Kezar Life Sciences Announces Orphan Drug Designations for KZR-616 for the Treatment of Polymyositis and Dermatomyositis
Kezar Life Sciences, a clinical-stage biotechnology firm, has announced that the FDA has granted Orphan Drug Designation for KZR-616, targeting polymyositis (PM) and dermatomyositis (DM). PM and DM are rare autoimmune diseases affecting up to 51,000 and 71,000 individuals in the U.S., respectively. KZR-616 is a first-in-class selective immunoproteasome inhibitor aimed at addressing the underlying causes of these diseases. The designation may offer significant benefits, including marketing exclusivity and tax credits. A Phase 2 trial for KZR-616 is currently enrolling patients.
- KZR-616 received Orphan Drug Designation from the FDA for PM and DM, signaling potential market advantages.
- The drug has shown a favorable safety and tolerability profile in earlier trials, supporting its development in chronic autoimmune diseases.
- There are currently no approved treatments for polymyositis, indicating a high level of unmet medical need.
- The forward-looking statements in the PR highlight uncertainties related to clinical trials and FDA approval timelines.
SAN FRANCISCO--(BUSINESS WIRE)--Kezar Life Sciences, Inc. (Nasdaq: KZR), a clinical-stage biotechnology company discovering and developing breakthrough treatments for immune-mediated and oncologic disorders, today announced that the U.S. Food and Drug Administration has granted Orphan Drug Designations (ODD) for KZR-616 for the treatment of polymyositis (PM) and dermatomyositis (DM). Both orphan diseases are autoimmune inflammatory myopathies that are chronic and debilitating diseases characterized by marked morbidity and mortality. The estimated prevalence of PM and DM in the United States is up to 51,000 and 71,000, respectively.
KZR-616 is a first-in-class selective immunoproteasome inhibitor with the potential to impact multiple drivers of immune-mediated diseases and inflammation. PRESIDIO, a Phase 2 placebo-controlled cross-over clinical trial to evaluate KZR-616 for the treatment of PM and DM, is currently enrolling subjects.
“We are very pleased to have received orphan drug designations for both polymyositis and dermatomyositis. This recognition spotlights the significant unmet need for patients living with these autoimmune myopathies. KZR-616 has potential to truly modify the underlying pathophysiology of these two diseases,” said Noreen R. Henig, MD, Kezar’s Chief Medical Officer. “We will share more detail on the preclinical scientific rationale for the use of selective immunoproteasome inhibition to treat these rare and debilitating diseases during the 2020 American College of Rheumatology Conference next month.”
Orphan Drug Designation is intended to advance drug development for rare diseases. The FDA defines rare diseases as those affecting fewer than 200,000 people in the United States. Orphan Drug Designation can provide certain benefits and incentives for KZR-616, including a period of marketing exclusivity for the first marketing application, if regulatory approval is received for the designated indication, potential tax credits for qualified clinical testing and waiver of certain administrative fees.
About KZR-616
KZR-616 is a novel, first-in-class, selective immunoproteasome inhibitor with broad therapeutic potential across multiple autoimmune diseases. Preclinical research demonstrates that selective immunoproteasome inhibition results in a broad anti-inflammatory response in animal models of several autoimmune diseases, while avoiding immunosuppression. Data generated from Phase 1a and 1b trials provide evidence that KZR-616 exhibits a favorable safety and tolerability profile for development in chronic autoimmune diseases. Phase 2 trials are underway in severe autoimmune diseases.
About Polymyositis and Dermatomyositis
Polymyositis (PM) and Dermatomyositis (DM) are two of the five types of autoimmune myositis diseases. Both are chronic, debilitating, inflammatory autoimmune myopathies that are distinguished by inflammation of the muscles as well as the skin (in DM). Up to ~120,000 people in the United States are living with these severe and progressive inflammatory myopathies that are characterized by marked morbidity and associated mortality. While debilitating muscle weakness is the hallmark of these myopathies, including compromised muscles of respiration, other internal organ system dysfunctions can be equally disabling. The aim of treatment for these diseases is to suppress inflammation, increase muscle strength and prevent long-term damage to muscles and extramuscular organs; however, treatment options are limited for DM, and there are currently no approved treatments for PM.
About Kezar Life Sciences
Based in South San Francisco, Kezar Life Sciences is combining courage, conviction and cutting-edge science to develop breakthrough treatments for immune-mediated and oncologic disorders. The company is pioneering first-in-class, small-molecule therapies that harness master regulators of cellular function and inhibit multiple drivers of disease via a single target. KZR-616, a first-in-class selective immunoproteasome inhibitor, is being evaluated in severe and underserved autoimmune diseases. Additionally, KZR-261, the first clinical candidate for the treatment of cancer from the company's protein secretion program targeting the Sec61 translocon, is undergoing IND-enabling activities.
Cautionary Note on Forward-looking Statements
This press release contains forward-looking statements within the meaning of the Private Securities Litigation Reform Act of 1995. Words such as “may,” “will,” “should,” “expect,” “believe” and similar expressions (as well as other words or expressions referencing future events, conditions or circumstances) are intended to identify forward-looking statements. These forward-looking statements are based on Kezar’s expectations and assumptions as of the date of this press release. Each of these forward-looking statements involves risks and uncertainties that could cause Kezar’s clinical development programs, future results or performance to differ materially from those expressed or implied by the forward-looking statements. Forward-looking statements contained in this press release include, but are not limited to estimates of the prevalence of polymyositis and dermatomyositis, the potential of KZR-616 to modify the underlying pathophysiology of polymyositis and dermatomyositis, statements about the likelihood that pre-clinical data will support the scientific rationale to develop KZR-616 in polymyositis and dermatomyositis, the design, progress, timing, scope and results of clinical trials, the anticipated timing of disclosure of results of clinical trials and the likelihood and timing of obtaining regulatory approval of KZR-616. Orphan Drug Designation does not provide any assurance of regulatory approval or expedite regulatory review. Many factors may cause differences between current expectations and actual results, including the impacts of the COVID-19 pandemic on the company’s business, clinical trials and financial position, unexpected safety or efficacy data observed during preclinical or clinical studies, clinical trial site activation or enrollment rates that are lower than expected, changes in expected or existing competition, changes in the regulatory environment, the uncertainties and timing of the regulatory approval process, and unexpected litigation or other disputes. Other factors that may cause actual results to differ from those expressed or implied in the forward-looking statements in this press release are discussed in Kezar’s filings with the U.S. Securities and Exchange Commission, including the “Risk Factors” contained therein. Except as required by law, Kezar assumes no obligation to update any forward-looking statements contained herein to reflect any change in expectations, even as new information becomes available.
