Personalis, Inc. to Present at IO Combinations 360˚

On October 1, 2020 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported the company’s participation at IO Combinations 360˚ which will be held online, October 1-2, 2020 (Press release, Personalis, OCT 1, 2020, View Source [SID1234567919]).

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The presentation, titled "Comprehensive immunogenomics to enable composite biomarkers for immunotherapy response using a sample sparing approach," will describe the Personalis universal cancer immunogenomics platform, ImmunoID NeXT. Travis Yates, PhD, will present for Personalis.

Dr. Yates will discuss the benefits of this unique, innovative design for immuno-oncology translational research, including mastering challenging samples, utilizing optimized algorithms, and obtaining accurate genomic data for identifying novel predictors of response. He will review a case study from a cohort of metastatic melanoma patients treated with an immune checkpoint blockade and will highlight the analytical capabilities provided by this immunogenomic profiling solution for understanding mechanisms of tumor evasion and creating composite biomarkers. Additionally, he will introduce NeXT Liquid Biopsy, an exome-wide liquid biopsy approach combined with ImmunoID NeXT, to further explore critical areas of tumor biology.

Codiak Initiates Patient Dosing in Phase 1/2 Clinical Trial of exoSTING™ for the Treatment of Solid Tumors

On October 1, 2020 Codiak BioSciences, Inc., a clinical-stage company focused on pioneering the development of exosome-based therapeutics as a new class of medicines, reported the initiation of patient dosing in its Phase 1/2 clinical trial of exoSTING (Press release, Codiak Biosciences, OCT 1, 2020, View Source [SID1234567918]). exoSTING is a novel exosome therapeutic candidate engineered with the company’s engEx Platform and designed to deliver Codiak’s proprietary STING (stimulator of interferon genes) agonist specifically to tumor-resident antigen presenting cells (APCs) to locally activate the innate immune response. The trial, which will study exoSTING in solid tumors, is Codiak’s second human clinical trial and the second clinical development program the Company has initiated in the past month.

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"We are enormously proud to now have both of our lead candidates in the clinic, the result of years of engineering and manufacturing innovation and a significant step forward towards fulfilling our goal of pioneering the development of engineered exosomes as a new class of medicines for diseases with high unmet medical needs," said Douglas E. Williams, Ph.D., CEO, Codiak. "With exoSTING, the data from our in vitro and in vivo preclinical studies support our desired product profile, demonstrating that we can achieve targeted engagement of the STING pathway to potentially overcome the lack of cell specificity, tolerability, and limited single-agent antitumor activity associated with previous STING agonists."

exoSTING is an exosome therapeutic candidate engineered with Codiak’s engEx Platform to incorporate its proprietary STING agonist inside the lumen of the exosome while expressing high levels of the exosomal protein, PTGFRN, on the surface. The high-level display of PTGFRN is designed to promote targeted delivery of Codiak’s proprietary STING agonist into APCs in the tumor microenvironment.

Engagement of the STING pathway has been validated to elicit an anti-tumoral response, yet therapeutic development has been generally limited by non-selective cell delivery, off-target toxicity to important immune cells in the tumor and dose-related toxicity due to leakage of the STING agonist into the circulation. In preclinical models of exoSTING, the targeted delivery of a STING agonist to tumor resident APCs promoted localized innate immune activation, T cell attraction and expansion in the tumor, and the development of systemic immunity not observed with a STING agonist delivered without exosomes (e.g., "free").

The Phase 1/2 dose escalation clinical trial of exoSTING is designed to investigate safety, tolerability, pharmacological activity, and objective tumor response in patients with advanced/metastatic, recurrent, injectable solid tumors, with a focus on tumors likely to be enriched in APCs. Examples of such tumors include metastatic head and neck squamous cell cancer (HNSCC), triple-negative breast cancer (TNBC), anaplastic thyroid carcinoma (ATC), and cutaneous squamous cell carcinoma (cSCC). Safety, biomarker and preliminary efficacy data from the dose-escalation phase of the trial is expected in mid-2021. As part of the Phase 2 portion of the trial, Codiak intends to enroll further expansion cohorts of patients at the optimal exoSTING dose to be identified in the Phase 1 portion of the clinical program.

About exoSTING

exoSTING is Codiak’s exosome therapeutic candidate engineered to incorporate a proprietary STING (stimulator of interferon genes) agonist inside the lumen of the exosome while expressing the exosomal protein, PTGFRN, on the exosome surface to facilitate specific uptake in tumor-resident antigen presenting cells (APCs). Codiak believes that exoSTING has the potential to overcome certain limitations of free STING agonists, and enhance the therapeutic index and selectivity of delivery to desired cells in the tumor microenvironment.

Codiak is developing exoSTING for the treatment of multiple solid tumors enriched in the target APCs. exoSTING has demonstrated encouraging activity in preclinical models and is now being evaluated in a Phase 1/2 clinical trial in patients with advanced/metastatic, recurrent, and injectable solid tumors. Future development of exoSTING may be expanded to neuro-oncology indications such as glioblastoma and leptomeningeal cancer disease.

About the engEx Platform

Codiak’s proprietary engEx Platform is designed to enable the development of engineered exosome therapeutics for a wide spectrum of diseases and to manufacture them reproducibly and at scale to pharmaceutical standards. By leveraging the inherent biology, function and tolerability profile of exosomes, Codiak is developing engEx exosomes designed to carry and protect potent drug molecules, provide selective delivery and elicit the desired pharmacology at the desired tissue and cellular sites. Through its engEx Platform, Codiak seeks to direct tropism and distribution by engineering exosomes to carry on their surface specific targeting drug moieties, such as proteins, antibodies/fragments, and peptides, individually or in combination. Codiak scientists have identified two exosomal proteins that serve as surface and luminal scaffolds. By engineering the exosome surface or lumen and optimizing the route of administration, Codiak aims to deliver engEx exosomes to the desired cell and tissue to more selectively engage the drug target, potentially enhancing the therapeutic index by improving potency and reducing toxicity.

Exicure Secures Debt Facility for Up to $25.0 Million

On October 1, 2020 Exicure, Inc. (Nasdaq: XCUR), a pioneer in gene regulatory and immunotherapeutic drugs utilizing spherical nucleic acid (SNA) constructs, reported it has entered into a $25.0 million senior secured term loan with MidCap Financial Trust (MidCap), as agent, and Silicon Valley Bank (SVB) (Press release, Exicure, OCT 1, 2020, View Source [SID1234567915]).

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The lenders under the debt facility have made available to Exicure an aggregate principal amount up to $25.0 million. Exicure has now received $17.5 million of proceeds and an additional $7.5 million can be drawn at Exicure’s discretion anytime between April 1, 2021 and September 30, 2021. The loan requires interest payments only for 24 months followed by 36 months of straight-line amortization after the interest only period.

"We believe this non-dilutive debt facility has enhanced Exicure’s financial flexibility," said David Giljohann, CEO of Exicure. "We are grateful to MidCap and SVB for the confidence they have shown in our preclinical and clinical programs in neurology and oncology, and plan to use these funds for the ongoing execution and expansion of our pipeline," concluded Dr. Giljohann.

Mustang Bio to Participate in Chardan’s Virtual 4th Annual Genetic Medicines Conference 2020

On October 1, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that Manuel Litchman, M.D., President and Chief Executive Officer, will participate in a fireside chat at Chardan’s Virtual 4th Annual Genetic Medicines Conference 2020 on Monday, October 5, 2020, at 1:00 p.m. EDT (Press release, Mustang Bio, OCT 1, 2020, View Source [SID1234567913]).

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A live webcast of the fireside chat will be available on the Events page of the Investor Relations section of Mustang’s website: www.mustangbio.com. An archived replay of the webcast will be available for approximately 30 days following the fireside chat.

Safety and efficacy to be evaluated in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm, acute myeloid leukemia and high-risk myelodysplastic syndrome

On October 1, 2020 Mustang Bio, Inc. ("Mustang") (NASDAQ: MBIO), a clinical-stage biopharmaceutical company focused on translating today’s medical breakthroughs in cell and gene therapies into potential cures for hematologic cancers, solid tumors and rare genetic diseases, reported that the first patient has been dosed in a Mustang-sponsored, open label, multicenter Phase 1/2 clinical trial to evaluate the safety and efficacy of MB-102 (CD123-targeted CAR T cell therapy) in patients with relapsed or refractory blastic plasmacytoid dendritic cell neoplasm (BPDCN), acute myeloid leukemia (AML) and high-risk myelodysplastic syndrome (hrMDS) (Press release, Mustang Bio, OCT 1, 2020, View Source [SID1234567910]). Study sites include City of Hope, where the CAR T cell therapy was initially developed and where the clinical data were generated to support Mustang’s current multicenter trial, Dana-Farber Cancer Institute, Duke University and MD Anderson Cancer Center.

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The Phase 1 portion of the trial will determine the maximum tolerated dose of MB-102 for the Phase 2 portion of the trial. Safety will be assessed at each dose level before proceeding to the next. The Phase 2 portion of the trial may be divided into as many as three arms to evaluate the efficacy of MB-102 in relapsed or refractory BPDCN (Arm 1), relapsed or refractory AML (Arm 2) and demethylation resistant hrMDS (Arm 3). The primary outcome that will be studied is the response rate at day 28 post infusion in all arms. Secondary outcome measures include duration of response, progression-free survival, overall survival and incidence of treatment-emergent adverse events, which will be followed for up to three years.

Manuel Litchman, M.D., President and Chief Executive Officer of Mustang, said, "This is a momentous occasion for Mustang, as it is the first clinical trial under Mustang’s investigational new drug application (IND) in which a patient was dosed with cells processed in our own manufacturing facility. We look forward to advancing the development of MB-102 and providing updates on the trial as we seek to help address the needs of patients suffering from the devastating diseases of BPDCN, AML and hrMDS."

Additional information about the trial can be found on www.clinicaltrials.gov using the identifier NCT04109482.

About MB-102 (CD123-targeted CAR T Cell Therapy)
MB-102 is a CAR T cell therapy that is produced by engineering patient T cells to recognize and eliminate CD123-expressing tumors. CD123 is widely expressed on bone marrow cells of patients with myelodysplastic syndromes, as well as in hematologic malignancies, including AML, and BPDCN.

In the first-in-human clinical trial at City of Hope (NCT02159495), MB-102 has demonstrated complete responses at low doses in AML and BPDCN without dose-limiting toxicities, as reported at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting in December 2017 and the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Special Conference on Tumor Immunology and Immunotherapy in November 2018. Dose escalation continues at City of Hope in both indications. MB-102 has received Orphan Drug Designations from the U.S. Food and Drug Administration for AML and BPDCN.