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WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT) received confirmation that the U.S. Food and Drug Administration (FDA) has accepted its New Drug Application (NDA) for vatiquinone, designed to treat Friedreich’s ataxia (FA) in children and adults. The FDA has granted the application Priority Review status, with a target action date set for August 19, 2025. The company, currently valued at $3.82 billion, has seen its stock surge over 90% in the past year, according to InvestingPro data.
Friedreich’s ataxia is a rare and life-shortening neuromuscular disorder that affects the central nervous system and the heart. With symptoms typically emerging in childhood or adolescence, the condition leads to progressive loss of coordination and muscle strength. Currently, approximately 25,000 people worldwide are living with FA.
The Priority Review designation is significant as it accelerates the FDA’s review time from the standard ten months to six months, reflecting the agency’s belief that vatiquinone could potentially offer a significant improvement in the safety or effectiveness of the treatment, diagnosis, or prevention of a serious condition.
Vatiquinone, a small molecule selective inhibitor of 15-Lipoxygenase, aims to mitigate the effects of mitochondrial dysfunction and oxidative stress that are characteristic of FA. The NDA submission is supported by data from the MOVE-FA study and two additional long-term studies, which have collectively demonstrated the drug’s potential to slow disease progression and its safety across different age groups. InvestingPro analysis shows the company maintains strong liquidity with a current ratio of 2.1, indicating sufficient resources to support its drug development programs. Get access to 10+ additional ProTips and comprehensive financial analysis with an InvestingPro subscription.
PTC Therapeutics’ CEO, Matthew B. Klein, M.D., expressed optimism about the FDA’s acceptance of the NDA, emphasizing the urgent need for an approved therapy for FA, particularly for pediatric patients. The company is prepared to collaborate with the FDA throughout the review process.
The MOVE-FA trial, while not achieving statistical significance in its primary endpoint, did find a significant effect on a pre-specified endpoint of upright stability, which aligns with improvements in the 1-minute walk distance test and the Modified Fatigue Rating Scale.
This development marks the fourth NDA submission by PTC Therapeutics that the FDA has accepted for review in 2024. As the company awaits the FDA’s decision, this news offers hope to those affected by Friedreich’s ataxia and their families. With annual revenue of $900.66 million and two analysts recently revising earnings expectations upward, investors can access detailed financial analysis and Fair Value estimates through InvestingPro’s comprehensive research reports, available for over 1,400 US stocks.
The information reported is based on a press release statement from PTC Therapeutics.
In other recent news, PTC Therapeutics has been the subject of several noteworthy developments. The biopharmaceutical company reported approximately $814 million in revenue for 2024, surpassing its revenue guidance. This financial performance was largely driven by its Duchenne Muscular Dystrophy franchise, which includes products like Translarna™ and Emflaza®.
Analysts at Cantor Fitzgerald have shown increased confidence in PTC Therapeutics, raising the stock target to $113 from a previous $76, maintaining an Overweight rating. This upgrade is based on promising results from a Phase 3 trial and a strong financial position. Cowen also revised its financial model for the company, raising the stock price target to $50 from $46, while maintaining a Hold rating.
An important business development for PTC Therapeutics in 2024 was the closure of a license and collaboration agreement with Novartis (SIX:NOVN) for the PTC518 program. This deal brought in $1.0 billion in upfront proceeds for PTC (NASDAQ:PTC), with potential for up to $1.9 billion in additional milestones. TD Cowen also highlighted several regulatory catalysts involving PTC Therapeutics’ products Vatiquinone, Sepiapterin, and Translarna, which are expected to generate significant investor interest in 2025.
In a recent adjustment to its lease agreement with Warren CC Acquisitions, LLC, PTC Therapeutics reduced its rented space by half, modifying its financial terms. These recent developments underscore the dynamic nature of PTC Therapeutics’ operations and the biopharmaceutical industry as a whole.
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