- Santhera Pharmaceutical Holding AG's drug Raxone failed to demonstrate any benefit over placebo in a small study of patients with primary progressive multiple sclerosis (PPMS), according to results released Monday by the Swiss biotech.
- Trial data reported by investigators showed no difference between treatment and control groups on a measure of disease progression, although Raxone proved relatively safe.
- Failure of the study, which was conducted by the National Institutes of Health over three years, deals a blow to Santhera's efforts to expand use of Raxone beyond its current approval in the EU as a treatment for a rare eye disease.
Shares in Santhera dropped by more than 20% in value on Swiss trading exchanges as investors digested another setback for the company.
In January, a key committee of the European Medicines Agency again declined to grant an approval of Raxone, or idebenone, in Duchenne muscular dystrophy, turning down an appeal from the company of an earlier rejection. As a result, Santhera announced it would also withdraw a regulatory application in Switzerland.
The EU drugs regulator will require Santhera to gather further data to support its claim Raxone slows the decline of respiratory function in DMD patients who aren't receiving glucocorticoids.
With the snub, Santhera is left with only Raxone's indication for Leber's hereditary optical neuropathy, granted by the EMA under "extraordinary circumstances" given the rarity of the condition. Sales of Raxone in 2017 for this disease totaled roughly CHF23 million (or $25 million).
Success in the multiple sclerosis study would have given Santhera another avenue of research to pursue to support Raxone's use in other populations.
Only one drug, Roche AG's Ocrevus (ocrelizumab) is currently approved in the U.S. for PPMS, a more severe and faster-moving form of the neurodegenerative disease.