Exonate Ltd. is preparing to advance its eye drop candidate to treat retinal vascular diseases, EXN-407, into a phase IIb study following promising tolerability and early signs of efficacy in a phase Ib/IIa study. A small-molecule SRPK1 inhibitor, EXN-407 is designed to be administered twice daily and works by selectively targeting specific (proangiogenic) isoforms of VEGF that lead to vascular retinal disease progression via aberrant growth of leaky blood vessels within the eye.
Apogee Therapeutics Inc.’s phase I home run put IL-13-targeting antibody APG-777 on an accelerated development path in atopic dermatitis, and the company touted its similarity to further-along IL-13 competitor lebrikizumab, from Eli Lilly and Co., as a likely indicator of further success.
The RNAi therapeutic zilebesiran, which was the focus of a 2023 $2.8 billion deal between Alnylam Pharmaceuticals Inc. and Roche Holding AG, demonstrated significant systolic blood pressure reductions in patients with mild to moderate hypertension who were part of the Kardia-2 phase II study.
While preparing a follow-on phase III study of its Cardiamp cell therapy, Biocardia Inc. has mined positive interim data at a mean 20-month follow-up of all patients in the original Cardiamp HF Trial.
Vivoryon Therapeutics NV said it will conduct an in-depth analysis after reporting top-line data showing the phase IIb European Viviad study testing its oral small molecule, varoglutamstat, in patients with early Alzheimer’s disease missed statistical significance on the primary endpoint measuring changes in cognition over time. Investors, however, opted for a more immediate reaction, sending company shares (Euronext Amsterdam:VVY) plunging 90% March 4, to close at €0.80 (US87 cents)
Akero Therapeutics Inc.’s phase IIb data with FGF21 analogue efruxifermin (EFX) in patients with pre-cirrhotic metabolic dysfunction-associated steatohepatitis (MASH) kept interest alive in the face-off between the company and 89bio Inc. with pegozafermin, another FGF21 drug targeting MASH, for which a phase III study is planned.
Amber Therapeutics Ltd. revealed positive data from the first-in-human investigation of its Amber-UI device, an implantable closed-loop bioelectrical therapy for urinary incontinence. Initial results highlight the safety and scalability of the Amber-UI therapy, which has the potential to meaningfully improve quality of life for patients dealing with the condition.
As Cardiff Oncology Inc. dosed the first patient in its phase II study of onvansertib (CRDF-004) in a first-line setting to treat RAS-mutated metastatic colorectal cancer (mCRC), the company also revealed positive efficacy data from its discontinued phase II of onvansertib as a second-line treatment of bevacizumab-naïve RAS-mutated mCRC.
Biovie Inc.’s double-barrelled blast of positive data with insulin sensitizer NE-3107 in Alzheimer’s disease (AD) and Parkinson’s disease (PD) launched shares (NASDAQ:BIVI) on a wild ride to close March 1 at $2.23, up 99 cents, or almost 80%, after trading as high as $3.31.
Positive phase III data from Ironwood Pharmaceuticals Inc. with once-weekly glucagon-like peptide-2 analogue apraglutide failed to impress Wall Street, and shares of the Boston-based firm (NASDAQ:IRWD) closed Feb. 29 at $9.43, down $5.69, or 37.6%. The experiment called Stars tested apraglutide to reduce parenteral support in adults with short bowel syndrome with intestinal failure. Rare and severe, the condition affects about 18,000 adults in the U.S., Europe and Japan. Based on the latest data, Ironwood plans to submit an NDA to the U.S. FDA and other regulatory filings.
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