Oncostellae

Oncostellae SL is a privately owned, clinical-stage biopharmaceutical company focused on the discovery and development of proprietary drug candidates with strong IP and worldwide rights for immunology and other indications with high unmet medical need.

According to its business model, Oncostellae intends to develop its novel compounds from the idea up to clinical proof-of-concept (Phase 2a) and, subsequently, sign a licensing or codevelopment agreement with a pharmaceutical company that continues the clinical development, registration and commercialization of the product. In return, Oncostellae will receive upfront and milestone payments as well as royalties on sales when the product reaches the market. The revenues from the license agreement can in turn be used to finance other developments.

This business model is well established in the biotech sector. Spanish companies that have consolidated this model are, for example, Oryzon Genomics (Roche 2014), Palobiofarma (Novartis 2015, Xoma 2019), Ability Pharmaceuticals (Sciclone 2016), Origo Biopharma (Agomab 2021) and Leukos Biotech (AOP Health 2022).

Oncostellae has currently three oral small-molecule programs for indications with high medical need in their development pipeline:

• OST-122 is an oral, gut-restricted JAK3/TYK2/ARK5 inhibitor for the safe local treatment of IBD in the gut,  overcoming systemic toxicities inherent in current JAKi and other immunosuppressive medication. 
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• OST-122 is the first non-systemic JAK inhibitor for the safe local treatment of IBD that acts exclusively in the gut. Unlike other IBD medication, OST-122 is practically absent from circulating plasma, even at high doses, offering an unprecedented treatment option that is free from causing systemic toxicities. In addition, OST-122 has a unique subtype-specific inhibitor activity targeting JAK3 and tyrosine kinase 2 (TYK2), making it a suitable treatment for UC and CD. With its additional potent AMPK-related protein kinase 5 (ARK5) inhibitor activity, OST-122 holds further potential to specifically benefit CD patients with fibrotic lesions, a condition with currently only limited treatment options. 

In a Phase 2a proof-of-concept trial in UC, OST-122 demonstrated excellent safety and tolerability, minimal systemic exposure and promising trends of clinical activity. We are currently preparing a confirmatory Phase 2b clinical trial in UC patients.

• OST-122 is protected in patent application WO2018220252 and has been granted worldwide rights and market exclusivity until 2038. Exclusivity can be extended by 5 years in Europe and the US, and by filing additional IP on a new solid form (in preparation).
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• OST-499 is a non-steroidal progesterone receptor (PR) antagonist for the treatment of uterine fibroids that has shown a good safety profile in a dose-ascending Phase Ia clinical trial in patients with solid tumors. 

Unlike current SPRM (selective PR modulators), OST-499 has a non-steroidal backbone and is devoid of chemical liabilities associated with hepatotoxicity, and has not shown any signs of liver alterations in preclinical 28-day tox studies, nor in the clinic. OST-499 has the potential to become a best-in-class SPRM alternative to ulipristal by reducing the potential for drug-induced liver injury, thus eliminating the need for liver function monitoring. 

In addition, OST-499 has the potential to become a treatment alternative to GnRH modulators, with a more favorable safety/tolerability profile, devoid of hypoestrogenic effects (hot flashes, bone mineral density loss, etc.) and as an alternative to surgery to preserve fertility. This addition to the UF armamentarium could expand treatment options and significantly improve the quality of life for women affected by this protracted condition.

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• OST-499 is protected in patent application WO2019086720 and has been granted worldwide rights and market exclusivity until 2039.
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• OST-664 is a selective ARK5 kinase inhibitor for the treatment of fibrosis, an indication with few treatment options, and is currently in preclinical optimization.