On November 14, 2024 TriSalus Life Sciences Inc., (Nasdaq: TLSI), an oncology company integrating novel delivery technology with immunotherapy to transform treatment for patients with liver and pancreatic tumors, reported its financial results for the third quarter ended September 30, 2024, and provided a business update (Press release, TriSalus Life Sciences, NOV 14, 2024, View Source [SID1234648426]).

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"We enter the final quarter of 2024 with great momentum, both commercially and clinically, and we are positioned well for an even greater 2025," stated Mary Szela, President and Chief Executive Officer of TriSalus Life Sciences. "Commercially, we enjoyed a strong third quarter highlighted by 42% revenue growth. We recently launched the TriNav LV system to address patients with larger vessels, an opportunity we believe will meaningfully expand our addressable market and provide full access to the $375 million liver embolization market."

"With the successful completion of our Phase 1 dose escalation study enrollment in UM-LM, we are actively pursuing a strategic partnership for nelitolimod," continued Ms. Szela. "This follows the successful presentation of positive Phase 1 results from our PERIO-01 dose escalation study in UM-LM at SITC (Free SITC Whitepaper). Additionally, by mid-2025, we anticipate data from our Phase 1 study in locally advanced pancreatic cancer, which will guide our next steps."

"Our clinical development efforts for the TriNav system have expanded with the launch of the DELIVER program, starting with the PROTECT registry trial for patients with multinodular goiter. There is significant potential to broaden our addressable market by $400 million, and we are committed to providing further updates on PROTECT and additional programs within the DELIVER initiative."

"Finally, we are initiating 2025 guidance that calls for greater than 50% revenue growth, a greater than 20% reduction in operating expenses, positive full-year EBITDA, and positive cash flow in the second half of the year," concluded Ms. Szela.

Third Quarter Business Update

TriNav System Large Vessel Launch

TriSalus recently expanded its portfolio of Pressure-Enabled Drug Delivery (PEDD) devices with the launch of the TriNav LV Infusion System and TriGuide Guiding Catheter to optimize therapeutic delivery for patients with larger vessels. The TriNav LV system is suitable for patients with vessels sized between 3.5 and 5.0 mm and is expected to allow the Company to meaningfully expand its addressable liver embolization market. The TriGuide Guiding Catheter has a larger inner diameter, lubricious inner lining, and reverse curve design to support femoral access for the TriNav LV system, which the Company believes will enhance procedural efficiency. These new products are eligible for the same HCPCS reimbursement codes as existing TriNav products, enabling seamless integration into current billing structures.

DELIVER and PROTECT Updates

During the quarter, TriSalus advanced the DELIVER clinical program, a series of clinical trials designed to demonstrate enhanced safety and efficacy across a broad spectrum of complex, difficult-to-treat patients through investigator-initiated studies, further underscoring the impact of PEDD technology. A key focus of the DELIVER program is to investigate the potential of combining use of the TriNav system with these therapies to enhance effectiveness and address resistance mechanisms in challenging cancers.

The first of these is a registry study called PROTECT (Pressure Enabled Retrograde Occlusive Therapy with Embolization for Control of Thyroid Disease), which has been initiated, and TriSalus intends to enroll 100 patients across five leading academic sites. It is estimated that approximately 5% of adults have multinodular goiters, and the prevalence in adults over 50 is estimated to be up to 50%. The Company estimates that this could expand the addressable market by approximately 50,000 procedures, representing an incremental $400 million market opportunity and putting the Company’s total addressable market at more than $1 billion in the U.S. This new procedure utilizing the TriNav system is also eligible for the same Healthcare Common Procedure Coding System (HCPCS) reimbursement code allowing for seamless integration into current billing approaches.

The Company anticipates opening additional DELIVER studies in the first half of 2025 and will provide more details as those studies commence.

PERIO Trial Update

TriSalus presented Phase 1 results from the PERIO-01 clinical trial at the recent SITC (Free SITC Whitepaper) meeting. This dose escalation trial investigated the use of the PEDD method of nelitolimod in patients with UM-LM. The results suggested that PEDD-administered nelitolimod, combined with immune checkpoint inhibitors, provides promising clinical benefits and durable survival in heavily pretreated patients with UM-LM and a favorable safety profile. The Company is actively exploring strategic partnerships to advance this indication further.

The Company also completed enrollment of 13 patients in its PERIO-03 Phase 1 dose escalation study of nelitolimod in locally advanced pancreatic cancer. Evidence gathered thus far supports a strong safety profile and further exploration of nelitolimod combined with the TriNav pancreatic infusion technology. The Company will outline the next steps once the final data are available in mid-2025.

Financial Results for Q3 2024

Revenue, all from the sale of the TriNav system, was $7.3 million and $21.2 million, respectively, for the three and nine months ended September 30, 2024. These were up 42% and 66%, respectively, compared to the same periods in 2023. Revenue growth was driven primarily by increased selling resources and increased market share.

Gross margins were 86% and 86% for the three and nine months ended September 30, 2024, respectively, compared to 89% and 84%, respectively, for the same periods in 2023. The year-to-date improvement is due to increased factory volumes and improved operational efficiency.

Operating losses were $8.7 million and $28.6 million, respectively, for the three and nine months ended September 30, 2024, respectively, compared to losses of $18.6 million and $40.2 million, respectively, for the same periods in 2023. These amounts include non-cash stock compensation and depreciation expenses of $1.6 million and $4.3 million for the three- and nine-month periods in 2024 and $0.4 million and $0.9 million for the same periods in 2023. Current year reductions in operating losses are due to increased sales, reduced general and administrative expenses due to non-recurrence of prior year costs related to becoming a public company, and reduced research and development spending associated with the ramp-down of clinical trial spending.

Net losses available to common stockholders were $2.4 million and $19.9 million, respectively, for the three and nine months ended September 30, 2024, compared to losses of $1.4 million and $23.7 million, respectively, for the same periods in 2023. Net losses in 2024 include non-cash related gains on change in fair value of various derivatives of $7.3 million and $10.5 million, respectively, for the three and nine months ended September 30, 2024, compared to gains of $17.1 million and $16.4 million, respectively, for the same periods in 2023. The basic and diluted loss per share for the three and nine months ended September 30, 2024, were $0.12 and $0.91, respectively, compared to $0.14 and $5.72 for the three and nine months ended September 30, 2023, respectively.

On September 30, 2024, cash and cash equivalents totaled $11.3 million. The Company expects existing liquidity sources and $25 million of available capacity on the OrbiMed debt facility to provide sufficient cash runway throughout 2025. In addition, the company expects to be EBITDA positive for 2025 and achieve positive cash flow by the second half of 2025, extending total cash runway beyond 2025.

2025 Guidance

The Company is providing guidance for 2025 for the first time, including:

Sales are expected to grow by more than 50% in 2025, driven by further market share increases in the TriNav system, the commercial launch of the TriNav LV system, and the TriNav target market expansion driven by the DELIVER program.
Operating expenses are expected to decline greater than 20% in 2025 due to reductions in R&D associated with completing the PERIO Phase 1 trials and reductions in G&A expenses due to the non-recurrence of certain costs related to becoming a public Company.
The Company expects to achieve positive full-year EBITDA and positive cash flow in the second half of the year.
Conference Call

TriSalus will host a webcast to discuss its third quarter 2024 financial results and business highlights on November 14, 2024, at 9:00 a.m. EST. The webcast can be accessed on the investor relations section of TriSalus’ website at View Source Following the conclusion of the event, a webcast replay will be available on the website. Interested parties participating by phone will need to register using this online form. After registering for the webcast, dial-in details will be provided in an auto-generated e-mail containing a link to the conference phone number and a personal pin.

On November 14, 2024 Cue Biopharma, Inc. (Nasdaq: CUE), a clinical-stage biopharmaceutical company developing a novel class of therapeutic biologics to selectively engage and modulate disease-specific T cells for the treatment of cancer and autoimmune disease, reported a business and financial update for the third quarter of 2024 (Press release, Cue Biopharma, NOV 14, 2024, View Source [SID1234648394]).

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Recent Business Highlights

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Presented positive updated data from the Phase 1 trials of CUE-101 and CUE-102 at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper)’s 39th Annual Meeting (SITC 2024) November 6-10
Updated data from Phase 1 trial of CUE-101 in combination with KEYTRUDA (pembrolizumab) continued to demonstrate enhanced benefit versus historical studies with pembrolizumab alone. Key findings included an objective response rate (ORR) of 46%, 12-month overall survival (OS) of 91.3% and a median overall survival (mOS) of 21.8 months in first line (1L) HPV+ R/M HNSCC patients, as well as an ORR of 50% in the subset of 1L patients with low PD-L1 expression (combined positive score (CPS) 1-19)
Updated data from Phase 1 monotherapy trial of CUE-102 included evidence of selective expansion of WT1-specific T cells and anti-tumor activity, as well as a favorable tolerability profile with no dose limiting toxicities (DLTs) in patients with Wilms’ Tumor 1 (WT1)-expressing colorectal, gastric, ovarian and pancreatic cancers
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Demonstrated disease control rate (DCR) of 67% in late-stage pancreatic cancer patients treated with CUE-102 monotherapy, including an unconfirmed partial response (PR) with a 40% decrease in tumor burden
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Announced pricing of $12.0 million public offering
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Appointed industry veteran Lucinda Warren as Chief Business Officer
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Continued advancement of preclinical programs, CUE-401 for induction and expansion of regulatory T cells, in collaboration with Ono Pharmaceutical, and CUE-501 for B cell depletion, positioning both programs towards drug candidate selection

"We are very pleased with the validating updated clinical data from our Phase 1 trials for both CUE-101 and CUE-102," said Daniel Passeri, chief executive officer of Cue Biopharma. "Importantly, we believe the maturing data further supports and strengthens our competitive differentiation and positioning for selective modulation of disease-specific T cells. This data further bolsters our confidence that the CUE-100 series, exemplified by CUE-101 and CUE-102, represents the potential of establishing a new standard of care for cancer patients. We are also very pleased with the continued progress of our preclinical autoimmune programs, both of which have moved closer towards drug candidate selection."

On November 14, 2024 Phio Pharmaceuticals Corp. (Nasdaq: PHIO) is a clinical-stage biotechnology company exploring new pathways towards a cancer-free future by using its INTASYL siRNA gene silencing technology designed to make the body’s immune cells more effective in killing cancer cells reported its financial results for the quarter ended September 30, 2024 and provided a business update (Press release, Phio Pharmaceuticals, NOV 14, 2024, View Source [SID1234648410]).

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Recent Corporate Updates

The Phase 1b dose escalation clinical trial for our lead product candidate, PH-762, previously received a positive safety recommendation to advance to the next highest dose from the Safety Monitoring Committee (SMC) for the first cohort. The second cohort is fully enrolled, and screening for the 3rd cohort will open on 10 Dec 2024. Each enrolled subject receives four (4) intratumoral (IT) doses of PH-762. The currently enrolled patients were diagnosed with cutaneous squamous cell carcinoma (6 patients) and metastatic melanoma (1 patient). Two of the three patients in the second cohort have completed the treatment and the follow-up phase.

At Day 36 (tumor excision), while each patient in the first cohort had stable disease, the following results were described for two patients who have completed treatment in the second cohort:

· Complete response (100% tumor clearance) reported for 1 patient with cutaneous squamous cell carcinoma
· Partial response (90% tumor clearance) reported for 1 patient with cutaneous squamous cell carcinoma

A sixth clinical trial site, Paradigm Clinical Research Centers in San Diego, CA, was selected for the Phase 1b study of PH-762 and will join the other five sites engaged in the study: Banner MD Anderson Cancer Center in Gilbert, Arizona. The George Washington University-Medical Faculty Associates in Washington, D.C; The University of Pittsburgh Medical Center (UPMC) Department of Dermatology; Integrity Research Clinical Associates in Delray Beach, Florida; and Centricity Research in Dublin Ohio.

Cancer Immunology, Immunotherapy, a peer reviewed journal, highlighted the INTASYL compound PH-804. The article, issued on Oct 3, 2024, was authored by M Maxwell et al. entitled "INTASYL Self-Delivering RNAi Decreases TIGIT Expression, Enhancing NK Cell Cytotoxicity: A Potential Application to Increase the Efficacy of NK-Adoptive Cell Therapy Against Cancer".

Presented new data on Phio’s INTASYL self-delivering siRNA technology applications for immuno-oncology therapy at several conferences:

· 9th Annual CAR-TCR Summit held on Sept 17th showcased INTASYL siRNA technology-an innovative and flexible solution designed specifically to enhance cell potency and improve overall yield for adoptive cell therapy.
· 20th Annual Oligonucleotide Therapeutics Society (OTS) held on October 8th showcased INTASYL’s role in helping immune cells target and kill cancer cells.
· The American Society of Gene and Cell Therapy’s 2024 Advancing Cancer Conference held Oct 16-17 reported on INTASYL Compound PH-894: A potent and specific silencing of BRD4 in NK Cells.
· 39th Annual meeting of the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) held November 9th reported on new clinical data from the Phase 1b clinical trial for its INTASYL Compound PH-762.

Phio Pharmaceuticals is currently participating in the Renmark Financial Virtual Non-Deal Roadshow series that features Phio’s CEO and Chairman of the Board, Robert Bitterman, presentation on its INTASYL technology. The presentations are directed to the retail investor communities including those in the Chicago, Denver, Dallas, Atlanta, and Boston areas. These roadshows have been on-going since late August 2024.

Financial Results

Cash Position

At September 30, 2024, the Company had cash of $5.4 million as compared with $8.5 million at December 31, 2023.

In July 2024, the Company entered into inducement letter agreements with certain holders of the Company’s existing warrants to purchase up to an aggregate of 545,286 shares of common stock at a reduced exercise price of $5.45 per share. In consideration for the immediate exercise of the existing warrants, the Company agreed to issue five and one-half year term Series C warrants to purchase up to 583,098 shares of common stock and eighteen month term Series D warrants to purchase up to 507,474 shares of common stock, both at an exercise price of $5.45. The net proceeds to the Company were approximately $2.6 million, after deducting placement agent fees and offering expenses.

Research and Development Expenses

Research and development expenses were $0.6 million for the three months ended September 30, 2024 as compared with $1.8 million for the three months ended September 30, 2023, a decrease of 64%. The decrease was primarily driven by non-recurring clinal program direct start-up costs in the 3rd Quarter 2023 and reductions in research compensation attributable to reductions in headcount of participants during the 3rd Quarter 2024.

General and Administrative Expenses

General and administrative expenses were $0.95 million for the three months ended September 30, 2024 as compared with $0.97 million for the three months ended September 30, 2023, a decrease of 2%. The decrease was primarily due to decreases in salary-related expenses compared to the prior year period.

Net Loss

Net loss was $1.5 million for the three months ended September 30, 2024 as compared with $2.8 million for the three months ended September 30, 2023. The decrease in net loss was primarily due to the changes in research and development expenses, as described above.

On November 14, 2024 Curis, Inc. ("Curis") (NASDAQ: CRIS), a biotechnology company focused on the development of emavusertib (CA-4948), an orally available, small molecule IRAK4 inhibitor, reported its business update and financial results for the quarter ended September 30, 2024 (Press release, Curis, NOV 14, 2024, View Source [SID1234648395]).

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"We continue to make excellent progress across our clinical programs. We are especially excited about the recent R/R PCNSL data released in September which continue to demonstrate the activity of emavusertib in combination with ibrutinib in salvage-line patients and that the CR/CRu responses appear to be durable," said James Dentzer, Curis Chief Executive Officer. "We are also excited to present additional clinical data in our TakeAim Leukemia study next month at ASH (Free ASH Whitepaper). We believe the positive momentum as we finish the year sets us up well for 2025."

Third Quarter 2024 and Recent Operational Highlights

Emavusertib (IRAK4 Inhibitor)

TakeAim Lymphoma

In September, at the 3rd Annual IRAK4 Symposium in Cancer, the Company released preliminary efficacy data in 10 response-evaluable patients who had progressed on treatment with a BTKi. The data showed 3 complete responses (CR), 1 unconfirmed complete response (CRu) and 2 partial responses (PR). The duration of response for 3 of the 4 patients with a CR/CRu was greater than 6 months.
The Company is actively engaged in discussions with regulatory authorities to gain alignment on the registrational path in PCNSL.
TakeAim Leukemia

The Company will have both an oral presentation and a poster presentation at the 66th American Society of Hematology (ASH) (Free ASH Whitepaper) annual meeting in December.

Oral Presentation:
Session Name: 616. Acute Myeloid Leukemias: Investigational Drug and Cellular Therapies: New Treatment Approaches for AML
Session Date: Monday, December 9, 2024
Presentation Time: 11:30 AM
Room: Manchester Grand Hyatt San Diego, Grand Hall B
Publication Number: 737
Title: Preliminary Safety, Efficacy, and Molecular Characterization of Emavusertib (CA-4948) in Relapsed/Refractory Acute Myeloid Leukemia Patients

Poster:
Session Name: 637. Myelodysplastic Syndromes: Clinical and Epidemiological: Poster II
Session Date: Sunday, December 8, 2024
Presentation Time: 6:00 PM – 8:00 PM
Location: San Diego Convention Center, Halls G-H
Publication Number: 3225
Title: Preliminary Safety, Efficacy and Molecular Characterization in Patients with Higher-Risk Myelodysplastic Syndrome Treated with Single Agent Emavusertib (CA-4948)

Corporate

In October 2024, Curis completed a registered direct offering and concurrent private placement of unregistered warrants ("October 2024 Offerings") with net proceeds of approximately $10.8 million.

Third Quarter 2024 Financial Results

For the third quarter of 2024, Curis reported a net loss of $10.1 million or $1.70 per share on both a basic and diluted basis as compared to $12.2 million or $2.13 per share on both a basic and diluted basis, for the same period in 2023. Curis reported a net loss of $33.8 million or $5.77 per share on both a basic and diluted basis, for the nine months ended September 30, 2024 as compared to a net loss of $35.7 million or $6.96 per share on both a basic and diluted basis for the same period in 2023.

Revenues for the third quarter of 2024 were $2.9 million as compared to $2.8 million for the same period in 2023. Revenues for both periods consist of royalty revenues from Genentech and Roche’s sales of Erivedge. Revenues for the nine months ended September 30, 2024 and 2023 were $7.6 million and $7.3 million, respectively.

Research and development expenses were $9.7 million for the third quarter of 2024, as compared to $10.4 million for the same period in 2023. The decrease was primarily attributable to lower consulting and employee related costs. Research and development expenses were $29.6 million for the nine months ended September 30, 2024, as compared to $29.5 million for the same period in 2023.

General and administrative expenses were $3.8 million for the third quarter of 2024, as compared to $4.8 million for the same period in 2023. The decrease was primarily attributable to lower legal and employee related costs. General and administrative expenses were $13.4 million for the nine months ended September 30, 2024, as compared to $13.8 million for the same period in 2023.

Other income, net was $0.5 million for the third quarter of 2024, as compared to $0.2 million for the same period in 2023. The increase was primarily attributable to a decrease in the non-cash expense related to the sale of future royalties. Other income, net was $1.8 million for the nine months ended September 30, 2024, as compared to $0.4 million for the same period in 2023.

Including the impact of the October 2024 Offerings, Curis’s cash and cash equivalents totaled $31.6 million, and the Company had approximately 8.5 million shares of common stock outstanding. Curis expects its existing cash and cash equivalents will enable its planned operations into mid-2025.

Conference Call Information

Curis management will host a conference call today, November 14, 2024, at 8:30 a.m. ET, to discuss the business update and these financial results.

To access the live conference call, please dial 800-836-8184 from the United States or 1-646-357-8785 from other locations, to access the webcast login to View Source shortly before 8:30 a.m. ET. The webcast can also be accessed via the Curis website in the ‘Investors’ section.

On November 14, 2024 Poseida Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage allogeneic cell therapy and genetic medicines company advancing differentiated non-viral treatments for patients with cancer, autoimmune and rare diseases, reported plans to share progress across its clinical- and earlier-stage pipeline of differentiated T stem cell memory cells (TSCM)-rich allogeneic CAR-T therapies in oncology and autoimmune diseases during a virtual R&D Day to be held today at 10:00am ET /7:00am PT (Press release, Poseida Therapeutics, NOV 14, 2024, View Source [SID1234648411]).

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"We believe Poseida is well positioned to be a cell therapy leader based on the unique capabilities of our proprietary non-viral technology platform and our allogeneic TSCM-rich CAR-T approach," said Kristin Yarema, Ph.D., President and Chief Executive Officer of Poseida Therapeutics. "We are organizing our pipeline around three pillars – hematologic malignancies, solid tumors and autoimmune diseases – with multiple candidates in each that give us a wide range of opportunities. In the emerging field of cell therapy for autoimmune diseases, we are optimistic that our platform can build upon the early success seen with autologous CAR-T by offering an off-the-shelf option that would expand accessibility and address the potential drivers of relapse with more complete B cell depletion."

"Building on the advantages of our non-viral allogeneic TSCM-rich approach, we are implementing several advanced technologies aimed at bringing the benefits of CAR-T therapy to patients with solid tumors," said Devon J. Shedlock, Ph.D., Chief Scientific Officer, Cell Therapy at Poseida Therapeutics. "This includes our collaboration with Astellas, which brings together the unique technologies from both companies to create a new class of CAR-T, convertibleCARs, which employs multi-antigen targeting and other enhancements to improve CAR-T potency and persistence."

The event will feature presentations by members of Poseida’s management team and will include a fireside chat with Peter Sandor, M.D., EVP and Head of Corporate Strategy at Astellas Pharma, that will cover the research collaboration and license agreement between Xyphos Biosciences, Inc., (a wholly owned subsidiary of Astellas, "Xyphos") and Poseida to develop novel convertibleCAR programs by combining the innovative cell therapy platforms from each of the companies. Poseida management will participate in a Q&A session at the end of the program.

Cell therapy technology platform: Poseida has built a full set of non-viral capabilities to design and develop allogeneic, TSCM-rich CAR-T therapies. TSCM cells are considered ideal for CAR-T therapy because they are long-lived, multi-potent and self-replicating, with the potential for an improved safety and efficacy profile. This compares to other approaches, which either use a different cell type or drive T cell differentiation (and therefore less stemness) as part of the process to manufacture the CAR-T cells. The key elements of Poseida’s approach include:

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Non-viral, transposon-based gene insertion system preferentially integrates genes into naïve and TSCM cells, with a high cargo capacity that allows for adding multiple genes to enhance functionality and add safety features

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Cas-CLOVER gene editing system preserves the TSCM cell type and operates effectively in resting T cells. It offers approximately 25-times greater fidelity than CRISPR-Cas9, supporting improved safety and quality

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Wholly-owned, onsite GMP manufacturing facility positioned to serve discovery through commercial needs across the Company’s pipeline. The Company’s Booster Molecule has enabled a scalable, lower cost manufacturing approach with the proven ability to generate cell yield up to over 100 doses per batch

Emerging leadership in allogeneic CAR-T for hematologic malignancies: Poseida will provide an overview of recently reported interim Phase 1 results for P-BCMA-ALLO1, its lead CAR-T program targeting BCMA for the treatment of multiple myeloma. New preclinical data suggest that P-BCMA-ALLO1 effectively targets mutations that are known to arise in patients with relapse after prior anti-BCMA therapies. P-BCMA-ALLO1 is part of Poseida’s collaboration with Roche and is currently enrolling a Phase 1b dose expansion study, which will be outlined in the presentation. In addition, the Company will highlight its emerging pipeline programs for hematologic malignancies:

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P-CD19CD20-ALLO1 is the Company’s first dual CAR-T program targeting CD19 and CD20 for the treatment of B-cell malignancies in collaboration with Roche. New preclinical data demonstrate that P-CD19CD20-ALLO1 delivers high in vitro potency and strong in vivo antitumor activity for either CD19 or CD20 single-positive target cells, as well as double-positive targets. A Phase 1 clinical trial is enrolling patients with selected B-cell malignancies, with initial clinical data anticipated in 2025. Additional information about the trial is available at www.clinicaltrials.gov using identifier: NCT06014762.

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Beyond hematologic malignancies, preclinical data also demonstrate robust in vitro activity against patient-derived B cells across multiple autoimmune diseases

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P-CD70-ALLO1 is a preclinical program targeting CD70 for the treatment of diseases including acute myeloid leukemia (AML). New preclinical data demonstrate P-CD70-ALLO1’s robust anti-AML effect, with no toxicity to hematopoietic stem cells. In addition, there is a growing body of clinical and preclinical evidence that targeting CD70 with cell therapy may be an effective treatment for solid tumors. Roche has an option to add this as a potential new program to the collaboration

Expanding allogeneic TSCM-rich CAR-T to autoimmune disease: Poseida’s cell therapy technology platform has the potential to create CAR-T therapies designed to address the challenges with existing emerging approaches to applying cell therapy to autoimmune disease, including autologous CAR-T, in vivo CAR-T and T cell engagers. The Company’s lead program for autoimmune disease is P-BCMACD19-ALLO1, a dual CAR-T targeting BCMA and CD19. New preclinical data demonstrate P-BCMACD19-ALLO1’s potential in both autoimmune disease and oncology:

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Demonstrated robust in vitro killing of patient-derived B cells across multiple autoimmune diseases, including rheumatoid arthritis (RA), systemic lupus erythematosus (SLE) and multiple sclerosis (MS), which collectively affect more than 5 million people in the U.S.

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Achieved dose-dependent depletion of primary human B cells in a humanized mouse model generated with human CD34+ cells

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Effectively eliminated primary human CD81+CD19+ multiple myeloma progenitor cells from patient bone marrow samples, addressing cells associated with relapse where BCMA-only targeted therapies were ineffective

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Demonstrated ability of P-BCMACD19-ALLO1 and P-BCMA-ALLO1 to kill tumor cells expressing known mutant forms of BCMA, which are linked to relapse in patients treated with autologous CAR-T and bispecific T cell engager therapies directed at BCMA

Poseida is conducting IND-enabling studies for P-BCMACD19-ALLO1 and plans to file one or more INDs for an autoimmune disease indication with the U.S. Food and Drug Administration.

Addressing historical barriers for CAR-T in solid tumors through a broad array of bold, innovative technologies enabled by the Company’s platform: There are currently no CAR-T therapies approved for solid tumors, with several key factors believed to be roadblocks: 1) antigen heterogeneity; 2) differing lymphodepletion needs compared to hematologic malignancies to enable CAR-T cell engraftment, tracking, and infiltration; 3) on-target off-tumor toxicity; and 4) hostile tumor microenvironment. Poseida is approaching these challenges with a suite of technologies across multiple solid tumor programs:

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P-MUC1C-ALLO1 is Poseida’s lead solid tumor CAR-T program targeting MUC1-C, a membrane protein overexpressed in many epithelial cancers. A Phase 1 clinical trial is enrolling patients with treatment-resistant breast, ovarian, colorectal and other solid tumors, with ongoing exploration of P-MUC1C-ALLO1 dosing and lymphodepletion regimens. A clinical data update is planned for the European Society for Medical Oncology Immuno-Oncology Congress 2024 (ESMO-IO) taking place in Geneva December 11-13, 2024. A patient case study from the Phase 1 trial demonstrated a 42% decrease in paraesophageal lymph node size with prolonged stable disease for nearly a year in a heavily pretreated appendiceal carcinoma patient. New preclinical data suggest adding low-dose methotrexate to standard lymphodepletion may enhance CAR-T expansion and persistence

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P-PSMA-ALLO1 is a preclinical program targeting PSMA for prostate cancer. In preclinical models, P-PSMA-ALLO1’s dual CAR format showed superior in vivo anti-tumor activity and cytotoxicity compared to single and tandem binder CAR-Ts

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Poseida and Xyphos are developing allogeneic convertibleCARs for solid tumors, combining Poseida’s allogeneic CAR-TSCM platform with Xyphos’ ACCEL technology to create controlled, long lasting, and highly active CAR-T therapies. New preclinical data shows positive results for an allogeneic convertibleCAR-TSCM targeting universal inert natural killer group 2 member D receptor (iNKG2D), paired with a solid tumor antigen-targeting MicAbody. Poseida and Xyphos are working to optimize this convertibleCAR and MicAbody pairing, and other platform technologies designed to maximize potency and persistence

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Poseida will highlight its allogeneic CAR-TCR-T cells to address antigen heterogeneity in solid tumors. The Company recently presented new preclinical data demonstrating enhanced potency to better target solid tumors at the Society for Immunotherapy of Cancer (SITC) (Free SITC Whitepaper) 39th Annual Meeting. Preclinical data highlight the potential of a combination of CAR-TCR-T cell treatment followed by a T cell engager (TCE). In this model, MUC1C-CAR and NY-ESO-1-TCR T cells effectively controlled a primary tumor (MUC1C+/NY-ESO-1+/CD70-), and later were reactivated and re-directed by a CD70 TCE to control challenge by a secondary tumor (MUC1C-/NY-ESO-1-/CD70+)

LOGO

In-house manufacturing to support Poseida’s broad cell therapy pipeline: the Company will highlight the capabilities of its GMP facility and team, including:

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New data demonstrated high-purity apheresis across different healthy donors, with consistency in CAR-T manufacturing (cellular expansion, gene editing, final phenotype) across collections from the same donor

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Future manufacturing platform enhancement opportunities including AI-assisted donor screening to improve efficiency; improving electroporation unit operations to improve gene editing efficiency and subsequent cell health; and leveraging dynamic bioreactor environments to increase yields

Video Webcast and Replay

This virtual event and access to the live webcast is available through the following registration link: View Source

Registration for this virtual event and access to a replay of the live webcast will be available on the Investors & Media section of www.poseida.com. A replay of the webcast will be available for approximately 90 days following the presentation.