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https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/novel-drug-approvals-2022

2022 FDA Drug Approval List, 2022 Biological Approvals and Approved Cellular and Gene Therapy Products

Posted in cell-based therapy, Drug Discovery Chemistry, FDA, FDA, CE Mark & Global Regulatory Affairs: process management and strategic planning - GCP, GLP, ISO 14155, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical R&D Informatics, Pharmacodynamics and Pharmacokinetics, Regenerative Biology and Medicine on January 17, 2023| Leave a Comment »

2022 FDA Drug Approval List, 2022 Biological Approvals and Approved Cellular and Gene Therapy Products

Reporter: Aviva Lev-Ari, PhD, RN

SOURCE

Tal Bahar’s post on LinkedIn on 1/17/2023

Novel Drug Approvals for 2022

FDA’s Center for Drug Evaluation and Research (CDER)

New Molecular Entities (“NMEs”)

Some of these products have never been used in clinical practice. Below is a listing of new molecular entities and new therapeutic biological products that CDER approved in 2022. This listing does not contain vaccines, allergenic products, blood and blood products, plasma derivatives, cellular and gene therapy products, or other products that the Center for Biologics Evaluation and Research approved in 2022.

Others are the same as, or related to, previously approved products, and they will compete with those products in the marketplace. See Drugs@FDA for information about all of CDER’s approved drugs and biological products.

Certain drugs are classified as new molecular entities (“NMEs”) for purposes of FDA review. Many of these products contain active moieties that FDA had not previously approved, either as a single ingredient drug or as part of a combination product. These products frequently provide important new therapies for patients. Some drugs are characterized as NMEs for administrative purposes, but nonetheless contain active moieties that are closely related to active moieties in products that FDA has previously approved. FDA’s classification of a drug as an “NME” for review purposes is distinct from FDA’s determination of whether a drug product is a “new chemical entity” or “NCE” within the meaning of the Federal Food, Drug, and Cosmetic Act.

No.	Drug Name	Active Ingredient	Approval Date	FDA-approved use on approval date*
37.	NexoBrid	anacaulase-bcdb	12/28/2022	To remove eschar in adults with deep partial thickness or full thickness thermal burns
36.	Briumvi	ublituximab-xiiy	12/28/2022	To treat relapsing forms of multiple sclerosis
35.	Xenoview	hyperpolarized Xe-129	12/23/2022	To evaluate pulmonary function and imaging
34.	Lunsumio	mosunetuzumab-axgb	12/22/2022	To treat adults with relapsed or refractory follicular lymphoma, a type of non-Hodgkin lymphoma
33.	Sunlenca	lenacapavir	12/22/2022	To treat adults with HIV whose HIV infections cannot be successfully treated with other available treatments due to resistance, intolerance, or safety considerations Press Release
32.	Krazati	adagrasib	12/12/2022	To treat KRAS G12C-mutated locally advanced or metastatic non-small cell lung cancer in adults who have received at least one prior systemic therapy
31.	Rezlidhia	olutasidenib	12/1/2022	To treat adults with relapsed or refractory acute myeloid leukemia with a susceptible isocitrate dehydrogenase-1 (IDH1) mutation
30.	Tzield	teplizumab-mzwv	11/18/22	To delay the onset of stage 3 type 1 diabetes Press Release
29.	Elahere	mirvetuximab soravtansine-gynx	11/14/2022	To treat patients with recurrent ovarian cancer that is resistant to platinum therapy
28.	Tecvayli	teclistamab-cqyv	10/25/2022	To treat relapsed or refractory multiple myeloma among adults who have received at least four specific lines of therapy
27.	Imjudo	tremelimumab	10/21/2022	To treat unresectable hepatocellular carcinoma
26.	Lytgobi	futibatinib	9/30/2022	To treat intrahepatic cholangiocarcinoma harboring fibroblast growth factor receptor 2 (FGFR2) gene fusions or other rearrangements
25.	Relyvrio	sodium phenylbutyrate/taurursodiol	9/29/2022	To treat amyotrophic lateral sclerosis (ALS) Press Release
24.	Omlonti	oomidenepag isopropyl ophthalmic solution	9/22/2022	To reduce elevated intraocular pressure in patients with open‑angle glaucoma or ocular hypertension
23.	Elucirem	gadopiclenol	9/21/2022	To detect and visualize lesions, together with MRI, with abnormal vascularity in the central nervous system and the body
22.	Terlivaz	terlipressin	9/14/2022	To improve kidney function in adults with hepatorenal syndrome with rapid reduction in kidney function
21.	Rolvedon	eflapegrastim	9/9/2022	To decrease the incidence of infection in patients with non-myeloid malignancies receiving myelosuppressive anti-cancer drugs associated with clinically significant incidence of febrile neutropenia
20.	Sotyktu	deucravacitinib	9/9/2022	To treat moderate-to-severe plaque psoriasis
19.	Daxxify	daxibotulinumtoixnA-lanm	9/7/2022	To treat moderate-to-severe glabellar lines associated with corrugator and/or procerus muscle activity
18.	Spevigo	spesolimab-sbzo	9/1/2022	To treat generalized pustular psoriasis flares
17.	Xenpozyme	Olipudase alfa	8/31/2022	To treat Acid Sphingomyelinase Deficiency Press Release
16.	Amvuttra	vutrisiran	6/13/2022	To treat polyneuropathy of hereditary transthyretin-mediated amyloidosis
15.	Vtama	tapinarof	5/23/2022	To treat plaque psoriasis
14.	Mounjaro	tirzepatide	5/13/2022	To improve blood sugar control in diabetes, in addition to diet and exercise Press Release
13.	Voquezna	vonoprazan, amoxicillin, and clarithromycin	5/3/2022	To treat Helicobacter pylori infection
12.	Camzyos	mavacamten	4/28/2022	To treat certain classes of obstructive hypertrophic cardiomyopathy
11.	Vivjoa	oteseconazole	4/26/2022	To reduce the incidence of recurrent vulvovaginal candidiasis (RVVC) in females with a history of RVVC who are not of reproductive potential
10.	Pluvicto	lutetium (177Lu) vipivotide tetraxetan	3/23/2022	To treat prostate-specific membrane antigen-positive metastatic castration-resistant prostate cancer following other therapies
9.	Opdualag	nivolumab and relatlimab-rmbw	3/18/2022	To treat unresectable or metastatic melanoma
8.	Ztalmy	ganaxolone	3/18/2022	To treat seizures in cyclin-dependent kinase-like 5 deficiency disorder
7.	Vonjo	pacritinib	2/28/2022	To treat intermediate or high-risk primary or secondary myelofibrosis in adults with low platelets
6.	Pyrukynd	mitapivat	2/17/2022	To treat hemolytic anemia in pyruvate kinase deficiency
5.	Enjaymo	sutimlimab-jome	2/4/2022	To decrease the need for red blood cell transfusion due to hemolysis in cold agglutinin disease
4.	Vabysmo	faricimab-svoa	1/28/2022	To treat neovascular (wet) aged-related macular degeneration and diabetic macular edema
3.	Kimmtrak	tebentafusp-tebn	1/25/2022	To treat unresectable or metastatic uveal melanoma
2.	Cibinqo	abrocitinib	1/14/2022	To treat refractory, moderate-to-severe atopic dermatitis
1.	Quviviq	daridorexant	1/7/2022	To treat insomnia

Showing 1 to 37 of 37 entries

SOURCE

INNOVATION PREDICTABILITY ACCESS FDA’s Center for Drug Evaluation and Research

January 2023

SOURCE

https://www.fda.gov/drugs/new-drugs-fda-cders-new-molecular-entities-and-new-therapeutic-biological-products/new-drug-therapy-approvals-2022

2022 Biological Approvals

The Center for Biologics Evaluation and Research (CBER) regulates products under a variety of regulatory authorities. See the Development & Approval Process page for a description of what products are approved as Biologics License Applications (BLAs), Premarket Approvals (PMAs), New Drug Applications (NDAs) or 510Ks.

Biologics License Applications and Supplements

New BLAs (except those for blood banking), and BLA supplements that are expected to significantly enhance the public health (e.g., for new/expanded indications, new routes of administration, new dosage formulations and improved safety).

2022 Biological License Application Approvals

2022 Biological License Application Supplement Noteworthy Approvals

2022 Biological New Drug Application (NDA) and Abbreviated New Drug Application (ANDA) Approvals

Other Applications Approved or Cleared by the Center for Biologics Evaluation and Research (CBER)

Medical devices involved in the collection, processing, testing, manufacture and administration of licensed blood, blood components and cellular products.

2022 Biological Device Application Approvals

Key Resources

SOURCE

https://www.fda.gov/vaccines-blood-biologics/development-approval-process-cber/2022-biological-approvals

Approved Cellular and Gene Therapy Products

Below is a list of licensed products from the Office of Tissues and Advanced Therapies (OTAT).

Approved Products

ABECMA (idecabtagene vicleucel)
Celgene Corporation, a Bristol-Myers Squibb Company

ADSTILADRIN
Ferring Pharmaceuticals A/S

ALLOCORD (HPC, Cord Blood)
SSM Cardinal Glennon Children’s Medical Center

BREYANZI
Juno Therapeutics, Inc., a Bristol-Myers Squibb Company

CARVYKTI (ciltacabtagene autoleucel)
Janssen Biotech, Inc.

CLEVECORD (HPC Cord Blood)
Cleveland Cord Blood Center

Ducord, HPC Cord Blood
Duke University School of Medicine

GINTUIT (Allogeneic Cultured Keratinocytes and Fibroblasts in Bovine Collagen)
Organogenesis Incorporated

HEMACORD (HPC, cord blood)
New York Blood Center

HEMGENIX
CSL Behring LLC

HPC, Cord Blood
Clinimmune Labs, University of Colorado Cord Blood Bank

HPC, Cord Blood – MD Anderson Cord Blood Bank
MD Anderson Cord Blood Bank

HPC, Cord Blood – LifeSouth
LifeSouth Community Blood Centers, Inc.

HPC, Cord Blood – Bloodworks
Bloodworks

IMLYGIC (talimogene laherparepvec)
BioVex, Inc., a subsidiary of Amgen Inc.

KYMRIAH (tisagenlecleucel)
Novartis Pharmaceuticals Corporation

LAVIV (Azficel-T)
Fibrocell Technologies

LUXTURNA
Spark Therapeutics, Inc.

MACI (Autologous Cultured Chondrocytes on a Porcine Collagen Membrane)
Vericel Corp.

PROVENGE (sipuleucel-T)
Dendreon Corp.

RETHYMIC
Enzyvant Therapeutics GmbH

SKYSONA (elivaldogene autotemcel)
bluebird bio, Inc.

STRATAGRAFT
Stratatech Corporation

TECARTUS (brexucabtagene autoleucel)
Kite Pharma, Inc.

YESCARTA (axicabtagene ciloleucel)
Kite Pharma, Incorporated

ZYNTEGLO (betibeglogene autotemcel)
bluebird bio, Inc.

ZOLGENSMA (onasemnogene abeparvovec-xioi)
Novartis Gene Therapies, Inc.

Resources For You

What Is Gene Therapy? How Does It Work?

SOURCE

https://www.fda.gov/vaccines-blood-biologics/cellular-gene-therapy-products/approved-cellular-and-gene-therapy-products

2022 forecast: Cell, gene therapy makers push past regulatory, payer hurdles to set up high hopes for next year

By Arlene Weintraub

Dec 22, 2021 03:00am

There are five FDA-approved CAR-T treatments for blood cancers and two gene therapies to treat rare diseases now on the market in the U.S. The late-stage pipeline could produce several more cancer CAR-Ts and gene therapies to treat a range of diseases.

RELATED: ASH: Bristol Myers’ Breyanzi, Gilead’s Yescarta lock horns in race to move CAR-T therapy to earlier lymphoma

One of the biggest races to watch in the cell therapy space will be that between Gilead Sciences’ Yescarta and Bristol Myers Squibb’s Breyanzi, both of which are gunning to move their CAR-Ts into earlier lines of treatment in large B-cell lymphoma (LBCL). At ASH, both companies rolled out impressive data from their trials in the second-line setting, but Gilead could have the upper hand by virtue of its three-year head start in the market, analysts said. Gilead expects to hear from the FDA on a label expansion in the second-line setting in April.

SOURCE

https://www.fiercepharma.com/pharma/cell-and-gene-therapy-makers-push-past-regulatory-payer-hurdles-to-set-up-high-hopes-for

https://www.cell.com/iscience/fulltext/S2589-0042(20)31218-9

A Platform called VirtualFlow: Discovery of Pan-coronavirus Drugs help prepare the US for the Next Coronavirus Pandemic

Posted in COVID-19, Drug Discovery Chemistry, Mechanisms of infection by SARS-CoV-2, Pharmaceutical Analytics, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Population Health Management, Genetics & Pharmaceutical, SARS-CoV-2, Virtual Drug Molecule Screening targeting SAR-CoV-2 proteins, Virus Infective Acute Respiratory Syndrome: SARS-CoV on February 1, 2021| Leave a Comment »

A Platform called VirtualFlow: Discovery of Pan-coronavirus Drugs help prepare the US for the Next Coronavirus Pandemic

Reporter: Aviva Lev-Ari, PhD, RN

ARTICLE|ONLINE NOW, 102021

A multi-pronged approach targeting SARS-CoV-2 proteins using ultra-large virtual screening

Open AccessPublished:January 04, 2021DOI:https://doi.org/10.1016/j.isci.2020.102021

SOURCE

The work was made possible in large part by about $1 million in cloud computing hours awarded by Google through a COVID-19 research grant program.

The work reported, below was sponsored by

a Google Cloud COVID-19 research grant. Funding was also provided by the
Fondation Aclon,
National Institutes of Health (GM136859),
Claudia Adams Barr Program for Innovative Basic Cancer Research,
Math+ Berlin Mathematics Research Center,
Templeton Religion Trust (TRT 0159),
U.S. Army Research Office (W911NF1910302), and
Chleck Family Foundation

Harvard University, AbbVie form research alliance to address emergent viral diseases

August 25, 2020 Research

This article is part of Harvard Medical School’s continuing coverage of medicine, biomedical research, medical education and policy related to the SARS-CoV-2 pandemic and the disease COVID-19.

Harvard University and AbbVie today announced a $30 million collaborative research alliance, launching a multi-pronged effort at Harvard Medical School to study and develop therapies against emergent viral infections, with a focus on those caused by coronaviruses and by viruses that lead to hemorrhagic fever.

The collaboration aims to rapidly integrate fundamental biology into the preclinical and clinical development of new therapies for viral diseases that address a variety of therapeutic modalities. HMS has led several large-scale, coordinated research efforts launched at the beginning of the COVID-19 pandemic.

“A key element of having a strong R&D organization is collaboration with top academic institutions, like Harvard Medical School, to develop therapies for patients who need them most,” said Michael Severino, vice chairman and president of AbbVie. “There is much to learn about viral diseases and the best way to treat them. By harnessing the power of collaboration, we can develop new therapeutics sooner to ensure the world is better prepared for future potential outbreaks.”

“The cataclysmic nature of the COVID-19 pandemic reminds us how vital it is to be prepared for the next public health crisis and how critical collaboration is on every level—across disciplines, across institutions and across national boundaries,” said George Q. Daley, dean of Harvard Medical School. “Harvard Medical School, as the nucleus of an ecosystem of fundamental discovery and therapeutic translation, is uniquely positioned to propel this transformative research alongside allies like AbbVie.”

AbbVie will provide $30 million over three years and additional in-kind support leveraging AbbVie’s scientists, expertise and facilities to advance collaborative research and early-stage development efforts across five program areas that address a variety of therapeutic modalities:

Immunity and immunopathology—Study of the fundamental processes that impact the body’s critical immune responses to viruses and identification of opportunities for therapeutic intervention.

Led by Ulirich Von Andrian, the Edward Mallinckrodt Jr. Professor of Immunopathology in the Blavatnik Institute at HMS and program leader of basic immunology at the Ragon Institute of MGH, MIT and Harvard, and Jochen Salfeld, vice president of immunology and virology discovery at AbbVie.

Host targeting for antiviral therapies—Development of approaches that modulate host proteins in an effort to disrupt the life cycle of emergent viral pathogens.

Led by Pamela Silver, the Elliot T. and Onie H. Adams Professor of Biochemistry and Systems Biology in the Blavatnik Institute at HMS, and Steve Elmore, vice president of drug discovery science and technology at AbbVie.

Antibody therapeutics—Rapid development of therapeutic antibodies or biologics against emergent pathogens, including SARS-CoV-2, to a preclinical or early clinical stage.

Led by Jonathan Abraham, assistant professor of microbiology in the Blavatnik Institute at HMS, and by Jochen Salfeld, vice president of immunology and virology discovery at AbbVie.

Small molecules—Discovery and early-stage development of small-molecule drugs that would act to prevent replication of known coronaviruses and emergent pathogens.

Led by Mark Namchuk, executive director of therapeutics translation at HMS and senior lecturer on biological chemistry and molecular pharmacology in the Blavatnik Institute at HMS, and Steve Elmore, vice president of drug discovery science and technology at AbbVie.

Translational development—Preclinical validation, pharmacological testing, and optimization of leading approaches, in collaboration with Harvard-affiliated hospitals, with program leads to be determined.

SOURCE

https://hms.harvard.edu/news/joining-forces

A Screen Door Opens

Virtual screen finds compounds that could combat SARS-CoV-2

By STEPHANIE DUTCHEN January 28, 2021 Research

This article is part of Harvard Medical School’s continuing coverage of medicine, biomedical research, medical education, and policy related to the SARS-CoV-2 pandemic and the disease COVID-19.

Less than a year ago, Harvard Medical School researchers and international colleagues unveiled a platform called VirtualFlow that could swiftly sift through more than 1 billion chemical compounds and identify those with the greatest promise to become disease-specific treatments, providing researchers with invaluable guidance before they embark on expensive and time-consuming lab experiments and clinical trials.

Propelled by the urgent needs of the pandemic, the team has now pushed VirtualFlow even further, conducting 45 screens of more than 1 billion compounds each and ranking the compounds with the greatest potential for fighting COVID-19—including some that are already approved by the FDA for other diseases.

“This was the largest virtual screening effort ever done,” said VirtualFlow co-developer Christoph Gorgulla, research fellow in biological chemistry and molecular pharmacology in the labs of Haribabu Arthanari and Gerhard Wagner in the Blavatnik Institute at HMS.

The results were published in January in the open-access journal iScience.

The team searched for compounds that bind to any of 15 proteins on SARS-CoV-2 or two human proteins, ACE2 and TMPRSS2, known to interact with the virus and enable infection.

Researchers can now explore on an interactive website the 1,000 most promising compounds from each screen and start testing in the lab any ones they choose.

The urgency of the pandemic and the sheer number of candidate compounds inspired the team to release the early results to the scientific community.

“No one group can validate all the compounds as quickly as the pandemic demands,” said Gorgulla, who is also an associate of the Department of Physics at Harvard University. “We hope that our colleagues can collectively use our results to identify potent inhibitors of SARS-CoV-2.”

In most cases, it will take years to find out whether a compound is safe and effective in humans. For some of the compounds, however, researchers have a head start.

Hundreds of the most promising compounds that VirtualFlow flagged are already FDA approved or being studied in clinical or preclinical trials for other diseases. If researchers find that one of those compounds proves effective against SARS-CoV-2 in lab experiments, the data their colleagues have already collected could save time establishing safety in humans.

Other compounds among VirtualFlow’s top hits are currently being assessed in clinical trials for COVID-19, including several drugs in the steroid family. In those cases, researchers could build on the software findings to investigate how those drug candidates work at the molecular level—something that’s not always clear even when a drug works well.

It shows what we’re capable of computationally during a pandemic.

Hari Arthanari

In another effort to speed successful transitions from computer to clinic, the team has joined in Harvard’s partnership with pharmaceutical company AbbVie.

If any successful drugs eventually arise from the VirtualFlow screens, they would complement other strategies in the fight against COVID-19. For instance, drugs that halt viral replication or prevent it from entering cells could help those who cannot be vaccinated.

The nature of the screens also raises the likelihood that drugs developed against current forms of SARS-CoV-2 would work against future mutations or other coronaviruses, the authors say.

Computing power

The work was made possible in large part by about $1 million in cloud computing hours awarded by Google through a COVID-19 research grant program.

Gorgulla and colleagues used that power to screen how well the compounds bound to more than 40 sites across the 15 viral proteins as well as to the two human proteins—effectively trying 1 billion 3D puzzle pieces in 40 places each.

The team selected the sites because blocking them with compounds would either prevent SARS-CoV-2 from entering cells, prevent the virus from replicating and thus lower viral load, reduce the virus’s ability to evade or subvert our immune defenses, or disrupt viral assembly and packaging.

Many of the sites have not yet been investigated by other groups, as far as the team is aware.

“We’re exploring new avenues on how to tackle the virus. This is one of the most exciting aspects of our project,” said Gorgulla.

One benefit of studying dozens of sites on 17 proteins at once is the possibility of identifying or developing multiple drugs that, when combined, combat SARS-CoV-2 infection on different fronts. Such drug cocktails would have a better chance of halting the virus in its tracks and overcoming future mutations than a single drug that works on one protein site.

Some sites included in the screen are shared among other coronaviruses of worldwide concern. Any compound that binds to one of those sites has a “high” chance of also combating SARS, MERS, or as-yet-unknown coronavirus diseases, said Arthanari.

“We are in a position to discover pan-coronavirus drugs and help prepare for the next coronavirus pandemic, should there ever be one,” said Gorgulla.

Regardless of the outcomes, the work raises the benchmark for virtual screening and demonstrates the current power of computing in biological research.

“It shows what we’re capable of computationally during a pandemic,” said Arthanari.

The project has also brought special satisfaction to team members who don’t typically work on projects so closely tied to human health.

SOURCE

https://hms.harvard.edu/news/screen-door-opens?utm_source=Silverpop&utm_medium=email&utm_term=field_news_item_1&utm_content=HMNews02012021

https://pharmaceuticalintelligence.com/2020/07/28/14th-annual-biopharma-healthcare-summit-friday-september-4-2020-8-am-est-to-3-30-pm-est-virtual-edition/

Tweet Collection by @pharma_BI and @AVIVA1950 and Re-Tweets for e-Proceedings 14th Annual BioPharma & Healthcare Summit, Friday, September 4, 2020, 8 AM EST to 3-30 PM EST – Virtual Edition

Posted in Big Data, BioSimilars, BioTechnology - Venture Creation, BioTechnology - Venture Creation, Venture Capital, Conference Coverage with Social Media, drug repurposing, Pharmaceutical Analytics, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment on September 4, 2020| Leave a Comment »

Tweet Collection by @pharma_BI and @AVIVA1950 and Re-Tweets for e-Proceedings 14th Annual BioPharma & Healthcare Summit, Friday, September 4, 2020, 8 AM EST to 3-30 PM EST – Virtual Edition

Reporter: Aviva Lev-Ari, PhD, RN

Real Time Press Coverage: Aviva Lev-Ari, PhD, RN

e-Proceedings 14^th Annual BioPharma & Healthcare Summit, Friday, September 4, 2020, 8 AM EST to 3-30 PM EST – Virtual Edition

Real Time Press Coverage: Aviva Lev-Ari, PhD, RN

Founder & Director, LPBI Group

Aviva Lev-Ari

@AVIVA1950

25m

#USAIC20 Dr. Hal Barron, Chief Scientific Officer and President R&D, GlaxoSmithKline GWAS not easy to find which gene drives the association Functional Genomics gene by gene with phenotypes using machine learning significant help

Aviva Lev-Ari

@AVIVA1950

44m

#USAIC20 Dr. Hal Barron, Chief Scientific Officer and President R&D, GSK GWAS not easy to find which gene drives the association Functional Genomics gene by gene with phenotypes using machine learning significant help

Srihari Gopal

@sgopal2

Enjoyed hearing enthusiasm for Neuroscience R&D by Roy Vagelos at #USAIC20. Wonderful interview by Mathai Mammen

@JanssenGlobal

Show this thread

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Nina Kjellson, General Partner, Canaan Data science is a winner in Healthcare Women – Data Science is an excellent match

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Arpa Garay, President, Global Pharmaceuticals, Commercial Analytics, Merck & Co. Data on Patients and identification who will benefit fro which therapy cultural bias risk aversion

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Najat Khan, Chief Operating Officer, Janssen R&D Data Sciences, Johnson & Johnson Data Validation Deployment of algorithms embed data by type early on in the crisis to understand the disease

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Sastry Chilukuri, President, Acorn AI- Medidata Opportunities in Data Science in Paharma COVID-19 and Data Science

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Maya Said, Chief Executive Officer, Outcomes4Me Cancer patients taking change of their care Digital Health – consumerization of Health, patient demand to be part of the decision, part the information FDA launched a Program Project Patient Voice

USAIC

@USAIC

We’re taking a quick break at #USAIC20 before our next panel on rare diseases starts at 12:20pm EDT. USAIC would like to thank our Sponsors and Partners for supporting this year’s digital event.

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Roy Vagelos, Chairman of the Board, Regeneron HIV-AIDS: reverse transcriptase converted a lethal disease to a chronic disease, tried hard to make vaccine – the science was not there

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Roy Vagelos, Chairman of the Board, Regeneron Pharmaceuticals Congratulates Big Pharma for taking the challenge on COVID-19 Vaccine, Antibody and anti-viral Government funding Merck was independent from Government – to be able to set the price

Dr Kapil Khambholja

@kapilmk

Christopher Viehbacher, Gurnet Point Capital touches very sensitive topic at #USAIC20 He claims that we are never going to have real innovation out of big pharma! Well this isn’t new but not entirely true either… any more thoughts?

Show this thread

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Daphne Zohar, Founder & CEO, PureTech Health Disease focus, best science is the decision factors

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Christopher Viehbacher, Managing Partner, Gurnet Point Capital Dream of every Biotech – get Big Pharma coming to acquire and pay a lot Morph and adapt

anju ghangurde

@scripanjug

Biogen’s chair Papadopoulos big co mergers is an attempt to solve problems; typically driven by patent expirations.. #usaic20

@USAIC

anju ghangurde

@scripanjug

Chris Viehbacher/Gurnet Point Capital on US election: industry will work with whoever wins; we’ll have to ‘morph & adapt’ #usaic20

@USAIC

Dr Kapil Khambholja

@kapilmk

@daphnezohar

@PureTechH

talks about various philosophies and key reasons why certain projects/molecules are killed early. My counter questions- What are chances of losing hope little early? Do small #biopharma publish negative results to aid to the knowledge pool? #USAIC20

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Aviva Lev-Ari

@AVIVA1950

#USAIC20 Rehan Verjee, President, EMD Serono Antibody cytoxic avoidance with precision

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Laurie Glimcher, President & CEO, Dana-Farber Cancer Institute DNA repair and epignetics are the future of medicine

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Laurie Glimcher, President & CEO, Dana-Farber Cancer Institute COlonorectal cancer is increasing immuno therapy 5 drugs marketed 30% cancer patients are treated early detection key vs metastatic 10% of cancer are inherited treatment early

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Rehan Verjee, President, EMD Serono Charities funding cancer research – were impacted and resources will come later and in decreased amount New opportunities support access to Medicine improve investment across the board

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Philip Larsen, Global Head of Research, Bayer AG Repurposing drugs as antiviral from drug screening innovating methods Cytokine storm in OCVID-19 – kinase inhibitors may be antiviral data of tested positive allows research of pathway in new ways

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Laurie Glimcher, President & CEO, Dana-Farber 3,000 Telemedicine session in the first week of the Pandemic vs 300 before – patient come back visits patient happy with Telemedicine team virtually need be reimbursed same rate working remotely

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Raju Kucherlapati, Professor of Genetics, Harvard Medical School New normal as a result of the pandemic role of personalized medicine

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Rehan Verjee, President, EMD Serono entire volume of clinical trials at Roche went down same at EMD delay of 6 month, some were to be initiated but was put on hold Charities funding cancer research were impacted and resources will come later smaller

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Laurie Glimcher, President & CEO, Dana-Farber Cancer Institute Dana Farber saw impact of COVID-19 on immunosuppressed patients coming in for Cancer Tx – switch from IV Tx to Oral 96% decrease in screenings due to Pandemic – increase with Cancer

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Kenneth Frazier, Chairman of the Board and Chief Executive Officer, Merck & Co. Pharma’s obligation for next generations requires investment in R&D vs Politicians running for 4 years Patients must come first vs shareholders vs R&D investment in 2011

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Kenneth Frazier, Chairman of the Board and Chief Executive Officer, Merck & Co. Antibiotic research at Merck – no market incentives on pricing for Merck to invest in antibiotics people will die from bacterial resistance next pandemic be bacterial

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Kenneth Frazier, Chairman of the Board and Chief Executive Officer, Merck & Co. Strategies of Merck = “Medicine is for the People not for Profit” – Ketruda in India is not reembureable in India and million are in need it Partnership are encouraged

Dr Kapil Khambholja

@kapilmk

@biogen

Chairman Stelios Papadopoulos asks #KennethFrazier if wealthy nations will try to secure large proportion of #COVID19 drugs/vaccines. #KennethFrazie rightly mentions: pharma industry’s responsibility to balance the access to diff countries during pandemic. #USAIC20

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USAIC

@USAIC

Merck CEO Ken Frazier interviewed by Biogen Chairman Stelios Papadopoulos: #COVID19 vaccine product profile should be 1 dose that can be deployed globally and administered easily #USAIC20 $MRK $BIIB usaindiachamber.org/agenda.php

Dr Kapil Khambholja

@kapilmk

Almost 60% participants at #USAIC20 feel that MNCs are more likely to run their #clinicalTrials in #INDIA seeing changing environment here, reveals the poll. Exciting time ahead for scientific fraternity as this can substantially increase the speed of #DrugDevelopment globally

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Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Barry Bloom, Professor & former Dean, Harvard School of Public Health Vaccine in clinical trials, public need to return for 2nd shot, hesitancy Who will get the Vaccine first in the US most vulnerable of those causing transmission Pharma’s risk

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr. Barry Bloom, Professor & former Dean, Harvard School of Public Health Testing – PCR expensive does not enable quick testing is expensive result come transmission occurred Antibody testing CRISPR test based Vaccine in clinical trials

Aviva Lev-Ari

@AVIVA1950

#USAIC20 Dr Andrew Plump, President of R&D, Takeda Pharmaceuticals COllaboration effort around the Globe in the Pandemic therapy solutions including Vaccines

Posted in Coronavirus Gene Expression, COVID-19, Drug Development Process, Drug Discovery Chemistry, drug repurposing, Personal Health Applications: Tech Innovations serves HealhCare, Personalized and Precision Medicine & Genomic Research, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacogenomics, Pharmacovigilance, Population Health Management, Population Health Management, Genetics & Pharmaceutical, SARS-CoV-2, Serology tests for coronavirus antibodies, Vaccinology, Virus Infective Acute Respiratory Syndrome: SARS-CoV on May 18, 2020| Leave a Comment »

The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Partnership on May 18, 2020

The Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) Partnership on May 18, 2020: Leadership of AbbVie, Amgen, AstraZeneca, Bristol Myers Squibb, Eisai, Eli Lilly, Evotec, Gilead, GlaxoSmithKline, Johnson & Johnson, KSQ Therapeutics, Merck, Novartis, Pfizer, Roche, Sanofi, Takeda, and Vir. We also thank multiple NIH institutes (especially NIAID), the FDA, BARDA, CDC, the European Medicines Agency, the Department of Defense, the VA, and the Foundation for NIH

Reporter: Aviva Lev-Ari, PhD, RN

May 18, 2020

Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) An Unprecedented Partnership for Unprecedented Times

Francis S. Collins, MD, PhD¹; Paul Stoffels, MD²

Article Information

JAMA. Published online May 18, 2020. doi:10.1001/jama.2020.8920

Author Affiliations

¹Office of the Director, National Institutes of Health, Bethesda, Maryland

²Johnson & Johnson, New Brunswick, New Jersey

It has been more than a century since the world has encountered a pandemic like coronavirus disease 2019 (COVID-19), and the rate of spread of COVID-19 around the globe and the associated morbidity and mortality have been staggering.¹ To address what may be the greatest public health crisis of this generation, it is imperative that all sectors of society work together in unprecedented ways, with unprecedented speed. In this Viewpoint, we describe such a partnership.

First reported in Wuhan, China, in December 2019, COVID-19 is caused by a highly transmissible novel coronavirus, SARS-CoV-2 (severe acute respiratory syndrome coronavirus 2). By March 2020, as COVID-19 moved rapidly throughout Europe and the US, most researchers and regulators from around the world agreed that it would be necessary to go beyond “business as usual” to contain this formidable infectious agent. The biomedical research enterprise was more than willing to respond to the challenge of COVID-19, but it soon became apparent that much-needed coordination among important constituencies was lacking.

Clinical trials of investigational vaccines began as early as January, but with the earliest possible distribution predicted to be 12 to 18 months away. Clinical trials of experimental therapies had also been initiated, but most, except for a trial testing the antiviral drug remdesivir,² were small and not randomized. In the US, there was no true overarching national process in either the public or private sector to prioritize candidate therapeutic agents or vaccines, and no efforts were underway to develop a clear inventory of clinical trial capacity that could be brought to bear on this public health emergency. Many key factors had to change if COVID-19 was to be addressed effectively in a relatively short time frame.

On April 3, leaders of the National Institutes of Health (NIH), with coordination by the Foundation for the National Institutes of Health (FNIH), met with multiple leaders of research and development from biopharmaceutical firms, along with leaders of the US Food and Drug Administration (FDA), the Biomedical Advanced Research and Development Authority (BARDA), the European Medicines Agency (EMA), and academic experts. Participants sought urgently to identify research gaps and to discuss opportunities to collaborate in an accelerated fashion to address the complex challenges of COVID-19.

These critical discussions culminated in a decision to form a public-private partnership to focus on speeding the development and deployment of therapeutics and vaccines for COVID-19. The group assembled 4 working groups to focus on preclinical therapeutics, clinical therapeutics, clinical trial capacity, and vaccines (Figure). In addition to the founding members, the working groups’ membership consisted of senior scientists from each company or agency, the Centers for Disease Control and Prevention (CDC), the Department of Veterans Affairs (VA), and the Department of Defense.

Figure.

Accelerating COVID-19 Therapeutic Interventions and Vaccines

NIH-ACTIV’s Working Groups

ACTIV’s 4 working groups, each with one cochair from NIH and one from industry, have made rapid progress in establishing goals, setting timetables, and forming subgroups focused on specific issues (Figure). The goals of the working group, along with a few examples of their accomplishments to date, include the following.

The Preclinical Working Group was charged to standardize and share preclinical evaluation resources and methods and accelerate testing of candidate therapies and vaccines to support entry into clinical trials. The aim is to increase access to validated animal models and to enhance comparison of approaches to identify informative assays. For example, through the ACTIV partnership, this group aims to extend preclinical researchers’ access to high-throughput screening systems, especially those located in the Biosafety Level 3 (BSL3) facilities currently required for many SARS-CoV-2 studies. This group also is defining a prioritization approach for animal use, assay selection and staging of testing, as well as completing an inventory of animal models, assays, and BSL 3/4 facilities.

The Therapeutics Clinical Working Group has been charged to prioritize and accelerate clinical evaluation of a long list of therapeutic candidates for COVID-19 with near-term potential. The goals have been to prioritize and test potential therapeutic agents for COVID-19 that have already been in human clinical trials. These may include agents with either direct-acting or host-directed antiviral activity, including immunomodulators, severe symptom modulators, neutralizing antibodies, or vaccines. To help achieve these goals, the group has established a steering committee with relevant expertise and objectivity to set criteria for evaluating and ranking potential candidate therapies submitted by industry partners. Following a rigorous scientific review, the prioritization subgroup has developed a complete inventory of approximately 170 already identified therapeutic candidates that have acceptable safety profiles and different mechanisms of action. On May 6, the group presented its first list of repurposed agents recommended for inclusion in ACTIV’s master protocol for adaptive clinical trials. Of the 39 agents that underwent final prioritization review, the group identified 6 agents—including immunomodulators and supportive therapies—that it proposes to move forward into the master protocol clinical trial(s) expected to begin later in May.

The Clinical Trial Capacity Working Group is charged with assembling and coordinating existing networks of clinical trials to increase efficiency and build capacity. This will include developing an inventory of clinical trial networks supported by NIH and other funders in the public and private sectors, including contract research organizations. For each network, the working group seeks to identify their specialization in different populations and disease stages to leverage infrastructure and expertise from across multiple networks, and establish a coordination mechanism across networks to expedite trials, track incidence across sites, and project future capacity. The clinical trials inventory subgroup has already identified 44 networks, with access to adult populations and within domestic reach, for potential inclusion in COVID-19 trials. Meanwhile, the survey subgroup has developed 2 survey instruments to assess the capabilities and capacities of those networks, and its innovation subgroup has developed a matrix to guide deployment of innovative solutions throughout the trial life cycle.

The Vaccines Working Group has been charged to accelerate evaluation of vaccine candidates to enable rapid authorization or approval.⁴ This includes development of a harmonized master protocol for adaptive trials of multiple vaccines, as well as development of a trial network that could enroll as many as 100 000 volunteers in areas where COVID-19 is actively circulating. The group also aims to identify biomarkers to speed authorization or approval and to provide evidence to address cross-cutting safety concerns, such as immune enhancement. Multiple vaccine candidates will be evaluated, and the most promising will move to a phase 2/3 adaptive trial platform utilizing large geographic networks in the US and globally.⁵ Because time is of the essence, ACTIV will aim to have the next vaccine candidates ready to enter clinical trials by July 1, 2020.

References

Desai A . Twentieth-century lessons for a modern coronavirus pandemic. JAMA. Published online April 27, 2020. doi:10.1001/jama.2020.4165
Article PubMed Google Scholar

NIH clinical trial shows remdesivir accelerates recovery from advanced COVID-19. National Institutes of Health. Published April 29, 2020. Accessed May 7, 2020. https://www.nih.gov/news-events/news-releases/nih-clinical-trial-shows-remdesivir-accelerates-recovery-advanced-covid-19

NIH to launch public-private partnership to speed COVID-19 vaccine and treatment options. National Institutes of Health. Published April 17, 2020. Accessed May 7, 2020. https://www.nih.gov/news-events/news-releases/nih-launch-public-private-partnership-speed-covid-19-vaccine-treatment-options

Corey L , Mascola JR , Fauci AS , Collins FS . A strategic approach to COVID-19 vaccine R&D. Science. Published online May 11, 2020. doi:10.1126/science.abc5312 PubMed Google Scholar

Angus DC . Optimizing the trade-off between learning and doing in a pandemic. JAMA. Published online March 30, 2020. doi:10.1001/jama.2020.4984
Article PubMed Google Scholar

Accelerating COVID-19 Therapeutic Interventions and Vaccines (ACTIV) portal. National Institutes of Health. Accessed May 15, 2020. https://www.nih.gov/ACTIV

Accelerating Medicines Partnership (AMP). National Institutes of Health. Published February 4, 2014. Accessed May 7, 2020. https://www.nih.gov/research-training/accelerating-medicines-partnership-amp

SOURCE

https://jamanetwork.com/journals/jama/fullarticle/2766371?guestAccessKey=5defc755-e585-47e5-b79a-fee2ec2dd42b&utm_source=For_The_Media&utm_medium=referral&utm_campaign=ftm_links&utm_content=tfl&utm_term=051820

https://advdrug.com/agenda/

Advancing Drug Development – 12/12/2019, 8:30AM – 8:30PM at The University of Massachusetts Club, One Beacon Street, Boston, MA

Posted in Drug Delivery Platform Technology, Drug Development Process, Drug Discovery Chemistry, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, Pharmacogenomics, Rapid automation of plasma protein pools on November 4, 2019| Leave a Comment »

Advancing Drug Development – 12/12/2019, 8:30AM – 8:30PM at The University of Massachusetts Club, One Beacon Street, Boston, MA

Reporter: Aviva Lev-Ari, PhD, RN

4th Advancing Drug Development Forum – Making the Impossible Possible – Harnessing Small Molecule Drug Development scheduled to take place December 12th, 2019 at The University of Massachusetts Club, in Boston, Massachusetts from 8:30 AM – 8:30 PM.

http://advdrug.com/agenda/

Scientists are more than just chipping away and kicking down the barricades to develop complex small molecule products better and faster. Successful companies are spending quality time finding novel and clever approaches and powerful technologies to better support their knowledgeable teams. Often it takes establishing strong partnerships with 1 or more specialized service providers, cleverly combining resources – always striving to raise the bar in order to make life threatening diseases more of a chronic and tolerable disease or eradicated completely.

Hear from key opinion leaders in pharma, biotech, the investment community and innovative service providers on how they are meeting the challenges. Keep in mind, it takes being open-minded, flexible and willing sometimes to redesigning a new formulation that better enhances bioavailability, optimizes drug-delivery profiles, reduces dosing frequency, or improves the patient experience to have the potential to deliver quicker returns on investments than developing a completely new drug.

PROGRAM AGENDA Thursday, December 12, 2019

8:30 AM Registration and Networking Continental Breakfast

9:00 AM Welcome Address and Opening Remarks
Kevin Bittorf, Ph.D., & Shelly Amster

9:15 AM Opening VC Keynote

9:45 AM Bridging the Gap between Experimentation and Implementation
Panel Discussion

10:15 AM Refreshment Break

10:45 AM Cross-Talk Between Clin-Ops and Tech-Ops
Panel Discussion

11:15 AM The Cost of Speed and Value in Drug Development
Panel Discussion

12:00 PM Networking Luncheon

1:00 PM Advances in the Delivery of Therapeutics to the Brain
Academic Keynote
Mansoor M. Amiji, Ph.D., University Distinguished Professor, Professor of Pharmaceutical Sciences & Professor of Chemical Engineering, Northeastern University

1:30 PM Advancing Drug Delivery and Controlled Release
Panel Discussion

2:00 PM Drowning in DATA

2:30 PM Disruptive AI Technologies Improving Drug Development

3:00 PM Refreshment Break

3:30 PM Small Specialty VS Full Service – What Makes Sense for US?
Panel Discussion

4:00 PM Fireside Chat
Michael Bonney, Executive Chair, Kaleido Biosciences
Heinrich Schlieker, Ph.D., SVP Technical Operations, Sage Therapeutics

5:00 PM – 8:00 PM Networking Social

PROGRAM AGENDA	Thursday, December 12, 2019
8:30 AM		Registration and Networking Continental Breakfast
9:00 AM		Welcome Address and Opening Remarks Kevin Bittorf, Ph.D., & Shelly Amster
9:15 AM		Opening VC Keynote
9:45 AM		Bridging the Gap between Experimentation and Implementation Panel Discussion
10:15 AM		Refreshment Break
10:45 AM		Cross-Talk Between Clin-Ops and Tech-Ops Panel Discussion
11:15 AM		The Cost of Speed and Value in Drug Development Panel Discussion
12:00 PM		Networking Luncheon
1:00 PM		Advances in the Delivery of Therapeutics to the Brain Academic Keynote Mansoor M. Amiji, Ph.D., University Distinguished Professor, Professor of Pharmaceutical Sciences & Professor of Chemical Engineering, Northeastern University
1:30 PM		Advancing Drug Delivery and Controlled Release Panel Discussion
2:00 PM		Drowning in DATA
2:30 PM		Disruptive AI Technologies Improving Drug Development
3:00 PM		Refreshment Break
3:30 PM		Small Specialty VS Full Service – What Makes Sense for US? Panel Discussion
4:00 PM		Fireside Chat Michael Bonney, Executive Chair, Kaleido Biosciences Heinrich Schlieker, Ph.D., SVP Technical Operations, Sage Therapeutics
5:00 PM – 8:00 PM		Networking Social

SOURCE

Direct electronic communication with Shelly Amster

Erin McCallisterSenior Editor at BioCentury

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

Posted in Big Data & Analytics, Data Science, Pharmaceutical R&D Informatics, REAL TIME Conference Coverage Twitter's Hashtags and Handles per Presentation/session, Scientific & Biotech Conferences: Press Coverage, Transcriptomics, tagged #pharmaIT, AI, Artificial Intelligence & Clinical Trials, Big data, Machine Learning, Neurological Disorders, Personalized and Precision Medicine & Genomic Research, Personalized medicine, precision medicine on June 5, 2019| Leave a Comment »

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

Reporter: Stephen J Williams PhD @StephenJWillia2

Speakers

Jennifer CozzoneGlobal Personalised Healthcare Lead, Specialty Care atRoche

Aurélie DeleforgePrecision Medicine Practice Lead at Bionest Partners

Ms. elizabeth DonleyCEO at NeuroPointDX

Precision Medicine has helped transform cancer care from one-size-fits-all chemotherapy to a new era, where patients’ tumors can be analyzed and therapy selected based on their genetic makeup. Until now, however, precision medicine’s impact has been far less in other therapeutic areas, many of which are ripe for transformation. Efforts are underway to bring the successes of precision medicine to neurology, immunology, ophthalmology, and other areas. This move raises key questions of how the lessons learned in oncology can be used to advance precision medicine in other fields, what types of data and tools will be important to personalizing treatment in these areas, and what sorts of partnerships and payer initiatives will be needed to support these approaches and their ultimate commercialization and use. The panel will also provide an in depth look at precision medicine approaches aimed at better understanding and improving patient care in highly complex disease areas like neurology.

Speaker panel: The big issue now with precision medicine is there is so much data and hard to put experimental design and controls around randomly collected data.

The frontier is how to CURATE randomly collected data to make some sense of it
One speaker was at a cancer meeting and the oncologist had no idea what to make of genomic reports they were given. Then there is a lack of action or worse a misdiagnosis.
So for e.g. with Artificial Intelligence algorithms to analyze image data you can see things you can’t see with naked eye but if data quality not good the algorithms are useless – if data not curated properly data is wasted

Data needs to be organized and curated.

If relying of AI for big data analysis the big question still is: what are the rates of false negative and false positives? Have to make sure so no misdiagnosis.

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

@Handles

@BIOConvention

# Hashtags

#BIO2019 (official meeting hashtag)

#itstartswithone

#RightMixMatters

Richard SollSenior Advisor, Strategic Initiatives at WuXi AppTec

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

Posted in Computational Biology/Systems and Bioinformatics, Conference Coverage with Social Media, Global Partnering & Biotech Investment, Investment in Technological Breakthrough, Life Sciences Breakthrough Prize, Pharmaceutical Discovery, Pharmaceutical Drug Discovery, Pharmaceutical Industry Competitive Intelligence, Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment, tagged academic advancement, Academic publishing, basic research, Big data, collaboration, data validity, Innovation, Pharmaceutical industry, Public–private partnership, research funding, tech transfer on June 3, 2019| Leave a Comment »

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

Reporter: Stephen J. Williams, PhD @StephenJWillia2

Article ID #267: Real Time Coverage @BIOConvention #BIO2019: Issues of Risk and Reproduceability in Translational and Academic Collaboration; 2:30-4:00 June 3 Philadelphia PA. Published on 6/3/2019

WordCloud Image Produced by Adam Tubman

Derisking Academic Science: The Unmet Need

Translating academic research into products and new therapies is a very risky venture as only 1% of academic research has been successfully translated into successful products.

Speakers

Jim AudiaExecutive Director atChicago Biomedical Consortium

Collaboration from Chicago area universities like U of Chicago, Northwestern, etc. First phase was enhance collaboration between universities by funding faculty recruitment and basic research. Access to core facilities across universities. Have expanded to give alternatives to company formation.

Curtis KeithCSO atBlavatnik Biomedical Accelerator, Harvard University

Half of the partnerships from Harvard and companies have been able to spin out viable startups.

Nadim ShohdyAssistant Dean, Therapeutics Alliances atNYU Langone Health

Most academic PI are not as savvy to start a biotech so they bring in biotechs and build project teams as well as developing a team of ex pharma and biotech experts. Derisk as running as one asset project. Partner as early as possible. A third of their pipeline have been successfully partnered. Work with investors and patent attorneys.

Dominique VerhelleSr Director, Strategic Academic Alliances, Center for External Innovation atTakeda

Focused on getting PIs to get to startup. Focused on oncology and vaccines and I/O. The result can be liscensing or partnership. Running around 50 to 60 projects. Creating a new company from these US PI partnerships.

Most projects from Harvard have been therapeutics-based. At Harvard they have a network of investors ($50 million). They screen PI proposals based on translateability and what investors are interested in.

In Chicago they solicit multiple projects but are agnostic on area but as they are limited they are focused on projects that will assist in developing a stronger proposal to investor/funding mechanism.

NYU goes around university doing due diligence reaching out to investigators. They shop around their projects to wet their investors, pharma appetite future funding. At Takeda they have five centers around US. They want to have more input so go into the university with their scientists and discuss ideas.

Challenges:

Takeda: Data Validation very important. Second there may be disconnect with the amount of equity the PI wants in the new company as well as management. Third PIs not aware of all steps in drug development. Harvard: Pharma and biotech have robust research and academic does not have the size or scope of pharma. PIs must be more diligent on e.g. the compounds they get from a screen… they only focus narrowly NYU: bring in consultants as PIs don’t understand all the management issues. Need to understand development so they bring in the experts to help them. Pharma he feels have to much risk aversion and none of their PIs want 100% equity. Chicago: they like to publish at early stage so publication freedom is a challenge

It Starts with One Health: A Guide to Translational Science from Research to Investment

115B, Level 100

Speakers

William KareshExecutive Vice President for Health and Policy atEcoHealth Alliance

Marianne de backerVP, Global M&A Operations and Divestitures (Pharmaceuticals Group), Global Infectious Diseases & Vaccines Business Development atJohnson & Johnson Innovation

Randal J. KirkChief Executive Officer atIntrexon

Dr. Hilary MarstonMedical Officer/Policy Advisor, Office of the Director, NIAID atNational Institute of Health

Tina ParkerProgram Officer – Translational Centers of Excellence and Research Coordination Section Office of Biodefense, Research Resources and Translational Research, DMID/NIAID atNIH

Novel Approaches to Improve Reproducibility in Academia-Industry Collaborations

112AB, Level 100

Speakers

Florence BietrixOperations Manager at EATRIS

Dr. Nathan CoussensSenior Research Scientist at National Center for Advancing Translational Sciences, National Institutes of Health

Leonard FreedmanCSO atFrederick National Laboratory for Cancer Research

Marian Hajduch Scientific Director at IMTM

Weida TongDirector Division of Bioinformatics and Biostatistics at NCTR/FDA

Dr. Freedman: Most scientists responding to Nature survey said yes a reproduceability crisis. The reasons: experimental bias, lack of validation techniques, reagents, and protocols etc.

And as he says there is a great ECONOMIC IMPACT of preclinical reproducability issues: to the tune of $56 billion of irreproducable results (paper published in PLOS Biology). If can find the core drivers of this issue they can solve the problem. STANDARDS are constantly used in various industries however academic research are lagging in developing such standards. Just the problem of cell line authentication is costing $4 billion.

Dr. Cousins: There are multiple high throughput screening (HTS) academic centers around the world (150 in US). So where does the industry go for best practices in assays? Eli Lilly had developed a manual for HTS best practices and in 1984 made publicly available (Assay Guidance Manual). To date there have been constant updates to this manual to incorporate new assays. Workshops have been developed to train scientists in these best practices.

NIH has been developing new programs to address these reproducability issues. Developed a method called

“Ring Testing Initiative” where multiple centers involved in sharing reagents as well as assays and allowing scientists to test at multiple facilities.

Dr.Tong: Reproduceability of Microarrays: As microarrays were the only methodology to do high through put genomics in the early 2000s, and although much research had been performed to standardize and achieve best reproduceability of the microarray technology (determining best practices in spotting RNA on glass slides, hybridization protocols, image analysis) little had been done on evaluating the reproducibility of results obtained from microarray experiments involving biological samples. The advent of Artificial Intelligence and Machine Learning though can be used to help validate microarray results. This was done in a Nature Biotechnology paper (Nature Biotechnology volume28, 827–838 (2010)) by an international consortium, the International MAQC (Microarray Quality Control) Society and can be found here

The MicroArray Quality Control (MAQC)-II study of common practices for the development and validation of microarray-based predictive models

However Dr. Tong feels there is much confusion in how we define reproduceability. Dr. Tong identified a few key points of data reproduceability:

Traceability: what are the practices and procedures from going from point A to point B (steps in a protocol or experimental design)
Repeatability: ability to repeat results within the same laboratory
Replicatablilty: ability to repeat results cross laboratory
Transferability: are the results validated across multiple platforms?

The panel then discussed the role of journals and funders to drive reproduceability in research. They felt that editors have been doing as much as they can do as they receive an end product (the paper) but all agreed funders need to do more to promote data validity, especially in requiring that systematic evaluation and validation of each step in protocols are performed.. There could be more training of PIs with respect to protocol and data validation.

Biotechnology & Pharma: 2018 Investment Budget in R&D – Top Ten Companies, Top R&D% AZ, Merck, BMS, Eli Lilly

Posted in Pharmaceutical R&D Informatics, Pharmaceutical R&D Investment on June 3, 2019| Leave a Comment »

Biotechnology & Pharma: 2018 Investment Budget in R&D – Top Ten Companies, Top R&D% AZ, Merck, BMS, Eli Lilly

Reporter: Aviva Lev-Ari, PhD, RN

AstraZeneca Budget 5,932,000,000 R&D 5,932,000,000 27.0%

Merck Budget 9,750,000,000 R&D 9,750,000,000 23.0%

BMS Budget 6,345,000,000 R&D 6,345,000,000 23.0%

Eli Lilly Budget 5,307,100,000 R&D 5,307,100,000 22.5%

The top 10 pharma R&D budgets in 2018

Annual pharma R&D budgets

Company	Measure Names	SUM(Budget (copy))	SUM(Budget)	SUM(R&D as percentage of revenue)
GlaxoSmithKline	Budget	5,196,000,000	5,196,000,000	12.6
Eli Lilly	Budget	5,307,100,000	5,307,100,000	22.5
AstraZeneca	Budget	5,932,000,000	5,932,000,000	27.0
Bristol-Myers Squibb	Budget	6,345,000,000	6,345,000,000	23.0
Sanofi	Budget	6,961,000,000	6,961,000,000	17.1
Pfizer	Budget	8,006,000,000	8,006,000,000	14.9
Novartis	Budget	9,074,000,000	9,074,000,000	17.5
Merck	Budget	9,750,000,000	9,750,000,000	23.0
Johnson & Johnson	Budget	10,800,000,000	10,800,000,000	13.2
Roche	Budget	11,060,000,000	11,060,000,000	19.3

Showing first 20 rows.

Download all rows as a text file

SOURCE

https://www.fiercebiotech.com/special-report/top-10-pharma-r-d-budgets-2018?mkt_tok=eyJpIjoiWkRCbE1XSTBaakJtTXpBeCIsInQiOiJUQjZNNkJRaWxEbkpWK25GV3lFY0NZSWI3ZFg4WHlrSmJuaVRMQlo4dlJnY1ZhaFJidDdtSHdqcytTZXNTODI5b1laRWNDXC9cL1FQOUN1RkJWdnZTZXFZOFhwaU80MlpHdlFhMlR0VHd3VHdpemFGZzlucXFNSVdsOGVrZUdNK2ZmIn0%3D&mrkid=993697