Poseida Therapeutics Provides Updates and Financial Results for the First Quarter of 2024
Expanded strategic relationship with Astellas with new research collaboration leveraging
Generated
Presented promising early data at AACR demonstrating clinical responses with P-BCMA-ALLO1 in multiple myeloma patients following progression after prior BCMA-targeted therapy
Presented non-human primate (NHP) data at ASGCT highlighting favorable safety and high-fidelity liver editing for lead non-viral investigational genetic medicine, P-KLKB1-101
"We see 2024 as a breakout year for
"The proprietary and powerful genetic engineering toolkit that we have used ex vivo to deliver our allogeneic CAR-T programs demonstrates compelling potential for the development of in vivo genetic medicines. Moving forward, we are focusing our gene therapy pipeline on fully nonviral approaches, highlighted by our lead gene editing and gene insertion programs, P-KLKB1-101 and P-FVIII-101. New preclinical data supporting the advancement of both programs was recently presented at the ASGCT annual meeting, further confirming their potential to address significant unmet patient need."
Recent Accomplishments
Cell therapy
Announced strategic research collaboration and license agreement with Astellas'
wholly owned subsidiary
,
Xyphos Biosciences, to
develop novel cell therapies for solid tumors. The collaboration will utilize
Presented new allogeneic CAR-T data at the
- The P-BCMA-ALLO1 data presented at AACR 2024 builds on the Company's earlier ASH 2023 data, which demonstrated a 100% overall response rate in patients who had not been previously treated with a BCMA-targeted therapy who received adequate lymphodepletion. This new data from a subset of patients in the ongoing Phase 1 study showed that three of five (60%) patients with relapsed/refractory multiple myeloma (RRMM) who had progressed following one or more prior BCMA-targeted therapies achieved clinical responses with P-BCMA-ALLO1. Overall, P-BCMA-ALLO1 was well tolerated, and findings suggest that P-BCMA-ALLO1 could be an appropriate treatment for a broad range of patients with multiple myeloma, including BCMA-naïve patients and those with relapsed/refractory disease whose cancer progressed following prior BCMA-targeted therapy.
- Following efforts to optimize its allogeneic CAR-T therapies,
Poseida presented new data underscoring the need for higher lymphodepletion chemotherapy doses when treating solid tumors vs. hematologic malignancies. Drawing from these insights, the Company is exploring higher lymphodepletion regimens in its Phase 1 clinical trial of P-MUC1C-ALLO1 for solid tumors.
P-BCMA-ALLO1 granted Orphan Drug Designation by the U.S. Food and Drug Administration (FDA) for the treatment of multiple myeloma, highlighting the potential of TSCM-rich allogeneic CAR-T therapies to offer the optimal combination of clinical results, on-demand availability, and high-volume production, while supporting broad access to off-the-shelf allogeneic CAR-T therapies.
Advanced Roche partnership including completion of certain program-related development milestones that resulted in the receipt of a
Genetic medicine
- The key advantages of
Poseida's fully non-viral approach include low immunogenicity, lower oncogenic risks, integrated and stable expression, personalized titratable dosing, and a favorable cost of goods compared to viral gene therapies. -
Poseida is focused on advancing its two fully non-viral lead programs in rare disease with significant unmet patient need: P-KLKB1-101 for Hereditary Angioedema (HAE), which is the Company's first in vivo gene editing program using Cas-CLOVER, and P-FVIII-101, which is the Company's gene insertion program for the treatment of Hemophilia A. -
Poseida's hybrid programs remain promising for applications where high levels of editing in liver cells are required andPoseida may opportunistically consider partnering transactions.
Presented multiple presentations at the
- P-KLKB1-101 interim data in a non-human-primate model showed that the Cas-CLOVER nuclease formulation was well tolerated and yielded levels of editing approaching therapeutic efficacy in early read-out doses.
- Studies in human cells and rodent models show high fidelity and high efficiency KLKB1 editing within a target range for correction of HAE. P-KLKB1-101 demonstrated a highly controlled dose-dependent reduction in kallikrein protein and confirmatory studies revealed minimal off-target editing.
- P-FVIII-101 preclinical data support advancing to non-human primate studies based on sustained FVIII expression over 13 months from a single dose, along with data supporting mitigated immunogenicity and ability to fine tune FVIII expression levels via repeat dosing and/or
Poseida's proprietary modulator switch. - Additional data describing the Company's advancements in lipid nanoparticle (LNP) technology, intracellular targeting agents and nuclease fidelity.
Anticipated Milestones
- P-BCMA-ALLO1 in RRMM: clinical update planned for the second half of 2024 subject to coordination with Roche.
- P-MUC1C-ALLO1 in solid tumors: clinical update planned for the second half of 2024.
- P-CD19CD20-ALLO1 in B-cell malignancies: interim data update anticipated in the second half of 2024 subject to coordination with Roche.
Other Operational Updates
Leadership Updates
Financial Results for the First Quarter 2024
Revenues
Revenues were
Research and Development Expenses
Research and development expenses were
General and Administrative Expenses
General and administrative expenses were
Net Loss
Net loss was
Cash Position
As of
About
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, reimbursements, milestones, royalty payments and other payments that the Company may receive pursuant to its collaboration agreements with Roche and Astellas, 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, safety and tolerability profile of such product candidates; the quote from
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STATEMENTS OF OPERATIONS |
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Three Months Ended |
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2024 |
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2023 |
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Revenues: |
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Collaboration revenue |
|
$ |
28,142 |
|
|
$ |
10,343 |
|
Total revenue |
|
|
28,142 |
|
|
|
10,343 |
|
Operating expenses: |
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|
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Research and development |
|
|
42,921 |
|
|
|
38,052 |
|
General and administrative |
|
|
9,798 |
|
|
|
11,807 |
|
Total operating expenses |
|
|
52,719 |
|
|
|
49,859 |
|
Loss from operations |
|
|
(24,577) |
|
|
|
(39,516) |
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Other income (expense): |
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Interest expense |
|
|
(2,253) |
|
|
|
(2,028) |
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Other income, net |
|
|
2,556 |
|
|
|
2,697 |
|
Net loss |
|
$ |
(24,274) |
|
|
$ |
(38,847) |
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|
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Net loss per share, basic and diluted |
|
$ |
(0.25) |
|
|
$ |
(0.45) |
|
Weighted-average number of shares outstanding, basic and diluted |
|
|
96,019,579 |
|
|
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86,265,223 |
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SELECTED BALANCE SHEET DATA |
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(Unaudited) |
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Cash, cash equivalents and short-term investments |
|
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$ 198,646 |
|
|
|
$ 212,202 |
|
Total assets |
|
|
262,580 |
|
|
|
273,885 |
|
Total liabilities |
|
|
177,986 |
|
|
|
170,184 |
|
Total stockholders' equity |
|
|
84,594 |
|
|
|
103,701 |
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