HELP STUDY

HELP OLE STUDY

REAL WORLD EMPOWER STUDY

SPRING STUDY

With 125 patients, HELP is the largest pivotal study in HAE prevention¹

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) months^1,3*†‡

^*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.^1,3

^‡Mean duration of the attack-free period in the OLE study was 14.8 (SD=12.4) months (N=209).³
SD=standard deviation.

TAKHZYRO was studied in a range of patients with HAE type I or II^1,3

*One month was defined as 28 days in the trial.^3
†Androgens or antifibrinolytics.³

HELP study design

^*Long-term preventive therapy washout was only for patients ≥18 years of age.^4
†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.²

^‡Treatments were administered as 2 separate 1-mL injections in the upper arm every 2 weeks to maintain the blind.²

^§One month was defined as 28 days in the trial.²

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).^1,2

Click here to jump to the HR-QoL data below

Rediscover effective prevention

Primary Endpoints

Significant reduction in mean attack rate* vs placebo at 6.5 months in the HELP study^1,2

• 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 period²:

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 months^1,2

^‡Adjusted P-values for multiple testing.¹

HELP prespecified exploratory endpoints

Subgroup results from the 300 mg Q4W arm of the HELP study

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.

SELECT IMPORTANT SAFETY INFORMATION
Adverse Reactions: The most commonly observed adverse reactions (≥10%) associated with TAKHZYRO were injection site reactions consisting mainly of pain, erythema, and bruising at the injection site; upper respiratory infection; headache; rash; dizziness; diarrhea; and myalgia. Less common adverse reactions observed included elevated levels of transaminases; one patient discontinued the trial for elevated transaminases.

HR-QoL prespecified exploratory endpoint

The burden of HAE goes beyond the attack

The unpredictable nature of HAE can affect various facets of a patient’s life.⁵

In the HELP study, quality of life (QoL) measures were evaluated using the AE-QoL and EQ-5D-5L questionnaires⁶

Limitations: These results should be interpreted with caution as they are based on patient recall and are observational/descriptive in nature. These data were also from an exploratory objective and had less evidentiary value than the primary and secondary objectives. The AE-QoL was administered to 10 adolescent patients in the study, an age group for which the instrument was not validated.⁶

For the EQ-5D-5L questionnaire, an instrument used to measure health status on a given day, no differences were observed. The nondisease-specific EQ-5D-5L questionnaire was administered on days 0, 98, and 182.⁶

Significantly more patients taking TAKHZYRO vs placebo experienced improvements in AE-QoL in the 6.5-month HELP study⁶:

• 81% of patients receiving TAKHZYRO 300 mg Q2W (95% CI, 61-93; n=26) experienced improvement in AE-QoL total score vs 37% of patients taking placebo (P<0.05; 95% CI, 22-54; n=38)^1,6
• Patients receiving TAKHZYRO 300 mg Q2W were 7.2 times more likely to achieve improvement in AE-QoL total score vs patients taking placebo (P<0.01)⁶

Definitions: The AE-QoL is a validated, angioedema-specific questionnaire in adults that was administered monthly, consisting of 4 domains (functioning, fatigue/mood, fears/shame, nutrition) and total scores. The minimal clinically important difference (MCID) is the minimum change in score that is meaningful to patients. For the AE-QoL total score, the predefined MCID is a reduction of 6 points.^6-8

AE-QoL=Angioedema Quality of Life Questionnaire; EQ-5D-5L=5-level EuroQol 5-dimensional; HR-QoL=health-related quality of life; Q2W=every 2 weeks.

Explore more studies

HELP OLE STUDY

REAL WORLD EMPOWER STUDY

SPRING STUDY

References: 1. Takhzyro. Prescribing information. Dyax Corp; 2025. 2. Banerji A, Riedl MA, Bernstein JA, et al. JAMA. 2018;320(20):2108-2121. doi:10.1001/jama.2018.16773 3. Banerji A, Bernstein JA, Johnston DT, et al; HELP OLE Investigators. Allergy. 2022;77(3):979-990. doi:10.1111/all.15011 4. Data on file, TAK743-303, Takeda Pharmaceuticals USA, Inc. 5. Busse PJ, Christiansen SC, Riedl MA, et al. J Allergy Clin Immunol Pract. 2021;9(1):132-150.e3. doi:10.1016/j.jaip.2020.08.046 6. Lumry WR, Weller K, Magerl M, et al; HELP Study Investigators. Allergy. 2021;76(4):1188-1198. doi:10.1111/all.14680 7. Weller K, Groffik A, Magerl M, et al. Allergy. 2012;67(10):1289-1298. doi:10.1111/all.12007 8. Weller K, Magerl M, Peveling-Oberhag A, Martus P, Staubach P, Maurer M. Allergy. 2016;71(8):1203-1209. doi:10.1111/all.12900