Tharimmune, Inc. (NASDAQ:THAR) has received positive feedback from the European Medicines Agency (EMA) regarding its TH104 clinical program for treating moderate-to-severe chronic pruritus in primary biliary cholangitis (PBC). The EMA's endorsement supports Tharimmune's clinical and regulatory strategy, potentially accelerating the development timeline for TH104. This progress offers a promising outlook for PBC patients suffering from this debilitating symptom.
EMA's Recommendations and Study Design
The EMA has indicated that no additional animal studies are required, and they have accepted the Article 10(3) hybrid application approach, which allows the company to reference existing safety data from approved products. The agency found the proposed Phase 2 study design acceptable and provided guidance for future Phase 3 studies. This streamlined approach could reduce development costs and expedite the approval process.
Upcoming Phase 2 Trial
Tharimmune plans to initiate a Phase 2 multiple-ascending dose trial in the coming months to assess the safety and tolerability of TH104. The trial will also evaluate the change from baseline in itch scores to assess the drug's effectiveness in treating chronic pruritus in PBC patients. Topline data from this trial is expected in 2025.
Management Commentary
"We are pleased with our interactions with both the FDA and EMA," said Randy Milby, CEO of Tharimmune. "The recent regulatory feedback from the EMA builds on the previous positive interactions with the FDA while providing a roadmap for TH104 for chronic pruritus in PBC, which continues to be a debilitating symptom for patients suffering from this rare condition."
About TH104
TH104 consists of nalmefene embedded in a proprietary transdermal buccal film that adheres to the inside of the mouth. It is being developed as a potential treatment for multiple liver-related and other pruritogenic inflammatory conditions. The drug has a dual mechanism of action, affecting both the μ-opioid receptor and the kappa-opioid receptor, and potentially inhibiting IL-17 inflammatory cytokine expression. These opioid receptors are known to be involved in the body's itch circuitry.
Pruritus and Primary Biliary Cholangitis (PBC)
Primary biliary cholangitis (PBC) is a chronic disease where the bile ducts in the liver become dysfunctional, leading to a buildup of bile and subsequent liver damage. According to the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK), PBC affects an estimated 58 out of every 100,000 U.S. women and about 15 out of every 100,000 U.S. men. Pruritus, or chronic itching, is one of the most common and distressing symptoms associated with PBC, affecting up to 75% of individuals during their disease course. It significantly impacts the quality of life, and current treatment options are limited. Many patients describe the itch as "bugs crawling under the skin," with over 65% reporting that it worsens at night (nocturnal pruritus), highlighting a significant unmet need.
