Mirum Pharmaceuticals raises $120m to advance Maralixibat into phase 3

Mirum Pharmaceuticals, a clinical stage pharma company in San Diego, has raised $120 million in a Series A funding round to support the development of its lead drug Maralixibat for the treatment of rare cholestatic liver diseases.

The financing round in the pharma company in California was led by New Enterprise Associates (NEA). Other firms like Deerfield Management, Frazier Healthcare Partners, Novo Holdings, Rock Springs Capital, Pappas Capital and RiverVest Venture Partners took part in the funding round of Mirum Pharmaceuticals.

The San Diego pharma company also signed an agreement with Shire to acquire the exclusive global rights for developing and commercializing maralixibat.

As per the deal, Shire will get an upfront payment, an equity stake in Mirum Pharmaceuticals and will also stand to earn potential future milestone payments and royalties.

Mirum Pharmaceuticals raises $120m to advance Maralixibat into phase 3.

Mirum Pharmaceuticals raises $120m to advance Maralixibat into phase 3. Photo courtesy of Stuart Miles/

Maralixibat is an oral inhibitor of the apical sodium-dependent bile acidic transporter (ASBT). The drug is being developed for the treatment of Alagille syndrome (ALGS) and also for progressive familial intrahepatic cholestasis (PFIC).

Ed Mathers – partner at New Enterprise Associates said: “The substantial investment we have made in Mirum and the outstanding syndicate of savvy biotech investors underscores the value we ascribe to maralixibat, as well as our confidence in the company’s management team to execute on an aggressive development plan.”

Maralixibat is being studied for its ability to prevent accumulation of excess bile acids and control extreme itching related to cholestatic liver diseases such as Alagille syndrome and progressive familial intrahepatic cholestasis. The San Diego pharma company is hoping that its ASBT inhibitor becomes the first-in-class oral drug for the two conditions, depending on regulatory approvals.

Mirum Pharmaceuticals revealed that it will initially focus on the development of maralixibat as treatments for the two pediatric indications.

Further, it said that there is a possibility for the ASBT inhibitor of being developed for additional pediatric and adult cholestatic liver disease indications.

So far, the drug has been tested on more than 230 cholestatic patients to provide a large safety data set.

The San Diego pharma company said that a Phase 2b trial, called ICONIC in ALGS patients, demonstrated a favorable outcome for the drug, based on the 48-week interim analysis the data.

Mirum Pharmaceuticals plans to launch phase 3 confirmatory studies of maralixibat in ALGS and PFIC patients in 2019.

Mike Grey – chairman, and CEO of Mirum Pharmaceuticals said: “The interim data we are announcing today from the Phase 2b ICONIC study in ALGS conducted by Shire underscores my continued confidence in maralixibat and its potential to help patients with these severely debilitating liver diseases.

“The study leveraged an improved trial design and in its interim analysis, patients taking maralixibat had reductions in bile acids and pruritus compared to placebo. Additionally, in a single-arm, open-label Phase 2 study, a subset of patients with PFIC2  responded to maralixibat, with a sustained (>2 years for some) reduction or normalization of serum bile acids and reduction of pruritus.

“These results led to the FDA’s Breakthrough Therapy designation for patients with PFIC2. These clinical studies demonstrate the potential of maralixibat to significantly impact patients’ lives.”

In addition to maralixibat, Mirum Pharmaceuticals also purchased the exclusive global rights from Shire for the development and marketing of another ASBT inhibitor called volixibat.

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