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.
This month sees mixed fortunes for uniQure, with the decision not to renew the marketing authorisation for Glybera, whilst at the same time releasing good news, in the form of European PRIME designation, for its hemophilia B product. Glybera was rejected three times by the European Medicines Agency before finally gaining approval in November 2012, only to see the product used commercially in just a handful of patients. The decision to discontinue the product is no great surprise, but must leave Chiesi Farmaceutici with questions about the long-term viability of its partnership with uniQure. On a more positive note, several companies have released positive clinical data, including early evidence of clinical benefit for Abeona’s cell-based therapy for epidermolysis bullosa, and confirmation of clinical benefit for Biogen’s recently approved, ASO-based treatment Spinraza in spinal muscular atrophy.
The use of T cells to treat autoimmune disorders is a relatively niche area of study, by comparison to the activity we are seeing in oncology, but is an application that holds promise. Atara Biotherapeutics announced positive interim data from an early study investigating ATA188, an autologous T-cell product targeting Epstein–Barr virus, in patients with primary and secondary progressive multiple sclerosis. The company is pursuing the technology on the premise that B cells and plasma cells expressing viral antigens can spur an autoimmune response and MS pathology. We saw Opexa recently try and fail in this space, albeit with a different approach, but given the limited entry of really game-changing therapeutics for MS patients over the years, a cell-based immunotherapy approach is one worth pursuing. What is most intriguing about the early data presented by Atara is the range of objective clinical outcomes experienced by patients, which include reduction in fatigue and nocturnal episodes, improvements in dexterity and walking ability, and improvements in eye sight.
Citation: Cell Gene Therapy Insights 2017;3(5),313-327.