Novartis releases results of first controlled, randomized clinical trial specifically designed for pediatric multiple sclerosis (MS)

• PARADIGMS trial showed reduced relapses in pediatric patients treated with Gilenya^® (fingolimod) versus interferon beta-1a¹
  
• MS is a highly debilitating disease which touches every aspect of young patients' daily lives, from school performance to family relations and friendships²

DORVAL, QC, Oct. 28, 2017 /CNW/ - Novartis has announced full results from the Phase III PARADIGMS study, investigating the safety and efficacy of Gilenya^® (fingolimod) versus interferon beta-1a, in children and adolescents (ages 10 to 17) with multiple sclerosis (MS). Results showed an 82% reduction in the rate of relapses (annualized relapse rate) over a period of up to two years with oral Gilenya compared to treatment with interferon beta-1a intramuscular injections (p <0.001)¹. Gilenya is not currently approved in Canada, or elsewhere, for the treatment of pediatric MS.

"We are pleased to contribute to the advancement of research in MS by conducting and announcing the results of this first controlled, randomized clinical trial specifically in pediatric patients. These findings add to the knowledge available in this population where there is a high unmet need," said Dawn Bell, Chief Scientific Officer, Novartis Pharmaceuticals Canada Inc.

PARADIGMS is the first controlled, randomized trial specifically designed for pediatric MS. The results were presented at the 7th Joint European and Americas Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS-ACTRIMS) meeting in Paris.

Canada has the highest rate of MS in the world, with the disease affecting more than 100,000 Canadians. While MS is most often diagnosed between the ages of 15 and 40, younger children and older adults are also diagnosed³.

"The results from PARADIGMS provide important new information," said Dr. Daniela Pohl, pediatric neurologist, Associate Professor of Pediatric Neurology at the University of Ottawa, and one of the study investigators. "Pediatric MS is challenging to manage because until now, we have been extrapolating data from clinical trials with adult patients to children."

Additional data from the study demonstrated¹:

• A significant reduction in the number of new / newly enlarging T2 and Gd-T1 lesions in the brain in Gilenya-treated patients compared to those treated with interferon beta-1a, as measured by magnetic resonance imaging (MRI).
• Individuals treated with Gilenya had significantly less brain shrinkage (measured by MRI as brain volume loss), compared to those treated with interferon beta-1a.
• The safety profile of Gilenya was overall consistent with that seen in previous clinical trials.

Gilenya (fingolimod) is indicated as monotherapy for the treatment of adult patients with the relapsing-remitting form of multiple sclerosis (MS) to reduce the frequency of clinical exacerbations and to delay the progression of physical disability. Gilenya is generally recommended in MS patients who have had an inadequate response to, or are unable to tolerate, one or more therapies for multiple sclerosis⁴.

About the Phase III PARADIGMS study
The Phase III PARADIGMS study (NCT01892722) is a flexible duration (up to two years), double-blind, randomized, multi-center study to evaluate the safety and efficacy of oral Gilenya compared to interferon beta-1a in children and adolescents with a confirmed diagnosis of multiple sclerosis (MS), followed by a five-year open label extension phase. The study enrolled 215 children and adolescents with MS, between the ages of 10 and 17 years with an Expanded Disability Status Scale (EDSS) score between 0 and 5.5. Patients were randomized to receive once-daily oral Gilenya (0.5 mg or 0.25 mg, dependent on patients' body weight) or intramuscular interferon beta-1a once weekly⁵.

The primary endpoint of the study was the frequency of relapses in patients treated up to 24 months (annualized relapse rate). Secondary endpoints include the number of new or newly enlarged T2 lesions, Gadolinium-enhancing T1 lesions, safety and the pharmacokinetic properties of Gilenya, all measured throughout the treatment period⁵.

The Phase III PARADIGMS study was conducted at 87 sites in 25 countries, including Canada, and was designed in partnership with the US Food and Drug Administration, European Medicines Agency and the International Pediatric Multiple Sclerosis Study Group.

About Multiple Sclerosis
Multiple sclerosis (MS) is a chronic auto immune disorder of the central nervous system (CNS) that attacks myelin, the protective covering of the nerves, causing inflammation and often damaging the myelin. Symptoms include extreme fatigue, lack of coordination, weakness, tingling, impaired sensation, vision problems, bladder problems, cognitive impairment and mood changes⁶. There are three main types of MS: relapsing-remitting MS (RRMS), secondary progressive MS (SPMS) and primary progressive MS (PPMS) and a more rare form, progressive-relapsing MS (PRMS)⁷. In children, RRMS accounts for nearly all cases (approximately 98 percent)⁸. Children with RRMS often experience more frequent relapses than adults with early MS and while evidence suggests a slower disease course in children with MS, significant disability can accumulate at an earlier age than with adult onset MS⁹.

About Novartis Pharmaceuticals Canada Inc.
Novartis Pharmaceuticals Canada Inc., a leader in the healthcare field, is committed to the discovery, development and marketing of innovative products to improve the well-being of all Canadians. In 2016, the company invested $48.8 million in research and development in Canada. Located in Dorval, Quebec, Novartis Pharmaceuticals Canada Inc. employs approximately 750 people in Canada and is an affiliate of Novartis AG, which provides innovative healthcare solutions that address the evolving needs of patients and societies. For further information, please consult www.novartis.ca.

About Novartis
Novartis provides innovative healthcare solutions that address the evolving needs of patients and societies. Headquartered in Basel, Switzerland, Novartis offers a diversified portfolio to best meet these needs: innovative medicines, cost-saving generic and biosimilar pharmaceuticals and eye care. Novartis has leading positions globally in each of these areas. In 2016, the Group achieved net sales of USD 48.5 billion, while R&D throughout the Group amounted to approximately USD 9.0 billion. Novartis Group companies employ approximately 119,000 full-time-equivalent associates. Novartis products are sold in approximately 155 countries around the world. For more information, please visit http://www.novartis.com.

Gilenya^® is a registered trademark of Novartis AG.

References

1		Chitnis T et al. PARADIGMS: A Randomised Double-blind Study of Fingolimod Versus Interferon β-1a in Paediatric Multiple Sclerosis. Late breaking news oral presentation presented at: the 7th Joint ECTRIMS-ACTRIMS meeting on October 28, 2017, Paris, France.
2		MS International Federation, Pediatric MS and other demyelinating disorders in childhood Current understanding, diagnosis and management, 2016, p. 4.
3		MS Society of Canada, About MS, accessed Oct. 22, 2017, at: https://mssociety.ca/about-ms
4		Novartis Pharmaceuticals Canada Inc., Gilenya Product Monograph, December 16, 2016, p. 3.
5		National Institutes of Health, U.S. National Library of Medicine, Clinical Trials. Safety and efficacy of fingolimod in pediatric patients with multiple sclerosis. Accessed Oct. 22, 2017, at:  https://clinicaltrials.gov/ct2/show/NCT01892722
6		MS Society of Canada, What is MS, accessed Oct. 22, 2017, at: https://mssociety.ca/about-ms/what-is-ms
7		MS Society of Canada, Types of MS, accessed Oct. 22, 2017, at: https://mssociety.ca/about-ms/types
8		Waldman A et al. Pediatric multiple sclerosis. Neurology. 2016;87(9): S74-S81
9		National Multiple Sclerosis Society, Pediatric multile sclerosis, accessed Oct. 22, 2017, at: https://www.nationalmssociety.org/What-is-MS/Who-Gets-MS/Pediatric-MS

SOURCE Novartis Pharmaceuticals Canada Inc.

For further information: Novartis Media Relations: Patricia Tiramani, Novartis Pharmaceuticals Communications, [phone], E-mail: [e-mail]; Spectrum Advisors, [phone], [e-mail]