PTC Therapeutics, Inc. (NASDAQ:PTCT) Reports 30% YoY Growth In Q2 2020 Sales Of Emflaza: PTC Will Net A Milestone Payment Of $15 Million Through MAA Filing

PTC Therapeutics, Inc. (NASDAQ:PTCT) reported a drop of 12% to $75.2 million in Q2 2020 revenues. It earned $38.6 million from the sale of Translarna products in Q2 2020. The company expects to recoup the revenues later this year on bagging a Group PO (Purchase Order) from Brazil.


PTC achieved a growth of 30% to $36.2 million in Q2 2020 for Emflaza through the Duchenne muscular dystrophy franchise. The company is identifying patients in LATAM, Europe, and other vital markets for Translarna products even during the ongoing coronavirus crises.

EMA (European Medicines Agency) confirmed its benefit/ risk profile for the 6th consecutive year as part of the Tranlarna renewal. According to a previous communiqué from PTC, CHMP (Committee for Medical Products for Human Use) recommended removal of the statement ‘efficacy is not demonstrated in the patients’. It allows healthcare professionals to make their decision to provide appropriate treatment for patients who are on Translarna and lost ambulation.

Acquires a late-stage asset for PKU

PTC took over the late-stage assets for PKU (phenylketonuria) in Q2 2020. The clinical-stage investigational therapy – PTC923 is targeted for the treatment of IEM (inborn errors of metabolism) that comprises PKU and other diseases related to defects in biochemical pathways. Nonclinical trials are underway by PTC to determine long term dose of Phase 3 clinical study in 2021 in PKU.

PTC will soon file for MAA (Market Authorization Application) for risdiplam to EMA. It will receive $15 million from Riche following MAA filing. Roche applied for commercialization of risdiplam in Russia, Chile, Brazil, Indonesia, Taiwan, and South Korea.

PTC applied for marketing authorization of Waylivra in people with FCS (familial chylomicronemia syndrome).  It expects the decision of ANVISA in 2021.

PTC expanded the collaboration pact with MassBiologics to produce and commercialize AADCD (aromatic L-amino acid decarboxylase deficiency).

PTC occupied 220,000 Sq. Ft. at Hopewell based BMS Site on lease basis to produce clinical materials for use in gene therapy programs. It will commence production in 2021.

To provide real-world experience for the recent graduates in the biopharmaceutical industry, PTC unveiled an international internship program called TPP (Talent Pipeline Program).