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Jazz Pharmaceuticals to Report 2021 First Quarter Financial Results on May 4, 2021

On April 19, 2021 Jazz Pharmaceuticals plc (Nasdaq: JAZZ) reported that it will report its 2021 first quarter financial results on Tuesday, May 4, 2021 after the close of the financial markets (Press release, Jazz Pharmaceuticals, APR 19, 2021, View Source [SID1234578203]). Company management will host a live audio webcast at 4:30 p.m. ET/9:30 p.m. IST to discuss first quarter 2021 financial results and provide a business and financial update.

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Interested parties may access the live audio webcast via the Investors section of the Jazz Pharmaceuticals website at www.jazzpharmaceuticals.com. A replay of the webcast will be archived on the website for at least one week.

AACR 2021-ImmVira presents the preclinical study results of MVR-T3011 via intravenous administration

On April 19, 2021 ImmVira reported the preclinical study results of MVR-T3011 via intravenous administration through publication in a virtual poster at the AACR (Free AACR Whitepaper) annual meeting (Press release, Immvira, APR 19, 2021, View Source [SID1234578202]). MVR-T3011 is a replication competent and genetically modified oncolytic herpes simplex virus-1 expressing human interleukin-12 and anti-PD-1 antibody. ImmVira owns the global right for development and commercialization of MVR-T3011 via intravenous administration.

Oncolytic virus(OV) has been proven to inhibit tumor growth in a number of superficial tumors through intratumoral injection. From clinical studies, intratumoral injection of OV has been proved to provide effective treatment against tumor via its abscopal effect, transitions from immunologically cold to hot tumor and combination with other cancer therapies. However, to advanced tumors, especially the lesions of multiple metastasis are ideally treated via a systemic route. At present, common types of viruses that have been studied clinically for intravenous administration are Vaccinia virus, Coxsackie virus and Reovirus etc. However, for various reasons, most virus types used in developing oncolytic viruses including HSV-1 and adenoviruses, have not been fully studied for intravenous administration preclinically and clinically.

In the preclinical study conducted by ImmVira, intravenous administration of MVR-T3011 demonstrated enrichment of viral DNA in tumor tissue. MVR-T3855, the surrogate of MVR-T3011 for mouse, was shown to significantly extend the survival time of several mouse with orthotopic tumors including primary and metastatic lung cancer and primary liver cancer, and repeated dose via intravenous administration has clearly enhanced antitumor effect. Furthermore, intravenous administration or intra-cavity of MVR-T3855 significantly delayed or prevented the formation of malignant ascites in mouse liver model. In terms of safety, the three-month pre-clinical study showed that the intravenous administration of MVR-T3011 and MVR-T3855 did not cause significant adverse reactions and had clinical significance. After administration, no histopathological change was found in non-target organs.

ImmVira submitted an IND application for MVR-T3011 intravenous administration to the U.S. FDA in January 2021 and expects to recruit patients in five leading U.S. research cancer centers within the next few weeks.

Veracyte Announces Data for Pulmonology Portfolio to be Presented at American Thoracic Society 2021 International Conference

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.

"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

Humanigen Reports Positive Data With Lenzilumab in the ZUMA-19 CAR-T Phase 1b Study in DLBCL and Plans to Initiate a Potential Registrational Study

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).

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."

Aptorum Group Limited Reports 2020 Fiscal Year End Financial Results and Provides Business Update

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]).

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.