Highlighted significant research, preclinical
advances and platform technology progress at third annual R&D
Day
Appointed Kristin
Yarema, Ph.D., as President, Cell Therapy, adding extensive
oncology and allogeneic T cell immunotherapy experience to the
Company's leadership team
SAN
DIEGO, May 9, 2023 /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 updates and
financial results for the first quarter ended March 31, 2023.
"In the first quarter, we continued to advance our clinical and
research stage efforts as well as enhanced our leadership with the
recent appointment of Kristin
Yarema, Ph.D., as President, Cell Therapy," said
Mark Gergen, Chief Executive Officer
of the Company. "In our allogeneic CAR-T portfolio, we are actively
enrolling patients in the Phase 1 studies for P-MUC1C-ALLO1 and
P-BCMA-ALLO1 and remain on track for an IND for P-CD19CD20-ALLO1
mid-year. These initial allogeneic clinical programs are designed
to evaluate many aspects of our unique high-TSCM
allogeneic platform including dosing strategies, pre-conditioning
regimens, potential biomarkers and many other factors. In our
gene therapy portfolio, we are in discussions with Takeda about our
collaboration following the news that they have made some strategic
decisions in their research priorities and will update when
appropriate. In the meantime, we remain excited about the strong
progress made on all gene therapy programs and look forward to
giving six preclinical presentations at the American Society of
Gene and Cell Therapy annual meeting next week."
Program Updates
CAR-T Programs
In cell therapy, the Company is focused on three allogeneic CAR-T
programs with two programs currently progressing in Phase 1
clinical trials and one expected IND during the
year:
MUC1-C Program
P-MUC1C-ALLO1 is an allogeneic CAR-T product candidate targeting
solid tumors derived from epithelial cells, including breast and
ovarian cancers. The Company is currently evaluating P-MUC1C-ALLO1
in a Phase 1 clinical trial and presented early clinical data in
December 2022 at the European Society
for Medical Oncology Immuno-Oncology 2022 Annual Congress (ESMO
I-O) in Geneva, Switzerland. The
Company currently expects to present further clinical updates for
the program at a medical meeting in 2023.
BCMA Program
P-BCMA-ALLO1 is an allogeneic CAR-T product candidate being
developed to target 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 shared early clinical
data from the program at ESMO I-O in December 2022. The Company currently expects to
present further clinical updates for the program at a medical
meeting in 2023, subject to clearance with Roche.
CD19CD20 Program
P-CD19CD20-ALLO1 is a preclinical
allogeneic 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. The
Company believes that by targeting both CD19 and CD20, there is
potential to overcome potential antigen escape that has been
observed by others. The Company expects to file an IND for
P-CD19CD20-ALLO1 in mid-2023.
Gene Therapy Programs
The Company is advancing multiple preclinical gene therapy programs
in liver-directed diseases:
OTC Program
P-OTC-101 is an in vivo program 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 and working on
an updated timeline for the program. The Company presented
data at its R&D Day in February
2023, highlighting continued advancements in preclinical
models leading towards a potential functional cure of OTC
Deficiency.
FVIII Program
The Company is advancing its P-FVIII-101 preclinical program
partnered with Takeda, which is in development for the in vivo
treatment of Hemophilia A. P-FVIII-101 utilizes piggyBac gene
modification delivered via lipid nanoparticle that has demonstrated
stable and sustained Factor VIII expression in animal models. The
Company is presenting preclinical data from this program at the
upcoming American Society of Gene and Cell Therapy (ASGCT) 26th
Annual Meeting being held in Los
Angeles on May 16-20,
2023.
PAH Program
Announced at the Company's R&D Day in
February 2023, P-PAH-101 is a
liver-directed gene therapy partnered with Takeda to treat
Phenylketonuria (PKU), an inherited genetic disorder caused by
mutations in the phenylalanine hydroxylase (PAH) gene resulting in
buildup of phenylalanine in the body. If left untreated, PKU can
affect a person's cognitive development. P-PAH-101 utilizes
piggyBac technology combined with its hybrid adeno-associated virus
(AAV) and nanoparticle delivery system. The Company's preclinical
data has demonstrated the potential to resolve phenylalanine to
normal levels following a single treatment in juvenile and adult
mice. P-PAH-101 is currently in preclinical development.
Leadership Updates
Kristin Yarema, Ph.D.,
Appointed President, Cell Therapy
In April 2023, the Company announced the appointment
of Kristin Yarema, Ph.D., as
President, Cell Therapy, to lead the Company's drug development
programs in cell therapy, including its collaboration with Roche.
She joined the Company after most recently serving as Chief
Commercial Officer at Atara Biotherapeutics, where she led the
commercialization of EBVALLO™, which became the world's first
marketed allogeneic T cell therapy after receiving regulatory
approval in Europe for the
treatment of a rare lymphoma. Prior to Atara, she held a series of
U.S. and global commercial leadership roles at Amgen and Novartis
in hematology-oncology among other therapeutic areas.
Rafael G. Amado, M.D.,
Appointed to Board of Directors
In April 2023, Rafael G.
Amado, M.D., was appointed to the Company's Board of
Directors. Dr. Amado is currently President, Head of Global
Oncology Research and Development at Zai Lab, a public
biopharmaceutical company. Prior to Zai Lab, Dr. Amado served as
Executive Vice President, Head of Research and Development and
Chief Medical Officer from September
2019 to December 2022 at
Allogene Therapeutics, Inc. and President of Research and
Development from August 2018 to
August 2019 and Chief Medical Officer
from March 2015 to August 2018 at Adaptimmune, LLC.
Financial Results for the First Quarter 2023
"We continue to be disciplined on spend and evaluate plans and
options to help us further manage our cash burn rate," said
Johanna Mylet, Chief Financial
Officer at Poseida. "We have been operating at the low end of our
guided cash utilization range and our current base plan should
allow us to preserve our runway into at least mid-2024, and
possibly further depending on other decisions, including business
development opportunities."
Revenues
Revenues were $10.3
million for the first quarter ended March 31, 2023, compared to $1.4 million for the same period in 2022. The
increase of revenues was primarily due to revenues earned from the
collaboration and license agreement with Roche, which became
effective in the third quarter of 2022.
Research and Development
Expenses
Research and development expenses were
$38.1 million for the first quarter
ended March 31, 2023, compared to $48.9
million for the same period in 2022. The decrease was
primarily driven by the wind-down of the Company's clinical
development activities associated with our autologous
programs and related contract termination expense in the
prior year and the transition of manufacturing to the Company's
internal pilot plant for P-BCMA-ALLO1, partially offset by
increases in the number of ongoing clinical trials, including
enrollment and manufacturing for the P-MUC1C-ALLO1 and P-BCMA-ALLO1
Phase 1 clinical trials, and increases related to the Company's
preclinical stage programs due to an increase in research
collaboration activity and personnel and facilities expenses as a
result of increased headcount.
General and Administrative Expenses
General and
administrative expenses for the first quarter ended March 31, 2023
were $11.8 million compared to
$9.5 million for the same period in
2022. The increase was primarily related to an increase in
stock-based compensation expense due to a one-time modification
associated with the retirement of the Company's former Executive
Chairman.
Net Loss
Net loss was $38.8
million for the first quarter ended March 31, 2023, compared
to $58.1 million for the same period
of 2022.
Cash Position
As of March 31,
2023, the Company's cash, cash equivalents and short-term
investments balance was $247.2
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 continue to be
sufficient to fund operations into at least mid-2024.
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 and nanoparticle and hybrid gene delivery
technologies. The Company has formed global strategic
collaborations with Roche and Takeda to unlock the promise of cell
and gene therapies for patients. Learn more at
www.poseida.com and connect with Poseida on
Twitter 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 agreements; 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; estimates of the Company's cash
balance, expenses, capital requirements, any future revenue, and
need for additional financing; 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 its
strategic partners devote to advancing development programs under
their respective collaboration agreements and the Company may not
receive the potential fees and payments under the collaboration
agreements and the ability of its strategic partners to early
terminate the collaborations, such that the Company may not
fully realize the benefits of such collaborations; 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
|
(Unaudited)
|
(In thousands,
except share and per share amounts)
|
|
STATEMENTS OF
OPERATIONS
|
|
|
|
Three Months Ended
March 31,
|
|
|
|
2023
|
|
|
2022
|
|
Revenues:
|
|
|
|
|
|
|
Collaboration
revenue
|
|
$
|
10,343
|
|
|
$
|
1,435
|
|
Total
revenue
|
|
|
10,343
|
|
|
|
1,435
|
|
Operating
expenses:
|
|
|
|
|
|
|
Research and
development
|
|
|
38,052
|
|
|
|
48,850
|
|
General and
administrative
|
|
|
11,807
|
|
|
|
9,546
|
|
Total operating
expenses
|
|
|
49,859
|
|
|
|
58,396
|
|
Loss from
operations
|
|
|
(39,516)
|
|
|
|
(56,961)
|
|
Other income
(expense):
|
|
|
|
|
|
|
Interest
expense
|
|
|
(2,028)
|
|
|
|
(1,077)
|
|
Other income
(expense), net
|
|
|
2,697
|
|
|
|
(19)
|
|
Net loss
|
|
$
|
(38,847)
|
|
|
$
|
(58,057)
|
|
|
|
|
|
|
|
|
Net loss per share,
basic and diluted
|
|
$
|
(0.45)
|
|
|
$
|
(0.93)
|
|
Weighted-average number
of shares outstanding, basic and diluted
|
|
|
86,265,223
|
|
|
|
62,555,915
|
|
SELECTED BALANCE
SHEET DATA
|
|
|
|
March 31,
2023
|
|
|
December 31,
2022
|
|
Cash, cash equivalents
and short-term investments
|
|
$
|
247,201
|
|
|
$
|
282,493
|
|
Total assets
|
|
|
313,621
|
|
|
|
351,837
|
|
Total
liabilities
|
|
|
156,056
|
|
|
|
164,242
|
|
Total stockholders'
equity
|
|
|
157,565
|
|
|
|
187,595
|
|
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SOURCE Poseida Therapeutics, Inc.