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
April 17, 2024
SAN
DIEGO, Jan. 4, 2024 /PRNewswire/ -- Poseida
Therapeutics, Inc. (Nasdaq: PSTX), a clinical-stage cell and gene
therapy company advancing a new class of treatments for patients
with cancer and rare diseases, today highlighted recent progress
across the company's cell and gene therapy portfolio and provided
updates on anticipated upcoming milestones.
"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 Kristin Yarema, Ph.D., President and Chief
Executive Officer of the Company. "In parallel, we expanded our
partnership with Roche, accelerating the expected timing of
achievement and increasing the certainty of certain milestones as
well as advancing programs at all stages in the Roche
collaboration. Notably, we cleared the IND for P-CD19CD20-ALLO1,
which we believe is the first known dual allogeneic CAR-T directed
against CD19 and CD20. In our non-viral gene therapy portfolio, we
are advancing P-FVIII-101 as a potentially durable treatment for
Hemophilia A and believe that the recent preclinical data presented
at ASH continue to support our liver-targeted non-viral delivery
platform. Together with our strong financial position and bolstered
by our robust business development and partnership activity, we
believe we are well positioned for success in 2024."
"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 Poseida at this transformative moment."
Program Highlights
Cell Therapy Programs
BCMA Program
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 American Society
of Hematology (ASH) Annual Meeting and Exposition in December 2023.
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.
In 2024, Poseida plans to continue development of P-BCMA-ALLO1
in partnership with Roche. This includes continuing the strong
enrollment in the Phase 1 study, refinement of dosing regimen for
both cell dose and lymphodepletion chemotherapy, sharing additional
details on clinical trial expansion strategies and continuing
product supply efforts. The Company plans to present additional
clinical data updates for P-BCMA-ALLO1 at scientific meetings in
2024, subject to coordination with Roche.
CD19CD20 Program
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.
MUC1C Program
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. Poseida plans to provide a data
update at a scientific meeting in the first half of 2024.
PSMA 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.
Early Stage Research
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 April 2024, the Company plans to share updates on
its programs and outcomes from its strategic review process.
FVIII Program
The Company is advancing its P-FVIII-101 preclinical program, a
fully non-viral liver-directed gene therapy combining Poseida's
proprietary piggyBac® DNA Delivery System with
nanoparticle delivery for the treatment of Hemophilia A. The
Company recently presented preclinical data from this program at
the ASH Annual Meeting in December
2023, demonstrating the capabilities of the piggyBac DNA
insertion system and non-viral approach in providing stable Factor
VIII transgene expression, along with the potential for redosing.
The P-FVIII-101 program is among the programs advanced through the
Company's former partnership with Takeda and Poseida received full
rights back after Takeda's change in strategy away from gene
therapy and rare disease.
OTC Program
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 July
2023.
PAH Program
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
Poseida has made substantial advancements
in its allogeneic cell therapy manufacturing platform
and capabilities. In 2023, Poseida established a fully
internal clinical manufacturing capability which is now supplying
all GMP products for all of its clinical trials across
three programs. In addition, the Company has achieved yield
improvements through optimization of unit operations, as described
at the CAR-TCR Summit in September 2023, capable of delivering cell yields
supporting up to 100+ doses per manufacturing run depending upon
cell dose.
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
In August 2023 the Company announced a $50 million strategic investment by Astellas and
granted Astellas certain strategic rights, further validating
Poseida's suite of genetic engineering technologies and its
allogeneic T cell platform.
Poseida continues to develop allogeneic CAR-T therapies
targeting hematological malignancies in partnership with Roche. The
Company announced in November 2023
that, based upon substantial progress in its P-BCMA-ALLO1 and
P-CD19CD20-ALLO1 programs, certain payments as well as the expected
timing of achievement of upcoming milestones in programs have been
accelerated to reflect progress in the programs and better align
with expected upcoming further clinical developments and
manufacturing needs and timelines. As a result of this progress,
the Company expects to receive certain payments sooner and/or with
more certainty than originally anticipated.
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.
Poseida will hold an R&D Day focusing on gene therapy on
April 17, 2024, when the Company
plans to provide an update on its strategic review of its gene
therapy and gene editing programs, as well as updates on its
pipeline and focus areas. An R&D Day focusing on cell therapy
will be hosted in the second half of 2024.
Organization
Kristin Yarema, Ph.D., has been
appointed to the Company's Board of Directors, effective
January 1st, 2024.
Cash Position and Runway
As previously announced, Poseida expects that its cash, cash
equivalents and short-term investments together with the remaining
near-term milestones and other payments from Roche and Astellas
will be sufficient to fund operations into the second half of 2025.
This cash runway reflects financial prudence but has mainly been
driven through business development and partnership activity, for
which Poseida continues to be well positioned.
About Poseida Therapeutics, Inc.
Poseida Therapeutics is a clinical-stage biopharmaceutical
company advancing differentiated cell and gene therapies with the
capacity to cure certain cancers and rare diseases. The Company's
pipeline includes allogeneic CAR-T cell therapy product candidates
for both solid and liquid tumors as well as in vivo gene therapy
product candidates that address patient populations with high unmet
medical need. The Company's approach to cell and gene therapies is
based on its proprietary genetic editing platforms, including its
non-viral piggyBac® DNA Delivery System, Cas-CLOVER™ Site-Specific
Gene Editing System, Booster Molecule, and nanoparticle and hybrid
gene delivery technologies as well as in-house GMP cell therapy
manufacturing. The Company has formed a global strategic
collaboration with Roche to unlock the promise of cell therapies
for patients with hematological malignancies. Learn more at
www.poseida.com and connect with us on X and LinkedIn.
Forward-Looking Statements
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 Dr. Yarema; estimates of
the Company's cash balance, expenses, capital requirements, and any
future revenue; the Company's ability to exploit and consummate
additional business development opportunities, including with Roche
and/or Astellas, and any anticipated impact on the Company's cash
balance and cash runway; the Company's ability to attract and/or
retain new and existing collaborators with relevant expertise and
its expectations regarding the potential benefits to be derived
from any such collaborations; and the Company's plans and strategy
with respect to developing its technologies and product candidates.
Because such statements are subject to risks and uncertainties,
actual results may differ materially from those expressed or
implied by such forward-looking statements. These forward-looking
statements are based upon the Company's current expectations and
involve assumptions that may never materialize or may prove to be
incorrect. Actual results could differ materially from those
anticipated in such forward-looking statements as a result of
various risks and uncertainties, which include, without limitation,
the Company's reliance on third parties for various aspects of its
business; risks and uncertainties associated with development and
regulatory approval of novel product candidates in the
biopharmaceutical industry; the Company's ability to retain key
scientific or management personnel; and the other risks described
in the Company's filings with the Securities and Exchange
Commission. All forward-looking statements contained in this press
release speak only as of the date on which they were made. The
Company undertakes no obligation to update such statements to
reflect events that occur or circumstances that exist after the
date on which they were made, except as required by law.
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SOURCE Poseida Therapeutics, Inc.
