FDA Grants Orphan Drug Status to Inhaled Imatinib Formulation

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by Steve Bryson PhD |

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AER-901, an inhaled formulation of imatinib being developed as a treatment for pulmonary arterial hypertension (PAH), has been granted orphan drug designation by the U.S. Food and Drug Administration (FDA).

The investigational therapy was designed with the goal of reducing the side effects reported among PAH patients using an approved oral formulation of imatinib.

The orphan drug designation, given to the therapy’s developer, Aerami Therapeutics, supports investigative treatments for rare diseases — those that affect fewer than 200,000 people in the U.S. — through financial incentives for development and commercialization. It allows for exclusivity in the U.S. market for seven years following approval, as well as FDA application fee exemptions and tax credits for clinical studies.

“Receiving orphan drug designation for imatinib in AER-901 is another important milestone to emerge from our PAH development program,” Steve Thornton, CEO of Aerami, said in a press release.

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The oral formulation of imatinib, marketed as Gleevec by Novartis, is currently approved to treat certain types of cancers. The therapy also has been shown to reduce the tightening and stiffness of pulmonary arteries, a hallmark of PAH. Based on these properties, Novartis launched IMPRES, a Phase 3 clinical trial (NCT00902174) to evaluate imatinib as add-on PAH therapy.

Although the therapy demonstrated clinically meaningful improvements in blood flow and physical exercise capacity, almost half of the participants experienced serious side effects thought to be due to the high oral dose used in the study.

Now, Aerami has reformulated imatinib as an inhaled therapy, dubbed AER-901, aiming to target the lungs only, thus avoiding the body-wide toxicity reported with the oral formulation.

The new treatment is designed to deliver consistent, well-tolerated, and effective levels of imatinib, once a day, via the Fox device licensed from the Vectura Group.

“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,” said Timm Crowder, PhD, president of Aerami.

“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,” Crower said.

Dosing with AER-901 has begun in a Phase 1 trial (NCT04903730) that is evaluating the therapy’s safety, tolerability, and pharmacokinetics. Pharmacokinetics is how a medicine moves into, through, and out of the body.

AER-901 is being tested at two doses, 5 and 40 mg/mL, and compared with a placebo, in up to 78 healthy adult volunteers. The Australian study is expected to be completed by the end of the year, the company said. Based on its findings, Aerami plans to initiate Phase 2/3 trials involving PAH patients early in 2022.

“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,” Thornton said.