STOK Stoke Therapeutics Inc

 

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 07, 2024

 

 

Stoke Therapeutics, Inc.

(Exact name of Registrant as Specified in Its Charter)

 

 

Delaware			001-38938	47-1144582
(State or Other Jurisdiction of Incorporation)			(Commission File Number)	(IRS Employer Identification No.)
45 Wiggins Ave
Bedford, Massachusetts				01730
(Address of Principal Executive Offices)				(Zip Code)

 

Registrant’s Telephone Number, Including Area Code: (781) 430-8200

 

(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:

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, $0.0001 par value per share		STOK		Nasdaq Global Select 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. ☐

 

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Item 2.02 Results of Operations and Financial Condition.

On August 7, 2024, Stoke Therapeutics, Inc. (the “Company”) issued a press release announcing its financial results for the quarter ended June 30, 2024. A copy of the press release is attached as Exhibit 99.1 to this report.

Item 9.01 Financial Statements and Exhibits.

(d) Exhibits.

Exhibit Number		Description
99.1		Press release issued by Stoke Therapeutics, Inc. regarding its Q2 2024 financial results, dated August 7, 2024
104		Cover Page Interactive Data File (embedded within the Inline XBRL document)

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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.

			STOKE THERAPEUTICS, INC.
Date:	August 7, 2024	By:	/s/ Thomas E. Leggett
			Thomas E. Leggett Chief Financial Officer

 

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Stoke Therapeutics Reports Second Quarter Financial Results and Provides Business Updates

 

– FDA removes Partial Clinical Hold as company advances toward a Phase 3 registrational study of zorevunersen (STK-001) in children and adolescents with Dravet syndrome –

 

– Company to provide an update on Phase 3 registrational plans for zorevunersen in the second half of 2024 –

 

– Company on track to initiate the Phase 1 study (OSPREY) of STK-002 for the treatment of Autosomal Dominant Optic Atrophy (ADOA) this year –

 

– As of June 30, 2024, Company had $282.0 million in cash, cash equivalents, and marketable securities –

 

 

BEDFORD, Mass., August 7, 2024 – Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine, today reported financial results for the second quarter of 2024 and provided business updates including those related to zorevunersen (STK-001), the Company’s proprietary antisense oligonucleotide (ASO) which is in development by Stoke as the first potential medicine to address the genetic cause of Dravet syndrome.

“The landmark data announced earlier this year provide strong support for zorevunersen as the first potential disease-modifying medicine for the treatment of Dravet syndrome by showing marked reductions in seizures and improvements in cognition and behavior in a heavily treated patient population,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “We continue to receive strong support and hear a great sense of urgency for zorevunersen among the Dravet syndrome community, including families, caregivers and clinicians. We thank the FDA for working with us to remove the Partial Clinical Hold and look forward to continuing our discussions with them and with other global regulatory agencies toward the goal of agreeing on a single, global Phase 3 registrational study design by year-end.”

 

Recent Program Highlights and Upcoming Milestones

 

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Second Quarter 2024 Financial Results

 

Year-to-Date 2024 Financial Results

 

About Dravet Syndrome

Dravet syndrome is a severe and progressive genetic epilepsy characterized by frequent, prolonged and refractory seizures, beginning within the first year of life. Dravet syndrome is difficult to treat and has a poor long-term prognosis. Complications of the disease often contribute to a poor quality of life for patients and their caregivers. The effects of the disease go beyond seizures and often include intellectual disability, developmental delays, movement and balance issues, language and speech disturbances, growth defects, sleep abnormalities, disruptions of the autonomic nervous system and mood disorders. The disease is classified as a developmental and epileptic encephalopathy due to the developmental delays and cognitive impairment associated with the disease. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. There are no approved disease-modifying therapies for people living with Dravet syndrome. One out of 16,000 babies are born with Dravet syndrome, which is not concentrated in a particular geographic area or ethnic group.

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About Zorevunersen (STK-001)

Zorevunersen is an investigational new medicine for the treatment of Dravet syndrome currently being evaluated in ongoing clinical trials. Stoke believes that zorevunersen, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. Zorevunersen is designed to upregulate NaV1.1 protein expression by leveraging the non-mutant (wild-type) copy of the SCN1A gene to restore physiological NaV1.1 levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities. Zorevunersen has been granted orphan drug designation by the FDA and the EMA, and rare pediatric disease designation by the FDA as a potential new treatment for Dravet syndrome.

 

About Autosomal Dominant Optic Atrophy (ADOA)

Autosomal dominant optic atrophy (ADOA) is the most common inherited optic nerve disorder. It is a rare disease that causes progressive and irreversible vision loss in both eyes starting in the first decade of life. Severity can vary and the rate of vision loss can be difficult to predict. Roughly half of people with ADOA fail driving standards and up to 46% are registered as legally blind. More than 400 OPA1 mutations have been reported in people diagnosed with ADOA. Currently there is no approved treatment for people living with ADOA. ADOA affects approximately one in 30,000 people globally with a higher incidence in Denmark of one in 10,000 due to a founder effect.

 

About STK-002

STK-002 is a proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA). Approximately 80% of individuals with ADOA experience symptoms before age 10, typically beginning between the ages of 4 and 6. Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. An estimated 65% to 90% of cases are caused by mutations in the OPA1 gene, most of which lead to a haploinsufficiency resulting in 50% OPA1 protein expression and disease manifestation. STK-002 is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene to restore OPA1 protein expression with the aim to stop or slow vision loss in patients with ADOA. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. STK-002 has been granted orphan drug designation by the FDA as a potential new treatment for ADOA and the company has received authorization of its CTA from the MHRA.

 

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to restoring protein expression by harnessing the body’s potential with RNA medicine. Using Stoke’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, Stoke is developing antisense oligonucleotides (ASOs) to selectively restore protein levels. Stoke’s first compound, zorevunersen (STK-001), is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy. Dravet syndrome is one of many diseases caused by a haploinsufficiency, in which a loss of ~50% of normal protein levels leads to disease. Stoke is

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pursuing the development of STK-002 for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder. Stoke’s initial focus is haploinsufficiencies and diseases of the central nervous system and the eye, although proof of concept has been demonstrated in other organs, tissues, and systems, supporting its belief in the broad potential for its proprietary approach. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/.

 

Cautionary Note Regarding Forward-Looking Statements

This press release contains forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1995, including, but not limited to: the Company’s quarterly results; its future operating results and current or future financial position and liquidity; the ability of zorevunersen (STK-001) to treat the underlying causes of Dravet syndrome and reduce seizures or show improvements in behavior and cognition at the indicated dosing levels or at all; and the timing and expected progress of clinical trials, data readouts, regulatory meetings, regulatory decisions and other presentations. Statements including words such as “expect,” “plan,” “will,” “continue,” or “ongoing” and statements in the future tense are forward-looking statements. These forward-looking statements involve risks and uncertainties, as well as assumptions, which, if they prove incorrect or do not fully materialize, could cause our results to differ materially from those expressed or implied by such forward-looking statements, including, but not limited to, risks and uncertainties related to: the Company’s ability to advance, obtain regulatory approval of, and ultimately commercialize its product candidates, including zorevunersen; the timing of data readouts and interim and final results of preclinical and clinical trials; the receipt and timing of potential regulatory decisions; positive results in a clinical trial may not be replicated in subsequent trials or successes in early stage clinical trials may not be predictive of results in later stage trials; the Company’s ability to fund development activities and achieve development goals; the Company’s ability to protect its intellectual property; the direct or indirect impact of global business, political and macroeconomic conditions, including inflation, interest rate volatility, cybersecurity events, uncertainty with respect to the federal budget, instability in the global banking system and volatile market conditions, and global events, including public health crises, and ongoing geopolitical conflicts, such as the conflicts in Ukraine and the Middle East; and other risks and uncertainties described under the heading “Risk Factors” in the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, its quarterly reports on Form 10-Q, and the other documents it files from time to time with the Securities and Exchange Commission. These forward-looking statements speak only as of the date of this press release, and the Company undertakes no obligation to revise or update any forward-looking statements to reflect events or circumstances after the date hereof.

 

Financial Tables Follow

 

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Stoke Therapeutics, Inc. and subsidiary

Consolidated balance sheets

(in thousands, except share and per share amounts)

(unaudited)

 

		June 30,				December 31,
		2024				2023
Assets
Current assets:
Cash and cash equivalents		$	193,476			$	191,442
Marketable securities			88,506				9,952
Prepaid expenses			10,345				11,320
Restricted cash - short-term			75				—
Interest receivable			305				64
Other current assets			4,287				2,561
Total current assets		$	296,994			$	215,339
Restricted cash - long-term			494				569
Operating lease right-of-use assets			5,499				6,611
Property and equipment, net			4,770				5,823
Total Assets		$	307,757			$	228,342
Liabilities and stockholders’ equity
Current liabilities:
Accounts payable		$	3,945			$	1,695
Accrued and other current liabilities			14,500				13,815
Deferred revenue - current portion			26,051				15,309
Total current liabilities		$	44,496			$	30,819
Deferred revenue - net of current portion			16,946				33,074
Other long term liabilities			3,606				4,884
Total long term liabilities			20,552				37,958
Total liabilities		$	65,048			$	68,777
Stockholders’ equity
Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 52,305,641 and 45,918,233 shares issued and outstanding as of June 30, 2024 and December 31, 2023, respectively			5				5
Additional paid-in capital			696,637				561,433
Accumulated other comprehensive loss			(15	)			(24	)
Accumulated deficit			(453,918	)			(401,849	)
Total stockholders’ equity		$	242,709			$	159,565
Total liabilities and stockholders’ equity		$	307,757			$	228,342

 

 

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Stoke Therapeutics, Inc. and subsidiary

Consolidated statements of operations and comprehensive loss

(in thousands, except share and per share amounts)

(unaudited)

 

		Three Months Ended June 30,								Six Months Ended June 30,
		2024				2023				2024				2023
Revenue		$	4,831			$	(2,481	)		$	9,048			$	2,671
Operating expenses:
Research and development			21,136				20,551				43,504				40,182
General and administrative			13,037				10,230				23,258				20,442
Total operating expenses			34,173				30,781				66,762				60,624
Loss from operations			(29,342	)			(33,262	)			(57,714	)			(57,953	)
Other income (expense):
Interest income (expense), net			3,695				2,567				6,121				4,670
Other income (expense), net			(48	)			41				(476	)			84
Total other income (expense)			3,647				2,608				5,645				4,754
Net loss		$	(25,695	)		$	(30,654	)		$	(52,069	)		$	(53,199	)
Net loss per share, basic and diluted		$	(0.46	)		$	(0.69	)		$	(1.02	)		$	(1.23	)
Weighted-average common shares outstanding, basic    and diluted			55,765,948				44,188,464				51,288,222				43,367,032
Comprehensive loss:
Net loss		$	(25,695	)		$	(30,654	)		$	(52,069	)		$	(53,199	)
Other comprehensive gain (loss):
Unrealized gain (loss) on marketable securities			(15	)			219				9				796
Total other comprehensive gain (loss)		$	(15	)		$	219			$	9			$	796
Comprehensive loss		$	(25,710	)		$	(30,435	)		$	(52,060	)		$	(52,403	)

 

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Stoke Media & Investor Contacts:

Dawn Kalmar

Chief Communications Officer

dkalmar@stoketherapeutics.com

781-303-8302

 

Doug Snow

Director, Communications & Investor Relations

IR@stoketherapeutics.com

508-642-6485

 

 

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v3.24.2.u1

Document And Entity Information	Aug. 07, 2024
Cover [Abstract]
Document Type	8-K
Amendment Flag	false
Document Period End Date	Aug. 07, 2024
Entity Registrant Name	Stoke Therapeutics, Inc.
Entity Central Index Key	0001623526
Entity Emerging Growth Company	true
Entity File Number	001-38938
Entity Incorporation, State or Country Code	DE
Entity Tax Identification Number	47-1144582
Entity Address, Address Line One	45 Wiggins Ave
Entity Address, City or Town	Bedford
Entity Address, State or Province	MA
Entity Address, Postal Zip Code	01730
City Area Code	(781)
Local Phone Number	430-8200
Written Communications	false
Soliciting Material	false
Pre-commencement Tender Offer	false
Pre-commencement Issuer Tender Offer	false
Entity Ex Transition Period	false
Title of 12(b) Security	Common Stock, $0.0001 par value per share
Trading Symbol	STOK
Security Exchange Name	NASDAQ

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