On January 10, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported tthat it 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 (Press release, PTC Therapeutics, JAN 10, 2022, View Source [SID1234598504]). 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.

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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.
Translarna growth was driven by new patients and high compliance in existing geographies and continued geographic expansion.
Emflaza growth was due to continued new prescriptions, high compliance, less patient discontinuations and more favorable access.
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:
APHENITY, a Phase 3 registration-directed trial of PTC923 in phenylketonuria (PKU) was initiated.
Phase 1 healthy volunteer trials of PTC518 and PTC857 were completed.
Advanced the oncology platform with the completion of Phase 1b studies of unesbulin in leiomyosarcoma and diffuse intrinsic pontine glioma (DIPG).
Completed enrollment for the vatiquinone MOVE-FA registration-directed trial with results expected in the second quarter of 2023.
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:
PTC expects an opinion from the European Medicine Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for PTC-AADC, the first gene therapy for AADC deficiency, in April 2022.
Submission of a Biologics License Application (BLA) to the FDA for PTC-AADC is expected in the second quarter of 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.
Non-GAAP Financial Measures:

In this press release, the financial results and financial guidance of PTC are provided in accordance with GAAP and using certain non-GAAP financial measures. In particular, the non-GAAP financial measures exclude non-cash, stock-based compensation expense. These non-GAAP financial measures are provided as a complement to financial measures reported in GAAP because management uses these non-GAAP financial measures when assessing and identifying operational trends. In management’s opinion, these non-GAAP financial measures are useful to investors and other users of PTC’s financial statements by providing greater transparency into the historical and projected operating performance of PTC and the company’s future outlook. Non-GAAP financial measures are not an alternative for financial measures prepared in accordance with GAAP. Quantitative reconciliations of the non-GAAP financial measures to their respective closest equivalent GAAP financial measures are included in the table below.