Paraplegia

30-Day Data from Endospan's NEXUS TRIOMPHE IDE Trial Demonstrate 63% Reduction in Major Adverse Event (MAE) Rate compared with Reference Performance Goal ATLANTA, May 5, 2025 /PRNewswire/ -- Artivion, Inc. (NYSE: AORT), a leading cardiac and vascular surgery company focused on aortic disease, today announced the presentation of new clinical data from Endospan's NEXUS TRIOMPHE trial at the 105th American Association for Thoracic Surgery (AATS) Annual Meeting in Seattle, Washington. The data presented analyzed clinical outcomes across a 54-patient chronic aortic dissection statistical cohort at 30 days following treatment with NEXUS. Patients enrolled were at high risk for open surgical repair. Dr. Bradley Leshnower, Professor of Surgery and Director of Thoracic Aortic Surgery, Emory University, presented the data from the NEXUS US IDE trial in an abstract titled, Endovascular Aortic Arch Repair Using a Novel Single-Branch Arch Stent Graft for Chronic Dissection: 30-Day Outcomes of the TRIOMPHE FDA Pivotal Study. The data demonstrate statistically significant improvement in clinical outcomes and device technical performance compared with the performance goals set forth in the FDA-approved investigational device exemption (IDE). *Major adverse events (MAEs) = mortality, disabling stroke, permanent paralysis/paraplegia, renal failure, aortic rupture, or development of new dissections in the thoracic aorta or brachiocephalic artery requiring intervention. Dr. Leshnower said, "The 30-day results from the TRIOMPHE study demonstrate that aneurysms due to chronic aortic arch dissection involving the aortic arch can be successfully treated with this novel off-the-shelf stent graft system. The NEXUS device provides an excellent, minimally invasive alternative to open aortic arch replacement in select patients at high risk for open surgery. This novel technology will extend treatment to patients who previously would not be offered therapy due to prohibitive risk" "We are pleased with the 30-day results and look forward to seeing the one-year outcomes," said Pat Mackin, Chairman, President and Chief Executive Officer of Artivion. "These results further validate this important therapeutic option for patients with aortic arch disease." About the NEXUS TRIOMPHE Clinical Trial The NEXUS TRIOMPHE trial is the US IDE study evaluating the NEXUS device in the endovascular treatment of chronic aortic dissection, either primary type B or residual dissection after prior type A repair, or aneurysm (not reported here). Inclusion criteria include the patient being high risk for open surgical repair. The clinical module of the PMA is anticipated to be filed after completion of one year of follow up with the 54 patients in the chronic aortic dissection statistical primary cohort. About Artivion, Inc. Headquartered in suburban Atlanta, Georgia, Artivion, Inc., is a medical device company focused on developing simple, elegant solutions that address cardiac and vascular surgeons' most difficult challenges in treating patients with aortic diseases. Artivion's four major groups of products include: aortic stent grafts, surgical sealants, On-X mechanical heart valves, and implantable cardiac and vascular human tissues. Artivion markets and sells products in more than 100 countries worldwide. For additional information about Artivion, visit our website, . References Leshnower B: Endovascular Aortic Arch Repair Using a Novel Single-Branch Arch Stent Graft for Chronic Dissection: 30-Day Outcomes of the TRIOMPHE FDA Pivotal Study. Presented May 3, 2025 at American Association of Thoracic Surgeons 105th annual meeting. Contacts: SOURCE Artivion, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

CHESHIRE, UK I April 28, 2025 I BlackfinBio Ltd, a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases, announces today that the U.S. Food and Drug Administration (FDA) has cleared its Investigational New Drug (IND) application for a Phase 1/2 clinical trial of its novel adeno-associated virus (AAV) gene therapy, BFB-101, in children with Hereditary Spastic Paraplegia, Type 47 (SPG47). The trial will be conducted in the U.S. at Boston Children’s Hospital and is expected to commence recruiting by the end of 2025. The FDA has granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to BFB-101 for the treatment of SPG47. SPG47 is a rare, autosomal-recessive, neurological disorder characterised by progressive lower-limb spasticity, developmental delays and intellectual disability in children. It is caused by deleterious changes in the AP4B1 gene. Currently, there is no cure or effective treatment available for this devastating condition. BFB-101 has been designed specifically to address the underlying genetic cause of SPG47 by delivering a functional copy of the AP4B1 gene, with the goal of halting or reversing disease progression. Preclinical results with BFB-101 have demonstrated promising activity and safety (1). The Phase 1/2 clinical trial is a single-center open-label study with a primary objective to assess the safety and efficacy of a single injection into the cerebrospinal fluid at the base of the brainstem near the spinal cord, termed an ‘ICM’ administration. This type of delivery generally allows for rapid and efficient delivery of agents to the central nervous system. BFB-101 will be administered by ICM in up to five children with AP4B1-associated SPG47. The secondary objectives will aim to evaluate the impact of BFB-101 on motor function, development and health-related quality of life. “The FDA’s clearance of the BFB-101 IND is an important milestone for our rare neurological disease program and the company. We look forward to initiating enrollment in the U.S. later this year and are working closely with the investigator team at Boston Children’s Hospital to evaluate the therapeutic utility of this gene therapy in children with SPG47,” said Peter Nolan, Founder and Chief Executive Officer of BlackfinBio Ltd. “We are proud to be leading this important clinical trial at Boston Children’s Hospital, bringing forward a precision medicine approach for children affected by SPG47. As clinicians, we see firsthand the impact of this disorder and the urgency for effective treatments. BFB-101 represents a promising gene therapy candidate, and this trial is a critical step toward delivering meaningful change for patients and families affected by AP-4-associated hereditary spastic paraplegia and related conditions,” said Dr. Darius Ebrahimi-Fakhari, M.D., Ph.D., Boston Children’s Hospital (Principal Investigator). “Having overseen this therapeutic innovation from discovery stage, it is a huge source of excitement to reach this important IND milestone. Given the incredibly high unmet need for children with this devastating condition, we are on a mission at BlackfinBio to transform treatment options for these young lives and make a real impact to the lives of their families,” added Professor Mimoun Azzouz, Founder and Chief Scientific Officer of BlackfinBio Ltd. Reference: (1): Pre-clinical development of AP4B1 gene replacement therapy for hereditary spastic paraplegia type 47 Disclosure Statement: Dr. Darius Ebrahimi-Fakhari has consulted for BlackfinBio. About BFB-101 BFB-101 aims to address the underlying genetic cause of Hereditary Spastic Paraplegia, Type 47 (SPG47) by delivering a functional copy of the AP4B1 gene, with the goal of halting or reversing disease progression. It is administered via the brain as a single lifetime dose and has shown potential in restoring AP-4 function in vitro and improving motor function in AP4B1 mutant mice. BFB-101 was originally developed by Professor Mimoun Azzouz at the University of Sheffield with the support of Cure AP-4 ( https://cureap4.org ), LifeArc ( www.lifearc.org ). This project has also received partial funding from the Innovative Medicines Initiative 2 Joint Undertaking under grant agreement No 945473 to support AAV9 capsid synthesis for immune response study. This Joint Undertaking receives support from the European Union’s Horizon 2020 research and innovation programme and EFPIA. The FDA has granted an orphan drug designation (ODD) and rare pediatric disease designation (RPDD) to BFB-101 for the treatment of SPG47. About BlackfinBio Ltd BlackfinBio is a clinical stage gene therapy company focused on the development of treatments for rare neurological diseases. The pipeline comprises BFB-101, a clinical stage AP4B1 replacement AAV gene therapy for spastic paraplegia 47 – an ultra-rare genetic neurological disease for which no treatment currently exists. BFB-201 is a preclinical stage gene therapy to treat several rare dopamine deficiency disorders. For more information, please visit https://blackfin.bio and follow us on LinkedIn . SOURCE: BlackfinBio

5-Year Data from the AMDS DARTS Trial Demonstrates Positive Aortic Remodeling with 94% of Patients Free from Unanticipated Reoperation 30-Day Data from the AMDS PERSEVERE Trial Shows Cerebral Malperfusion Resolution in 90% of Affected Subjects Post-AMDS Implantation 1-Year Data from the NEOS Study Indicate E-vita Open Neo is Safe and Effective in the Treatment of Aortic Arch Pathologies with Low Combined Major Adverse Events Rate ATLANTA, Oct. 10, 2024 /PRNewswire/ -- Artivion, Inc. (NYSE: AORT), a leading cardiac and vascular surgery company focused on aortic disease, today announced data from its AMDS DARTS and PERSEVERE trials and E-vita Open Neo study presented in Late-Breaking Science presentations at the 38th European Association for Cardio-Thoracic Surgery (EACTS) Annual Meeting in Lisbon, Portugal. The presentation regarding the AMDS DARTS trial highlighted 5-year aortic remodeling data that demonstrate positive, durable patient outcomes following AMDS implantation. The presentation on the 30-day AMDS PERSEVERE trial data showed cerebral malperfusion resolution for 90% of affected subjects at 30-days post-AMDS implantation. Additionally, the presentation on the 1-year data from the NEOS study showed E-vita Open Neo is safe and effective for the treatment of acute and chronic aortic arch pathologies with a lower combined major adverse event rate compared to the market leading alternative. "We were thrilled to command such a strong presence at EACTS, with data from our AMDS and E-vita Open Neo clinical trials dominating the aortic focused late-breaking science session," said Pat Mackin, Chairman, President, and Chief Executive Officer of Artivion. "Artivion remains committed to driving continued innovation in treating aortic disease, and these data further validate our best-in-class portfolio." Late-Breaking 5-Year Data from AMDS DARTS Trial: The abstract titled 5-Year Results on Aortic Remodeling in the Dissected Aorta Repair Through Stent (DARTS) Implantation Trial reports five years of clinical follow-up on the remaining 25 of 46 study participants with acute DeBakey Type I dissection who were treated with a proximal aortic repair and AMDS. The results demonstrate durable benefits out to at least 5 years over the baseline: 94% of patients were free from aortic reoperation compared to existing literature on hemiarch-only outcomes which report freedom from late aortic arch reoperation as low as 76%, 95% of patients were free from total aortic diameter (TAD) growth >5 mm between the 3-year and 5-year follow up periods at aortic zones 2 and 4; and In contrast, existing literature on hemiarch-only outcomes suggest a majority of patients have early TAD growth in the proximal descending aorta. Aortic growth can lead to increased risk of rupture or dissection, and reoperation. Principal investigator Dr. Michael Moon, Division of Cardiac Surgery, University of Alberta, Edmonton, Canada said, "Significant dilation of the ascending aorta, measured by changes to the TAD, increases the risk of spontaneous rupture or dissection of the aorta, a serious medical emergency. These long-term results from the DARTS study demonstrate a large majority of patients experience a stable or decreased TAD following treatment with AMDS, meaning these patients were successfully treated with AMDS and are at decreased risk for further aortic dissection and reoperation as far out as 5-years post implantation." Late-Breaking 30-Day Data from AMDS PERSEVERE Trial: The abstract titled AMDS Hybrid Prosthesis Improves Cerebral Malperfusion in Acute DeBakey Type I Dissection- Early Results of the PERSEVERE Study focuses on cerebral blood flow across the full 93 study participant IDE cohort at 30-days following AMDS implantation. 20% of patients (19 of 93 patients) presented with symptomatic pre-operative cerebral malperfusion including stroke, transient ischemic attack, delirium or other mental status changes. The 30-day data showed resolution of cerebral malperfusion in the vast majority of patients treated with AMDS. More specifically: 90% of patients experienced resolution of their cerebral malperfusion; and 63% of those who presented with cerebral malperfusion had complete resolution, while another 26% had no worsening of stroke symptoms The data compare favorably to hemiarch alone based on data from five articles in the literature, which showed a stroke occurrence rate of 20.9% compared to 10.8% (10 of 93 patients) in the PERSEVERE trial. Senior author, Dr. William Brinkman, Baylor Scott & White, The Heart Hospital Plano, Plano, Texas said, "Cerebral malperfusion, which often leads to stroke, is a devasting complication of Acute DeBakey Type I aortic dissection. The data from the PERSEVERE trial indicating that AMDS markedly reduces 30-day malperfusion and new disabling stroke compared to the baseline, is very encouraging." Late-Breaking 1-Year Data from E-vita Open Neo NEOS Study: The abstract titled E-vita Open Neo in the treatment of acute or chronic aortic pathologies: 1-year results of the European multicenter NEOS study and sub-group analysis includes 161 participants treated with E-vita Open Neo in Europe. The 1-year data suggests E-vita Open Neo is safe and effective in the treatment of aortic arch pathologies and compares favorably to the IDE study results of the current market leading device: The 1-year mortality rate observed for E-vita Open Neo (9.9%) compared favorably to current market leading device (10.8%); and Lower 1 year combined major adverse events rate of mortality, stroke, and paraplegia/ paraparesis occurring after treatment with E-vita Open Neo (17% vs 23.1% for current market leading device). Professor Martin Grabenwöger, Head, Department of Cardiovascular Surgery, Hietzing Hospital, Vienna, Austria said, "These exciting results from the NEOS study demonstrate the clinical benefits of E-vita Open Neo compare favorably to currently available options for treatment of aortic arch pathologies. This study is a major step forward in improving care for patients suffering from this life-threatening disease." About the AMDS DARTS Trial The AMDS DARTS (Dissected Aorta Repair Through Stent Implantation) trial is a prospective, multicenter, nonrandomized, single-arm trial of 47 patients evaluating the safety and efficacy of the AMDS Hybrid Prosthesis for the treatment of Acute DeBakey Type I (ADTI) dissection. Trial results supported regulatory approvals for AMDS in Canada, Europe, and other countries around the world. About the AMDS PERSEVERE Trial The AMDS PERSEVERE trial is a prospective, multicenter, non-randomized clinical trial to determine if patients with ADTI dissection can be treated safely and effectively using the AMDS Hybrid Prosthesis. The trial is designed to support the company's forthcoming application to the U.S. Food and Drug Administration (FDA) for premarket approval of the AMDS. The trial consists of 93 participants in the U.S., who have experienced an ADTI dissection. Each participant will be followed for up to 5 years. The combined 30-day safety and primary efficacy endpoints were used to determine the impact of the AMDS Hybrid Prosthesis on DANE prevention, reducing mortality, new disabling stroke, new onset renal failure requiring dialysis, and myocardial infarction, as well as remodeling of the aorta. Full 30-day IDE data from the PERSEVERE trial presented at the Society of Thoracic Surgeons in January 2024, demonstrated statistically significant reduction in all-cause mortality and primary major adverse events (MAEs) at 30 days with use of AMDS in ADTI dissections complicated by malperfusion. Data from the trial also demonstrated a 72% reduction in all-cause mortality and 54% reduction in primary major MAEs when compared to the current standard of care hemiarch procedure. Data presented from the PERSEVERE trial at the 104th American Association for Thoracic Surgery (AATS) Annual Meeting April 2024, showed positive aortic remodeling at 30-days after treatment with AMDS, as well as no occurrence of distal anastomotic new entry (DANE) tears. More specifically, at 30 days, the majority of patients demonstrated favorable remodeling with over 80% showing either stable or decreased total aortic diameter (TAD). About the E-vita Open Neo NEOS Study The E-vita Open Neo NEOS study is an observational, multicenter study to determine the safety and clinical performance of E-vita Open Neo Stent Graft System in the treatment of acute and chronic aneurysm and dissection in the ascending aorta, aortic arch and descending thoracic aorta. The observational study consists of 161 patients with acute or subacute type A aortic dissection, chronic type A aortic dissection or thoracic aortic aneurysm enrolled at 12 centers in Europe and Asia December 2020 to March 2022. The primary end point was the rate of all-cause mortality at 30 days. The secondary end points included further clinical and safety data that are reported up to 3–6 months postoperatively. About Artivion, Inc. Headquartered in suburban Atlanta, Georgia, Artivion, Inc. is a medical device company focused on developing simple, elegant solutions that address cardiac and vascular surgeons' most difficult challenges in treating patients with aortic diseases. Artivion's four major groups of products include: aortic stent grafts, surgical sealants, On-X mechanical heart valves, and implantable cardiac and vascular human tissues. Artivion markets and sells products in more than 100 countries worldwide. For additional information about Artivion, visit our website, . Contacts: SOURCE Artivion, Inc. WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED