Mirum Pharmaceuticals Reports Third Quarter 2024 Financial Results and Provides Business Update

Rhea-AI Summary

Mirum Pharmaceuticals (Nasdaq: MIRM) reported third-quarter 2024 financial results, showcasing a significant growth in net product sales, reaching $90.3 million, an 89% increase compared to Q3 2023. The company has raised its 2024 guidance to $330 - 335 million. Key developments include the breakthrough therapy designation for volixibat in PBC, with interim results to be presented at the AASLD Liver Meeting 2024. The PDUFA date for chenodiol in CTX is set for December 28, 2024. Mirum also in-licensed global rights to the PDE4D inhibitor MRM-3379, targeting Fragile X syndrome, with a Phase 2 study initiation expected in 2025. As of September 30, 2024, Mirum's cash, cash equivalents, and investments totaled $293.8 million. Operating expenses for Q3 2024 were $103.1 million, up from $72.9 million in Q3 2023. A business update conference call is scheduled for November 12, 2024.

Positive

• Third quarter 2024 net product sales of $90.3 million, up 89% YoY.
• 2024 guidance increased to $330 - 335 million.
• Volixibat granted breakthrough therapy designation for PBC.
• Strong cash position of $293.8 million as of September 30, 2024.

Negative

• Total operating expenses increased to $103.1 million in Q3 2024 from $72.9 million in Q3 2023.

Insights

Financial Analyst

The Q3 results demonstrate exceptional commercial momentum with $90.3 million in global net product sales, representing a robust 89% year-over-year growth. LIVMARLI leads revenue generation at $59.1 million, while CHOLBAM and CHENODAL contribute $31.2 million. The company's strong balance sheet shows $293.8 million in cash and investments, providing ample runway for pipeline development.

The raised full-year guidance to $330-335 million signals management's confidence in continued commercial execution. Operating expenses of $103.1 million reflect increased investment in growth initiatives, though $18 million is non-cash. The diversified revenue streams and breakthrough therapy designation for volixibat in PBC position Mirum for sustained growth in the rare disease market.

Medical Research Analyst

The pipeline developments significantly strengthen Mirum's rare disease portfolio. The breakthrough therapy designation for volixibat in PBC cholestatic pruritus accelerates the regulatory pathway and validates its potential clinical impact. The upcoming PDUFA date for chenodiol in CTX could expand the company's neurology franchise, while the initiation of the EXPAND study for LIVMARLI demonstrates commitment to label expansion.

The addition of MRM-3379 for Fragile X syndrome diversifies the pipeline into rare genetic neurocognitive disorders, representing a strategic expansion beyond liver diseases. The late-breaker presentation at The Liver Meeting® suggests potentially significant clinical data for volixibat, which could further validate its therapeutic potential.

• Third quarter global net product sales of $90.3 million; 2024 guidance increased to $330 - 335 million
• Volixibat granted breakthrough therapy designation for treatment of cholestatic pruritus in PBC, interim results to be presented as a Late Breaker presentation at AASLD’s The Liver Meeting® 2024
• PDUFA date for chenodiol in CTX is December 28, 2024
• In-licensed global rights to PDE4D inhibitor MRM-3379
• Conference call to provide business updates today, November 12 at 5:30 a.m. PT/8:30 a.m. ET

FOSTER CITY, Calif.--(BUSINESS WIRE)-- Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for the third quarter 2024 and provided a business update.

“The Mirum team executed across all aspects of the business in the third quarter with growth across all three commercial medicines highlighted by acceleration of LIVMARLI PFIC sales,” said Chris Peetz, chief executive officer of Mirum. “We also made noteworthy progress on the clinical pipeline with the granting of breakthrough designation for volixibat in PBC and the initiation of the EXPAND study for LIVMARLI in other indications with cholestatic pruritus. Our rare genetic neurology franchise is also taking shape with a December 28 PDUFA date for chenodiol for CTX and the addition of Phase 2 ready MRM-3379 for Fragile X syndrome.”

Commercial: Continued growth across all three commercial medicines

• Third quarter 2024 global net product sales of $90.3 million grew 89% compared to the third quarter 2023.
• Third quarter 2024 LIVMARLI net sales were $59.1 million.
• Third quarter 2024 CHOLBAM and CHENODAL net sales were $31.2 million.

Regulatory and Pipeline: Expanding Mirum’s leadership across multiple rare disease settings

• U.S. Food and Drug Administration (FDA) Prescription Drug User Free Act (PDUFA) date for chenodiol in cerebrotendinous xanthomatosis (CTX) is December 28, 2024.
• Late-breaker presentation for The Liver Meeting® accepted featuring volixibat interim data from the VANTAGE PBC study.
• Breakthrough therapy designation granted for volixibat in PBC.
• Phase 3 EXPAND study initiated, a label expansion opportunity for LIVMARLI.
• In-licensed MRM-3379, a PDE4D inhibitor for development in Fragile X syndrome (FXS), a rare genetic neurocognitive disorder; Phase 2 study initiation expected in 2025.

Corporate and Financial: Strong balance sheet

• As of September 30, 2024, Mirum had cash, cash equivalents and investments of $293.8 million compared to $286.3 million as of December 31, 2023.
• Total operating expenses were $103.1 million for the quarter ended September 30, 2024, compared to $72.9 million for the quarter ended September 30, 2023. Total operating expenses included $18.0 million of non-cash stock-based compensation and, depreciation and amortization for the quarter ended September 30, 2024.

Business Update Conference Call

Mirum will host a conference call today, November 12 at 5:30 a.m. PT/8:30 a.m. ET, to provide business updates. Join the call using the following details:

Conference Call Details:

U.S./Toll-Free: +1 833 470 1428
International: +1 404 975 4839
Passcode: 749358

You may also access the call via webcast by visiting the Events & Presentations section on Mirum’s website. A replay of this webcast will be available for 30 days.

About LIVMARLI® (maralixibat) oral solution

LIVMARLI® (maralixibat) oral solution 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 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 (≥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 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 U.S. by the FDA.

About Mirum Pharmaceuticals, Inc.

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 U.S. (three months and older), in Europe (two months and older), and in other regions globally. It is also approved in the U.S. in cholestatic pruritus in PFIC patients 12 months of age and older; in Europe, it is approved for patients with PFIC three months of age and older. Mirum is also initiating the Phase 3 EXPAND study, a label expansion opportunity for LIVMARLI in additional settings of cholestatic pruritus. CHOLBAM is FDA-approved for the treatment of bile acid synthesis disorders due to single enzyme deficiencies and adjunctive treatment of peroxisomal disorders in patients who show signs or symptoms or liver disease. CHENODAL has received medical necessity recognition by the FDA to treat patients with cerebrotendinous xanthomatosis (CTX).

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 U.S. Lastly, Mirum is planning for a Phase 2 study evaluating MRM-3379, a PDE4D inhibitor for the treatment of Fragile X syndrome, a rare genetic neurocognitive disorder.

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, continued commercial success for our approved products, including continued growth in year over year net product sales, being on track to achieve our revised financial guidance, delivering life changing medicines for patients suffering from rare diseases, the results, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, including newly in-licensed product candidates, and the regulatory approval path for its product candidates globally, including the anticipated PDUFA date for chenodiol for CTX. 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 “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 and the other risks described in Mirum’s 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. A further description of risks and uncertainties can be found in Mirum’s Annual Report on Form 10-K for the fiscal year ended December 31, 2023 and in its subsequent reports on Form 10-Q, including in the sections thereof captioned “Risk Factors,” as well as in its subsequent reports on Form 8-K, all of which are filed with the U.S. Securities and Exchange Commission and available at www.sec.gov

Mirum Pharmaceuticals, Inc. Condensed Consolidated Statement of Operations Data (in thousands, except share and per share amounts) (Unaudited)
		Three Months Ended September 30,								Nine Months Ended September 30,
			2024				2023				2024				2023
Revenue:
Product sales, net		$	90,302			$	47,725			$	236,979			$	109,320
License and other revenue			75				—				495				7,500
Total revenue			90,377				47,725				237,474				116,820
Operating expenses:
Cost of sales (1)			20,806				10,228				58,863				22,019
Research and development			31,710				26,117				96,604				71,674
Selling, general and administrative			50,545				36,528				145,391				99,696
Total operating expenses (2)			103,061				72,873				300,858				193,389
Loss from operations			(12,684	)			(25,148	)			(63,384	)			(76,569	)
Other income (expense):
Interest income			3,469				4,061				10,588				9,960
Interest expense			(3,586	)			(3,574	)			(10,732	)			(11,542	)
Loss from termination of revenue interest purchase agreement			—				—				—				(49,076	)
Other income (expense), net			(1,087	)			1,322				982				237
Net loss before provision for income taxes			(13,888	)			(23,339	)			(62,546	)			(126,990	)
Provision for income taxes			347				249				1,606				766
Net loss			(14,235	)			(23,588	)			(64,152	)			(127,756	)
Net loss per share, basic and diluted		$	(0.30	)		$	(0.57	)		$	(1.36	)		$	(3.28	)
Weighted-average shares of common stock outstanding, basic and diluted			47,782,619				41,098,920				47,316,789				38,973,060
(1) Amounts include intangible amortization expense as follows:
Intangible amortization		$	5,894			$	2,582			$	16,889			$	5,099
(2) Amounts include stock-based compensation expense as follows:
Cost of sales		$	319			$	—			$	637			$	—
Research and development			3,571				2,626				10,978				8,013
Selling, general and administrative			8,018				5,759				23,578				17,290
Total stock-based compensation		$	11,908			$	8,385			$	35,193			$	25,303

Mirum Pharmaceuticals, Inc. Condensed Consolidated Balance Sheet Data (Unaudited)
	September 30, 2024				December 31, 2023
Assets
Current assets:
Cash and cash equivalents	$	222,969			$	286,326
Short-term investments		61,476				—
Accounts receivable		68,530				67,968
Inventory		21,594				22,312
Prepaid expenses and other current assets		12,057				10,935
Total current assets		386,626				387,541
Restricted cash		425				—
Long-term investments		9,321				—
Intangible assets, net		255,794				252,925
Other noncurrent assets		15,727				6,155
Total assets	$	667,893			$	646,621
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	9,545			$	7,416
Accrued expenses		104,700				78,544
Operating lease liabilities, current		1,506				1,104
Total current liabilities		115,751				87,064
Operating lease liabilities, noncurrent		8,480				617
Convertible notes payable, net		307,666				306,421
Other liabilities		3,972				3,849
Total liabilities		435,869				397,951
Commitments and contingencies
Stockholders’ equity:
Preferred stock		—				—
Common stock		5				5
Additional paid-in capital		851,490				803,260
Accumulated deficit		(620,391	)			(556,239	)
Accumulated other comprehensive income		920				1,644
Total stockholders’ equity		232,024				248,670
Total liabilities and stockholders’ equity	$	667,893			$	646,621

 

View source version on businesswire.com: https://www.businesswire.com/news/home/20241112481474/en/

Investor Contact:

Andrew McKibben

ir@mirumpharma.com

Media Contact:

Erin Murphy

media@mirumpharma.com

Source: Mirum Pharmaceuticals, Inc.

FAQ

What were Mirum Pharmaceuticals' third quarter 2024 net product sales?

Mirum Pharmaceuticals' third quarter 2024 net product sales were $90.3 million.

What is the 2024 guidance for Mirum Pharmaceuticals?

Mirum Pharmaceuticals has increased its 2024 guidance to $330 - 335 million.

What breakthrough therapy designation did Mirum Pharmaceuticals receive in Q3 2024?

Mirum Pharmaceuticals received a breakthrough therapy designation for volixibat in PBC.

When is the PDUFA date for chenodiol in CTX?

The PDUFA date for chenodiol in CTX is December 28, 2024.

What is the cash position of Mirum Pharmaceuticals as of September 30, 2024?

As of September 30, 2024, Mirum Pharmaceuticals had cash, cash equivalents, and investments totaling $293.8 million.