PTC Therapeutics Announces Initiation of Phase 2/3 Clinical Trial in Australia to Evaluate PTC299 for the Treatment of COVID-19

PTC Therapeutics, Inc., today announced the initiation of a Phase 2/3 trial called FITE19 to investigate PTC299, a dihydroorotate dehydrogenase (DHODH) inhibitor, as a potential treatment for COVID-19. PTC299 is an oral investigational drug with a novel dual-mechanism of action that has the potential to address the two critical elements of COVID-19: 1) the high viral replication and 2) the uncontrolled inflammatory response that ensues after infection. Study sites are enrolling patients at Monash Health, Sunshine Hospital and Westmead.

"As we continue to feel the overwhelming and devastating effects of COVID-19 globally, we are hopeful for the potential of PTC299 to be part of the solution," said Nikhil Jayaram, Regional Head Australasia, PTC Therapeutics, Inc. "Initiating a clinical trial in Australia allows us to further investigate the unique ways in which PTC299 can combat COVID-19, by reducing the high viral replication and also selectively attenuating the immune response caused by the uncontrolled cytokine storm resulting from the infection."

PTC299 is an oral, small molecule tablet that inhibits the cellular enzyme DHODH which is used to produce the RNA building blocks for the production of SARS-CoV-2. PTC299 has demonstrated potent inhibition of viral replication in SARS-CoV-2 cell-based assays, which has led to PTC moving rapidly into the clinic. A number of RNA viruses require the same building blocks, which explains why in preclinical tests PTC299 has also shown broad-spectrum antiviral activity.

If you are interested in participating in the study, please visit PTC clinical trials. The primary objective of the trial is to evaluate the clinical efficacy of PTC299 compared with placebo assessed by time to respiratory improvement in adults hospitalized with COVID-19.

PTC also has sites that are actively recruiting in the U.S., Brazil and Spain as part of the trial.

About PTC299

PTC299 is an investigational oral small molecule that inhibits the cellular enzyme dihydroorotate dehydrogenase (DHODH). Recently, DHODH has been independently identified as one of the top-three most promising targets for intervention in COVID-19. PTC299 is being evaluated for the potential treatment of patients with COVID-19. With a novel dual-mechanism of action, PTC299 inhibits DHODH activity which leads to a reduction of pyrimidine biosynthesis and SARS-CoV-2 viral replication, and selectively modulates the immune response by attenuating the stress-induced inflammatory cytokine storm. PTC299 has broad-spectrum antiviral activity and potent inhibition of numerous RNA viruses with low nanomolar potency. PTC299 is also currently being evaluated in an ongoing clinical study in acute myeloid leukemia (AML).

About PTC Therapeutics, Inc.

PTC is a science-driven, global biopharmaceutical company focused on the discovery, development and commercialization of clinically differentiated medicines that provide benefits to patients with rare disorders. PTC's ability to globally commercialize products is the foundation that drives investment in a robust and diversified pipeline of transformative medicines and our mission to provide access to best-in-class treatments for patients who have an unmet medical need.