Oxford BioTherapeutics to research cell therapies for Gilead’s Kite

Gilead Sciences’ Kite unit has teamed up with the UK’s Oxford BioTherapeutics (OBT) to develop a new clutch of cell therapy products for solid tumours and blood cancers.

The partnership covers up to five oncology targets identified by OBT using its discovery platform, and the UK firm will try to develop antibodies against them. Kite and Gilead will then develop and commercialise therapies based on these targets or antibodies.

“Selecting the right target is fundamental for the successful development of first-in-class cell therapies,” said OBT chief executive Dr Christian Rohlff.

“We are delighted that Kite, the global leader in cell therapy, has recognised the potential of OBT’s OGAP discovery platform and antibody capabilities through this partnership.”

Under the agreement, OBT will receive an undisclosed upfront payment and may receive receive additional payments based on the achievement of discovery, clinical, and regulatory milestones, as well as royalties on any sales coming down the line.

Gilead acquired Kite in 2017 for almost $12 billion, at a time when enthusiasm for cell therapies for cancer, and particularly CAR-T, was riding high on the back of unprecedented, life-saving efficacy in trials involving patients with few treatment options left.

The commercial reality for CAR-Ts has been more challenging, and as the pipeline has become more crowded Gilead has cut the value of the experimental assets it acquired along with Kite on more than one occasion to the tune of around $1.6 billion.

The main draw for the Kite takeover for Gilead was lead CAR-T Yescarta (axicabtagene ciloleucel), approved for large B-cell lymphoma shortly after the company made its move, which was joined by Tecartus (brexucabtagene autoleucel) for mantle cell lymphoma last July.

Problems with tolerability, reimbursement and a complex manufacturing process has pegged back sales of CAR-Ts from earlier expectations, but Yescarta reached $450 million in 2019, and nearly matched figure that in the first nine months of 2020, with Tecartus adding a modest $9 million to the pot.

Gilead and Kite remain convinced that cell therapy can become the cornerstone of cancer treatment, however, and the deal with OBT isn’t the first that has boosted Kite’s portfolio.

In April last year, for example, Kite licensed a suite of antibodies from Teneobio against BCMA – a key target for multiple myeloma therapy – that will be used to generate CAR-T treatments for the blood cancer.

Last September it forged a second alliance with HiFiBiO Therapeutics to find novel targets and antibodies for acute myeloid leukaemia (AML), adding to an earlier agreement focusing on T cell receptor (TCR) therapies for cancer.

In 2018 it also formed a partnership with Gadeta of the Netherlands to develop new forms of CAR-T with greater activity against solid tumours.

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Why cell therapy manufacture is a team sport

Louis van de Wiel, Vice President, Site Head EU Manufacturing, Kite, a Gilead Company, reveals the complexity that sits behind the process of individualised cell therapy – and why team culture makes it work

This thought leadership series has been paid and developed by Kite, a Gilead Company.

In 2018, we were preparing to build a European facility to produce individualised cell therapies for the treatment of cancer.

Roll on two years and the team has achieved what at the time appeared a major challenge, putting 1,000 tonnes of steel, 1,800 solar panels and 176km of network cable into the creation of a centre of excellence for cell therapy near Amsterdam in the Netherlands.

In my experience, completing the design and build of a facility such as this, through to qualification, licence and becoming fully operational, would normally take four to five years. Our ambition was always to do this within two years, a goal we achieved despite the unprecedented challenge of a global coronavirus pandemic.

Indeed, while undoubtedly putting new hurdles in our path, the arrival of COVID-19 into our lives has transformed our business and operations by presenting an opportunity to be adaptable, flexible and responsive – and to continually evaluate and mitigate risk.

“There is huge complexity involved in cell therapy manufacture, with hundreds of personnel responsible for ensuring the quality and supply of an individual patient’s cells”

It’s been a complex process that’s required highly technical and skilled personnel. Not only did we build a specialised facility from the ground up, but we built an organisation, from 10-15 people two years ago to more than 400 now. We put energy and emphasis into creating the right team and a culture where everyone understands the values and drivers, allowing us to operate in a collaborative and cohesive way.

Now the new €130 million, 19,000m2 manufacturing facility near Amsterdam is able to support delivery of up to 4,000 cell therapies each year for eligible cancer patients across Europe.

But backtrack to August 2018 and the very first European patients were also receiving treatment, part of an expertly crafted operation that ran in parallel to the build. Our supply chain group worked with the existing US team to manage the shipment of patients’ cells to the US for modification and their return for treatment.

Having a fully operational site in Europe versus the US has several advantages; reducing transportation time, strengthening the chain of custody and, potentially, cutting lead time to the patient by approximately one week. This allows us to potentially provide the therapy quicker for eligible cancer patients who have stopped responding to or have progressed despite other treatments. At this stage of their disease, for patients who have no other options, a week can make a difference.

The journey of the cell

The very nature of cell therapy manufacture means employees work in tightly controlled environments to ensure adherence to good manufacturing practice standards and, ultimately, to ensure the quality and integrity of the product.

Ultimately, it’s a team sport between Kite and Gilead and the 100-plus qualifying hospitals across Europe, all of which have been individually trained and assessed to ensure they are fully compliant with the necessary procedures and meet exacting standards.

So, what does the journey of the cell look like?

To achieve consistent, timely delivery of a high-quality product requires a robust and efficient approach to engineering patient’s own T cells, which in itself encompasses apheresis, cell modification and final formulation – coupled with rigorous quality control testing throughout – reflecting the highly complex nature of the manufacturing process.

Understandably, teamwork is vital and requires an integrated network and seamless communication between Kite and the treating hospital. The journey starts with the hospital making a treatment reservation through KiteKonnect and shipping of the apheresis kit to enable the process of extracting the patient’s own white blood cells, kickstarting both the chain of identity and chain of custody.

Here, our quality and supply chain experts are integral to every stage of the cell therapy manufacturing continuum to ensure the product is returned to the patient in a timely manner.

As soon as apheresis has completed, the cells are shipped in temperature-controlled conditions to our facility near Amsterdam where they are assessed for quality and condition. One patient equals one individual treatment, so it is critical to preserve the chain of custody and chain of identity to ensure the product comes back to the same patient.

Why chain of custody and identity is critical

The chain of custody and chain of identity must, therefore, go hand-in-hand. In this way, not only do we know which cells belong to which patient, but we have precise location and up-to-the-minute feedback on storage conditions to ensure quality and safety is paramount at all times.

Once the cells have completed this first stage, the manufacturing process can begin, with T cell selection, activation, and genetic modification using viral vector technology to ensure the ability to recognise the patient’s cancer cells. Cell expansion follows to multiply the modified cells into their millions.

Further critical quality testing then takes place to ensure the cells are of a required standard and to create a finished purified product, which will be stored and returned to the originator hospital in temperature-controlled conditions (see diagram below).

Several quality attributes will be tested at this stage and the cells must meet these rigorous criteria and specifications. There is huge complexity involved in cell therapy manufacturing, with hundreds of personnel responsible for ensuring the quality and supply of an individual patient’s cells.

As part of this process, the supply chain team simultaneously coordinate with the hospital to prepare the individual so when the cells are infused back to the patient they are primed to potentially fight the cancer.

Individualising the approach

In stark contrast to basic biopharmaceutical products with a robust starting material, the cells of a patient with cancer who has already undergone multiple treatments will not have the same quality. Consequently, there can be unforeseen hurdles during the process and I am proud that the team has managed each situation to safeguard the patient’s cells and ensure they receive treatment in an efficient and timely way.

This is particularly important when you consider the turnaround for each individualised product from starting material to the patient is typically four weeks – versus months or even years for a standard biopharmaceutical product.

Additionally, each patient equals one product batch – we do not keep inventory – and the potential impact on the patient if something happens to that batch is why we are so passionate. From quality manufacturing, facility engineering, supply chain, we’re driven to make sure the batch is returned to the patient safely and effectively.

What of the future? For me, it’s all about leadership, clarity, direction, and commitment of the entire team. It’s about the opportunity to be involved in an innovative field of cancer therapy where the body is stimulated to fight cancer cells. It’s about optimising the manufacturing process to become more effective and efficient. But, most of all, it’s about the patients, their care partners and families

 

This was supported by Kite, a Gilead Company
UK-CTH-2020-11-0075 | Date of preparation: December 2020

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Chasing BMS, J&J files BCMA CAR-T for multiple myeloma to FDA

Johnson & Johnson’s Janssen unit has filed a rolling submission for its multiple myeloma CAR-T ciltacabtagene autoleucel (cilta-cel) to the FDA, in hot pursuit of Bristol-Myers Squibb’s delayed rival therapy.

Cilta-cel – which targets B-cell maturation antigen (BCMA) – has been submitted as a treatment for patients with relapsed or refractory myeloma, an incurable form of blood cancer, specifically in heavily treated adults who currently have few therapeutic options.

BMS’ CAR-T idecabtagene vicleucel (ide-cel) is going after the same indication and is due for a verdict from the US regulator on 27 March 2021.

The rolling application allows portions of the dossier to be filed as they become available, shortening the review time, although BMS is still in pole position to bring a multiple myeloma cell therapy to the US market, despite a setback earlier this year when the FDA rejected the filing for the CAR-T, asking for more data.

Cilta-cel’s filing triggers a $75 million payment from J&J to its development partner Legend Biotech under the terms of a deal signed in December 2017. It is J&J’s first application for a cell therapy.

The submission is based on results from phase 1b/2 CARTITUDE-1 study, which showed an overall response rate of 97% with cilta-cel, with 67% of patients achieving a stringent complete response despite having received a median of five prior therapies.

The median duration of response and progression-free survival still have not been reached in the study, suggesting the effect is long-lasting, according to Janssen.

Like other CAR-Ts the therapy isn’t without risk however, and there were 14 deaths reported during the study, including six due to complications related to treatment itself.

With all the caveats about comparing different trials, cilta-cel’s results look a little better than those of ide-cel’s KarMMa study, which had an overall response rate of 73%, including 33% complete responses, among patients who had a median of six prior treatments.

BMS is also developing another BCMA directed CAR-T – orvacabtagene autoleucel (orva-cel) – which generated positive results in the EVOLVE trial this year.

Ide-cel and cilta-cel aren’t the first BCMA-targeted drugs for myeloma, however. GlaxoSmithKline’s antibody-drug conjugate (ADC) Blenrep (belantamab mafodotin) has already been approved in the US and Europe, making almost $11 million in third-quarter sales in its first few weeks on the market.

There are other BCMA drugs coming through the pipeline as well, including a string of bispecific antibody therapies from Regeneron, Amgen, J&J and others that could sidestep the cumbersome and risky treatment and manufacturing process for CAR-T therapies.

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Catamaran joins the CAR-NK fleet, raising $42m in first round

Catamaran Bio has weighed anchor with a $42 million first-round financing that will be used to pull its off-the-shelf natural killer (NK) cell therapies for cancer through early-stage development.

The Cambridge, Massachusetts-based biotech is the latest player in the emerging field of chimeric antigen receptor (CAR) NK cell therapies, which unlike the current generation of CAR-T therapies can be sourced from healthy donors, rather than patients themselves.

CAR-NK therapies also have the potential to be used to treat solid tumours, something that has so far eluded CAR-Ts, which to date have only been approved for blood cancers. Solid tumours tend to be a tougher treatment proposition for cell therapies, as they are harder to penetrate and are often swamped with immunosuppressive factors.

CAR-NK therapies consist of NK cells that have been modified with a CAR molecule, which allow them to bind to and attack a cancer cell.

Earlier this year, researchers at MD Anderson Cancer Centre in the US – a pioneer of CAR-NK – said they have achieved a 73% response rate with a CD19-targeting therapy derived from donated cells in patients with relapsed or refractory non-Hodgkin’s lymphoma and chronic lymphocytic leukaemia.

Catamaran’s cash injection comes just nine months after Catamaran was founded to build on research conducted by its scientific founders – George Washington University’s Catherine Bollard and University of Minnesota’s Branden Moriarty.

The other founders include Houman Ashrafian, Tim Harris and Kevin Pojasek of SV Health Investors, and Obsidian Therapeutics and Serien co-founder Vipin Suri, who will serve as Catamaran’s chief scientific officer.

Catamaran’s proprietary platform – called Tailwind – consists of a novel way to design and engineer improved CAR-NK cells using synthetic biology.

One element is the “architecture” or the CAR molecule, and Catamaran says it has novel structures in play that stimulate production of immune cytokine molecules by the CAR-NK cell, enhancing the cell-killing action.

Another is the delivery of molecule switches to CAR-NK cells, used to activate biological pathways that can help overcome the immunosuppressive microenvironment around tumours and improve recruitment of other elements of an immune response.

Finally, Tailwind includes a non-viral delivery system for engineering the CAR-NK cells based on transposons that make them easier and potentially cheaper to manufacture.

Viral vectors are limited in the size of genetic payloads they can deliver, but using transposons – a “jumping” DNA sequence that can change its position within a genome – sidesteps this issue and allow multiple CARs and switches to be delivered in one go.

Catamaran has two lead CAR-NK programmes at the lead optimisation stage, both of which target the same, undisclosed antigen that is found on both solid tumour and blood cancer cells. The lead programme is targeting a blood cancer.

“Catamaran is focused on expanding the frontier of cell therapies to treat solid tumours and provide transformative benefit to cancer patients,” said Suri.

“We are doing this by creating allogeneic cell therapies that harness the innate cancer-fighting power of NK cells and enhancing them with new biologically-powerful attributes from our leading-edge technologies,” he added.

Sofinnova Partners and Lightstone Ventures co-led the Series A, with SV Health Investors, Takeda Ventures and Astellas Venture Management also taking part.

Other groups developing CAR-NK therapies include Takeda, which has partnered with MD Anderson, as well as Artiva, Nkarta, Fate Therapeutics, oNKo-innate, Kuur Therapeutics, and ONK Therapeutics.

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Sanofi to acquire Kiadis and NK cell tech for $353 million

Sanofi is to acquire Kiadis, a biotech specialising in therapies based around ‘off the shelf’ natural killer (NK) cells, for 308 million euros ($353 million).

The French pharma is buying Kiadis for 5.45 euros per share in cash, an offer price representing a premium of 272% over the biotech’s closing price on Friday evening on Amsterdam’s Euronext market.

It’s a substantial price to pay for the small pharma. Although the company’s share price has been notoriously volatile, the NK-cell technology is still largely unproven in clinic.

Allogeneic, or ‘off the shelf’, cell therapies are derived from banks of cells, and potentially offer the therapeutic benefits seen with autologous cell therapies derived from a patient’s own bodies but without the complex, costly and lengthy manufacturing process.

Autologous CAR-T cancer therapies based on T-cells are already on the market from Novartis and Gilead, but have had a slow start in terms of sales as health systems struggled to come to terms with their high price and the logistics of manufacturing them and delivering them to patients.

While Kiadis’ technology is mainly intended for cancer, the biotech announced plans in August to develop a NK-cell therapy for COVID-19, sending its shares soaring before they fell back due to profit-taking.

This followed a separate deal with Sanofi in July, where the pharma licensed Kiadis’ pre-clinical drug K-NK004 for multiple myeloma.

Kiadis has K-NK002 in phase 2 development to prevent patients with acute myeloid leukaemia (AML) and myelodysplastic syndromes from relapsing after transplant.

Also in phase 1 development is K-NK003, for patients with relapsed or refractory AML.

Kiadis is drawing up plans to begin a phase 1/2a clinical trial of KNK-ID-101, its COVID-19 therapy in high risk patients, with funding from the French government.

Acquiring Kiadis outright makes sense for Sanofi, which already has a foothold in cancer immunotherapy thanks to its Libtayo (cemiplimab), developed in partnership with Regeneron and already approved in a form of skin cancer.

Shareholders in Kiadis will likely be pleased with the substantial premium agreed and the deal has the unanimous support of its board.

Funds managed by Life Sciences Partners have committed to support the offer, accounting for 18.3% of shares in the Netherlands-based biotech.

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Bayer buys cell & gene therapy firm AskBio for $4 billion

Bayer is making clear its ambitions in cell and gene therapy with a $4 billion acquisition of US biotech Asklepios BioPharmaceutical, also known as AskBio.

AskBio’s portfolio includes pre-clinical and clinical stage candidates for the treatment of neuromuscular, central nervous system, cardiovascular and metabolic diseases.

Through the acquisition, Bayer will gain full rights to the company’s gene therapy technology and manufacturing platforms, including AskBio’s adeno-associated virus (AAV)-based platform, which has already demonstrated applicability across different therapeutic areas. 

The company’s most advanced programmes are in Pompe disease – a rare genetic disease causing buildup of a sugar molecule inside cells – Parkinson’s disease and congestive heart failure.

Under the terms of the agreement, Bayer will pay $2 billion, with a further $2 billion marked out for potential success-based milestone payments.

According to the company’s statement, some 75% of the potential success-based milestone payments are expected to be due during the course of the next five years and the remaining amount late thereafter.

Last year Bayer fully acquired another cell therapy company, BlueRock Therapeutics – which was created in 2016 via a joint venture between Bayer and Versant Ventures, and is also working on Parkinson’s Disease therapies. 

Like with BlueRock, Bayer says it wants AskBio to operate on an “arm’s-length basis” to “persevere its entrepreneurial culture as an essential pillar for nurturing successful innovation”.

Reuters reports that AskBio and BlueRock will exchange information and collaborate but will each operate as independent companies. In light of this, AskBio’s five main owners have pledged to remain with the firm.

“We are staying on board because of the unique structure that Bayer has provided,”  said CEO and co-founder Sheila Mikhail.  We’ll have the ability to make our science decisions.”

Cell and gene therapy is viewed as one of the most promising areas in pharma, with several biotech and big pharma firms making plays in the area.

These therapies offer new treatment options for many currently untreatable diseases, particularly genetic diseases caused by a single genetic defect.

AskBio has licensed some of its experimental drugs to external partners, such as Pfizer, which recently won fast-track designation in the US for a Duchenne Muscular Dystrophy candidate, PF-06939926, that was invented by AskBio.

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Triumvira Immunologics raises $55M in Series A funding round for new form of cancer cell therapy

The company hopes to use the money to bring its T-cell antigen coupler cell therapy technology – currently in preclinical development – into human clinical trials. German drugmaker Bayer’s venture capital arm led the round.

Patient-to-Patient Supply for Cell Immunotherapies Summit

Achieving Standardization & Operational Excellence in Vein-to-Vein Supply

As an increasing number of candidates enter the clinic and promise to be a mainstay in the future of cancer treatment, cell therapies pose unique supply chain challenges.

From temperature and time sensitivity, to patient-facing delivery and chain of identity, these supply chain challenges must be overcome to ensure the delivery of safe and efficacious cell therapies at scale.

The Patient-to-Patient Supply for Cell Immunotherapies Summit (October 29-30, 2020) is a unique two-day digital platform discussing best practises in implementing robust vein-to-vein supply chains.

Our community of supply chain experts are looking forward to connecting with you in the digital world, including:

  • Christopher Baldwin, Supply Chain Director, Cell & Gene Therapy¸ GSK
  • Reggie Foster, Senior Director Labelling, Logistics & Packaging, Kite: a Gilead Company
  • Sheng Lin-Gibson, Chief, NIST Biosystems & Biomaterials Division
  • Jean Stanton, Director Commercial Apheresis, Johnson & Johnson
  • Richard McFarland, President, Standards Coordinating Body
  • Neeraj Shah, Director Supply Chain Excellence, Bristol-Myers Squibb
  • Kawa Chiu, VP CMC Supply Chain, Lyell Immunopharma
  • Alexandra Gomez, Associate Director, Patient Services Operations, bluebird bio

Get your copy of the brand-new agenda for the full list of speakers and sessions.

Join the key speakers sharing industry-leading expertise and reconnect with your community to ensure your team is equipped to navigate this pandemic with robust, commercially ready supply chains.

Join this timely discussion by registering online today.

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2nd Macrophage-directed Therapies Summit

Harnessing the Potential of Macrophage Therapies Using Small Molecule, Antibody & Cell Therapy based Approaches for Oncology and Beyond

The $4.9 billion acquisition of Forty Seven by Gilead is an example of the explosive excitement and investment into macrophage-directed therapies, utilizing CD47 checkpoint blockades, repolarization approaches and engineered macrophages, that we have witnessed in recent months.

The digital 2nd Macrophage-directed Therapies Summit (October 27-29) is a comprehensive industry focused meeting dedicated to advancing current CD47 and CAR-macrophage therapies alongside the new and exciting macrophage targets being identified and studied.

View the full event guide to see all the leading speakers and their agenda sessions.

Some highlights on the 2020 agenda:

  • Nicholas Poirier, Chief Scientific Officer, OSE Immunotherapeutics will explore a novel ‘don’t eat me’ target regulating macrophages phagocytosis and dendritic cell antigen presentation
  • Michael Klichinsky, Co-Founder & VP, Discovery, Carisma Therapeutics will discuss genetically engineering CAR macrophages & CAR monocytes
  • Yang Liu, Founder, President and Chief Scientific Officer, Oncoimmune will explore the CD24–Siglec-10 pathway as a novel innate immune checkpoint to modulate anti-tumor immunity
  • Gloria Lin, Associate Director of Translational Research, Trillium Therapeutics Inc will reveal emerging clinical data on SIRPaFc decoy receptors that neutralize the suppressive effects of CD47

To see the full speaker line up, download the full agenda

We are excited to bring the community back together at the virtual Macrophage-directed Therapies Summit to advance the fundamental understanding of macrophage biology, avoiding on-target toxicities and the emerging field of macrophage cellular immunotherapy. 

Join this timely discussion by registering online today.

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Kiadis shares yo-yo on cell therapy trial for COVID-19

Shares in Netherlands biotech Kiadis rose sharply on the news that it would start developing a natural killer (NK) cell therapy for COVID-19, but later lost most of the gains on what looks like profit-taking.

Kiadis has joined forces with five Dutch biomedical groups on the R&D for its coronavirus candidate – called K-NK-ID101 – and has already submitted a grant application to the US government to try to raise money for the project.

For years, Amsterdam-based Kiadis focused its attention on the development of ATIR101, a T lymphocyte-based therapy in development to reduce side effects and improve efficacy in cancer patients who need to have a stem cell transplant, but that was abandoned last year.

The company has turned its attention to off-the-shelf NK cell therapies for use in cancer, headed by K-NK002, which is in phase 2 and going after the same indication as ATIR101.

It also has a therapy called K-NK003 in early-stage clinical development for patients with acute myeloid leukaemia (AML). The new COVID-19 project extends the use of Kiadis’ NK platform into infectious diseases for the first time.

NK cells are often viewed as the “first responders” when it comes to protecting the body against virally-infected or malignant cells.

The company will work with laboratory group Viroclinics-DDL, biotech Harbour BioMed Netherlands, blood donation service Sanquin Bloedvoorziening and two academic institutions – Erasmus Medical Centre and Utrecht University – on the development of K-NK-ID101.

They will study the biology of NK cells in COVID-19 patients and see if K-NK-ID101 can eliminate the SARS-CoV-2 virus and the cells that it infects. In time, the intention is to test the therapy alongside antibodies and vaccines targeting the coronavirus.

Using Kiadis’ platform, donor NK cells are selected for a particular disease target, such as a virally-infected cell, and their numbers are expanded using nanoparticles that cause the cells to rapidly divide. The cells can then be infused into the patient, mounting an attack against the infection.

Kiadis says that in COVID-19, the NK immune response becomes impaired, and in severe cases patients may have no functional NK cells at all.

If K-NK-ID101 can restore that immunity, it could target the infection without exacerbating inflammation, which is an issue in serious COVID-19 cases, it adds.

The company also points out that its NK cell therapies can be manufacturing rapidly at scale and kept frozen, so if they work could be “immediately and globally made available to patients”.

If this project is successful, K-NK cells could potentially serve as a universal countermeasure to fight future viral pandemics, according to Kiadis.

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Novartis plans to seek approval for CAR-T in slow-growing lymphoma after Phase II success

The company said the study of Kymriah in follicular lymphoma met its primary endpoint, though it did not disclose the trial data. It plans to submit approval applications to the FDA and EMA next year.

Preparing for an influx of cell and gene therapy approvals

Cell and gene therapies offer some of the most groundbreaking advancements in patient care the pharma industry has ever seen. However, to fully realise the potential of these innovative therapies, integration across the supply chain is critical – particularly with reimbursement and logistics.

As of the end of 2019, there were 17 cell and gene therapy products approved by the FDA. Now, there is more momentum than ever to bring these innovative medicines to market, and the FDA anticipates that it will approve 10 to 20 cell and gene therapy products a year within the next five years.

These therapies can offer new opportunities to patients with conditions where there are few treatment options and no cures. But the potential these products offer could remain largely unrealised if manufacturers and their partners are not prepared. Cell and gene therapy innovators and other stakeholders across the supply chain need to set themselves up for the greatest chance of success by addressing three key challenges: access barriers; logistics; and the need for stakeholder education.

Addressing access barriers through innovative payment models

While cell and gene therapies offer novel treatment to patients who have limited options, the cost associated with each product – anywhere between $375,000 and $2 million – can create significant access barriers. This challenge is compounded compared to traditional treatments that typically require multiple doses, as many cell and gene therapies are one-time treatments.

This situation increases the risk for payers covering the cell and gene therapy, given that the long-term magnitude and durability of the product is not known at the time of first regulatory approval and patients switch insurance carriers throughout their lifetimes.

“Stakeholders across the industry have recognised the increasing need to consider alternatives to the standard payment system if cell and gene therapies are to become widely available”

Stakeholders across the industry, such as manufacturers and payers, have recognised the increasing need to consider alternatives to the standard payment system if cell and gene therapies are to become widely available. As a result, a variety of payment models have been discussed:

  • Value-based model: The payer pays only a portion of the full price upfront. If the therapy achieves prespecified outcomes, the payer remits the remainder in full. This model spreads the financial risk, therefore, between the payer and manufacturer, and has been the most commonly employed method to date.
  • Pay-over-time model: The payer agrees to a fixed price for the therapy but pays in regular installments, like with an annuity, spreading the cost over time.
  • Subscription-based model: This model offers the payer a flat rate for coverage of various cell and gene therapy products, which provides predictability and helps them offset the potentially high upfront costs of these therapies and realise longer-term cost-savings.

We have already begun to see payers and manufacturers of cell and gene therapies attempt to adopt alternative payment models for their products, and more should continue to do so as additional therapies come through the approval pipeline. With a range of interdependencies that affect the success of cell and gene therapies, manufacturers need to develop their reimbursement strategy early in the commercialisation process. It’s critical for manufacturers to consider various payment models for cell and gene therapies ahead of approvals so that they can maximise patient access for their products.

Ensuring therapies reach their patients

Manufacturers have noted that the delivery of critical shipments is one of the biggest challenges facing the advanced therapy industry, as if you cannot connect cell and gene therapies with patients their efficacy is irrelevant. The inclusion of patients into the cell and gene therapy supply chain, the potentially life-altering impact of the therapies and their high cost leaves no room for failure.

These therapies require timely delivery and maintaining precise temperature control is integral for the patient and the product. It calls for near-perfect execution ranging from mapping the best transportation route and planning for multiple contingencies (such as closed international borders), to how the packaging itself is evaluated, validated and used to maintain product integrity in all conditions.

Successful execution of these processes requires both manufacturers and other supply chain partners to maintain a robust logistics platform. Currently, many manufacturers are developing different logistics plans for each of the stages of a clinical trial, only to find out these processes don’t scale when it is time to commercialise. Developing a plan early that can scale will position a product for success as more therapies are reviewed and approved. Manufacturers need to work with their 3PL and distribution partners to ensure control and oversight throughout the product journey to the patient – failure to do so will put patient outcomes and commercial success at risk.

Promoting stakeholder education

Many stakeholders – spanning payers, providers and patients – do not understand the full clinical, logistical, operational, financial or reimbursement components associated with cell and gene therapies. Manufacturers can leverage the preliminary data they’ve gathered throughout their initial commercialisation journey to support education and awareness efforts with these key stakeholders.

As payers conduct product reviews earlier and earlier in the development lifecycle, their demand for pre-approval information continues to grow. However, recent research shows that a gap still exists between the evidence sought by healthcare decision makers and what is being shared by manufacturers. COVID-19 has also caused delays in providing information in a timely and relevant manner, causing even more challenges for stakeholders.

The use of Pre-approval Information Exchange (PIE) is one way to combat these challenges. PIE allows manufacturers to communicate ahead of approval to partners with accurate, and unbiased information on products or indications, and share information early that may result in a “place saved at the table” for their product. This information equips stakeholders with the education needed to understand a product’s value story and positioning. Partners embedded in the industry – particularly those with a patient-centric focus – can also offer manufacturers the information they need to showcase the value of these products to patients.

The cell and gene therapy space is continuing to evolve. Through analysing payment models, working with partners to navigate logistical challenges and leveraging data, patients will have more opportunities than ever to access the next generation of medicines. Overall, the collaboration between stakeholders across the supply chain will facilitate a world in which we see 10 to 20 cell and gene therapies not only approved each year but out in the market directly impacting patients.

About the authors

Alex Guite is vice president services and alliances at World Courier. As strategy and services lead, Alex is responsible for developing and executing key strategic initiatives.Before joining World Courier in 2013 as head of pricing, Alex spent nearly 3 years with Oliver Wyman as a consultant in the Health and Life Sciences practice.

Ana Stojanovska is vice president, reimbursement & policy insights at Xcenda. She has extensive practical knowledge in working with key stakeholders to motivate local coverage of new products by both public and private payers and providing strategic compendia analyses and ongoing coding support. Prior to Xcenda, Ana worked for a bipartisan, non-profit health policy organization in Washington DC, where she helped lead research, health policy analysis, media outreach, and fundraising.

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BMS, bluebird refile FDA approval application for CAR-T therapy in myeloma

The companies’ resubmission of their application Wednesday seeking approval for idecabtagene vicleucel was in line with the timeline they provided in May, when the FDA sent a refuse-to-file letter in response to their initial submission.

Cell Therapy: A Potential Treatment for COVID-19?

With the success of first vaccine to reach phase I clinical trial and turning out to be safe, well-tolerated, and capable of generating an immune response against the virus in humans, a lot of hope has been created with this vaccine. However, the research is still ongoing to develop novel therapeutic treatments that could aid infected patients in the meantime. One such growing area of interest is the use of cell therapy. Cell therapies represent highly innovative therapeutic approaches that have revolutionized healthcare practices. Several studies from all over the world  has proposed stem-cells based therapy, specifically mesenchymal stem cells, as a suitable remedial approach in the treatment of acute respiratory distress syndrome (ARDS), which is the leading cause of death in COVID-19 patients. Even though there are no approved cell therapy-based approaches for the prevention or treatment of COVID 19, however, many clinical trials have begun, and scientists are trying relentlessly to develop a therapeutic to treat this disease.

Companies Engaged in the Manufacturing of Cell Therapies

Presently, over 100 industry players and 60 non-industry players are involved in the manufacturing of cell therapies; of these, 52% have the required capabilities for manufacturing T-cell therapies.

Cell Therapy: A Potential Treatment for COVID-19?

The Key Hubs of Cell Therapy Manufacturing

Majority of the industrial stakeholders (41%) are based in North America, followed by those based in Europe (31%) and the remaining in Asia Pacific. It is worth mentioning that within Asia Pacific, Japan (8) emerged as a popular hub for cell therapy manufacturers.

Cell Therapy: A Potential Treatment for COVID-19?

Demand for Cell Therapies (in terms of number of patients) is Anticipated to Grow at a CAGR of >21%, During 2019-2030

Given that advanced therapeutic medicinal products (ATMPs) is relatively a niche domain, the overall commercial demand for cell therapies is estimated to be more than 18,500 patients in 2019 and this value is likely to grow to close to 0.4 billion patients by 2030.

Cell Therapy: A Potential Treatment for COVID-19?

To get a detailed information on the key players, recent developments, capacity available, demand and the likely market evolution, visit this link

Cell Therapy: A Potential Treatment for COVID-19?

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