TSRI-invented compound ozanimod shows positive results in late-stage clinical trial for relapsing multiple sclerosis

February 21, 2017
Demyelination by MS. The CD68 colored tissue shows several macrophages in the area of the lesion. Original scale 1:100. Credit: Marvin 101/Wikipedia

Results from a new Phase 3 study conducted by the Celgene Corporation demonstrate that ozanimod, a drug candidate originally discovered and optimized at The Scripps Research Institute (TSRI), can reduce the frequency of multiple sclerosis relapse.

Relapsing multiple sclerosis is a form of the disease where patients experience a periodic worsening of symptoms. Sensory and motor loss of function leads to increased disability, and patients can need a cane or wheelchair. A signature of the disease is the appearance of lesions in the brain, which are linked to inflammation and can show up through MRI detection during active periods of multiple sclerosis relapse.

Ozanimod, discovered by TSRI Professors Hugh Rosen and Ed Roberts and their laboratories, acts as a sphingosine 1-phosphate 1 (S1PR1) receptor agonist—modulating S1PR1 signaling and blocking sources of inflammation. Rosen and Roberts went on to co-found Receptos, a clinical stage biopharmaceutical company that took ozanimod into Phase 1, 2 and 3 clinical trials and was then acquired by Celgene. Ozanimod is the first New Chemical Entity discovered from a starting point in the NIH Common Fund Molecular Libraries Initiative to reach and succeed in advanced clinical studies.

As reported by Celgene, results from the randomized, Phase 3, double-blind, double-dummy, active-controlled SUNBEAM study among 1,346 participants show that ozanimod met its primary endpoint in reducing annualized relapse rate (ARR) of relapsing , compared with an alternate drug treatment called weekly interferon (IFN) β-1a (Avonex).

Administered at doses of both 1 mg and 0.5 mg, ozanimod demonstrated statistically significant and clinically meaningful improvements, compared to Avonex, for the primary endpoint of ARR and the measured secondary endpoints of the number of MRI-detected lesions and the number of new or enlarging "T2" MRI lesions at after a year of treatment.

"It is exciting and rewarding to see the results of this new Phase 3 trial, which confirm the safety profile from the two-year extension data from the Phase 2 RADIANCE study and underscore ozanimod's efficacy in reducing the burden of MS symptoms on patients and their families," said Rosen. "We look forward to seeing the full study results, as well as the results from the Phase 3 study evaluating ozanimod in patients with ulcerative colitis."

Scientists involved in the trial plan to present the full Phase 3 trial results at an upcoming international scientific meeting.

Explore further: Study shows path to 'dial down' autoimmunity without compromising immune response

Related Stories

Study shows path to 'dial down' autoimmunity without compromising immune response

January 19, 2016
A new study led by scientists at The Scripps Research Institute (TSRI) shows how dangerous autoimmune responses, seen in diseases such as lupus and multiple sclerosis, might be "dialed down" without compromising the immune ...

Celgene will buy drug developer Receptos for $7.32 billion

July 14, 2015
Cancer drug maker Celgene said it will buy Receptos for $7.32 billion in cash, gaining a drug Receptos is studying as a treatment for multiple sclerosis and ulcerative colitis.

MS drug candidate also shows promise for ulcerative colitis

October 28, 2014
Positive new clinical data were released today on a drug candidate for ulcerative colitis that was first discovered and synthesized at The Scripps Research Institute (TSRI).

Study shows ozanimod as effective in treating ulcerative colitis

May 4, 2016
Researchers at University of California San Diego School of Medicine have shown that ozanimod (RPC1063), a novel drug molecule, is moderately effective in the treatment of ulcerative colitis. Results of the Phase II clinical ...

Study sheds light on the biology of progressive form of multiple sclerosis, suggests a new potential path for treatment

February 8, 2017
A research team led by scientists from Brigham and Women's Hospital has revealed how an FDA-approved drug works in the central nervous system in mice to suppress chronic inflammation. The drug, known as FTY720 (or Fingolimod) ...

Genentech announces favorable results for MS drug ocrelizumab

October 9, 2015
Swiss pharmaceutical company Genentech a member of the Roche Group, has released (at the European Committee for Treatment and Research in Multiple Sclerosis meeting) findings from Phase III clinical trials for its multiple ...

Recommended for you

Researchers create tool to measure, control protein aggregation

October 22, 2017
A common thread ties seemingly unlinked disorders like Alzheimer's disease and type II diabetes together. This thread is known as protein aggregation and happens when proteins clump together. These complexes are a hallmark ...

'Selfish brain' wins out when competing with muscle power, study finds

October 20, 2017
Human brains are expensive - metabolically speaking. It takes lot of energy to run our sophisticated grey matter, and that comes at an evolutionary cost.

Researchers find shifting relationship between flexibility, modularity in the brain

October 19, 2017
A new study by Rice University researchers takes a step toward what they see as key to the advance of neuroscience: a better understanding of the relationship between the brain's flexibility and its modularity.

Want to control your dreams? Here's how

October 19, 2017
New research at the University of Adelaide has found that a specific combination of techniques will increase people's chances of having lucid dreams, in which the dreamer is aware they're dreaming while it's still happening ...

Brain training can improve our understanding of speech in noisy places

October 19, 2017
For many people with hearing challenges, trying to follow a conversation in a crowded restaurant or other noisy venue is a major struggle, even with hearing aids. Now researchers reporting in Current Biology on October 19th ...

Investigating the most common genetic contributor to Parkinson's disease

October 19, 2017
LRRK2 gene mutations are the most common genetic cause of Parkinson's disease (PD), but the normal physiological role of this gene in the brain remains unclear. In a paper published in Neuron, Brigham and Women's Hospital ...

0 comments

Please sign in to add a comment. Registration is free, and takes less than a minute. Read more

Click here to reset your password.
Sign in to get notified via email when new comments are made.