CASI PHARMACEUTICALS ANNOUNCES FOURTH QUARTER AND FULL-YEAR 2023 BUSINESS AND FINANCIAL RESULTS

On March 28, 2024 CASI Pharmaceuticals, Inc. (Nasdaq: CASI), a Cayman incorporated biopharmaceutical company focused on developing and commercializing innovative therapeutics and pharmaceutical products, reported business and financial results for the year ended December 31, 2023, and provided an update on key highlights for 2023 (Press release, CASI Pharmaceuticals, MAR 28, 2024, View Source [SID1234641592]).

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CASI reported fourth quarter 2023 revenue of $6.9 million for EVOMELA, 33% lower than the same period in 2022. The 2023 full-year revenue of $34 million reflects an 11% of decrease compared to 2022. Wei-Wu He, Ph.D., CASI’s Chairman and Chief Executive Officer, said "CASI’s team has navigated our business through a challenging external environment. We have successfully brought the second commercial product FOLOTYN to China market as a treatment for patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). We will continue to strengthen the commercial franchise throughout 2024 and beyond. At the same time, our regulatory and development team made significant pipeline advancements."

Dr. He continued, "Advancement, development, and commercialization of the pipelines remain our strategic focus. 2023 marks a major milestone for CASI and our partner Juventas; Inaticabtagene Autoleucel (CNCT-19 CAR-T cell therapy) was approved by National Medical Products Administration (NMPA) in November 2023. We continue the development for BI-1206 in China, with the early clinical data from the ongoing phase I trial in China demonstrating promising preliminary clinical results for patients with relapsed/refractory non-Hodgkin lymphoma. CB-5339 received Clinical Trial Application approval from the NMPA in January 2023. We are currently preparing for CID-103 clinical study application in relapsed or refractory multiple myeloma in China. We will continue to drive our portfolio forward by executing on several milestones in the quarters ahead."

Key Highlights for 2023

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 therapeutic area experts to improve market awareness and expedite adoption in the Chinese market. In 2023, 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.

FOLOTYN (Pralatrexate)

On July 31, 2023, CASI entered into a tripartite assignment agreement with Mundipharma International Corporation Limited ("MICL"), Mundipharma Medical Company (MMCo), and Acrotech Biopharma Inc. (Acrotech) for the commercialization of FOLOTYN (Pralatrexate) in the People’s Republic of China. FOLOTYN (Pralatrexate) is a dihydrofolate reductase inhibitor indicated for the treatment of patients with relapsed or refractory peripheral T-cell lymphoma (PTCL). This product was approved by both the FDA and China’s NMPA for PTCL. CASI announced the first patient was dosed with FOLOTYN in China on February 15, 2024. CASI will continue to spend time, resources, and efforts on the commercialization of FOLOTYN in China.

BI-1206 (Anti-FcyRIIB antibody)

Along with CASI’s partner, BioInvent, CASI continues to progress the development and regulatory framework for BI-1206 in China. The 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 as combination agent 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. In 2022, 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).

Inaticabtagene Autoleucel (CNCT-19)

On November 8, 2023, The China National Medical Products Administration (NMPA) has granted market approval for Juventas’ Inaticabtagene Autoleucel (CNCT-19) for the treatment of relapsed and refractory B-cell acute lymphoblastic leukemia (r/r B-ALL) in China.

Inaticabtagene Autoleucel is a CD19 CAR-T cell therapy product comprised of a unique CD19 scFv(HI19a)structure and utilizes leading CMC manufacturing techniques. Inaticabtagene Autoleucel has demonstrated a high level of efficacy, with durable remissions, and substantially improved safety profile with reduced CAR-T related toxicities in the pivotal clinical study for the treatment of adults with r/r B-ALL.

CASI is currently involved in arbitration proceedings against Juventas in relation to Juventas’s purported termination of the CNCT-19 Agreements, between the Company and Juventas with respect to the commercialization of Juventas’ cell therapy, Inaticabtagene Autoleucel (CNCT-19). On March 2, 2024, CASI received a notice from Juventas, which purported to terminate the CNCT-19 Agreements. CASI responded to Juventas’s purported termination notice, noting that Juventas was not entitled to unilaterally terminate the CNCT-19 Agreements and further demanding that Juventas cease any conduct that may constitute further breach of the CNCT-19 Agreements and execute a written undertaking regarding compliance with the CNCT-19 Agreements by March 13, 2024. Juventas did not comply with CASI’s demands. On March 20, 2024, CASI submitted a Notice of Arbitration at the Hong Kong International Arbitration Centre ("HKIAC") against Juventas pursuant to the CNCT-19 Agreements’ dispute resolution clauses, claiming that Juventas’s purported termination was invalid and that Juventas breached the CNCT-19 Agreements and seeking, among other things, damages and injunctive reliefs. Together with the Notice of Arbitration, CASI also submitted an application for the appointment of an emergency arbitrator, seeking emergency injunctive reliefs. On the same day, Juventas also submitted a Notice of Arbitration at the HKIAC against CASI, alleging, among other things, that the CNCT-19 Agreements were validly terminated and that CASI breached the CNCT-19 Agreements. The HKIAC has appointed an emergency arbitrator in accordance with CASI’s application. The arbitration proceedings are ongoing.

CB-5339 (VCP/p97 inhibitor)

In March 2021, the Company entered into an exclusive license with Cleave Therapeutics, Inc. ("Cleave") for the development and commercialization of CB-5339, an oral novel VCP/p97 inhibitor, in both hematological malignancies and solid tumors, in Mainland China, Hong Kong, Macau and Taiwan. CB-5339 has been evaluated by Cleave in a Phase 1 clinical trial in patients with acute myeloid leukemia (AML) and myelodysplastic syndrome (MDS). Because CB-5339 has not yet reached technological feasibility and has no alternative future uses, the Company expensed the $5.5 million upfront payment as acquired in-process research and development in 2021.

On July 18, 2023, the Company entered into an assignment agreement (the "Assignment Agreement") with Cleave, pursuant to which the Company obtained the global intellectual property rights related to CB-5339. Pursuant to the Assignment Agreement and partially in exchange for the transfer of the global intellectual property rights for CB-5339 as well as all remaining CB-5339 drug substance and drug product to CASI.

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. CASI entered into a sublicense agreement with Precision Autoimmune Therapeutics, who will carry out the development activities for the autoimmune indications for CID-103.

Full-Year 2023 Financial Results

Revenues consist of product sales of EVOMELA. Revenue was $34.0 million for the year ended December 31, 2023 compared to $38 million for the year ended December 31, 2022. The decrease was mainly attributable to the launch of an undifferentiated generic formulation of melphalan for injection product by a Chinese domestic manufacture.

Costs of revenues were $13.8 million for the year ended December 31, 2023 compared to $15.8 million for the year ended December 31, 2022. Costs of revenues as a percentage of EVOMELA sales for 2023 and 2022 were 41% and 42%, respectively.

General and administrative expenses for the year ended December 31, 2023 were $25.4 million, compared with $23.4 million for the year ended December 31, 2022. The increase in general and administrative expenses was primarily attributable to incremental share-based compensation expense recognized due to the option modification in May 2023 amounted to US$2.2 million, and increased depreciation expense of US$1.2 million due to the full year depreciation expenses of CASI Wuxi’s leasehold improvement that started to depreciate in August 2022, offset by decrease of land vacancy fee of US$1.3 million in relation to the return of the Wuxi land use right.

Selling and marketing expenses for the year ended December 31, 2023, were $16.4 million, compared with $14.3 million for the year ended December 31, 2022. The increase was primarily due to increased travel and conference expenses incurred for our commercial activities after the Chinese health authority cancelled the stringent COVID-19 controlled measure in December 2022.

Research and development expenses for the year ended December 31, 2023 were $9.9 million, compared with $16.0 million for the year ended December 31, 2022. The decrease in R&D expenses is primarily due to CID-103 as we incurred less laboratory tests and decrease in the research and development expenses of generic pharmaceuticals in Wuxi manufacturing facility.

Net loss for the year ended December 31, 2023 was $26.3 million compared to $40.3 million for the year ended December 31, 2022.

As of December 31, 2023, CASI had cash, cash equivalents and short term investment of $29.1 million compared to $48.6 million as of December 31, 2022.
Further information regarding the Company, including its Annual Report on Form 20-F for the year ended December 31, 2023, can be found at www.casipharmaceuticals.com.

GenFleet and BeiGene Enter into Trial Collaboration for a Potentially First-in-class Combination Therapy to Initiate Phase Ib/II Study of GFH009 (CDK9 inhibitor) and BRUKINSA® (zanubrutinib) Treating Diffuse Large B Cell Lymphoma

On March 28, 2024 GenFleet Therapeutics, a clinical-stage biotechnology company focusing on cutting-edge therapies in oncology and immunology, reported that it has entered into a clinical trial collaboration and supply agreement with BeiGene Switzerland GmbH to start a combination study of GFH009 (CDK9 inhibitor) and BRUKINSA (zanubrutinib, BTK inhibitor) in a multicenter phase Ib/II trial treating diffuse large B cell lymphoma (DLBCL) (Press release, GenFleet Therapeutics, MAR 28, 2024, View Source [SID1234641591]). The first patient was dosed in the trial led by prominent Henan Cancer Hospital and Fudan University Shanghai Cancer Center.

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Under the terms of the agreement, GenFleet will conduct an open-label, single-arm and multi-center (10 hospitals in China) study of the combination therapy to evaluate the safety and efficacy among relapsed/refractory DLBCL patients. BeiGene will provide clinical drug supplies of BRUKINSA(zanubrutinib) for this trial. This study will be the first combination trial conducted by a Chinese biotech to combine CDK9 inhibitor and BTK inhibitor targeting DLBCL.

China’s National Cancer Center reports that around 100,000 patients are newly diagnosed non-Hodgkin’s lymphoma per year in China, with DLBCL patients accounting for 40-50% of new cases. Currently, R-CHOP (rituximab, cyclophosphamide, doxorubicin, vincristine, prednisone) is the standard of care in the first line setting for DLBCL globally, but 30-40% of DLBCL patients ultimately progressing into relapsed/refractory stage need more effective treatments.

The trials of GFH009 treating peripheral T-cell lymphoma and acute myeloid leukemia have entered into phase II stage in China and the U.S. respectively. Numerous patients achieved complete or partial response, and significant downregulation of anti-apoptotic proteins such as MYC, MCL1 were observed among patients.

Preclinical research demonstrated GFH009’s anti-proliferation effects on various tumor cell lines; the expression level of apoptosis markers including cleaved caspase-3 (CC3) and cleaved PARP increased dose-dependently with GFH009 treatment. According to academic publications, the treatment of CDK9 inhibitor in combination with BTK inhibitor resulted in accelerated induction of cleaved CC3 (the key protein in the cancer-cell killing mechanism of cytotoxic T lymphocytes).

"We are delighted to reach this agreement to move forward the innovative combinational therapy. We appreciate BeiGene’s recognition of GenFleet’s R&D capabilities and GFH009’s clinical potential. GFH009 has shown a promising activity in monotherapy trial and BRUKINSA(zanubrutinib) has been approved in scores of markets worldwide; we hope to explore more innovative therapies for relapsed/refractory DLBCL patients with our mutual efforts." stated Jiong Lan, Ph.D, Chief Executive Officer of GenFleet.

References:

1.Development and validation of a sensitive UPLC–MS/MS analytical method for GFH009 in rat plasma and its application to toxicokinetics studies, Biomedical Chromatography, 2023

2. Epidemiology of diffuse large B-cell lymphoma (DLBCL) and follicular lymphoma (FL) in the United States and Western Europe: population-level projections for 2020–2025, Leukemia & Lymphoma, 2021

3. Current status and progress of lymphoma management in China, International Journal of Hematology, 2018

About GFH009 & CDK9

As a family of serine & threonine kinases, the cyclin-dependent kinase (CDK) family plays an important role in cell cycle regulation and transcription; CDK9 activity is inversely correlated with the overall survival rate of patients with multiple tumors. Data from phase I trial and the preclinical research of GFH009 were posted at the 2002 Annual Meeting of the American Society of Hematology (ASH) (Free ASH Whitepaper). GFH009 monotherapy is well tolerated with preliminary clinical activity in patients with relapsed/ refractory lymphomas.

According to preclinical research, GFH009 reduces the expression of downstream oncogenes required for rapid cellular division and protein expression through specific, short-lived inhibition of CDK9. With more than 100 times selectivity over other CDK subtypes, this depletion via GFH009 inhibition of CDK9 likely deprives oncogene-addicted cancer cells of crucial survival signals, leading to senescence and death. GFH009 also exhibits strong anti-proliferative activities in multiple human cell lines, effectively inhibits the growth of tumor in various xenograft models and significantly improves survival of tumor bearing animals.

GFH009 has been granted with both fast track and orphan drug designation from the FDA for the treatment of adult patients with relapsed/refractory (r/r) peripheral T-cell lymphomas (PTCL) and r/r acute myeloid leukemia (AML). GenFleet received IND approval in 2020 for the GFH009 monotherapy to proceed into phase I trial treating patients with r/r hematological malignancies. In 2022, GenFleet and SELLAS Life Sciences Group (Nasdaq: SLS) entered into an exclusive license agreement across all therapeutic and diagnostic uses worldwide outside of Greater China (the Chinese mainland, Hong Kong, Macau and Taiwan).

About BRUKINSA (zanubrutinib)

BRUKINSA(zanubrutinib) is a small molecule inhibitor of BTK designed to deliver complete and sustained inhibition of the BTK protein by optimizing bioavailability, half-life, and selectivity. With differentiated pharmacokinetics compared with other approved BTK inhibitors, BRUKINSA has been demonstrated to inhibit the proliferation of malignant B cells within a number of disease-relevant tissues.

Aster Insights Announces AACR Schedule of ORIEN Presentations

On March 28, 2024 Aster Insights, the leading provider of scientific and clinical intelligence for oncology discovery, reported its schedule of research presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting in San Diego, California, April 5-10, 2024 (Press release, Aster Insights, MAR 28, 2024, View Source [SID1234641590]). The seven abstracts, which are a combination of oral and poster presentations, will be presented by researchers from institutions within the Oncology Research Information Exchange Network (ORIEN), a research consortium administered by Aster Insights. All abstracts utilize data from ORIEN and Aster Avatar, the best-in-class, deepest multimodal dataset for discovery research in oncology.

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The abstracts cover a broad range of research across several cancer types, including predictors of response to immunotherapy, the role of the tumor microbiome in patient outcomes, and the study of cancer in minority patient populations. Several of the presentations are ORIEN intermember studies developed by the consortium’s research interest groups, which are designed to increase multidisciplinary, collaborative research among premier national cancer centers. Additionally, most of the abstracts include co-investigators from Aster Insights’ scientific leadership, reflecting the unique roles of both the company and ORIEN in driving critical cancer discovery.

"There is no better demonstration of the versatility of Aster Insights and ORIEN than the selection of abstracts that will be presented at AACR (Free AACR Whitepaper) this year," said Dr. Anand Shah, CEO of Aster Insights. "The breadth of these investigations is a testament to the impact we have by bringing together the best expertise in cancer research to unlock new treatments and cures."

Official schedule of Aster Insights’ presentations at AACR (Free AACR Whitepaper):

Tarhini, A.A. Differential infiltration of key immune cell populations across malignancies varying by immunogenic potential and likelihood of response to immunotherapy (abstract 71/7)
Poster Presentation: Sunday, April 7, 2024, 1:30 PM – 5:00 PM
Section 3
Session PO.IM01.01 – Biomarkers, Immune Monitoring, and Immune Assays

Grencewicz, D. Gavage with Candida albicans leads to fungal colonization of colorectal tumors and decreased response to radiotherapy (abstract 1282)
Oral Presentation: Sunday, April 7, 2024, 4:05 PM – 4:20 PM
Room 16 – Convention Center Mezzanine Level
Session MS.TB11.01 – Microbes and Tumors: Time for Mechanisms

Obermeyer, A. Analysis of clonal heterogeneity within paired primary and metastatic tumor samples of patients with solid tumors and implications for neoantigen-based personalized cancer vaccines (abstract 3886)
Oral Presentation: Monday, April 8, 2024, 2:35 PM – 2:50 PM
Room 6 CF – Convention Center Upper Level
Session MS.CL10.01 – Application of Real-World Evidence to Cancer Care

Soupi, A.C. Genomic landscape and estimation of immune infiltration of soft tissue sarcoma histology subtypes from the ORIEN network (abstract 3928)
Oral Presentation: Monday, April 8, 2024, 2:35 PM – 2:50 PM
Room 31 – Convention Center Upper Level
Session MS.MCB08.01 – Advances in Cancer Genomics: Carcinogenesis, Tumor Evolution, and Heterogeneity

Villarreal Velazquez, E.I. Multi-omics characterization of molecular features and global-local genomic ancestry analysis of colorectal cancer in Hispanic-Latinos (abstract 3932) Oral Presentation: Monday, April 8, 2024, 3:35 PM – 3:50 PM
Room 31 – Convention Center Upper Level
Session MS.MCB08.01 – Advances in Cancer Genomics: Carcinogenesis, Tumor Evolution, and Heterogeneity

Garay Raygoza, J. Tumor gene expression patterns affecting response to BCl-2 inhibitor venetoclax in acute myeloid leukemia (abstract 5852/28)
Poster Presentation: Tuesday, April 9, 2024, 1:30 PM – 5:00 PM
Section 23
Session PO.ET03.07 – Drug Resistance 3: Regulation of Gene Expression

Chan, C.H.F. Epithelial-mesenchymal-transition gene signature changes and poor oncological outcome in Candida-positive pancreatic ductal adenocarcinoma (abstract 7615/9) Poster Presentation: Wednesday, April 10, 2024, 9:00 AM – 12:30 PM
Section 45
Session PO.CL01.13 – Predictive Biomarkers 7
"Fostering a rich culture of collaboration with multidisciplinary researchers is fundamental to the work Aster Insights has led for 10 years together with ORIEN," said Michelle Churchman, PhD, Scientific Director at Aster Insights. "This spirit of cooperation makes ORIEN truly unique in oncology, and we look forward to sharing our work with the broader cancer research community at AACR (Free AACR Whitepaper)."

Aster Insights leadership will be attending AACR (Free AACR Whitepaper). Please contact us to request a meeting.

Harbour BioMed Reports Full Year 2023 Financial Results

On March 28, 2024 Harbour BioMed ("HBM" or the "Company"; HKEX: 02142), a global biopharmaceutical company committed to the discovery, development and commercialization of novel antibody therapeutics focusing on immune-oncology and immunology, reported annual financial results of full year 2023 (Press release, Harbour BioMed, MAR 28, 2024, View Source [SID1234641589]).

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"We made progress in two significant undertakings in 2023, the third financial year since listing on the Main Board of the Stock Exchange of Hong Kong: preparing Harbour Therapeutics to operate as a faster, more focused clinical-stage next-generation therapeutics company and initiating Nona Biosciences to leverage our unique global patent-protected technology platforms to empower global therapeutic innovation. We recorded a significant increase in our revenue, demonstrating the Company’s excellent global business development capabilities," said Dr. Jingsong Wang, Founder, Chairman and CEO of Harbour BioMed. "We are navigating a fiercely competitive world that is going through rapid changes, facing human being’s fundamental quest for longevity and quality of life, and having a greater demand for biotechnological breakthroughs and innovative therapeutics. We’ve been well-positioned in the new era to achieve results that will propel the Company to new heights and create robust business value."

FULL YEAR 2023 FINANCIAL HIGHLIGHTS

Harbour BioMed recorded the profit of US$22.8 million for the year ended 31 December 2023. This is the first time that the Company has recorded a net profit on its annual financial statements. Other financial highlights include:

Revenue: The revenue for the full year 2023 is US$89.5 million, which increased significantly by US$48.8 million, or 119.9%, compared with US$40.7 million for the year ended 31 December 2022. Our revenue primarily consists of molecule license fee, research service fee and technology license fee. The increase is primarily attributed to license out and collaboration agreement with Pfizer, Cullinan Oncology and Kelun-Biotech.
Research and development costs: The research and development expenses decreased by 66.6%, from US$135.1 million in 2022 to US$45.1 million in 2023. This decrease was primarily attributable to the combined impact of (i) optimized investments in our clinical programs and our molecule assets in discovery and pre-clinical stages; and (ii) the efficient implementation of cost control measures.
Administrative expenses: The administrative expenses decreased by 28.6%, from US$27.3 million in 2022 to US$19.5 million in 2023.
ROBUST PORTFOLIO AND DIFFERENTIATED PIPELINE

Harbour Therapeutics, a sub-brand parallel to Nona Biosciences, is individually responsible for the development of the Company’s product pipeline. Focused on oncology and immunology, Harbour Therapeutics has a robust and diversified pipeline of more than ten potentially differentiated drug candidates, four of which are in clinical development stage. Batoclimab (HBM9161), porustobart (HBM4003), HBM7008 and HBM1020 are the main products.

Batoclimab, as the clinically most advanced FcRn inhibitor being developed in Greater China, has the potential to be a breakthrough treatment for a wide spectrum of autoimmune diseases in Greater China. Harbour Therapeutics completed the treatment of patients in early 2023 and announced the positive topline results of the phase III clinical trial of batoclimab for the treatment of generalized myasthenia gravis (gMG) in March, which is also the first positive pivotal trial outcome for batoclimab worldwide. This marks a major milestone as it is the Company’s first product to complete phase III clinical trial and be poised for commercialization to benefit the gMG patients. The Company also initiated open-label extension clinical trial in 2022 and completed enrolment in March 2023. As the cut-off of open-label extension clinical trial for gMG in November 2023, the data showed sustainable efficacy and safety of batoclimab in long-term disease management.
Porustobart (HBM4003) is the next-generation, fully human heavy chain only anti-CTLA-4 antibody generated from the HCAb platform. It is also the first fully human heavy chain only antibody which has entered into clinical development around the world. In 2023, Harbour Therapeutics conducted the global clinical development program of porustobart for multiple types of solid tumors, and positive data of efficacy and safety profile have been read out in the ongoing trials of neuroendocrine neoplasms and hepatocellular carcinoma.
Another example demonstrating Harbour Therapeutics’ strong research capabilities is the discovery of HBM7008, a novel product targeting B7H4 and 4-1BB. Developed from the immune cell engager platform HBICE, HBM7008 is the only bispecific antibody against these two targets globally. Leveraging and integrating the expertise in biology and antibody engineering and the unique characteristics of HBICE platform, HBM7008 showed exciting performance both in efficacy and safety profile at pre-clinical stage. In 2022, the Company conducted the phase I trials in the U.S. and Australia. In February 2023, to maintain a leading position in the development of this first-in-class asset, the Company entered into a co-development collaboration with Cullinan Oncology, to expand its study process in the U.S., Europe and Australia.
HBM1020 is a first-in-class fully human monoclonal antibody generated from Harbour Mice platform targeting B7H7. As a newly discovered member of the B7 family, B7H7 expression is found non-overlapping with PD-L1 expression in multiple tumor types, which potentially plays a more important role for tumor cells to escape immune surveillance. HBM1020 is the first product against B7H7 in clinical stage globally. With its excellent product design and target features, HBM1020 presents great potential to address huge unmet medical needs on solid tumors.
Driven by the Company’s leading drug innovation and discovery engine, new assets are continually being developed, of which HBM1020, HBM1022, HBM1007 and HBM9033 obtained the IND clearance from the U.S. FDA to initiate clinical study in the U.S. in 2023, and HBM9027 obtained the IND clearance from U.S. FDA in the first quarter of 2024.
In addition, we have a number of pre-clinical stage products in the pipeline, including HBM7004, HBM1047 and HBM9014, which have shown great potential for development.
PLATFORM-VALUE-MAXIMIZED BUSINESS COLLABORATIONS

In 2023, the Company continued to expand its business collaborations with leading academic institutions and select industrial partners focusing on innovation and efficiency across the world. The business collaboration model is not only limited to out-licensing, but also to engage with academic institutions or other leading innovative pioneers in the industry for co-development and incubation of joint ventures on next-generation innovative therapy. With flexible business models built around proprietary technologies and platforms, the Company can and will maximize its platform value to address global unmet medical needs.

Assets Collaboration of Harbour Therapeutics

Harbour Therapeutics has entered into several external collaborations in terms of pipeline licensing and collaborations. In 2023, Harbour Therapeutics has granted the regional out-licensing of HBM7008 in the U.S. to Cullinan Oncology, and HBM7022/AZD5863, which was licensed to AstraZeneca in 2022, has entered into clinical stage. Meanwhile, the three assets which have been licensed the Greater China Rights to Hualan Genetic obtained IND approvals from NMPA to initiate clinical study in China. In addition, HBM9378, which was developed in collaboration with Kelun-Biotech, completed its phase I clinical trial.

With these multiple collaborations based on the assets generated from HBICE, Harbour Therapeutics has shown its strength and unique advantages in building a comprehensive portfolio in immune cell engagers. And the co-development and collaboration of the pipeline is not only the recognition of the industry partners for the Company’s products and technology platforms, but will also help the Company to improve the efficiency of portfolio advancement, spread the costs and risks, and make the development of the Company more robust.

Multiple Collaborations of Nona Biosciences

In addition to collaboration through the molecules and pipeline generated from the platforms, the Company is also focusing the vision on more original and innovative collaborations on early stages. By integrating the industry leading Harbour Mice and HCAb PlusTM platforms with an experienced therapeutic antibody discovery team, Nona Biosciences provides a one-stop solution for therapeutic antibody discovery, engineering and development from I to ITM (Idea to IND) with flexible business models.

From the end of 2022, Nona Biosciences achieved big success in its launch as it has landed several international collaborations in multiple innovative formats. It is worth mentioning that Nona Biosciences has granted the global out-licensing of HBM9033, a potential best-in-class MSLN-targeted ADC, to Pfizer. Until now, Harbour Mice platforms have been validated by over 50 industry and academic partners.

Nona Biosciences has established four leading technology units based on HCAb, including protein engineering, conjugation technology, delivery technology and cell therapy to empower the next-generation therapies. With the multiple collaborations based on the assets generated from HCAb PlusTM, Nona Biosciences has demonstrated its robust capabilities in antibody discovery and development, exploring a new path to expand the collaboration network and maximize the value of the platforms.

Incubation on Cutting-Edge Collaborations

To give full play to the value of the unique platform technologies, the Company has continued to explore the expandability of platform technology application scenarios which generate impactful values and bring us new value growth points with minimal marginal investment. Representative projects include HBM Alpha Therapeutics, in partnership with Boston Children’s Hospital, and Shanghai NK Cell Technology Limited.

2024 OUTLOOK: EXTENSIVE GLOBALIZATION AND BREAKTHROUGH INNOVATION

Looking to the future, Harbour BioMed will keep driving business growth and accomplishing its mission through two key pillars, Harbour Therapeutics and Nona Biosciences. The former will advance multiple clinical trials of the internal pipeline to fully advance the global clinical development project, and the latter will keep providing integrated discovery solutions for biotechnology and pharmaceutical companies and ultimately create an innovation ecosystem to promote biological advancement.

A range of products based on the technology platform and generated from the concept of T-cell engager and NK cell engager, will be pushed forward to clinical stage in the following years. With a combination of in-house development and business collaborations, the Company will continuously form a portfolio of products with a differentiated competitive advantage in immuno-oncology.

The platform-valued-maximized business collaborations, driven by Nona Biosciences, will further walk the Company down the path of global development. Positive outcomes have been attained through platform-based collaborations with top institutions around the world and more extensive global collaborations are expected in 2024 as our preclinical products become increasingly mature.

Bridge Biotherapeutics Launches a Research Collaboration with Emory University School of Medicine to Explore Combination Therapy of BBT-877 for KRAS/P53 Mutant NSCLC Patients Resistant to Anti-PD-1 Blockade

On March 28, 2024 Bridge Biotherapeutics (KQ288330), a South Korean clinical-stage biotech company developing novel drugs for cancer, fibrosis, and inflammation, reported a research collaboration with Dr. Jessica M. Konen’s Lab at Emory University School of Medicine (Press release, Bridge Biotherapeutics, MAR 28, 2024, View Source [SID1234641588]).

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The collaboration will explore the potential therapeutic benefits of combination therapy of BBT-877, a novel autotaxin (ATX) inhibitor, with anti-PD-1 immunotherapy for the treatment of non-small cell lung cancer (NSCLC) in patients harboring KRAS and P53 (KP) mutations who are resistant to anti-PD-1 blockade.

As a member of the cancer immunology research program at Winship Cancer Institute of Emory University, Dr. Konen’s research has shown that autotaxin has a direct impact on the body’s immune response to tumors. Specifically, higher levels of ATX expression are associated with a decrease in the number of tumor-infiltrating CD8 T cells and an increase in inflammatory gene signatures, including those related to the cytolytic activity of CD8 T cells. Furthermore, an activated tumor-immune microenvironment upregulates ATX and thus provides opportunities for acquired resistance to anti-PD-1 treatment.[i]

From their in vitro studies, the company and the laboratory found that BBT-877 induces CD4 and CD8 T cell proliferation and activation markers, with a robust increase in CD8 T cells that express Granzyme B. The ongoing research collaboration is dedicated to investigating the potential benefits of combining BBT-877 with anti-PD-1 therapy as a treatment approach.

"We are excited to collaborate with Dr. Konen’s team at Emory University School of Medicine to explore the potential of BBT-877 in overcoming resistance to anti-PD-1 therapy in NSCLC patients with KRAS and P53 mutations as a combination therapy with immuno-oncology agents," said James Lee, CEO of Bridge Biotherapeutics. "We believe that Dr. Konen’s research on the role of autotaxin in immunosuppression has the potential to significantly improve treatment outcomes for those patients who are resistant to anti-PD-1 therapy."

"This collaboration presents a promising opportunity to translate our scientific understanding of autotaxin’s role in immunotherapy resistance into a novel therapeutic approach for KRAS/P53 mutant NSCLC patients," said Dr. Jessica Konen, Department of Hematology and Medical Oncology Instructor at Emory University School of Medicine. "We are pleased to work with Bridge Biotherapeutics to explore indication expansion into NSCLC through a combination of BBT-877 with anti-PD-1 agent and potentially offer new hope to those patients."

Under the terms of the collaboration, Bridge Biotherapeutics will provide financial support and access to BBT-877, while Dr. Jessica Konen’s Lab will contribute its expertise in immunology and oncology. Together, the two entities will conduct preclinical studies to evaluate the therapeutic potential of BBT-877 in enhancing anti-tumor immunity.