After a roller coaster of a year for Duchenne muscular dystrophy (DMD) gene therapy Elevidys (delandistrogene moxeparvovec), Sarepta Therapeutics Inc. looks to focus on the efficacy narrative in 2026, starting with newly unveiled three-year data showing durable efficacy across all key motor function assessments for treated DMD patients vs. external controls.

Citing the “totality of data,” Sanofi SA plans to file global regulatory submissions this year for amlitelimab in atopic dermatitis, despite mixed results from two additional phase III trials testing the OX40-ligand (OX40L) inhibitor the pharma firm has been positioning as a potential successor to its Regeneron Pharmaceuticals Inc.-partnered blockbuster, Dupixent (dupilumab).

Researchers have developed an AI-based wearable device, called Revoice, to help individuals communicate effectively after a stroke. The system, worn around the neck, uses sensors to capture carotid pulse signals and subtle vibrations from throat muscles to reconstruct intended words and sentences in real time, potentially transforming the lives of stroke patients living with dysarthria.

Lexicon Pharmaceuticals Inc. announced that it received no objections from the U.S. FDA to move pilavapadin, its non-opioid candidate for diabetic peripheral neuropathic pain, into phase III study, pushing company shares up near 20% on the day.

Six months shy of the PDUFA date for its oral, selective glucocorticoid receptor antagonist relacorilant to treat platinum-resistant ovarian cancer, Corcept Therapeutics Inc. reported a 35% reduction in the risk of death, meeting the second primary endpoint of overall survival after reaching statistical significance with progression-free survival last March.

After selling off amyotrophic lateral sclerosis therapeutics last month, Tanabe Pharma America Inc. emerged a forerunner in another rare disease space with positive top-line phase III data of dersimelagon (MT-7117) for erythropoietic protoporphyria/X-linked protoporphyria.

A trio of European cancer vaccine specialists have filed progress reports, with advances in technology and targeting, fresh infusions of cash, and off-the-shelf products moving into the clinic. Six years on from its formation, Ervimmune closed a series A at €17 million (US$19.9 million) to drive forward clinical development of lead program Ervac-01. Accession Therapeutics Ltd. raised a further £30.5 million (US$40.4 million) from its existing investors, following dosing of four patients with Trocept-01. And Infinitopes Ltd. added $15.4 million to its seed round, as it finalizes preparations for a phase I/IIa trial of the lead product ITOP-1.

Immunofoco Biotechnology Co. Ltd. reported encouraging early clinical data for its CLDN18.2-targeted CAR T therapy, IMC-002, in heavily pretreated gastric and gastroesophageal junction cancer, adding momentum to the push to extend cellular immunotherapy into solid tumors.

Neuropsychiatry specialist, Exciva GmbH raised €51 million (US$59.9 million) in a series B round to fund a phase II/III trial of Deraphan, a combination therapy for treating agitation in patients with Alzheimer’s disease.