INV-9956 is an orally available, highly selective inhibitor of CYP11A1, an enzyme crucial for the biosynthesis of pregnenolone and other androgens. By inhibiting CYP11A1, INV-9956 effectively reduces androgen production, which is essential for the growth of prostate cancer cells, particularly in castration-resistant prostate cancer (CRPC) with drug-resistant androgen receptor (AR) mutations.

Preclinical studies have demonstrated that INV-9956 suppresses the proliferation of prostate cancer cell lines harboring major clinically acquired drug-resistant AR ligand-binding domain mutations, such as AR L702H. In vivo experiments showed that orally administered INV-9956 exhibited pronounced tumor inhibition superior to known competitors in a CRPC VCaP xenograft model. Additionally, INV-9956 displayed a favorable toxicity profile in 14-day repeated dosing studies in rats and dogs, supported by high selectivity to CYP11A1 over other CYP family members and no significant off-target activity.

Currently, INV-9956 is being evaluated in a Phase 1, multi-center, open-label clinical trial to assess its safety, pharmacokinetics, pharmacodynamics, and preliminary evidence of antitumor activity in adult patients with advanced metastatic castration-resistant prostate cancer (mCRPC). The study involves dose escalation to determine the recommended dose range and/or maximum tolerated dose, followed by dose expansion to further evaluate its therapeutic potential.

The promising preclinical profiles of INV-9956 suggest its potential in addressing unmet needs in CRPC treatments, particularly for patients with drug-resistant AR mutations, and warrant its clinical development.

The trial is currently in recruitment phase.

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