WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) revealed that the U.S. Food and Drug Administration (FDA) has accepted for filing its New Drug Application (NDA) for sepiapterin, a treatment designed for both pediatric and adult patients with phenylketonuria (PKU). The company expects to receive a Prescription Drug User Fee Act (PDUFA) target action date in the upcoming Day 74 Letter.
This acceptance marks a significant step for PTC (NASDAQ:PTC) Therapeutics in its quest to provide a new therapeutic option for those affected by PKU, a rare metabolic disorder. The NDA submission is supported by data from the phase 3 APHENITY trial, which showed a notable reduction in blood phenylalanine levels among treated patients. Specifically, the trial reported a 63% mean decrease in these levels across the treated population, with a 69% reduction observed in subjects with classical PKU.
The majority of trial participants (84%) achieved phenylalanine control to the recommended levels, and 22% experienced normalization of these levels. Further evidence from the APHENITY open-label extension study suggests the long-term efficacy of sepiapterin, while the Phe tolerance sub-study indicates that around 60% of subjects could achieve protein intake above the recommended daily allowance without elevated phenylalanine levels.
Sepiapterin, known formerly as PTC923, is an oral synthetic compound that functions through a dual mechanism. It acts as a precursor to tetrahydrobiopterin (BH4), a vital cofactor for the phenylalanine hydroxylase (PAH) enzyme, and separately aids in preventing PAH misfolding, thereby enhancing enzyme function.
PKU is characterized by the inability to break down phenylalanine, an essential amino acid, due to a gene defect. Without proper management, phenylalanine can accumulate to toxic levels, leading to severe disabilities, including intellectual disability, seizures, and behavioral issues. Globally, an estimated 58,000 individuals live with PKU, with diagnosis typically occurring through newborn screening programs.
PTC Therapeutics focuses on developing medicines for rare disorders and believes that the potential approval of sepiapterin could address a significant unmet need for PKU patients. The company emphasizes that while there are risks in the development and commercialization of new pharmaceutical products, they are committed to advancing treatments for patients with limited options.
This news is based on a press release statement from PTC Therapeutics, Inc.
In other recent news, PTC Therapeutics reported noteworthy advancements in its drug development pipeline and financial performance. The company's Q2 2024 revenue hit $187 million, primarily driven by its Duchenne muscular dystrophy franchise. PTC Therapeutics also updated its full-year revenue guidance, projecting between $700 million to $750 million.
The FDA granted Fast Track Designation to PTC Therapeutics' drug candidate PTC518, developed for Huntington's disease treatment. The designation is seen as a significant step for PTC518, expediting the drug's development and review process. Baird, which issued an Outperform rating for the company, views this as a promising aspect of PTC Therapeutics' pipeline.
TD Cowen maintained its Hold rating on PTC Therapeutics, while the company also made progress in several clinical trials and regulatory submissions. This includes the submission of a New Drug Application for sepiapterin, a potential treatment for phenylketonuria, and the resubmission of the NDA for Translarna.
Furthermore, PTC Therapeutics 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 to focus on advancing its pipeline of rare disease treatments and meeting its projected milestones for the remainder of 2024.
InvestingPro Insights
PTC Therapeutics' recent FDA acceptance of its NDA for sepiapterin aligns with the company's focus on rare disorders and could potentially impact its financial outlook. According to InvestingPro data, PTC Therapeutics has shown strong market performance, with a 65.55% price total return over the past year and a 29.77% return in the last six months.
Despite these positive market indicators, InvestingPro Tips reveal that analysts anticipate a sales decline in the current year, and the company is not expected to be profitable this year. This information suggests that while the sepiapterin NDA acceptance is a positive development, it may take time for it to translate into financial gains.
The company's revenue for the last twelve months as of Q2 2023 stood at $900.45 million, with a revenue growth of 9.98%. However, the operating income for the same period was negative at -$192.45 million, indicating ongoing challenges in profitability.
It's worth noting that PTC Therapeutics' liquid assets exceed short-term obligations, which could provide financial flexibility as they navigate the regulatory process and potential commercialization of sepiapterin.
For investors seeking a more comprehensive analysis, InvestingPro offers 7 additional tips for PTC Therapeutics, providing a deeper understanding of the company's financial health and market position.
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