Avicenna gets patent for ROCK inhibitor compounds for ALS, more
Compounds show ability to reach central nervous system
Avicenna Biosciences has received its first patent covering new ROCK inhibitor compounds that may be used to treat amyotrophic lateral sclerosis (ALS) and similar disorders.
The U.S. patent grants Avicenna exclusive rights to pharmaceutical products containing inhibitors of the Rho associated protein kinases 1 and 2 (ROCK1/2). It also covers the use of such compounds in neurodegenerative conditions such as ALS and Parkinson’s disease.
“Securing this patent will protect and advance our work of pioneering much-needed treatments for diseases like ALS and Parkinson’s,” Thomas Kaiser, PhD, co-founder and chief scientific officer at Avicenna, said in a company press release.
The ROCK1 and ROCK2 proteins play essential roles in many cellular functions, including regulating cell shape and movement. But research has also demonstrated that ROCK can counteract neuronal survival, growth, and regeneration and contribute to inflammatory processes. Blocking these proteins could be a promising therapeutic strategy for ALS and other neurodegenerative diseases.
In ALS, targeting ROCK with an inhibitor called Bravyl has led to significant reductions in neurofilament light chain (NfL) levels, a sensitive and specific marker of nerve cell damage, and slower disease progression.
Reaching the brain
Using machine learning (ML) technology, Avicenna has developed ROCK inhibitors for ALS, Parkinson’s, and Charcot-Marie-Tooth disease. The company’s lead candidate for ALS, AV-016, “has demonstrated the greatest therapeutic benefit seen to date in an animal model” of ALS, the company says on its website.
Avicenna is conducting preclinical and pharmacological studies to file an investigational new drug application with the U.S. Food and Drug Administration requesting clearance to initiate clinical trials of AV-016 in humans.Â
The candidate being developed for Parkinson’s, AVI-009, has also shown potential in animal models of Parkinson’s, and Avicenna plans to launch human trials next year.
These oral ROCK inhibitors were selected based on their ability to reach the central nervous system (CNS), which consists of the brain and spinal cord. This is often a challenge that limits the success of most molecules in preclinical development.
“Our ML-driven medicinal chemistry platform has already enabled the discovery of the first orally available, CNS-penetrant ROCK inhibitor for the treatment of neurodegenerative diseases, with Investigational New Drug-enabling studies initiated just over one year since we began the Program,” Kaiser said.
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