Stoke Therapeutics(STOK) - 2025 Q2 - Quarterly Results

Stoke Therapeutics Reports Second Quarter 2025 Financial Results and Provides Business Updates Executive Summary and CEO Commentary The company reported Q2 2025 results, highlighting progress in its zorevunersen and STK-002 clinical programs and a strong cash position - First patient dosed in Phase 3 EMPEROR study of zorevunersen for Dravet syndrome[1] - New 3-year zorevunersen OLE data show durable reductions in seizures and improvements in cognition/behavior, with good tolerability[1] - Phase 1 study of STK-002 initiated for Autosomal Dominant Optic Atrophy (ADOA)[1] - CEO highlighted strong execution, foundational understanding of zorevunersen, and expansion into ADOA with STK-002[2] Cash, Cash Equivalents, and Marketable Securities | As of June 30, 2025 | Amount (Millions) | | :------------------ | :---------------- | | Cash, Cash Equivalents, and Marketable Securities | $355.0 | | Anticipated Funding Period | To mid-2028 | Recent Program Highlights The company reported significant progress across its clinical programs, including key study initiations and positive long-term data - First patient dosed in global Phase 3 EMPEROR study of zorevunersen for Dravet syndrome, with sites initiated in the U.S., UK, Japan, and planned for Europe[6] - New positive findings from long-term OLE studies of zorevunersen show substantial and durable reductions in convulsive seizure frequency through three years, alongside continued improvements in cognition and behavior[6] - Phase 1 OSPREY study of STK-002 in patients with Autosomal Dominant Optic Atrophy (ADOA) is now underway[6] Upcoming Anticipated Milestones The company plans to present additional zorevunersen data in 2025 and identify a clinical candidate for SYNGAP-1 in 2026 - Additional data from the zorevunersen clinical development program to be presented at upcoming medical congresses in 2025[6] - Lead optimization is underway to identify a clinical candidate for the treatment of SYNGAP-1 in 2026[6] Second Quarter 2025 Financial Results The company reported a significant increase in Q2 2025 revenue and a reduced net loss, driven by collaboration agreements Q2 2025 Financial Highlights | Metric | Q2 2025 (Thousands) | Q2 2024 (Thousands) | Change (YoY) | | :-------------------------------- | :------------------ | :------------------ | :----------- | | Revenue (Acadia Collaboration) | $10,600 | $4,800 | +120.8% | | Revenue (Biogen Collaboration) | $3,200 | $0 | N/A | | Total Revenue | $13,817 | $4,831 | +186.0% | | Net Loss | $(23,483) | $(25,695) | -8.6% | | Net Loss Per Share (Basic) | $(0.40) | $(0.46) | -13.0% | | R&D Expenses | $25,855 | $21,136 | +22.3% | | G&A Expenses | $15,262 | $13,037 | +17.1% | Year-to-Date 2025 Financial Results For the first six months of 2025, the company achieved substantial revenue growth and shifted to a net income from a net loss Year-to-Date 2025 Financial Highlights | Metric | YTD 2025 (Thousands) | YTD 2024 (Thousands) | Change (YoY) | | :-------------------------------- | :------------------- | :------------------- | :----------- | | Revenue (Acadia Collaboration) | $16,800 | $9,000 | +86.7% | | Revenue (Biogen Collaboration) | $155,600 | $0 | N/A | | Total Revenue | $172,386 | $9,048 | +1805.9% | | Net Income (Loss) | $89,397 | $(52,069) | N/A (Shift to profit) | | Net Income (Loss) Per Share (Diluted) | $1.50 | $(1.02) | N/A (Shift to profit) | | R&D Expenses | $58,531 | $43,504 | +34.5% | | G&A Expenses | $29,915 | $23,258 | +28.6% | - Increase in operating expenses for both three and six-month periods primarily relates to increases in costs associated with personnel and launch readiness expense[9] Stoke Webcast and Conference Call for Analysts and Investors Management will host a webcast and conference call on August 12, 2025, to discuss the financial results and business updates - Webcast and conference call for analysts and investors scheduled for Tuesday, August 12, 2025, at 4:30 PM Eastern Time[7] - Webcast available on the Investors & News section of Stoke's website; registration required for Q&A participation[7] About Dravet Syndrome Dravet syndrome is a severe genetic epilepsy with significant unmet medical needs, affecting an estimated 38,000 people - Dravet syndrome is a severe developmental and epileptic encephalopathy caused by SCN1A gene mutations, leading to insufficient NaV1.1 protein[8] - Over 90% of patients experience seizures despite current treatments, with complications leading to poor quality of life and higher SUDEP risk[8] Estimated Dravet Syndrome Prevalence | Region | Estimated Patients | | :----- | :----------------- | | U.S. | ~16,000 | | UK, EU-4, Japan | ~22,000 | | Total | ~38,000 | About Zorevunersen Zorevunersen is an investigational antisense oligonucleotide designed to treat Dravet syndrome by increasing NaV1.1 protein production - Zorevunersen is an investigational antisense oligonucleotide designed to increase NaV1.1 protein production from the non-mutated SCN1A gene to treat Dravet syndrome[9][10] - Aims to reduce seizure frequency and improve neurodevelopment, cognition, and behavior, with demonstrated potential for disease modification[10] - Granted Orphan Drug, Rare Pediatric Disease, and Breakthrough Therapy Designations by the FDA[10] - Stoke retains exclusive rights in the U.S., Canada, and Mexico; Biogen has exclusive rest-of-world commercialization rights under a strategic collaboration[10] About Autosomal Dominant Optic Atrophy (ADOA) ADOA is the most common inherited optic nerve disorder, causing progressive vision loss with no currently approved treatments - ADOA is the most common inherited optic nerve disorder, causing progressive and irreversible vision loss, with no approved treatment[11] - Primarily caused by OPA1 gene mutations, leading to haploinsufficiency and affecting approximately one in 30,000 people globally[11][12] - Roughly half of ADOA patients fail driving standards, and up to 46% are legally blind[11] About STK-002 STK-002 is a proprietary antisense oligonucleotide in clinical development for ADOA, designed to upregulate OPA1 protein expression - STK-002 is a proprietary antisense oligonucleotide for ADOA, designed to upregulate OPA1 protein expression from the non-mutant OPA1 gene[12] - Believed to be the first disease-modifying therapy for ADOA, aiming to maintain or improve vision[12] - Preclinical data demonstrate proof-of-mechanism and proof-of-concept; granted Orphan Drug designation by the FDA[12] - A Phase 1 study (OSPREY) of STK-002 in patients with ADOA is now underway[6][12] About Stoke Therapeutics Stoke Therapeutics is a biotechnology company developing RNA-based medicines to restore protein expression for genetic diseases - Stoke Therapeutics is a biotechnology company restoring protein expression using RNA medicine and its proprietary TANGO approach[13] - Develops antisense oligonucleotides (ASOs) to selectively restore naturally-occurring protein levels, focusing on CNS and eye diseases caused by haploinsufficiency[13] - Zorevunersen is the lead medicine, with proof of concept demonstrated in other organs, supporting broad potential[13] Cautionary Note Regarding Forward-Looking Statements This section outlines potential risks and uncertainties that could cause actual results to differ from forward-looking statements - Contains forward-looking statements regarding future financial results, clinical outcomes, regulatory approvals, and collaborations[15] - Highlights risks and uncertainties, such as ability to advance product candidates, success of collaborations, replication of clinical trial results, intellectual property protection, and funding[15] - Company undertakes no obligation to revise or update any forward-looking statements after the press release date[15] Reference This section provides the source for the Dravet syndrome prevalence estimate, citing a 2015 publication by Wu et al - Dravet syndrome prevalence estimate is based on Stoke Therapeutics' preliminary estimates, scaling annual incidence to prevalence using country-specific live birth rates and adjusted for Dravet-specific mortality[16] - Incidence rates published by Wu et al., Pediatrics, 2015, were used for the estimate[16] Consolidated Balance Sheets The balance sheet as of June 30, 2025, shows a significant increase in total assets and stockholders' equity compared to year-end 2024 Consolidated Balance Sheet Highlights (Selected Items) | Metric (in thousands) | June 30, 2025 | December 31, 2024 | Change | | :-------------------- | :------------ | :---------------- | :----- | | Cash and cash equivalents | $101,472 | $127,983 | $(26,511) | | Marketable securities - current | $146,236 | $88,916 | $57,320 | | Marketable securities - long-term | $107,256 | $29,824 | $77,432 | | Total current assets | $269,970 | $232,756 | $37,214 | | Total assets | $384,508 | $271,555 | $112,953 | | Total current liabilities | $38,678 | $40,056 | $(1,378) | | Deferred revenue - net of current portion | $9,632 | $0 | $9,632 | | Total liabilities | $49,565 | $42,534 | $7,031 | | Total stockholders' equity | $334,943 | $229,021 | $105,922 | Consolidated Statements of Operations and Comprehensive Income (Loss) The statements of operations show a shift to profitability for the six-month period driven by a substantial increase in collaboration revenue Consolidated Statements of Operations Highlights (Selected Items) | Metric (in thousands) | Q2 2025 | Q2 2024 | YTD 2025 | YTD 2024 | | :-------------------- | :------ | :------ | :------- | :------- | | Revenue | $13,817 | $4,831 | $172,386 | $9,048 | | R&D Expenses | $25,855 | $21,136 | $58,531 | $43,504 | | G&A Expenses | $15,262 | $13,037 | $29,915 | $23,258 | | Total Operating Expenses | $41,117 | $34,173 | $88,446 | $66,762 | | Income (loss) from operations | $(27,300) | $(29,342) | $83,940 | $(57,714) | | Net income (loss) | $(23,483) | $(25,695) | $89,397 | $(52,069) | | Net income (loss) per share (Diluted) | $(0.40) | $(0.46) | $1.50 | $(1.02) | Stoke Media & Investor Contacts This section provides contact information for the company's media and investor relations representatives - Contact details for Dawn Kalmar (Chief Communications Officer) and Doug Snow (Director, Communications & Investor Relations) are provided for inquiries[22]