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Calithera to Host Webcast Conference Call for Analysts and Investors During European Society for Medical Oncology (ESMO) Congress 2019 – GlobeNewswire

SOUTH SAN FRANCISCO, Calif., Sept. 26, 2019 (GLOBE NEWSWIRE) -- Calithera Biosciences, Inc. (Nasdaq: CALA), a clinical-stage biotechnology company focused on discovering and developing novel small molecule drugs for the treatment of cancer and other life-threatening diseases, today announced it will host a conference call and audio webcast on Monday, September 30 at 5:30 a.m. Pacific Time (8:30 a.m. ET) to review data from the Calithera clinical program presented at the European Society for Medical Oncology (ESMO) Congress 2019. Calithera management will review data from the ENTRATA study evaluating the glutaminase inhibitor telaglenastat and data presented on INCB001158, an oral arginase inhibitor.

Details of the ESMO 2019 oral late-breaking presentation for telaglenastat are as follows:

Date:Saturday, September 28, 2019, 8:30 a.m. CET, Barcelona AuditoriumSession Title: Proffered Paper 1-Genitourinary tumors, non-prostateAbstract Title: "ENTRATA: Randomized, double-blind, phase 2 study of telaglenastat (CB-839) + everolimus vs. placebo + everolimus in patients with advanced/metastatic renal cell carcinoma (RCC)."R. Motzer, et al.Abstract:LBA54Presenter: Chung-Han Lee, M.D., PhD of the Memorial Sloan Kettering Cancer Center

Details of the oral presentation for INCB001158 are as follows:

Date:Sunday, September 29, 2019, 4:30 p.m. CET, Malaga AuditoriumSession Title: Proffered Paper Developmental therapeuticsAbstract Title: "Phase 1 study of the arginase inhibitor INCB001158 (1158) alone and in combination with pembrolizumab in patients with advanced/metastatic solid tumors."A. Naing, et al.Abstract:440O Presenter: Aung Naing, M.D., FACP, Associate Professor, Department of Investigational Cancer Therapeutics, Division of Cancer Medicine, The University of Texas MD Anderson Cancer Center

Link: Abstract is available at https://www.esmo.org/Conferences/ESMO-Congress-2019

To participate in the Calithera ESMO 2019 Call, please dial (855) 783-2599 (domestic) or (631) 485-4877 (international) five minutes prior to the start of the call and provide the conference ID 1469186. To access the live audio webcast or the subsequent archived recording, visit the Investors section of the Calithera website at http://www.calithera.com. The webcast will be recorded and available for replay on Calitheras website for 30 days.

About Telaglenastat

Telaglenastat is an investigational first-in-class, novel glutaminase inhibitor specifically designed to block glutamine consumption in tumor cells. RCC tumors commonly exhibit metabolic alterations that increase their dependence on glutamine. In preclinical studies, telaglenastat produced synergistic antitumor effects when used in combination with standard-of-care RCC therapies. On June 17, 2019, Calithera announced that a randomized Phase 2 trial of telaglenastat plus everolimus versus everolimus plus placebo (ENTRATA) met its primary endpoint of improving progression free survival, demonstrating proof of concept for telaglenastat in patients with advanced RCC. The ongoing CANTATA trial is a global, randomized, double-blind trial designed to evaluate the safety and efficacy of telaglenastat plus cabozantinib versus placebo plus cabozantinib in patients with advanced or metastatic RCC.

About INCB001158 (CB-1158)

INCB001158 (CB-1158) is an investigational first-in-class, novel small molecule arginase inhibitor. Arginase is an enzyme that suppresses the immune-mediated destruction of tumors by depleting levels of a key amino acid, L-arginine, from the tumor microenvironment. A number of cell types in the tumor microenvironment, including myeloid-derived suppressor cells, macrophages, and neutrophils, can secrete arginase. L-arginine deprivation can act via nutrient sensor pathways to exert several suppressive effects on T-cell function, inhibiting proliferation, decreasing cytokine production, and diminishing expression of the T-cell receptor CD3 chain. Arginase activity may thus impair T-cell mediated anti-tumor responses. INCB001158 is being developed in a global collaboration with Incyte Corporation.

About Calithera

Calithera Biosciences is a clinical-stage biopharmaceutical company pioneering the discovery and development of targeted therapies that disrupt cellular metabolic pathways to preferentially block tumor cells and enhance immune-cell activity. Driven by a commitment to rigorous science and a passion for improving the lives of people impacted by cancer and other life-threatening diseases, Calithera is advancing a pipeline of first-in-clinic, oral therapeutics to meaningfully expand treatment options available to patients. Calithera is headquartered in South San Francisco, California. For more information about Calithera, please visit http://www.calithera.com.

Forward Looking Statements

Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as "may," "will," "expect," "anticipate," "estimate," "intend," "poised" and similar expressions (as well as other words or expressions referencing future events, conditions, or circumstances) are intended to identify forward-looking statements. These statements include those related to the safety, tolerability and efficacy of Calitheras product candidates, the overall advancement of Calitheras product candidates in clinical trials, the unmet need in the treatment of patients with advanced disease, and Calitheras plans to continue development of its product candidates. Because such statements are subject to risks and uncertainties, actual results may differ materially from those expressed or implied by such forward-looking statements. The product candidates that Calithera develops may not progress through clinical development or receive required regulatory approvals within expected timelines or at all. In addition, clinical trials may not confirm any safety, potency or other product characteristics described or assumed in this press release. Such product candidates may not be beneficial to patients or successfully commercialized. The failure to meet expectations with respect to any of the foregoing matters may have a negative effect on Calithera's stock price. Additional information concerning these and other risk factors affecting Calithera's business can be found in Calithera's most recent Quarterly Report on Form 10-Q filed with the Securities and Exchange Commission, and other periodic filings with the Securities and Exchange Commission at http://www.sec.gov. These forward-looking statements are not guarantees of future performance and speak only as of the date hereof, and, except as required by law, Calithera disclaims any obligation to update these forward-looking statements to reflect future events or circumstances.

SOURCE:Calithera Biosciences, Incorporated

CONTACT:

Jennifer McNealeyir@Calithera.com650-870-1071

MEDIA CONTACT:Hannah Hurdlehannahhurdle@sambrown.com 805-338-4752

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Calithera to Host Webcast Conference Call for Analysts and Investors During European Society for Medical Oncology (ESMO) Congress 2019 - GlobeNewswire

UCI School of Medicine researchers lead an international team for $4.3M NIH BRAIN Initiative award for new brain mapping tool – Newswise

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Newswise Irvine, CA - September 26, 2019 Professors Xiangmin Xu and Rozanne Sandri-Goldin, in collaboration with Professors Bert Semler and Todd Holmes at the University of California, Irvine, School of Medicine, were awarded a three-year, $4.3 million Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative grant. Working together with a dream team of US and Chinese scientists, they will lead the development of a new brain mapping tool for neuroscience research.

The mapping tool is based on genetically engineered herpesviruses. Herpes simplex virus (HSV) type 1, strain 129 (H129) will be harnessed as an anterograde, monosynaptic viral tracer with high labeling efficacy and low toxicity for neural circuit analysis.

Current versions of genetically modified H129 herpesviruses are limited primarily by high virulence and toxicity, said Xu. We have a comprehensive plan to reduce the toxicity and also to enhance signal outputs and generate variants carrying different functional payloads. Ultimately, we will be able to create a new set of safe, effective and validated anterograde-directed viral vectors.

Expected to have broad impact, the new tool will be made available through the UCI Center for Virus Research where it can be disseminated to the entire neuroscience community.

"The development of trans-synaptic viral tracers is an important component of the BRAIN Initiative, said Xu.At present, the lack of viral-based anterograde, monosynaptic tracing tools with high signal strength and low toxicity is a critical gap in neuroscience, preventing researchers from gaining a full understanding of how the brain works.

The team is excited about the prospect that new recombinant H129 viral vectors will lead to transformative tools for neuroscience research, and in the more distant future, clinical applications for human diseases such as Alzheimer's disease, Parkinson's disease and epilepsy. Xu and Sandri-Goldin have assembled a collaborative, interdisciplinary team composed of virologists and systems neuroscientists to develop the new neural analysis tool. Other principal investigators include Professor Gregory D. Horwitz from the University of Washington and Professor Min-Hua Luo from the Wuhan Institute of Virology, Chinese Academy of Sciences.

Their research proposal, "Genetically engineered anterograde monosynaptic viral tracers for multi-species neural circuit analysis, was submitted in response to a special RFA as part of the BRAIN Initiative: Development and Validation of Novel Tools to Probe Cell-Specific and Circuit-Specific Processes in the Brain.

About the NIH BRAIN Initiative

The Brain Research through Advancing Innovative Neurotechnologies (BRAIN) Initiative is aimed at revolutionizing our understanding of the human brain. By accelerating the development and application of innovative technologies, researchers will be able to produce a revolutionary new dynamic picture of the brain that, for the first time, shows how individual cells and complex neural circuits interact in both time and space. Long desired by researchers seeking new ways to treat, cure, and even prevent brain disorders, this picture will fill major gaps in our current knowledge and provide unprecedented opportunities for exploring exactly how the brain enables the human body to record, process, utilize, store, and retrieve vast quantities of information, all at the speed of thought. For more information, visit: https://www.braininitiative.nih.gov/

About the UCI School of Medicine

Each year, the UCI School of Medicine educates more than 400 medical students, as well as 200 doctoral and masters students. More than 600 residents and fellows are trained at UC Irvine Medical Center and affiliated institutions. The School of Medicine offers an MD; a dual MD/PhD medical scientist training program; and PhDs and masters degrees in anatomy and neurobiology, biomedical sciences, genetic counseling, epidemiology, environmental health sciences, pathology, pharmacology, physiology and biophysics, and translational sciences. Medical students also may pursue an MD/MBA, an MD/masters in public health, or an MD/masters degree through one of three mission-based programs: the Health Education to Advance Leaders in Integrative Medicine (HEAL-IM), the Leadership Education to Advance Diversity-African, Black and Caribbean (LEAD-ABC), and the Program in Medical Education for the Latino Community (PRIME-LC). The UCI School of Medicine is accredited by the Liaison Committee on Medical Accreditation and ranks among the top 50 nationwide for research. For more information, visit som.uci.edu.

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UCI School of Medicine researchers lead an international team for $4.3M NIH BRAIN Initiative award for new brain mapping tool - Newswise

Navigating the Clinical Trial Maze – Curetoday.com

Cutting-edge treatment is not out of reach, thanks to a little guidance from the Cancer Research Institute.

Currently, 4,000 active clinical trials involve immunotherapy drugs, which are examining T cell targets, cell therapy, vaccines and other immunomodulators. CRI works to help fund and advance this research.

Founded in 1953 by Helen Coley Nauts, the daughterof Dr. William B. Coley more commonly known as the Father of Cancer Immunotherapy and her friend Oliver R. Grace Sr., CRI has conducted research in this space for 65 years. Immunotherapy treats cancer by using the bodys immune system to prevent, target, control and eliminate the disease.

Decades of research have led to breakthrough treatments that mobilize our own immune systems to hunt down and eliminate cancer cells, wherever they are in the body, Jill ODonnell-Tormey, who holds a doctorate in philosophy, cell biology and anatomy, and serves as CEO and director of scientific affairs at CRI, said in an interview with CURE. Its a more sophisticated, dynamic and durable approach than other cancer treatments and may prove to be the best way to keep pace with cancers that evolve over time. Immunotherapies have also been proven to synergize with other forms of cancer treatment, increasing response rates while minimizing negative side effects and reducing risk of recurrence in some cancers.

The Food and Drug Administration has approved seven checkpoint inhibitors, a specific type of immunotherapy, to treat different cancers: Bavencio (avelumab), Imfinzi (durvalumab), Keytruda (pembrolizumab), Libtayo (cemiplimab), Opdivo (nivolumab), Tecentriq (atezoli- zumab) and Yervoy (ipilimumab).

A lot of research must still be done, according to ODonnell-Tormey. She encourages patients and their caregivers to be open to clinical trials, which give patients an opportunity to receive cutting-edge treatments under the watchful care of an expert team.

Many patients who participate in clinical trials describe the experience as very positive, often feeling part of the research team while cognizant that they are contributing to science and potentially helping future patients with cancer, she said. They can expect to receive lots of information about the study and the rationale supporting it, along with around-the-clock access to a clinical team that can answer any questions patients might have. While the treatments being tested are investigational, its important for patients to understand that new drugs or drug combinations undergo extensive laboratory testing before they are given to humans.

Often, the most challenging part of the process is finding a clinical trial. Not all health care providers discuss these studies with patients, and navigating some websitescan be difficult. CRI offers a Clinical Trial Finder to help patients learn about the basics and what to consider before enrolling, as well as connect with navigators who will walk them through the process.

Patients are asked to fill out a disease-specific questionnaire. The navigator needs to know the primary diagnosis, including tumor type; stage or extent of disease, such as if it has spread; and any treatment history. Then the navigator searches a national database of immunotherapy trials to find the best match for the patient.

Beyond locating trials and connecting to trial sites, our navigation is really customized education that varies by patient, said Caroline Melendez, director of client services at EmergingMed, the company that runs CRIs Clinical Trial Finder. Some people are very familiar with their diagnosis and treatment history, but those who have very little information are offered guidance about what to ask their health care team. Once a patient has identified trial matches, the navigator can help explain the differences between the types of treatment modalities and phases of trials being offered in their match results.

Enrollment time varies by trial and site, and patientsgo through an informed consent process, Melendez said. Next, they learn about the specific trial and undergo an examination to determine eligibility. The process can take several days, a few weeks or longer, she said.

We encourage every patient to ask this question: Is there an interesting clinical trial available to me today? Melendez said. The decision to enroll in a clinical trial is entirely dependent upon whether a compelling new therapy is in development. Everyones situation is unique, and patient interest in clinical trials should be informed by discussions with their medical team each time they have to make a treatment decision.

Several myths surround clinical trials, such as that they are a last resort or that patients are treated like guinea pigs. Another common misconception is that being on a trial means theres a chance youll receive placebo rather than treatment, ODonnell-Tormey said. This is not true except in cases where there are no existing treatments proven to improve patient outcomes. Generally, patients will receive some form of therapy, whether its standard of care, a new drug or a combination of the two.

Patients should investigate all options, educate them- selves and then make treatment decisions. Trials open and close all the time, but generally speaking, there are trialsfor patients at almost every phase of the journey, newly diagnosed to relapsed, Melendez said. Its never too early to get educated and connected to a clinical trial navigator, but it can be too late.

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Navigating the Clinical Trial Maze - Curetoday.com

The nanoengineer working to mend broken hearts – UNSW Newsroom

When Hossein Tavassoli first told his mother that he was researching heart disease, she responded with But, youre an engineer...

She was right, of course Hosseins undergraduate degree was in material engineering but at the time, he found it difficult to explain the intersections between biology and engineering. Now, not only is he a nanoengineer working in a tissue regeneration lab, but he is also an avid science communicator.

Its very interdisciplinary, says Hossein, who is completing the biology component of his PhD at UNSW Medicine and the engineering component at Swinburne University of Technology.

Hossein began working with the heart several years ago after finding himself interested in the heart-blood development and regeneration research of UNSW biologists Dr Vashe Chandrakanthan and Professor John Pimanda. Fortunately, they were intrigued by the technology Hossein was working on, and a collaboration began.

The heart is the most important organ, with heart disease the number one killer in the world, Hossein says.

My goal is to find a way for mending broken hearts or, in other words, to find a solution for cardiac problems.

During their first seven days of life, baby mice can self-repair broken heart tissue.

They develop this ability while in the embryo, which although impossible for scientists to view under a microscope could hold the clue to repairing heart damage in humans.

We cannot put a mouse embryo under the microscope, but what I do is model what is going on in the embryonic heart in a microchip, at a microscale.

The microchip that Hossein nanoengineered has been embedded with mice heart cells. It was built to mimic the embryonic heart cells natural environment, enabling the researchers to study how the regenerative cells communicate with each other. Knowledge of how this works could be applied to repairing human heart tissue, although Hossein speculates this technology is at least a decade away. In order to expand their research, additional project funding is needed.

Hossein sees many benefits to using nanotechnology in biomedicine.

First of all, its cheap. You dont need to have lots of plastic, petri dishes, culture media all those things we need for the cell culture. Secondly, you dont need too many animals or cells or patients. Instead of 10-100 million cells, what I need in that tiny family is like 100 or 1000 cells.

Or, in a simple way, instead of needing a hundred mice for each series of experiments, I just need one or two.

Hossein observes the cells interacting via a microscope connected to a supercomputer. Visually, it looks like a microchip. It has channels, it has valves it is like a simplified model of a heart.

When the cells interact in just the right way, it starts to pulse just like a heartbeat.

Hossein is lost for words when remembering the first time he saw the cells start beating.

His work, he explains, differs from many other PhD students. Instead of working from 9-5, he needs to visit the lab at all hours sometimes as late as midnight to feed the cells. One particular lab visit stands out to him.

I came to the lab on Friday to add culture media, which is food for the cells. The day after, I was at Coogee Beach with some friends and said, I should go, I need to check on my cells.

His friends were confused, but it was normal routine for Hossein. It was then he realised that the cells were effectively his babies.

Only when he arrived, they werent beating.

I thought, Oh no, something is wrong! He quickly added the culture media, put them in the incubator, and waited.

A few hours later, they started beating again.

It was incredible, Hossein remembers.

For Hossein, when the cells are beating, they are a symbol of hope and life.

"Everything in our world starts with that beating. And everything finishes with that beating. When that line becomes straight I've seen this in my life, with my family you see that everything is gone. As long as there is beating, there is hope."

Everything in our world starts with that beating. And everything finishes with that beating. When that line becomes straight I've seen this in my life, with my family you see that everything is gone.

As long as there is beating, there is hope.

Hossein knew that his family and friends were proud of his achievements but was frustrated at his inability to properly explain his research to them.

He remembers the specific moment when that changed.

Hossein was taking an Uber to university one night (to feed the cells, he recalls) when the driver asked what he studied. Knowing that the answer was convoluted, Hossein kept his response simple: Im an engineer, he said.

An engineer? the driver responded. Then why am I dropping you off at the medicine building?

Hossein then had a realisation: if he could explain his work to an Uber driver, then perhaps he could also explain it to his mum.

From that moment, he tried to articulate his research to any driver who asked. He tested new ways of describing his work and kept the ones that worked best.

Soon, something surprising happened Hossein developed a love for science communication.

I realised that it is important to explain what I'm doing, but in simple words, so that anyone can understand.

The practice paid off. Earlier this year, Hossein was a finalist in FameLab Australia (a live annual science communication competition), a shortlisted speaker at TEDx Melbournes Open Mic, and last year was a finalist in AMP Amplify Ignite (a national PhD pitch competition). He now shares his research via Twitter.

Science communication is an art, he says. For me, informing and educating is more important than doing just research all the time.

And, he adds, I now get five-star ratings on Uber!

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The nanoengineer working to mend broken hearts - UNSW Newsroom

OncLive Presents State of the Science Summit on Non-Small Cell Lung Cancer – Business Wire

CRANBURY, N.J.--(BUSINESS WIRE)--OncLive, the nations leading multimedia resource focused on providing oncology professionals with the most current and insightful information they need to offer the best patient care, will host its latest State of the Science Summit on Non-Small Cell Lung Cancer (NSCLC) on Thursday, Oct. 3, from 5-9 p.m. at the DoubleTree by Hilton Hotel Cleveland, in Ohio. The chair for the summit will be Nathan Pennell, M.D., Ph.D., director of the lung cancer medical oncology program and assistant professor of medicine at Cleveland Clinic.

This interactive and educational meeting will analyze and discuss novel treatments for patients with NSCLC. The expert presenters will explore a wide variety of informative topics surrounding NSCLC, such as updates in immunotherapy in stage 4 squamous NSCLC, epidermal growth factor receptor-positive NSCLC, screening for NSCLC, anaplastic lymphoma kinase-positive and ROS1 NSCLC, targetable biomarkers in NSCLC, new standard of care for stage III NSCLC, new immunotherapy combinations in stage 4 nonsquamous NSCLC and updates in the management of small-cell lung cancer. The presenters will also engage in a peer exchange and address audience questions.

The presenters for the summit include the following:

State of the Science Summit is a premier conference series hosted by OncLive that features medical experts from across the nation discussing treatment options. Each summit integrates academic and community-based physicians and health care professionals across key disciplines, from medical and surgical oncology to hematology.

Registration is free and open to all health care professionals, and food and beverages will be served. For more information and to register, visit https://www.onclive.com/meetings/soss or contact Kayla Collins at kcollins@onclive.com.

About OncLive

A digital platform of resources for practicing oncologists, OncLive offers oncology professionals information they can use to help provide the best patient care. OncLive is a brand of MJH Life Sciences, the largest privately held, independent, full-service medical media company in the U.S. dedicated to delivering trusted health care news across multiple channels.

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OncLive Presents State of the Science Summit on Non-Small Cell Lung Cancer - Business Wire

Greenleaf Health Expands Services to Support Cell & Gene Therapy – Business Wire

WASHINGTON--(BUSINESS WIRE)--Greenleaf Health, Inc., a leading Food and Drug Administration (FDA) regulatory consulting firm, today announces that the firm has expanded its portfolio of services to guide companies developing cell and gene therapy products. Greenleafs team of regulatory experts will be led by Karen Midthun, M.D., former Director of the FDAs Center for Biologics Evaluation and Research (CBER), and John Taylor, former FDA Counselor to the Commissioner and Principal Deputy Commissioner.

REGULATORY LANDSCAPE: Cell & Gene Therapy

The rapidly evolving fields of cell and gene therapy offer the possibility of novel treatments, and perhaps ultimately cures, for devastating and intractable illnesses. In response to what the FDA has called a "turning point in the development of these technologies and their application to human health, new policies have been introduced to address the development of safe and effective cell and gene therapies.

With innovation often comes uncertainty. In the case of cell and gene therapy products, the FDA has raised concerns about developers operating outside of the existing regulatory paradigm. To prevent this, the FDA has clarified the regulatory framework for regenerative medicine products and announced near-term enforcement actions aimed at ensuring compliance by companies developing and manufacturing cell and gene therapies.

Greenleafs expanded services support companies striving to introduce cell and gene therapy products to patients. The firms team of experts has a robust blend of technical skill and FDA institutional knowledge that spans all therapeutic areas and quality, manufacturing, and compliance systems. By working cross-functionally, Greenleaf ensures that clients have the comprehensive, specialized support needed to understand and navigate the complex regulatory landscape for cell and gene therapies.

FULL-SERVICE SUPPORT

Members of Greenleafs Drug and Biological Products Team work together with the firms Product Quality, Manufacturing, and Compliance Team to deliver guidance on cell and gene therapy products.

Product Development & Review

With the expert direction of Karen Midthun, M.D., Greenleafs team of advisors assists sponsors of cell and gene therapies by optimizing FDA interactions and submissions to support development and regulatory review. Greenleaf also helps sponsors understand and respond to the FDA requirements applicable to various cellular products, and provides guidance to sponsors of cell and gene therapies to treat rare and ultra-rare diseases on ways to maximize trial design using appropriate clinical endpoints and natural history study data to aid efficient product development.

Quality, Manufacturing & Compliance

Greenleafs Product Quality, Manufacturing, and Compliance Team, led by John Taylor and supported by the firms network of independent compliance experts, offers credible, informed guidance to help manufacturers of cell and gene therapies comply with the FDAs multiple current GXP regulations. Greenleaf experts provide strategic and technical support for establishing manufacturing and quality controls; pre- and postapproval inspection readiness; compliance assessments; evaluating and responding to FDA regulatory correspondence; and engaging with CBERs Advanced Technologies Team.

UNMATCHED EXPERTISE

Greenleaf is comprised of experts with a combined total of more than 250 years of FDA experience. The firms team of advisors demonstrates unmatched levels of skill in its specialties of drug and biological products and product quality, manufacturing, and compliance. Greenleafs Cell and Gene Therapy Team, led by Dr. Karen Midthun and John Taylor, is guided by decades of regulatory experience in senior FDA positions, global public health organizations, academia, and industry.

Karen Midthun, M.D.Principal, Drug & Biological Products

Dr. Midthun contributes specialized insight informed by her regulatory, research, and clinical experience to FDA-regulated entities developing cell and gene therapies. Dr. Midthun joined Greenleaf following a distinguished 28-year career in public health, of which 22 years were dedicated to the FDA.

An infectious disease physician by training, Dr. Midthun most recently served as the Director of the FDAs Center for Biologics Evaluation and Research (CBER). During her FDA tenure, Dr. Midthun played a critical role in facilitating policy and technology development in the areas of cell, tissue, and gene therapies, blood products, and vaccines.

John Taylor, J.D.President, Greenleaf Health, and Principal, Compliance & Regulatory Affairs

Taylor has held many high-profile positions at the FDA, as well as senior leadership roles within industry. Taylors wealth of regulatory experience, robust technical skills, and unique strategic perspective are unmatched. Clients working with Greenleafs Product Quality, Manufacturing, and Compliance Team benefit from Taylors vast FDA institutional knowledge.

Taylor joined Greenleaf following a distinguished 20-year career at the FDA, where he served in multiple leadership positions, including as the FDAs Acting Deputy Principal Commissioner, FDA Counselor to the Commissioner, Acting Deputy Commissioner for Global Regulatory Operations and Policy, and Associate Commissioner for Regulatory Affairs.

ABOUT GREENLEAF

Greenleaf Health provides strategic and technical guidance to pharmaceutical, biotechnology, and medical device companies researching, developing, and manufacturing innovative solutions to pressing global public health challenges.

The firm includes former leaders and regulatory professionals from the FDA, Capitol Hill, top global pharmaceutical and medical device companies, leading law firms, and the top U.S. biotechnology trade organization. Greenleafs blend of former FDA officials and industry experts provides a unique set of capabilities when advising entities regulated by the FDA.

For more information on Greenleafs cell and gene therapy services and Greenleaf Health, visit greenleafhealth.com.

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Greenleaf Health Expands Services to Support Cell & Gene Therapy - Business Wire

Emmaus withdraws European application for sickle cell drug that got US approval – MedCity News

A U.S. biotech company has withdrawn its application seeking approval for a drug to treat sickle cell disease in Europe following a negative review by a body of the E.U.s drug regulation authority.

Torrance, California-based Emmaus Life Sciences said Thursday that it was withdrawing its marketing authorization application with the European Medicines Agency for Xyndari (glutamine), which it markets as Endari in the U.S., after a negative opinion by the EMAs Committee for Medicinal Products for Human Use, or CHMP. The CHMP stated that the data in the application did not show the drug was effective at reducing the number of sickle cell disease crises or hospital visits. It had previouslyissueda negative opinion in May.

Shares of Emmaus were down 9 percent Friday morning on the over-the-counter market and were down more than 20 percent when markets closed.

The drug, marketed as Endari in the U.S., has had Food and Drug Administration approval to treat sickle cell disease in patients aged 5 and older since 2017. Earlier his month, the FDA accepted an application for accelerated approval for Global Blood Therapeutics voxelotor, another drug to treat sickle cell disease.

Concerns expressed in the CHMPs opinion in May included a higher discontinuation rate among patients taking the drug than among those taking placebo, and data on how the drug worked for them was not available, with the committee concluding that the way their data were dealt with was not appropriate. Many patients in a supportive study also dropped out early, and the higher number of patients in the Xyndari arm than in the placebo arm who had received another drug, hydroxyurea, may have influenced the results.

According to data from the Phase III study on the ClinicalTrials.gov database, the trial enrolled 230 patients, of whom 152 were randomized to the Endari/Xyndari arm and 78 of whom were randomized to placebo. Of those in the treatment arm, 55 did not complete the study, compared with 19 of those in the placebo arm, yielding respective discontinuation rates of about 36 percent and 24 percent.

One sickle cell disease expert noted that the drug seems to have been approved in the U.S. because of the limited treatment options for sickle cell disease and there being few problems with safety, but it still raised other issues.

There were multiple methodological concerns about the pivotal study of [Endari] to prevent acute complications of sickle cell anemia, wrote Dr. John Strouse, a hematologist and associate professor of medicine at Duke University, in an email.

The FDAs Oncologic Drugs Advisory Committee had expressed similar concerns to those of CHMP about the higher discontinuation rate in the treatment arm. ODAC nevertheless voted to support the drugs overall efficacy and safety profile.

According to the Phase III datathat led to FDA approval, patients in the Endari arm experienced a median three sickle cell crises, two hospitalizations and 6.5 days in the hospital, compared with a respective median four crises, three hospitalizations and 11 days in the hospital among those receiving placebo. The Endari arm also showed an increase in the number of days before a sickle cell crisis occurred and a lower number of patients experiencing acute chest syndrome.

Because we have demonstrated the efficacy of Xyndari, as supported by the data from the trials conducted, we are disappointed by the CHMPs position, Emmaus CEO Yutaka Niihara said in a statement Thursday. We remain committed to the patients who suffer from sickle cell disease and will continue to endeavor to broaden our global patient base while identifying new clinical uses for L-glutamine, obtaining additional patients and distribution partners, and through ongoing community and physician outreach.

Niihara said the company was seriously considering a decentralized approval procedure on a country-by-country basis, though he was not available to elaborate further.

Photo: virusowy, Getty Images

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Emmaus withdraws European application for sickle cell drug that got US approval - MedCity News

Disabling one protein might one day lead to a cure for the common cold – Science News

An uncommon way of thinkingmay be bringing scientists one step closer to a cure for the common cold.

Researchers have identifieda key protein in humans that some viruses use to multiply inside of human cells.Disabling that protein, instead of attacking the virus itself, may prevent infections from spreading. In mice and human cells engineered to lack thisprotein, the viruses couldnt replicate, Jan Carette, a microbiologist at Stanford University School of Medicine,and colleagues report September 16 in Nature Microbiology.

Its not quite a cure forthe common cold, but its an interesting step forward, says Ellen Foxman, animmunologist at Yale School of Medicine who was not involved in the study.

Colds are the most common infectious disease in humans. On average, adults catch a cold two or three times each year, while children get the sniffles even more often (SN: 2/12/09). Any one of a few hundred viruses, including rhinoviruses, can cause these infections. That fact and because these viruses can mutate quickly to become resistant to drugs makes it difficult to find a cure.

Headlines and summaries of the latest Science News articles, delivered Tuesdays and Thursdays

So researchers at Stanford and the University of California, SanFrancisco focused on the human host rather than the virus. Viruses hijack cells and rely on humans own cellularmachinery to make more virus and sicken their host. The team wanted to see if itcould identify human genes that make the proteins that many viruses hijack inorder to replicate.

Using the gene-editing tool CRISPR,Carette and colleagues systematically deleted chunks of DNA to build a libraryof human cells, each missing one gene and therefore unable to make that genescorresponding protein. The researchers then infected the cells with two typesof viruses, one that causes colds and another that has been linked toneurological diseases.

Using different viralproteins like hooks, the scientists pulled out human proteins that werephysically attached to a viral protein. That let the team identify which humanproteins were interacting with viral ones an indication that the virus was using that proteinto hijack the cell.

One human protein wasrepeatedly fished out of the cells: SETD3. And experiments indicated that theviruses needed SETD3 to take over the cell. Scientists knew this protein couldaffect actin proteins, which help muscles contract. But its role in viralinfections came as a surprise.

When the researchersinjected viruses into mice engineered to lack a working version of the SETD3 gene, the mice didnt get sick. Humanlung cells that also lacked the gene remained healthy. (Lung cells are oftenused in these types of studies because they are especially susceptible to manyrhinoviruses that cause colds.)

Repeating those experimentswith similar but potentially more serious viruses suggested the approach may beeffective against more than just the common cold. Engineered human cells didntbecome infected when they were exposed to viruses that cause hand, foot and mouthdisease and a polio-like spinal cord disease called acute flaccid myelitis. Andwhen mice were exposed to these viruses, the rodents that didnt have a functioningversion of SETD3 were much morelikely to survive than those that had the working gene.

We have identified an excellent target, says Carette, of SETD3. But its unclear whetherdisabling that gene and its protein could cause other problems. While theengineered mice survived and were healthy and fertile, they werent able to push their pups out of the wombduring birth, he says, which might be related to the proteins role in musclecontractions.

Scientists dont fully understand what this gene does in the human body, and getting rid of it completely could have unknown effects, says Vincent Racaniello, a virologist at Columbia University who wasnt involved in the work. The authors show that mice lacking the gene for SETD3 are viable and resistant to infection. However, this observation does not mean that SETD3 in humans is dispensable, he wrote in an e-mail.

Instead, the researchers think their best bet is to search for a drug that blocks the human protein and its viral counterparts from interacting, or one that destroys the human protein only when it is interacting with viral ones. But those types of drugs are still a long way off. The question is always When can I buy it over the counter? Carette says. Drug development takes time.

Continued here:
Disabling one protein might one day lead to a cure for the common cold - Science News

‘Provocative’ Results With Stem Cells in Progressive MS – Medscape

STOCKHOLM A new trial of autologous mesenchymal stem cells in progressive multiple sclerosis (MS) has shown encouraging results, with significant benefits vs placebo in several measures of disability.

The double-blind placebo-controlled phase 2 study described as "very pioneering" and "provocative" by outside commentators was presented at the recent 35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) 2019.

Both intravenous and intrathecal administration of the stem cells showed beneficial clinical effects compared with placebo in terms of Expanded Disability Status Scale (EDSS) changes and several other functional outcomes, but the intrathecal route appeared superior to intravenous administration, reported Dimitrios Karussis, Hadassah University Hospital, Jerusalem, Israel.

Benefits were also noted mainly in the intrathecal group in relapse rates and several other secondary endpoints vs placebo, including timed 25-foot walk test, 9-hole peg test, several measures of cognitive function, and the rate of change of T2 lesion load on magnetic resonance imaging (MRI), as well as newer biomarkers including optimal coherence tomography, retinal nerve fiber layer thickness, and functional MRI motor network.

The study showed both intravenous and intrathecal administration of the cells appeared safe with no serious adverse events observed vs placebo.

"A phase 3 trial is warranted to confirm these findings," Karussis concluded.

Commenting for Medscape Medical News, Robert Fox, MD, Cleveland Clinic, co-chair of the session at which the study was presented, said the study was "provocative" as it showed "quite a robust change in disability trajectory and inflammatory markers despite only including a small number of patients."

"The data suggested a marked impact on these patients with progressive MS," Fox noted. "We've seen other stem cell trials but this is the one with the most provocative results which need to be understood further," he added.

Karussis explained that two small open-label clinical trials of mesenchymal stem cells have previously shown some indications of clinical benefits in MS and amyotrophic lateral sclerosis (ALS) patients in terms of stabilization of disability and some functional improvements.

"This third study is a double-blind, placebo-controlled trial to try and establish safety and the optimal route of administration intrathecal or intravenous injection in progressive MS," he said.

The study included 48 progressive MS patients with activity who had failed on at least one MS therapy and had an EDSS score of 3.0 to 6.5.

Mesenchymal stem cells were aspirated from the bone marrow of each patient, expanded in vivo, and then transplanted back into the patient intrathecally or intravenously at a dose of 1 million stem cells per kg body weight.

Each patient received two injections. For the first injection, 16 patients received stem cells by intrathecal injection, 16 received stem cells by intravenous injection, and 16 received placebo.

After 6 months the patients were crossed over and all patients who first got placebo were given stem cells (half by intrathecal and half by intravenous injection); those who first received stem cells were divided into two subgroups half received a second injection with the same route of administration as the first injection and the other half received placebo.

There was no major difference in baseline demographics in the three groups. Most patients had secondary progressive MS, with about 20% having primary progressive MS. The average EDSS at baseline was about 5.8 with an average progression over the previous year of around +0.7.

Efficacy results showed statistically significant benefits in patients receiving intrathecal stem cell injections vs placebo, with EDSS scores reducing by 0.2 vs an increase of 0.3 in the placebo group. The ambulation score improved by 0.8 points in the intrathecal stem cell group vs an increase of 1.3 with placebo.

The sum of functional scores improved by 3 points in the intrathecal stem cell group vs a worsening by 1 point in the placebo group. The mean number of relapses per patient were 0.06 in the intrathecal stem cell group vs 0.56 in the placebo group, and 94% of the intrathecal stem cell group was relapse free at the end of the study vs 53% of the placebo group.

There were also some smaller but significant improvements in some of the endpoints in the intravenous stem cell group vs placebo but not in the relapse rates, Karussis reported.

For the secondary endpoints patients receiving intrathecal stem cells had significant benefits in the 25-foot walk test, an improvement of 6% to 10% in walking speed vs a deterioration in the placebo group. The 9-hole peg test also showed positive results in the intrathecal stem cell group.

When comparing two treatments vs one treatment, only intrathecally treated patients showed superiority in each of the primary efficacy parameters compared with one treatment.

When asked how much of the effect was thought to be anti-inflammatory, Karussis replied that there appeared to be some dissociation between anti-inflammatory and other effects. "Gadolinium enhancing lesions were much less affected than some other disability parameters, including walking and functional MRI, which may indicate that the main effect was not immunomodulatory," he said.

Fox commented: "It will take a bit of diving into the data to understand how much might have been from an anti-inflammatory effect and how much was truly addressing the progressive facet of the disease."

"One concern was that the second 6 months of treatment patients who then went on placebo seemed to decline quite dramatically," Fox added. "So this begs the question of whether this is just a short-lived benefit and how often will these stem cells treatments need to be given."

Karussis and Fox have disclosed no relevant financial relationships.

35th Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) 2019: Abstract 157. Presented September 12, 2039.

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'Provocative' Results With Stem Cells in Progressive MS - Medscape

‘I’ve Been Duped’: Disabled Veteran Says He Spent Thousands at Health Center With No Improvement – NBC Bay Area

Stephanie Burnette reached out to us after our first I-Team report about an expensive nerve treatment marketed to seniors. She says she and her husband still owe thousands of dollars for treatment they say didn't work for them.

While she and her husband both have neuropathy, Carlos Dominguez does not. This was confirmed by his neurologist in June and again in August.

But after his visit to Superior Health Centers in Aug. 2019, Dominguez says he didn't know what to think.

"They tell me they're going to put me through treatment to cure me," he said.

The disabled veteran says he was drawn to Superior Health Centers by an invitation offering a free dinner and the promise of stem cell treatments.

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"That's what I keep hearing is going to help people who have arthritis, which is what I have in my knee," Dominguez said of stem cells.

To his surprise, he says they also told him he had the debilitating nerve condition called neuropathy and Superior Health warned of dire consequences.

"He told me that later on you could basically have an amputation whether it be your toes or your feet ... so how are you feeling? Of course I'm scared," he said.

Dominguez said Superior Health bombarded him with paperwork he showed us the documents and pointed out more than a dozen places he had to initial and sign.

"They keep shoving one after another after another. They don't even give you a chance to stop and you know, get your thoughts straight."

When it was all said and done, Dominguez had signed up for 2 1/2 months treatment at a cost of $15,602.

The financing was arranged by Superior Health and none of the treatment costs were covered by insurance. Dominguez's neurologist confirmed after his visit with Superior that he does not have neuropathy.

And what about the promise of stem cells? Dominguez showed us a document which says Superior Health uses "human umbilical cord tissue."

"The tissuehas no stem cells or anything that could play any role in regenerating damaged organs or tissues or anything," Kevin McCormack, a spokesman for California Institute for Regenerative Medicine said.

CIRM is the state agency that funds stem cell research. McCormack warns many clinics are making big promises with no scientific proof.

"Long term it doesn't work, it doesn't repair the damage, it doesn't restore functions. It doesn't do anything. The only thing it improves is the bank account of these clinics," he said.

Chiropractor Philip Straw first came to the NBCLA I-Team's attention when viewers reached out to our investigative unit with complaints about the neuropathy treatment they received from Optimal Health/Straw Chiropractic. The I-Team started asking questions late last year. We were told in January that the business was closing its doors, but they appear to have reopened as Superior Health Centers where Carlos went.

For example, Straw is seen in television commercials advertising neuropathy treatment. When we've called the number displayed on the commercial, it connects to Superior Health Centers.

NBCLA has tried unsuccessfully to reach Straw and an attorney for Superior Health for comment.

But in a previously issued statement, the attorney wrote:

"Philip Straw is neither practicing at the facility nor is he a professional tenant of Superior Health Centers ... While patients acknowledge that there is no guaranty that they will improve from the treatment, many patients report significant improvement."

When Dominguez heard patient Michele Botts in our first I-Team report, he said her words resonated with him.

"I go, oh boy. I've been duped. That's the first thing I thought ... I've been taken," he said.

Taken for thousands of dollars and left, he says, with no improvement and no hope.

Dominguez never received any stem cell treatments. Instead, he says he told Superior Health he wanted out of his contract and cleared of his financial obligation. He says Superior has not agreed to that.

Consumers can contact the California Chiropractic Examiners Board online here or call 916-263-5355 to speak with someone.

This story is from our sister station, NBC Los Angeles. Click here for more investigative stories from NBC stations across the county.

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'I've Been Duped': Disabled Veteran Says He Spent Thousands at Health Center With No Improvement - NBC Bay Area