An experimental therapy developed to address the genetic cause of a Amyotrophic lateral sclerosis (ALS), rare form of motor neurone disease (MND) has shown encouraging results in early research supported by the NIHR.

The phase 1-2 study, supported by the National Institute for Health Research and published in the New England Journal of Medicine, has shown tofersen is safe and well tolerated in people with a genetic form of MND.

Around 5,000 people in the UK have MND, a disorder that affects the nerves in the brain and spinal cord that form the connection between the nervous system and muscles, enabling movement of the body.

In people with the disease, the messages from these nerves gradually stop reaching the muscles, leading them to weaken, stiffen and eventually waste, affecting a patient’s ability to walk, talk, use their arms and hands, eat and breathe.

Read more: http://www.pharmatimes.com/news/experimental_motor_neurone_disease_therapy_shows_early_promise_1344775

Experimental motor neurone disease therapy shows early promise - PharmaTimes

Phase I/II study results demonstrate proof-of-concept and proof-of-biology of tofersen - News - PharmaTimes

ALS is a severe, neurodegenerative disease which affects motor neurons leading to progressive muscle weakness, paralysis and ultimately death within a median time of two to four years from disease onset.
According to the firm, the ODD recognises the potential therapeutic role of aldesleukin in treating disease, and could provide a number of benefits for Clinigen should it obtain a marketing approval for this indication.
These benefits include seven years marketing exclusivity within the US upon launch, along with tax credits for clinical development costs and fee waivers.
Studies in ALS patients have shown that decreased levels of regulatory T-cells (Tregs) were associated with increased disease severity and predictive of disease progression and survival, suggesting that Tregs may be an attractive therapeutic target.


http://www.pharmatimes.com/news/clinigens_als_therapy_bags_us_orphan_status_1344779

Clinigen's ALS therapy bags US orphan status - PharmaTimes

Clinigen has bagged a US orphan drug designation (ODD) for its investigational Amyotrophic Lateral Sclerosis (ALS) treatment aldesleukin. - News - PharmaTimes

Melbourne-based Affinity Biosciences Pty Ltd (Affinity), a privately held company focused on antibody discovery for cancer therapeutics, today announces the discovery of potent antibodies with therapeutic potential against COVID-19.

In March, Affinity commenced screening its proprietary library of one hundred billion human antibodies to discover those that might neutralise SARS-CoV-2, the virus that causes COVID-19. After discovering a number of candidate antibodies that block the virus spike protein from binding to its human receptor, Affinity engaged The Peter Doherty Institute for Infection and Immunity (Doherty Institute) in April to conduct testing using the virus in the laboratory. The Doherty Institute has confirmed that Affinity's antibodies can completely neutralise the infectivity of SARS-CoV-2 at a single digit microgram/ml concentration in a five-day virus neutralisation assay. The antibodies have high affinity for the SARS-CoV-2 spike protein at around 20 picomolar, driven by an exceptionally slow off-rate, a measure of how long the antibody remains bound to its target.

"Our antibodies latch onto the virus spike protein and block its activity," said Affinity CEO, Dr Peter Smith. "These antibodies may be protective in humans by preventing the virus from gaining a foothold in healthy people exposed to the virus, and may be useful in arresting the progress of the virus in an infected person by blocking its ability to infect new cells and replicate."

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

Affinity discovers potent SARS-CoV-2 antibodies

/PRNewswire/ -- Melbourne-based Affinity Biosciences Pty Ltd (Affinity), a privately held company focused on antibody discovery for cancer therapeutics, today...

Carmine Therapeutics announced today that it has signed a research collaboration agreement with Takeda Pharmaceutical Company Limited ("Takeda") to discover, develop and commercialize transformative non-viral gene therapies for two rare disease targets using Carmine's REGENT(TM) technology, based on red blood cell extracellular vesicles. In addition, Takeda has committed a $5M convertible loan in support of the development of Carmine's novel REGENT(TM) platform.
Under the terms of the agreement, Carmine will receive an upfront payment, research funding support and is eligible for over $900M in total milestone payments plus tiered royalties. Takeda has an option to license the programs following the completion of pre-clinical proof of concept studies and would be responsible for clinical development and commercialization.

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Carmine Therapeutics and Takeda Collaborate to Discover and Develop Rare Disease Gene Therapies Using Novel Red Blood Cell Extracellular Vesicles Platform

/PRNewswire/ -- Carmine Therapeutics announced today that it has signed a research collaboration agreement with Takeda Pharmaceutical Company Limited...

Vertex has won a positive recommendation from the European Medicines Agency’s (EMA) Committee for Medicinal Products for Human Use (CHMP) for its triple combination cystic fibrosis therapy, alongside a host of other recommendations announced in the Committee’s June meeting.
The CHMP has recommended granting a conditional marketing authorisation for the triple therapy, comprised of elexacaftor/tezacaftor/ivacaftor, under the brand name Kaftrio. It is the first triple combination therapy for the treatment of CF in patients aged 12 years and older with one F508del mutation and one minimal function mutation and in patients with two F508del mutations.
The triple therapy was approved by the US Food and Drug Administration (FDA) in October last year, and scored that approval only a few months after filing the combination treatment with the agency. In the US, the drug was approved under the brand name Trikafta, and priced at $311,000 for a year of treatment.
The treatment provides a new treatment option for many CF patients, including for those with certain mutations where no treatment is currently available in the EU.

Read more: http://www.pmlive.com/pharma_news/vertexs_triple_cf_therapy_scores_recommendation_from_chmp_1343261

Vertex’s triple CF therapy scores recommendation from CHMP - PMLiVE

Vertex’s triple CF therapy scores recommendation from CHMP - PMLiVE

Gilead Sciences plans to start testing an inhaled formulation of its lead COVID-19 therapy remdesivir – currently dosed intravenously – that could allow it to be given to earlier-stage patients outside hospital.
In an open letter, Gilead’s chief executive Daniel O’Day said the new formulation of the drug – given via a nebuliser – will be given to healthy volunteers in a phase 1 trial due to start in August.
If it works, the inhaled version of the antiviral “could have significant implications in helping to stem the tide of the pandemic”, according to O’Day.
Remdesivir was the first drug to be shown to have an effect on COVID-19 in controlled clinical trials, and is currently the only drug with emergency use authorisation to treat COVID-19 in the US after the FDA pulled approval for chloroquine and hydroxychloroquine last week.
Gilead’s drug has shown that it can reduce the time spent in hospital for patients hospitalised with severe COVID-19 symptoms – down to 11 days from around 15 in a trial run by the National Institute of Allergy and Infectious Disease (NIAID) – although it hasn’t been proven to improve the chances of surviving the disease.

Read more: http://www.pmlive.com/pharma_news/gilead_will_test_inhaled_remdesivir_in_earlier-stage_covid-19_1342768

Gilead will test inhaled remdesivir in earlier-stage COVID-19 - PMLiVE

Gilead will test inhaled remdesivir in earlier-stage COVID-19 - PMLiVE

Sanofi Pasteur and Translate Bio have agreed to expand their existing 2018 collaboration and license agreement to develop mRNA vaccines for infectious diseases.
Under the expanded deal, Translate Bio will receive a total upfront payment of $425 million, consisting of a $300 million cash payment and a private placement common stock investment of $125 million at $25.59 per share, representing a 50% premium to the 20-day moving average share price prior to signing.
Translate Bio will also be eligible for potential future milestones and other payments up to $1.9 billion, including $450 million of milestones under the 2018 agreement.
Of these, around $360 million are anticipated over the next several years, inclusive of COVID-19 vaccine development milestones, and Translate Bio is also eligible to receive tiered royalty payments on worldwide sales of any vaccines making it to market.

Read more: https://bit.ly/3fQe0rA

Sanofi pays $300m upfront to expand deal with Translate Bio - PharmaTimes

The firms are working on the development of mRNA vaccines - News - PharmaTimes

US private biotech Cancer Prevention Pharmaceuticals has filed a marketing application in Europe for CPP-1X/sul, a preventative treatment for patients with familial adenomatous polyposis (FAP).

FAP is a rare genetic disease that, if left untreated, causes colorectal cancer in nearly 100% of patients.

Read more: https://bit.ly/2ChZ6eZ

EU filing for CPP's familial adenomatous polyposis drug - PharmaTimes

The drug prevented more than 90% of precancerous sporadic adenomas in trials - News - PharmaTimes

Mylan today announced that the U.S. District Court for the Northern District of West Virginia invalidated Biogen's Tecfidera® patent, U.S. Patent No. 8,399,514, for lack of written description. The ᾿514 patent claimed methods of treating multiple sclerosis (MS) using a dose of 480 mg/day of dimethyl fumarate delayed release capsules. Today's decision clears the way for Mylan's launch of its dimethyl fumarate product upon the receipt of FDA approval. The '514 patent could have otherwise delayed generic competition until 2028.

Mylan CEO Heather Bresch commented: "Today's win is significant in that it brings Mylan one step closer to providing expanded treatment options for the thousands of Americans living with relapsing forms of MS. The District Court decision clears the legal pathway for us to bring our dimethyl fumarate product to market, and we are working with the FDA to accelerate our regulatory approval target action date, which currently is November 16. Once approved and launched, we believe our generic Tecfidera will potentially be the first generic of any MS treatment in an oral solid dosage form available to patients in the U.S., further advancing our commitment to provide a broad portfolio of central nervous system medicines, which already includes glatiramer acetate injection."

Mylan President Rajiv Malik commented: "Mylan continues to address unmet patient needs across the value chain. Bringing a product like generic Tecfidera to market requires not only our extensive scientific and commercial expertise, but also our unwavering commitment to access through overcoming potential legal barriers to reach patients. We look forward to bringing this important medicine to market as soon as possible, pending final FDA approval."

Read more: https://prn.to/2zQ3BNe

Mylan Wins District Court Decision Against Biogen's Tecfidera® Patent

/PRNewswire/ -- Mylan N.V. (NASDAQ: MYL) today announced that the U.S. District Court for the Northern District of West Virginia invalidated Biogen's...

Armata Pharmaceuticals, Inc., a biotechnology company focused on precisely targeted bacteriophage therapeutics for antibiotic-resistant and difficult-to-treat bacterial infections, today announced that it has received a $15 million award for a three year program from the U.S. DoD through the Medical Technology Enterprise Consortium (MTEC) with funding from the Defense Health Agency and Joint Warfighter Medical Research Program. Armata will use the award to partially fund a Phase 1b/2, randomized, double-blind, placebo-controlled, dose escalation clinical study of Armata's therapeutic phage-based candidate, AP-SA02, for the treatment of complicated Staphylococcus aureus bacteremia infections.

"Today, I am pleased to announce that we have achieved our goal of receiving non-dilutive funding to support clinical development of our optimized phage candidate, AP-SA02, as a promising potential treatment for S. aureus bacteremia. We are excited to have exceeded the amount of funding we had originally targeted, which enables us to robustly examine the potential efficacy of our optimized phage candidate," stated Todd R. Patrick, Chief Executive Officer of Armata Pharmaceuticals. "This funding from the DoD validates the potential of phage-based therapeutics and helps us move AP-SA02 into clinical development while continuing to carefully manage our financial position. Drug-resistant S. aureus bacteremia infections carry mortality rates as high as 40%, reflecting the urgent need for novel and improved treatment options."

Read more: https://prn.to/3dbEdPk

Armata Pharmaceuticals Announces $15 Million Award from the U.S. Department of Defense (DoD) for Development of Bacteriophage Therapy to Treat S. aureus Bacteremia Infections

/PRNewswire/ -- Armata Pharmaceuticals, Inc. (NYSE American: ARMP) ("Armata" or the "Company"), a biotechnology company focused on precisely targeted...

Celltrion Healthcare has partnered with the University of Oxford and University Hospitals Birmingham to investigate the use of infliximab biosimilar CT-P13 as a treatment for COVID-19 symptoms.
As severe COVID-19 is associated with a major immune inflammatory response, researchers are testing the theory that therapies targeting this could also be used as a treatment for COVID-19.
Infliximab is an anti-TNF therapy designed to attach to TNF, a protein important in inflammation, and is currently used as a treatment for other inflammatory conditions.
“Using a treatment which is effective by blocking TNF in more than 10 inflammatory diseases to treat inflammation associated with COVID-19, in which levels of TNF are also high, makes a lot of sense,” said Sir Marc Feldmann, Professor of Immunology, University of Oxford, one of the inventors of infliximab.
“If treated with infliximab upon admission to hospital, the number of patients who may need to be admitted to intensive care may be reduced.”

https://bit.ly/2USZI1k

Celltrion's infliximab biosimilar to be tested for COVID-19 - PharmaTimes

Infliximab has been chosen because of its effective role in treating autoimmune and inflammatory conditions and established safety profile - News - PharmaTimes

AstraZeneca has agreed with Europe’s Inclusive Vaccines Alliance (IVA) that it will supply up to 400 million doses of the University of Oxford’s COVID-19 vaccine to broaden access in the region.
The deal, which will see deliveries start by the end of this year, allows the IVA aims to accelerate the supply of the vaccine and to make it available to other European countries that wish to participate in the initiative.
“This agreement will ensure that hundreds of millions of Europeans have access to Oxford University’s vaccine following approval,” said Pascal Soriot, AZ' chief executive.

Read more: http://www.pharmatimes.com/news/az_signs_deal_with_europes_iva_for_coronavirus_vaccine_supply_1342267

Koura (previously known as Mexichem Fluor), a pioneer in the development of next generation propellants, today announced FDA approval to proceed to clinical trials with Zephex® 152a. This new medical propellant has been under development for several years for use in metered-dose inhalers (MDIs) for treatment of respiratory disorders such as asthma and chronic obstructive pulmonary disease (COPD). Zephex® 152a reduces the carbon footprint of an MDI, helping to safeguard an invaluable therapeutic option for patients.
Koura (previously known as Mexichem Fluor), a pioneer in the development of next generation propellants, today announced FDA approval to proceed to clinical trials with Zephex® 152a. This new medical propellant has been under development for several years for use in metered-dose inhalers (MDIs) for treatment of respiratory disorders such as asthma and chronic obstructive pulmonary disease (COPD). Zephex® 152a reduces the carbon footprint of an MDI, helping to safeguard an invaluable therapeutic option for patients.

Read more: https://prn.to/3baPtvS

Researchers at the National Institutes of Health (NIH) have shown that inhibition of myeloid-derived suppressor cell (MDSC) trafficking into mouse solid tumors by SX-682 enhanced both PD-1 immune checkpoint blockade (ICB) and T cell receptor (TCR) engineered T cell immunotherapy, leading to significant tumor growth inhibition or eradication. The research, published last week in JCI Insight, suggests a promising new approach for treating patients with solid tumors using a combination of SX-682 and multiple forms of immunotherapy including ICB and adoptive cell transfer with engineered T cells. The results arose from a Cooperative Research and Development Agreement (CRADA) between Syntrix and NIH.

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

Indivior PLC today announced data from two studies evaluating efficacy and long-term safety of once-monthly SUBLOCADE™ (buprenorphine extended-release) injection for subcutaneous use (CIII) for the treatment of moderate to severe opioid use disorder.
• The RECOVER™ study found that 75 percent of patients who had received 12 once-monthly doses of SUBLOCADE in a Phase 3 clinical trial were abstinent from illicit opioids for a year after the study ended. In this observational study, abstinence was defined as self-report confirmed by urine drug screen. Some patients had the option to continue SUBLOCADE following the clinical trial or chose to continue another form of medication-assisted treatment during the RECOVER study period.
• An 18-month safety study among SUBLOCADE-treated patients reported no new safety issues.

SUBLOCADE is approved by the U.S. Food and Drug Administration (FDA) for the treatment of moderate to severe OUD in adult patients who have initiated treatment with a transmucosal buprenorphine-containing product, followed by dose adjustment for a minimum of seven days.

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

Luye Pharma Group has announced submission of a new drug application (NDA) to the U.S. Food and Drug Administration ("FDA") for Rykindo®, completed on March 28. Rykindo® (LY03004) Risperidone Extended-release Microsphere for Injection is expected to become the first Chinese innovative drug to receive U.S. FDA approval for marketing in the United States.
The NDA submission for Rykindo® is regarded as a milestone step for the company, expecting a big pay-off from the potential industrialization of its long-invested long-acting and extended-release technology R&D platform. This is not only a key step in Luye Pharma's globalization initiative, but also a major step for China in bringing innovative formulations to the world, receiving wide attention from all walks of life.
Rykindo® is an extended-release microsphere independently developed by Luye Pharma. It is administered once every two weeks by intramuscular injection to treat schizophrenia and bi-polar disorder.

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

Johnson & Johnson Vision today announced that results from two Phase 3 clinical studies evaluating the company's investigational antihistamine-releasing contact lens (etafilcon A with 0.019 mg ketotifen) were published in the journal Cornea. The results demonstrated that patients wearing the investigational contact lens had lower mean itching scores after their eyes were exposed to allergens compared to those wearing non-medicated control lenses, indicating that in these studies, the lenses reduced symptoms of eye itch.
The antihistamine-releasing contact lenses worn in the studies are daily disposable lenses containing ketotifen, a drug that inhibits certain substances in the body that are known to cause allergic reactions and inflammation. They were tested in two multicenter, double-masked, randomized, placebo-controlled trials that included 244 patients and used the conjunctival allergen challenge model (Ora-CAC®) to measure efficacy.

Read more: https://prn.to/2CEAc6O

Actelion Pharmaceuticals US, Inc., one of the Janssen Pharmaceutical Companies of Johnson & Johnson, announced today an interim analysis from the Right vEntricular Remodeling in Pulmonary ArterIal hypeRtension (REPAIR) study of OPSUMIT® (macitentan) that shows treatment with OPSUMIT in patients with pulmonary arterial hypertension (PAH) was associated with significant improvements in right ventricular (RV) function including reversal of RV remodeling and reduced pulmonary vascular resistance (PVR). The data are being presented at the American College of Cardiology's 68th Annual Scientific Session.

Pulmonary arterial hypertension (PAH) is a chronic, life-threatening disorder characterized by abnormally high blood pressure in the arteries between the heart and lungs of an affected individual. The symptoms of PAH are non-specific and can range from mild breathlessness and fatigue during normal daily activity to symptoms of right heart failure and severe restrictions on exercise capacity and ultimately reduced life expectancy.

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

Today, AARP announced the launch of 'Stop Rx Greed,' a nationwide campaign aimed at lowering prescription drug prices. The goal of AARP's sustained campaign is to help drive down drug prices for all Americans by advocating for a variety of legislative, executive, and regulatory actions at both the federal and state level.
"Americans are paying the highest prescription drug prices in the world," said Executive Vice President and Chief Advocacy & Engagement Officer Nancy LeaMond. "It's time for pharmaceutical companies to stop deflecting blame and ac

The Stop Rx Greed campaign will include national television, radio and digital ads, editorial content, emails to members, social media posts, ongoing advocacy and grassroots activity in D.C. and the states, and a petition calling on Congress and the Administration to take action now.

Read more: https://prn.to/2J7AAQT

AnTolRx, Inc., a private biotechnology company and a leader in immune tolerance, today announced that Pfizer Inc. has exercised the option granted under a June 2016 agreement to license an immune tolerance therapy from AnTolRx's Type 1 diabetes research program.
The option gives Pfizer exclusive rights to pursue development of AnTolRx's lead immunotherapeutic for Type 1 diabetes. Pfizer will be responsible for any further optimization, development and potential commercialization. AnTolRx will receive an upfront payment and is eligible to receive potential milestone and royalty payments from Pfizer.
AnTolRx is aiming to develop targeted antigen-specific therapies, based upon immune tolerance induction rather than broader immune suppression, for several inflammatory and autoimmune diseases.

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

Enzychem Lifesciences, Corp. announced that the company has achieved a huge milestone by completing enrollment of the required 24 patients in Stage 1 of the Phase 2 CRIOM (Chemoradiation Induced Oral Mucositis) study.
Stage 1 of the Phase 2 CRIOM study evaluates the maximum tolerable dose of EC-18 which will be used in Stage 2 of the study. Patient enrollment for Stage 2 is expected to begin in 2Q 2019. Enzychem has received Fast Track Designation by the U.S. Food and Drug Administration for the Phase 2 study evaluating lead investigational candidate EC-18 in CRIOM.
Enzychem Lifesciences, Corp. (KOSDAQ:183490) is a global biopharmaceutical company headquartered in Seoul, Korea, which is dedicated to developing new drugs and APIs (Active Pharmaceutical Ingredients). Since its foundation in 1999, Enzychem Lifesciences has been striving to develop new and innovative treatments capable of addressing unmet medical needs with R&D-driven approaches under its grand mission of "Saving Human Lives by Overcoming Unmet Medical Needs"

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

Amgen and Servier today announced that METEORIC-HF (Multicenter Exercise Tolerance Evaluation of Omecamtiv Mecarbil Related to Increased Contractility in Heart Failure), the second Phase 3 clinical trial of omecamtiv mecarbil, a novel cardiac myosin activator, is open to enrollment. Omecamtiv mecarbil is being developed for the potential treatment of heart failure with reduced ejection fraction (HFrEF).

Omecamtiv mecarbil is a novel, selective cardiac myosin activator that binds to the catalytic domain of myosin. Preclinical research has shown that cardiac myosin activators increase cardiac contractility without affecting intracellular myocyte calcium concentrations or myocardial oxygen consumption.1,2,3 Cardiac myosin is the cytoskeletal motor protein in the cardiac muscle cell that is directly responsible for converting chemical energy into the mechanical force resulting in cardiac contraction. Omecamtiv mecarbil is being developed for the potential treatment of heart failure with reduced ejection fraction (HFrEF) under a collaboration between Amgen and Cytokinetics, with funding and strategic support from Servier.

Read more: https://prn.to/2SgL143

The biotech drug industry, with encouragement from the Food and Drug Administration (FDA), is advancing efforts to modernize clinical trials, streamlining the process of figuring out whether a treatment actually works. "Clinical trials are getting more complex and increasing amounts of data are being collected," says Steve Cutler, chief operating officer of clinical research organization ICON. "We need better approaches and we need to apply our current systems and technology better in order to meet the challenges posed by this – such as increasing numbers of patient subgroups, difficulties in finding those patients, and trials in rare diseases and orphan drugs becoming more common." Advances in genetic testing now allow physicians and researchers to offer methods of identifying and treating cancer that were not considered, or even understood, not that long ago.

Read more: https://prn.to/2t972Y6

Takeda Pharmaceutical Company Limited today announced that it will feature 12 presentations, including 11 posters and one oral presentation, at the 15th annual WORLDSymposium™ 2019 in Orlando, Florida, February 4-8. Presentations and other company activities will focus on its research and development efforts in lysosomal storage disorders (LSDs) including Hunter syndrome (also known as Mucopolysaccharidosis type II or MPS II), type 1 Gaucher disease, Fabry disease and metachromatic leukodystrophy (also known as MLD).

The company will also sponsor a satellite symposium on the role of biomarkers in clinical management of lysosomal diseases, and a booth (Booth #105) in the WORLDSymposium exhibit hall.

The upcoming WORLDSymposium annual meeting focuses on the latest scientific and clinical research on LSDs, a collection of some 50 disorders caused by specific enzyme deficiencies and leading to significant disability and disease burden.

Read more: https://prn.to/2RDBX94

The Janssen Pharmaceutical Companies of Johnson & Johnson announced a worldwide collaboration and license agreement with MeiraGTx Holdings plc, a clinical-stage gene therapy company, to develop, manufacture and commercialize its clinical stage inherited retinal disease portfolio, including leading product candidates for achromatopsia (ACHM) caused by mutations in either CNGB3 or CNGA3, and X-linked retinitis pigmentosa (XLRP). Further, the companies have formed a research collaboration to explore new targets for other inherited retinal diseases and further develop adeno-associated virus (AAV) manufacturing technology.
Achromatopsia is an inherited retinal disease that prevents cone photoreceptors from functioning. Patients are legally blind from birth and usually suffer from severely reduced visual acuity of 20/200 or worse; a disabling sensitivity to light, or photophobia; total color blindness; and involuntary back and forth eye movements, or nystagmus. There are currently no approved treatments for achromatopsia.

Read more: https://prn.to/2Gg8f7L