On July 29, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and financial results for the second quarter ending June 30, 2021 (Press release, PTC Therapeutics, JUL 29, 2021, View Source [SID1234585359]).

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"I am delighted to say all facets of our business made substantial progress this quarter and we anticipate having four registration-directed trials ongoing by next quarter," said Stuart W. Peltz, Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "I am also proud of the execution of the global commercial team, which is driving the continued robust growth of the DMD franchise and has led us to raise our 2021 revenue guidance despite ongoing challenges from the pandemic."

Key Second Quarter and Other Corporate Updates:

The Duchenne muscular dystrophy (DMD) franchise had a total quarterly net product revenue of $102 million in the second quarter of 2021. This represents a 36% growth over the second quarter of 2020, continuing PTC’s trend of strong quarterly commercial revenues.
Translarna (ataluren) revenue growth was driven by expansion of the patient base, continued high compliance and broader access in existing geographies as well as continued geographic expansion including Russia, Central and Eastern Europe, Middle East and North Africa.
Emflaza (deflazacort) revenue growth was primarily due to new patient starts, continued high adherence and fewer discontinuations.
Evrysdi (risdiplam) received approval from the Ministry of Health Labor and Welfare in Japan in June 2021. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
Evrysdi is now approved in 54 countries.
In the U.S., more than 1800 patients are currently treated with Evrysdi, approaching 20% total market share.
Second Quarter Clinical Updates:

The registration-directed Phase 3 PTC923 phenylketonuria (PKU) trial, APHENITY, is expected to initiate in the third quarter of 2021.
PTC has multiple ongoing clinical trials, three of which are registration-directed clinical studies:
The MIT-E Phase 2/3 vatiquinone trial for mitochondrial epilepsy with data anticipated in the third quarter of 2022.
The MOVE-FA Phase 3 vatiquinone trial for Friedreich ataxia with data anticipated in 2023.
The FITE19 Phase 2/3 emvododstat trial in patients with COVID-19 is expected to be completed by year end 2021.
A Phase 1 healthy volunteer trial of the second Bio-e compound, PTC857, was recently completed and results demonstrated predictable pharmacology and no reported tolerability findings. This allows dose selection necessary to move forward to Phase 2, which we plan to initiate for amyotrophic lateral sclerosis (ALS) in the first quarter of 2022.
Results from additional cohorts are expected from the Phase 1 healthy volunteer trial of PTC518 for Huntington’s disease in the third quarter of this year. Phase 2 planning is already underway and is expected to be initiated by the end of 2021.
The Committee for Medicinal Products for Human Use (CHMP) imposed a clock stop in the aromatic L-amino acid decarboxylase deficiency (AADC-d) review process to allow for completion of its pre-approval inspections. This process is still ongoing and therefore the CHMP opinion is now expected in the fourth quarter of 2021.
For the Biologics License Application (BLA) for AADC-d, the third cannula surgery has been completed, and PTC will align with the FDA and expects to submit the BLA by the end of this year.
Second Quarter 2021 Financial Highlights:

Total revenues were $116.7 million for the second quarter of 2021, compared to total revenues of $75.2 million for the second quarter of 2020.
Total revenue includes net product revenue across the commercial portfolio of $103.1 million and royalty revenue of $13.6 million for the second quarter of 2021.
Translarna net product revenues were $52.6 million for the second quarter of 2021, compared to $38.6 million for the second quarter of 2020. These results reflect an increase in net product sales in existing markets as well as continued geographic expansion.
Emflaza net product revenues were $49.1 million for the second quarter of 2021, compared to $36.2 million for the second quarter of 2020. These results reflect new patient prescriptions, high compliance, and fewer discontinuations.
Roche reported first half of 2021 Evrysdi sales of approximately CHF 243 million, resulting in year-to-date royalty revenue of $20.2 million. Evrysdi is a product of a collaboration between PTC, Roche and the SMA Foundation.
U.S. GAAP (generally accepted accounting principles) research and development (R&D) expenses were $125.5 million for the second quarter of 2021, compared to $176.5 million for the second quarter of 2020. The decrease in research and development expenses is primarily related to one-time charges in the second quarter of 2020 of $53.6 million for our Censa merger, as well as $41.2 million for our commercial manufacturing service agreement with MassBiologics of the University of Massachusetts Medical School, or MassBio, related to dedicated manufacturing space for our lead gene therapy program, AADC deficiency. This was partially offset by increased investment in research programs and advancement of the clinical pipeline in the second quarter of 2021.
Non-GAAP R&D expenses were $112.0 million for the second quarter of 2021, excluding $13.4 million in non-cash stock-based compensation expense, compared to $168.0 million for the second quarter of 2020, excluding $8.6 million in non-cash stock-based compensation expense.
GAAP selling, general and administrative (SG&A) expenses were $68.9 million for the second quarter of 2021, compared to $53.7 million for the second quarter of 2020. The increase reflects our continued investment to support commercial activities including expanding our commercial portfolio, including an increase in rent and related expenses associated with entering into a long-term lease for our facility located in Hopewell Township.
Non-GAAP SG&A expenses were $56.6 million for the second quarter of 2021, excluding $12.3 million in non-cash stock-based compensation expense, compared to $45.3 million for the second quarter of 2020, excluding $8.3 million in non-cash stock-based compensation expense.
Change in the fair value of deferred and contingent consideration was $0.7 million for the second quarter of 2021, compared to $7.7 million for the second quarter of 2020. 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 $118.4 million for the second quarter of 2021, compared to net loss of $181.4 million for the second quarter of 2020.
Cash, cash equivalents and marketable securities was $947.1 million at June 30, 2021, compared to $1.1 billion at December 31, 2020.
Shares issued and outstanding as of June 30, 2021 were 70,559,330.
PTC Updates Full Year 2021 Guidance as Follows:

PTC now anticipates net product revenues for the DMD franchise for the full year 2021 to be between $370 and $390 million.
PTC continues to anticipate GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC continues to anticipate Non-GAAP R&D and SG&A expense for the full year 2021 to be between $725 and $755 million, excluding estimated non-cash, stock-based compensation expense of $100 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.

Today’s Conference Call and Webcast Reminder:

PTC will host a conference call to discuss the second quarter of 2021 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 7064479. 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.