Viela Bio, Inc.

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。 9月24日，Amgen宣布其OX40单抗rocatinlimab用于治疗特应性皮炎的III期试验和已获批上市的CD19抗体Uplizna在用于重症肌无力的III期试验中均取得成功。 rocatinlimab AMG 451（rocatinlimab）是一款全人源化抗OX40单克隆抗体在研新药，安进通过一项高达12.5亿美元的合作获得了共同开发和商业化该产品的权益。 Horizon研究是安进公司正在进行的有关rocatinlimab八项III期试验中的第一项，此外围绕rocatinlimab的研究还覆盖特应性皮炎、哮喘、结节性痒疹等多个适应症。 HORIZON研究（NCT05651711）中共入组726例特应性皮炎患者，按3:1随机分组接受Rocatinlimab单药或安慰剂治疗，主要终点为治疗24周的EASI-75和fIGA 0/1，旨在评估Rocatinlimab 单药治疗中度至重度特应性皮炎 (AD) 成年患者的疗效、安全性和耐受性。 rocatinlimab达到了主要终点，所有次要终点也都有显著的统计学意义。24周后，32.8%的患者的湿疹面积和严重程度指数（EASI-75）至少下降了75%，而服用安慰剂的患者只有13.7%；治疗组rIGA 0/1响应率为16.4%，安慰剂组为4.9%。总体安全性结果与2b期试验相似。 rocatinlimab的成功将巩固安进相对于赛诺菲amlitelimab的优势，赛诺菲将amlitelimab定位为其转向免疫学的主要增长动力之一。但该药的首个III期临床试验要到2026年底才能完成。与此同时，安进至少还有四项rocatinlimab的III期临床试验预计将在明年七月前报告数据，包括不同剂量的研究、该药物作为联合或维持疗法的使用，以及一项针对青少年的试验。 Uplizna 其罕见病药物Uplizna在治疗自身免疫性疾病重症肌无力的3期试验中达到了主要终点和关键次要终点。 Uplizna是一款对CD19具有高度亲和力的人源化单克隆抗体。CD19表达在多类B细胞上，其中包括分泌抗体的成浆细胞（plasmablasts）和浆细胞。通过与CD19抗原结合，Uplizna能够迅速将这些细胞从血循环中清除，从而降低自身抗体的产生，缓解患者症状。它最初由Viela Bio公司开发，在2020年首次获得FDA的批准，治疗视神经脊髓炎谱系疾病（NMOSD）患者。 MINT研究中共入组238例全身性重症肌无力患者，按1:1随机分组接受Uplizna单药或安慰剂治疗，主要终点为总体研究人群第26周时重症肌无力日常活动量表（MG-ADL）评分与基线相比的变化。 该研究达到了主要终点，与安慰剂相比，在第26周服用两剂UPLIZNA后，MG-ADL评分有临床意义和统计学意义的改善：总体改善-4.2，安慰剂调整后-1.9（p<0.0001）。也达到了次要终点，与安慰剂相比，在第26周服用UPLIZNA后，QMG评分有统计学意义的改善：总体改善-4.8，安慰剂调整后-2.5（p = 0.0002）。正在按方案评估其他疗效、反应持久性和安全性，未发现新的安全性信号，安进即将启动监管申报活动。 此前6月，MITIGATE 3期研究在IgG4-RD领域取得了令人瞩目的成果，这是全球首例达到主要终点的IgG4-RD随机对照试验。与安慰剂相比，52周时IgG4-RD复发风险降低了87%，具有临床意义和统计学意义（HR=0.13, p < 0.0001）。经多重性调整后，所有三个关键次要终点均达到统计学意义，首次给药后的给药间隔为 6个月，非常方便；采用类固醇间隔研究设计，以减少类固醇负担和毒性，未发现新的安全性信号。安进计划首先向美国FDA递交监管申请，随后在其他国家和地区递交监管申请以扩展Uplizna的适应症。 参考资料：公司公告 -END- 2025CMC-CHINA药博会 • 展位火热预定中！

▎药明康德内容团队编辑 日前，安进（Amgen）公布其与Kyowa Kirin共同开发的在研OX40靶向单抗rocatinlimab在HORIZON临床3期试验中的积极顶线结果。分析显示，该疗法达成共同主要终点与所有关键次要终点，rocatinlimab能够显著改善特应性皮炎（AD）患者的临床症状。两家公司将在未来的医学会议上公布试验详细结果。安进同时也公布旗下药品Uplizna（inebilizumab）在治疗重症肌无力（gMG）患者的3期试验中达到主要终点，公司已着手启动相关监管递交准备。 特应性皮炎是最常见的湿疹类型，是一种慢性炎症性疾病，会导致皮肤过度干燥、瘙痒并伴有疼痛。中度至重度特应性皮炎患者会出现慢性症状，不可预测的发作会加剧症状，可能带来疼痛并扰乱日常生活。这些患者中几乎有一半报告出现严重瘙痒，导致反复抓挠，这会导致皮肤增厚并容易受到感染。特应性皮炎影响15-20%的儿童和高达10%的成人。T细胞失衡是特应性皮炎的根本原因，导致包括疾病反复发作、不可预测的症状在内的临床表现。 HORIZON是一项为期24周的随机、双盲、安慰剂对照的3期研究，旨在评估rocatinlimab单药治疗中度至重度特应性皮炎成人患者的疗效、安全性和耐受性。HORIZON是全球ROCKET临床3期试验项目中的八项研究之一。 ▲HORIZON试验设计（图片来源：参考资料[1]） 分析显示，HORIZON达到了双主要终点。即在第24周时，与安慰剂组患者相比，rocatinlimab组患者中达到经验证特应性皮炎研究者总体评估（vIGA-ADTM）评分为0（皮肤症状清除）或1（皮肤症状几乎清除）且较基线减少≥2分的比例显著较高（19.3% vs. 6.6%，差异为12.8%，p<0.001）；达到湿疹面积和严重度指数较基线减少75%（EASI-75）的rocatinlimab组患者比例亦较高（32.8% vs. 13.7%，差异19.1%，p<0.001）。该试验还在第24周时达到修订后研究者总体评估（rIGA 0/1）这项终点，这是一种比vIGA 0/1更严格的疗效衡量标准，基于更严格的皮肤症状几乎消失的定义（16.4% vs. 4.9%，差异为11.5%，p<0.001）。 该研究还在所有关键次要终点上达到统计学上的显著差异，这些次要终点包括皮肤清除率测量（第16周的vIGA 0/1和EASI-75以及第24周的EASI-90）、瘙痒症数字评定量表、特应性皮炎皮肤疼痛量表、皮肤病学生活质量指数以及手部特应性皮炎和面部特应性皮炎的严重程度评分。 HORIZON试验的整体安全性结果与过往2b期研究中的结果一致。 ▲Rocatinlimab药物机制（图片来源：参考资料[1]） Rocatinlimab是一种OX40靶向人源单克隆抗体，目前正在研究用于治疗中度至重度特应性皮炎。Rocatinlimab有可能成为首个通过靶向OX40来抑制和减少致病T细胞，以达到体内T细胞再平衡的疗法。OX40是一种共刺激受体，参与驱动特应性皮炎和其他疾病中的全身和局部炎症反应。过往研究显示，表达OX40的效应T细胞存在于特应性皮炎患者的病变中，并且在疾病病理学中至关重要。该疗法也正在接受评估用于治疗中度至重度不受控制的哮喘、结节性痒疹和其他可能因T细胞失衡而导致炎症的疾病。该疗法最初是由Kyowa Kirin和La Jolla免疫学研究所合作发现的。 与此同时，安进也宣布其罕见病药物inebilizumab在治疗重症肌无力的3期试验中达到了主要终点。分析显示，在重症肌无力日常生活活动能力（MG-ADL）测量量表中，接受两剂inebilizumab治疗的患者，在26周后评分改善了4.2分，而安慰剂组则改善了1.9分（p<0.0001）。此外，该研究的五个次要终点中有四个有统计学显著差异。 今年6月，安进公布inebilizumab另一项临床3期试验的顶线结果，inebilizumab在治疗IgG4相关疾病（IgG4-RD）时达到了主要临床终点。在含安慰剂对照的52周试验期间，IgG4-RD发作风险与安慰剂相比显著降低87%（HR=0.13，p<0.0001）。基于这些积极数据，安进计划首先向美国FDA递交监管申请，随后在其他国家和地区递交监管申请以扩展inebilizumab的适应症。 ▲Inebilizumab药物机制（图片来源：参考资料[1]） Inebilizumab是一款对CD19具有高度亲和力的人源化单克隆抗体。CD19表达在多类B细胞上，其中包括分泌抗体的成浆细胞（plasmablasts）和浆细胞。通过与CD19抗原结合，inebilizumab能够迅速将这些细胞从血循环中清除，从而降低自身抗体的产生，缓解患者症状。它最初由Viela Bio公司开发，在2020年首次获得FDA的批准，治疗视神经脊髓炎谱系疾病（NMOSD）患者。 ▲欲了解更多前沿技术在生物医药产业中的应用，请长按扫描上方二维码，即可访问“药明直播间”，观看相关话题的直播讨论与精彩回放 参考资料： [1] Driving Long-Term Growth: Rocatinlimab and UPLIZNA® Update. Retrieved September 25, 2024 from https://investors.amgen.com/static-files/a7119a35-06de-497e-ba75-146404c54d95 [2] Kyowa Kirin Announces Top-line Data from Rocatinlimab Phase 3 ROCKET HORIZON Trial for Adults with Moderate to Severe Atopic Dermatitis. Retrieved September 25, 2024 from https://www.prnewswire.com/news-releases/kyowa-kirin-announces-top-line-data-from-rocatinlimab-phase-3-rocket-horizon-trial-for-adults-with-moderate-to-severe-atopic-dermatitis-302258420.html 免责声明：药明康德内容团队专注介绍全球生物医药健康研究进展。本文仅作信息交流之目的，文中观点不代表药明康德立场，亦不代表药明康德支持或反对文中观点。本文也不是治疗方案推荐。如需获得治疗方案指导，请前往正规医院就诊。 版权说明：本文来自药明康德内容团队，欢迎个人转发至朋友圈，谢绝媒体或机构未经授权以任何形式转载至其他平台。转载授权请在「药明康德」微信公众号回复“转载”，获取转载须知。 分享，点赞，在看，聚焦全球生物医药健康创新

→ The top leadership at Lexicon Pharmaceuticals is still undergoing a major makeover as president and COO Jeff Wade heads for the exit on Sept. 30 after 25 years with the Texas-based biotech. Wade won’t be around for the outcome of Lexicon’s latest attempt at an FDA nod in type 1 diabetes for sotagliflozin , which has a Dec. 20 decision date. The drug has already been approved for heart failure and is marketed as Inpefa . Lexicon laid off about 75 people in its sales and commercial teams in August “to right-size our promotional efforts around Inpefa for heart failure and reallocate resources to prepare for our potential launch” in type 1 diabetes and chronic kidney disease, CEO Mike Exton said in a statement. Exton succeeded Lonnel Coats as CEO a month earlier. → Sarepta Therapeutics has appointed Genmab chair Deirdre Connelly to the board of directors. Connelly had a 26-year career with Eli Lilly before pivoting to GSK in 2009 as president, North America pharmaceuticals until her retirement in 2015. She’s also a board member at Macy’s and Lincoln Financial Group . Like Janet Woodcock before him, CBER Director Peter Marks helped Sarepta cross the goal line with a label expansion in June for its Duchenne muscular dystrophy treatment Elevidys — overruling his own staff and befuddling experts. → Mitchell Chan has replaced Vit Vasista as CFO of Regenxbio at a time when the company plans to start a pivotal study for its own Duchenne gene therapy. It’s another big change for Regenxbio after Ken Mills became executive chairman and Curran Simpson succeeded Mills as CEO on July 1. Chan spent a year with Catalio Capital Management as operating partner after he was elevated to the CFO post at Viela Bio. Vasista had been finance chief since 2009 and will be an advisor until Jan. 3. → Here’s a look at some CEO news that we covered earlier this week: Former Kite Pharma commercial chief Warner Biddle has succeeded Peter Maag as CEO of Kyverna Therapeutics , which also added ex-Kite CEO Christi Shaw to the board. Shares $KYTX of Kyverna have taken a precipitous fall since its $319 million IPO in February. Ochre Bio co-founder Jack O’Meara is calling it quits after five years as CEO, but he tells Lei Lei Wu that he has an Alzheimer’s startup in the works. In a LinkedIn post announcing his departure, O’Meara also said that former F-star CEO and current Levicept chief Eliot Forster will be interim executive chair. And Shilpa Sambashivan has been promoted to CEO of Nura Bio , which stapled an additional $68 million to its Series A — bringing its total to $141 million. Sambashivan was a project team lead at Amgen before becoming a founding member at Nura Bio. → Matt Kane is taking the top spot at Celyad Oncology , a Belgian biotech that has seen considerable turnover at the top. Celyad co-founder Michel Lussier filled in as interim CEO for a second time after Georges Rawadi ’s sudden exit last December; he also took over when Filippo Petti stepped down in 2022. Kane co-founded Precision BioSciences with Derek Jantz and Jeff Smith , leading the company until he joined epigenome editing startup Tune Therapeutics as CEO in 2021. Kane’s LinkedIn page indicates that he left Tune in April, a year after he hired Jantz as CSO. → Ex- Prometheus Biosciences chief Mark McKenna has found a CFO at his new I&I bet, Mirador Therapeutics . Gregg Gilbert had been a managing director in the healthcare investment banking group for Bank of America Securities since 2021 and worked at Bank of America Merrill Lynch for 15 years. After selling Prometheus and its inflammatory bowel disease drug to Merck for $10.8 billion , McKenna wasted little time getting Mirador off the ground with a $400 million Series A in March, one of the largest private financing rounds of 2024. → GLP-1 competitor Structure Therapeutics has promoted Blai Coll to CMO and welcomed Ashley Hall as chief development officer. Coll, who replaces Mark Boch , had led clinical development for Raymond Stevens ’ crew since May 2022 and was cardiovascular and metabolic platform lead, medical affairs at the end of a six-year stint with Amgen. Hall is an Amgen vet in her own right who held the role of development chief at Reneo Pharmaceuticals . Structure’s stock price soared in June $GPCR when it produced efficacy data with its oral GLP-1 GSBR-1290 that was on par with Eli Lilly’s obesity drug orforglipron . → Novartis and Gilead veteran Matthias Will has succeeded Edgar Braendle as chief development officer at Autolus Therapeutics . Will had led clinical development at CRISPR Therapeutics and CytomX before his most recent gig as CMO of Dren Bio . The FDA is expected to make a decision by Nov. 16 on Autolus’ CD19-directed CAR-T obecabtagene autoleucel , or obe-cel . → According to an SEC filing , Crinetics chief commercial officer Jim Hassard will pack his bags on Oct. 14 in what’s described as a “termination without cause.” The 17-year Amgen vet came to Crinetics from Arrowhead Pharmaceuticals in March 2022. Phase 3 successes with its acromegaly drug paltusotine have propelled Crinetics to an NDA filing “later this year” and “an expected market launch in 2025,” CEO Scott Struthers said in the San Diego biotech’s second-quarter report. CFO Mark Wilson cited personal reasons for his own upcoming departure, which was also included in Crinetics’ Q2 update. → Allosteric drug discovery shop HotSpot Therapeutics has introduced Alison O’Neill as CMO. O’Neill is the former medical chief at Surface Oncology , which was acquired by Coherus BioSciences last year for $65 million. She had a senior leadership role in the global oncology division at Sanofi and was VP of clinical development for Radius Health . → Ex- Icosavax CEO Adam Simpson has landed at Frazier Life Sciences as a venture partner on its company creation team and will work out of Frazier’s office in San Diego. Simpson sold the vaccine developer to AstraZeneca last December, and his previous company — celiac disease specialist PvP Biologics — was purchased by Takeda in 2020. → Ruben Tommasi has been named CSO of Entact Bio , a Watertown, MA-based biotech led by ex- Ribon Therapeutics CEO Victoria Richon that is developing enhancement-targeting chimeric molecules, or ENTACs. Tommasi has also held this role at Dunad Therapeutics and AstraZeneca spinout Entasis Therapeutics . Entact Bio has kept a relatively low profile since its $81 million Series A in 2022, but it did show up in Peer Review this spring when it recruited fellow Entasis vet Michael Gutch as COO. → Third Rock ’s Treg developer Abata Therapeutics has resurfaced in Peer Review for the first time in more than a year, naming Joanne Beck as chief technical officer. The Shire and Celgene alum is the ex-technical chief at Aerium Therapeutics , the Covid biotech led by Rajeev Venkayya that recently shut down because of a surge in new variants. In April 2023, Abata poached Sonoma Biotherapeutics CMO Leonard Dragone for the same role . → Led by ex- Teva R&D chief Michael Hayden , neuro biotech Prilenia has tapped Rob Lauzen as CFO and Jason Marks as chief legal officer. Lauzen was VP, investor relations and head of financial planning & analysis at Dyne Therapeutics , which made a CMO change and lost its COO and CBO earlier this month. Marks just had a short stint with Teva as a senior legal advisor to global transactions and global finance. The Bausch Health alum has also been chief legal and compliance officer at Amarin . → San Francisco eye disease biotech Ray Therapeutics has selected Christopher Whitmore as CFO. Whitmore had held the same position with Immune-Onc Therapeutics since July 2022, and he was VP, finance & administration for Harpoon Therapeutics from 2018-21. Ray Therapeutics, which hauled in a $100 million Series A last year, will test its gene therapy RTx-015 in patients with retinitis pigmentosa. → Tris Pharma has promoted VP of marketing Manesh Naidu to the role of chief commercial officer. Naidu joined the New Jersey-based company in 2023 after a stint as chief marketing officer of Althera Pharmaceutical . Naidu’s résumé also includes stints at McKinsey , Mallinckrodt , Novartis, Pfizer and Bellerophon Therapeutics . In May, Tris snagged the first liquid non-stimulant ADHD drug approval in the US. Onyda XR will be used to treat ADHD in children at least 6 years of age. → Backed by Jeff Bezos , Seattle-based Nautilus Biotechnology has named Kentaro Suzuki as chief marketing officer. Suzuki most recently served as VP and general manager of Agilent Technologies’ mass spectrometry division. Before that, he was with Takeda and Hewlett-Packard . → San Francisco-based 89bio has appointed Teresa Perney as chief regulatory and quality officer. Perney held this title at EQRx after five years with Myovant Sciences as SVP of regulatory and quality. Madrigal Pharmaceuticals finally broke through with an FDA approval for metabolic dysfunction-associated steatohepatitis (also referred to as NASH), and 89bio is enrolling patients for late-stage studies of pegozafermin — one for non-cirrhotic MASH and another for MASH with compensated cirrhosis. “I’m eager to leverage my experience to navigate the regulatory landscape and help bring this innovative therapy to patients worldwide,” Perney said in a statement . → There’s also a new regulatory chief at Disc Medicine : Steve Caffé spent five years with CRISPR Therapeutics as head of regulatory affairs until his departure last December. Earlier, he had numerous stops as a regulatory exec with such companies as Ra Pharmaceuticals , Sucampo Pharmaceuticals , AMAG Pharmaceuticals and MedImmune . Shares of Disc $IRON fell sharply in early April after mixed data with its drug for a rare blood disorder called erythropoietic protoporphyria, but have rebounded since. → Although AI-designed drugs haven’t exactly lived up to the hype, companies like BPGbio are trying to flip the narrative. This week, GSK alum John Beeler has joined the team as SVP of business development. Beeler comes to BPGbio from Bristol Myers Squibb , where he took on the role of business development search & evaluation lead. → Macrocycle startup Circle Pharma has brought in Marie Evangelista as SVP and head of cancer biology, and promoted Constantine Kreatsoulas to SVP and head of discovery technology sciences. Evangelista spent the last two years as VP of oncology at one of the most ambitious AI drug developers , Recursion . The longtime Genentech vet also had a brief tenure with Frontier Medicines as senior director of translation medicine. Kreatsoulas had a nine-year run at GSK and was the pharma giant’s interim head, computational toxicology before coming to Circle in 2021 as head of research and VP, research informatics and computational chemistry. Circle disclosed its $54 million Series D in an SEC filing this summer. → South San Francisco-based Ideaya Biosciences has recruited Douglas Snyder as general counsel. Snyder most recently served as chief legal officer and secretary of GW Pharmaceuticals . Earlier in his career, Snyder was general counsel and secretary at Actelion and Eisai . Back in July, Ideaya inked a $406.5 million deal with Biocytogen . → Deerfield Group has rolled out the welcome mat for two new execs: Guy Varady as EVP, integrated marketing and Bob Josefsberg as EVP, communications. Before this, Varady was SVP, management supervisor for DDB Health and held a number of roles at IPG Health and Omnicom Health Group . Meanwhile, Josefsberg was VP, corporate communications at Karuna Therapeutics and managing director at Chamberlain Healthcare Public Relations . → Tempest Therapeutics , which revealed early PFS and OS data for its liver cancer combo last October, has brought on Sheldon Mullins as VP, regulatory affairs. Mullins joins the team after gigs at Arrowhead Pharmaceuticals and Ardelyx . Earlier in his career, Mullins was with Anacor Pharmaceuticals , Onyx Pharmaceuticals and Genentech. In addition to Mullins’ appointment, Darrin Bomba and Henry Johnson have been promoted to the titles of VP, development operations and VP, CMC & medicinal chemistry, respectively. → Entrada Therapeutics CEO Dipal Doshi has picked up a seat on the board of directors at āshibio , a California biotech that will push its ex-Gilead drug andecaliximab into a Phase 2/3 study for patients with an ultra-rare bone disorder called fibrodysplasia ossificans progressiva (FOP). Āshibio came out of stealth mode this summer with a $40 million raise . → Akari Therapeutics , which is developing PAS-nomacopan for geographic atrophy, has elected former Epizyme CEO Rob Bazemore to the board of directors. Bazemore is also a board member at Ardelyx and Nuvation Bio .