Mirum Pharmaceuticals, Inc. operates as a biopharmaceutical company. The company is dedicated to transforming the treatment of rare diseases.
The company has three approved medicines: LIVMARLI (maralixibat) oral solution (Livmarli), CHOLBAM (cholic acid) capsules (Cholbam), and CHENODAL or CTEXLI (chenodiol) tablets (Chenodal or Ctexli).
Livmarli is a novel, orally administered, minimally-absorbed ileal bile acid transporter (IBAT) inhibitor (IBATi) that is approved for the treatment of choles...
Mirum Pharmaceuticals, Inc. operates as a biopharmaceutical company. The company is dedicated to transforming the treatment of rare diseases.
The company has three approved medicines: LIVMARLI (maralixibat) oral solution (Livmarli), CHOLBAM (cholic acid) capsules (Cholbam), and CHENODAL or CTEXLI (chenodiol) tablets (Chenodal or Ctexli).
Livmarli is a novel, orally administered, minimally-absorbed ileal bile acid transporter (IBAT) inhibitor (IBATi) that is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the United States (U.S.) and various other countries around the world and for cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) in the U.S and for the treatment of PFIC in the European Union (EU). The company markets and commercializes Livmarli in the United States, Canada and certain countries in Europe through its specialized and focused commercial team. The company has also entered into license and distribution agreements with several rare disease companies for the commercialization of Livmarli in additional countries.
On August 31, 2023, the company completed the acquisition of assets of Travere Therapeutics, Inc. (Travere) that are primarily related to the development, manufacture (including synthesis, formulation, finishing or packaging) and commercialization of chenodiol and Cholbam (also known as Kolbam) (and together with chenodiol, the Bile Acid Medicines) pursuant to an asset purchase agreement dated July 16, 2023 (such acquisition, the Bile Acid Portfolio Acquisition).
The U.S. Food and Drug Administration (FDA) approved Cholbam in March 2015 as the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects and for adjunctive treatment of patients with peroxisomal disorders, including peroxisome biogenesis disorder-Zellweger spectrum disorder (PBD-ZSD). Chenodiol is standard of care for the treatment of cerebrotendinous xanthomatosis (CTX) in the United Sates with a medical necessity recognition by the FDA. Chenodiol is commercialized under the brand name Chenodal. The company submitted an NDA for chenodiol for the treatment of CTX in 2024 and received FDA approval for the treatment of adults with CTX in February 2025, which will be commercialized under the brand name Ctexli. The company commercializes Chenodal and Cholbam, and plan to commercialize Ctexli, in the United States through its specialized and focused commercial team. The company has also assumed license and distribution agreements with several rare disease companies for the commercialization of Cholbam and chenodiol in additional countries.
The company is advancing its product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. The company is developing volixibat in the setting of primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC). Volixibat has received breakthrough therapy and orphan drug designation from the FDA for cholestatic pruritus in PBC patients based on the positive interim analysis of the VANTAGE Phase 2b study. Volixibat has also completed a successful interim analysis of the VISTAS Phase 2b study in PSC patients. The company expects to complete enrollment of its VISTAS Phase 2b clinical trial in PSC in the second half of 2025 and of its VANTAGE Phase 2b clinical trial in PBC in 2026.
The company is also advancing its product candidate MRM-3379, a novel, oral, CNS-penetrant PDE4D inhibitor for the treatment of fragile X syndrome (FXS). The company is planning to initiate a dose ranging Phase 2 study of MRM-3379 in 2025.
Strategy
The key components of the company’s strategy are to commercialize and develop Livmarli for the treatment of ALGS, PFIC and other rare cholestatic conditions; commercialize and further develop Ctexli and Cholbam; develop and commercialize volixibat for the treatment of adults with PSC and PBC; develop and commercialize MRM-3379 for the treatment of FXS; and actively manage its product portfolio and expand its pipeline of novel product candidates.
Product Pipeline
Approved Medicines
Livmarli for cholestatic pruritus in patients with ALGS
Livmarli is a novel, orally administered, minimally-absorbed solution approved by the FDA and EMA for cholestatic pruritus in patients with ALGS. Livmarli is an IBATi that prevents absorption of bile acids in the ileum, thereby lowering serum bile acid (sBA) levels in settings of cholestasis where excess bile acids cause symptomatic and progressive disease burden.
Livmarli for cholestatic pruritus in patients with PFIC
Livmarli is a novel, orally administered, minimally-absorbed solution approved by the FDA for cholestatic pruritus in patients with PFIC and by the EMA for the treatment of patients with PFIC.
Ctexli (Chenodiol)
The company submitted a new drug application to the FDA for the approval of chenodiol in CTX in 2024 and received FDA approval to treat CTX in adults in February 2025, which will be commercialized under the brand name Ctexli. CTX is a rare, progressive and under diagnosed bile acid synthesis disorder affecting many parts of the body. In September 2022, chenodiol was granted Fast Track designation by the FDA for the treatment of CTX.
Clinical Product Candidates
Livmarli
The company is advancing its approved medicine Livmarli in additional indications. Through the EXPAND phase 3 study the company is exploring the treatment of pruritus in rare cholestatic settings other than ALGS, PFIC, PSC, intrahepatic cholestasis of pregnancy and PBC. The company expects to complete enrollment of this trial in 2026.
Volixibat
The company is advancing its product candidate, volixibat, a novel, oral, minimally-absorbed agent designed to inhibit IBAT, for the treatment of adult patients with cholestatic liver diseases. The company is developing volixibat for the treatment of PSC and PBC.
The company is conducting the VISTAS Phase 2b clinical trial of volixibat in patients with pruritus and PSC. VISTAS is an adaptive, randomized Phase 2b clinical trial evaluating the effect of volixibat on pruritus, sBA and fibrosis markers in patients with PSC and pruritus. The company expects to complete enrollment in the first half of 2025.
The companys is conducting the VANTAGE Phase 2b clinical trial of volixibat in patients with pruritus and PBC and it expects to complete enrollment in 2026.
MRM-3379
The company is advancing MRM-3379 for the treatment of FXS. MRM-3379 is a potent and selective allosteric inhibitor of PDE4D, an enzyme predominantly expressed in brain regions associated with learning, memory, and emotional regulation. The company expects to initiate a phase 2 clinical study of MRM-3379 in male FXS patients in 2025.
License, Finance, Royalty Agreements and Asset Purchases
Assignment and License Agreement with Shire International GmbH (Takeda)
In November 2018, the company entered into an assignment and license agreement (Shire License Agreement) with Shire International GmbH (Shire), which was subsequently acquired by Takeda Pharmaceutical Company Limited.
The company has sole authority and responsibility over development and commercialization activities for the Shire Licensed Products, and it is required to use commercially reasonable efforts to perform certain development, regulatory and commercialization activities with respect to the PFIC and ALGS indications for Livmarli and unspecified indications with respect to volixibat.
License Agreement with Pfizer Inc.
Through the Shire License Agreement, the company was assigned the rights to the license agreement (Pfizer Agreement), with Pfizer Inc. (Pfizer), pursuant to which it obtained an exclusive, worldwide license to Pfizer’s know-how related to Livmarli, (the Pfizer Know-How). Under the Pfizer Agreement, the company is permitted to research, develop, manufacture and commercialize products utilizing the Pfizer Know-How for the diagnosis, treatment, prevention, mitigation and cure of human diseases and disorders, and to sublicense such rights. Pfizer retained the right to use the Pfizer Know-How to conduct internal research and to use a third party to conduct research on Pfizer’s behalf.
The company has sole responsibility and control over development and commercialization activities for the Pfizer Know-How and products utilizing the Pfizer Know-How, and it is obligated to use commercially reasonable efforts to develop and commercialize products utilizing the Pfizer Know-How.
License Agreement with Sanofi-Aventis Deutschland GmbH
Through the Shire License Agreement, the company was assigned the rights to the license agreement, as amended (Sanofi Agreement), with Sanofi-Aventis Deutschland GmbH (Sanofi), under which it obtained an exclusive, worldwide license to certain patents and know-how controlled by Sanofi related to volixibat (Sanofi Technology). Under the Sanofi Agreement, the company is permitted to develop and commercialize products containing volixibat utilizing the Sanofi Technology. Additionally, under the Sanofi Agreement, it is permitted to manufacture products containing volixibat utilizing the Sanofi Technology and to sublicense such rights. In addition, Sanofi granted to the company, under certain conditions, an exclusive option to obtain an exclusive license to manufacture volixibat during the term of the Sanofi Agreement. The company exercised this option in May 2020 and is transferring manufacturing of volixibat to a third-party contract manufacturer. Sanofi retained the right to practice the Sanofi Technology outside the scope of the license granted to it under the Sanofi Agreement and to make and use for internal research purposes, provided that upon its request, Sanofi is obligated to provide it with a written summary of the results of any such research to the extent such results relate to the use of volixibat as an ASBT inhibitor (ASBTi).
Under the Sanofi Agreement, the company has sole authority and responsibility over development and commercialization activities for licensed products, and it is required to use diligent efforts to perform certain development, regulatory and commercialization activities.
License Agreement with Satiogen Pharmaceuticals, Inc.
Through the Shire License Agreement, the company was assigned the rights to the license agreement, as amended (Satiogen Agreement), with Satiogen Pharmaceuticals, Inc. (Satiogen), a wholly-owned Mirum entity, under which it obtained an exclusive, worldwide license to certain patents and know-how controlled by Satiogen related to ASBTis (ASBTi Technology), and TGR5 agonists (TGR5 Technology). Under the Satiogen Agreement, the company is permitted to develop, manufacture and commercialize products utilizing the ASBTi Technology or TGR5 Technology for the diagnosis, treatment, prevention, mitigation and cure of human diseases and disorders, other than diabetes, obesity or a combination thereof, and to sublicense such rights. In March 2017, the Satiogen Agreement was amended to terminate the license of certain patents related to the ASBTi Technology and TGR5 Technology as each relates to diabetes and obesity. In May 2022, the company completed the merger and acquisition of Satiogen in which Satiogen became its wholly-owned subsidiary.
The company has sole responsibility and control over development and commercialization activities for products utilizing the ASBTi Technology or TGR5 Technology under the Satiogen Agreement and it is required to use commercially reasonable efforts to develop and commercialize such licensed products.
License Agreement with Enthorin Therapeutics, LLC and Dart Neuroscience LLC
On October 22, 2024, the company entered into a license agreement (the MRM-3379 License) with Enthorin Therapeutics, LLC and Dart Neuroscience LLC (collectively Enthorin). The MRM-3379 License grants the company an exclusive, royalty bearing, sublicensable, worldwide license under certain regulatory materials, as well as patents and know-how, which it refers to collectively as the Enthorin IP, to develop, have developed, make, have made, use, sell, have sold, offer for sale or import ENT-3379, an allosteric inhibitor of Phosphodiesterase 4D, (PDE4D) for any medical diagnostic, therapeutic or prophylactic application in human health, including in FXS.
Commercial Agreements related to chenodiol
License and Manufacturing Agreement with LGM Pharma
Through the Bile Acid Portfolio Acquisition, the company was assigned the rights to the License and Manufacturing Agreement, dated November 4, 2009, between LGM Pharma, formerly Nexgen Pharma, Inc. (LGM), and Manchester Pharmaceuticals, Inc. (together with its affiliates, Manchester) related to the manufacture of chenodiol, pursuant to which it obtained an exclusive, perpetual license to market chenodiol in the U.S. under the terms of an abbreviated new drug application approved by the FDA and owned by LGM, which grants to LGM the authority to manufacture and sell chenodiol in the U.S.
Intellectual Property
The company has developed and continue to develop patent portfolios around its product candidates, Livmarli (maralixibat) and volixibat. The company has rights to pending patent applications in the United States, Europe, South Korea, Hong Kong, and Singapore covering the methods of treating various cholestatic liver indications using maralixibat and/or volixibat which, if issued, would expire in October 2032, absent any patent term adjustments or extensions. The company has rights to issued U.S. Patent No. 11,376,251, which is directed to methods of treating ALGS in a pediatric subject with maralixibat, expiring in October 2032. The company also has rights to U.S Patent Nos. 10,512,657 and 11,229,661, which are directed to methods of treating or ameliorating PFIC2 and methods of treating or ameliorating a pediatric disorder characterized by having a non-truncating BSEP mutation selected from PFIC2, BRIC2, and drug induced cholestasis in a pediatric subject comprising administering maralixibat, respectively, both of which expire in October 2032. These three U.S. patents are listed in the FDA’s Orange Book: Approved Drug Products with Therapeutic Equivalence Evaluations (Orange Book) for Livmarli. The company has rights to a U.S. patent that is directed to methods of treating PBC with volixibat, expiring in October 2032.
The company has rights to granted patents in Australia, Brazil, Canada, China, Israel, Japan, Mexico, South Korea, Armenia, Azerbaijan, Belarus, Kazakhstan, Kyrgyzstan, Russia, Tajikistan, Turkmenistan, South Africa and Macau covering the methods of treating cholestasis using IBATis that have limited systemic exposure, which expire in October 2032. The company also has rights to pending patent applications in United States, Europe, Eurasia, Hong Kong and Singapore, covering methods of treating pediatric cholestatic liver diseases using IBATis that have limited systemic exposure, which, if issued, would expire in October 2032, absent any patent term adjustments or extensions. The company has rights to granted patents in Australia, Brazil, Canada, China, Israel, Japan, Mexico, South Korea, South Africa, Singapore, Austria, Belgium, Czech Republic, Denmark, Estonia, Finland, France, Germany, Ireland, Italy, Netherlands, Norway, Poland, Portugal, Slovak Republic, Spain, Sweden, Switzerland, the United Kingdom, Armenia, Azerbaijan, Belarus, Kazakhstan, Kyrgyzstan, Russia, Tajikistan, Macau and Turkmenistan covering methods of treating pediatric cholestatic liver diseases using IBATis that have limited systemic exposure, which expire in October 2032. The company also has rights to a granted patent in South Africa covering pediatric dosage forms of IBATis that have limited systemic exposure, which expires in October 2032.
The company has rights to U.S. Patent Nos. 11,229,647 and 11,497,745, which are directed to methods of treating ALGS in a pediatric subject comprising administering maralixibat to the subject expiring in February 2040. The company also has rights to U.S. Patent No. 11,918,578, which is directed to a method of treating cholestatic pruritus in ALGS subjects expiring in February 2040. These three U.S. patents are listed in the Orange Book for Livmarli. The company has rights to granted patents in Austria, Belgium, Bulgaria, Croatia, Denmark, Finland, France, Germany, United Kingdom, Ireland, Italy, the Netherlands, Norway, Poland, Portugal, Romania, Sweden, Switzerland, Turkey, United Kingdom, Spain, and Mexico, which are directed to maralixibat or volixibat for use in treating cholestatic liver disease in a subject having BSEP deficiency but without total loss of BSEP activity, which expire in February 2040. The company has rights to pending patent applications in the United States, Europe, Canada, China, Japan, South Korea, Israel, Brazil, Russia, Mexico, Australia, New Zealand, UAE, and Saudi Arabia directed to methods for treating cholestatic liver disease and to methods for using patient genotype to predict response to IBATi administration in patients with BSEP deficiency. Any patents issuing from these applications would expire in February 2040, absent any patent term adjustments or extensions.
The company has rights to pending applications in the United States, Europe, Canada, China, Japan, South Korea, Israel, Brazil, Russia, Mexico, Australia, New Zealand, UAE, and Saudi Arabia directed to methods for treating cholestatic liver disease comprising administering higher dosages of IBATis. The company has rights to granted patents in Japan, Russia, Hong Kong, Croatia, Czech Republic, Ireland, Norway, Poland, Romania, Slovak Republic, Spain, Switzerland, and the United Kingdom directed to methods of increasing growth in pediatric subjects having cholestatic liver disease by administering IBATis, which expire in February 2040. The company has rights to pending applications in the United States, Europe, Canada, China, Japan, South Korea, Israel, Brazil, Russia, Mexico, Australia, New Zealand, UAE, and Saudi Arabia directed to methods of increasing growth in pediatric subjects having cholestatic liver disease by administering IBATis. Any patents issuing from these applications would expire in February 2040, absent any patent term adjustments or extensions.
The company has rights to pending applications in the United States, Argentina, Taiwan, Europe, Canada, China, Japan, Korea, Australia, India, Israel, Singapore, Brazil, Mexico, Eurasia, New Zealand, the United Arab Emirates, Saudi Arabia, Algeria, Morocco, Tunisia, Egypt, Chile, Colombia, Indonesia, Malaysia, the Philippines, Thailand, Vietnam, South Africa and Hong Kong directed to formulations of maralixibat which, if issued, would expire in October 2042, absent any patent term adjustments or extensions. The company has rights to a granted patent in Luxemburg directed to increased event-free survival of long-term maralixibat patients which expires in October 2042. The company has rights to pending applications in the United States, Taiwan, Europe, Canada, China, Japan, South Korea, Israel, Singapore, Brazil, Mexico, Eurasia, South Africa, and Hong Kong directed to methods of treatment with IBATis in the fasted state which, if issued, would expire in October 2042, absent any patent term adjustments or extensions. The company has rights to pending applications in the United States, Taiwan, Europe, Canada, China, Japan, South Korea, Australia, Israel, Singapore, Brazil, Mexico, South Africa, New Zealand, Chile, Malaysia, United Arab Emirates, Morocco, Tunisia, Lybia, Iraq, Algeria, and Hong Kong directed to increased event-free survival of long-term maralixibat patients which, if issued, would expire in November 2042, absent any patent term adjustments or extensions.
The company has rights to pending applications in the United States, Argentina, Taiwan, Uruguay, Paraguay and Patent Cooperation Treaty (PCT) directed to highly pure maralixibat forms and intermediates which, if issued, would expire in September 2043, absent any patent term adjustments or extensions. The company has rights to pending applications in the United States, PCT, and Taiwan directed to maralixibat compositions and solid dosage forms which, if issued, would expire in October 2043, absent any patent term adjustments or extensions. The company has rights to pending applications in the United States, Taiwan, and PCT directed to methods of treating PFIC with maralixibat which, if issued, would expire in October 2043, absent any patent term adjustments or extensions.
The company has licensed patent applications in the United States, Hong Kong, and Europe from Satiogen covering therapeutic uses of IBATis that have limited systemic exposure for treating inflammatory intestinal conditions, which, if issued, would expire in May 2031, absent any patent term adjustments or extensions. Two of these Satiogen applications have issued as United States Patent No. 10,251,880 and 11,260,053, the latter being Orange-Book listed for Livmarli. Thew company has licensed an issued United States patent, as well as issued foreign counterparts in Argentina, Austria, Australia, Belgium, Canada, Switzerland, China, Germany, Denmark, Spain, Finland, France, United Kingdom, Greece, Hong Kong, Ireland, Israel, India, Italy, Japan, South Korea, Liechtenstein, Mexico, Malaysia, the Netherlands, Norway, Portugal, Russia, Sweden, Singapore, Taiwan, Turkey, and Brazil from Sanofi, that cover the composition and methods of making volixibat and salts thereof, expiring in December 2027. Patents related to Livmarli and volixibat may be eligible for patent term extensions in certain jurisdictions, including the United States for volixibat, upon approval of a commercial use of the corresponding product by a regulatory agency in the jurisdiction where the patent was granted. Similar to the patent term-extensions in the United States, Supplementary Protection Certificates (SPCs) serve as an extension to a patent right in the EU for up to five years. SPCs have been granted for maralixibat for EP2771003 in Austria, Denmark, France, Italy, the Netherlands, Portugal, Spain, and Sweden and are pending in Belgium, Czech Republic, Germany, Estonia, Finland, the United Kingdom, Ireland, Norway, Poland, and Slovakia.
The company has rights to a patent portfolio and will continue to develop a patent portfolio around its product candidate MRM-3379. The company has rights to three issued U.S. patents covering MRM-3379 as a composition-of-matter, as well as a pending counterpart in the United States and issued foreign counterparts in Belgium, France, Germany, Norway, the Netherlands, Sweden, Switzerland, the United Kingdom, Denmark, Italy, Ireland, Spain, the United Kingdom, Australia, Canada, China, Japan, South Korea, Brazil, India, Israel, Mexico, New Zealand, Russia, Singapore, Hong Kong, and Taiwan, which are set to expire in March 2034, absent any patent term extensions. Patents related to MRM-3379 may be eligible for patent term extensions in certain jurisdictions, including the United States, upon approval of a commercial use of the corresponding product by a regulatory agency in the jurisdiction where the patent was granted.
Upon approval in the United States, as MRM-3379 has not previously been approved in the United States for any indication, it may be eligible for five years of NCE exclusivity, which would run concurrently with its seven years of orphan drug exclusivity if the company obtains orphan drug exclusivity for FXS or another approved orphan indication.
The company is a party to a number of license agreements under which it is granted intellectual property rights to know-how that are important to its business. Th company has licensed know-how related to Livmarli in the United States, Europe and other countries from Pfizer. The company has licensed know-how related to ASBTi Technology and TGR5 Technology from Satiogen. The company has licensed know-how related to volixibat from Sanofi. The company has licensed know-how related to MRM-3379 from Enthorin. The company’s existing license agreements as related to Livmarli, volixibat, and MRM-3379 impose various development, regulatory and/ commercial diligence obligations, payment of milestones and/or royalties and other obligations.
In addition, the company has orphan drug exclusivity for Livmarli for the treatment of ALGS and PFIC in the United States and the EU, providing seven years of market exclusivity in the United States, which can be extended to seven and a half years if trials are conducted in accordance with an agreed-upon pediatric investigational plan, and ten years of market exclusivity in the EU, which has been extended to 12 years in the EU for the treatment of ALGS in view of a pediatric award and may be extended to 12 years in the EU for PFIC. In addition, the company has orphan drug designation for volixibat for the treatment of PBC in the United States and in the EU, providing the opportunity to receive seven years of market exclusivity in the United States, which can be extended to seven and a half years if trials are conducted in accordance with an agreed-upon pediatric investigational plan, and ten years of market exclusivity in the EU, which can be extended to 12 years in the EU if trials are conducted in accordance with an agreed-upon pediatric investigational plan.
In the United States, maralixibat has been granted new chemical entity (NCE) exclusivity until September 29, 2026. This five years of post-FDA approval exclusivity runs concurrently with its seven years orphan drug exclusivity for the treatment of ALGS. Upon approval in the United States, as volixibat has not previously been approved in the United States for any indication, it may be eligible for five years of NCE exclusivity, which would run concurrently with its seven years of orphan drug exclusivity if the company obtains orphan drug exclusivity for an approved orphan indication.
Sales, Marketing and Distribution
The company has built the commercial infrastructure necessary to effectively support the commercialization of its approved medicines in North America and certain countries in Europe and are using strategic partners, and distributors to assist in the commercialization of the company’s approved medicines in other markets.
The company’s approved medicines are distributed in the U.S. and Canada, through a single specialty pharmacy in each country. In other geographies, the company’s approved medicines are sold direct to pharmacies by its third-party logistics providers, authorized distributors or licensed partners.
Research and Development
The company’s research and development expenses were $140.6 million for the year ended December 31, 2024.
History
Mirum Pharmaceuticals, Inc. was founded in 2018. The company was incorporated under the state of Delaware in 2018.
