Aileron Therapeutics' Drug in the FDA Approval Process
This section highlights FDA-related milestones and regulatory updates for drugs developed by Aileron Therapeutics (ALRN).
Over the past two years, Aileron Therapeutics has reported clinical trial outcomes, regulatory submissions, approvals, and other FDA events for drugs and therapies such as
LTI-03. For definitions of regulatory abbreviations such as NDA, BLA, or PDUFA, see the event status legend.
LTI-03 FDA Regulatory Timeline and Events
LTI-03 is a drug developed by Aileron Therapeutics for the following indication: In Idiopathic Pulmonary Fibrosis.
This drug is under review by the U.S. Food and Drug Administration (FDA).
Below is a timeline of key regulatory milestones for this therapy.
- Drug:
- LTI-03
- Announced Date:
- June 10, 2025
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Rein Therapeutics, Inc paused enrollment and patient dosing at its clinical trial sites in the United States (U.S.) for its Phase 2 RENEW trial of LTI-03.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- May 27, 2025
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Rein Therapeutics announced that the first patient has been dosed in the RENEW Phase 2 trial of its lead asset, LTI-03, a novel, multi-pathway, Caveolin-1-related peptide for the treatment of IPF.
AI Summary
Rein Therapeutics announced that the first patient has been dosed in its RENEW Phase 2 trial, evaluating its lead asset, LTI-03. LTI-03 is a new multi-pathway, Caveolin-1-related peptide designed for the treatment of idiopathic pulmonary fibrosis (IPF). The trial aims to assess the safety, tolerability, and efficacy of LTI-03 in patients with IPF. This multi-center study is randomized, double-blind, placebo-controlled, and is expected to enroll about 120 patients from up to 50 global sites. Topline interim data is anticipated in the first half of 2026. Rein Therapeutics expressed optimism about the potential of LTI-03 to offer a novel treatment option for patients whose needs are not met by current standard therapies, highlighting the progress made and collaboration with clinical research partners like IQVIA.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- May 12, 2025
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Rein Therapeutics announced that the RENEW Phase 2 trial of its lead asset, LTI-03, a novel, multi-pathway, Caveolin-1-related peptide for the treatment of idiopathic pulmonary fibrosis (IPF), has been initiated with screening and recruitment of patients underway.
AI Summary
Rein Therapeutics announced the initiation of the RENEW Phase 2 trial for its lead asset, LTI-03. LTI-03 is a new, multi-pathway, Caveolin-1-related peptide intended to treat idiopathic pulmonary fibrosis (IPF). This Phase 2 trial is a multi-center, randomized, double-blind, placebo-controlled study designed to evaluate the safety, tolerability, and effectiveness of the treatment in improving lung function.
Screening and recruitment for the trial have already begun, with plans to enroll up to 120 IPF patients. The study will assess various biomarkers and lung function measures over a 24-week treatment period, and topline results are expected in the first half of 2026. Rein’s approach focuses on protecting lung cells and reducing tissue scarring to potentially improve the condition for IPF patients.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- May 1, 2025
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Rein Therapeutics announced a collaboration for the integration of Qureight's deep-learning platform into Rein's planned Phase 2 trial of its lead asset LTI-03, a novel, multi-pathway, Caveolin-1-related peptide, for the treatment of idiopathic pulmonary fibrosis (IPF).
AI Summary
Rein Therapeutics has announced a new collaboration with Qureight Ltd to integrate Qureight’s deep-learning platform into its upcoming Phase 2 trial for LTI-03, a novel peptide aimed at treating idiopathic pulmonary fibrosis (IPF). LTI-03 is designed to work on multiple pathways related to Caveolin-1, potentially slowing lung tissue scarring and promoting healthy lung regeneration. In the trial, Qureight’s AI technology will analyze lung imaging data to measure changes in fibrotic, vascular, and airway compartments. This detailed analysis is expected to reveal correlations between lung volume changes and the activity of LTI-03 across various biomarkers, which were promising in earlier Phase 1b studies. By providing end-to-end imaging services, including site qualification, data handling, and quality control, the technology aims to enhance the trial’s efficiency and help researchers better understand LTI-03’s therapeutic effects in IPF patients.
Read Announcement - Drug:
- LTI-03
- Announced Date:
- April 23, 2025
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Rein Therapeutics announced that the U.S. Patent and Trademark Office (USPTO) granted two new patents related to Rein's lead product candidate, LTI-03, a novel, synthetic peptide with a dual mechanism targeting alveolar epithelial cell survival as well as inhibition of profibrotic signaling, which is administered through dry powder inhalation.
AI Summary
Rein Therapeutics received two new patents from the U.S. Patent and Trademark Office for its lead candidate, LTI-03. The patents, U.S. Patent Nos. 12,280,088 and 12,280,089, protect the dry powder inhalation formulation of LTI-03, a novel synthetic peptide. This innovative treatment is designed with a dual approach: it helps support alveolar epithelial cell survival and works to block profibrotic signaling in the lungs. The dual mechanism offers a promising new option for patients with various lung diseases such as interstitial lung disease, idiopathic pulmonary fibrosis, COPD, and asthma. The patent grant not only secures the intellectual property for LTI-03 but also strengthens Rein Therapeutics’ inhaled peptide program, supporting future clinical development and potentially addressing unmet needs in chronic respiratory conditions.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- October 12, 2024
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Aileron Therapeutics announced the presentation of two abstracts detailing LTI-03's pre-clinical and Phase 1b (NCT05954988) results in Idiopathic Pulmonary Fibrosis (IPF) at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF).
AI Summary
Aileron Therapeutics announced that it will present two abstracts at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF). These abstracts detail both the pre-clinical studies and results from Cohort 1 of its ongoing Phase 1b clinical trial (NCT05954988) in patients with Idiopathic Pulmonary Fibrosis (IPF). The low-dose study (2.5 mg BID) showed positive trends in seven of eight biomarkers, with statistically significant reductions in markers of collagen synthesis, inflammation, and fibrogenesis. These findings suggest that LTI-03 may improve lung function and potentially reverse IPF progression.
Pre-clinical data using precision cut lung slices further support LTI-03’s therapeutic potential by demonstrating reduced pro-fibrotic proteins and pathways. This presentation highlights the promising early clinical and pre-clinical results, reinforcing the potential of LTI-03 as a novel treatment option for IPF patients.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- October 12, 2024
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Aileron Therapeutics announced the presentation of two abstracts detailing LTI-03's pre-clinical and Phase 1b (NCT05954988) results in Idiopathic Pulmonary Fibrosis (IPF) at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF).
AI Summary
Aileron Therapeutics has announced the presentation of two new abstracts at the 22nd International Colloquium on Lung and Airway Fibrosis (ICLAF). These abstracts detail both pre-clinical and Phase 1b (NCT05954988) results for LTI-03, a novel peptide candidate for treating Idiopathic Pulmonary Fibrosis (IPF). In the Phase 1b trial, low-dose LTI-03 (2.5 mg BID) showed a positive trend in seven of eight evaluated biomarkers. Notably, significant reductions were observed in markers linked to collagen synthesis, inflammation, and fibrogenesis, suggesting LTI-03 may improve lung function by reversing disease progression.
Pre-clinical studies using precision cut lung slices further supported these findings by demonstrating LTI-03’s ability to attenuate multiple pro-fibrotic proteins. This promising presentation underscores the therapeutic potential of LTI-03 in addressing unmet needs in IPF treatment.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- September 23, 2024
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Aileron Therapeutics, Inc. announced the completion of enrollment in Cohort 2 of the ongoing Phase 1b clinical trial of LTI-03 in IPF patients.
AI Summary
Aileron Therapeutics has announced the successful enrollment of all patients in Cohort 2 of its ongoing Phase 1b clinical trial for the novel drug candidate LTI-03, aimed at treating idiopathic pulmonary fibrosis (IPF). This cohort will evaluate the safety and tolerability of a higher dose—5 mg administered twice daily via inhalation—in a group of 24 patients who have been randomized to receive either the drug or a placebo. The trial’s primary goal is to monitor the effects of this dosing on patient safety while also measuring various protein biomarkers linked to IPF. Following earlier promising trends seen in a lower dose group in Cohort 1, the completion of this high-dose cohort marks an important step in assessing whether LTI-03 can help improve lung function and potentially alter the progression of IPF. Topline data is expected to be reported soon.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- May 1, 2024
- Estimated Event Date Range:
- July 1, 2024 - September 30, 2024
- Target Action Date:
- Q3 2024
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Aileron Therapeutics, Inc. announced that Data from Cohort 2 evaluating high-dose LTI-03 (5 mg BID) is expected in the third quarter of 2024
AI Summary
Aileron Therapeutics, Inc. announced new progress in its Phase 1b clinical trial of inhaled LTI-03 for patients with idiopathic pulmonary fibrosis (IPF). Earlier results using a low dose of 2.5 mg BID showed promising trends, including reductions in proteins that promote fibrosis and improvements in biomarkers linked to lung health. The company now plans to advance the study by testing a higher dose.
Data from Cohort 2, evaluating high-dose LTI-03 (5 mg BID), is expected to be available in the third quarter of 2024. These upcoming results will be critical in determining the optimal dosing and overall safety of LTI-03. The findings could provide further insight into whether LTI-03 can offer a dual benefit by both protecting vital epithelial cells and reducing fibrotic processes that contribute to the progression of IPF.
Read Announcement- Drug:
- LTI-03
- Announced Date:
- May 1, 2024
- Indication:
- In Idiopathic Pulmonary Fibrosis
Announcement
Aileron Therapeutics, Inc. announced positive data from Cohort 1 of the ongoing Phase 1b clinical trial evaluating the safety and tolerability of inhaled LTI-03 in patients diagnosed with idiopathic pulmonary fibrosis (IPF). LTI-03 is a novel, Caveolin-1-related peptide that addresses both inhibition of pro-fibrotic signaling and survival of critical epithelial cells.
AI Summary
Aileron Therapeutics, Inc. announced encouraging results from Cohort 1 of its ongoing Phase 1b clinical trial of inhaled LTI-03 in patients with idiopathic pulmonary fibrosis (IPF). LTI-03 is a new, Caveolin-1-related peptide that works by blocking pro-fibrotic signaling and supporting the survival of lung epithelial cells. When patients received a low dose (2.5 mg twice daily), the treatment reduced several profibrotic proteins in basal-like cells and fibroblasts while increasing a biomarker linked to healthy epithelial cells. The therapy showed a positive trend in seven out of eight IPF biomarkers and was well-tolerated, with no concerning safety signals observed. These results suggest that LTI-03 may help slow, stabilize, or even reverse the progression of IPF. Further evaluation in higher dose cohorts is planned to more clearly determine its potential benefits for lung function.
Read Announcement
Aileron Therapeutics FDA Events - Frequently Asked Questions
As of now, Aileron Therapeutics (ALRN) has not received any FDA approvals for its therapy in the last two years.
In the past two years, Aileron Therapeutics (ALRN) has reported FDA regulatory activity for LTI-03.
The most recent FDA-related event for Aileron Therapeutics occurred on June 10, 2025, involving LTI-03. The update was categorized as "Enrollment Paused," with the company reporting: "Rein Therapeutics, Inc paused enrollment and patient dosing at its clinical trial sites in the United States (U.S.) for its Phase 2 RENEW trial of LTI-03."
Currently, Aileron Therapeutics has one therapy (LTI-03) targeting the following condition: In Idiopathic Pulmonary Fibrosis.
More FDA Event Resources from MarketBeat
FDA Event Stage Terminology & Abbreviation Guide
- NDA: New Drug Application
- ANDA: Abbreviated New Drug Application
- sNDA: Supplemental New Drug Application
- BLA: Biologics License Application
- sBLA: Supplemental Biologics License Application
- FDA Approved: Approved by the FDA
- EMA: European Medicines Agency
- CE Mark: European Union Certification
- NMPA: China National Medical Products Administration
- MHLW: Japanese Ministry of Health
- FDA Meeting: Consultation with FDA
- Pre-IND: Pre-Investigational New Drug Meeting
- Breakthrough Therapy: Special FDA designation for promising therapies
- Fast Track: Accelerated FDA approval pathway
- Orphan Drug: Designation for rare disease treatments
- RPD: Rare Pediatric Disease Designation
- RMAT: Regenerative Medicine Advanced Therapy
- DSMB Review: Data Safety Monitoring Board Review
- IDMC Review: Independent Data Monitoring Committee
- MAA: MHRA Marketing Authorization Application
- RTF: Refusal to File (Rejected Application)
- 510(k): FDA Clearance for Medical Devices
- Rolling Submission: Staggered regulatory review process
This page (NASDAQ:ALRN) was last updated on 7/18/2025 by MarketBeat.com Staff
