ECE2024 Poster Presentations Pituitary and Neuroendocrinology (120 abstracts)
A randomised phase 3 trial to assess efficacy and safety of CAM2029, an octreotide subcutaneous depot, in patients with acromegaly
Diego Ferone 1 , Julie Silverstein 2 , Pamela Freda 3 , Laurence Katznelson 4 , Federico Gatto 1 , Pinar Kadioglu 5 , Pietro Maffei 6 , Jochen Seufert 7 , Joanna L. Spencer-Segal 8 , Elena Isaeva 9 , Alexander Dreval 10 , Maria Harrie 11 , Agneta Svedberg 11 & Fredrik Tiberg 11
1Endocrinology Unit, Department of Internal Medicine, IRCCS Ospedale Policlinico San Martino, Genova, Italy; 2Division of Endocrinology, Metabolism and Lipid Research, Department of Neurosurgery, Washington University School of Medicine, St. Louis, MO, United States; 3Department of Medicine, Vagelos College of Physicians & Surgeons, Columbia University, New york, Ny, United States; 4Departments of Neurosurgery and Medicine, Stanford University School of Medicine, Stanford, CA, United States; 5Division of Endocrinology-Metabolism and Diabetes, Department of Internal Medicine, Istanbul University-Cerrahpasa, Istanbul, Turkey; 6Department of Medicine, Padua University Hospital, Padua, Italy; 7Division of Endocrinology and Diabetology, Department of Medicine II, Medical Faculty, University of Freiburg, Freiburg, Germany; 8Department of Internal Medicine and Michigan Neuroscience Institute, University of Michigan, Ann Arbor, MI, United States; 9Interregional Clinical Diagnostic Center, Kazan, Russian Federation; 10Endocrinology Department of Moscow Regional Research Clinical Institute, Moscow, Russian Federation; 11Camurus AB, Lund, Sweden
Background: Acromegaly is a rare endocrine disorder characterised by excess growth hormone (GH) and insulin-like growth factor 1 (IGF-1) that is associated with significant morbidity and impaired quality of life (QoL). Standard-of-care (SoC) treatments for acromegaly typically require healthcare provider administration, pose a substantial treatment burden, and leave scope for improved disease control. CAM2029, an octreotide subcutaneous depot with ~5x higher bioavailability than octreotide long-acting release (LAR), is designed for once-monthly self-administration as a ready-to-use syringe or injection pen. Here, we report findings from the pivotal trial of CAM2029 in patients with acromegaly.
Methods: In this Phase 3, multinational, randomised, double-blind, placebo-controlled trial (NCT04076462), patients on stable SoC treatment (octreotide LAR or lanreotide Autogel) who had IGF-1 ≤1x upper limit of normal (ULN) at screening were randomised 2:1 to once-monthly CAM2029 20 mg or placebo for 24 weeks. The primary endpoint was the proportion of patients with IGF-1 ≤ULN (mean of week 22/24 measurements). The key secondary endpoint was the proportion of patients with both IGF-1 ≤ULN (week 22/24 mean) and mean GH <2.5 µg/l (week 24). Patient-reported outcomes (PROs) were compared at baseline and week 24.
Results: Seventy-two patients were randomised to CAM2029 (n=48) or placebo (n=24). A significantly greater proportion of CAM2029 vs placebo recipients achieved the primary (IGF-1 response: 72.2% vs 37.5%; P=0.0018) and key secondary endpoints (IGF-1 and GH response: 70.0% vs 37.5%; P=0.0035). These results were supported by all sensitivity and supportive analyses. Median time to loss of response (IGF-1 >ULN) was 8.4 weeks in the placebo arm and was not reached in the CAM2029 arm. Mean IGF-1 remained below ULN throughout the study for patients receiving CAM2029 but not for patients receiving placebo. PROs showed improved QoL, treatment convenience, and patient satisfaction for CAM2029 vs baseline SoC, with numerically greater improvements vs placebo. CAM2029 was well tolerated with a comparable safety profile to SoC; no new or unexpected safety signals were observed. One treatment-related serious adverse event (cholecystitis) occurred in the placebo arm, and 5 patients discontinued treatment due to injection site erythema/induration (CAM2029: n=3; placebo: n=1) or migraine (CAM2029: n=1).
Conclusion: CAM2029 provided robust biochemical control of acromegaly superior to placebo, substantially improved PROs vs baseline SoC and placebo, and had a safety profile consistent with SoC. These findings support CAM2029 as a potential therapeutic alternative to SoC acromegaly treatments that addresses unmet patient needs.
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Endocrine Abstracts
ISSN 1470-3947 (print) | ISSN 1479-6848 (online)
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