Tharimmune, Inc. has announced positive feedback from the European Medicines Agency (EMA) regarding its clinical development program for TH104, a therapeutic candidate targeting moderate-to-severe pruritus (itching) in patients with primary biliary cholangitis (PBC). The EMA's guidance supports Tharimmune's planned Phase 2 trial and provides a roadmap for future Phase 3 studies, potentially accelerating the drug's path to market for this debilitating condition.
EMA Endorsement of Clinical Strategy
The EMA's feedback specifically addressed the Phase 2 and Phase 3 clinical program for TH104. The agency indicated that utilizing Article 10(3), a hybrid application, is an acceptable approach. This could allow Tharimmune to leverage existing non-clinical and safety data from approved products, potentially reducing the need for extensive new studies. The EMA endorsed Tharimmune's non-clinical strategy, stating that no additional animal studies are needed and that human exposure data is sufficient to proceed.
Phase 2 Trial Design and Future Development
The EMA found the design and main features of the proposed Phase 2 study to be overall acceptable, with some comments provided to further refine the protocol. The agency also offered general guidance for the design of a future Phase 3 study, providing a clear regulatory pathway for TH104's development.
Randy Milby, CEO of Tharimmune, stated, "We are pleased with our interactions with both the FDA and EMA. 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."
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 efficacy in alleviating chronic pruritus in PBC patients. Topline data from the Phase 2 trial is expected in 2025.
About TH104 and its Mechanism of Action
TH104 is a novel formulation of nalmefene delivered via a proprietary transdermal buccal film. It is being developed for various 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: An Unmet Need
Primary biliary cholangitis (PBC) is a chronic disease affecting the bile ducts in the liver, leading to bile buildup and liver damage. It is estimated to affect 58 out of every 100,000 U.S. women and about 15 out of every 100,000 U.S. men. Pruritus is a common and debilitating symptom of PBC, affecting up to 75% of patients. It significantly impacts quality of life, and current treatment options are limited. Many patients describe the itch as feeling like "bugs crawling under the skin," and it is often worse at night (nocturnal pruritus), representing a significant unmet medical need.
