US-based biotechnology company AI Therapeutics has begun a Phase II clinical trial of LAM-001, an inhaled form of sirolimus, to treat pulmonary arterial hypertension (PAH).
The open-label, single-arm study aims to assess LAM-001’s tolerability, safety and activity in WHO-functional Class III adults with advanced PAH.
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A total of 15 patients aged 18 years and older who are symptomatic despite background therapy will receive LAM-001 for 24 weeks.
The study’s primary endpoints are the safety, tolerability and change from baseline in peak oxygen uptake (VO₂ max), as recorded by invasive cardiopulmonary exercise testing (iCPET), after 24 weeks.
Its secondary endpoints include changes in cardiac index, pulmonary capillary wedge pressure, pulmonary vascular resistance and cardiac output, as determined by iCPET, after the same period.
The study will also assess pharmacokinetics, time to clinical worsening, change from baseline in WHO functional class and six-minute walking distance in patients treated with LAM-001.
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By GlobalDataAI Therapeutics CEO Dr Brigette Roberts said: “LAM-001 offers an innovative solution to delivering sirolimus directly to diseased lungs, thereby potentially minimising systemic exposure.
“Recognising that there is still an acute need to offer additional treatments for this population, we are excited to have begun dosing in our Phase II LAM-001 study in PAH.”
Patients will be followed for an extension period of 12 months after they have completed the first 24 weeks of treatment.
The trial’s principal investigator is Dr Aaron Waxman, director of the Pulmonary Vascular Disease Programme at Brigham and Women’s Hospital.
Dr Waxman said: “By modulating both the mTOR and BMPR2 pathways, LAM-001 has the potential to reduce smooth muscle cell hyperproliferation and ameliorate endothelial cell dysfunction in the pulmonary vasculature, thereby altering the long-term course of the disease.
“Even as new disease-modifying agents become available, because of its unique mechanism of action, LAM-001 may become an important addition to our growing armamentarium of drugs used to treat this devastating disease.”
