Results From a Study of a Potential Therapy for HAE Have Been Shared

Initial results from a clinical trial investigating the effects of an experimental drug called BCX7353 in patients with hereditary angioedema suffering from acute attacks. For more detailed information, you can view the source press release here, at BioCryst’s website.

About Hereditary Angioedema

Hereditary angioedema (HAE) is a rare genetic condition that can be potentially life-threatening. According to the US Hereditary Angioedema Society, HAE is estimated to affect one in 10,000 to 50,000 people. HAE can cause recurrent episodes of swelling known as ‘attacks’. The swelling can cause different symptoms depending on where it occurs. For example, one common area to be affected is the intestinal tract, and this can cause excruciating abdominal pain. The airway can also be affected by swelling, which can be life-threatening if someone is unable to breathe. The frequency and severity of attacks may vary a lot over time and between patients. However, according to the NIH, untreated people tend to have an attack every one to two weeks, with each attack lasting approximately three to four days.

About the Zenith-1 Clinical Trial

The Zenith-1 clinical trial investigated the effects of an oral investigational drug (BCX7353) in patients with HAE suffering from acute attacks. Adults who have HAE types I or II took a drug within one hour of attack symptom onset for three attacks. Two of the drugs were active, and one was a placebo, and the patients didn’t know which were which. The patients’ attack symptoms and severity were recorded using the visual analogue scale (VAS) and a questionnaire.

The Results

BioCryst has just announced initial results from this trial, which show that BCX7353 was better than the placebo for most of the study’s endpoints, and well-tolerated in patients.

The researchers found patients that took BCX7353 showed an improvement in symptoms and VAS scores compared to placebo as early as one hour after taking the drug. This was continued for 24 hours, during which SOC medication use was approximately one third lower for those taking the drug compared to those taking a placebo. In addition, approximately patients reported no or mild symptoms for two-thirds of attacks treated with BCX7353, compared to approximately one-third of those for which patients took a placebo.

No serious adverse events were reported, but two patients discontinued treatment (one due to a rash, and another due to abdominal pain).

For more information about the study and these results, you can view the original press release here.


Anna Hewitt

Anna Hewitt

Anna is from England and recently finished her undergraduate degree. She has an interest in medicine and enjoys writing. In her spare time she likes to cook, hike, and hang out with cats.

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