Denileukin Diftitox

Trial achieves statistically significant primary endpoint (p=0.0006) Secondary endpoint demonstrates statistically significant overall success of Mino-Lok therapy with a greater percentage of patients retaining their catheters (p=0.0025) CRANFORD, N.J., May 21, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products, today announced positive topline results of its pivotal Phase 3 clinical trial of Mino-Lok®, a novel antibiotic lock solution designed to salvage catheters in patients with central line-associated infections (CLABSI) or catheter-related bloodstream infections (CRBSI). The study met its primary endpoint with a statistically significant improvement in the time to failure event in patients receiving Mino-Lok compared to Control arm patients receiving clinician-directed anti-infective lock solution. The data demonstrate that Mino-Lok is well-tolerated. "We are extremely pleased by the strong results of the trial, which demonstrate the safety and efficacy of Mino-Lok in preserving indwelling catheters in patients with bloodstream infections. The data indicate, with a high degree of statistical significance, that Mino-Lok outperforms anti-infective lock solutions utilized in patients with CLABSI or CRBSI in the study's control arm. We believe Mino-Lok could potentially set a new standard of care (SOC) as an adjunct therapy in the treatment of patients with bloodstream infections. This therapy offers a non-invasive treatment option compared to catheter removal and replacement," stated Leonard Mazur, Chairman and Chief Executive Officer of Citius. "We look forward to engaging with the US Food and Drug Administration (FDA) to determine the optimal path forward for Mino-Lok. Our focus remains on improving outcomes for patients and offering a much-needed alternative to the current practice of catheter removal and replacement," added Mazur. Mino-Lok Phase 3 Trial Design MDA 2013-0039 (NCT02901717) is a completed Phase 3 multicenter, randomized, open-label, active-controlled assessor-blinded trial conducted to evaluate the safety and efficacy of Mino-Lok as an adjunctive therapy to systemic anti-infectives for the treatment of catheter-related bloodstream infections (CRBSI) and central line-associated infections (CLABSI). A total of 241 patients in the U.S. and India, with an indwelling central venous catheter before the onset of a bloodstream infection, were randomized in a 1:1 ratio to receive either Mino-Lok or SOC site-specific anti-infective lock solution plus SOC systemic antibiotics. Enrollment was not limited to patients with specific types of catheters. Patients in the Mino-Lok arm received one Mino-Lok dose daily with a dwell time of two to four hours for a total of seven doses over a period of up to 15 days. The primary endpoint for this study is the time to a catheter failure event between randomization and test of cure (TOC) at six weeks, measured in days following randomization. Additional secondary outcome measures include overall success, microbiological eradication, and clinical cure, among others. For subjects in the Control arm, the investigator determined the antibiotic used in the lock, dose, dwell time, and number of days of administration based on institutional standards or Infectious Diseases Society of America (IDSA) guidelines. TOPLINE RESULTS Efficacy The primary endpoint in the study was time to catheter failure between randomization and six-weeks following the first dose of Mino-Lok or SOC lock solution. Catheter failure was defined as the inability to administer study lock solution, catheter removal for any infection-related reason (including worsening clinical signs and symptoms or persistence or recurrence of baseline pathogen, or new infection), and all-cause mortality. Results of blinded-assessor clinical outcome analyses in all randomized patients demonstrated that Mino-Lok achieved its primary endpoint: Time to catheter failure events in patients receiving Mino-Lok therapy occurred substantially later than in patients in the Control arm (p value = 0.0006) Median time-to-failure (MTF) of Control arm: 33 days; 95% confidence interval (CI) (14 days – 44 days; n = 122) Median time-to-failure (MTF) of Mino-Lok arm: MTF exceeded the time the patients were on trial and therefore not estimable (NE); 95% CI (50 days - NE; n = 119) The hazard ratio of this study was 0.53 The critical secondary endpoints included the proportion of patients at six weeks with overall treatment success including no catheter failure, clinical cure (absence of baseline clinical signs and symptoms of infection or improvement of clinical signs and symptoms such that no additional therapy was necessary), or microbiological eradication (absence of the baseline pathogen). Results in all randomized patients who received at least one dose of study lock solution demonstrate overall treatment success in 57.1% of patients in the Mino-Lok arm and 37.7% of patients in the Control arm (p=0.0025). Safety Mino-Lok is designed for intracatheter instillation and is not for intravenous injection or systemic circulation. No serious adverse events were drug-related Serious adverse events occurred in 45.1% of patients in the Mino-Lok arm and 46.1% of patients in the Control arm About Mino-Lok Mino-Lok (MLT) is a novel antibiotic lock solution that combines minocycline, ethanol with edetate disodium designed to treat patients with catheter-related blood stream infections. Citius licensed Mino-Lok from an affiliate of The University of Texas MD Anderson Cancer Center. Mino-Lok is designed to offer an alternative to removing and replacing a central venous catheter (CVC), which may lead to a reduction in serious adverse events and cost savings to the healthcare system. If approved, Mino-Lok would be the first and only FDA-approved treatment that salvages central venous catheters that cause central line-related blood stream infections. About Citius Pharmaceuticals, Inc. Citius is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products. The Company's diversified pipeline includes two late-stage product candidates: Mino-Lok® and LYMPHIRTM. In May 2024, Citius announced positive topline results of Mino-Lok, its antibiotic lock solution to salvage catheters in patients with bloodstream infections. The Biologics License Application for LYMPHIR, a novel IL-2R immunotherapy for an initial indication in cutaneous T-cell lymphoma, is currently under review by the FDA with August 13, 2024 assigned as the PDUFA target action date. Citius previously announced plans to form Citius Oncology, a standalone publicly traded company with LYMPHIR as its primary asset. LYMPHIR received orphan drug designation by the FDA for the treatment of CTCL and PTCL. In addition, Citius completed enrollment in its Phase 2b trial of CITI-002 (Halo-Lido), a topical formulation for the relief of hemorrhoids. For more information, please visit . Forward-Looking Statements 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. 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 "will," "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: risks to an approval of Mino-Lok by the FDA; our ability to commercialize our products, including Mino-Lok, if approved by the FDA; 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; the early stage of products under development; our need for substantial additional funds; our dependence on third-party suppliers; our ability to procure cGMP commercial-scale supply; the estimated markets for our product candidates and the acceptance thereof by any market; the ability of our product candidates to impact the quality of life of our target patient populations; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; 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 identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; 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 and could be impacted by any future public health risks. 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 SEC filings which are available on the SEC's website at , including in our Annual Report on Form 10-K for the year ended September 30, 2023, filed with the SEC on December 29, 2023, 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. Investor Contact: Ilanit Allen [email protected] 908-967-6677 x113 Media Contact: STiR-communications Greg Salsburg [email protected] SOURCE Citius Pharmaceuticals, Inc.

Mino-Lok data analysis on track with topline results anticipated this quarter LYMPHIR biologics license application accepted with PDUFA target action date set for August 13, 2024 CRANFORD, N.J., May 14, 2024 /PRNewswire/ -- Citius Pharmaceuticals, Inc. ("Citius" or the "Company") (Nasdaq: CTXR), a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products today reported business and financial results for the fiscal second quarter 2024 ended March 31, 2024. Second Quarter 2024 Business Highlights and Subsequent Developments LYMPHIR™ (denileukin diftitox) biologics license application (BLA) accepted by the U.S. Food and Drug Administration (FDA) with August 13, 2024, assigned as Prescription Drug User Fee Act (PDUFA) target action date; Data analysis underway for completed Mino-Lok® Pivotal Phase 3 trial with topline results anticipated in calendar 2Q 2024; Continued engagement with FDA following end of Phase 2b meeting to determine next phase in the development of Halo-Lido for the treatment of hemorrhoids; Merger of our wholly owned subsidiary with TenX Keane Acquisition (Nasdaq: TENK) to form publicly listed Citius Oncology, Inc. is progressing and pending review by Securities and Exchange Commission (SEC) and TENK shareholder approval; Robert Smith elected to the Citius Board of Directors at the Annual Meeting of Stockholders; and, Completed $15 million registered direct offering in April 2024 extending the Company's cash runway. Financial Highlights Cash and cash equivalents of $12.6 million as of March 31, 2024; $15 million in gross proceeds from a registered direct offering on April 30, 2024, extends the Company's cash runway through December 2024; R&D expenses were $3.6 million and $6.2 million for the three and six months ended March 31, 2024, respectively, compared to $4.7 million and $8.2 million for the three and six months ended March 31, 2023, respectively; G&A expenses were $4.3 million and $7.9 million for the three and six months ended March 31, 2024, respectively, compared to $4.8 million and $7.4 million for the three and six months ended March 31, 2023, respectively; Stock-based compensation expense was $3.1 million and $6.1 million for the three and six months ended March 31, 2024, respectively, compared to $1.2 million and $2.4 million for the three and six months ended March 31, 2023, respectively; and, Net loss was $8.5 million and $17.8 million, or ($0.05) and ($0.11) per share for the three and six months ended March 31, 2024, respectively, compared to a net loss of $10.5 million and $14.1 million, or ($0.07) and ($0.10) per share for the three and six months ended March 31, 2023, respectively. "I am pleased to share that we made solid progress this quarter as we focused on execution and managing our finances. The data analysis of our late-stage asset, Mino-Lok, the only treatment of its kind in development to salvage infected catheters, remains on track. We look forward to reporting the topline results later this quarter. Once we review the results, we plan to engage with the FDA to determine the optimal next steps in the program and look forward to advancing this much-needed alternative to the current standard of care, which often involves painful and costly catheter removal and replacement," stated Leonard Mazur, Chairman and CEO of Citius. "Importantly, the BLA submission for LYMPHIR, our novel IL-2 receptor targeted oncology therapy, was accepted by the FDA, and assigned a late summer 2024 PDUFA target action date. In anticipation of potential approval, we continue to align the organization for a successful launch," added Mazur. "Despite a tough capital market environment for pre-revenue companies, we successfully completed a $15 million registered direct offering, expanding our cash runway and providing capital to support the execution of our strategic plan. We believe that the merger of our oncology subsidiary with TenX to form a publicly listed company will make our company more attractive to investors and increase the value of our assets. This transaction is progressing, and we expect it to be completed in the coming months as we finalize SEC review and await approval by TENK shareholders. As we continue to meet our goals, we believe additional opportunities to strengthen our capital structure will become available," concluded Mazur. SECOND QUARTER 2024 FINANCIAL RESULTS: Liquidity As of March 31, 2024, the Company had $12.6 million in cash and cash equivalents. As of March 31, 2024, the Company had 159,094,781 common shares outstanding. Based on our cash and cash equivalents as of March 31, 2024, and after giving effect to a capital raising that closed on April 30, 2024, we expect to have sufficient funds to continue our operations through December 2024. We expect to raise additional capital in the future to support our operations beyond December 2024. Research and Development (R&D) Expenses R&D expenses were $3.6 million for the quarter ended March 31, 2024, compared to $4.7 million for the quarter ended March 31, 2023. For the six months ended March 31, 2024, R&D expenses were $6.2 million as compared to $8.2 million during the six months ended March 31, 2023, a decrease of $1.9 million. The decrease primarily reflects incremental costs related to the completion of the Mino-Lok Phase 3 trial and remediation activities for the LYMPHIR BLA resubmission, offset by lower costs in the current period due to the completion of the Halo-Lido Phase 2b trial. We expect that research and development expenses will stabilize at current levels in fiscal 2024 as we focus on the commercialization of LYMPHIR, complete our Phase 3 trial for Mino-Lok, and analyze the data from our Phase 2b trial and begin planning our Phase 3 trial for Halo-Lido General and Administrative (G&A) Expenses G&A expenses were $4.3 million for the quarter ended March 31, 2024, compared to $4.8 million for the quarter ended March 31, 2023. The decrease was primarily due to lower costs for pre-launch and market research activities associated with LYMPHIR during the period. For the six months ended March 31, 2024, G&A expenses were $7.9 million as compared to $7.4 million during the six months ended March 31, 2023. The primary reason for the increase was higher costs for pre-launch and market research activities associated with LYMPHIR. General and administrative expenses consist primarily of compensation costs, professional fees for legal, regulatory, accounting, and corporate development services, and investor relations expenses. Stock-based Compensation Expense For the quarter ended March 31, 2024, stock-based compensation expense was $3.1 million as compared to $1.2 million for the quarter ended March 31, 2023. For the six months ended March 31, 2024, stock-based compensation expense was $6.1 million as compared to $2.4 million for the six months ended March 31, 2023. The increase is primarily due to the Citius Oncology stock plan. Net loss Net loss was $8.5 million, or ($0.05) per share for the quarter ended March 31, 2024, compared to a net loss of $10.5 million, or ($0.07) per share for the quarter ended March 31, 2023. The $2 million decrease in the net loss was due to decreases of $1.1 million in research and development expenses and $0.5 million in general and administrative expenses, and the increase in other income of $2.3 million, being partially offset by the increase in stock-based compensation expense of $1.9 million. Net loss was $17.8 million, or ($0.11) per share for the six months ended March 31, 2024, compared to a net loss of $14.1 million, or ($0.10) per share for the six months ended March 31, 2023. The increase in the net loss was primarily due to the increase in stock-based compensation expense. About Citius Pharmaceuticals, Inc. Citius Pharma is a late-stage biopharmaceutical company dedicated to the development and commercialization of first-in-class critical care products. The Company's diversified pipeline includes two late-stage product candidates. At the end of 2023, Citius completed enrollment in a Phase 3 pivotal superiority trial of Mino-Lok®, an antibiotic lock solution to salvage catheters in patients with catheter-related bloodstream infections. The Biologics License Application for LYMPHIR™, a novel IL-2R immunotherapy for an initial indication in cutaneous T-cell lymphoma, is currently under review by the FDA with August 13, 2024 assigned as the PDUFA target action date. Citius previously announced plans to form Citius Oncology, a standalone publicly traded company with LYMPHIR as its primary asset. LYMPHIR received orphan drug designation by the FDA for the treatment of CTCL and PTCL. In addition, Citius completed enrollment in its Phase 2b trial of CITI-002 (Halo-Lido), a topical formulation for the relief of hemorrhoids. For more information, please visit . Forward-Looking Statements 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. 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 "will," "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: risks relating to the results of research and development activities, including those from existing and new pipeline assets; our need for substantial additional funds; uncertainties relating to preclinical and clinical testing; the FDA may not approve LYMPHIR; our ability to commercialize our products if approved by the FDA; our dependence on third-party suppliers; our ability to procure cGMP commercial-scale supply; the estimated markets for our product candidates and the acceptance thereof by any market; the ability of our product candidates to impact the quality of life of our target patient populations; our ability to obtain, perform under and maintain financing and strategic agreements and relationships; 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; our ability to realize some or all of the benefits expected to result from the anticipated spinoff of Citius Oncology or the delay of such benefits; our ongoing businesses which may be adversely affected and subject to certain risks and consequences as a result of the anticipated spinoff transaction; patent and intellectual property matters; our ability to identify, acquire, close and integrate product candidates and companies successfully and on a timely basis; 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 and could be impacted by any future public health risks. 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 SEC filings which are available on the SEC's website at , including in our Annual Report on Form 10-K for the year ended September 30, 2023, filed with the SEC on December 29, 2023, 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. Investor Relations for Citius Pharmaceuticals: Investor Contact: Ilanit Allen [email protected] 908-967-6677 x113 Media Contact: STiR-communications Greg Salsburg [email protected] -- Financial Tables Follow – SOURCE Citius Pharmaceuticals, Inc.