Oncoheroes Biosciences, a US-based start-up focuses exclusively on advancing and developing new therapies for childhood cancer, has out-licensed volasertib’s global development and commercialisation rights for cancer in adults to the US biopharmaceutical firm Notable Labs, a pioneer biotech developing preventive and precision medicine. Oncoheroes, that will continue clinical development of volasertib to treat childhood cancer, was co-founded by Catalan entrepreneur Ricardo García and Italian scientist Cesare Spadoni, both fathers affected by childhood cancer. The company, headquartered in Boston (United States), centralises the drug discovery activity in its Lab at the Barcelona Science Park.

Volasertib is an inhibitor of the kinase PLK-1, an enzyme shown to occur in a wide range of solid tumours and haematological cancers. The compound was discovered and developed by Boehringer Ingelheim for acute myeloid leukaemia (AML). However, the company decided to stop the development of volasertib for strategic reasons after the results of a phase III study in adults did not meet its expectations.

In 2019, Oncoheroes obtained the exclusive global licence of volasertib to continue its clinical development for younger oncology patients. In 2020, volasertib was awarded Orphan Drug Designation (ODD) and Rare Paediatric Disease Designation (RPDD) by the US Food and Drug Administration (FDA) to treat paediatric rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in children and adolescents.

“Getting RPDD status for volasertib is an important milestone for Oncoheroes because it means we are eligible for a Priority Review Voucher (PRV) once the drug is approved. Companies holding a PRV can use it at any time to accelerate FDA review of one of their drug candidates, helping them reach the market faster”, explains Ricardo Garcia, Oncoheroes founder and CEO. “PRVs are only granted to companies developing drugs for rare or orphan diseases, like Oncoheroes is doing in paediatric oncology. However, companies that are granted with a PRV but do not want to use it are allowed to transfer it. Big pharma companies are very interested in getting them and are willing to pay. The average selling rate for a PRV is $100 million. Most of the new drugs Oncoheroes is planning to develop are eligible for a PRV”.

Oncoheroes has started manufacturing volasertib and is now working with the European Innovative Therapies for Children with Cancer (ITCC) Consortium to begin a phase Ib/II trial to treat paediatric rhabdomyosarcoma, sponsored by the University of Birmingham and Cancer Research UK, in 2022.

Committed to childhood cancer

Oncoheroes’ business model is to develop a portfolio of new drug candidates for childhood cancer using three strategies: proprietary R&D, in-licensing drugs in the clinical phase and partnerships with the pharmaceutical industry, academia, hospitals, foundations and patients’ associations.

“Childhood cancer is not like adult cancer; it requires a different treatment as some of the most devastating childhood cancers only occur in children. Unfortunately, in most cases, paediatric oncologists are forced to adapt adult treatments to the children. Currently, there are only five drugs available that have been developed and approved specifically for childhood cancer, despite being the number one cause of death by disease in children. Moreover, treating children with drugs that were not designed for them, results in more survivors living with lifelong side effects associated with drug toxicity, which in some cases can be very serious”, explains Cesare Spadoni, co-founder and COO of Oncoheroes.

Oncoheroes centralises its global R&D for drug discovery at its lab at the Barcelona Science Park. The centre is led by Dr. Eva Méndez, Oncoheroes’ Chief Discovery Officer. Dr. Mendezhas over 20 years of experience in academic and industrial biomedical research. “As far as we know, our lab is the only one in the industry dedicated 100% to discovering new targets, biomarkers and drug candidates for childhood cancer. In 2019, we launched our first drug discovery project, 2HIT-Medulloblastoma to treat paediatric medulloblastoma, the most common brain tumour in children. Our goal is to select and characterise a preclinical candidate that will be ready to enter the regulatory preclinical phase by the end of 2023. The lab was initially funded through Neotec, ENISA, Torres Quevedo and Industrial Doctorates non dilutive funds”, notes Dr. Eva Méndez.

Its determination and exclusive commitment to childhood cancer has led Oncoheroes to design a strategy that will allow the company to continue funding its growing portfolio by out-licensing some of its drug candidates for the adult market. “Volasertib’s out-licence for adults not only reaffirms our solely focus on childhood cancer but also validates one of the business model. The potential approval of volasertib for the adult cancer market by Notable would bring in revenue to Oncoheroes for milestones and royalties, which we can re-invest to develop new drugs for childhood cancer”, says Ricardo García.

To date, Oncoheroes has raised $8.8 million from private investors, foundations and governmental organisations. This capital has allowed the company to set up its drug discovery lab at the Barcelona Science Park and to continue advancing development of its first two assets (volasertib and the 2HIT Medulloblastoma project). “Furthermore”, adds Ricardo, “we are working to incorporate two assets that are closer to entering the clinical phase to complete our portfolio”.

Active role of foundations and affected families 

To date, $2.9 million of the capital raised by Oncoheroes comes from cancer foundations and/or families affected. “14 non-profit organisations have invested in our project. They are organizations that have supported childhood cancer research for the past 40 years with more than $300 million. For many of them, this is the first time they have invested in a biotech company. We are optimistic that with the progress we have shown so far, most will invest again, as we saw recently with the first childhood cancer foundation that invested in Oncoheroes, the Andrew McDonough B+ Foundation”, highlights the co-founder of Oncoheroes.

In September 2021, Oncoheroes announced an investment from Fundación Olivares, a non-profit organisation to fight childhood cancer based in Málaga (Spain). It was the first time this organisation decided to support childhood cancer research and invested in a biotech company.

“Before Fundación Olivares, Oncoheroes received investment from several US childhood cancer foundations, some considered global references in funding research. We’ve very excited to be working with the first Spanish childhood cancer organisation to join our project”, concludes Ricardo García.