Pancreatitis

MB-111 is a potential first-in-class in vivo ultracompact CRISPR therapy for Familial Chylomicronemia Syndrome and Severe Hypertriglyceridemia Dr. Brown brings deep expertise in nucleic acid drug development as Mammoth advances its lead program, MB-111, to IND-enabling stage and continues to build a leading company in genetic medicines BRISBANE, CA, USA I May 05, 2025 I Mammoth Biosciences, Inc. , a biotechnology company harnessing its proprietary next-generation CRISPR gene editing platform to create potential one-time curative therapies, today announced the nomination of its first clinical development candidate, MB-111, and the appointment of biotechnology industry veteran, Bob D. Brown, Ph.D., to its Board of Directors. MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. Share MB-111 utilizes CasPhi — an ultracompact CRISPR in vivo gene editing system that is less than half the size of first-generation, Cas9-based systems — encapsulated in a lipid nanoparticle for delivery to the liver after IV administration. MB-111 has the potential to be a first-in-class, one-time treatment for patients with very high triglycerides including familial chylomicronemia syndrome (FCS) and severe hypertriglyceridemia (SHTG). MB-111 is designed to permanently disrupt the expression of the APOC3 gene in the liver, and thus to reduce expression of ApoC-III protein. ApoC-III is a critical driver of lipid metabolism and disrupting its production has been shown to reduce plasma triglycerides in patients with pathologically elevated levels, including those with FCS and SHTG. These patients suffer from recurrent episodes of acute pancreatitis, leading to frequent hospitalizations, as well as an increased risk of cardiovascular disease. Mammoth Biosciences is on track to initiate IND-enabling studies this year. “The nomination of our lead program as a development candidate is a major milestone for Mammoth and the gene editing field, as well as the first proof point of the therapeutic potential of our novel ultracompact CRISPR systems and gene editing technologies,” said Trevor Martin, Ph.D., co-founder and Chief Executive Officer of Mammoth Biosciences. “The preclinical data on MB-111 is compelling, and we’re excited about its potential to be a first-in-class genetic cure for debilitating diseases such as FCS and SHTG. We believe Dr. Brown’s extensive drug discovery and development experience, and track record of building leading companies in siRNA and antisense oligonucleotide therapeutics across rare and chronic diseases will be invaluable to our goal of increasing the number of patients who can derive benefit from Mammoth Biosciences’ genetic medicines.” Dr. Bob D. Brown brings over 30 years of experience in multidisciplinary biotechnology research and drug development, including in the US and ex-US jurisdictions. Most recently, Dr. Brown served as Chief Scientific Officer and Executive Vice President of R&D at Dicerna Pharmaceuticals, an RNAi-focused therapeutics company that was acquired by Novo Nordisk. At Novo, he served as President and Head of the Dicerna Transformation Research Unit and SVP. During his time at Dicerna, he led the discovery and early clinical development of numerous genetic medicines, including nedosiran for the treatment of primary hyperoxaluria, RG6346 for the treatment of chronic hepatitis B, belcesiran for the treatment of alpha-1 antitrypsin deficiency, and several other drug candidates now in the clinical development pipelines of large pharmaceutical companies. Prior to Dicerna, Dr. Brown held various positions at Genta, a clinical-stage antisense oligonucleotide therapeutics company, most recently as its Vice President of Research and Technology. Previously, he was a co-founder and Vice President of R&D of Oasis Biosciences, which was acquired by Gen-Probe. Dr. Brown is an inventor or co-inventor of more than 85 issued US patents, and earned a Ph.D. in molecular biology from the University of California, Berkeley and B.S. degrees in chemistry and biology from the University of Washington, Seattle. “I’m honored to join Mammoth Biosciences at such a pivotal moment, as CRISPR technology stands ready to transform healthcare and redefine the future of medicine,” said Dr. Brown. “I look forward to working with the exceptional Mammoth Biosciences team to harness the power of their groundbreaking platform to tackle some of the most pressing medical challenges and improve patients’ lives. Drawing on my experiences in drug discovery and clinical development, I aim to help advance Mammoth Biosciences’ ultracompact CRISPR gene editing therapies—starting with MB-111—toward clinical application.” About Mammoth Biosciences Mammoth Biosciences is a biotechnology company focused on leveraging its proprietary ultracompact CRISPR systems to develop potential long-term curative therapies for patients with life-threatening and debilitating diseases. Founded by CRISPR pioneer and Nobel laureate Jennifer Doudna and Trevor Martin, Janice Chen, and Lucas Harrington, the company’s ultracompact systems are designed to be more specific and enable in vivo gene editing in difficult to reach tissues utilizing both nuclease applications and new editing modalities beyond double stranded breaks, including base editing, reverse transcriptase editing, and epigenetic editing. The company is building out its wholly owned pipeline of potential in vivo gene editing therapeutics and capabilities and has partnerships with leading pharmaceutical and biotechnology companies to broaden the reach of its innovative and proprietary technology platform. Mammoth Biosciences’ deep science and industry experience, along with a robust and differentiated intellectual property portfolio, have enabled the company to further its mission to transform the lives of patients and deliver on the promise of CRISPR technologies. SOURCE: Mammoth Biosciences

PRINCETON, N.J., May 1, 2025 /PRNewswire/ -- CytoSorbents Corporation (NASDAQ: CTSO), a leader in blood purification therapies for life-threatening conditions in the intensive care unit and cardiac surgery, today provided a regulatory update for DrugSorb™-ATR, its FDA-designated Breakthrough Device. DrugSorb-ATR is designed to reduce the severity of bleeding in patients undergoing coronary artery bypass grafting (CABG) surgery within 2 days of stopping the antiplatelet drug Brilinta® (ticagrelor, AstraZeneca), a commonly used blood thinner. On April 25, 2025, the U.S. Food and Drug Administration (FDA) issued a denial letter regarding the Company's De Novo Request for DrugSorb-ATR, identifying remaining deficiencies that must be addressed before the De Novo Request can be granted, and the device can be authorized for commercialization in the U.S. CytoSorbents has since met with the FDA and will continue to have interactive conversations with the agency to try to resolve the remaining issues. However, if these conversations do not lead to a resolution, a formal appeal will be filed within 60 days. "We remain deeply committed to bringing DrugSorb-ATR to market as an important solution to address the serious, unmet medical need of preventing life-threatening bleeding in CABG patients treated with Brilinta®," said Dr. Phillip Chan, Chief Executive Officer of CytoSorbents. "With no therapies currently available in the U.S., tens of thousands of patients remain at risk every year. We will continue working closely with FDA and believe we can resolve the remaining issues in a timely manner and still anticipate a final regulatory decision in 2025." About CytoSorbents Corporation (NASDAQ: CTSO) CytoSorbents Corporation is a leader in the treatment of life-threatening conditions in the intensive care unit and cardiac surgery through blood purification. CytoSorbents' proprietary blood purification technologies are based on biocompatible, highly porous polymer beads that can actively remove toxic substances from blood and other bodily fluids by pore capture and surface adsorption. Cartridges filled with these beads can be used with standard blood pumps already in the hospital (e.g. dialysis, continuous renal replacement therapy or CRRT, extracorporeal membrane oxygenation or ECMO, and heart-lung machines), where blood is repeatedly recirculated outside the body, through our cartridges where toxic substances are removed, and then back into the body. CytoSorbents' technologies are used in a number of broad applications. Specifically, two important applications are 1) the removal of blood thinners during and after cardiothoracic surgery to reduce the risk of severe bleeding, and 2) the removal of inflammatory agents and toxins in common critical illnesses that can lead to massive inflammation, organ failure and patient death. The breadth of these critical illnesses includes, for example, sepsis, burn injury, trauma, lung injury, liver failure, cytokine release syndrome, and pancreatitis as well as the removal of liver toxins that accumulate in acute liver dysfunction or failure, and the removal of myoglobin in severe rhabdomyolysis that can otherwise lead to renal failure. In these diseases, the risk of death can be extremely high, and there are few, if any, effective treatments. CytoSorbents' lead product, CytoSorb®, is approved in the European Union and distributed in over 70 countries worldwide, with more than 270,000 devices used cumulatively to date. CytoSorb was originally launched in the European Union under CE mark as the first cytokine adsorber. Additional CE mark extensions were granted for bilirubin and myoglobin removal in clinical conditions such as liver disease and trauma, respectively, and for ticagrelor and rivaroxaban removal in cardiothoracic surgery procedures. CytoSorb has also received FDA Emergency Use Authorization in the United States for use in adult critically ill COVID-19 patients with impending or confirmed respiratory failure. CytoSorb is not yet approved or cleared in the United States. In the U.S. and Canada, CytoSorbents is developing the DrugSorb™-ATR antithrombotic removal system, an investigational device based on an equivalent polymer technology to CytoSorb, to reduce the severity of perioperative bleeding in high-risk surgery due to blood thinning drugs. It has received two FDA Breakthrough Device Designations: one for the removal of ticagrelor and another for the removal of the direct oral anticoagulants (DOAC) apixaban and rivaroxaban in a cardiopulmonary bypass circuit during urgent cardiothoracic procedures. In September 2024, the Company submitted a De Novo Request to the U.S. FDA requesting marketing authorization for the DrugSorb-ATR medical device to reduce the severity of perioperative bleeding in CABG patients on the antithrombotic drug ticagrelor, which was accepted for substantive review in October 2024. In November 2024, the Company received its MDSAP certification and submitted its Medical Device License (MDL) application to Health Canada. DrugSorb-ATR has not been authorized for commercialization in the U.S. or Canada. The Company has numerous marketed products and products under development based upon this unique blood purification technology protected by many issued U.S. and international patents and registered trademarks, and multiple patent applications pending, including ECOS-300CY®, CytoSorb-XL™, HemoDefend-RBC™, HemoDefend-BGA™, VetResQ®, K+ontrol™, DrugSorb™, ContrastSorb, and others. For more information, please visit the Company's website at or follow us on Facebook and X. Forward-Looking Statements This press release includes forward-looking statements intended to qualify for the safe harbor from liability established by the Private Securities Litigation Reform Act of 1995. These forward-looking statements include, but are not limited to, statements about our plans, objectives, future targets and outlooks for our business, representations and contentions, and the outcome of our regulatory submissions, and are not historical facts and typically are identified by use of terms such as "may," "should," "could," "expect," "plan," "anticipate," "believe," "estimate," "predict," "potential," "continue" and similar words, although some forward-looking statements are expressed differently. You should be aware that the forward-looking statements in this press release represent management's current judgment and expectations, but our actual results, events and performance could differ materially from those in the forward-looking statements. Factors which could cause or contribute to such differences include, but are not limited to, our restructuring of our direct sales team and strategy in Germany, our ability to resolve remaining deficiencies in the FDA denial letter and/or successfully appeal the FDA's decision, and the risks discussed in our Annual Report on Form 10-K, filed with the SEC on March 31, 2025, as updated by the risks reported in our Quarterly Reports on Form 10-Q, and in the press releases and other communications to shareholders issued by us from time to time which attempt to advise interested parties of the risks and factors which may affect our business. We caution you not to place undue reliance upon any such forward-looking statements. We undertake no obligation to publicly update or revise any forward-looking statements, whether as a result of new information, future events, or otherwise, other than as required under the Federal securities laws. Please Click to Follow Us on Facebook and X U.S. Company Contact: Peter J. Mariani, Chief Financial Officer 305 College Road East Princeton, NJ 08540 [email protected] Investor Relations Contact: Aman Patel, CFA & Adanna G. Alexander, PhD ICR Healthcare [email protected] SOURCE Cytosorbents Corp WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

WALTHAM, Mass., April 29, 2025 /PRNewswire/ -- Alcresta Therapeutics, Inc., a leading commercial-stage company focused on developing and commercializing novel enzyme-based products, today announced that Massachusetts General Hospital (MGH) has enrolled the first patient in an investigator-initiated trial evaluating the efficacy of RELiZORB in managing exocrine pancreatic insufficiency (EPI) in tube-fed pancreatitis patients. "We are excited about the enrollment of the first patient in this study to evaluate the potential benefits of RELiZORB use in tube-fed pancreatitis patients. Acute pancreatitis is one of the leading gastrointestinal causes of hospital admissions, and there is a growing focus on managing long-term complications of pancreatitis, which includes EPI," said Casey Luckhurst, MD, Pancreatic and General Surgeon and Inpatient Director of Massachusetts General Hospital's Pancreatitis Treatment Center. Fat malabsorption is often associated with EPI conditions including pancreatitis, cystic fibrosis, and gastrointestinal cancers and can result in weight loss and serious gastrointestinal symptoms. For patients with fat malabsorption who require tube feeding, RELiZORB is the only FDA-cleared enzyme device designed to mimic the function of pancreatic lipase to improve absorption of fats for tube-fed patients. Studies have demonstrated that RELiZORB is safe, well-tolerated, and effective in improving fat absorption for patients with cystic fibrosis. "The initiation of study recruitment marks an important step toward expanding the body of clinical evidence that supports RELiZORB use in Alcresta's target therapeutic areas," said Michael Yeh, MD, Senior Vice President of Medical Affairs at Alcresta Therapeutics. "This is one of four clinical trials evaluating the safety and efficacy of RELiZORB in malabsorptive conditions supporting our mission to improve nutritional care and outcomes for tube-fed patients living with rare diseases." For more information about RELiZORB clinical trials, visit ClinicalTrials.gov: Pancreatitis: NCT06691893 Short Bowel Syndrome: NCT03530852 and NCT05635747 Critical Care: NCT05710315 About RELiZORB®  (iMMOBILIZED LIPASE) CARTRIDGE RELiZORB is a first-of-its-kind digestive enzyme cartridge designed to mimic the function of pancreatic lipase and is indicated for use in pediatric patients (ages 1 year and above) and adult patients to hydrolyze fats in enteral formula. RELiZORB was developed using Alcresta's proprietary enzyme immobilization technology, iLipase®, which is the digestive enzyme lipase bound to small polymeric bead carriers. RELiZORB connects in-line to enteral feeding systems. As enteral formula passes through RELiZORB, the bound lipase breaks down formula fats into an absorbable form prior to ingestion. RELiZORB was FDA-cleared in 2015 for use in adult patients and was cleared in 2017 for use in children as young as five years old. Use was expanded to pediatric patients as young as two years old in August 2023 and expanded again for patients as young as one year old in January 2025. The next-generation RELiZORB device was introduced to market in May 2024 with broader formula compatibility, an increase in the number of devices used per day and use in both continuous and bolus-feeding set ups. About Alcresta  Therapeutics, Inc. Alcresta Therapeutics, Inc. is dedicated to developing and commercializing novel, enzyme-based products designed to address challenges faced by patients living with gastrointestinal disorders and rare diseases. Alcresta currently markets RELiZORB for enterally-fed patients with pancreatic insufficiency, which occurs in cystic fibrosis, pancreatic cancer, and pancreatitis, and short bowel syndrome, which is marked by significant malabsorption due to limited absorptive area as a result of resection. Alcresta is currently developing a platform application for prematurely born infants treated in the NICU. Alcresta Therapeutics, Inc. is backed by Linden Capital Partners and HealthQuest Capital. More information can be found at . Internal Media Contact: Natalie Bronfin Alcresta Therapeutics, Inc. [email protected] 617-431-3600 SOURCE Alcresta Therapeutics, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED