WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for sepiapterin, a proposed treatment for phenylketonuria (PKU) in pediatric and adult patients. The application encompasses a full spectrum of ages and disease subtypes.
The NDA is supported by data from the phase 3 APHENITY trial, which reported a mean reduction in blood phenylalanine (Phe) levels of 63% among treated participants, and 69% in those with classical PKU. The majority of subjects in the trial achieved Phe control, with 84% reaching levels below 360 µmol/L, a threshold in line with treatment guidelines. Additionally, 22% of participants reached normal Phe levels.
Matthew B. Klein, M.D., CEO of PTC (NASDAQ:PTC) Therapeutics, stated that the submission is a significant step toward addressing the unmet medical needs of children and adults with PKU in the United States.
The submission also includes findings from an open-label extension study and a Phe tolerance sub-study, indicating that around 60% of subjects could consume protein above the recommended daily allowance without elevated Phe levels.
Sepiapterin, previously known as PTC923, is an oral synthetic precursor to tetrahydrobiopterin, a key enzyme cofactor involved in metabolizing several biological substances. It is positioned to treat a broad range of PKU patients and is more bioavailable than externally administered synthetic BH4.
PKU is a rare inherited disorder that affects the brain by impairing the breakdown of phenylalanine, leading to its accumulation and potentially causing severe and irreversible disabilities if not properly managed. Global estimates suggest that approximately 58,000 individuals are living with PKU.
PTC Therapeutics is a biopharmaceutical company focusing on the development and commercialization of medicines for rare disorders. The company's strategy includes leveraging scientific and clinical expertise to deliver treatments to patients with limited options.
The European marketing authorization application for sepiapterin is under review, and submissions for Japan and Brazil are anticipated later in 2024.
PTC Therapeutics has been making significant strides in its ongoing clinical trials and regulatory engagements. The company reported encouraging interim results from Part 2 of the Phase 2 PIVOT-HD trial, which is exploring 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.
Several analysts have updated their stance on PTC Therapeutics, with BofA Securities raising its price target to $32 while maintaining an Underperform rating. Goldman Sachs also reiterated a Sell rating with a price target of $32.
The company also received a boost from regulatory developments impacting its other products. The FDA granted Priority Review to Upstaza, a gene therapy candidate, and 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.
InvestingPro Insights
As PTC Therapeutics advances its treatment for phenylketonuria (PKU) with the recent submission of a New Drug Application, investors are closely monitoring the company's financial health and market potential. According to InvestingPro data, PTC Therapeutics has a market capitalization of approximately $2.49 billion. Despite a revenue growth of 20.39% over the last twelve months as of Q1 2024, analysts have expressed concern over the company's profitability. The revenue growth is a positive sign of the company's capacity to generate income, yet the company faces challenges as it has not been profitable during the same period, with a negative P/E ratio of -6.52.
Two key InvestingPro Tips for PTC Therapeutics include analysts' anticipation of a sales decline in the current year and the expectation that the company will not be profitable this year. These insights are particularly relevant for investors considering the potential impact of the FDA's decision on the company's financial performance. Additionally, PTC Therapeutics does not pay a dividend to shareholders, which may influence investment decisions for those seeking regular income from their investments.
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