Revolutionary Treatment for Liver Disease: Mirum's Livmarli

/ CASE STUDY

Mirum Pharmaceuticals: Getting patients with rare liver disease the relief they need

/ BIOPHARMA COMPANY / IPO – JULY 2019 (NASDAQ: MIRM)

Mirum was founded upon a drug treatment that RiverVest helped develop through Lumena – a Fund II company acquired by Shire in 2014.

THE SNAPSHOT

THE IMPACT

LIVMARLI, a minimally absorbed ileal bile acid transporter inhibitor, is the first and only FDA- approved medication for treating a rare liver disease that affects 2,000 to 2,500 children in the United States. Mirum is advancing LIVMARLI and volixibat, also acquired from Shire, across five additional late-stage trials for high-need rare cholestatic liver diseases.

OUR ROLE

After Shire deprioritized maralixibat trials in 2018, RiverVest helped reclaim the asset, provided scientific guidance to get the approval process back on track, and assembled a world-class leadership team to bring the drug to market.

THE NEED

Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC) are severe genetic diseases affecting 4,500 children in the U.S. and U.K. Most children with these diseases suffer from debilitating itching that disrupts sleep, stunts growth, and compels them to scratch themselves sometimes to the bone. The toll this takes on the psychological and social well-being of these children and their families is devastating. Many will require biliary diversion surgery or liver transplantation. Others will die from liver failure.

CEO Chris Peetz discusses Mirum’s robust pipeline

RiverVest helped build the company (Lumena) that initiated these studies and was instrumental in pulling together Mirum’s Series A financing, advising us through an IPO, and supporting our success as a public company.

CHRIS PEETZ, CEO,  MIRUM PHARMACEUTICALS

Dr. Binita Kamath discusses the impact of GALA

A GAME-CHANGING STUDY

The Lumena team had the foresight to initiate a global database described by principal investigator Dr. Binita Kamath as a model for Academia and Industry collaboration. More than 100 physicians, scientists, and research coordinators from 35 countries share clinical, genetic, and laboratory data from ALGS patients in what has become The Global ALagille Alliance (GALA) Study.

This type of research doesn’t get funded from the standard federal agencies. It just doesn’t. And it’s been game changing!

DR. BINITA KAMATH, PRINCIPAL INVESTIGATOR, GALA STUDY

The Story

Mirum was founded upon a drug treatment (maralixibat) that RiverVest helped develop through Lumena – a Fund II company acquired by Shire in 2014.

After acquiring Lumena, Shire continued to run the randomized withdrawal study on maralixibat, the oral drug being evaluated in children with Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC).

Former Lumena CEO Mike Grey and CMO Niall O’Donnell – a RiverVest Managing Director – remained in touch with the families and physicians. By all accounts, the treatment was working!

But Shire – now part of Takeda – deprioritized the program in 2018 before launching a Phase III clinical study. Undeterred, the Lumena team bought back the asset and brought on Chris Peetz and his team to move maralixibat swiftly through FDA approval (based on additional analyses of the ALGS Phase II data) and a successful commercial launch.

Mirum went public in July 2019, and LIVMARLI was approved by the FDA in September 2021 for the treatment of cholestatic pruritus in patients with ALGS one year of age and older.

LIVMARLI, a minimally absorbed ileal bile acid transporter inhibitor, is the first and only FDA-approved medication for treating this rare liver disease that affects 2,000 to 2,500 children in the United States.

In conjunction with the approval, Mirum received a rare pediatric disease priority review voucher to apply toward speedier FDA review of one of its other products.

Mirum is advancing LIVMARLI and volixibat, also acquired from Shire, across five additional late-stage trials for high-need rare cholestatic liver diseases.

Read the latest Mirum news here.

Six years of data emerging from the GALA Study demonstrate significant, sustained event-free survival outcomes with maralixibat treatment vs. natural history in patients with ALGS.