Rattle and hum: Roche, PTC post update on their SMA drug as a shaken Biogen faces yet another franchise buster
Right on the heels of Novartis’ latest data on its SMA gene therapy for spinal muscular atrophy, PTC and its partners at Roche/Genentech posted their own positive take on the latest data for their oral rival risdiplam. And a shaky Biogen will have to take it into account as well as they assess the future for their blockbuster drug Spinraza, which has led the way on treating this disease for more than 2 years.
Totting up their latest scores, the risdiplam team chalked up:
- 9 infants (53%) were able to maintain an upright head position (HINE-2, n=17)
- 10 infants (59%) demonstrated rolling (rolling to the side, prone to supine, or supine to prone) (HINE-2, n=17)
- 7 infants (41%) were able to sit without support for at least 5 seconds (BSID-III, n=17)
- 1 infant (6%) was able to stand (HINE-2, n=17)
- 85.7% (18/21) babies were event-free overall
Three patients had died, though. And while the researchers were quick to note that none of the infants died as a result of therapy, both Spinraza and Novartis’ treatment have racked up prolonged periods with no deaths.
That sets the stage for an FDA application later this year, as Novartis awaits word from the agency any day now on its pitch for a gene therapy.
SVB Leerink’s Mani Foroohar looked it over and pronounced himself satisfied.
Topline risdiplam data released by Roche prior to the presentations at AAN later this afternoon continue to demonstrate an efficacy and safety profile that appears supportive of registration across spinal muscular atrophy (SMA) subtypes.
The jury is still out on how this rivalry will play out, but the prospect of a gene therapy as well as an oral drug hitting the market may well have a profound effect on Spinraza. Biogen sells this drug for $750,000 for the first year, and then half that for each year thereafter. That gives Novartis plenty of headroom to look for around $2 million for a claimed once-and-done therapy. And then Roche alongside PTC could cause serious disruption with whatever price they pick.
Whatever parents prefer, insurers will have a lot of say over who gets what.
None of that bodes well for Biogen, which can ill afford to see its franchise eaten up by rivals after its big late-stage Alzheimer’s drug aducanumab just crashed and burned.
“Bottom line — we could see a 1/3, 1/3, 1/3 split of market over time, Jefferies analysts recently said that the three drugs could end up dividing things evenly. Based on their discussions with physicians, gene therapy “could be 30-33% share of new SMA Type I-III patients over time. An estimated 20-25% could look to switch from Spinraza to gene therapy (depends on weight of patient). 2) Similarly, docs suggest 1/3 share for oral risdiplam, too, after 3-5 years; 3) Commentary by docs suggests an even split of opinions on the market as well — from oral pills ‘very appealing’ and ‘game changer’ and ‘especially attractive for teens/adults’ to other therapies need more data and likely combo.”