MS Patient’s Pick of the Week’s News: Siponimod, Thymosin Patent, Orphan Drug, Lifestyle, and Lymphopenia Risk

Here’s my Pick of the Week’s News, as published in Multiple Sclerosis News Today.

Gilenya-like Therapy Shows Benefit in Secondary Progressive MS Patients in Phase 3 Trial

Here’s a potentially encouraging development for anyone with SPMS.

Patients with secondary progressive multiple sclerosis (SPMS) who were treated with BAF312 (siponimod), a sphingosine-1-phosphate (S1P) inhibitor, in a Phase 3 clinical trial showed a significantly reduced risk for disability progression compared to placebo,  Novartis recently announced.

BAF312 is a selective modulator of specific types of the S1P receptor. This receptor is commonly found at the surface of immune cells that infiltrate and induce damage in the central nervous system (CNS), leading to loss of function in MS patients.

Similar to Gilenya (fingolimod), BAF312 works by binding to these specific receptors and preventing the migration and activation of immune cells in the CNS. BAF312, however, binds more selectively to specific S1P receptors than Gilenya, particularly to S1P-1 and S1P-5, and is thus expected to avoid lymphopenia (an abnormally low level of lymphocytes, a type of white blood cell, in the blood), one of the more common side effects of Gilenya.

If the expectations prove to be correct, avoiding the abnormally low levels of lymphocytes white blood cells would be a real step in the right direction.

RegeneRx to Receive EU Patent for Molecule That May Lead to MS Remyelination Therapy

Yet another advance in the race to find a workable remyelination treatment.

RegeneRx Biopharmaceuticals announced that it has received an Intent to Grant notice from the European Patent Office (EPO) regarding a patent for its proprietary molecule Thymosin beta 4 (Tβ4), a potential therapy for multiple sclerosis designed to promote remyelination.

The patent will cover the use of Tβ4 in a composition for treating or reducing deterioration that results in injury or damage to tissues caused by MS. It will expire on Jan. 13, 2026.

An injectable formulation known as RGN-352 was developed based on Tβ4, and is designed to be administered systemically to prevent and repair damage that results from central nervous system (CNS) tissue-associated disorders, such as MS, cardiac damage from heart attacks, and traumatic injuries such as stroke. The drug candidate is Phase 2 clinical trial ready, the company said in a press release.

“This patent expands our exclusivity for the potential administration of RGN-352 in patients with multiple sclerosis in key countries in Europe and broadens our patent portfolio related to the systemic use of RGN-352 for CNS and neurodegenerative disorders. We are pleased patent applications for our technologies, some of which were initially filed years ago as part of our intellectual property strategy, continue to be granted throughout the world,” said J.J. Finkelstein, president and chief executive officer of RegeneRx.

It’s only ready to start a Phase 2 clinical trial, so it’s a bit of a way to go yet.

TG Therapeutics’ Potential Neuromyelitis Optica Treatment Named Orphan Drug by FDA

More good news that a special development status has been given to a drug seen to be a potential therapy for two diseases for which the FDA has no approved treatments.

The U.S. Food and Drug Administration (FDA) has designated TG-1101 (ublituximab), a glycoengineered anti-CD20 monoclonal antibody by TG Therapeutics, an orphan drug to advance its development. The drug is a potential treatment for neuromyelitis optica (NMO) and neuromyelitis optica spectrum disorder (NMOSD), two relapsing-remitting autoimmune diseases with similarities to multiple sclerosis.

Currently, there are no FDA-approved treatments for these disorders.

“We are pleased to announce our first orphan drug designation for TG-1101 in a non-oncology indication, providing additional proprietary protection for TG-1101,” Michael S. Weiss, executive chairman and interim CEO of TG Therapeutics, said in a press release.

“NMO is closely related to Multiple Sclerosis, an area of significant interest to us. We look forward to presenting early data from our current Phase 1b study of TG-1101 in NMO at the ECTRIMS (European Committee for the Treatment and Research in Multiple Sclerosis) conference this September, which we believe will provide an early peek into the effects of TG-1101 in patients with autoimmune diseases,” Weiss added.

An orphan drug designation offers special status to a drug or biological product that is intended for the effective and safe prevention, diagnosis, or treatment of a rare disease or condition that affects less than 200,000 people in the United States. It qualifies the treatment’s sponsor for various development incentives, including tax credits for qualified clinical testing.

I have to say that it is good to see the FDA taking supportive action, for once.

Lifestyle May Impact MS Disability, Study Suggests

Hmm, no surprise that a healthier lifestyle, including diet and exercise, can help manage MS symptoms, but a new study goes further.

Previous studies have pointed toward certain lifestyle factors such as nutrition, sedentary behavior, and stress as possible key determinants in multiple sclerosis (MS) progression but few studies have been dedicated to learning more about the impact of lifestyle risk factors on patient disability and disease progression.

A recent study supports the idea that a healthier lifestyle is strongly associated with less MS patient disability. The study, “Associations of Lifestyle, Medication, and Socio-Demographic Factors with Disability in People with Multiple Sclerosis: An International Cross-Sectional Study,” by George Jelinek, PhD, and colleagues from the University of Melbourne, Australia, was published in the journal PLoS One.

The study followed 2,469 participants with a definite or possible MS diagnosis and a sub-sample of 1,493 participants with relapsing-remitting MS (RRMS). Participants were recruited from around the world by internet platforms such as MS online forums and websites and Facebook. They responded to a cross-sectional survey with several questionnaires regarding lifestyle, disability status, and relapses. The large sample allowed the authors to analyze a wide variety of lifestyle factors and to uncover important associations that would have been difficult to detect in a more homogeneous sample population.

I always worry about the validity of any research that relies on self-reporting like this. There are likely to be errors in the data because of this.

MS Treatments May Lower Levels of Immune White Blood Cells

Not so good! Treatments being given to Relapsing MS patients are increasing their risk of having extremely low levels of white blood cells that are essential to defense of their immune systems.

Multiple sclerosis patients are at risk of developing lymphopenia, or abnormally low levels of immune defense white blood cells, called lymphocytes, according to a study that investigated lymphocyte counts in people with relapsing MS both before and after the start of treatment. The study, “Lymphopenia in treatment-naive relapsing multiple sclerosis,” was published in the journal Neurology.

Various therapies are today available to help treat relapsing MS, with the primary goal of regaining function after an attack, preventing new attacks, and, particularly, preventing disability progression. However, as with many other medical treatments, those designed for MS management can cause adverse effects. Some studies have suggested that some medications cause lymphopenia, which is risky in MS because lymphocytes are essential to protecting the body from infection.

Researchers investigated the prevalence of lymphopenia in pretreatment MS patients. The team collected and analyzed data, acquired between 2012 and 2014, from the Southampton regional MS service in the U.K. Patients were classified by age, sex, comorbidities, category of relapsing MS, age at disease symptom onset, number of affected functional systems, dates related to pre- and post-treatment lymphocyte counts, relapse date and severity, and date of starting treatment. For control reasons, lymphocyte data were also taken from healthy individuals, matched by age and sex, treated at the same hospital for cosmetic procedures.

Once again, side effects associated with drug therapies are a cause of concern.

Note: Multiple Sclerosis News Today is strictly a news and information website about the disease. It does not provide medical advice, diagnosis, or treatment. This content is not intended to be a substitute for professional medical advice, diagnosis, or treatment. Always seek the advice of your physician or other qualified health provider with any questions you may have regarding a medical condition. Never disregard professional medical advice or delay in seeking it because of something you have read on this website. The opinions expressed in this column are not those of Multiple Sclerosis News Today, or its parent company, Bionews Services, and are intended to spark discussion about issues pertaining to multiple sclerosis.