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Design Therapeutics Announces Third Quarter 2024 Financial Results and Reviews Near-term Milestones for GeneTACTM Portfolio

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Design Therapeutics (NASDAQ: DSGN) announced its Q3 2024 financial results and pipeline updates. The company initiated a Phase 1 trial for FECD with data expected in H1 2025, and plans to start a Phase 1 trial for Friedreich Ataxia in H1 2025. The company reported R&D expenses of $11.9M, G&A expenses of $4.4M, and a net loss of $13.0M for Q3 2024. With $254.1M in cash and securities, Design Therapeutics expects to fund operations into 2029, supporting up to four potential clinical proof-of-concept data sets. The company is also advancing preclinical programs in Huntington's Disease and Myotonic Dystrophy Type-1.

Design Therapeutics (NASDAQ: DSGN) ha annunciato i risultati finanziari per il terzo trimestre del 2024 e aggiornamenti sulla sua pipeline. L'azienda ha avviato uno studio di Fase 1 per FECD, con dati previsti per la prima metà del 2025, e prevede di iniziare uno studio di Fase 1 per Atassia di Friedreich nella prima metà del 2025. L'azienda ha riportato spese in R&S di $11,9 milioni, spese generali di $4,4 milioni e una perdita netta di $13,0 milioni per il terzo trimestre del 2024. Con $254,1 milioni in contante e titoli, Design Therapeutics si aspetta di finanziare le operazioni fino al 2029, supportando fino a quattro potenziali set di dati clinici di prova di concetto. L'azienda sta inoltre avanzando programmi preclinici in Malattia di Huntington e Distrofia Miotonica Tipo-1.

Design Therapeutics (NASDAQ: DSGN) anunció sus resultados financieros del tercer trimestre de 2024 y actualizaciones de su pipeline. La compañía inició un ensayo de Fase 1 para FECD con datos esperados en la primera mitad de 2025, y planea iniciar un ensayo de Fase 1 para Ataxia de Friedreich en la primera mitad de 2025. La empresa reportó gastos en I+D de $11.9 millones, gastos administrativos de $4.4 millones y una pérdida neta de $13.0 millones para el tercer trimestre de 2024. Con $254.1 millones en efectivo y valores, Design Therapeutics espera financiar sus operaciones hasta 2029, apoyando hasta cuatro posibles conjuntos de datos clínicos de prueba de concepto. La empresa también está avanzando en programas preclínicos para Enfermedad de Huntington y Distrofia Miotónica Tipo-1.

Design Therapeutics (NASDAQ: DSGN)는 2024년 3분기 재무 결과 및 파이프라인 업데이트를 발표했습니다. 이 회사는 FECD에 대한 1상 시험을 시작했으며, 데이터는 2025년 상반기에 예상됩니다. 또한, 2025년 상반기에 프리드리히 운동실조증에 대한 1상 시험을 시작할 계획입니다. 이 회사는 2024년 3분기에 R&D 비용으로 1,190만 달러, 일반 관리비로 440만 달러, 순손실로 1,300만 달러를 보고했습니다. 현금 및 유가증권으로 2억 5,410만 달러를 보유하고 있는 Design Therapeutics는 2029년까지 운영 자금을 지원할 것으로 기대하고 있으며, 최대 4개의 잠재적인 임상 개념 증명 데이터 세트를 지원할 예정입니다. 이 회사는 또 헌팅턴병미오토닉 근위축증 1형에 대한 전임상 프로그램을 진행하고 있습니다.

Design Therapeutics (NASDAQ: DSGN) a annoncé ses résultats financiers pour le troisième trimestre 2024 et des mises à jour sur son pipeline. L'entreprise a lancé un essai de Phase 1 pour FECD, avec des données attendues au premier semestre 2025, et prévoit de démarrer un essai de Phase 1 pour Ataxie de Friedreich au premier semestre 2025. L'entreprise a déclaré des dépenses de R&D de 11,9 millions de dollars, des dépenses générales et administratives de 4,4 millions de dollars, et une perte nette de 13,0 millions de dollars pour le troisième trimestre 2024. Avec 254,1 millions de dollars en liquidités et titres, Design Therapeutics s'attend à financer ses opérations jusqu'en 2029, soutenant jusqu'à quatre ensembles de données cliniques potentielles de preuve de concept. L'entreprise fait également progresser des programmes précliniques pour la maladie de Huntington et la dystrophie miotonique de type 1.

Design Therapeutics (NASDAQ: DSGN) gab seine finanziellen Ergebnisse für das dritte Quartal 2024 und Aktualisierungen zu seiner Pipeline bekannt. Das Unternehmen begann eine Phase-1-Studie für FECD, von der die Daten in der ersten Hälfte von 2025 erwartet werden, und plant, in der ersten Hälfte von 2025 mit einer Phase-1-Studie für Friedreich-Ataxie zu beginnen. Das Unternehmen berichtete über Forschungs- und Entwicklungsausgaben von 11,9 Millionen US-Dollar, Verwaltungskosten von 4,4 Millionen US-Dollar und einen Nettoverlust von 13 Millionen US-Dollar für das dritte Quartal 2024. Mit 254,1 Millionen US-Dollar in Bargeld und Wertpapieren erwartet Design Therapeutics, seinen Betrieb bis 2029 zu finanzieren und dabei bis zu vier potenzielle klinische Nachweisdaten zu unterstützen. Das Unternehmen entwickelt auch präklinische Programme in der Huntington-Krankheit und der Myotone Dystrophie Typ-1.

Positive
  • Strong cash position of $254.1M providing runway into 2029
  • Initiated Phase 1 trial for FECD program
  • Multiple clinical milestones expected in H1 2025
Negative
  • Net loss of $13.0M in Q3 2024
  • Increased R&D expenses of $11.9M in Q3 2024

Insights

The Q3 results and pipeline update reveal significant progress in Design Therapeutics' GeneTAC™ portfolio. The $254.1 million cash position provides substantial runway into 2029, supporting multiple clinical programs through proof-of-concept stages. The quarterly net loss of $13 million and R&D expenses of $11.9 million reflect appropriate investment in clinical development.

Two key catalysts are approaching in H1 2025: data readout from the FECD Phase 1 trial and initiation of the FA program's Phase 1 study. The advancement of preclinical programs in HD and DM1 demonstrates pipeline expansion beyond lead programs. The company's focus on rare genetic diseases with its GeneTAC™ platform represents a novel therapeutic approach in areas of high unmet need.

The burn rate appears well-managed relative to cash reserves, suggesting efficient capital allocation. The extended runway provides multiple shots on goal without immediate financing pressure.

Fuchs Endothelial Corneal Dystrophy (FECD) Phase 1 Trial Initiated with Data Expected in the First Half of 2025

Friedreich Ataxia (FA) Program on Track to Initiate Phase 1 Single Ascending Dose, Healthy Volunteer Study in the First Half of 2025

Active Research Pipeline Advancing with Programs in Myotonic Dystrophy Type-1 (DM1) and Huntington’s Disease (HD)

Well-Capitalized with Cash and Securities of $254.1 Million to Fund Operations Through Up to Four Potential Clinical Proof-of-Concept Data Sets

CARLSBAD, Calif., Nov. 07, 2024 (GLOBE NEWSWIRE) -- Design Therapeutics, Inc. (Nasdaq: DSGN), a clinical-stage biotechnology company developing treatments for serious degenerative genetic diseases, today announced its third quarter 2024 financial results and reviewed anticipated upcoming program milestones for its portfolio of GeneTAC™ candidates.

“Thanks to our progress so far this year, the first half of 2025 will be a busy one, with data expected from our ongoing Phase 1 trial in FECD and the start of clinical activities for 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 is on track to initiate the Phase 1 single ascending dose, normal healthy volunteer trial for DT-216P2 in the first half of 2025. The company anticipates beginning FA patient dosing later in 2025. 
  • Fuchs Endothelial Corneal Dystrophy (FECD) The company has initiated Phase 1 development for DT-168 in normal healthy volunteers, with initial data expected in the first half of 2025. In parallel, Design continues its ongoing FECD observational study.
  • Pipeline programs Design also continues to advance preclinical characterization of several lead molecules toward the selection of development candidates for Huntington’s disease (HD) and myotonic dystrophy type-1 (DM1) in anticipation of future IND submissions.

Third Quarter 2024 Financial Results

  • R&D Expenses: Research and development (R&D) expenses were $11.9 million for the quarter ended September 30, 2024.
  • G&A Expenses: General and administrative (G&A) expenses were $4.4 million for the quarter ended September 30, 2024.
  • Net Loss: Net loss was $13.0 million for the quarter ended September 30, 2024.
  • Cash Position and Operating Runway: Cash, cash equivalents and marketable securities were $254.1 million as of September 30, 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 lead GeneTAC™ small molecule, DT-216, in development for patients with Friedreich ataxia, the company is advancing programs in Fuchs endothelial corneal dystrophy, Huntington’s disease and myotonic dystrophy type-1. 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 June 30, 2024, as filed with the SEC on August 5, 2024, and under the “Risk Factors” heading of Design’s Quarterly Report on Form 10-Q for the quarter ended September 30, 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)
      
 Three Months Ended September 30,  Nine Months Ended September 30, 
 2024  2023  2024  2023 
 (unaudited) 
Operating expenses:           
Research and development$11,876  $13,257  $32,193  $46,051 
General and administrative 4,370   5,565   13,496   17,018 
Total operating expenses 16,246   18,822   45,689   63,069 
Loss from operations (16,246)  (18,822)  (45,689)  (63,069)
Other income, net 3,207   3,033   9,752   8,049 
Net loss$(13,039) $(15,789) $(35,937) $(55,020)
            
Net loss per share, basic and diluted$(0.23) $(0.28) $(0.64) $(0.98)
Weighted-average shares of common stock outstanding, basic and diluted 56,620,731   55,988,691   56,555,312   55,948,867 
                


DESIGN THERAPEUTICS, INC.
CONDENSED BALANCE SHEETS

(in thousands)
      
 September 30,  December 31, 
 2024  2023 
 (unaudited)    
Assets     
Current assets:     
Cash, cash equivalents and investment securities$254,074  $281,798 
Prepaid expenses and other current assets 3,168   2,786 
Total current assets 257,242   284,584 
Property and equipment, net 1,559   1,691 
Right-of-use asset, related party 2,401   2,938 
Other assets 427   430 
Total assets$261,629  $289,643 
Liabilities and Stockholders’ Equity     
Current liabilities:     
Accounts payable$1,470  $1,940 
Accrued expenses and other current liabilities 5,962   7,682 
Total current liabilities 7,432   9,622 
Operating lease liability, net, related party 1,744   2,334 
Total liabilities 9,176   11,956 
Total stockholders’ equity 252,453   277,687 
Total liabilities and stockholders’ equity$261,629  $289,643 
        

FAQ

What is Design Therapeutics' (DSGN) cash position as of Q3 2024?

Design Therapeutics reported cash, cash equivalents and marketable securities of $254.1 million as of September 30, 2024.

When will Design Therapeutics (DSGN) report FECD Phase 1 trial data?

Design Therapeutics expects to report initial data from the FECD Phase 1 trial in the first half of 2025.

What was Design Therapeutics' (DSGN) net loss in Q3 2024?

Design Therapeutics reported a net loss of $13.0 million for the quarter ended September 30, 2024.

When will Design Therapeutics (DSGN) start the Friedreich Ataxia Phase 1 trial?

Design Therapeutics plans to initiate the Phase 1 single ascending dose trial in healthy volunteers for Friedreich Ataxia in the first half of 2025.

Design Therapeutics, Inc.

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