On January 13, 2020 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported a corporate update that will be presented at the 38th Annual J.P. Morgan Healthcare Conference on Wednesday, January 15th at 8:30 a.m. PST. Stuart W. Peltz, Ph.D., PTC’s Chief Executive Officer, will provide an update on the 2019 accomplishments and highlight value creating events in the upcoming year (Press release, PTC Therapeutics, JAN 13, 2020, View Source [SID1234553078]). Additionally, the Company is providing preliminary 2019 financial results and 2020 financial guidance. The presentation will be webcast live on the Events and Presentations page under the investors’ section of PTC Therapeutics’ website at www.ptcbio.com
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Corporate Highlights:
Gene Therapy Platform Update:
PTC has submitted a Marketing Authorization Application (MAA) for the potential approval of a gene therapy treatment, PTC-AADC, for AADC deficiency with the European Medicines Agency (EMA). PTC expects the Committee for Medicinal Products for Human Use (CHMP) opinion in 2H 2020.
In a recent interaction with the U.S. Food and Drug Administration (FDA) there was a request for additional information concerning the use of the commercial delivery system for PTC-AADC in young patients. Patients have been identified who are immediately available for treatment with the commercial delivery system. We anticipate that this will cause a short delay of the Biologics License Application (BLA) and should allow for submission to the FDA in 2Q 2020.
PTC has identified 200 AADC patients to date and continues to anticipate that over 300 AADC patients will be identified by launch.
PTC-FA gene therapy for Friedreich ataxia is progressing and is anticipated to enter the clinic in 3Q 2020.
In order to control and accelerate its gene therapy platform, PTC secured a 15-year lease on ~185,000 sq. ft. of space, which includes a state-of-the-art biologics production facility with supporting research and operations buildings in NJ. PTC expects manufacturing to begin at this facility in 2020.
Risdiplam Regulatory & Clinical Updates:
The risdiplam Prescription Drug User Fee Act (PDUFA) date for a decision by the FDA is May 24, 2020. Risdiplam is expected to be indicated for spinal muscular atrophy (SMA) type 1, 2 & 3 patients, if approved.
In November 2019, PTC announced that the pivotal part of the SUNFISH trial in SMA type 2/3 patients was successful, meeting its primary endpoint. Data from the pivotal part of SUNFISH will be presented at the SMA Europe Conference from February 5 – 7, 2020.
Topline results from the pivotal portion of the open label FIREFISH study in type 1 SMA patients are anticipated in 1Q 2020.
The SMA program has studied the broadest cohort of SMA patients in clinical trials to date. This program is a collaboration between PTC, Roche and the SMA Foundation.
Expanding commercial platform:
The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna and Emflaza, continues to grow with 2019 unaudited revenue of approximately $291 million.
Translarna continues to grow with further penetration into existing territories, geographic expansion into new territories, increased awareness and earlier diagnosis. PTC plans to re-submit the Translarna New Drug Application (NDA) to the FDA with the data from the dystrophin study in mid-year 2020.
Growth in Emflaza revenue was based on a recent label expansion in the 2-5-year-old patient population, and reduced payer restrictions resulting from multiple publications showing benefit of Emflaza relative to prednisone. One such recent publication, which included real-world data from Cincinnati Children’s hospital, specifically highlighted the benefit of switching from prednisone to Emflaza.
Tegsedi (inotersen) received approval from the Brazilian health regulatory authority (ANVISA) for the treatment of stage 1 or 2 polyneuropathy in adult patients with hereditary transthyretin amyloidosis (hATTR), to delay disease progression and improve quality of life. The commercial launch for Tegsedi is ongoing with patient and physician services to support the launch, including medical education and diagnosis efforts.
Growing pipeline and R&D capabilities:
In 2020, PTC plans to initiate three trials in its newly acquired redox platform with two unique compounds that regulate inflammation and oxidative stress.
These include potential registrational trials in mitochondrial epilepsy and Friedreich ataxia with PTC743. Additionally, a Phase 1 trial with PTC857 is also planned targeting GBA Parkinson’s disease.
In late 2020, PTC expects to file an investigational new drug (IND) application for its development candidate in Huntington disease. PTC has several additional programs from the splicing platform in development with undisclosed targets.
Preliminary Unaudited 2019 Financial Results
Total unaudited revenue for 2019 was approximately $306 million. This includes expected net product revenue for the Duchenne muscular dystrophy franchise of approximately $291 million for the full year 2019 and a $15 million milestone payment from Roche for the risdiplam NDA acceptance.
PTC expects to report net product revenue for Translarna of approximately $190 million for full year 2019. As a reminder, the ANVISA approval in Brazil should lead to expanded market access in 2020 and beyond but led to a price discount that impacted 2019 net sales.
Additionally, PTC expects to report net product revenue for Emflaza of approximately $101 million for the full year 2019.
PTC expects to report 2019 year-end cash and cash equivalents of approximately $686 million.
PTC is currently in the process of finalizing its financial results for the 2019 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2019, subject to the completion of PTC’s financial closing procedures. In addition, the above information is subject to revision as PTC completes its financial closing procedures for fiscal 2019.
2020 Guidance
PTC anticipates full year net product revenues for the DMD franchise to be between $320 and $340 million. New product launches, including Tegsedi, Waylivra, PTC-AADC and risdiplam, could contribute revenue in 2020. Remaining milestones and royalties from Roche on the SMA program are outlined in the table below.
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.
The increase in R&D and SG&A expense are based in part on highly leverageable and scalable investments towards the $1.5 billion projected revenue in 2023, including gene therapy manufacturing, an increase in the number of programs advancing into the clinic and launches.