ABLi Therapeutics Launches to Develop c-Abl Kinase-Based Neurodegenerative Treatments

ABLi Therapeutics, a newly launched biotechnology firm, is making strides in the fight against neurodegenerative diseases like Parkinson’s. Spearheaded by Dr. Milton H. Werner, the company is focusing on the development of its lead candidate, Risvodetinib (ABLi-148009). This oral, once-daily medication is a selective c-Abl kinase inhibitor designed to penetrate the brain effectively. It holds the potential to modify the course of Parkinson's disease and related conditions by targeting the root causes rather than merely alleviating symptoms.

The vision for ABLi was born from Dr. Werner’s extensive research into c-Abl kinases, proteins known to play a role in numerous diseases. Alongside a team of seasoned drug development professionals, Dr. Werner has spent the last decade developing Risvodetinib and other promising drug candidates. The goal is to improve patient outcomes through therapies that are not only disease-modifying but also suitable for long-term systemic use.

A significant challenge in treating Parkinson’s is the lack of therapies that can stop or reverse disease progression. Dr. Werner emphasizes the importance of ABLi’s RAMP™ drug innovation engine, which has been crucial in developing Risvodetinib. This engine has produced a series of novel c-Abl inhibitors that excel in potency and selectivity compared to existing options. These inhibitors achieve effective concentrations both in the brain and throughout the body, showing initial promising results in terms of safety, tolerability, and efficacy in Parkinson’s-related conditions.

ABLi boasts a leadership team with vast experience in pharmaceutical innovation and company growth. The team includes Milton H. Werner, PhD, as Chairman and CEO, Garth Lees-Rolfe as Chief Financial Officer, and Dr. Karl Kieburtz, serving as the Interim Chief Medical Officer. Furthermore, ABLi's Scientific Advisory Board is composed of distinguished experts like Dr. Robert Hauser, Dr. Jeffrey Kordower, Dr. Ken Marek, Dr. Wolfgang Singer, and Dr. Wassilios Meissner, who support and guide the company's scientific pursuits.

Risvodetinib stands out for its potential as a disease-modifying therapy that combats Parkinson's at its core, potentially halting disease progression and reversing functional loss. Unlike existing treatments that only manage symptoms, this drug aims to change the disease's trajectory. Recent clinical trials have shown Risvodetinib to be the first monotherapy to enhance quality of life in Parkinson’s patients while simultaneously addressing the underlying disease pathology. The drug is protected by intellectual property rights extending beyond 2036.

ABLi Therapeutics leverages its deep understanding of medicinal chemistry and disease biology to create small molecule therapeutics. The company focuses on diseases stemming from the activation or dysfunction of Abelson Tyrosine Kinases, such as Parkinson’s disease and related conditions like Multiple System Atrophy and Dementia with Lewy Bodies. With a commitment to addressing the causes of these diseases, ABLi aims to develop treatments that offer improved brain penetration, heightened potency, and better safety profiles.

By advancing these cutting-edge therapies, ABLi Therapeutics aspires to transform the treatment landscape for neurodegenerative diseases, offering new hope to patients affected by these debilitating conditions.