The FDA has granted orphan drug designation to Aerami Therapeutics’s AER-901 inhaled imatinib for the treatment of pulmonary arterial hypertension (PAH), the company said. Aerami initiated a Phase 1 trial of AER– 901 in June 2021, with results expected by the end of the year.
In June 2020, Aerami announced a partnership with inhalation CDMO Vectura for development of the nebulized imatinib for PAH, which will be delivered via Vectura’s Fox nebulizer. Earlier this year, Hangzhou Chance Pharmaceuticals acquired the rights to develop and commercialize AER-901 for the treatment of pulmonary arterial hypertension in China, Hong Kong, Macau, and Taiwan.
Aerami CEO Steve Thornton commented, “Receiving orphan drug designation for imatinib in AER-901 is another important milestone to emerge from our PAH development program. We are pleased with the progress we are making in our Phase 1 trial and believe that AER- 901, which is targeted to enter Phase 2/3 trials in the first half of 2022, might, for the first time, provide the opportunity to modify the course of this terrible disease, and offer an important therapeutic option for patients.”
Aerami President Timm Crowder said, “By delivering Aerami’s proprietary inhaled imatinib directly to the site of the disease, we believe AER-901 has the potential to significantly reduce the dose necessary to achieve therapeutic benefit thereby avoiding the adverse events seen with oral imatinib. Our nebulized formulation and delivery system has the potential to improve efficacy through deeper lung penetration and better drug uptake while reducing the potential for side effects like cough, which is commonly associated with other inhaled technologies such as dry powder formulations.”
Read the Aerami Therapeutics press release.
