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PTC Therapeutics Provides a Corporate Update

February 21, 2023

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the fourth quarter and full year ending December 31, 2022.

“2023 will be a very exciting year at PTC, including a celebration of our 25th anniversary,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “Together our marketed products grew 30% in revenue in 2022 despite significant foreign exchange headwinds. We expect our revenue growth to accelerate further in 2023, potentially reaching $1 billion in total revenue. We are also advancing a broad and deep pipeline of new therapies that we expect to provide substantial growth in the coming years.”

Key Corporate Updates:

  • 2022 total revenue of $699 million, representing 30% year-over-year growth, or $740 million when calculated at CER, representing a 37% growth
  • 2022 revenue for the Duchenne muscular dystrophy (DMD) franchise was $507 million, representing 20% year-over-year growth, or $539 million when calculated at CER, representing a 27% growth. The DMD franchise fourth quarter total net product revenue was $114 million
    • Translarna™ (ataluren) total net product revenue was $56 million, with growth coming from new patients in existing geographies and continued geographic expansion
    • Emflaza® (deflazacort) total net product revenue was $58 million, driven by new patient starts, broader access, continued high compliance, and appropriate weight-based dosing

Key Clinical and Regulatory Updates:

  • 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 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. Data from the first 12 weeks of the placebo-controlled trial is anticipated in the second quarter of 2023.

Fourth-Quarter and Full-Year 2022 Financial Highlights:

  • Total revenues were $167.4 million for the fourth quarter of 2022, compared to $165.2 million for the fourth quarter of 2021. Total revenues were $698.8 million for the full year 2022, compared to $538.6 million for the full year 2021.
  • Total revenue includes net product revenue across the commercial portfolio of $127.5 million for the fourth quarter of 2022 and $535.2 million for the full year 2022, compared to $118.9 million for the fourth quarter of 2021 and $428.9 million for the full year 2021. Total revenue also includes collaboration and royalty revenue of $39.9 million in the fourth quarter of 2022 and $163.6 million for the full year 2022, compared to $46.3 million for the fourth quarter of 2021 and $109.7 million for the full year 2021.
  • Translarna net product revenues were $55.8 million for the fourth quarter of 2022, compared to $69.7 million for the fourth quarter of 2021. Translarna net product revenues were $288.6 million for the full year 2022, compared to $236.0 million for the full year 2021. These results were driven by treatment of new patients, continued high compliance, and geographic expansion.
  • Emflaza net product revenues were $58.1 million for the fourth quarter of 2022, compared to $47.5 million for the fourth quarter of 2021. Emflaza net product revenues were $218.3 million for the full year 2022, compared to $187.3 million for the full year 2021. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
  • Roche reported Evrysdi 2022 year to date sales of approximately CHF 1,119 million, resulting in full year 2022 royalty revenue of $113.5 million to PTC, as compared to $54.6 million for full year 2021.
  • Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $188.7 million for the fourth quarter of 2022, compared to $149.8 million for the fourth quarter of 2021. GAAP R&D expenses were $651.5 million for the full year 2022, compared to $540.7 million for the full year 2021. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
  • Non-GAAP R&D expenses were $174.7 million for the fourth quarter of 2022, excluding $14.0 million in non-cash, stock-based compensation expense, compared to $136.4 million for the fourth quarter of 2021, excluding $13.4 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $595.6 million for the full year 2022, excluding $55.9 million in non-cash, stock-based compensation expense, compared to $487.1 million for the full year 2021, excluding $53.6 million in non-cash, stock-based compensation expense.
  • GAAP SG&A expenses were $92.7 million for the fourth quarter of 2022, compared to $86.5 million for the fourth quarter of 2021. GAAP SG&A expenses were $326.0 million for the full year 2022, compared to $285.8 million for the full year 2021. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
  • Non-GAAP SG&A expenses were $79.3 million for the fourth quarter of 2022, excluding $13.4 million in non-cash, stock-based compensation expense, compared to $73.7 million for the fourth quarter of 2021, excluding $12.8 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $271.5 million for the full year 2022, excluding $54.5 million in non-cash, stock-based compensation expense, compared to $235.9 million for the full year 2021, excluding $49.9 million in non-cash, stock-based compensation expense.
  • Intangible asset impairment was $33.4 million for the fourth quarter and full year 2022, which represents a non-cash charge. The partial impairment was related to a decrease in projected cash flows due to refinements in current market assumptions and the timing of patient treatments.
  • Change in the fair value of deferred and contingent consideration was a loss of $6.3 million for the fourth quarter of 2022, compared to a gain of $12.1 million for the fourth quarter of 2021. Change in the fair value of deferred and contingent consideration was a gain of $25.9 million for the full year 2022, compared to a gain of $0.5 million for the full year 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 $170.9 million for the fourth quarter of 2022, compared to net loss of $143.3 million for the fourth quarter of 2021. Net loss was $559.0 million for the full year 2022, compared to net loss of $523.9 million for the full year 2021.
  • Cash, cash equivalents, and marketable securities was $410.7 million at December 31, 2022, compared to $773.4 million at December 31, 2021.
  • Shares issued and outstanding as of December 31, 2022, were 73,104,692.

PTC Reaffirms Full Year 2023 Financial Guidance:

  • 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 million and $565 million.
  • PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between $1.01 billion and $1.06 billion.
  • PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2023 to be between $890 million and $940 million, excluding estimated non-cash, stock-based compensation expense of $120 million. PTC anticipates up to $80 million of one-time payments upon achievement of potential clinical and regulatory success-based milestones from previous acquisitions.

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.