A Phase 2, Randomized, DB, Placebo-controlled Study to Determine the Efficacy, Safety and PK Profile of ARV-1801 in Combination With Optimized Background Therapy for the Treatment of Pulmonary Exacerbations in Patients With Cystic Fibrosis

This study will evaluate the efficacy and safety of an oral ARV-1801(ACG-701) plus optimized background therapy (OBT) compared to oral placebo plus OBT, each administered for 14 days, in the treatment of participants with Cystic Fibrosis-related pulmonary exacerbations (PEx).

Start Date10 Feb 2023

Sponsor / Collaborator

Aceragen, Inc.

NCT05105035 / CompletedPhase 2

A Randomized, Double-Blind, Placebo-Controlled, Exploratory Study to Assess the Efficacy, Safety and Tolerability of Oral ARV-1801 Given in Combination With Intravenous Ceftazidime or Meropenem for Intensive Phase Therapy of Melioidosis in Hospitalized Patients

The study investigates the effect of 14 days of twice daily doses of ARV-1801 or placebo in combination with meropenem or ceftazidime in patients hospitalized with melioidosis.

Start Date14 Jun 2022

Sponsor / Collaborator

Arrevus, Inc.Startup

ISRCTN14019371 / RecruitingPhase 2

A randomized, double-blind, placebo-controlled, exploratory study to assess the efficacy, safety, and tolerability of oral ARV-1801 given in combination with intravenous ceftazidime or meropenem for intensive phase therapy of melioidosis in hospitalized patients

Start Date01 May 2022

Sponsor / Collaborator

Arrevus, Inc.Startup

100 Clinical Results associated with Sodium Fusidate

100 Translational Medicine associated with Sodium Fusidate

100 Patents (Medical) associated with Sodium Fusidate

7,061

Literatures (Medical) associated with Sodium Fusidate

01 Feb 2025·FOOD CHEMISTRY

Dual cross-linking with tannic acid and transglutaminase improves microcapsule stability and encapsulates lemon essential oil for food preservation

Article

Author: Gong, Yuxi ; Wang, Yansong ; Zhang, Xiangyu ; Wu, Chao ; Wu, Dongmei ; Yang, Wei ; Li, Jinlian

The microencapsulation of essential oils by complex coacervation technology has attracted considerable attention. This paper deals with the preparation of gelatin-chitosan microcapsules through dual cross-linking using transglutaminase (TGase) and tannic acid (TA). Lemon essential oil (LEO) was successfully encapsulated with 82.5 % encapsulation efficiency. Compared to single cross-linking microcapsules (TG), dual cross-linking microcapsules (TG-TA) exhibit superior thermal stability and swell stability. In vitro release studies demonstrated that TG-TA exhibited better controlled-release behavior with a longer duration of action. Meanwhile, the lipid oxidation of TG-TA was 1.45 mg MDA/kg that of control group was 2.23 mg MDA/kg which showed their excellent antioxidant effects. Moreover TG-TA have higher antibacterial rate, more inhibition zone diameters and better effect for preventing the growth of total viable count than TG and LEO. This study has theoretical implications for the use of TG-TA ideal carriers for protecting various active substances, thus facilitating their applications in food preservation.

01 Jan 2025·JOURNAL OF ETHNOPHARMACOLOGY

Lavender improves sleep through olfactory perception and GABAergic neurons of the central amygdala

Article

Author: Gao, Jin-Xian ; Wang, Can ; Yang, Xing-Wen ; Xin, Le ; Lin, Jian-Sheng ; Ren, Yan-Li ; Hou, Yi-Ping ; Chen, Yu-Nong ; Xie, Jun-Fan ; Spruyt, Karen ; Chu, Wei-Wei ; Shao, Yu-Feng ; Yan, Gui-Zhong

ETHNOPHARMACOLOGICAL RELEVANCE:

The use of lavender as sleep aid or hypnotic agent can be traced back as early as ancient Romans and Greeks. Yet, objective experimental data on whether and how lavender enhances sleep duration or/and sleep quality remain lacking.

AIM OF THE STUDY:

We aimed to characterize the sleep-wake regulating effects of lavender in the mouse and to demonstrate the brain targets and neural circuits involved.

MATERIALS AND METHODS:

A self-made precise odor delivery system combined with chronic polysomnographic recordings was employed to assess the sleep-wake effects of inhalation with lavender essential oil (LEO, extracted from lavender) and its different constituents during the light and dark phases in free-moving C57BL/6J mice. Neuroviral labeling, in situ hybridization and pharmacogenetics were combined to identify the neural circuits and targets involved. Finally, an insomniac model of DL-4-Chlorophenylalanine (PCPA)-treated mice was established to examine the sleep-inducing potential of LEO.

RESULTS:

We found that inhalation of LEO with a concentration at 25.0% during the light (inactive) phase significantly shortened the latency to non-rapid eye movement (NREM) sleep, increased the total amount of NREM sleep at the expense of wakefulness (W), and enhanced cortical EEG slow wave activities, notably delta power spectra density. We further identified linalool, d-limonene, 1,8-cineole, linalyl acetate and terpinene-4-ol as the major effective sleep-promoting monomer components. Importantly, we found that LEO no longer produced any of the above sleep-promoting effect following either nasal injection of zinc sulfate which interrupts the olfactory pathway, or pharmacogenetics silencing of central amygdala GABAergic neurons. Finally, LEO reestablished NREM sleep with short latency in PCPA-treated insomniac mice, effects comparable with those induced by a potent sedative diazepam.

CONCLUSIONS:

We have characterized the quantitative and qualitative sleep-promoting effects of LEO and its effective components via the olfactory pathway and central amygdala GABA neuronal targets. The hypnotic property of LEO is reinforced by its ability to restore sleep in insomnia. Our study thus establishes a neurobiological basis for aromatherapy of sleep disorders using lavender.

01 Dec 2024·Applied microbiology and biotechnology

Metabolomic analysis of hydroxycinnamic acid inhibition on Saccharomyces cerevisiae

Article

Author: Chen, Junxiao ; Yi, Wenbo ; Gu, Jie ; Xu, Shujie ; Tan, Liping ; Liu, Tongjun ; Ge, Xiaoli

Abstract:

Ferulic acid (FA) and p-coumaric acid (p-CA) are hydroxycinnamic acid inhibitors that are mainly produced during the pretreatment of lignocellulose. To date, the inhibitory mechanism of hydroxycinnamic acid compounds on Saccharomyces cerevisiae has not been fully elucidated. In this study, liquid chromatography-mass spectrometry (LC–MS) and scanning electron microscopy (SEM) were used to investigate the changes in S. cerevisiae cells treated with FA and p-CA. In this experiment, the control group was denoted as group CK, the FA-treated group was denoted as group F, and the p-CA-treated group was denoted as group P. One hundred different metabolites in group F and group CK and 92 different metabolites in group P and group CK were selected and introduced to metaboanalyst, respectively. A total of 38 metabolic pathways were enriched in S. cerevisiae under FA stress, and 27 metabolic pathways were enriched in S. cerevisiae under p-CA stress as identified through Kyoto Encyclopaedia of Genes and Genomes (KEGG) analysis. The differential metabolites involved included S-adenosine methionine, L-arginine, and cysteine, which were significantly downregulated, and acetyl-CoA, L-glutamic acid, and L-threonine, which were significantly upregulated. Analysis of differential metabolic pathways showed that the differentially expressed metabolites were mainly related to amino acid metabolism, nucleotide metabolism, fatty acid degradation, and the tricarboxylic acid cycle (TCA). Under the stress of FA and p-CA, the metabolism of some amino acids was blocked, which disturbed the redox balance in the cells and destroyed the synthesis of most proteins, which was the main reason for the inhibition of yeast cell growth. This study provided a strong scientific reference to improve the durability of S. cerevisiae against hydroxycinnamic acid inhibitors.

Key points:

• Morphological changes of S. cerevisiae cells under inhibitors stress were observed.• Changes of the metabolites in S. cerevisiae cells were explored by metabolomics.• One of the inhibitory effects on yeast is due to changes in the metabolic network.

News (Medical) associated with Sodium Fusidate

06 Feb 2023

·www.globenewswire.com

Aceragen Announces Recommendation of Data Monitoring Committee in Terra Study

DURHAM, N.C. and EXTON, Pa., Feb. 06, 2023 (GLOBE NEWSWIRE) -- Aceragen, Inc. (Nasdaq: ACGN) a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases, today announced the recommendation of its independent Data Monitoring Committee (DMC) in regard to the on-going TERRA Phase 2 study of ACG-701 in patients with melioidosis. The DMC is responsible for providing oversight of safety and efficacy for this study and has recommended the TERRA study continue without modification. The DMC review was a planned meeting held in order to monitor the safety of the seriously ill subjects participating in the TERRA study. “This safety review represents an important milestone for ACG-701, as it allows the TERRA study to continue to evaluate ACG-701 in these hospitalized melioidosis patients. We are very pleased that the DMC recommended that the trial continue. We look forward to delivering the data from the study later this year,” stated Carl Kraus, M.D., Aceragen’s Chief Medical Officer. ACG-701 for MelioidosisAceragen has executed an ~$50 million development partnership with the Department of Defense’s Defense Threat Reduction Agency (“DTRA”) to investigate ACG-701 as a potential medical countermeasure for melioidosis, a life-threatening infection caused by the B. pseudomallei pathogen. This program is centered around a Phase 2 trial, the TERRA study (NCT05105035), which is a randomized double-blind, placebo-controlled trial conducted in hospitalized melioidosis patients. TERRA was initiated in May 2022 and continues to actively enroll patients with a data read-out expected in the fourth quarter of 2023. About Aceragen, Inc. Aceragen is a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases. Our lead product candidate, ACG-701, is an oral, loading dose formulation of sodium fusidate that is in development for the treatment of melioidosis and acute pulmonary exacerbations associated with cystic fibrosis. ACG-701 has received Fast Track Designation, Orphan Drug Designation, and Qualified Infectious Disease Product (QIDP) from the FDA in acute exacerbations with cystic fibrosis. Aceragen is also developing ACG-801, recombinant human acid ceramidase, as an enzyme replacement therapy for an untreated lysosomal storage disorder called Farber disease. To learn more about us and our programs, please visit Aceragen.com. Forward-Looking StatementsThis press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements, other than statements of historical fact, included or incorporated in this press release, including, without limitation, statements regarding the Company's new development opportunities, clinical trials and studies, product designation and/or status, financial position, funding for continued operations, cash reserves, projected costs, prospects, clinical trials, plans, expectations, strategies, projections and objectives of management, are forward-looking statements. The words "believes," "anticipates," "estimates," "plans," "expects," "intends," "may," "could," "should," "potential," "likely," "projects," "continue," "will," “schedule,” and "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements are predictions based on our current expectations and projections about future events and various assumptions. We cannot guarantee that we will achieve the plans, intentions, or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. These forward-looking statements involve known and unknown risks, uncertainties, and other factors, which may be beyond our control, and which may cause our actual results, performance, or achievements to differ materially from future results, performance, or achievements expressed or implied by such forward-looking statements. There are a number of important factors that could cause our actual results to differ materially from those indicated or implied by its forward-looking statements including, without limitation: whether we will be able to successfully integrate the acquired operations; whether our cash resources will be sufficient to fund continuing operations; and newly acquired operations; whether our products will advance into or through the clinical trial process when anticipated or at all or warrant submission for regulatory approval; whether such products will receive approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies; whether our products receive approval, they will be successfully distributed and marketed; and whether our collaborations will be successful. All forward-looking statements included in this press release are made as of the date hereof and are expressly qualified in their entirety by this cautionary notice, including, without limitation, those risks and uncertainties described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, and otherwise in the Company’s subsequent filings and reports filed with Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements and it disclaims any intention or obligation to update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise, except as may be required by law. Please direct questions to: John KirbyChief Financial OfficerAceragen, Inc.

Fast TrackPhase 2Orphan Drug

25 Jan 2023

·www.biospace.com

Methicillin-Resistant Staphylococcus aureus Infection Pipeline Clinical Trials Featuring 25+ companies

New York, USA, Jan. 25, 2023 (GLOBE NEWSWIRE) -- Methicillin-Resistant Staphylococcus aureus Infection Pipeline Clinical Trials Featuring 25+ companies| DelveInsight Methicillin-resistant Staphylococcus aureus (MRSA) is an infection caused by antibiotic-resistant strains of the pathogen Staphylococcus aureus. Rising incidence of Methicillin-resistant Staphylococcus aureus (MRSA) infections is creating health concerns among the population. Manufacturers are focusing on product development and launches for the treatment of Methicillin-resistant Staphylococcus aureus (MRSA). DelveInsight’s 'Methicillin-Resistant Staphylococcus aureus Infection Pipeline Insight 2023' report provides comprehensive global coverage of available, marketed, and pipeline methicillin-resistant Staphylococcusaureus infection therapies in various stages of clinical development. Major pharmaceutical companies are working to advance the pipeline space and future growth potential of the methicillin-resistant Staphylococcusaureus infection pipeline domain. Key Takeaways from the Methicillin-Resistant Staphylococcus aureus Infection Pipeline Report DelveInsight’s methicillin-resistant Staphylococcusaureus infection pipeline report depicts a robust space with 25+ companies and 28+ pipeline drugs for methicillin-resistant Staphylococcusaureus infection treatment. Key methicillin-resistant Staphylococcusaureus infection companies include Trellis Bioscience, LegoChem Biosciences, Aptorum Group, ContraFect, Crystalgenomics, Cellics Therapeutics, MicuRx, Oxford Antibiotic Group, Destiny Pharma, TSRL, Inc., TAXIS Pharmaceuticals, Basilea Pharmaceutica, Helperby Therapeutics, Akagera Medicines, Histogen, Alphamab Co. Ltd, Biocidium Pharmaceuticals, and others are evaluating novel methicillin-resistant Staphylococcusaureus infection drugs candidate to improve the treatment landscape. Promising methicillin-resistant Staphylococcusaureus infection pipeline therapies in various stages of development include TRL1068, Delpazolid, ALS 4, Teicoplanin inhalation, CG 400549, SAAP 148, Exebacase, Marinus, CTI 005, Iclaprim, MRX 4, MRX 7, XF-73, TXA709, ARV-1801, ACX-375C, ABX-605, IBT-V02, BCM-0184, Contezolid, TetramOX, Ceftobiprole, HY-004B8b, HY-005B8a, CG-549, Emricasan, KN043, and several others. Ceftobiprole medocaril, the prodrug of the active moiety ceftobiprole, is a cephalosporin antibiotic for intravenous administration, with rapid bactericidal activity against a wide range of Gram-positive and Gram-negative bacteria. This includes methicillin-susceptible and resistant Staphylococcus aureus (MSSA, MRSA) and susceptible Pseudomonas spp. In October 2022, Basilea Pharmaceutica announced the results from Phase III ERADICATE study, evaluating ceftobiprole in the treatment of adult patients with bacterial bloodstream infections caused by Staphylococcus aureus. Results for the primary efficacy outcomes were consistent in key subgroups including in patients with MSSA or MRSA bloodstream infections at baseline, and in various categories of underlying conditions such as skin and skin structure infections, abdominal abscesses, chronic dialysis, septic arthritis, osteomyelitis, definite right-sided infective endocarditis and in patients with persistent SAB. Basilea also planned to submit a New Drug Application (NDA) for ceftobiprole to the US Food and Drug Administration (FDA) around year-end 2022. Exebacase (CF-301) being developed by ContraFect Corporation is a lysin with potent activity against Staphylococcus aureus ("Staph aureus"). Exebacase has the potential to be the first-in-class of a new treatment for patients with Staph aureus bacteremia. The FDA has granted Breakthrough Therapy designation to exebacase for the treatment of MRSA bloodstream infections (bacteremia), including right-sided endocarditis, when used in addition to SOC antistaphylococcal antibiotics in adult patients, based on the final data from the Phase II superiority trial of exebacase. In July 2022, Destiny Pharma plc announced that it had received an award from the Cystic Fibrosis Foundation. The research project will establish the potential of the Company’s proprietary XF-73 drug as a novel treatment for cystic fibrosis patients infected with methicillin-resistant Staphylococcus aureus (MRSA). CTI-005 is for the treatment of Methicillin Resistance Staphylococcus Aureus (MRSA) Pneumonia and has been granted IND allowance from the US FDA. The potent RBC-NS has the membrane lipids and receptors that a normal red blood cell has, however, the high surface-volume ratio of the RBC-NS allows it to act as a decoy. This characteristic allows the RBC-NS to bind and neutralize toxins, auto-antibodies, and bacterial infections. The pore-forming toxins emitted by MRSA can be effectively absorbed and neutralized by RBC-NS. Request a sample and discover the recent advances in methicillin-resistant Staphylococcusaureus infection treatment drugs @ Methicillin-Resistant Staphylococcus aureus Infection Pipeline Report The methicillin-resistant Staphylococcusaureus infection pipeline report provides detailed profiles of pipeline assets, a comparative analysis of clinical and non-clinical stage methicillin-resistant Staphylococcusaureus infection products, inactive and dormant assets, a comprehensive assessment of driving and restraining factors, and an assessment of opportunities and risks in the methicillin-resistant Staphylococcusaureus infection pipeline landscape. Methicillin-Resistant Staphylococcus aureus Infection Overview Methicillin-resistant Staphylococcus aureus (MRSA) infections are caused by bacteria that have attained resistance from several antibiotics that treat diseases caused by Staphylococcus bacteria. The common patients of Methicillin-resistant Staphylococcus aureus (MRSA) infections are patients that have been in hospitals or healthcare settings for a long duration where the bacteria is found commonly. MRSA infections are known as healthcare-associated MRSA (HA-MRSA) if infected in hospitals. Such infections can spread to healthcare workers through invasive procedures, which are further transmitted to several patients by touching and operating with bare hands. Other MRSA infections occur in a considerable population of healthy people. People associated with community-associated MRSA (CA-MRSA) show symptoms appearing as painful skin boils. It further spreads through physical contact between individuals. The population at higher risk includes people surrounded by a significant number of people and childcare workers. MRSA infections might look like red bumps, pimples, or spider bites, accompanied by pus and fever. Find out more about methicillin-resistant Staphylococcusaureus infection treatment drugs @ Drugs for Methicillin-Resistant Staphylococcus aureus Infection Treatment A snapshot of the Methicillin-Resistant Staphylococcus aureus Infection pipeline drugs mentioned in the report: Drug Company Phase MoA RoA Ceftobiprole Basilea Pharmaceutica Phase III Cell wall inhibitors; Peptidyltransferase inhibitors Intravenous CG 549 CrystalGenomics Phase II Enoyl-ACP reductase inhibitors; Fatty acid synthase expression inhibitors; Fatty acid synthetase complex inhibitors Oral BCM-0184 Biocidium Pharmaceuticals Phase I NA Oral/Topical TRL1068 Trellis Bioscience Phase I Bacterial biofilm inhibitors Intravenous ALS 4 Aptorum Group Phase I Bacterial virulence inhibitors Oral EVX-B1 Evaxion Biotech Preclinical Immunostimulants NA Emricasan Histogen Preclinical Apoptosis inhibitors; Caspase inhibitors NA TarO inhibitor Prokaryotics Discovery Cell membrane inhibitors; Cell wall inhibitors NA Learn more about the emerging methicillin-resistant Staphylococcusaureus infection pipeline therapies @ Methicillin-Resistant Staphylococcus aureus Infection Clinical Trials Methicillin-Resistant Staphylococcus aureus Infection Therapeutics Assessment The methicillin-resistant Staphylococcus aureus infection pipeline report proffers an integral view of methicillin-resistant Staphylococcusaureus infection emerging novel therapies segmented by stage, product type, molecule type, mechanism of action, and route of administration. Scope of the Methicillin-Resistant Staphylococcus aureus Infection Pipeline Report Coverage: Global Therapeutic Assessment By Product Type: Mono, Combination, Mono/Combination Therapeutic Assessment By Clinical Stages: Pre-clinical, Discovery, Discontinued, Inactive, Phase I, Phase II, Phase III Therapeutics AssessmentBy Route of Administration: Oral, Parenteral, Intravenous, Subcutaneous, Topical. Therapeutics AssessmentBy Molecule Type: Monoclonal Antibody, Peptides, Polymer, Small molecule, Gene therapy Therapeutics AssessmentBy Mechanism of Action: Cell wall inhibitors; Peptidyltransferase inhibitors, Enoyl-ACP reductase inhibitors; Fatty acid synthase expression inhibitors; Fatty acid synthetase complex inhibitors, Bacterial biofilm inhibitors, Bacterial virulence inhibitors, Immunostimulants, Apoptosis inhibitors; Caspase inhibitors, Cell membrane inhibitors; Cell wall inhibitors Key Methicillin-Resistant Staphylococcus aureus Infection Companies: Trellis Bioscience, LegoChem Biosciences, Aptorum Group, ContraFect, Crystalgenomics, Cellics Therapeutics, MicuRx, Oxford Antibiotic Group, Destiny Pharma, TSRL, Inc., TAXIS Pharmaceuticals, Basilea Pharmaceutica, Helperby Therapeutics, Akagera Medicines, Histogen, Alphamab Co. Ltd, Biocidium Pharmaceuticals, and others. Key Methicillin-Resistant Staphylococcus aureus Infection Pipeline Therapies: TRL1068, Delpazolid, ALS 4, Teicoplanin inhalation, CG 400549, SAAP 148, Exebacase, Marinus, CTI 005, Iclaprim, MRX 4, MRX 7, XF-73, TXA709, ARV-1801, ACX-375C, ABX-605, IBT-V02, BCM-0184, Contezolid, TetramOX, Ceftobiprole, HY-004B8b, HY-005B8a, CG-549, Emricasan, KN043, and others. Dive deep into rich insights for new drugs for methicillin-resistant Staphylococcusaureus infection treatment; visit @ Methicillin-Resistant Staphylococcus aureus Infection Drugs Table of Contents 1. Methicillin-Resistant Staphylococcus aureus Infection Pipeline Report Introduction 2. Methicillin-Resistant Staphylococcus aureus Infection Pipeline Report Executive Summary 3. Methicillin-Resistant Staphylococcus aureus Infection Pipeline: Overview 4. Analytical Perspective In-depth Commercial Assessment 5. Methicillin-Resistant Staphylococcus aureus Infection Pipeline Therapeutics 6. Methicillin-Resistant Staphylococcus aureus Infection Pipeline: Late Stage Products (Pre-registration) 7. Methicillin-Resistant Staphylococcus aureus Infection Pipeline: Late Stage Products (Phase III) 7.1 Ceftobiprole: Basilea Pharmaceutica 8. Methicillin-Resistant Staphylococcus aureus Infection Pipeline: Mid Stage Products (Phase II) 8.1 CG549: CrystalGenomics 9. Methicillin-Resistant Staphylococcus aureus Infection Pipeline: Early Stage Products (Phase I) 9.1 TRL1068: Trellis Bioscience 10. Methicillin-Resistant Staphylococcus aureus Infection Pipeline Therapeutics Assessment 11. Inactive Products in the Methicillin-Resistant Staphylococcus aureus Infection Pipeline 12. Company-University Collaborations (Licensing/Partnering) Analysis 13. Key Companies 14. Key Products in the Methicillin-Resistant Staphylococcus aureus Infection Pipeline 15. Unmet Needs 16. Market Drivers and Barriers 17. Future Perspectives and Conclusion 18. Analyst Views 19. Appendix For further information on the methicillin-resistant Staphylococcusaureus infection pipeline therapeutics, reach out @ Methicillin-Resistant Staphylococcus aureus Infection Treatment Drugs Related Reports Methicillin-Resistant Staphylococcus aureus Epidemiology Forecast Methicillin-Resistant Staphylococcus aureus Epidemiology Forecast – 2032 report delivers an in-depth understanding of the disease, historical and forecasted epidemiology, and the methicillin-resistant Staphylococcusaureus epidemiology trends. Methicillin-Resistant Staphylococcus aureus Infection Market Methicillin-Resistant Staphylococcus aureus Market Insights, Epidemiology, and Market Forecast – 2032 report delivers an in-depth understanding of the market trends, market drivers, market barriers, and key methicillin-resistant Staphylococcusaureus infection companies, including Trellis Bioscience, LegoChem Biosciences, Aptorum Group, Allergan, iNtRON Biotechnology, Neupharma, ImmuPharma, Lytix Biopharma, CrystalGenomics, AIMM Therapeutics, among others. Staphylococcus aureus Bacteremia Pipeline Staphylococcus aureus Bacteremia Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug pro including clinical and non-clinical stage products, and the key Staphylococcus aureus Bacteremia companies, including ContraFect, Armata Pharmaceuticals, iNtRON Biotechnology, Durata Therapeutics, Cipher Pharmaceuticals, XBiotech, Basilea Pharmaceutica, Genentech, among others. Hospital Acquired Methicillin-Resistant Staphylococcus aureus Infections Pipeline Hospital Acquired Methicillin-Resistant Staphylococcus aureus Infections Pipeline Insight – 2023 report provides comprehensive insights about the pipeline landscape, pipeline drug pro including clinical and non-clinical stage products, and the key Hospital Acquired Methicillin-Resistant Staphylococcus aureus Infections companies, including Trellis Bioscience, LegoChem Biosciences, Aptorum Group, Allergan, iNtRON Biotechnology, Neupharma, ImmuPharma, Lytix Biopharma, CrystalGenomics, AIMM Therapeutics, among others. 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Connect with us atLinkedIn Contact Us Shruti Thakur info@delveinsight.com +1(919)321-6187

Phase 3Phase 2Clinical Result

17 Jan 2023

·www.biospace.com

Idera Pharmaceuticals Announces Name Change to Aceragen, Inc. and Provides Near-Term Strategic Outlook

Company completes preferred stock conversion, implements reverse stock split, and regains Nasdaq compliance Company positioned for success with late-stage rare disease portfolio and multiple clinical milestones in 2023 DURHAM, N.C. and EXTON, Pa., Jan. 17, 2023 (GLOBE NEWSWIRE) -- Idera Pharmaceuticals, Inc. (“Idera”) (Nasdaq: IDRA), a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases, today announced the Company has changed its name and symbol to Aceragen, Inc. (“Aceragen,” the “Company,” “we,” “us,” or “our”) and “ACGN”. Additionally, the Company’s stockholders approved the conversion into common stock of the Series Z preferred shares resulting from the previously announced merger with Aceragen and authorized a reverse split of common stock. The Company’s Board of Directors has approved the reverse stock split at a ratio of 1-for-17 shares. As a result of these changes, which will be effective upon the market open on Wednesday, January 18, 2023, the Company will be in compliance with all applicable Nasdaq listing standards. Nasdaq has issued an approval letter confirming Aceragen’s listing. “We are excited to have completed the transformation of Aceragen via our merger with Idera and the subsequent adjustments to our stock and Nasdaq listing. We believe this transition strengthens our portfolio of late-stage clinical assets in cystic fibrosis and Farber disease and aligns with our goal of delivering important therapies for people living with rare diseases,” stated John Taylor, Aceragen’s Chief Executive Officer. “During the course of this year, we anticipate the achievement of significant clinical milestones that include two Phase 2 data read-outs for ACG-701, as well as lifting of the clinical hold and advancing toward the initiation of our Phase 2/3 trial in Farber disease for ACG-801.” “With the positive result of the stockholder vote behind us, I share in John’s excitement and optimism for Aceragen and look forward to the progress that this team will make for patients in need,” added Vincent J. Milano, Chair of Aceragen’s Board of Directors. Clinical Development Overview Aceragen has a portfolio of late-stage clinical assets in cystic fibrosis and Farber disease with clinical milestones anticipated in 2023. ACG-701 for Acute Pulmonary Exacerbations in Cystic Fibrosis ACG-701 is a proprietary oral, loading dose formulation of sodium fusidate being developed as a treatment for acute pulmonary exacerbations (“PEx”) associated with cystic fibrosis (“CF”), a major factor driving lung function decline in people living with CF. Sodium fusidate has an established clinical efficacy and safety pro more than 50 years of use in other countries, including as part of CF PEx treatment guidelines in the United Kingdom and Australia. Despite this, the compound has never been approved by the FDA and represents a new and potentially powerful approach in the United States to address the infection, inflammation, and enhanced mucin expression that are hallmark features of CF PEx. A Phase 2 trial of ACG-701 in CF PEx (the REPRIEVE study), a randomized double-blinded, placebo-controlled study, was initiated in December 2022 at clinical sites in the United States in collaboration with the CF Foundation’s Therapeutic Development Network (TDN). The CF Foundation has also provided funding of $3.5 million in support of the study. If approved, ACG-701 would represent the first product in the United States indicated for the treatment of newly diagnosed CF PEx patients. Data from the REPRIEVE study is expected in 2H 2023. The FDA has granted Orphan Drug Designation, Fast Track and Qualified Infectious Disease Product (QIDP) status to ACG-701 for CF PEx. ACG-701 for Melioidosis Aceragen has also executed an ~$50 million development partnership with the Department of Defense’s Defense Threat Reduction Agency (“DTRA”) to investigate ACG-701 as a potential medical countermeasure for melioidosis, a life-threatening infection caused by the B. pseudomallei pathogen. This program is centered around a Phase 2 trial, the TERRA study (NCT05105035), which is a randomized double-blind, placebo-controlled trial conducted in hospitalized melioidosis patients. TERRA was initiated in May 2022 and continues to actively enroll patients with an independent DMC review planned during Q1 2023 and a data read-out expected in 2H 2023. ACG-801 for Farber Disease ACG-801, recombinant human acid ceramidase, is an investigational biologic in development to be the first-ever enzyme replacement therapy for the treatment of Farber disease, a progressive, severe, and life-threatening lysosomal storage disorder that is caused by the monogenic deficiency of acid ceramidase. The biochemical hallmark of Farber disease is the loss of acid ceramidase enzyme activity leading to abnormal accumulation of ceramide, profound macrophage-driven inflammation and multi-organ disease affecting bone and joints, cartilage, the immune system, central nervous system, and the lungs. Complications of the disease are life threatening, with many patients dying in the first years of life. There are no ceramide-targeted medications currently available that can alter the disease natural history. The Company expects to initiate the ADVANCE clinical study for ACG-801 in Farber disease, a randomized, double-blind, placebo-controlled, first-in-human study, in the first quarter of 2024 with data expected in the first quarter of 2025. Due to the ultra-rare nature of Farber disease, if successful, this study has the potential to support registration of the product. The FDA has granted Orphan Drug, Fast Track, and Rare Pediatric Disease designations for ACG-801. Rare pediatric disease designation permits priority review voucher eligibility, upon FDA marketing authorization. Clinical Milestone Summary ACG-701 - REPRIEVE study in CF PEx, data expected 2H 2023 ACG-701 – TERRA study in melioidosis, data expected 2H 2023 ACG-801 – ADVANCE study in Farber disease, initiation expected in Q1 2024 Projected cash available is expected to provide the Company with capital runway into Q3’2023. About Aceragen, Inc. Aceragen is a clinical-stage biopharmaceutical company committed to transforming the care of people living with rare pulmonary and rheumatic diseases. Our portfolio includes late-stage programs based on well-established biological principles that we are developing to be innovative therapeutics capable of addressing the unmet medical needs of individuals living with rare diseases. To learn more about us and our programs, please visit Aceragen.com. Forward-Looking Statements This press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933, as amended, and Section 21E of the Securities Exchange Act of 1934, as amended. All statements, other than statements of historical fact, included or incorporated in this press release, including, without limitation, statements regarding the Company's new development opportunities, clinical trials and studies, product designation and/or status, financial position, funding for continued operations, cash reserves, projected costs, prospects, clinical trials, plans, expectations, strategies, projections and objectives of management, are forward-looking statements. The words "believes," "anticipates," "estimates," "plans," "expects," "intends," "may," "could," "should," "potential," "likely," "projects," "continue," "will," “schedule,” and "would" and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. These forward-looking statements are predictions based on our current expectations and projections about future events and various assumptions. We cannot guarantee that we will achieve the plans, intentions, or expectations disclosed in our forward-looking statements and you should not place undue reliance on our forward-looking statements. These forward-looking statements involve known and unknown risks, uncertainties, and other factors, which may be beyond our control, and which may cause our actual results, performance, or achievements to differ materially from future results, performance, or achievements expressed or implied by such forward-looking statements. There are a number of important factors that could cause our actual results to differ materially from those indicated or implied by its forward-looking statements including, without limitation: whether we will be able to successfully integrate the acquired operations; whether our cash resources will be sufficient to fund continuing operations; and newly acquired operations,; whether our products will advance into or through the clinical trial process when anticipated or at all or warrant submission for regulatory approval; whether such products will receive approval from the U.S. Food and Drug Administration or equivalent foreign regulatory agencies; whether our products receive approval, they will be successfully distributed and marketed; and whether our collaborations will be successful. All forward-looking statements included in this press release are made as of the date hereof and are expressly qualified in their entirety by this cautionary notice, including, without limitation, those risks and uncertainties described in the Company’s Annual Report on Form 10-K for the year ended December 31, 2021, and otherwise in the Company’s subsequent filings and reports filed with Securities and Exchange Commission. The Company does not assume any obligation to update any forward-looking statements and it disclaims any intention or obligation to update or revise any forward-looking statement, whether as a result of new information, future events, or otherwise, except as may be required by law. Please direct questions to: John Kirby Chief Financial Officer Aceragen, Inc. jkirby@aceragen.com

Fast TrackOrphan DrugPhase 2

100 Deals associated with Sodium Fusidate

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KEGG	Wiki	ATC	Drug Bank
-	-	D09AA02 S01AA13 J01XC01	-

R&D Status

Approved

10 top approved records.

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Indication	Country/Location	Organization	Date
Bacterial Infections	CA	Leo Pharma, Inc.	01 Jan 1984
Secondary infection	JP	Alfresa Pharma Corp.	22 Apr 1972

Developing

10 top R&D records.

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Indication	Highest Phase	Country/Location	Organization	Date
Bone and joint infections	Phase 3	US	Arrevus, Inc.Startup	01 Dec 2015
Staphylococcal Infections	Phase 3	US	Arrevus, Inc.Startup	01 Dec 2015
Skin and skin structure infections	Phase 3	US	Arrevus, Inc.Startup	01 Nov 2015
Skin and skin structure infections	Phase 3	PR	Arrevus, Inc.Startup	01 Nov 2015
Pulmonary Cystic Fibrosis	Phase 2	US	Aceragen, Inc.	10 Feb 2023
Melioidosis	Phase 2	TH	Arrevus, Inc.Startup	14 Jun 2022
Prosthetic joint infection	Phase 2	US	Arrevus, Inc.Startup	01 Dec 2012
Skin Diseases, Bacterial	Phase 2	US	Arrevus, Inc.Startup	01 Aug 2009

Clinical Result

Indication

Phase

Evaluation

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Study	Phase	Population	Analyzed Enrollment	Group	Results	Evaluation	Publication Date


NCT02569541 (CTgov)	Phase 2/3	Bone and joint infections \| Staphylococcal Infections	30	sodium fusidate	hcsejcpigu(secgormeiv) = eoxcwfzgrz zqbvxxfjdi (vdorwhetju, pkfmtcsjjx - whcngvzdzl) View more	-	30 Jan 2020
NCT02570490 (GlobeNewswire) Manual	Phase 3	Skin and skin structure infections	716	Fusidic acid	sqfcjwlggv(bkgisirebf) = bqhrbjjshr iplebahnsa (lcpbmnvrxp ) View more	Non-inferior	24 Feb 2017
				linezolid	sqfcjwlggv(bkgisirebf) = srsqdwltov iplebahnsa (lcpbmnvrxp ) View more
NCT00986856 (CTgov)	Phase 4	Impetigo	58	Fucidin® cream (Fucidin® Cream)	cdkufnnbjv(vrgtkkqsyq): Odds Ratio (OR) = 13.2 (95% CI, 1.4 - 120.4), P-Value = <0.01 View more	-	29 Sep 2010
				Fucidin® cream (Fucidin® Cream Vehicle)

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