HEMATOLOGY

Merck's presentations at ASH 2024 showcased its robust pipeline of hematologicresearch, with over 20 abstracts covering results from trials on multiple investigationalassets. Their presentations included positive results from Phase 2 studies onzilovertamab vedotin (ZV) and nemtabrutinib in indications such as diffuse large B-celllymphoma (DLBCL) and mantle cell lymphoma. ZV in combination with R-CHPdemonstrated a complete response rate of 100% at 1.75 mg/kg dose in the Phase 2waveLINE-007 trial of treatment-naive patients with DLBCL. Merck also presentedresults from a selection of the 1,600 clinical trials on KEYTRUDA, their largest revenuedriver that is seeking label expansion across multiple indications.

GENE THERAPIES

SonoThera took to ASH 2024 to showcase theirultrasound-mediated gene delivery (UMGD) platform'sability to noninvasively deliver nonviral genetic payloadswith durable and titratable FVIII and FIX geneexpression. The UMGD platform aims to treathematological diseases like Hemophilia A and B bydelivering oversized DNA payloads to the liver. The preclinical data demonstrated the platform's safety andredosability, overcoming the two largest challengesfaced by existing delivery mechanisms.

GENE THERAPIES

Long-term data on Vertex's CRISPR/Cas9 gene-editedtherapy CASGEVY, presented at ASH 2024,demonstrated efficacy in both severe sickle cell disease(SCD) and transfusion-dependent beta thalassemia(TDT). The clinical trials reported that 93% of SCD patientswere free from vaso-occlusive crises, and 98% of TDTpatients were transfusion-independent for at least 12consecutive months. Evidence of CASGEVY's safety andefficacy have facilitated approvals in the US, UK, andthe EU as the first therapy of its kind in each market.

MULTIPLE MYELOMA

J&J's ASH 2024 presentations were headlined by data fromthe Phase 3 MajesTEC-4 trial, which highlighted Tecvayli'spotential as a therapy option for newly diagnosed multiplemyeloma (NDMM) patients undergoing autologous stem celltransplantation. Results demonstrated MRD-negativity atfollow-up for all patients and a cumulative 40% incidence ofgrade 3-4 neutropenia. J&J also presented results from theGRIFFIN study, demonstrating significant improvements inPFS at 36 months for NDMM transplant-eligible patientsreceiving the lenalidomide, bortezomib, and dexamethasone(RVd) combination in comparison with the standard of care.

LYMPHOMA

BeiGene presented its latest research in chroniclymphocytic leukemia (CLL) at ASH 2024. TheSEQUOIA study demonstrated that BRUKINSAreduced the risk of progression or death by 71%over five years compared to bendamustinerituximab in treatment-naïve patients. Thecompany also showcased data from the Phase1/1b trial of sonrotoclax combined with BRUKINSAthat achieved a 99% overall response rate, andthe Phase 1/2 study of the BTK degrader BGB16673 that showed a 94% overall response rate intreatment-resistant CLL cases.

LYMPHOMA

Results from Lilly's Phase 3 BRUIN CLL-321 trial presentedat ASH 2024 demonstrated that pirtobrutinib reduced therisk of disease progression or death by 46% compared tostandard treatments in 238 BTK inhibitor pre-treatedCLL/SLL patients. Pirtobrutinib extended medianprogression-free survival to 14.0 months and time to nexttreatment to 23.9 months, while also showing a favorablesafety profile with fewer grade 3 or higher adverse events.Pirtobrutinib is currently approved under the FDA'sAccelerated Approval pathway, and will likely receiveapproval in additional markets based on these results.