\n\u00a0<\/p>\n<\/td>\n
Difference at end of double-blind period (placebo relative to Chenodal)<\/b><\/p>\n<\/td>\n
\nP-value<\/p>\n<\/td>\n<\/tr>\n
Primary Endpoint<\/i><\/p>\n<\/td>\n
\n\u00a0<\/p>\n<\/td>\n
\n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\nUrine 23S-Pentol (bile alcohol) ng\/mL<\/p>\n<\/td>\n
\n20-fold increase (CI: 10.3, 43.5)<\/p>\n<\/td>\n
\n<0.0001<\/p>\n<\/td>\n<\/tr>\n
Key Secondary Endpoints<\/i><\/p>\n<\/td>\n
\n\u00a0<\/p>\n<\/td>\n
\n\u00a0<\/p>\n<\/td>\n<\/tr>\n
\nRescue medication*<\/p>\n<\/td>\n
\n61.5% of placebo patients (CI: 31.6%, 86.1%)<\/p>\n<\/td>\n
\n0.0006<\/p>\n<\/td>\n<\/tr>\n
\nPlasma cholestanol \u00b5g\/mL<\/p>\n<\/td>\n
\n2.8-fold increase (CI: 1.5, 5.2)<\/p>\n<\/td>\n
\n0.0083<\/p>\n<\/td>\n<\/tr>\n
\nPlasma 7\u03b1C4 ng\/mL<\/p>\n<\/td>\n
\n50-fold increase (CI: 25.0, 66.7)<\/p>\n<\/td>\n
\n<0.0001<\/p>\n<\/td>\n<\/tr>\n
\n* Proportion of patients on placebo requiring rescue Chenodal during double-blind withdrawal periods<\/p>\n<\/td>\n<\/tr>\n<\/table>\n
\nThe most commonly reported adverse events while on Chenodal were diarrhea (n=5) and headache (n=3). The majority of adverse events reported were mild or moderate in severity and not considered to be treatment-related.<\/p>\n
\n\u201cBecause the diagnostic odyssey for CTX is typically long and arduous and the complications serious, it is critical that patients are treated quickly following diagnosis to ameliorate or prevent symptoms that can severely impact their lives,\u201d said Robert Steiner, MD, professor, geneticist, and CTX Alliance medical and scientific advisory board member. \u201cThese data demonstrate a strong potential to help patients avoid the devastating effects of CTX.\u201d<\/p>\n
\n\u201cCTX is a rare multi-symptom disease that affects each patient very differently, and can significantly impact a person\u2019s quality of life,\u201d said Jean Pickford, executive director, CTX Alliance. \u201cFor many patients and their families, the diagnostic journey is challenging and navigating the symptoms is ongoing. Once diagnosed, patients and their families are thrust into the world of a rare disease that can feel isolating and overwhelming. However, we are thrilled to see these impressive data from the RESTORE study. A change in bile alcohols and cholestanol have the potential to reduce the progressive symptoms associated with this rare disease.\u201d<\/p>\n
\nData from the RESTORE study will be presented at an upcoming scientific congress, including forthcoming results from the open-label pediatric group of patients. In addition, Mirum will be submitting a new drug application to the U.S. FDA in the first half of 2024.<\/p>\n
About the RESTORE Phase 3 Study<\/b><\/p>\n
\nThe Phase 3 RESTORE study is a randomized withdrawal, placebo-controlled clinical trial which evaluated the safety and efficacy of Chenodal in patients with cerebrotendinous xanthomatosis (CTX). Chenodal is administered at 250 mg three times daily in tablet format. The objective of the RESTORE study is to understand how the body responds, as measured by change in blood and urine biomarkers associated with CTX, when treated with Chenodal. The study involved a screening period (4 weeks), four treatment periods (totaling 6 months), and a follow-up phone call (30 days after last dose was administered). The four treatment periods consisted of: an 8-week open-label Chenodal period, a 4-week randomized withdrawal period (placebo or Chenodal), a second 8-week open-label Chenodal period for all patients, and a second 4-week randomized withdrawal period (alternate treatment to first withdrawal period).<\/p>\n
\nThe primary analysis assessed change at the end of each double-blind withdrawal period. The study also included an open-label pediatric treatment group where all patients received liquid Chenodal.<\/p>\n
\nThe study was conducted at multiple sites in the United States and Brazil.<\/p>\n
About Cerebrotendinous Xanthomatosis<\/b><\/p>\n
\nCerebrotendinous xanthomatosis (CTX) is an autosomal, recessive, progressive genetic disorder resulting from a deficiency of a key enzyme in the bile acid synthesis pathway. CTX is characterized by fatty yellow nodules (xanthomas) located in the connective tissues within the brain. These deposits can cause progressive damage to the brain and other areas of the body. As the clinical course progresses, irreversible neurological deterioration leads to premature death. CTX is a rare disease affecting one to two thousand people in the United States.<\/p>\n
About CHENODAL\u00ae (chenodiol) tablets<\/b><\/p>\n
\nChenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. More recently, the US Food and Drug Administration (FDA) granted Chenodal orphan drug designation for cerebrotendinous xanthomatosis (CTX). CTX is a rare progressive disorder that can affect the brain, spinal cord, tendons, eyes and arteries. Chenodal is not indicated for the treatment of CTX but has received a medical necessity determination in the U.S. by the FDA. Full data from the Phase 3 RESTORE clinical trial are expected to be presented at an upcoming medical congress.<\/p>\n
About Mirum Pharmaceuticals, Inc.<\/b><\/p>\n
\nMirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI\u00ae (maralixibat) oral solution<\/a>, Cholbam\u00ae (cholic acid) capsules,<\/a> and Chenodal\u00ae (chenodiol) tablets.<\/a><\/p>\n \nLIVMARLI, an IBAT inhibitor, is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older and in Europe in PFIC for patients two months of age and older. Cholbam is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. Chenodal has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).<\/p>\n \nMirum\u2019s late-stage pipeline includes three investigational treatments for debilitating liver diseases. The LIVMARLI development program includes the Phase 2b EMBARK study for biliary atresia. Mirum\u2019s second investigational IBAT inhibitor is volixibat, which is being evaluated in two potentially registrational studies including the Phase 2b VISTAS<\/a> study for primary sclerosing cholangitis and Phase 2b VANTAGE<\/a> study for primary biliary cholangitis. Lastly, Chenodal, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX.<\/p>\n \nTo learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook<\/a>, LinkedIn<\/a>, Instagram<\/a> and Twitter<\/a>.<\/p>\n Forward-Looking Statements<\/b><\/p>\n This press release contains \u201cforward-looking statements” within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. Such forward-looking statements, including those regarding the clinical potential and regulatory process for Chenodal, the data resulting from the RESTORE trial, and the development success of Mirum products and candidates involve risks and uncertainties. The Company\u2019s experience and results may differ materially from the experience and results anticipated in such statements. The accuracy of such statements is subject to a number of risks, uncertainties and assumptions including, but are not limited to, the following factors: the uncertainties inherent in research and development; the uncertainties inherent in business and financial planning, including, without limitation, risks related to Mirum\u2019s business and prospects, adverse developments in our focused markets, or adverse developments in the U.S. or global regulatory environment or economies generally; the continued impact of COVID-19 on our business, operations and financial results; and competitive developments. Other factors that might cause such a difference include those discussed in the Company\u2019s filings with the SEC. All forward-looking statements contained in this press release speak only as of the date on which they were made and are based on management\u2019s assumptions and estimates as of such date. Mirum undertakes no obligation to update such statements to reflect events that occur or circumstances that exist after the date on which they were made, except as required by law.<\/i><\/p>\n View source version on businesswire.com: <\/span>https:\/\/www.businesswire.com\/news\/home\/20231002522656\/en\/<\/a><\/span><\/p>\n Media<\/b>: Investors: \nSam Martin KEYWORDS:<\/b> California United States North America<\/p>\n INDUSTRY KEYWORDS:<\/b> Biotechnology Pharmaceutical Health Clinical Trials<\/p>\n<\/p>\n
\n
Erin Murphy
\n
510-508-6521
\n
media@mirumpharma.com<\/a><\/p>\n
\n<\/b>
Andrew McKibben
\n
ir@mirumpharma.com<\/a><\/p>\n
\n
Argot Partners
\n
ir@mirumpharma.com<\/a><\/p>\n
![\"Logo\"](\"https:\/\/mms.businesswire.com\/media\/20231002522656\/en\/746217\/3\/Mirum_Inline_FullColor_RGB.jpg\")
<\/td>\n<\/tr>\n