This news is based on a press release statement. PTC Therapeutics (NASDAQ:PTCT) will hold a conference call today at 8:30 am EST to discuss the agreement further. For deeper insights into PTC (NASDAQ:PTC) Therapeutics' financial health and growth prospects, including 10 exclusive ProTips and comprehensive valuation metrics, visit InvestingPro, where you'll find detailed analysis in our Pro Research Report, part of our coverage of over 1,400 US stocks. For deeper insights into PTC Therapeutics' financial health and growth prospects, including 10 exclusive ProTips and comprehensive valuation metrics, visit InvestingPro, where you'll find detailed analysis in our Pro Research Report, part of our coverage of over 1,400 US stocks.
The agreement, announced today, positions PTC518 as a leading oral disease-modifying therapy for Huntington's disease (HD), a fatal genetic disorder. PTC's CEO, Matthew B. Klein, M.D., highlighted the potential of PTC518 to meet the urgent needs of HD patients globally and noted the deal's alignment with the treatment's promise. With a healthy current ratio of 2.1, indicating strong liquidity, and last twelve months revenue of $900.66 million, PTC plans to invest the proceeds to further develop its splicing platform and support its commercial and development portfolio.
Novartis (SIX:NOVN) CEO Vas Narasimhan expressed the company's commitment to advancing neuroscience and addressing neurodegenerative diseases with high unmet needs. Novartis will take over global development, manufacturing, and commercialization responsibilities for PTC518 following the completion of the placebo-controlled segment of the ongoing PIVOT-HD study, expected in the first half of 2025.
PTC518, discovered from PTC's validated splicing platform, has shown promising results in Phase 2 trials, including a reduction in mutant Huntingtin protein levels and early signs of clinical benefit. The treatment has maintained a favorable safety profile throughout the studies.
The financial arrangement between PTC and Novartis includes a 40/60 profit and loss share in the U.S. market. The deal's completion is subject to regulatory approvals and customary closing conditions, with closure anticipated in the first quarter of 2025.
The press release indicates that Huntington's disease affects the central nervous system, causing a wide range of symptoms, including abnormal movements and cognitive issues. Currently, there is no cure or approved treatment to delay the onset or progression of HD.
This news is based on a press release statement. PTC Therapeutics will hold a conference call today at 8:30 am EST to discuss the agreement further.
In other recent news, PTC Therapeutics has experienced several notable developments. The financial services firm Baird raised the company's stock price target to $52.00, up from $48.00, maintaining an Outperform rating. This adjustment followed an event focusing on Friedreich's ataxia, a rare genetic disease, and reflects optimism about the potential of sepiapterin, a drug in PTC Therapeutics' pipeline.
Additionally, PTC Therapeutics sold its Rare Pediatric Disease Priority Review Voucher for $150 million, capitalizing on regulatory achievements. The company also raised its 2024 revenue outlook to between $750 million and $800 million, following strong third-quarter earnings with a total revenue of $197 million. Much of this revenue, $124 million, was generated by the Duchenne muscular dystrophy franchise, including Emflaza.
PTC Therapeutics has submitted two New Drug Applications to the FDA and is preparing for global product launches, including those of sepiapterin and vatiquinone. The launch of sepiapterin is planned across 50 countries, with potential revenue exceeding $1 billion in the U.S. alone. The company has also received positive feedback from key opinion leaders on vatiquinone's efficacy and safety. Despite these developments, PTC Therapeutics maintains a solid financial foundation, with cash reserves exceeding $1 billion.
This article was generated with the support of AI and reviewed by an editor. For more information see our T&C.