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
Forward Looking Statements
This press release contains “forward-looking statements.” Forward-looking statements reflect our current view about future events. When used in this press release, the words “anticipate,” “believe,” “estimate,” “expect,” “future,” “intend,” “plan,” or the negative of these terms and similar expressions, as they relate to us or our management, identify forward-looking statements. Such statements, include, but are not limited to, statements contained in this press release relating to our business strategy, our future operating results, continuing development of our product candidates, including development timelines, timing of FDA submissions and expected endpoints, long-term visions and strategies, evaluations and judgements and beliefs regarding potential efficacy and safety of our product candidates, future clinical development of our product candidates, including any implication that results or observations in initial data or earlier clinical trials will be representative of results or observations in later data or clinical trials, the expected timing of such results and the potential market size for our product candidates. Forward-looking statements are based on our current expectations and assumptions regarding our business, the economy and other future conditions. Because forward-looking statements relate to the future, they are subject to inherent uncertainties, risks and changes in circumstances that are difficult to predict. Our actual results may differ materially from those contemplated by the forward-looking statements. They are neither statements of historical fact nor guarantees of assurance of future performance. We caution you, therefore, against relying on any of these forward-looking statements. Important factors that could cause actual results to differ materially from those in the forward-looking statements include, without limitation, our ability to raise capital to fund continuing operations; our ability to protect our intellectual property rights; the impact of any infringement actions or other litigation brought against us; competition from other providers and products; our ability to develop and commercialize products and services; changes in government regulation; our ability to complete capital raising transactions; that our product candidates may not realize the anticipated responses discussed in this release or that their development may suffer delays that materially and adversely affects future commercial viability; that the market for our product candidates may not grow at the rates anticipated or at all; and other factors relating to our industry, our operations and results of operations. Actual results may differ significantly from those anticipated, believed, estimated, expected, intended or planned, including: the uncertainties related to market conditions and other factors described more fully in the section entitled ‘Risk Factors’ in Immix Biopharma’s Annual Report on Form 10-K for the year ended December 31, 2022, and other periodic reports subsequently filed with the Securities and Exchange Commission. Any forward-looking statements contained in this press release speak only as of the date hereof, and we specifically disclaim any obligation to update any forward-looking statement, whether as a result of new information, future events or otherwise. Factors or events that could cause our actual results to differ may emerge from time to time, and it is not possible for us to predict all of them. We cannot guarantee future results, levels of activity, performance or achievements.
Contacts
Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com
Company Contact
irteam@nexcella.com
