Multiple sclerosis is a chronic disease of the central nervous system (CNS). As an unpredictable disease that affects people differently, some patients with multiple sclerosis may only have mild symptoms, while others may lose their ability to see clearly, write, speak, or walk when communication between the brain and other parts of the body becomes disrupted.
Multiple sclerosis is caused by the body’s immune system attacking myelin, a protein and fatty substance that surrounds and protects nerve fibers. Consequently, the myelin becomes destroyed in many areas and it is this loss of myelin that forms scar tissue called sclerosis, also referred to as plaques or lesions. When the nerves are damaged in this way, they can’t conduct electrical impulses normally to and from the brain.
With nearly one million people thought to be living with the disease in the U.S. alone, biotech companies are trying to expand therapeutic options for the disease, which is currently treated with disease-modifying drugs like interferons to manage symptoms, and provide treatment options for different types of multiple sclerosis – the main ones being relapsing, primary progressive, and secondary progressive.
In this article, we take a look at six multiple sclerosis companies trying to make a difference to patients by advancing new treatments for the debilitating disease.
Table of contents
Biogen
Leading multiple sclerosis drug candidate of Biogen: BIIB091
Biogen is known for its pioneering work in the neuroscience field. It has been discovering and developing treatments for CNS disorders for more than 40 years, including treatments for multiple sclerosis, amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, depression, lupus, and spinal muscular atrophy (SMA).
With regards to multiple sclerosis, the company already has several approved treatments for the disease, including Avonex, Plegridy, and Vumerity, all of which are used to treat relapsing forms of multiple sclerosis. Furthermore, in its existing pipeline, Biogen is working on the development of a next-generation Burton’s tyrosine kinase (BTK) inhibitor, known as BIIB091, for adults with relapsing multiple sclerosis. It is currently in phase 2 of development.
BTK is a non-receptor tyrosine kinase that regulates the development and signaling of B cells and myeloid cells that are thought to contribute to multiple sclerosis pathogenesis. By reversibly inhibiting BTK in myeloid cells in addition to its effects in B cells, BIIB091 is expected to provide additional clinical benefits over B cell-only therapies and could address the drug resistance seen in covalent BTK inhibitors.
Immunic Therapeutics
Leading multiple sclerosis drug candidate of Immunic Therapeutics: Vidofludimus calcium
Immunic Therapeutics is working on the development of small-molecule therapies for chronic inflammatory and autoimmune disorders. Its lead candidate is called vidofludimus calcium (IMU-838) and is being developed for the treatment of multiple sclerosis.
Vidofludimus calcium works by potently activating the neuroprotective transcription factor called nuclear receptor-related 1 (Nurr1), which is associated with direct neuroprotective properties and may enhance the potential benefit for patients. Vidofludimus calcium also inhibits dihydroorotate dehydrogenase (DHODH), a key enzyme in the metabolism of overactive immune cells and virus-infected cells. This dual action not only targets inflammatory and antiviral processes, but also addresses the underlying neurodegenerative aspects of multiple sclerosis, making it stand out from currently available therapies, which, despite being able to manage multiple sclerosis relapses, do not stop patients from experiencing worsening disability over time.
Vidofludimus calcium is being tested in several ongoing multiple sclerosis trials, including twin phase 3 trials in relapsing multiple sclerosis and a supportive phase 2 trial in progressive multiple sclerosis. In October 2023, Immunic reported positive interim data from the phase 2 trial, as it showed biomarker evidence that vidofludimus calcium’s activity extends beyond the previously observed anti-inflammatory effects. Ultimately, this further reinforces its neuroprotective potential. Furthermore, in January 2024, Immunic announced a private placement of up to $240 million.
Kyverna Therapeutics
Leading multiple sclerosis drug candidate of Kyverna Therapeutics: KYV-101
Although CAR-T therapy is best known for its ability to treat certain blood cancers, some companies are now trying to expand its scope by investigating its potential to treat autoimmune disorders, too. This is what Kyverna Therapeutics is focusing on, as it is attempting to develop cell therapies for patients suffering from B-cell-driven autoimmune diseases.
The multiple sclerosis company has autologous and allogeneic CAR compounds that target CD19 – a molecule that plays an important role in the regulation of the development, activation, and differentiation of B-lymphocytes – and are designed to reduce cytokine release, deplete B cells, and improve clinical tolerability. Its lead candidate, KYV-101, is currently being evaluated to treat multiple sclerosis – as well as myasthenia gravis – in a phase 2 trial. In January, it was granted fast track designation by the U.S. Food and Drug Administration (FDA) in the treatment of patients with refractory progressive multiple sclerosis.
Furthermore, Kyverna brought in an impressive $366 million in gross proceeds from an initial public offering (IPO) in February 2024.
Progentos Therapeutics
Progentos Therapeutics launched only a few months ago, armed with $65 million in series A financing to advance treatments for multiple sclerosis and other degenerative diseases. As touched upon previously, in diseases like multiple sclerosis, disability is caused by demyelination – meaning damage to the myelin sheaths – that support the function and survival of axons. Here, the immune system attacks myelin sheaths, leading to inflammation and impaired nerve cell function. That is why Progentos is developing first-in-class small molecules designed to induce remyelination of axons affected by multiple sclerosis.
The company acquired its multiple sclerosis program from Frequency Therapeutics, a spinout from the Massachusetts Institute of Technology (MIT) that entered a reverse merger with RNA editing company Korro Bio in 2023. According to Progentos, its molecules appear to outperform previous approaches in animal models when it comes to generating the cells that create and maintain myelin.
The funding brought in from the multiple sclerosis company’s recent series A round will enable it to begin advancing its multiple sclerosis program through human proof of concept studies and expand its pipeline in additional degenerative diseases.
TG Therapeutics
Leading multiple sclerosis drug candidate of TG Therapeutics: Ublituximab
Founded with a mission to leverage scientific advances in B-cell biology to develop novel treatments for patients, TG Therapeutics is particularly concentrated on developing therapies for autoimmune disorders. With multiple sclerosis as its lead focus, the company announced in December 2022 that the FDA had approved its drug BRIUMVI (ublituximab) for the treatment of patients with relapsing forms of multiple sclerosis, including clinically isolated syndrome, relapsing-remitting disease, and active secondary progressive disease. Furthermore, in September 2024, TG Therapeutics reported that, after five years of treatment with BRIUMVI, 92% of patients with relapsing multiple sclerosis were free from disability progression.
Ublituximab is a monoclonal antibody that targets a unique epitope on CD20-expressing B-cells. When the drug binds to the B-cell, it triggers a series of immunological reactions, including antibody-dependent cellular cytotoxicity [ADCC] and complement-dependent cytotoxicity [CDC], ultimately leading to destruction of the cell. Additionally, ublituximab is designed to lack certain sugar molecules normally expressed on the antibody, as removal of these sugar molecules – a process called glycoengineering – has been shown to enhance the potency of ublituximab, especially with regards to ADCC activity.
As part of its partnership with Precision BioSciences, TG Therapeutics is also developing an allogeneic CAR-T therapy, called azer-cel, for the treatment of progressive forms of multiple sclerosis. The candidate, which was discovered by Precision BioSciences and licensed to TG Therapeutics, recently received investigational new drug (IND) clearance to bring it into clinical trials, potentially commencing later this year.
Zenas BioPharma
Leading multiple sclerosis drug candidate of Zenas BioPharma: Obexelimab
Zenas BioPharma’s focus is on immunology-based therapies, and it is currently working on the development of its lead candidate obexelimab for the treatment of various immunology and inflammation diseases, including multiple sclerosis.
Obexelimab is a bifunctional monoclonal antibody that is designed to deplete B cells by binding to the proteins CD19 and FcγRIIb, which are broadly present across B cell lineage. According to the company, the co-engagement of these targets results in the suppression of B cell activity.
Zenas BioPharma is currently enrolling patients in a phase 2 trial to evaluate the efficacy and safety of obexelimab in patients with relapsing multiple sclerosis. The company has also raised impressive funds of late. In May 2024, the company announced that it had bagged $200 million in a series C round, before also announcing a $225 million IPO last month. The proceeds from both of these rounds will help the company to further advance obexelimab.
Multiple sclerosis drug market expected to grow in coming years
According to Fortune Business Insights, the global multiple sclerosis drug market size is projected to grow from $21.16 billion in 2024 to $38.94 billion by 2032, exhibiting a compound annual growth rate (CAGR) of 7.9% during the forecast period. This is likely because the increasing prevalence of relapsing remitting multiple sclerosis and primary progressive multiple sclerosis has increased the demand for effective diagnosis and treatment of the disorder globally.
So, with several companies and researchers trying to come up with different approaches to tackle multiple sclerosis, such as BTK inhibitors, CAR-T therapies, bispecific antibodies, and even stem cell therapy, the future looks bright for patients living with the disease, as more effective options for treating different forms of the multiple sclerosis are likely to become available in the coming years.
Partnering 2030: The Biotech Perspective 2023