MS Research Update 2020

Written and compiled by Tom Garry and Pete Kelly

Reviewed by Barry Hendin, MD, MSAA Chief Medical Officer

Edited by Susan Courtney, MSAA Senior Writer

The Multiple Sclerosis Association of America (MSAA) is pleased to present this 2020 edition of its MS Research Update. The Update highlights important new data on approved and experimental treatments for MS, and is provided to serve as a comprehensive resource for the entire MS community. Please note that the MS Research Update focuses on research related to approved and experimental medications and therapies for the long-term treatment of multiple sclerosis. It does not include information on symptom-management medications or therapies.

For additional information about MS, symptoms and symptom management, as well as MSAA’s programs and services, please visit or call (800) 532-7667.

Also, please note that due to the timing of national and international MS conferences, study data from 2020 conferences generally could not be included in the Update. Information in this publication includes data presented at 2019 conferences, as well as important updates that occurred in the early months of 2020. Please visit MSAA’s website at for future summaries of 2020 conference highlights.

The 2020 MS Research Update is made possible through contributions in honor of Dr. Jules Kernan and Ms. Hannah Dennehy Lee and an anonymous supporter.


The 2020 MS Research Update reviews new data and findings on:

  • Disease-modifying therapies (DMTs) approved by the FDA, including recently approved medications and those that have been available for several years
  • Experimental drugs under investigation for the long-term treatment of multiple sclerosis (MS)
  • New therapeutic approaches and treatment targets, such as stem cell therapy and the gut microbiome
  • Promising areas of inquiry that are enhancing researchers’ and clinicians’ understanding of MS, such as genetics and biomarkers

While this 2020 edition of MSAA’s MS Research Update provides a comprehensive overview of important areas of inquiry and study findings, it is not an exhaustive compilation of all relevant data released in the past year. There is – fortunately – far too much ongoing research for summation in a single report. Rather, the items presented here were selected for their relevance to current or future patient care, and with a view toward showcasing the breadth of work being done to understand and treat MS. The information that follows is drawn from a variety of sources, including journal literature on MS and its management, a review of ongoing clinical trials, and presentations at major national and international conferences.

Please note that this MS Research Update reports on the most recent study results available at the time of publication. While every effort has been made to provide meaningful, timely, and balanced information, keeping the amount of information equal for each medication covered is not possible. Please understand that the different degree of coverage given to the various therapies should in no way be considered as favoritism toward any one medication or treatment approach. Additionally, references have been cited only for newer study results.

While medications for management of MS symptoms are beyond the scope of this report, information on the specific symptoms of MS and their treatment is available in the symptoms section of MSAA’s website.

Providing these resources is at the heart of MSAA’s mission of being a leading resource for the entire MS community, improving lives today through vital services and support. Feedback and thoughts on the 2020 MS Research Update are welcomed. These can be directed to MSAA at

Overview of MS Research Progress

Never before have so many people been so keenly aware of the importance of medical research. With the arrival of the COVID-19 pandemic in early 2020, people who just a few months earlier had only a passing familiarity with the drug-development process, now understand more about the different trial phases.

This widespread appreciation for therapeutic innovation is just one of many ways the coronavirus has changed life for all people. For those with MS, however, the pandemic brings additional concerns and considerations. Does taking a disease-modifying therapy (DMT) that affects the immune system increase the risk of contracting COVID-19? Does having a chronic condition, such as MS, suggest more severe symptoms and worse outcomes should a person become infected? For information on those topics and related issues, visit the Coronavirus and MS section of MSAA’s website.

Beyond its impact on the daily lives of people with MS, the COVID-19 pandemic also is affecting the course of multiple sclerosis research. For example, GeNeuro, a biotechnology company investigating the monoclonal antibody temelimab for use in primary- and secondary-progressive MS, in March announced the postponement of a planned Phase II trial of the medication in order to “prioritize healthcare resources behind the fight of COVID-19 and to reduce the risk for MS patients.”1 Meanwhile, MediciNova, the company developing the oral medication ibudilast for potential use in relapsing forms of MS, announced in April that it also will study the medication for use in acute respiratory distress syndrome (ARDS) caused by COVID-19.2

While some temporary delays, detours, and distractions may be part of the near-term impact of COVID-19 on the MS research agenda, the long-term effect hopefully will include an enduring commitment to funding research against the full range of conditions – acute and chronic – that harm health and threaten lives. Meanwhile, investigators in clinics, hospitals, and laboratories around the world continue to explore the biological processes that lead to MS onset and progression, and the treatment approaches that can reduce or even halt disease activity.

There is abundant evidence that this research effort is making progress on many fronts. One of the most tangible markers of success is the expanding array of disease-modifying therapies (DMTs) available to treat MS.

Since the publication of MSAA’s 2019 MS Research Update, the FDA has approved three more DMTs. In April 2020, Bafiertam™ (monomethyl fumarate) received approval for use in relapsing forms of MS. The Banner Life Sciences’ medication is an oral agent taken twice daily. It is a fumarate-type medication, as is Biogen’s Tecfidera® (dimethyl fumarate). Bafiertam obtained FDA approval after Banner Life Sciences showed that the medication is a “bioequivalent alternative” to Tecfidera, meaning that the active ingredient and site of action do not differ significantly between the two medications.3 Because Tecfidera can cause gastrointestinal (GI) effects – including nausea, vomiting, and diarrhea – in some patients,4 biopharmaceutical companies have explored formulations of fumarate medications that would have efficacy against MS – as Tecfidera has demonstrated – with fewer GI side effects. In the case of Bafiertam, this goal is being pursued through use of a daily dose lower than that for Tecfidera. 4, 5 However, whether or not Bafiertam causes fewer GI side effects has not yet been determined and has not been evaluated in clinical trials in people with relapsing forms of MS.

Another of the three recently approved medications pursues the same goal of reducing GI side effects by other means. In October 2019, the FDA authorized use of Vumerity® (diroximel fumarate) in relapsing forms of MS. Also an oral medication taken twice daily, Vumerity was developed by Biogen – which markets Tecfidera – and the Irish pharmaceutical company Alkermes plc. In this case, the chemical structure of Vumerity differs from that of Tecfidera, and the recently approved medication has been shown to cause fewer GI side effects than Tecfidera. 6

The third medication recently approved by the FDA is Zeposia® (ozanimod), a once-daily oral medication from Bristol Myers Squibb. In March 2020, the FDA approved the sphingosine 1-phosphate (S1P1)-receptor modulator for use in relapsing forms of MS. Two other S1P1-receptor modulators, Gilenya® (fingolimod) and Mayzent® (siponimod), also are FDA-approved for MS. How this class of medication exerts a therapeutic effect in MS is not completely understood. However, the mechanism of action may involve reducing the number of lymphocytes – white blood cells involved in immune function – that migrate to the central nervous system (CNS), where they may contribute to damaging the myelin sheath that protects nerves.7

Turning from medications approved by the FDA to those now being evaluated by the FDA, Janssen/Johnson & Johnson is requesting that another S1P1-receptor modulator, ponesimod, be approved for treating adults with relapsing forms of MS.8 The request is based on data from the Phase III OPTIMUM trial, in which patients treated with ponesimod had lower average annualized relapse rates than those receiving an already approved DMT, Aubagio® (teriflunomide).9, 10

Meanwhile, the FDA is conducting a priority review of ofatumumab for the treatment of relapsing forms of MS. Novartis, which markets the agent collaboratively with Genmab, says ofatumumab could be approved as early as this summer.11 The monoclonal antibody, which already is indicated for treating chronic lymphocytic leukemia,12 binds to the CD20 molecule located on the surface of lymphocytes, a type of white blood cell. Lymphocytes trigger the abnormal immune response that damages the protective sheath (myelin) surrounding nerve cells in the brain and spinal cord. By binding to CD20, the lymphocytes are destroyed and neuronal damage is prevented or delayed.

Ofatumumab is self-injected subcutaneously once a month, allowing for at-home administration.13 Novartis and Genmab are seeking approval for use of ofatumumab in MS based on data from the Phase III ASCLEPIOS I and ASCLEPIOS II trials, in which ofatumumab outperformed Aubagio in slowing disease progression in RMS.11

Phase III trials, which generate the main data used to pursue FDA approval, are under way or planned for other potential MS treatments. TG Therapeutics hopes to provide results later this year from two simultaneous Phase III trials assessing the safety and effectiveness of its monoclonal antibody ublituximab in relapsing forms of MS.15 EMD Serono will evaluate its oral investigational agent evobrutinib in patients with relapsing MS in the Phase III EVOLUTION RMS 1 and EVOLUTION RMS 2 studies.16 Evobrutinib inhibits Bruton’s tyrosine kinase (BTK), an enzyme that contributes to the development and function of B lymphocytes, a type of white blood cell that can attack and destroy the neuroprotective myelin sheath that surrounds nerve cells.16

MediciNova is organizing a Phase III trial that will determine whether its oral medication ibudilast, also known as MN-166, can slow disease progression in more-severe, non-relapsing MS. Ibudilast is a small molecule macrophage migration inhibitory factor (MIF) inhibitor and phosphodiesterase (PDE) -4 and -10 inhibitor that suppresses pro-inflammatory molecules and promotes nerve-growth factors.17

On another research front, investigators continue to assess previously approved medications, examining their long-term effectiveness and safety, both in the overall MS population and in specific groups of people with MS. This MS Research Update reports on studies evaluating the impact of starting specific DMTs earlier rather than later following diagnosis, and of switching from one DMT to another. Inquiries are also examining how approved DMTs are affecting efficacy measures beyond relapse rates and MRI findings, such as confirmed disability progression, and the need for wheelchair use.

In terms of safety, research is examining the frequency and nature of adverse events with multi-year use, how DMTs affect maternal and fetal outcomes when used during pregnancy, and whether altering dosing schedules can reduce the incidence of adverse effects. Beyond evaluating pharmacologic agents, a plethora of research is probing the therapeutic potential of interventions ranging from stem cell therapy and dietary adjustments, to Vitamin D supplementation and altering the gut microbi­ome. Other studies are looking at the role genetics and other factors may play in the development and course of MS, and at how biomarkers such as serum neurofilament light (NfL) can help in monitoring MS status and informing treatment decisions. All of these topics are addressed in the sections that follow.

In short, thousands of clinicians and researchers around the world are exploring various aspects of MS, and their collective efforts promise even further progress in the years just ahead. It is important to remember, however, that the work of these physicians, nurses, biochemists, pharmacologists, and others would not be possible without the selfless participation of even larger numbers of people with MS. The patients who enroll in clinical trials, submit data to registries, and otherwise contribute to research, play an invaluable role in the effort to better understand, more effectively manage, and one day defeat multiple sclerosis. If you already are counted among their ranks, you have our deepest gratitude. If you have not participated to date, we would encourage interested readers to ask their providers about possible opportunities to become involved in MS research. For more information about participating in clinical trials for the treatment of MS and its symptoms, readers may visit

Trial Phases for Investigating Treatments

Phase I

Phase I studies are primarily concerned with assessing the drug’s safety. This initial phase of testing in humans is done in a small number of healthy volunteers, and is designed to determine what happens to the drug in the human body – how it is absorbed, metabolized, and excreted.

Phase II

Once a drug has been shown to be safe, it must be tested for efficacy. This second phase of testing may last from several months to two years, and involve up to several hundred patients. Phase II studies are often “doubleblinded,” meaning that the participants, medical staff, and investigators are not told who is receiving the drug and who is receiving the placebo.

Phase III

In a Phase III study, a drug is usually tested in several hundred to several thousand patients, usually in multiple medical facilities around the world. Phase III studies typically last two or more years. Only after a Phase III study is successfully completed can a pharmaceutical company request FDA approval for marketing the drug.

Phase IV

Phase IV clinical trials are conducted after a drug has been approved. Participants are enrolled to further monitor safety and side effects, while evaluating long-term efficacy.

FDA-Approved Medications: Recently Approved

Three more medications have joined the ranks of FDA-approved therapies for MS since the 2019 MS Research Update was posted. They are: Zeposia® (ozanimod), approved in March 2020; Bafiertam™ (monomethyl fumarate), approved in April 2020; and Vumerity® (diroximel fumarate), approved in October 2019. To follow is information on these medications, their approved uses and dosages, and their clinical data.

FDA-Approved Medications: New Data on Previously Approved Medications

Please note that not all of the approved treatments for MS have been included in this section. For a full listing, please see MSAA’s treatment chart giving an overview of the approved DMTs.

Infused Medications

Oral Medications

Injectable Medications

Experimental Medications

Monoclonal Antibodies

About Monoclonal Antibodies

Monoclonal antibodies are medications derived from identical cells that are cloned from a single cell and then replicated. They are produced from animal tissue, most commonly laboratory mice. Humanized monoclonal antibodies are antibodies from non-human species, again, commonly a mouse, whose protein sequences have been modified to increase their similarity to antibodies produced naturally in humans.

Monoclonal antibodies can be extremely powerful and effective, as they can specifically target a certain segment of one of the body’s systems – such as the immune system – while leaving the other parts of the system unaffected. This can be very desirable when trying to impact a structure as complex as the immune system.

The names of all monoclonal antibodies end with “mab,” including alemtuzumab (Lemtrada®), ocrelizumab (Ocrevus®), and natalizumab (Tysabri®), which are already approved for MS. Several other monoclonal antibodies have shown promise in MS, and five of these are reviewed in this section.

S1P Receptor Modulators

About S1P Receptor Modulators

Sphingosine-1-phosphate receptor (S1P) modulators confine certain immune cells in the lymph nodes so that they cannot reach the central nervous system (CNS) and contribute to the formation of the lesions that are hallmarks of MS. Two FDA-approved disease-modifying therapies (DMTs), Gilenya® and Mayzent®, work in this manner, and other S1P receptor modulators are being evaluated for a possible role in treating MS. The company developing one of those investigational medications, ponesimod, recently asked the FDA and the European regulators to authorize use of its medication in relapsing forms of MS based on the results of the following study.8

Administered Orally

New Directions in MS Research


While the development of new disease-modifying therapies (DMTs) remains a major focus of multiple sclerosis research, considerable attention is also being devoted to other avenues of inquiry. These include stem cell therapy, the role of diet and vitamin supplementation in the onset and management of MS, the impact of genetics, and the significance of various biomarkers in tracking disease onset, progression, and response to therapy. (Examples of biomarkers include substances in the blood or cerebrospinal fluid, as well as findings on imaging). This section of the 2020 MS Research Update reports on several interesting studies addressing these topics.

Closing Notes

This 2020 edition of the MS Research Update highlights a representative selection of some of the most important studies shaping the care of people with MS. The dozens of clinical trials showcased here are but the proverbial “tip of the iceberg.” Thousands of researchers in hundreds of clinics and laboratories around the world are exploring questions and possibilities that offer considerable hope for progress.

Of course, this global research effort is taking place against the backdrop of the COVID-19 pandemic. The coronavirus outbreak poses particular challenges and concerns for people with MS in terms of uncertainty regarding disease susceptibility and severity, among other issues. While some MS-related research initiatives have been halted due to the COVID-19 crisis, other investigators are exploring whether MS therapies may help speed recovery from the viral infection. Above all else, the terrible toll of COVID-19 has underscored the importance of research into the causes and treatment of both acute and chronic conditions.

As people with MS continue to be proactive partners in their care, their participation in clinical trials has enabled the development of several new DMTs, including three approved by the FDA in the past several months and others now being assessed by the FDA or in late stages of investigation.

The challenges and complexities that 2020 has presented thus far are, in the end, no match for the resilience of people with MS and the dedication of their clinicians. That is why MSAA is proud to provide patients and clinicians alike with this edition of its annual MS Research Update. We hope that it will be a valuable resource for all who are committed to enhancing the lives and health of people with MS. We also hope that you will turn to MSAA throughout the year for the latest information in this exciting time of frequent, significant advances in the treatment of MS. For information about opportunities to participate in clinical trials, please visit For more information about MS, its treatments, and MSAA’s programs and services, please contact MSAA at (800) 532-7667, or visit

View References