On November 15, 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 Q1 ended September 30, 2021 (Press release, Benitec Biopharma, NOV 15, 2021, View Source [SID1234595648]). The Company has filed its quarterly report on Form 10-Q for the quarter ended September 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 provided 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 Pilot Dosing Study in Large Animals: On September 8th the Company disclosed updated analyses for the animal subjects dosed with BB-301 in the Pilot Dosing Study. The updated data continued to demonstrate dose-dependent transduction of the target pharyngeal muscle tissues by BB-301, dose-dependent gene expression for the three distinct components of the therapeutic BB-301 transgene, and biologically significant levels of gene silencing ("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.
Regulatory Interactions in Europe: 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 in large animals was viewed as an appropriate dose range finding study.
The design of the ongoing GLP Toxicology and Biodistribution 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 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.
Regulatory Interactions in the United States: Benitec has been granted a Type C Meeting with the U.S. Food and Drug Administration ("FDA") in the fourth quarter of 2021.
Regulatory Interactions in Canada: Benitec has been granted a Pre-CTA Consultation Meeting with Health Canada 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.
Financial Highlights

Total Revenues for the quarter ended September 30, 2021 were $0 compared to fifty-five thousand dollars in total revenue for three months ended September 30, 2020. The decrease in revenues from customers is due to the decrease in licensing and royalty revenues in the current period.

Total Operating Expenses were $4.8 million for the quarter ended September 30, 2021 compared to $2.7 million for the comparable period in 2020. For the quarters ended September 30, 2021 and 2020, respectively, Benitec incurred $0 and $134 thousand in royalties and license fees. During the three months ended September 30, 2021 and 2020, respectively, the Company incurred $2.8 million and $0.8 million in research and development expenses. The increase in research and development expenses is primarily related to the commencement of the BB-301 GLP Toxicology and Biodistribution Study in large animals at Charles River Laboratories. Additionally, the Company incurred expenses related to the production of GMP-grade drug product to facilitate submissions of the Clinical Trial Applications outside of the United States and the Investigational New Drug Application in the United States, as these activities are critical to the planned initiation of the Phase 1b/2a clinical trial for BB-301 in 2022. General and administrative expenses were $2.0 million and $1.8 million for the three months ended September 30, 2021 and 2020, respectively. The increase during this three-month period was due to small increases in insurance costs, consultant fees, and legal and accounting fees.

Jerel A. Banks, M.D., Ph.D., Executive Chairman and Chief Executive Officer of Benitec Biopharma commented, "Our team continues to focus on the core development activities that will ensure the advancement of BB-301 into the first-in-human study. Our primary goal has been, and will continue to be, the improvement of the lives of patients suffering from genetic disorders for which no curative interventions exist. We believe that the initiation of the Phase 1b/2a study is critical for patients in the Oculopharyngeal Muscular Dystrophy community and represents an important milestone with respect to our long-term goals as a company."

On November 15, 2021 Athos Therapeutics, Inc., a late preclinical stage biotechnology company pioneering the development of first-in-class precision therapeutics for patients with autoimmune diseases and cancer, reported the completion of an oversubscribed $15MM Series A financing (Press release, Athos Therapeutics, NOV 15, 2021, View Source [SID1234595647]).

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The funding will be used to advance the company’s pre-clinical and clinical programs, including the initiation of a first in human phase I clinical trial of ATH-63 in 2022. Athos is developing ATH-63 for Inflammatory Bowel Disease, Lupus, other autoimmune disorders, and select cancer indications. Funding will also be used to develop additional novel targets for autoimmune diseases and two first-in-class small molecule inhibitors for solid cancers and AML. Additionally, Athos will further advance its proprietary artificial intelligence drug discovery platform, and substantially expand its patient biomaterial omics database through collaborations with large academic medical centers all over the world, including the Cleveland Clinic Foundation.

Athos Therapeutics completes an oversubscribed $15MM Series A financing

"I am delighted at the progress Athos has made since our Seed round of $4.25MM in March of 2020. Those seed funds enabled us to hire a stellar team, develop a powerful drug discovery engine, perform multi-omics analysis of annotated patient samples from key IBD centers around the world, file 8 patents, and create a medicinal chemistry platform that produced 100’s of highly selective and safe compounds with excellent pharmacologic profiles. The addition of more than $15MM in new funding will allow us to complete a Phase 1 human clinical trial with ATH-63 and progress our autoimmune and novel cancer programs towards the clinic," said Dimitrios Iliopoulos, PhD, MBA, President & Chief Executive Officer of Athos. "Furthermore, we are excited about the development of a new small molecule inhibitor that addresses the unmet needs of a subpopulation of AML patients," he added.

ATH-63 is a small molecule, first-in-class, approach to the massive unmet medical need of IBD. Medications currently prescribed for IBD are associated with limited efficacy, serious side effects, and were not developed for any specific IBD patient subtype. The development of ATH-63 was the result a meticulous systems biology approach where Athos was able to characterize IBD subtypes at the molecular level, using a proprietary biologic and network computational analysis of multi-omics data from highly annotated IBD patient samples.

"The field of IBD is in dire need of more precise, long-lasting, and far safer therapeutic options for millions of patients that suffer from the disease. In response to this need, we developed a highly targeted, precision medicine drug development platform to specifically identify compounds, like ATH-63, that we think will be game-changers for patients," said Allan J. Pantuck, MD, MS, Chairman and Chief Medical Officer of Athos. "Our entire team and Board want to extend our sincere thanks to our original seed funders, the many seed holders who also participated in the Series A, and our new Series A investors. We are honored to have all of you on Team Athos," he added.

On November 15, 2021 BioAtla, Inc. (Nasdaq: BCAB), a global clinical-stage biotechnology company focused on the development of Conditionally Active Biologic (CAB) antibody therapeutics, reported financial results for the third quarter of 2021 and provided an update on its clinical progress and business (Press release, BioAtla, NOV 15, 2021, View Source [SID1234595646]).

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"BioAtla continues to advance the progress of the potentially registration-enabling Phase 2 clinical trials for our two lead CAB product candidates as well as our other preclinical and pipeline programs. The increase in our financial resources in the third quarter through our equity placement provides the funding into the first half of 2024 to develop these key assets and the several additional ADC and bispecific CAB candidates in our development pipeline," stated Jay M. Short, Ph.D., Chairman, Chief Executive Officer and co-founder of BioAtla, Inc. "We are aggressively advancing all of our clinical programs and are on track for a sarcoma Phase 2 interim data read out by year-end for BA3011, CAB-AXL-ADC. Additionally, a clinical trial for CAB-CTLA-4 is expected to be initiated by the end of the year," added Scott Smith, President of BioAtla.

Advancing clinical trials for lead candidates
Mecbotamab Vedotin (BA3011)
We are developing BA3011, CAB-AXL-ADC, a conditionally and reversibly active antibody drug conjugate targeting the receptor tyrosine kinase AXL, as a potential therapeutic for multiple solid tumor types, including soft tissue and bone sarcoma, non-small cell lung cancer (NSCLC) and ovarian cancer, with other potential indications in the future. The Office of Orphan Drug Products (OODP) at FDA granted Orphan Drug Designation to BA3011 for the treatment of soft tissue sarcoma, and Phase 1 results in sarcoma patients were presented at the recent Connective Tissue Oncology Society (CTOS) 2021 Annual Meeting. BA3011 was generally well tolerated in this refractory sarcoma population. Few patients discontinued due to an adverse event (2 patients out of 26 or 7.7%). No clinically meaningful on-target toxicity to normal AXL-expressing tissue was observed. Dose-limiting toxicities were limited to monomethyl auristatin E (MMAE) conjugate-associated toxicity at the highest dose tested, including reversible neutropenia.The degree of AXL tumor membrane expression correlated with response to treatment. Of the seven sarcoma patients who had an AXL tumor membrane percent score of greater than or equal to 70, four of these obtained a confirmed partial response, including patients with leiomyosarcoma, undifferentiated pleomorphic sarcoma, and Ewing sarcoma. Prolonged response to therapy was observed in this ongoing study with the duration of response ranging from 33 to more than 60 weeks. Overall, mecbotamab vedotin may have a favorable benefit-risk profile, and importantly this is one of the few studies with a putative biomarker which is not only highly expressed in sarcomas, but also may help select patients across multiple sarcoma subtypes who may benefit from therapy. In the ongoing potentially registration-enabling sarcoma Phase 2 study, patients are enrolled for therapy by prescreening for AXL expression. We also are conducting a Phase 2 study (BA3011-002) in AXL high NSCLC patients who have previously progressed on PD-1/L1, EGFR, or ALK inhibitor therapy. We currently expect to have enrolled more than 70 patients by year end in our sarcoma Phase 2 trial in the U.S. Interim analyses in the sarcoma and NSCLC trials are anticipated this year and in early 2022, respectively. In addition, a multi-center investigator-initiated Phase 2 clinical trial of BA3011 in combination with a PD-1 inhibitor in patients with platinum-resistant ovarian cancer is expected to begin enrollment by year end.

BA3021 (Ozuriftamab Vedotin)
BA3021, CAB-ROR2-ADC, is a conditionally and reversibly active antibody drug conjugate directed against ROR2, a receptor tyrosine kinase that is overexpressed across many different solid tumors including lung, head and neck, melanoma and breast. We are developing BA3021 as a potential therapeutic for multiple solid tumor types, including NSCLC, melanoma, squamous cell cancer of the head and neck (SCCHN) and ovarian cancer. Based on Phase 1 data we believe BA3021 has broad potential as a cancer therapy for patients with advanced solid tumors that have previously progressed on a PD-1 inhibitor. We are enrolling a Phase 2 trial of BA3021 monotherapy or in combination with a PD-1 inhibitor in patients with ROR2 high melanoma who have previously progressed on PD-1/L1 inhibitor and patients with ROR2 high NSCLC who have previously progressed on PD-1/L1, EGFR or ALK inhibitor therapy. A Phase 2 study in patients with ROR2 high SCCHN is anticipated to enroll in early 2022. In addition, a multi-center investigator-initiated Phase 2 clinical trial of BA3021 in combination with a PD-1 inhibitor in patients with platinum-resistant ovarian cancer is expected to begin enrollment by year end.

BA3071
BA3071, is a CAB anti-CTLA-4 antibody that is being developed as an immuno-oncology agent with the goal of delivering efficacy comparable to the approved anti-CTLA-4 antibody, ipilimumab, but with lower toxicities due to the CAB’s tumor microenvironment-restricted activation. The development of BA3071 is the subject of a 2019 Global Co-Development and Collaboration Agreement between BioAtla and BeiGene, Ltd. Like BA3011, BA3021 and our other CAB candidates, BA3071 is designed to be conditionally and reversibly activated in the tumor microenvironment via the Protein-associated Chemical Switch or PaCS mechanism discovered by BioAtla scientists. This proprietary system enables reduction of systemic toxicity and potentially enables safer combination therapies, such as with anti-PD-1 antibody checkpoint inhibitors in the case of BA3071. BA3071 is being developed as a potential therapeutic for multiple solid tumor indications, including renal cell carcinoma, NSCLC, small cell lung cancer, hepatocellular carcinoma, melanoma, bladder cancer, gastric cancer and cervical cancer. We are currently in advanced discussions with BeiGene regarding the allocation of roles and responsibilities for global development and commercialization of BA3071 under our Global Co-Development and Collaboration Agreement with BeiGene. As part of these ongoing discussions, BeiGene is planning to initiate the transfer of the IND for BA3071 to BioAtla and BioAtla anticipates initiating the Phase I trial for BA3071 during 2021, with dosing commencing in the first half of 2022.

Plans to advance development of several bispecific CAB candidates
We have also leveraged our CAB technology to develop bispecific antibodies, which bind both a tumor-specific antigen and a T cell receptor (CD3) using CAB antigen-binding domains. With this design, bispecific antibodies can induce potent T cell responses against tumors expressing the tumor target antigen. We have shown in preclinical experiments that our CAB bispecific molecules meet or exceed the activity of conventional bispecifics and reduce systemic activation of potentially severe immune responses. We are conducting IND-enabling studies for two CAB bispecific antibody product candidates, EpCAM/CD3 and B7-H3/CD3, and presently plan to file INDs in mid-2022 and by year end 2022, respectively. We also are evaluating additional candidates including EGFR and Nectin-4 for CAB CD3 bispecific modalities. Nectin-4 is also progressing as a CAB ADC candidate. Overall, we are advancing multiple pre-clinical assets with the potential to submit up to four US INDs over the next 18 months for our CAB bispecific or ADC molecules.

Sheri Lydick joins to lead commercial strategy
BioAtla is building its capabilities to plan for and execute the commercial launches of its CAB portfolio of product candidates. A key element of this strategy is the recent hiring of Sheri Lydick as the company’s Senior Vice President, Commercial Strategy. Ms. Lydick will be leading commercial strategy, which includes long-range portfolio planning, assessing strategic business opportunities and delivering on these plans. Ms. Lydick has more than 20 years of leadership and commercialization experience in the biotechnology and pharmaceutical industry. In her most recent role at Bristol-Meyers Squibb Company, Ms. Lydick was responsible for building the sales and marketing teams and leading the commercial launch of Zeposia across multiple therapeutic areas (multiple sclerosis and ulcerative colitis). In her twelve years (2007-2019) at Celgene her leadership role expanded from Director of Global Marketing for the Inflammation and Immunology (I&I) Franchise to Executive Director, US Rheumatology Lead, to Vice President, US Commercial Lead for Zeposia. Among her accomplishments, she was instrumental in building Celgene’s Inflammation and Immunology franchise and led the planning and launch of the multi-billion dollar drug Otezla. BioAtla president Scott Smith commented, "Sheri has deep experience and success in global commercial marketing and sales and in building and leading multidisciplinary teams. We are excited for her to bring that expertise to BioAtla."

Third quarter 2021 financial results
Cash and cash equivalents as of September 30, 2021 were $269.9 million. We expect current cash and cash equivalents will be sufficient to fund planned operations into the first half of 2024. In September 2021, we executed a private placement of common stock yielding gross proceeds of $75.0 million before deducting placement agent fees and other offering expenses. The investors included both current and new institutional investors.

Research and development (R&D) expenses were $16.6 million for the quarter ended September 30, 2021 compared to $4.9 million for the same quarter in 2020. We expect our R&D expenses to increase substantially for the foreseeable future as we continue to invest in R&D activities to advance our product candidates, and our clinical programs and expand our product candidate pipeline.

General and administrative (G&A) expenses were $7.1 million for the quarter ended September 30, 2021 compared to $3.3 million for the same quarter in 2020. We expect our G&A expenses to increase as a result of operating as a public company. In addition, we expect our intellectual property expenses to increase as we expand our intellectual property portfolio.

Net loss for the third quarter ended September 30, 2021 was $22.9 million compared to a net loss of $11.6 million for the same quarter in 2020. Net cash used in operating activities for the first nine months of 2021 was $41.3 million compared to net cash used in operating activities of $22.3 million for the same period in 2020.

On November 15, 2021 Immunome, Inc. (Nasdaq: IMNM), a biopharmaceutical company that utilizes its human memory B cell platform to discover and develop first-in-class antibody therapeutics, reported financial results for the third quarter ended September 30, 2021 and provided a corporate update (Press release, Immunome, NOV 15, 2021, View Source [SID1234595645]).

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"We made significant progress in advancing our three-antibody cocktail to combat SARS-CoV-2, IMM-BCP-01, reporting additional activity against emerging variants, Lambda and Delta Plus. We now have demonstrated compelling neutralizing activity of IMM-BCP-01 in all of the variants of concern, as identified by the CDC, and look forward to submitting our IND this quarter and reporting topline data in the first half of 2022," commented Purnanand Sarma, Ph.D., President and CEO of Immunome. "We continue to advance IMM-ONC-01, our novel innate immune checkpoint that targets IL-38, towards an IND submission that we expect in early 2022."

Third Quarter and Subsequent Highlights

Demonstrated Potent Neutralization Activity of IMM-BCP-01 Against SARS-CoV-2 Lambda and Delta Plus Variants. In September 2021, Immunome announced that its three-antibody cocktail (IMM-BCP-01) has demonstrated robust neutralizing activity against SARS-CoV-2 Lambda (C.37) and Delta AY.1/2 (Delta Plus) variants. Furthermore, IMM-BCP-01 has been shown to neutralize all of the Centers for Disease Control (CDC) variants of concern in preclinical testing*.
Presented Data on Anti-IL38 Antibody Program at AACR (Free AACR Whitepaper)-NCI-EORTC Molecular Targets and Cancer Therapeutics Conference. In October 2021, Immunome presented an oral presentation of its anti-IL-38 monoclonal antibody that demonstrated the mAb inhibits tumor growth in two different murine syngeneic tumor models and induces protection following implantation of secondary tumors. Additionally, Immunome’s mAb binds to human IL-38 while inhibiting binding to its putative receptors, IL1RAPL1 and IL-36R, as well as inhibiting IL-38-mediated suppression of myeloid cell activity in vitro.
Submitted Preclinical Research of IMM-BCP-01 Against SARS-CoV-2 for Publication in bioRxiv. In October 2021, Immunome announced its submission of a preprint manuscript on its preclinical findings of its antibody cocktail, IMM-BCP-01, to bioRxiv. The manuscript detailed IMM-BCP-01’s potent activation of phagocytosis and complement fixation, which is known to be a component of in vivo treatment efficacy, as well as potent efficacy in pseudovirus neutralization against the Delta variant of SARS-CoV-2*.
* Immunome received funding from the U.S. Department of Defense’s (DOD) Joint Program Executive Office for Chemical, Biological, Radiological and Nuclear Defense (JPEO-CBRND), in collaboration with the Defense Health Agency (DHA), for these studies. (Contract number: W911QY-20-9-0019)

Financial Highlights

Research and development (R&D) expenses: R&D expenses for the three months ended September 30, 2021 were $4.5 million.
General and administrative (G&A) expenses: G&A expenses for the three months ended September 30, 2021 were $3.2 million.
Net loss: Net loss for the three months ended September 30, 2021 was $7.7 million.
Cash and cash equivalents: As of ended September 30, 2021, cash and cash equivalents totaled $56.3 million.

On November 15, 2021 SQZ Biotechnologies (NYSE:SQZ), focused on unlocking the full potential of cell therapies for multiple therapeutic areas, reported that management will be presenting at the Stifel 2021 Virtual Healthcare Conference on November 17 (Press release, SQZ Biotech, NOV 15, 2021, View Source [SID1234595644]). Armon Sharei, Ph.D., Chief Executive Officer, will present a corporate overview and the company will be hosting one-on-one meetings.

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PRESENTATION DETAILS

Wednesday, November 17
Stifel 2021 Virtual Healthcare Conference
10:00 am ET
Webcast

Specific conference webcast details and the company’s corporate overview presentation will be available on the Investors & Media section of the SQZ website. The webcast will be available for 90 days following the presentation.