On June 15, 2021 NanoString Technologies, Inc. (NASDAQ: NSTG), a leading provider of life science tools for discovery and translational research, and the Parker Institute for Cancer Immunotherapy (PICI) reported that they are collaborating on an expansive molecular characterization project for cellular therapies(Press release, NanoString Technologies, JUN 15, 2021, View Source [SID1234584086]). The collaboration will define the characteristics that make a cell therapy effective, providing a standardized approach to developing CAR-T regimens that may improve patient outcomes across all cancer types, especially solid tumors.

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The collaboration will leverage the cell therapy expertise of PICI’s network, including world-class academic research centers, and empower research teams with NanoString’s widely recognized nCounter Analysis System and CAR-T Characterization Panel. The technology utilizes a standardized gene expression panel of 780 genes, which the team will use to profile eight different biological characteristics of existing cell therapies. By analyzing the factors that correlate with optimal cellular therapies, the team will define the characteristics that make a therapy more likely to be effective. The team will also make the findings publicly available to the scientific community through PICI’s Cancer Data and Evidence Library (CANDEL) analysis platform.

CAR-T cell therapy has produced significant advancements in the treatment of hematological malignancies, which has led to an explosion of research aimed at adapting these therapies for solid tumors. Despite this activity, several significant challenges remain. There is a particular need in the field to define key aspects of CAR-T activity, including molecular pathways that regulate effectiveness, toxicity, and persistence. This challenge is compounded by varying strategies used during the design and manufacturing process, and the variability associated with these patient-derived treatments. Overcoming these barriers can help unlock the benefits of cellular therapies for cancer patients currently in need of more effective treatments.

"By working together, NanoString and PICI will generate meaningful information from our scientific community to develop a standardized approach to cell therapy development, allowing us to better understand the attributes that make treatments effective and to ultimately improve patient outcomes," said Joseph Beechem, Ph.D., NanoString’s chief scientific officer and senior vice president of Research and Development.

"PICI’s approach is based on bringing together the brightest minds to solve cancer’s toughest problems," said Lisa Butterfield, Ph.D., PICI’s vice president of Research and Development. "This collaboration provides an important opportunity to deeply examine cell therapies and layout a road map for future development and manufacturing that can overcome the challenges of treating solid tumors."

NanoString and PICI are working together on other collaborative projects in parallel, including a multi-site validation of the GeoMx Digital Spatial Profiler and the GeoMx as a tool to characterize patient response to immunotherapy. Work is underway for both projects, which includes processing nearly 1,000 cell therapy samples from across PICI’s network with nCounter.

The team aims to provide updates on its work to the community through conferences and round table discussions.

On June 15, 2021 RayzeBio which is discovering and developing a broad pipeline of innovative targeted radiopharmaceutical drugs for cancer, reported a $108 million Series C financing led by Venrock Healthcare Capital Partners alongside new investors Perceptive Advisors, Vivo Capital, Acuta Capital Partners, Deerfield Management, and TCG X (Press release, RayzeBio, JUN 15, 2021, View Source [SID1234584085]). Also participating were all of Rayze’s existing investors: venBio Partners, Versant Ventures, Samsara BioCapital, Redmile Group, Viking Global Investors, Cormorant Asset Management, OrbiMed, LifeSci Venture Partners, Logos Capital, Alexandria Venture Investments, and others.
"The targeted radiopharmaceuticals field represents a highly clinically validated modality that, until recently, has been underappreciated," said Ken Song, M.D., president and CEO of RayzeBio. "The interest and confidence from our exceptional group of investors that has funded the company enables us to fully invest in our plans to be the leader in targeted radiopharmaceuticals for a multitude of cancer types."
With over $258 million of capital raised since the company first debuted in the second half of 2020, RayzeBio has rapidly expanded its team, infrastructure, capabilities, and pipeline.

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On June 15, 2021 OncoMyx Therapeutics, a privately-held oncolytic immunotherapy company, has extended its intellectual property (IP) portfolio to include an exclusive option to license IP rights for the use and delivery of myxoma virus to treat certain cancers from the University of Florida Research Foundation (UFRF)(Press release, OncoMyx Therapeutics, JUN 15, 2021, View Source [SID1234584084]). This IP was developed by OncoMyx cofounder Grant McFadden, Ph.D., and his collaborators, when he was a Professor at the University of Florida College of Medicine.

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"We are pleased to enter this licensing agreement with OncoMyx, the leading company for multi-armed systemic delivery in oncology and hematology," said Christopher Cogle, Professor of Medicine at the University of Florida and a Leukemia & Lymphoma Society Clinical Research Scholar. "As practicing oncologists in blood cancer clinics, we struggle to find effective treatments for our patients. The myxoma virus technology has tremendous potential to help our patients suffering from multiple myeloma, AML, myelofibrosis, lymphomas, and other blood conditions, such as graft versus host disease. Rather than using single mechanism agents, myxoma therapy combines multiple arming strategies delivered concurrently to ignite the immune system – establishing a new treatment paradigm in blood cancers."

"We have been working in collaboration with Professor McFadden on multiple myeloma for a number of years, and an important part of this technology will be to partner with leaders in myeloma centers," said Rafael Fonseca, M.D., interim director of Mayo Clinic Cancer Center and a longstanding clinical collaborator of Dr. McFadden in myeloma. "Rather than single agent approaches, there is an unmet need to simultaneously target multiple mechanisms in one delivery system to improve the treatment of multiple myeloma."

"As we develop our plans for solid tumor intravenous programs with our multi-armed myxoma platform, the unmet need in hematological malignancies is striking," said Steve Potts, Ph.D., MBA, Cofounder and Chief Executive Officer of OncoMyx. "There is no other viral approach that currently offers true multi-arming combined with systemic delivery against hematologic cancers. There have been more than a dozen peer-reviewed preclinical publications at multiple institutions demonstrating the ability of the myxoma virus to have curative potential against multiple myeloma when delivered systemically. In other hematologic malignancies like AML, the virus has also been shown in peer-reviewed studies to systemically infect and kill tumor cells, while additionally dampening the graft versus host disease response after allogeneic transplants. With the exclusive option to license these IP rights, we further strengthen our IP portfolio protecting the use of myxoma virus and myxoma virotherapies for the systemically delivered treatment of cancer."

"In modern multi-arming approaches in oncolytic viral therapy, the myxoma virus is the only platform that can be both multi-armed and systemically delivered in hematologic cancers," said Dr. McFadden. "OncoMyx has built a leading technology platform generating multi-armed myxoma virotherapies with demonstrated ability to modulate anti-tumor immunity and be systemically delivered for broad cancer killing potential. I am pleased to see OncoMyx continue to add to their IP portfolio as the company builds a pipeline of oncolytic immunotherapies that has the potential to be a new pillar in cancer care."

About Myxoma Virotherapy for the Treatment of Cancer
Myxoma is a highly immuno-stimulatory, oncolytic virus with unique qualities that make it ideal for developing multi-armed, targeted, systemic virotherapies. Because myxoma virus is not pathogenic to humans, myxoma virotherapy does not have to overcome pre-existing immunity and is highly amenable to IV delivered multi-dosing. As a large dsDNA poxvirus, myxoma is engineerable to express multiple transgenic payloads, such as immunomodulatory proteins, to target multiple points in the cancer immunity cycle. OncoMyx’s preclinical data demonstrates efficacy of multi-armed myxoma virotherapies via intravenous (IV) and intratumoral (IT) delivery in a number of tumor models across multiple cancer indications and supports a pan-tumor approach to expand the therapeutic effectiveness of immunotherapies.

On June 15, 2021 Kazia Therapeutics Limited (NASDAQ: KZIA; ASX: KZA), an oncology-focused drug development company, is pleased to report that it has entered a collaboration with the Joan & Sanford I Weill Medical College of Cornell University in the United States, to launch a phase II clinical study investigating the use of Kazia’s investigational new drug, paxalisib, in combination with ketogenesis, for glioblastoma(Press release, Kazia Therapeutics, JUN 15, 2021, View Source [SID1234584083]).

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Key Points

Research by Professor Lew Cantley, who discovered the PI3K pathway, suggests that ketogenesis may enhance the activity of PI3K inhibitors in glioblastoma, with impressive preclinical data previously published in Nature
Ketogenesis represents an alternative biochemical mechanism in which the body is fueled by fats and proteins rather than by glucose; it occurs in states such as starvation, and also in response to a ‘ketogenic diet’
Data from this study has the potential to significantly enhance the activity of paxalisib in glioblastoma, and to minimize certain side effects, including hyperglycemia (high blood sugar)
Dr Howard Fine, founding Director of the Brain Tumor Center at New York-Presbyterian Weill Cornell Medical Center, will serve as Principal Investigator; Professor Cantley will be a scientific advisor to the study
Kazia will provide support including study drug and a financial grant
Dr Fine, Principal Investigator to the study, commented, "glioblastoma remains an immensely challenging disease, and we need the most potent array of tools at our disposal in order to treat it. My lab has extensive experience of translational research in this area, and I am excited to explore the potential for a brain-penetrant PI3K inhibitor in combination with ketogenesis."

Professor Cantley, who is a scientific advisor to the study, added, "the interplay between the PI3K pathway, insulin signaling, and tumor growth has been a focus of scientific interest for some time now. Our research clearly shows the synergistic benefits of PI3K inhibition and ketosis in animal models of glioblastoma. This is an important project, designed to verify these laboratory findings in the human setting."

Ketogenesis and Glioblastoma

Cells in the human body generally rely on glucose as ‘fuel’ for their energy requirements. However, when glucose is not readily available, cells can metabolise fats and proteins to provide energy. The fats and proteins are broken down to an intermediate form known as ketones, and so this biochemical pathway is referred to as ‘ketogenesis’.

Unlike healthy cells, most tumour cells are poorly able to metabolise ketones, and so depend on glucose for their energy needs. Consequently, many researchers have experimented with ‘ketogenic diets’ as a potential treatment for cancer.[1]

In addition, scientists in Professor Cantley’s lab have shown that insulin has the potential to counteract the anti-tumor effects of PI3K inhibitors.[2] Insulin is a hormone produced by the body in response to high levels of glucose. When the body is in a state of ketosis, glucose is absent, and so insulin falls to very low levels.

For these reasons, there is a sound rationale to explore a combination of ketogenic diet and paxalisib in glioblastoma. In this study, patients will also receive metformin, a common anti-diabetic drug, which will help to further lower insulin levels.

[1] A Kapelner & M Vorsanger (2015). Medical Hypotheses. 84(3):162-168

[2] B Hopkins et al. (2018). Nature. 560:499-503

Clinical Trial Design

This study will comprise two arms. The first will contain patients with newly diagnosed glioblastoma who have unmethylated MGMT promotor status. These patients are essentially resistant to temozolomide, the existing standard-of-care therapy. The second arm will contain patients with recurrent disease, who have progressed after taking standard-of-care therapy.

In each arm, paxalisib will be combined with metformin and with a ketogenic diet. The diet will be overseen by expert clinical dieticians to ensure that it is scientifically appropriate and that patients are compliant.

An initial cohort of approximately sixteen patients will be recruited to each arm. If there are signals of activity in a given arm, that arm will be expanded to approximately thirty patients. The primary endpoint will be progression-free survival at six months (PFS6). In addition to efficacy and safety, the study will examine a range of metabolic and pharmacodynamic biomarkers to help inform future research and clinical practice. The study is expected to take approximately two years to complete.

Dr Howard Fine will serve as Principal Investigator to the study. Dr Fine is the founding Director of the Brain Tumor Center at New York-Presbyterian Weill Cornell Medical Center, and Associate Director for Translational Research at the Sandra and Edward Meyer Cancer Center at Weill Cornell Medicine. He is an internationally recognized leader in the field of neuro-oncology, with more than 30 years of experience in both laboratory and clinical research as well as in the care of patients with brain tumors. Dr Fine has built large multidisciplinary brain tumor programs at top academic institutions such as the Dana Farber Cancer Institute / Harvard Medical School and the National Institutes of Health, has cared for nearly 20,000 patients with brain and spinal cord tumors in his career, has conducted over 100 clinical trials, published over 250 papers and book chapters on brain tumors, and for over two decades has run a continuously operating translational genetic / molecular laboratory devoted to a better understanding of, and better therapies for, brain tumors.

On June 15, 2021 PTC Therapeutics, Inc. (NASDAQ: PTCT) reported that on June 11, 2021 it approved non-statutory stock options to purchase an aggregate of 118,350 shares of its common stock and 30,835 restricted stock units ("RSUs"), each representing the right to receive one share of its common stock upon vesting, to 66 new employees(Press release, PTC Therapeutics, JUN 15, 2021, View Source [SID1234584037]). The awards were made pursuant to the NASDAQ inducement grant exception as a component of the new hires’ employment compensation.

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The inducement grants were approved by PTC’s Compensation Committee on June 11, 2021 and are being made as an inducement material to each employee’s acceptance of employment with the Company in accordance with NASDAQ Listing Rule 5635(c)(4).

All stock option awards have an exercise price of $42.95 per share, the closing price of PTC’s common stock on June 11, 2021, the date of the grant. The stock options each have a 10-year term and vest over four years, with 25% of the original number of shares vesting on the first anniversary of the applicable employee’s new hire date and 6.25% of the original number of shares vesting at the end of each subsequent three-month period thereafter until fully vested, subject to the employee’s continued service with the Company through the applicable vesting dates. The RSUs each will vest over four years with 25% of the original number of shares vesting on each annual anniversary of the applicable employee’s new hire date until fully vested, subject to the employee’s continued service with the Company through the applicable vesting dates.