With patent con cerns loom ing, Roche gets a new pri or i ty re view on block buster IPF drug


2021-01-22 11:04:51 ENDPOINTS NEWS


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 Swiss phar­ma is bid­ding for a new in­di­ca­tion at an acute pe­ri­od for their long­time block­buster. One of two ma­jor id­io­path­ic pul­monary fi­bro­sis drugs along­side Boehringer In­gel­heim’s Ofev, Es­bri­et con­tin­ues to bring over $1 bil­lion per year. But sales growth has slowed, par­tic­u­lar­ly in the US, and the com­pa­ny’s or­phan des­ig­na­tion pro­tec­tions in IPF be­gin to ex­pire this year. With that dead­line loom­ing, Roche has sought to guard against would-be gener­ics, fil­ing around a dozen law­suits to stop Te­va, San­doz and oth­ers from re­leas­ing cheap­er, copy­cat ver­sions of the drug. Es­bri­et’s an­nu­al whole­sale cost is around $100,000. Roche ac­quired Es­bri­et in their $8.3 bil­lion buy­out of the Cal­i­for­nia biotech In­ter­Mune, two months be­fore the FDA would give the drug a green light. The agency had ini­tial­ly re­ject­ed the drug in 2010, de­mand­ing In­ter­Mune run a sec­ond Phase III tri­al. The Big Phar­ma, though, would not put the drug in­to a piv­otal tri­al for un­clas­si­fi­able in­ter­sti­tial lung dis­ease un­til 2017. In 2019, in­ves­ti­ga­tors pub­lished Phase II re­sults from 253 un­clas­si­fi­able ILD pa­tients, show­ing pa­tients who took the drug had sig­nif­i­cant­ly more sta­ble lung func­tion, as mea­sured by an end­point called forced vi­tal ca­pac­i­ty. The new in­di­ca­tion is just one route Roche is us­ing to try to keep its fi­bro­sis fran­chise alive. The com­pa­ny has al­so in­vest­ed in find­ing a new block­buster for IPF, ac­quir­ing Prome­dior and its pipeline of an­ti-fi­brot­ic mol­e­cules for up to $1.4 bil­lon in 2019. The lead drug, which has re­ceived break­through des­ig­na­tion, went in­to Phase III ear­li­er this year. So far, Roche has stayed ahead of Boehringer in that search; two months af­ter Roche start­ed their Phase III, the Ger­man phar­ma dropped out of its own $1 bil­lion fi­bro­sis pact, cit­ing tox­i­c­i­ty with their part­ner’s drug. 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. 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. 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. 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. 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. 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. 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. 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FDA首次认可爱思必利的七年后,罗氏IPF这一重磅药品正在接受第二次公开的审查。 周四,该机构对未破裂的肺内质部疾病,或不容易与200多种已知ILD相匹配的肺内炎和瘢痕进行了优先复查。这一举动是在埃斯布里特公司重新获得突破设计10个月后,并为罗氏公司在5月前的设计做好准备。 瑞士法玛公司正在竞标一个新的标书,在一个尖锐的时期,他们的长期大片。Esbriet是与勃林格英格海姆Ofev一起的两种主要治疗肺纤维疾病的药物之一,每年将带来超过100万美元的收益。但销售增长已经放缓,尤其是在美国,而且康帕尼公司的Orphan Designation保护计划今年将在IPF展销。 随着最后期限的临近,罗氏一直在寻求防范可能成为仿制药的产品,提交了大约12起诉讼,以阻止Teva,Sandoz和Others重新出租更便宜的仿制产品。Esbriets Annual的整体销售成本在10万左右。 罗氏公司以83亿美元收购了加州生物技术公司Esbriet,两个月后,FDA将为该药开绿灯。该机构曾在2010年对该药进行了初步评估,取消了Intermune进行的第二次III期试验的资格。 不过,大法尔马公司在2017年之前不会将该药用于治疗难治性肺间质病变的临床试验。2019年,Investigators公布了253例无法识别的ILD患者的II期结果,显示服用该药的患者有明显更稳定的肺功能,这是通过一个被称为强迫性死亡的终点来测量的。 这个新的计划只是罗氏试图让它的Fibrosis Franchise存活下来的一个途径。该公司还投资于为IPF寻找一个新的重磅产品,与Promedior和它的管道的抗纤纤维分子在2019年高达1.4比伦。主导药物,已经获得突破设计,进入第三阶段提前在今年。 到目前为止,罗氏在这方面一直领先于勃林格;两个月后,罗氏开始了他们的第三阶段,German Pharma退出了自己的1百万纤维连接协议,因为他们的合作伙伴的药物有毒。 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。 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。 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。 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。 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。 在更名和重大重组几个月后,Kuur Therapeutics公司向少数患者提供了初步的结果,这些患者使用他们与贝勒医学院合作开发的CAR-NKT细胞疗法进行治疗。 根据Houston生物技术中心的研究,在10名接受Kuurs lead候选药物治疗的神经母细胞瘤患者中,研究人员注意到1例完全应答,1例部分应答,3例病情稳定。Kuurs lead候选药物是一种自体靶向GD2的CAR-NKT疗法。 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。 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。