Poised for an FDA pitch, Novartis lays out all its PhIII cards on MS drug siponimod

Novartis researchers are laying out all of their Phase III cards for an experimental therapy that aims to become the first approved for cases of secondary progressive multiple sclerosis, a fate that tends to involve everyone diagnosed with the relapsing-remitting form of the disease.

As we were told earlier, investigators tracked a significant 21% reduction in the risk of disease progression among patients taking siponimod (BAF312) after three months, making the primary goal of the study. The full results published Thursday evening in The Lancet provide a more complete portrait. Secondaries in the study demonstrated the therapy:

  • Slowed the rate of brain volume loss by 23%.
  • Limited the increase of T2 lesion volume by a mean of about 80%.
  • Reduced the annual relapse rate by 55%.
  • And raised the bar on reducing disease progression to 26% at month 6.

Danny Bar-Zohar

What the drug did not do was significantly improve walking performance.

Nevertheless, Novartis is on a glide path to regulators now with marketing pitches for a drug that they hope to usher into the MS field relatively soon. Novartis execs tell me they plan to file with the FDA in a matter of weeks, with an EMA filing to follow.

“This is pretty much the first and only study in secondary progressive MS that showed meaningful results,” says Novartis’ Danny Bar-Zohar, the global head of neuroscience development at Novartis.

The drug works by binding to the S1P1 sub-receptor on lymphocytes, which prevents them from penetrating the central nervous system.

As for the missed secondary on walking, Bar-Zohar notes that over half of the patients in the study had an EDSS disability score of 6 or higher, meaning they already needed a significant amount of assistance to walk. That may have influenced the outcome.

“We saw a high variability in these patients,” he adds. “It may not be statistically significant but makes us wonder whether these results reflect the reality” for this group of patients.

For Novartis, an approval would help protect its $3 billion in threatened Gilenya revenue, which has been growing fast. Analysts are expecting generic rivals to crowd into the blockbuster market in a little more than 2 years, and Novartis is lining up a new product while Teva has been mounting a final, losing defense against knockoffs of Copaxone.

Roche, meanwhile, is expected to hit pay dirt with Ocrevus (ocrelizumab), which has been successful for both relapsing/remitting MS — where the bulk of the market is — and primary progressive MS patients (PPMS). Recently Celgene’s S1P drug ozanimod was unexpectedly stymied at the FDA, which issued a refuse-to-file notice on their application for MS. Days later, the little biotech Arena Pharmaceuticals impressed a range of analysts with mid-stage results for their S1P drug etrasimod for ulcerative colitis, raising the prospect of another one-day rival for the throne.

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Research Scientist - Immunology
Recursion Pharmaceuticals Salt Lake City, UT
Director of Operations
Atlas Venture Cambridge, MA

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