Restoring Health Growth Life

Creating Transformative Medicine for Children
With Rare Cholestatic Liver Disease

About Our Company Name

Mirum is a Latin word that means extraordinary, amazing or remarkable. We chose Mirum as our company name because it speaks to the impact we hope we can make for patients and their families.

About Us

Mirum Pharmaceuticals is dedicated to creating life-changing therapies for patients with liver diseases. We are advancing our lead product candidate for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) — conditions with a profound and devastating impact on quality of life for young patients and their families.

Untreated, these conditions can lead to liver failure and inflict life-altering symptoms including stunted growth and severe, non-stop itching. Under current standard of care, patients often require a liver transplant and are at higher risk of many serious health issues. Working closely with patients and caregiver communities, we intimately understand the challenges of living with cholestatic liver diseases.

Based on a wealth of data from clinical trials spanning multiple years and involving more than one hundred patients, we believe our approach can address the underlying cause of symptoms and liver damage. Our goal is to give families their lives back.

Mike Grey
Executive Chairman

Leadership

Mirum has assembled some of the world’s top experts in liver disease and pharmaceutical development.

Chris Peetz

Chief Executive Officer

Chris Peetz is chief executive officer and board member of Mirum Pharmaceuticals, bringing deep experience in therapeutic development and commercialization. He also serves as an entrepreneur-in-residence… more

Pamela Vig, Ph.D.

Chief Scientific Officer

Pamela Vig, Ph.D. is chief scientific officer for Mirum Pharmaceuticals, where she leads scientific and clinical aspects of drug development. Pam brings 25 years of industry experience… more

Lara Longpre

Chief Development Officer

Lara Longpre is the chief development officer for Mirum Pharmaceuticals, where she leads development operations, program management and alliance management. Lara brings to Mirum… more

Thomas Jaecklin, M.D. and M.Sc.

Senior Vice President, Clinical Development

Thomas Jaecklin, M.D. and M.Sc. is senior vice president of clinical development and the medical lead of the pediatric cholestasis development program at Mirum Pharmaceuticals… more

Sherin Halfon, Ph.D.

Vice President, CMC and Project Management

Sherin Halfon, Ph.D.  leads Chemistry, Manufacturing, and Controls (CMC) at Mirum Pharmaceuticals. Sherin has worked in the biopharmaceutical industry. Prior to joining Mirum… more

Board of Directors

Mike Grey

Executive Chairman, Mirum Pharmaceuticals

Chris Peetz

Chief Executive Officer, Mirum Pharmaceuticals

Ed Mathers

Partner, New Enterprise Associates

Patrick Heron

Managing General Partner, Frazier Healthcare Partners

Jonathan Leff

Partner, Deerfield Management

Tiba Aynechi, Ph.D.

Partner, Novo Ventures

Niall O’Donnell, Ph.D.

Managing Director, RiverVest

Investors

 

Pipeline

MARALIXIBAT

PHASE 1

PHASE 2

PHASE 3

Progressive Familial Intrahepatic Cholestasis (PFIC)

Phase 3 initiation in first half of 2019

Phase 3

Alagille Syndrome (ALGS)

Phase 3 initiation in the second half of 2019

Phase 2

VOLIXIBAT

PHASE 1

PHASE 2

PHASE 3

Adult Cholestasis

Indication to be selected in 2019

Phase 1

About Maralixibat

Maralixibat is an orally administered therapy being evaluated as a treatment for children with rare cholestatic liver diseases, including Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Both of these cholestatic diseases involve an interruption in the flow of bile acid from the liver, resulting in excess bile acid in the liver and systemically. This leads to liver disease and a variety of severe and life-altering symptoms, including stunted growth and chronic and severe itch. Maralixibat works by inhibiting an important carrier protein known as the apical sodium dependent bile acid transporter (ASBT), thereby reducing systemic levels of bile acids.

Encouraged by promising clinical trial data, Mirum Pharmaceuticals is now advancing maralixibat in late-stage clinical trials. To date, more than 100 patients have received the therapy as an investigational treatment for cholestatic liver diseases.

Among patients with ALGS, recent clinical trial data has shown that maralixibat reduces severe itching (also known as pruritus), an often-debilitating symptom of the disease. Results from the Phase 2 INDIGO study of maralixibat in PFIC patients supported Breakthrough Therapy designation from the U.S. Food and Drug Administration for PFIC2.

Mirum is preparing to initiate separate Phase 3 studies for pediatric patients with ALGS and PFIC in 2019. Mirum also is exploring maralixibat as a treatment for other cholestatic liver diseases including biliary atresia.

About Volixibat

Volixibat is an investigational oral inhibitor of ASBT, a protein that is primarily responsible for recycling bile acids from the intestine to the liver. Volixibat has been evaluated in dose ranging studies and Mirum plans to develop it in adult liver diseases.

Patients / Families

Mirum is focused on developing a safe and effective treatment for children with rare cholestatic liver diseases. What we want, more than anything, is to enable patients and their families to live a healthy and happy life, liberated from around-the-clock disruptions of disease.

But we don’t just work on behalf of patients, we work with them. Through clinical trials for our lead program, maralixibat, patients and their families play an integral role in helping us evaluate the therapy and obtain the strong, detailed evidence we need for regulatory approval in the U.S. and beyond.

We are currently developing maralixibat for two rare liver conditions: Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) .

Kira (center, with her family) received miralixibat as part of a clinical trial for ALGS.

Therapeutic Areas

Alagille Syndrome (ALGS)

ALGS is a rare genetic condition that can affect many systems of the body including the liver, heart, skeleton, eyes and kidneys. Patients have fewer than the normal number of small bile ducts inside the liver, which can interfere with liver function and the digestive system. Symptoms and severity of the disease can vary greatly from one person to another. Symptoms often emerge during the first three months of life, although the disease can be difficult to diagnose because it is so rare. Patients often experience cholestasis, or blockage of the flow of bile from the liver, as well as symptoms such as jaundice, poor weight gain and growth, severe itching (pruritus) and pale, loose stools.

Symptoms often interfere with sleep for patients and their families and can significantly affect quality of life in a multitude of other ways. Bile acid accumulation leads to progressive liver damage, with many patients needing a liver transplant. Current standard of care involves an invasive and disfiguring surgical procedure or liver transplant. No disease-modifying drug therapy exists today.

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 secrete bile, leading to a buildup of bile that causes severe and constant itching that becomes a major life disruption for the patients and their families. Itching typically begins in infancy along with other symptoms including jaundice, failure to gain weight and grow at the expected rate (failure to thrive), high blood pressure in the vein that supplies blood to the liver (portal hypertension), and an enlarged liver and spleen (hepatosplenomegaly). Current standard of care involves an invasive and disfiguring biliary diversion surgery. No disease-modifying drug therapy exists today.

Clinical Trials

Mirum’s investigational therapy maralixibat has been tested rigorously in multiple Phase 1 and Phase 2 clinical studies. These studies have yielded extensive safety and efficacy data to support its further development. Phase 3 studies will begin in 2019.

Enrolling for Alagille Syndrome (ALGS)

In 2019, Mirum will begin enrolling patients in a Phase 3 clinical trial to study the efficacy of maralixibat in treating children with ALGS. For more information about how to enroll, please contact us at clinicaltrials@mirumpharma.com.

Enrolling for Progressive Familial Intrahepatic Cholestasis (PFIC)

In 2019, Mirum will begin enrolling patients in a Phase 3 clinical trial to study the efficacy of maralixibat in treating children PFIC.

Join Our Mission

Mirum Pharmaceuticals is driven by a mission to create transformative medicine for children with rare cholestatic liver disease. Our growing team brings a long history of liver disease expertise, and we are very passionate about the patient population we serve. As we expand, we continue to welcome other passionate and talented individuals who share our desire to create new possibilities in medicine.

We prioritize our people, and offer a competitive compensation package with a rewarding work environment that fosters innovation. Are you ready to make a difference? If you think Mirum may be the right place for you, but didn’t find a perfect job match on our website, please submit your resume to careers@mirumpharma.com.

Opportunities

Program Manager

© Copyright 2019 - Mirum Pharmaceuticals

Mike Grey
Executive Chairman

Mike Grey is executive chairman of Mirum Pharmaceuticals, a company he founded in 2018 to continue the work he began seven years earlier with Mirum’s predecessor company, Lumena Pharmaceuticals. Mike formed Lumena in 2011 to develop treatments for rare liver diseases, and the company was acquired in 2014 by Shire Plc. When Shire de-prioritized the programs, Grey spun them out into a focused company that could build on extensive safety data and proof of efficacy for two rare liver diseases: ALGS and PFIC.

In addition to leading Mirum, Mike is executive chairman of Amplyx Pharmaceuticals, Reneo Pharmaceuticals and Spruce Biosciences, as well as venture partner at Pappas Capital. He has 40 years of experience in the pharmaceutical and biotechnology industries, and has held senior positions at a number of companies, including president and CEO of Auspex Pharmaceuticals, president and CEO of SGX Pharmaceuticals (acquired by Eli Lilly), president and CEO of Trega Biosciences (acquired by Lion Bioscience), and president of BioChem Therapeutic. For approximately 20 years, Mike served in various roles with Glaxo and Glaxo Holdings plc, culminating as vice president, corporate development and head of international licensing. Mike serves on the board of directors of BioMarin Pharmaceutical, Horizon Pharma plc, and Mirati Therapeutics. Mike holds a Bachelor of Science in chemistry from University of Nottingham.

Chris Peetz
Chief Executive Officer

Chris Peetz is chief executive officer and board member of Mirum Pharmaceuticals, bringing deep experience in therapeutic development and commercialization. He also serves as an entrepreneur-in-residence at Frazier Healthcare Partners, focusing on liver diseases and related therapeutic areas.

Previously, served as chief financial officer and head of corporate development at Tobira Therapeutics, a leading developer of therapeutics to treat non-alcoholic steatohepatitis (NASH). While at Tobira, he led the company’s efforts to become a public company through a reverse merger, expand its pipeline through partnerships and in-licensing, and sell the company to Allergan for up to $1.7 billion. Prior to Tobira, Chris held business leadership roles at Jennerex Biotherapeutics, Onyx Pharmaceuticals and Abgenix. Chris holds a Bachelor of Science in Business Administration from Washington University in St. Louis and an MBA from the Stanford Graduate School of Business.

Pamela Vig, Ph.D.
Chief Scientific Officer

Pamela Vig, Ph.D. is chief scientific officer for Mirum Pharmaceuticals, where she leads scientific and clinical aspects of drug development.

Pam brings 25 years of industry experience in clinical development, medical affairs and translational science in various liver diseases including HBV, HCV, NASH and cholestatic liver disease, as well as HIV and other infectious diseases. Most recently, she has held leadership roles at Allergan and Tobira Therapeutics as VP of clinical research and discovery, as well as VP of clinical research at Presidio Pharmaceuticals. Pam previously served as director, global medical affairs, at Johnson & Johnson in London, UK, and also held positions at Idenix Pharmaceuticals and Gilead Sciences. Pam holds a Ph.D. from Imperial College London in the departments of hepatology and histopathology.

Lara Longpre
Chief Development Officer

Lara Longpre is the chief development officer for Mirum Pharmaceuticals, where she leads development operations, program management and alliance management. Lara brings to Mirum more than 25 years of experience developing innovative and complex therapies, most recently as chief operating officer at MedGenesis Therapeutix and Jennerex Biotherapeutics where she held responsibilities for program management, alliance management, clinical operations, technical operations, regulatory affairs, and quality assurance. She previously held several leadership roles with PRA International and CroMedica including operations, regulatory affairs, and proposals & contracts.

Lara holds a Bachelor of Arts in biology from Cornell University, a Master of Medical Science in from Harvard Medical School, and an MBA from Queen’s University in Ontario, Canada.

Shelly Xiong, Ph.D.
Senior Vice President, Regulatory

Shelly Xiong, Ph.D., is senior vice president of regulatory affairs for Mirum Pharmaceuticals. Shelly brings to Mirum more than 25 years of experience in the biopharmaceutical industry. Prior to joining Mirum, Shelly served as a senior vice president of regulatory affairs at Eiger BioPharmaceuticals and held multiple leadership roles in regulatory affairs at InterMune and Gilead Sciences and in operations at Covance. Shelly also has extensive experience in drug discovery at Gilead Sciences. She is an author on over 30 publications and primary inventor on several patents. She holds a Bachelor of Science in chemistry from Peking University in Beijing, China, a Master of Science in chemistry from University of Minnesota, and a Ph.D. in biochemistry from University of Wisconsin-Madison.

Sherin Halfon, Ph.D.
Vice President, CMC and Project Management

Sherin Halfon, Ph.D. leads Chemistry, Manufacturing, and Controls (CMC) at Mirum Pharmaceuticals. Sherin has worked in the biopharmaceutical industry, including Asterias Biotherapeutics, Presidio Pharmaceuticals and Relypsa, spanning more than 25 years in both scientific and management roles. She holds a BA in chemistry from the University of Chicago, and a PhD in Organic Chemistry from Columbia University.

Thomas Jaecklin, M.D. and M.Sc.
Senior Vice President of Clinical Development

Thomas Jaecklin, M.D. and M.Sc. is senior vice president of clinical development and the medical lead of the pediatric cholestasis development program at Mirum Pharmaceuticals.

Thomas brings 20 years of academic and industry experience in several pediatric rare disease indications having recently served as senior medical director of the maralixibat program at Shire Pharmaceuticals, before it was acquired by Mirum Pharmaceuticals. Prior to Shire, Thomas was medical director at Novartis Pharma AG for strategic business development and several pediatric rare-disease development programs. Thomas is Swiss board-certified in pediatrics, neonatology and critical care medicine. Thomas holds an M.D. from the University of Geneva and an M.Sc., in medical sciences from the University of Toronto.