Weill Cornell Medicine, NewYork-Presbyterian Hospital, and Illumina Collaborate on Scalable Clinical Whole-Genome Sequencing Initiative

NEW YORK, Dec. 3, 2020 /PRNewswire/ — Seeking to advance the scope of precision medicine, Weill Cornell Medicine, NewYork-Presbyterian Hospital, and Illumina, Inc. are entering into a collaboration to sequence the complete human genomes of thousands of consenting patients, in order to identify genetic alterations driving disease and potentially reveal previously unidentified therapies for treatment. The initiative, which also includes a collaboration between Weill Cornell Medicine, NewYork-Presbyterian Hospital, and the New York Genome Center (NYGC), aims to evaluate the diagnostic potential of whole-genome sequencing at scale, which allows the interrogation of the full genome sequence of a patient’s DNA. The goal is to better understand health problems and potential disease risks of individual patients, and to design more effective treatments, including the choice of specific drugs and their dosing.

Investigators will study the feasibility and viability of large-scale implementation of whole-genome sequencing within an academic medical center that is part of a major metropolitan health care system in the United States. Whole-genome sequencing has already been shown to improve patient care and disease prevention in specific clinical contexts, but few systems have deployed whole-genome sequencing across multiple care pathways. Weill Cornell Medicine, through its Caryl and Israel Englander Institute for Precision Medicine, and NewYork-Presbyterian/Weill Cornell Medical Center, which have applied this precision medicine approach to investigate cancer’s molecular underpinnings since 2015, will be among the first medical institutions to examine the feasibility of large-scale whole-genome sequencing across multiple diseases. In addition to revealing the role individual genes play in disease and therapeutic responses, the study could also yield promising new avenues for scientific inquiry.

Under the initiative, which originates from Weill Cornell Medicine’s Englander Institute for Precision Medicine, doctors at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center will offer qualifying patients the option to have their genomes sequenced as part of their diagnostic workups. NYGC will leverage its high-throughput whole-genome sequencing clinical sequencing expertise to investigate patients’ DNA, using Illumina’s patented Next-Generation Sequencing technology. NYGC was the first sequencing center in the country to gain regulatory approval for clinical whole-genome sequencing tests for genetic diseases and cancer from the New York State Department of Health Clinical Laboratory Evaluation Program. Board-certified molecular geneticists at NYGC will interpret and share the results with ordering physicians, who will then share them with their patients. The initiative will focus on the disease areas of oncology, cardiovascular, metabolic and neurodegenerative diseases. This first phase will inform the next steps to expand infrastructure to support more widespread testing in years to come.  

“We are committed to expanding whole-genome sequencing to cancer and other common diseases more broadly, so that the approach can eventually become a routine part of healthcare, an essential source of data for biomedical research and, importantly, enhance patient care,” said Dr. Olivier Elemento, director of the Englander Institute for Precision Medicine at Weill Cornell Medicine, who also leads joint precision medicine efforts at Weill Cornell Medicine and NewYork-Presbyterian/Weill Cornell Medical Center. “This project and the network of participating

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Clinical trials are key to advancing medicine, including COVID-19 vaccine

MIAMI – Registered clinical trials reached record levels in the past year, increasing the demand for people willing to participate, and Miami resident Marilyn Strauss Diaz was eager to join a COVID-19 vaccine trial underway at the University of Miami Health System.

“We can’t depend on a lab rat. We have to have human trials for there to be a successful vaccine, so we have to have humans come forward and be a part of trials,” she said.

“Before COVID, we usually would have a couple of hundred clinical trials here going at the same time at the University of Miami that are focused on a lot of different diseases. With COVID, there was a slowdown and now we’re ramping up again and starting a lot of these studies,” said Dr. Olveen Carassquillo, who is part of the research team.

“COVID has the most urgency. That’s a public health crisis so a lot of research is focused on that, but we have a lot of landmark cancer studies going on with different treatment regimens and prevention studies. We also have a lot of cardiovascular disease, a lot of other studies focused on stroke, on preventing dementia,” Carassquillo added.

And South Florida’s diverse population makes it a major focus of research efforts, something Strauss Diaz is proud to be a part of.

“At the end, it will be beneficial to so many,” she said.

To learn more about clinical trials at the University of Miami Health System go to: https://umiamihealth.org/clinical-trials

Copyright 2020 by WPLG Local10.com – All rights reserved.

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Ionis announces AstraZeneca’s initiation of the Phase 2b clinical study of its antisense medicine targeting PCSK9 to lower LDL-cholesterol

CARLSBAD, Calif., Nov. 30, 2020 /PRNewswire/ — Ionis Pharmaceuticals, Inc. (NASDAQ:IONS) today announced that the biopharmaceutical company AstraZeneca has initiated a Phase 2b clinical trial of ION449 (AZD8233), an investigational antisense medicine designed to reduce blood cholesterol levels in patients with dyslipidemia by targeting proprotein convertase subtilisin/kexin type 9 (PCSK9), an important regulator of low-density lipoprotein cholesterol (LDL-C). PCSK9 is an enzyme that controls the number of LDL receptors on the surface of cells. People with genetic variations that reduce PCSK9 function have lower LDL-C levels in the blood and a lower risk for major cardiovascular events. ION449 is a LIgand Conjugated Antisense (LICA) medicine being developed by AstraZeneca as part of a collaboration between Ionis and AstraZeneca.


(PRNewsfoto/Ionis Pharmaceuticals, Inc.)

The Phase 2b, randomized, double-blind, placebo-controlled trial will enroll approximately 108 participants, aged 18-75, who have LDL-C levels between 70 and 190 mg/dL and are receiving moderate- or high-intensity statin therapy as defined by the American College of Cardiology/American Heart Association (ACC/AHA) guidelines on blood cholesterol management. The primary objective is to assess the effect of different doses of ION449 on LDL-C compared to placebo at Week 12 in patients taking baseline statin therapy.  The study will evaluate three dose levels of ION449 versus placebo, all administered once a month by subcutaneous injection. Safety and tolerability will be evaluated along with a number of secondary endpoints. Learn more about the trial at: https://clinicaltrials.gov/ct2/show/NCT04641299. 

In a Phase 1 study reported at the American Heart Association (AHA) Scientific Sessions on November 13, single subcutaneous doses of ION449 (AZD8233) demonstrated dose-dependent mean reductions in circulating plasma PCSK9 and LDL-C levels of >90 percent and up to 70 percent, respectively, in subjects who had a baseline LDL-C between 100 and 190 mg/dL without concomitant statin therapy.Doses up to 120 mg were evaluated. ION449 was observed to be safe and well-tolerated at all dose levels. 

“Results from the Phase 1 study showed that ION449 potently reduces PCSK9 and LDL cholesterol. ION449 demonstrated best-in-class potential for PCSK9 inhibition and LDL-C reduction, supporting larger clinical trials that are now underway to further evaluate efficacy and safety,” said Sotirios “Sam” Tsimikas, M.D., senior vice president, clinical development and cardiovascular franchise leader at Ionis. “The growing evidence supporting Ionis’ advanced LICA technology in cardiovascular disease holds promise for more effective approaches to lower LDL-C and to address cardiovascular disease, the leading cause of death worldwide.”

Dr. Tsimikas will provide an update on Ionis’ cardiovascular programs during Ionis’ Virtual Investor Day, Dec. 7, 2020, beginning at 12 p.m. EST.

Ionis earned a milestone payment of $20 million from AstraZeneca for the Phase 2b clinical trial initiation of ION449. Ionis and AstraZeneca are collaborating on potential treatments for kidney disease, cardiometabolic disease and cancer.

About Ionis Pharmaceuticals, Inc.

As the leader in RNA-targeted drug discovery and development, Ionis has created an efficient, broadly applicable, drug discovery platform called antisense technology that can treat diseases where no other therapeutic approaches have proven effective. Our drug

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ZYUS Life Sciences Strengthens Leadership in Evidence-Based Medicine with Appointment of Clinical Advisory Committee

SASKATOON, Saskatchewan–(BUSINESS WIRE)–ZYUS Life Sciences Inc. (“ZYUS”), a Canadian life sciences company leading scientific research and development in phyto-therapeutics, is pleased to announce the formation of a Clinical Advisory Committee to provide strategic advice and guidance on ZYUS’ clinical trial programs. Five committee members have been appointed to the Clinical Advisory Committee, adding additional medical, scientific, and clinical expertise to its experienced leadership team. This announcement is an important step in ZYUS’ integrated clinical development program and supports the company’s ambitions to pioneer the next generation of life sciences through an evidence-based approach.

Chaired by ZYUS’ Chief Medical Officer, Dr. Lionel Marks de Chabris, a leading expert in chronic pain management and addictions medicine, the ZYUS Clinical Advisory Committee includes four initial committee members including Dr. Mary Lynch, Dr. David J. Shulkin, Dr. Cedric Francois and Dr. Alice Zwerling. As ZYUS scales its clinical trial programs, the Clinical Advisory Committee will work closely with the research team to provide input and guidance on clinical development strategies and evaluate the clinical trial progress.

Each of the named Clinical Advisory Committee members bring remarkable medical leadership and expertise to their advisory roles. Dr. Mary Lynch ranks among North America’s leading experts in pain management. She is a Professor in the Departments of Anesthesiology, Pain Management and Perioperative Medicine, Psychiatry and Pharmacology in the Faculty of Medicine at Dalhousie University. In addition to being the founding director of the Canadian Consortium for the Investigation of Cannabinoids, Dr. Lynch co-chairs the Canadian Pain Strategy and is a member of the Royal College of Physicians and Surgeons Working Group on Pain Management. Her expertise in pain management is directly aligned with ZYUS’ research into the potential of phyto-therapeutics to manage chronic and neuropathic pain, and Dr. Lynch’s scientific research and thought-leadership in this space will provide valuable guidance to ZYUS’ clinical approach.

Providing cross-border expertise and widely respected medical leadership, Dr. David J. Shulkin will bring his decades of experience leading some of North America’s largest and most sophisticated medical networks to the ZYUS Clinical Advisory Committee. Dr. Shulkin previously served as the ninth Secretary of the US Department of Veterans Affairs and prior to holding that role, was appointed Under Secretary for Health by President Obama and confirmed unanimously by the U.S. Senate. In his role as Secretary of the U.S. Department of Veterans Affairs, he represented 21 million American veterans and was responsible for the country’s largest integrated health care system with over 1,200 sites of care. In addition to his roles in the public sector, Dr. Shulkin has served as the chief executive of multiple hospitals and health systems, and has been named as one of the “One Hundred Most Influential People in American Healthcare” by Modern Healthcare. Dr. Shulkin’s experience as a physician, hospital CEO and Secretary of the Department of Veterans Affairs provides unique insights into the medical, societal and political implications of ZYUS’ clinical trial programs.

The co-founder and Chief Executive Officer and President of Apellis Pharmaceuticals,

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Spring Hills Names Dr. Pierantonio Russo as Chief Medical Officer and Chief Clinical Transformation Officer

Veteran Cardiac Surgeon, Heart Transplant Surgeon and Health Care Executive to Lead Expansion of Organization’s Population Health Management Program

Spring Hills announced today that it has named Pierantonio Russo, MD, Chief Medical Officer and Chief Clinical Transformation Officer of the Population Health Management Program. Russo will oversee the integration of Population Health into Spring Hills’ clinical programs and offer guidance to providers, payers, and policymakers on IT and AI enabled comprehensive population management, including complex care for high-risk, high cost, high needs patients.

Following two decades of practicing cardiac and heart transplant surgery, first at the Mayo Clinic, Russo assumed executive leadership roles providing expertise in medical informatics, machine learning (ML) and predictive modeling applications in health care, value-based insurance and outcome-based contracts, and population medicine. Prior to joining Spring Hills, Russo served as the Chief Medical Officer of HVH/Eversana, a leading provider of artificial intelligence (AI) and ML for health care applications and global commercial services to the life sciences industry. Among other projects, Russo led an initiative with the National Institutes of Health (NIH) on early predictors and medical cost of several rare diseases.

In 2016-17, Russo served as Vice President of Health Services at Boston-based Harvard Pilgrim Health Care and Insurance Council Chair and board member of the Massachusetts Health Care Quality Partners. He supported the creation of a framework for utilization, quality management and reimbursement that facilitated the launch of the virtual hospital or hospital-at-home program, Medicallyhome. From 2007-2016, Russo was Medical Director at Independence Blue Cross in Philadelphia, where he leveraged predictive algorithms codesigned with the data science team to implement disease management and telemedicine initiatives for patients with heart failure.

“Dr. Russo is a clinician with a notable track record of driving new innovation to improve patient care and outcomes,” said Alex Markowits, Founder and President/CEO of Spring Hills. “He is keenly aware of the numerous factors that can impact a person’s health and has created programs over the past decade to close critical gaps in communication and care. His background and expertise make him an ideal fit to lead the growth of our population health management program. With his experience in cardiology, Dr. Russo also will be a key asset to our specialized cardiac program for people transitioning from hospital to home after a cardiac event.”

Spring Hills operates Post-Acute Care, Assisted Living, and Memory Care communities and Home Care services for seniors and those with chronic health needs. The company is expanding to include clinical programs that will offer residents and patients uniquely comprehensive health care. Population Health Management is fully integrated into the clinical programs, including the recently established Cardiac Program.

Spring Hills Population Health associates build relationships with the patient and family while in the hospital to ensure a smooth transition to Spring Hills and provide support during their stay and for 90 days post-discharge. Services include care coordination, personal communication, follow-up appointments, home visits, remote monitoring and removing social or financial barriers to care. Through Population Health

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Rhythm Pharmaceuticals Announces Publication of Results from Phase 3 Clinical Trials of Setmelanotide in The Lancet Diabetes & Endocrinology

Largest studies in POMC and LEPR deficiency obesities demonstrate that treatment with setmelanotide reduced body weight and hunger

BOSTON, Oct. 30, 2020 (GLOBE NEWSWIRE) — Rhythm Pharmaceuticals, Inc. (Nasdaq:RYTM), a late-stage biopharmaceutical company aimed at developing and commercializing therapies for the treatment of rare genetic disorders of obesity, announced today that results from two pivotal Phase 3 studies evaluating setmelanotide in proopiomelanocortin (POMC) deficiency obesity and leptin receptor (LEPR) deficiency obesity were published in The Lancet Diabetes & Endocrinology. As previously reported, data from the studies demonstrate that treatment with setmelanotide, the company’s melanocortin-4 receptor (MC4R) agonist, led to statistically significant and clinically meaningful reductions of weight and hunger.

“Results from Rhythm’s pivotal Phase 3 studies, which are the largest studies to date in POMC and LEPR deficiency obesities, provide evidence regarding the safety and efficacy of setmelanotide and we believe they validate its potential long-term use as a novel treatment for severe obesity and hyperphagia,” said co-author Peter Kühnen, M.D., Institute for Experimental Pediatric Endocrinology, Charité Universitätsmedizin Berlin, Germany. “It is important to recognize the signs of these rare genetic disorders because we may soon have a targeted treatment option available for the first time for obesity disorders caused by impairments of the MC4R pathway.”

Rhythm initially reported positive topline data from the Phase 3 studies in August 2019 and subsequently presented updated data in a late-breaking research forum during the 37th Annual Meeting of The Obesity Society at ObesityWeek® 2019.

Eight of 10 participants with POMC deficiency obesity (80%; P<0.0001 compared with historical data) and five of 11 participants with LEPR deficiency obesity (45%; P=0.0001 compared with historical data) achieved at least 10 percent weight loss at approximately one year. The mean percent change in “most hunger” score in participants aged 12 years and older was -27.1 percent (n=7; P=0.0005) in POMC deficiency obesity and -43.7 percent (n=7; P<0.0001) in LEPR deficiency obesity. Consistent with prior clinical experience, setmelanotide was generally well-tolerated in both trials. The most common adverse events were injection site reaction, skin hyperpigmentation, and nausea.

“These results are significant because, as we know from natural history data, individuals living with POMC or LEPR deficiency obesity consistently experience substantial weight gain each year beginning in early childhood, and we would not expect any of these patients to be able to achieve 10 percent weight loss over the course of a year without continued treatment,” said co-author Karine Clément, professor of nutrition at Pitié-Salpêtrière hospital and Sorbonne University in Paris. “These data and the significant unmet need to address the obesity and hyperphagia caused by rare genetic disorders of obesity underscore the importance of testing for genetic variants that may impair MC4R activation and lead to severe obesity.”

In May 2020, Rhythm announced that the U.S. Food and Drug Administration (FDA) accepted the company’s New Drug Application (NDA) for setmelanotide for the treatment of POMC deficiency obesity and LEPR deficiency obesity, granted Priority Review of the NDA and assigned a Prescription Drug

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Genprex Unveils New Branding for Upcoming Combination Clinical Trials in Non-Small Cell Lung Cancer

New branding will support upcoming combination clinical trials, including the Company’s trial combining REQORSA™ immunogene therapy drug with AstraZeneca’s Tagrisso®, which received FDA Fast Track Designation earlier in 2020

Genprex, Inc. (“Genprex” or the “Company”) (NASDAQ: GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today announced the launch of new branding for its upcoming oncology clinical trials combining its lead drug candidate, REQORSA(quaratusugene ozeplasmid), with AstraZeneca’s Tagrisso® (osimertinib), which received U.S. Food and Drug Administration (FDA) Fast Track Designation earlier this year, and for the combination of REQORSA with Merck’s Keytruda® (pembrolizumab), for the treatment of non-small cell lung cancer (NSCLC).

These trials will use the trial brand “Acclaim,” which the Company believes evokes its enthusiasm and the hope these trials represent for NSCLC patients and the oncology community. Acclaim-1 will be used to identify the REQORSA and Tagrisso combination clinical trial, and Acclaim-2 will be used to identify the REQORSA and Keytruda combination clinical trial.

“We are enthusiastically preparing for our upcoming clinical trials and are excited to launch the adoption of this branding,” said Rodney Varner, President and Chief Executive Officer of Genprex. “We believe the Acclaim brand communicates our passion for providing hope to NSCLC patients for important new treatment options in the fight against this devastating disease and aligns us with the clinical, medical and patient communities.”

The trial brand was developed in order to encourage early exposure of the Company’s clinical programs to the broad audience that Genprex’s business addresses, including patients, healthcare practitioners, clinical investigators, investors, employees and others. Genprex plans to initiate the Acclaim-1 clinical trial and the Acclaim 2 clinical trial in the first-half of 2021. Acclaim-1 is a Phase 1/2 clinical trial using a combination of REQORSA with Tagrisso in patients with late stage NSCLC with mutated epidermal growth factor receptors (“EGFRs”) whose disease progressed after treatment with Tagrisso. Acclaim-2 is a Phase 1/2 clinical trial using a combination of REQORSA with Keytruda in NSCLC patients who are low expressors (1 to 49%) of the protein, programmed death-ligand 1 (PD-L1).

About Genprex, Inc.

Genprex, Inc. is a clinical-stage gene therapy company focused on developing potentially life-changing therapies for patients with cancer and diabetes. Genprex’s technologies are designed to administer disease-fighting genes to provide new therapies for large patient populations with cancer and diabetes who currently have limited treatment options. Genprex works with world-class institutions and collaborators to develop drug candidates to further its pipeline of gene therapies in order to provide novel treatment approaches. The Company’s lead product candidate, REQORSA™ (quaratusugene ozeplasmid), is being evaluated as a treatment for non-small cell lung cancer (NSCLC). REQORSA has a multimodal mechanism of action that has been shown to interrupt cell signaling pathways that cause replication and proliferation of cancer cells; re-establish pathways for apoptosis, or programmed cell death, in cancer cells; and modulate the immune response against cancer cells. REQORSA has also

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Northwestern Medicine Partners with Caption Health to Introduce New Artificially Intelligent Ultrasound Systems into Clinical Practice

Northwestern Medicine Partners with Caption Health to Introduce New Artificially Intelligent Ultrasound Systems into Clinical Practice

PR Newswire

CHICAGO, Oct. 28, 2020

Northwestern Memorial Hospital First in Country to Adopt Caption AI in Multiple Clinical Settings

CHICAGO, Oct. 28, 2020 /PRNewswire/ — Northwestern Memorial Hospital is the first hospital in the United States to purchase Caption Health’s artificial intelligence (AI) technology for ultrasound, Caption AI. The FDA cleared, AI-guided ultrasound system enables healthcare providers to acquire and interpret quality ultrasound images of the human heart, increasing access to timely and accurate cardiac assessments at the point of care.

Performing an ultrasound exam is a complex skill that takes years to master. Caption AI enables clinicians—including those without prior ultrasound experience—to quickly and accurately perform diagnostic-quality ultrasound exams by providing expert turn-by-turn guidance, automated quality assessment and intelligent interpretation capabilities. The systems are currently in the hospital’s emergency department, medical intensive care unit, cardio-oncology clinic and in use by the hospital medicine group.

“Through our partnership with Caption Health, we are looking to democratize the echocardiogram, a stalwart tool in the diagnosis and treatment of heart disease,” said Patrick McCarthy, MD, chief of cardiac surgery and executive director of the Northwestern Medicine Bluhm Cardiovascular Institute, a group involved in the early development of the technology. “Our ultimate goal is to improve cardiovascular health wherever we need to, and Caption AI is increasing access throughout the hospital to quality diagnostic images.”

Caption AI emulates the expertise of a sonographer by providing real-time guidance on how to position and manipulate the transducer, or ultrasound wand, on a patient’s body. The software shows clinicians in real time how close they are to acquiring a quality ultrasound image, and automatically records the image when it reaches the diagnostic-quality threshold. Caption AI also automatically calculates ejection fraction, or the percentage of blood leaving the heart when it contracts, which is the most widely used measurement to assess cardiac function.

“Northwestern Medicine has been a tremendous partner in helping us develop and validate Caption AI. We are thrilled that they are bringing Caption AI into key clinical settings as our first customer,” said Charles Cadieu, chief executive officer and co-founder of Caption Health. “The clinical, economic and operational advantages of using AI-guided ultrasound are clear. Most important, this solution increases access to a safe and effective diagnostic tool that can be life-saving for patients.”

Point-of-care ultrasound (POCUS) has a number of benefits. Increased usage of POCUS contributes to more timely and accurate diagnoses, more accurate monitoring and has been shown to lead to changes in patient management in 47% of cases for critically ill patients. POCUS also allows patients to avoid additional visits to receive imaging, as well as providing real-time results that can be recorded into a patient’s electronic medical record.

“I think the most exciting part is that Caption AI allows our intensive care unit (ICU) providers to do a point-of-care, real-time ultrasound for a sick patient,” said James “Mac”

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Is Northwest Biotheraputics a Buy Ahead of Its Brain Tumor Vaccine Clinical Data Release?

When it comes to winning biotech stocks this year, coronavirus vaccine developers automatically come to many investors’ minds. However, one of the top-performing stocks in the sector is Northwest Biotherapeutics (OTC:NWBO), which focuses on immunotherapies that treat cancer, and has returned a staggering 408% since January.

The main reason why investors are so excited about Northwest Biotherapeutics’ prospects is that the company’s 14-year-long phase 3 clinical trial to evaluate its one and only immunotherapy candidate, DCVax-L, as a treatment for glioblastoma, has concluded. Should you consider buying the stock in anticipation of the data release? Let’s find out together. 

3-D illustration of a dendritic cell.

Image source: Getty Images.

Treatment background

Glioblastoma is a deadly form of brain cancer prevalent in up to 15% of people with brain tumors. Even after patients receive standard of care (SOC) treatments consisting of surgery, chemotherapy, and radiotherapy, their median survival time comes down to just 15.5 months in historical studies. DCVax-L is an experimental immunotherapy that seeks to stimulate patients’ own immune systems to fight cancer growth. 

The potential biologic has been in phase 3 clinical trials since December 2006. In the study, all glioblastoma patients receive SOC treatments, while a random portion also receives DCVax-L via upper arm injections. A key trial endpoint requires at least 233 patient deaths out of a total of 331 participants enrolled to calculate a survival benefit for DCVax-L, if any. The company completed its study on July 24, and the data is currently being reviewed by statisticians. In the meantime, speculations on the results have ranged from wildly enthusiastic to pessimistic from excited investors and short-sellers. 

The bullish case

The bullish case for Northwest Biotherapeutics stock is straightforward: The DCVax-L clinical trial was supposed to wrap up as early as November 2016, but had to keep going because the projected number of deaths had not occurred by then.

Around the time when the trial was enrolling, only 3% of glioblastoma patients who received SOC survived over five years. Due to extremely low survival rates for patients who receive SOC treatments, bullish investors argue that there is no other logical explanation for the clinical study going into overtime than DCVax-L keeping patients alive longer than expected. 

The bearish case

The bearish case is a lot more complicated.

Clinical trials investigating experimental biologics for deadly diseases with a lack of therapeutic options usually have pre-planned interim analyses. DCVax-L’s phase 3 trial had two such analyses built into the study.

The analysis is conducted by an independent data-monitoring committee (DMC) that can recommend that the trial stop early if an experimental therapy demonstrates statistically meaningful efficacy against SOC treatments. This way, the biologic can quickly advance to the approval stage in order to save more lives.

Northwest Biotherapeutics’ DMC carried out two interim analyses on the DCVax-L study in 2017 and 2018 (more on this later). Both times, however, the company published the DMC’s findings as blinded, and the trial continued. Unfortunately, that doesn’t make any sense at all in the context of

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Study in Nature Medicine shows superior patient outcomes in LLS’s Beat AML clinical trial

Rye Brook, NY (Monday, October 26, 2020) – Patients participating in The Leukemia & Lymphoma Society’s (LLS) groundbreaking precision medicine Beat AML Master Clinical Trial had superior outcomes compared to acute myeloid leukemia (AML) patients who opted for standard chemotherapy treatment, according to findings published today in the prestigious Nature Medicine journal.

The Beat AML trial achieved its primary endpoint by showing genomic analysis of the leukemia cells to identify AML subtypes can be completed within an unprecedented seven days, giving patients, caregivers and their doctors ample time to make a more personalized treatment decision without risking the patient’s chance for survival.

In other key findings, the study demonstrated a paradigm shift in how patients diagnosed with AML should be treated, proving that using genetic information to match patients to targeted therapies leads to better survival rates than the traditional one-size-fits all treatment approach.

AML is an extremely fast-moving cancer of the marrow and blood, affecting 21,000 people in the U.S. a year, and killing 10,000. For decades patients have been given the same treatments almost immediately upon diagnosis because waiting allows the cancer cells to grow out of control. This standard of care involves either infusion of a combination of two chemotherapies, cytarabine and daunorubicin, or treatment with a so-called hypomethylating agent, a drug that unleashes signals allowing the cancer cells to die.

“The study shows that delaying treatment up to seven days is feasible and safe, and that patients who opted for the precision medicine approach experienced a lower early death rate and superior overall survival compared to patients who opted for standard of care,” said John C. Byrd, MD, D. Warren Brown Chair of Leukemia Research of The Ohio State University, and one of the Beat AML leads and corresponding author of the study. “This patient-centric study shows that we can move away from chemotherapy treatment for patients who won’t respond or can’t withstand the harsh effects of the same chemotherapies we’ve been using for 40 years and match them with a treatment better suited for their individual case.”

Going on the Offensive Against AML


Recognizing the urgent need to do better for AML patients, LLS launched this clinical trial in fall 2016 to test multiple novel targeted therapies at major cancer centers across the U.S., in newly diagnosed AML patients aged 60 and older. In a historic first for cancer clinical trials, LLS is the first non-profit health organization to sponsor a trial and hold the IND (Investigational New Drug) application from the U.S. Food and Drug Administration. Beat AML partnered with Foundation Medicine Inc. to employ next generation genomic sequencing to rapidly analyze the patients’ cancer cells, and identify the patients’ AML subtype so they can be given a targeted therapy within a safe timeframe.

“The breadth of this collaboration, with every clinician, cancer center, pharmaceutical partner and all of the many operations and technical support companies, all unified in working toward the common goal of building a new model for tackling this challenging

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