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U.S. Emergency Labs Ready To Work On Ebola Drugs If Asked

U.S. Emergency Labs Ready To Work On Ebola Drugs If Asked | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
(Repeating story first sent on Aug 9 to additional subscribers) By Sharon Begley and Toni Clarke NEW YORK/WASHINGTON, Aug 9 (Reuters) - All three U.S. facilities established to quickly make vaccines and therapeutics in...
Krishan Maggon 's insight:

The US emergency labs and production facilities are ready to scale up producing Ebola drugs, mabs and vaccines if funded.

 

The facilities are mainly for US use but can supply drugs to Allies and WHO.

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Pharma Biotech Industry Review (Krishan Maggon)
Review and updates of latest developments in the pharma biotech industry dealing with new drugs, market research and global health. It will mainly cover FDA and EMA approvals of new drugs, regulatory filings ( NDA, BLA, MAA ), sales and marketing data of to selling drugs, blockbuster drugs,  best selling drugs and R&D budget and R&D productivity/efficiency. Sales and marketing forecast and cost of newly approved drugs will be discussed.  <a href="<a href="http://knol.google.com/k/krishan-maggon-knols#"" rel="nofollow">http://knol.google.com/k/krishan-maggon-knols#"</a>; rel="nofollow"><a href="http://knol.google.com/k/krishan-maggon-knols#" rel="nofollow">http://knol.google.com/k/krishan-maggon-knols#</a></a>;
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FDA New Drug Approvals 2014

FDA New Drug Approvals 2014 | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

The FDA has approved 42 new  drugs (NCE/NME) so far in 2014 and may approve additional drugs in the last week. The approval of new drugs has picked up in December with 9 new drugs approved and 6 in one week, a record for the FDA.

 

The CHMP of EMA has recommended approval of 82  new medicines in 2014, out of which 57 were approved by the EC for marketing in the EU. 

 

 

to be updated ASAP.

 

 

Krishan Maggon 's insight:

For a free updated ongoing review of new drugs (NCE/NME) approved by the FDA in 2014 with a list of new NDA/BLA filings, PDUFA dates. 

 

FDA first approved drug Farxiga (dapaglifozin, Astra Zeneca, BMS) to treat type 2 diabetes is reviewed with links, references,

clinical data and regulatory history and its competition with J&J first approved SGTP2 inhibitor Invokana. 

 

Approvals of generics, combination products or new formulations is not covered.

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BioWorld's top 10: The biggest news makers and trending stories of 2014 - BioWorld Online

BioWorld's top 10: The biggest news makers and trending stories of 2014 - BioWorld Online | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
To say that 2014 was a good year for the biopharma industry would be a radical understatement. The flourishing capital markets, the record-breaking number of companies successfully going public via
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Actelion submits US FDA New Drug Application for selexipag (Uptravi) in patients with pulmonary arterial hypertension

Actelion submits US FDA New Drug Application for selexipag (Uptravi) in patients with pulmonary arterial hypertension | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

ALLSCHWIL, SWITZERLAND - 23 December 2014 - Actelion Ltd (SIX: ATLN) today announced that it has submitted a New Drug Application (NDA) to the US Food and Drug Administration (FDA) seeking approval for selexipag (Uptravi®) for the treatment of patients with pulmonary arterial hypertension (PAH).

 

Selexipag, the first selective oral IP prostacyclin receptor agonist, was studied in the pivotal Phase III GRIPHON study in 1,156 patients with pulmonary arterial hypertension (PAH). As reported in June 2014, the study demonstrated that selexipag decreased the risk of a morbidity/mortality event versus placebo by 39% (p<0.0001). Efficacy observed was consistent across the key subgroups; age, gender, WHO Functional Class, PAH etiology and background PAH therapy. Patients were treated for up to 4.2 years. The overall tolerability profile of selexipag in GRIPHON was consistent with prostacyclin therapies. Adverse reactions occurring more frequently (>5%) on selexipag compared to placebo are headache, diarrhea, jaw pain, nausea, myalgia, vomiting, pain in extremity, and flushing.

 

THE ROLE OF THE PROSTACYCLIN PATHWAY

The prostacyclin pathway is one of the 3 essential pathways involved in the pathophysiology and treatment of PAH. Prostacyclin is a prostanoid and serves as a signaling molecule in the human body. It is produced, like other vasoactive substances, by endothelial cells. Prostacyclin induces vasodilation, is anti-proliferative, has anti-inflammatory effects and inhibits platelet aggregation. In certain disease conditions, the production of prostacyclin by the endothelium is impaired, allowing for example, the deleterious effects of excessive levels of endothelin to predominate.

 

ABOUT SELEXIPAG

 

Selexipag selectively targets the prostacyclin receptor (also called IP-receptor). The IP receptor is one of 5 types of prostanoid receptor. Prostacyclin activates the IP receptor inducing vasodilation and inhibiting proliferation of vascular smooth muscle cells. Selexipag, unlike prostacyclin analogs, is selective for the IP receptor over other prostanoid receptors. In preclinical models selective IP receptor agonism has shown to maintain efficacy and reduce the risk of side effects mediated by activation of other prostanoid receptors, such as EP1 and EP3 receptors. [1,2,3]

Selexipag was previously evaluated in a Phase II, 43-patient, placebo-controlled, double-blind study, where patients were randomized in a 3:1 ratio receiving selexipag or placebo on top of PDE-5 inhibitor and/or ERA [4]

 

Krishan Maggon 's insight:

Selexipag, originally discovered and synthesized by Nippon Shinyaku, is a potent, orally available, selective IP prostacyclin receptor agonist.


NIPPON SHINYAKU

 

http://www.nippon-shinyaku.co.jp/english/index.html



REGULATORY STATUS OF SELEXIPAG

Submission of a centralized Marketing Authorisation Application (MAA) was made to the European Medicines Agency (EMA) for selexipag (Uptravi®) in the treatment of pulmonary arterial hypertension in December 2014. The application is now pending validation. 

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GEN | Insight & Intelligence™:Top Charitable Funds and Foundations

GEN | Insight & Intelligence™:Top Charitable Funds and Foundations | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
These eleven organizations are known for their charitable contributions to life-sci researchers and their institutions.
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Finding the Ebola virus' vulnerable points - Ars Technica

Finding the Ebola virus' vulnerable points - Ars Technica | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
We know what antibodies stop it in its tracks—we now know where they attach.
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Saxenda® (liraglutide,Novo Nordisk) approved by the FDA for obesity under REMS

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FDA approves weight-management drug Saxenda ((liraglutide, Novo Nordisk)

FDA approves weight-management drug Saxenda ((liraglutide, Novo Nordisk) | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
The U.S. Food and Drug Administration today approved Saxenda (liraglutide [rDNA origin] injection) as a treatment option for chronic weight management in addition to a reduced-calorie diet and physical activity.

 

The drug is approved for use in adults with a body mass index (BMI) of 30 or greater (obesity) or adults with a BMI of 27 or greater (overweight) who have at least one weight-related condition such as hypertension, type 2 diabetes, or high cholesterol (dyslipidemia).

 

Saxenda is a glucagon-like peptide-1 (GLP-1) receptor agonist and should not be used in combination with any other drug belonging to this class, including Victoza, a treatment for type 2 diabetes. Saxenda and Victoza contain the same active ingredient (liraglutide) at different doses (3 mg and 1.8 mg, respectively). However, Saxenda is not indicated for the treatment of type 2 diabetes, as the safety and efficacy of Saxenda for the treatment of diabetes has not been established. 

 

The safety and effectiveness of Saxenda were evaluated in three clinical trials that included approximately 4,800 obese and overweight patients with and without significant weight-related conditions. All patients received counseling regarding lifestyle modifications that consisted of a reduced-calorie diet and regular physical activity.

 

Results from a clinical trial that enrolled patients without diabetes showed that patients had an average weight loss of 4.5 percent from baseline compared to treatment with a placebo (inactive pill) at one year. In this trial, 62 percent of patients treated with Saxenda lost at least 5 percent of their body weight compared with 34 percent of patients treated with placebo. Results from another clinical trial that enrolled patients with type 2 diabetes showed that patients had an average weight loss of 3.7 percent from baseline compared to treatment with placebo at one year. In this trial, 49 percent of patients treated with Saxenda lost at least 5 percent of their body weight compared with 16 percent of patients treated with placebo.

Patients using Saxenda should be evaluated after 16 weeks to determine if the treatment is working. If a patient has not lost at least 4 percent of baseline body weight, Saxenda should be discontinued, as it is unlikely that the patient will achieve and sustain clinically meaningful weight loss with continued treatment.

Saxenda has a boxed warning stating that tumors of the thyroid gland (thyroid C-cell tumors) have been observed in rodent studies with Saxenda but that it is unknown whether Saxenda causes thyroid C-cell tumors, including a type of thyroid cancer called medullary thyroid carcinoma (MTC), in humans. Saxenda should not be used in patients with a personal or family history of MTC or in patients with multiple endocrine neoplasia syndrome type 2 (a disease in which patients have tumors in more than one gland in their body, which predisposes them to MTC).

Serious side effects reported in patients treated with Saxenda include pancreatitis, gallbladder disease, renal impairment, and suicidal thoughts. Saxenda can also raise heart rate and should be discontinued in patients who experience a sustained increase in resting heart rate.

In clinical trials, the most common side effects observed in patients treated with Saxenda were nausea, diarrhea, constipation, vomiting, low blood sugar (hypoglycemia), and decreased appetite.

 

 

Krishan Maggon 's insight:

The FDA is requiring the following post-marketing studies for Saxenda:

clinical trials to evaluate dosing, safety, and efficacy in pediatric patients;a study to assess potential effects on growth, sexual maturation, and central nervous system development and function in immature rats;an MTC case registry of at least 15 years duration to identify any increase in MTC incidence related to Saxenda; andan evaluation of the potential risk of breast cancer with Saxenda in ongoing clinical trials.

In addition, the cardiovascular safety of liraglutide is being investigated in an ongoing cardiovascular outcomes trial.

The FDA approved Saxenda with a Risk Evaluation and Mitigation Strategy (REMS), which consists of a communication plan to inform health care professionals about the serious risks associated with Saxenda.

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BioCryst Ebola drug BCX 4430 promising in rhesus macaques trial

BioCryst Ebola drug BCX 4430 promising in rhesus macaques trial | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

BioCryst Pharmaceuticals, Inc.(Nasdaq:BCRX) today announced results from a proof-of-concept study of its broad spectrum antiviral BCX4430 for the treatment of experimental Ebola virus infection in rhesus macaques, conducted at the United States Army Medical Research Institute of Infectious Diseases (USAMRIID).

 

The primary goal of the study was to assess the effect of BCX4430 treatment on survival through Day 41 in animals infected with Ebola virus. Dosing of placebo or BCX4430 by intramuscular (i.m.) injection was initiated 30-120 minutes after virus challenge and continued twice a day (BID) for 14 days.

 

Animals were dosed with either placebo, 16 mg/kg of BCX4430 BID or 25 mg/kg of BCX4430 BID. Survival at day 41 in the 16 mg/kg group of BCX4430 treated animals was 4 of 6 (66.7%, p < 0.001 compared to 0% survival in controls) and 6 of 6 in the 25 mg/kg treated group (100%, p < 0.001 compared to controls). The overall survival rate for BCX4430 treated animals at day 41 was 10 of 12 (83%, p < 0.001 compared to controls). Preliminary evaluation of the quantity of virus in the blood showed an approximate 3-log reduction in Ebola virus RNA copies/mL of plasma, compared with control animals.   

Krishan Maggon 's insight:

BCX4430 is an RNA dependent-RNA polymerase inhibitor that has demonstrated broad-spectrum activity in vitro against more than 20 RNA viruses in nine different families, including filoviruses, togaviruses, bunyaviruses, arenaviruses, paramyxoviruses, coronaviruses and flaviviruses. BioCryst is developing BCX4430 in collaboration with U.S. Government Agencies.

 

The National Institute of Allergy and Infectious Diseases, part of the National Institutes of Health, is funding this project under Contract No. HHSN272201300017C.

 

United States Army Medical Research Institute of Infectious Diseases (USAMRIID).

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Can We Unlock The Body's Ability To Regenerate Lost Hearing?

Can We Unlock The Body's Ability To Regenerate Lost Hearing? | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Researchers at Novartis are developing an experimental gene therapy which may lead to a medical breakthrough in potentially restoring damaged and lost hearing in patients.

 

cochlear implants, microphones mounted behind the ear that transmit sounds to electrodes implanted in the inner ear, bypassing the hair cells to create an electronic approximation of natural hearing.

 

For new patients with similar hearing loss, however, an exciting option is on the horizon. In May 2014, an experimental drug from Novartis that could potentially restore lost hearing entered early-phase clinical trials. Rather than providing an electronic solution, the drug appears to unlock the body’s ability to regenerate delicate hair cells and repair the ear’s natural auditory mechanics. Much research remains to be done, but if the experimental drug, known by its research designation CGF166, proves effective and safe in this trial, it could lead to a major breakthrough for the treatment of hearing loss.

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ContraFect Lysins fast acting antibacterial agents

ContraFect Lysins fast acting antibacterial agents | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Lysins are enzymes that digest the cell wall of bacteria. Once the cell wall is breached, the bacteria lyses in a virtually explosive manner. We believe lysins are unlike standard-of-care antibiotics, especially regarding their mechanism and speed of action. Traditional antibiotics, and most cytotoxic agents, require bacterial cell division and metabolism to occur in order to exert their effect (i.e., cell death or cessation of growth). Based on in vitro tests, lysins, however, are fundamentally different in that they rapid bactericidal activity. Lysins have the ability to kill bacteria immediately upon contact. 

Our lysins target the conserved regions of bacteria. Based on our research and experimentation to date, bacteria have shown minimal resistance to our lysins. It is our intention to maintain this attribute for all of our product candidates.Additional key features of our lysins that distinguish them from many standard-of-care antibiotics include full activity against antibiotic-resistant bacteria (while sparing "good" bacteria), synergy with antibiotics, and the ability to eradicate biofilms. 


Based upon our data, we believe our lysins combined with standard of care antibiotics, have the potential to reduce treatment times, improve patient outcomes and shorten hospital stays, while not contributing to the global crisis of drug-resistance. 

We have an in-house lysin discovery platform that consists of bio-informatic and metagenomic-based functional screening of genomic material recovered directly from the environment for large scale identification of lysins, enabling the production of lysin banks specific for any particular bacterial pathogen. The ability to rapidly identify lysins specific for any bacteria of interest provides a steady pipeline of novel lysins for consideration as potential antimicrobial therapeutic candidates. In addition to our in-house lysin discovery program, we have acquired worldwide exclusive rights to nine lysins from The Rockefeller University. Each lysin targets specific gram-positive bacteria, including drug-sensitive and drug-resistant forms of Staph aureus, pneumococcus, group B streptococcus, enterococcus and anthrax.

Attributes of Lysins

 

Novel Mechanism of Action.Lysins represent a first in class approach to treatment of bacterial infections. Traditional antibiotics, and most cytotoxic agents, require bacterial cell division and metabolism to occur in order to exert their effect (i.e., cell death or cessation of growth). Lysins, however, are fundamentally different in that they kill bacteria immediately upon contact. Lysins are recombinant forms of naturally occurring bacteriophage enzymes thatdirectly digest the cell wall of bacteria. Based on in vitro tests, once the cell wall is breached, the bacteria lyses in a virtually explosive manner due to the high internal osmotic pressure of its cytoplasm, and bacterial cell death ensues. 

 

Rapid bactericidal activity. Lysins kill bacteria immediately upon contact. In vitro experiments demonstrate CF-301 to be 12-18x faster than the standard-of-care agents used to treat Staph aureus Bacteremia. Currently, mortality from Staph bacteremia remains close to 30% with treatment on standard-of-care drugs. We believe ourlysins, combined with standard-of-care antibiotics, have the potential to improve patient outcomes, shorten treatment times and reduce the length of hospital stays.

Narrow spectrum, broad-acting anti-infective activity. Unlike standard-of-care antibiotics, which are "broad spectrum" and kill the body's natural flora (including the good bacteria) in addition to the disease-causing bacteria, we consider lysins to be "narrow spectrum, broad-acting" anti-infective agents. Due to the specificity of a lysin's binding domain and catalytic activity, they are highly specific for given species of bacteria (i.e., Staph aureus, pneumococcus, etc.), making them a narrow spectrum anti-infective.However, lysins possess full activity against all resistant strains of that bacteria and are considered broad-acting. For example, CF-301 exhibits activity specific to all forms of Staph aureus, including isolates that are resistant to the antibiotics methicillin (methicillin-resistant Staph aureus; "MRSA"),vancomycin (vancomycin-resistant Staph aureus; "VISA";"VRSA"), and daptomycin (daptomycin-resistant Staph aureus; "DRSA"). We believe the narrow spectrum of activity will avoid damaging side effects that often occur when conventional antibiotic treatments kill the body's healthy, desirable bacteria.

Synergy with standard-of-care antibiotics. We have discovered a strong synergistic effect between CF-301 and several standard-of-care antibiotics, including daptomycin, vancomycin and oxacillin. Synergy is defined as the interaction of two or more agents so that their combined effect is greater than the sum of their individual effects. We intend to seek approval for CF-301 in combination with these standard-of-care antibiotics for Staph aureus bacteremia. We believe that the use of CF-301 in combination with, rather than as a replacement for, standard-of-care antibiotics, may help speed adoption of our product by physicians.

Eradicates biofilms. Biofilms render infections up to 1,000-fold more resistant to penetration by antibiotics. Infected human tissues, such as the heart valve in endocarditis or bone in osteomyelitis, or indwelling medical devices, such as central venous catheters, prosthetic joints and pacemakers, are common sites for biofilm formation, providing a hurdle for effective treatment with antibiotics alone. There is currently nothing available to eradicate biofilms. CF-301 eradicates biofilms that protect bacterial infections in the body (heart valve) and on indwelling devices (prosthetics) from antibiotics.

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Accelerating Medicines Partnership | Foundation for the National Institutes of Health

Accelerating Medicines Partnership | Foundation for the National Institutes of Health | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

This unprecedented public private partnership brings together the National Institutes of Health, (NIH), 10 biopharmaceutical companies and several not-for-profit organizations, in a mission to transform the current model for developing new diagnostics and treatments by jointly identifying and validating promising biological targets of disease.  This is the first pre-competitive public private partnership of its kind, since a main goal of the collaboration will be to generate pre-competitive, disease-specific data that will be made publicly available.   

 

AMP currently has projects in three disease areas — Alzheimer’s Disease,Type 2 Diabetes and Rheumatoid Arthritis and Systemic Lupus Erythematosus (Lupus) — each project will last from three to five years in duration.

Krishan Maggon 's insight:

The AMP Partners include the Food and Drug Administration (FDA) and the NIH from the government sector; AbbVie, Biogen Idec, Bristol-Myers Squibb, GlaxoSmithKline, Janssen Research & Development, Eli Lilly and Company, Merck & Co., Inc., Pfizer, Inc., Sanofi, and Takeda Pharmaceuticals from the industry sector.

 

Additional support is provided by the non-profit sector partners Alliance for Lupus Research, The Alzheimer’s Association, the American Diabetes Association, Arthritis Foundation, The Foundation for the NIH, Geoffrey Bean Foundation, Juvenile Diabetes Research Foundation, Lupus Foundation of America, The Lupus Research Institute, PhRMA, Rheumatology Research Foundation, and US Against Alzheimer’s.

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CytoSorb® - A First-in-Class Cytokine Filter Approved in the European Union. Cytosorbents

CytoSorb® - A First-in-Class Cytokine Filter Approved in the European Union. Cytosorbents | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

CytoSorb® is a potentially breakthrough critical care immunotherapy that attempts to address one of the most challenging unmet medical needs in medicine…the ability to prevent or mitigate multiple organ failure - the leading cause of death in the intensive care unit (ICU) from any cause. 

In response to life-threatening conditions such as sepsis and infection, trauma, serious burn injuries, severe lung injury, and pancreatitis, the body frequently overreacts and produces a massive excess of cytokines, or “cytokine storm”. Cytokines normally help the body cope with injury, but at these levels, cytokine storm is toxic to the body, driving severe inflammation and a cascade of pathophysiologic changes in the body that cause cell damage, organ failure and often death. It follows that reduction of cytokine storm may limit this cascade of events, thereby reducing the severity of illness, and helping patients recover and survive. Until recently, however, there were no effective ways to reduce cytokine storm broadly. 

Krishan Maggon 's insight:

CytoSorb® is a first-in-class extracorporeal cytokine adsorber, now approved in the European Union, and broadly indicated for use in any clinical situation where cytokines are elevated. It is compatible with standard hemodialysis machines and blood pumps found in most hospitals. Blood is pumped out of the body, through the CytoSorb® cartridge that contains the company’s proprietary blood compatible porous polymer beads, and the “purified” blood is recirculated back to the patient. In a six hour period, a patient's entire blood volume can be treated approximately 20 times. 

CytoSorb® is clinically proven to reduce cytokines in the company's multi-center, randomized, controlled European Sepsis Trial conducted in Germany. Treatment was safe and well-tolerated in more than 300 human treatments in very sick patients with the worst forms of sepsis and lung injury, and treatment has been safe in nearly 4,000 human treatments overall. Early data suggests that CytoSorb® can reduce organ injury and improve survival in patients at high risk of cytokine injury, particularly those patients with very high cytokine levels, and patients older than age 65. 


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FDA approves RAPIVAB(TM) (peramivir injection, BioCryst ) a new antiviral flu shot for Acute Influenza.

FDA approves RAPIVAB(TM) (peramivir injection, BioCryst ) a new antiviral flu shot for Acute Influenza. | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

RESEARCH TRIANGLE PARK, N.C., Dec. 22, 2014 (GLOBE NEWSWIRE) -- BioCryst Pharmaceuticals, Inc., (Nasdaq:BCRX) a pharmaceutical company focused on the development and commercialization of treatments for rare and infectious diseases, today announced that the U.S. Food and Drug Administration (FDA) has approved RAPIVAB (peramivir injection), an intravenous (i.v.) neuraminidase inhibitor for the treatment of acute uncomplicated influenza in patients 18 years and older who have been symptomatic for no more than two days.

 

About RAPIVAB (peramivir injection)

RAPIVAB's approval was supported by data from over 2,700 subjects treated with peramivir in 27 clinical trials. InJanuary 2010, Shionogi & Co., Ltd. launched intravenous peramivir in Japan under the name RAPIACTA® and in August 2010, Green Cross Corporation announced that it had received marketing and manufacturing authorization for i.v. peramivir in Korea under the name PeramiFlu®. It is estimated that more than one million patients have received peramivir treatment to date. The recommended dose of RAPIVAB, in most adult patients 18 years of age or older with acute uncomplicated influenza, is a single 600 mg dose administered via intravenous infusion for 15 to 30 minutes. RAPIVAB was developed under contract number HHSO10020070032C from the Biomedical Advanced Research and Development Authority (BARDA/HHS), a $234.8 million contract.

 

Krishan Maggon 's insight:

Peramivir is already approved and marketed in Japan.

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Modern Drug Development

Modern Drug Development | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Opinion from JAMA — Modern Drug Development — Which Patients Should Come First?
Krishan Maggon 's insight:
Modern Drug DevelopmentWhich Patients Should Come First?Muthiah Vaduganathan, MD, MPH1; Vinay Prasad, MD, MPH2[+] Author AffiliationsJAMA. 2014;312(24):2619-2620. doi:10.1001/jama.2014.15221.
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West African Ebola Epidemic after One Year — Slowing but Not Yet under Control — NEJM

West African Ebola Epidemic after One Year — Slowing but Not Yet under Control — NEJM | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Correspondence from The New England Journal of Medicine — West African Ebola Epidemic after One Year — Slowing but Not Yet under Control
Krishan Maggon 's insight:
West African Ebola Epidemic after One Year — Slowing but Not Yet under Control

December 24, 2014DOI: 10.1056/NEJMc1414992

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Notable advances 2014 : Nature Medicine : Nature Publishing Group

Notable advances 2014 : Nature Medicine : Nature Publishing Group | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
This year's most notable research included studies that opened new avenues for regenerative medicine, paved the way to editing out vulnerability to disease and unraveled the genetic complexities underlying diseases such as leukemia and schizophrenia. Here are some of the papers that captured our attention and moved their fields forward in 2014.
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Drugs that made headlines in 2014 : Nature Medicine : Nature Publishing Group

Drugs that made headlines in 2014 : Nature Medicine : Nature Publishing Group | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
This year's newsworthy drugs made major strides against infectious diseases, cancer and more. Some others received attention for controversies they stemmed or stomped. Here is a look at the therapies that leapt forward, some that are in limbo, and others that fell by the wayside.
Krishan Maggon 's insight:

NATURE MEDICINE | NEWS

 Drugs that made headlines in 2014Amanda KeenerNature Medicine 20, 1370–1371 (2014) doi:10.1038/nm1214-1370Published online 04 December 2014
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European regulatory experience with drugs for central nervous system disorders

European regulatory experience with drugs for central nervous system disorders | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

Although great advances have been made in the understanding of the pathophysiology of central nervous system (CNS) disorders during the past two decades, this has not been translated into a wider choice of pharmacological treatments. The pharmaceutical industry has substantially reduced its presence in the field.



Krishan Maggon 's insight:

NATURE REVIEWS DRUG DISCOVERY | NEWS AND ANALYSIS | FROM THE ANALYST'S COUCH

European regulatory experience with drugs for central nervous system disordersFlorence Butlen-Ducuing,Malgorzata Zienowicz,Frank Pétavy,Manuel Haas,Tomas Salmonson,Hans-Georg Eichler& Guido RasiAffiliationsCorresponding authorNature Reviews Drug Discovery (2014) doi:10.1038/nrd4511Published online 19 December 2014
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A decade of innovation in pharmaceutical R&D: the Chorus model

A decade of innovation in pharmaceutical R&D: the Chorus model | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it

Chorus is a small, operationally independent clinical development organization within Eli Lilly and Company that specializes in drug development from candidate selection to clinical proof of concept. The mission of Chorus is to achieve proof of concept rapidly and at a low cost while positioning successful projects for 'pharma-quality' late-stage development. Chorus uses a small internal staff of experienced drug developers and a network of external vendors to design and implement chemistry, manufacturing and control processes, preclinical toxicology and biology, and Phase I/II clinical trials. In the decade since it was established, Chorus has demonstrated substantial productivity improvements in both time and cost compared to traditional pharmaceutical research and development. Here, we describe its development philosophy, organizational structure, operational model and results to date.

Krishan Maggon 's insight:

NATURE REVIEWS DRUG DISCOVERY | PERSPECTIVES | OUTLOOK

 A decade of innovation in pharmaceutical R&D: the Chorus modelPaul K. Owens,Eyas Raddad,Jeffrey W. Miller,John R. Stille,Kenneth G. Olovich,Neil V. Smith,Rosie S. Jones& Joel C. SchererAffiliationsCorresponding authorNature Reviews Drug Discovery (2014) doi:10.1038/nrd4497Published online 15 December 2014
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Immune system may play role in obesity - 13abc Action News

Immune system may play role in obesity - 13abc Action News | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Toledo news and weather from 13abc. Breaking news updates, local sports coverage and the area's only live doppler radar
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A Fresh Look at Fighting Glaucoma

A Fresh Look at Fighting Glaucoma | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
A team of Novartis researchers is going back to basics in search of the roots of glaucoma and new opportunities to fight this stealthy thief of eyesight.

 

Glaucoma ultimately robs some 60 million people worldwide of their sight. Researchers at Novartis want to cut that number dramatically. The company is building a new research team specifically to learn more about what makes glaucoma tick at the cellular level—and to see if any of the 1.6 million compounds on the company's shelves can stop the disease in its tracks. Over time it quietly increases the fluid pressure in the eye (what doctors call intraocular pressure), slowly destroying optic nerve cells, narrowing vision to an ever-shrinking tunnel.



Krishan Maggon 's insight:
Flow in the glaucoma eye

The aqueous humor is continuously made inside the eye to nourish the clear, avascular tissues such as the cornea and lens. It drains out of the front of the eye through a plumbing network comprised of a series of three spongy tissues and ducts: the trabecular meshwork, the juxtacanalicular tissue, and Schlemm's canal. In patients with glaucoma:

the trabecular meshwork has fewer cellsthe remaining cells in the meshwork contract more and are stiffer than usualthe spongy tissues within the drainage pathway are more compact and less porous than those in a healthy eye

It's not clear why these changes occur or how significant each is to the disease, though there are several hypotheses. For instance, the loss of cells in the trabecular meshwork may make it harder for this spongy tissue to sense the humor's pressure as it drains from the eye. What is known is that even though this pathway accounts for the majority of aqueous humor drainage, it has never before been modeled for use as a drug screen.

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FDA approves Opdivo (nivolumab, BMS) for advanced melanoma

FDA approves Opdivo (nivolumab, BMS) for advanced melanoma | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
The U.S. Food and Drug Administration today granted accelerated approval to Opdivo (nivolumab), a new treatment for patients with unresectable (cannot be removed by surgery) or metastatic (advanced) melanoma who no longer respond to other drugs.

 

Melanoma is the fifth most common type of cancer in the United States. It forms in the body’s melanocyte cells, which develop the skin’s pigment. The National Cancer Institute estimates that 76,100 Americans will be diagnosed with melanoma and 9,710 will die from the disease this year.

Opdivo works by inhibiting the PD-1 protein on cells, which blocks the body’s immune system from attacking melanoma tumors. Opdivo is intended for patients who have been previously treated with ipilimumab and, for melanoma patients whose tumors express a gene mutation called BRAF V600, for use after treatment with ipilimumab and a BRAF inhibitor.

 

 

Opvido is being approved under the FDA’s accelerated approval program, which allows approval of a drug to treat a serious or life-threatening disease based on clinical data showing the drug has an effect on a surrogate endpoint reasonably likely to predict clinical benefit to patients. This program provides earlier patient access to promising new drugs while the company conducts additional clinical trials to confirm the drug’s benefit.

Opdivo’s efficacy was demonstrated in 120 clinical trial participants with unresectable or metastatic melanoma. Results showed that 32 percent of participants receiving Opdivo had their tumors shrink (objective response rate). This effect lasted for more than six months in approximately one-third of the participants who experienced tumor shrinkage.   

Opdivo’s safety was evaluated in the overall trial population of 268 participants treated with Opdivo and 102 participants treated with chemotherapy. The most common side effects of the drug were rash, itching, cough, upper respiratory tract infections, and fluid retention (edema). The most serious side effects are severe immune-mediated side effects involving healthy organs, including the lung, colon, liver, kidneys and hormone-producing glands.

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Krishan Maggon 's curator insight, December 23, 2:00 AM

The FDA granted Opvido breakthrough therapy designation, priority review and orphan product designation because the sponsor demonstrated through preliminary clinical evidence that the drug may offer a substantial improvement over available therapies; the drug had the potential, at the time of the application was submitted, to be a significant improvement in safety or effectiveness in the treatment of a serious condition; and the drug is intended to treat a rare disease, respectively.


Other FDA-approved treatments for melanoma include ipilimumab (2011), peginterferon alfa-2b (2011), vemurafenib (2011), dabrafenib (2013), trametinib (2013) and pembrolizumab (2014). Opdivo is being approved more than three months ahead of the prescription drug user fee goal date of March 30, 2015, the date when the agency was scheduled to complete its review of the application.

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The Most Disappointing Clinical Failures in 2014 - Motley Fool

The Most Disappointing Clinical Failures in 2014 - Motley Fool | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
Three Motley Fool contributors tell us what they think were the biggest drug trial failures this year. - Motley Fool Staff - Health Care
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Good overview of the major clinical trial failures of much hyped potential blockbuster drugs

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Conservancy of mAb Epitopes in Ebolavirus Glycoproteins of Previous and 2014 Outbreaks – PLOS Currents Outbreaks

Conservancy of mAb Epitopes in Ebolavirus Glycoproteins of Previous and 2014 Outbreaks – PLOS Currents Outbreaks | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
ABSTRACT

Background: Several monoclonal antibodies (mAb) are being evaluated as treatment options for the current 2014 Ebola outbreak. But they were derived from and tested for protection against the older 1976 Mayinga or 1995 Kikwit Zaire Ebolaviruses (EBOV). The EBOV sequences reported for the current outbreak contain several mutations whose significance remained to be established.

 

Methods: We analyzed sequence and structural conservation of the Ebolavirus glycoprotein (GP) epitopes for all experimentally identified protective mAbs published to date.

 

Results: The conservancy analysis of protective mAb epitopes in the Ebolavirus glycoprotein sequences spanning all Ebola virus lineages since 1976 showed that conservancy within the Zaire EBOV lineage was high, with only one immunodominant epitope of mAb 13F6-1-2 acquiring two novel mutations in the 2014 outbreak that might potentially change the antibody specificity and neutralization activity. However, the conservation to other Ebola viruses was unexpectedly low.

 

Conclusion: Low conservancy of Zaire EBOV mAb epitopes to other EBOV lineages suggests that these epitopes are not indispensable for viral fitness, and that alternative mAbs could be developed to broadly target all EBOV. However, average percent sequence identity of the epitopes for mAbs used in current cocktails to the Zaire EBOV is high with only one epitope differing in the 2014 outbreak. These data bode well for general usefulness of these antibodies in the context of the current outbreak.

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PLoS Currents Outbreak

 

Conservancy of mAb Epitopes in Ebolavirus Glycoproteins of Previous and 2014 OutbreaksNOVEMBER 3, 2014 · RESEARCHPrint Article   AUTHORSJulia PonomarenkoKerrie VaughanAlessandro SetteSebastian Maurer-Stroh
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Tolerance Induction and Reversal of Diabetes in Mice Transplanted with Human Embryonic-Stem-Cell-Derived Pancreatic Endoderm: Cell Stem Cell

Tolerance Induction and Reversal of Diabetes in Mice Transplanted with Human Embryonic-Stem-Cell-Derived Pancreatic Endoderm: Cell Stem Cell | Pharma Biotech Industry Review (Krishan Maggon) | Scoop.it
From UCSF Bluestone lab: using #immunotherapy to allow transplant of insulin-producing cells in #diabetes treatment http://t.co/BeeslRGTnI

 

Summary

Type 1 diabetes (T1D) is an autoimmune disease caused by T cell-mediated destruction of insulin-producing β cells in the islets of Langerhans. In most cases, reversal of disease would require strategies combining islet cell replacement with immunotherapy that are currently available only for the most severely affected patients. Here, we demonstrate that immunotherapies that target T cell costimulatory pathways block the rejection of xenogeneic human embryonic-stem-cell-derived pancreatic endoderm (hESC-PE) in mice. The therapy allowed for long-term development of hESC-PE into islet-like structures capable of producing human insulin and maintaining normoglycemia. Moreover, short-term costimulation blockade led to robust immune tolerance that could be transferred independently of regulatory T cells. Importantly, costimulation blockade prevented the rejection of allogeneic hESC-PE by human PBMCs in a humanized model in vivo. These results support the clinical development of hESC-derived therapy, combined with tolerogenic treatments, as a sustainable alternative strategy for patients with T1D.

Krishan Maggon 's insight:

Highlights

 

•Costimulation blockade prevents rejection of xenogeneic hESC-derived islets•Short-term treatment induces long-term tolerance to xenogeneic hESC-derived islets•Tolerance induced by costimulation blockade is transferable independently of Tregs•Costimulation blockade prevents rejection of allogeneic hESC islets by human PBMCs  Tolerance Induction and Reversal of Diabetes in Mice Transplanted with Human Embryonic-Stem-Cell-Derived Pancreatic EndodermGregory L. Szot, Mahesh Yadav, Jiena Lang, Evert Kroon, Justin Kerr, Kuniko Kadoya, Eugene P. Brandon, Emmanuel E. Baetge, Hélène Bour-Jordan3,Jeffrey A. Bluestone33Co-senior author DOI: http://dx.doi.org/10.1016/j.stem.2014.12.001Publication stage: In Press Corrected Proof

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