Category Archives: Collaborations

Roche and SQZ expand cell therapy deal to more than $1.3bn

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US biotech SQZ Biotechnologies has expanded a collaboration with Roche to develop cancer therapies based on antigen presenting cells, which may be able to target solid tumours and which are cheaper to make than the recently approved CAR-T drugs.

The deal announced late yesterday expands a collaboration that began in 2015 to a deal worth more than $1.375 billion if all development targets are met.

SQZ’s technology is based around antigen presenting cells (APCs), which occur naturally in the body and present antigen on their surface to stimulate a T-cell response against that particular antigen.

SQZ is able to genetically engineer APCs with tumour antigens, that when injected into the body can produce powerful responses from CD8 T-cells – also known as killer T-cells.

The hope is that these killer T-cells will produce a strong response against any tumour target of interest, across a wide range of cancers.

Roche already has a cancer immunotherapy on the market – its Tecentriq (atezolizumab) is already approved in certain kinds of bladder and lung cancer.

But unlike Novartis and Gilead it does not have cell therapy approved yet and is looking to develop cell therapies that could be used in solid tumours, instead of blood cancer like its rivals’ CAR-T therapies.

Under the collaboration, SQZ may receive up to $125 million in upfront payment and near-term milestones.

SQZ could earn up to $250 million in clinical, regulatory and sales milestones per product that emerges from the collaboration.

The Massachusetts-based biotech may receive development milestone payments of over $1 billion. Within the collaboration, SQZ and Roche could share commercial rights for certain approved products.

Howard Bernstein, chief scientific officer of SQZ, said: “This collaboration allows for a SQZ APC product engine that could potentially generate products with more potent immunologic responses through a simplified, more efficient manufacturing process.”

Chimeric antigen receptor T-cell (CAR-T) drugs are potentially curative in the patients that respond – but are very expensive to make as they involve harvesting a patient’s T-cells, genetically modifying them in a lab, and then re-injecting them to fight cancer.

SOURCE: www.pharmaphorum.com/news

FDA gives go-ahead for CRISPR-based sickle cell disease trial

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Development of a stem cell therapy for sickle cell disease from Vertex and development partner CRISPR therapeutics can go ahead, after the FDA lifted a hold on a review.

The companies had applied to begin an early stage trial of CTX001, a gene therapy derived from a patient’s own stem cells, for beta-thalassemia and sickle cell disease.

Although it’s a long way from the market, the drug could be one of the first to use the revolutionary CRISPR/Cas9 gene editing technology to correct a genetic defect causing a disease.

After an application in April for a phase 1/2 trial in the UNS for adults with sickle cell disease, the FDA had further questions that needed to be resolved.

Without revealing further details, the companies said the trial had been put on hold until they could provide the information the FDA required.

The FDA has now lifted the clinical hold and allowed development to go ahead, although the companies gave no further information about the information required by the regulator.

CRISPR and Vertex have obtained approval for clinical trial applications for several countries outside the US for beta-thalassemia and SCD.

They said they are on track to begin a phase 1/2 study in SCD by the end of 2018 and are enrolling patients transfusion dependent beta-thalassemia in a phase 1/2 trial in Europe.

CTX001 uses the CRISPR gene editing technique to make a patient’s haematopoietic stem cells produce high levels of foetal haemoglobin (HbF) in red blood cells.

HbF is a form of the oxygen carrying molecule haemoglobin naturally present at birth, which is replaced by the adult form of haemoglobin.

The elevation of HbF by CTX001 could alleviate transfusion requirements for beta-thalassemia patients and painful and debilitating sickle crises for sickle cell patients.

CRISPR and Vertex began a strategic research collaboration in 2015 to discover and develop gene editing treatments using the CRISPR/Cas9 technology to correct defects in genes known to cause or contribute to certain diseases.

Vertex has exclusive rights to license up to six new CRISPR/Cas9-based treatments that emerge from the collaboration, and CTX001 represents the first treatment to emerge from the joint research program.

For CTX001, CRISPR and Vertex will equally share all research and development costs and profits worldwide.

Novartis yesterday unveiled data showing its crizanlizumab reduced occurrence of the painful and potentially fatal vaso-occlusive crises that occur when blood cells become stacked in patients with SCD, blocking arteries and cutting the oxygen supply to vital organs.

SOURCE: www.pharmaphorum.com/news/fda

Roche buys Tusk, plus cancer immunotherapy drug, for £62m

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Roche has bought immuno-oncology biotech firm Tusk Therapeutics for an upfront payment of £62 million.

Tusk’s shareholders will receive the upfront cash payment, plus additional contingent payments of up to £521 million, if and when certain milestones are achieved.

Tusk, which is based in Stevenage, has developed a first-in-class antibody, CD25, for the depletion of regulatory T-cells (TRegs), which suppress the body’s immune reaction to cancer cells.

This novel antibody enables the body’s other immune cells to fight tumours while leaving healthy tissue unharmed. Clinical trials are expected to start next year.

Luc Dochez, CEO of Tusk Therapeutics, said: “We are delighted that Roche will further develop this novel antibody and drive the development ahead.

“The remaining portfolio of our immune-oncology targets will be further developed by Black Belt Therapeutics, a newly formed company spun out of Tusk Therapeutics.”

Droia Oncology Ventures, Tusk’s majority shareholder, founded the company in 2014. Droia is a specialist investor, which focuses on fighting cancer.

It invests in promising new cancer therapies and accelerates their progress by actively supporting young drug development companies to achieve clinical proof of concept with their lead programmes. The deal will expand Roche’s oncology pipeline.

Also, Roche today announced a new collaboration with Novo Nordisk, which specialises in the treatment and management of diabetes and obesity.

The plan is to integrate insulin dosage information from Novo Nordisk’s connected pen technology into Roche’s open ecosystem, whereby it will communicate with its digital diabetes management solutions including mySugr, which allows people to monitor their glucose levels.

Marcel Gmuender, global head of Roche Diabetes Care, said: “The integration of insulin pen data in our digital health solutions such as mySugr will make it much easier for people with diabetes and their caregivers to track the effect of insulin on blood glucose levels.

“This enables more efficient and targeted decision support, as they can act on near real-time insights to optimise the personalised diabetes management, thereby reducing the risk of costly secondary complications and contributing to improved therapy outcomes and better quality of life.”

Anders Toft, corporate vice president of commercial innovation at Novo Nordisk, echoed this, adding: “Digital health solutions like mySugr are already helping thousands of patients. By integrating Novo Nordisk connected technology with mySugr, we can further ease the day-to-day burden of disease management and provide data-based insights to improve the dialogue between patients and caregivers.”

SOURCE: www.pharmaphorum.com/news

Six new startups win funding and expert support from Bayer

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Changing the experience of health: that’s the focus of the six startups which the Bayer G4A team has included in the Accelerator program this year.

The young companies came out ahead of more than 1,800 competitors from 100 countries.

They now have 100 days in which to intensively drive the further development of their products and solutions with expertise and investment from Bayer. The company will provide them with offices in Berlin, pharmaceutical executives and industry experts as mentors and EUR 50,000 in funding for each project.

The startups that have applied are developing digital solutions that cover the entire value chain within healthcare. Also this year, a patient jury was asked to rank startups according to the impact that their solutions would have on patient experience.

This year’s winners are:
• Agamon (Israel, GB): A healthcare intelligence platform that can be used to compile and structure health-related data from various sources in order to derive new information. www.agamon.io
• Cyclica (Canada): A cloud platform that aims to use artificial intelligence and biophysics to accelerate drug development. www.cyclicarx.com
• KinAptic (USA): An accelerated learning system for VR stroke rehabilitation using electric stimulation that analyses and detects neural signals to stimulate nerves in stroke patients. www.kinaptic.com
• OME (GB): Personalised health coaching that uses extensive data analyses to compile individualized health programs (nutrition, sleep, physical activity) in order to prevent disease. www.ome.health
• S-There Technologies (Spain): A smart device that analyses health data through urine in the toilet and gives patients insights into their health. https://s-there.com/
• Zencorlabs (Germany): A smartphone software and device that uses artificial intelligence to warn patients of heart failure.

Six years ago, Bayer started cooperating with startups in the healthcare sector through the G4A program headed by Eugene Borukhovich, he said, “It’s incredible to see the tremendous impact that some of our startups have had in the industry. I’m impressed to see the maturity and significance of their innovative solutions. Through the Accelerator program, I’m convinced we will be able to make a significant contribution to truly change the experience of health as we know it. We will continue to put people at the centre of their health and care every single day.”

Dieter Weinand, Member of the Board of Management of Bayer AG and head of the Pharmaceuticals Division commented, “Digital solutions are essential to driving innovation in an evolving healthcare environment. Bayer is seeking to apply them across the pharmaceutical value chain in order to detect diseases at an earlier stage, to develop medicines faster, and to deliver individual treatments with a meaningful outcome for patients. In this endeavour, we benefit immensely from collaborations and the exchange of knowledge and skills with innovative startups.”

SOURCE: www.pharmafield.co.uk/pharma_news

Sartorius and Repligen collaborate on perfusion enabled bioreactors

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Designing the bioreactors to control cell growth, fluid management and cell retention to ultimately simplify the development and cGMP manufacture of biological drugs.

Sartorius Stedim Biotech (SSB) and Repligen Corporation have partnered to integrate Repligen’s XCell ATF cell retention control technology into SSB’s BIOSTAT STR large-scale single-use bioreactors to create novel perfusion-enabled bioreactors.

SSB is an international supplier for the biopharmaceutical industry and Repligen Corporation is a global life sciences company focused on bioprocessing technologies.

Christine Gebski, VP of Product Management at Repligen, said: “We are excited to partner with SSB, a global innovator in bioreactor technology. The integration of our market-leading XCell ATF control technology with SSB’s high-performance bioreactors offers a simplified perfusion-enabled bioreactor solution for end users to develop cell culture processes more quickly and implement perfusion more efficiently.”

As a result of this collaboration, end users will stand to benefit from a single control system for 50–2000 litre bioreactors used in perfusion cell culture applications. This single interface is designed to control cell growth, fluid management and cell retention in continuous and intensified bioprocessing and ultimately, simplify the development and cGMP manufacture of biological drugs.

Through the partnership, SSB and Repligen will further collaborate to equip SSB’s recently launched ambr 250ht perfusion single-use mini bioreactor system with Repligen’s KrosFlo hollow fibre filter technology.

The bioreactor system will be sold by SSB as a complete single-use assembly. This optimal design conserves the hollow fibre filter technology across scales, enabling customers to fast track development and scale up their cell culture perfusion processes.

“Sartorius Stedim Biotech has continuously expanded its integrated upstream portfolio for the past years with a focus on robust and scalable, automated single-use solutions, optimized for high-cell-density applications. The collaboration with Repligen will result in easy-to-implement, high-performance and perfusion-ready bioreactors ranging from process development to commercial manufacturing scale,” commented Stefan Schlack, Head of Marketing at SSB.

SOURCE: www.manufacturingchemist.com/news

Can pharma halt the world’s obesity crisis?

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Major research published in the Lancet this week comes as no surprise, but the findings are still sobering: across the world there are too many people who are not doing enough exercise, putting themselves at risk of diseases such as obesity and type 2 diabetes.

The research published in Lancet Global Health showed that more than a quarter (1.4 billion) adults are at risk from not doing enough physical activity – these diseases are hugely costly to society and to individuals affected.

The levels of insufficient physical activity varied widely across income groups – 16% in low-income countries, compared with 37% in high-income countries.

And in 55 (33%) of 168 countries, more than a third of the population was insufficiently active according to the figures collated in 2016.

In four countries more than half of adults were insufficiently active – Kuwait (67%), American Samoa (53%), Saudi Arabia (53%) and Iraq (52%).

But the regions with the highest increase in insufficient activity over time were high-income Western countries (from 31% in 2001 to 37% in 2016), and Latin America and the Caribbean (33% to 39%).

Countries from these regions driving this trend include Germany, New Zealand, the USA, Argentina, and Brazil.

Authors also identified several socioeconomic forces at work behind the problem – including urbanisation, sedentary occupations, and motorised transport in the richer countries where lack of exercise is most prevalent.

This research will be of interest to the pharma companies that are attempting to tackle diabetes and obesity related diseases, not just with medications but by working with governments to try and influence policy to reduce incidence of the disease.

Leaders in the field such as Novo Nordisk and AstraZeneca are actively campaigning to try and encourage governments to think about how they can encourage people to become more active, and reducing the levels of obesity in society.

With networks of experts in diabetes in countries across the world big pharma companies have realised that there is a huge opportunity to reach out to health systems using corporate social responsibility programmes that aim to tackle the issues outlined in the Lancet research.

For instance Novo has created an initiative entitled “Cities Changing Diabetes” that specifically aims to tackle the problem of “urban diabetes”.

The project involves collecting qualitative and quantitative evidence that could lead to better understanding of the problem and the contributing factors.

It has built up a network of partners across the world, including city leads, city administrations, academia, diabetes associations, health insurances, community centres and business corporations.

So far it has built relations with 16 cities across the world, representing 100 million citizens, including Beirut, Copenhagen, Leicester and Shanghai.

The project is driven by the recognition that the problem with diabetes is only going to get worse unless immediate action is taken.

According to modelling from Novo Nordisk, in order to hold the rise in prevalence at 10%, the world must set itself a target of reducing obesity by 25% by 2045.

Novo organised a Cities Changing Diabetes Summit last year, where it made the call for joint working across sectors and disciplines in order to unite them behind the cause.

Novo has launched an Urban Diabetes Toolbox that gives policy makers tools on how to tackle the problem, including diabetes vulnerability assessment tools, and tips about how to promote healthy living.

AstraZeneca has also been active in this regard, taking part in the multi-year Action in Diabetes initiative and participating in the Global Diabetes Policy Forum in Rome last October.

Now in its third year, the event brought together more than 100 leading global experts in type 2 diabetes care to discuss best practice in policy-making.

Inspired and funded by AstraZeneca, the initiative operates in partnership with the Internatioinal Diabetes Federation, the World Heart Federation, and Primary Care Diabetes Europe, among other organisations.

AstraZeneca’s work aims to demonstrate the interconnectivity between metabolic, cardiovascular, and renal diseases and foster policies that deal with these diseases in an holistic manner.

Eli Lilly is also known for its work in diabetes, and has launched its non-communicable disease partnership with a similar aim.

It has three aims  – piloting new approaches to strengthen diabetes care, advocating to governments for better disease management, and increasing appropriate use of and compliance with medicines to improve outcomes.

The scale of the problem is daunting, but pharma’s focus on raising awareness about the issue, and bringing different stakeholders together towards the common goal of reducing obesity is an example of how industry can help to tackle one of the major social problems of our times.

SOURCE: www.pharmaphorum.com/views-and-analysis

Ionis/Akcea’s ultra-rare disease drug rejected by FDA

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The FDA has opted to refuse approval to Akcea and Ionis’ Waylivra (volanesorsen) for the treatment of the ultra-rare hereditary condition familial chylomicronemia syndrome (FCS), despite the submission of Phase 3 data from the largest-ever study of the disease.

The US regulator alerted the manufacturers via a complete response letter (CRL), originating from its Division of Metabolism and Endocrinology Products, but the reason for the rejection was not given. Submitted data had shown that Waylivra reduced triglycerides by 94% in patients compared to placebo, which raised levels by 18%

FCS is characterised by extremely elevated triglyceride levels in the blood – levels which can’t be adequately metabolised due to a deficiency off lipoprotein lipase; it severely impacts daily life and can cause a range of damaging conditions including unpredictable and potentially fatal acute pancreatitis, chronic complications due to permanent organ damage.

“We are extremely disappointed with the FDA’s decision. FCS is an ultra-rare and debilitating disease. Our disappointment extends to the patient and physician community who currently do not have a treatment available to them,” commented Paula Soteropoulos, Chief Executive Officer of Akcea Therapeutics. “We continue to feel strongly that Waylivra demonstrates a favourable benefit/risk profile in people with FCS as was reflected in the positive outcome from our Advisory Committee hearing in May. We will continue to work with the FDA to confirm the path forward.”

Dr Brett P Monia, Chief Operating Officer of Ionis Pharmaceuticals, added: “We are fully supportive of WAYLIVRA and the many patients, physicians and researchers who are working to provide the first therapeutic option for FCS, a truly life-altering disease that deserves a treatment.”

SOURCE: www.pharmafile.com/news/518434

LEO Pharma to develop and commercialise JW Pharmaceutical’s JW1601

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Denmark-based LEO Pharma has entered into a global licensing agreement to develop and commercialise South Korean JW Pharmaceutical’s JW1601 drug candidate in a deal worth approximately $402m.

Under the terms of the deal, LEO Pharma will get exclusive global rights to JW1601. However, JW Pharmaceutical will retain its exclusivity in South Korea.

JW Pharmaceutical’s JW1601 is a new drug candidate for the treatment of atopic dermatitis. It was developed by one of the company’s affiliates C&C Research Laboratories.

Last year, JW Pharmaceutical acquired the exclusive rights to develop and commercialise the drug candidate globally. An investigational new drug application for a Phase I clinical trial is expected to be submitted over the coming months.

JW1601 is designed to prevent the activation and migration of immune cells that cause atopic dermatitis. The therapeutic selectively targets the histamine H4 receptor and inhibits histamine signalling responsible for itching.

The anti-pruritic and anti-inflammatory effects are expected to deliver better efficacy, while the selectivity towards H4 receptor is expected to show a good safety profile.

LEO Pharma Global R&D executive vice-president Kim Kjoeller said: “At LEO Pharma, we continuously seek to expand our pipeline with new innovative solutions with the ultimate aim of bringing real life-changing medicines to the many patients we serve.

This compound is a perfect fit with our existing biologics currently in phase III (Tralokinumab) and phase I (LP0145) and our topical Delgocitinib currently in phase II.”

As part of the agreement, JW Pharmaceutical will receive an upfront fee of $17m, followed by up to $385m in stepwise development and sales milestones.

In addition to the milestone payments, LEO Pharma will pay royalties on net sales of the drug candidate.

SOURCE: www.pharmaceutical-technology.com/news

Collaborative working is key for the NHS and pharma

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Both the NHS and pharma recognise the need and the opportunity to collaborate in order to improve patient care and outcomes, and this was a key theme for discussions at the recent HSJ Life Sciences Forum, which united thought leaders across the healthcare spectrum. 

One way in which industry can work in mutually beneficial partnership with the NHS is by looking beyond the usual promotional plans and tactics, to how it can share valuable clinical evidence and information online by accessing NHS staff intranets. This can enable pharma to build its reputation and strengthen customer relationships whilst improving NHS staff knowledge and expertise.

There is a particular need to support NHS staff on the health economic benefits of complex speciality medicines, as indicated in McKinsey & Company’s recent Medical Affairs: Key Imperatives for Engaging and Educating Physicians in a Digital World report.

It states: “There is an increasing need for education and high-quality information, given the proliferation of specialty and more complex medicine.” The industry has to play an important role in collaborating with the NHS to keep staff abreast of the latest innovations and evidence to support the drive for outcomes-focused reimbursement models.

However, it is clear that pharma needs to modify its approach and consider different multi-channel methods, since the McKinsey report shows that although physicians’ use of digital content for discussion, research and collaboration continues to grow, “81% of physicians are dissatisfied with their interactions with biopharmaceutical companies, and more than 40% no longer perceive a ‘need’ for medical support from pharma”. Physicians’ dissatisfaction was said to be driven by a perceived lack of personalised, relevant content (28%) and appropriate communication channels (17%).

The highly regulated environment in which pharma operates is referenced in the McKinsey report as being partly to blame for industry’s failure to provide the kind of targeted, personalised digital communications for HCPs that are commonplace for customers in many other industries.

This isn’t helped by the rapid pace of structural change within the NHS, where new care models are emerging in different areas across England, key stakeholders often hold a number of roles and responsibilities, and collaborative decision-making is becoming increasingly important.

Despite these barriers, industry already has a wealth of approved and compliant information that can be shared through NHS staff intranets, as part of a more collaborative partnership approach both on and offline. This can enable NHS staff to gain easy access to vital information to help improve outcomes in key care pathways.

Content could range from clinical evidence papers, to trials results reported in conference highlights and clinical guidelines. Reports and outcomes from joint working initiatives that show, for example, how the re-design of a care pathway, combined with new treatments, is improving patient outcomes, could also provide valuable insight nationally and regionally.

By supporting continuing professional development (CPD), with knowledge-improving modules/quizzes, industry can add value to the cash-strapped NHS and help HCPs, who are finding it increasingly difficult to access traditional CPD courses, owing to funding cuts.

While historically the industry has prioritised promotional budgets and activity to drive sales performance, now is the time to explore equally measurable and focused routes to sharing the huge range of valuable clinical information at its disposal in order to work more closely with the NHS and help to optimise the performance of NHS staff. This, in turn, will build relationships, reputation and value for both the NHS and pharma.

As the resource constrained and cash strapped NHS looks for ways to collaborate with the life sciences industry to further improve and optimise patient outcomes, there is a substantial opportunity for the industry to align its own objectives by capitalising on its data, information and evidence.

SOURCE: www.pharmaphorum.com/views-and-analysis

 

Bristol diabetes spin-out company acquired by Novo Nordisk

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The global healthcare company has acquired all of the shares of the Bristol supramolecular chemistry company, of which total payments under the agreement could ultimately exceed $800 million.

Novo Nordisk has acquired all of the shares of Ziylo, a University of Bristol spin-out company based at the Unit DX science incubator in Bristol, UK.

Ziylo has been pioneering the use of its platform technology – synthetic glucose binding molecules – for therapeutic and diagnostic applications.

The acquisition gives Novo Nordisk full rights to Ziylo’s glucose binding molecule platform to develop glucose responsive insulins.

The development of glucose responsive insulins is a key strategic area for Novo Nordisk in its effort to develop this next generation of insulin, which would lead to a safer and more effective insulin therapy.

A glucose responsive insulin would help eliminate the risk of hypoglycaemia, which is the main risk associated with insulin therapy and one of the main barriers for achieving optimal glucose control. Thus, a glucose responsive insulin could also lead to better metabolic control and overall reduce the burden of diabetes for people living with the disease.

Prior to closing of the acquisition, certain research activities have been spun out of Ziylo to a new company, Carbometrics. Carbometrics has entered into a research collaboration with Novo Nordisk to assist with ongoing optimisation of glucose binding molecules for use in glucose responsive insulins.

Carbometrics has licenced rights to develop non-therapeutic applications of glucose binding molecules, with a focus on developing continuous glucose monitoring applications.

Ziylo’s glucose binding molecules are synthetic molecules that were designed by Professor Anthony Davis at the University of Bristol. These stable, synthetic molecules exhibit an unprecedented selectivity to glucose in complex environments such as blood.

The combination of this technology with engineered insulin pioneered by Novo Nordisk aspires to realise the world’s first glucose responsive insulin and transform the treatment of diabetes.

“We believe the glucose binding molecules discovered by the Ziylo team together with Novo Nordisk world-class insulin capabilities have the potential to lead to the development of glucose responsive insulins, which we hope can remove the risk of hypoglycaemia and ensure optimal glucose control for people with diabetes,” said Marcus Schindler, senior VP, Global Drug Discovery, Novo Nordisk.

“Novo Nordisk is the ideal company to maximise the potential of the Ziylo glucose binding molecules in glucose responsive insulins and diabetes applications, and it brings hope of a truly groundbreaking treatment to diabetes patients,” said Dr Harry Destecroix, CEO and cofounder of Ziylo.

Novo Nordisk has acquired all shares in Ziylo for an upfront payment and earn-outs with contingent milestone payments. Total payments under the agreement could ultimately exceed $800 million upon the achievement of certain development, regulatory and sales milestones by Novo Nordisk.

SOURCE: www.manufacturingchemist.com/news