On January 8, 2023 Biocytogen Pharmaceuticals (Beijing) Co., Ltd. ("Biocytogen", HKEX: 02315), a global biotechnology company focused on the development of novel antibody therapeutics, reported that it has entered into an exclusive option and license agreement with Radiance Biopharma Inc. ("Radiance"), a biotechnology company that specializes in the development of next-generation antibody-drug conjugates (Press release, Biocytogen, JAN 8, 2024, View Source [SID1234639130]).

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 agreement grants Radiance an option to license from Biocytogen a first-in-class, fully human anti-HER2/TROP2 bispecific antibody drug conjugate (BsADC) for the development, manufacture and commercialization of therapeutic products for all human indications worldwide. HER2 and TROP2 are two tumor-associated antigens (TAAs) that are commonly expressed and co-expressed by various tumor types, including breast, gastric, colorectal, bladder, pancreatic, and non-small cell lung cancers.

Under the terms of the agreement, upon exercise of the option, Biocytogen will be entitled to an option fee, a royalty, development and commercialization milestone payments, and single-digit royalties on net sales. In addition, Biocytogen has the right to collect any sublicensing fee between Radiance and a third party.

Dr. Yuelei Shen, President and CEO of Biocytogen, said , " We are pleased to collaborate with Radiance, a strong team with extensive drug development experience, to develop a leading proprietary, fully human bispecific antibody-drug conjugate. We are optimistic that the combination of our strength in BsADC discovery and the extensive experience of the Radiance team will help accelerate the commercialization of this dual BsADC."

Marc Lippman, MD, Chairman of the Board of Radiance, said , " We are pleased to enter into this exclusive option and license agreement with Biocytogen for a novel human bispecific antibody-drug conjugate targeting HER2 and Trop2. Preclinical data from in vitro and in vivo testing with this BsADC show promising high potency of anti-tumor activities in leading tumor indications. We look forward to working with Biocytogen to bring the product into practice for the benefit of patients."

On January 8, 2024 Nuvalent, Inc. (Nasdaq: NUVL), a clinical-stage biopharmaceutical company focused on creating precisely targeted therapies for clinically proven kinase targets in cancer, reported its "OnTarget 2026" operating plan to guide efforts towards having its first potential approved product in 2026 (Press release, Nuvalent, JAN 8, 2024, View Source [SID1234639162]).

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!

"Over the last three years, Nuvalent has evolved from an emerging preclinical-stage start-up to an established biotech leader in advanced clinical development. In this period, we have disclosed three novel programs, demonstrated preliminary proof-of-concept for our two lead clinical programs, and launched our first registration-directed study," said James Porter, Ph.D., Chief Executive Officer at Nuvalent. "These accomplishments demonstrate a track record of successful execution and resolute focus on our founding goal: to translate our expertise in chemistry and structure-based drug design into potential best-in-class treatments for patients with cancer."

Dr. Porter continued, "2024 marks the beginning of the ‘next 3 years’ culminating in our first potential FDA approval expected in 2026, a critical milestone towards fulfilling our commitment to patients. Throughout this year, our priority is on execution of the global registrational strategies for our ROS1 and ALK programs that underpin our ultimate goal of moving up the treatment paradigm, including the potential launch of our first-line ALK strategy. We expect pivotal data from at least one of our parallel-lead programs in 2025 in support of potential New Drug Application submissions in 2026. By 2026, we also anticipate further advancing our HER2 program and our pipeline of discovery programs."

"‘OnTarget 2026’ outlines a clear set of mission-driven priorities that also deliver multiple transformative catalysts for our stakeholders over the short, intermediate, and long term," said Alexandra Balcom, Chief Financial Officer at Nuvalent. "Backed by strong product candidates, scientific rigor, and a bolstered balance sheet, we believe we are well positioned to continue achieving our goals as a growing team aligned around a firm commitment to patient impact."

OnTarget 2026: The Path to Patient Impact

OnTarget 2026 delineates Nuvalent’s 3-year operating plan towards bringing new, potential best-in-class medicines to patients with cancer. As part of this plan, Nuvalent expects to achieve the following anticipated milestones throughout 2024, leading to the company’s first potential pivotal data in 2025 and first potential approved product in 2026:

•
2024: Execute on Global Registrational Strategies
o
Progress the Phase 2 portion of its ARROS-1 trial of NVL-520 in patients with advanced ROS1-positive NSCLC with registrational intent;
o
Initiate the Phase 2 portion of its ALKOVE-1 trial of NVL-655 in patients with advanced ALK-positive NSCLC with registrational intent;

o
Launch the front-line development strategy for its ALK program;
o
Initiate the Phase 1 trial for its HER2 program; and,
o
Present interim data from its ongoing ARROS-1 and ALKOVE-1 clinical trials at medical meetings.
•
2025: First Pivotal Data
•
2026: First Approved Product

2023 Year-End Cash and Guidance

Nuvalent ended 2023 with approximately $719.9 million in cash, cash equivalents and marketable securities (unaudited), which, based on its current operating plans, is expected to fund its operations into 2027. This amount is a preliminary, unaudited estimate only as of today, could change following completion of year-end closing procedures, and does not present all information necessary for an understanding of our financial position as of December 31, 2023.

Presentation at 42nd Annual J.P. Morgan Healthcare Conference

Dr. Porter will present at the 42nd Annual J.P. Morgan Healthcare Conference in San Francisco on Tuesday, January 9, 2024 at 7:30 a.m. PT in San Francisco. A live webcast will be available in the Investors section of Nuvalent’s website at www.nuvalent.com, and will be archived for 30 days following the conference.

About NVL-520

NVL-520 is a brain-penetrant ROS1-selective inhibitor created with the aim to overcome limitations observed with currently available ROS1 inhibitors. NVL-520 is designed to remain active in tumors that have developed resistance to currently available ROS1 inhibitors, including tumors with treatment-emergent ROS1 mutations such as G2032R. In addition, NVL-520 is designed for brain penetrance to potentially improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ROS1 inhibitors and to drive deep, durable responses for patients across all lines of therapy. NVL-520 has received orphan drug designation for ROS1+ non-small cell lung cancer (NSCLC) and is currently being investigated in the ARROS-1 trial (NCT05118789), a first-in-human Phase 1/2 clinical trial for patients with advanced NSCLC and other solid tumors.

About NVL-655

NVL-655 is a novel brain-penetrant ALK-selective inhibitor created with the aim to overcome limitations observed with currently available ALK inhibitors. NVL-655 is designed to remain active in tumors that have developed resistance to first-, second-, and third-generation ALK inhibitors, including tumors with both single or compound treatment-emergent ALK mutations such as those involving G1202R. In addition, NVL-655 is designed for CNS penetrance to improve treatment options for patients with brain metastases, and to avoid inhibition of the structurally related tropomyosin receptor kinase (TRK) family. Together, these characteristics have the potential to avoid TRK-related CNS adverse events seen with dual TRK/ALK inhibitors and to drive deep, durable responses for patients across all lines of therapy. NVL-655 has received orphan drug designation for ALK+ non-small cell lung cancer (NSCLC) and is currently being investigated in the ALKOVE-1 clinical trial (NCT05384626), a first-in-human Phase 1/2 clinical trial for patients with advanced ALK-positive NSCLC and other solid tumors.

About NVL-330

NVL-330 is a novel, brain-penetrant, and HER2-selective tyrosine kinase inhibitor designed to address the combined medical need of treating HER2-mutant tumors, including those with HER2 exon 20 insertion mutations, avoiding treatment related adverse events due to off-target inhibition of wild-type EGFR, and treating brain metastases.

On January 8, 2024 bluebird bio, Inc. (Nasdaq: BLUE) (the Company; bluebird) reported updates to be presented at the 42nd Annual J.P. Morgan Healthcare conference including commercial launch progress, 2024 program milestones and financial outlook (Press release, bluebird bio, JAN 8, 2024, View Source [SID1234639059]). Andrew Obenshain, chief executive officer, is scheduled to present Tuesday, January 9 at 10:30 a.m. PT/1:30 p.m. ET.

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!

"In 2023 bluebird cemented our status as a gene therapy leader, securing our third FDA approval in under two years and establishing a commercial footprint that will support growth in the coming year and beyond," said Andrew Obenshain, chief executive officer, bluebird bio. "In 2024 we are leveraging our validated commercial strategy to accelerate the launch of LYFGENIA and drive continued strong uptake for ZYNTEGLO. We are extremely pleased with the indicators of demand from both patients and providers in the weeks following FDA approval of LYFGENIA and are focused on using our real-world experience to support timely and equitable access and deliver a positive treatment experience."

Highlights from the Company’s update include:

Synergies with ZYNTEGLO commercial network are accelerating LYFGENIA commercial launch in 2024

Established Qualified Treatment Center (QTC) network in place, with 48 centers activated for ZYNTEGLO for beta-thalassemiai as of January 5, 2024.
35 of 48 centers ready to receive referrals for LYFGENIA for sickle cell disease as of January 5, 2024.
All centers are anticipated to be ready to treat with both ZYNTEGLO and LYFGENIA by end of Q1 2024.
Validated access and reimbursement strategy is driving favorable coverage landscape for LYFGENIA and ZYNTEGLO

bluebird has signed outcomes-based agreements for LYFGENIA with national payer organizations representing dozens of downstream plans and covering approximately 200 million U.S. lives.
Advanced discussions are ongoing with additional commercial payers and with more than 15 Medicaid agencies representing 80% of individuals with sickle cell disease in the U.S.
bluebird has designed outcomes-based agreements that are unique to LYFGENIA and offer payers meaningful risk sharing tied to VOE-related hospitalizations, with patients followed for three years. Informed by input from state Medicaid agencies, the Company has designed an offering specifically for Medicaid that addresses the need for predictability and operational ease that is essential for states grappling with resource constraints.
Outcomes-based agreements are in place for ZYNTEGLO with both commercial and Medicaid payers, and more than 200 million U.S. lives are covered through contracts or favorable coverage policies. Timely access to ZYNTEGLO for people living with beta-thalassemia continues, with zero ultimate denials across both Medicaid and commercial payers.
bluebird also continues to engage with Center for Medicare and Medicaid Innovation (CMMI) on its Cell and Gene Therapy Access Model, which is anticipated to be implemented in 2025.
Strong commercial momentum is poised to translate into sustained revenue recognition

26 patient starts were completed in 2023 across bluebird’s commercial portfolio, including 20 for ZYNTEGLO and 6 for SKYSONA. 2023 patient starts will drive revenue recognition in 2024 as patients complete the gene therapy treatment journey.
bluebird anticipates the first patient start for LYFGENIA in Q1 2024.
The Company anticipates 85 to 105 patient starts combined across all three of its FDA approved therapies (LYFGENIA, ZYNTEGLO, SKYSONA) in 2024.
Liquidity and Cash Runway Update

The Company’s preliminary unaudited cash and cash equivalents and marketable securities balance was approximately $275 million, including restricted cash of approximately $53 million, as of December 31, 2023. bluebird expects its cash, cash equivalents, and marketable securities, excluding restricted cash, will be sufficient to meet bluebird’s planned operating expenses and capital expenditure requirements into the first quarter of 2025 as bluebird progresses its launch of LYFGENIA gene therapy for sickle cell disease and continues to scale its launches of ZYNTEGLO and SKYSONA for beta-thalassemia and cerebral adrenoleukodystrophy, respectively.

The Company has taken additional steps to strengthen its financial position by entering into an accounts receivable factoring agreement which will accelerate cash collection related to patient starts across its portfolio of approved therapies.

Presentation at the 2024 J.P. Morgan Healthcare Conference

Andrew Obenshain, chief executive officer, bluebird bio, will present a corporate update on Tuesday, January 9 at 10:30 a.m. PT/1:30 p.m. ET. A live webcast of the presentation will be available on the "Events & Presentations" page within the Investors & Media section of the bluebird bio website at View Source A replay of the webcast will be available on the bluebird bio website for 30 days following the event.

On January 8, 2024 Fate Therapeutics, Inc. (NASDAQ: FATE), a clinical-stage biopharmaceutical company dedicated to bringing a first-in-class pipeline of induced pluripotent stem cell (iPSC)-derived cellular immunotherapies to patients with cancer and autoimmune disorders, reported the initiation of enrollment for its Phase 1 clinical trial of FT825 / ONO-8250, a multiplexed-engineered, chimeric antigen receptor (CAR) T-cell product candidate targeting human epidermal growth factor receptor 2 (HER2) (Press release, Fate Therapeutics, JAN 8, 2024, View Source [SID1234639076]). The iPSC-derived CAR T-cell product candidate incorporates a novel HER2-targeted antigen binding domain and is designed to overcome unique challenges in treating solid tumors. The Phase 1 study of FT825 / ONO-8250 is being conducted under a strategic collaboration with Ono Pharmaceutical Co., Ltd. (Ono).

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!

"Since the formation of our partnership with Ono in 2018, we have worked closely together to pioneer the manufacture of CD8 alpha-beta T cells from iPSCs and to discover and integrate novel synthetic controls of cell function into our iPSC-derived CAR T-cell product platform for safe and effective treatment of solid tumors, including functional elements designed to promote cell trafficking, resist immune suppression in the tumor microenvironment, and preferentially target cancer cells," said Scott Wolchko, President and Chief Executive Officer of Fate Therapeutics. "The preclinical data for FT825 / ONO-8250 indicate a highly-differentiated therapeutic profile across a broad range of solid tumors, with the novel HER2-targeted antigen binding domain demonstrating selective targeting of cancer cells expressing HER2 including those with low expression. We are excited to initiate the Phase 1 study in collaboration with Ono and assess the potential to benefit patients with hard-to-treat advanced solid tumors who currently have limited treatment options."

Designed using the Company’s iPSC Product Platform, FT825 / ONO-8250 incorporates seven novel synthetic controls of cell function including a CXCR2 receptor to promote cell trafficking, a chimeric TGFβ receptor to redirect immunosuppressive signals in the tumor microenvironment, and a high-affinity, non-cleavable CD16a receptor to enable antibody-dependent cellular cytotoxicity. Preclinical data of FT825 / ONO-8250 presented at the 2023 Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) Annual Meeting demonstrated that the profile of the novel HER2-targeted antigen binding domain is unique and differentiated from that of trastuzumab, exhibiting similar potency with greater specificity for cancer cells expressing HER2.

The Phase 1 study is designed to investigate a single dose of FT825 / ONO-8250 as monotherapy and in combination with monoclonal antibody therapy in previously-treated patients with advanced solid tumors. The dose escalation and dose expansion portions of the trial are expected to evaluate safety, tolerability, and pharmacokinetics as well as anti-tumor activity by overall response rate, duration of response and disease control rate.

Under the terms of its Collaboration and Option Agreement with Ono, Fate will jointly develop and commercialize FT825 / ONO-8250 with Ono in the U.S. and Europe, and Ono maintains exclusive development and commercialization rights for FT825 / ONO-8250 in the rest of the world. Fate is eligible to receive clinical, regulatory and commercial milestone payments as well as tiered royalties on net sales outside of the United States and Europe by Ono. The parties are currently conducting preclinical development of an additional solid tumor program targeting an undisclosed tumor-associated antigen.

About Fate Therapeutics’ iPSC Product Platform
The Company’s proprietary induced pluripotent stem cell (iPSC) product platform enables mass production of off-the-shelf, multiplexed-engineered cell products that are selectively designed, incorporate novel synthetic controls of cell function, and are intended to deliver multiple mechanisms of therapeutic importance to patients. Human iPSCs possess the unique dual properties of unlimited self-renewal and differentiation potential into all cell types of the body. The Company’s platform combines multiplexed engineering and single-cell selection of human iPSCs to create clonal master iPSC lines. Analogous to master cell lines used to mass produce biopharmaceutical drug products such as monoclonal antibodies, the Company utilizes its clonal master iPSC lines as a renewable cell source to manufacture multiplexed-engineered cell products which are well-defined and uniform in composition, can be stored in inventory for off-the-shelf availability, can be combined and administered with other therapies, and can potentially reach a broad patient population. As a result, the Company’s platform is uniquely designed to overcome numerous limitations associated with the manufacture of cell therapies using patient- or donor-sourced cells. Fate Therapeutics’ iPSC product platform is supported by an intellectual property portfolio of over 400 issued patents and 450 pending patent applications.

On January 8, 2024 Iktos, a company specialized in Artificial Intelligence for new drug design, and Nerviano Medical Sciences S.r.l (NMS), a leading Italian clinical stage biotech discovering and developing innovative therapies for the treatment of cancer, reported a collaboration agreement in AI for unprecedented kinase project (Press release, Nerviano Medical Sciences, JAN 8, 2024, View Source [SID1234639092]). As per the agreement, NMS will leverage Iktos’s expertise in drug design services, with the ultimate goal of identifying at least one promising candidate molecule. Iktos’s innovative generative modeling technology platform, Makya, will be employed to apply a ligand- and structure-based approach in designing novel molecules that align with NMS’s candidate drug target profile (CDTP); Iktos’s generative AI approach uniquely enables the exploration of chemical space and produces innovative molecule designs with greater freedom to operate and good synthetic tractability thanks to integration with Iktos’s retrosynthesis AI technology platform Spaya.

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!

"We are excited about the prospect of collaborating with IKTOS, a proven leader in AI-powered drug discovery platforms to delivery one candidate drug for a truly, unprecedented novel kinase" said Hugues Dolgos, Pharm.D., Chief Executive Officer of NMS.

"We are excited and proud to collaborate with Nerviano Medical Sciences S.r.l, a leading company focused on the discovery and development of oncology drugs and the largest oncological R&D company in Italy. In the framework of our collaboration, the NMS team will use Makya, Iktos proprietary Gen AI software in their discovery of a novel candidate drug" said Dr. Quentin Perron, Co-founder and CSO of Iktos. "Indeed, at Iktos, we are committed to developing innovative technologies that enhance the chance of success of small molecule discovery. Our mission is to expedite drug discovery through the application of AI, which we achieve by integrating our robust algorithmic technology, leveraging our expertise from numerous successful collaborations."