PTC Therapeutics Q3 2024 Corporate Update and Financial Results

15 November 2024
PTC Therapeutics, Inc. (NASDAQ: PTCT), headquartered in Warren, N.J., released its corporate update and financial results for the third quarter ending September 30, 2024. Dr. Matthew Klein, CEO, expressed pride in the team's performance and highlighted the company's robust financial position and strategic achievements. The third quarter of 2024 saw total revenue of $197 million, with $124 million attributed to its DMD franchise.

PTC's DMD franchise showcased significant performance, with Translarna™ (ataluren) contributing $72 million and Emflaza® (deflazacort) bringing in $52 million. These figures were bolstered by consistent patient access, geographic expansion, compliance, adherence, and dose adjustments.

Among the key clinical and regulatory milestones, PTC submitted a New Drug Application (NDA) to the FDA for sepiapterin for treating PKU, with a regulatory action date set for July 29, 2025. Additionally, PTC resubmitted the NDA for Translarna™ for treating nmDMD, though a regulatory action date remains unspecified. The Biologics License Application (BLA) for gene therapy targeting AADC deficiency is aligned for a regulatory action by November 13, 2024. The company also received Fast Track Designation for its PTC518 Huntington's disease program and plans to submit an NDA for vatiquinone to treat Friedreich ataxia by December 2024. Furthermore, PTC is scheduled to announce topline data from the CardinALS trial for utreloxastat in ALS during the fourth quarter of 2024.

In terms of financial performance, PTC's third-quarter 2024 total revenue was $196.8 million, marking a slight increase from $196.6 million in the same period in 2023. Product revenues were $135.4 million compared to $144.0 million in the previous year, while royalty and manufacturing revenue stood at $61.4 million, up from $52.5 million.

Translarna™ net product revenue increased to $72.3 million from $69.0 million, whereas Emflaza® revenue dropped to $51.9 million from $67.4 million in the same period of 2023. Notably, Roche reported Evrysdi® year-to-date sales of approximately CHF 1.2 billion, resulting in royalty revenue of $61.4 million to PTC for the third quarter, an increase from $50.2 million in 2023.

On the expense side, GAAP R&D expenses were $161.4 million, slightly down from $164.2 million in the third quarter of 2023, reflecting a strategic focus on high-potential R&D programs. Non-GAAP R&D expenses, excluding stock-based compensation, were $152.0 million, compared to $150.2 million in the previous year.

GAAP SG&A expenses decreased to $73.5 million from $80.9 million, mainly due to reduced workforce costs. Non-GAAP SG&A expenses, excluding stock-based compensation, were $63.1 million, down from $67.9 million in 2023.

The net loss for the third quarter of 2024 was $106.7 million, an improvement from a net loss of $133.0 million in the prior year. As of September 30, 2024, cash, cash equivalents, and marketable securities totaled $1,013.4 million, up from $876.7 million on December 31, 2023. The company's shares issued and outstanding were 76,952,124 as of September 30, 2024.

PTC also updated its full-year 2024 financial guidance, projecting total revenues between $750 million and $800 million. GAAP R&D and SG&A expenses are expected to range from $740 million to $835 million, including anticipated R&D milestone payments of up to $65 million. Non-GAAP R&D and SG&A expenses are forecasted between $660 million and $755 million, excluding stock-based compensation expenses. The company anticipates up to $90 million in payments for potential regulatory milestones in 2024, with $65 million recorded as R&D expenses and $20 million as contingent consideration.

Overall, PTC Therapeutics continues to make significant strides in its revenue performance and strategic objectives, driven by strong clinical and regulatory progress and a solid financial foundation.

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