In the wake of Keros Therapeutics Inc.’s voluntary halt of dosing in two arms of its phase II study of pulmonary arterial hypertension, the company’s stock (NASDAQ:KROS) has crumpled after a year’s growth. Keros said “unanticipated” adverse events involving pericardial effusion, an excess accumulation of fluid in the sac around the heart, was the cause of the stoppage. Keros halted the 3-mg and 4.5-mg dosing arms of the activin inhibitor cibotercept but is continuing the 1.5-mg arm. The company’s stock had plunged 75% at midday, with shares trading at $17.50 each. The setback comes a week after Takeda Pharmaceutical Co. Ltd. licensed another activin inhibitor, elritercept, in a global development and commercialization deal worth up to $1.3 billion.
Trevi stock jumps; no tweaks needed in IPF cough phase II
Shares of Trevi Therapeutics Inc. (NASDAQ:TRVI) were trading at $4.02, up $1.52, or 61%, on word from the phase IIb Coral trial with investigational therapy Haduvio (oral nalbuphine extended release) for the treatment of chronic cough in idiopathic pulmonary fibrosis and refractory chronic cough. New Haven, Conn.-based Trevi disclosed a positive outcome from the planned sample size re-estimation for the ongoing experiment. No change to the current sample size for the 160-patient study will be necessary. The trial has reached 75% of targeted enrollment, and top-line results from the full effort are still expected in the first half of 2025, Trevi said.
Marinus, Sage fall on clinical data as neurology stocks decline
By the end of November 2024, the BioWorld Neurological Diseases Index had dropped 13.4%, deepening its decline of 9.4% at the end of August. In recent months, shares of Wave Life Sciences Ltd. climbed following results from its ongoing phase Ib/IIa trial of an A-to-I RNA editing oligonucleotide. Meanwhile, Marinus Pharmaceuticals Inc. plunged after an oral ganaxolone phase III miss and Sage Therapeutics Inc. discontinued development of dalzanemdor for Huntington’s disease.
Caliway’s CBL-514 meets endpoints in reducing abdominal fat
Caliway Biopharmaceuticals’ lipolysis candidate, CBL-514, met all the primary and secondary efficacy endpoints in a phase IIb study for subcutaneous fat reduction, paving the way for the company to begin a global pivotal phase III study in 2025. In October, Taiwan-based Caliway raised NT$6.4 billion (US$206 million) in its IPO on the Taipei Stock Exchange, making it the largest IPO in Taiwan’s biotech industry history and valuing the company at nearly $3 billion following the listing, BioWorld previously reported. CBL-514 is a small-molecule lipolysis drug known to upregulate the capsase-3 and ratio of Bax-to-Bcl2 expression, helping to trigger adipocyte apoptosis. The non-invasive injection works to reduce subcutaneous fat without causing tissue necrosis or other side effects.
SK Bioscience to start mRNA vax trial for Japanese encephalitis
SK Bioscience Co. Ltd. gained approval from the Human Research Ethics Committee in Australia to start phase I/II trials of GBP-560, its mRNA-based vaccine candidate for mosquito-borne Japanese encephalitis virus, with funding from Coalition for Epidemic Preparedness Innovations (CEPI). SK Bioscience expects to kick off the phase I/II study in Australia in February 2025, with interim results expected by 2026. The mRNA platform is being developed for both Japanese encephalitis virus and Lassa fever virus, which is transmitted through exposure to urine or feces of infected Mastomys rats. CEPI committed up to $40 million (₩57.4 billion) in initial funding to support Seoul, South Korea-based SK Bioscience’s preclinical and early clinical trials.