ALPROLIX^®: the first EHL FIX
WITH >10 years OF REAL-WORLD EXPERIENCE^1–3

ALPROLIX PROPHYLAXIS RESULTED IN LOW ABR
IN A REAL-WORLD SETTING⁸

In a real-world switch from previous FIX in France (N=91*), ALPROLIX demonstrated:⁸

Effective bleed treatment with ALPROLIX on-demand⁸

Primary endpoints in patients who received ALPROLIX on-demand during the prospective period (n=7):*⁸

Median (IQR) ALPROLIX dose used to treat a bleeding episode: 42.7 (41.4–51.7) IU/kg

Median (IQR) number of injections used to treat a bleeding episode: 1.0 (1.0–1.0)

6 patients experienced a total of 18 bleeding episodes⁸

(5 patients experienced 14 traumatic bleeds and 3 patients experienced 4 spontaneous bleeds)

Low ABRs among those receiving ALPROLIX prophylaxis⁸

Primary endpoints in patients who received ALPROLIX prophylaxis during the
prospective period (n=84):⁸

	Mean/median (IQR) ABR	Mean (SD) annualised injection frequency	Mean annualised dispensed ALPROLIX consumption, IU/kg/year (SD)
Patients with prior FIX prophylaxis (n=68)	1.6/1.2 (0.0–2.5)	47.45 (13.13)	2,844 (1,043)
Patients with prior FIX on-demand (n=15)†	1.4/1.0 (0.0–1.6)	31.00 (13.52)	1,900 (944)

Mean (range) observation period for the overall population was 21.6 months (3.1–30.6).⁸
*Since a limited number of patients were treated with on-demand ALPROLIX, these results should be interpreted with caution.^{8 †}Data are missing for one patient.⁸
ABR, annualised bleeding rate; FIX, factor IX; IQR, interquartile range; SD, standard deviation.

IMPROVED BLEED PROTECTION AND
REDUCED INJECTION FREQUENCY VS PRIOR FIX⁸

Improved ABRs⁸

ABRs numerically reduced with ALPROLIX prophylaxis vs pre-switch FIX⁸

Secondary endpoint: ABR pre- and post-switch to ALPROLIX⁸

Figures adapted from Chambost H, et al. 2025.⁸

Reduced factor consumption⁸

Median annualised factor consumption numerically reduced by
436 IU/kg/year with ALPROLIX vs pre-switch FIX prophylaxis⁸

Secondary endpoint: Median factor consumption pre- and post-switch to ALPROLIX (n=57)^‡8

Reduced injection frequency⁸

Secondary endpoint: Median injection frequency numerically
reduced by 44 injections per year vs prior prophylaxis (n=57)^‡8

Pre-switch: 94.8 injections/year
Post-switch: 51.2 injections/year

*63/65 patients with available data both pre- and post-switch to ALPROLIX prophylaxis were included.^{8 †}14/18 patients with available data both pre- and post-switch to ALPROLIX prophylaxis were included.^{8 ‡}57/65 patients with available data both pre- and post-switch to ALPROLIX prophylaxis were included.⁸
ABR, annualised bleeding rate; FIX, factor IX; IQR, interquartile range.

EFFECTIVE JOINT PROTECTION AND
TREATMENT SATISFACTION WITH ALPROLIX⁸

Resolved target joints⁸

Secondary endpoint:

• At enrolment, 7 patients had 1 target joint, and 2 patients had 2 target joints⁸
• At the end of the observation period, 9 target joints were resolved*⁸

Patient and physician satisfaction⁸

Secondary endpoint:

• 83% (n=70/84) of physicians and 77% (n=65/84) of patients were satisfied or highly satisfied with ALPROLIX prophylaxis^†8

*A total of 11 target joints were identified at enrolment; however, the resolution status for two target joints from one patient were not assessed as they ended the study less than 12 months after the enrolment visit.^{8 †}At last assessment. Patients' and physicians' satisfaction were evaluated by asking them to rate satisfaction with the outcome of their treatment on a scale of 1–5, with 5 being highly satisfied and 1 being highly dissatisfied.⁸

ALPROLIX WAS WELL TOLERATED, AND SAFETY
DATA WERE CONSISTENT WITH PHASE 3 STUDIES⁸

Only serious adverse events (SAEs) and non-serious adverse events (non-SAEs) that
led to ALPROLIX discontinuation were collected, from the first ALPROLIX dose to
the patient’s last study visit.⁸

In the overall population, 26.4% of patients (n=24/91) experienced
at least one SAE (n=48 SAEs were recorded in total).*⁸

• The most frequently reported SAEs (each reported for n=3) were fall, rectal haemorrhage, chest pain and haematuria⁸
• One patient developed two SAEs (hypersensitivity and a low-titre FIX inhibitor) which were assessed by the investigator as related to treatment and led to discontinuation of ALPROLIX^†8
• No other AEs were assessed as treatment-related during the prospective period⁸

*One patient experienced a fatal SAE (acute pulmonary oedema) but this was deemed unrelated ALPROLIX treatment.^{8 †}The patient (severe haemophilia B; aged 32 years) received prior on-demand FIX treatment and switched to 50 IU/kg ALPROLIX weekly prophylaxis. It was reported they had a family history of inhibitors and a previous history of low-titre inhibitors associated with an allergic reaction. Recovery from the hypersensitivity reaction occurred on the day of the event.⁸
AE, adverse event; FIX, factor IX.

IMPROVED BLEED PROTECTION WITH
ALPROLIX VS PRIOR FIX⁸

A multicentre, prospective, non-interventional study to evaluate the real-world use and effectiveness of ALPROLIX treatment over a 24-month period in France.⁸

Adapted from Chambost H, et al. 2025.⁸

Patients and duration of observation period⁸

• Males with haemophilia B, enrolled at 21 HTCs in France*
• 89% (n=81/91) had severe haemophilia B
• 94.5% (n=86/91) completed the 24-month study period
• Median (range) age: 34.2 (4–84) years
• Mean (range) observation period for the overall study population: 21.6 (3.1–30.6) months

Primary endpoints (patients on ALPROLIX prophylaxis):^†8

• ABR
• Annualised injection frequency
• Annualised factor consumption

Patients with ≥3 months prospective follow-up on ALPROLIX⁸

Primary endpoints (patients on ALPROLIX on-demand):⁸

The amount of ALPROLIX and the number of injections used to treat a bleeding episode

Treatment regimen pre- and post-switch to ALPROLIX⁸

Adapted from Chambost H, et al. 2025.⁸

Study outcomes used descriptive statistics only; no formal statistical testing was performed.⁸
*A total of 21/22 participating haemophilia treatment centres in France enrolled patients in B-SURE.^{8 †}ABR was based on bleeding episodes reported by the healthcare professional at study visits, annualised injection frequency was assessed by prescription and usage at regular visits and annualised factor consumption was assessed by prescription and usage.^{8 ‡}One patient had multiple switches between on-demand and prophylaxis regimens following ALPROLIX prophylaxis initiation, but the final regimen was on-demand.^{8 §}Final ALPROLIX regimen refers to the last documented regimen.⁸
ABR, annualised bleeding rate; FIX, factor IX; HTC, haemophilia treatment centre.