Lalo Flores is confident he’s found the way forward in cell therapy.
The feeling, to be sure, is not exclusive. Thanks to splashy companies like Allogene, Lyell and Artiva, biopharma is well aware of the promises of allogeneic therapies as next-generation alternatives to the autologous CAR-Ts that have already made it to the market. But fewer are talking about a finer distinction within that allogeneic group: Where do you find the cells to engineer and turn into a final off-the-shelf product?
While most have opted for donor cells, Century Therapeutics — where Flores is CEO — is banking on induced pluripotent stem cells.
Almost two years after launching with $250 million in cash and three employees, it’s collected $160 million to fuel the now 100-person team’s dash to the clinic.
In parallel with all the advances in cell engineering over the years, there’s been a similar evolution in the science of reprogramming skin or blood cells into an embryonic-like state and coaxing them into T or NK cells. Fate Therapeutics, a J&J-partnered pioneer now in the clinic, has done much to educate the field about it, and more recently we’ve seen startups like Notch emerge; Century wants to join them in the leading spot, Flores said.
The room for improvement, after all, is significant. Even in the few lymphoma indications where Kymriah and Yescarta have been approved, not all patients achieve a complete response and many who do would relapse.
“So clearly what that means is that there was not enough pressure on the tumor, there were enough cells that survived that treatment […] and that’s the reason patients relapse,” Flores said. “Our goal at Century is to increase the durability of the responses.”
Century is “platform agnostic” and therefore developing both CAR-T and CAR-NKs created with its iPSCs. The lead program is a CD19 CAR-T that they hope to steer into a human trial by 2022.
In addition to the antigen targets, the biotech has devised a combination of three genetic edits to prevent immune rejection of the therapeutic cells — opening up the possibility to dose several times until the patient responds.
With each new program, once they finalize all the modifications Century would isolate a single clone to start what they call a master cell bank.
“It may take you a couple of years to get to that point,” Flores said. “But once you have a master cell bank, that is the beginning of your manufacturing process. And from there it only takes you 30 days to make a manufacturing run that will supply thousands of doses.”
Ultimately, he envisions Century’s therapies to be stored as a frozen product in hospitals. The preclinical pipeline goes after a slate of hematological malignancies as well as solid tumors like glioblastoma, where they would deploy a bispecific CAR. The substantial backing from Versant and Bayer in its Series B has helped Flores recruit a experts in protein engineering, gene editing and manufacturing, with him managing the headquarters in Philadelphia, R&D chief Hyam Levitsky leading a team in Seattle and the group that was formerly Empirica Therapeutics setting up a third office in Canada after Century acquired the University of Toronto spinout.
More recently, it’s leased the space for an manufacturing facility in New Jersey, just 45 minutes from the HQ, adding in-house capacity to complement the existing production under a partnership with Fujifilm Cellular Dynamics.
“Each time you develop a product, you begin the whole process of learning how to scale up manufacturing,” he said. “Going to this journey, there’s many learnings and it’s really important to capture all those learnings within your own employees, your own scientists, or within your close partner as we have in FCDI.”
They could be taking those learnings to Nasdaq soon. Though Flores notes Century now has two to three years of runway, the Series C syndicate — Fidelity, the Federated Hermes Kauffman Funds, RA Capital, Logos Capital, OrbiMed, Marshall Wace, Qatar Investment Authority, Avidity Partners, and Octagon Capital — features well-known crossover players with whom he will be exploring an IPO.
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.
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.
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.
The outsourcing market for cell and gene therapies has stayed red-hot in recent months as some of the biggest CDMOs — and even major drugmakers — are diving in. Now, a Swiss CDMO wants a bigger piece of the action, and it’s looking to set up shop in Novartis’ fledgling life sciences hub in Stein.
Celonic plans to build a 91,500-square-foot manufacturing center focused on cell and gene therapies, next-gen vaccines and other “innovative” drugs as part of a two-part expansion at Novartis’ Rhine Valley hub, the CDMO said last week.
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.
Janux Therapeutics had kept a relatively low profile since being founded back in 2017 but burst onto the scene late last year when Merck plunked down more than $1 billion in promised milestones for its T cell engagers. Now, less than three months later, the small biotech has clinched its first round of private funding led by some prominent backers.
As it prepares its first programs for INDs, Janux completed a $56 million Series A on Wednesday morning, with Jay Lichter’s Avalon Ventures joining forces with new investors OrbiMed and RA Capital Management to fund the company. Janux will use the cash to primarily advance its T cell engagers targeting PSMA and TROP2, which are expected to hit the clinic in the first and second quarters of 2022, respectively.
La lo Flo-res相信他在牢房里找到了路。
当然，这种感觉并不明显。多亏了Allo-Gene、Lyell和Arti-Va这样的知名公司，Bio-Pharma很清楚Allo-Geneic There-a Pies作为Au-Tol-O-Gous Car的下一代企业的前景，这些企业已经准备好进入市场。但是，很少有人在谈论与这个有限基因集团的一个潜在的差异：你从哪里找到细胞来进行消化，然后转到一个独立的现成产品中去呢？
与这些年来细胞免疫的所有变化一样，在将皮肤或血细胞转化为细胞样状态并诱导其转化为T细胞或NK细胞的科学中也存在着一种类似的发展趋势。Fate Thera Peutics,一个现在在临床的J&J部分的Pio-neer,已经做了很多关于它的领域的教育，更多的是我们看到像Notch这样的明星出现；弗洛雷斯说：“Centu-ry希望和他们一起成为领头羊。”
CENtu-RY是“Plat-Form Ag-Nos”，因此CAR-T和CAR-NKs都与它的IP SCs相结合。主导项目是一辆CD19汽车-T，他们希望在2022年前转向一辆Hu-Man Trial。
最后，他的馅饼将作为冷冻产品存放在寄存的地方。前临床管道去了一个石板上的血细胞和解决像胶质瘤，在那里，他们会制造一个双PE的汽车。这位来自Versant和Bayer的副总裁帮助Flores重新组建了一位项目工程师，gene Editing和Man Fac,他在费城担任首席执行官，研发总监Hyam Levit Sky领导了一个团队，在Cana设立了三分之一业务的团队请求Toron的Uni-sit退出。
更重要的是，它为一个在新的工厂工作的人租赁了空间，离总部只有45分钟的路程，增加了内部包装，以与Fuji-Film Cel-lar Dy-namics合作。
他们可能很快就会把这些学到的东西拿到NAS DAQ上。尽管有两三年的经营之路，但Cen'tu're Sen'di'del'ty、Fed'er'at Her'mes Kauff'man Funds、RA'cap'i'tal、Lo'gos Cap'i'tal、or'bimed、Mar'shallwace、Catal'in'mentau'thor'i'ty、Avid'i'ty Parter'ners、Oc'ta'gonCap'i'tal都是知名的跨界投资者，他将与他们进行IPO。
Janux Therapeutics自2017年成立以来一直保持着相对低调的姿态，但去年年底，默克公司斥资逾10亿美元，承诺为其T细胞参与器提供一系列里程碑项目，这让Janux Therapeutics突然崭露头角。现在，不到三个月后，这家小型生物技术公司已经获得了由一些知名支持者牵头的第一轮私人融资。
Janux在为INDs准备第一个项目的同时，于周三上午完成了5600万美元的a轮融资，由Jay Lichter的Avalon Ventures联合新的投资者OrbiMed和RA Capital Management为该公司提供资金。Janux公司将利用这笔资金主要推进针对PSMA和TROP2的T细胞接合器，预计将分别于2022年第一季度和第二季度投入临床应用。