Applied Therapeutics Announces Acceptance of Late-Breaking Abstract for Oral Presentation at the Peripheral Nerve Society 2025 Annual Meeting
Applied Therapeutics (NASDAQ: APLT) announced that full 12-month clinical data from the INSPIRE Phase 3 trial of govorestat (AT-007) has been accepted for a late-breaking oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting in Edinburgh, Scotland. The presentation will also include new topline 18-month and 24-month data from patients before their transition to the open-label extension study.
The INSPIRE Phase 3 trial was a double-blind, placebo-controlled registrational study evaluating govorestat's effect in patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease. The company previously released topline results from the 12-month group-level data analysis in February 2024.
Applied Therapeutics (NASDAQ: APLT) ha annunciato che i dati clinici completi a 12 mesi dello studio di fase 3 INSPIRE su govorestat (AT-007) sono stati accettati per una presentazione orale di rilievo durante il Peripheral Nerve Society (PNS) 2025 Annual Meeting a Edimburgo, Scozia. La presentazione includerà anche nuovi dati principali a 18 e 24 mesi provenienti dai pazienti prima della loro transizione allo studio di estensione in aperto.
Lo studio di fase 3 INSPIRE è stato uno studio registrativo in doppio cieco, controllato con placebo, che ha valutato l'effetto di govorestat in pazienti con Deficienza di Sorbitolo Deidrogenasi (SORD), una sottocategoria della malattia di Charcot-Marie-Tooth (CMT). L'azienda aveva già diffuso i risultati principali dell'analisi dei dati a 12 mesi a livello di gruppo nel febbraio 2024.
Applied Therapeutics (NASDAQ: APLT) anunció que los datos clínicos completos de 12 meses del ensayo de fase 3 INSPIRE de govorestat (AT-007) han sido aceptados para una presentación oral de última hora en la Reunión Anual 2025 de la Peripheral Nerve Society (PNS) en Edimburgo, Escocia. La presentación también incluirá nuevos datos principales a 18 y 24 meses de pacientes antes de su transición al estudio de extensión abierto.
El ensayo de fase 3 INSPIRE fue un estudio registracional doble ciego, controlado con placebo, que evaluó el efecto de govorestat en pacientes con Deficiencia de Sorbitol Deshidrogenasa (SORD), un subtipo de la enfermedad de Charcot-Marie-Tooth (CMT). La compañía ya había publicado los resultados principales del análisis de datos a nivel de grupo a 12 meses en febrero de 2024.
Applied Therapeutics (NASDAQ: APLT)는 govorestat (AT-007)의 INSPIRE 3상 임상시험 12개월 전체 데이터가 스코틀랜드 에든버러에서 열리는 Peripheral Nerve Society (PNS) 2025 연례회의에서 긴급 구두 발표로 채택되었다고 발표했습니다. 발표에는 오픈라벨 연장 연구로 전환하기 전 환자들의 18개월 및 24개월 신규 주요 데이터도 포함될 예정입니다.
INSPIRE 3상 시험은 이중맹검 위약 대조 등록 연구로, 샤르코-마리-투스(CMT) 질환의 하위 유형인 소르비톨 탈수소효소(SORD) 결핍증 환자에서 govorestat의 효과를 평가했습니다. 회사는 2024년 2월에 12개월 그룹 수준 데이터 분석의 주요 결과를 이미 발표한 바 있습니다.
Applied Therapeutics (NASDAQ : APLT) a annoncé que les données cliniques complètes sur 12 mois de l'essai de phase 3 INSPIRE concernant govorestat (AT-007) ont été acceptées pour une présentation orale de dernière minute lors de la réunion annuelle 2025 de la Peripheral Nerve Society (PNS) à Édimbourg, en Écosse. La présentation inclura également de nouvelles données principales à 18 et 24 mois issues des patients avant leur passage à l'étude d'extension en ouvert.
L'essai de phase 3 INSPIRE était une étude d'enregistrement en double aveugle, contrôlée par placebo, évaluant l'effet de govorestat chez des patients atteints de déficit en sorbitol déshydrogénase (SORD), un sous-type de la maladie de Charcot-Marie-Tooth (CMT). La société avait déjà publié les résultats principaux de l'analyse des données à 12 mois au niveau du groupe en février 2024.
Applied Therapeutics (NASDAQ: APLT) gab bekannt, dass die vollständigen 12-Monats-Klinikdaten der INSPIRE Phase-3-Studie zu govorestat (AT-007) für eine kurzfristige mündliche Präsentation auf der Peripheral Nerve Society (PNS) 2025 Jahrestagung in Edinburgh, Schottland, angenommen wurden. Die Präsentation wird auch neue Topline-Daten von 18 und 24 Monaten von Patienten vor ihrem Übergang zur Open-Label-Verlängerungsstudie enthalten.
Die INSPIRE Phase-3-Studie war eine doppelblinde, placebokontrollierte Zulassungsstudie, die die Wirkung von govorestat bei Patienten mit Sorbitol-Dehydrogenase (SORD)-Mangel, einem Subtyp der Charcot-Marie-Tooth (CMT)-Krankheit, untersuchte. Das Unternehmen hatte bereits im Februar 2024 die Topline-Ergebnisse der 12-Monats-Gruppendatenanalyse veröffentlicht.
- None.
- None.
Late-breaking oral presentation to include full 12-month clinical data from INSPIRE registrational Phase 3 trial in addition to topline 18-month and 24-month data from patients prior to open-label extension
NEW YORK, May 09, 2025 (GLOBE NEWSWIRE) -- Applied Therapeutics, Inc. (Nasdaq: APLT) (the “Company”), a clinical-stage biopharmaceutical company dedicated to creating transformative treatments for rare diseases, today announced that it will present full 12-month clinical results from the INSPIRE Phase 3 trial of govorestat (AT-007) in a late-breaking abstract selected for oral presentation at the Peripheral Nerve Society (PNS) 2025 Annual Meeting, being held May 17-20, 2025 in Edinburgh, Scotland. The presentation will also include new topline 18-month and 24-month data prior to the transition to the open-label extension study. The INSPIRE Phase 3 trial was a double-blind, placebo-controlled registrational trial to evaluate the effect of govorestat in patients with Sorbitol Dehydrogenase (SORD) Deficiency, a subtype of Charcot-Marie-Tooth (CMT) disease. The Company previously shared topline results from an analysis of 12-month group-level data from the INSPIRE trial in February 2024.
Presentation Details
Title: INSPIRE CMT-SORD Clinical Trial: Month 18 and Month 24 INSPIRE Trial Results and Designing the Next Randomized Study
Oral Presentation Number: 597
Presenter: Evan Bailey, MD, Senior Vice President, Clinical Development, Applied Therapeutics Inc.
Date and Time: Sunday, May 18, 2025, 12:25 - 12:40 p.m. BST
About Govorestat (AT-007)
Govorestat is a central nervous system (CNS) penetrant Aldose Reductase Inhibitor (ARI) being developed for the treatment of multiple rare diseases including Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD), Classic Galactosemia, and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
Govorestat has received Orphan Medicinal Product Designation from the European Medicines Agency (EMA) for both Galactosemia and CMT disease. Govorestat has also received Orphan Drug Designation from the U.S. Food and Drug Administration (FDA) for the treatment of Galactosemia, phosphomannomutase 2 deficiency, and SORD Deficiency; Rare Pediatric Disease designation for Galactosemia and PMM2-CDG; and Fast Track designation for Galactosemia.
About Applied Therapeutics
Applied Therapeutics is a clinical-stage biopharmaceutical company committed to the development of novel drug candidates against validated molecular targets in rare diseases. The Company’s lead drug candidate, govorestat, is a novel central nervous system penetrant Aldose Reductase Inhibitor (ARI) for the treatment of CNS rare metabolic diseases, including Classic Galactosemia, Charcot-Marie-Tooth Sorbitol Dehydrogenase Deficiency (CMT-SORD) and phosphomannomutase 2 congenital disorder of glycosylation (PMM2-CDG).
To learn more, please visit www.appliedtherapeutics.com and follow the company on X at @Applied_Tx.
Forward-Looking Statements
This press release contains “forward-looking statements” that involve substantial risks and uncertainties for purposes of the safe harbor provided by the Private Securities Litigation Reform Act of 1995. Any statements, other than statements of historical fact, included in this press release regarding the strategy, future operations, prospects, plans and objectives of management, including words such as “may,” “will,” “expect,” “anticipate,” “plan,” “intend,” “predicts” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are forward-looking statements. These include, without limitation, expressed or implied statements regarding the (i) continued development and advancement of AT-007 and (ii) potential of AT-007 for the treatment of multiple rare diseases, including CMT-SORD, Classic Galactosemia and CDG. Forward-looking statements in this release involve substantial risks and uncertainties that could cause actual results to differ materially from those expressed or implied by the forward-looking statements, and we, therefore cannot assure you that our plans, intentions, expectations or strategies will be attained or achieved.
Such risks and uncertainties include, without limitation, (i) our plans to develop, market and commercialize our product candidates, (ii) the initiation, timing, progress and results of our current and future preclinical studies and clinical trials and our research and development programs, (iii) our ability to take advantage of expedited regulatory pathways for any of our product candidates, (iv) our estimates regarding expenses, future revenue, capital requirements and needs for additional financing, (v) our ability to successfully acquire or license additional product candidates on reasonable terms and advance product candidates into, and successfully complete, clinical studies, (vi) our ability to maintain and establish collaborations or obtain additional funding, (vii) our ability to obtain and timing of regulatory approval of our current and future product candidates, (viii) the anticipated indications for our product candidates, if approved, (ix) our expectations regarding the potential market size and the rate and degree of market acceptance of such product candidates, (x) our ability to fund our working capital requirements and expectations regarding the sufficiency of our capital resources, (xi) the implementation of our business model and strategic plans for our business and product candidates, (xii) our intellectual property position and the duration of our patent rights, (xiii) developments or disputes concerning our intellectual property or other proprietary rights, (xiv) our expectations regarding government and third-party payor coverage and reimbursement, (xv) our ability to compete in the markets we serve, (xvi) the impact of government laws and regulations and liabilities thereunder, (xvii) developments relating to our competitors and our industry, (xviii) our ability to achieve the anticipated benefits from the agreements entered into in connection with our partnership with Advanz Pharma and (xix) other factors that may impact our financial results. In light of the significant uncertainties in these forward-looking statements, you should not rely upon forward-looking statements as predictions of future events. Although we believe that we have a reasonable basis for each forward-looking statement contained in this press release, we cannot guarantee that the future results, levels of activity, performance or events and circumstances reflected in the forward-looking statements will be achieved or occur at all. Factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in our filings with the U.S. Securities and Exchange Commission, including those described in the “Risk Factors” section contained therein. Except as otherwise required by law, we disclaim any intention or obligation to update or revise any forward-looking statements, which speak only as of the date they were made, whether as a result of new information, future events or circumstances or otherwise.
Contacts
Investors:
Julie Seidel / Andrew Vulis
(212) 600-1902 or
appliedtherapeutics@argotpartners.com
Media:
media@appliedtherapeutics.com
FAQ
What will Applied Therapeutics (APLT) present at the PNS 2025 Annual Meeting?
What is the INSPIRE Phase 3 trial studying for APLT stock?
When and where will APLT present their INSPIRE trial results in 2025?
What is govorestat (AT-007) being developed for by Applied Therapeutics?
