Zellweger Syndrome

10 scientists have been selected for OHC's first annual award to advance academic discoveries into clinical practice for rare diseases OXFORD, United Kingdom and CLEVELAND, Oct. 24, 2024 /PRNewswire/ -- The Oxford-Harrington Rare Disease Centre – a partnership between the University of Oxford, UK and Harrington Discovery Institute at University Hospitals, Cleveland, Ohio – today announces the first class of Oxford-Harrington Rare Disease Scholars. The scholar awards will support breakthrough treatments for rare and ultra-rare diseases across neurologic, oncologic, and metabolic disease areas, using small molecule, nucleic acid, viral, and cellular therapies. The recipients represent institutions across the UK, US, and Canada. The OHC brings together world-leading research, expertise in therapeutics development and new financial models to advance solutions to critical challenges in rare diseases. Each Scholar will receive £100,000 (UK) / $100,000 (US and Canada), personalised therapeutics development support from industry experts to help bring new therapeutics to market, and the opportunity to receive acceleration and investment funds of up to £1,000,000 based on project milestones. Matthew Wood, MD, PhD, Director and Chief Scientific Officer of the OHC, commented: "This first cohort of OHC Scholars is a testament to the outstanding quality of proposals we received from academics at institutions across the UK and North America. The breadth and standard of research into innovative approaches to treat rare diseases made our selection process very difficult, with successful proposals representing breakthrough discoveries defined by innovation, creativity and potential for clinical impact. Our congratulations go to the 2024 OHC Scholars and our sincere thanks go to all who submitted proposals – their continued efforts to make a meaningful difference for patients in need and their families are an inspiration to us all." Jonathan S. Stamler, MD, President of Harrington Discovery Institute, added: "By supporting each OHC Scholar with funding and an experienced therapeutics development team, we are maximising the potential of their discoveries to advance into the clinic. We have seen success with this approach at Harrington Discovery Institute and we are excited about replicating the model at the OHC. I look forward to seeing the progress of these innovative initiatives and sharing their advancements as the OHC continues to work towards providing scalable solutions to address the rare disease challenge globally." The 2024 OHC Rare Disease Scholar Award recipients, their organisations and fields of research are: Jacquelyn Bower, PhD – University of North Carolina at Chapel Hill, NC, USA Novel viral gene therapy to treat lethal rare eye tumours Louis Chesler, MD, PhD – Institute of Cancer Research, London, UK Regulatable cell therapies to treat brain cancers Charles Gersbach, PhD – Duke University, Durham, NC, USA Reprogramming immune cells for treatment of Hodgkin's lymphoma Xianxin Hua, MD, PhD – University of Pennsylvania, Philadelphia, PA, USA A new cellular strategy for treating acute myeloid leukaemia Michele Jacob, PhD – Tufts University School of Medicine, Medford, MA, USA A drug to treat brain abnormalities caused by CTNNB1 defects, an ultra-rare disease Bowen Li, PhD – University of Toronto, Toronto, Canada A nucleic acid therapy to treat cystic fibrosis, an inherited lung disease Michael Pacold, MD, PhD – New York University, New York City, NY, USA Drugs restoring brain function in rare metabolic diseases Carlo Rinaldi, MA, MD, PhD – University of Oxford, Oxford, UK Nucleic acid therapy for spinal and bulbar muscular atrophy (SBMA), a rare X-linked neuromuscular condition Timothy Yu, MD, PhD – Boston Children's Hospital, Boston, MA, USA Custom DNA-based therapy for a rare genetic disorder (PEX1 Zellweger Syndrome) Haiyan Zhou, MD, PhD – University College London, London, UK Nucleic acid therapy to treat loss of sensation and paralysis caused by a rare mutation For those interested in submitting a proposal for the Oxford-Harrington Rare Disease Scholar Award, the next funding call will open on the 15th of January 2025. For more information, visit: OxfordHarrington.org/funding/overview SOURCE Harrington Discovery Institute WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

First quarter 2024 total revenue of $69.2 million, on track to achieve full-year guidance of $310 to $320 million FDA approval of LIVMARLI for cholestatic pruritus in PFIC patients achieved in March Volixibat VISTAS and VANTAGE interim analyses scheduled for June of 2024 Cash balance of $302.8 million as of March 31, 2024 Conference call to provide business updates today, May 8 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 first quarter 2024 and provided a business update. “In the first quarter, we made great progress across a number of our strategic objectives that support both the near- and long-term growth of Mirum while maintaining a strong financial position,” said Chris Peetz, chief executive officer at Mirum. “Our commercial business is performing well, and we saw continued growth in demand for LIVMARLI globally putting us on track for our total revenue guidance target for the year. The FDA’s approval of LIVMARLI for cholestatic pruritus in patients with PFIC was also an important step in bringing our high impact medicines to the rare disease patient community. We look forward to continuing this momentum with our upcoming NDA submission for CHENODAL in CTX, and important interim analyses in our PSC and PBC studies in June.” Commercial: Continued demand growth and reiterate full year guidance of $310-$320 million First quarter 2024 global product sales, net of $68.9 million grew 137% compared to first quarter 2023. LIVMARLI first quarter net sales were $42.8 million, representing 47% growth compared to first quarter 2023. LIVMARLI approved in the US for treatment of cholestatic pruritus in patients with PFIC. First quarter 2024 CHOLBAM and CHENODAL net sales were $26.1 million. Regulatory and Pipeline: H1 Milestones on track Interim analyses for volixibat in PSC (VISTAS study) and in PBC (VANTAGE study) expected in June of 2024. New Drug Application (NDA) submission for CHENODAL in CTX on track for first half 2024. PFIC CHMP approval recommendation anticipated in the first half of 2024. Corporate and Financial: Strong balance sheet Total revenue for the quarter ended March 31, 2024, was $69.2 million compared to $31.6 million for the quarter ended March 31, 2023. Total operating expenses were $95.7 million for the quarter ended March 31, 2024, compared to $58.7 million for the quarter ended March 31, 2023. Total operating expense included $17.1 million of non-cash stock-based compensation and depreciation and amortization expense for the quarter ended March 31, 2024, compared to $9.9 million for the quarter ended March 31, 2023. As of March 31, 2024, Mirum had cash and cash equivalents of $302.8 million compared to $286.3 million as of December 31, 2023. Business Update Conference Call Mirum will host a conference call today, May 8, 2024, 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: 479107 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 LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older and progressive familial intrahepatic cholestasis (PFIC) five years of age and older. LIVMARLI is also the only approved IBAT inhibitor approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older, and by Health Canada for the treatment of cholestatic pruritus in ALGS. For more information for U.S. residents, please visit LIVMARLI.com. Mirum has also submitted LIVMARLI for approval in Europe in PFIC for patients two months of age and older. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. 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 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. US Prescribing Information 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. Phase 1 and Phase 2 studies of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of ASBT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (VANTAGE study). 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 CHENODAL® (chenodiol) tablets CHENODAL® is a synthetic oral form of chenodeoxycholic acid (CDCA), a naturally occurring primary bile acid synthesized from cholesterol in the liver. The FDA approved CHENODAL for the treatment of people with radiolucent stones in the gallbladder. In 2010, CHENODAL was granted orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare autosomal recessive lipid storage disease. While CHENODAL® is not currently approved for CTX, it received a medical necessity determination in the U.S. by the FDA and has been used as the standard of care for more than three decades. A Phase 3 clinical trial for this indication has recently been completed and based on the positive results, a New Drug Application (NDA) submission for CHENODAL in CTX planned in first half 2024. We believe the prevalent patient population in the United States with CTX exceeds 1,000. 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, CHOLBAM® (cholic acid) capsules, and CHENODAL® (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 five years of age and older. Mirum has submitted for approval in Europe for the treatment of PFIC in 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). Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. To learn more about Mirum, visit mirumpharma.com 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, progress across Mirum’s strategic objectives, its near- and long-term growth, the strength of its financial position, the performance of its commercial business, growth in demand for LIVMARLI, achievement of full-year revenue guidance, the results, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, the timing and results of interim analyses of Mirum’s ongoing studies and the regulatory approval path for its product candidates globally. 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,” “forecasts,” “forward,” “planned,” “poised,”, “positioned” “potential”, “will” 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 on Form 10-K for the year ended December 31, 2023 and subsequent filings with the Securities and Exchange Commission. 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 Statement of Operations Data (in thousands, except share and per share amounts) (Unaudited) Three Months Ended March 31, 2024 2023 Revenue: Product sales, net $ 68,917 $ 29,098 License and other revenue 305 2,500 Total revenue 69,222 31,598 Operating expenses: Cost of sales (1) 17,830 4,979 Research and development 32,222 23,548 Selling, general and administrative 45,638 30,219 Total operating expenses (2) 95,690 58,746 Loss from operations (26,468 ) (27,148 ) Other income (expense): Interest income 3,633 2,272 Interest expense (3,577 ) (4,242 ) Other income (expense), net 1,757 (811 ) Net loss before provision for income taxes (24,655 ) (29,929 ) Provision for income taxes 624 201 Net loss (25,279 ) (30,130 ) Net loss per share, basic and diluted $ (0.54 ) $ (0.80 ) Weighted-average shares of common stock outstanding, basic and diluted 46,927,550 37,675,306 (1) Amounts include intangible amortization expense as follows: Intangible amortization $ 5,402 $ 1,259 (2) Amounts include stock-based compensation expense as follows: Research and development $ 3,861 $ 2,715 Selling, general and administrative 7,589 5,846 Total stock-based compensation $ 11,450 $ 8,561 Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (Unaudited) March 31, 2024 December 31, 2023 Assets Current assets: Cash and cash equivalents $ 302,843 $ 286,326 Accounts receivable 54,999 67,968 Inventory 21,606 22,312 Prepaid expenses and other current assets 10,017 10,935 Total current assets 389,465 387,541 Intangible assets, net 257,443 252,925 Other noncurrent assets 5,054 6,155 Total assets $ 651,962 $ 646,621 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 15,983 $ 7,416 Accrued expenses 89,568 78,544 Operating lease liabilities 358 1,104 Total current liabilities 105,909 87,064 Operating lease liabilities, noncurrent 328 617 Convertible notes payable, net 306,835 306,421 Other liabilities 4,287 3,849 Total liabilities 417,359 397,951 Commitments and contingencies Stockholders’ equity: Preferred stock — — Common stock 5 5 Additional paid-in capital 816,129 803,260 Accumulated deficit (581,518 ) (556,239 ) Accumulated other comprehensive (loss) income (13 ) 1,644 Total stockholders’ equity 234,603 248,670 Total liabilities and stockholders’ equity $ 651,962 $ 646,621 View source version on businesswire.com: Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Erin Murphy media@mirumpharma.com Source: Mirum Pharmaceuticals, Inc. 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- Net product sales totaled $178.9 million, and full year 2023 revenue totaled $186.4 million, delivering 142% growth over 2022 total revenue - 2024 expected global net product sales of $310 million to $320 million - LIVMARLI PFIC PDUFA date on track for March 13, 2024 - Volixibat VISTAS and VANTAGE interim analyses expected in first half of 2024 - Conference call to provide business updates today, February 28 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 fourth quarter and year-end 2023 and provided a business update. “2023 was a landmark year for Mirum as we significantly expanded our commercial footprint, realizing $179 million in net product sales, and achieved another year of strong global LIVMARLI growth. We also successfully integrated CHOLBAM and CHENODAL into our portfolio, which strengthened our position as a leader in rare disease,” said Chris Peetz, chief executive officer at Mirum. “In 2024, we look forward to further expanding the reach of our approved medicines, potential LIVMARLI and CHENODAL label expansions and interim analyses for our volixibat adult indications. Operationally and financially, we are well positioned for this next phase of growth and remain committed to our mission of delivering life changing medicines to patients in need.” Commercial: Leading Pediatric Hepatology Franchise Net product sales totaled $178.9 million, and full year 2023 revenue totaled $186.4 million, representing 142% growth over 2022 total revenue. 2023 LIVMARLI net product sales totaled $141.8 million, representing approximately 89% growth over 2022 net product sales. International business grew to 18 countries with reimbursed access to LIVMARLI. Expect continued revenue growth across all approved medicines in 2024 with expected global net product sales of $310 million to $320 million. Regulatory & Pipeline: Multiple Upcoming 2024 Milestones U.S. Food and Drug Administration (FDA) Prescription Drug User Free Act (PDUFA) date for LIVMARLI in progressive familial intrahepatic cholestasis (PFIC) is March 13, 2024. New Drug Application (NDA) submission for CHENODAL in CTX planned in first half 2024. Volixibat VISTAS study in primary sclerosing cholangitis blinded interim analysis expected in first half 2024. Volixibat VANTAGE study in primary biliary cholangitis interim analysis expected in first half 2024. Corporate and Financial: Strong Balance Sheet, Financially Sustaining Scale Total net product sales for the full year ended December 31, 2023, was $178.9 million compared to $75.1 million for the full year ended December 31, 2022. Total operating expenses were $293.0 million for the full year ended December 31, 2023, compared to $208.3 million for the full year ended December 31, 2022. The increase in year-over-year operating expense was due to a $32.2 million increase in cost of goods sold and a $56.8 million increase in sales, general and administrative expense, partially offset by a $4.2 million decrease in research and development expense. As of December 31, 2023, Mirum had cash and cash equivalents of $286.3 million. Announced the appointment of Joanne Quan, M.D. as Chief Medical Officer. The foregoing financial information is unaudited and subject to change, and actual results may vary from the foregoing. Business Update Conference Call Mirum will host a conference call today, February 28, 2024, at 1:30 p.m. PT/4:30 p.m. ET, to discuss its financial results, guidance, and 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: 089049 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 LIVMARLI® (maralixibat) oral solution is an orally administered, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) three months of age and older. LIVMARLI is also approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older. For more information for U.S. residents, please visit LIVMARLI.com. Mirum has also submitted LIVMARLI for approval in the U.S. for the treatment of cholestatic pruritus in patients with PFIC three months of age and older, and in Europe for the treatment of PFIC in patients two months of age and older. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website. IMPORTANT SAFETY INFORMATION LIVMARLI can cause side effects, including: Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests 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) or loss of appetite. Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. 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 is caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment. Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding. US Prescribing Information EU SmPC 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. Phase 1 and Phase 2 studies of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of ASBT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (VANTAGE study). 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 CHENODAL® (chenodiol) tablets CHENODAL® is a synthetic oral form of chenodeoxycholic acid (CDCA), a naturally occurring primary bile acid synthesized from cholesterol in the liver. The FDA approved CHENODAL for the treatment of people with radiolucent stones in the gallbladder. In 2010, CHENODAL was granted orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare autosomal recessive lipid storage disease. While CHENODAL® is not currently approved for CTX, it received a medical necessity determination in the U.S. by the FDA and has been used as the standard of care for more than three decades. A Phase 3 clinical trial for this indication has recently been completed and based on the positive results, a New Drug Application (NDA) submission for CHENODAL in CTX planned in first half 2024. We believe the prevalent patient population in the United States with CTX exceeds 1,000. 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, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets. LIVMARLI, 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 other regions globally. 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 three 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). Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter. 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 commercial success for our approved products, including growth in year over year net product sales, having sufficient cash and cash equivalent reserves, planned expansion into additional international markets, LIVMARLI label expansion into PFIC, the timing and results of LIVMARLI’s PDUFA, CHENODAL potential full indication in CTX, delivering life changes medicines and becoming a global leader in rare disease, the results, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for its product candidates globally. 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,” “forecasts,” “forward,” “planned,” “poised,”, “positioned” “potential”, “will” 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2023 and subsequent filings with the Securities and Exchange Commission. 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 Statement of Operations Data (in thousands, except share and per share amounts) (Unaudited) Three Months Ended Year ended December 31, December 31, 2023 2022 2023 2022 Revenue: Product sales, net $ 69,554 $ 27,906 $ 178,874 $ 75,062 License revenue - - 7,500 2,000 Total revenue 69,554 27,906 186,374 77,062 Operating expenses: Cost of sales (1) 22,510 4,494 44,529 12,374 Research and development 30,935 31,105 102,609 106,842 Selling, general and administrative 46,184 26,468 145,880 89,066 Total operating expenses (2) 99,629 62,067 293,018 208,282 Loss from operations (30,075 ) (34,161 ) (106,644 ) (131,220 ) Other income (expense): Interest income 3,775 2,143 13,735 3,857 Interest expense (3,563 ) (4,359 ) (15,105 ) (15,979 ) Change in fair value of derivative liability - 674 - 906 Loss from termination of revenue interest purchase agreement - - (49,076 ) - Other (expense) income, net (3,061 ) (588 ) (2,824 ) 365 Net loss before for income taxes (32,924 ) (36,291 ) (159,914 ) (142,071 ) Provision for (benefit from) income taxes 225 140 991 (6,406 ) Net loss $ (33,149 ) $ (36,431 ) $ (160,905 ) $ (135,665 ) Net loss per share: Basic $ (3.94 ) $ (4.01 ) Diluted $ (3.94 ) $ (4.02 ) Weighted-average shares of common stock outstanding: Basic 40,885,124 33,839,072 Diluted 40,885,124 33,982,493 (1) Amounts include intangible amortization expense as follows: Intangible amortization $ 5,305 $ 1,078 $ 10,404 $ 2,893 (2) Amounts include stock-based compensation expense as follows: Research and development $ 2,879 $ 2,531 $ 10,892 $ 10,050 Selling, general and administrative 6,841 4,365 24,131 16,957 Total stock-based compensation $ 9,720 $ 6,896 $ 35,023 $ 27,007 Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (Unaudited) December 31, 2023 December 31, 2022 Assets Current assets: Cash and cash equivalents $ 286,326 $ 28,003 Short-term investments - 123,716 Accounts receivable 67,968 23,994 Inventory 19,590 5,565 Prepaid expenses and other current assets 10,935 8,947 Total current assets 384,819 190,225 Restricted cash and cash equivalents - 100,000 Intangible assets, net 252,925 58,954 Other noncurrent assets 6,155 3,727 Total assets $ 643,899 $ 352,906 Liabilities and Stockholders’ Equity Current liabilities: Accounts payable $ 7,416 $ 8,690 Accrued expenses 77,161 54,018 Other current liabilities 1,104 2,021 Total current liabilities 85,681 64,729 Revenue interest liability, net - 140,351 Convertible notes, net 306,421 - Other noncurrent liabilities 617 5,789 Total liabilities 392,719 210,869 Commitments and contingencies Stockholders' equity: Preferred stock - - Common stock 5 4 Additional paid-in capital 803,260 535,074 Accumulated deficit (553,729 ) (392,824 ) Accumulated other comprehensive income (loss) 1,644 (217 ) Total stockholders' equity 251,180 142,037 Total liabilities and stockholders' equity $ 643,899 $ 352,906