Armed with new data from what Genentech is touting as one of the biggest and most diverse studies of previously treated spinal muscular atrophy (SMA) patients, the Roche unit is padding its case for Evrysdi in people who may have already received a pricey therapy for their disease.
Wednesday, Genentech pulled back the curtain on two-year data from its Jewelfish study, which looked at patients between 6 months old and 60 years old with type 1, 2 or 3 SMA. The trial specifically enrolled patients who’d previously received other approved or investigational SMA-targeting therapies.
Alongside Evrysdi, which Genentech licensed from developer PTC Therapeutics, Biogen’s and Novartis’ respective gene therapies Spinraza and Zolgensma make up the treatment armamentarium available for SMA. The disease hinders production of the survival motor neuron (SMN) protein, which in turn causes “debilitating and potentially fatal muscle weakness” in patients, Genentech explained.
After an original approval in August 2020 for adults and children over the age of 2 months with SMA, Evrysdi in May clinched a green light in infants younger than 2 months old, deepening the rivalry with Spinraza and Zolgensma, which already boasted approvals in that age group.
Meanwhile, new data from Jewelfish published this week show Evrysdi helped previously treated patients improve or maintain motor function and led to “rapid increases” in SMN protein levels. Those SMN levels remained intact after two years of treatment, Genentech said.
Breaking down the trial, Jewelfish enrolled 174 patients, 63 of whom were adults. Of the group, 105 patients had “very severe” disease according to the Hammersmith Functional Motor Scale Expanded (HFMSE), and 139 patients had scoliosis.
Forty-four percent of patients enrolled (76 patients) had previously received Biogen’s Spinraza, versus just 14 patients who’d been treated in the past with Novartis’ Zolgensma. Aside from those approved therapies, 71 patients in Jewelfish had received the now defunct Trophos SMA prospect olesoxime, and another 13 patients had been treated with Roche’s RG7800.
Olesoxime and RG7800 are no longer in development for SMA, Genentech noted in its release.
The trial found Evrysdi helped double patients’ median SMN protein levels versus baseline after four weeks “in all patient groups,” regardless of which prior treatment they’d received. Exploratory efficacy endpoints further suggested Evrysdi helped patients sustain motor function over two years when compared to the natural history of the disease in untreated patients.
Genentech points out that a recent survey by the patient advocacy group SMA Europe found more than 96% of SMA patients saw disease stabilization as “progress” when it came to their treatment expectations.