Cell Therapies
Over 140 biopharma leaders share their views on developments to watch for in key therapeutic areas this year. Advances in multiple scientific fields are opening up new avenues for treatment.
Rimqarto is poised to become the first homegrown South Korean CAR-T therapy to gain domestic approval and looks set to provide new local competition to Kymriah.
Cancer retained its crown as 37 novel drugs were approved for pan-EU marketing last year; treatments for blood disorders followed closely behind in what was another slow year at the European Medicines Agency.
The FDA placed a clinical hold on the cell therapy firm’s active INDs that affects Ebvallo and ATA3219 and is connected to the recent complete response letter for Ebvallo.
The Pierre Fabre-partnered allogeneic T-cell immunotherapy is already available in Europe but hopes of a US approval have been hit by a complete response letter citing problems at a third-party manufacturing facility.
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.
CARsgen's anti-Claudin 18.2 CAR-T therapy satricabtagene autoleucel has met its endpoint in a pivotal Phase II trial in later-stage gastric/gastroesophageal junction adenocarcinoma, paving the way for a China NDA filing.
The cell therapy won approval for pediatric steroid-refractory acute graft versus host disease after two complete response letters over the last four years.
The struggling Japanese pharma group is aiming to grow its new cell therapy business in the US and Japan to $2.3bn by the late 2030s.
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.
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.
Both companies are seeking to license their products as they try to stretch their dwindling cash.
Aucatzyl will look to take market share from Gilead’s Tecartus, and the US approval is a boost for the firm and the wider UK biotech sector.
The drug maker said in its third quarter earnings report that competition from other therapeutic classes, particularly bispecifics, resulted in a slight decline of sales for the CAR-T.
The otherwise encouraging data were marred by one patient death, but the company attributed it to the busulfan conditioning regimen and not BEAM-101.
Lyell believes ImmPACT’s dual CD19/20-targeted CAR-T could offer better response rates than Yescarta or Breyanzi and will focus on IMPT-314 while terminating much of its own pipeline.
Multiple Japanese ventures presented updates on their novel pipeline assets and modalities at BioJapan, including cell and gene therapies and combination treatments, despite lingering financing challenges.
ARM’s Cell and Gene Meeting on the Mesa offered optimism that a biopharma financial market recovery is under way, but cell and gene therapies still are seen as risky investments.
Emerging Company Profile: The Maryland-based biotech is working on a functional cure for type 1 diabetes that is similar to Vertex’s VX-880 – but with some key differences.