ASH 2024 conference has been buzzing with exciting news and developments in the field of hematology. Here is a detailed roundup of some of the key highlights from the conference:
Johnson & Johnson Seeks Approval for Smoldering Myeloma Treatment:
One of the major highlights from the ASH 2024 conference was the presentation of data from the AQUILA trial by Johnson & Johnson. The trial evaluated the use of daratumumab (Darzalex) as a monotherapy for high-risk smoldering myeloma, a precursor to multiple myeloma. The results showed a significant improvement in outcomes for patients who received Darzalex compared to those who underwent active monitoring. The data indicated a 51% reduced risk of disease progression or death in patients treated with Darzalex, with a progression-free survival rate of 63.1% at five years. This groundbreaking data has led Johnson & Johnson to seek approval from regulatory agencies for the use of Darzalex in treating high-risk smoldering myeloma.
Blenrep’s Comeback:
Another highlight from the conference was the resurgence of GSK’s drug Blenrep in the treatment of relapsed or refractory multiple myeloma. Interim data from the DREAMM-7 trial showed a 42% reduction in the risk of death among patients receiving Blenrep, with an impressive projected median overall survival period of 84 months compared to 51 months for the comparator. After a setback following a failed confirmatory study, GSK has refiled Blenrep with the FDA, and the drug is expected to receive approval next summer. The success of Blenrep in the DREAMM-7 trial has reignited hopes for its use in earlier lines of treatment.
Efforts to Ease Conditioning Before Genetic Treatments:
The ASH 2024 conference also shed light on emerging efforts to address the challenges associated with conditioning regimens required for genetic treatments in diseases like sickle cell anemia. Beam Therapeutics presented research on a new conditioning regimen that eliminates the need for the toxic chemotherapy busulfan, making the treatment more accessible to patients. This development could potentially remove a major barrier to accessing curative genetic therapies for debilitating diseases.
Kura Oncology’s Promising Results in AML:
Kura Oncology presented encouraging early-stage trial results for its menin inhibitor in acute myeloid leukemia (AML) at the ASH conference. The Phase 1 trial showed promising outcomes with complete responses in newly diagnosed AML patients, particularly those with specific gene mutations. The oral drug, ziftomenib, when combined with intensive chemotherapy, demonstrated high efficacy and tolerability in patients. This breakthrough therapy has the potential to transform the treatment landscape for AML and offer patients a more manageable and chronic condition.
Overall, the ASH 2024 conference has been a platform for groundbreaking research and developments in hematology, showcasing the potential for innovative treatments to improve outcomes for patients with various blood disorders. Stay tuned for more updates from the conference as researchers continue to unveil exciting advancements in the field. Kura Pharmaceuticals is currently facing competition from Syndax, a Massachusetts-based biotech company that recently received approval for a menin inhibitor in relapsed or refractory AML patients with mutations in KMT2A. Despite this competition, Kura’s CEO, Troy Wilson, remains confident in the market potential of their drug, ziftomenib. Wilson believes that the greatest market opportunity lies in the first-line setting for AML treatment.
One key advantage that Wilson points out is that Kura’s drug does not have the same negative effects on heart rhythms or blood cell counts as Syndax’s drug. This could give Kura a competitive edge in the market, especially in terms of safety and tolerability for patients.
Wilson envisions that ziftomenib could potentially bring in $5 billion in U.S. sales, contingent on the results of two global Phase 3 studies that Kura and its development partner Kyowa Kirin plan to launch next year. These studies will be crucial in determining the drug’s efficacy and safety profile in a larger patient population.
In other news related to pharmaceuticals, Pfizer recently faced scrutiny over the withdrawal of Oxbryta, a drug used for sickle cell disease, due to safety concerns. A special meeting was held to discuss the withdrawal, but Pfizer representatives did not provide much clarity on the situation, leaving attendees with unanswered questions.
Experts like John Strouse from Duke University and Alexis Thompson from the Children’s Hospital of Philadelphia expressed the need for timely and transparent disclosures regarding the safety of pharmaceutical products. The European Medicines Agency is expected to release a report on the matter later this month, while the FDA inquiry may take longer to conclude.
Overall, the pharmaceutical industry continues to be a dynamic and competitive landscape, with companies like Kura Pharmaceuticals and Syndax vying for market share and striving to bring innovative treatments to patients in need. The outcome of ongoing clinical trials and regulatory investigations will ultimately shape the future of these companies and the treatments they offer.
