Pipeline

We are advancing three clinic-ready compounds to be tested exclusively in pediatric cancers. Our focus is simple and unwavering: accelerate the development of new therapies for children who have been left behind by traditional drug models.

Volasertib

Volasertib is a selective Polo-like kinase 1 (PLK1) inhibitor.

Originally developed by Boehringer Ingelheim for the treatment of acute myeloid leukemia (AML), the compound advanced to Phase III clinical trials but was later discontinued after results did not meet expectations.
As part of its pediatric regulatory obligations, Boehringer Ingelheim conducted Phase I trials in children with leukemia and solid tumors, demonstrating tolerability, and defining dosing and pharmacokinetics.
Following this work, Boehringer transferred full patent rights to Oncoheroes Biosciences, granting worldwide exclusivity for all indications.
Oncoheroes is now preparing to initiate a signal-generating Phase I/II multi-arm clinical trial to further explore Volasertib’s potential across several pediatric cancer types.
Rhabdomyosarcoma, Ewing sarcoma, medulloblas!oma, neuroblastoma, and (DMG-DIPG).
Volasertib was granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD).

Dovitinib

Dovitinib is a multi–tyrosine kinase inhibitor (TKI), a small molecule that targets FGFR, VEGFR, and other receptor tyrosine kinases (RTKs) involved in tumor growth and angiogenesis.
The compound was originally developed by Novartis and later in-licensed by Allarity Therapeutics. After Novartis concluded its licensing agreement with Allarity, it granted Oncoheroes Biosciences the worldwide pediatric license for Dovitinib.
The U.S. FDA’s “Relevant Pediatric Molecular Target List” (July 19, 2018) identifies FGFR as a molecular target implicated in several pediatric cancers, supporting Dovitinib’s potential clinical relevance in this population.
Novartis previously demonstrated clinical activity of Dovitinib in Phase III trials for renal cell carcinoma (RCC) and Phase II trials for other malignancies, including GIST, hepatocellular carcinoma (HCC), breast cancer, and endometrial cancer.
Oncoheroes is now preparing a biomarker-supported Phase I/II pediatric study to evaluate Dovitinib’s safety and efficacy across selected childhood cancers.
Osteosarcoma (OS) and other pediatric bone sarcomas.
Dovitinib was granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD).

Stenoparib

Stenoparib is a first-in-class, small molecule inhibitor that targets key DNA damage repair enzymes (PARP) and telomerase maintenance enzymes (Tankyrase)—a dual mechanism designed to enhance efficacy, reduce myelotoxicity, and minimize drug resistance.
The compound was originally developed by Eisai and later licensed to Allarity Therapeutics, which subsequently granted Oncoheroes Biosciences the worldwide pediatric license.
Stenoparib has demonstrated clinical activity and safety in adults, completing a Phase I study in solid tumors with a recommended Phase II dose (RP2D) of 600 mg/day and an overall response rate of 4.9%. A Phase II trial in ovarian cancer is currently ongoing.
Validated through Drug Response Predictor (DRP®) technology in both ovarian and pancreatic cancers, Stenoparib shows broad potential as a monotherapy or in combination with immuno-oncology or chemotherapy agents, supported by its favorable safety profile and absence of myelosuppression at clinically relevant doses.
These characteristics make Stenoparib a strong candidate for biomarker-driven pediatric trials across multiple cancer indications.
PARP inhibitors are relevant agents in various pediatric cancers, such as advanced neuroblastoma, Ewing sarcoma and osteosarcoma. Pre-clinical work will be conducted to select the primary pediatric.
In progress.