Paromomycin or Metronidazole for Symptomatic Dientamoeba Fragilis in Adults (COMFORTER Trial) - Protocol for a Superiority Double Blind, Randomized Controlled Trial

Dientameba Fragilis (D.fragilis) is a protozoan found in the digestive tract - in the human colon. there are disagreements regarding the preferred treatment for these cases, with several regimens tested in mostly small observational studies. Several drugs are currently recommended for D.fragilis, with metronidazole most commonly used. However, metronidazole therapy for treating dientamoebiasis in children was not associated with better clinical outcomes in a randomized, double-blinded and placebo-controlled clinical trial.
Hence, we aim to perform a double blind, randomized controlled trial, evaluating the clinical and microbiological efficacy of paromomycin versus metronidazole for the treatment of symptomatic adults with PCR positive dientamoeba fragilis.
The primary outcomes would be clinical improvement or resolution. Secondary outcomes include clinical improvement evaluated by a visual analogue scale; microbiological eradication, quality of life, and adverse events related to therapy.
We plan to include 60 patients (30 per arm)

Start Date22 Jan 2024

Sponsor / Collaborator

Sheba Medical Center

RBR-4ypn69

/ Not yet recruitingPhase 3IIT

Open, randomized clinical trial of efficacy and safety of sequential use ofsingle dose of intralesional meglumine antimoniate and topical paromomycin gelcompared to three intralesional applications of meglumine antimoniate in thetreatment of cutaneous leishmaniasis

Start Date01 Feb 2021

Sponsor / Collaborator

Fundação Oswaldo Cruz

NCT03829917

/ CompletedPhase 2/3IIT

Oral Miltefosine Plus Topical Paromomycin In American Cutaneous Leishmaniasis

Cure rate for L braziliensis bolivian CL has been 70%-80% for standard systemic and local monotherapies. It would benefit patients if cure rates could be consistently >90%, so testing a combination of two treatments is proposed. The most attractive systemic therapy is the only oral agent, miltefosine during 28 days, and the most attractive local therapy is application of Paromomycin cream for 28 days.

Start Date01 Feb 2019

Sponsor / Collaborator

Alfred Foundation

100 Clinical Results associated with Paromomycin Sulfate

100 Translational Medicine associated with Paromomycin Sulfate

100 Patents (Medical) associated with Paromomycin Sulfate

2,584

Literatures (Medical) associated with Paromomycin Sulfate

01 Oct 2025·Journal of Computational Biophysics and Chemistry

Utilizing Non-β-Lactam Antibiotics to Combat Antimicrobial Resistance by Targeting Multiple Virulence Factors of Pseudomonas aeruginosa

Author: Arshad, Mohammed ; Parveen, Nagma ; Husain, Fohad Mabood ; Furkan, Mohammad ; Khan, Altaf ; Qais, Faizan Abul ; Khan, Rizwan Hasan ; Ahmad, Iqbal ; Adil, Mohd

In pre-antibiotic times, various highly contagious diseases like cholera, smallpox and tuberculosis were widespread worldwide. Penicillin discovery in the late 1920s was a groundbreaking moment in medical history, saving countless lives. However, over the next few decades, microbes developed antibiotic resistance, leading to a global public health threat known as antimicrobial resistance (AMR). Pseudomonas aeruginosa is a major contributor to hospital-acquired infections, affecting millions of patients and causing numerous deaths annually. Several non-[Formula: see text]-lactam antibiotics combat these infections effectively, while their effect on P. aeruginosa quorum sensing (QS) has been insufficiently explored. We have undertaken comprehensive research to understand the effect of non-[Formula: see text]-lactam antibiotics on various targets of P. aeruginosa. Using molecular simulations, we scrutinize these antibiotics” dynamic behavior and stability. Based on toxicity, binding energy and binding site, platensimycin and sulfasalazine were identified as promising candidates against various targets of P. aeruginosa. The binding energies for sulfasalazine and platensimycin with LasA were found to be −8.1 and −8.6 kcal/mol, respectively. Both of these leading antibiotics were interacting at the active sites of all tested proteins (LasA, LasI and PqsR). The examination of molecular dynamics confirmed the stable complex formation of the lead non-[Formula: see text]-lactam antibiotics with all selected target proteins under normal physiological conditions. These findings emphasize the potential efficacy of platensimycin and sulfasalazine. They could potentially be repurposed for targeting the QS of P. aeruginosa.

01 Oct 2025·EUROPEAN JOURNAL OF MEDICINAL CHEMISTRY

Investigation into the binding domains of platelet factor 4 unlocks new avenues for the design and synthesis of selective sulfated pseudo-tetrasaccharide aminoglycoside ligands

Article

Author: Nguyen, Hien M ; Philip, Livia ; Abdulsalam, Hawau ; Singh, Kartikey

Platelet factor 4 (PF4) is a natural chemokine that binds to negatively charged glycosaminoglycans (GAGs), including the anticoagulant heparin. The formation of the PF4-heparin complex elicits an immune response that results in platelet activation, leading to serious thrombotic complications. This study explores the structure-activity relationships (SAR) of sulfated pseudo-tetrasaccharide aminoglycoside ligands. The binding interactions of these synthetically designed compounds with heparanase (HPSE) and PF4 were systematically elucidated. Through computational design, a library of sulfated aminoglycoside ligands was synthesized in 10-13 steps from readily available paromomycin and neomycin. The SAR studies revealed that hydroxyl-capped ligands interacted with the fondaparinux-binding domain of PF4, while hydrophobic-capped ligands bound to the heparin-binding domain. Notably, steric hindrance imposed by hydrophobic groups impedes the binding of the ligands to PF4's shallow binding site. In contrast, these hydrophobic-capped ligands demonstrated a strong binding affinity for HPSE. The most selective ligands reduced the viability of HPSE-overexpressing cancer cells, highlighting their potential efficacy in modulating the enzymatic activity of HPSE. This SAR study provides a foundational framework for the design of sulfated aminoglycoside-based therapeutics with minimized adverse effects associated with PF4.

01 Aug 2025·EXPERIMENTAL EYE RESEARCH

Whole genome sequencing-based prediction of antibiotic-resistance of ocular Staphylococcus aureus across six continents

Article

Author: Shen, Jiawei ; Yasir, Muhammad ; Willcox, Mark

Staphylococcus aureus is a leading cause of ocular infections, resulting in vision loss in severe cases. Understanding the antibiotic resistance profiles of ocular S. aureus can help customize treatments. However, there is a lack of global data on the resistance patterns of ocular isolates and comparative regional analyses. Hence, WGS data from 195 ocular S. aureus isolates across six continents were analysed to identify antibiotic resistance genes (ARGs) and predict antibiotic resistance phenotypes in this study. A total of 40 ARGs were detected, involving resistance mechanisms against aminoglycosides, beta-lactams, macrolide-lacosamide-streptogramin B (MLS_B), glycopeptides, tetracyclines, other antibiotic classes, and efflux pump regulators. Notably, the prevalences of ARGs associated with efflux pump regulators and beta-lactams were particularly high (>80 %). Resistance to 45 antibiotics was predicted across the isolates, with 51 % identified as multidrug-resistant (MDR), while only 8 % were predicted to be fully susceptible to all predicted antibiotics. Regional data varied, with isolates from North America and Asia exhibiting the most extensive resistance patterns, showing predicted resistance to 45 and 41 antibiotics, respectively. In contrast, Oceanian isolates were predicted to be resistant to only 14 antibiotics. Beta-lactams showed the highest predicted resistance prevalence among all antibiotic classes. Notably, North American isolates showed markedly higher resistance to MLS_B antibiotics. A high proportion of cloud genes highlights the need for monitoring regional resistance. This study provides antibiotic resistance profiles among ocular S. aureus using WGS prediction, emphasizing the importance of regional surveillance and antimicrobial stewardship to suggest effective treatment strategies. It is recommended that WGS of more strains be deposited to overcome limited data, and laboratory tests be performed to analyse the consistency between genetic predicted and phenotypic resistance.

News (Medical) associated with Paromomycin Sulfate

14 Nov 2024

·www.globenewswire.com

Appili Therapeutics Reports Financial and Operational Results for Second Quarter of Fiscal Year 2025

Overwhelming shareholder support in favour of take-private transaction with Aditxt Studies presented at IDWeek 2024™ demonstrate ATI-1701 provides full protection against lethal tularemia in animal models after one year Alignment with U.S. Food and Drug Administration on development requirements for ATI-1801 NDA Submission HALIFAX, Nova Scotia, Nov. 14, 2024 (GLOBE NEWSWIRE) -- Appili Therapeutics Inc. (TSX:APLI; OTCPink: APLIF) (the “Company” or “Appili”), a biopharmaceutical company focused on drug development for infectious diseases and medical countermeasures, today announced its financial and operational results for the second quarter of its fiscal year 2025, which ended on September 30, 2024. All figures are stated in Canadian dollars unless otherwise stated. “During fiscal year 2025, Appili shareholders voted overwhelmingly in favor of the transaction with Aditxt, facilitating our access to capital and activating promising opportunities for Appili,” said Dr. Don Cilla, President and CEO of Appili. “The shareholder vote is an important milestone for Appili and brings us one step closer to ‘Making Promising Innovations Possible Together’ through Aditxt’s social incubator platform.” ATI-1701, Biodefense Vaccine Candidate with Strong U.S. Air Force Academy Funding Commitments ATI-1701, a novel live-attenuated vaccine for preventing F. tularensis, has previously secured US$14 million in awards from the United States Air Force Academy (“USAFA”). Under the cooperative agreement with USAFA, Appili manages a comprehensive development program, including nonclinical studies, CMC/manufacturing, clinical preparatory, and regulatory activities supporting an IND submission in 2025. Recent data presented by Appili's scientific team at the Military Health System Research Symposium and IDWeek 2024™ demonstrated strong efficacy results. Studies showed that a single dose of ATI-1701 provided full protection against lethal tularemia in animal models, with protection for one year after vaccination. Additional research highlighted the heightened risk of tularemia outbreaks in conflict zones and positioned ATI-1701 as a potentially valuable tool in protecting warfighters. Appili has recently had positive interactions with the United States Food and Drug Administration (“FDA”) in the form of a pre-IND meeting, confirming the development pathway for the majority of Appili’s proposed development plan. Appili is currently in the process of implementing suggested changes in its development effort. ATI-1801, Demonstrated safety and efficacy across multiple Phase 2 and Phase 3 studies ATI-1801, a novel topical formulation of paromomycin (15% w/w), is under advanced clinical development for treating cutaneous leishmaniasis, a disfiguring skin infection affecting hundreds of thousands globally. In a recent response to Appili’s Type B meeting request, the FDA agreed with the Company’s proposed strategy to establish a scientific bridge between previous clinical trial material and new drug product batches. The approach enables Appili to leverage key results from the clinical dossier for ATI-1801 licensed from U.S. Army Medical Materiel Development Activity, including the successful results of a Phase 3 study, and allows the completion of an New Drug Application much sooner than if additional clinical data were required. ATI-1801 has received Orphan Drug Designation from the FDA for certain forms of cutaneous leishmaniasis. The company is evaluating ATI-1801’s eligibility for a priority review voucher (“PRV”), which, if confirmed, would make it the second PRV-eligible program at Appili, alongside ATI-1701, which is potentially eligible for a PRV pending renewal of certain U.S. legislation. LIKMEZ™ (ATI-1501), FDA Approved Liquid Oral Reformulation of the Antibiotic Metronidazole In September 2023, Appili, together with its U.S. partner, Saptalis Pharmaceuticals LLC., announced the approval by the FDA of LIKMEZ™ (ATI-1501), LIKMEZ® is the first and only FDA-approved liquid formulation of metronidazole providing a novel prescribing option for patients with difficulty swallowing or experiencing taste-related hurdles. LIKMEZ, with patent coverage through 2039, addresses the unmet need in patients with dysphagia to avoid risks associated with drug compounding, and discontinuation related anti-microbial resistance. In November 2023, Saptalis, through a commercialization partner, launched LIKMEZ in the U.S. and the product is now available to patients and doctors. Appili earned milestone payments from Saptalis and is entitled to receive additional sales-based milestone payments and royalties from Saptalis based on sale of the product. Saptalis is currently in the process of identifying an alternative commercialization partner. Aditxt Arrangement On November 6, 2024, shareholders of the Company overwhelmingly voted in favour of a special resolution approving a plan of arrangement pursuant to which Aditxt Inc. (NASDAQ:ADTX) (“Aditxt”), through its wholly-owned subsidiary, Adivir, Inc. will acquire all of the issued and outstanding Class A common shares of the Company by way of a court-approved plan of arrangement under the Business Corporations Act (Ontario) (the “Transaction”). Approximately 29.50% of the outstanding Appili Shares were voted by shareholders and the Transaction was approved by 99.79% of the votes cast. For further details regarding the Transaction, please see Appili’s press release dated April 2, 2024 and Appili’s management information circular (the “Circular”) dated October 4, 2024, a copy of which is available under Appili’s profile on SEDAR+ (sedarplus.ca). Appili and Aditxt are in the process of satisfying the remaining closing conditions, which include: (1) the Company obtaining final court approval for the Transaction, and (2) Aditxt securing sufficient financing to complete the Transaction. Subject to satisfaction or waiver of the remaining closing conditions, Appili expects the Transaction to close in December 2024. Financial Results The Company prepares its financial statements in accordance with IFRS Accounting Standards as issued by the International Accounting Standard Board and Part I of Chartered Professional Accountants of Canada Handbook–Accounting. All figures are stated in Canadian dollars unless otherwise stated. The net loss and comprehensive loss of $0.8 million or $0.01 loss per share for the three months ended September 30, 2024, was $0.2 million lower than the net loss and comprehensive loss of $1 million or $0.01 loss per share during the three months ended September 30, 2023. This relates mainly to an increase in research and development expenses of $1 million, an increase in financing costs of $0.1 million and a decrease in revenue of $0.3 million. This increase was offset by a decrease in general and administrative expenses by $0.1 million, an increase in government assistance of $1.3 million, and an increase of $0.2 million in foreign exchange gain. As of September 30, 2024, the Company had cash of $0.7 million, compared to $0.1 million on March 31, 2024. As of November 13, 2024, the Company had 121,266,120 issued and outstanding Common Shares, 11,520,281 stock options, and 41,356,874 warrants outstanding. This press release should be read in conjunction with the Company’s unaudited interim condensed consolidated financial statements for the second quarter of the 2025 fiscal year and the related MD&A, copies of which are available under the Company’s profile on SEDAR+ at www.sedarplus.ca. About Appili TherapeuticsAppili Therapeutics is an infectious disease biopharmaceutical company that is purposefully built, portfolio-driven, and people-focused to fulfill its mission of solving life-threatening infections. By systematically identifying urgent infections with unmet needs, Appili’s goal is to strategically develop a pipeline of novel therapies to prevent deaths and improve lives. The Company is currently advancing a diverse range of anti-infectives, including an FDA approved ready-made suspension of metronidazole for the treatment of antimicrobial infections, a vaccine candidate to eliminate a serious biological weapon threat, and a topical antiparasitic for the treatment of a disfiguring disease. Led by a proven management team, Appili is at the epicenter of the global fight against infection. For more information, visit www.AppiliTherapeutics.com. Forward looking statementsThis news release contains “forward-looking statements”, including with respect to the funding commitment from USAFA and expected timing of invoice payment, advancing program towards IND, further anticipated milestones and the timing thereof, the Company’s development plans and timelines with respect to ATI-1501, ATI-1701 and ATI-1801, the timing of any milestone and/or royalty payments in respect of ATI-1501, consideration to be received by shareholders in connection with the proposed Transaction, the expected timing and process to complete the Transaction, and the Company’s expectations with respect to its ability to operate as a going concern and satisfy its ongoing working capital requirements. Wherever possible, words such as “may,” “would,” “could,” “should,” “will,” “anticipate,” “believe,” “plan,” “expect,” “intend,” “estimate,” “potential for” and similar expressions have been used to identify these forward-looking statements. These forward-looking statements reflect the current expectations of the Company’s management for future growth, results of operations, performance and business prospects and opportunities and involve significant known and unknown risks, uncertainties and assumptions, including, without limitation, those listed in (i) the annual information form of the Company dated June 25, 2024, (ii) the Cicular, and (iii) the other filings made by the Company with the Canadian securities regulatory authorities (which may be viewed at www.sedarplus.ca). Should one or more of these risks or uncertainties materialize or should assumptions underlying the forward-looking statements prove incorrect, actual results, performance or achievements may vary materially from those expressed or implied by the forward-looking statements contained in this news release. These factors should be considered carefully, and prospective investors should not place undue reliance on the forward-looking statements. The Company disclaims any intention or obligation to revise forward-looking statements whether as a result of new information, future developments or otherwise, except as required by law. All statements included in this press release relating to the Arrangement are qualified by reference to the “Forward Looking Statements” section included in the Arrangement Press Release. Media Contact:Jenna McNeil, Communications ManagerAppili Therapeutics E: JMcNeil@AppiliTherapeutics.com Investor Relations Contact:Don Cilla, President and CEOAppili TherapeuticsE: Info@AppiliTherapeutics.com

Clinical ResultDrug ApprovalVaccineOrphan DrugPhase 3

06 Nov 2024

·www.globenewswire.com

Appili Therapeutics Announces Results of Special Meeting of Shareholders

NOT FOR DISTRIBUTION TO UNITED STATES NEWSWIRE SERVICES OR FOR DISSEMINATION IN THE UNITED STATES HALIFAX, Nova Scotia, Nov. 06, 2024 (GLOBE NEWSWIRE) -- Appili Therapeutics Inc. (TSX: APLI; OTCPink: APLIF) (the “Company” or “Appili”), a biopharmaceutical company focused on drug development for infectious diseases and medical countermeasures, today announced the results from its special meeting of shareholders held earlier today (the “Meeting”). Shareholders of the Company (the “Shareholders”), overwhelmingly voted in favour of a special resolution approving a plan of arrangement pursuant to which Aditxt Inc. (NASDAQ:ADTX) (“Aditxt”), through its wholly-owned subsidiary, Adivir, Inc. will acquire all of the issued and outstanding Class A common shares of the Company (the “Appili Shares”) by way of a court-approved plan of arrangement under the Business Corporations Act (Ontario) (the “Transaction”). A total of 35,770,287 Appili Shares were voted at the Meeting, representing approximately 29.50% of the outstanding Appili Shares. The Transaction was approved by (i) 99.79% of the votes cast by Shareholders at the Meeting and (ii) 99.65% of the votes cast by Shareholders at the Meeting (excluding the votes cast by persons whose votes may not be included in determining minority approval of a “business combination” in accordance with Multilateral Instrument 61-101 – Protection of Minority Security Holders in Special Transactions). The successful close of this transaction is an important milestone for Appili as it focuses on advancing its pipeline of anti-infective therapies and delivering on its mission to protect patients from severe infectious diseases. “The alignment with Aditxt will facilitate our access to capital and activates promising opportunities for Appili across multiple fronts. Through Aditxt’s social incubator platform, we can amplify our impact and reach in the infectious diseases and medical countermeasure landscape.” said Dr. Don Cilla, President and CEO of Appili. “By leveraging Aditxt’s mission to "Make Promising Innovations Possible Together", we aim to prepare ATI-1701 for IND submission, implement FDA’s agreed upon strategy for ATI-1801, and enhance commercialization for LIKMEZ™ and capitalize on the strategic value of two potential PRV eligible programs.” The Transaction is conditional upon certain customary closing conditions and Aditxt raising at least US$20 million in financing. The Transaction is expected to close in Q4 2024. The Company has filed a report of the voting results on the resolution voted on the Meeting on the Company’s SEDAR+ profile at www.sedarplus.ca. About Appili Therapeutics Appili Therapeutics is an infectious disease biopharmaceutical company that is purposefully built, portfolio-driven, and people-focused to fulfill its mission of solving life-threatening infections. By systematically identifying urgent infections with unmet needs, Appili’s goal is to strategically develop a pipeline of novel therapies to prevent deaths and improve lives. The Company is currently advancing a diverse range of anti-infectives, including an FDA approved ready-made suspension of metronidazole for the treatment of antimicrobial infections, a vaccine candidate to eliminate a serious biological weapon threat, and a topical antiparasitic for the treatment of a disfiguring disease. Led by a proven management team, Appili is at the epicenter of the global fight against infection. For more information, visit www.AppiliTherapeutics.com. About Aditxt Aditxt, Inc.® is an innovation platform dedicated to accelerating promising health innovations. Aditxt’s ecosystem of research institutions, industry partners, and shareholders collaboratively drives their mission to "Make Promising Innovations Possible Together." The innovation platform is the cornerstone of Aditxt’s strategy, where multiple disciplines drive disruptive growth and address significant societal challenges. Aditxt operates a unique model that democratizes innovation, ensures every stakeholder’s voice is heard and valued and empowers collective progress. Aditxt currently operates two programs focused on immune health and precision health. The Company plans to introduce two additional programs dedicated to public health and women’s health. For these, Aditxt has entered into an Arrangement Agreement with Appili Therapeutics, Inc. (“Appili”) (TSX: APLI; OTCPink: APLIF), which focuses on infectious diseases, and a Merger Agreement with Evofem Biosciences, Inc. Each program will be designed to function autonomously while collectively advancing Aditxt’s mission of discovering, developing, and deploying innovative health solutions to tackle some of the most urgent health challenges. The closing of each of the transactions with Appili and Evofem is subject to several conditions, including but not limited to approval of the transactions by the respective target shareholders and Aditxt raising sufficient capital to fund its obligations at closing. No assurance can be provided that all of the conditions to closing will be obtained or satisfied or that either of the transactions will ultimately close. For more information, www.aditxt.com. CAUTIONARY NOTE REGARDING FORWARD-LOOKING STATEMENTS This news release contains “forward-looking information” within the meaning of applicable securities laws. Forward-looking information contained in this press release may be identified by the use of words such as, “may”, “would”, “could”, “will”, “likely”, “expect”, “anticipate”, “believe, “intend”, “plan”, “forecast”, “project”, “estimate”, “outlook” and other similar expressions. Forward-looking information is not a guarantee of future performance and is based upon a number of estimates and assumptions of management in light of management’s experience and perception of trends, current conditions and expected developments, as well as other factors relevant in the circumstances, including assumptions in respect of current and future market conditions, the current and future regulatory environment, and the availability of licenses, approvals and permits. Forward-looking statements in this news release include, but are not limited to closing of the Transaction and the timing thereof. Actual results and developments may differ materially from those contemplated by these statements. Such forward-looking statements are based on certain assumptions regarding Aditxt and Appili, including the successful completion of the conditions precedent of the Transaction. While Appili considers these assumptions to be reasonable, based on information currently available, they may prove to be incorrect. Among other things, there can be no assurance that the Transaction will be completed. Forward-looking information is subject to a variety of risks and uncertainties that could cause actual events or results to differ materially from those projected in the forward-looking information. Consequently, readers are cautioned not to place undue reliance on the forward-looking statements and information contained in this press release. The statements in this press release are made as of the date of this release. Appili disclaims any intent or obligation to update any forward-looking information, whether as a result of new information, future events or results or otherwise, other than as required by applicable securities laws. No securities regulatory authority has either approved or disapproved of the contents of this press release. This press release is for information purposes only and shall not constitute an offer to sell or the solicitation of an offer to buy, nor shall there be any sale of these securities in any state or jurisdiction in which such offer, solicitation or sale would be unlawful prior to registration or qualification under the securities laws of any such state or jurisdiction. Neither CIRO nor any stock exchange or other securities regulatory authority accepts responsibility for the adequacy or accuracy of this release. Media Contact:Jenna McNeil, Corporate Affairs and Communications ManagerAppili TherapeuticsE: JMcNeil@AppiliTherapeutics.com Investor Relations Contact:Don Cilla, Pharm.D. M.B.A.Appili TherapeuticsE: Info@AppiliTherapeutics.com

Vaccine

31 Oct 2024

·www.globenewswire.com

Appili Therapeutics Announces Alignment with FDA on Development Requirements for ATI-1801 Topical Antiparasitic Product NDA Submission

Demonstrated safety and efficacy across multiple Phase 2 and Phase 3 studies Appili provides update on Aditxt transaction and encourages shareholders to vote their shares as soon as possible for the November 6, 2024 shareholders meeting HALIFAX, Nova Scotia, Oct. 31, 2024 (GLOBE NEWSWIRE) -- Appili Therapeutics Inc. (TSX: APLI; OTCPink: APLIF) (the “Company” or “Appili”), a biopharmaceutical company focused on drug development for infectious diseases and medical countermeasures, today announced that the U.S. Food and Drug Administration (“FDA”) has provided positive feedback regarding the development strategy for ATI-1801, and agreed on the necessary registration package to support a New Drug Application (“NDA”). ATI-1801, a novel topical formulation of paromomycin (15% w/w), is under advanced clinical development for treating cutaneous leishmaniasis, a disfiguring skin infection affecting hundreds of thousands globally. “This positive response from the FDA simplifies and de-risks our development program for ATI-1801,” said Don Cilla, President and CEO of Appili Therapeutics. “The agency's agreement with our proposed strategy enables Appili to leverage key results from the clinical dossier for ATI-1801 licensed from U.S. Army Medical Materiel Development Activity (“USAMMDA”), including the successful results of a Phase 3 study, and provides a clear path towards an NDA submission.” In a recent response to Appili's Type B meeting request, the FDA agreed with the Company's proposed strategy to establish a scientific bridge between previous clinical trial material and new drug product batches. This approach includes developing an appropriately validated in-vitro release test (“IVRT”) method and manufacturing a new reference standard to use in IVRT studies to support the scientific bridge to products used in prior studies. This will allow completion and submission of an NDA much sooner than if additional clinical data were required. Subject to securing the requisite funding Appili expects to implement the agreed-upon strategy and continue to advance ATI-1801 topical paromomycin cream (15% w/w) program to NDA submission. ATI-1801 has received Orphan Drug Designation from the FDA for certain forms of cutaneous leishmaniasis. The company is evaluating ATI-1801’s eligibility for a priority review voucher (“PRV”), which, if confirmed, would make it the second PRV-eligible program at Appili, alongside ATI-1701, which is potentially eligible for a PRV pending renewal of certain U.S. legislation. Aditxt Arrangement On April 2, 2024, the Company announced that it had entered into a definitive arrangement agreement (the “Arrangement Agreement”) pursuant to which Aditxt Inc. (NASDAQ: ADTX) (“Aditxt”), through its wholly-owned subsidiary, Adivir, Inc., agreed to acquire all of the issued and outstanding Class A common shares of the Company. Under the terms of the Arrangement Agreement, shareholders of the Company will receive (i) US$ 0.0467 in cash and (ii) 0.0000686251 of a share of Adixt common stock (the “Transaction”). This Transaction represents a premium to Appili’s recent trading price, and assuming the completion of the transaction, provides shareholders with immediate value. On October 1, 2024, the Company obtained an interim order from the Ontario Superior Court of Justice in connection with the Transaction. The order permits the mailing of all requisite meeting materials in connection with the special meeting of Appili shareholders (the “Meeting”) scheduled to be held virtually on November 6, 2024 at 11:00 am ET. Shareholders are encouraged to vote their shares as soon as possible. The notice of the special meeting of shareholders and management information circular containing all relevant details with respect to the Meeting (including voting instructions) and the Transaction are available on the Company’s website at: www.appilitherapeutics.com/proxy-info/ as well as under Appili’s profile on SEDAR+ www.sedarplus.ca. About ATI-1801Licensed from the U.S. Department of Defense through the U.S. Army Medical Materiel Development Activity, Appili’s ATI-1801 is a novel topical product with demonstrated safety and efficacy across multiple Phase 2 and Phase 3 studies. As current treatments are often invasive and require hospitalization, ATI-1801 has the potential to significantly reduce suffering from cutaneous leishmaniasis by providing patients in need with a safe and effective topical therapy that can be used in the outpatient setting. About Appili TherapeuticsAppili Therapeutics is an infectious disease biopharmaceutical company that is purposefully built, portfolio-driven, and people-focused to fulfill its mission of solving life-threatening infections. By systematically identifying urgent infections with unmet needs, Appili’s goal is to strategically develop a pipeline of novel therapies to prevent deaths and improve lives. The Company is currently advancing a diverse range of anti-infectives, including an FDA approved ready-made suspension of metronidazole for the treatment of antimicrobial infections, a vaccine candidate to eliminate a serious biological weapon threat, and a topical antiparasitic for the treatment of a disfiguring disease. Led by a proven management team, Appili is at the epicenter of the global fight against infection. For more information, visit www.AppiliTherapeutics.com. Forward Looking StatementsThis news release contains “forward-looking statements”, including with respect to (i) the potential for ATI-1801 as a treatment for leishmaniasis and the proposed development plan for ATI-1801(including the NDA submission and the expected timing thereof) and (ii) the potential that certain of Appili’s programs may be PRV-eligible. Wherever possible, words such as “may,” “would,” “could,” “should,” “will,” “anticipate,” “believe,” “plan,” “expect,” “intend,” “estimate,” “potential for” and similar expressions have been used to identify these forward-looking statements. These forward-looking statements reflect the current expectations of the Company’s management for future growth, results of operations, performance and business prospects and opportunities and involve significant known and unknown risks, uncertainties and assumptions, including, without limitation, those listed in the annual information form of the Company dated June 25, 2024, and the other filings made by the Company with the Canadian securities regulatory authorities (which may be viewed at www.sedarplus.ca). Should one or more of these risks or uncertainties materialize or should assumptions underlying the forward-looking statements prove incorrect, actual results, performance or achievements may vary materially from those expressed or implied by the forward-looking statements contained in this news release. These factors should be considered carefully, and prospective investors should not place undue reliance on the forward-looking statements. The Company disclaims any intention or obligation to revise forward-looking statements whether as a result of new information, future developments or otherwise, except as required by law. Media Contact:Jenna McNeil, Corporate Affairs and Communications ManagerAppili TherapeuticsE: JMcNeil@AppiliTherapeutics.com Investor Relations Contact:Don Cilla, Pharm.D. M.B.A.Appili TherapeuticsE: Info@AppiliTherapeutics.com

Phase 3NDAOrphan Drug

100 Deals associated with Paromomycin Sulfate

External Link

KEGG	Wiki	ATC	Drug Bank
-	Paromomycin Sulfate	A07AA06	DB01421

R&D Status

Approved

10 top approved records.

to view more data

Indication	Country/Location	Organization	Date
Amebiasis	Japan	Pfizer Japan, Inc.	25 Dec 2012
Cryptosporidiosis	-	-	01 Jan 1959
Giardiasis	-	-	01 Jan 1959

Developing

10 top R&D records.

to view more data

Indication	Highest Phase	Country/Location	Organization	Date
Leishmaniasis, Cutaneous	Phase 3	United States	Appili Therapeutics, Inc.	13 Apr 2022
Acquired Immunodeficiency Syndrome	Clinical	United States	Genetics Institute LLC	31 Aug 2001
Optic Atrophy, Autosomal Dominant	Preclinical	China	Shantou University	30 May 2025
Optic Atrophy, Autosomal Dominant	Preclinical	China	The Chinese University of Hong Kong	30 May 2025
Optic Atrophy, Autosomal Dominant	Preclinical	Hong Kong	The Hong Kong Polytechnic University	30 May 2025

Clinical Result

Indication

Phase

Evaluation

View All Results

Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT03399955 (not available, Pubmed \| PLoS Negl Trop Dis)	Phase 2	Post-Kala-azar dermal leishmanioid	110	Paromomycin/Miltefosine	jlrucokuas(swbjbzrajj) = ggydjhmpdh uauhzhdfkx (gkcmcpmdoi, 90.3 - 100)	Positive	21 Nov 2023
				Liposomal Amphotericin B/Miltefosine	jlrucokuas(swbjbzrajj) = nqlisbqhif uauhzhdfkx (gkcmcpmdoi, 70.2 - 91.9)
NCT03129646 (Pubmed)	Phase 3	Leishmaniasis, Visceral	439	Paromomycin plus Miltefosine	sljckpoxkv(ggbbagrkgt) = pfvebwpyuw xymbkhkmmv (qnjmwjwyrq, -6.2 to 7.4)	Positive	27 Sep 2022
				Sodium Stibogluconate plus Paromomycin	sljckpoxkv(ggbbagrkgt) = trxgdnepgw xymbkhkmmv (qnjmwjwyrq, -6.2 to 7.4)
NCT03759886 (Pubmed \| Int J Colorectal Dis)	Not Applicable	-	120	Intervention group (oral paromomycin and metronidazole)	rnicccrooq(mtefbodbur) = showed a tendency towards decreased frequencies in the intervention group wcyrskrrrt (qfpbcqqsvy )	-	25 Apr 2021
				paromomycine (Control group (no intervention))
NCT03096457 (Pubmed \| Clin Infect Dis)	Phase 2/3	Leishmaniasis, Cutaneous	80	15% Paromomycin-Aquaphilic	myftgzwwqf(gnrkvnyekw) = dymlbnoukb jfntrqozax (xhmdptdlpe, 62.5 - 88)	-	15 Feb 2019
				Aquaphilic vehicle	myftgzwwqf(gnrkvnyekw) = qipakfbtnk jfntrqozax (xhmdptdlpe, 3 - 30)
NCT01328457 (Pubmed \| PLoS Negl Trop Dis)	Not Applicable	Leishmaniasis, Visceral	120	Paromomycin Intramuscular Injection (PMIM)	svwtrftyfs(vcnvezaxik) = Injection site pain was the most commonly reported adverse event xajppcafmb (qgkgtfmlst ) View more	Positive	01 Jan 2015
NCT00255567 (Pubmed \| PLoS Negl Trop Dis)	Phase 2	Leishmaniasis, Visceral	42	Paromomycin 20 mg/kg/day for 21 days	omzmpofwuk(zeybakmqof) = urzsdxxzwf hjheqycalo (plniyqahno, 63.7% - 97.0%)	-	26 Oct 2010
				Paromomycin 15 mg/kg/day for 28 days	omzmpofwuk(zeybakmqof) = pdgqzpnlox hjheqycalo (plniyqahno, 69.6% - 98.8%)
NCT00629031 (Pubmed \| Clin Infect Dis)	Phase 3	Leishmaniasis, Visceral	329	paromomycin (Group A)	lebzebyozz(ixbrllcmas) = Mild grade injection site pain was the most common adverse event jugclwfxzy (horijmdrbw ) View more	-	15 Sep 2009
				paromomycin (Group B)
NCT00216346 (Pubmed \| N Engl J Med)	Phase 3	Leishmaniasis, Visceral	667	Paromomycin	xvtmavxlwr(vfutcdxzer) = fevers qjnkgfchul (mpleqvsazl ) View more	Positive	21 Jun 2007
				Amphotericin B

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