Billions on the line: BioMarin bustles past enthused rival, planning to launch world’s first hemophilia A gene therapy
BioMarin isn’t going to let a rival’s success over the weekend get in the way of making a bee-line to the FDA with an imperfect, but presentable, gene therapy for hemophilia A.
On Monday the biotech $BMRN reported that its talks with the FDA and the EMA convinced execs to make a leap for an accelerated approval with data on just a handful of patients. And they’re handing over Phase I/II and interim Phase III results in Q4 to make their case.
That decision was made despite a considerable backlash against the latest snapshot of the data, with worsening efficacy over time and a lower initial response in their pivotal trial — but excellent bleed rates, so far. BioMarin says that with a breakthrough drug designation in the USA and Europe’s prime status, they have good reason to believe they are on track to field the world’s first gene therapy.
Investors appear somewhat skeptical though, with BioMarin shares down 2.4% in Monday afternoon trading.
Here’s the bet they’re making, outlined in their latest statement:
Factor VIII levels sustained over a three-year period were sufficient to achieve striking hemostatic efficacy. Factor VIII expression has entered a plateau phase where the rate of decline has substantially slowed, which could be indicative of durable, long-term expression.
Leerink’s Joseph Schwartz offers a bullish assessment of BioMarin’s gamble:
Based on BMRN’s Ph.3 data, the company met their pre-specified criteria for accelerated approval – 8 pts achieving mean factor VIII/FVIII levels above 40 IU/dL. With only 17 pts (out of 20 pts) evaluated so far, it is possible that the remaining 3 could provide more confidence in Valrox although it is not required for accelerated approval since BMRN has already met the bar. The company is focused on getting Valrox to market as soon as possible by submitting their filing in 4Q19 based on their Ph.3 interim analysis and aims to complete GENEr8-1 (6E13 vg/kg) enrollment in early fall 2019. Assuming BMRN could increase and optimize efficacy — recall, chromogenic measures were higher in Ph.1/2 at weeks 23-26 than Ph.3 (68 IU/dL mean/57 IU/dL median in Ph.1/2 vs 36 IU/dL mean/33 IU/dL median Ph.3) — we believe that potential studies BMRN may conduct later could further boost sales and increase Valrox’s attractiveness among hemophilia A pts, especially as competing programs (i.e., SGMO/PFE) appear to be delivering competitive efficacy.
Spark Therapeutics $ONCE is widely viewed as the runner up for now, while Roche struggles to complete its $4.3 billion buyout as soon as possible. But Sangamo/Pfizer garnered some careful second looks a few days ago as they posted better efficacy and a fast response among 4 evaluable patients. That team also outlined plans to make a leap forward on an accelerated pathway as they sought to catch up with the two frontrunners.
Any stumble at this point could prove a setback, but if they stay on track Sangamo and Pfizer may set up a situation where patients can choose an imperfect gene therapy or wait for a better one. And BioMarin has outlined plans to charge in the range of $2 million to $3 million per treatment — with the capability of earning $10 billion to $15 billion a year — adding sticker shock for payers to the list of challenges.