On March 2, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update and reported financial results for the fourth quarter and full year ending December 31, 2019 (Press release, PTC Therapeutics, MAR 2, 2020, View Source [SID1234555058]).

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"2019 was a year of outstanding execution for PTC. I’m proud of the progress made in every area of the business," said Stuart Peltz, Ph.D., CEO of PTC Therapeutics. "We’ve significantly expanded our rare disease portfolio, strengthened our global commercial engine and added vital gene therapy manufacturing capabilities, all while delivering on our revenue guidance. The innovation in our diverse, multiplatform pipeline continues to create value for stakeholders."

Key Fourth Quarter and Other Corporate Highlights:

Expanding commercial footprint including continued revenue growth

Total revenue for the full year 2019 was $307 million. The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna (ataluren) and Emflaza (deflazacort), continues to grow with 2019 revenue of $291 million.

PTC continues to leverage its strong Latin American infrastructure to support the launch of multiple products. In 2019, Translarna received approval from the Brazilian health regulatory authority (ANVISA) for ambulatory nmDMD patients 5 years and older. In addition, Tegsedi (inotersen) received ANVISA approval for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR). In 2019, PTC established an early access program for patients with Familial Chylomicronemia Syndrome (FCS) in Latin America. PTC anticipates filing for approval of Waylivra (volanesorsen) for the treatment of FCS from ANVISA in 2H 2020.

Real-world data from the Translarna STRIDE registry demonstrated that boys with nonsense mutation Duchenne muscular dystrophy (nmDMD) treated with Translarna and standard of care, preserved the ability to walk for 3.5 years longer than compared with those in a matched natural history cohort. Importantly, pulmonary function was also preserved in those treated with Translarna.

A recent publication on Emflaza adds to the growing body of evidence showing its relative benefit and supports the advantage of switching from prednisone to Emflaza. The data in the publication demonstrated that patients treated with Emflaza had better ambulatory function and were able to walk longer, had a lower risk of scoliosis, greater percentage lean body mass and lower weight than patients treated with prednisone.

Positive pivotal trials for risdiplam support planned global launch in broad range of SMA patient types

Following the U.S. Food and Drug Association’s (FDA) acceptance of the New Drug Application (NDA) for risdiplam, a Prescription Drug User Fee Act (PDUFA) date for risdiplam has been set for May 24, 2020.

Roche’s Marketing Authorization Application (MAA) filing with the European Medicines Agency (EMA) for risdiplam is expected mid-year 2020.

Data from part 2 of the FIREFISH trial will be presented during the American Academy of Neurology (AAN) 2020 Annual Meeting, from April 25 to May 1, 2020.

PTC gene therapy platform continues to make progress with regulatory filings and long-term lease of manufacturing facility

In January 2020, the EMA accepted the MAA for the potential approval of PTC-AADC for the treatment of Aromatic l-amino acid decarboxylase (AADC) deficiency. PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion by year end 2020.

PTC expects to submit a Biologics License Application (BLA) to the FDA in 2Q 2020.

PTC continues to anticipate that more than 300 global, addressable AADC patients will be identified by launch.

In 2019, PTC leased a state-of-the-art biologics manufacturing facility with supporting research and operations buildings in N.J. PTC expects manufacturing at this facility to begin in 2020.

Continued progress with PTC’s multi-platform, multi-product pipeline

The Phase 2 STAR trial in patients with nonsense mutation aniridia evaluating the effect of ataluren did not meet its primary endpoint of Maximum Reading Speed as measured by MNREAD. The results did show a trend in favor of ataluren and the results will be discussed with experts in the coming weeks, after which a decision will be made on a path forward for the program in this indication.

Topline data from the U.S. study to assess dystrophin levels in subjects with nmDMD after treatment with ataluren are expected in 2Q 2020.

In 2019, PTC declared a development candidate in its Huntington’s Disease program. The compound is currently in GLP safety toxicology studies and PTC expects to initiate a clinical trial by the end of the year. The Huntington’s Disease program was developed through PTC’s alternative splicing platform.

In 2020, PTC plans to initiate three clinical trials in two compounds that regulate inflammation and oxidative stress from its Bio-e (redox) platform. PTC acquired the Bio-e platform through an acquisition of assets from BioElectron Technology Corporation in 2019.

Potential registrational trial of PTC743 in patients with Mitochondrial Epilepsy (MEDS) expected to begin in 2Q 2020. MEDS is estimated to affect 5,000-6,000 patients in the U.S. and E.U. combined.

Potential registrational trial of PTC743 in patients with Friedreich Ataxia (FA) expected to begin in 3Q 2020. This is anticipated to be complementary to PTC-FA, a gene therapy candidate for the treatment of FA, which is expected to enter the clinic in 3Q 2020. FA is estimated to affect 25,000 patients worldwide.

PTC857 will enter into a Phase 1 healthy volunteer study in 3Q 2020. The first indication for PTC857 is planned to be GBA Parkinson’s disease based on strong pre-clinical rationale. This condition affects approximately 50,000-90,000 patients in the U.S.

Additional updates on PTC’s platforms and pipeline will be provided at PTC’s Analyst Day on June 16, 2020.

Fourth Quarter and Full-year 2019 Financial Highlights:

Total revenues were $96.5 million for the fourth quarter of 2019, compared to $86.3 million for the fourth quarter of 2018. Total revenues were $307 million for the full year 2019, compared to $264.7 million for the full year 2018.

Translarna net product revenues were $48.4 million for the fourth quarter of 2019, compared to $56 million for the fourth quarter of 2018. Translarna net product revenues were $190 million for the full year 2019, compared to $171 million for the full year 2018.

Emflaza net product revenues were $32.7 million for the fourth quarter of 2019, compared to $29.8 million for the fourth quarter of 2018. Emflaza net product revenues were $101 million for the full year 2019, compared to $92 million for the full year 2018.

GAAP R&D expenses were $81.8 million for the fourth quarter of 2019, compared to $53.6 million for the fourth quarter of 2018. GAAP R&D expenses were $257.4 million for the full year 2019, compared to $172 million for the full year 2018. The increase in R&D expenses reflects costs associated with advancing the gene therapy platform, increased investment in research programs, advancement of the clinical pipeline and the upfront $10M payment for the acquisition of the BioElectron assets.

Non-GAAP R&D expenses were $76.2 million for the fourth quarter of 2019, excluding $5.6 million in non-cash, stock-based compensation expense, compared to $49.7 million for the fourth quarter of 2018, excluding $4 million in non-cash, stock-based compensation expense. Non-GAAP R&D expenses were $236.6 million for the full year 2019, excluding $20.8 million in non-cash, stock-based compensation expense, compared to $155.9 million for the full year 2018, excluding $16.1 million in non-cash, stock-based compensation expense.

GAAP SG&A expenses were $63.5 million for the fourth quarter of 2019, compared to $48.7 million for the fourth quarter of 2018. GAAP SG&A expenses were $202.5 million for the full year 2019, compared to $153.6 million for the full year 2018. The increase in SG&A expenses was primarily due to continued investment to support our commercial activities.

Non-GAAP SG&A expenses were $57.7 million for the fourth quarter of 2019, excluding $5.8 million in non-cash, stock-based compensation expense, compared to $44.2 million for the fourth quarter of 2018, excluding $4.5 million in non-cash, stock-based compensation expense. Non-GAAP SG&A expenses were $181.2 million for the full year 2019, excluding $21.3 million in non-cash, stock-based compensation expense, compared to $136.4 million for the full year 2018, excluding $17.2 million in non-cash, stock-based compensation expense.

Change in the fair value of deferred and contingent consideration was $12.4 million for the quarter ended December 31, 2019, compared to $19.3 million for the quarter ended December 31, 2018. Change in the fair value of deferred and contingent consideration was $48.4 million for the year ended December 31, 2019, compared to $19.3 million for the year ended December 31, 2018. The change is related to the fair valuation of the potential future consideration to be paid to former equity holders of Agilis, as a result of our merger with Agilis which closed in August 2018. Changes in the fair value were due to the re-calculation of discounted cash flows for the passage of time and changes to certain other estimated assumptions.

Net loss was $77.7 million for the fourth quarter of 2019, compared to net loss of $48.3 million for the fourth quarter of 2018. Net loss was $251.6 million for the full year 2019, compared to net loss of $128.1 million for the full year 2018.

Cash, cash equivalents, and marketable securities was $686.6 million at December 31, 2019, compared to $227.6 million at December 31, 2018.

Shares issued and outstanding as of December 31, 2019 were 61,935,870.

PTC Reaffirms Full Year 2020 Guidance as Follows

PTC anticipates full year DMD franchise net product revenues to be between $320 and $340 million.

PTC anticipates GAAP R&D and SG&A expense for the full year 2020 to be between $610 and $640 million.

PTC anticipates Non-GAAP R&D and SG&A expense for the full year 2020 to be between $545 and $575 million, excluding estimated non-cash, stock-based compensation expense of approximately $65 million.

Today’s Conference Call and Webcast Reminder:
Today’s conference call will take place at 4:30 pm ET and can be accessed by dialing (877) 303-9216 (domestic) or (973) 935-8152 (international) five minutes prior to the start of the call and providing the passcode 1732477. 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. The accompanying slide presentation will be posted on the investor relations section of the PTC website. 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.