On May 3, 2022 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the first quarter ending March 31, 2022 (Press release, PTC Therapeutics, MAY 3, 2022, View Source [SID1234613404]).
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"We are continuing to build a robust pipeline of potential new therapeutics that at steady state we anticipate delivering a new product every 2-3 years," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "With multiple ongoing registration-directed trials, we are well on the way to fulfill this vision."
Key First Quarter Corporate Updates:
The Duchenne muscular dystrophy (DMD) franchise continued to show strong growth, with total net product revenue of $128 million for Translarna (ataluren) and Emflaza (deflazacort) in the first quarter of 2022.
Translarna total net product revenue of $79 million, with growth coming from new patients in existing markets and continued geographic expansion as PTC drives its robust and globally diversified business.
Emflaza total net product revenue of $49 million, with growth driven by continued increases in new patients and high compliance and appropriate weight-based dosing.
Evrysdi (risdiplam) net sales in the first quarter of 2022 resulted in $19 million in royalty revenue to PTC. Evrysdi is a product of the SMA collaboration among PTC, the SMA Foundation and Roche.
First Quarter Clinical Updates:
The PIVOT-HD Phase 2 study of PTC518 for the treatment of Huntington’s disease was initiated in the first quarter of 2022.
In April, Scientific Advisory Group and Oral Explanation meetings with the CHMP were successfully completed. The CHMP opinion on the PTC-AADC marketing authorization application is now expected in May 2022.
PTC expects to submit the BLA for PTC-AADC in AADC deficiency to the FDA in the third quarter of 2022.
PTC anticipates reporting results of Study 041 for ataluren by the end of the second quarter of 2022, after data analysis is completed.
PTC continues to make progress in three additional ongoing registration-directed clinical studies:
The MIT-E Phase 2/3 vatiquinone trial for mitochondrial disease associated seizures, with results anticipated in the fourth quarter of 2022.
The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia, with results anticipated in the second quarter of 2023.
The APHENITY Phase 3 trial of PTC923 for PKU, with results anticipated by the end of this year.
Two additional registration-directed trials were initiated:
The CardinALS Phase 2 trial of PTC857 for the treatment of ALS.
The SUNRISELMS Phase 2 trial of unesbulin for the treatment of leiomyosarcoma.
First Quarter 2022 Financial Highlights:
Total revenues were $148.7 million for the first quarter of 2022, compared to $117.9 million for the first quarter of 2021.
Total revenues included net product revenue across the commercial portfolio of $129.8 million for the first quarter of 2022, compared to $91.3 million for the first quarter of 2021. Total revenues also included collaboration and royalty revenue of $18.9 million in the first quarter of 2022, compared to $26.7 million for the first quarter of 2021.
Translarna net product revenues were $79.2 million for the first quarter of 2022, compared to $46.5 million for the first quarter of 2021. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion.
Emflaza net product revenues were $48.6 million for the first quarter of 2022, compared to $43.5 million for the first quarter of 2021. These results reflect continued addition of new patients, continued high compliance and appropriate weight-based dosing.
Roche reported Evrysdi sales of approximately CHF 226 million for the first quarter of 2022, resulting in royalty revenue of $18.9 million to PTC in the first quarter of 2022, as compared to $6.7 million for the first quarter of 2021. In the first quarter of 2021, the first commercial sale of Evrysdi in the EU triggered a $20 million milestone payment to PTC. This 2021 achievement was reported as collaboration revenue.
Based on U.S. GAAP (Generally Accepted Accounting Principles) R&D expenses were $140.1 million for the first quarter of 2022, compared to $134.5 million for the first quarter of 2021. The increase reflects additional investment in research programs and advancement of the clinical pipeline.
Non-GAAP R&D expenses were $127.0 million for the first quarter of 2022, excluding $13.0 million in non-cash, stock-based compensation expense, compared to $120.8 million for the first quarter of 2021, excluding $13.7 million in non-cash, stock-based compensation expense.
GAAP SG&A expenses were $73.3 million for the first quarter of 2022, compared to $61.1 million for the first quarter of 2021. The increase reflects our continued investment to support commercial activities, including expanding our commercial portfolio.
Non-GAAP SG&A expenses were $59.7 million for the first quarter of 2022, excluding $13.6 million in non-cash, stock-based compensation expense, compared to $49.1 million for the first quarter of 2021, excluding $12.0 million in non-cash, stock-based compensation expense.
The change in the fair value of deferred and contingent consideration was $11.7 million for the first quarter of 2022, compared to $0.1 million for the first 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.
The net loss was $126.7 million for the first quarter of 2022, compared to a net loss of $128.6 million for the first quarter of 2021.
Cash, cash equivalents, and marketable securities were $587.8 million on March 31, 2022, compared to $773.4 million at December 31, 2021.
Shares issued and outstanding as of March 31, 2022 were 71,337,041.
PTC Reaffirms 2022 Financial Guidance:
PTC anticipates total revenues for full year 2022 to be between $700 and $750 million.
PTC anticipates net product revenues for the DMD franchise for full year 2022 to be between $475 and $495 million.
PTC anticipates GAAP R&D and SG&A expenses for full year 2022 to be between $915 and $965 million.
PTC anticipates Non-GAAP R&D and SG&A expense for 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:
PTC will host a conference call to discuss the first quarter corporate updates and financial results today at 4:30 pm ET and can be access by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 9963177. A live, listen-only webcast of the conference call can be accessed on the investor relations section of the PTC website at www.ptcbio.com. A webcast 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.