On August 4, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the second quarter ending June 30, 2022 (Press release, PTC Therapeutics, AUG 4, 2022, View Source [SID1234617534]).

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"We made significant progress in the second quarter toward achieving our ambitious goals for 2022. We continued to generate strong revenue growth, to expand our product portfolio, and to advance our pipeline," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "PTC has a broad and deep pipeline across a range of diseases, and we eagerly anticipate important results for several registration-directed studies during the next six to twelve months and this will continue our transformational growth."

Key Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise continued to show strong growth, with second quarter total net product revenue of $134 million, or 32% year-over-year growth.
Translarna total net product revenue increased 46% year-over-year to $77 million, with growth coming from new patients in existing geographies and continued geographic expansion.
Emflaza total net product revenue grew 16% year-over-year to $57 million, driven by new patient starts, broader access, continued high compliance and appropriate weight-based dosing.
The FDA approved a label extension for Evrysdi (risdiplam) to include infants under 2 months old with SMA. Evrysdi is a product of the SMA collaboration among PTC, the SMA Foundation and Roche.
In July 2022, Upstaza (eladocagene exuparvovec), our gene therapy for AADC deficiency, received marketing authorization by the European Commission.
First group purchase order for Tegsedi in Brazil was fulfilled.
Key Clinical/Regulatory Updates:

PTC recently announced positive topline results of the placebo-controlled portion of Study 041 for Translarna. The Study 041 results add to the totality of evidence demonstrating the clinical benefit of Translarna.
PTC will update topline results of FITE-19 study of emvododstat for COVID-19 on second quarter call.
PTC continues to make progress in additional ongoing clinical studies:
The APHENITY Phase 3 trial of PTC923 for PKU, with results anticipated by the end of 2022.
The PIVOT-HD Phase 2 trial of PTC518 for Huntington’s disease, with data from the first 12 weeks of the placebo-controlled trial anticipated by the end of 2022.
The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures, with results anticipated in the first quarter of 2023.
The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia, with results anticipated in the second quarter of 2023.
Second Quarter 2022 Financial Highlights:

Total revenues were $165.5 million for the second quarter of 2022, compared to $116.7 million for the second quarter of 2021.
Total revenue includes net product revenue across the commercial portfolio of $143.7 million for the second quarter of 2022, compared to $103.1 million for the second quarter of 2021. Total revenue also includes royalty revenue of $21.8 million in the second quarter of 2022, compared to $13.6 million for the second quarter of 2021.
Translarna net product revenues were $77.0 million for the second quarter of 2022, compared to $52.6 million for the second quarter of 2021. These results reflect an increase in net product sales coming from new patients in existing geographies and continued geographic expansion.
Emflaza net product revenues were $56.8 million for the second quarter of 2022, compared to $49.1 million for the second quarter of 2021. These results reflect new patient starts, broader access, continued high compliance, and appropriate weight-based dosing.
Roche reported year to date 2022 Evrysdi sales of approximately CHF 500 million, resulting in royalty revenue of $21.8 million to PTC in the second quarter of 2022, as compared to $13.6 million for the second quarter of 2021.
Based on U.S. GAAP (Generally Accepted Accounting Principles), GAAP R&D expenses were $157.3 million for the second quarter of 2022, compared to $125.5 million for the second quarter of 2021. The increase reflects increased investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $143.5 million for the second quarter of 2022, excluding $13.8 million in non-cash, stock-based compensation expense, compared to $112.0 million for the second quarter of 2021, excluding $13.4 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $79.9 million for the second quarter of 2022, compared to $68.9 million for the second quarter of 2021. The increase reflects our continued investment to support commercial activities including our expanding commercial portfolio.
Non-GAAP SG&A expenses were $66.0 million for the second quarter of 2022, excluding $13.9 million in non-cash, stock-based compensation expense, compared to $56.6 million for the second quarter of 2021, excluding $12.3 million in non-cash, stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was a $15.2 million gain for the second quarter of 2022, compared to a $0.7 million loss for the second quarter of 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 $152.1 million for the second quarter of 2022, compared to net loss of $118.4 million for the second quarter of 2021.
Cash, cash equivalents, and marketable securities was $505.5 million on June 30, 2022, compared to $773.4 million at December 31, 2021.
Shares issued and outstanding as of June 30, 2022 were 71,505,889.
PTC Reaffirms Full Year 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 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.

Today’s Conference Call and Webcast Reminder:

To access the call by phone, please click here to register and you will be provided with dial-in details. To avoid delays, we recommend participants dial in to the conference call 15 minutes prior to the start of the call. The webcast conference call can be accessed on the Investor section of the PTC website at View Source A replay of the call will be available approximately two hours after completion of the call and will be archived on the company’s website for 30 days following the call.