- Eight abstracts accepted, including three
oral presentations
- Maralixibat data highlighting predictors of
event-free survival in patients with Alagille syndrome nominated
for prestigious Alex Mowat Prize for best oral presentation in
hepatology
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) will
present data at the upcoming International Liver Congress™ of the
European Association for the Study of the Liver (EASL) and the 54th
Annual European Society for Paediatric Gastroenterology,
Hepatology, and Nutrition Annual Meeting (ESPGHAN), taking place in
London and Copenhagen, respectively. The Company will present data
from its LIVMARLI® (maralixibat) clinical studies and will unveil a
multi-national survey of patient-reported outcomes data assessing
pruritus and overall disease burden in adults with primary
sclerosing cholangitis.
Mirum will also host a scientific symposium during each
meeting:
- EASL: “Patient, caregiver, and
physician: Understanding perspectives in adult and pediatric
cholestasis”
- Thursday, June 23, 2022 from 6:30-8:00 p.m. BST
- ESPGHAN: “Cholestatic Disease in
Children: From Diagnosis to Patient Management”
- Thursday, June 23, 2022 from 6:15-7:15 p.m. CEST
**ESPGHAN: June 22-25, 2022,
Copenhagen, Denmark**
All abstracts are available via the Journal of Paediatric
Gastroenterology and Nutrition and the page number for each
abstract is enclosed with the title below.
Oral presentations
H-O-008: Maralixibat treatment response is associated with
improved health-related quality of life in patients with bile salt
export pump (BSEP) deficiency (pg. 650) Oral presentation: June
23, 2022 at 5:00 p.m. CEST Presenter: Dr. Douglas Mogul (on behalf
of Dr. Kathy Loomes)
H-O-012: Predictors of 6-year event-free survival in patients
with Alagille syndrome treated with maralixibat, an IBAT inhibitor
(pg. 658) Oral presentation: June 24, 2022 at 8:45 a.m. CEST
**Nominated for the Alex Mowat Prize for Best Oral Presentation in
Hepatology** Presenter: Dr. Ronald Sokol
H-O-018: Maralixibat-treated patients with Alagille syndrome
(ALGS) demonstrate improved event-free survival in a natural
history comparison with patients from the GALA database:
application of real-world evidence analytics (pg. 670) Oral
presentation: June 24, 2022 at 9:45 a.m. CEST Presenter: Dr.
Bettina Hansen
Poster presentations
H-P-058: Maralixibat improves growth in patients with
Alagille syndrome: a 4-year analysis (pg. 778) Poster: June 24,
2022 from 2:00-2:45 p.m. CEST Authors: Dr. Binita Kamath, et al
H-P-057: Response to treatment with maralixibat in Alagille
syndrome is associated with improved health-related quality of life
(pg. 776) Poster Walk: June 24, 2022 at 2:00-2:45 p.m. CEST
Author: Dr. Binita Kamath, et al
H-P-047: Significant improvement in cholestatic pruritus in
patients with Alagille syndrome treated with maralixibat, an ileal
bile acid transporter inhibitor: Real-world experience (pg.
763) Poster Walk: June 24, 2022 from 2:00-2:45 p.m. CEST
Authors: Dr. Regino Gonzalez-Peralta and Dr. Doug Mogul
**International Liver Congress – EASL:
June 22-26, 2022, London, United Kingdom**
All abstracts are available via the EASL website and the page
number for each abstract is enclosed with the title below.
Poster Presentations
Poster 634: Dosing ileal bile acid transporter
inhibitors in the fasted state minimizes gastrointestinal adverse
effects while maintaining pharmacodynamic effect (pg. S599)
Author: Dr. Cory Kostrub, et al.
Poster 756: Impact of pruritus in primary sclerosing
cholangitis (PSC): a multinational survey (pg. S312) Author:
Dr. Kris Kowdley, et al.
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered,
once-daily, ileal bile acid transporter (IBAT) inhibitor approved
by the U.S. Food and Drug Administration for the treatment of
cholestatic pruritus in patients with Alagille syndrome (ALGS) one
year of age and older and is the only FDA-approved medication to
treat cholestatic pruritus associated with Alagille syndrome. For
more information, please visit LIVMARLI.com.
LIVMARLI is currently being evaluated in late-stage clinical
studies in other rare cholestatic liver diseases including
progressive familial intrahepatic cholestasis (PFIC) and biliary
atresia. LIVMARLI has received Breakthrough Therapy designation for
ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and
biliary atresia. LIVMARLI is not approved for use in PFIC or
biliary atresia. To learn more about ongoing clinical trials with
LIVMARLI, please visit Mirum’s clinical trials section on the
company’s website.
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause serious side effects, including:
Changes in liver tests. Changes in certain liver tests
are common in patients with Alagille syndrome and can worsen during
treatment with LIVMARLI. These changes may be a sign of liver
injury and can be serious. Your healthcare provider should do blood
tests 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) or loss of
appetite.
Stomach and intestinal (gastrointestinal) problems.
LIVMARLI can cause stomach and intestinal problems, including
diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with
Alagille syndrome but may worsen during treatment. Your healthcare
provider should do blood tests before starting and during
treatment.
Other common side effects reported during treatment were bone
fractures and gastrointestinal bleeding.
Prescribing information
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 IBAT 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 Phase 2b clinical trial),
intrahepatic cholestasis of pregnancy (OHANA Phase 2b clinical
trial), and primary biliary cholangitis (VANTAGE Phase 2b clinical
trial).
About Mirum Pharmaceuticals
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company
dedicated to transforming the treatment of rare liver diseases.
Mirum’s approved medication is LIVMARLI® (maralixibat) oral
solution which is approved in the U.S. for the treatment of
cholestatic pruritus in patients with Alagille syndrome one year of
age and older.
Mirum’s late-stage pipeline includes two investigational
treatments for debilitating liver diseases affecting children and
adults. LIVMARLI, an oral ileal bile acid transporter (IBAT)
inhibitor, is currently being evaluated in clinical trials for
pediatric liver diseases and includes the MARCH Phase 3 clinical
trial for progressive familial intrahepatic cholestasis (PFIC) and
the EMBARK Phase 2b clinical trial for patients with biliary
atresia. In addition, Mirum has an expanded access program open
across multiple countries for eligible patients with ALGS and
PFIC.
Mirum has submitted a Marketing Authorization Application to the
European Medicines Agency for LIVMARLI for the treatment of
cholestatic liver disease in patients with Alagille syndrome.
Mirum’s second investigational treatment, volixibat, an oral
IBAT inhibitor, is being evaluated in three potentially
registrational studies including the VISTAS Phase 2b clinical trial
for adults with primary sclerosing cholangitis, the OHANA Phase 2b
clinical trial for pregnant women with intrahepatic cholestasis of
pregnancy, and the VANTAGE Phase 2b clinical trial for adults with
primary biliary cholangitis.
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Forward-Looking Statements
This press release includes forward-looking statements
pertaining to the Company’s planned participation at a scientific
conference, which may include discussion of the Company’s clinical
and research data, including the discovery, development and
commercialization of our product candidates and technologies, and
the therapeutic potential thereof, the continuation of our clinical
trials, and the success of our collaborations with partners and any
potential future collaborations. Such forward-looking statements
are subject to risks and uncertainties that could cause actual
results to differ materially from those expressed or implied in
such statements. Applicable risks and uncertainties include those
relating to our preclinical research and clinical programs and
other risks identified under the heading “Risk Factors” included in
our most recent Form 10-Q and Form 10-K filings and in other future
filings with the SEC. The forward-looking statements contained in
this press release reflect Mirum's current views with respect to
future events, and Mirum does not undertake and specifically
disclaims any obligation to update any forward-looking statements,
except as required by law.
View source
version on businesswire.com: https://www.businesswire.com/news/home/20220613005346/en/
Media Contact: Erin Murphy media@mirumpharma.com
Investor Contacts: Ian Clements, Ph.D. ir@mirumpharma.com
Sam Martin Argot Partners ir@mirumpharma.com
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