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BioWorld - Wednesday, December 31, 2025
Home » Topics » Clinical

Clinical
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Caribou adds hoofprint, multiple developers ‘herd’ from in ROR1

Jan. 27, 2023
By Randy Osborne
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.
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After phase II miss, Ocuphire to seek a visual from FDA on next steps in DR

Jan. 26, 2023
By Randy Osborne
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.
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Staphylococcus aureus

Analysts positive, market not so much, on Aridis phase III MAb data for S. aureus VAP

Jan. 26, 2023
By Karen Carey
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.
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Neon brain

Small Pharma takes big step forward with positive phase IIa psychedelic drug data

Jan. 25, 2023
By Caroline Richards
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.
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Layoff illustration

Finch drops phase III of CP-101 for C. difficile, reduces staff by 95%

Jan. 24, 2023
By Karen Carey
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 Clostridium difficile infection – on the sidelines.
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Red coronavirus with long shadow

Playing the long game: Axcella moves toward a global long COVID study

Jan. 24, 2023
By Lee Landenberger
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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Dialysis patient Vasc-Alert

Vasc-Alert algorithm reliably predicts vascular access blockages in dialysis patients

Jan. 23, 2023
By Annette Boyle
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.
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Pliant posts positive phase IIa data in IPF

Jan. 23, 2023
By Lee Landenberger
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).
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IV drips

FDA lifts clinical hold on Astellas Fortis AAV gene therapy trial in Pompe disease

Jan. 20, 2023
By Tamra Sami
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.
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GBS navigation system directs Annexon toward pivotal study; effort in HD on track, too

Jan. 19, 2023
By Randy Osborne
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.
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