Reflections on ASH 2024: Transformative Breakthroughs in Hematology-Oncology

The 66th American Society of Hematology (ASH) Annual Meeting, held from December 7-10 in San Diego and online, delivered on its promise of showcasing groundbreaking advancements in hematology and oncology. Cromos Pharma’s CEO Vlad Bogin attended this event and prepared his takeaways on the most exciting and impactful insights shared at the conference.

ASH 2024 highlighted not only the remarkable strides made in treatments and therapies but also the evolving regulatory environment that is enabling faster clinical progress.

Here are the top takeaways from ASH 2024:

Ascentage Pharma

Olverembatinib (HQP1351) once again took center stage, with multiple studies presented, including an Oral Report for the seventh consecutive year. This investigational therapy continues to demonstrate promise in hematologic malignancies, underscoring Ascentage Pharma’s commitment to addressing unmet needs in hematologic cancer treatments.

Cogent Biosciences

The APEX and SUMMIT Phase 2 trials of bezuclastinib, a selective KIT D816V tyrosine kinase inhibitor, emphasized its potential in systemic mastocytosis, a challenging area with limited options. These findings solidify Cogent’s position as one of the leaders in targeted therapies in this space.

Nurix Therapeutics

With its BTK degradation approach, Nurix presented encouraging data from the NX-5948 Phase 1a/b study. Improvements in T-cell functionality through the NX-2127 program suggest innovative directions for treating relapsed or refractory chronic lymphocytic leukemia (CLL).

Cartesian Therapeutics

The mRNA CAR-T therapy Descartes-08 phase 2 trial of patients with Myasthenia Gravis observed a 5.5-point reduction in MG-ADL at Month 4. The phase 3 trial is being planned for Q1 2025.

IN8bio

Gamma-delta T-cell therapy data from the INB-100 phase 1 study demonstrated continued progression-free survival in patients with acute myeloid leukemia (AML).

CellCentric

Phase II data for inobrodib, a novel oral p300/CBP inhibitor, demonstrated a 75% ORR in relapsed/refractory multiple myeloma when combined with pomalidomide and dexamethasone. This development reaffirms the potential of targeted combination therapies.

Genmab A/S

Epcoritamab-bysp (EPKINLY®) stood out with data from the EPCORE® NHL-2 trial showing that treatment with epcoritamab combination led to an overall response rate (ORR) of 100 percent and a complete response (CR) rate of 87 percent in high-risk patients with previously untreated diffuse large B-cell lymphoma (DLBCL).

Disc Medicine

Disc Medicine announced promising results from its Phase 1b trial of DISC-0974 in patients with myelofibrosis and anemia. The study demonstrated significant hematologic improvements, including elevated hemoglobin levels and reduced transfusion requirements across diverse patient populations, such as those receiving JAK inhibitors. Encouraged by these findings, Disc Medicine has launched a Phase 2 trial to further assess DISC-0974 in a broader cohort of myelofibrosis patients with anemia.

Bluebird Bio

Long-term data on LYFGENIA™ (lovotobegligene autotemcel) gene therapy demonstrated stable anti-sickling hemoglobin production and significant reductions or elimination of vaso-occlusive events (VOEs) across all patient subgroups, sustained through follow-ups exceeding nine years. Additionally, in the first focused analysis of patients with a history of stroke, no recurrent strokes were observed, underscoring the therapy’s potential to provide transformative and durable.

Rigel Pharmaceuticals

Rigel’s Phase 1b trial of R289, a dual IRAK1/4 inhibitor, showed that 40% of transfusion-dependent patients receiving doses ≥500 mg achieved significant hematologic responses, including transfusion independence. The therapy was generally well tolerated, demonstrating a favorable safety profile and supporting its potential as a novel treatment option for heavily pretreated lower-risk myelodysplastic syndrome (LR-MDS) patients.

Eli Lilly and Company

The BRUIN CLL-321 Phase III trial demonstrated that pirtobrutinib significantly improved progression-free survival (PFS) compared to investigator’s choice of Idelalisib plus Rituximab or Bendamustine plus Rituximab in heavily pretreated CLL/SLL patients who had previously received covalent BTK inhibitors. With a favorable safety profile and consistent efficacy across high-risk subgroups, including those with complex karyotype or TP53 mutations, pirtobrutinib offers a promising treatment option for this challenging patient population.

Bristol Myers Squibb

BMS presented data that demonstrated the long-term efficacy and safety of Breyanzi® (lisocabtagene maraleucel) and Abecma® (idecabtagene vicleucel) across blood cancers. Promising updates on pipeline therapies, including the first survival data for arlocabtagene autoleucel (a GPRC5D-directed CAR T for multiple myeloma) and Phase 1 results for CD19 NEX-T CAR T (BMS-986353) in severe refractory autoimmune diseases, highlight the company’s commitment to expanding cell therapy’s transformative potential beyond hematology into autoimmune indications.

Conclusion: Charting the Future of Hematology-Oncology

ASH 2024 underscored the transformative potential of ongoing innovations in hematology-oncology. From gene and cell therapies to targeted treatments and combination regimens, the conference highlighted promising advancements poised to reshape patient outcomes. The positive trends in regulatory timelines and patient-centric approaches further enhance the global momentum in clinical research.

For biopharma professionals, ASH 2024 wasn’t just a conference — it was a glimpse into the future of hematology-oncology, where collaboration, innovation, and efficiency drive progress. As these breakthroughs transition from research to practice, the hematology community is well-positioned to address the most pressing challenges in cancer and blood disorders.

At Cromos Pharma, a CRO with years of expertise in hematology and oncology, we are proud to support the development of innovative therapies that can transform patient care. With our deep understanding of clinical trial management and a commitment to excellence, we are ready to partner with you to advance your research.

Stay tuned for the next wave of updates as these developments continue to influence the landscape of hematology-oncology. For tailored support in your clinical trial needs, reach out to us at inquiry@cromospharma.com. Let’s shape the future of hematology-oncology together!