AI Therapeutics (formerly LAM Therapeutics) said that it is seeking a partner to advance its LAM-001 inhaled rapamycin into Phase 3 trials for the treatment of lymphangioleiomyomatosis (LAM disease) and into Phase 2 for the treatment of pulmonary arterial hypertension (PAH). The company said that LAM-001 has been granted Orphan Drug designation for LAM in Europe and for both indications in the US.
According to AI, “Through low-dose inhalation into the lung, LAM-001 has the potential to deliver efficacious doses of rapamycin while minimizing the systemic side effects observed with oral formulations of rapamycin.”
AI cited Simon Johnson of the University of Nottingham, who agreed that, “LAM-001 may offer an opportunity for women with LAM disease to stabilize their lung function and reduce the side effects associated with oral rapamycin,” and Lewis Rubin of the University of California, San Diego School of Medicine, who said, “I also see tremendous potential for LAM-001 as a new mechanistic approach to treat pulmonary arterial hypertension (PAH) since rapamycin has effects on two pathways – mammalian target of rapamycin (mTOR) and bone morphogenetic protein receptor type II (BMPR2) that are involved in the pathogenesis of PAH.”
Read the AI Therapeutics press release.
