Verge Genomics and Stealth Bio Advance Novel Therapeutics in ALS


As the ALS space rides the momentum from the recent approval of Amylyx's Relyvrio, Verge Genomics and Stealth Biotherapeutics are each moving forward in early-stage clinical trials.

Verge Moves AI-Powered Therapeutic into Human Trials

Verge Genomics has dosed its first subject in a Phase I trial studying VRG50635, a therapeutic discovered through its AI-powered platform, the company announced Monday.

VRG50635 is a small molecule inhibitor of PIKfyve, a therapeutic target for ALS discovered by Verge’s proprietary platform, CONVERGE. CONVERGE is powered completely by AI and uses RNA, DNA and protein profiles to pinpoint new targets in CNS diseases. 

Jane Rhodes, chief business officer at Verge, told BioSpace the company's AI platform differs from others in that it uses human tissue instead of animal models.

“We use machine learning to map the complex causes of disease and develop those insights into proprietary drug candidates on our internal biology and chemistry platforms," Rhodes said. 

In a press release, Alice Zhang, CEO of Verge, touted the company’s ability to move the therapeutic into clinical trials in just four years. She said this achievement “offers clear proof” of Verge's commitment to the space and is a “leading indicator” of the potential of tech-enabled drug discovery. 

This is only the start of the company's plan to develop a robust clinical pipeline based on a human-to-human rather than an animal-to-human approach, she added. 

To that end, Rhodes told BioSpace that in addition to ALS, Verge is working on “a dozen breakthrough opportunities” in diseases with “no effective treatments to date,” like Parkinson’s disease and frontotemporal dementia. 

Stealth Bio’s SBT-272 Granted Orphan Designation

Stealth Bio’s ALS therapeutic, SBT-272, has been granted Orphan Drug Designation by the FDA, the company announced Tuesday. 

The designation was based on positive Phase I data in which SBT-272, a small molecule designed to restore mitochondrial function, showed promise in patients with ALS.

Preclinical data on SBT-272 will be presented Tuesday at the Northeast Amyotrophic Lateral Sclerosis (NEALS) conference in Clearwater, FL. 

The therapeutic targets mitochondria in upper motor neurons that are diseased with TDP-43 pathology, according to Hande Ozdinler, Ph.D., associate professor of neurology, Feinberg School of Medicine, Northwestern University. TDP-43 has previously been identified as a major pathological protein that drives neurodegeneration in ALS. 

“This also provides neuroprotection and reduces neuroinflammation in the motor cortex of a TDP-43 model of ALS",  Ozdinler said, adding that there is “compelling support” for the therapeutic’s potential to target mitochondria in ALS. 

Like Verge, Stealth Bio indicated in its announcement that it plans to evaluate the drug in other neurodegenerative diseases of mitochondrial dysfunction. 

SBT-272 has demonstrated mitochondria-protective and neuroprotective effects in models of ALS, FTD, alpha-synucleinopathy, Huntington's disease and ischemic stroke, according to the company.

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