BEDFORD, Mass.--(BUSINESS WIRE)--Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company pioneering a new way to treat the underlying cause of genetic diseases by precisely upregulating protein expression, today reported financial results for the second quarter of 2020 and provided business updates.

“Today we are announcing that the first patient has been dosed with STK-001, which we believe has the potential to be the first-disease modifying medicine for Dravet syndrome, a severe and progressive genetic epilepsy that is characterized by developmental delays and cognitive impairment, in addition to seizure activity,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “The start of MONARCH also marks Stoke’s official transition to a clinical-stage biotech company. We enter this new stage in a strong financial position to execute on our plans for STK-001 in Dravet syndrome and continue to advance the potential of our TANGO platform for additional genetic diseases.”

Second Quarter 2020 Business Highlights and Recent Developments

• Stoke announced today that the first patient was enrolled and has been dosed with STK-001 in Part A of the Phase 1/2a MONARCH study of children and adolescents ages 2 to 18 years old with Dravet syndrome. Part A of the study is designed to evaluate two dose cohorts of STK-001. The U.S. FDA placed a partial clinical hold on Part B of the study, which is designed to evaluate higher doses of STK-001. Stoke has generated additional data and is in the process of preparing its response to the FDA.
• On June 12, Stoke presented additional data on the use of Stoke’s TANGO technology to address OPA1 protein deficiency at the Association for Research in Vision and Ophthalmology (ARVO). OPA1 protein deficiency is the primary cause of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder.
• On July 9, the journal Nature Communications published data supporting Stoke’s Targeted Augmentation of Nuclear Gene Output (TANGO) approach to addressing severe genetic diseases by precisely upregulating protein expression.
• The BUTTERFLY observational study is ongoing. Despite experiencing a slowing in new patient enrollment earlier this year due to the impact of COVID-19, new patient enrollment has resumed and we believe we have achieved sufficient participation in the study to provide informative data about the natural progression of Dravet syndrome.

Upcoming Anticipated Milestones

• Nomination of a second product candidate for the treatment of an additional genetic disease is expected in the second half of 2020.

Second Quarter and Year-to-Date Results

• Net loss for the three months ended June 30, 2020 were $13.0 million, or $0.39 per share compared to $7.8 million or $1.54 per share for the same period in 2019.
• Research and development expenses for the three months ended June 30, 2020 were $8.0 million, compared to $6.0 million for the same period in 2019.
• General and administrative expenses for the three months ended June 30, 2020 were $5.0 million, compared to $2.4 million for the same period in 2019.
• Net loss for the first six months of 2020 was $24.0 million or $0.73 per share, compared to net loss of $13.6 million or $4.57 per share for the same period in 2019.
• Research and development expenses for the six months ended June 30, 2020 were $15.2 million, compared to $10.2 million for the same period in 2019.
• General and administrative expenses for the six months ended June 30, 2020 were $9.6 million, compared to $4.6 million for the same period in 2019.
• The increase in expenses for the three and six month periods in 2020 over the same periods in 2019 primarily relate to increases in costs associated with personnel, third party contracts, consulting, facilities and others associated with development activities for STK-001, research on additional therapeutics and growing a public corporation.
• As of June 30, 2020, Stoke had approximately $202.1 million in cash, cash equivalents and restricted cash, which is anticipated to fund operations into 2023.

About STK-001

STK-001 is an investigational new medicine for the treatment of Dravet syndrome. Stoke believes that STK-001, a proprietary antisense oligonucleotide (ASO), has the potential to be the first disease-modifying therapy to address the genetic cause of Dravet syndrome. STK-001 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. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-001. STK-001 has been granted orphan drug designation by the FDA as a potential new treatment for Dravet syndrome.

About Phase 1/2a Clinical Study (MONARCH)

The MONARCH study is a Phase 1/2a open-label study of children and adolescents ages 2 to 18 who have an established diagnosis of Dravet syndrome and have evidence of a pathogenic genetic mutation in the SCN1A gene. The primary objectives for the study will be to assess the safety and tolerability of STK-001, as well as to characterize human pharmacokinetics. A secondary objective will be to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 12-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as quality of life as secondary endpoints. Stoke plans to enroll approximately 40 patients across 20 sites in the United States.

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 severe intellectual disabilities, severe developmental disabilities, motor impairment, speech impairment, autism, behavioral difficulties and sleep abnormalities. Compared with the general epilepsy population, people living with Dravet syndrome have a higher risk of sudden unexpected death in epilepsy, or SUDEP. Dravet syndrome affects approximately 35,000 people in the United States, Canada, Japan, Germany, France and the United Kingdom, and it is not concentrated in a particular geographic area or ethnic group.

About Stoke Therapeutics

Stoke Therapeutics (Nasdaq: STOK) is a biotechnology company pioneering a new way to treat the underlying causes of severe genetic diseases by precisely upregulating protein expression to restore target proteins to near normal levels. Stoke aims to develop the first precision medicine platform to target the underlying cause of a broad spectrum of genetic diseases in which the patient has one healthy copy of a gene and one mutated copy that fails to produce a protein essential to health. These diseases, in which loss of approximately 50% of normal protein expression causes disease, are called autosomal dominant haploinsufficiencies. Stoke is headquartered in Bedford, Massachusetts with offices in Cambridge, Massachusetts. For more information, visit https://www.stoketherapeutics.com/ or follow the company on Twitter at @StokeTx.