STAT+: Revolution Medicines Touts ‘Unprecedented’ Data for Pancreatic Cancer Pill
Revolution Medicines reported that its oral KRAS‑G12C inhibitor daraxonrasib more than doubled survival for patients with metastatic pancreatic cancer. In a head‑to‑head trial, the daily pill yielded a median overall survival of 13.2 months versus 6.7 months for standard chemotherapy. The result, described as "unprecedented" by analysts, marks a rare breakthrough in a disease where median survival is typically under a year. The data could reshape treatment standards and accelerate regulatory review.
STAT+: Allogene Therapeutics’ CAR-T Treatment Eliminates Residual Cancer Cells in B-Cell Lymphoma Patients
Allogene Therapeutics reported that its off‑the‑shelf CAR‑T therapy, cema‑cel, eliminated residual cancer cells in B‑cell lymphoma patients at three times the rate of standard care, meeting the interim goal of its Phase 3 trial. In the interim analysis, 58% of treated...
STAT+: Spyre Therapeutics IBD Drug Shows Promise in Early Trial
Spyre Therapeutics reported positive Phase 2 data for its ulcerative colitis candidate SPY001, showing safety and meeting the trial's primary endpoint. In the SKYLINE study, patients experienced a 9.2‑point drop in a disease‑activity index, and roughly 40% entered remission after 12...
STAT+: 5 Years After Lupus Breakthrough, CAR-T Is Still Surprising Autoimmunity Researchers
Five years after a pioneering CAR‑T treatment rescued a teenage lupus patient, the therapy has sustained remission and reshaped expectations for autoimmune disease management. The case, led by German rheumatologist Georg Schett, proved that engineered T cells could safely target...
STAT+: Biotech VCs, Used to a Winning Formula in Drug Development, Face Disruption
For decades, biotech venture capitalists have followed a formula—seed university science, add seasoned pharma executives, and invest tens of millions—to generate drugs and returns. That playbook is now under pressure as Chinese biotech firms deliver research faster and cheaper, and...
STAT+: A Decade Ago, These Drugs Tore Apart the FDA. Today, They Might Be some Patients’ Best Hope
Exon‑skipping therapies for Duchenne muscular dystrophy, once a source of controversy at the FDA, are now delivering unexpected clinical benefits. A recent trial involving 39 patients, including 5‑year‑old Hawken Miller, showed functional improvements that have surprised leading experts. The drugs,...
STAT+: Many Cancer Patients Don’t Get Genomic Tests to Guide Treatment, Study Finds
Genomic sequencing, a key component of precision oncology, is underused in metastatic cancer care. A JAMA Network Open study of five cancer types found that only about half of patients received tumor genetic testing, with lower rates among low‑income, Medicare/Medicaid,...
STAT+: Stealth Biotech Stipple Bets on Secretive ADCs
Stealth biotech Stipple is quietly advancing secretive antibody‑drug conjugates (ADCs) as the market races toward targeted cancer therapies. A recent four‑month FDA review delay forced a cash‑strapped small biotech to shut down, underscoring the existential risk of regulatory setbacks for...
STAT+: How a Four-Month FDA Delay Forced a Small Biotech Company to Close Its Doors
Kezar Life Sciences, a small biotech developing a treatment for autoimmune hepatitis, saw a critical FDA meeting cancelled four months late, derailing its trial timeline. The delay forced investors to withdraw, prompting the company to lay off most of its...
STAT+: Lilly’s Obesity Pill Enters the Oral GLP-1 Game, Novo Responds
The FDA approved Eli Lilly’s oral GLP‑1 obesity pill orforglipron, marking the first FDA‑cleared oral weight‑loss drug. The approval puts Lilly into direct competition with Novo Nordisk, which is developing its own oral GLP‑1 candidate. Simultaneously, a draft Trump administration order could...
STAT+: Trump Administration Prepares 100% Tariffs on some Imported Drugs
The Trump administration is poised to issue an order that would levy a 100% tariff on imports of patented medicines and their active pharmaceutical ingredients. A draft of the order suggests the tariffs could be announced as early as Thursday,...
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...
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...