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
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.
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。
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。
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。
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。