Associated tags: Systemic, Patient, Amyloidosis, Tissue, Incidence, Bone marrow, Cell, B cell, AL amyloidosis, Pharmaceutical industry, BCMA, LOS, AL, Society, MD, Hematopoietic stem cell transplantation, FDA, Multiple myeloma
Locations: EUROPE, AL
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Immunotherapy One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update.
Key Points:
- One new patient and additional follow-up data from an additional 8 patients (9 total) are included in this update.
- “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.
- Median follow-up was 7.3 months (range: 2.5 – 16.5 months) as of the September 20, 2023 data cutoff.
- Data highlights:
Best responder had a duration of response of 19.2 months as of the data cutoff of September 20, 2023, with response ongoing
For the 7 relapsed/refractory AL Amyloidosis patients with cardiac involvement:
4 patients with t(11;14) relapsed/refractory AL Amyloidosis:
The 20th IMS AL Amyloidosis presentation can be accessed on the ImmixBio corporate website at this link: https://immixbio.com/pipeline/#publications
20th International Myeloma Society Annual Meeting, Athens, Greece
Retrieved on:
Thursday, September 21, 2023
ODD,
MD,
Patient,
AL amyloidosis,
Diagnosis,
LOS,
Rare,
FDA,
Regulation of food and dietary supplements by the U.S. Food and Drug Administration,
Food,
Pharmaceutical industry,
Amyloidosis 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).
Key Points:
- FDA Orphan Drug Designation (“ODD”) qualifies one-time treatment NXC-201 for:
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.”
Retrieved on:
Thursday, September 21, 2023
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Amyloidosis LOS ANGELES, Sept. 21, 2023 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio” or the “Company”), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, 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).
Key Points:
- FDA Orphan Drug Designation (“ODD”) qualifies one-time treatment NXC-201 for:
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) -- Immix Biopharma, Inc. (Nasdaq: IMMX) (“ImmixBio” or the “Company”), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, 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, Immix Biopharma Chief Executive Officer, 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, Immix Biopharma Chief Financial Officer, added, “We believe one-time treatment NXC-201 could offer AL amyloidosis patients a convenient therapeutic option.”
Retrieved on:
Wednesday, August 16, 2023
LOS ANGELES, Aug. 16, 2023 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, today announced that additional NXC-201 clinical data for relapsed/refractory AL Amyloidosis has been selected for oral presentation at the 20th International Myeloma Society Annual Meeting to be held in Athens, Greece, September 27-30, 2023.
Key Points:
- LOS ANGELES, Aug. 16, 2023 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“ImmixBio”, “Company”, “We” or “Us”), a clinical-stage biopharmaceutical company pioneering personalized therapies for oncology and immunology, today announced that additional NXC-201 clinical data for relapsed/refractory AL Amyloidosis has been selected for oral presentation at the 20th International Myeloma Society Annual Meeting to be held in Athens, Greece, September 27-30, 2023.
- “We are pleased to present additional clinical data in the NEXICART-1 clinical study evaluating NXC-201 in patients with relapsed/refractory light chain AL amyloidosis at the upcoming International Myeloma Society Annual Meeting in September,” said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and NXC-201 principal study investigator.
- “We continue to make progress in this important indication, in the hope of providing patients with new treatment options.”
20th International Myeloma Society Annual Meeting, Athens, Greece
Retrieved on:
Wednesday, August 16, 2023
LOS ANGELES, Aug. 16, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, today announced that additional NXC-201 clinical data for relapsed/refractory AL Amyloidosis has been selected for oral presentation at the 20th International Myeloma Society Annual Meeting to be held in Athens, Greece, September 27-30, 2023.
Key Points:
- LOS ANGELES, Aug. 16, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), a clinical-stage biopharmaceutical company engaged in the discovery and development of novel cell therapies for oncology and other indications, today announced that additional NXC-201 clinical data for relapsed/refractory AL Amyloidosis has been selected for oral presentation at the 20th International Myeloma Society Annual Meeting to be held in Athens, Greece, September 27-30, 2023.
- “We are pleased to present additional clinical data in the NEXICART-1 clinical study evaluating NXC-201 in patients with relapsed/refractory light chain AL amyloidosis at the upcoming International Myeloma Society Annual Meeting in September,” said Polina Stepensky, M.D., Director of the Hadassah Medical Organization’s Department of Bone Marrow Transplantation and Immunotherapy for Adults and Children, and NXC-201 principal study investigator.
- “We continue to make progress in this important indication, in the hope of providing patients with new treatment options.”
20th International Myeloma Society Annual Meeting, Athens, Greece
LOS ANGELES, July 10, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the completion of its initial CAR-T NXC-201 engineering batch at its U.S. manufacturing site.
Key Points:
- LOS ANGELES, July 10, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the completion of its initial CAR-T NXC-201 engineering batch at its U.S. manufacturing site.
- This represents an important step forward in the planned U.S. expansion of the Company’s ongoing Phase 1b/2a NEXICART-1 (NCT04720313) study of its novel BCMA-targeted chimeric antigen receptor T (CAR-T) cell therapy NXC-201.
- This milestone is completed following the selection of a U.S. GMP manufacturer in February 2023 and commencement of U.S. engineering batches in May 2023.
- In the 5% of U.S medical centers that do offer CAR-T treatment, patients are hospitalized for a current market-standard 14 days,” said Gabriel Morris, President of Nexcella.
LOS ANGELES, July 10, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the completion of its initial CAR-T NXC-201 engineering batch at its U.S. manufacturing site.
Key Points:
- LOS ANGELES, July 10, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the completion of its initial CAR-T NXC-201 engineering batch at its U.S. manufacturing site.
- This milestone is completed following the selection of a U.S. GMP manufacturer in February 2023 and commencement of U.S. engineering batches in May 2023.
- Ph.D. Chief Executive Officer of Immix Biopharma.
- In the 5% of U.S medical centers that do offer CAR-T treatment, patients are hospitalized for a current market-standard 14 days,” said Gabriel Morris, Chief Financial Officer of Immix Biopharma.
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Hematology “AL amyloidosis is a rare and life-threatening disease in which patients have no treatment options despite the high fatality rate.
Key Points:
- “AL amyloidosis is a rare and life-threatening disease in which patients have no treatment options despite the high fatality rate.
- For two secondary endpoints, birtamimab demonstrated statistically significant improvements over placebo in a post hoc assessment of patients with Mayo Stage IV AL amyloidosis.
- In safety evaluations, the rates of treatment emergent adverse events (TEAEs) were balanced between treatment arms among patients with Mayo Stage IV AL amyloidosis (38 TEAEs in patients treated with birtamimab compared to 39 TEAEs in patients receiving placebo).
- Cardiac disorder was the most common class of fatal TEAEs, which is consistent with patients who have AL amyloidosis.
GMP,
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AL amyloidosis,
Multiple myeloma LOS ANGELES, May 26, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the commencement of CAR-T NXC-201 Engineering Batches at its U.S. Manufacturing Site. These U.S. engineering batches will support the planned U.S. expansion of the Company’s ongoing Phase 1b/2a NEXICART-1 (NCT04720313) study of its novel BCMA-targeted chimeric antigen receptor T (car-T) cell therapy NXC-201. This manufacturing milestone represents a crucial next step in transferring the Company’s existing CAR-T GMP manufacturing process to the United States following the selection of a U.S. GMP manufacturer in February 2023.
Key Points:
- Commencement of U.S. engineering batches follows selection of U.S. GMP manufacturer in February 2023
LOS ANGELES, May 26, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the commencement of CAR-T NXC-201 Engineering Batches at its U.S. Manufacturing Site.
- These U.S. engineering batches will support the planned U.S. expansion of the Company’s ongoing Phase 1b/2a NEXICART-1 (NCT04720313) study of its novel BCMA-targeted chimeric antigen receptor T (car-T) cell therapy NXC-201.
- This manufacturing milestone represents a crucial next step in transferring the Company’s existing CAR-T GMP manufacturing process to the United States following the selection of a U.S. GMP manufacturer in February 2023.
- “NXC-201 is the first CAR-T being developed in AL Amyloidosis, and in Multiple Myeloma, patients face significant obstacles when seeking BCMA-targeted CAR-T treatments like NXC-201.
GMP,
Hospital,
Engineering,
Doctor of Philosophy,
AL,
Patient,
Amyloidosis,
LOS,
BCMA,
Pharmaceutical industry,
Vaccine,
AL amyloidosis,
Multiple myeloma LOS ANGELES, May 26, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the commencement of CAR-T NXC-201 Engineering Batches at its U.S. Manufacturing Site. These U.S. engineering batches will support the planned U.S. expansion of the Company’s ongoing Phase 1b/2a NEXICART-1 (NCT04720313) study of its novel BCMA-targeted chimeric antigen receptor T (car-T) cell therapy NXC-201. This manufacturing milestone represents a crucial next step in transferring the Company’s existing CAR-T GMP manufacturing process to the United States following the selection of a U.S. GMP manufacturer in February 2023.
Key Points:
- Commencement of U.S. engineering batches follows selection of U.S. GMP manufacturer in February 2023
LOS ANGELES, May 26, 2023 (GLOBE NEWSWIRE) -- Nexcella, Inc., a subsidiary of Immix Biopharma, Inc. (“Nexcella”, “Company”, “We” or “Us”), today announced the commencement of CAR-T NXC-201 Engineering Batches at its U.S. Manufacturing Site.
- These U.S. engineering batches will support the planned U.S. expansion of the Company’s ongoing Phase 1b/2a NEXICART-1 (NCT04720313) study of its novel BCMA-targeted chimeric antigen receptor T (car-T) cell therapy NXC-201.
- This manufacturing milestone represents a crucial next step in transferring the Company’s existing CAR-T GMP manufacturing process to the United States following the selection of a U.S. GMP manufacturer in February 2023.
- “NXC-201 is the first CAR-T being developed in AL Amyloidosis, and in Multiple Myeloma, patients face significant obstacles when seeking BCMA-targeted CAR-T treatments like NXC-201.
