Welcome to the Weekly Rundown where the DDN editors cover this week’s top biotech and pharma news.
Genentech's oral breast cancer drug gets priority FDA review — first major endocrine therapy advance in two decades
Genentech announced this week that the FDA has accepted its New Drug Application (NDA) for giredestrant under priority review, an investigational oral selective estrogen receptor degrader for adults with estrogen receptor (ER)-positive, HER2 (Human Epidermal growth factor Receptor 2)-negative early-stage breast cancer, with a decision expected November 30, 2026. The submission is based on results from the Phase 3 lidERA Breast Cancer study, which enrolled more than 4,100 patients and showed giredestrant reduced the risk of invasive disease recurrence or death by 30 percent compared with standard-of-care endocrine therapy. At three years, 92.4 percent of patients on giredestrant were alive and free of invasive disease versus 89.6 percent on standard therapy, with consistent benefit across all subgroups. The drug was also better tolerated, with a treatment discontinuation rate of 5.3 percent versus 8.2 percent on standard care. If approved, giredestrant would represent the first significant advance in adjuvant endocrine therapy for early-stage breast cancer in more than 20 years — a meaningful bar given that ER (Estrogen receptor)-positive disease accounts for roughly 70 percent of all breast cancer diagnoses and up to a third of early-stage patients eventually experience recurrence on current therapies. The FDA separately accepted a second Genentech NDA for giredestrant in combination with everolimus for advanced breast cancer with ESR1 (Estrogen Receptor 1) mutations, with that decision expected in December 2026. – Andrea Corona
Travere and Everest Medicines collaborate on $1B deal for BTK inhibitor
In an exclusive licensing agreement with Everest Medicines, Travere Therapeutics will develop and commercialize civorebrutinib, a reversible Bruton’s tyrosine kinase (BTK) inhibitor to treat immune-mediated rare kidney diseases. Past proof-of-concept Phase 1/2 data has shown “rapid and sustained reductions in anti-PLA2R autoantibodies and proteinuria, with high rates of immunologic and clinical remission and stable kidney function through 52 weeks of follow-up,” according to the press release. The deal gives Travere exclusive rights excluding markets in China and certain countries in East and Southeast Asia, according to the statement. “As a differentiated, potential best-in-class therapy, civorebrutinib has demonstrated encouraging efficacy in primary membranous nephropathy. With its highly selective and reversible covalent mechanism of action, it is well positioned to advance in development across multiple immune-mediated kidney indications,” said Yifang Wu, Chairman of the Board of Everest Medicines. – Allison Whitten
Vertex's kidney disease drug clears FDA review hurdle with a November decision date
Vertex Pharmaceuticals announced this week that the FDA has accepted its Biologics License Application for povetacicept, an investigational dual inhibitor of the B cell activating factor and APRIL (A PRoliferation-Inducing Ligand) cytokines, for adults with immunoglobulin A nephropathy (IgAN), the most common cause of primary glomerulonephritis worldwide. The FDA has set a Prescription Drug User Fee Act target action date of November 30, 2026. The submission is supported by a pre-specified interim analysis of the Phase 3 RAINIER trial, the largest IgAN trial ever conducted, in which povetacicept achieved a 49.8 percent reduction in urine protein to creatinine ratio compared to placebo at week 36, a key marker of kidney disease progression. The trial also hit both secondary endpoints, including a 79.3 percent reduction in galactose-deficient immunoglobulin A1 compared to placebo, a protein central to the disease's underlying biology, and hematuria resolution in 85.1 percent of treated patients versus 23.4 percent on placebo. If approved, povetacicept would mark Vertex's first commercial entry into nephrology, expanding well beyond its established cystic fibrosis franchise. The drug has also received Breakthrough Therapy designation and is being studied in additional autoimmune indications including primary membranous nephropathy and generalized myasthenia gravis. – Andrea Corona
Revolution’s daraxonrasib doubles survival in phase 3 pancreatic trial
The full data on Revolution Medicines’ pancreatic cancer candidate daraxonrasib were presented on Sunday to multiple rounds of applause at the American Society of Clinical Oncology annual meeting in Chicago, alongside publication in The New England Journal of Medicine. In the global Phase 3 RASolute 302 trial, once-daily oral daraxonrasib more than doubled both overall survival (OS) and progression-free survival compared to standard cytotoxic chemotherapy in patients with previously treated metastatic pancreatic ductal adenocarcinoma, and nearly tripled the objective response rate. In the intent-to-treat population, median OS was 13.2 months with daraxonrasib versus 6.7 months for chemotherapy, showing a roughly 60 percent reduction in the risk of death. The drug was generally well tolerated, with fewer high-grade treatment-related adverse events and lower discontinuation rates than chemotherapy, and patients reported delayed deterioration in pain and quality of life. Revolution Medicines said it plans to submit the data to regulators, including the FDA under the Commissioner’s National Priority Voucher program, and has already received an FDA “safe to proceed” letter to begin an expanded access program for daraxonrasib in eligible US patients. – Bree Foster
Servier bets on muscular dystrophy in $2.65B deal
On Monday, Servier announced that it will acquire Edgewise Therapeutics’ muscular dystrophy business for $1.55 billion upfront and up to $1.1 billion in regulatory and commercial milestone payments. Data on Edgewise’s oral skeletal myosin inhibitor, sevasemten, is expected in Q4 this year. Currently in Phase 2 and 3 trials, the drug candidate works by inhibiting fast skeletal myosin and allowing for more gentle muscle contractions. “The acquisition of Edgewise Therapeutics’ muscular dystrophy business is a key step forward to achieve our Servier 2030 ambition in neurology with a team of talented experts and a promising asset in muscular dystrophies,” said Olivier Laureau, President of Servier, in the press release. The deal marks continued growth in neurology for Servier, which currently has three other drugs in development in this space. For Edgewise, the deal removes their neurology program and narrows their focus to their cardiovascular indications. – Allison Whitten
Allen Institute leads $400m collaborative effort to decode brain disorders
The Allen Institute on Tuesday launched the Brain Health accelerator, a $400 million global research initiative aimed at pinpointing the specific brain cells and neural circuits disrupted in neurodegenerative disease and using that insight to speed the development of new treatments. Backed by $200 million from the Allen Institute, $100 million from the Bezos family, and a further $100 million from partners including National Institutes of Health and Amazon Web Services, the program brings together academia, technology groups, philanthropy and disease advocates in an open and collaborative, human-first research model. The effort will focus initially on Alzheimer’s disease, Parkinson’s disease, Lewy body dementia, Huntington’s disease, and amyotrophic lateral sclerosis, using advances in single-cell genomics, high-resolution imaging and AI-driven data analysis to map vulnerable cell types and circuits directly in human brain tissue. – Bree Foster
