Redx gains FDA orphan drug status for potential IPF treatment

 (), a clinical-stage biotechnology company, has been awarded orphan drug status by the Food and Drug Administration (FDA) for Zelasudil, a potential treatment for Idiopathic Pulmonary Fibrosis (IPF). 

IPF is a life-threatening disease of the lungs that progressively causes scarring and a reduction in lung function. 

Zelasudil targets ROCK2, an enzyme central to fibrosis or lung-scarring, and is currently in Phase 2a clinical study for IPF, with data expected in Q1 2024.

ROCK2 selectivity is important to avoid systemic hypotension, a serious cardiovascular side effect that has been seen in product candidates that systemically inhibit both ROCK1 and ROCK2, both enzymes linked to scarring. Zelasudil, has the potential to treat several fibrotic diseases and has demonstrated robust anti-fibrotic effects in a range of industry-standard in vivo preclinical models.

The FDA gives Orphan Drug Designation to help create new treatments for rare diseases or conditions that affect fewer than 200,000 people in the US. 

Dr Jane Robertson, Chief Medical Officer of Redx Pharma commented: "We are delighted that the FDA has recognised the potential of zelasudil for the treatment of IPF and granted Orphan Drug Designation. Selectively targeting ROCK2 is an exciting, novel approach which could provide a new treatment option for patients with IPF, and with potential applications in other interstitial lung diseases and cancer-associated fibrosis.  We are encouraged by both the strength of our preclinical package as well as the clinical results to date and we look forward to reporting Phase 2a topline data in Q1 2024."

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Rare diseases are a global health priority, with about 95% of all rare diseases not yet having treatments. More than 170,000 patients suffer from IPF, while an additional 53,000 people receive new diagnoses annually across the United States, the five countries of the European Union, and Japan combined. Those afflicted by IPF face an estimated life expectancy of 3 to 5 years, with no known cure - current treatment only slows progression of the disease. 

Along with zelasudil, Redx has two other drug candidates, the Porcupine inhibitor RXC004 being developed as a targeted treatment for Wnt-ligand dependent cancers, which are influenced by abnormal activity in the Wnt signaling pathway and can lead to uncontrolled cell growth and tumor development. This candidate is expected to report combination with anti-PD-1 Phase 2 data during 2023. 

Redx's third drug candidate, RXC008, a GI-targeted ROCK inhibitor for the treatment of fibrostenotic Crohn's disease, is progressing towards a CTA application at the end of 2023.  

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