Welcome to your weekly roundup of EP Vantage’s snippets – short takes on smaller news items.
This week, January 1-5, 2018, we had thoughts on the following: Takeda’s cautious move on Tigenix; Biontech enters the private financing record books; outcomes-based rebates should soften blow of Luxturna’s price tag.
These snippets were previously published daily via twitter.
Takeda’s cautious move on Tigenix
January 5, 2018
Takeda’s proposed €520m ($626m) acquisition of Tigenix seems a logical extension of the companies’ 2016 licensing agreement, but nevertheless it shows an unusual degree of caution from the Japanese group. The fine print reveals the takeover bid to be conditional on Cx601, Tigenix’s lead asset, securing EU approval, and on “the absence of a material adverse event occurring”. Cx601, a therapy based on cells derived from liposuction procedures, could become Europe’s first ever allogeneic stem cell therapy, and has been developed for treating complex anal fistulas in Crohn’s disease. Still, the acquisition is unlikely to be driven solely by EU approval – Takeda cites a recently begun US phase III trial of Cx601, and says it is exploring regulatory filings in Japan, Canada and emerging markets. And, however cautious it is being, it seems that the chances of a setback in Europe are low: Cx601 secured a positive recommendation from the EU regulator in December.
Source: Company statement.
Biontech enters the private financing record books
January 4, 2018
Biontech’s monster $270m capital raise, announced just before next week’s JP Morgan conference, ranks as the seventh-biggest biotech venture capital round of all time. It is the third-biggest series A round for a biotech company, and could be the biggest ever for a European bio. It will not go unnoticed that Biontech’s focus includes mRNA, which is now officially the hottest area for private biotechs, including Moderna and Curevac along with Biontech as its most prominent exponents. Germany’s Handelsblatt newspaper puts Biontech’s post-money valuation at €2.0-2.5bn ($2.4-3.0bn), meaning that three of biotech’s four biggest unicorns are mRNA players, though Biontech has not revealed this. And there appears to be little solace for those bemoaning the exclusivity of biotech VC financing: the $270m raise reinforces the trend whereby ever bigger amounts of private cash are being scooped up by fewer and fewer companies.
Outcomes-based rebates should soften blow of Luxturna’s price tag
January 3, 2018
Spark Therapeutics’s eye-watering $850,000 price tag for the first US-approved gene therapy was accompanied by a peace offering to payers – namely, a promise only to seek full reimbursement if patients’ vision improves both on short and long-term measures. The Pennsylvania-based group’s “agreements in principle” with the insurer Harvard Pilgrim and affiliates of Express Scripts on payment for Luxturna will likely set a precedent for other gene therapies in the pipeline, such as haemophilia treatments from Biomarin and Spark itself. Spark said it would link payments for the gene therapy for confirmed biallelic RPE65 mutation-associated retinal dystrophy, which costs $475,000 per eye, to improvements in full-field light sensitivity threshold testing scores at 30 and 90 days and at 30 months. The group also created a reimbursement model in which commercial insurers’ speciality pharmacies would pay Spark for Luxturna, rather than requiring hospitals to buy the treatment and bill insurers. To accommodate this model Spark has asked the Centers for Medicare and Medicaid Services to create a demonstration project for Luxturna that will ease price reporting requirements to allow instalment payments and value-based reimbursement at higher levels. Assuming $850,000 per patient, Luxturna would be the highest-priced US drug; outcomes-based pricing means that it will only earn this title if it works as well as advertised.