Rain Therapeutics Inc., a privately-held, clinical stage biotechnology company focused on targeted therapies for patients with cancer, 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 CEO 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.”

A Potential Cancer Therapy

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.

Deal terms from the license agreement were not disclosed.