/ CASE STUDY

Doubling Down on Rare Liver Disease

RiverVest re-invests in drug it helped develop, brings it back into the clinic

Mirium-2-1

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

After the drug compound they developed was deprioritized, the Lumena team bought it back so they could finish what they started – getting kids with rare liver disease the relief they need.

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.

RiverVest Role

In addition to the financing, RiverVest Managing Director Niall O’Donnell helped reclaim the Lumena drug (maralixibat), provided scientific guidance to get the approval process back on track, and helped assemble Mirum’s world-class leadership team to bring the drug to market.

(listening time: 1:22 min)

“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

THE UNMET 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.

MIRUM’S SOLUTION

LIVMARLI® (maralixibat) is an oral ileal bile acid transporter (IBAT) inhibitor, approved in the U.S. in September 2021 for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

 

Mirum is advancing LIVMARLI and another IBAT inhibitor, volixibat, across five additional late-stage trials for high-need rare cholestatic liver diseases in children and adults.

Voice of the Patient

Patients are the real experts on their disease; after all, they live with and struggle through its impacts every minute of every day.  That is why it is incumbent upon researchers and drug developers to listen to and learn from patients and their caregivers.

 

Learn about the realities of living with Alagille Syndrome by watching Kira’s Story, now available on PBS’s Medical Stories.

 

Watch the PBS documentary here.

“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

Better Patient Outcomes

Initiated by Lumena, the Global ALagille Alliance (GALA) Study has become a role 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.

(listening time: 2:00 min)

Real-World Data

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.

The Details

After the drug compound they developed was deprioritized, the Lumena team bought it back so they could finish what they started – getting kids with rare liver disease the relief they need. Here’s how this deal came together.

 

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), and 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, a minimally absorbed ileal bile acid transporter inhibitor, 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 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.

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