Latest news with #Peetz
Business Wire
6 days ago
- Business
- Business Wire
Mirum Pharmaceuticals Reports Second Quarter 2025 Financial Results and Provides Business Update
FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the second quarter 2025 and provided a business update. 'Our second quarter results once again underscore the strength of our commercial programs with notable outperformance from our International business and U.S. PFIC launch,' said Chris Peetz, chief executive officer of Mirum. 'The momentum we are seeing with Livmarli globally reinforces our belief that the medicine will reach and help more patients than we initially projected, allowing us to raise guidance with confidence.' Peetz added, 'We are carrying that same momentum into our pipeline, where we are on course for three important late stage clinical milestones in 2026. Notably, the VISTAS study of volixibat in PSC will complete enrollment this quarter, and we plan to report topline data in the second quarter of next year.' Commercial: Raising full year revenue guidance to $490 to $510 million Second quarter 2025 global net product sales of $127.8 million. Second quarter 2025 LIVMARLI net product sales were $88.2 million, representing 87% growth over second quarter 2024 net product sales. LIVMARLI single oral tablet dose U.S. launch in June. Bile Acid Medicines second quarter 2025 net product sales were $39.6 million, representing 30% growth over second quarter 2024 net product sales. Pipeline: Advancing toward multiple 2026 milestones Volixibat VISTAS study in primary sclerosing cholangitis (PSC) topline data expected in the second quarter of 2026. 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 the fourth quarter of 2025. Corporate and Financial: Strong balance sheet and financial independence Total revenue for the quarter ended June 30, 2025, was $127.8 million compared to $77.9 million for the quarter ended June 30, 2024. Total operating expenses were $132.8 million for the quarter ended June 30, 2025, compared to $102.1 million for the quarter ended June 30, 2024. Total operating expenses for the quarter ended June 30, 2025, included $24.5 million of non-cash stock-based compensation, intangible amortization, and other non-cash expenses compared to $17.7 million for the quarter ended June 30, 2024. As of June 30, 2025, Mirum had unrestricted cash, cash equivalents, and investments of $321.7 million compared to $292.8 million as of December 31, 2024. Business Update Conference Call Mirum will host a conference call today, August 6 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 Access Code: 448461 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) 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 2024, 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, 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, continued positive 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 initiation, results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, the timing and results of interim analyses of, and topline data for, 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) December 31, 2024 Assets Current assets: Cash and cash equivalents $ 228,122 $ 222,503 Short-term investments 76,430 57,812 Accounts receivable 106,836 78,286 Inventory 22,941 22,403 Prepaid expenses and other current assets 19,069 11,784 Total current assets 453,398 392,788 Restricted cash 480 425 Long-term investments 17,112 12,526 Intangible assets, net 237,870 249,819 Other noncurrent assets 16,965 15,196 Total assets $ 725,825 $ 670,754 Liabilities and Stockholders' Equity Current liabilities: Accounts payable $ 17,657 $ 14,618 Accrued expenses and other current liabilities 127,139 111,933 Total current liabilities 144,796 126,551 Operating lease liabilities, noncurrent 7,884 7,972 Convertible notes payable, net, noncurrent 308,933 308,082 Other liabilities 9,054 2,509 Total liabilities 470,667 445,114 Commitments and contingencies Stockholders' equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 919,520 870,189 Accumulated deficit (664,719 ) (644,181 ) Accumulated other comprehensive income (loss) 352 (373 ) Total stockholders' equity 255,158 225,640 Total liabilities and stockholders' equity $ 725,825 $ 670,754 Expand
Chicago Tribune
05-02-2025
- Sport
- Chicago Tribune
Katie Peetz, whose mom played for Doug Bruno at DePaul, is a chip off the old block for Lockport. ‘Do everything.'
When it comes to her style of play for Lockport, sophomore guard/forward Katie Peetz is taking after her mother. Sylvia, whose maiden name is Blakeslee, played basketball at DePaul. In 1998, legendary Blue Demons coach Doug Bruno told the Chicago Tribune that she was 'our jack of all trades.' Twenty-seven years later, Lockport coach Darrien Jacobs has a similar appraisal of Peetz. 'Katie's role is to do everything,' Jacobs said. 'And she's good at that.' Peetz showed off that versatility Tuesday night, coming up with nine points, six rebounds and four steals for the host Porters in a 51-47 overtime victory over Lincoln-Way West in a battle between the top two teams in the SouthWest Suburban Conference. Senior forward Alaina Peetz, Katie's sister, led the Porters (24-4, 13-2) with 12 points and added six rebounds and five assists. Evelyn Ingram contributed 10 points, four rebounds and two steals. Amanda Way came off the bench and scored five points, including the go-ahead basket with 1:25 remaining in the OT to give the Porters a 48-46 lead. They never trailed after that. Molly Finn led Lincoln-Way West (19-8, 11-2) with 17 points, while Ava Tisch added 10 points. Caroline Smith tallied nine points and 13 rebounds, with 10 rebounds in the first half. Katie Peetz, meanwhile, knows her role for Lockport and completely embraces it. 'I will play any position that the coaches want me to,' she said. 'Sometimes I'll make blocked shots or sometimes I'll shoot threes. It depends on the day.' What part of the game does she like best? 'I like a mix,' she said. 'It's been perfect, honestly.' Jacobs, who has a variety of talented players with various skills on his team, pointed out that some of the pressure for Peetz to perform has been spread out. 'Katie doesn't have a lot of pressure on her shoulders because we don't need her to come out and score in double digits,' Jacobs said. 'We don't need her to be our primary ballhandler. 'We just need her to hustle, and that's what she does. She gets the loose balls. She gets the rebounds. She plays solid defense. That's all you can ask from her.' Asked and answered. Way, a junior guard, enjoys watching Peetz go to work for the Porters. 'Katie's shot was on and so was her rebounding,' Way said. 'She is always a great player, but we needed that in this game.' And Lockport also needed Way's conversion of that 10-foot jump shot in OT. 'It was awesome to have that opportunity,' Way said. 'I saw an opening in the lane so when I cut. I got the ball. I was nervous, but it was the best opening I might get, so I just knew to take it.' If Lockport wins its last conference game Tuesday against Sandburg, the Porters are guaranteed at least share of the title. Since the conference was formed for the 2005-2006 season, Lockport has never won a conference title. Even more impressive, this is the first season the conference hasn't been split up into two divisions. Not bad for a team that ended up with a 13-19 record last season and also went winless in eight games in the SouthWest Suburban Blue. No doubt, it's something few people saw coming. 'It's a ton different this year,' Peetz said. 'Last year, we would have fun and play but we knew we weren't going to be great. This year, it's like, 'Oh my gosh, we're actually good.' 'We could seriously do something this year. This year is still fun. There is some pressure, but it's good pressure. You want to have this type of pressure. I think everybody would.' Jeff Vorva is a freelance reporter for the Daily Southtown. Originally Published: February 4, 2025 at 11:23 PM CST
