Inhaled IPF Therapy LTI-03 Well-Tolerated in Healthy Volunteers

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by Steve Bryson, PhD |

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LTI-03 inhaled IPF therapy | Pulmonary Fibrosis News | illustration of clinical trial findings

Lung Therapeutics’ LTI-03, an investigational inhaled therapy for idiopathic pulmonary fibrosis (IPF), was safe and well-tolerated in healthy volunteers, with no reports of serious adverse events (side effects) or discontinuations, according to a Phase 1a trial.

“We are pleased that LTI-03 has passed this important safety milestone as we continue development,” Brian Windsor, PhD, president and CEO of Lung Therapeutics, said in a press release.

“We believe that the mechanism LTI-03 is unique among drugs in development for IPF, and we are excited to move LTI-03 into the next stage of clinical development in IPF patients later this year,” Windsor said.

The process of wound healing in the body in response to tissue damage normally begins with the activation of cells called fibroblasts. In IPF, the most common type of pulmonary fibrosis, chronic injury leads to uncontrolled fibroblast activation and excessive deposits of scar tissue, or fibrosis. This is particularly the case in alveolar epithelial cells, which line the lungs’ tiny air sacs where gas exchanges occur. As a result, breathing becomes progressively more difficult.

“IPF is a lethal lung disease for which novel and well-tolerated therapies are urgently needed,” said Fernando Martinez, MD, chief of pulmonary and critical care medicine at New York-Presbyterian Hospital/Weill Cornell Medical Center.

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LTI-03 contains a small portion, or peptide, of Caveolin-1 (Cav1), a protein that plays an essential role in preventing fibrosis by maintaining a balance between the pathways that initiate and stop the wound healing process. Cav1 production is lower in IPF lungs and also in mouse models with induced fibrosis.

According to Lung Therapeutics, the therapy — formulated as a dry powder for direct lung administration — aims to restore the balance of pathway signals to slow lung function decline, but also restore lung function by protecting healthy alveolar epithelial cells.

In preclinical studies, LTI-03 successfully suppressed pro-fibrotic signaling pathways and preserved alveolar epithelial cells in the lungs.

“Lung Therapeutics LTI-03 uniquely inhibits both fibroblast activation and promotes the survival and function of alveolar epithelial cell subtypes needed for lung regeneration in IPF,” Martinez said.

The Phase 1a ascending dose study (NCT04233814) enrolled 71 healthy, non-smoking male and female volunteers, ages 18–55. The study’s objectives were to assess LTI-03’s initial safety, tolerability, and pharmacological properties.

Groups of participants were randomly assigned a single, escalating dose of LTI-03 or a placebo on day 1, then multiple ascending doses (LTI-03 or placebo) once daily for 14 days.

Doses between 2.5 and 10 mg were well-tolerated with no reported serious adverse events or discontinuations. According to preclinical data, LTI-03 was effective when given at doses equivalent to 1–10 mg in humans.

“The established safety of LTI-03 in normal volunteers clears the advancement of this inhaled therapeutic into IPF patients,” Martinez said.

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