On October 28, 2019 Ziopharm Oncology, Inc. ("Ziopharm" or "the Company") (Nasdaq:ZIOP), and The University of Texas MD Anderson Cancer Center reported a new research and development agreement relating to Ziopharm’s Sleeping Beauty immunotherapy program to use non-viral gene transfer to stably express and clinically evaluate neoantigen-specific T-cell receptors (TCRs) in T cells (referred to as TCR-T) (Press release, Ziopharm, OCT 28, 2019, View Source [SID1234549911]).

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"We are delighted to deepen our relationship with MD Anderson, which provides treatment to a large and diverse population of cancer patients with solid tumors," said Laurence Cooper, M.D., Ph.D., Chief Executive Officer of Ziopharm. "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."

"Cell-based immunotherapies have emerged as a powerful new option for treating patients with hematological cancers, but we have not yet had the same success for patients with solid tumors," said Ferran Prat, Ph.D., J.D., senior vice president for Research Administration and Industry Ventures at MD Anderson. "We are pleased to be working with Ziopharm to advance a new generation of cell therapies, and we are hopeful they can one day be effective in treating a broader group of our patients."

Under the terms of the new 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, the Food and Drug Administration cleared an IND application for a phase 1 clinical trial to evaluate CD19-specific CAR-T, manufactured and infused within two days of gene transfer using Ziopharm’s rapid personalized manufacture (RPM), 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.

In addition to the new research and development agreement, Ziopharm has entered a lease agreement with MD Anderson through which the company accesses additional laboratory and office space within the institution’s campus. This new facility will serve as home for Ziopharm’s expanded Houston office, under the direction of Eleanor de Groot, Ph.D., EVP, GM Cell Therapy and Drew Deniger, Ph.D., head of Ziopharm’s TCR-T cell therapy program.

On October 28, 2019 Inflammatory Breast Cancer (IBC) Research Foundation, Susan G. Komen, and the Milburn Foundation reported that they have again come together to become a powerful force against inflammatory breast cancer (IBC) – an aggressive subtype of breast cancer that is challenging to diagnose and treat (Press release, IBC Research Foundation, OCT 28, 2019, View Source [SID1234549929]). Together, the organizations raised more than $850,000 for research aimed at finding new, innovative diagnostic tools and treatment options for IBC.

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Earlier this year, IBC Research Foundation and Milburn matched all donations to Komen up to $50,000, and far exceeded their goal to raise $250,000 in funds specifically for research into IBC.

IBC is a particularly aggressive form of breast cancer because it can be missed on a mammogram, doesn’t usually present with a lump, is often misdiagnosed, and spreads quickly. In fact, about 30 percent of those diagnosed with IBC are initially diagnosed with metastatic disease, meaning their cancer has already spread to other parts of the body. Clinical trials for new therapies often either restrict enrollment of IBC patients or combine their outcomes with non-IBC patients, limiting our understanding of this form of breast cancer.

Through this partnership, a focus group of clinicians, researchers and advocates who are experts in the field of IBC convened to identify the most critical questions in IBC and IBC research. Through this patient directed conversation, several vital challenges were identified, including how inflammatory breast cancer is defined in the clinic and for research. The group has developed a set of recommendations to address the challenge of diagnosis, which will be presented at the San Antonio Breast Cancer Symposium in December.

"In our twenty years of IBC research and patient advocacy, we have consistently stressed the need for better definition and diagnostic guidelines for IBC," said Ginny Mason, Executive Director of the IBC Research Foundation. "We are very pleased that this year’s impressive matching campaign will support continued collaborative work to resolve these long-standing issues, resulting in more timely diagnosis and hopefully saving lives that would have been stolen by IBC."

"We know treatment options for IBC are lacking, and with few clinical trials focused solely on IBC, there have been few improvements in treatment," said Paula Schneider, President and CEO of Susan G. Komen. "We must continue to make IBC and metastatic breast cancer a research priority in order to help save the more than 42,000 lives lost each year in the U.S. to metastatic breast cancer. Partnerships like this help us make an even greater impact than we ever could alone."

"By working hard together, this partnership has amplified the strengths of each organization to bring a potentially significant advancement in IBC to fruition," said Bryon Davis, President and CEO of the Milburn Foundation. "Our collective work being presented at the San Antonio Breast Cancer Symposium is the first of multiple critical milestones aimed at changing the research landscape in IBC."

The partnership between the three organizations has now raised more than $2.44 million to date, thanks to earlier matching gift campaigns beginning in 2016. Funds raised have made it possible to invest in breakthrough research, including studies in IBC, aggressive and metastatic breast cancers.

On October 28, 2019 PharmaCyte Biotech (OTCQB: PMCB) reported that it has a technology that could very well change the way a host of hard-to-treat diseases are treated for the foreseeable future (Press release, PharmaCyte Biotech, OCT 28, 2019, View Source [SID1234549912]). The company is closer than ever to its upcoming Phase 2b clinical trial in locally advanced, inoperable pancreatic cancer (LAPC) that could give patients a new lease on life by shrinking their tumors enough so that the tumors can be removed surgically. It is an outcome that would not go unnoticed.

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Clinical trials are essential for the development of new treatments, and PharmaCyte will enter this upcoming trial with two essential goals in mind—the future of its pancreatic cancer treatment and the future of its technology. First, successfully shrinking tumors would address a real unmet medical need for a group of patients that no longer realizes any benefit from either of the two first-line therapies. Second, and of equal importance for the small California-based biotech, will be to utilize the company’s first-ever clinical trial to validate or prove that its signature live-cell encapsulation technology, Cell-in-a-Box, is both effective and safe to use in humans.

When asked about the importance of validating its technology in a clinical trial setting in relation to the company’s future, PharmaCyte’s Chief Executive Officer, Kenneth L. Waggoner, said, "PharmaCyte is on the precipice of completely changing the paradigm for how diseases are treated in the future. New cell lines are constantly being developed to treat a variety of diseases, particularly genetically engineered cells lines. With the validation of our technology, Cell-in-a-Box knows no bounds in helping these cell lines succeed in treating a disease in the way the cell line was designed to treat it."

During PharmaCyte’s clinical trial, it will introduce its Cell-in-a-Box technology to a public that is mostly unfamiliar with it and how it works. Cell-in-a-Box refers to a porous microcapsule that acts as a "protective cocoon" for the more than 20,000 genetically modified live cells inside. If this cellular therapy proves it can shrink tumors in a large group of patients during an FDA trial, while at the same time proving that the technology can remain where it’s placed inside the body and the cells inside the capsules can remain viable, it would certainly be welcomed news that would reverberate throughout the pancreatic cancer community—and likely well beyond that.

Commenting on PharmaCyte’s clinical trial, renowned oncologist and clinician Dr. Manuel Hidalgo, who will be the Principal Investigator for the trial, said, "The primary goal of the study is to determine the efficacy of this approach, compared to a standard of care treatment, in controlling LAPC. If this endpoint is met, the technology will be validated as a therapeutic option in patients with LAPC."

Dr. Hidalgo also knows the significance of shrinking tumors in patients with LAPC and stated, "Patients who undergo a successful operation may be indeed cured of the disease. So, if this strategy is able to increase the number of patients that can undergo surgery to remove their previously inoperable tumors, that could be a very important finding."

Prof. Walter H. Gunzburg, the co-founder and Chief Technical Officer of Austrianova, said of targeted cellular therapies and specifically Cell-in-a-Box, which Austrianova helped to develop, "This is the future of medicine, this is the new generation of how we’re going to be able to treat diseases—not only cancer but a whole plethora of other diseases. We’re going to be able to take cells; we’re going to program them to do what we want, and we’re going to put them back into patients’ bodies and they’re going to react to signals in the patient to produce medicines in the right way. So, what PharmaCyte is doing and what we’re doing with PharmaCyte is we’re actually taking the first few bold steps for the new medicines for the next generation."

Validating the Cell-in-a-Box technology in an FDA clinical trial will confirm that PharmaCyte has a role in shaping the future of treating diseases using cellular therapies. As monumental as it would be for PharmaCyte to shrink what were once considered inoperable tumors to the point that they are now made operable, validation of the technology is equally as monumental for patients worldwide who suffer from other hard-to-treat diseases and who could now find hope in Cell-in-a-Box to specifically treat some of those diseases.

PharmaCyte’s Chief Operating Officer, Dr. Gerald W. Crabtree, commenting on the upcoming clinical trial said, "Validating the technology simply means that we may have open to us a way to treat diseases for which any type of human cell can be employed—for example, stem cells. All that is needed is that the cells be pure and free from adventitious agents, so validating our technology would mean they can be derived from another human and/or genetically modified."

When Dr. Crabtree offers stem cells as an example, obviously a treatment for Type1 and insulin-dependent Type 2 diabetes has to be one of the hard-to-treat diseases at the forefront of his thinking. After all, an encapsulation technology that can live inside the body—staying exactly where that technology is implanted and acting as a protective home for the insulin-producing cells inside without any threat from the body’s immune response (eliminating the need for harmful immunosuppressive drugs), all while delivering insulin to diabetic patients—is considered the "Holy Grail" for a diabetes treatment, a disease that represents the largest healthcare crisis in the world.

Dr. Crabtree added, "The clinical trial in LAPC, if successful, will prove that the Cell-in-a-Box capsules with genetically altered human cells inside them are safe to use in other human beings. This safety aspect is very important to the FDA and this is why we have made it one of two primary objectives in our clinical trial protocol. A successful trial should also put to rest any doubts about whether the capsules can protect the cells inside from the body’s immune system attack for a reasonable amount of time."

Dr.’s Crabtree and Hidalgo agree that the treatment of numerous different cancers could very well be the future for both PharmaCyte and Cell-in-a-Box once the technology is validated. Dr. Crabtree points to the results of a published Phase 1/2 study using the Cell-in-a-Box technology in dogs that developed spontaneous mammary tumors as evidence that certain types of breast cancer where there are discrete tumor nodules could be a target for the technology.

Dr. Hidalgo expanded the list of possibilities saying, "As this technology is extremely versatile in the sense that different cell types with different genetic manipulations can be used, the range of cancer types that can be treated is vast. It is envisioned that this clinical trial will open the opportunity to explore the same technology in other hard-to-treat cancers like liver, cholangiocarcinoma, head and neck cancer, brain tumors and others. Furthermore, the range of cells that can be encapsulated in Cell-in-a-Box as well as the genetic modifications that can be made to these cells is just huge."

And the list doesn’t stop there. In addition to potential treatments for a number of cancers and for diabetes using different types of cells, the list of treatments grows exponentially when we discuss stem cells. In addition to encapsulating stem cells to treat diabetes, the encapsulation of stem cells can also be used to regenerate and repair diseased or damaged tissues in patients. These stem cell therapies are available for people with spinal cord injuries, Parkinson’s disease, Alzheimer’s disease, heart disease, strokes, burns and even cancer.

Researchers continue to develop new ways in which stem cells can be conducive to better regenerative medicine approaches, to be eventually applied in transplants. Again, protecting stem cells using PharmaCyte’s technology would solve the long-standing concern over safety and efficacy of stem cell therapy to treat diseases and conditions of organs and tissues in patients.

Today’s healthcare market offers few effective ways to treat the root causes of many diseases or conditions. In many cases, typical treatments can only manage a patients’ symptoms with medications or devices, providing only temporary symptomatic relief.

PharmaCyte’s CEO believes that validating Cell-in-a-Box will open many doors to the future of cellular therapies for his company. "What PharmaCyte is involved with can lead to a game-changing approach to treating numerous degenerative diseases. By encapsulating certain types of stem cells and protecting them, the body is given a chance to heal itself. This type of therapy is the future of medicine—medicine that offers the realistic promise of repairing damaged tissue and reversing the effects of many degenerative conditions, offering solutions for people with diseases that today are beyond repair."

It’s clear that cellular therapies could offer PharmaCyte a real opportunity to: (i) expand its own pipeline; (ii) partner with other biotechnology and pharmaceutical companies on the development of cellular treatments; and/or (iii) entertain suitors for specific indications, who have an eye on developing their own treatment(s)—all contingent upon Cell-in-a-Box performing as well as it has in the past and validating itself once and for all on the world’s biggest stage, an FDA clinical trial.

To learn more about PharmaCyte’s pancreatic cancer treatment and how it works inside the body to treat locally advanced inoperable pancreatic cancer, watch the company’s documentary video complete with medical animations at: View Source

On October 28, 2019 PierianDx, the leading clinical genomics informatics company, reported that it has closed a $27 million Series B funding round led by ATW Partners and SJF Ventures that also includes existing investors Health Catalyst Capital, Inova Health Systems, RTI International, and ARUP Laboratories (Press release, PierianDx, OCT 28, 2019, View Source [SID1234549930]).

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PierianDx provides a SaaS platform that enables the practice of clinical genomics as a standard of care and empowers the world’s most advanced molecular diagnostic labs. Founded in 2014 out of Washington University in St. Louis, PierianDx is focused on advancing cancer diagnostics and making targeted therapeutics more accessible to healthcare systems, laboratories, and patients worldwide.

Dr. Rakesh Nagarajan, Founder and Executive Chairman of PierianDx, said, "PierianDx has grown tremendously over the past 18 months and is continuing to add the clinical expertise and resources necessary to execute on our vision. Our team is uniquely skilled and dedicated to the adoption of clinical NGS around the globe."

Michael L. Sanderson, CEO of PierianDx, said, "Our new capital infusion will accelerate the commercial expansion of PierianDx’s leading clinical genomics platform in the U.S. and global markets throughout Europe, Asia, Australia, Latin America, and North America, as we continue to work with leading sequencer, assay and pharma partners in our explosive space. PierianDx is fueled by making the most advanced clinically actionable cancer care accessible to everyone, regardless of location or ability to pay."

"We are excited about leading the Series B investment in PierianDx. We believe PierianDx will change the way cancer is treated worldwide and we are proud to be investors," said Mr. Kerry Propper, Co-Founder and Managing Partner of ATW Partners.

On October 28, 2019 Applied BioMath (www.appliedbiomath.com), the industry-leader in applying systems pharmacology and mechanistic modeling, simulation, and analysis to de-risk drug research and development, reported their participation at The Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) occurring November 6th-10th in National Harbor, Maryland (Press release, Applied BioMath, OCT 28, 2019, View Source [SID1234549946]). They will present two posters at the conference Friday, November 8th.

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Katie Williams, PhD, Associate Director, Business Development, Applied BioMath will present the poster titled "A semi-mechanistic platform model to capture individual animal responses to checkpoint inhibitors in a syngeneic mouse model." In this work, we describe the generation of a model platform that captures essential aspects of the pharmacokinetics, cellular and tumor growth effects of murine surrogates of two checkpoint therapeutic antibodies, anti-PD1 and anti-CTLA4, in the CT26 syngeneic tumor model. The model describes individual animal responses regarding drug exposure, key intra-tumoral cell kinetics and tumor volume changes and provides biologically plausible explanations for the observed differences between good and poor responders to treatment with anti-PD1 or anti-CTLA4.

Jennifer Park, PhD, Director, Business Development, Applied BioMath will present the poster titled "Semi-mechanistic PK and target-occupancy modeling to support dose justification for anti-PD-L1 clinical candidate CK-301 (TG-1501) in oncology patients." In this work, a semi-mechanistic pharmacokinetic/target-occupancy (PKTO) model was developed with in vitro, preclinical and clinical data to facilitate dose selection of CK-301 (also known as TG-1501, cosibelimab), an anti-PD-L1 monoclonal antibody (mAb), for ongoing and future clinical trials in oncology patients. The model was used to compare the PK and tumor target occupancy (TO) at steady state under various dosing regimens with cosibelimab to those with three marketed anti-PD-L1 mAbs (i.e. atezolizumab, durvalumab and avelumab).

"We are excited to participate at SITC (Free SITC Whitepaper) for the first time this year!" said John Burke, PhD, Co-founder, President and CEO, Applied BioMath. "We hope to introduce the benefits of systems modeling and simulation to SITC (Free SITC Whitepaper) attendees, and how this analysis can be applied to cancer immunotherapy research and development from very early development through clinical trials."

For more information about all of Applied BioMath’s events, visit View Source