Category Archives: Genetic medicine

Did you know that viruses can kill bacteria? It's true and the process holds much promise for medicine.

For the first time ever, researchers successfully used bacteriophages to treat an antibiotic-resistant mycobacterial lung infection. This process led the way for a young National Jewish Health patient with cystic fibrosis to receive a life-saving lung transplant, according to a press release by the institution published on Friday.

We had tried unsuccessfully for years to clear the mycobacterial infection with a variety of antibiotics, said Jerry Nick, MD, lead author of the study and director of the Adult Cystic Fibrosis Program at National Jewish Health. When we used the bacterias own natural enemies, we were able to clear the infection which resulted in a successful lung transplant.

No one was happier with the results of the treatment than the patient himself.

I am so grateful for the effort, persistence, and creativity of all the people who were involved in my treatment, said Jarrod Johnson, recipient of the lung transplant. I thought I was going to die. They have literally saved my life.

Johnson is a 26-year-old cystic fibrosis patient who has been plagued with repeated lung infections starting from childhood and causing him to be admitted to various hospitals several times a year.

In adulthood, he suffered from a rapid decline in his lung function due to a persistentMycobacterium abscessusinfection over a six-year period that caused doctors to believe he was likely to die in only a few years without a lung transplant. However, hehad been refused transplants by three transplant centers because of his continuous mycobacterial infection.

That's when Nick and his team decided to treat him with phages (viruses that attack bacteria) for the first time ever. The treatment ran for 200 days but proved so successful that Johnson was ready to undergo a lung transplant.

This research can serve as a roadmap for future use of phages to treat patients with severeMycobacterium abscessuslung infection and to save lives, concluded Nick.

Abstract:Two mycobacteriophages were administered intravenously to a male with treatment-refractoryMycobacterium abscessuspulmonary infection and severe cystic fibrosis lung disease. The phages were engineered to enhance their capacity to lyseM.abscessusand were selected specifically as the most effective against the subjects bacterial isolate. In the setting of compassionate use, the evidence of phage-induced lysis was observed using molecular and metabolic assays combined with clinical assessments.M.abscessusisolates pre and post-phage treatment demonstrated genetic stability, with a general decline in diversity and no increased resistance to phage or antibiotics. The anti-phage neutralizing antibody titers to one phage increased with time but did not prevent clinical improvement throughout the course of treatment. The subject received lung transplantation on day 379, and systematic culturing of the explanted lung did not detectM.abscessus. This study describes the course and associated markers of a successful phage treatment ofM.abscessusin advanced lung disease.

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Researchers have used a virus to fight a drug-resistant lung infection - Interesting Engineering

For countries that could get their hands on it, the rapid development of mRNA vaccines for COVID-19 was something close to a miracle, saving an estimated 1.1 million lives in the United States alone.

By capitalizing on recent developments in gene sequencing, chemical synthesis and new delivery mechanisms, scientists were able to start the first clinical trials for mRNA vaccines just four months after the World Health Organization declared a pandemic. This success, industry observers say, could act as a kind of blueprint to tackle other hard-to-treat diseases.

It was really the development of several technologies coming together that put us in a position where we were able to respond very, very quickly, says Richard Bozzato, a senior adviser on health at MaRS. It showed we can do it.

Researchers are using these advances to speed up all aspects of drug development from discovery to how a medication is administered with the potential to cut costs, save time and help more patients. The whole objective here, says Bozzato, is to be able to cure as many patients as we can.

Using AI to speed up drug development

Fewer than 10 per cent of new compounds ever make it from the test tube to clinical use. Most turn out to be either less effective or more toxic than originally believed. Even then, many drugs that are approved can have serious side effects.

Right now, the process of discovery and development is hit and miss, says Bozzato. You do an experiment and see what happens. Then you discover something you didnt expect. This is where artificial intelligence and machine learning can help: by running tests through computational models, they can identify issues as well as promising new compounds much faster.

If you can combine the speed of computational approaches with the intuition and brilliance of the human mind, you can start asking different questions for a different problem, says Naheed Kurji, co-founder, president and CEO of Toronto-based Cyclica. It means more medicines, better medicines faster. We wont have to wait 12 years to get them.

Researchers have been developing computational techniques to pinpoint the molecular drivers of disease for more than 20 years, but the area has expanded considerably in the last five years as more genetic data becomes available. A major breakthrough came last year when Googles artificial intelligence startup Deep Mind allowed free access to its AlphaFold database of three-dimensional protein structures.

The program predicts those structures with more than 90 per cent accuracy twice the accuracy of previous programs. Work that would have taken months or years can now be accomplished in a few hours or days, says Bozzato.

More than 400 companies worldwide are now working in the space, targeting diseases from Alzheimers and Parkinsons disease to various forms of cancer. Cyclica is looking into new treatments for a range of ailments that are hard to treat, including some central nervous system disorders such as pain and spinal disease. And Deep Genomics, another Toronto-based venture, is preparing its first AI-discovered drug programs for clinical trials.

Finding a better delivery mechanism

Medications are only effective if they end up in the right place, at the right time. When theyre on target, they can treat disease in the affected tissues with minimal side effects. Researchers have been developing safe and effective drug delivery technologies for decades, but new, more complex therapies such as mRNA and gene editing need more complex delivery systems. Thats where lipid nanoparticles come in.

Lipid nanoparticles are the tiny envelopes that deliver the active ingredient in mRNA COVID vaccines. Their success in the mRNA vaccines suggests they have much greater potential to safely deliver other innovative therapies, such as small drugs, proteins or genetic material.

Seeing the safety and efficacy of these vaccines was proof you could use this for a new modality, says Brent Stead, co-founder and chief executive officer of Specific Biologics, a discovery-stage gene-editing company thats experimenting with lipid nanoparticles to deliver gene therapy. If it works in this application, then fundamentally theres no reason it cannot work in others.

The particles, which are fatty molecules that closely resemble human cells, act as a protective layer for small molecule therapies as they make their way to their target cells. Their close resemblance to human cells means they can evade the bodys natural defences, making them non-toxic. Theyre especially effective at reaching specific organs or cells because they can have chemical structures attached to their surface that recognize unique molecules on their targets.

Researchers are looking to see whether lipid nanoparticles can be used to deliver the CRISPR gene-editing technology to target rare diseases that are caused by a single genetic mutation, such as ALS, Huntingtons Disease and ocular diseases that cause blindness. You can envision a treatment where you do the treatment once and that permanently corrects the disease, says Stead.

California-based Intellia Therapeutics recently started the first clinical safety trials using the combination to target liver diseases, and Specific Biologics is doing laboratory research on illnesses that affect the lungs, such as cystic fibrosis.

Treatments are still several years away, however. Although CRISPR works in animal models, researchers are still determining how to get it to consistently target a specific genetic mutation without damaging other cells. Another challenge, Stead says, is the fact that lipid nanoparticles tend to accumulate in the liver, making them most effective for targeting liver disease. Biotech companies are now working on next-generation LNPs that target different tissues and organs.

Creating a tailored treatment

One of the challenges in treating cancer is that cancer cells are constantly looking for ways to survive. They may respond initially to a treatment but then mutate and develop a resistance. Doctors then need to find a new treatment. In other cases, patients dont respond to therapy at all. Scientists are turning to next-generation sequencing to find the treatment that can be tailored to the individual patient, as well as determine why a treatment has failed.

Xue Wu, a co-founder of Geneseeq, one of the companies specializing in this field, recalls one woman who wasnt responding to traditional chemotherapy for an advanced sarcoma and was on life support in the ICU. Using next-generation sequencing, Wu discovered that a rare genetic mutation might have caused the resistance. She helped her join a clinical trial for a new drug targeting that mutation. Within 10 days, the tumour had dramatically reduced in size, and the woman lived for another 10 months before she developed drug resistance to this drug and died.

Next-generation sequencing can map hundreds of genes at a time and find the mutation in as little as five days, so that doctors can find treatments that target only those cells. Since the U.S.-based Foundation Medicine made the technique commercially available a decade ago, its application in the U.S. and other countries has grown rapidly. The technique has been especially useful in lung cancer, Wu says, for which several generations of precision treatments are available. You have the option to choose different drugs, so the patient will have a prolonged lifespan. It significantly improves the five-year survival rate of these patients.

The next frontier, Wu adds, is developing more precise treatments for other types of cancer mutations currently without viable treatment options. Researchers are now looking at whether technologies such as CRISPER gene editing and mRNA vaccines, could be deployed.

Scaling up treatments

The other big challenge for Canadian companies working to speed up drug development lies outside the laboratory, in the commercial realm, says Bozzato. The success of COVID vaccines proved that many Canadian researchers are leaders in drug development. For example, Peter Culis, now a professor emeritus at the University of British Columbia, was one of the pioneers in developing lipid nanoparticles. The B.C. company Genevent Sciences Corporation supplies LNPs to mRNA vaccine manufacturers Pfizer-BioNTech and Moderna.

However, as a recent report from the Innovation Economy Council notes, Canada doesnt currently have the large-scale manufacturing facilities that could help translate homegrown research into homegrown therapies. The federal government has started to address the issue, investing $1.2 billion in various projects, which could help Canada secure a slice of the growing global biotech industry.

The MaRS Impact Health conference, being held May 25 to 27, explores the latest innovations in biotech.

Anita Elash is a freelancer who write about technology for MaRS. Torstar, the parent company of the Toronto Star, has partnered with MaRS to highlight innovation in Canadian companies.

Disclaimer This content was produced as part of a partnership and therefore it may not meet the standards of impartial or independent journalism.

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The success of mRNA vaccines could act as a blueprint to tackle other diseases - Toronto Star

NEW YORK & CAMBRIDGE, Mass.--(BUSINESS WIRE)--Apertura Gene Therapy, a biotechnology company opening opportunities for treating debilitating diseases with limited options for patients, today announced that it has launched with a Series A financing of up to $67M from Deerfield Management Company to develop genetic medicines using platform technologies that address key limitations of genetic medicine delivery and expression. Deerfield Management has also committed additional operational support to further strengthen the companys ability to advance gene therapy discoveries.

Apertura is founded on a pair of platform technologies developed in the labs of Ben Deverman, Ph.D., Senior Director of Vector Engineering and Institute Scientist at the Broad Institute of MIT and Harvard, and Michael Greenberg, Ph.D., the Nathan Marsh Pusey Professor of Neurobiology and Chair of the Department of Neurobiology at Harvard Medical School (HMS). The companys platform leverages machine learning and high-throughput assays to engineer novel capsids, gene regulatory elements, such as promoters and enhancers, and payloads to simultaneously enhance multiple functions of gene therapies for greater translational potential.

With these platform technologies from the Broad Institute and Harvard University, Deerfield saw an opportunity to bring together and support a unique and comprehensive platform that could address technical challenges that have prevented gene therapy from reaching its full potential, said Dave Greenwald, Ph.D., Acting Chief Executive Officer of Apertura and Vice President, Business Development at Deerfield Management Company. While next-generation approaches to gene therapy have largely focused on the innovation of delivery vectors, Apertura has the potential to innovate simultaneously across delivery, expression, and payloads.

Ben Deverman, Scientific Founder of Apertura, said: When developing a gene therapy, it has been common to use naturally occurring serotype AAV capsids. The technology we have developed uses proprietary assays and machine learning to design custom AAV capsids that have the chosen characteristics for treating specific diseases, and we believe this approach will result in new and effective gene therapies.

Apertura has certain exclusive rights to AAV capsids developed in the Deverman Lab at the Broad Institute.

A separate sponsored research and licensing agreement with Harvard University, spearheaded by the Harvard Office of Technology Development, grants Apertura exclusive access to certain powerful methods of identifying cell type-specific genetic regulatory elements (GREs), including access to the Paralleled Enhancer Single-Cell Assay (PESCA) platform, developed in the Greenberg Lab at Harvard University.

A major challenge in developing effective gene therapies is having the payload of the therapy expressed at the correct level in target cells, said Greenberg. "The technology we have developed at Harvard Medical School overcomes this hurdle by targeting transgene expression to specific cell types, fine-tuning expression levels in these cells, and, at the same time, avoiding expression of the transgene in non-target cell types. The Greenberg labs PESCA platform was advanced to commercial readiness through the strategic support of the Q-FASTR program at HMS and the Blavatnik Biomedical Accelerator at Harvard University.

The two technology platforms have unique capabilities to simultaneously engineer AAV capsids to exhibit enhanced cellular tropism, evasion of pre-existing immunity, while maintaining and potentially improving manufacturability. The companys GRE platform focuses on GREs and enhancers that drive cell type-specific expression, disease state-specific expression, and tunable expression levels. These capabilities together are expected to enable Apertura to develop best-in-class gene therapies designed for specific indications.

Our platform has the potential to unlock many new indications for gene therapy, said Kristina Wang, Director of Corporate Development and Board Member of Apertura. We aim to maximize our impact through dedicated internal programs and meaningful partnerships with other biopharma companies and academic groups. Committed to advancing the field of gene therapy, Apertura seeks to collaborate broadly to accelerate impact to patients.

About Apertura Gene TherapyApertura is a biotechnology company opening opportunities for treating currently intractable diseases. We are uniquely positioned to develop genetic medicines by simultaneously engineering AAV capsids, genetic regulatory elements, and payloads to overcome limitations in cellular access, gene expression, pre-existing immunity, and manufacturability. Apertura is committed to growing the field of gene therapy and believes that together we maximize our impact by working with corporate and academic partners, patients, and foundations. Founded on technologies from the Broad Institute and Harvard University, and with support from Deerfield Management Company, the company is based at the Cure, Deerfields innovation campus in New York City. For more information, please visit our website at http://www.aperturagtx.com and follow us on LinkedIn and Twitter.

About Deerfield ManagementDeerfield is an investment management firm committed to advancing healthcare through investment, information and philanthropy. The Firm works across the healthcare ecosystem to connect people, capital, ideas and technology in bold, collaborative and inclusive ways. For more information, please visit https://deerfield.com/.

About Harvard Universitys Office of Technology DevelopmentHarvards Office of Technology Development (OTD) promotes the public good by fostering innovation and translating new inventions made at Harvard University into useful products that are available and beneficial to society. Our integrated approach to technology development comprises sponsored research and corporate alliances, intellectual property management, and technology commercialization through venture creation and licensing. More than 90 startups have launched to commercialize Harvard technologies in the past 5 years, collectively raising more than $4.5 billion in financing. To further bridge the academic-industry development gap, Harvard OTD manages the Blavatnik Biomedical Accelerator and the Physical Sciences & Engineering Accelerator. For more information, please visit https://otd.harvard.edu.

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Apertura Gene Therapy Launches with $67M Series A Financing from Deerfield and an Innovative Technology Platform to Develop Genetic Medicines -...

CAMBRIDGE, Mass., April 27, 2022 (GLOBE NEWSWIRE) -- Sarepta Therapeutics, Inc. (NASDAQ:SRPT), the leader in precision genetic medicine for rare diseases, will report first quarter 2022 financial results after the Nasdaq Global Market closes on Wednesday, May 4, 2022. Subsequently, at 4:30 p.m. E.T., the Company will host a conference call to discuss its first quarter 2022 financial results and to provide a corporate update.

The conference call may be accessed by dialing (800) 895-3361 for domestic callers and (785) 424-1062 for international callers. The passcode for the call is SAREPTA. Please specify to the operator that you would like to join the "Sarepta Therapeutics First Quarter 2022 Earnings Call." The conference call will be webcast live under the investor relations section of Sarepta.com and will be archived there following the call for 90 days. Please connect to Sarepta's website several minutes prior to the start of the broadcast to ensure adequate time for any software download that may be necessary.

About Sarepta TherapeuticsSarepta is on an urgent mission: engineer precision genetic medicine for rare diseases that devastate lives and cut futures short. We hold leadership positions in Duchenne muscular dystrophy (DMD) and limb-girdle muscular dystrophies (LGMDs), and we currently have more than 40 programs in various stages of development. Our vast pipeline is driven by our multi-platform Precision Genetic Medicine Engine in gene therapy, RNA and gene editing. For more information, please visitwww.sarepta.com or follow us on Twitter, LinkedIn, Instagram and Facebook.

Internet Posting of InformationWe routinely post information that may be important to investors in the 'For Investors' section of our website atwww.sarepta.com. We encourage investors and potential investors to consult our website regularly for important information about us.

Source: Sarepta Therapeutics, Inc.

Investor Contact: Ian Estepan, 617-274-4052iestepan@sarepta.com

Media Contact: Tracy Sorrentino, 617-301-8566tsorrentino@sarepta.com

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Sarepta Therapeutics to Announce First Quarter 2022 Financial Results and Recent Corporate Developments on May 4, 2022 | Sarepta Therapeutics, Inc. -...

Elizabeth Ruzzo, PhD, discusses the findings of a report published by the UNFPA, which found women were abandoning effective methods of contraception due to misinformation.

Elizabeth Ruzzo, PhD completed her graduate work at Duke University and postdoctoral research at UCLA. Throughout her 10+ year academic career in human genomics and medical genetics (where she discovered nearly 40 genetic causes for various human diseases) she repeatedly encountered knowledge gaps that hindered discovery in non-Europeans and in females. After her symptoms from birth control side effects were dismissed by medical professionals, she decided to dedicate her expertise in precision medicine to close gaps caused by historic inequity in medical research and demand a new standard of care. She founded adyn in late 2019 and was accepted into Y Combinator in the summer of 2020. Shes a recipient of Rock Healths 2021 Top 50 in Digital Health award. Elizabeths work is also recognized by CB Insights which included adyn in its Digital Health 150 list, an annual ranking of the 150 most promising digital health startups in the world.

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Report: Women abandoning contraception due to misinformation - Contemporary Obgyn

DUBLIN--(BUSINESS WIRE)--The "Spatial Genomics and Transcriptomics Market by Technique, Product Type, Application, and End User: Global Opportunity Analysis and Industry Forecast, 2021-2030" report has been added to ResearchAndMarkets.com's offering.

Global spatial genomics and transcriptomic market size was valued at $0.62 billion in 2020 and is projected to reach $2.15 billion by 2030 registering a CAGR of 13.6% from 2021 to 2030.

Spatial genomics & transcriptomic is a new discipline involving the identification of high-performance information concerning the organizational composition of tissue and cell content. It is a method that uses mRNA readouts to assign cell types to their positions in histological sections. This method may also be used to determine the subcellular localization and cellular heterogeneity of mRNA molecules in cancers and organs.

In addition, spatial genomics and transcriptomics provide information on immune cells and characterize the subcellular distribution of transcripts in a variety of situations. Spatial genomics has a lot of potential in disease control because it provides quantitative gene expression data as well as DNA and RNA visualization mapping into tissue components. In addition, sequencing techniques have a significant role in spatial transcriptomics, due to the widespread availability of sequencing platforms that aid in the study of RNA architecture in cells or tissues as well as the employment of advanced techniques inside a laboratory setup to investigate biological organisms.

The major factors that impact the growth of the spatial genomics and transcriptomic market include increase in adoption of spatial genomics and transcriptomics technologies, owing to their various applications. In addition, growth in prevalence of genetic disorders and applications of spatial genomics and transcriptomics in determination of disease and treatment markers also boost the growth of the market. Furthermore, rise in preference toward personalized medicines also fuels the growth of the market.

However, unavailability of skilled professionals and ethical & legal limitations associated with the use of spatial genomics and transcriptomic technologies hamper the growth of the market. On the other hand, growth in innovations in spatial genomics and transcriptomic technologies are expected to offer profitable opportunities for the growth of the market during the forecast period.

COVID-19 is a large family of viruses that causes illness ranging from common cold to more severe respiratory diseases. The global spatial genomics and transcriptomics market also declined in 2020 due to global economic recession led by COVID-19. However, market witness recovery in the year 2021, owing to increase in adoption of spatial genomics & transcriptomics to profile COVID-19 tissue autopsies as well as rise in biomarker identification and its use in drug discovery and development programs.

The global spatial genomics and transcriptomic market is segmented on the basis of technique, product type, application, end user, and region. By technique, it is bifurcated into spatial transcriptomics, and spatial genomics analysis. The spatial transcriptomics is further segmented into microdissection, fluorescent in-situ hybridization, in-situ sequencing, in-situ capturing, and in-silico construction.

In addition, the spatial genomics analysis segment is segmented into, fluorescence in-situ sequencing, and fluorescence microscopy. The spatial genomics analysis segment was the major shareholder in 2020, owing to emerging potential of spatial genomic analysis as a cancer diagnostic tool, and advent of the fourth generation of sequencing.

By product type, it is segmented into instruments, consumables, and software. The consumables segment dominated the global spatial genomics and transcriptomic market in 2020, and is anticipated to continue this trend during the forecast period. This is attributed to increase in adoption of consumables that are required to run the instruments across various stages of genome mapping from sample preparation to result derivation. Based on application, it is segmented into translational research, and drug discovery & development.

The drug discovery & development acquired the largest share in 2020, and is expected to remain dominant throughout the forecast period. This is attributed to increase in application of spatial genomic and transcriptomics technologies in drug discovery & development. By end user, it is segmented into pharmaceutical & biotechnology companies, contract research organizations, and academic & research institutes.

The academic & research institutes segment acquired the largest share in 2020, and is anticipated to continue this trend during the forecast period. This is attributed to surge in genomic research projects conducted by academic research institutes as well as collaboration among major players and universities for development of novel approaches to cure chronic conditions through genetic medicine.

Key Findings Of The Study

LIST OF KEY PLAYERS PROFILED IN THE REPORT

KEY MARKET SEGMENTS

By Technique

By Product Type

By Application

By End User

By Region

For more information about this report visit https://www.researchandmarkets.com/r/avh6t0

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$2.15 Billion Spatial Genomics and Transcriptomics Markets - Global Opportunity Analysis and Industry Forecast, 2021-2030 - ResearchAndMarkets.com -...

Participants taking tirzepatide lost up to 52 lb. (24 kg) in this 72-week phase 3 study

63% of participants taking tirzepatide 15 mg achieved at least 20% body weight reductions as a key secondary endpoint

INDIANAPOLIS, April 28, 2022 /PRNewswire/ -- Tirzepatide (5 mg, 10 mg, 15 mg) achieved superior weight loss compared to placebo at 72 weeks of treatment in topline results from Eli Lilly and Company's (NYSE: LLY) SURMOUNT-1 clinical trial, with participants losing up to 22.5% (52 lb. or 24 kg) of their body weight for the efficacy estimandi. This study enrolled 2,539 participants and was the first phase 3 global registration trial evaluating the efficacy and safety of tirzepatide in adults with obesity, or overweight with at least one comorbidity, who do not have diabetes. Tirzepatide met both co-primary endpoints of superior mean percent change in body weight from baseline and greater percentage of participants achieving body weight reductions of at least 5% compared to placebo for both estimandsii. The study also achieved all key secondary endpoints at 72 weeks.

For the efficacy estimand, participants taking tirzepatide achieved average weight reductions of 16.0% (35 lb. or 16 kg on 5 mg), 21.4% (49 lb. or 22 kg on 10 mg) and 22.5% (52 lb. or 24 kg on 15 mg), compared to placebo (2.4%, 5 lb. or 2 kg). Additionally, 89% (5 mg) and 96% (10 mg and 15 mg) of people taking tirzepatide achieved at least 5% body weight reductions compared to 28% of those taking placebo.

In a key secondary endpoint, 55% (10 mg) and 63% (15 mg) of people taking tirzepatide achieved at least 20% body weight reductions compared to 1.3% of those taking placebo. In an additional secondary endpoint not controlled for type 1 error, 32% of participants taking tirzepatide 5 mg achieved at least 20% body weight reductions. The mean baseline body weight of participants was 231 lb. (105 kg).

"Obesity is a chronic disease that often does not receive the same standard of care as other conditions, despite its impact on physical, psychological and metabolic health, which can include increased risk of hypertension, heart disease, cancer and decreased survival," said Louis J. Aronne, MD, FACP, DABOM, director of the Comprehensive Weight Control Center and the Sanford I. Weill Professor of Metabolic Research at Weill Cornell Medicine, obesity expert at NewYork-Presbyterian/Weill Cornell Medical Center and Investigator of SURMOUNT-1. "Tirzepatide delivered impressive body weight reductions in SURMOUNT-1, which could represent an important step forward for helping the patient and physician partnershiptreat this complex disease."

For the treatment-regimen estimandiii, results showed:

The overall safety and tolerability profile of tirzepatide was similar to other incretin-based therapies approved for the treatment of obesity. The most commonly reported adverse events were gastrointestinal-related and generally mild to moderate in severity, usually occurring during the dose escalation period. For those treated with tirzepatide (5 mg, 10 mg and 15 mg, respectively), nausea (24.6%, 33.3%, 31.0%), diarrhea (18.7%, 21.2%, 23.0%), vomiting (8.3%, 10.7%, 12.2%) and constipation (16.8%, 17.1%, 11.7%) were more frequently experienced compared to placebo (9.5% [nausea], 7.3% [diarrhea], 1.7% [vomiting], 5.8% [constipation]).

Treatment discontinuation rates due to adverse events were 4.3% (5 mg), 7.1% (10 mg), 6.2% (15 mg) and 2.6% (placebo). The overall treatment discontinuation rates were 14.3% (5 mg), 16.4% (10 mg), 15.1% (15 mg) and 26.4% (placebo).

Participants who had pre-diabetes at study commencement will remain enrolled in SURMOUNT-1 for an additional 104 weeks of treatment following the initial 72-week completion date to evaluate the impact on body weight and the potential differences in progression to type 2 diabetes at three years of treatment with tirzepatide compared to placebo.

"Tirzepatide is the first investigational medicine to deliver more than 20 percent weight loss on average in a phase 3 study, reinforcing our confidence in its potential to help people living with obesity," said Jeff Emmick, MD, Ph.D., vice president, product development, Lilly. "Obesity is a chronic disease that requires effective treatment options, and Lilly is working relentlessly to support people with obesity and modernize how this disease is approached. We're proud to research and develop potentially innovative treatments like tirzepatide, which helped nearly two thirds of participants on the highest dose reduce their body weight by at least 20 percent in SURMOUNT-1."

Tirzepatide is a novel investigational once-weekly GIP (glucose-dependent insulinotropic polypeptide) receptor and GLP-1 (glucagon-like peptide-1) receptor agonist, representing a new class of medicines being studied for the treatment of obesity. Tirzepatide is a single peptide that activates the body's receptors for GIP and GLP-1, two natural incretin hormones.Obesity is a chronic, progressive disease caused by disruptions in the mechanisms that control body weight, often leading to an increase in food intake and/or a decrease in energy expenditure. These disruptions are multifactorial and can be related to genetic, developmental, behavioral, environmental and social factors. To learn more, visit Lilly.com/obesity.

Lilly will continue to evaluate the SURMOUNT-1 results, which will be presented at an upcoming medical meeting and submitted to a peer-reviewed journal. Additional studies are ongoing for tirzepatide as a potential treatment for obesity or overweight.

About tirzepatide

Tirzepatide is a once-weekly GIP (glucose-dependent insulinotropic polypeptide) receptor and GLP-1 (glucagon-like peptide-1) receptor agonist that integrates the actions of both incretins into a single novel molecule. GIP is a hormone that may complement the effects of GLP-1 receptor agonists. In preclinical models, GIP has been shown to decrease food intake and increase energy expenditure therefore resulting in weight reductions, and when combined with GLP-1 receptor agonism, may result in greater effects on markers of metabolic dysregulation such asbody weight, glucose and lipids. Tirzepatide is in phase 3 development for adults with obesity or overweight with weight-related comorbidity and is currently under regulatory review as a treatment for adults with type 2 diabetes. It is also being studied as a potential treatment for non-alcoholic steatohepatitis (NASH) and heart failure with preserved ejection fraction (HFpEF). Studies of tirzepatide in obstructive sleep apnea (OSA) and in morbidity/mortality in obesity are planned as well.

About SURMOUNT-1 and the SURMOUNT clinical trial program

SURMOUNT-1 (NCT04184622) is a multi-center, randomized, double-blind, parallel, placebo-controlled trial comparing the efficacy and safety of tirzepatide 5 mg, 10 mg and 15 mg to placebo as an adjunct to a reduced-calorie diet and increased physical activity in adults without type 2 diabetes who have obesity, or overweight with at least one of the following comorbidities: hypertension, dyslipidemia, obstructive sleep apnea or cardiovascular disease. The trial randomized 2,539 participants across the U.S., Argentina, Brazil, China, India, Japan, Mexico, Russia and Taiwan in a 1:1:1:1 ratio to receive either tirzepatide 5 mg, 10 mg or 15 mg or placebo. The co-primary objectives of the study were to demonstrate that tirzepatide 10 mg and/or 15 mg is superior in percentage of body weight reductions from baseline and percentage of participants achieving 5% body weight reduction at 72 weeks compared to placebo. Participants who had pre-diabetes at study commencement will remain enrolled in SURMOUNT-1 for an additional 104 weeks of treatment following the initial 72-week completion date to evaluate the impact on body weight and potential differences in progression to type 2 diabetes at three years of treatment with tirzepatide compared to placebo.

All participants in the tirzepatide treatment arms started the study at a dose of tirzepatide 2.5 mg once-weekly and then increased the dose in a step-wise approach at four-week intervals to their final randomized maintenance dose of 5 mg (via a 2.5 mg step), 10 mg (via steps at 2.5 mg, 5 mg and 7.5 mg) or 15 mg (via steps at 2.5 mg, 5 mg, 7.5 mg, 10 mg and 12.5 mg).

The SURMOUNT phase 3 global clinical development program for tirzepatide began in late 2019 and has enrolled more than 5,000 people with obesity or overweight across six clinical trials, four of which are global studies. Results from SURMOUNT-2, -3, and -4 are anticipated in 2023.

About Lilly

Lilly unites caring with discovery to create medicines that make life better for people around the world. We've been pioneering life-changing discoveries for nearly 150 years, and today our medicines help more than 47million people across the globe. Harnessing the power of biotechnology, chemistry and genetic medicine, our scientists are urgently advancing new discoveries to solve some of the world's most significant health challenges, redefining diabetes care, treating obesity and curtailing its most devastating long-term effects, advancing the fight against Alzheimer's disease, providing solutions to some of the most debilitating immune system disorders, and transforming the most difficult-to-treat cancers into manageable diseases. With each step toward a healthier world, we're motivated by one thing: making life better for millions more people. That includes delivering innovative clinical trials that reflect the diversity of our world and working to ensure our medicines are accessible and affordable. To learn more, visitLilly.comandLilly.com/newsroomor follow us onFacebook,Instagram,Twitterand LinkedIn. P-LLY

Lilly Cautionary Statement Regarding Forward-Looking Statements

This press release contains forward-looking statements (as that term is defined in the Private Securities Litigation Reform Act of 1995) about tirzepatide as a potential treatment for adults with obesity or overweight and the timeline for future readouts, presentations and other milestones relating to tirzepatide and its clinical trials, and reflects Lilly's current belief and expectations. However, as with any pharmaceutical product, there are substantial risks and uncertainties in the process of research development and commercialization. Among other things, there can be no guarantee that the studies will be completed as planned, that future study results will be consistent with the results to date or that tirzepatide will receive regulatory approvals.For further discussion of these and other risks and uncertainties, see Lilly's most recent Form 10-K and Form 10-Q filings with the United States Securities and Exchange Commission. Except as required by law, Lilly undertakes no duty to update forward-looking statements to reflect events after the date of this release.

Dr. Aronne is co-founder, chief scientific advisor and member of the board of directors for Intellihealth. Dr. Aronne is also a paid scientific advisory board member for Eli Lilly and Company.

iEfficacy estimand represents efficacy prior to discontinuation of study drug.iiTreatment differences for two estimands efficacy and treatment-regimen were evaluated for three tirzepatide doses (5 mg, 10 mg and 15 mg) compared to placebo.iiiTreatment-regimen estimand represents the estimated average treatment effect regardless of treatment discontinuation.

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SOURCE Eli Lilly and Company

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Lilly's tirzepatide delivered up to 22.5% weight loss in adults with obesity or overweight in SURMOUNT-1 | Eli Lilly and Company - Eli Lilly

Expectant parents, doctors, and regulators need to reconsider the rising use of gee-whiz genetic testing as doubts emerge about popular blood screenings to detect rare prenatal disorders and a costly test relied on by couples undergoing in-vitro fertilization (IVF) treatment.

This is what the New York Times reported about what researchers have found about preimplantation genetic testing for aneuploidy, or PGT-A. It is an increasingly common screening in IVF and has led potential parents to discard embryos as unfeasible or unacceptable due to abnormalities to carry to term:

PGT-A has, over the last two decades, become a standard add-on to already pricey IVF procedures. But the test, which can cost anywhere from $4,000 to $10,000, has become controversial over the years as studies have cast doubt on whether it increases birthrates from IVF at all. A growing number of scientists have questioned the widespread use of the test, which leads to tens of thousands of discarded embryos per year and causes many women to believe they may not be able to carry biological children. A new study published last week details 50 patients who underwent transfers of abnormal embryos at the Center for Human Reproduction in New York City The study reported eight births after 57 transfer cycles of embryos with abnormal genetic testing results since 2015. Seven of the babies were born healthy. The average age of the women in the study was 41 years old.

The study is a follow-up to a 2015 study also led by the center that first showed that selected abnormal embryos could still be viable. Since then, other fertility clinics worldwide have also started to transfer such embryos. The research was funded by the clinic and by the Foundation for Reproductive Medicine, a nonprofit research organization also based in New York. The new study supports concerns that I and others have had for several years now about the accuracy of these tests, Josephine Johnston, a bioethicist and director of research at the Hastings Center who was not involved in the research, said by email. The study strengthens the argument that PGT-A tests have been prematurely incorporated into fertility medicine and strongly suggests that these tests will have led patients to discard potentially viable embryos.

The newspaper reported this key criticism of the test:

[The problem with PGT-A, the authors of the study argue, is that it provides an incomplete picture that is often interpreted as a very definitive result. The test relies on sampling a handful of cells from the outer shell of the developing embryo and testing to see if each one has 23 pairs of chromosomes [structures, as shown in Harvard single-cell, color photo]. The point of PGT was to select embryos that would give somebody a better chance of achieving pregnancy, said Dr. David Barad, an OB-GYN at the Center for Human Reproduction and a co-author of the study. But doing genetic testing doesnt make embryos better, it just kind of gives us some idea of who they are.

Researchers caution that embryos flagged by the PGT-A test and that prove viable to carry to term have risks of miscarriage or of infants having abnormalities at birth, the newspaper reported. These can be the result, though, of conditions and circumstances such as the mothers age that lead couples into the challenging, costly IVF process.

The PGT-A test, with its results presented without nuance and complete explanation, can derail complex choices about IVF embryos, especially if couples proceed with those now declined due to the PGA-T test, the New York Times reported, quoting experts, including Laura Hercher, director of student research at the Sarah Lawrence College genetic counseling program.

We dont really know what to tell them about the long-term potential consequences to a child thats born. Will there be an increased number of birth defects? Will we find that they have problems down the road? Theres a lot of I dont knows.

The New York Times article, by the way, reports on Anna and Brian Dahlquist, a couple undergoing IVF treatment who declined to implant an embryo due to the PGT-A test but later switched specialists and clinics and went ahead successfully, years later. As she told the newspaper about her healthy son:

I was 39 when I made his embryo. And I was 46 when I became pregnant with him. Thats a long time and a lot of valuable years in both my childs upbringing and my life.

U.S. warns about prenatal blood tests

The federal Food and Drug Administration, meantime, has offered a sharp caution for expectant couples who have experimented with prenatal blood tests to screen fetuses for rare genetic conditions, the New York Times reported, separately. The newspaper quoted a statement from Dr. Jeff Shuren, director of the FDAs Center for Devices and Radiological Health:

While genetic noninvasive prenatal screening tests are widely used today, these tests have not been reviewed by the FDA and may be making claims about their performance and use that are not based on sound science.

The New York Times independently investigated the blood tests with expert assistance recently, reporting:

[A] New York Times examination in January reported on the incidence of false positive results in some of these tests, known as noninvasive prenatal screening tests, or NIPTs. That article quoted women who received false positive screening results for extremely rare conditions; highlighted examples of misleading marketing by companies selling the tests; and described some reports of pregnancies that were terminated based on a screening without a confirmatory test. The agency warned doctors not to diagnose genetic disorders based on these results alone and stressed the need to follow positive screenings with more reliable diagnostic testing. It said it was aware of reports that some women have ended pregnancies based only on the results of these genetic screening tests.

The newspaper had further information about the burgeoning use of these blood tests:

The tests are taken by over a third of pregnant women in the United States. But they fall into a category called lab-developed tests, which are not regulated or approved by the FDA. In its safety notice, the agency said it was working with Congress on legislation to establish a modern regulatory framework for all tests, including the category that covers these screenings. The agencys warning comes after numerous accounts, dating back years, of misinterpretations of the screenings as being definitive. But the criticism has intensified in recent months, with 97 Republican legislators sending a letter to the FDA in January, prompted by the Times article. This is an area that has concerned the agency for a long time, said Alberto Gutierrez, the former director of the FDA office that oversees many medical test The lack of regulation of lab-developed tests has led to many cases where the laboratories have claimed more than they should based on the data that is available, he added. The warning does not come with any consequence for prenatal test manufacturers. But it urged them to submit their products for voluntary approval. A spokeswoman for the agency declined to say whether it was currently working with any manufacturers on this front.

In my practice, I see not onlythe harms that patients suffer while seeking medical services, but also the damage that can be inflicted on them and their loved ones bydefensive and excessive over testing, over diagnosis, misdiagnosis, and over-treatment. Patients struggle too much already to afford needed medical services much less those that are invasive, painful, wasteful, and unnecessary.

Up to a third of medical spending goes for over-treatment and over-testing, withan estimated $200 billion in the U.S. expended on medical services with little benefit to patients. Butgetting doctors and hospitals to stop this excessisnt easy, nor is it a snap to get patients to understand what this problems all about so theyll push their health care providers to do something about it.

With medical tests, like the dubious rare-disease prenatal blood screen promoted by Silicon Valley firms, serious questions must be asked about the dearth of federal oversight. It has been opposed by those who argue that patients benefit best by government staying out of the way of innovation

Caveat emptor, consumers. Patients must insist that their doctors explain fully any test or procedure. This is patientsfundamental right to informed consent. This means they are told clearly and fully all the important facts they need to make an intelligent decision about what treatments to have, where to get them, and from whom. Patients, in turn, must put in the effort to safeguard themselves by improving their knowledge and comprehension about their care a constant learning process that is crucial for those who wish to bring new lives into the world and to nurture them for decades after.

We have much work to do to rein in costly, wasteful, and harmful testing.

Original post:
Cautions issued over prenatal genetic tests in IVF and for rare diseases - JD Supra

Immunotherapy is an increasingly important form of cancer treatment. It encompasses a class of cancer therapies that harness the immune systems natural ability to recognize and eliminate tumor cells. Most immunotherapies work by boosting the power of a type of white blood cell called T cells, so they can better recognize and attack cancer.

Investigators at Memorial Sloan Kettering Cancer Center (MSK) are developing a variety of tactics for creating new immunotherapy treatments. One focus is on finding ways to engineer Tcells to recognize mutated proteins that are unique to cancer cells. A paper reporting a new advance in this area was published in Nature Medicine on April 28, 2022.

The genesis of this project was to try to come up with a new form of cellular immunotherapy that might give an inroad against cancers that are largely unresponsive to current forms of immunotherapy treatment, says physician-scientist Christopher Klebanoff, the papers senior author. Were using a genetic engineering approach to help supercharge T cells in a new way.

Cancer is caused by the accumulation of mutated genes, some of which result in the generation of novel proteins, called neoantigens, that can be perceived by the immune system as foreign. Tcells have a natural ability to recognize neoantigens, and they can be trained to do so even more effectively. The MSK researchers thought that by focusing on neoantigens resulting from recurrent driver mutations (those that actually cause cancer to grow and spread), they could develop an off-the-shelf therapy applicable to many patients. The study was done in collaboration with researchers from the University of Notre Dame.

The driver approach is different from immunotherapies that target neoantigens resulting from patient-specific mutations that are simply along for the ride (called passenger mutations). Therapies targeting neoantigens derived from passenger mutations are only applicable to a single patient because they target mutations unique to that persons cancer.

We're using a genetic engineering approach to help supercharge T cells in a new way.

Dr. Klebanoff and his colleagues decided to focus on a neoantigen created by a common driver mutation in a gene called PIK3CA. Their rationale was that this particular mutation is frequently found in breast cancer, endometrial cancer, and many other types of tumors that could benefit from more effective therapies.

There are already drugs that target this protein, but they cause a lot of side effects, says Smita Chandran, a scientific research lead in Dr. Klebanoffs lab and the Nature Medicine papers first author. We thought that targeting this driver gene with a cell-based immunotherapy might be more effective while at the same time reducing the risks of side effects.

One challenge in immunologically targeting mutant PIK3CA is that its found on the inside of cancer cells, a location that is inaccessible to antibody therapies. Its also unreachable using chimeric antigen receptor (CAR)T therapy, which is a form of cellular immunotherapy that has shown clinical benefit in several types of blood cancers. Antibody and CART therapies can only engage target proteins displayed on the outside of cancer cells. To get around this limitation, the researchers employed a tactic called Tcell receptor (TCR) therapy, which co-opts the T cells unique ability to survey the interior of cells for the presence of abnormal proteins.

We thought that targeting this driver gene with a cell-based immunotherapy might be more effective while at the same time reducing the risks of side effects, says researcher Smita Chandran.

This technology works because both cancer cells and normal cells regularly break down and recycle their proteins. As part of this process, fragments of degraded proteins within a cell are placed in a molecular basket, called a human leukocyte antigen (HLA). The loaded HLA molecule is then shuttled to the cell surface for display to Tcells. The researchers discovered that a common PIK3CA mutation results in a neoantigen that is compatible with an HLA type shared by a large number of patients, creating an elite class of shared tumor-exclusive targets called public neoantigens.

Building on this finding, the investigators went on to demonstrate that Tcells genetically engineered to express a TCR specific to the PIK3CA public neoantigen can attack and eliminate tumor cells while leaving healthy cells that express the normal version of protein unharmed.

TCR-engineered T cells were therapeutically effective against cancer cells in a petri dish and also against patient-derived tumor samples and in laboratory mice implanted with PIK3CA-mutated tumors. Some of the cancer types studied included breast cancer and an aggressive form of endometrial cancer called uterine serous carcinoma. These are cancers for which current immunotherapies have typically not been effective.

In addition to having a large number of side effects, the drugs that are currently approved to target mutant PIK3CA work only for a limited period. Most tumors ultimately develop resistance and begin growing again.

Based on their research in the lab, Drs. Klebanoff and Chandran expect that targeting mutated PIK3CA with immunotherapy rather than targeted therapy will not result in the same degree of resistance.

The researchers plan to work in collaboration with a pharmaceutical company to determine whether this approach could be evaluated in a clinical trial in the future. Its possible that trials could enroll any patient whose tumor carries the PIK3CA public neoantigen, regardless of where in the body the tumors originated. The fact that this therapy targets public neoantigens is the reason this treatment would be considered off-the-shelf, rather than having to be manufactured individually for each patient.

Theres an evolving appreciation that its genetic changes that contribute to cancer formation in the first place, as well as the likelihood a cancer will respond to a given therapy, Dr.Klebanoff says. We want to take the approach of developing treatments that are target-specific but tissue-agnostic and apply this to new immunotherapies, including the PIK3CA public neoantigen described in our paper.

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New Immunotherapy Approach Targets a Commonly Mutated Driver Protein Unique to Cancer Cells - On Cancer - Memorial Sloan Kettering

By Ed Miseta, Chief Editor, Clinical LeaderFollow Me On Twitter @EdClinical

Recent years have seen unprecedented innovation in the clinical space. Precision medicine, cell and gene therapies, decentralized trials, real-world data, and the promise of artificial intelligence (AI) and machine learning (ML) are just a few of the reasons to be excited about the future of clinical research. But what can we expect to see in the next three years, and what are the challenges sponsor companies will need to overcome?

A webinar hosted by IBM Watson hoped to answer those questions. The discussion featured Lorraine Marchand, general manager of life sciences at IBM Watson Health; Nimita Limaye, research VP, Life Sciences R&D Strategy and Technology at IDC Health Insights; and Greg Cunningham, director of the RWE Center of Excellence at Eli Lilly and Company. The three shared insights into what we might expect to impact trials over the next three years.

In this article the panel discusses precision medicine and real-world data. In part 2 of this article the panel looks at the future of decentralized clinical trials.

The Growth Of Precision Medicine

The first game changer the panel discussed is the advancement of precision medicine. It has moved from exploring single gene mutations to performing research using combinations of genes. This change has the potential to bring better drug targets forward and get the best products to patients faster.

This has been playing out in the last decade in oncology real-world evidence, notes Cunningham. We've seen an evolution in precision medicine as we've built out the patient record. As we have done that, the marketplace has evolved rapidly, particularly for electronic medical record data and genomic data.

Pharma companies were happy to get their hands on electronic medical record data. When genetic test results were combined with that data, researchers gained the ability to look at a single mutation and develop better patient outcomes.

Where precision medicine will continue to evolve in 2022 and beyond is the growing use of genetic testing in oncology. This will provide the industry with more data about patients. With more genes at their disposal, researchers can look at groups of genes and the complex combinations of gene mutations. This has the potential to open the door for tools like artificial intelligence to help researchers analyze the complex number of permutations.

RWD Creates More Efficient Research

Next the panel discussed RWD and the ability to utilize it across several use cases from discovery and development to commercial. Limaye likes the prospect of being able to create a data exchange where researchers can bring together claims, clinical, EMR, and genomics data directly from patients to create an intelligent and digital patient health record. That record gives researchers the digital equivalent of a real-life patient which can be used as a natural history or synthetic control arm in randomized control clinical trials.

These data can allow drug developers to track patient response to drugs and look at outcomes after being exposed to new therapies. The promise of data and technology is using tools like AI to advance therapies and get them to patients faster. This will be done with better information and a much more efficient way to perform drug development and track and monitor outcomes in patients.

Big data has been a topic of discussion in pharma for years. The volume of clinical data is now growing exponentially. Approximately 30% of the world's datavolume is being generated by the healthcare industry and by 2025, the compound annual growth rate will hit 36%. That's 6% faster than manufacturing, 10% faster than financial services, and 11% faster than media & entertainment.

In addition to getting bigger, data is also getting broader. Researchers can not only look at a patients medical history but can now consider factors such as social determinants of health and behavioral data.

Since most EHRs do not include genomic data, researchers need the ability to look at patient data more holistically. Type 2 diabetes was one example discussed. Today, 40% to 70% of it is genetically inherited and there are over 500 different genetic loci which could be involved in causing the disease. The earlier strategy of looking at genetic risk scoring was single trait. That is now transitioning to multi-trait research with an integrated view that will drive a precision medicine strategy. This creates an interesting situation where drug discovery will continue to get more specific and focused towards an individual while also getting bigger and broader.

The Challenge Of RWD

With access to RWD, drug developers can benefit from data they may not have known existed. Although the data is rich and robust, it can be difficult to access. One of the biggest challenges the industry faces is data stored in silos. The panel notes data is stored in patient claims, electronic medical records, in lab apps, images, and genetic files on a smartphone. Having the technology to tap into those sources to identify quality data is the primary challenge.

The data must be de-identified for patient privacy, cleaned, curated to remove noise, and enriched, which means bringing together the various components that will be meaningful to drug development. That will allow researchers to have a patient record that is useful across pharma, from development through to commercial. An exchange would enable that exact process a platform where various entities can bring their data to have it linked, integrated, cleaned, and enriched, creating a data package that can be plugged into studies.

An important component of that exchange is the data being housed in a place where various third parties can feel comfortable bringing their data to match it with data from other third parties.

Cunningham cites lupus as an example of where pharma could benefit from such an exchange. I would like to have a complete data set of lupus, he says. Lupus is an autoimmune condition, and a quintessential data set could be used for a number of uses, such as preparing a Phase 1 trial, selecting patients, or understanding patient responses to different therapies when designing studies. Specific data sets could be created for each therapeutic area, and pharma companies need that hard work of bringing the data together removed.

Data Assembly And Analysis

Currently, drug developers spend 80% of their time assembling data and 20% of their time analyzing it. The situation must be flipped so that 80% of the time is spent performing analysis. The panel recommends rethinking how health records are created. The healthcare and life science industries require the ability to easily put data together. That comes back to investing in data standards everyone can agree upon. With the right standards and technology, the industry can spend its time improving lives as opposed to assembling data.

The FDA has indicated it is aware and supportive of the fact that pharma needs use RWD in drug discovery. The industry now needs to create the interoperability, standards, and methods to ensure that data can be included in regulatory submissions. This evolution may be akin to the critical path initiative. When the FDA embraced the idea of the critical path and allowing more in silico modeling of clinical trial design and development, it took the industry almost 10 years to adopt and apply the guidance.

The FDA has said it recognizes the importance of RWD, but that acknowledgement has resulted in few approvals. Looking at the use of synthetic control arms and RWD in regulatory submissions over the last five years shows just 10 submissions and all were in oncology. Only one was a successful submission, and the rest were rejected because of lack of completeness of the data.

Those numbers should tell the industry the FDA is not going to dictate how to get to approvals. The industry is going to have to figure out the interoperability and how to apply the standards. Regulators are always going to require quality data. Industry will need to enrich the data and create the cohort that is going to be equivalent to a patient in the real world.

In part 2 of this article, the panel discusses the role of technology in clinical trials, how decentralized trials will continue to evolve, what capabilities sponsor companies will need, and whether decentralized trials might offer cost benefits to companies.

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The Next Three Years Of Clinical Trials DCTs RWE And Beyond - Clinical Leader