Latest news with #CMSC
Yahoo
12-06-2025
- Health
- Yahoo
Positive PoNSTEP Study Results Presented at Consortium of Multiple Sclerosis Centers Annual Meeting
-PoNS Therapeutic Experience Program (PoNSTEP) study demonstrates durable long-term beneficial effects of PoNS Therapy® on gait deficit improvement in people with Multiple Sclerosis- -Presentation of full PoNSTEP study results expanded upon top line data previously reported- NEWTOWN, Pa., June 12, 2025 (GLOBE NEWSWIRE) -- Helius Medical Technologies, Inc. (NASDAQ: HSDT), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait deficits, today announced that Deborah Backus, PT, Ph.D., FACRM- Vice President of Research and Innovation at Atlanta's Shepard Center presented the positive results from the PoNS Therapeutic Experience Program, or PoNSTEP, study for people with multiple sclerosis (MS) at the 2025 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting on May 29 in Pheonix, Arizona. Dr. Backus' presentation highlighted the importance of treatment adherence as a key requirement for achieving a clinically meaningful improvement in gait disability over 14 weeks of therapy and its sustained therapeutic effect well into 6 months after the end of treatment. The study results confirm controlled and real-world clinical evidence of PoNS Therapy's long-term benefits already reported in people with balance deficit due to traumatic brain injury (TBI). 'The results from this study were received with great interest as they provide the first clinical trial evidence of the long-term therapeutic benefits of PoNS Therapy for functional rehabilitation in the MS population,' stated Antonella Favit-Van Pelt, M.D., Ph.D., Helius' Chief Medical Officer. 'We were excited to engage with both US healthcare professionals and Canadian MS specialists and offer additional insights on the body of clinical and mechanistic evidence of PoNS Therapy's effect in MS. We are encouraged by the growing recognition of the importance of PoNS' direct effect on the central nervous system and its targeted mechanism of action as well as how it correlates with a lasting and sustained effect on gait rehabilitation, especially when comparing PoNS Therapy to other interventions limited to peripheral neuromuscular or transcutaneous stimulation.' CMSC attendees were encouraged at Helius' recent successes in obtaining initial reimbursement from federal and private payers and appreciated the Company's relentless effort to engage with health insurance providers, viewing it as a much-needed endeavor to expand access to PoNS Therapy to all people with MS. 'We are excited by the positive conversations had at CMSC regarding both the therapeutic impact of the data presented and the recent announcement of reimbursement from the VA and out of network commercial payers for PoNS,' stated Dane Andreeff, Helius' President and Chief Executive Officer. 'We anticipate that the increase in reimbursement coverage will significantly lower the barrier to prescribe PoNS and allow for greater access to this important therapy.' About PoNS Therapeutic Experience Program (PoNSTEP) The PoNS Therapeutic Experience Program ('PoNSTEP') is a Helius-sponsored, open-label, observational, interventional multi-center outcome research study designed to assess adherence to on-label PoNS Therapy for improvement in gait deficits for patients with multiple sclerosis ('MS') in a real-world clinical setting. The study aims to understand better the relationship between adherence to on label (100-120 minute per day) PoNS Therapy, which combines the PoNS device with physical therapy, and the therapeutic outcome on gait deficit improvement over 14 weeks of study treatment, as measured by changes in the Dynamic Gait Index (DGI) scores. PoNS Therapy is applied in a supervised clinical setting for the first two weeks (Phase 1) and, independently, at home for the remaining 12 weeks (Phase 2). The study also includes a six-month no-treatment follow-up phase aimed at establishing the durability of the therapeutic effect (Phase 3). The primary endpoint of the study is maintenance of gait improvement from the end of supervised therapy (Phase 1) to the end of unsupervised therapy (Phase 2) in relation to the subject's adherence to PoNS Therapy. The secondary endpoints are, among others, maintenance of improvement of gait and balance deficit over a 6-month timeframe and clinical global impression of change. The study was performed at six Centers of Excellence across the United States, including Neurology Center of New England in Foxboro (MA), the Shepherd Center in Atlanta (GA), Montefiore Medical Center ('Montefiore') in NY (NY), Oregon Health & Science University ('OHSU') in Portland (OR), MGH Institute of Health Professions in Boston (MA), NYU Langone Health in NY (NY), and recruited 43 MS participants with gait deficit. About the PoNS Device and PoNS Therapy The Portable Neuromodulation Stimulator ('PoNS') is an innovative, non-implantable, orally applied therapy that delivers neurostimulation through a mouthpiece connected to a controller and it's used, primarily at home, with physical rehabilitation exercise, to improve balance and gait. The PoNS device, which delivers mild electrical impulses to the tongue, is indicated for use in the United States as a short-term treatment of gait deficit due to mild-to-moderate symptoms from MS and is to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and over by prescription only. PoNS has shown effectiveness in treating gait or balance and a significant reduction in the risk of falling in stroke patients in Canada, where it received authorization for sale in three indications: (i) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from stroke and is to be used in conjunction with physical therapy; (ii) for use as a short-term treatment (14 weeks) of chronic balance deficit due to mild-to-moderate traumatic brain injury ('mmTBI') and is to be used in conjunction with physical therapy; and (iii) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from MS and is to be used in conjunction with physical therapy. PoNS is also authorized for sale in Australia for short-term use by healthcare professionals as an adjunct to a therapeutic exercise program to improve balance and gait. For more information visit About Helius Medical Technologies, Inc. Helius Medical Technologies is a leading neurotech company in the medical device field focused on neurologic deficits using orally applied technology platform that amplifies the brain's ability to engage physiologic compensatory mechanisms and promote neuroplasticity, improving the lives of people dealing with neurologic diseases. The Company's first commercial product is the Portable Neuromodulation Stimulator. For more information about the PoNS or Helius Medical Technologies, visit Cautionary Disclaimer Statement Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as 'believe,' 'expect,' 'continue,' 'will,' 'goal,' 'aim' and similar expressions. Such forward-looking statements include, among others, statements regarding future presentation and uses of the PoNSTEP study results, the availability of commercial reimbursement and the uses and effectiveness of PoNS and PoNS Therapy. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Company's expectations include uncertainties associated with the Company's capital requirements to achieve its business objectives, availability of funds, the Company's ability to find additional sources of funding, manufacturing, labor shortage and supply chain risks, including risks related to manufacturing delays, the Company's ability to obtain national Medicare insurance coverage and to obtain a reimbursement code, the Company's ability to continue to build internal commercial infrastructure, secure state distribution licenses, market awareness of the PoNS device, future clinical trials and the clinical development process, the product development process and the FDA regulatory submission review and approval process, other development activities, ongoing government regulation, and other risks detailed from time to time in the 'Risk Factors' section of the Company's Annual Report on Form 10-K for the year ended December 31, 2023, and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at or The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law. Investor Relations Contact Philip Trip TaylorGilmartin Groupinvestorrelations@ in retrieving data Sign in to access your portfolio Error in retrieving data Error in retrieving data Error in retrieving data Error in retrieving data
Hamilton Spectator
12-06-2025
- Health
- Hamilton Spectator
Positive PoNSTEP Study Results Presented at Consortium of Multiple Sclerosis Centers Annual Meeting
-PoNS Therapeutic Experience Program (PoNSTEP) study demonstrates durable long-term beneficial effects of PoNS Therapy® on gait deficit improvement in people with Multiple Sclerosis- -Presentation of full PoNSTEP study results expanded upon top line data previously reported- NEWTOWN, Pa., June 12, 2025 (GLOBE NEWSWIRE) — Helius Medical Technologies, Inc. (NASDAQ: HSDT), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait deficits, today announced that Deborah Backus, PT, Ph.D., FACRM- Vice President of Research and Innovation at Atlanta's Shepard Center presented the positive results from the PoNS Therapeutic Experience Program, or PoNSTEP, study for people with multiple sclerosis (MS) at the 2025 Consortium of Multiple Sclerosis Centers (CMSC) Annual Meeting on May 29 in Pheonix, Arizona. Dr. Backus' presentation highlighted the importance of treatment adherence as a key requirement for achieving a clinically meaningful improvement in gait disability over 14 weeks of therapy and its sustained therapeutic effect well into 6 months after the end of treatment. The study results confirm controlled and real-world clinical evidence of PoNS Therapy's long-term benefits already reported in people with balance deficit due to traumatic brain injury (TBI). 'The results from this study were received with great interest as they provide the first clinical trial evidence of the long-term therapeutic benefits of PoNS Therapy for functional rehabilitation in the MS population,' stated Antonella Favit-Van Pelt, M.D., Ph.D., Helius' Chief Medical Officer. 'We were excited to engage with both US healthcare professionals and Canadian MS specialists and offer additional insights on the body of clinical and mechanistic evidence of PoNS Therapy's effect in MS. We are encouraged by the growing recognition of the importance of PoNS' direct effect on the central nervous system and its targeted mechanism of action as well as how it correlates with a lasting and sustained effect on gait rehabilitation, especially when comparing PoNS Therapy to other interventions limited to peripheral neuromuscular or transcutaneous stimulation.' CMSC attendees were encouraged at Helius' recent successes in obtaining initial reimbursement from federal and private payers and appreciated the Company's relentless effort to engage with health insurance providers, viewing it as a much-needed endeavor to expand access to PoNS Therapy to all people with MS. 'We are excited by the positive conversations had at CMSC regarding both the therapeutic impact of the data presented and the recent announcement of reimbursement from the VA and out of network commercial payers for PoNS,' stated Dane Andreeff, Helius' President and Chief Executive Officer. 'We anticipate that the increase in reimbursement coverage will significantly lower the barrier to prescribe PoNS and allow for greater access to this important therapy.' About PoNS Therapeutic Experience Program (PoNSTEP) The PoNS Therapeutic Experience Program ('PoNSTEP') is a Helius-sponsored, open-label, observational, interventional multi-center outcome research study designed to assess adherence to on-label PoNS Therapy for improvement in gait deficits for patients with multiple sclerosis ('MS') in a real-world clinical setting. The study aims to understand better the relationship between adherence to on label (100-120 minute per day) PoNS Therapy, which combines the PoNS device with physical therapy, and the therapeutic outcome on gait deficit improvement over 14 weeks of study treatment, as measured by changes in the Dynamic Gait Index (DGI) scores. PoNS Therapy is applied in a supervised clinical setting for the first two weeks (Phase 1) and, independently, at home for the remaining 12 weeks (Phase 2). The study also includes a six-month no-treatment follow-up phase aimed at establishing the durability of the therapeutic effect (Phase 3). The primary endpoint of the study is maintenance of gait improvement from the end of supervised therapy (Phase 1) to the end of unsupervised therapy (Phase 2) in relation to the subject's adherence to PoNS Therapy. The secondary endpoints are, among others, maintenance of improvement of gait and balance deficit over a 6-month timeframe and clinical global impression of change. The study was performed at six Centers of Excellence across the United States, including Neurology Center of New England in Foxboro (MA), the Shepherd Center in Atlanta (GA), Montefiore Medical Center ('Montefiore') in NY (NY), Oregon Health & Science University ('OHSU') in Portland (OR), MGH Institute of Health Professions in Boston (MA), NYU Langone Health in NY (NY), and recruited 43 MS participants with gait deficit. About the PoNS Device and PoNS Therapy The Portable Neuromodulation Stimulator ('PoNS') is an innovative, non-implantable, orally applied therapy that delivers neurostimulation through a mouthpiece connected to a controller and it's used, primarily at home, with physical rehabilitation exercise, to improve balance and gait. The PoNS device, which delivers mild electrical impulses to the tongue, is indicated for use in the United States as a short-term treatment of gait deficit due to mild-to-moderate symptoms from MS and is to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and over by prescription only. PoNS has shown effectiveness in treating gait or balance and a significant reduction in the risk of falling in stroke patients in Canada, where it received authorization for sale in three indications: (i) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from stroke and is to be used in conjunction with physical therapy; (ii) for use as a short-term treatment (14 weeks) of chronic balance deficit due to mild-to-moderate traumatic brain injury ('mmTBI') and is to be used in conjunction with physical therapy; and (iii) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from MS and is to be used in conjunction with physical therapy. PoNS is also authorized for sale in Australia for short-term use by healthcare professionals as an adjunct to a therapeutic exercise program to improve balance and gait. For more information visit . About Helius Medical Technologies, Inc. Helius Medical Technologies is a leading neurotech company in the medical device field focused on neurologic deficits using orally applied technology platform that amplifies the brain's ability to engage physiologic compensatory mechanisms and promote neuroplasticity, improving the lives of people dealing with neurologic diseases. The Company's first commercial product is the Portable Neuromodulation Stimulator. For more information about the PoNS or Helius Medical Technologies, visit . Cautionary Disclaimer Statement Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as 'believe,' 'expect,' 'continue,' 'will,' 'goal,' 'aim' and similar expressions. Such forward-looking statements include, among others, statements regarding future presentation and uses of the PoNSTEP study results, the availability of commercial reimbursement and the uses and effectiveness of PoNS and PoNS Therapy. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Company's expectations include uncertainties associated with the Company's capital requirements to achieve its business objectives, availability of funds, the Company's ability to find additional sources of funding, manufacturing, labor shortage and supply chain risks, including risks related to manufacturing delays, the Company's ability to obtain national Medicare insurance coverage and to obtain a reimbursement code, the Company's ability to continue to build internal commercial infrastructure, secure state distribution licenses, market awareness of the PoNS device, future clinical trials and the clinical development process, the product development process and the FDA regulatory submission review and approval process, other development activities, ongoing government regulation, and other risks detailed from time to time in the 'Risk Factors' section of the Company's Annual Report on Form 10-K for the year ended December 31, 2023, and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at or . The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law. Investor Relations Contact Philip Trip Taylor Gilmartin Group investorrelations@
Medscape
09-06-2025
- Health
- Medscape
Perspective Shifts on the Use of MS Meds During Pregnancy
PHOENIX — Two new datasets support the use of potent disease-modifying therapies (DMTs) during pregnancy in women with active multiple sclerosis (MS), reinforcing a growing shift toward treatment when the potential benefit to the mother outweighs the risks. Both studies — one involving the anti-CD20 monoclonal antibody ofatumumab and the other the integrin receptor antagonist natalizumab — are observational, but they provide a foundation for taking a proactive rather than reactive approach to treating pregnant women with active MS, said Riley M. Bove, MD, associate professor of neurology, University of California San Francisco. Bove emphasized that a 'we-don't-know' approach is no longer acceptable when counseling pregnant women with active disease. As first author of the ofatumumab study — presented on May 29 at Consortium of Multiple Sclerosis Centers (CMSC) 2025 Annual Meeting— she followed up with a lecture the next day, explaining that evidence is now available to guide treatment decisions. Relevant Data While significant knowledge gaps remain about the relative risks of DMTs to fetal development and pregnancy outcomes, it is well established that women with active disease 'will be harmed if we do not help,' Bove said. She and others now believe that the available data, even if observational, are relevant and useful in guiding decisions that affect both maternal and fetal health. The new data on ofatumumab and natalizumab offer a clear example. In an ongoing registry, ofatumumab exposure has been documented in 669 pregnancies. Of these, 221 were reported prior to 2023, allowing for assessment of both short- and long-term outcomes. In most cases (87%), exposure occurred during the first trimester. Adverse outcomes were reported, including spontaneous abortions in 12.6% of cases, preterm births in 9.6%, and minor congenital malformations in two infants (< 1%). However, these rates are 'in line with the background rates observed in the general population,' Bove noted. She emphasized that such data are important when weighing treatment decisions against the known risks of active disease, which can lead to irreversible brain injury in the mother. The natalizumab data, presented as a late-breaking abstract at CMSC on May 30, reflected 16 years of experience at a single center. Tracking began in 2008, when two pregnancies were identified in women already receiving natalizumab. Through 2024, no increased risk for adverse pregnancy outcomes was observed, while natalizumab treatment was associated with meaningful improvements in MS disease control. A total of 58 pregnancies in 43 women have been tracked at the Rocky Mountain MS Clinic in Salt Lake City, according to Katrina Bawden, FNP-C, a nurse practitioner who has been involved since the registry began. Reevaluating Natalizumab She noted that outcomes were analyzed in women who discontinued natalizumab after learning they were pregnant as well as those who continued treatment into the third trimester. Among the 38 pregnancies in which natalizumab was stopped, 13 women experienced clinical relapses, and four others showed new lesions on MRI. In contrast, among the 20 pregnancies where treatment was continued, there were no relapses and no MRI evidence of disease activity. Pregnancy complications were observed, including one fetal malformation and 10 miscarriages, but Bawden noted that these figures — like those in the ofatumumab registry — are consistent with background rates. 'Of the three fetal deaths, all occurred in those who discontinued natalizumab,' she said. She noted that all 10 of the women who miscarried had healthy term full term deliveries in a subsequent pregnancy while remaining on natalizumab. Compared to the start of the tracking period, Bawden said the data have prompted clinicians at her clinic to reevaluate the benefit-risk balance of using natalizumab during pregnancy. 'Women at the Rocky Mountain MS Clinic who become pregnant while treated with natalizumab, using shared decision-making, are now given the option of continuing natalizumab every 8 weeks throughout pregnancy with the last dose scheduled at 34 weeks' gestation,' Bawden said. 'Illogical Guidance' Caring for pregnant women with MS is a complex challenge, given the incomplete information available. However, Bove — co-author of a 2024 paper on practical considerations for weighing the risks and benefits of DMT use during pregnancy — said that strictly following drug labeling is not helpful in guiding clinical decisions. She noted that current recommendations are inconsistent across drug classes, vary between the FDA and the European Medicines Agency, and often fail to reflect the latest science — resulting in guidance that is ultimately 'illogical.' Moreover, labeling continues to evolve, and pregnancy-related use of DMTs remains a dynamic area, with new data — such as the recent studies presented at CMSC — shaping clinical strategies. Bove emphasized that it is the clinician's responsibility to stay informed as the evidence develops, in order to support shared decision-making. This includes staying up to date on when to treat active disease during pregnancy, when to restart therapy if it was paused, and how to weigh the benefit-risk profile of DMTs for women who choose to breastfeed. When making MS treatment decisions during pregnancy, the adage 'first, do no harm' has traditionally focused on fetal risk. But Bove pointed out that withholding treatment may, in many cases, pose greater harm to the mother, underscoring the need for a balanced discussion that considers risks to both mother and fetus.
Medscape
09-06-2025
- Health
- Medscape
Evidence Shifts Perspective on MS Drug Use in Pregnancy
PHOENIX — Two new datasets support the use of potent disease-modifying therapies (DMTs) during pregnancy in women with active multiple sclerosis (MS), reinforcing a growing shift toward treatment when the potential benefit to the mother outweighs the risks. Both studies — one involving the anti-CD20 monoclonal antibody ofatumumab and the other the integrin receptor antagonist natalizumab — are observational, but they provide a foundation for taking a proactive rather than reactive approach to treating pregnant women with active MS, said Riley M. Bove, MD, associate professor of neurology, University of California San Francisco. Bove emphasized that a 'we-don't-know' approach is no longer acceptable when counseling pregnant women with active disease. As first author of the ofatumumab study — presented on May 29 at Consortium of Multiple Sclerosis Centers (CMSC) 2025 Annual Meeting— she followed up with a lecture the next day, explaining that evidence is now available to guide treatment decisions. Relevant Data While significant knowledge gaps remain about the relative risks of DMTs to fetal development and pregnancy outcomes, it is well established that women with active disease 'will be harmed if we do not help,' Bove said. She and others now believe that the available data, even if observational, are relevant and useful in guiding decisions that affect both maternal and fetal health. The new data on ofatumumab and natalizumab offer a clear example. In an ongoing registry, ofatumumab exposure has been documented in 669 pregnancies. Of these, 221 were reported prior to 2023, allowing for assessment of both short- and long-term outcomes. In most cases (87%), exposure occurred during the first trimester. Adverse outcomes were reported, including spontaneous abortions in 12.6% of cases, preterm births in 9.6%, and minor congenital malformations in two infants (< 1%). However, these rates are 'in line with the background rates observed in the general population,' Bove noted. She emphasized that such data are important when weighing treatment decisions against the known risks of active disease, which can lead to irreversible brain injury in the mother. The natalizumab data, presented as a late-breaking abstract at CMSC on May 30, reflected 16 years of experience at a single center. Tracking began in 2008, when two pregnancies were identified in women already receiving natalizumab. Through 2024, no increased risk for adverse pregnancy outcomes was observed, while natalizumab treatment was associated with meaningful improvements in MS disease control. A total of 58 pregnancies in 43 women have been tracked at the Rocky Mountain MS Clinic in Salt Lake City, according to Katrina Bawden, FNP-C, a nurse practitioner who has been involved since the registry began. Reevaluating Natalizumab She noted that outcomes were analyzed in women who discontinued natalizumab after learning they were pregnant as well as those who continued treatment into the third trimester. Among the 38 pregnancies in which natalizumab was stopped, 13 women experienced clinical relapses, and four others showed new lesions on MRI. In contrast, among the 20 pregnancies where treatment was continued, there were no relapses and no MRI evidence of disease activity. Pregnancy complications were observed, including one fetal malformation and 10 miscarriages, but Bawden noted that these figures — like those in the ofatumumab registry — are consistent with background rates. 'Of the three fetal deaths, all occurred in those who discontinued natalizumab,' she said. She noted that all 10 of the women who miscarried had healthy term full term deliveries in a subsequent pregnancy while remaining on natalizumab. Compared to the start of the tracking period, Bawden said the data have prompted clinicians at her clinic to reevaluate the benefit-risk balance of using natalizumab during pregnancy. 'Women at the Rocky Mountain MS Clinic who become pregnant while treated with natalizumab, using shared decision-making, are now given the option of continuing natalizumab every 8 weeks throughout pregnancy with the last dose scheduled at 34 weeks' gestation,' Bawden said. 'Illogical Guidance' Caring for pregnant women with MS is a complex challenge, given the incomplete information available. However, Bove — co-author of a 2024 paper on practical considerations for weighing the risks and benefits of DMT use during pregnancy — said that strictly following drug labeling is not helpful in guiding clinical decisions. She noted that current recommendations are inconsistent across drug classes, vary between the FDA and the European Medicines Agency, and often fail to reflect the latest science — resulting in guidance that is ultimately 'illogical.' Moreover, labeling continues to evolve, and pregnancy-related use of DMTs remains a dynamic area, with new data — such as the recent studies presented at CMSC — shaping clinical strategies. Bove emphasized that it is the clinician's responsibility to stay informed as the evidence develops, in order to support shared decision-making. This includes staying up to date on when to treat active disease during pregnancy, when to restart therapy if it was paused, and how to weigh the benefit-risk profile of DMTs for women who choose to breastfeed. When making MS treatment decisions during pregnancy, the adage 'first, do no harm' has traditionally focused on fetal risk. But Bove pointed out that withholding treatment may, in many cases, pose greater harm to the mother, underscoring the need for a balanced discussion that considers risks to both mother and fetus.
Medscape
06-06-2025
- Health
- Medscape
Experimental MS Drug Nearly Eliminates Disease Activity
PHOENIX — Frexalimab, a second-generation anti-CD40 ligand monoclonal antibody provides extended tight control of multiple sclerosis (MS) whether measured by relapse or brain imaging at 2-year follow-up, results of an open-label extension (OLE) of a phase 2 trial showed. 'At week 96, there was almost complete suppression of new gadolinium-enhancing lesions with very similar pattern seen with new or enhancing T2 lesions,' said study investigator Stephen Krieger, MD, professor of neurology, Icahn School of Medicine at Mount Sinai, New York City. Two phase 3 international studies with this drug are already enrolling. 'Part of the interest in frexalimab and anti-CD40 therapies is the idea that one can modulate both B- and T-cell activity without cell depletion,' explained Krieger, who presented the long-term open-label data on May 29 at the Consortium of Multiple Sclerosis Centers (CMSC) 2025 Annual Meeting. Near Complete Disease Suppression The latest data suggest frexalimab is fulfilling its promise. Over follow-up to date, there has been nearly complete suppression of gadolinium-enhancing (Gd+) lesions on MRI among those taking the dose now being tested in the phase 3 trials. At 2 years, with an annualized relapse rate of 0.08%, 92% of patients were relapse-free. The randomized portion of this phase 2 trial attracted attention when it was published a year ago in The New England Journal of Medicine , but the 2-year results showed that the efficacy and safety observed at 12 weeks persist. In the controlled trial, 129 patients with relapsing MS were randomized to 300-mg, 400-mg, 600-mg, or 1200-mg frexalimab or matching placebos. Suppression of Gd+ lesions was the primary endpoint. At 12 weeks, the adjusted mean of new Gd+ lesions was 1.4 in the combined placebo groups but 0.3 in the 300-mg frexalimab group and 0.2 in the 1200-mg group. Of those who participated in the randomized portion of the phase 2 trial, 97% continued into the long-term OLE. The OLE consisted of two arms: 1200-mg frexalimab administered intravenously every 4 weeks or 300-mg frexalimab administered subcutaneously every 2 weeks. At the end of 2 years, when 82% of those enrolled in the OLE were still on medication, the adjusted mean for new T1-weighted Gd+ lesions ranged from 0.1 to 0.3 across study arms whether on continuous frexalimab or switched from placebo to frexalimab. For those who were initiated on the 1200-mg dose in the controlled portion of the trial and remained on this dose for the OLE, the mean was 0.1. For the secondary endpoint of new or enlarging T2 lesions, the suppression at 2 years was almost the same. Again, the adjusted mean for new lesions across all arms ranged from 0.1 to 0.3. For those receiving the 1200-mg dose, the mean was 0.2. Mean T2 lesion volume increased in the placebo arm but not in the treatment arms during the randomized phase. After entering the OLE, T2 lesion volume fell in placebo patients now on active therapy. In the 1200-mg arm, the fall in lesion volume during the randomized phase continued into the first 24 weeks of the OLE. After 24 weeks, the lesion volume remained suppressed with no return toward baseline. Those initiated on placebo never caught up after switching to frexalimab. Relapse Rare — 2% at 96 Weeks On the 1200-mg dose of frexalimab, only 8% had any relapse recorded over the extended follow-up. In half, there was a single relapse. Only 2% had three or more relapses. While the Expanded Disability Status Scale score declined slightly among placebo patients once started on active therapy, there was no change from baseline through 96 weeks in patients started on any active therapy. As postulated by earlier preclinical and clinical studies, frexalimab had no effect on lymphocyte counts over time. Over the 96-week follow-up, levels of immunoglobulins remained unchanged, according to Krieger who showed graphs with straight lines for these values over the course of the OLE. Due to the potential of suppressing activation of both T and B cells over time, anti-CD40 therapies have long been considered a promising mechanism for control of MS. However, clinical development of first-generation drugs was abandoned because of an association with thromboembolism. 'Frexalimab has been engineered to avoid these events through a change in the Fc receptors with reduces downstream inflammatory events,' said Krieger. The long-term data support this premise. Over 2 years, there was one pulmonary embolism, but this exception was observed in a patient with a viral illness and a genetic predisposition for an inflammatory response, according to Krieger. When surveying other adverse events, 'nothing jumps out' in the OLE relative to the randomized phase. One potential exception is a rise in liver function tests observed in two (4%) patients on the 1200-mg dose. Only one of these patients discontinued therapy, and the levels returned to normal over time in both. The effects of the anti-CD40 mechanism on both the adaptive and innate immune systems suggest frexalimab might offer efficacy for both progressive and relapsing MS. In the ongoing phase 3 program, one of the trials (FREXALT) is enrolling patients with relapsing MS. The other (FREVIVA) is enrolling patients with progressive disease. Fulfilling its Promise Commenting on the results, Amit Bar-Or, MD, Chief of the Multiple Sclerosis Division, the Department of Neurology, University of Pennsylvania Perelman School of Medicine, Philadelphia, described frexalimab as 'a very interesting drug.' He agreed that the CD40 ligand is a promising target in MS but cautioned that these phase 2 data cannot answer the most interesting questions. This includes the more robust evidence of safety and efficacy from phase 3 trials, but it remains unclear whether the benefits extend beyond controlling relapsing disease. 'I think there is particular interest in whether it will also show extended benefit in progressive MS, and this will be a major focus of interest from the next set of studies,' Bar-Or said.
