Inozyme Pharma
- Country
- 🇺🇸United States
- Ownership
- Public
- Established
- 2016-01-01
- Employees
- 59
- Market Cap
- $367.7M
- Website
- http://www.inozyme.com
- Introduction
Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company. It engages in the business of developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton. The company was founded by Axel Bolte, Joseph P. Schlessinger, and Demetrios T. Braddock in September 2015 and is headquartered in Boston, MA.
Clinical Trials
14
Trial Phases
3 Phases
Drug Approvals
0
Drug Approvals
No drug approvals found
This company may not have drug approvals in our database
Clinical Trials
Distribution across different clinical trial phases (11 trials with phase data)• Click on a phase to view related trials
The ENABLE Study: Safety and Efficacy Study of INZ-701 in Patients With ENPP1 Deficiency
- Conditions
- Ectonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic RicketsGeneralized Arterial Calcification of Infancy 1
- Interventions
- First Posted Date
- 2024-12-18
- Last Posted Date
- 2025-05-01
- Lead Sponsor
- Inozyme Pharma
- Target Recruit Count
- 40
- Registration Number
- NCT06739980
ADAPT Study: Long-term Safety Study of INZ-701 in Patients With ENPP1 Deficiency and ABCC6 Deficiency
- Conditions
- Gene MutationsPseudoxanthoma ElasticumArterial CalcificationEctonucleotide Pyrophosphatase/phosphodiesterase1 DeficiencyAutosomal Recessive Hypophosphatemic Rickets Type 2
- Interventions
- First Posted Date
- 2024-06-17
- Last Posted Date
- 2024-11-28
- Lead Sponsor
- Inozyme Pharma
- Target Recruit Count
- 200
- Registration Number
- NCT06462547
- Locations
- 🇺🇸
Mayo Clinic, Rochester, Minnesota, United States
🇺🇸Clinilabs Drug Development Corporation, Eatontown, New Jersey, United States
🇫🇷Necker-Enfants Malades Hospital, Paris, France
PROPEL - A Prospective Observational Patient Registry to Evaluate ENPP1 and ABCC6 Deficiency
- Conditions
- Ectonucleotide Pyrophosphatase/Phosphodiesterase 1 DeficiencyATP-Binding Cassette Subfamily C Member 6 Deficiency
- First Posted Date
- 2024-03-08
- Last Posted Date
- 2025-06-10
- Lead Sponsor
- Inozyme Pharma
- Target Recruit Count
- 1000
- Registration Number
- NCT06302439
- Locations
- 🇨🇦
CHU Sainte-Justine Research Centre, Montreal, Quebec, Canada
🇯🇵The University of Tokyo Hospital, Tokyo, Japan
🇴🇲Royal Hospital Muscat, Muscat, Oman
The SEAPORT 1 Study: Evaluation of the Safety and Tolerability of INZ-701 in Adults With End-Stage Kidney Disease Undergoing Hemodialysis
- First Posted Date
- 2024-02-28
- Last Posted Date
- 2025-02-05
- Lead Sponsor
- Inozyme Pharma
- Target Recruit Count
- 11
- Registration Number
- NCT06283589
- Locations
- 🇺🇸
South Florida Nephrology Research, Coral Springs, Florida, United States
🇺🇸Elixia Health, Hollywood, Florida, United States
The ENERGY 3 Study: Evaluation of Efficacy and Safety of INZ-701 in Children With ENPP1 Deficiency
- Conditions
- Ectonucleotide Pyrophosphatase/Phosphodiesterase1 DeficiencyGeneralized Arterial Calcification of InfancyAutosomal Recessive Hypophosphatemic Rickets
- Interventions
- Drug: Control Arm (Conventional Therapy)
- First Posted Date
- 2023-09-21
- Last Posted Date
- 2025-05-01
- Lead Sponsor
- Inozyme Pharma
- Target Recruit Count
- 27
- Registration Number
- NCT06046820
- Locations
- 🇺🇸
Children's Hospital of Colorado, Aurora, Colorado, United States
🇦🇺Queensland Children's Hospital, South Brisbane, Australia
🇸🇦King Faisal Specialist Hospital and Research Centre, Riyadh, Saudi Arabia
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News
BioMarin Acquires Inozyme Pharma for $270 Million, Adding Late-Stage Enzyme Therapy for Rare Genetic Disorder
BioMarin Pharmaceutical has entered into a definitive agreement to acquire Inozyme Pharma for $270 million, strengthening its enzyme therapy portfolio with the addition of INZ-701.
Inozyme's INZ-701 Shows Promise in ENPP1 Deficiency Trial with Sustained Phosphate Increases
Interim data from Inozyme Pharma's ENERGY 3 pivotal trial demonstrates INZ-701's potential to modify disease course in pediatric ENPP1 Deficiency patients, with sustained phosphate increases and favorable safety profile.
Big Pharma M&A Activity Surges as Companies Target Undervalued Biotechs Amid Market Recovery
Pharmaceutical M&A activity is experiencing a significant rebound with major deals including Novartis's $1.7 billion acquisition of Regulus Therapeutics and Merck KGaA's $3.9 billion purchase of SpringWorks Therapeutics.
Congress's Failure to Renew Priority Review Voucher Program Threatens Rare Disease Drug Development
The FDA's rare pediatric disease Priority Review Voucher program expired in December 2023, despite unanimous congressional support for renewal, leaving biotech companies unable to receive new vouchers worth approximately $150 million.
Inozyme's INZ-701 Shows Promise in ENPP1 and ABCC6 Deficiencies
• Inozyme Pharma's INZ-701 demonstrated improved survival rates in infants with ENPP1 Deficiency, with 80% surviving beyond one year compared to a historical 50%. • The ENERGY 3 pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency has completed enrollment, with topline data expected in early 2026. • Preliminary regulatory support has been received for the ASPIRE pivotal trial of INZ-701 in children with ABCC6 Deficiency, targeting major adverse clinical events. • INZ-701 was well-tolerated in trials, showing substantial reductions or stabilization of arterial calcifications and improvements in heart function in treated patients.