Camurus AB · OMXSTO:CAMX
Camurus AB — Technical Presentation (June 2023)
33 slides from Camurus AB's technical presentation, Jun 20, 2023
Slide 1: ACROINNOVA 1 Phase 3 results
ACROINNOVA 1 Phase 3 results Topline results from a 24-week, randomized, double-blind, placebo-controlled trial evaluating octreotide SC depot in adult patients with acromegaly 20 June 2023 For investor communication only. Octreotide SC depot (CAM2029) is an investigational medical product.
Slide 2: Forward looking statements
2 Forward looking statements This presentation contains forward-looking statements that provide our expectations or forecasts of future events such as new product developments and regulatory approvals and financial performance. Camurus is providing the following cautionary statement. Such forward-lo
Slide 3: Agenda
3 Agenda • Acromegaly and octreotide SC depot Fredrik Tiberg, PhD President & CEO, CSO • The ACROINNOVA program Alberto M. Pedroncelli, MD, PhD • Phase 3 topline results of ACROINNOVA 1 Chief Medical Officer • Next steps towards regulatory submissions Agneta Svedberg, MSc, MBA Vice President Clinica
Slide 4: Octreotide SC depot (CAM2029) showed high level of disease control and positive PRO outcomes
4 Octreotide SC depot (CAM2029) showed high level of disease control and positive PRO outcomes Primary endpoint of superiority met with 72.2% IGF-1 response in the CAM2029 group versus 37.5% in the placebo group Key secondary endpoint of superiority met with 70.0% IGF-1 and GH response in the CAM202
Slide 5: CAM2029 – octreotide subcutaneous depot in Phase 3 development
CAM2029 – octreotide subcutaneous depot in Phase 3 development Investigational treatment of three, serious, rare diseases with high unmet medical needs • Acromegaly • Gastroenteropancreatic neuroendocrine tumors (GEP-NET) • Polycystic liver disease (PLD) Designed for enhanced efficacy and improved c
Slide 6: Acromegaly – a rare growth hormone disorder with significant morbidity
6 Acromegaly – a rare growth hormone disorder with significant morbidity • Rare pituitary disorder caused by a benign tumor and excess PREVALENCE: of growth hormone 50-70 cases per million1 • Slowly developing chronic disease with progressive physical changes or disability, often unrecognized, leadi
Slide 7: Acromegaly symptoms and morbidity
7 Acromegaly symptoms and morbidity •Common symptoms •Benign pituitary tumor •Pulmonary ‒ Fatigue causes excess GH secretion ‒ Sleep apnea ‒ Headache leading to increased IGF-1 levels ‒ Narcolepsy ‒ Paresthesia ‒ Obstruction of upper airways ‒ Soft tissue swelling •Bone overgrowth and ‒ Excessive sw
Slide 8: SRLs are first-line medical treatment
8 SRLs are first-line medical treatment •Transsphenoidal surgery of micro- and macro-adenomas •First-line medical treatment is first-generation, long-acting somatostatin receptor ligands (SRLs) ‒ Octreotide LAR and lanreotide Autogel (rarely dopamine agonist) ‒ Second-line therapy often includes peg
Slide 9: First generation SRLs are standard of care for acromegaly – with improvement potential
9 First generation SRLs are standard of care for acromegaly – with improvement potential •Complex handling and inconvenient dosing •Efficacy limitations with modest response rates ‒ Complex reconstitution – prone to handling errors ‒ Octreotide LAR has low octreotide bioavailability and needle clogg
Slide 10: CAM2029 – targeting key unmet medical needs in acromegaly
10 CAM2029 – targeting key unmet medical needs in acromegaly •Convenient dosing allowing for easy patient self-administration Octreotide subcutaneous depot ‒ Ready to use, with no need for mixing, reconstitution, or temperature (CAM2029) conditioning, and is stored at room temperature ‒ Subcutaneous
Slide 11: ACROINNOVA 1 Topline results
ACROINNOVA 1 Topline results 24-week, Phase 3, randomized, double-blind, placebo-controlled trial of octreotide SC depot
Slide 12: Patient demographics by treatment arm
12 Patient demographics by treatment arm Balanced demographics with patients of different ages: Intention to Treat (ITT) analysis set CAM2029 PLACEBO OVERALL (N=72) Parameter (unit) Statistics or category (N=48) (N=24) n (%) Age (years) Mean (SD) 57 (11.2) 52 (15.1) 55 (12.8) Min-Max 29-79 20-82 20-
Slide 13: Phase 3 trial enrolled adult patients with acromegaly on stable treatment with SoC
13 Phase 3 trial enrolled adult patients with acromegaly on stable treatment with SoC Patients with documented acromegaly history (Intention-to-treat Analysis Set) CAM2029 PLACEBO OVERALL (N=48) (N=24) (N=72) Parameter Category n (%) n (%) n (%) Time since Diagnosis (years) Mean (SD) 10.8 (6.8) 13.0
Slide 14: Phase 3 RCT evaluated efficacy and safety of octreotide SC depot in patients with acromegaly
14 Phase 3 RCT evaluated efficacy and safety of octreotide SC depot in patients with acromegaly • Primary objective • Key secondary endpoints • Other secondary endpoints ‒ To assess the superiority of ‒ Proportion of patients with mean ‒ Biochemical response (IGF-1 and GH) CAM2029 compared to placeb
Slide 15: Phase 3 RCT evaluated efficacy and safety of octreotide SC depot in patients with acromegaly
15 Phase 3 RCT evaluated efficacy and safety of octreotide SC depot in patients with acromegaly • Primary objective • Key secondary endpoints • Other secondary endpoints ‒ To assess the superiority of ‒ Proportion of patients with mean ‒ Biochemical response (IGF-1 and GH) CAM2029 compared to placeb
Slide 16: ACROINNOVA 1 trial patient disposition
16 ACROINNOVA 1 trial patient disposition Randomized 2:1 N=72 CAM2029 PLACEBO N=48 N=24 Withdrawn from trial Withdrawn consent: N=1 Received CAM2029 Received Placebo N=47 N=24 Withdrawn from trial Withdrawn consent: N=1 Completed treatment Discontinued treatment Completed treatment Discontinued trea
Slide 17: Topline efficacy results
Acrolnnova’ Topline efficacy results
Slide 18: Octreotide SC depot achieved superiority for IGF-1 and GH response
18 Octreotide SC depot achieved superiority for IGF-1 and GH response Primary and key secondary endpoints met with high statistical significance (ITT) CAM2029- CAM2029 % Placebo % In favor of Placebo In favor of CAM2029 Upper-tailed Placebo % responders responders p-value (95% CI) Primary efficacy e
Slide 19: Sensitivity analyses confirmed the main analysis
19 Sensitivity analyses confirmed the main analysis Main and sensitivity analyses of the primary endpoint (ITT) CAM2029- CAM2029 % Placebo % Upper-tailed Placebo % In favor of Placebo In favor of CAM2029 responders responders p-value (95% CI) Primary efficacy endpoint 72.2 37.5 34.6 (11.3, 57.9) 0.0
Slide 20: Supportive analyses confirmed the main analysis
20 Supportive analyses confirmed the main analysis Main and supportive analyses of the primary endpoint CAM2029- CAM2029 % Placebo % Upper-tailed Placebo % In favor of Placebo In favor of CAM2029 responders responders p-value (95% CI) Primary efficacy endpoint 72.2 37.5 34.6 (11.3, 57.9) 0.0018 Supp
Slide 21: High statistical difference in time to loss of biochemical response: IGF-1>ULN
21 High statistical difference in time to loss of biochemical response: IGF-1>ULN •Cox regression analysis (ITT): Hazard ratio=0.1; p<0.0001 1 0,9 0,8 0,7 Proportion of responders 0,6 0,5 0,4 0,3 CAM2029 Placebo 0,2 censored 0,1 0 0 4 8 12 16 20 24 28 Weeks
Slide 22: IGF-1 over time confirms efficacy of CAM2029
22 IGF-1 over time confirms efficacy of CAM2029 •IGF-1 values for CAM2029 stable over time (mean, 95% CI) 1,8 IGF-1/ULN Week 24 CAM2029 (N=48) vs. Baseline 1,6 Placebo (N=24) LS Mean CAM2029 0.04 LS Mean Placebo 0.51 1,4 Mean Difference -0.47 (CAM2029 – Placebo) Mean IGF-1/ULN 1,2 95% CI of Differen
Slide 23: Patient reported outcomes
Acrolnnova’ Patient reported outcomes
Slide 24: Significant improvement in patient's QoL indicated versus SoC at baseline
24 Significant improvement in patient’s QoL indicated versus SoC at baseline Significant treatment differences with CAM2029 versus baseline LS Mean of Change AcroQoL Treatment arm Worsening Improving p-value from Baseline CAM2029 4.685 (1.510, 7.861) 0.0038 Total Score Placebo 2.237 (-2.246, 6.721)
Slide 25: High patient reported treatment satisfaction with CAM2029 versus baseline
25 High patient reported treatment satisfaction with CAM2029 versus baseline Answers to Patient Satisfaction Scale at Week 24 versus previous treatment by treatment arm 40 CAM2029 Mean score and 95% CI 30 Placebo % of patients 20 CAM2029 10 Placebo 0 Much worse Slightly worse About the same Slightly
Slide 26: Safety and adverse events overview
Safety and adverse events overview
Slide 27: Summary of adverse events by treatment arm
27 Summary of adverse events by treatment arm Adverse events (AEs) were mostly mild, and none of the serious or severe AEs were related to octreotide SC depot (Safety analysis set) CAM2029 PLACEBO OVERALL (N=47) (N=24) (N=71) Category n (%) n (%) n (%) Any AE 37 (78.7) 19 (79.2) 56 (78.9) Any Relate
Slide 28: Adverse event summary by preferred term
28 Adverse event summary by preferred term Adverse events reported by at least 5% of patients in any treatment arm by preferred term (Safety Analysis Set) CAM2029 PLACEBO OVERALL (N=47) (N=24) (N=71) Preferred term n (%) n (%) n (%) Any AE 37 (78.7) 19 (79.2) 56 (78.9) Injection site erythema 12 (25
Slide 29: Summary and conclusions
Acrolnnova’ Summary and conclusions
Slide 30: Positive topline Phase 3 results from ACROINNOVA 1
30 Positive topline Phase 3 results from ACROINNOVA 1 •Primary and key secondary endpoints met with robust, statistical significance ‒ All sensitivity and supportive analyses confirmed efficacy ‒ Trial met multiple additional secondary endpoints on biochemical control of IGF-1 and GH with octreotide
Slide 31: Upcoming key milestones: ACROINNOVA program
31 Upcoming key milestones: ACROINNOVA program Full results from ACROINNOVA 1 trial, Q3 2023 Interim results ACROINNOVA 2, Q3 2023 Pre-NDA meeting Q3/Q4 2023 NDA submission around the turn of the year 2023/24 Further regulatory submission in EU and RoW
Slide 32: CAM2029 Phase 3 programs progressing
32 CAM2029 Phase 3 programs progressing NDA filing NDA/MAA approval ACRO est. decisions ACRO est. 1 Phase 3 RCT NDA/MAA 2 Phase 3 LST Trial extension approval decisions GEP-NET est. GEP-NET Phase 3 RCT PLD Phase 2/3 RCT Phase 3 continuation -2022 2023 2024 2025 2026 ACRO Phase 3 RCT ACRO Phase 3 LST
Slide 33: Questions
Acrolnnova’ Questions