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WARREN, N.J. - PTC Therapeutics, Inc. (NASDAQ: NASDAQ:PTCT), a biopharmaceutical company with a market capitalization of $3.4 billion and an impressive 60.8% stock return over the past year according to InvestingPro, announced that its unaudited total revenue for the year 2024 reached approximately $814 million, exceeding its revenue guidance. The company's financial performance was bolstered by its Duchenne Muscular Dystrophy (DMD) franchise, which generated significant sales from its products Translarna™ and Emflaza®.
In the past year, PTC Therapeutics also submitted four regulatory approval applications to the U.S. Food and Drug Administration (FDA), including for its gene therapy Kebilidi™, which received FDA approval in November 2024. The company is preparing for the global launch of sepiapterin for Phenylketonuria (PKU), with regulatory decisions expected in the second quarter and July 2025 from the Committee for Medicinal Products for Human Use (CHMP) and the FDA, respectively.
A significant business development for PTC (NASDAQ:PTC) in 2024 was the closure of a license and collaboration agreement with Novartis (SIX:NOVN) for the PTC518 program. The deal brought in $1.0 billion in upfront proceeds for PTC, and the company stands to gain up to $1.9 billion in additional milestones. PTC will also receive a 40% profit share on U.S. sales and tiered royalties on sales outside the U.S. Novartis will take on global development, manufacturing, and commercial responsibilities post-completion of the PIVOT-HD study's placebo-controlled part, expected in the first half of 2025.
The company also reported a strong cash balance of approximately $1.1 billion as of December 31, 2024. InvestingPro data shows a healthy current ratio of 2.1, indicating liquid assets exceed short-term obligations. This financial position is expected to support its operations and investment in its pipeline of medicines targeting rare disorders. InvestingPro's Financial Health Score rates PTC Therapeutics as "GOOD," suggesting solid operational fundamentals.
For the upcoming year, PTC anticipates total revenues to be between $600 million and $800 million, which includes projections for current products, potential new product launches, and royalty revenue. According to InvestingPro, analysts have revised their earnings upwards for the upcoming period, though they anticipate a sales decline in the current year. For deeper insights into PTC Therapeutics' financial outlook and access to comprehensive Pro Research Reports covering 1,400+ top stocks, consider exploring InvestingPro's advanced analytics platform. The GAAP R&D and SG&A expense for 2025 is estimated to be between $805 million and $835 million, with a non-GAAP expense projection, excluding stock-based compensation, of $730 million to $760 million.
These financial highlights and corporate updates were presented by Dr. Matthew B. Klein, Chief Executive Officer of PTC, at the 43rd Annual J.P. Morgan Healthcare Conference. The information provided in this update is based on preliminary unaudited data and management estimates for the full year 2024, subject to the completion of PTC's financial closing procedures. This update is based on a press release statement from PTC Therapeutics.
In other recent news, financial analysts have been adjusting their expectations for PTC Therapeutics following several key developments. The company has submitted an FDA application for its Friedreich ataxia treatment, vatiquinone, and has adjusted its lease terms, reducing its rented space. Moreover, PTC Therapeutics has entered into a substantial collaboration with Novartis for the drug candidate PTC518. This partnership includes a $1.0 billion upfront payment and potential for up to $1.9 billion in future milestone payments.
Morgan Stanley (NYSE:MS) has upgraded PTC Therapeutics from Equalweight to Overweight, setting a new price target of $67.00. This comes after the company reported strong third-quarter earnings with total revenue of $197 million, largely driven by the Duchenne muscular dystrophy franchise. However, Goldman Sachs and Baird have noted ongoing risks for the company, including forthcoming regulatory decisions in the US for vatiquinone.
TD Cowen has outlined ten anticipated catalysts for the year 2025 in the biotech sector. Among them, ANAB is expected to release Phase II rheumatoid arthritis data for Rosnilimab, and UTHR is looking to extend the success of its Tyvaso treatment. QURE is working towards submitting an accelerated approval application for AMT-130 in Huntington's disease, and ALKS is preparing to release Phase II datasets for its orexin candidate '2680. These recent developments in the biotech sector are expected to generate significant investor interest.
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