Biogen's Spinraza gets a boost from NICE as competitors loom

As analysts speculate about the future of Biogen’s pricey spinal muscular atrophy (SMA) treatment Spinraza, the company is hoping it can corner the market before competitors take the field. A nod from England’s drug-price watchdog could help.

England’s National Institute for Health and Care Excellence (NICE) recommended Spinraza for use on the country's NHS for treatment of presymptomatic, infantile and late-onset SMA after Biogen agreed to a confidential discount.

The backing makes Spinraza the first and only treatment recommended for use in England for SMA, a rare and often fatal disease that is estimated to affect between 650 and 1,300 patients in the U.K., Biogen said. With its recommendation in hand, Biogen said it would seek to immediately roll out Spinraza for treatment of infantile—or Type 1—SMA patients.

Spinraza’s win in England will help it make its case as the SMA treatment of choice even as competition looms and analysts split opinions on the drug’s future chances.

Novartis’ one-dose gene therapy Zolgensma is waiting in the wings for an FDA approval after the Swiss drugmaker released positive results in March in a series of clinical trials for treatment of multiple SMA types. In phase 3 trials for Type 1 and presymptomatic SMA, dubbed Str1ve and Spr1nt, respectively, Zolgensma extended patients’ lives and improved motor function, Novartis said.

A separate phase 1 trial for treatment of Type 2 SMA patients diagnosed between 7 and 18 months of age showed that all 30 patients survived and 10 achieved major motor function milestones 6.5 months after treatment. Spinraza received its own FDA approval in 2016.

But Zolgensma isn’t the only competitor nipping at Biogen’s heels. Investigational study data for Roche’s SMA hopeful risdiplam, released last week, showed significant motor function improvement in 17 Type 1 SMA patients and 15 surviving at the one-year mark. The study, called Firefish, also showed promise in Types 2 and 3 SMA, Roche said.

With two potential competitors approaching approval, analysts have mixed opinions on when Spinraza can expect a challenge. However, they largely agree that Spinraza’s long-term chances are in serious trouble, which is bad news for Biogen.

Eliana Merle, equity researcher with Cantor Fitzgerald, told investors last week that Zolgensma’s most recent trial data—including slim info on the therapy’s high-dose cohort in Type 2 SMA—didn’t show that the gene therapy was prepped this month for FDA approval in that indication as many analysts predicted. That could give Spinraza some breathing room.

"Our take is that the data are too early to draw a meaningful conclusion relative to Spinraza, although the gene therapy is clearly active in Type 2," she said.

RBC Capital Markets’ Brian Abraham said in a note to investors that Zolgensma’s data wasn’t strong enough to show a serious challenge to Spinraza in the short term, but he did highlight the long-term difficulties the drug faces.

Despite Zolgensma’s modest showing in Type 2, Merle said the gene therapy and risdiplam have already made headway with doctors. In a conference call with a physician after the American Academy of Neurology annual meeting, Merle said the doctor hoped to "have no patients on Sprinraza" in five to 10 years, saying he favored gene therapies over Spinraza’s intrathecal, or spinal injection, administration.

If Spinraza’s sales do dip, it could spell trouble for Biogen, which is dealing with the fallout from the spectacular trial flop of its Alzheimer’s hopeful aducanumab.

In March, Biogen management announced a $5 billion buyback to ease shareholder concerns after aducanumab missed a phase 3 trial's primary endpoints and the company’s stock plummeted, wiping $18 billion off Biogen's market cap. In addition, Biogen’s multiple sclerosis blockbuster Tecfidera is under a patent challenge from Mylan, which could threaten an additional 10% to 15% of the company’s value, analysts have said.

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