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Genotype And Dose-Dependent Response To An Asbti In Patients With Bile Salt Export Pump Deficiency - EP3923943

The patent EP3923943 was granted to Mirum Pharmaceuticals on Jul 31, 2024. The application was originally filed on Feb 12, 2020 under application number EP20755296A. The patent is currently recorded with a legal status of "Granted And Under Opposition".

EP3923943

MIRUM PHARMACEUTICALS
Application Number
EP20755296A
Filing Date
Feb 12, 2020
Status
Granted And Under Opposition
Jun 27, 2024
Publication Date
Jul 31, 2024
External Links
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Patent Oppositions (2)

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GENERICS UKApr 30, 2025RUSSELLADMISSIBLE
SANDOZApr 30, 2025MAIWALDADMISSIBLE

Patent Citations (4) New

Patent citations refer to prior patents cited during different phases such as opposition or international search.

Citation PhasePublication Number
INTERNATIONAL-SEARCH-REPORTUS2014243281
INTERNATIONAL-SEARCH-REPORTUS9040518
INTERNATIONAL-SEARCH-REPORTWO2017167935
INTERNATIONAL-SEARCH-REPORTWO2018002827

Non-Patent Literature (NPL) Citations (45) New

NPL citations refer to non-patent references such as research papers, articles, or other publications cited during examination or opposition phases.

Citation PhaseReference Text
EXAMINATION- THOMPSON RICHARD, "Oral Abstracts (Abstracts 1-263)", HEPATOLOGY, US, vol. 66, no. S1, doi:10.1002/hep.29500, ISSN 0270-9139, (20171001), pages 1 - 148, URL: https://api.wiley.com/onlinelibrary/tdm/v1/articles/10.1002%2Fhep.29500, XP093090080
INTERNATIONAL-SEARCH-REPORT- "PubChem COMPOUND SUMMARY CID9939892 | C36H45N3O7S2", PubChem COMPOUND, (20061025), Database accession no. CID9939892, URL: NCBI, XP055731571 [A] 6, 59 * pages 1-24. pg 2, structure *
INTERNATIONAL-SEARCH-REPORT- Anne Davit-Spraul, Monique Fabre, Sophie Branchereau, Christiane Baussan, Emmanuel Gonzales, Bruno Stieger, Olivier Bernard, Emmanuel Jacquemin, "ATP8B1 and ABCB11 Analysis in 62 Children with Normal Gamma- Glutamyl Transferase Progressive Familial Intrahepatic Cholestasis (PFIC): Phenotypic Differences Between PFIC1 and PFIC2 and Natural History", HEPATOLOGY, (20100000), vol. 51, no. 5, doi:10.1002/hep.23539, pages 1645 - 1655, XP055731568 [Y] 54-63, 69-79 * . abstract; pg 1652, col 2, para 1 *
OPPOSITION- Anonmyous, "A Study to Evaluate the Efficacy and Safety of Maralixibat in Subjects With Progressive Familial lntrahepatic Cholestasis (MARCH-PFIC) (MARCH-PFIC)", Clinicat Trials - NCT03905330, (20231211), pages 1 - 13, XP093276292
OPPOSITION- Anonymous, "ANNEX I SUMMARY OF PRODUCT CHARACTERISTICS Livmarli", European Medicines Agency, (20240701), pages 1 - 38, European Medicines Agency, URL: https://www.ema.europa.eu/en/documents/product-information/livmarli-epar-product-information_en.pdf, XP093310109
OPPOSITION- Anonymous, "Final Results of Albireo’s Phase 2 Study ofA4250 in Children with Cholestatic Liver Disease Presented at The Liver Meeting® 2017", GlobeNewswire, (20171020), pages 1 - 3, GlobeNewswire , URL: https://www.globenewswire.com/news-release/2017/10/20/1150901/0/en/Final-Results-of-Albireo-s-Phase-2-Study-of-A4250-in-Children-with-Cholestatic-Liver-Disease-Presented-at-The-Liver-Meeting-2017.html, XP093276237
OPPOSITION- Anonymous, "How ASBT Inhibition Works", Mirum Pharma, (20191210), pages 1 - 16, Mirum Pharma, URL: https://web.archive.org/web/20191210065339/https://mirumpharma.com/programs/, XP093276241
OPPOSITION- Anonymous, "Mirum Pharmaceuticals Initiates Phase 3 Clinical Trial for Pediatric Patients with Progressive Familial lntrahepatic Cholestasis", Mirum, (20190709), pages 1 - 1, XP093276294
OPPOSITION- Anonymous, "Mi rum Pharmaceuticals Presents Data Demonstrating Long-term Durability of Treatment Effect of Maralixibat in Children With Cholestatic Liver Diseases", Mirum, (20191108), pages 1 - 3, XP093276277
OPPOSITION- Anonymous, "Mirum Pharmaceuticals Presents New Data Demonstrating Durable Improvements in Clinical Outcome Measures in Patients with PFIC2 and Alagille Syndrome Treated with Maralixibat", Mirum, (20190415), pages 1 - 3, Mirum, URL: https://www.prnewswire.com/news-releases/mirum-pharmaceuticals-presents-new-data-demonstrating-durable-improvements-in-clinical-outcome-measures-in-patients-with-pfic2-and-alagille-syndrome-treated-with-maralixibat-300831721.html, XP093276255
OPPOSITION- Anonymous, "Mirum Pharmaceuticals Presents New Data Demonstrating Durable Improvements in Clinical Outcome Measures in Patients with PFIC2 and Alagille Syndrome Treated with Maralixibat", PR Newswire, (20190415), pages 1 - 6, PR Newswire, URL: https://www.prnewswire.com/news-releases/mirum-pharmaceuticals-presents-new-data-demonstrating-durable-improvements-in-clinical-outcome-measures-in-patients-with-pfic2-and-alagille-syndrome-treated-with-maralixibat-300831721.html, XP093275919
OPPOSITION- Anonymous, "Mirum Pharmaceuticals to Present New Data at The Liver Meeting 2019", Mirum, (20191001), pages 1 - 2, XP093276274
OPPOSITION- Anonymous, "Mirum Pharmaceuticals to Present Phase 2 Clinical Results of Maralixibat in Alagille Syndrome and Progressive Familial lntrahepatic Cholestasis at the International Liver Congress", Mirum, (20190327), pages 1 - 1, Mirum, URL: https://www.prnewswire.com/news-releases/mirum-pharmaceuticals-to-present-phase-2-clinical-results-of-maralixibat-in-alagille-syndrome-and-progressive-familial-intrahepatic-cholestasis-at-the-international-liver-congress-300819248.html, XP093276243
OPPOSITION- Anonymous, "Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 (Maralixibat) in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis (INDIGO)", Clinical Trials, (20231023), pages 1 - 23, XP093276295
OPPOSITION- Anonymous, "Public summary of opinion on orphan designation", European Medicines Agency, (20140121), pages 1 - 6, XP093275954
OPPOSITION- D24 - Wayback Machine search results for ‘https://mirumpharma.com/wpcontent/uploads/2019/07/INDIGO501_EASL2019_oral_presentation_final.pdf’ which is the URL of D5 cited by the opponents
OPPOSITION- D25 - Wayback Machine search results for ‘https://mirumpharma.com/wpcontent/uploads/2019/11/AASLD_-501-Genetics-FINAL.pdf’ which is the URL of D16b cited by O2
OPPOSITION- D26 - Wayback Machine search results for ‘https://mirumpharma.com/wpcontent/uploads/2019/07/AASLD-2017-INDIGO-presentation.pdf’ which is the URL of D20b cited by O2.
OPPOSITION- D2-The International Liver Congress 2019 Abstract Book
OPPOSITION- D4- Publications & Presentations Mirum Pharma, Obtained 16/04/2025
OPPOSITION- Gonzalez-Peralta Regino P., Miethke Alexander, Aqul Amal A., Mogul Douglas B., Nunes Tiago, Garner Will, Vig Pamela, Thompson Richard J., "Maralixibat improves growth in patients with progressive familial intrahepatic cholestasis: data from the MARCH/MARCH-ON trials", (20240608), URL: https://mirumpharma.com/wp-content/uploads/2024/06/Thompson-RJ-EASL-2024-MARCH-ON-Growth.pdf, XP093310692
OPPOSITION- Thompson Richard, Jaecklin Thomas, Peetz Chris, Vig Pamela, "Genotype and dose-dependent response to maralixibat in patients with bile salt export pump deficiency", Mirum Pharma, (20191110), pages 1 - 17, Mirum Pharma, URL: https://mirumpharma.com/wp-content/uploads/2021/06/AASLD_-501-Genetics-FINAL.pdf, XP093276270
OPPOSITION- Thompson Richard J, Kelly Deirdre A, Mcclean Patricia, Miethke Alexander G, Soufi Nisreen, Rivet8 Christine, Jankowska Irena, Mack Cara L, Karthikeyan Palaniswamy, Gu Joan, Jaecklin Thomas, Squires Robert H, Leen Kath, Loomes M, Rivet Christine, Loomes Kathleen M, "Phase 2 open-label efficacy and safety study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in chil dren with progressive familial intrahepatic cholestasis: 48-week interim efficacy analysis", Hepatology, (20190701), pages 57A - 57A, XP093276291
OPPOSITION- Thompson Richard J, Kelly Deirdre A, Mcclean Patricia, Miethke Alexander G, Soufi Nisreen, Rivet Christine, Jankowska Irena, Mack Cara L, Karthikeyan Palaniswamy, Gu Joan, Jaecklin Thomas, Squires Robert H, Loomes Kathleen M, "Phase 2 open-label efficacy and safety study of the apical sodium-dependent bile acid transporter inhibitor maralixibat in children with progressive familial intrahepatic cholestasis: 48-week interim efficacy analysis", AASLD - American association for the study of liver diseases, (20171001), pages 1 - 18, AASLD - American association for the study of liver diseases, URL: https://mirumpharma.com/wp-content/uploads/2021/06/AASLD-2017-INDIGO-presentation.pdf, XP093276284
OPPOSITION- Thompson Richard J, Thomas Jaecklin , Chris Peetz , Pamela Vig, "Genotype and dose-dependent response to maralixibat in patients with bile salt export pump deficiency ", Hepatology, (20191001), pages 56A - 56A, XP093276267
OPPOSITION- Thompson Richard, Kelly Deirdre, Rajwal Sanjay, Miethke Alexander, Soufi Nisreen, Rivet Christine, Jankowska Irena, Mack Cara, Jaecklin Thomas, Squires Robert H, Loomes Kathleen M, "Growth analysis in children with PFIC treated with the ASBT inhibitor maralixibat", The International Liver Congress 2019, (20190101), pages 1 - 16, XP093275942
OPPOSITION- Malatack Jeffrey, Daniel Doyle, "A Drug Regimen for Progressive Familial Cholestasis Type 2", Pediatrics, (20180101), doi:10.1542/peds.2016-3877, pages 1 - 5, XP055975631
OPPOSITION- Shirley Matt, "Maralixibat: First Approval", Drugs, Auckland, (20220101), vol. 82, no. 1, doi:10.1007/s40265-021-01649-0, ISSN 0012-6667, pages 71 - 76, XP093276280
OPPOSITION- Kubitz Ralf, Dröge Carola, Stindt Jan, Weissenberger Katrin, Häussinger Dieter, "The bile salt export pump (BSEP) in health and disease", Clinics and Research in Hepatology and Gastroenterology, FR , (20121201), vol. 36, no. 6, doi:10.1016/j.clinre.2012.06.006, ISSN 2210-7401, pages 536 - 553, XP093275950
OPPOSITION- Baker Alastair, Kerkar Nanda, Todorova Lora, Kamath Binita M., Houwen Roderick H.J., "Systematic review of progressive familial intrahepatic cholestasis", Clinics and Research in Hepatology and Gastroenterology, FR , (20190201), vol. 43, no. 1, doi:10.1016/j.clinre.2018.07.010, ISSN 2210-7401, pages 20 - 36, XP093275951
OPPOSITION- Thompson Rischard, "Growth Analysis in Children with Progressive Familial Intrahepatic Cholestasis Treated with the Apical Sodiumdependent Bile Acid Transporter Inhibitor Maralixibat", Journal of Hepatology, (20190101), vol. 70, no. 1, pages e131 - e132, XP093264272
OPPOSITION- Miethke Alexander G, Moukarzel Adib, Porta Gilda, Covarrubias Esquer Joshue, Czubkowski Piotr, Ordonez Felipe, Mosca Antonella, Aqul Amal A, Squires Robert H, Sokal Etienne, D'agostino Daniel, Baumann Ulrich, D'antiga Lorenzo, Kasi Nagraj, Laborde Nolwenn, Arikan Cigdem, Lin Chuan-Hao, Gilmour Susan, Mittal Naveen, Chiou Fang Kuan, Horslen Simon P, Huber Wolf-Dietrich, Jaecklin Thomas, Nunes Tiago, Lascau Anamaria, Longpre Lara, Mogul Douglas B, Garner Will, Vig Pamela, Hupertz Vera F, Gonzalez-Peralta Regino P, Ekong Udeme, Hartley Jane, Laverdure Noemie, Ovchinsky Nadia, Thompson Richard J, "Maralixibat in progressive familial intrahepatic cholestasis (MARCH-PFIC): a multicentre, randomised, double-blind, placebo-controlled, phase 3 trial", The lancet. Gastroenterology & hepatology, Elsevier Ltd, Netherlands, Netherlands, (20240701), vol. 9, no. 7, doi:10.1016/S2468-1253(24)00080-3, ISSN 2468-1253, pages 620 - 631, XP093310698
OPPOSITION- Evason Kimberley , Bove Kevin E. , Finegold Milton J. Knisely, A.S. Rhee, Sue , Rosenthal, Philip , Miethke, Alexander G. , Karpen, Saul J. , Ferrell, Linda D. , Kim, Grace E, "Evason, Morphologic findings in progressive familial intrahepatic cholestasis 2 (PFIC2): correlation with genetic and immunohistochemical studies", Am J Surg Pathol, (20110501), vol. 35, no. 5, doi:10.1097/PAS.0b013e318212ec87, pages 687 - 696, XP009561088
SEARCH- BAUMANN U, "Effects of the ileal bile acid transport inhibitor A4250 on pruritus and serum bile acids in patients with biliary atresia: phase 2 study results", EMBASE, ELSEVIER SCIENCE PUBLISHERS, AMSTERDAM, NL, (20190501), Database accession no. EMB-628093576, XP002807947 [XP] 1,4,5,11,14,16 * the whole document * [Y] 1-16
SEARCH[ ] - BAUMANN U, "Effects of the ileal bile acid transport inhibitor A4250 on pruritus and serum bile acids in patients with biliary atresia: phase 2 study results", JOURNAL OF PEDIATRIC GASTROENTEROLOGY AND NUTRITION 20190501 LIPPINCOTT WILLIAMS AND WILKINS NLD, (20190501), vol. 68, no. Supplement 1, ISSN 1536-4801, pages 767 20190605 to 20190608 Glasgow - 768 CONF
SEARCH- Clinical Trials.Gov, "Study Record Versions Contacts/Locations and Study Status History of Changes for Study: NCT03566238 This Study Will Investigate the Efficacy and Safety of A4250 in Children With PFIC 1 or 2 (PEDFIC 1)", clinical trials .gov, (20190208), pages 1 - 16, URL: https://clinicaltrials.gov/ct2/history/NCT03566238?V_18=View#StudyPageTop, (20221028), XP055975866 [Y] 1-16 * pages 5-7, 9 *
SEARCH- Clinical Trials.Gov, "Study Record Versions History of Changes for Study: NCT02057718 Open Label Study to Evaluate Efficacy and Long Term Safety of LUM001 in the Treatment of Cholestatic Liver Disease in Patients With Progressive Familial Intrahepatic Cholestasis (INDIGO)", (20170905), pages 1 - 13, URL: https://clinicaltrials.gov/ct2/history/NCT02057718?V_16=View#StudyPageTop, (20221028), XP055975938 [Y] 1-16 * page 5 * * page 9 *
SEARCH- Clinical Trials.Gov, "Study Record Versions History of Changes for Study: NCT03659916 Long Term Safety & Efficacy Study Evaluating The Effect of A4250 in Children With PFIC Types 1 & 2", clinical trials.gov, (20190211), pages 1 - 11, URL: https://clinicaltrials.gov/ct2/history/NCT03659916?V_5=View#StudyPageTop, (20221028), XP055975930 [Y] 1-16 * page 5 - page 8 *
SEARCH- Eudract 2015-001157-32, "Clinical Trial Results: An exploratory Phase II Study to demonstrate the Safety and Efficacy of A4250 in Children with Cholestatic Pruritus Summary", EU clinical Trials register, EU Clinical Trials Register, (20171014), pages 1 - 19, URL: https://www.clinicaltrialsregister.eu/ctr-search/trial/2015-001157-32/results, (20221028), XP055975770 [X] 1,4-6,10,11,16 * page 4 * * page 9 - page 10 * * page 11; table bottom * * pages 12-13 * [Y] 1-16
SEARCH- Malatack Jeffrey ET AL, "Case RepoRt A Drug Regimen for Progressive Familial Cholestasis Type 2", PEDIATRICS, doi:10.1542/peds.2016-3877, (20180101), pages 1 - 5, URL: https://publications.aap.org/pediatrics/article/141/1/e20163877/37702/A-Drug-Regimen-for-Progressive-Familial, (20221027), XP055975631 [X] 1,2,4-7,10,16 * the whole document * [Y] 1-16
SEARCH- THOMPSON RICHARD, "Oral Abstracts (Abstracts 1-288)", HEPATOLOGY, US, vol. 70, no. S1, doi:10.1002/hep.30940, ISSN 0270-9139, (20191001), pages 1 - 1, URL: https://onlinelibrary.wiley.com/doi/full-xml/10.1002/hep.30940, (20221031), XP055977389 [XP] 1,2,4-7,9-11,14-16 * the whole document * [Y] 1-16
SEARCH- HEGADE VINOD S ET AL, "Effect of ileal bile acid transporter inhibitor GSK2330672 on pruritus in primary biliary cholangitis: a double-blind, randomised, placebo-controlled, crossover, phase 2a study", THE LANCET, ELSEVIER, AMSTERDAM, NL, (20170208), vol. 389, no. 10074, doi:10.1016/S0140-6736(17)30319-7, ISSN 0140-6736, pages 1114 - 1123, XP029946669 [A] 1,4-16 * the whole document *
SEARCH- STRAUTNIEKS S S ET AL, "Severe Bile Salt Export Pump Deficiency: 82 Different ABCB11 Mutations in 109 Families", GASTROENTEROLOGY, ELSEVIER INC, US, vol. 134, no. 4, doi:10.1053/J.GASTRO.2008.01.038, ISSN 0016-5085, (20080401), pages 1203 - 1214.e8, (20080118), XP022584853 [A] 1-16 * the whole document *
SEARCH- MELISSA PALMER ET AL, "A randomised, double-blind, placebo-controlled phase 1 study of the safety, tolerability and pharmacodynamics of volixibat in overweight and obese but otherwise healthy adults: implications for treatment of non-alcoholic steatohepatitis", BMC PHARMACOLOGY AND TOXICOLOGY, BIOMED CENTRAL LTD, LONDON, UK, (20180316), vol. 19, no. 1, doi:10.1186/S40360-018-0200-Y, pages 1 - 13, XP021254505 [Y] 3 * the whole document *
SEARCH- CHEN HUEY-LING ET AL, "Expression of Hepatocyte Transporters and Nuclear Receptors in Children With Early and Late-Stage Biliary Atresia", PEDIATRIC RESEARCH, US, (20080601), vol. 63, no. 6, doi:10.1203/PDR.0b013e318170a6b5, ISSN 0031-3998, pages 667 - 673, XP055978248 [A] 1-16 * page 668, column right, paragraph 7; figure 1 *

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