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.

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"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).

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"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.

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"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."

On January 8, 2024 Royalty Pharma plc (Nasdaq: RPRX) reported an update on its business performance, including recent key accomplishments, and the full year 2023 outlook for Portfolio Receipts (Press release, Royalty Pharma , JAN 8, 2024, View Source [SID1234639108]). Pablo Legorreta, Royalty Pharma’s founder and Chief Executive Officer, will discuss these updates tomorrow as part of a webcast presentation at the 42nd Annual J.P. Morgan Healthcare Conference to be held at 12:00 p.m. Eastern Time / 9:00 a.m. Pacific Time.

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"Royalty Pharma delivered record performance in 2023," said Pablo Legorreta. "We expect to achieve an all-time high for Portfolio Receipts of approximately $3.05 billion, representing our third consecutive year of double-digit underlying growth since our initial public offering in 2020. We deployed substantial capital to acquire royalties on eight therapies, including our strongest year ever for synthetic royalty transactions. We remain in an excellent position to deliver compounding growth in the years to come while executing on our mission and vision to accelerate innovation in life sciences and transform patient lives globally."

Record 2023 Financial Performance

Based on preliminary unaudited fourth quarter 2023 results, Royalty Pharma now expects to deliver Portfolio Receipts for full year 2023 of approximately $3,050 million, which includes a $50 million payment related to oral zavegepant and exceeds the upper end of its previous guidance range of $2,950 million to $3,000 million. This represents underlying growth of 11% year-over-year prior to Biohaven-related payments and reflects the strong performance of Royalty Pharma’s diversified royalty portfolio. Royalty Pharma also expects Net cash provided by operating activities to be approximately $2,980 million to $2,990 million for full year 2023.

Portfolio Receipts was previously referred to as Adjusted Cash Receipts. The calculation of Portfolio Receipts will result in the same total as under Royalty Pharma’s previous presentation of Adjusted Cash Receipts. This change of presentation will facilitate increased transparency into the economics of individual royalties, as Royalty Receipts by product and franchise, will be reported net of legacy non-controlling interests.

Royalty Pharma’s preliminary unaudited fourth quarter 2023 results provided in this press release are subject to change in connection with the completion of the company’s final adjustments and other developments that may arise during the preparation and audit of its financial statements. Royalty Pharma’s management will host a conference call to discuss Royalty Pharma’s fourth quarter and full year 2023 results in February 2024.

Strong Capital Deployment Added Innovative Therapies, Enhancing Long-term Growth

Since 2020, Royalty Pharma has announced transactions of approximately $13 billion, including $4.0 billion in 2023. Important additions to Royalty Pharma’s portfolio in the past year have included incremental royalties on Evrysdi, the fast-growing therapy for spinal muscular atrophy, as well as the potentially practice-changing, development-stage compounds pelacarsen for cardiovascular disease and KarXT for schizophrenia. Royalty Pharma also had its strongest year ever for synthetic royalties with announced transactions of $775 million, including Adstiladrin for bladder cancer, which is the first gene therapy added to its portfolio, Skytrofa, an approved therapy for growth hormone deficiency and TEV-‘749, an exciting development-stage compound for schizophrenia.

In total, 34 unique therapies have been added to the company’s portfolio since 2020 (of which 17 are either currently or projected to be blockbusters that generate annual sales of $1 billion or more based on consensus estimates). In aggregate, based on consensus sales forecasts, investments made since 2020 are estimated to add approximately $1.2 billion to Royalty Pharma’s Portfolio Receipts in 2025.

Biopharma Funding Environment Driving New Royalty Opportunities

The biopharmaceutical ecosystem is generating significant demand for capital to fund the ongoing wave of healthcare innovation. Reflecting this positive market backdrop, between 2019 and 2023, the number of in-depth reviews of new royalty opportunities conducted by Royalty Pharma increased by 133%, resulting in an 80% increase in announced annual transaction value (from $2.2 billion in 2019 to $4.0 billion in 2023).

Given Royalty Pharma’s unique role at the heart of funding life sciences innovation, Royalty Pharma believes that there will be significant opportunity to deploy capital and fund innovation, while creating value for its stakeholders. This is reflected in the company’s capital deployment target of $10 billion to $12 billion from 2022 to 2026 and in Royalty Pharma’s expectation that it has the potential over the longer term to double its average annual capital deployment to $4 billion to $5 billion.

Webcast of J.P. Morgan Healthcare Conference

Royalty Pharma will present at the 42nd Annual J.P. Morgan Healthcare Conference at 12:00 p.m. ET / 9:00 a.m. PT tomorrow. The webcast will be accessible from Royalty Pharma’s "Events" page at View Source The webcast will also be archived for a minimum of thirty days.

On January 8, 2024 GC Cell, a fully integrated cell therapy pioneer, reported the approval from both the Australian Human Research Ethics Committee (HREC) and the Korean Ministry of Food and Drug Safety (MFDS) for a Phase 1 Investigational New Drug (IND) trial for its AB-201 cancer treatment, a HER2 targeted chimeric antigen receptor-natural killer (CAR-NK) cell therapy that shows great promise (Press release, GC Cell, JAN 8, 2024, View Source [SID1234639126]). Additionally, GC Cell is excited to reveal a strategic partnership with Lunit, the medical AI leader, to enhance precision and clinical intelligence of AB-201’s efficacy evaluation by applying its advanced AI technology, in addition to traditional Immunohistochemistry (IHC) based assessments. After participating in the US White House’s Cancer Moonshot initiative this past October, both accomplishments represent major milestones in GC Cell’s efforts to revolutionize immunotherapy-based cancer treatment while harnessing AI and digital transformation to fight cancer.

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"Our preclinical studies suggest the possibility that AB-201 causes complete cancer remission and tumor suppression," remarked James Park, CEO of GC Cell. "We are thrilled to move forward to Phase 1 trials — the first Korean company to do so for CAR-NK cell therapy — and we are grateful to Lunit for joining hands with us in our digital transformation and the fight against cancer — now armed with the power of AI. This initiative is part of GC Cell’s broader strategy to integrate digital & AI technology into all aspects of healthcare, from research and development to patient care. I believe it will prepare our company for the upcoming era of digital healthcare."

Promising treatment to begin clinical trials

AB-201, a novel CAR-NK cell therapy targeting solid tumors, represents a breakthrough in cancer immunotherapy, capable of killing malignant cells. While existing NK cell treatments typically dissipate within a few weeks, AB-201 has demonstrated persistence for over three months in preclinical studies, highlighting its potential in managing long-term, advanced cancers. A multi-country study, the Phase 1 trial will evaluate safety and efficacy in 48 patients with HER2-overexpressing breast, gastric, and gastroesophageal junction cancers. GC Cell holds exclusive rights to AB-201 in the Asia Pacific region, while ex-Asia-Pacific rights were licensed to Artiva Biotherapeutics, Inc. in an exclusive partnership.

Lunit partnership for AI-powered pharma development

A recent McKinsey report found that AI has the potential to increase the value of the pharmaceutical R&D industry by six to eleven billion US dollars. Employing the technology, GC Cell anticipates that AI-driven imaging can analyze vast amounts of data with a high degree of precision and consistency while reducing variability by individual interpretation. This leads to a more accurate assessment of HER2 expression levels, which is vital for appropriate treatment decisions. With such quantitative analysis, AI can help standardize the assessment of cancer target expression across different laboratories and geographical locations. This standardization is essential for multicenter clinical trials and for ensuring that patients receive consistent care regardless of where they are treated. It’s a pivotal step in GC Cell’s journey towards a digital future, aligning with the broader healthcare industry’s shift towards technology-enhanced solutions.

About AB-201

AB-201 is an allogeneic HER2-targeted chimeric antigen receptor NK (CAR-NK) cell therapy for the treatment of solid tumors in the outpatient setting with the option for repeat dosing. A novel, high affinity anti-HER2 antibody converted to scFv structure confers highly specific tumor targeting and is coupled with a unique costimulatory structure and IL-15 expression for enhanced activity and persistence. AB-201 has demonstrated potent anti-tumor activity in multiple preclinical HER2-positive tumor model systems. The underlying NK cell is derived from umbilical cord blood donors preselected for advantageous attributes including the high affinity variant of the CD16 receptor and a KIR-B haplotype. AB-201 cell products maintain high expression of CD16, as well as other activating innate cell tumor engaging receptors, enabling the potential for dual targeting therapeutic approaches via monoclonal antibody combinations. The resulting CAR-NK is manufactured at very large scale and cryopreserved in infusion-ready media to enable repeat clinical administrations in the outpatient setting.

GC Cell holds exclusive rights to AB-201 in the Asia-Pacific region, while ex-Asia-Pacific rights were licensed to Artiva Biotherapeutics, Inc. in an exclusive partnership. The U.S. Food and Drug Administration previously cleared Artiva’s investigational new drug (IND) application for AB-201 in 2022.