– 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 –
NEW
YORK, Oct. 9, 2023 /PRNewswire/ -- 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
* The only approved 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."
"The 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."
"We 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 stronger
results."
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.
CALLIPER 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).
Anticipated MS Clinical Milestones
- Top-line data from the phase 2 CALLIPER trial of vidofludimus
calcium in PMS is expected in April of 2025.
- Data from the interim analysis of the phase 3 ENSURE program of
vidofludimus calcium in relapsing MS is expected in late 2024, with
the top-line readout of the first of the ENSURE trials at the end
of 2025.
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.
Webcast Information
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
Multiple
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.
About Vidofludimus Calcium (IMU-838)
Vidofludimus
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.
About Immunic, Inc.
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 trials for the treatment of relapsing and progressive
multiple sclerosis, respectively, and has shown therapeutic
activity in phase 2 clinical trials in patients suffering from
relapsing-remitting multiple sclerosis and moderate-to-severe
ulcerative colitis. Vidofludimus calcium combines neuroprotective
effects, through its mechanism as a first-in-class nuclear receptor
related 1 (Nurr1) activator, with additional anti-inflammatory and
anti-viral effects, by selectively inhibiting the enzyme
dihydroorotate dehydrogenase (DHODH). IMU-856, which targets the
protein Sirtuin 6 (SIRT6), is intended to restore intestinal
barrier function and regenerate bowel epithelium, which could
potentially be applicable in numerous gastrointestinal diseases,
such as celiac disease, where it is currently in preparations for a
phase 2 clinical trial. IMU-381, which currently is in preclinical
testing, is a next generation molecule being developed to
specifically address the needs of gastrointestinal diseases. For
further information, please visit: www.imux.com.
Cautionary Statement Regarding Forward-Looking
Statements
This press release contains "forward-looking
statements" that involve substantial risks and uncertainties for
purposes of the safe harbor provided by the Private Securities
Litigation Reform Act of 1995. All statements, other than
statements of historical facts, included in this press release
regarding strategy, future operations, future financial position,
future revenue, projected expenses, sufficiency of cash, expected
timing, development and results of clinical trials, prospects,
plans and objectives of management are forward-looking statements.
Examples of such statements include, but are not limited to,
statements relating to Immunic's development programs and the
targeted diseases; the potential for vidofludimus calcium to safely
and effectively target diseases; preclinical and clinical data for
vidofludimus calcium; the timing of current and future clinical
trials and anticipated clinical milestones; the nature, strategy
and focus of the company and further updates with respect thereto;
and the development and commercial potential of any product
candidates of the company. Immunic may not actually achieve the
plans, carry out the intentions or meet the expectations or
projections disclosed in the forward-looking statements and you
should not place undue reliance on these forward-looking
statements. Such statements are based on management's current
expectations and involve substantial risks and uncertainties.
Actual results and performance could differ materially from those
projected in the forward-looking statements as a result of many
factors, including, without limitation, the COVID-19 pandemic,
increasing inflation, impacts of the Ukraine – Russia conflict on planned and ongoing
clinical trials, risks and uncertainties associated with the
ability to project future cash utilization and reserves needed for
contingent future liabilities and business operations, the
availability of sufficient financial and other resources to meet
business objectives and operational requirements, the fact that the
results of earlier preclinical studies and clinical trials may not
be predictive of future clinical trial results, the protection and
market exclusivity provided by Immunic's intellectual property,
risks related to the drug development and the regulatory approval
process and the impact of competitive products and technological
changes. A further list and descriptions of these risks,
uncertainties and other factors can be found in the section
captioned "Risk Factors," in the company's Annual Report on Form
10-K for the fiscal year ended December 31,
2022, filed with the SEC on February
23, 2023, and in the company's subsequent filings with the
Securities and Exchange Commission. Copies of these filings are
available online at www.sec.gov or ir.imux.com/sec-filings.
Any forward-looking statement made in this release speaks only as
of the date of this release. Immunic disclaims any intent or
obligation to update these forward-looking statements to reflect
events or circumstances that exist after the date on which they
were made. Immunic expressly disclaims all liability in respect to
actions taken or not taken based on any or all the contents of this
press release.
Contact Information
Immunic, Inc.
Jessica Breu
Head of Investor Relations and Communications
+49 89 2080 477 09
jessica.breu@imux.com
US IR Contact
Rx Communications Group
Paula Schwartz
+1 917 633 7790
immunic@rxir.com
US Media Contact
KOGS Communication
Edna Kaplan
+1 617 974 8659
kaplan@kogspr.com
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SOURCE Immunic, Inc.