On May 13, 2024 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products for cancer and autoimmune disease, reported corporate updates, and announced financial results for the quarter ended March 31, 2024 (Press release, Allogene, MAY 13, 2024, View Source [SID1234643128]).

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"We are very proud of the progress we’ve made across our portfolio, in particular the transformative potential of our pivotal ALPHA3 trial with cema-cel which is expected to readout in 2026. Securing the EU and UK rights reinforces our conviction in the cema-cel program," said David Chang, M.D., Ph.D., President, Chief Executive Officer and Co-Founder of Allogene. "We will continue to focus all of our resources on advancing these core programs and believe we are well-positioned to change the CAR T treatment landscape for the benefit of patients."

Key Corporate Updates

Expansion of CD19 Oncology Program Rights
The Company has obtained development and commercialization oncology rights to all EU Member States and the United Kingdom (Extended Territory) for its CD19-directed allogeneic cell therapy products from Servier.

These new expanded rights, combined with the U.S. rights already owned by the Company, substantially increase the potential total market opportunity from more than $6 billion in the U.S. alone to more than $9.5 billion in the combined U.S. and Extended Territory. It is expected that the future multibillion-dollar revenue potential for cema-cel now could increase by 50% with these expanded rights. The Company also has the ability to obtain the development and commercialization rights for cema-cel in Japan and China in the future at no additional cost, subject to Allogene demonstrating the resources to pursue those markets.

Program Updates

Cema-Cel: Pivotal Phase 2 ALPHA3 1L Consolidation Trial in Large B Cell Lymphoma (LBCL)
The Company continues to focus on the development of its investigational product cemacabtagene ansegedleucel, or cema-cel (previously known as ALLO-501A), as part of the first line (1L) treatment plan for LBCL patients who are at risk of relapse following 1L chemoimmunotherapy. The ALPHA3 trial will be conducted in a wide array of cancer treatment centers, including community cancer centers where most earlier line patients seek care.

This innovative trial will identify patients at high risk for relapse after 1L treatment by utilizing a novel and highly accurate test for minimal residual disease, or MRD. This investigational test is being developed by our partners Foresight Diagnostics and aims to offers improved prediction over existing methods for future relapse after completion of 1L treatment. ALPHA3 takes advantage of the allogeneic attributes of cema-cel. With off-the-shelf availability and convenience, cema-cel will be administered as a one-time infusion immediately upon detection of MRD at the completion of six cycles of R-CHOP or other standard 1L chemoimmunotherapy regimen. The outcome of this consolidation treatment could potentially improve the cure rate and uniquely position cema-cel to become the standard "7th cycle" of frontline treatment available to all eligible patients with MRD.

Start-up activities for the ALPHA3 trial are ongoing with a planned study initiation in mid-2024. This randomized study will enroll approximately 240 patients and is designed to demonstrate a meaningful improvement in event free survival (EFS) in patients treated with cema-cel relative to patients who receive the current standard of care (observation). Efficacy analyses are expected to occur in 2026 and will include the Independent Data Safety Monitoring Board (IDSMB) interim EFS analysis in 1H 2026 and the data readout of the primary EFS analysis in 2H 2026 with a Biologics License Application (BLA) submission targeted for 2027.

Cema-Cel: Phase 1 Trial in Chronic Lymphocytic Leukemia (CLL)
Enrollment is ongoing in the relapsed/refractory (r/r) CLL cohort of the Phase 1 ALPHA2 trial of cema-cel. While recent approval of an autologous CD19 CAR T therapy has been a positive step for patients with r/r CLL T cell dysfunction, and high circulating leukemia burden often found in patients with CLL make the isolation of functional T cells for autologous CAR T manufacturing difficult. This trial has been driven by investigator enthusiasm for an allogeneic CAR T to potentially boost the curative power of CAR T.

Initial data readout from the Phase 1 ALPHA2 CLL cohort (n=12) is projected by year-end 2024 with a Phase 2 pivotal study expected in 2025.

ALLO-329: CD19/CD70 Dual CAR with Dagger Technology in Autoimmune Disease (AID)
The Company has applied its deep understanding of CAR T research and development to design next-generation allogeneic CAR T investigational products that could sustain the scale of the AID market while also meeting the unique requirements for these patients.

ALLO-329, the Company’s CRISPR-based AlloCAR T investigational product for AID, incorporates the Dagger technology which is intended to eliminate the need for lymphodepletion while targeting CD19+ B-cells and CD70+ activated T-cells, both of which are likely to play a role in AID.

The Company plans to file an Investigational New Drug (IND) application in Q1 2025 and expects to have proof-of-concept by YE 2025.

ALLO-316: TRAVERSE Trial in Renal Cell Carcinoma (RCC)
In April 2024, the Company announced a $15 million grant from the California Institute for Regenerative Medicine (CIRM) to support the ongoing TRAVERSE trial with ALLO-316 in RCC.

The Company has developed and implemented a diagnostic and treatment algorithm in the TRAVERSE trial that may mitigate the treatment-associated hyperinflammatory response without compromising the CAR T function needed to eradicate solid tumors. This builds upon the field’s understanding of how certain drugs can act as a "safety key" to mitigate hyperinflammatory response without compromising CAR T function or efficacy.

Details on this potentially cornerstone discovery in the Phase 1 TRAVERSE trial are planned for release Q2 2024. A Phase 1 data update from approximately 20 patients with CD70 positive RCC is planned by YE 2024.

2024 First Quarter Financial Results
•Research and development expenses were $52.3 million for the first quarter of 2024, which includes $3.8 million of non-cash stock-based compensation expense.
•General and administrative expenses were $17.3 million for the first quarter of 2024, which includes $8.1 million of non-cash stock-based compensation expense.
•Net loss for the first quarter of 2024 was $65.0 million, or $0.38 per share, including non-cash stock-based compensation expense of $11.9 million.
•The Company had $397.3 million in cash, cash equivalents, and investments as of March 31, 2024.

Based on the cash runway as of March 31, 2024, the Company expects its cash runway to fund operations into 2026. The Company expects a decrease in cash, cash equivalents, and investments of approximately $200 million in 2024. GAAP

Operating Expenses are expected to be approximately $300 million, including estimated non-cash stock-based compensation expense of approximately $60 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 2:00 p.m. Pacific Time / 5:00 p.m. Eastern Time to discuss financial results and provide a business update. If you would like the option to ask a question on the conference call, please use this link to register. Upon registering for the conference call, you will receive a personal PIN to access the call, which will identify you as the participant and allow you the option to ask a question. The listen-only webcast will be made available on the Company’s website at www.allogene.com under the Investors tab in the News and Events section. Following the live audio webcast, a replay will be available on the Company’s website for approximately 30 days.

About Cemacabtagene Ansegedleucel (Previously Known as ALLO-501A) Cemacabtagene ansegedleucel, or cema-cel is a next generation anti-CD19 AlloCAR T investigational product for the treatment of large B cell lymphoma (LBCL). The ALPHA3 pivotal Phase 2 trial in first line (1L) consolidation for the treatment of LBCL is expected to begin mid-2024. In June 2022, the U.S. Food and Drug Administration granted Regenerative Medicine Advanced Therapy (RMAT) designation to cema-cel in third line (3L) r/r LBCL.