Travere Therapeutics, Inc., (Nasdaq: TVTX) today announced a voluntary pause of enrollment in the Phase 3 HARMONY Study evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). The voluntary enrollment pause enables the Company to work to address necessary process improvements in manufacturing scale-up to support commercial scale manufacturing as well as full enrollment in the HARMONY Study. Patients currently enrolled in pegtibatinase studies continue to receive study medication from small scale batches which are unaffected by the scale-up process. Currently enrolled patients will be able to continue on study medication as scheduled for the duration of the trials they are participating in.
The voluntary enrollment pause was enacted following the Company’s determination that the desired drug substance profile was not achieved in the recent scale-up process. The Company is in the process of notifying all study investigators of the decision to pause enrollment of new patients into the study until additional material is available.
The Company expects to further evaluate the necessary commercial process improvements to enable the continuation of the Phase 3 program and anticipates the earliest date to restart enrollment in the Phase 3 HARMONY Study will be in 2026. Previously planned investments related to clinical enrollment in HARMONY and large-scale production are expected to be delayed beyond 2025. Together with the expected decline in costs associated with the development of sparsentan as the Phase 3 programs advance towards completion, the Company now estimates that research and development expenses may be reduced by more than $30 million in 2025 compared to 2024. The Company continues to anticipate that its cash, cash equivalents and marketable securities of $325.4 million as of June 30, 2024 can support its operations into 2028.
Conference call information
Travere Therapeutics will host a conference call and webcast today, Thursday, September 26, 2024 at 4:30 p.m. ET to discuss the voluntary pause in enrollment. To participate in the conference call, dial +1 (323) 994-2093 (U.S.) or +1 (888) 394-8218 (International), confirmation code 5880214. The webcast can be accessed on the Investor page of Travere’s website at ir.travere.com/events-presentations. Following the live webcast, an archived version of the call will be available for 30 days on the Company’s website.
No comments:
Post a Comment
Note: Only a member of this blog may post a comment.