On January 9, 2023 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported an update on its commercial progress and R&D pipeline at the 41st Annual J.P. Morgan Healthcare Conference today, Monday January 9, at 10:30am EST/7:30am PST. Matthew Klein, M.D., Chief Operating Officer of PTC Therapeutics, will provide an update on 2022 accomplishments and highlight upcoming 2023 potential value-creating milestones. Preliminary 2022 unaudited financial results and 2023 financial guidance will also be provided (Press release, PTC Therapeutics, JAN 9, 2023, View Source [SID1234626096]). 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 2022 Corporate Highlights:
Upstaza, the first direct-administered gene therapy into the brain, was approved in the EU and UK for AADC deficiency patients.
Unaudited net product revenue of $535 million in 2022 representing 25% year-over-year growth.
Strong year-over-year growth for the Duchenne muscular dystrophy (DMD) franchise, with unaudited net product revenue of $289 million for Translarna (ataluren) and $218 million for Emflaza (deflazacort) in 2022.
Translarna growth was driven by new patients in existing geographies and continued geographic expansion.
Emflaza growth was due to continued new prescriptions, high compliance, fewer patient discontinuations and more favorable access.
Upstaza unaudited net product revenue was $13 million driven by patients being treated through early access programs and commercial access.
Evrysdi (risdiplam) is now approved in more than 90 countries. It has established market leadership in all major markets and is on track to become the global market leader in treatment of spinal muscular atrophy (SMA). Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche.
PTC successfully advanced its clinical pipeline in 2022:
CardinALS, a registration-directed Phase 2 clinical trial of PTC857 in amyotrophic lateral sclerosis, was initiated.
PIVOT-HD, a Phase 2 clinical trial of PTC518 in Huntington’s disease, was initiated.
SunriseLMS, a registration-directed Phase 2 trial of unesbulin in leiomyosarcoma, was initiated.
The placebo-controlled portion of Study 041, a Phase 3 clinical trial of Translarna for nmDMD, was completed. Study 041 results supported submission of a Type II variation for conversion to standard marketing authorization in the EU.
FITE-19, a Phase 2/3 clinical trial of emvododstat for COVID-19, was completed.
2023 Potential Key Value-Creating Milestones:
Results from the placebo-controlled portion of APHENITY, the Phase 3 registration-directed clinical trial of sepiapterin in patients with PKU, are expected in the first quarter of 2023.
Results from MIT-E, the Phase 2/3 registration-directed clinical trial of vatiquinone in mitochondrial disease associated seizures, are expected in the first quarter of 2023.
Results from MOVE-FA, the Phase 3 registration-directed clinical trial of vatiquinone in Friedreich ataxia, are expected in the second quarter of 2023.
Results from the 12-week portion of PIVOT-HD, the Phase 2 study of PTC518 in Huntington’s disease, are expected in the second quarter of 2023.
Submission of a Biologics License Application (BLA) to the FDA for Upstaza is expected in the first half of 2023.
A Phase 2/3 clinical trial of unesbulin in diffuse intrinsic pontine glioblastoma is expected to initiate in the fourth quarter of 2023.
Preliminary Unaudited 2022 Financial Results:
Total unaudited net revenue for full year 2022 was approximately $710 million.
Total unaudited net product revenue for full year 2022 was approximately $535 million.
DMD franchise unaudited revenue for full year 2022 was approximately $507 million, including net product revenue for Translarna of approximately $289 million and for Emflaza of approximately $218 million.
PTC expects to report approximately $175 million in 2022 collaboration and royalty revenue associated with Evrysdi.
PTC is currently in the process of finalizing its financial results for the 2022 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2022, subject to the completion of PTC’s financial closing procedures. Evrysdi royalty revenue estimates are based on sell side analyst consensus estimates.
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 and $565 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2023 to be between $1.01 and $1.06 billion.
PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2023 to be between $890 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.
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.
PTC Therapeutics, Inc.
Reconciliation of GAAP to Non-GAAP Projected Full Year 2023 R&D and SG&A Expense (In thousands)
Low End of Range
High End of Range
Projected GAAP R&D and SG&A Expense
$ 1,010,000
$ 1,060,000
Less: projected non-cash, stock-based compensation expense
120,000
120,000
Projected non-GAAP R&D and SG&A expense
$ 890,000
$ 940,000