Mirum Pharmaceuticals Unveils New Liver Disease Treatment Data
Written by Emily J. Thompson, Senior Investment Analyst
Updated: May 30 2026
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Source: Newsfilter
- VISTAS Study Results: In patients with primary sclerosing cholangitis (PSC), treatment with volixibat led to a significant average reduction of 2.72 points in pruritus scores, with 56% of patients achieving a ≥2 point reduction, indicating the drug's potential to enhance patient quality of life.
- AZURE-1 Study Findings: In chronic hepatitis delta virus (HDV) patients, brelovitug demonstrated a 100% virologic response rate at 24 weeks, with ALT normalization rates of 45% and 40%, showcasing its efficacy and safety in advanced disease patients, potentially offering new treatment options for HDV.
- Improved EFS with LIVMARLI: PFIC patients treated with LIVMARLI (maralixibat) showed a significant improvement in event-free survival (EFS) compared to a control cohort, with an HR of 0.29 (p=0.0001), suggesting the drug's potential in long-term PFIC management and improved patient outcomes.
- Future Plans: Mirum is set to submit an NDA for volixibat to the FDA in the second half of 2026 and anticipates the commercial launch of brelovitug in 2027, reflecting the company's ongoing development and market potential in rare liver diseases.
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Analyst Views on MIRM
Wall Street analysts forecast MIRM stock price to rise
9 Analyst Rating
9 Buy
0 Hold
0 Sell
Strong Buy
Current: 102.040
Low
81.00
Averages
102.33
High
140.00
Current: 102.040
Low
81.00
Averages
102.33
High
140.00
About MIRM
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company. The Company is focused on the treatment of rare diseases affecting children and adults. It has three medicines: LIVMARLI (maralixibat) oral solution (Livmarli), Cholbam (cholic acid) capsules, and Chenodal or Ctexli (chenodiol) tablets. Livmarli is an orally administered, minimally-absorbed ileal bile acid transporter (IBAT) inhibitor (IBATi) that is used for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the United States and various other countries and for cholestatic pruritus in patient with progressive familial intrahepatic cholestasis (PFIC) in the United States, Canada and Japan. Cholbam is used for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders. The Company is also advancing its product candidate, volixibat, for the treatment of adult patients with cholestatic liver diseases.
About the author
Emily J. Thompson
Emily J. Thompson, a Chartered Financial Analyst (CFA) with 12 years in investment research, graduated with honors from the Wharton School. Specializing in industrial and technology stocks, she provides in-depth analysis for Intellectia’s earnings and market brief reports.
Mirum and Incyte Report Positive Results for FOP Study
- Clinical Trial Results: Mirum and Incyte's Phase II PROGRESS study of zilurgisertib showed that only 3.1% of patients developed new lesions compared to 16.7% in the placebo group, achieving an 81% reduction, indicating the drug's potential in treating this ultra-rare disease.
- Sustained Efficacy: In the open-label extension phase, no new lesions were observed among patients continuing zilurgisertib treatment at 48 weeks, with total lesion volume continuing to decline, demonstrating the drug's long-term efficacy and safety, addressing the urgent need for effective treatments.
- FDA Priority Review: Following positive clinical data, the FDA has accepted the NDA for zilurgisertib and granted Priority Review status, with a decision expected by September 26, 2026, which will expedite the drug's market entry to meet the demand for new therapies.
- Market Performance: Year-to-date, shares of Mirum and Incyte have risen by 28.5% and 1%, respectively, against a 1.4% decline in the biotech sector, reflecting investor optimism regarding their clinical advancements and enhancing their competitive position in the market.
See More
Mirum and Incyte Report Positive Phase 2 Data for Zilurgisertib
- Clinical Trial Results: Mirum Pharmaceuticals and Incyte have reported positive pivotal Phase 2 data for their oral FOP candidate, zilurgisertib, with results from Cohort 1 of the PROGRESS study showing consistent treatment effects on disease activity and durability through Week 48, indicating the drug's therapeutic potential.
- Safety Assessment: During the open-label extension, no new HO lesions were observed in patients continuing zilurgisertib treatment or in placebo patients who crossed over to active treatment at week 24, demonstrating the drug's safety and efficacy in long-term use.
- FDA Review Progress: The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application for zilurgisertib and granted Priority Review, with a target action date set for September 26, 2026, laying the groundwork for the drug's market launch.
- Market Outlook: Incyte's Chief Medical Officer Steven Stein stated that the findings represent an important milestone for the zilurgisertib program, further strengthening the clinical evidence supporting its potential as a treatment for FOP, signaling future market opportunities in the rare disease sector.
See More
Mirum and Incyte Announce Positive Phase 2 Results for Zilurgisertib
- Clinical Trial Results: Mirum and Incyte presented positive results from the PROGRESS study at the 2026 ENDO meeting, showing that Zilurgisertib reduced new heterotopic ossification (HO) lesions by 81% in patients aged 12 and older over 24 weeks, indicating significant efficacy.
- Efficacy Metrics: Patients treated with Zilurgisertib experienced a 99.9% reduction in the total volume of new HO lesions at week 24, suggesting the drug's high potential in managing Fibrodysplasia Ossificans Progressiva (FOP) and possibly transforming treatment outcomes for patients.
- Safety Assessment: Zilurgisertib was generally well tolerated during the 24-week placebo-controlled period, with no new HO lesions observed, and total HO lesion volume continued to decrease from week 24 to week 48, indicating potential long-term benefits.
- Market Outlook: With approximately 300 patients in the U.S. and 900 worldwide affected by FOP, Mirum's NDA for Zilurgisertib is expected to be decided by the FDA in September 2026, which could present significant market opportunities for the company.
See More
Mirum and Incyte Announce New Progress in FOP Treatment
- Clinical Trial Results: At the ENDO 2026 conference, Mirum and Incyte presented results from Cohort 1 of the PROGRESS study, showing an 81% reduction in new heterotopic ossification lesions in patients treated with zilurgisertib, indicating significant efficacy in treating FOP.
- FDA Priority Review: The U.S. FDA has accepted the New Drug Application for zilurgisertib and granted Priority Review, with a decision expected by September 26, 2026, which could provide urgently needed treatment options for FOP patients and potentially reshape the market landscape.
- Patient Data Analysis: During the 24-week double-blind period, patients receiving zilurgisertib had significantly fewer new lesions compared to the placebo group, and no new lesions were observed during the 48-week open-label extension, demonstrating sustained treatment effects and enhancing the drug's market potential.
- Safety Assessment: The study indicated that zilurgisertib was well-tolerated during the 24-week controlled period, with most adverse events being mild or moderate and no treatment discontinuations, paving the way for future commercialization and boosting investor confidence.
See More
Mirum and Incyte Announce FOP Study Results
- Clinical Trial Results: At ENDO 2026, Mirum and Incyte presented data showing that zilurgisertib significantly reduced new HO lesions by 81% in FOP patients over 24 weeks, indicating its potential as a new treatment option for this rare disease.
- FDA Priority Review: The U.S. FDA has accepted the New Drug Application for zilurgisertib and granted it Priority Review, with a target action date of September 26, 2026, which could expedite its market entry to address urgent patient needs in the FOP community.
- Study Design and Findings: The PROGRESS study's first cohort included 63 patients, revealing significant reductions in HO lesion volume during the 24-week double-blind period, with no new lesions observed during the open-label extension, suggesting sustained efficacy.
- Safety Profile: Zilurgisertib demonstrated good tolerability during the 24-week study, with most adverse events being mild or moderate, and no treatment discontinuations, highlighting its safety and feasibility for clinical use.
See More
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- Knight-Swift Outlook: Susquehanna upgraded Knight-Swift from neutral to positive, highlighting strong earnings prospects in the trucking sector, although it noted that much of the good news is already priced in.
- Mobia Medical Coverage Initiation: Goldman Sachs initiated coverage on Mobia Medical with a buy rating and a $31 price target, indicating approximately 130% upside potential, reflecting the company's strong growth prospects in the medtech space.
- Intuit Downgrade: Goldman Sachs downgraded Intuit from neutral to sell, expressing concerns over heightened competition and noting a 46% year-to-date decline in stock price, which raises doubts about its future profitability.
See More
Mirum and Incyte Report Positive Results for FOP Study
- Clinical Trial Results: Mirum and Incyte's Phase II PROGRESS study of zilurgisertib showed that only 3.1% of patients developed new lesions compared to 16.7% in the placebo group, achieving an 81% reduction, indicating the drug's potential in treating this ultra-rare disease.
- Sustained Efficacy: In the open-label extension phase, no new lesions were observed among patients continuing zilurgisertib treatment at 48 weeks, with total lesion volume continuing to decline, demonstrating the drug's long-term efficacy and safety, addressing the urgent need for effective treatments.
- FDA Priority Review: Following positive clinical data, the FDA has accepted the NDA for zilurgisertib and granted Priority Review status, with a decision expected by September 26, 2026, which will expedite the drug's market entry to meet the demand for new therapies.
- Market Performance: Year-to-date, shares of Mirum and Incyte have risen by 28.5% and 1%, respectively, against a 1.4% decline in the biotech sector, reflecting investor optimism regarding their clinical advancements and enhancing their competitive position in the market.
See More
Mirum and Incyte Report Positive Phase 2 Data for Zilurgisertib
- Clinical Trial Results: Mirum Pharmaceuticals and Incyte have reported positive pivotal Phase 2 data for their oral FOP candidate, zilurgisertib, with results from Cohort 1 of the PROGRESS study showing consistent treatment effects on disease activity and durability through Week 48, indicating the drug's therapeutic potential.
- Safety Assessment: During the open-label extension, no new HO lesions were observed in patients continuing zilurgisertib treatment or in placebo patients who crossed over to active treatment at week 24, demonstrating the drug's safety and efficacy in long-term use.
- FDA Review Progress: The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application for zilurgisertib and granted Priority Review, with a target action date set for September 26, 2026, laying the groundwork for the drug's market launch.
- Market Outlook: Incyte's Chief Medical Officer Steven Stein stated that the findings represent an important milestone for the zilurgisertib program, further strengthening the clinical evidence supporting its potential as a treatment for FOP, signaling future market opportunities in the rare disease sector.
See More
Mirum and Incyte Announce Positive Phase 2 Results for Zilurgisertib
- Clinical Trial Results: Mirum and Incyte presented positive results from the PROGRESS study at the 2026 ENDO meeting, showing that Zilurgisertib reduced new heterotopic ossification (HO) lesions by 81% in patients aged 12 and older over 24 weeks, indicating significant efficacy.
- Efficacy Metrics: Patients treated with Zilurgisertib experienced a 99.9% reduction in the total volume of new HO lesions at week 24, suggesting the drug's high potential in managing Fibrodysplasia Ossificans Progressiva (FOP) and possibly transforming treatment outcomes for patients.
- Safety Assessment: Zilurgisertib was generally well tolerated during the 24-week placebo-controlled period, with no new HO lesions observed, and total HO lesion volume continued to decrease from week 24 to week 48, indicating potential long-term benefits.
- Market Outlook: With approximately 300 patients in the U.S. and 900 worldwide affected by FOP, Mirum's NDA for Zilurgisertib is expected to be decided by the FDA in September 2026, which could present significant market opportunities for the company.
See More
Mirum and Incyte Announce New Progress in FOP Treatment
- Clinical Trial Results: At the ENDO 2026 conference, Mirum and Incyte presented results from Cohort 1 of the PROGRESS study, showing an 81% reduction in new heterotopic ossification lesions in patients treated with zilurgisertib, indicating significant efficacy in treating FOP.
- FDA Priority Review: The U.S. FDA has accepted the New Drug Application for zilurgisertib and granted Priority Review, with a decision expected by September 26, 2026, which could provide urgently needed treatment options for FOP patients and potentially reshape the market landscape.
- Patient Data Analysis: During the 24-week double-blind period, patients receiving zilurgisertib had significantly fewer new lesions compared to the placebo group, and no new lesions were observed during the 48-week open-label extension, demonstrating sustained treatment effects and enhancing the drug's market potential.
- Safety Assessment: The study indicated that zilurgisertib was well-tolerated during the 24-week controlled period, with most adverse events being mild or moderate and no treatment discontinuations, paving the way for future commercialization and boosting investor confidence.
See More
Mirum and Incyte Announce FOP Study Results
- Clinical Trial Results: At ENDO 2026, Mirum and Incyte presented data showing that zilurgisertib significantly reduced new HO lesions by 81% in FOP patients over 24 weeks, indicating its potential as a new treatment option for this rare disease.
- FDA Priority Review: The U.S. FDA has accepted the New Drug Application for zilurgisertib and granted it Priority Review, with a target action date of September 26, 2026, which could expedite its market entry to address urgent patient needs in the FOP community.
- Study Design and Findings: The PROGRESS study's first cohort included 63 patients, revealing significant reductions in HO lesion volume during the 24-week double-blind period, with no new lesions observed during the open-label extension, suggesting sustained efficacy.
- Safety Profile: Zilurgisertib demonstrated good tolerability during the 24-week study, with most adverse events being mild or moderate, and no treatment discontinuations, highlighting its safety and feasibility for clinical use.
See More
Latest Rating Changes on Wall Street
- TripAdvisor Upgrade: Wedbush upgraded TripAdvisor from neutral to outperform, citing the stock's undervaluation and expected governance transformation post-LTRIP merger, alongside activist involvement as catalysts for future growth.
- Knight-Swift Outlook: Susquehanna upgraded Knight-Swift from neutral to positive, highlighting strong earnings prospects in the trucking sector, although it noted that much of the good news is already priced in.
- Mobia Medical Coverage Initiation: Goldman Sachs initiated coverage on Mobia Medical with a buy rating and a $31 price target, indicating approximately 130% upside potential, reflecting the company's strong growth prospects in the medtech space.
- Intuit Downgrade: Goldman Sachs downgraded Intuit from neutral to sell, expressing concerns over heightened competition and noting a 46% year-to-date decline in stock price, which raises doubts about its future profitability.
See More
