On April 19, 2021 Veracyte, Inc. (Nasdaq: VCYT) reported that new data demonstrating the clinical capability of the Percepta Genomic Atlas for informing lung cancer treatment decisions, as well as the technical feasibility of enabling the company’s Envisia Genomic Classifier on the nCounter Analysis System, will be presented at the American Thoracic Society (ATS) 2021 International Conference (Press release, Veracyte, APR 19, 2021, View Source [SID1234578201]). Multiple posters reinforcing the Envisia classifier’s diagnostic performance will also be presented at the meeting, which will be held virtually May 14-19, 2021.

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"We’re also very excited about the new data demonstrating that we can successfully enable the Envisia classifier on the nCounter system, maintaining the test’s clinical performance while enabling access for patients globally through local labs."

"The data being presented at ATS suggest that the Percepta Genomic Atlas can provide information that will accelerate appropriate treatment for patients following a lung cancer diagnosis and, importantly, that the test provides this information regardless of disease stage," said Bonnie Anderson, Veracyte’s chairman and chief executive officer. "We’re also very excited about the new data demonstrating that we can successfully enable the Envisia classifier on the nCounter system, maintaining the test’s clinical performance while enabling access for patients globally through local labs."

The Percepta Genomic Atlas provides comprehensive genomic profiling information on cancerous lung nodules, utilizing small samples from the same biopsy used for diagnosis. Data to be shared at ATS show that the in-development test accurately detects known gene variants in lung cancer using bronchoscopy samples, potentially enabling earlier and more appropriate treatment with currently available and in-development targeted therapies.

The Envisia Genomic Classifier is a genomic test that detects a genomic pattern of usual interstitial pneumonia (UIP) to improve interstitial lung disease (ILD) diagnostic and prognostic confidence. The classifier is performed in Veracyte’s U.S.-based CLIA laboratory. In December 2019, Veracyte acquired the exclusive global diagnostics rights to the nCounter Analysis System, a CE-marked and FDA-cleared decentralized molecular testing platform. The company plans to make the Envisia classifier available on the nCounter platform in international markets by the end of this year.

Following are details of the Percepta Genomic Atlas and Envisia classifier posters accepted for presentation at ATS. These posters will be available to meeting registrants on demand beginning May 14 through July 2, 2021:

Title:

Identification of Driver Mutations in Transbronchial Needle Aspirates of Suspicious Lung Nodules Concurrent with Diagnostic Bronchoscopy, Abstract #A4825

First Author:

Joshua Babiarz, Ph.D., Veracyte

Title:

Envisia Genomic Classifier Demonstrates Consistent Performance Across Gender, Age Group, and Smoking Status. Abstract #A1839

First Author:

Luca Richeldi, M.D., Ph.D., Università Cattolica del Sacro Cuore, Rome, Italy

Title:

Envisia Genomic Classifier Helps Improve Multidisciplinary Diagnoses of Complex Interstitial Lung Diseases, Abstract #A1877

First Author:

Lisa H. Lancaster, M.D., Vanderbilt University Medical Center

Title:

Cryobiopsy and Genomic Classifier (Envisia) in the Diagnosis of Usual Interstitial Pneumonia, Abstract #A4236

First Author:

R. Ronaghi, M.D., University of California, Los Angeles

Title:

Role of the Envisia Genomic Classifier in Establishing a Diagnosis of Idiopathic Pulmonary Fibrosis, Abstract #1837

First Author:

M. Abdalla, M.D., Pulmonary and Critical Care Medicine, Medical College of Wisconsin

Title:

Bridging the Envisia Genomic Classifier to the nCounter Platform: A Proof-of-Concept Study, Abstract #A4352

First Author:

Huimin Jiang, Ph.D., Veracyte

On April 19, 2021 Humanigen, Inc. (Nasdaq: HGEN) ("Humanigen"), a clinical-stage biopharmaceutical company focused on preventing and treating an immune hyper-response called ‘cytokine storm’ with its lead drug candidate, lenzilumab, reported positive data from the Phase 1b portion of ZUMA-19 evaluating the efficacy and safety of lenzilumab in patients treated with CAR-T in diffuse large B-cell lymphoma (DLBCL) (Press release, Humanigen, APR 19, 2021, View Source [SID1234578200]). At the recommended Phase 2 dose of lenzilumab, the ORR was 100% and no patient experienced severe cytokine release syndrome (CRS) or severe neurotoxicity (NT).

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ZUMA-19 was a clinical study designed to evaluate the efficacy and safety of lenzilumab and CAR-T (axicabtagene ciloleucel, Axi-Cel) in patients with relapsed or refractory DLBCL.

This study was a standard 3+3 design with three patients administered 600 mg lenzilumab (cohort 1) and three patients administered 1,800 mg lenzilumab (cohort 2) just prior to CAR-T. The recommended Phase 2 dose was determined to be 1,800 mg.

In the six study patients, the ORR was 83% (n=5) which included four complete responses (CR). In cohort 1, there was no severe CRS (≥ grade 3). One patient experienced grade 3 NT with a two-day duration. At the recommended Phase 2 dose (cohort 2), ORR was 100% (n=3) and the toxicity-free CR (CRS and NT < grade 2) was 66% (n = 2). There was no severe CRS or severe NT at the recommended Phase 2 dose. There were no adverse events attributed to lenzilumab across the study.

Inflammatory markers were correlated with reduced rates of CRS and NT. Lenzilumab dose-dependently reduced myeloid cytokines IL-6, IL-8, MCP-1, and IP-10 (CXCL-10) and systemic inflammatory markers CRP, ferritin, and SAA.

"These encouraging results from ZUMA-19 provide further proof of concept that lenzilumab may break the linkage between efficacy and toxicity (CRS and NT) widely-associated with CAR-T, and may improve durability of response," said Dale Chappell, MD, MBA, Chief Scientific Officer, Humanigen. "We believe these data warrant a larger study involving multiple CAR-T therapies."

Humanigen will initiate a randomized, multicenter, potentially registrational, Phase 2 study to evaluate the efficacy and safety of lenzilumab combined with all commercially available CD19 CAR-T therapies in DLBCL. The study plans to enroll approximately 150 patients and the protocol is being submitted to FDA.

Humanigen has terminated the clinical collaboration agreement with Kite related to ZUMA-19 and both parties will collaborate to wind down current study activities.

"Humanigen is pleased to be in a position to proactively develop lenzilumab across the CAR-T landscape and further expand its pipeline," said Cameron Durrant, MD, MBA, Chief Executive Officer, Humanigen. "We thank Kite for their sponsorship and contribution that has allowed Humanigen to progress to this exciting point."

On April 19, 2021 Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM), a clinical stage biopharmaceutical company dedicated to meeting unmet medical needs in oncology and infectious diseases, reported financial results for the fiscal year ended December 31, 2020, and provided an update on clinical and corporate developments (Press release, Aptorum, APR 19, 2021, View Source [SID1234578199]).

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Mr. Darren Lui, President and Executive Director of Aptorum Group Limited, commented "Aptorum’s operational plans remain on track. In particular, one of our lead programs, ALS-4 (targeting infections caused by Staphylococcus aureus including Methicillin-resistant Staphylococcus aureus (MRSA)) has already commenced dosing of the first cohort of human subjects in its Phase I clinical trial as announced in March 2021. With the additional capital raised in October 2020, the Company is also on track to commence validation of our novel liquid biopsy based rapid pathogen identification and detection diagnostics ("RPIDD"). Our other lead program SACT-1, which is targeting an orphan indication for the treatment of neuroblastoma, is also on track in its preparation to open an IND with US FDA for the commencement of clinical trials in 2021. The Company is also focused on the distribution of a non-hormonal based Dioscorea opposita bioactive nutraceutical supplement targeting woman’s health during menopausal or post-menopausal cycles in 2021 in major markets including UK, Europe and Asia initially. In light of the global coronavirus situation, the Company would like to report that its day-to-day operations continue as normal.

Clinical Pipeline Update and Upcoming Milestones

In March 2021, Aptorum Group announced dosing of our first human subject in its Phase I clinical trial for one of our lead programs, ALS-4, an orally administered small molecule drug for the treatment of infections caused by Staphylococcus aureus including MRSA. The Phase I clinical trial is currently conducted in Canada and will target to recruit up to 48 and 24 healthy volunteers for the single-ascending dose (SAD) and multiple-ascending dose (MAD) cohorts, respectively.

In February 2021, Aptorum Group completed a Pre-IND meeting with US FDA with encouraging outcomes regarding SACT-1, another lead program employing an orally administered small molecule repurposed drug for the treatment of neuroblastoma. The Company is now on track in its preparation to open an IND with US FDA for the commencement of clinical trials in 2021.

In September 2020, Aptorum Group initiated an additional R&D program. Our RPIDD technology enables rapid and accurate identification and detection of existing or emerging unknown pathogens (including DNA/RNA-based viruses such as coronavirus, antibiotic-resistant bacteria, fungi, etc.), in a cost-effective, unbiased and broad-spectrum manner, by employing liquid biopsy (patients’ blood samples and is potentially adaptable for other sample types) in conjunction with New Generation Sequencing (NGS) and Artificial Intelligence (AI) driven software analytics. The Company is now on track to commence validation of the RPIDD program in further human blood samples to support its eventual commercialization.

Aptorum Group is commencing the commercialization and distribution of its non-hormonal based Dioscorea oppposita bioactive nutraceutical supplement ("NativusWell") in 2021, targeting woman’s health during menopausal or post-menopausal cycles. The product is currently being manufactured in Canada.

Corporate Highlights

On July 24, 2020, our Class A Ordinary Shares began to trade on the Professional Compartment of the regulated market of Euronext Paris under the symbol "APM" and are denominated in Euros on Euronext Paris.

On September 25, 2020, Aptorum Innovations Holding Pte. Limited ("AIHPL"), one of the Company’s wholly-owned subsidiaries, entered into an Exclusive License Agreement with Accelerate Technologies Pte Ltd. ("Accelerate Technologies"), commercialization arm of the Singapore’s Agency for Science, Technology and Research, to co-develop novel molecular-based RPIDD technology.

In October 2020, Aptorum Group completed a capital raise of approximately $9 million by a public offering of 2,769,231 Class A ordinary shares and warrants to purchase 2,769,231 of its Class A ordinary shares.

On December 30, 2020, AIHPL entered into an Evaluation Agreement with Illumina Inc ("Illumina") to evaluate the data and performance of Illumina’s sequencing technology based on the workflow of AIHPL’s RPIDD technology, at AIHPL’s Singapore based evaluation site.

Full year 2020 research and development expenses of $11.6 million, an increase of $4.6 million over the full year 2019.

Fiscal Year End Financial Results

Aptorum Group reported a net income of $4.9 million in 2020, as compared to net loss of $20.1 million in 2019. The net income in 2020 was mainly driven by the gain on investments in marketable securities, net of $25.2 million.

Research and development expenses were $11.6 million in 2020 as compared to $6.9 million in 2019. The increase in research and development expenses in 2020 was primarily due to the increase in services provided by our consultants, advisors and contracted research organizations as a result of the progress of our projects’ development.

General and administrative fees were $4.9 million in 2020 as compared to $7.4 million in 2019. The decrease in general and administration fees was primarily due to a decrease in compensation expenses and business expenses as well as general travel restrictions due to the COVID 19 outbreak.

Legal and professional fees were $2.9 million in 2020 as compared to $3.4 million in 2019. The decrease in legal and professional fees was primarily due to the reduction of professional services required during 2020.

Aptorum Group reported $3.6 million of cash and restricted cash as of December 31, 2020 compared to $5.3 million as of December 31, 2019. The decrease in cash and restricted cash was mainly the result of the cash used in operating activities of $15.8 million during the year and net repayment of loan from related parties of $4.4 million, partly offset by the net proceeds from issuance of Class A Ordinary Shares and warrants of $16.8 million in 2020 and proceeds from disposal of fixed assets and investment in marketable securities of $1.0 million and $0.9 million, respectively, in 2020.

In addition, Aptorum Group had $28.4 million marketable securities and $13.0 million undrawn line of credit as of December 31, 2020, compared to $1.1 million marketable securities and $8.7 million undrawn line of credit as of December 31, 2019. The increase in marketable securities was due to the public listing and subsequent stock price appreciation of one of our pre-IPO investments.

On April 19, 2021 NorthShore University HealthSystem (NorthShore) and Sema4, a patient-centered health intelligence company, reported the availability of a system-wide data-driven genomics program that utilizes predictive analytics to help clinicians and patients prevent, detect, and treat diseases at an early stage, when they are most treatable (Press release, NorthShore University HealthSystem, APR 19, 2021, View Source [SID1234578198]). As part of the program, NorthShore clinicians and patients will have access to Sema4’s industry-leading, information-rich genomic solutions for hereditary cancer, cardiovascular diseases, pharmacogenomics, and rare diseases.

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"Unlocking the power of genomics in patient care requires us to make information available in a way that clinicians and patients can easily act upon over time," said Peter Hulick, MD, director of the Mark R. Neaman Center for Personalized Medicine at NorthShore. "The partnership between NorthShore and Sema4 will allow us to get even better at delivering on the promise of genomic insights to prevent, detect, and manage diseases through one of the largest and most comprehensive personalized medicine programs in the country."

Advanced Clinical Offerings

By combining clinical information with genomic analysis, Sema4’s technology platform structures and evaluates data sets to draw insights on how genetic variants will impact patients’ chances of developing disease and to determine the most appropriate treatment options, helping their doctors administer more personalized, holistic care plans. NorthShore will have the opportunity to benefit from disease network models developed by Sema4. For example, disease models for cancers will provide more comprehensive views of cancer patients, their tumor progression, and how best to diagnose or treat them.

Amidst the pandemic, as evidence reflects people are delaying preventative care, these more personalized genomic insights are particularly relevant in helping patients and physicians make informed decisions about prioritizing care.

"By narrowing the significant gap between available biomedical data and health and disease information, we are enabling NorthShore clinicians to make real-time use of the latest insights that allow for highly informed decisions at the point-of-care and improved health outcomes," said Eric Schadt, PhD, Founder and Chief Executive Officer of Sema4. "NorthShore has done what few health systems in the country have – brought the potential of genomics from the research labs to the front lines of care. Through the implementation of Sema4’s innovative health intelligence platform in a clinical-care setting, we are together advancing industry standards."

Improved Patient and Clinician Experiences

In addition to guiding clinicians, the program makes it easier for NorthShore patients to understand the implications of genomic findings. Novel digital tools and resources will uncover personalized insights that shape the care of multiple conditions throughout the lifetimes of patients. Patients also have an opportunity to consent to share de-identified data to improve research programs focused on expanding the understanding of genomic-guided care.

The strategic alliance between NorthShore and Sema4 will significantly expand on NorthShore’s existing clinical genomics offerings, which were already the largest in routine primary care in the U.S. More than 1,000 physicians and approximately 300,000 NorthShore patients will have access to Sema4’s offerings.

"Precision medicine represents the next major transformation in health care, allowing us to predict and prevent disease, to identify the right treatment at the right time, and to empower patients and clinicians to make better choices about health," said J.P. Gallagher, President and Chief Executive Officer of NorthShore. "This advanced genomics and health intelligence initiative with Sema4 builds on our long history of pioneering technology and genomic advancements to improve patient care – and aligns with our strong commitment to improving the accessibility of our best-in-class care to address documented disparities in health care services, disease prevalence, and outcomes."

Addressing Disparities in Care

Due to NorthShore’s intentional focus on increasing access to advanced clinical programs, the program is launching in conjunction with an investment to engage culturally diverse communities. In addition to understanding and addressing barriers to access, NorthShore, via a gift from the Swedish Hospital Foundation, will subsidize genetic testing costs for patients who do not have the financial resources to pay for testing.

Earlier this month, NorthShore began offering the program to patients of Swedish Hospital, a State of Illinois Safety Net Hospital located in a federally designated Medically Underserved Area and a member of the NorthShore family. Through the program, the system expects to gain insights about the views of diverse patient populations towards personalized medicine and barriers to receiving genetic information and testing.

On April 19, 2021 Oncotelic Therapeutics, Inc. ("Oncotelic" or the "Company") (OTCQB:OTLC) reported a presentation at AACR (Free AACR Whitepaper)-2021 (American Association for Cancer Research/ Annual Meeting) entitled: Combination therapy of anti-sense oligonucleotide targeting TGF-β2 (TASO) and IL-2 (Proleukin) has anti-cancer effect in solid cancer (Press release, Oncotelic, APR 19, 2021, View Source [SID1234578197]).

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TGF-β (transforming growth factor-beta) is an essential cytokine for tumor proliferation and metastasis. The expression of TGF-Β correlates with malignancy of various cancers and involves immunosuppression and angiogenesis of a tumor. IL-2 is a major cytokine to proliferate T cells and NK cells which are major players of cancer immunity. However, the toxicity of high dose IL-2 limits its use in cancer therapy. Combination treatment of TGF-β inhibitor and IL-2 would have an anti-tumor effect by immune cells through diminishing immunosuppression by TGF-β and enforcement of immune cells by IL-2. Trabedersen is an anti-sense oligonucleotide targeting human TGF-β mRNA. It is shown that Trabedersen is well tolerable in cancer patients and effective reagent to treat pancreatic cancer, melanoma, and glioblastoma. Proleukin is the only approved IL-2 reagent to treat Renal cell carcinoma and Melanoma.

Trabedersen and Proleukin activated human PBMC (Peripheral Blood Mononuclear Cell) are treated to several solid cancer cell lines, such as Breast cancer, Pancreatic cancer, Melanoma, Lung cancer, and Colon cancer to see the cytotoxicity effect of combination therapy of Trabedersen and Proleukin. NSG mouse (NOD Scid Gamma mouse, NOD.Cg-Prkdcscid Il2rgtm1Wjl/SzJ) which are humanized by human PBMC engraftment and the tumor growth of Melanoma and TNBC (Triple Negative Breast Cancer cell) cell line are monitored.

The combination treatment of Trabedersen and low dose Proleukin decreased cancer cell viability in vitro experiment in solid cancer cell lines. Melanoma and TNBC tumor growth was delayed in humanized NSG mouse model by Trabedersen and low dose IL-2 combination therapy and tumor growth delay was statistically significant to Trabedersen alone or IL-2 alone group. Tumor infiltrating lymphocyte population was increased in Trabedersen treated group and FoxP3+ regulatory T cell population in blood and tumor microenvironment was decreased by treatment of Trabedersen.

TGF-β inhibitor (Trabedersen) and low dose IL-2 (Proleukin) combination treatment is expected to be an effective regimen in solid cancer treatment than individual treatment by alteration of tumor environment. Modulation of the dose of Proleukin expects to help reduce the toxicity of IL-2 and increase the anti-cancer effect by combination with Trabedersen.

Oncotelic has previously announced the regulatory approval from the Ministry of Food and Drug Safety of Korea for the phase 1b clinical trial of a patented OT-101/IL-2 combination. This phase 1b clinical trial will confirm the safety and effectiveness of OT-101/IL-2 in solid cancer patients in cooperation with the UK global pharmaceutical company Clinigen Group. The study will be conducted together with Autotelic BIO- a partner of Mateon on OT-101/IL-2 combination.

OT-101 has received orphan drug designation for glioblastoma, melanoma, and pancreatic cancer. Furthermore, FDA recently granted Rare Pediatric Designation for OT-101 against diffuse intrinsic pontine glioma (DIPG). OT-101 is also effective against coronavirus including COVID-19 and being deployed against the COVID-19 epidemic.

OT-101 has demonstrated robust efficacy against pancreatic cancer, glioblastoma, and melanoma during phase 2 clinical trials. The demonstration that OT-101 will synergize with IL-2 further demonstrate its utility as adjunct to other immunotherapies. Interleukin-2 (IL-2, Aldesleukin, PROLEUKIN) Immunotherapy is cancer treatment that stimulates the body’s immune system to fight cancer, such as melanoma.