Design Therapeutics Announces Third Quarter 2026 Financial Results and Recent Business Updates

Expected 2027 clinical readouts accompany a wider quarterly net loss and lower cash and securities than at year-end 2025.

Rhea-AI Summary

Design Therapeutics (DSGN) reported third-quarter 2026 financial results and outlined upcoming clinical milestones for its portfolio of genetic disease treatments.

Quarterly net loss widened to $20.288 million from $16.997 million a year earlier; loss per share increased to $0.32 from $0.30. Research and development expenses were $16.926 million, versus $14.589 million. Cash, cash equivalents and investment securities totaled $208.608 million at September 30, versus $219.845 million at December 31, 2025.

Design expects a DT-216P2 registrational plan update in Q4 2026 and 12-week RESTORE-FA trial data in Q1 2027. It also expects data in 2027 from the ongoing Phase 1 DT-818 trial in myotonic dystrophy type-1 and Phase 2 DT-168 trial in Fuchs endothelial corneal dystrophy. Preclinical characterization continues for Huntington’s disease candidates.

5 points · 0 major

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0 major · 10 points

Hollow bars mark forward-looking points. How the balance works

Positive

• Moderate point. Forward-looking: it has not happened yet and may not happen.DT-216P2 milestones expected: registrational plan update in Q4 2026 and 12-week RESTORE-FA data in Q1 2027.
• Minor point. Forward-looking: it has not happened yet and may not happen.DT-818 Phase 1 trial in myotonic dystrophy type-1 continues, with data anticipated in 2027.
• Minor point. Forward-looking: it has not happened yet and may not happen.DT-168 Phase 2 trial in Fuchs endothelial corneal dystrophy continues, with data expected in 2027.
• Minor pointHuntington’s disease program continues preclinical characterization of several candidate molecules.
• Minor pointNine-month loss per share narrowed to $0.93 from $0.95 in 2025.

Negative

• Moderate pointQuarterly net loss widened to $20.288 million from $16.997 million a year earlier.
• Minor pointQuarterly loss per share increased to $0.32 from $0.30 a year earlier.
• Minor pointCash and securities decreased to $208.608 million at September 30, 2026, from $219.845 million at year-end 2025.
• Minor pointQuarterly research and development expenses rose to $16.926 million from $14.589 million a year earlier.
• Minor pointQuarterly general and administrative expenses rose to $5.304 million from $4.722 million a year earlier.

5 minor points

• Minor pointQuarterly other income, net declined to $1.942 million from $2.314 million a year earlier.
• Minor pointNine-month net loss widened to $58.089 million from $53.795 million in 2025.
• Minor pointNine-month research and development expenses rose to $47.693 million from $45.704 million in 2025.
• Minor pointNine-month general and administrative expenses rose to $16.420 million from $15.594 million in 2025.
• Minor pointNine-month other income, net declined to $6.024 million from $7.503 million in 2025.

Key Figures

Registrational plan update: Q4 2026 RESTORE-FA data timing: 12-week data in Q1 2027 R&D expenses: $16.9 million +3 more

Registrational plan update

Q4 2026

DT-216P2

RESTORE-FA data timing

12-week data in Q1 2027

DT-216P2

R&D expenses

$16.9 million

Quarter ended September 30, 2026

G&A expenses

$5.3 million

Quarter ended September 30, 2026

Net loss

$20.3 million

Quarter ended September 30, 2026

Cash and securities

$208.6 million

As of September 30, 2026

Previous Earnings Reports

1 past event · Latest: Apr 28

Same Type 1 event

1. Apr 28

   Q1 earnings

   24h Move

   +23.3%

   Reported $222.8 million cash and securities and expected operations funding into 2029.

24h Move is the share-price change in the day after each event; other market factors may also have contributed.

Key Terms

multiple-ascending dose, biomarker, allele

3 terms

multiple-ascending dose medical

"The Phase 1 multiple-ascending dose (MAD) trial of DT-818"

A multiple-ascending dose study is an early-stage clinical trial where groups of volunteers receive a drug repeatedly at progressively higher dose levels to see how the body handles it and whether repeated dosing causes side effects. Think of it as gradually increasing the weight on a workout machine to find the safe, effective range; for investors, results reveal safety, how the drug behaves over time, and whether the program can move into larger, more costly trials.

biomarker medical

"A Phase 2 biomarker trial of DT-168 is ongoing"

A biomarker is a measurable indicator found in the body, such as in blood or tissues, that provides information about health, disease, or how the body responds to treatment. For investors, biomarkers can signal the potential success or risk of medical products or therapies, influencing the value of related companies and industry trends. They act like signals or clues that help assess the progress of medical advancements and their market impact.

allele medical

"selectively reduce transcription of the mutant DMPK allele"

One of two or more alternative forms of the same gene at a specific location (locus) on a chromosome. Alleles are different DNA sequences that can arise from single‑base changes, insertions, deletions or larger rearrangements; in organisms with paired chromosomes an individual can carry two identical alleles (homozygous) or two different alleles (heterozygous) at a locus. Different alleles can produce different molecular products or levels of gene activity, which may affect traits, biochemical function, or disease susceptibility, but the term itself only denotes the variant form of the gene.

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DT-216P2 Anticipated Milestones: Registrational Plan Update in Q4 2026 and 12-Week RESTORE-FA Data in Q1 2027

Cash and securities of $208.6 million at quarter-end provide strong financial runway

CARLSBAD, Calif., Nov. 04, 2026 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today reported third quarter 2026 financial results and highlighted business updates and upcoming milestones across its GeneTAC^® portfolio.

“We remain focused on evaluating DT-216P2 over longer treatment durations and preparing for the next stage of clinical development in Friedreich ataxia,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “In DM1, our Phase 1 trial of DT-818 is evaluating an approach designed to selectively reduce transcription of the mutant DMPK allele, targeting the source of toxic RNA. Clinical readouts expected in 2027 will help guide these programs as we pursue our vision for the GeneTAC^® platform: to address the underlying causes of serious genetic diseases and deliver meaningful benefits for patients.”

Corporate Highlights

• Friedreich ataxia (FA): The Phase 1/2 multiple ascending dose RESTORE-FA trial of DT-216P2 is ongoing. Design expects to provide an update on its registrational plans in the fourth quarter of 2026, with data following 12 weeks of treatment expected in the first quarter of 2027.
• Myotonic Dystrophy Type-1 (DM1): The Phase 1 multiple-ascending dose (MAD) trial of DT-818, a GeneTAC^® small molecule designed to selectively reduce transcription of the mutant DMPK allele in patients with DM1, is ongoing. Design anticipates reporting data from this trial in 2027.
• Fuchs Endothelial Corneal Dystrophy (FECD): A Phase 2 biomarker trial of DT-168 is ongoing to evaluate safety, tolerability and corneal endothelium biomarkers in FECD patients who are scheduled for corneal transplant surgery, with data expected in 2027.
• Huntington’s disease (HD): Design continues to advance preclinical characterization of several candidate molecules for its Huntington’s disease program.
  
  

Third Quarter 2026 Financial Results

• R&D Expenses: Research and development (R&D) expenses were $16.9 million for the quarter ended September 30, 2026.
• G&A Expenses: General and administrative (G&A) expenses were $5.3 million for the quarter ended September 30, 2026.
• Net Loss: Net loss was $20.3 million for the quarter ended September 30, 2026.
• Cash Position: Cash, cash equivalents and investment securities were $208.6 million as of September 30, 2026.
  
  

About Design Therapeutics
Design Therapeutics is a clinical-stage biotechnology company developing a new class of therapies based on its platform of GeneTAC^® gene targeted chimera small molecules. The company’s GeneTAC^® molecules are designed to either dial up or dial down the expression of a specific disease-causing gene to address the underlying cause of disease. In addition to its clinical-stage GeneTAC^® programs, DT-216P2, in development for patients with Friedreich ataxia, DT-168, for Fuchs endothelial corneal dystrophy, and DT-818, for myotonic dystrophy type-1, the company is advancing a program in Huntington’s disease. Discovery efforts are underway for multiple genomic medicines. For more information, please visit designtx.com.

Forward-Looking Statements
Statements in this press release that are not purely historical in nature are “forward-looking statements” within the meaning of the Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to: the progression or completion of certain development activities; the initiation and progression of studies and clinical trials for DT-216P2, DT-168 and DT-818 and the timing thereof; the anticipated timing for data readouts and other program updates; Design's ability to advance the GeneTAC^® platform; Design’s estimated cash runway and the sufficiency of its resources to support its planned operations; and the capabilities and potential advantages of Design’s pipeline of GeneTAC^® molecules. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. Words such as “believes,” “designed to,” “anticipates,” “capable of,” “plans to,” “expects,” “estimate,” “intends,” “will,” “potential” and similar expressions are intended to identify forward-looking statements. These forward-looking statements are based upon Design’s current expectations and involve assumptions that may never materialize or may prove to be incorrect. Actual results and the timing of events could differ materially from those anticipated in such forward-looking statements as a result of various risks and uncertainties, which include, without limitation, risks and uncertainties associated with: the data we observe from early clinical and nonclinical studies may impact our clinical development plans; pursuing a biomarker-driven clinical development strategy carries increased risks as there are currently a limited number of approved biomarker-specific therapies; nonclinical development activities and results of nonclinical studies; conducting a clinical trial and patient enrollment and retention, which are affected by many factors, and any difficulties or delays encountered with such clinical trial or patient enrollment or retention may delay or otherwise adversely affect Design’s clinical development plans; the process of discovering and developing therapies that are safe and effective for use as human therapeutics and operating as a development stage company; undesirable side effects or other undesirable properties, which could cause Design or regulatory authorities to suspend or discontinue clinical trials and thereby delay or prevent Design’s product candidates’ development or regulatory approval; Design’s ability to develop, initiate or complete nonclinical studies and clinical trials for its product candidates on the timeframe anticipated, or at all; whether promising early research or clinical trials will result in demonstrated safety and/or efficacy in later clinical trials; changes in Design’s plans to develop its product candidates; reliance on third parties to successfully conduct clinical trials and nonclinical studies; competitive products, which may make any products we develop or seek to develop obsolete or noncompetitive; Design’s reliance on third parties, including contract manufacturers and contract research organizations; interactions with regulatory authorities; Design’s ability to raise any additional funding it will need to continue to pursue its business and product development plans; regulatory developments in the United States and foreign countries; Design’s ability to obtain and maintain intellectual property protection for its product candidates; and Design’s ability to recruit and retain key scientific or management personnel. For a more detailed discussion of these and other factors, please refer to Design’s filings with the Securities and Exchange Commission (“SEC”), including under the “Risk Factors” heading of Design’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2026, as filed with the SEC on August 3, 2026, and under the “Risk Factors” heading of Design’s Quarterly Report on Form 10-Q for the quarter ended September 30, 2026, being filed with the SEC later today. You are cautioned not to place undue reliance on these forward-looking statements, which speak only as of the date hereof. All forward-looking statements are qualified in their entirety by this cautionary statement and Design undertakes no obligation to revise or update this press release to reflect events or circumstances after the date hereof, except as required by law.

Contact:
Renee Leck, THRUST
renee@thrustsc.com

DESIGN THERAPEUTICS, INC.
CONDENSED STATEMENTS OF OPERATIONS
(in thousands, except share and per share data)
			Three Months Ended September 30,								Nine Months Ended September 30,
			2026				2025				2026				2025
			(unaudited)								(unaudited)
Operating expenses:
Research and development		$	16,926			$	14,589			$	47,693			$	45,704
General and administrative			5,304				4,722				16,420				15,594
Total operating expenses			22,230				19,311				64,113				61,298
Loss from operations			(22,230	)			(19,311	)			(64,113	)			(61,298	)
Other income, net			1,942				2,314				6,024				7,503
Net loss		$	(20,288	)		$	(16,997	)		$	(58,089	)		$	(53,795	)
Net loss per share, basic and diluted		$	(0.32	)		$	(0.30	)		$	(0.93	)		$	(0.95	)
Weighted-average shares of common stock outstanding, basic and diluted			63,035,529				56,950,999				62,330,974				56,856,779

DESIGN THERAPEUTICS, INC.
CONDENSED BALANCE SHEETS
(in thousands)
		September 30,			December 31,
		2026			2025
		(unaudited)
Assets
Current assets:
Cash, cash equivalents and investment securities		$	208,608		$	219,845
Prepaid expenses and other current assets			5,355			3,939
Total current assets			213,963			223,784
Property and equipment, net			573			981
Right-of-use asset			2,272			1,438
Total assets		$	216,808		$	226,203
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable		$	2,078		$	2,312
Accrued expenses and other current liabilities			10,675			10,743
Total current liabilities			12,753			13,055
Operating lease liability			1,859			645
Total liabilities			14,612			13,700
Total stockholders’ equity			202,196			212,503
Total liabilities and stockholders’ equity		$	216,808		$	226,203

FAQ

AI-generated questions and answers. How Rhea-AI works. Not financial advice.

When does Design Therapeutics expect DT-216P2 trial data and a registrational plan update?

Design expects a registrational plan update in Q4 2026 and data following 12 weeks of treatment in Q1 2027. The Phase 1/2 RESTORE-FA trial is ongoing in Friedreich ataxia.

What was Design Therapeutics’ third-quarter 2026 net loss?

Design Therapeutics recorded a $20.288 million net loss for the quarter ended September 30, 2026, compared with $16.997 million a year earlier. Basic and diluted loss per share was $0.32, versus $0.30.

What is Design Therapeutics evaluating in the DT-168 Phase 2 trial?

The DT-168 Phase 2 trial evaluates safety, tolerability and corneal endothelium biomarkers in patients with Fuchs endothelial corneal dystrophy who are scheduled for corneal transplant surgery.