The startup Oncoheroes Biosciences , focused exclusively on the advancement of new therapies for childhood cancer, has licensed the rights to develop and commercialize volasertib for adult cancer worldwide to the North American biopharmaceutical company Notable Labs, a pioneering biopharmaceutical company in preventive and precision medicine. Oncoheroes - which will retain the global license in pediatric oncology - was co-founded in 2017 by Catalan entrepreneur Ricardo García and Italian scientist Cesare Spadoni, two parents affected by childhood cancer. The company is headquartered in Boston (United States) and centralizes all its drug discovery activity in its laboratory at the Barcelona Science Park (PCB).
Original News: https://www.pcb.ub.edu/ (18/10/2021)
Volasertib is an inhibitor of the PLK-1 kinase, an enzyme involved in a wide range of solid tumors and hematological cancers. The compound was discovered and developed by Boehringer Ingelheim for acute myeloid leukemia, until the company decided to stop its development for strategic reasons, after a phase III study in adults failed to meet its expectations.
In 2019, Oncoheroes obtained the exclusive global license for the drug with the aim of continuing clinical development in younger oncology patients and, in 2020, it achieved Orphan Drug Designation ( ODD) and Rare Pediatric Disease Designation (RPDD) designation by the Food and Drug Administration (FDA) for the treatment of pediatric rhabdomyosarcoma (RMS), the most common soft tissue cancer in children and adolescents.
“The granting of RPDD status to volasertib is an important milestone for Oncoheroes, as it allows the possibility of obtaining a Priority Review Voucher (PRV) at the time of its approval. Companies holding the PRV can use it at any time to reduce the time for the FDA to review one of their candidates to become a new drug, allowing them to come to market earlier,” explains Ricardo García , co-founder and CEO of Oncoheroes. “The PRV is only granted to companies that are developing drugs for rare or orphan diseases, as is the case of Oncoheroes with childhood cancer. However, the companies that receive a PRV are not interested in using it, but in selling it, since the FDA allows us to transfer it and the big pharmaceutical companies are very interested in getting it and are willing to pay for it. The current market value of the PRV is $100M. Most of the new drugs that Oncoheroes plans to develop are eligible to receive a PRV.”
Oncoheroes has already started the production of volasertib and is working with the European consortium ITCC (Innovative Therapies for Children with Cancer) with the aim of starting a phase Ib/II clinical trial in pediatric rhabdomyosarcoma in 2022, sponsored by the University of Birmingham and Cancer Research UK.
Committed to childhood cancer
Oncoheroes' business model is to develop a portfolio of potential new drugs for childhood cancer, through three strategies: in-house R&D, licensing of drugs in the clinical phase and collaborations with the pharmaceutical industry, academia, hospitals, foundations and patient associations.
“Childhood cancer is not like adult cancer and requires different treatment. So much so that some of the most devastating childhood cancers only occur in children. Unfortunately, in most cases, pediatric oncologists are forced to adjust adult treatments for them. Today, only 5 drugs are available specifically developed and approved for childhood cancer, despite it remaining the leading cause of death from disease in children. Furthermore, treating children with drugs that have not been designed for them means that a large percentage of survivors will experience lifelong side effects associated with toxicity, which, in some cases, can be very serious,” explains Cesare Spadoni, co-founder and COO of Oncoheroes.
Oncoheroes centralizes its global R&D activity in drug discovery in its laboratory in the Barcelona Science Park. The center is led by Dr. Eva Méndez, current director of this area of the company, who has more than 20 years of experience in academic and industrial biomedical research. “To our knowledge, our laboratory is the only one in the industry dedicated 100% to the discovery of new targets, biomarkers and drug candidates for childhood cancer. In 2019 we launched our first drug discovery project, 2HIT-Medulloblastoma for the treatment of pediatric medulloblastoma, the most common brain tumor in children. Our goal is to select and characterize a candidate that is ready to enter the regulatory preclinical phase by the end of 2023. The laboratory has been funded in part with a Neotec, an Eninsa, a Torres Quevedo and two industrial doctorates, one from the Generalitat de Catalunya and another from the Ministry of Science and Innovation”, notes Dr. Méndez.
The determination and exclusive commitment to childhood cancer has also led Oncoheroes to design a strategy that allows it to continue financing the construction of its portfolio through licensing agreements for some of its potential drugs for the adult market. “The license of volasertib for its development in adults not only reaffirms our 100% focus on childhood cancer, but also validates one of the scenarios foreseen in our business model. The possible approval of volasertib for the adult cancer market by Notable will provide Oncoheroes with revenue from milestones and royalties to continue investing in the development of new drugs for pediatric cancer”, states Ricardo García.
To date, Oncoheroes has raised around $8.8 million from private investors, foundations and government agencies. This capital has allowed them to create the drug discovery laboratory at the Barcelona Science Park and to advance the development of their first two assets (volasertib and the 2HIT Medulloblastoma project). “On the other hand - adds Ricardo - we are in the process of incorporating two more assets ready to enter the clinical phase and thus complete our portfolio”.
The active role of foundations and affected families
Of the capital raised so far by Oncoheroes, $2.9M has come from cancer foundations and affected families. “14 non-profit organizations have already invested in our project. They are entities that have supported childhood cancer research over the last 40 years with more than $300M. For many of them, this is the first time they have invested in a biotech. It is likely that after the progress we have shown so far, most of them will invest again, as the first childhood cancer foundation to invest in Oncoheroes, The Andrew McDonough B+ Foundation , has recently done,” emphasizes the Oncoheroes co-founder.
In September 2021, Oncoheroes announced the investment of the Olivares Foundation , a non-profit organization against childhood cancer in Malaga (Spain). It was the first time that this organization took a step forward by supporting pediatric cancer research and investing in a biotechnology company. “Until the arrival of the Olivares Foundation, Oncoheroes had received investment from several North American childhood cancer foundations, some of them considered world leaders in financing research projects. We are very excited to also have the first Spanish childhood cancer organization joining our project”, concludes Ricardo García.
