RARE Daily

PTC Cuts 25 Percent of Workforce in Further Strategic Prioritization

September 29, 2023

Rare Daily Staff

Rare disease focused biotech PTC Therapeutics announced further strategic prioritization and an associated workforce reduction of 25 percent.

The portfolio prioritization continues the process initiated in May 2023 as the company continues to focus its resources on its differentiated, high potential R&D programs and its global commercial infrastructure. The company also confirmed its plans to submit the re-examination request for the CHMP opinion on Translarna.

The CHMP of the European Medicines Agency gave a negative opinion on the conversion of the conditional marketing authorization to full marketing authorization of Translarna (ataluren) for the treatment of nonsense mutation Duchenne muscular dystrophy (nmDMD). The negative opinion also applies to the renewal of the existing conditional authorization. Translarna received conditional marketing authorization in Europe in 2014 and the conditional authorization was renewed in 2017. As part of the renewal of the conditional marketing authorization, PTC agreed to a specific obligation to conduct a third placebo-controlled trial, Study 041. PTC shared the results of Study 041, which included nominally statistically significant results on several key endpoints in the overall enrolled Intent-to-Treat population of 359 boys even though it did not meet statistical significance in the primary analysis subgroup.

Primarily affecting males, Duchenne is a rare and fatal genetic disorder that results in progressive muscle weakness from early childhood and leads to premature death in the mid-20’s due to heart and respiratory failure. It is a progressive muscle disorder caused by the lack of functional dystrophin protein. Dystrophin is critical to the structural stability of all muscles, including skeletal, diaphragm, and heart muscles. Patients with Duchenne can lose the ability to walk (loss of ambulation) as early as 10 years old, followed by loss of the use of their arms. Duchenne patients subsequently experience life-threatening lung complications, requiring the need for ventilation support, and heart complications in their late teens and 20s.

“We remain confident that we have the data to address the concerns raised by CHMP in its negative opinion,” said Matthew Klein, CEO of PTC Therapeutics. “The totality of evidence collected in three placebo-controlled trials and in the STRIDE registry provide clear evidence of Translarna’s benefit. In addition, the patient and physician communities strongly believe in the benefits of Translarna and have shared their motivation to support the efforts to maintain authorization for the only approved Duchenne muscular dystrophy therapy in Europe.”

The workforce reduction impacted approximately 25 percent of the organization, and primarily included employees working on early-stage research programs, employees at the gene therapy manufacturing facility located in Hopewell, NJ, and associated SG&A functions. Implementation of these cost saving measures is expected to result in an approximate 20 percent reduction in annualized operating expenses compared to 2023 OPEX guidance.

Photo: Matthew Klein, CEO of PTC Therapeutics

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