Biocryst to present new Orladeyo, navenibart data at AAAAI meeting
Findings include pediatric Orladeyo results and interim navenibart data
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Biocryst Pharmaceuticals will present new clinical trial and real-world data on its treatments for hereditary angioedema (HAE) at this year’s American Academy of Allergy, Asthma & Immunology (AAAAI) annual meeting, the company announced.
The data include six abstracts reporting updated findings on Orladeyo (berotralstat), an approved oral medication used to help prevent swelling attacks in people ages 2 and older with HAE. These include interim results from the ongoing Phase 3 APeX-P trial (NCT05453968), which is evaluating the safety and pharmacological properties of once-daily Orladeyo oral granules in 29 children ages 2 to less than 12, with follow-up lasting up to nearly three years.
Additional abstracts highlight investigational therapy navenibart
Three additional abstracts will present new clinical trial results on navenibart, an investigational treatment designed to help prevent HAE attacks and potentially reduce how often injections are needed. These include a late-breaking abstract reporting positive interim results from the ongoing Phase 2 ALPHA-SOLAR trial (NCT06007677), an open-label extension study evaluating long-term outcomes in 29 adults with HAE who previously enrolled in the Phase 2 ALPHA-STAR trial (NCT05695248).
According to the company, eight posters will be presented on Friday, Feb. 27, at the Pennsylvania Convention Center in Philadelphia.
“The breadth of data to be presented at AAAAI, including a late-breaking abstract, reflect the continued evolution of our strategy to expand and diversify our HAE portfolio in ways that matter to patients and their care teams,” Charlie Gayer, Biocryst’s CEO, said in a company press release. “From advancing programs like navenibart to the launch of an expanded pediatric indication for ORLADEYO, we are focused on delivering meaningful treatment options that align with individual patient needs, preferences, and lifestyles, supported by rigorous and innovative clinical evidence.”
HAE is caused by excessive production of bradykinin, an inflammatory molecule that widens blood vessels and leads to swelling.
Orladeyo works by blocking kallikrein, an enzyme that mediates bradykinin’s production, helping prevent swelling attacks. The therapy was initially approved in the U.S. and other regions to prevent swelling attacks in people with HAE ages 12 and older.
APeX-P trial data supported expanded pediatric approval of Orladeyo
Data from the APeX-P supported the recent extended approval of Orladeyo by the U.S. Food and Drug Administration to children ages 2-11, authorizing a new oral pellet formulation.
Early trial data showed the therapy was generally well tolerated, reduced attack rates within the first month, and lowered the percentage of days with HAE symptoms as early as three months. After one year, 70.4% of children were attack-free, and the median monthly attack rate fell to zero early in treatment and remained at that level for most of the first year. At that time point, 93.1% of participants had completed at least 48 weeks, or roughly 11 months, of treatment.
New interim data, titled “Oral Berotralstat Reduces the Rate of Moderate and Severe Attacks and Percentage of Days with HAE Symptoms Over 48 Weeks in Children Aged 2 to Less Than 12 Years: Interim Data from APeX-P,“ further showed reductions in disease burden over 48 weeks of treatment. Among 29 children, the median monthly rate of moderate attacks declined from 0.33 during standard care to 0.16 with Orladeyo, while the rate of severe attacks fell from 0.31 to zero per month. The percentage of days with HAE symptoms also decreased, from a median of 9% during standard care to 3% during treatment. No patients discontinued due to adverse events.
Navenibart designed as long-acting preventive treatment for HAE
Navenibart, which recently joined Biocryst’s pipeline through its acquisition of Astria Therapeutics, is a long-acting antibody therapy designed to block kallikrein, the same target as Orladeyo. The therapy is designed to be given as subcutaneous (under-the-skin) injection every 3 or 6 months, a less frequent schedule than many existing injectable treatments.
Earlier results from ALPHA-SOLAR showed that treatment with navenibart every three to six months reduced swelling attacks by a mean of about 92% over 17.4 months of follow-up across the ALPHA-STAR and ALPHA-SOLAR trials. Updated interim findings, titled “Long-Term, Sustained, Robust Hereditary Angioedema Attack Suppression with Navenibart Administered Every 3 and 6 Months: ALPHA-SOLAR Interim Results,” showed these reductions were maintained for up to 24 months (about two years). Among people receiving navenibart every three months, attack rates were reduced by a mean of 92%, and by 90% in those treated every six months.
Navenibart is now being evaluated in the Phase 3 ALPHA-ORBIT trial (NCT06842823), which is recruiting patients, ages 12 and older, with HAE types 1 or 2, at sites worldwide. Top-line results are expected in 2027 and may support future regulatory approval.
