Dovitinib, a potential new treatment for osteosarcoma, receives Orphan Drug Designation from the U.S. FDA

Boston, MA., Oct. 13, 2023. – Oncoheroes Biosciences, a biotech focused on advancing new therapies for childhood cancer, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designation (ODD) to dovitinib for its use in treating osteosarcoma.

 Key Points

• FDA awards Orphan Drug Designation to potential therapies addressing unmet needs of underserved patients with rare diseases.

• ODD may enable Oncoheroes to obtain partial tax credits for clinical trial expenditures.

• Dovitinib previously received Rare Pediatric Disease Designation by the FDA, which may also provide substantial financial incentives to Oncoheroes with the related Priority Review Voucher program.

The U.S. FDA has established initiatives aimed at incentivizing pharmaceutical companies to pursue drug development for rare diseases, which are characterized as conditions impacting fewer than 200,000 individuals in the U.S. at the time of designation. Following the enactment of the Orphan Drug Act in 1983, the FDA has granted Orphan Drug Designations (ODD) and subsequently approved the release of numerous drugs tailored to treat such rare diseases.

Under the Orphan Drug status, Oncoheroes will qualify for various development incentives, including a tax credit on expenditures incurred in clinical studies, exemption from filing fees such as the user fee, substantial cost savings, or eligibility for a research grant awarded by the FDA. Being granted orphan designation does not modify the standard regulatory requirements to obtain marketing approval.

“We are thrilled to have received another favorable notice from the FDA. This underscores the imperative demand for better treatments for children and adolescents with osteosarcomas,” stated Cesare Spadoni, Oncoheroes’ Founder and COO. “We anticipate that this excellent news will expedite the drug's clinical development, benefiting not just osteosarcoma patients but also other pediatric solid tumors given the design of dovitinib's Phase 1/2a clinical trial, which has been recently allowed to proceed by the FDA”.

In September 2022, dovitinib was also granted the Rare Pediatric Disease Designation (RPDD), making Oncoheroes eligible for a fast-track review and for a Priority Review Voucher (PRV) that is fully transferable to other sponsor companies and has a current average market value of $100 million.

Bone tumors are a group of histologically diverse diseases. Osteosarcoma is the most common cause of bone cancer in children and adolescents, associated with high treatment failure rates and morbidity. Annually, the United States sees 800-900 new osteosarcoma cases, and those that experience disease progression or recurrence have a low prognosis. The 5-year overall survival rates remain at approximately 20% for patients who develop metastatic disease.

About dovitinib:

Dovitinib is a pan-tyrosine kinase inhibitor (TKI) of multiple proteins known to be overexpressed in osteosarcomas and other bone sarcomas, including fibroblast growth factor receptor (FGFR), vascular endothelial growth factor receptor (VEGFR), and other receptor tyrosine kinases (RTKs). Oncoheroes acquired the pediatric exclusive license of dovitinib from Allarity Therapeutics in 2022. This drug class is considered interesting for the treatment of pediatric bone sarcomas, and the clinical development of dovitinib is supported by an exploratory biomarker that would allow the identification of potential responders to the drug. In September 2023, Oncoheroes received the green light from the FDA to move forward an Investigational New Drug (IND) application for dovitinib for the treatment of advanced or relapsed pediatric solid tumors, including osteosarcoma.

About Oncoheroes Biosciences:

Oncoheroes Biosciences is a Boston-based biotech company exclusively focused on the discovery and development of better drugs for children and adolescents with cancer. Our vision is to deliver benefits to young cancer patients and create value in the process. Oncoheroes is actively looking for in-licensing opportunities in the pediatric oncology space while working to generate new proprietary assets for a number of pediatric cancer indications with high unmet medical needs.

FDA Allows Oncoheroes to Begin Two Pediatric Oncology Clinical Trials

Boston, MA., October 2, 2023. – Oncoheroes Biosciences, Inc. (“Oncoheroes”) announced today that the U.S. Food and Drug Administration (FDA) has allowed the Boston-based biotech to proceed with the investigation of two potential drugs for pediatric oncology.

In August, Oncoheroes submitted two Investigational New Drug (IND) applications to the FDA, seeking permission to initiate clinical trials for two compounds in their portfolio: volasertib and dovitinib. After FDA’s thorough review and evaluation, Oncoheroes reported that both INDs have received the green light from the FDA to move forward, opening the door to innovative treatment options for pediatric cancer patients.

"Receiving these announcements in the final days of Childhood Cancer Awareness Month has been an extraordinary gift," remarked Ricardo Garcia, Co-founder and CEO of Oncoheroes. "Our ongoing mission is to reshape the landscape of pediatric oncology, and these milestones accomplished stand as a testament to the dedication and determination of the entire Oncoheroes team."

This achievement is not just a cause for celebration within the company but also a beacon of hope for the countless children and their families whose lives may be positively impacted by these potential drugs. 

Oncoheroes extended their gratitude to everyone who has contributed to this journey— team, consultants, collaborators, investors, supporters, and the patients and families who inspire them every day.

About volasertib:

Volasertib is an inhibitor of Polo-like-kinase 1 (PLK1), an enzyme known to be involved in disease progression in a number of cancers. Oncoheroes licensed the compound from Boehringer Ingelheim, which was developing it for the treatment of Acute Myeloid Leukemia until the company decided to discontinue the compound for strategic reasons. Preclinical data support further development of volasertib for rhabdomyosarcoma and other pediatric cancer indications. 

About dovitinib:

Dovitinib is a pan-tyrosine kinase inhibitor targeting fibroblast growth factor receptor (FGFR), vascular endothelial growth factor receptor (VEGFR), and other receptor tyrosine kinases (RTKs). Oncoheroes acquired a pediatric exclusive license of dovitinib from Allarity Therapeutics in 2022. This drug class is considered interesting for the treatment of pediatric bone sarcomas and the clinical development of dovitinib is supported by an exploratory biomarker that would allow the identification of potential responders to the drug.

About Oncoheroes:

Oncoheroes is a Boston-based biotech company exclusively focused on the discovery and development of better drugs for children and adolescents with cancer. Our vision is to deliver benefits to young cancer patients and create value in the process. Oncoheroes is actively looking for in-licensing opportunities in the pediatric oncology space while working to generate new proprietary assets for a number of pediatric cancer indications with high unmet medical needs.

FDA Grants Rare Pediatric Disease Designation (RPDD) to dovitinib for osteosarcoma

• Rare Pediatric Disease Designation qualifies Oncoheroes to receive fast track review, and a priority review voucher (PRV) at the time of marketing approval of dovitinib

• The PRV holder can benefit from an expedited six-month review of a new drug application for any disease by the FDA

• PRVs are transferable to other sponsor companies and have had a recent average selling price of $105 million

Boston, MA., September 22, 2022. – Oncoheroes Biosciences, Inc. (“Oncoheroes”) is pleased to announce that the United States Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to dovitinib, an investigational treatment for osteosarcoma.

The US FDA actively supports companies that develop drugs for rare diseases, defined as diseases affecting less than 200,000 Americans. One of the relevant programs created by the agency is for rare diseases primarily affecting individuals younger than 18 years old, called the Rare Pediatric Disease Designation (RPDD) which comes with the related priority review voucher opportunity.

Upon drug approval, the RPDD program may provide substantial financial incentives by making companies eligible for a Priority Review Voucher (PRV) that is fully transferable. The PRV grants accelerated FDA review of a drug candidate, for any indication, reducing the review period to 6 months and potentially gaining early market access. To date, 18 out of 34 PRVs received for pediatric indications have been sold for a cumulative sale price of USD 2.3 billion. 

Osteosarcoma (OS) is a rare disease and the most common cause of bone cancer in children and adolescents; OS is diagnosed in approximately 750-1,000 new individuals each year in the United States. About 450 are children or adolescents under the age of 20. The demonstration of utility of chemotherapy for all patients in the early 1980s led to the current survival rate of > 65% but this has not changed over the past three decades. The 5-year overall survival rates are approximately 20% for patients who develop metastatic disease. Additionally, of patients who experience disease progression or recurrence, less than 30% survive.  

“We are delighted that dovitinib has been granted RPDD by the FDA. This is an acknowledgement of the urgent need for better treatments for children and adolescents with osteosarcoma. We hope this fantastic news can speed up the drug’s clinical development process,” stated Ricardo Garcia, Oncoheroes’ Co-Founder and CEO.

Dovitinib is a pan-tyrosine kinase inhibitor targeting fibroblast growth factor receptor (FGFR), vascular endothelial growth factor receptor (VEGFR) and other receptor tyrosine kinases (RTKs). In January 2022, Oncoheroes signed an exclusive pediatric licensing agreement for dovitinib with Allarity Therapeutics, Inc. to continue the clinical development of this drug candidate for the benefit of younger cancer patients. Dovitinib will be developed together with Allarity’s DRP® companion diagnostics.

Several RTKs inhibited by dovitinib are involved in the growth, differentiation, and survival of different types of tumor cells as well as in tumor angiogenesis and development and maintenance of the tumor stroma microenvironment that is essential for proliferation of tumor cells and angiogenesis. Many RTKs must be inhibited simultaneously to generate a clinical response. Generally, the efficacy of tyrosine kinase inhibitors (TKIs) in the clinic depends on achieving a wide therapeutic index (vs off-tumor/on-target toxicity). Resistance can quickly occur to single TKI through mutations and therefore resistance is less likely to quickly develop with TKIs as these hit multiple targets simultaneously. Given dovitinib’s multi-target mechanism of action, the drug’s utility in pediatric osteosarcoma is promising.

“A major gap in the field, and one that continues to challenge the development of clinically effective TKIs in osteosarcoma, is the lack of reliable biomarkers”, said Cesare Spadoni, Oncoheroes’ Co-Founder and COO.  “Our partnership with Allarity brings the potential added value of further validating Allarity’s DRP® companion diagnostics for the selection of patients that are most likely to respond to dovitinib”.

 

About Oncoheroes Biosciences

Oncoheroes Biosciences is a ground-breaking biotech company exclusively focused on the discovery and development of better drugs for children and adolescents with cancer. Our vision is to deliver benefits to young cancer patients and create value in the process. The company is headquartered in Boston, US, with a discovery lab in Barcelona, Spain. Oncoheroes is actively looking for in-licensing opportunities in the pediatric oncology space while working to generate new proprietary assets for a number of pediatric cancer indications with high unmet medical needs.

For more information please visit: oncoheroes.com