Semicircular Canal Dehiscence

Interest continues to grow in developing new strategies to reduce systemic inflammation as a means of improving outcomes in heart failure patients DENVER, Feb. 27, 2024 (GLOBE NEWSWIRE) -- SeaStar Medical Holding Corporation (Nasdaq: ICU), a medical device company developing proprietary solutions to reduce the consequences of hyperinflammation on vital organs, announces publication of a manuscript discussing the role of chronic dysregulated systemic inflammation in heart failure and the potential application of the adult Selective Cytopheretic Device, part of the Quelimmune™ product family, in enabling previously ineligible patients with severe disease to receive a left ventricular assist device (LVAD) or heart transplant in the peer-reviewed European Journal of Heart Failure February 2024, Pitt, B., Iyer, S.P.N. and Humes, H.D. (2024), Eur J Heart Fail. https://doi.org/10.1002/ejhf.3177. The manuscript, titled “New Opportunity for Targeting Systemic Inflammation in Patients with Heart Failure through Leukocyte Immunomodulation” cites increasing evidence of the role of chronic systemic inflammation in patients with heart failure and discusses the Adult SCD’s potential to improve hypertensive heart failure and mortality following hospitalization for acute or worsening heart failure. The manuscript reviews the positive effect of the Adult SCD in a previously announced first-in-human case report of a 71-year-old patient with cardiorenal syndrome who was deemed ineligible for cardiac transplantation or a LVAD due to worsening renal function and right ventricular dysfunction. Following treatment with the Adult SCD, this patient was effectively bridged to LVAD three days after discontinuing treatment and was subsequently discharged without further complications. This study was the basis for Adult SCD receiving a Breakthrough Device Designation in cardiorenal syndrome from the U.S. Food and Drug Administration (FDA) Center for Biologics Evaluation and Research (CBER) in September 2023. “Systemic inflammation is one component in the multi-factorial pathophysiology of heart failure, and the use of anti-inflammatory strategies in patients with acute or worsening heart failure is not novel ,” said lead manuscript author and heart failure expert Bertram Pitt, MD, FACC, Division of Internal Medicine (Emeritus), University of Michigan School of Medicine. “Prior attempts targeting singular proinflammatory factors as well as systemic immunosuppression with glucocorticoids in patients with acute heart failure have yielded unclear results, raising the need to identify new strategies. An alternative strategy may be to target cellular sources of proinflammatory molecules such as activated leucocytes.” “Our novel extracorporeal device has shown significant promise in targeting active neutrophils and monocytes to quell systematic inflammation, leading to improved outcomes in hyperinflammatory acute conditions including acute kidney injury, acute respiratory distress syndrome and cardiorenal syndrome,” said manuscript co-author Sai Prasad N. Iyer, Ph.D., SeaStar Medical’s Vice President, Medical Affairs and Clinical Development. “While our manuscript discusses a positive outcome in one patient following treatment for six hours daily over six consecutive days, the results suggest that more study with Adult SCD is warranted for patients with heart failure and hyperinflammation.” On February 22, 2024 SeaStar Medical announced the Humanitarian Device Exemption (HDE) FDA Approval Order for the Pediatric SCD (SCD-PED now known as Quelimmune) for use in children weighing 10 kilograms or more with acute kidney injury due to sepsis or a septic condition requiring kidney replacement therapy (KRT). About Hyperinflammation Hyperinflammation is the overproduction or overactivity of inflammatory cells that can lead to damage of vital organs. It occurs when the body overproduces inflammatory effector cells and other molecules that can be toxic, damaging to vital organs and result in multi-organ failure and even death. This is known as the cytokine storm. About the Selective Cytopheretic Device (SCD)The SCD is a patented cell-directed extracorporeal device that employs immunomodulating technology to selectively target proinflammatory neutrophils and monocytes during CKRT and reduces the hyperinflammatory milieu including the cytokine storm that causes inflammation, organ failure and possible death in critically ill patients. Unlike pathogen removal and other blood-purification tools, the device is integrated with CKRT hemofiltration systems to selectively target and transition proinflammatory monocytes to a reparative state and promote activated neutrophils to be less inflammatory. The SCD selectively targets the most highly activated proinflammatory neutrophils and monocytes. These cells are then returned back into the body through the blood, and the body is signaled to lower its inflammatory environment and focus on repair. This unique immunomodulation approach may promote long-term organ recovery and eliminate the need for future KRT, including dialysis. About SeaStar MedicalSeaStar Medical is a medical technology company that is redefining how extracorporeal therapies may reduce the consequences of excessive inflammation on vital organs. SeaStar Medical’s novel technologies rely on science and innovation to provide life-saving solutions to critically ill patients. The Company is developing and commercializing cell-directed extracorporeal therapies that target the effector cells that drive systemic inflammation, causing direct tissue damage and secreting a range of pro-inflammatory cytokines that initiate and propagate imbalanced immune responses. For more information visit www.seastarmedical.com or visit us on LinkedIn or X. Forward-Looking StatementsThis press release contains certain forward-looking statements within the meaning of the “safe harbor” provisions of the Private Securities Litigation Reform Act of 1955. These forward-looking statements include, without limitation, the SCD’s ability to treat hyperinflammation and the expected results of clinical trials and studies. Words such as “believe,” “project,” “expect,” “anticipate,” “estimate,” “intend,” “strategy,” “future,” “opportunity,” “plan,” “may,” “should,” “will,” “would,” “will be,” “will continue,” “will likely result,” and similar expressions are intended to identify such forward-looking statements. Forward-looking statements are predictions, projections and other statements about future events that are based on current expectations and assumptions and, as a result, are subject to significant risks and uncertainties that could cause the actual results to differ materially from the expected results. Most of these factors are outside SeaStar Medical’s control and are difficult to predict. Factors that may cause actual future events to differ materially from the expected results include, but are not limited to: (i) the risk that SeaStar Medical may not be able to obtain regulatory approval of its SCD product candidates; (ii) the risk that SeaStar Medical may not be able to raise sufficient capital to fund its operations, including clinical trials; (iii) the risk that SeaStar Medical and its current and future collaborators are unable to successfully develop and commercialize its products or services, or experience significant delays in doing so, including failure to achieve approval of its products by applicable federal and state regulators, (iv) the risk that SeaStar Medical may never achieve or sustain profitability; (v) the risk that SeaStar Medical may not be able to access funding under existing agreements, including the equity line of credit and forward purchase agreements; (vi) the risk that third-parties suppliers and manufacturers are not able to fully and timely meet their obligations, (vii) the risk of product liability or regulatory lawsuits or proceedings relating to SeaStar Medical’s products and services, (viii) the risk that SeaStar Medical is unable to secure or protect its intellectual property, and (ix) other risks and uncertainties indicated from time to time in SeaStar Medical’s Annual Report on Form 10-K, including those under the “Risk Factors” section therein and in SeaStar Medical’s other filings with the SEC. The foregoing list of factors is not exhaustive. Forward-looking statements speak only as of the date they are made. Readers are cautioned not to put undue reliance on forward-looking statements, and SeaStar Medical assume no obligation and do not intend to update or revise these forward-looking statements, whether as a result of new information, future events, or otherwise. Contact: LHA Investor Relations Jody Cain (310) 691-7100 Jcain@lhai.com # # #

Pictured: Illustration of sickle cells in the bloodstream/iStock, Artur Plawgo Despite winning FDA approval on Friday for its sickle cell disease gene therapy Lyfgenia (lovotibeglogene autotemcel), bluebird bio’s investors were less than bullish on the company. The Massachusetts-based biotech closed the day down nearly 35%. Much of the negative share reaction could have been driven by Lyfgenia’s list price. Shortly after its regulatory win, bluebird announced that the gene therapy would have a wholesale acquisition cost of $3.1 million. In its news announcement, bluebird said it arrived at this price “in recognition of the value the therapy may deliver through robust and sustained clinical benefits and the estimated lifetime impact,” including savings from lower healthcare utilization, patients’ future earnings and their life opportunities. By comparison, Vertex Pharmaceuticals’ and CRISPR Therapeutics’ Casgevy (exagamglogene autotemcel), which was approved on the same day as Lyfgenia, will go for $2.2 million. Casgevy is a CRISPR-based gene edited therapy, is also indicated for sickle cell disease (SCD) and will likely be a strong competitor for Lyfgenia. Over the weekend, at the 65th Annual Meeting & Exposition of the American Society of Hematology, experts were cautiously optimistic about what the dual approvals of Casgevy and Lyfgenia might mean for patients, STAT News reported. However, many expressed their confusion, if not shock, at bluebird’s pricing. “For two therapies which do pretty much the same thing and are targeting exactly the same population, a price tag that’s $1 million apart, I think, is a major deciding factor for many patients and providers and even insurance programs,” Akshay Sharma, who treats pediatric SCD patients at St. Jude Children’s Research Hospital, told STAT. “It’s baffling to me why Bluebird Bio priced their therapy so high — or maybe Vertex priced theirs low, I don’t know,” Sharma said. Also potentially contributing to bluebird’s drop on Friday is Lyfgenia’s boxed warning, which flags instances of hematologic malignancies in patients who had been treated with the gene therapy. Additionally, bluebird failed to secure an FDA priority review voucher, which it had planned to sell to Novartis for $103 million in a bid to improve its cash position. In its third-quarter financial report, bluebird said that it had $227 million in cash, cash equivalents, marketable securities and restricted cash, which would be enough to support its operations only through the second quarter of 2024. Seemingly lost in the discussion about Lyfgenia’s pricing are new long-term follow-up data, which bluebird posted on Saturday. Over five years of follow-up, 94% patients treated with the gene therapy achieved complete resolution of severe vaso-occlusive events, while 88.2% saw complete resolution of all vaso-occlusive events. Tristan Manalac is an independent science writer based in Metro Manila, Philippines. He can be reached at tristan@tristanmanalac.com or tristan.manalac@biospace.com.

Renowned, Expert Care Team to Deliver Transformative New Therapy to Eligible Adult Patients Suffering from Sickle Cell Disease BOSTON, Dec. 8, 2023 /PRNewswire/ -- Boston Medical Center (BMC), a national leader in the treatment of sickle cell disease for more than 50 years, announced today that it will offer the first-ever gene therapy treatment program for sickle cell disease that uses a type of novel genome editing technology. The new therapy leverages the latest advances in medical science to alleviate the severe painful vaso-occlusive crises (VOCs) associated with sickle cell disease, in a long-awaited step toward equity for a disproportionally impacted Black patient population. The announcement comes as the FDA today granted approval of this new therapy, jointly developed by Boston-based Vertex Pharmaceuticals and CRISPR Therapeutics of Switzerland, along with approval of another cell-based gene therapy for sickle cell disease. BMC is the only hospital in New England to offer the Vertex Pharmaceuticals and CRISPR Therapeutics therapy to eligible adults with sickle cell disease. "Boston Medical Center has a longstanding commitment to providing advanced clinical care to those with sickle cell disease," said Dr. Jean-Antoine Ribeil, MD, PhD, Clinical Director of the Center of Excellence in Sickle Cell Disease at Boston Medical Center and an internationally renowned hematologist who has dedicated his career to the development of gene therapies for patients with sickle cell disease and beta thalassemia. "The new gene therapies can be life changing for eligible patients who are impacted by sickle cell disease and the extreme pain that it causes, with previously limited treatment options." Sickle cell disease is a debilitating and chronic disease that afflicts more than 100,000 Americans and predominantly impacts people of African descent, representing 1 out of every 365 births1. The disease is caused by a gene mutation that makes blood cells misshapen which can lead to strokes, organ damage and episodes of agonizing pain. Those with sickle cell disease often rely on regular blood transfusions to ease pain and reduce risk of additional complications. The new Vertex Pharmaceuticals treatment is the first therapy to treat a genetic disease with the CRISPR gene-editing technique. Vertex began clinical trials on the therapy in the United States in 2018. The SCD clinical trial enrolled a patient population that was predominantly of African ancestry. The FDA reviewed the safety and effectiveness of the treatment in that trial before today's approval. The treatment will be offered to select, eligible adult sickle cell patients at BMC as part of its Center of Excellence in Sickle Cell Disease, which is the largest center of its kind in New England, serving approximately 600 adult and pediatric patients annually. The introduction of this therapy gives those with sickle cell disease more options to try to achieve a life without chronic pain. The BMC Center of Excellence in Sickle Cell Disease has long taken an innovative and multidisciplinary approach to caring for patients suffering from this debilitating disease. The Center pairs hematologists with specialists, such as pulmonologists, nephrologists, and primary care doctors who understand the needs and many complications of patients with sickle cell disease. The addition of cutting-edge gene therapy to its suite of services reflects BMC's ongoing commitment to this population and to being a leader in offering advanced, equitable care to its patients. For more information on BMC's Center of Excellence in Sickle Cell Disease visit their website or connect on Facebook, Instagram, X (formerly Twitter), and LinkedIn. About Boston Medical Center Boston Medical Center models a new kind of excellence in healthcare, where innovative and equitable care empowers all patients to thrive. We combine world-class clinicians and cutting-edge treatments with compassionate, quality care that extends beyond our walls. As an award-winning health equity leader, our diverse clinicians and staff interrogate racial disparities in care and partner with our community to dismantle systemic inequities. And as a national leader in research and the teaching affiliate for Boston University Chobanian & Avedisian School of Medicine, we're driving the future of care. Media Contact: David Kibbe 617.638.8499 [email protected] 1(SCT). SOURCE Boston Medical Center