Mirum Pharmaceuticals Announces Preliminary Unaudited 2024 Net Product Sales and Cash Balance and Provides Corporate Updates
Mirum Pharmaceuticals (NASDAQ: MIRM) reported preliminary unaudited 2024 net product sales of approximately $336 million, exceeding their guidance. The company projects 2025 global net product sales between $420-435 million. LIVMARLI contributed approximately $213 million in 2024 sales, while CHOLBAM and CHENODAL generated approximately $123 million.
Key 2024 achievements include FDA approval for LIVMARLI in PFIC patients, positive interim results for volixibat in PSC and PBC studies, and expansion to 30 countries with commercial access. The company maintained financial strength with approximately $287 million in cash and investments as of December 31, 2024, achieving positive cash flow from operations in Q3 2024.
Looking ahead, Mirum expects to complete enrollment for the VISTAS study in PSC in second half 2025, with topline data in 2026. The FDA PDUFA date for chenodiol in CTX is set for March 28, 2025.
Mirum Pharmaceuticals (NASDAQ: MIRM) ha riportato vendite di prodotto nette preliminari non verificate per il 2024 pari a circa 336 milioni di dollari, superando le loro previsioni. L'azienda prevede vendite globali di prodotto netto per il 2025 tra 420 e 435 milioni di dollari. LIVMARLI ha contribuito per circa 213 milioni di dollari nelle vendite del 2024, mentre CHOLBAM e CHENODAL hanno generato circa 123 milioni di dollari.
Le principali realizzazioni del 2024 includono l'approvazione della FDA per LIVMARLI nei pazienti con PFIC, risultati intermedi positivi per volixibat negli studi PSC e PBC, e l'espansione in 30 paesi con accesso commerciale. L'impresa ha mantenuto una solidità finanziaria con circa 287 milioni di dollari in contante e investimenti al 31 dicembre 2024, raggiungendo un flusso di cassa positivo dalle operazioni nel terzo trimestre del 2024.
Guardando al futuro, Mirum prevede di completare l'arruolamento per lo studio VISTAS in PSC nella seconda metà del 2025, con i dati preliminari nel 2026. La data PDUFA della FDA per chenodiolo in CTX è fissata per il 28 marzo 2025.
Mirum Pharmaceuticals (NASDAQ: MIRM) reportó ventas netas de productos preliminares no auditadas para 2024 de aproximadamente 336 millones de dólares, superando sus expectativas. La compañía proyecta ventas netas de productos globales para 2025 entre 420 y 435 millones de dólares. LIVMARLI contribuyó con aproximadamente 213 millones en ventas de 2024, mientras que CHOLBAM y CHENODAL generaron aproximadamente 123 millones de dólares.
Los logros clave de 2024 incluyen la aprobación de la FDA para LIVMARLI en pacientes con PFIC, resultados interinos positivos para volixibat en estudios de PSC y PBC, y expansión a 30 países con acceso comercial. La empresa mantuvo fortaleza financiera con aproximadamente 287 millones de dólares en efectivo e inversiones al 31 de diciembre de 2024, logrando un flujo de caja positivo de operaciones en el tercer trimestre de 2024.
Mirum espera completar la inscripción para el estudio VISTAS en PSC en la segunda mitad de 2025, con datos preliminares en 2026. La fecha PDUFA de la FDA para chenodiol en CTX está programada para el 28 de marzo de 2025.
Mirum Pharmaceuticals (NASDAQ: MIRM)는 2024년 비감사된 잠정 순제품 매출이 약 3억 3천6백만 달러에 달해 가이던스를 초과했다고 보고했습니다. 이 회사는 2025년 전세계 순제품 매출이 4억 2천만에서 4억 3천5백만 달러 사이가 될 것이라고 예상하고 있습니다. LIVMARLI는 2024년에 약 2억 1천3백만 달러의 매출에 기여했으며, CHOLBAM과 CHENODAL은 약 1억 2천3백만 달러를 생성했습니다.
2024년의 주요 성과로는 PFIC 환자에 대한 LIVMARLI의 FDA 승인, PSC 및 PBC 연구에서의 volixibat의 긍정적인 중간 결과, 그리고 상업적 접근이 가능한 30개국으로의 확장이 있습니다. 회사는 2024년 12월 31일 기준으로 약 2억 8천7백만 달러의 현금 및 투자로 금융 건전성을 유지했으며, 2024년 3분기 운영에서 긍정적인 현금 흐름을 달성했습니다.
앞으로 Mirum은 2025년 하반기에 PSC에 대한 VISTAS 연구의 등록을 완료할 것으로 예상하며, 2026년에는 주요 데이터를 발표할 계획입니다.chenodiol의 FDA PDUFA 날짜는 2025년 3월 28일로 설정되어 있습니다.
Mirum Pharmaceuticals (NASDAQ: MIRM) a annoncé des ventes nettes de produits préliminaires non auditées pour 2024 d'environ 336 millions de dollars, dépassant ainsi ses prévisions. La société prévoit des ventes nettes de produits mondiaux comprises entre 420 et 435 millions de dollars pour 2025. LIVMARLI a contribué à environ 213 millions de dollars de ventes en 2024, tandis que CHOLBAM et CHENODAL ont généré environ 123 millions de dollars.
Les réalisations clés de 2024 comprennent l'approbation de la FDA pour LIVMARLI chez les patients atteints de PFIC, des résultats intermédiaires positifs pour volixibat dans les études PSC et PBC, et une expansion dans 30 pays avec un accès commercial. L'entreprise a maintenu sa solidité financière avec environ 287 millions de dollars en liquidités et en investissements au 31 décembre 2024, atteignant un flux de trésorerie positif provenant des opérations au troisième trimestre 2024.
Dans les perspectives d'avenir, Mirum prévoit de terminer l'inscription pour l'étude VISTAS dans la PSC dans la seconde moitié de 2025, avec des données préliminaires en 2026. La date PDUFA de la FDA pour le chenodiol dans le CTX est fixée au 28 mars 2025.
Mirum Pharmaceuticals (NASDAQ: MIRM) berichtete über vorläufige, nicht testierte Netto-Produktverkäufe im Jahr 2024 von etwa 336 Millionen US-Dollar, was ihre Prognose übertrifft. Das Unternehmen erwartet für 2025 globale Netto-Produktverkäufe zwischen 420 und 435 Millionen US-Dollar. LIVMARLI trug im Jahr 2024 mit etwa 213 Millionen US-Dollar zu den Verkäufen bei, während CHOLBAM und CHENODAL etwa 123 Millionen US-Dollar generierten.
Wichtige Erfolge im Jahr 2024 umfassen die FDA-Zulassung für LIVMARLI bei PFIC-Patienten, positive Zwischenresultate für volixibat in PSC- und PBC-Studien sowie die Expansion in 30 Länder mit kommerziellem Zugang. Das Unternehmen hielt eine finanzielle Stabilität mit etwa 287 Millionen US-Dollar in bar und Investitionen zum 31. Dezember 2024 aufrecht und erzielte im dritten Quartal 2024 einen positiven Cashflow aus dem operativen Geschäft.
Blick in die Zukunft erwartet Mirum den Abschluss der Rekrutierung für die VISTAS-Studie bei PSC in der zweiten Hälfte von 2025, mit Topline-Daten im Jahr 2026. Das FDA PDUFA-Datum für Chenodiol bei CTX ist auf den 28. März 2025 festgesetzt.
- Net product sales of $336M in 2024, exceeding guidance
- Projected 2025 sales growth to $420-435M
- LIVMARLI sales reached $213M in 2024
- Positive cash flow from operations achieved in Q3 2024
- Cash position maintained at $287M
- Commercial expansion to 30 countries
- FDA and European approvals for LIVMARLI in PFIC
- None.
Insights
The preliminary 2024 results showcase exceptional commercial execution, with net product sales of
The 2025 guidance of
The expansion to 30 countries with successful reimbursement in major European markets significantly enlarges the addressable market. This geographic diversification, combined with multiple catalysts including the chenodiol PDUFA date and potential label expansions, creates multiple revenue growth vectors.
The clinical development pipeline shows strategic depth across multiple rare diseases. The VISTAS study in PSC and VANTAGE study in PBC position volixibat as a potential breakthrough therapy, particularly noteworthy given the FDA's breakthrough therapy designation for PBC. The positive interim analyses in both indications suggest promising efficacy.
LIVMARLI's label expansion for PFIC patients and the EXPAND study initiative demonstrate a methodical approach to market expansion. The acquisition of MRM-3379 for Fragile X Syndrome represents a strategic entry into rare neurological disorders, diversifying beyond liver diseases.
The combination of commercial products and late-stage pipeline assets creates a balanced portfolio in rare diseases. The focus on both ultra-rare conditions and larger orphan indications provides multiple growth opportunities while maintaining pricing power in niche markets.
- 2024 net product sales of approximately
- 2025 expected global net product sales of
- VISTAS study of volixibat in primary sclerosing cholangitis expected to complete enrollment in second half 2025; topline data expected 2026
“2024 marked a significant year for Mirum as we accelerated our commercial business and achieved significant development milestones,” said Chris Peetz, chief executive officer of Mirum. “We continued our leadership in cholestatic disease with a label expansion for LIVMARLI and positive interim analyses for volixibat in PSC and PBC. Additionally, we advanced our rare genetic neurology efforts with the NDA submission for chenodiol and the in-licensing of MRM-3379 for Fragile X syndrome. With proven commercial execution in ultra-rare disease and a compelling pipeline in larger indications, we believe we are well-positioned for sustained growth in the years ahead.”
Future Expectations and Milestones
-
2025 Guidance: expect continued revenue growth with global net product sales of approximately
$420 million $435 million - Volixibat VISTAS study in primary sclerosing cholangitis (PSC) expected to complete enrollment in second half 2025; topline data expected in 2026
- Volixibat VANTAGE study in primary biliary cholangitis (PBC) expected to complete enrollment in 2026
- LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions expected to complete enrollment in 2026
- FDA Prescription Drug User Fee Act (PDUFA) date for chenodiol in cerebrotendinous xanthomatosis (CTX) is March 28, 2025
- Expect to initiate Phase 2 study for MRM-3379 in Fragile X Syndrome (FXS) in 2025
2024 Highlights
Commercial: Continued leadership in rare disease with a franchise in hepatology and genetic neurology
-
2024 estimated LIVMARLI net product sales of approximately
$213 million $123 million -
Total estimated net product sales of approximately
$99 million $64 million $35 million - Expanded global footprint; 30 countries with commercial access, including successful reimbursement negotiation and launch in the four major European markets
Regulatory and Pipeline: Expanding Mirum’s leadership across multiple rare diseases and larger orphan settings
- Positive interim results for volixibat in VISTAS PSC and VANTAGE PBC studies
-
Volixibat granted breakthrough therapy designation for treatment of cholestatic pruritus in PBC by the
U.S. Food and Drug Administration (FDA) - Volixibat granted orphan drug designation for the treatment of cholestatic pruritus in PBC by the FDA
- LIVMARLI approved by the FDA for cholestatic pruritus in progressive familial intrahepatic cholestasis (PFIC) patients 12 months and older
-
LIVMARLI approved in
Europe for treatment of PFIC in patients three months and older - Initiated the LIVMARLI EXPAND Phase 3 study for pruritus in rare cholestatic conditions
- New Drug Application (NDA) submitted for chenodiol in CTX
- In-licensed global rights to PDE4D inhibitor MRM-3379 for FXS
Corporate and Financial: Sustained financial strength
- Achieved positive cash flow from operations in Q3 2024
-
Cash, cash equivalents and investments of approximately
$287 million $286.3 million - Announced the appointment of Joanne Quan, MD as Chief Medical Officer
The foregoing amounts relating to 2024 financial data are unaudited and preliminary and are subject to completion of financial closing procedures. Additional information and disclosure would be required for a more complete understanding of the company’s financial position and results of operations as of December 31, 2024.
Mirum will present at the 43rd annual J.P. Morgan Healthcare Conference in
About LIVMARLI® (maralixibat) oral solution
LIVMARLI® (maralixibat) oral solution is an orally administered, ileal bile acid transporter (IBAT) inhibitor approved by the
LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for 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 Alagille syndrome 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 Alagille syndrome 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 Volixibat
Volixibat is an 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 (VISTAS study), and primary biliary cholangitis (VANTAGE study). In June, Mirum announced positive interim results from the Phase 2b VANTAGE study showing statistically significant improvement in pruritus as well as meaningful reductions in serum bile acids and improvements in fatigue for patients treated with volixibat. No new safety signals were observed, and the most common adverse event was diarrhea with all cases mild to moderate.
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 (≥
Please see full Prescribing Information for additional Important Safety Information.
About Cerebrotendinous Xanthomatosis
Cerebrotendinous xanthomatosis (CTX) is a rare, progressive and underdiagnosed disorder of cholesterol metabolism affecting many parts of the body. In people with CTX, the body is unable to break down cholesterol properly causing toxins (e.g., cholestanol and bile alcohols) to build up throughout the body over time. The disorder is inherited in an autosomal recessive genetic manner. Signs and symptoms of CTX include neonatal cholestasis (jaundice or bile flow interruption), chronic diarrhea, the development of bilateral cataracts before the age of 18, development of tendon xanthomas (fatty deposits in the tendons) during teenage years or later, and neurologic deterioration. The types, combinations and severity of symptoms can be different from person to person making diagnosis challenging and often delayed.
About chenodiol tablets
Chenodiol tablets 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. More recently, the US Food and Drug Administration (FDA) granted chenodiol orphan drug designation for cerebrotendinous xanthomatosis (CTX). CTX is a rare progressive disorder that can affect the brain, spinal cord, tendons, eyes and arteries. Chenodiol is not yet indicated for the treatment of CTX but has received a medical necessity determination in the
About Mirum Pharmaceuticals
Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare diseases affecting children and adults. Mirum has three approved medications: LIVMARLI® (maralixibat) oral solution, CHOLBAM® (cholic acid) capsules, and CHENODAL® (chenodiol) tablets.
LIVMARLI, an IBAT inhibitor, is approved for the treatment of two rare liver diseases affecting children and adults. It is approved for the treatment of cholestatic pruritus in patients with Alagille syndrome in the
Mirum's late-stage pipeline includes investigational treatments for several rare diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2 VISTAS study for primary sclerosing cholangitis (PSC) and Phase 2b VANTAGE study for primary biliary cholangitis. Volixibat has been granted Breakthrough Therapy Designation for the treatment of cholestatic pruritus in patients with PBC. Chenodiol, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023. Mirum has submitted a new drug application with the FDA for the approval of chenodiol to treat CTX in the
To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter (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, being on track to achieve our revised financial guidance, our expected financial results as of December 31, 2024, including our net product sales and cash, cash equivalents and investments, delivering life changing medicines for patients suffering from rare diseases, the results, enrollment, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, including newly in-licensed product candidates, the timing and results of interim analyses of our ongoing studies and the regulatory approval path for its product candidates globally, including the anticipated PDUFA date for chenodiol for CTX and additional international launches expected in 2025. 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” 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 Quarterly Report on Form 10-Q for the quarter ended September 30, 2024 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.
View source version on businesswire.com: https://www.businesswire.com/news/home/20250113693031/en/
Investor Contacts:
Andrew McKibben
ir@mirumpharma.com
Media Contact:
Erin Murphy
media@mirumpharma.com
Source: Mirum Pharmaceuticals, Inc.
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