Rare Thyroid Therapeutics International AB

▲速抢免费参会名额 | 大湾区(广州)生物医药创新者峰会 导语 根据公开资料不完全统计，药融云为大家检测到了2021年2月16日至2021年2月22日的全球新药重大研发进展40条。其中涉及药物上市2条，药物提交上市申请4条，临床重大进展33条，2款药物研究终止。 9款治疗“新型肺炎冠状病毒感染(COVID-19)”的药物研发阶段发生重大进展，康希诺和拜耳提交上市申请。根据《疫苗管理法》、《药品管理法》相关规定，新冠疫苗按照药品特别审批程序，进行应急审评审批，而且新冠疫苗的研发过程中企业就与药审中心进行滚动资料提交，因此从递交上市申请到获批上市用时非常短，预计康希诺Ad5-nCoV近期也会加快获批上市。 康希诺的新冠疫苗上市申请获受理，康希诺生物B盘中大涨逾10%。                                  数据来源：东方财富网 表1  40款药物基本信息 数据来源：药融云全球药物研发数据库 据统计，40款药物的治疗领域主要是感染、肿瘤等。 据统计，40款药物的适应症主要是新型肺炎冠状病毒感染(COVID-19)。 表2  40款药物适应症(TOP10) 数据来源：药融云全球药物研发数据库  上市药物  生物药evinacumab（依维苏单抗）和rVSV-EBOV获批上市。 01 evinacumab（依维苏单抗） 近日，FDA批准再生元研发的evinacumab上市，用于治疗纯合子家族性高胆固醇血症（HoFH）。人源化单克隆抗体evinacumab是一种血管生成素相关蛋白-3（ANGPTL3）抑制剂，曾获“突破性疗法（2017年）、快速通道、孤儿药、儿科用药研究计划、优先审评（2020年）”资格认定。 evinacumab之所以可以降低HoFH患者的胆固醇含量，是因为它主要靶向ANGPTL3，可以有效抑制蛋白脂肪酶和内皮质酶，并且可以在脂蛋白的代谢中起到关键作用。由于evinacumab的独特靶点和作用机制，再生元还正在进行evinacumab 治疗“顽固型高胆固醇血症”和“重度高甘油三酯血症”的二期临床试验。 evinacumab的获批是基于一项评估evinacumab治疗HoFH的疗效性和安全性试验。此项试验，65例HoFH患者被随机分为两组：evinacumab组（43例）和安慰剂组（22例）。在基线时，evinacumab组和安慰剂组患者的基线平均低密度脂蛋白胆固醇（LDL-C）水平相似，分别为259.5mg/dl和246.5 mg/dl。evinacumab组患者每4周静脉注射evinacumab 15mg/kg，为期24周。主要终点为基线至24周时LDL-C的降低。研究显示，在第24周时，evinacumab组患者的LDL-C水平降低了47.1%，安慰剂组则增加了1.9%（LS平均差异为-49.0%，P＜0.0001）。 evinacumab的获批对于HoFH患者是一个分水岭，为HoFH患者提供了新的治疗选择。根据药融云全球药物研发数据库预测，evinacumab未来5年的销售额有可能会逐年增加，预计今年的销售额可达到0.12亿万美元，2025年或可达到1亿美元。 02 rVSV-EBOV 近日，默沙东研发的治疗“埃博拉病毒传染”的糖蛋白rVSV-EBOV在多个国家/地区上市，曾获“加速批准、突破性疗法、优先审评、EMA优先药物”。rVSV-EBOV最初由加拿大微生物实验室公共卫生署研发，之后授予默沙东研发和市场化权利。 根据药融云全球药物研发数据库预测，rVSV-EBOV未来5年的销售额有可能会逐年增加，预计今年的销售额可达到0.3亿万美元，2025年或可达到1亿美元。  提交上市申请的药物  Ad5-nCoV、omalizumab biosimilar（奥马珠单抗类似药）、SARS-CoV-2 mRNA vaccine和ARI-0001提交上市申请。 01 Ad5-nCoV 康希诺生物与中国人民解放军军事医学科学院生物工程研究所合作研发的COVID19刺突糖蛋白调节剂Ad5-nCoV提交上市申请，用于治疗“新型肺炎冠状病毒感染(COVID-19)”，曾获“紧急使用授权”资格。 Ad5-nCoV的Ⅲ期临床试验结果显示：在单针接种疫苗28天后，疫苗对所有症状的总体保护效力为65.28%；在单针接种疫苗14天后，疫苗对所有症状总体保护效力为68.83%。疫苗对重症的保护效力分别为：单针接种疫苗28天后为90.07%；单针接种疫苗14天后为95.47%。康希诺生物新冠疫苗接种一针即可提供保护力。储运条件也相对便捷，从目前的研究结果来看，在2-8℃ 条件下可稳定保存至少3个月，在25℃条件下可稳定保存至少2个月，在37℃ 条件下可稳定保存至少3周。 目前，国内已经有两款疫苗获批附条件上市，均为灭活疫苗，分别来自国药集团中国生物和北京科兴。如果本次康希诺生物最终顺利获批，则意味着将成为国内获得附条件上市的第三款疫苗，同时也是国内获批的首款腺病毒载体新冠疫苗。 02 omalizumab biosimilar（奥马珠单抗类似药） 人源化单克隆抗体奥马珠单抗类似药是一款治疗哮喘的免疫球蛋白E拮抗剂。 03 SARS-CoV-2 mRNA vaccine 拜耳研发的SARS-CoV-2 mRNA vaccine 是一款治疗新型肺炎冠状病毒感染(COVID-19)和SARS病毒感染的COVID19刺突糖蛋白调节剂。 04 ARI-0001 B淋巴细胞抗原CD19调节剂ARI-0001是一款治疗“急性淋巴细胞白血病、慢性淋巴细胞白血病、非霍奇金淋巴瘤”的嵌合抗原受体T细胞疗法，此次提交上市申请的适应症是“急性淋巴细胞白血病”。  进入临床三期的药物  glofitamab、upamostat、Mim-8、tiratricol（替拉曲考）和posoleucel进入临床三期。 01 glofitamab 罗氏研发的glofitamab是双特异性T细胞衔接子抗体，用于治疗“B淋巴细胞瘤、弥漫性大B细胞淋巴瘤、滤泡中心淋巴瘤、多发性骨髓瘤、非霍奇金淋巴瘤”。此次进入临床三期的适应症是“弥漫性大B细胞淋巴瘤”。 glofitamab是靶向CD20和CD3的双特异性抗体，靶向B细胞表面的CD20和T细胞表面的CD3。其中CD20位点使用的是二代人源化CD20单抗奥妥珠单抗的位点。基于一种新颖的结构形式，称之为“ 2：1”， 指的是具有两个与CD20结合的“ Fab”区域和一个与CD3结合的“ Fab”区域。这种结构的优势在于glofitamab能够与B细胞表面CD20的结合性更高。即使肿瘤细胞表达CD20含量较低，glofitamab也可以找到肿瘤细胞发挥作用。CD20和CD3首尾相连的结构也可以诱导快速T细胞活化、增殖、细胞因子释放，导致靶细胞裂解。而且“ 2：1”的结构形式也能够使glofitamab与其他抗CD20抗体联用，为后续研发联合治疗方案提供可能性。 02 upamostat RedHill Biopharma Ltd研发的upamostat是一种新型且有效的胰蛋白酶、丝氨酸蛋白酶抑制剂，靶向参与为病毒侵入目标细胞准备刺突蛋白的人类细胞因子，有望有效对抗新出现的带刺突蛋白突变的病毒变种，用于治疗“乳腺肿瘤、新型肺炎冠状病毒感染(COVID-19)、胃肠道炎症、胃肠道肿瘤、胰腺肿瘤”，曾获“孤儿药”资格认定。此次进入临床三期的适应症是“新型肺炎冠状病毒感染(COVID-19)”。 近日，RedHill Biopharma Ltd宣布，其研发的口服胶囊upamostat在美国启动三期临床试验，该实验旨在评估upamostat治疗轻症新冠肺炎患者的疗效。 03 Mim-8 诺和诺德研发的免疫球蛋白G同型抗体Mim-8，是一种凝血因子IX和X拮抗剂，用于治疗“凝血因子VIII缺乏症”。 04 tiratricol（替拉曲考） Rare Thyroid Therapeutics International AB研发的tiratricol用于治疗“X连锁遗传病”，甲状腺激素受体激动剂，曾获“孤儿药、罕见儿科疾病用药”资格认定。 05 posoleucel AlloVir研发的posoleucel，是一种T淋巴细胞疗法，用于治疗“腺病毒感染、BK病毒感染、膀胱炎、巨细胞病毒感染、EB病毒感染、造血干细胞移植、疱疹病毒6型感染、多瘤病毒感染”，曾获“孤儿药、EMA优先药物、再生医学先进疗法”资格认定。此次进入临床二期的适应症是“巨细胞病毒感染、腺病毒感染、BK病毒感染、EB病毒感染、多瘤病毒感染、疱疹病毒6型感染”。 版权声明：本文转自药融云，如不希望被转载的媒体或个人可与我们联系，我们将立即删除 【关于药融圈】药融圈围绕我国生物医药产业链，针对生物医药大数据、技术和资本投资、药融园(产业园)等开展系列系统性工作，促进我国生物医药产业健康发展，完善产业链，共同面对全球合作和竞争。 点分享 点点赞点在看

PledPharma AB (publ) has today completed the acquisition of Rare Thyroid Therapeutics International AB. As previously announced, PledPharma AB (publ) (“PledPharma” or the “Company”) entered into an agreement on 5 October 2020 to acquire all outstanding shares in Rare Thyroid Therapeutics International AB (“RTT” or “Rare Thyroid Therapeutics”), a privately held drug development company with the ultra-orphan asset Emcitate. The purchase price for the shares in RTT consists of a cash purchase price in the amount of MSEK 60, funded from own cash-in-hand and a share purchase price consisting of 63,773,345 new shares in PledPharma. In addition, the sellers of RTT are entitled to earnout payments based on the future net sales of Emcitate as well an earnout which is payable in connection with a potential sale of a so-called US Rare Pediatric Disease Priority Review Voucher. On 5 October 2020 the board of directors also resolved, subject to the general meeting’s subsequent approval, on an issue of up to 63,773,345 shares to the current shareholders of RTT against payment in kind in the form of shares in RTT. The extraordinary general meeting in PledPharma held on 28 October 2020 resolved to approve the board of directors’ resolution regarding the in-kind issue as part of the purchase consideration for RTT. Today on 3 November 2020 the acquisition of RTT was completed. The share issue in kind will lead to an increase in the number of shares of PledPharma by 63,773,345 from 53,533,321 to 117,306,666, which will result in a dilution of approx. 54,4 percent for existing shareholders. The completion of the acquisition has also resulted in that Peder Walberg assumes the position as member of the board of directors of PledPharma, in accordance with the resolution on the extraordinary general meeting on 28 October 2020. “The acquisition of RTT is an important step, building a new company with a strategic focus on the attractive orphan drug segment, with Aladote and Emcitate as the company’s key assets in late stage development. The RTT team, specialized in orphan drugs, will complement PledPharma’ s late stage development focused organization, building a new orphan drug company named Egetis Therapeutics, dedicated to development and commercialization of therapies for rare diseases,” said Nicklas Westerholm, CEO, PledPharma.

PledPharma AB (publ) (STO: PLED) (“PledPharma” or the “Company”) has today entered into an agreement to acquire all outstanding shares in Rare Thyroid Therapeutics International AB (“RTT” or “Rare Thyroid Therapeutics”), a privately held drug development company with the ultra-orphan asset Emcitate (the “Transaction”). The purchase price for the shares in RTT consists of a cash purchase price in the amount of 60 MSEK, funded from own cash-in-hand, which will be paid on closing and a share purchase price consisting of 63,773,345 new shares in PledPharma. In addition, the sellers of RTT are entitled to earnout payments based on the future net sales of Emcitate as well an earnout which is payable in connection with a potential sale of a so-called US Rare Pediatric Disease Priority Review Voucher. The Board of Directors in PledPharma has resolved, subject to the general meeting’s subsequent approval, on an issue of 63,773,345 shares to the current shareholders off RTT against payment in kind in the form of shares in RTT. The Board of Directors has further resolved, subject to the general meeting’s subsequent approval, on a fully guaranteed share issue with preferential rights for existing shareholders in the amount of approx. 200 MSEK and to propose that the general meeting authorizes the Board of Directors to resolve on an over-allotment option in the amount of approx. 50 MSEK, please refer to a separate press release for more information. The Transaction is conditioned on that the general meeting approves the Board of Directors’ resolutions regarding the rights issue and the in-kind issue. The Transaction is expected to be completed in early November 2020 provided that the condition described above has been fulfilled. The company has the intention to change its name to Egetis Therapeutics AB.