Aileron Therapeutics to Present Previously Announced Data from the Phase 1b Clinical Trial Evaluating Low-Dose LTI-03 in Idiopathic Pulmonary Fibrosis (IPF) at the 22nd International Colloquium on Lung and Airway Fibrosis
Aileron Therapeutics (NASDAQ: ALRN) is presenting data from its Phase 1b clinical trial of LTI-03 for Idiopathic Pulmonary Fibrosis (IPF) at the 22nd International Colloquium on Lung and Airway Fibrosis. The trial's Cohort 1, evaluating low-dose LTI-03 (2.5 mg BID), showed positive trends in seven out of eight biomarkers, with three showing statistically significant improvements in collagen synthesis, inflammation, and fibrogenesis.
The company has also completed enrollment for Cohort 2, testing high-dose LTI-03 (5 mg BID), with topline data expected soon. Pre-clinical data using precision cut lung slices (PCLS) further supports LTI-03's potential effectiveness in IPF treatment. The ongoing Phase 1b trial aims to evaluate the safety and tolerability of LTI-03 in IPF patients over 14 days of treatment.
Aileron Therapeutics (NASDAQ: ALRN) sta presentando dati dal suo trial clinico di Fase 1b su LTI-03 per Fibrosi Polmonare Idiopatica (IPF) al 22° Colloquio Internazionale sulla Fibrosi Polmonare e delle Vie Aeree. Il Cohort 1 della sperimentazione, che valuta LTI-03 a bassa dose (2,5 mg BID), ha mostrato tendenze positive in sette su otto biomarcatori, con tre che presentano miglioramenti statisticamente significativi nella sintesi del collagene, nell'infiammazione e nella fibrogenesi.
L'azienda ha inoltre completato l'arruolamento per il Cohort 2, che testerebbe LTI-03 ad alta dose (5 mg BID), con i dati preliminari attesi a breve. I dati preclinici utilizzando sezioni polmonari tagliate con precisione (PCLS) supportano ulteriormente l'efficacia potenziale di LTI-03 nel trattamento dell'IPF. L'attuale trial di Fase 1b mira a valutare la sicurezza e la tollerabilità di LTI-03 nei pazienti con IPF per un periodo di trattamento di 14 giorni.
Aileron Therapeutics (NASDAQ: ALRN) está presentando datos de su ensayo clínico de Fase 1b sobre LTI-03 para Fibrosis Pulmonar Idiopática (IPF) en el 22º Coloquio Internacional sobre Fibrosis Pulmonar y de Vías Respiratorias. El Cohorte 1 del ensayo, que evalúa LTI-03 en dosis bajas (2,5 mg BID), mostró tendencias positivas en siete de ocho biomarcadores, con tres que presentaron mejoras estadísticamente significativas en la síntesis de colágeno, inflamación y fibrogénesis.
La empresa también ha completado la inscripción para el Cohorte 2, que prueba LTI-03 en dosis altas (5 mg BID), con datos preliminares que se esperan pronto. Los datos preclínicos utilizando cortes de pulmón de precisión (PCLS) respaldan aún más la potencial efectividad de LTI-03 en el tratamiento de la IPF. El ensayo de Fase 1b en curso tiene como objetivo evaluar la seguridad y tolerabilidad de LTI-03 en pacientes con IPF durante un tratamiento de 14 días.
아이레론 테라퓨틱스(Aileron Therapeutics, NASDAQ: ALRN)는 제22회 국제 폐 및 기도 섬유증 심포지엄에서 LTI-03에 대한 특발성 폐섬유증(IPF)의 1b상 임상시험 데이터를 발표하고 있습니다. 저용량 LTI-03(2.5mg BID)을 평가한 1군의 시험 결과는 8개의 바이오마커 중 7개에서 긍정적인 추세를 보여주었으며, 그 중 3개는 콜라겐 합성, 염증 및 섬유세포 생성에서 통계적으로 유의미한 개선을 보였습니다.
회사는 또한 고용량 LTI-03(5mg BID)을 시험하는 2군의 등록을 완료했으며, 곧 전체 데이터를 발표할 예정입니다. 정밀 절단 폐 슬라이스(PCLS)를 사용한 전임상 데이터는 IPF 치료에서 LTI-03의 잠재적 효과를 더욱 지지합니다. 현재 진행 중인 1b상 시험은 IPF 환자에 대한 14일 치료 기간 동안 LTI-03의 안전성과 내약성을 평가하는 것을 목표로 하고 있습니다.
Aileron Therapeutics (NASDAQ: ALRN) présente des données de son essai clinique de Phase 1b de LTI-03 pour la Fibrose Pulmonaire Idiopathique (IPF) lors du 22ème Colloque International sur la Fibrose Pulmonaire et des Voies Respiratoires. Le Cohorte 1 de l'essai, évaluant une faible dose de LTI-03 (2,5 mg BID), a montré des tendances positives dans sept des huit biomarqueurs, trois montrant des améliorations statistiquement significatives dans la synthèse de collagène, l'inflammation et la fibrogénèse.
L'entreprise a également terminé l'inscription pour le Cohorte 2, testant une dose élevée de LTI-03 (5 mg BID), avec des données préliminaires attendues prochainement. Les données précliniques utilisant des coupes pulmonaires de précision (PCLS) soutiennent davantage l'efficacité potentielle de LTI-03 dans le traitement de l'IPF. L'essai de Phase 1b en cours vise à évaluer la sécurité et la tolérabilité de LTI-03 chez les patients atteints d'IPF sur une durée de traitement de 14 jours.
Aileron Therapeutics (NASDAQ: ALRN) präsentiert Daten aus seiner klinischen Phase-1b-Studie zu LTI-03 für idiopathische Lungenfibrose (IPF) auf dem 22. Internationalen Kolloquium über Lungen- und Atemwegfibrose. Die Kohorte 1 der Studie, die die niedrige Dosis von LTI-03 (2,5 mg BID) bewertet, zeigte positive Tendenzen in sieben von acht Biomarkern, wobei drei statistisch signifikante Verbesserungen in der Kollagensynthese, Entzündung und Fibrogenese aufwiesen.
Das Unternehmen hat zudem die Rekrutierung für die Kohorte 2, die die Hochdosis von LTI-03 (5 mg BID) testet, abgeschlossen, wobei bald erste Ergebnisse erwartet werden. Präklinische Daten zur Verwendung von präzise geschnittenen Lungenschnitten (PCLS) unterstützen weiterhin die potenzielle Wirksamkeit von LTI-03 in der Behandlung von IPF. Die laufende Phase-1b-Studie zielt darauf ab, die Sicherheit und Verträglichkeit von LTI-03 bei IPF-Patienten über einen Zeitraum von 14 Tagen zu bewerten.
- Positive trends observed in 7 out of 8 biomarkers in Phase 1b trial Cohort 1
- Statistically significant improvements in 3 biomarkers: collagen synthesis, inflammation, and fibrogenesis
- Completed enrollment for Cohort 2 (high-dose LTI-03) with topline data expected soon
- Pre-clinical data supports LTI-03's potential effectiveness in IPF treatment
- None.
Insights
The presentation of Phase 1b data for LTI-03 in IPF at ICLAF is a significant milestone for Aileron Therapeutics. The positive trends observed in 7 out of 8 biomarkers, with 3 showing statistical significance, suggest potential efficacy in addressing key aspects of IPF pathology:
- Reduced expression of profibrotic proteins
- Improvements in collagen synthesis, inflammation and fibrogenesis
These results, combined with the pre-clinical data from precision cut lung slices, provide a strong foundation for LTI-03's mechanism of action. The completion of enrollment for Cohort 2 (high-dose) is also noteworthy, as it will provide important dose-response data.
However, investors should note that this is still early-stage data. The small sample size (12 patients) and short treatment duration (14 days) limit the conclusions that can be drawn. The upcoming topline data from Cohort 2 will be critical in determining the drug's potential and informing future development plans.
For a
This news has positive implications for Aileron Therapeutics' financial outlook and potential market position:
- Positive early-stage data in IPF, a market projected to reach
$5.9 billion by 2027, could attract investor interest and potential partnerships. - The novel mechanism of action (caveolin-1 scaffolding domain peptide) differentiates LTI-03 from current IPF treatments, potentially addressing unmet needs in this space.
- Completion of Cohort 2 enrollment suggests efficient clinical trial execution, which is important for cash runway management in small-cap biotech companies.
However, investors should consider:
- Aileron's
$74 million market cap reflects early-stage risk; significant value inflection points likely lie ahead with more advanced clinical data. - Cash position and burn rate will be critical metrics to watch, as further clinical development will require substantial funding.
- The path to market in IPF is long and costly, often requiring large Phase 3 trials.
While promising, this news represents an early step in a lengthy and capital-intensive drug development process.
First scientific presentation of previously announced data from Cohort 1 of the ongoing Phase 1b clinical trial evaluating low-dose LTI-03 (2.5 mg BID) in IPF, affirms positive trends in seven of the eight biomarkers evaluated, suggesting potential therapeutic effect
Recently completed enrollment of Cohort 2 evaluating high-dose LTI-03 (5 mg BID) in mid-September; topline data expected in the near-term
The Company previously announced positive data from Cohort 1 of the ongoing Phase 1b clinical trial evaluating low-dose LTI-03 (2.5 mg BID) in patients with IPF. Following inhaled administration of low-dose LTI-03 in 12 patients over the course of 14 days, a positive trend was observed in seven out of eight biomarkers with evidence of reduced expression among multiple profibrotic proteins produced by basal-like cells and fibroblasts that contribute to the progression of IPF, including data from three biomarkers (collagen synthesis, inflammation, and fibrogenesis) that was statistically significant, reinforcing the potential of LTI-03 to improve lung function and reverse the course of IPF. The [poster][abstracts] being presented at ICLAF will summarize the previously disclosed data from Cohort 1.
Pre-clinical data presented at ICLAF further supports the potential therapeutic effectiveness of LTI-03 for IPF through precision cut lung slices (PCLS) performed ex-vivo. Pre-clinical studies demonstrated molecular activity in IPF PCLS explants indicative of fibrosis during five days in culture and LTI-03 broadly attenuated pro-fibrotic proteins and pathways.
Additionally, the Company recently announced completion of enrollment in Cohort 2 of the ongoing Phase 1b clinical trial evaluating high-dose LTI-03 (5 mg BID) in 12 patients with IPF. In the trial, eligible patients (n=24) are randomly assigned (3:1) to receive either inhaled LTI-03 or placebo. The primary objective of the trial is to evaluate the safety and tolerability of LTI-03 in patients with IPF after treatment for 14 consecutive days, with measurement of multiple protein biomarkers as exploratory endpoints. The Company expects to report topline data for this cohort in the near-term.
Details of the [poster] presentations are as follows:
Presentation Title: Anti-Fibrotic Activity of Caveolin-1 scaffolding domain Peptide LTI-03 in Ex Vivo Precision Cut Lung Slices from Patients with Idiopathic Pulmonary Fibrosis
Abstract #: 0186
Presenter: Professor Cory M. Hogaboam, Cedars Sinai Medical Center,
Date & Time: Tuesday, October 15, 2024 at 3:30 pm EEST/ 8:30 am ET
Presentation Title: Inhalation of LTI-03 Modulates Multiple Targets in a Phase 1B Placebo Controlled Clinical Trial for IPF
Abstract #: 0183
Presenter: Professor Cory M. Hogaboam, Cedars Sinai Medical Center,
Date & Time: Tuesday, October 15, 2024 at 3:30 pm EEST/ 8:30 am ET
About the Phase 1 Clinical Trial of LTI-03
The Phase 1b clinical trial of LTI-03 is a randomized, double-blind, placebo controlled, multi-center, dose escalation trial in patients recently diagnosed with IPF that have not received prior treatment with anti-fibrotic agents for at least two months (NCT05954988). Eligible patients are randomly assigned (3:1) to receive one of two doses of inhaled LTI-03 or placebo. The primary objective of the trial is to investigate the safety and tolerability of LTI-03 in patients with IPF after treatment for 14 consecutive days, with measurement of multiple protein biomarkers as exploratory endpoints.
About IPF
IPF is a chronic lung disease characterized by progressive tissue scarring that prevents proper lung function. It is a progressive, fatal, age-associated lung disease affecting approximately 100,000 people in the
About LTI-03 and Caveolin-1 (Cav1)
LTI-03 is a seven amino acid peptide, the sequence of which is derived from the caveolin scaffolding domain (CSD), an important binding region of the Cav1 protein. Cav1 normally serves a critical function in the prevention of fibrosis by maintaining a balance between pathways that both initiate and arrest lung repair and cell movement. Through the CSD, caveolin interacts with a large number of signaling molecules, all of which possess a caveolin binding domain region. Cav1 expression is decreased in IPF lung tissues and has been demonstrated to decrease during the fibrotic phase of bleomycin lung injury in mice. Restoring the balance of important biological signals in the lung may not only slow lung function decline but could also restore healthy lung function through the protection of healthy epithelial cells.
About Aileron Therapeutics
Aileron Therapeutics is a biopharmaceutical company advancing a novel pipeline of first-in-class medicines to address significant unmet medical needs in orphan pulmonary and fibrosis indications. Aileron's lead product candidate, LTI-03, is a novel, synthetic peptide with a dual mechanism targeting alveolar epithelial cell survival as well as inhibition of profibrotic signaling. Currently, LTI-03 is being evaluated in a Phase 1b clinical trial for the treatment of idiopathic pulmonary fibrosis. Aileron's second product candidate, LTI-01, is a proenzyme that has completed Phase 1b and Phase 2a clinical trials for the treatment of loculated pleural effusions. LTI-01 has received Orphan Drug Designation in the US and EU and Fast Track Designation in the US.
References
1 Pergolizzi, Jr., J., LeQuang, J., Varrassi, M., Breve, F., Magnusson, P., Varrassi, G., (2023). What Do We Need to Know About Rising Rates of Idiopathic Pulmonary Fibrosis? A Narrative Review and Update. Springer Nature, Published online 2023 Jan 24. Doi: 10.1007/s12325-022-02395-9.
2 Nathan et al. "Long-term Course and Prognosis of Idiopathic Pulmonary Fibrosis in the New Millennium". Chest Journal Volume 140, ISSUE 1, P221-229, July 2011.
Forward-Looking Statements
This press release may contain forward-looking statements of Aileron Therapeutics, Inc. ("Aileron", the "Company", "we", "our" or "us") within the meaning of the Private Securities Litigation Reform Act of 1995, including statements with respect to: the timing and expectation of the topline results of Cohort 2 of the Phase 1b clinical trial of LTI-03; future expectations, plans and prospects for the Company, the sufficiency of the Company's cash resources; the status and plans for clinical trials, including the timing of data; future product development; and the potential commercial opportunity of LTI-03 and LTI-01. We use words such as "anticipate," "believe," "estimate," "expect," "hope," "intend," "may," "plan," "predict," "project," "target," "potential," "would," "can," "could," "should," "continue," and other words and terms of similar meaning to help identify forward-looking statements, although not all forward-looking statements contain these identifying words. Actual results may differ materially from those indicated by such forward-looking statements as a result of various important factors, including risks and uncertainties related to, changes in applicable laws or regulations, the possibility that the Company may be adversely affected by other economic, business, and/or competitive factors, including risks inherent in pharmaceutical research and development, such as: adverse results in the Company's drug discovery, preclinical and clinical development activities, the risk that the results of preclinical studies and early clinical trials may not be replicated in later clinical trials or that partial results of a trial such as the Cohort 1 results from the Company's ongoing Phase 1b clinical trial will be indicative of the full results of the trial, the Company's ability to enroll patients in its clinical trials, and the risk that any of its clinical trials may not commence, continue or be completed on time, or at all; decisions made by the U.S. Food and Drug Administration and other regulatory authorities, investigational review boards at clinical trial sites and publication review bodies with respect to our development candidates; our ability to obtain, maintain and enforce intellectual property rights for our platform and development candidates; competition; uncertainties as to the sufficiency of the Company's cash resources to fund its planned activities for the periods anticipated and the Company's ability to manage unplanned cash requirements; and general economic and market conditions; as well as the risks and uncertainties discussed in the "Risk Factors" section of the Company's Annual Report on Form 10-K for the year ended December 31, 2023 and Quarterly Report on Form 10-Q for the quarter ended June 30, 2024, which are on file with the United States Securities and Exchange Commission (the "SEC"), and in subsequent filings that the Company makes with the SEC. These forward-looking statements should not be relied upon as representing the Company's view as of any date subsequent to the date of this press release, and we expressly disclaim any obligation to update any forward-looking statements, whether as a result of new information, future events or otherwise, except as required by law.
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