Category Archives: Neurology

Amgen CEO says Aimovig is “one of most successful launches”

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As biosimilars start to eat into sales of its blockbuster biologic drugs, Amgen needs new products to deliver growth – and it says migraine therapy Aimovig is off to a great start.

Chief executive Bob Bradway told investors on the company’s third-quarter results call that Aimovig (erenumab) is “shaping up to be one of the industry’s most successful recent launches” with sales reaching $22m in the quarter, ahead of analyst expectations.

“People suffering from migraine and their physicians have been waiting for years for an effective new therapy and they’ve reacted very well to Aimovig,” he said. The company estimates that more than 100,000 patients have taken the Novartis-partnered CGRP inhibitor since its launch in May as a once-monthly, self-injected preventive treatment for migraines.

The strong take-up has come despite approvals for rival CGRP inhibitors from Teva and Lilly, and Amgen says its first-mover advantage is backed up by a patient-friendly delivery device, as well as a willingness by healthcare payers to cover the product thanks to a price that encourages access.

The company is also making much of the fact that Aimovig is the only drug that targets the CGRP receptor rather than the CGRP protein itself, although for now there’s no strong evidence to suggest that confers a clinical advantage.

There’s no doubt that the rapid roll-out comes from pent-up demand for the drug among people with migraine who still suffer headaches despite other therapies. However Amgen’s commercial head Murdo Gordon said he believes “we’re scratching the surface of helping chronic migraine sufferers [and] we think that we’ll see continued demand growth.”

Overall, Amgen posted a 2% rise in revenues to $5.78bn in the quarter, despite pressure on older drugs like while blood cell booster Neulasta (pegfilgrastim) which showed the first signs of weakness from biosimilar competition after Mylan/Biocon launched their Fulphila rival in July. TNF inhibitor Enbrel (etanercept) also fell 5% in the face of competition from newer drugs in indications such as psoriasis.

There was also disappointing news once again for cholesterol drug Repatha (evolocumab) which rose 35% to $120m but missed expectations. Last week, Amgen reduced its price by 60% to $5,850 in a bid to kickstart sales growth and overcome payer resistance to the drug.

“This should substantially lower their out-of-pocket costs and lower the abandonment rate which is as high as 75%,” said Gordon, who said the cut was necessary to compete and would build much-needed volumes at the expense of near-term sales.

The Repatha discount comes as the US administration is ramping up the pressure on the biopharma industry on drug pricing, most recently proposing a reference pricing model to bring process closer into line with other affluent nations.

Commenting on the overall environment and how it looks likely to develop in 2019, Bradway said that the US has benefitted enormously from a market which allows patients to access new therapies “more quickly and more comprehensively than in other countries.”

“We think what’s required is more innovation, not less, and the system that enables people to get access to that innovation swiftly,” he suggested.

SOURCE: www.pmlive.com/pharma_news

Urine exRNA may be source of biomarkers for muscular dystrophy

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Massachusetts General Hospital (MGH) researchers have found that extracellular RNA (exRNA) in urine may be a source of biomarkers for the two most common forms of muscular dystrophy, noninvasively providing information about whether therapeutic drugs are having the desired effects on a molecular level.

The report published in online journal Nature Communications, is the first to show that urine exRNA can be used to monitor systemic diseases that do not directly affect the urinary tract.

“Our findings could facilitate drug development by offering a convenient, painless and relatively low-cost assay that may reduce and perhaps eventually eliminate the need for multiple invasive muscle biopsies to track disease activity and therapeutic response,” says Thurman Wheeler, MD, MGH Department of Neurology, senior author of the report. “Urine exRNA monitoring could determine whether a drug is reaching its target long before clinical effects on muscle function could be detected and may enable early identification of whether dosage adjustments may be required, something that would be impossible with invasive muscle biopsies.”

There are several types of muscular dystrophy, all of which lead to progressive muscle weakness and loss of muscle mass. Duchenne muscular dystrophy (DMD) is the most common form, with symptoms that usually begin in children under the age of 5. Myotonic muscular dystrophy (DM) is the most common adult-onset form and has two subtypes – DM1 and DM2. Each form is caused by a different gene mutation. In DMD, the mutation affects the gene for dystrophin, a protein essential to the strength of muscle fibers. DM-associated mutations – in the DMPK gene for DM1 and in the CNBP gene for DM2 – involve excessive repeats of nucleotides, leading to abnormal processing of RNA molecules.

The mutation associated with DM1 affects RNA splicing, the process that removes non-coding segments from an RNA molecule. A single gene can normally give rise to several different proteins, with the differences being determined by alternative RNA splicing patterns. The DM1 mutation interferes with appropriate splicing of RNAs encoding several other proteins, and analysis of RNA splice variants in muscle biopsies has been used to determine disease severity in patients. In animal models, splice variants in muscle tissue have been used to indicate whether potential therapies are reaching their molecular target. A less invasive way of assessing disease severity and therapeutic response could expand the number of patients who could receive therapeutic drugs or participate in clinical trials. For example, a recent clinical trial for a DM1 drug was restricted to adult patients partially because of the need for repeat muscle biopsies.

Carried through bodily fluids like blood and urine in membrane bubbles called vesicles, exRNA encompasses messenger, non-coding, and microRNA molecules and can reflect mutations, deletions and other molecular variants. A few muscular-dystrophy-associated RNA or protein biomarkers have been identified in the blood of patients. Even though it seemed unlikely that exRNA from the skeletal and cardiac muscle tissues affected by DM1 could pass through the kidney’s filtration system into the urine, urine is such an easily accessible fluid that Wheeler’s team analyzed vesicles from both blood and urine for exRNAs that could reflect results of the DM1 mutation.

Their experiments comparing urine exRNAs from DM1 patients, patients with two other forms of muscular dystrophy and unaffected control volunteers identified 10 transcripts that are alternatively spliced in a pattern unique to DM1 patients, most of which had been previously found in patient muscle biopsies. A composite biomarker incorporating these 10 transcripts was 100 percent accurate in distinguishing DM1 patients from unaffected controls in a different group of participants. Samples taken from untreated DM1 patients over several months indicated consistency of splicing patterns within an individual, suggesting that repeat sampling could accurately reflect disease state and treatment response.

Along with developing a more precise assay for rapid measurement of alternative RNA splicing in urine and other bodily fluids, the team showed that splicing patterns in total RNA from the cells in the urine were different from and less useful as biomarkers than those from exRNAs and found that exRNAs in blood could not distinguish between DM1 patients and controls. They also found that the splicing patterns of some urine exRNA transcripts reflected the severity of DM1 symptoms, and that a small group of asymptomatic patients with the DM1 mutation had urine exRNA splicing patterns midway between those of symptomatic patients and unaffected controls.

A group of drugs being evaluated for the treatment of DMD manipulate splicing of the dystrophin gene in order to remove a specific exon – a protein-coding segment of RNA – producing a shortened but partially functional version of the dystrophin protein. The MGH team showed that urine exRNAs from six untreated DMD patients accurately reflected the specific gene mutation in each patient. In two DMD patients being treated with eteplirsen – an FDA-approved DMD drug that induces skipping of the target exon – analysis of urine exRNA was able to confirm the drug was reaching its molecular target, the first such confirmation not provided by muscle biopsy.

“Our demonstration of disease-specific splice variants in urine exRNA suggests the value of biofluids as a means of identifying novel splice variants that may help correlate gene variants with symptoms for several diseases for which noninvasive biomarkers are unavailable,” says Wheeler, an assistant professor of Neurology at Harvard Medical School. “These findings support studies of exRNA from urine, blood or cerebrospinal fluid as biomarker replacements for tissue biopsies in other conditions with altered RNA splicing – including other types of muscular dystrophy, spinal muscular atrophy and amyotrophic lateral sclerosis.”

SOURCE: www.news-medical.net/news

JM to offer a new process to synthesise CBD

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Johnson Matthey to offer a new process to synthesise ultra-pure cannabidiol, expanding on existing cannabinoid offerings.

Johnson Matthey, a global leader in science that enables a cleaner and healthier world has announced an expansion to its API and controlled substances portfolio by establishing a new synthetic method for ultra-pure cannabidiol, a medicinal component of the cannabis plant.

This synthesis will help Johnson Matthey (JM) support companies in developing novel treatments and medicines to help patients across a range of disease areas.

Cannabidiol (CBD) is one of the many chemical compounds in the cannabis plant and is known to possess medicinal and therapeutic properties. Unlike tetrahydrocannabinol (THC), another molecule in the cannabis plant, CBD does not cause intoxication or euphoria, two unwanted side-effects for medicines.

In light of CBD’s medicinal applications and the absence of psychoactive side-effects, demand for GMP-grade CBD from pharmaceutical companies has increased extensively in recent years.

It is now being investigated as a potential therapeutic treatment for various illnesses and diseases, including multiple cancers, seizures, dermatological conditions and anxiety.

JM has established more than 15 years’ experience in developing and commercialising a portfolio of ultra-pure synthetic cannabinoids and other controlled substances. By adding cannabidiol into this portfolio and filing a US drug master file (DMF) with a validated synthetic process, JM is excited to support companies looking to explore CBD’s medicinal applications further.

With manufacturing sites based in the US, JM is able to apply its knowledge of synthetic chemistry and purification techniques to CBD synthesis. In particular, JM’s solid form chemistry expertise has enabled the development of a free flowing crystalline powder, which is able to be particle size adjusted for a variety of formulation requirements.

Furthermore, JM has established a full suite of references of standards and impurities to facilitate CBD product development, which helps to ensure molecules are synthesised to an ultra-pure standard.

As well as synthetic chemistry expertise in controlled substances, JM also offers API manufacturing capabilities for botanical extraction and purification of cannabinoids. Based on the growing popularity of medicinal cannabinoids, JM is actively investigating the development of other cannabinoid compounds.

“As a leader in API development, we are delighted to add the high-value synthesis of cannabidiol to our expanding portfolio of Pharma solutions,” said Paul Evans, VP Generic Products and Solutions at JM.

“This will enable companies to easily explore the medicinal properties of cannabinoids, and combined with our development and manufacturing capabilities, deliver novel treatments and medicines to patients.”

SOURCE: www.manufacturingchemist.com/news

Teva wins FDA OK for crucial migraine drug

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Vital to Teva’s turnaround, Ajovy will challenge Novartis/Amgen’s Aimovig.

Teva has secured US approval for migraine drug Ajovy, a key part of new chief execeutive Kåre Schultz’s turnaround plan for the company.

The FDA approved the CGRP inhibitor for the prevention of migraine in adults with either monthly or quarterly dosing, based on two phase 3 studies showing that it could reduce the number of migraine days over a 12 -week period compared to placebo.

Ajovy (fremanezumab) is the second CGRP inhibitor to be approved for marketing after Novartis and Amgen’s Aimovig (erenumab), which got the green light in May and is dosed monthly, and ahead of late-stage rivals from Eli Lilly (galcanezumab) and Alder Biopharma (eptinezumab).

Teva would have been earlier to market but the FDA review for its products was delayed by three months while it sorted out some manufacturing issues. With Lilly due to hear from the FDA any day now, potentially setting up a three-way marketing battle, the Israeli drugmaker will be hoping the three-monthly dosing option will be popular with doctors and patients – particularly as there seems to be little to separate the antibodies when it comes to efficacy.

In a statement, Teva said that it had set a list price of $575 for the monthly dose and $1,725 for the quarterly dose, giving an annual cost of $6,900 before rebates and discounts – exactly the same level as Novartis and Amgen positioned Aimovig, and in line with stablished therapies like Allergan’s Botox.

It is estimated that with discounts the annual cost of the drugs could be around the $5,000 mark. However, the US-based Institute for Clinical and Economic Review (ICER) said recently that – even at that lower price – the new drugs should only be used with prior authorisation and after other preventive options such as Botox and anti-epilepsy drugs like topiramate because their long-tern safety is still unclear.

It is estimated that more than 36 million people in the US suffer from migraines, with around 40% of these would be suitable for preventive treatment, and that has led some analysts to predict buoyant sales of the CGRP inhibitors.

GlobalData said recently that it expects the market for migraine drugs to grow at more than 10% a year to reach almost $9bn by 2026, with the prevention category driven by the injectable CGRP inhibitors.

”Market share is going to come down to…variables such as frequency of administration, position to market, and market access strategy,” according to GlobalData analyst Rahael Maladwala.

“Each drug has its own set of advantages; Aimovig will be the first to market, Eli Lilly’s has significant experience in marketing drugs, and an extensive sales force, while both of the other drugs have a quarterly dosing regimen compared to monthly.”

On balance, GlobalData is putting Aimovig out in front in 2026 given its first-mover advantage generating nearly $1.4bn in sales in the seven major pharma markets (US, France, Germany, Italy, Spain, UK, and Japan).  Teva filed Ajovy in Europe earlier this year, but here it will also lag behind Aimovig as Novartis and Amgen picked up EMA approval for their drug in July.

Analysts at Jefferies have suggested Ajovy could become a $500m product in 2022, which will go some way towards counteracting the decline in sales of Teva’s blockbuster multiple sclerosis therapy Copaxone (glatiramer acetate), its rapidly ageing cash-cow product.

Since Schultz took the helm last year, Teva has been shedding staff and restructuring the business to cut costs, selling off its women’s health business for almost $2.5bn in order to pay down a very high level of debt stemming from its $39bn acquisition of Allergan’s generics business Actavis in 2015.

Ajovy is one of 23 New Drug Application (NDA) approvals Teva is targeting between fiscal 2019 and fiscal 2023 to help replace Copaxone and drive top-line growth, along with other new products such as movement disorder therapy Austedo (deutetrabenazine).

SOURCE: www.pmlive.com/pharma_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

Low dose Aspirin daily – benefits and risks measured in two studies

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Low dose Aspirin has been advocated to reduce the risk of a second heart attack or a stroke or other heart problems in persons who have had one episode.

A major new study published in the Lancet, has found that taking Aspirin to prevent the first heart attack fails to work. In fact the risks of taking low dose Aspirin to prevent the first heart attack outweigh the benefits.

There have been numerous studies on the prophylactic and protective effect of this low cost medication. While certain studies have shown that it can prevent first strokes or heart attacks in persons who are at a moderate risk of heart attacks and strokes, some studies have not shown significant benefit.

Aspirin has also been tested in people with cardiac disease risk factors such as diabetes, high blood pressure, smoking, high cholesterol etc. In diabetes for example Aspirin intake on a regular basis may offer some protection against heart attacks or strokes but the risk of serious bleeding as a side effect of the drug remains high. Aspirin was also purported to have anti-cancer or cancer preventing properties. This has also been negated in studies. In the studies the researchers have used 100 milligrams per day.

Study leader Dr. Jane Armitage of the University of Oxford in England said, “There’s been a lot of uncertainty among doctors around the world about prescribing aspirin. If you’re healthy, it’s probably not worth taking it.” The study was presented this Sunday (26th August 2018) at the European Society of Cardiology meeting in Munich.

In this latest study, the team of researchers from Boston used aspirin or placebo pills on 12,546 participants. These participants had a moderate risk of suffering a heart attack or stroke and other health problems. The participants were followed up for five years and it was seen that 4 percent persons in each group had a heart problem. These participants took medications for lowering blood pressure and cholesterol explained Dr. J. Michael Gaziano of Brigham and Women’s Hospital. Side effects such as internal bleeding or stomach bleeding, was mild but seen twice as much with Aspirin than the placebo pills. Aspirin users also complained more of reflux, abdominal pain, indigestion etc. This new study was sponsored by Bayer.

Low dose aspirin/fish oil supplements in diabetics

In yet another study, researchers from Oxford, randomly assigned 15,480 adults with Type 1 or 2 diabetes with aspirin or 1 gram of fish oil, both the aspirin and fish oil or placebo pills. The pills were administered each day.

The participants were followed for seven and a half years. Heart problems were lower among aspirin users. The risk of bleeding however was raised with aspirin. Fish oil supplements did not help though said study leader Dr. Louise Bowman of the University of Oxford. This study was published by the New England Journal of Medicine.

The British Heart Foundation sponsored the study. Bayer and Mylan provided aspirin and the fish oil supplements, respectively.

SOURCE: www.news-medical.net/news

Roche SMA drug shines in study as costly new therapies advance

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A drug being co-developed by Roche to treat spinal muscular atrophy (SMA) helped improve development scores in babies with the genetic disease, a study released on Monday showed, as the race heats up for therapies destined to be among the drug industry’s most expensive.

PTC Therapeutics, which struck a licensing deal with Roche in 2011 for its SMA program, said more than 90 percent of babies with severe Type 1 SMA given the RG7916 drug achieved a greater than four-point increase in a test to measure their neuromuscular progress six months after treatment began.

PTC shares, which are listed on the Nasdaq, rose as much as 30 percent.

The companies hope their medicine, also known as risdiplam, will be approved to take on rival drug Spinraza from Biogen, which sells for $750,000 for the first year of therapy and about $375,000 annually after that.

Novartis is also quickly advancing in the SMA field with its $8.7 billion acquisition this year of U.S.-based Avexis that is working on a gene therapy for the disorder.

Analysts from Barclays project Novartis’s one-time treatment could run to $1.25 million per patient.

“We are delighted that up to 6.5-fold increase of protein production has translated into clinical impact for these babies,” PTC Chief Executive Stuart Peltz said in a statement about the RG7916 study, adding no babies required a tracheostomy or permanent ventilation since the study began, and no baby lost his or her ability to swallow.

SMA is an often-deadly genetic neuromuscular disorder caused by a missing or defective gene that normally produces a protein needed for development of motor neurons in the spinal cord. This leads to muscle wasting. Many babies with the severest form of the disorder die, while others never stand or walk.

Barclays analyst Emmanuel Papadakis expects that if Roche and PTC’s data holds up on RG7916 with regulators, it could become a fierce competitor to Spinraza, now the only licensed SMA therapy.

Cowen analysts also said on Monday they view PTC’s update as very encouraging for approval — and for competitiveness versus Spinraza, which is administered into the spine about four times a year. Roche’s and PTC’s drug is taken orally.

Novartis Chief Executive Vas Narasimhan also has high hopes for his own newly acquired SMA treatment, saying in April after the Swiss company bought Avexis that its gene therapy has “multi-billion dollar peak sales potential”.

SOURCE: www.pmlive.com/pharma_news

Prothena guts workforce by more than half, after trial failure

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Prothena Therapeutics has acted drastically by slashing staff numbers by more than half, after the failure of two pivotal trials for its lead candidate.

The company subsequently ditched NEOD001, when trials revealed that placebo treatment outperformed the drug candidate in the treatment of AL amyloidosis.

The reorganisation sees 63 positions cut away from the Dublin-based company, representing a shedding of 57% of the entire staff at the company.

As is usually the case, the process was necessitated to stymie cash losses during 2018 – it projected its estimated net cash burn for the year to be $40 million to $50 million, driven by a net loss of $170 million to $185 million.

Luckily for the biotech, it’s had some backers that were betting that its work would produce results and so estimates that it still have a relatively healthy $421 million in cash to end the year on.

One notable backer is renowned UK investor, Neil Woodford, who had to defend his investment in biotech in a blog post immediately after the trial failures were announced – pointing out strengths from within the Prothena’s pipeline and suggesting its partnership with Roche was one reason to keep faith with the biotech.

In the announcement regarding its restructuring, Prothena followed suit, with Gene Kinney, President and Chief Executive Officer of Prothena, saying: “As we move forward, we have the resources to support the advancement of our pipeline through meaningful milestones and we will focus on developing neuroscience programs that we believe have a potential to offer significant benefit to patients. This includes our two clinical-stage programs PRX002/RG7935, currently in Phase 2 development in the PASADENA study in patients with early Parkinson’s disease, and PRX004, which recently initiated a Phase 1 study in patients with ATTR amyloidosis.”

PRX002/RG7935 is both being developed in collaboration with Roche and there will be significant hopes placed on this candidate to pull the biotech out of a tricky spot; however, with the treatment being for patients with Parkinson’s disease, it’s a fairly risky bet given the dearth of disease-modifying treatments for the condition.

SOURCE: www.pharmafile.com/news/517476

ABPI expert urges to find new ‘blockbuster treatments’ for brain tumors

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With the Government set to invest an additional £20 million into the research, diagnosis and development of treatments for brain tumours, we need to talk more about how we are going to find the next blockbuster treatments for these devastating diseases.

Nearly 11,500 people are diagnosed with a brain tumour every year in the UK with fewer than 15% surviving beyond 10 years. This week’s announcement from the from the Department of Health and Social Care – following the death of Dame Tessa Jowell – that they would be doubling investment for brain cancer research to £40 million is a welcome commitment to helping achieve a goal our industry shares: finding innovative new treatments and cures for these diseases.

The science is advancing in laboratories here in the UK and around the world, funded and supported by charities, universities and the pharmaceutical industry, collectively we are working to fight back against this terrible disease.

Among the 7,000 medicines currently being developed by the global pharmaceutical industry, there are 58 medicines in the pipeline for brain tumours, including gliomas. Companies are actively working to find better ways to speed up medicines development to get treatments to patients sooner.

In her speech to the House of Lords in January, Dame Tessa Jowell talked candidly about her glioblastoma diagnosis and called for greater collaboration in the fight against cancer. She also talked about the speeding up of drug trials by testing more than one at a time, saying: “I am not afraid, but I am fearful that this new and important approach may be put into the ‘too difficult’ box.”

The type of clinical trials Tessa Jowell talked about have many different names: adaptive randomisation, drop-the-loser, adaptive dose-finding, adaptive seamless and the list goes on.

The one thing they all have in common is flexibility. In trials like this – that we call adaptive design clinical trials – researchers can see how patients are responding to treatments and then change or stop parts of the trial in real time.

When used appropriately, trials like this may improve efficiency, reduce cost, maximize information gained and minimize risk to the patients and sponsors. Ultimately, drug development can be accelerated so that the right treatments can be delivered rapidly to the right patients. The UK is seen as a pioneer of innovative clinical trials and this involves collaboration between academia, the NHS, industry and medical research charities –  we must ensure we keep it that way in the future.

The issue is that these clinical trial types are not easy to design, plan or execute. An adaptive design will not rescue a poorly planned trial or ineffective treatment.

We need to make sure the regulatory authorities in the UK are not seen as a barrier to innovation; the MHRA and HRA are open to discussion and we need to encourage researchers and pharmaceutical companies to start conversations with them early in the process of planning an innovative clinical trial.

We think that adaptive design clinical trials could be the solution to speeding up the research and development of not only brain tumor treatments, but for all sorts of diseases. Research into small or rare patient populations could really benefit from these trials since they help us quickly rule out the drugs or drug combinations that aren’t working and give more patients the option to contribute to research and clinical trials.

We’re not alone. In February, the Department of Health and Social Care published their brain tumor research report which stated that, because brain tumors are one of the areas that have small patient populations, we need to think differently about how we conduct clinical trials and incorporate innovative trial designs.

The report provided practical recommendations for how we can work collaboratively to make quicker progress in this area. The next steps are to build on the UK’s existing strengths, ensure we have access to researchers with the right skills, and make sure that the right infrastructure is in place for us to make really make progress in this area.

Alongside their funding announcement, we welcome the Government’s commitment this week to accelerate the use of adaptive design trials. When used appropriately, drug development can be accelerated so that the right treatments can be delivered rapidly to the right patients – and that’s where the real benefit lies.

As we look to the future of cutting-edge research and development for blockbuster treatments, we know we need to make the case for innovative clinical trial design, talk more about the amazing science our researchers, companies and NHS are pioneering and encourage them to have open conversations with the UK regulators to ensure that innovative clinical research is safe and effective.

Together, we won’t rest until devastating brain tumours are a thing of the past.

SOURCE: www.news-medical.net/news

Boehringer partners with Bactevo on drug discovery

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Boehringer Ingelheim is bolstering its drug discovery efforts with a new collaboration with UK tech-enabled research firm Bactevo.

Enabled by advance machine learning, Bactevo claims that its Totally Integrated Medicines Engine platform (TIME) will be able to bring about a paradigm shift in the speed, efficiency and quality of drug discovery, as well as dramatically enhanced safety profiling.

Aiming to identify novel small molecule lead compounds, the deal will see Bactevo use its TIME and synthetic chemistry technology to further enhance speed, efficiency and quality when detecting novel in vivo enabled leads.

In addition to working with partners to develop novel first-in-class medicines, Bactevo is also developing breakthrough medicines for the treatment of diseases that involve defects in mitochondrial function, such as MELAS and LHON. It is also targeting diseases of the central nervous system, such as Parkinson’s, Alzheimer’s and Amyotrophic Lateral Sclerosis (ALS).

Bactevo will receive upfront payments and research funding, although that specific amount was not disclosed at the time of writing.

The tech group could also be eligible to receive payments for certain research, development and commercialisation milestones.

David Williams, chief executive officer of Bactevo, said: “We are pleased to be commencing this highly complementary collaboration with Boehringer Ingelheim.

“It combines our cutting-edge TIME drug discovery platform with the powerful therapeutic drug development and commercialisation experience at Boehringer Ingelheim to create much needed new medicines in areas outside of our current therapeutic focus.”

SOURCE: www.pmlive.com/pharma_news