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]

Core Viewpoint - Design Therapeutics, Inc. (DSGN) has seen a 23.7% increase in share price over the past four weeks, closing at $5.01, with a potential upside of 39.7% based on Wall Street analysts' mean price target of $7 [1] Price Targets - The average price target consists of three short-term estimates ranging from a low of $4 to a high of $12, with a standard deviation of $4.36, indicating variability among analysts [2] - The lowest estimate suggests a decline of 20.2% from the current price, while the highest estimate indicates a potential upside of 139.5% [2] Analyst Sentiment - Analysts show strong agreement regarding DSGN's ability to report better earnings than previously predicted, which supports the view of potential upside [4] - The Zacks Consensus Estimate for the current year has increased by 7.4% over the past month, with two estimates revised higher and no negative revisions [10] Zacks Rank - DSGN holds a Zacks Rank 2 (Buy), placing it in the top 20% of over 4,000 ranked stocks based on earnings estimate factors, indicating a strong potential upside [11]

Financial Performance - The company reported a net loss of $200.5 million as of June 30, 2024, with cash and cash equivalents totaling $261.0 million[65]. - The company has not generated any revenue from product sales to date[65]. - The company anticipates continued net losses and negative cash flows from operations for the foreseeable future, primarily funded through equity and debt financing[82]. - The company reported a net cash used in operating activities of $(23,699,000) for the six months ended June 30, 2024, an improvement from $(31,228,000) in the same period of 2023[86]. Research and Development - Research and development expenses for Q2 2024 were $10.516 million, a decrease of $6.548 million from $17.064 million in Q2 2023[77]. - Research and development expenses for the three months ended June 30, 2024, totaled $10,516,000, a decrease of 38.4% from $17,064,000 in the same period of 2023[78]. - Research and development expenses for the six months ended June 30, 2024, were $20,317,000, a decrease of 38.1% from $32,794,000 in the same period of 2023[80]. - The company aims to complete GLP studies for the new product candidate DT-216P2 by the end of 2024 and expects to initiate clinical trials in 2025[61]. - The company has received FDA clearance for the IND of DT-168 and plans to initiate Phase 1 development in 2024[62]. - In preclinical studies, the HD GeneTAC candidate molecules showed over 50% reduction in mutant HTT RNA and protein in an animal model[63]. - The company plans to recruit 200 patients for an observational study in FECD to confirm disease characteristics and identify rapid progression risks[62]. Operating Expenses - General and administrative expenses for Q2 2024 were $4.527 million, down by $1.005 million from $5.532 million in Q2 2023[77]. - Total operating expenses for Q2 2024 were $15.043 million, a reduction of $7.553 million compared to $22.596 million in Q2 2023[77]. - For the six months ended June 30, 2024, total operating expenses were $29,443,000, down 33.4% from $44,247,000 in the same period of 2023[79]. - General and administrative expenses for the six months ended June 30, 2024, were $9,126,000, down 20.3% from $11,453,000 in the same period of 2023[81]. Cash and Funding - As of June 30, 2024, the company had $261.0 million in cash, cash equivalents, and investment securities, a decrease of $20.8 million from $281.8 million at December 31, 2023[82]. - The company expects its existing cash and investments to be sufficient to fund planned operating expenses for more than the next 12 months[85]. - Future capital requirements will depend on various factors, including the scope and costs of clinical trials and the emergence of competing therapies[87]. - The company has not sold any shares under the $100.0 million ATM Program as of June 30, 2024, which is part of the $300.0 million shelf registration statement filed in April 2022[84]. 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[101]. - 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[102]. - The company will cease to be an emerging growth company upon reaching at least $1.235 billion in annual revenue or if certain market conditions are met[103].

Financial Performance - Research and development (R&D) expenses for Q2 2024 were $10.5 million, a decrease from $17.1 million in Q2 2023[4] - General and administrative (G&A) expenses for Q2 2024 were $4.5 million, down from $5.5 million in Q2 2023[4] - The net loss for Q2 2024 was $11.8 million, compared to a net loss of $19.9 million in Q2 2023[4] - Total operating expenses for Q2 2024 were $15.0 million, down from $22.6 million in Q2 2023[8] Cash Position - Cash, cash equivalents, and marketable securities totaled $261.0 million as of June 30, 2024, expected to fund operations into 2029[4] Clinical Development - The company is on track to start patient trials for DT-216P2 in Friedreich Ataxia (FA) in 2025[3] - DT-168 for Fuchs Endothelial Corneal Dystrophy (FECD) is advancing toward Phase 1 development later this year, with 200 patients to be enrolled in an observational study[3] - The company aims to complete GLP studies for DT-216P2 by year-end 2024[3] - Design Therapeutics is progressing preclinical programs for Huntington's Disease (HD) and Myotonic Dystrophy Type-1 (DM1) in anticipation of future IND submissions[3] Share Information - The weighted-average shares of common stock outstanding for Q2 2024 were approximately 56.6 million, compared to 55.9 million in Q2 2023[8]

Friedreich Ataxia (FA) and Fuchs Endothelial Corneal Dystrophy (FECD) Programs on Track and Advancing Toward Clinical Trials Active Research Pipeline with Programs Progressing in Myotonic Dystrophy Type-1 (DM1) and Huntington's Disease (HD) Cash and Securities of $261.0 Million Supports Operations Through Potentially Four Clinical Proof-of-Concept Data Sets CARLSBAD, Calif., Aug. 05, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious ...

Friedreich Ataxia (FA) and Fuchs Endothelial Corneal Dystrophy (FECD) Programs on Track and Advancing Toward Clinical Trials Active Research Pipeline with Programs Progressing in Myotonic Dystrophy Type-1 (DM1) and Huntington's Disease (HD) Cash and Securities of $261.0 Million Supports Operations Through Potentially Four Clinical Proof-of-Concept Data Sets CARLSBAD, Calif., Aug. 05, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a biotechnology company developing treatments for serious ...

UNITED STATES SECURITIES AND EXCHANGE COMMISSION WASHINGTON, DC 20549 FORM 10-Q (Mark One) ☒ QUARTERLY REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the quarterly period ended March 31, 2024 OR ☐ TRANSITION REPORT PURSUANT TO SECTION 13 OR 15(d) OF THE SECURITIES EXCHANGE ACT OF 1934 For the transition period from to Commission File Number: 001-40288 Design Therapeutics, Inc. (Exact Name of Registrant as Specified in its Charter) Delaware 82-3929248 (State or other jurisd ...