Clinical Study for Progressive Familial
Intrahepatic Cholestasis (PFIC)

Mirum Pharmaceuticals is striving to bring transformative medicines to patients
who have rare cholestatic liver conditions. In keeping with this mission, we have
initiated a Phase 3 study called MARCH to evaluate the safety and efficacy of
maralixibat in pediatric patients with progressive familialintrahepatic cholestasis (PFIC).
Eligible participants in this clinical study may help provide new insights into PFIC
and could lead to the first approved drug therapy.

Clinical Study for Progressive Familial Intrahepatic Cholestasis (PFIC)

Mirum Pharmaceuticals is striving to bring transformative medicines to patients who have rare cholestatic liver conditions. In keeping with this mission, we have initiated a Phase 3 study called MARCH to evaluate the safety and efficacy of maralixibat in pediatric patients with progressive familial intrahepatic cholestasis (PFIC). Eligible participants in this clinical study may help provide new insights into PFIC and could lead to the first approved drug therapy.

About

What is Progressive Familial Intrahepatic Cholestasis (PFIC)?

PFIC is a rare genetic condition that causes progressive liver disease, often leading to liver failure and requiring a liver transplant. In patients with PFIC, liver cells are less able to eliminate bile, leading to a buildup of bile which causes itching so severe and constant that it becomes a major life disruption for the patients and their families. Itching typically begins in infancy along with other symptoms including jaundice, failure to grow at the expected rate (failure to thrive), and increasingly severe liver disease. Current standard of care involves an invasive and disfiguring surgery (external biliary diversion) or liver transplantation. There is currently no approved drug therapy to treat PFIC or pruritus associated with PFIC.

The MARCH Study

Mirum has recently initiated a Phase 3 study called MARCH (MARalixibat in CHolestasis) to evaluate the safety and efficacy of a drug called maralixibat as a potential novel treatment for pediatric patients with PFIC. The study is enrolling 30 to 90 participants ages 1 to 17 years old to assess the change in itch severity and frequency, as well as reduction of bile acids in the blood. Learn more about MARCH and to see if your child is eligible.

Maralixibat

Maralixibat is an orally-administered liquid. Maralixibat is an investigational medicine being evaluated as a treatment for children with rare cholestatic liver diseases, including PFIC.

Maralixibat works by blocking an important bile acid transport protein on the surface of the small bowel, thereby reducing systemic levels of bile acids.

Encouraged by promising Phase 2 clinical data, Mirum Pharmaceuticals is now advancing maralixibat into Phase 3 clinical studies. To date, more than 119 children and 69 adults have received maralixibat as a treatment for their cholestatic liver diseases, many of them for more than 4 years. Maralixibat has also been tested in > 1400 subjects in other studies.

Results from the Phase 2 INDIGO study supported maralixibat’s Breakthrough Therapy Designation for the treatment of PFIC2 from the US FDA. Breakthrough Therapy designation is a process designed to expedite the development and review of drugs by the FDA that are intended to treat a serious condition and preliminary clinical evidence indicates that the drug may demonstrate substantial improvement over available therapy.

Who Can Take Part?

Your child may be eligible for the MARCH study if they:

Have been diagnosed with PFIC, carry the gene that causes the disease and are 1-17 years old
Have elevated bile acids in the blood and are moderately to severely itchy
Do not have presence of any other disease/condition known to interfere with the drug; are not experiencing severe diarrhea; and do not have a previous or planned liver transplant

For questions about the trial please contact:

medinfo@mirumpharma.com (U.S & International [non-Europe])
medinfoEU@mirumpharma.com (Europe)

Your child must meet all other study criteria to take part in the MARCH study. See the full list of eligibility criteria.

I Would Like to Learn More About the MARCH Clinical Study

Where is the nearest MARCH Clinical Study site to me?

Trial Locations

We have open sites in North America, Europe, Middle East, Asia and Latin America.

Americas Europe/Middle East Asia/Pacific

FAQs

What are clinical trials?

It can take up to 10 years to develop a new medication. During this process plenty of information to learn if and how a new medication works need to be gathered before doctors are allowed to prescribe it. This information is collected in clinical research studies (also known as clinical trials) that test investigational medications (also known as study drugs) in humans. Each study follows a plan (or “protocol”) that has been reviewed and approved by various groups of people, including health authorities, like the Food and Drug Administration (FDA) in the United States, as well as scientists, doctors, lawyers, and members of the public.

Why do people participate in clinical trials?

Clinical trial participants can play a more active role in their health care, gain access to new research treatments before they are widely available and help others by contributing to medical research.

What are the different phases of clinical trials?

There are four phases of clinical trials. The trials at each phase have a different purpose and help answer different questions.

Phase I – test a new drug or treatment in a small group of people for the first time in order to evaluate the treatment’s safety, how much can be given safely, and to identify possible side effects from the treatment.
Phase II – typically test the treatment in a larger group of people in order to see if the treatment is effective and to further evaluate the safety of the treatment. Patients are usually given either active medication or placebo without knowing which one they are taking.
Phase III – typically test the treatment in a large group of people to confirm that the treatment is effective, monitor side effects, compare the treatment to other commonly used treatments, and collect additional information which should help allow safe use of the treatment. Again, patients are usually given either active medication or placebo without knowing which one they are taking.
Phase IV – are usually studies that are conducted once a drug is on the market and establish additional information about the treatment’s risks, benefits, and best use of the treatment.

The number of people included in each stage of a trial increases from one stage to the next, with the total number in a given trial depends on the type of disease the trial is addressing. For example, a clinical trial for a rare disease like PFIC would enroll far fewer patients than a clinical trial for a common condition that impacts millions of people, such as heart disease or diabetes.

What is an investigational medication?

An investigational medication is a drug that is not approved for marketing by the health authorities. It is generally only available for use in clinical research studies to determine its safety and effectiveness.

How often does PFIC occur?

Approximately 3,000 people in the United States and 5,000 people in Europe are affected by PFIC. Approximately 60% of PFIC patients are estimated to be PFIC2.

Are there other available therapeutics for PFIC?

There is currently no therapy approved for PFIC and available medical approaches have limited efficacy. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues.

Who is conducting the MARCH clinical study?

Mirum Pharmaceuticals Inc. is a clinical-stage therapeutics company developing a novel approach for treating liver diseases, with an immediate focus on rare pediatric conditions. The company’s lead product candidate, maralixibat, is an investigational orally-administered drug with an established safety profile and efficacy data in several indications, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). Mirum is dedicated to bringing innovative medicines to patients as quickly and efficiently as possible. For more information, visit www.mirumpharma.com.

Clinical Study for Progressive FamilialIntrahepatic Cholestasis (PFIC)