Atara Biotherapeutics and Pierre Fabre Laboratories Announce Publication of Phase 3 ALLELE Tab-cel® Data in The Lancet Oncology

Rhea-AI Summary

Atara Biotherapeutics and Pierre Fabre Laboratories announced the publication of pivotal Phase 3 ALLELE study data for tab-cel, an allogeneic T-cell therapy for EBV+ PTLD patients. The study showed a 51.2% objective response rate and a 23.0 month median duration of response. The U.S. BLA submission for tab-cel is on track for Q2 2024. The study met its primary endpoint, and tab-cel was well-tolerated with no reported adverse events. These results highlight the clinical value of tab-cel and its potential to address an urgent unmet medical need for patients with limited treatment options.

Insights

Medical Research Analyst

The recent publication of Phase 3 results for Atara Biotherapeutics' allogeneic T-cell therapy, tabelecleucel, marks a significant milestone in the field of immunotherapy and has implications for the business landscape of biopharmaceutical companies. This therapy targets Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD), a condition with limited treatment options. The reported 51.2% objective response rate (ORR) and a median duration of response of 23.0 months suggest a substantial clinical benefit, which is crucial for patients with r/r EBV+ PTLD.

The data indicating a favorable safety profile, with no reports of severe adverse effects commonly associated with T-cell therapies, such as cytokine release syndrome or graft-versus-host disease, could position tabelecleucel as a preferable treatment option. This could lead to increased demand and market penetration upon FDA approval. For investors, the potential for expanding into broader indications, as suggested by the ongoing Phase 2 EBVision trial, represents an opportunity for growth in Atara's product portfolio and an increase in its addressable market.

Healthcare Economist

From an economic standpoint, the successful development and commercialization of tabelecleucel can have a significant impact on the healthcare system. The treatment of rare diseases such as EBV+ PTLD often involves high costs due to the complexity of the therapy and the specialized care required. The introduction of tabelecleucel, particularly with its long duration of response, could potentially reduce the overall healthcare burden by decreasing the need for subsequent treatments and hospitalizations.

However, the cost-effectiveness of this therapy will be a critical factor for its adoption. Payers will evaluate the long-term benefits against the upfront costs, which are typically high for novel cellular therapies. For Atara Biotherapeutics and its partner, Pierre Fabre Laboratories, pricing strategies will need to balance profitability with access to ensure market uptake. The partnership's ability to navigate reimbursement landscapes across different countries will be pivotal in realizing the full economic potential of the drug.

Market Research Analyst

In terms of market dynamics, the European Commission's approval of tabelecleucel under the brand name EBVALLO™ and the planned U.S. BLA submission in Q2 2024 represent key steps in Atara's strategy to establish a foothold in the oncology market. The company's expanded global partnership with Pierre Fabre Laboratories could enhance its commercial capabilities, leveraging Pierre Fabre's existing oncology infrastructure for effective market penetration.

Investors should monitor Atara's progress towards FDA approval, as the U.S. market presents a significant opportunity for revenue growth. The company's stock performance could be influenced by regulatory developments, commercialization efforts and the reception of tabelecleucel in the medical community. The real-world data from the Expanded Access Program study indicating a 66.7% ORR further supports the therapy's efficacy and may bolster confidence among stakeholders.

First Phase 3 Results Published for an Allogeneic T-Cell Therapy

Significant 51.2% Objective Response Rate and 23.0 Month Median Duration of Response in Relapsed or Refractory EBV+ PTLD Patients

U.S. BLA on Track for Submission in Q2 2024 Based on Strong Clinical File

THOUSAND OAKS, Calif. & CASTRES, France--(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, and Pierre Fabre Laboratories, a global player in oncology and responsible for worldwide commercialization of tabelecleucel (tab-cel^® or EBVALLO™), today announced that data from the pivotal Phase 3 ALLELE study of tab-cel, approved in the European Union in adults and children two years of age and older with relapsed or refractory (r/r) Epstein-Barr virus positive post-transplant lymphoproliferative disease (EBV+ PTLD) following solid organ transplant (SOT) or hematopoietic cell transplant (HCT), were published for the first time online in The Lancet Oncology.

The data were published in an article titled, “Tabelecleucel for allogeneic haematopoietic stem-cell or solid organ transplant recipients with Epstein–Barr virus-positive post-transplant lymphoproliferative disease after failure of rituximab or rituximab and chemotherapy (ALLELE): a phase 3, multicentre, open-label trial,” and can be accessed at the following link: https://www.thelancet.com/journals/lanonc/article/PIIS1470-2045(23)00649-6/fulltext

“The results of the ALLELE study highlight the clinical value of tab-cel, which is now EMA and MHRA approved, and is being made available to patients in Europe through our partner Pierre Fabre Laboratories as a first-of-its-kind treatment for those with a devastating disease that previously had limited treatment options,” said Pascal Touchon, President and Chief Executive Officer of Atara. “As we prepare for our tab-cel BLA submission in the second quarter 2024, we look forward to interacting with the FDA to progress towards approval based on our robust clinical data.”

As reported in The Lancet Oncology publication, the ALLELE study met its primary endpoint. 22 of 43 EBV+ PTLD patients achieved an objective response (51.2% objective response rate, or ORR). Those that responded to tab-cel had longer survival, with an estimated one-year overall survival of 84.4% (95% CI: 58.9, 94.7) for responders versus 34.8% (95% CI: 14.6, 56.1) for non-responders. The median duration of response was 23.0 months and the median overall survival was 18.4 months. Tab-cel was well tolerated with no reports of tumor flare reaction, cytokine release syndrome or immune effector cell-associated neurotoxicity syndrome, and no events of graft-versus-host disease or SOT rejection as related to tab-cel. These interim data were previously presented at the 2022 American Society of Hematology (ASH) Annual Meeting.

These pivotal trial data are supported by a recent updated analysis from the October 2023 data cut of the ongoing ALLELE study that continued to demonstrate a statistically significant 49% ORR (p<0.0001), consistent durability of response, estimated OS, and favorable safety profile in the intended population for the proposed U.S. label. ln addition, real-world results from the multicenter Expanded Access Program study in Europe demonstrated an ORR of 66.7% in 24 EBV+ PTLD patients and were presented at the 2023 American Society of Clinical Oncology (ASCO) Annual Meeting.

“Patients with relapsed or refractory EBV+ PTLD have limited treatment options and poor overall survival measured in only weeks to months,” said Susan Prockop, MD, lead investigator, Boston Children’s Hospital-Dana Farber Cancer Institute. “These clinically meaningful data reinforce the life-saving potential of tabelecleucel for these patients, for whom there are no approved therapies in the U.S. and helps address an urgent unmet medical need.”

Tab-cel was granted marketing authorization under the brand name EBVALLO™ in December 2022 by the European Commission (EC) as a monotherapy for the treatment of adult and pediatric patients two years of age and older with r/r EBV+ PTLD who have received at least one prior therapy. For solid organ transplant patients, prior therapy includes chemotherapy unless chemotherapy is inappropriate. In the United States, Atara plans to submit a biologics license application (BLA) to the U.S. Food and Drug Administration for tab-cel for the treatment of EBV+ PTLD in the second quarter of 2024. Additionally, in December 2023, Atara reported the first results from the ongoing Phase 2 EBVision trial, which has the potential to further extend the clinical experience and potential of tab-cel into broader indications.

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 tabelecleucel, building on an initial partnership covering Europe, Middle East, Africa, and other select emerging markets.

"Current results from the first global, multicenter, open-label Phase 3 study of the new allogeneic T-cell therapy, tabelecleucel, show significant clinical benefit and a favorable safety profile in a severely affected population. These results bring a lot of hope for patients, and confirm the innovative nature of this treatment, also recognized through the Prix Galien prize that we received in France. With the recent EU marketing authorization, EBVALLO™ is the first EBV-specific allogeneic T-cell therapy available for patients with r/r EBV+ PTLD after HCT or SOT and their families. All this resonates perfectly with our purpose ‘every time we care for a single person, we make the whole world better,’” said Núria Perez-Cullell, Director of Medical Affairs, Patients & Consumers at Pierre Fabre Laboratories.

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 within days. 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 Epstein-Barr virus (EBV) 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.

About Pierre Fabre Laboratories

Pierre Fabre Laboratories is a leading French medical and beauty care company with 4 decades of experience in innovation, development, manufacturing, and commercialization in oncology. The company dedicated about 80% of its R&D spendings to oncology in 2022 with a focus on targeted therapies. Its current commercial portfolio in oncology covers colorectal, breast and lung cancers, melanoma, hematology, and pre-cancerous skin conditions like actinic keratosis.

In 2022, Pierre Fabre Laboratories posted 2.7 billion euros in revenues, 69% of which came from international sales in 120 countries. Established in the South-West of France since its creation in 1962, the Group manufactures 90% of its products in France and employs some 10 000 people worldwide. The company is 86%-owned by the Pierre Fabre Foundation, a government-recognized public-interest foundation, and by its own employees through an international employee stock ownership plan. Pierre Fabre Laboratories’ sustainability policy has been assessed by the independent AFNOR Certification body at the "Exemplary" level of its CSR label (ISO 26 000 standard for sustainable development).

Further information about Pierre Fabre Laboratories can be found at www.pierre-fabre.com, @PierreFabre.

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, data, timing and progress, as applicable, of: (1) the tab-cel program, including a potential BLA submission for tab-cel in the United States; (2) the potential characteristics and benefits of tab-cel, including data and analyses from the ALLELE study and timing of when such data will be received and communicated; and (3) the amended and restated commercialization agreement with Pierre Fabre. 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 ongoing 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’s 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/20240131800324/en/

Atara Investor and Media Relations:

Alex Chapman

Vice President, Corporate Communications & Investor Relations

(805) 456-4772

achapman@atarabio.com

Jason Awe, Ph.D.

Senior Director, Corporate Communications & Investor Relations

(805) 217-2287

jawe@atarabio.com

Pierre Fabre Laboratories Media Contact:

Laurence Marchal

+33 7 88 88 54 47

laurence.marchal@pierre-fabre.com

Source: Atara Biotherapeutics, Inc.

FAQ

What is the objective response rate for tab-cel in the Phase 3 ALLELE study?

The objective response rate for tab-cel in the Phase 3 ALLELE study was 51.2%.

What is the median duration of response for tab-cel in the Phase 3 ALLELE study?

The median duration of response for tab-cel in the Phase 3 ALLELE study was 23.0 months.

When is the U.S. BLA submission for tab-cel scheduled?

The U.S. BLA submission for tab-cel is on track for the second quarter of 2024.

What were the primary endpoint results of the ALLELE study for tab-cel?

The ALLELE study for tab-cel met its primary endpoint.

What adverse events were reported for tab-cel in the ALLELE study?

Tab-cel was well-tolerated with no reports of adverse events in the ALLELE study.