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Clinical Trial for the Use of Nitric Oxide to Treat Severe COVID-19 Infection

Reporter and Curator: Aviva Lev-Ari, PhD, RN

 

UPDATED 5/26/2020

2009 Dec 5;395(1):1-9.

doi: 10.1016/j.virol.2009.09.007. Epub 2009 Oct 1.

Dual Effect of Nitric Oxide on SARS-CoV Replication: Viral RNA Production and Palmitoylation of the S Protein Are Affected

Affiliations expand

Free PMC article

Abstract

Nitric oxide is an important molecule playing a key role in a broad range of biological process such as neurotransmission, vasodilatation and immune responses. While the anti-microbiological properties of nitric oxide-derived reactive nitrogen intermediates (RNI) such as peroxynitrite, are known, the mechanism of these effects are as yet poorly studied. Severe Acute Respiratory Syndrome coronavirus (SARS-CoV) belongs to the family Coronaviridae, was first identified during 2002-2003. Mortality in SARS patients ranges from between 6 to 55%. We have previously shown that nitric oxide inhibits the replication cycle of SARS-CoV in vitro by an unknown mechanism. In this study, we have further investigated the mechanism of the inhibition process of nitric oxide against SARS-CoV. We found that peroxynitrite, an intermediate product of nitric oxide in solution formed by the reaction of NO with superoxide, has no effect on the replication cycle of SARS-CoV, suggesting that the inhibition is either directly effected by NO or a derivative other than peroxynitrite. Most interestingly, we found that NO inhibits the replication of SARS-CoV by two distinct mechanisms.

  • Firstly, NO or its derivatives cause a reduction in the palmitoylation of nascently expressed spike (S) protein which affects the fusion between the S protein and its cognate receptor, angiotensin converting enzyme 2.
  • Secondly, NO or its derivatives cause a reduction in viral RNA production in the early steps of viral replication, and this could possibly be due to an effect on one or both of the cysteine proteases encoded in Orf1a of SARS-CoV.

 

UPDATED ON 4/21/2020

A Possible Explanation for the COVID-19 Racial Disparity

— And a possible solution

While the pathophysiology of hypertension is complex and multifaceted, there are notable racial differences. In the context of COVID-19, the most suspicious difference is a comparative deficiency of L-arginine and subsequently nitric oxide (NO). In this lies a potential explanation for the COVID-19 race disparity

NO is a gas synthesized by our cells and has multiple roles, but perhaps is best known for vascular dilation. In short, NO facilitates relaxation of vascular smooth muscle allowing vessel dilation and increased blood flow.

This on its own has potential implications in acute respiratory distress syndrome (ARDS), a condition that results from severe COVID-19 infection. By improving blood flow across the entire lung, this theoretically results in improved gas exchange and oxygenation of the blood. In fact, there is research that inhaled NO improved oxygenation and other clinical outcomes in SARS-1 patients, and current research in COVID-19 coronavirus (SARS-CoV-2) supports this previously demonstrated efficacy.

Additionally, abnormal blood clotting is an increasingly recognized complication of this disease, both systemically and within the pulmonary circulation. In fact, one of the greatest predictors of death is a serum blood test that indicates elevated clotting activity. Most recently, some physicians have suggested that small clots within the lungs are central to pathogenesis and have administered clot busting drugs known as thrombolytics which abruptly improve oxygenation, albeit transiently, as the medication effect weans and the predisposition to clot formation persists. NO inhibits clot formation, and deficiency may contribute to a prothrombotic state. In fact, it has been shown that inhaled NO decreases the propensity of clotting in ARDS.

However, perhaps the most convincing role of nitric oxide in this disease is its antiviral properties. SARS-CoV-2 infects cells by attaching to a receptor on the lining of the airways called angiotensin-converting enzyme 2 (ACE2). This is the same mechanism by which SAR-1 infects cells. NO specifically alters a surface protein on SARS-1, known as the spike protein, such that it cannot attach to the ACE2 receptor. This results in blocking viral entry into the cell as well as the subsequent replication of the virus. Since SARS-CoV-2 shares the same mechanism of cell entry, we can relatively confidently assume that NO would have a similar effect regarding this novel virus.

Knowing that NO deficiency is common in African Americans and that this population is disproportionately dying from an infection that can be blocked by this gas, augmenting NO seems like a reasonable therapeutic target. While NO is being used as an inhaled gas via mechanical ventilation, this is only suitable for someone ill enough to require mechanical ventilation.

A better way to increase nitric oxide in the minimally ill or even uninfected is to augment the body’s ability to create it. There are many pharmacologic ways to do this; however, potentially the most effective, cheapest, and lowest risk is to supplement with the precursor amino-acids L-arginine and L-citrulline. We already know these nutritional supplements result in this very effect and that there seems to be a more potent effect of supplementation on NO production in L-arginine-deficient African Americans.

Therefore, a reasonable action is to expedite clinical trials to further investigate this theory. At a minimum, we need to start a conversation to improve our understanding of the role of nitric oxide deficiency as a risk factor for disease severity. It is my strong belief that augmenting NO via L-arginine and L-citrulline not only has potential for treatment and reducing progression to severe illness, but given the safety profile, it may be most valuable as a preventative measure.

It could save many lives at a minimal cost.

Jason Kidde, MS, MPAS, is a physician assistant at University of Utah Health in Salt Lake City.

Last Updated April 21, 2020
SOURCE

 

Previous research found nitric oxide has antiviral properties against coronaviruses.

ummary: A new clinical trial is enrolling patients with severe COVID-19 symptoms to assess the effect of nitric oxide in treating the virus. Previous research found nitric oxide has antiviral properties against coronaviruses. The effect was tested and demonstrated during the SARS outbreak in the early 2000s.

Source: University of Alabama at Birmingham

The University of Alabama at Birmingham has been selected to begin enrolling patients in an international study assessing the use of inhaled nitric oxide (iNO) to improve outcomes for COVID-19 patients with severely damaged lungs.

iNO has been used for the treatment of failing lungs, but it was also found to have antiviral properties against coronaviruses

“In humans, nitric oxide is generated within the blood vessels and regulates blood pressure, and prevents formation of clots and also destroys potential toxins,” Arora said.

The UAB team says this pandemic has led to an extraordinary unifying response by the medical community, including ICU physicians, nurses, respiratory therapists, clinical trial specialists, reviewers and medical administrators, allowing for faster than normal approvals for potentially lifesaving research studies.

“The fact that we are able to get this trial started quickly was due to collaborations across specialties and fields of expertise at UAB with the common goal of providing the highest quality of scientifically proven care for our COVID-19 patients,” Arora said. “We are all trying to fight this together, and I hope, with our resilience, we shall overcome these difficult times.”

SOURCE
Source:
University of Alabama at Birmingham
Media Contacts:
Adam Pope – University of Alabama at Birmingham
Image Source:
The image is credited to University of Alabama at Birmingham.

Other related articles published in this Open Access Online Scientific Journal include the following:

  • Clinical Indications for Use of Inhaled Nitric Oxide (iNO) in the Adult Patient Market: Clinical Outcomes after Use of iNO in the Institutional Market, Therapy Demand and Cost of Care vs. Existing Supply Solutions

Curator: Aviva Lev-Ari, PhD, RN

https://pharmaceuticalintelligence.com/2013/06/03/clinical-indications-for-use-of-inhaled-nitric-oxide-ino-in-the-adult-patient-market-clinical-outcomes-after-use-therapy-demand-and-cost-of-care/

 

Series A: e-Books on Cardiovascular Diseases

 

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Six Volumes

  1. Cardiovascular Diseases, Volume One: Perspectives on Nitric Oxide in Disease Mechanisms. On com since 6/21/2013 https://lnkd.in/8DANfq
  2. Cardiovascular Diseases, Volume Two: Cardiovascular Original Research: Cases in Methodology Design for Content Co-Curation. On com since 11/30/2015 https://lnkd.in/ekbuNZ3
  3. Cardiovascular Diseases, Volume Three: Etiologies of Cardiovascular Diseases: Epigenetics, Genetics and Genomics. On com since 11/29/2015 https://lnkd.in/ecp5mrA
  4. Cardiovascular Diseases, Volume Four: Regenerative and Translational Medicine: The Therapeutics Promise for Cardiovascular Diseases. On com since 12/26/2015 https://lnkd.in/dwqM3K3
  5. Cardiovascular Diseases, Volume Five: Pharmacological Agents in Treatment of Cardiovascular Diseases. On com since 12/23/2018 https://lnkd.in/e3r87cQ
  6. Cardiovascular Diseases, Volume Six: Interventional Cardiology for Disease Diagnosis and Cardiac Surgery for Condition Treatment. On com since 12/24/2018 https://lnkd.in/e_CTb4R

  • Cardiovascular Diseases, Volume One: Perspectives on Nitric Oxide in Disease Mechanisms. On Amazon.com since 6/21/2013

http://www.amazon.com/dp/B00DINFFYC

Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1) by [Margaret Baker PhD, Tilda Barliya PhD, Anamika Sarkar PhD, Ritu Saxena PhD, Stephen J. Williams PhD, Larry Bernstein MD FCAP, Aviva Lev-Ari PhD RN, Aviral Vatsa PhD]

Perspectives on Nitric Oxide in Disease Mechanisms (Biomed e-Books Book 1) Kindle Edition

Table of Contents

Chapter 1:

Nitric Oxide Basic Research

1.1 Discovery of Nitric Oxide

1.1.1 Discovery of Nitric Oxide and its Role in Vascular Biology

Aviral Vatsa, PhD, MBBS

1.1.2 Nitric Oxide: The Nobel Prize in Physiology or Medicine

Aviva Lev-Ari, PhD, RN

1.2 Nitric Oxide Synthase(s)

1.2.1 Nitric Oxide: A Short Historic Perspective

Aviral Vatsa, PhD, MBBS

1.2.2 Nitric Oxide: Role in Cardiovascular Health and Disease

Aviral Vatsa, PhD, MBBS

1.3 Endothelial Blood Cell Interactions: Platelet, Leukocyte and Monocyte

1.3.1 Nitric Oxide: Chemistry and Function

Aviral Vatsa, PhD, MBBS

1.4 Signaling Pathways

1.4.1 Nitric Oxide Signaling Pathways

Aviral Vatsa, PhD, MBBS

1.4.2 Nitric Oxide has a Ubiquitous Role in the Regulation of Glycolysis – with a Concomitant Influence on Mitochondrial Function

Larry H. Bernstein, MD, FCAP

1.5 Oxidative Stress

1.5.1 Mitochondrial Damage and Repair under Oxidative Stress

Larry H. Bernstein, MD, FCAP

1.6 Oxygen and Nitrogen Reactive Species

1.6.1 Interaction of Nitric Oxide and Prostacyclin in Vascular Endothelium

Larry H Bernstein, MD, FCAP

1.6.2 Prostacyclin and Nitric Oxide: Adventures in vascular biology –  a tale of two mediators

Aviva Lev-Ari, PhD, RN

 

Chapter 2:

Nitric Oxide and Circulatory Diseases

2.1 Endothelial Dysruption and Denudation

2.1.1 Blood-vessels-generating Stem Cells Discovered

Ritu Saxena, PhD

2.1.2 Differential Distribution of Nitric Oxide – A 3-D Mathematical Model

Anamika Sarkar, PhD

2.1.3 Nitric Oxide Nutritional Remedies for Hypertension and Atherosclerosis. It’s 12AM: Do you know where your electrons are?

Meg Baker, PhD

2.2 Endothelin and ET Receptors

2.2.1 Statins’ Nonlipid Effects on Vascular Endothelium through eNOS Activation

Larry H Bernstein, MD, FCAP

2.2.2 Endothelial Function and Cardiovascular Disease

Larry H Bernstein, MD, FCAP

2.2.3 Endothelin Receptors in Cardiovascular Diseases: The Role of eNOS Stimulation: Observations on Intellectual Property Development for an Unrecognized Future Fast Acting Therapy for Patients at High Risk for Macrovascular Events

Aviva Lev-Ari, PhD, RN

Chapter 3:

Therapeutic Cardiovascular Targets

3.1 Nitric oxide and therapeutic Targets

3.1.1 Cardiovascular Disease (CVD) and the Role of Agent Alternatives in Endothelial Nitric Oxide Synthase (eNOS) Activation and Nitric Oxide Production

Aviva Lev-Ari, PhD, RN

3.1.2 Telling NO to Cardiac Risk

Stephen W Williams, PhD

3.1.3 Nitric Oxide and its Impact on Cardiothoracic Surgery

Tilda Barliya PhD

3.2 Therapeutic opportunities for Endothelial Progenitor Cells

3.2.1 Inhibition of ET-1, ETA and ETA-ETB, Induction of Nitric Oxide production, stimulation of eNOS and Treatment Regime with PPAR-gamma agonists (TZD): eEPCs Endogenous Augmentation for Cardiovascular Risk Reduction – A Bibliography

Aviva Lev-Ari, PhD, RN

3.2.2 Bystolic’s generic Nebivolol – Positive Effect on circulating Endothelial Progenitor Cells Endogenous Augmentation

Aviva Lev-Ari, PhD, RN

3.2.3 Positioning a Therapeutic Concept for Endogenous Augmentation of cEPCs — Therapeutic Indications for Macrovascular Disease: Coronary, Cerebrovascular and Peripheral

Aviva Lev-Ari, PhD, RN

3.2.4 Endothelial Dysfunction, Diminished Availability of cEPCs, Increasing CVD Risk for Macrovascular Disease – Therapeutic Potential of cEPCs

Aviva Lev-Ari, PhD, RN

3.3 Hypertension, Congestive Heart Failure and Endothelin Biomarker

3.3.1 Clinical Trials Results for Endothelin System: Pathophysiological Role in Chronic Heart Failure, Acute Coronary Syndromes and MI – Markers of Disease Severity or Genetic Determination?

Aviva Lev-Ari, PhD, RN

3.4 Hypotension and Shock: Cardiovascular Collapse

3.4.1 Nitric Oxide and Sepsis, Hemodynamic Collapse and the Search for Therapeutic Options

Larry H Bernstein, MD, FCAP

3.4.2 Sepsis, Multi-organ Dysfunction Syndrome, and Septic Shock: A Conundrum of Signaling Pathways Cascading Out of Control

Larry H Bernstein, MD, FCAP

3.5 Hemorrhagic and Thrombo-embolic Events

3.5.1 Nitric Oxide Function in Coagulation

Larry H Bernstein, MD, FCAP

Chapter 4:

Nitric Oxide and Neurodegenerative Diseases

4.1 Nitric Oxide Covalent Modifications: A Putative Therapeutic Target?

Stephen J. Williams, PhD

Chapter 5:

Bone Metabolism

5.1 Nitric Oxide in Bone Metabolism

Aviral Vatsa, PhD, MBBS

Chapter 6:

Nitric Oxide and Systemic Inflammatory Disease

6.1 Nitric Oxide and Immune Responses: Part 1

Aviral Vatsa, PhD, MBBS

6.2 Nitric Oxide and Immune Responses: Part 2

Aviral Vatsa, PhD, MBBS

6.3 Nitric Oxide Production in Systemic Sclerosis

Aviral Vatsa, PhD. MBBS

Chapter 7:

Nitric Oxide: Lung and Alveolar Gas Exchange

7.1 ’Lung on a Chip’

Ritu Saxena, Ph.D.

7.2 Low Bioavailability of Nitric Oxide due to Misbalance in Cell Free Hemoglobin in Sickle Cell Disease – A Computational Model

Anamika Sarkar, Ph.D.

7.3 The Rationale and Use of Inhaled Nitric Oxide in Pulmonary Artery Hypertension and Right Sided Heart Failure

Larry H Bernstein, MD, FCAP

7.4 Transposon-mediated Gene Therapy improves Pulmonary Hemodynamics and attenuates Right Ventricular Hypertrophy: eNOS gene therapy reduces Pulmonary vascular remodeling and Arterial wall hyperplasia

Aviva Lev-Ari, PhD, RN

Chapter 8:

Nitric Oxide and Kidney Dysfunction

8.1 Part I: The Amazing Structure and Adaptive Functioning of the Kidneys: Nitric Oxide

Larry H. Bernstein, MD, FCAP

8.2 Part II: Nitric Oxide and iNOS have Key Roles in Kidney Diseases

Larry H. Bernstein, MD, FCAP

8.3 Part III: The Molecular Biology of Renal Disorders: Nitric Oxide

Larry H. Bernstein, MD, FCAP

8.4 Part IV: New Insights on Nitric Oxide Donors

Larry H. Bernstein, MD, FCAP

8.5 The Essential Role of Nitric Oxide and Therapeutic Nitric Oxide Donor Targets in Renal Pharmacotherapy

Larry H. Bernstein, MD, FCAP

Chapter 9:

Nitric Oxide and Cancer

9.1 Crucial role of Nitric Oxide in Cancer

Ritu Saxena, Ph.D.

Summary

Nitric oxide and its role in vascular biology

 

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Injectable inclisiran (siRNA) as 3rd anti-PCSK9 behind mAbs Repatha and Praluent

 

Reporter: Aviva Lev-Ari, PhD, RN

Next stop, filing for approval. The Medicines Company has said it plans to submit inclisiran for FDA review by the end of 2019 and EMA review in the first quarter of 2020. If the drug’s approved it’ll be the third anti-PCSK9 behind mAbs Repatha and Praluent, and could try to compete on price to gain market share.

The company’s been very careful not to disclose its pricing plans for inclisiran, said ORION-10 principal investigator Dr. Scott Wright, professor and cardiologist at the Mayo Clinic. But, Wright said, The Medicines Co. and other companies he advises on clinical trial design “have learned the lesson from the sponsors of the monoclonal antibodies [against PCSK9], they’re not going to come in and price a drug that’s out of proportion to what the market will bear.” 

Because the anti-PCSK9 mAbs were initially priced beyond what patients and insurers were willing to pay, “now most of the physicians that I meet have a resistance to using them just because they’re fearful about the pre-approval process” with insurers, said Wright. “They’re much easier to get approved and paid for today than they’ve ever been, but that message is not out in the medical community yet.”

SOURCE

From: “STAT: AHA in 30 Seconds” <newsletter@statnews.com>

Reply-To: “STAT: AHA in 30 Seconds” <newsletter@statnews.com>

Date: Monday, November 18, 2019 at 2:59 PM

To: Aviva Lev-Ari <AvivaLev-Ari@alum.berkeley.edu>

Subject: Interim look at Amarin data, an inclisiran update, & Philly’s giant heart

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Transthyretin amyloid cardiomyopathy (ATTR-CM): U.S. FDA APPROVES VYNDAQEL® AND VYNDAMAX™ for this Rare and Fatal Disease

 

Reporter: Aviva Lev-Ari, PhD, RN

UPDATED on 11/22/2019

Trialists Attack $225K Heart Drug Price Tag

Cardiologists who helped run the pivotal study of Pfizer’s heart drug tafamidis (Vyndaqel/Vyndamax) are criticizing the drug’s $225,000 annual price tag, Bloomberg reports.

Mathew Maurer, MD, of Columbia University, and three other doctors involved in the trial started speaking out after seeing patients’ financial struggles after the drug’s market launch earlier this year.

For example: John Rufenacht, a 73-year-old interior designer in Kansas City, Missouri, has Medicare but his out-of-pocket cost was $6,000 for a 90-day supply of the drug, which treats cardiac transthyretin amyloidosis. Rufenacht doesn’t qualify for Pfizer’s patient assistance programs, most of which direct patients to charities to help them pay.

Maurer aired his complaints in front of colleagues at the Heart Failure Society of America meeting in September, and at the American Heart Association meeting earlier this week, where he and colleagues reported a cost-effectiveness study on the drug, showing it’s only cost-effective with a more than 90% price reduction — a cost of $16,563 a year.

Pfizer says its price is appropriate, given the small number of patients in the U.S. with the condition who will receive it — some 100,000 to 150,000, the company estimates. But Maurer and critics say that’s likely an underestimate. Diagnosis requires an invasive heart biopsy; there was little incentive to do that when no approved treatment was available.

The company promised to cut the price if more patients start taking the drug.

SOURCE

https://www.medpagetoday.com/publichealthpolicy/ethics/83459?xid=nl_badpractice_2019-11-22&eun=g99985d0r&utm_source=Sailthru&utm_medium=email&utm_campaign=BadPractice_112219&utm_term=NL_Gen_Int_Bad_Practice%20-%20Active

 

Click here to learn more about Pfizer’s Rare Disease portfolio and how we empower patients, engage communities in our clinical development programs, and support programs that heighten disease awareness.

 

U.S. FDA APPROVES VYNDAQEL® AND VYNDAMAX™ FOR USE IN PATIENTS WITH TRANSTHYRETIN AMYLOID CARDIOMYOPATHY, A RARE AND FATAL DISEASE

— First and only medicines approved for patients with either wild-type or hereditary transthyretin amyloid cardiomyopathy —

Monday, May 6, 2019 – 6:45am
EDT

NEW YORK–(BUSINESS WIRE)–Pfizer Inc. (NYSE:PFE) announced today that the U.S. Food and Drug Administration (FDA) has approved both VYNDAQEL® (tafamidis meglumine) and VYNDAMAX (tafamidis) for the treatment of the cardiomyopathy of wild-type or hereditary transthyretin-mediated amyloidosis (ATTR-CM) in adults to reduce cardiovascular mortality and cardiovascular-related hospitalization. VYNDAQEL and VYNDAMAX are two oral formulations of the first-in-class transthyretin stabilizer tafamidis, and the first and only medicines approved by the FDA to treat ATTR-CM.

Transthyretin amyloid cardiomyopathy is a rare, life-threatening disease characterized by the buildup of abnormal deposits of misfolded protein called amyloid in the heart and is defined by restrictive cardiomyopathy and progressive heart failure. Previously, there were no medicines approved to treat ATTR-CM; the only available options included symptom management, and, in rare cases, heart (or heart and liver) transplant. It is estimated that the prevalence of ATTR-CM is approximately 100,000 people in the U.S. and only one to two percent of those patients are diagnosed today.

“The approvals of VYNDAQEL and VYNDAMAX are a testament to the significant research and development investment in our innovative cardiovascular outcomes trial, ATTR-ACT. We are proud to bring these medicines to ATTR-CM patients who are in dire need of treatment,” said Brenda Cooperstone, MD, Senior Vice President and Chief Development Officer, Rare Disease, Pfizer Global Product Development. “VYNDAQEL and VYNDAMAX reduce cardiovascular mortality and the frequency of cardiovascular-related hospital stays in patients with wild-type or hereditary forms of this rare disease, giving them a chance for more time with their loved ones.”

“Pfizer’s purpose is to deliver breakthrough medicines that change patients’ lives. The approvals of VYNDAQEL and VYNDAMAX deliver on this promise for patients with ATTR-CM,” said Paul Levesque, Global President, Rare Disease. “This milestone is a gamechanger for patients, who until today had no approved medicines for this rare, debilitating and fatal disease. We will continue to focus efforts on working with the physician community to increase awareness and ultimately detection and diagnosis of this disease.”

The recommended dosage is either VYNDAQEL 80 mg orally once-daily, taken as four 20 mg capsules, or VYNDAMAX 61 mg orally once-daily, taken as a single capsule. VYNDAMAX was developed for patient convenience; VYNDAQEL and VYNDAMAX are not substitutable on a per milligram basis.

“ATTR-CM is not only fatal, but also significantly underdiagnosed, with some patients cycling through multiple doctors and a myriad of tests over a period of years while the disease progresses,” said Isabelle Lousada, Founder and CEO, Amyloidosis Research Consortium. “ATTR-CM is a rare disease for which more education and awareness is needed. The approval of these medicines represents an important advance for patients; however, it is equally important that we work as a community to recognize the critical importance of early diagnosis.”

The FDA approval was based on data from the pivotal Phase 3 Transthyretin Amyloidosis Cardiomyopathy Clinical Trial (ATTR-ACT), the first global, double-blind, randomized, placebo-controlled clinical study to investigate a pharmacological therapy for the treatment of this disease. In ATTR-ACT, VYNDAQEL significantly reduced the hierarchical combination of all-cause mortality and frequency of cardiovascular-related hospitalizations compared to placebo over a 30-month period (p=0.0006). Additionally, individual components of the primary analysis demonstrated a relative reduction in the risk of all-cause mortality and frequency of cardiovascular-related hospitalization of 30% (p=0.026) and 32% (p<0.0001), respectively, with VYNDAQEL versus placebo. Approximately 80% of total deaths were cardiovascular-related in both treatment groups. VYNDAQEL also had significant and consistent treatment effects compared to placebo on functional capacity and health status first observed at six months and continuing through 30 months. Specifically, VYNDAQEL reduced the decline in performance on the six-minute walk test (p<0.0001) and reduced the decline in health status as measured by the Kansas City Cardiomyopathy Questionnaire – Overall Summary score (p<0.0001). VYNDAQEL was well tolerated in this study, with an observed safety profile comparable to placebo. The frequency of adverse events in patients treated with VYNDAQEL was similar to placebo, and similar proportions of VYNDAQEL-treated patients and placebo-treated patients discontinued the study drug because of an adverse event.

Pfizer is committed to helping eligible ATTR-CM patients who have been prescribed VYNDAQEL or VYNDAMAX gain appropriate access. Pfizer supports patients by helping them understand their insurance coverage requirements and can connect eligible patients with financial assistance resources which may be available including the Pfizer Patient Assistance Program.*

About ATTR-CM
Transthyretin amyloid cardiomyopathy (ATTR-CM) is a rare and fatal condition that is caused by destabilization of a transport protein called transthyretin, which is composed of four identical sub units (a tetramer). When unstable transthyretin tetramers dissociate, they result in misfolded proteins that aggregate into amyloid fibrils and deposit in the heart, causing the heart muscle to become stiff, eventually resulting in heart failure. There are two sub-types of ATTR-CM: hereditary, also known as variant, which is caused by a mutation in the transthyretin gene and can occur in people as early as their 50s and 60s; or with no mutation and associated with aging, known as the wild-type form, which is thought to be more common and usually affects men after age 60. Often ATTR-CM is diagnosed only after symptoms have become severe. Once diagnosed, the median life expectancy in patients with ATTR-CM, dependent on sub-type, is approximately two to 3.5 years.

About VYNDAQEL (tafamidis meglumine) and VYNDAMAX (tafamidis)
VYNDAQEL (tafamidis meglumine) and VYNDAMAX (tafamidis) are oral transthyretin stabilizers that selectively bind to transthyretin, stabilizing the tetramer of the transthyretin transport protein and slowing the formation of amyloid that causes ATTR-CM.

VYNDAMAX 61 mg is a once-daily oral capsule developed for patient convenience. VYNDAQEL and VYNDAMAX are not substitutable on a per milligram basis.

VYNDAQEL was granted Orphan Drug Designation for ATTR-CM in both the EU and U.S. in 2012 and in Japan in 2018. In June 2017 and May 2018, respectively, the FDA granted VYNDAQEL Fast Track and Breakthrough Therapy designations for ATTR-CM. In November 2018, the FDA granted Priority Review for the new drug application (NDA) for VYNDAQEL.

In March 2019, the Ministry of Labor Health and Welfare in Japan approved VYNDAQEL, under SAKIGAKE designation, for patients with wild-type and variant forms of ATTR-CM. Regulatory submissions for the use of VYNDAQEL in patients with ATTR-CM have been submitted to the European Medicines Agency (EMA) and are under review.

VYNDAQEL was first approved in 2011 in the EU for the treatment of transthyretin amyloid polyneuropathy (ATTR-PN), in adult patients with early-stage symptomatic polyneuropathy to delay peripheral neurologic impairment. ATTR-PN is a neurodegenerative form of amyloidosis that leads to sensory loss, pain and weakness in the lower limbs and impairment of the autonomic nervous system, Currently, it is approved for ATTR-PN in 40 countries, including Japan, countries in Europe, Brazil, Mexico, Argentina, Israel, Russia, and South Korea. VYNDAQEL and VYNDAMAX are not approved for the treatment of ATTR-PN in the U.S.

SOURCE

https://www.pfizer.com/news/press-release/press-release-detail/u_s_fda_approves_vyndaqel_and_vyndamax_for_use_in_patients_with_transthyretin_amyloid_cardiomyopathy_a_rare_and_fatal_disease

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eProceedings for BIO 2019 International Convention, June 3-6, 2019 Philadelphia Convention Center; Philadelphia PA, Real Time Coverage by Stephen J. Williams, PhD @StephenJWillia2

 

CONFERENCE OVERVIEW

Real Time Coverage of BIO 2019 International Convention, June 3-6, 2019 Philadelphia Convention Center; Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/05/31/real-time-coverage-of-bio-international-convention-june-3-6-2019-philadelphia-convention-center-philadelphia-pa/

 

LECTURES & PANELS

Real Time Coverage @BIOConvention #BIO2019: Machine Learning and Artificial Intelligence: Realizing Precision Medicine One Patient at a Time, 6/5/2019, Philadelphia PA

Reporter: Stephen J Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/05/real-time-coverage-bioconvention-bio2019-machine-learning-and-artificial-intelligence-realizing-precision-medicine-one-patient-at-a-time/

 

Real Time Coverage @BIOConvention #BIO2019: Genome Editing and Regulatory Harmonization: Progress and Challenges, 6/5/2019. Philadelphia PA

Reporter: Stephen J Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/05/real-time-coverage-bioconvention-bio2019-genome-editing-and-regulatory-harmonization-progress-and-challenges/

 

Real Time Coverage @BIOConvention #BIO2019: Precision Medicine Beyond Oncology June 5, 2019, Philadelphia PA

Reporter: Stephen J Williams PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/05/real-time-coverage-bioconvention-bio2019-precision-medicine-beyond-oncology-june-5-philadelphia-pa/

 

Real Time @BIOConvention #BIO2019:#Bitcoin Your Data! From Trusted Pharma Silos to Trustless Community-Owned Blockchain-Based Precision Medicine Data Trials, 6/5/2019, Philadelphia PA

Reporter: Stephen J Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/05/real-time-bioconvention-bio2019bitcoin-your-data-from-trusted-pharma-silos-to-trustless-community-owned-blockchain-based-precision-medicine-data-trials/

 

Real Time Coverage @BIOConvention #BIO2019: Keynote Address Jamie Dimon CEO @jpmorgan June 5, 2019, Philadelphia, PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/05/real-time-coverage-bioconvention-bio2019-keynote-address-jamie-dimon-ceo-jpmorgan-june-5-philadelphia/

 

Real Time Coverage @BIOConvention #BIO2019: Chat with @FDA Commissioner, & Challenges in Biotech & Gene Therapy June 4, 2019, Philadelphia, PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/04/real-time-coverage-bioconvention-bio2019-chat-with-fda-commissioner-challenges-in-biotech-gene-therapy-june-4-philadelphia/

 

Falling in Love with Science: Championing Science for Everyone, Everywhere June 4 2019, Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/04/real-time-coverage-bioconvention-bio2019-falling-in-love-with-science-championing-science-for-everyone-everywhere/

 

Real Time Coverage @BIOConvention #BIO2019: June 4 Morning Sessions; Global Biotech Investment & Public-Private Partnerships, 6/4/2019, Philadelphia PA

Reporter: Stephen J Williams PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/04/real-time-coverage-bioconvention-bio2019-june-4-morning-sessions-global-biotech-investment-public-private-partnerships/

 

Real Time Coverage @BIOConvention #BIO2019: Understanding the Voices of Patients: Unique Perspectives on Healthcare; June 4, 2019, 11:00 AM, Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/04/real-time-coverage-bioconvention-bio2019-understanding-the-voices-of-patients-unique-perspectives-on-healthcare-june-4/

 

Real Time Coverage @BIOConvention #BIO2019: Keynote: Siddhartha Mukherjee, Oncologist and Pulitzer Author; June 4 2019, 9AM, Philadelphia PA

Reporter: Stephen J. Williams, PhD. @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/04/real-time-coverage-bioconvention-bio2019-keynote-siddhartha-mukherjee-oncologist-and-pulitzer-author-june-4-9am-philadelphia-pa/

 

Real Time Coverage @BIOConvention #BIO2019:  Issues of Risk and Reproduceability in Translational and Academic Collaboration; 2:30-4:00 June 3, 2019, Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/03/real-time-coverage-bioconvention-bio2019-issues-of-risk-and-reproduceability-in-translational-and-academic-collaboration-230-400-june-3-philadelphia-pareal-time-coverage-bioconvention-bi/

 

Real Time Coverage @BIOConvention #BIO2019: What’s Next: The Landscape of Innovation in 2019 and Beyond. 3-4 PM June 3, 2019, Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/03/real-time-coverage-bioconvention-bio2019-whats-next-the-landscape-of-innovation-in-2019-and-beyond-3-4-pm-june-3-philadelphia-pa/

 

Real Time Coverage @BIOConvention #BIO2019: After Trump’s Drug Pricing Blueprint: What Happens Next? A View from Washington; June 3, 2019 1:00 PM, Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/03/real-time-coverage-bioconvention-bio2019-after-trumps-drug-pricing-blueprint-what-happens-next-a-view-from-washington-june-3-2019-100-pm-philadelphia-pa/

 

Real Time Coverage @BIOConvention #BIO2019: International Cancer Clusters Showcase June 3, 2019, Philadelphia PA

Reporter: Stephen J. Williams PhD @StephenJWillia2

https://pharmaceuticalintelligence.com/2019/06/03/real-time-coverage-bioconvention-bio2019-international-cancer-clusters-showcase-june-3-philadelphia-pa/

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Real Time Coverage @BIOConvention #BIO2019: Genome Editing and Regulatory Harmonization: Progress and Challenges

Reporter: Stephen J Williams, PhD @StephenJWillia2

 

Genome editing offers the potential of new and effective treatments for genetic diseases. As companies work to develop these treatments, regulators are focused on ensuring that any such products meet applicable safety and efficacy requirements. This panel will discuss how European Union and United States regulators are approaching therapeutic use of genome editing, issues in harmonization between these two – and other – jurisdictions, challenges faced by industry as regulatory positions evolve, and steps that organizations and companies can take to facilitate approval and continued efforts at harmonization.

 

CBER:  because of the nature of these gene therapies, which are mainly orphan, there is expedited review.  Since they started this division in 2015, they have received over 1500 applications.

Spark: Most of the issues were issues with the primary disease not the gene therapy so they had to make new endpoint tests so had talks with FDA before they entered phase III.   There has been great collaboration with FDA,  now they partnered with Novartis to get approval outside US.  You should be willing to partner with EU pharmas to expedite the regulatory process outside US.  In China the process is new and Brazil is behind on their gene therapy guidance.  However there is the new issue of repeat testing of your manufacturing process, as manufacturing of gene therapies had been small scale before. However he notes that problems with expedited review is tough because you don’t have alot of time to get data together.  They were lucky that they had already done a randomized trial.

Sidley Austin:  EU regulatory you make application with advance therapy you don’t have a national option, the regulation body assesses a committee to see if has applicability. Then it goes to a safety committee.  EU has been quicker to approve these advance therapies. Twenty five percent of their applications are gene therapies.  Companies having issues with manufacturing.  There can be issues when the final application is formalized after discussions as problems may arise between discussions, preliminary applications, and final applications.

Sarepta: They have a robust gene therapy program.  Their lead is a therapy for DMD (Duchenne’s Muscular Dystrophy) where affected males die by 25. Japan and EU have different regulatory applications and although they are similar and data can be transferred there is more paperwork required by EU.  The US uses an IND for application. Global feedback is very challenging, they have had multiple meetings around the world and takes a long time preparing a briefing package….. putting a strain on the small biotechs.  No company wants to be either just EU centric or US centric they just want to get out to market as fast as possible.

 

Please follow LIVE on TWITTER using the following @ handles and # hashtags:

@Handles

@pharma_BI

@AVIVA1950

@BIOConvention

# Hashtags

#BIO2019 (official meeting hashtag)

 

 

 

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Real Time Coverage @BIOConvention #BIO2019: Chat with @FDA Commissioner, & Challenges in Biotech & Gene Therapy June 4 Philadelphia

Reporter: Stephen J. Williams, PhD @StephenJWillia2

 

  • taking patient concerns and voices from anecdotal to data driven system
  • talked about patient accrual hearing patient voice not only in ease of access but reporting toxicities
  • at FDA he wants to remove barriers to trial access and accrual; also talk earlier to co’s on how they should conduct a trial

Digital tech

  • software as medical device
  • regulatory path is mixed like next gen sequencing
  • wearables are concern for FDA (they need to recruit scientists who know this tech

Opioids

  • must address the crisis but in a way that does not harm cancer pain patients
  • smaller pain packs “blister packs” would be good idea

Clinical trial modernization

  • for Alzheimers disease problem is science
  • for diabetes problem is regulatory
  • different diseases calls for different trial design
  • have regulatory problems with rare diseases as can’t form control or placebo group, inhumane. for example ras tumors trials for MEK inhibitors were narrowly focused on certain ras mutants
Realizing the Promise of Gene Therapies for Patients Around the World

103ABC, Level 100

Speakers
Lots of promise, timeline is progressing faster but we need more education on use of the gene therapy
Regulatory issues: Cell and directly delivered gene based therapies have been now approved. Some challenges will be the ultrarare disease trials and how we address manufacturing issues.  Manufacturing is a big issue at CBER and scalability.  If we want to have global impact of these products we need to address the manufacturing issues
 of scalability.
Pfizer – clinical grade and scale is important.
Aventis – he knew manufacturing of biologics however gene therapy manufacturing has its separate issues and is more complicated especially for regulatory purposes for clinical grade as well as scalability.  Strategic decision: focusing on the QC on manufacturing was so important.  Had a major issue in manufacturing had to shut down and redesign the system.
Albert:  Manufacturing is the most important topic even to the investors.  Investors were really conservative especially seeing early problems but when academic centers figured out good efficacy then they investors felt better and market has exploded.  Now you can see investment into preclinical and startups but still want mature companies to focus on manufacturing.  About $10 billion investment in last 4 years.

How Early is Too Early? Valuing and De-Risking Preclinical Opportunities

109AB, Level 100

Speakers
Valuing early-stage opportunities is challenging. Modeling will often provide a false sense of accuracy but relying on comparable transactions is more art than science. With a long lead time to launch, even the most robust estimates can ultimately prove inaccurate. This interactive panel will feature venture capital investors and senior pharma and biotech executives who lead early-stage transactions as they discuss their approaches to valuing opportunities, and offer key learnings from both successful and not-so-successful experiences.
Dr. Schoenbeck, Pfizer:
  • global network of liaisons who are a dedicated team to research potential global startup partners or investments.  Pfizer has a separate team to evaluate academic laboratories.  In Most cases Pfizer does not initiate contact.  It is important to initiate the first discussion with them in order to get noticed.  Could be just a short chat or discussion on what their needs are for their portfolio.

Question: How early is too early?

Luc Marengere, TVM:  His company has early stage focus, on 1st in class molecules.  The sweet spot for their investment is a candidate selected compound, which should be 12-18 months from IND.  They will want to bring to phase II in less than 4 years for $15-17 million.  Their development model is bad for academic labs.  During this process free to talk to other partners.

Dr. Chaudhary, Biogen:  Never too early to initiate a conversation and sometimes that conversation has lasted 3+ years before a decision.  They like build to buy models, will do convertible note deals, candidate compound selection should be entering in GLP/Tox phase (sweet spot)

Merck: have MRL Venture Fund for pre series A funding.  Also reiterated it is never too early to have that initial discussion.  It will not put you in a throw away bin.  They will have suggestions and never like to throw out good ideas.

Michael Hostetler: Set expectations carefully ; data should be validated by a CRO.  If have a platform, they will look at the team first to see if strong then will look at the platform to see how robust it is.

All noted that you should be completely honest at this phase.  Do not overstate your results or data or overhype your compound(s).  Show them everything and don’t have a bias toward compounds you think are the best in your portfolio.  Sometimes the least developed are the ones they are interested in.  Also one firm may reject you however you may fit in others portfolios better so have a broad range of conversations with multiple players.

 

 

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Real Time Coverage @BIOConvention #BIO2019: After Trump’s Drug Pricing Blueprint: What Happens Next? A View from Washington; June 3 2019 1:00 PM Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

 

Speaker: Dan Todd, JD

Dan Todd is the Principal of Todd Strategy, LLC, a consulting firm founded in 2014 and based in Washington, DC. He provides legislative and regulatory strategic guidance and advocacy for healthcare stakeholders impacted by federal healthcare programs.

Prior to Todd Strategy, Mr. Todd was a Senior Healthcare Counsel for the Republican staff of the Senate Finance Committee, the Committee of jurisdiction for the Medicare and Medicaid programs. His areas of responsibility for the committee included the Medicare Part B and Part D programs, which includes physician, medical device, diagnostic and biopharmaceutical issues.

Before joining the Finance Committee, Mr. Todd spent several years in the biotechnology industry, where he led policy development and government affairs strategy. He also represented his companies’ interests with major trade associations such as PhRMA and BIO before federal and state representatives, as well as with key stakeholders such as physician and patient advocacy organizations.

Dan also served as a Special Assistant in the Office of the Administrator at the Centers for Medicare & Medicaid Services (CMS), the federal agency charged with the operation of the Medicare and Medicaid programs. While at CMS, Dan worked on Medicare Part B and Part D issues during the implementation of the Medicare Modernization Act from 2003 to 2005.

Cost efficiencies were never measured.

Removing drug rebates would cost 180 billion over 10 years. CBO came up with similar estimate.  Not sure what Congress will do. It appears they will keep the rebates in.

  • House  Dems are really going after PBMs; anytime the Administration makes a proposal goes right into CBO baseline estimates;  negotiations appear to be in very early stages and estimates are up in the air
  • WH close to meet a budget cap but then broke down in next day; total confusion in DC on budget; healthcare is now held up, especially the REBATE rule; : is a shame as panel agrees cost savings would be huge
  • they had initiated a study to tie the costs of PartB to international drug prices; meant to get at disparity on international drug prices; they currently are only mulling the international price index; other option is to reform Part B;  the proposed models were brought out near 2016 elections so not much done; unified agenda;
  • most of the response of Congress relatively publicly muted; a flat fee program on biologics will have big effect on how physicians and health systems paid; very cat and mouse game in DC around drug pricing
  • administration is thinking of a PartB “inflation cap”;  committees are looking at it seriously; not a rebate;  discussion of tiering of physician payments
  • Ways and Means Cmmtte:  proposing in budget to alleve some stresses on PartB deductable amounts;
  • PartD: looking at ways to shore it up; insurers 80% taxpayers 20% responsible; insurers think it will increase premiums but others think will reduce catastrophic costs; big part of shift in spending in Part D has been this increase in catastrophic costs
  • this week they may actually move through committees on this issue; Administration trying to use the budgetary process to drive this bargain;  however there will have to be offsets so there may be delays in process

Follow or Tweet on Twitter using the following @ and # (hashtags)

@pharma_BI

@AVIVA1950

@BIOConvention

@PCPCC

#BIO2019

#patientcost

#PrimaryCare

 

Other articles on this Open Access Journal on Healthcare Costs, Payers, and Patient Care Include:

The Arnold Relman Challenge: US HealthCare Costs vs US HealthCare Outcomes

Centers for Medicare & Medicaid Services announced that the federal healthcare program will cover the costs of cancer gene tests that have been approved by the Food and Drug Administration

Trends in HealthCare Economics: Average Out-of-Pocket Costs, non-Generics and Value-Based Pricing, Amgen’s Repatha and AstraZeneca’s Access to Healthcare Policies

Can Blockchain Technology and Artificial Intelligence Cure What Ails Biomedical Research and Healthcare

Live Conference Coverage @Medcity Converge 2018 Philadelphia: Oncology Value Based Care and Patient Management

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Real Time Coverage of BIO 2019 International Convention, June 3-6, 2019 Philadelphia Convention Center, Philadelphia PA

Reporter: Stephen J. Williams, PhD @StephenJWillia2

Please follow LIVE on TWITTER using the following @ handles and # hashtags:

@Handles

@pharma_BI

@AVIVA1950

@BIOConvention

# Hashtags

#BIO2019 (official meeting hashtag)

Please check daily on this OPEN ACCESS JOURNAL for updates on one of the most important BIO Conferences of the year for meeting notes, posts, as well as occasional PODCASTS.

 

The BIO International Convention is the largest global event for the biotechnology industry and attracts the biggest names in biotech, offers key networking and partnering opportunities, and provides insights and inspiration on the major trends affecting the industry. The event features keynotes and sessions from key policymakers, scientists, CEOs, and celebrities.  The Convention also features the BIO Business Forum (One-on-One Partnering), hundreds of sessions covering biotech trends, policy issues and technological innovations, and the world’s largest biotechnology exhibition – the BIO Exhibition.

The BIO International Convention is hosted by the Biotechnology Innovation Organization (BIO). BIO represents more than 1,100 biotechnology companies, academic institutions, state biotechnology centers and related organizations across the United States and in more than 30 other nations. BIO members are involved in the research and development of innovative healthcare, agricultural, industrial and environmental biotechnology products.

 

Keynote Speakers INCLUDE:

Fireside Chat with Margaret (Peggy) Hamburg, MD, Foreign Secretary, National Academy of Medicine; Chairman of the Board, American Association for the Advancement of Science

Tuesday Keynote: Siddhartha Mukherjee (Author of the bestsellers Emperor of All Maladies: A Biography of Cancer and  The Gene: An Intimate History)

Fireside Chat with Jeffrey Solomon, Chief Executive Officer, COWEN

Fireside Chat with Christi Shaw, Senior Vice President and President, Lilly BIO-Medicines, Eli Lilly and Company

Wednesday Keynote: Jamie Dimon (Chairman JP Morgan Chase)

Fireside Chat with Kenneth C. Frazier, Chairman of the Board and Chief Executive Officer, Merck & Co., Inc.

Fireside Chat: Understanding the Voices of Patients: Unique Perspectives on Healthcare

Fireside Chat: FDA Town Hall

 

ALSO SUPERSESSIONS including:

Super Session: What’s Next: The Landscape of Innovation in 2019 and Beyond

Super Session: Falling in Love with Science: Championing Science for Everyone, Everywhere

Super Session: Digital Health in Practice: A Conversation with Ameet Nathawani, Chief Digital Officer, Chief Medical Falling in Love with Science: Championing Science for Everyone, Everywhere

Super Session: Realizing the Promise of Gene Therapies for Patients Around the World

Super Session: Biotech’s Contribution to Innovation: Current and Future Drivers of Success

Super Session: The Art & Science of R&D Innovation and Productivity

Super Session: Dealmaker’s Intentions: 2019 Market Outlook

Super Session: The State of the Vaccine Industry: Stimulating Sustainable Growth

 

See here for full AGENDA

Link for Registration: https://convention.bio.org/register/

The BIO International Convention is literally where hundreds of deals and partnerships have been made over the years.

 

BIO performs many services for members, but none of them are more visible than the BIO International Convention. The BIO International Convention helps BIO fulfill its mission to help grow the global biotech industry. Profits from the BIO International Convention are returned to the biotechnology industry by supporting BIO programs and initiatives. BIO works throughout the year to create a policy environment that enables the industry to continue to fulfill its vision of bettering the world through biotechnology innovation.

The key benefits of attending the BIO International Convention are access to global biotech and pharma leaders via BIO One-on-One Partnering, exposure to industry though-leaders with over 1,500 education sessions at your fingertips, and unparalleled networking opportunities with 16,000+ attendees from 74 countries.

In addition, we produce BIOtechNOW, an online blog chronicling ‘innovations transforming our world’ and the BIO Newsletter, the organization’s bi-weekly email newsletter. Subscribe to the BIO Newsletter.

 

Membership with the Biotechnology Innovation Organization (BIO)

BIO has a diverse membership that is comprised of  companies from all facets of biotechnology. Corporate R&D members range from entrepreneurial companies developing a first product to Fortune 100 multinationals. The majority of our members are small companies – 90 percent have annual revenues of $25 million or less, reflecting the broader biotechnology industry. Learn more about how you can save with BIO Membership.

BIO also represents academic centers, state and regional biotech associations and service providers to the industry, including financial and consulting firms.

  • 66% R&D-Intensive Companies *Of those: 89% have annual revenues under $25 million,  4% have annual revenues between $25 million and $1 billion, 7% have annual revenues over $1 billion.
  • 16% Nonprofit/Academic
  • 11% Service Providers
  • 7% State/International Affiliate Organizations

Other posts on LIVE CONFERENCE COVERAGE using Social Media on this OPEN ACCESS JOURNAL and OTHER Conferences Covered please see the following link at https://pharmaceuticalintelligence.com/press-coverage/

 

Notable Conferences Covered THIS YEAR INCLUDE: (see full list from 2013 at this link)

  • Koch Institute 2019 Immune Engineering Symposium, January 28-29, 2019, Kresge Auditorium, MIT

https://calendar.mit.edu/event/immune_engineering_symposium_2019#.XBrIDc9Kgcg

http://kochinstituteevents.cvent.com/events/koch-institute-2019-immune-engineering-symposium/event-summary-8d2098bb601a4654991060d59e92d7fe.aspx?dvce=1

 

  • 2019 MassBio’s Annual Meeting, State of Possible Conference ​, March 27 – 28, 2019, Royal Sonesta, Cambridge

http://files.massbio.org/file/MassBio-State-Of-Possible-Conference-Agenda-Feb-22-2019.pdf

 

  • World Medical Innovation Forum, Partners Innovations, ARTIFICIAL INTELLIGENCE | APRIL 8–10, 2019 | Westin, BOSTON

https://worldmedicalinnovation.org/agenda-list/

https://worldmedicalinnovation.org/

 

  • 18th Annual 2019 BioIT, Conference & Expo, April 16-18, 2019, Boston, Seaport World Trade Center, Track 5 Next-Gen Sequencing Informatics – Advances in Large-Scale Computing

http://www.giiconference.com/chi653337/

https://pharmaceuticalintelligence.com/2019/04/22/18th-annual-2019-bioit-conference-expo-april-16-18-2019-boston-seaport-world-trade-center-track-5-next-gen-sequencing-informatics-advances-in-large-scale-computing/

 

  • Translating Genetics into Medicine, April 25, 2019, 8:30 AM – 6:00 PM, The New York Academy of Sciences, 7 World Trade Center, 250 Greenwich St Fl 40, New York

https://pharmaceuticalintelligence.com/2019/04/25/translating-genetics-into-medicine-april-25-2019-830-am-600-pm-the-new-york-academy-of-sciences-7-world-trade-center-250-greenwich-st-fl-40-new-york/

 

  • 13th Annual US-India BioPharma & Healthcare Summit, May 9, 2019, Marriott, Cambridge

https://pharmaceuticalintelligence.com/2019/04/30/13th-annual-biopharma-healthcare-summit-thursday-may-9-2019/

 

  • 2019 Petrie-Flom Center Annual Conference: Consuming Genetics: Ethical and Legal Considerations of New Technologies, May 17, 2019, Harvard Law School

http://petrieflom.law.harvard.edu/events/details/2019-petrie-flom-center-annual-conference

https://pharmaceuticalintelligence.com/2019/01/11/2019-petrie-flom-center-annual-conference-consuming-genetics-ethical-and-legal-considerations-of-new-technologies/

 

  • 2019 Koch Institute Symposium – Machine Learning and Cancer, June 14, 2019, 8:00 AM-5:00 PM  ET MIT Kresge Auditorium, 48 Massachusetts Ave, Cambridge, MA

https://pharmaceuticalintelligence.com/2019/03/12/2019-koch-institute-symposium-machine-learning-and-cancer-june-14-2019-800-am-500-pmet-mit-kresge-auditorium-48-massachusetts-ave-cambridge-ma/

 

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World’s first artificial pancreas

Reporter: Irina Robu, PhD

Diabetes is a life-long condition where your body does not produce enough insulin (Type 1) or your body cannot use the insulin it has effectively. Since there is no cure for diabetes, the artificial pancreas system comes as a relief for patients that are suffering with this disease.

The artificial pancreas, MiniMed 670G hybrid closed loop system designed by Medtronic is the first FDA-approved device that measures glucose levels and delivers the appropriate dose of basal insulin. The system comprises Medtronic’s MiniMed 670G insulin pump that is strapped to the body, an infusion patch that delivers insulin via catheter from the pump and a sensor which measures glucose levels under the skin and can be worn for 7 days at a time. While the device regulates basal, or background, insulin, patients must still manually request bolus insulin at mealtimes.

The device is intended for people age 14 or older with Type 1 diabetes and is intended to regulate insulin levels with “little to no input” from the patient. The artificial pancreas measures blood sugar levels using a constant glucose monitor (CGM) and communicates the information to an insulin pump which calculates and releases the required amount of insulin into the body, just as the pancreas does in people without diabetes.

The 2016 FDA approval was done in just three months which is a record for any medical device. The agency evaluated data from a clinical trial in which 123 patients with Type 1 diabetes used the system’s hybrid closed-loop feature as repeatedly during a three-month period. The trial presented the device to be safe for use in those 14 and older, showing no serious adverse events. The system is on sale since spring 2017.

While further clinical research is needed to ensure that the strength of the device in different settings is consistent, several researchers support the view that “artificial pancreas systems are a safe and effective treatment approach for people with type 1 diabetes. Medtronic counts this device as a step toward a fully automated, closed-loop system.

SOURCE

https://www.fiercebiotech.com/medical-devices/fda-approves-medtronic-s-artificial-pancreas-world-s-first

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One or More Clinical Trials to get FDA Approve a Drug?

 

Reporter: Aviva Lev-Ari, PhD, RN

Almost half of all new drug approvals in 2018 relied on one clinical trial

SOURCE

https://endpts.com/almost-half-of-all-new-drug-approvals-in-2018-relied-on-one-clinical-trial/?utm_medium=email&utm_campaign=726%20JJ%20has%20a%20new%20list%20of%20blockbusters-to-be%20it%20wants%20you%20to%20know%20about%20Top%20Biogen%20exec%20jumps%20ship&utm_content=726%20JJ%20has%20a%20new%20list%20of%20blockbusters-to-be%20it%20wants%20you%20to%20know%20about%20Top%20Biogen%20exec%20jumps%20ship+CID_15fe600050d8a9e0e22fba39d1651c9a&utm_source=ENDPOINTS%20emails&utm_term=Almost%20half%20of%20all%20new%20drug%20approvals%20in%202018%20relied%20on%20one%20clinical%20trial

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