Zelasudil

Phalguni Deswal @Phalguni_GD With a limited number of approved drugs for IPF in the market, Esbriet and Ofev dominate the sales in the field. Image Credit: eamesBot / Shutterstock. Avalyn Pharma secured $175m in a Series C financing round to fund the clinical development of inhaled pulmonary therapies. The US-based company plans to advance idiopathic pulmonary fibrosis (IPF) therapies, AP01 (inhaled pirfenidone) and AP02 (inhaled nintedanib), to Phase IIb and IIa trials, respectively. Approved oral drugs— Roche’s Esbriet/Pirespa (pirfenidone) and Boehringer Ingelheim’s Ofev (nintedanib) —are commonly used to reduce scarring. Avalyn is developing inhaled formulations in an effort to reduce the systemic side effects of the drugs. Avalyn CEO Lyn Baranowski highlighted the need for an alternate mode of administration stating : “Fewer than 30% of patients in the US are treated with either of the two approved oral medicines today due to their significant tolerability challenges.” AP01 is currently in an open-label extension of the Phase Ib trial in IPF patients, whilst AP02 has been evaluated in a Phase I study with healthy volunteers. With a limited number of approved drugs for IPF in the market, Esbriet and Ofev dominate the sales in the field. Esbriet generated SFr119m ($129.5m) in sales in H1 2023, as per Roche’s financials. However, there are more than 300 drugs currently in development to treat IPF, with 30% of these drugs currently in clinical development (Phase I or later), as per GlobalData. GlobalData is the parent company of Pharmaceutical Technology . Other drugs currently in development for IPF include Redx Pharma’s zelasudil (RXC007), which was granted orphan drug designation by the US Food and Drug Administration (FDA) based on Phase II data. United Therapeutic’s Tyvaso (treprostinil) is also being studied in two Phase III trials (NCT04708782 and NCT04905693).

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. googletag.cmd.push(function () { googletag.display('text-ad1'); }); 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.