Mark Curtis & Richard Philipson
Providing a critical overview of the sector’s commercial developments – M&As, licensing agreements & collaborations, financial results, IPOs and clinical/regulatory updates, with commentary from our Expert Contributors.
The race to get the first product approved in hemophilia continues apace, as both Shire and uniQure release positive news this month, albeit in different types of the condition. It’s a crowded space – competitors include Biomarin, Dimension Therapeutics, Sangamo Biosciences and Spark Therapeutics. Biomarin leads the field in hemophilia A with a product already in Phase 1/2, so Shire has some catching up to do following the announcement of its IND submission for SHP654. In hemophilia B, uniQure presented clinical data showing that patients may be eligible for treatment with AMY-060 even when they have pre-existing neutralising antibodies; however, the news is somewhat tarnished by the announcement that Chiesi has withdrawn from an agreement to co-develop the treatment, citing “changes in strategic priorities”.
The cell and gene therapy industry is inching closer to approvals in major markets for the world’s first CART therapies. Both Novartis and Kite have BLAs submitted in the USA for pediatric acute lymphoblastic leukemia (ALL) and relapsed or refractory non-Hodgkin lymphoma (NHL), respectively. In July, Kite Pharma became the first company to submit a Marketing Authorization Application to the EMA for a CART product (axicabtagene ciloleucel). While the first indications being targeted by Novartis and Kite are different, there will be overlap in patient populations in the future. It will be interesting to see how the company’s position their products as approvals are granted following the first set of indications and what the dynamics of market uptake will look like. The latter will be driven by reimbursement, though prospects are strong in both cases.
Citation: Cell Gene Therapy Insights 2017; 3, 509-520.