On April 26, 2023 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a Cayman incorporated biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported financial results for the year ended December 31, 2022, and provided an update on key highlights for 2022 (Press release, CASI Pharmaceuticals, APR 26, 2023, View Source [SID1234630571]).

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Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, commented, "We are pleased to report $10.2 million in EVOMELA revenues for the fourth quarter, $38 million for the full-year 2022. We have achieved our goal for our full-year 2022 revenue growth to exceed 2021 revenues, with a 26.7% increase in year on year sales revenue. Our team in China delivered strong performance despite the adverse impact of COVID-19 including of a Q3-4 rapid national spread of the pandemic and lockdowns in several major cities in China. Through the efforts of the CASI team and our commercial group of hematology sales and medical marketing specialists in China, we have built a strong foundation for our commercial franchise. We plan to continue building our commercial franchise throughout 2023 and beyond."

Dr. He continued, "Advancement, development, and commercialization of the portfolio remains our strategic focus. 2022 marks a major milestone for CASI and our partner Juventas; the CNCT-19 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, as we continue the development of the regulatory framework for BI-1206 in China. We completed dosing the first patient in the BI-1206 phase I trial in China. CB-5339 also received Clinical Trial Application approval from the NMPA in January 2023. The Phase 1 dose escalation and expansion study of CID-103, in patients with previously treated, relapsed or refractory multiple myeloma is closed to further accrual in France and the UK. We plan to build on the momentum to drive our portfolio forward by executing on several milestones in the quarters ahead."

Key Highlights for 2022

EVOMELA (melphalan for injection)

Prior to EVOMELA’s entry into the Chinese market, an average of 800 stem cell transplants per year were conducted in the multiple myeloma (MM) treatment setting. Following EVOMELA’s launch in August of 2019, CASI worked closely with KOLs to drive market awareness and expedite adoption in the Chinese market. In 2022, nearly 10,000 patients were treated with EVOMELA. CASI continues to pursue a similar strategy with respect to marketing efforts and physician visits to further the adoption of stem cell transplantation as a standard of care in the MM treatment setting and will continue working to address the persistent high unmet need in this patient population.

CNCT19 (CD19 targeted CAR-T)

Our partner, Juventas Cell Therapy Ltd ("Juventas"), continues the development of CNCT19, an autologous CD19 CAR-T investigative product for which CASI has co-commercial and profit-sharing rights. CNCT19 is being developed as a potential treatment for patients with hematological malignancies which express CD19 including, B-cell acute lymphoblastic leukemia ("B-ALL") and B-cell non-Hodgkin lymphoma ("B-NHL"). The Phase 1 studies in B-ALL and B-NHL in China have been completed by Juventas. The Phase 2 B-ALL and B-NHL registration studies are both currently enrolling. In December 2020, CNCT19 received Breakthrough Therapy Designation based on initial data from the ongoing single-arm, open-label, non-randomized, dose-escalation, Phase 1 study designed to determine the safety and efficacy of CNCT19 in B-ALL. Since then, the National Medical Products Administration (NMPA) granted CTA approval for CNCT19 in two indications (relapsed/refractory B-All and B-NHL) in Nov. 2019. Additionally, earlier this year, the U.S. Food and Drug Administration (FDA) granted Orphan Drug Designation (ODD) to Juventas, for CNCT19, for the treatment of patients with Acute Lymphoblastic Leukemia (ALL). Currently, there are no domestically developed CD-19 CAR-T therapy marketed in China. CASI intends for CNCT (CD19 CAR-T) to be locally developed and manufactured to be more affordable and widely accessible to patients. Dec. 15, 2022, China National Medical Products Administration (NMPA) has accepted the new drug application (NDA) from Juventas Cell Therapy, Ltd., (Juventas) for CNCT19.

CB-5339 (VCP/p97 inhibitor)

CB-5339 CTA application for the Multiple Myeloma indication is in preparation after receiving an acceptance letter for the CB-5339 IND package from the China Center of Drug Evaluation. Cleave Therapeutics is responsible for the ex-China development of CB-5339, an oral second-generation, small molecule VCP/p97 inhibitor, and is evaluating the molecule in a Phase 1 clinical trial in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS).

BI-1206 (Anti-FcyRIIB antibody)

Along with our partner, BioInvent, we continue to progress the development and regulatory framework for BI-1206 in China. The National Medical Products Administration (NMPA) granted BI-1206 Clinical Trial Application (CTA) approval in December 2021. EC approval from a leading investigational site was granted in January 2022. BI-1206 is currently being investigated in two Phase 1/2 trials. One is evaluating the BI-1206 combination with rituximab for the treatment of non-Hodgkin lymphoma, which includes patients with FL, MCL and marginal zone lymphoma (MZL) who have relapsed or are refractory to rituximab. A second Phase 1/2 trial is investigating BI-1206 in combination with anti-PD1 therapy Keytruda (pembrolizumab) in solid tumors. Earlier this year the U.S. FDA granted orphan drug designation, for BI-1206, for the treatment of follicular lymphoma (FL), the most common form of slow-growing non-Hodgkin lymphoma (NHL).

CID-103 (Anti-CD38 Mab)

CID-103 is a fully human IgG1 anti-CD38 monoclonal antibody recognizing a unique epitope that has demonstrated encouraging preclinical efficacy and safety profile compared to other anti-CD38 monoclonal antibodies. CASI maintains exclusive global rights and is developing CID-103 for the treatment of patients with multiple myeloma. The Phase 1 dose escalation and expansion study of CID-103 in patients with previously treated relapsed or refractory multiple myeloma is closed to further accrual in France and the UK. Future multiple myeloma development activities will be focused on China. In May 2022, CASI entered into a sublicense agreement with Precision Autoimmune Therapeutics, who will carry out the development activities for the autoimmune indications for CID-103.

Fourth Quarter & Full-Year 2022 Financial Results

Revenues mainly consist of product sales of EVOMELA. Total revenue was $43.1 million for the year ended December 31, 2022, including EVOMELA sales of $38.0 million, compared to $30 million for the year ended December 31, 2021.
Costs of revenues were $15.8 million for the year ended December 31, 2022 compared to $12.6 million for the year ended December 31, 2021.
General and administrative expenses for the year ended December 31, 2022 were $23.4 million, compared with $23.8 million for the year ended December 31, 2021.
Selling and marketing expenses for the year ended December 31, 2022, were $14.3 million, compared with $14.7 million for the year ended December 31, 2021.
Research and development expenses for the year ended December 31, 2022 were $16.0 million, compared with $14.4 million for the year ended December 31, 2021.
Net loss for the year ended December 31, 2022 was $40.3 million compared to $35.8 million for the year ended December 31, 2021. As of December 31, 2022, CASI had cash and cash equivalents of $47.1 million compared to $38.7 million as of December 31, 2021.

Further information regarding the Company, including its Annual Report on Form 20-F for the year ended December 31, 2022, can be found at www.casipharmaceuticals.com.

On April 26, 2023 Compugen Ltd. (Nasdaq: CGEN) (TASE:CGEN) a clinical-stage cancer immunotherapy company and a pioneer in computational target discovery, reported that it will present pre-clinical data on its lead pre-clinical asset COM503 in an oral presentation at the 20th CIMT (Free CIMT Whitepaper) annual meeting, Europe’s Cancer Immunotherapy Meeting, on May 3-5, 2023, in Mainz, Germany (Press release, Compugen, APR 26, 2023, https://www.prnewswire.com/news-releases/compugen-to-present-data-on-its-lead-pre-clinical-asset-com503-in-an-oral-presentation-at-the-20th-cimt-annual-meeting-301805591.html [SID1234630570]).

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"We are very excited to be presenting our pre-clinical data on COM503, a novel approach to harness cytokine biology to treat cancer, discovered using our proprietary computational discovery platform," said Eran Ophir, Ph.D., SVP Research and Drug Discovery at Compugen. "The inflammasome derived cytokine IL-18 is abundantly expressed in the tumor microenvironment (TME) but is blocked naturally by IL-18 binding protein, thereby preventing IL-18 from activating NK and T cells and its resulting anti-tumor activity. During our oral presentation at CIMT (Free CIMT Whitepaper) we will present pre-clinical data showing that COM503 binds with high affinity to IL-18 binding protein, freeing IL-18 and restoring NK and T cell activity. We will also show that blocking IL-18 binding protein prevents tumor growth and releases IL-18 to activate immunity in the TME without affecting peripheral immunity in murine tumor models. This is a differentiated approach to harness cytokine biology for cancer therapeutics. IL-18 is one of the rare cytokines which is naturally blocked by an endogenous binding protein, presenting a potentially unique opportunity to use a blocking antibody to release IL-18 at the site where it is needed to fight cancer, thereby potentially overcoming the limitations of systemically administered cytokines."

Presentation details:

Abstract number: 213
Title: Unleashing natural IL18 activity using an anti-IL18BP blocker antibody induces potent immune stimulation and anti-tumor activity
First Author: Menachem, A
Poster presentation: Thursday, May 4, 2023
Oral presentation: Friday, May 5, 2023
Presenter: Ophir, E

The abstract is available on the publication section of Compugen’s website at www.cgen.com. The poster and presentation will be made available on the publication section of Compugen’s website at www.cgen.com following presentation.

On April 26, 2023 Avenge Bio, Inc. ("Avenge"), a clinical stage, oncology-focused biotechnology company developing the LOCOcyte Immunotherapy platform for the precision administration of potent immune effector molecules to treat solid tumors, reported the successful completion of the first dose cohort in a Phase 1/2 clinical trial of AVB-001 in patients with refractory ovarian cancer (Press release, Avenge Bio, APR 26, 2023, View Source [SID1234630569]).

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The dose escalation trial evaluates the safety and tolerability, as well as preliminary efficacy, of AVB-001 administered intraperitoneally across a series of ascending dose-level cohorts. In the first cohort, the administration of AVB-001 has been well tolerated. No dose-limiting toxicities, on-target or off-target toxicities, or other unexpected events were observed. As such, investigators have initiated dosing in the second dose level cohort.

"We are pleased to complete the first dose cohort in this Phase 1/2 clinical trial. Although early, we are encouraged by the initial observations in this first dose level indicating the potential for this allogeneic cell-based immunotherapy. We look forward to announcing additional data on this program in the second half of 2023," said Claudio Dansky Ullmann, MD, Chief Medical Officer of Avenge Bio.

The Phase 1/2 clinical trial is a first-in-human, single-arm, open-label, dose-escalation and expansion study (NCT05538624) designed to evaluate the safety, tolerability, pharmacokinetics, pharmacodynamics, and preliminary antitumor activity of AVB-001 delivered intraperitoneally (IP) to patients with high grade serous adenocarcinoma of the ovary, primary peritoneum, or fallopian tube.

In addition to advancing the lead clinical trial in ovarian cancer, Avenge is also developing programs for additional conditions of high unmet needs in other peritoneal malignancies, and pleural cancers such as malignant pleural mesothelioma.

About LOCOcyte Platform

Our LOCOcyte allogeneic cell-based immunotherapy platform enables potent localized modulation of the immune system which also precipitates a systemic immune response, allowing us to treat previously intractable cancers. The technology leverages three unique advantages:

Potent immune effector molecules are generated by synthetically engineering allogeneic cells creating a ready-to-use therapy,

Therapy is localized in proximity to the primary tumor site and generates innate and adaptive immune response, and

The immunomodulator trains the patient’s immune system generating a robust immune response that seeks and eradicates distal metastasis without systemic toxicity.

On April 26, 2023 TC BioPharm (Holdings) PLC ("TC BioPharm" or the "Company") (NASDAQ: TCBP) a clinical stage biotechnology company reported a fundamental shift in the ongoing development and manufacture of its lead product TCB-008, an allogeneic gamma-delta T cell therapies for cancer, and the Company’s CAR modified gamma-delta T cell (Press release, TC Biopharm, APR 26, 2023, View Source [SID1234630568]).

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Following an analysis of current and emerging trends in the cell therapy landscape, the Company intends to pursue exclusive partnerships for test research and combination trial with complimentary therapies using allogeneic gamma delta t-cells, subvariant 2 ("GDT v2s"), the Company’s lead asset TCB-008. The Company’s current plans around the advancement of TCB-008 as a monotherapy for Acute Myeloid Leukemia and the ability to manufacture the product to GMP standards will continue with the FDA IND filing in Q3 of 2023 will continue as scheduled and as the final monotherapy trial for TCB-008.

A collaborative integration of TC BioPharm’s primary asset, TCB-008, will further enable the Company to expand operations into CAR Therapy with a focus on binder B7H3 for the treatment of ovarian cancer. The shift should enable the company to maximize the potential of its therapies and increase the value of the assets and the company. At present the Company has executed three research collaborations around TCB-008 in combination with complimentary approaches and expects to finalize a partnership from one of these collaborations in 2023 for trial in 2024.

"Our 2023 plan is rooted in enabling research teams to work together on preclinical and clinical studies to evaluate the potential for gamma-delta T cell therapies and better understand the behaviors of these cells in patients," said Bryan Kobel, CEO of TC BioPharm. "TCB-008 is an extremely promising therapeutic in cancer, and given its versatility, its mechanism of action, and the numerous arenas where other companies are attempting to activate, interact with or draw gamma delta t-cells to tumors, we believe TCB-008 will be a best-in-class asset for these companies to partner with in the future. This will give TCBP multiple shots on goal and maintain our leadership position as the de facto partner for companies looking to explore gamma delta t-cells sub variant 2 in combination with their asset. Our approach is already beginning to show signs of success, with multiple research collaborations and increasing interest from other potential strategic partners. In today’s capital markets environment, we firmly believe partnering will have an increased importance for development stage biotech companies and with TCB-008 as a best-in-class asset, we hope to leverage our success into non-dilutive capital and expanded data sets with partners. Having just completed a capital raise on the heels of extending our cash runway via corporate restructuring, I am confident we are better positioned for long-term growth."

TCBP will increase the focus on the CAR program, looking to shorten the timeline to completing a pre-clinical package and pursuing a US Phase I trial. The current target indication will be Ovarian cancer, with more than 313,000 new cases of ovarian cancer in 2020. The Five year Relative Survival rate is 49.7%

This approach should drive an increase in the company’s patent portfolio and IP, increasing the value of the Company in a potential acquisition scenario as well as increasing the competitive advantage of the Company with IP and patent protection.

Clinical development to-date has enabled the company to pivot to logical, more agile clinical strategy towards U.S. FDA trials for TCB-008 for AML. Completing dosing of the safety cohort was another step in TCBP’s efforts and firmly plant TCBP as the leader in the allogeneic gamma delta space.

TCBP will review external assets with pre-clinical packages/early data Cell therapies across the spectrum including TCR/CAR/Other immunotherapy opportunities and other areas where TCB-008 might be effective. Potential near-term collaborations could include fungal infection therapies in Rare Disease indications and combination research to assist private companies seeking capital where it can leverage equity capital to stockpile assets.

"Prioritizing partnerships as we head towards US trials realigns us with our long-term goal of becoming a leading commercial stage company," Kobel said. "These partnerships will expand the data set on TCB-008, give us more knowledge around both our product and GDTs and provide investors with multiple shots on goal and additional inflection points at a lower cash expenditure. We firmly believe in the potential of TCB-008 and we continue to position the Company to be more economically efficient."

On April 26, 2023 I-Mab (the "Company") (Nasdaq: IMAB), a clinical-stage biopharmaceutical company committed to the discovery, development, and commercialization of novel biologics, reported that a poster featuring the latest clinical data of uliledlimab, the Company’s proprietary and highly differentiated CD73 antibody, in combination with PD-1 therapy in non-small-cell lung cancer (NSCLC), will be presented at the 2023 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting, taking place June 2-6 in Chicago, Illinois (Press release, I-Mab Biopharma, APR 26, 2023, View Source [SID1234630567]).

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Presentation details:

Abstract Title:

Uliledlimab and Toripalimab Combination Therapy in Treatment Naïve Advanced
NSCLC: Phase 1b/2 Clinical Trial Results Using CD73 as a Potential Predictive Biomarker

Abstract Number:

2570

Presenting Author:

Prof. Qing Zhou, Guangdong Provincial People’s Hospital

Session:

Developmental Therapeutics – Immunotherapy

Location:

Hall A, McCormick Place Convention Center, Chicago, Illinois

Presentation Date/Time:

June 3, 2023, 8:00 am – 11:00 am E.T.

About Uliledlimab

Uliledlimab (also known as TJD5) is a differentiated, humanized antibody against CD73, an ecto-enzyme expressed on stromal cells and tumors that converts extracellular adenosine monophosphate (AMP) to adenosine. Adenosine, in turn, binds to adenosine receptors on relevant immune cells and inhibits anti-tumor immune responses in the tumor microenvironment. Uliledlimab is expected to offer clinical benefits by suppressing tumor growth in concert with checkpoint therapies such as PD-(L)1 antibodies. Uliledlimab is effective in anti-tumor activities through a unique intra-dimer binding, leading to differentiated and favorable functional properties, as evident in preclinical studies.