Sumitomo Dainippon Pharma Oncology Announces First Patient Dosed in Phase 1/2 Study of TP-0184 for the Treatment of Anemia in Patients with Low or Intermediate Risk Myelodysplastic Syndromes

CAMBRIDGE, Mass., Jan. 19, 2021 /PRNewswire/ -- Sumitomo Dainippon Pharma Oncology, Inc., a developer of novel cancer therapeutics, today announced that the first patient has been dosed in a Phase 1/2 study evaluating the investigational agent TP-0184, an activin receptor-like kinase 2 (ALK2) and ALK5 inhibitor, for the treatment of anemia in adult patients with low or intermediate risk myelodysplastic syndromes (MDS).

"The management of anemia in patients with MDS is challenging as this patient population tends to be older and often have non-hematological comorbidities which can make them relatively intolerant to therapy," said Patricia S. Andrews, Chief Executive Officer and Global Head of Oncology, Sumitomo Dainippon Pharma Oncology (SDP Oncology). "Given the high unmet medical need, we are looking forward to learning more about the potential of TP-0184 in this patient population."

The primary objectives of the Phase 1 part of the open-label, dose-escalation study are to assess the safety and tolerability of oral TP-0184 as well as determine the maximum tolerated dose (MTD) or maximum administered dose (MAD), and recommended dose for future expansion arms in the Phase 2 part of the study.

Following the completion of the Phase 1 portion of the study, the study will move into a Phase 2 expansion. The primary objectives of the Phase 2 portion of the study are to determine the effect of TP-0184 on the treatment of anemia in terms of hemoglobin increase, reduction in red blood cell transfusions and being red blood cell transfusion-free for at least eight weeks.

The trial is being conducted at sites in the United States. Additional information on this trial, including comprehensive inclusion and exclusion criteria, can be accessed at www.ClinicalTrials.gov (NCT04623996).

About TP-0184

TP-0184 is an activin receptor-like kinase 2 (ALK2) and ALK5 inhibitor. ALK2, also known as activin A receptor type 1 (ACVR1), is mutated in multiple types of cancers, including diffuse intrinsic pontine glioma (DIPG), a malignancy with high morbidity and mortality affecting the pediatric population.1,2,3 ALK2 is also involved in regulation of iron homeostasis, through stabilization of hepcidin and reduction of bioavailable iron, which is associated with anemia of chronic disease.1,4 TP-0184 also has activity against ALK5, a transforming growth factor beta (TGFβ) family member that is involved in myelodysplastic syndrome (MDS). TP-0184 is currently being evaluated in a Phase 1 clinical trial in advanced solid tumors (NCT03429218) and in a Phase 1/2 clinical trial in treatment of anemia associated with myelodysplastic syndromes (MDS) (NCT04623996).

About Sumitomo Dainippon Pharma Oncology

Sumitomo Dainippon Pharma Oncology, Inc., is a wholly owned subsidiary of Sumitomo Dainippon Pharma Co., Ltd. As a global oncology organization with teams in the U.S. and Japan, SDP Oncology is relentlessly committed to advancing purposeful science by transforming new discoveries into meaningful treatments for patients with cancer. The company's robust and diverse pipeline of preclinical and advanced-stage assets spans multiple areas, including oncogenic pathways, survival mechanisms and novel protein interactions, which aim to address unmet clinical needs in oncology.

For more information, visit www.sdponcology.com.

About Sumitomo Dainippon Pharma

Sumitomo Dainippon Pharma is among the top-10 listed pharmaceutical companies in Japan, operating globally in major pharmaceutical markets, including Japan, the U.S., China and the European Union. Sumitomo Dainippon Pharma aims to create innovative pharmaceutical products in the Psychiatry & Neurology area, the Oncology area and Regenerative medicine/Cell therapy field, which have been designated as the focus therapeutic areas. Sumitomo Dainippon Pharma is based on the merger in 2005 between Dainippon Pharmaceutical Co., Ltd., and Sumitomo Pharmaceuticals Co., Ltd. Today, Sumitomo Dainippon Pharma has more than 6,000 employees worldwide. Additional information about Sumitomo Dainippon Pharma is available through its corporate website at www.ds-pharma.com.

Disclaimer Regarding Forward-Looking Statements

This press release contains "forward-looking statements," as that term is defined in the Private Securities Litigation Reform Act of 1995 regarding the research, development and commercialization of pharmaceutical products. The forward-looking statements in this press release are based on management's assumptions and beliefs in light of information presently available and involve both known and unknown risks and uncertainties. Any forward-looking statements set forth in this press release speak only as of the date of this press release. We do not undertake to update any of these forward-looking statements to reflect events or circumstances that occur after the date hereof. Information concerning pharmaceuticals (including compounds under development) contained within this material is not intended as advertising or medical advice.

References

1. Peterson P, Kim W, Haws H, et al. The ALK-2 inhibitor, TP-0184, demonstrates high distribution to the liver contributing to significant preclinical efficacy in mouse models of anemia of chronic disease [Abstract]. Blood. 2016;128:263.

2. Taylor KR, Mackay A, Truffaux N, et al. Recurrent activating ACVR1 mutations in diffuse intrinsic pontine glioma. Nature Genetics. 2014; 46; 457–461. doi:10.1038/ng.2925

3. Wu G, Diaz AK, Paugh BS, et al. The genomic landscape of diffuse intrinsic pontine glioma and pediatric non-brainstem high-grade glioma. Nature Genetics. 2014;46(5):444-450. doi:10.1038/ng.2938.

4. Steinbicker AU, Bartnikas TB, Lohmeyer LK, et al. Perturbation of hepcidin expression by BMP type I receptor deletion induces iron overload in mice. Blood. 201; 118(15):4224–4230. doi:10.1182/blood-2011-03-339952.

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