Allogene Therapeutics Reports Fourth Quarter and Full Year 2022 Financial Results and Provides Business Update
- Initiated Industry’s First Potentially Pivotal Phase 2 Allogeneic CAR T Trial, ALPHA2, in Relapsed/Refractory (R/R) Large B Cell Lymphoma (LBCL)
- Presented Data Highlighting Industry-Leading Allogeneic CAR T Portfolio
- CD19 (ALLO-501/A): Six Month Durability and Progression Free Survival in R/R LBCL Comparable with Approved Autologous CAR T Therapies
- BCMA (ALLO-715): First Allogeneic CAR T to Demonstrate Deep and Durable Responses in R/R Multiple Myeloma (MM)
- CD70 (ALLO-316): Initial Proof-of-Concept for an Allogeneic CAR T in Solid Tumors
- Published Phase 1 Data from the UNIVERSAL Study of ALLO-715 in Nature Medicine
- Presented Dagger™ Technology, a Next Generation Allogeneic Platform Designed to Control Rejection of AlloCAR T Cells
- Ended 2022 with $576 Million in Cash, Cash Equivalents and Investments
- Conference Call and Webcast Scheduled for Today at
2:00 PM PT/ 5:00 PM ET
“We are very proud that data from our pipeline candidates continues to break new ground in the field of allogeneic cell therapy. From our CD19 program for non-Hodgkin lymphoma to our BCMA program for multiple myeloma, we have established that our AlloCAR T technology can induce deep, clinically meaningful responses in patients,” said
ALLO-501A: Anti-CD19 AlloCAR T Program
- As of the
October 25, 2022data cutoff, the ORR and Complete Response (CR) rate was 67% and 58%, respectively, among the 12 patients treated with the Single Dose FCA90 regimen using Alloy™ process material. The median duration of response was 23.1 months.
- Of patients who received single dose FCA90 and evaluable at six months, the ongoing CR rate was 50% and all CRs at six months were durable at 12 months. The longest CR ongoing at 26+ months.
- Phase 1 trials demonstrated a manageable safety profile with no observed dose limiting toxicities (DLTs), graft-vs-host disease (GvHD) or severe immune effector cell-associated neurotoxicity syndrome (ICANS).
- Among patients treated with Single Dose FCA90, there was no Grade 3+ cytokine release syndrome (CRS). One patient (8%) experienced a Grade 3+ infection and two (17%) experienced prolonged Grade 3+ cytopenia.
- 92% of all enrolled patients received product with 100% of infused product manufactured and released per product specifications. Patients were able to initiate treatment within two days of enrollment.
The Company is preparing for a Phase 3 study in earlier line LBCL targeting trial initiation in 1H 2024.
The Company is developing ALLO-647, its proprietary anti-CD52 monoclonal antibody intended to enable expansion and persistence of AlloCAR T product candidates, including ALLO-501A. The EXPAND trial, which is intended to demonstrate the contribution of ALLO-647 to the lymphodepletion regimen, will be open to enrollment early in the second quarter.
ALLO-715: Anti-BCMA AlloCAR T Programs
Data from the Phase 1 UNIVERSAL trial with ALLO-715 for the treatment of r/r multiple myeloma (MM) was also presented at the Company’s R&D Showcase and subsequently published in Nature Medicine, accompanied by an editorial. The UNIVERSAL trial is the first allogeneic anti-BCMA CAR T to demonstrate proof-of-concept in MM with response rates that are similar to an approved autologous CAR T therapy. Highlights include:
- Dose expansion cohorts demonstrated substantial and durable responses.
- Through a median follow-up of 14.8 months as of the
October 11, 2022data cutoff, the ORR was 67% in the FCA60 cohort and the very good partial response or better rate (VGPR+) was 42%. All VGPR+ were minimal residual disease (MRD) negative.
- The median DoR was 9.2 months, with the longest response ongoing at 24 months.
- 92% of all enrolled patients received product with 100% of infused product manufactured and released as per product specifications. None of the patients received bridging therapy and patients were able to initiate treatment immediately following enrollment. Median time from enrollment to lymphodepletion was 5 days.
- Safety profile was manageable with low-grade and reversible neurotoxicity and no GvHD. Eight patients (29%) experienced Grade 3+ infections and eight patients experienced prolonged Grade 3+ cytopenias.
The Company is evaluating manufacturing processes improvements across its BCMA candidates to achieve optimal performance.
ALLO-316: Anti-CD70 AlloCAR T Program
ALLO-316, the Company’s first AlloCAR T candidate for solid tumors, targets CD70, an antigen expressed on clear cell renal cell carcinoma (RCC) and other malignancies. At the Company’s R&D Showcase, the Company presented initial data demonstrating promising anti-cancer activity in the subset of nine patients with confirmed CD70-positive RCC from the ongoing Phase 1 TRAVERSE trial. Highlights include:
- As of the data cutoff date of
November 17, 2022, the disease control rate (DCR) in patients who were CD70+ was 100% including three patients who achieved a partial response (PR) (two confirmed and one unconfirmed with the longest response lasting until month eight).
- Cell expansion in patients with CD70 positive tumor was robust, and there was a trend toward greater tumor shrinkage in patients with high CD70 expression.
- Across all patients treated in the trial, ALLO-316 has demonstrated a generally manageable safety profile with no GvHD. One dose limiting toxicity of auto-immune hepatitis occurred in the second dose level. Grade 3+ prolonged cytopenia was observed in three patients (18%). Grade 3 CRS was observed in one patient. Neurotoxicity was low grade, reversible and seen in only three patients (18%).
The Company is deploying a new investigational in vitro companion diagnostic (IVD) assay designed to prospectively assess CD70 expression levels to enhance patient selection. TRAVERSE will continue to explore varying cell dose and lymphodepletion regimens, including FC and
Next Generation Platform Technology
Allogene has pursued an integrated strategy within Research and Development aimed at matching technology with insights obtained from the clinic to create solutions designed to advance patient outcomes. One of these is Dagger™, a proprietary technology designed to control rejection of AlloCAR T cells by the host immune cells. This technology deploys a CD70 CAR on AlloCAR T cells in an effort to recognize and deplete CD70 positive alloreactive host T cells. Preclinical data indicate that CD70 Dagger CARs can be combined with other anti-tumor CARs in a single cell, providing both protection from allorejection and dual specificity killing capability, thus offering a differentiated next generation product candidate profile.
Fourth Quarter and Year-End Financial Results
- Research and development expenses were $75.4 million for the fourth quarter of 2022, which includes $7.4 million of non-cash stock-based compensation expense. For the full year of 2022, research and development expenses were
$256.4 million. Research and development expense for the year includes $42.5 millionof non-cash stock-based compensation expense.
- General and administrative expenses were $21.0 million for the fourth quarter of 2022, which includes $9.8 million of non-cash stock-based compensation expense. For the full year of 2022, general and administrative expenses were
$79.3 million, which includes $41.1 millionof non-cash stock-based compensation expense.
- Net loss for the fourth quarter of 2022 was $94.8 million, or $0.66 per share, including non-cash stock-based compensation expense of $17.2 million. For the full year of 2022, net loss was
$332.6 million, or $2.32per share, including non-cash stock-based compensation expense of $83.6 million.
- The Company had
$576.5 millionin cash, cash equivalents, and investments as of December 31, 2022.
2023 Financial Guidance
- The Company expects a decrease in cash, cash equivalents, and investments of approximately
$250 millionin 2023. Based on current expectation, the Company expects the cash runway to be sufficient to fund operations into 2025. GAAP Operating Expenses are expected to be approximately $350 million, including estimated non-cash stock-based compensation expense of approximately $90 million. These estimates exclude any impact from potential business development activities.
Conference Call and Webcast Details
Allogene will host a live conference call and webcast today at
Cautionary Note on Forward-Looking Statements
This press release contains forward-looking statements for purposes of the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. The press release may, in some cases, use terms such as "predicts," "believes," "potential," "proposed," "continue," "estimates," "anticipates," "expects," "plans," "intends," "may," "could," "might," "will," "should" or other words that convey uncertainty of future events or outcomes to identify these forward-looking statements. Forward-looking statements include statements regarding intentions, beliefs, projections, outlook, analyses or current expectations concerning, among other things: the timing and ability to progress the ALPHA2 and TRAVERSE trials; the timing and ability to initiate the EXPAND trial; clinical outcomes, which may materially change as more patient data become available; the likelihood of success of the Phase 2 ALPHA2 trial, which is based on limited data from the Phase 1 ALPHA trials across two different product candidates and various doses of ALLO-501 or ALLO-501A; the ability to optimize manufacturing or manufacture AlloCAR T products, including with the Alloy process, with consistent and reproducible product characteristics; the design and potential benefits of our Dagger technology, including its ability to control rejection of allogeneic CAR T cells; the potential for our product candidates to be approved; the potential benefits of AlloCAR T products and 2023 financial guidance. Various factors may cause material differences between Allogene’s expectations and actual results, including, risks and uncertainties related to: our product candidates are based on novel technologies, which makes it difficult to predict the time and cost of product candidate development and obtaining regulatory approval; Servier’s discontinuation of its involvement in the development of all CD19 products pursuant to our Exclusive License and Collaboration Agreement, including disagreements relating to development cost contributions and audit rights related to such contributions, and the timeframe during which we have the right to elect a license to CD19 Products outside of
Caution should be exercised regarding statements comparing autologous CAR T data. There are differences in the clinical trial design, patient populations, published data, follow-up times and the product candidates themselves, and the results from the clinical trials of autologous products may have no interpretative value on our existing or future results.
AlloCAR T™, TurboCAR™, Alloy™ and Dagger™ are trademarks of Allogene Therapeutics, Inc.
Allogene’s AlloCAR T™ programs utilize Cellectis technologies. ALLO-501 and ALLO-501A are anti-CD19 products being jointly developed under a collaboration agreement between
SELECTED FINANCIAL DATA
(unaudited; in thousands, except share and per share data)
STATEMENTS OF OPERATIONS
|Three Months Ended
|Collaboration revenue - related party||$||47||$||51||$||243||$||38,489|
|Research and development||75,419||53,983||256,387||220,176|
|General and administrative||21,002||19,961||79,305||74,105|
|Total operating expenses||96,421||73,944||335,692||294,281|
|Loss from operations||(96,374||)||(73,893||)||(335,449||)||(255,792||)|
|Other income (expense), net:|
|Interest and other income, net||2,757||186||4,566||1,714|
|Total other income (expense), net||1,527||(975||)||2,817||(1,213||)|
|Net loss per share, basic and diluted||$||(0.66||)||$||(0.54||)||$||(2.32||)||$||(1.89||)|
|Weighted-average number of shares used in computing net loss per share, basic and diluted||144,149,240||139,173,761||143,147,165||135,820,386|
SELECTED BALANCE SHEET DATA
|Cash, cash equivalents and investments||$||576,471||$||809,481|
|Total stockholders’ equity||665,870||916,406|
Allogene Media/Investor Contact:
Chief Communications Officer
Source: Allogene Therapeutics, Inc.