FPR1

ADS051 is the first and only MRP2 / FPR1 inhibitor to demonstrate favorable safety and tolerability with signals of clinical efficacy with relief from disruptive symptoms of inflammatory bowel disease in a multiple ascending dose (MAD) study of moderately to severely active Ulcerative Colitis (UC) patients in relapse ADS051 was demonstrated to be safe in a single ascending dose (SAD) study in healthy volunteers across five dosing cohorts CONCORD, Mass., March 6, 2025 /PRNewswire/ -- Adiso Therapeutics, Inc. announced the publication of the results of two Phase 1 studies, which showed that healthy subjects and ulcerative colitis (UC) patients given oral doses of ADS051, a novel modulator of neutrophils being developed for Inflammatory Bowel Disease (IBD), could be safely administered and well-tolerated in IBD patients. These studies showed consistent safety and tolerability of ADS051 with highly encouraging signals of clinical response as determined by Mayo Endoscopic Scoring blinded to the investigator. Data from the two trials has been published in the American Journal of Gastroenterology, the official journal of the American College of Gastroenterology (ACG). The first of the two publications presents the results from the single ascending dose (SAD) trial of ADS051. This trial was the intensive first in human study of ADS051 across five (5) dosing cohorts of healthy subjects and included a deep-reaching pharmacokinetic analysis of this first and only modulator of neutrophils. There were no dose-limiting toxicities, serious adverse events (AEs), or trial discontinuation due to AEs. Pharmacokinetic data demonstrated minimal systemic absorption of ADS051, with the majority of drug excreted in the stool as expected. There was no clinical evidence of adverse immunosuppressive activity. The second publication presents the results of a multiple ascending dose (MAD) study of ADS051 in patients with moderately to severely active UC. The MAD study included three (3) placebo controlled dosing cohorts, measuring signals of clinical response using assessments typical for later stage studies, such as Mayo Endoscopic Scoring which included Clinical Remission (CR), Endoscopic Improvement (EI), and Endoscopic Response (ER). The study also looked at neutrophil-related markers of inflammation and histologic assessment. As detailed in the publication, this study achieved its primary endpoint of safety and tolerability with encouraging signs of efficacy across multiple outcome measures. "The SAD publication documents the successful completion of a very important first step in establishing patient safety. The completion of the study provided an understanding and foundation for safe dosing of ADS051 prior to exposure in patients with IBD," said Adam Cheifetz, M.D., Director, Center for Inflammatory Bowel Disease, Beth Israel Lahey Health, Beth Israel Deaconess Medical Center, Harvard Medical School Teaching Hospital. "UC is an inflammatory disease with complex underlying biology. We are now amid a rapidly expanding therapeutic landscape of treatment options for individuals with IBD, yet ADS051 is different and represents a unique therapeutic approach of neutrophil modulation," said Jessica Allegretti, MD, MPH., Crohn's and Colitis Center, Associate Professor of Medicine, Harvard Medical School, Gastroenterology, Hepatology and Endoscopy, Internal Medicine, Brigham and Women's Hospital. "Participants in the multiple ascending dose (MAD) trial with moderately to severely active UC were repeatedly dosed with ADS051. Highly favorable signals of pharmacologic activity and clinical benefit were seen over a relatively short 28-day period. These Phase 1b MAD data indicate that ADS051 may represent an important therapeutic advancement as a safe and effective, once-daily oral therapy for patients with UC." ADS051 is a novel, oral, gut-restricted, small molecule consisting of an active moiety or warhead, covalently linked to a 2,000 molecular weight polyethylene glycol (PEG). ADS051 inhibits two neutrophil-related receptors, multidrug resistant protein 2 (MRP2) which activates hepoxilin A3 (HXA3) neutrophil recruitment or transmigration and formyl peptide receptor 1 (FPR1), a known GPCR found on the surface of neutrophils, important for activation and signaling recruitment. Due to the design of ADS051, it largely remains in the colonic lumen, avoiding wide-spread systemic exposure and subsequent T-cell activation or general immunosuppression. Unlike currently available therapies, ADS051 is intended to positively affect neutrophil-mediated tissue damage, a hallmark of IBD pathology. The ADS051 data generated from these two studies has been submitted to the United States (U.S.) Food and Drug Administration (FDA) within an end-of-Phase 1 meeting. Adiso is currently planning a Phase 2 trial for the treatment of moderately to severely active UC in adults. Publication Links: Small-Molecule Neutrophil Modulator ADS051 is Safe and Well-Tolerated in a Phase 1 Single Ascending Dose Study (Open Press), Safety, Pharmacokinetics, and Clinical Efficacy of ADS051, a Neutrophil Modulator, in Ulcerative Colitis: Results of a Randomized Phase 1b Trial (Open Press) About ADS051 ADS051 is a novel, oral, gut-restricted, small molecule that blocks multidrug resistant protein 2 (MRP2)/hepoxilin A3 (HXA3) and formyl peptide receptor 1 (FPR1) mediated neutrophil epithelial transmigration and activation in human cell-based systems. Its lack of both systemic exposure and blockage of T cell activation is intended to limit general immunosuppression. Unlike currently available therapies, it addresses neutrophil-mediated tissue damage, a hallmark of UC pathology. In a Phase 1b MAD study in patients with moderate to severe ulcerative colitis, ADS051 demonstrated favorable safety and tolerability administered orally, once daily, in patients with moderate-to-severe UC. PK data demonstrated that ADS051 is gut-restricted, as designed, with minimal systemic drug exposure. ADS051 efficacy measures indicate encouraging signals of pharmacologic activity and clinical benefit versus placebo after 28 days of treatment. The Phase 1b MAD data indicate that ADS051 may be an important therapeutic advancement as a safe and effective, once-daily oral therapy for patients with UC. Adiso is currently pursuing additional investigation of ADS051 in a larger Phase 2 clinical study. About Adiso Adiso is a clinical-stage biopharmaceutical company dedicated to improving the health of patients suffering from debilitating inflammatory diseases. This dedication is epitomized by our lead clinical candidates, ADS051, an oral gut-restricted modulator of neutrophil trafficking and activation via inhibition of MRP2 and FPR1 for the treatment of inflammatory bowel disease; and ADS032, a dual NLRP1/NLRP3 inflammasome inhibitor initially being developed for inflammatory diseases of the lung. Adiso has built these development programs upon a rich history of institutional and academic collaboration, including the University of Massachusetts Chan Medical School, the Hudson Institute of Medical Sciences Centre for Innate Immunity and Infectious Diseases in Australia, the University of Edinburgh Centre for Inflammation Research and the University College Cork, Ireland, the APC Microbiome Institute. For more information, please visit or our LinkedIn page Refer to: Scott Megaffin; [email protected] (media) SOURCE Adiso Therapeutics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Adiso to Utilize Noninvasive RNA Technology Developed by Geneoscopy as a Precision Immunology Measure in ADS051 Phase 2 IBD Trial ST. LOUIS, Mo. & CONCORD, Mass.--(BUSINESS WIRE)-- Geneoscopy, Inc., a life sciences company focused on developing noninvasive diagnostic and precision medicine tools for gastrointestinal health, and Adiso Therapeutics, Inc., a clinical-stage biotechnology company advancing novel medicines to treat inflammatory diseases, today announced a strategic collaboration to further treatment options available for patients with inflammatory bowel disease (IBD). The agreement allows Geneoscopy’s patented RNA biomarker technology to be used in conjunction with ADS051, a first-in-class, oral, gut-restricted, small molecule modulator of neutrophil trafficking and activation being developed by Adiso for the treatment of IBD. This press release features multimedia. View the full release here: “Utilizing our RNA biomarker platform to assess stool samples from IBD patients and categorize response to treatment with ADS051 is an exciting opportunity,” said Andrew Barnell, CEO of Geneoscopy. “Our technology shows significant promise to enhance IBD clinical trials by identifying patients likely to respond to a particular therapeutic and, through noninvasive methods, determining therapeutic effectiveness through the measurement of RNA expression profiles which signal mucosal healing in the colon.” “The addition of noninvasive stool-based RNA technology from Geneoscopy within our Phase 2 trial of ADS051 is a perfect complement to our neutrophil-targeted, gut-restricted, non-systemic therapy,” said Scott Megaffin, CEO of Adiso. “Employing precision immunology within our trial holds the potential to significantly advance our ability to provide IBD patients with a novel and highly differentiated treatment option that targets their specific needs.” As part of the Adiso ADS051 Phase 2 trial in patients with ulcerative colitis, Geneoscopy will prospectively assess stool-based transcriptome changes in patients treated with ADS051 to identify immunological markers that may predict individual response. Beyond its utility in identifying likely responders to therapy, preliminary research has also indicated that Geneoscopy’s RNA biomarker technology could provide a noninvasive method of determining clinical remission that is more accurate than traditional diagnostics such as fecal calprotectin. In subsequent clinical trials, Adiso will leverage the Geneoscopy biomarker panel to employ a precision immunology approach that uses stool-derived eukaryotic RNA (seRNA) to assess mucosal healing and predict patient response to therapy. This collaboration comes at a critical time for the estimated 3 million Americans living with IBD. Despite an increased focus on improving treatment and patient care, effective tools are lacking to inform therapy selection, and significant opportunities exist to advance and refine methods for disease monitoring. The Adiso ADS051 Phase 2 clinical study will be conducted globally and begin recruiting in 2024. About Geneoscopy, Inc. Geneoscopy Inc. is a life sciences company focused on developing diagnostic tests for gastrointestinal health. Leveraging its proprietary, patented stool-derived eukaryotic RNA (seRNA) biomarker platform, Geneoscopy’s mission is to empower patients and providers to transform gastrointestinal health through innovative diagnostics. Beyond colorectal cancer screening, Geneoscopy is developing diagnostic tests for treatment selection and therapy monitoring in other disease areas in partnership with leading universities and biopharmaceutical companies. For more information, visit and follow the company on LinkedIn. About Adiso Adiso is a clinical-stage biopharmaceutical company dedicated to improving the health of patients suffering from debilitating inflammatory diseases. This dedication is epitomized by our lead clinical candidates, which exemplify 'healthruption' (disruption in healthcare and drug development) with novel mechanisms of action and a distinct approach to the treatment of inflammatory diseases. ADS051, an oral, gut-restricted modulator of neutrophil trafficking and activation via inhibition of MRP2 and FPR1 for the treatment of ulcerative colitis; and ADS032, a dual NLRP1/NLRP3 inflammasome inhibitor initially being developed for inflammatory diseases of the lung. Adiso has built development programs upon a rich history of institutional and academic collaboration, including, the University of Massachusetts Chan Medical School, the Hudson Institute of Medical Sciences Centre for Innate Immunity and Infectious Diseases in Australia, the University of Edinburgh Centre for Inflammation Research and the University College Cork, Ireland, the APC Microbiome Institute. For more information, please visit or our LinkedIn page. About ADS051 ADS051 is a novel first-in-class, oral, gut-restricted, small molecule modulator of neutrophil trafficking and activation via MRP2 and FPR1 receptor inhibition. ADS051 is a locally acting targeted therapy that addresses the underlying pathophysiology of disease while sparing the peripheral role of neutrophil function. Unlike currently available therapies, ADS051 addresses neutrophil-mediated tissue damage, a hallmark of UC. UC is a form of IBD that causes inflammation and ulcers in the colon. Neutrophils are a type of white blood cell that plays a key role in the body's immune response. In UC, neutrophils are recruited to the colon in large numbers, where they release a variety of inflammatory mediators that cause tissue damage. ADS051 gut restriction, safety and tolerability after oral dosing has been demonstrated in a healthy subject Phase 1a clinical study. In a healthy volunteer Single Ascending Dose study and in a moderate to severe UC patient Phase 1b Multiple Ascending Dose study, oral doses of ADS051 were safe and well-tolerated and largely restricted to the gut with limited systemic exposure while demonstrating encouraging signals of pharmacologic activity and clinical benefit.

ADS051 was safe and well-tolerated at all doses with no safety signal observed Clinical remission was achieved in 22% of patients after only 28 days on drug CONCORD, Mass., Oct. 22, 2023 /PRNewswire/ -- Adiso Therapeutics, Inc., a clinical-stage biotechnology company committed to creating medicines that treat inflammatory diseases and improve the lives of patients and their families, today announced positive topline data from its Phase 1b multiple ascending dose (MAD) clinical trial evaluating ADS051 (BT051) in patients with moderate-to-severe ulcerative colitis (UC). The data were presented today at the American College of Gastroenterology (ACG) 2023 Annual Scientific Meeting being held October 20-25, in Vancouver, Canada. ADS051 is a first-in-class, oral, gut-restricted, small molecule modulator of neutrophil trafficking and activation via inhibition of MRP2 and FPR1. Unlike currently available therapies, ADS051 addresses neutrophil-mediated tissue damage, a hallmark of UC pathology. The randomized, double-blind, placebo-controlled, MAD Phase 1b study enrolled 24 moderately-to-severely active UC patients, all of whom completed the trial as planned over the 28-day treatment period (NCT05084261). ADS051 was found to be safe and well-tolerated at three dose levels (200, 800, and 3200 mg) administered orally, once daily for 28 days. PK data demonstrate that ADS051 was restricted to the gut with limited systemic exposure. On Day 28, for pooled ADS051 participants vs. pooled placebo participants, clinical remission was achieved in 22.2% vs. 0%, endoscopic improvement (investigator blinded, centrally-read) in 33.3% vs 0%, and endoscopic response (centrally-read) (UCEIS) was achieved in 50% vs. 17%. "We are very pleased with these positive results indicating that ADS051 clearly attained the objectives of this Phase 1b trial and may be an important therapeutic advancement as a safe and effective, once-daily oral therapy for patients with ulcerative colitis," said Scott Megaffin, Chief Executive Officer of Adiso. "ADS051 efficacy measures reveal encouraging signals of pharmacologic activity and clinical benefit versus placebo, after 28 days of treatment. Given these findings and a recently completed positive end-of-Phase 1 FDA interaction, we plan to advance ADS051 in a Phase 2 clinical study in early 2024." "Despite recent therapeutic advancements in the treatment of UC, there still remains a significant unmet need for safe and efficacious therapeutic options," said Jessica Allegretti, M.D., M.P.H Medical Director, Crohn's and Colitis Center, Brigham & Women's Hospital Boston and Associate Professor of Medicine, Harvard Medical School. "The results of this Phase 1b study demonstrate that not only is ADS051 safe and well-tolerated, it also may offer a potentially efficacious solution for patients with moderate-to-severe UC. I look forward to assessing this therapy further in a Phase 2 clinical study." The MAD clinical trial followed a healthy subject Phase 1a single ascending dose (SAD) study which demonstrated ADS051 was gut-restricted with minimal systemic drug exposure, with no dose-limiting toxicities or serious adverse events. One treatment-emergent adverse event was reported in 16.7% of patients in the pooled ADS051 group vs. 66.7% in the pooled placebo group. Safety and tolerability of the 3200-mg dose group were equivalent to the lower dose cohorts with substantially higher fecal concentrations of ADS051. The poster detailing these findings #P0172: ADS051, an Oral, Gut-Restricted, Small Molecule Neutrophil Modulator in Patients With Moderate-To-Severe Ulcerative Colitis: Results From a Multiple Ascending Dose Study, was presented at the ACG 2023 Annual Scientific Meeting, October 22, 2023, and will be available on the Company's website. About ADS051 ADS051 is a first-in-class, oral, gut-restricted, small molecule modulator of neutrophil trafficking and activation via MRP2 and FPR1 receptor inhibition. Unlike currently available therapies, ADS051 addresses neutrophil-mediated tissue damage, a hallmark of UC. UC is a chronic IBD that causes inflammation and ulcers in the colon. Neutrophils are a type of white blood cell that plays a key role in the body's immune response. In UC, neutrophils are recruited to the colon in large numbers, where they release a variety of inflammatory mediators that cause tissue damage. ADS051 gut restriction, safety and tolerability after oral dosing has been demonstrated in a healthy subject Phase 1a clinical study. About Adiso: Adiso is a clinical-stage biopharmaceutical company dedicated to improving the health of patients suffering from debilitating inflammatory diseases. This dedication is epitomized by our lead clinical candidates, which exemplify 'healthruption' (disruption in healthcare and drug development) with novel mechanisms of action and a distinct approach to the treatment of inflammatory diseases. ADS051, an oral, gut-restricted modulator of neutrophil trafficking and activation via inhibition of MRP2 and FPR1 for the treatment of ulcerative colitis; and ADS032, a dual NLRP1/NLRP3 inflammasome inhibitor initially being developed for inflammatory diseases of the lung. Adiso has built these development programs upon a rich history of institutional and academic collaboration, including, the University of Massachusetts Chan Medical School, the Hudson Institute of Medical Sciences Centre for Innate Immunity and Infectious Diseases in Australia, the University of Edinburgh Centre for Inflammation Research and the University College Cork, Ireland, the APC Microbiome Institute. For more information, please visit or our LinkedIn page. Contacts Argot Partners Media: Sarah Sutton/Liza Sullivan IR: Jason Finkelstein [email protected] 212.600.1902 SOURCE Adiso Therapeutics