Bruton Type Agammaglobulinemia

TEANECK, N.J., Sept. 9, 2024 /PRNewswire/ -- GC Biopharma USA, Inc. announced the launch and distribution of its immunoglobulin (IG) product ALYGLO (immune globulin intravenous, human-stwk), the company's first 10% intravenous immunoglobulin therapy for the treatment of adult patients aged 17 years and older with primary humoral immunodeficiency (PI). ALYGLO is a treatment option for patients with PI. This rare and chronic disorder occurs when the body's ability to produce antibodies is impaired, affecting the immune system's ability to fight infections. ALYGLO, an immune globulin formulation, has met the primary endpoints for both safety and efficacy as established by the FDA recommended guidelines in our Phase 3 clinical trials. Its novel manufacturing process may help improve patient safety by using Cation Exchange Chromatography (CEX) to remove coagulation factor XIa (FXIa) to undetectable levels. ALYGLO was approved by the U.S. Food and Drug Administration (FDA) on Dec. 15, 2023. Dr. Eun Chul Huh, President and CEO of GC Biopharma, said, "The US launch of ALYGLO by GC Biopharma USA is a historic milestone for the company and patients with primary immunodeficiency disease. It executes GC Biopharma's mission to improve the health and lives of individuals who suffer from rare and chronic diseases globally. GC Biopharma's commitment to the US further reflects its investment in R&D and a pipeline of future products to the US marketplace that will benefit patients and healthcare professionals." ALYGLO® is available to patients and healthcare professionals through a limited distribution network of top national Specialty Pharmacy providers. These partnerships provide a national footprint, delivering high-quality patient care for the primary immune deficiency population. ALYGLO is also available through specialty distributors throughout the United States. "ALYGLO expands patients' treatment options and improves access within the US marketplace," stated Dr. Lisa Betts, Chief Operating Officer for GC Biopharma USA, Inc. "GC Biopharma is committed to providing life-saving therapies to people with rare diseases. We are excited to offer the primary immune deficiency population another immune globulin therapeutic option." Please see Important Safety Information for ALYGLO on the following pages and refer to the full Prescribing Information (PI) or visit Alyglo.com For any medical or scientific questions regarding ALYGLO, call the GC Biopharma USA medical department at 1-833-426-6426. About ALYGLO ALYGLO is a liquid solution containing 10% immunoglobulin G (100 mg/mL) for intravenous infusion, manufactured from pooled human plasma from US donors. The manufacturing process includes multiple steps to reduce the risk of virus transmission. These include fractionation, solvent/detergent treatment, and nanofiltration. ALYGLO also uses its novel Cation Exchange Chromatography (CEX) in the manufacturing process for removing FXIa to undetectable levels. About Primary Humoral Immunodeficiency (PI) refers to a group of disorders where the body's ability to produce antibodies is impaired, affecting the immune system's ability to fight infections. In PI, there's a deficiency or absence of B cells or plasma cells, which are key in producing antibodies, particularly immunoglobulin G. This leads to increased susceptibility to infection, especially bacterial infections affecting the respiratory and gastrointestinal tracts. The PI indication for ALYGLO includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. About GC Biopharma Corp. GC Biopharma Corp. (formerly known as Green Cross Corporation) is a biopharmaceutical company that delivers lifesaving and life-sustaining protein therapeutics and vaccines. Headquartered in Yongin, South Korea, GC Biopharma is a leading global plasma protein and vaccine product manufacturer dedicated to quality healthcare solutions for over half a century. About GC Biopharma USA, Inc. GC Biopharma USA, Inc., headquartered in Teaneck, NJ, established its sales, marketing, and business operations in 2018 to serve customers and patients throughout the US. Our foundation is built on the expertise of our parent company, GC Biopharma's, expertise—a leading biopharmaceutical company delivering plasma therapies and vaccines worldwide. With GC Biopharma USA, Inc., GC Biopharma further intends to extend its footprint, bringing its expertise and legacy to the United States. Forward-Looking Statements This press release may contain forward-looking statements that express the current beliefs and expectations of GC Biopharma USA management within the meaning of the Private Securities Litigation Reform Act of 1995. We intend such forward-looking statements to be covered by the safe harbor provisions for forward-looking statements contained in Section 27A of the Securities Act of 1933, as amended and Section 21E of the Securities Exchange Act of 1934, as amended. All statements contained in this press release other than statements of historical fact should be considered forward-looking statements and do not represent any guarantee by GC Biopharma USA or its management of future performance and involve known and unknown risks, uncertainties, and other factors. When used herein, words including "anticipate," "believe," "can," "continue," "could," "designed," "estimate," "expect," "forecast," "goal," "intend," "may," "might," "plan," "possible," "potential," "predict," "project," "should," "target," "will," "would" and similar expressions are intended to identify forward-looking statements, though not all forward-looking statements use these words or expressions. In addition, any statements or information that refer to expectations, beliefs, plans, projections, objectives, performance or other characterizations of future events or circumstances, including any underlying assumptions, are forward-looking. All forward-looking statements are based upon the Company's current expectations and various assumptions. The Company believes there is a reasonable basis for its expectations and beliefs, but they are inherently uncertain. The Company may not realize its expectations, and its beliefs may not prove correct. GC Biopharma USA undertakes no obligation to update or revise any forward-looking statement contained in this press release or any other forward-looking statements it may make, except as required by law or stock exchange rule. INDICATION ALYGLO™ is indicated for the treatment of primary humoral immunodeficiency (PI) in adults aged 17 years and older. This includes, but is not limited to, congenital agammaglobulinemia, common variable immunodeficiency (CVID), Wiskott-Aldrich syndrome, and severe combined immunodeficiencies. IMPORTANT SAFETY INFORMATION Contraindications: ALYGLO is contraindicated in patients who have a history of anaphylactic or severe systemic reaction to the administration of human immune globulin and in IgA-deficient patients with antibodies to IgA and a history of hypersensitivity. Hypersensitivity: In case of hypersensitivity, discontinue infusion immediately and institute appropriate treatment. Epinephrine should be available for immediate treatment of severe acute hypersensitivity reactions. Hyperproteinemia, Increased Serum Viscosity, and Hyponatremia: Hyperproteinemia, increased serum viscosity, and hyponatremia may occur. Aseptic Meningitis Syndrome (AMS): Aseptic meningitis syndrome (AMS) may occur, especially with high doses or rapid infusion. AMS usually begins within several hours to 2 days following ALYGLO treatment. Discontinuation of treatment has resulted in remission of AMS within several days without sequelae. Hemolysis: Delayed hemolytic anemia due to enhanced red blood cell (RBC) sequestration and acute hemolysis consistent with intravascular hemolysis have been reported. Cases of severe hemolysis-related renal dysfunction/failure or disseminated intravascular coagulation have occurred following infusion of IGIV. Closely monitor patients for clinical signs and symptoms of hemolysis, particularly patients with risk factors. Transfusion-Related Acute Lung Injury: Noncardiogenic pulmonary edema (transfusion-related acute lung injury [TRALI]) may occur. TRALI is characterized by severe respiratory distress, pulmonary edema, hypoxemia, normal left ventricular function, and fever. Patients with TRALI may be managed using oxygen therapy with adequate ventilator support. Monitor patients for pulmonary adverse reactions. Transmissible Infectious Agents: Because ALYGLO is made from human blood, it may carry a risk of transmitting infectious agents (eg, viruses, the variant Creutzfeldt-Jakob disease [vCJD] agent and, theoretically, the Creutzfeldt-Jakob disease [CJD] agent). Interference with Laboratory Tests: After infusion of immunoglobulin, the transitory rise of the various passively transferred antibodies in the patient's blood may yield positive serological testing results, with the potential for a misleading interpretation. Adverse reactions (observed in ≥ 5% of study subjects) were headache, nausea/vomiting, fatigue, nasal/sinus congestion, rash, arthralgia, diarrhea, muscle pain/aches, infusion site pain/swelling, abdominal pain/discomfort, cough, and dizziness. It is recommended that ALYGLO be administered separately from other drugs or medications. ©2024 GC Biopharma USA, Inc. All rights reserved. ALYGLO is a trademark of Green Cross Corporation. SOURCE GC Biopharma USA Inc.

Potential to be the first IVD for the screening of Wilson Disease, Wiskott-Aldrich Syndrome, X-linked Agammaglobulinemia and Adenosine Deaminase Deficiency SEATTLE, March 29, 2024 /PRNewswire/ -- Key Proteo, a pioneering proteomics diagnostics company specializing in the enhanced early detection of rare but treatable genetic disorders, today announced that it has submitted a de novo classification request to the U.S. Food and Drug Administration (FDA) for its first in vitro diagnostic Key Proteo Newborn Screening Kit. The novel proteomics-based screening panel addresses a critical, unmet need to aid in the early identification of four treatable rare genetic disorders that current newborn screening programs do not typically test for, including Wilson Disease (WD), Wiskott-Aldrich Syndrome (WAS), X-linked Agammaglobulinemia (XLA) and Adenosine Deaminase Deficiency (ADA). Early screening has the potential to enable timely intervention that can help change the clinical trajectory and relieve the burden of disease for patients and their families afflicted by these potentially devastating disorders. The submission of Key Proteo's Newborn Screening Kit 1 comes after screening over 22,000 newborn samples in an ongoing pilot study in Washington state. Subsequently, Key Proteo successfully completed a clinical study that included 3,294 newborn samples analyzed at 3 sites across North America. "We are excited to lead the charge towards changing the clinical course of treatable rare genetic disorders that are currently being missed," said Mark Willig, President and CEO of Key Proteo. "Enhanced early screening is key to enabling clinical interventions that can significantly help reduce morbidity and mortality. I am extremely proud of the tireless efforts that our team has made over the past five years to reach this important milestone not just for our company, but for the patients and their families that will benefit from these life-altering screening tools." With its initial focus on addressing unmet needs for WD, WAS, XLA and ADA in the newborn screening space, nearly 100 treatable genetic disorders have been identified that could potentially be adapted to Key Proteo's dynamic proteomics-based platform. The in vitro diagnostic Key Proteo Newborn Screening Kit 1 will only be available for commercialization once the FDA has completed its process. Currently, Key Proteo Newborn Screening Kit 1 is available as an RUO offering. In parallel path, Key Proteo is also expanding its operations to include a CLIA-certified laboratory to serve unaddressed newborn screening needs among public health laboratories, reference laboratories, hospital laboratories and other providers associated with neonatal care that may prefer to send-out for these and other critically important newborn screening tests. ABOUT WILSON DISEASE, WISKOTT-ALDRICH SYNDROME, XLA and ADA Wilson Disease, affecting approximately 1 in 30,000 individuals, is characterized by excess copper accumulation in the liver, brain, and eyes, and can lead to liver disease, central nervous system dysfunction and death. Timely diagnosis and treatment are vital to prevent long-term disability and life-threatening complications. Impacting 1 in 100,000, Wiskott-Aldrich Syndrome is characterized by immunodeficiency and impaired blood clotting. Symptoms include bruising, susceptibility to infections and immune and inflammatory disorders, and heightened cancer risk. Eczema often accompanies the syndrome. WAS patients face increased vulnerability to infections, bleeding, and cancers, often leading to premature mortality. X-linked Agammaglobulinemia, impacting around 1 in 250,000 individuals, mainly males, is marked by severely low immunoglobulin levels. Typically, symptoms manifest as recurrent bacterial infections from around 6 months of age, with commonly diagnosed infections including lung, ear, sinus, eye, and skin infections, along with those linked to chronic diarrhea. Adenosine Deaminase Deficiency (ADA), impacting roughly 1 in 500,000 individuals, is a primary cause of severe combined immunodeficiency (SCID), impairing the body's ability to combat infections. Symptoms typically emerge before 6 months, ranging from pneumonia to chronic diarrhea and delayed development. While most symptoms surface early, some may manifest later in life. ABOUT KEY PROTEO Key Proteo is on a mission to save lives and relieve the burden of disease for patients and their families afflicted by rare but treatable genetic disorders through early screening. Many treatable genetic disorders are difficult to detect, but when found early, intervention can be enabled to help change the clinical course. With its initial focus on newborns, Key Proteo's dynamic proteomics-based platform can be adapted to screen for rare but treatable genetic disorders across other patient populations. Key Proteo's patent-pending Immuno-SRM platform and associated reagent kits can perform targeted proteomic analysis of extremely low abundance peptide biomarkers from a few drops of blood. This proprietary technology expands the scope of detecting previously undetectable genetic disorders in newborns with high accuracy, effectiveness and efficiency, and has demonstrated faster turnaround times and lower costs when compared to next-generation sequencing (NGS). FORWARD-LOOKING STATEMENTS Certain information contained in this press release constitutes forward-looking statements. These forward-looking statements involve a number of risks and uncertainties that could cause actual future results to differ materially from those anticipated. Forward-looking statements represent the Company's estimates only as of the date such statements are made and the Company undertakes no obligation to update forward-looking statements except as required by applicable securities laws. Readers are cautioned that forward-looking statements are not guarantees of future performance and are cautioned not to place undue reliance on any forward-looking statements. For more information, visit . #KeyProteo #DeNovoClassification #NewbornScreening #RareDisease #WilsonDisease #WilsonDiseaseAssociation #WiskottAldrichSyndrome #XLA #AdenosineDeaminaseDeficiency #ImmuneDeficiencyFoundation #Proteomics #Biotech #VentureCapital #LifeSciences #HealthTech #BioTechNews CONTACT Sean Sandin Chief Operating Officer [email protected] SOURCE Key Proteo

The DynamX BTK System is intended for the treatment of BTK vessels impacted by CLTI by widening the use of the company’s novel bioadaptor platform technology beyond the treatment of coronary artery disease DynamX BTK system treats patients with CLTI. (Credit: Arek Socha from Pixabay) US-based medical technology company Elixir Medical has secured the US Food and Drug Administration (FDA) Breakthrough Device Designation for its DynamX BTK System. DynamX is a novel, adaptive implant intended for use in the treatment of narrowed or blocked vessels below the knee (BTK) in patients with chronic limb-threatening ischemia (CLTI). The system leverages the DynamX Bioadaptor platform, a medical technology designed to uncage the vessel and restore normal vessel motion and function after PCI or BTK intervention. With a unique mechanism of action (MOA), the technology maintains dynamic support of the diseased vessel after uncaging, establishing a new standard of care for vascular interventions. Elixir Medical CEO Motasim Sirhan said: “The Bioadaptor platform was developed to transform treatment of coronary and peripheral artery disease. “We appreciate the FDA recognition of our innovation for treating the CLTI population with BTK disease and its potential impact on the patients suffering from vascular disease.” Peripheral arterial disease (PAD) is a condition characterised by narrowing or blockage of the blood vessels in the leg, primarily caused by the buildup of fatty plaque in the arteries. CLTI is an advanced stage of PAD and is associated with risk to both life and limb, with one-year estimates of mortality over 20% across a range of observational studies. According to Elixir, DynamX is the only metallic device designed to support the vessel during the healing phase, after which it unlocks the vessel. The system also provides essential dynamic support to restore vessel function and maintain an open lumen. The unique design and mechanism of action make bioadaptor a promising therapy in BTK revascularisation, said the medical device maker. DynamX Bioadaptor provides high acute lumen gain in the coronary vessels and maintains the gain over time, which is a common challenge in existing BTK therapies. The bioadaptor also restores vessel motion and function, including positive adaptive remodelling, vessel pulsatility, improved vessel dynamic compliance, and enhanced blood flow.