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Immix Biopharma Awarded European Union Orphan Drug Designation for NXC-201 in AL Amyloidosis

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The orphan drug designation (ODD) granted by the European Commission to NXC-201 for the treatment of AL Amyloidosis is a significant regulatory milestone for Immix Biopharma. This status is particularly important for therapies targeting rare diseases, as it provides a range of incentives designed to support drug development. These include market exclusivity for a decade post-authorization, which can protect the company's investment in the drug by reducing competition. Additionally, reduced regulatory fees and access to the centralized authorization procedure can lower the financial barriers to market entry and expedite the approval process.

In the context of AL Amyloidosis, a rare and life-threatening condition, the ODD could facilitate Immix Biopharma's efforts to bring a new treatment option to market. The company's focus on a 'Single Day CRS' protocol and the potential for NXC-201's application in other autoimmune diseases could further amplify its market impact. The increasing prevalence of relapsed/refractory AL Amyloidosis, growing at an annual rate of 12%, underscores the urgency and potential market for such treatments.

The grant of ODD to NXC-201 in the EU could be a precursor to increased investor confidence in Immix Biopharma's stock. Investors often view regulatory milestones as indicators of a company's progress and potential for future revenue growth. The designation suggests that NXC-201 has a unique treatment proposition for a growing patient population estimated to reach nearly 30,000 in 2023. The financial implications include not only the potential revenue from the drug's sales but also the cost savings associated with reduced regulatory fees and streamlined approval processes.

Furthermore, the company's ability to potentially expand the drug's application to other autoimmune diseases could lead to a diversification of its product pipeline, reducing the risk profile of the company's portfolio. This diversification strategy could attract a broader range of investors and positively influence the company's valuation in the stock market.

The economic implications of ODD for a drug like NXC-201 extend beyond Immix Biopharma's financials. From a healthcare economics perspective, the cost-effectiveness of new treatments for rare diseases is critical. The incentives provided by the ODD, such as market exclusivity, can help offset the high costs of research and development for drugs that serve a small patient population. This is particularly relevant in the EU, where healthcare systems are often publicly funded and cost containment is a priority.

Moreover, the potential for NXC-201 to serve as a one-time treatment option could lead to long-term savings for healthcare systems by reducing the need for ongoing treatment and management of relapsed/refractory AL Amyloidosis. The societal impact of introducing effective treatments for rare diseases also includes improved patient outcomes and quality of life, which are important considerations for healthcare policy makers.

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

U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing 12% per year according to Staron, et al Blood Cancer Journal, estimated to reach 29,712 patients in 2023

Los Angeles, Feb. 07, 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 AL Amyloidosis.

“European Union orphan drug designation for NXC-201 represents an important milestone in our global strategy,” said Ilya Rachman, MD PhD, Chief Executive Officer, Immix Biopharma. “We believe we are addressing an urgent need to establish a new potential one-time treatment option for relapsed/refractory AL Amyloidosis patients.” 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 to-date enables our observed complete responses (ongoing) in heavily pretreated relapsed/refractory AL Amyloidosis patients and potential expansion into other autoimmune diseases.”

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 autoimmune disease. Our lead cell therapy asset is next generation CAR-T NXC-201 for autoimmune disease, relapsed/refractory AL Amyloidosis, and relapsed/refractory multiple myeloma, currently being evaluated in our ongoing Phase 1b/2a NEXICART-1 (NCT04720313) clinical trial. 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. Our second program, a tissue specific therapeutic (TSTx) asset IMX-110, is being evaluated in Phase 1b/2a clinical trials as a monotherapy and IMMINENT-01 combination clinical trial with BeiGene’s anti-PD-1 antibody tislelizumab in relapsed/refractory solid tumors. IMX-110 has been awarded Orphan Drug Designation (ODD) and Rare Pediatric Disease Designation (RPDD) by the FDA. Learn more at www.immixbio.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, data observed to date, 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 and benefits 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 obtain the marketing approval for our product candidates by the FDA and the regulatory authorities in foreign jurisdictions in which we intend to market our product candidates, of which there can be no assurance; clinical testing is expensive, is difficult to design and implement, can take many years to complete and is uncertain as to outcome; success in early phases of pre-clinical and clinical trials does not ensure that later clinical trials will be successful, and interim results of a clinical trial do not necessarily predict final results; 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;; 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@immixbio.com


The ticker symbol for Immix Biopharma, Inc. is IMMX.

The European Orphan Drug Designation provides 10 years of market exclusivity in the EU, access to the centralized authorization procedure, and reduced fees for various regulatory processes.

The U.S. observed prevalence of relapsed/refractory AL Amyloidosis is growing at 12% per year, estimated to reach 29,712 patients in 2023.

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.
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