Peroxisomal Disorders

GAITHERSBURG, Md., May 01, 2025 (GLOBE NEWSWIRE) -- Altimmune, Inc. (Nasdaq: ALT), a late clinical-stage biopharmaceutical company developing next-generation peptide-based therapeutics for liver and cardiometabolic diseases, today announced that the Company will present analyses of pemvidutide-treated subjects with metabolic dysfunction-associated steatotic liver disease (MASLD) utilizing the MASH Resolution Index (MASHResInd; Loomba 2024), a highly sensitive non-invasive measure of MASH histologic response, in a poster session at the European Association for the Study of the Liver (EASL) International Liver Congress™ 2025 in Amsterdam, The Netherlands. The abstract will be featured in the conference poster session and poster tour. Details of the presentations are as follows: Session: MASLD: Therapy, Track Hub 7 – Metabolism, Alcohol, and Toxicity (Poster# SAT-453) Date/Time: Thursday, May 8, 2025, at 9:45 am CET Session: MASLD Therapy Section (Poster# SAT-453) Date/Time: Saturday, May 10, 2025, from 12:00 PM to 1:00 PM CET A copy of the poster will be accessible on the Events section of the Altimmune website. About Pemvidutide Pemvidutide is a novel, investigational, peptide-based 1:1 GLP-1/glucagon dual receptor agonist in development for the treatment of obesity, MASH, Alcohol Use Disorder (AUD) and Alcohol Liver Disease (ALD). Activation of the GLP-1 and glucagon receptors is believed to mimic the complementary effects of diet and exercise on weight loss, with GLP-1 suppressing appetite and glucagon increasing energy expenditure. Glucagon is also recognized as having direct effects on hepatic fat metabolism, which is believed to lead to rapid reductions in levels of liver fat and serum lipids. In clinical trials to date, once-weekly pemvidutide has demonstrated compelling weight loss with class-leading lean mass preservation, and robust reductions in triglycerides, LDL cholesterol, liver fat content and blood pressure. The U.S. FDA has granted Fast Track designation to pemvidutide for the treatment of MASH. Pemvidutide recently completed the MOMENTUM Phase 2 obesity trial and is being studied in the ongoing IMPACT Phase 2b MASH trial. IND applications in AUD and ALD have received FDA clearance with Phase 2 trials to commence in mid-2025. About Altimmune Altimmune is a late clinical-stage biopharmaceutical company focused on developing innovative next-generation peptide-based therapeutics. The Company is developing pemvidutide, a GLP-1/glucagon dual receptor agonist for the treatment of obesity, MASH, alcohol use disorder as well as alcohol related liver disease. For more information, please visit www.altimmune.com. Follow @Altimmune, Inc. on LinkedIn Follow @AltimmuneInc on Twitter Company Contact: Greg Weaver Chief Financial Officer Phone: 240-654-1450 ir@altimmune.com Investor Contact: Lee Roth Burns McClellan Phone: 646-382-3403 lroth@burnsmc.com Media Contact: Jason Spark Inizio Evoke, Biotech Phone: 619-849-6005 Jason.spark@canalecomm.com This press release was published by a CLEAR® Verified individual. Source: Altimmune, Inc

- 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

bluebird has been under financial pressure for several years. Credit: SOPA Images via Getty Images. Bluebird bio (bluebird) has reaffirmed its support for a proposed acquisition by Carlyle and SK Capital after rival bidder Ayrmid failed to deliver a binding offer or secure financing by the extended deadline. The announcement marks the second time Ayrmid has initiated discussions with bluebird without producing a fully financed proposal. Bluebird said it had engaged with Ayrmid for three weeks following the private equity firm’s unsolicited bid in March, including extending the original deadline to allow more time for due diligence and deal structuring. However, Ayrmid did not submit a definitive proposal by the agreed-upon 15 April cut-off date and has yet to secure the necessary capital to proceed. As a result, bluebird’s board of directors is reiterating its unanimous support for the acquisition agreement with Carlyle and SK Capital, which was first announced in February 2025. The board is urging shareholders to tender their shares by the updated deadline of 2 May, extending the previous expiration date of 18 April. bluebird’s board chairman Mark Vachon said: “Ayrmid’s proposal remains highly conditional. bluebird has engaged with Ayrmid on two separate occasions—neither of which has resulted in a binding or fully-financed offer. The board unanimously reaffirms its support of the previously announced agreement with Carlyle and SK Capital in the strongest possible terms.” Bluebird has been under significant financial pressure for several years. The company currently faces the risk of defaulting on a $175m loan from Hercules Capital, secured in 2024 to support ongoing operations. Without a new capital injection, the board said, the company’s liquidity position remains precarious. Carlyle and SK Capital’s proposal offers $3 per share in cash, along with a potential additional payment of $6.84 per share if certain clinical and commercial milestones are met. Ayrmid‘s offer, disclosed last month, proposed $4.50 per share upfront and the same milestone-based payout structure. However, bluebird confirmed that Ayrmid has not demonstrated access to committed funding. bluebird noted that Ayrmid is continuing to seek financing and may provide further updates in the coming days. bluebird’s financial position has deteriorated over time, despite achieving several key regulatory milestones. The company currently markets three US Food and Drug Administration (FDA)- approved gene therapies – Zynteglo (betibeglogene autotemcel) for transfusion-dependent beta-thalassemia, Skysona (elivaldogene autotemcel) for cerebral adrenoleukodystrophy, and Lyfgenia (lovotibeglogene autotemcel) for sickle cell disease – but has struggled with slow uptake, high costs, and reimbursement hurdles. In 2023, the company suffered a major setback when the FDA denied a priority review voucher (PRV) for Lyfgenia. Bluebird had already arranged to sell the voucher to Novartis for $103m, but the agency ruled that the therapy’s active ingredient had previously been used in Zynteglo, for which the company had already received a PRV in 2022. The loss of the expected funds deepened bluebird’s financial strain. To fill the gap, bluebird pursued emergency financing through a $150m public stock offering underwritten by Goldman Sachs and JP Morgan, as well as up to $100m in receivables financing from Alterna Capital Solutions. The company has also undergone repeated cost-cutting efforts, including workforce reductions of 30% in 2022 and an additional 25% last year. Despite these efforts, bluebird’s therapies continue to face stiff competition. Lyfgenia is priced at $3.1m per patient, while a competing treatment, Casgevy (exagamglogene autotemcel) from Vertex Pharmaceuticals and CRISPR Therapeutics, is listed at $2.2m. Both therapies were approved on the same day in December 2023 to treat sickle cell disease. Going public in 2013, bluebird has seen its stock decline sharply in recent years. Following the 16 April update, shares closed down by 7%. Cell & Gene Therapy coverage on Pharmaceutical Technology is supported by Cytiva . Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content. Free Whitepaper Optimise your cell therapy process: a guide to cell thawing Typically carried out at the point of care, errors in cell therapy thawing could compromise treatment efficacy, leading to significant patient impact as well as high costs and a compromised reputation for the product’s developer. This guide addresses how cell thawing has historically developed into the new techniques used today, along with the physical and biological implications of key metrics and components such as warming rate and ice structure. Also included are reviews of key studies from scientific literature and a consideration of the interactions between cooling and warming rates, as applicable to cell and gene therapies. Thank you. You will receive an email shortly. Please check your inbox to download the Whitepaper. By Cytiva Thematic You have a right to withdraw your consent at any time, by clicking here . We may still continue to send you service-related and other non-promotional communications. For more information relating to our privacy practices, we invite you to review our privacy policy .