CARLSBAD, Calif., April 21, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (NASDAQ:DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced that it will present an update on the progress of its DT-168 program for Fuchs endothelial corneal dystrophy (FECD) at Eyecelerator @ Park City 2025 on Friday, May 2, 2025, at 1:30 p.m. MT in Park City, UT. The presentation will include safety findings from the Phase 1 single- and multiple-ascendi ...

CARLSBAD, Calif., April 17, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced the appointment of Chris M. Storgard, M.D., as Chief Medical Officer (CMO). Dr. Storgard brings over two decades of leadership and hands-on drug development experience, having successfully advanced multiple assets from preclinical stages through global regulatory approvals. “We are thrilled to we ...

Design Therapeutics, Inc. (DSGN) could be a solid addition to your portfolio given its recent upgrade to a Zacks Rank #2 (Buy). This upgrade primarily reflects an upward trend in earnings estimates, which is one of the most powerful forces impacting stock prices.A company's changing earnings picture is at the core of the Zacks rating. The system tracks the Zacks Consensus Estimate -- the consensus measure of EPS estimates from the sell-side analysts covering the stock -- for the current and following years. ...

Financial Reporting and Compliance Risks - Future changes in financial accounting standards may lead to unexpected revenue fluctuations and affect reported financial results [434]. - Changes in tax laws could materially impact the company's business operations and financial performance, potentially increasing future U.S. tax expenses [435]. - The company's disclosure controls may not prevent all errors or fraud, leading to potential misstatements [465]. - Non-compliance with environmental, health, and safety laws could result in substantial fines and penalties, adversely affecting business success [450]. - The company does not maintain insurance for environmental liability, which may expose it to significant risks [451]. Cybersecurity and Technology Risks - The company is increasingly dependent on information technology systems, which may be vulnerable to security incidents and data breaches [436]. - Cyberattacks and online fraud are on the rise, posing significant risks to the company's operations and data security [437]. - The company faces challenges in monitoring third-party contractors' cybersecurity practices, which could lead to adverse consequences if security incidents occur [441]. Operational and Market Risks - Natural disasters could disrupt operations, as the company lacks comprehensive disaster recovery and business continuity plans [447]. - Unstable market conditions, including high inflation and rising interest rates, may adversely affect the company's financial performance and stock price [462]. - Geopolitical tensions, such as the U.S.-China conflict and the Russia/Ukraine war, could materially impact global trade and the company's operations [463]. Governance and Shareholder Risks - Anti-takeover provisions in Delaware law may hinder potential mergers or acquisitions, potentially depressing the stock price [453]. - The company's bylaws include provisions that could delay stockholder actions, impacting governance and control [454]. - The company has not opted out of Section 203 of the DGCL, which may prohibit large stockholders owning 15% or more from merging or combining with the company for a certain period [455]. - The exclusive forum provisions in the amended and restated certificate of incorporation may limit stockholders' ability to bring claims in favorable judicial forums, potentially increasing litigation costs [460]. - The company faces risks of securities class action litigation, particularly following declines in stock price, which could divert management's attention and resources [461]. - Substantial sales of common stock by directors or significant stockholders could lead to a decline in the stock price [464]. Analyst and Market Perception Risks - Inaccurate or unfavorable research from analysts could negatively affect the company's stock price and trading volume [467]. - The company is classified as a "smaller reporting company" and is not required to provide certain market risk disclosures [545].

Clinical Trials - Design Therapeutics initiated a Phase 1 trial of DT-216P2 for Friedreich Ataxia (FA) with patient dosing expected to begin in mid-2025[2] - The company completed dosing in the Phase 1 trial of DT-168 for Fuchs Endothelial Corneal Dystrophy (FECD) and anticipates data release in the first half of 2025[4] - The company achieved enrollment of approximately 250 patients in the FECD observational study, with 100 selected for future follow-ups[4] - Design Therapeutics plans to select a development candidate for myotonic dystrophy type-1 (DM1) in 2025[4] Financial Performance - Research and development (R&D) expenses for Q4 2024 were $12.2 million, a 10.4% increase from $11.0 million in Q4 2023[8] - General and administrative (G&A) expenses for Q4 2024 were $4.5 million, compared to $4.1 million in Q4 2023, reflecting a 9.8% increase[8] - The net loss for Q4 2024 was $13.7 million, up from a net loss of $11.8 million in Q4 2023, indicating a 16.1% increase in losses[8] - Total operating expenses for the year ended December 31, 2024, were $62.4 million, down from $78.2 million in 2023, a decrease of 20.2%[12] - The total stockholders' equity as of December 31, 2024, was $242.1 million, down from $277.7 million in 2023[14] Cash Position - Cash, cash equivalents, and marketable securities totaled $245.5 million as of December 31, 2024, expected to fund operations into 2029[8]

Core Insights - Design Therapeutics, Inc. has initiated a Phase 1 single ascending dose trial of DT-216P2 for Friedreich Ataxia (FA) with patient dosing expected to begin in mid-2025 [1][6] - The company has completed dosing in the Phase 1 trial for DT-168 and anticipates data release in the first half of 2025 [1][6] - Design Therapeutics is well-capitalized with cash and securities amounting to $245.5 million, which is expected to fund operations through multiple potential clinical proof-of-concept data sets [1][4] Clinical Development Progress - The Phase 1 trial for DT-216P2 is being conducted in healthy volunteers in Australia, focusing on safety and pharmacokinetics [6] - A Phase 1/2 multiple ascending dose trial for FA patients is anticipated to start in mid-2025, with data expected in 2026 [6] - The company has achieved its enrollment target for the observational study in Fuchs Endothelial Corneal Dystrophy (FECD), recruiting approximately 250 patients [6] Financial Performance - Research and development (R&D) expenses for Q4 2024 were $12.2 million, totaling $44.4 million for the year [6][10] - General and administrative (G&A) expenses for Q4 2024 were $4.5 million, with an annual total of $18.0 million [6][10] - The net loss for Q4 2024 was $13.7 million, with a total net loss of $49.6 million for the year [6][10] Future Outlook - The company expects to select a development candidate for myotonic dystrophy type-1 (DM1) in 2025 [1][6] - Design Therapeutics aims to advance its GeneTAC platform, with multiple clinical proof-of-concept data sets anticipated over the next few years [2][6]

Company Overview - Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases [3] - The company utilizes a platform of GeneTAC gene targeted chimera small molecules to address the underlying causes of diseases by modulating the expression of specific disease-causing genes [3] Product Development - Design Therapeutics is advancing several programs, including DT-216P2 for Friedreich ataxia and DT-168 for Fuchs endothelial corneal dystrophy [3] - The company is also working on therapies for myotonic dystrophy type-1 and Huntington's disease, with ongoing discovery efforts for multiple genomic medicines [3] Upcoming Events - Management will participate in a fireside chat at Leerink's Global Healthcare Conference on March 10, 2025, at 1:40 p.m. ET in Miami [1] - A live webcast of the event will be available on the company's website and archived for at least 30 days [2]

Company Overview - Design Therapeutics, Inc. is a clinical-stage biotechnology company focused on developing treatments for serious degenerative genetic diseases [2] - The company utilizes its GeneTAC™ platform to create gene-targeted chimera small molecules that can modulate the expression of disease-causing genes [2] Product Development - The lead product, DT-216, is in development for Friedreich ataxia, with additional programs targeting Fuchs endothelial corneal dystrophy, Huntington's disease, and myotonic dystrophy type-1 [2] - Discovery efforts are ongoing for multiple genomic medicines [2] Upcoming Events - Management will participate in the 2024 Jefferies London Healthcare Conference on November 19, 2024, at 10:00 a.m. GMT in London, UK [1] - The company will also engage in a fireside chat at the Piper Sandler 36th Annual Healthcare Conference on December 3, 2024, at 11:30 a.m. ET in New York, NY [1] - Live webcasts of these presentations will be available and archived for at least 30 days [1]

Financial Position - As of September 30, 2024, the company had an accumulated deficit of $213.6 million and cash, cash equivalents, and investment securities of $254.1 million[79]. - As of September 30, 2024, the company had $254.1 million in cash, cash equivalents, and investment securities, a decrease of $27.7 million from $281.8 million at December 31, 2023[99]. - The company anticipates continuing to incur net losses and negative cash flows from operations for the foreseeable future[98]. - The company expects its existing cash and investments to be sufficient to fund planned operating expenses for more than the next 12 months[103]. - The company may need to finance its cash needs through public or private equity offerings, debt financings, or strategic collaborations[106]. Operating Expenses - Research and development expenses for Q3 2024 were $11.9 million, a decrease of $1.4 million (approximately 10.4%) from $13.3 million in Q3 2023, primarily due to the completion of clinical activities for the FA program[93]. - General and administrative expenses for Q3 2024 were $4.4 million, down $1.2 million (approximately 21.5%) from $5.6 million in Q3 2023[93]. - Total operating expenses for Q3 2024 were $16.2 million, a decrease of $2.6 million (approximately 13.7%) from $18.8 million in Q3 2023[93]. - For the nine months ended September 30, 2024, total operating expenses were $45.689 million, down $17.380 million (27.6%) from $63.069 million in the same period in 2023[95]. - The company reported a significant decrease in research and development expenses for the nine months ended September 30, 2024, totaling $32.193 million, down $13.858 million (30.1%) from $46.051 million in the same period in 2023[96]. Clinical Development - The company plans to initiate a Phase 1 clinical trial of the new product candidate DT-216P2 in healthy volunteers in the first half of 2025, with patient dosing expected later in 2025[72]. - The second GeneTAC™ small molecule, DT-168, is currently in a Phase 1 clinical trial, with initial data expected in the first half of 2025[74]. - In preclinical studies for Huntington's disease, the company observed over a 50% reduction in mutant HTT RNA and protein in an animal model after eight weeks of treatment[75]. - The company is continuing to evaluate DM1 GeneTAC™ molecules, which have shown robust reductions in nuclear foci and splicing defects in patient muscle cells[76]. Future Outlook - The company anticipates that expenses and operating losses will increase substantially as it continues clinical trials and expands its capabilities[80]. - The company has not generated any revenue from product sales and expects to incur significant commercialization expenses if any product candidates receive marketing approval[79]. - Future capital requirements will depend on various factors, including the scope and costs of drug discovery and clinical trials, as well as regulatory approval processes[105]. Company Classification - The company remains classified as an emerging growth company until at least December 31, 2026, allowing it to rely on certain exemptions from public company reporting requirements[122]. - The company has irrevocably elected not to avail itself of the exemption from new or revised accounting standards, thus will adhere to the same standards as other public companies[123]. - The company will cease to be an emerging growth company upon reaching at least $1.235 billion in annual revenue or if certain market value thresholds are met[124]. Share Issuance - The company has not sold any shares under the $100 million ATM Program as of September 30, 2024, which is part of the $300 million shelf registration statement filed in April 2022[102].

Financial Performance - Research and development (R&D) expenses for Q3 2024 were $11.9 million, compared to $13.3 million in Q3 2023, reflecting a decrease of approximately 10.3%[5] - General and administrative (G&A) expenses for Q3 2024 were $4.4 million, down from $5.6 million in Q3 2023, a reduction of about 20.3%[5] - The net loss for Q3 2024 was $13.0 million, compared to a net loss of $15.8 million in Q3 2023, indicating an improvement of approximately 17.5%[5] - The total operating expenses for Q3 2024 were $16.2 million, down from $18.8 million in Q3 2023, a decrease of approximately 13.8%[10] Cash Position - As of September 30, 2024, the company had cash, cash equivalents, and marketable securities totaling $254.1 million, expected to fund operations into 2029[6] Clinical Development - The company is on track to initiate the Phase 1 trial for Friedreich Ataxia (FA) in healthy volunteers in the first half of 2025, with patient dosing anticipated later in 2025[2] - Phase 1 development for Fuchs Endothelial Corneal Dystrophy (FECD) has been initiated, with initial data expected in the first half of 2025[3] - The company is advancing preclinical programs for Huntington's Disease (HD) and Myotonic Dystrophy Type-1 (DM1) in preparation for future IND submissions[4] - The company aims to have multiple clinical proof-of-concept data sets from its GeneTAC™ pipeline over the next few years[2] Share Information - The weighted-average shares of common stock outstanding for Q3 2024 were 56,620,731, compared to 55,988,691 in Q3 2023[10]