Stoke Therapeutics completes enrollment in Phase III EMPEROR study for zorevunersen

BridgeBio Pharma reported positive results from a late-stage clinical trial of its experimental drug encaleret, showing improvements in symptoms and bone health among patients with a rare genetic disorder that affects the body's ability to regulate calcium. The company said Sunday that all patients treated with the drug in its Phase 3 CALIBRATE trial reported improvement in at least one symptom of autosomal dominant hypocalcemia type 1, or ADH1, compared with 46% of patients receiving standard treatment. After 24 weeks of treatment, 91% of patients receiving the experimental therapy had parathyroid hormone levels at or above the lower limit of normal, compared with none of those receiving standard care. About 61% of patients receiving the drug reported reduced fatigue, compared with 27% of those receiving standard care, while muscle cramps improved in 58% of treated patients, compared with 36% in the comparison group, and nearly half of patients taking the experimental treatment reported less brain fog, compared with 9% of patients receiving conventional care. The FDA has accepted BridgeBio's new drug application and granted it priority review, setting a target decision date of May 8, 2027, and the company has also submitted an application seeking approval from European regulators. BridgeBio is also pursuing a potentially larger market through a separate Phase 3 trial called RECLAIM-HP involving patients with chronic hypoparathyroidism, which plans to enroll approximately 160 adults and adolescents and will compare the drug with a placebo over 24 weeks.

Becton Dickinson agreed to invest $3 billion to bring manufacturing of essential medical products to the United States, part of a broader plan to invest $19 billion in the country over several years across capital, operations and supply-chain initiatives, President Donald Trump said Monday in a post on Truth Social. More than $1 billion of that $3 billion will go to Nebraska to ramp up production of numerous products, including needles made with American steel, and BD expects to increase domestic production of essential medical consumables by about 5 billion units annually, raising the share of such products supplied from within the U.S. to roughly 80%. Separately, Viatris entered a definitive agreement to acquire all outstanding shares of Pacira BioSciences for $36.50 per share in cash, an aggregate equity value of $1.65 billion, in a deal expected to close by the end of 2026 that adds the patent-protected pain therapies EXPAREL and ZILRETTA; Pacira generated approximately $746 million in total revenue and about $177 million in adjusted EBITDA in the twelve months ended June 30, 2026. CSL and Alentis Therapeutics entered an exclusive global partnership to co-develop and co-promote lixudebart for rare kidney, liver and other diseases, with CSL making an initial payment of $355 million and Alentis eligible for up to $1.2 billion in commercial milestone payments, and the companies sharing global profits 55% to CSL and 45% to Alentis once the drug is commercialized. Novavax and Moderna traded flat in the premarket on Tuesday after the WHO said Russian officials had traced all contacts of a plague research lab employee who died of pneumonia of an unknown cause, though Novavax closed 20% higher and Moderna added 7% on Monday after Russia's Rospotrebnadzor reported the death of the 28-year-old worker at the Irkutsk Research Anti-Plague Institute of Siberia.

Vertex Pharmaceuticals announced new interim data showing that ALYFTREK, or vanzacaftor/tezacaftor/deutivacaftor, restored exocrine pancreatic function in some children with cystic fibrosis ages 2 to 5, allowing them to stop pancreatic enzyme replacement therapy. The data, presented as a late breaker poster at the North American Cystic Fibrosis Conference, came from 48 children enrolled in the PERT discontinuation substudy VX22-121-106 Cohort 2, part of an ongoing open-label extension study in that age group. After at least 48 weeks of treatment, mean fecal elastase-1 was 239.7 micrograms per gram, a mean increase of 110.2 micrograms per gram from baseline, and 48.6 percent of children reached the pancreatic sufficiency threshold of at least 200 micrograms per gram. Of the 48 children eligible for the substudy, 18, or 37.5 percent, successfully discontinued pancreatic enzyme replacement therapy for a mean duration of 8.6 weeks, and across the full enrolled cohort of 66 children, 50 percent were not on the therapy at the data cut. Carmen Bozic, Vertex's chief medical officer, said the findings represent a profound shift in understanding the benefits of treating cystic fibrosis in its earliest stages, noting that pancreatic failure had long been considered irreversible. Vertex also presented additional abstracts at the conference, held October 7 to 10 in Atlanta, on clinical and real-world evidence for CFTR modulators, including a post-hoc analysis of reduced intravenous antibiotic use with VNZ/TEZ/D-IVA in patients 12 and older and a natural history study of exocrine pancreatic function in infants under 12 months. The use of ALYFTREK in children 2 to 5 years old is investigational.

Scholar Rock has resubmitted its Marketing Authorisation Application for apitegromab to the European Medicines Agency for the treatment of children and adults with spinal muscular atrophy. The resubmitted application replaces the Catalent Indiana fill-finish facility with Scholar Rock's alternate fill-finish facility, the same U.S.-based site that supported the September 2026 FDA approval of ISEMBYLD, also known as apitegromab-mstn. The company withdrew its previous MAA in August 2026 after a lack of GMP compliance at the Catalent Indiana facility, and now anticipates a Committee for Medicinal Products for Human Use opinion in the first half of 2027. Chief Executive Officer David L. Hallal said the company is working with urgency to bring this first-ever muscle-targeted therapy to children and adults living with SMA in Europe, where more than 35,000 SMA patients globally are receiving an SMN-targeted therapy. Apitegromab is the first and only muscle-targeted treatment to demonstrate motor function improvement in individuals with SMA currently receiving a survival motor neuron 2-targeted treatment, based on the Phase 3 SAPPHIRE study.