Researchers at The University of Texas at Austin have developed a new approach to treating cancer using enzyme therapy. The enzyme, PEG-KYNase, does not directly kill cancer cells but instead empowers the immune system to eradicate unwanted cells on its own. PEG-KYNase is designed to degrade kynurenine, a metabolite produced by numerous tumors that suppresses the immune system. The UT team's findings were published in a recent issue of Nature Biotechnology.
A healthy, fully functioning immune system can combat the spread of cancer cells and eliminate tumors by itself. However, tumors have evolved in multiple ways to suppress the immune system, leading to the growth and metastasis of cancer cells.

"Our immune system constantly polices the body and normally recognizes and eliminates cancerous cells," said Everett Stone, research assistant professor in the College of Natural Sciences' Department of Molecular Biosciences and co-author of the study. "Kynurenine acts as a roadblock to immune cells that impedes normal surveillance; our drug removes this obstacle."

Read more: http://www.worldpharmanews.com/research/4522-new-cancer-treatment-uses-enzymes-to-boost-immune-system-and-fight-back

Dublin, Ireland-based Jazz Pharmaceuticals will be celebrating news of European approval of its leukaemia drug Vyxeos.

The European Commission is allowing use of the drug to treat adults with newly diagnosed, therapy-related acute myeloid leukaemia (t-AML) or AML with myelodysplasia-related changes (AML-MRC).

Vyxeos is an advanced liposomal formulation that delivers a synergistic molar ratio of daunorubicin and cytarabine, and is “the first chemotherapy to demonstrate an overall survival advantage versus the standard of care in a Phase III study of older adult patients” with these conditions, said Daniel Swisher, Jazz’ president and chief operating officer.

Read more: http://www.pharmatimes.com/news/jazz_vyxeos_wins_eu_nod_for_certain_acute_myeloid_leukaemias_1250597

NHS funding for Ipsen’s Cabometyx is now being recommended by the National Institute for Health and Care Excellence to treat kidney cancer.

In a Final Appraisal Determination, the cost watchdog concluded that the drug can be used as a first-line treatment for adults with advanced renal cell carcinoma (RCC).

Cabometyx (cabozantinib) is currently routinely available on the NHS as a treatment for adults with advanced RCC following prior vascular endothelial growth factor (VEGF)-targeted therapy.

Earlier this year, the drug’s scope was expanded in Europe to include treatment-naïve adults with advanced RCC, on the back of data showing patients given Cabometyx had a median progression free survival of 8.6 months versus 5.3 months for those treated initially with sunitinib (Pfizer’s Sutent), marking a 52 percent reduction in the hazard for progression or death.

Read more: http://www.pharmatimes.com/news/nice_u-turn_recommends_ipsens_cabometyx_for_kidney_cancer_1250596

US regulators have further expanded the scope of Bristol Myers Squibb’s Opdivo, approving its use for a previously treated small cell lung cancer.

The immunotherapy is now the first and only immuno-oncology treatment option for patients with metastatic small cell lung cancer (SCLC) whose cancer has progressed after platinum-based chemotherapy and at least one other line of therapy.

Clearance for this indication was granted by the US Food and Drug Administration under the regulator’s accelerated approval pathway based on overall response rate (ORR) and duration of response (DOR) data, so continued approval may be contingent upon verification of clinical benefit in confirmatory trials.

Read more: http://www.pharmatimes.com/news/us_nod_for_opdivo_in_pre-treated_small_cell_lung_cancer_1249436

Ziylo and Novo Nordisk A/S today announced that Novo Nordisk has acquired all of the shares of Ziylo, a University of Bristol spin-out company based at 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 thus overall reduce the burden of diabetes for people living with the disease.

Read more: http://www.worldpharmanews.com/novonordisk/4515-novo-nordisk-acquires-ziylo-ltd-to-accelerate-its-development-of-glucose-responsive-insulins

Patient-centred healthcare is here to stay, so how can pharma sales embrace it?

Patient centricity is the new rallying call in healthcare with the potential to change the way pharma does business. It’s no longer just a talking point either; increasing numbers of companies are looking to incorporate the ethos into multiple aspects of their businesses with a concerted effort of putting the patient and their needs firmly at the centre. This is being seen in the new wave of patient education and disease awareness materials, as well as in drug development and innovative clinical trial designs. Indeed, the globalisation of the patient voice has prompted industry associations, such as the International Federation of Pharmaceutical Manufacturers and Associations, to provide guidance and education about ethical and effective patient involvement.

“Developing medicines for patients is not new but developing medicines with patients is,” explains Professor Karen Woolley, global lead, patient partnerships at Envision Pharma Group. “We are seeing more and more clients move beyond the ‘Why involve patients?’ to ‘How do we involve patients?’, and this shift is changing the way companies do business.” She believes the voice of the patient will get stronger, providing positive and transformational change for the industry. However, she adds, the science of ethical and effective patient involvement is still in its infancy.

Read more: http://www.pharmatimes.com/magazine/2018/julyaugust_2018/patient_centricity_through_the_shop_window

GlaxoSmithKline unit ViiV has shown that a two-drug HIV regimen given just once a month by injection can match the efficacy of daily, oral dosing with triple therapy.
The results of the ATLAS study enrolled people with HIV who were already controlling the virus using an oral regimen of two nucleoside reverse transcriptase inhibitors (RTIs) plus a third drug. After switching to the injectable regimen of integrase inhibitor cabotegravir and Johnson & Johnson’s non-nucleoside RTI rilpivirine, the patients showed similar efficacy after 48 weeks’ treatment, with ViiV’s combination just as good at suppressing viral load.

If approved, the new regimen could allow HIV patients to be freed from having to take daily doses of antiretrovirals for the first time drug treatment for the infection first became available in 1987. It also gives ViiV another fillip in its long-standing battle for supremacy in the HIV market with arch-rival Gilead Sciences.

Read more: http://www.pmlive.com/pharma_news/viiv_chalks_up_another_win_for_two-drug_hiv_regimen_1248938

Researchers at Lund University in Sweden, have in collaboration with colleagues in Copenhagen and Singapore, mapped how the body's own peptides act to reduce infection and inflammation by deactivating the toxic substances formed in the process. The study is published in Nature Communications and the researchers believe their discovery could lead to new drugs against infection and inflammation, for example in wound healing.

The toxins that are neutralised, known as lipopolysaccharides (LPS), come from the bacterial cell walls and generate an inflammatory reaction. The reaction is a necessary part of our immune defence system in which our bodies respond quickly and fight invasive bacteria. However, it can be over-activated and become harmful, as observed in infected skin wounds, infections in various organs, or in the case of bacteria spreading to the blood, which can lead to sepsis.

Read more: http://www.worldpharmanews.com/research/4511-the-medicine-of-the-future-against-infection-and-inflammation

For the first time, Mount Sinai researchers have identified a way to make large numbers of immune cells that can help prevent cancer reoccurrence, according to a study published in August in Cell Reports. The researchers discovered a way to grow the immune cells, called dendritic cells, at large scale in the lab to study them for their potential use in highly refined cancer vaccines to prevent patients' cancer from coming back. Dendritic cells are very rare in the body, so it has not been possible to isolate them from patients for generating vaccines without great expense and highly complicated methods.
The ability to grow many types of dendritic cells, which act as sentinels that warn the immune system to gear up with weapons specific to the disease it is attacking, will allow researchers to study their roles in the immune system. This discovery is especially important because dendritic cells aren't limited to one type of cancer and can attack all types of cancer with very limited side effects.

"The ability to generate large numbers of distinct types of human dendritic cells in vitro is critical for accelerating our understanding of dendritic cell biology and to harness them clinically," said Nina Bhardwaj, MD, PhD, Director of Immunotherapy at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai. "Our system will also be useful for translational applications including in vitro drug and vaccine testing on different dendritic cell types."

Read more: http://www.worldpharmanews.com/research/4512-researchers-artificially-generate-immune-cells-integral-to-creating-cancer-vaccines

Scientists have discovered a marker in the blood that could predict the risk of developing kidney cancer.

The research, supported by Cancer Research UK, the IARC and the NIH, found that measuring levels of the protein KIM-1 could determine whether a person is at risk of developing kidney cancer in the following five years.

The findings also suggest that the higher the concentration of KIM-1 the greater the their risk of developing the disease.

The researchers believe that in future KIM-1 levels could be used with imaging to either confirm kidney cancer or rule out the disease.

“This work is a big step forward; KIM-1 is the only blood biomarker shown prospectively to distinguish between people at high and low risk of kidney cancer. But there’s a lot more work to do before we could envisage this in the clinic,” said Dr David Muller, Cancer Research UK-funded co-first author based at Imperial College London.

Read more: http://www.pharmatimes.com/news/blood_test_could_detect_kidney_cancer_up_to_five_years_earlier_1248937

Pfizer's Herceptin (trastuzumab) biosimlar Trazimera has been approved by the European Comission.

Trazimera is the company's first oncology biosimilar and fourth biosimilar overall to receive European approval. Like its originator it will be licesnced to treat human epidermal growth factor (HER2) overexpressing breast cancer and HER2 overexpressing metastatic gastric or gastroesophageal junction adenocarcinoma.

Read more: http://www.pharmatimes.com/news/ema_approves_pfizers_herceptin_biosimilar_1246372

For years, Indivior has made the bulk of its revenues from opioid addiction treatment Suboxone. Now, it’s taken a step towards reducing its reliance on that cash cow, which is under pressure from generic competition.
The UK drugmaker has just won FDA approval for Perseris, a monthly injectable formulation of atypical antipsychotic risperidone, to treat schizophrenia.
It will launch in competition to other monthly therapies, such as Lundbeck/Otsuka’s market leading Abilify Maintena (aripiprazole) and Johnson & Johnson’s Invega Sustenna (paliperidone) but, for now, Indivior isn’t predicting when that will take place. In a press release, the company says its launch – originally slated for the fourth quarter – will now be reviewed in light of a recent court decision relating to Suboxone (buprenorphine/naloxone) in the US.

Read more: http://www.pmlive.com/pharma_news/indivior_push_to_diversify_boosted_by_schizophrenia_drug_approval_1246373

The EMA has approved Norvartis’ Aimovig (erenumab), making it Europe’s first treatment specifically designed to prevent migraine.

The drug works by blocking the calcitonin gene-related peptide (CGRP) receptor, thought to be involved in the transmission of the pain signals associated with migraine.

Migraine is a complex and debilitating neurological condition that affects each individual differently. Over 610,000 people in the UK are estimated to experience chronic migraine, with the 2010 Equality Act classing migraine as a disability should the condition have a long term, substantial impact on day to day or work-related activities.

Read more: http://www.pharmatimes.com/news/novartis_migraine_drug_gets_ema_approval_1246290

Celgene has new phase III data that could add yet another string to blockbuster Revlimid’s bow – as a treatment for patients with non-Hodgkin’s lymphoma who have relapsed or failed to respond to front-line therapy.
The AUGMENT trial paired Revlimid (lenalidomide) with Roche’s established lymphoma therapy Rituxan (rituximab) in patient with two types of NHL – follicular lymphoma (FL) and marginal zone lymphoma (MZL) – and showed a significant increase in progression-free survival (PFS) compared to Rituxan plus placebo.
There was also a trend towards an improvement in overall survival with Revlimid/Rituxan (known as R2), although the trial still has a while to run and that may reach statistical significance when the data matures.
Celgene is planning to file for approval of Revlimid in the new indication early next year, adding to its current uses in multiple myeloma, mantle cell lymphoma, and anaemia associated with myelodysplastic syndromes from which it generated sales of $8.19bn last year.

Read more: http://www.pmlive.com/pharma_news/celgenes_revlimid_gets_a_win_in_late-stage_lymphoma_1245500

The Lancet has published full results of a Phase II trial showing the benefit of Lilly’s JAK inhibitor Olumiant in the treatment of global systemic lupus erythematosus (SLE).

According to the data, a statistically significant proportion of patients treated with 4mg of Olumiant (baricitinib) achieved resolution of their SLE-related arthritis or rash compared to placebo at week 24, thus meeting the primary endpoint of the trial.

Also, compared to placebo, treatment with the drug resulted in a statistically significantly greater proportion of patients with lower overall disease activity, a key secondary endpoint.

Based on these data, Lilly will initiate Phase III trials to evaluate the safety and efficacy of Olumiant for the treatment of SLE.

Read more: http://www.pharmatimes.com/news/lilly_to_progress_olumiant_into_phiii_for_lupus_1245497

Life-threatening fungal infections affect more than two million people worldwide. Effective antifungal medications are very limited. Until now, one of the major challenges is that the fungal cell wall is poorly understood, which has impeded the development of effective antifungal medications that target the cell wall. However, an LSU chemist has identified for the first time the cell wall structure of one of the most prevalent and deadly fungi, which could usher in a new era of antifungal drug development to help save millions of lives.
LSU Department of Chemistry Assistant Professor Tuo Wang and colleagues have identified the high-resolution architecture of the cell wall of one of the most common fungi, Aspergillus fumigatus. Aspergillus fumigatus is airborne and can be found indoors and outdoors. In people with compromised immune systems, the fungi multiplies at an extraordinary rate. It affects more than 200,000 people annually including a quarter of all leukemia patients, and kills more than half of these patients.

"This is the first time anyone has looked at the whole cell of this fungi in its native state at such high resolution. Our work provides the molecular basis to engineer more effective antifungal drugs," Wang said.

Read more: http://www.worldpharmanews.com/research/4492-chemists-characterize-the-fatal-fungus-among-us

Sangamo has agreed a €72m deal to take control of French biotech TxCell, eyeing a regulatory T cell (Treg) platform that it says would accelerate its plans to enter the cell therapy market.
Sangamo’s zinc finger nuclease (ZFN) gene-editing technology is already being used to create chimeric antigen receptor T cell (CAR-T) cancer therapies under a $3b-plus deal with Gilead’s Kite unit, signed earlier this year, and it also has an early-stage programmes in stem cell therapies for blood diseases and off-the-shelf (allogeneic) CAR-Ts for cancer.
By acquiring TxCell, Sangamo thinks it will be able to start trials of cell-based immunotherapies as early as next year, headed by TxCell’s TX200 candidate to prevent graft rejection in solid organ transplant patients and with follow-up projects in other autoimmune diseases and inflammatory such as Crohn’s disease and multiple sclerosis. It plans to a start a phase I/II trial in Europe before the end of 2019.

Read more: http://www.pmlive.com/pharma_news/sangamo_bulks_up_in_cell_therapy_with_txcell_acquisition_1245315

GlaxoSmithKline is reportedly considering whether to split its business into a consumer health spin-off and standalone pharmaceutical and vaccines group.

According to a report in the Financial Times, which cited people familiar with the situation, chairman Philip Hampton has had discussions with major investors over breaking up the business.

“I have had these conversations [with Sir Philip]. The logic of [splitting] the business is pretty clear . . . The financial dynamics of consumer and pharma are pretty different,” one top-10 shareholder told the paper.

Another reportedly told the FT: “It has been a question on the table for a long time. We have been asking ‘what are you? Are you pharma? Are you consumer goods’? Emma came in with a fresh pair of eyes - there is potential for thinking that [a spin-off] would make sense . . . We have spoken to the board about it.”

Read more:http://www.pharmatimes.com/news/gsk_considers_splitting_up_1245326

Roche (SIX: RO, ROG; OTCQX: RHHBY) announced that the U.S. Food and Drug Administration (FDA) has granted Breakthrough Therapy Designation for Tecentriq® (atezolizumab) in combination with Avastin® (bevacizumab) as an initial (first-line) treatment for people with advanced or metastatic hepatocellular carcinoma (HCC), the most common form of liver cancer. The designation is based on data from a Phase Ib study assessing the safety and clinical activity of the combination of Tecentriq and Avastin.
"Hepatocellular carcinoma is an aggressive cancer with limited treatment options and a major cause of cancer deaths worldwide," said Sandra Horning, MD, Roche's Chief Medical Officer and Head of Global Product Development. "Preliminary data from the combination of Tecentriq and Avastin in this disease are promising and we look forward to working with health authorities to make this potential treatment regimen available to people with hepatocellular carcinoma as soon as possible."

Breakthrough Therapy Designation is designed to expedite the development and review of medicines intended to treat serious or life-threatening diseases and to help ensure people have access to them through FDA approval as soon as possible. This is the 22nd Breakthrough Therapy Designation for Roche’s portfolio of medicines and the 3rd for Tecentriq.

Read more: http://www.worldpharmanews.com/roche/4491-fda-grants-breakthrough-therapy-designation-for-roche-s-tecentriq-in-combination-with-avastin-as-first-line-treatment-for-advanced-or-metastatic-hepatocellular-carcinoma-hcc

US regulators have approved the first targeted therapy for patients with relapsed or refractory acute myeloid leukemia carrying a certain genetic mutation.

Agios Pharmaceuticals’ Tibsovo (ivosidenib) is the first drug in the isocitrate dehydrogenase-1 (IDH1) inhibitor class of medicines to win a green light, and has been approved with a companion diagnostic able to detect specific mutations in the IDH1 gene in patients with the disease.

“Tibsovo is a targeted therapy that fills an unmet need for patients with relapsed or refractory AML who have an IDH1 mutation,” said Richard Pazdur, director of the FDA’s Oncology Center of Excellence and acting director of the Office of Hematology and Oncology Products in the FDA’s Center for Drug Evaluation and Research.

“The use of Tibsovo is associated with a complete remission in some patients and a reduction in the need for both red cell and platelet transfusions.”

Read more: http://www.pharmatimes.com/news/us_approves_first-in-class_targeted_aml_therapy_1245302

Without T cells, we could not survive. They are a key component of our immune system and have highly sensitive receptors on their surface that can detect pathogens. The exact way that these receptors are distributed over the surface of the T cells is still not completely understood, but the analyses by TU Wien show that previous ideas are no longer tenable.
It was previously thought that the T cell would concentrate the receptors at certain points in order to achieve the highest possible sensitivity. As a current publication by the biophysics research group at TU Wien shows, T cells are actually programmed to react as quickly as possible, and therefore their receptors are arranged at random. These results were made possible by close collaboration between the Medical University of Vienna (MUW) and the Max Planck Institute (MPI) of Biophysics in Göttingen. These new findings not only help to better understand the immune response, but are also key in developing new methods of medical treatment. These findings have now been published in the specialist journal Nature Immunology.

Read more: http://www.worldpharmanews.com/research/4484-the-immune-system-t-cells-are-built-for-speed

Pfizer Inc. (NYSE:PFE) and Spark Therapeutics (NASDAQ:ONCE) announced that Pfizer initiated a Phase 3 open-label, multi-center, lead-in study (NCT03587116) to evaluate the efficacy and safety of current factor IX prophylaxis replacement therapy in the usual care setting. The factor IX prophylaxis efficacy data obtained in the lead-in study will serve as the within-subject control group for those patients that enroll into the next part of the Phase 3 study, which will evaluate the investigational gene therapy fidanacogene elaparvovec for the treatment of hemophilia B. The interventional portion of this pivotal Phase 3 study will enroll patients who have completed at least six months in the lead-in study. Fidanacogene elaparvovec is the official United States Adopted Name (USAN) and will become the Recommended International Nonproprietary Name (INN) for the therapy formerly known as SPK-9001 and PF-06838435.

Read more: http://www.worldpharmanews.com/pfizer/4485-pfizer-initiates-pivotal-phase-3-program-for-investigational-hemophilia-b-gene-therapy

Janssen’s Symtuza has been approved US regulators as the first and only complete, darunavir-based single-tablet regimen for the treatment of HIV in adults.

According to Janssen, single table regimens have the capacity to boost adherence and reduce the medicine burden

Symtuza darunavir (cobicistat/emtricitabine/tenofovir alafenamide) offers patients “the proven protective barrier to resistance of darunavir in a formulation designed for improved tolerability and the convenience,” it said.

Read more: http://www.pharmatimes.com/news/us_approves_janssens_hiv_pill_symtuza_1244683

GlaxoSmithKline’s new game plan for R&D under new R&D chief Hal Barron continues to take shape, and in the latest move the firm has sold off its lead immuno-inflammation disease asset.
Psoriasis and atopic dermatitis (eczema) candidate tapinarof is being transferred to Roivant Sciences group company Dermavant in return for £150m upfront and another £100m in the offing if it meets development targets. The deal covers all markets except for China.
Tapinarof, a therapeutic aryl hydrocarbon receptor modulating agent (TAMA), is being developed as a once-daily, topical treatment for the skin disorders that could provide an alternative to topical and oral steroids, which have side effects. In phase II trials the drug had a dose-dependent impact on plaque psoriasis lesions, and it is ready to move to phase III testing.

Read more:

http://www.pmlive.com/pharma_news/gsk_sells_eczema_and_psoriasis_drug_to_dermavant_for_250m_1244181

Pfizer Inc. (NYSE:PFE) announced it will organize the company into three businesses: a science-based Innovative Medicines business which will now include biosimilars and a new hospital business unit for anti-infectives and sterile injectables; an off-patent branded and generic Established Medicines business operating with substantial autonomy within Pfizer and a Consumer Healthcare business. These changes will be effective at the beginning of the company's 2019 fiscal year.


"This new structure represents a natural evolution of these businesses given the ongoing strength of our in-market products and our late-stage pipeline and the expected significant reduction in the impact of patent protection losses post-2020 following the loss of exclusivity for Lyrica in the U.S which is expected to occur in or after December 2018. As we transition to a period post-2020 where we expect a higher and more sustained revenue growth profile we see this new structure better positioning each business to achieve its growth potential," said Ian Read, Pfizer Chairman and Chief Executive Officer.


Read more: http://www.worldpharmanews.com/pfizer/4479-pfizer-to-organize-for-future-growth