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Inozyme Pharma, Inc. (Nasdaq: INZY) is a clinical-stage biopharmaceutical company dedicated to developing novel therapeutics aimed at treating rare metabolic diseases. Focusing on disorders linked to paradoxical mineralization, Inozyme is pioneering solutions for conditions impacting vasculature, soft tissue, and skeletal health. Their lead drug, INZ-701, is an enzyme replacement therapy designed to correct defects in pathways involving ENPP1 and ABCC6 deficiencies.
INZ-701 showcases promise in treating diseases such as pseudoxanthoma elasticum (PXE) and generalized arterial calcification of infancy (GACI). This drug, currently in Phase 1/2 clinical trials, has demonstrated positive preliminary results, including safety, pharmacokinetic, and pharmacodynamic data, as well as trends toward clinical improvement. The trials indicate that INZ-701 increases plasma pyrophosphate (PPi) levels, a crucial marker for these conditions.
During the trials, INZ-701 was generally well tolerated with favorable safety profiles. Inozyme has undertaken comprehensive natural history studies to better understand the progression of ABCC6 Deficiency, especially in pediatric populations at high risk for severe complications like strokes.
In addition to their clinical advancements, Inozyme is committed to expanding its pipeline and therapeutic focus, planning pivotal trials for both pediatric and adult populations affected by these genetic disorders. The company also actively engages with patient communities and global health institutions to further research and raise awareness.
For real-time updates and detailed information, visit Inozyme Pharma or follow them on LinkedIn, X (formerly Twitter), and Facebook.
Inozyme Pharma announced that the FDA has granted Fast Track designation to INZ-701 for treating ABCC6 Deficiency, a rare, severe condition affecting children and leading to significant clinical events such as stroke and severe neurological and cardiovascular diseases. The decision is based on nonclinical pharmacology data and preliminary safety and efficacy data from the ongoing Phase 1/2 trial in adults. This designation will facilitate more frequent FDA engagement and expedited regulatory review. Inozyme aims to finalize plans for a pivotal pediatric study by the end of 2024.
Inozyme Pharma (Nasdaq: INZY), a clinical-stage biopharmaceutical company, will present findings from a radiographic study on pediatric patients with ENPP1 Deficiency at the 11th International Conference on Children’s Bone Health (ICCBH) in Salzburg, Austria, from June 22-25, 2024.
The presentation titled, 'Expanding the spectrum of radiographic features in children with ENPP1 Deficiency: Novel skeletal findings from an international collaboration,' will be delivered by Dr. Leanne Ward on June 24, 2024, between 11:00-12:00 CEST. Additionally, Inozyme will sponsor a symposium on June 22, 2024, from 13:45-14:45 CEST, focusing on ENPP1 Deficiency as an overlooked cause of hypophosphatemic rickets, chaired by Dr. Agnes Linglart and featuring presentations by Dr. Zulf Mughal and Dr. David Weber.
Inozyme Pharma (Nasdaq: INZY), a clinical-stage biopharmaceutical company specializing in rare diseases, announced that its CEO and Chairman, Doug Treco, Ph.D., will present at the Jefferies Global Healthcare Conference. The presentation is scheduled for Thursday, June 6, 2024, from 12:30 to 12:55 pm ET. A live webcast will be available on the Investor Relations section of Inozyme's website, with a replay accessible for a time.
Inozyme Pharma (Nasdaq: INZY) announced that it will present topline data from its Phase 1/2 clinical trials of INZ-701 in adults with ENPP1 Deficiency and ABCC6 Deficiency at two upcoming medical conferences. The presentations will occur at the European Calcified Tissue Society Congress (ECTS) 2024 in Marseille, France, from May 25-28, and at the Endocrine Society's Annual Meeting (ENDO) 2024 in Boston, Massachusetts, from June 1-4. Key topics include the impact of INZ-701 on bone and mineral metabolism biomarkers and clinical outcomes in adults with ENPP1 Deficiency, as well as the safety and exploratory efficacy of INZ-701 in adults with ABCC6 Deficiency manifesting as pseudoxanthoma elasticum (PXE).
Inozyme Pharma, Inc. (Nasdaq: INZY) will participate in the Bank of America Securities 2024 Health Care Conference with CEO Doug Treco. The company focuses on developing therapeutics for rare diseases related to mineralization and intimal proliferation.
Inozyme Pharma, Inc. (Nasdaq: INZY) reported positive interim data from Phase 1 trials of INZ-701 in end-stage kidney disease and ENPP1 Deficiency. Cash on hand expected to fund operations into Q4 2025. The company aims to address unmet needs in ABCC6 Deficiency and calciphylaxis. Financially, cash, cash equivalents, and short-term investments were $166.2 million, with R&D expenses at $19.1 million and net loss of $23.3 million for Q1 2024.
Inozyme Pharma, Inc. (Nasdaq: INZY) granted stock options to a new employee under Nasdaq Listing Rule 5635(c)(4). The options allow the purchase of up to 20,000 shares at $4.61 per share, vesting over four years.
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