Five Prime Therapeutics to Present at the Guggenheim Healthcare Talks Idea Forum

On February 4, 2019 Five Prime Therapeutics, Inc. (NASDAQ:FPRX), a clinical-stage biotechnology company focused on discovering and developing innovative immuno-oncology protein therapeutics reported that Aron Knickerbocker, Chief Executive Officer, will present at the Guggenheim Healthcare Talks Idea Forum on Thursday, February 14, 2019 at 2:00 pm PT (Press release, Five Prime Therapeutics, FEB 4, 2019, View Source [SID1234533041]).

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The presentation will be webcast and may be accessed at the "Events & Presentations" section of the Company’s website at: View Source Five Prime will maintain an archived replay of the webcast on its website for 30 days after the conference.

Syntrix Wins $3.4M NIH Grant to Conduct Phase 1/2 Trial of SX-682 in Myelodysplastic Syndrome

On February 4, 2019 Syntrix Pharmaceuticals reported that it has been awarded a three-year grant worth $3.4 million from the National Heart Lung and Blood Institute of the National Institutes of Health to assess its investigational CXCR1/2 inhibitor SX-682 in patients with low- and high-risk myelodysplastic syndrome (MDS) who had progression or were intolerant to prior therapy (Press release, Syntrix, FEB 4, 2019, View Source [SID1234553882]). The phase 1/2 clinical trial will be carried out in collaboration with researchers at the Moffitt Cancer Center led by Dr. Rami Kamrokji.

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"Patients with low-risk MDS have an expected median survival measured in years, but suffer from hematologic deficits and related symptoms that lead to transfusion dependence," said Syntrix’s President John Zebala. "Patients with high-risk MDS have debilitating cytopenias and borderline leukemia, and can have expected survival measured in months."

Only three drugs have received regulatory approval for MDS treatment, all with suboptimal response rates (<50%) and of limited durability (1-2 years). Once these agents fail in patients, there is no second-line treatment. Prognosis after failure is dismal, with median survival estimated at <6 months for higher-risk patients, and <18 months for lower-risk patients.

The Phase 1/2 clinical trial of SX-682 builds on groundbreaking discoveries by investigators at the Moffitt Cancer Center and the Albert Einstein College of Medicine who showed CXCR1/2 is pivotal in MDS and that its inhibition is a therapeutic strategy against the disease.

This Phase 1/2 trial in MDS patients will test the hypothesis that targeting CXCR1/2 with SX-682 will be efficacious in the disease by eliminating the MDS stem cells and bone marrow MDSCs. The FDA approved the protocol for the study in an IND sponsored by Syntrix.

ABOUT SX-682: SX-682 is a clinical-stage oral allosteric small-molecule inhibitor of CXCR1 and CXCR2 (CXCR1/2). Inhibiting both human receptors is believed essential. CXCR1/2 are a combined "master switch" of the immunosuppressive tumor microenvironment. Clinical studies in melanoma, breast, ovarian, prostate and colon cancer have shown a direct correlation between serum levels of CXCR1/2 ligands and disease progression. SX-682 has been validated in all major solid tumor models, where it exhibits mono-agent anti-tumor activity, blocks metastasis, depletes immunosuppressive myeloid cells, activates tumor killing by effector cells, reverses chemo-resistance, and potently synergizes with anti-CTLA-4 and anti-PD1. SX-682 is also being evaluated in solid tumors supported by the National Cancer Institute.

Torque and Thermo Fisher Scientific Announce Collaboration to Build State-of-the-Art Facility to Manufacture Torque’s Deep-Primed T Cell Immunotherapies

On February 4, 2019 Torque, an immuno-oncology company developing first-in-class Deep Primed T Cell Therapeutics to direct immune power deep within the tumor microenvironment, and Thermo Fisher Scientific Inc. (NYSE: TMO), reported a collaboration to build a dedicated Slipstream manufacturing facility for high-efficiency production of Torque’s Deep-Primed T cell immunotherapies (Press release, Torque Therapeutics, FEB 4, 2019, View Source [SID1234553883]).

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Buildout of the Torque/Thermo Fisher manufacturing facility has begun, and the companies anticipate processing patient cells by the end of 2019. The Slipstream platform will initially be used in the clinical development of Torque’s lead Deep-Primed T cell candidate, TRQ-1501, in solid and hematologic tumors; followed by TRQ-1201, also for solid and hematologic tumors.

"Torque’s cell manufacturing technology platform is critical to achieving our goal of developing and commercializing a new class of cellular immunotherapy for cancer patients with both early and advanced disease, particularly for the large population of solid tumor patients in need of new treatments," said Bart Henderson, CEO of Torque. "We are very excited about partnering with Thermo Fisher to build this state-of-the-art manufacturing facility because of their vision and exceptional track record in building high-efficiency, globally integrated operations. This is a pioneering manufacturing partnership with the goal of dramatically improving the efficiency and scalability of producing best-in-class cellular immunotherapy products that have the potential to benefit a significant number of cancer patients."

"Thermo Fisher is dedicated to advancing precision medicine, and our collaboration with Torque is a tangible example of the progress we are making to become a recognized leader in cell therapy manufacturing," said Michel Lagarde, President of Pharma Services for Thermo Fisher Scientific. "We are pleased to partner with Torque to build and launch this unique and advanced manufacturing facility utilizing the Slipstream technology, Torque’s innovative cellular immunotherapy product, which has the potential to transform cancer treatment and patients’ lives."

The Slipstream platform technology situated in Princeton is a revolutionary design that uses a fully closed, semi-automated system that surpasses conventional cell therapy manufacturing techniques. The Slipstream process leverages advanced logistics that enable a substantially smaller manufacturing footprint that is less capital- and labor-intensive. Production capacity can be expanded modularly by adding arrays in Lego-like fashion.

About Slipstream Cell Therapy Manufacturing Technology
Slipstream is a proprietary, high-efficiency T cell manufacturing platform engineered by Torque. Currently marketed immune cell therapies are produced using open, complex, labor- and cost-intensive processes that require a substantial manufacturing facility. In contrast, Slipstream production is semi-automated and fully closed, which eliminates contamination risk between transfers and can dramatically reduce staffing requirements and the factory footprint. The cell engineering process uses a modular design that enables both large-scale and decentralized manufacturing. Slipstream has the potential to improve manufacturing efficiency beyond what is possible with currently used cell therapy manufacturing processes and to move cell therapy production closer to the point of care.

About Torque’s Deep-Primed Immune Cell Therapy Platform
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.

BioLineRx Receives Orphan Drug Designation from the FDA for its Lead Therapeutic Candidate BL-8040 for the Treatment of Pancreatic Cancer

On February 4, 2019 BioLineRx Ltd. (NASDAQ/TASE:BLRX), a clinical-stage biopharmaceutical company focused on oncology, reported that the US Food and Drug Administration (FDA) has granted Orphan Drug Designation to its lead oncology candidate, BL-8040, for the treatment of pancreatic cancer (Press release, BioLineRx, FEB 4, 2019, View Source [SID1234533026]).

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"Orphan Drug Designation in pancreatic cancer is a very important milestone in the development plan of BL-8040, and joins previously approved orphan designations by the FDA for BL-8040 in AML and stem-cell mobilization," stated Philip Serlin, Chief Executive Officer of BioLineRx. "Despite advances in the treatment of various cancers with immune checkpoint inhibitors, pancreatic cancer is refractory to these treatment options, and remains an area of significant unmet medical need. We have previously reported encouraging clinical data supporting the potential of BL-8040 as part of an immunotherapy combination treatment in pancreatic cancer, and we look forward to top-line results from our ongoing pancreatic clinical studies later this year."

BL-8040 is currently being investigated in clinical studies for the treatment of pancreatic cancer under two separate immuno-oncology collaborations – one with Merck & Co., Inc., Kenilworth, N.J., USA (known as MSD outside the United States and Canada), and a second collaboration with Genentech, a member of the Roche Group.

Orphan Drug Designation by the FDA entitles BioLineRx to seven years of market exclusivity for the use of BL-8040 for the treatment of pancreatic cancer, if approved, plus significant development incentives, including tax credits related to clinical trial expenses, an exemption from the FDA-user fee, and FDA assistance in clinical trial design.

About BL-8040
BL-8040 is a short synthetic peptide for the treatment of hematological malignancies, solid tumors, and stem cell mobilization. It functions as a high-affinity best-in-class antagonist for CXCR4, a chemokine receptor that is directly involved in tumor progression, angiogenesis, metastasis and cell survival. CXCR4 is over-expressed in many human cancers and its expression often correlates with disease severity. In a number of clinical and pre-clinical studies, BL-8040 has shown robust mobilization of cancer cells and immune-cells, sensitization of cancer cells to chemo- and bio-based anti-cancer therapies, and direct anti-cancer effect by inducing programmed cell death (apoptosis). BL-8040 was licensed by BioLineRx from Biokine Therapeutics and was previously developed under the name BKT-140.

About Pancreatic Cancer
Pancreatic cancer has a low rate of early diagnosis and a poor prognosis. Its incidence rate in the US is estimated at 3.2% of new cancer cases. Each year, about 185,000 individuals globally are diagnosed with this condition, and an estimated 55,000 individuals were diagnosed with pancreatic cancer in the US during 2018. Symptoms are usually non-specific and as a result, pancreatic cancer is often not diagnosed until it reaches an advanced stage. Surgical resection does not offer adequate treatment since only 20% of patients have resectable tumors at the time of diagnosis. Even among patients who undergo resection for pancreatic cancer and have tumor-free margins, the five-year survival rate is only 10%-25%. The overall five-year survival rate among pancreatic cancer patients is 7-8%, which constitutes the highest mortality rate among solid tumor malignancies. The overall median survival is less than one year from diagnosis, highlighting the need for the development of new therapeutic options.

Despite advances in chemotherapeutics and immunotherapy, increases in median and overall survival rates in pancreatic cancer have been modest. Pancreatic cancer remains an area of unmet medical need, with no new approved therapies since the approval of nab-paclitaxel in combination with gemcitabine (Abraxane) for first-line treatment in 2013 and Onivyde in combination with fluorouracil and leucovorin for second-line treatment in 2015. The limited clinical benefits demonstrated by these existing standard treatment options reinforce the need for additional approaches.

Onconova Therapeutics, Inc. to Present Corporate Update at the BIO CEO & Investor Conference in New York City

On February 4, 2019 Onconova Therapeutics, Inc. (NASDAQ: ONTX), a Phase 3-stage biopharmaceutical company focused on discovering and developing novel products to treat cancer, with a primary focus on myelodysplastic syndromes (MDS), reported that Dr. Steven M. Fruchtman, President and Chief Executive Officer, will present a company update at the BIO CEO & Investor Conference February 11-12, 2019 (Press release, Onconova, FEB 4, 2019, https://investor.onconova.com/news-releases/news-release-details/onconova-therapeutics-inc-present-corporate-update-bio-ceo [SID1234533027]). Dr. Fruchtman and members of management will be available for 1×1 meetings during the conference.

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Presentation details: 1:15 – 1:30p EST Monday, February 11th
Gramercy, 7th Floor
Marriott Marquis 1535 Broadway, New York, NY 10036
A webcast of the presentation will be available one hour after the conclusion of the live event at http://www.veracast.com/webcasts/bio/ceoinvestor2019/71117204463.cfm and can also be accessed via View Source on the Company’s website after the event.