Presage teams with Mer ck on its Phase 0 test ing; Kem Pharm AD HD drug wins ap proval in chil dren aged 6 and up

在其第0阶段试验中采用Mer ck进行预演;Kem Pharm AD HD药物在6岁及6岁以上的儿童中获得成功

2021-03-04 03:30:12 ENDPOINTS NEWS


Seat­tle-based Presage Bio­sciences, which ap­proach­es drug de­vel­op­ment through its mi­cro­dos­ing plat­form, has some new part­ner­ships and cash to come with them. Presage closed a $13 mil­lion fi­nanc­ing round Tues­day, aim­ing to ex­pand its net­work of clin­i­cal tri­al sites and ad­vance de­vel­op­ment of its mi­cro­dos­ing in­jec­tion de­vices. They al­so closed part­ner­ship deals with Mer­ck and Mav­er­ick Ther­a­peu­tics. The fi­nanc­ing in­clud­ed $7 mil­lion from new in­vestors, in­clud­ing the Lab­Corp Ven­ture Fund, Bris­tol My­ers Squibb, and In­Harv Part­ners. An ad­di­tion­al $6 mil­lion con­vert­ible note from Take­da Ven­tures will con­vert to eq­ui­ty. Presage’s big idea cen­ters around what they call Phase 0 test­ing, which us­es mi­cro­dos­es of mul­ti­ple drugs and com­bos on pa­tients to gauge their phar­ma­co­dy­nam­ic ef­fects. Their tech al­lows re­searchers to pep­per a tu­mor with mul­ti­ple ther­a­pies while in use by pa­tients, pro­vid­ing a bet­ter snap­shot of rel­a­tive ac­tiv­i­ty. Tues­day’s new re­search col­lab­o­ra­tions with Mer­ck and Mav­er­ick will uti­lize Presage’s plat­form to eval­u­ate new on­col­o­gy pro­grams in these Phase 0 tri­als, bring­ing their to­tal num­ber of part­ners to five. Kem­Pharm scored a new ap­proval on Wednes­day, and it’s send­ing their stock soar­ing. The Cel­e­bra­tion, FL-based biotech notched the FDA OK for Azs­tarys, a once-dai­ly treat­ment for AD­HD in chil­dren aged six and old­er. Azs­tarys, for­mer­ly dubbed KP415, con­sists of serdexmethylphenidate, Kem­Pharm’s pro­drug of the drug Fo­calin, co-for­mu­lat­ed with an im­me­di­ate-re­lease ver­sion of the prod­uct. Shares of Kem­Pharm $KMPH sky­rock­et­ed as much as 90% in ear­ly Wednes­day trad­ing, set­tling in at around a 70% in­crease lat­er in the morn­ing. Azs­tarys will be com­mer­cial­ly avail­able as soon as the sec­ond half of this year, and that process will be led by a sub­sidiary of Gur­net Point Cap­i­tal. The ap­proval will al­so trig­ger a mile­stone pay­ment from Gur­net Point Cap­i­tal to Kem­Pharm, which may to­tal up to $468 mil­lion. AD­HD has been a lu­cra­tive mar­ket for drug­mak­ers, ac­count­ing for near­ly $18 bil­lion of rev­enue in 2019, Kem­Pharm said. The pe­di­atric field has proven tough for com­peti­tors, though, with the FDA spurn­ing Su­per­nus’ pitch last No­vem­ber. Reg­u­la­tors cit­ed con­cerns about an in-house lab used to an­a­lyze their ex­per­i­men­tal drug. 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. 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. Bob Nelsen’s new, state-of-the-art drug manufacturing initiative is taking shape. Just 3 months after gathering $800 million of launch money, a dream team board and a plan to shake up a field where he found too many bottlenecks and inefficiencies for the era of Covid-19, Resilience has snapped up a pair of facilities now in line for a retooling. The company has acquired a 310,000-square-foot plant in Boston from Sanofi along with a 136,000-square-foot plant in Ontario to add to a network which CEO Rahul Singhvi says is just getting started on building his company’s operations up. The Sanofi deal comes with a contract to continue manufacturing one of its drugs. One of Europe’s most high-profile biopharma investors is getting $540 million to invest in new crossover deals for late-stage companies. The Paris-based VC says the fresh Sofinnova Crossover Fund raise positions them as the “largest crossover investor in Europe dedicated to late-stage biopharma and medtech investments.” They got a leg up in France after winning a special “Tibi” designation from the French government, giving them access to a pool of €6 billion that helped them gain an edge with institutional investors. Since they were founded close to 50 years ago, the venture group has backed more than 500 companies and currently has more than €2 billion under management.
基于座位的Presage Bio-Sciences公司通过其微型平台来推动药物研发,有了一些新的零部件和现金。 Presage在周二前后关闭了一个1300万美元的LION Fience,目的是退出它的三个临床站点网络,并在Jection De上进行销售。他们与merck和maverick thereutics达成了零配件交易。 该基金从new In'vestors,In'cluding The Lab'Corp Ven'ture Fund,Bris tol My'ers Squibb和In'harv Part公司获得了700万美元。从Take da venures开出的一张价值600万美元的假钞将会兑换成Equuity。 Presage的大主意围绕着他们所谓的0阶段试验,即对多种药物和药物进行微观测试,以评估其药效。他们的技术低能让搜索者在用户使用时与多个其他馅饼一起打气,从而更好地拍摄用户的产品。 周二与Merck和Maver'ick的新研究公司实验室将使用Presage的平台来评估这些第0阶段的三个项目中的新的研究公司项目,使他们的部分数量达到5个。 Kempharm在周三获得了一个新的收益,这使得他们的股票飙升。 这家总部位于佛罗里达州的生物技术公司获得了FDA对Azstarys的认可,这是一种曾经用于治疗6岁和6岁儿童AD HD的药物。AZS·TARYS,简称KP415,是哌酸塞地克甲酯的产品,是药厂生产该产品的原料药,同时也是该产品的中间体。 Kempharm Kmph Skyrock的股价在周三的交易中上涨了90%,在早上的交易中上涨了70%左右。“Kempharm Kmph Skyrock”的股价在周三的交易中上涨了90%。“Kempharm Kmph Skyrock”的股价在周三的交易中上涨了90%。AZStarys将在今年下半年开始使用,这一过程将由Gurnet Point Capital的一个分支机构领导。 这款产品将从Gurnet Point Capital到Kempharm提供一英里长的支付,最高可达4.68亿美元。 Kempharm说,AD-HD已经成为药品制造商的一个重要标志,据统计,2019年的销售额接近18亿美元。 然而,事实证明,体育领域对企业来说很艰难,因为FDA最近一次否决了企业的提议。管理人员介绍了一个内部实验室用来检验他们以前的药物的情况。 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。 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。 鲍勃尼尔森的新的,最先进的药物制造计划正在形成。 仅在筹集了8亿美元的启动资金、一个梦之队董事会以及一个在Covid-19时代发现有太多瓶颈和低效率的领域进行改革的计划之后的三个月,复原力就抢购了两个正在等待重新改造的设施。 该公司从赛诺菲手中收购了位于波士顿的一家31万平方英尺的工厂,并在安大略省收购了一家13.6万平方英尺的工厂,以补充公司的运营网络。该公司首席执行官拉胡尔•辛格维表示,公司的运营才刚刚起步。与赛诺菲的交易伴随着一份继续生产其一种药物的合同。 欧洲最受瞩目的生物制药投资者之一将获得5.4亿美元,投资于新的跨界交易,为处于后期阶段的公司投资。 这家总部位于巴黎的风投公司表示,新的Sofinnova跨界基金融资使他们成为“欧洲最大的跨界投资者,致力于后期生物制药和医疗技术投资”。 他们在法国获得了法国政府颁发的“Tibi”特许权,从而获得了60亿欧元的资金池,帮助他们在机构投资者中获得了优势。自50年前成立以来,这家风险集团已经支持了500多家公司,目前管理着超过20亿欧元的资金。