Thrombophilia

Submission of a Prior Approval Supplement for a reduction in the number of patients in the NEPHRO CRRT study is expected within the coming week The FDA agreed to two additional protocol changes expected to accelerate enrollment in the NEPHRO CRRT study SAN MATEO, Calif., Jan. 14, 2025 /PRNewswire/ -- Talphera, Inc. (Nasdaq: TLPH), ("Talphera"), a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings, today announced that following a meeting with the U.S. Food and Drug Administration (FDA), the agency has agreed to review a Prior Approval Supplement (PAS) requesting a reduction in the number of patients in the NEPHRO CRRT clinical study. A PAS is reviewed by the FDA within 30 days, and its approval by the agency is required before formally amending the study protocol. During this same meeting, the agency agreed to two other changes to broaden the clinical study inclusion criteria which would allow the Company to enroll patients already on continuous renal replacement therapy (CRRT) beyond 48 hours as well as heparin-tolerant patients at certain institutions. These changes are being made through a five-day protocol amendment notice to the FDA with no additional FDA review required. "We continue to evaluate and focus on opportunities to support completion of the NEPHRO CRRT study by the end of the year. In addition to new clinical sites, our continuous improvement efforts in study execution were strengthened following our positive face-to-face meeting with the FDA where the agency agreed we should submit a PAS to reduce the number of patients in the NEPHRO CRRT study. We plan to submit the PAS in the coming week which requires a review period of up to 30 days," stated Shakil Aslam, MD, Talphera's Chief Medical Officer. "Our breakthrough designation status and close collaboration with the FDA continue to support our confidence in the importance of nafamostat as an alternative anticoagulant for patients on CRRT," continued Dr. Aslam. About Talphera, Inc. Talphera, Inc. is a specialty pharmaceutical company focused on the development and commercialization of innovative therapies for use in medically supervised settings. Talphera's lead product candidate, Niyad™ is a lyophilized formulation of nafamostat and is currently being studied under an investigational device exemption (IDE) as an anticoagulant for the extracorporeal circuit, and has received Breakthrough Device Designation status from the U.S. Food and Drug Administration (FDA). This release is intended for investors only. For additional information about Talphera, please visit www.talphera.com. About Niyad and nafamostat Nafamostat is a broad spectrum, synthetic serine protease inhibitor with anticoagulant, anti-inflammatory and potential anti-viral activities. Niyad™ is a lyophilized formulation of nafamostat and is currently being studied under an IDE, as an anticoagulant for the extracorporeal circuit, and has received Breakthrough Device Designation Status from the FDA. Talphera's registrational study of Niyad™ is named the NEPHRO CRRT (Nafamostat Efficacy in Phase 3 Registrational Continuous Renal Replacement Therapy) study. An ICD-10 procedural code, XY0YX37, has been issued for the extracorporeal introduction of nafamostat. The ICD-10 code is a specific/billable code that can be used to indicate a procedure. LTX-608 is a proprietary nafamostat formulation for direct IV infusion that may be investigated and developed for the treatment of acute respiratory distress syndrome (ARDS), disseminated intravascular coagulation (DIC), acute pancreatitis or as an anti-viral treatment, amongst other potential targets. About the NEPHRO CRRT Study The NEPHRO Study, which has received central IRB approval, is designed as a prospective, double-blinded trial to be conducted at up to 14 U.S. hospital intensive care units. The study will enroll and evaluate 166 adult patients undergoing renal replacement therapy, who cannot tolerate heparin or are at risk for bleeding. The primary endpoint of the study is mean post-filter activated clotting time using Niyad versus placebo over the first 24 hours. Key secondary endpoints include filter lifespan, number of filter changes over 72 hours, number of transfusions over 72 hours and dialysis efficacy (based on urea concentration) over the first 24 hours. Forward-looking statements This press release contains forward-looking statements based upon Talphera's current expectations and assumptions. These and any other forward-looking statements are made pursuant to the safe harbor provisions of the Private Securities Litigation Reform Act of 1995. These statements may be identified by the use of forward-looking terminology such as "believe," "expect," "anticipate," "may," "if," "intends," "plans," "potential," "projected," "will," or the negative of these words or other comparable terminology, and include: Talphera's expectation that the NEPHRO study enrollment rates will accelerate due to the protocol changes, and will support the study completion by the end of 2025, and that the PAS will be submitted in the coming week. Talphera's discussion of its strategy, plans and intentions also include forward-looking statements, which are predictions, projections and other statements about future events that are based on current expectations and assumptions. These forward-looking statements involve risks and uncertainties that could cause actual results to differ materially from those projected, anticipated or implied by such statements, including: (i) risks relating to Talphera's product development activities, including that clinical studies may not be fully enrolled or completed and/or confirm any safety, efficacy or other potential developmental product characteristics described or assumed in this press release; (ii) Talphera's developmental product candidates may not be beneficial to patients or healthcare providers or be successfully commercialized; (iii) risks relating to Talphera's ability to obtain regulatory approvals for its developmental product candidates; (iv) risks related to the ability of Talphera and its business partners to implement development plans, commercial launch plans, forecasts and other business expectations; and (v) risks related to Talphera's liquidity and its ability to maintain capital resources sufficient to conduct its clinical studies. Although it is not possible to predict or identify all such risks and uncertainties, they may include, but are not limited to, those described under the caption "Risk Factors" and elsewhere in Talphera's annual, quarterly and current reports (i.e., Form 10-K, Form 10-Q and Form 8-K) as filed or furnished with the SEC and any subsequent public filings. You are cautioned not to place undue reliance on any such forward-looking statements, which speak only as of the date such statements were first made. To the degree financial information is included in this press release, it is in summary form only and must be considered in the context of the full details provided in Talphera's most recent annual, quarterly or current report as filed or furnished with the SEC. Talphera's SEC reports are available at www.talphera.com under the "Investors" tab. Except to the extent required by law, Talphera undertakes no obligation to publicly release the result of any revisions to these forward-looking statements to reflect new information, events or circumstances after the date hereof, or to reflect the occurrence of unanticipated events. View original content to download multimedia:https://www.prnewswire.com/news-releases/talphera-announces-agreement-with-the-fda-for-prior-approval-supplement-review-to-reduce-the-number-of-patients-in-the-nephro-crrt-study-302349947.html SOURCE Talphera, Inc. Investor Contacts: Talphera, Raffi Asadorian, CFO, 650-216-3500, investors@talphera.com; LifeSci Advisors, Kevin Gardner, 617-283-2856, kgardner@lifesciadvisors.com; Chris Calabrese, 917-680-5608, ccalabrese@lifesciadvisors.com

TGD001 is being developed to offer patients with clotting disorders a faster, safer and more effective treatment than today's standard of care – saving lives and reducing disability Following the Phase 1 safety study, TargED intends to further develop TGD001 in patients with AIS and iTTP (a rare micro-thrombotic disorder) in the second half of 2025 UTRECHT, the Netherlands, Dec. 19, 2024 /PRNewswire/ -- TargED Biopharmaceuticals ("TargED"), a private biotechnology company focused on developing improved treatments for thrombotic diseases, today announces the start of clinical development with the dosing of the first participant with TGD001. TGD001 is a groundbreaking thrombolytic designed to break down clots of all sizes and compositions – from large clots which cause AIS, down to 'micro-clots' which cause conditions such as the rare, life-threatening disorder immune-mediated thrombotic thrombocytopenic purpura (iTTP). The first study participant was successfully dosed yesterday [Dec 18] at a Phase 1 clinic in Germany. The study's design is a randomized, double-blind, placebo-controlled single-ascending dose study to evaluate TGD001's safety, tolerability and pharmacokinetics in healthy volunteers (EU CT ID number: 2024-514931-63-00). First safety read-out from the Phase 1 study is expected by mid-2025. TargED plans to continue clinical development with two proof-of-concept trials in the second half of 2025: a Phase 2a trial in AIS patients and a Phase 1b trial in iTTP patients, for which TargED received EU Orphan Drug Designation in July 2024 from the European Commission. Kristof Vercruysse, Chief Executive Officer and co-founder of TargED, said: "The dosing of the first participant with our lead compound TGD001 marks a watershed in the treatment of thrombotic disorders. Rapid treatment with highly effective thrombolytics that are well tolerated and don't exaggerate the risk of bleeding is essential to reduce the risk of serious disability or even death from thrombotic disorders. Today's thrombolytics show efficacy but their significant drawbacks greatly limit their safety and effectiveness, and result in restricted utility. TGD001 is specifically designed to address these shortcomings. Its unique mode of action allows for blood clots of all sizes and compositions to be targeted and rapidly dissolved without increasing bleeding risk, resulting in clinical benefit for a broad population of patients." Steven de Maat, Chief Scientific Officer and co-founder of TargED, said: "Acute ischemic stroke is one of the biggest causes of preventable death and disability in the world today, but up to 80% of AIS patients cannot be treated with currently available thrombolytics such as intravenous alteplase (tPA). Meanwhile, there is significant room for improvement in the treatment of iTTP. Despite recent advances, 15% of iTTP patients who receive treatment do not survive, and half of those who live still suffer long term health impairment. TGD001 has the potential to greatly improve outcomes in both these indications and, we believe, in other thrombotic indications as well." TGD001 is a first-in-class 'fusion protein' drug with a unique two-step mode of action. It targets clots via an antibody fragment that binds to a non-functional domain of von Willebrand Factor (VWF), a protein present in all types of thrombi. Then, in the immediate vicinity of the thrombus, it activates the endogenous enzyme system that degrades both VWF and fibrin, two key clotting ingredients which form the structural bond in the thrombus. This mode of action enables rapid and effective thrombolysis without systemic activation of the lysis cascade and the resulting bleeding risk. TGD001 is administered as short or bolus injection, making it a suitable therapeutic for the emergency, in-patient and first-response setting. About TargED Biopharmaceuticals TargED Biopharmaceuticals B.V. is a Netherlands based biotechnology company developing first-in-class biological drugs to improve treatment of thrombosis. TargED stands for Targeted Enzyme Delivery. TargED's biological drugs are unique by using antibody fragments ("VhH") to deliver enzymes to sites of thrombosis, enabling 'targeted' thrombolysis. Its lead compound TGD001 is currently under development for the treatment of immune-mediated thrombotic thrombocytopenic purpura (iTTP) and acute ischemic stroke (AIS). The objective is to accelerate thrombolysis in all forms of thrombosis, irrespective of the thrombus composition. TargED, a spin-off of the University Medical Center Utrecht, was founded in July 2020. Since then, TargED raised more than €45 million of funding, supporting its growth to become a clinical-stage company. For more information please visit Acute Ischemic Stroke (AIS), which occurs when one or more clots block blood flow to part of the brain, accounts for around 90% of all strokes. Worldwide, around 18 million AIS incidents take place every year, resulting in 5 million deaths and 5 million people permanently disabled. A delay in diagnosis and treatment can lead to loss of brain tissue, causing permanent disability or death. Standard of care thrombolysis must be initiated within 4.5 hours of ischemic stroke, since after this time the risk of tPA-induced bleeding outweighs the potential benefit. Due to their limitations, up to 80% of AIS patients are ineligible for current thrombolytics like tPA. Immune-mediated thrombotic thrombocytopenic purpura (iTTP) is a rare, life-threatening blood disorder characterized by sudden episodes of clot formation in small blood vessels throughout the body. Blood flow to organs such as the kidney, heart, and brain is disrupted, causing functional failure. There are about 7,500 incidents of iTTP a year worldwide and it particularly affects young women, sometimes after pregnancy. Without treatment, up to 90% of cases are fatal. Treatment in specialized centers cuts mortality to 15%; however, of the survivors, half still suffer long-term complications. Current treatment is complex, involving blood plasma exchange, immune suppression and administration of the drug caplacizumab, which stops new clot formation. To date, no thrombolytic – i.e. a drug that clears existing thrombi – is licensed in iTTP due to the perceived inherent bleeding risk of patients with thrombotic microangiopathies and the systemic thrombolytic activity of currently licensed thrombolytics. TGD001 has the potential to resolve iTTP attacks very rapidly, saving organ tissue and preventing disability and death. SOURCE TargED WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

New packaging solution enhances sustainability, workflow efficiency, and patient throughput PRINCETON, N.J., Dec. 16, 2024 /PRNewswire/ -- Bracco Diagnostics Inc. (BDI), a U.S. subsidiary of Bracco Imaging S.p.A., one of the world's leading companies in the diagnostic imaging business, and a pioneer in healthcare innovation, proudly unveils its latest breakthrough in barium solutions: the "Mini" Packaging for VARIBAR® (barium sulfate) oral suspension. Designed to meet the diverse needs of healthcare providers, this packaging option offers smaller volumes of VARIBAR products, the gold standard in Modified Barium Swallow Studies (MBSS). Mini packaging will be available for VARIBAR® NECTAR (barium sulfate) oral suspension, VARIBAR® THIN HONEY (barium sulfate) oral suspension, VARIBAR® HONEY (barium sulfate) oral suspension, and VARIBAR® PUDDING (barium sulfate) oral paste. Time-tested, trusted, and FDA-approved, the legacy of standardized viscosities of the VARIBAR product line helps eliminate the unpredictability of varied barium preparations.1-4 The "Mini" Packaging is a significant milestone as it enhances procedural efficiency and patient care. "The VARIBAR line of products, with standardized viscosities, consistently addresses the most important needs in Modified Barium Swallow Studies," said Cosimo De Pinto, Senior Vice President of Sales and Marketing at BDI. "Now, with this new additional packaging solution, VARIBAR products are available to clinicians in sustainable, smaller volume options." The key features of this innovative packaging include: Tailored Solutions: Healthcare providers can now access VARIBAR products in smaller quantities, optimizing resource utilization and reducing waste. Sustainability: BDI prioritizes using recyclable and biodegradable materials in manufacturing the "Mini" packaging, as it promotes a circular economy thereby reducing reliance on single-use plastics. Streamlined Workflow: The "Mini" Packaging facilitates smoother administration of VARIBAR contrast, enhancing workflow efficiency and patient throughput. "This new packaging minimizes product waste, thereby reducing the environmental footprint of healthcare procedures," said Amy King, Director of Sustainability at BDI. "Compact packaging optimizes storage space and transportation efficiency, further reducing consumption and emissions." By offering VARIBAR products in a more versatile and accessible format, BDI continues to invest in the future of abdominal radiology. The "Mini" Packaging for VARIBAR products is now available for order from all BDI wholesalers and distributors. For more information, please visit . Please see Important Safety Information below. VARIBAR® (barium sulfate) INDICATIONS VARIBAR® THIN HONEY (barium sulfate) oral suspension, VARIBAR® NECTAR (barium sulfate) oral suspension, and VARIBAR® THIN LIQUID (barium sulfate) oral suspension are radiographic contrast agents indicated for use in modified barium swallow examinations to evaluate the oral and pharyngeal function and morphology in adult and pediatric patients. VARIBAR® HONEY (barium sulfate) oral suspension and VARIBAR® PUDDING (barium sulfate) oral paste are radiographic contrast agents indicated for use in modified barium swallow examinations to evaluate the oral and pharyngeal function and morphology in adult and pediatric patients 6 months of age and older. IMPORTANT SAFETY INFORMATION CONTRAINDICATIONS These products should not be used in patients with known or suspected perforation of the gastrointestinal (GI) tract; known obstruction of the GI tract; high risk of GI perforation such as those with a recent GI perforation, acute GI hemorrhage or ischemia, toxic megacolon, severe ileus, post GI surgery or biopsy, acute GI injury or burn, or recent radiotherapy to the pelvis; high risk of aspiration such as those with known or suspected tracheo-esophageal fistula or obtundation; known severe hypersensitivity to barium sulfate or any of the excipients of the product used. WARNINGS AND PRECAUTIONS Hypersensitivity Reactions Barium sulfate preparations contain a number of excipients, including natural and artificial flavors, and may induce serious hypersensitivity reactions. The manifestations include hypotension, bronchospasm and other respiratory impairments, and dermal reactions including rashes, urticaria, and itching. A history of bronchial asthma, atopy, food allergies, or a previous reaction to a contrast agent may increase the risk for hypersensitivity reactions. Emergency equipment and trained personnel should be immediately available for treatment of a hypersensitivity reaction. Intra-abdominal Barium Leakage The use of VARIBAR PRODUCTS is contraindicated in patients at high risk of perforation of the GI tract. Administration of VARIBAR PRODUCTS may result in leakage of barium from the GI tract in the presence of conditions such as carcinomas, GI fistula, inflammatory bowel disease, gastric or duodenal ulcer, appendicitis, or diverticulitis, and in patients with a severe stenosis at any level of the GI tract, especially if it is distal to the stomach. The barium leakage has been associated with peritonitis and granuloma formation. Delayed Gastrointestinal Transit and Obstruction Orally administered barium sulfate may accumulate proximal to a constricting lesion of the colon, causing obstruction or impaction with development of baroliths (inspissated barium associated with feces) and may lead to abdominal pain, appendicitis, bowel obstruction, or rarely perforation. Patients with the following conditions are at higher risk for developing obstruction or baroliths: severe stenosis at any level of the GI tract, impaired GI motility, electrolyte imbalance, dehydration, on a low residue diet, taking medications that delay GI motility, constipation, pediatric patients with cystic fibrosis or Hirschsprung disease, and the elderly. To reduce the risk of delayed GI transit and obstruction, patients should maintain adequate hydration after the barium sulfate procedure. When administering VARIBAR PUDDING, consider the administration of laxatives. Aspiration Pneumonitis The use of VARIBAR PRODUCTS is contraindicated in patients with trachea-esophageal fistula. Oral administration of barium is associated with aspiration pneumonitis, especially in patients with a history of food aspiration or with compromised swallowing mechanism. Vomiting following oral administration of barium sulfate may lead to aspiration pneumonitis. In patients at risk for aspiration, begin the procedure with a small ingested volume of VARIBAR PRODUCTS. Monitor the patient closely for aspiration, discontinue administration of VARIBAR PRODUCTS if aspiration is suspected, and monitor for development of aspiration pneumonitis. Systemic Embolization Barium sulfate products may occasionally intravasate into the venous drainage of the GI tract and enter the circulation as a "barium embolus" leading to potentially fatal complications which include systemic and pulmonary embolism, disseminated intravascular coagulation, septicemia and prolonged severe hypotension. Although this complication is exceedingly uncommon after oral administration of a barium sulfate suspension, monitor patients for potential intravasation when administering barium sulfate. ADVERSE REACTIONS The most common adverse reactions are nausea, vomiting, diarrhea, and abdominal cramping. Serious adverse reactions and fatalities include aspiration pneumonitis, barium sulfate impaction, intestinal perforation with consequent peritonitis and granuloma formation, vasovagal and syncopal episodes. You are encouraged to report negative side effects of prescription drugs to the FDA. Visit or call 1-800-FDA-1088. Please click here for full Prescribing Information for VARIBAR® THIN LIQUID (barium sulfate) oral suspension. Please click here for full Prescribing Information for VARIBAR® THIN HONEY (barium sulfate) oral suspension. Please click here for full Prescribing Information for VARIBAR® NECTAR (barium sulfate) oral suspension. Please click here for full Prescribing Information for VARIBAR® HONEY (barium sulfate) oral suspension. Please click here for full Prescribing Information for VARIBAR® PUDDING (barium sulfate) oral paste. VARIBAR is manufactured by E-Z-EM Canada Inc., for E-Z-EM, Inc., a subsidiary of Bracco Diagnostics Inc., Princeton, NJ 08540. VARIBAR is a registered trademark of E-Z-EM, Inc. For additional information about Bracco's products, and for full prescribing information, please visit . About Bracco Imaging Bracco Imaging S.p.A. ("Bracco Imaging"), part of the Bracco Group, is an innovative world leader delivering end-to-end products and solutions through its comprehensive portfolio across diagnostic imaging modalities. Headquartered in Milan, Italy, Bracco Imaging's purpose is to improve people's lives by shaping the future of prevention and precision diagnostic imaging. The Bracco Imaging portfolio includes products and solutions for all key diagnostic imaging modalities: X-ray imaging, magnetic resonance imaging (MRI), Contrast Enhanced Ultrasound (CEUS), and Nuclear Medicine through radioactive tracers and novel PET imaging agents. Bracco Imaging has 3,800 employees and operates in more than 100 markets globally. Bracco Imaging has a well-skilled and innovative Research and Development (R&D) organization with an efficient process-oriented approach and track record in the diagnostic imaging industry. Discover Bracco at Bracco Diagnostics Inc. Media Relations (USA) Kimberly Gerweck Senior Compliance & Communications Manager [email protected] D: +1 609-524-2777 ______________________________ Hazelwood RJ, Armeson KE, Hill EG, et al. Identification of swallowing tasks from a modified barium swallow study that optimize the detection of physiological impairment. J Speech Lang Hear Res. 2017 Jul;60(7):1855-1863. Hind J, Divyak E, Zielinski J, et al. Comparison of standardized bariums with varying rheological parameters on swallowing kinematics in males. J Rehabil Res Dev. 2012;49(9):1399-1404. doi: 10.1682/jrrd.2011.09.0180. Robbins J, Nicosia M, Hind JA, et al. Defining physical properties of fluids for dysphagia evaluation and treatment. Perspectives on Swallowing and Swallowing Disorders (dysphagia). 2002 Jun;11(2):16-19. Martin-Harris, B., Humphries, K., Garand, K.L. The Modified Barium Swallow Impairment Profile (MBSImP™©) – Innovation, Dissemination and Implementation. Perspectives of the ASHA Special Interest Groups. 2017 Dec;2(4):129-138. SOURCE Bracco Group WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED