• 100% (9/9) overall response rate and 67% (6/9) complete response rate (MRD 10^-5) observed in heavily pre-treated patients with daratumumab relapsed/refractory AL Amyloidosis as of the September 20, 2023 data cutoff
• 100% (4/4) overall response rate and 75% (3/4) complete response rate observed in t(11;14) relapsed/refractory AL Amyloidosis
• Best responder duration of response was 19.2 months with response ongoing; median follow-up of 7.3 months (range: 2.5 – 16.5 months) as of the data cutoff date of September 20, 2023
• Nexcella plans to submit a BLA for FDA approval in AL Amyloidosis once 40 patients are treated with NXC-201
• The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate

LOS ANGELES, Oct. 03, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced presentation of additional AL Amyloidosis clinical data from its Phase 1b/2a NEXICART-1 (NCT04720313) study of novel, autologous, BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy, NXC-201, at an oral presentation at the 20^th International Myeloma Society Annual (IMS) Meeting being held in Athens, Greece on September 27-30 2023. One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update. All patients were DARZALEX® (daratumumab) combination therapy relapsed/refractory and experienced a median of 6 earlier treatments that failed to stop worsening of disease (lines of therapy) prior to receiving NXC-201.

“There are no approved drugs for relapsed/refractory AL Amyloidosis,” said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and principal study investigator. “NXC-201’s 100% response rate in relapsed/refractory AL amyloidosis patients, including t(11;14) and cardiac involved, indicates a potential broad mechanism of action.  Additionally, as a one-time treatment, NXC-201 would present an attractive alternative to multi-drug combination, long-term daily or weekly regimens for relapsed/refractory AL amyloidosis patients.”

“Currently, AL Amyloidosis treatment involves repeat dosing and weekly distant travel to academic medical centers,” said Ilya Rachman, M.D., Ph.D., Executive Chairman of Nexcella. “One-time treatment with NXC-201 could fill the void for relapsed/refractory AL Amyloidosis, where there are no therapies approved today, while restoring quality of life.”  

Gabriel Morris, President of Nexcella, added, “NXC-201’s uniquely favorable CAR-T tolerability profile and an apparent ability to clear disease-causing amyloid chains from the body within ~30 days could make it particularly suitable for treatment in a potential outpatient setting.”

Nexcella Announces Complete Response in 9th Relapsed/Refractory AL Amyloidosis Patient in NXC-201 Clinical Trial at IMS 20th Annual Meeting

Data were presented from 9 relapsed/refractory AL amyloidosis patients (including one new patient) in the ongoing Phase 1b/2a NEXICART-1 study of NXC-201 (formerly HBI0101). Patients were infused with CAR+T cells at doses of 150 x 10⁶ (n=1), 450 x 10⁶ (n=2), and 800 x 10⁶ (n=6).
Of the 9 patients, 7/9 patients had cardiac involvement and 4/9 patients were t(11;14) relapsed/refractory.  Median follow-up was 7.3 months (range: 2.5 – 16.5 months) as of the September 20, 2023 data cutoff.  Data highlights:

 9 relapsed/refractory AL amyloidosis patients (median 6 lines of prior therapy):

• Overall response rate of 100% (9/9)
• Complete response rate of 67% (6/9) (MRD 10^-5)
• Organ response rate of 56% (5/9)
• Best responder had a duration of response of 19.2 months as of the data cutoff of September 20, 2023, with response ongoing
• There were no immune effector cell-associated neurotoxicity syndrome (ICANS) events or grade 4 cytokine release syndrome (CRS) events reported
• 44% (4/9) had MAYO-stage IIIa/IIIb disease

For the 7 relapsed/refractory AL Amyloidosis patients with cardiac involvement:

• Overall response rate of 100% (7/7)
• Complete response rate of 57% (4/7) (MRD 10^-5)
• Organ response rate of 57% (4/7)

4 patients with t(11;14) relapsed/refractory AL Amyloidosis:

• Overall response rate of 100% (4/4)
• Complete response rate of 75% (3/4) (MRD 10^-5)
• Organ response rate of 50% (2/4)

The 20^th IMS AL Amyloidosis presentation can be accessed on the Nexcella corporate website at this link: https://nexcella.com/publications/
Oral Presentation:

About NEXICART-1

NEXICART-1 (NCT04720313) is an ongoing Phase 1b/2a, open-label study evaluating the safety and efficacy of NXC-201 (formerly HBI0101), in adults with relapsed or refractory multiple myeloma and AL amyloidosis.

The primary objective of the Phase 1b portion of the study was to characterize the safety and confirm the recommended Phase 2 dose (RP2D) and Phase 2 dose of NXC-201. The Phase 2 portion of the study will evaluate the efficacy and safety of NXC-201 with endpoints of overall survival, progression-free survival and response rates, according to International Myeloma Working Group (IMWG) Uniform Response Criteria.

The Phase 1b portion of the ongoing Phase 1b/2a clinical trial has been successful in determining the recommended Phase 2 dose (RP2D) of 800 million CAR+T cells. The expected primary endpoint for the Phase 2 portion of the ongoing Phase 1b/2a clinical trial of NXC-201 in relapsed/refractory multiple myeloma is overall response rate and duration of response.  Nexcella plans to submit data to the FDA in multiple myeloma once 100 patients are treated with NXC-201. The expected primary endpoint for NXC-201 in relapsed/refractory AL Amyloidosis is overall response rate. Nexcella plans to submit data to the FDA in AL amyloidosis once 30-40 patients are treated with NXC-201.

About NXC-201

NXC-201 (formerly HBI0101) is a BCMA-targeted investigational chimeric antigen receptor T (CAR-T) cell therapy that is being studied in a comprehensive clinical development program for the treatment of patients with relapsed or refractory multiple myeloma and AL amyloidosis.

About AL Amyloidosis

AL amyloidosis is a rare systemic disorder caused by an abnormality of plasma cells in the bone marrow. Misfolded amyloid proteins produced by plasma cells buildup in and around tissues, nerves and organs, gradually affecting their function. This can cause progressive and widespread organ damage, and high mortality rates.

AL amyloidosis affects roughly 30,000 – 40,000 patients in total throughout the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases of AL amyloidosis annually in the U.S. The annual global incidence of AL Amyloidosis is ~15,000 patients.

The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research.

About Nexcella, Inc.

Nexcella, Inc. is a Los Angeles, California based clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications. Our lead candidate, next generation BCMA-targeted CAR-T NXC-201 for relapsed/refractory multiple myeloma and relapsed/refractory AL amyloidosis, has produced 95% and 100% response rates in each indication, respectively, as of July 17, 2023, across 72 patients. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both multiple myeloma and AL Amyloidosis. We believe NXC-201 has potential to be the world’s first outpatient CAR-T. Our N-GENIUS platform allows us to discover, develop, and manufacture cutting-edge cell therapies for patients in need. To learn more about Nexcella, Inc. visit us at www.nexcella.com

Forward Looking Statements

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Contacts

Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com

Company Contact

irteam@nexcella.com

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• Nexcella Announces Complete Response in 9th Relapsed