After getting hit with a setback that forced the pharma giant Roche to abandon its lead drug for spinal muscular atrophy, company execs are back with a new drug in-licensed from PTC $PTCT that they believe has legs.
It’s an interim, updated look at a tiny data set for risdiplam (or RG7916), but researchers spotlighted evidence that the drug could help significant percentages of infants sit, kick and control their head movements — all elements that the disease destroys as it relentlessly pushes these children to an early death.
This is a single-arm study being pursued by Genentech, the same kind of approach that AveXis scored on in the summer of 2017 for the gene therapy AVXS-101, when noted investigator Jerry Mendell and his team highlighted their own impressive results in a small study. That set of data propelled the biotech to outline plans for an accelerated approval, as well as inspiring Novartis’ Vas Narasimhan to buy the company for $8.7 billion. It’s still widely considered the top contender.
Novartis also has an oral drug similar to Roche’s called LMI070 (branaplam), which got back into the clinic last fall after it was forced into a 2-year hiatus triggered by safety fears raised by animal studies that had highlighted the risk of nerve damage.
Roche turned to its oral SMN2 splicing modifier last summer after regulators on both sides of the Atlantic demanded new late-stage work before they would consider approving their drug olesoxime. Roche punted the drug instead.
Both Novartis and Roche are now hot on the trail of Spinraza, the pioneering drug in the field from Biogen, which was priced at $750,000 — making it one of the most expensive drugs on the planet. It’s also ripe for being overtaken by others. And Baird’s Brian Skorney likes Roche’s chances with this drug. He noted:
The data is compelling so far, and suggests that risdiplam could impact Spinraza’s market share, if it comes to market, given its convenience as an oral small molecule.
In the FIREFISH study, researchers reported that a key score on disease progression showed more than half of the infants with type 1 SMA hit 40-plus. They noted:
The median CHOP-INTEND scores increased over time (37.5 at 6 months [n=20] compared to 41.5 at eight months [n=14]). The median age at first dose in FIREFISH was 6.7 months and median treatment duration was 9.5 months. Nineteen out of 21 infants enrolled (90 percent) remain alive with two having discontinued due to the fatal progression of their disease. Three patients are now over 24 months old. No infant has required a tracheostomy or permanent ventilation since study initiation, and no infant has lost the ability to swallow.
In the Sunfish study for type 2 and 3 SMA, they found:
Of the patients treated with risdiplam for at least one year (n=30), the median change from baseline in Motor Function Measure (MFM), the primary endpoint in the confirmatory part of SUNFISH and a scale used to assess motor function in neuromuscular diseases, was a 3.1-point improvement. Sixty-three percent of patients experienced an improvement in MFM over baseline of three points or more after one year. Such improvements were seen both in patients under 12 years old (76 percent; n=17) and over 12 years old (46 percent; n=13).
“SMA therapies that produce a sustained increase in SMN protein in both the CNS and periphery may provide comprehensive benefits to people diagnosed with SMA, and we look forward to sharing additional data on risdiplam as the clinical program progresses,” said CMO Sandra Horning in a statement.
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