BioCryst Presents New Real-World Evidence Showing High Adherence and Persistence Rates with ORLADEYO® (berotralstat)

Additional real-world studies show sustained HAE attack rate reduction after initiating ORLADEYO, regardless of C1-INH status, and patient preference for an oral LTP therapy

RESEARCH TRIANGLE PARK, N.C., Oct. 24, 2024 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc. (Nasdaq: BCRX) today announced new real-world comparative research on the use of oral, once-daily ORLADEYO® (berotralstat) that found high rates of adherence and persistence for ORLADEYO, similar to the rates observed with two other long-term prophylactic (LTP) therapies for hereditary angioedema (HAE).

The company also announced new real-world evidence showing statistically significant and sustained HAE attack rate reductions after initiating ORLADEYO in patients with HAE, regardless of their C1-inhibitor (C1-INH) deficiency status, and new findings from an HAE patient survey confirming patient preference for an oral LTP therapy.

“These results build on previous research that highlights how the real-world effectiveness and convenience of our oral, once-daily prophylactic therapy are differentiators for patients. The strong adherence, persistence and preference for ORLADEYO we report here provide further evidence that enables physicians to optimize individualized treatment recommendations for each of their patients. We continue to generate real-world evidence demonstrating that ORLADEYO is a powerful, potentially life-changing treatment for many people with HAE,” said Dr. Donald S. Fong, chief medical officer of BioCryst.

The data are being presented at the Annual Scientific Meeting of the American College of Allergy, Asthma & Immunology (ACAAI), which is taking place in Boston from October 24-28, 2024.

High Adherence and Persistence Rates for ORLADEYO and Other LTP Therapies

Poster #R072 explores adherence and persistence rates among patients with HAE following initiation of ORLADEYO (n=90) compared with lanadelumab (n=189) and subcutaneous plasma-derived C1-inhibitor (SC-pdC1-INH) (n=78).

“Our findings reveal that among HAE patients managed with LTP, high adherence and persistence rates were demonstrated for ORLADEYO. There was a striking consistency between first line therapies with comparable results for lanadelumab and SC-pdC1-INH. ORLADEYO rates were equivalent to – and in some cases better than – these LTP counterparts. The outcomes of our study provide further support for a shared decision-making approach within the physician-patient partnership to inform LTP selection, incorporating effectiveness, safety, treatment burden and patient preference for each individual,” said Sandra Christiansen, MD, professor of medicine and director of translational research at the US HAEA Angioedema Center at the University of California, San Diego.

• Adherence And Persistence Among Hereditary Angioedema Patients Treated With Berotralstat, Lanadelumab, And Subcutaneous Plasma-Derived C1-Inhibitor; ePoster #R072; Friday, October 25, 2:30-2:45 p.m. ET; Monitor #19, Hall A
  • Eighty-six percent of patients who took ORLADEYO (n=77), 91 percent who took lanadelumab (n=172) and 83 percent who took SC-pdC1-INH (n=65) filled at least two prescriptions during the 12-month follow-up period.
  • Adherence rates among patients with at least two fills were high at 12 months of follow-up. Seventy-seven percent of patients taking ORLADEYO were adherent compared to 76 percent of patients taking lanadelumab and 80 percent of patients taking SC-pdC1-INH.
  • Persistence rates among patients with at least two fills were high at 12 months of follow-up, with 71 percent of patients taking ORLADEYO persistent compared to 63 percent of patients taking lanadelumab and 63 percent of patients taking SC-pdC1-INH.
  • No statistically significant differences in adherence or persistence were observed between the LTP patient cohorts.

Methods

• Electronic health records linked to claims data were used to select mutually exclusive cohorts of patients aged 12 and above who initiated ORLADEYO, lanadelumab or pdC1-INH between June 22, 2017, and September 12, 2023.
• Adherence was defined as having a proportion of days covered greater than or equal to 0.80.
• Persistence was defined as fewer than 45 consecutive days without the index LTP treatment.

Sustained HAE Attack Rate Reduction After Beginning ORLADEYO Regardless of C1-INH Status

Posters #R092 and #R093 evaluate self-reported HAE attacks prior to and following ORLADEYO initiation among patients with and without HAE with C1-inhibitor deficiency (HAE-nl-C1-INH), respectively.

“ORLADEYO was associated with statistically significant and sustained reductions in attack rates through 18 months (540 days) following ORLADEYO initiation, regardless of patients’ C1-INH status. Importantly, in this real-world study, patients with zero attacks at baseline were included, compared to the minimum of two attacks that was required at baseline in APeX-2. These data confirm that many already well-controlled HAE patients enjoy continued or improved attack control once they initiate ORLADEYO,” Dr. Fong added.

• Sustained Real-World Attack Reductions Following Berotralstat Initiation Among Patients with Hereditary Angioedema with C1-Inhibitor Deficiency; ePoster #R092; Friday, October 25, 3:30-3:45 p.m. ET; Monitor #20, Hall A
  • Patients (n=466) had significantly lower attack rates at each follow-up interval, which ranged from 0.62 attacks per month at days 361-450 (n=185) to 0.79 at days 271-360 (n=217) versus baseline (2.62 at days 361-450 to 2.50 at days 271-360).
  • Mean monthly attack rate reduction was -1.71 (p