CS-1103

The Phase 1 study for CS-1103 met its objective, demonstrating a favorable safety and tolerability profile with no serious adverse events Phase 2 studies for the treatment of methamphetamine and fentanyl overdose are planned for early 2025 CAMBRIDGE, Mass., Sept. 10, 2024 /PRNewswire/ -- Clear Scientific, Inc., a clinical-stage biopharmaceutical company focused on developing treatments for conditions and diseases caused by an excess of harmful substances in the body, today announced positive topline data from the First-in-Human Phase 1 clinical trial of its therapeutic CS-1103 that demonstrate a favorable safety and tolerability profile. All thirty-two healthy participants recruited, divided into 4 cohorts, completed the Phase 1 single ascending dose, randomized, double-blinded, placebo-controlled, safety, tolerability and pharmacokinetics study. In this study, single doses of CS-1103, administered via intravenous injection, were well tolerated. There were no serious adverse events reported. The Phase 1 study supports the dose requirement for the Phase 2 studies on meth and fentanyl overdose, planned for early 2025. "The phase 1 results clear a critical hurdle, as together with the pre-clinical studies, convincingly demonstrate both safety and efficacy of CS-1103. Based on these strong results, we are working toward an accelerated registration pathway with Phase 2 studies to begin in early 2025. We thank the National Institute on Drug Abuse (NIDA) of the NIH for their continued support," said Shekar Shetty, Clear Scientific CEO. About the Overdose Epidemic The US overdose crisis continues unabated, with 1.9M emergency department (ED) visits and over 100,000 deaths annually. According to the CDC, drug overdose mortality rates are at the highest level they've ever been, and overdose is the leading cause of death for Americans under the age of 50. There are currently approved medications for opioid overdose, however, there are no approved treatments for overdose with stimulants or overdoses that result from drug combinations. About CS-1103 CS-1103 is a first-in-class injectable small molecule sequestrant that immediately binds to and inactivates target compounds, rapidly reversing their toxic effects and accelerating clearance into urine. Clear Scientific is developing CS-1103 for treatment of overdose and selected metabolic, central nervous system, and neurodegenerative disorders. About Clear Scientific Clear Scientific, a clinical-stage biopharmaceutical company co-founded by George Whitesides of Harvard University, is pioneering a new paradigm for treatment of conditions and diseases that involve an excess of specific compounds in the body. This approach focuses on development of highly selective, small molecule sequestrants that bind, immediately inactivate, and rapidly clear excess compounds from the body. Research reported in this press release was supported by the National Institute on Drug Abuse of the National Institutes of Health under award number U01DA053054. The content is solely the responsibility of the authors and does not necessarily represent the official views of the National Institutes of Health. SOURCE Clear Scientific

There is a push for disease-modifying therapies in the ALS space as both approved drugs for the condition only manage the disease. Credit: Green Apple via Shutterstock. Ionis and Biogen are halting the development of their experimental treatment for amyotrophic lateral sclerosis (ALS) after it failed to show improvement in patients in a Phase I/II study. Data from the 99-patient ALSpire study (NCT04494256) demonstrated that BIIB105 failed to significantly reduce neurodegeneration or improve functional measures such as breathing. BIIB105, an investigational antisense oligonucleotide was designed to reduce the expression of ataxin-2 (ATXN2) protein. Despite demonstrating statistically significant ATXN2 reductions in the study, the drug showed no evidence of clinical benefit in any subgroup evaluated in the study. This marks another setback in the search for new treatments for ALS, a progressive neurodegenerative disease that causes muscle weakness and paralysis. Last month, Amylyx Pharmaceuticals axed its ALS drug Relyvrio (AMX0035) from the market after its Phase III Phoenix trial failed to meet any of its endpoints. However, this trial failure does not mark the end of Ionis and Biogen’s quest with ALS. The ALS candidate Qalsody (tofersen), which was developed by both, received an accelerated approval from the US Food and Drug Administration (FDA) in April 2023. The decision followed a recommendation from an FDA advisory committee where experts agreed that Qalsody’s ability to reduce levels of plasma neurofilament light (NfL), a biomarker of nerve injury and neurodegeneration, provided enough basis for a conditional approval. However, the panel voted against a full approval after a failed Phase III trial back in 2021. GlobalData’s Pharmaceutical Intelligence Centre predicts global sales of Qalsody to reach $127m in 2030. See Also: ION-356 by Ionis Pharmaceuticals for Pelizaeus-Merzbacher Disease (PMD): Likelihood of Approval CS-1103 by Clear Scientific for Substance (Drug) Abuse: Likelihood of Approval GlobalData is the parent company of Pharmaceutical Technology. Last month, BrainStorm Cell Therapeutics announced that it will be initiating a Phase IIIb confirmatory trial of its ALS candidate NurOwn (debamestrocel) following a previous Phase III failure. The FDA provided a written agreement under a special protocol assessment (SPA), on the design for the Phase IIIb trial of the stem cell therapy. There are only two drugs approved for the treatment of ALS: Mitsubishi Tanabe’s Radicava (edaravone) and generic drug riluzole. According to a report on GlobalData’s Pharma Intelligence Center, there will be 74,614 cases of ALS in the eight major markets (UK, US, Spain, Germany, Italy, France, Japan, and Canada). In the announcement accompanying the trial results, Stephanie Fradette, head of the neuromuscular development unit at Biogen said: “While BIIB105 lowered ATXN2 protein, it did not reduce neurofilament, which gives us confidence that BIIB105 did not slow the disease process.”