Poseida Therapeutics Highlights Recent Progress, Strategic Priorities and Anticipated 2024 Key Milestones
Reported positive early clinical data for allogeneic myeloma cell therapy P-BCMA-ALLO1 at ASH 2023 supporting potential of TSCM-rich allogeneic CAR-T to offer a differentiated and compelling efficacy, safety, and reliability profile that could compete with autologous CAR-T
Plan to report clinical data updates for P-BCMA-ALLO1and P-MUC1C-ALLO1 allogeneic CAR-T programs at scientific meetings in 2024
Expect to dose first patient in the Phase 1 P-CD19CD20-ALLO1 trial in early 2024
Company to host Gene Therapy R&D Day
"In 2023, we made significant progress in delivering on the promise of our pipeline of allogeneic cell therapies through both substantial manufacturing advancements and compelling clinical data. At the ASH Annual Meeting, we were thrilled to highlight the ability of P-BCMA-ALLO1, our TSCM-rich CAR-T therapy for multiple myeloma, to offer a compelling emerging efficacy and safety profile that we believe will be competitive with autologous cell therapies and bispecific antibodies, and to treat patients without lengthy waiting times," said
"As we work to build on this momentum in 2024, we plan to report interim Phase 1 data in both our BCMA and MUC1C programs as well as dose the first patient in our allogeneic CD19CD20 dual CAR program targeting B-cell malignancies, which is partnered with Roche. We also expect to advance IND-enabling studies directed toward our wholly-owned allogeneic PSMA program in prostate cancer. In our gene therapy portfolio, we look forward to providing a comprehensive strategic update at our upcoming gene therapy-specific R&D Day in April, as we continue advancing our non-viral DNA delivery platform to unlock potential for genetic diseases. We believe we are entering a new era of cell and gene therapies, and I am thrilled to step into the role of President and CEO of
Cell Therapy Programs
P-BCMA-ALLO1 is a potential first-in-class allogeneic, TSCM-rich CAR-T product candidate being developed to target B-cell maturation antigen (BCMA) for the treatment of relapsed/refractory multiple myeloma (R/R MM) in partnership with Roche. The Company is currently evaluating P-BCMA-ALLO1 in a Phase 1 clinical trial and recently shared positive early safety and preliminary efficacy data at the 65th
Data highlighted at the meeting showed that P-BCMA-ALLO1 was a well-tolerated off-the-shelf therapy with a favorable emerging safety profile, delivered to 100% of patients in the intent-to-treat population with no use of bridging chemotherapy or other anti-myeloma bridging therapies. There were no cases of GvHD or dose-limiting toxicities and low incidences of CRS and neurotoxicity observed (all ≤ Grade 2). The data showed an 82% overall response rate (ORR) and deep clinical responses, including stringent complete responses (sCRs) in MRD-negative patients, from the off-the-shelf, allogeneic BCMA-targeted CAR-T in heavily pretreated patients in study arms receiving adequate lymphodepletion. Of those patients, a 100% ORR was seen in patients who were not previously treated with a BCMA-targeted bispecific T cell-engaging antibody. The median time from study enrollment to start of lymphodepletion chemotherapy was 1 day and was 7 days to P-BCMA-ALLO1 infusion. Preliminary data show allogeneic TSCM-rich CAR-T cells trafficking to bone marrow, differentiating to cell-killing effector T cells and persisting at least 6 weeks after treatment, which supports the hypothesis of cell persistence at tumor-relevant sites. At the time of data cutoff, 8 of 9 responding patients were still in clinical response.
P-CD19CD20-ALLO1 is an allogeneic, TSCM-rich CAR-T product being developed to target B-cell malignancies in partnership with Roche. P-CD19CD20-ALLO1 is the Company's first dual CAR program and contains two fully functional CAR molecules to target cells that express either CD19 or CD20 or both. The Company believes P-CD19CD20-ALLO1 is the first known dual allogeneic CAR-T directed against CD19 and CD20 to receive IND clearance from the FDA. The Company expects to dose the first patient in the Phase 1 trial of P-CD19CD20-ALLO1 for B-cell malignancies in early 2024 and provide a data update later in the year, subject to coordination with Roche.
P-MUC1C-ALLO1 is an allogeneic, TSCM-rich CAR-T product candidate targeting the C-terminal domain of the mucin-1 protein (MUC1C), which is prevalent in solid tumors of epithelial origin, including breast, ovarian, and multiple other cancers. The Company is currently evaluating P-MUC1C-ALLO1 in a Phase 1 clinical trial with a basket study design. Building on learnings from the allogeneic BCMA CAR-T program, the Company is evaluating multiple dosing strategies, including higher lymphodepletion, cell dose, and schedule in its MUC1C program.
P-PSMA-ALLO1 is an allogeneic CAR-T product candidate targeting prostate-specific membrane antigen, or PSMA, to treat prostate cancer. This allogeneic program utilizes VH-based binding technology, similar to the approach used in the Company's P-BCMA-ALLO1 program, but targeting PSMA. When compared to the previous autologous PSMA program that used a Centyrin binder, in preclinical models, enhanced anti-tumor activity was observed using this newer binder. Based upon progress and learnings from its autologous P-PSMA-101-001 clinical trial and across its allogenic platform, the Company plans to advance IND-enabling work for P-PSMA-ALLO1 in 2024.
The Company plans to continue early pipeline research activities and advance IND-enabling work for other allogeneic pipeline programs, including exploring opportunities in autoimmune disease.
Gene Therapy Programs
At its Gene Therapy R&D Day event in
The Company is advancing its P-FVIII-101 preclinical program, a fully non-viral liver-directed gene therapy combining
P-OTC-101 is a liver-directed gene therapy for the treatment of urea cycle disease caused by congenital mutations in the ornithine transcarbamylase (OTC) gene. The Company is developing the P-OTC-101 program utilizing a hybrid delivery system (AAV+LNP) and working on an updated timeline for the program. P-OTC-101 received orphan drug designation from the FDA in
P-PAH-101 is a liver-directed gene therapy for the treatment of Phenylketonuria (PKU), an inherited genetic disorder caused by mutations in the phenylalanine hydroxylase (PAH) gene resulting in a buildup of phenylalanine in the body. The Company is developing P-PAH-101 program utilizing a hybrid system (AAV+LNP) and is currently in preclinical development.
Other Business Highlights
Fully Operationalized Internal Manufacturing Capabilities
In 2024, the Company plans to continue to invest in its allogeneic platform for TSCM-rich CAR-T, including proprietary non-viral technologies, and will continue to refine and improve its manufacturing processes in ways that will potentially further increase product yield generally.
Partnerships and Collaborations
The Company continues to explore potential strategic and business development options for its technology platforms and gene therapy programs, as well as other potential opportunities in cell therapy.
Poseida R&D Days
In recognition of its continued development and growth, and to highlight its proprietary platform technologies and preclinical research in 2024, the Company plans to hold two R&D Days focusing on gene therapy and cell therapy respectively.
Cash Position and Runway
As previously announced,
Statements contained in this press release regarding matters that are not historical facts are "forward-looking statements" within the meaning of the Private Securities Litigation Reform Act of 1995. Such forward-looking statements include statements regarding, among other things, expected plans with respect to clinical trials, including timing of regulatory submissions and approvals and clinical data updates; anticipated timelines and milestones with respect to the Company's development programs and manufacturing activities and capabilities; the potential capabilities and benefits of the Company's technology platforms and product candidates, including the efficacy and safety profile of such product candidates; the quotes from
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Investor Contact:Alex Lobo, Stern Investor Relations IR@poseida.com; Media Contact:Sarah Thailing, Senior Director, Corporate Communications and IR, Poseida Therapeutics, Inc., PR@poseida.com