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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

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]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

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."

On August 11, 2023 Asieris Pharmaceuticals (688176), a global biopharma company specializing in discovering, developing and commercializing innovative drugs for the treatment of genitourinary tumors and other related diseases, reported that the Phase III bridging clinical trial of Hexvix for bladder cancer diagnosis met the primary endpoint (Press release, Asieris Pharmaceuticals, AUG 11, 2023, View Source [SID1234634290]). The results of the study will be submitted to the Chinese National Medical Products Administration (NMPA) for a new drug application (NDA) in the coming months.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

The study is a prospective, self-controlled, multicenter Phase Ⅲ trial aimed at investigating the additional detection rate and safety of Hexvix and blue light cystoscopy (BLC) versus white light cystoscopy in patients with non-muscle invasive bladder cancer (NMIBC) including tumors with stage carcinoma in situ (CIS), Ta, and T1.

A total of 158 patients were enrolled in this study. The completed statistical analysis results indicate that the study has been successfully conducted and has achieved the primary endpoint. The results of the study will be presented at upcoming academic conferences.

In January 2021, Asieris entered into a license agreement with Photocure ASA (Photocure, OSE:PHO), a bladder cancer specialty company based in Oslo, Norway, to obtain the exclusive registration and commercialization rights of Hexvix in mainland China and Taiwan.

"The clinical data of Hexvix further confirms its outstanding clinical performance. We sincerely thank all the researchers and participants for their dedication and efforts in advancing this clinical study." said Dr. Linda Wu, Chief Development Officer of Asieris, "This marks a significant milestone in our company’s comprehensive efforts to create an integrated diagnostic and therapeutic solution in the field of bladder cancer. We will continue to vigorously advance the follow-up work, aiming to achieve commercialization and benefit more bladder cancer patients as soon as possible."

On August 11, 2023 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the quarter ended June 30, 2023 (Press release, CASI Pharmaceuticals, AUG 11, 2023, View Source [SID1234634289]).

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We are pleased to report $9.8 million sales revenue for the second quarter of 2023. This is a 15% increase compared to the same period last year and an 18% of increase from the first quarter of 2023."

Dr. He continued, "Advancement, development, and commercialization of the portfolio remains our strategic focus. We have achieved a major milestone with our partner Juventas Biotechnology (Tianjin) Co., Ltd. ("Juventas") on the CNCT-19 CAR-T Cell therapy. CNCT-19’s New Drug Application (NDA) was accepted by National Medical Products Administration (NMPA) in December 2022. We are now diligently preparing for the anticipated CNCT-19 launch in China. We are advancing the clinical development of BI-1206 in combination with rituximab and presently undertaking the enrollment of patients for the second cohort of the phase I trial in China. We are transitioning the development of CID-103 into China for the malignant hematology indications. In addition, we acquired global rights for CB-5339 from Cleave Therapeutics in July. On August 1st, we announced the transfer of license of Folotyn in China from Mundipharma International Corporation Limited, Mundipharma Medical Company, and Acrotech Biopharma Inc.. We continue to advance our portfolio by executing on several milestones as well as bringing in new potential opportunities for synergy in the quarters ahead."

Second Quarter 2023 Financial Highlights

Revenue was $9.8 million for the three months ended June 30, 2023, compared to $8.6 million for the three months ended June 30, 2022.
Costs of revenues were $4.0 million for the three months ended June 30, 2023, compared to $3.6 million for the three months ended June 30, 2022.
Research and development expenses for the three months ended June 30, 2023 were $2.6 million, compared with $3.9 million for the three months ended June 30, 2022.
General and administrative expenses for the three months ended June 30, 2023 were $7.7 million, compared with $5.5 million for the three months ended June 30, 2022. The increase was mainly attributable to the $2.2 million share-based compensation expenses allocated to general and administrative expenses related to the Company’s modification to certain share options granted under the 2011 Long-term Incentive Plan and 2021 Long-term Incentive Plan.
Selling and marketing expenses for the three months ended June 30, 2023 were $4.8 million, compared with $3.4 million for the three months ended June 30, 2022. The increase was mainly attributable to travel and conference expenses incurred for our commercial activities.
As of June 30, 2023, CASI had cash, cash equivalents and short-term investments of $36.8 million.

On August 11, 2023 Zumutor Biologics Inc "Zumutor", a Boston based biopharmaceutical company focused on developing unique novel therapeutics in immuno-oncology (I-O) reported that the US Food and Drug Administration (FDA) has granted the company’s Investigational New Drug (IND) application for the novel drug ZM008 to initiate a Phase 1, first-in-human, clinical study for the treatment of multiple solid cancers (Press release, Zumutor Biologics, AUG 11, 2023, View Source [SID1234634288]). ZM008 is a human IgG1 monoclonal antibody against LLT1 (CLEC2D), which disrupts the interaction of LLT1-CD161 between human immune cells and Tumor cells resulting in antitumor effects of ZM008 in monotherapy. The disruption of LLT1-CD161 interaction with ZM008 leads to reversal from "cold" or less immune responsive Tumor Microenvironment (TME) to "hot" highly immune responsive TME – the Holy Grail of I-O mechanisms.

Schedule your 30 min Free 1stOncology Demo!
Discover why more than 1,500 members use 1stOncology™ to excel in:

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

                  Schedule Your 30 min Free Demo!

FDA also accepted the "safety first" staggered parallel clinical design of ZM008 combination arm with Pembrolizumab to benefit a wider range of patients whose immune response could be boosted with ZM008 followed by Pembrolizumab treatment to obtain favourable disease responses.

"The U.S. Food and Drug Administration’s approval of ZM008 IND application is an important milestone for Zumutor and a clear recognition of the intriguing preclinical data supporting novel/non overlapping mode of actions, generated thus far. Our work to translate the promise of Zumutor’s INABLRä platform into improved and more potent human antibody products for patients in need will delve further into unique immune pathways involving NK cells and other innate immune compartments," said Dr Maloy Ghosh, PhD, Chief Scientific Officer, Zumutor Biologics. "We are now focusing on clinical execution of ZM008, closely working with oncologists for Phase I trial at NEXT Oncology, San Antonio and other sites in US, targeting first patient recruitment soon. We strongly believe this unique product will influence immune pathways involving both NK cell and T cell compartments with amazing potential for combination with Anti PD1 antibody like Pembrolizumab to benefit patients with advanced solid tumors".

"We believe that newer therapies like ZM008 will usher in options for patients and translate to better clinical outcomes. We anticipate start of Phase 1 studies in Q4 2023," said Kavitha Iyer Rodrigues, Founder/ CEO of Zumutor

ZM008 Phase 1 study design will provide important information about the safety, tolerability, pharmacokinetics, pharmacodynamics, preliminary efficacy of monotherapy treatments as well as Recommended Phase 2 Dose (RP2D) of this novel drug in patients with solid cancers who are not benefited from existing ‘standard of care’.

About ZM008:

ZM008 is a full length human IgG1 monoclonal antibody against LLT1 antigen on surface of various tumor cells. LLT1 – CD161 interaction leads to immune repressive condition in the TME allowing tumor cells to bypass the human immune system. ZM008 disrupts the LLT1-CD161 interaction and release the break from the immune cells – the activated immune cells then recognize and kill the tumor cells. The antitumor effects of ZM008 was demonstrated in elegant assays performed in humanized mice xenograft model and an ex vivo platform with biopsies from lung cancer and bladder cancer patients in presence of autologous PBMC.