Camurus AB

The FDA handed a CRL to the Swedish biotech Camurus for its hormone disorder therapy over “facility-related deficiencies” at a third-party manufacturer. The therapy, dubbed CAM2029, had a PDUFA date of Oct. 21 for acromegaly, but production issues were found at a third-party factory in September. There were no issues with the candidate’s efficacy or safety, Camurus said in a Tuesday release. CAM2029 does not have a new decision date, but Camurus said “timely resolution of the CRL” will depend on the unnamed third-party manufacturer’s responses. Labeling discussions for the asset were already in the final stages when the CRL was given, the company said. Acromegaly occurs when the body makes too much growth hormone, which leads to abnormally large hands and feet. Subcutaneously administered CAM2029 features a peptide inhibitor of growth hormones called octreotide. Camurus announced data from a pair of Phase 3 trials investigating CAM2029 in June and July 2023, showing acromegaly symptoms improved in patients. In May, Camurus said the EMA had received its regulatory application. Jefferies analysts said the delay could lead to Camurus launching its drug later than expected. Crinetics Pharmaceuticals has its own acromegaly candidate and announced the submission of its FDA application last month. CAM2029 is also being investigated for gastroenteropancreatic neuroendocrine tumors and polycystic liver disease.

Lund, Sweden — 22 October 2024 — Camurus (NASDAQ STO: CAMX) today announced that the U.S. Food and Drug Administration (FDA) has issued a complete response letter (CRL) regarding the new drug application (NDA) for CAM2029 (octreotide) extended-release injection for the treatment of patients with acromegaly. The CRL is attributed to facility-related deficiencies identified during a Current Good Manufacturing Practices (cGMP) inspection of a third-party manufacturer completed in September 2024. FDA has communicated that its determination of responses from the facility as satisfactory is needed for a timely resolution of the CRL and approval of the NDA. The CRL did not state any other concerns, including related to clinical efficacy or safety of CAM2029. “The CRL is disappointing, however, we are confident in the data supporting our NDA and the potential of CAM2029 to address unmet medical needs of patients with acromegaly”, says Fredrik Tiberg, Camurus’ President & CEO. “Camurus is committed to working with the FDA and the third-party manufacturer to bring CAM2029 to patients living with acromegaly as soon as possible.” Labelling discussions with the FDA were held during the final stage of the NDA review and the prescribing information for CAM2029 is well advanced. In parallel with the US NDA process, a Market Authorization Application for CAM2029 for the treatment of acromegaly is under review in the EU. In addition, two development programs for CAM2029 for treatment of gastroenteropancreatic neuroendocrine tumors and polycystic liver disease are progressing. The CRL does not impact on the advancement of these programs. 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 acromegaly Acromegaly is a rare, slowly progressive disease, typically caused by a tumor of the pituitary gland producing excess growth hormone and stimulating increased insulin growth factor-1 (IGF-1) levels. This results in abnormal growth of bone and tissue, enlarged hands, feet, facial features and inner organs, and symptoms such as fatigue, joint pain, headache, visual field defects, excessive sweating, and paresthesia.1 Inadequate biochemical and symptom control can have detrimental impacts on quality of life and mortality of patients with acromegaly.2,3 The prevalence of acromegaly is estimated to about 60 cases per million.4 About CAM2029 Octreotide SC depot, CAM2029, is an investigational, ready-to-use octreotide for subcutaneous administration under development for the treatment of acromegaly, as well as gastroentero-pancreatic neuroendocrine tumors (GEP-NET), and polycystic liver disease (PLD). CAM2029 is designed for enhanced octreotide exposure and convenient, once-monthly administration with a prefilled pen to facilitate easy self-administration by patients. The CAM2029 clinical program for acromegaly comprises of seven clinical trials, including four Phase 1 studies, one Phase 2 study, and two Phase 3 studies within the ACROINNOVA clinical program. CAM2029 has demonstrated an approximate five-fold higher bioavailability compared to the currently approved, long-acting, intramuscular (IM) octreotide.5 In the Phase 3 ACROINNOVA program, CAM2029 showed superior biochemical control compared to placebo as well as improvements in symptom control, treatment satisfaction, and quality of life compared to SoC at baseline with first-generation somatostatin receptor ligands (SRLs), octreotide and lanreotide. The safety profile of CAM2029 was consistent with SoC with no new findings.6,7 CAM2029 has received orphan drug designation for acromegaly (EU) and for polycystic liver disease (EU and US). About Camurus Camurus is a Swedish, science-led biopharmaceutical company committed to developing and commercializing innovative, long-acting medicines for the treatment of severe and chronic conditions. New drug products with best-in-class potential are conceived based on the company’s proprietary FluidCrystal® drug delivery technologies and its extensive R&D expertise. Camurus’ clinical pipeline includes products for the treatment of dependence, pain, cancer and endocrine diseases, which are developed in-house and in collaboration with international pharmaceutical companies. The company’s shares are listed on Nasdaq Stockholm under the ticker CAMX. For more information, visit www.camurus.com. References Colao A., et al. Acromegaly. Nat Rev Dis Primers. 2019;5(1):20. Webb SM, et al. Quality of Life in Acromegaly. Neuroendocrinology. 2016;103(1):106-111. Fleseriu M, et al Acromegaly: pathogenesis, diagnosis, and management. Lancet Diabetes Endocrinol. 2022 Nov;10(11):804-826. Crisafulli S., et al. Global epidemiology of acromegaly: a systematic review and meta-analysis. Eur J Endocrinology. 2021; 185:251-63. Prescribing Information SANDOSTATIN® LAR, https://www.accessdata.fda.gov/drugsatfda_docs/label/2024/021008Orig1s047Corrected_lbl.pdf Ferone, D., et al. Octreotide subcutaneous depot for acromegaly: A randomized, double-blind, placebo-controlled phase 3 trial, ACROINNOVA 1. J Clin Endocrinol Metab. Published 8 October, 2024. https://doi.org/10.1210/clinem/dgae707 Press release 15 July, 2024: https://www.camurus.com/media/press-releases/2024/camurus-announces-positive-phase-3-results-from-the-acroinnova-2-study-of-octreotide-sc-depot-cam2029-in-acromegaly-patients/ 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 1:00 am CET on 22 October 2024.

iStock, hapabapa The regulator cited deficiencies at a third-party manufacturing facility. Camurus is seeking approval for its extended-release subcutaneous formulation of octreotide, which would allow more convenient once-monthly dosing for patients with acromegaly. The FDA has rejected Camurus’ investigational subcutaneous formulation of octreotide, dubbed CAM2029, for the treatment of the rare hormonal disorder acromegaly, the Swedish drugmaker announced on Tuesday. In a Complete Response Letter (CRL), the regulator cited “facility-related deficiencies” discovered last month during an inspection of a third-party manufacturer as the reason for the rejection and did not point to other issues, particularly as regards CAM2029’s clinical efficacy or safety data. The FDA needs to receive “satisfactory” responses from Camurus’ third-party provider before it can resolve the CRL, according to the company. CEO Fredrik Tiberg in a statement called the CRL “disappointing” but noted that the company remains “confident in the data supporting our [New Drug Application] and the potential of CAM2029 to address unmet medical needs of patients with acromegaly.” CRLs from the FDA rejecting drug candidates due to manufacturing issues are on the rise . Out of a total of 238 CRLs issued between 2017 and 2023, 30% involved quality/manufacturing issues —particularly problems with manufacturing processes, facility inspections, or chemical, manufacturing and controls. Camurus will work with its external manufacturer and with the FDA “to bring CAM2029 to patients living with acromegaly as soon as possible,” Tiberg said. To this end, the company said that it has already held labelling discussions with the regulator and that CAM2029’s prescribing information is “well advanced.” Afflicting around sixty patients per one million people, acromegaly is a rare and slowly progressive disease characterized by the abnormal growth of bones and enlarged hands, feet and facial features. Acromegaly also affects internal organs and can typically include symptoms such as headaches, joint pain and fatigue. Acromegaly is often caused by a tumor in the pituitary gland, which leads to the excess production of growth hormone and insulin growth factor-1. When uncontrolled, acromegaly worsens patients’ quality of life or can heighten their risk of death. Octreotide, the active ingredient of CAM2029, is a peptide drug that is well-known as an acromegaly therapy. It works by mimicking the function of somatostatin, a naturally occurring hormone, and activating its corresponding receptor. This mechanism of action helps suppress growth hormone, addressing the underlying cause of acromegaly. Octreotide also lowers the levels and activity of insulin and glucagon. The pharma industry has long taken advantage of octreotide’s therapeutic effects for acromegaly. Sun Pharmaceuticals won FDA approval for Bynfezia Pen (octreotide acetate) in 1988, which is indicated for the three-times-daily subcutaneous treatment of acromegaly. The drug is also approved for carcinoid tumors and vasoactive intestinal peptide tumors. Camurus’ investigational formulation is an extended-release version of octreotide that would allow for once-monthly dosing, giving patients a more convenient treatment option. In the Phase III ACROINNOVA 2 study, CAM2029 led to better biochemical control of acromegaly versus the current standard of care. Camurus’ injection also improved quality of life and symptom burden.