Travere halts enrollment in Phase 3 HARMONY Study

SAN DIEGO, CA - Travere Therapeutics, Inc. (NASDAQ:TVTX) announced today a voluntary pause in enrolling new patients in its Phase 3 HARMONY Study, which is evaluating pegtibatinase for the treatment of classical homocystinuria (HCU). The decision was made to facilitate process improvements in manufacturing scale-up, which are necessary for supporting commercial-scale production and the complete enrollment of the HARMONY Study.

Patients who are already participating in the pegtibatinase studies will continue to receive their medication from existing small-scale batches, which are not affected by the scale-up process. These patients will be able to maintain their treatment schedules throughout the duration of the trials.

The company identified that the recent scale-up process did not yield the desired drug substance profile, prompting the enrollment pause. Travere Therapeutics is in the process of informing all study investigators of this development and has indicated that enrollment is expected to resume at the earliest in 2026, once the necessary improvements have been made.

This announcement contains forward-looking statements regarding the company's plans to address manufacturing scale-up issues and the anticipated timeline for resuming enrollment in the Phase 3 HARMONY Study. These statements are subject to various risks and uncertainties, including challenges related to manufacturing processes and regulatory approvals. The company's ability to successfully implement these improvements and the impact on its clinical trials and commercial products are not guaranteed.

Travere Therapeutics is also navigating risks associated with its current commercial products, the market reception of its therapies, and the development of its product pipeline. The company acknowledges the potential for global and macroeconomic factors, such as health epidemics and pandemics, to disrupt clinical trials, commercial activities, and manufacturing operations.

This report is based on information contained in a press release statement filed with the Securities and Exchange Commission on the same date.

In other recent news, Travere Therapeutics has seen significant developments, with its drug Filspari gaining full FDA approval for the treatment of adult patients with primary Immunoglobulin A nephropathy (IgAN) at risk of disease progression. The approval, based on long-term results from the PROTECT Study, is expected to increase the eligible patient population, leading to potential sales growth. Financially, Travere reported Filspari sales reaching $27.1 million in the second quarter of 2024, a 37% increase over the first quarter, and maintains a solid financial position with $325.4 million in cash and securities.

Analysts from Canaccord Genuity, Citi, and BofA Securities have all maintained Buy ratings on Travere, with Canaccord Genuity and Citi raising their price targets to $23.00 and BofA Securities to $19.00. These adjustments reflect the FDA's approval of Filspari and its potential to expand the market size. In addition, Canaccord Genuity revised its model to reflect an increased peak U.S. market penetration forecast for Filspari, now estimated at 13.8% by 2031.

These are recent developments for Travere Therapeutics and highlight a period of significant progress, particularly with the successful launch of Filspari. The company is also in discussions with the FDA regarding a potential modification to the liver-monitoring Risk Evaluation and Mitigation Strategy (REMS). Travere Therapeutics anticipates further growth acceleration for Filspari following the upcoming PDUFA date for full approval.

InvestingPro Insights

As Travere Therapeutics (NASDAQ:TVTX) faces challenges with its Phase 3 HARMONY Study, investors and stakeholders might be interested in the company's financial health and market performance. According to real-time data from InvestingPro, the company currently has a market capitalization of approximately $1.15 billion. Despite a significant revenue growth of 47.42% in the last twelve months as of Q2 2024, Travere's gross profit margin remains negative at -31.44%, indicating the company is spending more than it earns on its cost of goods sold.

InvestingPro Tips highlight that Travere Therapeutics is quickly burning through cash and analysts do not anticipate the company will be profitable this year. Moreover, the Relative Strength Index (RSI) suggests the stock is in overbought territory, which could signal a potential retraction in its stock price. On a more positive note, the company's liquid assets exceed its short-term obligations, and it has managed a high return over the last year. For investors seeking a deeper dive into Travere Therapeutics' financials and performance metrics, there are over 13 additional InvestingPro Tips available at InvestingPro.

With the company trading near its 52-week high and at a high Price/Book multiple of 75.9, it is essential to consider these financial metrics in the context of the company's recent announcements and the volatile nature of the biotech industry. The InvestingPro Fair Value estimate is currently set at $15.53, which may serve as a reference point for investors gauging the stock's potential value against its recent trading price of $14.54.

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