On October 28, 2019 Innovation Pharmaceuticals (OTCQB:IPIX) ("the Company"), a clinical stage biopharmaceutical company, is reported to highlight preclinical data by independent cancer researchers supporting the therapeutic potential of Kevetrin, the Company’s novel p53-modulating anti-cancer drug candidate, in treating Acute Myeloid Leukemia (AML) (Press release, Innovation Pharmaceuticals, OCT 28, 2019, View Source [SID1234549913]).

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Building on Kevetrin data in AML presented at the 2017 European Hematology Association (EHA) (Free EHA Whitepaper) Annual Meeting, a group of independent cancer researchers recently presented additional data at the June 2019 EHA (Free EHA Whitepaper) Annual Meeting, and at the October 2019 Italian Society of Hematology Annual Meeting.

Results showed that Kevetrin alters cellular metabolism and several key genes, including TP53 and MYC, both of which when dysfunctional are implicated in many types of cancers, including AML.

The researchers’ conclusion as presented at the June 2019 EHA (Free EHA Whitepaper) Annual Meeting:

Our results show Kevetrin alters several key genes and cellular metabolism. Along with cellular data, this study could provide a rationale for an experimental trial in AML patients, especially those carrying TP53 mutation who actually have very few therapeutic options.

A related scientific article remains under review for publication. The paper details Kevetrin’s treatment potential in AML by targeting p53 and several key leukemia-related genes.

The continued publication flow of encouraging studies related to Kevetrin’s potential in AML is particularly promising. AML accounts for almost one-third of all leukemias worldwide and has a 5-year survival rate of only 25 percent. Pre-clinical research and academic literature also support Kevetrin’s potential in combination with cancer immunotherapies.

Kevetrin was shown to be well-tolerated in a completed Phase 1 clinical trial in Advanced Solid Tumors, with a separate Phase 2a clinical trial in late-stage Ovarian Cancer showing intra-tumor p53 modulation. P53 is the most studied gene of all time, eliciting significant private and public investment—e.g., see Aprea Therapeutics, PMV Pharma, Aileron Therapeutics—given an approved p53 drug would likely have a large impact in fighting many types of cancer. Next steps within the Innovation Pharmaceuticals cancer program is to complete the necessary remaining bridging toxicology work toward developing Kevetrin in oral formulation, both to leverage its pharmacokinetics (PK) profile and provide a more patient-friendly mode of administration.

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 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 Y-mAbs Therapeutics, Inc. (the Company or Y-mAbs) (Nasdaq: YMAB) a late-stage clinical biopharmaceutical company focused on the development and commercialization of novel, antibody-based therapeutic products for the treatment of cancer, reported a clinical update on omburtamab for the treatment of central nervous system (CNS) leptomeningeal metastases from neuroblastoma and a number of additional cancer indications (Press release, Y-mAbs Therapeutics, OCT 28, 2019, View Source [SID1234549910]). Data was presented at the International Society of Pediatric Oncology (SIOP) Annual Congress in Lyon, France on October 26, 2019 by Dr. Kim Kramer from Memorial Sloan Kettering Cancer Center (MSK) in New York.

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An updated data readout as of June 30, 2019 from a single-center study at MSK (Study 03-133), where patients with CNS/leptomeningeal metastases from neuroblastoma received up to two doses of radiolabelled omburtamab, showed that for the 107 evaluable patients, the median survival had increased to 50.8 months, with the final median not yet being reached. This compared well to a median survival of 47.1 months at the prior data readout based on the first 93 patients in the study.

In addition, 68 patients diagnosed with other CNS cancers, including metastatic tumors had received a total of 201 injections of omburtamab. Injections were routinely administered in an outpatient setting. Rare self-limited adverse events included grade 1 or 2 fever, headache, vomiting; 3 injections were associated with grade 3 toxicities requiring discontinuation of therapy including chemical meningitis and increasing communicating hydrocephalus. Although not a dose limiting toxicity, myelosuppression occurred in patients who had received craniospinal radiation and at dose levels exceeding 60mCi. The primary CNS diagnoses included medulloblastoma (n=27), ependymoma (n=9), and embryonal tumors with multilayered rosettes (n=4), while metastatic tumors included sarcoma (n=9), melanoma (n=5), and other tumors (n=14). As of today, 26 of the 68 patients with these highly lethal diagnoses remain alive.

"We are excited to announce this updated data for omburtamab in CNS/leptomeningeal metastases from high-risk neuroblastoma further confirming the importance of omburtamab in addressing this unmet medical need. Further very interesting proof of concept data for other CNS cancers suggests potential clinical utility for use of omburtamab beyond neuroblastoma. Overall survival data have already been accepted by the FDA as supportive for our Breakthrough Therapy Designation, and we still plan to initiate the rolling BLA submission for omburtamab in December this year," said Thomas Gad, Founder, Chairman, President and Head of Business Development and Strategy.

Dr. Claus Moller, Chief Executive Officer further notes, "We are very pleased to see the variety of other CNS cancers and metastatic cancers treated in this trial at MSK. The experience gained from these 68 patients pave the way for future potential label expansion of compartmental use of the radiolabeled omburtamab as well as our new clinical program for omburtamab-DTPA, for which we expect to file an IND within the next few months."

Researchers at MSK developed the therapeutic products referenced in this statement, which are exclusively licensed by MSK to Y-mAbs. As a result of this licensing arrangement, MSK has institutional financial interests in the products and in Y-mAbs.

On October 28, 2019 TG Therapeutics, Inc. (NASDAQ: TGTX), a biopharmaceutical company developing medicines for patients with B-cell mediated diseases, reported that the follicular lymphoma (FL) cohort of the UNITY-NHL Phase 2b pivotal trial evaluating single agent umbralisib, the Company’s novel, once daily, PI3K delta inhibitor, met the primary endpoint of overall response rate (ORR) as determined by Independent Review Committee (IRC) for all treated patients (n=118) who have received at least two prior lines of therapy including an anti-CD20 monoclonal antibody and an alkylating agent (Press release, TG Therapeutics, OCT 28, 2019, View Source [SID1234549909]). The results met the Company’s prespecified ORR target of 40-50%. Importantly, umbralisib monotherapy appeared to be well tolerated with a safety profile consistent with previous reports.

The Company plans to present the data at a future medical conference as well as discuss the data with the U.S. Food and Drug Administration (FDA).

Michael S. Weiss, Executive Chairman and Chief Executive Officer of TG Therapeutics stated, "We are extremely pleased to announce that the UNITY-NHL follicular lymphoma cohort evaluating umbralisib monotherapy met the primary endpoint of ORR. There are no fully approved drugs for patients with follicular lymphoma that have progressed following two or more prior lines of therapy and we are excited by the potential to offer a novel treatment for this underserved population. We look forward to sharing these results with the FDA and discussing submission opportunities for accelerated approval of umbralisib in follicular lymphoma." Mr. Weiss continued, "These are very exciting times for TG and with two additional major events targeted to occur over the next several months, including commencing our first NDA filing for umbralisib in patients with relapsed/refractory marginal zone lymphoma and results from our UNITY-CLL Phase 3 trial, we expect that excitement to continue. Taken together, we see 2020 shaping up as a pivotal year where we transition from a development-stage company into a fully-integrated development and commercial organization."

ABOUT THE UNITY-NHL PHASE 2b STUDY—FOLLICULAR LYMPHOMA COHORT

The multicenter, open-label, UNITY-NHL Phase 2b study – Follicular Lymphoma cohort was designed to evaluate the safety and efficacy of single agent umbralisib, the Company’s novel, once daily, PI3K delta inhibitor, in patients with FL who have received at least two prior lines of therapy, including an anti-CD20 regimen and an alkylating agent. The primary endpoint is overall response rate (ORR) as determined by Independent Review Committee (IRC) assessment. Secondary endpoints include safety, duration of response, and progression-free survival (PFS).

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The positive ORR outcome announced today was based on 118 FL patients that received at least one dose of umbralisib and who previously had received at least two prior lines of therapy, including an anti-CD20 regimen and an alkylating agent.

CONFERENCE CALL INFORMATION

The Company will host a conference call today, Monday, October 28, 2019 at 8:30 AM ET to discuss the UNITY-NHL FL news. In order to participate in the conference call, please call 1-877-407-8029 (U.S.), 1-201-689-8029 (outside the U.S.), Conference Title: TG Therapeutics Update Call.

A live audio webcast of this call will be available on the Events page, located within the Investors & Media section, of the Company’s website at www.tgtherapeutics.com. An audio recording of the conference call will also be available for replay at www.tgtherapeutics.com, for a period of 30 days after the call.

ABOUT FOLLICULAR LYMPHOMA

Follicular lymphoma (FL) is typically a slow-growing or indolent form of non-Hodgkin lymphoma (NHL) that arises from B-lymphocytes, making it a B-cell lymphoma. Follicular lymphoma is usually not considered to be curable, and is a chronic disease. Patients can live for many years with this form of lymphoma. With an annual incidence in the United States of approximately 15,000 newly diagnosed patients1, FL is the most common indolent lymphoma accounting for approximately 20 percent of all NHL cases2.