Inozyme Pharma, Inc. - INZY STOCK NEWS

Inozyme Pharma presented new data revealing the significant disease burden of ENPP1 and ABCC6 Deficiencies at the ACMG annual meeting. The study showcased how these rare genetic diseases impact patients differently across age groups, highlighting severe symptoms such as cardiac issues and skeletal abnormalities. The research included responses from 38 patients across nine countries, emphasizing the need for improved diagnosis and management strategies. Inozyme is preparing to start Phase 1/2 trials for its therapy INZ-701 targeting these disorders in 2021.

Inozyme Pharma (Nasdaq: INZY) announced the presentation of a study on the burden of illness associated with infantile onset ABCC6 and ENPP1 deficiency at the ACMG Annual Clinical Genetics Meeting from April 13-16, 2021. The poster titled 'From the Voice of Patients and Caregivers: Burden of Illness in Infantile Onset ABCC6 and ENPP1 Deficiency' will be displayed starting April 14, 2021. The research focuses on these rare diseases affecting mineralization pathways. The results will be available on Inozyme’s website post-conference.

Inozyme Pharma has announced changes to its Scientific Advisory Board (SAB), welcoming three experts in vascular calcification and renal disease: W Charles O’Neill IV, Jouni Uitto, and Paul B. Yu. David Thompson, former Chief Scientific Officer, joins the SAB as well. The transition follows the departure of three members, aiming to strengthen the company's focus on rare diseases linked to mineralization abnormalities. The new appointees bring valuable expertise that will aid Inozyme in its research and development efforts.

Inozyme Pharma, Inc. (Nasdaq: INZY) received Orphan Drug Designation from the FDA for INZ-701, targeting ABCC6 deficiency. The company plans to initiate Phase 1/2 trials for both ENPP1 and ABCC6 deficiencies in 2021. As of December 31, 2020, cash reserves stood at $159.9 million, projected to fund operations into the second half of 2022. R&D expenses increased significantly to $46.5 million, driven by asset acquisitions and clinical preparations. The net loss reported was $56.4 million, translating to a loss of $5.11 per share.

Inozyme Pharma announces the feature of ENPP1 deficiency on The Balancing Act® to raise awareness about this rare, life-threatening disease with no approved treatments. The episode, airing on March 15 and March 24, 2021, will include personal stories from patients and medical professionals. The ENPP1 gene is vital for regulating mineralization and its deficiency can lead to severe health issues, especially in infants. Inozyme aims to develop therapies for these conditions, emphasizing the need for early diagnosis and proper treatment.

Inozyme Pharma (Nasdaq: INZY) announced the appointment of Dr. Deborah Wenkert as Senior Vice President and Chief Medical Officer, effective February 2, 2021. With over 20 years of experience in pediatric rheumatology and metabolic bone disorders, Dr. Wenkert will lead clinical development programs and scientific communications. She succeeds Dr. Pedro Huertas, who has left the position. Dr. Wenkert has previously held significant roles at PreciThera and Amgen, enhancing her qualifications for the new role as Inozyme advances its clinical trials for rare bone diseases, specifically ENPP1 and ABCC6 deficiencies.

Inozyme Pharma (Nasdaq: INZY) announced regulatory approvals for its investigational drug INZ-701, targeting ENPP1 deficiency. The U.S. FDA cleared its IND application, while the UK MHRA authorized its Clinical Trial Application for a Phase 1/2 trial. The company plans to enroll its first subject in H1 2021 and provide preliminary safety data in H2 2021. The trial aims to assess safety, tolerability, and pharmacokinetics of INZ-701, with a multi-center approach involving nine subjects across three dose cohorts.

Inozyme Pharma submitted a Clinical Trial Application for INZ-701 to treat ENPP1 deficiency, receiving both Rare Pediatric Disease and Fast Track Designations from the FDA. The company aims to initiate Phase 1/2 trials in H1 2021. Financially, Inozyme reported a net loss of $28.1 million for Q3 2020, increasing from $4.0 million in Q3 2019. R&D expenses surged to $25.2 million, attributed to a non-recurring acquisition and rising employee costs. Cash reserves stood at $171.7 million, expected to sustain operations into H2 2022.

Inozyme Pharma (Nasdaq: INZY) announced the appointment of Dr. Kevin B. Johnson as senior vice president of regulatory affairs. Dr. Johnson, who has over 25 years of experience in global regulatory strategies for rare diseases, will spearhead Inozyme's regulatory initiatives. His previous roles include senior vice president at Magenta Therapeutics and head of regulatory affairs at IMARA Inc., where he achieved key designations for orphan diseases. Inozyme focuses on developing treatments for rare metabolic diseases linked to mineralization disorders, particularly targeting ENPP1 and ABCC6 deficiencies.