On January 5, 2024 Vor Biopharma presented its corporate presentation (Presentation, Vor BioPharma, JAN 5, 2024, View Source [SID1234639023]).

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On January 5, 2024 TG Therapeutics, Inc. (NASDAQ: TGTX), reported that Michael S. Weiss, the Company’s Chairman and Chief Executive Officer, will present at the 42nd Annual J.P. Morgan Healthcare Conference. The presentation is scheduled to take place on Tuesday, January 9, 2024, at 4:30 PM PT (Press release, TG Therapeutics, JAN 5, 2024, View Source [SID1234639022]).

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A live webcast of this presentation will be available on the Events page, located within the Investors & Media section, of the Company’s website at View Source A replay of the webcast will be available on TG’s website following the event.

On January 5, 2024 Sana Biotechnology, Inc. (NASDAQ: SANA), a company focused on changing the possible for patients through engineered cells, reported the U.S. Food and Drug Administration (FDA) has cleared the company’s Investigational New Drug (IND) application to initiate a study of SC262 in patients with relapsed or refractory B-cell malignancies, initially in patients who have received prior CD19-directed CAR T therapy (Press release, Sana Biotechnology, JAN 5, 2024, View Source [SID1234639020]).

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Engineered CAR T cell therapies for B-cell malignancies use binders to target proteins expressed on the surface of B cells. One such protein, CD19, has been the target of all approved autologous CAR T therapies for B-cell lymphoma and B-cell acute lymphoblastic leukemia to date. Unfortunately, incomplete responses or relapses occur in approximately 60% of CD19 CAR T-treated patients. CD22, which is also a B-cell surface protein, has emerged as an alternative to address failure to achieve durable complete responses with CD19-directed CAR T therapy. SC262 expresses the same CAR, including the same CD22 binder, used in CD22-directed CAR T therapies tested in multiple academic clinical trials. To date, these trials have shown durable complete responses in a substantial number of patients in the relapse setting following treatment with a CD19-directed CAR T therapy.

"Patients who have failed a CD19-directed CAR T therapy represent a significant unmet need, and this population is growing as more patients receive these therapies," said Doug Williams, PhD, Sana’s President of Research and Development. "SC262 represents an important potential option for these patients and is the next step in building Sana’s hypoimmune CAR T therapy platform. Over the past twelve months, Sana has received three IND regulatory clearances, as well as supported the authorization of an investigator-sponsored CTA, to begin new studies utilizing our hypoimmune platform in seven different indications in oncology, B-cell mediated autoimmune diseases, and type 1 diabetes. We look forward to presenting data from all of these studies this year, including initial proof of concept data for SC262 later this year."

About SC262 in B-cell Malignancies
SC262 is a hypoimmune, CD22-directed allogeneic CAR T cell therapy derived from healthy donor CD4+ and CD8+ T cells. SC262 is developed with Sana’s hypoimmune platform, which is designed to overcome the immunologic rejection of allogeneic cells and may result in longer CAR T cell persistence and a higher rate of durable complete responses for patients with B-cell malignancies. CD22 is expressed on lymphoma and leukemia cells in most patients with these diseases, including those that have failed CD19- and/or a CD20-directed therapies. SC262 is initially being explored in patients that have failed a previous CD19-directed therapy. The hypoimmune technology includes disruption of major histocompatibility (MHC) class I and MHC class II expression to allow cells to evade the adaptive immune system, which includes antibody and T cell responses, as well as overexpression of CD47 to evade the innate immune cell system, in particular macrophages and natural killer (NK) cells. The company has presented data across multiple preclinical models highlighting the potential of this platform to cloak cells from immune recognition and the potential of SC262 as a therapeutic for patients with B-cell malignancies.

About Hypoimmune Platform
Sana’s hypoimmune platform is designed to create cells ex vivo that can "hide" from the patient’s immune system to enable the transplant of allogeneic cells without the need for immunosuppression. We are applying the hypoimmune technology to both donor-derived allogeneic T cells, with the goal of making potent and persistent CAR T cells at scale, and pluripotent stem cells, which can then be differentiated into multiple cell types at scale. Preclinical data published in peer-reviewed journals demonstrate across a variety of cell types that these transplanted allogeneic cells are able to evade both the innate and adaptive arms of the immune system while retaining their activity. Our most advanced programs utilizing this platform include an allogeneic CAR T program targeting CD19+ cancers, an allogeneic CAR T program for B-cell mediated autoimmune diseases, an allogeneic CAR T program targeting CD22+ cancers, and stem-cell derived pancreatic islet cells for patients with type 1 diabetes.

On January 5, 2024 Personalis, Inc. (Nasdaq: PSNL), a leader in advanced genomics for precision oncology, reported unaudited preliminary revenue for the fourth quarter and full year ended December 31, 2023 (Press release, Personalis, JAN 5, 2024, View Source [SID1234639019]).

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Preliminary Fourth Quarter Revenue and Cash Balance

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Preliminary total company revenue is estimated to be $19.7 million in the fourth quarter of 2023, an increase of 18% compared with $16.7 million in the fourth quarter of 2022

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Preliminary revenue from pharma tests, enterprise sales, and other customers is estimated to be $18.7 million in the fourth quarter of 2023 compared with $15.8 million in the fourth quarter of 2022; revenue from enterprise customers includes revenue from Natera which is estimated to be $7.1 million in the fourth quarter of 2023, compared with $8.2 million from Natera in the fourth quarter of 2022

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Preliminary revenue from the Veteran’s Administration Million Veteran’s Program (VA MVP) is estimated to be $1.0 million in the fourth quarter of 2023, compared with $0.9 million in the fourth quarter of 2022

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Preliminary cash, cash equivalents, and short-term investments is estimated to be $114.0 million as of December 31, 2023, and is expected to last through the first quarter of 2026, as a result of 2023 cost-cutting measures which reduced expenses by approximately $35 million annually

Preliminary Full Year 2023 Revenue and Cash Usage

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Preliminary total company revenue is estimated to be $73.5 million in the full year of 2023, an increase of 13% compared with $65.0 million in the full year of 2022

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Preliminary revenue from pharma tests, enterprise sales, and other customers is estimated to be $64.1 million in the full year of 2023 compared with $56.6 million in the full year of 2022; revenue from enterprise customers includes revenue from Natera which is estimated to be $31.7 million in the full year of 2023, compared with $26.6 million from Natera in the full year of 2022

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Preliminary revenue from the VA MVP is estimated to be $9.4 million in the full year of 2023, compared with $8.4 million in the full year of 2022

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Cash usage is expected to be approximately $54 million in the full year of 2023, reduced from $119 million in 2022

"Our strong fourth quarter performance caps off a remarkable year where we delivered on our commitments to investors," stated Chris Hall, President and CEO of Personalis. "To touch on a few highlights, over this past year we launched our NeXT Personal Dx ultrasensitive MRD product, deepened clinical evidence to support obtaining reimbursement, presented unprecedented clinical evidence for early-stage lung cancer detection at medical conferences, expanded our relationships with research collaborators, and forged a new strategic commercial partnership with Tempus to commercialize and ramp our MRD product, all while reducing our annualized expenses by $35 million. I’m very proud of our progress and we expect to further leverage our clinical and commercial strength to drive more success in 2024."

The above information is preliminary and subject to Personalis’ normal quarter and year-end accounting procedures and external audit by the company’s independent registered public accounting firm. In addition, these preliminary unaudited results are not a comprehensive statement of the company’s financial results for the year ended December 31, 2023, should not be viewed as a substitute for full, audited financial statements prepared in accordance with generally accepted accounting principles, and are not necessarily indicative of the company’s results for any future period.

On January 5, 2024 Nimbus Therapeutics, LLC ("Nimbus Therapeutics" or "Nimbus"), a biotechnology company that designs and develops breakthrough medicines through its powerful computational drug discovery engine, reported the advancement and expansion of its pipeline with the addition of discovery programs targeting innate immunity pathways (Press release, Nimbus Therapeutics, JAN 5, 2024, View Source [SID1234639017]). These programs, targeting the salt-inducible kinase (SIK) family and cyclic GMP-AMP synthase (cGAS), represent promising opportunities to leverage Nimbus’ industry-leading computational and structure-based drug design expertise to develop highly selective, potent medicines addressing areas of significant unmet need.

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"Building on the success of our TYK2 program, we are broadening our drug discovery engine to unlock new difficult-to-drug targets with compelling biology," said Peter Tummino, Ph.D., Chief Scientific Officer of Nimbus. "SIK and cGAS are critical targets in highly prevalent diseases that are well suited to Nimbus’ structure-based drug design approach. We look forward to advancing our discovery and development programs across oncology, immunology, and metabolism to deliver transformative medicines to patients."

Nimbus is advancing its Phase 1/2 trial (NCT05128487) of NDI-101150, a small molecule inhibitor of hematopoietic progenitor kinase 1 (HPK1), and has reported positive dose escalation data showing potential monotherapy clinical benefit for patients with solid tumors. Furthermore, the company expects to initiate IND-enabling activities this year for its oncology program targeting Werner syndrome helicase (WRN). In collaboration with Eli Lilly and Company, Nimbus continues to progress the development of novel targeted therapies that activate AMPK to potentially treat a broad range of metabolic disorders.

Nimbus continues to expand its platform capabilities with ongoing investments in cutting-edge technology for drug discovery. Alongside proprietary computational tools which the company has developed in-house, Nimbus’ platform leverages state-of-the-art technology through collaborations such as its recently announced partnership with Anagenex, a leader in generative AI for drug design.

Jeb Keiper, M.S., MBA, Nimbus’ Chief Executive Officer, will provide an overview of the company’s progress and pipeline and anticipated milestones for 2024 and beyond at the 42nd Annual J.P. Morgan Healthcare Conference on Monday, January 8, 2024 at 7:30 am PT.