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.
Vasc-Alert LLC’s surveillance technology successfully predicts which dialysis patients are at risk of experiencing stenosis, a study published in the Journal of Vascular Access shows. Patients assigned high scores by the company’s algorithm had seven times the risk of stenosis and blockage compared to those with low-risk scores.
A lack of available options for treating idiopathic pulmonary fibrosis (IPF) is driving plenty of development into the indication. The latest data come from Pliant Therapeutics Inc., which posted positive 12-week interim data from 21 participants in the 320-mg dose group’s active arm and eight in the placebo arm in its phase IIa study of PLN-74809 (bexotegrast).
The U.S. FDA has lifted the clinical hold on Astellas Pharma Inc.’s Fortis phase I/II trial evaluating adeno-associated virus (AAV) gene replacement therapy AT-845 in adults with late-onset Pompe disease.
Upcoming catalysts from Annexon Biosciences Inc. put some joy into shares as the firm talked up its prospects during the recent J.P. Morgan Healthcare Conference, where attendees heard Jan. 8 about the news ahead with C1q protein complex inhibitor ANX-005 in Huntington’s disease (HD) as well as progress in Guillain-Barre syndrome (GBS), and more. The stock (NASDAQ:ANNX) enjoyed a 43% stock boost in the days after JPM, rising from $4.79 on Jan 8 to $6.84 on Jan. 13.
A year and a half gone, two HIV vaccine studies shut down. That’s the case with the Janssen Pharmaceutical Cos. of Johnson and Johnson as the phase III Mosaico study of its HIV vaccine regimen was not effective in preventing infection compared to placebo. Based on a data and safety monitoring board’s report saying the study was not expected to hit its primary endpoint, Janssen discontinued the clinical trial more than a year ahead of its estimated March 2024 completion date.