mirm-8k_20200806.htm

 

 

UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

Washington, D.C. 20549

 

FORM 8-K

 

CURRENT REPORT

Pursuant to Section 13 or 15(d)

of the Securities Exchange Act of 1934

Date of Report (Date of earliest event reported): August 6, 2020

 

Mirum Pharmaceuticals, Inc.

(Exact name of registrant as specified in its charter)

 

 

Delaware

 

001-38981

 

83-1281555

(State or other jurisdiction

of incorporation)

 

(Commission

File Number)

 

(I.R.S. Employer

Identification No.)

 

950 Tower Lane, Suite 1050

Foster City, California

 

 

 

94404

(Address of principal executive offices)

 

 

 

(Zip Code)

 

Registrant’s telephone number, including area code: (650) 667-4085

(Former name or former address, if changed since last report.)

 

Check the appropriate box below if the Form 8-K filing is intended to simultaneously satisfy the filing obligation of the registrant under any of the following provisions:

 

 

Written communications pursuant to Rule 425 under the Securities Act (17 CFR 230.425)

 

 

 

 

Soliciting material pursuant to Rule 14a-12 under the Exchange Act (17 CFR 240.14a-12)

 

 

 

 

Pre-commencement communications pursuant to Rule 14d-2(b) under the Exchange Act (17 CFR 240.14d-2(b))

 

 

 

 

Pre-commencement communications pursuant to Rule 13e-4(c) under the Exchange Act (17 CFR 240.13e-4(c))

 

Securities registered pursuant to Section 12(b) of the Act:

 

Title of each class

 

Trading

Symbol(s)

 

Name of each exchange

on which registered

Common stock, par value $0.0001 per share

 

MIRM

 

Nasdaq Global Market

 

Indicate by check mark whether the registrant is an emerging growth company as defined in Rule 405 of the Securities Act of 1933 (§ 230.405 of this chapter) or Rule 12b–2 of the Securities Exchange Act of 1934 (§ 240.12b–2 of this chapter).

Emerging growth company  

If an emerging growth company, indicate by check mark if the registrant has elected not to use the extended transition period for complying with any new or revised financial accounting standards provided pursuant to Section 13(a) of the Exchange Act.  

 

 

 

 


 

Item 2.02Results of Operations and Financial Condition.

On August 6, 2020, Mirum Pharmaceuticals, Inc. (the “Company”) issued a press release providing a corporate update and announcing its financial results for the quarter ended June 30, 2020. The full text of the press release is furnished as Exhibit 99.1 to this Current Report on Form 8-K and is incorporated herein by reference.

The information in this Current Report on Form 8-K (including Exhibit 99.1) shall not be deemed “filed” for purposes of Section 18 of the Securities Exchange Act of 1934, as amended (the “Exchange Act”), or otherwise subject to the liabilities of that Section, nor shall it be deemed to be incorporated by reference into any filing of the Company under the Securities Act of 1933, as amended, or the Exchange Act, except as expressly set forth by specific reference in such filing.

Item 9.01Financial Statements and Exhibits.

(d) Exhibits.

 

Exhibit

No.

 

Description

 

 

99.1

 

Press Release dated August 6, 2020

 

 


 

SIGNATURES

Pursuant to the requirements of the Securities Exchange Act of 1934, the registrant has duly caused this report to be signed on its behalf by the undersigned hereunto duly authorized.

 

 

 

Mirum Pharmaceuticals, Inc.

 

 

 

 

 

Date: August 6, 2020

 

By:

 

/s/ Christopher Peetz

 

 

 

 

Christopher Peetz

 

 

 

 

President and Chief Executive Officer

 

 

mirm-ex991_6.htm

Exhibit 99.1

 

Mirum Pharmaceuticals Announces Second Quarter 2020 Financial Results and Provides Clinical Program Updates

 

 

-

Rolling NDA submission for maralixibat in Alagille syndrome (ALGS) to be initiated in third quarter 2020; planning for potential launch in second half of 2021.

 

-

Submission of European marketing authorization application for maralixibat in PFIC2 planned for fourth quarter of 2020.

 

-

Expanded Access Program for maralixibat in ALGS planned to open for registration in September 2020.

 

-

Orphan Drug Designation received by European Medicines Agency for maralixibat in biliary atresia.

 

-

Cash, cash equivalents and investments of $149.3 million.

 

FOSTER CITY, Calif. – August 6, 2020 - Mirum Pharmaceuticals, Inc. (Nasdaq: MIRM), a biopharmaceutical company focused on the development and commercialization of novel therapies for debilitating liver diseases, today announced financial results and business updates for the quarter ended June 30, 2020.

 

“We made significant progress toward making maralixibat available as an alternative to liver transplantation in devastating pediatric cholestatic settings in the United States and Europe,” said Chris Peetz, president and chief executive officer at Mirum. “In the United States, we are initiating a rolling NDA submission for maralixibat in ALGS and are planning to start an expanded access program broadening availability to this important medicine for eligible patients. By the end of this year, we intend to submit a marketing authorization application for maralixibat in PFIC2 in Europe, dramatically shifting our timeline for a potential launch in this region.”

 

Second Quarter Highlights

 

Released maralixibat data demonstrating five-year transplant-free survival for pediatric patients with PFIC2. Data will be presented at the upcoming Digital International Liver Congress, August 27-29, 2020.

 

Completed pre-submission meeting with European regulators resulting in feedback enabling submission of a marketing authorization application (MAA) for maralixibat in the treatment of patients with PFIC2 in Europe in the fourth quarter of 2020.

 

Investigational new drug (IND) application cleared by FDA for the initiation of a Phase 2 study evaluating volixibat in adult patients with pruritus associated with primary sclerosing cholangitis.

 

Received orphan drug designation in Europe for maralixibat in the study of patients with biliary atresia; planning for a Phase 2 study is underway.

 

As of June 30, 2020, Mirum had cash, cash equivalents and investments of $149.3 million.

 

Upcoming Anticipated Program Milestones

 

Maralixibat in ALGS:

 

o

Initiation of rolling new drug application (NDA) submission for maralixibat in ALGS on track for third quarter of 2020.

 

Expect to complete NDA submission in the first quarter of 2021.

 

Planning for launch in ALGS in the second half of 2021.

 

o

Maralixibat ALGS Expanded Access Program planned to open for registration in the U.S. and Canada in September 2020.

 

Maralixibat in PFIC:

 

o

Submission of MAA to European regulators for maralixibat in the treatment of patients with PFIC2 on track for fourth quarter of 2020.

 

o

Enrollment ongoing in Phase 3 MARCH clinical trial in PFIC.


 

Maralixibat in biliary atresia:

 

o

Phase 2 study initiation planned.

 

Volixibat:

 

o

Planned expansion into adult patients with studies in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy.

 

Digital International Liver Congress™ 2020

New findings from maralixibat studies will be presented in a late-breaker and poster sessions at the Digital International Liver Congress 2020, August 27-29, 2020. Featured presentations to include the following abstracts:

 

Late-breaker Oral Presentation

August 29, 2020 – 1:45-2:00pm CET

Oral Presentation LB008: Serum bile acid control in long-term maralixibat-treated patients is associated with native liver survival in children with progressive familial intrahepatic cholestasis due to bile salt export pump deficiency.

 

Poster Presentations

August 28, 2020

Poster FRI-131: Psychometric evaluation of the adult itch reported outcome tool, a worst-itch numeric rating scale in adults with cholestatic liver disease.

 

August 28, 2020

Poster FRI-296: Differential expression of bile acid subspecies with maralixibat treatment in pruritus responders.

 

About Maralixibat

Maralixibat is a novel, minimally absorbed, orally administered investigational drug being evaluated in several rare cholestatic liver diseases. Maralixibat inhibits the apical sodium dependent bile acid transporter, resulting 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 1,600 individuals have received maralixibat, including more than 120 children who have received maralixibat as an investigational treatment for Alagille syndrome (ALGS) and progressive familial intrahepatic cholestasis (PFIC). In the ICONIC Phase 2b ALGS clinical trial, patients taking maralixibat had significant reductions in bile acids and pruritus compared to placebo. In a Phase 2 PFIC study, a genetically defined subset of BSEP (bile salt export pump) deficient (PFIC2), patients responded to maralixibat. The FDA has granted maralixibat Breakthrough Therapy designation for pruritus associated with ALGS in patients one year of age and older and for PFIC2. Maralixibat was generally well-tolerated throughout the studies. The most frequent adverse events were diarrhea, abdominal pain, and vomiting. For more information about the North American Expanded Access Program please visit ALGSEAP.com. For more information about the Phase 3 study for maralixibat in pediatric patients with PFIC, visit PFICtrial.com.

 

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 Alagille syndrome (ALGS), progressive familial intrahepatic cholestasis (PFIC), and biliary atresia. The company is also developing volixibat, also an oral ASBT-inhibitor, in primary sclerosing cholangitis and intrahepatic cholestasis of pregnancy. 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 ongoing and planned studies for maralixibat and volixibat, as well as Mirum’s Expanded Access Program for


maralixibat, the regulatory approval path for maralixibat and volixibat, the strength of Mirum’s balance sheet and the adequacy of cash, cash equivalents and investments on hand, the impacts of the COVID-19 pandemic, and commercial readiness activities. 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, 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)

 

 

 

Three Months Ended June 30,

 

 

Six Months Ended June 30,

 

 

 

 

2020

 

 

2019

 

 

2020

 

 

2019

 

 

 

 

(Unaudited)

 

 

(Unaudited)

 

 

Operating expenses:

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

18,555

 

 

$

11,589

 

 

$

35,895

 

 

$

16,452

 

 

General and administrative

 

 

5,042

 

 

 

2,445

 

 

 

9,734

 

 

 

3,766

 

 

Total operating expenses (1)

 

 

23,597

 

 

 

14,034

 

 

 

45,629

 

 

 

20,218

 

 

Loss from operations

 

 

(23,597

)

 

 

(14,034

)

 

 

(45,629

)

 

 

(20,218

)

 

Interest income

 

 

405

 

 

 

468

 

 

 

1,154

 

 

 

700

 

 

Other income (expense), net

 

 

(56

)

 

 

9

 

 

 

(79

)

 

 

4

 

 

Net loss before provision for income taxes

 

 

(23,248

)

 

 

(13,557

)

 

 

(44,554

)

 

 

(19,514

)

 

Provision for income taxes

 

 

3

 

 

 

 

 

 

7

 

 

 

 

 

Net Loss

 

$

(23,251

)

 

$

(13,557

)

 

$

(44,561

)

 

$

(19,514

)

 

Net loss per share, basic and diluted

 

$

(0.93

)

 

$

(5.31

)

 

$

(1.79

)

 

$

(7.70

)

 

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

 

 

25,056,123

 

 

 

2,551,822

 

 

 

24,880,387

 

 

 

2,534,877

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

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

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

Research and development

 

$

1,260

 

 

$

638

 

 

$

2,301

 

 

$

709

 

 

General and administrative

 

 

1,714

 

 

 

1,003

 

 

 

3,246

 

 

 

1,150

 

 

Total stock-based compensation

 

$

2,974

 

 

$

1,641

 

 

$

5,547

 

 

$

1,859

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 

 



Mirum Pharmaceuticals, Inc.

Selected Consolidated Balance Sheet Data

(in thousands)

 

 

 

June 30,

2020

 

 

December 31,

2019

 

 

 

(Unaudited)

 

 

 

 

 

Cash, cash equivalents and investments

 

$

149,336

 

 

$

139,952

 

Working capital

 

 

135,495

 

 

 

106,287

 

Total assets

 

 

155,766

 

 

 

146,712

 

Accumulated deficit

 

 

(114,462

)

 

 

(69,901

)

Total stockholders' equity

 

 

136,208

 

 

 

130,349

 

 

Contacts

 

Investor Contact:

Ian Clements, Ph.D.

ir@mirumpharma.com

 

Media Contact:

Erin Murphy

media@mirumpharma.com