On January 11, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that it will present an update on its R&D pipeline and commercial progress at the 39th Annual J.P. Morgan Healthcare Conference, Monday, Jan. 11 at 7:30 a.m. EST. Stuart W. Peltz, Ph.D., Chief Executive Officer of PTC Therapeutics, will provide an update on 2020 accomplishments and highlight upcoming potential value-creating milestones (Press release, PTC Therapeutics, JAN 11, 2021, View Source [SID1234576139]). Preliminary 2020 unaudited financial results and 2021 financial guidance will also be provided. The presentation will be webcast live on the Events and Presentations page of the Investors section of PTC Therapeutics website at www.ptcbio.com.

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Key 2020 Corporate Highlights:

The Duchenne muscular dystrophy (DMD) franchise, consisting of Translarna (ataluren) and Emflaza (deflazacort), continue to demonstrate year-over-year growth, with 2020 preliminary unaudited revenue of approximately $331 million.
Translarna growth is primarily driven by geographical expansion and label modifications allowing broader access.
Emflaza experienced strong 38% year-over-year revenue growth in 2020 due to increased patient prescriptions and high compliance.
In August, Evrysdi (risdiplam) was approved by the FDA for patients with Spinal Muscular Atrophy (SMA). A strong global launch is underway with increasing U.S. patient uptake across all disease subtypes in both treatment naïve and switch patients. The European Medicines Agency’s Committee for Medicinal Products for Human Use (CHMP) opinion for Evrysdi is expected in 1H 2021, and priority review of the Japanese New Drug Application (NDA) is ongoing. Evrysdi is the first at-home, orally administered SMA treatment and was developed from PTC’s proprietary splicing platform. Evrysdi is a product of the SMA collaboration between PTC, the SMA Foundation and Roche.
PTC initiated three clinical trials from its novel Bio-e platform, including two registration-directed trials with vatiquinone (PTC743) in Mitochondrial epilepsy (MIT-E) and Friedreich ataxia (MOVE-FA) that are actively enrolling.
2021 Potential Value-Creating Milestones:

Top-line results from the Translarna dystrophin study are expected in 1Q 2021. With positive results, PTC plans to submit an NDA to the FDA.
PTC is preparing to launch its first potential gene therapy, PTC-AADC, for the highly morbid genetic pediatric disorder aromatic L-amino acid decarboxylase deficiency (AADC-d). Launch efforts include expanding genetic testing programs and the identification and preparation of expert pediatric neurosurgical centers.
PTC-AADC expected regulatory milestones include a CHMP opinion for a potential approval and the submission of a Biologics License Application (BLA) to the FDA in 1H 2021.
Results from PTC’s Huntington disease program Phase 1 study of PTC518 in healthy volunteers are expected in 1H 2021. PTC518 was discovered from PTC’s validated splicing platform.
A registration-directed clinical trial of PTC299 for COVID-19 (FITE19) continues to enroll patients. PTC anticipates completing the study in 1H 2021.
A registration-directed study, APHENITY, for PTC923 in patients with phenylketonuria (PKU) is expected to initiate in mid-2021.
Results by year end are expected from ongoing clinical trials evaluating PTC596 in Leiomyosarcoma and Diffuse Intrinsic Pontine Glioma (DIPG).
Gene therapy manufacturing for clinical trials will begin at PTC’s state-of-the-art biologics production facility in Hopewell, N.J.
Preliminary Unaudited 2020 Financial Results:

Total unaudited net product revenue for full year 2020 was approximately $333 million.
DMD franchise revenue for year end 2020 included net product revenue for Translarna of approximately $192 million with $69 million in revenue in the fourth quarter and approximately $139 million year end 2020 revenue for Emflaza with $37 million in revenue in fourth quarter.
PTC expects to report $42.5 million in 2020 revenue associated with Evrysdi milestones. PTC will report fourth quarter royalty revenue for Evrysdi on the company’s next earnings call.
PTC expects to report 2020 year-end cash and cash equivalents of approximately $1.1 billion.
PTC is currently in the process of finalizing its financial results for the 2020 fiscal year. The above information is based on preliminary unaudited information and management estimates for the full year 2020, subject to the completion of PTC’s financial closing procedures.

2021 Financial Guidance:

PTC anticipates full year net product revenues for the DMD franchise to be between $355 and $375 million.
PTC anticipates GAAP R&D and SG&A expense for the full year 2021 to be between $825 and $855 million.
PTC anticipates 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 approximately $100 million.