CLINICAL TRIALS PROFILE FOR SIPONIMOD

All Clinical Trials for Siponimod

Trial ID	Title	Status	Sponsor	Phase	Start Date	Summary
NCT01185821 ↗	Long-term Safety, Tolerability and Efficacy of BAF312 Given Orally in Patients With Relapsing-remitting Multiple Sclerosis	Completed	Novartis Pharmaceuticals	Phase 2	2010-08-30	This study consisted of a two year dose blinded phase during which patients received one of five doses of siponimod (10, 2, 1.25, 0.5 or 0.25mg) following which patients were switched to open label treatment with siponimod 2mg for approximately a further 3 years. It will provide data on long term safety, tolerability and efficacy of siponimod in the RRMS patient population
NCT01665144 ↗	Exploring the Efficacy and Safety of Siponimod in Patients With Secondary Progressive Multiple Sclerosis (EXPAND)	Active, not recruiting	Novartis Pharmaceuticals	Phase 3	2012-12-20	Evaluate the safety and efficacy of Siponimod (BAF312) versus placebo in a variable treatment duration in patients with secondary progressive multiple sclerosis (Core Part) followed by extended treatment with open-label BAF312 to obtain data on long-term safety, tolerability and efficacy (Extension Part).
NCT02029274 ↗	Safety and Efficacy of BAF312 in Dermatomyositis	Terminated	Novartis Pharmaceuticals	Phase 2	2013-08-25	This study investigated the dose response relationship for the efficacy and safety of BAF312 compared to placebo in active DM patients over a treatment period of 6+6 months and to determine the minimum dose required for a maximal clinical effect. The study was composed of 2 periods: a double-blind period 1 with BAF312 administered at different daily doses (0.5, 2, 10 mg and placebo) and a fixed-dose Period 2 in which BAF312 was administered at the dose of 2 mg daily .
NCT03338998 ↗	Efficacy, Safety and Tolerability of BAF312 Compared to Placebo in Patients With Intracerebral Hemorrhage (ICH).	Completed	Novartis Pharmaceuticals	Phase 2	2017-12-24	This is a randomized, placebo-controlled, subject and investigator-blinded study to evaluate efficacy, safety and tolerability of BAF312 in participants with intracerebral hemorrhage (ICH)
NCT03498131 ↗	Melatonin in Patients With Multiple Sclerosis (MS).	Active, not recruiting	Providence Health & Services	Early Phase 1	2018-05-09	To date, there are no published data on the role of melatonin supplementation or the appropriate dose for patients with multiple sclerosis. Because of the potential benefits of melatonin, this pilot study will be an exploratory investigation to evaluate the effect of supplementing melatonin in subjects with multiple sclerosis who are taking an oral disease modifying therapy (DMT) for 6 months or longer. It is our intent that the results of this study will support the rationale and be a prelude to a larger trial which can focus on clinical efficacy of melatonin therapy outcomes.
NCT03623243 ↗	Safety and Tolerability of Conversion From Oral, Injectable, or Infusion Disease Modifying Therapies to Dose-titrated Oral Siponimod (Mayzent) in Advancing RMS Patients.	Recruiting	Novartis Pharmaceuticals	Phase 3	2019-02-14	To assess safety and tolerability of patients converting from approved Relapsing Multiple Sclerosis (RMS) Disease Modifying Therapies (DMTs) to siponimod.
NCT04792567 ↗	Exploring the Immune Response to SARS-CoV-2 modRNA Vaccines in Patients With Secondary Progressive Multiple Sclerosis (AMA-VACC)	Recruiting	Novartis Pharmaceuticals	Phase 4	2021-04-19	The purpose of this study is to understand whether participants can mount an immune response to SARS-CoV-2 modRNA vaccines administered either during continuous siponimod treatment or during a treatment break.

Clinical Trial Conditions for Siponimod

Condition Name

Condition Name for Siponimod
Intervention	Trials
Relapsing Remitting Multiple Sclerosis	2
Secondary Progressive Multiple Sclerosis	2
Hemorrhagic Stroke	1
Immune System Diseases	1

Condition MeSH

Condition MeSH for Siponimod
Intervention	Trials
Multiple Sclerosis	7
Sclerosis	7
Neoplasm Metastasis	3
Multiple Sclerosis, Chronic Progressive	3

Clinical Trial Locations for Siponimod

Trials by Country

Trials by Country for Siponimod
Location	Trials
United States	78
Spain	12
Japan	12
Canada	8
Italy	8

Trials by US State

Trials by US State for Siponimod
Location	Trials
Florida	5
California	4
Ohio	4
Oregon	4
Massachusetts	3

Clinical Trial Progress for Siponimod

Clinical Trial Phase

Clinical Trial Phase for Siponimod
Clinical Trial Phase	Trials
Phase 4	2
Phase 3	3
Phase 2	3

Clinical Trial Status

Clinical Trial Status for Siponimod
Clinical Trial Phase	Trials
Recruiting	4
Active, not recruiting	2
Completed	2

Clinical Trial Sponsors for Siponimod

Sponsor Name

Sponsor Name for Siponimod
Sponsor	Trials
Novartis Pharmaceuticals	7
Providence Health & Services	1
Robert Zivadinov, MD, PhD	1

Sponsor Type

Sponsor Type for Siponimod
Sponsor	Trials
Industry	7
Other	2

Siponimod: A Comprehensive Update on Clinical Trials, Market Analysis, and Projections

Introduction

Siponimod, marketed as Mayzent by Novartis, is a significant advancement in the treatment of multiple sclerosis (MS), particularly for patients with secondary progressive multiple sclerosis (SPMS). This article delves into the clinical trials, market analysis, and future projections for this drug.

Clinical Trials: EXPAND and AMASIA

EXPAND Trial

The EXPAND trial, a pivotal Phase III clinical trial, evaluated the efficacy and safety of siponimod in patients with SPMS. This multicenter, randomized, double-blind, placebo-controlled study involved 1099 patients with SPMS, comparing those treated with siponimod to those receiving a placebo. The trial demonstrated that siponimod reduced the risk of disability progression by 21% compared to the placebo group. Brain changes measured using MRI showed that siponimod decreased brain tissue loss and the number of new and active lesions[4].

AMASIA Study

The AMASIA study is an ongoing observational study that aims to analyze the real-world treatment effects of siponimod in SPMS patients. Initiated in February 2020 and set to continue until February 2025, this study involves 1500 SPMS patients treated with siponimod in up to 250 neurological centers in Germany. The study focuses on evaluating the long-term benefits of siponimod, including changes in the Expanded Disability Status Scale (EDSS), quality of life, and socioeconomic aspects[3].

Efficacy and Safety of Siponimod

Efficacy

The EXPAND trial showed that siponimod significantly slowed the progression of disability in SPMS patients. The primary endpoint, 6-month confirmed disability progression, was lower in the siponimod group compared to the placebo group. Additionally, siponimod reduced the rate of brain tissue loss and the formation of new and active lesions, as observed through MRI[4].

Safety

While siponimod demonstrated efficacy, it also came with side effects, including low white blood cell counts, cardiac and liver abnormalities. These side effects are consistent with other treatments in the same class of drugs targeting the sphingosine-1-phosphate (S1P) receptor[4].

Market Analysis and Projections

Current Market Size

As of 2023, the global market for siponimod was estimated to be worth US$ 357 million. This market is expected to grow significantly, driven by the increasing demand for effective treatments for SPMS[2].

Future Projections

The global market for siponimod is forecasted to reach US$ 967.9 million by 2030, with a compound annual growth rate (CAGR) of 13.2% during the forecast period of 2024-2030. This growth is attributed to the drug's efficacy in treating SPMS, a condition with limited treatment options, and the expanding patient base seeking effective therapies[2].

Economic and Cost-Effectiveness Analysis

Cost-Effectiveness

The Institute for Clinical and Economic Review (ICER) conducted an analysis on the cost-effectiveness of siponimod. The report indicated that siponimod is estimated to cost $1.15 million per quality-adjusted life year (QALY) and $3.76 million per life year gained (LYG) for the overall SPMS trial population. For the active SPMS sub-population, the costs were estimated at $433,000 per QALY and $1.57 million per LYG. These figures highlight the high cost of the treatment relative to commonly accepted thresholds[5].

Real-World Impact and Patient Perspectives

Quality of Life and Socioeconomic Aspects

The AMASIA study is designed to evaluate not only the clinical efficacy but also the real-world impact of siponimod on patients' quality of life and socioeconomic conditions. This includes assessments of functional domains, cognitive worsening, and the perspectives of patients, physicians, and relatives on disability progression[3].

Patient Hope and Clinical Significance

For patients with SPMS, siponimod offers a glimmer of hope in a landscape where treatment options are scarce. Even small changes in EDSS scores can translate into significant improvements in neurological function and daily activities, as noted by the authors of the EXPAND trial[4].

Conclusion

Siponimod represents a crucial advancement in the treatment of secondary progressive multiple sclerosis, a condition that has long lacked effective therapeutic options. The clinical trials, particularly the EXPAND trial, have demonstrated its efficacy in slowing disability progression and reducing brain tissue loss. The market for siponimod is projected to grow substantially, driven by its clinical benefits and the increasing demand for effective MS treatments.

Key Takeaways

• Clinical Efficacy: Siponimod has been shown to reduce the risk of disability progression in SPMS patients.
• Market Growth: The global market for siponimod is expected to reach US$ 967.9 million by 2030.
• Cost-Effectiveness: Siponimod is costly relative to accepted thresholds, but it offers significant clinical benefits.
• Real-World Impact: The AMASIA study is evaluating the long-term benefits of siponimod on quality of life and socioeconomic aspects.
• Patient Hope: Siponimod provides a new treatment option for SPMS patients, offering improvements in neurological function and daily activities.

FAQs

What is the primary use of siponimod?

Siponimod is primarily used to treat adults with relapsing forms of multiple sclerosis, including active secondary progressive multiple sclerosis.

What were the key findings of the EXPAND trial?

The EXPAND trial showed that siponimod reduced the risk of disability progression by 21% and decreased brain tissue loss and the number of new and active lesions in SPMS patients.

What is the projected market size for siponimod by 2030?

The global market for siponimod is forecasted to reach US$ 967.9 million by 2030.

What are the common side effects of siponimod?

Common side effects include low white blood cell counts, cardiac and liver abnormalities.

How does siponimod compare in terms of cost-effectiveness?

Siponimod is estimated to cost $1.15 million per QALY and $3.76 million per LYG, which is higher than commonly accepted thresholds.

Sources

1. Stanford Health Care: Exploring the Efficacy and Safety of Siponimod in Patients With Secondary Progressive Multiple Sclerosis (EXPAND)[1].
2. Valuates Reports: Siponimod - Market, Report Size, Worth, Revenue, Growth, Industry[2].
3. JMIR Research Protocols: Gaining First Insights on Secondary Progressive Multiple Sclerosis[3].
4. MS Australia: Siponimod offers hope for people with secondary progressive MS[4].
5. ICER: ICER Publishes Evidence Report on the Use of Siponimod to Treat Secondary Progressive Multiple Sclerosis[5].