Athenex, Texas Children’s Cancer Center, and the Center for Cell and Gene Therapy at Baylor College of Medicine to Present Phase 1 Clinical Data for KUR-501, an autologous GD2 CAR-NKT Cell Therapy for Relapsed/Refractory High Risk Neuroblastoma at the ASGCT 25th Annual Meeting

Rhea-AI Summary

Athenex (NASDAQ: ATNX) announces promising results from the KUR-501 trial, presented at the ASGCT 25th Annual Meeting. The trial demonstrated a 25% Overall Response Rate (ORR) in patients with relapsed/refractory neuroblastoma, with a Disease Control Rate (DCR) of 58%. Notably, a durable Complete Response was observed in one patient lasting 12 months. The therapy was well tolerated, with no severe toxicities reported. The study aims to validate CAR-NKT cell therapy targeting GD2 in high-risk neuroblastoma and is ongoing at Texas Children’s Cancer Center and Baylor College of Medicine.

Positive

• 25% Overall Response Rate (ORR) in the trial.
• 58% Disease Control Rate (DCR).
• Durable Complete Response lasting 12 months.
• Well tolerated with no dose-limiting toxicities.

Negative

• None.

• Data presented by investigators from the Texas Children’s Cancer Center’s Center for Advanced Innate Cell Therapy and Baylor College of Medicine
• Data demonstrate expansion of CAR-NKT cells post-transfer in all patients, and objective responses in patients with relapsed/refractory neuroblastoma
• Evidence of therapeutic efficacy with 25% (3/12) Overall Response Rate (ORR), 58% (4 stable disease, 2 partial responses, 1 complete response/12) Disease Control Rate (DCR), and 2 out of 3 responses at dose level 4 (1x10⁸/m²)
• Durable Complete Response persisting 12 months
• Analysis found that responders correlate with CD62L+ NKT frequency in the pre-infusion products as well as CAR-NKT area under the curve (AUC) in the peripheral blood post-infusion
• Well tolerated with no dose limiting toxicity; no immune associated neurotoxicity syndrome (ICANS) at the first four dose levels and one case of grade 2 CRS

BUFFALO, N.Y., May 02, 2022 (GLOBE NEWSWIRE) -- Athenex (NASDAQ: ATNX), a global biopharmaceutical company dedicated to the discovery, development, and commercialization of novel therapies for the treatment of cancer and related conditions, today announced that the Company will present at the upcoming ASGCT 25^th Annual Meeting, taking place May 16-19, 2022, in Washington, D.C. The abstract associated with the presentation is now available on the conference website.

Details of the presentations are as follows:

Oral Presentation Details

Abstract 54: Anti-GD2 CAR NKT cells are safe and produce antitumor responses in patients with relapsed/refractory neuroblastoma
Date/Time: Monday, May 16, 2022, 2:30 PM – 2:45 PM ET
Session: Gene and Cell Therapy Trials in Progress
Presenter: Dr. Andras Heczey, Baylor College of Medicine
Location: Room 201, Walter E. Washington Convention Center, 801 Mt Vernon Pl NW Washington, D.C.

“Observing evidence of a dose response is extremely encouraging, and we are excited to see two out of three responses at 100 million cells/m²,” said Dan Lang, M.D., President of Athenex Cell Therapy. “We are hopeful that we will see additional responses as we move to higher doses. We and our partners at Baylor College of Medicine (BCM) and Texas Children’s Hospital (TCH) continue to be motivated by the data and look forward to our next update.”

About KUR-501

KUR-501, is an autologous NKT cell CAR product that targets GD2, a tumor-associated antigen that is expressed on almost all neuroblastomas as well as melanomas, some sarcomas, and a variety of other tumors. GINAKIT2, a phase 1 single arm clinical study (NCT03294954) of KUR-501, in patients with refractory or recurrent high-risk neuroblastoma will evaluate up to six dose levels of KUR-501 following lymphodepletion with cyclophosphamide and fludarabine.

Neuroblastoma, a pediatric cancer of the sympathetic nervous system, typically the adrenal gland, is associated with a poor prognosis in children with high-risk disease and is a significant unmet medical need. The KUR-501 development program will provide autologous proof-of-concept for CAR-NKT cells in solid tumors using a validated target.

The GINAKIT2 study is supported by Athenex, Inc., which acquired Kuur Therapeutics in May 2021, as well as by Alex’s Lemonade Stand Foundation. This study is currently recruiting patients at BCM/TCH.

About Athenex, Inc.

Founded in 2003, Athenex, Inc. is a global clinical-stage biopharmaceutical company dedicated to becoming a leader in the discovery, development, and commercialization of next generation cell therapy drugs for the treatment of cancer. In pursuit of this mission, Athenex leverages years of experience in research and development, clinical trials, regulatory standards, and manufacturing. The Company’s current clinical pipeline is derived mainly from the following core technologies: (1) Cell therapy based on NKT cells, (2) Orascovery, based on a P-glycoprotein inhibitor, and (3) Src Kinase Inhibition. Athenex’s employees worldwide are dedicated to improving the lives of cancer patients by creating more active, accessible and tolerable treatments. For more information, please visit www.athenex.com.

Forward-Looking Statements

Except for historical information, all of the statements, expectations, and assumptions contained in this press release are forward-looking statements. These forward-looking statements are typically identified by terms such as “believe,” “look forward to,” “potential,” and similar expressions. Actual results might differ materially from those explicit or implicit in the forward-looking statements. Important factors that could cause actual results to differ materially include: our history of operating losses and the substantial doubt about our ability to continue as a going concern; our strategic pivot to focus on our cell therapy platform and our plan to dispose of non-core assets; our ability to obtain financing to fund operations, successfully redirect our resources and reduce our operating expenses; our ability to refinance, extend or repay our substantial indebtedness owed to our senior secured lender; the development stage of our primary clinical candidates, including NKT Cell Therapy and related risks involved in drug development, clinical trials, regulation, uncertainties around regulatory reviews and approvals; the preclinical and clinical results for Athenex’s drug candidates, which may not support further development of such drug candidates; the Company’s ability to successfully demonstrate the safety and efficacy of its drug candidates and gain approval of its drug candidates on a timely basis, if at all; the uncertainty of ongoing legal proceedings; risks related to our ability to successfully integrate the business of Kuur into our existing businesses, including uncertainties associated with maintaining relationships with customers, vendors and employees, as well as differences in operations, cultures, and management philosophies that may delay successful integration and our ability to support the added cost burden of Kuur’s business; risks related to counterparty performance, including our reliance on third parties for success in certain areas of Athenex’s business; risks and uncertainties inherent in litigation, including purported stockholder class actions; the impact of the COVID-19 pandemic and other macroeconomic factors, like the war in Ukraine, and their ongoing impact on our operations, supply chain, cash flow and financial condition; competition; intellectual property risks; risks relating to doing business internationally and in China; the risk of development, operational delays, production slowdowns or stoppages or other interruptions at our manufacturing facility as well as our ability to find alternative sources of supply to meet our obligations and requirements; and the other risk factors set forth from time to time in our SEC filings, copies of which are available for free in the Investor Relations section of our website at http://ir.athenex.com/phoenix.zhtml?c=254495&p=irol-sec or upon request from our Investor Relations Department. All information provided in this release is as of the date hereof and we assume no obligation and do not intend to update these forward-looking statements, except as required by law.

Athenex Contacts

Investors

Daniel Lang, MD
Athenex, Inc.
Email: danlang@athenex.com

Caileigh Dougherty
Athenex, Inc.
Email: cdougherty@athenex.com

FAQ

What were the results of the KUR-501 clinical trial presented by Athenex?

The KUR-501 trial showed a 25% Overall Response Rate and a 58% Disease Control Rate in patients with relapsed/refractory neuroblastoma.

When and where was the KUR-501 trial data presented?

The data was presented at the ASGCT 25th Annual Meeting on May 16, 2022, in Washington, D.C.

What is the purpose of the KUR-501 clinical trial?

The trial aims to evaluate the safety and therapeutic efficacy of CAR-NKT cells targeting GD2 in patients with high-risk neuroblastoma.

What findings were reported regarding the safety of KUR-501?

KUR-501 was well tolerated, with no severe toxicities reported during the first four dose levels.

What implications do the trial results have for Athenex's market outlook?

The positive trial results for KUR-501 may enhance Athenex's market position and pave the way for future developments in cancer therapy.