FOSTER CITY, Calif. / Nov 02, 2023 / Business Wire / Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM) today reported financial results for third quarter 2023 and provided a business update.
“We made significant progress in the third quarter with record LIVMARLI revenues in both the United States and Internationally, the closing of the acquisition of CHENODAL and CHOLBAM and the positive top line results from the unblinding of the RESTORE trial of CHENODAL in CTX,” said Chris Peetz, president and chief executive officer at Mirum. “The company is well positioned to maintain this momentum with continued strong revenue growth and we look forward to multiple exciting upcoming clinical and regulatory catalysts.”
LIVMARLI®: Continued growth in Alagille syndrome worldwide net sales and progressive familial intrahepatic cholestasis (PFIC) near-term label expansion opportunity
Completed acquisition of CHENODAL and CHOLBAM: establishes Mirum’s leading growth franchise in pediatric hepatology
Pipeline: Three upcoming late-stage data catalysts
Corporate and financial: Strong balance sheet and experienced management team driving growth
Business Update Conference Call
Mirum will host a conference call today, November 2, 2023, at 1:30 p.m. PT/4:30 p.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: 979561
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 U.S. Food and Drug Administration for the treatment of cholestatic pruritus in patients with Alagille syndrome (ALGS) one year of age and older. LIVMARLI is also approved by the European Commission for the treatment of cholestatic pruritus in patients with ALGS two months and older. It is the only approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information for U.S. residents, please visit LIVMARLI.com.
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 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
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
ADVERSE REACTIONS
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 U.S. by the FDA and has been used as the standard of care for more than three decades. Efforts are being made to obtain FDA approval of CHENODAL for the treatment of CTX and a Phase 3 clinical trial for this indication was initiated in January 2020. We believe the prevalent patient population in the United States with CTX exceeds 1,000.
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 cholestatic pruritus in patients with Alagille syndrome in the U.S. (three months and older), in Europe (two months and older), and in Canada. Mirum has also submitted LIVMARLI for approval in the U.S. in cholestatic pruritus in PFIC patients three months of age and older and in Europe in PFIC for patients two months of age and older. 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 three investigational treatments for debilitating liver diseases. The LIVMARLI development program includes the Phase 2b EMBARK study for biliary atresia. Mirum’s second investigational IBAT inhibitor is volixibat, which 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.
To learn more about Mirum, visit mirumpharma.com and follow Mirum on Facebook, LinkedIn, Instagram and Twitter.
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, CHENODAL and CHOLBAM including growth in year over year net product sales, planned LIVMARLI launches in additional international markets, label expansion into PFIC, CHENODAL potential full indication in CTX, 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 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.
| Mirum Pharmaceuticals, Inc. | |||||||||||||||
| Condensed Consolidated Statement of Operations Data | |||||||||||||||
| (in thousands, except share and per share amounts) | |||||||||||||||
| (Unaudited) | |||||||||||||||
Three Months Ended |
| Nine Months Ended | |||||||||||||
September 30, |
| September 30, | |||||||||||||
2023 |
| 2022 |
| 2023 |
| 2022 | |||||||||
| Revenue: | |||||||||||||||
| Product sales, net | $ | 47,725 |
| $ | 18,780 |
| $ | 109,320 |
| $ | 47,156 |
| |||
| License revenue |
| - |
|
| - |
|
| 7,500 |
|
| 2,000 |
| |||
| Total revenue |
| 47,725 |
|
| 18,780 |
|
| 116,820 |
|
| 49,156 |
| |||
| Operating expenses: | |||||||||||||||
| Cost of sales (1) |
| 10,228 |
|
| 2,932 |
|
| 22,019 |
|
| 7,880 |
| |||
| Research and development |
| 26,117 |
|
| 26,217 |
|
| 71,674 |
|
| 75,737 |
| |||
| Selling, general and administrative |
| 36,528 |
|
| 22,513 |
|
| 99,696 |
|
| 62,598 |
| |||
| Total operating expenses (2) |
| 72,873 |
|
| 51,662 |
|
| 193,389 |
|
| 146,215 |
| |||
| Loss from operations |
| (25,148 | ) |
| (32,882 | ) |
| (76,569 | ) |
| (97,059 | ) | |||
| Other income (expense): | |||||||||||||||
| Interest income |
| 4,061 |
|
| 1,352 |
|
| 9,960 |
|
| 1,714 |
| |||
| Interest expense |
| (3,574 | ) |
| (3,971 | ) |
| (11,542 | ) |
| (11,620 | ) | |||
| Change in fair value of derivative liability |
| - |
|
| - |
|
| - |
|
| 232 |
| |||
| Loss on extinguishment of RIPA |
| - |
|
| - |
|
| (49,076 | ) |
| - |
| |||
| Other income (expense), net |
| 1,322 |
|
| (192 | ) |
| 237 |
|
| 953 |
| |||
| Net loss before for income taxes |
| (23,339 | ) |
| (35,693 | ) |
| (126,990 | ) |
| (105,780 | ) | |||
| (Benefit) provision for income taxes |
| 249 |
|
| 13 |
|
| 766 |
|
| (6,546 | ) | |||
| Net loss | $ | (23,588 | ) | $ | (35,706 | ) | $ | (127,756 | ) | $ | (99,234 | ) | |||
| Net loss per share, basic and diluted | $ | (0.57 | ) | $ | (1.02 | ) | $ | (3.28 | ) | $ | (3.03 | ) | |||
| Weighted-average shares of common stock outstanding, basic |
| 41,098,920 |
|
| 34,927,790 |
|
| 38,973,060 |
|
| 32,809,365 |
| |||
| Weighted-average shares of common stock outstanding, diluted |
| 41,098,920 |
|
| 34,927,790 |
|
| 38,973,060 |
|
| 32,825,314 |
| |||
(1) Amounts include intangible amortization expense as follows: | |||||||||||||||
| Intangible amortization | $ | 2,582 |
| $ | 1,040 |
| $ | 5,099 |
| $ | 1,815 |
| |||
(2) Amounts include stock-based compensation expense as follows: | |||||||||||||||
| Research and development | $ | 2,626 |
| $ | 2,517 |
| $ | 8,013 |
| $ | 7,519 |
| |||
| Selling, general and administrative |
| 5,759 |
|
| 4,391 |
|
| 17,290 |
|
| 12,592 |
| |||
| Total stock-based compensation | $ | 8,385 |
| $ | 6,908 |
| $ | 25,303 |
| $ | 20,111 |
| |||
| Mirum Pharmaceuticals, Inc. | ||||||||
| Condensed Consolidated Balance Sheet Data | ||||||||
| September 30, 2023 | December 31, 2022 | |||||||
| (unaudited) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ | 303,059 |
| $ | 28,003 |
| ||
| Short-term investments |
| 2,971 |
|
| 123,716 |
| ||
| Accounts receivable |
| 47,834 |
|
| 23,994 |
| ||
| Inventory |
| 22,250 |
|
| 5,565 |
| ||
| Prepaid expenses and other current assets |
| 12,465 |
|
| 8,947 |
| ||
| Total current assets |
| 388,579 |
|
| 190,225 |
| ||
| Restricted cash and cash equivalents |
| - |
|
| 100,000 |
| ||
| Intangible assets, net |
| 258,338 |
|
| 58,954 |
| ||
| Other noncurrent assets |
| 3,725 |
|
| 3,727 |
| ||
| Total assets | $ | 650,642 |
| $ | 352,906 |
| ||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 7,369 |
| $ | 8,690 |
| ||
| Accrued expenses |
| 66,213 |
|
| 54,018 |
| ||
| Other current liabilities |
| 1,082 |
|
| 2,021 |
| ||
| Total current liabilities |
| 74,664 |
|
| 64,729 |
| ||
| Revenue interest liability, net |
| - |
|
| 140,351 |
| ||
| Convertible notes, net |
| 306,022 |
|
| - |
| ||
| Other noncurrent liabilities |
| 875 |
|
| 5,789 |
| ||
| Total liabilities |
| 381,561 |
|
| 210,869 |
| ||
| Commitments and contingencies | ||||||||
| Stockholders' equity: | ||||||||
| Preferred stock |
| - |
|
| - |
| ||
| Common stock |
| 5 |
|
| 4 |
| ||
| Additional paid-in capital |
| 791,114 |
|
| 535,074 |
| ||
| Accumulated deficit |
| (520,580 | ) |
| (392,824 | ) | ||
| Accumulated other comprehensive loss |
| (1,458 | ) |
| (217 | ) | ||
| Total stockholders' equity |
| 269,081 |
|
| 142,037 |
| ||
| Total liabilities and stockholders' equity | $ | 650,642 |
| $ | 352,906 |
| ||
| Last Trade: | US$44.50 |
| Daily Change: | -1.76 -3.80 |
| Daily Volume: | 588,393 |
| Market Cap: | US$2.120B |
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