Stoke Therapeutics: Positive Dravet Data Sends Shares Soaring, One For The Watchlist

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Stoke Therapeutics' stock has surged after positive data from studies of its lead drug candidate, STK-001, in children with Dravet syndrome.Dravet syndrome is a severe form of epilepsy that causes ...

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An opportunity in ophthalmology and a partnership with Acadia Pharmaceuticals add further value to the company.   tadamichi Investment Overview Stoke Therapeutics' (NASDAQ:STOK) stock has been soaring in response to the positive data the company shared from two open-label Phase 1/2a studies and two open-label extension (\"OLE\") studies of its lead drug candidate STK-001 in children and adolescents ages 2 to 18 with Dravet syndrome, on March 25th this year. Stoke, which raised ~$142m in its 2019 IPO, at $18 per share, leverages its RNA therapeutics platform TANGO (\"Targeted Augmentation of Nuclear Gene Output\") to create antisense oligonucleotides (\"ASO\"), and STK-001 is its most advanced asset to date. According to Stoke's 2023 annual report / 10K submission:  Dravet syndrome is characterized by multiple seizure types and may progress to status epilepticus or prolonged seizures lasting more than five minutes that require immediate intervention. Patients typically experience their first seizure before 12 months of age. More than 90% of patients suffer from at least one non-seizure comorbidity, including severe intellectual and developmental disabilities, motor and speech impairment, autism, attention deficit hyperactivity disorder and behavioral difficulties. After eight years of age, nearly all Dravet syndrome patients exhibit evidence of substantial developmental delay. The symptoms of the disease result in remarkably low quality of life and shortened life expectancy, and as a result impose an immense burden on individuals and families. The incidence of Dravet syndrome is approximately 64 per million births, which translates to an overall prevalence of approximately 35,000 patients across the United States, Canada, Japan, Germany, France and the United Kingdom, with approximately 16,000 patients in the United States.  Stoke says that \"current treatment strategy involves the use of multiple anti-seizure medications (\"ASMs\"), including combinations of cannabidiol, stiripentol, fenfluramine, clobazam, valproate, topiramate and others\" - none are disease modifying, however, and in short, newer, better treatment options are desperately sought. The headline results from Stoke's studies were shared in a press release as follows:  Phase 1/2a End of Study Data: 70mg doses demonstrated substantial and sustained reductions in convulsive seizure frequency on top of the best available anti-seizure medicines; Median reductions of 85% (n=10) at 3 months and 74% (n=9) at 6 months after last dose – – Open Label Extension Studies: Durable reductions in seizures and clinically meaningful improvements in multiple measures of cognition and behavior were maintained over 12 months with continued dosing at 30mg and 45mg –  Analysis - Is Stoke Set For Success In Phase 3, Potential Approval? Stoke's share price has certainly been in need of a boost. In January 2021, Stoke stock briefly reached an all-time high of ~$67 per share, which I trace back to early updates from the same Phase 1/2a clinical study reported on last week, named MONARCH. Findings shared in December 2020 revealed that eight out of eleven patients experienced a reduction in convulsive seizure frequency, and that \"single doses of STK-001 up to 30mg and multiple doses of 20mg were well-tolerated with no safety concerns related to the study drug\". Concerns around the safety of higher doses of STK-001, however, which included a partial clinical hold imposed by the FDA on use of a 70mg dose, appear to have driven down Stoke's value. Having fallen to a price of ~$12 in mid-2023, the share price reached an all-time low of >$3.5 in late 2024 after updated data from Monarch revealed 32% of 74 patients experienced an adverse safety event after dosing, with 20% of patients experiencing a serious adverse event, while efficacy data at a 45mg dose underwhelmed, with a 19% reduction in convulsive seizure frequency at 3 months, rising to 45% after 6 months.   Monarch study results (Stoke Therapeutics presentation ) As we can see above (slide taken from Stoke's study results presentation) the 70mg dose is substantially the most efficacious, and Stoke has also revealed that it has: clearance from the U.S. Food and Drug Administration (FDA) that allows patients to receive three doses of 70mg followed by continued dosing at 45mg. Based on this regulatory update and these data, the Company plans to meet with regulatory agencies to discuss a registrational study that includes initial doses of 70mg followed by continued dosing at 45mg. From a safety perspective, 30% (24/81) patients experienced a treatment emergent adverse event (\"TEAE\"), related to STK-001 - the most common being cerebrospinal fluid (\"CSF\") protein elevations and procedural vomiting - and 22% of patients experienced a serious adverse event, albeit the majority of these were \"assessed as unrelated to study drug\", the exception being a single patient who \"experienced Suspected Unexpected Serious Adverse Reactions (SUSARs)\". While it should be noted that these safety event data cover patients across all dose ranges - the percentages are likely to be higher in the higher dose cohorts - it does seem as though, based on Phase 1/2 data, Stoke may have an approvable drug on its hands. Given that the Monarch study had been running for over 3 years, investors may face a lengthy wait for results from a Phase 3 study whose protocols are yet to be agreed, with any pivotal study unlikely to be initiated until late this year, or early next. In terms of the competition and market opportunity, Stoke lists numerous competitors in its 2023 annual report / 10K submission, including Jazz Pharmaceuticals' cannabinoid therapy epidiolex, and \"GABA receptor agonists, such as clobazam and stiripentol, and glutamate blockers, which is one of the mechanisms of action of topiramate\". Epidiolex earned $845m of revenues last year, although it is approved to treat Lennoz Gastaut Syndrome (\"LGS\") and tuberous sclerosis complex (\"TSC\") as well as Dravet Syndrome. Stoke is not a cash rich company, reporting $201m in cash as of the end of 2023, and a net loss for the year of $(105m), or $(2.38) per share. Management did complete a substantial fundraising of $125m fundraising at $13.5 per share immediately after announcing the successful Monarch results last week, but funding a Phase 3, and potentially, an approval push and commercial launch, will be cash intensive and likely require the company to complete further fundraisings down the line. Perhaps most importantly, however, the latest study results appear to give STK-001 a \"best-in-class\" profile in the field of seizure reduction, and provide proof of concept (\"PoC\") for its TANGO platform, and validate the mechanism of action (\"MoA\") of STK-001, described as follows by Stoke:  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. SCN1A codes for the alpha subunit of the voltage-gated sodium channel, or Nav1.1 protein, an ion channel that is essential for the generation and propagation of action potentials. More than 95% of the disease-causing mutations of SCN1A cause loss-of-function, resulting in haploinsufficiency (approximately 50% reduction) of the Nav1.1 protein in select neurons in the brain. This loss of Nav1.1 channels in inhibitory interneurons and other nerve cells results in Dravet syndrome.  Other Opportunities May Offer Nearer Term Catalysts Luckily for shareholders, given there may now be a long wait for the next set of STK-001 data (although it may well be worth waiting for), there are other positives to consider in relation to buying Stoke Therapeutics stock. As per Stoke's 10K submission:  STK-002 is our 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. To date, we have generated preclinical data demonstrating proof-of-mechanism and proof-of-concept for STK-002. ADOA is the most common inherited optic nerve disorder seen in clinical practice. ADOA causes progressive and irreversible vision loss in both eyes starting in the first decade of life.  With a global prevalence of ~250k, ADOA is not a large indication, but there are no approved therapies to treat the disease. The data establishing signs of efficacy early stage: 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 Ophthalmology is a lucrative space to be in, nonetheless, but from a near-term revenue perspective, of more interest may be Stoke's partnership with commercial stage Pharma Acadia Pharmaceuticals (ACAD). The agreement apparently focuses on \"targets SYNGAP1, MECP2 (Rett syndrome), and an undisclosed neurodevelopmental target of mutual interest.\" Acadia, which already markets and sells a Rett Syndrome drug (Rett is a rare genetic disease that results in progressive loss of motor skills and language), Daybue, approved in March last year, and earning $177m last year, has made an upfront payment of $60m to Stoke (in January last year), and pledged up to $907m in development, regulatory and commercial milestones. The deal provides some additional validation of Stoke's technology, and even if it has not proven lucrative to date, with collaboration revenue reported as $8.8m in 2023, and $12.4m in 2022, if the company can identify a product candidate and secure an investigational new drug (\"IND\") from the FDA, allowing in-human studies to begin, a much larger payout ought to be secured. This could happen within 1-2 years, I suspect. Looking Ahead - Is Stoke Therapeutics Stock A Buy, Sell Or Hold After Sharing Positive STK-001 Data Stoke's data has helped derisk its technology platform somewhat, validating its ASO approach - another company that uses a similar approach is Ionis Pharmaceuticals, the $6.3bn market cap Pharma that has secured four commercial drug approvals, and has multiple other late-stage opportunities, in partnership with some of the world's largest Pharmas, including Novartis (NVS), GSK (GSK), Roche (OTCQX:RHHBY) and Biogen (BIIB). Whilst I don't expect progress into a pivotal study to be rapid with STK-001, the drugs' ability to dramatically reduce seizure frequency in Dravet patients, if repeated in a Phase 3 setting, seems bound to secure a commercial approval, barring any safety concerns - likely the biggest threat, given the reasonably high levels of adverse events in studies to date, and highest doses planned for the Phase study. I am speculating, but perhaps it may be possible for Stoke to push for a rolling approval process / accelerated approval in which data from the pivotal study is submitted to the FDA as soon as it is available, or a priority review (\"PRV\"), in which the FDA's review process is shortened from 10 months, to a maximum of 6 months, given the level of unmet need. Last week's fundraising eases any short-to-medium term funding concerns, and allows Stoke to run a Phase 3 for STK-001, whilst hopefully bringing up to four more assets into the clinic in the next couple of years. Perhaps the best chance for investors to realise a return on investment buying Stoke stock today would be for a larger Pharma to acquire the company, hoping to leverage its fairly unique technology and approach to drug development, before any approvals push its price tag up. Given the recent upside, and based on what happened post January 2021 - a substantial, long-term, downward drift, my suspicion would be that Stoke stock will drift downward in value over the next few months (barring M&A), due to a lack of near-term catalysts.  With that said, I do believe there is a good chance the share price will rise again, as the STK-001 pivotal study is initiated and progresses, and therefore, while I would not invest today, I plan to keep a close eye on the company, as I believe STK-001 has a good chance of approval and therefore the share price has just as good a chance of trading higher than it does today, and perhaps substantially so, in 18-24 months' time.","kind":null,"is_publish_news":null,"is_publish_highlight":null,"is_publish_live":null,"is_publish_wemedia":null,"editions":null,"symbols":[],"gpt_button":1},"commentList":[],"isCommentEnd":true,"newsSizeData":{"likeSize":0,"commentSize":0,"repostSize":0,"favoriteSize":0,"likeStatus":false,"favoriteStatus":false},"APP":{"userAgent":"Mozilla/5.0 AppleWebKit/537.36 (KHTML, like Gecko; compatible; ClaudeBot/1.0; +claudebot@anthropic.com)","isDev":false,"isTTM":true,"deviceId":"web-server-community-laohu8-v3","version":"4.22.4","shortVersion":"4.22.4","platform":"web","vendor":"web","appName":"ttm","isIOS":false,"isAndroid":false,"isTiger":false,"isTHS":false,"isWeiXin":false,"isWeiXinMini":false,"isWeiBo":false,"isQQ":false,"isBaiduSwan":false,"isBaiduBox":false,"isDingTalk":false,"isToutiao":false,"isOnePlus":false,"isHuaWei":false,"isXiaomi":false,"isXiaomiWebView":false,"isOppo":false,"isVivo":false,"isSamsung":false,"isMobile":false},"href":"/m/news/2424337050"}