Citius Pharmaceuticals, Inc. Hosts Pre-BLA Meeting with FDA for I/ONTAK (denileukin diftitox) for the Treatment of Cutaneous T-Cell Lymphoma


Submission of Biological License Application (BLA) planned for the second half of 2022

CRANFORD, NJ , July 12, 2022 /PRNewswire/ — Citius Pharmaceuticals, Inc. (“Citius” or the “Company”) (Nasdaq: CTXR), a late-stage biopharmaceutical company developing and commercializing first-class critical care products, today announced Today that the Company held a pre-BLA Type B meeting with the U.S. Food and Drug Administration (FDA) to discuss I/ONTAK (denileukin diftitox), an IL-2-diphtheria toxin fusion protein designed for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma (CTL). I/ONTAK is a purified and more bioactive formulation of ONTAK previously approved by the FDA®. The pre-BLA meeting was held with representatives from the FDA’s Center for Drug Evaluation and Research (CDER).

The purpose of the pre-BLA meeting with the FDA was to discuss the content and acceptability of the company’s planned BLA for I/ONTAK. The briefing document provided to the FDA included a review of clinical and non-clinical studies and minutes of previous meetings with the FDA.

“We appreciate the continued guidance from the FDA on the path to developing a more purified formulation of ONTAK. Based on feedback from the FDA pre-BLA meeting, we intend to move forward. forward with our planned BLA submission for I/ONTAK in the second half of 2022. We look forward to our continued engagement with the FDA as we advance this program to provide CTCL patients with a potential new treatment option,” said declared Leonard MazurChairman and CEO of Citius.

I/ONTAK is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent binds specifically to IL-2 receptors on the cell surface, causing inhibition of protein synthesis by diphtheria toxin fragments that have entered the cells. I/ONTAK, a purified version of the denileukin diftitox, is a reformulation of the previously FDA-approved oncology treatment ONTAK. ONTAK was marketed in the United States from 1999 until 2014, when it was voluntarily withdrawn from the market. Manufacturing improvements have resulted in a new formulation, which retains the same amino acid sequence but features improved purity and bioactivity. The new formulation received regulatory approval in Japan for the treatment of CTCL and PTCL. In 2011 and 2013, the FDA granted Orphan Drug Designation (ODD) to I/ONTAK for the treatment of PTCL and CTCL, respectively, making it potentially eligible for seven years of market exclusivity after approval for each indication.

About cutaneous T-cell lymphoma
Cutaneous T-cell lymphoma is a type of cutaneous non-Hodgkin’s lymphoma (NHL) that comes in various forms and is the most common type of cutaneous lymphoma. In CTCL, T cells, a type of lymphocyte that plays a role in the immune system, become cancerous and develop into skin lesions, leading to a decrease in the quality of life of patients with this disease due to severe pain and pruritus. Mycosis fungoides (MF) and Sézary syndrome (SS) represent the majority of cases of CTCL. Depending on the type of CTCL, the disease can progress slowly and can take from several years to more than ten years to potentially reach the tumor stage. However, once the disease reaches this stage, the cancer is highly malignant and can spread to lymph nodes and internal organs, resulting in a poor prognosis. Given the duration of the disease, patients typically use multiple systemic agents to control disease progression. CTCL affects men twice as often as women and is usually first diagnosed in patients between the ages of 50 and 60. Other than allogeneic stem cell transplantation, for which only a small fraction of patients are eligible, there is currently no cure for advanced CTCL. About 3,000 new cases are reported in United States each year, with around 30,000 to 40,000 people living with the disease.

About Citius Pharmaceuticals, Inc.
Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-class critical care products, with a focus on oncology, anti-infectives in complementary cancer care, unique prescription products and stem cell therapies. The Company has two late-stage product candidates, Mino-Lok®an antibiotic lock solution for the treatment of patients with catheter-related bloodstream infections (CRBSI), which is currently enrolling patients in a pivotal Phase 3 superiority trial, and I/ONTAK (E7777), a novel IL immunotherapy -2R for a first indication in cutaneous T-cell lymphoma (CTCL), for which a BLA submission is being prepared for the second half of 2022. Mino-Lok® has obtained Fast Track designation from the United States Food and Drug Administration (FDA). I/ONTAK has received orphan drug designation from the FDA for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). For more information, please visit

Safe Harbor
This press release may contain “forward-looking statements” within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. These statements are made on the basis of our expectations and beliefs regarding the future events impacting Citius. You can identify these statements by the fact that they use words such as “believe”, “anticipate”, “estimate”, “expect”, “plan”, “should” and “may” and others words and terms of similar meaning or the use of future dates. Forward-looking statements are based on management’s current expectations and are subject to risks and uncertainties that could adversely affect our business, results of operations, financial condition and stock price. Factors that could cause actual results to differ materially from those currently anticipated are: we may encounter unexpected delays or obstacles in our efforts to submit a BLA for I/ONTAK and we may not be able to submit the BLA on schedule or at all; the FDA may find inadequacies and deficiencies in our BLA for I/ONTAK, including in the data we submit, and may decide not to accept the BLA filing; our ability to successfully initiate and complete clinical and non-clinical trials and the results of such trials for our product candidates; the estimated markets for our product candidates and their acceptance by any market; our ability to market our products if approved by the FDA; our reliance on third-party vendors; the ability of our product candidates to impact the quality of life of our target patient populations; risks relating to the results of research and development activities, including those of existing and new pipeline assets; uncertainties related to preclinical and clinical trials; our need for substantial additional funds; the early stage of products under development; market and other conditions; our ability to attract, integrate and retain key personnel; risks related to our growth strategy; patents and intellectual property; our ability to obtain, execute and maintain financing and strategic agreements and relationships; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; our ability to obtain commercial-scale cGMP supply; government regulation; competition; and other risks described in our filings with the SEC. These risks have been and could be further impacted by Covid-19. Accordingly, these forward-looking statements are not guarantees of future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks relating to our business are described in detail in our filings with the Securities and Exchange Commission (“SEC”) which are available on the SEC’s website at www.sec.govincluding in our Annual Report on Form 10-K for the fiscal year ended September 30, 2021filed with the SEC on December 15, 2021 and updated by our subsequent filings with the SEC. These forward-looking statements speak only as of the date hereof, and we expressly disclaim any obligation or undertaking to release any updates or revisions to any forward-looking statements contained herein to reflect any change in our expectations or any change in the events, conditions or circumstances on which such statement is based, except as required by law.

Investor Relations for Citius Pharmaceuticals:
Ilanit Allen
Vice President, Investor Relations and Corporate Communications
Such. : 908-967-6677 ext. 113
E : [email protected]

SOURCECitius Pharmaceuticals, Inc.


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