EXTON, Pa., Nov. 13, 2018 /PRNewswire/ -- The Institute for Clinical and Economic Review (ICER) released their draft background and scope document on October 31st in support of their evaluation of the health and economic outcomes of Novartis's Mayzent (siponimod) for the treatment of secondary progressive multiple sclerosis (SPMS). According to Spherix Global Insights' recently published RealWorld Dynamix™: Progressive Forms of Multiple Sclerosis (US) audit, ICER's recognition of progressive disease as "active" or "not active" is a clinically relevant distinction. In the current audit, as well as previous reports, Spherix confirms significant differences in patient demographics, disability progression, and treatment patterns between the active and not active SPMS subgroups ─ insights not available through traditional secondary data or audit sources which do not capture metrics at the subtype level. While disease-modifying therapies (DMTs) approved to treat relapsing forms of multiple sclerosis (MS) can be used to treat active SPMS patients with ongoing relapses, there are currently no approved DMTs* for the not active form of SPMS resulting in a high unmet need for these progressing patients.
In the audit, which analyzed over 400 SPMS patient records, DMT-treated not active SPMS patients tend to have a longer disease duration, yet similar mean age and EDSS scores, compared to active SPMS patients suggesting earlier disease onset with slower progression. Not active SPMS patients are also more likely to have no documentation of a last relapse or to have had their last relapse three or more years ago and to have a lower gadolinium-enhanced lesion burden. Among those that did have a relapse, not active SPMS patients are more likely to have had a full recovery implying continued functional reserve for a subset of patients. Although fewer not active SPMS patients are experiencing ongoing disability progression compared to active SPMS, among the two out of five patients who are progressing, a large majority are considered to be experiencing progression independent of relapse activity (PIRA) highlighting the importance of a therapy shown to be efficacious on disability progression independent of anti-inflammatory activity.
With no approved therapies and patients presenting with low inflammatory disease activity, the not active SPMS segment represents a slow churn segment with a low treatment rate and a low DMT switch rate compared to the active SPMS segment. Not active SPMS patients have been on therapy for significantly longer than active SPMS patients but are more likely to still be on their first- or second-line DMT. While the switch rate is higher among not active SPMS audit patients compared to US neurologists' self-reported estimate, only one-third have switched in the past year (compared to more than half of active SPMS patients) frequently compelled by patients' progression of clinical disability. With most switches going to an oral DMT, not active SPMS patients are most likely to currently be treated with Genzyme's Aubagio, Novartis' Gilenya, or Biogen's Tecfidera. As a result, the ICER's comparators of interest, which include Genentech's Ocrevus and Rituxan, Biogen's Tysabri, and interferons, may not be as relevant for gauging siponimod's effectiveness and value in the not active SPMS segment. In addition, the not active SPMS landscape could evolve in the not so distant future with anticipated pivotal trial read outs over the next 18 months for MedDay Pharmaceuticals' Qizenday (MD-1003) and AB Science's masitinib, both of which are in development specifically for the segment.
Turning to the primary progressive MS (PPMS) segment, in their Q3 2018 earnings press release, Sanofi announced that they would not be pursuing a PPMS indication for Lemtrada (marketed by the Genzyme subsidiary in the US). With Ocrevus being the only approved therapy for the treatment of PPMS, it is not surprising that the majority of the over 300 DMT-treated PPMS audit patients were treated with the brand. However, data from the Phase 3 ORATORIO trial of Ocrevus versus placebo found no disability progression benefit in women. In our audit, collaborating US neurologists who believed that Ocrevus is more effective in male compared to female PPMS patients prescribed significantly more Lemtrada and less Ocrevus to their PPMS patients. In addition, more than half of neurologists who prescribed monoclonal antibody (mAb) DMTs to their PPMS patients used two or more mAb DMTs suggesting that neurologists believe that other mAb DMTs, like Lemtrada, could have efficacy within this patient type and challenge market-leading Ocrevus ─ a belief that could only have been strengthened with a robust clinical program.
*Mitoxantrone is approved for SPMS but not commonly used due to toxicity concerns.
RealWorld Dynamix™: Progressive Forms of Multiple Sclerosis (US) blends attitudinal and demographic physician survey data (n=184) with patient record data (n=755) to uncover how practice type and setting and certain beliefs influence the progressive MS treatment pathway and to understand how marketed DMTs are being used by physicians and for what patient types. The report also captures physician's perspectives about products in development and the impact they will have on the current treatment paradigm among patients with progressive forms of multiple sclerosis. Parallel audits include the third annual RealWorld Dynamix™: DMT New Starts in Multiple Sclerosis (US), publishing in February 2019, and the fourth annual RealWorld Dynamix™: DMT Switching in Multiple Sclerosis (US), publishing in April 2019.
About Spherix Global Insights
Spherix Global Insights is an independent business intelligence and market research company, specializing in renal, autoimmune, neurologic and rare disease markets. Our aim is to apply our commercial experience and unique relationships within core specialty markets to translate data into insight, enabling our clients to make smarter business decisions.
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SOURCE Spherix Global Insights