PTC Therapeutics Provides a Corporate Update

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2023.

“We are pleased to report strong revenue growth in the first quarter, with double-digit year-over-year revenue increases for all six of our marketed and partnered products,” said Matthew Klein, M.D., Chief Executive Officer, PTC Therapeutics, Inc. “In addition, we remain on track to share readouts from four clinical studies, including three registration-directed studies, in the second quarter.”

Key Corporate Updates:

  • First quarter 2023 revenue for the Duchenne muscular dystrophy (DMD) franchise was $170 million, representing 33% year-over-year growth
    • Translarna™ (ataluren) quarterly net product revenue was $115 million, with growth coming from treatment of new patients, continued high compliance and continued geographic expansion
    • Emflaza® (deflazacort) quarterly net product revenue was $55 million, driven by new patients, high compliance and broader access

Key Clinical and Regulatory Updates:

  • PTC continues to make progress in ongoing registration-directed clinical studies:
    • The APHENITY Phase 3 trial of sepiapterin (PTC923) for PKU, with results anticipated in May 2023
    • The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures, with results anticipated in the second quarter of 2023
    • The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia, with results anticipated in the second quarter of 2023
  • Enrollment in the PIVOT-HD Phase 2 trial of PTC518 for Huntington’s disease is active and ongoing at study sites in Europe and Australia. Interim data from the first 12 weeks of the placebo-controlled trial is anticipated in the second quarter of 2023.
  • Given the anticipated imminent results from these studies, PTC will not be discussing these programs from April 28 until results are disclosed for each study.
  • For Upstaza, the FDA requested additional bioanalytical data in support of comparability analyses between the clinical and commercial drug product. We have received initial feedback from the FDA on these data and are in the process of responding to additional queries prior to submitting the BLA, which could result in a BLA submission occurring in the third quarter of 2023.
  • PTC submitted a Type II variation to EMA to support the conversion of the conditional marketing authorization for Translarna to a standard marketing authorization. PTC expects a CHMP opinion in the second quarter of 2023.

First Quarter 2023 Financial Highlights:

  • Total revenues were $220.4 million for the first quarter of 2023, compared to $148.7 million for the first quarter of 2022.
  • Total revenue includes net product revenue across the commercial portfolio of $187.6 million for the first quarter of 2023, compared to $129.8 million for the first quarter of 2022. Total revenue also includes collaboration, royalty and manufacturing revenue of $32.8 million in the first quarter of 2023, compared to $18.9 million for the first quarter of 2022.
  • Translarna net product revenues were $115.1 million for the first quarter of 2023, compared to $79.2 million for the first quarter of 2022. These results were driven by treatment of new patients, continued high compliance and geographic expansion.
  • Emflaza net product revenues were $54.6 million for the first quarter of 2023, compared to $48.6 million for the first quarter of 2022. These results reflect new patients, high compliance, and broader access.
  • Roche reported Evrysdi 2023 year to date sales of approximately CHF 363 million, resulting in royalty revenue of $30.8 million to PTC for the first quarter of 2023, as compared to $18.9 million for the first quarter of 2022.
  • Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $195.1 million for the first quarter of 2023, compared to $140.1 million for the first quarter of 2022. The increase reflects additional investment in research programs and advancement of the clinical pipeline. The increase in R&D expenses includes the achievement of a $30.0 million success-based development milestone for the completion of enrollment of a Phase 3 clinical trial for sepiapterin for PKU.
  • Non-GAAP R&D expenses were $179.8 million for the first quarter of 2023, excluding $15.3 million in non-cash, stock-based compensation expense, compared to $127.0 million for the first quarter of 2022, excluding $13.0 million in non-cash, stock-based compensation expense.
  • GAAP SG&A expenses were $86.9 million for the first quarter of 2023, compared to $73.3 million for the first quarter of 2022. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
  • Non-GAAP SG&A expenses were $73.4 million for the first quarter of 2023, excluding $13.5 million in non-cash, stock-based compensation expense, compared to $59.7 million for the first quarter of 2022, excluding $13.6 million in non-cash, stock-based compensation expense.
  • Change in the fair value of deferred and contingent consideration was a loss of $2.4 million for the first quarter of 2023, compared to a gain of $11.7 million for the first quarter of 2022. The change in fair value of deferred and contingent consideration is related to the fair valuation of potential future consideration to be paid to former equity holders of Agilis Biotherapeutics, Inc. (Agilis) in connection with PTC’s acquisition of Agilis, which closed in August 2018.
  • Net loss was $139.0 million for the first quarter of 2023, compared to net loss of $126.7 million for the first quarter of 2022.
  • Cash, cash equivalents, and marketable securities was $286.3 million at March 31, 2023, compared to $410.7 million at December 31, 2022.
  • Shares issued and outstanding as of March 31, 2023, were 74,012,034.

PTC Reaffirms Full Year 2023 Revenue Guidance as Follows:

  • PTC anticipates total revenues for the full year 2023 to be between $940 million and $1.0 billion.
  • PTC anticipates net product revenues for the DMD franchise for the full year 2023 to be between $545 and $565 million.

About PTC Therapeutics, Inc.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC’s ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need. The company’s strategy is to leverage its strong scientific expertise and global commercial infrastructure to maximize value for its patients and other stakeholders. To learn more about PTC, please visit us at www.ptcbio.com and follow us on Facebook, on Twitter at @PTCBio, and on LinkedIn.