#Drug development

Ultragenyx reported that its antisense oligonucleotide candidate for Angelman syndrome did not meet the primary endpoint in a Phase 3 trial. The failure represents a significant blow to efforts to develop a disease-modif…

Top officials at the US Food and Drug Administration are intensifying oversight of clinical trial data collected outside the country, aiming to verify its adequacy and reliability. The move responds to growing pressure f…

Newly released FDA documents show that Asha Das, director of the biologics center's clinical evaluation office, overrode the internal review team's rejection to grant accelerated approval to Replimune's melanoma drug Tud…

The deaths of two children in China who received CRISPR-based treatments have intensified scrutiny of viral vectors used to deliver such therapies. U.S. scientists working on similar genetic medicines are reassessing the…

Teva Pharmaceuticals reported that a single injection of its experimental antibody significantly reduced intestinal damage in celiac disease patients who consumed gluten daily. The topline results from the gluten challen…

The UK's Medicines and Healthcare products Regulatory Agency has suspended sales of Tavneos, following a similar action by the European Medicines Agency. UniQure has submitted a gene therapy for Huntington's disease for …

Typewriter has raised $56 million to develop a gene-editing approach using transposable elements, which could enable the delivery of full genes into the body without viral vectors or CRISPR enzymes. The funding will supp…

ARPA-H has allocated up to $125 million across five groups to develop on-demand manufacturing for personalized RNA therapies. The initiative aims to make custom genetic treatments available within days rather than months…

Arsenal Biosciences is reducing its headcount again, letting go of most employees as it shifts its focus toward in vivo CAR-T therapies. This follows a previous layoff of half its staff about a year ago. The company's st…

Eli Lilly has completed its 13th acquisition of 2026, with at least 50 distinct venture investors receiving returns from the company's dealmaking this year. The activity is funneling early-stage investment dollars back i…

Roche has entered into a collaboration with Chinese biopharma Simcere Zaiming, marking its second such deal with a Chinese company in two weeks. The agreement involves a preclinical trispecific T cell engager, with Roche…

GSK is progressing its mRNA-based seasonal flu vaccine into a Phase 3 study following a Phase 2 trial that indicated stronger immune responses compared with conventional vaccines. The UK-based pharmaceutical company disc…

In two identical late-stage studies, Novartis' experimental BTK inhibitor Rhapsido markedly lowered the frequency of multiple sclerosis relapses. The oral therapy, already approved for chronic itch, could now gain a foot…

ARCH Venture Partners, a prominent biotech investment firm, is aiming to secure $3 billion for its next venture fund. If successful, the 14th fund would rank among the largest capital pools in the life sciences sector. T…

Novartis has halted trials of its CD19-directed CAR-T therapy after three patient deaths linked to a severe immune response. Bristol Myers Squibb has also paused a similar program to review safety concerns. The pauses hi…

Electra Therapeutics, backed by Sanofi, has submitted an IPO filing with U.S. regulators, aiming to begin trading on the Nasdaq after the Labor Day holiday. The company plans to use the proceeds to support a Phase 2/3 tr…

Eli Lilly has agreed to acquire Merida Biosciences, a startup focused on immunology, in a deal valued at up to $2.875 billion. This marks Lilly's 13th acquisition this year, underscoring its aggressive push into immune-r…

Alnylam is defending its heart disease therapies following a failed clinical trial of AstraZeneca's Wainua. The company contends that its own drugs operate through different mechanisms and remain clinically valuable. The…

At the ESC 2026 meeting in Munich, Arrowhead presented results from two Phase 3 trials of its RNA interference drug Redemplo in patients with elevated triglycerides. The therapy demonstrated efficacy and a favorable safe…

At the ESC 2026 meeting in Munich, AstraZeneca presented results from a mid-stage trial of its oral relaxin agonist for heart failure. The drug improved cardiac function in a subset of patients, though not across the ent…

AstraZeneca analyzed the unexpected failure of a major heart disease trial involving a silencer drug. The outcome has implications for other companies developing similar therapies in a competitive market. The findings ra…

Cytokinetics announced successful trial data for a potential new drug. The company, which took 27 years to get its first approval, may now see a faster path for this candidate. The results could lead to a second approval…

This newsletter covers recent pharmaceutical industry news, including a failed AstraZeneca trial and a controversial conflict of interest. It also highlights other developments in the sector. The items are curated from t…

A drug acquired by Novartis reduced markers of inflammation in a trial. The key question now is whether this translates into better cardiovascular outcomes for patients. Further studies will determine if the treatment ca…

In the AMUNDSEN trial, patients with acute myocardial infarction who received an immediate injection of evolocumab before percutaneous coronary intervention were more likely to achieve LDL cholesterol targets at 1 year t…

In the LIBREXIA ACS trial, milvexian did not reduce the composite of cardiovascular death, myocardial infarction, or ischemic stroke compared with placebo in patients with recent acute coronary syndrome on antiplatelet t…

In the SINGLE-AF trial, patients with atrial fibrillation and one thromboembolic risk factor who received a direct oral anticoagulant had a lower rate of composite cardiovascular events at 24 months than those without an…

A phase III trial involving 517 patients with symptomatic nonobstructive hypertrophic cardiomyopathy found that aficamten significantly boosted peak oxygen uptake and patient-reported health status at 36 weeks compared w…

AstraZeneca and Ionis have disclosed a surprising failure in a clinical trial for a heart disease drug. The unexpected outcome has drawn attention from Alnylam, which is monitoring the situation. The news is part of a br…

The FDA has approved rusfertide, now to be sold as Mimrylo, for the treatment of polycythemia vera. The drug, developed by Takeda and Protagonist Therapeutics, addresses a rare blood disorder characterized by overproduct…

CRISPR Therapeutics presented one-year data from an early-stage trial of its gene editing treatment for high cholesterol, showing inconsistent durability. Despite the mixed results, the company plans to advance the highe…

The FDA granted Eli Lilly's Mounjaro an expanded approval to lower the risk of serious heart problems. In a separate deal, Teva plans to purchase an existing approved medication. The announcement also touched on developm…

A high-profile study of AstraZeneca and Ionis Pharmaceuticals' Wainua for heart disease failed unexpectedly, according to the trial's lead investigator. The failure may be linked to patients concurrently taking a Pfizer …

At the European Society of Cardiology meeting, Cytokinetics unveiled detailed data supporting Myqorzo for a specific heart muscle condition. The company expects to pursue an expanded approval next year, which could doubl…

Roivant Sciences received FDA approval for brepocitinib, marketed as Lisraya, to treat dermatomyositis. This is the first oral therapy for the autoimmune condition and marks a significant regulatory win for the company. …

Moderna is offering convertible senior notes to raise $2 billion, aiming to fund its oncology expansion and repay debt. Separately, AstraZeneca and Amgen, along with Biokin, announced positive Phase 3 trial results. Addi…

Eli Lilly has decided to no longer publicly disclose its Phase 1 pipeline, a shift from its previous transparency. The move comes as the company's GLP-1 drug leads global sales and its market valuation tops all other hea…

Lonza has established a new executive role for commercial operations, appointing Samanta Cimitan, currently CEO of Celonic, to the position effective January 1. The move is intended to strengthen the company's management…

Generate:Biomedicines presented initial clinical data for its respiratory disease candidate at the European Respiratory Society meeting, after its posters were accidentally made public two weeks early. The company had in…

Executives argue that a proposed law aimed at shielding U.S. biotech from Chinese rivals contains a significant gap. The loophole could allow companies to circumvent the restrictions through certain geographic arrangemen…

The personalized cancer vaccine from Moderna and Merck is seen as a major advance. The key to its effectiveness might be a proprietary algorithm that guides its design. This algorithm could be as important as the biologi…

The European Society of Cardiology meeting will unveil results from the STAREE trial, which examined statin use for primary prevention in adults aged 70 and older. Other studies will address heart attack triage and new a…

Elevated levels of Lp(a), a common cardiovascular risk factor, currently lack effective drug therapies. Several pharmaceutical companies are advancing candidates toward market approval, aiming to address this unmet need.

A retrospective study of 300 patients with ulcerative colitis found that those using liraglutide or semaglutide had significantly higher remission rates at 4, 8, and 12 weeks compared with non-users. The association rema…

In an interview, Insitro's Daphne Koller explained how artificial intelligence can streamline clinical trial processes and reduce delays. The discussion also covered broader applications of AI in health care.

Biotech observers are awaiting crucial trial results for a novel category of heart disease drugs. The upcoming data could shape future treatment options.

Haisco Pharmaceutical has granted US startup Sentivera ex-China rights to an unnamed inflammatory disease drug. Separately, ViiV Healthcare's HIV treatment has received approval for use in newborns. The two developments …

Biohaven has entered a licensing agreement with South Korea's SK Biopharmaceuticals for its epilepsy drug candidate. The deal could be worth up to $795 million, covering global rights to Biohaven's Kv7 ion channel platfo…

Cellares is disputing Bristol Myers Squibb's characterization of its cell therapy manufacturing technology after the drugmaker terminated a contract. The startup says the technology meets standards, pushing back against …

Aurora Therapeutics, which pioneered bespoke CRISPR-based treatments, has cut staff and halted its primary clinical program. The company had been applying base editing to correct specific genetic mutations. Its future no…

Spyre Therapeutics is deprioritizing its anti-TL1A antibody for rheumatoid arthritis after the drug failed to meet the company's threshold for success in a mid-stage trial. The decision casts doubt on the broader potenti…

San Diego-based Faro AI has raised $37 million in a Series B round to expand its platform that applies artificial intelligence to accelerate clinical trial execution. The company focuses on the post-discovery phase, aimi…

Massachusetts' biopharmaceutical sector experienced its first employment decline since 2002 last year, but 2026 has seen a significant recovery. The turnaround is attributed to renewed investment and pipeline activity. T…

The FDA approved zanidatamab (Ziihera) combined with tislelizumab and chemotherapy as first-line treatment for unresectable or metastatic HER2-positive gastroesophageal adenocarcinoma. The approval is based on the phase …

The FDA approved nipocalimab (Imaavy) for warm autoimmune hemolytic anemia in adults and adolescents who have used corticosteroids. The drug blocks the neonatal Fc receptor to reduce pathogenic IgG autoantibodies while p…

OmicsBank has secured $2.25 million in funding to provide biopharmaceutical companies with patient data from outside the US. The company aims to capitalize on the growing need for diverse health data to train AI models i…

Haisco Pharmaceutical has formed a new alliance with ARCH Venture Partners and Population Health, adding to its series of summer partnerships. The collaboration is part of a broader trend of Western biotech investors eng…

Johnson & Johnson's monoclonal antibody Imaavy has received an expanded FDA approval, making it the first treatment specifically for warm autoimmune hemolytic anemia. The condition causes severe anemia and fatigue due to…

The FDA is working with stakeholders including Amgen and Memorial Sloan Kettering to design a pilot that could shorten the timeline for early-stage drug studies. The initiative aims to make the US more competitive with c…

Bristol Myers Squibb has terminated its partnership with Cellares after evaluating the manufacturer's Cell Shuttle platform. The decision will lead to a corporate reorganization at Cellares. The move reflects a shift in …