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Passage Bio Presents Preclinical and Interim Clinical Data for PBFT02 in FTD-GRN at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Conference

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Passage Bio (NASDAQ: PASG) presented preclinical and interim clinical data for PBFT02, their gene therapy treatment for frontotemporal dementia (FTD-GRN), at the ESGCT 31st Annual Conference. Key findings showed that AAV1 vector achieved superior human progranulin levels compared to other vectors, and PBFT02 improved lysosomal histopathology and reduced neuroinflammation in test subjects.

The interim clinical data from the upliFT-D trial demonstrated that PBFT02 was well-tolerated and produced consistent, durable increases in CSF progranulin levels, maintained up to 12 months post-administration. The therapy showed promising results in achieving widespread vector distribution throughout the nervous system.

Passage Bio (NASDAQ: PASG) ha presentato dati preclinici e dati clinici intermedi per PBFT02, il loro trattamento con terapia genica per la demenza frontotemporale (FTD-GRN), durante la 31ª Conferenza Annuale ESGCT. Le principali scoperte hanno mostrato che il vettore AAV1 ha raggiunto livelli di progranulina umana superiori rispetto ad altri vettori, e PBFT02 ha migliorato l'istopatologia lisosomiale e ridotto la neuroinfiammazione nei soggetti testati.

I dati clinici intermedi provenienti dallo studio upliFT-D hanno dimostrato che PBFT02 è stato ben tollerato e ha prodotto aumenti costanti e duraturi nei livelli di progranulina nel liquido cerebrospinale, mantenuti fino a 12 mesi dopo la somministrazione. La terapia ha mostrato risultati promettenti nel raggiungere una distribuzione diffusa del vettore in tutto il sistema nervoso.

Passage Bio (NASDAQ: PASG) presentó datos preclínicos y datos clínicos intermedios para PBFT02, su tratamiento de terapia génica para la demencia frontotemporal (FTD-GRN), en la 31ª Conferencia Anual de ESGCT. Los hallazgos clave mostraron que el vector AAV1 logró niveles superiores de progranulina humana en comparación con otros vectores, y PBFT02 mejoró la histopatología lisosómica y redujo la neuroinflamación en los sujetos de prueba.

Los datos clínicos intermedios del ensayo upliFT-D demostraron que PBFT02 fue bien tolerado y produjo aumentos consistentes y duraderos en los niveles de progranulina en el líquido cefalorraquídeo, mantenidos hasta 12 meses después de la administración. La terapia mostró resultados prometedores en lograr una distribución amplia del vector a lo largo del sistema nervioso.

Passage Bio (NASDAQ: PASG)는 ESGCT 제31회 연례 회의에서 전두측두엽 치매(FTD-GRN)에 대한 유전자 치료제 PBFT02의 전임상 및 중간 임상 데이터를 발표했습니다. 주요 발견 사항에 따르면 AAV1 벡터는 다른 벡터에 비해 인간 프로그란울린 수준을 더 높게 달성하였으며, PBFT02는 테스트 대상의 리소좀 조직병리학을 개선하고 신경 염증을 감소시켰습니다.

upliFT-D 시험의 중간 임상 데이터는 PBFT02가 잘 견디는 것으로 나타났으며, CSF 프로그란울린 수준에서 일관되고 지속적인 증가를 이끌어냈고, 이는 투여 후 최대 12개월 동안 유지되었습니다. 이 치료법은 신경계 전체에 걸쳐 벡터 분포를 널리 달성하는 데 있어 유망한 결과를 보였습니다.

Passage Bio (NASDAQ: PASG) a présenté des données précliniques et des données cliniques intermédiaires pour PBFT02, leur traitement de thérapie génique pour la démence frontotemporale (FTD-GRN), lors de la 31e Conférence Annuelle de l'ESGCT. Les principales conclusions ont montré que le vecteur AAV1 atteignait des niveaux de progranuline humaine supérieurs à ceux d'autres vecteurs, et que le PBFT02 améliorait l'histopathologie lysosomale et réduisait la neuroinflammation chez les sujets testés.

Les données cliniques intermédiaires de l'essai upliFT-D ont démontré que le PBFT02 avait été bien toléré et produisait des augmentations cohérentes et durables des niveaux de progranuline dans le liquide céphalorachidien, maintenues jusqu'à 12 mois après l'administration. La thérapie a montré des résultats prometteurs pour atteindre une distribution généralisée du vecteur dans l'ensemble du système nerveux.

Passage Bio (NASDAQ: PASG) hat auf der 31. Jahreskonferenz der ESGCT präklinische und interimistische klinische Daten für PBFT02, ihre Gentherapie für die frontotemporale Demenz (FTD-GRN), vorgestellt. Wesentliche Ergebnisse zeigten, dass der AAV1-Vektor im Vergleich zu anderen Vektoren höhere menschliche Progranulinwerte erreichte und PBFT02 die lysosomale Histopathologie verbesserte sowie die Neuroinflammation bei den Testpersonen reduzierte.

Die interimistischen klinischen Daten aus der upliFT-D-Studie zeigten, dass PBFT02 gut vertragen wurde und konsistente, langanhaltende Erhöhungen der Progranulinwerte im Liquor cerebrospinalis hervorrief, die bis zu 12 Monate nach der Verabreichung aufrechterhalten wurden. Die Therapie zeigte vielversprechende Ergebnisse bei der Erreichung einer weitreichenden Vektordistribution im gesamten Nervensystem.

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Preclinical data demonstrated that an AAV1 vector achieved superior human progranulin levels in the CSF as compared to AAV5 and AAVhu68 (an AAV9 variant)

Nonclinical data showed PBFT02 improved lysosomal histopathology and reduced neuroinflammation in Grn knockout mice, and achieved widespread vector distribution throughout the nervous system in non-human primates

Company delivered data during an oral presentation on Thursday, October 24 at ESGCT

PHILADELPHIA, Oct. 24, 2024 (GLOBE NEWSWIRE) -- Passage Bio, Inc. (Nasdaq: PASG), a clinical stage genetic medicines company focused on improving the lives of patients with neurodegenerative diseases, today announced the company delivered preclinical and interim clinical data as part of an oral presentation at the European Society of Gene & Cell Therapy (ESGCT) 31st Annual Congress being held October 22-25, 2024, in Rome, Italy.

Details of the oral presentation are below:

Abstract Title:Non-clinical and early clinical development of PBFT02, an AAV gene therapy for frontotemporal dementia with GRN mutations (FTD-GRN)
Session:7d: CNS gene therapy
Date & Time:Thursday, October 24 from 9:00 a.m.-11:00 a.m. CEST (3:00 a.m.-5:00 a.m. ET)
Presenter:Sue Browne, Ph.D., Chief Scientific Officer


Today's oral presentation at ESGCT outlined the robust preclinical data generated in the development of PBFT02, including studies that informed vector and dose selection and demonstrated the positive effects of elevating progranulin levels in vivo. Additionally, the presentation highlighted interim safety and biomarker data from the upliFT-D clinical trial, which validate the preclinical findings and position PBFT02 as a potential best-in-class progranulin-raising therapy.

“We are excited to share a detailed overview of the preclinical studies that support our PBFT02 program and informed our clinical development strategy,” said Will Chou, M.D., president and chief executive officer of Passage Bio. “These strong preclinical results gave us confidence in choosing the AAV1 vector and ICM administration as a differentiated approach to administer our gene therapy to patients in our ongoing upliFT-D clinical study. It’s also encouraging to see these preclinical findings translate into the clinic, with interim PBFT02 data demonstrating a well-tolerated safety profile and consistent, durable increases in CSF progranulin levels. We remain committed to advancing PBFT02 in FTD and look forward to exploring its therapeutic potential in additional neurodegenerative diseases that could benefit from elevated progranulin levels.”

Data Summary

  • Capsid comparison study in non-human primates (NHPs) showed the AAV1 vector achieved superior human PGRN levels in the CSF as compared to AAV5 and AAVhu68 (an AAV9 variant) following intra-cisterna magna (ICM) administration
  • Dose escalation study in Grn knockout mice showed PBFT02 improved lysosomal histopathology and reduced neuroinflammation throughout the brain following intra-CSF delivery, including evidence suggesting that higher levels of PGRN may provide additional benefits
  • NHP biodistribution study showed ICM administration of PBFT02 achieved high levels of gene distribution throughout the nervous system, including vector delivery to the cortical and sub-cortical brain regions affected in FTD, and to the spinal cord
  • PBFT02 was well tolerated in NHPs, and ICM administration resulted in dose-dependent PGRN elevations in NHP CSF
  • Interim safety and biomarker data from the upliFT-D clinical study demonstrated that Dose 1 of PBFT02 was generally well-tolerated after ICM administration and led to consistent, durable increases in levels of CSF progranulin in all treated Cohort 1 patients, with elevated CSF progranulin levels sustained up to 12 months post-administration

Additional details on the meeting can be found at the ESGCT 31st Annual Congress website, and a copy of the oral presentation deck will be available on the Investor Events and Presentations page of the Passage Bio corporate website.

About PBFT02

PBFT02 utilizes an AAV1 viral vector to deliver, through ICM administration, a functional GRN gene that encodes for PGRN. This vector construct and delivery approach aim to elevate PGRN levels in the central nervous system to alter the course of neurodegenerative diseases. Interim clinical data from the upliFT-D Phase 1/2 study in FTD-GRN participants shows that ICM administration of PBFT02 resulted in robust PGRN elevations in the CSF.

The potential clinical benefit of PBFT02 is supported by extensive preclinical studies. In non-human primates, a single ICM administration of PBFT02 led to broad vector distribution throughout the CNS, and robust, dose-dependent elevations in PGRN levels in CSF. An NHP study also demonstrated that AAV1 was particularly proficient at transducing ependymal cells. In a murine FTD model, PBFT02 administration improved lysosomal function and reduced neuroinflammation.

About Passage Bio

Passage Bio (Nasdaq: PASG) is a clinical stage genetic medicines company on a mission to improve the lives of patients with neurodegenerative diseases. Our primary focus is the development and advancement of cutting-edge, one-time therapies designed to target the underlying pathology of these conditions. Passage Bio’s lead product candidate, PBFT02, seeks to treat neurodegenerative conditions, including frontotemporal dementia, by elevating progranulin levels to restore lysosomal function and slow disease progression.

To learn more about Passage Bio and our steadfast commitment to protecting patients and families against loss in neurodegenerative conditions, please visit: www.passagebio.com.

Forward-Looking Statements

This press release contains “forward-looking statements” within the meaning of, and made pursuant to the safe harbor provisions of, the Private Securities Litigation Reform Act of 1995, including, but not limited to: our expectations about timing and execution of anticipated milestones, including the progress of clinical studies and the availability of clinical data from such trials; our expectations about our collaborators’ and partners’ ability to execute key initiatives; and the ability of our product candidates to treat their respective target CNS disorders. These forward-looking statements may be accompanied by such words as “aim,” “anticipate,” “believe,” “could,” “estimate,” “expect,” “forecast,” “goal,” “intend,” “may,” “might,” “plan,” “potential,” “possible,” “will,” “would,” and other words and terms of similar meaning. These statements involve risks and uncertainties that could cause actual results to differ materially from those reflected in such statements, including: our ability to develop and obtain regulatory approval for our product candidates; the timing and results of preclinical studies and clinical trials; risks associated with clinical trials, including our ability to adequately manage clinical activities, unexpected concerns that may arise from additional data or analysis obtained during clinical trials, regulatory authorities may require additional information or further studies, or may fail to approve or may delay approval of our drug candidates; the occurrence of adverse safety events; the risk that positive results in a preclinical study or clinical trial may not be replicated in subsequent trials or success in early stage clinical trials may not be predictive of results in later stage clinical trials; failure to protect and enforce our intellectual property, and other proprietary rights; our dependence on collaborators and other third parties for the development and manufacture of product candidates and other aspects of our business, which are outside of our full control; risks associated with current and potential delays, work stoppages, or supply chain disruptions; and the other risks and uncertainties that are described in the Risk Factors section in documents the company files from time to time with the Securities and Exchange Commission (SEC), and other reports as filed with the SEC. Passage Bio undertakes no obligation to publicly update any forward-looking statement, whether written or oral, that may be made from time to time, whether as a result of new information, future developments or otherwise.

For further information, please contact:

Investors:
Stuart Henderson
Passage Bio
267.866.0114
shenderson@passagebio.com

Media:
Mike Beyer
Sam Brown Inc. Healthcare Communications
312.961.2502
MikeBeyer@sambrown.com


FAQ

What were the key results from Passage Bio's PBFT02 clinical trial presented at ESGCT 2024?

The upliFT-D clinical trial showed PBFT02 was well-tolerated after ICM administration and produced consistent, durable increases in CSF progranulin levels in all Cohort 1 patients, with elevated levels sustained up to 12 months post-administration.

How did PBFT02's AAV1 vector perform compared to other vectors in the preclinical studies?

The AAV1 vector achieved superior human progranulin levels in the CSF compared to AAV5 and AAVhu68 (an AAV9 variant) following intra-cisterna magna administration.

What therapeutic benefits did PBFT02 show in preclinical studies for FTD-GRN treatment?

In Grn knockout mice, PBFT02 improved lysosomal histopathology and reduced neuroinflammation throughout the brain following intra-CSF delivery, with evidence suggesting higher levels of progranulin may provide additional benefits.

When did Passage Bio (PASG) present their PBFT02 data at the ESGCT conference?

Passage Bio presented their PBFT02 data during an oral presentation on Thursday, October 24, 2024, at the ESGCT 31st Annual Congress in Rome, Italy.

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