/ CASE STUDY

Lumena Pharmaceuticals: Treating life-threatening pediatric liver diseases

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/ BIOPHARMA COMPANY / ACQUIRED BY SHIRE IN 2014, SOLD TO MIRUM IN 2019

RiverVest co-founded and seed funded Lumena and built a clinical strategy to fast-track its lead drug compound through clinical trials.

THE SNAPSHOT

THE IMPACT

Drawing on deep scientific expertise and relationships, the Lumena team tackled a significant unmet need and paved the way for safe, effective oral treatments for life-threatening liver diseases. Lumena was acquired by Shire less than 3 years after formation, delivering ~$400 million to investors.  The story continues with Mirum.

OUR ROLE

RiverVest Managing Director Niall O’Donnell developed rapid proof-of-concept clinical programs and led interactions with U.S. and European regulatory bodies. RiverVest’s John McKearn joined Lumena’s board of directors and Niall became acting chief medical officer.

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.

NIALL O’DONNELL ON RIVERVEST PUTTING PATIENTS FIRST

Niall dug into the science and developed the clinical plans while I assumed the role of CEO. This marked the beginning of a partnership that spans six early-stage biopharma companies … and counting!

MIKE GREY, BOARD CHAIRMAN, MIRUM PHARMACEUTICALS

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A NEW WAY OF LOOKING AT A DEPRIORITIZED DRUG

Lumena was built upon a clinical-stage, cholesterol-busting drug candidate developed while RiverVest Managing Director John McKearn was head of discovery research at Searle. The compound was deprioritized by Searle/Pharmacia (now Pfizer), but had been proven safe and stable and ripe for a new clinical proposal.

Niall O’Donnell and entrepreneur Mike Grey worked backward to define their clinical focus; first understanding the problem the compound could solve (lowering high levels of bile acids) and then identifying a rare disease in which to solve that problem (cholestatic liver disease).

the team

Key members of the Lumena team who went on to co-found Mirum were Mike Grey, Niall O’Donnell, Ciara Kennedy, Susan Dubé, and Alex Dorenbaum.

Also notable were the deep connections forged with the Alagille Syndrome Alliance and the ALGS families as well as the creation of a “Lumena Pay-it-Forward” database, which became the foundation for the groundbreaking Global ALagille Alliance (GALA) Study.

The Story

RiverVest leverages a unique innovation network to find and cultivate compelling investment opportunities. We invest in products with a known path to clinical approval, doing our due diligence to assess markets, viability, and regulatory hurdles.
In 2009, Ted Greene, CEO of Satiogen Pharmaceuticals, called longtime colleague RiverVest Managing Director John McKearn about a LDL cholesterol-targeting compound developed during John’s tenure as head of discovery research at Searle/Pharmacia (now Pfizer). Ted believed the compound could be effective as a bile acid reuptake inhibitor, which could potentially induce weight loss and correct type 2 diabetes.
RiverVest Managing Director Niall O’Donnell joined John in diving into this hypothesis under Mike Grey, an experienced pharmaceutical executive, serial entrepreneur, and venture partner at Pappas Ventures, who licensed technologies from Satiogen and formed Lumena with seed funding from Pappas and RiverVest.
While diabetes was, and may still be, an exciting opportunity for this mechanism, the team identified an urgent, unmet medical need: children with rare, life-threatening pediatric liver diseases. Niall began building a fast-track strategy for the clinical trials to get these children and their families the relief they needed.
An estimated 4,500 children in the U.S. and U.K. are diagnosed with Alagille syndrome (ALGS) and Progressive familial intrahepatic cholestasis (PFIC), which cause excruciating itching, failure to thrive, and for many, the need for liver transplant in order to reach adulthood. The lead Lumena asset, LUM001 (maralixibat), offered hope for these children and their families.
John joined Lumena’s board of directors and Niall became acting chief medical officer, helping guide the company through interactions with U.S. and European regulatory bodies and develop rapid proof-of-concept clinical programs. Ciara Kennedy, a long-time colleague of Niall, was brought in as chief operating officer to execute the clinical programs.
Just as clinical studies were nearing completion and Lumena began exploring the potential for an initial public offering (IPO), Shire offered to acquire the company, returning ~$400 million to Lumena investors. Maralixibat, now in the hands of Mirum Pharmaceuticals (a RiverVest Fund IV company), received FDA approval in September 2021 for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.
The story continues with Mirum.