With 125 patients, HELP is the largest pivotal study in HAE prevention with the longest active duration1-5
TAKHZYRO is clinically proven to help prevent HAE attacks with every-2-weeks dosing that takes ≤1 minute to administer—patients experienced long-term freedom from attacks for an average of 14.8 (SD=12.4) months1,5*†‡
*In clinical studies, the majority of patients self-administered TAKHZYRO within 10 to 60 seconds. These injection times are based on vial adminstration.1
†Evaluated in a 6.5-month study and a 2.5-year open-label extension (OLE) study.1,5
‡Mean duration of the attack-free period in the OLE study was 14.8 (SD=12.4) months (N=209).5
SD=standard deviation.
TAKHZYRO was studied in a range of patients with HAE type I or II1,5
125
PATIENTS1
- 70% Female6
- 30% Male6
- 12 to 73 Years of Age7
6.5
MONTHS OF ACTIVE TREATMENT
(26 weeks)1,6*
56%
PREVIOUSLY TREATED WITH LONG-TERM PREVENTIVE THERAPY1
- 48% were on C1-INH alone6
- 3.2% were on oral therapy6†
- 4.8% were on both6
52%
HAD ≥3 ATTACKS/MONTH DURING RUN-IN PERIOD1
*One month was defined as 28 days in the trial.6
†Androgens or antifibrinolytics.6
44% of patients at baseline in HELP had not previously received long-term preventive therapy.6
HELP study design
*Long-term preventive therapy washout was only for patients ≥18 years of age.7
†Run-in period could be shortened if the patient experienced ≥3 HAE attacks before completion of the 4 weeks, and the period could be extended to 8 weeks if the patient did not experience any attacks during the 4 weeks. During the 8 weeks, the patient needed to have ≥2 attacks to proceed to enrollment and randomization.6
‡Treatments were administered as 2 separate 1-mL injections in the upper arm every 2 weeks to maintain the blind.6
§One month was defined as 28 days in the trial.6
The pivotal trial was a multicenter, double-blind, parallel group, placebo-controlled, dose-ranging study, which assessed the safety and efficacy of TAKHZYRO in 125 patients with HAE type I or II (≥12 years of age). Patients were randomized to receive TAKHZYRO 150 mg every 4 weeks (n=28), TAKHZYRO 300 mg every 4 weeks (n=29), TAKHZYRO 300 mg every 2 weeks (n=27), or placebo (n=41) for 26 weeks (6.5 months, where 1 month was defined as 28 days). Prior to randomization, patients ≥18 years of age were required to complete a ≥2-week long-term prophylaxis washout period. All patients then entered a 4-week run-in period to determine the baseline HAE attack rate. Patients with ≥1 investigator-confirmed HAE attack during the run-in period were eligible for study enrollment and randomization. The primary efficacy endpoint was the rate of investigator–confirmed attacks during the treatment period (time frame: from Day 0 to Day 182).1,6
Rediscover effective prevention
Primary Endpoint
Significant reduction in mean attack rate‡ vs placebo at 6.5 months in the HELP study1,6
- TAKHZYRO 300 mg every 4 weeks resulted in a 73% reduction in attacks vs placebo (Adjusted P<0.001)1§
Mean monthly attack rate during the run-in period6:
| 3.52 for Q2W arm (n=27) |
3.71 for Q4W arm (n=29) |
4.02 for placebo arm (n=41) |
Mean monthly attack rate during the treatment period1:
| 0.26 for Q2W arm |
0.53 for Q4W arm |
1.97 for placebo arm |
All data presented are for TAKHZYRO 300 mg every 2 weeks unless otherwise indicated.1
‡Mean monthly attack rate: number of attacks/4 weeks.1
§Adjusted P-values for multiple testing.1
Q2W=every 2 weeks; Q4W=every 4 weeks.
SELECT IMPORTANT SAFETY INFORMATION
Hypersensitivity reactions have been observed. In case of a severe hypersensitivity reaction, discontinue TAKHZYRO administration and institute appropriate treatment.
Secondary Endpoints
Significant reduction in moderate and severe attacks and attacks requiring acute treatment vs placebo at 6.5 months1,6
‡Adjusted P-values for multiple testing.1
HELP prespecified exploratory endpoints
Subgroup results from the 300 mg Q4W arm of the HELP study
Attack History7
- 80% reduction in attacks on average vs placebo for patients who had 1 to <2 HAE attacks per month at baseline (n=9)*
- 77% reduction in attacks on average vs placebo for patients that had 2 to <3 attacks per month (n=5)
- 71% reduction in attacks on average vs placebo for patients that had 2 ≥3 attacks per month (n=15)
Body Mass Index (BMI)7
- 86% reduction in attacks on average vs placebo for patients with a normal BMI (n=6)†‡
- 70% reduction in attacks on average vs placebo for patients with an overweight BMI (n=5)§
- 74% reduction in attacks on average vs placebo for patients with an obese BMI (n=8)¶
These studies were prespecified exploratory analyses in the pivotal HELP study to evaluate the efficacy and safety of TAKHZYRO compared to placebo in patients of varying BMls and in patients with different baseline run-in attack rates.
Your patient may be considered for less frequent dosing with TAKHZYRO if they are well controlled (eg, attack free) for more than 6 months.
*In the HELP study, TAKHZYRO provided reductions in monthly attack rates relative to placebo in patients with HAE, regardless of baseline attack rate.
†In the HELP study, TAKHZYRO reduced the HAE attack rate compared with placebo, regardless of patients’ BMI.
‡A normal BMI was defined as 18.5 to <25 kg/m2 (n=35).
§An overweight BMI was defined as 25 to <30 kg/m2 (n=43).
¶An obese BMI was defined as ≥30 kg/m2 (n=36).
Exploratory Endpoints
Freedom from HAE attacks in the HELP study
All data presented are for TAKHZYRO 300 mg every 2 weeks unless otherwise indicated.