Inozyme Pharma ( INZY) is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton
The Company’s lead product candidate, INZ-701, is a soluble, recombinant, or genetically engineered, fusion protein that is designed to correct a defect in the mineralization pathway caused by ENPP1 and ABCC6 deficiencies.
INZY is developing INZ-701, a potential first-in-class enzyme therapy, to address pathologic mineralization and intimal proliferation which can drive morbidity and mortality in these severe diseases.
Company estimates addressable patient population with ENPP1 Deficiency at 37,000 worldwide; 2,800 in North America, 4,100 in Europe and 900 in Japan
Cash, cash equivalents, and investments were $141.5 million as of September 30, 2022. Based on its current plans, the Company expects that its cash, cash equivalents, and investments together with the remainder of the first tranche of its debt facility as of September 30, 2022 will enable the Company to fund its cash flow requirements into the second quarter of 2024.
Upcoming INZY Catalysts (Q1 2023)
INZ-701: ABCC6 deficiency: Phase 1/2 trial initiated, noted April 12, 2022. Preliminary safety and biomarker data reported that all three subjects showed rapid and significant increases in PPi levels, noted July 19, 2022. Dosing in next cohort planned for 3Q 2022 and topline data in the 1Q 2023.
INZ-701: ENPP1 deficiency: Phase 1/2 trial initiated November 15, 2021. Phase 1/2 portion 1 preliminary biomarker, safety, and pharmacokinetic (PK) data from the first three patients treated showed rapid, significant, and sustained increases in PPi levels, noted April 4, 2022. Open label extension dosing commenced November 3, 2022. Topline data expected in 1Q 2023.
ENPP1 Deficiency is a progressive condition that manifests as a spectrum of diseases. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI), which is characterized by extensive vascular calcification and neointimal proliferation (overgrowth of smooth muscle cells inside blood vessels), resulting in myocardial infarction, stroke, or cardiac or multiorgan failure. Approximately 50% of infants with ENPP1 Deficiency die within six months of birth. Children with ENPP1 Deficiency typically experience rickets, a condition also known as autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adults experience osteomalacia (softened bones), and they can exhibit a range of signs and symptoms that include hearing loss, arterial calcification, and cardiac and/or neurological involvement. There are no approved therapies for ENPP1 Deficiency**.**
@Fllwoman raised a good point about to review more the design clinical trial with the focus on the size (#particpants) of the trial as a small group increase significant the risk of failure.
INZ-701 in ENPP1 Deficiency Phase 1/2 Clinical Trial Design
The ongoing Phase 1/2 open-label clinical trial is expected to enroll up to nine adult patients with ENPP1 Deficiency at sites in North America and Europe. The trial will primarily assess the safety and tolerability of INZ-701 in adult patients with ENPP1 Deficiency, as well as characterize the pharmacokinetic (PK) and pharmacodynamic (PD) profile of INZ-701, including evaluation of plasma pyrophosphate (PPi) and other biomarker levels. In the Phase 1 dose-escalation portion of the trial, Inozyme is assessing INZ-701 for 32-days at doses of 0.2 mg/kg, 0.6 mg/kg, and 1.8 mg/kg administered via subcutaneous injection twice weekly, with three patients per dose cohort. Doses were selected based on preclinical studies and PK/PD modeling. The Phase 1 dose-escalation portion of the trial seeks to identify a safe, tolerable dose that increases PPi levels, and that can be used for further clinical development. The open-label Phase 2 extension portion of the trial is assessing long-term safety, pharmacokinetics, and pharmacodynamics of continued treatment with INZ-701 for up to 48 weeks, where patients may receive doses of INZ-701 at home depending on site-specific protocols. Exploratory endpoints will include evaluations of skeletal, vascular, physical function and patient-reported outcomes.
So super high risk here.
Update on the Position: Covered cost basis on Shares & Calls given the 100% run up in the last month.
…will host an Investor and Analyst Event on Thursday, Feb. 16, 2023 at 8 a.m. ET. During the webcast and conference call members of Inozyme management will review topline pharmacokinetic, pharmacodynamic (PK/PD), and safety data from the ongoing Phase 1/2 clinical trials of INZ-701 in ENPP1 Deficiency and ABCC6 Deficiency (PXE).
topline data today demonstrating that INZ-701 rapidly restored PPi to normal levels in all dose cohorts in the ENPP1 Deficiency trial, with normal PPi levels sustained during the trial. Additionally, emerging patient-reported outcome data, as measured by GIC, showed six of eight patients reported concordant improvements in overall health compared to baseline on clinician-GIC and patient-GIC
Data reported today are highly encouraging and show that INZ-701 significantly increased PPi levels into normal physiological ranges with a favorable safety profile
INZ-701 has shown in our ongoing clinical trials encouraging safety data, a predictable pharmacokinetic profile, and significant pharmacodynamic activity, all of which provide a strong foundation for advancing our trials.
estimated cash position ($127.9 million as of December 31, 2022) expected to fund cash flow requirements into the fourth quarter of 2024
Given the 100% run up its only 15-20% up in pre-market. We could see as well a “sell the news” action. As i covered my CB already i am planning to hold long-term. Lets see if there sharing additional information on the call at 8am EST.
Edit: Up 30% pre-market, i am taking profits as my average price is 1.4$