Triptorelin Pamoate

Debiopharm’s expertise in extended-release formulations could lighten the burden of treatment for children with Central Precocious Puberty (CPP) and their families by reducing injection frequency to once a year Lausanne, Switzerland – September 9th, 2024 – Debiopharm (www.debiopharm.com), a privately-owned, Swiss-based biopharmaceutical company aiming to develop innovative therapies and to improve patient quality of life, today announced the dosing of the first sentinel patients in its open-label, single-arm, multi-center Phase III study, NCT06129539 ‘A Study to Assess the Efficacy, Safety and Pharmacokinetics of Debio 4326 in Pediatric Participants Receiving Gonadotropin-Releasing Hormone Agonist Therapy for Central Precocious Puberty (LIBELULA)’. Triptorelin is an established treatment for CPP as a 1-, 3- and 6-month formulation. This new phase III study evaluating the novel 12-month triptorelin formulation is being carried out in North and South America. In pursuit of developing safe, effective, and convenient treatment options for patients, Debiopharm, is applying its expertise in extended-release formulations to achieve the first yearly injectable Gonadotropin-Releasing Hormone agonist (GnRHa). Reducing treatment burden while preserving triptorelin’s well-known efficacy and safety are key elements that Debiopharm is eager to assess through this study. “It is always disheartening to see such young children burdened by the effects of central precocious puberty and the lifelong complications it may cause. We are thankful that treatments like triptorelin are already on the market, and that companies like Debiopharm continue to invest in lightening the patient care burden with formulations that reduce injection frequency.” expressed Prof. Fernando Cassorla, Principal Investigator, Pediatrics Department at the Hospital San Borja Arriarán in Santiago, Chile. “As patients are at the center of all we do, we recognize that convenience matters. Reducing injection frequency to once a year can lower the burden for CPP patients and their caretakers by reducing children’s stress and improving overall compliance. We are enthusiastic about the first patients enrolled in this phase III trial that can bring both medical and practical advancements.” expressed Bertrand Ducrey, CEO of Debiopharm. About Central Precocious Puberty Central precocious puberty (CPP) occurs at an unusually early age, before 8 years of age in girls and before 9 years of age in boys [1-2]. It is characterized by a premature development of secondary sexual characteristics (e.g. breasts for girls and enlarged testicles for boys), accelerated growth, and bone maturation leading to reduced adult height. CPP is triggered by the early release of the gonadotropin-releasing hormone in the brain and premature activation of the hypothalamic-pituitary-gonadal axis. This early activation can be due to specific genetic alterations, central nervous system lesions, and social stressors but frequently has no identified etiology [3]. The approximative prevalence of CPP is 1 in 5,000-10,000 among Caucasians, more frequent in girls than in boys globally [4]. Precocious puberty may be associated with psychosocial difficulties and carries potential negative implications for long-term health including increased risk of metabolic complications, such as type 2 diabetes, weight gain, obesity, cardiovascular disease, as well as depression, and even premature death [5-10]. Early puberty has also been associated with an increased risk of breast cancer in women. In men, it may increase the risk of prostate cancer [11-13]. Since the early 1980s, Gonadotropin-Releasing Hormone agonists (GnRHa) such as triptorelin have been the standard of care for the treatment of CPP [14-16]. Treatment aims to preserve adult height and prevent social and psychological difficulties and the various potential consequences on long-term health. Currently, there are several different extended-release GnRHa formulations ranging from monthly injections to subcutaneous implants for annual use [17]. While the latter may have a longer duration of action, it requires yearly surgical positioning. About Debio 4326 Debio 4326 is a unique injectable, biodegradable 12-month extended-release formulation of triptorelin designed to further reduce the frequency of injections and burden of administrations, particularly considering its intended use in a pediatric population. Based on favorable efficacy and safety data with the different triptorelin 1-, 3- and 6-month formulations, Debio 4326 aims to preserve efficacy while providing increased comfort, ensure long-term compliance, and reduce stress for children and their parents. Debiopharm’s commitment to patients Debiopharm aims to develop innovative therapies that target high unmet medical needs primarily in oncology and bacterial infections. Bridging the gap between disruptive discovery products and real-world patient reach, we identify high-potential compounds and technologies for in-licensing, clinically demonstrate their safety and efficacy, and then hand stewardship to large pharmaceutical commercialization partners to maximize patient access globally. For more information, please visit www.debiopharm.com We are on X. Follow us @DebiopharmNews at http://twitter.com/DebiopharmNews Sources [1] Latronico AC, et al. Lancet Diabetes Endocrinol. 2016;4(3):265–274. [PubMed: 26852255] [2] Sanctis V, et al. Acta biomedica: Atenei Parmensis. 2019 90 (3): 345-359. [3] Maione L, et al. Clin Endocrinol (Oxf). 2021 Oct;95(4):542-555. doi: 10.1111/cen.14475. Epub 2021 Apr 20. PMID: 33797780; PMCID: PMC8586890 [4] National Organization for Rare Disorders (NORD) 2022 [5] Day FR, et al. 2015. Sci Rep, 5: 11208. [6] Elks CE, et al. 2013. Diabetes Care, 36 (11): 3526-34. [7] Prentice P, and Viner RM. 2013. Int J Obes (Lond), 37 (8): 1036-43 [8] Ong KK, et al. 2012. J Clin Endocrinol Metab, 97 (8): 2844-52. [9] Hamlat EJ, et al. 2014. J Abnorm Child Psychol, 42 (4): 527-38. [10] Charalampopoulos D, et al. 2014. Am J Epidemiol, 180 (1): 29-40. [11] Day FR, et al. 2017. Nat Genet, 49 (6): 834-41. [12] Bonilla C, et al. 2016. BMC Med, 14: 66. [13] Bräuner EV, et al. 2020. JAMA Netw Open, 3 (10): e2015665 [14] Carel JC, et al. 2009. Pediatrics, 123 (4): e752-62. [15] Bertelloni S, et al. 2015. Horm Res Paediatr, 84 (6): 396-400. [16] Bangalore Krishna K, et al. 2019. Horm Res Paediatr, 91 (6): 357-72. [17] Aguirre RS, Eugster EA. Best Pract Res Clin Endocrinol Metab. 2018;32(4):343–354.

TAIPEI, Aug. 9, 2024 /PRNewswire/ -- Foresee Pharmaceuticals (TPEx: 6576), ("Foresee") today announced several new developments related to its SIF long-acting injectable (LAI) technologies, namely the disclosure of its four novel and proprietary SIF LAI delivery systems: SIF-ADAGIOTM, SIF-LEGATOTM, SIF-DUOTM, and SIF-MEZZOTM. Continue Reading View PDF Building on the tremendous success of its first SIF-derived product CAMCEVI®, Foresee has been continuously developing and expanding its SIF technology platform as well as its industry dominance and leadership in the area of LAI's, from the technology, intellectual property and product development perspective. In view of the complexity of LAI's, requiring a remarkable depth of know-how, expertise, and experience, very few organizations have developed a LAI technology platform with the level of flexibility and adaptability that can be found in the SIF platform. In fact, Foresee has now developed four distinct SIF LAI delivery systems that will enable even greater flexibility and allow to continue to push the boundaries of innovation in complex LAI products. Foresee will be disclosing more information on these new SIF delivery systems over the upcoming months. Below is a summary: Foresee's commercial product CAMCEVI® was developed using its SIF-ADAGIOTM delivery system, based on biodegradable polymers. Foresee is also applying its SIF-ADAGIOTM system to other products in the portfolio, namely FP-014, its best-in-class ready-to-use, subcutaneous, 3 and 6-month triptorelin LAI. The SIF-LEGATOTM system is a novel combination lipid-based system that has the unique advantage of permitting very high drug loading, making it highly adaptable to various drug substances and product profiles. This SIF LAI delivery system is currently being employed for the development of several early neurology products in our portfolio. SIF-DUOTM is a solution-based system which Foresee is using for the development of some of its early psychiatry LAI programs that benefit from this unique delivery system, while our SIF-MEZZOTM delivery system remains in development and optimization phase. "We are pleased to announce these recent developments in our SIF LAI technologies," said Dr. Ben Chien, Foresee's Chairman and CEO. "While we remain very committed to the advancement of our product portfolio across both SIF and NCE programs, we continue to make significant strides in advancing the technology and intellectual property foundations on which we have built our company. A key part of our mission is to remain a leader in complex LAI products and technologies." "We are very enthusiastic about the promise of these emerging SIF delivery systems. We have proven the value of our technology and expertise through the application of our SIF-ADAGIOTM system to CAMCEVI®, a best-in-class product growing successfully in the market, now we are ready to embark in the second phase of our mission by demonstrating the commercial potential of the new delivery systems," said Dr. Yuhua Li, Foresee's Chief Technology Officer. About Foresee Pharmaceuticals Co., Ltd. Foresee is a Taiwan and US-based biopharmaceutical company listed on the Taipei Exchange (TPEx: 6576). Foresee's R&D efforts are focused in two key areas, namely its unique Stabilized Injectable Formulation (SIF) long-acting injectable technology with derived drug products targeting specialty markets and secondly, its transformative preclinical and clinical first-in-class NCE programs targeting rare and severe disease areas with high unmet needs. Foresee's product portfolio includes late and early-stage programs. CAMCEVI® 42 mg, for the treatment of advanced prostate cancer, is now approved in the U.S., Canada, EU, Taiwan and Israel and launched in the U.S. in April 2022. Additionally, U.S. and EU regulatory submissions are under preparation for CAMCEVI® 21 mg. For the second indication of CAMCEVI® 42 mg, central precocious puberty (CPP), the Casppian Phase 3 clinical study, has been initiated. FP-025, a highly selective oral MMP-12 inhibitor targeting inflammatory and fibrotic diseases, has been investigated in a Phase 2 proof-of-concept study in allergic asthmatic patients. The study has been completed with positive outcomes, with future development in rare immune-fibrotic diseases. FP-020, a follow-on oral MMP-12 inhibitor currently in Phase 1, with development targeted in severe asthma, COPD and IBD. FP-045 is a highly selective oral small molecule allosteric activator of ALDH2, a mitochondrial enzyme, for which the FuschiA Phase 1b/2 Fanconi Anemia study is currently being initiated, and the Phase 2 WINDWARD study in pulmonary hypertension-interstitial lung disease (PH-ILD) patients is in planning with targeted initiation in the fourth quarter of 2024. Building on the compelling biology of ALDH2, and translational data from several Foresee ALDH2 activators, a follow-on candidate from a new series of compounds is being selected for development in metabolic syndrome/healthy weight loss and the broader cardiovascular-renal-metabolic space. SOURCE Foresee Pharmaceuticals Co., Ltd.