The Snippet: The Success of Cannabidiol

GW Pharmaceuticals, a UK based company, believes in growing its own cannabis plants, search for its ingredients and then come up with medicines that help the company in taking one more step towards approval of Epidiolex, as seen in the announcement of positive phase 3 trial results earlier this week.

The drug is for the treatment of epilepsy including Dravet syndrome and lennox-Gastaut syndrome. Pateints who were on 20mg dosage of Epidiolex had 42% reduction in monthly seizures compared to 17% of control patients. The company is expecting to submit a new drug application with US FDA in the first half of 2017. On Monday, a sudden rise of more than 16% in the stocks of the company was noted.

The drug so far has got Orphan Drug Designation from FDA for the treatment of Dravet syndrome and lennox-Gastaut syndrome and Fast track designation for Dravet syndrome. The company has, thus, set a new standard in the market by announcing promising results of Epidiolex.

To know more, please click on the link below:

http://www.gwpharm.com/PR260916.aspx

Business Cocktail

Bayer AG and Evotec AG enter a multi-target research partnership agreement

Bayer and Evotec have agreed to develop novel treatments for chronic kidney disease in diabetic patients. According to the agreement, Bayer will have all the rights to select clinical candidates and Evotec’s Cure Nephron target pipeline. It will pay approximately $15.64m to Evotec during the contract period.

Plasticell and CellSpring AG have entered into an agreement

Plasticell and CellSpring have entered an agreement which aims to examine and validate the capability of osteogenic cell therapy and 3D cell cultures in drug screening. According to this agreement, the companies will be responsible for developing 3D tissue models that accurately respond to the drug treatment. These 3D models are used in pharmaceutical industry in all stages of drug discovery, to evaluate the efficacy of lead compounds.

Allergan Acquires Akarna Therapeutics

Allergan plans to develop novel treatments for non-alcoholic steatohepatitis (NASH) and other liver diseases through the acquisition of Akarna Therapeutics. The acquisition has been completed for a purchase consideration of $50m. Allergan will gain all rights to AKN-083 and a portfolio of additional development stage FXR compounds. Akarna Therapeutics will receive additional clinical, regulatory, developmental and commercialisation milestones of its drug candidate.

Cambrex Acquires Pharmacore

Cambrex Corporation, a manufacturer of small molecule innovator and generic active pharmaceutical ingredients (APIs), has acquired PharmaCore, which specializes in developing, manufacturing and scaling up small molecule APIs for clinical phase projects, for approximately $25 million. The company is licensed with the US Drug Enforcement Administration (DEA) to manufacture Schedule II to Schedule V controlled substances.

 

The Snippet : Merck Focuses its Research on Hard to Treat Cancers

Merck is a leading science and technology company, and has many projects that are involved in research to find treatments for diseases that are hard to treat. Keeping in view with this agenda, Merck has announced that the new research that has been conducted from their marketed and pipeline compounds will be presented at this year’s European Society for Medical Oncology annual meeting in Denmark. The presentations are to be focussed on hard-to-treat cancers, and will include results for Erbitux® (cetuximab) in metastatic colorectal cancer (mCRC) and squamous cell carcinoma of the head and neck (SCCHN); preliminary study results in bladder cancer and renal cell carcinoma (RCC) for avelumab, which is being developed in collaboration with Pfizer; and updates on the Phase II program for tepotinib* in non-small cell lung cancer (NSCLC).

To know more, click on the link below:

http://www.bioportfolio.com/news/article/2854515/Merck-to-Present-New-Research-Focused-on-Hard-to-Treat-Cancers-at.html

 

Notizia

US FDA grants approval for Novartis’ Ilaris to treat Periodic Fever Syndromes

Novartis received approval to market its Periodic Fever Syndrome drug Ilaris (cancakinumab). The drug targets the syndrome, and will be used to treat a group of diseases that cause serious recurrent fever and pathogenic inflammation through non-infectious activation of the immune system. Ilaris inhibits Interleukin-1 (IL-1) beta, and is already approved in the US to treat Cryopyrin-Associated Periodic Syndromes (CAPS) and Systemic Juvenile Idiopathic Arthritis (SJIA).

Sanaria receives FDA fast-track designation for malaria vaccine

PfSPZ, the malaria vaccine of Sanaria, has received fast-track designation from the US Food and Drug Administration (FDA).PfSPZ is currently in clinical trials that are intended to finalise an immunisation regimen to be taken forward into pivotal Phase III clinical trials. If successful, the clinical trials provide the necessary data for licensing the vaccine, according to the company.

Sanofi and Regeneron’s next wannabe blockbuster tagged ‘priority’ by Regulators

FDA has accepted the application for atopic dermatitis candidate dupilumab, from Sanofi and Regeneron, and have given it a priority review tag that should hasten its trip to approval. Dupilumab has already shown it can beat out those topical steroids, too. In June, its makers rolled out Phase III data showing that a dupilumab/corticosteroid combo topped steroids alone. analysts see dupilumab generating more than $2.5 billion a year–with some predicting up to $4 billion or $5 billion.

Allele receives NIH grant to develop new antibody therapy for Alzheimer’s disease

Allele Biotechnology and Pharmaceuticals (Allele) has secured a grant from the US National Institutes of Health’s (NIH) National Institute on Aging to develop a new antibody therapy for Alzheimer’s disease. Allele will develop a panel of antibodies that identify this protein, one of which would be used as a therapeutic drug candidate. The antibodies will feature a distinct shape and size, allowing them to pass the blood-brain barrier to reach the critical areas of the brain. Allele researchers also aim to modify and engineer each antibody to enhance its therapeutic potential.

Cervical Cancer: Current Scenario

Cervical cancer is the second most common cause of death in women globally. Increased awareness and early diagnosis has reduced the death toll over the years; but still there is considerable risk that exists. According to the estimated of American Cancer Society, approximately 12,990 new cases of invasive cervical cancer will be diagnosed and about 4,120 women might die from cervical cancer in United States in 2016. There are many risk factors associated with the cause of cervical cancer and the most common is the persistent HPV (Human papillomavirus) infection which accounts 90% of the total cases.

Research showed that psychosocial stress is one of the crucial contributors of the prolonged infection of HPV. Women who smoke, take drugs etc. are more prone to HPV infection which further increases the chances of developing cervical cancer. Women suffering from Systemic Lupus Erythematosus (SLE) and Inflammation Bowel Syndrome (IBD) like Crohn’s disease should take extra care as they can be more likely to develop cervical cancer. Age is also an important factor. Trends indicate that occurrence of cervical cancer rises between late teens and mid 30s. Women with lowered immunity are at high risk of developing this cancer. Other factors such as multiple pregnancies, oral contraceptives, more than one sex partners etc. are more likely to contribute to the occurrence of cervical cancer.

Since cervical cancer has a large scope for treatment if detected in early stages, researchers are focusing on developing better ways of detecting precancer and cervical cancer. Prevention has always been better than cure, and in case of cancer early diagnosis increases the survival rate. Regular screening (Pap test and HPV Test) and vaccination is suggested for prevention. Cervical Cancer treatment is based on use of surgery, Chemotherapy, Radiation therapy and targeted therapy.

Several chemotherapeutics such as Cisplatin (Platinol; Bristol-Myers Squibb Company), Carboplatin (Paraplatin; Bristol-Myers Squibb Company), Paclitaxel (Taxol; Bristol-Myers Squibb), Topotecan (Hycamtin; GlaxoSmithKline), Gemcitabine (Gemzar; Eli Lilly and Company), Bevacizumab (Avastin; Genentech) are available for the treatment of advanced stages of cervical cancer. Researchers and pharmaceutical companies are focusing on targeted therapies, drugs for treating early detection, palliative care and use of biomarkers for accurate detection of cervical cancer.

Insight by
Jyoti Kumari
Associate Analyst 
DelveInsight Business Research, LLP

The Snippet : FDA Approves Sarepta’s Muscular Dystrophy Drug after Months of Debate

The Food and Drug Administration approved the recent controversial drug to treat Duchenne muscular Dystrophy, which is a rare disease that confines boys to wheelchairs and condemns them to an early death. The decision was made after months of debate between the Agency and Sarepta Therapeutics, regarding the evidence needed to demonstrate that its medication, called eteplirsen, had a meaningful impact on patients. Sarepta said the drug would be priced at about $300,000 a year in the US. As a condition of the approval, Sarepta will have to conduct a two-year, randomized controlled trial to verify the clinical benefit of the drug. The purpose is to determine whether the drug actually improves motor functions. If the trial fails, the FDA could move to withdraw approval.

To read more, Click on the link below:

https://www.statnews.com/pharmalot/2016/09/19/sarepta-wins-dmd-drug-approval/

Business Cocktail

Collaboration between Sygnis AG and ECACC for licensing and distribution of Caco-2 cell line

Sygnis AG enters into a collaboration agreement with ECACC to significantly expand Sygnis’ Caco-2 business. Under the collaboration, ECACC will organize that companies obtain the license for the use of the Caco-2 cell line from Sygnis in the US and afterwards will provide the Caco-2 materials directly.

Partnership between BioNTech and Genentech to develop individualized mRNA cancer therapies

BioNTech has agreed to collaborate with Roche’s Genentech to develop and commercialize mRNA based, individualized cancer vaccines. The agreement will see the companies jointly develop individually tailored mRNA cancer vaccines applicable against a wide range of cancers, in a bid to potentially provide a new treatment paradigm for cancer patients.

Partnership between Zydus Cadila and Takeda to develop vaccine for Chikungunya virus

Zydus Cadila and Takeda Pharmaceutical have agreed to partner to develop a vaccine for an emerging infectious disease known as Chikungunya. The agreement will see both parties collaborating from an early stage development of the vaccine up to its final commercialization.

Global Agreement between Regeneron Pharmaceuticals, Inc. and Teva Pharmaceutical to develop Regeneron’s Fasinumab

Regeneron and Teva entered a global agreement for the development and commercialization of the former’s NGF antibody, fasinumab. According to the developmental deal, Regeneron will receive payment of $250 million along with development and regulatory milestone payments, plus additional payments based on net sales. Regeneron will be responsible for the global development and commercialization of fasinumab in the U.S., and Teva will be responsible for the candidate’s development and commercialization outside the U.S.

Big Pharma companies including Cipla & Wockhardt in global alliance to fight drug resistance

13 global pharmaceutical companies have joined hands in laying out a roadmap to combat antimicrobial resistance (AMR) by 2020. The roadmap by these 13 drug firms follows the Industry Declaration signed in January 2016 at the World Economic Forum by more than 100 companies and trade associations, the companies said in a joint statement. The other companies in the alliance include global majors such as Johnson & Johnson, Novartis, Pfizer, Sanofi and AstraZeneca.

Notizia

GSK appoints its first female CEO

Emma Walmsley has been appointed as GlaxoSmithKline‘s new CEO, and has broken the mould as she is the first woman to lead a top global drug maker, being a consumer brands guru rather than a prescription medicines expert. Walmsley’s appointment suggests GSK will continue the diversified strategy pioneered by outgoing CEO Andrew Witty. “Obviously, R&D is the beating heart of our company and our success is and will continue to be defined most fundamentally by the strength of our pipeline,” she said in an in-house video recorded to mark her appointment.

Johnson & Johnson to Buy Abbott’s Vision Unit for $4.33B

Johnson & Johnson announced that it will acquire Abbott Medical Optics for $4.325 billion. The deal is expected to close in the first quarter of 2017. The deal comes as JNJ plans to grow its vision business by entering into the cataract surgery market, apart from its Acuvue contact lenses. Abbott has decided to sell this part of business so that it can focus more on treatment for heart and arteries and disease testing equipment.

Essai Launches Collaborative R&D Unit in Boston Area

Eisai Inc., the U.S. subsidiary of Japan’s Eisai Co., Ltd.announced  that it is launching a research-and-discovery unit in the Boston area. The new unit will be called the Eisai Andover Innovative Medicines (AiM) Institute. It will target three therapeutic areas, immuno-dementia, immuno-oncology, and autoimmune indications. Within immuno-dementia, AiM will focus on a subset of dementia patients whose disease is driven by immune dysfunction. In immuno-oncology, it will focus on discovering precision immunotherapies that target myeloid lineage cells. In autoimmune studies, it will look at toll-like receptors and prostaglandins to develop therapies for diseases like systemic lupus erythematosus (SLE).

Novartis intensifies marketing plan for Entresto

Novartis on Monday intensified its bid to convince doctors to prescribe its heart failure drug Entresto, releasing an analysis that concluded the medicine contributed to higher quality of life scores compared with an older drug. Launched with much fanfare last year, Entresto sales have so far disappointed investors and the company, forcing Novartis to spend hundreds of millions more on marketing as it seeks to hit a modest target of $200 million in revenue from the drug this year.

U.S. FDA approves Bayer contraceptive device Kyleena

The U.S. Food and Drug Administration approved Bayer AG‘s hormonal contraceptive device, Kyleena, to prevent pregnancy for up to five years. Kyleena, which will be available from October, is a long-acting reversible contraceptive (LARC), a category of potent contraceptives that has returned to popularity. LARCs, including IUDs and implants, are more effective than other contraceptives such as pills and patches, and are nearly as effective as sterilization, according to the U.S. Centers for Disease Control and Prevention.

 

The Snippet : PI3Kγ is a molecular switch that controls immune suppression

Macrophages play critical role in acute and chronic inflammation and cancer. In response to pathogens or injury, inflammatory macrophages express cytokines that stimulate cytotoxic T cells. Macrophage PI(3)Kinase γ controls a critical switch between immune stimulation and suppression during inflammation and cancer. PI3KC signaling through Akt and mTor inhibits NFκB activation while stimulating C/EBPβ activation. This induces a transcriptional program that promotes immune suppression during inflammation and tumor growth. However, selective inactivation of macrophage PI3Kγ stimulates and prolongs NFκB activation and inhibits C/EBPβ activation, thus promoting an immunostimulatory transcriptional program that restores CD8+ T cell activation and cytotoxicity and synergizes with checkpoint inhibitor therapy to promote tumor regression and extend survival. The therapeutic targeting of intracellular signaling pathways that regulate the switch between macrophage polarization states can control immune suppression in cancer and other disorders.

To know more about PI3Kγ, click on the link below:

http://www.nature.com/nature/journal/vaap/ncurrent/full/nature19834.html

Graft vs. Host Disease: Gruesome complications of allogeneic bone marrow transplant- Treatment is on the way

It is a well-known fact that where there is stem cell transplantation, there is Graft vs Host Disease (GVHD). The concept arose 2 decades ago and is not rare- it is a common complication of allogeneic hematopoietic stem cell transplantation (HSCT) that occurs when the donated (graft) cells are rejected, and attack the host’s cells as foreign. Chronic GVHD is a major area of concern as it can last for years or a lifetime, leading to debilitated and life-threatening condition.

Approximately 40 percent of patients develop chronic GVHD after 100 days of transplant. The number of cases of GVHD depends on the number of allogeneic cell transplants (HCTs) and the transplantation occurs more than 20,000 times annually. So, the utmost concern is how GVHD should be treated.

The most effective approach includes Monoclonal antibodies. The use of Monoclonal antibodies in Graft Versus Host Disease has been increasing from last 20 years. Several drugs such as Alemtuzumab, Infliximab and Rituximab are already approved for the treatment of GVHD. Pipeline of GVHD contains Neihulizumab, Begelomab, Milatuzumab and MGD010 that targets activated T Cells.

Cell therapy is also making its way for the treatment as Enlivex Therapeutics and Kiadis Pharma are actively involved in developing their own technologies. Besides that, more than 52 companies are in the development race for GVHD treatment. Novartis Pharmaceuticals is currently leading the race with 3 products in pipeline. Incyte Corporation and ImmuNext are the next leaders in the upcoming GVHD market with two products respectively. Endonovo Therapeutics is developing a next-generation; off-the-shelf treatment for Graft-Versus-Host Disease (GVHD) using Cytotronics expanded and ex vivo enhanced stem cells from the human umbilical cord. Dr. Falk Pharma GmbH, Mallinckrodt and Adienne Pharma & Biotech are expected to launch their products in coming years as the products are in Phase III stage of development.

GVHD is treated by suppressing the immune system, which means the treatment also increases the risk of infection. Each year, thousands of patients with hematologic malignancies undergo allogeneic (donor) stem cell transplantation (SCT). Thus, treatment of GVHD by enhancing immune system is very important. Pharmaceutical companies, clinicians and researchers are continually working to reduce the rate of GVHD occurrence and improve patient outcomes.

Insight by Shakshi Sikrewal
Associate Analyst
DelveInsight Business Research, LLP