WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ:PTCT) disclosed today that it will request a re-examination of the Committee for Medicinal Products for Human Use (CHMP) negative opinion regarding the renewal of the conditional marketing authorization for its drug, Translarna™ (ataluren). The CHMP's decision follows a previous negative stance by the European Commission (EC), which was sent back for further review.
Translarna's marketing authorization continues to be valid while the re-examination process is underway, with the expectation from PTC (NASDAQ:PTC) that the drug will be available through the end of 2024, even if the negative opinion is ultimately confirmed and ratified by the EC.
Chief Executive Officer of PTC Therapeutics, Dr. Matthew B. Klein, expressed disappointment with the CHMP's reaffirmed negative opinion, despite considering new evidence including the STRIDE real-world data.
He highlighted that the Scientific Advisory Group, part of the review, advised that the efficacy evidence from STRIDE should not be disregarded, aligning with the opinions of key leaders in Duchenne muscular dystrophy (DMD) treatment.
Translarna, a product of PTC's research and development, is a protein restoration therapy aimed at treating genetic disorders caused by nonsense mutations. It is currently approved in several countries for treating nonsense mutation Duchenne muscular dystrophy (nmDMD) in ambulatory patients aged two and older. In the United States, ataluren is classified as an investigational new drug.
Duchenne is a rare, fatal genetic disorder predominantly affecting males, characterized by progressive muscle weakness leading to early loss of mobility and premature death in the mid-20s due to heart and respiratory failure.
PTC Therapeutics is a biopharmaceutical company dedicated to the discovery, development, and commercialization of medicines for rare disorders. The company's mission is to provide access to treatments for patients with limited or no therapeutic options.
This announcement is based on a press release statement. The company's forward-looking statements are subject to various risks and uncertainties, including the outcome of negotiations with third-party payors, the ability to maintain marketing authorization, and the commercial potential of its products. These statements represent the company's position as of the date of the press release and are subject to change after that date.
In other recent news, PTC Therapeutics has seen significant developments in its clinical trials and regulatory interactions. The company revealed interim 12-month results from Part 2 of the Phase 2 PIVOT-HD trial, evaluating PTC518 for the treatment of Huntington's disease. The treatment led to a dose-dependent reduction in mutant huntingtin protein in both peripheral blood and cerebrospinal fluid.
BofA Securities updated its stance on PTC Therapeutics shares, increasing its price target to $32 from $25, while maintaining an Underperform rating. Goldman Sachs (NYSE:GS) also reiterated a Sell rating on PTC Therapeutics, with a price target of $32.00. Meanwhile, JPMorgan (NYSE:JPM) maintained its Overweight rating and $53.00 price target for PTC Therapeutics, citing the encouraging clinical data.
The U.S. Food and Drug Administration (FDA) lifted a partial clinical hold on PTC518, allowing PTC Therapeutics to proceed with further development. Regulatory developments also impacted other products of the company, with the FDA granting Priority Review to Upstaza, a gene therapy candidate.
The European Commission decided not to endorse a negative opinion on Translarna, leading to adjustments in the company's stock ratings and price targets by firms such as Jefferies, and Raymond James.
Jefferies raised the price target to $46, maintaining a Buy rating, and Raymond James upgraded PTC Therapeutics from Underperform to Market Perform. These are the latest developments in PTC Therapeutics' pursuit of advancing treatments for rare disorders.
InvestingPro Insights
PTC Therapeutics (NASDAQ:PTCT) is navigating a challenging period with its drug Translarna™, and the financial metrics reflect a company in a state of flux. According to recent data from InvestingPro, the company's market capitalization stands at approximately $2.66 billion USD.
Despite a notable revenue growth of 20.39% over the last twelve months as of Q1 2024, analysts are not optimistic about the company's profitability in the near term, with a current P/E Ratio at -4.54, and an adjusted P/E Ratio for the last twelve months at -6.94.
The company's stock has experienced a strong return over the last three months, with a 19.15% increase, which could be indicative of investor confidence in the company's long-term prospects or a response to specific market conditions. It is important to note that PTC Therapeutics does not pay a dividend, which may affect the investment decisions of income-focused shareholders.
InvestingPro Tips reveal that analysts anticipate a sales decline in the current year and do not expect the company to be profitable this year. These insights, coupled with the fact that the company was not profitable over the last twelve months, may be crucial for investors considering the potential risks and rewards associated with PTCT's stock.
For those looking to delve deeper into the financials and forecasts for PTC Therapeutics, there are additional InvestingPro Tips available at https://www.investing.com/pro/PTCT. To access these valuable insights, use the coupon code PRONEWS24 to get an additional 10% off a yearly or biyearly Pro and Pro+ subscription.
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