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Camurus' POSITANO study shows treatment effects with CAM2029 in polycystic liver disease patients

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Camurus announced positive topline results from its POSITANO Phase 2b study evaluating CAM2029 for polycystic liver disease (PLD). The study met its primary endpoint, showing a 4.3% reduction in height-adjusted liver volume and 8.7% reduction in liver cyst volume compared to placebo at week 53. The trial involved 71 participants randomized across two CAM2029 dosing regimens and placebo. CAM2029, which has received Orphan Drug Designation in both US and EU, demonstrated good tolerability with no unexpected safety concerns. Common side effects included diarrhea, gastrointestinal disturbances, and injection site reactions. PLD, affecting approximately 37,000 people in the US and EU, currently has no approved treatments. Based on these results, Camurus plans to discuss a Phase 3 study design with regulatory authorities.
Camurus ha annunciato risultati positivi preliminari dallo studio POSITANO di Fase 2b che valuta CAM2029 per la malattia policistica del fegato (PLD). Lo studio ha raggiunto l'endpoint primario, mostrando una riduzione del 4,3% del volume epatico corretto per altezza e una riduzione dell'8,7% del volume delle cisti epatiche rispetto al placebo alla settimana 53. La sperimentazione ha coinvolto 71 partecipanti randomizzati in due regimi di dosaggio di CAM2029 e placebo. CAM2029, che ha ottenuto la Designazione di Farmaco Orfano sia negli USA che in Europa, ha dimostrato una buona tollerabilità senza preoccupazioni di sicurezza inattese. Gli effetti collaterali comuni includevano diarrea, disturbi gastrointestinali e reazioni nel sito di iniezione. La PLD, che colpisce circa 37.000 persone negli USA e in Europa, non dispone attualmente di trattamenti approvati. Sulla base di questi risultati, Camurus prevede di discutere il disegno di uno studio di Fase 3 con le autorità regolatorie.
Camurus anunció resultados positivos preliminares de su estudio POSITANO de Fase 2b que evalúa CAM2029 para la enfermedad poliquística hepática (PLD). El estudio cumplió con su objetivo primario, mostrando una reducción del 4.3% en el volumen hepático ajustado por altura y una reducción del 8.7% en el volumen de quistes hepáticos en comparación con placebo en la semana 53. El ensayo incluyó a 71 participantes asignados aleatoriamente a dos regímenes de dosificación de CAM2029 y placebo. CAM2029, que ha recibido la Designación de Medicamento Huérfano tanto en EE.UU. como en la UE, demostró buena tolerabilidad sin preocupaciones de seguridad inesperadas. Los efectos secundarios comunes incluyeron diarrea, trastornos gastrointestinales y reacciones en el sitio de inyección. La PLD, que afecta aproximadamente a 37,000 personas en EE.UU. y la UE, no cuenta actualmente con tratamientos aprobados. Basándose en estos resultados, Camurus planea discutir el diseño de un estudio de Fase 3 con las autoridades regulatorias.
Camurus는 다낭성 간 질환(PLD) 치료를 위한 CAM2029의 POSITANO 2b상 시험에서 긍정적인 주요 결과를 발표했습니다. 본 연구는 53주차에 키에 맞게 조정된 간 용적이 4.3% 감소하고 간 낭종 용적이 위약 대비 8.7% 감소하는 1차 평가변수를 충족했습니다. 총 71명의 참가자가 두 가지 CAM2029 투여 요법과 위약군에 무작위 배정되었습니다. 미국과 유럽에서 희귀의약품 지정(Orphan Drug Designation)을 받은 CAM2029는 예상치 못한 안전성 문제 없이 우수한 내약성을 보였습니다. 흔한 부작용으로는 설사, 위장 장애 및 주사 부위 반응이 있었습니다. 약 37,000명이 영향을 받는 PLD는 현재 승인된 치료제가 없습니다. Camurus는 이 결과를 바탕으로 규제 당국과 3상 시험 설계에 대해 논의할 계획입니다.
Camurus a annoncé des résultats positifs préliminaires de son étude POSITANO de phase 2b évaluant CAM2029 pour la maladie polykystique du foie (PLD). L'étude a atteint son critère principal, montrant une réduction de 4,3 % du volume hépatique ajusté à la taille et une réduction de 8,7 % du volume des kystes hépatiques par rapport au placebo à la semaine 53. L'essai a inclus 71 participants répartis aléatoirement entre deux schémas posologiques de CAM2029 et un placebo. CAM2029, qui a obtenu la désignation de médicament orphelin aux États-Unis et en Europe, a démontré une bonne tolérance sans problèmes de sécurité inattendus. Les effets secondaires courants comprenaient diarrhée, troubles gastro-intestinaux et réactions au site d'injection. La PLD, qui touche environ 37 000 personnes aux États-Unis et en Europe, ne bénéficie actuellement d'aucun traitement approuvé. Sur la base de ces résultats, Camurus prévoit de discuter de la conception d'une étude de phase 3 avec les autorités réglementaires.
Camurus gab positive Zwischenergebnisse der POSITANO Phase-2b-Studie bekannt, in der CAM2029 zur Behandlung der polyzystischen Lebererkrankung (PLD) untersucht wurde. Die Studie erreichte den primären Endpunkt und zeigte in Woche 53 eine 4,3%ige Reduktion des höhenkorrigierten Lebervolumens sowie eine 8,7%ige Verringerung des Leberzystenvolumens im Vergleich zu Placebo. An der Studie nahmen 71 Teilnehmer teil, die auf zwei Dosierungsschemata von CAM2029 und Placebo randomisiert wurden. CAM2029, das sowohl in den USA als auch in der EU die Orphan-Drug-Designation erhalten hat, zeigte eine gute Verträglichkeit ohne unerwartete Sicherheitsbedenken. Häufige Nebenwirkungen waren Durchfall, gastrointestinale Störungen und Reaktionen an der Injektionsstelle. PLD betrifft etwa 37.000 Menschen in den USA und der EU und es gibt derzeit keine zugelassenen Therapien. Basierend auf diesen Ergebnissen plant Camurus, das Studiendesign für eine Phase-3-Studie mit den Zulassungsbehörden zu besprechen.
Positive
  • Met primary endpoint with statistically significant 4.3% reduction in liver volume (p=0.044)
  • Achieved 8.7% reduction in liver cyst volume (p=0.016)
  • Demonstrated improvements in disease symptoms and patient outcomes
  • Received Orphan Drug Designation in both US and EU
  • Addresses an unmet medical need with no current approved treatments
  • Well-tolerated safety profile consistent with established treatments
Negative
  • Most common side effects included diarrhea, gastrointestinal disturbances, and injection site reactions
  • Will require additional Phase 3 study before potential approval
  • Limited market size of 37,000 patients in US and EU combined

Insights

CAM2029 shows promise for PLD patients with significant liver and cyst volume reductions, addressing a critical unmet need.

The POSITANO Phase 2b study represents a significant clinical milestone for Camurus, demonstrating that CAM2029 (octreotide SC depot) can effectively reduce both liver and cyst volume growth in polycystic liver disease patients. The trial met its primary endpoint with a 4.3% relative reduction in height-adjusted liver volume compared to placebo (p=0.044), and an even more impressive 8.7% reduction in total liver cyst volume (p=0.016).

These results are particularly meaningful given PLD's status as a rare genetic disorder affecting approximately 37,000 people in the US and EU, with no currently approved treatments. The statistical significance achieved despite the relatively small sample size (71 participants) is encouraging. The p-values of 0.044 and 0.016 clear the standard threshold of 0.05, though the liver volume reduction p-value is close to this cutoff.

The safety profile appears consistent with established somatostatin receptor ligands, with primarily gastrointestinal side effects and injection site reactions – an acceptable profile for a chronic condition with significant symptom burden. The reported improvements in disease symptoms and patient-reported outcomes further strengthen the clinical relevance of these findings.

The Orphan Drug Designation in both US and EU markets provides regulatory advantages and market exclusivity potential. With plans to discuss Phase 3 study design with regulatory authorities, Camurus has a clear development pathway. The ongoing open extension phase will provide valuable long-term data that could strengthen the eventual regulatory submission and address durability questions.

  • CAM2029 reduces liver and cyst volume growth compared to placebo 
  • Well tolerated with no new or unexpected safety findings
  • A follow-up Phase 3 study will be discussed with regulatory authorities

LUND, Sweden, June 18, 2025 /PRNewswire/ -- Camurus (NASDAQ STO: CAMX) today announced topline results from the 12-month, randomized, double-blind, placebo-controlled POSITANO Phase 2b study (NCT05281328), evaluating efficacy and safety of octreotide subcutaneous (SC) depot (CAM2029) in patients with symptomatic polycystic liver disease (PLD). 

PLD is a rare genetic disorder causing progressive liver cysts, leading to severe symptoms and reduced quality of life. Approximately 37,000 people in the US and EU have PLD, most of whom are women.1 Currently, no approved treatment exists for PLD.

"The POSITANO study shows that CAM2029 significantly reduces liver and liver cyst volume compared to placebo while showing improvements in symptoms as assessed by relevant patient reported outcomes", says Dr Joost P.H. Drenth, Professor of Hepatology, Amsterdam University Medical Center and coordinating investigator for the POSITANO study. "There is a high unmet medical need for treatment of patients with polycystic liver disease and the POSITANO study brings hope for our patients."

POSITANO enrolled 71 participants with symptomatic PLD randomized to treatment with one out of two dosing regimens of CAM2029 or placebo in a 1:1:1 ratio. The study met the primary endpoint showing a statistically significant relative reduction of the height-adjusted liver volume (htTLV) from baseline to week 53 of 4.3% (p=0.044) for the combined CAM2029 groups compared to placebo. The corresponding relative reduction in total liver cyst volume was 8.7% (p=0.016). Treatment with CAM2029 also resulted in improvements in disease symptoms and other patient and clinical reported outcomes.

"The POSITANO study met the primary endpoint showing that CAM2029 reduces liver and cyst growth and can improve disease symptoms and other outcomes in patients with polycystic liver disease", says Fredrik Tiberg, Camurus' President & CEO, CSO. "Based on the results, Camurus intends to discuss the design of a confirmatory Phase 3 study with regulatory authorities in the US and Europe."

CAM2029 was well tolerated with a safety profile consistent with the established safety profile of approved injectable somatostatin receptor ligands, octreotide and lanreotide. The most frequently reported adverse effects were diarrhea, and mild to moderate gastrointestinal disturbances and injection site reactions. No new or unexpected safety findings were noted. Detailed results from the POSITANO study will be presented at future scientific meetings and in publications.

After completion of the randomized part of POSITANO, the treatment of patients continues with CAM2029 in a 2.5-year, open extension phase of the study where further long-term efficacy and safety data are being collected. CAM2029 has been granted Orphan Drug Designation (ODD) for the treatment of autosomal dominant PLD in the US by the U.S. Food and Drug Administration (FDA) and in the EU by the European Commission.

For more information
Fredrik Tiberg, President & CEO
Tel. +46 (0)46 286 46 92
fredrik.tiberg@camurus.com
 

Fredrik Joabsson, Chief Business Development Officer
Tel. +46 (0)70 776 17 37
ir@camurus.com
 

About polycystic liver disease
Polycystic liver disease (PLD) is a rare genetic and chronic disorder characterized by progressive growth of fluid-filled cysts in the liver, which can cause severe symptoms such as abdominal pain and discomfort, shortness of breath (dyspnea), indigestion (dyspepsia), gastro-esophageal reflux, and limited mobility. Rare complications are hepatic cyst hemorrhage, infection or rupture.2-5 Age and gender contribute to disease severity; increasing age is positively associated with both cyst sizes and numbers, and women are highly overrepresented among symptomatic patients.6-8 Most patients with PLD are diagnosed in their 30s after reporting a sudden and accelerated increase of waist width together with PLD-related symptoms.7 There is currently no approved pharmacological treatment for PLD. Clinical studies indicate that somatostatin receptor ligands, e.g., octreotide, can slow down cyst growth, decrease fluid secretion, and reduce the liver volume.9-11

About octreotide SC depot (CAM2029)
CAM2029 is a ready-to-use, long-acting subcutaneous depot of octreotide under development for the treatment of three rare disease indications: acromegaly, gastroenteropancreatic neuroendocrine tumors (GEP-NET), and polycystic liver disease (PLD). CAM2029 has been evaluated in a comprehensive clinical program, including five Phase 1 and 2 studies, two Phase 3 studies in acromegaly (ACROINNOVA 1 and 2), an ongoing Phase 3 study in patients with GEP-NET (SORENTO), and the Phase 2/3 study in patients with PLD (POSITANO). CAM2029 is designed for enhanced octreotide exposure and convenient, once-monthly administration with a prefilled autoinjector pen to facilitate easy self-administration by patients.

About Camurus
Camurus is an international, science-led biopharmaceutical company committed to developing and commercializing innovative, long-acting medicines for improving the lives of patients with severe and chronic diseases. New drug products with best-in-class potential are conceived based on the company's proprietary FluidCrystal® technology and its extensive R&D expertise. The R&D pipeline includes products for the treatment of dependence, pain, cancer, and endocrine diseases. Camurus has operations across Europe, the US, and Australia, with headquarters in Lund, Sweden. The company's shares are listed on Nasdaq Stockholm under the ticker CAMX. For more information, visit www.camurus.com and LinkedIn.

References

  1. Est. in US and EU4+UK. Globe Life Sciences report 2020; data on file.
  2. Abu-Wasel, B., et al. Pathophysiology, epidemiology, classification and treatment options for polycystic liver diseases. World J Gastroenterol. 2013. 19(35): p. 5775-86.
  3. Perugorria, M.J., et al. Polycystic liver diseases: advanced insights into the molecular mechanisms. Nat Rev Gastroenterol Hepatol. 2014. 11(12): p. 750-61.
  4. Neijenhuis, M.K., et al. Impact of liver volume on polycystic liver disease-related symptoms and quality of life. United European Gastroenterol J. 2018. 6(1): p. 81-88.
  5. Olaizola P., et al. Genetics, pathobiology and therapeutic opportunities of polycystic liver disease. Nat Rev Gastroenterol Hepatol. 2022 May 13.
  6. van Keimpema L., et al. Patients with isolated polycystic liver disease referred to liver centres: clinical characterization of 137 cases. Liver international. 2011;31(1):92-8.
  7. van Aerts RMM, et al. Clinical management of polycystic liver disease. J Hepatol. 2018;68(4):827-37.50.
  8. van Aerts RMM, et al. Severity in polycystic liver disease is associated with aetiology and female gender: Results of the International PLD Registry. Liver international. 2019;39(3):575-82.
  9. Gevers T. J. G., et al. Effect of lanreotide on polycystic liver and kidneys in autosomal dominant polycystic kidney disease: an observational trial. Liver International. 2015 May;35(5):1607-14.
  10. Pisani A., et al. Long-term Effects of Octreotide on Liver Volume in Patients With Polycystic Kidney and Liver Disease. Clin Gastroenterol Hepatol. 2016 Jul;14(7):1022-1030.
  11. van Aerts RMM, et al., Lanreotide Reduces Liver Growth In Patients With Autosomal Dominant Polycystic Liver and Kidney Disease. Gastroenterology. 2019 Aug;157(2):481-491.

This information is information that Camurus AB is obliged to make public pursuant to the EU Market Abuse Regulation. The information was submitted for publication, through the agency of the managing director, at 8:00 am CET on 18 June 2025.

This information was brought to you by Cision http://news.cision.com

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FAQ

What were the main results of Camurus' POSITANO Phase 2b study for PLD?

The study met its primary endpoint, showing a 4.3% reduction in height-adjusted liver volume and 8.7% reduction in liver cyst volume compared to placebo after 53 weeks of treatment with CAM2029.

How many patients were enrolled in the CAMX POSITANO trial?

The POSITANO study enrolled 71 participants who were randomized into three groups (1:1:1 ratio) - two different CAM2029 dosing regimens and one placebo group.

What is the market size for Camurus' PLD treatment?

Approximately 37,000 people in the US and EU have polycystic liver disease (PLD), with the condition primarily affecting women.

What are the side effects of Camurus' CAM2029 for PLD?

The most common side effects were diarrhea, mild to moderate gastrointestinal disturbances, and injection site reactions, with no new or unexpected safety findings.

What is the regulatory status of CAM2029 for PLD treatment?

CAM2029 has received Orphan Drug Designation from both the FDA in the US and the European Commission for treating autosomal dominant PLD. Camurus plans to discuss Phase 3 study design with regulators.
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