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

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BioCryst Pharmaceuticals presented new real-world evidence for ORLADEYO® (berotralstat), showing high adherence and persistence rates comparable to other HAE long-term prophylactic therapies. The study revealed that 86% of ORLADEYO patients filled at least two prescriptions, with 77% adherence and 71% persistence rates at 12 months.

Additional data demonstrated significant HAE attack rate reductions after ORLADEYO initiation, regardless of C1-inhibitor status. Patient survey results indicated a preference for oral therapy, with 54% choosing oral daily treatment over injectable options when efficacy is equal.

BioCryst Pharmaceuticals ha presentato nuove evidenze di vita reale per ORLADEYO® (berotralstat), mostrando elevati tassi di aderenza e persistenza comparabili ad altre terapie profilattiche a lungo termine per l'HAE. Lo studio ha rivelato che l'86% dei pazienti in trattamento con ORLADEYO ha effettuato almeno due prescrizioni, con un tasso di aderenza del 77% e un tasso di persistenza del 71% a 12 mesi.

Dati aggiuntivi hanno dimostrato riduzioni significative del tasso di attacchi di HAE dopo l'inizio del trattamento con ORLADEYO, indipendentemente dallo stato del C1-inibitore. I risultati delle indagini tra i pazienti hanno indicato una preferenza per la terapia orale, con il 54% che seleziona il trattamento orale giornaliero rispetto alle opzioni iniettabili quando l'efficacia è equivalente.

BioCryst Pharmaceuticals presentó nuevas evidencias del mundo real para ORLADEYO® (berotralstat), mostrando altas tasas de adherencia y persistencia comparables a otras terapias profilácticas a largo plazo para el HAE. El estudio reveló que el 86% de los pacientes tratados con ORLADEYO rellenaron al menos dos recetas, con un 77% de adherencia y un 71% de persistencia a los 12 meses.

Datos adicionales demostraron reducciones significativas en la tasa de ataques de HAE después del inicio de ORLADEYO, sin importar el estado del inhibidor C1. Los resultados de la encuesta a los pacientes indicaron una preferencia por la terapia oral, con un 54% eligiendo el tratamiento oral diario sobre las opciones inyectables cuando la eficacia es igual.

BioCryst Pharmaceuticals는 ORLADEYO® (berotralstat)에 대한 새로운 실제 증거를 발표하며, 높은 순응도와 지속성 비율이 다른 HAE 장기 예방 요법과 유사하다는 것을 보여주었습니다. 연구 결과, ORLADEYO 환자의 86%가 최소 두 번의 처방을 받았으며, 12개월 기준으로 77%의 순응도와 71%의 지속성 비율을 보였습니다.

추가 데이터는 ORLADEYO 사용 시작 후 HAE 공격률의 유의미한 감소가 있음을 보여주었으며, 이는 C1 억제제 상태와 관계없이 발생합니다. 환자 설문조사 결과, 경구 요법에 대한 선호도가 나타났으며, 54%가 효과가 동일할 경우 주사 요법보다 경구 일일 치료를 선택했습니다.

BioCryst Pharmaceuticals a présenté de nouvelles preuves du monde réel concernant ORLADEYO® (berotralstat), montrant des taux d'adhérence et de persistance élevés comparables à d'autres thérapies prophylactiques à long terme pour l'HAE. L'étude a révélé que 86% des patients prenant ORLADEYO ont rempli au moins deux prescriptions, avec un taux d'adhérence de 77% et un taux de persistance de 71% à 12 mois.

Des données supplémentaires ont montré des réductions significatives du taux d'attaques HAE après le début d'ORLADEYO, quel que soit le statut du C1-inhibiteur. Les résultats de l'enquête auprès des patients ont indiqué une préférence pour la thérapie orale, 54% choisissant le traitement oral quotidien plutôt que les options injectables lorsque l'efficacité est équivalente.

BioCryst Pharmaceuticals hat neue Real-World-Daten zu ORLADEYO® (berotralstat) präsentiert, die zeigen, dass hohe Adhärenz- und Persistenzraten mit anderen langfristigen prophylaktischen Therapien für HAE vergleichbar sind. Die Studie ergab, dass 86% der ORLADEYO-Patienten mindestens zwei Rezepte ausgefüllt haben, mit 77% Adhärenz und 71% Persistenzraten nach 12 Monaten.

Zusätzliche Daten zeigten signifikante Reduzierungen der HAE-Anfallrate nach Beginn von ORLADEYO, unabhängig von dem Status des C1-Inhibitors. Die Ergebnisse der Patientenumfrage wiesen auf eine Präferenz für die orale Therapie hin, wobei 54% die tägliche orale Behandlung den injizierbaren Optionen bei gleicher Wirksamkeit vorzogen.

Positive
  • High adherence (77%) and persistence (71%) rates for ORLADEYO, comparable to other HAE therapies
  • Significant reduction in HAE attack rates across all patient groups
  • Strong patient preference (54%) for oral therapy over injectable options
Negative
  • None.

Insights

This real-world evidence study demonstrates high adherence rates for ORLADEYO, with 86% of patients filling at least two prescriptions and 77% maintaining adherence over 12 months. The data shows statistically significant attack rate reductions in both C1-INH deficient and non-deficient patients, sustaining effectiveness through 18 months.

The comparative analysis against injectable alternatives (lanadelumab and SC-pdC1-INH) reveals ORLADEYO achieved similar or better persistence rates at 71% versus 63% for competitors. Patient preference data indicating 54% would choose oral over injectable therapy when efficacy is equal suggests strong market positioning.

These findings validate ORLADEYO's commercial viability and potential for continued market share growth in the HAE prophylaxis space, particularly given its unique oral administration advantage and demonstrated real-world effectiveness.

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<0.05) at days 271-360 (n=217) and -1.96 (p<0.05) at days 451-540 (n=157).
  • Sustained Real-World Attack Reductions Following Berotralstat Initiation Among Patients with Hereditary Angioedema without C1-Inhibitor Deficiency; ePoster #R093; Friday, October 25, 3:45-4:00 p.m. ET; Monitor #20, Hall A

    • Patients (n=353) had significantly lower attack rates at each follow-up interval, which ranged from 1.63 attacks per month at days 181-270 (n=191) to 2.04 at days 1-90 (n=304) versus baseline (4.33 at days 181-270 to 4.63 at days 1-90).
    • Mean monthly attack rate reduction was -2.91 (p<0.05) at days 271-360 (n=157) and -2.53 (p<0.05) at days 451-540 (n=108).

Methods

  • Outcomes were collected through BioCryst’s sole-source pharmacy and included U.S. patients who actively received ORLADEYO 110 or 150 mg QD from December 15, 2020, to January 8, 2024.
  • Patient-reported HAE attack rates were collected at baseline and each refill (approximately every 30 days).
  • To be included in the analysis of a given follow-up period, patients were required to have responded to a follow-up assessment within the period of interest and to the onboarding self-assessment of attacks. Approximately 96 percent of patients met these criteria at each interval.

Patients with HAE Prefer Oral LTP

Poster #R081 examines the LTP treatment preferences of patients with HAE, as collected through an online survey.

“We are encouraged to see both effectiveness and convenience are drivers of treatment preference with patients preferring oral therapy over injectable therapy when efficacy is assumed equal. Given the benefits of treatment with ORLADEYO for patients who remain on therapy over the long term, we believe we will continue to see the preference towards oral therapy reflected in the strong commercial performance of our oral, once-daily prophylactic therapy for HAE,” Dr. Fong noted.

  • Long-Term Prophylactic Treatment Preferences of Patients With Hereditary Angioedema; ePoster #R081; Friday, October 25, 4:45-5:00 p.m. ET; Monitor #19, Hall A

    • LTP treatment attributes rated most important were effectiveness in attack prevention and effectiveness in reducing attack severity.
    • When presented with the option of an equally effective oral or injectable LTP, 54 percent preferred an oral daily therapy over a biweekly or monthly subcutaneous injection.
    • More than half (52 percent) preferred oral over injectable LTP as infrequently as every three months.

Methods

  • Results from an online survey of U.S. patients with HAE who were at least 18 years of age, self-reported their diagnosis and were receiving an HAE treatment (LTP, on-demand or both) or had experienced at least one HAE attack in the preceding three months (n=150).

In addition to displaying in the exhibit hall at the noted times, ePosters are accessible online and on demand to registered attendees on ACAAI’s website.

About ORLADEYO® (berotralstat)
ORLADEYO® (berotralstat) is the first and only oral therapy designed specifically to prevent attacks of hereditary angioedema (HAE) in adult and pediatric patients 12 years and older. One capsule of ORLADEYO per day works to prevent HAE attacks by decreasing the activity of plasma kallikrein.

U.S. Indication and Important Safety Information

INDICATION
ORLADEYO® (berotralstat) is a plasma kallikrein inhibitor indicated for prophylaxis to prevent attacks of hereditary angioedema (HAE) in adults and pediatric patients 12 years and older.

Limitations of use
The safety and effectiveness of ORLADEYO for the treatment of acute HAE attacks have not been established. ORLADEYO should not be used for the treatment of acute HAE attacks. Additional doses or dosages of ORLADEYO higher than 150 mg once daily are not recommended due to the potential for QT prolongation.

IMPORTANT SAFETY INFORMATION

An increase in QT prolongation was observed at dosages higher than the recommended 150 mg once-daily dosage and was concentration dependent.

The most common adverse reactions (≥10% and higher than placebo) in patients receiving ORLADEYO were abdominal pain, vomiting, diarrhea, back pain, and gastroesophageal reflux disease.

A reduced dosage of 110 mg taken orally once daily with food is recommended in patients with moderate or severe hepatic impairment (Child-Pugh B or C).

Berotralstat is a substrate of P-glycoprotein (P-gp) and breast cancer resistance protein. P-gp inducers (eg, rifampin, St. John’s wort) may decrease berotralstat plasma concentration, leading to reduced efficacy of ORLADEYO. The use of P-gp inducers is not recommended with ORLADEYO.

ORLADEYO at a dose of 150 mg is a moderate inhibitor of CYP2D6 and CYP3A4. For concomitant medications with a narrow therapeutic index that are predominantly metabolized by CYP2D6 or CYP3A4, appropriate monitoring and dose titration is recommended. ORLADEYO at a dose of 300 mg is a P-gp inhibitor. Appropriate monitoring and dose titration is recommended for P-gp substrates (eg, digoxin) when coadministering with ORLADEYO.

The safety and effectiveness of ORLADEYO in pediatric patients <12 years of age have not been established.

There are insufficient data available to inform drug-related risks with ORLADEYO use in pregnancy. There are no data on the presence of berotralstat in human milk, its effects on the breastfed infant, or its effects on milk production.

To report SUSPECTED ADVERSE REACTIONS, contact BioCryst Pharmaceuticals, Inc. at 1-833-633-2279 or FDA at 1-800-FDA-1088 or www.fda.gov/medwatch.

Please see full Prescribing Information.

About BioCryst Pharmaceuticals
BioCryst Pharmaceuticals is a global biotechnology company with a deep commitment to improving the lives of people living with complement-mediated and other rare diseases. BioCryst leverages its expertise in structure-guided drug design to develop first-in-class or best-in-class oral small-molecule and protein therapeutics to target difficult-to-treat diseases. BioCryst has commercialized ORLADEYO® (berotralstat), the first oral, once-daily plasma kallikrein inhibitor, and is advancing a pipeline of small-molecule and protein therapies. For more information, please visit www.biocryst.com or follow us on LinkedIn.

Forward-Looking Statements

This press release contains forward-looking statements, including statements regarding future results, performance or achievements and statements relating to ORLADEYO performance and patient preferences. These statements involve known and unknown risks, uncertainties and other factors which may cause actual results, performance or achievements to be materially different from any future results, performance or achievements expressed or implied by the forward-looking statements. These statements reflect our current views with respect to future events and are based on assumptions and are subject to risks and uncertainties. Given these uncertainties, you should not place undue reliance on these forward-looking statements. Some of the factors that could affect the forward-looking statements contained herein include: BioCryst’s ability to successfully implement or maintain its commercialization plans for ORLADEYO, which could take longer or be more expensive than planned; the commercial viability of ORLADEYO, including its ability to achieve sustained market acceptance; the FDA or other applicable regulatory agency may require additional studies beyond the studies planned for products and product candidates, may not provide regulatory clearances which may result in delay of planned clinical trials, may impose certain restrictions, warnings, or other requirements on products and product candidates, may impose a clinical hold with respect to product candidates, or may withhold, delay, or withdraw market approval for products and product candidates; BioCryst’s ability to successfully manage its growth and compete effectively; and risks related to the international expansion of BioCryst’s business. Please refer to the documents BioCryst files periodically with the Securities and Exchange Commission, specifically BioCryst’s most recent Annual Report on Form 10-K, Quarterly Reports on Form 10-Q, and Current Reports on Form 8-K, which identify important factors that could cause the actual results to differ materially from those contained in BioCryst’s forward-looking statements.

BCRXW

Contact:
John Bluth
+1 919 859 7910
jbluth@biocryst.com


FAQ

What are the adherence rates for ORLADEYO (BCRX) compared to other HAE treatments?

ORLADEYO showed 77% adherence rate, compared to 76% for lanadelumab and 80% for SC-pdC1-INH, with no statistically significant differences between treatments.

How effective is ORLADEYO (BCRX) in reducing HAE attacks?

ORLADEYO demonstrated significant attack rate reductions, with patients experiencing between 0.62 to 0.79 attacks per month during follow-up compared to 2.50-2.62 at baseline.

What percentage of HAE patients prefer ORLADEYO (BCRX) oral therapy over injectable treatments?

54% of patients preferred oral daily therapy over injectable options when efficacy is equal, and 52% preferred oral over injectable even when injections were as infrequent as every three months.

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