COVID-19 Delays Planned Trial of PTC743 and Gene Therapy Work, PTC Says

David Melamed, PhD avatar

by David Melamed, PhD |

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The COVID-19 pandemic will delay development work on a potential gene therapy, and the start of a planned and possibly pivotal clinical trial in the small molecule treatment PTC743, both intended for people with Frederich’s ataxia (FA), PTC Therapeutics said in a quarterly report.

PTC now expects to start a registrational study of PTC743 in patients toward this year’s end. Registrational studies are those intended to support a request for regulatory approval of the treatment under investigation.

PTC743 is designed to work by regulating inflammation and oxidative stress, which occurs when the production of toxic free radicals outweighs the body’s antioxidant defenses.

Besides FA, this therapy is also intended to treat refractory mitochondrial epilepsy. It is part of the Bio-e platform, whose potential treatments were acquired from from BioElectron Technology Corporation.

The company also expects that the demands of working under an ongoing pandemic will delay its gene therapy program for Friedreich’s ataxia by at least three months. It was anticipating bringing this therapy into an initial clinical trial between July and September, pending U.S. Food and Drug Administration (FDA) agreement. Now, a trial launch is more likely to take place between October and December.

Further updates on these delays will be given as the effects of COVID-19 are better known.

“COVID-19 has impacted multiple investigational new drug application (IND) enabling activities” for its gene therapy program, PTC stated  in the report. “Therefore, we now anticipate the IND filings [necessary to open a trial] will be delayed by at least one quarter and we will provide an update on timing as we better understand the impact of COVID-19.”

FA is caused by a genetic mutation that reduces production of frataxin (FXN). This protein is required for the proper functioning of mitochondria, the cells’ structures responsible for producing energy.

In its acquisition of Agilis Biotherapeutics in 2018, PTC gained rights to a potential therapy (then called AGIL-FA), intended to restore a functional FXN gene in the central nervous system (brain and spinal cord) of people with Friedreich’s ataxia.

Company officials, in a March investor update, noted that PTC743 might be a complementary treatment to this gene therapy.

PTC added that an estimated 25,000 people worldwide are diagnosed with Friedreich’s ataxia.