U.S. Food and Drug Administration Approves Orphan Drug Designation for Nexcella NXC-201 as a Treatment for Amyloid Light Chain (AL) Amyloidosis

FDA Orphan Drug Designation (“ODD”) qualifies one-time treatment NXC-201 for:

• 7 years of U.S. market exclusivity after approval
• Tax credits for qualified clinical testing
• Waiver of the Prescription Drug User Fee (currently at almost $3 million for a new drug)
• The Amyloidosis market was $3.6 billion in 2017, expected to reach $6 billion in 2025, according to Grand View Research

LOS ANGELES, Sept. 21, 2023 (GLOBE NEWSWIRE) — Nexcella, Inc. (“Nexcella” or the “Company”), a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug Designation (ODD) designation for NXC-201 for the treatment of a life-threatening blood disorder, Amyloid Light Chain (AL) Amyloidosis. NXC-201, a next generation CAR-T cell therapy, is currently being evaluated in a Phase 1b/2a clinical trial NEXICART-1 (NCT04720313).

The FDA’s Office of Orphan Products Development grants orphan designation status to drugs and biologics that are intended for the safe and effective treatment, diagnosis or prevention of rare diseases, or conditions that affect fewer than 200,000 people in the U.S. Orphan Drug Designation provides certain benefits, including financial incentives, to support clinical development and the potential for up to 7 years of market exclusivity in the U.S. upon regulatory approval.

“We are pleased to receive FDA’s orphan drug designation in AL amyloidosis for NXC-201, the only clinical-stage CAR-T cell therapy in development for AL amyloidosis,” said Ilya Rachman, MD PhD, Executive Chairman of Nexcella, adding, “We are thrilled to potentially expand therapeutic options for relapsed and refractory AL amyloidosis patients, where we have observed to date in our NXC-201 clinical trials a 100% hematologic response rate and demonstrated organ responses in patient hearts, livers and kidneys, for AL amyloidosis patients who received a median of 6 earlier treatments that previously failed to halt the disease.” Gabriel Morris, President of Nexcella, added, “We believe one-time treatment NXC-201 could offer AL amyloidosis patients a convenient therapeutic option.”

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 these cells cause a buildup of misfolded immunoglobulin proteins 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 the U.S. and Europe, and it is estimated that there are approximately 3,000 – 4,000 new cases annually in the U.S. The estimated annual global incidence ~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, CA 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 92% and 100% response rates in each indication, respectively, as of February 9, 2023, across 58 patients. 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  

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Contacts
Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com

Company Contact
irteam@nexcella.com