Mirum Pharmaceuticals Inc.

MIRM

Held by 8 specialist biotech funds

High Convergence

8 funds opened new positions. Next phase2 readout (LUM001): Feb 2015. Short interest: 13.8% of float.

BiotechnologyCEO: Christopher PeetzIPO: 2019Website ↗

Mirum Pharmaceuticals, Inc. is a biopharmaceutical firm dedicated to discovering and commercializing groundbreaking therapies for serious rare and orphan conditions. Its lead experimental drug, LIVMARLI, an oral treatment, is currently undergoing trials for progressive familial intrahepatic cholestasis (PFIC), Alagille syndrome, and biliary atresia. Furthermore, the company is advancing Volixibat to address intrahepatic cholestasis of pregnancy and primary sclerosing cholangitis. Founded in 2018, Mirum Pharmaceuticals, Inc. operates from its headquarters in Foster City, California.

Catalysts

Phase 3Q4 2009Cholic AcidsInfantile Refsum's Disease, Zellweger Syndrome, AdrenoleukodystrophyNCT00007020

Primary: Number of Participants With Excretion of Atypical Bile Acids in Urine by Category (Baseline, then every 1, 3, or 6 months (depending on protocol version) for an average of 2.8 years)Enrollment:85/85Sites: 1NA, TREATMENT

Phase 3PositiveQ3 2010Cholic acidInborn Errors of Bile Acid SynthesisNCT01115582

Phase 2PositiveQ1 2015LUM001Alagille SyndromeNCT01903460

Primary: Change From Baseline to Week 13 (End of Treatment) in Fasting Serum Bile Acid Level (Baseline to 13 weeks or end of treatment)Enrollment:20/20Sites: 3RANDOMIZED, TRIPLE, TREATMENT

Phase 2Q2 2015LUM001PBC, Primary Biliary CirrhosisNCT01904058

Primary: Change From Baseline in Pruritus Using Adult Itch Reported Outcome (ItchRO) Weekly Sum Score at Week 13/ Early Termination (ET) (Baseline and Week 13/ET)Enrollment:66/66Sites: 24RANDOMIZED, DOUBLE, TREATMENT

Phase 2PositiveQ1 2016LUM001Primary Sclerosing Cholangitis (PSC)NCT02061540

Primary: Number of Participants With Treatment-Emergent Adverse Events (TEAEs) (From start of study drug administration until Week 18)Enrollment:27/27Sites: 9NA, TREATMENT

Phase 3PositiveQ3 2016Cholic AcidBile Acid Synthesis DefectNCT01438411

Primary: Change in Atypical Urinary Bile Acid Excretion by FAB-MS (Fast-Atom-Bombardment Ionization-Mass Spectrometry) (At baseline, then every 12 months for an average of 3.5 years)Enrollment:53/53Sites: 1NA, TREATMENT

Phase 2PositiveQ2 2020LUM001 (Maralixibat)Progressive Familial Intrahepatic Cholestasis (PFIC)NCT02057718

Primary: Change From Baseline to Endpoint (Week 13) in Fasting sBA Level (Baseline (Day 0) to Week 13)Enrollment:33/33Sites: 10NA, TREATMENT

Phase 2PositiveQ2 2020LUM001 (Maralixibat)Alagille SyndromeNCT02160782

Primary: Change From Week 18 to Week 22 in Fasting sBA Levels in Participants Who Had a Reduction in sBA ≥50% From Baseline to Week 12 or Week 18 (Week 18 to Week 22)Enrollment:31/31Sites: 9RANDOMIZED, TRIPLE, TREATMENT

Phase 2PositiveQ2 2020LUM001 (Maralixibat)Alagille SyndromeNCT02117713

Phase 2PositiveQ2 2020LUM001 (Maralixibat)Alagille SyndromeNCT02047318

Phase 2NegativeQ4 2022VolixibatIntrahepatic Cholestasis of PregnancyNCT04718961

Primary: Assess the Safety and Tolerability of Volixibat in Participants With ICP (Through to end of treatment, up to 21 weeks)Enrollment:4/4Sites: 20RANDOMIZED, DOUBLE, TREATMENT

Phase 3Q3 2023Blinded CDCA 250 mg TIDCTXNCT04270682

Phase 2Q3 2024MaralixibatCholestatic Liver DiseaseNCT04168385

Primary: Incidence of Treatment-Emergent Adverse Events (From informed consent through approximately 4.5 years, including 30 days after last dose.)Enrollment:52/52Sites: 17NA, TREATMENT

Phase 2Q2 2026VolixibatPrimary Sclerosing CholangitisNCT04663308

Primary: Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire (Baseline through to Week 28)Enrollment:182/182Sites: 103RANDOMIZED, DOUBLE, TREATMENT

Phase 3Q3 2026Brelovitug 300 mgChronic Hepatitis D InfectionNCT07298330

Primary: Percentage of participants with a composite endpoint of virologic response and ALT normalization at Week 24 in brelovitug arms compared to response at Week 12 of delayed-treatment arm (Week 24)Enrollment:Target 80Sites: 27RANDOMIZED, TREATMENT

Phase 3Q4 2026MaralixibatCholestatic Liver Disease (Except ALGS, PFIC, PBC and PSC)NCT06553768

Primary: Mean change in the ItchRO(Obs) severity score (From baseline to average of week 13 to week 20)Enrollment:Target 90Sites: 23RANDOMIZED, QUADRUPLE, TREATMENT

Phase 2Q1 2027VolixibatPrimary Biliary Cholangitis, PBCNCT05050136

Primary: Mean change in the daily itch scores using the Adult Itch Reported Outcome (Adult ItchRO) questionnaire (Baseline to week 28)Enrollment:333/333Sites: 130RANDOMIZED, DOUBLE, TREATMENT

Phase 3Q2 2027Brelovitug 300 mgChronic Hepatitis D InfectionNCT07200908

Phase 3Q3 2027Brelovitug (BJT-778)Chronic Hepatitis DNCT07454837

Phase 2Q4 2027Low dose of MRM-3379Fragile X SyndromeNCT07209462

Phase 2Mar 31, 2028MaralixibatIntrahepatic Cholestasis of PregnancyNCT07389031

Phase 2Q1 2029MaralixibatIntrahepatic Cholestasis of PregnancyNCT07823127

Phase 3Q2 2029Brelovitug 300 mgChronic Hepatitis D InfectionNCT06907290

Mirum Pharmaceuticals Inc.

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