Key Takeaways
- BioMarin to Acquire Inozyme Pharma for $270M
The acquisition brings INZ-701, a promising enzyme replacement therapy for ENPP1 deficiency and GACI, into BioMarin’s rare disease pipeline. - INZ-701 Shows Strong Early Results in Pediatric Trials
Positive data from the ENERGY 1 and Expanded Access Program studies highlight improved survival, reduced arterial calcifications, and favorable safety in infants with ENPP1 deficiency. - Fast Track and Pivotal Trials Signal Regulatory Momentum
INZ-701 earned FDA Fast Track designation for ABCC6 deficiency and is advancing in the pivotal ENERGY 3 and ASPIRE trials targeting broader pediatric populations.
BioMarin Pharmaceutical has announced a definitive agreement to acquire Inozyme Pharma, a clinical-stage biopharmaceutical company. The deal includes INZ-701, a late-stage enzyme replacement therapy targeting ectonucleotide pyrophosphatase/phosphodiesterase 1 (ENPP1) deficiency.
What Are the Strategic Financial Implications of BioMarin’s Inozyme Acquisition?
Under terms of the deal, BioMarin will pay approximately $270 million in an all-cash transaction at a price of $4 per share. Inozyme’s board of directors unanimously recommended that stockholders accept the tender offer and sell their shares.1
"BioMarin has been deeply committed to advancing enzyme therapies for children and adults living with serious genetic conditions for more than 25 years, and today's agreement builds on our legacy," said Alexander Hardy, president, CEO, BioMarin, in a press release. "This acquisition brings to BioMarin an important medicine that has the potential to be the first treatment for children and adults with ENPP1 Deficiency, improving care for people living with this serious condition. As BioMarin continues our transformation and delivers on our corporate strategy, we will continue to evaluate external innovation alongside internal innovation. We are in a strong financial position to bring in additional assets as we accelerate the development of medicines for patients with significant unmet need."
INZ-701 Shows Survival and Safety Benefits in ENPP1-Deficient Infants
In January, Inozyme announced positive interim results from its ENERGY 1 trial, which consisted of three infants, and Expanded Access Program (EAP), which included two infants and one child 2.5 years old. Both trials evaluated INZ-701 in generalized arterial calcification of infancy (GACI), a severe manifestation of ENPP1 deficiency. Patients were treated for durations ranging from three weeks to 22 months. Results demonstrated an 80% survival rate beyond the first year of life, compared to a historical survival rate of approximately 50%. Additionally, data showed reduction or resolution of arterial calcifications, improved heart function, no evidence of rickets in eligible patients, and a favorable safety profile.
ENERGY 3 to Evaluate INZ-701 in Older Children With ENPP1 Deficiency
That same month, the company completed enrollment in the pivotal ENERGY 3 trial of INZ-701, a randomized study involving 25 pediatric patients from one to under 13 years of age. The trial is designed to detect meaningful improvements in rickets using both biochemical and clinical endpoints, with topline results expected in early 2026.2 Inozyme is currently planning a supportive study of INZ-701 for adolescents and adults.1
FDA Fast Track and ASPIRE Trial Advance ABCC6 Deficiency Program
In July 2024, the FDA granted Fast Track designation to INZ-701 in patients with ABCC6 deficiency, based on preliminary safety and efficacy data from a Phase I/II trial.3 Building on this momentum, Inozyme is preparing to launch the ASPIRE trial, a pivotal, randomized study that will evaluate INZ-701 in approximately 70 pediatric patients with ABCC6 deficiency, aiming to measure major adverse clinical events over a two-year treatment period.2
"Today's announcement gives greater hope to patients who may benefit from INZ-701, a potentially transformative therapy that aims to address the underlying causes and systemic impacts of ENPP1 deficiency," said Douglas A. Treco, PhD, CEO, chairman, Inozyme, in the press release. "BioMarin has paved the way over the past two and a half decades, successfully launching five first-in-disease enzyme therapies. I'd like to thank the team at Inozyme and our partners for their outstanding work and dedication, as we pass this important potentially life-changing therapy to the leading innovator in genetically defined conditions."
Deal Closing Awaits Regulatory and Shareholder Approvals
The completion of the tender offer remains subject to customary closing conditions, including the tender of a majority of Inozyme’s outstanding shares, the expiration or early termination of the waiting period under the Hart-Scott-Rodino Antitrust Improvements Act of 1976, and other customary conditions.1
References
1. BioMarin Strengthens Enzyme Therapy Business with Acquisition of Inozyme Pharma. BioMarin. May 16, 2025. Accessed May 19, 2025. https://investors.biomarin.com/news/news-details/2025/BioMarin-Strengthens-Enzyme-Therapy-Business-with-Acquisition-of-Inozyme-Pharma/default.aspx
2. Inozyme Pharma Announces Positive Interim Data for INZ-701 in Infants and Young Children with ENPP1 Deficiency and Key Program Updates. GlobeNewswire. January 10, 2025. Accessed May 19, 2025. https://www.globenewswire.com/news-release/2025/01/10/3007647/0/en/Inozyme-Pharma-Announces-Positive-Interim-Data-for-INZ-701-in-Infants-and-Young-Children-with-ENPP1-Deficiency-and-Key-Program-Updates.html
3. FDA Grants Fast Track Designation to Inozyme Pharma’s INZ-701, a Treatment for Patients with ABCC6 Deficiency. PharmExec. July 2, 2024. Accessed May 19, 2025. https://www.pharmexec.com/view/fda-grants-fast-track-designation-inozyme-pharma-inz-701-treatment-patients-abcc6-deficiency
