DUBLIN – Shares in Idorsia Ltd. were off almost 4% Oct. 11 on news that lucerastat missed the primary endpoint of the Modify phase III trial in Fabry disease. The double-blinded, placebo-controlled study, which recruited 118 adult patients, was designed to evaluate the effect of lucerastat on neuropathic pain. Patients were randomized to drug or placebo in a 2-to-1 ratio.
Clinical updates, including trial initiations, enrollment status and data readouts and publications: 4D, Aligos, Astrazeneca, Bicycle, Diurnal, Idorsia, Galmed, Oxurion, Regenxbio, Reistone, Repertoire, Sarepta.
On the last day of this year’s Molecular Targets meeting, an annual joint conference of the American Association for Cancer Research, the National Cancer Institute and the European Organization for the Research and Treatment of Cancer, the final plenary went from molecular to macro in a lively discussion of the biggest roadblock in cancer drug development, and what can be done to improve it.
Shares of Protagonist Therapeutics Inc. (NASDAQ:PTGX) climbed 93.9% on Oct. 11 after the FDA removed a full clinical hold on studies of the rusfertide, the company's investigational treatment for the blood disorders polycythemia vera and hereditary hemochromatosis. Triggered by a finding of malignant skin tumors in mice treated with the drug disclosed on Sept. 17, the FDA's three-weeks-ago hold had pushed Protagonist shares down by as much as 72%.
Limbix Health Inc. released data demonstrating a 21% remission rate and 29% response rate for Sparkrx, its prescription digital therapeutic, in adolescents who completed the program as recommended. The company presented study results on Oct. 8 at the 2021 American Academy of Pediatrics National Conference in San Francisco.
Allogene Therapeutics Inc. officials took many questions but had few answers during a conference call regarding the FDA’s clinical hold after the report of a chromosomal abnormality in ALLO-501A CAR T cells in a patient treated in the phase I/II Alpha2 study.
It looks like Shanghai Miracogen Inc.’s antibody-drug conjugate (ADC) partnership with Synaffix BV is paying off as MRG-004A, an ADC designed to treat solid tumors, has entered a U.S. phase I/II trial to treat solid tumors.
A lack of toxicity and the shrinking of tumors were at the heart of new interim data produced from a two-stage phase II study by Relay Therapeutics Inc. The company reported that the oral small molecule RLY-4008, designed to elicit responses across a broad spectrum of resistance mutations and in multiple tumor types, selectively inhibited fibroblast growth factor receptor (FGFR) 2 in ways that are not limited by off-target toxicities of hyperphosphatemia (FGFR1) and diarrhea (FGFR4).
PERTH, Australia – After meeting with the FDA, Medlab Clinical Ltd. is reformulating its cannabinoid pain product into a synthetic formulation because the agency indicated it was more likely to register a synthetic compared to a botanic.