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Mirum Pharmaceuticals Reports Third Quarter Financial Results and Provides Corporate Update

November 6, 2019

FOSTER CITY, Calif.--(BUSINESS WIRE)--Nov 6, 2019--

Mirum Pharmaceuticals, Inc. (NASDAQ: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today reported financial results for the quarter ended September 30, 2019, and provided an update on its clinical programs.

“The first nine months of 2019 have seen great progress toward bringing maralixibat to market for Alagille syndrome and progressive familial intrahepatic cholestasis,” said Chris Peetz, president and chief executive officer of Mirum. “The granting of breakthrough designation by the Food & Drug Administration and an oral late breaking presentation at the Liver Meeting are testimony of the tremendous potential of maralixibat for Alagille syndrome. The next 12 months are poised to be a transformational year as we prepare for commercialization and complete the MARCH-PFIC Phase 3 study.”

Key Operational Highlights

Third Quarter 2019 Financial Results

As of September 30, 2019, Mirum had cash, cash equivalents and investments of $153.6 million.

Upcoming Milestones and Expectations

About Maralixibat

Maralixibat is a novel, minimally-absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases for pediatric populations. Maralixibat inhibits the apical sodium dependent bile acid transporter, which results in more bile acids being excreted in the feces, leading to lower levels of bile acids systemically, thereby potentially reducing bile acid mediated liver damage and related effects and complications. More than 1,500 individuals have received maralixibat, including more than 100 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In a Phase 2 PFIC study, a genetically defined subset of BSEP (bile salt export pump) deficient, or PFIC2, patients responded to maralixibat, which led to maralixibat receiving Breakthrough Therapy designation from the U.S. Food and Drug Administration for PFIC2. In a Phase 2b ALGS study, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. Maralixibat was generally well-tolerated throughout the studies. The most frequent adverse events were diarrhea, abdominal pain and vomiting.

About Mirum Pharmaceuticals

Mirum Pharmaceuticals, Inc. is a clinical-stage biopharmaceutical company focused on the development and commercialization of a late-stage pipeline of novel therapies for debilitating liver diseases. The company’s lead product candidate, maralixibat, is an investigational oral drug in development for progressive familial intrahepatic cholestasis (PFIC) and Alagille syndrome (ALGS). For more information, visit MirumPharma.com. Follow Mirum on Twitter, Facebook and LinkedIn.

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, the results, conduct, progress and timing of Mirum’s clinical trials, and the regulatory approval path for maralixibat. 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 “plans,” “will,” “believes,” “anticipates,” “expects,” “intends,” “goal,” “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, 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. 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.
Selected Consolidated Statement of Operations Data
(in thousands, except per share amounts)
     
    Period from 
 

Three Months Ended

 

Nine Months Ended

 

May 2, 2018 to

 

September 30,

 

September 30,

 

September 30, 2018

 

 

2019

 

 

 

2018

 

 

 

2019

 

 

 

2018

 

     
Operating expenses:    
Research and development 

 

              12,159

 

 

 

                        3

 

 

 $

                  28,611

 

 

 

                        3

 

General and administrative 

 

                3,708

 

 

 

                      62

 

 

 $

                    7,474

 

 

 

                      72

 

Total operating expenses 

 

              15,867

 

 

 

                      65

 

 

 

                     36,085

 

 

 

                      75

 

     
Loss from operations 

 

            (15,867

)

 

 

                    (65

)

 

 

                    (36,085

)

 

 

                    (75

)

Interest income (expense) 

 

                    785

 

 

 

                       (2

)

 

 $

                    1,485

 

 

 

                       (2

)

Other income (expense), net 

 

                       (5

)

 

 

                         -

 

 

 $

                           (1

)

 

 

                         -

 

     
Net loss 

 $

         (15,087

)

 

 $

                 (67

)

 

 $

                (34,601

)

 

 $

                 (77

)

     
Net loss per common share, basic and diluted 

 $

              (0.84

)

 

 $

              (0.14

)

 

 $

                     (4.47

)

 

 $

              (0.26

)

     
Weighted-average common shares outstanding, basic and diluted 

 

      17,996,065

 

 

 

            476,905

 

 

 

                7,745,241

 

 

 

            290,570

 

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

September 30,

 

December 31,

 

2019

 

 

 

2018

 

 
Cash, cash equivalents and investments

$

153,559

 

$

51,963

 

Working capital

 

118,408

 

 

49,526

 

Total assets

 

158,273

 

 

51,975

 

Accumulated deficit

 

(51,949

)

 

(17,348

)

Total stockholders' equity (deficit)

 

146,203

 

 

(17,313

)

 

 

View source version on businesswire.com:https://www.businesswire.com/news/home/20191106005841/en/

CONTACT: Media Contact:

Heidi Chokeir, Ph.D.

Canale Communications

619-203-5391

heidi@canalecomm.comInvestor Contact:

Ian Clements, Ph.D.

650-667-4085

ir@mirumpharma.com

KEYWORD: CALIFORNIA UNITED STATES NORTH AMERICA

INDUSTRY KEYWORD: BIOTECHNOLOGY FDA HEALTH PHARMACEUTICAL CLINICAL TRIALS

SOURCE: Mirum Pharmaceuticals, Inc.

Copyright Business Wire 2019.

PUB: 11/06/2019 04:05 PM/DISC: 11/06/2019 04:05 PM

http://www.businesswire.com/news/home/20191106005841/en