Full Press Release Details
Positive Outcome of Interim Analysis of Phase 3 ENSURE Program of Vidofludimus Calcium in Relapsing Multiple Sclerosis
- Based on a Review of
Unblinded Data, an Independent Data Monitoring Committee (IDMC) Confirmed that Predetermined Futility Criteria Have Not Been Met -
- IDMC Also Recommended
Continuing Trial without Changes, Including no Need for a
Potential Upsizing -
- ENSURE Program Remains
on Track to be Completed in 2026 -
- Webcast to be Held Today,
October 22, at 8:00 am ET -
NEW YORK, October 22, 2024 - 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 a positive outcome of the non-binding, interim futility analysis of
its phase 3 ENSURE program, investigating lead asset, nuclear receptor related 1 (Nurr1) activator, vidofludimus calcium (IMU-838), for
the treatment of relapsing multiple sclerosis (RMS). Based on the outcome of the interim futility analysis, an unblinded Independent Data
Monitoring Committee (IDMC) has recommended that the trials are not futile and should continue as planned.
"While Immunic remains blinded to all data,
the IDMC's favorable recommendations in this interim analysis corroborate our initial assumptions for the design, powering and relapse
rate of the twin phase 3 trials of vidofludimus calcium in RMS, and suggest that they are in line with the data observed so far,"
stated Andreas Muehler, M.D., M.B.A., Chief Medical Officer of Immunic. "In particular, the planned sample size seems appropriate
to address the primary endpoint of time to first relapse. As the IDMC recommends, we are continuing the ENSURE trials unchanged, with
completion expected in 2026."
excited about the positive outcome of the interim analysis of our phase 3 ENSURE trials, marking the successful achievement of a critical
milestone for the program," added Daniel Vitt, Ph.D., Chief Executive Officer of Immunic. "We are confident in vidofludimus
calcium's potential to transform the oral MS market and continue to believe that the phase 3 program provides a clear and straightforward
path towards seeking potential regulatory approval in RMS. Our next clinical milestone for vidofludimus calcium is the top-line readout
of our phase 2 CALLIPER trial in patients with progressive multiple sclerosis (PMS), which we expect to release in April of next year.
If this data set continues to show a neuroprotective effect for vidofludimus calcium, we believe our drug may be positioned as first-in-class
oral treatment option for PMS, a form of MS with highest unmet medical needs."
The interim futility analysis of the phase 3 ENSURE
program was performed by an unblinded IDMC and based on a pre-specified assessment after approximately half of the planned first relapse
events occurred in the double-blind treatment periods of each of the twin ENSURE-1 and ENSURE-2 trials. The analysis was intended to inform
potential sample size adjustment and help prevent the final study readout from occurring before sufficient events have been achieved.
The unblinded IDMC was asked to make two decisions: The first question, as to whether the trials are futile, was answered by the IDMC
with "futility criteria have not been met." The second question, as to whether the sample size in each trial should be increased,
was answered by the IDMC with "continue as planned." Both decisions were based on the conditional power of the trials at the
time of the interim analysis. Immunic has remained blinded during the interim analysis and has not seen any of the data available to the
IDMC to make their recommendations.
The ongoing ENSURE program
comprises two identical multicenter, randomized, double-blind phase 3 trials designed to evaluate the efficacy, safety and tolerability
of vidofludimus calcium versus placebo in RMS patients. Each of the trials, titled ENSURE-1 and ENSURE-2, is expected to enroll approximately
1,050 adult patients with active RMS at more than 100 sites in more than 15 countries, including the United States, India and countries
in Latin America, Central and Eastern Europe. Patients are being randomized in a double-blinded fashion to either 30 mg daily doses of
vidofludimus calcium or placebo and the primary endpoint for both trials is time to first relapse up to 72 weeks. Key secondary endpoints
include time to confirmed disability worsening based on Expanded Disability Status Scale (EDSS) disability progression, volume of new
T2-lesions, time to sustained clinically relevant changes in cognition, and percentage of whole brain volume change, grey matter volume
and white matter volume. As previously reported, completion of ENSURE-1 is anticipated in the second quarter of 2026, with completion
of ENSURE-2 expected in the second half of 2026.
webcast today at 8:00 am ET. To participate in the webcast, please register in advance at: https://imux.zoom.us/webinar/register/WN_fSJNHWuxRMGRPaMl3hUlqg
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.
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 highly selective 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 1,800 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 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, progressive 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, for which 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 and cash runway, 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; announcements regarding
the positive outcomes of the interim analysis of the phase 3 ENSURE trials; 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 and the conflict in the Middle
East on planned and ongoing clinical trials, risks and uncertainties associated with the ability to project future cash utilization
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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
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