FDA Grants Fast Track Designation for Chemomab’s Therapy for Primary Sclerosing Cholangitis

Rare Daily Staff

The U.S. Food and Drug Administration has granted Fast Track designation to Chemomab Therapeutics’ CM-101 for the treatment in adult patients of primary sclerosing cholangitis, a fibrotic liver disease that can result in liver transplant, cancer, and early death.

Primary Sclerosing Cholangitis (PSC) is a rare, progressive liver disease, characterized by inflammation and fibrosis (scarring) of the bile ducts. Eventually, it can lead to cirrhosis of the liver and liver failure. PSC also increases the risk of various cancers, which account for about half of PSC deaths. PSC affects an estimated 30,000 patients in the U.S. and about 80,000 worldwide. The underlying cause of PSC is unknown, but about 75 percent of individuals with PSC also have inflammatory bowel disease. Currently there are no FDA or EMA-approved therapies for patients with PSC. Liver transplant is common in advanced cases, but even then, PSC re-occurs in about 20 percent of transplanted patients. There is a high unmet need for therapeutic options to address the symptoms and modify the progression of this devastating illness.

CM-101 is a first-in-class monoclonal antibody that neutralizes the soluble protein CCL24, which in preclinical and clinical studies has been associated with key pathways underlying PSC pathophysiology. CM-101’s dual anti-inflammatory and anti-fibrotic activity, which is designed to break the vicious cycle driving these pathways, has demonstrated the potential for disease modifying activity in preclinical and early clinical studies of PSC-related processes. CM-101 was safe and well tolerated in phase 1 and phase 2 clinical trials to date.

Fast Track is a process developed by the FDA to facilitate and expedite the development of new treatments that demonstrate a potential to address unmet medical needs in serious or life-threatening conditions. Programs with Fast Track designation can benefit from early and more frequent interactions with the FDA during the clinical development process. Therapeutic candidates with Fast Track designation may also be eligible for priority review and accelerated approval if supported by clinical data.

“This FDA Fast Track designation is an important validation of CM-101’s potential to have a major impact on this devastating disease that attacks people in their prime years and lacks any approved treatments,” said Adi Mor, co-founder, CEO, and chief scientific officer of Chemomab. “We designed the CM-101 phase 2 SPRING trial to be supportive of a registrational trial in patients with PSC, and we welcome the enhanced opportunities for working closely with the FDA and for acceleration of the development and review process provided by Fast Track status.”

CM-101 has Orphan Drug designation from the FDA and Europe’s EMA, and is currently being evaluated in PSC patients in the Phase 2 SPRING trial, a double-blind, placebo-controlled study assessing the safety and tolerability of CM-101 in PSC patients. The trial is also measuring a wide range of relevant biomarkers and physiological parameters. Patient enrollment in the trial is advancing towards completion and Chemomab anticipates reporting a top-line readout in the second half of 2024.

Photo: Adi Mor, co-founder, CEO, and chief scientific officer of Chemomab