Atara Biotherapeutics Submits Tabelecleucel (Tab-cel®) Biologics License Application for Treatment of Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease with U.S. FDA

Rhea-AI Summary

Atara Biotherapeutics has submitted a Biologics License Application (BLA) to the U.S. FDA for tabelecleucel (tab-cel®), targeting Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) in patients aged two and older.

This marks the first allogeneic T-cell therapy BLA submission for the FDA. If approved, tab-cel would be the first FDA-approved therapy for EBV+ PTLD, potentially triggering milestone payments of up to $80 million from Pierre Fabre Laboratories.

Supported by data from over 430 patients, including a 48.8% Objective Response Rate from the ALLELE study, tab-cel has shown a favorable safety profile. The BLA submission is part of a broader global partnership with Pierre Fabre, which includes double-digit tiered royalties on net sales and reimbursement for development costs.

Positive

• Submission of BLA for tabelecleucel (tab-cel®) to U.S. FDA.
• First potential FDA-approved therapy for EBV+ PTLD.
• Potential milestone payments: $20 million upon BLA acceptance and $60 million upon FDA approval.
• Supported by pivotal data with a 48.8% Objective Response Rate.
• Favorable safety profile demonstrated in studies.
• Strategic global partnership with Pierre Fabre.

Negative

• Clinical trial success doesn't guarantee FDA approval.
• Potential delays and costs associated with FDA review process.
• Dependence on milestone payments, contingent on regulatory success.
• Market competition from existing and emerging therapies.

Financial Analyst

Atara Biotherapeutics' submission of a Biologics License Application (BLA) for tabelecleucel (tab-cel) is a noteworthy event for retail investors. Should the FDA approve this application, it would trigger a $20 million milestone payment from Pierre Fabre Laboratories, with an additional $60 million upon approval. These payments represent significant revenue inflows for Atara, which can positively impact their short-term liquidity and financial stability.

The breakthrough therapy designation and orphan drug status of tab-cel suggest potential market exclusivity, offering Atara a competitive advantage. Investors should consider that while regulatory approvals in Europe and the UK add credibility, the decisive financial impact will stem from the U.S. market due to its size and healthcare spending capacity. Potential double-digit tiered royalties on net sales also provide a substantial long-term revenue stream.

However, investors must be aware of the inherent risks associated with FDA reviews and the competitive landscape of T-cell immunotherapies. The acceptance and approval by the FDA could take several months and competition from other biotech firms remains a variable that could influence Atara's market share and stock performance.

Oncology Doctor

The submission of tab-cel for FDA approval is a promising development in the treatment of Epstein-Barr Virus Positive Post-Transplant Lymphoproliferative Disease (EBV+ PTLD), a condition currently lacking approved therapies. As an allogeneic T-cell therapy, tab-cel is designed to target and eliminate EBV-infected cells. The pivotal ALLELE study data demonstrating a 48.8% objective response rate (ORR) is impressive, especially in a disease context where treatment options are limited.

The favorable safety profile reported aligns with previous analyses, suggesting that tab-cel could become a valuable option for managing EBV+ PTLD, especially for patients refractory to rituximab. The clinical community will closely monitor the FDA's review process, as approval could change the treatment landscape for post-transplant patients facing lymphoproliferative disease.

Market Research Analyst

Atara Biotherapeutics' partnership with Pierre Fabre Laboratories is strategically significant. The partnership not only includes milestone payments contingent upon FDA acceptance and approval but also involves reimbursement for global development costs and inventory purchases. This approach mitigates Atara's financial burden while ensuring readiness for market launch.

The previous approvals in Europe, the UK and Switzerland for Ebvallo reflect the drug's potential efficacy and safety, which can positively influence its acceptance in the U.S. market. Investors should consider the potential market size for EBV+ PTLD treatments and the growth trajectory, especially given the expanding global reach that this partnership facilitates. Nevertheless, market penetration and competitive positioning will be important metrics to track post-approval.

First Allogeneic T-Cell Therapy BLA Submission to U.S. Food and Drug Administration

If Approved, Tab-cel Would be First Approved Therapy in U.S. for EBV+ PTLD

Acceptance of BLA Will Trigger $20 Million Milestone Payment from Pierre Fabre Laboratories, with Potential for Additional $60 Million Milestone Upon FDA Approval

THOUSAND OAKS, Calif.--(BUSINESS WIRE)-- Atara Biotherapeutics, Inc. (Nasdaq: ATRA), a leader in T-cell immunotherapy, leveraging its novel allogeneic Epstein-Barr virus (EBV) T-cell platform to develop transformative therapies for patients with cancer and autoimmune diseases, today announced that Atara has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for tabelecleucel (tab-cel^®) indicated as monotherapy for treatment of adult and pediatric patients two years of age and older with Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. There are no FDA approved therapies in this treatment setting.

“The BLA submission for tab-cel represents a significant moment for Atara, our partner Pierre Fabre, and the broader allogeneic T-cell therapy field, and is a critical step towards our goal of delivering this first-of-its-kind treatment to EBV+ PTLD patients in the U.S.,” said Pascal Touchon, President and Chief Executive Officer of Atara. “I would like to thank the patients and physicians who participated in the tab-cel clinical trials, our long-time collaborators at Memorial Sloan Kettering Cancer Center, as well as our internal teams for their remarkable dedication and hard work. We now look forward to continued collaboration with the FDA on its review and with Pierre Fabre as they actively prepare for the potential launch of this innovative therapy in the U.S.”

Tab-cel is an allogeneic, EBV-specific T-cell immunotherapy designed to target and eliminate EBV-infected cells. The BLA is supported by pivotal and supportive data covering more than 430 patients treated with tab-cel across multiple life-threatening diseases including the latest pivotal ALLELE study data that demonstrated a statistically significant 48.8% Objective Response Rate (ORR) (p<0.0001) and favorable safety profile consistent with previous analyses.

Tab-cel has been granted Breakthrough Therapy Designation for the treatment of rituximab-refractory EBV-associated lymphoproliferative disease by the U.S. FDA and has orphan drug designation.

In December 2023, Atara announced the closing of the expanded global partnership with Pierre Fabre Laboratories for the U.S. and remaining global commercial markets for tab-cel, building on an initial partnership covering Europe, Middle East, Africa, and other select emerging markets. With the completion of the tab-cel BLA submission, Atara continues to advance the Pierre Fabre expanded global partnership, which includes potential milestone payments of $20 million and $60 million from Pierre Fabre contingent upon the successful FDA acceptance and approval of the tab-cel BLA, respectively. In addition, Pierre Fabre is reimbursing Atara for expected tab-cel global development costs through the BLA transfer and purchasing tab-cel inventory through the manufacturing transfer date. Atara is also eligible to receive double-digit tiered royalties on net sales of tab-cel in the U.S. and remaining global commercial markets referenced above.

Tab-cel was granted marketing authorization under the brand name Ebvallo™ in December 2022 by the European Commission. Marketing authorization was also granted by the Medicines and Healthcare Products Regulatory Agency in the United Kingdom in May 2023 and by Swissmedic in Switzerland in May 2024. In all three territories, Ebvallo is indicated as monotherapy for the treatment of adult and pediatric patients two years of age and older with relapsed or refractory EBV+ PTLD who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate.

About Atara Biotherapeutics, Inc.

Atara is harnessing the natural power of the immune system to develop off-the-shelf cell therapies for difficult-to-treat cancers and autoimmune conditions that can be rapidly delivered to patients from inventory. With cutting-edge science and differentiated approach, Atara is the first company in the world to receive regulatory approval of an allogeneic T-cell immunotherapy. Our advanced and versatile T-cell platform does not require T-cell receptor or HLA gene editing and forms the basis of a diverse portfolio of investigational therapies that target EBV, the root cause of certain diseases, in addition to next-generation AlloCAR-Ts designed for best-in-class opportunities across a broad range of hematological malignancies and B-cell driven autoimmune diseases. Atara is headquartered in Southern California. For more information, visit atarabio.com and follow @Atarabio on X and LinkedIn.

Forward-Looking Statements

This press release contains or may imply "forward-looking statements" within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. For example, forward-looking statements include statements regarding the development, timing and progress of tab-cel^®, including a potential BLA acceptance, the potential characteristics and benefits of tab-cel^®, the indication(s) for which tab-cel could potentially obtain FDA approval for, and the progress and results of, and prospects for, the expanded global partnership with Pierre Fabre Laboratories involving tab-cel^®, including Pierre Fabre activities relating to the potential commercialization of tab-cel, and the potential financial benefits to Atara as a result of the expanded global partnership with Pierre Fabre Laboratories, including any payments thereunder. Because such statements deal with future events and are based on Atara’s current expectations, they are subject to various risks and uncertainties and actual results, performance or achievements of Atara could differ materially from those described in or implied by the statements in this press release. These forward-looking statements are subject to risks and uncertainties, including, without limitation, risks and uncertainties associated with the costly and time-consuming pharmaceutical product development process and the uncertainty of clinical success; the COVID-19 pandemic and the wars in Ukraine and the Middle East, which may significantly impact (i) our business, research, clinical development plans and operations, including our operations in Southern California and Denver and at our clinical trial sites, as well as the business or operations of our third-party manufacturer, contract research organizations or other third parties with whom we conduct business, (ii) our ability to access capital, and (iii) the value of our common stock; the sufficiency of Atara’s cash resources and need for additional capital; and other risks and uncertainties affecting Atara and its development programs, including those discussed in Atara’s filings with the Securities and Exchange Commission, including in the “Risk Factors” and “Management’s Discussion and Analysis of Financial Condition and Results of Operations” sections of the Company’s most recently filed periodic reports on Form 10-K and Form 10-Q and subsequent filings and in the documents incorporated by reference therein. Except as otherwise required by law, Atara disclaims any intention or obligation to update or revise any forward-looking statements, which speak only as of the date hereof, whether as a result of new information, future events or circumstances or otherwise.

View source version on businesswire.com: https://www.businesswire.com/news/home/20240520985176/en/

Investor and Media Relations

Jason Awe, Ph.D.

Senior Director, Corporate Communications & Investor Relations

(805) 217-2287

jawe@atarabio.com

Source: Atara Biotherapeutics, Inc.

FAQ

What is the latest update on Atara's tabelecleucel (tab-cel®)?

Atara Biotherapeutics has submitted a Biologics License Application (BLA) to the U.S. FDA for tabelecleucel (tab-cel®), targeting EBV+ PTLD.

What is the significance of the BLA submission for Atara's stock (ATRA)?

The BLA submission could lead to the first FDA-approved therapy for EBV+ PTLD, potentially triggering milestone payments of up to $80 million and positively impacting ATRA stock.

How has tab-cel performed in clinical studies?

Tab-cel has demonstrated a 48.8% Objective Response Rate and a favorable safety profile in the ALLELE study and other trials.

What are the financial implications of Atara's partnership with Pierre Fabre?

The partnership includes potential milestone payments, reimbursement for development costs, and double-digit tiered royalties on net sales.

What are the risks associated with tabelecleucel's (tab-cel®) FDA approval?

There are risks of potential delays, costs associated with the FDA review process, and the possibility of not gaining approval.