Design Therapeutics, Inc.
For the period ended Sep 30, 2025. Filed Nov 5, 2025.
View on SEC.govAbout this filing
A company's report for each of its first three quarters. The financial statements are not audited. It shows how the business has moved since the last annual report, and what management says changed.
Sep 30, 2025 · Filed Nov 5, 2025
DSGN$10.21+5.6%Stored close · as of Nov 6, 2026
What changed in Management’s Discussion and Analysis?
Read the current filingPrevious report: 10-Q
Period ended 2025-06-30; filed 2025-08-07.
0000950170-25-105062
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsCurrent report: 10-Q
Period ended 2025-09-30; filed 2025-11-05.
0001193125-25-266970
Item 2. Management’s Discussion and Analysis of Financial Condition and Results of OperationsThe baseline is this issuer’s latest earlier reporting period of the same form that was filed before the current report. A previous 10-Q is a different quarter, not a year-over-year comparison. Amendments are excluded.
Compared 121 previous paragraphs and 121 current paragraphs.
102 unchanged; 17 reworded; 2 added; 2 removed; 0 previous and 0 current paragraphs rearranged.
Tables are not compared: 5 previous and 5 current tables excluded. Dates, whitespace, punctuation and numbering changes are ignored; changes to financial figures remain significant.
A changed paragraph is not evidence that risk increased or decreased. An unchanged paragraph does not establish that risk stayed the same.
Added paragraphs (2)
Current report
In November 2025, we nominated our third GeneTAC® small molecule, DT-818, as a development candidate for the treatment of DM1. The underlying cause of DM1 is a CTG repeat expansion in the DMPK gene, which DT-818 is designed to address by selectively reducing transcription of the mutant expanded allele. In preclinical studies, DT-818 has demonstrated a potential best-in-disease profile for DM1, including a greater than 90% reduction in toxic RNA foci in DM1 patient cells, corresponding splicing correction and selective targeting of mutant DMPK. In preclinical models of DM1 (HSALR mouse model with CTG repeats under the control of the actin promoter), DT-818 treatment resulted in improved myotonia and foci reduction. In tissue distribution studies in NHPs, DT-818 levels were observed to be at expected pharmacologic levels in key target tissues at well-tolerated doses. We have obtained regulatory clearance to initiate clinical development of DT-818 and plan to begin dosing DM1 patients in a Phase 1 MAD trial in Australia to assess safety and correction of mis-splicing in the first half of 2026, with splicing data expected in 2027.
Current report
Comparison of the Nine Months Ended September 30, 2025 and 2024
Removed paragraphs (2)
Previous report
Our third program based on the GeneTAC® platform is focused on DM1. Our DM1 GeneTAC® molecules are specifically designed to reduce the formation of CUG repeat hairpin structures that trap splicing factors and form pathogenic nuclear foci that cause DM1. Blocking the formation of CUG foci has demonstrated phenotypic benefit in DM1 patients. We plan to continue evaluating the properties of our DM1 GeneTAC® molecules in preclinical studies in order to nominate a development candidate in 2025.
Previous report
Comparison of the Six Months Ended June 30, 2025 and 2024
Reworded paragraphs (17)
Previous wording
We are conducting our RESTORE-FA (Reactivating Expression Suppressed Through Overcoming Repeat Expansion for FA) Phase 1/2 MAD clinical trial of DT-216P2. The RESTORE-FA trial is designed to evaluate the safety, tolerability, PK and pharmacodynamics (PD) of IV and SC of DT-216P2 in patients with FA. We anticipate providing an update from the RESTORE-FA trial on the effect of DT-216P2 on endogenous FXN levels following 12 weeks of dosing in 2026.
Current wording
We are conducting our RESTORE-FA (Reactivating Expression Suppressed Through Overcoming Repeat Expansion for FA) Phase 1/2 MAD clinical trial of DT-216P2. The RESTORE-FA trial is designed to evaluate the safety, tolerability, PK and pharmacodynamics (PD) of IV and SC of DT-216P2 in patients with FA. We anticipate providing an update from the RESTORE-FA trial on the effect of DT-216P2 on endogenous FXN levels following 12 weeks of dosing in the second half of 2026.
Previous wording
In May 2025, we reported results from a completed Phase 1, double-masked, placebo-controlled, randomized, SAD/MAD clinical trial evaluating the safety, tolerability and systemic PK of DT-168 ophthalmic solution in normal healthy volunteers. Twenty-four normal healthy volunteers received either placebo or single- and multiple-ascending doses of DT-168 eye drops twice daily for seven days (up to a maximum dose of two 0.5% drops twice-daily). DT-168 eye drops were well-tolerated in all participants. There were no serious adverse events, no ocular adverse events (AEs) and no treatment discontinuations due to AEs in the trial. All observed AEs were deemed not related to DT-168 by the trial investigator. PK analysis demonstrated systemic exposure below the limit of quantitation for all participants across all timepoints and all dose groups. In parallel with the Phase 1 trial, we conducted reference range studies which showed consistently different splicing in the corneal endothelium between unaffected eye donors and surgical samples from mutant TCF4 FECD patients, supporting the potential for corneal endothelium biomarkers as a clinical proof-of-concept measure of drug activity. We have initiated a Phase 2 biomarker trial of DT-168 to evaluate safety, tolerability, and corneal endothelium biomarkers in patients with FECD. FECD Patients will receive 0.5% DT-168 eye drops twice-daily for approximately four weeks or more before corneal transplant surgery. Following surgery, tissues from the treated eyes of FECD patients will undergo testing to assess corneal endothelium biomarkers including the abnormal splicing of genes known as spliceopathy. We anticipate reporting data from the Phase 2 biomarker trial in 2026.
Current wording
In May 2025, we reported results from a completed Phase 1, double-masked, placebo-controlled, randomized, SAD/MAD clinical trial evaluating the safety, tolerability and systemic PK of DT-168 ophthalmic solution in normal healthy volunteers. Twenty-four normal healthy volunteers received either placebo or single- and multiple-ascending doses of DT-168 eye drops twice daily for seven days (up to a maximum dose of two 0.5% drops twice-daily). DT-168 eye drops were well-tolerated in all participants. There were no serious adverse events, no ocular adverse events (AEs) and no treatment discontinuations due to AEs in the trial. All observed AEs were deemed not related to DT-168 by the trial investigator. PK analysis demonstrated systemic exposure below the limit of quantitation for all participants across all timepoints and all dose groups. In parallel with the Phase 1 trial, we conducted reference range studies which showed consistently different splicing in the corneal endothelium between unaffected eye donors and surgical samples from mutant TCF4 FECD patients, supporting the potential for corneal endothelium biomarkers as a clinical proof-of-concept measure of drug activity. We are conducting a Phase 2 biomarker trial of DT-168 to evaluate safety, tolerability, and corneal endothelium biomarkers in patients with FECD. FECD Patients will receive 0.5% DT-168 eye drops twice-daily for approximately four weeks or more before corneal transplant surgery. Following surgery, tissues from the treated eyes of FECD patients will undergo testing to assess corneal endothelium biomarkers including the abnormal splicing of genes known as spliceopathy. We anticipate reporting data from the Phase 2 biomarker trial in the second half of 2026.
Previous wording
To date, we have incurred net losses and negative cash flows from operations since our inception and as of June 30, 2025 had an accumulated deficit of $264.0 million. Our cash, cash equivalents and investment securities balance as of June 30, 2025 was $216.3 million. Our net losses have resulted primarily from costs incurred in connection with organizing and staffing our company, business planning, raising capital, developing and optimizing our technology platform, identifying potential product candidates, undertaking research and nonclinical studies, clinical development activities, engaging in manufacturing for our development programs, and providing general and administrative support for these operations. We do not have any products approved for sale and have not generated any revenue from product sales.
Current wording
To date, we have incurred net losses and negative cash flows from operations since our inception and as of September 30, 2025 had an accumulated deficit of $281.0 million. Our cash, cash equivalents and investment securities balance as of September 30, 2025 was $206.0 million. Our net losses have resulted primarily from costs incurred in connection with organizing and staffing our company, business planning, raising capital, developing and optimizing our technology platform, identifying potential product candidates, undertaking research and nonclinical studies, clinical development activities, engaging in manufacturing for our development programs, and providing general and administrative support for these operations. We do not have any products approved for sale and have not generated any revenue from product sales.
Previous wording
Expenses for our FECD program decreased compared to the same period of the prior year primarily due to nonclinical activities that took place in 2024. These decreases were partially offset by costs associated with DT-168 clinical activities and costs for additional development activities.
Current wording
Expenses for our FECD program increased compared to the same period of the prior year primarily due to costs associated with DT-168 clinical activities and costs for additional development activities.
Previous wording
General and Administrative Expenses. The increase in general and administrative expenses was primarily due to a $0.8 million increase in employee compensation, including $0.6 million in stock-based compensation expenses, and a $0.5 million increase in professional services expenses including a $0.4 million one-time charge to write off previously deferred financing costs during the three months ended June 30, 2025 as compared to the same period in 2024.
Current wording
General and Administrative Expenses. The increase in general and administrative expenses was primarily due to a $0.3 million increase in stock-based compensation expenses and $0.1 million in other expenses incurred during the three months ended September 30, 2025 as compared to the same period in 2024.
Previous wording
The following table summarizes our operating expenses for the six months ended June 30, 2025 (in thousands):
Current wording
The following table summarizes our operating expenses for the nine months ended September 30, 2025 (in thousands):
Previous wording
Research and Development Expenses. Research and development expenses for our FA program increased during the six months ended June 30, 2025 compared to the same period of the prior year primarily due to costs attributable to DT-216 clinical activities and costs for additional development activities.
Current wording
Research and Development Expenses. Research and development expenses for our FA program increased during the nine months ended September 30, 2025 compared to the same period of the prior year primarily due to costs attributable to DT-216 clinical activities and costs for additional development activities.
Previous wording
Expenses for our FECD program increased compared to the same period of the prior year primarily due to costs associated with DT-168 clinical activities and costs for additional development activities. These increases were partially offset by decreases associated with nonclinical activities that took place in 2024.
Current wording
Expenses for our FECD program increased compared to the same period of the prior year primarily due to costs associated with DT-168 clinical activities and costs for additional development activities.
Previous wording
The following table summarizes our research and development expenses by program, direct and indirect costs for the six months ended June 30, 2025 (in thousands):
Current wording
The following table summarizes our research and development expenses by program, direct and indirect costs for the nine months ended September 30, 2025 (in thousands):
Previous wording
General and Administrative Expenses. The increase in general and administrative expenses was primarily due to a $1.1 million increase in employee compensation, including a $0.9 million in stock-based compensation expenses, a $0.5 million increase in professional services expenses including a $0.4 million one-time charge to write off previously deferred financing costs and $0.1 million in other expenses incurred during the six months ended June 30, 2025 as compared to the same period in 2024.
Current wording
General and Administrative Expenses. The increase in general and administrative expenses was primarily due to a $1.4 million increase in employee compensation, including a $1.2 million in stock-based compensation expenses, a $0.6 million increase in professional services expenses including a $0.4 million one-time charge to write off previously deferred financing costs and $0.1 million in other expenses incurred during the nine months ended September 30, 2025 as compared to the same period in 2024.
Previous wording
As of June 30, 2025, we had $216.3 million of combined cash, cash equivalents and investment securities, a decrease of $29.2 million from the $245.5 million of cash, cash equivalents and investment securities at December 31, 2024. Further detail of the change in our cash and cash equivalents for the six months ended June 30, 2025 and 2024 is summarized below (in thousands):
Current wording
As of September 30, 2025, we had $206.0 million of combined cash, cash equivalents and investment securities, a decrease of $39.5 million from the $245.5 million of cash, cash equivalents and investment securities at December 31, 2024. Further detail of the change in our cash and cash equivalents for the nine months ended September 30, 2025 and 2024 is summarized below (in thousands):
Previous wording
Operating Activities. The increase in our net cash used in operating activities was primarily due to the $13.9 million increase in net loss, partially offset by an increase in non-cash expenses and net working capital for the six months ended June 30, 2025 compared to the six months ended June 30, 2024.
Current wording
Operating Activities. The increase in our net cash used in operating activities was primarily due to the $17.9 million increase in net loss, partially offset by an increase in non-cash expenses and net working capital for the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024.
Previous wording
Investing Activities. The decrease in net cash provided by investing activities was primarily due to a net decrease in cash provided from the maturities and purchases of our investment securities during the six months ended June 30, 2025 compared to the six months ended June 30, 2024. We have classified our investment securities as available-for-sale and all investments are made in accordance with our investment policy.
Current wording
Investing Activities. The decrease in net cash provided by investing activities was primarily due to a net decrease in cash provided from the maturities and purchases of our investment securities during the nine months ended September 30, 2025 compared to the nine months ended September 30, 2024. We have classified our investment securities as available-for-sale and all investments are made in accordance with our investment policy.
Previous wording
Financing Activities. Net cash provided by financing activities for the six months ended June 30, 2025 and 2024 was comprised of proceeds received from the issuance of common stock through our employee stock purchase plan and from employee stock option exercises.
Current wording
Financing Activities. Net cash provided by financing activities for the nine months ended September 30, 2025 and 2024 was comprised of proceeds received from the issuance of common stock through our employee stock purchase plan and from employee stock option exercises.
Previous wording
The Company recorded a one-time non-cash charge to general and administrative expenses of approximately $0.4 million during the three and six months ended June 30, 2025. The one-time charge was previously capitalized in other assets on the Company's balance sheets and related to deferred financing costs for the 2022 Registration Statement.
Current wording
The Company recorded a one-time non-cash charge to general and administrative expenses of approximately $0.4 million during the nine months ended September 30, 2025. The one-time charge was previously capitalized in other assets on the Company's balance sheets and related to deferred financing costs for the 2022 Registration Statement.
Previous wording
For the six months ended June 30, 2025, no payments were made pursuant to the License Agreement. In 2022, pursuant to the License Agreement, we paid $0.1 million to WARF upon the acceptance of an IND in the U.S. We will be required to make further aggregate milestone payments of up to $17.5 million upon achievement of certain other regulatory and commercial milestones. We may also be required to pay royalties based on annual net product sales in the low single digits on our or our sublicensees’ net product sales on a country-by-country and product-by-product basis, and are subject to a minimum royalty of $0.1 million per calendar year upon first commercial product sale. Further, we may be required to pay sublicense fees in the mid-single digits percentage for fees, royalties or other payments earned from the granting of sublicenses to the WARF patents and know-how. The Company will recognize these milestone payments and royalties when paid or payable. There were no additional payments or royalties recorded through June 30, 2025.
Current wording
For the nine months ended September 30, 2025, no payments were made pursuant to the License Agreement. In 2022, pursuant to the License Agreement, we paid $0.1 million to WARF upon the acceptance of an IND in the United States. We will be required to make further aggregate milestone payments of up to $17.5 million upon achievement of certain other regulatory and commercial milestones. We may also be required to pay royalties based on annual net product sales in the low single digits on our or our sublicensees’ net product sales on a country-by-country and product-by-product basis, and are subject to a minimum royalty of $0.1 million per calendar year upon first commercial product sale. Further, we may be required to pay sublicense fees in the mid-single digits percentage for fees, royalties or other payments earned from the granting of sublicenses to the WARF patents and know-how. The Company will recognize these milestone payments and royalties when paid or payable. There were no additional payments or royalties recorded through September 30, 2025.
Previous wording
We are responsible for reimbursing WARF for costs incurred in connection with prosecuting and maintaining patent rights that are specific to the License Agreement. Expenses recognized in connection with legal patent fees under this License Agreement were immaterial for each of the six months ended June 30, 2025 and 2024.
Current wording
We are responsible for reimbursing WARF for costs incurred in connection with prosecuting and maintaining patent rights that are specific to the License Agreement. Expenses recognized in connection with legal patent fees under this License Agreement were immaterial for each of the nine months ended September 30, 2025 and 2024.