With a shout-out to the ALS community, the FDA opened an advisory committee meeting today on Amylyx Pharmaceutical Inc.’s AMX-0035, a fixed-dose combination of sodium phenylbutyrate and taurursodiol, as a treatment for amyotrophic lateral sclerosis (ALS). The FDA recognizes the urgent unmet need for a therapy to slow the progression of ALS and that patients would welcome even small advances, the agency’s Teresa Buracchio said in her opening remarks to the Peripheral and Central Nervous System Drugs Advisory Committee. Cognizant of the number of ALS patients and caregivers listening to the virtual meeting and the 516 comments received prior to the meeting, Buracchio said the FDA has heard the concerns of the ALS community and has been responsive to them, as it considered the unmet need throughout its review.

Roche’s TIGIT drug fails in phase III trial in aggressive lung cancer

Roche Holding AG’s attempt to find a next-generation cancer immunotherapy has suffered a setback after the first ever phase III readout for an anti-TIGIT drug, tiragolumab, turned out negative. With big pharma investing heavily in TIGIT (T-cell immunoreceptor with immunoglobulin and ITIM domains), results from the company’s Skyscraper-02 trial were closely watched. An interim analysis, however, showed tiragolumab failed to meet its progression-free survival endpoint in the study, which is testing the candidate in people with extensive stage small-cell lung cancer (ES-SCLC).

Rejuvenate gains clearance for ‘ground-breaking’ sarcopenia trial

LONDON – In a first of its kind phase I trial, Rejuvenate Biomed NV has secured ethical clearance to induce acute sarcopenia in 42 healthy subjects, aged 65 to 75 years old. That will be achieved by placing a cast on one leg to induce sarcopenia through muscle disuse. The volunteers in the double-blind trial will then receive either RJx-01, an oral small molecule for treating sarcopenia, or placebo.

Results with intranasal flu vaccine punish Revelation shares

Shares of Revelation Biosciences Inc. (NASDAQ:REVB) took a $44.4% hit, trading midday at $1.19, down 95 cents, as the firm disclosed results from an interim statistical analysis of its phase IIb challenge study of intranasal REVTx-99a for the prevention of H3N2 influenza (influenza A) in healthy adults. REVTx-99a missed its primary endpoint, area under the curve of viral load by quantitative RT-PCR from nasopharyngeal swabs. Preliminary data suggest the difference between the compound and placebo was not statistically significant, Revelation said.

Adagio MAb posts strong data in a mostly pre-omicron study

ADG-20 (adintrevimab), Adagio Therapeutics Inc.’s lead candidate, a monoclonal antibody (MAb) for preventing and treating COVID-19, met the primary endpoints with statistical significance in phase II/III studies. Most of the data was collected when pre-omicron variants dominated and showed the risk of symptomatic COVID-19 was reduced by 71% compared to placebo in pre-exposure prophylaxis and 75% compared to placebo in post-exposure prophylaxis. Also, in a subset of the pre-exposure cohort after omicron emerged, there was a clinically meaningful reduction in cases of symptomatic COVID-19 found with adintrevimab compared to placebo. Adagio said it plans to file for emergency use authorization in the second quarter of 2022. The Waltham, Mass.-based company’s stock (NASDAQ:ADGI) surged 43% in midday trading.

Neuron23 adds $100M in series C, nominates LRRK2 inhibitor for clinical testing

Neuron23 Inc., a precision medicine company that came out of stealth mode in late 2020 with a $113.5 million combined series A and B round, has added another $100 million in a series C financing as it gears up to move into clinical testing this year with its lead candidate, an LRRK2 inhibitor for Parkinson’s disease. Funds also will advance a second candidate, targeting TYK2, which plays a role in pathological immune signaling, as well as support work on three undisclosed early stage programs. The series C was led by Softbank Vision Fund 2, with participation from series A and B investors Westlake Village Biopartners, Kleiner Perkins, Redmile Group, Cowen Healthcare Investments, Acorn Bioventures, HBM Partners, Perceptive Advisors and Surveyor Capital.

Elix and Shionogi partner to validate retrosynthetic analysis model to discover drugs

Elix Inc. has teamed up with Shionogi & Co. Ltd. to validate a retrosynthetic analysis model for drug discovery that will employ chemical reaction data from Shionogi to explore various routes to synthesizing new molecules. The project will employ Elix Synthesize, which uses parallel computation to explore various synthetic routes by processing large amounts of data at high speed. Financial terms were not disclosed.

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