Mirum Pharmaceuticals, Inc. [MIRM] Filings

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10-Q 1 mirm-20230930.htm 10-Q 10-Q

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, 2023


☐	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

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.

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 

The number of shares of registrant’s common stock, par value $0.0001 per share, outstanding as of October 27, 2023 was 46,614,274.

Table of Contents

SUMMARY OF RISKS ASSOCIATED WITH OUR BUSINESS


		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	28
Item 3.	Quantitative and Qualitative Disclosures About Market Risk	41
Item 4.	Controls and Procedures	42
PART II.	OTHER INFORMATION	43
Item 1.	Legal Proceedings	43
Item 1A.	Risk Factors	43
Item 2.	Unregistered Sales of Equity Securities, Use of Proceeds, and Issuer Purchases of Equity Securities	93
Item 3.	Defaults Upon Senior Securities	93
Item 4.	Mine Safety Disclosures	93
Item 5.	Other Information	93
Item 6.	Exhibits	94
Signatures		96

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.

•

The success of our business depends, in part, on our ability to market and sell our approved medicines profitably.

•

If we are unable to adequately grow, 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 approved medicines, we may not be able to generate sufficient revenues to be viable.

•

Our approved medicines or any one of our product candidates, if approved, may fail to achieve the market acceptance among physicians, patients and others in the medical community necessary for commercial success.

•

We rely completely on third parties to manufacture and distribute our clinical and commercial drug supplies, including certain sole-source suppliers and manufacturers. These third parties may fail to obtain and maintain regulatory approval for their facilities, fail to provide us with sufficient quantities of drug substance, drug product, or labeled finished product in a timely fashion, or fail to do so at acceptable quality levels or prices.

•

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 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 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.

•

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 may fail to realize all of the anticipated benefits of the Bile Acid Portfolio Acquisition (defined below) or those benefits may take longer to realize than expected.

•

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.

•

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

•

We do not currently have patent protection or regulatory exclusivity for certain of our approved medicines or may rely solely upon regulatory exclusivity. If we are unable to obtain and maintain sufficient intellectual property protection for our approved medicines 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 our approved medicines and our other product candidates, if approved, may be adversely affected.

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,
	2023			2022
	(Unaudited)			(Note 2)
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 equivalents		—			100,000
Property and equipment, net		787			914
Operating lease right-of-use assets		1,428			1,431
Intangible assets, net		258,338			58,954
Other assets		1,510			1,382
Total assets	$	650,642		$	352,906
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable	$	7,369		$	8,690
Accrued expenses		66,213			54,018
Operating lease liabilities		1,082			931
Derivative liability		—			1,090
Total current liabilities		74,664			64,729
Revenue interest liability, net		—			140,351
Operating lease liabilities, noncurrent		868			1,257
Convertible notes payable, net		306,022			—
Other liabilities		7			4,532
Total liabilities		381,561			210,869
Commitments and contingencies
Stockholders’ equity:
Preferred stock, $0.0001 par value; 10,000,000 shares authorized, and no shares issued and outstanding as of September 30, 2023 and December 31, 2022		—			—
Common stock, $0.0001 par value; 200,000,000 shares authorized; 46,595,745 and 36,956,345 shares issued and outstanding as of September 30, 2023 and December 31, 2022, respectively		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

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

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,
	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		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		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 from termination of revenue interest purchase agreement		—			—			(49,076	)		—
Other income (expense), net		1,322			(192	)		237			953
Net loss before provision for (benefit from) income taxes		(23,339	)		(35,693	)		(126,990	)		(105,780	)
Provision for (benefit from) 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

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

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Statements of Comprehensive Loss

(Unaudited)

(In thousands)


	Three Months Ended September 30,						Nine Months Ended September 30,
	2023			2022			2023			2022
Net loss	$	(23,588	)	$	(35,706	)	$	(127,756	)	$	(99,234	)
Other comprehensive income (loss):
Unrealized gain (loss) on available-for-sale investments		11			(191	)		230			(305	)
Cumulative translation adjustments		(1,545	)		(20	)		(1,471	)		(73	)
Comprehensive loss	$	(25,122	)	$	(35,917	)	$	(128,997	)	$	(99,612	)

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

Mirum Pharmaceuticals, Inc.

Condensed Consolidated Statements of Stockholders’ Equity

(Unaudited)

(In thousands, except share amounts)


	Common Stock				Additional Paid-In		Accumulated			Accumulated Other Comprehensive			Total Stockholders’
	Shares		Amount		Capital		Deficit			Income (Loss)			Equity
Balance as of December 31, 2022		36,956,345	$	4	$	535,074	$	(392,824	)	$	(217	)	$	142,037
Issuance of common stock in connection with equity award plans		197,703		—		1,390		—			—			1,390
Issuance of common stock in at-the-market offerings, net of issuance costs of $518		658,206		—		14,480		—			—			14,480
Issuance of common stock in connection with achievement of Contingent Milestone		199,993		—		4,292		—			—			4,292
Stock-based compensation		—		—		8,728		—			—			8,728
Net loss		—		—		—		(30,130	)		—			(30,130	)
Other comprehensive income		—		—		—		—			353			353
Balance as of March 31, 2023		38,012,247		4		563,964		(422,954	)		136			141,150
Issuance of common stock in connection with equity award plans		101,699		—		803		—			—			803
Issuance of common stock in connection with employee stock purchase plan		76,481		—		1,294		—			—			1,294
Issuance of common stock in connection with settlement of Indemnification Holdback liability		31,631		—		896		—			—			896
Stock-based compensation		—		—		8,565		—			—			8,565
Net loss		—		—		—		(74,038	)		—			(74,038	)
Other comprehensive loss		—		—		—		—			(60	)		(60	)
Balance as of June 30, 2023		38,222,058		4		575,522		(496,992	)		76			78,610
Issuance of common stock in private placement, net of issuance costs of $7,761		8,000,000		1		202,238		—			—			202,239
Issuance of common stock in connection with equity award plans		373,687		—		4,766		—			—			4,766
Stock-based compensation		—		—		8,588		—			—			8,588
Net loss		—		—		—		(23,588	)		—			(23,588	)
Other comprehensive loss		—		—		—		—			(1,534	)		(1,534	)
Balance as of September 30, 2023		46,595,745	$	5	$	791,114	$	(520,580	)	$	(1,458	)	$	269,081


	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		—

Mirum Pharmaceuticals, Inc. [MIRM] Filings
10-Q	[2023-11-02]
10-Q	[2023-08-07]
10-Q	[2023-05-04]
10-K	[2023-03-08]
10-Q	[2022-11-09]
10-Q	[2022-08-04]
10-Q	[2022-05-05]
10-K	[2022-03-09]
10-Q	[2021-11-15]
10-Q	[2021-08-05]
10-Q	[2021-05-06]
10-K	[2021-03-09]
10-Q	[2020-11-12]
10-Q	[2020-08-06]
10-Q	[2020-05-07]
10-K	[2020-03-12]
S-1	[2020-01-06]
10-Q	[2019-11-06]
10-Q	[2019-08-28]
S-1	[2019-06-21]