On October 28, 2019 Deciphera Pharmaceuticals, Inc. (Nasdaq:DCPH), a clinical-stage biopharmaceutical company addressing key mechanisms of tumor drug resistance, reported data from its ongoing Phase 1b/2 clinical study of rebastinib, an oral TIE2 kinase inhibitor, in combination with paclitaxel at the AACR (Free AACR Whitepaper)-NCI-EORTC AACR-NCI-EORTC (Free AACR-NCI-EORTC Whitepaper) International Conference on Molecular Targets and Cancer Therapeutics (EORTC-NCI-AACR) (Free ASGCT Whitepaper) (Free EORTC-NCI-AACR Whitepaper) in Boston (Press release, Deciphera Pharmaceuticals, OCT 28, 2019, View Source [SID1234549915]). In addition, the Company also presented data from preclinical studies of DCC-3116, a potential first-in-class autophagy inhibitor to treat mutant RAS cancers.

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"Both of these datasets highlight the broad applicability of Deciphera’s kinase switch control platform and our potential to address unmet needs in oncology," said Matthew L. Sherman, M.D., Executive Vice President and Chief Medical Officer of Deciphera. "We look forward to continuing Part 2 of our Phase 1b/2 study of rebastinib in combination with paclitaxel with the insights garnered from Part 1 of the study. We also look forward to advancing the IND-enabling studies for DCC-3116."

Rebastinib

The Phase 1b/2 study of rebastinib in combination with paclitaxel is a two-part, open-label, multicenter study assessing the safety, tolerability, anti-tumor activity and pharmacokinetics of multiple doses of rebastinib in patients with advanced or metastatic solid tumors. Data presented today are from 43 patients from Part 1 of the study, including 24 patients from the rebastinib 50 mg oral twice a day (BID) with paclitaxel 80 mg/m2 IV cohort and 19 patients from the rebastinib 100 mg oral BID with paclitaxel 80 mg/m2 IV cohort. Preliminary results from Part 1 included:

Encouraging preliminary anti-tumor activity was observed in both dose cohorts, with objective responses seen across a heavily pre-treated patient population, including patients with prior exposure to paclitaxel. Objective responses were seen in eight patients including ovarian (3), breast (2), carcinosarcoma (2), and peritoneal mesothelioma (1), seven of whom had prior therapy with paclitaxel or docetaxel. A best response of partial response (PR) was observed in 5 of 24 patients in the 50 mg BID dose cohort and 3 of 19 patients in the 100 mg BID dose.
Exposure to rebastinib was dose-proportional at the 50 mg BID and 100 mg BID doses when given in combination with paclitaxel.
Mean circulating Ang-2 levels increased with exposure to higher doses of rebastinib in combination with paclitaxel, indicating TIE2 inhibition.
Rebastinib in combination with paclitaxel was generally well-tolerated, with similar frequency of treatment-emergent adverse events (TEAEs) between the two dose cohorts, and most TEAEs were consistent with first-in-human studies of rebastinib or known to be associated with treatment with paclitaxel.
Based on the observed frequency of muscular weakness in preliminary data from the ongoing Part 2 portion of the study with the 100 mg BID dose, the recommended phase 2 dose (RP2D) was changed to 50 mg BID.
DCC-3116

DCC-3116 is designed as a potential autophagy inhibitor by selectively targeting ULK kinase. Autophagy is a cellular pathway that has been shown to be upregulated in mutant RAS cancers and that also mediates resistance to inhibitors of the RAS signaling pathway. Subject to favorable investigational new drug (IND)-enabling studies and filing and activation of an IND application, Deciphera intends to develop DCC-3116 for the potential treatment of mutant RAS cancers in combination with inhibitors of downstream effector targets including RAF, MEK, or ERK inhibitors (MAPK inhibitors) as well as with direct inhibitors of mutant RAS. Preclinical data presented today included the following:

DCC-3116 was shown to be a potent, selective, and tight-binding inhibitor of ULK kinase.
DCC-3116 inhibited phosphorylation of the ULK substrate ATG13 in cancer cells and exhibited synergy in vitro in combination with MAPK inhibitors in inhibiting cancer cell growth.
Oral doses of DCC-3116 led to sustained inhibition of ULK activity as shown by the inhibited phosphorylation of the ULK substrate ATG13 in vivo.
DCC-3116 exhibited synergy with MAPK inhibitors in tumor growth inhibition in mouse models.
A copy of each poster presentation is available at www.deciphera.com.

About Rebastinib

Rebastinib is an investigational, orally administered, potent and selective inhibitor of the TIE2 kinase, the receptor for angiopoietins, an important family of vascular growth factors in the tumor microenvironment that also activate pro-tumoral TIE2 expressing macrophages. In a Phase 1 clinical study, biomarker data have demonstrated rebastinib-induced increases in the TIE2 ligand angiopoietin 2, providing evidence of TIE2 inhibition. Rebastinib is currently being evaluated in a Phase 1b/2 clinical study in combination with paclitaxel (NCT03601897) and in a Phase 1b/2 clinical study in combination with carboplatin (NCT03717415).

About DCC-3116

DCC-3116 is a potential first-in-class small molecule designed to inhibit cancer autophagy, a key tumor survival mechanism, by inhibiting the ULK kinase. Subject to favorable investigational new drug (IND)-enabling studies and filing and activation of an IND application, expected in mid-2020, Deciphera intends to develop DCC-3116 for the potential treatment of mutant RAS cancers in combination with inhibitors of downstream RAS effector targets including RAF, MEK, or ERK inhibitors as well as with direct inhibitors of mutant RAS.

On October 28, 2019 AstraZeneca reported positive progression-free survival (PFS) results for Imfinzi (durvalumab) and tremelimumab, an anti-CTLA4 antibody, when added to chemotherapy, from the Phase III POSEIDON trial in previously-untreated Stage IV (metastatic) non-small cell lung cancer (NSCLC) (Press release, AstraZeneca, OCT 28, 2019, View Source [SID1234549914]).

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The trial met a primary endpoint by showing a statistically significant and clinically meaningful improvement in the final PFS analysis in patients treated with the combination of Imfinzi and a broad choice of five standard-of-care platinum-based chemotherapy options vs. chemotherapy alone. The triple combination of Imfinzi plus tremelimumab and chemotherapy also demonstrated a statistically significant and clinically meaningful PFS improvement vs. chemotherapy alone as a key secondary endpoint. The safety and tolerability of Imfinzi was consistent with its known safety profile. The triple combination delivered a broadly similar safety profile to the Imfinzi and chemotherapy combination and did not result in increased discontinuation of therapy.

José Baselga, Executive Vice President, Oncology R&D, said: "The POSEIDON trial provides evidence of the efficacy of Imfinzi in patients with Stage IV non-small cell lung cancer. Clinical benefit was observed in a trial population that included a high proportion of patients with squamous disease and multiple choices of chemotherapy regimens. Additionally, the potential to add tremelimumab to Imfinzi and chemotherapy may present an important treatment approach in this challenging setting, especially taking into consideration the favourable safety profile."

The POSEIDON trial will continue to assess the additional primary endpoint of overall survival (OS) with data anticipated in 2020. AstraZeneca will submit the results for presentation at a forthcoming medical meeting and plans to share the results with health authorities.

Imfinzi is also being tested in Stage IV NSCLC as monotherapy in the Phase III PEARL trial, and in earlier stages of disease as part of an extensive Immuno-Oncology programme in lung cancer.

Imfinzi is approved in the curative-intent setting of unresectable, Stage III NSCLC after chemoradiation therapy in 53 countries, including the US, Japan and across the EU, based on the Phase III PACIFIC trial.

About POSEIDON

The POSEIDON trial is a randomised, open-label, multi-centre, global, Phase III trial of Imfinzi plus platinum-based chemotherapy or Imfinzi, tremelimumab and chemotherapy vs. chemotherapy alone in the 1st-line treatment of patients with metastatic NSCLC. The trial population included patients with either non-squamous or squamous disease and the full range of PD-L1 expression levels. POSEIDON excluded patients with a mutation in the epidermal growth factor receptor (EGFR) or anaplastic lymphoma kinase (ALK) gene. In the experimental arms, patients were treated with a flat dose of 1,500mg of Imfinzi with four cycles of chemotherapy once every three weeks or Imfinzi plus 75mg of tremelimumab, followed by maintenance therapy with Imfinzi or Imfinzi and one dose of tremelimumab on a once-every-four-weeks dosing schedule. In comparison, the control arm allowed up to six cycles of chemotherapy. Pemetrexed maintenance therapy was allowed in all arms in patients with non-squamous disease if given during the induction phase.

The trial is being conducted in 153 centres across 18 countries, including the US, Europe, South America, Asia and South Africa. Primary endpoints include PFS and OS for the Imfinzi plus chemotherapy arm. Key secondary endpoints include PFS and OS in the Imfinzi plus tremelimumab and chemotherapy arm.

About Stage IV NSCLC

Lung cancer is the leading cause of cancer death among both men and women and accounts for about one-fifth of all cancer deaths.1 Lung cancer is broadly split into NSCLC and SCLC, with 80-85% classified as NSCLC.2 Within NSCLC, patients are classified as squamous, representing 25-30% of patients, or non-squamous, the most common type representing approximately 70-75% of NSCLC patients.2 Stage IV is the most advanced form of lung cancer and is often referred to as metastatic disease.3 Lung cancer patients are most commonly diagnosed after the tumour has spread outside of the lung.4 For these patients with metastatic disease, prognosis is particularly poor, as only 1 in 10 will be alive five years after diagnosis.5

About Imfinzi

Imfinzi is a human monoclonal antibody that binds to PD-L1 and blocks the interaction of PD-L1 with PD-1 and CD80, countering the tumour’s immune-evading tactics and releasing the inhibition of immune responses.

Imfinzi is approved for unresectable, Stage III NSCLC in 53 countries including the US, Japan, and across the EU, based on the Phase III PACIFIC trial. Imfinzi is also approved for previously-treated patients with advanced bladder cancer in 11 countries, including the US.

As part of a broad development programme, Imfinzi is also being tested as a monotherapy and in combination with tremelimumab, an anti-CTLA4 monoclonal antibody and potential new medicine, as a treatment for patients with NSCLC, small-cell lung cancer, bladder cancer, head and neck cancer, liver cancer, biliary tract cancer, cervical cancer and other solid tumours.

About tremelimumab

Tremelimumab is a human monoclonal antibody and potential new medicine that targets the activity of cytotoxic T-lymphocyte-associated protein 4 (CTLA-4). Tremelimumab blocks the activity of CTLA-4, contributing to T cell activation and boosting the immune response to cancer. Tremelimumab is being tested in a clinical trial programme in combination with Imfinzi in NSCLC, bladder cancer, head and neck cancer, liver cancer and blood cancers.

About AstraZeneca in lung cancer

AstraZeneca has a comprehensive portfolio of approved and potential new medicines in late-stage clinical development for the treatment of different forms of lung cancer spanning several stages of disease, lines of therapy and modes of action. We aim to address the unmet needs of patients with EGFR-mutated tumours as a genetic driver of disease, which occur in 10-15% of NSCLC patients in the US and EU and 30-40% of NSCLC patients in Asia, with our approved medicines Iressa (gefitinib) and Tagrisso (osimertinib), and ongoing Phase III trials ADAURA, LAURA, and FLAURA2 as well as the Phase II combination trials SAVANNAH and ORCHARD.6-8

Our extensive late-stage Immuno-Oncology programme focuses on lung cancer patients without a targetable genetic mutation which represents approximately three-quarters of all patients with lung cancer.9 Imfinzi, an anti-PDL1 antibody, is in development for patients with advanced disease (Phase III trials POSEIDON, PEARL, and CASPIAN) and for patients in earlier stages of disease including potentially-curative settings (Phase III trials AEGEAN, ADJUVANT BR.31, PACIFIC-2, PACIFIC-4, PACIFIC-5, and ADRIATIC) both as monotherapy and in combination with tremelimumab and/or chemotherapy.

About AstraZeneca’s approach to Immuno-Oncology (IO)

IO is a therapeutic approach designed to stimulate the body’s immune system to attack tumours. Our IO portfolio is anchored by immunotherapies that have been designed to overcome anti-tumour immune suppression. We believe that IO-based therapies offer the potential for life-changing cancer treatments for the clear majority of patients.

We are pursuing a comprehensive clinical-trial programme that includes Imfinzi (anti-PDL1) as monotherapy and in combination with tremelimumab (anti-CTLA4) in multiple tumour types, stages of disease, and lines of therapy, using the PD-L1 biomarker as a decision-making tool to define the best potential treatment path for a patient. In addition, the ability to combine our IO portfolio with radiation, chemotherapy, small targeted molecules from across our Oncology pipeline, and from our research partners, may provide new treatment options across a broad range of tumours.

About AstraZeneca in oncology

AstraZeneca has a deep-rooted heritage in oncology and offers a quickly-growing portfolio of new medicines that has the potential to transform patients’ lives and the Company’s future. With at least six new medicines to be launched between 2014 and 2020, and a broad pipeline of small molecules and biologics in development, the Company is committed to advance oncology as a key growth driver for AstraZeneca focused on lung, ovarian, breast and blood cancers. In addition to AstraZeneca’s main capabilities, the Company is actively pursuing innovative partnerships and investments that accelerate the delivery of our strategy, as illustrated by the investment in Acerta Pharma in haematology.

By harnessing the power of four scientific platforms – Immuno-Oncology, Tumour Drivers and Resistance, DNA Damage Response and Antibody Drug Conjugates – and by championing the development of personalised combinations, AstraZeneca has the vision to redefine cancer treatment and, one day, eliminate cancer as a cause of death.

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.