Manufacturing issues are the latest problem for Ultragenyx Pharmaceutical Inc. to solve after last week’s disappointment in a phase III study to treat brittle bones. The U.S. FDA gave the company a complete response letter (CRL) regarding the BLA for its gene therapy to treat Sanfilippo syndrome type A, saying it needs more details and improvements made about CMC after having finished manufacturing facility inspections. Read More
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. Read More
Takeda Pharmaceutical Co. Ltd.’s dual phase III victories in narcolepsy type 1 (NT1) with oral orexin receptor 2 (OX2R)-selective agonist oveporexton (also known as TAK-861) had Wall Street mulling what the outcome might mean for others trying the mechanism of action, and questions linger about side effects on eyesight. Read More
The complete remission of a testicular cancer patient receiving Mink Therapeutics Inc.’s allogeneic, off-the-shelf invariant natural killer T-cell therapy Agent-797 with nivolumab drove the company’s shares up by 730% July 11. The results, published in Nature’s Oncogene, described the complete, durable remission of a 49-year-old man with a germ cell neoplasm, showing no evidence of disease two years after receiving a single infusion. Read More
China’s National Medical Products Administration approved Ascentage Pharma Group Corp. Ltd.’s Bcl-2 selective inhibitor lisaftoclax (APG-2575) for treating adults with chronic lymphocytic leukemia/small lymphocytic lymphoma who previously received at least one systemic therapy including Bruton’s tyrosine kinase inhibitors. Read More
Once again, U.S. legislative reforms to rein in pharmacy benefit manager (PBM) business practices missed a ride to finally becoming law. This time, they were kicked out of the Trump administration’s budget reconciliation bill that was signed into law July 4. House Resolution 1, as first passed in the lower chamber, included a few PBM reforms, but they were deleted from the Senate version that ultimately became law because the parliamentarian ruled they didn’t meet the restrictions placed on reconciliation measures. Read More
Biopharma happenings, including deals and partnerships, grants, preclinical data and other news in brief: Cel-Sci, Century, Perpetual, TandemAI. Read More
Regulatory snapshots, including global drug submissions and approvals, clinical trial approvals and other regulatory decisions and designations: Capricor, Cognition, Milestone. Read More
