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PTC Therapeutics Provides a Corporate Update

May 4, 2021

PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced a corporate update and financial results for the first quarter ending March 31, 2021.

“Overall PTC has had a strong performance this quarter through all aspects of the company from discovery to commercial revenue,” said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. “I would like to highlight the continued strong growth of the DMD franchise which has had one of our largest quarterly revenues to date. The other key milestone was the positive preliminary results in our PTC518 Huntington’s disease program demonstrating dose dependent lowering of the HTT mRNA. Analogous to the SMA program we are now well positioned with a clear path for success.”

Key First Quarter and Other Corporate Updates:

  • The Duchenne muscular dystrophy (DMD) franchise had a total net product revenue of $90 million for Translarna™ (ataluren) and Emflaza® (deflazacort) in the first quarter of 2021. This represents 32% growth over the first quarter of 2020 and one of PTC’s strongest quarterly commercial revenues to date.
    • Broader uptake due to new patients in existing geographies and geographic expansion drove Translarna growth.
    • Emflaza revenue growth was primarily due to increased new prescriptions, high compliance, and fewer discontinuations.
  • In March 2021, the European Medicines Agency (EMA) approved Evrysdi™ (risdiplam) in the European Union (EU). The first sale of Evrysdi in this region was recorded the following day, triggering a $20 million milestone payment to PTC. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
  • Preliminary results from the PTC518 Phase 1 healthy volunteer trial demonstrated dose-dependent reduction of Huntington mRNA beyond the 30-50% target.
  • PTC received Gallup’s Don Clifton Strengths-Based Culture Award, which reflects the Company’s ongoing deep commitment to its employees.

First Quarter Clinical Updates:

  • PTC has multiple clinical trials ongoing, three of which are registration-directed clinical studies:
    • The MIT-E Phase 2/3 trial with vatiquinone for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
    • The MOVE-FA Phase 3 trial with vatiquinone for Friedreich ataxia with data anticipated in 2023.
    • The FITE19 Phase 2/3 clinical trial for PTC299 in patients with COVID-19 with an expected data readout in the second half of 2021.
  • The second Bio-e compound, PTC857 healthy volunteer study was recently completed, and data will be communicated in the second quarter.
  • The registration-directed Phase 3 PTC923 phenylketonuria (PKU) trial, APHENITY, is expected to initiate in mid-2021.
  • The Committee for Medicinal Products for Human Use (CHMP) has requested a clock stop in the aromatic L-amino acid decarboxylase (AADC) deficiency review process to allow for completion of its pre-approval inspections, which were delayed due to COVID-19. The CHMP opinion is now anticipated in the third quarter of 2021.
  • Due to COVID-related surgical delays, the AADC-deficiency biologics license application submission to the U.S. Food and Drug Administration is anticipated to be delayed by at least one quarter.

First Quarter 2021 Financial Highlights:

  • Total revenues were $117.9 million for the first quarter of 2021, compared to total revenues of $68.3 million for the first quarter of 2020, a 32% increase. Total revenue includes net product revenue of $91.3 million and collaboration and royalty revenue of $26.7 million in the first quarter of 2021.
  • Translarna net product revenues were $46.5 million for the first quarter of 2021, compared to $40.5 million for the first quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion into new territories.
  • Emflaza net product revenues were $43.5 million for the first quarter of 2021, compared to $27.5 million for the first quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
  • Roche reported Evrysdi first quarter 2021 sales of approximately CHF 80 million. During the first quarter of 2021, the first commercial sale of Evrysdi in the EU triggered a $20 million milestone payment to PTC, which was reported as collaboration revenue.
  • U.S. GAAP (generally accepted accounting principles) R&D expenses were $134.5 million for the first quarter of 2021, compared to $90.1 million for the first quarter of 2020. The increase in R&D expenses reflects costs associated with increased investment in research programs, and advancement of the clinical pipeline.
  • Non-GAAP R&D expenses were $120.8 million for the first quarter of 2021, excluding $13.7 million in non-cash, stock-based compensation expense, compared to $81.9 million for the first quarter of 2020, excluding $8.2 million in non-cash, stock-based compensation expense.
  • GAAP SG&A expenses were $61.1 million for the first quarter of 2021, compared to $58.2 million for the first quarter of 2020. The increase in SG&A expenses was associated with entering into a long-term lease for the Hopewell facility that commenced on July 1, 2020.
  • Non-GAAP SG&A expenses were $49.1 million for the first quarter of 2021, excluding $12.0 million in non-cash, stock-based compensation expense, compared to $51.2 million for the first quarter of 2020, excluding $7.0 million in non-cash, stock-based compensation expense.
  • Change in the fair value of deferred and contingent consideration was $0.1 million for the first quarter of 2021, compared to $0.9 million for the first quarter of 2020. 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 $128.6 million for the first quarter of 2021, compared to net loss of $112.7 million for the first quarter of 2020.
  • Cash, cash equivalents and marketable securities was $988.4 million at March 31, 2021, compared to $1.1 billion at December 31, 2020.
  • Shares issued and outstanding as of March 31, 2021 were 70,405,905.

PTC Reaffirms Full Year 2021 Guidance as Follows:

  • PTC anticipates net product revenues for the DMD franchise for the full year 2021 to be between $355 and $375 million.
  • PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
  • PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 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.