20 March 2018

Pistoia Alliance calls life sciences firms to support ‘lab of the future’ 

Global non-profit organisation The Pistoia Alliance is urging the life sciences industry to join its efforts in creating the ‘lab of the future’ (LoTF) through greater collaboration.

At the alliance’s annual EU member conference held in London, companies including Merck, GlaxoSmithKline and SAP discussed how artificial intelligence (AI) and real-world data will contribute to the development of the lab.

The conference, which involved 165 participants from ten pharma companies, biotechs and tech firms, was intended to pinpoint potential partnerships aimed at improving research and patient outcomes.

The Pistoia Alliance president Dr Steve Arlington said: “If pharmaceutical companies want to support R&D outcomes that truly benefit patients, then building the ‘lab of the future’ is paramount.

“They must now seek ways to amplify their budgets through greater collaboration, cooperation, and data sharing, and by exploring future technologies that will change the lives of patients.”

The conference included a briefing on existing pharmaceutical R&D models and commercial challenges faced by the industry.

The conference also showcased The Pistoia Alliance’s President’s Series Hackathon, held on 12-13 March.

The hackathon entailed a series of challenges, focused on AI insight, the ‘future corner’, and health and safety for the LoTF.

Participants had to build prototypes that use AI to comprehend instrument logs in order to analyse the effect of new tools on the lab and determine new uses for datasets and related lab data.

19 March 2018

Thermo Fisher to invest in new pharma services facility in Germany

Thermo Fisher Scientific has announced plans to invest $35m in a new pharma services supply chain facility in Germany.

The plans are aimed at expanding the company’s presence in the European Union (EU) in response to a growing demand for global clinical supply services.

Thermo Fisher will use the latest technology and infrastructure to increase its European capacity for cold and ambient clinical trial materials. The new facility is slated to create more than 200 new jobs.

The facility will be scalable for mixed-use space and will offer a strategic logistical location for shipping either by road or air.

Construction of the 8,000m² facility is expected to start in the fourth quarter of this year, with building predicted to take 12-18 months.

The investment will also include the option for additional expansion with the potential for doubling in size.

Fisher Clinical Services Europe vice president and general manager Astrid Frank said: “This is an exciting time for our business, and it reinforces our commitment to best serve our clients globally.

“By creating a best-in-class supply chain facility in the EU, we open new opportunities to support client and patient need.”

The new facility will comply with GDP guidelines, Clinical Trial Regulations Annex 6 and the new Falsified Medicines Directive 2019.

It will join Thermo Fisher’s 20 purpose-built cGMP facilities worldwide.

19 March 2018

Lundbeck to buy Netherlands’ Prexton Therapeutics for €905m

Danish pharmaceutical company Lundbeck has agreed to purchase Netherlands-based Prexton Therapeutics for up to €905m.

Under the deal, Lundbeck will make an upfront payment of €100m, followed by €805m on achieving development and sales milestones.

The acquisition will add Prexton’s drug candidate, foliglurax, to Lundbeck’s pipeline of treatments for patients with Parkinson’s disease.

Foliglurax is a selective, small-molecule, positive allosteric group III metabotropic glutamate receptor 4 (mGluR4 PAM) stimulator formulated to trigger a compensatory neuronal system in the brain that is mostly unaffected in Parkinson’s.

It is being developed to address motor symptoms such as muscle rigidity, resting tremor and uncontrolled movements (dyskinesia).

Lundbeck interim CEO and CFO Anders Götzsche said: “By acquiring Prexton, Lundbeck will obtain global rights to foliglurax, an exciting first-in-class compound, and gain full control of the asset.

“Foliglurax addresses high unmet needs with its potential indication in Parkinson’s fitting perfectly within Lundbeck’s core areas and this treatment option also appears to be highly interesting for patients, physicians and payors.”

The compound will be investigated in a double-blinded, randomised, placebo-controlled, parallel-arm Phase II clinical trial measuring changes in OFF-time (time where the treatment does not work) in Parkinson’s and dyskinesia, including levodopa-induced dyskinesia.

The trial is set to recruit 165 subjects across the UK, Germany, France, Austria, Spain and Italy.

Foliglurax’s safety, tolerability and efficacy in minimising motor complications of levodopa therapy will be evaluated during the trial in patients suffering from end-of-dose wearing off and levodopa-induced dyskinesia.

Initial results from the Phase II programme are expected in the first half of 2019.

16 March 2018

China FDA replaced by State Drug Administration in proposed reform

The China Food and Drug Administration (CFDA) will merge with other administrative bodies to form a national market supervision administration.

In an overhaul of ministries proposed by the Chinese Government, known as the State Council, the CFDA will be replaced by the State Drug Administration. The CFDA is currently a standalone agency, but under the new plans it will become part of the newly-formed State Administration of Market Supervision.

The proposal was announced by China’s State Council during an annual conference in March, and is one of several restructurings the Chinese Government is rolling out. It will mean that the China State Council will be made up of 26 ministries and commissions.

The new entity will not be responsible for food administration, allowing the regulator to focus more on medical technologies. The proposed changes are designed to raise China’s drug regulations to international standards.

Under the reforms, drug sales will not be monitored by the national regulator but by local market-supervision agencies.

There will also be changes to health insurance and drug pricing, which will be administrated by the newly-formed State Administration for Health Security division.

The plan has been submitted to the 13th National People’s Congress for final approval, with national legislators due to vote on the draft plan later.

The regulator has been restructured several times since it was founded in 1998, most recently in March 2013 when the State Food and Drug Administration was rebranded and restructured as the China Food and Drug Administration.

The CFDA introduced a series of reforms in 2017 aimed at creating increased regulatory transparency and encouraging greater collaboration with the rest of the world. In August last year, the CFDA joined the International Conference on Harmonisation of Technical Requirements for Registration of Pharmaceuticals for Human Use, a project that brings together different regulatory authorities to achieve greater regulatory co-operation.

The agency went on to announce plans to accept and use data generated from clinical trials run overseas in October.

16 March 2018

NIH-funded trials for H7N9 influenza vaccine begin enrolment in US

Two Phase II clinical trials have started enrolling participants across the US to test an experimental inactivated influenza vaccine, 2017 H7N9 IIV.

Developed by Sanofi Pasteur, the vaccine candidate is intended to prevent infection from the bird influenza virus H7N9, which was first reported in humans in China in 2013.

The trials will evaluate various dosages of the vaccine, vaccination schedules and the candidate’s effect on immune responses when given in combination with GlaxoSmithKline’s AS03 vaccine.

The trials are funded by the National Institute of Allergy and Infectious Diseases (NIAID) division of the National Institutes of Health. The NIAID-funded network of vaccine and treatment evaluation units (VTEUs) will perform both of the trials.

NIAID director Anthony Fauci said: “These new clinical trials will build upon initial studies of earlier versions of an H7N9 vaccine candidate to provide a more detailed picture of its safety and ability to generate a protective immune response to current H7N9 strains.”

The new version of the vaccine candidate uses an inactivated form of the H7N9 influenza virus collected in 2017 to increase the likelihood of the vaccine providing immunity against a newly-evolved strain of H7N9.

The first of the trials will be conducted at VTEU sites in Georgia, Iowa, Maryland, North Carolina and Washington, where 420 participants aged 19-64 and 300 participants over 65 will be recruited.

The volunteers will receive two vaccinations of 2017 H7N9 IIV either with or without the AS03 adjuvant.

Set to take place in Alabama, Maryland, Ohio and Tennessee, the second trial will recruit 150 healthy individuals aged 19-64. The trial will test a combination of the vaccine candidate, AS03 adjuvant, and a quadrivalent seasonal influenza vaccine.

In this trial, investigators intend to evaluate whether the H7N9 vaccine candidate affects the immune response to the seasonal influenza vaccine, and vice versa.

Subjects in both the trials will be monitored for seven days after each vaccination and followed-up for around one year.

15 March 2018

Canadian and US scientists partner to fight brain diseases

Neuroscientists from the National Research Council of Canada (NRC) and US-based biotechnology company Biogen have collaborated to develop new therapies for brain disorders such as dementia.

The partners will use the NRC’s molecular map of blood vessels that form the blood-brain barrier (BBB) to create new treatment strategies.

While the BBB blocks bacteria and viruses, it also inhibits more than 95% of drugs from entering the brain, thereby hindering the treatment of conditions such as Alzheimer’s, brain cancer, Parkinson’s and amyotrophic lateral sclerosis.

Over a period of three years, a team of more than 30 scientists from the NRC and Biogen will work to identify new pathways through which medications can cross the barrier.

NRC Life Sciences vice president Dr Roman Szumski said: “We are working with Biogen on advancing knowledge of the brain and its internal workings, so that new treatments that ease the burden of brain diseases for patients and their families can be developed.”

The scientists plan to explore a mechanism that allows essential nutrients to pass through the interior of BBB cells as a potential route for drug delivery.

They will use the NRC’s molecular map to study the surface of the barrier cells and use algorithms to identify the best targets for drug transport.

The scientists will engineer antibodies that can bind to the isolated targets and ‘sneak’ their integrated medicine into the brain.

The ability of the new medicines to cross the BBB and treat the target disease will then be assessed, and the most promising candidates will be advanced to clinical trial.

Biogen Canada managing director Marina Vasiliou said: “With forty years of expertise in neuroscience, we know the tremendous need of advances in areas like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis. We welcome the opportunity to be part of this important initiative.”

14 March 2018

Boehringer and Vanderbilt University project targets cancer

Boehringer Ingelheim and Vanderbilt University have entered their third collaboration project to discover and develop treatments for difficult-to-treat cancers.

The partners intend to develop chemical therapeutics designed to block the pro-survival myeloid cell leukaemia 1 (MCL1) protein, which is highly prevalent in difficult-to-treat cancers and prevents their programmed cell death.

Currently, there are no therapies targeting this protein. Researchers will work towards developing a molecule that binds to MCL1, triggering the on-target, mechanism-based cell death.

Boehringer Ingelheim Austria vice president and research site head Darryl McConnell said: “Boehringer Ingelheim and Vanderbilt University have the expertise and are jointly focused on discovering breakthrough medicines against the cancer-causing proteins KRAS, SOS and now, MCL1.

“Together, we are committed to driving scientific research and development forward to help patients win the fight against cancer.”

Set to be carried out in the Stephen Fesik laboratory at Vanderbilt University School of Medicine, the collaboration will involve researchers from Boehringer and Vanderbilt’s team of structural biologists, medicinal chemists, and cell biologists.

Vanderbilt University School of Medicine, Basic Sciences dean Lawrence Marnett said: “MCL1 is one of the top ten overexpressed genes in human cancer where it plays a role as a survival factor.

“It is a great target for therapy but candidate drugs need to disrupt high-affinity protein-protein interactions, which is very challenging.

“The Fesik laboratory has made impressive strides in developing such compounds and it is exciting to see them advanced toward clinical development through the partnership with Boehringer Ingelheim.”

13 March 2018

Researchers discover ‘addiction gene’ to beat triple-negative cancer

Researchers have discovered a promising approach for treating triple-negative breast cancer by blocking an ‘addiction gene’, KIFC1.

In a study funded by Breast Cancer Now and conducted by researchers from King’s College London and The Institute of Cancer Research, it was found that triple-negative tumours depend on a gene called KIFC1 to survive. They found that blocking this gene could slow tumour growth but did not affect healthy cells.

Researchers performed a genome analysis of 182 patient breast cancer samples to identify the genes either most overactive or present in the greatest numbers that triple negative breast cancers are particularly dependent on. They then narrowed these down to focus on the genes which were known to be associated with features likely to drive tumour growth and progression.

KIFC1 was one of 37 new genes that the researchers found triple-negative breast cancers were ‘addicted’ to. Researchers then performed experiments to test how lacking these genes affected tumour cells’ and healthy cells’ survival.

Published in Nature Communications, the research suggested that KIFC1, also known as HSET, could be a drug target in patients with triple-negative breast cancer.

Targeted therapies are designed to stop cancer from growing and spreading. They work by targeting specific genes or proteins related to cancer growth. Whereas chemotherapy can also kill normal cells when eliminating cancer cells, normal cells can survive targeted therapy.

In triple-negative breast cancer, patients lack the three molecules routinely used to guide treatment for breast cancers: the oestrogen receptor, progesterone receptor, and human epidermal growth factor receptor 2. This means that the cancer can be highly aggressive and cannot be treated with targeted drugs commonly used to interfere with these receptors. Around 15% of all breast cancers are ‘triple negative’ with 7,500 women in the UK diagnosed each year.

Breast Cancer Now CEO Baroness Delyth Morgan said: “Patients diagnosed with triple negative breast cancer unfortunately remain limited to chemotherapy and radiotherapy and surgery because there are currently no targeted treatments available. This can be extremely gruelling, so we desperately need to find them new options. These incredibly exciting findings could give us 37 new avenues of hope for thousands of women.”

“If we could develop drugs to block these ‘addiction genes’ to kill triple negative cancer cells, while leaving healthy cells unscathed, this could be the chink in the armour we’ve long been searching for.”

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