Months af ter a ma jor re brand ing around CAR-NKT cells, Ku ur Ther a peu tics is of fer ing a peek at ear ly re sults


2021-01-22 07:00:15 ENDPOINTS NEWS


Sev­er­al months af­ter a name change and ma­jor re­struc­tur­ing, Ku­ur Ther­a­peu­tics has of­fered an ear­ly glimpse at re­sults from a small num­ber of pa­tients treat­ed with their CAR-NKT cell ther­a­pies de­vel­oped with the Bay­lor Col­lege of Med­i­cine. Out of 10 evalu­able neu­rob­las­toma pa­tients dosed with Ku­ur’s lead can­di­date, an au­tol­o­gous GD2-tar­get­ing CAR-NKT ther­a­py, re­searchers not­ed one com­plete re­sponse, one par­tial re­sponse, and three pa­tients with sta­ble dis­ease, ac­cord­ing to the Hous­ton-based biotech. The pa­tient with a com­plete re­sponse had pre­vi­ous­ly achieved a par­tial re­sponse be­fore re­ceiv­ing a sub­se­quent dose of the drug. The drug ap­peared safe, ac­cord­ing to Ku­ur, with one case of Grade 2 cy­tokine re­lease syn­drome (CRS) oc­cur­ring in the Phase I tri­al, dubbed GI­NAKIT2. “We have some very good ear­ly signs of NKT cells be­ing safe, hom­ing to the site of tu­mor, which is very im­por­tant go­ing af­ter sol­id tu­mors … and now we’re show­ing signs of ef­fi­ca­cy as well,” CEO Kevin Boyle told End­points News. “So, based on that, we want the com­pa­ny to be fo­cused on just this plat­form.” The drug con­sists of nat­ur­al killer T cells en­gi­neered with a CAR tar­get­ing GD2, a ma­jor gan­glio­side ex­pressed on many neu­rob­las­toma tu­mors. It’s de­signed to se­crete IL-15, which has been shown to bol­ster the ef­fi­ca­cy and per­sis­tence of CAR-NKT cells in the im­muno­sup­pres­sive tu­mor mi­croen­vi­ron­ment. NKT cells are rare lym­pho­cytes that aren’t fre­quent­ly found in the pe­riph­er­al blood, and as a re­sult, re­searchers tend not to work with them, Boyle ex­plained. The sci­en­tist lead­ing Bay­lor’s re­la­tion­ship with Ku­ur has been work­ing with the cells for two decades, he said. “One rea­son that we like this cell type is it homes nat­u­ral­ly to the tis­sue in sol­id tu­mors… It al­so has an in­vari­ant T-cell re­cep­tor… and that means it’s the same in every­one so it won’t cause graft-ver­sus-host dis­ease when we use it in an­oth­er per­son as off-the-shelf ther­a­py. And that means we don’t need to gene ed­it the TCR and that puts us in a fun­da­men­tal­ly dif­fer­ent po­si­tion from the peo­ple work­ing with CAR-T cells,” CMO Kurt Gunter said. Ku­ur’s sec­ond can­di­date, an off-the-shelf CD19-di­rect­ed CAR-NKT ther­a­py, achieved a PR and CR in the two pa­tients with hema­to­log­i­cal ma­lig­nan­cies who were evalu­able in the Phase I study, AN­CHOR. The re­searchers saw no CRS cas­es, and no ev­i­dence of graft ver­sus host dis­ease (GvHD) — a con­di­tion where donor cells at­tack their host, the com­pa­ny said. Boyle, who took the helm last March dur­ing a ma­jor re­struc­tur­ing, ex­pects to read out full da­ta from both the Phase I stud­ies next year. And if all goes well, he’s plan­ning to launch a Phase II ex­pan­sion tri­al of the AN­CHOR study in 2021. Un­til last March, Ku­ur went by the name Cell Med­ica. The Lon­don-based up­start had lo­ca­tions across two con­ti­nents and three coun­tries, Boyle said. But in­stead of work­ing on a va­ri­ety of cell ther­a­pies, the com­pa­ny chose to laser-fo­cus on the CAR-NKT pro­gram un­der an ex­clu­sive li­cense with Bay­lor. It set­tled on one lo­ca­tion in Hous­ton, right next door to their col­lab­o­ra­tors, and for­mer CEO Chris Now­ers was re­placed by Boyle, who had pre­vi­ous­ly served as CFO. The com­pa­ny hopes to close a pri­vate fi­nanc­ing round this quar­ter, Boyle hint­ed, some of which will be used to trans­form the sin­gle-cen­ter stud­ies in­to mul­ti-cen­ter stud­ies. “The sit­u­a­tion with neu­rob­las­toma is still kind of bleak es­pe­cial­ly for these high risk re­lapsed re­frac­to­ry pa­tients,” Gunter said. “They have gen­er­al sur­vivals of less than one year, once they’ve re­lapsed,” he con­tin­ued, adding that he be­lieves the com­pa­ny can bring a sig­nif­i­cant ben­e­fit to pa­tients. By RBC Capital Markets With Andy Schwab, Co-Founder and Managing Partner at 5AM Ventures Key Points Prescription Digital Therapeutics, cell therapy technologies, and in silico medicines will be a vital part of future treatment modalities. Unlocking the potential of the microbiome could be the missing link to better disease diagnosis. Growing links between academia, industry, and venture capital are spinning out more innovative biotech companies. Biotech is now seen by investors as a growth space as well as a safe haven, fuelling the recent IPO boom. GlaxoSmithKline’s HIV unit ViiV was dealt a stinging loss back in late 2019 when the FDA slammed the brakes on its application for a once-monthly injection based on manufacturing issues. Now, with that roadblock in the rearview, ViiV has finally made good on its promise to change the HIV game. The FDA on Thursday approved ViiV’s Cabenuva (cabotegravir and rilpivirine) as a long acting, once-monthly therapy for HIV-positive adults who are virologically suppressed and on a stable antiviral regimen. It’s official: Janet Woodcock is now the acting chief of the FDA. And — according to an Endpoints poll — most industry readers would like her to stay there, although a significant minority is strongly opposed. To recap: Joe Biden is reportedly choosing between Woodcock and former deputy FDA commissioner Joshua Sharfstein as his nominee for the permanent position. Given their respective track records, the decision is set to determine the agency’s lodestar for years to come. The firehose of biopharma news is gushing these days. That’s why broader and deeper is the theme for 2021 at Endpoints. You can expect new coverage outside our core R&D focus, with deeper reporting in some key areas. When John Carroll and I launched Endpoints nearly five years ago, we were wading in waist-high waters. Now we’re a team of 25 full-time staffers (and growing) with plans to cover the flood of biopharma news, Endpoints-style. Close to 2 years ago, GSK’s R&D team eagerly agreed to pay up to $4 billion-plus to ally itself with Merck KGaA on a mid-stage bispecific called bintrafusp alfa, which intrigued them with the combination of a TGF-β trap with the anti-PD-L1 mechanism in one fusion protein. But today the German pharma company says that their lead study on lung cancer was a bust, as independent monitors said there was no reason to believe that the experimental drug — targeting PD-L1/TGF-Beta — could beat Keytruda. Eli Lilly says bamlanivimab lowered the risk of contracting symptomatic Covid-19 in a first-of-its-kind trial involving nursing home residents and staff, paving the way for a new option to protect against the virus. But how big of an impact it might have, and what role it will play, at a time vaccines are being rolled out to the exact population it is targeting still remains unclear. Among 965 participants in the study — all of whom tested negative for the coronavirus at baseline — the number of symptomatic cases reported in the bamlanivimab arm was 57% lower than that in the placebo arm (odds ratio 0.43, p=0.00021). In addition to that primary endpoint, all secondary endpoints reached statistical significance. Tony Coles’ team at Cerevel Therapeutics is adding two high-profile board members, including an ex-governor that has lots of connections to the Boston area where the biotech is based. Former Massachusetts Gov. Deval Patrick is hopping on Cerevel’s board of directors, the company announced Thursday, joining less than three months after Cerevel went public on the backs of Perceptive’s ARYA II SPAC. And in a twist, Pfizer’s new business development chief Deborah Baron is joining the board as well, about three years after the Big Pharma shuttered the neuroscience pipeline that Cerevel is seeking to revive. Seven years after the FDA first approved Esbriet, the blockbuster Roche IPF drug is getting an expedited review for a second indication. On Thursday, the agency gave Esbriet priority review for unclassified interstitial lung diseases, or forms of pulmonary inflammation and scarring that don’t fit easily into the over 200 known types of ILD. The move comes 10 months after Esbriet received breakthrough designation and sets Roche up for a decision by May. The biopharma industry, on the whole, had a red-letter year in 2020 amid Covid-19, with fundraising at an all-time high and major players speeding vaccines ahead to approval. But for BIO, the industry’s leading trade organization, the pandemic has prompted a reconsideration of the game plan. BIO will pivot to digital as the Covid-19 pandemic continues to rage, making “some staff reductions” as it looks to bring its roughly 37,000 in-person meetings each year to the web, the organization said Thursday.
在改名和Major重组后的几个月里,Kuur Therapeutics从一小部分病人用他们与医学贝勒学院开发的CAR-NKT细胞治疗中初步看到了结果。 在10例可评价的神经母细胞瘤患者中,根据基于Houston的生物技术,使用Kuurs Candidate铅(一种自体GD2靶向CAR-NKT疗法),研究人员发现1例完全反应,1例部分反应,3例出现刺痛。 有完全反应的病人在服用次剂量的药物之前已有部分反应。与Kuur一致,该药物是安全的,在I期试验中出现了一例2级细胞因子再租赁综合征(CRS),称为Ginakit2。 我们有一些非常好的早期迹象表明NKT细胞是安全的,回到了Tumor的位置,这在固体Tumor之后是非常重要的……现在我们也显示出了有效的迹象,首席执行官凯文·博伊尔告诉端点新闻。因此,基于此,我们希望companny只在这个平台上。 该药物含有一个以GD2为靶点的CAR激活的天然杀伤T细胞,GD2是一种在许多神经母细胞瘤上表达的Major ganglioside。在免疫抑制的Tumor microenvironment环境中,它能促进CAR-NKT细胞的活性和持久性。 波义耳说,NKT细胞是一种罕见的淋巴细胞,在外周血中并不常见,因此研究人员倾向于不使用它们。他说,领导Baylors与Kuur合作的科学学者已经研究这些细胞20年了。 我们喜欢这种细胞类型的一个原因是它天然地在固体管中寄居在组织中…它也有一个不变的T细胞受体…这意味着它在每个人身上都是一样的,所以当我们用它作为现成的实验品时,它不会引起移植物对宿主的不适。CMO Kurt Gunter说:“这意味着我们不需要对TCR进行基因编辑,这使我们处于与使用CAR-T细胞的人完全不同的位置。” Kuurs Second Candidate,一种现成的CD19导向的CAR-NKT疗法,在I期研究中可评估的两例血液肿瘤患者中获得了PR和CR。研究人员没有发现CRS病例,也没有移植物抗宿主病(GvHD)的迹象,GvHD是供体细胞攻击宿主的一种情况。 博伊尔去年3月在Major公司重组期间掌舵,明年他将宣读第一阶段研究的全部数据。如果一切顺利,他计划在2021年启动安丘尔研究的第二阶段试验。 直到去年三月,Kuur被命名为Cell Medica。博伊尔说,这位总部位于伦敦的新贵在两个地区和三个国家都有业务。但是,公司不是在大量的细胞实验中工作,而是在与Baylor的排除性接触下,对CAR-NKT程序进行了激光加工。它坐落在豪斯顿的一个地方,就在他们的Collabrators隔壁,Former的首席执行官Chris Nowers由之前担任CFO的Boyle接替。 该公司希望在这四个季度结束一个Private Finance,Boyle Hinted,其中一些将被用来将零星的研究转变为多个研究。 Gunter说,神经母细胞瘤的治疗仍然有些暗淡,尤其是对于那些高危复发的难治性患者。他们一般都有不到一年的随访,一旦复发,他继续说,他相信这种疗法能给病人带来明显的好处。 加拿大皇家银行资本市场 与5AM Ventures联合创始人兼管理合伙人安迪•施瓦布合作 要点 处方数字治疗技术,细胞治疗技术和硅胶药物将是未来治疗模式的重要组成部分。 开发微生物组的潜力可能是更好地诊断疾病所缺少的一环。 学术界,工业界和风险投资之间日益紧密的联系正在催生出更多创新的生物技术公司。 生物技术现在被投资者视为一个增长空间,同时也是一个安全的避风港,推动了最近的IPO热潮。 葛兰素史克公司HIV病毒第VIV单元在2019年末遭受了惨痛的损失,当时FDA基于生产问题对其每月注射一次的申请急刹车。现在,有了这个障碍,ViiV终于兑现了改变HIV游戏的承诺。 FDA周四批准VIIVS Cabenuva(卡博特格拉韦和利匹韦林)作为长期的,每月一次的治疗方案,用于病毒学受抑制且使用稳定的抗病毒方案的HIV阳性成人。 ITS官员:珍妮特·伍德科克现在是FDA的代理局长。 根据Endpoints的一项民意调查,大多数行业读者都希望她留在那里,尽管有相当一部分人强烈反对。 重述一下:据报道,乔·拜登正在伍德科克和前FDA副专员约书亚·沙夫斯坦之间选择他的永久职位提名人。鉴于它们各自的成绩记录,这一决定将确定原子能机构今后几年的方向。 这几天生物制药新闻的火龙喷涌而出。 这就是为什么2021年的主题更广泛,更深入。你可以期待我们的核心研发重点之外的新报道,在一些关键领域有更深入的报道。大约五年前,当约翰·卡罗尔和我推出Endpoints的时候,我们正趟着一条不起眼的水路。现在我们有一个由25名全职员工组成的团队(并且还在不断增长),计划覆盖生物制药新闻的洪流,终点式的。 将近两年前,GSKS的研发团队急切地同意支付高达40多亿美元与默克集团联合研制一种名为bintrafusp alfa的中期双特异性药物,该药物将TGF?陷阱与一种融合蛋白中的抗PD-L1机制结合在一起,引起了他们的兴趣。 但今天这家德国制药公司表示,他们在肺癌方面的领先研究是失败的,因为独立的监督员说,没有理由相信靶向PD-L1/TGF-beta的试验性药物能够打败Keytruda。 礼来公司表示,在一项涉及疗养院居民和工作人员的首次同类试验中,班拉尼单抗降低了感染有症状的柯维19病毒的风险,为预防病毒的新选择铺平了道路。 但在疫苗正向其针对的确切人群推出之际,它可能会产生多大影响,以及它将发挥何种作用,目前仍不清楚。 在965名基线冠状病毒检测呈阴性的研究参与者中,班拉维单抗组报告的有症状病例数比安慰剂组低57%(优势比0.43,P=0.00021)。除主要终点外,所有次要终点均达到统计学意义。 Cerevel Therapeutics的Tony Colesteam将增加两名高知名度的董事会成员,其中包括一名前州长,他与该生物技术公司所在的波士顿地区有很多联系。 Cerevel公司周四宣布,前马萨诸塞州州长德瓦尔•帕特里克将加入该公司董事会,而在不到三个月前,Cerevel公司在艾雅二世公司的支持下上市。此外,Pfizer新的业务发展主管黛博拉·巴伦也加入了董事会,大约三年前,这家大型制药公司关闭了Cerevel正在寻求重振的神经科学项目。 在FDA首次批准艾司布瑞特七年后,罗氏IPF这一重磅药物正获得第二个适应症的加速审查。 周四,该机构对未分类的间质性肺疾病,即不容易归入200多种已知ILD的肺部炎症和瘢痕进行了Esbriet优先审查。10个月前,Esbriet获得了突破性的称号,罗氏将在5月前做出决定。 总体而言,生物制药行业在2020年的Covid-19中迎来了喜人的一年,筹资达到了历史最高水平,各大公司加快了疫苗的审批速度。但对于该行业的主要贸易组织BIO来说,这场大流行促使他们重新考虑自己的游戏计划。 该组织周四表示,随着Covid-19疫情的持续肆虐,BIO将转向数字化,并将裁减一些员工,以实现每年约37,000次面对面会议的网络化。