Design Therapeutics Highlights Progress Across Lead GeneTAC® Programs and Reports Fourth Quarter and Full Year 2024 Financial Results

Rhea-AI Summary

Design Therapeutics (DSGN) has reported significant progress in its GeneTAC® programs and financial results for Q4 and full year 2024. The company has initiated a Phase 1 Single Ascending Dose trial of DT-216P2 in healthy volunteers for Friedreich Ataxia (FA), with patient dosing planned for mid-2025. Dosing is complete in the DT-168 Phase 1 healthy volunteer trial for Fuchs Endothelial Corneal Dystrophy (FECD), with data expected in H1 2025.

The company has achieved its enrollment target for the FECD observational study with 250 patients. Additionally, Design expects to select a development candidate for Myotonic Dystrophy Type-1 (DM1) in 2025.

Financial highlights include:

• R&D expenses: $12.2M for Q4 2024, $44.4M for full year
• G&A expenses: $4.5M for Q4 2024, $18.0M for full year
• Net loss: $13.7M for Q4 2024, $49.6M for full year
• Cash position: $245.5M as of December 31, 2024

Positive

• Strong cash position of $245.5M expected to fund operations into 2029
• Phase 1 trial initiation for FA program with DT-216P2
• Completed Phase 1 dosing for FECD program
• Successful enrollment of 250 patients in FECD observational study

Negative

• Net loss of $49.6M for full year 2024
• Increased R&D expenses to $44.4M for 2024
• Clinical proof-of-concept data not expected until 2026

Insights

Biotechnology Clinical Development Expert

Design Therapeutics has reported meaningful progress across their GeneTAC® pipeline, highlighted by two key clinical programs advancing as planned. For Friedreich Ataxia (FA), the company has initiated a Phase 1 single ascending dose trial of DT-216P2 in healthy volunteers with a clear pathway to patient dosing in mid-2025. For Fuchs Endothelial Corneal Dystrophy (FECD), they've completed dosing in the Phase 1 healthy volunteer trial with data expected in H1 2025, while successfully enrolling their observational study.

What's particularly significant is the company's financial position. With $245.5 million in cash and securities, Design has an exceptionally long runway extending into 2029, providing sufficient capital to reach up to four clinical proof-of-concept data readouts without requiring additional financing. This runway substantially de-risks their development pathway compared to most clinical-stage biotechs.

While these programs remain in early clinical stages without efficacy data yet, the company is executing well on their development timelines across multiple indications. Their pipeline diversity (FA, FECD, DM1, and HD) provides strategic risk mitigation, and the anticipated clinical readouts in 2025-2026 will serve as important value inflection points. For a company with $289.9 million market cap, their robust cash position covers almost 85% of their current valuation, creating a favorable risk-reward profile as they approach these critical data milestones.

Financial Analyst

Design Therapeutics' financial results reveal a well-capitalized clinical-stage biotech with contained cash burn and substantial runway. Their $245.5 million cash position against a $289.9 million market cap creates an enterprise value of just $44.4 million - unusually low for a company with multiple clinical programs. This cash is projected to fund operations into 2029, representing approximately 4-5 years of runway, which is exceptionally long in the biotech sector.

The quarterly R&D expense of $12.2 million and G&A of $4.5 million suggest an annual burn rate of $60-70 million, aligning with their long runway projection. With net losses of $13.7 million for Q4 and $49.6 million for the full year, their spending remains disciplined despite advancing multiple programs.

What stands out is the capital efficiency - their current cash position could potentially fund four clinical proof-of-concept readouts, representing significant value inflection points. This contrasts with many comparable biotechs that require frequent dilutive financings between major milestones. For investors, this creates a unique proposition where the downside is partially protected by cash value while maintaining upside exposure to multiple clinical catalysts over the next 1-4 years. The diversified pipeline targeting multiple genetic diseases (FA, FECD, DM1, HD) further enhances the company's risk/reward profile by not being dependent on a single asset's success.

Initiated Phase 1 Single Ascending Dose Trial of DT-216P2 in Healthy Volunteers; Friedreich Ataxia (FA) Patient Dosing to Begin in mid-2025

Dosing Complete in DT-168 Phase 1 Healthy Volunteer Trial, with Data on Track for the First Half of 2025; Enrollment Target Achieved in Fuchs Endothelial Corneal Dystrophy (FECD) Observational Study

Selection of Development Candidate for Myotonic Dystrophy Type-1 (DM1) Expected in 2025

Well-Capitalized with Cash and Securities of $245.5 Million to Fund Operations Through Up to Four Potential Clinical Proof-of-Concept Data Sets

CARLSBAD, Calif., March 10, 2025 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced progress across its portfolio of GeneTAC^® candidates and reported fourth quarter and full year 2024 financial results.

“Thanks to the progress we have achieved so far this year, the first half of 2025 will be a busy one for Design, with data expected from our Phase 1 trial in FECD and the advancement of clinical activities in our FA program,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “We believe these programs lead a pipeline of GeneTAC^® small molecules capable of transforming the status quo in genomic medicines, with the potential for multiple clinical proof-of-concept data sets over the next few years.”

Corporate Highlights and Anticipated Upcoming Milestones

• Friedreich Ataxia (FA) Design has initiated dosing in a Phase 1 clinical trial in healthy volunteers in Australia to evaluate the safety and pharmacokinetics (PK) of single ascending doses of DT-216P2 via multiple routes of administration (intravenous infusion, subcutaneous infusion and subcutaneous injection). A Phase 1/2 multiple ascending dose (MAD) clinical trial to assess safety, PK and pharmacodynamics (PD) in FA patients is anticipated to begin in mid-2025. Data based on twelve weeks of DT-216P2 dosing in patients is anticipated in 2026.  
• Fuchs Endothelial Corneal Dystrophy (FECD) Design has completed dosing in a Phase 1 MAD clinical trial of DT-168 in healthy volunteers and expects to report data in the first half of 2025. The company achieved its enrollment goal for the observational study by recruiting and completing baseline assessments on approximately 250 FECD patients. Based on the baseline characteristics data, Design has chosen approximately 100 patients for future follow-up visits.
• Pipeline programs Design continues to advance preclinical work toward the selection of a development candidate for myotonic dystrophy type-1 (DM1) later in 2025. Preclinical characterization of several candidate molecules also continues in Huntington’s disease (HD).
  

Fourth Quarter and Full Year 2024 Financial Results

• R&D Expenses: Research and development (R&D) expenses were $12.2 million for the quarter ended December 31, 2024, and $44.4 million for the year ended December 31, 2024.
• G&A Expenses: General and administrative (G&A) expenses were $4.5 million for the quarter ended December 31, 2024, and $18.0 million for the year ended December 31, 2024.
• Net Loss: Net loss was $13.7 million for the quarter ended December 31, 2024, and $49.6 million for the year ended December 31, 2024.
• Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $245.5 million as of December 31, 2024, which the company expects to fund its planned operating expenses into 2029.

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, and DT-168, for Fuchs endothelial corneal dystrophy, the company is advancing programs in myotonic dystrophy type-1 and 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 projections from early-stage programs, nonclinical data and early-stage clinical data; the progression or completion of certain development activities, including the selection of development candidates; the initiation and progression of studies and clinical trials for DT-216P2 and DT-168 and the timing thereof; the expected timing for data readouts; Design’s pipeline, including the potential to have four programs with clinical proof-of-concept with Design’s current cash runway; 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,” “on track to,” “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 acceptance of INDs by the FDA or similar applications by foreign regulatory agencies for the conduct of planned clinical trials of our product candidates and our proposed design of future clinical trials; nonclinical development activities and results of nonclinical studies; conducting a clinical trial and patient enrollment, which are affected by many factors, and any difficulties or delays encountered with such clinical trial or patient enrollment 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; whether promising early research or clinical trials will demonstrate safety and/or efficacy in later nonclinical studies or 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 key third parties, including contract manufacturers and contract research organizations; 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; Design’s ability to recruit and retain key scientific or management personnel; and market conditions. 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 September 30, 2024, as filed with the SEC on November 7, 2024, and under the “Risk Factors” heading of Design’s Annual Report on Form 10-K for the fiscal year ended December 31, 2024, 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 Strategic Communications
renee@thrustsc.com

DESIGN THERAPEUTICS, INC. CONDENSED STATEMENTS OF OPERATIONS (in thousands, except share and per share data)
		Quarter Ended December 31,								Year Ended December 31,
		2024				2023				2024				2023
		(unaudited)
Operating expenses:
Research and development		$	12,157			$	11,012			$	44,350			$	57,063
General and administrative			4,537				4,109				18,033				21,127
Total operating expenses			16,694				15,121				62,383				78,190
Loss from operations			(16,694	)			(15,121	)			(62,383	)			(78,190	)
Interest income			3,043				3,279				12,795				11,328
Net loss		$	(13,651	)		$	(11,842	)		$	(49,588	)		$	(66,862	)
Net loss per share, basic and diluted		$	(0.24	)		$	(0.21	)		$	(0.88	)		$	(1.19	)
Weighted-average shares of common stock outstanding, basic and diluted			56,681,940				56,090,912				56,587,142				55,984,670

DESIGN THERAPEUTICS, INC. CONDENSED BALANCE SHEETS (in thousands)
		December 31,				December 31,
		2024				2023
Assets
Current assets:
Cash, cash equivalents and investment securities		$	245,477			$	281,798
Prepaid expenses and other current assets			2,563				2,786
Total current assets			248,040				284,584
Property and equipment, net			1,410				1,691
Right-of-use asset, related party			2,216				2,938
Other assets			427				430
Total assets		$	252,093			$	289,643
Liabilities and Stockholders’ Equity
Current liabilities:
Accounts payable		$	2,186			$	1,940
Accrued expenses and other current liabilities			6,276				7,682
Total current liabilities			8,462				9,622
Operating lease liability, net, related party			1,534				2,334
Total liabilities			9,996				11,956
Total stockholders’ equity			242,097				277,687
Total liabilities and stockholders’ equity		$	252,093			$	289,643

FAQ

What is the cash runway for Design Therapeutics (DSGN) as of Q4 2024?

DSGN reported $245.5M in cash and securities as of December 31, 2024, expected to fund planned operations into 2029.

When will Design Therapeutics begin patient dosing for its Friedreich Ataxia (FA) program?

DSGN plans to begin Phase 1/2 multiple ascending dose trials in FA patients in mid-2025.

What was Design Therapeutics' net loss for full year 2024?

DSGN reported a net loss of $49.6 million for the year ended December 31, 2024.

When will Design Therapeutics report Phase 1 data for its FECD program?

DSGN expects to report Phase 1 data from the DT-168 healthy volunteer trial in the first half of 2025.

How many patients were enrolled in DSGN's FECD observational study?

Design Therapeutics enrolled approximately 250 FECD patients, with 100 selected for future follow-up visits.