Mirum Pharmaceuticals Reports First Quarter 2024 Financial Results and Provides Business Update
- First quarter 2024 total revenue of
- FDA approval of LIVMARLI for cholestatic pruritus in PFIC patients achieved in March
- Volixibat VISTAS and VANTAGE interim analyses scheduled for June of 2024
- Cash balance of
- Conference call to provide business updates today,
“In the first quarter, we made great progress across a number of our strategic objectives that support both the near- and long-term growth of Mirum while maintaining a strong financial position,” said
Commercial: Continued demand growth and reiterate full year guidance of
-
First quarter 2024 global product sales, net of
$68.9 million grew 137% compared to first quarter 2023. -
LIVMARLI first quarter net sales were
$42.8 million , representing 47% growth compared to first quarter 2023. - LIVMARLI approved in the US for treatment of cholestatic pruritus in patients with PFIC.
-
First quarter 2024 CHOLBAM and CHENODAL net sales were
$26.1 million .
Regulatory and Pipeline: H1 Milestones on track
- Interim analyses for volixibat in PSC (VISTAS study) and in PBC (VANTAGE study) expected in June of 2024.
- New Drug Application (NDA) submission for CHENODAL in CTX on track for first half 2024.
- PFIC CHMP approval recommendation anticipated in the first half of 2024.
Corporate and Financial: Strong balance sheet
-
Total revenue for the quarter ended
March 31, 2024 , was$69.2 million compared to$31.6 million for the quarter endedMarch 31, 2023 . -
Total operating expenses were
$95.7 million for the quarter endedMarch 31, 2024 , compared to$58.7 million for the quarter endedMarch 31, 2023 . Total operating expense included$17.1 million of non-cash stock-based compensation and depreciation and amortization expense for the quarter endedMarch 31, 2024 , compared to$9.9 million for the quarter endedMarch 31, 2023 . -
As of
March 31, 2024 , Mirum had cash and cash equivalents of$302.8 million compared to$286.3 million as ofDecember 31, 2023 .
Business Update Conference Call
Mirum will host a conference call today,
Conference Call Details:
International: +1 404 975 4839
Passcode: 479107
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, once-daily, ileal bile acid transporter (IBAT) inhibitor approved by the
LIVMARLI is also the only approved IBAT inhibitor approved by the
Mirum has also submitted LIVMARLI for approval in
LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS and PFIC. 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. Phase 1 and Phase 2 studies of volixibat demonstrated on-target fecal bile acid excretion, a pharmacodynamic marker of ASBT inhibition, in addition to decreases in LDL cholesterol and increases in 7αC4 which are markers of bile acid synthesis. Volixibat has been evaluated in more than 400 individuals across multiple clinical trials. The most common adverse events reported were mild to moderate gastrointestinal events observed in the volixibat groups.
Volixibat is currently being evaluated in Phase 2b studies for primary sclerosing cholangitis (VISTAS study), and primary biliary cholangitis (VANTAGE study).
About CHOLBAM® (cholic acid) capsules
The FDA approved CHOLBAM® (cholic acid) capsules in
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 CHENODAL® (chenodiol) tablets
CHENODAL® is a synthetic oral form of chenodeoxycholic acid (CDCA), a naturally occurring primary bile acid synthesized from cholesterol in the liver. The FDA approved CHENODAL for the treatment of people with radiolucent stones in the gallbladder. In 2010, CHENODAL was granted orphan drug designation for the treatment of cerebrotendinous xanthomatosis (CTX), a rare autosomal recessive lipid storage disease.
While CHENODAL® is not currently approved for CTX, it received a medical necessity determination in the
About
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 two investigational treatments for debilitating liver diseases. Volixibat, an IBAT inhibitor, is being evaluated in two potentially registrational studies including the Phase 2b VISTAS study for primary sclerosing cholangitis and Phase 2b VANTAGE study for primary biliary cholangitis. Lastly, CHENODAL, has been evaluated in a Phase 3 clinical study, RESTORE, to treat patients with CTX, with positive topline results reported in 2023.
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, progress across Mirum’s strategic objectives, its near- and long-term growth, the strength of its financial position, the performance of its commercial business, growth in demand for LIVMARLI, achievement of full-year revenue guidance, the results, conduct and progress of Mirum’s ongoing and planned studies for its product candidates, the timing and results of interim analyses of Mirum’s ongoing studies and the regulatory approval path for its product candidates globally. 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,” “forecasts,” “forward,” “planned,” “poised,”, “positioned” “potential”, “will” 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 on Form 10-K for the year ended
Condensed Consolidated Statement of Operations Data (in thousands, except share and per share amounts) (Unaudited) |
||||||||
|
|
Three Months Ended |
||||||
|
|
2024 |
|
2023 |
||||
Revenue: |
|
|
|
|
||||
Product sales, net |
|
$ |
68,917 |
|
|
$ |
29,098 |
|
License and other revenue |
|
|
305 |
|
|
|
2,500 |
|
Total revenue |
|
|
69,222 |
|
|
|
31,598 |
|
Operating expenses: |
|
|
|
|
||||
Cost of sales (1) |
|
|
17,830 |
|
|
|
4,979 |
|
Research and development |
|
|
32,222 |
|
|
|
23,548 |
|
Selling, general and administrative |
|
|
45,638 |
|
|
|
30,219 |
|
Total operating expenses (2) |
|
|
95,690 |
|
|
|
58,746 |
|
Loss from operations |
|
|
(26,468 |
) |
|
|
(27,148 |
) |
Other income (expense): |
|
|
|
|
||||
Interest income |
|
|
3,633 |
|
|
|
2,272 |
|
Interest expense |
|
|
(3,577 |
) |
|
|
(4,242 |
) |
Other income (expense), net |
|
|
1,757 |
|
|
|
(811 |
) |
Net loss before provision for income taxes |
|
|
(24,655 |
) |
|
|
(29,929 |
) |
Provision for income taxes |
|
|
624 |
|
|
|
201 |
|
Net loss |
|
|
(25,279 |
) |
|
|
(30,130 |
) |
|
|
|
|
|
||||
Net loss per share, basic and diluted |
|
$ |
(0.54 |
) |
|
$ |
(0.80 |
) |
Weighted-average shares of common stock outstanding, basic and diluted |
|
|
46,927,550 |
|
|
|
37,675,306 |
|
|
|
|
|
|
||||
|
|
|
|
|
||||
(1) Amounts include intangible amortization expense as follows: |
|
|
|
|
||||
|
|
|
|
|
||||
Intangible amortization |
|
$ |
5,402 |
|
|
$ |
1,259 |
|
|
|
|
|
|
||||
(2) Amounts include stock-based compensation expense as follows: |
|
|
|
|
||||
|
|
|
|
|
||||
Research and development |
|
$ |
3,861 |
|
|
$ |
2,715 |
|
Selling, general and administrative |
|
|
7,589 |
|
|
|
5,846 |
|
Total stock-based compensation |
|
$ |
11,450 |
|
|
$ |
8,561 |
|
Condensed Consolidated Balance Sheet Data (Unaudited) |
|||||||
|
|
|
|
||||
|
|
|
|
||||
Assets |
|
|
|
||||
Current assets: |
|
|
|
||||
Cash and cash equivalents |
$ |
302,843 |
|
|
$ |
286,326 |
|
Accounts receivable |
|
54,999 |
|
|
|
67,968 |
|
Inventory |
|
21,606 |
|
|
|
22,312 |
|
Prepaid expenses and other current assets |
|
10,017 |
|
|
|
10,935 |
|
Total current assets |
|
389,465 |
|
|
|
387,541 |
|
Intangible assets, net |
|
257,443 |
|
|
|
252,925 |
|
Other noncurrent assets |
|
5,054 |
|
|
|
6,155 |
|
Total assets |
$ |
651,962 |
|
|
$ |
646,621 |
|
Liabilities and Stockholders’ Equity |
|
|
|
||||
Current liabilities: |
|
|
|
||||
Accounts payable |
$ |
15,983 |
|
|
$ |
7,416 |
|
Accrued expenses |
|
89,568 |
|
|
|
78,544 |
|
Operating lease liabilities |
|
358 |
|
|
|
1,104 |
|
Total current liabilities |
|
105,909 |
|
|
|
87,064 |
|
Operating lease liabilities, noncurrent |
|
328 |
|
|
|
617 |
|
Convertible notes payable, net |
|
306,835 |
|
|
|
306,421 |
|
Other liabilities |
|
4,287 |
|
|
|
3,849 |
|
Total liabilities |
|
417,359 |
|
|
|
397,951 |
|
Commitments and contingencies |
|
|
|
||||
Stockholders’ equity: |
|
|
|
||||
Preferred stock |
|
— |
|
|
|
— |
|
Common stock |
|
5 |
|
|
|
5 |
|
Additional paid-in capital |
|
816,129 |
|
|
|
803,260 |
|
Accumulated deficit |
|
(581,518 |
) |
|
|
(556,239 |
) |
Accumulated other comprehensive (loss) income |
|
(13 |
) |
|
|
1,644 |
|
Total stockholders’ equity |
|
234,603 |
|
|
|
248,670 |
|
Total liabilities and stockholders’ equity |
$ |
651,962 |
|
|
$ |
646,621 |
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20240508006127/en/
Investor Contact:
ir@mirumpharma.com
Media Contact:
media@mirumpharma.com
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