Novartis is buying US gene therapy group AveXis in a deal valued at $8.7 billion.

The firm has agreed to pay $218 per share for the business, just days after it banked $13 billion from the sale of its stake in the consumer healthcare joint venture owned with GlaxoSmithKline.

The Swiss drug giant is expecting the move to expand its position in the gene therapy space, supporting both its ambition to be a leader in neurodegenerative diseases “and our Neuroscience franchise priorities to strengthen our position in devastating paediatric neurological diseases such as SMA,” said Paul Hudson, chief executive of Novartis Pharmaceuticals.

Read more: http://www.pharmatimes.com/news/novartis_snaps_up_avexis_in_$8.7bn_deal_1230899

Researchers at RMIT University in Australia have developed an artificial enzyme that uses visible light to break down and destroy bacteria.

The NanoZymes work by combining light with moisture to create a biochemical reaction that results in OH radicals that break down bacteria.

When tested using visible light sources the activity of these enzymes increased 20-fold, creating holes in the bacterial cells and killing them.

Researchers hope that in the future the enzymes could be activated by sunlight.

RMIT lead researcher Vipul Bansal said: “For a number of years we have been attempting to develop artificial enzymes that can fight bacteria, while also offering opportunities to control bacterial infections using external triggers and stimuli. Now we have finally cracked it.

Read more: https://www.pharmaceutical-technology.com/news/new-artificial-enzyme-employs-light-bacteria/

Biogen and Samsung Bioepis have laid to rest a long-running patent wrangle with AbbVie regarding a biosimilar form of the latter’s blockbuster Humira.

The parties have now come to an agreement over the commercialisation of Biogen and Samsung Bioepis’ Imraldi (biosimilar adalimumab), the specific terms of which are being kept under wraps.

It was disclosed, however, that under the settlement AbbVie will grant patent licenses for the use and sale of Imraldi in Europe, on a country-by-country basis, while Biogen and Samsung Bioepis will make royalty payments in return.

Read more: http://www.pharmatimes.com/news/biogen,_samsung_bioepis_and_abbvie_settle_patent_dispute_1230636

Boehringer Ingelheim and OSE Immunotherapeutics, a biotechnology company focused on the development of innovative immunotherapies, have announced a collaboration and exclusive worldwide collaboration and license agreement to jointly develop OSE-172, a SIRP-alpha antagonist targeting myeloid lineage cells.
SIRP-alpha is a receptor expressed by myeloid lineage cells such as Dendritic Cells (DCs), tumor-associated macrophages (TAMs) and Myeloid-Derived Suppressor Cells (MDSCs). In targeting SIRP- alpha, OSE-172 prevents the ligand CD47 from binding to and triggering the cellular inhibitory effects of SIRP-alpha. OSE-172 has the potential to enhance anti-tumor immunity by improving T cell activity through enhancement of DC antigen presentation functionality, potentiating the phagocytic and inflammatory properties of macrophages in the tumor microenvironment and enabling differentiation of MDSCs to an effector state.

Read more: http://www.worldpharmanews.com/boehringer-ingelheim/4352-boehringer-ingelheim-and-ose-immunotherapeutics-announce-global-immuno-oncology-partnership-to-develop-a-pioneering-checkpoint-inhibitor-for-the-treatment-of-advanced-solid-tumors

AstraZeneca’s MedImmune has inked a deal with Israeli group Compugen giving it access to the firm’s experimental bi- and multi-specific antibody products.

Bispecific antibodies are genetically engineered to simultaneously bind to two (bi-specific) or more (multi-specific) different types of antigen. They enable multiple mechanisms of action for treating disease within a single molecule.

Under the deal, MedImmune has gained the right to create multiple products, and will be solely responsible for all research, development and commercial activities.

In return, Compugen will receive a $10 million upfront payment and potentially up to $200 million in development, regulatory and commercial milestones for the first product, as well as tiered royalties on future product sales.

Read more: http://www.pharmatimes.com/news/az_signs_up_for_access_to_compugens_pipeline_1230099

Takeda, Japan's largest drugs firm by sales, has said it is considering a possible offer for the UK's Shire.
The Takeda announcement sent shares in the UK pharmaceuticals company up as much as 25% in morning trading.
Big drugs names have long been rumoured to have been circling Shire after a takeover by US firm Abbvie fell through in 2014.
In mid-December last year, speculation on a possible takeover bid sent Shire shares to the top of the FTSE 100.
Takeda said its offer "is at a preliminary and exploratory stage and no approach has been made to the board of Shire".
However, Takeda said it was interested in the firm to strengthen its cancer, stomach and brain drugs offerings.
Takeda must announce that it intends to make a firm offer for Shire by the end of 25 April.

Read more: http://www.bbc.co.uk/news/business-43568725

Roche’s defence of its haemophilia drug Hemlibra against a patent infringement challenge by Shire has been buoyed by a Japanese ruling.
A Tokyo court has found in favour of the Swiss group and recommended that Shire’s challenge against majority-owned Japanese affiliate Chugai should be dismissed. While the ruling doesn’t have a direct bearing on additional challenges outside Japan, Roche has been buoyed by the result.
In a statement, the company said the court ruled that Hemlibra (emicizumab) “does not infringe patent 4313531, held by Shire’s wholly-owned subsidiaries Baxalta Inc and Baxalta GmbH”.

Read more: http://www.pmlive.com/pharma_news/first_blood_to_roche_in_hemlibra_patent_fight_with_shire_1229612

Ipsen’s Cometriq can now be routinely funded by the NHS to treat patients with medullary thyroid cancer (MTC) in England and Wales.

The National Institute for Health and Care Excellence has issued guidelines recommending the drug as an option for treating progressive MTC in adults with unresectable, locally advanced or metastatic disease.

Read more: http://www.pharmatimes.com/news/final_nice_green_light_for_ipsens_cometriq_1229604

TiGenix and Takeda have announced that Alofisel (darvadstrocel) has received EU approval for the treatment of complex perianal fistulas in Crohn’s disease.

The drug has been approved for the treatment of complex perianal fistulas in adult patients with non-active/mildly active luminal Crohn’s disease where fistulas have not responded to at a conventional or biologic therapy.

Perianal fistulas occur when a channel develops between the end of the bowel and the skin near the anus and usually require surgical treatment. They are a severe and debilitating complication of Crohn’s disease for which there is currently no effective treatment.

Alofisel, which is TiGenix’s lead product, works by reducing the release of pro-inflammatory cytokines at inflammation sites.

It is the first allogeneic stem cell therapy to receive central marketing authorisation approval in Europe.

Read more: https://www.pharmaceutical-technology.com/news/eu-allogenic-crohns-alofisel/

A Vancouver-based research team led by Canada's most cited neuroscientist, Dr. Patrick McGeer, has successfully carried out studies suggesting that, if started early enough, a daily regimen of the non-prescription NSAID (nonsteroidal anti-inflammatory drug) ibuprofen can prevent the onset of Alzheimer's disease. This means that by taking an over-the-counter medication, people can ward off a disease that, according to Alzheimer's Disease International's World Alzheimer Report 2016, affects an estimated 47 million people worldwide, costs health care systems worldwide more than US$818 billion per year and is the fifth leading cause of death in those aged 65 or older.
The Alzheimer's Association estimates that there are more than 5 million cases in the United States alone, with a new case being identified every 66 seconds. The annual cost to the country in 2017 is estimated have been $259 billion, with that figure predicted to potentially rise to $1.1 trillion by 2050.

Dr. McGeer, who is President and CEO of Vancouver-based Aurin Biotech, and his wife, Dr. Edith McGeer, are among the most cited neuroscientists in the world. Their laboratory is world-renowned for their 30 years of work in neuroinflammation and neurodegenerative diseases, particularly Alzheimer's disease. A paper detailing Dr. McGeer's most recent discoveries were published Friday in the prestigious Journal of Alzheimer's Disease. (Journal of Alzheimer's Disease 62 (pp. 1219-1222).

Read more: http://www.worldpharmanews.com/research/4343-canadian-neuroscientists-say-daily-ibuprofen-can-prevent-alzheimer-s-disease

GlaxoSmithKline is buying Novartis’ stake in the firms’ consumer healthcare unit, the announcement coming just days after the drug giant said it would not pursue acquisition of Pfizer’s consumer healthcare business.

The UK drugmaker said it is buying Novartis’ 36.5-percent stake in the JV for $13 billion, securing full control of the business, which was established as part of a three-part transaction between GSK and Novartis approved by shareholders in 2014.

Under the terms of the original deal, Novartis holds the right, exercisable from 2 March 2018 to 2 March 2035, to require GSK to purchase its stake (or specified tranches of it) in the JV.

Read more: http://www.pharmatimes.com/news/gsk_to_buy-out_novartis_stake_in_consumer_health_jv_1229403

As we get older, our endurance declines, in part because our blood vessels lose some of their capacity to deliver oxygen and nutrients to muscle tissue. An MIT-led research team has now found that it can reverse this age-related endurance loss in mice by treating them with a compound that promotes new blood vessel growth.

The study found that the compound, which re-activates longevity-linked proteins called sirtuins, promotes the growth of blood vessels and muscle, boosting the endurance of elderly mice by up to 80 percent.

If the findings translate to humans, this restoration of muscle mass could help to combat some of the effects of age-related frailty, which often lead to osteoporosis and other debilitating conditions.

"We'll have to see if this plays out in people, but you may actually be able to rescue muscle mass in an aging population by this kind of intervention," says Leonard Guarente, the Novartis Professor of Biology at MIT and one of the senior authors of the study. "There's a lot of crosstalk between muscle and bone, so losing muscle mass ultimately can lead to loss of bone, osteoporosis, and frailty, which is a major problem in aging."

Read more: https://www.sciencedaily.com/releases/2018/03/180322140926.htm

Novartis has received approval from the US Food and Drug Administration (FDA) for the use of its Tasigna (nilotinib) drug to treat Philadelphia chromosome-positive chronic myeloid leukaemia in the chronic phase (Ph+ CML-CP) in children.

Ph+CML is caused by the abnormal production of protein from a gene called BCR-ABL1. A signal sent by the BCR-ABL1 protein is responsible for the production of leukemic cells. Tasigna is designed to block this signal to allow the growth of healthy blood cells.

Tasigna has been approved for use in first and second-line paediatric patients aged one year or above.

Previously, the drug was indicated for newly diagnosed Ph+CML-CP in adults and paediatric patients with Ph+CML-CP resistant or intolerant to prior tyrosine kinase inhibitor (TKI) therapy. It is also indicated for adult with Ph-CML in chronic and accelerated phase, or resistant to prior therapy.

Read more: https://www.pharmaceutical-technology.com/news/novartis-tasigna-gets-approval-for-rare-leukaemia-in-us-children/

Tuberculosis can be cured and could be eradicated. For this to happen, however, patients have to receive the right treatment. Researchers at the Makerere University and the University of Zurich were able to demonstrate that the levels of medication used are often too low. As a result, patients remained contagious with the dangerous disease for longer than necessary.
More than 4,000 people worldwide die from tuberculosis every day. This means the disease tops the list of the most common causes of death that result from a single pathogen. The people who are most affected are those also suffering from an immune deficiency, such as an HIV infection. However, the high rate of mortality isn't unavoidable: Tuberculosis can be cured if the relevant drugs are used correctly.

But this is precisely where the problem lies. The most important medications for treating tuberculosis - in Switzerland, too - date back to the 1960s: Treatment is lengthy, involves several pills, and comes with numerous side effects. People thus often discontinue their treatment, which means that patients continue to suffer from the disease and also pass it on to others. In addition, the optimal dose of tuberculosis drugs has also not yet been determined.

Read more: http://www.worldpharmanews.com/research/4338-north-and-south-cooperation-to-combat-tuberculosis

AstraZeneca’s Lokelma has been approved in Europe to treat hyperkalaemia in adult patients.

Hyperkalaemia is characterised by elevated potassium levels in the blood. Risk of developing the condition increases significantly for patients with chronic kidney disease and for those who take common life-saving medications for heart failure, such as renin-angiotensin-aldosterone system (RAAS) inhibitors, which can increase potassium in the blood.

To help prevent the recurrence of hyperkalaemia, RAAS-inhibitor therapy is often modified or discontinued, but this can compromise cardio-renal outcomes and increase the risk of death, the firm noted.

Read more: http://www.pharmatimes.com/news/eu_approves_az_potassium_binder_for_hyperkalaemia_1229022

JERUSALEM (Reuters) - Israel will invest nearly 1 billion shekels ($287 million) in a project to make data about the state of health of its population available to researchers and private companies, Prime Minister Benjamin Netanyahu said on Sunday.

Almost all of Israel’s nine million citizens belong to four health maintenance organizations (HMOs) who keep members’ records digitally, thus comprising a huge medical database.

“This is a major asset and we want to make it accessible to researchers and developers in order to achieve two things: one is preventive medicine, and the second is personal medicine tailored to each individual,” Netanyahu told his cabinet.

Nadav Davidovitch, head of the Public Health School at Ben Gurion University in southern Israel, said the country’s push to harness big data for healthcare had huge potential, but also held risks in terms of privacy and medical confidentiality.

Read more: https://www.reuters.com/article/us-israel-health/israel-to-launch-big-data-health-project-idUSKBN1H10LW

US regulators have approved Seattle Genetics’ Adcetris in combination with chemotherapy for adult patients with previously untreated Stage III or IV classical Hodgkin lymphoma.

The decision gives patients in this setting the first new treatment option in more than 40 years, the firm notes.

Read more: http://www.pharmatimes.com/news/us_expands_scope_of_seattle_genetics_adcetris_1228673

LONDON, March 21 (Reuters) - In January, GlaxoSmithKline's new head of pharmaceuticals Luke Miels issued a blunt challenge to his managers: find budget savings of 20 percent.

The plan was to pool the savings and reallocate the money to priority medicines and markets for Britain's biggest drugmaker, according to people familiar with the meeting.

The tough demand startled some GSK veterans who are used to a more consultative approach, said the sources. But it is a sign of the harder commercial edge new Chief Executive Emma Walmsley - almost 12 months into the job - is bringing to a company whose shares have moved sideways for years.

Miels - a long-time protege of AstraZeneca CEO Pascal Soriot, whose departure sparked a legal spat between the rival drugmakers - is a key lieutenant for Walmsley as she tries to overhaul the core pharma business.

The unit has lagged rivals like Novartis and Merck & Co in producing multibillion-dollar blockbusters, so Walmsley has prioritised plans to refocus both its research and marketing efforts in a drive for fewer but bigger new drugs.

Miels declined to comment in detail on his budget demand, including the savings figure of 20 percent, but confirmed the strategic goal of achieving a leaner, meaner commercial footprint to match a recently pared-down research and development (R&D) operation.

The aim is to concentrate sales and marketing resources on new products, particularly lung drug Trelegy and shingles vaccine Shingrix, in 10 big markets - ranging from the United States, major European countries and Japan to China, India and Russia, he told Reuters.

"In R&D, we're trying to pick which assets are most productive and are going to add the most value to patients. Well, we need to do the same thing in the commercial organisation," Miels said.

"That is something that will become more visible over time and it is a process that we are going through now ... the strategy is more advanced than is visible externally."

https://www.cnbc.com/2018/03/21/reuters-america-focus-glaxosmithkline-prescribes-commercial-reboot-for-pharma-division.html

A switch has been discovered that instructs blood vessel cells to become blood stem cells during embryonic development in mice. Using single-cell technology, researchers from the Wellcome Sanger Institute in Cambridge and the European Molecular Biology Laboratory in Rome discovered that two sets of specific factors in the cells work against each other, and when the balance of these changes, the vascular tube cells convert to free blood cells.

Reported in eLife, these findings could pave the way for further research into creating new blood cells for transplants and for understanding cancer development.

Blood vessels and blood cells develop from stem cells in the embryo. In fact, the blood stem cells, responsible for the generation of all blood cell types, develop from the vascular cells which line the walls of blood vessels. This process happens in fish, birds and mammals, and is critical for the formation of blood cells. However how these vascular cells decide when to transform into blood stem cells was unknown.

Read more: https://www.sciencedaily.com/releases/2018/03/180320084320.htm

Heron Therapeutics’ pain drug HTX-011 has succeeded in two phase 3 trials in patients undergoing bunionectomy and hernia repair by meeting all their primary and key secondary points.
HTX-011, which is a combination of local anesthetic bupivacaine and anti-inflammatory meloxicam, had produced statistically significant reductions in both pain intensity and need for opioids through 72 hours post-surgery.

The primary and key secondary endpoints for the Study 301/EPOCH1 in patients undergoing bunionectomy and Study 302/EPOCH2 phase 3 studies in patients going through hernia repair were identical. HTX-011 was evaluated against placebo and bupivacaine in both the trials.

The primary endpoint was pain intensity, which was assessed by the Area Under the Curve (AUC) score from 0 to 72 hours post-surgery (AUC 0-72), in comparison to placebo.

Key secondary endpoints include comparison of AUC 0-72 of pain intensity to bupivacaine solution and the total amount of opioid rescue medication used compared to placebo during 72 hours after surgery.

Read more: http://clinicaltrials.pharmaceutical-business-review.com/news/herons-pain-drug-succeeds-in-phase-3-bunionectomy-and-hernia-repair-trials-200318-6088474

March 19 (Reuters) - Arena Pharmaceuticals Inc said on Monday a higher dose of its experimental drug succeeded in improving symptoms associated with ulcerative colitis, a chronic bowel disease, in a mid-stage study, sending its shares up 24 percent.

The company's shares, which surged as much as 62 percent to $50 after the bell, were trading at $38.20.

Arena said patients receiving the oral treatment showed statistically significant improvement in stool frequency, rectal bleeding and findings on endoscopy, compared with a placebo.

The company said it planned to move the drug, etrasimod, to late-stage trials based on the data.

Read more: https://www.cnbc.com/2018/03/19/reuters-america-update-1-arena-pharmas-bowel-disease-drug-meets-study-goal-shares-surge.html

The cost of providing care for Americans with Alzheimer’s disease has hit $277 billion – eclipsing a quarter of a trillion dollars – as baby boomers age and the U.S. healthcare system struggles to find people to care for this fast-growing population.

The annual cost estimate for the deadly disease from the Alzheimer’s Association comes as Republican members of Congress led by U.S. House Speaker Paul Ryan consider cutting entitlement programs like Medicaid health coverage for poor Americans and Medicare health insurance for the elderly.

Read more: https://www.forbes.com/sites/brucejapsen/2018/03/20/alzheimers-costs-reach-277-billion/#23c313985c09

In July 2015, 86-year-old Douglas Waters developed severe age-related macular degeneration (AMD). He struggled to see things clearly, even when up close. A few months later, he became part of a clinical trial that used stem cell-derived ocular cells developed in part by researchers at UC Santa Barbara. His retinal eyepatch was implanted at Moorfields Eye Hospital, a National Health Service (NHS) facility in Waters' hometown of London, England.
In the months before Waters' surgery, his vision was poor and he couldn't see anything out of his right eye. After the surgery, his eyesight improved so much that he could read the newspaper and help his wife with gardening.

The results of this groundbreaking clinical study, published in Nature Biotechnology, describe the safe and effective implantation of a specially engineered patch of retinal pigment epithelium cells derived from stem cells to treat people with sudden severe sight loss from wet AMD - the form that afflicted Waters. This is the first description of a completely engineered tissue that has been successfully used in this way.

Read more: http://www.pharmanews.eu/research/1796-stem-cells-treat-macular-degeneration

A stem cell transplant may be an effective treatment for multiple sclerosis, an international study suggests.

The treatment involves using cancer drugs to decimate a patient's immune system and then restarting it with a stem cell transplant, BBC News reported.


The study included 100 patients in the United States, England, Sweden and Brazil with relapsing remitting MS, in which attacks are followed by periods of remission.

The patients received either stem cell transplantation or drug treatment (control group). After one year, only one patient of 52 in the stem cell transplant group had a relapse, compared with 39 of 50 in the drug group, BBC News reported.

Read more: https://www.drugs.com/news/stem-cell-transplant-ms-shows-promise-69046.html

Pfizer and Astellas Pharma have obtained priority review status from the US Food and Drug Administration (FDA) for the supplemental new drug application (sNDA) of Xtandi (enzalutamide) to treat patients with non-metastatic castration-resistant prostate cancer (CRPC).

Xtandi is an inhibitor of androgen receptor and received FDA approval in 2012 for metastatic CRPC patients who were previously treated with docetaxel. In 2014 it was expanded to include patients with metastatic CRPC.

Patients with non-metastatic CRPC lack clinically detectable evidence of the cancer spreading, apart from a rapid increase in their prostate-specific antigen (PSA) levels. The majority of patients go on to develop metastatic CRPC.

Read more: https://www.pharmaceutical-technology.com/news/pfizers-snda-xtandi-gets-priority-review-prostate-cancer/