FDA Names AMP945, Potential IPF Treatment, an Orphan Drug

The U.S. Food and Drug Administration (FDA) has designated AMP945, Amplia Therapeutics’ investigational anti-fibrotic therapy for idiopathic pulmonary fibrosis (IPF), an orphan drug, supporting its development, the company announced.

Its decision came about one month after AMP945 also received orphan drug status as a potential pancreatic cancer treatment.

This designation carries certain benefits, including incentives for further testing and possible commercialization, U.S. market exclusivity for seven years if approved, clinical trial protocol assistance, and fee exemptions and reductions. It is set aside for treatments being developed for rare disorders.

AMP945 is an inhibitor of the enzyme focal adhesion kinase (FAK) that plays a key role in the formation of scar tissue (fibrosis) both in cancer and diseases like IPF. By blocking FAK’s activity, AMP945 is expected to lessen tissue scarring and make cancer treatments, which are often rendered useless in the presence of fibrosis, more effective.

Preclinical studies in the bleomycin animal model of pulmonary fibrosis found that AMP945 was able to reduce, and even reverse, the formation of scar tissue in the animals’ lungs.

The company is planning to launch a Phase 1 trial this year to evaluate the safety, tolerability, pharmacokinetic, and  pharmacodynamic properties of AMP945 in a group of healthy volunteers.

Pharmacokinetic properties are analyzed to understand how a medication moves through the body; how it is absorbed, distributed, metabolized and then eliminated. Pharmacodynamic analyses look at the effects a particular therapy has in the body.

If this trial supports AMP945 being safe for human use, Amplia is planning to launch Phase 2 trials in 2021 investigating the safety and efficacy of this potential treatment in cancer and IPF patients.

“This Orphan Drug Designation further highlights the extensive opportunities provided by Amplia’s pipeline. With the preclinical toxicology program for AMP945 nearing completion, the company is rapidly moving toward commencement of a Phase 1 study in healthy volunteers later this year,” John Lambert, CEO and managing director of Amplia, said in a press release.

“This … Designation for the use of AMP945 to treat patients with IPF provides further validation of the pipeline of opportunities we are putting in place for our proprietary FAK inhibitors,” Lambert added.