STAT+: Insilico Medicine CEO on How Best to Use AI in Drug Development
Insilico Medicine, a veteran AI‑driven drug discovery firm, announced a partnership with Eli Lilly that includes a $115 million upfront payment and up to $2.75 billion in milestone‑based total consideration. The deal leverages Insilico’s generative‑AI platform to co‑develop novel therapeutics, primarily targeting metabolic diseases. Insilico’s CEO emphasized a pragmatic AI philosophy that blends deep learning with traditional pharmacology to shorten timelines and improve hit rates. The collaboration marks one of the largest AI‑centric contracts in biotech to date.
STAT+: The Biotech Scorecard for the Second Quarter: 23 Stock-Moving Events to Watch
STAT’s quarterly biotech scorecard lists 23 upcoming events that could move biotech stocks in Q2 2026. Highlights include Phase 3 results from Abivax’s obefazimod in ulcerative colitis and Allogene Therapeutics’ interim data on its cema‑cel CAR‑T therapy for B‑cell lymphoma. The...
STAT+: Over-the-Top Psychedelic Promos Could Undermine the Field’s Drug Development Efforts
Psychedelic biotech firms Helus Pharma and AtaiBeckley have hired marketing agencies to produce YouTube videos that tout exaggerated efficacy claims and alleged FDA fast‑track status for their investigational drugs. The ads, labeled only as “informational,” suggest near‑perfect Phase 2 results and...
STAT+: In Private Meetings, White House Works to Win Pharma Companies’ Support for Drug Pricing Bill
The White House has drafted a drug‑pricing bill and is privately meeting with more than a dozen major pharmaceutical companies to secure their backing. The proposed legislation mirrors voluntary pricing agreements the administration previously struck, and notably would allow cash‑paid...
STAT+: FDA Approves Rocket Gene Therapy for Rare Immune Disorder
The FDA has granted approval to Rocket Pharma's gene therapy Kresladi for severe leukocyte adhesion deficiency type 1 (LAD‑1), an ultra‑rare immune disorder. The therapy was previously rejected in 2024 due to manufacturing concerns, but the agency cleared it after the...
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...
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...
STAT+: How an Outsider Crept Into Eli Lilly’s Top Ranks — and Plans to Drive Its Business Forward
Jacob Van Naarden, formerly COO of Loxo Oncology, joined Eli Lilly after its $8 billion cash acquisition of Loxo in 2018. He now serves as president of Lilly Oncology and head of business development, overseeing the company’s dealmaking and pipeline expansion. The purchase...
STAT+: Insmed Drug Benefits Patients with Rare, Bacterial Lung Disease, Study Shows
Insmed announced that a Phase 3 trial showed adding its inhaled antibiotic Arikayce to standard therapy significantly improved respiratory symptoms and boosted culture conversion rates in patients with newly diagnosed mycobacterium avium complex (MAC) lung infection. The study met its primary...
STAT+: Pfizer’s Lyme Vaccine Shows Efficacy, but Misses Key Statistical Hurdle
Pfizer and Valneva’s experimental Lyme vaccine cut the risk of infection by more than 70% in a late‑stage trial, offering a promising preventive tool for a disease that affects roughly 476,000 Americans annually. The study, however, missed its primary statistical...
STAT+: Apogee Therapeutics Data Show Long-Acting Eczema Drug Induced Relief with Less Frequent Injections
Apogee Therapeutics reported that its experimental long‑acting eczema biologic, zumilokibart, achieved sustained skin‑clearance in a mid‑stage trial. Seventy‑five percent of patients receiving the drug every three months and 85 % of those dosed every six months maintained an EASI‑75 response after...
Drug Development Is Booming in China. Should the U.S. View It as a Threat or an Opportunity?
China’s biotech sector is experiencing a rapid surge, now hosting more CAR‑T cell trials than the United States. The growth is driven by a dual‑track regulatory framework that enables fast‑track, investigator‑initiated trials with minimal red tape. U.S. experts warn that...
STAT+: Eli Lilly’s ‘Triple-G’ Drug Leads to Significant Blood Sugar, Weight Reductions in Diabetes Trial
Eli Lilly’s investigational injectable retatrutide achieved a 1.9‑point HbA1c reduction versus 0.8 points for placebo after 40 weeks, while participants on the highest dose shed 15.3% of body weight compared with 2.6% on placebo. The weight loss was still progressing at...
STAT+: Clearing Tumors in Mice, Azalea Therapeutics Advances Dream of in Vivo CAR-T Therapy
Azalea Therapeutics, a spinout from Jennifer Doudna’s lab, reported in Nature that its in vivo CAR‑T approach can generate functional CAR‑T cells directly within mice and eradicate both solid and hematologic tumors. The technique uses infused gene‑editing particles that precisely...
STAT+: A Huntington’s Researcher on the UniQure-FDA Fray
UniQure’s experimental gene‑therapy for Huntington’s disease, which previously reported a 75% slowdown in disease progression, has received a third consecutive rejection from the FDA. The trial’s lead investigator, Ed Wild of University College London, praised the early data but warned...
STAT+: J&J Wins Approval for First-of-Its-Kind Psoriasis Pill
Johnson & Johnson received FDA clearance for Icotyde, the first oral daily pill for moderate‑to‑severe plaque psoriasis. The drug, originally called icotrokinra, is approved for patients aged 12 and older and is designed to replicate the efficacy of injectable biologics...
STAT+: VC Firm Dimension Scouting Out $700 Million for New Fund
Dimension Ventures, founded by former Lux Capital and Obvious Ventures partners, is launching its third fund targeting the intersection of artificial intelligence and life sciences. The firm aims to raise roughly $700 million, following a 2023 debut fund and a second...
STAT+: White House Digs in on ‘Most-Favored Nation’ Drug Pricing Despite Congress’ Cool Reception
The White House is intensifying pressure on Congress to pass a “most‑favored nation” drug‑pricing bill that would cap U.S. medication prices at levels paid by peer nations. Administration officials say the proposal would overhaul pricing for providers, insurers, federal programs...
STAT+: Structure Therapeutics Reports Significant Weight Loss From Mid-Stage GLP-1 Pill
Structure Therapeutics announced that its daily oral GLP‑1 obesity pill produced an average 16% body‑weight reduction versus placebo after 44 weeks in a Phase 2 trial. The result outperforms Eli Lilly’s orforglipron, which showed about 11% loss over 72 weeks, and rivals...
STAT+: Cancer Cells Can ‘Barf’ Proteins Onto Their Cell Surface. That May Create New Targets for Immunotherapies
Researchers at UCSF discovered the Src kinase, traditionally an intracellular signaling protein, displayed on the outer membrane of malignant cells. The finding, published in Science, showed surface Src was absent from healthy donor tissue, suggesting a tumor‑specific marker. This unexpected...
STAT+: FDA Approves Leucovorin for Rare Disorder without Trial Data
The FDA has granted approval for leucovorin, a folinic‑acid formulation, to treat a rare metabolic disorder despite the absence of new clinical trial data. The decision leans on decades of off‑label use and historical safety records rather than prospective studies....
STAT+: Novo Nordisk Is Warned by the FDA for Failing to Report Side Effects Tied to GLP-1 Drugs
Novo Nordisk received an FDA warning letter on March 5, 2026 for failing to report suspected side effects of its GLP‑1 medicines. The violations were uncovered during a 2025 inspection of the company’s facilities and were described as “serious.” The agency warned...
STAT+: The FDA, Urged to Avoid Controversy, Creates a New Headache with Attack Against UniQure
The FDA staged a media call where an anonymous senior official publicly criticized UniQure’s experimental Huntington’s disease gene therapy. The official, identified only as a practicing hematology‑oncology professor, framed his comments as serving the public interest while hinting at personal...
STAT+: Servier to Acquire Day One, Maker of Pediatric Cancer Drug, for $2.5B
Servier announced a $2.5 billion cash deal to acquire Day One Biopharmaceuticals, paying $21.50 per share—a 68% premium to the biotech’s closing price. The acquisition centers on Day One’s Ojemda, the first FDA‑approved therapy for pediatric low‑grade glioma, the most common...
How the ‘Holy Grail’ Weight Loss Pill Became a Reality, and What Comes Next
The pharmaceutical industry has finally delivered an oral GLP‑1 weight‑loss pill, with Novo Nordisk launching an oral version of Wegovy earlier this year. Eli Lilly’s oral GLP‑1 candidate, orforglipron, is expected to receive approval imminently. Oral formulations overcome the injection barrier that...
STAT+: FDA Is ‘Not Convinced’ UniQure’s Huntington’s Therapy Has Benefit, Senior Official Says
The FDA concluded that UniQure’s experimental gene therapy for Huntington’s disease has not demonstrated therapeutic benefit based on existing clinical data. Reviewers said they are not persuaded by the evidence, prompting the agency to block the company from submitting a...
STAT+: FDA Rejection Is a Reality Check on Agency Rhetoric
The U.S. Food and Drug Administration rejected a rare‑disease cell therapy that had already secured approval in Europe, despite earlier internal support from the agency. The decision comes under the FDA’s new leadership and has sparked debate over whether the...
STAT+: Rare Disease Advocates Fume over FDA’s Mixed Signals
The FDA has rejected Regenxbio’s gene therapy for mucopolysaccharidosis type 2, citing a need for additional data that could postpone approval by years. This decision adds to at least four other recent rejections of cell or gene therapies targeting deadly rare...
STAT+: Sarepta Therapeutics CEO Doug Ingram Will Retire After a Tumultuous Decade
Sarepta Therapeutics CEO Doug Ingram announced his retirement, ending a decade that lifted the biotech to a $15 billion valuation by securing approvals for three Duchenne muscular dystrophy (DMD) therapies. The company’s market value subsequently collapsed amid a safety investigation into...
STAT+: A Rare Disease Drug Was Approvable, Then It Wasn’t. Inside a Surprise Rejection by the FDA
An experimental cell therapy for a rare post‑transplant blood cancer, developed by Atara Biotherapeutics and Pierre Fabre, was initially deemed approvable by FDA reviewers but was abruptly rejected in February 2026 over alleged clinical data deficiencies. The disease affects roughly...
STAT+: Novo Doubles Down on Oral Peptides
Novo Nordisk announced a major expansion of its oral peptide platform, committing additional capital to bring next‑generation oral GLP‑1 candidates to market within the next few years. At the same time, Alkermes CEO Richard Pops will retire after a 35‑year...
STAT+: Drugmakers Lay Out Their Legal Arguments Against Trump’s Drug Pricing Experiments
Pharmaceutical and biotech firms are mobilizing against two Trump administration proposals that would tie Medicare drug prices to those paid in other affluent nations. The initiatives – the GLOBE model for Part B physician‑administered drugs and the GUARD model for Part D...
STAT+: New Treatment Approach Could Give IBD Patients Hope, and Be a Bonanza for Drugmakers
A new combination‑therapy approach for inflammatory bowel disease (IBD) aims to boost remission rates beyond the current 30 % success benchmark. By pairing agents that target distinct immune pathways, early trials show remission climbing to roughly 55 % and faster mucosal healing....
STAT+: Gossamer Lung Disease Drug Fails Late-Stage Study, but Company Will Still Seek FDA Approval
Gossamer Bio announced that its Phase 3 trial of seralutinib for pulmonary arterial hypertension missed its primary endpoint. Patients on seralutinib walked 13 meters farther than placebo over six minutes, but the difference lacked statistical significance. The trial also reported cough...
STAT+: Gilead to Buy Arcellx in Nearly $8B Deal
Gilead Sciences announced a $7.8 billion acquisition of Arcellx, pricing the deal at $115 per share—a 79% premium to the prior close. The agreement includes an additional $5 per share contingent on future sales milestones. Central to the transaction is anito‑cel,...
STAT+: Key Study of Grail’s Cancer Detection Test Fails in Setback for Company
Grail’s multi‑cancer blood test Galleri failed to meet its primary endpoint in a large NHS‑partnered study, casting doubt on its early‑detection claims. The test, priced at $1,000, generated $136.8 million from 185,000 units sold in 2025 but remains unapproved by the...
STAT+: In First Speech to Her FDA Staff, Høeg Says She’ll Scrutinize RSV Shots and SSRIs in Pregnancy
FDA Commissioner Tracy Beth Høeg, in her inaugural staff address, announced a renewed focus on evaluating the safety of antidepressants prescribed during pregnancy and monoclonal antibody RSV prophylaxis for infants. She highlighted gaps in existing safety monitoring and pledged more...
STAT+: What to Expect From Gossamer Bio’s Late-Stage Lung Disease Study
Gossamer Bio is set to announce results from its Phase 3 trial in pulmonary arterial hypertension (PAH) before the end of February. The readout follows a Phase 2 study that delivered modest, sub‑par efficacy, which the company attributes to an...
STAT+: The FDA’s Moderna Pirouette Is One More Sign of Chaos at the Agency
The FDA initially issued a refuse‑to‑file letter for Moderna’s mRNA influenza vaccine, then reversed course within a week and agreed to review the product. The agency will now consider two separate pathways: full approval for adults 50‑64 and accelerated approval...
STAT+: Biotech Startup Raises $175 Million to Advance Alzheimer’s Antibody Therapy
Korsana Biosciences announced a $175 million financing round led by Fairmount, Venrock, Wellington Management and TCGX to develop its Alzheimer’s antibody candidate KRSA‑028. The startup positions KRSA‑028 as a next‑generation “brain shuttle” designed to clear amyloid plaques more efficiently than existing...
STAT+: Ocular’s Experimental Eye Drug Beats Low Dose of Regeneron’s Eylea in Late-Stage Trial
Ocular Therapeutix announced that its experimental drug Axpaxli met the primary endpoint in a Phase 3 trial for wet age‑related macular degeneration, maintaining vision in 74% of patients at nine months and 66% at one year after a single injection....
STAT+: Compass Says Its Psilocybin Drug Helped Patients with Severe Depression in Two Trials
Compass Pathways announced that its synthetic psilocybin therapy, COMP360, achieved statistically significant improvements in two identical Phase 3 trials for severe major depressive disorder. Both studies met primary endpoints, showing greater reduction in depression scores than placebo‑controlled psychotherapy. The data suggest...
STAT+: As China’s Drug Industry Races Ahead, Its GLP-1 Race Is Accelerating Too
China’s pharmaceutical sector is rapidly expanding, now generating its own GLP‑1 drugs alongside imported treatments. Novo Nordisk reported a 5% decline in Ozempic sales in China last year, the first dip since its 2021 launch, while sales grew elsewhere. The...
STAT+: FDA Rejects Rare Disease Therapy From Disc Medicine, Early Recipient of Commissioner’s Voucher
The FDA rejected bitopertin, Disc Medicine’s experimental therapy for acute hepatic porphyria, marking the first drug reviewed under Commissioner Marty Makary’s fast‑track voucher program. The agency cited uncertainties about the link between the trial’s blood‑based biomarker and actual clinical benefit....
STAT+: Pediatricians Confront HHS in Vaccine Showdown
The FDA unexpectedly refused to review Moderna’s mRNA‑based flu vaccine, sparking alarm across biotech firms. Pediatricians are confronting the Department of Health and Human Services (HHS), demanding transparent guidance on pediatric flu immunization. The decision highlights perceived regulatory volatility under...
STAT+: BridgeBio Drug for Genetic Cause of Dwarfism Succeeds in Key Study
BridgeBio Pharma's oral drug infigratinib met its primary endpoint in a pivotal trial of more than 100 children with achondroplasia, delivering an average growth increase of 2.1 cm per year versus placebo. Adjusted analysis showed a 1.74 cm per year advantage, both...
STAT+: European VCs Band Together to Improve the Landscape for Biotech Startups
European venture capital firms have launched the European Life Sciences Coalition, a new alliance aimed at strengthening funding pipelines for biotech startups across the continent. The coalition highlights a stark financing gap: of the 67 EU‑based biotech firms that went...
STAT+: The Unusual Prasad Missive in the FDA’s Rejection of the Moderna Flu Shot Application
Moderna announced that the FDA rejected its mRNA‑based flu vaccine, a move that caught the company off guard. The decision sparked a public critique from oncologist Vinay Prasad, who questioned the agency’s evaluation process. At the same time, regulators are...
STAT+: Eczema Drug From Nektar Therapeutics Maintains Skin Responses in One-Year Study
Nektar Therapeutics reported that its experimental eczema biologic rezpeg sustained stringent skin‑lesion improvements in a year‑long maintenance study. Patients receiving monthly injections maintained responses in 71% of cases, while quarterly dosing achieved an 83% maintenance rate. The data met or...
STAT+: Biotech Doubles Financing in Quest to Silence Genes Behind Neurological Diseases
Aerska, a Dublin‑London biotech, announced a $39 million financing round, just four months after its initial seed raise. The company’s platform uses iron‑mimicking antibodies to ferry siRNA molecules across the blood‑brain barrier, a technique it calls a “brain shuttle.” By silencing...
