Immix Biopharma Awarded European Union Orphan Drug Designation for NXC-201 in Multiple Myeloma
Immix Biopharma, Inc. has been granted European Orphan Drug Designation for NXC-201 in the treatment of multiple myeloma, providing 10 years of market exclusivity, access to centralized authorization procedure, and reduced fees for various applications. The Company aims to address unmet medical needs in frail patients with relapsed/refractory multiple myeloma, with potential benefits in terms of tolerability and clinical impact. The designation signifies recognition of the therapy's potential for this patient population, opening up opportunities for further development.
Granting of European Orphan Drug Designation for NXC-201 in multiple myeloma, showcasing potential clinical impact and addressing unmet medical needs in frail patients.
Potential for NXC-201 to provide an attractive treatment option for frail relapsed/refractory multiple myeloma patients, along with expansion into other autoimmune diseases.
Recognition of NXC-201's favorable tolerability profile and 'Single Day CRS' across a robust clinical dataset, indicating promising therapeutic potential.
- None.
European Orphan Drug Designation (“ODD”) qualifies NXC-201 for:
- 10 years of market exclusivity once authorized in the EU
- Access to the EU centralized authorization procedure
- Reduced fees for: EU protocol assistance, marketing authorization applications, inspections before authorization, applications for changes to marketing authorizations made after approval, and reduced annual fees
LOS ANGELES, April 29, 2024 (GLOBE NEWSWIRE) -- Immix Biopharma, Inc. (“Immix Biopharma”, “Company”, “We” or “Us”, Nasdaq:IMMX), a clinical-stage biopharmaceutical company trailblazing cell therapies in autoimmune disease, today announced that the European Commission (EC) has granted orphan drug designation to NXC-201 for the treatment of multiple myeloma.
“Frail patients, heavily represented in our NEXICART-1 clinical trial, remain an area of unmet medical need and are a significant portion of the relapsed/refractory multiple myeloma population,” said Ilya Rachman, MD PhD, Chief Executive Officer, Immix Biopharma. “We believe EU orphan drug designation for NXC-201 affirms the potential clinical impact of NXC-201 in this sizable population.”
Gabriel Morris, Chief Financial Officer, Immix Biopharma added, “We believe NXC-201’s observed favorable tolerability profile and ‘Single Day CRS’ across a robust clinical dataset could enable an attractive option for frail relapsed/refractory multiple myeloma patients in addition to our lead indication, relapsed/refractory AL Amyloidosis, and our active NXC-201 expansion into other autoimmune diseases.”
According to Davis et al., 2023 Transplantation and Cellular Therapy, commercial CAR-Ts produced 6.9 months median progression free survival in frail relapsed/refractory multiple myeloma patients in a real-world setting.
Orphan drug designation in the European Union (EU) is granted by the EC based on a positive opinion issued by the European Medicines Agency (EMA) Committee for Orphan Medicinal Products. To qualify for orphan drug designation, a candidate therapy must be intended for the treatment, prevention or diagnosis of a life-threatening or chronically debilitating disease that occurs in not more than five in 10,000 people in the EU. The designation provides regulatory, financial and commercial incentives to develop therapies for rare diseases where there are either no satisfactory treatment options or significant benefit to those affected by the disease.
About Immix Biopharma, Inc.
Immix Biopharma, Inc. (ImmixBio) (Nasdaq: IMMX) is a clinical-stage biopharmaceutical company trailblazing cell therapies in AL Amyloidosis and other autoimmune diseases. Our lead cell therapy is FDA IND cleared next generation CAR-T NXC-201, currently being evaluated in our ongoing Phase 1b/2 NEXICART-1 (NCT04720313) clinical trial, initiated in February 2021. NXC-201 has the potential to be the world’s first “Single-Day CRS” CAR-T (CRS median onset day 1, median duration 1 day), enabling the potential for a faster return home for patients and supporting ongoing expansion into autoimmune indications. NXC-201 has been awarded Orphan Drug Designation (ODD) by the FDA in both AL Amyloidosis and multiple myeloma, and ODD by the European Commission (EMA) in AL Amyloidosis. Learn more at www.immixbio.com and www.BeProactiveInAL.com.
Forward Looking Statements
This press release contains forward-looking statements regarding Immix Biopharma, Inc., including the potential benefits of our initial product candidates, CAR-T NXC-201 and IMX-110. Forward-looking statements include, but are not limited to, our plans, objectives, expectations and intentions and other statements that contain words such as “expects,” “contemplates,” “anticipates,” “plans,” “intends,” “believes”, “estimates”, “potential” and variations of such words or similar expressions that convey the uncertainty of future events or outcomes, or that do not relate to historical matters. Those forward-looking statements involve known and unknown risks, uncertainties and other factors that could cause actual results to differ materially. Among those factors are: (i) the risk that the further data from the ongoing Phase 1b/2a clinical trials for CAR-T NXC-201 and IMX-110 will not be favorably consistent with the initial data initial data readouts, (ii) the risk that the Company may not be able to advance to registration-enabling studies for the CAR-T NXC-201 and IMX-110 product candidates, (iii) success in early phases of pre-clinical and clinicals trials do not ensure later clinical trials will be successful; (iv) no drug product developed by the Company has received FDA pre-market approval or otherwise been incorporated into a commercial drug product, (v) the risk that the Company may not be able to obtain additional working capital with which to continue the clinical trials for CAR-T NXC-201 or IMX-110, or advance to the initiation of registration-enabling studies, for such product candidates as and when needed and (vii) those other risks disclosed in the section “Risk Factors” included in the Company’s Annual Report on Form 10-K filed with the SEC on March 29, 2024 and other periodic reports subsequently filed with the Securities and Exchange Commission. Immix Biopharma cautions readers not to place undue reliance on any forward-looking statements. Immix Biopharma does not undertake, and specifically disclaims, any obligation to update or revise such statements to reflect new circumstances or unanticipated events as they occur, except as required by law.
Contacts:
Mike Moyer
LifeSci Advisors
mmoyer@lifesciadvisors.com
Company Contact
irteam@immixbio.com
FAQ
What is the significance of the European Orphan Drug Designation granted to Immix Biopharma for NXC-201?
The designation recognizes NXC-201's potential in treating multiple myeloma, offering market exclusivity, access to centralized authorization, and reduced fees for various applications.
Who is the Chief Executive Officer of Immix Biopharma and what did they say about the orphan drug designation?
The Chief Executive Officer of Immix Biopharma is Ilya Rachman, MD PhD. He mentioned that the designation affirms the potential clinical impact of NXC-201 in frail patients with relapsed/refractory multiple myeloma.
What are the benefits of orphan drug designation in the European Union?
The designation provides regulatory, financial, and commercial incentives to develop therapies for rare diseases by offering reduced fees and market exclusivity.
How does NXC-201 compare to commercial CAR-Ts in treating frail relapsed/refractory multiple myeloma patients?
According to a study, commercial CAR-Ts produced 6.9 months median progression free survival in frail patients, highlighting the potential significance of NXC-201 in this patient population.
