from the National Institutes of Health (NIH) to its rights on a patent granted
in the USA for the use of idebenone for the treatment of primary-progressive MS
(PPMS), a currently untreatable disease affecting about 40,000 patients in the
Unites States. The NIH is investigating the efficacy of
Catena® (idebenone) in PPMS in a placebo-controlled Phase II clinical
trial (the IPPoMS trial).
trial agreement which gives Santhera the rights to the results. Santhera has
now obtained the exclusive rights to the use patent for idebenone in PPMS
granted in the USA. Patients who complete the IPPoMS trial can enter into a
12-months open-label extension trial for which Santhera and the NIH recently
signed a Materials Cooperative Research and Development Agreement (M-CRADA).
“Patients with PPMS do not
respond to immunomodulatory therapies with proven efficacy in relapsing
remitting MS,” said Bibiana Bielekova, M.D, the principal investigator of the
IPPoMS trial. “Accumulating data indicate that mitochondrial dysfunction and
related oxidative stress may play a major role in the pathogenesis of
progressive MS. Idebenone enhances mitochondrial function and acts as an
anti-oxidant against membrane damage in laboratory models and is a rational
treatment choice in PPMS based on these pharmacological properties.”
Effective treatments for
relapsing-remitting MS (RRMS) are limited to immune-modulatory therapies which
have not been proven to be effective in people with PPMS. This subtype develops
much slower than RRMS but presents a steady functional decline without any
distinct episodes of regeneration or acute relapses. IT is estimated there are
about 40,000 people with PPMS living in the United States.
ranging from none to moderately severe are eligible to enroll in the trial.
This is a Phase I/II safety/efficacy trial with an adaptive trial design:
one year of pretreatment baseline period serves the dual purpose of collecting
patient-specific biomarkers of disease progression and collecting longitudinal
neuroimaging and clinical data for selection of primary outcome measures. This
baseline period is then followed by a double-blind, idebenone
(2250 mg/day) versus placebo treatment phase for a total of 2 years.
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