On September 20, 2021 Benitec Biopharma Inc. (NASDAQ: BNTC) ("Benitec" or "the Company"), a development-stage, gene therapy-focused, biotechnology company developing novel genetic medicines based on the proprietary DNA-directed RNA interference ("ddRNAi") platform, reported the financial results for its fiscal year ended June 30, 2021 (Press release, Benitec Biopharma, SEP 20, 2021, View Source [SID1234588056]). The Company has filed its annual report on Form 10-K for the year ended June 30, 2021, with the U.S. Securities and Exchange Commission.

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Operational Updates

The key milestones related to the investigational agents under development by the Company and other corporate updates are outlined below:

BB-301 (Oculopharyngeal Muscular Dystrophy Program)

On September 8, 2021, Benitec announced three key updates related to the progress of the BB-301 development program, including: updated results for the BB-301 Pilot Dosing Study in large animals, updates on European and North American regulatory interactions for the BB-301 development program, and a comprehensive overview of the design of, and key primary and secondary endpoints for, the Phase 1b/2a clinical trial which is planned for initiation in 2022. All of the updates were positive and demonstrated the significant progress that has been achieved for the BB-301 development program; below is a summary of each update:
BB-301 Large Animal Pilot Dosing Study: On September 8th the Company disclosed updated analyses that continue to demonstrate robust, dose-dependent target tissue transduction for BB-301, dose-dependent gene expression for the three distinct components of the therapeutic transgene, and biologically significant knock-down of the target PABPN1 protein. These updated data provide continued support for the planned advancement of BB-301 into the Phase 1b/2a clinical study in 2022.
European Regulatory Interaction: Following the disclosure in February 2021 of the positive interim data from the BB-301 Pilot Dosing Study in large animals, Benitec completed a Scientific Advice Meeting with The National Agency for the Safety of Medicines and Health Products in France (L’Agence nationale de sécurité du médicament et des produits de santé or "ANSM") in the first half of 2021. At the conclusion of the meeting:
The BB-301 Pilot Dosing Study was viewed as an appropriate dose range finding study.
The design of the ongoing GLP Biodistribution and Toxicology study was viewed as appropriate to support Phase 1b/2a testing of BB-301.
The manufacturing plan for clinical grade BB-301 drug product can be conducted under GMP conditions with a production process analogous to that that employed in prior large-scale production runs for BB-301.
The design of the Phase 1b/2a clinical trial can support the evaluation of BB-301 safety and clinical efficacy in key populations of OPMD patients.
North American Regulatory Interaction: Benitec has been granted a Type C Meeting with the U.S. Food and Drug Administration ("FDA") in the fourth quarter of 2021.
BB-301 Phase 1b/2a Clinical Study Design: On September 8th the Company provided a comprehensive overview of the key design elements of the upcoming BB-301 Phase 1b/2a clinical trial. The Phase 1b/2a study is planned for 2022. In addition to the determination of the safety and tolerability profiles of BB-301, the secondary endpoints of the trial will facilitate the accurate and reproducible characterization of the key physiological processes underlying the successful completion of the pharyngeal phase of swallowing. The core analytical tools and methods that will be employed during the clinical study will focus on functional measures of swallowing efficiency for OPMD patients during the pharyngeal phase of swallowing.
Corporate Updates

On April 30, 2021, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents (the "April 2021 Capital Raise"). The Company received gross proceeds of approximately $14.3 million and net proceeds of approximately $12.7 million from the offering.
On October 6, 2020, the Company announced the closing of an underwritten public offering of common stock and common stock equivalents (the "October 2020 Capital Raise"). The Company received gross proceeds of approximately $11.5 million and net proceeds of approximately $9.9 million from the offering.
Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma commented, "Our team has made significant progress towards the goal of advancing BB-301 into clinical studies, and we remain focused on improving the lives of patients suffering from Oculopharyngeal Muscular Dystrophy. In the near future, we will begin to apply the ddRNAi platform to the treatment of other fatal genetic disorders with significant unmet medical need."

Financial Highlights

Total Revenues for the year ended June 30, 2021 were fifty-nine thousand dollars compared to $102,000 for the year ended June 30, 2020. Benitec recognized fifty-nine thousand dollars in customer revenues for the year ended June 30, 2021 compared to $97,000 for the comparable year ended June 30, 2020. The decrease in revenues from customers is due to the decrease in licensing and royalty revenues in the current year. During the year ended June 30, 2021, the Company recognized no revenue from government research and development grants, as compared $5 million for the comparable year ended June 30, 2020. The decrease in grant revenue is a result of Benitec no longer claiming the grant from the Australian government due to the Re-domiciliation of the Company to the United States of America.

Total expenses were $13.7 million for the year ended June 30, 2021 compared to $8.4 million for the comparable period in 2020. Royalty and license fees, research and development costs, and general and administrative costs comprise the primary corporate expenses. For the year ended June 30, 2021, Benitec incurred one hundred twenty-three thousand dollars in royalties and license fees compared to a gain of one hundred eighty-five thousand dollars for the comparable year ended June 30, 2020. The change is primarily due to a reversal of an accrual which created the negative balance for the year ended June 30, 2020. Benitec incurred $7 million of research and development expenses compared to $3 million for the comparable year ended June 30, 2020. The increase in research and development expenses are related to the pre-clinical studies associated with BB-301 as well as an increase in research and development costs related to stock-based compensation expenses. General and administrative expenses were $6.5 million and $5.5 million for the years ended June 30, 2021 and 2020, respectively. The increase for the year was due to the increase in insurance, consultants, legal and accounting fees.

The loss from operations for fiscal 2021 was $13.9 million compared to a loss of $8.3 million for fiscal 2020. Net loss attributable to shareholders for the fiscal year ended 2021 was $13.9 million, or $3.23 per basic and diluted share, compared to a net loss of $8.3 million, or $8.10 per basic and diluted share in earnings for the fiscal year ended 2020. At the end of fiscal year 2021 the Company had $19.8 million in cash and cash equivalents.

On September 20, 2021 Vaxart, Inc. (NASDAQ: VXRT) reported that Chief Executive Officer Andrei Floroiu and Chief Scientific Officer Dr. Sean Tucker will present at the 2021 Cantor Virtual Global Healthcare Conference at 10:40 a.m. ET on Thursday, September 30, 2021 (Press release, Vaxart, SEP 20, 2021, View Source [SID1234588055]).

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The presentation will be available by clicking here and on Vaxart’s website. The Company will also participate in one-on-one meetings during the conference.

Vaxart’s oral tablet COVID-19 vaccine is "The Pill that Moves the Needle" and the only oral COVID-19 vaccine whose potential is backed by clinical data. The Company’s oral tablet COVID-19 vaccine candidate has completed phase I trials and will enter phase II clinical trials this year.

On September 20, 2021 PharmAbcine Inc. (KOSDAQ: 208340ks), a clinical-stage biotech company focusing on the development of next generation antibody therapeutics, reported that the Company will virtually participate in the 27th annual BIO-Europe which will take place from October 25 – 28, 2021 (Press release, PharmAbcine, SEP 20, 2021, View Source [SID1234588053]).

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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BIO-Europe is Europe’s largest annual partnering conference serving the global life science industry. It attracts a wide range of business leaders, including senior executives of leading biotech companies, business development teams from large and midsize pharmaceutical companies, investors and other industry experts. This year, more than 3,500 executives from over 1,000 life sciences companies around the world are expected to participate in the event.

During the event, PharmAbcine’s business development team will conduct one-to-one partnering meetings with biotech companies around the world to broaden its global network, explore mutual interests, and identify recent trends in drug development market. Also, the Company will present a pre-recorded video which will be made available on-demand. The video will feature Dr. Jin-San Yoo, CEO of PharmAbcine, who will introduce the Company’s core technology and give updates on the R&D progress of the main pipelines such as olinvacimab, PMC-403, and PMC-309.

Olinvacimab, an anti-VEGFR2 (Vascular Endothelial Growth Factor Receptor 2) fully human antibody, is the Company’s leading pipeline and is undergoing multiple global clinical trials. In early September 2021, PharmAbcine initiated a Phase II olinvacimab and pembrolizumab combo study for the treatment of mTNBC (metastatic Triple-Negative Breast Cancer) in Australia. Also, a Phase II olinvacimab mono study for bevacizumab-nonresponding rGBM (recurrent glioblastoma multiforme) patients at multiple sites in both US and Australia and two Phase Ib olinvacimab and pembrolizumab combo trials in mTNBC and rGBM in Australia are still ongoing.

PMC-403 is a novel TIE2-activating fully human antibody designed to stabilize and repair damaged blood vessels in a variety of diseases. PMC-403 is currently in development for treating AMD (Age-related Macular Degeneration), DME (Diabetic Macular Edema), and DR (Diabetic Retinopathy) which are common abnormal vascular-related eye diseases. The Company expects PMC-403 to enter global clinical trials for both ophthalmology and oncology in 2022.

PMC-309 is a novel anti-VISTA (V-domain Ig Suppressor of T cell Activation) antagonizing antibody in development for the treatment of various tumor types. VISTA plays a pivotal role in maintaining the immunosuppressive environment around the tumor cells and is expressed primarily on MDSC (Myeloid-Derived Suppressor Cells) and Tregs (regulatory T cells). Blocking VISTA pathways activates T cells’ immune responses and leads to better anti-tumor effects. PMC-309 is expected to enter a global clinical trial in 2022.

To request a partnering meeting with PharmAbcine, register for the event here.

On September 20, 2021 Lipocine Inc. (NASDAQ: LPCN), a clinical-stage biopharmaceutical company focused on metabolic and endocrine disorders, reported that the Company will present at the Cantor Fitzgerald Virtual Global Healthcare Conference (Press release, Lipocine, SEP 20, 2021, View Source [SID1234588051]). The conference will be held virtually. Presentation details are below.

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Cantor Fitzgerald Virtual Global Healthcare Conference September 27-30, 2021

Presentation time: September 30, 2021 starting at 1:20 p.m. EST
Webcast link: View Source

The webcast of this presentation will also be available on Lipocine’s corporate website under "Events and Presentations" in the Investors section or using the webcast link above. The webcast will be available on Lipocine’s website for 90 days.

On September 20, 2021 miR Scientific reported that Data presented from a cross-validation study at the American Urological Association’s (AUA) 2021 Annual Meeting confirms that the miR Sentinel Prostate Cancer Test can detect and risk-classify prostate cancer at the molecular level with predictive accuracy of over 90%, based on a single urine sample (Press release, miR Scientific, SEP 20, 2021, View Source [SID1234588048]). This validation study follows and confirms the data on more than 1,400 patients published in The Journal of Urology in September 2020.

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Early/Late Stage Pipeline Development - Target Scouting - Clinical Biomarkers - Indication Selection & Expansion - BD&L Contacts - Conference Reports - Combinatorial Drug Settings - Companion Diagnostics - Drug Repositioning - First-in-class Analysis - Competitive Analysis - Deals & Licensing

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When comparing the results of the miR Sentinel Prostate Cancer Test to systematic core needle biopsy in men presenting with initial suspicion of prostate cancer, the latest validation study demonstrated a 93% concordance between the two methods of screening and detection regarding the classification of clinically significant cancer. This 93% sensitivity of the miR Sentinel Prostate Cancer Test sets a new bar for detecting and classifying prostate cancer. The data further demonstrate the strong negative predictive value of the miR Sentinel Prostate Cancer Test with results showing the test correctly identified 96% (371/387) of men as having non-clinically significant cancer.

"Our mission to inclusively transform cancer management is further validated in this prospective study, demonstrating that the miR Sentinel Prostate Cancer Test is able to provide an accurate non-invasive means to identify the presence or absence of any prostate cancer and classify the molecular risk of disease becoming metastatic, and thereby lethal, prior to biopsy" says Sam Salman, Chairman and CEO of miR Scientific. "We believe that the accuracy, accessibility, and non-invasive features of our award-winning technology will impact the lives of millions of men and forever positively evolve the standard of care for urological cancers."

Cross-Validation Study Results

The data reported in this late-breaking abstract and presented by Laurence Klotz, MD, FRCSC at the AUA, is based on a study of 763 men over 45 years old, eligible for their first core needle biopsy. Pathology based on the core needle biopsy showed 354 (46%) were biopsy negative and 409 (54%) were biopsy positive. Of those 409 men found to have pathologic evidence of disease, 189 (25%) were classified as Grade Group 1 (GG1) representing non-clinically significant cancer, and 220 were classified as Grade Group 2 (GG2) or higher representing clinically significant cancer.

With just a urine liquid biopsy, the miR Sentinel Prostate Cancer Test identified 204 men with clinically significant cancer (intermediate or high risk), demonstrating 93% sensitivity (204/220) with the core needle biopsy. When evaluating men with non-clinically significant cancer, the miR Sentinel Prostate Cancer Test identified 371 out of 387 men, delivering 96% (371/387) sensitivity.

The data further examined the apparent false positive rate for clinically significant cancer by analyzing the sub-set of men who underwent both systematic biopsy and MRI-fusion biopsy, and who were negative on systematic biopsy. The data demonstrates >95% concordance been the positive MRI-guided biopsy and the miR Sentinel Prostate Cancer Test, indicating that the discordance between systematic biopsy results and the miR Sentinel Prostate Cancer Test are largely attributable to false negatives of biopsy.

Laurence Klotz, MD, FRCSC, Professor of Surgery at the University of Toronto, and Chief Medical Officer of miR Scientific LLC, said, "The key features of the test are the very high negative predictive value and the very high accuracy [in] identifying cancer. In addition to its quick turnaround, the test is highly scalable: the company will be able to perform a very large number of these tests right away."

Innovative Urine-based Liquid Biopsy

The miR Sentinel Prostate Cancer Test, which already received the Breakthrough Device Designation by the FDA, is a non-invasive molecular test based on the analysis of small non-coding RNAs (sncRNA) isolated from non-DRE urinary exosomes. It provides an innovative method to analyze sncRNAs derived from a simple, non-invasive urine specimen from age-eligible men. Using only the expression dynamics of these sncRNAs, a proprietary Statistical Classification Algorithm derives scores that classify patients according to the likelihood of being in the following four possible groups: no molecular evidence of prostate cancer; low-risk; intermediate-risk; or high-risk prostate cancer.

The miR Sentinel Prostate Cancer Test is expected to be commercially available in the United States and Puerto Rico at the end of this year.