Potential New Oral Treatment for Hereditary Angioedema Scores Well in Safety and Efficacy

Hereditary angioedema (HAE) is a rare condition which causes severe swelling in various areas of the body. Takhzyro has been approved as a treatment for this disease. The drug was created by Shire and it works by inhibiting kallikrein. Kallikrein activates bradykinin. Bradykinin is a molecule that causes fluid to escape blood vessels and enter the body’s tissues, causing them to swell. So, by blocking the activator of bradykinn, the drug prevents swelling attacks.

However, Takhzyro is administered by injection and must be taken every two weeks. Attune Pharmaceuticals began working to develop a treatment which also inhibits kallikrein but can be administered orally. They created ATN-249 and initiated a Phase 1 trial to investigate the drug’s efficacy and safety in healthy volunteers.

The Trial

Attune’s Phase 1 trial is close to completion. Findings have already been presented at the 2019 Annual Scientific Session of the WSAAI. It indicated safety, tolerability, and efficacy of ATN-249. Participants who received the drug achieved high levels of blood plasma and pharmacokinetics were dose-dependent.

The trial has 48 participants- 30 who received ATN-249 (50mg to 8000mg) and 12 who received placebo. Six additional volunteers were specifically monitored to examine any effect food might have on ATN-249’s stability or exposure in the body. Food was found to have no impact on the levels of ATN-249 recognized by the body.

Across all groups in the study, ATN-249 was well tolerated. All adverse events documented in the trial were mild and none were determined to have occurred as a result of the treatment.

Looking Forward

Attune will finish this Phase 1 trial soon. Following completion, they plan to initiate a Phase 2 trial for ATN-249. This trial will last for 4 weeks and will further investigate the drug’s efficacy and safety.

“ATN-249 has the potential to significantly improve disease management and patient quality of life.”

Stay tuned to hear future updates on this investigation. In the meantime, you can read more about ATN-249 and Attune’s work in hereditary angioedema here.


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