New Drug Candidates Debut in Atlanta
At the ACS Spring 2026 meeting in Atlanta, the Medicinal Chemistry division unveiled six new drug candidates transitioning from discovery to clinical testing. The molecules, presented by researchers from Biohaven, Bristol Myers Squibb, Regor Therapeutics, Olema Oncology, FoRx Therapeutics, and Iambic Therapeutics, target mechanisms such as TRPM3 for neuropathic pain, IKZF transcription factors in solid tumors, CDK4/2 in HER2‑negative breast cancer, KAT6 epigenetic regulation, PARG DNA repair, and HER2 signaling. All six are in Phase 1 to Phase 2 trials, with identifiers NCT06603623 through NCT07356453. The session also underscored the growing role of AI‑driven drug design and heightened market activity around molecular glues and CDK inhibitor acquisitions.
How Seaport Is Hedging Against Failure in Phase 2b Depression Study
Seaport Therapeutics is embedding a fail‑safe mechanism into its Phase 2b trial of SPT‑300, an experimental therapy for major depressive disorder. The study will enroll roughly 300 patients at multiple U.S. sites and uses an adaptive design that can halt...
BREAKING STUDY: COVID-19 “Vaccines” DISRUPT THE BLOOD-BRAIN BARRIER — 63 Serious Brain & Spinal Cord Safety Signals Identified
A recent Substack post cites a study claiming COVID‑19 mRNA vaccines increase reports of rare neurological disorders by dozens to thousands of times compared with flu shots, based on VAERS data from 1990‑2024. The post lists specific conditions such as...
FDA Approves Relacorilant with Nab-Paclitaxel for Platinum-Resistant Epithelial Ovarian, Fallopian Tube, or Primary Peritoneal Cancer
The FDA has approved relacorilant (Lifyorli), a glucocorticoid‑receptor antagonist, in combination with nab‑paclitaxel for adults with platinum‑resistant epithelial ovarian, fallopian tube or primary peritoneal cancer who have received up to three prior regimens, including bevacizumab. The approval is based on...
Hemodynamic and Biochemical Effects of Intravenous versus Oral Fluids in Healthy Volunteers – A Protocol for a Randomized Crossover Trial
A Phase 1 randomized crossover trial will compare the hemodynamic impact of 1 liter oral tap water versus 1 liter intravenous Ringer’s lactate in 20 healthy volunteers. The primary endpoint is the change in mean arterial pressure 30 minutes after fluid administration, with secondary...
The Brain's Cleaning System Can Be Boosted to Rid Alzheimer's Proteins
Researchers identified a drug combination that enhances the brain's glymphatic system, improving clearance of Alzheimer‑related proteins. The regimen pairs a widely used sedative with a medication that prevents dangerously low blood pressure, showing safe and effective removal of amyloid and...
Curiosity Fuels AGI: Autonomous Labs Powered by GPT‑5
Awesome being on the @Sequoia Training Data podcast with @gradypb and @sonyatweetybird. Topic links in the 🧵 The most human part of intelligence is curiosity, and science is formalized curiosity. If we want AGI — doing the work of science is...
Bridging Virtual Cells to Real Patients: The Next Frontier
Virtual biology burst onto the scene a few years ago. The idea was simple. Model the cell well enough, and you can predict biology before you test it. Large pharma has been thinking about this longer than most. PK/PD models, systems biology,...
STAT+: FDA Approves Denali Therapeutics Drug for Hunter Syndrome
On March 27, 2026, the U.S. Food and Drug Administration granted approval to Denali Therapeutics’ drug Avlayah for the treatment of Hunter syndrome, a rare lysosomal storage disorder. The decision arrives amid a recent wave of stricter FDA scrutiny of...
Two Polyunsaturated Lipids Demonstrate Senolytic Activity
Researchers identified two conjugated polyunsaturated fatty acids, α‑eleostearic acid (α‑ESA) and its methyl ester (α‑ESA‑me), as potent senolytics that selectively eliminate senescent cells. In mouse models, short‑term dosing reduced senescence markers and SASP factors across liver, heart, kidney, and lung...
NOVOS Trial Shows Vascular Aging Biomarker Improvements in Midlife Adults
NOVOS launched a randomized, double‑blind, placebo‑controlled trial of a multi‑component supplement in adults over 40, enrolling 61 participants and completing six months with 43 finishers. Early preprint data show a 2.9% improvement in flow‑mediated dilation, a 1.18 m/s reduction in pulse‑wave...
Akeso Secures NMPA Clearance for Phase II Trials of First-in-Class ADCs
Akeso, Inc. has obtained clearance from China’s National Medical Products Administration to begin Phase II trials of two novel antibody‑drug conjugates, AK146D1 and AK138D1. The studies will combine the ADCs with the company’s approved bispecific immunotherapies cadonilimab and ivonescimab, marking the...
Shanghai Fosun Pharma Posts $472 Million Profit, Boosting R&D Outlook
Shanghai Fosun Pharmaceutical (Group) Co Ltd announced full‑year profit of RMB3.371 bn ($472 m), up 22% from the prior year, while revenue edged 1.4% higher to RMB41.498 bn. The earnings lift expands the group’s capacity to fund R&D and health‑technology projects amid a...
FDA Clears Denali's AVLAYAH, 91% CSF
FDA clears Denali's AVLAYAH (macro), 91% CSF HS drop and analysts lift estimates (key). Risk: COMPASS confirmatory readout, infusion reactions. Trade: buy DNLI on 50‑day pullback 🚀 — Viktor Kopylov, PhD, CFA More insights: t.me/si14Kopylov
Rosen Law Sets April 14 Lead Plaintiff Deadline in REGENXBIO Securities Fraud Suit
Rosen Law Firm announced that investors who bought REGENXBIO shares between Feb. 9, 2022 and Jan. 27, 2026 must file to become lead plaintiff by April 14, 2026. The class‑action alleges the gene‑therapy company misled investors about its RGX‑111 trial, underscoring heightened scrutiny of biotech disclosures.
Scout-Triggered Proteomics Sharpens HCP Control
Host-cell proteins (HCPs) remain a persistent impurity risk in biomanufacturing, with regulators demanding levels below 100 ppm in final drug products. Traditional ELISAs measure total HCPs but cannot identify individual proteins, while conventional multiple reaction monitoring (MRM) suffers from retention‑time shifts...
How Inflammation May Prime the Gut for Cancer
Researchers at the Broad Institute and Harvard uncovered that chronic intestinal inflammation imprints lasting epigenetic scars on gut cells, even after tissue appears healed. In mouse models, these epigenetic memories persist through many cell divisions and, when paired with a...
Improving Early Detection and Treatment of Liver Cancer
Early-stage liver cancer survival hinges on timely diagnosis, yet ultrasound and AFP tests miss many cases. Helio Genomics has introduced the HelioLiver blood test, a multi‑analyte cfDNA and protein assay that flags tumor‑associated epigenetic signals earlier than imaging. A new...
Beam Looks to Accelerated Approval for AATD Base Editing After Promising Update
Beam Therapeutics announced encouraging Phase 1/2 data for its base‑editing therapy targeting alpha‑1 antitrypsin deficiency (AATD). The trial demonstrated a 30% reduction in the disease‑causing SERPINA1 mutation and a 45% rise in functional protein levels, while confirming a clean safety profile....
Guidance Recap Podcast | Use of Bayesian Methodology in Clinical Trials of Drug and Biological Products
The FDA released a draft guidance on using Bayesian methodology in clinical trials, outlining how sponsors should incorporate prior data, define success criteria, and document analyses. It emphasizes systematic prior construction, transparent operating characteristics, and extensive simulation documentation. The guidance...
Guidance Snapshot Pilot
The FDA has launched a Guidance Snapshot Pilot that distills complex guidance documents into visual, plain‑language one‑page snapshots complemented by podcasts, timelines, and social‑media links. The pilot targets cross‑cutting topics such as Bayesian trial methodology, decentralized clinical elements, and neonatal...
Boehringer Ingelheim Plans for Dealmaking and R&D Spending Amid US Price Pressure
Boehringer Ingelheim announced it will maintain its high‑level R&D spending while exploring dealmaking opportunities in China to counteract mounting U.S. drug‑price pressure. The company is prioritising an obesity‑focused pipeline, betting on the fast‑growing weight‑loss market to sustain revenue. By keeping...
Ozempic Pill Improves Multiple Cardiometabolic Risk Factors
A post‑hoc analysis of the SOUL trial shows that once‑daily oral semaglutide (Ozempic pill) significantly improves several cardiometabolic risk factors in adults with type 2 diabetes at high risk for atherosclerotic cardiovascular disease. Compared with placebo, participants experienced a 3.2 mm Hg greater...
Rentschler Highlights Milford Site Progress and Growth
Rentsc hler Biopharma announced that its Milford, Massachusetts CDMO site has entered a new growth phase, adding a 22,000‑square‑foot cleanroom and four 2,000‑liter single‑use bioreactors. The expansion, the largest in the company’s 150‑year history, brings the U.S. facility to the...
Pharma AI Shifts From Cloud to On‑Device
Most AI companies believe the future is cloud-first. Pharma is different. In this industry, data is the kingdom. Experimental data. Molecular designs. Clinical insights. Some of the most valuable intellectual property on Earth. And in many cases, it cannot leave the lab. This is where the typical...
Escitalopram Yields -16.6 HAM‑D Drop After 8 Weeks
Anyone have a PDF of this? Perhaps there isn't a digital copy... This one showed an impressive -16.6 HAM-D drop with 8 weeks of escitalopram. It would be good to know their methodology. https://t.co/P5GCYcbxjk
An Indication Selection Resource for Longevity Companies
Norn Group has released a free, detailed spreadsheet that maps 47 age‑related disease indications, providing mechanisms, incidence, market size, clinical endpoints, animal models, and trial cost estimates. The guide is designed to help longevity‑focused startups choose FDA‑approved disease targets rather...
Morgan Stanley Sets $20 Target, 120% Upside for IMMX
$IMMX - Morgan Stanley initiates coverage with a $20 PT and an outperform rating, implying ~120% upside. https://t.co/Pd4uIH5BkI
Engineered Immune Cells Target Refractory Myeloma, Broader Cancer Potential
Steady progress towards engineering immune cells in the body now for refractory myeloma , with major implications for many autoimmune diseases and cancers https://t.co/K3IksrCFlg https://t.co/1AQ5rLuH1g
Novo Reports More Triple-G Data From China; Grifols Plots IPO for Biopharma Unit
Novo Nordisk announced that its triple‑agonist candidate UBT251 achieved a mean HbA1c reduction of up to 2.16% after 24 weeks in a phase‑2 study of Chinese patients with type‑2 diabetes. The trial, involving roughly 200 participants, underscores the drug’s potential...
STAT+: Sarepta Therapeutics Shares Rise on Early Promise for Rare Disease Drugs
Sarepta Therapeutics reported that early‑stage trials of two experimental drugs, SRP‑1001 and SRP‑1003, demonstrated safety and signs of efficacy for rare muscle‑wasting disorders. The data sparked a more than 20% surge in the company’s stock during early trading. The results...
UCB Investing $2B To Build Manufacturing Facility in Georgia
UCB announced a $2 billion investment to build a 460,000‑square‑foot biologics manufacturing plant in Gwinnett County, Georgia, its first U.S. production site. The facility will create about 330 permanent jobs and over 1,000 construction positions, leveraging AI, robotics and automation. Georgia...
Xaira’s First Virtual Cell Model Is Largest To-Date, Toward Complex Biology
Xaira Therapeutics unveiled X-Cell, a 4.9‑billion‑parameter virtual cell model that predicts transcriptome‑level responses to genetic perturbations. The model leverages the company’s 25.6 million‑cell X‑Atlas/Pisces CRISPRi Perturb‑seq dataset and demonstrates zero‑shot performance on unseen T‑cell and iPSC contexts. X-Cell uses a diffusion...
GSK’s Two-Speed Strategy: Broad Sourcing and Selective Bets
GSK has accelerated its pipeline build‑out by pairing broad early‑stage partnerships with selective, later‑stage acquisitions such as the up‑to‑$950 million purchase of 35Pharma’s pulmonary‑hypertension candidate HS235. After spinning off Haleon, the company now leans on specialty medicines—accounting for over 40% of...
Why Maze Therapeutics Plunged 20%-Plus Despite 'Overwhelming Positive' Data
Maze Therapeutics reported Phase 2 results for its kidney drug MZE829, showing a 35.6% average reduction in proteinuria after 12 weeks and a 61.8% drop in the FSGS subgroup. The data were hailed as "overwhelmingly positive" but the stock fell more...
CEO Amy Burroughs Discusses M&A Post‑ASH25 Data
I asked $TERN CEO Amy Burroughs right after the #ASH25 data was out how she felt about M&A. Congrats to her and her team. https://t.co/2HemPoiqWO
New Treatments Target Faulty Genetic Heart Signals
A new DNA‑methylation (episignature) test can differentiate harmful from benign NOTCH1 variants in congenital heart disease, giving families definitive genetic answers. The assay scans over 740,000 genomic sites to identify a characteristic methylation pattern linked to disease‑causing mutations. Positive results...
Thousands of P-Values, only a Handful Are Real
1/ You ran 20,000 differential expression tests. 1,000 genes came back with p < 0.05. How many are real? Maybe 50. Maybe fewer. Most bioinformaticians learn p-values in stats class. Almost nobody learns why they break at scale. Let me explain: https://t.co/btC71ABX5N
Stanford Study Shows Gut‑Brain Modulation Reverses Age‑Related Memory Loss in Mice
Stanford Medicine and the Arc Institute reported that stimulating gut‑brain signaling and altering the microbiome reversed age‑related cognitive decline in mice, making old animals perform on memory tests like their younger peers. The finding points to a new, peripheral route...
Merck & Co. Nears $6 Billion All‑Cash Deal for Terns Pharma, Boosting Oncology Portfolio
Merck & Co. is in advanced negotiations to acquire U.S. biotech Terns Pharma for an all‑cash price of about $6 billion. The news lifted Merck shares 0.5% to $116.99 and sent Terns stock soaring 12% to $55.97, underscoring the deal’s market...
Microneedle Vaccine Patch Company Raises $50M for Pivot to GLP-1 Delivery
Terrestrial Bio, the microneedle vaccine patch pioneer originally founded as Vaxess Technologies, announced a $50 million Series B financing round to shift its focus from vaccines to GLP‑1 peptide delivery. The capital, led by a consortium of biotech investors, will fund clinical...
FDA Halts Elevidys Trial, Fueling New Right‑to‑Try Debate for Duchenne Families
A mother’s plea after the FDA halted the Elevidys gene‑therapy trial for her son with Duchenne muscular dystrophy has reignited criticism of the 2018 Right‑to‑Try Act. The agency’s decision, triggered by two deaths in a broader trial, cuts off a...
MRK Likely to Rerun TERN Study for Data
Based on previous form $MRK will probably now rerun the $TERN study, just to prove to itself that the data were real.
TERN's Termination Terms Hint MRK Could Be Outbid
For those wondering if $MRK might be outbid, here are the deal termination terms from $TERN https://t.co/TbM3rWyEoG
Study Shows How Lymph Node Architecture Affects Cancer Growth
Researchers from EMBL Heidelberg and partner institutions have created the first detailed map of immune and stromal cell organization within human lymph nodes, revealing how this architecture deteriorates in lymphomas. They discovered an inflammatory vicious cycle where T‑cell interferon signals...
Merck Spends $6.7B on CML Drug Despite Gleevec Era
It's amazing that 25 years after Gleevec we are still watching drug companies pay billions for new drugs for CML. And that, in Adam's previous story, a once deadly cancer was referred to as a chronic disease. Anyway, $MRK buys $TERN...
Amy Burrough
Adding $TERN to this list (Merck $6.7B reported) led by Amy Burroughs. Congrats to Amy and team 💪 https://t.co/KZ8CtA2GA0?
RA Capital Looks to China for Next Startup To Put on SPAC Track to Nasdaq
RA Capital Management has filed a prospectus for a new SPAC, Research Alliance III, with up to $57.5 million of capital. The blank‑check vehicle is openly scouting life‑science startups, emphasizing Chinese drug developers as potential targets. While the SPAC has not yet...
AVXL Withdraws Alzheimer's Drug From EMA After Rejection
$AVXL pulled its blarcamesine Alzheimer's application from the EMA after regulators there said there was no fking way it was going to approve a drug that does not work.