Fragile X Syndrome

FDA Authorizes Expanded Access Program (EAP) to Help Provide Access to SPG302 for 200 ALS Individuals Ineligible for Clinical Study EAP Supports Real-world Data Collection Concurrent to Ongoing Clinical Studies of SPG302 LOS ANGELES, May 5, 2025 /PRNewswire/ -- Spinogenix, Inc., a clinical-stage biopharmaceutical company pioneering first-in-class therapeutics that restore synapses to improve the lives of patients worldwide, today announced that it has received notification from the Food and Drug Administration (FDA) to begin its Expanded Access Program (EAP) for SPG302 in the United States (U.S.) for people living with Amyotrophic Lateral Sclerosis (ALS) that meet program eligibility criteria. SPG302 is being developed as the first synaptic regenerative approach to treating ALS, with the potential to slow or reverse declines in cognitive and motor function. Spinogenix CEO and Founder Dr. Stella Sarraf stated, "FDA clearance to launch this new EAP supports its awareness of SPG302's safety and potential efficacy, and the need for ALS patients to have access to novel treatments. This EAP reflects our dedication to giving hope to people living with ALS and their families, providing treatment to those who do not qualify for, or may be unable to participate in, our ongoing clinical trial. Innovation is urgently needed to tackle ALS, a disease with devastating impact, and we are committed to ensuring that no patients who may benefit are left behind." EAPs are designed to allow individuals with serious or life-threatening conditions to access investigational treatments outside of a clinical trial that are not yet approved by the FDA. Dan Doctoroff, founder and chairman of Target ALS, and the first individual to receive SPG302 in the U.S. as part of a separate EAP protocol commented, "The notion that ALS is an untreatable disease is outdated. My own experience with SPG302, the first synaptic regenerative therapy in clinical development, has shown that partial stabilization of the disease is a possibility. I am thrilled that the FDA has recognized the value of allowing Spinogenix to expand access to SPG302, allowing others battling ALS to have a similar beneficial experience." In addition to serving the ALS community by providing access to an innovative investigational therapy, the EAP enables the collection of real-world data on SPG302 safety and efficacy that can support its clinical development. The FDA previously granted SPG302 Orphan Drug Designation for the treatment of ALS. Spinogenix recently completed a Phase 2 ALS trial in Australia, and patients have been offered a continuation of treatment in an open label extension. Additional information on the trial (NCT05882695) and open label extension (NCT06903286) may be found on ClinicalTrials.gov. About SPG302 SPG302 is a once-a-day pill being developed as a regenerative treatment for neurodegenerative and neuropsychiatric diseases with the unique ability to restore synapses, the key connections between neurons that allow people to think, plan, remember, and control movement. The synaptic regenerative activity of SPG302 represents a first-in-class approach to treating these diseases and has the potential to reverse declines in cognitive, respiratory, and motor function. SPG302 has been granted U.S. FDA Orphan Drug Designation for the treatment of ALS and has received preclinical support from the U.S. National Institutes of Health and the Department of Defense. Spinogenix has completed a Phase 1/2 study in Australia (NCT05882695) assessing the safety, pharmacokinetics and pharmacodynamics of once-daily dosing of SPG302 in healthy volunteers and up to 48 weeks of treatment in ALS patients. Additional information on the global clinical trials evaluating SPG302 for the treatment of schizophrenia and Alzheimer's disease can be found on ClinicalTrials.gov (NCT06442462 and NCT06427668). About Expanded Access to SPG302 Access to SPG302 is available in the U.S. for certain adults with ALS who meet eligibility criteria for participation in the FDA-authorized Expanded Access Program for SPG302. More information about the U.S. EAP can be requested through [email protected]. About Spinogenix Current treatments for neurodegenerative, neuropsychiatric and neurodevelopmental conditions primarily focus on slowing disease progression or minimizing symptoms, leaving many without hope for improvement. Spinogenix is aiming to transform the treatment of these conditions through its pioneering first-in-class and paradigm-shifting synaptic regenerative and synaptic corrective therapeutics designed to restore depleted synapses and reverse synaptic degeneration and dysfunction – offering patients and their families a new reality of hope. Spinogenix is developing two novel therapeutics: SPG302, which triggers neurons to produce new glutamatergic synapses and restore cognitive, motor, and other functions in ALS, Alzheimer's disease, schizophrenia and other diseases; and SPG601, which works at the synaptic level to correct specific dysfunctions in Fragile X Syndrome (FXS) that underlie many core symptoms. The company has received FDA Orphan Drug designation for both ALS and FXS, as well as FDA Fast Track designation for FXS. More information on Spinogenix can be found at or follow us on LinkedIn. Media Relations Nechama Rosengarten FINN Partners [email protected] Investor Relations Dan Albosta Spinogenix, Inc. [email protected] SOURCE Spinogenix WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- 28-week data from Phase 2b VANTAGE PBC study highlighting improvements in itch and fatigue will be presented at EASL - Data from Mirum studies presented at DDW, EASL, and ESPGHAN congresses FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM) today announced that it will present data at three upcoming medical congresses. Digestive Disease Week (DDW) will be held in San Diego, May 3-6, 2025. The European Association for the Study of the Liver (EASL) will take place May 7-10 in Amsterdam, the Netherlands. The 57th European Society for Paediatric, Gastroenterology, Hepatology, and Nutrition (ESPGHAN) Annual Meeting will follow May 14-17 in Helsinki, Finland. Data from Mirum’s LIVMARLI and volixibat studies will be highlighted in oral and poster presentations during the congresses. “We are excited to see the continued evidence generated by Mirum medicines and product candidates across a number of settings,” said Joanne Quan, MD, chief medical officer at Mirum. “In particular, we look forward to sharing updated data from the interim analysis of the VANTAGE study of volixibat in patients with pruritus due to PBC. We are encouraged by the positive results which show a meaningful decrease in pruritus that lasts for the duration of the placebo-controlled period.” EASL – May 7-10, 2025 Abstract #OS-059 (Oral): Volixibat for the treatment of cholestatic pruritus in primary biliary cholangitis: an adaptive, randomized, placebo-controlled phase 2B trial (VANTAGE): 28-week interim analysis Friday, May 9 from 8:30-8:45am CEST Immune-mediated and Cholestatic Diseases Session, McIntyre Room Presented by: Michael Heneghan, MD, MMedSc, FRCPI, King’s College, London, UK New 28-week data were reported from the Phase 2 VANTAGE study evaluating volixibat in patients with cholestatic pruritus related to primary biliary cholangitis (PBC). Thirty-one patients with moderate-to-severe pruritus were randomized (20mg VLX=10, 80mg VLX=10, placebo=11). Data showed that volixibat led to early and significant reductions in PBC-associated cholestatic pruritus and were maintained throughout the duration of the study. Further, 70% of volixibat-treated patients had a ≥50% reduction in serum bile acid levels from baseline. Additionally, patients treated with volixibat experienced improvements in fatigue and sleep. No new safety signals were observed through 28 weeks of treatment. The most common treatment-emergent adverse event was diarrhea which was mild to moderate in severity and transient in nature. Additional data will be shared following the formal presentation on May 9. EASL Symposium Wednesday, May 7 from 1:30-2:30pm CEST Adult Cholestatic Liver Disease: Focusing on Clinical Outcomes and the Future of IBAT Inhibitors An archive of the symposium will be available following the event. DDW Oral Presentations Presentation 784: Volixibat for the Treatment of Cholestatic Pruritus in Primary Biliary Cholangitis: An Adaptive, Randomized, Placebo-controlled Phase 2B Trial (VANTAGE): Interim Results Monday, May 5 from 2:15-2:30pm PT Cholestatic Liver Disease session, Room 6C Presented by Mitchell Schiffman, MD, Bon Secours Liver Institute, Virginia, USA Presentation 1086: Comprehensive Analysis of Cholestasis Genetic Panel Results From 2016–2022 in Children and Young Adults: Insights into Diagnostic Yield Tuesday, May 6 from 10:00-10:15am PT Human and Experimental Cholestatic and Autoimmune Liver Diseases, Room 6F Presented by Brett Hoskins, DO, Indiana University School of Medicine, Indiana, USA Poster Presentations Poster Su1756: Genetic Insights Into PFIC-associated Genes in Unexplained Chronic Cholestasis and Liver Disease: Frequency and Implications of Variant Combinations Sunday, May 4 from 12:30-1:30pm PT Pediatric Hepatology Session, DDW Poster Hall, C-E Presented by Brett Hoskins, DO, Indiana University School of Medicine, Indiana, USA Poster Mo1627: Improvements in Pruritus, Serum Bile Acids, and Total Bilirubin Following Treatment with Maralixibat in Patients with Primary Sclerosing Cholangitis Monday, May 5 from 12:30-1:30pm PT Human and Experimental Cholestatic and Autoimmune Liver Diseases, DDW Poster Hall C-E Presented by Jessica Hochberg, MD, University of Miami Miller School of Medicine, Florida, USA ESPGHAN – May 14-17, 2025 Oral presentations Abstract 1066: Improvements in pruritus are associated with growth in patients with progressive familial intrahepatic cholestasis: Data from the MARCH-ON trial Saturday, May 17 from 9:15-11:15am EEST Nutrition and Hepatology Session, Hall 3G Presented by Professor Richard Thompson, King’s College, London, UK Abstract 1089: The relationship between serum bile acids and event-free survival following the use of maralixibat for progressive familial intrahepatic cholestasis (PFIC): Data from the MARCH/MARCH-ON trials Saturday, May 17 from 1:10-2:40pm EEST Hepatology Abstracts, Hall 3G Presented by Professor Richard Thompson, King’s College, London, UK Abstract 1110: Improvements in pruritus after maralixibat treatment are associated with improved health-related quality of life for patients with cholestatic liver disease Saturday, May 17 from 1:10-2:40pm EEST Hepatology Abstracts, Hall 3G Presented by Dr. Emmanuel Gonzales, Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP. Université Paris-Saclay, Le Kremlin-Bicêtre, France Abstract 1098: Bile acid subspecies are correlated with pruritus and bilirubin improvement in PFIC patients treated with maralixibat: Data from MARCH and MARCH-ON Saturday, May 17 from 1:10-2:40pm EEST Hepatology Abstracts, Hall 3G Presented by Dr. Henkjan Verkade, University of Groningen, Beatrix Children’s Hospital and University Medical Center Groningen, Netherlands Poster presentations Abstract 1102: Greater improvements in bilirubin were observed in pruritus responders after maralixibat treatment in patients with PFIC: data from the MARCH/MARCH-ON trials Friday, May 16 from 3:30-4:20pm EST General Hepatology 02 Session, e-Poster Station 08 Presented by Professor Richard Thompson, King’s College, London, UK Abstract 1094: Impact of long-term maralixibat treatment on concomitant medication use for the treatment of cholestatic pruritus in Alagille syndrome: Real-world experience in the United States Friday, May 16 from 3:30-4:20pm EEST General Hepatology 03 & Transplantation Session, e-Poster Station 09 Presented by Dr. Tony Tokman, Mirum Pharmaceuticals, Inc., Foster City, California USA Abstract 1107: Clinical benefits of maralixibat for patients with Alagille syndrome are durable through 7 years of treatment: Data from the MERGE study Friday, May 16 from 3:30-4:20pm EEST General Hepatology Session 02, e-Poster Station 08 Presented by Dr. Emmanuel Gonzales, Hépatologie Pédiatrique, Hôpital Bicêtre, AP-HP. Université Paris-Saclay, Le Kremlin-Bicêtre, France ESPGHAN Symposium Thursday, May 15 from 12:45-1:45pm EEST Hall 3C Growing Knowledge and Evidence: Long-term impact of IBAT inhibition Full presentations will be available within the Publications and Presentations section on Mirum’s website. 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 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.com. 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. US Prescribing Information EU SmPC Canadian Product Monograph 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 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, the anticipated presentations of data from Mirum’s LIVMARLI and volixibat studies, including the VANTAGE study of volixibat in patients with pruritus due to PBC. 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 www.sec.gov. 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. Media Contact: Erin Murphy media@mirumpharma.com Investor Contact: Andrew McKibben ir@mirumpharma.com

LIVMARLI Tablet formulation approved for patients with Alagille Syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC) FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today announced that the U.S. Food and Drug Administration (FDA) has approved a new tablet formulation of LIVMARLI® (maralixibat) for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) and Progressive Familial Intrahepatic Cholestasis (PFIC). “The approval of LIVMARLI in tablet form provides a meaningful additional treatment option for patients with ALGS and PFIC. It allows flexibility for patients and physicians, with the liquid dosing used by the youngest patients and a convenient one-tablet per dose option for older patients,” said Peter Radovich, president and chief operating officer at Mirum. “We have had tremendous success with LIVMARLI since its launch and we hope that the availability of the tablet will provide convenience that positively impacts patients’ lives.” LIVMARLI tablets are planned to be available in June through Mirum Access Plus. 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.com. 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. US Prescribing Information EU SmPC Canadian Product Monograph 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 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, the potential benefits of a tablet formulation of LIVMARLI versus a liquid formulation, the availability of a tablet formulation for all patients taking LIVMARLI and the real life usage patterns of patients on LIVMARLI. 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 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’s Quarterly Report on Form 10-K for the quarter ended December 31, 2024 and subsequent filings with the U.S. Securities and Exchange Commission and available at www.sec.gov. Media Contact: Erin Murphy media@mirumpharma.com Investor Contact: Andrew McKibben ir@mirumpharma.com