On September 9, 2019 Biodesix, Inc. reported positive clinical data from Oncimmune’s EarlyCDT Lung blood test, which harnesses the power of the immune system to detect evidence of the body’s natural response to cancer (Press release, Biodesix, SEP 9, 2019, View Source [SID1234539380]). The technology can detect cancer on average four years or more before standard clinical diagnosis . Results from the randomized controlled study of 12,209 people at high risk of developing cancer, Early detection of Cancer of the Lung Scotland (ECLS), were presented today at the International Association for the Study of Lung Cancer’s (IASLC) 2019 World Conference on Lung Cancer in Barcelona. In June, Biodesix announced acquisition of Oncimmune and the EarlyCDT Lung test in the United States.

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Prof. Frank Sullivan, Professor of Primary Care Medicine at the University of St. Andrews and Chief Investigator for the ECLS trial, noted the global significance of a blood test, followed by CT scans, to increase the number of patients diagnosed at an earlier stage of disease, when surgery is still possible and prospects for survival are higher. This early diagnosis is especially important in the case of lung cancer because approximately 85 percent of patients are diagnosed with the disease at a late stage, when the cancer has spread to other parts of the body and the chances of survival are low.

Biodesix CEO David Brunel

"This represents a significant opportunity to improve the standard of care in screening for lung cancer. Current screening programs in the United States have seen very slow adoption rates, with only two percent of the eligible eight million U.S. high risk individuals undergoing screening in 2016. The EarlyCDT Lung test is a blood test that can be administered in primary care and used to direct at-risk patients into further investigation with imaging, thereby increasing the proportion of lung cancers diagnosed in early stages. We look forward to continuing our work with Oncimmune to extend the utility of the EarlyCDT Lung test in the U.S. to include lung cancer screening."

In addition to its potential in lung cancer screening, the EarlyCDT Lung test also helps physicians to determine if a pulmonary nodule is potentially cancerous, helping physicians manage nodule patients. The Biodesix Nodify XL2 test, is used to help physicians rule-out malignancy in low-to-moderate risk incidental lung nodules. Biodesix will offer complementary tests that help empower physicians to stratify patients into distinct nodule management pathways.

On September 9, 2019 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for cancer, reported that it has entered into a research agreement with Invectys, a biopharmaceutical company focused on the development of innovative immunotherapy approaches to treat cancers (Press release, Personalis, SEP 9, 2019, View Source [SID1234539379]). Under the terms of the agreement, Personalis will provide comprehensive tumor immunogenomics capabilities to facilitate biomarker discovery in chronic lymphocytic leukemia (CLL) patients enrolled in a Phase II clinical trial assessing the efficacy of INVAC-1, a DNA plasmid encoding a modified human telomerase reverse transcriptase (hTERT) protein, as monotherapy or in combination with ibrutinib.

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"Personalis is known for their innovative and expansive cancer immunogenomics expertise, and we’re delighted to partner with them on this project to evaluate biomarker signatures in responders and non-responders to our leading cancer vaccine candidate, INVAC-1," said Pierre Langlade Demoyen, CEO of Invectys. "INVAC-1 has the potential to become a key component of combination therapy approaches for the treatment of various solid and hematologic cancer types. Our work with Personalis will help us to determine which patients are most likely to respond favorably to these novel treatment regimens."

Invectys is now enrolling CLL patients in this Phase II, open-label, single-arm trial consisting of two patient groups: 45 patients receiving INVAC-1 as monotherapy and an additional 45 patients receiving INVAC-1 in combination with ibrutinib, a Bruton’s Tyrosine Kinase inhibitor. The primary goal of the study is to achieve minimal residual disease negativity in both groups. The trial is being conducted at the MD Anderson Cancer Center in Houston, Texas. For more information, please visit clinicaltrials.gov identifier NCT03265717.

"Invectys has built an exciting pipeline of novel, next-generation immunotherapies, and we’re thrilled to help drive their lead program forward," said John West, CEO of Personalis. "Having already established ourselves as a leader in comprehensive tumor immunogenomic profiling and biomarker discovery for solid tumor indications, we’ve seen increasing use of our capabilities for biomarker evaluation in hematologic cancer types. This bodes well as we continue to rollout and onboard customers for our newly-launched, universal cancer immunogenomics platform, ImmunoID NeXT."

On September 9, 2019 Aptorum Group Limited (Nasdaq: APM) ("Aptorum Group"), a biopharmaceutical company focused on the development of novel therapeutics to address global unmet medical needs, reported the development of two preclinical drug candidates which target obesity and neuroblastoma, respectively (Press release, Aptorum, SEP 9, 2019, View Source [SID1234539378]).

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About CLS-1: Treatment of obesity via modulation of chemical signaling relating to gut microbiota

Under the recently-announced microbiota modulation platform operated by Aptorum Group’s wholly-owned subsidiary Claves Life Sciences Limited, we have commenced the preclinical development of macromolecule candidate CLS-1 targeting the treatment of obesity. CLS-1 is undergoing lead optimization and is expected to progress into the IND enabling stage in 2020.

The prevalence of obesity continues to escalate globally; however, there is no current optimal therapy for this condition1. For the majority of obese patients, conventional medical therapies (i.e., diet, exercise, behavioral counseling) often have a high failure rate for the long term2. We believe current pharmacotherapy has limited efficacy and is associated with substantial safety issues, and this will provide immeasurable market opportunity for CLS-1.

Chemical signaling of gut microbiota is known to be one of the major causes of obesity1. CLS-1 is an orally administered non-absorbable macromolecule that modulates the metabolite excreted by gut microbiota with high affinity and specificity. In this way, we believe the absorption of this particular metabolite, which is linked to obesity, can be inhibited.

Aptorum Group is also pursuing two further indications based on the modulation of microbiota based chemical signaling involving the above large molecule technology, which we believe to be highly scalable and we hope to be making further announcements regarding our efforts in due course.

About SACT-1: Repurposed Drug Candidates for the Treatment of Neuroblastoma

Under the recently announced Smart-ACTTM computational drug discovery platform operated by our wholly-owned subsidiary Smart Pharma Group, Aptorum Group has completed computational screening of approximately 1,615 marketed drugs against 3 therapeutic target proteins to potentially tackle poor prognosis of neuroblastoma, i.e., a rare type of children’s cancer that forms in certain types of nerve tissue and most frequently in the adrenal glands as well as spine, chest, abdomen or neck, especially in children3. For the high-risk group, the 5-year survival rate of this condition is around 40-50% as observed by the American Cancer Society.4 Aptorum Group has identified an array of repurposed candidates and has proceeded to evaluate them in cell-based and animal models in order to validate the candidates’ usage for such new indication and potential efficacies.

Aptorum Group is also pursuing two further indications under the Smart-ACTTM drug discovery platform and hopes to be making further announcements regarding our research in due course.

On September 9, 2019 Mateon Therapeutics Inc. (OTCQB:MATN), reported that it has executed agreements with WideTrial Inc. for an Expanded Access program (EAP) in pancreatic cancer (Press release, Mateon Therapeutics, SEP 9, 2019, View Source [SID1234539377]). Using WideTrial’s novel platform, the investigational therapeutic OT-101 will be made available for elective treatment-use to patients who do not meet the inclusion criteria of OT-101 research trials. The partnership may also support Expanded Access for OT-101 in additional patients with advanced solid tumors, excluding brain cancers (AA, GBM, and other gliomas). Separately, the companies executed data access agreements to support the development of Mateon’s AI/Blockchain technologies with WideTrial’s data assets in cancer and other therapeutic areas.

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OT-101 is a first-in-class RNA therapeutic targeting TGF beta and is the lead immune-oncology drug candidate of Oncotelic, a wholly owned subsidiary of Mateon Therapeutics Inc.. The investigational drug exhibited single-agent activity in relapsed/refractory cancer patients during multiple Phase 2 clinical trials.

Like research trials, cohort Expanded Access programs (EAPs) are centrally monitored trials that enroll patients at designated sites under a single protocol. They differ from research trials in that their primary objective is to provide the option of treatment-use to patients who cannot take part in the product’s research trials.

"This Expanded Access program will allow us to meet the spirit of clinical trial inclusivity, as described in the 2017 FDA Reauthorization Act. We are pleased to have found a viable way to accommodate greater numbers of patients in need and the doctors who seek to treat them," said Dr. Vuong Trieu, Chairman and CEO of Mateon Therapeutics Inc.. "Though the primary objective is treatment, we intend to utilize generated data to improve the power and targeting of future research trials."

"The PointR platform applies cutting edge algorithms to clinical datasets and could be used to quickly and cost-effectively identify the best therapy tailored fit to a single individual. The ability to access EAP data is helpful to the development of this platform" said Saran Saund, CEO of PointR Data, which is becoming a wholly owned subsidiary of Mateon Therapeutics Inc.. "We are thrilled at the opportunity of vertically integrating artificial intelligence and drug development capabilities under one roof to quickly identify promising new therapeutic opportunities for various diseases and to deliver compelling business value."

"We built WideTrial to solve the economic and operational barriers that drug developers face with Expanded Access," said Jess Rabourn, CEO of WideTrial. "The new platform enables larger numbers of gravely ill patients to choose to try investigational medicines under a properly designed protocol. Although treatment is the primary objective, EAP sponsors can learn a lot from patients’ experiences in these programs."

On September 9, 2019 Phio Pharmaceuticals Corp. (NASDAQ: PHIO), a biotechnology company developing the next generation of immuno-oncology therapeutics based on its proprietary self-delivering RNAi (sd-rxRNA) therapeutic platform, reported that it has entered into a research collaboration with Carisma Therapeutics to evaluate the potential of Phio’s self-delivering RNAi compounds to synergistically modify Carisma’s chimeric antigen receptor macrophages (CAR-M) (Press release, Phio Pharmaceuticals, SEP 9, 2019, View Source [SID1234539376]). Silencing of key genes using sd-rxRNA compounds may enhance the immune function of these cells as a novel adoptive cell therapy for use in cancer treatment.

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"Overcoming local tumor immunosuppression remains a significant challenge in the treatment of cancer," said Dr. John A. Barrett, Chief Development Officer of Phio Pharmaceuticals. "We have established collaborations with several companies and academic institutions to validate our technology’s ability to improve the activity of T cells, TILS, NK cells and dendritic cells towards tumors. Macrophages are sentinel cells of the innate immune system that robustly accumulate in most solid tumors. Combining Phio’s gene silencing technology with Carisma’s CAR-M technology has the potential to enhance aspects of macrophage anti-tumor activity and may further tip the scale in favor of an immunostimulatory phenotype and enhance tumor cell killing. We believe we can leverage both technologies to turn cold tumors hot and ultimately improve outcomes for patients."

Dr. Gerrit Dispersyn, Phio Pharmaceuticals’ President and CEO stated "We are very excited to work with the Carisma Therapeutics team, which represents our first collaboration addressing macrophages utility as an immunotherapeutic. By combining Carisma’s unique approach to adoptive cell therapy using CAR macrophages with our self-delivering RNAi technology, we expect to demonstrate potential synergies between our platforms that could enable us to bring new and improved forms of adoptive cell therapy to patients in the future. Our overall strategy in this setting is to generate robust data that validate our platform for the weaponization of a broad range of immune cells against tumors in order to generate future partnerships and build shareholder value."

"Metastatic solid tumors have been an intractable challenge for engineered cell therapies. Our platform technology, based on the genetically engineered macrophage, has a unique opportunity to provide benefit in this disease state," said Dr. Michael Klichinsky, co-Founder and Vice President of Discovery at Carisma. "We are excited to work with Phio to evaluate sd-rxRNA mediated silencing of genes that have the potential to enhance macrophage immunotherapy."