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Company Overview

Innovation: Focused discovery, development and commercialization of better drugs for children and adolescents with cancer
Targeted Cancer(s): All types of pediatric cancer
Leadership: Ricardo Garcia, Co-founder and CEO; Cesare Spadoni, Ph.D., MBA, Co-founder and COO; Marc Goldberg, J.D., MBA, Co-founder; Marco Munoz, Co-founder (Click here to view our team)
Stage of Business: $3 million seed


Oncoheroes Biosciences is building a multi-asset pediatric drug portfolio by, over the short-term, in-licensing clinical or near-clinical stage assets and, over the long-term, identifying novel targets in its own labs for existing compounds and creating new intellectual property. The company leverages ecosystem partners to support asset selection, development and clinical trials. Its business model is the commercialization of pediatric oncology drugs through optimal pathways and out-licensing products with adult use.

Unmet Medical Need
For several types of pediatric cancers there has been no substantial improvement in cure rates over the past few decades, and too many children and adolescents still face a very grim prognosis.
Childhood cancers are rare diseases and industry has so far disregarded the development of specific pediatric cancer drugs. Due to insufficient specific therapies for children with cancer, pediatric oncologists are forced to adjust adult cancer treatments for childhood cancer.
However, the current regulatory landscape now supports the development of new therapies specifically for children with cancer. This includes such U.S. initiatives and laws as the Orphan Drug Designation Program, Pediatric Research Exclusivity Act, RACE for Children Act and Creating Hope Act, and a two-year market exclusivity extension to orphan-designated pediatric drugs in Europe.
Oncoheroes has emerged with an unprecedented combination of ingredients for success in pediatric oncology.
The Oncoheroes management team is formed by drug development and industry professionals in the U.S. and Europe, and the company has already established strong links with major international pediatric oncology networks.
The company has established a discovery laboratory in Barcelona and has operations there, elsewhere in Europe and in Boston.
The company has licensed from Boehringer Ingelheim its first asset: volasertib—a compound that has been shown to have enormous potential to treat rhabdomyosarcoma (RMS) in children.
Innovative Therapies for Children with Cancer, Europe’s primary body of key opinion leaders for pediatric oncology drug development, lauds volasertib’s potential.
Asset Profile & Development Plan
Mechanism of Action: A gene fusion between the PAX3 or PAX7 and FOXO1 genes is found in the majority of cases with alveolar RMS. Volasertib is a highly potent Polo-like kinase 1 (PLK1) inhibitor. PLK1 inhibition directly reduces the activity and stability of the PAX3/7- FOXO1 fusion protein. Potential biomarkers of efficacy within the PAX3/7-FOXO1 fusion positive cell line models have been identified. PLK1 inhibition is synergistic in combination with microtubule-destabilizing agents such as vincristine. This synergism has been demonstrated in vitro for both fusion gene positive and negative RMS.
Proof of Concept: Volasertib has been studied as single agent in Phase I in children with leukemia and refractory solid tumors. The recommended Phase 2 dose (RP2D) for children has been defined and lies above the adult RP2D. There is substantial preclinical evidence suggesting that volasertib might be an effective therapeutic agent against RMS.
Intellectual Property: Patent protection on volasertib through until at least 2027, and multiple additional assets are under assessment.
Clinical Development Plan: Ready for key go/no-go Phase Ib/II trial of volasertib for pediatric RMS patients in the fourth quarter of 2020.
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