SAT-026 - Patient-Reported Outcomes of Debio 4126, A First 12-Week Formulation of Octreotide in Acromegaly Patients: Results from the OXTEND-01 Trial

Background: OXTEND-01, a Phase 1b, open label, multicenter trial in patients with controlled acromegaly was conducted to characterize pharmacokinetics, pharmacodynamics, safety, and tolerability of Debio 4126, a novel long-acting octreotide formulation administered intramuscularly every 12 weeks. Descriptive Patient-Reported Outcomes (PROs) data were also evaluated as exploratory endpoints to assess quality of life and treatment satisfaction.

Methods: OXTEND-01 enrolled patients with biochemically controlled acromegaly (Insulin-like growth factor 1 [IGF-1] ≤1.3x upper limit of normal [ULN]) who were stable on standard-of-care (SoC) octreotide or lanreotide monotherapy. Patients switched from SoC to receive Debio 4126 every 12 weeks, for a duration of 12 months. Key efficacy endpoints were IGF-1 and Growth hormone (GH) levels.
PROs were assessed using exploratory endpoints focused on Health-Related Quality of Life (HRQoL) and treatment satisfaction. The Acromegaly Quality of Life Questionnaire (AcroQoL), a disease-specific questionnaire, was used to measure the impact and burden of acromegaly symptoms on quality of life. Changes in AcroQoL scores were evaluated from baseline to 24 weeks (Day 1 of injection 3) and 48 weeks (End of treatment: EoT).
The Treatment Satisfaction Questionnaire for Medication (TSQM) was used to measure satisfaction and convenience. Changes in TSQM scores were assessed from baseline to 7 days after the first Debio 4126 injection and 21 days after the third injection. For both questionnaires, higher scores indicate an improvement.

Results: OXTEND-01 trial enrolled 16 patients with acromegaly. Of the 16 participants, 15 (93.8%) participants completed the study. Median age of the participants was 57.0 years. Most participants maintained their IGF-1 levels throughout treatment (13 of 15 participants ((86.7%) below 1.3× ULN at 48 weeks).
PRO scores progressively improved over time. The AcroQoL questionnaire results (range 0-100) showed a mean global score of 60.9 at 48 weeks (EoT), representing a mean increase of 3.1 from the baseline (interquartile range (IQR): -0.2, 6.7). Results from the TSQM (range 0-100) showed an overall improvement in participant satisfaction after the third injection compared to baseline on SoC. This improvement was evidenced across all TSQM sub-domains: ‘convenience’ improved by 9.6 points (IQR: 0.0, 22.2), ‘effectiveness’ by 8.1 points (IQR:0.0, 22.2), self-reported side-effects by 11.3 points (IQR:0.0, 31.2) and ‘global satisfaction’ by 8.6 (IQR: 0.0, 14.3).

Conclusions: Debio 4126, administered every 12 weeks maintained biochemical control of acromegaly throughout treatment in the majority of the participants and showed favourable safety and tolerability profiles, similar to SoC. Patients reported numerical improvements in QoL, treatment convenience and satisfaction with Debio 4126 vs SoC.

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