- $536 million unaudited 2021 total revenue representing impressive 41% year-over-year growth -
- $700 - 750 million 2022 total revenue guidance -
- Results are expected in four registration-directed trials this year -
- Three additional registration-directed clinical trials expected to initiate in 2022
PTC Therapeutics, Inc. (NASDAQ: PTCT) will present an update on its commercial progress and R&D pipeline at the 40th Annual J.P. Morgan Healthcare Conference today, Monday Jan. 10th at 7:30am EST. Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2021 accomplishments and highlight upcoming potential value-creating milestones. Preliminary 2021 unaudited financial results and 2022 financial guidance will also be provided. The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.
Key 2021 Corporate Highlights:
Unaudited net product revenue of $429 million in 2021 representing 29% year-over-year growth.
Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of $424 million for Translarna™ (ataluren) and Emflaza® (deflazacort) in 2021. Cumulative net product revenue for Translarna exceeds $1 billion and Emflaza exceeds $500 million, since respective launches.
Evrysdi® (risdiplam) has shown continued rapid uptake in the United States and is now approved in all major markets including the European Union and Japan. Evrysdi is a product of the Spinal Muscular Atrophy (SMA) collaboration between PTC, the SMA Foundation and Roche.
Waylivra® (volanesorsen), the only treatment for familial chylomicronemia syndrome was approved by Brazilian Health Regulatory Agency, Agência Nacional de Vigilância Sanitária (ANVISA), and both Waylivra and Tegsedi® (inotersen) received Category 1 classification from Câmara de Regulação do Mercado de Medicamentos - CMED (Drug Market Regulation Chamber) in Brazil. Category 1 classification is given to innovative treatments that provide greater efficacy than the standard of care and allows for pricing in line with international markets.
PTC successfully advanced its clinical pipeline in 2021:
2022 Potential Key Value-Creating Milestones:
Results from Study 041 for Translarna are expected mid-year 2022 and if positive could support
re-submission of a New Drug Application (NDA) to the Food and Drug Administration (FDA).Results for MIT-E, the registration-directed study of vatiquinone in mitochondrial disease associated seizures, are expected in the fourth quarter of 2022.
Results are expected by year end 2022 for the Phase 3 registration-directed study, APHENITY, for PTC923 in patients with PKU.
From the splicing platform, the PIVOT-HD Phase 2 study of PTC518 in Huntington's disease patients is planned to initiate in the first quarter of 2022.
From the Bio-e platform, the registration-directed CardinALS study of PTC857 in amyotrophic lateral sclerosis (ALS) patients is expected to be initiated in the second quarter of 2022.
Progress in the gene therapy platform is anticipated in 2022:
Preliminary Unaudited 2021 Financial Results:
Total unaudited net revenue for full year 2021 was approximately $536 million.
Total unaudited net product revenue for full year 2021 was approximately $429 million.
DMD franchise revenue for year end 2021 included net product revenue for Translarna of approximately $236 million, with $70 million in revenue in the fourth quarter, and Emflaza of approximately $188 million, with $48 million in revenue in the fourth quarter.
PTC expects to report approximately $107 million in 2021 collaboration and royalty revenue associated with Evrysdi.
PTC expects to report 2021 year-end cash, cash equivalents and marketable securities of approximately $773 million.
PTC is currently in the process of finalizing its financial results for the 2021 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2021, subject to the completion of PTC's financial closing procedures.
2022 Financial Guidance:
PTC anticipates total revenues for the full year 2022 to be between $700 and $750 million.
PTC anticipates net product revenues for the DMD franchise for the full year 2022 to be between $475 and $495 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2022 to be between $915 and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2022 to be between $800 and $850 million, excluding estimated non-cash, stock-based compensation expense of $115 million.
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