RegulationPriority Review (United States), Fast Track (United States), Orphan Drug (United States), Orphan Drug (European Union), Breakthrough Therapy (China)

Structure/Sequence

Molecular FormulaC34H46N4O9

InChIKeyTYGOIKFMUXDYPB-NZTRXVCXSA-N

CAS Registry2173390-29-7

Clinical Trials associated with Zilurgisertib

NCT05090891

/ RecruitingPhase 2

A Phase 2, Randomized, Double-Blind, Placebo-Controlled Study to Evaluate the Efficacy, Safety, and Tolerability of INCB000928 in Participants With Fibrodysplasia Ossificans Progressiva

This Phase 2, Randomized, Double-Blind, Placebo-Controlled Study is intended to evaluate the Efficacy, Safety, and Tolerability and PK of INCB000928 administered to participants with a clinical diagnosis of fibrodysplasia ossificans progressiva (FOP).

Start Date05 May 2022

Sponsor / Collaborator

Incyte Corp.

NCT05099445

/ CompletedPhase 1

A Phase 1 Study to Evaluate the Pharmacokinetics, Safety, and Pharmacodynamics of INCB000928 in Participants With Impaired Renal Function and Hemodialysis

This is a multicenter, open-label parallel-group to evaluate single oral doses of INCB000928 in participants with varying level of renal function or impairment.

Start Date14 Dec 2021

Sponsor / Collaborator

Incyte Corp.

NCT04582539

/ TerminatedPhase 1/2

A Phase 1/2, Open-Label, Multicenter Study of INCB000928 Administered as a Monotherapy in Participants With Anemia Due to Myelodysplastic Syndromes or Multiple Myeloma

This Phase 1/2, open-label, dose-finding study is intended to evaluate the safety and tolerability, PK, PD, and efficacy of INCB000928 administered as monotherapy in participants with MDS or MM who are transfusion-dependent or present with symptomatic anemia.

Start Date19 Aug 2021

Sponsor / Collaborator

Incyte Corp.

100 Clinical Results associated with Zilurgisertib

100 Translational Medicine associated with Zilurgisertib

100 Patents (Medical) associated with Zilurgisertib

Literatures (Medical) associated with Zilurgisertib

01 May 2026·BRITISH JOURNAL OF CLINICAL PHARMACOLOGY

Effects of Renal and Hepatic Impairment on the Pharmacokinetics of Zilurgisertib

Article

Author: Phillip Wang ; Yan-ou Yang ; Xiang Liu ; Jay Getsy ; Kevin Rockich ; Yunlan Fang

Aim:

Small‐molecule activin receptor‐like kinase‐2 inhibitor zilurgisertib (INCB000928) is under investigation in a pivotal trial for the treatment of fibrodysplasia ossificans progressiva (FOP), an ultrarare genetic condition. This analysis assessed effects of renal and hepatic impairment on zilurgisertib pharmacokinetics.

Methods:

Two open‐label, parallel‐group studies were conducted. Participants with renal or hepatic impairment were matched with healthy controls and received a single 200‐mg dose of zilurgisertib; those with end‐stage renal disease (ESRD) received doses before and after haemodialysis. Plasma concentrations were quantified via validated assay; pharmacokinetic parameters were derived from non‐compartmental analysis. Geometric mean ratios (GMRs) and 90% confidence intervals (CIs) for C max , AUC 0‐t and AUC 0‐∞ were compared by ANOVA.

Results:

Compared with matched controls, GMRs (90% CIs) for C max and AUC 0‐∞ were as follows: mild renal impairment, 0.92 (0.64–1.34) and 0.98 (0.77–1.24); moderate, 1.22 (0.92–1.63) and 1.64 (1.28–2.10); severe, 1.00 (0.74–1.35) and 1.70 (1.33–2.16), respectively. Among participants with ESRD, values were the following: before haemodialysis, 1.17 (0.91–1.49) and 1.47 (1.15–1.88); after, 1.32 (1.00–1.75) and 1.68 (1.29–2.19), respectively. Among participants with hepatic impairment, values were as follows: moderate, 1.06 (0.80–1.42) and 1.13 (0.93–1.38); severe: 1.36 (1.05–1.76) and 1.25 (1.06–1.48), respectively.

Conclusions:

Zilurgisertib exposure was unchanged with mild renal or moderate hepatic impairment, but increased by 30%–70% with moderate or more severe renal impairment and by 25% with severe hepatic impairment. These findings, alongside the therapeutic index, to be defined by the ongoing pivotal FOP trial (NCT05090891), will guide dosing in these populations.

01 Feb 2026·Clinical Pharmacology in Drug Development

Evaluation of clinical cardiac safety of zilurgisertib, an activin receptor‐like kinase‐2 (ALK2) inhibitor, in healthy participants

Article

Author: Liu, Xing ; Yang, Hong ; Gong, Xiaohua ; Yang, Yan‐ou ; Getsy, Jay ; Rockich, Kevin

Abstract:

The oral, small molecule inhibitor of activin receptor‐like kinase‐2, zilurgisertib (INCB000928), is under evaluation in fibrodysplasia ossificans progressiva. Cardiac safety was assessed using electrocardiogram (ECG) parameters and a plasma concentration‐heart rate‐corrected QT (C‐QTc) interval analysis of pooled data from single ascending dose (SAD) and multiple ascending dose (MAD) studies of zilurgisertib in healthy adult participants (SAD: 10‐500 mg; INCB00928‐102: 50‐400 mg QD, 300 mg BID). Overall, 91 (SAD) and 79 (MAD) participants provided at least one pair of PK/ECG data. As both studies indicated a dose‐dependent effect of zilurgisertib on heart rate, individualized QT correction (QTcI) was used as the primary endpoint for QTc analysis. Estimated population slope of the individualized C‐ΔQTc (C‐ΔQTcI) relationship was shallow (0.02 ms per µ m [90% CI, −0.60, 0.65]) and not statistically significantly different from 0; treatment effect–specific intercept was small and not significant (−0.83 ms [90% CI, −2.26, 0.61]). No significant relationship between zilurgisertib plasma concentration and change in QTcI was identified; zilurgisertib did not have a clinically relevant effect on QTc prolongation. QT effect > 10 ms could therefore be excluded within the dose range studied (up to 300 mg BID). No clinically meaningful effects on cardiac conduction (PR and QRS intervals) or any categorical PR or QRS outliers were observed. These data support further clinical development of zilurgisertib.

13 Nov 2025·ACS Medicinal Chemistry Letters

Discovery and Characterization of Zilurgisertib, a Potent and Selective Inhibitor of Activin Receptor-like Kinase-2 (ALK2) for the Treatment of Fibrodysplasia Ossificans Progressiva

Article

Author: Sylvie Teixeira-Fouchard ; Alexandra Hinniger ; Victoria Head ; Ina Kramer ; Michaela Kneissel ; Sven Weiler ; Luca Arista ; Thomas Ullrich ; Nikolaus Stiefl ; Sabine Guth ; Carien Dekker

Fibrodysplasia ossificans progressiva (FOP) is a rare autosomal-dominant disease leading to progressive soft tissue heterotopic ossification (HO) with no curative treatment available to date. It is caused by gain-of-function mutations in the activin A type-1 receptor ACVR1/ALK2, a member of the bone morphogenetic protein (BMP) type I receptor family. Most recent clinical trials in FOP have adopted for the first time on-target therapies to normalize the aberrant ALK2 receptor activity. Here we describe the discovery and preclinical characterization of zilurgisertib, a novel small-molecule inhibitor of ALK2 kinase with high biochemical and cellular potency, selectivity over other BMP and TGFβ signaling receptor kinases, and excellent oral bioavailability in preclinical species. Zilurgisertib fully suppresses HO in a pediatric mouse model of injury-induced FOP and therefore holds great potential as a novel targeted disease-modifying therapy for FOP. The candidate is being evaluated in clinical trials.

News (Medical) associated with Zilurgisertib

29 Sep 2026

·www.biospace.com

Mirum Pharmaceuticals and Incyte Announce U.S. FDA Approval of Atebrioz™ (zilurgisertib) for Adult and Pediatric Patients with Fibrodysplasia Ossificans Progressiva

-Once-daily oral ALK2 inhibitor approved to reduce the volume of total new heterotopic ossification in patients aged 12 years and older with FOP -Atebrioz expected to be available in the United States in October through Mirum Access Plus (MAP), with eligible patients paying as little as $0 per month -PROGRESS pediatric development program continues to evaluate zilurgisertib in children aged 2 to <12 years FOSTER CITY, Calif. & WILMINGTON, Del.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq:MIRM) and Incyte (Nasdaq:INCY) today announced that the U.S. Food and Drug Administration (FDA) has approved Atebrioz™ (zilurgisertib) tablets to reduce the volume of total new heterotopic ossification (HO) in adult and pediatric patients aged 12 years and older with fibrodysplasia ossificans progressiva (FOP). The recommended dose of Atebrioz is 100 mg administered orally, once daily. “Today marks an important milestone for people living with FOP, bringing a new treatment option to adult and pediatric patients living with this devastating disease,” said Chris Peetz, Chief Executive Officer at Mirum. “At Mirum, we are driven to serve rare disease communities where the unmet need is significant and the opportunity to make a difference is profound. The approval of Atebrioz reflects what can be achieved when industry, researchers and patient communities work together, and we remain committed to fostering that spirit of collaboration in FOP.” Atebrioz was developed by Incyte and licensed to Mirum Pharmaceuticals, Inc. for worldwide development and commercialization. Atebrioz is a once-daily oral activin receptor-like kinase 2 (ALK2) inhibitor designed to target the disease-driving pathway at the center of FOP biology. In people living with FOP, pathogenic variants in the ACVR1 gene result in the abnormal activation of ALK2, leading to the formation of bone in muscles, tendons, ligaments and other soft tissues through a process known as heterotopic ossification (HO). As HO lesions develop and accumulate over time, they can progressively restrict movement and lead to significant disability. “For families living with FOP, having additional treatment options means having greater flexibility in managing a complex, lifelong disease,” said Michelle Davis, Executive Director at the International Fibrodysplasia Ossificans Progressiva Association (IFOPA). “Every person’s experience with FOP is different, and expanding treatment options gives patients, families and their physicians the opportunity to consider what may be right for their individual needs.” “FOP is a lifelong disease in which the accumulation of HO leads to increasing disability and loss of function,” said Robert Pignolo, M.D., Ph.D., Robert and Arlene Kogod Professor of Geriatric Medicine at the Mayo Clinic College of Medicine and lead investigator for the PROGRESS study. “Having another treatment option is meaningful in a progressive disease like FOP, particularly for adolescents who may be earlier in the course of their disease.” Atebrioz was approved based on data from Cohort 1 of the PROGRESS study evaluating zilurgisertib in adult and pediatric patients aged 12 years and older with FOP. Efficacy was established based on total new HO lesion volume. Total new HO lesion volume includes expansion of baseline HO lesion burden as well as any new discrete HO that developed during the 24-week double-blind period. At Week 24, mean total new HO lesion volume decreased by 3.2 cm 3 in patients receiving zilurgisertib compared with an increase of 24.6 cm 3 in placebo-treated patients. Treatment effects were maintained through Week 48 of the open-label extension. Zilurgisertib was generally well tolerated during the 24-week placebo-controlled period of the study. The most common adverse reactions were headache, arthralgia, upper respiratory tract infection, epistaxis and nausea. Most adverse events were mild or moderate in severity, and no adverse events led to treatment discontinuation or dose reduction. Atebrioz will be available through Mirum Access Plus (MAP), a patient support program designed to help patients, families and healthcare providers navigate treatment access. MAP provides insurance coverage and access support, financial assistance for eligible patients, personalized patient support and educational resources for patients and caregivers. Atebrioz is expected to be commercially available in the U.S. in October, with eligible patients paying as little as $0 per month through MAP. To learn more about MAP, call 855-MRM-4YOU (1-855-676-4968). With this approval, the FDA also issued a Rare Pediatric Disease Priority Review Voucher (PRV) to Incyte. The voucher can be used for a subsequent drug application that would not otherwise qualify for a priority review. In the European Union, a marketing authorization application (MAA) for zilurgisertib is currently under review by the European Medicines Agency (EMA), supported by data from Cohort 1 (patients aged 12 years and older) of the PROGRESS study. The PROGRESS development program also continues to advance, with enrollment completed in Cohort 2 of children aged 6 to <12 years, and enrollment underway in Cohort 3 of children aged 2 to <12 years. About Atebrioz™ (zilurgisertib) tablets Atebrioz™ (zilurgisertib) tablets are a once-daily oral activin receptor-like kinase 2 (ALK2) inhibitor approved by the U.S. Food and Drug Administration (FDA) to reduce the volume of total new heterotopic ossification in adult and pediatric patients aged 12 years and older with Fibrodysplasia Ossificans Progressiva (FOP). In people living with FOP, pathogenic variants in the ACVR1 gene result in abnormal activation of ALK2, leading to the formation of bone in muscles, tendons, ligaments and other soft tissues through a process known as heterotopic ossification (HO). Mirum Pharmaceuticals, Inc. licensed zilurgisertib from Incyte for worldwide development and commercialization. IMPORTANT SAFETY INFORMATION Atebrioz can cause fetal harm based on data from animal studies. Patients of reproductive potential should use effective contraception and should immediately discontinue Atebrioz and contact their healthcare provider if pregnancy occurs. US Prescribing Information 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 63 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. Results from Cohort 1 have been reported through Week 48. The PROGRESS pediatric development program is evaluating the safety and efficacy of zilurgisertib in younger patients with FOP, including patients aged 6 to <12 years in Cohort 2 and patients aged 2 to <12 years in Cohort 3. About Fibrodysplasia Ossificans Progressiva (FOP) Fibrodysplasia ossificans progressiva (FOP) is an ultra-rare, progressive genetic disease affecting approximately 300 people in the United States and 900 worldwide. FOP is characterized by heterotopic ossification (HO), a process in which bone forms in muscles, tendons, ligaments and other soft tissues. Symptoms typically become apparent in early childhood and the number and volume of HO lesions increase over time, progressively restricting movement and limiting mobility, daily function, and independence. 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), Atebrioz™ (zilurgisertib) for fibrodysplasia ossificans progressiva (FOP), 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) 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, Mirum’s continued advancement of zilurgisertib with Incyte, expectations regarding the timing or results of clinical trials for zilurgisertib, the potential benefit of zilurgisertib in real world settings versus clinical trial settings, the importance of an additional therapy for the treatment of FOP, and the expected commercial availability of Atebrioz™, including the expected timing, cost to patients and methods of availability. 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 whether and when Atebrioz will become available as a treatment option for patients with FOP; the potential offered by Atebrioz for patients with FOP; expectations regarding the cost of Atebrioz for eligible patients; expectations regarding ongoing and future clinical trials for zilurgisertib, including the PROGRESS development program; expectations regarding the regulatory review of the MAA for zilurgisertib by the EMA; 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 June 30, 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 ResultDrug ApprovalPhase 2Priority Review

28 Sep 2026

·firstwordpharma.com

Mirum tees up filing for hepatitis delta hopeful after Phase III win

Mirum Pharmaceuticals said Monday that brelovitug passed a key test on its way to become a new treatment to patients with chronic hepatitis delta virus (HDV), with the antibody meeting the primary endpoint in the Phase III portion of AZURE-1. The findings put Mirum a step closer to a potential US filing."These results were achieved in a broad patient population, including those with significant liver inflammation, cirrhosis and clinically significant portal hypertension," said Nancy Shulman, EVP of clinical development at the company. "We look forward to reporting topline results from AZURE-4 later this year and advancing toward our planned BLA submission in the first half of 2027."The Phase III portion of AZURE-1 enrolled 153 treatment-naive patients who were randomised to a self-administered, 300-mg subcutaneous brelovitug injection once weekly, 900 mg once every four weeks, or delayed treatment starting at week 24.According to the company, 56% of patients in the weekly arm and 45% in the monthly arm achieved the primary endpoint, which required both a virologic response and ALT normalisation at week 24, compared to 0% in the delayed-treatment group (p<0.0001 for both comparisons). Virologic response — defined as undetectable HDV RNA or decline in HDV RNA ≥2 log10 IU/mL — was seen in 86% and 85% of patients in the two brelovitug groups, respectively, while ALT normalised in 63% and 54%.There were no serious adverse events (AEs) in either treatment group, nor any drop-outs as a result of AEs. Mirum said the drug was well tolerated in both dose arms.Deepening responsesMeanwhile, Mirum also reported longer-term data from the 53-patient Phase IIb portion of AZURE-1, with responses generally deepening over time. In the 300-mg weekly group, the proportion achieving the combined endpoint rose from 45% at week 24 to 55% at week 48, while the proportion with HDV RNA below 10 IU/mL went from 45% to 80% at those two timepoints.Among those receiving 900 mg every four weeks, the combined response rose from 35% to 55%, while HDV RNA below 10 IU/mL increased from 10% to 40%.ALT normalisation increased from 45% to 55% in the 300-mg weekly group and from 40% to 55% in the 900-mg every-four-week group between weeks 24 and 48."Seeing virologic response alongside ALT normalisation is encouraging in a long-term therapy," commented Norah Terrault, an AZURE-1 investigator. "For patients facing the most aggressive form of viral hepatitis, these are important results."Monday's readout comes on the heels of an FDA approval for Mirum just last week. On Friday, US regulators cleared its oral drug Atebrioz (zilurgisertib) for fibrodysplasia ossificans progressiva.Upcoming Vir readoutsCommenting on the AZURE-1 results, Stifel analysts said Mirum will face competition from Vir Biotechnology, which is expected to report Phase III data for a combination regimen consisting of investigational siRNA elebsiran with mAb tobevibart. Topline data from ECLIPSE 1 are due in the fourth quarter, followed by results from ECLIPSE 2 and ECLIPSE 3 in early 2027. Vir's regimen is administered once monthly as two separate sequential subcutaneous injections.Vir has said 96-week data from the Phase II SOLSTICE study data showed that 88% (28/32) of patients receiving the elebsiran/tobevibart combo achieved undetectable HDV RNA, compared to 53% (17/32) of those on tobevibart monotherapy.Meanwhile, Gilead Sciences' Hepcludex (bulevirtide-gmod) became the first treatment greenlit in the US for chronic HDV infection, after the FDA granted it accelerated approval earlier this year. However, a poll conducted by FirstWord suggested a lack of disease awareness could be a hurdle to uptake (see – Physician Views Results: Hepcludex marks meaningful advance in HDV, but first it must find its people).

Clinical ResultPhase 3Drug ApprovalPhase 2

28 Sep 2026

·www.biospace.com

Mirum Pharmaceuticals Announces Primary Endpoint Met in Phase 3 AZURE-1 Study of Brelovitug in Chronic Hepatitis Delta Virus

– Phase 3 primary endpoint (virologic response and ALT normalization) achieved in both dose arms at Week 24 – 48-week data from Phase 2b portion of AZURE-1 demonstrate deepening viral suppression and increased rates of ALT normalization with longer-term treatment – Favorable safety and tolerability profiles observed; no new safety signals – Mirum to host conference call to discuss topline results today, September 28, at 8:30 a.m. ET FOSTER CITY, Calif.--(BUSINESS WIRE)--Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today announced the primary endpoint was met in the Phase 3 portion of the AZURE-1 study evaluating brelovitug, an investigational fully human monoclonal antibody that binds to hepatitis B surface antigen (HBsAg), for the treatment of chronic hepatitis delta virus (HDV). In addition, 48-week data from the Phase 2b portion of AZURE-1 demonstrated deepening viral suppression, with a greater proportion of patients achieving HDV RNA below the lower limit of quantification (LLOQ, <10 IU/mL) and target not detected (TND), and increased rates of alanine aminotransferase (ALT) normalization. AZURE-1 is one of two pivotal Phase 3 studies intended to form the basis of Mirum’s U.S. registration package for brelovitug. “These results demonstrate the potential of brelovitug as a well-tolerated, convenient, single agent to deliver both deep viral suppression and ALT normalization, with responses that continue to deepen through 48 weeks of treatment. Notably, these results were achieved in a broad patient population, including those with significant liver inflammation, cirrhosis and clinically significant portal hypertension,” said Nancy Shulman, M.D., Executive Vice President of Clinical Development at Mirum. “We look forward to reporting topline results from AZURE-4 later this year and advancing toward our planned BLA submission in the first half of 2027.” “The goal of treating chronic HDV is to prevent progression to cirrhosis, liver cancer and liver failure, and that requires controlling both the virus and liver inflammation,” said Norah Terrault, M.D., MPH, Professor of Medicine and Chief of the Division of GI and Liver at the Keck School of Medicine of USC, and an AZURE-1 investigator. “ALT is a marker of liver injury, so seeing virologic response alongside ALT normalization is encouraging in a long-term therapy. For patients facing the most aggressive form of viral hepatitis, these are important results.” “Chronic hepatitis delta is a lifelong disease, and people living with it need treatment options that are effective, safe and tolerable enough to stay on over time. That’s especially true for patients with more advanced disease, who often have the fewest choices,” said Chari A. Cohen, DrPH, MPH, President of the Hepatitis B Foundation. “Results like these bring hope to a community that has had few options until recently.” Phase 3 AZURE-1 Results: Brelovitug Met the Primary Endpoint at Week 24 The Phase 3 portion of the AZURE-1 study enrolled 153 treatment-naive patients randomized in a 2:2:1 ratio to receive brelovitug 300 mg self-administered once weekly (QW) by subcutaneous (SC) injection, brelovitug 900 mg administered once every four weeks (Q4W) by SC injection, or delayed treatment starting at Week 24. This global study enrolled a broad patient population including patients with advanced disease. At Week 24, treatment with brelovitug met the primary endpoint, with 56% of patients in the 300 mg QW arm and 45% in the 900 mg Q4W arm achieving the combined endpoint of virologic response (≥2 log10 reduction in HDV RNA from baseline or undetectable HDV RNA [

Clinical ResultPhase 2Breakthrough TherapyPhase 3Orphan Drug

100 Deals associated with Zilurgisertib

External Link

KEGG	Wiki	ATC	Drug Bank
D12547 D12548	-	-	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Myositis Ossificans	United States	Incyte Corp.	25 Sep 2026

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Ossification, Heterotopic	Phase 2	United States	Incyte Corp.	31 Jan 2022
Ossification, Heterotopic	Phase 2	United States	Incyte Biosciences Shanghai Co., Ltd.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Australia	Incyte Biosciences Shanghai Co., Ltd.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Australia	Incyte Corp.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Brazil	Incyte Corp.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Brazil	Incyte Biosciences Shanghai Co., Ltd.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Canada	Incyte Corp.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Canada	Incyte Biosciences Shanghai Co., Ltd.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Chile	Incyte Corp.	31 Jan 2022
Ossification, Heterotopic	Phase 2	Chile	Incyte Biosciences Shanghai Co., Ltd.	31 Jan 2022

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT05090891 (PROGRESS, ENDO 12026 \| ENDO 22026)	Phase 2	Myositis Ossificans	63	Zilurgisertib 100 mg QD	nuyacshaex(khsogjmwcm) = opnsyvwphh vzaqmeajaa (fghsiqohku ) View more	Positive	14 Jun 2026
				Placebo	nuyacshaex(khsogjmwcm) = mwauqszqua vzaqmeajaa (fghsiqohku ) View more
NCT04455841 (INCB00928-104, ASH2025)	Phase 1/2	Anemia \| Myelofibrosis	84	Zilurgisertib alone (TGA)	hdsssaofza(isepeghumu) = oaprttczzz bycuxjxbyj (hjeizekreq ) View more	Negative	06 Dec 2025
				Zilurgisertib + ruxolitinib (TGB)	uharpifhsk(efshtaybln) = qblkgeujqt gdnoabjbjy (ohpxnprfst ) View more
NCT04582539 (LIMBER, CTgov)	Phase 1/2	Anemia \| Multiple Myeloma \| Myelodysplastic Syndromes	22	Zilurgisertib (Zilurgisertib 50 mg QD)	iiysekguol = fjsbheebsh bjbwkmimql (vtquprhvcz, byvhycjabs - dehpnewrmr) View more	-	22 Oct 2025
				Zilurgisertib (Zilurgisertib 100 mg QD)	iiysekguol = hnlpvnoein bjbwkmimql (vtquprhvcz, ajkqmkwqzf - bzfblyncrk) View more
NCT04455841 (INCB 00928-104, EHA2024) Manual	Phase 1/2	Anemia \| Myelofibrosis hepcidin	45	Zilurgisertibb alone	-	Positive	14 May 2024
				Zilurgisertibb wRuxolitinibinib	fkyptnwcvi(adkymyrjyo) = sdaekjskak muomddtntx (zhvmxwhveu ) View more
NCT04455841 (INCB 00928-104, ASH2023) Manual	Phase 1/2	Anemia	36	zilurgisertib (TGA)	ftytxazwnj(xtiwdjjvti) = No DLTs occurred in either treatment group. izirpqqmpb (vupnouxrvs ) View more	Positive	10 Dec 2023
				zilurgisertib+ruxolitinib (TGB)
NCT04455841 (ASCO 12023 \| ASCO 22023) Manual	Phase 1/2	Primary Myelofibrosis	31	zilurgisertib (TGA)	wynnzsagcw(rutlwqovml) = No dose-limiting toxicities (DLTs) or study drug-related serious adverse events (AEs) occurred in either treatment group. xjfitogcud (drzofmcemz ) View more	Positive	31 May 2023
				ruxolitinib+zilurgisertib (TGB)
NCT04455841 (INCB 00928-104, ASH 12022 \| ASH 22022) Manual	Phase 1/2	Primary Myelofibrosis	15	INCB000928	azwdljuzei(cqjankdiyb) = uifoapjlpg tiygqzbzzk (nkgxdcngqj ) View more	Positive	15 Nov 2022
				INCB000928+Ruxolitinib	azwdljuzei(cqjankdiyb) = iynjahesjp tiygqzbzzk (nkgxdcngqj ) View more

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