The clinical stage biotech made the announcement about its oral, selective, rho associated coiled-coil containing protein kinase (ROCK2) today (August 21).
The drug is currently in a phase 2a clinical study for IPF with topline data expected in the first quarter of next year (2024).
The FDA can grant ODD to support the development and evaluation of new treatments to prevent, diagnose or treat a rare disease or condition that affects fewer than 200,000 in the US.
IPF - unmet need for patients
The designation provides Redx with various development and commercial incentives, including market exclusivity, in order to address this unmet need for patients suffering from IPF.
Dr Jane Robertson, chief medical officer, Redx Pharma said: “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 the first quarter of next year.”
Zelasudil is an orally available, highly selective small molecule inhibitor that targets ROCK2 which sits at a nodal point in a cell signalling pathway, believed to be central to fibrosis.
ROCK2 selectivity is important to avoid systemic hypotension, a serious cardiovascular side effect which has been seen in product candidates that systemically inhibit both ROCK1 and ROCK2.
Potential to treat several fibrotic diseases
As a selective ROCK2 inhibitor 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, results of which were presented at the International Colloquium on Lung and Airway Fibrosis (ICLAF) and the Antifibrotic Drug Development Summit (AFDD) last year (2022).
Redx is evaluating zelasudil initially as a treatment for IPF, a severe and life-threatening chronic lung condition with limited treatment options.
IPF is a lung disease that causes scarring and reduction in lung function slowly over time. Primarly older adults mainly aged 50 and over get the disease which involves irreversible and variable scarring, stiffening, and thickening of the lung tissues leading to sufferers experiencing shortness of breath and lack of oxygen absorption. There are more than 170,000 people suffering from IPF according to Redx’s research with a further 53,000 people diagnosed every year in the US, five EU countries and Japan.
The company says patients diagnosed with IPF have an estimated life expectancy of 3 to 5 years. There is no known cure and current treatment only slows progression of the disease.
