UCB Pharma GmbH

Biogen's litifilimab "stands out as an especially strong competitor" to Benlysta and Saphnelo, according to Spherix Global Insights analysts. New biologics such as GSK's Benlysta and AstraZeneca's Saphnelo, along with off-label drugs, have been the mainstay for systemic lupus erythematosus (SLE) treatment for many years. However, the landscape may be about to change with the introduction of new medicines and indications from other approved treatments.That's according to Spherix Global Insights analysts who spoke to 101 U.S. rheumatologists about current and future treatments for SLE, a landscape they see as “poised for evolution.”There are several types of lupus, with SLE the most common. The condition is caused by an autoimmune disease in which the immune system attacks its own tissues, causing widespread inflammation and tissue damage. Organ damage can occur in up to 48% of people living with lupus, most within five years of diagnosis, with lupus flares also upping the risk of organ damage.GSK’s SLE drug Benlysta, which was approved by the FDA in 2011, has now become a blockbuster medicine, while AstraZeneca’s new challenger Saphnelo, FDA-approved in 2021, remains some way behind the market leader in Benlysta in terms of sales.Doctors have also relied on off-label use of other older medicines, including rituximab. However, Spherix found in a 2023 report, this use has begun to lose favor among rheumatologists as they turn to newer biologics.Now, Spherix has found that an experimental drug from Biogen, litifilimab, “stands out as an especially strong competitor” to Benlysta and Saphnelo, according to its report. Talking to rheumatologists, Spherix analysts said that they believe litifilimab’s latest midstage data, notably its 26% improvement in SRI-4 response (a measurement on how well a patient responds to treatment) over placebo, “demonstrates a superior treatment profile compared to existing biologics and other promising therapies.”There is also hope from new indications from already-approved drugs in the form of AbbVie’s blockbuster immunology drug Rinvoq, which is in phase 3 testing for SLE, and Bristol Myers Squibb’s Sotyktu, which presented positive data in SLE at the start of the year.Spherix found that these oral therapies “offer convenience for patients and may appeal to rheumatologists seeking alternative treatment options.”Getting patients to stay on them, however, can be a “challenge,” while black box warnings on JAKs (i.e., Rinvoq) “may prevent use in earlier lines of SLE treatment,” according to Spherix.  Despite that, rheumatologists “are most eager to see Rinvoq approved for SLE, followed by Sotyktu and litifilimab, all of which are believed to be potentially effective for skin manifestations,” the report found.There are also other investigational contenders in the form of UCB Pharma’s dapirolizumab pegol and Idorsia’s cenerimod.All new entrants must, however, be mindful of the “dominance” of Benlysta, the analysts conclude, which “remains a preferred choice” in both SLE and lupus nephritis. 

声明：因水平有限，错误不可避免，或有些信息非最及时，欢迎留言指出。本文仅作医疗健康相关药物介绍，非治疗方案推荐（若涉及）;本文不构成任何投资建议。 过去十多来，孤儿药的增长速度超过了非孤儿处方药，在全球处方药销售额中所占的份额翻了一番，从2014年的不到10%增至如今的近20%。预计孤儿药2024年的全球销售额将达到1850亿美元，到2028年将接近2700亿美元。 近日，行业知名媒体Evaluate发布的一份最新报告，预测了2028年十大最有价值的孤儿药，在去年由于一些罕见病领域的竞争加剧和《通货膨胀削减法》的实施，药品排名出现了进一步的调整。前三名分别是：强生公司的血癌药物Darzalex、Vertex公司治疗囊性纤维化的Trikafta和罗氏公司治疗A型血友病的Hemlibra。 两个国产药物进入榜单，分别为传奇生物与强生合作的CAR-T疗法Carvykti和百济神州的BTK抑制剂Brukinsa，在2028年有望创下44.06亿美元和38.35亿美元的销售额。 Brukinsa在2022年对先前接受过治疗的慢性淋巴细胞白血病和小淋巴细胞淋巴瘤患者进行的头对头试验中击败了lmbruvica，这可能是因为Brukinsa具有更强的BTK特异性。与阿斯利康的同类药物Calquence相比，新晋前十名的Brukinsa在药代动力学方面也可能具有优势。 Calquence的2028年预测销售排名在过去一年中从第五位下滑到第七位。Argenx的重症肌无力药物efgartigimod alfa (Vyvgart) 跌至第14位，预计2028年销售额为30亿美元。因为2023年UCB Pharma的两款全身型重症肌无力新药获批，分别是C5补体抑制剂Zylbrysq和新生儿Fc受体靶向抗体Rystiggo，与其分割市场。 2028 年，十大最有价值的孤儿药将合计创造超过570亿美元的收入。 1、强生：Darzalex Darzalex（达雷妥尤单抗）是一种抗CD38单克隆抗体，能结合多发性骨髓瘤细胞上的CD38并激活免疫系统，从而杀伤肿瘤细胞，主要用于多发性骨髓瘤的治疗。Darzalex也是全球首个靶向CD38的药物。 Darzalex 2023年的全球销售额为97.4亿美元，占强生全年总收入的11.4%。2024 H1该药物销售额为55.7亿美元，同比增长18.6%。根据Evaluate发布的2024年全球畅销药TOP10榜单，该药预计将在今年取得近120亿美元的营收。预计2028年销售额170.22亿美元。 强生的这种药物与硼替佐米、百时美施贵宝的来那度胺和类固醇地塞米松（VRd）的先进组合疗法显示出了良好的前景。7月30日，FDA已经批准达雷妥尤单抗皮下注射制剂（商品名：Darzalex Faspro）的补充生物制品许可申请（sBLA），用于联合硼替佐米、来那度胺和地塞米松作为适合接受自体干细胞移植（ASCT）的新诊断多发性骨髓瘤（NDMM）成人患者的诱导治疗和巩固治疗。 不过Darzalex同样面临着专利悬崖的危机，Darzalex在美国、欧洲和日本的组合物专利将于2026年3月到期。已有生物类似药蓄势待发，比如复宏汉霖的HLX15今年4月已经成功完成I期临床试验。 2、Vertex：Trikafta Vertex早在2001年开始试水囊性纤维化（CF）赛道，2019年囊性纤维化跨膜调节因子（CFTR）调节剂Trikafta（elexacaftor、tezacaftor和ivacaftor）获FDA批准上市，成为首款CF三联药物。Trikafta直接针对CFTR蛋白，与彼时市场上只能缓解症状的CF药物形成鲜明差异化优势，垄断近90%的CF市场。 一经上市，Trikafta很快就成为了重磅药，去年为Vertex创下89.45亿美元销售额。预计2028年创收87.53亿美元。 7月2日，美国FDA受理了Vertex新一代三联疗法（vanzacaftor/tezacaftor/deutivacaftor，简称vanza三联疗法）的新药申请（NDA）并将其纳入优先审评名单，审评周期从原来的10个月缩短至6个月。PDUFA日期为2025年1月2日。vanza三联疗法具备成为重磅药甚至销售额超过Trikafta的潜力。 2、罗氏：Hemlibra Hemlibra（艾美赛珠单抗）是靶向凝血因子IXa与凝血因子X的双特异性抗体，用于治疗A型血友病。Hemlibra是一种预防性治疗药物，可通过皮下注射即用溶液的方式给药。Hemlibra由中外制药有限公司开发，并由中外制药、罗氏和基因泰克在全球范围内共同开发。 2017年11月，Hemlibra获得FDA批准上市，用于预防或减少产生了VIII因子抑制物A型血友病成人和儿童患者的出血频率。2018年4月，Hemlibra被FDA扩增新适应症，用于不含VIII因子抑制物A型血友病治疗的突破性疗法认定。该双抗药物是目前唯一一款在血友病领域应用的双抗药物。 Hemlibra自上市以来，年复合增速超300%，并于2020年突破20亿美元销售大关，成为重磅品种。Hemlibra2023年销售额41.47亿瑞士法郎（约45.75亿美元），同比增长16%。2024年依然保持着增长趋势，2024上半年销售额为21.43亿瑞士法郎（约25.29亿美元）。预计2028年销售额62.03亿美元。 4、阿斯利康：Ultomiris 瑞利珠单抗是一款长效补体C5抑制剂，最早于2018年12月获FDA批准上市，用于治疗阵发性睡眠性血红蛋白尿症（PNH），商品名为Ultomiris；2021年9月，瑞利珠单抗新适应症获FDA批准，用于治疗非典型溶血性尿毒症综合征（aHUS）的成人及儿童（一个月以上）患者；2022年4月，瑞利珠单抗第3项适应症获FDA批准，用于治疗成人全身型重症肌无力（gMG）。 3月25日，阿斯利康宣布FDA已经批准Ultomiris（ravulizumab-cwvz）新适应症上市，用于治疗抗水通道蛋白4（AQP4）抗体阳性（Ab+）视神经脊髓炎（NMOSD）。新闻稿指出，这是首款也是唯一一款能够使得此类患者摆脱复发的长效C5补体抑制剂。 该批准得到了一项III期试验的数据的支持，该试验达到了其主要疗效终点，在中位73.5周内防止了所有58名接受治疗的患者的复发，而阿斯利康的其他NMOSD疗法Soliris试验的安慰剂组有20例复发。 根据2023年的数据，Ultomiris的销售额达到了29.65亿美元，2024上半年销售额18亿美元，同比增长32%。预计2028年销售额51.84亿美元。 5、强生&传奇生物：Carvykti 传奇/强生合作开发的Carvykti是一款针对BCMA的基因修饰自体T细胞免疫疗法。2017年12月，强生旗下公司杨森制药（现为强生创新制药）与传奇生物签订了全球独家许可和合作协议，以开发和商业化西达基奥仑赛。2022年2月，Carvykti获FDA批准上市，用于治疗既往至少接受过四线治疗的复发或难治性多发性骨髓瘤成人患者。 Carvykti上市首年销售额就达到1.33亿美元，2023年销售额上涨276%至5亿美元。2024 H1销售额3.43亿美元，同比增长81%。预计2028年销售额44.06亿美元。 8月27日，根据中国NMPA官网最新公示，传奇生物递交的西达基奥仑赛注射液的新药上市申请已正式获批。根据CDE优先审评公示，该产品本次获批适应症为：用于治疗既往接受过一种蛋白酶体抑制剂和一种免疫调节剂治疗后复发或难治性多发性骨髓瘤（R/R MM）成人患者，是第6 款在国内获批的CAR-T疗法。 6、Incyte&诺华：Jakafi  芦可替尼是Incyte开发的一种小分子JAK1/JAK2抑制剂，其片剂（商品名：Jakafi）于2011年12月在美国获批上市，其乳膏（商品名：Opzelura）于2021年9月在美国获批上市。片剂适用于真性红细胞增多症、骨髓纤维化和移植物抗宿主病，乳膏则适用于特应性皮炎和非节段性白癜风。 2009年11月，诺华与Incyte达成协议，获得芦可替尼在美国以外的开发和商业化权益，不过仅限于血液瘤适应症。康哲药业于2022年12月引进了芦可替尼乳膏的中国和东南亚权益。鉴于白癜风患者的临床需求巨大，芦可替尼乳膏的销售增长表现一直不错，2023年销售额已达到3.4亿美元。 Incyte公司2024年H1总收入19.25亿美元，同比增长9%；其中Incyte最重要的产品JAK1/2抑制剂芦可替尼占所有产品总销售额的90%以上，其中Jakafi销售额达12.78亿美元，同比增长1%，Opzelura销售额为2.07亿美元，同比增长52%。Incyte预计Jakafi的销售额将从2023年的25.9亿美元增长到2024年的26.9亿至27.5亿美元。Evaluate预计2028该产品销售额42.35亿美元。 7、阿斯利康：Calquence  Calquence（阿可替尼）是新一代BTK选择性抑制剂，通过与BTK共价结合抑制其活性。2016年，阿斯利康先斥资40亿美元收购Acerta Pharma 55%股权获得。 Calquence曾获得美国FDA授予优先审评资格、突破性疗法认定和孤儿药资格。2017年，获得FDA的加速批准，用于治疗二线套细胞淋巴瘤（MCL）。后被批准用于初始疗法或后续疗法治疗慢性淋巴性白血病（CLL）或小细胞淋巴瘤（SLL）成人患者。 2023年3月，中国NMPA批准阿可替尼胶囊（acalabrutinib，商品名：Calquence/康可期）首次上市。用于治疗既往至少接受过一种治疗的成人套细胞淋巴瘤（MCL）患者，2023年8月，单药适用于既往至少接受过一种治疗的成人慢性淋巴细胞白血病（CLL）/小淋巴细胞淋巴瘤（SLL）患者，第2项适应症在中国获批；2024年8月28日，中国CDE官网最新公示，阿斯利康申报的acalabrutinib（通用名：阿可替尼胶囊，商品名：康可期）上市申请已获得受理：推测此次阿可替尼申报上市的新适应症可能是一线治疗慢性淋巴细胞白血病（CLL）。 截至2023年，Calquence的全球销售额已达到25.14亿美元，2024 H1全球销售额为15.08亿美元，预计到2028年销售额将增长至40.16亿美元，反映出其在治疗B细胞恶性肿瘤领域的持续增长潜力和商业前景。 8、百济神州：Brukinsa Brukinsa（泽布替尼）是一款强效BTK抑制剂，作为单药或与其他疗法联合用药。临床上主要用于治疗成人T细胞淋巴瘤、成人急性淋巴细胞白血病或小淋巴细胞淋巴瘤等疾病。 2024年3月7日，百济神州宣布FDA加速批准Brukinsa与抗CD20单克隆抗体obinutuzumab联用，用于治疗复发或难治性（R/R）滤泡性淋巴瘤（FL）成人患者，经过两个或多个疗程后的全身治疗。Brukinsa是迄今为止唯一一种对这种恶性肿瘤显示疗效的BTK抑制剂，目前在全球同类药物中拥有最广泛的标签，包括五种肿瘤学适应症。 2024年上半年，泽布替尼在全球销售金额达到了创纪录的80.18亿元人民币，同比增速高达122.0%。去年泽布替尼全年销售金额首次突破10亿美元，已经引发了市场很大的震动，当时很多人可能都没有想到，今年仅仅用了半年时间，其销售金额就轻松突破了10亿美元大关。 这再次说明，作为“best-in-class”的BTK抑制剂，随着在全球主要市场都开始大放异彩，这款药物的上限，可能远超此前市场对其的一致预期。预测2028年该药物销售额38.35亿美元。 9、阿斯利康：Lynparza  Lynparza（olaparib，奥拉帕利）是一种口服PARP抑制剂，可破坏癌细胞的 DNA 修复，导致癌细胞死亡。该药物已被批准用于治疗卵巢癌、输卵管癌或原发性腹膜癌，以及乳腺癌、胰腺癌和前列腺癌。奥拉帕利是由英国的KuDOS制药公司开发的，后来被阿斯利康收购。2017年美国首获批，2018年中国批准进口，2019年被纳入国家医保药品目录。 2023年Lynparza销售额为28.11亿美元，2024上半年销售额为1.45亿美元，同比增长9%。预测2028年该药物销售额34.51亿美元。 其化合物专利已于今年3月到期，制剂专利在2029年10月到期，最早在国内布局奥拉帕利的仿制厂商是齐鲁制药，拿下首仿；科伦药业的奥拉帕利片为国产第2家。除此之外，山香药业、南京方生\医药科技、华东制药、石药集团以及宣泰医药等多个企业也加入到激烈的竞争中。 10、艾伯维：Venclexta Venclexta是一款“first-in-class”BCL-2抑制剂，已知BCL-2蛋白在血癌中过度表达，介导肿瘤存活和治疗抵抗。Venetoclax最初于2016年获得FDA批准，用于治疗存在17p缺失的慢性淋巴细胞白血病（CLL）。2018年美国FDA批准其扩展适应症至无论是否有17p缺失，之前已接受至少一种治疗的复发/难治CLL患者都可以使用该药物。 Venclexta近几年维持了较好的增长势头，2023年达到了22.88亿美元，同比增长13.9%。Venclexta（维奈克拉）增速稳定，上半年销售额达到12.51亿美元，同比增长16%。预测2028年该药物销售额33.44亿美元。 参考资料： [1]www.evaluate.com [2]各公司官网

CLEVELAND, April 30, 2024 (GLOBE NEWSWIRE) -- Discovery Therapeutics Caribe (DTC), a clinical-stage biotechnology company dedicated to improving the health outcomes of Americans through the development of regenerative therapies for serious and life-threatening diseases, today announced it has achieved a significant milestone reflecting progress in its clinical program. In the first quarter of 2024, DTC submitted an Investigational New Drug (IND) application to the United States Food and Drug Administration (FDA). The IND submission included a phase 3 protocol for a randomized, double-blind, placebo-controlled trial of the company’s lead product, an intralesional recombinant human Epidermal Growth Factor (rhEGF) for the treatment of complex and complicated refractory neuropathic, ischemic, and neuroischemic diabetic foot ulcers (DFU). The clinical grades of complex and complicated refractory DFUs are comparable to Wagner’s classification system grades 3 and 4; University of Texas classification system grades 2 and 3; and Wound/Ischemia/Foot Infection (WIfI) classification system stages 3 and 4. These severe ulcers are difficult to heal and carry a greater risk of amputation and death. The FDA completed its review of the IND and concluded that DTC may proceed with its proposed Phase 3 clinical investigation. DTC received a Study May Proceed (SMP) letter from the FDA on April 10, 2024. Building upon the clinical efficacy and safety data gathered from international studies, DTC management believes they are on track to initiate this pivotal U.S.-based trial of intralesional rhEGF by year-end 2024. While intralesional rhEGF, globally marketed as Heberprot-P®, is available to patients in twenty-six countries, this is the first time it will be studied in U.S. patients suffering from DFU. Dr. David Armstrong, DPM, MD, PhD, a distinguished podiatric surgeon at the University of Southern California and a globally recognized researcher in diabetic foot ulcers, emphasized the critical need for innovative therapies. “There is a pressing need for treatments that can halt the progression of diabetic foot ulcers before amputation becomes the inevitable solution. Historically, treatment options have been limited, but with the introduction of advanced therapies like intralesional rhEGF, which I have seen used effectively abroad, we are hopeful. The upcoming Phase 3 trial in the U.S. will rigorously assess its efficacy for DFU patients. This trial represents an exciting potential to shift the current paradigm and provide new hope for those who desperately need it.” Lee Weingart, Co-Founder and President of DTC commented, “We are pleased to commence the next stage of intralesional rhEGF’s global journey here in the United States. The upcoming Phase 3 clinical trial will help us to understand this biologic’s clinical profile within the FDA’s regulatory framework and build upon rhEGF’s extensive international clinical experience.” Dr. Charles Zelen, DPM FACFAS, President of the Professional Education & Research Institute (PERI) said, “Preventing the severe outcomes associated with diabetic foot ulcers is critical in saving American limbs and lives. Alarmingly, nearly half of patients who undergo DFU-related lower extremity amputation do not survive beyond five years. Among U.S. veterans, the prognosis is even more grim, where survival past two years is uncommon for patients presenting with gangrene. Particularly concerning is the disproportionate impact on our African American communities, where Medicare beneficiaries are nearly twice as likely to undergo lower limb amputation within a year of DFU diagnosis compared to their non-Hispanic White counterparts. Innovative therapeutic options for severe diabetic foot ulcers are not just necessary—they are long overdue for patients in need. PERI is committed to making these important clinical trials a reality to evaluate the efficacy and safety of new biologics like intralesional rhEGF.” About intralesional rhEGF Naturally occurring human Epidermal Growth Factor (hEGF) plays a crucial role in the body’s healing process. This well-understood protein works by activating the EGF receptor on the surface of cells, a process that can stimulate cell growth, migration, and differentiation. With respect to wound healing, the primary function of EGF is to encourage the growth of essential cells, such as those that form the skin (keratinocytes), connective tissue (fibroblasts), and blood vessels (vascular endothelial cells). Additionally, EGF aids in guiding these cells to the wound site, helps them develop into mature cells, and promotes the formation of new blood vessels—a process known as angiogenesis. Together, these actions accelerate the formation of new tissue and help wounds heal effectively. In a process developed by the Center for Genetic Engineering and Biotechnology (Spanish: Centro de Ingeniería Genética y Biotecnología [CIGB]) in Havana, Cuba, hEGF is expressed using genetically engineered yeast (Saccharomyces cerevisiae), utilizing recombinant DNA technology. Unlike currently available therapies that use growth factors applied directly to the surface of wounds, intralesional injection of rhEGF delivers the active ingredient directly in the target area, a process intended to penetrate past the chronic wound environment that can otherwise degrade and reduce the effectiveness of EGF treatments. Heberprot-P® currently has marketing authorization in 26 countries for the treatment of DFUs. During 16 years of foreign post-marketing surveillance, an estimated 426,232 DFU patients (as of December 30, 2022) have been administered Heberprot-P® worldwide since its initial marketing authorization in the Republic of Cuba in June 2006. In the international markets where Heberprot-P® is commercially available, the drug is an important option in the therapeutic armamentarium for patients with this serious condition. About diabetic foot ulcers DFUs are a severe and common complication of diabetes, marked by wounds that do not heal properly or quickly. These ulcers significantly contribute to morbidity and are a major reason for hospitalizations, lower limb amputations, and even death among the more than 37 million Americans living with diabetes, both diagnosed and undiagnosed. It is estimated that up to 34% of individuals with diabetes will develop a DFU in their lifetime. Annually, the United States sees approximately 1.6 million new cases of DFUs. Of these, around 154,000 patients with non-healing ulcers undergo amputation each year, a number that has been on the rise. About Discovery Therapeutics Caribe Discovery Therapeutics Caribe (DTC) is a clinical stage biotechnology company developing rhEGF for marketing in the United States under an exclusive collaboration agreement. DTC is an Ohio-based biotechnology company following the BLA licensure pathway under the provisions of 351(a) of the Public Health Service (PHS) Act. DTC has also executed an exclusive license agreement to market Heberprot-P® in the United States and other territories. Forward-Looking Statements This press release contains forward-looking statements. Any statements contained herein that do not describe historical facts are considered forward-looking statements. Words such as “believe,” “anticipate,” “plan,” “expect,” “will,” “may,” “intend,” “estimate,” “potential,” and similar expressions are designed to identify such forward-looking statements. These statements include, without limitation, discussions regarding our future operations, business strategies, research and development efforts; the ongoing approval of intralesional rhEGF, which may hinge on the verification and delineation of clinical benefits in confirmatory trials; the anticipated advantages and potential risks associated with rhEGF; and our strategic plans and milestones, notably our aim to expediently proceed with a Biologics License Application (BLA) submission following the anticipated completion of the Phase 3 trial. Actual outcomes may significantly differ from those projected in these forward-looking statements due to a variety of crucial factors and uncertainties, including, but not limited to, the timing and results of our Phase 3 clinical trial; the process and outcomes of a BLA submission to the FDA; challenges in patient enrollment for clinical trials; the results of future discussions and inspections by the FDA or other regulatory bodies, pertaining to both clinical and preclinical programs; the capacity to secure necessary regulatory endorsements; the effects of shifts in financial markets, global economic conditions, or U.S. foreign policy; and the complexities tied to data analysis and reporting. Discovery Therapeutics Caribe expressly disclaims any obligation to update or revise these forward-looking statements to reflect any change in expectations or events, conditions, or circumstances on which any such statements may be based, except as required by federal securities laws. Media Contact Lee WeingartPresidentmedia@discothera.com Investor Contact Jason M. SmithSenior Vice President, Program Management & Business Developmentinvest@discothera.com For more information, visit https://www.discothera.com/