A third child with X-linked myotubular myopathy (XLMTM) has died after receiving the higher of two doses of an experimental gene therapy for the rare disease under development at Audentes Therapeutics Inc. Preliminary findings indicate that the immediate cause of death was gastrointestinal bleeding, Audentes said. The trial, testing AT-132 (resamirigene bilparvovec), was already on an FDA clinical hold.
San Diego-based Anaptysbio Inc.’s phase II blowup with anti-IL-33 monoclonal antibody etokimab in chronic rhinosinusitis with nasal polyps (CRwNP), disclosed Aug. 10, followed the compound’s mid-stage atopic dermatitis (AD) fizzle in November of last year, and raised questions not only about the lead candidate but also about the target.
Karuna Therapeutics Inc. remains on track to launch a phase III study by the end of this year with Karxt against acute psychosis in schizophrenia by way of the broad-based program called Emergent, and the company continues planning for a phase II study testing the combo drug as adjunctive therapy with standard of care in the same indication.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Annovis, Ascletis, Aquestive, CNS, Entera, Immunic, Matinas, Orgenesis, Puma, Regeneron, Revive, Viiv.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: 4D Molecular, Acelrx, Albireo, Biolinerx, Epitopoietic Research, Jaguar Health, Kamada, Merck, Northwest, PCI, Pluristem, Qu, Trefoil, Turning Point, Verona.
HONG KONG – China’s Lianyungang-based Jiangsu Hengrui Medicine Co. Ltd. has obtained approval from Korea’s Ministry of Food and Drug Safety (MFDS) to begin a phase III trial for a combination therapy of its PD-1 inhibitor candidate camrelizumab and VEGFR-2 inhibitor rivoceranib.
Following a patient’s death in Poseida Therapeutics Inc.’s phase I trial of P-PSMA-101 in metastatic castrate-resistant prostate cancer (mCRPC), the FDA has put the study on clinical hold.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Acepodia, Aligos, Arrowhead, Clovis, Deciphera, Follicum, GSK, Immatics, Immunicum, Immutep, Intravacc, Maxcyte, Novartis, Organicell, Poseida, Telix, The University of Texas MD Anderson Cancer Center, Ultimovacs, Vanda.
Shares of Unity Biotechnology Inc. (NASDAQ:UBX) fell 66.6% to $4.15 on Aug. 17 after UBX-0101, its one-time lead candidate, failed to best a placebo on an established measure of osteoarthritis pain in a phase II study.
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