the drug’s secondary measures were mixed. For example, 19.7 percent of patients achieved a new motor milestone after 15 months, compared to 5.9 percent on placebo, but the results weren’t statistically significant. Other secondary measures calculating the ability to stand alone or walk with assistance also failed to meet statistical significance.
Lotze says those gains are still meaningful, considering how old these patients were and how far along their disease was when the trial started. But others disagree.
“Those are the things we ultimately care about more,” says Alexander Fay, a pediatric neurologist and neuromuscular specialist at UCSF Benioff Children’s Hospital in San Francisco, CA, who also wasn’t involved with the trial.
Fay says while CHERISH shows that nusinersen helps Type 2 and Type 3 SMA patients, the study was too short to understand the magnitude of the benefit. Fay still wants to know how it will impact patients in the long term, such as their breathing problems or the spinal curvature that many develop. How significant will the positive effects be, he asks, and will any side effects emerge?
“There’s definitely reason to be optimistic,” he says, ”but we just don’t know yet what the impact is going to be.”
Biogen touts the drug’s safety profile, and clinicians interviewed by Xconomy mainly agree. They say the most common side effects so far are headaches and pain related to the lumbar puncture used to administer nusinersen. Taken together, will the data be good enough for payers to change their policies? “It’s going to create increased pressure and momentum for all children with different types of SMA to be treated, and to be treated as early as possible,” Fay says.
Lotze says he hopes there will be an impact on policies, but expects insurers—as many already have—to demand proof over time that the drug is working to continue treatment.
Analyst Geoffrey Porges of Leerink Partners believes the results leave wiggle room for insurers to “push back on approving Type 2’s and older adolescents and adults” given the “less than spectacular motor milestones results,” he wrote in a note to investors.
As Xconomy has detailed, some frustrated patients and their families have had to wait months before receiving treatment. Others are still awaiting insurance coverage. Treatment centers in California (Stanford University), Pennsylvania (Children’s Hospital of Philadelphia) and New Jersey (Goryeb Children’s Hospital) reported to Xconomy that a minority of their patients had started on drug so far, with many tied up with payers. Biogen CEO Vounatsos said Tuesday that while some “leading” centers have treated more than 10 patients, most have only dosed one or two.
Biogen also said 100 commercial plans and 65 Medicaid plans have approved “individual cases” of nusinersen, that 88 sites in 36 states have administered it, and 203 total sites have submitted “start forms,” which patients fill out to try to gain access to the drug.
A treatment delay can be critical. For a small child with SMA, getting nusinersen could mean more time being able to sit up, stand, or walk. What’s more, patients tend to respond better if they’re treated quickly after diagnosis, says Wildon Farwell, a senior director of medical development at Biogen. “The data are just quite clear across the program that that is in the best interest of the patients,” he says.
Nusinersen’s launch is critical for Biogen, which has no other near-term commercial opportunities in its pipeline. Biogen’s most advanced experimental treatment, aducanumab, won’t produce Phase 3 data until 2019 in the high-risk field of Alzheimer’s disease, which is littered with late-stage failures.
Meanwhile, revenue from the company’s core franchise of multiple sclerosis drugs is flattening, and more competition is on the way, chiefly from a new MS drug from Roche/Genentech, ocrelizumab (Ocrevus).
“It’s a battle every day, and it’s not yet where we want to be,” Vounatsos said of nusinersen’s launch.
