Y-mAbs Therapeutics (YMAB) 2024 Cantor Fitzgerald Global Healthcare Conference summary

Pipeline and platform overview

• Commercial-stage biotech with a marketed product for high-risk, relapsed/refractory neuroblastoma in children.

• Radiopharmaceutical pipeline centers on the SADA platform, with two clinical-stage products: GD2 SADA and CD38 SADA.

• GD2 SADA basket trial includes sarcoma, melanoma, small cell lung, and now adult neuroblastoma.

• CD38 SADA trial targets refractory non-Hodgkin's lymphoma, with trial sites now open.

• Four disclosed targets in development: GD2, CD38, HER2, and B7-H3, with more in preclinical stages.

SADA platform and differentiation

• SADA (self-assembling and disassembling) is a tetramer complex enabling a two-step, in vivo radiopharmaceutical approach.

• The platform separates drug and isotope administration, reducing off-target toxicity and improving tumor targeting.

• Enables use of standard infusion centers and existing infrastructure, broadening physician participation.

• Isotope-agnostic design allows for flexibility in using different radioisotopes for therapy and diagnostics.

• Proprietary chelators in development to support multiple isotopes.

Clinical development and trial design

• GD2 SADA phase I trial uses a three-part design: Part A (protein safety), Part B (isotope dose escalation), Part C (multiple cycles for efficacy).

• Part A focuses on determining safe and optimal protein dosing and timing for isotope injection.

• Imaging and dosimetry data collected using lutetium; patients imaged for GD2 expression before therapeutic dosing.

• No dose-limiting toxicities observed in first 17 patients; heterogeneous GD2 expression confirmed.

• Part A data expected early 2025, with Part B and C to follow for efficacy and expanded dosing.