On June 18, 2019 Torque, a clinical-stage immuno-oncology company developing Deep-Primed T Cell Therapeutics to direct immune power deep within the tumor microenvironment, reported that the U.S. Food and Drug Administration (FDA) granted Fast Track designation for Torque’s first Deep-Primed T cell immunotherapy program, TRQ-1501 (Deep IL-15 Primed T cells) (Press release, Torque Therapeutics, JUN 18, 2019, View Source [SID1234537152]). The Fast Track designation is for the treatment of relapsed or refractory solid tumors and lymphomas that express any of five tumor-associated antigens (PRAME, WT-1, SSX2, Survivin, and NY-ESO-1). Torque is currently conducting a Phase 1/2 clinical trial of TRQ-1501 for this indication.

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"Patients with relapsed or refractory solid tumors and lymphomas have a poor prognosis and limited treatment options. We are delighted to receive this Fast Track designation of TRQ-1501 for a broad, tumor-agnostic indication, which provides significant flexibility for our clinical trial program," said Becker Hewes, MD, Chief Medical Officer of Torque. "Working in close collaboration with the FDA supports our goal of improving patient outcomes in multiple difficult-to-treat solid and hematologic cancers."

The FDA’s Fast Track designation is designed to facilitate the development and expedite the regulatory review of drugs and biologics that have shown the potential to address an unmet medical need associated with a serious or life-threatening disease. Fast Track status provides for more frequent interactions with the FDA during drug development and the possibility of Priority Review of New Drug or Biologic Licensing Applications.

About TRQ-1501
TRQ-1501 is an investigational immune cell therapy produced from a patient’s own T cells, which are primed to target multiple tumor-associated antigens (PRAME, WT-1, SSX2, Survivin, NY-ESO-1) and loaded with Deep IL-15 (a multimer of IL-15 cytokine) anchored to the T cells’ surface. A Phase 1/2 clinical trial of TRQ-1501 in solid cancers and lymphoma is currently enrolling (NCT03815682) and will evaluate TRQ-1501 both as a single agent and in combination with KEYTRUDA (pembrolizumab), Merck’s anti-PD-1 therapy.

About Torque’s Deep-Primed Immune Cell Therapy Platform
Torque is developing a new class of Deep-Primed cellular immunotherapy designed to overcome the key challenges limiting broad use of cellular therapy in oncology, including the capability to target tumors that express multiple heterogeneous antigens, the ability to overcome the immunosuppressive microenvironment that shuts down T cell function, and the need for outpatient treatment with a high margin of safety. Torque uses its Deep-Priming technology to develop multi-targeted, antigen-primed T cells that carry surface-anchored immune-stimulatory drugs to drive a full immune response within the tumor microenvironment against tumors with heterogenous antigens.

Torque’s Deep-Priming platform uses advanced cell process engineering to:

prime and activate T cells to target multiple tumor antigens and
tether immune-stimulatory drugs to the surface of these multi-target T cells to direct immune activation in the tumor microenvironment
using a proprietary technology platform, without genetic engineering, for a high margin of safety.
Deep-Primed T cells both target multiple tumor antigens and pharmacologically activate an immune response with anchored cytokines. This process does not require genetic engineering of the T cells and so preserves the natural T cell receptor for delivering a regulated immune response, with the potential for a high margin of safety. In addition to antigen priming, immunomodulators are tethered to the surface of Deep-Primed T cells—initially IL-15 and IL-12 cytokines, and TLR agonists—that activate both innate and adaptive immunity. Administering these immunomodulators systemically to a patient can cause lethal toxicity by activating immune cells throughout the body. By loading precise doses of cytokines onto the surface of T cells, Deep Priming focuses the immune response to target the tumor, without systemic exposure.

In hematologic cancers, this new class of immune cell therapeutics has the potential to improve on the initial success of single-target CAR T therapeutics with expanded efficacy and also move cell therapy treatment out of the hospital with a high margin of safety. For solid tumors, Deep-Primed T cells have the potential to enable efficacy against tumors with heterogeneous antigens protected by hostile microenvironments, which are not readily addressable with the first generation of immune cell therapies.

On June 18, 2019 Medicenna Therapeutics Corp. ("Medicenna" or "the Company") (TSX: MDNA,OTCQB: MDNAF), a clinical stage immunotherapy company developing first-in-class Superkines and Empowered Cytokines, reported that it will present preliminary top-line clinical results from their Phase 2b trial of MDNA55, an IL4-guided toxin, in patients with recurrent Glioblastoma (rGBM), the most common and uniformly fatal form of brain cancer, at the Inaugural Immuno-Oncology Pharma Congress held from June 18-20, 2019 during World Pharma Week in Boston, MA (Press release, Medicenna Therapeutics, JUN 18, 2019, View Source [SID1234537151]).

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The presentation by Dr. Fahar Merchant, Chairman, President & CEO of Medicenna, will focus on safety and preliminary efficacy results from the Phase 2b clinical trial MDNA55-05, which recently completed its enrollment (N=46).

"The preliminary top-line clinical results of the Phase 2b trial of MDNA55 are very promising when compared to approved therapies for rGBM especially in patients exhibiting high levels of IL4R expression, a biomarker for more aggressive forms of brain cancer," said Dr. Fahar Merchant. "While treatment options for patients with rGBM are very limited, MDNA55 has shown early evidence of substantial clinical benefit as well as improved survival for patients. We believe these results are evidence of the opportunity for MDNA55 to become a leading treatment option for a sizeable patient population with this devastating disease."

Highlights from the presentation included:

Disease control rate of up to 83% overall achieved
To account for initial local inflammation (called pseudo-progression) that can occur up to six months after treatment with MDNA55 in some subjects (and which can mask underlying tumor response), assessment of response from the nadir (largest tumor size) was also conducted.

This demonstrated 35 of 42 subjects with tumor shrinkage or stabilization following MDNA55 treatment (disease control rate of 83%). Use of advanced imaging techniques (such as perfusion and diffusion MRI) was able to show underlying tissue response amidst inflammation and edema in some subjects.

Strong safety and promising survival results
Safety data from the Phase 2b clinical trial show a similar safety profile to previous MDNA55 trials, with no systemic toxicities, no clinically significant laboratory abnormalities and no drug-related deaths.

Current median Overall Survival (mOS) in subjects treated with low doses of MDNA55 (median 63µg; n=21) is 11.8 months. When stratified by IL4 receptor (IL4R) expression status, a biomarker for more aggressive GBM, IL4R+ve subjects (mOS 15.2 months; n=8) showed a survival advantage of seven months compared to IL4R-ve subjects (mOS 8.1 months; n=10).

The clinical trial also showed that there may be an association between pseudo-progression and longer survival. Subjects showing disease stabilization or better from nadir (indicating possible pseudo-progression) were seen to live longer than those with progressive disease (mOS of 13.7 months versus 8.5 months, respectively). These results are consistent with earlier reports suggesting that occurrence of initial pseudoprogression and immunogenic cell death following treatment with MDNA55 is associated with improved clinical prognosis and survival (Leshem and Pastan, 2019: Toxins, 11, p20).

"Glioblastoma is a uniformly fatal disease where virtually all tumors will recur, and where the Blood Brain Barrier (BBB) blocks transport of therapies to the tumor," says Dr. John H. Sampson, MD, PhD, Chair of Neurosurgery at Duke University. "New drug delivery methods, like those undertaken with MDNA55, are needed to change the treatment paradigm and potentially improve patient outcomes."

"This trial is advancing neurosurgical methods in a number of very important ways, including more precise drug delivery to the brain, potential use of IL4R expression as a biomarker, and optimal use of advanced imaging techniques to support clinical decision making," states Dr. Martin Bexon, Head of Clinical Development at Medicenna. "These findings should be of great benefit both to those impacted by rGBM, but to the broader neurosurgical community as a whole."

Key Opinion Leader Call
Medicenna will host a Key Opinion Leader call and webcast for the investment community today at 1 PM EDT

To access the conference audio:
Local dial in: 416-764-8609
North American Toll Free: 1-888-390-0605
Conference ID No.: 65571448

To access the webcast and slide presentation:View Source

Following the event, the archived webcast and Medicenna presentation will be available on the Company’s website at www.medicenna.com. The webcast will be archived for 30 days after the event.

About the MDNA55-05 Clinical Trial
MDNA55-05 is a Phase 2b study of the safety and efficacy of MDNA55, an IL4R-directed toxin, in patients with de novo GBM at first or second relapse where the tumor is not amenable to surgical resection. In the study, investigators administer MDNA55 once directly into the brain tumor using a technique known as Convection Enhanced Delivery (CED). CED allows precision delivery of MDNA55 into the tumor and the surrounding healthy brain containing infiltrative tumor cells, while avoiding systemic exposure.

The primary endpoint of the study is median Overall Survival (mOS) comparing a null survival rate of 8.0 months (based on historical control) with an alternative pursue rate of 11.5 months (1-sided alpha = 0.10 and 80% power for 46 ITT subjects). The secondary endpoint is objective response rate (ORR) assessed by the modified Response Assessment in Neuro-Oncology (mRANO)-based criteria incorporating advanced imaging modalities according to a null response rate of 6% with alternative pursue rate of 18% (1-sided alpha = 0.10 and 80% power for at least 35 subjects evaluable for response).

On June 18, 2019 Prelude Corporation (PreludeDx), a leader in molecular diagnostics and personalized medicine for early stage breast cancer, reported its compelling data from research on stage 1 breast cancer patients presented at the 2019 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting in Chicago, IL (Press release, Prelude Therapeutics, JUN 18, 2019, View Source [SID1234537150]). The abstract titled "Risk stratification in early stage luminal breast cancer patients treated with and without RT" shows that PreludeDx’s newly-developed assay can stratify risk of local recurrence in luminal stage 1 breast cancer patients.

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The study data shared by Dr. Troy Bremer, Chief Scientific Officer of PreludeDx, builds on work presented in December 2018 at SABCS. Results of the study demonstrated that the new test was able to stratify patients where those in the low risk group had a 4% local 10-yr risk of either invasive breast cancer or DCIS with surgery alone and 3% risk of local recurrence with surgery and radiation therapy. Patients in the elevated risk group had a 15% local recurrence risk when treated with surgery alone but, after radiation therapy, had their local recurrence risk reduced to 3% at 10 years.

According to Rakesh Patel, MD, a leading breast cancer radiation oncologist and Medical Director of Breast Cancer Services at Good Samaritan Hospital in Los Gatos, CA, "These findings are important as the patient population (stage 1 luminal breast cancer patients over 50 years old) represents a large share of the patients that we see in clinic and we currently have very little data guiding us on which patients would truly benefit from conventional adjuvant radiation therapy or as importantly, if radiation can be more limited or deferred altogether. Specifically, randomized data have yet to identify patients that do not at least statistically benefit from treatment after a lumpectomy; however, we know that the degree of benefit may not be clinically significant in some patients. Likewise, there are traditionally ‘low risk’ patient populations that have an elevated risk of developing breast cancer and should be considered for additional treatment, such as radiation therapy, tamoxifen or even chemotherapy. The PreludeDx test can help us resolve these cases and in turn allow us to tailor treatments to the patients biologic risk profile addressing the concern of both over and under treatment."

"Our focus is on developing new technologies that improve the lives of patients with early breast cancer, and the data presented at ASCO (Free ASCO Whitepaper) is a great example of how we plan to continue to innovate in this space," said Daniel Forche, PreludeDx President and CEO. He continued by saying, "We believe it is critically important to offer new radiogenomic tools to radiation oncologists so that they can deliver precision medicine to their patients in an environment where they have an ever-increasing armament of therapies at their disposal. We look forward to results of future validations and ultimately getting this new radiogenomic technology into the hands of radiation oncologists."

About DCISionRT for Breast DCIS
DCISionRT is the only radiogenomic risk assessment test for patients with ductal carcinoma in situ (DCIS), which affects over 60,000 women in the US each year. The test was developed by PreludeDx and built on research that began with funding from the National Cancer Institute to better understand the biology of DCIS. DCISionRT assesses a woman’s individual tumor biology and other risk factors to provide a personalized Decision Score that identifies a woman’s risk as low or elevated. DCISionRT’s intelligent reporting provides a woman’s recurrence risk after breast conserving surgery alone and with the addition of radiation therapy, effectively allowing patients and their physicians to make more informed treatment decisions.

On June 18, 2019 Vanda Pharmaceuticals Inc. (Vanda) (Nasdaq: VNDA), reported that it will deliver a corporate presentation at the JMP Securities Life Sciences Conference in New York City on Wednesday, June 19, 2019 at 2:00 p.m. Eastern Time (Press release, Vanda Pharmaceuticals, JUN 18, 2019, View Source [SID1234537149]).

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The corporate presentation at the JMP Securities Life Sciences Conference will be available live on Vanda’s corporate website, where it also will be archived for 30 days. To access the presentation, go to Vanda’s website at www.vandapharma.com and click on the Presentations tab on the Investor Relations page. Please connect to the website several minutes prior to the start of the live presentation.

On June 18, 2019 EXUMA Biotechnology, a clinical-stage biotechnology company discovering and developing CAR-T products for solid tumors and affiliate of F1 Oncology, Inc., reported the expansion of its leadership team with the appointment of Wendy Li, M.D. as Chief Medical Officer and James Samuels as Chief Financial Officer (Press release, EXUMA Biotechnology, JUN 18, 2019, View Source [SID1234537147]).

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"It’s been a successful quarter for EXUMA with the positive announcement of interim results from two first-in-human solid tumor CAR-T products," said Gregory Frost, Ph.D., Chairman of EXUMA. "It is my pleasure to welcome Dr. Li and James to the team; their expertise and broad experience will play a valuable role as EXUMA continues on its trajectory."

Dr. Li has more than 20 years of medical and scientific leadership in oncology research and other therapeutic areas, as well as clinical practice experience. Dr. Li brings her experience in oncology development from Genentech, Sanofi, and Pfizer, as well as from her former position as Vice President of Clinical Development and CMO at Sihuan Pharma/Co-Xuanzhu Pharma, providing leadership on global early and late stage clinical studies, responding to FDA/CFDA and related agencies, and advising clinical, marketing, regulatory, and pharmacovigilance teams. Dr. Li received her M.D. from the Sun Yat-Sen University of Medical Studies in China.

"EXUMA Biotechnology has a meaningful pipeline of exciting clinical and preclinical cellular therapy candidates," said Dr. Li. "I look forward to bringing my clinical and research experience to the team and for the opportunity to develop life changing therapies for patients."

James Samuels has nearly 20 years of experience managing finance for both public and private companies. Most recently, he served as the CFO of Vetsource, a venture capital-backed provider of outsourced veterinary pharmacy services. Previously, he served as the CFO of Fusheng Industrial, a Taiwan based global manufacturer of industrial compressors. Additionally, he has held various CFO roles at Johnson & Johnson operating companies in Asia-Pacific in the areas of medical devices, diagnostics and pharmaceuticals. Prior to leaving Johnson & Johnson, Samuels served as CFO of Xian-Janssen, Johnson & Johnson’s pharmaceutical joint-venture in China. He holds an MBA from The Wharton School at the University of Pennsylvania.

"It’s a tremendous opportunity to be part of this talented team of individuals dedicated to finding treatments that will help patients across the world," said Samuels. "I look forward to helping EXUMA build on its success to date."