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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 (Nasdaq: INZY) has begun dosing its first patient in a Phase 1/2 clinical trial for INZ-701, targeting ABCC6 Deficiency. Meanwhile, the second cohort in the trial for ENPP1 Deficiency is fully enrolled, with topline results anticipated in the second half of 2022. Preliminary safety and biomarker data from the ABCC6 trial are expected by Q2 2022. The trials aim to assess the safety and efficacy of INZ-701, which has shown promise in earlier models by increasing plasma pyrophosphate levels and preventing soft tissue calcification, significant factors in both conditions.
Inozyme Pharma (Nasdaq: INZY) announced that its CEO Axel Bolte will participate in a fireside chat at the 21st Annual Needham Virtual Healthcare Conference on April 13, 2022, at 8:45 AM ET. The event will provide insights into the company’s development of INZ-701, a novel therapy targeting rare genetic diseases caused by ENPP1 and ABCC6 deficiencies. A replay of the chat will be available for 30 days in the Investor & News section of Inozyme’s website.
Inozyme Pharma reported positive preliminary data from its Phase 1/2 clinical trial of INZ-701 for treating ENPP1 Deficiency, with three patients in the lowest dose cohort (0.2 mg/kg) showing significant increases in plasma pyrophosphate (PPi) levels. The PPi levels rose to a mean of 1356 nM, a 5-fold increase from baseline. INZ-701 was well-tolerated without serious adverse events. The trial is progressing to the next dose level (0.6 mg/kg), and topline data is expected in the second half of 2022, addressing a critical medical need as no approved therapies currently exist for this condition.
Inozyme Pharma (Nasdaq: INZY) reported a net loss of $56.6 million, or $2.40 per share, for the year ended December 31, 2021. R&D expenses decreased to $37.7 million from $46.5 million in 2020, while G&A expenses rose to $18.9 million. The company is advancing its clinical programs for INZ-701, with patient dosing underway for ENPP1 Deficiency and initial patient enrollment expected for ABCC6 Deficiency. Preliminary safety and biomarker data are anticipated in the first half of 2022. Cash reserves stood at $111.8 million, sufficient to fund operations into Q1 2023.
Inozyme Pharma (Nasdaq: INZY) announced the appointment of Sanjay S. Subramanian as CFO and Soojin Kim, Ph.D., as CTOO. Subramanian brings over 20 years of financial expertise, previously raising over $200 million at Ocugen. Kim, with more than 25 years in pharmaceutical R&D, will enhance the company's manufacturing operations for INZ-701, currently in Phase 1/2 trials for ENPP1 Deficiency. Both executives aim to advance Inozyme's mission in treating rare diseases caused by abnormal mineralization.
Inozyme Pharma (Nasdaq: INZY) announced that co-founder and CEO Axel Bolte will engage in a fireside chat at the Cowen 42nd Annual Health Care Conference on March 8, 2022, at 11:10 AM ET. This event highlights the company’s commitment to advancing therapies for rare genetic conditions caused by abnormal mineralization, particularly focusing on ENPP1 and ABCC6 deficiencies. A replay of the discussion will be available for 30 days post-event on the Inozyme website.
Inozyme Pharma (Nasdaq: INZY) announced the initiation of its first-in-human Phase 1/2 clinical trial for INZ-701, an enzyme replacement therapy targeting ENPP1 Deficiency. The trial's first patient has been dosed, with preliminary biomarker and safety data expected by mid-2022. This trial will evaluate the safety, tolerability, and pharmacokinetic profiles of INZ-701 in nine adult patients across the U.S. and Europe. ENPP1 Deficiency is a life-threatening condition with no approved treatments, making this milestone significant for patient care and potential market impact.
Inozyme Pharma, a clinical-stage biopharmaceutical company focused on rare diseases of abnormal mineralization, announced that co-founder and CEO Axel Bolte will present at two upcoming virtual investor conferences. The 2021 Jefferies London Virtual Healthcare Conference will feature an on-demand presentation starting November 18, 2021, at 3:00 a.m. ET, while the Piper Sandler 33rd Annual Virtual Healthcare Conference will host a fireside chat available on-demand from November 22, 2021, at 10:00 a.m. ET. Presentations will be accessible for 60 days on the company’s website.
Inozyme Pharma, Inc. (Nasdaq: INZY) reported its third-quarter 2021 financial results, highlighting progress in the INZ-701 program for ENPP1 and ABCC6 Deficiencies. The company anticipates starting Phase 1/2 clinical trials in Q4 2021, with preliminary data expected in early 2022. As of September 30, 2021, cash, cash equivalents, and investments totaled $125.3 million, expected to support operations into Q1 2023. R&D expenses decreased to $9.3 million, while G&A expenses rose to $4.9 million. The net loss improved to $14.3 million, or $0.60 per share, compared to $28.1 million, or $1.55 per share, year-over-year.
Inozyme Pharma (Nasdaq: INZY) presented findings from its ENPP1 Deficiency Natural History Study and gene therapy program at the ASBMR 2021 Annual Meeting. The study, involving 74 patients, showed that 46% had skeletal disease, with 58% experiencing cardiac issues. AAV-ENPP1 gene therapy demonstrated potential in preclinical models, preventing soft tissue calcification and restoring bone parameters. Inozyme plans to begin Phase 1/2 trials for its therapy INZ-701 within Q4 2021 to address this life-threatening condition. Currently, no approved treatments exist for ENPP1 Deficiency.
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