Science Programs

Precision oncology underlies Rain’s developmental philosophy across all its pipeline programs.


Stating the Problem

Our lead product candidate, milademetan, is a small molecule, oral inhibitor of MDM2 and is being developed in patients with MDM2-dependent cancers. Historically, MDM2 inhibition has presented treatment challenges due to dose-limiting, on-target hematologic toxicities. We believe an MDM2-targeted therapy must possess certain pharmacological characteristics related to potency, pharmacokinetics and drug accumulation to allow for the design of an optimized dosing schedule. An optimized dosing schedule is intended to improve peak drug exposure leading to apoptosis and cell cycle arrest during the dosing period, while permitting hematopoietic precursor cell recovery during the dosing break, thereby minimizing hematologic toxicity.

The Rain Approach

Precision oncology underlies Rain’s developmental philosophy across all its pipeline programs.  Each program targets aberrant genetic or cell signaling networks in cancers with an appropriate clinical development strategy employing diagnostic assays to identify biomarkers predictive of efficacy.  We've learned from the past to prioritize pairing drug and patient. The precision strategy is aimed squarely at improving historical rates for program success by maximizing patient benefit.

Our approach leverages currently available tumor genetic testing strategies to enroll and treat patients with advanced cancer. We anticipate employing companion diagnostics and partnerships with industry-leading assay developers to identify patients based on molecular biomarkers indicating oncogene addiction and dependency on specific cellular signaling networks.

Milademetan focuses on MDM2-dependent, and p53 wild-type cancers, and may effectively treat up to50% of all cancers.

learn how milademetan woRKS

learn how milademetan woRKS

Rain has an exciting precision oncology pipeline.