Among severe insulin-deficient diabetes patients, 12 weeks of 100-mg, once-daily dosing of Biomea Fusion Inc.’s icovamenib lowered hemoglobin A1c by 1.8% from placebo at the 52-week timepoint, an increased benefit over and above what was seen at 26 weeks.
Washington University in St. Louis has disclosed new autophagy inducers reported to be useful for the treatment of alpha-1 antitrypsin deficiency, amyotrophic lateral sclerosis, Alzheimer’s, Huntington’s and Parkinson’s disease.
A top-line readout of the 26-week phase IIa Cbeyond trial showed nimacimab, Skye Bioscience Inc.’s peripherally restricted CB1 inhibitor antibody for weight loss, fell short of statistical significance as a monotherapy vs. placebo on the primary endpoint of weight loss, sending the company’s shares down 60%. Skye executives, however, offered a more optimistic outlook for the findings, which they said provide clear direction for moving forward.
A major contributor to obesity-related pathology is inflammation involving a shift in macrophage polarization toward the inflammatory M1 phenotype and accumulation of such macrophages in the liver and adipose tissue. Activation of Toll-like receptor 4 (TLR4) on macrophages leads the downstream adaptor protein MyD88 to promote M1 polarization through altered gene expression mediated by the transcription factor NF-κB.
Although type 2 diabetes tends to get more airtime, type 1 diabetes also had drawn a number of the developers to the table. Recently winning the attention of Wall Street is SAB Biotherapeutics Inc., which offered data during the European Association for the Study of Diabetes annual meeting. Vertex Pharmaceuticals Inc. and Biomea Fusion Inc. are among the other players.
A U.S. FDA complete response letter (CRL) citing manufacturing concerns has stepped into the way of a rare genetic disorder treatment. Fortress Biotech Inc. and its subsidiary, Cyprium Therapeutics Inc., said the letter in response to the NDA for CUTX-101, an injectable formulation of copper histidinate, noted cGMP deficiencies at the facility where the Menkes disease treatment is manufactured.
Hanmi Pharmaceutical Co. Ltd. and Health Hope Pharma Ltd. may be getting a second wind for encequidar and the Orascovery platform, with Gilead Sciences Inc. picking up exclusive global rights to develop and commercialize encequidar in the field of virology for $2.5 million up front.
Phase IIb data of Metsera Inc.’s lead GLP-1 receptor agonist, MET-097i, showed significant weight loss and good tolerability, supporting a phase III start later this year, and validating Pfizer Inc.’s decision a week ago to buy the obesity-focused company for $7.3 billion.
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