Cellectar Biosciences (CLRB) Corporate presentation summary

Pipeline and clinical development

• Advancing phospholipid drug conjugates (PDCs) targeting solid and hematologic tumors, with iopofosine | 131 showing significant efficacy in Waldenström's macroglobulinemia (WM) and multiple myeloma, and additional programs in triple negative breast cancer (CLR 125) and pancreatic cancer (CLR 225).

• Phase 2b CLOVER WaM study for iopofosine | 131 achieved statistically significant response rates, leading to FDA Breakthrough, Fast Track, and EMA PRIME designations.

• Phase 3 confirmatory study for iopofosine | 131 in WM is planned, with accelerated approval application targeted for 1H27 and potential full approval by late 2027 or early 2028.

• CLR 125 Phase 1b study in triple negative breast cancer is ongoing, and CLR 225 is ready for Phase 1 in pancreatic cancer.

• Preclinical and early clinical data support broad activity of the PDC platform across multiple cancer types.

Mechanism of action and platform advantages

• PDCs target lipid rafts, which are overabundant and stable in tumor cells, enabling pan-cancer targeting and intracellular delivery of diverse payloads.

• Platform allows for conjugation of various radioisotopes, offering flexibility and the ability to match isotopes to tumor types.

• Demonstrated rapid uptake, CNS penetration, and improved toxicity profile with limited resistance.

• Proof-of-concept studies confirm selective uptake by tumor cells and reduced off-target effects.

Clinical efficacy and safety

• Iopofosine | 131 in WM showed an overall response rate (ORR) of 80–83.6%, major response rate (MRR) of 58.2–61.8%, and disease control rate (DCR) of 98.2%.

• Median duration of response (DoR) reached 17.8 months and progression-free survival (PFS) was 13.5 months, with better outcomes in earlier-line patients.

• Safety profile characterized by manageable hematologic toxicities and low rates of severe non-hematologic adverse events.

• Additional clinical activity observed in multiple myeloma, CNS lymphoma, pediatric glioma, and other refractory cancers.