Ozanimod Hydrochloride

Trinity Life Sciences最近发布了一份报告，对 2023 年新药上市情况进行了分析，结果显示，针对未满足临床需求疾病的药物以及由具有复杂商业运营经验丰富的公司推出的药物最有可能在上市第一年销售超出预期。 高度未满足临床需求药物 在高度未满足临床需求领域，无论将产品推向市场的公司类型/规模如何，也无论市场类型/规模如何，在未得到满足的高需求领域推出的产品在第一年都取得成功的机会都是最大的。 这些上市产品包括对没有现有治疗方法的疾病具有强大临床价值的产品，如RSV疫苗、弗里德里希共济失调症、地理萎缩症、雷特综合症和睑缘炎。特别是对于罕见病公司来说，通过早期发现/识别患者和快速获得治疗机会进行早期市场开发工作，可开辟一条成功之路。 如三项成功的RSV疫苗上市，每一项都大大超出了预期。这些疫苗包括葛兰素史克的Arexvy、辉瑞的Abrysvo、赛诺菲和阿斯利康的Beyfortus。GSK的Arexvy超越了2023年推出的所有新产品，销售额达到15亿美元，大大超出了分析师的一致预测。 高度未满足临床需求药物商业化最成功的的案例还有Reata的治疗罕见退行性疾病弗里德赖希共济失调(FA)药物Skyclarys，Tarsus Pharmaceuticals首次推出蠕形螨性睑缘炎眼病药物Xdemvy、Apellis推出地理萎缩(GA)治疗药物Syfovre和IvericBio推出GA治疗药物Izervay。 在更成熟或更复杂市场中的上市经验和良好记录 对于在成熟市场或竞争更激烈的市场推出的产品，在治疗领域拥有丰富经验的“老手”比首次推出药物的“新手”表现更好。这种趋势在肿瘤领域药物的上市中表现得尤为明显。 礼来推出Jaypirca就是一个很好的例子，该药物最初被批准用于治疗套细胞淋巴瘤，随后获得了用于治疗慢性淋巴细胞白血病和小淋巴细胞淋巴瘤。 Genmab和艾伯维针对弥漫性大B细胞淋巴瘤患者推出的Epkinly表现也超出了预期。 另一方面，有三个肿瘤药物的例子是由经验不足的公司带来的，表现不佳。它们是Gamida的细胞疗法Omisirge、BioLineRx的多发性骨髓瘤治疗药物Aphexda和Coherus/Junshi的头颈癌药物Loqtorzi。 尽管不是在肿瘤学领域，Sarepta利用其治疗领域的专业知识推出新产品杜氏肌营养不良症(DMD)基因疗法Elevidys，这是该公司自2016年以来向市场推出的第四种DMD治疗药物。虽然近年来基因疗法的上市普遍举步维艰，但Elevidys仍能获得市场认可。 值得注意的是，首次（或第二次）推出肿瘤药物的公司（如Gamida、BioLineRx、Coherus）在上市第一年的市场表现较为有限。除了商业上的复杂性，新兴肿瘤公司还经常遇到生产/供应方面的挑战。如果尽快解决这些问题，起步缓慢也有可能获得成功。如ZEPOSIA®(BMS)在2020年缓慢起步后，ZEPOSIA稳步增长，2022年的年同比销售额增长了79%，2023年又增长了83%。 参考资料：Trinity Life Sciences 本文由「新药前沿」微信公众号根据公开资料整理编辑，欢迎个人转发至朋友圈。媒体或机构转载授权请在「新药前沿」微信公众号留言公众号名称，审核通过后开通白名单获取转载授权，转载请标识来源。 免责声明：本文仅作信息交流之目的，非投资建议或者治疗方案推荐，「新药前沿」微信公众号不对任何主体因使用本文内容而导致的任何损失承担责任。 限于作者水平和专业知识所限，如有谬误，欢迎指正！

SAN DIEGO--( BUSINESS WIRE )-- Orphagen Pharmaceuticals, Inc. , a biotech company pioneering the screening, discovery, and development of small molecule ligands that modulate orphan or unexplored members of the nuclear receptor family, today announced an award of up to $1.7 million from the National Institute of Diabetes and Digestive and Kidney Diseases (NIDDK) through the Small Business Innovation Research (SBIR) program. The funding will support the preclinical development of the small molecule OR-812, a selective nuclear receptor antagonist, as a novel oral therapeutic for treatment of ulcerative colitis and Crohn’s disease, two forms of inflammatory bowel disease (IBD). “This award recognizes the innovative preclinical work that Orphagen has done to characterize retinoic acid receptor alpha (RARα) as a target for inhibiting autoimmune inflammation of the intestinal mucosa,” said Scott Thacher, Ph.D., CEO of Orphagen. “We’ll use this funding from the NIDDK to further evaluate the properties of our lead compound, OR-812, a potent and selective RARα antagonist with promising preclinical efficacy, safety, and pharmacokinetics for the treatment of IBD.” “Innovative therapeutics for autoimmune diseases of the intestinal mucosa have largely been dominated by injectable drugs. Our small molecule, OR-812, has the potential to offer IBD patients a novel oral medicine with a differentiated mechanism of action from current therapies,” added Dr. Thacher. Up to two million Americans suffer from inflammatory bowel disease (IBD), a lifelong, debilitating inflammatory condition of the gut in which modulation of the immune system is the major therapeutic strategy. However, despite development of novel therapies including monoclonal antibodies to TNFα and IL-12/IL-23, and inhibitors of T cell gut homing such as vedolizumab and ozanimod, less than half of patients experience a sustained therapeutic benefit. The research leading to this award grows out of investigations at Orphagen into unexplored members of the nuclear receptor family, which has been the source of major drugs in cancer, metabolic disease, and inflammation. One of these unexplored targets, RARα, has a central role in the intestinal immune response and regulates the induction of homing markers that drive activated T cells to migrate to the intestinal mucosa. In vivo pharmacological studies at Orphagen have shown that RARα antagonists will, with very high potency, block the induction of the gut homing integrin α4β7, the target for vedolizumab, a major inflammatory bowel disease drug. Induction of a second important gut homing receptor, CCR9, is also blocked by OR-812 during T cell activation. Potential for efficacy in this drug class was supported by studies carried out in the laboratory of Professor Casey Weaver, the Leonard H. Robinson Endowed Chair in Pathology at the University of Alabama, Birmingham, a noted pioneer in mucosal immunology. His group showed that an RARα antagonist will inhibit α4β7 expression and cause a significant reduction in accumulation of inflammatory T cells in the lamina propria of the colon of Citrobacter rodentium -infected mice, a model of gut mucosal inflammation resembling IBD. Separately, OR-812 markedly suppressed gut mucosal inflammation and erosion in a T cell transfer model of colitis in mouse, which is known to respond to most major classes of drug for IBD. Preliminary safety studies carried out at Orphagen are consistent with a robust safety margin for OR-812. Dr. Weaver commented, “Globally, there is a pressing need for new therapies as the incidence of IBD continues to increase. Selectively blocking the action of retinoic acid through RARα is clearly one promising approach.” About Crohn’s Disease and Ulcerative Colitis Inflammatory bowel disease (IBD) is an umbrella term used to describe disorders, including Crohn’s disease (CD) and ulcerative colitis (UC), that involve chronic inflammation of the gastrointestinal tract. UC is an idiopathic, chronic disorder of the large intestine mucosa, characterized by a continuous stretch of inflammation in the innermost lining of the colon wall. Half of UC patients suffer debilitating disease flares 4-5 times a year, significantly affecting their quality of life. Patients may recover after a single attack, while others require frequent hospitalizations and surgery. CD differs from UC in that it can affect any part of the intestinal tract, and it occurs in all layers of the intestinal wall within localized regions of inflammation scattered throughout the GI tract. About Orphagen Pharmaceuticals Orphagen unlocks the power of orphan nuclear receptors. Our scientists have discovered small molecule ligands to these largely unexplored drug targets. Starting with an agnostic approach to therapeutic area, we explore proprietary lead molecules with potential in autoimmune disease and oncology. Orphagen successfully partnered its first program for ROR-gamma antagonists in autoimmune disease with a mid-size pharmaceutical company ahead of all competitors in the field. Funding from our partnerships and other non-dilutive sources, including federal grants, has allowed Orphagen to advance its proprietary first-in-class drug discovery programs, including OR-812 for inflammatory bowel disease. For more info, visit www.orphagen.com .