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

Vall d’Hebron Research Institute (VHIR) and Oncoheroes Biosciences to Partner for the Discovery and Development of Novel Therapies for Childhood Sarcomas

BARCELONA, July 14th, 2022 – Vall d’Hebron Research Institute (VHIR), a world-leading healthcare institution where healthcare, research, innovation and education go hand in hand, and Oncoheroes Biosciences, a biotech company exclusively focused on advancing new therapies for children with cancer, today announced they have entered into a partnership to collaborate in the discovery and development of innovative therapies for pediatric sarcomas.

Survival rates in childhood cancer have improved considerably in recent decades with an overall survival close to 80% in developed countries. However, some of the most common solid tumors, such as soft tissue or bone sarcomas, still show an adverse prognosis in a high percentage of patients (nearly 40%). Sarcomas in children account for approximately 14% of cases and 20% of deaths in childhood cancers.

Oncoheroes Biosciences Discovery Lab, located at Barcelona Science Park and led by Dr. Eva Mendez, focuses on discovering and developing novel oncology drugs for those pediatric cancers with the highest unmet needs. “Pediatric sarcoma patients have not seen any significant improvement in treatment options in decades. We are excited to initiate this collaboration with Dr. Josep Roma, a recognized expert in this type of childhood cancer,” stated Dr. Eva Mendez, Oncoheroes’ Chief Discovery Officer. “We strongly believe that academic-industry collaborations are key to discover the new generation of drugs for childhood sarcomas.”

Dr. Roma is leading the Sarcoma Laboratory in the Childhood Cancer and Blood Disorders research group at VHIR. His current research focuses on discovering new therapeutic targets in rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in the pediatric population, by characterizing the role of embryonic pathways: Notch, Wnt, and Hedgehog. These pathways are essential during embryonic development and could have high oncogenic potential when an abnormal activation in childhood sarcomas is present.

Oncoheroes and VHIR’s collaboration will focus on the Hedgehog pathway, where recent studies led by Dr. Roma have identified novel targets for pediatric sarcomas with high therapeutic potential, not disclosed yet. The academic group has demonstrated that the inhibition of some specific targets results in killing selectively cancerous cells in both in vitro and in vivo models. Oncoheroes will rationally design new inhibitors and conduct high throughput screening campaigns for identifying new active compounds that will be later optimized in collaboration with Dr. Roma.

“The oncogenic mechanisms that we will evaluate in this collaboration are not exclusive to a single tumor type but are activated in a wide spectrum of sarcomas and other cancers,” added Dr. Josep Roma, VHIR’s researcher. “By combining the academic know-how generated at our research group, and the drug development and pediatric expertise from Oncoheroes’ team, we are confident we can accelerate the development of new targeted therapies for this underserved pediatric population”.

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

About Vall d’Hebron Research Institute

The Vall d'Hebron Research Institute (VHIR) is a public sector institution that promotes and develops biosanitary research, innovation and teaching at the Vall d'Hebron University Hospital, aimed at finding solutions to the health problems of citizens and with the desire to contribute to the scientific, educational, social and economic development of its area of competence. Founded more than 25 years ago, the VHIR is part of the Vall d’Hebron Barcelona Hospital Campus, the Catalan Institute of Health (ICS), and is a CERCA center of the Department of Economy and Knowledge of the Generalitat de Catalunya, as well as a University Research Institute from to the UAB.

Oncoheroes Biosciences and Allarity Therapeutics Sign Exclusive Pediatric Licensing Agreement for Dovitinib and Stenoparib

  • Oncoheroes will lead the pediatric clinical development of dovitinib and stenoparib in childhood cancers, utilizing Allarity’s DRP® companion diagnostics

  • Oncoheroes is Allarity’s partner of choice to fulfill FDA’s requirements mandated by the RACE for Children Act

  • The RACE for Children Act authorizes the FDA to require pediatric clinical studies when a molecular target of an adult cancer drug is relevant to a children’s cancer

Boston, MA., January 3, 2022 —Oncoheroes Biosciences, Inc. (“Oncoheroes”), and Allarity Therapeutics, Inc. (Nasdaq: ALLR) (“Allarity”) have signed a worldwide, exclusive pediatric licensing agreement for dovitinib, a pan-targeted kinase inhibitor (pan-TKI), and stenoparib, a PARP inhibitor, both being developed by Allarity.

Under the terms of the licensing agreements, Oncoheroes acquires global, exclusive rights to both clinical-stage therapeutic compounds for pediatric cancer clinical development. Allarity will support Oncoheroes’ pediatric clinical trials by providing access to its DRP® companion diagnostic for the selection of patients that are most likely to respond to both drugs. Financial terms of the licenses were not disclosed.

“Oncoheroes is a leader in pediatric oncology and Allarity’s ideal pediatric-focused partner to advance both dovitinib and stenoparib as potential new therapeutic options for children and adolescents with cancer,” explained Steve R. Carchedi, CEO of Allarity Therapeutics. “We are very pleased to support Oncoheroes’ pediatric clinical development of our therapeutic candidates together with our DRP® companion diagnostics, in a true personalized cancer care approach.”

 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). The drug was originally licensed by Allarity from Novartis and is currently under NDA submission to the U.S. FDA  for treatment of renal cell carcinoma.

“This agreement fits very well with the requirements set forth in the Research and Accelerate Cures and Equity (RACE) for Children Act, requiring companies to test adult oncology drugs in pediatric patients too, when there is a scientific rationale,” said Ricardo Garcia, Oncoheroes’ co-founder and CEO. “ Allarity’s drug candidate dovitinib, initially developed for renal cell carcinoma, might provide significant benefit to osteosarcoma pediatric patients and Oncoheroes is excited to make this happen”.

 The use of dovitinib in osteosarcoma patients is based on the results of preclinical studies suggesting a significant role of dovitinib in preventing metastases formation in the lungs. More in general, the potential of this class of multi-tyrosine kinase inhibitors for pediatric bone sarcomas, is suggested by several clinical studies demonstrating significant responses in subsets of patients.

Stenoparib is a unique, small molecule dual targeted inhibitor of Poly ADP-Ribose Polymerases (PARP 1 and 2) and telomerase maintenance enzymes (Tankyrase 1 and 2), currently in a Phase 2 clinical trial for advanced ovarian cancer at Dana-Farber Cancer Institute (Boston, MA).

“Previous preclinical and clinical studies have demonstrated the potential of PARP inhibitors in pediatric oncology. We are excited to bring this class of molecules into Oncoheroes’ portfolio,” explained Cesare Spadoni, Oncoheroes’ co-founder and COO.  “Stenoparib has the potential to be tested across many different pediatric cancers, including brain tumors. More importantly, for both licensed molecules, we would have a powerful tool in the DRP® companion diagnostic to select potential responders.”

 

About Oncoheroes Biosciences

Oncoheroes 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

 

About Allarity Therapeutics

Allarity Therapeutics, Inc. (Nasdaq: ALLR) develops drugs for personalized treatment of cancer guided by its proprietary and highly validated companion diagnostic technology, the DRP® platform. The Company has a mature portfolio of five drug candidates, including: Stenoparib, a PARP inhibitor in Phase 2 development for ovarian cancer; Dovitinib, a pan-TKI submitted for NDA review by the FDA for 3rd line renal cell carcinoma; IXEMPRA® (Ixabepilone), a microtubule inhibitor approved in the U.S. for the treatment of 2nd line metastatic breast cancer and in Phase 2 development, in Europe, for the treatment of the same indication; LiPlaCis®, a liposomal formulation of cisplatin in Phase 2 development for metastatic breast cancer;  and 2X-111, a liposomal formulation of doxorubicin in Phase 2 development for metastatic breast cancer and/or glioblastoma multiforme (GBM).  The LiPlaCis® and 2X-111 programs are partnered, via out-license, to Smerud Medical Research International AS.  In 2021, Allarity sold the global rights to Irofulven, a DNA-damaging agent in Phase 2 for prostate cancer, back to Lantern Pharma, Inc. The Company maintains an R&D facility in Hoersholm, Denmark.


For more information: allarity.com

Oncoheroes Biosciences Inc. Out-licences volasertib to Notable Labs for the Global Adult Development of Its Anticancer Therapy

  • Agreement enables parallel development of volasertib for the benefit of all patients

  • Oncoheroes will continue the development of volasertib in childhood cancers while Notable will focus on the adult market using its high-fidelity Predictive Precision Medicine Platform

  • Volasertib is a PLK-1 inhibitor with therapeutic potential across a range of tumor types

Boston, MA, Nov, 11th, 2021 – Oncoheroes Biosciences Inc. (“Oncoheroes”), a biotech focused on advancing new therapies for childhood cancer, has out-licensed worldwide the adult rights of volasertib to Notable Labs, Inc. (“Notable”), a clinical-stage predictive precision medicine company. Under the terms of this agreement, Oncoheroes will retain the license for the development and commercialization of volasertib for pediatric cancers and continue its commitment towards childhood cancer.  Notable will have exclusive rights to develop and commercialize volasertib in adult cancer indications. Financial details of this deal are not disclosed.

Volasertib is a Polo-like kinase 1 (PLK-1) inhibitor with evidence of clinical activity in relapsed/refractory acute myeloid leukemia (AML). PLK-1 overexpression has been shown to occur in a wide range of both solid tumors and hematological cancers. Volasertib was previously granted Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the US Food and Drug Administration (FDA) for its use in treating pediatric rhabdomyosarcoma (RMS), a soft tissue sarcoma. Oncoheroes is planning to take volasertib into the clinic with a Phase Ib/II clinical trial for the treatment of RMS.

Notable will leverage its high-fidelity Predictive Precision Medicine Platform to identify and select volasertib-responsive patients prior to their treatment and fast-track volasertib’s clinical development with phase 2/3 clinical trials in AML and other cancers.

 “In-licensing clinical-stage therapies that pass our platform’s rigorous demands like volasertib represents an exciting milestone towards fulfilling Notable’s mission to deliver life-changing therapies precisely to those who will benefit,” said Thomas Bock, M.D., CEO of Notable. “We hope that this agreement will also contribute and accelerate Oncoheroes’ mission”.

 “As a company solely committed to childhood cancer, we do not want to deviate our attention from developing new therapies for pediatric oncology. However, we are delighted about this out-licensing agreement as other patients can also benefit from our drug candidates,” said Ricardo Garcia, Oncoheroes’ co-founder and CEO. “Notable’s predictive precision medicine approach can revolutionize the traditional clinical development process and we are excited that volasertib will be one of their first assets to be assessed”.

About Oncoheroes Biosciences

Oncoheroes is a ground-breaking biotech company exclusively focused on the discovery and development of better drugs for children and adolescents with cancer. Oncoheroes’ 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

 

About Notable

Notable is a clinical-stage platform therapeutic company developing a new class of precision medicines: predictive precision medicines. Notable aims to be the leader in predicative precision medicine and revolutionize the way in which patients seek and receive treatment: patient population by patient population, and cancer by cancer. By transforming historical standards of care, Notable expects to provide dramatic impact for patients and the healthcare community.

For more information please visit: notablelabs.com

SHEPHERD Therapeutics and Oncoheroes Biosciences to Partner for the Discovery, Development, and Commercialization of Oncology Therapeutics For Rare Cancers

20210210_ShepherdPartnership_Wide.jpg

BOSTON, Feb 15th, 2021 – SHEPHERD Therapeutics, a company dedicated to catalyzing lifesaving treatments for rare cancer patients, and Oncoheroes Biosciences, a biotech company focused on advancing new therapies for childhood cancer, today announced they have entered into a partnership to collaborate in the discovery, development and commercialization of innovative drug products for rare oncology indications.

Out of 400 known distinct cancers, 380 are considered rare according to the American Cancer Society’s metric of an incidence of less than 6 per 100,000. Rare cancers affect almost one in three new patients, or over half a million Americans each year. Despite the prevalence of rare cancers, 2012-2016 data shows that 75% of all clinical trials did not specifically include even one rare cancer by name.

Childhood cancer is considered a rare disease that accounts for about 1% of all U.S. cancer diagnoses. Every year, cancer takes the lives of 90,000 children and adolescents worldwide, despite treatment advances in recent decades. Although cancer is the leading cause of death by disease among children in the United States, pediatric oncology treatments are lacking: Only five pediatric-specific drugs have been granted U.S. Food and Drug Administration (FDA) approval in the last 20 years versus more than 200 for adults.

Oncoheroes and SHEPHERD’s collaboration advances a paradigm change in drug development for rare cancers. The companies will use DELVE, SHEPHERD’s next-generation, precision-oncology platform that integrates bioinformatics, machine learning and mathematics, to unveil unprecedented insights into rare cancers, including childhood cancer. DELVE enables the companies to move beyond a single target-based approach to identify all of the primary mechanisms of action responsible for drug response and resistance within the human transcriptome.

The companies’ first project of the collaboration will identify additional pediatric and rare adult indications for Oncoheroes’ lead asset, 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. Volasertib has recently been granted Rare Pediatric Disease Designation (RPDD) and Orphan Drug Designation (ODD) by the FDA for the treatment of rhabdomyosarcoma, the most common soft tissue sarcoma in children and young adults, representing 3-4% of all childhood cancers.

“Getting a diagnosis of cancer as a child should not be a death sentence, yet too many lives are being lost or diminished because of a dearth of pediatric cancer research and treatments,” said David Hysong, CEO of SHEPHERD. “SHEPHERD and Oncoheroes are coming together in hope and in action, leveraging the full extent of our combined technology, experience, and expertise to help save lives.”

“We are excited about this partnership between two mission-driven companies determined to address high unmet medical needs in the rare oncology space. We are confident that SHEPHERD’s technology, combined with Oncoheroes’ expertise in the pediatric oncology space, will deliver new therapeutics options to children and adolescents with cancer,” stated Ricardo Garcia, CEO of Oncoheroes.

 

About Oncoheroes Biosciences

Oncoheroes 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

 

About DELVE

DELVE is SHEPHERD’s next-generation, precision-oncology platform that integrates bioinformatics, machine learning, and mathematics to unveil unprecedented insights into cancer. DELVE moves beyond a single-target-based approach to identify complex and interconnected mechanisms responsible for drug response and resistance revealed in the human transcriptome. Using DELVE, SHEPHERD can identify the cancers for which each therapy potentially will work best – and the therapies that will work best for each cancer. This approach enables SHEPHERD to work faster and differently across over 100 cancers, multiplying the potential patient impact for any therapy, speeding development, and improving patient outcomes by optimizing for maximum clinical trial success.

 

About SHEPHERD

SHEPHERD is on a mission to change the way rare cancer is treated. Through SHEPHERD Therapeutics and the SHEPHERD Foundation, we attack rare cancer from every angle, with the goal of saving millions of lives. SHEPHERD Therapeutics pursues drug development differently than any other company: Using our precision-oncology platform, DELVE, we are moving beyond a single-target-based approach to identify complex and interconnected mechanisms responsible for drug response and resistance revealed in the human transcriptome, which inform the identification of promising potential treatments for specific cancers. The SHEPHERD Foundation is a non-profit fighting to generate industry-wide change related to rare cancer awareness and therapeutic availability. The Foundation drives transformative healthcare policy at the federal level, connects and advocates for rare cancer patients and their families, and ultimately aims to accelerate the availability of and access to rare cancer-related data, basic science and therapies – to ensure that no patient is left to die. Visit www.SHEPHERD.bio to learn more and join the fight.

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

Boston, Oct. 14, 2020. – 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 volasertib for its use in treating pediatric rhabdomyosarcoma and other rare soft tissue sarcomas

Key Points

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

  • ODD may enable Oncoheroes to retain seven years of U.S. market exclusivity upon marketing approval and obtain partial tax credits for clinical trial expenditures.

  • Volasertib 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 programs to encourage companies to develop drugs for rare diseases, defined as diseases affecting 200,000 people in the U.S. at the time of designation. Since the Orphan Drug Act was signed into law in 1983, the FDA has granted Orphan Drug Designations (ODD) and later approved hundreds of drugs for rare diseases.

“All the ODD-related incentives will help us to accelerate the clinical development and commercialization of our first asset volasertib, an investigational treatment for rhabdomyosarcoma. Each potential new therapy for rhabdomyosarcoma gives fresh hope to the around 450 American families diagnosed each year with this type of childhood cancer,” stated Ricardo Garcia, Oncoheroes’ Founder, and CEO.

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, a cost savings of around $2.9M in 2020, eligibility for a research grant awarded by the FDA, and, most importantly, seven years of U.S. market exclusivity upon approval. Being granted orphan designation does not modify the standard regulatory requirements to obtain marketing approval.

“The fact that volasertib was granted FDA’s Rare Pediatric Disease Designation last month, and now, Orphan Drug Designation speaks to the unmet medical need for better treatments for patients with rhabdomyosarcoma and the strength of the translational data package supporting volasertib for rhabdomyosarcoma,” stated Cesare Spadoni, Oncoheroes’ Founder and COO.

In September 2020, volasertib was also granted with 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 historically have had a selling price range of USD 67 to 350 million.

Around 450 new patients each year in the U.S. are diagnosed with rhabdomyosarcoma (RMS), the most common soft tissue sarcoma in children and young adults, representing around 3% of childhood cancers. It is an aggressive and highly malignant form of cancer that develops from skeletal muscle cells that have failed to fully differentiate. Despite efforts to improve treatment, survival rates have only improved in the medium or low-risk groups. In contrast, over the past 25 years, a threshold was reached with no improvement in survival for high-risk patients. Therefore, there is a clear unmet medical need to treat the most aggressive forms of this disease.

Volasertib is an inhibitor of Polo-like-kinase 1 (PLK1), an enzyme known to be involved in disease progression in a number of cancers. The compound was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid Leukemia, until the company decided to discontinue the compound for strategic reasons. Meanwhile, independent academic groups generated strong data in support of further development of volasertib for rhabdomyosarcoma and, possibly, a few other pediatric cancer indications. In 2019, Oncoheroes in-licensed volasertib from Boehringer Ingelheim to continue the clinical development of this drug candidate for the benefit of younger cancer patients.

Preclinical research in rhabdomyosarcoma

A number of publications highlight the potential of volasertib in rhabdomyosarcoma. High PLK1 expression has been associated with poor prognosis in a number of cancers, including rhabdomyosarcoma. It was shown that the drug may have a specific anti-cancer effect in this disease, which is driven by the PAX3-FOXO1 fusion protein in a large subset of patients. PAX3-FOXO1 is a challenging drug target. However, it was shown that PLK1 inhibition by volasertib reduces the stability of this fusion protein leading to its degradation and cancer growth inhibition in PDX models. Interestingly, in vivo data also point to a strong synergy between volasertib and vincristine, a drug already in the standard treatment protocol for rhabdomyosarcoma. Most of these data were generated by European laboratories that are part of the Innovative Therapies for Children with Cancer (ITCC) consortium. Oncoheroes is planning to collaborate with ITCC for the clinical development of volasertib.

About Oncoheroes Biosciences Inc.

Oncoheroes 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

 

FDA Grants Rare Pediatric Disease Designation (RPDD) to volasertib for rhabdomyosarcoma

Oncoheroes Biosciences, a biotech focused on advancing new therapies for childhood cancer, is pleased to announce that the United States Food and Drug Administration (FDA) has granted the designation of rare pediatric disease to volasertib, an investigational treatment for rhabdomyosarcoma.

 Key Points

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

  • PRV holders 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 historically have had a selling price range of USD 67 to 350 million.

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

“We are delighted that volasertib has been awarded with RPDD by the FDA. This recognition acknowledges the unmet medical need for better treatments for children and adolescents with rhabdomyosarcoma. We hope this big news will allow Oncoheroes to speed up the drug development process of volasertib,” stated Ricardo Garcia, Oncoheroes’ Founder and CEO. 

Upon drug approval, the RPDD 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, 12 out of 25 PRVs received for pediatric indications have been sold for a cumulative sale price of USD 1.6 billion. 

“Current rhabdomyosarcoma treatments are based on decades-old therapies and generally lack efficacy against the most aggressive subtypes of the disease, for which the 5-year survival rate is currently 20-30%. We are excited about upcoming clinical studies and we hope that volasertib could be a game-changer for rhabdomyosarcoma patients,” explained Cesare Spadoni, PhD, Oncoheroes’ Founder and COO.

 Around 500 new patients each year in the US are diagnosed with rhabdomyosarcoma, an aggressive and highly malignant form of cancer (soft tissue sarcoma) that develops from skeletal muscle cells that have failed to fully differentiate. There is a clear unmet medical need for the treatment of the most aggressive forms of this disease. 

Volasertib is an inhibitor of Polo-like-kinase 1 (PLK1), an enzyme known to be involved in disease progression in a number of cancers. The compound was originally discovered and developed by Boehringer Ingelheim for the treatment of Acute Myeloid Leukemia until the company decided to discontinue the compound for strategic reasons. Meanwhile, independent academic groups generated strong data in support of further development of volasertib for rhabdomyosarcoma and, possibly, a few other pediatric cancer indications. In 2019, Oncoheroes in-licensed volasertib from Boehringer Ingelheim to continue the clinical development of this drug candidate for the benefit of younger cancer patients.

Preclinical research in Rhabdomyosarcoma

A number of publications highlight the potential of volasertib in rhabdomyosarcoma. High PLK1 expression has been associated with poor prognosis in a number of cancers, including rhabdomyosarcoma. It was shown that the drug may have a specific anti-cancer effect in this disease, which is driven by the PAX3-FOXO1 fusion protein in a large subset of patients. PAX3-FOXO1 is a challenging drug target. However, it was shown that PLK1 inhibition by volasertib reduces the stability of this fusion protein leading to its degradation and cancer growth inhibition in PDX models. Interestingly, in vivo data also point to a strong synergy between volasertib and vincristine, a drug already in the standard treatment protocol for rhabdomyosarcoma. Most of these data were generated by European laboratories that are part of the Innovative Therapies for Children with Cancer (ITCC) consortium. Oncoheroes is planning to collaborate with ITCC for the clinical development of volasertib.

About Oncoheroes Biosciences Inc.

Oncoheroes 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

 

Dreamers Startup Ventures signs an agreement to invest up to $1.1M in Oncoheroes Biosciences.

Oncoheroes Biosciences Inc. (“Oncoheroes”), a biotech company exclusively focused on the development of innovative medicines to treat cancer in children and adolescents, today announced that it has closed the first investment with Dreamers Startup Ventures. Under the terms of the agreement, Dreamers Startup Ventures has committed to invest up to $1.1M, disbursed in installments subject to Oncoheroes milestone achievements in the coming months.

The agreement goes beyond a traditional VC investment. Ricardo Garcia, Oncoheroes’ CEO, stated: “This is a great opportunity for Oncoheroes to take advantage of Dreamers Startup Ventures hands-on approach to support our efforts in all directions. We feel honored and acknowledge the responsibility of being the first investment of Dreamers Startup Ventures in the biotech sector.”

Cesare Spadoni, Oncoheroes' Chief Operations Officer added: “This funding is an important financial achievement for Oncoheroes and enables us to focus on continue working on the clinical development plan for volasertib, our first asset in-licensed from Boehringer Ingelheim, and identify two new assets and complete our pipeline”.

Gorka Fius, cofounder of Dreamers Startup Ventures further notes: “From the beginning, we realized that Oncoheroes holds great potential as a biotech company, which made it a very strategic investment for us. The first reason that led us to invest in Oncoheroes is the uniqueness of being 100% focused on developing drugs for pediatric cancer, an estimated niche market of $5-10B/year. We also value the multi-asset portfolio strategy that contributes to diversifying our investment risk and the fact they already have volasertib, a very strong drug candidate in their pipeline. Moreover, Oncoheroes' first-class team has wide expertise over all of the drug development spectrum, a clear strategy, and a very well-defined road map. Finally, we are confident that we are diminishing the risk of our first operation in the biotech sector by investing in the rare disease space, where normally the cost to develop a drug is lower and the time to reach the market is faster.”

About Oncoheroes Biosciences Inc.

Oncoheroes 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. The company was co-founded by Ricardo Garcia (serial entrepreneur), Cesare Spadoni (drug development professional), Marc Goldberg (savvy Life Science VC investor) and Marco Muñoz (seasoned fundraiser). The management team is formed by drug development and industry professionals in the US and Europe and the company has already established strong links with the major international pediatric oncology network as well as the childhood cancer community at large.

For more information please visit: oncoheroes.com


About Dreamers Startup Ventures

Dreamers Startup Ventures, based in Bilbao (Spain) and Boston (USA), is led by entrepreneurs Jose Antonio Borge, Eneko Knörr and Gorka Fius, as well as by a group of private investors such us Albia Capital who complete a team with extended experience on inception, management, transformation, acceleration and exit of companies. They support startups in early-stage with global ambition and seek to position themselves in markets in a competitive way. With the participation in technology companies both in Spain and the USA, Dreamers Startup Ventures is positioned as a leader in establishing connections between startups on both sides of the Atlantic, facilitating its rapid growth and completing its strategic plan for future investment.

For more information please visit: http://dreamers-vc.com/