On May 13, 2024 Ajax Therapeutics, Inc., a biopharmaceutical company developing next generation JAK inhibitors for patients with myeloproliferative neoplasms (MPNs), reported that it has received clearance for its Investigational New Drug (IND) application from the U.S. Food and Drug Administration (FDA) to initiate a Phase 1clinical study of AJ1‑11095, a first-in-class Type II JAK2 inhibitor, for the treatment of patients with myelofibrosis (Press release, Ajax Therapeutics, MAY 13, 2024, View Source [SID1234643179]).

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"We are thrilled to obtain clearance to advance AJ1-11095 into the clinic and excited to bring this innovative new medicine to patients with myelofibrosis," said Martin Vogelbaum, co-founder and CEO of Ajax Therapeutics. "This is an important milestone for our company and our first program to enter the clinic and the first clinical study to ever evaluate a Type II JAK2 inhibitor in patients."

"We look forward to the clinical development of AJ1-11095 in myelofibrosis and to initiating our Phase 1 dose escalation study, AJX-101, later this year," said David Steensma, MD, FACP, Chief Medical Officer at Ajax. "As a first-in-class therapy with a unique mechanism as a Type II inhibitor of JAK2, AJ1-11095 was developed to provide a much-needed new treatment for patients with myelofibrosis by offering the potential for improved efficacy with disease modifying effects compared to existing therapies."

About AJ1-11095

AJ1-11095 was designed by Ajax, through our collaboration with Schrödinger, to be a next generation JAK2 inhibitor by using structure-based drug design and computational methods at scale to selectively bind the Type II conformation of the JAK2 kinase and to provide greater efficacy with disease modification compared to all currently approved JAK2 inhibitors which bind the Type I conformation of JAK2. Additionally, AJ1-11095 has been shown in preclinical studies to reverse marrow fibrosis, reduce mutant allele burden and maintain efficacy against MPN cells that become resistant to chronic Type I JAK2 inhibition.

About Myelofibrosis

Myelofibrosis (MF) is a rare blood cancer that affects approximately 20,000 patients in the United States. The disease is characterized by spleen enlargement, scarring (fibrosis) in the bone marrow, progressive anemia, and debilitating symptoms, such as fatigue, night sweats, itching, and abdominal discomfort, which can impair a patient’s’ quality of life. The most widely used treatment for MF patients are Type I JAK2 inhibitors which can reduce spleen size and provide symptomatic improvement but have little effect on the underlying cause of disease. Over time, most MF patients stop Type I JAK2 inhibitor therapy. The most common causes for treatment discontinuation include a lack of benefit or loss of response, adverse events, and disease progression, leaving significant unmet treatment needs for these patients.

On May 13, 2024 Legend Biotech Corporation (NASDAQ: LEGN) (Legend Biotech), a global leader in cell therapy, reported its first quarter 2024 unaudited financial results and key corporate highlights (Press release, Legend Biotech, MAY 13, 2024, View Source [SID1234643178]).

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"Legend made great progress in the first quarter, culminating in our exciting announcements in recent weeks. We received label expansions for CARVYKTI in the U.S., Europe, and Brazil that have changed the treatment paradigm for multiple myeloma and will enable more patients to receive our transformative therapy earlier in the course of their disease," said Ying Huang, Ph.D., Chief Executive Officer of Legend Biotech. "With more patients needing access to CARVYKTI, we have increased our manufacturing capacity and have scaled up our operations to reach our goal of 10,000 annual doses by the end of 2025. The expansion of our partnership with Novartis demonstrates our commitment to ensuring every patient who needs CARVYKTI can access it."

Regulatory Updates

The U.S. Food and Drug Administration (FDA) approved CARVYKTI for the treatment of adult patients with relapsed or refractory multiple myeloma who have received at least one prior line of therapy including a proteasome inhibitor (PI) and an immunomodulatory agent (IMiD) and are refractory to lenalidomide following the Oncologic Drug Advisory Committee’s (ODAC) unanimous (11 to 0) vote recommending the approval of CARVYKTI.
The European Commission (EC) granted approval for the label expansion of CARVYKTI for the treatment of adult patients with relapsed and refractory multiple myeloma who have received at least one prior therapy, including an immunomodulatory agent and a proteasome inhibitor, have demonstrated disease progression on the last therapy, and are refractory to lenalidomide.
The Brazilian Health Regulatory Agency, ANVISA (Agência Nacional de Vigilância Sanitária), approved CARVYKTI for the treatment of adult patients with multiple myeloma, who previously received a proteasome inhibitor and are refractory to lenalidomide, as well as adult patients with relapsed or refractory multiple myeloma, who previously received a proteasome inhibitor, an immunomodulatory agent and anti-CD38 antibody.
Key Business Developments

Legend and Johnson & Johnson* entered into a Master Manufacturing and Supply Services Agreement with Novartis Pharmaceuticals Corporation to supplement our existing manufacturing capabilities and increase commercial supply of CARVYKTI
Published inaugural Environmental, Social & Governance (ESG) report which aligns with the Sustainable Accounting Standards Board (SASB) Biotechnology and Pharmaceutical sector standards, shares ESG data collection and disclosure roadmap, and future growth strategy for good corporate citizenship
* In December 2017, Legend Biotech entered into an exclusive worldwide collaboration and license agreement with Janssen Biotech, Inc., a Johnson & Johnson company, to develop and commercialize cilta-cel (the Janssen Agreement).

First Quarter 2024 Financial Results

License Revenue: License revenue was $12.2 million for the first quarter of 2024 and consisted of the recognition of deferred revenue in connection with the global license agreement with Novartis Pharma AG to develop, manufacture, and commercialize LB2102 and other potential CAR-T therapies selectively targeting DLL3. Legend did not recognize any license revenue for the first quarter of 2023.
Collaboration Revenue: Collaboration revenue was $78.5 million for the first quarter of 2024 compared to $36.3 million for the first quarter of 2023. The increase was primarily due to an increase in revenue generated from sales of CARVYKTI in connection with the Janssen Agreement.
Collaboration Cost of Revenue: Collaboration cost of revenue was $49.1 million for the first quarter of 2024 compared to $35.6 million for the first quarter of 2023. The increase was primarily due to Legend Biotech’s share of the cost of sales in connection with CARVYKTI sales under the Janssen Agreement.
Cost of License and Other Revenue: Cost of license and other revenue for the three months ended March 31, 2024 was $5.6 million and consisted of costs in connection with the global license agreement with Novartis Pharma AG to develop, manufacture, and commercialize LB2102 and other potential CAR-T therapies selectively targeting DLL3. The Company did not incur any cost of license and other revenue for the three months ended March 31, 2023.
Research and Development Expenses: Research and development expenses were $101.0 million for the first quarter of 2024 compared to $84.9 million for the first quarter of 2023. The increase was primarily driven by continuous research and development activities in cilta-cel, including start up costs for clinical production in Belgium and continued investment in Legend’s solid tumor programs.
Administrative Expenses: Administrative expenses were $31.9 million for the first quarter of 2024 compared to $22.2 million for the first quarter of 2023. The increase was primarily due to the expansion of administrative functions and infrastructure to increase manufacturing capacity.
Selling and Distribution Expenses: Selling and distribution expenses were $24.2 million for the first quarter of 2024 compared to $18.0 million for the first quarter of 2023. The increase was primarily driven by costs associated with commercial activities for cilta-cel, including the expansion of the sales force and second line indication launch preparation.
Net Loss: Net loss was $59.8 million for the first quarter of 2024, compared to a net loss of $112.1 million for the first quarter of 2023.
Cash Position: Cash and cash equivalents, time deposits, and short-term investments were $1.3 billion as of March 31, 2024.
Webcast/Conference Call Details:
Legend Biotech will host its quarterly earnings call and webcast today at 8:00 am ET. To access the webcast, please visit this weblink.

A replay of the webcast will be available on Legend Biotech’s website at View Source

On May 13, 2024 Pheast Therapeutics ("Pheast"), a biotech developing novel macrophage checkpoint therapies to defy cancer, reported the first presentation of preclinical data for PHST001, an anti-CD24 antibody drug candidate that is designed to block a key macrophage "don’t eat me" signal on cancer cells (Press release, Pheast Therapeutics, MAY 13, 2024, View Source [SID1234643177]). The data to be presented at the 20th Annual PEGS Boston Summit demonstrate that PHST001 can powerfully induce an anti-cancer immune response and drive therapeutic efficacy in in vivo models.

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"Pheast’s lead drug candidate, PHST001, is an exceptionally well-engineered, well-expressed antibody against an exciting new target, CD24," said Roy Maute, Ph.D., Cofounder and CEO, Pheast Therapeutics. "Our preclinical studies show potent efficacy in challenging mouse model systems, which supports the possibility of clinical efficacy in cancers where other immunotherapies have failed and the unmet need remains high. The Pheast team is making rapid progress to support filing an investigational new drug application with the goal to enter the clinic in patients in the first half of next year."

CD24 is a newly discovered immune checkpoint that is highly expressed by many human cancers, including ovarian and triple negative breast cancer (TNBC). High expression of CD24 is a negative prognostic factor in multiple cancer indications. CD24 interacts with the macrophage receptor Siglec-10, and shields cancer cells from attack by macrophages. Pheast has engineered PHST001 to bind recombinant CD24 and cell surface CD24 with high affinity and specificity and to block Siglec-10 binding.

Pheast scientists have demonstrated that blocking CD24 with PHST001 induces macrophage phagocytosis of multiple cancer subtypes in vitro, and have shown potent efficacy for PHST001 in vivo. In some preclinical models, PHST001 monotherapy is sufficient to shrink and fully eradicate tumors in all treated mice.

"These data demonstrate Pheast’s robust in-house antibody discovery and protein engineering capabilities, allowing rapid discovery and development of therapeutic monoclonal antibodies," said John Burg, Ph.D., Senior Director, Protein Sciences. "Our innovative platform for discovery of tumor-specific immune checkpoints for precision immuno-oncology treatments will support exploring additional targets and developing new pipeline programs."

Dr. Burg will present these data on Monday, May 13, 2024, at 1:15 pm ET in a talk titled, "Engineering a Therapeutic Monoclonal Antibody Targeting CD24, a Macrophage Checkpoint."

On May 13, 2024 Aldevron, a leading global manufacturer of DNA, RNA, and proteins used in cell and gene therapies and vaccine development, reported it has entered a strategic partnership with Acuitas Therapeutics, Inc., a private biotechnology company specializing in the development of delivery systems for nucleic acid therapeutics based on lipid nanoparticles (LNPs) (Press release, Aldevron, MAY 13, 2024, View Source [SID1234643176]). This partnership will allow Aldevron to expand its capabilities in mRNA LNP encapsulation by incorporating Acuitas’ proprietary LNP encapsulation platform, increasing Aldevron’s services and capabilities as an mRNA sequence-to-vial custom manufacturer.

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In May of 2023, Aldevron announced the expansion of its mRNA production capabilities to include Cytiva’s mRNA LNP encapsulation and aseptic fill-finish platform. This new strategic partnership with Acuitas Therapeutics, working with its encapsulation platform, equips Aldevron with a diverse range of mRNA LNP technologies, significantly enhancing its mRNA end-to-end drug product service capabilities. The partnership will enable Aldevron to support Acuitas’ LNP formulations and licensees.

"This collaboration with Acuitas is an exciting addition to our existing mRNA sequence-to-vial services and will enable Acuitas partners to leverage Aldevron’s extensive mRNA manufacturing experience and comprehensive end-to-end capabilities," said Mark Wetzel, Vice President mRNA CDMO at Aldevron. "We look forward to our continued partnership that is poised to enhance the landscape of mRNA-based therapies, bringing faster timelines and hope, to patients worldwide."

"We have long known of Aldevron’s reputation and broad experience in the manufacturing of nucleic acids and their commitment to quality," said Chris Barbosa, Vice President of Technology Development at Acuitas Therapeutics, Inc. "We are pleased to work with Aldevron to provide Acuitas partners with an integrated path to clinical drug products using Acuitas’ lipid nanoparticle manufacturing processes." He added, "We look forward to Aldevron’s contribution to working with us to support our global partners in bringing new mRNA-based therapies to patients as quickly and efficiently as possible."

"The Acuitas technology, combined with Aldevron’s extensive experience providing cutting-edge solutions for advancing therapies and rigorous quality processes, provides an exceptional advantage," continued Wetzel. "With our two decades of experience in mRNA technologies, Aldevron has developed a seamless workflow from mRNA sequence-to-vial, and this partnership provides clients LNP platform flexibility for the appropriate development stage for their program."

On May 13, 2024 Leads Biolab reported that the 2024 American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Annual Meeting will be held in Chicago from May 31 to June 4, 2024 (Press release, Nanjing Leads Biolabs, MAY 13, 2024, View Source [SID1234643175]). As the largest and most academically prestigious international oncology conference in the world, the ASCO (Free ASCO Whitepaper) Annual Meeting gathers the latest and most cutting-edge research findings in the field of oncology. Leads Biolabs will present the exciting clinical data on its four innovative clinical programs at the ASCO (Free ASCO Whitepaper) meeting. These presentations will include one oral presentation, two posters, and one online publication, showcasing advancements in various cancer indications such as nasopharyngeal cancer (NPC), extrapulmonary neuroendocrine carcinoma (EP-NEC), hepatocellular carcinoma (HCC), and renal cell carcinoma (RCC).

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Oral Presentation：A novel and uniquely designed bispecific antibody (LBL-024) against PD-L1 and 4-1BB in patients with advanced malignant tumors and neuroendocrine carcinoma: A report of safety and robust efficacy of LBL-024 monotherapy in phase Ⅰ/Ⅱ first in human, open-label, multicenter, dose escalation/expansion Study
Date and Time：2024.06.02 4:30 PM-6:00 PM CDT
Abstract Number：4010

LBL-024, a bispecific antibody composed of an anti-Programmed Cell Death Ligand-1 (PD-L1) and an anti-4-1BB (CD137) antibody. LBL-024 blocks the immunosuppressive pathway of tumor cells by targeting PD-L1 and effectively localizes 4-1BB co-stimulation to the tumor microenvironment, to improve the anti-tumor immune response.

LBL-024 received IND approvals from FDA and NMPA on July 30, 2021 and September 9, 2021 respectively to conduct phase Ⅰ/Ⅱ clinical research. Subsequently, the therapy has achieved outstanding results. On April 30, 2024, it received approval from China’s Center for Drug Evaluation (CDE) of the National Medical Products Administration (NMPA) to conduct a single-arm pivotal study for registration and market authorization. According to the results in the oral presentation, LBL-024 demonstrated good safety profile and very promising antitumor effects in patients with advanced malignant tumors, particularly extrapulmonary neuroendocrine carcinomas (EP-NEC) patients who failed at least one line of chemotherapy. Notably, the efficacy observed in EP-NEC surpassed historic reports with immunotherapy and chemotherapy. The extremely robust efficacy and good safety profiles support the advancement to a pivotal study to accelerate the development of LBL-024 in EP-NEC, a deadly disease of high unmet medical need.

Poster：Anti-LAG-3 antibody LBL-007 in combination with anti-PD-1 antibody Tislelizumab with or without chemotherapy, in patients with advanced nasopharyngeal cancer and other malignant tumors A phase Ⅰb/Ⅱ dose escalation/expansion Study
Date and Time：2024.06.02 9:00 AM-12:00 PM CDT
Abstract Number：6033

LBL-007 is a fully humanized monoclonal antibody against Lymphocyte Activation Gene-3 (LAG-3). By specifically binding to human LAG-3, it relieves the inhibitory effect of LAG-3 on T cells, restores the immune function, and inhibits tumor growth.

Dual inhibition of programmed cell death receptor-1 (PD-1) and LAG-3 is expected to synergistically increase immune response against tumor growth while chemotherapy can enhance the efficacy of immunotherapy through various mechanisms. This phase Ⅰb/Ⅱ study indicates that the combination of LBL-007 and tislelizumab is well-tolerated in patients with advanced malignant tumors.

Poster：Anti-PD-1/TGF-βRII Bispecific Antibody Fusion Protein LBL-015 in patients with advanced malignant tumors：A phase Ⅰ, first-in-human, open-label, multicenter, dose-escalation Study
Date and Time：2024.06.01 9:00 AM-12:00 PM CDT
Abstract Number：2592

LBL-015 is a bifunctional antibody fusion protein targeting PD-1 and Transforming Growth Factor Beta Receptor 2 (TGF-βR2). LBL-015 blocks both PD-1/PD-L1 and TGF-β/TGF-βR2 signaling pathways to reverse immunosuppression and boost immune responses. In addition, LBL-015 can boost immune cell response and inhibit tumor metastasis by blocking TGF-β in the tumor microenvironment. In a Phase 1 clinical study, LBL-015 has demonstrated a good safety profile and encouraging preliminary efficacy signals in patients with advanced solid tumors.

Online Publication：Anti-TNFR2 monoclonal antibody LBL-019 in patients with advanced malignant tumors: A phase Ⅰ, first-in-human, open-label, multicenter, dose escalation Study
Date and Time：2024.05.23 5:00 PM EDT
Abstract Number：e14580

LBL-019 is a humanized IgG1 monoclonal antibody against Tumor Necrosis Factor Receptor 2 (TNFR2). LBL-019 binds specifically to TNFR2 with high affinity and co-stimulates TNFR2 in an Fc crosslinking-dependent manner. This leads to the activation of NF-κB signaling, T cell activation and expansion, thereby promoting antitumor immunity. The data from the Phase 1 study indicate that LBL-019 is well tolerable and has demonstrated preliminary efficacy in patients with advanced malignant tumors.

Dr. Charles Cai, Chief Medical Officer of Leads Biolabs, said "Leads Biolabs will be presenting four clinical projects at 2024 ASCO (Free ASCO Whitepaper). Among them, the efficacy of LBL-024 is significantly higher than the historic reports with immunotherapy and chemotherapy, providing tremendous support for the approval and accelerated development of our pivotal study. The encouraging efficacy and safety profile of LBL-007 is impressive, which is conducive to advancing the pivotal study of LBL-007 in combination with tislelizumab, gemcitabine and cisplatin for NPC. We have adopted a differentiated, innovative, and efficient clinical development strategy, aiming to bring more effective therapies to market sooner. This is a manifestation of Leads Biolabs’ unwavering commitment to addressing patient needs and delivering clinical value, aligning with the company’s mission of ‘care for life’."