UNITED STATES

SECURITIES AND EXCHANGE COMMISSION

WASHINGTON, D.C. 20549

FORM 10-Q

(Mark One)

☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the quarterly period ended September 30, 2022

OR

☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934

For the transition period from ________to ________

Commission File Number: 001-38981

Mirum Pharmaceuticals, Inc.

(Exact name of registrant as specified in its charter)

Delaware	83-1281555
( State or other jurisdiction of incorporation or organization)	(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

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	The Nasdaq Global Market

Indicate by check mark whether the registrant (1) has filed all reports required to be filed by Section 13 or 15(d) of the Securities Exchange Act of 1934 during the preceding 12 months (or for such shorter period that the registrant was required to file such reports), and (2) has been subject to such filing requirements for the past 90 days. Yes  No ☐

Indicate by check mark whether the registrant has submitted electronically every Interactive Data File required to be submitted pursuant to Rule 405 of Regulation S-T (§232.405 of this chapter) during the preceding 12 months (or for such shorter period that the registrant was required to submit such files). Yes ☒ No ☐

Indicate by check mark whether the registrant is a large accelerated filer, an accelerated filer, a non-accelerated filer, a smaller reporting company, or an emerging growth company. See the definitions of “large accelerated filer,” “accelerated filer,” “smaller reporting company,” and “emerging growth company” in Rule 12b-2 of the Exchange Act.

Large accelerated filer	☐	Accelerated filer	☐
Non-accelerated filer	☒	Smaller reporting company	☒
		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. 

Indicate by check mark whether the registrant is a shell company (as defined in Rule 12b-2 of the Exchange Act). Yes ☐ No 

As of November 4, 2022 the registrant had 36,870,380 shares of common stock, $0.0001 par value per share, outstanding.

Table of Contents

		Page
PART I.	FINANCIAL INFORMATION
Item 1.	Financial Statements (Unaudited)	1
	Condensed Consolidated Balance Sheets	1
	Condensed Consolidated Statements of Operations	2
	Condensed Consolidated Statements of Comprehensive Loss	3
	Condensed Consolidated Statements of Stockholders’ Equity	4
	Condensed Consolidated Statements of Cash Flows	6
	Notes to Unaudited Condensed Consolidated Financial Statements	7
Item 2.	Management’s Discussion and Analysis of Financial Condition and Results of Operations	24
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	37
Item 4.	Controls and Procedures	38
PART II.	OTHER INFORMATION
Item 1.	Legal Proceedings	39
Item 1A.	Risk Factors	39
Item 2.	Unregistered Sales of Equity Securities and Use of Proceeds	91
Item 3.	Defaults Upon Senior Securities	91
Item 4.	Mine Safety Disclosures	91
Item 5.	Other Information	91
Item 6.	Exhibits	92
Signatures		93

i

SUMMARY OF RISKS ASSOCIATED WITH OUR BUSINESS

An investment in shares of our common stock involves a high degree of risk. Below is a list of the more significant risks associated with our business. This summary does not address all of the risks that we face. Additional discussion of the risks listed in this summary, as well as other risks that we face, are set forth under Part I, Item 1A, “Risk Factors” in this Quarterly Report on Form 10-Q.

•LIVMARLI® (maralixibat) oral solution (“Livmarli”) is our only approved product and the success of our business depends, in part, on our ability to market and sell Livmarli profitably.

•As a company we currently have limited marketing and sales experience. If we are unable to adequately maintain and scale our marketing and sales capabilities or enter into or maintain rights pursuant to agreements with third parties to market and sell our products, we may not be able to generate viable product revenues. Even if we adequately establish and further maintain such capabilities, market acceptance of our products may be lower than expected.

•Livmarli may fail to achieve the broad degree of physician and patient adoption and use necessary for commercial success.

•We rely completely on third parties to supply, manufacture and distribute drug supplies for Livmarli, including certain sole-source suppliers and manufacturers.

•We have a very limited operating history, and we have incurred significant losses since our inception and anticipate that we will continue to incur significant losses for the foreseeable future.

•Our business depends, in part, on the success of our product candidates, each of which requires significant clinical testing before we can seek regulatory approval and potentially launch commercial sales.

•We have encountered and may continue to encounter delays and difficulties enrolling patients in our clinical trials, and as a result, our clinical development activities could be delayed or otherwise adversely affected.

•Our product candidates are subject to extensive regulation and compliance, which is costly and time consuming, and such regulation may cause unanticipated delays or prevent the receipt of the required approvals to commercialize our product candidates.

•Our clinical trials may fail to adequately demonstrate the safety and efficacy of our product candidates, which could prevent or delay regulatory approval and commercialization.

•Clinical drug development involves a lengthy and expensive process with uncertain outcomes, and results of earlier studies and trials may not be predictive of future trial results.

•Any delays in the commencement or completion, or termination or suspension, of our clinical trials could result in increased costs for us, delay or limit our ability to generate revenue and adversely affect our commercial prospects.

•Our applications for marketing authorization with regulatory authorities may not be accepted or may require additional studies, regulatory actions, or manufacturing requirements to be completed before marketing authorization is granted.

•Even if we obtain regulatory approval for our product candidates, our product candidates may not gain market acceptance among physicians, patients, tertiary care centers, transplant centers and others in the medical community.

•We face significant competition from other biotechnology and pharmaceutical companies with products that may directly or indirectly compete with ours, and our operating results will suffer if we fail to compete effectively.

•We will need substantial additional financing to continue our commercialization efforts for Livmarli, develop our product candidates and implement our operating plans. If we fail to obtain additional financing, we may be forced to delay, reduce or eliminate our product development programs or commercialization efforts.

•We depend on intellectual property licensed from third parties and termination of any of these licenses could result in the loss of significant rights, which would harm our business.

•If we are unable to obtain and maintain sufficient intellectual property protection for Livmarli and our product candidates, or if the scope of the intellectual property protection is not sufficiently broad, our competitors could develop and commercialize products similar or identical to ours, and our ability to successfully commercialize Livmarli and our other product candidates, if approved, may be adversely affected.

ii

PART I—FINANCIAL INFORMATION

Item 1. Financial Statements.

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Balance Sheets

(In thousands, except share and per share data)

	September 30,			December 31,
	2022			2021
	(Unaudited)			(Note 2)
Assets
Current assets:
Cash and cash equivalents	$	35,759		$	31,340
Short-term investments		149,588			125,201
Accounts receivable		16,337			3,267
Inventory		6,943			1,513
Prepaid expenses and other current assets		6,929			5,271
Total current assets		215,556			166,592
Restricted cash equivalents		100,000			100,000
Long-term investments		—			4,983
Property and equipment, net		795			981
Operating lease right-of-use assets		1,254			1,569
Intangible assets, net		45,032			18,740
Other assets		1,710			1,786
Total assets	$	364,347		$	294,651
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	10,548		$	9,166
Accrued expenses		38,311			30,723
Operating lease liabilities		775			711
Derivative liability		1,764			1,996
Total current liabilities		51,398			42,596
Revenue interest liability, net		138,017			129,923
Operating lease liabilities, noncurrent		1,311			1,903
Other liabilities		4,324			17
Total liabilities		195,050			174,439
Commitments and contingencies
Stockholders’ equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized,    and zero shares issued and outstanding as of September 30, 2022    and December 31, 2021, respectively		—			—
Common stock, $0.0001 par value; 200,000,000 shares    authorized; 36,836,024 shares issued and 36,813,754 shares outstanding,    excluding 22,270 shares subject to repurchase as of September 30, 2022;    and 30,705,060 shares issued and 30,582,596 shares outstanding,    excluding 122,464 shares subject to repurchase as of December 31, 2021		4			3
Additional paid-in capital		526,099			377,403
Accumulated deficit		(356,393	)		(257,159	)
Accumulated other comprehensive loss		(413	)		(35	)
Total stockholders’ equity		169,297			120,212
Total liabilities and stockholders’ equity	$	364,347		$	294,651

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

1

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Statements of Operations

(Unaudited)

(In thousands, except share and per share data)

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
Revenue:
Product sales, net	$	18,780		$	—		$	47,156		$	—
License revenue		—			5,000			2,000			16,000
Total revenue		18,780			5,000			49,156			16,000
Operating expenses:
Cost of sales		2,932			—			7,880			—
Research and development		26,217			30,471			75,737			103,653
Selling, general and administrative		22,513			17,353			62,598			40,185
Total operating expenses		51,662			47,824			146,215			143,838
Loss from operations		(32,882	)		(42,824	)		(97,059	)		(127,838	)
Other income (expense):
Interest income		1,352			72			1,714			301
Interest expense		(3,971	)		(5,667	)		(11,620	)		(13,824	)
Change in fair value of derivative liability		—			1,355			232			417
Other (expense) income, net		(192	)		(35	)		953			(565	)
Net loss before income taxes		(35,693	)		(47,099	)		(105,780	)		(141,509	)
Provision (benefit) for income taxes		13			9			(6,546	)		25
Net loss	$	(35,706	)	$	(47,108	)	$	(99,234	)	$	(141,534	)
Net loss per share, basic and diluted	$	(1.02	)	$	(1.55	)	$	(3.03	)	$	(4.68	)
Weighted-average shares of common stock outstanding, basic		34,927,790			30,367,727			32,809,365			30,250,127
Weighted-average shares of common stock outstanding, diluted		34,927,790			30,367,727			32,825,314			30,250,127

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

2

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Statements of Comprehensive Loss

(Unaudited)

(In thousands)

	Three Months Ended September 30,						Nine Months Ended September 30,
	2022			2021			2022			2021
Net loss	$	(35,706	)	$	(47,108	)	$	(99,234	)	$	(141,534	)
Other comprehensive loss:
Unrealized (loss) gain on available-for-sale investments		(191	)		7			(305	)		(83	)
Cumulative translation adjustments		(20	)		—			(73	)		(6	)
Comprehensive loss	$	(35,917	)	$	(47,101	)	$	(99,612	)	$	(141,623	)

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

3

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Statements of Stockholders’ Equity

(Unaudited)

(In thousands, except share and per share data)

	Common Stock				Additional Paid-In		Accumulated			Accumulated Other Comprehensive			Total Stockholders’
	Shares		Amount		Capital		Deficit			Loss			Equity
Balance as of December 31, 2021		30,582,596	$	3	$	377,403	$	(257,159	)	$	(35	)	$	120,212
Issuance of common stock in connection with    equity award plans		100,951		—		1,477		—			—			1,477
Issuance of common stock in at-the-market offerings,    net of issuance costs of $601		995,897		—		17,384		—			—			17,384
Restricted common stock vested in the period		33,398		—		—		—			—			—
Stock-based compensation	—			—		6,561		—			—			6,561
Net loss	—			—		—		(36,606	)		—			(36,606	)
Other comprehensive loss	—			—		—		—			(96	)		(96	)
Balance as of March 31, 2022		31,712,842	$	3	$	402,825	$	(293,765	)	$	(131	)	$	108,932
Issuance of common stock in connection with asset acquisition		609,305		—		15,585		—			—			15,585
Issuance of common stock in connection with    equity award plans		92,593		—		1,408		—			—			1,408
Issuance of common stock in at-the-market offerings,    net of issuance costs of $184		165,018		—		3,905		—			—			3,905
Restricted common stock vested in the period		33,398		—		—		—			—			—
Issuance of common stock in connection with employee stock purchase plan		76,099		—		1,032		—			—			1,032
Stock-based compensation		—		—		6,818		—			—			6,818
Net loss		—		—		—		(26,922	)		—			(26,922	)
Other comprehensive loss		—		—		—		—			(71	)		(71	)
Balance as of June 30, 2022		32,689,255	$	3	$	431,573	$	(320,687	)	$	(202	)	$	110,687
Issuance of common stock in connection with    equity award plans		91,101		—		1,378		—			—			1,378
Issuance of common stock in public offerings,    net of issuance costs of $5,922		4,000,000		1		86,077		—			—			86,078
Restricted common stock vested in the period		33,398		—		—		—			—			—
Stock-based compensation		—		—		7,071		—			—			7,071
Net loss		—		—		—		(35,706	)		—			(35,706	)
Other comprehensive loss		—		—		—		—			(211	)		(211	)
Balance as of September 30, 2022		36,813,754	$	4	$	526,099	$	(356,393	)	$	(413	)	$	169,297

The accompanying notes are an integral part of these unaudited condensed consolidated financial statements.

4

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Statements of Stockholders’ Equity

(Unaudited)

(In thousands, except share and per share data)

	Common Stock				Additional Paid-In		Accumulated			Accumulated Other Comprehensive		Total Stockholders’
	Shares		Amount		Capital		Deficit			Income (Loss)		Equity
Balance as of December 31, 2020		29,776,544	$	3	$	345,180	$	(173,171	)	$	83	$	172,095
Issuance of common stock in connection with   &#