The Time Has Come to Present Mirum Pharmaceuticals Inc

Mirum Pharmaceuticals Inc

Mirum Pharmaceuticals Inc (MIRM) is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases.

Maralixibat, is Mirum Pharmaceuticals lead product candidate. The product is an investigational oral drug in development for the following conditions:  

  • Alagille syndrome (ALGS)
  • Progressive familial intrahepatic cholestasis (PFIC) and
  • Biliary atresia (BA)

It is important to note that Mirum Pharmaceuticals Inc has submitted an NDA for maralixibat for the treatment of cholestatic pruritus in patients with ALGS in the U.S. We were motivated to post about Mirum Pharmaceuticals today because the NDA has been accepted for priority review by the FDA with a PDUFA action date of September 29, 2021,

We believe the FDA will approve maralixibat this upcoming Wednesday.

And that’s not all…

The Company has submitted a marketing authorization application for maralixibat to the European Medicines Agency for the treatment of cholestatic liver disease for patients with ALGS.

Volixibat – another oral ASBT-inhibitor, for primary sclerosing cholangitis, intrahepatic cholestasis of pregnancy and primary biliary cholangitis.

To augment its pipeline in cholestatic liver disease Mirum acquired, from Vivet Therapeutics, SAS, an exclusive option to develop and commercialize the following gene therapy programs: VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively following preclinical evaluation and investigational new drug-enabling studies.

The Latest News About Mirum Pharmaceuticals Inc

On September 21, 2021 Mirum Pharmaceuticals and Takeda Pharmaceutical (TAK) announced that they have entered into an exclusive licensing agreement for the development and commercialization of maralixibat in Japan for ALGS, PFIC and BA.

Under the terms of the agreement, Takeda will be responsible for regulatory approval and commercialization of maralixibat in Japan. Takeda will also be responsible for development, including conducting clinical studies in cholestatic indications.

From Mirum Pharmaceuticals Inc & Takeda Pharmacuetical Ltd

Chris Peetz, President and Chief Executive Officer of Mirum Pharmaceuticals, said, “Takeda is a leading global biopharmaceutical company with extensive experience in development and commercialization of novel therapies to treat rare diseases as well as gastroenterology and hepatology, making them an ideal partner as we look to accelerate the delivery of maralixibat to children living with rare liver diseases in Japan. As we approach potential commercialization in the United States and complete the recent filing for Alagille syndrome in Europe, our goal is to partner with top companies outside of North America and Europe to ensure global reach for patients with these terrible diseases. We are excited for Takeda to engage in the development of maralixibat and collaborate in our effort to advance this potentially life-changing therapy.”

Dr. Naoyoshi Hirota, General Manager of Takeda Development Center in Japan, said, “There is a significant unmet medical need for a treatment to help patients with cholestatic diseases such as ALGS and PFIC in Japan and developing novel treatment for those patients suffering from rare liver diseases is a top priority for Takeda’s global R&D strategy. This agreement reinforces Takeda’s commitment to developing highly differentiated medicines to improve the health and quality of life of patients.”

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter (ASBT), resulting in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated effects.

More than 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for ALGS and PFIC. In the ICONIC Phase 2b ALGS trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo.

In a Phase 2 PFIC study, a genetically defined subset of BSEP deficient (PFIC2), patients responded to maralixibat with an increase in transplant-free survival. The U.S. FDA has granted maralixibat Breakthrough Therapy designation for the treatment of pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat demonstrated a tolerable safety profile in the studies. The most frequent treatment-related adverse events were diarrhea and abdominal pain. Maralixibat has been studied extensively and its safety database represents the largest database for an ASBT inhibitor.

About Takeda Pharmaceutical Company Limited

Takeda Pharmaceutical Company Limited (TSE: 4502/NYSE: TAK) is a global, values-based, R&D-driven biopharmaceutical leader headquartered in Japan, committed to discover and deliver life-transforming treatments, guided by our commitment to patients, our people and the planet. Takeda focuses its R&D efforts on four therapeutic areas: Oncology, Rare Genetics and Hematology, Neuroscience, and Gastroenterology (GI). We also make targeted R&D investments in Plasma-Derived Therapies and Vaccines. We are focusing on developing highly innovative medicines that contribute to making a difference in people’s lives by advancing the frontier of new treatment options and leveraging our enhanced collaborative R&D engine and capabilities to create a robust, modality-diverse pipeline. Our employees are committed to improving quality of life for patients and to working with our partners in health care in approximately 80 countries and regions.

Prohost Observations

We repeat that Mirum Pharmaceuticals’ PDUFA is on September 29, 2021, this Wednesday, for the approval of the firm’s most needed product maralixibat. From all the news about the Company, its product, the recent deal with Takeda, as well as the way the FDA has considered the NDA and the needs for the product, we are optimistic about the PDUFA outcome.

The stock is trading at $18.22, which is still affordable; the firm’s market cap is $564.24 million, which is low when you consider the firm is expected to go from clinical stage to revenue generating right after the maralixibat approval.


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