ASH 2024 Conference Highlights: Exciting Developments in Blood Cancer Treatments
The ASH 2024 conference is in full swing, and Monday’s news brought forth some promising developments in the field of blood cancer treatments. Here’s a detailed roundup of the key highlights from the conference:
Johnson & Johnson Seeks First Drug Approval for Smoldering Myeloma Treatment:
One of the significant announcements at the ASH 2024 conference was Johnson & Johnson’s pursuit of the first drug approval for the treatment of smoldering myeloma. The AQUILA trial results showed that treatment with daratumumab (Darzalex) alone significantly improved outcomes for patients with high-risk smoldering myeloma. This condition precedes multiple myeloma, and the data from the trial indicated a 51% reduced risk of disease progression or death for patients receiving Darzalex. These results have led J&J to submit applications for Darzalex monotherapy approval to treat high-risk smoldering myeloma to the FDA and EMA.
The Controversy Around Early Treatment of Smoldering Myeloma:
The debate around early treatment of smoldering myeloma has been ongoing among myeloma physicians, with differing opinions on the benefits of early intervention. While some experts believe that early treatment is beneficial, others argue that patients who are fated to develop multiple myeloma may require stronger therapies than Darzalex alone. Despite the exciting data from the AQUILA trial, challenges remain in adopting Darzalex as a standard treatment for smoldering myeloma.
Blenrep’s Comeback Story:
GSK’s drug Blenrep made a significant comeback at the ASH 2024 conference, with interim data from the DREAMM-7 trial showing a 42% reduction in the risk of death among patients with relapsed or refractory multiple myeloma. After its initial approval in 2020 and subsequent withdrawal from the market due to a failed confirmatory study, GSK has kept up the program and refiled Blenrep with the FDA following positive results from DREAMM-7 and DREAMM-8 studies.
Efforts to Ease Conditioning Before Genetic Treatments:
One of the key obstacles to accessing genetic medicines for sickle cell disease has been the toxic chemotherapy conditioning required before administering the treatments. New research presented at the conference highlighted emerging efforts to develop softer conditioning regimens that eliminate the need for toxic chemotherapy like busulfan, making treatment more accessible for patients.
Kura Oncology’s Promising Results in AML Treatment:
Kura Oncology presented encouraging early-stage trial results for its menin inhibitor in the treatment of acute myeloid leukemia (AML). The Phase 1 trial showed promising outcomes with complete responses in newly diagnosed AML patients, raising hopes for a potential game-changing therapy in the field. Market analysts have reacted positively to the news, recognizing the blockbuster potential of Kura’s drug in the treatment of AML.
Overall, the ASH 2024 conference has been a platform for groundbreaking research and advancements in blood cancer treatments, offering hope to patients and clinicians alike. Stay tuned for more updates from the conference as it unfolds. Kura Pharmaceuticals is currently facing stiff competition from Syndax, a Massachusetts-based biotech company that recently obtained approval for a menin inhibitor in relapsed or refractory AML patients with mutations in KMT2A. Despite this competition, Kura’s CEO, Wilson, remains optimistic about the market potential for their drug, ziftomenib.
Wilson argues that the real market opportunity lies in the first-line setting, emphasizing that Kura’s drug does not have the same negative effects on heart rhythms or blood cell counts as Syndax’s drug. He believes that ziftomenib could potentially generate $5 billion in U.S. sales, depending on the results of two upcoming global Phase 3 studies that the company plans to launch next year in collaboration with Kyowa Kirin.
On a separate note, Pfizer recently made headlines with the withdrawal of Oxbryta, their drug for sickle cell disease, due to safety concerns. However, a special meeting held to discuss this issue did not provide much clarity on what went wrong with the drug. Attendees, including experts like John Strouse and Alexis Thompson, expressed frustration at the lack of information and unanswered questions surrounding the withdrawal.
There is hope that a report from the European Medicines Agency will shed some light on the situation later this month, although an FDA inquiry is expected to take longer. The timely disclosure of scientific investigations is crucial in situations like this, and the lack of transparency in the case of Oxbryta’s withdrawal has raised concerns among healthcare professionals and industry experts.
Overall, the pharmaceutical industry is constantly evolving, with new drugs entering the market and facing various challenges along the way. Companies like Kura Pharmaceuticals and Syndax are at the forefront of innovation, striving to develop groundbreaking treatments for serious medical conditions like AML and sickle cell disease. It will be interesting to see how these companies navigate the competitive landscape and address the safety concerns that can arise in the development of new drugs.