Duodenal Diseases

HONG KONG, Jan. 20, 2025 /PRNewswire/ -- Researchers from the School of Engineering of the Hong Kong University of Science and Technology (HKUST) have successfully developed the world's smallest multifunctional biomedical robot, which is 60% smaller than current models. Capable of imaging, high-precision motion, and multifunctional operations like sampling, drug delivery, and laser ablation, the robot offers competitive imaging performance and a tenfold improvement in obstacle detection, paving the way for robotic applications in narrow and challenging channels of the human body, such as the lung's end bronchi and the oviducts. With a slim profile of just 0.95 mm—60% smaller than current endoscopic robots—this robot has accomplished an "impossible trinity" by simultaneously integrating all three significant specifications in one robot. It offers competitive imaging performance and extends obstacle detection distance up to ~9.4 mm, a tenfold improvement from theoretical limits. It also achieves remarkable motion precision (less than 30 μm) and substantially widens the imaging region by ~25 times the inherent view. Developed by Prof. SHEN Yajing, Associate Professor at the Department of Electronic and Computer Engineering (ECE) and his team, the robot's tiny size is made possible with four major components. These include an optical fiber array for capturing images inside the body, a custom tool for delivering treatments precisely where needed, a hollow skeleton to hold the fibers and tools in place, and a functionalized skin that enables precise control of the robot's movements. The hollow skeleton is created by a microscale 3D printer, while the functionalized skin is produced through a magnetic spray technique, which helps keep the robot small and allows it to glide easily during surgery. It also features a gel-like outer layer that reduces friction. The team has tested this robot within in vitro bronchial models and ex-vivo porcine lungs, demonstrating smooth navigation in tight spaces while successfully capturing clear images and performing treatments on difficult areas. Prof. Shen said that this groundbreaking robot holds tremendous potential for clinical applications. "Small-scale continuum robots hold promise for interventional diagnosis and treatment, yet existing models often struggle with compactness, precise navigation, and visualized functional treatment all in one. Our study provides a significant solution for developing a surgical robot aimed at achieving early diagnosis and therapeutic goals in hard-to-reach areas of the body. With ongoing technological advancements, we believe that the fiberscopic robot will make greater contributions to human health in the foreseeable future," he said. Thanks to their ability to navigate narrow cavities while enabling quick recovery and low infection risk, small continuum robots have been employed in the treatment of several diseases, including heart disease—through the deployment of stents and electrophysiology catheters—as well as the repair of perforations in gastric and duodenal ulcers using single-port laparoscopy, among other applications. Building on this successful invention, the research team plans to further refine the robot's features to fit them into practical settings. "We aim to further optimize the design and control of the fiberscopic robot, prioritizing safety and reliability during interventional surgery. We look forward to implementing in vivo trials to demonstrate its performance in clinical scenarios," said Dr. ZHANG Tieshan, a postdoctoral fellow at HKUST. He is one of the two co-first authors of the study, along with Dr. LI Gen. Other co-authors from HKUST include Research Assistant Professor Dr. YANG Xiong and PhD student ZHAO Haoxiang, also from the ECE Department. Their findings have been recently published in Nature Communications. Download photos here: Watch the introduction video here: SOURCE The Hong Kong University of Science and Technology WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

New Commercial Supply Contracts for Vafseo® (vadadustat) secured, bringing availability and dialysis organization coverage to nearly 100% of patients on dialysis in U.S. Vafseo tablets now shipping in the U.S. Plan to start Phase 3 trial in mid-2025 to potentially expand Vafseo label to include treatment of late-stage non-dialysis CKD patients Company to discuss business updates at 43rd Annual J.P. Morgan Healthcare Conference CAMBRIDGE, Mass., Jan. 13, 2025 /PRNewswire/ -- Akebia Therapeutics®, Inc. (Nasdaq: AKBA), a biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease, today announced multiple positive business updates. As of January, Vafseo® (vadadustat) tablets are available in the U.S. for adult patients with anemia due to chronic kidney disease (CKD) on dialysis for at least three months, and shipment of product to dialysis centers has commenced. In addition, Akebia recently signed a Vafseo commercial supply agreement with a leading dialysis organization. Akebia now has contracts in place with dialysis organizations caring for nearly 100% of dialysis patients in the U.S. As previously announced, Vafseo qualifies for the Transitional Drug Add-On Payment Adjustment (TDAPA) reimbursement as directed by the Centers for Medicare & Medicaid Services (CMS), and CMS has published billing guidance and reimbursement rates. Akebia also announced its plans to begin a Phase 3 trial for the use of vadadustat in treating anemia in late-stage CKD patients who are not on dialysis. Akebia received feedback from the U.S. Food and Drug Administration (FDA) on its protocol submission for a label expansion study and is incorporating feedback as appropriate. Akebia expects to begin the trial in mid-2025. "We believe Akebia is entering a transformational year with the U.S. market availability of Vafseo, which we believe could become a new standard of care for patients with CKD," said John P. Butler, Chief Executive Officer of Akebia. "During 2025 our commercial organization will remain focused on executing the U.S. launch of Vafseo, while our development team pursues label expansion of Vafseo into the non-dialysis population, which represents a potential multiple billion-dollar market opportunity in the U.S. We believe our commercial products and pipeline have the potential to generate significant shareholder value as Akebia strengthens its leadership position in the treatment of kidney disease and the hypoxia-inducible factor (HIF) space." Vafseo Commercial Update: Vafseo began shipping to dialysis centers and authorized distributors on January 9, 2025. Akebia has secured broad access for Vafseo as the company has now entered into commercial supply agreements with dialysis organizations treating nearly 100% of patients on dialysis in the U.S., including recently signing agreements with a leading dialysis organization and a mid-size dialysis provider. Akebia market research suggests 99% of nephrologists would consider prescribing Vafseo to certain eligible patients, with 75% intending to do so by 6 months after product availability. In December 2024, Akebia announced that U.S. Renal Care enrolled the first patients in the VOICE collaborative clinical trial of Vafseo. U.S. Renal Care has now enrolled more than 650 patients in the trial. Planned Phase 3 Trial of Vadadustat in U.S. Non-Dialysis CKD Patients: In March 2024, the FDA approved Vafseo for the treatment of anemia due to chronic kidney disease in adults who have been receiving dialysis for at least three months. The FDA acknowledged an unmet need for safer and orally available therapies to treat anemia due to CKD in certain non-dialysis patients. Akebia has since engaged with the FDA on potentially expanding vadadustat treatment to this patient population and has received feedback on a protocol submitted for a Phase 3 cardiovascular outcome study of approximately 1,500 U.S. subjects with late-stage CKD anemia not on dialysis, which is expected to begin in mid-2025. Akebia plans to request a Type C meeting with the FDA to continue to discuss the statistical analysis plan and regulatory path. Akebia expects its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including the U.S. Vafseo launch and planned pipeline advancement, for at least two years. Presentation at 43rd J.P. Morgan Healthcare Conference John Butler, Chief Executive Officer, will present on Thursday, January 16th, 2025 at 7:30 a.m. PST. A webcast of the presentation can be accessed through the "Investors" section of Akebia's website for 30 days following the conference. About Akebia Therapeutics Akebia Therapeutics, Inc. is a fully integrated biopharmaceutical company with the purpose to better the lives of people impacted by kidney disease. Akebia was founded in 2007 and is headquartered in Cambridge, Massachusetts. For more information, please visit our website at , which does not form a part of this release. About Vafseo® (vadadustat) tablets Vafseo® (vadadustat) tablets is a once-daily oral hypoxia-inducible factor prolyl hydroxylase inhibitor that activates the physiologic response to hypoxia to stimulate endogenous production of erythropoietin, increasing hemoglobin and red blood cell production to manage anemia. Vafseo is approved for use in 37 countries. INDICATION VAFSEO is indicated for the treatment of anemia due to chronic kidney disease (CKD) in adults who have been receiving dialysis for at least three months. Limitations of Use VAFSEO has not been shown to improve quality of life, fatigue, or patient well-being. VAFSEO is not indicated for use: As a substitute for red blood cell transfusions in patients who require immediate correction of anemia. In patients with anemia due to CKD not on dialysis. IMPORTANT SAFETY INFORMATION about VAFSEO (vadadustat) tablets CONTRAINDICATIONS Known hypersensitivity to VAFSEO or any of its components Uncontrolled hypertension WARNINGS AND PRECAUTIONS Increased Risk of Death, Myocardial Infarction (MI), Stroke, Venous Thromboembolism, and Thrombosis of Vascular Access A rise in hemoglobin (Hb) levels greater than 1 g/dL over 2 weeks can increase these risks. Avoid in patients with a history of MI, cerebrovascular event, or acute coronary syndrome within the 3 months prior to starting VAFSEO. Targeting a Hb level of greater than 11 g/dL is expected to further increase the risk of death and arterial and venous thrombotic events. Use the lowest effective dose to reduce the need for red blood cell (RBC) transfusions. Adhere to dosing and Hb monitoring recommendations to avoid excessive erythropoiesis. Hepatotoxicity Hepatocellular injury attributed to VAFSEO was reported in less than 1% of patients, including one severe case with jaundice. Elevated serum ALT, AST, and bilirubin levels were observed in 1.8%, 1.8%, and 0.3% of CKD patients treated with VAFSEO, respectively. Measure ALT, AST, and bilirubin before treatment and monthly for the first 6 months, then as clinically indicated. Discontinue VAFSEO if ALT or AST is persistently elevated or accompanied by elevated bilirubin. Not recommended in patients with cirrhosis or active, acute liver disease. Hypertension Worsening of hypertension was reported in 14% of VAFSEO and 17% of darbepoetin alfa patients. Serious worsening of hypertension was reported in 2.7% of VAFSEO and 3% of darbepoetin alfa patients. Cases of hypertensive crisis, including hypertensive encephalopathy and seizures, have also been reported in patients receiving VAFSEO. Monitor blood pressure. Adjust anti-hypertensive therapy as needed. Seizures Seizures occurred in 1.6% of VAFSEO and 1.6% of darbepoetin alfa patients. Monitor for new-onset seizures, premonitory symptoms, or change in seizure frequency. Gastrointestinal (GI) Erosion Gastric or esophageal erosions occurred in 6.4% of VAFSEO and 5.3% of darbepoetin alfa patients. Serious GI erosions, including GI bleeding and the need for RBC transfusions, were reported in 3.4% of VAFSEO and 3.3% of darbepoetin alfa patients. Consider this risk in patients at increased risk of GI erosion. Advise patients about signs of erosions and GI bleeding and urge them to seek prompt medical care if present. Serious Adverse Reactions in Patients with Anemia Due to CKD and Not on Dialysis The safety of VAFSEO has not been established for the treatment of anemia due to CKD in adults not on dialysis and its use is not recommended in this setting. In large clinical trials in adults with anemia of CKD who were not on dialysis, an increased risk of mortality, stroke, MI, serious acute kidney injury, serious hepatic injury, and serious GI erosions was observed in patients treated with VAFSEO compared to darbepoetin alfa. Malignancy VAFSEO has not been studied and is not recommended in patients with active malignancies. Malignancies were observed in 2.2% of VAFSEO and 3.0% of darbepoetin alfa patients. No evidence of increased carcinogenicity was observed in animal studies. ADVERSE REACTIONS The most common adverse reactions (occurring at ≥ 10%) were hypertension and diarrhea. DRUG INTERACTIONS Iron supplements and iron-containing phosphate binders: Administer VAFSEO at least 1 hour before products containing iron. Non-iron-containing phosphate binders: Administer VAFSEO at least 1 hour before or 2 hours after non-iron-containing phosphate binders. BCRP substrates: Monitor for signs of substrate adverse reactions and consider dose reduction. Statins: Monitor for statin-related adverse reactions. Limit the daily dose of simvastatin to 20 mg and rosuvastatin to 5 mg. USE IN SPECIFIC POPULATIONS Pregnancy: May cause fetal harm. Lactation: Breastfeeding not recommended until two days after the final dose. Hepatic Impairment : Not recommended in patients with cirrhosis or active, acute liver disease. Please note that this information is not comprehensive. Please click here for the Full Prescribing Information, including BOXED WARNING and Medication Guide. Forward-Looking Statements Statements in this press release regarding Akebia Therapeutics, Inc.'s ("Akebia's") strategy, plans, prospects, expectations, beliefs, intentions and goals are forward-looking statements within the meaning of the U.S. Private Securities Litigation Reform Act of 1995, as amended, and include, but are not limited to, statements regarding: Akebia's plans with respect to its U.S. commercial launch of Vafseo, including Akebia's statements regarding commercial supply contract coverage for Vafseo with dialysis organizations and Akebia's ability to make Vafseo available to nearly 100% of patients on dialysis in the U.S.; Akebia's plans and expectations with respect to potential Vafseo label expansion to treat anemia in late-stage CKD patients who are not on dialysis, including Akebia's expectations as to the timing of a Phase 3 trial, Akebia's statements regarding feedback from the FDA on its protocol submission for a label expansion study and Akebia's plans to request a Type C meeting with the FDA to discuss the statistical plan and regulatory path; Akebia's belief that it is entering a transformational year with U.S. market availability of Vafseo, and Akebia's belief that Vafseo could become a new standard of care for patients with CKD; Akebia's expectations regarding label expansion of Vafseo into the non-dialysis population, including a potential multiple billion-dollar market opportunity in the U.S.; Akebia's belief that its commercial products and pipeline have the potential to generate significant shareholder value as Akebia strengthens its leadership position in the treatment of kidney disease and the HIF space; Akebia's expectations regarding the demand for Vafseo from prescribers; and Akebia's expectations that its existing cash resources and cash from operations will be sufficient to fund its current operating plan, including the U.S. Vafseo launch and planned pipeline advancement, for at least two years. The terms "intend," "believe," "plan," "goal," "potential," "anticipate, "estimate," "expect," "future," "will," "continue," derivatives of these words, and similar references are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results, performance or experience may differ materially from those expressed or implied by any forward-looking statement as a result of various risks, uncertainties and other factors, including, but not limited to, risks associated with: the commercial availability of Vafseo; the potential demand and market potential and acceptance of, as well as coverage and reimbursement related to, Auryxia® and Vafseo, including estimates regarding the potential market opportunity; the competitive landscape for Auryxia and Vafseo, including potential generic entrants; the ability of Akebia to attract and retain qualified personnel; Akebia's ability to implement cost avoidance measures and reduce operating expenses; decisions made by health authorities, such as the FDA, with respect to regulatory filings and other interactions; the potential therapeutic benefits, safety profile, and effectiveness of Vafseo; the results of preclinical and clinical research; manufacturing, supply chain and quality matters and any recalls, write-downs, impairments or other related consequences or potential consequences; and early termination of any of Akebia's collaborations. Other risks and uncertainties include those identified under the heading "Risk Factors" in Akebia's Quarterly Report on Form 10-Q for the quarter ended September 30, 2024, and other filings that Akebia may make with the U.S. Securities and Exchange Commission in the future. These forward-looking statements (except as otherwise noted) speak only as of the date of this press release, and, except as required by law, Akebia does not undertake, and specifically disclaims, any obligation to update any forward-looking statements contained in this press release. Akebia Therapeutics®, Auryxia and Vafseo® are registered trademarks of Akebia Therapeutics, Inc. and its affiliates. Akebia Therapeutics Contact Mercedes Carrasco [email protected] SOURCE Akebia Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

- 2024 net product sales of approximately $336 million exceeds upper end of guidance range; preliminary and unaudited estimate - 2025 expected global net product sales of $420 million to $435 million - VISTAS study of volixibat in primary sclerosing cholangitis expected to complete enrollment in second half 2025; topline data expected 2026 FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today provided its preliminary and unaudited estimates for full-year 2024 net product sales, year-end cash balance, corporate updates, and full-year 2025 outlook. “2024 marked a significant year for Mirum as we accelerated our commercial business and achieved significant development milestones,” said Chris Peetz, chief executive officer of Mirum. “We continued our leadership in cholestatic disease with a label expansion for LIVMARLI and positive interim analyses for volixibat in PSC and PBC. Additionally, we advanced our rare genetic neurology efforts with the NDA submission for chenodiol and the in-licensing of MRM-3379 for Fragile X syndrome. With proven commercial execution in ultra-rare disease and a compelling pipeline in larger indications, we believe we are well-positioned for sustained growth in the years ahead.” Future Expectations and Milestones 2025 Guidance: expect continued revenue growth with global net product sales of approximately $420 million to $435 million and positive cash flow from operations Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in second half 2025; topline data expected in 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 FDA Prescription Drug User Fee Act (PDUFA) date for chenodiol in cerebrotendinous xanthomatosis (CTX) is March 28, 2025 Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025 2024 Highlights Commercial: Continued leadership in rare disease with a franchise in hepatology and genetic neurology 2024 estimated LIVMARLI net product sales of approximately $213 million and CHOLBAM and CHENODAL net product sales of approximately $123 million Total estimated net product sales of approximately $99 million in Q4 2024 including approximately $64 million in LIVMARLI net sales and approximately $35 million in net sales from CHOLBAM and CHENODAL Expanded global footprint; 30 countries with commercial access, including successful reimbursement negotiation and launch in the four major European markets Regulatory and Pipeline: Expanding Mirum’s leadership across multiple rare diseases and larger orphan settings Positive interim results for volixibat in VISTAS PSC and VANTAGE PBC studies Volixibat granted breakthrough therapy designation for treatment of cholestatic pruritus in PBC by the U.S. Food and Drug Administration (FDA) Volixibat granted orphan drug designation for the treatment of cholestatic pruritus in PBC by the FDA LIVMARLI approved by the FDA for cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) patients 12 months and older LIVMARLI approved in Europe for treatment of PFIC in patients three months and older Initiated the LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions New Drug Application (NDA) submitted for chenodiol in CTX In-licensed global rights to PDE4D inhibitor MRM-3379 for FXS Corporate and Financial: Sustained financial strength Achieved positive cash flow from operations in Q3 2024 Cash, cash equivalents and investments of approximately $287 million as of December 31, 2024 compared to $286.3 million as of December 31, 2023 Announced the appointment of Joanne Quan, MD as Chief Medical Officer The foregoing amounts relating to 2024 financial data are unaudited and preliminary and are subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of December 31, 2024. Mirum will present at the 43rd annual J.P. Morgan Healthcare Conference in San Francisco on Wednesday, January 15, 2025, at 9:45 a.m. PT. The presentation and question and answer session will be webcast live and can be accessed by visiting the Investors and Media section of Mirum’s corporate website. The replay of the webcast will be available for 30 days. About LIVMARLI® (maralixibat) oral solution 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.com. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and 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 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. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (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. 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 Cerebrotendinous Xanthomatosis Cerebrotendinous xanthomatosis (CTX) is a rare, progressive and underdiagnosed disorder of cholesterol metabolism affecting many parts of the body. In people with CTX, the body is unable to break down cholesterol properly causing toxins (e.g., cholestanol and bile alcohols) to build up throughout the body over time. The disorder is inherited in an autosomal recessive genetic manner. Signs and symptoms of CTX include neonatal cholestasis (jaundice or bile flow interruption), chronic diarrhea, the development of bilateral cataracts before the age of 18, development of tendon xanthomas (fatty deposits in the tendons) during teenage years or later, and neurologic deterioration. The types, combinations and severity of symptoms can be different from person to person making diagnosis challenging and often delayed. About chenodiol tablets Chenodiol tablets is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. More recently, the US Food and Drug Administration (FDA) granted chenodiol orphan drug designation for cerebrotendinous xanthomatosis (CTX). CTX is a rare progressive disorder that can affect the brain, spinal cord, tendons, eyes and arteries. Chenodiol is not yet indicated for the treatment of CTX but has received a medical necessity determination in the U.S. by the FDA. 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 , 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 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating 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 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 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. Chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the U.S. Lastly, Mirum is 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, commercial results for our approved products, including continued growth in year over year net product sales, being on track to achieve our revised financial guidance, our expected financial results as of December 31, 2024, including our net product sales and cash, cash equivalents and investments, delivering life changing medicines for patients suffering from rare diseases, the results, enrollment, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, including newly in-licensed product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for its product candidates globally, including the anticipated PDUFA date for chenodiol for CTX and additional international launches expected in 2025. 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” 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, 2024 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. Investor Contacts: Andrew McKibben ir@mirumpharma.com Media Contact: Erin Murphy media@mirumpharma.com