Pharmaceutical Industry 15 – 21 June: Ft Anocca and NICE
- Anocca has treated the first patients with its precision TCR T cell therapy, ANOC 001.
- NICE has recommended the D VRd combination for certain adults with untreated multiple myeloma.
Anocca Reaches Clinical Milestone with ANOC 001
Swedish biotechnology company Anocca has dosed the first patients in its phase 1 VIDAR 1 clinical programme, marking the initial clinical use of its precision TCR T cell therapy, ANOC 001.
The company confirmed that the treatment has now been administered at multiple clinical sites across Europe. The milestone represents the first time that a product developed through Anocca’s pancreatic ductal adenocarcinoma programme has progressed into clinical evaluation.
ANOC 001 is designed to treat patients whose tumours contain the KRAS G12V mutation. KRAS mutations are found in the vast majority of pancreatic cancer cases and have historically proved difficult to target effectively using conventional pharmaceutical approaches.
Pancreatic ductal adenocarcinoma is an especially aggressive form of cancer. It is frequently diagnosed at an advanced stage and has a five year survival rate of below 10 per cent. These poor outcomes have created an urgent need for treatments capable of identifying and attacking cancer cells with greater precision.
By targeting a specific mutation rather than treating pancreatic cancer as a single, uniform disease, ANOC 001 reflects the pharmaceutical industry’s wider movement towards highly personalised cancer therapies.
A Precision TCR T Cell Therapy Built in Sweden
ANOC 001 was discovered, developed and manufactured at Anocca’s facilities in Sweden. According to the company, it is the first non viral gene edited T cell therapy to enter clinical evaluation in Europe.
TCR T cell therapies are created by equipping a patient’s T cells with carefully selected T cell receptors. These receptors are intended to recognise specific targets associated with cancer cells, enabling the immune system to identify and attack the disease more effectively.
Anocca developed ANOC 001 using its proprietary analytical platform, which maps potential therapeutic targets and supports the identification, characterisation and engineering of suitable T cell receptors.
The use of non viral gene editing is another important feature of the programme. Many existing cell and gene therapies rely on viral vectors to introduce new genetic material into cells. Anocca’s approach is intended to offer a precise and potentially more scalable alternative for producing engineered T cell therapies.
The company believes this technology could eventually support the delivery of advanced treatments to broader patient populations, particularly when several products are developed against different mutations and patient immune system profiles.
VIDAR 1 Designed to Evaluate Multiple Products
VIDAR 1 has been structured as more than a conventional single product clinical study. The programme is designed to evaluate several TCR T cell candidates targeting distinct KRAS mutations and different human leukocyte antigen combinations.
Human leukocyte antigens, commonly referred to as HLA types, play an important role in how the immune system presents and recognises cellular targets. Matching a therapy to both a tumour mutation and an appropriate HLA type is therefore central to the precision of Anocca’s treatment strategy.
ANOC 001 is the first candidate to enter the programme. Anocca intends to introduce additional products targeting other forms of mutated KRAS as the clinical study progresses.
This structure could allow the company to assess a family of related therapies within a single coordinated clinical programme, rather than treating every candidate as an entirely separate development project.
Recruitment and manufacturing for the phase 1 stage of VIDAR 1 remain ongoing. The multi centre trial is operating across eight university hospitals in Sweden, Denmark, Germany and the Netherlands.
Anocca’s Chief Scientific Officer said the programme had been designed to investigate multiple products directed at different KRAS mutations and HLA combinations. The company also thanked its clinical collaborators, participating patients and their families for supporting the study.
Anocca Demonstrates End to End Development Capability
Anocca’s co-founder and Chief Executive Officer described the first patient dosing as an important milestone for the company.
The development demonstrates Anocca’s ability to identify a potential cancer target, engineer an appropriate T cell receptor, manufacture the resulting therapy and deliver it into a clinical environment.
That end to end capability is particularly significant within the cell therapy sector, where the transition from laboratory research to reproducible clinical manufacturing can be extremely challenging.
Advanced therapy medicinal products require complex production systems, strict quality controls and carefully managed supply chains. Unlike traditional tablets or injectable medicines, engineered cell therapies often involve living material, individual patient samples and time sensitive manufacturing processes.
Anocca acknowledged the contribution of its team, investors, partners and clinical collaborators in reaching the first patient dosing stage.
While the phase 1 study remains at an early point, the beginning of clinical treatment provides the first opportunity to evaluate ANOC 001 in patients and gather evidence on its safety, tolerability and potential activity.
NICE Supports New First Line Multiple Myeloma Combination
In a separate development within cancer treatment, the National Institute for Health and Care Excellence has issued final draft guidance recommending a four medicine combination for adults with untreated multiple myeloma who are unsuitable for an autologous stem cell transplant.
The recommended combination brings together daratumumab, bortezomib, lenalidomide and dexamethasone. It is commonly referred to as D VRd.
The decision means eligible patients in England and Wales are expected to gain access to the UK’s only licensed subcutaneous CD38 directed antibody quadruplet treatment for first line use once the guidance is finalised.
Daratumumab, developed by Johnson & Johnson, is an antibody treatment that targets the CD38 protein found on the surface of multiple myeloma cells. It is used alongside three established medicines to create a broader first line treatment approach.
Addressing an Incurable and Relapsing Cancer
Multiple myeloma is a form of blood cancer that affects plasma cells within the bone marrow. Around 33,000 people in the UK are currently living with the disease.
Although treatment has improved considerably, multiple myeloma remains incurable. Patients frequently experience periods of remission followed by relapse, and the disease can become increasingly difficult to control with each recurrence.
This makes the choice of first line treatment especially important. An effective initial therapy may help patients achieve a deeper response and potentially delay the point at which additional treatment becomes necessary.
Approximately two thirds of people newly diagnosed with multiple myeloma are unsuitable for an autologous stem cell transplant. This may be due to age, general health, frailty or other clinical considerations.
For these patients, access to a wider range of effective treatments at the beginning of their care can be particularly valuable.
Patient Campaigners Welcome NICE Reconsideration
Myeloma UK welcomed the recommendation and described it as a significant victory for patients following NICE’s earlier decision not to approve the treatment combination.
The charity’s Access Manager said patients had felt let down by the initial outcome and that Myeloma UK had worked hard to encourage NICE to reconsider its position.
The organisation believes access to D VRd could provide newly diagnosed patients with an important additional option from the beginning of their treatment journey.
Myeloma UK also highlighted the potential effect on quality of life. More effective first line treatment may reduce the burden of disease progression and could mean that some patients and their families spend less time attending hospitals for further treatment.
The charity stressed that until a cure for multiple myeloma is found, patients should be able to access suitable therapies as early as possible following diagnosis.
CEPHEUS Trial Supports the Recommendation
NICE’s recommendation is supported by evidence from the phase 3 CEPHEUS clinical trial.
The study compared D VRd with a three medicine regimen consisting of daratumumab, lenalidomide and dexamethasone.
At a median follow up period of 58.7 months, the overall minimal residual disease negativity rate was 60.9 per cent among patients receiving D VRd. This compared with 39.4 per cent among those receiving the three medicine combination.
Minimal residual disease negativity means that highly sensitive testing is unable to detect remaining cancer cells at the specified level of measurement. It can provide an indication of how deeply a patient has responded to treatment.
The higher rate reported in the D VRd group was therefore an important consideration in the evaluation of the quadruplet regimen.
A Haematology Consultant at The Royal Wolverhampton NHS Trust, who participated as an investigator in the CEPHEUS trial in Wolverhampton, described the inclusion of bortezomib as an example of how a relatively small change to an established treatment combination can produce a meaningful effect.
The consultant also emphasised the importance of expanding first line options for patients who cannot undergo a transplant, particularly because this group represents the majority of the multiple myeloma population.
Johnson & Johnson Welcomes NHS Access
Johnson & Johnson welcomed NICE’s decision and thanked the patients, clinicians, campaigners and other participants involved in the appraisal process.
The company’s UK Director of Strategic Access, Pricing and Operations said Johnson & Johnson’s long history of working in multiple myeloma had demonstrated the importance of providing patients with a strong range of first line options.
The company said it hoped the positive outcome would be welcomed by patients and their loved ones.
The recommendation also reflects the importance of collaboration between pharmaceutical manufacturers, clinical researchers, health technology assessment bodies and patient organisations.
Clinical evidence alone is a central part of the approval process, but the introduction of a medicine into routine NHS care also depends on assessments of clinical value, affordability and the practical needs of the healthcare system.
What These Developments Mean for Pharmaceutical Manufacturing and Production
The Anocca and Johnson & Johnson developments demonstrate two different but increasingly connected directions for pharmaceutical manufacturing.
ANOC 001 represents the growth of individualised and highly complex biological production. Manufacturing precision TCR T cell therapies requires specialist facilities, controlled gene editing processes, sophisticated analytical testing and close coordination between hospitals and production teams.
As more therapies of this kind enter clinical trials, manufacturers will need scalable systems capable of maintaining product consistency while handling complex or patient specific materials.
The D VRd recommendation highlights the manufacturing and supply implications of combining several established treatments into a first line regimen.
Wider NHS access could increase demand for daratumumab, bortezomib, lenalidomide and dexamethasone, requiring manufacturers and healthcare providers to maintain reliable production, distribution and treatment capacity.
Together, the developments show that future pharmaceutical production will not be defined by a single manufacturing model. The industry will need to support both highly specialised cell therapies produced through advanced biotechnology and larger scale medicine combinations intended for broad use within national health systems.
Cancer Care Moves Towards Greater Precision and Earlier Intervention
Although ANOC 001 and D VRd address different cancers and sit at different stages of their development, both reflect a common objective: delivering more effective treatment at the most appropriate point in a patient’s care.
Anocca is attempting to identify and attack cancer through a highly specific genetic mutation. Johnson & Johnson’s multiple myeloma combination seeks to strengthen first line treatment by adding an established medicine to an existing regimen.
One development is beginning an early phase clinical study, while the other is moving closer to routine NHS use. Both, however, demonstrate how laboratory science, clinical research, manufacturing capability and patient access must work together if medical innovation is to produce meaningful change.
Conclusion
The first patient dosing of ANOC 001 marks a major step for Anocca and for the European development of non viral gene edited T cell therapies. Through the VIDAR 1 programme, the company is building a platform that could eventually support several precision treatments directed at different KRAS mutations and patient HLA profiles.
At the same time, NICE’s recommendation of D VRd offers renewed hope to adults with newly diagnosed multiple myeloma who cannot receive a stem cell transplant. Supported by findings from the CEPHEUS trial, the decision could provide eligible patients with a more effective first line treatment option through the NHS.
From personalised T cell engineering to the carefully evaluated expansion of an established medicine combination, these developments illustrate the breadth of innovation now taking place across cancer care.
They also underline the responsibility facing pharmaceutical manufacturers to translate scientific progress into treatments that can be produced reliably, delivered safely and made accessible to the patients who need them.
News Credits:
Anocca doses first patients with precision TCR‑T therapy
NICE backs daratumumab quadruplet for newly diagnosed transplant‑ineligible myeloma
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