Acetylcysteine

Study AER-01-002 is using a novel and precision imaging strategy to enroll 100 mucus plug-high chronic obstructive pulmonary disease (COPD) patients AER-01-002 is well powered to determine if 28-days of once daily dosing of fexlamose is safe and improves lung function Fexlamose has been shown in Phase 1 studies of 96 healthy volunteers to be safe, well tolerated and highly differentiated from first generation thiol-based mucolytic drugs RALEIGH, N.C., Jan. 08, 2025 (GLOBE NEWSWIRE) -- Aer Therapeutics (“Aer”), a biopharmaceutical company developing novel inhaled treatments for mucus-associated lung disease, announced today that the first patient has been dosed in its Phase 2a proof-of-concept (“POC”) clinical study of fexlamose (formerly “AER-01”) in Chronic Obstructive Pulmonary Disease (“COPD”). The Phase 2a POC study (“AER-01-002”) is a randomized, double-blind, placebo controlled, parallel group study of fexlamose given once daily for 28 days to patients with COPD. Aer is utilizing a novel approach to identify and treat mucus plug-high COPD patients. Specifically, AER-01-002 will enroll 100 patients with moderate to severe COPD who are mucus plug-high. A mucus plug score is generated by analysis of computed tomography (CT) lung scans using a mucus plug scoring system developed at the University of California, San Francisco. This precision imaging approach to enrich AER-01-002 with mucus plug-high, moderate to severe COPD patients maximizes the sensitivity of the trial to detect the treatment benefits of fexlamose. AER-01-002 was designed and powered to determine if once daily dosing of fexlamose, administered by nebulizer for 28 days, improves lung function as measured by forced expiratory volume in one second (“FEV1"), patient reported outcomes (“PRO”) including the St. George's Respiratory Questionnaire (SGRQ-C), CT mucus plug score, and other secondary and exploratory functional, imaging and PRO endpoints. Both FEV1 and SGRQ-C are primary outcome measures accepted by regulatory agencies such as the United States Food and Drug Administration (“FDA”) and European Medicines Agency (“EMA”) for approval in COPD. AER-01-002 is being conducted in clinical sites in Australia and New Zealand, with plans to expand to the United Kingdom. Top-line data are expected in January 2026. “We are highly encouraged by the rapid start-up of AER-01-002 and enrollment of our first patient in Australia. Our clinical investigators are very excited by the novel approach Aer is taking with fexlamose to potentially improve lung function and symptoms in COPD patients with moderate to severe disease. These patients have a large unmet medical need and targeting mucus plugs represents a rational and differentiated approach to increase their lung function and improve their lung health. We expect to complete AER-01-002 by the end of this year,” said Dr. John Fahy, founder and Chief Clinical Consultant for Aer. About FexlamoseFexlamose is being developed as a chronic treatment for COPD and asthma in conjunction with standard therapies, to improve lung function, lessen symptoms and improve quality of life. Fexlamose may also be developed for the treatment of other muco-obstructive lung diseases, including cystic fibrosis (CF) and non-CF bronchiectasis. Fexlamose previously demonstrated a strong safety and tolerability profile in a Phase 1 Single Ascending Dose and Multiple Ascending Dose clinical studies in 96 healthy volunteers. Adverse events were mild to moderate and self-resolving, and there were no serious adverse events (SAEs). There was no indication of bronchoconstriction and no clinically significant hematology or chemistry abnormalities. Pharmacokinetic data indicate suitability of fexlamose for once daily dosing. Importantly, fexlamose was deemed to be tolerable from a taste and smell profile due to its proprietary chemistry and manufacturing process and its physicochemical properties. This safety and tolerability profile potentially differentiates fexlamose from first generation thiol-based mucolytics such as Mucomyst® (N-Acetylcysteine, Bristol Myers Squibb). Disulfide bridges cross-link mucin polymers to increase their elasticity and propensity to form mucus plugs. Fexlamose is a thiol-modified carbohydrate (“thiol-saccharide”) which cleaves mucin disulfide bridges to liquefy (“lyse”) mucus plugs. Carbohydrate scaffolds are natural and non-toxic, and their polar nature and high aqueous solubility allows them to easily penetrate mucus plugs. Fexlamose is a potent and fast acting mucolytic that is well-suited for pulmonary delivery as liquid inhalation solution by nebulizer or as dry powder. Primary market research indicates that fexlamose sales could exceed $1 billion in the U.S. alone for the treatment of moderate to severe COPD and asthma. About COPDCOPD is a group of lung diseases that include chronic bronchitis and emphysema that are characterized by breathlessness and chronic cough. COPD can be caused by inhaling pollutants, including tobacco smoke or workplace-related fumes, chemicals and dusts. Genetics can also play a causal role in some COPD patients. COPD is one of the leading causes of death in the United States. In 2021, it was the fourth leading cause of death worldwide, responsible for about 5% of all deaths. COPD, which affects millions of patients worldwide, has seen very limited treatment innovation in recent years. The airway component of COPD involves airway smooth muscle tightening, airway inflammation, and airway blockage with mucus plugs. Although several drugs are approved to treat the smooth muscle and inflammation components of COPD, none are approved to treat mucus plugs. The development of treatments for mucus plugs in COPD and other muco-obstructive lung diseases has been slowed by a lack of predictive and monitoring biomarkers for airway mucus plug pathology. AER-01-002 will use CT mucus plug scoring as a novel protocol design element to enrich the trial in patients who are more likely to demonstrate the treatment benefits of fexlamose. About Aer TherapeuticsAer Therapeutics is a clinical stage biopharmaceutical company with a mission to develop novel treatments for mucus-associated lung diseases such as COPD, asthma, cystic fibrosis (CF) and non-CF bronchiectasis. The company leverages the expertise of its scientific founders in airway mucus biology and pathology, medicinal glycochemistry, and inhaled pharmaceutics and the experience of its management team in drug development for lung disease. Aer is supported by a syndicate of premier life science industry investors including Canaan, OrbiMed, and Hatteras Venture Partners. Learn more at: www.aertherapeutics.com Forward-Looking StatementsCertain of the statements made in this press release are forward looking, such as those, among others, relating to Aer’s expectations for fexlamose’s efficacy and safety, clinical trial enrollment goals and the timing of future clinical trials. Actual enrollment results, use of cash and other developments may occur that differ materially from those projected or implied in these forward-looking statements. Factors that may cause such a difference include risks that enrollment goals will not be met, trials may not be commenced or successful or may take longer to complete than anticipated, and projected cash needs and expected financial results may be different. Aer disclaims any intention or obligation to update or revise any forward-looking statements, whether as a result of new information, future events or otherwise. CONTACT Thomas P. MathersExecutive ChairmanAer Therapeutics400 W North Street, Suite 112Raleigh, NC 27603+1 (617) 642-8677tom.mathers@aertherapeutics.com

- New Dosing Regimen Simplifies Administration - NASHVILLE, Tenn., Dec. 9, 2024 /PRNewswire/ -- Cumberland Pharmaceuticals Inc. (Nasdaq: CPIX), a specialty pharmaceutical company focused on delivering high-quality products to improve patient care, announced today the FDA has approved a supplemental New Drug Application (sNDA) for its Acetadote® (N-acetylcysteine for injection) product. Acetadote is an intravenous (IV) formulation of N-acetylcysteine (NAC) indicated to prevent or lessen liver injury after ingestion of potentially toxic quantities of acetaminophen1. Acetaminophen, a common over-the-counter pain reliever and fever reducer, is the leading cause of acute liver failure in the United States. Each year, thousands of individuals experience accidental or intentional acetaminophen poisoning, leading to serious liver damage. The newly approved dosing regimen simplifies the administration of Acetadote by combining the first two bags of the standard regimen into a single, slower infusion. This streamlined approach has been implemented in hospitals across multiple countries and demonstrated to reduce the frequency of medication errors and potentially serious non-allergic anaphylactoid reactions (NAARs) without compromising the effectiveness of Acetadote2-6. By simplifying the dosing regimen, health care providers can administer the life-saving treatment more efficiently, potentially improving patient outcomes. "This FDA approval is a significant step forward in the treatment of acetaminophen overdose," said Rick Dart, MD, PhD, Director at the Rocky Mountain Poison and Drug Center. "By streamlining the administration of NAC, we can improve patient outcomes and reduce the risk of adverse events. This simplified dosing regimen is a valuable tool for health care providers in managing this potentially life-threatening condition." "We are thrilled to announce the FDA approval of this simplified dosing regimen for Acetadote," said A.J. Kazimi, Cumberland's Chief Executive Officer. "This important milestone underscores our commitment to improving patient care and providing innovative solutions for urgent medical needs. By streamlining the administration process, we aim to enhance patient outcomes and reduce the burden on health care providers." Key Highlights: FDA-approved sNDA adds a simplified, IV NAC dosing regimen to the product prescribing information. New IV NAC dosing regimen is both safe and effective. New dosing regimen aims to minimize interruptions in care, medication errors and incidence of dose-related reactions. About Cumberland Pharmaceuticals Cumberland Pharmaceuticals Inc. is the largest biopharmaceutical company founded and headquartered in Tennessee and is focused on providing unique products that improve the quality of patient care. The company develops, acquires, and commercializes products for the hospital acute care, gastroenterology and oncology market segments. The company's portfolio of FDA-approved brands includes: Acetadote® (acetylcysteine) injection, for the treatment of acetaminophen poisoning; Caldolor® (ibuprofen) injection, for the treatment of pain and fever; Kristalose® (lactulose) oral, a prescription laxative, for the treatment of constipation; Sancuso® (granisetron) transdermal, for the prevention of nausea and vomiting in patients receiving certain types of chemotherapy treatment; Vaprisol® (conivaptan) injection, to raise serum sodium levels in hospitalized patients with euvolemic and hypervolemic hyponatremia; and Vibativ® (telavancin) injection, for the treatment of certain serious bacterial infections including hospital-acquired and ventilator-associated bacterial pneumonia, as well as complicated skin and skin structure infections. The company also has a series of Phase II clinical programs underway evaluating its ifetroban product candidate in patients with cardiomyopathy associated with Duchenne Muscular Dystrophy, Systemic Sclerosis and Idiopathic Pulmonary Fibrosis. For more information on Cumberland's approved products, including full prescribing information, please visit the individual product websites, which can be found on the company's website www.cumberlandpharma.com. References: Forward-Looking Statements This press release contains forward-looking statements, which are subject to certain risks and reflect Cumberland's current views on future events based on what it believes are reasonable assumptions. No assurance can be given that these events will occur. Forward-looking statements include, among other things, statements regarding the company's intent, belief or expectations, and can be identified by the use of terminology such as "may," "will," "expect," "believe," "intend," "plan," "estimate," "should," "seek," "anticipate," "look forward" and other comparable terms or the negative thereof. As with any business, all phases of Cumberland's operations are subject to factors outside of its control, and any one or combination of these factors could materially affect Cumberland's operation results. These factors include macroeconomic conditions, including rising interest rates and inflation, competition, an inability of manufacturers to produce Cumberland's products on a timely basis, failure of manufacturers to comply with regulations applicable to pharmaceutical manufacturers, natural disasters, public health epidemics, maintaining an effective sales and marketing infrastructure, and other events beyond the company's control as more fully discussed in its most recent annual report on Form 10-K as filed with the U.S. Securities and Exchange Commission ("SEC"), as well as the company's other filings with the SEC from time to time. There can be no assurance that results anticipated by the company will be realized or that they will have the expected effects. Readers are cautioned not to place undue reliance on forward-looking statements, which speak only as of the date hereof. The company does not undertake any obligation to publicly revise these statements to reflect events after the date hereof. View original content to download multimedia:https://www.prnewswire.com/news-releases/fda-approves-acetadote-snda-302326652.html SOURCE Cumberland Pharmaceuticals Inc. Investor Contact: Shayla Simpson, Cumberland Pharmaceuticals, (615) 255-0068; Media Contact: Molly Aggas, Dalton Agency, (704) 641-6641