Lead asset P-BCMA-ALLO1 Phase 1 clinical trial
data presented at ASH 2023 demonstrated 82% ORR and favorable
emerging safety and reliability profile
Initiated clinical trial of Phase 1
P-CD19CD20-ALLO1, the Company's first allogeneic dual CAR-T
program
Allogeneic CAR-T data to be presented at AACR
in April 2024 describing activity of
P-BCMA-ALLO1 in BCMA-experienced patients and comparison of
lymphodepletion needs in heme vs. solid tumor settings
P-FVIII-101 gene therapy data presented at ASH
and Company to host Gene Therapy R&D Day April 17, 2024
SAN
DIEGO, March 7, 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 announced business updates and
financial results for the fourth quarter and full year ended
December 31, 2023.
"2024 looks to be a breakout year for Poseida as we build on the
recent allogeneic BCMA clinical data presented at ASH, which
demonstrated the potential of our
high-TSCM allogeneic CAR-T therapies to offer
effective, safe, and reliable treatment addressing unmet needs in
multiple myeloma. We are able to achieve high levels of
CAR-TSCM in our products because of our proprietary,
nonviral technologies," said Kristin
Yarema, Ph.D., President and Chief Executive Officer. "Over
the coming months we are planning data readouts for each of our
three clinical-stage CAR-T programs, all of which are manufactured
at our own GMP facility and will discuss the latest progress of our
gene therapy programs at our R&D Day in April."
Recent 2023 Accomplishments
Cell therapy
- Presented positive data from the Phase 1
study of P-BCMA-ALLO1 in relapsed/refractory multiple
myeloma (RRMM) at ASH: In December 2023, the Company presented data from
its Phase 1 study of P-BCMA-ALLO1 at the 65th American
Society of Hematology (ASH) Annual Meeting and Exposition. These
early data support the Company's belief that TSCM-rich
allogeneic CAR-Ts have the potential to offer promising product
response rates, safety profiles, and rapid patient access which if
confirmed could provide differentiation from currently available
therapies, including autologous CAR-T therapies. The study used
product from 6 different CAR-T lots produced from 6 different
donors with data demonstrating:
- 82% ORR in deep clinical responses, including sCRs and
MRD-negative patients from off-the-shelf, allogeneic BCMA-targeted
CAR-T in heavily pretreated patients receiving adequate
lymphodepletion.
- 100% ORR in those patients who were not previously treated with
a BCMA-targeted bispecific T-cell engaging antibody.
- Favorable emerging safety and reliability profile, with all
(100%) intent-to-treat patients receiving therapy with no use of
bridging chemotherapy or other anti-myeloma bridging therapies and
low incidences of CRS and neurotoxicity observed (all ≤ Grade
2).
- Preliminary data suggest that allogeneic TSCM-rich
CAR-T cells traffic to bone marrow, differentiate to cell-killing
effector T cells and persist at least 6 weeks, supporting the
hypothesis of cell persistence at tumor-relevant sites.
- 8 of 9 responding patients still in response at data cut
off.
- Continued enrollment of P-MUC1C-ALLO1: The Company
continued enrollment in the Phase 1 clinical trial of P-MUC1C-ALLO1
for solid tumors. Drawing from insights gained in allogeneic BCMA
CAR-T, it is exploring various dosing approaches, including higher
lymphodepletion, cell dose, and scheduling in the MUC1C program.
Data from the allogeneic BCMA CAR-T program, presented at ASH,
underscored the important role of cyclophosphamide conditioning
dose in CAR-T cell expansion and persistence. Patients in arms
receiving higher doses (500 mg/m2 and 1,000
mg/m2) demonstrated significantly elevated P-BCMA-ALLO1
levels compared to those in the 300 mg/m2 cohort.
Consequently, the Company is assessing lymphodepleting conditioning
regimens with doses exceeding 300 mg/m2 in the MUC1C
program.
- Advanced programs within hematologic malignancies at all
stages, in partnership with Roche, throughout 2023:
- Previously announced the expected acceleration of achieving
milestones, resulting in the receipt of a $30 million payment in the first quarter of
2024.
- Expanded the protocol and continued to enroll patients in the
Phase 1 study of P-BCMA-ALLO1, incorporating lymphodepletion
learnings from ASH 2023.
- Continued site activation activities and initiation of
enrollment for P-CD19CD20-ALLO1, the Company's first dual,
allogeneic CAR-T for B-cell malignancies.
- Advanced research programs optioned to Roche (P-BCMACD19-ALLO1
for multiple myeloma and P-CD70-ALLO1 for AML).
- Developed and deployed manufacturing platform improvements to
all programs that delivered higher and more consistent levels of
cell expansion.
Gene therapy
- Presented proof-of-principle data
supporting P-FVIII-101 as a potential treatment
option for Hemophilia A at ASH: New
preclinical data presented at ASH demonstrated the capabilities of
the Company's non-viral approach in providing stable Factor VIII
(FVIII) transgene expression through genomic integration.
Additionally, the data highlighted re-dosing, or titrating to
efficacy, along with favorable tolerability data for this approach,
including 52-week durability data in an adult Hemophilia A mouse
model supporting sustained and robust expression.
Anticipated Milestones
- P-BCMA-ALLO1: The Company plans to present data at the
American Association for Cancer Research (AACR) Annual Meeting in
San Diego. At AACR, the Company
will present data on a subset of recently enrolled patients
refractory to initial BCMA targeting therapy. The poster
presentation, titled "Clinical Activity of P-BCMA-ALLO1, a B-cell
Maturation Antigen (BCMA) Targeted Allogeneic Chimeric Antigen
Receptor T-cell (CAR-T) Therapy, in Relapsed Refractory Multiple
Myeloma (RRMM) Patients (pts) Following Progression on Prior BCMA
Targeting Therapy," is anticipated to take place on April 8, 2024, 9:00 AM to
12:30 PM PT. Subject to coordination with Roche, the Company
plans to provide an additional clinical update on this program at a
scientific meeting in the second half of 2024.
- P-MUC1C-ALLO1: The Company plans to review initial
clinical findings from the Phase 1 clinical trial in solid tumors
at AACR related to cell expansion and lymphodepletion regimens. The
poster presentation, titled "Solid Tumor Patients Require Higher
Cyclophosphamide Dose than Multiple Myeloma Patients to Achieve
Adequate Lymphodepletion (LD) Necessary to Enable Allogeneic CAR-T
Expansion," is anticipated to take place on April 8, 2024, 9:00 AM to
12:30 PM PT. In addition to the AACR presentation, the
Company plans to present a more fulsome clinical update at an
appropriate forum in the second half of 2024.
- P-CD19CD20-ALLO1: Following the initiation of the Phase
1 trial targeting B-cell malignancies in late 2023, the Company
expects to provide an interim data update in the second half of
2024, subject to coordination with Roche.
- P-PSMA-ALLO1: In January
2024, Poseida announced that it expects to advance its next
allogeneic solid tumor program, P-PSMA-ALLO1 in prostate cancer,
into IND-enabling studies incorporating the progress and learnings
of its previous autologous study and recent advancements in its
allogeneic platform in 2024.
The Company believes its TSCM-rich CAR-T platform and
associated proprietary technologies have strong potential to
deliver new therapeutic approaches in autoimmune disease. The
Company is actively developing a strategy in inflammation and
autoimmune disease and will provide an update at an appropriate
time.
Other Operational Updates and Upcoming Events
Poseida R&D Days
The Company plans to host two
R&D Days in 2024 focusing on gene therapy and cell therapy
respectively.
The Company plans to host its gene therapy specific R&D Day
on April 17, 2024, which will feature
presentations from gene therapy management as well as key opinion
leaders. The event will highlight the Company's progress across its
proprietary non-viral gene insertion and editing technologies, an
update following a strategic review of its portfolio and new
preclinical data across in vivo gene therapy programs.
The Company plans to host an R&D Day focusing on cell
therapy in the second half of 2024.
Evaluating Opportunities in CAR-T Beyond Oncology
The
Company believes its TSCM-rich CAR-T platform and
associated proprietary technologies have strong potential to
deliver new therapeutic approaches in autoimmune disease. The
Company is developing a strategy in inflammation and autoimmune
disease and will provide an update at an appropriate time.
Leadership Updates
Alexander
Chapman joined Poseida on February
26, 2024, as Senior Vice President, Investor Relations &
Corporate Communications. He has over 20 years' experience spanning
a broad range of strategic roles across the biotech sector. Prior
to Poseida, he was the Head of Corporate Communications &
Investor Relations at Atara Biotherapeutics, the first company in
the world to receive regulatory approval for an allogeneic T-cell
immunotherapy. Previously, Mr. Chapman held a series of global and
U.S. leadership roles at Amgen, including Corporate Affairs, Value,
Access & Pricing, and U.S. Business Operations.
Loren Wagner was promoted to the
role of Chief Operations Officer in February
2024. Mr. Wagner joined the Company in March 2021 and most recently served as Senior
Vice President, Global Operations. He has more than 30 years of
industry experience across a variety of manufacturing, quality, and
engineering roles. Prior to the Company, Mr. Wagner was the Head of
CAR-T Operations at Bristol Myers Squibb/Celgene. Prior to Celgene,
he worked for 12 years at Allergan, where he was responsible for
the production and distribution of investigational supplies
globally.
Brent Warner, who has served as
the Company's President, Gene
Therapy, will depart Poseida to pursue an external career
opportunity effective April 1,
2024.
Financial Results for the Fourth Quarter and Full Year
2023
Revenues
Revenues were $25.0
million for the fourth quarter ended December 31, 2023, compared to $10.1 million for the same period in 2022. The
increase was primarily due to revenue earned as a result of a
milestone achieved in the fourth quarter under the amendment to the
collaboration and license agreement with Roche and increased
activity under the collaboration, offset by a decrease in revenue
related to Takeda due to the termination of its collaboration
agreement in the third quarter of 2023.
For the full year ended December 31, 2023, revenues were
$64.7 million, compared to
$130.5 million for the same period in
2022. The decrease was primarily due to initial license revenue
recognized from the collaboration and license agreement with Roche,
which became effective in the third quarter of 2022, offset by the
revenue recognized related to the research services performed under
the collaboration and license agreements with Roche and Takeda,
including $8.9 million of previously
deferred revenue recognized as a result of the termination of its
collaboration agreement with Takeda in the third quarter of
2023.
Research and Development Expenses
Research and
development expenses were $42.0 million for the fourth quarter ended
December 31, 2023, compared to $33.9
million for the same period in 2022. The increase was
primarily due to an increase in preclinical stage programs and
other unallocated expenses due to an increase in research
collaboration activity, an increase in personnel expenses as a
result of increased headcount, and an increase in clinical stage
allogeneic programs, partially offset by a decrease in clinical
stage autologous programs driven by the wind-down of the Company's
clinical development activities for such programs.
For the full year ended December 31, 2023, research and
development expenses were $156.8 million, compared to $152.9 million for the same period in 2022. The
increase was primarily due to an increase in preclinical stage
programs and other unallocated expenses due to an increase in
research collaboration activity, an increase in personnel expenses
as a result of increased headcount, an increase in clinical stage
allogeneic programs, and an increase in internal costs related to
facilities and other expenses, offset by a decrease in clinical
stage autologous programs driven by the wind-down of the Company's
clinical development activities for such programs.
General and Administrative Expenses
General and
administrative expenses were $8.9
million for the fourth quarter ended December 31, 2023,
compared to $9.4 million for the same period in 2022. The
decrease was primarily due to lower insurance costs.
For the full year ended December 31, 2023, general and
administrative expenses were $37.4 million, compared to $37.5 million for the same period in 2022.
The decrease was primarily due to lower insurance costs, offset by
an increase in personnel costs due to an accelerated stock-based
compensation expense in the first quarter of 2023 related to a
one-time expense associated with the retirement of the Company's
former Executive Chairman.
Net Loss
Net loss was $25.3
million and $123.4 million for the fourth quarter and
full year ended December 31, 2023, respectively, compared to
net loss of $33.3 million and
$64.0 million for the fourth quarter
and full year ended December 31, 2022, respectively.
Cash Position
As of December 31, 2023, the
Company's cash, cash equivalents and short-term investments balance
was $212.2 million. The Company
expects that its cash, cash equivalents and short-term investments
together with the remaining near-term milestones and other payments
from Roche will be sufficient to fund operations into the second
half of 2025. Potential additional payments under
the amended Roche collaboration and license
agreement and/or potential additional business development
could further extend cash runway.
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 Poseida 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;
potential fees, milestones and other payments that the Company may
receive pursuant to its collaboration agreement with Roche,
including related timing; 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 quote 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 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; the fact that the Company will
have limited control over the efforts and resources that Roche
devotes to advancing development programs under its collaboration
agreement and the Company may not receive the potential fees and
payments under the collaboration agreement and the ability of Roche
to early terminate the collaboration, such that the Company may not
fully realize the benefits of the collaboration; 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.
Poseida
Therapeutics, Inc.
Selected Financial
Data
(In thousands,
except share and per share amounts)
STATEMENTS OF
OPERATIONS
|
|
|
|
|
|
Three Months
Ended
December 31,
|
|
|
Twelve Months
Ended
December 31,
|
|
|
|
2023
|
|
|
2022
|
|
|
2023
|
|
|
2022
|
|
|
|
(Unaudited)
|
|
|
|
|
|
|
|
Revenues:
|
|
|
|
|
|
|
|
|
|
|
|
|
Collaboration
revenue
|
|
$
|
24,995
|
|
|
$
|
10,051
|
|
|
$
|
64,703
|
|
|
$
|
130,492
|
|
Total
revenue
|
|
|
24,995
|
|
|
|
10,051
|
|
|
|
64,703
|
|
|
|
130,492
|
|
Operating
expenses:
|
|
|
|
|
|
|
|
|
|
|
|
|
Research and
development
|
|
|
42,045
|
|
|
|
33,904
|
|
|
|
156,772
|
|
|
|
152,899
|
|
General and
administrative
|
|
|
8,859
|
|
|
|
9,368
|
|
|
|
37,435
|
|
|
|
37,539
|
|
Total operating
expenses
|
|
|
50,904
|
|
|
|
43,272
|
|
|
|
194,207
|
|
|
|
190,438
|
|
Loss from
operations
|
|
|
(25,909)
|
|
|
|
(33,221)
|
|
|
|
(129,504)
|
|
|
|
(59,946)
|
|
Other income
(expense):
|
|
|
|
|
|
|
|
|
|
|
|
|
Interest
expense
|
|
|
(2,267)
|
|
|
|
(1,975)
|
|
|
|
(8,671)
|
|
|
|
(6,370)
|
|
Other income,
net
|
|
|
2,827
|
|
|
|
2,170
|
|
|
|
14,852
|
|
|
|
2,858
|
|
Net loss before income
tax
|
|
|
(25,349)
|
|
|
|
(33,026)
|
|
|
|
(123,323)
|
|
|
|
(63,458)
|
|
Income tax
expense
|
|
|
—
|
|
|
|
(292)
|
|
|
|
(107)
|
|
|
|
(544)
|
|
Net loss
|
|
$
|
(25,349)
|
|
|
$
|
(33,318)
|
|
|
$
|
(123,430)
|
|
|
$
|
(64,002)
|
|
|
|
|
|
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.27)
|
|
|
$
|
(0.39)
|
|
|
$
|
(1.37)
|
|
|
$
|
(0.89)
|
|
Weighted-average number
of shares outstanding, basic and diluted
|
|
|
95,623,219
|
|
|
|
85,953,375
|
|
|
|
90,185,096
|
|
|
|
71,953,703
|
|
SELECTED BALANCE
SHEET DATA
|
|
|
|
|
|
December 31,
|
|
|
|
2023
|
|
|
2022
|
|
Cash, cash equivalents
and short-term investments
|
|
$
|
212,202
|
|
|
$
|
282,493
|
|
Total assets
|
|
|
273,885
|
|
|
|
351,837
|
|
Total
liabilities
|
|
|
170,184
|
|
|
|
164,242
|
|
Total stockholders'
equity
|
|
|
103,701
|
|
|
|
187,595
|
|
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SOURCE Poseida Therapeutics, Inc.