Mirum Pharmaceuticals Reports First Quarter 2022 Financial Results and Provides Business Update
Mirum Pharmaceuticals reported a total revenue of $12.9 million for Q1 2022, with net product revenue from LIVMARLI® at $10.9 million. This marks significant growth compared to the previous year's figures. Operating expenses decreased to $45.6 million from $47.6 million in Q1 2021. Despite a net loss of $36.6 million (or $1.17 per share), it was an improvement from last year’s loss of $50.5 million. The company anticipates key clinical trial data and regulatory approvals in late 2022, enhancing prospects for future revenue growth.
- Total revenue increased to $12.9 million in Q1 2022, driven by $10.9 million from LIVMARLI.
- Net product revenue for LIVMARLI showed significant growth over Q1 2021.
- Operating expenses reduced to $45.6 million compared to $47.6 million in the previous year.
- Research and development expenses decreased by $14 million from the prior year.
- Net loss of $36.6 million represents a sizable financial burden.
- Selling, general and administrative expenses increased to $19.1 million, a significant rise from $9.5 million in Q1 2021.
- Total revenue for the first quarter of 2022 was
- Conference call to provide business updates today,
“The first quarter was tremendously successful for Mirum. Rapid growth in LIVMARLI revenue means that children suffering from pruritus associated with Alagille syndrome finally have a treatment option and are getting the care they need,” said
-
Hepatology Communications recently published data from the maralixibat Phase 2 INDIGO study evaluating patients with PFIC. -
The
European Medical Journal published an article summarizing predictors of six-year event-free survival in patients with Alagille syndrome treated with maralixibat.
Upcoming Anticipated Milestones
-
LIVMARLI (maralixibat)
- Topline data evaluating more than 90 patients in the MARCH-PFIC Phase 3 clinical trial expected in the fourth quarter of 2022.
-
LIVMARLI approval by the
European Medicines Agency for cholestatic liver disease in patients with Alagille syndrome, expected in the fourth quarter of 2022. - EMBARK Phase 2b clinical trial for biliary atresia currently enrolling; topline data anticipated in 2023.
-
Volixibat
- Interim analysis expected for VISTAS Phase 2b clinical trial for primary sclerosing cholangitis in the fourth quarter of 2022.
- Interim analysis expected for OHANA Phase 2b clinical trial for intrahepatic cholestasis of pregnancy in the fourth quarter of 2022.
- Enrollment ongoing for VANTAGE Phase 2b clinical trial for primary biliary cholangitis; interim analysis expected in 2023.
Financial Results
-
Net product revenue of LIVMARLI for the quarter ended
March 31, 2022 was$10.9 million -
License revenue for the quarter ended
March 31, 2022 was$2.0 million -
Total operating expenses for the quarter ended
March 31, 2022 were$45.6 million $47.6 million -
Research and development expenses for the first quarter ended
March 31, 2022 were$24.1 million $38.1 million $17.0 million -
Selling, general and administrative (SG&A) expenses for the first quarter of 2022 were
$19.1 million $9.5 million
-
Research and development expenses for the first quarter ended
-
For the quarter ended
March 31, 2022 , Mirum reported a net loss of$36.6 million $1.17 $50.5 million $1.68 -
As of
March 31, 2022 , Mirum had cash, cash equivalents, restricted cash equivalents, and investments of$239.9 million
Business Update Conference Call
Mirum will host a conference call today,
Conference Call Details:
International: 1-646-904-5544
Passcode: 717854
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 progressive familial intrahepatic cholestasis (PFIC) and 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 serious 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.
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 IBAT 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 Phase 2b clinical trial), intrahepatic cholestasis of pregnancy (OHANA Phase 2b clinical trial), and primary biliary cholangitis (VANTAGE Phase 2b clinical trial).
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 MARCH Phase 3 clinical trial for progressive familial intrahepatic cholestasis (PFIC) and 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 has submitted a Marketing Authorization Application to the
Mirum’s second investigational treatment, volixibat, an oral IBAT inhibitor, is being evaluated in three potentially registrational studies including the VISTAS Phase 2b clinical trial for adults with primary sclerosing cholangitis, the OHANA Phase 2b clinical trial for pregnant women with intrahepatic cholestasis of pregnancy, 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, meeting the disclosed milestones, continued commercial approvals and success for LIVMARLI, the ongoing and planned clinical trials for Mirum’s 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 “will,” “anticipate,” “expect,” “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 the COVID-19 pandemic, and the other risks described in Mirum’s filings with the
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Condensed Consolidated Statement of Operations Data |
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(in thousands, except share and per share amounts) |
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(Unaudited) |
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Three Months |
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Three Months |
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Ended |
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Ended |
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| Revenue: | |||||||||||
| Product sales, net | $ |
10,892 |
|
$ |
- |
|
|||||
| License revenue |
|
2,000 |
|
|
- |
|
|||||
| Total revenue |
|
12,892 |
|
|
- |
|
|||||
| Operating expenses: | |||||||||||
| Cost of sales | $ |
2,424 |
|
$ |
- |
|
|||||
| Research and development | 24,088 |
|
38,134 |
|
|||||||
| Selling, general and administrative |
|
19,116 |
|
|
9,479 |
|
|||||
| Total operating expenses (1) |
|
45,628 |
|
|
47,613 |
|
|||||
| Loss from operations |
|
(32,736 |
) |
|
(47,613 |
) |
|||||
| Other income (expense): | |||||||||||
| Interest income |
|
69 |
|
|
149 |
|
|||||
| Interest expense |
|
(3,774 |
) |
|
(3,381 |
) |
|||||
| Change in fair value of derivative liability |
|
- |
|
|
334 |
|
|||||
| Other expense, net |
|
(154 |
) |
|
(16 |
) |
|||||
| Net loss before provision for income taxes |
|
(36,595 |
) |
|
(50,527 |
) |
|||||
| Provision for income taxes |
|
11 |
|
|
5 |
|
|||||
| Net loss | $ |
(36,606 |
) |
$ |
(50,532 |
) |
|||||
| Net loss per share, basic and diluted | $ |
(1.17 |
) |
$ |
(1.68 |
) |
|||||
| Weighted-average shares of common stock outstanding, basic and diluted | |||||||||||
|
31,296,223 |
|
|
30,105,017 |
|
||||||
(1) |
Amounts include stock-based compensation expense as follows: | ||||||||||
| Research and development | $ |
2,585 |
|
$ |
2,743 |
|
|||||
| Selling, general and administrative |
|
3,976 |
|
|
2,542 |
|
|||||
| Total stock-based compensation | $ |
6,561 |
|
$ |
5,285 |
|
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|
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Selected Condensed Consolidated Balance Sheet Data |
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(in thousands) |
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2022 |
|
2021 |
||||
|
|
(Unaudited) |
|
|
||||
| Cash, cash equivalents, restricted cash equivalents and investments | $ |
239,908 |
|
$ |
261,524 |
|
||
| Working capital |
|
120,715 |
|
|
123,996 |
|
||
| Total assets |
|
278,148 |
|
|
294,651 |
|
||
| Accumulated deficit |
|
(293,765 |
) |
|
(257,159 |
) |
||
| Total stockholders' equity |
|
108,932 |
|
|
120,212 |
|
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View source version on businesswire.com: https://www.businesswire.com/news/home/20220505005401/en/
Investor Contacts:
ir@mirumpharma.com
ir@mirumpharma.com
Media Contact:
media@mirumpharma.com
Source:
FAQ
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