Boehringer Ingelheim GmbH’s zongertinib enters the market as the first oral HER2-targeted therapy for patients with non-small-cell lung cancer, following an accelerated approval by the U.S. FDA. Branded Hernexeos, the drug is cleared for use in adults with unresectable disease or whose tumors have HER2 tyrosine kinase domain activating mutations and who have received prior systemic therapy.
Jazz Pharmaceuticals plc is prepped and ready for launch following U.S. FDA accelerated approval of dordaviprone as the first systemic therapy for H3 K27M-mutant diffuse glioma. Branded Modeyso, the orally administered first-in-class imipridone was acquired via Jazz’s acquisition earlier this year of Chimerix Inc. in a deal valued at about $935 million.
Spine Biopharma Inc. cited an “inconsistent sham control response” among some of the sites in its phase III study testing SB-01 as an intradiscal treatment for patients with chronic low back pain associated with degenerative disc disease. The study, dubbed MODEL, fell short of statistical significance on pain intensity and pain-related function, despite showing numerical and clinically meaningful improvements.
PTC Therapeutics Inc. will be launching its oral phenylketonuria therapy, Sephience (sepiapterin) in both the U.S. and Europe this summer, following the U.S. FDA approval just ahead of its July 29 PDUFA date. Sephience previously gained marketing authorization by the European Commission, roughly three months after a thumbs up from the EMA’s Committee for Medicinal Products for Human Use.
A total of 33 states, plus the District of Columbia and Puerto Rico, agreed to participate in the U.S. CMS’ voluntary, outcomes-based program aimed at helping state Medicaid programs cover high-priced cell and gene therapies, starting with therapies for sickle cell disease.
Astrazeneca plc reported results from its BaxHTN phase III trial showing aldosterone synthase inhibitor (ASI) baxdrostat met primary and secondary endpoints in patients with uncontrolled or treatment-resistant hypertension. The news marks a positive outcome of the company’s $1.8 billion gamble on Cincor Pharma Inc. in 2023, and keeps baxdrostat in the race with Mineralys Therapeutics Inc., which has said it anticipates a pre-NDA meeting this year for its ASI, lorundrostat, after reporting pivotal hypertension data in March 2025.
It may be a year later than originally planned, but Regeneron Pharmaceuticals Inc. is set to commercialize Lynozyfic (linvoseltamab) in the U.S. following FDA approval for use in adults with relapsed and refractory multiple myeloma. A bispecific antibody targeting B-cell maturation antigen (BCMA) and CD3, Lynozyfic was cleared under the accelerated approval pathway, with continued approval contingent upon a confirmatory trial.
Celldex Inc.’s KIT inhibitor, barzolvolimab, which hit its phase II endpoints in late 2023, kicking off phase III development in chronic spontaneous urticaria, offered up some impressive long-term findings at the EAACI Congress 2025, including complete responses in patients for as long as seven months after the cessation of therapy, which analysts said could position the drug as a potentially best-in-class option.
Philochem AG’s ligand-targeting approach drew to the table Bristol Myers Squibb Co. in a potential $1.35 billion agreement granting BMS subsidiary Rayzebio Inc. exclusive worldwide rights to OncoACP3, a diagnostic and therapeutic candidate targeting prostate cancer.
One of the big questions going into the phase I readout for Metsera Inc.’s amylin analogue, MET-233i, was whether findings would support once-monthly dosing for the potential obesity candidate. They did. Results also indicated solid and dose-dependent weight loss activity, and Metsera was able to identify well-tolerated starting doses for subsequent studies, said Steve Marso, chief medical officer. “So we exceeded expectations on all three scientific objectives.”
