Stoke Therapeutics Stock

Stoke Therapeutics, Inc. (Stoke) engages in addressing the underlying causes of severe diseases by upregulating protein expression with RNA-based medicines.

Using the company’s proprietary TANGO (Targeted Augmentation of Nuclear Gene Output) approach, the company is developing antisense oligonucleotides (‘ASOs’) to selectively restore protein levels. The company’s first compound, STK-001, is in clinical testing for the treatment of Dravet syndrome, a severe and progressive genetic epilepsy.

The company is also pursuing treatment for a second haploinsufficient disease, autosomal dominant optic atrophy (‘ADOA’), the most common inherited optic nerve disorder. The company’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 the company’s belief in the broad potential for the company’s proprietary approach.

strategy

The company is using its proprietary RNA therapeutics platform to create ASOs for the treatment of severe diseases. The critical components of the company’s strategy are to rapidly advance its lead program, STK-001, to clinical proof-of-concept, approval and commercialization; advance STK-002 to the clinic, for the potential treatment of ADOA; expand the company’s pipeline through internal discovery and collaboration to fully exploit the potential of the company’s proprietary platform (TANGO); maintain broad commercial rights to the company’s product candidates where the company can realize maximum value; and continue to strengthen and expand the company’s intellectual property portfolio.

Proprietary RNA Therapeutics Platform (TANGO)

TANGO (Targeted Augmentation of Nuclear Gene Output) is the company’s proprietary research platform. The company’s initial applications 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 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.

TANGO exploits unique mechanisms for modulation of splicing to prevent the synthesis of naturally occurring non-productive mRNA and increase the synthesis of productive mRNA, resulting in increased production of functional protein.

Human cells naturally regulate protein production to maintain health. Pre-mRNA splicing, including alternative splicing, is an important mechanism used to regulate how much protein and which protein variant is produced. During splicing, introns are removed and exons are joined together to generate the mRNA template that carries the code to synthesize proteins. More than one third of alternative splicing events in mammals do not produce functional proteins and lead to mRNA degradation through nonsense-mediated mRNA decay (‘NMD’). TANGO ASOs act at the pre-mRNA level and prevent non-productive alternative splicing so that the body produces more protein-coding mRNA and thus more protein. This approach is particularly applicable to diseases that are caused by insufficient protein production.

In July 2020, the company published data in the journal Nature Communications that support the company’s proprietary approach to precisely upregulate protein expression using TANGO ASOs. To evaluate the approach broadly, Stoke researchers selected four gene targets that vary in type and abundance of non-productive splicing events, gene size and protein function: PCCA (propionic acidemia); SYNGAP1 (autosomal dominant mental retardation 5); CD274 (autoimmune diseases, including uveitis); and SCN1A (Dravet syndrome). Stoke researchers designed TANGO ASOs to target the non-productive splicing events in these genes and their activity was evaluated. Dose-dependent reductions of non-productive mRNA were observed to lead to increases in both productive mRNA and protein levels for each of the target genes.

Treatment of Autosomal Dominant Haploinsufficiency Diseases with TANGO ASOs

The company is initially focused on applying the transformative potential of the company’s platform to developing precision medicines for autosomal dominant haploinsufficiencies, or disorders in which only one allele of a gene is mutated, resulting in approximately 50% of normal protein expression.

TANGO Mechanisms of Action

The company’s ASOs are specifically designed to bind to a desired RNA sequence inside the nuclei of patients’ cells to prevent the occurrence of non-productive splicing. By doing so, the company’s ASOs decrease the amount of non-productive mRNA and increase the level of productive mRNA, leading to the generation of more protein. TANGO operates in a mutation-independent manner, given it utilizes one wild-type allele, and does not alter protein coding splicing isoforms. The net effect is increased expression of functional protein from the wild-type allele.

The company’s ASOs bind to the pre-mRNA and redirect the splicing machinery to prevent inclusion of the NMD exon. This splice-switching decreases non-productive mRNA and increases productive mRNA, which is translated into increased protein expression from the wild-type allele. In contrast to current exon skipping therapies, which remove a coding exon and result in a truncated protein, the company’s TANGO mechanism skips out a non-coding NMD exon and yields a full-length functional protein.

Approach

The company employs a systematic and capital-efficient approach to develop ASOs for genetically defined patient populations. The company relies on the company’s proprietary database to identify novel drug targets and corroborate these findings with existing knowledge to improve the company’s probability of success in the clinic.

Target Identification

The company continues to make significant investments in its infrastructure to accelerate the pace and scale of target identification. The company has built a significant bioinformatics capability, which includes proprietary bioinformatics algorithms and extensive in-house expertise in whole transcriptome RNA sequencing, also referred to as RNAseq. RNAseq uses next-generation sequencing to determine the quantity and sequences of RNA in a sample. The company leverages large internal datasets of RNAseq from key tissues known to be addressable with antisense, such as the CNS, eye, liver and kidney, that are purpose-built to enhance the capture of non-productive events.

The company employs machine learning to iteratively refine the company’s search and scoring criteria for the most addressable non-productive mRNA elements based on internal target validation and hit identification data. The company’s technology is amenable to a large number of mutations and can thereby potentially provide a single-drug approach for diseases that are caused by many loss-of-function mutations in a single gene. The company has identified approximately 1,200 monogenic, or single gene, diseases containing at least one NMD-inducing nonproductive event, which may be amenable to TANGO. The company’s approach is highly predictive and enables rapid and systematic identification of those targets that are most likely to have clinical relevance, thereby increasing the probability for clinical success and accelerating the expansion of the company’s emerging pipeline.

Hit Identification

Once a TANGO target is validated in cells and tissues that are relevant to the disease, the company employs cell lines to rapidly screen for hit ASOs that can increase the target protein expression by specifically preventing the occurrence of the non-productive event in the target mRNA. ASO arrays utilize clinically translatable previously-validated ASO chemistries, such as 2’ methoxyethyl phosphorothioate and PMO. Hit compounds are evaluated in vivo to identify lead ASOs that possess suitable efficacy and safety to merit preclinical development. Lead ASOs are subsequently evaluated in animal disease models or ex vivo disease model systems.

Lead Evaluation and Prioritization

After the company has identified lead compounds, the company evaluates and prioritizes the advancement of new development candidates based on both program-specific and portfolio-wide considerations. Program-specific criteria include, among other relevant factors, the severity of the unmet medical need, the likelihood of therapeutic utility, the feasibility of clinical development, the costs of development and the commercial opportunity. Portfolio-wide considerations include the ability to demonstrate technical success for the company’s platform, thereby increasing the probability of success and learnings for subsequent programs. The learnings from the company’s lead Dravet syndrome program will significantly reduce the uncertainty of development of subsequent programs in the company’s pipeline, particularly those targeting the CNS.

Clinical Trial and Regulatory Execution

The company employs a multi-pronged approach to bring new product candidates forward as rapidly as possible. The company’s approach leverages previously-validated ASO chemistry and a modality that has been successfully utilized for other diseases, to minimize potential safety concerns and development risk. The company has two ongoing Phase 1/2a open-label studies of STK-001 for Dravet syndrome, MONARCH in the United States and ADMIRAL in the United Kingdom, each with a design and endpoints common to the trials for approved anti-seizure medications (‘ASMs’).

Commercialization

The company intends to retain broad commercial rights and independently bring the company’s therapies to patients through a lean, targeted internal commercial organization where the company can realize maximum value. To do this, the company is focused on ensuring that the company can effectively identify and access those patients who may benefit from the company’s product candidates. The company targets diseases in which genetic testing is routinely performed, thereby shortening the diagnostic odyssey and enabling rapid identification of patients who harbor the relevant genetic mutations. The company has partnered with Invitae, a leading genetic information company, to provide genetic testing for pediatric epilepsy at no cost to patient. Lastly, to maximize patient access, the company intends to leverage an established network of academic and tertiary centers with extensive experience with analogous drug administration.

Therapeutic Focus and Product Candidates

The company’s ASOs can be applied to treat a wide range of severe diseases, and the company has carefully designed and prioritized the company’s pipeline strategy to maximize this opportunity. The company is focused on applying the transformative potential of its TANGO platform to developing medicines for patients with diseases where the genetic abnormality is known and is found in a single gene. The company therefore knows for a given disease precisely which gene will need to be upregulated, thus mitigating against the uncertainty of the disease biology. The company is focused on developing product candidates to treat autosomal dominant haploinsufficiency diseases, or disorders in which one copy of a gene is mutated and results in approximately 50% of normal protein expression. Within haploinsufficiencies, the company is prioritizing genetic diseases of the CNS and the eye for the company’s near-term development efforts.

Development Programs

The company’s technology, development experience and scientific knowledge in the field of biologics, RNA splicing, and antisense oligonucleotide chemistry has enabled the company to build a pipeline of programs targeting the underlying cause of severe diseases.

STK-001 for the Treatment of Dravet Syndrome

STK-001 is an investigational new medicine for the treatment of Dravet syndrome being evaluated in ongoing clinical trials. STK-001 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 protein levels, thereby reducing both occurrence of seizures and significant non-seizure comorbidities.

Preclinical Data

The company has generated compelling preclinical data that demonstrate proof-of-mechanism for STK-001. The company’s initial target engagement, pharmacology and efficacy studies were performed in mice, including both wild-type and a Dravet syndrome mouse model. The targeted non-productive splicing event in SCN1A is highly conserved across multiple species, including mouse, non-human primates and humans. The target sequence for STK-001 is also identical across species.

The company evaluated STK-001 pharmacology and efficacy in transgenic mice with a heterozygous deletion of Scn1a. This model was created by introducing a targeted deletion in the first coding exon of the Scn1a gene; these mice exhibit many aspects of the Dravet syndrome phenotype, including seizures and premature lethality.

The company also investigated the pharmacology, distribution and tolerability of STK-001 in a study with cynomolgus monkeys. As a pilot experiment, this study was not required to be performed under Good Laboratory Practices, or GLP.

Single dose GLP toxicology studies in rats and cynomolgus monkeys, that characterized the pharmacology, exposure and tolerability of STK-001 were included in the investigational new drug application (the ‘IND’) that was submitted to the FDA in late 2019. Additional multiple dose GLP toxicology data have subsequently been submitted to the FDA and the MHRA (UK regulatory agency) to support multiple dosing in the clinic.

Clinical Program and Data

The company designed its lead product candidate, STK-001, to treat Dravet syndrome, a severe and progressive genetic epilepsy. This program draws on a well-defined patient population based on routine genetic testing and learnings from drugs approved for the treatment of Dravet syndrome to inform the clinical and regulatory pathways for STK-001.

MONARCH and ADMIRAL Phase 1/2a Studies

The company has two ongoing Phase 1/2a open-label studies of STK-001, MONARCH in the United States and ADMIRAL in the United Kingdom. The MONARCH study is designed to evaluate single and multiple ascending dose levels of STK-001 administered intrathecally in children and adolescents with Dravet syndrome. Patients are eligible for the trial if they are between the ages of 2 to 18, have an established diagnosis of Dravet syndrome and have evidence of a pathogenic genetic mutation in the SCN1A gene. Requiring an SCN1A mutation for trial enrollment allows for a clear and definitive etiologic diagnosis, a more homogeneous patient population and tailored treatment based on a precision medicine approach. Eligible patients will also have failed at least two epilepsy treatments in the past and be taking at least one ASM. All medications and interventions will remain unchanged throughout the trials, which will allow for assessment of STK-001 with a variety of ASMs.

The primary objectives are the assessment of the safety and tolerability of STK-001, as well as to characterize blood pharmacokinetics (‘PK’) and cerebrospinal fluid (‘CSF’) exposure levels. A secondary objective is to assess the efficacy of STK-001 as an adjunctive ASM treatment with respect to the percent change from baseline in convulsive seizure frequency over a 12-week treatment period. The company is measuring non-seizure aspects of the disease, such as quality of life as secondary endpoints.

In March 2020, the company announced the FDA had placed a partial clinical hold on doses of STK-001 above 20mg in the MONARCH study, pending additional preclinical testing to determine the safety profile of doses higher than the current no observed adverse effect level (‘NOAEL’). When intrathecal doses above the NOAEL were administered to non-human primates (‘NHPs’), adverse hind limb paresis was observed. This finding is known to occur following intrathecal delivery of ASOs to NHPs and is not known to translate to the human experience. When extremely high dose levels were administered, acute convulsions were observed immediately following STK-001 administration. The dose levels were well above the range of corresponding human doses that would ever be administered in the clinic and were delivered in a formulation that was at a higher concentration than would be administered in the clinic. There is no apparent correlation of these acute adverse events with the mechanism of action of STK-001.

Since March 2020, the FDA has agreed to allow the company to add additional higher dose levels to the MONARCH study. The study is permitted to evaluate single doses (10mg, 20mg, 30mg or 45mg) and multiple doses administered monthly for three months (20mg, 30mg or 45mg). Dosing above 45mg in this study remains on FDA partial clinical hold.

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 up to 70mg, as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. 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. The company is measuring non-seizure aspects of the disease, such as overall clinical status and quality of life, as secondary endpoints.

In the second half of 2021, the company announced positive interim safety, PK, CSF exposure, and efficacy data from the MONARCH study based on data from patients who were treated in the 20mg MAD dose cohort, most of whom had received three monthly doses of STK-001, or the single 10mg, 20mg or 30mg dose cohorts. Key findings from the interim analysis included that single doses of STK-001 up to 30mg, and three 20mg doses of STK-001 given every four weeks were found to be well tolerated. The most common treatment-emergent adverse events (‘TEAEs’) were headache, vomiting, seizure, irritability, and back pain.

In November and December 2022, the company announced additional positive interim safety, PK, CSF exposure and efficacy data from the MONARCH and ADMIRAL studies, which, along with other data, was presented at the American Epilepsy Society (AES) 2022 Annual Meeting. The pooled data from these two studies demonstrated that, as of the time of the 2022 interim analysis, single and multiple doses of STK-001 up to 45mg were well-tolerated.

MONARCH and ADMIRAL continue to progress. In the United States, MONARCH enrollment is nearing completion, and dosing is ongoing in the expanded 45mg multiple dose cohort. In the United Kingdom, ADMIRAL enrollment is now complete and dosing is ongoing at the 70mg multiple dose cohort. The company expects to announce preliminary clinical data on 45mg MAD doses of STK-001 in mid-2023 and additional preliminary clinical data on 70mg MAD doses of STK-001 in the second half of 2023.

SWALLOWTAIL and LONGWING Open Label Extension Studies

In January 2021, the company initiated enrollment and dosing in its SWALLOWTAIL Open Label Extension (OLE) study of STK-001 for children and adolescents with Dravet syndrome in the United States, and in May 2022, the company began dosing patients in the company’s LONGWING OLE study of STK-001 for children and adolescents with Dravet syndrome in the United Kingdom. Patients who participated in the MONARCH study in the United States or the ADMIRAL study in the United Kingdom and meet study entry criteria are eligible to continue treatment in SWALLOWTAIL or LONGWING, respectively, both of which are designed to evaluate the long-term safety and tolerability of repeated doses of STK-001. SWALLOWTAIL and LONGWING will also provide valuable information on the preliminary effects of STK-001 on seizures along with non-seizure aspects of the disease, such as quality of life and cognition. Following interactions with regulatory agencies, the company has agreed to limit chronic dosing in SWALLOWTAIL to 30mg and in LONGWING to 45mg.

In December 2021 the company presented initial data from the SWALLOWTAIL study that demonstrated that multiple doses up to 30mg of STK-001 intrathecally every four months appears to be well tolerated. In addition, preliminary data from a small cohort of patients treated in the SWALLOWTAIL OLE was presented at the AES 2022 Annual Meeting and showed the effects of ongoing treatment with 30mg of STK-001, including a safety profile consistent with MONARCH and ADMIRAL; reductions in convulsive seizure frequency that were observed in MONARCH were maintained with ongoing treatment in the SWALLOWTAIL OLE; and a trend toward improvement in non-seizure comorbidities as measured by the BRIEF-P, an assessment of executive function that was one of several exploratory endpoints, was observed among patients in the SWALLOWTAIL OLE, while one-year data from the BUTTERFLY observational study, which did not include treatment with STK-001, showed little change from baseline to 12 months in the mean BRIEF-P Scores and other commonly used cognition assessments.

The company has not yet discussed with regulatory authorities the evidence necessary for approval of STK-001. However, if the company sees evidence of clinical efficacy as the company dose escalate, then the company would plan to meet with regulatory authorities to discuss expedited regulatory pathways.

STK-002 for the Treatment of Autosomal Dominant Optic Atrophy (ADOA)

STK-002 is the company’s lead clinical candidate for the treatment of 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 slow vision loss in patients with ADOA. The company has generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002.

Preclinical Data

The company previously identified a novel exon inclusion event (‘Exon X’) in OPA1 that leads to non-productive mRNA due to introduction of a premature termination codon (‘PTC’). The company’s preclinical studies showed that its ASOs blocked the incorporation of Exon X with consequent dose-dependent increase in productive OPA1 mRNA and protein due to reduction of Exon X-directed non-sense mediated decay (‘NMD’) of OPA1 mRNA. The company has now demonstrated that a single injection of ASO-14 surrogate in the rabbit eye leads to a dose-dependent increase in ASO accumulation in the retina that correlated with an increase in target engagement (removal of Exon X) and an increase in OPA1 protein. The study was conducted using female New Zealand white rabbits that were injected with a single dose of vehicle alone or vehicle containing ASO (n=3/group). On Days 15 and 29, the retinal tissue was collected and analyzed. Retinal exposure of ASO-14 surrogate (ST-1102) was elevated with increased dosing, dose-dependent target engagement was seen at all three time-points examined, and protein increase of OPA1 protein was observed at both Day 15 and Day 29 of the study. The company further showed that in OPA1 haploinsufficient human cells, ASO-14-mediated increase in OPA1 protein translates to improved mitochondrial function as measured by the substantial restoration of ATP levels in the treated cells. ATP is produced by the mitochondria and is the key energy carrying molecule in cells.

In May 2021, the company presented new preclinical efficacy data at the Association for Research in Vision and Ophthalmology (ARVO) Annual Meeting demonstrating that the company’s TANGO ASOs can increase OPA1 protein levels and improve mitochondrial function in human cells derived from ADOA patients with different OPA1 mutations.

In May 2022, the company presented further preclinical data for STK-002 demonstrating in-vivo, dose-related target engagement and OPA1 protein upregulation with sustained effect in NHP retinal tissue following administration of STK-002. Additionally, a dose-related increase in OPA1 protein was detected in retinal ganglion cells of NHPs treated with STK-002.

Clinical Plans

In August 2022, the company announced enrollment of the first patient in its two-year prospective natural history study of people ages 8 to 60 who have a confirmed diagnosis of ADOA that is caused by an OPA1 mutation (FALCON study). The FALCON study is designed to evaluate the rate of change in structural and functional ophthalmic assessments. Data collected from the FALCON study will support the clinical development of STK-002. The company submitted a CTA in the United Kingdom in the first half of 2023 to enable a potential Phase 1/2 trial for STK-002 in 2024.

Additional product opportunities

The company is also advancing additional programs focused on multiple targets, including haploinsufficiency diseases of the CNS and eye. These tissues are affected in many severe genetic diseases.

Acadia License and Collaboration Agreement

In January 2022, the company entered into a license and collaboration agreement with Acadia Pharmaceuticals Inc. (‘Acadia’) for the discovery, development and commercialization of novel RNA-based medicines for the treatment of severe and rare genetic neurodevelopmental diseases of the CNS. The agreement focuses on the targets SYNGAP1, MECP2 (Rett syndrome), and an undisclosed neurodevelopmental target of mutual interest. In connection with each target, the company will collaborate with Acadia to identify potential treatments for further development and commercialization as licensed products. With respect to SYNGAP1, the company has agreed with Acadia to co-develop and co-commercialize licensed products for such target globally, and in connection therewith the company granted to Acadia worldwide, co-exclusive (with the company) licenses for such licensed products. With respect to MECP2 and the neurodevelopmental target, the company granted to Acadia worldwide, exclusive licenses to develop and commercialize licensed products for such targets.

Intellectual Property

With respect to the company’s TANGO platform, the company has exclusively licensed intellectual property for the company’s TANGO technology from the University of Southampton and Cold Spring Harbor Laboratory, which includes issued U.S. and foreign patents and pending U.S. and foreign patent applications that cover the TANGO mechanisms. As of December 31, 2022, the issued U.S. patents, issued foreign patents, pending U.S. patent applications and pending foreign patent applications that the company had licensed from the University of Southampton are anticipated to expire between 2035 and 2036, absent any patent term adjustments or extensions. As of December 31, 2022, the issued U.S. patents, pending U.S. patent applications and pending foreign patent applications that the company had licensed from Cold Spring Harbor Laboratory are anticipated to expire in 2035, absent any patent term adjustments or extensions.

As of December 31, 2022, the issued U.S. patents, the issued foreign patents and any patents that may issue from the pending patent applications, including PCT international applications, U.S. patent applications, and foreign patent applications, were expected to expire between 2036 and 2043, absent any patent term adjustments or extensions.

With respect to STK-001, as of December 31, 2022, the company had exclusively licensed U.S. patents that cover the mechanism of action of STK-001, as well as foreign patents and pending foreign patent applications. The issued patents and any patents that may issue from these pending patent applications are expected to expire between 2035 and 2036, absent any patent term adjustments or extensions. As of December 31, 2022, the company also owned the U.S. patents, pending U.S. patent applications, foreign patents and pending foreign patent applications relating to STK-001, and the U.S. patents any patents that may issue from these pending patent applications are expected to expire between 2038 and 2043, absent any patent term adjustments or extensions.

With respect to STK-002, as of December 31, 2022, the company had exclusively licensed U.S. patents that cover the mechanism of action of STK-002, as well as foreign patents and pending foreign patent applications. The issued patents and any patents that may issue from these pending patent applications are expected to expire between 2035 and 2036, absent any patent term adjustments or extensions. As of December 31, 2022, the company also owned pending U.S. patent applications, and pending foreign patent applications relating to STK-002, and any patents that may issue from these pending patent applications are expected to expire between 2038 and 2041, absent any patent term adjustments or extensions.

The company’s issued patents will likely expire on dates ranging from 2035 to 2038, unless the company receives patent term extension or patent term adjustment, or both. If patents are issued on the company’s pending patent applications, the resulting patents are projected to expire on dates ranging from 2036 to 2043, unless the company receives patent term extension or patent term adjustment, or both.

The company has filed for trademark protection of the ‘Stoke Therapeutics’ mark with the United States Patent and Trademark Office and foreign trademark organizations. The company has registered, and intend to maintain, the trademark ‘Stoke Therapeutics’ in the United States Patent and Trademark Office and in numerous other jurisdictions, including but not limited to the European Union, China, India, and Canada.

License and Research Agreements

In July 2015, the company entered into a worldwide license agreement, or the CSHL Agreement, with CSHL, with respect to TANGO patents. Under the CSHL Agreement, the company receives an exclusive (except with respect to certain government rights and non-exclusive licenses), worldwide license under certain patents and applications relating to TANGO.

In April 2016, the company entered into an exclusive, worldwide license agreement with the University of Southampton (the ‘Southampton Agreement’), whereby the company acquired rights to foundational technologies related to the company’s TANGO technology. Under the Southampton Agreement, the company receives an exclusive, worldwide license under certain licensed patents and applications relating to TANGO.

Research and Development Expenses

The company’s research and development expenses were $77.8 million for the year ended December 31, 2022.

History

The company was founded in 2014. It was incorporated under the laws of the state of Delaware in 2014. The company was formerly known as ASOthera Pharmaceuticals, Inc. and changed its name to Stoke Therapeutics, Inc. in 2016.