Fibrosis Treatment Based on Porcupine Inhibitor Being Readied for Clinical Testing

By developing a drug that blocks a molecule — going under the curious name, porcupine — Redx Pharma hopes to develop a new kind of treatment for idiopathic pulmonary fibrosis.

The company’s porcupine program also aims to come up with drugs that can improve responses to cancer immunotherapy.

“We’re delighted with the excellent progress our team has made with the Porcupine inhibitor RXC004,” said Dr. Neil Murray, CEO of Redx, in a press release. “As we move ever closer to the start of first-in-human clinical studies with RXC004, it is clear that not just ourselves, but key opinion leaders … are getting increasingly excited about Porcupine inhibition as an enhancer of immuno-oncology drugs.”

“In addition, we are moving full speed ahead with our second Porcupine inhibitor which provides a major opportunity as a potential treatment for some debilitating fibrotic diseases,” Murray added.

Porcupine is part of a molecular signaling pathway centered on a factor called Wnt (pronounced “wint”). Researchers are paying increasing attention to the role of molecular chains of events in cancer, but the pathway is known to be involved in numerous other disease processes, including the development of fibrosis in various organs.

The pathway is tightly controlled, and studies have shown that the porcupine molecule allows Wnt to be secreted upon the transfer of a chemical group called acyl. Redx hopes that by harnessing the actions of porcupine and preventing Wnt from being secreted, it may be possible to prevent disease processes linked to overactive Wnt signaling.

Currently, Redx has made most progress with the porcupine blocker RXC004 — a drug the company has shown to possess anticancer properties. But the drug’s strength is in improving the response to cancer immunotherapy with a checkpoint inhibitor targeting PD-1.

In an upcoming clinical trial, Redx will include the combination of RXC004 and a PD-1 blocker in one of the treatment groups. Redx is in the final stages of putting together a trial application with the U.K. Medicines and Healthcare products Regulatory Agency (MHRA).

The compound being developed for fibrotic diseases belongs to a different chemical class. In addition to idiopathic pulmonary fibrosis, Redx is developing the treatment for other fibrotic conditions, such as diabetic nephropathy and non-alcoholic steatohepatitis, a type of liver fibrosis.