Mirum Pharmaceuticals (NASDAQ: MIRM) lifts 2026 sales outlook after $176M Q2
Rhea-AI Filing Summary
Mirum Pharmaceuticals reported Q2 2026 net product sales of $176.2 million, up from $127.8 million in Q2 2025, and raised 2026 net product sales guidance to $680 million to $700 million. LIVMARLI generated $128.7 million and bile acid medicines $47.5 million, with year-over-year growth of 46% and 20%, respectively.
The company recorded a Q2 net loss of $67,227 (in thousands) as total operating expenses rose to $218.8 million, including $16.4 million of non-recurring acquired in-process R&D associated with a zilurgisertib license and higher R&D and SG&A. Mirum issued $690.0 million of 0.00% convertible senior notes due 2032, settled $237.2 million of 4.00% notes due 2029, and ended June 30, 2026 with unrestricted cash, cash equivalents and investments of $561.3 million.
Pipeline highlights include FDA Breakthrough Therapy and Orphan Drug designations for volixibat in PSC, a pre-NDA meeting where the agency recommended conducting a Phase 3 study with further FDA discussions planned before a potential NDA submission targeted in the first half of 2027, completion of enrollment in the VANTAGE PBC study, and a PDUFA target action date of September 26, 2026 for zilurgisertib in FOP.
Positive
- Q2 2026 net product sales rose to $176.2 million, with LIVMARLI and bile acid medicines delivering 46% and 20% year-over-year growth and management raising 2026 net product sales guidance to $680–$700 million.
Negative
- Six-month net loss reached $857,382 (in thousands), driven largely by $742,737 (in thousands) of acquired in-process R&D expense, and stockholders’ equity shifted to a deficit of $10,422 at June 30, 2026 from $314,690 at December 31, 2025.
Filing Explained
At June 30, Mirum still reported $750,079 thousand of net convertible notes payable after settling about 75% of its 2029 notes.
The financing actions reported for the second quarter were completed: Mirum issued
The filing says the settlement covered approximately
At June 30, the balance sheet reported
This 8-K does not provide the notes’ conversion terms, potential share issuance, or use of proceeds, leaving common-holder dilution and financing use unresolved in this disclosure.
8-K Event Classification
2 items: 2.02, 9.01
2 items
Item 2.02
Results of Operations and Financial Condition
Financial
Disclosure of earnings results, typically an earnings press release or preliminary financials.
Item 9.01
Financial Statements and Exhibits
Exhibits
Financial statements, pro forma financial information, and exhibit attachments filed with this report.
Key Figures
Q2 2026 net product sales: $176.2 million
LIVMARLI Q2 2026 sales: $128.7 million
Bile Acid Medicines Q2 2026 sales: $47.5 million
+5 more
8 metrics
Q2 2026 net product sales
$176.2 million
Global net product sales for the quarter ended June 30, 2026, compared to $127.8 million in Q2 2025
LIVMARLI Q2 2026 sales
$128.7 million
Second quarter 2026 LIVMARLI net product sales, 46% growth over second quarter 2025
Bile Acid Medicines Q2 2026 sales
$47.5 million
Second quarter 2026 Bile Acid Medicines net product sales, 20% growth over second quarter 2025
Total operating expenses Q2 2026
$218.8 million
Total operating expenses for the quarter ended June 30, 2026
Non-recurring IPR&D expense Q2 2026
$16.4 million
Acquired in-process research and development expense tied to zilurgisertib license in Q2 2026
Q2 2026 net loss
$67,227 (in thousands)
Net loss for the quarter ended June 30, 2026
Unrestricted cash and investments
$561.3 million
Unrestricted cash, cash equivalents and investments as of June 30, 2026, versus $391.4 million at December 31, 2025
0.00% convertible notes issued
$690.0 million
Aggregate principal amount of convertible senior notes due 2032 issued in 2026
Key Terms
Breakthrough Therapy Designation, Orphan Drug Designation, acquired in-process research and development (IPR&D), convertible senior notes, +1 more
5 terms
Breakthrough Therapy Designation regulatory
"U.S. FDA granted volixibat Breakthrough Therapy Designation for the treatment of cholestatic pruritus due to PSC"
A breakthrough therapy designation is a regulatory fast-track given to a drug or treatment that shows early signs of providing a major improvement over existing options for a serious condition. Think of it as a VIP lane that can speed up development and more intensive guidance from regulators, which matters to investors because it can shorten time to market, reduce development risk and potentially increase a company’s value — though it does not guarantee approval.
Orphan Drug Designation regulatory
"U.S. FDA granted volixibat Breakthrough Therapy Designation and Orphan Drug Designation for PSC"
Orphan drug designation is a special status given to medicines developed to treat rare diseases affecting only a small number of people. This status often provides benefits like faster approval processes and financial incentives, making it more attractive for companies to develop these drugs. For investors, it signals potential for exclusive market rights and reduced competition, which can impact the drug’s profitability.
acquired in-process research and development (IPR&D) financial
"Non-recurring acquired in-process research and development (IPR&D) expense associated with the exclusive license of worldwide rights to zilurgisertib"
convertible senior notes financial
"Issued $690.0 million aggregate principal amount of 0.00% convertible senior notes due 2032"
Convertible senior notes are a type of loan that a company issues to investors, which can be turned into company shares later on. They are called "senior" because they are paid back before other debts if the company runs into trouble. This allows investors to earn interest like a loan but also have the chance to own part of the company if its value rises.
PDUFA target action date regulatory
"PDUFA target action date for the zilurgisertib NDA is September 26, 2026"
The PDUFA target action date is the deadline set by the U.S. Food and Drug Administration (FDA) by which it aims to decide whether to approve or reject a new drug application. This date helps investors gauge when a company’s new medication might reach the market, potentially influencing sales and revenue expectations. It acts as a key milestone signaling progress in the drug approval process.
Earnings Snapshot
Net product sales Q2 2026: $176.2 million · Guidance included
quarter ended June 30, 2026
Net product sales Q2 2026
$176.2 million
compared to $127.8 million in Q2 2025
Net loss Q2 2026
$67,227 (in thousands)
compared to $5,861 (in thousands) net loss in Q2 2025
Guidance
2026 net product sales guidance of $680 million to $700 million
AI-generated analysis. How Rhea-AI works. Not financial advice.
FAQ
What were Mirum Pharmaceuticals’ (MIRM) Q2 2026 net product sales?
Mirum reported $176.2 million in net product sales for Q2 2026, up from $127.8 million in Q2 2025. LIVMARLI contributed $128.7 million and bile acid medicines $47.5 million, reflecting 46% and 20% year-over-year growth, respectively.
What 2026 net product sales guidance did Mirum Pharmaceuticals (MIRM) provide?
The company increased its 2026 net product sales guidance to $680 million to $700 million. The higher outlook is supported by continued commercial momentum for LIVMARLI and bile acid medicines and expectations around upcoming product and indication launches.
Why did Mirum Pharmaceuticals (MIRM) post a large net loss in the first half of 2026?
Mirum reported a $857,382 net loss (in thousands) for the first half of 2026, primarily due to $742,737 (in thousands) of acquired in-process research and development expense, alongside higher research and development and selling, general and administrative costs.
What is the regulatory status of volixibat at Mirum Pharmaceuticals (MIRM)?
Volixibat has FDA Breakthrough Therapy and Orphan Drug designations for PSC-related cholestatic pruritus. A pre-NDA meeting was held; the FDA recommended a Phase 3 study, and Mirum plans further discussions ahead of a potential NDA submission targeted for the first half of 2027.
When is the FDA decision date for zilurgisertib at Mirum Pharmaceuticals (MIRM)?
Zilurgisertib’s NDA for fibrodysplasia ossificans progressiva is under Priority Review with a Prescription Drug User Fee Act (PDUFA) target action date of September 26, 2026, following pivotal Phase 2 PROGRESS study results supporting the submission.
What is Mirum Pharmaceuticals’ (MIRM) cash and debt position as of June 30, 2026?
As of June 30, 2026, Mirum held $561.3 million in unrestricted cash, cash equivalents and investments. During the period, it issued $690.0 million of 0.00% convertible senior notes due 2032 and settled $237.2 million of 4.00% notes due 2029.
UNITED STATES
SECURITIES AND EXCHANGE COMMISSION
WASHINGTON, D.C. 20549
____________________________________________________
FORM 8-K
____________________________________________________
CURRENT REPORT
Pursuant to Section 13 or 15(d) of the Securities Exchange Act of 1934
Date of Report (Date of earliest event reported): August 5, 2026
____________________________________________________
(Exact name of Registrant as Specified in Its Charter)
____________________________________________________
(State or Other Jurisdiction of Incorporation) | (Commission File Number) | (IRS Employer Identification No.) | ||||||
989 East Hillsdale Boulevard Suite 300 | ||||||||
| (Address of Principal Executive Offices) | (Zip Code) | |||||||
Registrant’s Telephone Number, Including Area Code: (650) 667-4085
N/A
(Former Name or Former Address, if Changed Since Last Report)
____________________________________________________
Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:
Securities registered pursuant to Section 12(b) of the Act:
| Title of each class | Trading Symbol(s) | Name of each exchange on which registered | ||||||||||||
Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b-2 of the Securities Exchange Act of 1934 (§ 240.12b-2 of this chapter).
Emerging growth company ☐
If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act. ☐
Item 2.02 Results of Operations and Financial Condition.
On August 5, 2026, Mirum Pharmaceuticals, Inc. (the “Company”) issued a press release providing a corporate update and announcing its financial results for the quarter ended June 30, 2026. The full text of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.
The information in this Current Report on Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.
Item 9.01 Financial Statements and Exhibits.
(d)Exhibits.
Exhibit No. | Description | |||||||
99.1 | Press Release dated August 5, 2026 | |||||||
| 104 | Cover Page Interactive Data File (embedded within the Inline XBRL document) | |||||||
SIGNATURES
Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned thereunto duly authorized.
| Mirum Pharmaceuticals, Inc. | ||||||||
Date: August 5, 2026 | By: | /s/ Christopher Peetz | ||||||
| Christopher Peetz | ||||||||
| Chief Executive Officer | ||||||||
Exhibit 99.1
Mirum Pharmaceuticals Reports Second Quarter 2026 Financial Results and Provides Business Update
–Q2 2026 net product sales of $176 million
–2026 net product sales guidance increased to $680 million to $700 million
–Volixibat granted Breakthrough Therapy and Orphan Drug Designations for cholestatic pruritus due to PSC
–Pre-NDA meeting held for volixibat in cholestatic pruritus due to PSC; additional discussions planned before potential NDA submission, now targeted in H1 2027
–Enrollment completed in VANTAGE study of volixibat in cholestatic pruritus due to PBC; data expected Q1 2027
–Conference call to provide business updates today, August 5 at 1:30 p.m. PT / 4:30 p.m. ET
FOSTER CITY, Calif. – August 5, 2026 – Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a leading rare disease company, today reported financial results for the second quarter 2026 and provided a business update.
“Mirum delivered another strong quarter, with continued commercial momentum supporting an increase to our full-year net product sales guidance and launch readiness for the upcoming potential approval of zilurgisertib,” said Chris Peetz, Chief Executive Officer of Mirum. “The FDA’s decision to grant volixibat Breakthrough Therapy and Orphan Drug Designations underscores the strength of the VISTAS efficacy data and the serious unmet need in PSC. VISTAS was designed with FDA input as a pivotal study and met its primary endpoint with highly significant results. However, at our recent pre-NDA meeting, the agency recommended conducting a Phase 3 study. We intend to hold further discussions with the FDA before a potential NDA submission in the first half of 2027 based on VISTAS.”
Q2 and Recent Highlights
Commercial: Raising Full Year Net Product Sales Guidance to $680 Million to $700 Million
•Second quarter 2026 global net product sales of $176.2 million.
•Second quarter 2026 LIVMARLI® net product sales were $128.7 million, representing 46% growth over second quarter 2025 net product sales.
•Second quarter 2026 Bile Acid Medicines net product sales were $47.5 million, representing 20% growth over second quarter 2025 net product sales.
Regulatory and Pipeline: Advancing Toward Multiple Milestones
•U.S. FDA granted volixibat Breakthrough Therapy Designation for the treatment of cholestatic pruritus due to primary sclerosing cholangitis (PSC) and Orphan Drug Designation for PSC.
•Participated in pre-NDA meeting with U.S. FDA for volixibat in cholestatic pruritus due to PSC; planning additional discussions with the FDA before potential NDA submission, now targeted in H1 2027.
•Brelovitug AZURE-1 and AZURE-4 Phase 3 studies in chronic hepatitis delta virus (HDV) topline results expected in Q3 and Q4 2026, respectively.
•LIVMARLI EXPAND Phase 3 study in cholestatic pruritus due to additional rare cholestatic conditions topline results expected in Q4 2026.
•Completed enrollment in the volixibat VANTAGE Phase 2b study in cholestatic pruritus due to primary biliary cholangitis (PBC); topline results expected in Q1 2027.
•Presented positive pivotal Phase 2 results from the PROGRESS study of zilurgisertib in fibrodysplasia ossificans progressiva (FOP) at ENDO 2026; Prescription Drug User Fee Act (PDUFA) target action date for the zilurgisertib NDA is September 26, 2026.
Corporate & Financial: Strong Balance Sheet and Financial Independence
•Total revenue for the quarter ended June 30, 2026 was $176.2 million compared to $127.8 million for the quarter ended June 30, 2025.
•Total operating expenses were $218.8 million for the quarter ended June 30, 2026 compared to $132.8 million for the quarter ended June 30, 2025. Total operating expenses for the quarter ended June 30, 2026 included:
◦Cost of sales of $23.2 million, excluding intangible amortization and stock-based compensation expense.
◦Non-recurring acquired in-process research and development (IPR&D) expense associated with the exclusive license of worldwide rights to zilurgisertib of $16.4 million.
◦Research and development expense of $76.3 million, including $28.8 million for the development of brelovitug, excluding stock-based compensation expense.
◦Selling, general, and administrative expense of $65.6 million, excluding stock-based compensation expense.
◦$37.3 million of stock-based compensation, intangible amortization, and other non-cash expenses.
•Issued $690.0 million aggregate principal amount of 0.00% convertible senior notes due 2032.
•Settled $237.2 million aggregate principal amount of 4.00% convertible senior notes due 2029, which represented approximately 75% of the then-outstanding notes.
•As of June 30, 2026, Mirum had unrestricted cash, cash equivalents, and investments of $561.3 million compared to $391.4 million as of December 31, 2025.
Business Update Conference Call
Mirum will host a conference call today, August 5 at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates. Join the call using the following details:
Conference Call Details:
US/Toll-Free: +1 833 461 5787
International: +1 585 542 9983
Access Code: 789239699
You may also access the call via webcast by visiting the Investors section of Mirum’s corporate website. The archived webcast will be available for replay.
About LIVMARLI® (maralixibat) oral solution and LIVMARLI® (maralixibat) tablets
LIVMARLI® (maralixibat) is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the U.S. Food and Drug Administration for two pediatric cholestatic liver diseases. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) in the U.S. three months of age and older and in Europe for patients two months of age and older. It is also approved in the U.S. for the treatment of cholestatic pruritus in patients with progressive familial intrahepatic cholestasis (PFIC) 12 months of age and older and in Europe for the treatment of PFIC in patients three months of age and older. For more information for U.S. residents, please visit LIVMARLI.com.
LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for the treatment of ALGS and PFIC. LIVMARLI is currently being evaluated in the Phase 3 EXPAND study in additional settings of cholestatic pruritus. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
Limitation of Use: LIVMARLI is not for use in PFIC type 2 patients who have a severe defect in the bile salt export pump (BSEP) protein.
LIVMARLI can cause side effects, including:
Liver injury. Changes in certain liver tests are common in patients with ALGS and PFIC but can worsen during treatment. These changes may be a sign of liver injury. In PFIC, this can be serious or may lead to liver transplant or death. Your healthcare provider should do blood tests and physical exams before starting and during treatment to check your liver function. Tell your healthcare provider right away if you get any signs or symptoms of liver problems, including nausea or vomiting, skin or the white part of the eye turns yellow, dark or brown urine, pain on the right side of the stomach (abdomen), bloating in your stomach area, loss of appetite or bleeding or bruising more easily than normal.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea and stomach pain. Your healthcare provider may advise you to monitor for new or worsening stomach problems including stomach pain, diarrhea, blood in your stool or vomiting. Tell your healthcare provider right away if you have any of these symptoms more often or more severely than normal for you.
A condition called Fat Soluble Vitamin (FSV) Deficiency caused by low levels of certain vitamins (vitamin A, D, E, and K) stored in body fat is common in patients with ALGS and PFIC but may worsen during treatment. Your healthcare
provider should do blood tests before starting and during treatment and may monitor for bone fractures and bleeding which have been reported as common side effects.
US Prescribing Information
EU SmPC
Canadian Product Monograph
About CHOLBAM® (cholic acid) capsules
The FDA approved CHOLBAM® (cholic acid) capsules in March 2015, the first FDA-approved treatment for pediatric and adult patients with bile acid synthesis disorders due to single enzyme defects, and for adjunctive treatment of patients with peroxisome biogenesis disorder-Zellweger spectrum disorder. The effectiveness of CHOLBAM has been demonstrated in clinical trials for bile acid synthesis disorders and the adjunctive treatment of peroxisomal disorders. An estimated 200 to 300 patients are current candidates for therapy.
CHOLBAM (cholic acid) Indication
CHOLBAM is a bile acid indicated for
◦Treatment of bile acid synthesis disorders due to single enzyme defects.
◦Adjunctive treatment of peroxisomal disorders, including Zellweger spectrum disorders, in patients who exhibit manifestations of liver disease, steatorrhea, or complications from decreased fat-soluble vitamin absorption.
LIMITATIONS OF USE
The safety and effectiveness of CHOLBAM on extrahepatic manifestations of bile acid synthesis disorders due to single enzyme defects or peroxisomal disorders, including Zellweger spectrum disorders, have not been established.
IMPORTANT SAFETY INFORMATION
WARNINGS AND PRECAUTIONS – Exacerbation of liver impairment
Monitor liver function and discontinue CHOLBAM in patients who develop worsening of liver function while on treatment.
Concurrent elevations of serum gamma glutamyltransferase (GGT) and alanine aminotransferase (ALT) may indicate CHOLBAM overdose.
Discontinue treatment with CHOLBAM at any time if there are clinical or laboratory indicators of worsening liver function or cholestasis.
ADVERSE REACTIONS
The most common adverse reactions (≥1%) are diarrhea, reflux esophagitis, malaise, jaundice, skin lesion, nausea, abdominal pain, intestinal polyp, urinary tract infection, and peripheral neuropathy.
Please see full Prescribing Information for additional Important Safety Information.
About CTEXLI® (chenodiol) tablets
CTEXLI® (chenodiol) tablets is FDA-approved for the treatment of adults with cerebrotendinous xanthomatosis (CTX). Chenodiol is another name for chenodeoxycholic acid (CDCA). CDCA is a naturally occurring bile acid that was originally approved for the treatment of people with radiolucent stones in the gallbladder. CTEXLI was evaluated as part of the Phase 3 RESTORE study, the first and only clinical trial for CTX. CTX is a rare progressive disease that can affect the brain, spinal cord, tendons, eyes and arteries.
IMPORTANT SAFETY INFORMATION
CTEXLI can cause side effects, including:
Liver Injury: You will need to undergo laboratory testing before starting and while taking CTEXLI to check your liver function. Changes in certain liver tests may occur during treatment and may be a sign of liver injury. This can be serious. Stop taking CTEXLI immediately and tell your healthcare provider right away if you get any signs or symptoms of liver problems, including, stomach (abdomen) pain, bruising, dark-colored urine, feeling tired (fatigue), bleeding, yellowing of the skin and eyes, nausea, and itching.
Most Common Side Effects: Diarrhea, headache, stomach pain, constipation, high blood pressure, muscular weakness, and upper respiratory tract infection.
Tell your healthcare provider about all the medications that you take, as CTEXLI may interact with other medicines.
US Prescribing Information
About Volixibat
Volixibat is an investigational oral, minimally absorbed agent designed to selectively inhibit the ileal bile acid transporter (IBAT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of IBAT, thereby reducing bile acids systemically and in the liver. Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (PSC) (VISTAS study) and primary biliary cholangitis (PBC) (VANTAGE study).
In 2026, Mirum shared that the Phase 2b VISTAS study of volixibat in PSC met its primary endpoint, with statistically significant and clinically meaningful reductions in pruritus observed in patients treated with volixibat. Volixibat’s safety profile in the study was generally consistent with the known effects of IBAT inhibition. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of cholestatic pruritus due to PSC.
In 2024, Mirum announced positive interim results from the Phase 2b VANTAGE study of volixibat in PBC. No new safety signals were observed in the study. Volixibat has been granted FDA Breakthrough Therapy designation for the treatment of cholestatic pruritus due to PBC.
About Brelovitug
Brelovitug is an investigational, highly potent, pan-genotypic, fully human immunoglobulin G1 (IgG1) monoclonal antibody (mAb) that targets the surface antigen (anti-HBsAg) on both the hepatitis delta virus (HDV) and the hepatitis B virus (HBV). Brelovitug is designed to neutralize and remove hepatitis B and hepatitis D virions and deplete HBsAg-containing subviral particles. Brelovitug has FDA Breakthrough Therapy designation for the treatment of chronic HDV infection and PRIME and Orphan designations from the European Medicines Agency.
In 2026, Mirum announced that in the Phase 2b portion of the AZURE-1 study in HDV, treatment with brelovitug demonstrated strong antiviral activity in HDV and achieved the primary composite endpoint of virologic response and alanine aminotransferase (ALT) normalization at Week 24 in both brelovitug dose arms as compared to the delayed treatment arm. Favorable safety and tolerability profiles were observed. Brelovitug is currently being evaluated in the global Phase 3 AZURE clinical program. Mirum owns worldwide rights to brelovitug.
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 MRM-3379
MRM-3379 is an in-licensed investigational oral therapy being evaluated for the treatment of Fragile X syndrome (FXS). It is a selective phosphodiesterase-4D (PDE4D) inhibitor designed to enhance cAMP signaling. MRM-3379 may offer a novel approach to improving cognition, language, and daily function in individuals with FXS. MRM-3379 has been granted FDA Fast Track designation for the treatment of FXS.
The BLOOM Phase 2 clinical study of MRM-3379 is currently underway in FXS. Males ages 16 to 45 will be randomly assigned to receive one of three dose levels of MRM-3379 or placebo for 12 weeks. An open-label cohort of boys ages 13 to 16 will receive the lowest dose, in order to explore effects of treatment in younger boys, closer to the age of diagnosis. The study’s primary endpoint is safety and tolerability, the key secondary endpoint is the NIH Toolbox Crystallized Cognition Composite (CCC), and several exploratory endpoints will assess potential effects on mood, behavior, and other symptoms that are relevant to this population. Mirum owns worldwide rights to MRM-3379.
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 www.mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and X.
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, commercial results for our approved products, including continued growth in year-over-year net product sales, achievement of our 2026 financial guidance, our anticipated successes in 2026, including continued commercial momentum, the results, enrollment, conduct and progress of our ongoing and planned studies for our product candidates, including the timing and results of interim and topline analyses of our ongoing studies, the occurrence, timing and results of our discussions with the FDA regarding volixibat, potential submission and approval of NDA filings, the timing of any submissions and approvals of NDA filings and the potential commercial launch of our product candidates. 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 “anticipate,” “expected,” “will,” “could,” “would,” “guidance,” “target,”“intend,”“plan,” “potential” 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, risks and uncertainties associated with pharmaceutical development and commercialization in general, the impact of geopolitical and macroeconomic events, and the other risks described in Mirum’s Annual Report on Form 10-K for the year ended December 31, 2025 and subsequent filings with the Securities and Exchange Commission. 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.
Mirum and the Mirum logo are trademarks of Mirum Pharmaceuticals, Inc.
Mirum Pharmaceuticals, Inc.
Condensed Consolidated Statement of Operations Data
(in thousands, except share and per share amounts)
(Unaudited)
| Three Months Ended June 30, | Six Months Ended June 30, | ||||||||||||||||||||||
| 2026 | 2025 | 2026 | 2025 | ||||||||||||||||||||
| Revenue: | |||||||||||||||||||||||
| Product sales, net | $ | 176,243 | $ | 127,785 | $ | 336,125 | $ | 239,370 | |||||||||||||||
| Operating expenses: | |||||||||||||||||||||||
| Cost of sales (1) | 30,348 | 23,421 | 59,153 | 46,439 | |||||||||||||||||||
| Research and development | 90,528 | 46,067 | 188,438 | 87,111 | |||||||||||||||||||
| Acquired in-process research and development | 16,435 | — | 742,737 | 5,000 | |||||||||||||||||||
| Selling, general and administrative | 81,469 | 63,286 | 177,799 | 120,992 | |||||||||||||||||||
| Total operating expenses (2) | 218,780 | 132,774 | 1,168,127 | 259,542 | |||||||||||||||||||
| Loss from operations | (42,537) | (4,989) | (832,002) | (20,172) | |||||||||||||||||||
| Other income (expense): | |||||||||||||||||||||||
| Interest income | 3,851 | 3,033 | 7,252 | 6,056 | |||||||||||||||||||
| Interest expense | (2,618) | (3,589) | (6,234) | (7,185) | |||||||||||||||||||
| Other (expense) income, net | (24,234) | 86 | (24,234) | 2,194 | |||||||||||||||||||
| Net loss before provision for income taxes | (65,538) | (5,459) | (855,218) | (19,107) | |||||||||||||||||||
| Provision for income taxes | 1,689 | 402 | 2,164 | 1,431 | |||||||||||||||||||
| Net loss | $ | (67,227) | $ | (5,861) | $ | (857,382) | $ | (20,538) | |||||||||||||||
| Net loss per share, basic and diluted | $ | (1.06) | $ | (0.12) | $ | (14.03) | $ | (0.42) | |||||||||||||||
| Weighted-average shares of common stock used to compute net loss per share, basic and diluted | 63,374,183 | 49,726,823 | 61,125,097 | 49,310,255 | |||||||||||||||||||
| (1) Amounts include intangible amortization expense as follows: | |||||||||||||||||||||||
| Intangible amortization | $ | 6,685 | $ | 5,894 | $ | 13,370 | $ | 11,788 | |||||||||||||||
| (2) Amounts include stock-based compensation expense as follows: | |||||||||||||||||||||||
| Cost of sales | $ | 461 | $ | 238 | $ | 809 | $ | 569 | |||||||||||||||
| Research and development | 14,227 | 7,624 | 38,416 | 12,853 | |||||||||||||||||||
| Selling, general and administrative | 15,887 | 10,606 | 48,824 | 20,849 | |||||||||||||||||||
| Total stock-based compensation | $ | 30,575 | $ | 18,468 | $ | 88,049 | $ | 34,271 | |||||||||||||||
Mirum Pharmaceuticals, Inc.
Condensed Consolidated Balance Sheet Data
(in thousands)
(Unaudited)
| June 30, 2026 | December 31, 2025 | ||||||||||
| Assets | |||||||||||
| Current assets: | |||||||||||
| Cash and cash equivalents | $ | 330,728 | $ | 296,683 | |||||||
| Short-term investments | 107,563 | 86,644 | |||||||||
| Accounts receivable | 145,360 | 123,330 | |||||||||
| Inventory | 25,769 | 24,887 | |||||||||
| Prepaid expenses and other current assets | 26,890 | 18,140 | |||||||||
| Total current assets | 636,310 | 549,684 | |||||||||
| Restricted cash | 1,734 | 1,482 | |||||||||
| Long-term investments | 123,030 | 8,105 | |||||||||
| Intangible assets, net | 247,388 | 260,921 | |||||||||
| Other noncurrent assets | 36,519 | 22,621 | |||||||||
| Total assets | $ | 1,044,981 | $ | 842,813 | |||||||
| Liabilities and Stockholders’ (Deficit) Equity | |||||||||||
| Current liabilities: | |||||||||||
| Accounts payable | $ | 23,427 | $ | 9,614 | |||||||
| Holdback liabilities, current | 24,800 | — | |||||||||
| Accrued expenses and other current liabilities | 174,561 | 196,185 | |||||||||
| Total current liabilities | 222,788 | 205,799 | |||||||||
| Operating lease liabilities, noncurrent | 10,465 | 7,516 | |||||||||
| Convertible notes payable, net, noncurrent | 750,079 | 309,797 | |||||||||
| Holdback liabilities, noncurrent | 61,154 | — | |||||||||
| Other liabilities | 10,917 | 5,011 | |||||||||
| Total liabilities | 1,055,403 | 528,123 | |||||||||
| Commitments and contingencies | |||||||||||
| Stockholders’ (deficit) equity: | |||||||||||
| Preferred stock | — | — | |||||||||
| Common stock | 6 | 5 | |||||||||
| Additional paid-in capital | 1,515,172 | 981,878 | |||||||||
| Accumulated deficit | (1,524,926) | (667,544) | |||||||||
| Accumulated other comprehensive (loss) income | (674) | 351 | |||||||||
| Total stockholders’ (deficit) equity | (10,422) | 314,690 | |||||||||
| Total liabilities and stockholders’ (deficit) equity | $ | 1,044,981 | $ | 842,813 | |||||||
Contacts
Investor Contact:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Meredith Kiernan
media@mirumpharma.com