Equilibration disorder

Lovell is an approved supplier to the U.S. Department of Veterans Affairs (VA) and Department of Defense (DoD) Over 28,000 cases of multiple sclerosis (MS) are reported to the VA annually NEWTOWN, Pa., April 03, 2024 (GLOBE NEWSWIRE) -- Helius Medical Technologies, Inc. (Nasdaq:HSDT) (“Helius” or the “Company”), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait deficits, today announced it has partnered with Lovell Government Services (“Lovell”), an SBA-certified Service Disabled Veteran Owned Small Business (“SDVOSB”), to make the Company’s Portable Neuromodulation Stimulator (“PoNS®”) device available to federal healthcare systems. PoNS is indicated in the U.S. for use as a short-term treatment of gait deficit in adults with mild-to-moderate symptoms from MS when used in conjunction with physical therapy. “Through their Multiple Sclerosis Centers of Excellence, the VA is dedicated to maximizing the quality of life for veterans suffering from MS, and we are thrilled to partner with Lovell to expand the reach of our innovative PoNS device. In a study of real-world results, after 14 weeks of PoNS Therapy, 100% of MS patients experienced a clinically meaningful improvement in gait. More than 28,000 cases of MS are reported to the VA annually, making PoNS a potential game changer for veterans and their families,” said Dane Andreeff, President and Chief Executive Officer of Helius. “As the largest integrated healthcare system in the U.S., the VA provides services to veterans with MS from the time of diagnosis through the rest of their lives. Veterans have given their best to our country and should have access to the most innovative and effective resources available. Lovell is proud to introduce this important product to the VA and other federal healthcare providers,” said Chris Lovell, Major, USMC (Ret.), CEO of Lovell Government Services. “Recently, Helius highlighted the real-life story of Kevin Byrne, a retired U.S. veteran who’s been suffering from MS since 1999. Gait difficulties had taken away his most valued treasure, quality adventures with his 13-year-old daughter, but PoNS Therapy helped him improve his walking by increasing speed, endurance, and distance. After treatment with PoNS, he was able to take his daughter to New York City, where they enjoyed walking the streets and seeing Broadway shows, experiences he thought were lost forever. While clinical results have demonstrated the effectiveness of PoNS Therapy, it’s firsthand accounts like Captain Byrne’s that are the most gratifying,” concluded Andreeff. About Lovell® Government Services Lovell Government Services has been a trusted SDVOSB vendor since 2013 with a proven track record of successfully introducing suppliers to the government market. Lovell is a two-time Inc. 5000 honoree and leader in the federal space. They partner with medical and pharmaceutical companies looking to better serve veteran and military patient populations, increase their federal revenue stream, and win government contracts. Learn more at . About Helius Medical Technologies, Inc. Helius Medical Technologies is a leading neurotech company in the medical device field focused on neurologic deficits using orally applied technology platform that amplifies the brain’s ability to engage physiologic compensatory mechanisms and promote neuroplasticity, improving the lives of people dealing with neurologic diseases. The Company’s first commercial product is the Portable Neuromodulation Stimulator. For more information about the PoNS® or Helius Medical Technologies, visit . About the PoNS Device and PoNS Therapy The Portable Neuromodulation Stimulator (“PoNS”) is an innovative, non-implantable, orally applied therapy that delivers neurostimulation through a mouthpiece connected to a controller and it’s used, primarily at home, with physical rehabilitation exercise, to improve balance and gait. The PoNS device, which delivers mild electrical impulses to the tongue, is indicated for use in the United States as a short-term treatment of gait deficit due to mild-to-moderate symptoms from multiple sclerosis (“MS”) and is to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and over by prescription only. PoNS has shown effectiveness in treating gait or balance and a significant reduction in the risk of falling in stroke patients in Canada, where it received authorization for sale in three indications: (i) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from stroke and is to be used in conjunction with physical therapy; (ii) for use as a short-term treatment (14 weeks) of chronic balance deficit due to mild-to-moderate traumatic brain injury (“mmTBI”) and is to be used in conjunction with physical therapy; and (iii) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from MS and is to be used in conjunction with physical therapy. PoNS is also authorized for sale in Australia for short term use by healthcare professionals as an adjunct to a therapeutic exercise program to improve balance and gait. For more information visit . Cautionary Disclaimer Statement Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as “believe,” “expect,” “continue,” “will,” “goal,” “aim” and similar expressions. Such forward-looking statements include, among others, statements regarding the Company’s partnership with Lovell and the uses and effectiveness of PoNS and PoNS Therapy. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include uncertainties associated with the Company’s capital requirements to achieve its business objectives, availability of funds, the Company’s ability to find additional sources of funding, manufacturing, labor shortage and supply chain risks, including risks related to manufacturing delays, the Company’s ability to obtain national Medicare insurance coverage and to obtain a reimbursement code, the Company’s ability to continue to build internal commercial infrastructure, secure state distribution licenses, market awareness of the PoNS device, future clinical trials and the clinical development process, the product development process and the FDA regulatory submission review and approval process, other development activities, ongoing government regulation, and other risks detailed from time to time in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at or . The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law. Investor Relations Contact Lisa M. Wilson, In-Site Communications, Inc. T: 212-452-2793 E: lwilson@insitecony.com

-- Company to host call at 4:30pm today -- NEWTOWN, Pa., March 28, 2024 (GLOBE NEWSWIRE) -- Helius Medical Technologies, Inc. (Nasdaq:HSDT) (“Helius” or the “Company”), a neurotech company focused on delivering a novel therapeutic neuromodulation approach for balance and gait deficits, today announced results for the quarter and full year ended December 31, 2023. Fourth Quarter and Recent Business Updates Secured Healthcare Common Procedure Coding System (“HCPCS”) Level II codes for the Portable Neuromodulation Stimulator (“PoNS®”) mouthpiece and controller from the Centers for Medicare & Medicaid Services (“CMS”), representing a significant step toward reimbursement.Further expanded the registrational program in stroke by adding a second site and initiating an open-label study; reached alignment with the U.S. Food and Drug Administration (“FDA”) on optimizing the stroke development plan.Signed a letter of intent (“LOI”) with the Québec Ministry of Health and Social Services (“MSSS”) to purchase up to 30 PoNS systems that will be distributed in five separate administrative regions within Québec as part of a government-funded initiative designed to evaluate the effectiveness of PoNS Therapy™ when used by patients suffering the effects of stroke.Released study results from collaborative project between Pacific Blue Cross (“PBC”) and HealthTech Connex (“HTC”), demonstrating that PoNS Therapy can drastically improve return-to-work (“RTW”) outcomes for patients suffering from traumatic brain injury (“TBI”).Q4 2023 revenue of $134 thousand, compared to $282 thousand in Q4 2022, primarily attributable to the June 30, 2023 expiration of PTAP in the United States and lower Canada product sales.Total operating expenses of $2.3 million in Q4 2023, a decrease of $0.5 million compared to Q4 2022.Generated $1.3 million of net proceeds from the sale of shares of Company common stock under its At-The-Market (“ATM”) in Q1 2024, extending cash runway into Q3 2024. “We are pleased to have reached a key milestone toward Medicare and broad third-party reimbursement last month when CMS assigned HCPCS codes for the PoNS mouthpiece and controller, effective April 1, 2024. We plan to engage with CMS in the coming months with the objective of securing Medicare reimbursement for the PoNS controller and mouthpiece to be effective as soon as October 1, 2024. In addition, these codes will facilitate our ability to concurrently pursue third-party payer coverage,” said Dane Andreeff, President and Chief Executive Officer of Helius. “Delivering PoNS Therapy to stroke patients suffering from gait and balance deficit is one of our chief goals and I'm proud to report that we’ve made several meaningful strides toward potential U.S. authorization. In recent weeks, we’ve added a second site to our ongoing investigator-initiated, placebo-controlled study, begun enrollment of an open-label study at this same site, and met with the FDA to optimize our overall stroke development plan. We are now targeting an early 2025 regulatory submission with possible commercialization by the end of next year.” “With $1.3 million raised under our ATM program since year end, we now have the cash runway to take us into the third quarter of 2024 as we continue progressing our stroke registrational program and pursuing widespread reimbursement,” concluded Andreeff. Fourth Quarter 2023 Financial Results Total revenue for the fourth quarter of 2023 was $134 thousand, a decrease of $148 thousand compared to $282 thousand in the fourth quarter of 2022 primarily attributable to the June 30, 2023 expiration of PTAP in the United States and lower Canada product sales. Cost of revenues was $90 thousand for the three months ended December 31, 2023, compared to $150 thousand for the comparable period in 2022, primarily due to decreased revenues. Gross profit for the fourth quarter of 2023 was $44 thousand, compared to gross profit of $132 thousand in the fourth quarter of 2022. Selling, general, and administrative expenses for the fourth quarter of 2023 decreased to $1.6 million, compared to $2.0 million in the fourth quarter of 2022 due primarily to a decrease in compensation related expenses. Research and development expenses for the fourth quarter of 2023 decreased to $0.7 million compared to $0.8 million in the fourth quarter of 2022, resulting primarily from a decrease in clinical and product development expenses in the current year period. Operating loss for the fourth quarter of 2023 decreased to a loss of $2.2 million, compared to an operating loss of $2.7 million in the fourth quarter of 2022. Net loss was $1.0 million for the fourth quarter of 2023, compared to a net loss of $4.9 million in the fourth quarter of 2022. The basic and diluted net loss per share for the fourth quarter 2023 was $1.47, compared to net loss per share of $8.66 in the fourth quarter 2022. Full Year 2023 Financial Results Total revenue for the full year 2023 was $644 thousand, compared to $787 thousand for the full year 2022, due to lower Canada product sales resulting from the prior year inclusion of approximately $120 thousand of revenue recognized in connection with the delivery PoNS devices related to noncash consideration paid in the Company’s acquisition of Heuro. Cost of revenue for the full year 2023 increased $120 thousand to $583 thousand, compared to cost of revenue of $463 thousand for the full year 2022, primarily attributable to fixed overhead costs, including salaries and benefits of employees involved in management of the supply chain and certain production costs. Gross profit for the full year 2023 was $61 thousand, compared to gross profit of $324 thousand for the full year 2022. Selling, general, and administrative expenses for the full year 2023 decreased to $9.3 million, compared to $10.6 million for the full year 2022, due primarily to a $0.6 million decrease in stock-based compensation and a $0.6 million decrease in payroll taxes including the $0.5 million Employee Retention Credit claims we filed for the prior eligible periods. Research and development expenses for the full year 2023 decreased to $2.9 million, compared to $4.3 million for the full year 2022, due primarily to decreases in product development expenses and clinical trial activities as we transitioned our focus from product development and clinical trials to U.S. commercialization activities beginning in 2022. Operating loss for the full year 2023 decreased $3.1 million to a loss of $12.4 million, compared to a loss of $15.5 million for the full year 2022. Net loss for the full year 2023 was $8.9 million, compared to $14.1 million for the full year 2022. The basic and diluted net loss per share for the full year 2023 was $14.56 per share, compared to $52.13 per share, for the full year 2022. Cash and Liquidity Cash used in operating activities for the three months ended December 31, 2023, was $2.0 million, compared to $2.1 used in operating activities in the fourth quarter of 2022, reflecting the results of our continued focus on managing cash burn. As of December 31, 2023, the Company had cash of $5.2 million, compared to $14.5 million at December 31, 2022. In Q1 2024, the Company generated $1.3 million of net proceeds from the sale of shares of its common stock under its ATM program at an average share price of $9.27 per share, extending the cash runway into Q3 2024. The Company had no debt outstanding at December 31, 2023. Conference Call Date:Thursday, March 28, 2024Time:4:30 p.m. Eastern TimeRegister (Audio only):Click hereWebcast:Click here The webcast will be archived under the Newsroom section of the Company’s investor relations website. About Helius Medical Technologies, Inc. Helius Medical Technologies is a leading neurotech company in the medical device field focused on neurologic deficits using orally applied technology platform that amplifies the brain’s ability to engage physiologic compensatory mechanisms and promote neuroplasticity, improving the lives of people dealing with neurologic diseases. The Company’s first commercial product is the Portable Neuromodulation Stimulator. For more information about the PoNS® or Helius Medical Technologies, visit www.heliusmedical.com. About the PoNS Device and PoNS Therapy The Portable Neuromodulation Stimulator (PoNS) is an innovative, non-implantable, orally applied therapy that delivers neurostimulation through a mouthpiece connected to a controller and it’s used, primarily at home, with physical rehabilitation exercise, to improve balance and gait. The PoNS device, which delivers mild electrical impulses to the tongue, is indicated for use in the United States as a short-term treatment of gait deficit due to mild-to-moderate symptoms from multiple sclerosis (“MS”) and is to be used as an adjunct to a supervised therapeutic exercise program in patients 22 years of age and over by prescription only. PoNS has shown effectiveness in treating gait or balance and a significant reduction in the risk of falling in stroke patients in Canada, where it received authorization for sale in three indications: (i) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from stroke and is to be used in conjunction with physical therapy; (ii) for use as a short-term treatment (14 weeks) of chronic balance deficit due to mild-to-moderate traumatic brain injury (“mmTBI”) and is to be used in conjunction with physical therapy; and (iii) for use as a short-term treatment (14 weeks) of gait deficit due to mild and moderate symptoms from MS and is to be used in conjunction with physical therapy. PoNS is also authorized for sale in Australia for short term use by healthcare professionals as an adjunct to a therapeutic exercise program to improve balance and gait. For more information visit www.ponstherapy.com. Cautionary Disclaimer Statement Certain statements in this news release are not based on historical facts and constitute forward-looking statements or forward-looking information within the meaning of the U.S. Private Securities Litigation Reform Act of 1995 and Canadian securities laws. All statements other than statements of historical fact included in this news release are forward-looking statements that involve risks and uncertainties. Forward-looking statements are often identified by terms such as “believe,” “expect,” “continue,” “will,” “goal,” “aim” and similar expressions. Such forward-looking statements include, among others, statements regarding the Company’s expected results for its business and financial performance in 2024, the sufficiency of the Company’s future cash position, the development, commercialization and success of the Company’s PoNS and PoNS Treatment, future decisions and approvals from applicable regulatory entities in the U.S. and Canada, the Company’s strategic operating plans, and the uses and effectiveness of PoNS and PoNS Therapy. There can be no assurance that such statements will prove to be accurate and actual results and future events could differ materially from those expressed or implied by such statements. Important factors that could cause actual results to differ materially from the Company’s expectations include uncertainties associated with the Company’s capital requirements to achieve its business objectives, availability of funds, the Company’s ability to find additional sources of funding, manufacturing, labor shortage and supply chain risks, including risks related to manufacturing delays, the Company’s ability to obtain national Medicare insurance coverage and to obtain a reimbursement code, the Company’s ability to continue to build internal commercial infrastructure, secure state distribution licenses, market awareness of the PoNS device, future clinical trials and the clinical development process, the product development process and the FDA regulatory submission review and approval process, other development activities, ongoing government regulation, and other risks detailed from time to time in the “Risk Factors” section of the Company’s Annual Report on Form 10-K for the year ended December 31, 2023, and its other filings with the United States Securities and Exchange Commission and the Canadian securities regulators, which can be obtained from either at www.sec.gov or www.sedar.com. The reader is cautioned not to place undue reliance on any forward-looking statement. The forward-looking statements contained in this news release are made as of the date of this news release and the Company assumes no obligation to update any forward-looking statement or to update the reasons why actual results could differ from such statements except to the extent required by law. Investor Relations Contact Lisa M. Wilson, In-Site Communications, Inc.T: 212-452-2793E: lwilson@insitecony.com Helius Medical Technologies, Inc.Unaudited Condensed Consolidated Statements of Operations(in thousands, except share and per share data) Three Months Ended Years Ended December 31, December 31, 2023 2022 2023 2022 Revenue Product sales, net $123 $281 $605 $778 Other revenue 11 1 39 9 Total revenue 134 282 644 787 Cost of revenue 90 150 583 463 Gross profit 44 132 61 324 Operating expenses Selling, general and administrative expenses 1,632 1,967 9,271 10,640 Research and development expenses 650 794 2,942 4,262 Amortization expense 8 40 117 181 Goodwill and fixed asset impairment — — 159 757 Total operating expenses 2,290 2,801 12,489 15,840 Loss from operations (2,246) (2,669) (12,428) (15,516)Nonoperating income (expense) Interest income (expense), net — 85 257 (834)Change in fair value of derivative liability 915 (2,462) 2,966 3,027 Foreign exchange (loss) gain 213 154 275 (756)Other income (expense), net 73 6 80 7 Nonoperating income (expense), net 1,201 (2,217) 3,578 1,444 Loss before provision for income taxes (1,045) (4,886) (8,850) (14,072)Provision for income taxes — — — — Net loss $(1,045) $(4,886) $(8,850) $(14,072)Loss per share Basic $(1.47) $(8.66) $(14.56) $(52.13)Diluted $(1.47) $(8.66) $(14.56) $(52.13)Weighted average number of common shares outstanding Basic 708,603 564,014 607,890 269,929 Diluted 708,603 564,014 607,890 269,929 Helius Medical Technologies, Inc.Unaudited Condensed Consolidated Balance Sheets(in thousands, except share and per share data) December 31, 2023 December 31, 2022ASSETS Current assets Cash and cash equivalents $5,182 $14,549 Accounts receivable, net 117 71 Other receivables 520 272 Inventory, net 457 589 Prepaid expenses and other current assets 1,162 1,216 Total current assets 7,438 16,697 Property and equipment, net 178 347 Intangible assets, net 24 140 Operating lease right-of-use asset, net 52 103 Total assets $7,692 $17,287 LIABILITIES AND STOCKHOLDERS’ EQUITY Current liabilities Accounts payable $531 $627 Accrued and other current liabilities 1,260 1,280 Current portion of operating lease liabilities 45 54 Current portion of deferred revenue 43 27 Total current liabilities 1,879 1,988 Operating lease liabilities, net of current portion 12 56 Deferred revenue, net of current portion 128 175 Derivative liability 3,323 6,917 Total liabilities 5,342 9,136 STOCKHOLDERS’ EQUITY Class A common stock, $0.001 par value; 150,000,000 shares authorized; 714,590 and 564,094 shares issued and outstanding as of December 31, 2023 and December 31, 2022, respectively 1 1 Additional paid-in capital 162,979 159,645 Accumulated deficit (159,957) (151,107)Accumulated other comprehensive loss (673) (388)Total stockholders' equity 2,350 8,151 Total liabilities and stockholders' equity $7,692 $17,287

– 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 – – STK-001 generally well-tolerated – – Company to meet with regulatory agencies to discuss registrational study design with initial doses of 70mg followed by continued dosing at 45mg – – Webcast and conference call for analysts and investors at 4:30 p.m. Eastern Time today – BEDFORD, Mass.--(BUSINESS WIRE)-- Stoke Therapeutics, Inc. (Nasdaq: STOK), a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines, today announced landmark new data from two open-label Phase 1/2a studies and two open-label extension (OLE) studies of children and adolescents ages 2 to 18 with Dravet syndrome who were treated with STK-001. Data from these studies showed clinically meaningful effects, including substantial and durable reductions in convulsive seizure frequency and improvements in multiple measures of cognition and behavior that support the potential for disease modification. These improvements were observed among a highly refractory group of patients who were already taking the best available anti-seizure medicines. STK-001 has been generally well-tolerated in studies to date. Today, the Company also announced 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. “The totality of these data provide compelling evidence that support the potential for STK-001 to be a disease-modifying medicine for patients with Dravet syndrome by treating the underlying cause of the disease, rather than just the symptoms,” said Edward M. Kaye, M.D., Chief Executive Officer of Stoke Therapeutics. “STK-001 is the first medicine in development to demonstrate substantial and durable reductions in seizure frequency and improvements in multiple measures of cognition and behavior. These effects were observed in patients who were already taking the best available anti-seizure medicines, which confirms our highly differentiated mechanism of action and approach to treating this disease. We look forward to meeting with regulatory agencies to discuss our plans for a randomized, controlled registrational study and to providing an update coming out of those discussions later in 2024.” “For decades, the primary goal of treating Dravet syndrome has been to control the frequency and severity of seizures, but, as we can now see from natural history data, many patients still experience high rates of seizure frequency and fall further and further behind in their neurodevelopment,” said Joseph Sullivan, M.D., FAES, Professor of Neurology and Pediatrics and Director of the Pediatric Epilepsy Center of Excellence at the University of California San Francisco, and a prominent researcher in Dravet syndrome. “A 50% reduction in seizures is an important measure of clinical efficacy, so an 80% reduction on top of any benefit patients may already be getting from their baseline anti-seizure regimen is profound. The further evidence of improvements in skills like communication, behavior, socialization and movement distinguish this approach from anything we have seen to date and mark our entry into a new era in the treatment of Dravet syndrome.” Phase 1/2a Study Results: Substantial and Sustained Reductions in Convulsive Seizure Frequency The Phase 1/2a studies were multi-center and included children and adolescents who have an established diagnosis of Dravet syndrome. Patients enrolled in these studies were highly refractory to treatment and taking the best available anti-seizure medicines: 85% of patients were taking at least three and 54% were taking at least four medicines to control seizures. Half the patients in the studies were taking concomitant fenfluramine. New data from a combined analysis of 19 clinically evaluable patients who were treated with one, two or three doses of 70mg demonstrated substantial reductions in convulsive seizure frequency compared to baseline at 3 months and at 6 months after the last dose, one of several secondary endpoints in each study. Observed Reductions in Convulsive Seizure Frequency Among Patients Treated with 70mg Doses of STK-001 in the Phase 1/2a Studies Median % Reduction from Baseline in Convulsive Seizure Frequency 70mg (1 dose, n=8) 70mg (2 or 3 doses, n=11) At 3 Months After Last Dose 43% (n=8) 85% (n=10†) At 6 Months After Last Dose 57% (n=7*) 74% (n=9†) *Seizure data excluded from month 5-6 for 1 patient because >50% seizure diary was missing †Seizure data excluded for 2 patients (1 patient prior to 3m after last dose, 1 prior to 6m after last dose) following a change in background anti-seizure medicines Open Label Extension Studies: Durable reductions in seizures and clinically meaningful improvements in multiple measures of cognition and behavior over 12 months with continued dosing at 30mg and 45mg Eligible patients who completed treatment in the Phase 1/2a studies continued treatment with STK-001 in one of two OLEs. At the time of the analysis, 92% (68/74) of eligible patients had enrolled in the OLEs and 84% (57/68) remained in the studies. Durable reductions in convulsive seizure frequency were observed throughout the course of treatment. This analysis only included patients who received >30mg of STK-001 in the Phase 1/2a studies and then continued treatment with STK-001 (30mg or 45mg) every four months in the OLEs. Clinically meaningful improvements from baseline through 12 months were observed in multiple measures of cognition and behavior, including multiple sub-domains of the Vineland Adaptive Behavior Scale (VINELAND-3). These improvements are in stark contrast to recent natural history study data that showed that, on average, patients with Dravet syndrome experienced no meaningful improvement in convulsive seizure frequency and exhibited widening gaps in cognition and behavior compared to neurotypical peers, despite treatment with the best available anti-seizure medicines. Key Safety Findings At the time of the analyses, 81 patients had been treated with STK-001. Safety findings are summarized below. STK-001 was generally well-tolerated across the Phase 1/2a and OLE studies. In the Phase 1/2a studies: 30% (24/81) of patients experienced a treatment-emergent adverse event (TEAE) that was related to study drug. The most common were CSF protein elevations and procedural vomiting; and 22% (18/81) of patients had a treatment-emergent serious adverse event. These events were assessed as unrelated to study drug except for the previously reported case of one patient who experienced Suspected Unexpected Serious Adverse Reactions (SUSARs). A greater incidence of CSF protein elevation was observed in the OLEs. 74% (50/68) of patients in the OLEs had at least 1 CSF protein value >50 mg/dL. No clinical manifestations have been observed in these patients. Across the studies, one patient discontinued treatment due to study drug. As previously reported, this patient discontinued treatment in the OLE due to elevated CSF protein. Stoke Webcast and Conference Call for Analysts and Investors Stoke will host a webcast and conference call for analysts and investors at 4:30 p.m. Eastern Time on March 25, 2024, to present landmark new data from two Phase 1/2a studies and two ongoing open-label extension (OLE) studies in children and adolescents ages 2 to 18 with Dravet syndrome. The webcast will be broadcast live on the Investors & News section of Stoke’s website at . Participants who want to join the call and ask a question may register here to receive the dial-in numbers and unique PIN to seamlessly access the call. Otherwise please access the listen-only webcast by clicking here. An archived replay of the webcast will be available for at least 90 days following the event. 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 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. STK-001 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 the U.S. Studies: MONARCH (Phase 1/2a) and SWALLOWTAIL (OLE) The MONARCH study was 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 were to assess the safety and tolerability of STK-001, as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. A secondary objective was to assess the efficacy as an adjunctive antiepileptic treatment with respect to the percentage change from baseline in convulsive seizure frequency. Following completion of MONARCH, patients who met study entry criteria were 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. The study is also evaluating the long-term effects of STK-001 on convulsive seizure frequency and on behavior, cognition and overall quality of life. Dosing in SWALLOWTAIL is ongoing. About the UK Studies: ADMIRAL (Phase 1/2a) and LONGWING (OLE) The ADMIRAL study was 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 were to assess the safety and tolerability of multiple doses of STK-001, as well as to determine the pharmacokinetics in plasma and exposure in cerebrospinal fluid. A secondary objective was 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. Overall clinical status and quality of life were secondary endpoints of ADMIRAL. Following completion of ADMIRAL, patients who met study entry criteria were eligible to continue treatment in LONGWING, an open-label extension (OLE) study designed to evaluate the long-term safety and tolerability of repeat doses of STK-001. The study is also evaluating the long-term effects of STK-001 on convulsive seizure frequency and on behavior, cognition and overall quality of life. Dosing in LONGWING is ongoing. About the BUTTERFLY Observational Study The BUTTERFLY study was a multicenter, longitudinal, prospective, observational study of children and adolescents ages 2 to 18 who have been diagnosed with Dravet syndrome as a result of an SCN1A gene mutation. This study was designed to evaluate neurodevelopmental status and change from baseline to 24 months. Secondary and exploratory endpoints in the study evaluated changes in other disease measures, including seizures and additional non-seizure comorbidities. No investigational medications or other treatments were provided. Participants continued to receive their usual care, including anti-seizure medications, and were observed for up to two years. The study was conducted at approximately 20 sites in the United States. Two-year results were presented at the American Epilepsy Society in December 2023 and showed that, on average, patients experienced no meaningful improvement in convulsive seizure frequency and exhibited widening gaps in cognition and behavior compared to neurotypical peers, despite treatment with the best available anti-seizure medicines. About Stoke Therapeutics Stoke Therapeutics (Nasdaq: STOK), is a biotechnology company dedicated to addressing the underlying cause of severe diseases by upregulating protein expression with RNA-based medicines. 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, 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 . 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 ability of 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 STK-001; 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 into 2025; 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.