Full Press Release Details
Immunic Reports Positive
Interim Data from Phase 2 CALLIPER Trial of Vidofludimus Calcium in Progressive Multiple Sclerosis
24-Week Data from First Half of Patients Shows Improvements in Biomarker NfL, Consistent Throughout the Overall Progressive Multiple Sclerosis
Population as well as All Subtypes -
NfL Effect in Non-Active Subpopulation Reinforces Vidofludimus Calcium's Neuroprotective Potential -
Brain Volume Data of the Full 467 CALLIPER Patient Cohort Expected in April 2025 -
Phase 3 ENSURE Program in Relapsing Multiple Sclerosis Ongoing -
Conference Call and Webcast to be Held Tomorrow, October 10, 2023 at 8:00 am ET -
October 9, 2023 - Immunic, Inc. (Nasdaq: IMUX), a biotechnology company
developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory and autoimmune diseases, today
announced positive interim data from its phase 2 CALLIPER trial of nuclear receptor related 1 (Nurr1)
activator, vidofludimus calcium (IMU-838), in patients with progressive multiple sclerosis (PMS). The Company believes that this data
shows biomarker evidence that vidofludimus calcium's activity extends beyond the previously observed anti-inflammatory effects,
thereby further reinforcing its neuroprotective potential.
The predefined interim analysis examined the change
from baseline to 24 weeks in serum neurofilament light chain (NfL) and glial fibrillar acidic protein (GFAP) levels among approximately
the first half of patients enrolled in this trial. Serum NfL results are as follows:
| Total N | 45 mg vidofludimus calcium versus placebo | ||
| Change in serum NfL at 24 weeks in % of baseline as compared to placebo | All PMS | N=203 | -22.4% |
| PPMS* | N=59 | -18.8% | |
| Non-Active SPMS | N=124 | -20.1% | |
| Active SPMS | N=20 | -43.3% |
PPMS: primary progressive multiple sclerosis;
SPMS: secondary progressive multiple sclerosis
drug for PPMS, Ocrevus (ocrelizumab), showed a 12.4% reduction versus placebo in 24-week serum NfL levels in the phase 3 ORATORIO
trial (Bar-Or A. et al., EBioMedicine. 2023 Jul;93:104662).
Serum NfL responses were consistently observed
for vidofludimus calcium across progressive MS disease and all subpopulations. In the overall PMS population at 24 weeks (N=203), vidofludimus
calcium was associated with a 6.7% reduction from baseline in serum NfL, compared to a
15.8% increase over baseline in placebo (p=0.01, post hoc). At 48 weeks (N=79), vidofludimus calcium reduced
serum NfL by 10.4% from baseline, compared to a 6.4%
increase in placebo. Substantial reductions were also seen across all PMS subtypes,
as well as in patients that show or do not show disease and/or magnetic resonance imaging (MRI) activity.
Although early, interim
GFAP data also showed a promising signal: at 24 weeks (N=203), GFAP increased by 3.7% for vidofludimus calcium, and 4.4% for placebo.
At 48 weeks (N=79), the change was only 2.7% for vidofludimus calcium, with a 6.4% increase for placebo. Progression of GFAP response
is generally thought to evolve more slowly than NfL, and the Company believes that a longer follow-up may further strengthen this signal.
"Serum NfL has been consistently shown to
capture disease activity and to predict future disability in MS. Vidofludimus calcium shows a separation in serum NfL over placebo in
this interim analysis, an effect also seen across different subgroups," stated Prof. Jens Kuhle, M.D., Ph.D., Senior Physician,
Head of Neuroimmunology Unit and Multiple Sclerosis Centre, University Hospital Basel, Switzerland. "Particularly remarkable, the
non-active progressive MS population, which represents the highest unmet medical need in MS, also showed differences in NfL levels over
this relatively short observation period in favor of vidofludimus calcium. Meanwhile, although longer follow-up is needed, the GFAP data
set also shows a potential promising early signal. Overall, the interim biomarker data further support vidofludimus calcium's possible
activity beyond an anti-inflammatory effect, which may be related to its potent Nurr1 activation."
clear separation observed in serum NfL for vidofludimus calcium over placebo in the PMS patient
population represents another major step forward for, what potentially could be, a first-in-class
Nurr1 activator for MS," commented Daniel Vitt, Ph.D., Chief Executive Officer and
President of Immunic. "Although no head-to-head data is available, it is encouraging to see that vidofludimus calcium's improvement
in NfL over placebo appears at least as good as, and is in fact numerically higher than that observed with historical studies of other
therapeutic approaches for PMS. We believe that, if the top-line CALLIPER data, expected
in April of 2025, continue to show a neuroprotective effect, we may be able to position vidofludimus calcium as the first oral treatment
option for non-active SPMS. Additionally, the drug's first-in-class ability to activate
Nurr1, a known neuroprotective target, should also significantly benefit our ongoing phase 3 ENSURE
program in relapsing MS where prevention of disability progression independent of relapse
activity (PIRA), serves as a key outcome."
are very pleased to see such strong improvements in serum NfL for vidofludimus calcium over placebo in the overall PMS population
of this interim analysis, as well as across all PMS subtypes and in patients with and without disease
activity, and with and without MRI activity. We even saw evidence in
non-active SPMS, a population where the medical need for new therapies is high as there is currently no relevant treatment available in
the US," added Andreas Muehler, M.D., Chief Medical Officer of Immunic. "Finally, we were also excited to see an encouraging
early signal with GFAP. This is a newer biomarker which is thought to evolve more slowly and with lower amplitude than NfL, and longer
follow-up will hopefully allow us to see even
The Company believes that these results corroborate
separate findings from its phase 2 EMPhASIS trial in relapsing-remitting multiple sclerosis (RRMS), where vidofludimus calcium was associated
with a decrease in serum NfL at 24 weeks (-17.0% for 30 mg and -20.5% for 45 mg) as compared to baseline values, as contrasted with a
6.5% increase in serum NfL over baseline among placebo patients.
is a multicenter, randomized, double-blind, placebo-controlled phase 2 trial which enrolled 467 patients with primary PMS or active or
non-active secondary PMS at more than 70 sites throughout North America as well as Western, Central and Eastern Europe. Patients were
randomized to either 45 mg daily doses of vidofludimus calcium or placebo, and the trial's primary endpoint is the annualized rate
of percent brain volume change up to 120 weeks. Key secondary endpoints include the annualized rate of change in whole brain atrophy and
time to 24-week confirmed disability progression based on the expanded disability status scale (EDSS).
MS Clinical Milestones
For more information on the phase 2 CALLIPER trial,
please visit: www.clinicaltrials.gov, NCT05054140.
The interim data of the phase 2 CALLIPER trial
of vidofludimus calcium in PMS will be filed on a Form 8-K and discussed during the management presentation to be held tomorrow at 8:00
am ET. The presentation will also be accessible on the "Events and Presentations" section of Immunic's website at: https://ir.imux.com/events-and-presentations.
Immunic will host a webcast tomorrow, October
10, 2023, at 8:00 am ET to discuss these results. To participate in the webcast, please register in advance at: https://imux.zoom.us/webinar/register/WN_evpnpdOKQX2AKEXpCacoUA
or on the "Events and Presentations" section of Immunic's website at: ir.imux.com/events-and-presentations.
Registrants will receive a confirmation email containing a link for online participation or a telephone number for dial in access.
An archived replay of the webcast will be available
approximately one hour after completion on Immunic's website at: ir.imux.com/events-and-presentations.
About Progressive Multiple Sclerosis
sclerosis (MS) is an autoimmune disease that affects the brain, spinal cord and optic nerve. In MS, myelin, the coating that protects
the nerves, is attacked and damaged by the immune system. Thus, MS is considered an immune-mediated demyelinating disease of the central
nervous system. Progressive multiple sclerosis (PMS) includes both primary progressive MS (PPMS) and secondary progressive MS (SPMS).
PPMS is characterized by steadily worsening neurologic function from the onset of symptoms without initial relapse or remissions. SPMS
is identified following an initial relapsing-remitting course, after which the disease becomes
more steadily progressive, with (active SPMS) or without (non-active SPMS) other
disease activity present.
calcium is a small molecule investigational drug in development as an oral next-generation treatment option for patients with multiple
sclerosis and other chronic inflammatory and autoimmune diseases. The selective immune modulator activates the neuroprotective
transcription factor nuclear receptor related 1 (Nurr1), which is associated with direct neuroprotective properties. Additionally,
vidofludimus calcium is a known inhibitor of the enzyme dihydroorotate dehydrogenase (DHODH), which is a key enzyme in the metabolism
of overactive immune cells and virus-infected cells. This mechanism is associated with the anti-inflammatory and anti-viral effects of
vidofludimus calcium. Vidofludimus calcium has been observed to selectively act on hyperactive T and B cells while leaving other immune
cells largely unaffected and enabling normal immune system function, e.g., in fighting infections. To date, vidofludimus calcium has been
tested in more than 1,400 individuals and has shown an attractive pharmacokinetic, safety and tolerability profile. Vidofludimus calcium
is not yet licensed or approved in any country.
Immunic, Inc. (Nasdaq:
IMUX) is a biotechnology company developing a clinical pipeline of orally administered, small molecule therapies for chronic inflammatory
and autoimmune diseases. The company's lead development program, vidofludimus calcium (IMU-838), is currently in phase 3 and phase 2 clinical