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. Ultragenyx said the problems are fixable and not directly related to the quality of the AAV gene therapy UX-111. By midday, the company’s stock (NASDAQ:RARE) had climbed out of a deep premarket hole to tread water at $29.83 per share, an upward bump of a little more than 1%.

Astrazeneca’s baxdrostat solid in phase III hypertension trial

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.

Takeda scores pair of wins with OX2R narcolepsy drug

Takeda Pharmaceutical Co. Ltd. said all primary and secondary endpoints were met in a pair of phase III randomized, double-blind, placebo-controlled studies testing oveporexton (TAK-861), a potential first-in-class oral orexin receptor 2 (OX2R)-selective agonist, in narcolepsy type 1. The condition is caused by the loss of orexin-producing neurons in the brain. For the first time, Tokyo-based Takeda noted, the OX2R agonist mechanism of action has been validated in late-stage trials.

Mink surges on salvage therapy success with Agent-797

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. It is the second landmark case following another one revealed earlier this year of a metastatic gastric cancer patient who achieved a 42% tumor reduction and more than nine months of progression-free survival after a single infusion of the combination therapy. New York-based Mink’s shares (NASDAQ:INKT) skyrocketed from a $7.73 close on July 10 to a $64.17 close on July 11. They have slipped back by 41% in early trading July 14 to about $37.83.

China approves Ascentage’s lisaftoclax, its first Bcl-2 inhibitor

China’s National Medical Products Administration (NMPA) approved Ascentage Pharma Group Corp. Ltd.’s Bcl-2 selective inhibitor lisaftoclax (APG-2575) for treating adults with chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL) who previously received at least one systemic therapy including Bruton’s tyrosine kinase inhibitors. The approval makes lisaftoclax the first Bcl-2 inhibitor receiving conditional approval and marketing authorization for patients with CLL/SLL in China, and the second Bcl-2 inhibitor approved globally. Lisaftoclax is an orally administered small-molecule Bcl-2 selective inhibitor that blocks the antiapoptotic protein Bcl-2 and restores the normal apoptosis process in cancer cells.

PBM practices a threat to future of biosimilars, FTC, DOJ told

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. The original version of House Resolution 1 (H.R. 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. Meanwhile, PBM practices continue to threaten the future of biosimilar competition, industry groups warned at a recent FTC-Department of Justice (DOJ) listening session on biopharma competition.

Glia 2025: Microglial crosstalk could be early Alzheimer’s targeting opportunity

“Loss of synapses and dysfunctional synapses in a region-specific way is important in Alzheimer’s. It’s actually the strongest correlate of cognitive decline, far more so than plaques and tangles, which are the pathological hallmarks,” Soyon Hong told the audience at the XVII Meeting on Glial Cells in Health and Disease, which was held in Marseille last week. The link between synapse loss and cognitive decline in Alzheimer’s disease was first described decades ago, and soon followed by a mechanistic explanation of how those synapses are eaten: by microglial cells that recognize upregulated complement. However, Hong pointed out, that understanding leads directly to the next question: “How do microglia know which synapses are engulfed? … Is there a master orchestrator which coordinates which synapses to be eliminated?”

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