Stoke Therapeutics Reports Third Quarter Financial Results and Provides Business Updates
Stoke Therapeutics, Inc. (Nasdaq: STOK) has nominated STK-002 as a clinical candidate targeting autosomal dominant optic atrophy (ADOA), an inherited optic nerve disorder. The company reported financial results for Q3 2021, showing a net loss of $22.6 million, or $0.61 per share, compared to $13.7 million, or $0.41 per share in 2020. Research and development expenses rose to $14.4 million from $8.1 million year-over-year. With $236.9 million in cash as of September 30, 2021, Stoke expects to fund operations through 2023. Positive interim data from the MONARCH study of STK-001 was also highlighted.
- Nominated STK-002 for ADOA treatment, addressing a significant unmet medical need.
- Positive interim data from MONARCH study supports STK-001 progress.
- Cash reserves of $236.9 million expected to fund operations until end of 2023.
- Net loss increased to $22.6 million in Q3 2021 from $13.7 million in Q3 2020.
- R&D expenses grew significantly to $14.4 million in Q3 2021 from $8.1 million in Q3 2020.
– Company nominates STK-002 as clinical candidate for the treatment of autosomal dominant optic atrophy (ADOA), the most common inherited optic nerve disorder –
– Additional data from an interim analysis of the Phase 1/2a MONARCH study of STK-001 including 20mg multiple ascending dose (MAD) seizure data submitted for presentation at the
– Phase 1/2a studies MONARCH in the
– As of
“The recent positive interim data from the Phase 1/2a MONARCH study in patients with Dravet syndrome continue to support our efforts to develop STK-001 as the first potential new medicine to target the underlying cause of this severe and progressive genetic epilepsy,” said
Third Quarter 2021 Business Updates
- The Company announced positive interim data from the Phase 1/2a MONARCH study of STK-001 in children and adolescents with Dravet syndrome.
- The Phase 1/2a studies MONARCH and ADMIRAL continue to progress with enrollment and dosing of patients ongoing in the 30mg MAD cohorts.
- The FDA will allow the evaluation of an additional higher dose level of STK-001 (45mg) in the MONARCH study.
Recent Developments
-
The Company has nominated STK-002 as the clinical candidate for the treatment of ADOA, a severe and progressive optic nerve disorder. There are currently no approved treatments for ADOA, which is primarily caused by loss-of-function mutations in the OPA1 gene, resulting in
50% OPA1 protein expression and disease manifestation. STK-002, a proprietary antisense oligonucleotide (ASO), is designed to upregulate OPA1 protein expression by leveraging the non-mutant (wild-type) copy of the OPA1 gene. Based on preclinical data generated to date, the Company believes that STK-002 has the potential to be a disease-modifying therapy for people living with ADOA.
Upcoming Anticipated Milestones
-
Additional data from an interim analysis of the Phase 1/2a MONARCH study of STK-001, including 20mg MAD seizure data, were submitted for presentation at the
American Epilepsy Society (AES) 2021 Annual Meeting,December 3-7, 2021 inChicago . - The Company is on track to initiate the collection of natural history data of people living with ADOA by year-end. The data will be used to better understand the natural progression of this disease and to support future clinical development of STK-002 for the treatment of ADOA.
-
The Company is on track to demonstrate in vivo proof of mechanism and safety for a third
TANGO ASO program by the end of 2021. - The Company plans to initiate preclinical studies in 2022 to support future clinical trial applications.
- The Company expects to share preliminary clinical data on 30mg MAD doses of STK-001 in the second half of 2022.
Third Quarter 2021 Financial Results
-
Net loss for the three months ended
September 30, 2021 was$22.6 million $0.61 $13.7 million $0.41 -
Research and development expenses for the three months ended
September 30, 2021 were$14.4 million $8.1 million -
General and administrative expenses for the three months ended
September 30, 2021 were$8.3 million $5.6 million -
Net loss for the nine months ended
September 30, 2021 was$61.4 million $1.67 $37.7 million $1.14 -
Research and development expenses for the nine months ended
September 30, 2021 were$38.4 million $23.3 million -
General and administrative expenses for the nine months ended
September 30, 2021 were$23.2 million $15.2 million - The increase in expenses for the three and nine month periods in 2021 over the same periods in 2020 primarily relate to increases in costs associated with personnel, third party contracts, consulting, facilities and others associated with development activities for STK-001 and STK-002, research on additional therapeutics and growing a public corporation.
-
As of
September 30, 2021 , Stoke had approximately$236.9 million
About TANGO
TANGO (Targeted Augmentation of Nuclear Gene Output) is Stoke’s proprietary research platform. Stoke’s initial application for this technology are diseases in which one copy of a gene functions normally and the other is mutated, also called haploinsufficiencies. In these cases, the mutated gene does not produce its share of protein, so the body does not function normally. Using the TANGO approach and a deep understanding of RNA science, Stoke researchers design antisense oligonucleotides (ASOs) that bind to pre-mRNA and help the target genes produce more protein. TANGO aims to restore missing proteins by increasing – or stoking – protein output from healthy genes, thus compensating for the non-functioning copy of the gene.
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 STK-001
STK-001 is an investigational new medicine for the treatment of Dravet syndrome that is currently being evaluated in ongoing clinical trials. 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 MONARCH Study (
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 are to assess the safety and tolerability of STK-001, as well as to characterize blood pharmacokinetics. A secondary objective is 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 90 patients in the study across 20 sites in
Patients who participated in the MONARCH study are eligible to continue treatment in SWALLOWTAIL, an open label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001. Enrollment and dosing in SWALLOWTAIL are underway.
About Phase 1/2a ADMIRAL Study (
The ADMIRAL 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 genetic mutation in the SCN1A gene. The primary objectives for the study are to assess the safety and tolerability of multiple doses of STK-001, as well as to characterize blood pharmacokinetics. A secondary objective is to assess the effect of multiple doses of STK-001 as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency over a 24-week treatment period. Stoke also intends to measure non-seizure aspects of the disease, such as overall clinical status and quality of life, as secondary endpoints. Stoke plans to enroll up to 60 patients in the study across multiple sites in the
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. Symptoms typically begin between the ages of 4 and 6 years old, affecting males and females equally. The severity of the condition by adolescence reflects the overall level of visual function to be expected throughout most of the individual’s adult life. Roughly half of people with ADOA fail driving standards and up to
About STK-002
STK-002 is a proprietary antisense oligonucleotide (ASO) in preclinical development for the treatment of Autosomal Dominant Optic Atrophy (ADOA). Stoke believes that STK-002 has the potential to be the first disease-modifying therapy for people living with ADOA. 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 reverse vision loss in patients with ADOA. Stoke has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002.
About
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: our quarter-end results and cash runway, our future operating results, financial position and liquidity, the ability of STK-001 to treat the underlying causes of Dravet syndrome and reduce seizures, the ability of STK-002 to treat the underlying causes of ADOA, the timing and expected progress of clinical trials, data readouts and presentations, the timing or receipt of regulatory approvals, the ability of TANGO to design medicines to increase protein production and the expected benefits thereof. Statements including words such as “plan,” “will,” “continue,” “expect,” 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 do not fully materialize or prove incorrect, could cause our results to differ materially from those expressed or implied by such forward-looking statements. Forward-looking statements are subject to risks and uncertainties that may cause the company’s actual activities or results to differ significantly from those expressed in any forward-looking statement, including risks and uncertainties related to the Company’s ability to advance its product candidates, obtain regulatory approval of and ultimately commercialize its product candidates, the timing and results of preclinical and clinical trials, the risk that positive results in a clinical trial may not be replicated in subsequent trials or success 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 intellectual property, the risks associated with the direct and indirect impacts of the ongoing COVID-19 pandemic on our business, and other risks and uncertainties described under the heading “Risk Factors” in documents the company files from time to time with the
Financial Tables Follow
| Condensed consolidated balance sheets | ||||||||
| (in thousands, except share and per share amounts) | ||||||||
| (unaudited) | ||||||||
|
|
|||||||
|
2021 |
|
|
2020 |
|
|||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash and cash equivalents | $ |
149,038 |
|
$ |
287,308 |
|
||
|
87,118 |
|
|
— |
|
|||
| Prepaid expenses and other current assets |
|
8,468 |
|
|
6,435 |
|
||
| Restricted cash - short-term |
|
147 |
|
|
— |
|
||
| Deferred financing costs |
|
117 |
|
|
181 |
|
||
| Interest receivable |
|
45 |
|
|
6 |
|
||
| Total current assets | $ |
244,933 |
|
$ |
293,930 |
|
||
| Restricted cash |
|
569 |
|
|
205 |
|
||
| Operating lease right-of-use assets |
|
5,326 |
|
|
1,115 |
|
||
| Property and equipment, net |
|
2,943 |
|
|
2,675 |
|
||
| Total assets | $ |
253,771 |
|
$ |
297,925 |
|
||
| Liabilities and stockholders’ equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ |
655 |
|
$ |
1,495 |
|
||
| Accrued and other current liabilities |
|
11,229 |
|
|
9,930 |
|
||
| Total current liabilities | $ |
11,884 |
|
$ |
11,425 |
|
||
| Long term liabilities |
|
4,374 |
|
|
422 |
|
||
| Total liabilities | $ |
16,258 |
|
$ |
11,847 |
|
||
| Commitments and contingencies | ||||||||
| Stockholders’ equity | ||||||||
| Common stock, par value of |
|
4 |
|
|
4 |
|
||
| Additional paid-in capital |
|
409,166 |
|
|
396,352 |
|
||
| Accumulated other comprehensive loss |
|
(22 |
) |
|
— |
|
||
| Accumulated deficit |
|
(171,635 |
) |
|
(110,278 |
) |
||
| Total stockholders’ equity | $ |
237,513 |
|
$ |
286,078 |
|
||
| Total liabilities and stockholders’ equity | $ |
253,771 |
|
$ |
297,925 |
|
||
|
||||||||||||||||
| Condensed consolidated statements of operations and comprehensive loss | ||||||||||||||||
| (in thousands, except share and per share amounts) | ||||||||||||||||
| (unaudited) | ||||||||||||||||
| Three Months Ended |
Nine Months Ended |
|||||||||||||||
|
2021 |
|
|
2020 |
|
|
2021 |
|
|
2020 |
|
|||||
| Revenue | $ |
— |
|
$ |
— |
|
$ |
— |
|
$ |
— |
|
||||
| Operating expenses: | ||||||||||||||||
| Research and development |
|
14,358 |
|
|
8,109 |
|
|
38,366 |
|
|
23,293 |
|
||||
| General and administrative |
|
8,325 |
|
|
5,602 |
|
|
23,173 |
|
|
15,165 |
|
||||
| Total operating expenses |
|
22,683 |
|
|
13,711 |
|
|
61,539 |
|
|
38,458 |
|
||||
| Loss from operations |
|
(22,683 |
) |
|
(13,711 |
) |
|
(61,539 |
) |
|
(38,458 |
) |
||||
| Other income: | ||||||||||||||||
| Interest income (expense), net |
|
44 |
|
|
(1 |
) |
|
84 |
|
|
703 |
|
||||
| Other income (expense), net |
|
42 |
|
|
28 |
|
|
98 |
|
|
73 |
|
||||
| Total other income |
|
86 |
|
|
27 |
|
|
182 |
|
|
776 |
|
||||
| Net loss | $ |
(22,597 |
) |
$ |
(13,684 |
) |
$ |
(61,357 |
) |
$ |
(37,682 |
) |
||||
| Net loss per share, basic and diluted | $ |
(0.61 |
) |
$ |
(0.41 |
) |
$ |
(1.67 |
) |
$ |
(1.14 |
) |
||||
| Weighted-average common shares outstanding, basic and diluted |
|
36,759,319 |
|
|
33,273,597 |
|
|
36,706,647 |
|
|
32,954,727 |
|
||||
| Comprehensive loss: | ||||||||||||||||
| Net loss | $ |
(22,597 |
) |
$ |
(13,684 |
) |
$ |
(61,357 |
) |
$ |
(37,682 |
) |
||||
| Other comprehensive loss: | ||||||||||||||||
| Unrealized gain (loss) on marketable securities |
|
20 |
|
|
— |
|
|
(22 |
) |
|
— |
|
||||
| Total other comprehensive loss | $ |
20 |
|
$ |
— |
|
$ |
(22 |
) |
$ |
— |
|
||||
| Comprehensive loss | $ |
(22,577 |
) |
$ |
(13,684 |
) |
$ |
(61,379 |
) |
$ |
(37,682 |
) |
||||
View source version on businesswire.com: https://www.businesswire.com/news/home/20211108005364/en/
Stoke Media & Investor:
Chief Communications Officer
dkalmar@stoketherapeutics.com
781-303-8302
Vice President, Investor Relations
IR@stoketherapeutics.com
617-312-2754
Source:
FAQ
What is the latest financial result for Stoke Therapeutics in Q3 2021?
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What are the ongoing clinical studies for Stoke Therapeutics?
