Why diversity in clinical trials is matter of life or death – PBS NewsHour

Cat Wise:

Brittani Powell is now-cancer free, thanks to the clinical trial. But she was lucky. According to the Food and Drug Administration, only about 30 percent of clinical trial participants for cancer drugs come from minority groups. The rest are white.

In an era of precision medicine, when drugs are being developed for and tailored to specific segments of the population, diversity is essential, because some diseases and drugs impact racial groups in different ways.

George Ocampo has been part of the Lazarex push to reverse those numbers. He couldn't work during five grueling rounds of chemo for pancreatic cancer. A clinical trial for a new treatment, two hours from home, didn't seem like an option.

Lazarex has footed the bill for his trips to the University of California, San Francisco the gas, tolls, parking, and hotel stays while he participated in the trial. They also pay for airfare for those traveling longer distances.

Those seemingly small interventions have helped Ocampo and other patients access cutting-edge care they otherwise wouldn't have received.

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Why diversity in clinical trials is matter of life or death - PBS NewsHour

InGeneron Announces Publication of Preclinical Results for its Cell Therapy in Chronic Ischemic Heart Failure – Business Wire

HOUSTON--(BUSINESS WIRE)--InGeneron, Inc., a regenerative medicine and cell therapy company, today announced the publication of promising results in developing a novel treatment for chronic ischemic heart failure using its regenerative cell therapy platform.

A newly-released research paper published in the World Journal of Stem Cells provides missing pieces of evidence for a fundamental change in the treatment of chronic ischemic heart failure, showing efficacy and safety of a novel stem cell treatment in cardiology. Patients with heart failure as a consequence of previous myocardial infarction are a large and currently underserved patient population, due to the lack of regenerative treatment options.

The publication, performed in a pig model for the study of chronic myocardial infarction, evidences for the first time that regeneration of the damaged tissue in the heart - responsible for chronic ischemic heart failure - is possible. Specifically, the study demonstrates that InGenerons fresh, uncultured, autologous adipose derived regenerative cells (UA-ADRCs) - isolated and administered at point of care - provide a significant improvement of cardiac circulatory parameters in chronic ischemic heart failure. The results show that the mean cardiac output increased by 37%, the mean left ventricular mass increased by 29% and the mean relative amount of scar volume of the left ventricular wall decreased by 21% six weeks after treatment with the cells. All results were statistically significant compared to the control group. Notably, on average only 18 gram of adipose tissue were required to recover the averaged 18 million cells injected to achieve the reported effects.

The findings represent an important step in research, laying the foundation for new frontiers on cardiac regeneration of chronic ischemic heart failure in human patients. While previous studies indicated that stem cells (including UA-ADRCs) might be of benefit in acute myocardial infarction, this benchmark had previously not been achieved by studies of autologous stem cells for chronic heart failure following myocardial infarction.

Haenel et al., the authors of the publication, attribute the success of the study to two important improvements over previous attempts. The primary success factor was the use of InGeneron's technology for isolating the stem cells at point of care. In this regard, a recent publication by Winnier et al. (PLoS One 2019;14:e0221457) demonstrated that the technology used (TransposeRT / Matrase; InGeneron, Inc., Houston, TX, USA), provides the highest published number of living, uncultured, autologous, adult pluripotent stem cells recovered per gram of adipose tissue.

The second differentiator to all previously published results for myocardial regeneration is the application method to the damaged heart. Haenel et al. administered the stem cells retrograde through the hearts venous system, precisely to the area in need of regeneration. This retrograde injection technique, combined with a temporary blockage of the coronary vein at the level of a previous arterial occlusion, allowed the stem cells to overcome the endothelial barrier and thereby created a homogenous distribution of injected cells throughout the damaged myocardial tissue.

Dr. Eckhard Alt, Executive Chair of InGeneron, Inc. and senior author of the study, commented "this therapy, which may be performed in an ambulatory setting without the known risks associated with major anticoagulation, delivers the stem cells in about 15 minutes and involves a total treatment time of approximately 3 hours. This gives hope that millions of patients suffering from chronic ischemic heart failure might benefit from rebuilding the heart with their own stem cells".

The study, entitled "Unmodified autologous stem cells at point of care for chronic myocardial infarction", by Haenel et al. was published in the World Journal of Stem Cells on October 26, 2019.

While the company is advancing its ongoing clinical programs for key orthopedic conditions, additional studies are designed to validate the clinical potential of stem cells in patients with coronary artery disease and chronic heart failure.

About InGeneron

InGeneron is a clinical stage cell therapy company enabling novel, safe and evidence-based regenerative medicine therapies. Our purpose is to set new therapeutic standards by developing treatments that unlock the healing potential of each patients own regenerative cells processed at the point of care for same-day application. We focus on helping patients who are impacted by musculoskeletal indications and are pursuing research to extend the application of our platform technology to additional treatment areas.

http://www.ingeneron.com

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InGeneron Announces Publication of Preclinical Results for its Cell Therapy in Chronic Ischemic Heart Failure - Business Wire

For 20 Years This Company Has Helped to Shape the Field of Cell Therapy – BioBuzz

How MaxCyte Has Evolved to Become Leading Technology Provider for Cell Engineering and Leveraged Its Flow Electroporation Technology and Know-How to Launch Its Own Robust CAR Pipeline

MaxCyte, located in Gaithersburg, Maryland, is a unique biotech company that is a key anchor of the robust and growing Maryland cell and gene therapy industry. Founded in 1999, MaxCyte has been helping to unlock the full potential of cell and gene therapies by empowering the product development of its many blue chip life science partners and collaborators.

The companys core technology is a proprietary, advanced cell engineering platform that is enabling leading companies like Kite Pharma, a Gilead Company, and CRISPR Therapeutics, among other major life science clients ten of the top ten pharmaceutical companies to be exact to advance their cell-based therapies for patients with high unmet medical needs. MaxCyte is a truly unique organization in that it is both a leading life sciences instrumentation company and is advancing its own portfolio of clinical-stage, cell-based therapies.

The organization has been in the cell therapy field since 2000 and was founded on proprietary technology that came from the Center for Blood Research in Boston and was spun out of Entremed, which is now known as CASI Pharmaceuticals. Among MaxCytes founders was the late Dr. John W. Holaday who served as the companys first Chairman. Dr. Holaday was a tireless advocate and champion of MaxCytes mission.

Our founding technology was for increasing oxygen offloading of red blood cells to treat hypoxia or low oxygen in the body. The whole premise of the business was to develop an instrument system that could effectively engineer cells at high volumes and high efficiency and do it close to the patient in a sterile, closed system. This is the technology we invented and what we have been focused on since we formed the company. Our mission is really pretty simple: To identify high value applications of this technology in making better medicines, stated MaxCytes CEO Doug Doerfler.

In 2004, MaxCyte secured its first deal with fellow Maryland biotech, United Therapeutics, to engineer a cell to treat pulmonary hypertension. This was the companys first official foray into the cell therapy field. Since then, the company has become a technology partner of choice for companies developing engineered cell therapies across a variety of applications.

Over the years the company has gone through several stages of evolution.

The financial crisis of 2008 required MaxCyte to adjust its approach because investments were not readily available. The company quickly had to figure out how to be a cash-neutral company: In 2009, Doerfler and his team pivoted to licensing and selling its technology to life science organizations.

Once we realized we had to survive with the revenues we could generate from our technology, we became very commercially oriented and that has really changed the dynamics of the company in a good way. Through this experience we built a lot of important relationships, stated Doerfler.

Today, the company is having a remarkable impact in the field: it now has more than 80 partnered program licenses in cell therapy with more than 45 licensed for clinical use, including six announced commercial licenses (the most recent with leading genome editing company Editas Medicine, Inc.). One such program that was announced in March 2019 is a multi-drug clinical and commercial agreement with Kite, a Gilead Company, who is using MaxCytes Flow Electroporation Technology to enable non-viral cell engineering for the development of multiple CAR-T drug candidates for up to 10 targets.

MaxCytes Flow Electroporation Technology is highly robust and is efficient in how it engineers cells and increases product potency and efficacy for companies that utilize it. MaxCyte, whose team designs, develops and manufactures its instruments at its facilities in Gaithersburg, ships its systems around the world to companies for small molecule discovery, biologics production and to make vaccines.

We provide the technology, field support, core team expertise and regulatory support to help our clients succeed, Doerfler stated. We provide the whole package to our partners that buy an instrument or license our technology.

Weve been really true to this one mission and focusing on this one technology. Weve been doing so for 20 years and its a highly robust, best-in-class technology, added Doerfler.

Having successfully navigated through the turbulence of thegreat recession that began in 2008, MaxCyte leveraged new financing strategies and entered the clinic withits first CARMA immuno-oncology cell therapy program in 2018, marking another major milestone for MaxCyte and the beginning of its own therapy pipeline. CARMA is a novel and proprietary platform for next-generation chimeric antigen receptor (CAR)-engineered cell or CAR therapies.

With MCY-M11, the first CARMA therapeutic candidate in the clinic, MaxCyte has demonstrated it can manufacture its product in a single day, which can get these critical medicines to patients faster and ultimately may reduce manufacturing costs.

When we first entered the cell therapy space with our own drug development platform, we were a bit concerned about how our licensees would feel about us competing with them in the cell therapy field. We were cautious but it has been anything but that. We now talk to our licensees and they say now youre like one of us and understand the trials and tribulation of cell therapy. Its helped us as a company to become a better partner, stated Doerfler. This field is so huge that you dont see any negative competitiveness. Theres a lot of opportunity for almost every company in the field and theres a real benefit to companies working together.

Doerfler credits the original MaxCyte core team as a major factor in the companys success. Many of these core team members are still with the company today and have contributed to building out an outstanding staff and developing the companys vibrant culture.

Its critical that we hire people who are curious and cooperative and help us solve problems. We see an application and we help our customers use our technology to build better medicines. Every time you bring a person in, the culture changesits always evolving. We are careful about who we hire and how we integrate them and help them grow, shared Doerfler.

We try to respect peoples differences and diversity is a vital part of what we doWe really work hard to make sure we are best-in-class in benefits and how we treat one another, added Doerfler. Its always a work in progress.

As a company you have to follow the science, have the people that allow you to do that and then you have to trust the data, do the work and figure out how to solve the next big problem that no ones solved before. Its really fun and its a great place to be and Maryland is a wonderful place to do it, stated Doerfler.

Over the past 8 years, Chris has grown BioBuzz into a respected brand that is recognized for its community building, networking events and news stories about the local biotech industry. In addition, he runs a Recruiting and Marketing Agency that helps companies attract top talent through a blended model that combines employer branding and marketing services together with a high powered recruiting solution.

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For 20 Years This Company Has Helped to Shape the Field of Cell Therapy - BioBuzz

Oz Azam beefs up Tmunity’s total raise to $231M as they steer toward human data in the drive to cell therapy 2.0 – Endpoints News

Stephen Hahns nomination as the new commissioner of the FDA was delivered right on schedule Friday, with the clock ticking down on the November 1 deadline President Donald Trump faced in finding a permanent replacement for Scott Gottlieb. The pending news had become the worst kept secret in Washington. But the full meaning of the move is still undetermined.

The Chief Medical Executive at MD Anderson had a rep for steering straight into confrontation, when the future of the institution was at stake. More problematic, perhaps, will be his role in dismissing Chinese researchers at a time the Trump administration has been bearing down on the Asian giant. Just how Hahn will manage drug development, which is my primary arena of interest, is going to take some time to figure out.

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Oz Azam beefs up Tmunity's total raise to $231M as they steer toward human data in the drive to cell therapy 2.0 - Endpoints News

AACR Project GENIE begins five-year research project with $36M in industry funding – The Cancer Letter

publication date: Nov. 1, 2019

Project GENIE (Genomics Evidence Neoplasia Information Exchange), an initiative by the American Association for Cancer Research, is launching a five-year, $36 million research collaboration with nine biopharmaceutical companies to obtain clinical and genomic data from an estimated 50,000 de-identified patients.

The patients are treated at institutions participating in AACR Project GENIE. The additional clinical data furthers the project goals of advancing precision oncology and powering clinical decision making through open and transparent data sharing.

The nine biopharmaceutical companies participating in the collaborative project are:

Amgen Inc.

AstraZeneca

Bayer HealthCare Pharmaceuticals Inc.

Boehringer Ingelheim

Bristol-Myers Squibb Company

Genentech, member of the Roche Group

Janssen Research & Development, LLC

Merck

Novartis

AACR Project GENIE is a publicly accessible international cancer registry of real-world data assembled through data sharing between 19 cancer centers across the world. Through the efforts of strategic partners Sage Bionetworks and cBioPortal, the registry aggregates, harmonizes, and links clinical-grade, next-generation cancer genomic sequencing data with clinical outcomes obtained during routine medical practice from cancer patients treated at these institutions.

Currently, AACR Project GENIEs registry contains clinical-grade cancer genomic sequencing data from nearly 71,000 patients. These data are linked to a limited set of clinical data, such as age, sex, primary diagnosis, and type of tumor sample analyzed (primary or metastatic).

The new collaboration will greatly expand the scope and accelerate the speed of clinical data collection.

In the first two years, the project will add prior cancer treatments, tumor pathology and clinical outcomes to the clinical data already linked with the genomic profiles of nearly 8,000 bladder, breast, colorectal, lung, pancreatic and prostate cancer patients treated at three of the institutions participating in AACR Project GENIE: Dana-Farber Cancer Institute, Memorial Sloan Kettering Cancer Center and Vanderbilt-Ingram Cancer Center.

In years three through five, this data collection will be expanded to as many cancer types as possible from all active participating institutions.

Recognizing the importance of the outputs of this project to the broader research and patient communities, and in alignment with the guiding principles of openness, transparency, and inclusion, all data generated will be made publicly available 12 months following data lock, Shawn M. Sweeney, director of the AACR Project GENIE Coordinating Center, said in a statement.

European Commission approves Astellas Xospata indication for relapsed or refractory AML

The European Commission has approved Astellas oral once-daily therapy Xospata (gilteritinib) as a monotherapy for the treatment of adult patients with relapsed or refractory (resistant to treatment) acute myeloid leukemia with a FLT3 mutation. Gilteritinib has the potential to improve treatment outcomes for AML patients with two forms of the most common mutationFLT3 internal tandem duplication and FLT3 tyrosine kinase domain mutation.

This approval is based on results from the phase III ADMIRAL trial, which investigated gilteritinib versus salvage chemotherapy in patients with relapsed or refractory FLT3mut+ AML. Patients treated with gilteritinib had significantly longer overall survival than those who received salvage chemotherapy.

Median OS for patients who received gilteritinib was 9.3 months, compared to 5.6 months for patients who received salvage chemotherapy (Hazard Ratio = 0.64 [95% CI 0.49, 0.83], P=0.0004). Rates of one-year survival were 37% for patients who received gilteritinib, compared to 17% for patients who received salvage chemotherapy.

The EC marketing authorization for gilteritinib in relapsed or refractory FLT3mut+ AML is applicable to the European Union member countries, and is also valid in Iceland, Norway and Liechtenstein. Gilteritinib was designated an orphan medicinal product and also received accelerated assessment from the European Medicines Agency earlier this year, which reduced the timeframe for approval.

Patients FLT3mut+ status can change over the course of AML treatment, even after relapse. Due to the poor outcomes associated with FLT3mut+ AML, patients FLT3 mutation status may be confirmed to help inform the best treatment approach.

FDA grants Cytotron Breakthrough Device Designation for breast, liver and pancreatic cancers

FDA granted Shreis Scalene Sciences Breakthrough Device Designation for the Cytotron, a CE-marked, whole-body therapeutic medical device.

The Center for Devices and Radiological Health granted the designation.

The companys designation request stated that The Cytotron is intended to be used to cause degeneration of uncontrolled growth of tissues. It is indicated for treating protein-linked, abnormally regenerating disorders such as neoplastic disease, by selectively targeting and enabling tissue apoptosis, allowing extended progression-free survival, with pain relief, palliation, improved quality and dignity of life. It is indicated for the treatment of solid tumors of the breast, liver, and pancreas.

Shreis, while actively pursuing collaborations for clinical trials in the current proposed indications for use, intend to also submit a request for Breakthrough designation in other solid tumors such as adult and pediatric brain tumors, lung cancer, and other life-limiting diseases.

MD Anderson, Ziopharm Oncology to expand TCR-T Program

Ziopharm Oncology Inc. and MD Anderson Cancer Center established a research and development agreement relating to Ziopharms Sleeping Beauty immunotherapy program to use non-viral gene transfer to stably express and clinically evaluate neoantigen-specific T-cell receptors in T cells.

This new agreement is a launch point to expand our TCR library and execute two new clinical trials; a trial for utilizing TCRs from the library targeting hotspot mutations in KRAS, TP53 and EGFR, and a second trial for personalized TCRs targeting patient-specific neoantigens, Ziopharm CEO Laurence Cooper said in a statement.

Under the agreement, Ziopharm commits to fund an additional $20 million for this expanded work in the TCR-T program through 2023, as well as certain milestone payments for clinical development or regulatory approval in the U.S., European Union, Japan and the rest of the world. The funding for this new agreement was included within the budget forecast provided by Ziopharm in its second quarter 2019 financial results news release and webcast commentary.

MD Anderson will receive low, single-digit royalties on net sales in the U.S. and international markets, as well as warrants for Ziopharm common stock which vest upon achievement of clinical milestones. According to institutional guidelines, MD Anderson has implemented an Institutional Conflict of Interest Management and Monitoring Plan to manage this research.

This new agreement expands the relationship between Ziopharm and MD Anderson, established under a 2015 research agreement related to CD19-specific CAR T. Earlier this month, FDA cleared an IND application for a phase I clinical trial to evaluate CD19-specific CAR T, manufactured and infused within two days of gene transfer using Ziopharms rapid personalized manufacture, as an investigational treatment for patients with relapsed CD19+ leukemias and lymphomas. Ziopharm has approximately $20 million of pre-funded R&D at MD Anderson under the prior agreement, which may now be used under the new agreement, for both the CAR T or TCR-T initiatives.

Ziopharm has entered a lease agreement with MD Anderson to access laboratory and office space within the institutions campus. This new facility will serve as home for Ziopharms expanded Houston office, under the direction of Eleanor de Groot, of GM Cell Therapy, and Drew Deniger, head of Ziopharms TCR-T cell therapy program.

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AACR Project GENIE begins five-year research project with $36M in industry funding - The Cancer Letter

Intellia Therapeutics Announces Third Quarter 2019 Financial Results – GlobeNewswire

CAMBRIDGE, Mass., Oct. 31, 2019 (GLOBE NEWSWIRE) -- Intellia Therapeutics, Inc. (NASDAQ:NTLA), reported operational highlights and financial results for the third quarter ended September 30, 2019.

In 2019, we continued to leverage the breadth of our genome editing platform to advance our in vivo and engineered cell therapy programs. We have demonstrated that we can knock out a disease-causing gene as well as introduce a functional gene to restore normal protein production. Now, we have achieved consecutive editing in vivo by combining both these edit types, further highlighting the versatility of our modular platform, said Intellia President and Chief Executive Officer, John Leonard, M.D. Our full-spectrum strategy and platform capabilities are enabling Intellias development of a robust pipeline to address a range of severe diseases. We look forward to the planned nomination of our first engineered cell therapy development candidate for acute myeloid leukemia by year-end and the submission of our first IND application for NTLA-2001 for the treatment of transthyretin amyloidosis in mid-2020.

Third Quarter 2019 and More Recent Operational Highlights

Upcoming Milestones

The Company has set forth the following for pipeline progression:

Upcoming Events

The Company will participate in the following investor events:

Third Quarter 2019 Financial Results

Financial Guidance

Intellia expects that its cash, cash equivalents and marketable securities as of September 30, 2019, as well as technology access and funding from Novartis and Regeneron, will enable Intellia to fund its anticipated operating expenses and capital expenditure requirements into the second half of 2021. This expectation excludes any potential milestone payments or extension fees that could be earned and distributed under the collaboration agreements withNovartisand Regeneron or any strategic use of capital not currently in the Companys base-case planning assumptions.

Conference Call to Discuss Third Quarter 2019 Earnings

The Company will discuss these results on a conference call today, October 31, 2019, at 8 a.m. ET.

To join the call:

A replay of the call will be available through the Events and Presentations page of the Investor Relations section on Intellias website, beginning on October 31, 2019 at 12 p.m. ET.

About Intellia Therapeutics

Intellia Therapeutics is a leading genome editing company focused on developing curative therapeutics using the CRISPR/Cas9 system. Intellia believes the CRISPR/Cas9 technology has the potential to transform medicine by permanently editing disease-associated genes in the human body with a single treatment course, and through improved cell therapies that can treat cancer and immunological diseases, or can replace patients diseased cells. The combination of deep scientific, technical and clinical development experience, along with its leading intellectual property portfolio, puts Intellia in a unique position to unlock broad therapeutic applications of the CRISPR/Cas9 technology and create a new class of therapeutic products. Learn more about Intellia Therapeutics and CRISPR/Cas9 at intelliatx.com and follow us on Twitter @intelliatweets.

Forward-Looking Statements

This press release contains forward-looking statements of Intellia Therapeutics, Inc. (Intellia or the Company) within the meaning of the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, express or implied statements regarding Intellias beliefs and expectations regarding its planned submission of an investigational new drug (IND) application for NTLA-2001 for the treatment of transthyretin amyloidosis (ATTR) in mid-2020; its plans to nominate a first T cell receptor (TCR)-directed engineered cell therapy development candidate for its acute myeloid leukemia (AML) program by the end of 2019; its plans to advance and complete preclinical studies, including non-human primate studies for its ATTR program, AML program and other in vivo and ex vivo programs; develop our proprietary LNP-AAV hybrid delivery system to advance our complex genome editing capabilities, such as gene insertion; its presentation of additional data at upcoming scientific conferences, and other preclinical data by the end of 2019; the advancement and expansion of its CRISPR/Cas9 technology to develop human therapeutic products, as well as maintain and expand its related intellectual property portfolio; the ability to demonstrate its platforms modularity and replicate or apply results achieved in preclinical studies, including those in its ATTR and AML programs, in any future studies, including human clinical trials; its ability to develop other in vivo orex vivocell therapeutics of all types, and those targeting WT1 in AML in particular, using CRISPR/Cas9 technology; the ability to continue its growth and realize the anticipated contribution of the members of its board of directors and executives to its operations and progress; the impact of its collaborations on its development programs, including but not limited to its collaborations with Regeneron Pharmaceuticals, Inc. and Novartis Institutes for BioMedical Research; statements regarding the timing of regulatory filings regarding its development programs; its use of capital, including ATM receivables, expenses, future accumulated deficit and other financial results during the third quarter of 2019; and the ability to fund operations into the second half of 2021.

Any forward-looking statements in this press release are based on managements current expectations and beliefs of future events, and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in or implied by such forward-looking statements. These risks and uncertainties include, but are not limited to: risks related to Intellias ability to protect and maintain our intellectual property position, including through our arbitration proceedings against Caribou; risks related to Intellias relationship with third parties, including our licensors; risks related to the ability of our licensors to protect and maintain their intellectual property position; uncertainties related to the initiation and conduct of studies and other development requirements for our product candidates; the risk that any one or more of Intellias product candidates will not be successfully developed and commercialized; the risk that the results of preclinical studies or clinical studies will not be predictive of future results in connection with future studies; the risk that Novartis will not continue to pursue programs it has selected through its collaboration with Intellia; and the risk that Intellias collaborations withNovartisor Regeneron or its otherex vivo collaborations will not continue or will not be successful. For a discussion of these and other risks and uncertainties, and other important factors, any of which could cause Intellias actual results to differ from those contained in the forward-looking statements, see the section entitled Risk Factors in Intellias most recent annual report on Form 10-K as well as discussions of potential risks, uncertainties, and other important factors in Intellias other filings with theSecurities and Exchange Commission. All information in this press release is as of the date of the release, andIntellia undertakes no duty to update this information unless required by law.

Intellia Contacts:

Investors:Lina LiAssociate DirectorInvestor Relations+1 857-706-1612lina.li@intelliatx.com

Media:Jennifer Mound SmoterSenior Vice PresidentExternal Affairs & Communications+1 857-706-1071jenn.smoter@intelliatx.com

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Intellia Therapeutics Announces Third Quarter 2019 Financial Results - GlobeNewswire