- Maralixibat demonstrates significant transplant-free survival
for patients with progressive familial intrahepatic cholestasis
(PFIC2) who achieved serum bile acid (sBA) control in five-year
analysis (p=0.0006).
- sBA responders remain on maralixibat for more
than five years with improvements across multiple parameters
including normalization of liver enzyme and bilirubin levels,
decreased pruritus and improvement in growth.
Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical
company focused on the development and commercialization of novel
therapies for debilitating liver diseases, presented an analysis
from its Phase 2 INDIGO study in an oral late-breaker session at
the Digital International Liver Congress™ 2020. The five-year
analysis showed that patients with PFIC2, also known as bile salt
export pump (BSEP) deficiency, who achieved sBA control on
long-term maralixibat treatment have a significant improvement in
transplant-free survival.
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“The maralixibat data demonstrate a well-tolerated, long-term
treatment option to address PFIC2, which may provide patients with
not only a viable treatment alternative to liver transplantation,
but also a possible path to addressing many of the quality of life
challenges that can make PFIC a burdensome disease for patients and
their families,” said Richard Thompson, professor of molecular
hepatology at King’s College London, who presented the data.
Late-breaker oral presentation
August 29, 2020 – 2:45-3:00pm CET LB008: Serum bile acid
control in long-term maralixibat-treated patients is associated
with native liver survival in children with progressive familial
intrahepatic cholestasis due to bile salt export pump
deficiency.
In February 2020, the NAPPED consortium published an analysis
showing that surgical interruption of the enterohepatic circulation
of bile acids can lead to transplant-free survival when sBAs are
reduced by 75% or below 102 µmol/L. Data presented today
demonstrate that, similar to the findings of NAPPED, patients
exhibiting sBA control with pharmacological interruption of the
enterohepatic circulation with maralixibat have significantly
improved transplant-free survival (p=0.0006). Seven of the 19
non-truncating patients with PFIC2 achieved this response
threshold. In addition, the data showed that maralixibat responders
achieved improvement across numerous parameters including quality
of life scores, normalization of liver enzyme and bilirubin levels
if abnormal at baseline, decreased pruritus and accelerated
growth.
“These data further underscore the potential for maralixibat as
an alternative to liver transplantation in children with PFIC2,
addressing a major unmet medical need for a very burdensome
disease,” said Chris Peetz, president and chief executive officer
at Mirum. “These children are in dire need of an effective
pharmacologic treatment option, and we want to ensure patients have
appropriate treatments beyond surgery. Given these efficacy data
and the encouraging safety profile, we are planning to file a
marketing authorization application in Europe this year with the
hope of bringing maralixibat to patients with PFIC2 as quickly as
possible.”
Long-term treatment with maralixibat was well-tolerated. The
most frequent treatment-emergent adverse events (TEAEs) were
nasopharyngitis, vomiting, cough, diarrhea, pyrexia and abdominal
pain. The majority of TEAEs were mild to moderate in severity and
transient.
To view the presentation as well as other maralixibat posters
presented during the meeting, please visit the Publications and
Presentations section of our corporate website.
About the Maralixibat Phase 2 INDIGO Study
The INDIGO Phase 2 study was an open-label study evaluating the
long-term treatment effects of pharmacological interruption of
enterohepatic circulation with maralixibat in children with PFIC.
INDIGO included 19 PFIC2 patients with non-truncating BSEP
mutations who received maralixibat 280 µg/kg once or twice daily.
Study endpoints were sBA, pruritus, quality of life, safety and
tolerability.
About Maralixibat
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. The FDA has granted
maralixibat Breakthrough Therapy designation for 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, abdominal pain, and vomiting. The North American
Expanded Access Program for ALGS is planned to open for
registration in September 2020. For more information, please visit
ALGSEAP.com. For more information about the Phase 3 study for
maralixibat in pediatric patients with PFIC, visit
PFICtrial.com.
About Mirum Pharmaceuticals
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. The company’s lead product candidate,
maralixibat, is an investigational oral drug in development for
Alagille syndrome (ALGS), progressive familial intrahepatic
cholestasis (PFIC), and biliary atresia. The company is also
developing volixibat, also an oral ASBT-inhibitor, in primary
sclerosing cholangitis and intrahepatic cholestasis of pregnancy.
For more information, visit MirumPharma.com. Follow Mirum on
Twitter, Facebook and LinkedIn.
Forward-Looking Statements
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 results, conduct, progress and
timing of Mirum’s ongoing and planned studies for maralixibat and
volixibat, as well as Mirum’s Expanded Access Program for
maralixibat and the regulatory approval path for 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 “plans,” “will”, “believes,” “anticipates,” “expects,”
“intends,” “goal,” “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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version on businesswire.com: https://www.businesswire.com/news/home/20200829005005/en/
Investor Contact: Ian Clements, Ph.D. ir@mirumpharma.com
Media Contact: Erin Murphy media@mirumpharma.com
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