The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating AAV gene replacement therapy AT-845 in adults with late-onset Pompe disease.
Vera Therapeutics Inc.’s latest results from the phase IIb Origin trial with atacicept in patients with IgA nephropathy (IgAN) provided cause for optimism with regard to the phase III experiment targeted for the first half of this year – which should yield 36-week data in the first half of 2025 – and the company is budgeting to make the later-stage effort happen.
Equilibre Biopharmaceuticals Corp. has positive top-line data from the phase II study of its lead asset, EQU-001, an anti-inflammatory adjunctive therapy for treating focal seizures for adults with epilepsy.
A meta-analysis found no difference in outcomes between autograft nerve repair and repair using Axogen Inc.’s Avance nerve grafts across all types of nerves and gap lengths up to 70 millimeters. Axogen’s allograft nerves allow patients to avoid the potential complications associated with harvesting the individual’s own nerve to use in repair. The study was published in Plastic and Reconstructive Surgery in January.
Caribou Biosciences Inc.’s disclosure last December that it has chosen the target for CB-020, an induced pluripotent stem cell-derived allogeneic CAR-NK cell therapy for solid tumors, added impetus to the growing interest in receptor tyrosine kinase-like orphan receptor 1 (ROR1), where a number of parties are advancing programs.
Ocuphire Pharma Inc. is marching ahead despite falling short of the primary endpoint in top-line data from the Zeta-1 phase II study testing oral APX-3330 in diabetic retinopathy (DR). The trial did not achieve its goal with regard to the percentage of patients with a ≥2-step improvement in Diabetic Retinopathy Severity Score at week 24 in the study eye. Ocuphire plans to schedule an end-of-phase II meeting with the U.S. FDA to review the results.
Low enrollment in a phase III trial and a missed primary endpoint sent Aridis Pharmaceuticals Inc.’s shares tumbling by 35% on Jan. 26, although the company’s AR-301 (tosatoxumab) showed superior efficacy over the control group in Staphylococcus aureus ventilator-associated pneumonia (VAP) patients.
The burgeoning psychedelic therapeutics market experienced a boost Jan. 25 as Small Pharma Inc. announced its synthetic, intravenous formulation of N,N-dimethyltryptamine (DMT), SPL-026, led to a statistically significantly, rapid reduction in depression symptoms in patients with major depressive disorder in a phase IIa trial, thus meeting its primary endpoint. DMT is a powerful hallucinogenic tryptamine substance which works as a 5-HT receptor agonist. It is found naturally in various plants, and is a schedule I controlled substance in the U.S. Although lesser known than other psychedelics such as magic mushrooms and LSD, it produces short-lived but intense auditory and visual hallucinogenic effects.
Microbiome company Finch Therapeutics Group Inc. has suffered one blow after another in the past year with dwindling cash, delayed programs, a terminated deal and three workforce reductions. The latest reduction will bring the once healthy 189-person company down to a handful of employees, and it places the lead program – the oral microbiota product, CP-101, for Clostridiumdifficile infection – on the sidelines.
Axcella Therapeutics Inc. is working on its comeback to make something else go away. Just before Christmas, Axcella said it was repositioning itself to focus on long COVID-19 and was just granted regulatory guidance on a primary endpoint and study design from the U.K.’s MHRA to conduct a single registration trial of AXA-1125 for those with long COVID fatigue. Axcella will be meeting with the MHRA in the near term to discuss getting an innovative licensing and access pathway application, which is designed to accelerate the approval process. Axcella also has just submitted its IND to the U.S. FDA for a global phase IIb/III study for treating long COVID.
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