Wiskott-Aldrich Syndrome

FDA Approval Provides for Room Temperature (25°C) Storage Conditions for up to 4 Weeks at Any Point During the 36-Month Approved Shelf Life Extends the Prior Room Temperature Storage Allowance for the Full 36-months for ASCENIV and BIVIGAM Provides for Improved Inventory Management and Ease of Product Administration to Patients Approval of Extended Ambient Storage Conditions for ASCENIV & BIVIGAM is Immediately Effective and Now Commercially Available to U.S. Healthcare Providers RAMSEY, N.J. and BOCA RATON, Fla., March 11, 2024 (GLOBE NEWSWIRE) -- ADMA Biologics, Inc. (NASDAQ: ADMA) (“ADMA” or the “Company”), an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty biologics, today announced the United States Food and Drug Administration’s (“FDA”) approval for its supplemental Biologics License Applications (BLAs) for both ASCENIV and BIVIGAM to extend the approved 4-week room temperature (25°C) storage conditions during the first 24 months of shelf life, to allow for a 4-week room temperature storage at any time during the entire 36-month approved shelf life. The room temperature approval applies to all existing ASCENIV and BIVIGAM lots currently in the commercial supply chain as well as to future production of ASCENIV and BIVIGAM. “With the FDA-approved extension of room temperature storage conditions, the Company expects to reach more customers who were previously inaccessible due to limited refrigeration space and cold chain capacity constraints,” said Adam Grossman, President and Chief Executive Officer of ADMA. “We believe that this added storage flexibility for both ASCENIV and BIVIGAM will meaningfully enhance our products’ market offerings, enabling more versatile utilization and better inventory management for providers.” The newly approved extension of room temperature storage conditions for both ASCENIV and BIVIGAM is immediately effective, and both products are commercially available to U.S. healthcare providers and patients. About ADMA Biologics, Inc. (ADMA) ADMA Biologics is an end-to-end commercial biopharmaceutical company dedicated to manufacturing, marketing and developing specialty biologics for the treatment of immunodeficient patients at risk for infection and others at risk for certain infectious diseases. ADMA currently manufactures and markets three United States Food and Drug Administration (FDA)-approved plasma-derived biologics for the treatment of immune deficiencies and the prevention of certain infectious diseases: BIVIGAM® (immune globulin intravenous, human) for the treatment of primary humoral immunodeficiency (PI); ASCENIV™ (immune globulin intravenous, human – slra 10% liquid) for the treatment of PI; and NABI-HB® (hepatitis B immune globulin, human) to provide enhanced immunity against the hepatitis B virus. ADMA manufactures its immune globulin products at its FDA-licensed plasma fractionation and purification facility located in Boca Raton, Florida. Through its ADMA BioCenters subsidiary, ADMA also operates as an FDA-approved source plasma collector in the U.S., which provides its blood plasma for the manufacture of its products. ADMA’s mission is to manufacture, market and develop specialty biologics, human immune globulins targeted to niche patient populations for the treatment and prevention of certain infectious diseases and management of immune compromised patient populations who suffer from an underlying immune deficiency, or who may be immune compromised for other medical reasons. ADMA holds numerous U.S. and foreign patents related to and encompassing various aspects of its products and product candidates. For more information, please visit . About ASCENIV™ ASCENIV (immune globulin intravenous, human – slra 10% liquid) is a plasma-derived, polyclonal, intravenous immune globulin (IVIG). ASCENIV was approved by the United States Food and Drug Administration (FDA) in April 2019 and is indicated for the treatment of primary humoral immunodeficiency (PI), also known as primary immune deficiency disease (PIDD), in adults and adolescents (12 to 17 years of age). ASCENIV is manufactured using ADMA’s unique, patented plasma donor screening methodology and tailored plasma pooling design, which blends normal source plasma and respiratory syncytial virus (RSV) plasma obtained from donors tested using the Company’s proprietary microneutralization assay. ASCENIV contains naturally occurring polyclonal antibodies, which are proteins that are used by the body’s immune system to neutralize microbes, such as bacteria and viruses and prevent against infection and disease. ASCENIV is protected by U.S. Patents: 9,107,906, 9,714,283 and 9,815,886. Certain data and other information about ASCENIV can be found by visiting . Information about ADMA and its products can be found on the Company’s website at . About BIVIGAM® BIVIGAM (immune globulin intravenous, human – 10% liquid) is a plasma-derived, polyclonal, intravenous immune globulin (IVIG). BIVIGAM was approved by the FDA in May 2019 and is indicated for the treatment of primary humoral immunodeficiency (PI), including, but not limited to, the following group of genetic disorders: X-linked and congenital agammaglobulinemia, common variable immunodeficiency, Wiskott-Aldrich syndrome and severe combined immunodeficiency. BIVIGAM contains a broad range of antibodies similar to those found in normal human plasma. These antibodies are directed against bacteria and viruses and help to protect PI patients against serious infections. BIVIGAM is a purified, sterile, ready-to-use preparation of concentrated human Immunoglobulin antibodies. Certain data and other information about BIVIGAM or ADMA and its products can be found on the Company’s website at . Cautionary Note Regarding Forward-Looking Statements This press release contains “forward-looking statements” pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995 about ADMA Biologics, Inc. and its subsidiaries (collectively, “our” or the “Company”). Forward-looking statements include, without limitation, any statement that may predict, forecast, indicate, or imply future results, performance or achievements, and may contain such words as “anticipate,” “intend,” “target,” “plan,” “expect,” “believe,” “will,” “is likely,” “will likely,” “should,” “could,” “would,” “may,” or, in each case, their negative, or words or expressions of similar meaning. These forward-looking statements also include, but are not limited to, the anticipated benefits and significance of the FDA’s room temperature storage approval. Actual events or results may differ materially from those described in this press release due to a number of important factors. Current and prospective security holders are cautioned that there also can be no assurance that the forward-looking statements included in this press release will prove to be accurate. Except to the extent required by applicable laws or rules, ADMA does not undertake any obligation to update any forward-looking statements or to announce revisions to any of the forward-looking statements. Forward-looking statements are subject to many risks, uncertainties and other factors that could cause our actual results, and the timing of certain events, to differ materially from any future results expressed or implied by the forward-looking statements, including, but not limited to, the risks and uncertainties described in our filings with the U.S. Securities and Exchange Commission, including our most recent reports on Form 10-K, 10-Q and 8-K, and any amendments thereto. COMPANYCONTACT: Skyler Bloom Senior Director, Business Development & Corporate Strategy | 201-478-5552 | sbloom@admabio.com INVESTOR RELATIONS CONTACT: Michelle Pappanastos Senior Managing Director, Argot Partners | 212-600-1902 | michelle@argotpartners.com

Before her arrival, Stefanski was an associate professor of pediatrics at the University of Minnesota and was actively involved in clinical care and research involving children with life-threatening blood and immune system disorders. One of her areas of focus was the use of cord blood units in stem cell transplants and other cellular therapy. She is board certified in both Pediatrics and Pediatric Hematology-Oncology. In the last two decades, there has been a rapid evolution in the diagnosis and treatment of primary immunodeficiencies and the recognition of immune dysregulation, could you explain a) why this is and b) what point we are at currently with the diagnosis and treatment? ​ We are more sophisticated with our molecular techniques of detecting genes in these disorders.  There are approximately 500 inborn errors of immunity causing genes and with the widely available sequencing methods this number continues to grow rapidly. Established treatment options currently consist of immune modulatory drugs, antimicrobial prophylaxis, immunoglobulin replacement therapy and, allogeneic hematopoietic stem cell transplantation (HSCT) as well as gene therapy. Could you explain what causes inborn errors of immunity? ​ Inborn errors of immunity (IEI) are caused by genetic mutations. They can be x-linked, autosomal dominant or autosomal recessive.  They can also be novel mutations that are not inherited. IEI present clinically as increased susceptibility to infections, autoimmunity, autoinflammatory diseases, allergy, bone marrow failure, and/or malignancy. googletag.cmd.push(function () { googletag.display('text-ad1'); }); Until recently, management of treatment options were limited to prompt treatment of infections, gammaglobulin replacement and even bone marrow transplant depending on what was wrong. Could you outline the newer therapies including small molecule inhibitors, biologics, gene therapy etc? ​ In all types of gene therapy for IEI, ex vivo ​ gene therapy is used, in which a viral vector  is used to genetically modify cells from the patients with therapeutic intent. Gene editing can also be used where the DNA is repaired. Gene therapy has been used for multiple IEI including X-linked SCID, Artemis SCID, X-SCID, Wiskott-Aldrich Syndrome and Chronic Granulomatous Disease. What difference have these made to the patients? ​ These patients live longer, healthier lives and have a much better quality of life.  They are not hospitalized as frequently, they can attend school, they no longer need transfusions.  These therapies are life changing. What efforts have been made to increase awareness of IEI? ​ The advancement of genetic testing and diagnostic techniques have increased the awareness of IEI.  There are educational efforts to recruit young immunologists to the field as well as encouraging international collaborations. The development and widespread use of newborn screening have helped to identify severe combined immune deficiency (SCID) has also increased awareness.  These patients are identified immediately and have improved outcomes.  There are continual improvements and accessibility of genetic sequencing has accelerated identification of new IEI diseases. Advances and improvements in both gene therapy and hematopoietic stem cell transplant have made treatments possible in otherwise fatal diseases. Could you explain ‘newborn screening’ and the advantages or any disadvantages this may have? ​ Newborn screening identifies conditions that can affect a child’s long-term health or survival and is done within 24 hours after birth. Early detection, diagnosis, and intervention can prevent death or disability and enable children to reach their full potential.  The advantages are detecting life-threatening disorders early will impact that child’s health. Each year, new disorders, including IEI, can be detected on newborn screen.  The number of disorders detected varies by state. Where would you like to see this field in terms of development within the next decade or two? ​ A number of gene therapy trials using viral gene addition have been successful for ADA-SCID. Importantly, gene therapy approaches have become safer, more efficient and applicable to more IEI. Techniques for gene therapy continue to evolve and challenges include targeting the specific gene and minimizing the off-targets mutagenesis. The biggest obstacles remain cost and complex regulatory pieces. Where is the industry at with inborn errors of immunity? ​ We are continuing to improve gene therapy and expanding it to other IEIs.  We are making hematopoietic stem cell transplant safer for those that require transplant. The treatment for these disorders will change significantly in the next 10-20 years with more patients receiving gene therapy and additional IEIs being treated with gene therapy. We are truly on the cusp of novel therapies for these patients.

YOGIN, South Korea, Dec. 17, 2023 /PRNewswire/ -- GC Biopharma Corp (006280.KS) today announces that the US Food and Drug Administration (FDA) has approved ALYGLO (immune globulin intravenous, human-stwk) 10% Liquid, formerly referred to as "GC5107," for the treatment of adult patients aged 17 years and older with primary humoral immunodeficiency (PI). The ALYGLO pivotal phase 3 clinical study followed FDA guidance for the treatment of patients with PI. The clinical trial (NCT02783482) GC5107B was a prospective, open-label, single-arm, historically controlled, multicenter phase 3 study to assess the efficacy and safety of GC5107B in patients with a confirmed diagnosis of PI. The studies were conducted in the United States and Canada. Key findings from the phase 3 clinical trial for patients aged 17 years and older include the following: A primary efficacy end point of 0.03 acute serious bacterial infections (aSBIs) per patient-year, which met the FDA efficacy requirement of less than one aSBI per patient-year. The proportion of infusions with temporally associated adverse events occurring during or within 72 hours after infusion was 0.22 (95% one-sided upper confidence bound: 30%), which met the FDA-required prespecified end point of less than 0.40. Secondary analyses were annual rate or days of other infections, antibiotic use, days out of work/school/day care or unable to perform normal activities due to infection, and days of hospitalization due to infection. ­­The results of the phase 3 trial by GC Biopharma are published in Frontiers in Immunology (2021). Dr. Elena Perez, MD., Allergy Immunologist from Allergy Associates of the Palm Beaches, and lead study investigator, said, "Alyglo will significantly impact clinical practice in the U.S. due to its strong safety and efficacy profile. It is an important treatment option for primary immune-deficient patients. This product provides additional confidence in treatment options for this important patient population." In addition, ALYGLO uses its novel Cation Exchange Chromatography (CEX) in the manufacturing process for removing coagulation factor XIa (FXIa) to undetectable levels. The presence of residual activated FXIa in some commercial IGIV products has been identified as the root cause of a small number of thromboembolic events in patients receiving immunoglobulin infusions. A recent study published in Frontiers in Cardiovascular Medicine details the extensive testing of ALYGLO and its results of the GC Biopharma manufacturing process. GC Biopharma is committed to evaluating how this new manufacturing process serves to further improve product safety. Dr. Eun-chul Huh, President and CEO of GC Biopharma, said, "The approval of ALYGLO by the US FDA is a significant milestone for GC Biopharma and patients with primary immunodeficiency disease. It reinforces GC Biopharma's commitment to patients and health care professionals by expanding our product portfolio globally to serve individuals with rare diseases. We are pleased to offer patients another important treatment option in the coming months that is both safe and effective." For any medical or scientific questions regarding ALYGLO, call the GC Biopharma USA medical department at 1-833-426-6426, or email [email protected]. 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 three 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 PI 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. People with PI might experience frequent infections, some of which can be severe or chronic. Treatment often involves immunoglobulin replacement therapy to boost the immune system. Vaccinations and antibiotics may also be used to prevent and treat infections. The exact type and severity of PI can vary widely among individuals. 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 and has been dedicated to quality health care 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 extends its footprint, bringing its expertise and legacy to the United States. This press release may contain forward-looking statements that express the current beliefs and expectations of GC Biopharma Corp. management. Such statements do not represent any guarantee by GC Biopharma Corp. or its management of future performance and involve known and unknown risks, uncertainties, and other factors. GC Biopharma Corp. 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 WARNING: THROMBOSIS, RENAL DYSFUNCTION and ACUTE RENAL FAILURE Thrombosis may occur with immune globulin intravenous (IGIV) products, including ALYGLO. Risk factors may include: advanced age, prolonged immobilization, hypercoagulable conditions, history of venous or arterial thrombosis, use of estrogens, indwelling vascular catheters, hyperviscosity, and cardiovascular risk factors. Renal dysfunction, acute renal failure, osmotic nephropathy, and death may occur with the administration of IGIV products in predisposed patients. Renal dysfunction and acute renal failure occur more commonly in patients receiving IGIV products containing sucrose. ALYGLO does not contain sucrose. For patients at risk of thrombosis, renal dysfunction or renal failure, administer ALYGLO at the minimum dose and infusion rate practicable. Ensure adequate hydration in patients before administration. Monitor for signs and symptoms of thrombosis and assess blood viscosity in patients at risk for hyperviscosity. 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. For more information about ALYGLO, please see full Prescribing Information. GC Biopharma Contact Seunghyun Baek [email protected] Sohee Kim [email protected] Yelin Jun [email protected] GC Biopharma USA Contact (Media) Robin Zvonek [email protected] (312) 636-5744 ©2023 GC Biopharma USA, Inc. All rights reserved. ALYGLO is a trademark of Green Cross Corporation. SOURCE GC Biopharma