Know What's Happening in Pharma
Know What's Happening in Pharma
FDA approves KRESLADI, first CIRM‑funded gene‑editing therapy for LAD‑I
The FDA has granted approval to KRESLADI, a gene‑editing therapy from Rocket Pharmaceuticals that treats severe leukocyte adhesion deficiency‑I in children without a matching bone‑marrow donor. This marks the first FDA‑approved product directly funded by California’s Institute for Regenerative Medicine (CIRM), highlighting the agency’s impact on translational research.
Also developing:
By the numbers: Lupin Limited acquires VISUfarma from GHO Capital
The Xtalks Life Science Podcast featured Dr. Steve Levine, Chief Patient Officer at Compass Pathways, discussing the company’s push to develop psilocybin‑based therapies for treatment‑resistant depression (TRD). Levine, a board‑certified psychiatrist and founder of Actify Neurotherapies, highlighted the clinical promise of psychedelic medicine and the need for scalable patient‑access models. The episode also explored broader gaps in mental‑health care and how evidence‑driven innovation can address them. Listeners can stream the interview on Spotify, Apple Podcasts, and other platforms.
The FDA issued a warning letter to ImmunityBio for misleading promotional claims about its bladder‑cancer drug Anktiva, triggering a roughly 26 percent drop in the company’s shares and giving it 15 days to submit a corrective plan. In parallel, Merck announced a...
Researchers at the University of Guelph announced that their sugar‑based vaccine candidate against Campylobacter jejuni demonstrated safety and immunogenicity in a small Phase 1 human trial. Participants experienced only mild side effects, and the formulation generated measurable antibody responses even at...
Inspired by the man who built a personalized cancer vaccine for his dog, I’ve written an open-source guide to DIY mRNA vaccine production: philfung.github.io/openvaxx Drawing on my background in running lab startups, the guide covers the entire process - from sequencing...
Anavex Life Sciences withdrew its European marketing authorisation application for blarcamesine, an add‑on therapy for early‑stage Alzheimer’s, after the EMA’s CHMP signaled a likely negative opinion. The committee criticized the trial’s efficacy data, methodological flaws, and safety reporting, including concerns...
The FDA’s Center for Drug Evaluation and Research (CDER) released its FY 25 Drug Quality Sampling and Testing results, showing that the vast majority of tested pharmaceuticals met USP specifications. The program uses a risk‑based, data‑driven approach introduced in 2018 to...
One thing that really makes the in-person summits we run at STAT so amazing: the people in the room. Here's what happened when we asked some of them for the last big breakthrough they saw. Featuring: the infectious @DrBlytheAdamson, genomcis pioneer...
A Senate investigation led by Sen. Ron Johnson uncovered that federal health officials identified a statistically significant ischemic stroke risk associated with the Pfizer COVID‑19 booster for adults 65 and older as early as November 2022. Internal HHS records show...
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,...
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...
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...
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...
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...
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,...
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...
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 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, 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 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 (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
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...
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 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....
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...
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 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...
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...
Fujitsu and the University of Osaka have unveiled a new technology that couples the third version of Fujitsu’s STAR quantum architecture with a novel molecular model optimization technique. The combined approach dramatically lowers the quantum resources needed for chemical energy...
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...
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...
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...
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 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 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 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...
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...
Merck announced a $6.7 billion cash deal to acquire Terns Pharmaceuticals, paying a 31% premium and targeting the oral BCR::ABL1 inhibitor TERN‑701 for treatment‑resistant chronic myeloid leukemia. The transaction, slated to close in Q2 2026, adds an orphan‑drug‑designated oncology asset to Merck’s...
The U.S. Federal Trade Commission has filed a proposed settlement with CVS Caremark, accusing the pharmacy‑benefit manager of artificially inflating insulin prices and limiting patient access. The deal, which still requires FTC chair approval, mirrors a recent settlement with Cigna’s...
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...
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...
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...
The view from my hotel room in Chamonix is quite spectacular. I spoke yesterday at a global event organized by a major pharma company. They organized it near Mt. Blanc to make the country managers literally aim higher and higher. I...
When people ask "How can we make #AAV #GeneTherapy accessible and affordable" --> this is how 👇
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
See this for just 1 of the TAD trials from recent @psybalazs and @QuantPsychiatry comparative analysis. 1 of several deck stacks favoring TADs. This trial reported a surprising -15.2 HAM-D drop with venlafaxine. Not so surprising when you look closer......
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
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
Based on previous form $MRK will probably now rerun the $TERN study, just to prove to itself that the data were real.
For those wondering if $MRK might be outbid, here are the deal termination terms from $TERN https://t.co/TbM3rWyEoG
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...
