Design Therapeutics Highlights Progress Across Lead GeneTAC® Programs and Reports Second Quarter 2025 Financial Results
Design Therapeutics (NASDAQ:DSGN) reported progress in its GeneTAC® programs and Q2 2025 financial results. The company announced encouraging early pharmacokinetics data for DT-216P2 in Friedreich Ataxia (FA), showing favorable translation from non-human primates to humans via both intravenous and subcutaneous administration.
Key developments include the ongoing RESTORE-FA Phase 1/2 trial of DT-216P2 outside the US, while addressing an FDA clinical hold notice. The company also initiated a Phase 2 biomarker trial for DT-168 in Fuchs Endothelial Corneal Dystrophy (FECD) patients. Design reported $216.3 million in cash and securities, with a Q2 net loss of $19.1 million.
[ "Favorable pharmacokinetics data translation from non-human primates to humans for DT-216P2", "DT-216P2 showed improved exposure and PK parameters compared to previous formulation", "Strong cash position of $216.3 million to support pipeline development", "Advancement of multiple clinical programs including FA and FECD treatments" ]Design Therapeutics (NASDAQ:DSGN) ha riportato progressi nei suoi programmi GeneTAC® e i risultati finanziari del secondo trimestre 2025. L'azienda ha annunciato dati farmacocinetici preliminari incoraggianti per DT-216P2 nella Friedreich Ataxia (FA), evidenziando una traduzione favorevole dai primati non umani all'uomo sia tramite somministrazione endovenosa che sottocutanea.
Gli sviluppi chiave includono il proseguimento dello studio RESTORE-FA di fase 1/2 di DT-216P2 al di fuori degli Stati Uniti, con la gestione di una sospensione clinica da parte della FDA. Inoltre, l'azienda ha avviato uno studio biomarcatori di fase 2 per DT-168 in pazienti con distrofia corneale endoteliale di Fuchs (FECD). Design Therapeutics ha riportato 216,3 milioni di dollari in liquidità e titoli, con una perdita netta di 19,1 milioni di dollari nel secondo trimestre.
- Traduzione favorevole dei dati farmacocinetici dai primati non umani all'uomo per DT-216P2
- DT-216P2 ha mostrato un'esposizione e parametri farmacocinetici migliorati rispetto alla formulazione precedente
- Solida posizione di cassa di 216,3 milioni di dollari a sostegno dello sviluppo della pipeline
- Avanzamento di più programmi clinici, inclusi trattamenti per FA e FECD
Design Therapeutics (NASDAQ:DSGN) informó avances en sus programas GeneTAC® y los resultados financieros del segundo trimestre de 2025. La compañía anunció datos farmacocinéticos preliminares alentadores para DT-216P2 en Ataxia de Friedreich (FA), mostrando una traducción favorable de primates no humanos a humanos mediante administración intravenosa y subcutánea.
Los desarrollos clave incluyen el continuo ensayo RESTORE-FA Fase 1/2 de DT-216P2 fuera de los EE. UU., mientras se aborda una suspensión clínica de la FDA. También iniciaron un ensayo de biomarcadores de fase 2 para DT-168 en pacientes con Distrofia Endotelial Corneal de Fuchs (FECD). Design reportó 216,3 millones de dólares en efectivo y valores, con una pérdida neta en el segundo trimestre de 19,1 millones de dólares.
- Traducción favorable de datos farmacocinéticos de primates no humanos a humanos para DT-216P2
- DT-216P2 mostró una mejor exposición y parámetros farmacocinéticos comparados con la formulación previa
- Fuerte posición de efectivo de 216,3 millones de dólares para apoyar el desarrollo de la cartera
- Avance de múltiples programas clínicos, incluyendo tratamientos para FA y FECD
Design Therapeutics (NASDAQ:DSGN)는 GeneTAC® 프로그램과 2025년 2분기 재무 실적에서 진전을 보고했습니다. 회사는 프리드라이히 운동실조증(FA)에서 DT-216P2의 초기 약동학 데이터가 비인간 영장류에서 인간으로 IV 및 피하 투여 모두에서 유리하게 전환됨을 발표했습니다.
주요 개발 사항으로는 미국 외부에서 진행 중인 RESTORE-FA 1/2상 임상시험과 FDA 임상 보류 통지에 대응하는 것이 포함됩니다. 또한, Fuchs 각막내피이영양증(FECD) 환자를 대상으로 DT-168 2상 바이오마커 시험을 시작했습니다. Design Therapeutics는 현금 및 증권 2억 1,630만 달러를 보유하고 있으며 2분기 순손실은 1,910만 달러였습니다.
- DT-216P2의 비인간 영장류에서 인간으로의 유리한 약동학 데이터 전환
- 이전 제형 대비 DT-216P2의 노출 및 PK 파라미터 개선
- 파이프라인 개발을 지원하는 2억 1,630만 달러의 강력한 현금 보유
- FA 및 FECD 치료제를 포함한 다수의 임상 프로그램 진전
Design Therapeutics (NASDAQ:DSGN) a annoncé des progrès dans ses programmes GeneTAC® ainsi que ses résultats financiers du deuxième trimestre 2025. La société a présenté des données pharmacocinétiques préliminaires encourageantes pour DT-216P2 dans l'ataxie de Friedreich (FA), montrant une bonne transposition des primates non humains aux humains par administration intraveineuse et sous-cutanée.
Les développements clés comprennent l'essai RESTORE-FA de phase 1/2 en cours pour DT-216P2 hors des États-Unis, tout en répondant à une mise en attente clinique de la FDA. La société a également lancé un essai de biomarqueurs de phase 2 pour DT-168 chez des patients atteints de dystrophie cornéenne endothéliale de Fuchs (FECD). Design a déclaré disposer de 216,3 millions de dollars en liquidités et titres, avec une perte nette de 19,1 millions de dollars au deuxième trimestre.
- Bonne transposition des données pharmacocinétiques des primates non humains aux humains pour DT-216P2
- DT-216P2 a montré une exposition et des paramètres PK améliorés par rapport à la formulation précédente
- Une solide trésorerie de 216,3 millions de dollars pour soutenir le développement du pipeline
- Avancement de plusieurs programmes cliniques, y compris des traitements pour la FA et la FECD
Design Therapeutics (NASDAQ:DSGN) meldete Fortschritte in seinen GeneTAC®-Programmen und die Finanzergebnisse für das zweite Quartal 2025. Das Unternehmen gab ermutigende frühe pharmakokinetische Daten für DT-216P2 bei Friedreich-Ataxie (FA) bekannt, die eine günstige Übertragung von nicht-menschlichen Primaten auf Menschen sowohl bei intravenöser als auch subkutaner Verabreichung zeigen.
Zu den wichtigsten Entwicklungen gehört die laufende RESTORE-FA Phase 1/2 Studie von DT-216P2 außerhalb der USA, während eine klinische Aussetzung der FDA adressiert wird. Außerdem startete das Unternehmen eine Phase-2-Biomarker-Studie für DT-168 bei Patienten mit Fuchs Endothelialer Hornhautdystrophie (FECD). Design meldete 216,3 Millionen US-Dollar in bar und Wertpapieren, mit einem Nettoverlust von 19,1 Millionen US-Dollar im zweiten Quartal.
- Günstige pharmakokinetische Datenübertragung von nicht-menschlichen Primaten auf Menschen für DT-216P2
- DT-216P2 zeigte verbesserte Exposition und PK-Parameter im Vergleich zur vorherigen Formulierung
- Starke Barreserve von 216,3 Millionen US-Dollar zur Unterstützung der Pipeline-Entwicklung
- Fortschritte in mehreren klinischen Programmen, einschließlich Behandlungen für FA und FECD
- None.
- FDA clinical hold on DT-216P2 IND application regarding US starting dose
- Increased net loss of $19.1 million in Q2 2025
Insights
Design Therapeutics shows promising PK data for Friedreich Ataxia drug and advances FECD program while maintaining strong cash position.
Design Therapeutics has shared encouraging early pharmacokinetic (PK) data for their lead Friedreich Ataxia (FA) candidate DT-216P2, demonstrating favorable translation from non-human primates to humans with both intravenous and subcutaneous administration routes. The new formulation shows improved exposure parameters compared to the previous version, including higher AUC and sustained plasma levels at comparable doses, which could enhance clinical efficacy.
While the FDA placed a clinical hold on the U.S. IND application for DT-216P2 regarding the starting dose, the company continues to dose patients in its RESTORE-FA Phase 1/2 multiple-ascending dose trial outside the U.S. This suggests confidence in the safety profile, further supported by their statement that the injection site thrombophlebitis issue seen with the prior formulation appears resolved.
The company has also initiated a Phase 2 biomarker trial for DT-168 in Fuchs Endothelial Corneal Dystrophy (FECD), a condition with no approved disease-modifying therapies. This represents a potential first-in-class opportunity in an underserved market.
With
The GeneTAC platform targeting transcriptional activation of genes silenced by repeat expansions continues to show promise across multiple indications, positioning Design as a potentially significant player in genetic medicine for previously untreatable conditions.
Early Human Pharmacokinetics Data for DT-216P2 Demonstrates Favorable Translation from Non-Human Primates (NHPs) to Humans and Improved DT-216 Product Profile for Friedreich Ataxia (FA) Program
RESTORE-FA Phase 1/2 Multiple-Ascending Dose Trial of DT-216P2 in FA Patients Underway
Phase 2 Biomarker Study for DT-168 Initiated in Patients with Fuchs Endothelial Corneal Dystrophy (FECD)
Cash and Securities of
CARLSBAD, Calif., Aug. 07, 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 financial results for the second quarter 2025.
“We’ve made meaningful progress across our pipeline this quarter,” said Pratik Shah, Ph.D., chairperson and chief executive officer of Design Therapeutics. “Early human PK data for DT-216P2 demonstrate the consistency of human plasma exposure profiles with NHP data across both IV and subcutaneous routes. We’re also pleased to have initiated our Phase 2 biomarker trial in patients with FECD, a disease with no approved disease-modifying therapies. Our preclinical programs also continue to advance as we work to deliver a new class of genomic medicines for patients with serious degenerative diseases.”
Corporate Highlights
- Friedreich Ataxia (FA)
- Today, Design announced early pharmacokinetics (PK) data for DT-216P2 demonstrating favorable translation from NHPs to humans with both intravenous (IV) and subcutaneous (SC) administration and an improved product profile compared to the prior DT-216 formulation (DT-216P1).
- Human plasma PK profiles of DT-216P2 were consistent with NHP data following both IV and SC single-dose administration.
- DT-216P2 exhibited improved exposure and PK parameters compared to DT-216P1, including higher AUC and sustained plasma levels at comparable doses.
- DT-216P2 has been generally well-tolerated, and based on clinical and non-clinical data, Design believes the injection site thrombophlebitis seen with DT-216P1 is no longer an issue limiting continued development of DT-216.
- In June, Design announced that it had received a clinical hold notice from the U.S. Food and Drug Administration (FDA) regarding its Investigational New Drug (IND) application for DT-216P2. FDA’s request pertains to the starting dose in the U.S., which the company plans to address with clinical data and, if needed, nonclinical data, in order to initiate studies for DT-216P2 in the U.S. Design continues to dose patients in its RESTORE-FA Phase 1/2 MAD trial of DT-216P2 outside the U.S.
- Today, Design announced early pharmacokinetics (PK) data for DT-216P2 demonstrating favorable translation from NHPs to humans with both intravenous (IV) and subcutaneous (SC) administration and an improved product profile compared to the prior DT-216 formulation (DT-216P1).
- Fuchs Endothelial Corneal Dystrophy (FECD): Design has initiated a Phase 2 biomarker trial of DT-168 to evaluate safety, tolerability and corneal endothelium biomarkers in FECD patients who are scheduled for corneal transplant surgery.
- Pipeline programs: Design continues to progress preclinical activities for its myotonic dystrophy type-1 (DM1) program toward the selection of a development candidate later in 2025. In Huntington’s disease, the company also continues to advance preclinical characterization of several candidate molecules.
Second Quarter 2025 Financial Results
- R&D Expenses: Research and development (R&D) expenses were
$15.7 million for the quarter ended June 30, 2025. - G&A Expenses: General and administrative (G&A) expenses were
$5.8 million for the quarter ended June 30, 2025. - Net Loss: Net loss was
$19.1 million for the quarter ended June 30, 2025. - Cash Position: Cash, cash equivalents and investment securities were
$216.3 million as of June 30, 2025.
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; Design’s pipeline; Design’s plan to address the FDA clinical hold with clinical data and, if needed, nonclinical data, in order to initiate studies for DT-216P2 in the U.S.; Design’s belief that the injection site thrombophlebitis seen with DT-216P1 is no longer an issue limiting continued development of DT-216; Design's ability to advance the GeneTAC® platform 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 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; the data we intend to provide the FDA to resolve the clinical hold may not be sufficient, which could further delay our ability to commence U.S. clinical trials of DT-216P2; the data we observe from earlier 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, 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 March 31, 2025, as filed with the SEC on May 7, 2025, and under the “Risk Factors” heading of Design’s Quarterly Report on Form 10-Q for the quarter ended June 30, 2025, 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 June 30, | Six Months Ended June 30, | |||||||||||||||
| 2025 | 2024 | 2025 | 2024 | |||||||||||||
| (unaudited) | ||||||||||||||||
| Operating expenses: | ||||||||||||||||
| Research and development | $ | 15,738 | $ | 10,516 | $ | 31,115 | $ | 20,317 | ||||||||
| General and administrative | 5,831 | 4,527 | 10,872 | 9,126 | ||||||||||||
| Total operating expenses | 21,569 | 15,043 | 41,987 | 29,443 | ||||||||||||
| Loss from operations | (21,569 | ) | (15,043 | ) | (41,987 | ) | (29,443 | ) | ||||||||
| Other income, net | 2,486 | 3,250 | 5,189 | 6,545 | ||||||||||||
| Net loss | $ | (19,083 | ) | $ | (11,793 | ) | $ | (36,798 | ) | $ | (22,898 | ) | ||||
| Net loss per share, basic and diluted | $ | (0.34 | ) | $ | (0.21 | ) | $ | (0.65 | ) | $ | (0.41 | ) | ||||
| Weighted-average shares of common stock outstanding, basic and diluted | 56,859,388 | 56,555,960 | 56,808,888 | 56,522,244 | ||||||||||||
| DESIGN THERAPEUTICS, INC. CONDENSED BALANCE SHEETS (in thousands) | ||||||||
| June 30, | December 31, | |||||||
| 2025 | 2024 | |||||||
| (unaudited) | ||||||||
| Assets | ||||||||
| Current assets: | ||||||||
| Cash, cash equivalents and investment securities | $ | 216,276 | $ | 245,477 | ||||
| Prepaid expenses and other current assets | 3,518 | 2,563 | ||||||
| Total current assets | 219,794 | 248,040 | ||||||
| Property and equipment, net | 1,258 | 1,410 | ||||||
| Right-of-use asset, related party | 1,834 | 2,216 | ||||||
| Other assets | — | 427 | ||||||
| Total assets | $ | 222,886 | $ | 252,093 | ||||
| Liabilities and Stockholders’ Equity | ||||||||
| Current liabilities: | ||||||||
| Accounts payable | $ | 1,013 | $ | 2,186 | ||||
| Accrued expenses and other current liabilities | 7,734 | 6,276 | ||||||
| Total current liabilities | 8,747 | 8,462 | ||||||
| Operating lease liability, net, related party | 1,102 | 1,534 | ||||||
| Total liabilities | 9,849 | 9,996 | ||||||
| Total stockholders’ equity | 213,037 | 242,097 | ||||||
| Total liabilities and stockholders’ equity | $ | 222,886 | $ | 252,093 | ||||
FAQ
What are the latest developments in Design Therapeutics' (DSGN) DT-216P2 program for Friedreich Ataxia?
How much cash does Design Therapeutics (DSGN) have as of Q2 2025?
What is the status of Design Therapeutics' (DSGN) FECD program?
What were Design Therapeutics' (DSGN) Q2 2025 financial results?
What pipeline programs is Design Therapeutics (DSGN) developing?
