Black Diamond Therapeutics (BDTX) H.C. Wainwright 28th Annual Global Investment Conference summary

Pipeline and clinical development

• Silevertinib is a CNS-penetrant EGFR inhibitor targeting a broad spectrum of oncogenic EGFR mutations in non-small cell lung cancer (NSCLC) and glioblastoma (GBM).

• Recent ASCO data showed silevertinib's confirmed overall response rate of 60% and median progression-free survival (PFS) of over 15 months in newly diagnosed NSCLC patients with non-classical EGFR mutations.

• Silevertinib demonstrated CNS activity with an 86% confirmed response rate for brain metastases and no new brain metastases developed during the study.

• A phase II trial in newly diagnosed EGFR-mutated GBM patients was initiated in spring, focusing on those with high unmet need (MGMT unmethylated status).

• The GBM study includes an interim analysis that could pivot the trial to a registrational study if results are compelling.

Competitive landscape and differentiation

• Silevertinib covers a broader spectrum of EGFR mutations than competitors, with confirmed responses in 29 unique mutation types.

• It is the only EGFR small molecule with strong penetration into both enhancing and non-enhancing brain tumor regions, critical for efficacy.

• Competing agents like furmonertinib and amivantamab/lazertinib show limited CNS activity and narrower mutation coverage.

• Silevertinib does not target T790M, but this is not seen as a liability due to the mutation's rarity in the target population.

• The focus remains on non-classical EGFR mutations, which represent about 25% of newly diagnosed EGFR-mutated NSCLC cases.

Regulatory and development strategy

• Positive ASCO data supports ongoing FDA discussions for pivotal development in NSCLC.

• The strong CNS activity may enable an expedited pivotal trial with a smaller, faster design, aiming to double median PFS in CNS patients compared to current therapies.

• FDA feedback on pivotal trial design is expected in Q4 2026, with trial initiation targeted for mid-2027.

• Updated data from the frontline NSCLC study and further regulatory milestones are anticipated within the next 12-18 months.