Category Archives: CAR-T

Novartis’ CAR T therapy Kymriah to become available on the NHS

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Novartis’ Kymriah is set to become the first CAR T therapy to become available on the NHS after it was revealed that the cancer treatment will be offered to children and young adults up to the age of 25 years old with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse.

While both Gilead and Novartis’ CAR T therapy were awarded marketing authorisation in the European Union just last week, NICE were quick to reject Gilead’s CAR T therapy Yescarta on the grounds that it was too expensive.

However Novartis’ one time cancer treatment is now set to become available through the UK’s national healthcare system.

Mari Scheiffele, Novartis Oncology General Manager, UK & Ireland, said: “This decision to make our revolutionary CAR-T therapy, Kymriah (tisagenlecleucel) available so soon after being licensed is the result of our close collaboration with NHS England and NICE, with flexibility shown by all parties to ensure young patients can access this life-saving treatment as quickly as possible.”

The custom made treatment, which uses an individual’s own immune cells to combat cancer, has the potential to extend survival and significantly improve quality of life for children and young adults whose prognosis is poor.

However the cancer therapy comes with a high price tag, costing $475,000 in the United States. Meanwhile the list price for Gilead’s alternative Yescarta is just $373,000 in the US. Nevertheless the price that has been negotiated between Novartis and NICE will be kept confidential.

SOURCE: www.pharmafile.com/news/518554

NICE rejects Gilead’s CAR-T, immediately after EU approval

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Novartis and Gilead’s CAR-T therapies have been approved in Europe – and the UK’s NICE immediately slapped down the latter, saying it is too expensive for regular NHS use in England and Wales.

Novartis’ CAR-T, Kymriah (tisagenlecleucel) has not yet been reviewed by NICE’s committees, as the cost-effectiveness body received the manufacturer’s dossier much later.

But if NICE’s decision on Gilead’s CAR-T (chimeric antigen receptor T-cell) therapy, Yescarta (axicabtagene ciloleucel) is anything to go by, Novartis may also have difficulties securing market access on England’s NHS.

In its document summarising its assessment of Gilead’s drug, NICE noted the good response rates, overall survival and progression-free survival data from clinical trials of Yescarta in diffuse large B-cell lymphoma and primary mediastinal B-cell lymphoma in people who have had two or more systemic therapies.

But NICE said there was a lack of comparator data with standard care of salvage chemotherapy, so it was unable to quantify the exact size of the drug’s benefit.

Gilead has kept the list price a secret, and NICE merely said that the cost per Quality Adjusted Life Year was in excess of £50,000, its upper limit for medicines given to patients at the end of their lives.

However the medicine does not come cheaply – in the US Gilead set a list price of $373,000, for the therapy, a single shot of a patient’s own T-cells, harvested and genetically modified to destroy certain blood cancer.

NICE’s independent assessment committee also considered whether Yescarta could be reimbursed on an interim basis by the Cancer Drugs Fund until new clinical data comes to light.

But the committee said Yescarta does not have plausible potential to be cost-effective.

Meindert Boysen, director of the centre for health technology evaluation at NICE, said: “CAR-T is an exciting innovation in very difficult to treat cancers, with a promise of cure for some patients.”

“We have been working with the companies involved, and with NHS England, with the aim of ensuring that patients in England are among the first to have access to these new treatments in Europe.”

“Although promising, there is still much more we need to know about CAR-T, and unfortunately, in this case, we are not able to recommend axicabtagene ciloleucel for use in the NHS in England at the cost per patient set by (Gilead’s subsidiary) Kite Pharma.”

However in the long term, it looks likely that CAR-T therapies will be available for NHS patients: as revealed by pharmaphorum earlier this year, the NHS has been doing extensive groundwork ahead of their approval, including setting up specialist centres.

The approved indications for Yescarta are adult patients with relapsed or refractory diffuse large B-cell lymphoma (DLBCL) and primary mediastinal large B-cell lymphoma (PMBCL), after two or more lines of systemic therapy.

Kymriah has been approved for the treatment of paediatric and young adult patients up to 25 years of age with B-cell acute lymphoblastic leukaemia (ALL) that is refractory, in relapse post-transplant or in second or later relapse; and for the treatment of adult patients with relapsed or refractory (r/r) diffuse large B-cell lymphoma (DLBCL) after two or more lines of systemic therapy.

SOURCE: www.pharmaphorum.com/news

Veteran analyst Eric Schmidt moves over to biotech as Allogene’s new CFO

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Longtime Cowen analyst Eric Schmidt is joining Arie Belldegrun’s latest CAR-T startup, Allogene, as its chief financial officer.

The announcement comes six weeks after Schmidt confirmed that he was considering a job offer from the company, which is working on off-the-shelf CAR-T therapies.

“Eric is an enormously talented individual with a rare blend of insight into multiple facets of the industry and deep relationships within the financial community. We are very fortunate to have him join the Allogene executive team and look forward to him applying his expertise to our vision for allogeneic cell therapy,” said Allogene CEO and President, David Chang, M.D., Ph.D, in a statement.

Schmidt joined Cowen in 1998, where he served as managing director and senior biotechnology analyst. Though he’s leaving his post at Cowen, he will stay on as a senior advisor to the company.

After creating the first CAR-T for non-Hodgkin lymphoma and selling their company off to Gilead, the former Kite Pharma executives Belldegrun and Chang returned for chapter two—allogeneic, or off-the-shelf CAR-T. They launched the startup in April, with $300 million in series A capital and a suite ofe early-stage allogeneic CAR-T assets, licensed from Cellectis, by way of Pfizer.

Pfizer licensed 16 CAR-T assets from Cellectis in 2014 for $80 million up front and another $185 million per product up for grabs, making the deal potentially worth as much as $2.9 billion. A year later, Pfizer and Servier gained the rights to another CAR-T candidate, UCART19, which has now become Allogene’s lead asset, with phase 2 trials slated to start in 2019.

While Kite and Novartis earned the first approvals for CAR-T treatments in 2017, there remains much room for improvement. Their therapies, Yescarta and Kymriah, are both indicated for blood cancers, with most CAR-Ts seeing limited success in solid tumors. Moreover, CAR-T cells are made by collecting a patient’s T cells, modifying them to better target and destroy cancer and then putting them back into the patient. This makes manufacturing complex and time-consuming. An allogeneic approach uses donor cells, which can be stored and used off the shelf.

“I’ve watched and analyzed dozens of companies as they worked to bring innovative and transformative new therapies to the market,” Schmidt said. “I have greatly admired the team and investors behind Allogene and consider myself very fortunate to be in a position to help make a difference in the lives of patients who are facing cancer.”

SOURCE: www.fiercebiotech.com

Gilead signs $3bn-plus gene-editing deal with Sangamo

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Gilead Sciences has claimed another stake in the emerging cell therapy sector with a $3bn agreement to use Sangamo Biosciences gene-editing platform for new cell-based cancer therapies.

The company seems determined to stay in the forefront of the pharma industry’s push into cell therapies, with the latest deal coming shortly after it acquired CAR-T specialist Kite Pharma for $12bn and almost immediately bolted on another CAR-T tech through the takeover of Cell Design a few weeks later.

Sangamo is getting $150m upfront from Gilead in the deal, which will see Kite claim an exclusive license to use the biotech’s zinc finger nuclease (ZFN) technology to develop up to ten off-the-shelf (allogeneic) as well as autologous CAR-T therapies. It also stands to receive up to $3bn in milestones – $300m per product – as well as tiered royalties on sales.

Kite will be responsible for all development, manufacturing and commercialisation of products under the collaboration, and will be responsible for agreed upon expenses incurred by Sangamo.

Gene-editing techniques are used to modify the cells – either harvested from patients or taken from donor stocks – that are infused into cancer patients in order to mount an immunotherapeutic assault on cancers. The deal with Sangamo means rivals in the CAR-T category such as Novartis, Celgene/Juno and off-the-shelf specialist Cellectis won’t be able to use the ZFN technology in their own gene-editing toolboxes.

ZFN is a gene-editing approach that uses a DNA-cutting nuclease enzyme attached to zinc finger -binding proteins to recognize and edit specific sequences of DNA. Other techniques include CRISPR/Cas9 – used by Novartis and Juno – and Cellectis’ favoured TALENS.

However, according to CEO Sandy Macrae, Sangamo has made significant strides in improving the precision, efficiency and specificity of ZFN, which means the technique now “sets the standard on what therapeutic genome editing should be”.

He said that Kite’s “financial strength and clear determination” to bring new cellular therapies products forward makes it an ideal partner for ZFN in this setting.

Kite highlighted the potential of the technology for developing allogeneic CAR-Ts, which have been put forward as potentially a major advance over autologous therapies, as they should be much quicker and cheaper to deliver – an important consideration given the current debate over the escalating costs of cancer treatment.

“The emergence of gene editing as a tool to edit immune cells holds promise in the development of therapies with potentially improved safety, efficacy and efficiency,” commented Gilead’s CEO John Milligan.

SOURCE: www.pmlive.com/pharma_news