HP-001 / Helioson Pharmaceutical - LARVOL DELTA

BEYOND-MM 001: HP-001 Plus Dexamethasone for Relapsed/Refractory Multiple Myeloma With Extramedullary Disease (clinicaltrials.gov) - P2 | N=20 | Not yet recruiting | Sponsor: Institute of Hematology & Blood Diseases Hospital, China

New P2 trial • Hematological Malignancies • Multiple Myeloma • Oncology

Across-cities transportable 13C hyperpolarization using UV-induced labile radicals. (PubMed, Nat Commun) - "We validate this concept through the first across-cities HP 13C-MRSI experiments in vivo, injecting healthy female rats with both a perfusion/angiography ([1-13C]HP001) and a metabolic ([U-13C, d7]glucose) contrast agent. Our findings advance the feasibility of decentralized, scalable HP MRI workflows, removing the barrier of on-site infrastructure."

Journal

Preclinical profiling of CY002-243-1: A highly potent PDE3A-SLFN12 molecular glue for cancer therapy (AACR 2025) - "In the SK-MEL-3 Melanoma tumor xenograft model, administration of HP-001 (0.1 mpk, QD) promoted tumor regression (~95% tumor growth inhibition by 14 days), compared to ~90% tumor growth inhibition by 14 days in BAY2666605 (1 mpk, QD) group. CY002-243-1 is a highly potent, PDE3A-SLFN12 molecular glue, with marked anti-tumor activity as a single agent across diverse tumor cell."

Late-breaking abstract • Preclinical • Lung Cancer • Melanoma • Non Small Cell Lung Cancer • Oncology • Solid Tumor • BCL2 • MAGEE1 • MCL1 • PDE3A

HP-001: A highly potent IKZF1/3 degrader that demonstrates superior safety profile (AACR 2025) - "IMiDs (e.g. pomalidomide, CC-220, CC-92480. HP-001 is a highly potent, catalytic degrader of IKZF1/3, with marked antitumor activity as a single agent and in combination with dexamethasone. HP-001 is currently in phase 1 clinical trial (CDE registration number: CTR20242943), a first-in-human, open-label, multicenter study to evaluate it as monotherapy in patients with r/r MM or NHL."

Clinical • First-in-human • Late-breaking abstract • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Multiple Myeloma • Oncology • CRBN • GSPT1 • IKZF1 • SALL4

HP-001, AN ORAL IKZF 1/3 MOLECULAR GLUE, DEMONSTRATED SUPERIOR DEGRADATION POTENCY AND SPECIFITY IN THE PRE-CLINICAL MODEL (EHA 2025) - "Background: Immunomodulatory drugs (IMiDs) such as lenalidomide and pomalidomide, which hijack the CRBN E3 ligase to degrade IKZF1/3, remain central to hematologic malignancy treatment...Pre-clinical studies demonstrate HP-001's superior degradation potency and selectivity compared to approved IMiDs and next-generation candidates (e.g., mezigdomide, iberdomide, golcadomide, et al), overcoming resistance mechanisms while minimizing off-target effects... HP-001 is a highly potent and specific degrader of IKZF1/3, with marked antitumor activity as a single agent and in combination with dexamethasone. HP-001 is currently in phase 1 clinical trial for a variety of hematologic malignancies. CDE registration number: CTR20242943."

Preclinical • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Multiple Myeloma • Non-Hodgkin’s Lymphoma • Oncology • Targeted Protein Degradation • CRBN • IKZF1