Mirum Pharmaceuticals Reports Fourth Quarter and Year-End 2022 Financial Results and Provides Business Update
Mirum Pharmaceuticals reported financial results for 2022, marking a successful first full year for LIVMARLI® with net product sales of $75.1 million. Total revenue reached $77.1 million, a significant increase from $3.1 million in 2021. The company anticipates a 50% year-over-year growth in net sales for 2023, fueled by recent launches in Germany and potential label expansions. The fourth quarter saw a 48% sales increase over Q3 2022. Strong data from the Phase 3 MARCH-PFIC study highlights LIVMARLI's efficacy in treating pruritus and serum bile acids. Mirum has raised $121.9 million in capital since January 2022, strengthening its balance sheet.
- Net product sales of LIVMARLI reached $75.1 million in 2022, up from $3.1 million in 2021.
- Projected 50% growth in net product sales for 2023.
- Fourth quarter 2022 net sales increased by 48% compared to Q3 2022.
- Successful launch of LIVMARLI in Germany in early 2023.
- Groundbreaking data from Phase 3 MARCH-PFIC study demonstrated efficacy in treating new PFIC types.
- Operating expenses rose from $192.6 million in 2021 to $208.3 million in 2022.
- Net product sales for LIVMARLI® (maralixibat) oral solution totaled
- Conference call to provide business updates today,
“We are proud of our strong commercial, clinical and regulatory execution in 2022 that realized
LIVMARLI Alagille syndrome: Strong launch continues fueled by
-
Fourth quarter 2022 net sales grew
48% as compared to third quarter 2022. -
U.S. business positioned for50% net product sales growth in 2023. -
Launched LIVMARLI in
Germany in early 2023 followingEuropean Commission approval. - Approximately 600 Alagille syndrome patients have received LIVMARLI worldwide to date.
- Publications highlighting long-term impact:
LIVMARLI PFIC: Compelling profile emerges from Phase 3 MARCH-PFIC study
- Presented groundbreaking data from the LIVMARLI Phase 3 MARCH-PFIC study at AASLD, showcasing an unprecedented reduction in pruritus and serum bile acids.
- First study to demonstrate efficacy of ileal bile acid transporter (IBAT) inhibitor in new PFIC types.
-
Submitted a supplemental New Drug Application (sNDA) to
U.S. Food and Drug Administration .
Pipeline: On track for catalyst rich 2023
- LIVMARLI in biliary atresia on-track for top-line data in second half of 2023.
- Volixibat in primary sclerosing cholangitis on-track for interim analysis in mid-2023.
- Volixibat in primary biliary cholangitis on track for interim analysis in second half of 2023.
Corporate & financial: Strong financial performance & balance sheet
-
Strengthened balance sheet with additional capital raises totaling
$121.9 million January 2022 . - Lowered net royalty obligations to high single digits to mid-teens by acquiring Satiogen.
-
Total net product revenue from the sale of LIVMARLI for the year ended
December 31, 2022 was$75.1 million $3.1 million -
Stable operating expenses. For the years ended
December 31, 2022 and 2021, total operating expenses were$208.3 million $192.6 million -
As of
December 31, 2022 , Mirum had cash, cash equivalents, restricted cash equivalents, and short-term investments of$251.7 million
Business Update Conference Call
Mirum will host a conference call today,
Conference Call Details:
International: 1 929-526-1599
Passcode: 596100
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 currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including biliary atresia. LIVMARLI has received Breakthrough Therapy designation for ALGS and PFIC type 2 and orphan designation for ALGS, PFIC and biliary atresia. To learn more about ongoing clinical trials with LIVMARLI, please visit Mirum’s clinical trials section on the company’s website.
IMPORTANT SAFETY INFORMATION
LIVMARLI can cause side effects, including:
Changes in liver tests. Changes in certain liver tests are common in patients with Alagille syndrome and can worsen during treatment with LIVMARLI. These changes may be a sign of liver injury and can be serious. Your healthcare provider should do blood tests 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) or loss of appetite.
Stomach and intestinal (gastrointestinal) problems. LIVMARLI can cause stomach and intestinal problems, including diarrhea, stomach pain, and vomiting during treatment. 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. FSV deficiency is common in patients with Alagille syndrome but may worsen during treatment. Your healthcare provider should do blood tests before starting and during treatment.
Other common side effects reported during treatment were bone fractures and gastrointestinal bleeding.
US Prescribing Information
EU SmPC
About Volixibat
Volixibat is an oral, minimally absorbed agent designed to selectively inhibit the apical sodium dependent bile acid transporter (ASBT). Volixibat may offer a novel approach in the treatment of adult cholestatic diseases by blocking the recycling of bile acids, through inhibition of ASBT, 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
Mirum’s late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. LIVMARLI, an oral ileal bile acid transporter (IBAT) inhibitor, is currently being evaluated in clinical trials for pediatric liver diseases and includes the EMBARK Phase 2b clinical trial for patients with biliary atresia. In addition, Mirum has an expanded access program open across multiple countries for eligible patients with ALGS and PFIC.
Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in two potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis and the VANTAGE Phase 2b clinical trial for adults with primary biliary cholangitis.
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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 LIVMARLI, including growth in year over year net product sales, planned LIVMARLI launches in additional international markets, label expansion into PFIC, becoming a global leader in rare disease, the results, conduct and progress of Mirum’s ongoing and planned studies for its product candidates 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 “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 the COVID-19 pandemic, and the other risks described in Mirum’s filings with the
| Condensed Consolidated Statement of Operations Data | |||||||||||||||
| (in thousands, except per share and per share amounts) | |||||||||||||||
| Three Months Ended | Year Ended | ||||||||||||||
2022 |
2021 |
2022 |
2021 |
||||||||||||
| (Unaudited) | |||||||||||||||
| Revenue: | |||||||||||||||
| Product sales, net | $ |
27,906 |
|
$ |
3,138 |
|
$ |
75,062 |
|
$ |
3,138 |
|
|||
| License revenue |
|
- |
|
|
- |
|
|
2,000 |
|
|
16,000 |
|
|||
| Total revenue |
|
27,906 |
|
|
3,138 |
|
|
77,062 |
|
|
19,138 |
|
|||
| Operating expenses: | |||||||||||||||
| Cost of sales |
|
4,494 |
|
|
1,903 |
|
|
12,374 |
|
|
1,903 |
|
|||
| Research and development |
|
31,105 |
|
|
27,775 |
|
|
106,842 |
|
|
131,428 |
|
|||
| Selling, general and administrative |
|
26,468 |
|
|
19,035 |
|
|
89,066 |
|
|
59,220 |
|
|||
| Total operating expenses (1) |
|
62,067 |
|
|
48,713 |
|
|
208,282 |
|
|
192,551 |
|
|||
| Loss from operations |
|
(34,161 |
) |
|
(45,575 |
) |
|
(131,220 |
) |
|
(173,413 |
) |
|||
| Other income (expense): | |||||||||||||||
| Interest income |
|
2,143 |
|
|
65 |
|
|
3,857 |
|
|
366 |
|
|||
| Interest expense |
|
(4,359 |
) |
|
(3,766 |
) |
|
(15,979 |
) |
|
(17,590 |
) |
|||
| Change in fair value of derivative liability |
|
674 |
|
|
(1,149 |
) |
|
906 |
|
|
(732 |
) |
|||
| Other income (expense), net |
|
(588 |
) |
|
(17 |
) |
|
365 |
|
|
(582 |
) |
|||
| Gain from sale of priority review voucher, net |
|
- |
|
|
108,000 |
|
|
- |
|
|
108,000 |
|
|||
| Net loss before provision for income taxes |
|
(36,291 |
) |
|
57,558 |
|
|
(142,071 |
) |
|
(83,951 |
) |
|||
| (Benefit from) provision for income taxes |
|
140 |
|
|
12 |
|
|
(6,406 |
) |
|
37 |
|
|||
| Net loss | $ |
(36,431 |
) |
$ |
57,546 |
|
$ |
(135,665 |
) |
$ |
(83,988 |
) |
|||
| Net loss per share: | |||||||||||||||
| Basic | $ |
(4.01 |
) |
$ |
(2.77 |
) |
|||||||||
| Diluted | $ |
(4.02 |
) |
$ |
(2.77 |
) |
|||||||||
| Weighted-average shares outstanding: | |||||||||||||||
| Basic |
|
33,839,072 |
|
|
30,321,722 |
|
|||||||||
| Diluted |
|
33,982,493 |
|
|
30,321,722 |
|
|||||||||
(1) Amounts include stock-based compensation expense as follows: |
|||||||||||||||
| Research and development | $ |
2,532 |
|
$ |
2,095 |
|
$ |
10,050 |
|
$ |
9,888 |
|
|||
| Selling, general and administrative |
|
4,364 |
|
|
3,397 |
|
|
16,957 |
|
|
13,128 |
|
|||
| Total stock-based compensation | $ |
6,896 |
|
$ |
5,492 |
|
$ |
27,007 |
|
$ |
23,016 |
|
|||
| Selected Consolidated Balance Sheet Data | |||||||
| (in thousands) | |||||||
2022 |
2021 |
||||||
| Cash, cash equivalents, restricted cash equivalents and investments | $ |
251,719 |
|
$ |
261,524 |
|
|
| Working capital |
|
125,496 |
|
|
123,996 |
|
|
| Total assets |
|
352,906 |
|
|
294,651 |
|
|
| Accumulated deficit |
|
(392,824 |
) |
|
(257,159 |
) |
|
| Total stockholders' equity |
|
142,037 |
|
|
120,212 |
|
|
View source version on businesswire.com: https://www.businesswire.com/news/home/20230308005769/en/
Investor Contacts:
ir@mirumpharma.com
ir@mirumpharma.com
Media Contact:
media@mirumpharma.com
Source:
FAQ
What were the net product sales for Mirum Pharmaceuticals in 2022?
What is the expected growth for LIVMARLI sales in 2023?
What was the total revenue for Mirum Pharmaceuticals in 2022?
What significant data was presented from the Phase 3 MARCH-PFIC study?
What was the company’s operating expense for 2022?
