Caphosol

罕见病赛道，国内布局者寡，国外却并购如潮。这不，12月12日，美国安进公司宣布以超280亿美元收购Horizon Therapeutics公司，扩展罕见病产品管线。此次参与竞标收购Horizon的企业，还有赛诺菲和强生。实际上，这只是外资药企在罕见病赛道频繁并购的“冰山一角”。基于此，罕见病赛道正从以往的“冷门”、小众，逐渐升温为热门赛道。一、全球约3亿罕见病患者，市场存在巨大商业利益由于存在患者人群少、药物研发难度大、成本高等显著特征，罕见病赛道一直是人尽皆知的“冷门”、小众赛道，并不像抗肿瘤、自身免疫等大热门赛道，吸引众多药企竞相扎堆、厮杀激烈。发病率低、患者人群少，是罕见病赛道最为显著的特征。罕见病，是指患病率极低、患者总数少的非常见疾病，例如白化病、血友病、成骨不全症、多发性硬化症、肌萎缩侧索硬化症（渐冻人）等。美国定义的罕见病为患病率低于万分之五或患者总数低于20万，但预期其治疗药品销售额难以收回研发成本的疾病。根据《中国罕见病定义研究报告2021》，将“新生儿发病率小于1/万、患病率小于1/万、患病人数小于14万的疾病”列入罕见病。从患者人数看，目前全球约有3亿罕见病患者，中国有2000多万罹患罕见病的患者，并且每年新出生的罕见病患儿达20万人。药物研发难度大、成本高，是罕见病赛道的另一个显著特征。由于发病原因复杂以及对罕见病的研究不足，导致许多罕见病“无药可医、少药可医”。据统计，目前全球已知的罕见病有7000多种（占人类总疾病的10%），其中约95%无有效治疗药物。另外，由于罕见病药物用药人群少，且药物研发成本高，药企可能没法收回成本，导致研发积极性有限。不过，虽然罕见病市场参与者寡，但同时也意味着市场存在着巨大的商业利益。由于罕见病药物（又称孤儿药）研发难度高，形成了极高的行业进入壁垒，市场竞争相对缓和，药企可通过高技术壁垒打造护城河，享受高利润。而且，罕见病市场有着极高的政策壁垒，药企可享有较高的定价权。一直以来，世界各国及地区对罕见病药物的研发十分重视，给予了许多强有力的政策支持，包括市场独占期、临床试验的税收抵免、研发资助、加快审批等。市场独占期，是阻止另一种具有相同活性部分的产品用于相同的疾病或病症，保护品牌药不受仿制药竞争的影响。例如，澳大利亚、韩国制定的市场独占期分别为5年、6年，美国为7年，欧盟、日本和新加坡的市场独占期更是长达10年。国内方面，近几年我国从研发、注册、支付等各环节对罕见病进行支持。2022年5月，国家药监局明确指出，“对批准上市的罕见病新药，在药品上市许可持有人承诺保障药品供应情况下，给予最长不超过7年的市场独占期，期间不再批准相同品种上市。”我国罕见病领域支持政策 资料来源：国家卫健委、平安证券研究所二、国内已获批超60种罕见病用药，进口产品占主导正是看中罕见病市场存在的巨大商业利益，使得外资药企不惜砸重金强势布局。例如，默沙东以115亿美元收购了罕见病新药研发公司Acceleron Pharma；赛诺菲豪掷201亿美元现金全资收购了罕见病“独角兽”健赞；阿斯利康以390亿美元收购了Alexion Pharmaceuticals（亚力兄制药）；武田制药以620亿美元收购了欧洲罕见病巨头夏尔（Shire）。除了上述百亿级并购事件以外，亿级、十亿级并购更不在少数，涉及葛兰素史克、诺和诺德等，外资药企在罕见病领域的野心和布局可见一斑。相较之下，由于我国药企在罕见病领域布局较少，导致国内市场仍以进口药物为主。而且，在罕见病药物上市数量上，我国与美国仍存在较大差距。不过，近年来我国在罕见病领域也取得了许多良好成效。国家卫健委制定的《第一批罕见病目录》，共收录了121种罕见病。目前国内共有60多种罕见病用药获批上市，其中已有40多种被纳入国家医保目录，涉及25种疾病。另外，根据东亚前海证券研报披露，2018年11月至今，国家药监局、国家卫健委陆续发布三批次临床急需境外新药名单，涵盖的药品数量分别为40个、26个、7个，共计73款。在三批名单的73款药品中，罕见病药物达到37个，占比达到50.68%。在已经获批的46个药物中，罕见病药物达到21个，占比45.65%。 从市场前景看，由于我国存在大量未被医治的罕见病患者群体，以及随着诊疗意识的提高、精准疗法的进步，我国罕见病药物市场有望实现快速增长。根据弗若斯特沙利文资料显示，中国罕见病药物市场总额从2016年的5亿美元增至2020年的13亿美元，期间复合年增长率为29.9%，远高于全球罕见病药物市场增速（5.5%），并预计2020年至2030年将以34.5%的复合年增长率增长至259亿美元，而美国和世界其他地区于同期的市场增长率分别为10.5%和10%。三、孤儿药认定，国产创新药出海的“另一条腿”虽然我国罕见病市场尚处于起步阶段，但是受巨大的商业利益和市场前景以及热门赛道同类型药物扎堆的影响，目前国内也有不少上市药企抢滩布局罕见病赛道。北海康成主要专注于开发和商业化罕见病药物和罕见肿瘤特药，于2021年底在港交所挂牌上市，被称为“罕见病第一股”，目前已打造13个创新产品管线（拥有其中7项的全球专利），包括3个已上市产品、4个处于临床阶段的候选药物、1个处于IND准备阶段、2个处于临床前阶段，另外3个基因治疗专案处于先导识别阶段。其中，在罕见病领域，拥有7种生物制剂及小分子产品及候选产品，用于治疗粘多糖贮积症II型（即亨特氏综合症）及其他溶酶体贮积病（LSDs）、A型血友病等；在罕见肿瘤领域，也布局了治疗胶质母细胞瘤的CAN008，及Caphosol®（CAN002）和Nerlynx®（CAN030）等肿瘤产品。北海康成研发管线 来源：2022年中报除了北海康成以外，国内也有不少药企通过自主研发或引进境外创新管线的方式入局罕见病赛道。例如，截至2022年中报，亚盛医药已获得两项FDA授予的快速通道资格认定与两项儿童罕见病资格认定，以及FDA与欧盟委员会（EC）授予的16项孤儿药资格认定，继续创下中国生物制药公司获得FDA孤儿药资格认定的最高记录。具体来看，亚盛医药研发的奥雷巴替尼（耐立克），已获得FDA授予急性髓系白血病（AML）、急性淋巴细胞白血病等三项孤儿药认定和一项快速通道资格认定，这也是中国首个且唯一获批上市的第三代BCR-ABL抑制剂；新型口服Bcl-2选择性抑制剂Lisaftoclax（APG-2575），已获得FDA授予的五项孤儿药认证，包括滤泡性淋巴瘤、华氏巨球蛋白血症、慢性淋巴细胞白血病、多发性骨髓瘤等；靶向MDM2-p53 PPI的小分子抑制剂Alrizomadlin (APG-115)，已获得FDA授予的六项孤儿药资格认定，包括软组织肉瘤、胃癌、急性髓系白血病、视网膜母细胞瘤、IIB-IV期黑色素瘤及神经母细胞瘤；Pelcitoclax（APG-1252）为新型高效小分子药物，可通过双重抑制Bcl-2及Bcl-xL蛋白恢复细胞凋亡以治疗小细胞肺癌（SCLC）、非小细胞肺癌、神经内分泌肿瘤、非霍奇金淋巴瘤，其中已获得FDA授予治疗SCLC的孤儿药资格认定。另外，石药集团、君实生物、康宁杰瑞等国产药企，也有不少产品获得孤儿药认定，并借此逐步打开国际化市场。例如，在国际化方面已积累许多经验的石药集团，截至2021年已有10余项药物在海外开展临床研究，取得了10项孤儿药或快速通道资格，其中SYSA1801用于治疗胰腺癌及NBL-015用于治疗胃癌（包括食道胃结合部癌）获得FDA授予孤儿药资格。君实生物也在积极推进国际化进展，目前核心产品特瑞普利单抗已获得五项FDA颁发的孤儿药资格认定，适应症包括黏膜黑色素瘤、鼻咽癌、软组织肉瘤、食管癌和小细胞肺癌。康宁杰瑞研发的KN035（恩沃利单抗），是全球首个及唯一获批上市的可皮下注射的PD-L1抑制剂，也是中国第一个针对跨瘤种适应症的免疫治疗药物、首个国产PD-L1药物，于2021年获得FDA授予分别用于治疗晚期胆道癌及软组织肉瘤的孤儿药资格。可以看到，先通过孤儿药认定打入国际化市场，再逐步拓展更多适应症的另辟蹊径“出海”模式，已经成为国产创新药出海的“另一条腿”。👇欢迎关注药渡数据媒体矩阵往期推荐

WAKEFIELD, MA / ACCESSWIRE / July 22, 2022 / Innovation Pharma (Formerly known as Cellceutix) (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, today provided an update on the Company's Brilacidin program in Oral Mucositis (OM). Brilacidin, delivered as an oral rinse, was shown in Phase 2 clinical testing (NCT02324335) to reduce incidence, delay onset and decrease duration of severe OM (WHO Grade ≥ 3) in Head and Neck Cancer (HNC) patients receiving chemoradiation. The Company and FDA have completed an End-of-Phase 2 meeting and agreed to an acceptable Phase 3 program. To assess current insurance programs and Brilacidin's overall commercialization potential in OM, the Company engaged separate consulting firms to analyze the provider and payer landscape -- specifically, the likelihood of clinicians to prescribe and insurers to reimburse a novel OM treatment, such as Brilacidin, should it eventually gain marketing approval. Results of this analysis are summarized below. Large Unmet Need: Oral Mucositis (OM) presents a significant socioeconomic cost to the healthcare system and to patients, as it can substantially impact quality of life, result in hospitalization, and lead to disruptions in anti-cancer therapy and poorer outcomes. Current standard of care (SOC) treatment options (primarily devices) are lacking because they only act as symptom management and do not decrease incidence of severe OM (SOM). Competitive Positioning: Brilacidin's oral sachet formulation was perceived as convenient and highly preferred by key opinion leaders (KOLs) in comparison to intravenous (IV) options, and KOLs are willing to prescribe Brilacidin for all HNC patients because the compound's preventative properties and ease of use as an oral rinse. Brilacidin has the potential to be a first-line product for prevention and treatment of SOM due to efficacy perceived as being highly clinically meaningful. Development Considerations: KOLs viewed the primary endpoint of reducing SOM incidence as favorable, though recommend additional secondary endpoints to promote greater uptake (i.e., reduction of opioid usage, overall pain level, and time to onset of SOM). Maximizing overall efficacy (reduction of SOM) was identified as a higher KOL priority than the specific mechanism of action (anti-inflammatory activity) by which efficacy is achieved. Coverage Perspectives: Developing Brilacidin as a New Drug Application (NDA), via the 505(b)(1) pathway, significantly improves Brilacidin's commercialization prospects in the OM therapeutic area. OM products, such as MuGard, GelClair, Episil, and Caphosol, which were developed as devices and not drugs, have struggled to show clinical benefit, as they are primarily palliative in nature, and from a government payer perspective are not covered under Medicare Part D. Reimbursement Landscape: At least 2 approved drugs in all therapeutic drug classes must be made available under Medicare Part D, per USP Medicare Model Guidelines. The guidelines establish the framework (the rules) that drive payer formularies. Given there are no approved drugs for OM in HNC, should Brilacidin be approved, it is highly likely it would be included in payer formularies as a pharmacy benefit. Commercial Opportunity: Annually, in the U.S., Brilacidin has the potential to address 52,000 OM patients in HNC, with potential future extension to 20,000 OM patients in Hematopoietic Stem Cell Transplantation (HSCT), and 160,000 OM patients in all chemotherapy categories. Based on physician estimates of Brilacidin adoption and other pricing-based assumptions, forecasts of U.S. annual sales are $188 million for OM in HNC patients in 2030, with a potential additional upside of $106 million for OM in HSCT and $598 million for OM in all chemotherapy categories. "This analysis reinforces Brilacidin's potential to become a breakthrough OM treatment and commercialization success," said Leo Ehrlich, Chief Executive Officer at Innovation Pharmaceuticals. "While our focus on Brilacidin over the last few years has been in the antiviral space, given COVID-19, we have made meaningful strides to advance Brilacidin in OM, including refinement of the oral rinse formulation in sachet form. We continue to pursue partnering opportunities so Brilacidin can be further advanced in oral mucositis in the clinic." Additional information on the Brilacidin-OM revenue opportunity based on this analysis is available on the Company's website at the link below: About Oral Mucositis Oral Mucositis (OM) is a painful and debilitating complication of chemoradiation.1 Head and Neck Cancer (HNC) patients, comprising an estimated 66,000 newly diagnosed cases in the U.S. in 20212, and an estimated 900,000 worldwide3, are at high risk of developing OM. By 2030, the global incidence of HNC cases is expected to exceed 1 million per year. Moreover, between 25 and 60 percent of cancer patients, regardless of cancer type, also will experience OM. Characterized by inflammation and ulceration, patients suffering from OM are often unable to speak and eat (requiring the insertion of a feeding tube) and are more susceptible to infections, with severe cases leading to hospitalization at increased treatment costs of up to $25,000. There currently are no approved medications for the prevention of OM in the HNC population, with only limited palliative care options available. Worldwide, the OM market was estimated to be $1.5bn in 2021.4 1 The broadening scope of oral mucositis and oral ulcerative mucosal toxicities of anticancer therapies - Elad - 2022 - CA: A Cancer Journal for Clinicians - Wiley Online Library 2 Cancer Statistics, 2021 - PubMed (nih.gov) 3 Global Cancer Observatory (iarc.fr) 4 Oral Mucositis Market Size, Epidemiology, Market Research 2032 (delveinsight.com) Alerts Sign-up for Innovation Pharmaceuticals email alerts is available at: About Innovation Pharmaceuticals Innovation Pharmaceuticals Inc. (IPIX) is a clinical stage biopharmaceutical company developing a portfolio of innovative therapies addressing multiple areas of unmet medical need, including inflammatory diseases, cancer, and infectious diseases. The Company is also active in evaluating other potential investment opportunities that can add value and diversify its portfolio. Forward-Looking Statements: This press release contains forward-looking statements made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 including, without limitation, statements concerning future product development plans, including with respect to specific indications; statements regarding the therapeutic potential and capabilities of the StingRay System; future regulatory developments; and any other statements which are other than statements of historical fact. These statements involve risks, uncertainties and assumptions that could cause actual results and experience to differ materially from anticipated results and expectations expressed in these forward-looking statements. The Company has in some cases identified forward-looking statements by using words such as "anticipates," "believes," "hopes," "estimates," "looks," "expects," "plans," "intends," "goal," "potential," "may," "suggest," and similar expressions. Among other factors that could cause actual results to differ materially from those expressed in forward-looking statements are risks related to conducting pre-clinical studies and clinical trials and seeking regulatory and licensing approvals in the United States and other jurisdictions, including without limitation that compounds and devices may not successfully complete pre-clinical or clinical testing, or be granted regulatory approval to be sold and marketed in the United States or elsewhere; prior test results may not be replicated in future studies and trials; the Company's need for, and the availability of, substantial capital in the future to fund its operations and research and development, including the amount and timing of the sale of shares of common stock under securities purchase agreements; and the Company's licensee(s) may not successfully complete pre-clinical or clinical testing and the Company will not receive milestone payments. A more complete description of these and other risk factors is included in the Company's filings with the Securities and Exchange Commission. Many of these risks, uncertainties and assumptions are beyond the Company's ability to control or predict. You should not place undue reliance on any forward-looking statements. The forward-looking statements speak only as of the information currently available to the Company on the date they are made, and the Company undertakes no obligation to release publicly the results of any revisions to any such forward-looking statements that may be made to reflect events or circumstances after the date of this press release or to reflect the occurrence of unanticipated events, except as required by applicable law or regulation. INVESTOR AND MEDIA CONTACTS Innovation Pharmaceuticals Inc. Leo Ehrlich info@ipharminc.com SOURCE: Innovation Pharmaceuticals Inc. View source version on accesswire.com: