A fourth child has died after developing liver complications on a trial of Astellas Pharma Inc.’s gene therapy for rare neuromuscular disease, after FDA advisers noted the problems on the ASPIRO study in a discussion on gene therapy safety. Last week, Astellas announced that it had stopped dosing on ASPIRO after a safety issue involving liver function emerged in the trial of the gene therapy AT-132, aimed at the life-threatening rare disease X-linked myotubular myopathy.
The NIH and CDC granted researchers at the Feinstein Institutes for Medical Research at Northwell Health $700,000 to investigate an in-the-ear stimulator as a treatment for post-traumatic stress disorder (PTSD) in first responders to the attack on the World Trade Center 20 years ago. The researchers will enroll 30 World Trade Center first responders with chronic PTSD in a sham-controlled trial to test the effectiveness of a device that uses transcutaneous auricular stimulation of the vagus nerve, which has shown promise in preclinical studies.
The share price of Renovorx Inc. has spiked following FDA clearance for its Renovocath delivery system. Since the company announced the clearance, its stock (NASDAQ:RNXT) has climbed by more than 13%. Last month the company completed an IPO for approximately $16.6 million, issuing shares at $9-$11.
Shares of antibody drug conjugate developer Mersana Therapeutics Inc. (NASDAQ:MRSN) fell 39.1% to $8.56 on Sept. 10 after the company disclosed a second death from pneumonitis in the ongoing phase I trial testing its antibody drug conjugate, upifitamab rilsodotin against tumor types likely to express the sodium-dependent phosphate transport protein NaPi2b, such as ovarian cancer.
Apellis Pharmaceuticals Inc.’s win with one phase III trial and narrow miss with an identical one testing pegcetacoplan in geographic atrophy secondary to age-related macular degeneration caused Wall Street to punish the company while rewarding competitor Iveric Bio Inc.
DUBLIN – In biopharma, U.S. patent grants hardly represent big news. Without them, you simply don’t get to sit at the table. So Onk Therapeutics Ltd.’s receipt of U.S. patent no. 11104735 covering CISH gene knockouts in natural killer (NK) cell therapies for cancer is not a major event in the general scheme of things. At the same time, it is a vitally important enabler for a company that is, paradoxically, both an early mover in the field but also a laggard in the highly competitive race to move NK cells into clinical development.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: Alvotech, Apellis, Astrazeneca, Azurx, Cartargia, Cassiopea, Cytodyn, Mersana, Rezolute, Scpharmaceuticals, Senhwa, Sunovion.
Savicell Diagnostics Ltd. has reported high accuracy results from a study validating its liquid biopsy platform Immunobiopsy. Haifa, Israel-based Savicell said the platform achieved high accuracy identifying lung cancer across stages I to IV. In a cohort of 245 participants, the Immunobiopsy test achieved 97% sensitivity and 97% specificity among patients with lung cancer.
Astrazeneca plc is eyeing an addition to its respiratory diseases portfolio after a potential first-in-class “rescue” asthma inhaler, PT-027, met its goals in two phase III trials.
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