- Data from five years of treatment with
maralixibat reinforces safety and tolerability in patients with
- Intrahepatic cholestasis of pregnancy
patient-reported data demonstrates significant burden of disease
and underscores urgent need for new treatment options.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced the
presentation of analyses from its rare liver disease programs
during the EASL International Liver Congress™. The posters being
presented feature an integrated safety analysis of maralixibat in
patients with Alagille syndrome (ALGS) and the unveiling of a
multi-national survey of patient reported outcomes from pregnant
women with intrahepatic cholestasis of pregnancy (ICP). The posters
are now available on the congress website.
Abstract PO-1285: An
integrated analysis of long-term clinical safety in
maralixibat-treated participants with Alagille syndrome
Data from more than five years of maralixibat treatment across
three Phase 2 clinical studies (and their extension studies) were
analyzed to assess the overall clinical safety of maralixibat in
patients with ALGS (n=86). The analysis evaluated
treatment-emergent adverse events (TEAEs) and laboratory parameters
including reported rates, severity and seriousness, actions taken
with maralixibat (i.e., dose reductions and discontinuations), time
to onset and potential dose-response relationships. A sub-analysis
of safety data was also conducted evaluating data from 13-week
The analysis concluded:
- Maralixibat was well-tolerated for more than five years.
- The most common TEAEs were diarrhea and abdominal pain.
- Mild to moderate gastrointestinal events (GI events) were
observed in the first weeks of treatment, were transient, and
lasted less than one week in duration.
- In placebo-controlled studies, GI events occurred at similar
rates between maralixibat and placebo in the background of
- There were no discontinuations of maralixibat due to diarrhea
or abdominal pain.
- No clinically significant trends or patterns in laboratory
measures were observed.
- ALT levels were consistent with natural history comparisons of
Abstract PO-2657: Patient perspectives on pruritus in
intrahepatic cholestasis of pregnancy: a multinational
The multinational survey was conducted in collaboration with ICP
Support, a leading patient advocacy group focused on ICP and based
in the United Kingdom.
The survey evaluated the burden of cholestatic pruritus, the
impact on quality of life, and the effectiveness of available
treatments as reported by women who have or have had ICP (n=688).
Participants in the survey were asked to assess severity of their
itch, sleep disturbance, and impact of medications received, using
a numerical rating scale (NRS) of 0-10, with 0 representing none
and 10 representing the worst imaginable, for each symptom.
Responses from the survey demonstrated that pruritus has
debilitating effects on patients living with ICP, including a
substantial impact on sleep and quality of life. The results found
- The median worst itch reported was a 9 out of 10 on the
- 94% of women reported itch-related sleep disturbances, which
were associated with degree of itch severity.
- Itch was associated with mood changes and often led to a
disruption of day-to-day responsibilities and routines.
- 71% of women reported that they were not asked about itch by
their healthcare provider.
- A majority of women reported taking ≥2 medications; however,
most reported either only partial or no resolution of ICP-related
These responses underscore the significant impact pruritus can
have on women with ICP as well as the high unmet need for the
development of safe and effective therapies to treat this rare
“This integrated safety analysis of maralixibat with more than
five years of evaluation across 86 patients with ALGS continues to
underscore the safety and tolerability profile of maralixibat for
use in this chronic disease setting,” said Pam Vig, Ph.D., chief
scientific officer at Mirum. “We are also excited to showcase the
ICP survey data which demonstrates the significant burden that
pruritus can have on these women, and the tremendous need for new
therapies. As we continue to enroll patients in our OHANA study, we
remain hopeful that volixibat’s potential to reduce cholestasis and
pruritus associated with ICP may improve quality of life.”
“Understanding the severity of pruritus and the impact on a
woman’s quality of life whilst pregnant is of critical importance
to advancing research and identifying a new treatment option for
ICP,” said Jenny Chambers, chief executive officer, ICP Support.
“We surveyed nearly 700 women globally to assess the scale of how
debilitating this condition can be and to champion research that
will help us better understand the effects of the condition. We
hope this in turn will lead to improved outcomes for women with
this uncommon and unrelenting liver disease.”
Maralixibat is a novel, minimally absorbed, orally administered
investigational drug being evaluated in several rare cholestatic
liver diseases. Maralixibat inhibits the apical sodium dependent
bile acid transporter (ASBT), resulting in more bile acids being
excreted in the feces, leading to lower levels of bile acids
systemically, thereby potentially reducing bile acid mediated liver
damage and related effects and complications. More than 1,600
individuals have received maralixibat, including more than 120
children who have received maralixibat as an investigational
treatment for Alagille syndrome (ALGS) and progressive familial
intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS
clinical trial, patients taking maralixibat had significant
reductions in bile acids and pruritus compared to placebo, as well
as reduction in xanthomas and accelerated growth long-term. In a
Phase 2 PFIC study, a genetically defined subset of BSEP deficient
(PFIC2), patients responded to maralixibat with an increase in
transplant-free survival. The U.S. Food and Drug Administration has
granted maralixibat Breakthrough Therapy designation for the
treatment of pruritus associated with ALGS in patients one year of
age and older and for PFIC2. Maralixibat was generally
well-tolerated throughout the studies. The most frequent
treatment-related adverse events were diarrhea and abdominal pain.
Maralixibat has been studied extensively and its safety database
represents the largest database for an ASBT inhibitor.
Until maralixibat is approved and available for prescribing, the
medication is available to patients with ALGS through Mirum’s
expanded access program. For more information, please visit
ALGSEAP.com. For further information about maralixibat’s ongoing
studies in pediatric liver disease, please visit the study
websites: Phase 3 MARCH study for PFIC and Phase 2b EMBARK study
for biliary atresia.
Volixibat is an oral, minimally absorbed agent designed to
selectively inhibit the apical sodium dependent bile acid
transporter (ASBT). Volixibat may offer a novel approach in the
treatment of adult cholestatic diseases by blocking the recycling
of bile acids, through inhibition of ASBT, 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 intrahepatic
cholestasis of pregnancy (OHANA study). Mirum plans to initiate a
Phase 2b study for primary biliary cholangitis later this year.
About Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc. is a clinical-stage
biopharmaceutical company focused on the development and
commercialization of a late-stage pipeline of novel therapies for
debilitating liver diseases. Mirum’s lead product candidate,
maralixibat, is an investigational oral drug in development for
Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia. Mirum has submitted an NDA
for maralixibat in the treatment of cholestatic pruritus in
patients with ALGS. The NDA has been accepted for priority review
by the FDA with a PDUFA action date of September 29, 2021.
Additionally, Mirum’s marketing authorization application for the
treatment of pediatric patients with PFIC2 has been accepted for
review (validated) by the European Medicines Agency. Mirum is also
developing volixibat, also an oral ASBT-inhibitor, in primary
sclerosing cholangitis, intrahepatic cholestasis of pregnancy, and
primary biliary cholangitis. For more information, visit
To augment its pipeline in cholestatic liver disease, Mirum has
acquired the exclusive option to develop and commercialize gene
therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2,
respectively, from Vivet Therapeutics SAS, following preclinical
evaluation and investigational new drug-enabling studies.
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Statements contained in this press release regarding matters
that are not historical facts are “forward-looking statements”
within the meaning of the Private Securities Litigation Reform Act
of 1995. Such forward-looking statements include statements
regarding, among other things, the potential benefits and an
assessment on the severity of side effects of maralixibat and
volixibat. 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.
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