Le Lézard
Classified in: Health, Science and technology, Business
Subjects: EARNINGS, Conference Call, Webcast

Mirum Pharmaceuticals Reports Third Quarter 2021 Financial Results and Provides Business Update


Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), today reported financial results for the quarter ended September 30, 2021, and provided a business update.

"The FDA approval and launch of LIVMARLI capped a transformational third quarter for Mirum and the Alagille syndrome patient community. The launch of LIVMARLI, the first and only approved medication for the treatment of cholestatic pruritus in patients one year of age and older with Alagille syndrome, is off to a strong start," said Chris Peetz, president and chief executive officer at Mirum. "We believe the launch of LIVMARLI, coupled with a strong balance sheet and an advancing late-stage clinical pipeline, positions Mirum for sustained growth as we lead the way in rare liver disease worldwide."

Recent Key Operational Highlights

Financial Results

Upcoming Anticipated Milestones

Business Update Conference Call

Mirum will host a conference call today, November 15, 2021 at 1:30 p.m. PT/4:30 p.m. ET, to provide business updates and discuss data presented today at AASLD. Join the call using the following details:

Conference Call Details:

U.S. toll-free:

844-200-6205

International:

646-904-5544

Passcode:

588077

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 LIVMARLItm (maralixibat) oral solution

LIVMARLItm (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 one year of age and older and is the only FDA-approved medication to treat cholestatic pruritus associated with Alagille syndrome. For more information, please visit LIVMARLI.com.

LIVMARLI is currently being evaluated in late-stage clinical studies in other rare cholestatic liver diseases including progressive familial intrahepatic cholestasis 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.

Prescribing information

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 7?C4 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), intrahepatic cholestasis of pregnancy (OHANA study) and primary biliary cholangitis (VANTAGE study).

About Mirum Pharmaceuticals, Inc.

Mirum Pharmaceuticals, Inc. is a biopharmaceutical company dedicated to transforming the treatment of rare liver diseases. Mirum's approved medication is LIVMARLItm (maralixibat) oral solution which is approved in the U.S. for the treatment of cholestatic pruritus in patients with Alagille syndrome one year of age and older.

Mirum's late-stage pipeline includes two investigational treatments for debilitating liver diseases affecting children and adults. Maralixibat (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 study for progressive familial intrahepatic cholestasis (PFIC) and the EMBARK Phase 2b study for patients with biliary atresia. In addition, Mirum has an expanded access program open in Canada, Australia, the UK and several countries in Europe for eligible patients with Alagille syndrome.

Mirum has submitted a Marketing Authorization Application to the European Medicines Agency for maralixibat for the treatment of cholestatic liver disease in patients with Alagille syndrome.

Mirum's second investigational treatment, volixibat, also an oral IBAT inhibitor, is being evaluated in three registrational studies including the OHANA Phase 2b study for pregnant women with intrahepatic cholestasis of pregnancy, the VISTAS Phase 2b study for adults with primary sclerosing cholangitis, and the VANTAGE Phase 2b study for primary biliary cholangitis.

To augment its pipeline in cholestatic liver disease, Mirum has acquired the exclusive option to develop and commercialize gene therapy programs VTX-803 and VTX-802 for PFIC3 and PFIC2, respectively, from Vivet Therapeutics SAS, following preclinical evaluation and investigational new drug-enabling studies.

Follow Mirum on Twitter, Facebook, LinkedIn and Instagram.

Forward-Looking Statements

This press release includes forward-looking statements pertaining to the Company's planned participation at a scientific conference, which may include discussion of the Company's revenue, business, and operations, including the discovery, development and commercialization of our product candidates and technologies, and the therapeutic potential thereof, the continuation of our clinical trials, and the success of our collaborations with partners and any potential future collaborations. Such forward-looking statements are subject to risks and uncertainties that could cause actual results to differ materially from those expressed or implied in such statements. Applicable risks and uncertainties include those relating to our preclinical research and clinical programs and other risks identified under the heading "Risk Factors" included in our most recent Form 10-Q and Form 10-K filings and in other future filings with the SEC. The forward-looking statements contained in this press release reflect Mirum's current views with respect to future events, and Mirum does not undertake and specifically disclaims any obligation to update any forward-looking statements.

 
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,

 

2021

 

 

 

2020

 

 

 

2021

 

 

 

2020

 

 
License revenue

$

5,000

 

$

-

 

$

16,000

 

$

-

 

 
Operating expenses:
Research and development

 

30,471

 

 

15,984

 

 

103,653

 

 

51,879

 

General and administrative

 

17,353

 

 

5,732

 

 

40,185

 

 

15,466

 

Total operating expenses (1)

 

47,824

 

 

21,716

 

 

143,838

 

 

67,345

 

 
Loss from operations

 

(42,824

)

 

(21,716

)

 

(127,838

)

 

(67,345

)

Other income (expense):
Interest income

 

72

 

 

237

 

 

301

 

 

1,391

 

Interest expense

 

(5,667

)

 

-

 

 

(13,824

)

 

-

 

Change in fair value of derivative liability

 

1,355

 

 

-

 

 

417

 

 

-

 

Other expense, net

 

(35

)

 

(30

)

 

(565

)

 

(109

)

 
Net loss before provision for income taxes

 

(47,099

)

 

(21,509

)

 

(141,509

)

 

(66,063

)

Provision for (benefit from) income taxes

 

9

 

 

(3

)

 

25

 

 

4

 

Net loss

$

(47,108

)

$

(21,506

)

$

(141,534

)

$

(66,067

)

 
Net loss per share, basic and diluted

$

(1.55

)

$

(0.86

)

$

(4.68

)

$

(2.65

)

Weighted-average shares of common stock outstanding, basic and diluted

 

30,367,727

 

 

25,132,916

 

 

30,250,127

 

 

24,965,178

 

 
 

(1) Amounts include stock-based compensation expense as follows:

 
Research and development

$

3,035

 

$

1,361

 

$

7,792

 

$

3,662

 

General and administrative

 

4,380

 

 

2,067

 

 

9,731

 

 

5,313

 

Total stock-based compensation

$

7,415

 

$

3,428

 

$

17,523

 

$

8,975

 

 
 
Mirum Pharmaceuticals, Inc.
Selected Condensed Consolidated Balance Sheet Data
(in thousands)

 

 

 

September 30,

 

December 31,

 

2021

 

 

 

2020

 

(Unaudited)
 
Cash, cash equivalents and short-term investments

$

205,031

 

$

231,820

 

Working capital

 

146,418

 

 

217,888

 

Total assets

 

235,167

 

 

240,864

 

Accumulated deficit

 

(314,705

)

 

(173,171

)

Total stockholders' equity

 

56,396

 

 

172,095

 

 


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