Thetis Pharmaceuticals Receives Orphan Drug Designation for Resolvin E1 Program in Pediatric Ulcerative Colitis

Thetis Pharmaceuticals公司获得儿童溃疡性结肠炎Resolvin E1计划孤儿药称号

2020-12-15 01:03:22 BioSpace


Thetis’ drug candidate TP-317 is a first-in-class small molecule oral therapy in development for inflammatory bowel disease RIDGEFIELD, CT – (December 14, 2020) – Thetis Pharmaceuticals (“Thetis”), a biopharmaceutical company developing novel therapies for autoimmune and inflammatory disorders, announced today that it has received U.S. Food and Drug Administration orphan drug designation for TP-317 as a small molecule, oral therapy for treatment of pediatric ulcerative colitis. “We are pleased that the FDA has granted orphan drug designation for TP-317 for treatment of pediatric ulcerative colitis, highlighting the medical need for new therapeutic options for this patient population,” said Gary Mathias, CEO of Thetis. “Based on encouraging preclinical efficacy and Phase 1 safety data, we believe that TP-317 could offer a major benefit before escalation to biologics or as adjunct to biologics.” Dr. Jeffrey Hyams, Head of Gastroenterology and Director of the IBD Center at Connecticut Children’s Hospital and a clinical advisor to Thetis, commented, “TP-317 has the potential to address a critical unmet need for safe, oral therapies for children that face a lifetime of chronic illness. I am excited to see the TP-317 program advancing toward clinical investigation.” About TP-317 The active agent in TP-317 is Resolvin E1 (RvE1), an endogenous immunoresolvent discovered by biomedical researchers at Harvard Medical School. When administered therapeutically in preclinical models of IBD, RvE1 activates multiple pathways to resolve inflammation and promote tissue repair without compromising the host’s ability to fight infection, indicating a unique pharmacological profile compared to approved and other investigational IBD drugs. In addition, Phase 1 studies indicate that RvE1 is safe and well tolerated in healthy volunteers, providing support that TP-317 could offer a favorable safety profile compared to immunosuppressive agents such as biologics and JAK inhibitors. About Ulcerative Colitis Ulcerative colitis is a chronic inflammatory bowel disease that mainly affects the large intestine. Symptoms typically include diarrhea mixed with blood, abdominal pain, increased bowel movements and in severe cases, weight loss and fatigue. Children can also develop complications related to growth, development, nutrition, pubertal maturation, bone mineral density accretion and psychological impacts. Although ulcerative colitis can present at any age, it typically begins in teenagers and young adults. Various studies estimate that there are between 63,000 and 110,000 cases of pediatric ulcerative colitis in the United States, with the majority of these occurring in children aged 10 to 17. Currently available medications alleviate inflammation and reduce symptoms, but do not provide a cure or prevent long-term complications, and some have serious adverse effects, including increased risk of severe infection and malignancy. As a result, there continues to be a high unmet medical need for additional oral agents that are safe and effective for the induction and maintenance of remission of ulcerative colitis and Crohn’s disease. About Thetis Pharmaceuticals Thetis is a biopharmaceutical company dedicated to improving the lives of patients suffering from inflammatory diseases. Thetis’ proprietary HEALER™ technology platform enables the pharmaceutical development of Resolvins, a class of endogenous lipid mediators that promote inflammation resolution and tissue repair. Thetis’ HEALER™ technology overcomes the stability, manufacturing, and formulation hurdles that have limited the development of Resolvins as pharmaceutical agents, unlocking their robust pharmacology to be developed as first-in-class drugs. For more information, please visit Thetis Pharmaceuticals’ website ( and follow Thetis on Twitter (@thetispharma). Contact Information Aaron Mathias Director of Business Development
Thetis公司的候选药物TP-317是一种正在开发中的用于炎症性肠病的一流小分子口服疗法 美国康涅狄格州里奇菲尔德-(2020年12月14号)-Thetis Pharmaceuticals(“Thetis”),一家为自身免疫性和炎症性疾病开发新疗法的生物制药公司,今天宣布,它已经获得美国食品和药物管理局的孤儿药命名,将TP-317作为一种小分子口服疗法用于治疗儿童溃疡性结肠炎。 Thetis公司首席执行官加里·马蒂亚斯说:“我们很高兴FDA批准TP-317为孤儿药,用于治疗儿童溃疡性结肠炎,这突出了对这一患者群体新的治疗选择的医学需要。基于令人鼓舞的临床前疗效和1期安全性数据,我们相信TP-317在升级为生物制剂或作为生物制剂的辅助制剂之前可以提供重大的益处。“ 康涅狄格州儿童医院胃肠病学主任兼IBD中心主任,Thetis公司临床顾问Jeffrey Hyams博士评论说:“TP-317有潜力解决那些面临终生慢性疾病的儿童对安全口服疗法的迫切需求。我很高兴看到TP-317项目向临床研究迈进。“ 关于TP-317 TP-317中的活性剂是Resolvin E1(RvE1),一种由哈佛医学院生物医学研究人员发现的内源性免疫Resolvin。当在IBD临床前模型中进行治疗性给药时,RvE1激活多种途径以解决炎症和促进组织修复,而不损害宿主抗感染的能力,这表明与批准的和其他研究性IBD药物相比具有独特的药理学特征。此外,1期研究表明,RvE1在健康志愿者中是安全的,耐受性良好,这为TP-317与生物制剂和JAK抑制剂等免疫抑制剂相比提供了有利的安全性支持。 关于溃疡性结肠炎 溃疡性结肠炎是一种主要影响大肠的慢性炎症性肠病。典型的症状包括腹泻和血液混合,腹痛,大便运动增加,在严重的情况下,体重减轻和疲劳。儿童还可出现与生长,发育,营养,青春期成熟,骨密度增高和心理影响有关的并发症。 虽然溃疡性结肠炎可以出现在任何年龄,它典型的开始于青少年和年轻的成年人。各种研究估计,美国有63000到11万儿童溃疡性结肠炎病例,其中大多数发生在10到17岁的儿童。 目前可用的药物可减轻炎症和减轻症状,但不能提供治疗或预防长期并发症,有些药物具有严重的不良影响,包括增加严重感染和恶性肿瘤的风险。因此,对于安全有效的用于诱导和维持溃疡性结肠炎和克罗恩病缓解的额外口服制剂,仍然存在着高度未满足的医疗需求。 Thetis制药公司简介 Thetis是一家生物制药公司,致力于改善患有炎症性疾病患者的生活。Thetis公司的专有Healer™技术平台使Resolvins(一类促进炎症消解和组织修复的内源性脂质介质)的药物开发成为可能。Thetis公司的Healer™技术克服了稳定性,制造和配方方面的障碍,这些障碍限制了Resolvins作为药物制剂的开发,使其强大的药理得以开发成为一流的药物。 欲了解更多信息,请访问Thetis制药公司的网站(,并在Twitter上关注Thetis(@thetispharma)。 联系方式 阿伦·马蒂亚斯 业务发展总监 电子邮件