Phase 2 Trial of C21, Potential Oral Therapy for IPF, Opens

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by Joana Carvalho |

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Phase 2 trial opens

A first patient has been enrolled in a proof-of-concept Phase 2 trial of C21 (VP01), Vicore Pharma’s investigational oral therapy for idiopathic pulmonary fibrosis (IPF) and other lung diseases.

“We are pleased to announce that we can now start our IPF trial despite the ongoing COVID-19 pandemic,” Anne Katrine Cohrt, senior director of clinical operations at Vicore Pharma, said in a press release.

The patient was recruited to the study (NCT04533022) at a site in India. Other sites are to open in the U.K., where the trial has been approved, and preparations are underway to expand the study to other countries. Contact and site information will become available here.

This open-label and single-arm trial aims to assess the safety, efficacy, and pharmacological properties of C21 in around 60 adults with IPF (ages 40 and older). Enrolled participants will receive the experimental therapy twice daily for six months.

After completing this initial treatment period, patients will have the option to continue treatment with C21 for an additional three months.

The study’s main goal is to assess the frequency and severity of side effects occurring over the first six months of treatment with C21.  Its effect on patients’ lung function and its pharmacokinetic properties — how C21 is distributed and processed in the body, and then eliminated — will also be evaluated.

C21 is a first-in-class activator of angiotensin II receptor type 2 (AT2R), a protein receptor that when active promotes blood vessel widening and prevents tissue scarring (fibrosis) by activating the “protective arm” of the renin-angiotensin system (RAS) signaling cascade.

Through this mechanism of action, C21 is expected to improve blood flow and reduce the formation of scar tissue in the lungs.

While C21 is mainly being investigated as a possible IPF treatment, its potential to treat people with systemic sclerosis or acute COVID-19 infection is also being explored.

Preclinical studies in animal models of pulmonary fibrosis and pulmonary hypertension are reported to have shown C21 as effective at lowering tissue fibrosis. More recently, the investigational therapy was also found to successfully block the activity of transforming growth factor-beta 1, a key regulator of fibrosis, in lung tissue samples taken from an IPF patient.

C21 has been named an orphan drug by regulatory authorities in the U.S. and Europe, supporting its testing and further development.