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PTC Therapeutics Provides a Corporate Update

October 27, 2022

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the third quarter ending September 30, 2022.

“Our mission is to build an enduring biopharmaceutical company that treats diseases with significant unmet medical needs,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “We continue to achieve our ambitious goals for 2022 of generating strong revenue growth while advancing our broad and deep pipeline to continue to fulfill this vision.”

Key Corporate Updates:

  • The Duchenne muscular dystrophy (DMD) franchise continued to show strong growth, with third quarter total net product revenue of $131 million, or 15% year-over-year growth.
    • Translarna™ (ataluren) total net product revenue was $77 million, with growth coming from new patients in existing geographies and continued geographic expansion
    • Emflaza® (deflazacort) total net product revenue was $55 million, driven by new patient starts, broader access, continued high compliance and appropriate weight-based dosing
  • Evrysdi milestone of $50 million from Roche achieved for surpassing annual net sales of $750 million.
  • Strategic financing with Blackstone to grow pipeline. As part of the partnership, Blackstone provides PTC with an initial $350 million in capital at close, with an option for an additional $650 million in funding.

Key Clinical and Regulatory Updates:

  • PTC submitted a type II variation to EMA to convert the conditional marketing authorization for Translarna to a standard marketing authorization. PTC expects a CHMP opinion in the first half of 2023.
  • PTC submitted a meeting request to the FDA to gain clarity on the regulatory pathway for the NDA for Translarna in the US. While the FDA has provided initial written feedback that Study 041 does not provide substantial evidence of effectiveness, PTC is planning follow up discussions with the agency to understand whether the evidence in the ITT population in Study 041 along with confirmatory evidence from other studies could support approval.
  • PTC held a type C meeting with the FDA to discuss the details of a potential submission package for Upstaza. The FDA asked for additional bioanalytical data in support of comparability between the drug product used in the clinical studies and the commercial drug product. PTC is currently working with the FDA to address this request and expects to submit a BLA for Upstaza in the first half of 2023.
  • PTC continues to make progress in additional ongoing registration-directed clinical studies:
    • The APHENITY Phase 3 trial of sepiapterin (PTC923) for PKU, with results anticipated by the end of the fourth quarter
    • The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures, with results anticipated in the first 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. Enrollment in the US is paused as the FDA has requested additional data to support trial conduct. Data from the first 12 weeks of the placebo-controlled trial anticipated in the first half of 2023.

Third Quarter 2022 Financial Highlights:

  • Total revenues were $217.1 million for the third quarter of 2022, compared to $138.7 million for the third quarter of 2021.
  • Total revenue includes net product revenue across the commercial portfolio of $134.2 million for the third quarter of 2022, compared to $115.6 million for the third quarter of 2021. Total revenue also includes royalty and collaboration revenue of $82.9 million in the third quarter of 2022, compared to $23.1 million for the third quarter of 2021.
  • Translarna net product revenues were $76.6 million for the third quarter of 2022, compared to $67.2 million for the third quarter of 2021. These results reflect an increase in net product sales from new patients in existing markets as well as continued geographic expansion.
  • Emflaza net product revenues were $54.8 million for the third quarter of 2022, compared to $47.1 million for the third quarter of 2021. These results reflect new patient starts, broader access, continued high compliance and appropriate weight-based dosing.
  • Roche reported Evrysdi 2022 year to date sales of approximately CHF 793 million, resulting in royalty revenue of $32.9 million to PTC in the third quarter of 2022, as compared to $13.1 million for the third quarter of 2021. Also in the third quarter of 2022, PTC recorded a sales milestone of $50 million for the achievement of $750 million in worldwide annual net sales from Evrysdi. This sales milestone was recorded as collaboration revenue.
  • Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $165.5 million for the third quarter of 2022, compared to $130.8 million for the third quarter of 2021. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
  • Non-GAAP R&D expenses were $150.4 million for the third quarter of 2022, excluding $15.1 million in non-cash, stock-based compensation expense, compared to $117.8 million for the third quarter of 2021, excluding $13.0 million in non-cash, stock-based compensation expense.
  • GAAP SG&A expenses were $80.1 million for the third quarter of 2022, compared to $69.3 million for the third quarter of 2021. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
  • Non-GAAP SG&A expenses were $66.5 million for the third quarter of 2022, excluding $13.6 million in non-cash, stock-based compensation expense, compared to $56.4 million for the third quarter of 2021, excluding $12.8 million in non-cash, stock-based compensation expense.
  • Change in the fair value of deferred and contingent consideration was $5.3 million for the third quarter of 2022, compared to $10.8 million for the third quarter of 2021. 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 $109.3 million for the third quarter of 2022, compared to net loss of $133.6 million for the third quarter of 2021.
  • Cash, cash equivalents, and marketable securities was $288.4 million as of September 30, 2022, compared to $773.4 million as of December 31, 2021.
  • Shares issued and outstanding as of September 30, 2022, were 71,854,892.

PTC Updates Full Year 2022 Financial Guidance as Follows:

  • PTC anticipates total revenues for the full year 2022 to be between $710 and $750 million, compared to previous guidance of between $700 and $750 million.
  • PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between $490 and $500 million, compared to previous guidance of between $475 and $495 million.
  • PTC anticipates GAAP R&D and SG&A expenses for the full year 2022 to be between $925 and $965 million, compared to previous guidance of between $915 and $965 million.
  • PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between $810 and $850 million, compared to previous guidance of between $800 and $850 million, both excluding estimated non-cash, stock-based compensation expense of $115 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.