Eli Lilly and Company announced that the U.S. Food and Drug Administration (FDA) has granted approval for a new indication for ALIMTA® (pemetrexed for injection) in combination with KEYTRUDA® (pembrolizumab), developed and marketed by Merck (known as MSD outside the U.S. and Canada), and platinum chemotherapy for the first-line treatment of patients with metastatic nonsquamous non-small cell lung cancer (NSCLC), with no EGFR or ALK genomic tumor aberrations. This indication is approved based on data from Merck's Phase 3 KEYNOTE-189 trial, which enrolled patients regardless of PD-L1 expression and had dual primary endpoints of overall survival (OS) and progression-free survival (PFS).

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Customer focus. Patient centricity. Innovation. Changing mindsets. These have all become “buzzwords” in our industry, but who is actually making headway, changing outcomes, going beyond the status quo?
Instead of following the beaten path that predecessors and competitors had already well-trodden, the companies below are actually doing it - evidenced by the fact they have reached the final of the eyeforpharma Awards.
Over 20 of pharma’s most innovative and impactful projects have been shortlisted by eyeforpharma’s independent judging panel. One of the judges Alvaro Herreros, Vice President - Global Neuroscience Franchise Head at Shire said, “It was great to see different initiatives presented with a variety of diseases and approaches, from general awareness to patient adherence,”.
You can see exactly how these companies are doing it and learn from them at the eyeforpharma Awards Pitch Day (Europe - 11 March 2019, North America – April 15 2019).

Find out more about the Awards Pitch Day Europe here: http://bit.ly/2Rj42C7

Find out more about the Awards Pitch Day North America here: http://bit.ly/2RiVkUq

Any questions? Contact Lucy Fisher, Awards Manager on lucy@eyeforpharma.com.

#healthcare #pharmaceutical #medical #clinicalresearch

Merck KGaA, Darmstadt, Germany, a leading science and technology company, which operates its healthcare business in the U.S. and Canada as EMD Serono, announced that it has entered into an exclusive licensing agreement with Vertex Pharmaceuticals Incorporated, Boston, USA for two DNA-dependent protein kinase (DNA-PK) inhibitors - M9831 (formerly known as VX-984) and an additional pre-clinical compound - in the field of gene editing for six specific genetic disease indications. Merck KGaA, Darmstadt, Germany will receive an upfront payment in addition to milestones and royalties on future net sales and retains the rights to both assets in all other disease areas, including oncology, with the ability to develop both these compounds in-house, or to license them to future partners in the gene editing field. Vertex has the option to add indications to the license grant. Both molecules were acquired in a licensing agreement from Vertex in 2017, and are part of the company's broad portfolio of DNA Damage Response (DDR) inhibitors.

Read more: https://prn.to/2UgiU66

Biohaven Pharmaceutical Holding Company Ltd. announced the U.S. Food and Drug Administration (FDA) has notified the Company that it may proceed with its clinical investigation of BHV-3241, a novel myeloperoxidase (MPO) inhibitor, which Biohaven is developing as a potential treatment for multiple system atrophy (MSA). The FDA May Proceed Letter was received following Biohaven's reactivation of the investigational new drug (IND) application initially filed by AstraZeneca prior to licensing the compound to Biohaven.

BHV-3241 is a potential first-in-class, oral, brain-penetrant, irreversible inhibitor of MPO, an enzyme that acts as a key driver of pathological oxidative stress and inflammation in the brain. MPO inhibition is a promising therapeutic strategy for treating a number of neurodegenerative diseases, including MSA.

Read more: https://prn.to/2R3BCfv

REACH-2 is a global, randomized, double-blind, placebo-controlled Phase 3 study of CYRAMZA and best supportive care (BSC) compared to placebo and BSC in hepatocellular carcinoma (HCC) patients who were intolerant to, or who had disease progression while on or following treatment with, sorafenib and had a high alpha-fetoprotein (AFP-High), defined as an AFP of ≥400 ng/mL.

Initiated in 2015, the study has enrolled 292 patients across 20 countries in North America, Asia, Europe and Latin America. Patients were assigned to 8 mg/kg of intravenous CYRAMZA (n=197) or placebo (n=95) at every two weeks until disease progression, unacceptable toxicity, or withdrawal of consent, with all patients receiving BSC. The primary endpoint of the REACH-2 trial is overall survival (OS) and key secondary endpoints include progression-free survival, objective response rate, quality of life and safety.

Read more: https://prn.to/2HnKexP

EVENITY is a bone forming agent that both increases bone formation and reduces bone resorption to increase bone mineral density (BMD) and reduce the risk of fracture. The approval is based on results from two pivotal Phase 3 studies: FRAME,3 which included 7,180 postmenopausal women with osteoporosis, and BRIDGE,4 which included 245 men with osteoporosis. The Japanese Pharmaceuticals and Medical Devices Agency undertook a thorough review of the safety profile of EVENITY, including the cardiovascular safety findings in the ARCH trial.

"In Japan, osteoporotic fracture is one of the leading causes for patients losing independence and needing nursing care. As the aged population of Japan increases, preventing such fractures should be given high priority," said Steve Sugino, Amgen vice president and president and representative director of AABP. "Japanese patients will be the first in the world to have a new therapeutic option for osteoporosis that reduces the risk of fracture by not only increasing bone formation but also decreasing bone resorption."

Read more: https://prn.to/2sky0LW

A fully integrated biopharmaceutical company – Uni-Bio Science Group Limited , together with its subsidiaries, is pleased to announce that the bioequivalence study ("BE Study") and its clinical materials on Acarbose tablets jointly developed by Beijing Genetech Pharmaceutical Co., Limited (a wholly-owned subsidiary of the Company) and Beijing Baiao Pharmaceutical Co., Ltd. has been formally accepted by the National Medical Products Administration ("NMPA") with reference number of CYHS1900008GUO and CYHS1900007GUO, corresponding to the oral tablets under two specifications of 50mg and 100mg for this submission respectively. This submission, if approved, means that the Company will be qualified for batch production of such product, which will be produced and launched to the Mainland China market as newly approved generic drugs.

Acarbose tablet is an oral anti-diabetic drug which belongs to the Alpha-Glucosidase Inhibitors class. It is used to treat Type 2 diabetes and is reimbursed under the National Reimbursement Drug List. It targets patients with pre-diabetes condition who need to be treated in early stage, and those with post prandial hyperglycemia under control.

Read more: https://www.prnewswire.com/news-releases/uni-bio-sciences-anti-diabetic-acarbose-oral-tablets-be-study-application-accepted-by-the-national-medical-products-administration-300773017.html

Eli Lilly and Company and Incyte Corporation announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to baricitinib, which is being studied for the treatment of systemic lupus erythematosus (SLE). The Fast Track designation process aims to facilitate the development and expedite the review of new medicines that treat serious conditions and fill unmet medical needs, with the goal of delivering potentially important therapies to patients sooner.

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Viracor Eurofins announced the recent launch of a new 16S Next Generation Sequencing (NGS) Bacterial Meningitis test for use in patients suspected of having bacterial meningitis. Physicians will receive test results for difficult-to-diagnose immunocompromised patients within 3 business days from specimen receipt. Knowing the specific pathogen involved in central nervous system (CNS) infection is crucial for selection of the appropriate therapeutic to achieve rapid recovery and to minimize the risk of permanent CNS damage. Viracor is expanding its test menu to include this and other NGS testing in support of enhanced pathogen detection and transplant rejection assessment.

Read more: https://prn.to/2QWwXA9

Biohaven Pharmaceutical Holding Company Ltd. today announced initial positive results from its ongoing long-term, open-label safety study of its oral calcitonin gene-related peptide (CGRP) receptor antagonist, rimegepant, for the acute treatment of migraine. Based upon this interim analysis (database cutoff of November 21, 2018) of Study BHV3000-201 (NCT03266588 or "Study 201") it appears that rimegepant may be safe and well tolerated with long-term dosing in patients with migraine. In this study, patients are allowed to treat migraine attacks of all severities (mild to severe) up to once daily for up to a full year.
Migraine is both a widespread and disabling neurological disorder. The Migraine Research Foundation ranks migraine as the world's third most prevalent illness, affecting approximately 36 million people or 1 out of 4 households in the United States. And the Global Burden of Disease Study 2015 rates migraine as the seventh highest specific cause of disability worldwide. More than 90% of migraine sufferers are unable to work or function normally during an attack.

Read more: https://prn.to/2QspYQ0

Chimeric antigen receptor (CAR) T-cell therapies have quickly shifted the treatment paradigm for many people with several types of aggressive blood cancers, in whom treatment options have been woefully limited. Five studies being presented today at the 60th American Society of Hematology (ASH) Annual Meeting and Exposition spotlight the long-term effectiveness of these therapies in some patients, analyze how combination therapies might affect treatment responses, and examine if transplantation following CAR T-cell therapy affects remission rate.

CAR T-cell therapies are designed by harvesting a patient's own T cells (the immune system's primary cancer-killing cells), reengineering them to target specific proteins on the surface of cancer cells, and reintroducing the modified T cells back into the patient's immune system.

Read more: https://prn.to/2KUlw6q

The U.S. Food and Drug Administration today approved Firdapse (amifampridine) tablets for the treatment of Lambert-Eaton myasthenic syndrome (LEMS) in adults. LEMS is a rare autoimmune disorder that affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. This is the first FDA approval of a treatment for LEMS.

"There has been a long-standing need for a treatment for this rare disorder," said Billy Dunn, M.D., director of the Division of Neurology Products in the FDA's Center for Drug Evaluation and Research. "Patients with LEMS have significant weakness and fatigue that can often cause great difficulties with daily activities."

In people with LEMS, the body's own immune system attacks the neuromuscular junction (the connection between nerves and muscles) and disrupts the ability of nerve cells to send signals to muscle cells. LEMS may be associated with other autoimmune diseases, but more commonly occurs in patients with cancer such as small cell lung cancer, where its onset precedes or coincides with the diagnosis of cancer. The prevalence of LEMS is estimated to be three per million individuals worldwide.

Read more: https://prn.to/2Sox985

Moleculin Biotech, a clinical stage pharmaceutical company focused on the development of oncology drug candidates, all of which are based on license agreements with The University of Texas System on behalf of the M.D. Anderson Cancer Center, today announced that data from an independent test in animal models confirmed, as previously believed, that its immuno-stimulating STAT3 inhibitor achieves disproportionately high accumulation in the pancreas.

"Our own sponsored research suggested that WP1732 might be an ideal candidate for treating pancreatic cancer," commented Dr. Donald Picker, Moleculin's Chief Science Officer. "Now, we have independent testing with radiolabeled drug confirming this in animal models. The propensity for such enhanced pancreatic distribution could be highly beneficial for a new pancreatic cancer drug."

Read more: https://prn.to/2ra1tYL

Biohaven Pharmaceutical Holding Company Ltd. today announced that its 505(b)(2) new drug application (NDA) for BHV-0223, an investigational sublingual form of riluzole for the treatment of people living with amyotrophic lateral sclerosis (ALS), has been accepted for review by the U.S. Food and Drug Administration (FDA). Biohaven previously announced that the FDA granted orphan drug designation for BHV-0223 in ALS. BHV-0223 is a novel, lower dose formulation of riluzole designed to be placed under the tongue where it dissolves in seconds and is absorbed sublingually. The active ingredient, riluzole, is the only FDA-approved treatment to extend tracheostomy-free survival in people living with ALS.
ALS is a progressive, neurodegenerative motor neuron disease that affects nerve cells in the brain and the spinal cord. The disease belongs to a group of disorders known as motor neuron diseases, which are characterized by the gradual degeneration and death of motor neurons.

Read more: https://prn.to/2THkHlo

Synthetic Biologics, Inc. (NYSE American: SYN), a late-stage clinical company developing therapeutics designed to preserve the microbiome to protect and restore the health of patients, today announced that it has successfully completed an End-of-Phase 2 meeting with the U.S. Food and Drug Administration (FDA) to discuss development of SYN-004 (ribaxamase) for the prevention of antibiotic-mediated Clostridium difficile infection (CDI). Pursuant to the meeting, the FDA has proposed criteria for Phase 3 clinical efficacy and safety which, if achieved, may support submission for marketing approval of ribaxamase on the basis of a single Phase 3 clinical trial. Final agreement on these criteria is contingent on FDA evaluation of a detailed Phase 3 clinical trial protocol.

Read more: https://lnkd.in/gd7V-Se

MRI brain scans perform better than common clinical tests at predicting which people will go on to develop Alzheimer's disease, according to a study being presented next week at the annual meeting of the Radiological Society of North America (RSNA).

Alzheimer's disease is a progressive, irreversible brain disorder that destroys memory and thinking skills. The disease affects 5.5 million Americans, according to the National Institutes of Health.

"Alzheimer's disease is the most common cause of dementia in the world and is expected to increase globally, and especially in the U.S., as the population gets older," said the study's lead author Cyrus A. Raji, M.D., Ph.D., assistant professor of radiology at the Mallinckrodt Institute of Radiology at Washington University School of Medicine in St. Louis. "As we develop new drug therapies and study them in trials, we need to identify individuals who will benefit from these drugs earlier in the course of the disease."

Read more: https://lnkd.in/g2F8hjW

Applied BioMath (www.appliedbiomath.com), the industry-leader in applying mechanistic modeling to drug research and development, today announced a collaboration with Obsidian Therapeutics to provide semi-mechanistic models and analysis services. Applied BioMath will create semi-mechanistic models, using in vitro and in vivo preclinical data, to aid in dose prediction and knowledge gap identification for Obsidian's promising therapeutic strategy. "Our Destablizing Domain platform is exceedingly powerful but also pharmacologically complex," said Steve Shamah, Senior Vice President of Research, of Obsidian Therapeutics. "We chose to work with Applied BioMath because of their proven track record of delivering impactful results in a very short timeframe."

Obsidian Therapeutics, founded by Atlas Venture in 2015, is developing next-generation cell and gene therapeutics that employ precise exogenous control of transgenes for improved safety and efficacy. Applied BioMath will leverage their proprietary algorithms and software to develop semi-mechanistic models for Obsidian using rigorous fit-for-purpose model development processes and optimization techniques to estimate model parameters and variability.

Read more: https://www.prnewswire.com/news-releases/applied-biomath-llc-announces-collaboration-with-obsidian-therapeutics-for-semi-mechanistic-quantitative-modeling-of-a-novel-therapeutic-platform-300753053.html

Royal Philips, a global leader in health technology, today announced the Philips V60 Plus ventilator has received CE mark approval. This comprehensive solution expands on Philips' noninvasive ventilation (NIV) gold standard platform, integrating both NIV and high flow therapy (HFT) in a single device. Clinicians can rapidly adjust therapies around constantly changing patient conditions without having to switch devices. Designed for early intervention in respiratory failure, the V60 Plus enables clinicians to further enhance patient outcomes with less invasive respiratory care therapies, while improving clinician workflow and maximizing equipment investments.

Read more: https://prn.to/2A18yia

The letter from the FDA gives permission for Vyera to proceed with dosing human subjects in its Phase 1 SAD study of VYR-006. Vyera intends to begin a multiple ascending dose study pending satisfactory completion of the SAD study.
Toxoplasmosis is caused by the parasite Toxoplasma gondii, and more than 40 million people in the United States may be infected by the parasite, which can persist in the body for a lifetime. A toxoplasma infection could cause serious health problems ranging from flu-like symptoms to causing damage to the brain, eyes and other organs, particularly in pregnant women or individuals who have compromised immune systems.

Read more: https://prn.to/2PyT0gg

Eli Lilly and Company has announced the submission of a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) for lasmiditan for the acute treatment of migraine with or without aura in adults.

Lasmiditan is an investigational, oral, centrally-penetrant, selective serotonin 5-HT1F agonist that is structurally and mechanistically distinct from other approved migraine therapies and lacks vasoconstrictive activity. It is the first and only molecule in the "-ditan" class under evaluation for the acute treatment of migraine in adults. If approved, it could represent the first significant innovation for the acute treatment of migraine in more than two decades.

Read more: https://prn.to/2PtMwz6

REGENXBIO Inc. (Nasdaq: RGNX), a leading clinical-stage biotechnology company seeking to improve lives through the curative potential of gene therapy based on its proprietary NAV® Technology Platform, today announced the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation to RGX-181, a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease caused by mutations in the tripeptidyl peptidase 1 (TPP1) gene.

"We believe RGX-181 has the potential to correct the underlying genetic condition, halt progression and address many of the serious and life-threatening symptoms of CLN2 disease," said Kenneth T. Mills, President and Chief Executive Officer of REGENXBIO. "CLN2 disease is an extremely debilitating disease in children with no cure and limited treatment options. Receiving Orphan Drug Designation from the FDA signifies our continued progress and commitment to develop RGX-181 as a potential one-time treatment for children with CLN2 disease."

FDA Orphan Drug Designation is granted to investigational therapies addressing rare medical diseases or conditions that affect fewer than 200,000 people in the United States. Orphan drug status provides benefits to drug developers including assistance in the drug development process, tax credits for clinical costs, exemptions from certain FDA fees and seven years of marketing exclusivity.

Read more: https://www.prnewswire.com/news-releases/fda-grants-orphan-drug-designation-to-rgx-181-gene-therapy-for-the-treatment-of-cln2-form-of-batten-disease-300749729.html

Evox Therapeutics Ltd, a leading exosome therapeutics company, today announces that it has secured £655,000 in funding from Duchenne UK. The grant will be invested to support exploration of the Company's exosome-based therapeutic platform to deliver either full-length dystrophin or its shorter variants in pre-clinical models of Duchenne muscular dystrophy ('Duchenne').

Duchenne is a highly debilitating, progressive, muscle-wasting disorder caused by the lack of functional dystrophin protein, for which there is currently no cure. Delivery of dystrophin or its shorter variants to these patients has the potential to be a highly effective treatment option in Duchenne.

Evox is engineering exosomes, the body's natural vesicular delivery system, to enable a wide variety of drugs to reach previously inaccessible tissues and compartments, such as crossing the blood brain barrier to deliver drugs to the central nervous system, intracellular delivery of proteins, and extra-hepatic delivery of RNA therapeutics.

Read more: https://prn.to/2PW3B4f

Animated by Phospho Biomedical: https://www.phospho.co.uk

Enable Injections, Inc., a patient-centric drug delivery combination product company developing advanced wearable devices for subcutaneous delivery of high-volume therapeutics, today announced it has entered into development agreements with Apellis Pharmaceuticals, and most recently, UCB. Enable Injections currently has partnerships with five companies to deliver products that shift treatment to the home with easy, convenient self-administration of new large-volume drugs.

Read more: https://prn.to/2B3gYr6

Theravance Biopharma, Inc. and Mylan today announced that the U.S. Food and Drug Administration (FDA) has approved the New Drug Application (NDA) for YUPELRITM (revefenacin) inhalation solution for the maintenance treatment of patients with chronic obstructive pulmonary disease (COPD). YUPELRI, a long-acting muscarinic antagonist (LAMA), is the first and only once-daily, nebulized bronchodilator approved for the treatment of COPD in the US. The companies expect YUPELRI to be available to COPD patients in the US before the end of the year. COPD is the third leading cause of death and the fourth leading cause of hospital readmissions in the US, affecting approximately 16 million Americans.

Read more: https://prn.to/2z6XBfo

"Excessive daytime sleepiness associated with narcolepsy or obstructive sleep apnea can be debilitating for patients and challenging for the medical community to diagnose and treat," said Jed Black, M.D., senior vice president, Sleep and CNS Medicine at Jazz Pharmaceuticals and adjunct professor, Stanford University Medical Center, Stanford Center for Sleep Sciences and Medicine. "Jazz is committed to addressing unmet needs in sleep medicine by delivering meaningful treatment options."

"In the European Union, there is no approved treatment for people with excessive daytime sleepiness associated with obstructive sleep apnea, and people with excessive daytime sleepiness due to narcolepsy may benefit from the availability of new treatment options," said Professor Yves Dauvilliers, M.D., Ph.D. Head of Sleep and Wake Disorders at the University Hospital of Montpellier, France. "As the sleep medical community improves its understanding and diagnosis of these conditions, it is increasingly important that patients have multiple treatment options available to help them manage their condition more effectively."

Jazz has studied solriamfetol extensively via the Treatment of OSA and Narcolepsy Excessive Sleepiness (TONES) Phase 3 program, which was comprised of four studies that evaluated:

solriamfetol in EDS in adult patients with narcolepsy (TONES 2) or OSA (TONES 3 and TONES 4), and
the long-term safety and maintenance of efficacy for solriamfetol as a treatment for EDS in patients with narcolepsy or OSA (TONES 5).
Jazz presented long-term safety and maintenance of efficacy results from the TONES 5 Phase 3 study of solriamfetol in adult patients with EDS associated with OSA or with narcolepsy at the annual meeting of the Associated Professional Sleep Societies (APSS) in June 2018. TONES 2, 3 and 4 data were presented at APSS in 2017.

Read more: https://prn.to/2QAugRn