FDA Gives Fast Track to BCX7353 for Preventing Attacks in HAE Patients

FDA Gives Fast Track to BCX7353 for Preventing Attacks in HAE Patients

The U.S. Food and Drug Administration (FDA) has granted fast track status to BCX7353, an investigational therapy being developed by BioCryst Pharmaceuticals, for the prevention of angioedema attacks in patients with hereditary angioedema.

That status will support the development of BCX7353, speed its review and, depending on how it performs in clinical trials, expedite its entry into the market.

“Fast Track Designation from the FDA serves as another indicator of the importance of BCX7353 to meet an unmet medical need for HAE patients in the U.S.,” Jon P. Stonehouse, president and CEO of BioCryst Pharmaceuticals, said in a press release.

Most angioedema attacks in patients with hereditary angioedema are mediated by a molecule called bradykinin. Bradykinin is a vasodilator, meaning it widens blood vessels, and increases their permeability. When produced in excess, bradykinin may cause too much fluid to escape blood vessels, causing swelling (edema).

BCX7353 was made to inhibit kallikrein, a precursor of bradykinin, and prevent angioedema attacks.

Researchers have been studying BCX7353 as a prophylactic treatment for hereditary angioedema patients in a Phase 2 trial, called APeX-1 (NCT02870972). Recent results showed that attacks are reduced by more than 70 percent with once-daily BCX7353 (125 mg), compared to a placebo, and that more patients remain attack-free for the four week duration of the study.

The investigational medicine also is safe and well-tolerated, with gastrointestinal symptoms being the most common adverse side effect.

Based on the positive results, BioCryst launched two additional clinical trials — the APeX-2 Phase 3 trial (NCT03485911) and the long-term APeX-S Phase 2 trial (NCT03472040) — to continue studying the safety and effectiveness of BCX7353 in reducing the rates of attacks in patients with hereditary angioedema.

APeX-2 is already underway and is expected to include 96 patients across 47 clinical sites in Canada, the U.S., and Europe. Participants will be assigned randomly once-daily BCX7353 or a placebo for a total of 24 weeks.

APeX-S will evaluate the long-term impact of the investigational treatment. It will enroll 160 patients who will receive BCX7353 for 48 weeks.

“We remain focused on completing the Phase 3 program and preparing for an NDA [new drug application] filing in the second half of 2019,” Stonehouse said.

The company is also conducting the ZENITH-1 Phase 2 trial (NCT03240133), a proof-of-concept study to evaluate the potential of an oral liquid formulation of BCX7353 for the acute treatment of angioedema attacks.

Those interested in taking part in any of these trials can find additional information in the clinical trials linked above.