Promising results regarding safety, efficacy, and side effects are generally needed at each major phase of development.
Sometimes they approve it for a subtype, like highly-active MS (HAMS, inside RRMS), rapidly-worsening MS (RWMS, inside PPMS), or "active SPMS" (previous progressive-relapsing)[1] As of 2021[update], the approved drugs for relapsing-remitting multiple sclerosis (RRMS) are: There are reports comparing these treatments in front of each other.
Besides, the regulatory agencies treat sometimes apart the cases aSPMS (Active Secondary progressive), nSPMS(non-active SPMS), HAMS (highly active) and RPMS (rapidly progressive) Phase III programs consist of studies on large patient groups (300 to 3,000 or more) and are aimed at being the definitive assessment of how effective and safe a test drug will be.
In July 2021, the FDA gave the go-ahead for an investigational new drug application (IND) for the phase 3 ENSURE program, which will evaluate IMU-838 in patients with relapsing-remitting multiple sclerosis (RRMS).
Immunic also announced that a separate IND application for the supportive phase 2 CALLIPER trial of IMU-838 in patients with progressive multiple sclerosis has been cleared as well.
The ENSURE program consists of two identical, double-blind, twin phase 3 trials, titled ENSURE-1 and ENSURE-2, designed to evaluate the efficacy, safety, and tolerability of IMU-838 in a 30-mg daily dose versus placebo in patients with RRMS.
Approximately 1050 adult patients with active RRMS are expected to be enrolled in the studies and will be evaluated on time to first relapse as the primary end point.
Both trials will run concurrently, with dosing of the first patient expected in the second half of 2021.2[32] Phase II studies are performed on mid-sized groups of patients (20 to 300) and are designed to assess whether a drug works in the targeted disease area, as well as to continue earlier safety assessments obtained in healthy volunteers.
Cyclophosphamide (Revimmune) is in Phase III trials for secondary progressive MS.[78] It was also studied for RRMS but the company does not pursue actively this path.
[81] Some PPMS patients with a special biomarker (Immunoglobulin M oligoclonal bands) have been shown to respond to standard RRMS medications, though there is only preliminary evidence waiting to be confirmed[82] Several combinations of drugs have been tested.
[145] Lymphocyte subpopulations in peripheral blood is a promising tool to select RRMS candidate for fingolimod treatment.