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.

Rhythm Biosciences Ltd. launched a new genetype colorectal cancer risk assessment clinical test that incorporates clinical and lifestyle risk factors alongside its established 140-SNP polygenic risk score to identify people at elevated risk of colorectal cancer. The test supports more targeted screening and complements Melbourne-based Rhythm’s Colostat blood-based CRC detection test, which debuted in Australia in December 2025.

Early data of Waldenström’s non-Hodgkin lymphoma patients enrolled in a trial testing Immunitybio Inc.’s off-the-shelf allogeneic CD19 chimeric antigen receptor natural killer cell therapy (CAR-NK), used in combination with anti-CD20 drug rituximab, led to durable complete responses without lymphodepletion.

The EMA had its second busiest year ever in 2025, approving 104 drugs, of which 38 had a new active substance. Sixteen of these are for treating rare diseases, and four are advanced therapy medicinal products.