A Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Biliary Cholangitis

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Biliary Cholangitis (PBC), and to assess the possible impact on the disease progression of PBC.

Start Date22 Sep 2021

Sponsor / Collaborator

Mirum Pharmaceuticals, Inc.

NCT04718961

/ TerminatedPhase 2

A Phase 2a/2b Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in Adult Women With Intrahepatic Cholestasis of Pregnancy and Elevated Serum Bile Acid Concentrations (OHANA).

Part 1 is an open-label randomized study of volixibat in patients with Intrahepatic Cholestasis of Pregnancy (ICP) and elevated serum bile acid concentrations (sBA) to evaluate safety and tolerability of two doses of volixibat. Part 2 is a double-blind, placebo controlled, study designed to evaluate the safety and efficacy of a selected volixibat dose.

Start Date04 Jan 2021

Sponsor / Collaborator

Mirum Pharmaceuticals, Inc.

NCT04663308

/ Active, not recruitingPhase 2

A Randomized Double-Blind Placebo-Controlled Study to Evaluate the Efficacy and Safety of Volixibat in the Treatment of Cholestatic Pruritus in Patients With Primary Sclerosing Cholangitis

The purpose of this clinical research study is to learn more about the use of the study medicine, volixibat, for the treatment of pruritus (itching) associated with Primary Sclerosing Cholangitis (PSC), and to assess the possible impact on the disease progression of PSC.

Start Date18 Dec 2020

Sponsor / Collaborator

Mirum Pharmaceuticals, Inc.

100 Clinical Results associated with Volixibat

100 Translational Medicine associated with Volixibat

100 Patents (Medical) associated with Volixibat

Literatures (Medical) associated with Volixibat

01 Mar 2026·Liver International

Efficacy, Safety and Tolerability of Volixibat, an IBAT Inhibitor, in Patients With Intrahepatic Cholestasis of Pregnancy

Article

Author: Li, Furong ; Stone, Sophia ; Chien, Elaine ; Kahng, Jennifer ; Mogul, Douglas B. ; Vig, Pamela ; Nunes, Tiago ; Ovadia, Caroline ; Sibai, Baha ; Krishnaswami, Jayshree ; Williamson, Catherine ; Warsi, Qurratul Ann

ABSTRACT:

Intrahepatic cholestasis of pregnancy (ICP) presents with cholestatic pruritus, elevated sBA and increased risk of adverse perinatal outcomes. Volixibat is a minimally absorbed IBAT inhibitor that interrupts enterohepatic recirculation. We describe four patients with ICP with pruritus and sBA > ULN treated with volixibat (20 or 80 mg BID orally until delivery), with dose modifications permitted for tolerability. Daily pruritus scores, sBA, liver enzymes, perinatal outcomes and TEAEs were assessed. Over 1000 patients were invited to participate; 26 were screened, and four received volixibat. Three patients experienced reductions in pruritus from baseline regardless of volixibat dose, with intermittent relief coinciding with resumption of dosing when doses were interrupted. sBA nadir values all reached < 6 μmol/L following volixibat. No clinically meaningful changes in laboratory parameters were observed. All patients had healthy live births. Most frequent TEAEs were gastrointestinal. Volixibat demonstrated improvements in pruritus and sBA in patients with ICP.Trial Registration: NCT04718961

01 Dec 2025·Gastroenterology & hepatology

Volixibat Leads to Improvements in Fatigue and Sleep for Adults With PBC: Data From VANTAGE.

Article

01 May 2022·ALIMENTARY PHARMACOLOGY & THERAPEUTICS

Meta‐analysis: analysis of mechanistic pathways in the treatment of non‐alcoholic steatohepatitis. Evidence from a Bayesian network meta‐analysis

Article

Author: Nicholas Chew ; Eunice Tan ; Jie Ning Yong ; Xin‐Hui Pan ; Mazen Noureddin ; Phoebe Tay ; Mark D. Muthiah ; Daniel Q. Huang ; Arun J. Sanyal ; Cheng Han Ng ; Mohammad Shadab Siddiqui ; Darren Jun Hao Tan ; Jieling Xiao ; Rohit Loomba ; Wen Hui Lim ; Nicholas Syn ; Grace Lim ; Jeffery Wei Heng Koh ; Yip Han Chin

Summary:

Background and Aims:

Non‐alcoholic steatohepatitis (NASH) is the most common cause of liver disease. However, there is lack of comparison of efficacy between different NASH drug classes. We conducted a network meta‐analysis evaluating drug classes through comparing histological outcomes and targets of drugs.

Approach and Results:

Medline, EMBASE and CENTRAL were searched for randomised controlled trials evaluating NASH drugs in biopsy‐proven NASH patients. Primary outcomes included NASH resolution without worsening of fibrosis, at least 2‐point reduction in Non‐alcoholic fatty liver disease Activity Score (NAS) without worsening of fibrosis and at least 1‐point reduction in fibrosis. Treatments were classified into inflammation, energy, bile acid and fibrosis modulators. The analysis was conducted with Bayesian network model and surface under the cumulative ranking curve (SUCRA) analysis. Among 49 included trials, treatments modulating energy (Risk ratio (RR): 1.92, Credible intervals (Crl): 1.59‐2.34) were most likely to achieve NASH resolution followed by treatments modulating fibrosis (RR 1.66, Crl: 0.65‐4.50), bile acids (RR: 1.37, Crl: 0.99‐1.92) and inflammation (RR: 1.00, Crl: 0.75‐1.33). Energy and bile acids modulation were effective in at least 2‐point NAS reduction without worsening of fibrosis (RR: 1.52, Crl 1.30‐1.77; RR: 1.69, Crl 1.41‐2.03) and at least 1‐point reduction in fibrosis (RR: 1.26, Crl:1.05‐1.49; RR: 1.54, Crl: 1.20‐1.97).

Conclusions:

This network analysis demonstrates the relative superiority of drugs modulating energy pathways and bile acids in NASH treatment. This guides the development and selection of drugs for combination therapies.

201

News (Medical) associated with Volixibat

14 Jul 2026

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Ligand Completes Acquisition of XOMA Royalty, Creating a Portfolio of More than 200 Biopharmaceutical Royalty Assets

Acquisition adds seven commercial products including VABYSMO® (faricimab-svoa), OJEMDA™ (tovorafenib), and MIPLYFFA® (arimoclomol), and more than 100 development and commercial-stage assets, creating long-term opportunities to drive growth Ligand to provide updated 5-year outlook during Investor Day on December 8, 2026 JUPITER, Fla., July 14, 2026 (GLOBE NEWSWIRE) -- Ligand Pharmaceuticals Incorporated (Nasdaq: LGND) today announced it has completed its acquisition of XOMA Royalty Corporation (“XOMA Royalty”), a biotechnology royalty aggregator, for $39.00 per share of common stock in cash, for a total equity value of approximately $739 million. XOMA Royalty stockholders also received one non-transferable Contingent Value Right (“CVR”) per share entitling the holder to receive a portion of 75% of the net proceeds that may result from certain pending litigation at XOMA Royalty. “At Ligand, we are focused on building a diversified portfolio of high-value royalty assets tied to innovative medicines that have the potential to improve patient outcomes around the world,” said Todd Davis, CEO of Ligand. “Over the past several years, we have executed a disciplined strategy to expand, diversify and strengthen our royalty portfolio, and the acquisition of XOMA Royalty meaningfully advances that vision. This transaction adds exposure to high-quality commercial and development-stage assets and enhances our ability to generate durable, long-term shareholder value, marking an important milestone in our evolution as a leading biopharmaceutical royalty aggregator.” This acquisition strengthens Ligand’s royalty portfolio by adding seven commercial products, including Roche’s VABYSMO® (faricimab-svoa), Servier’s OJEMDA™ (tovorafenib), and Zevra Therapeutics’ MIPLYFFA® (arimoclomol). Additionally, it includes 14 late-stage development programs, featuring Takeda’s mezagitamab and certain assets from Takeda’s externalized asset portfolio, such as osavampator, volixibat, and OHB-607, along with more than 100 assets in various stages of development. As a result, Ligand’s portfolio has more than doubled in size, now comprising over 200 commercial, clinical, and preclinical stage royalty assets. The closing of the transaction met Ligand’s original timeline expectations. The transaction is expected to be immediately accretive and to add approximately $0.50 and $1.50 per share to Ligand’s projected 2026 and 2027 adjusted earnings per share 1 , respectively. Ligand will provide an updated 5-year outlook during the Company’s Investor Day on December 8, 2026. In connection with the completion of the transaction, XOMA Royalty common stock ceased trading on The Nasdaq Global Market. Advisors Stifel served as lead financial advisor, Citi served as financial advisor, Paul Hastings LLP served as legal advisor, and Collected Strategies served as strategic communications advisor to Ligand. Leerink Partners served as lead financial advisor, H.C. Wainwright & Co. served as financial advisor, and Gibson, Dunn & Crutcher LLP served as legal advisor to XOMA Royalty. About Ligand Ligand is a leading royalty aggregator, partnering with biopharmaceutical companies to finance and advance late-stage clinical development programs. Ligand owns and manages one of the largest and most diversified portfolios of biopharmaceutical royalties in the industry, with economic interests in more than 200 development and commercial-stage assets. Ligand funds high-value programs in exchange for long-term economic interests, aligning capital with clinical and commercial success. Ligand’s royalty portfolio is designed to deliver consistent and predictable revenue streams across a broad range of therapeutic assets. Ligand also licenses its proprietary technologies, Captisol® and NITRICIL™, to support drug development and formulation across its global partner network. For more information, visit or follow Ligand on X and LinkedIn . Forward-Looking Statements This press release contains “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995 that involve substantial risks and uncertainties, including information about, among other topics, Ligand’s acquisition of XOMA Royalty, Ligand’s and XOMA Royalty’s products pipeline and the substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by such statements. Risks and uncertainties include, among other things, risks related to the ability to realize the anticipated benefits of the acquisition, including the possibility that the expected benefits from the acquisition will not be realized or will not be realized within the expected time period; the risk that the businesses will not be integrated successfully; the risk that XOMA Royalty stockholders may receive no payment with respect to the CVRs; disruption from the transaction making it more difficult to maintain business and operational relationships, including the ability to attract and retain highly qualified management and other clinical and scientific personnel; negative effects of this announcement or the completion of the acquisition on the market price of Ligand’s common stock and/or operating results; significant transaction costs; unknown liabilities; the risk of litigation and/or regulatory actions related to the acquisition of XOMA Royalty’s business; other business effects and uncertainties, including the effects of industry, market, business, economic, political or regulatory conditions; future exchange and interest rates; risks and uncertainties related to issued or future executive orders or other new, or changes in, laws, regulations or policy; changes in tax and other laws, regulations, rates and policies; the uncertainties inherent in business and financial planning, including, without limitation, risks related to Ligand’s business and prospects, adverse developments in Ligand’s markets, or adverse developments in the U.S. or global capital markets, credit markets, regulatory environment, tariffs and other trade policies or economies generally; future business combinations or disposals; uncertainties regarding the commercial success of commercialized and/or pipeline products; risks associated with drug development; Ligand’s reliance on collaborative partners for milestone payments, royalties, materials revenue, contract payments and other revenue projections, which may not be received; the uncertainties inherent in research and development, including the ability of Ligand’s partners to meet anticipated clinical endpoints, commencement and/or completion dates for clinical trials, regulatory submission dates, regulatory approval dates and/or launch dates, as well as the possibility of unfavorable new clinical data and further analyses of existing clinical data; risks associated with initial, preliminary or interim data; the risk that clinical trial data are subject to differing interpretations and assessments by regulatory authorities; whether regulatory authorities will be satisfied with the design of and results from the clinical trials conducted by Ligand’s partners; whether and when drug applications may be filed in any jurisdictions for pipeline products for any potential indications by Ligand’s partners; whether and when any such applications may be approved by regulatory authorities, which will depend on myriad factors, including making a determination as to whether the product’s benefits outweigh its known risks and determination of the product’s efficacy and, if approved, whether any such products will be commercially successful; and decisions by regulatory authorities impacting labeling, manufacturing processes, safety and/or other matters that could affect the availability or commercial potential of such products. You should carefully consider the foregoing factors and the other risks and uncertainties that affect the businesses of Ligand and XOMA Royalty described in the “Risk Factors” and “Cautionary Note Regarding Forward-Looking Statements” (in the case of Ligand) and “Forward Looking Statements” (in the case of XOMA Royalty) sections of their respective Annual Reports on Form 10-K, Quarterly Reports on Form 10-Q and other documents filed by either of them from time to time with the U.S. Securities and Exchange Commission (the “SEC”), all of which are available at . These filings identify and address other important risks and uncertainties that could cause actual events and results to differ materially from those contained in the forward-looking statements. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and Ligand assumes no obligation to, and does not intend to, update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise, unless required by law. Ligand gives no assurance that it will achieve its expectations. Contacts Investors: Melanie Herman investors@ligand.com (858) 550-7761 Media: Nick Lamplough/Jude Gorman Ligand-CS@collectedstrategies.com (917) 885-1013 1 The financial outlook, expectations and other forward-looking statements provided by Ligand for 2026 and beyond reflect Ligand's judgment based on the information available at the time of this release. Please see the Forward-looking Statements section in this release for factors that may impact Ligand's ability to meet expectations. In reliance upon Item 10(e)(1)(i)(B) of Regulation S-K, reconciliations of forward-looking core adjusted earnings per diluted share for 2027 is not provided because of the unreasonable effort associated with providing such reconciliations due to the variability in the occurrence and the amounts of certain components thereof. For the same reasons, we are unable to address the significance of the unavailable information, which could be material to future results.

13 Jul 2026

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Mirum Pharmaceuticals Reports Inducement Grants Under Nasdaq Listing Rule 5635(c)(4) - July 13, 2026

FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced that on July 10, 2026, the Compensation Committee of Mirum’s Board of Directors granted inducement awards consisting of non-qualified stock options to purchase 6,300 shares of common stock and 9,300 restricted stock units (“RSUs”) to 6 new employees under Mirum’s 2020 Inducement Plan. The Compensation Committee of Mirum’s Board of Directors approved the awards as an inducement material to the new employees’ employment in accordance with Nasdaq Listing Rule 5635(c)(4). Each stock option has an exercise price of $122.13 per share, which is equal to the closing price of Mirum’s common stock on July 10, 2026, and will vest over four years, with 25% of the underlying shares vesting on the one-year anniversary of the applicable vesting commencement date, and the balance of the underlying shares vesting monthly thereafter over 36 months, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The RSUs will vest over three years, with 33% of the underlying shares vesting on each anniversary of the applicable initial vesting date, subject to the new employees’ continued service relationship with Mirum through the applicable vesting dates. The awards are subject to the terms and conditions of Mirum’s 2020 Inducement Plan and the terms and conditions of an applicable award agreement covering the grant. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI ® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM ® (cholic acid) for bile-acid synthesis disorders, and CTEXLI ® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X . Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. Contacts Investor Contact: Andrew McKibben ir@mirumpharma.com Media Contact: Meredith Kiernan media@mirumpharma.com

Drug Approval

15 Jun 2026

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Mirum Pharmaceuticals and Incyte Announce Positive Pivotal Phase 2 Results from PROGRESS Study of Zilurgisertib in Fibrodysplasia Ossificans Progressiva

- Cohort 1 results presented at ENDO 2026 demonstrate meaningful reductions in total heterotopic ossification (HO) lesion volume, new HO lesions and flare activity in adolescents and adults with FOP - U.S. Food and Drug Administration (FDA) accepted the New Drug Application (NDA) for zilurgisertib in FOP under Priority Review FOSTER CITY, Calif. & WILMINGTON, Del.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq:MIRM) and Incyte (Nasdaq:INCY) today announced pivotal Phase 2 results from Cohort 1 of the PROGRESS study evaluating zilurgisertib, an investigational oral activin receptor-like kinase 2 (ALK2) inhibitor, in adolescents and adults (≥12 years of age) with fibrodysplasia ossificans progressiva (FOP). Results were shared in a late-breaking rapid-fire presentation at ENDO 2026, the Endocrine Society’s annual meeting. Results from Cohort 1 of the PROGRESS study demonstrated a consistent treatment effect across measures of disease activity and durability through Week 48. During the open-label extension, no new HO lesions were observed among patients who continued to receive zilurgisertib or among placebo-treated patients who crossed over to active treatment at Week 24. "The findings presented at ENDO represent an important milestone for the zilurgisertib program and further strengthen the growing body of clinical evidence supporting its potential as a treatment for FOP," said Steven Stein, M.D., Executive Vice President, Chief Medical Officer and Head of Late-Stage Development at Incyte. "People living with FOP and their families urgently need additional treatment options," said Joanne Quan, M.D., Chief Medical Officer at Mirum Pharmaceuticals. "These results reinforce our confidence in the potential of zilurgisertib and our commitment to working with Incyte to bring this important program forward as we prepare for potential commercialization and support the FOP community." Cohort 1 of the PROGRESS study evaluated zilurgisertib 100 mg once-daily in 63 adolescents and adults (≥12 years of age) with FOP. Patients were randomized 1:1 to receive zilurgisertib (n=32) or placebo (n=31) during a 24-week, placebo-controlled, double-blind period, followed by an open-label extension period. Baseline demographics and disease characteristics were generally balanced between treatment groups, with a mean age of approximately 21 years and evidence of recent disease activity prior to enrollment. A total of 61 patients had 48-week whole-body CT scan data available at the time of the open-label extension analysis. Key efficacy findings included: Fewer patients receiving zilurgisertib developed new HO lesions at Week 24, with an 81% reduction versus placebo (p=0.0986). 99.9% reduction in total volume of new HO lesions in patients receiving zilurgisertib versus placebo at Week 24 (nominal p-value<0.0001). Reduction in total existing HO lesion volume compared with an increase observed in placebo-treated patients at Week 24 (nominal p-value=0.004). Among patients receiving zilurgisertib, no new HO lesions were observed and total HO lesion volume continued to decrease from Week 24 to Week 48. Among patients who crossed over from placebo to zilurgisertib, no new HO lesions were observed and total HO lesion volume decreased from Week 24 to Week 48. Key Efficacy Findings (Week 24 Placebo-Controlled Period and Week 48 Crossover) Endpoint Zilurgisertib (ZGB) (n=32) Week 24 Placebo (n=31) Week 24 Key Finding Open-Label Extension Week 48 Number (%) of patients who developed new HO lesions 1 (3.1) 5 (16.7) 81% reduction vs placebo No patients with new HO lesions observed at Week 48 (n=61) P-value 0.0986 Mean (SD) total number of new HO lesions 0.06 (0.35) 0.23 (0.63) Fewer new lesions vs placebo No new lesions observed (n=61) Mean (SD) new lesion volume, cm³ 0.003 (0.02) 6.57 (20.70) 99.9% reduction vs placebo No new lesions observed (n=61) P-value <0.0001* Mean (SD) change in total lesion volume, cm³ -3.24 (19.86) 24.64 (51.94) Reduction vs increase on placebo Continued reduction from Week 24 -6.37 (19.43) ZGB (n=32) -5.32 (20.91) crossover (n=29) P-value 0.004* Mean (SD) new flares (annualized) 2.34 (6.06) 4.55 (7.71) Lower flare activity vs placebo Low flare activity maintained 1.01 (3.26) ZGB (n=32) 1.22 (2.54) crossover (n=30) *Nominal P-value Zilurgisertib was generally well-tolerated during the 24-week placebo-controlled period of the study. Data showed: Most adverse events were mild or moderate in severity. No adverse events led to treatment discontinuation or dose reduction. Serious adverse events and Grade ≥3 adverse events occurred at low rates in both treatment groups. The most commonly reported adverse events among patients receiving zilurgisertib were FOP flare-up or aching/pain due to FOP (25%), headache (21.9%), upper respiratory tract infection (21.9%), arthralgia (18.8%), epistaxis (12.5%), and nausea (12.5%). The full abstract is available on the Endocrine Society’s ENDO 2026 website. Detailed analyses are also posted on the Publications & Presentations section of Mirum’s website. The U.S. Food and Drug Administration (FDA) has accepted the New Drug Application (NDA) for zilurgisertib for the treatment of FOP in patients 12 years of age and older and granted Priority Review. The Prescription Drug User Fee Act (PDUFA) target action date for zilurgisertib is September 26, 2026. About Zilurgisertib Zilurgisertib is an investigational, oral, small molecule, activin receptor-like kinase 2 (ALK2) inhibitor in development for the treatment of Fibrodysplasia Ossificans Progressiva (FOP). Zilurgisertib is designed to inhibit the ALK2 receptor, which is abnormally active in most patients with FOP and leads to bone formation in soft tissues, a process known as heterotopic ossification (HO). FOP is an ultra-rare genetic disease that affects approximately 300 patients in the U.S. and 900 worldwide, with diagnosis typically occurring in early childhood. Zilurgisertib was evaluated in the PROGRESS pivotal Phase 2 study, which formed the basis of a new drug application (NDA). The FDA has accepted the NDA for zilurgisertib in FOP under Priority Review with a Prescription Drug User Fee Act (PDUFA) date of September 26, 2026. Mirum Pharmaceuticals, Inc. licensed zilurgisertib from Incyte for worldwide development and commercialization. About the PROGRESS Study PROGRESS is a global, randomized, double-blind, placebo-controlled Phase 2 study evaluating the efficacy and safety of zilurgisertib in patients with fibrodysplasia ossificans progressiva (FOP). PROGRESS Cohort 1 enrolled patients 12 years of age and older who were randomized 1:1 to receive zilurgisertib 100 mg once daily or placebo during a 24-week double-blind treatment period, followed by an open-label extension. Additional PROGRESS cohorts will evaluate the efficacy and safety of zilurgisertib in patients ages 6 to <12 years of age (Cohort 2) and in patients ages 2 to <12 years of age (Cohort 3). The primary endpoint of the study is the proportion of Cohort 1 patients with new heterotopic ossification (HO) lesions at Week 24 as assessed by whole-body CT scan data. Key secondary endpoints include the number and total volume of new HO lesions, changes in total HO lesion volume and flare activity through Week 24. About Mirum Pharmaceuticals Mirum Pharmaceuticals (NASDAQ: MIRM) is a leading rare disease company with a global footprint of approved products and a broad pipeline of investigational medicines. Purpose-built to bring forward breakthrough medicines for people with overlooked conditions, Mirum focuses on rare liver and rare genetic diseases, where it has built deep expertise and strong connections to patient communities. The company’s commercial portfolio includes LIVMARLI® (maralixibat) for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC), CHOLBAM® (cholic acid) for bile-acid synthesis disorders, and CTEXLI® (chenodiol) for cerebrotendinous xanthomatosis (CTX). Mirum’s clinical-stage pipeline includes volixibat, an IBAT inhibitor in late-stage development for primary sclerosing cholangitis (PSC) and primary biliary cholangitis (PBC), brelovitug, a fully human monoclonal antibody in late-stage development for chronic hepatitis delta virus (HDV), zilurgisertib, an ALK2 inhibitor under regulatory review with the FDA for fibrodysplasia ossificans progressiva (FOP), and MRM-3379, a PDE4D inhibitor being evaluated for Fragile X syndrome (FXS). Mirum’s success is driven by a team dedicated to advancing high impact medicines through strategic development, disciplined execution and purposeful collaboration across the rare disease ecosystem. Learn more at and follow Mirum on Facebook , LinkedIn , Instagram and X . About Incyte® Incyte is redefining what’s possible in biopharmaceutical innovation. Through deep scientific expertise and a relentless focus on patients, we have built an established portfolio of first-in-class medicines and an extensive portfolio of next-generation medicines across our key franchises: Hematology, Oncology and Inflammation & Autoimmunity. To learn more, visit Incyte.com and Investor.Incyte.com . Follow us on social media: LinkedIn , X and Instagram . Mirum 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 Company’s planned participation at a scientific congress, Mirum’s continued advancement of zilurgisertib with Incyte, the likelihood of a FDA approval pathway for zilurgisertib and the potential benefit of zilurgisertib in real world settings versus scientific presentations of data. 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,” “continue” 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 Annual Report for the year ended December 31, 2025, filed with the Securities and Exchange Commission on February 25, 2026, and subsequent filings with the Securities and Exchange Commission, which are available at . 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. Incyte Forward-Looking Statements This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995 and other federal securities laws, including statements regarding the presentation of data from the PROGRESS study; the potential for zilurgisertib to become a treatment option for people living with FOP; expectations regarding ongoing and future clinical trials for zilurgisertib, including the timing of such trials; and Incyte’s aspirations and goals as set forth under the heading “About Incyte.” Actual results may differ materially from those indicated in the forward-looking statements as a result of various important factors, including the sufficiency of clinical trial data to meet applicable regulatory standards or warrant continued development; the ability to enroll sufficient numbers of subjects in clinical trials and the ability to enroll subjects in accordance with planned schedules; actions of regulatory agencies, which may affect the initiation, timing and progress of clinical trials and marketing approval; the efficacy or safety of Incyte’s and its partners’ products; the ability of Incyte and its partners to achieve commercial success for their marketed products and product candidates, if approved; Incyte’s and its partners’ ability to obtain and maintain protection of intellectual property for their products and technology; Incyte’s reliance on third parties and partners; the acceptance of Incyte’s and its partners’ products in the marketplace; market competition, sales, marketing, manufacturing and distribution requirements; greater than expected expenses, including expenses relating to litigation or strategic activities; and those risks and uncertainties discussed in greater detail in Incyte’s reports filed with the U.S. Securities and Exchange Commission, including its annual report on Form 10-K for the year ended December 31, 2025, and its quarterly report on Form 10-Q for the quarter ended March 31, 2026. Incyte disclaims any intent or obligation to update these forward-looking statements. Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc. Contacts Mirum Investor Contact: Andrew McKibben ir@mirumpharma.com Mirum Media Contact: Meredith Kiernan media@mirumpharma.com Incyte Investor Contact: ir@incyte.com Incyte Media Contact: media@incyte.com

Clinical ResultPhase 2NDAPriority Review

100 Deals associated with Volixibat

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KEGG	Wiki	ATC	Drug Bank
-	Volixibat	-	DB13914

R&D Status

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Primary Biliary Cholangitis	Phase 2	United States	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	China	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Japan	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Argentina	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Belgium	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Brazil	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Canada	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	France	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Germany	Mirum Pharmaceuticals, Inc.	22 Sep 2021
Primary Biliary Cholangitis	Phase 2	Israel	Mirum Pharmaceuticals, Inc.	22 Sep 2021

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05050136 (VANTAGE, Company_Website) Manual	Phase 2	Cholestatic pruritus \| Primary Biliary Cholangitis	31	Volixibat	qspwkmfmny(lobbkkyesu) = mqfguglxeh atsgajyipv (prwgcvgauo ) View more	Positive	09 May 2025
				Volixibat 20 mg	qspwkmfmny(lobbkkyesu) = lpgqopgyih atsgajyipv (prwgcvgauo )
NCT04718961 (OHANA, CTgov)	Phase 2	Intrahepatic Cholestasis of Pregnancy	4	Placebo	dchntvaoyy(upzsgrywze) = eegppcybec qmkkbohaen (zfbegvliba, vfaahtcwat - cfyjfhyieq) View more	-	06 Aug 2024
NCT05050136 (VANTAGE, Biospace) Manual	Phase 2	Primary Biliary Cholangitis	30	volixibat	ptreeasqoy(xxtyjibhqw) = scccpnirgj ikevwqutcx (kjnzhngjhr, 0.414) View more	Positive	17 Jun 2024
				volixibat (20mg)	ptreeasqoy(xxtyjibhqw) = eymocwssni ikevwqutcx (kjnzhngjhr, 0.609)
NCT04718961 (OHANA, AASLD2023) Manual	Phase 2	Intrahepatic Cholestasis of Pregnancy	4	Volixibat	quibnltrir(uleduoknlg) = ptdvqnhxdq xctxmkoopk (vfavqyoiwn ) View more	Positive	13 Nov 2023
NCT02787304 (Pubmed \| J Hepatol) Manual	Phase 2	Metabolic Dysfunction Associated Steatohepatitis	197	volixibat	beamnxxdap(mnxqqgiqbw) = hynibzsfiz wbkayogeuj (oouekeasat ) View more	Negative	01 Aug 2020
				Placebo	beamnxxdap(mnxqqgiqbw) = coivcxmybt wbkayogeuj (oouekeasat )
NCT02787304 (CTgov)	Phase 2	Metabolic Dysfunction Associated Steatohepatitis	197	SHP626 (SHP626 5 Milligram (mg))	jarowvlvlj = drlmjwhqdv budynkezyl (ehogvscqgj, oedqbwqfhn - tdgmtcxkyv) View more	-	25 Nov 2019
				SHP626 (SHP626 10 Milligram (mg))	jarowvlvlj = bcjvydgmok budynkezyl (ehogvscqgj, wnywjssyvn - apfoyjmffc) View more
NCT02287779 (Pubmed \| BMC Pharmacol Toxicol) Manual	Phase 1	Metabolic Dysfunction Associated Steatohepatitis	84	volixibat	hlihlubaww(zshscuqrgc) = no serious adverse events eygsjgjuwm (kjdjptqxqb )	Positive	16 Mar 2018
				Placebo
NCT02571192 (Pubmed \| Eur J Drug Metab Pharmacokinet) Manual	Phase 1	-	8	[ 14 C]-Volixibat	riofejucyl(gexdlcgmrk) = No radioactivity was observed in plasma or whole blood. The percentage (mean ± standard deviation) of total radioactivity in urine was 0.01 ± 0.007%. The vast majority (92.3 ± 5.25%) of volixibat was recovered in feces (69.2 ± 33.1% within 24 h). lrfjmyhifx (ucdolrlmuz ) View more	Positive	01 Feb 2018
NCT02287779 (CTgov)	Phase 1	Metabolic Dysfunction Associated Steatohepatitis \| Obesity	84	Placebo	ezpgyyojpn = ufokphrmct dxffkgljdi (kusjmhsvvq, lyqkpladhx - zkcpdwiqxd) View more	-	07 Dec 2016

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