On August 12, 2023 Bio-Thera Solutions, Ltd (688177:SH), a commercial-stage biopharmaceutical company developing a pipeline of innovative assets and biosimilars, reported positive Phase 1 clinical data for BAT8006 (Folate-Receptor-α-ADC) as part of a presentation of the clinical results of a Phase 1 dose escalation study evaluating the safety and efficacy of BAT8006 at the Bethune Obstetrics and Gynecology Forum in China (Press release, BioThera Solutions, AUG 12, 2023, View Source;adc-and-presents-phase-1-dose-escalation-study-at-the-bethune-obstetrics-and-gynecology-forum-301899142.html [SID1234634299]). BAT8006 is an antibody-drug conjugate (ADC) that is composed of an anti-FRα antibody and an ADC linker-payload combination that is composed of a proprietary cleavable linker that is highly systemically stable and a small molecule topoisomerase I inhibitor.

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As a co-principal investigator of the BAT8006 Phase 1 study that was performed at First Bethune Hospital of Jilin University, Professor Songling Zhang presented dose-escalation results from the study at the Bethune Obstetrics and Gynecology Forum. July 6, 2023 served as the cut-off date for the data presented at the conference. At that time twenty-nine (29) subjects with advanced solid tumors were recruited into four (4) dose cohorts (1.2 mg/kg, 1.8 mg/kg, 2.1 mg/kg and 2.4 mg/kg) in the dose-escalation study. FRα expression was not an eligibility criterion in the study and patients with multiple tumor types, such as ovarian cancer, breast cancer, non-small cell lung cancer (NSCLC) and cervical cancer, were include in the study. Ovarian cancer patients accounted for approximately 60% of the subjects in the study.

All twenty-nine (29) subjects enrolled in the study (regardless of tumor type) had at least one tumor assessment with an Overall Objective Response Rate (ORR) of 31.0% and a Disease Control Rate (DCR) of 86.2%. For the ovarian cancer subgroup fifteen (15) subjects were recruited in the 2.1 mg/kg and 2.4 mg/kg cohorts. Among the twelve (12) ovarian cancer subjects with TPS >25%, the ORR was 58.3% and the DCR was 91.7%. Based on FRα expression epidemiological studies, it is estimated that approximately 75% of ovarian cancer patients have FRα expression >25%. To be noted, most of these ovarian cancer patients had received ＞3 prior anti-tumor therapies that included bevacizumab and PARPi. In addition, two (2) subjects with non-ovarian tumors, including breast cancer and endometrial carcinoma, experienced partial responses.

BAT8006 demonstrated a manageable safety profile with the main treatment related adverse events (TRAEs) being hematological toxicity such as neutropenia, thrombocytopenia, anemia, and gastrointestinal toxicity, including nausea and vomiting. Safety issues such as interstitial lung disease, ocular toxicity and severe hepatotoxicity were not observed in the study and no subjects were withdrawn from the study due to TRAEs.

BAT8006 is currently being evaluated in a Phase 1b study in China with four dose expansion cohorts. Two different patient subgroups are being examined in the Phase 1b study. One patient subgroup consists of FRα-positive ovarian cancer patients while the second patient subgroup consists of FRα-positive patients with non-ovarian cancers. Two different doses of BAT8006 are being evaluated with each patient subgroup. Bio-Thera is also working to expand the BAT8006 clinical program to the United States and Europe.

About BAT8006

FRα is a folic acid-binding protein located on cell membranes that is overexpressed in a variety of solid tumors such as ovarian, lung, breast cancer, etc., but has a limited distribution and a lower level of expression in normal human tissues. Differences in expression levels make FRα an attractive target for ADC drug development. BAT8006 was developed using Bio-Thera’s anti-FRα antibody and Bio-Thera’s proprietary ADC linker-payload combination that includes a systemically stable and cleavable linker and a small molecule topoisomerase I inhibitor. The small molecule topoisomerase I inhibitor payload carried by BAT8006 has a strong cell membrane penetration ability, so when the target cancer cells are killed, the payload can be released and further kill nearby cancer cells, producing a bystander effect which may effectively overcome the heterogeneity of the tumor.

BAT8006 is in the dose optimization and dose expansion studies. More subjects with ovarian cancer, endometrial cancer, NSCLC and breast cancers will be enrolled in the study to further evaluate the exposure-safety and exposure-efficacy response, which will support the final determination of recommended dose in Phase 2.

On August 11, 2023 Zetagen Therapeutics, a private, clinical-stage, biopharmaceutical company focused on driving breakthrough innovation in the treatment of metastatic cancers to bone and soft tissue organs as well as osteologic interventions, reported the close of $9.79 million USD Series B funding round (Press release, Zetagen Therapeutics, AUG 11, 2023, View Source [SID1234643708]).

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"We are pleased to have reached this key milestone in the advancement of Zetagen as a biopharmaceutical company," said Joe C. Loy, CEO of Zetagen Therapeutics. "We have a robust and diverse pipeline of oncologic treatments in various stages of development and this funding will accelerate each asset, as well as build out the necessary expertise required to bring these important therapies to fruition."

The Company has been issued two Breakthrough Device Designations from the U.S. Food and Drug Administration (FDA) for ZetaMet (Zeta-BC-003) and ZetaFuse and issued multiple patents from the U.S. Patent and Trademark Office (USPTO). Zetagen is funded through Series A and B rounds of funding, Phase I and II National Institute of Health (NIH) / National Cancer Institute (NCI) grants and angel investors.

The Series B financing will support the advancement of the Company’s lead pipeline asset ZetaMet (Zeta-BC-003) which is currently in Phase 2a clinical trials for the treatment of metastatic breast cancer bone lesions as part of advanced stage cancer therapy. In addition to ZetaMet (Zeta-BC-003), the funding will propel the development of ZetaMAST (Zeta-MBC-005) for the treatment of metastatic lesions to soft tissue organs, as well as other oncologic and osteologic pipeline assets. The Company will also add new talent to support these advancing programs.

"The work being done by Zetagen is aligned with our mission of supporting research which is potentially life-changing," said Nikhil Thakur, MD, Co-founder. "We believe the development of ZetaMet (Zeta-BC-003) and ZetaMAST (Zeta-MBC-005) – as well as the Company’s other clinical programs – have the potential to make a significant contribution to the treatment of late-stage cancers and more importantly, to the lives of those living with late-stage cancer."

Participating in the financing round were New York Ventures and Consolidated Capital Investments, LLC, and an amalgam of private investors.

On August 11, 2023 Zetagen Therapeutics, a private, clinical-stage, biopharmaceutical company focused on driving breakthrough innovation in the treatment of metastatic cancers to bone and soft tissue organs as well as osteologic interventions, reported the close of $9.79 million USD Series B funding round (Press release, Zetagen Therapeutics, AUG 11, 2023, View Source [SID1234634293]).

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"We are pleased to have reached this key milestone in the advancement of Zetagen as a biopharmaceutical company," said Joe C. Loy, CEO of Zetagen Therapeutics. "We have a robust and diverse pipeline of oncologic treatments in various stages of development and this funding will accelerate each asset, as well as build out the necessary expertise required to bring these important therapies to fruition."

The Company has been issued two Breakthrough Device Designations from the U.S. Food and Drug Administration (FDA) for ZetaMet and ZetaFuse and issued multiple patents from the U.S. Patent and Trademark Office (USPTO). Zetagen is funded through Series A and B rounds of funding, Phase I and II National Institute of Health (NIH) / National Cancer Institute (NCI) grants and angel investors.

The Series B financing will support the advancement of the Company’s lead pipeline asset ZetaMet which is currently in Phase 2a clinical trials for the treatment of metastatic breast cancer bone lesions as part of advanced stage cancer therapy. In addition to ZetaMet, the funding will propel the development of ZetaMAST for the treatment of metastatic lesions to soft tissue organs, as well as other oncologic and osteologic pipeline assets. The Company will also add new talent to support these advancing programs.

"The work being done by Zetagen is aligned with our mission of supporting research which is potentially life-changing," said Nikhil Thakur, MD, Co-founder. "We believe the development of ZetaMet and ZetaMAST– as well as the Company’s other clinical programs – have the potential to make a significant contribution to the treatment of late-stage cancers and more importantly, to the lives of those living with late-stage cancer."

Participating in the financing round were New York Ventures and Consolidated Capital Investments, LLC, and an amalgam of private investors.

About ZetaMet and ZetaMAST

ZetaMet is a synthetic, small-molecule, inductive biologic technology being developed to target and resolve metastatic bone lesions while inhibiting future tumor growth and regenerating bone. The small molecule has been approved by the FDA since 1971. Zetagen scientists have discovered an entirely new pathway for this established molecule which, if proven successful in human clinical trials, could create a new treatment paradigm for patients living with metastatic bone lesions.

ZetaMAST is designed as a single, percutaneous, intra-tumoral injection, utilizing a proprietary hydrogel carrier containing a precise, tailored concentration of our small molecule. Preclinical studies have demonstrated ZetaMAST may cease lesion growth, with the goal of enabling improved quality of life as part of a cancer treatment program, including longevity.

On August 11, 2023 CEL-SCI Corporation (NYSE American: CVM) reported financial results for the quarter ended June 30, 2023, as well as key clinical and corporate developments (Press release, Cel-Sci, AUG 11, 2023, View Source [SID1234634292]).

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Clinical and Corporate Developments this quarter include:

CEL-SCI had a productive pre-submission meeting with Canada’s regulator, Health Canada, to determine the best regulatory path toward market approval. Health Canada advised CEL-SCI to request advance consideration for approval under a Notice of Compliance with Conditions (NOCC) policy which facilitates earlier access for physicians and patients to promising new drugs for patients suffering from serious, life-threatening, or severely debilitating diseases. CEL-SCI is preparing an application for the NOCC approval as suggested and plans to file it by early next year. If Health Canada grants the NOCC, then it is possible that CEL-SCI could begin commercialization in 2024.
Europe is a priority market for CEL-SCI, as Europe has more than twice the number of head and neck cancer cases diagnosed each year as compared to the United States (US). CEL-SCI is seeking conditional marketing authorization for Multikine in Europe. Based on the published guidelines, the Company believes it meets the requirements for conditional approval of Multikine and has plans for meetings with regulators in the fall.

CEL-SCI had a collaborative and positive meeting with the U.S. Food and Drug Administration (FDA). The FDA acknowledged the great need for improved treatments for head and neck cancer, particularly the locally advanced oral cavity that CEL-SCI is targeting and is open to close collaboration with CEL-SCI to help demonstrate that Multikine could fill this need. Preliminary feedback from the FDA included that the selection criteria developed by CEL-SCI from its Phase 3 data could be used to determine which locally advanced oral cavity cancer patients might benefit from Multikine treatment. CEL-SCI is preparing additional information about its Multikine development plan for a follow-up meeting with the FDA based on this feedback. A confirmatory clinical trial will be conducted based on the agreed upon selection criteria for patients that will be treated with Multikine as assessed by methods including PET-CT/MRI screening. CEL-SCI will collaborate closely with the FDA on the design of a clinical protocol that will allow the Company to generate, as expeditiously as possible, the confirmatory data they will require for approval of Multikine in the US. Importantly, this study is also expected to have intermediate endpoints during study enrollment for potential accelerated approval based on interim results.

Potentially quite impactful PD-L1 biomarker data from CEL-SCI’s Phase 3 study was presented at the American Head and Neck Cancer Society’s 11th Annual International Conference on Head and Neck Cancer titled "Tumor cell PD-L1 biomarker confirms Leukocyte Interleukin Injection (LI) treatment (Tx) survival outcome advantage in naïve locally advanced primary head & neck squamous cell carcinoma (SCCHN), the IT-MATTERS Study". The data demonstrated that the tumors of patients who responded best to Multikine in the Phase 3 study had low levels of the PD-L1 biomarker. Currently approved checkpoint inhibitors (Keytruda and Opdivo) which are indicated for treatment of unresectable or recurrent or metastatic head and neck cancer are known to work best in patients whose tumors express high PD-L1 levels and are less likely to work in patients whose tumors express low PD-L1. These contrasting PD-L1 data observed in patients responsive to Multikine vs those observed in patients treated with checkpoint inhibitors are very significant.

The data are expected to be helpful in key ways:

Supporting marketing approval by using PD-L1 as a marker to select patients who are most likely to benefit from Multikine

Positioning Multikine as a combination therapy with checkpoint inhibitors

Combination studies may be conducted in partnership with a larger pharma company that has an approved PD-L1 checkpoint inhibitor

Should combination studies with checkpoint inhibitors be successful not only would patients benefit substantially, but the financial benefits to CEL-SCI could be very large
The global PD-L1/PD-1 therapeutics market was valued at $34.8 billion in 2022
Data presented at the European Society for Radiotherapy and Oncology (ESTRO) 2023 Congress in May confirmed that Multikine significantly prolonged overall survival in head and neck cancer. The presentation titled "Histopathology population (HPP) confirms Multikine* [Leukocyte Interleukin Injection (LI)] treatment (Tx) outcome in naïve locally advanced primary head & neck squamous cell carcinoma SCCHN)" provided findings from a histopathology and tumor biomarker analysis of its Phase 3 study that confirmed Multikine-treated subjects had improved 5-year survival, showed improved progression free survival and improved local regional control, and a significantly lowered death rate compared to control subjects who received standard of care alone.
"The growing body of data on the efficacy of Multikine presented at peer-reviewed conferences is highly encouraging as we move forward with regulatory meetings and submissions with the world’s most respected regulators in the world in addition to FDA. The data has also allowed us to define very well the population of patients who have the greatest benefit from Multikine treatment. This is a crucial part of our approval strategy in this unmet medical need as we are aiming for conditional/accelerated approval pathways with multiple regulators," stated CEL-SCI CEO, Geert Kersten. "We are also excited about the new prospect of developing Multikine in conjunction with a pharma partner as a combination therapy with a checkpoint inhibitor to boost patient outcomes.

Financial Results

CEL-SCI reported a loss per share for the quarter ending June 30, 2023 of $0.19 versus a loss of $0.23 for the quarter of June 30, 2022.

On August 11, 2023 HebeCell Corp (HebeCell), a biotechnology company developing ProtoNK cell therapeutics based on its proprietary 3D PSC-NK manufacture platform, and Logomix Inc (Logomix), a Tokyo-based synthetic biology company, with a platform technology to engineer-in complex genomic messages of ~Mb-scale into human cells, reported a strategic partnership to research and develop gene-edited NK cells and discover genetic modifications that can create next generation designer NK cells (Press release, HebeCell, AUG 11, 2023, View Source [SID1234634291]).

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Under the agreement, the companies enter a strategic partnership where Logomix first provides genome editing capabilities to HebeCell for development of next generation designer protoNK cells. The combination of PSC-based platform with genome editing capabilities will significantly broaden and accelerate new development for screening de novo genetic modifications that enhance the persistence and efficacy of protoNK cells against cancers.

"We know our protoNK cells are strong cytotoxic cells. Our goal is to make PSC-derived protoNK more efficacious in vivo. This collaboration with Logomix will give us a very powerful toolbox to achieve that goal," said John Lu, Ph.D., Chief Executive Officer at HebeCell Corp. "This collaboration expands our efforts to develop unique NK cell therapies, and has the potential to create something new and never seen before in the NK therapeutic field."

"Logomix’s proprietary technology, Geno-Writing is a genome-writing platform that grants cell therapy developers unprecedented freedom to screen, design and write-in large-scale genomic alterations in human cells. Because our technology allows Mb-scale and scarless modifications, applied to any sequences at either or both alleles, it is an ideal tool to create source of allogeneic cell therapy with desired gene edits," said Taiki Ishikura, CEO at Logomix Inc. "This collaboration with HebeCell demonstrates how our genome engineering technologies can be applied to the field of immuno-oncology and develop the next generation of cell therapy modalities more efficacious and safe to cancer patients."