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Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates

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Stoke Therapeutics (NASDAQ: STOK) reported Q3 2024 financial results and business updates. The company ended Q3 with $269.2 million in cash and equivalents. Revenue was $4.9 million, up from $3.3 million in Q3 2023. Net loss was $26.4 million ($0.47 per share) compared to $24.5 million ($0.55 per share) last year. The company plans to share new data on zorevunersen's dosing regimen and complete regulatory discussions for Phase 3 study design by year-end. The FDA has removed the Partial Clinical Hold on zorevunersen, their lead program for Dravet syndrome.

Stoke Therapeutics (NASDAQ: STOK) ha riportato i risultati finanziari del terzo trimestre 2024 e aggiornamenti aziendali. L'azienda ha chiuso il terzo trimestre con 269,2 milioni di dollari in contanti e equivalenti. I ricavi sono stati pari a 4,9 milioni di dollari, in aumento rispetto ai 3,3 milioni di dollari del terzo trimestre 2023. La perdita netta è stata di 26,4 milioni di dollari (0,47 dollari per azione) rispetto ai 24,5 milioni di dollari (0,55 dollari per azione) dell'anno precedente. L'azienda pianifica di condividere nuovi dati sul regime di dosaggio di zorevunersen e di completare le discussioni regolatorie per la progettazione dello studio di Fase 3 entro la fine dell'anno. La FDA ha rimosso il Clinical Hold Parziale su zorevunersen, il loro programma principale per la sindrome di Dravet.

Stoke Therapeutics (NASDAQ: STOK) reportó los resultados financieros del tercer trimestre de 2024 y actualizaciones sobre el negocio. La empresa terminó el tercer trimestre con 269,2 millones de dólares en efectivo y equivalentes. Los ingresos fueron de 4,9 millones de dólares, un aumento desde los 3,3 millones de dólares en el tercer trimestre de 2023. La pérdida neta fue de 26,4 millones de dólares (0,47 dólares por acción) en comparación con los 24,5 millones de dólares (0,55 dólares por acción) del año pasado. La empresa planea compartir nuevos datos sobre el régimen de dosificación de zorevunersen y completar las discusiones regulatorias para el diseño del estudio de Fase 3 para fin de año. La FDA ha levantado la Suspensión Clínica Parcial sobre zorevunersen, su programa principal para el síndrome de Dravet.

스톡 테라퓨틱스 (NASDAQ: STOK)는 2024년 3분기 재무 결과와 사업 업데이트를 보고했습니다. 회사는 3분기를 2억 6천 9백 20만 달러의 현금 및 현금성 자산으로 마감했습니다. 수익은 490만 달러로, 2023년 3분기의 330만 달러에서 증가했습니다. 순손실은 2천 640만 달러 (주당 0.47 달러)로, 작년의 2천 450만 달러 (주당 0.55 달러)와 비교됩니다. 회사는 zorevunersen의 복용 요법에 대한 새로운 데이터를 공유할 계획이며, 해마다 말까지 3상 연구 설계에 대한 규제 논의를 완료할 예정입니다. FDA는 드라베 증후군에 대한 주요 프로그램인 zorevunersen의 부분 임상 보류를 해제했습니다.

Stoke Therapeutics (NASDAQ: STOK) a annoncé les résultats financiers du troisième trimestre 2024 et des mises à jour sur ses activités. L'entreprise a terminé le troisième trimestre avec 269,2 millions de dollars en espèces et équivalents. Le chiffre d'affaires s'est établi à 4,9 millions de dollars, en hausse par rapport aux 3,3 millions de dollars du troisième trimestre 2023. La perte nette s'est élevée à 26,4 millions de dollars (0,47 dollar par action), contre 24,5 millions de dollars (0,55 dollar par action) l'année dernière. L'entreprise prévoit de partager de nouvelles données sur le schéma posologique de zorevunersen et de finaliser les discussions réglementaires concernant la conception de l'étude de Phase 3 d'ici la fin de l'année. La FDA a levé la suspension clinique partielle concernant zorevunersen, leur programme phare pour le syndrome de Dravet.

Stoke Therapeutics (NASDAQ: STOK) hat die finanziellen Ergebnisse des dritten Quartals 2024 und Unternehmensupdates veröffentlicht. Das Unternehmen schloss das dritte Quartal mit 269,2 Millionen Dollar in bar und Barmitteln ab. Der Umsatz betrug 4,9 Millionen Dollar, ein Anstieg von 3,3 Millionen Dollar im dritten Quartal 2023. Der Nettoverlust betrug 26,4 Millionen Dollar (0,47 Dollar pro Aktie) im Vergleich zu 24,5 Millionen Dollar (0,55 Dollar pro Aktie) im letzten Jahr. Das Unternehmen plant, neue Daten zum Dosierungsregime von zorevunersen zu teilen und die regulatorischen Gespräche zum Studiendesign der Phase 3 bis zum Jahresende abzuschließen. Die FDA hat die partielle klinische Sperre für zorevunersen, ihr Hauptprogramm für das Dravet-Syndrom, aufgehoben.

Positive
  • Revenue increased 48.5% YoY to $4.9 million in Q3 2024
  • Strong cash position of $269.2 million
  • FDA removal of Partial Clinical Hold on zorevunersen
  • Loss per share improved from $0.55 to $0.47 YoY
Negative
  • Net loss increased to $26.4 million from $24.5 million YoY
  • R&D expenses increased 9.4% to $22.2 million
  • G&A expenses rose 23.3% to $12.7 million
  • Delay in Phase 1 study of STK-002 for ADOA treatment

Insights

The Q3 results reveal mixed financial signals. Revenue from the Acadia partnership grew 48.5% YoY to $4.9 million, while the net loss increased to $26.4 million. The strong cash position of $269.2 million provides a healthy runway, but operating expenses continue to climb with R&D at $22.2 million and G&A at $12.7 million.

The delay in STK-002's Phase 1 study signals a strategic focus on zorevunersen, their lead candidate. The upcoming year-end data presentation and potential Phase 3 study alignment could be pivotal catalysts. However, the widening operating expenses and increased net loss per share warrant attention, despite the revenue growth from the Acadia collaboration.

The removal of FDA's Partial Clinical Hold on zorevunersen marks a significant regulatory milestone. The company's strategy to present comprehensive data on the 70mg loading/45mg maintenance dosing regimen aligns with their Phase 3 planning. The focus on both seizure control and cognitive improvements demonstrates a holistic approach to Dravet syndrome treatment.

The decision to delay STK-002 for ADOA indicates resource prioritization towards zorevunersen's development. The upcoming AES meeting presentations and regulatory discussions could provide important validation of the treatment's efficacy across multiple disease measures, particularly in behavior and cognition domains.

– Company plans to provide seizure as well as cognition and behavior data from all patients treated with initial 70mg doses followed by 45mg maintenance dosing in studies of zorevunersen by year-end –

– Discussions with FDA and global regulatory agencies related to a single global Phase 3 study of zorevunersen continue to progress; Company to provide an update by year-end –

– As of September 30, 2024, Company had $269.2 million in cash, cash equivalents, and marketable securities –

BEDFORD, Mass.--(BUSINESS WIRE)-- 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 third quarter of 2024 and provided business updates including those related to zorevunersen, 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.

“We are headed into a busy year-end as we prepare to share new data which is representative of our proposed Phase 3 dosing regimen and complete our regulatory discussions toward alignment on that study design,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “Key to our regulatory discussions are the assessments of behavior and cognition in patients with Dravet syndrome. In our studies of zorevunersen, we have demonstrated substantial and sustained effects across multiple measures of disease, starting within the first year of treatment. These data give us confidence in our Phase 3 planning and the potential for zorevunersen to go beyond seizure management by addressing the root cause of the disease.”

The Company announced today that it has submitted abstracts for presentation at the American Epilepsy Society meeting taking place December 6-10, in Los Angeles, California. Included among these planned presentations are new data from all patients treated in the clinical studies with initial 70mg doses followed by 45mg maintenance dosing in studies of zorevunersen. A Phase 3 regimen of two or three loading doses of 70mg followed by maintenance doses of 45mg is currently under discussion with global regulatory agencies. As the Company continues to focus on zorevunersen as its lead program, it will delay the start of the Phase 1 study of STK-002, its clinical candidate for the treatment of autosomal dominant optic atrophy (ADOA).

Recent Program Highlights and Upcoming Milestones

  • In August, the Company announced that the U.S. Food and Drug Administration (FDA) has removed the Partial Clinical Hold on zorevunersen.
  • In September, the Company shared data from the Phase 1/2a and open-label extension (OLE) studies of zorevunersen in children and adolescents with Dravet syndrome at the 15th European Epilepsy Congress (EEC).
  • Company management will present at the Jefferies London Healthcare Conference on Wednesday, November 20, at 6:30am EST (11:30am GMT). A live webcast of the presentation will be available on the Investors & News section of Stoke’s website.
  • The Company expects to present data at the American Epilepsy Society (AES) 2024 Annual Meeting, pending acceptance of the abstracts.
  • The Company plans to provide an update on Phase 3 registrational plans for zorevunersen by year-end.

Third Quarter 2024 Financial Results

  • As of September 30, 2024, the Company had $269.2 million in cash, cash equivalents, and marketable securities.
  • Revenue recognized for upfront license fees and services provided from the License and Collaboration Agreement with Acadia Pharmaceuticals for the three months ended September 30, 2024 was $4.9 million, compared to $3.3 million for the same period in 2023.
  • Net loss for the three months ended September 30, 2024 was $26.4 million, or $0.47 per share, compared to $24.5 million, or $0.55 per share, for the same period in 2023.
  • Research and development expenses for the three months ended September 30, 2024 were $22.2 million, compared to $20.3 million for the same period in 2023.
  • General and administrative expenses for the three months ended September 30, 2024 were $12.7 million, compared to $10.3 million for the same period in 2023.

Year-to-Date 2024 Financial Results

  • Revenue recognized for upfront license fees and services provided from the License and Collaboration Agreement with Acadia Pharmaceuticals for the nine months ended September 30, 2024 was $13.9 million, compared to $6.0 million for the same period in 2023.
  • Net loss for the nine months ended September 30, 2024 was $78.5 million, or $1.48 per share, compared to $77.7 million, or $1.78 per share, for the same period in 2023.
  • Research and development expenses for the nine months ended September 30, 2024 were $65.7 million, compared to $60.5 million for the same period in 2023.
  • General and administrative expenses for the nine months ended September 30, 2024 were $36.0 million, compared to $30.7 million for the same period in 2023.
  • The increase in operating expenses for the three and nine month periods ending September 30, 2024 over the same periods in 2023 primarily relates to increases in costs associated with personnel, third party contracts, consulting, facilities and other costs associated with development activities for zorevunersen and STK-002, research on additional therapeutics and growing a public corporation.

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.

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 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 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 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, including expectations regarding its collaboration with Acadia Pharmaceuticals; 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

Stoke Therapeutics, Inc. and subsidiary

Consolidated balance sheets

(in thousands, except share and per share amounts)

(unaudited)

 

 

 

 

 

 

 

September 30,

 

December 31,

 

 

 

2024

 

 

 

2023

 

Assets
Current assets:
Cash and cash equivalents

$

150,016

 

$

191,442

 

Marketable securities - current

 

89,184

 

 

9,952

 

Prepaid expenses

 

9,837

 

 

11,320

 

Restricted cash - current

 

75

 

 

 

Interest receivable

 

691

 

 

64

 

Other current assets

 

3,809

 

 

2,561

 

Total current assets

$

253,612

 

$

215,339

 

Marketable securities - long-term

 

29,952

 

 

 

Restricted cash - long-term

 

494

 

 

569

 

Operating lease right-of-use assets

 

4,928

 

 

6,611

 

Property and equipment, net

 

4,333

 

 

5,823

 

Total assets

$

293,319

 

$

228,342

 

Liabilities and stockholders’ equity
Current liabilities:
Accounts payable

$

2,485

 

$

1,695

 

Accrued and other current liabilities

 

15,768

 

 

13,815

 

Deferred revenue - current portion

 

31,612

 

 

15,309

 

Total current liabilities

$

49,865

 

$

30,819

 

Deferred revenue - net of current portion

 

8,291

 

 

33,074

 

Other long term liabilities

 

3,050

 

 

4,884

 

Total long term liabilities

 

11,341

 

 

37,958

 

Total liabilities

$

61,206

 

$

68,777

 

Stockholders’ equity
Common stock, par value of $0.0001 per share; 300,000,000 shares authorized, 52,941,191 and 45,918,233 shares issued and outstanding as of September 30, 2024 and December 31, 2023, respectively

 

5

 

 

5

 

Additional paid-in capital

 

712,290

 

 

561,433

 

Accumulated other comprehensive income (loss)

 

166

 

 

(24

)

Accumulated deficit

 

(480,348

)

 

(401,849

)

Total stockholders’ equity

$

232,113

 

$

159,565

 

Total liabilities and stockholders’ equity

$

293,319

 

$

228,342

 

Stoke Therapeutics, Inc. and subsidiary
Consolidated statements of operations and comprehensive loss
(in thousands, except share and per share amounts)
(unaudited)
 

Three Months Ended September 30,

 

Nine Months Ended September 30,

 

2024

 

 

 

2023

 

 

 

2024

 

 

 

2023

 

Revenue

$

4,894

 

$

3,308

 

$

13,941

 

$

5,978

 

Operating expenses:
Research and development

 

22,205

 

 

20,271

 

 

65,710

 

 

60,453

 

General and administrative

 

12,692

 

 

10,271

 

 

35,950

 

 

30,712

 

Total operating expenses

 

34,897

 

 

30,542

 

 

101,660

 

 

91,165

 

Loss from operations

 

(30,003

)

 

(27,234

)

 

(87,719

)

 

(85,187

)

Other income (expense):
Interest income (expense), net

 

3,545

 

 

2,651

 

 

9,668

 

 

7,321

 

Other income (expense), net

 

28

 

 

41

 

 

(448

)

 

125

 

Total other income (expense)

 

3,573

 

 

2,692

 

 

9,220

 

 

7,446

 

Net loss

$

(26,430

)

$

(24,542

)

$

(78,499

)

$

(77,741

)

Net loss per share, basic and diluted

$

(0.47

)

$

(0.55

)

$

(1.48

)

$

(1.78

)

Weighted-average common shares outstanding, basic and diluted

 

56,341,074

 

 

44,266,017

 

 

52,991,015

 

 

43,669,987

 

Comprehensive loss:
Net loss

$

(26,430

)

$

(24,542

)

$

(78,499

)

$

(77,741

)

Other comprehensive gain:
Unrealized gain on marketable securities

 

181

 

 

232

 

 

190

 

 

1,028

 

Total other comprehensive gain

$

181

 

$

232

 

$

190

 

$

1,028

 

Comprehensive loss

$

(26,249

)

$

(24,310

)

$

(78,309

)

$

(76,713

)

 

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

Source: Stoke Therapeutics, Inc.

FAQ

What was Stoke Therapeutics (STOK) Q3 2024 revenue?

Stoke Therapeutics reported revenue of $4.9 million in Q3 2024, compared to $3.3 million in Q3 2023.

What is the cash position of STOK as of Q3 2024?

As of September 30, 2024, Stoke Therapeutics had $269.2 million in cash, cash equivalents, and marketable securities.

What is the status of STOK's zorevunersen FDA clinical hold?

The FDA has removed the Partial Clinical Hold on zorevunersen, as announced by Stoke Therapeutics in August 2024.

What was STOK's net loss per share in Q3 2024?

Stoke Therapeutics reported a net loss of $0.47 per share in Q3 2024, compared to $0.55 per share in Q3 2023.

Stoke Therapeutics, Inc.

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Biotechnology
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