WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) today announced significant advancements in its vatiquinone treatment program for Friedreich ataxia (FA), a rare neuromuscular disorder. The company confirmed that the primary endpoint was met in long-term extension studies of vatiquinone, showing a substantial slowing of disease progression.
The MOVE-FA long-term extension study demonstrated a 3.7-point benefit on the modified Friedreich Ataxia Rating Scale (mFARS) after 144 weeks of treatment, indicating a 50% reduction in disease progression over three years compared to a natural history cohort. Moreover, vatiquinone has maintained a strong safety profile throughout the trials, with no serious adverse events related to the treatment reported.
Matthew B. Klein, M.D., Chief Executive Officer of PTC (NASDAQ:PTC) Therapeutics, expressed confidence in vatiquinone's potential, particularly for children and adolescents who currently lack approved therapies for FA. PTC is preparing for a New Drug Application (NDA) submission to the FDA in December 2024, which will include data from the MOVE-FA study and long-term treatment analyses.
The MOVE-FA trial, which enrolled 146 patients, did not show statistical significance in the overall mFARS score change but did reveal a significant effect on the upright stability subscale, particularly relevant for pediatric and young adult patients. The planned NDA will also feature mechanistic data demonstrating vatiquinone's impact on biomarkers of disease pathology.
Jennifer Farmer, Chief Executive Officer of the Friedreich's Ataxia Research Alliance (FARA), praised the trial's conduct and the open-label extension data, highlighting the positive results on clinical endpoints and the ongoing safety and tolerability of the treatment.
Vatiquinone, a first-in-class selective inhibitor of 15-Lipoxygenase, addresses mitochondrial dysfunction and oxidative stress in FA, which can lead to cell death through ferroptosis. The treatment has been evaluated in various clinical studies, showing an impact on mortality risk and symptoms of neurological and neuromuscular diseases.
FA affects the central nervous system and the heart, leading to a range of symptoms, including loss of coordination, muscle strength, and serious heart conditions. Approximately 25,000 people globally suffer from this genetic disorder.
This announcement is based on a press release statement from PTC Therapeutics, Inc.
In other recent news, PTC Therapeutics has made significant strides in its drug development pipeline. The U.S. Food and Drug Administration (FDA) accepted the company's New Drug Application for sepiapterin, a potential treatment for phenylketonuria (PKU). Data from the phase 3 APHENITY trial supports this submission, showing a significant reduction in blood phenylalanine levels among treated patients. The FDA also granted Fast Track Designation to PTC Therapeutics' drug candidate PTC518, developed for Huntington's disease treatment. This designation expedites the drug's development and review process.
Baird has initiated coverage on PTC Therapeutics with an Outperform rating, while TD Cowen maintained its Hold rating on the company. In financial news, PTC Therapeutics reported Q2 2024 revenue of $187 million, primarily driven by its Duchenne muscular dystrophy franchise, and updated its full-year revenue guidance to project between $700 million to $750 million.
The company also completed the sale of their gene therapy manufacturing business, receiving an upfront payment of $27.5 million. These are the recent developments surrounding PTC Therapeutics, as the company continues its focus on advancing treatments for patients with rare disorders.
InvestingPro Insights
PTC Therapeutics' recent announcement about its vatiquinone treatment program for Friedreich ataxia aligns with some key financial insights from InvestingPro. The company's focus on developing innovative treatments for rare diseases is reflected in its financial performance and market position.
According to InvestingPro data, PTC Therapeutics has shown strong revenue growth, with a 9.98% increase in the last twelve months as of Q2 2024. This growth trajectory supports the company's ability to invest in research and development for treatments like vatiquinone. However, it's worth noting that analysts anticipate a sales decline in the current year, which could be influenced by various factors including the timing of new drug approvals and market launches.
Despite the positive news on vatiquinone, InvestingPro Tips indicate that PTC Therapeutics is not currently profitable, with a negative P/E ratio of -7.64 over the last twelve months. This is not uncommon for biotech companies investing heavily in drug development. The company's liquid assets exceeding short-term obligations suggest a stable financial position to support ongoing clinical trials and potential regulatory submissions.
Investors seem optimistic about PTC Therapeutics' prospects, as evidenced by the large price uptick over the last six months, with a 30.17% total return. This aligns with the positive long-term extension study results for vatiquinone and the planned NDA submission.
For readers interested in a more comprehensive analysis, InvestingPro offers 8 additional tips for PTC Therapeutics, providing a deeper understanding of the company's financial health and market position.
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