Travere halts enrollment in homocystinuria study

Published 26/09/2024, 21:10
Travere halts enrollment in homocystinuria study

SAN DIEGO – Travere Therapeutics, Inc. (NASDAQ: TVTX), a biopharmaceutical company, today announced a temporary halt to patient enrollment in its Phase 3 HARMONY Study, which is assessing the efficacy of pegtibatinase in treating classical homocystinuria (HCU). The company cited the need for process improvements in manufacturing scale-up as the reason for the pause.

This voluntary suspension is designed to address and optimize the scale-up process for commercial manufacturing, which did not meet the desired drug substance profile in recent attempts. Despite the setback, Travere maintains that patients currently enrolled in pegtibatinase trials will not be affected, as they will continue to receive their medication from existing small-scale batches.

Senior Vice President of Research and Development, Bill Rote, Ph.D., expressed confidence in the company's ability to rectify the manufacturing issues, emphasizing the extensive biologics expertise of both their internal team and external partners.

The earliest anticipated date for resuming enrollment in the HARMONY Study is projected to be in 2026. As a result, related investments in clinical enrollment and large-scale production are expected to be postponed beyond 2025. However, Travere anticipates a reduction in research and development expenses by more than $30 million in 2025 compared to 2024, attributed to the progression of other Phase 3 programs, particularly for sparsentan, towards completion.

Travere also reported that its financial reserves, including cash, cash equivalents, and marketable securities totaling $325.4 million as of June 30, 2024, are projected to fund operations into 2028.

Classical HCU is a rare metabolic disorder that leads to elevated levels of homocysteine, causing life-threatening complications. Pegtibatinase, if successful, could become the first disease-modifying therapy for this condition. The drug has already received Breakthrough Therapy designation, Rare Pediatric Disease and Fast Track designations by the FDA, as well as Orphan Drug designation in the U.S. and Europe.

The company will host a conference call later today to discuss the enrollment pause further. This article is based on a press release statement from Travere Therapeutics.


In other recent news, Travere Therapeutics has paused enrollment in its Phase 3 HARMONY Study to facilitate improvements in manufacturing scale-up. The pause will not affect patients already participating in the study. Enrollment is expected to resume in 2026. On a positive note, Travere's drug Filspari has gained full FDA approval for the treatment of adult patients with primary Immunoglobulin A nephropathy (IgAN), potentially leading to sales growth. In fact, Filspari sales reached $27.1 million in the second quarter of 2024, a 37% increase over the first quarter. Analysts from Canaccord Genuity, Citi, and BofA Securities have maintained Buy ratings on Travere, with Canaccord Genuity and Citi raising their price targets. These recent developments highlight a period of significant progress for Travere Therapeutics.


InvestingPro Insights


As Travere Therapeutics faces a temporary halt in patient enrollment for its HARMONY Study, investors may be closely monitoring the company's financial health and stock performance. InvestingPro data reveals a market capitalization of $1.15 billion, which indicates the size of the company in the competitive biopharmaceutical landscape. Despite the challenges in manufacturing scale-up, the company's liquid assets are robust, with enough reserves to fund operations well into the future.

InvestingPro Tips suggest that Travere Therapeutics is experiencing a strong return over the past year, with a 70.46% price total return, reflecting investor optimism. Additionally, the company is operating with a moderate level of debt, which may provide some financial flexibility as they work through the current manufacturing issues. For investors looking for more detailed analysis and additional tips, there are 14 InvestingPro Tips available, which can be accessed for Travere Therapeutics at InvestingPro.

Moreover, the company's stock has been trading near its 52-week high, at 98.24% of the peak, which could signal market confidence or, as per another InvestingPro Tip, suggest that the stock is in overbought territory. With a Price / Book ratio of 75.9, the stock is trading at a high multiple, which may be of interest to value-focused investors. However, it's important to note that analysts do not anticipate the company will be profitable this year, a fact that could weigh on the stock's performance in the short term.

Overall, while Travere Therapeutics navigates through its current challenges, the InvestingPro insights provide a snapshot of the company's financial metrics and stock performance that could be valuable for investors considering their positions in the biopharmaceutical sector.

This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.

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