Acthar Gel (repository corticotropin injection) leads to significant improvements after two months of use in people with multiple sclerosis (MS) still having acute relapses despite treatment, and who failed to respond to other high-dose corticosteroids, topline data from an observational study show.
The gel’s developer, Mallinckrodt Pharmaceuticals, also announced that its OPTIONS trial, evaluating the safety of Acthar Gel in relapsing-remitting MS (RRMS) patients who have not responded to treatment with high-dose steroids — a standard of care treatment — is now about half enrolled.
Acthar Gel is currently approved by the U.S. Food and Drug Administration (FDA) for several indications, including the treatment of acute MS flares (disease exacerbations) in adults with frequent and recurring relapses.
In MS, treatment that can effectively prevent a relapse is critical for minimizing disability.
Mallinckrodt conducted an observational registry study (NCT02633033) to monitor the course of MS, and to document relapses and the recovery process, in a group of MS patients treated with Acthar Gel.
The study’s primary goal was to assess the effect of Acthar Gel on symptoms associated with MS relapses after two months of treatment, based on the MSIS-29 impact scale, a tool that measures the impact of MS in physical and psychological aspects from a patient’s perspective.
In the MSIS-29 physical subscale, participants rate 20 physical disease symptoms from one (no impact) to five (extreme impact). Higher scores represent a greater impact of disease symptoms on day-to-day life.
It enrolled 145 adults (mean age of 47) in the U.S., who had MS for about 10 years. Eighty-two completed the study.
More than half of the participants (60%) had a history of intolerance or poor response to treatment with high-dose corticosteroids, and 58.4% reported having had at least one relapse in the two years before enrolling in the study.
Topline results showed that people given Acthar Gel had a significant reduction in their symptoms after two months of treatment. These improvements were still seen at month six.
Specifically, patients treated with Acthar Gel showed a decrease in the MSIS-29 physical subscale of 7.99 at two months, and 9.64 at six months, from a baseline (study start) score of 55.69 .
Physicians also reported significant improvements in patients’ physical symptoms, as shown by a decrease in the mean EDSS scores after two months — from 3.92 (at baseline) to 0.37. At six months, the EDSS score was still significantly lower (0.45) than at the trial’s start (3.92).
The CGI-I scores also indicated a significant improvement at two months in more than half of the patients (63.4%). Once again, these improvements were sustained in 61.4% of patients at six months.
Regarding safety, a total of 83 adverse events were reported in 35 patients (28.0%). Serious adverse events occurred in 11 patients (8.8%).
Overall, the results “support the efficacy and tolerability of RCI [repository corticotropin injection; Acthar Gel] as a treatment for MS relapse,” the researchers wrote.
OPTIONS aims to enroll 66 people, who will be randomized to subcutaneous injections of Acthar Gel (1 mL per day, corresponding to 80 Units) or a matching placebo for 14 days.
The study’s main goal is to assess the response rate based on EDSS at 42 days after treatment. Response is defined as the percentage of patients with an improvement in EDSS scores of at least one point (for those with a EDSS score equal or lower than 5.5 at the trial’s start), or at least 0.5 points (for those with an EDSS score higher than 5.5).
Additional parameters include patients’ scores in the MSIS-29 impact scale and CGI-I scale. Safety will also be assessed.
The 32 adults enrolled (mean age, 42.2) have been living with MS for an average of 13.7 years. The majority (78.1%) are women, and Caucasian (84.4%), with a mean EDSS score at the trial’s start of 3.91.
Researchers hope that data from the OPTIONS trial will “support RCI as an effective treatment for acute exacerbations of RRMS in patients with inadequate response to high-dose steroids,” they wrote.
OPTIONS is recruiting across multiple clinical sites in the U.S. More information can be found here.
“These studies underscore our commitment to patients with difficult-to-treat MS relapse who continue to have symptoms and disease exacerbations that don’t resolve with standard therapy,” Tunde Otulana, MD, senior vice president and chief medical officer at Mallinckrodt, said in a press release.
The two studies presented at ACTRIMS “seek to better understand this patient population and Acthar Gel’s potential role in MS relapse,” Otulana concluded.
Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.
Patrícia holds her PhD in Medical Microbiology and Infectious Diseases from the Leiden University Medical Center in Leiden, The Netherlands. She has studied Applied Biology at Universidade do Minho and was a postdoctoral research fellow at Instituto de Medicina Molecular in Lisbon, Portugal. Her work has been focused on molecular genetic traits of infectious agents such as viruses and parasites.
Patricia holds her Ph.D. in Cell Biology from University Nova de Lisboa, and has served as an author on several research projects and fellowships, as well as major grant applications for European Agencies. She also served as a PhD student research assistant in the Laboratory of Doctor David A. Fidock, Department of Microbiology & Immunology, Columbia University, New York.