Latest news with #Mirum
Business Wire
19-05-2025
- Business
- Business Wire
Mirum Pharmaceuticals Appoints Doug Sheehy, JD, as Chief Legal Officer
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced the appointment of Doug Sheehy, JD, as chief legal officer. Mr. Sheehy brings nearly two decades of experience leading global legal and compliance operations for biopharmaceutical companies. Mr. Sheehy was most recently chief legal officer and secretary at Sonoma Biotherapeutics, Inc., which is developing regulatory T cell therapies for autoimmune and inflammatory diseases. Prior to Sonoma, from 2016 to 2020, he served as general counsel and secretary for Aimmune Therapeutics, Inc., a biopharmaceutical company that specialized in the development and commercialization of treatments for life-threatening food allergies. Mr. Sheehy served at Aimmune until its acquisition by Nestle Health Science in 2020. Before Aimmune, from 2007-2016, he was executive vice president, chief administrative officer, general counsel, and secretary of Codexis, Inc., a company focused on the development of synthetic enzymes for the pharmaceutical and food industries. Earlier in his career, he served in several roles within the legal department at CV Therapeutics. Mr. Sheehy began his career as a corporate and securities lawyer in Silicon Valley representing emerging growth companies and venture funds. Mr. Sheehy received an A.B. in History from Dartmouth College and a J.D. from American University's Washington College of Law. 'Doug brings extensive global legal experience that will strengthen Mirum's capabilities as we continue to grow our commercial business and advance our rare disease pipeline,' said Chris Peetz, chief executive officer at Mirum. 'Doug will be a great addition to Mirum's leadership team, and I look forward to leveraging his experience in guiding transformative growth for the company.' 'Mirum is an impressive company that has established its place as a leader in rare disease, and I am excited to have the opportunity to join the leadership team and contribute to the exciting work underway,' said Doug Sheehy. 'The company has a focused growth strategy and is poised for continued success as it works to make a meaningful difference in the lives of people impacted by rare disease.' About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It 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 other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X).
Business Wire
09-05-2025
- Health
- Business Wire
Volixibat Data from Mirum's VANTAGE PBC Study Showcased at EASL
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today presented new data from its Phase 2b VANTAGE study at the European Association for the Study of the Liver (EASL) meeting in Amsterdam, the Netherlands. The VANTAGE study is evaluating volixibat in patients with cholestatic pruritus caused by primary biliary cholangitis (PBC). 'The extended VANTAGE analysis provides encouraging insights into the potential impact that volixibat may have in treating the most severe and bothersome symptoms for patients living with PBC, including pruritus and fatigue,' said Joanne Quan, MD, chief medical officer at Mirum. 'We are also encouraged by the reductions seen in IL-31 in conjunction with sBA reductions, showing the potential for volixibat treatment to help alter the inflammatory condition present in PBC.' Data from the interim analysis of VANTAGE were highlighted with new analyses through week 28. Patients were randomized to receive volixibat 20mg BID, 80mg BID, or placebo BID. The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale, from baseline to Week 17-Week 28. Volixibat treatment led to rapid (as early as week 1) and sustained reductions in ItchRO with the volixibat combined dose group achieving a statistically significant 3.78-point reduction from Baseline (p<0.0001) and a placebo-adjusted response of 2.51 (p=0.0004). Each volixibat dose achieved statistically significant and similar responses (20mg BID: -2.4 [P=0.0039]; 80mg BID: -2.6 [P=0.0010]). The analysis also provided data across secondary endpoints showing reductions in serum bile acids (sBA) and quality of life (QoL) measures. Overall, 70% of patients who received volixibat achieved ≥50% reduction in sBA. Further, the data showed improvement from baseline in health-related quality of life, including fatigue and sleep, for patients treated with volixibat. Reductions from baseline were observed in inflammatory biomarker IL-31 in patients treated with volixibat. No new safety signals were observed, and adverse events were similar between the 20 mg and 80 mg treatment groups. The most common adverse event was diarrhea (77%) with all cases mild to moderate; one case resulted in discontinuation. Given the similar results with both efficacy and safety between the 20mg BID and 80mg doses BID, the 20mg BID dose was selected for the confirmatory portion of the VANTAGE study. To view the presentation, please visit the Publications & Presentations on Mirum's website. About the VANTAGE Study VANTAGE is a randomized, double-blind, placebo-controlled study evaluating the efficacy and safety of volixibat in patients with pruritus caused by primary biliary cholangitis (PBC). Patients were randomized to receive volixibat 20mg BID (n=10), 80mg BID (n=10), or placebo BID (n=11). The primary endpoint of the study was mean change in weekly averaged daily itch score, as measured by the adult ItchRO scale (an 11 point scale where 0 = no itch and 10 = worst itch imaginable), from baseline to Week 17-Week 28. VANTAGE is designed as a two-part study: the first part is intended to confirm dose (completed) and the second intended to be the confirmatory portion (currently enrolling patients). In the first part of the study, patients were randomized to receive either volixibat 20mg BID, 80mg BID, or placebo BID; the second part of the study randomized patients to receive either volixibat 20mg BID (the selected dose based on part one) or placebo BID. About Primary Biliary Cholangitis Primary Biliary Cholangitis (PBC) is a chronic, progressive, immune-mediated condition leading to the destruction of small intrahepatic bile ducts. The disease impacts an estimated 230,000 patients in the United States and the European Union, with women predominantly affected (a sex ratio of 9:1). Key clinical features and symptoms include cholestatic pruritus, fatigue, sicca syndrome, abdominal pain, cirrhosis, and hepatocellular carcinoma. Cholestatic pruritus (itch) and fatigue are two of the most debilitating symptoms and greatly affect overall health-related quality of life (HRQoL). Cholestatic pruritus can affect up to 80% of individuals and is thought to result in part from accumulation of toxic bile acids. About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI ® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases, in both liquid and tablet formulations. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website. U.S. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including: Liver injury. Changes in certain liver tests are common in patients with Alagille syndrome and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. LIVMARLI is available in oral solution or tablet formulations and is taken by mouth 30 minutes before a meal. For Alagille syndrome, LIVMARLI is taken one time each day in the morning. For PFIC, LIVMARLI is taken two times each day. If you take the oral solution, use the oral dosing dispenser to measure your dose. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It 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 other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, data from Mirum's volixibat studies, including the VANTAGE study of volixibat in patients with pruritus due to PBC and such implications for patients in real world settings. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'will,' 'could,' 'can,' 'would,' 'potential,' 'hope,' 'opportunity,' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of macroeconomic and geopolitical developments, and the other risks described in Mirum's Annual Report on Form 10-K for the year ended December 31, 2024, filed with the U.S. Securities and Exchange Commission on February 26, 2025, and subsequent filings with the U.S. Securities and Exchange Commission, which are available at 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's 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.
Business Wire
07-05-2025
- Business
- Business Wire
Mirum Pharmaceuticals Reports First Quarter 2025 Financial Results and Provides Business Update
- - First quarter 2025 total revenue of $111.6 million; 2025 guidance increased to $435 to $450 million - VISTAS study in PSC expected to complete enrollment in third quarter of 2025; topline data expected in second quarter of 2026 - LIVMARLI oral tablet formulation FDA approved - Conference call to provide business updates today, May 7 at 1:30 p.m. PT/4:30 p.m. ET FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the first quarter 2025 and provided a business update. 'It's been a strong start to the year with commercial growth and multiple milestones achieved across our pipeline,' said Chris Peetz, chief executive officer of Mirum. 'We're pleased with the FDA's approval of LIMARLI's tablet formulation, which provides more options for the ALGS and PFIC community in the U.S. in the form of a convenient single tablet dose. Our VISTAS study of volixibat in PSC is enrolling well and we expect to complete enrollment in the third quarter of this year. We're excited to continue our strong execution across our commercial medicines and pipeline throughout the year.' Commercial: Raising full year revenue guidance to $435 to $450 million First quarter 2025 global net product sales of $111.6 million. First quarter 2025 LIVMARLI net product sales were $73.2 million representing 71% growth over first quarter 2024 net product sales. Bile Acid Medicines net product sales were $38.4 million representing 47% growth over first quarter 2024 net product sales. LIVMARLI oral tablet formulation approved. LIVMARLI approved in Japan for ALGS and PFIC. CTEXLI (chenodiol) approved in the US for cerebrotendinous xanthomatosis. Regulatory and Pipeline: Clinical milestones on track Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in third quarter of 2025; topline data expected in the second quarter of 2026. Oral presentation of 28-week interim data from volixibat in PBC (VANTAGE study) at EASL on Friday May 9th at 9:30am CEST. Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026. LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026. Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025. Corporate and Financial: Strong balance sheet and financial independence Total revenue for the quarter ended March 31, 2025, was $111.6 million compared to $69.2 million for the quarter ended March 31, 2024. Total operating expenses were $126.8 million for the quarter ended March 31, 2025 compared to $95.7 million for the quarter ended March 31, 2024. Total operating expenses for the quarter ended March 31, 2025 included $21.9 million of non-cash stock-based compensation, intangible amortization, and other non-cash expenses compared to $17.1 million for the quarter ended March 31, 2024. As of March 31, 2025, Mirum had unrestricted cash, cash equivalents and investments of $298.6 million compared to $292.8 million as of December 31, 2024. Business Update Conference Call Mirum will host a conference call today, May 7 th at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates. Join the call using the following details: Conference Call Details: U.S./Toll-Free: +1 833 470 1428 International: +1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI has received orphan designation for ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum's clinical trials section on the company's website. IMPORTANT SAFETY INFORMATION Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein. LIVMARLI can cause side effects, including Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you. A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with Alagille syndrome and PFIC but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects. EU SmPC Canadian Product Monograph About Volixibat Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study), and primary biliary cholangitis (PBC) (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate. Volixibat has been granted breakthrough therapy designation for the treatment of PBC. About CHOLBAM® (cholic acid) capsules The FDA approved CHOLBAM (cholic acid) capsules in March 2015, 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 peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy. CHOLBAM® (cholic acid) Indication CHOLBAM is a bile acid indicated for Treatment of bile acid synthesis disorders due to single enzyme defects. Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption. LIMITATIONS OF USE The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established. IMPORTANT SAFETY INFORMATION WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment. Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose. Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis. ADVERSE REACTIONS The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy. Please see full Prescribing Information for additional Important Safety Information. About CTEXLI™ (chenodiol) tablets CTEXLI™ (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol 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. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries. IMPORTANT SAFETY INFORMATION CTEXLI can cause side effects, including: Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching. Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection. Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines. US Prescribing Information About Mirum Pharmaceuticals Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It 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 other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). Forward-Looking Statements Statements contained in this press release regarding matters that are not historical facts are 'forward-looking statements' within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, significant increase or continued commercial results for our approved medicines, including continued financial growth, the potential achievement of our yearly financial guidance, continued strong execution across our approved medicines and pipeline, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for our product candidates in any indication or any specific territory. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as 'expected,' 'will,' 'could,' 'would,' 'guidance,' 'potential,' 'continue' and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Mirum's current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with Mirum's business in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum's Annual Report for the year ended December 31, 2024, filed with the Securities and Exchange Commission on February 26, 2025, and subsequent filings with the Securities and Exchange Commission, which are available at 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's 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. Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (in thousands) (Unaudited) March 31, 2025 2024 Assets Current assets: Cash and cash equivalents $ 211,822 $ 222,503 Short-term investments 65,852 57,812 Accounts receivable 95,852 78,286 Inventory 22,418 22,403 Prepaid expenses and other current assets 14,874 11,784 Total current assets 410,818 392,788 Restricted cash 425 425 Long-term investments 20,912 12,526 Intangible assets, net 243,845 249,819 Other noncurrent assets 14,245 15,196 Total assets $ 690,245 $ 670,754 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 11,639 $ 14,618 Accrued expenses and other current liabilities 115,938 111,933 Total current liabilities 127,577 126,551 Operating lease liabilities, noncurrent 7,003 7,972 Convertible notes payable, net, noncurrent 308,509 308,082 Other liabilities 13,900 2,509 Total liabilities 456,989 445,114 Commitments and contingencies Stockholders' equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 892,367 870,189 Accumulated deficit (658,858 ) (644,181 ) Accumulated other comprehensive loss (258 ) (373 ) Total stockholders' equity 233,256 225,640 Total liabilities and stockholders' equity $ 690,245 $ 670,754 Expand Contacts Investor Contact: Andrew McKibben ir@ Media Contact: Erin Murphy media@ Industry: Biotechnology Pharmaceutical Health Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025 FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025... Mirum Pharmaceuticals to Present Data at Upcoming Medical Congresses
Yahoo
02-05-2025
- Business
- Yahoo
High Growth Tech Stocks To Watch In The US May 2025
The U.S. stock market is experiencing a notable upswing, with the S&P 500 on track for its longest winning streak since 2004, buoyed by strong employment data and potential trade talks between the U.S. and China. In this environment of optimism and recovery, identifying high-growth tech stocks requires a focus on companies that demonstrate resilience in volatile conditions, adaptability to policy changes, and robust financial health amidst evolving economic landscapes. Name Revenue Growth Earnings Growth Growth Rating Super Micro Computer 20.35% 34.10% ★★★★★★ Travere Therapeutics 28.65% 66.06% ★★★★★★ Arcutis Biotherapeutics 26.11% 58.46% ★★★★★★ TG Therapeutics 26.06% 37.69% ★★★★★★ Alnylam Pharmaceuticals 23.29% 67.39% ★★★★★★ Clene 62.08% 64.01% ★★★★★★ Alkami Technology 20.59% 90.79% ★★★★★★ AVITA Medical 27.81% 55.17% ★★★★★★ Ascendis Pharma 32.75% 59.64% ★★★★★★ Lumentum Holdings 21.34% 120.49% ★★★★★★ Click here to see the full list of 236 stocks from our US High Growth Tech and AI Stocks screener. We're going to check out a few of the best picks from our screener tool. Simply Wall St Growth Rating: ★★★★★☆ Overview: Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to developing and commercializing novel therapies for debilitating rare and orphan diseases, with a market cap of $2.15 billion. Operations: Mirum Pharmaceuticals focuses on developing and commercializing therapies for rare and orphan diseases, generating revenue of $336.89 million from its pharmaceuticals segment. Mirum Pharmaceuticals, a key player in the biotech industry, is navigating its path toward profitability with significant advancements and strategic presentations. Recently, the company announced its participation in the EASL Symposium to present promising data from its VANTAGE study on May 9, 2025. This follows a robust year where Mirum reported a notable revenue increase to $336.89 million, up from $186.37 million the previous year, reflecting an annualized growth rate of 20.9%. Despite current unprofitability with a net loss reduction to $87.94 million from $163.42 million last year, these developments are pivotal as Mirum aims for profitability within three years amidst high expectations of earnings growth at an annual rate of 61.75%. This trajectory is supported by recent FDA approvals and continuous R&D efforts that underscore its commitment to addressing rare diseases through innovative treatments. Click here and access our complete health analysis report to understand the dynamics of Mirum Pharmaceuticals. Gain insights into Mirum Pharmaceuticals' historical performance by reviewing our past performance report. Simply Wall St Growth Rating: ★★★★☆☆ Overview: BioCryst Pharmaceuticals, Inc. is a biotechnology company focused on developing oral small-molecule and injectable protein therapeutics for rare diseases, with a market cap of $1.85 billion. Operations: BioCryst focuses on developing therapeutics for rare diseases, generating revenue primarily from its biotechnology segment, which reported $450.71 million. The company operates within the biotechnology sector and targets niche markets with its specialized products. BioCryst Pharmaceuticals, amid a transformative phase, is steering towards profitability with an expected annual profit growth of 56.3%, outpacing the industry average. The company's R&D commitment is evident from its expenditure trends, which are integral to its strategy in developing treatments for rare diseases—a sector marked by high entry barriers and significant patient need. Recent leadership enhancements, including the appointment of a seasoned investment banker to its board, align with BioCryst's aim to fortify its financial and strategic acumen as it anticipates revenue growth of 13.4% annually. This trajectory is supported by increased revenue guidance for 2025, projecting sales between $560 million and $575 million due to robust performance of their flagship product ORLADEYO® in clinical trials. Unlock comprehensive insights into our analysis of BioCryst Pharmaceuticals stock in this health report. Explore historical data to track BioCryst Pharmaceuticals' performance over time in our Past section. Simply Wall St Growth Rating: ★★★★☆☆ Overview: trivago N.V. operates a hotel and accommodation search platform across various international markets, with a market cap of approximately $292.77 million. Operations: The company generates revenue primarily through advertising services on its hotel and accommodation search platform, leveraging a cost-per-click model. Operating in key markets like the United States, Germany, and the United Kingdom, it focuses on connecting users with online travel agencies and hotel chains. The business has shown variability in its net profit margin over recent periods. Trivago N.V. is navigating a transformative landscape with a recent uptick in revenue, marking a 9.3% annual growth, surpassing the U.S. market average of 8.3%. This surge is underscored by their Q1 2025 earnings where sales rose to EUR 124.11 million from EUR 101.43 million year-over-year, despite a net loss reduction to EUR 7.8 million from EUR 8.38 million previously reported in the same period last year. The appointment of Dr. Wolf Schmuhl as CFO hints at strategic shifts aimed at reinforcing governance and financial oversight amidst these challenging yet promising times for the company, which has historically struggled with profitability but is now on a path forecasted to achieve profit growth of approximately 70.8% annually over the next three years. Take a closer look at trivago's potential here in our health report. Examine trivago's past performance report to understand how it has performed in the past. Embark on your investment journey to our 236 US High Growth Tech and AI Stocks selection here. Are you invested in these stocks already? Keep abreast of every twist and turn by setting up a portfolio with Simply Wall St, where we make it simple for investors like you to stay informed and proactive. Join a community of smart investors by using Simply Wall St. It's free and delivers expert-level analysis on worldwide markets. Explore high-performing small cap companies that haven't yet garnered significant analyst attention. Fuel your portfolio with companies showing strong growth potential, backed by optimistic outlooks both from analysts and management. Find companies with promising cash flow potential yet trading below their fair value. This article by Simply Wall St is general in nature. We provide commentary based on historical data and analyst forecasts only using an unbiased methodology and our articles are not intended to be financial advice. It does not constitute a recommendation to buy or sell any stock, and does not take account of your objectives, or your financial situation. We aim to bring you long-term focused analysis driven by fundamental data. Note that our analysis may not factor in the latest price-sensitive company announcements or qualitative material. Simply Wall St has no position in any stocks mentioned. Companies discussed in this article include NasdaqGM:MIRM NasdaqGS:BCRX and NasdaqGS:TRVG. Have feedback on this article? Concerned about the content? with us directly. Alternatively, email editorial-team@ Sign in to access your portfolio
Yahoo
30-04-2025
- Business
- Yahoo
Mirum Pharmaceuticals to Announce First Quarter 2025 Financial Results and Host Conference Call on May 7, 2025
FOSTER CITY, Calif., April 30, 2025--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will report first quarter 2025 financial results on Wednesday, May 7, 2025. Mirum will also host a conference call to discuss the first quarter 2025 financial results and recent corporate progress. Conference call details: Wednesday, May 7, 2025 4:30 p.m. ET / 1:30 p.m. PT Dial-in: U.S./Toll-Free: + 1 833 470 1428 International: + 1 404 975 4839 Passcode: 549600 You may also access the call via webcast by visiting the Events & Presentations section on Mirum's website. A replay of this webcast will be available for 30 days. About Mirum Pharmaceuticals, Inc. Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution/LIVMARLI® (maralixibat) tablets, CHOLBAM® (cholic acid) capsules, and CTEXLI™ (chenodiol) tablets. LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It 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 other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum has initiated the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. 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 of liver disease. CTEXLI is FDA-approved for the treatment of cerebrotendinous xanthomatosis (CTX) in adults. Mirum's late-stage pipeline includes two investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Mirum is also planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder. To learn more about Mirum, visit and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (X). View source version on Contacts Investor Contacts:Andrew McKibbenir@ Media Contact:Erin Murphymedia@ Sign in to access your portfolio
