- VANTAGE PBC interim analysis shows 3.8 point
reduction from baseline and 2.3 point placebo-adjusted (p=0.0026)
reduction in primary endpoint of pruritus
- VISTAS PSC interim analysis exceeds efficacy
threshold for study continuation
- Mirum to host conference call to discuss
analyses, today, June 17 at 8:30 a.m. ET/5:30 a.m. PT
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced
interim results from two Phase 2b studies evaluating volixibat, an
oral ileal bile acid transporter (IBAT) inhibitor in patients with
primary biliary cholangitis (PBC) and primary sclerosing
cholangitis (PSC).
Interim results from the VANTAGE study evaluating volixibat in
patients with PBC demonstrated a statistically significant (-3.82,
p<0.0001) improvement in pruritus for volixibat and a
placebo-adjusted difference of -2.32 points in the primary
endpoint, p=0.0026, as measured by the Adult ItchRO scale. 75% of
patients on volixibat achieved a greater than 50% reduction in
serum bile acids. In addition, there was a significant improvement
in fatigue at week 16 with volixibat compared to placebo.
No new safety signals were observed, and adverse events were
similar between the 20 mg and 80 mg treatment groups. The most
common adverse event was diarrhea (77%) with all cases mild to
moderate, and mostly transient; one case resulted in
discontinuation. Four patients experienced serious adverse events,
including one in the placebo arm. There were no clinically
meaningful changes in liver biomarkers.
Adult ItchRO Change from Baseline
Mean change in Adult ItchRO
score*
20 mg
(n=10)
80 mg
(n=10)
20+80 mg
(n=20)
PBO
(n=10)
LSMean (SE)
P-value
-3.84 (0.609)
<0.0001
-3.79 (0.564)
<0.0001
-3.82 (0.414)
<0.0001
-1.50 (0.585)
0.0149
Difference between VLX and PBO
P-value
-2.34
0.0090
-2.29
0.0075
-2.32
0.0026
*Adult ItchRO is a 0-10 numerical rating
scale; MMRM weekly averaged worst daily itch score, assessed over
weeks 17-28 of the treatment period.
Based on these results, the VANTAGE PBC trial will continue with
a volixibat dose of 20 mg twice daily.
Concurrently, the interim analysis for the VISTAS PSC study was
conducted and the independent data review committee recommended
that the study continue with the selected volixibat dose of 20 mg
twice daily, with no changes to the study. The criteria for
continuation included safety as well as a predefined threshold for
efficacy. The sponsor and investigators are blinded to the interim
results and analysis.
“The interim data from the VANTAGE study provide outstanding
results in relation to what has been shown for treatment of
pruritus in PBC,” said Joanne Quan, MD, chief medical officer at
Mirum. “With both VISTAS and VANTAGE advancing to enroll their
confirmatory portions, we are excited about volixibat as a
potential future option to help patients overcome one of the most
prevalent and burdensome symptoms of these rare liver
diseases.”
“The results of the interim analyses are very impressive as they
confirm the potential of volixibat in targeting bile acids in PBC
and PSC,” said Kris Kowdley, MD, Washington State University and an
investigator for VANTAGE and VISTAS. “I look forward to seeing the
final data with the goal of having additional therapies available
to address the burden of disease in adult cholestasis.”
“The symptomatic burden in PBC is significant and often an
underappreciated aspect of this disease. Both itch and fatigue are
devastating hallmarks of PBC that can significantly decrease
quality of life,” said Carol Roberts, president of the PBCers
Organization. “It is incredibly encouraging for PBC patients to see
such promising results with volixibat.”
Conference Call to Discuss Interim Analysis
Mirum will be hosting a conference call to discuss the interim
analyses from VISTAS and VANTAGE today, Monday, June 17 at 8:30
a.m. ET/5:30 a.m. PT. Join the call by dialing (404) 975-4839
(local/int’l) or (833) 470-1428 (toll-free) and using the access
code: 205511. You may also access the webcast through Mirum’s
Investor Relations website.
About Volixibat
Volixibat is an oral, minimally absorbed agent designed to
selectively inhibit the ileal bile acid transporter (IBAT).
Volixibat may offer a novel approach in the treatment of adult
cholestatic diseases by blocking the recycling of bile acids,
through inhibition of IBAT, thereby reducing bile acids
systemically and in the liver. Phase 1 and Phase 2 studies of
volixibat demonstrated on-target fecal bile acid excretion, a
pharmacodynamic marker of ASBT inhibition, in addition to decreases
in LDL cholesterol and increases in 7αC4 which are markers of bile
acid synthesis. Volixibat has been evaluated in more than 400
individuals across multiple clinical trials. The most common
adverse events reported were mild to moderate gastrointestinal
events observed in the volixibat groups. Volixibat is currently
being evaluated in Phase 2b studies for primary sclerosing
cholangitis (VISTAS study), and primary biliary cholangitis
(VANTAGE study).
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company
dedicated to transforming the treatment of rare diseases affecting
children and adults. Mirum has three approved medications:
LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid)
capsules, and CHENODAL® (chenodiol) tablets.
LIVMARLI, an IBAT inhibitor, is approved for the treatment of
two rare liver diseases affecting children and adults. It is
approved for the treatment of cholestatic pruritus in patients with
Alagille syndrome in the U.S. (three months and older), in Europe
(two months and older), and in other regions globally. It is also
approved in the U.S. in cholestatic pruritus in PFIC patients five
years of age and older and in Europe the Committee for Medicinal
Products for Human Use (CHMP) has adopted positive opinion of
LIVMARLI to treat patients with PFIC three months and older. A
decision by the European Commission is expected in the third
quarter 2024. CHOLBAM is FDA-approved for the treatment of bile
acid synthesis disorders due to single enzyme deficiencies and
adjunctive treatment of peroxisomal disorders in patients who show
signs or symptoms or liver disease. CHENODAL has received medical
necessity recognition by the FDA to treat patients with
cerebrotendinous xanthomatosis (CTX).
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases. Volixibat, an IBAT
inhibitor, is being evaluated in two potentially registrational
studies including the Phase 2b VISTAS study for primary sclerosing
cholangitis and Phase 2b VANTAGE study for primary biliary
cholangitis. Lastly, CHENODAL has been evaluated in a Phase 3
clinical study, RESTORE, to treat patients with CTX, with positive
topline results reported in 2023.
To learn more about Mirum, visit mirumpharma.com and follow
Mirum on Facebook, LinkedIn, Instagram and Twitter (X).
LIVMARLI® (maralixibat) Oral Solution IMPORTANT
SAFETY INFORMATION
Limitation of Use: LIVMARLI is not for use in PFIC type 2
patients who have a severe defect in the bile salt export pump
(BSEP) protein.
LIVMARLI can cause side effects, including:
Liver injury. Changes in certain liver tests are common
in patients with Alagille syndrome and PFIC but can worsen during
treatment. These changes may be a sign of liver injury. In PFIC,
this can be serious or may lead to liver transplant or death. Your
healthcare provider should do blood tests and physical exams before
starting and during treatment to check your liver function. Tell
your healthcare provider right away if you get any signs or
symptoms of liver problems, including nausea or vomiting, skin or
the white part of the eye turns yellow, dark or brown urine, pain
on the right side of the stomach (abdomen), bloating in your
stomach area, loss of appetite or bleeding or bruising more easily
than normal.
Stomach and intestinal (gastrointestinal) problems.
LIVMARLI can cause stomach and intestinal problems, including
diarrhea and stomach pain. Your healthcare provider may advise you
to monitor for new or worsening stomach problems including stomach
pain, diarrhea, blood in your stool or vomiting. Tell your
healthcare provider right away if you have any of these symptoms
more often or more severely than normal for you.
A condition called Fat Soluble Vitamin (FSV) Deficiency
caused by low levels of certain vitamins (vitamin A, D, E, and K)
stored in body fat is common in patients with Alagille syndrome and
PFIC but may worsen during treatment. Your healthcare provider
should do blood tests before starting and during treatment and may
monitor for bone fractures and bleeding which have been reported as
common side effects.
US Prescribing Information EU SmPC
Forward-Looking Statements
This press release contains “forward-looking statements" within
the meaning of Section 27A of the Securities Act of 1933, as
amended, and Section 21E of the Securities Exchange Act of 1934, as
amended. Such forward-looking statements include those regarding
the clinical potential and regulatory process for volixibat, the
ability for volixibat to impact bile acids or pruritus in patients
with PSC or PBC, and the effectiveness of volixibat in a real-world
population, if ever approved by the FDA. Because such statements
are subject to risks and uncertainties, actual results may differ
materially from those expressed or implied by such forward-looking
statements. Words such as “will,” “could,” “would,” “potential” and
similar expressions are intended to identify forward-looking
statements. These forward-looking statements are based upon Mirum’s
current expectations and involve assumptions that may never
materialize or may prove to be incorrect. Actual results could
differ materially from those anticipated in such forward-looking
statements as a result of various risks and uncertainties, which
include, without limitation, risks and uncertainties associated
with Mirum’s business in general, the impact of the COVID-19
pandemic, and the other risks described in Mirum’s filings with the
Securities and Exchange Commission. All forward-looking statements
contained in this press release speak only as of the date on which
they were made and are based on management’s assumptions and
estimates as of such date. Mirum undertakes no obligation to update
such statements to reflect events that occur or circumstances that
exist after the date on which they were made, except as required by
law. A further description of risks and uncertainties can be found
in Mirum’s Annual Report on Form 10-K for the fiscal year ended
December 31, 2023 and in its subsequent reports on Form 10-Q,
including in the sections thereof captioned “Risk Factors,” as well
as in its subsequent reports on Form 8-K, all of which are filed
with the U.S. Securities and Exchange Commission and available at
www.sec.gov.
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version on businesswire.com: https://www.businesswire.com/news/home/20240617178174/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contact: Andrew McKibben ir@mirumpharma.com
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