Full Press Release Details
Pharmaceuticals, Inc. Submits Biologics License Application to the U.S. Food and Drug Administration for Denileukin Diftitox for the
Treatment of Patients with Persistent or Recurrent Cutaneous T-Cell Lymphoma
N.J., September 28, 2022 -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage
biopharmaceutical company developing and commercializing first-in-class critical care products, today announced that the Company
has submitted a Biologics License Application (BLA) to the U.S. Food and Drug Administration (FDA) for denileukin diftitox (I/ONTAK),
an engineered IL-2-diphtheria toxin fusion protein for the treatment of patients with persistent or recurrent cutaneous T-cell lymphoma
(CTCL). I/ONTAK is a purified and more bioactive formulation of previously FDA-approved ONTAK . The BLA is supported by
a pivotal Phase 3 study (NCT01871727). Results of the study were consistent with the prior FDA-approved formulation.
treatment of advanced cutaneous T-cell lymphoma remains a complex and challenging unmet medical need. Each year, thousands of patients
are diagnosed with CTCL, a debilitating orphan disease with no single standard of care. Patients are often treated with multiple alternate
therapies. Citius is proud to advance the only potential CTCL therapeutic with a mechanism of action that delivers a cytotoxic protein
by binding to the IL-2 receptors found in malignant T-cells and immunosuppressive T-regulatory cells. We look forward to continuing to
engage with the FDA as they review our BLA and bringing this treatment option to patients, if approved," stated Leonard Mazur,
Chairman and CEO of Citius. "The BLA filing for denileukin diftitox marks the first of our pipeline candidates to be submitted
is a recombinant fusion protein that combines the interleukin-2 (IL-2) receptor binding domain with diphtheria toxin fragments. The agent
specifically binds to IL-2 receptors on the cell surface, causing diphtheria toxin fragments that have entered cells to inhibit protein
synthesis. I/ONTAK, a purified version of denileukin diftitox, is a reformulation of previously FDA-approved oncology treatment ONTAK.
ONTAK was marketed in the U.S. from 1999 to 2014, when it was voluntarily withdrawn from the market. Manufacturing improvements resulted
in a new formulation, which maintains the same amino acid sequence but features improved purity and bioactivity. The new formulation
received regulatory approval in Japan in 2021 for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). In 2011 and 2013, the
FDA granted orphan drug designation (ODD) to I/ONTAK for the treatment of PTCL and CTCL, respectively.
Cutaneous T-cell Lymphoma
T-cell lymphoma is a type of cutaneous non-Hodgkin lymphoma (NHL) that comes in a variety of 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
S zary Syndrome (SS) comprise the majority of CTCL cases. Depending on the type of CTCL, the disease may progress slowly and can
take anywhere from several years to upwards of ten to potentially reach tumor stage. However, once the disease reaches this stage, the
cancer is highly malignant and can spread to the lymph nodes and internal organs, resulting in a poor prognosis. Given the duration of
the disease, patients typically cycle through multiple systemic agents to control disease progression. CTCL affects men twice as often
as women and is typically first diagnosed in patients between the ages of 50 and 60 years of age. Other than allogeneic stem cell transplantation,
for which only a small fraction of patients qualify, there is currently no curative therapy for advanced CTCL. Approximately 3,000 new
cases are reported in the United States every year, with an estimated 30,000 - 40,000 individuals living with the disease.
Citius Pharmaceuticals, Inc.
is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products,
with a focus on oncology, anti-infectives in adjunct cancer care, unique prescription products, and stem cell therapies. The Company's
diversified pipeline includes two late-stage product candidates, Mino-Lok , an antibiotic lock solution for the treatment
of patients with catheter-related bloodstream infections (CRBSIs), which is currently enrolling patients in a Phase 3 Pivotal superiority
trial, and I/ONTAK (E7777), a novel IL-2R immunotherapy for an initial indication in cutaneous T-cell lymphoma (CTCL), for which a BLA
has been submitted. Mino-Lok was granted Fast Track designation by the U.S. Food and Drug Administration (FDA).
I/ONTAK has received orphan drug designation by the FDA for the treatment of CTCL and peripheral T-cell lymphoma (PTCL). In the first
half of 2022, Citius initiated a Phase 2b trial for Halo-Lido, a topical formulation for the relief of hemorrhoids. The Company anticipates
completing enrollment in the Halo-Lido trial by the end of 2022. For more information, please visit www.citiuspharma.com.
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. Such statements are made based on our expectations and beliefs concerning 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 other words and terms of similar
meaning or use of future dates. Forward-looking statements are based on management's current expectations and are subject to risks and
uncertainties that could negatively affect our business, operating results, financial condition and stock price. Factors that could cause
actual results to differ materially from those currently anticipated are: 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 for filing; our ability to successfully undertake and
complete clinical and non-clinical trials and the results from those trials for our product candidates; the estimated markets for our
product candidates and the acceptance thereof by any market; our ability to commercialize our products if approved by the FDA; our dependence
on third-party suppliers; 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 from existing and new pipeline assets; uncertainties
relating to preclinical and clinical testing; 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; patent
and intellectual property matters; our ability to obtain, perform under 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 procure cGMP commercial-scale supply; government regulation; competition; as well as other risks described in our SEC filings. These
risks have been and may be further impacted by Covid-19. Accordingly, these forward-looking statements do not constitute guarantees of
future performance, and you are cautioned not to place undue reliance on these forward-looking statements. Risks regarding our business
are described in detail in our Securities and Exchange Commission ("SEC") filings which are available on the SEC's
website at www.sec.gov, including in our Annual Report on Form 10-K for the year ended September 30, 2021, filed 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 publicly any updates or revisions to any forward-looking statements contained
herein to reflect any change in our expectations or any changes in events, conditions or circumstances on which any such statement is
based, except as required by law.