ASH

Following promising Phase II data presented at ASH on the company's allogenic leukemia-derived dendritic cell vaccine, Mendus has secured support from the FDA and the EMA for pivotal studies starting later this year.

Data presented at the ASH conference showed high overall response and complete response rates in heavily pretreated CLL patients with Epkinly.

With two products on the market, a third on the way and a fourth under development too, the question becomes how they will fit in relative to each other and also CAR-T cell therapies.

BMS presented Phase I/II data for its next-generation small molecule drugs mezigdomide and iberdomide at ASH.

Hematologists discussed the still limited treatment options for sickle cell disease at ASH, weighing the risks and benefits of disease-modifying versus curative therapies after the withdrawal of Pfizer’s Oxbryta (voxelotor).

The drug maker presented Phase I data for PF-08046040 at ASH, having designed it to avoid some of the toxicities associated with antibody-drug conjugates.

In an interview at the conference, a J&J exec noted that bleximenib in frontline AML did not show differentiation syndrome or QTc prolongation.

Roche is highlighting the fixed-duration dosing benefits of Lunsumio and Columvi over AbbVie/Genmab’s Epkinly in B-cell lymphomas as they fight for market dominance. However, the speed at which the companies complete Phase III studies could also be a deciding factor.

China's Belief BioMed presented promising new Phase III results at ASH for its AAV gene therapy for hemophilia B, although the wider China CGT space continues to be dominated by oncology.

Hematologists’ concerns about hepatoxicity with hemophilia A gene therapies may limit use, but Phase III data for Pfizer’s candidate at ASH show a lower rate of ALT increases than seen with BioMarin’s Roctavian.

BMS presented Phase I data for arlocabtagene autoleucel, showing a strong median PFS and lower incidence and severity of trouble swallowing, a common side effect of anti-GPRC5D drugs.

Multiple myeloma patients in the iMMagine-1 study were less heavily pretreated than in the CARTITUDE-1 trial of Carvykti, but Gilead noted that more patients receiving anito-cel had high-risk features.

Overall survival data presented at ASH and filed with the US FDA puts Blenrep on course for a comeback – something that GSK’s oncology leader said he had never really doubted.

Scemblix proved more efficacious than older TKIs, but the older drugs may maintain a presence for economic reasons, though Takeda’s Iclusig may see a diminished role due to tolerability concerns.

Beam presented data from three more patients than during its last update for BEAM-101, showing stem cell engraftment and efficacy consistent with the first four patients.

Patients treated with Novo’s PKR activator had a nearly 50% reduction in vaso-occlusive events relative to placebo in Phase II, VOC occurrence was delayed and blood biomarkers improved.

The Swiss drug maker presented Phase II data at ASH for rapcabtagene autoleucel, also called YTB323, showing a 65% CR rate in relapsed/refractory diffuse large B-cell lymphoma.

The first gene editing medicine and gene therapy for sickle cell disease were approved as the ASH meeting got under way, but novel oral drugs may be key to making treatment more accessible.

The recent ASH meeting began with US FDA approvals of the first genetic medicines for sickle cell disease, but doctors and biopharma companies still see a need for new oral therapies and other options. 

The drug maker provided updates at ASH on newer agents, including a GPRC5D-directed CAR-T therapy, a member of the CELMoD class and its entry into an increasingly crowded BCMA bispecific ring.