On December 28, 2022 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the fiscal year ended September 30, 2022, as well as key clinical and corporate developments (Press release, Cel-Sci, DEC 28, 2022, View Source [SID1234625651]).

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Clinical and Corporate Developments:

CEL-SCI’s main focus throughout fiscal year 2022 has been to complete and submit a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for approval of Multikine* (Leukocyte Interleukin, Injection) in the treatment of advanced primary head and neck cancer.

Current drug therapies for squamous cell carcinoma of the head and neck (SCCHN) from Merck and Bristol Myers Squibb (BMS) are approved as a last resort for recurrent tumors after treatments have failed or for patients who are not candidates for surgery. By contrast, Multikine is given to newly-diagnosed patients following initial diagnosis and ahead of surgery— it’s the first of its kind with substantial survival benefit in a randomized Phase 3 trial in locally advanced primary SCCHN.

The Multikine treated study population who receive surgery and radiotherapy as their standard of care treatments showed the following advantages over control:
A median overall survival improvement of 46.5 months— nearly four years.
62.7% of Multikine patients were alive after five years vs. 48.6% in the control.
Nearly one out of every six patients had their tumors shrink by more than 30% in just 3 weeks and prior to surgery.
Five patients had their tumors completely disappear in just 3 weeks and prior to surgery.
Tumor shrinkage/disappearance cut the death rate by a factor of three.
CEL-SCI published and presented data from its pivotal randomized controlled Phase 3 Multikine head and neck cancer clinical trial at two prestigious cancer conferences.
2022 ASCO (Free ASCO Whitepaper) Annual Meeting in Chicago, Illinois
"Novel algorithm for assigning risk/disease-directed treatment (DDT) choice in locally advanced primary squamous cell carcinoma of the head and neck (SCCHN): Using pretreatment data only." – Abstract
"Leukocyte interleukin injection (LI) immunotherapy extends overall survival (OS) in treatment-naive low-risk (LR) locally advanced primary squamous cell carcinoma of the head and neck: The IT-MATTERS study." – Abstract and Poster Presentation
European Society for Medical Oncology (ESMO) (Free ESMO Whitepaper) annual Congress in Paris, France
Early response to Neoadjuvant Leukocyte Interleukin Injection (LI) immunotherapy extends overall survival (OS) in locally advanced primary squamous cell carcinoma (SCC) of the head & neck (HN): the IT-MATTERS Study – Poster Presentation and Video Presentation
Histopathology (HP) biomarkers confirm Leukocyte Interleukin Injection (LI) treatment (Tx) outcome in naïve locally advanced primary head & neck squamous cell carcinoma (SCCHN) the IT-MATTERS Study – Poster Presentation
Additional results from the Phase 3 clinical trial of Multikine in advanced primary head and neck cancer were posted to the U.S. government clinical trial website www.clinicaltrials.gov.
CEL-SCI has submitted additional abstracts/manuscripts to be published/presented.
CEL-SCI’s dedicated manufacturing facility in which it manufactures Multikine is being validated following the completion of its commercial scale build out during the first quarter of 2022. The construction was designed to ensure the facility will be compliant with all FDA GMP and European regulations.
As of September 30, 2022, CEL-SCI had $22.7 million in cash and cash equivalents.
"We are the first to have achieved successful neoadjuvant (before surgery) immunotherapy in head and neck cancer. Keytruda and Opdivo, the two most successful cancer drugs today, have not shown a survival benefit as a neoadjuvant for advanced primary head and neck cancer, and the last FDA approval for this disease was decades ago. We have shown a very large improvement in survival for the patients scheduled to receive surgery and radiotherapy as their standard of care treatments. We have presented at top cancer conferences. We are very proud of this success, but I would like to increase the speed of our progress towards submitting a BLA and gaining marketing approval in the U.S. as well as in Canada. A number of factors are responsible for the rate at which we have been progressing. First, we have succeeded at something that has never been done successfully before—show improvement in survival from a neoadjuvant for advanced primary head and neck cancer patients. Second, the study that supports this achievement was long, enrolled a large number (928) of patients, and produced an extensive amount of data that had to be analyzed and interpreted. Third, because we are not a big pharma company with virtually unlimited staff and resources, we have not been able to complete our milestones as quickly as bigger companies would have done. But, as has always been the case with CEL-SCI, our tenacity gets the job done and done well. The Phase 3 study results bear this out. I am looking forward to a lot of positive activity and progress next year as we continue in our efforts to bring Multikine to market for patients with a severe unmet medical need," stated CEL-SCI CEO, Geert Kersten.

Financial Results

CEL-SCI incurred a net operating loss of approximately $36.1 million for the twelve months ended September 30, 2022, of which approximately $11.4 million was non-cash expense including stock-based employee compensation and approximately $3.8 million in depreciation and amortization expense while cash spent was $18.2 million. The cash balance on September 30, 2022 was $22.7 million.

During the year ended September 30, 2022, research and development expenses increased by approximately $2.2 million, or 10%, and general and administrative expenses decreased by approximately $2.4 million, or 18%, compared to the year ended September 30, 2021.

CEL-SCI’s audited financial statements contained an audit opinion from its independent registered public accounting firm that included an explanatory paragraph related to CEL-SCI’s ability to continue as a going concern.

Biomica Ltd., an emerging biopharmaceutical company developing innovative microbiome-based therapeutics, and a subsidiary of Evogene Ltd. (NASDAQ: EVGN) (TASE: EVGN), reported that it will be in attendance at the Healthcare Week taking place together with the J.P. Morgan Healthcare Conference in San Francisco (Press release, Biomica, DEC 28, 2022, View Source [SID1234625649]). The conference is one of the industry’s largest conferences of the year.

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This follows Biomica’s recent successful financing round of $20 million, led by Shanghai Healthcare Capital, a leading Chinese private equity fund. In attendance will be Dr. Elran Haber, CEO of Biomica, throughout the week from Monday, January 9 until Wednesday, January 11, 2023.

Dr. Haber will be available for one-on-one meetings throughout the week, and those interested should be in touch with Biomica’s investor or public relations team.

On December 28, 2022 Mission Bio, the pioneer in high-throughput single-cell DNA and multi-omics analysis, and Abiosciences, the single-cell bioinformatics leader, reported a new partnership to co-develop bioinformatics packages for translational and clinical research applications in hematological cancers (Press release, Mission Bio, DEC 28, 2022, View Source [SID1234625647]). The goal is to combine Mission Bio’s industry-leading single-cell DNA and multi-omics capabilities with Abiosciences’ cutting-edge artificial intelligence expertise, to uncover new disease signatures and address therapeutic resistance. These new capabilities can be leveraged in patient stratification for clinical trials and potentially, as a companion diagnostic tool.

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Mission Bio’s Tapestri Platform is a powerful tool that has been adopted increasingly for translational research and clinical trials, both at biopharma companies and in academia. In the past month alone, presentations by gene therapy biotech AVROBIO at the Gene Therapy Analytical Development (GTAD) Summit and Servier Pharmaceuticals at the American Society of Hematology (ASH) (Free ASH Whitepaper) Annual Meeting demonstrated the platform’s versatility in clinical trials, helping identify mechanisms of disease resistance in a Phase 3 trial and measuring critical quality attributes for clinical samples, respectively. Academic teams at ASH (Free ASH Whitepaper) from City of Hope, Memorial Sloan Kettering Cancer Center, and more described a range of clinical research use cases for Tapestri-based single-cell multi-omics assays, such as matching patients with leukemia to personalized care that reduced mortality in a clinical trial, and screening for dangerous cancer clones linked to therapeutic resistance.

Abiosciences will leverage its AI platform and bioinformatics expertise to create a unique and advanced bioinformatic analytical algorithm, in order to gain deeper and more comprehensive insights into disease progression, development of resistance, and MRD after single-cell multi-omics analysis for researchers in China. Mission Bio will support Abiosciences with its unique Tapestri single-cell multi-omics platform, custom panels, subject matter experts, and R&D expertise.

"Abiosciences has been committed to applying our single cell research platform and our big data/AI bioinformatics platform to unravel the complex heterogeneity underlying major diseases, such as cancer. Through the numerous algorithms and AI platforms exclusively developed by the company, we can more flexibly and effectively process the huge amount of data generated by single cell sequencing, find more valuable clinical therapeutic biomarkers, and help the translate the technology for clinical use," said Tao Hong, Co-Founder & Chief Operating Officer at Abiosciences, "We are very happy to work with Mission Bio to provide more powerful technical support for clinical and translational researchers and implementation of single cell technology by combining our big data/AI platform of bioinformatics with Mission Bio’s single-cell multi-omics technology."

"Elucidating tumor heterogeneity and clonal evolution are important to understand therapeutic resistance, and will be a crucial collaboration for the hematological oncology community," said Yan Zhang, PhD, CEO of Mission Bio. "It also represents our continued efforts to support biotechnology companies in China that share our aim of transforming healthcare through collaboration and scientific advancement. We look forward to integrating our strength in single-cell multi-omics with Abiosciences’ complementary strengths in AI to advance analytical bioinformatics, and discover new biomarkers with potential clinical utility."

To learn more about Mission Bio and the Tapestri Platform, please visit www.missionbio.com.

On December 28, 2022, Novavax, Inc. (Nasdaq: NVAX), a biotechnology company (the "Company" or "Novavax") dedicated to developing and commercializing next-generation vaccines for serious infectious diseases, reported the completed closing of its previously announced offering of $150 million aggregate amount of 5.00% Senior Convertible Notes due 2027 (the "Notes"), and on December 23, 2022, the Company completed the closing of the initial purchasers’ option to purchase up to an additional $25.25 million aggregate principal amount of the Notes, which was exercised in full (Press release, Novavax, DEC 28, 2022, View Source [SID1234625646]). On December 20, 2022, the Company also completed the closing of its previously announced underwritten public offering of 7,475,000 shares of its common stock, including the exercise in full of the underwriters’ option to purchase up to an additional 975,000 shares of its common stock (the "Common Stock Offering") resulting in total gross proceeds of $74.75 million. Total combined gross proceeds to the Company from both the offering of Notes and the Common Stock Offering was $250 million.

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The Notes were offered and sold only to persons reasonably believed to be qualified institutional buyers pursuant to Rule 144A under the Securities Act of 1933, as amended (the "Securities Act"). J.P. Morgan, Jefferies and Cowen acted as joint book-running managers and representatives of the initial purchasers for the offering of the Notes. J. Wood Capital Advisors served as financial advisor to the Company in relation to the offering of the Notes.

The Notes bear cash interest at a rate of 5.00%, payable on June 15 and December 15 of each year, beginning on June 15, 2023. The Notes will be redeemable, in whole or in part (subject to certain limitations), for cash at Novavax’ option at any time, and from time to time, on or after December 22, 2025, if the last reported sale price of Novavax’ common stock has been at least 130% of the conversion price then in effect for at least 20 trading days (whether or not consecutive), during any 30 consecutive trading day period (including the last trading day of such period) ending on and including the trading day immediately preceding the date on which Novavax provides notice of redemption at a redemption price equal to 100% of the principal amount of the Notes to be redeemed, plus any accrued and unpaid interest to, but excluding, the redemption date. The Notes are convertible at an initial conversion rate of 80.0000 shares of the Company’s common stock per $1,000 principal amount of Notes, which is equivalent to an initial conversion price of $12.50 per share of Novavax’ common stock, representing a 25% conversion premium of the public offering price in the Company’s concurrent common stock offering that closed on December 20, 2022. The Company will settle conversions by paying or delivering, as applicable, cash, shares of common stock, or a combination of cash and shares of common stock, at the Company’s election.

Novavax estimates that the net proceeds from the offering of the Notes are approximately $166.3 million, after deducting the initial purchasers’ discounts and commissions and estimated offering expenses.

The offer and sale of the Notes and any shares of common stock issuable upon conversion of the Notes have not been, and will not be, registered under the Securities Act or any other securities laws, and the Notes and any such shares cannot be offered or sold absent registration or except pursuant to an applicable exemption from, or in a transaction not subject to, the registration requirements of the Securities Act and any other applicable securities laws.

For the Common Stock Offering, shares were issued at $10.00 per share, resulting in total gross proceeds from the Common Stock Offering of $74.75 million before deducting the underwriters’ discount and offering expenses. J.P. Morgan, Jefferies and Cowen acted as joint book-running managers and representatives of the underwriters for the Common Stock Offering. B. Riley Securities and H.C. Wainwright & Co. acted as co-lead managers for the Common Stock Offering.

Novavax estimates that the net proceeds from the Common Stock Offering are approximately $69.8 million, after deducting the underwriters’ discounts and commissions and estimated offering expenses.

A registration statement relating to the Common Stock Offering was filed with the Securities and Exchange Commission ("SEC") on March 11, 2020, and is effective. The Common Stock Offering was made by means of a prospectus supplement and the accompanying prospectus. An electronic copy of the preliminary prospectus supplement and accompanying prospectus relating to the Common Stock Offering are available on the SEC’s website at www.sec.gov, and may also be obtained from: J.P. Morgan, Attention: Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, by telephone at (866) 803-9204 or by email at [email protected]; Jefferies LLC, Attention: Equity Syndicate Prospectus Department, 520 Madison Avenue, New York, NY 10022, by telephone at (877) 821-7388 or by email at [email protected]; or Cowen and Company, LLC, c/o Broadridge Financial Solutions, 1155 Long Island Avenue, Edgewood, NY 11717, Attn: Prospectus Department, by telephone at (833) 297-2926, or by email at [email protected].

On December 28, 2022 PDS Biotechnology Corporation (Nasdaq: PDSB), a clinical-stage immunotherapy company developing a growing pipeline of targeted immunotherapies for cancer and infectious disease, reported expanded interim data in a Phase 2 clinical trial investigating the PDS0101-based triple combination therapy in advanced human papillomavirus (HPV)-positive cancers (Press release, PDS Biotechnology, DEC 28, 2022, View Source [SID1234625644]). The triple combination of PDS0101 with the tumor-targeting IL-12 fusion protein M9241 (formerly known as NHS-IL12), and bintrafusp alfa, a bifunctional fusion protein targeting two independent immunosuppressive pathways (PD-L1 and TGF-β), is being studied in CPI-naïve and CPI-refractory patients with advanced HPV-positive anal, cervical, head and neck, vaginal, and vulvar cancers.

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The triple combination Phase 2 trial (NCT04287868) is being conducted at the Center for Cancer Research (CCR) at the National Cancer Institute (NCI), one of the Institutes of the National Institutes of Health.

All patients in the study had failed prior treatment with chemotherapy and 90% had failed radiation treatment. The interim efficacy data (n=50) involves 37 HPV16-positive evaluable patients, including 29 patients who have, in addition, failed treatment with CPIs (CPI refractory). Highlights of the expanded interim data are as follows and are consistent with the results presented at American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting 2022 and prior interim data announced in October:

Median OS is 21 months in 29 checkpoint inhibitor refractory patients who received the triple combination. The reported historical median OS in patients with CPI refractory disease is 3-4 months.

In CPI naïve subjects, 75% remain alive at a median follow-up of 27 months. As a result, median OS has not yet been reached. Historically median OS for similar patients with platinum experienced CPI naïve disease is 7-11 months.

Objective response rate (ORR) in CPI refractory patients who received the optimal dose of the triple combination is 63% (5/8). In current approaches ORR is reported to be less than 10%.

ORR in CPI naïve patients with the triple combination is 88%. In current approaches ORR is reported to be less than 25% with FDA-approved CPIs in HPV-associated cancers.

Safety data have not changed since October’s update. 48% (24/50) of patients experienced Grade 3 (moderate) treatment-related adverse events (AEs), and 4% (2/50) patients experienced Grade 4 (severe) AEs, compared with approximately 70% of patients receiving the combination of CPIs and chemotherapy reporting Grade 3 and higher treatment-related AEs.

"The expanded data continue to demonstrate the durability and tolerability of the PDS0101-based triple combination therapy in advanced HPV-positive cancers, an extremely challenging population of refractory and previously untreatable HPV-positive patients," stated Dr. Frank Bedu-Addo, President and Chief Executive Officer of PDS Biotech. "We are pleased to see the continued consistency in the data with each update and we look forward to meeting with the FDA to discuss the registrational pathway."

Both M9241 and bintrafusp alfa are owned by Merck KGaA, Darmstadt, Germany, and its affiliates.