Healthcare -95% 30 -80% 131

Microcap+ (>50M) Biotech Belowcash 57
61 as of 2022.01.10
74 (2022.01.20)
77 (2022.02.17)
110 (2022.06.26)
98 (2022.10.30)
106(2023.03.08)
77(2023.06.20)
113(2023.10.18)
72(2024.02.18)
74(2024.06.17)
72(2024.07.22)
69(2024.08.19)
67(2024.09.20)
76(2024.10.23)
81(2024.11.18)

RFK Jr. HHS pick sends shockwave to biotech industry

Two late-breaking poster at AASLD 11.15-19
AB-101 part 3 28-dosing expect data 1H25
Moderna trial set 09/24/2025 (less than a year)
Pfizer/BNTX construct hearing 12/18/2024 (Moderna hearing 02/08/2024)
did not issue any ATM shares this quarter. Selling pressure gone.

Healthcare 37; Y+500% 11

181 <=1; 319 <=2 (674 Biotech); price 293 <=3

173 <=1; 311 <=2 (676 Biotech); price 301 <=3

Healthcare 19; Y+500% 7
SMMT
SLNO
WGS
CRBP
TIL in-licensed PDL1xVEGF bispecific from 宜明昂科
CADL viral immunotherapies for solid tumors ADV, HSV
RNA antibody RNAi conjugate

Healthcare -95% 34 -80% 139

24% yoy Q2  record 70.2M Heplisav sales. Reaffirms FY guidance. Market share back to 42%. TDap extension results 24Q4.

cut research to focus on clinical development, reduce staff by 40%. Stock only quick spike dip at open, then recovered. lean and mean. More confidence in clinic, no potential in the pipeline.

Healthcare -95% 32 -80% 154

AB-101 SAD OK. MAD 2H24. AB729+Nivo+VTP-300 results 2H24

P/C level has strong correlation with XBI/FBIOX indices level, but not IBB. Divergence of IBB/XBI indicates reversal.

Healthcare -95% 31 -80% 155

10% increase Q1 sales, market share 41% increase from Q1Y23, but down from Q4 42%. Growth slows. Z-1018 IND clears.  Tdap to do duration Ph1 extension before Ph2. Market soft due to extended cold cough and flu season. Will recover.

FDA to hold AdCom on Alzheimer’s drug donanemab

FDA official maintains that the accelerated approval for Sarepta’s Duchenne therapy last year was “exceptional” rather than typical. Internal documents released earlier this year shed light on both the review process and the influence of patient advocates on Marks’ decision.

Endpts feature article: Once a scientific backwater, protein research attracts billion-dollar deals and high-profile publications

new CEO to build new empire with expected Humira revenue loss

Iovance Biotherapeutics won accelerated approval for Amtagvi, a cell therapy for patients with advanced melanoma. 1st cell therapy for solid tumor; 1st approval based on TILs.

weight loss candidate MariTide to contend with $LLY tirzepatide’s leading position

stop sales and development of Aduhelm, while better drug  Lecanemab (Leqembi) already in place.

189 <=1; 322 <=2 (701 Biotech); price 304 <=3

Healthcare 12; Y+500% 4
$SLNO
$AMAM
$EYPT pan-VEGF PDGF (excl. TIE2) TKI delivered via DURASERT IVT. ready for Ph3.
$CRBP Nectin-4 ADC

FDA adds box warning to six CART about "secondary cancer"

CG oncology, lead candidate, cretostimogene grenadenorepvec, is a targeted oncolytic intravesically delivered immunotherapy agent that is currently in two phase three trials for the treatment of BCG unresponsive and intermediate risk NMIBC disease. It is also in Phase 2 study in combination with pembrolizumab in the same indication.

Autoimmune CAR-T

owns 32.8% of LNP Patent value. 20%+16% of Genevant (80%)

buys Calypso Biotech and its autoimmune “pipeline-in-a-drug.”

Ambrx ADC buyout by $JNJ, slightly above 2021 IPO

After leading research at Amgen and Merck, Roger Perlmutter’s third act is lighting up the biological dark with superpowered microscopes.

lost 99% from 2018 peak, 6 years

Vaccitech now Barinthus Biotherapeutics, published data at AASLD, 1. VTP-300 + Opdivo low dose works best with HBsAg < 200U/ml, now only recruit such patients. 2. +AB-729 combo show robust promise. VTP-300 cohort stable after off-treatment, more IFNg response

$AZN buyout Icosavax for $838M upfront  and its Ph3-ready bivalent vaccine candidate against both respiratory syncytial virus and human metapneumovirus (hMPV) $GSK $PFE

The drugmaker’s attempt to improve on Novartis’ Piqray and deliver a first-line PI3K inhibitor has yielded an improved progression-free survival (PFS) in phase 3, teeing up talks with regulatory authorities.

The investigational drug, inavolisib, hits the same target as Piqray. What differentiates the molecule, in Roche’s view, is significantly better potency and selectivity against other PI3K isoforms.

buying CDK2 inhibtor from Ensem Therapeutics to compete with Bayer, Blueprint Medicines, Incyte and Pfizer #deepkinase

repotrectinib (Augtyro) acquired from Turning Point buyout, now approved by FDA  to treat non-small cell lung cancer (NSCLC) patients whose tumors have ROS1 oncogenic fusions #deepkinase

Kronos but stuff after positive CDK9 inhibitor data to ensure enough fund for clinical development #deepkinase

$CRMO buyout in IPO queue, bigpharma racing into GLP-1

$NVS slimmer pipeline after Sandoz spinoff.
$LLY oral GLP-1, why he sees a “slower ramp” for Alzheimer’s and how the company is looking at deals on ‘underappreciated’ assets;
$GSK “very busy” business development scene
$JNJ 20 new drugs in cancer, immunology and other areas
$SNY pitched 12 blockbusters

altimmune GLP-1 see turnaround with new data after previous year failure

buy out by $ABBV for $10.1B, for its ELAHERE® (mirvetuximab soravtansine-gynx) first-in-class ADC targeting folate receptor alpha (FRα) with a maytansinoid payload DM4. IMGN-529 anti-CD37-ADC also ImmunoGen

"inferior efficacy" next-gen blood thinner asundexian; recall a lot of its solid tumor drug Vitrakvi due to microbial contamination; contend with legal losses in cases relating to its weedkiller. Plan to focus on specialty drugs, lower costs and fewer big bets.

Carmo IPO, GLP-1 assets

$CRSP and Vertex's CRISPR therapy for sickle cell disease and transfusion-dependent beta-thalassemia approved by MHRA, soon by FDA.
$BLUE stock tanked due to competition

Bayer to cut management structure and non-medicine business. Voluntarily withdraw Aliqopa (Copanlisib, pan-PI3K inhibitor) for R/R Folicular Lymphoma

CEO retire, cut 24% stuff, Imdusiran +NA+VT300 data ok

deal with $AZN

$GSK buy JNJ-3989 (ARO-HBV) despite its own Bepirovirsen ASO.

report Q3 financials, 66% yoy Q3 Heplisav sales, raise guidance again $210-220M (+67-75%),  reassures positive cashflow,  market share increased to 41% (39% prior Q). Pipeline continues:  TDP, Shingles, plaque, seasonal and universal influenza

ADC come back, $ESMO23, a combination of Seagen and Astellas Pharma’s Padcev with Merck’s Keytruda — which earned a standing ovation for changing standard-of-care in first-line bladder cancer — to AstraZeneca and Daiichi Sankyo’s TROP2 candidate to new combination approaches.
$AMGN lay off 350 after Horizon deal; Idorsia lay off 300 cost reduction and revamp.

 agreed to acquire an antibody-drug conjugate biotech, swooping in for France-based Mablink Bioscience.

264 <=1; 404 <=2 (714 Biotech); price 383 <=3

Healthcare 9; Y+500% 4
$SLNO: Diazoxide Choline for Prader-Willi Syndrome met Primary Endpoint. #RareDisease but generic drug, commercial failure?
$CABA: obtained Autoimmune (SLE etc.) CD19-CAR-T rights and development plan in 2022.10
$AMAM: ADC story (anti-HER2 ADC, anti-PSMA ADC)
$MLTX: nanobodies anti-IL17A/F

after FDA rejection of ONpattro indication expansion ATTR cardiomyopathy, give up on the indication for Onpattro, focusing instead on newer drugs.

buyout $MRTX Mirati for KRAS and PRMT5 assets $AMGN rivalry

UniQure layoffs as gene therapy players adapt
Less than a year after winning its first gene therapy approval — for Hemgenix in hemophilia B — uniQure is undergoing a broad overhaul that includes laying off 20% of its staff, saying goodbye to its CSO and cutting its research and technology projects by half. Elsewhere in the gene therapy world, retinal-focused Atsena Therapeutics also reduced its staff as it reels in more cash from existing investors and looks for a partner. Orchard Therapeutics $ORTX, meanwhile, found a new home as Japan’s Kyowa Kirin bought out the biotech for $387.4 million in cash.

1.4B buyout $PNT point biopharma, a radiopharma company with two cancer therapies in Phase III
its GLP-1 agonist tirzepatide nearly 70% of patients had an adverse event.

Google DeepMind Demis Hassabis AI for Bio

shaking up the way it does research, creating three unique teams — each with their own leaders — to zero in on vaccines and infectious diseases, respiratory and immunology, and oncology, and steer programs all the way to Phase II before the development group takes over. John Lepore, head of research, will be moving on.

RayzeBio IPO radiopharmaceutical therapy (RPT) similar to ADC

FDA cleared updated booster from $PFE and $MRNA. $NVAX left out

2021 IPO. slashing headcount by 40% — or 176 roles — and capping spending on some CAR-T programs to focus on approved BCMA therapy, Abecma. Sales of Abecma to decrease due to manufacture problem

clinical trial investigators see the potential of semaglutide and other GLP-1 agonists in treating NASH

bought another obesity-focused biotech to buy. Copenhagen-based Embark Biotech

Ipsen FDA approval Sohonos (palovarotene) in fibrodysplasia ossificans progressiva despite concerns about its efficacy and safety profile.  Previously Palovarotene received its first approval on 21 January 2022 to reduce the formation of heterotopic ossification (HO)  in adults and children aged 8 years and above for females and 10 years and above for males with FOP in Canada.

orally bioavailable selective retinoic acid receptor (RAR)γ agonist being developed by Ipsen for the reduction of heterotopic ossification (HO) formation in patients with fibrodysplasia ossificans progressiva (FOP). By binding to RARγ, palovarotene inhibits bone morphogenetic protein and SMAD 1/5/8 signalling: interfering with these pathways prevents chondrogenesis and ultimately HO by permitting normal muscle tissue repair or regeneration to occur.

 Pozelimab anti-C5 complement inhibitor FDA approval, recombinant monoclonal antibody used for CD55-deficient protein-losing enteropathy, (CHAPLE disease)

cut sales, executive departure, SEC probe

semaglutide reduce CV events by 20%. acquiring Inversago Pharma and its suite of drugs targeting cannabinoid receptors, including a lead candidate for weight loss.

report Q2 financials, 73% yoy Q2 Heplisav sales, raise guidance $200-215M (+59-71%), reassures positive cashflow, market share increased to 39% (37% prior Q). Pipeline continues.

Roche buy Zilebesiran from $ALNY (pronounced “zile-BEE-siran”) is an  subcutaneously RNA interference (RNAi) therapeutic targeting liver-expressed angiotensinogen (AGT) in development for the treatment of hypertension for $310M upfront and upto $2.8B

Novartis is back on siRNA with Novartis has signed a $1 billion deal to acquire DTx Pharma - DTx Pharma shareholders will receive $500 million upfront and up to $500 million in milestones.  leveraging its proprietary fatty acid ligand-conjugated oligonucleotide (FALCON) platform. Deal includes DTx-1252, a potential therapy for the neuromuscular disorder Charcot-Marie-Tooth disease type 1A (CMT1A), and two additional preclinical programs for other neuroscience indications

Eli Lilly announced it submitted its experimental Alzheimer’s drug donanemab to the FDA after unveiling detailed data

Apogee Therapeutics, half life extended antibodies to target IL13 IL-4Ra OX-40L for AD, COPD, Asthma. IPO raise 300M

to buyout private Versanis for $1.9B, for its only asset Bimagrumab (anti-ActRII) bimagrumab (BYM338)是一种与激活素II型受体(activitin type II receptor, ActRII)结合的全人单克隆抗体，可以阻止天然配体的作用，从而负调节骨骼肌生长。

尽管此前bimagrumab已经获得了美国FDA授予的突破性药物认证，2016年4月诺华宣布bimagrumab用于治疗散发性包涵体肌炎在一项临床IIb/III期研究中未能达到主要终点，终止了该药物临床。

直到2021年1月13日，一篇发表在JAMA Network Open上的一篇研究确定了bimagrumab对超重或肥胖及2型糖尿病成年患者的身体和血糖控制的有效性和安全性，bimagrumab的新适应症扩展才初见眉目。

CEO warns against AI, https://endpts.com/how-will-ai-change-drug-development-schrodinger-ceo-warns-of-hype/
'The more overhype, the harder you fall': Schrödinger CEO warns on AI craze

... “Nobody in the field is actually using AI,” Schrödinger CEO Ramy Farid said in an interview at the company’s New York headquarters overlooking Times Square. “That’s not what they mean. It’s machine learning. To me, describing ourselves as a machine-learning company would be like describing ourselves as a company that uses Microsoft Office.” ...
... “They changed the name （QSAR定量构效关系） to machine learning. That sounds better,” Farid said. “There’s no difference. Then it was called deep learning, and that got people really excited. And then artificial intelligence.” ...
...But he’s also nervous about what’s to come in the near term: The AI hype will inevitably come back down to earth....
...“Those companies that stuck ‘dot AI’ in their name, it’s going to seem kind of foolish,” Farid said...

Generate:Biomedicines, a flagship company, using AI to design de novo protein drugs

Roctavian, the first gene therapy to treat the genetic bleeding disorder hemophilia A, finally FDA approved. $2.9M dose

type I diabetes cell therapy promising data from Vertex and Sernova, building manufacture capacity with lonza.
FDA also approved a first-of-its-kind cell therapy, which takes pancreatic cells from deceased donors, for type 1 diabetes from Chicago biotech CellTrans.
Eli Lilly announced it will buy its partner in the space, Sigilon Therapeutics, with $34.6 million upfront and much more in milestones.

obeticholic acid (OCA) for NASH received 2nd CRL.

196 <=1; 334 <=2 (728 Biotech); price 347 <=3

Healthcare 18; Y+500% 8
$CABA
$VKTX
$RXDX
$ARDX
$TGTX
$AKRO
$ETNB
$AVDL

Greg Verdine, personal story, drug the undruggable. “God didn’t make it undruggable,” he said. “It’s going to be biology, unmet medical need, that decides what actually gets drugged, and not people’s notions.”
... In another world, Greg Verdine would still be building boats and tearing apart car engines in his New Jersey front yard.

CDK4/6 inhibitor Kisqali reduced relapse rate by 25% from 12.9% to 9.6%  in patients with Stage II or III HR-positive, HER2-negative breast cancer.

Rain oncology (2021 IPO), MDM2 is a key negative regulator of p53 responsible for controlling and limiting p53 levels. If MDM2 levels are high, p53 levels may be significantly suppressed, aiding in tumor growth. Milademetan may reactivate p53, restoring the protection it provides. Laying off 65% workers.

Eikon Therapeutics super-resolution microscopy, Single Molecule Tracking (SMT) across the drug discovery process. 2019 founding, having raised nearly $775 million, Nobel-prize winning technology (2014 Chemistry Prize)

Ironwood buy vectivbio (2021 IPO)  with a focus on a Phase III program for the rare condition known as short bowel syndrome.  Apraglutide is an investigational drug being developed as a once-weekly treatment for patients who have SBS with intestinal failure (SBS-IF). It is a next-generation, long-acting, synthetic GLP-2 analog that acts as a selective, full agonist of the GLP-2 receptor.

cuts PI3Kd and AXL/MER programs due to regulatory landscape and poor early efficacy, respectively

sector downturn caused, In the last 16 months, drugmakers have canceled or paused development on 26 rare disease programs that were financially supported by patient groups.

$AMGN $GMAB CD3xCD20 bispecific epcoritamab Epkinly approved for relapsed/refractory large B cell lymphoma.

OCA for NASH AdCom voted against accelerated approval

FDA AdCom 8-6 on its new DMD gene therapy

$OPHT rebranded to IVERIC BIO, now buyout by Astellas for $5.9B

Q1 ER/CC: 1. AB-729 1.5 years off-treatment data, IFN combo to release 2Q23, VTP-300 in 2H23, opdivo+VTP-300 to be dosed 2Q
2. AB-161 dosed in March, data due 2H23
3. AB-101 on hold
4. AB-343 Ph1 initiate 2H23
5. ATM raise 19.9M, 165.1M shares+19.7M options

develop its own weight loss drug tirzepatide Mounjaro against Nova Nordisk's $NVO Wegovy semaglutide

10.8B buyout by Merck. $MRK founded in 1995. Almost 30 years of accumulation on IBD. 2019 BD with Takeda to get several pre-clinical pipeline, with self-developed companion diagnostics, then had clinical success. 
不同于一般靠做创新药研发起家的Biotech，Prometheus Laboratories一开始专注于胃肠道、自身免疫炎症性疾病和病症精准诊断和检测试剂和工具的开发，并很早就有了正向的现金流，而非像一般Biotech需要烧钱度日。
在McKenna治理下，Prometheus Biosciences完成了了多个BD项目。其中最引入注目的莫过于与武田之间数亿美元的BD交易，借助于此BD交易，Prometheus Biosciences拿下了多个处于临床前的IBD创新药项目。
检测理解疾病+BD临床管线+自身开发伴随诊断 =成功精准医疗
链接是Prometheus上市时的材料, 特别是涉及与武田交易的资料在113-115页和F17页
RXDX Rx是药 Dx是诊断 在一起就是药伴随诊断
https://www.insideprecisionmedicine.com/topics/molecular-dx-topic/takeda-to-use-prometheus-bioinformatics-platform-cdx-tools-in-ibd-drug-diagnostic-partnership/

Novo Nordisk team up with Canada’s Aspect Biosystems around four cell therapy candidates for diabetes

$170M upfront and $1.5B milestones (biobuck haha) pact with a Shanghai biotech named Duality Biologics 映恩生物 . The deal focuses on two ADCs, including one targeting HER2-expressing solid tumors.

partnership with Regeneron for CARTreg

Schoizophrenia drug another Ph3 success, parent company puretech sold its part of Royalty to Royalty Pharma for $100M cash and $400M milestones.

ALS drug tofersen to be conditionally approved based on surrogate biomarker neurofilament despite failed PH3 trial

report Q1 financials, 109% yoy Q1 Heplisav sales, reaffirms $165-185M sales (30-47% growth), expects positive cash flow, market share increased to 37% (35% prior Q). Pipeline: TDP, Shingles, plaque, univesal influenza, new Queensland poly T cell eipitope proteins

$40M upfront and $36M equity with $MRNA to develop novel nucleic acid therapeutics

Ainos, the feasibility of Merdury’s potential investment and participation in the completion of a Phase 3 trial of VELDONA drug candidate for the treatment of oral warts in HIV-seropositive patients.

AB-161 , RNA destablizer dose first subject

present AB-343 mpro COVID preclinical data at ICAR

242 <=1; 365 <=2 (737 Biotech); price 361 <=3

Healthcare 3; Y+500% 0
CABA CART for autoimmunity
TMDX  organ transplantation
ARDX Tenapanor first-in-class inhibit the sodium/hydrogen exchanger 3 (NHE3) for hyperphosphatemia, a condition of elevated levels of phosphate in the blood, common among patients with chronic kidney disease on dialysis.

Ph2 #failure NYX-458 in patients with cognitive impairment associated with Parkinson’s disease and dementia with Lewy bodies

announce zotatifin (eIF4A inhibitor) for COVID data

ARX517 anti-PSMA ADC for prostate cancer, reports Ph1 data

report preclinical data REVTx-300 in a model of acute and chronic kidney disease. Data reported a significant reduction in circulating transforming growth factor-β in a dose dependent manner relative to the positive control group.

Biophytis Sarconeos (BIO101) activates the MAS receptor in muscle cells, a key component of the Renin-Angiotensin System (RAS) repurposed for COVID-19

MyoVista Wavelet Technology was granted a patent in Korea by the Korean Intellectual Property Office, better than traditional ECG.

Endogenous Metabolic Modulators (EMMs) AXA-1125 EMM composition of six amino acids and derivatives, for LONG COVID fatigue and NASH. hype?

流式细胞肺癌早期诊断LDT产品 CyPath Lung 用痰液, 针对影像发现20mm以下小结节患者。92% sensitivity, 87% specificity

abandon ANG-3070 TKI for chronic fibrotic diseases kidney lung,  targets DDR1/2 and PDGFRα/β, reverse merger, gave up everything. #deepkinase #safety an unexpected and substantial decline in kidney function

released briefing documents for its upcoming advisory committee meeting evaluating Rezafungin (CD101) as a treatment for Candidemia. noted “consistent efficacy and safety findings” and a “favorable benefit-risk profile.” -43% still. novel, once-weekly antifungal

data from Alzheimer biomarker study show a novel biomarker TDP-43

acquires and change its name to Aceragen, abandons TLR9 CpG agonist development $ACGN

Tumor metabolism. Now liquidated.

Cincor 2022.01 IPO, buyout by $AZN, cardio-renal diseases, such as hypertension

submit EUA application to the FDA for over-the-counter (OTC) use of its molecular COVID-19 & Flu test.

Probody platform, protease cleavable masked antibody/cytokines to target protease rich tumor microenvironment. Announced deal with Moderna to see if mRNA can be similarly masked

2023 CpG revenue minimal, 2022 HeplisavB sales may not cover expense. If 100%+ sales again, maybe.
Shingles vaccine data OK. To initiate Dynavax manufactured gE vaccine trial

announce 2023 corporate objectives:
AB-729 four data: off-treatment follow up, +IFN, +VT300, +Nivonumab
AB161 RNA destabilizer, AB101 PD-L1: Ph1 data
AB343 Mpro initiate Ph1
Nsp12: nominate

Impact Biomedicines, John Hood, fedratinib, sold to Sanofi, failed with Wernicke’s encephalopathy, revive, turn around, sold again to $CELG for $7B, patient appeal stories, #kinase #deepkinase San Diego 2017 2018 2019

Ripretinib Qinlock phase 3 failure, revive hope with subgroup analysis

FDA Fast Track FTD to EB05 anti-TLR4 for acute respiratory distress syndrome (ARDS) +115%. Other pipeline: sPLA2 (Secreted phospholipases A2) inhibitor for anti-inflammation

IsoPlexis SingleCell Proteomics example company 2021.10 IPO $11+ now $1. All stock merger with Berkeley Lights ($BLI). NewCo called PhenomeX, $CELL.
$BLI cell-based therapy development product. IPOed in 2020.07 price at $22, skyrocketed first  day to close at $65.45, peak closed at $106, now at $2-3. Totally hype.

success plinabulin (blocks the polymerization of tubulin in a unique manner) to treat docetaxel-induced neutropenia (DIN) in non-small cell lung cancer (NSCLC) patients and breast cancer.

Ph1b THB001 (KIT inhibitor)for chronic inducible urticaria stopped after two subjects experienced asymptomatic liver transaminitis.  focus on mast cell mediated inflammatory dermatology diseases. $CLDX anti-KIT mAb, same indications.

THB-001 terminated due to liver tox. Looking for other WT KIT inhibitors and several candidates going through preclinical studies.

Novartis granted Third Harmonic a license to three compounds, including THB001, in 2019 in return for $0.4 million upfront, a stake in the biotech, up to $31.7 million in development milestones and up to $200 million in commercial paydays. $155M IPO.

issue 125M debt + 125M stock + 15 % 30 day option for additional, now well below $DVAX

terminates GS-3583 FLT3 inhibitor in solid tumors.

+NA+IFN Ph2a lead-in phase data release as expected, complete enrollment, IFN phase data release in 1H2023.

Prometheus Biosciences reported twp Ph2 PRA023 trials positive data. offer $500M. +177%
PRA023 anti-TL1A cytokine for intestine inflammation and fibrosis, for Ulcerative Colitis, Crohn’s Disease, and Systemic Sclerosis-associated Interstitial Lung Disease (SSc-ILD), along with companion diagnostic products designed to help predict responses to identify the right patients for PRA023, with the goal of ensuring maximal response. 

Pharvaris has a platform of B2-receptor antagonist small molecules for bradykinin-mediated diseases. to treat all sub-types of hereditary angioedema (HAE). Ph2 success +339%

$abus chronology
2011.3 Tekmira sues Alnylam
2011.4 Alnylam has filed its answer
2011.10 Discovery is underway. Tekmira has requested a trial date be set for the fall of 2012
2012.11 settlement agreement. As a result of that, Tekmira will receive $65 million within 10days

REM-001 is a second-generation photodynamic therapy (“PDT”) photosensitizer agent which has undergone late stage clinical development in previous clinical trials showing a complete response in approximately 80% of evaluable tumor sites.
FDA Fast Track

acquire $IMGO, LSD1 inhibitor IMG-7289 Bomedemstat, Ph2 for Myelofibrosis, Thrombocythemia

4D Molecular Therapeutics, gene therapy vector evolution 37 AAV out of 1B. We believe our customized vectors have the potential to enable targeted delivery, efficient transduction, reduced inflammation and resistance to pre-existing antibodies.

interim data Ph1/2 intravitreal 4D-150 for wet AMD. annualized anti-VEGF injection rate was reduced by 96.7% and 80% of patients have remained anti-VEGF injection-free.

Luxeptinib (CG-806, panFLT3/BTK) to treat Acute myeloid leukemia (AML). Ph1b

ADP-A2M4CD8 for solid tumors Ph1 ORR improve from 44% to 52%, MAGE-A4 T cell therapy

buyout $AMZN $3.7B

GSK ASO Ph3 to commence in 1H23, 9-10% HBsAg LLoQ NEJM. ARO-HBV no news

$JNJ buyout $16B

gene therapy hearing company buyout by Eli Lily

Court rejects Moderna motion to dismiss "sueing the wrong  party"

AB-729 9 patient off all drugs, no relapse 12-44 weeks; AB-101 more preclinical data; core inhibitor no additional effect; AB-836 discontinue.

 “We believe AB-729 is the only RNAi therapeutic in development for hepatitis B virus that has clinically shown its ability to suppress HBV DNA, reduce HBsAg and immunologically control HBV – three pillars that we consider to be key to developing a functional cure for HBV.”

Healthcare -95% 33 -80% 289

ESSA Pharma EPI-8207 (ARV-110) ANITAC (PROTAC) Ph1/2 data anti-tumor activity in six patients enrolled, with four of six of these patients achieving a PSA90 by 12 weeks of dosing and five of six patients to date have achieved a PSA90. Preclinical data noted robust potency degrading AR, including AR splice variants and clinically relevant AR mutants that can potentially drive disease progression.

deal w. Forma Therapeutics ($FMTX) olutasidenib (IDH1 inhibitor) for R/R AML

to merge with $ENLV, trade under $ENLV

Ph3 #failure REL-1017 to treat Major Depressive Disorder (MDD).

FDA award an accelerated approval for INBRX-101  for alpha-1 antitrypsin deficiency (AATD).

mavorixafor anti-CXCR4 to treat people with idiopathic, cyclic, or congenital chronic neutropenia (CN). 100% of participants reported a robust response and achieved normalized neutrophil counts.

Ph1 PGN-EDO51 #success to treat Duchenne Muscular Dystrophy. exon skipping therapy bound to a peptide to help the therapy reach the muscle cells. PGN-EDO51 targets those amenable to exon 51 skipping.

awarded patents covering its radiation sensitizing HDAC inhibitor technology platform in the US and Hong Kong. EU pending.

AL102 gamma-secretase inhibitor Positive Interim Data Ph2/3 in Desmoid Tumors, little price move

Vimseltinib (switch-control kinase inhibitor of CSF1R) positive Ph1/2 results in Tenosynovial Giant Cell Tumour (TGCT)
Phase 1 Single Agent Dose Escalation Data for First-in-Class switch-control inhibitor ULK1/2 Inhibitor of Autophagy, DCC-3116, positive results. to combine with MEK, KRAS inhibitors

Foundation Medicine and Relay Therapeutics Collaborate to Develop FoundationOne®CDx as a Companion Diagnostic for Relay's Investigational FGFR2 Inhibitor, RLY-4008
88% overall response rate (15 out of 17) from interim data of pan-FGFR treatment (FGFRi)-naïve FGFR2-fusion cholangiocarcinoma (CCA) patients treated at the pivotal dose

sold Cardurion Pharmaceuticals Tovinontrine and other assets of the PDE9 program for ~$34.75M upfront + $60M in milestones.

#AI Evaxion Biotech grant and collaboration with UMass Chan Medical School to develop a gonorrhea lead vaccine candidate

Exscientia anounced its 3 year, €240 million deal with Bayer AG to develop Artificial Intelligence (AI)-designed compounds to potentially treat pain, cardiovascular diseases and cancer was mutually terminated.  Call it outdated. $100M deal with $SNY still underway

CDO leaving YE

Virios IMC-1 antiviral for  fibromyalgia did not meet its primary endpoint.

$GSK exclusive license agreement to commercialize Spero’s antibiotic for complicated urinary tract infections (cUTI) tebipenem HBr in all territories, except Japan and certain other Asian countries. $66M upfront +  $9 million equity

 released data Aquadex FlexFlow System. Aquadex FlexFlow is a medical device to treat hypervolemia (fluid overload) that had a 100% 30-day survival rate.

removed from NASDAQ

join monkeypox hype

policy change to allow OTC hearing aids sale

Ph3 ensifentrine RPL554 for COPD success +94%

buyout rumor

Ph3 Patisiran for transthyretin-mediated (ATTR) amyloidosis with cardiomyopathy success +54%

buyout by $AMGN $3.7B, less than 2021 peak

not losing any order yet cut guidance by half

to pursue EUA vilobelimab anti-C5a for critically ill COVID-19 patients in 3Q2022.

FDA identified deficiencies in NDA linzagolix for uterine fibroids. CSO and head R&D resigns.

 previous Y+500%. Ph3 #failure PH94B for the acute treatment of anxiety with social anxiety disorder (SAD) not seasonal affective disorder. continue PH94B's development in the ongoing Ph3 and Ph2 trials in adjustment disorder with anxiety. PH94B is an odorless nasal spray, synthetic investigational neurosteroid developed from proprietary compounds called pherines.

Ofranergene obadenovec, VB-111, Ph3 failure to treat platinum-resistant ovarian cancer. is an anti-angiogenic gene therapy. ongoing Ph2 trials in metastatic colorectal cancer and recurrent glioblastoma multiforme

The vector is a non-replicating adenovirus 5. The payload is a chimeric gene encoding a fusion protein that combines the extracellular and intramembrane domains of the human TNF receptor 1 and the intracellular domain of the Fas receptor, under the control of a modified version of the murine promoter for endothelin-1, which is called PPE-1. PPE-1 drives tissues specific expression and also has a hypoxia responsive element. The FasR part of the payload is intended to drive cell death in the endothelium of blood vessels where the chimeric protein is expressed; the TNF-R1 part is intended to provide some tumor specificity.[1]

Ph2 setmelanotide to treat Hypothalamic Obesity. 17.2 reduction in BMI, a 15.8% mean change in body weight and a 35 lbs mean weight loss. +110%

Contrafect. Exebacase (CF-301) is a lysin with potent activity against Staphylococcus aureus ("Staph aureus"). DSMB recommend stop Ph3 for futility

DiaMedica Ph2/3 DM199 to treat acute ischemic stroke (AIS) report three serious adverse events of transient hypotension.

announce a new blood sensor, HYLA, to monitor in real time patient conditions

min Mcap 2.19B, medium 4.71B
$MRNA #3 $BNTX #4 $NVAX #25 $CVAC #56
$CRSP #23 $NTLA #31 $BEAM #41
$MRVI holding company for Trilink, Glen and Cygnus
$APLS IPO to top60, Pegcetacoplan, Pegylated C3 inhibitor (11-AA peptide)
$DNA Ginkgo Bioworks
$CERE neurology
$CYTK muscle company doubled in last year
$TPTX kinase buyout
$KRTX M1-M4 muscarinic agonist, neurology
$HRMY Pitolisant is a histamine-3 receptor (H3R) antagonist/inverse agonist, Narcolepsy
$ABCM AbCam 2020 IPO, down less then market
$ABCL AbCellera 2020 IPO, COVID ab, down less
$PTCT SMA
$FOLD chaperone
$ROIV
$CORT Corcept has focused on the adverse effects of excess cortisol
$RCUS anti-TIGIT Wang Ning
$EQRX low price me too drugs

Ph1b DB-020 to prevent hearing loss in cancer patients release data. 87% of 17 were partially or completely protected from ototoxicity. +75% down 90% since IPO

Healthcare -95% 27 -80% 294

traded higher following the US Supreme Court decision to overturn Roe v. Wade on Friday. Evofem’s PHEXXI (Amphora), a contraceptive vaginal gel, was approved in 2020. $AGRX no move

Athira Ph2 fosgonimeton (ATH-1017) for Alzheimer's  #failure. hepatocyte growth factor receptor (c-Met/HGFR) agonist. #DeepKinase

AstraZeneca $AZN buyout rumor

to launch Inbrija in Germany

Ph3 XHANCE (liquid Exhalation Delivery System (EDS) fluticasone propionate )  for chronic sinusitis met both co-primary endpoints. 

FDA rebutted proposed outcome measures planned Phase 2b trial of adalimumab to treat Dupuytren's disease. "did not meet the requirement for the demonstration of efficacy in registrational studies".

EASL 2022.  1) AB-729 out of 32 patients, 11 eligible to stop NA 34%; (out of 5 enough follow-up, 100% no relapse DNA or HBsAg, 5/5). 2) AB-836 data disappointing (IP-10 (exploratory core inhibitor tox biomarker) and ALT/AST), yet collaboration with $ASMB should still make the POC of siRNA + Core Inhibitor. 3) PD-1L preclinical data success.

FTX-6058 for sickle cell disease (SCD) reports positive data, 3/6 not evaluated due to being non-adherent or discontinued due to protocol deviation. -41%

Ph3 #failure fostamatinib for warm autoimmune hemolytic anemia (wAIHA). fosta SYK inhibitor already approved. -62%

Bezuclastinib (formerly PLX 9486) is a highly potent small molecule selective inhibitor of KIT D816V and other mutations in KIT exon 17. Ph2 data 100% patients ≥50% reduction in serum tryptase levels. +77%.

two FDA AdCom positive recommendations for BLAs betibeglogene autotemcel (beti-cel, β-globin for β-thalassemia) and elivaldogene autotemcel (eli-cel, ABCD1, for early active CALD, a rare neurodegenerative disease). was $116 in 2018 now $3 (after 1:2 split)

license camonsertib (RP-3500, Serine/threonine-protein kinase Ataxia telangiectasia and Rad3 related ATR inhibitor ) to Roche $RHHBY $125M + up to 1.2B. 2020 IPO

$4B buyout by $BMY TKI #DeepKinase ROS1, MET, ALK

180 Life Science Corp regulatory updates from UK MHRA and FDA. FDA will provide written response to Ph2b data and type C meeting request. anti-TNF for Dupuytren’s Disease. Founders are Remicade infliximab pioneers

CEO planned transition

Ph2 data adagrasib (KRAS G12C) for NSCLC. adagrasib spurred slightly more responses and led to a longer overall survival than Amgen Lumakras, duration appears shorter and the safety profile continues to spark concern. -37%

 C-144-01 Ph3 trial  for Lifileucel/LN-144 for melanoma (post anti-PD-1) cohort 4 data worse than cohort 2 (ORR 29% vs 35), DOR 10m. #TIL

zilovertamab (prev. Cirmtuzumab) release ASCO abstract Ph1/2 combo with ibrutinib in mantle cell lymphoma (MCL) or chronic lymphocytic leukemia (CLL). ORR 85% CR 41%

FDA 501(k) clearance for Renuvion Dermal Handpiece for specific dermal resurfacing procedures.

reports Sentinel Lymphnode (SLN+) data for surgery exercised melanoma, trial stop early, will move on to 5y and 10y PFS and OS. Very long horizon.

preclinical COVID-19 antibody cocktail IMM-BCP-01 data showing neutralizing Omicron in hampster models

monkeypox speculations make vaccine stocks trade higher, small caps $SIGA $GOVX $EBS $CMRX $NNVC

FDA reduced REMS requirements, both for audit site number and frequency

expanded license agreement with St. Jude Children's Research Hospital to design genetic constructs to express bacterial proteins on the surface of Streptococcus pneumoniae.

Ph2a PRAX-944 to treat essential tremor (ET) positive results. Antiepileptic drug T type calcium channel antagonist. "Genetic NeuroScience"

FDA lifted hold on IND for LB-001 Ph1/2 trial for methylmalonic acidemia.

-11% $MRNA dismiss patent infringement, hook on US government

revenue declined from $141M to $94M.

Connect Biopharma, CBP-307 for ulcerative colitis (UC) fail Ph2.
2021 IPO, 康乃德生物医药，China company. $18.5 IPO, $30 peak, now $0.61. Pipeline: CBP-201 anti-IL4R (Th2 mod), CBP-307 S1P1 inhibitor, Th1 mod., CBP-174, peripheral H3 Receptor antagonist

2017 IPO, peak 2020.12 $22, now $2. FDA find deficiencies in NDA of tebipenem to treat complicated urinary tract infections (cUTI)。 a novel oral formulation of tebipenem, a carbapenem-class antibiotic marketed in Japan since 2009 for specific pediatric infections.
Antibiotics is hard.

Acuitas sued Arbutus to avoid patent dispute, futile move

Hu MeiMei, previous COVAXX, now COVID-19 vaccine in Ph3 (multitope), multiple pipeline, endobody, another theranos E. Holmes? or another Vivek?

2021.06 IPO, genomic defined cancer. NRG1 fusion lead. to announce data at ASCO

$NVS announced ensovibep EUA require additional data

Offers $45M. Ibrexafungerp (formerly SCY-078) is the first representative of a novel class of structurally-distinct glucan synthase inhibitors, triterpenoids. It has shown activity against Aspergillus and Candida, including against multidrug-resistant (MDR) species Candida auris as well as Pneumocystis. Ibrexafungerp combines the well-established activity of glucan synthase inhibitors with the flexibility of oral and IV formulations, offering a broad use across different settings (in-patient and out-patient).

NY Times article covering the retractions of data for simufilam, to treat Alzheimer disease.

nano cap. Hype on Jatenzo (testosterone undecanoate, T therapy) data release speculation at Androgen Society's annual meeting

lost 90% since peak. After partial clinical hold, now voluntarily withdraw BLA and sNDA for CLL and SLL.

Mysterious Ampion is a novel biologic drug containing a blood-derived cyclized peptide and small molecules that target multiple pathways in the innate immune response characteristic of inflammatory disease.
FDA did not agree that AP-013 could serve as a second pivotal trial based on the change in the analysis population and pain only instead of the original prespecified co-primary endpoints.

Ph3 data sabizabulin 50% reduction COVID death. orally bioavailable bis-indole that binds to the “colchicine binding site” of a and b tubulin and inhibits tubulin polymerization at low nanomolar concentrations.

2019 IPO, kinase inhibitors with structural guide design so that it will not be affected by  solvent-front, gatekeeper or compound resistance mutations. #DeepKinase
Reported data for repotrectinib, 4CR, 52PR, ORR 79%

nano cap release data  showing its LB-100 evidence of DNA damage and mitotic stress. Announce $5.8M offering

$REGN buyout $250M, market cap $50M, 5X premier

failed Ph2b NYX-2925 to treat painful diabetic peripheral neuropathy (DPN)

biotechs recovering after hope for china-us regulator collaboration

revealed at a fireside chat that Ph3 has only 170 enrolled with a target of 1750.

IGALMI (dexmedetomidine, BXCL-501) sublingual film for agitation with schizophrenia or bipolar I or II disorder in adults. Still down. sell on news. Bad overall market.

2019 IPO, $SNY deal to develop IgM antibody agonists for $150M upfront on Oncology, Immunology, and Inflammation Targets. lost ~90% in 2021 biotech bear

Ph1 success Lomecel-B to treat mild Alzheimer’s disease (AD). 忽悠？

vadadustat (HIF prolyl-hydroxylase inhibitor, 马宇) for CKD anemia FDA CRL, see previous note for safety and competition concerns.

discontinue ORIC-101 (glucocorticoid receptor GR antagonist) after futile 2 Ph1b studies. 2020.04 IPO,  other pipeline CD73, EGFR/HER2 exon 20, PRC2, PLK4 inhibitors.

LB1148, oral liquid formulation of the well-characterized digestive enzyme inhibitor, tranexamic acid (“TXA”), to reduce abdominal adhesions and help restore bowel function following surgery. for administration prior to major surgeries that are at risk of disrupting the intestinal epithelial barrier. Reverse split: 1:6

PI3K delta (PI3Kδ) inhibitor zandelisib no longer submit NDA based on single arm Ph2 trial results. FDA requesting additional trial.

2021 revenue $86M up 33% from 2020

2021.03 IPO biomarker based cancer therapy, Hippo, RAS pathways, also AHR, EP4 pathways. 20M research revenue

REVTx-99 Ph2b for influenza completed enrollment. Intranasal formulation of synthetic TLR4 ligand PHAD.

FDA approved ZTALMY (ganaxolone) to treat seizures associated with CDKL5 deficiency disorder

NexImmune 2021.02 peak bio IPO, The AIM (Artificial Immune Modulation) technology construct “synthetic dendritic cells” (beads), AIM nanoparticles, directing a specific T cell-mediated immune response.

$NVS Novartis license $VYGR's AAV capsids for $54M upfront. +53%. $VYGR form a round bottom pattern.

Xipere approved in 2021.10, records 4Q21 revenue $26M, not bad. +39%

1. XBI指数已经跌去一半
2. 大量曾经的热门股跌去超80%
3. 一年以前两位朋友询问我要不要买ARKG，那时候正好ARKG见顶，当时ARKG占股超过10%的公司达到25家。很多业绩一般的测序股，ai股。如今ARKG已跌去64%。业绩一般却随ARKG那时候猛涨的PacBio Schrodinger都已经跌去80%
4. 价格低于3的生物公司超过250家，历史最高
5. Price/Cash低于2的生物公司超过300家，低于1的超过125，两个数据都是历史最高，已经持续在这位置三个月了
班群

-33% due to USPTO ruled in litigation regarding CRISPR/Cas9 editing patents that the patents in question belonged to The Broad Institute. $NTLA has no license. Dispute will continue despite latest ruling

2021.03 IPO was $22.5, now 6.6, microbiome CP101 (complete consortia for C. diff) versus enriched consortia and target consortia. FDA clinical hold on Ph3.

NuCana 2017 IPO, post IPO triple peak ~$30, now <$1. Protide tech: specific combinations of aryl, ester and amino acid groups (phosphoramidate motifs) that protect the activated nucleotide analog.
IDMC conclude Ph3 NUC-1031 futile for advanced biliary tract cancer. Acelarin is comprised of gemcitabine and a phosphoramidate,  can enter the cancer cell independently of nucleoside transporters, is protected from breakdown by deaminases, and, delivers the pre-activated form of gemcitabine.

YE21 ER: revenue up, market share up, still working on influenza in 10-K, both universal and seasonal.

EPS +264% comparing to 2020, +63% at $47, imaging diagnostics for prostate cancer, 20X comparing to 2016, 4X to 2020

Y2021 revenue up 28% to $137.9M,  +28%, was $116 2021.02, recent low $23. MCap now $2.3B, nromalized

Ph3 KSI-301 anti-VEGF ABC miss primary endpoints, not enough to stop vision worse. other trials on going. $164 in 2021.01, now $8. Baker Brothers had significant insider buying activity.

EU CHMP negative vote on arimoclomol for Niemann-Pick disease type C (NPC). Previously FDA CRL. -38% already low.

FDA approval pain med in 2020, now 1:35 reverse split, lost 98%

zuranolone (SAGE-217/BIIB125) release Ph3 data, met primary endpts. Day 3 effect not present at Day 15, durability issue. Was $190 in 2018/19

联拓生物 专做中国跨境医药市场。cross border and local markets for China. 吴人伟 as director，Perceptive Advisors managing director Konstantin Poukalov as CEO. 2021.11 IPO quite a few collaboration projects, including:
Infigratinib (FGFR1-3 inhibitor QEDTx; BridgeBio); BBP-398 (SHP2 inhibitor Navire; BridgeBio); TP-03 topical ophthalmic formulation of lotilaner for Demodex blepharitis
联拓生物成立两年火速纳斯达克上市，“买买买”模式如此吃香吗？ https://vcbeat.top/52932

FDA maintains clinical hold on its CTI-1601, previous hold on 2021.05

Healthcare -95% 7 -80% 187

2022
01.20 GapIndex <1, P/C, Price and Belowcash continue low, FINRA bouncing off high line. JPM leaderships from giants $BMY and Bayer say biotech valuations are still too high.
2.16 P/C Belowcash stable at low level, index shows signs of reversal, FINRA declined big to 0.44 in Jan.

Sepofarsen QR-110 failed Ph2/3 ADAR RNA editting to restore vision in Leber congenital amaurosis 10, or LCA10, due to the most common p.Cys998X mutation in CEP290 gene. -74% week
Used to >20, now lost 95%., disappoints

Dermata 2021.08 IPO, down since, now offering shares at ~$2.
derived from a natural source of Spongilla lacustris, which contains multiple active components to treat a variety of inflammatory skin diseases with once weekly applications. It is currently under development for the treatment of moderate to severe acne, mild to moderate psoriasis and moderate to severe papulopustular rosacea.  It contains a unique freshwater sponge that is harvested from a select region of the world, under specific environmental conditions to ensure the sponge retains its distinctive anti-inflammatory, anti-microbial and mechanical properties.  After processing, the sponge powder contains precisely sized and shaped silica spicules that upon application may help exfoliate the skin, promote collagen production, open closed comedones (creating an aerobic environment to help kill C. acne bacteria) and create microchannels to facilitate penetration of the sponge’s naturally occurring chemical compounds.

4Q revenue increase from $19M to $24M, up 14% week, small news, previous line expansion failure

BioCardia CardiAMP Cell Therapy System FDA Breakthrough Device Designation for heart failure. 60ml bone marrow aspirate concentrated at bed side and delivered by injection using a catheter. nano Cap

Ph3 failure tradipitant (NK-1R antagonist) in gastroparesis (2018 Ph2 success) only -18%

NHS agreement to allow access to metachromatic leukodystrophy (MLD) drug, Libmeldy (atidarsagene autotemcel) for all children in England and Wales. lost 95% since IPO peak. It contains an autologous CD34⁺ cell enriched population that contains haematopoietic stem and progenitor cells transduced using a lentiviral vector encoding the human arylsulfatase A (ARSA) gene.

3 publications in a row.

sales up 68% at $35M more than $31M expected

SBP-101 is a proprietary polyamine analogue that accumulates in the pancreatic acinar cells due to its unique chemical structure. and disrupts normal polyamine metabolism. started Ph2 SBP-101 with gemcitabine and nab-paclitaxel to treat metastatic pancreatic ductal adenocarcinoma.

partial clinical hold on U2 combination Ukoniq (umbralisib) and ublituximab for chronic lymphocytic leukemia (CLL) and non-Hodgkin’s lymphoma (NHL). PDUFA and AdCom pending.

announce 2022 goals:
1. multiple AB-729 AB-836 trial results
2. IND for PD-L1 AB-101 in 2H22
3. IND for oral RNA destabilizer AB-161 in 2H22 conducted extensive non-clinical safety evaluations with AB-161 that gives us confidence in this molecule’s ability to circumvent the peripheral neuropathy findings seen in non-clinical safety studies with the Company’s first-generation oral RNA destabilizer, AB-452. 
4. nominate and optimize SARS-COV-2 mpro nsp5 and nsp12 polymerase, respectively

Yumanity 2017 IPO, lost a lot, FDA partial clinical hold on YTX-7739 for Parkinson's Disease. a SCD (Stearoyl-CoA Desaturase) inhibitor.

previous wechat hotpick. release data Ph1/2 onvansertib +SOC for metastatic colorectal cancer (mCRC). 29% ORR. -45%. peak $22 YE20. PLK1 inhibitor.

reduce workforce by 25% including 10 executives, CPO, CCO, CSO

Black Diamond Therapeutics FDA IND clearance for BDTX-1535 for glioblastoma multiforme (GBM) and non-small cell lung cancer (NSCLC). BDTX1535, a brain penetrant and irreversible MasterKey inhibitor of EGFR mutations that expressed in glioblastoma multiforme (GBM) and intrinsic and acquired resistance EGFR mutations in non-small cell lung cancer (NSCLC).

Immuron $4.5M funding from DoD for military strength regime of Travelan. Travelan® contains high levels of antibodies that target bacteria such as E.coli (ETEC) and the toxins they produce in the gut. for Diarrhoea.

CMO quit without replacement, lost a lot since last April peak

FDA removed its clinical hold. Also ALLO-501A in LBCL to initiate in mid 2022.

mRNA deal with Acuitas for LNP technology upto 10 targets $ABUS

Ph1/2 EDIT-301 to treat sickle cell disease will begin in 1H2022.  beta-thalassemia program with $BMY

$300M deal with $PFE for Beam's in vivo base editing programs. target a single base without a double-stranded break in the DNA.

52wk relative price the only one above 20 (47)

Announce 2022 goals:
1. expect Heplisav growth
2. expect COVID vaccine milestones and CpG deliveries and capacity expansions
3. Pipeline expansion including Shingles Ph1 (YE22 data), Tdap Ph1 (1H22 adults, 2H adolescent), DOD funded Ph2 plaque to initiate 2H22.
4. universal and seasonal influenza R&D ongoing.

2015 IPO. flat since.  Release Ph1 data showing AVTX-002 anti-LIGHT heal Crohn's disease. Expand to Non-eosinophilic Asthma. AVTX-007 anti-IL18 to discontinue in multiple myeloma due to futility. Still's Disease ongoing.

2019 IPO, down since. 505(b)(2) HT-ALZ release animal model showing reduction of Aβ. Way too early. +44%, penny stock

was Y+300% a year ago, has been retracing for entire 2021. REQORSA TUSC2 gene therapy combo with Keytruda get Fast Track Designation for NSCLC.  +47% week. Previosuly in January 2020, FTD for combo with AstraZeneca’s Tagrisso® (osimertinib, EGFRi) in NSCLC with EFGR mutations progressed after Tagrisso.

BBIO, a hub-and-spike form biotech, 2019 IPO, reports Ph3 data acoramidis for symptomatic transthyretin (TTR) amyloid cardiomyopathy (ATTR-CM). Acoramidis is a small molecule bind to transthyretin and stabilize it, reduces amyloid formation. Pfizer’s Vyndaqel also a small molecule transthyretin stabilizer, but BridgeBio its better in head-to-head lab test, achieving “near-complete stabilization” of transthyretin.
$ALNY Onpattro also this indication. -20%.

another article from SA promoting. up 36%

CX-2029 is a masked form of a proprietary anti-CD71 (transferrin receptor 1) antibody conjugated to monomethyl auristatin E. Ph2 results CX-2029 to treat lung, head and neck cancer. ORR 18.8%. -33%.

Ph3 reproxalap ophthalmic solution (reproxalap) to treat dry eye disease failure. -40%

public offering at 0.39, down to 0.29

2021.08 IPO, in vitro data reporting that ADG20 mAb is less effective than previously thought in neutralizing COVID-19's omicron variant. -68%

Galera Therapeutics GC4419 avasopasem manganese previously failed Ph3 for severe oral mucositis (SOM), now statistically significant, blame the statistically error on CRO.

Valneva, booster trial data after all very good. Previous UK COV-Boost flawed design

85 <=1; 238 <=2 (724 biotech); price 224 <= 3

GH001, is formulated for 5-MeO-DMT administration via a proprietary inhalation approach, release Ph2 data 87.5% remission after 1-week dosing. +24%, June 2021 IPO.

eganelisib PI3K-gamma data in TNBC ongoing Ph2. 89% ORR, 93% DCR, 47%+ PFS. up 24%

published real world data showing 30% reduction in Reduced Hospital Length of Stay and Reduced Opioid Utilization

QiLu license deal: $40 upfront, $15 equity, double digits royalty, milestones 245M.

announce identifies several unique compound series that inhibit the SARS-CoV-2 nsp5 main protease, validated Pfizer target

PDUFA pacritinib to treat myelofibrosis (MF) extended 3 months.

FDA plans for an Advisory Committee meeting for ublituximab and UKONIQ (umbralisib) to treat chronic lymphocytic leukemia (CLL) and small lymphocytic lymphoma (SLL).  Timing in Mar/Apr will not meet PDUFA.

UK booster trial phase2 results published in Lancet showing $VLA is the worst: S Ab, S T and COVID cases. Not a good booster, but only two dose vaccine.

Gracell, CAR-T company from China. 亘喜生物, 2021.01 IPO  management team to use personal fund to buy $2M ADR -18%

offering over subscribed, price increase to $1.25

Ph1b APVO436 for AML data release. 1 high-risk AML patient CR. +61%.

 raise $15.5M at $3.3

financing deal $42M up 43%

2019 Belowcash after Lupus failure, Come back after 2+ years interim Ph2 4/5 patients reported PR/CR, 3/5 >=50% reduction in UPCR

ONCR-177 (Oncolytic HSV combines six immuno-oncology approaches in one – HSV, IL-12, CCL4, FLT3LG, anti-PD-1 and anti-CTLA-4) Ph1 data 1PR 1SD, -47%
2020.10 IPO HSV or synthetic oncolytic viruses for solid tumor

Aditxt 2020.07 IPO, AditxtScore, a multiple antibody  algorithm test for COVID-19 vaccine and treatment recommendation system. Appili Ph3 Avigan and Reeqonus (favipiravir, an influenza drug) failed using AditxtScore as gudiance (maybe could not find evidence, BPC put ADTX on the new item about those two drugs from Appili TSX: APLI; OTCQX: APLIF)

Compass Pathways Ph2b COMP360 psilocybin (natural product found in fungi 迷幻蘑菇) for treatment-resistant depression. 25mg meet endpts yet more frequent TE-SAE than  10mg 1mg, which missed endpts. -28%. 2020.09 IPO

Shattuck Labs and Takeda terminated deal for SL-279252 (PD1-Fc-OX40L) and SL-115154 (CSF1R-Fc-CD40L) bi-specific fusion proteins. 2020.10 IPO

US Government cancelled $180M contract with its Baltimore facilities

QINLOCK (ripretinib, DCC-2618 dual KIT PDGFRa inhibitor) failed Ph3 expansion trial from previously approved fourth line into second line GIST. Comparing to sunitinib (pan-TK) after failed imatinib. use to be $70 now $9. Disappoints are normal in biotech. #deepkinase

YZX 推荐 SHP2 FGFR2 PI3Ka. kinase phosphatase pipelines

First Patient Enrolled in Phase 3 VISION-2 Trial of MicroLine for Presbyopia

ACIP recommended universal HBV vaccination.
ER pretty good.
Q3 Revenue 108M,  increase full year guidance. Operating income 16.1M. 15% 414M cash
HEPLISAV-B revenue record 22.7, market share 33.5%
TDAP Ph1 results due 1Q22
$PFE new oral drug 89% efficacy.

Viaskin Peanut (mVP) development program and STAMP trial initiation are further delayed as DBV awaits FDA review. $50 in 2017. Now $3.

Q3 new prescription growth slowing down, company says additional capital will be required to reach profitability, even after raise. Only $1.3M sales, now reflects actual market performance.

Angion 2021.02 IPO, HGF mimetic (AGN-3777) failed Ph3 for delayed graft function (DGF) after a kidney transplant. Other pipeline: ANG-3070 TKI, ROCK2, CYP11B2 Aldosterone Synthase Inhibitor for organ injury and fibrosis diseases.

忘了保存！ Microcap+ (>50M) Biotech Belowcash 27:
SBTX
TCRR
ORTX
NXTC
ADVM
GMTX
BCEL
ASMB
SVRA
ARDX
IFRX
FRLN
KALA
GLYC
CNCE
ATXS
GLTO
TARA
LUMO
MBRX
ELDN
AIKI
GRAY
CLBS
NBRV
SYN
GRTX

Healthcare -95% None -80% 12

Galera Therapeutics 2019 IPO. lost 90%. GC4419 avasopasem manganese failed Ph3 for severe oral mucositis (SOM) in head and neck cancer (HNC). Could find detailed MOA, related to conversion of superoxide to hydrogen peroxide.

Ph3 success elacestrant to treat ER+/HER2- advanced or metastatic breast cancer (mBC). elacestrant (RAD-1901, ER-306323) is a nonsteroidal combined selective estrogen receptor modulator (SERM) and selective estrogen receptor degrader (SERD) (described as a "SERM/SERD hybrid (SSH)") that was discovered by Eisai and is under development by Radius Health and Takeda for the treatment estrogen receptor (ER)-positive advanced breast cancer.
2014 IPO then 10X peak in summer 2015 with Biotech bubble. Mediocre since then. Also has two Ph3 assets new SC and transdermal formulations of approved drug Abaloparatide (brand name Tymlos) is a parathyroid hormone-related protein (PTHrP) analog drug used to treat osteoporosis.

OTX-CSI (cyclosporine intracanalicular insert) to treat dry eye disease (DED) failed Ph2. Down a lot from YE20 peak.

Aviptadil open label study reported 81% v 21% SOC 60-day sruvival. Aviptadil (INN) is an injectable synthetic formulation of human vasoactive intestinal peptide (VIP). VIP was discovered in 1970, and has been used to treat various inflammatory conditions, such as acute respiratory distress syndrome (ARDS), asthma and chronic obstructive pulmonary disease (COPD).

FDA CRL for its Botox copy DaxibotulinumtoxinA for moderate to severe glabellar lines. Due to "deficiencies related to the FDA’s onsite inspection at Revance’s manufacturing facility".
3 days earlier it was still announcing "anticipating FDA approval despite inspection letter"
A public forum post saying a new cell bank needs to be identified after a working cell bank being identified as a cause of rejected GMP lots.

Indian government approved its partner Bharat Biotech's COVID-19 vaccine for children aged 2-18.

CEO CFO and Perceptive Advisors bought shares at offering. #insider

Valneva success, GMT ratio=1.39, >95% SCR, superior tolerability profile. No big jump. Need orders.

raise $22.7M @$0.85 bring total shares to 114.3M, similar to my other holdings. Half amount warrant at same price. Commercialization is hard for not-so-hot fields.

Prelude 2020 IPO, PRT-543 PRT-811 PRMT5 (Protein Arginine Methyltransferase 5) inhibitors, reports disappointing  Ph1 data. was 95 now $16.

FDA approved TAVNEOS (avacopan) to treat ANCA-associated vasculitis (AAV). up 109% at $38.41. Back to pre-AdCom level. $2.7B

Federal Appeal Board hearing seems clear a win for $ABUS.
@cstingling: From the court of appeal $abus lawyer nailed all questions.  It was like in his DNA. All $mrna lawyer did was shifting blames, making complains, moaning and more moanings.
@Biohazard3737 I spoke to Roivant a couple of weeks ago with regards to $MRNA potentially infringing on Genevant/ $ABUS IP. Interestingly, they thought the strongest patent is a Method of Use patent that expires 2023. $ROIV It was largely ignored, as $MRNA never expected to have a product on the market by 2023. But with COVID, things obviously changed.
@PunditPatent $ABUS Just listened to the oral arguments at the Federal Circuit.  I think ABUS wins the '435 appeal based on standing, and it wins the '069 appeal on the merits.
See link for a youtube video.
Odds: 80% affirms (half rubber stamp in a few days; half opinion in a few months) 10% reverse 10% mixed opinion
3 judges: 1 oldest pro-patent 1 young one lso pro-patent 1 former district judge, no patent background, also good. Cannot be better for $ABUS and patent holder.
ABUS is waiting for the patent dispute decision for a litigation. Jury and litigation judges will refer to USPTO examiner, PTAB, Fed Circuit judges and decisions. Re: Apple v. Qualcomm.
'069 likely in 10 days, '435 likely written opinion or even return to PTAB.

Acceleron acquired by $MRK Merck for $11.5B. Main asset  sotatercept (TGF-beta ligand trap) for pulmonary arterial hypertension (PAH).
8X since IPO. Most gain occur in last 2 years. Prior 6 years flat and 2X

$MRK interim data oral molnupiravir to treat COVID-19. reduce risk of hospitalization or death by approximately 50%. vs antibody 70-80%. Price to be 1/3. Vaccine panic decline yet still valuable. molnupiravir was developed by Emory in 2014 later acquired by Ridgeback. Originally for Venezuelan equine encephalitis virus (VEEV), later show efficacy against other RNA viruses including influenza and coronaviruses.

deal with DOD to develop CpG 1018 adjuvant with the DOD's rF1V plague vaccine. Ph2 in 2022

partnership with U.S. Women’s Soccer legend, Carli Lloyd.

Plinabulin Ph3 results in combo with docetaxel only 1month longer OS vs docetaxel alone (10.5 vs 9.4). Plinabulin blocks the polymerization of tubulin in a unique manner, resulting in multi-factorial effects including an enhanced immune-oncology response, activation of the JNK pathway and disruption of the tumor blood supply.

FDA 2nd CRL VP-102 for molluscum contagiosum. Previous CRL 07.2020. general quality issues” at the CMO’s facility. To resolve soon.

$PFE somatrogon extended release hGH PDUFA extended 3-months

lead drug candidate Mupadolimab, is also an anti-CD73 antibody, up 109% on news Innate Pharma and AstraZeneca’s anti-CD73 monoclonal antibody to treat non-small cell lung cancer (NSCLC).

AR-15512 for dry eye disease fail Ph2b. AR-15512, small-molecule selective agonist of the transient receptor potential melastatin 8 (TRPM8) cold thermoreceptor, a novel therapeutic target for dry eye.
TRPM8 channels, located on the eyelid and cornea, are cold-sensitive thermoreceptors that play a central role in tear film homeostasis.
TRPM8 channels detect drops in corneal temperature associated with evaporation on the ocular surface. TRPM8 nerve terminals exhibit continuous (tonic) and spontaneous activity which rapidly increases with cooling. Increased TRPM8 activity increases basal tear secretion and blink rate. Dysfunction in TRPM8 sensing of evaporation-induced temperature changes may play a role in development of dry eye.

Ph1/2 gavo-cel (TCR) for mesothelin-expressing solid tumors. ORR 31%. 1 Grade 3 1 Grade 5. -45%

Rusfertide PTG-300 on clinical hold due to findings from a non-clinical 26-week rasH2 transgenic mouse model study, likely carcinogen test.
rasH2 (HRAS transgenic model) Useful as a rapid, cost-effective, and sensitive carcinogenicity testing model for detection of nongenotoxic and genotoxic carcinogens, as supported by studies conducted by ILSI.

was 9x, now back to start point

FDA votes on booster dose, emergency use for 65y or older, at high risk, or healthcare professionals. Against general use. $PFE

deal with Genentech for its cancer-targeted allogeneic T-cell therapies for $150M upfront, $150M in 5y, and upto $3B in milestones.

Kadmon ROCK inhibitor  buyout by $SNY Sanofi, not much gain since IPO

raise $20M at market price

IV Empaveli (pegcetacoplan, complement C3 inhibitor) for geographic atrophy (GA, a type of wet AMD) met endpts in one of two Ph3 trials. Miss the other one. -37%

AC Immune announce Ph2 semorinemab (anti-Tau antibody) for Alzheimer AD. Met one of two primary endpoint. to present at CTAD.

Pliant Therapeutics 2020 IPO, 52w peak $40, now $19, lead product candidate, PLN-74809, is an oral, small molecule, dual-selective inhibitor of αvβ6 and αvβ1 for idiopathic pulmonary fibrosis (IPF) and primary sclerosing cholangitis (PSC).
Released Ph2a results showing target engagement. No efficacy results yet due to long horizon. Speculation based on biomarker prediction.

Trudhesa (dihydroergotamine mesylate) is a nasally administered dihydroergotamine (DHE) formulation. FDA approval for acute treatment of migraine.
Impel NeuroPharma 2021.04 IPO

EU says '069 patent is valid

Theravance gut specific pan-JAK inhibitor izencitinib for ulcerative colitis failed Ph2b trial. Tofacitinib systemic inhibitor already approved for the indication. 2014 IPO, I never recorded a single item here at biolog. Mediocre stock performance.

delayed decision on AXS-05 NDA for MDD, previously "identified deficiencies". Requested no additional information. Up 11%, probably will CRL like $DVAX delays.

Citizen Petition to halt Ph2 and pending Ph3 trial until data audit complete regarding allegations of false or misleading data in its Alzheimer’s trials. FDA only commented it received petition. $SAVA named Quanterix as the company generated results in question. Quanterix refuted claims that they only collected the data, didnot do analysis for AAIC meeting. $SAVA confirmed that in a later release.

Stock down 44%.

release interim Ph1 data of FT516 FT596 NK therapy combo w. rituximab for r/r B lymphoma. durability concern.

following CRL news, a further 12-month confirmatory trial is also required with a BLA to be resubmitted in 2023 at the earliest.

Medigen rolls out vaccine with only Phase 2 results. "political will".
Valneva start rolling submission in UK, start Ph3 trial in senior people with new comparing arm VLA2101 targeting variant.
$MRNA $BNTX $NVAX drop big the next day after big rise on PFE/BNT final approval. $DVAX still rise 10%.

$SESN
$FGEN
$SPPI
$ARDX
$ITRM
$INCY
$PRVB
$VCEL/$MDWD
$ORPH
$FBIO/ATXI
$ETON
$PLX
$ACAD

positive interim Ph1 results FTX-6058 for sickle cell disease, proof of mechanism. +184%.
FTX-6058 inhibits polycomb repressive complex 2 inhibitors (PRC2) via binding to embryonic ectoderm development protein (EED). It induced fetal hemoglobin better than others.
Failure with another pipeline DUX4 a year ago. down to ~$7. Now back to $25

Cortexyme 2019 IPO, Ph3 Alzheimer data due mid-Nov 2021, narrowing from 4Q21, up 49%. Burst with data? Atuzaginstat (COR388) / Lysine gingipain (protease) inhibitor
A bunch of enzyme in neurodegenetive diseases.

update NDA bardoxolone for chronic kidney disease caused by Alport syndrome. FDA has identified four significant clinical and statistical review issues for the company to address. AdCom 12/8/21, PDUFA 2/25/22

FDA CRL on Vicinium ADC for BCG-unresponsive non-muscle invasive bladder cancer (NMIBC). FDA noted clinical/statistical data and analyses plus CMC issues.

acquires by $SNY, 2X since IPO, uses LNP $ABUS

ROLONTIS (eflapegrastim) CRL due to CMC issues. RE-inspection required. was delayed due to pandemic.

In trial Alzheimer's and Parkinson’s disease, placebo saw improved cognitive impairment compared with Posiphen ANVS-401. -71%. Other AAIC presenters such as $SAVA also sold off. used to be Y+300%.

Infinity released eganelisib PI3K-gamma in Ph2 Urothelial Cancer (UC) and Triple Negative Breast Cancer (TNBC). 2X PFS in UC, less in TNBC. Analyst worried competition from similar MOA, orPD-1 and Cellular therapy (CAR-T etc.) and commercial potential due to small target population. Some speculate not directly comparable to Roche trial. Expect NDA for UC, but not positive for TNBC.

Q2 Total Rx up 171% New Rx 103% Refill up 355%.
Overall, Twirla has access in approximately 55% of the commercial and government CHC market based on claims.
1.2mil sales, still not compararable due to inventory walk down. still $31.1M cash, 92.9M shares.
See investor presentation excel model for trends.
Tinder marketing exceeding expectation.
Trend comparable to Lo Loestrin FE despite COVID.
Curve is bending up.

-22% ATRC-101 anti-tumor-specific ribonucleoprotein (RNP) complex. 8/20 Stable Disease.

to pay $35m to settle an opioid-related case involving counties and cities in Tennessee vs $2B initially sought. up 41% to $5.

MGTA-117  Anti-CD117 (cKit) amanitin ADC FDA clinical hold for Ph1/2 in acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS)

-64% on SER-287 Ph2 failure for mild-to-moderate ulcerative colitis (UC). Company disappointed not deterred, citing SER-109 C. difficile. Share price back to pre Ph3 success level.

FDA identified deficiencies in Tenapanor NDA for serum phosphorus control in CKD. FDA noting the size of the treatment effect and its clinical relevance as one of the key issues. -78%

CRL issued the following week. Little price change.

CohBar, mitochondria company founded by Nir Barzilai, M.D. Pinchas Cohen, M.D. David Sinclair, Ph.D. COHenBARzilai. data release Ph1a/b CB4211 for NASH and obesity delayed from early July to Labor Day. -28%. Low in cash MCap 62M, cash <20M
CB4211 is a novel peptide analog of MOTS-c, a mitochondrially encoded peptide with a potential role in metabolic homeostasis.
Competitor: Khondrion, Mitobridge. Both private.

negative data from 2 of 3 readouts GLPG3970, success signs for psoriasis, failed both rheumatoid arthritis and ulcerative colitis.
Toledo program is a strategic effort to develop assets inhibiting serine/threonine-protein kinase salt-inducible kinases (SIK). Our lead molecule, GLPG3970, inhibits SIK2/SIK3 and effectuates a dual mode of action on inflammation by stimulating anti-inflammatory cytokines and inhibiting pro-inflammatory cytokines.
peaked 2020.02 at $274, now at $50. still in down trend. Wait for stablization.

rise due to inclusion in SP500, potential boom and burst

up 24% on deal with french company Laboratoire Aguettant to receive up to approximately $55 million in combined up-front and sales-based milestone payments for DZUVEO licensing agreement, obtains the rights to file NDAs and commercialize two innovative pre-filled syringe product candidates (ready-to-use ephedrine and phenylephrine) for the U.S.

FDA clinical hold on Ph1/2 SIG-001 for hemophilia A after 1 of 3 patients (3rd) developed FVIII inhibitor, common complications in FVIII therapies.
2020.12 IPO Sigilon tech is Shielded Living Therapeutics™ (SLTx) platform, encapsulated cell therapy to produce a wide range of missing or deficient therapeutic molecules (e.g. proteins, antibodies and enzymes) Benefit: off-the-shelf, durable, controllable and redosable, without requiring modification of the patient’s genes or immunosuppression.

filed amendment to avacopan (C5aR) NDA addressing AdCom points. delay PDUFA to 10/7/2021.

Microcap+ (>50M) Biotech Belowcash 14:
$ADVM AAV VEGF reports SAE
$SVRA inhaled rhGM-CSF
$DBTX Decibel, gene therapy for hearing and balance, down since IPO 2021.02
$ASMB HBV core inhibitor
$VYGR Voyager continue to down since 2019 Parkinson failure
$IFRX continue to down since 2019 C5a failure
$GLYC continue down since 2018/19 dual failure glycobiology
$ELDN Eledon CD40/CD40L pathway
$CLBS
$LUMO
$AIKI
$SYN
$ABIO
$PULM

Healthcare -95% None -80% 9:
$ASMB Core inhibitor not as great as hoped. Was $55 2.5 years ago. Now $3.5
$TCDA continue down

min Mcap 3.82B, medium 7.08B
$MRNA #1 $BNTX #4 $NVAX #16 $CVAC #17
$CRSP #19 $NTLA #20 $BEAM #33
$MRVI Maravai italian word for miracle, sounds fishy, a portfolio of CDMOs.
$DNLI big portfolio
$FATE rose a lot in the past year NK
$MRTX KRAS
$XLRN TGF-b success
$ASND hGH once weekly $OPK
$HALO Halozyme’s primary technology is based on its proprietary recombinant human hyaluronidase PH20 enzyme (rHuPH20) that degrades hyaluronan, or HA. HA is a glycosaminoglycan, a chain of natural sugars that is a component of normal tissue, such as skin and cartilage. The effects of rHuPH20 are local and transient. By degrading HA, rHuPH20 facilitates the dispersion and absorption of other drugs and fluids that are injected under the skin.
$RXRX April 2021 IPO, already top60. "One of the largest, broadest and deepest pipelines of any technology-enabled drug discovery company." "The industrial revolution of drug discovery is here." Recursion OS ... so grand.
$LEGN July 2020 IPO, already top60, cell therapy
$SAVA stuning come back of Alzheimer candidate

Too many new faces, ignore #41-59

Ph2a IND approved by FDA, AB-729+NA+IFN. After a 24-week dosing period of AB-729 (60 mg SC every 8 weeks (Q8W)), subjects will be randomized into one of 4 groups:
    A1: AB-729 + NA + weekly Peg-IFNα-2a for 24 weeks (N = 12)
    A2: NA + weekly Peg-IFNα-2a for 24 weeks (N = 12)
    B1: AB-729 + NA + weekly Peg-IFNα-2a for 12 weeks (N = 8)
    B2: NA + weekly Peg-IFNα-2a for 12 weeks (N = 8)

collaboration again with VacciTech AB-729, combo with Immunotherapeutic, VTP-300 for Ph2a HBV who are already receiving standard-of-care nucleos(t)ide reverse transcriptase inhibitor (NrtI) therapy. split all costs. VTP-300 utilizes Vaccitech’s ChAdOx1-HBV/MVA-HBV prime-boost combination to elicit an immune response against HBV.

CEL-SCI Ph3 Multikine (injection of 14 cytokines) for HNSCC fails. -68% week.

FDA identified deficiencies that preclude the discussion of labeling and post marketing requirements/commitments for its NDA for sulopenem. -38%. Previously note up 27% when FDA deemed AdCom no longer necessary.

its Fc-engineered antibody report results. A single dose only last a few days, then let go normally. It cannot cure the disease like in other model and experiment design. $ABUS I was worry about RNAi got out compete. It turns out fine. Negative expectation turned out OK.

to discontinue nasal spray COVID vaccine and treatment pipelines.

collaboration with Antios Therapeutics to combo AB-729 with its proprietary active site polymerase inhibitor nucleotide (ASPIN), ATI-2173, and Viread in a Ph2a single cohort.

cinrebafusp alfa  PRS-343 (see previous note for MOA) orphan drug designation for HER2-high and HER2-low expressing gastric cancers. Ph2 to start summer.

2020.08 IPO -40% on Ph2a failure GEM103 in geographic atrophy (GA) secondary to dry AMD. indistinguishable to placebo.

a recombinant form of the human complement factor H protein (“CFH”), and is designed to address both complement hyperactivity and restore retinal health in patients with AMD

FDA awarded Breakthrough Therapy designation for donanemab Alzheimer's Disease. aka N3pG, is a humanized IgG1 monoclonal antibody developed from mouse mE8-IgG2a. This biologic drug recognizes Aβ(p3-42), a pyroglutamate form of Aβ that is aggregated in amyloid plaques. Most Aβ antibodies in therapeutic development bind various soluble or insoluble species but have low affinity to deposited amyloid plaques. The rationale behind donanemab is that targeting deposited plaque itself is necessary to clear existing amyloid burden from the brain, rather than merely prevent deposition of new plaques or growth of existing plaques. Some previous plaque-binding antibodies have been abandoned because they caused microhemorrhages in the brain. The mE8 antibody was reported to clear plaques in mice without causing microhemorrhages.

down 50% FDA CRL for arimoclomol (amplifies heat shock proteins HSPs) in Niemann-Pick disease type C (NPC), previously failed ALS. Licensed from $CYTR. also down 62%

CRL based on needing additional qualitative and quantitative evidence to further substantiate the validity and interpretation of the 5-domain NPC Clinical Severity Scale (NPCCSS) and, in particular, the swallow domain. Further, the FDA noted in the CRL that additional data are needed to bolster confirmatory evidence beyond the single phase 2/3 clinical trial to support the benefit-risk assessment of the NDA

zuranolone (SAGE-217/BIIB125) 50 mg  for major depressive disorder showing statistically significant improvement in depressive symptoms compared with placebo at Day 15. Market still worry its durability. -28% week.
It is a synthetic, orally active, inhibitory pregnane neurosteroid, and acts as a positive allosteric modulator of the GABAA receptor.

Clover announced commercial agreement with $DVAX and COVAX pre-order of 414M doses.

release US Ph3 results. 90.4% efficacy, 100% protection against moderate and severe disease. 93.2% against VOC. sell off 17%

7X since 2020.3, 15month.
FDA gave Breakthrough Therapy Designation.
EHA Ph2 Data Supporting Long-Term Efficacy of Rusfertide PTG-300 in Polycythemia Vera. Rusfertide demonstrated sustained hematocrit control, reversal of iron deficiency, and a reduction in phlebotomies, for up to 18 months, in both high risk and low risk patients
Rusfertide (PTG-300) is currently being evaluated for various disorders associated with iron overload and/or excessive erythrocytosis (red blood cell production). Rusfertide is a synthetic mimetic of the natural hormone hepcidin that regulates iron homeostasis and controls the absorption, storage, and distribution of iron in the body. Discovered through our peptide technology platform, rusfertide is currently in a Phase 2 study in polycythemia vera (PV), a rare chronic blood disorder that affects about 160,000 patients in the U.S. In addition, it is also being evaluated in a small open-label Phase 2 study in hereditary hemochromatosis (HH), a broader population-based blood disorder affecting over a million people in the U.S. alone and arising primarily from absence or deficiency of the hepcidin pathway in these patients.
Hormone replacement therapy.

Flt3/BTK inhibitor new data fails to impress at EHA, I did not fully understand details. Could be sell off on news.

reports SB-206 Ph3 success molluscum contagiosum. Previous failed. No adverse event and to NDA by 3Q22.

Lyra Therapeutics, 2020.05 IPO, FDA agrees on LYR210 trial design for chronic rhinosinusitis CRS.  The single primary endpoint will evaluate improvement at week 24 using a composite score of three cardinal symptoms (3CS) of CRS: nasal blockage, nasal discharge, and facial pain.
LYR-210 is an anti-inflammatory implantable drug matrix based upon Lyra’s XTreo™ platform designed to elute mometasone furoate consistently and locally to the inflamed mucosal tissue of CRS patients for up to six months from a single treatment. LYR-210 is being developed as an alternative to sinus surgery for CRS patients.

aducanumab approved against AdCom, 3/11 member resigned, FDA ask confirmatory trial complete by 2029.08, data by 2030. at least 9 years of sales. annual cost $56,000

previous CMC CRL, release Ph3 data. pegunigalsidase alfa (PRX–102) for Fabry disease miss changes in eGFR when compared with agalsidase beta (Fabrazyme), were below the non-inferiority margin pre-specified for the interim analysis in ITT. eGFR, or Estimated glomerular filtration rate, measures the level of kidney function. The company also noted that one patient discontinued due a related treatment emergent adverse event.

through its affiliate QED Therapeutics Inc and Helsinn Group, FDA accelerated approval Truseltiq (infigratinib) forcholangiocarcinoma (CCA) with previously treated locally advanced or metastatic CCA harboring an FGFR2 fusion or rearrangement. Truseltiq is an orally administered, ATP-competitive, tyrosine kinase inhibitor of FGFR. The approval of Truseltiq is based on Ph2 results.
Little price move.

was #BelowCash. up 17% to $9.60 FDA approved sNDA for oral-only dosing regimen of NUZYRA for Community-Acquired Bacterial Pneumonia (CABP).

Liminal BioSciences FDA approved Ryplazim (plasminogen replacement therapy) for plasminogen deficiency type 1 (hypoplasminogenia)., the first FDA approved therapy for this rare genetic disorder. Up 55% AH.
Previous TSE stock, 2019 listed to NASDAQ. Mediocre performance since.

5 abstracts at EASL

FDA CRL for dehydrated alcohol injection for the treatment of methanol poisoning due to COVID restriction prevented a Pre-Approval Inspection (PAI) of its European contract manufacturer. -17%

2015 IPO Lantheus, we are dedicated to the development, manufacture and commercialization of innovative diagnostic imaging agents, targeted therapeutics, and artificial intelligence to find, fight, and follow disease. IPO: "a global leader in developing, manufacturing, selling and distributing innovative diagnostic imaging agents and products".
up 13% to $24.25 following its announcement the FDA approved PYLARIFY, an F 18-labeled prostate-specific membrane antigen (PSMA) targeted positron emission tomography (PET) imaging agent to identify suspected metastasis or recurrence of prostate cancer.

5y 10X

Larimar Therapeutics CTI-1601 is a recombinant fusion protein intended to deliver human frataxin into the mitochondria of patients with Friedreich’s ataxia (FA) $RETA. which is the cause of the disease. FDA clinical hold after NHP toxicology study show mortality.
Chondrial Therapeutics and Zafgen Complete Merger and Begin Operating as Larimar Therapeutics 2020.05

Genentech licensed its Anticalin tech for respiratory and ophthalmology therapies, $20M upfront upto 1.4B milestones

licensed Heplisav-B to Bavarian Nordic for Germany.

CEO CFO both bought 20K shares in May 2021

2017 IPO reverse merger with Aadi Bioscience, Inc, precision therapies for genetically-defined cancers.

2020.01 IPO $6 now $56 with $98 intraday peak. ANVS401 Ph2 Alzheimer's Disease AD Success, 25 days show  statistically significant cognitive improvement as measured by an 11-part test.
ANVS401 small lipophilic molecule  enters the brain and inhibits "translation" of all three Alzheimer proteins APP, tau and αSYN. Did now say how.

FDA ask company to withdraw type C meeting and request pre-NDA meeting on Omaveloxolone for Friedreich's Ataxia.
Bardoxolone in NDA now. $RGLS competitor

Begin manufacturing, EUA by July. More revenue for DVAX.
Btw, FDA in this news is not US FDA, it means Taiwan Province, China FDA.
The revenue in Taiwan Province, China will be small. So someone bigger have to license or buy it to make it significant for DVAX.
Both vaccines (Medigen and United Biomedical) from Taiwan Province, China will be EUA with only Ph2 trials..... kind of risky, but I guess they really need them asap.

clover announce pre-clinical data for next generation booster. Adjuvant is not necessary. numerically 1.7-2.4x less titer. So still better with adjuvant.

Clover Health, SPAC deal 2020, false claim of subscribers in SPAC deck
@Dirk:
Never invest in a stock where main backer + chief pumper posts naked upper body pictures of himself on Twitter...kind of common sense.

Ginkgo Bioworks grabs $DNA ticker in a SPAC deal

sell off 17% on FDA approval ZYNRELEF (HTX-011, bupivacaine and meloxicam) post-operative pain management for use in adults for soft tissue or periarticular instillation to produce postsurgical analgesia after bunionectomy, open inguinal herniorrhaphy and total knee arthroplasty.
peak was in 2018

more data on IRAK-4 success: BM blast reduction in 8/9. 1 complete recovery, 1 CRi, 2 BM CR.

Island reversal. I speculate that FDA will reject Matrix-M adjuvant. It won't be easy to get a new adjuvant pass FDA  $DVAX
-25% week due to "not expected to seek emergency use authorization for its Covid-19 vaccine until June at the earliest, with data not expected until late-May". Also announceda revised timeline to reach manufacturing capacity. It expects capacity of 100 million doses per month by the end of the third quarter of 2021 before ramping up to 150 million does per month during the fourth quarter. The company had previously guided that full capacity would be reached in mid-2021.

Athenex acquired Kuur Therapeutics, Inc., a developer of off-the-shelf CAR-NKT cell immunotherapies for the treatment of solid and hematological malignancies.  70M upfront + up to $115M in milestone.
ATNX Previous CRL on P-gp pump inhibitor, encequidar.

Ph2 results monthly dose of SRP-5051 30mg/kg produced 8x (6.55% v. 0.86%) dystrophin as weekly dose of Exondys 51, its current approved drug for DMD.
SRP-5051 is next generation eteplirsen, in that it targets the same population, those amenable to exon 51 skipping, but the compound is “charged”, meaning that its cell-penetrating capacity is increased.

AVR-RD-01 ex vivo lentiviral gene therapy agalsidase beta no longer eligible for accelerated approval due to the approval of agalsidase beta Fabrazyme from Sanofi Genzyme. lost 80% since 2018 IPO hype.

not to pursue NDA until 1H2022  citing delays in manufacturing, in part caused by the global COVID-19 pandemic.
Soligenix, 2013 IPO, SGX301 for Cutaneous T-Cell Lymphoma. Synthetic hypericin, the active ingredient in SGX301, tends to accumulate in T-cells. Once the hypericin is in the T-cells, it can be activated by safe, visible fluorescent light. When synthetic hypericin is activated it creates oxygen radicals which subsequently cause cellular toxicity, killing the targeted T-cells.

panic selling upon vaccine IP waiver comment from Biden. Same day reversal. Bottom for Biotech?
My holding review: ABUS, ACRX, AGRX, DVAX all doing well on track with their business. nothing has changed. Still above 2020 Mar and 2020 Oct bottoms.

ER: nothing new. Continue to sell ATM.

Sales (M)/Px (K):
2021:  0.1/1.9,

twitter argument:Replying to @GuptaBioDD @DudeBiotech and 2 others
1. No COVID vaccine uses AS01B, all GSK deals uses AS03
2. Medicago the only one use CpG w.o. alum, hence weak, everyone else + alum
3. AS01B has MPLA = strong side effects, see Shingrix reports.

ADVM-022 (AAV.7m8-aflibercept) anti-VEGF gene therapy Ph2 trial for diabetic macular edema (DME) report a Suspected Unexpected Serious Adverse Reaction (SUSAR) of hypotony (decrease in ocular pressure). AAV continues to deliver SUSAR. How many more SAEs and deaths will it take for the gene therapy field to switch to LNP? $ABUS

Was Y+300%, now lost 80% more from 2020 peak.

plant cell based expression for protein biologics. 2006 reverse marger, Pegunigalsidase alfa for Fabry disease FDA CRL due to COVID travel restriction on CMC inspection.

EU still in talk with Valneva, 10 states want Valneva's vaccine, fear of prioritize UK. "Conditions not met": "solid protection in the areas of liability safety, effectiveness of the vaccine and also solid rules and arrangements for the delivery of the vaccines."

Epsolay NDA unable to complete since a pre-approval inspection of the production site of EPSOLAY (benzoyl peroxide 5% topical cream for Inflammatory lesions of rosacea) still needs to be conducted.

CpG1018 is safer(!) than Alum. $DVAX has the safest vaccine adjuvant in the world: SAE: Heplisav 1.5% v. Engerix 2.1%; AMI 1.67 v 1.86; Fever 1.3% x2 doses v. 1.8% x3.
I am not worried. In science, good science may be late but never absent.
$VXRT host KOL event to show T cell is important in COVID vaccine
$NVAX Biden mentions Novavax name in speech

Ph1 data SNDX-5613 in mixed lineage leukemia rearranged (MLLr) and nucleophosmin (NPM1c) mutant relapsed/refractory (R/R) acute leukemias. 48% ORR (15/31). Stock down -24% due to only 5 CR. Previosuly (2020.05) 2/3 ORR 1PR1CR.

will it announce co-promotion in the USA?

partner, Bharat Biotech, 2ns interim analysis shows 78% efficacy, 100% against severe disease. 45% profit in US market. +67%

香港上市 估值9.6亿美元

Valneva enters Ph3. this pivotal phase 3 is much smaller, 4000 enrollment. It is also a comparative trial against $AZN. Is this the first comparative COVID Ph3? AFAIK. Salute to UK regulators.

-20% shake out on no news, overwhelmingly bullish odds https://wallstreetodds.com/DVAX/#sameday

Valneva Switches Focus to Bilateral Discussions to Supply its Inactivated, Adjuvanted COVID-19 Vaccine Candidate VLA2001, could not get deal with EU. The enthusiasm of UK and lukewarm of EU are in sharp contrast.
EU is really committed to $PFE $BNTX; they have frustrated $AZN first, then $NVAX, now $VLA.PA $DVAX.  Sadly, EU is in short supply of any vaccine. What makes it so proud? #CovidVaccine

use physics Tumor Treating Fields (TTFields) to treat cancer. report Ph3 success in NSCLC. Interim analysis enrollment  can be reduced, save one year completion time. +60% week. China right owner $ZLAB up 24%

EU 50M more -> 250M total deal, potential 1.8B doses more deal from 2021 to 2023 $PFE, $DVAX

a doctor at newtalk claim valneva may be the best at variant.
Valneva planning Nasdaq IPO

reports data Ph1 cord blood-derived natural killer (cbNK) cells with its innate cell engager (ICE) AFM13 (CD16A/CD30) in recurrent/refractory CD30-positive lymphomas. 4 patients 2 CR 2 PR 100% ORR @Dirk heavy in

FDA approved Qelbree SPN-812 for ADHD, another ADHD drug!

 down 48% to $18.57 following a disclosure regarding its anemia treatment, roxadustat, in chronic kidney disease. as it was preparing for AdCom meeting NDA, it realize their analysis  had included altered data to make the treatment appear safer than it was. Now it cannot conclude that roxadustat reduces the risk of (or is superior to) MACE+ (Major Adverse Cardiovascular Events) in dialysis, and MACE and MACE+ in incident dialysis compared to epoetin-alfa. begun an internal review to ensure such issues do not occur in the future.

It is always sobering to see a shell company like $OCGN has higher market cap then a company $DVAX with an approved product, providing adjuvant to five #CovidVaccine candidates, and a pipeline of other vaccines.

FDA IDE approval to begin VenoValve trial in US. Still no share price rise.

hepatic chemo injection system. filter out drug in blood before returning it to patient. PH3 HEPZATO KIT for liver dominant metastatic ocular melanoma (mOM). 29% (95% Cl%: 20.1, 39.8) ORR vs. predefined goal of 21% disappointing.

discontinue WVE-120102 and WVE-120101 for Huntington's disease due to lack of efficacy.

$JNJ vaccine manufacture failure, wasting up to 15mil doses. NYTimes reports ingradients mixed up between $JNJ and $AZN vaccines.

COVID anti-GM-CSF Lenzilumab Ph3 54% greater chance of survival. $KNSA

deal with X-Chem (DNA-tagged small molecule library for high through put screening), Proteros Biostructures on nsp5 main protease M^pro. to combine with assets arising from internal nucleoside program targeting the SARS-CoV-2 nsp12 viral polymerase

Aptose CG-806 luxeptinib, the only Flt3/BTK dual inhibitor reported a CR in Ph1 R/R AML. Also in B malignancies CLL and NHL.
Also has MYC pipeine #嘉华

up 45% Ph1/2  QR-421a (exon 13 skipping USH2A) in Usher syndrome and non-syndromic retinitis pigmentosa (nsRP). n=14, 6 letters improvement at week 48 in the treated eyes compared to untreated after a single injection. Down big since 2018 notes.

FDA says data to date would likely not be sufficient for FDA approval. discontinue development tesetaxel, winding down company

$50M ATM offering, CEO CFO insider buy 62.5k shares at $2, similar to $ACRX, rise may be far away.

$PFE going alone on mRNA medicine mostly vaccines. Shameless: “We are the best positioned company right now to take it to the next step because of our size and our expertise." ... "He said the company’s scientists and engineers gained a decade’s worth of experience in the past year."

donanemab  (anti-Amyloid-beta) Alzheimer's disease falling short of investor expectations.

MRT5005 LNP+mRNA 2nd interim analysis Ph1/2 cystic fibrosis (CF) exhibited no lung function increase by ppFEV1. $ABUS

RTX-240 is an allogeneic, off-the-shelf cellular therapy product candidate that is designed to simultaneously present hundreds of thousands of copies of the costimulatory molecule 4-1BB ligand (4-1BBL) and IL-15TP (trans-presentation of IL-15 on IL-15Rα) in their native forms. RTX-240 is expected to broadly stimulate the immune system by activating and expanding both NK and memory T cells to generate a potent anti-tumor response.

Reports 1PR and 1 unconfirmed PR in hard to treat cold tumor and metastatic tumor patients

高探生物 高瓴独家投资 三千万 天使轮 TCRT 单细胞测序 实体瘤靶向TCRT

Valneva talking about licensing its vaccine out of Europe

CMO leaving company on 4.16. Abandon ship?

Recent IPO 2020.09, was $38, now $8. GB-102 for wet AMD data release, 9 letter diff than placebo, still disappoints.
GB-102 (pan-VEGF inhibitor sunitinib) formulated with Graybug's formulation tech for extended release. #嘉华

announces final UK data, 96% original strain, 86% UK variant, 49% in SA (down), 55% in HIV-.

KD025, belumosudil, PDUFA extended 3 months.

FDA identified deficiencies that preclude discussion of labeling and post-marketing requirements for its sNDA of Pimavanserin for allucinations and delusions associated with dementia-related psychosis.  down 45% to $27.04.

Ph2 luvadaxistat (NBI-1065844/TAK-831, oral inhibitor of d-amino acid oxidase (DAAO, developed by Takeda)) fail in negative symptoms of schizophrenia

unexpected announcement of AdCom, PDUFA likely to be delayed, already postponed 3 months.

CRL for oral paclitaxel plus encequidar (oral enabler) for metastatic breast cancer. need new trial.

buyout by $AMGN for $38/share. was #belowcash $4 then. Zai Lab deal; was superstar before that.

2.47 gap closed. !!!

4Q20, FY20 CC: revenue 1M as guided.  Loss 17.6M. COVID impact uncertainty.
Increases Credit Facility to $45 million, with $25 million potentially available.
Announces Agreement with Sterling Specialty Pharmacy
New CMO Paul Korner, M.D. evaluating pipeline and BD opportunities
achieved approximately 40-45% commercial formulary coverage so far, to close gap at $2.48?

Vascepa results controversial, new trial negative results, success trial control arm is mineral oil, potentially increase control arm rates. Advocates argue Japanese open label trial.

FDA says Ph3 NurOwn data in Amyotrophic lateral sclerosis ALS does not provide the threshold of substantial evidence to support a BLA

FDRR denied

$GSK invest $120M equity and pay $225 upfront to expand collaboration. COVID Ph3 due 1Q21

Chromadex see Los Angeles article.  Stock up on 40% faster recovery for COVID patients. 谭君子感兴趣. chat 20min regret.

4Q FY20 CC, FY20 revenue $46.6M, up 32% from 2019 $35.2M, Heplisav 4Q 11.5M 26% Market share; 1Q2021 CpG 1018 revenue $40 ~ $60M
ACIP discussing universal HepB vaccination to vote in October

Insilico Medicine (Hong Kong) 人工智能发现 IPF新靶点和候选药物

$MRNA filed appeal today by the 3rd extended deadline. No deal, but something is there. Why appealing if no infringement?

Dociparstat sodium is a potential first-in-class glycosaminoglycan compound derived from porcine heparin that has low anticoagulant activity In vitro and in vivo animal model data support DSTAT’s potential to reduce the inflammation and cellular infiltration associated with acute lung injury and address coagulation disorders associated with COVID-19 pathology.

gap closed

Added 339 shares at $4.449 ($1.5K). anticipating Moderna and Genevant deal next Wed, in 3 trading days. Decided not to wait for $4 50MA level (original order). Better than $5. Not a big deal. Low next days: $4.25
2021.2.23 Market crash, three -10%, 我的预感是对的
2021.2.24 bounce back on appeal file

Only 2 Biotech Belowcash:
$ASMB ($194M MC) Capsid inhibitor not enough for mono
$ABIO ($44M MC) new beta-blocker COVID spike in 2020.05

Healthcare -95% None -80% 4:
$TCDA non-absorbed polymer removes HCl from GI and blood. CRL, was blockbuster potential, then FDA think biomarker not enough for efficacy. See main item for details
$SONN reverse merger in 2020. low dose IL-6. Fully human albumin binding Fv to deliver cancer payloads.
$RMED excimer laser systems are tools for use in the treatment of vascular and dermatological diseases
$NBRV 1:10 reverse split in 2020.12 antibiotics CRL.

Another Investigator-Initiated Study of DSUVIA® in Patients Undergoing Plastic Surgery Procedures (Orange County)

RA capital digram $NVAX Matrix-M side effects are stronger than AS03, especially for headache, fatigue and muscle pain. Good news for $DVAX, which is way safer than AS03 already in Clover and Medicago trial as well as Heplisav trial.

2021.2.16 Correction: Shingrix uses AS01B, contains MPLA, a TLR4 agonist. TLR4 is a bad pathway for specificity, hence poor side effects. Matrix-M side effects ~> AS01B > AS03 >> CpG. $NVAX $DVAX

Eagle, mediore since 2015 patent win rise

Phase 3 with $GILD of ziritaxestat for idiopathic pulmonary fibrosis discontinued due to insufficient efficacy -70% from peak

eganelisib IPI-549 PI3K-gamma inhibitor + PD-1 in platinum-refractory, I/O naïve patients with advanced urothelial cancer: 30% ORR vs 25% PD-1 only. In PD-1 negative, 26% vs 14%, up 13%
A company  with long history 1:4 split in 2006, founded by Steve Holtzman in 1995. Mediocre stock performance.

Philadelphia based Ocugen to bring India Bharat Biotech's vaccine to the US, stock price rise from $0.29 on December 21 to $18.77 as of 2.8.2021 in ~6 weeks. Crazy! @Dirk, use alum, likely will be rejected by FDA for inadequate data/trial, don't expected to be distributed in the US, pump and dump.

@Dirk, $OCGN ingenious to raise paltry (now >$2B mc) $23M at eye-watering premium ($7.65 vs $5.90 2.5 close) to con the market into believing that 'real' investors see that much value in stock.

FcRn is the primary protein responsible for preventing the degradation of IgG antibodies and albumin, the most abundant protein found in the blood. The role of FcRn is to bind to the IgG antibodies in the endosome, and transport them to the cell surface, where they are released back into circulation. Other proteins are degraded if non-receptor bound.

IMVT-1401 is designed to selectively bind to and inhibit FcRn, thus blocking the recycling of IgG antibodies.

Trial voluntary hold due to a physiological signal consisting of elevated total cholesterol and LDL levels in IMVT-1401-treated patients in ASCEND GO-2, a Phase 2b trial in Thyroid Eye Disease (TED).

failed in 2018,  to initiate confirmatory trial of Birtamimab for newly diagnosed, treatment naïve patients with AL amyloidosis. Target p value <= 0.1 unprecedented.

Arcutis Biotherapeutics 2020.01 IPO Topical roflumilast cream (ARQ-151) is a small molecule inhibitor of phosphodiesterase type 4 (PDE4), an intracellular enzyme that increases the production of proinflammatory mediators and decreases production of anti-inflammatory mediators, and has been implicated in a wide range of inflammatory diseases including psoriasis, eczema, and chronic obstructive pulmonary disease (COPD).
ARQ-151  2 Ph3 positive data for plaque psoriasis

FDA approved Posimir (bupivacaine solution) to produce post-surgical analgesia following arthroscopic subacromial decompression. AdCom 6-6. Twice the mcap of $ACRX

buyout $VIE for $53 in cash $3B 2.5x since IPO only 15 months

@Dirk find that CpG1018 patent expired in 2018. would think that agreements would prohibit 'partners' from going off on their own.
I wonder the same. This is a new field: generic CpG oligo adjuvant. Will it be like a generic chemical? or will it be like biosimilar? Sure, everyone can develop their own CpG adjuvant, that will cost billions $ and 2 decades.
MF59 went clinic in 1992, 30 years later, patent should have expired, no generic.

bioRxiv paper showing "Neutralization of SARS-CoV-2 VOC 501Y.V2 by human antisera elicited by both inactivated BBIBP-CorV and recombinant dimeric RBD ZF2001 vaccines"

1)If true, very good news for the world, no need to develop new vaccine, just use adjuvant to boost existing vaccine.
2) Also potential advantage of WT versus S.2P. Interesting development.
3) ZF2001 RBD vaccine (with alum) is weaker than inactivated BBIBP-CorV.

T cell immunity important in protection against severe COVID of variant, says JNJ executives.
T cell epitopes little overlap with antibody, key in protection against variants. CD4 T cell epitope little RBD, S protein OK.
Comprehensive analysis of T cell immunodominance and immunoprevalence of SARS-CoV-2 epitopes in COVID-19 cases

@Steam_Sports: $MRNA recently filed for another (3rd one) extension of time to file its principal brief in its appeal v. $ABUS. Moderna's brief is now due 02/24. I imagine there are settlement discussions ongoing as the date keeps getting pushed out.
@DeborahDolen: "Unopposed" is the key word here.  If they were not close to an agreement it would be VERY opposed.  $ABUS  More so the third extension.

aducanumab PDUFA extended 3 months

Medigen 1 dose did not happen; NVAX tox?

announced 2021 goals, cash increased 5mil ($40M ATM?) 10-15M shares sold. Announced two more Ph2a trials with AB-729 in 2H21; AB-836 Ph1 in 1H21.

anti-LAG3 developed for autoimmune disease GSK2831781 (licensed from french company Immutep). Ph2 trial for active ulcerative colitis discontinued.
formerly Prima Biotech, changed its name to Immutep, a subsidiary at the time.

Ph2/3 troriluzole for mild-to-moderate Alzheimer's disease (AD) fail

TI-450, an Oral MK2 Inhibitor as TNF mab alternative, Ph2 success, was #BelowCash from $0.7 to $18. Company accept commercial failure, refocus, management change, fall below cash, rebound eventually.

Jan 2021 corp ppt mentioned ARX-02 ARX-03 for the first time for a long time.

pop to $2.94 then raise AH, just as I feared. Suddenly break out upwards in huge volume after H.C. Wainwright Bioconnect 2021 Conference

US government buying additional upto 1.25m doses of casirivimab and imdevimab antibody cocktail delivered by June 30, 2021, in addition to the current supply of doses to treat 300,000 people under a previous agreement

anti-beta-amyloid Donanemab Ph2 success, met the primary endpoint, slowing the decline of patients on the Integrated Alzheimer's Disease Rating Scale (iADRS) by 32% relative to placebo. can it translate to Ph3? $BIIB also up on the news

Corp PPT Medigen data due early Q1, Ph2 to start mid Q1; Medigen announced 12.30.2020 TFDA approved Ph2 trial based on interim Ph1 data.

struggling BlueBird Bio split into two companies
@RNAianalyst:
Once the most adored genome editing Co, $EDIT, + most pop gene Rx Co, $BLUE, illustrating that you cannot 'manufacture' good biotechs.
$EDIT 'transitions' CSO, $BLUE splits up.

discontinued lead candidate MLE-301 (NK3R inhibitor) after reviewing PKPD data Ph1a SAD healthy volunteers.  "highly competitive NK3R antagonist market". to liquidate or merger.

telaglenastat (Glutaminase Inhibitor, CB-839) in combo with cabozantinib (kinase inhibitor) fail advanced or metastatic renal cell carcinoma (RCC). No PFS difference. Tumor metabolism. Lay off 35% staff. -45%

IV board really quiet no post since 2020.12.16

Phase 3 HELIOS-A trial of vutrisiran for transthyretin-mediated (ATTR) amyloidosis, met its primary and both secondary endpoints, compared to historical placebo data from the APOLLO Phase 3 study of patisiran. Self innovation.

previous TapImmune (peptide vaccine for cancer) merged with Marker in 2018 and adopted its name and Multi-Antigen Targeted Cell Therapy tech.
MT-401 for post-transplant AML FDA lifted the partial clinical hold on its Phase 2.

relugolix deal with $PFE $650M upfront, up to $4.2B