Biogen Inc. BIIB announced that it plans to initiate a global phase IV study on its spinal muscular atrophy (SMA) drug Spinraza (nusinersen). Named RESPOND, the study will evaluate the clinical benefit and safety of Spinraza for treating SMA in infants and children who still have unmet clinical needs following treatment with Novartis’ NVS new gene therapy Zolgensma (onasemnogene abeparvovec).

Per Biogen’s press release, some patients in a long-term study of Zolgensma shifted to treatment with Spinraza. The efficacy will be assessed by change from baseline on motor function measures, additional clinical outcomes and caregiver burden.

Biogen plans to submit the study protocol to regulatory authorities in the coming months and aims to enroll the first patient in the first quarter of 2021.

Shares of Biogen have lost 5.6% so far this year against the industry’s increase of 11.8%.

We note that SMA is a rare, genetic neuromuscular disease caused by the lack of a functional SMN1 gene resulting in the rapid and irreversible loss of motor neurons, affecting muscle functions including breathing, swallowing and basic movements.

The two-year, open-label RESPOND study will analyze whether the proven efficacy of Spinraza and its continuous production of SMN protein might also benefit those who were previously on gene therapy.

Notably, a few-years ago, Biogen in-licensed the global rights to develop, manufacture and commercialize Spinraza from Ionis Pharmaceuticals IONS, a leader in antisense therapeutics. The former pays royalties to the latter on the net sales of spinraza

Spinraza is approved for the treatment of SMA in the United States. The drug already performed beyond expectations witnessing a strong uptake among patients in the United States and internationally, and now became the standard of care in SMA. The drug generated sales worth $565 million in the first quarter of 2020, reflecting a year-over-year increase of 9%, driven by higher sales in the United States as well as ex-U.S. markets.

However, management believes that Spinraza’s new patient starts and regimen compliance might have declined in the United States during the second quarter, particularly among adults due to the ongoing COVID-19 pandemic. As a result, Spinraza’s sales growth rate in 2020 is expected to be moderate from the 2019-levels.

Moreover, competition looms large on Spinraza. Biogen had earlier acknowledged that Zolgensma’s launch negatively impacted Spinraza’s U.S. sales within the infant population. Meanwhile, Roche RHHBY and PTC Therapeutics’ regulatory application seeking an approval for risdiplam regarding a broad range of patients with SMA is under priority review with the FDA. The decision is pending on Aug 24, 2020, which may build a competitive pressure on Spinraza.

Zacks Rank

Biogen currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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