Novartis' latest Zolgensma data bode well for use in older patients

SMA patients ages 2 to 5 who received Novartis' SMA gene therapy Zolgensma showed significant improvements in motor function, the company says. (Zolgensma)

Biogen and Novartis have been battling in spinal muscular atrophy ever since Novartis won approval for its gene therapy Zolgensma, threatening Biogen’s blockbuster Spinraza. Now, Novartis is the latest to tout data for its offering in patients between 2 and 5 years old, and the new data are "extremely encouraging," one exec said.  

In a phase 1/2 study called Strong, SMA Type 2 patients between ages 2 and 5 achieved a mean increase of 5.9 points from baseline on a scale called Hammersmith Functional Motor Scale-Expanded, a commonly used means of measuring physical abilities and motor function among SMA patients in clinical trials. The results came on average 9.3 months after treatment with Zolgensma.

Those numbers are almost double what investigators consider a clinically meaningful result, and they also represent an improvement on earlier results presented in May. At that time, investigators saw a mean increase of 4.2 points on the scale. The study had enrolled 31 patients as of the latest data cutoff.  

Though the results remain early, they're “extremely encouraging for families who hope to see their children with SMA Type 2 experience meaningful improvement in motor function and important milestones, like standing and walking, following a one-time intrathecal administration” with the medicine, AveXis SMA therapeutic head Douglas Sproule said in a statement.

Without treatment, SMA Type 2 patients will never walk without assistance and more than 30% die by age 25, according to Novartis.

Novartis purchased AveXis last year to bolster its gene therapy pipeline and get access to Zolgensma, the company's lead drug. Zolgensma won approval in May for patients under 2 years old. 

RELATED: Novartis to put AveXis into protective custody after data manipulation scandal 

In August, though, the FDA made public a data manipulation episode at AveXis that had occurred before the drug’s approval. Regulators said Novartis knew about alleged data manipulation while the FDA was reviewing the drug and didn’t notify the agency until after approval. Still, the drug should remain on the market, Novartis and the FDA insist.  

Since that scandal, Novartis has terminated scientists who were involved in the manipulation and brought AveXis’s quality control operations into its own group. 

RELATED: With 'remarkable' data, Biogen's Spinraza sharpens its case against Novartis gene therapy 

Novartis' latest data release follows some "remarkable” data for Biogen’s Spinraza in patients with presymptomatic SMA. In an ongoing study of 25 SMA patients on Spinraza, all are alive and don’t require permanent breathing assistance from a ventilator, Biogen said in July.  

The company’s executive director and head of late-stage neuromuscular said it’s a “remarkable result” and “well beyond anything that has been previously reported or ever seen in the natural history of patients with SMA.” 

Meanwhile, both companies face a threat from Roche and PTC Therapeutics' risdiplam, which has been generating eye-turning data as well. The companies have said they plan to file for approval later this year.