Unit ed ax es PAH pro gram Trevyent, once bought for cheap in ac qui si tion of a ri val, af ter FDA feed back

单位ed ax es PAH pro gram Trevyent,曾在一个ri val的基础上廉价购买,得到FDA的反馈

2021-03-04 04:00:12 ENDPOINTS NEWS


About three years af­ter ac­quir­ing SteadyMed and its ex­per­i­men­tal pul­monary ar­te­r­i­al hy­per­ten­sion drug Trevyent, Unit­ed Ther­a­peu­tics has de­cid­ed to kick the pro­gram to the curb. In an 8-K form filed with the SEC on Tues­day, Unit­ed re­vealed that they are shut­ting down de­vel­op­ment of Trevyent af­ter re­ceiv­ing feed­back from the FDA last week. The move marks the end of a long and bumpy road for the pro­gram, which had pre­vi­ous­ly earned an RTF in 2017 and a CRL last April, as Unit­ed moves for­ward with new Ty­va­so for­mu­la­tions. Reg­u­la­tors had di­rect­ed Unit­ed to both re­design the Trevyent prod­uct and con­duct an en­tire­ly new clin­i­cal study. That not on­ly would have added sig­nif­i­cant de­lays to the pro­gram, Unit­ed wrote, but may have proved un­suc­cess­ful in an­swer­ing the FDA’s con­cerns. Specif­i­cal­ly, the FDA took is­sue with the prod­uct’s pump de­vice. Trevyent us­es a pump to de­liv­er a re­for­mu­lat­ed ver­sion of Unit­ed’s main­stay PAH drug Re­mod­ulin. The ac­cu­ra­cy of the pump, how­ev­er, was not up to snuff in reg­u­la­tors’ eyes, and Unit­ed said the agency want­ed the com­pa­ny to im­prove the ac­cu­ra­cy “in cer­tain re­spects.” So Unit­ed has de­cid­ed to throw in the tow­el. They’re halt­ing all de­vel­op­ment of the pro­gram af­ter de­ter­min­ing it wouldn’t be com­mer­cial­ly rea­son­able to move for­ward. It marks an un­cer­e­mo­ni­ous end to a pro­gram they once picked up for a bar­gain from their com­peti­tors at SteadyMed in 2018. With the $216 mil­lion buy­out, Unit­ed had been look­ing to head off a PAH clash be­tween the two com­pa­nies by ac­quir­ing Trevyent it­self. In ad­di­tion to that pro­gram, SteadyMed at the time had been try­ing to in­val­i­date one of Unit­ed’s patents for the ac­tive in­gre­di­ent in three of its promi­nent fran­chis­es: Re­mod­ulin, Ty­va­so and Oren­itram. Though Unit­ed moved for­ward with Trevyent, sub­mit­ting its NDA to US reg­u­la­tors in Sep­tem­ber 2019, the agency re­ject­ed its pitch in an April 2020 CRL. The FDA had said that some of the de­fi­cien­cies pre­vi­ous­ly point­ed out had “not yet been ad­dressed to its sat­is­fac­tion,” Unit­ed said at the time. Unit­ed had been work­ing on a new pitch and Tues­day’s de­ci­sion stemmed from its sub­se­quent meet­ing with the FDA to re­sub­mit Trevyent for ap­proval. It’s the lat­est in a cou­ple of pipeline set­backs Unit­ed has seen over the last few years. In 2019, the com­pa­ny dropped PAH hope­ful es­uber­aprost af­ter it failed a com­bo Phase III study, fail­ing to show ben­e­fit when com­bined with Ty­va­so over Unit­ed’s old­er Re­mod­ulin drug. Then, in Feb­ru­ary, Unit­ed re­vealed that a Phase II/III tri­al com­bin­ing their Uni­tux­in an­ti­body with a stan­dard treat­ment, irinote­can, failed to help small cell lung can­cer pa­tients live longer. Unit­ed is in­stead mov­ing for­ward with an in­haled re­for­mu­la­tion of Ty­va­so af­ter snag­ging a pri­or­i­ty re­view vouch­er from Y-mAbs Ther­a­peu­tics last De­cem­ber for $105 mil­lion. The vouch­er had orig­i­nal­ly been slat­ed for Y-mAbs’ ap­pli­ca­tion for Danyelza in neu­rob­las­toma, and it wasn’t used for the FDA’s ap­proval in No­vem­ber. In what could be an early shot in the battle against drugmakers that whiff on confirmatory studies to support accelerated approvals, the FDA ordered Bristol Myers Squibb late last year to give up Opdivo’s approval in SCLC. Now, Merck is next on the firing line — are we seeing the FDA buckling down on post-marketing offenders? Merck has withdrawn its marketing approval for PD-(L)1 inhibitor Keytruda in metastatic small cell lung cancer as part of what it describes as an “industry-wide evaluation” by the FDA of drugs that do not meet the post-marketing checkpoints on which their accelerated nods were based, the company said Monday. The global pandemic may have roiled economies, killed hundreds of thousands and throttled entire industries, but the only effect it had on biopharma venture investing was to help turbocharge the field to giddy new heights. Below you’ll find the new top 100 venture investors in the industry, ranked by the number of deals they were publicly involved in, as tracked by DealForma chief Chris Dokomajilar. The numbers master then calculated the estimated amount of money they put into each deal — divvying up the cash by the number of players — to indicate how they managed their syndicates. CRLs. 483s. CBER, CDER and RWE. For biopharma professionals, these acronyms command attention because of the fundamental role FDA plays in drug development. Now Endpoints is doubling down on regulatory coverage, and launching a weekly report focusing on developments out of White Oak, with analysis and insight into what it all means. Coverage will be led by our new senior editor, Zachary Brennan. He joins Endpoints from POLITICO, where he covered pharma. Prior to that he was the managing editor for Regulatory Focus, a news publication from the Regulatory Affairs Professionals Society. GlaxoSmithKline and Vir Biotechnology were hopeful that one of their partnered antibodies would carve out a win after getting the invite to a major NIH study in hospitalized Covid-19 patients. But just like Eli Lilly, the pair’s drug couldn’t hit the mark, and now they’ll be left to take a hard look at the game plan. The NIH has shut down enrollment for GSK and Vir’s antibody VIR-7831 in its late-stage ACTIV-3 trial after the drug showed negligible effect in achieving sustained recovery in hospitalized Covid-19 patients, the partners said Wednesday. A little more than a week after BrainStorm acknowledged that regulators at the FDA had informed them that the biotech needed more data before it could expect to gain an approval for its ALS treatment NurOwn — while still touting a “clear signal” of efficacy and not ruling out an application — the agency has decided to clarify the record in a most unusual statement. The FDA statement amounts to a straight slap own, offering a different set of efficacy numbers from the company’s public presentation last November and ruling out any chance of statistical significance. All the pieces needed to trigger a third wave of Covid-19 vaccine supply to start washing over the US fell neatly into place over the weekend. After providing for a brief mime of regulatory judiciousness, the FDA stamped their emergency approval on J&J’s Covid-19 vaccine Saturday, adding to the Biden administration’s plan aimed at ending the pandemic in the near term — at least in the US. The CDC came through on Sunday with its stamp of approval and J&J is reportedly expected to start delivering vaccine sometime in the next few days. Over the last decade, drugmakers have proven JAK inhibitors can treat a smattering of immune-related diseases ranging from rheumatoid arthritis to Covid-19. Now Eli Lilly has pulled out a new one. Lilly and its biotech partner Incyte announced Wednesday that their JAK inhibitor baricitinib effectively regrew patients’ hair in a Phase III trial for alopecia areata, an autoimmune condition that can cause sudden, severe and patchy hair loss. Lilly didn’t break down the results from the 546-patient trial, but the primary endpoint was improvement on a standard score for alopecia symptoms. Soticlestat made it. Takeda is bringing the drug back into its fold more than four years after first entrusting the team at Ovid with the mid-stage clinical work. For all that — generating what they saw as positive Phase II data in Dravet syndrome and Lennox-Gastaut syndrome — the biotech has been rewarded with $196 million in upfront cash, with another $660 million reserved for regulatory and commercial milestones. FibroGen will likely have to delay its US rollout for roxadustat once again. In an unexpected move, the FDA is convening its Cardiovascular and Renal Drugs Advisory Committee to review the NDA in an advisory committee meeting. The date is yet to be confirmed. Just a few weeks ago, SVB Leerink analyst Geoffrey Porges predicted that the roxa approval could come ahead of the PDUFA date on March 20 — effusive despite already being let down once by the FDA’s extension of its review back in December. AstraZeneca, which is partnered with FibroGen on the chronic kidney disease-related anemia drug, disclosed regulators had requested further clarifying analyses of clinical data.
大约三年后,SteadyMed和它的前Pul ar-te hy-10的药物Trevyent,单位已经破灭,将该项目踢到了路边。 在周二提交给SEC的一份8-K表格中,UNITED表示,根据FDA上周的反馈,他们正在关闭Trevyent的生产。这一举措标志着该项目漫长而坎坷的道路的结束,该项目在2017年获得了RTF奖,去年4月获得了CRL,因为该项目的单位向新的TYVA-SO进军。 专家们已经决定了重新设计Trevyent产品和进行一项新的临床研究的单位。United写道,如果不这样做的话,就会在计划中增加签署协议,但可能会被证明不符合FDA的规定。 具体来说,FDA采取的是与制造商的泵设备的起诉。Trevyent我们是一个泵,以使单位的主要停留药物恢复模式的恢复。泵的性能,怎么可能,在专家的眼里是不合格的,单位说,机构想让公司改进“在专家看来”的性能。 所以单位决定把拖车扔进去。他们停止了这一项目的所有执行,因为它将无法继续移动。这标志着他们曾在2018年SteadyMed展会上从同行那里获得过一笔利润的项目的一个不合时宜的结束。 有了2.16亿美元的雄狮收购,United一直在寻找避免两家公司之间的冲突,通过特雷文特自己。在这个项目的基础上,SteadyMed当时一直在尝试将United的一项专利用于其三个项目:Remodulin、Tyva-So和Orenitram。 尽管United与Trevyent合并,并于2019年9月将其NDA转至美国注册表,但该机构在2020年4月的CRL中对其进行了评估。FDA曾表示,一些被指出的“尚未被装订成符合其标准的”,该部门当时说到。(美国食品和药物管理局曾表示,该部门曾表示,美国食品和药物管理局曾表示,该部门曾表示,美国食品和药物管理局曾指出,一些“尚未被装订成符合其标准的标准”。单位已经在一个新的球场上工作,周二的决定是因为它与FDA的一次会议,将特雷维特重新分配给了美国食品药品监督管理局。 这是最近几年来,在一系列管道挫折中,单位看到的最新情况。2019年,该公司在一项公司III期研究失败后,放弃了一种有希望的药物,即与Tyva公司的老药合用时,没有显示出合用的效果。然后,在二月份,研究小组发现,一种II/III期的三种病毒在一个身体中与一种标准的治疗--伊利诺特病毒--不能帮助小细胞肺癌患者更长的寿命。 单位购买取代了以一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位购买一个单位。这张凭单是在Neuroblastoma的Y-Mabs对Danyelza的援助中被指责的,而在No.Vember的FDA援助中却没有使用这张凭单。 去年年底,FDA命令百时美施贵宝放弃Opdivo在SCLC的审批,这可能是对那些利用验证性研究来支持加速审批的制药商的一次早期打击。现在,默克是下一个上火线的--我们看到FDA对上市后违法者的严厉打击了吗? 默克公司周一表示,该公司已经撤回了对转移性小细胞肺癌的PD-(L)1抑制剂Keytruda的上市批准,作为FDA对不符合上市后检查点的药物进行“全行业评估”的一部分。 这场全球大流行可能扰乱了经济,夺走了数十万人的生命,扼杀了整个行业,但它对生物制药风险投资的唯一影响是帮助该领域达到令人眩晕的新高度。 根据DealForma首席执行官Chris Dokomajilar的记录,以下是业内前100名风险投资家的排名,根据他们公开参与的交易数量排名。然后,数字大师计算出他们投入每笔交易的估计金额--按玩家人数分配现金--以表明他们是如何管理自己的辛迪加的。 CRLS.483s.CBER、CDER和RWE。对于生物制药专业人员来说,这些首字母缩略词之所以引起注意,是因为FDA在药物开发中起着重要作用。现在Endpoints正在加倍扩大监管覆盖范围,并推出一份关注White Oak公司发展的周报,并对这一切意味着什么进行分析和洞察。 报道将由我们新的高级编辑Zachary Brennan领导。他加入了POLITICO的端点,在那里他报道了制药。在此之前,他是管理事务专业人士协会的新闻刊物《管理焦点》的执行编辑。 葛兰素史克公司和Vir Biotechnology公司在接到NIH一项针对住院Covid-19患者的重大研究的邀请后,希望他们合作的抗体之一能够赢得胜利。但就像礼来一样,这对组合的药物不能击中目标,现在他们将被留在认真看比赛计划。 合作伙伴周三表示,NIH已经停止了GSK和Vir的抗体VIR-7831在其晚期Active-3试验中的注册,因为该药物在实现住院的Covid-19患者的持续康复方面显示出可忽略不计的效果。 一周多一点后,BrainStorm公司承认FDA的监管机构已经通知他们,该生物技术公司需要更多的数据才能获得ALS治疗NurOwn的批准--同时仍在吹捧疗效的“明确信号”,并不排除申请--该机构决定在一份最不寻常的声明中澄清记录。 FDA的声明完全是对自己的一记耳光,它提供的药效数据与该公司去年11月的公开声明不同,并且排除了任何统计意义的可能性。 引发第三波Covid-19疫苗供应开始席卷美国所需的所有部件都在周末顺利就绪。 在短暂地展示了监管的明智性后,FDA于周六紧急批准强生公司的新冠疫苗,为拜登政府旨在近期结束疫情的计划增添了一笔--至少是在美国。CDC于周日通过了批准,强生公司预计将在未来几天内开始提供疫苗。 过去十年,制药商已经证明JAK抑制剂可以治疗从类风湿性关节炎到COVID-19等一小部分免疫相关疾病。现在礼来又拿出了一个新的。 礼来及其生物技术合作伙伴Incyte周三宣布,他们的JAK抑制剂baricitinib在一项针对斑秃的III期试验中有效地修复了患者的头发。斑秃是一种会导致突然、严重和斑片状脱发的自身免疫性疾病。礼来没有对546名患者的试验结果进行分析,但主要终点是脱发症状标准评分的改善。 索蒂克莱斯塔特成功了。 在委托奥维德大学的研究小组进行中期临床工作四年多后,竹田正将这种药物重新纳入自己的阵营。由于所有这些--在Dravet综合征和Lennox-Gastaut综合征中产生了他们认为是积极的第二阶段数据--生物技术公司获得了1.96亿美元的预付现金,另外6.6亿美元用于监管和商业里程碑。 FibroGen很可能不得不再次推迟在美国推出roxadustat产品。 在一个意想不到的举动中,FDA正在召集其心血管和肾脏药物咨询委员会,在一次咨询委员会会议上审查NDA。日期尚待确认。 就在几周前,SVB Leerink的分析师Geoffrey Porges预测,roxa的批准可能会在3月20日PDUFA的日期之前--尽管FDA在去年12月延长了审查期,但这一批准已经让人失望了一次。阿斯利康与FibroGen合作开发慢性肾病相关贫血药物,该公司透露,监管机构已要求对临床数据进行进一步的澄清分析。