Rain Therapeutics Inc. (“Rain”), a privately-held, clinical stage biotechnology company focused on targeted therapies for patients with cancer, announced it has licensed worldwide rights to a clinical stage, small molecule, oral MDM2 inhibitor, DS-3032 (milademetan) from Daiichi Sankyo Company, Limited. Rain will re-designate the program as RAIN-32.

RAIN-32 is a potent and selective MDM2 inhibitor that has been evaluated in clinical trials for solid tumors and hematologic malignancies. The profile and dosing schedule of RAIN-32 may lend a differentiated tolerability profile, enabling longer-term therapy as compared to other MDM2 programs in development.

“We’re excited to add RAIN-32, a clinical program with the potential to significantly impact cancers characterized by MDM2 amplification or overexpression, to Rain’s pipeline of targeted cancer therapies,” said Avanish Vellanki, cofounder and chief executive officer of Rain Therapeutics. “Daiichi Sankyo has done extensive work that we intend to build upon as we implement a biomarker-driven strategy to complete RAIN-32’s development.”

MDM2 has emerged as a potentially valuable target for cancer therapies due to its inhibitory effects on p53, a critical tumor suppressor. Rain intends to evaluate RAIN-32 in multiple indications where patients demonstrate MDM2 gene amplification or overexpression, with an initial focus on liposarcoma, where two-thirds of patients display MDM2 amplification.

Robert Doebele, MD, PhD, cofounder of Rain Therapeutics, added, “We hope to pursue a rapid registrational path for RAIN-32 in a challenging tumor type that lacks effective therapies, and look forward to rationally developing RAIN-32 for additional oncology indications where MDM2 activity plays a central role.”

Rain anticipates the presentation of clinical data from the ongoing Daiichi Sankyo clinical trial in patients with advanced solid tumors at an upcoming medical conference.

Rain has licensed global rights for all indications of RAIN-32. Deal terms from the license agreement were not disclosed.

About RAIN-32

RAIN-32 has been evaluated in patients with various solid tumors, acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). RAIN-32 also has been evaluated in continuous and intermittent dose schedules that may offer a differentiated tolerability profile as compared to other MDM2 programs.

Two clinical studies for RAIN-32 are ongoing, including a study evaluating the safety and efficacy of RAIN-32 in patients with liposarcoma and in patients with FLT3-ITD AML treated with RAIN-32 and the FLT3 inhibitor, quizartinib. In addition, multiple investigator sponsored studies are being conducted by MD Anderson Cancer Center (MDACC) as well as National Cancer Center Hospital (NCCH) in Tokyo, Japan.

About Rain Therapeutics Inc.

Rain Therapeutics Inc. is a privately-held biotechnology company developing targeted therapies for patients with cancer. Rain is investigating tarloxotinib in a Phase 2 clinical trial, a hypoxia-activated prodrug of a potent pan-ErbB inhibitor as a treatment for non-small cell lung cancer patients with EGFR exon 20 insertions and HER2 mutations as well as any solid tumors with gene fusions involving NRG1, EGFR, HER2, and HER4. Rain Therapeutics Inc. has worldwide development and commercialization rights for tarloxotinib. Rain is also developing novel inhibitors of RAD52 in the DNA Damage Response (DDR) pathway as a synthetic lethal strategy in BRCA1/2-deficient tumors. For more information, visit www.rainthera.com