Inozyme Pharma

Inozyme Pharma is now a wholly owned subsidiary of Pharmaceutical Inc. We look forward to advancing our shared mission in translating the promise of genetic discovery into medicines that make a profound impact on the life of each patient.

Thanks to STAT for highlighting our new Chief Medical Officer, Dr. Petra Duda. We’re thrilled to have her join us in our fight against rare diseases that affect bone health and blood vessel function.

You can read more about what she does in her free time here:

Up and down the ladder: The latest comings and goings From new hires to departures, promotions and transfers, here are the latest comings and goings in the pharmaceutical industry.

We’re proud to share that Inozyme Pharma has entered into a definitive agreement to be acquired by Biomarin, a global leader in developing and delivering therapies for rare genetic diseases. This marks an exciting new chapter in our mission to bring a potential first-in-class treatment to patients living with ENPP1 Deficiency—a severe, progressive, and life-threatening disorder with no approved therapies.

Joining forces with BioMarin will help accelerate the development and potential global reach of INZ-701, our investigational enzyme replacement therapy. Pivotal Phase 3 data are expected in early 2026, with potential regulatory filings anticipated in 2027.

We are deeply grateful to the patients, families, clinicians, and partners who have supported this journey. We look forward to the future with continued determination and hope.

Read the full press release:

BioMarin Strengthens Enzyme Therapy Business with Acquisition of Inozyme Pharma Acquisition is Strong Strategic Fit for BioMarin, Adding INZ-701, a Phase 3 Enzyme Replacement Therapy Being Developed for Treatment of ENPP1 Deficiency First Pivotal Data Readout in Children Expected in Early 2026 with Potential Launch in 2027; Additional Clinical Programs to Expand to Patients of....

Today we announced that Petra Duda, M.D., Ph.D. has been appointed our Chief Medical Officer. Dr. Duda will help guide the development of our lead program, INZ-701, which is being studied in a pivotal clinical trial in children with ENPP1 Deficiency. She brings more than two decades of global clinical development, medical affairs, and regulatory leadership to Inozyme Pharma, and has deep expertise in rare and ultra-rare diseases.

You can read more about Dr. Duda in our press release: https://bit.ly/4kgr4Ya

Today, we announced our Q1 2025 financial results and provided important updates on the ENPP1 Deficiency indication for our lead program, INZ-701, including:

* Encouraging Interim ENERGY 3 data highlighting INZ-701’s potential to modify disease course in ENPP1 Deficiency, with sustained phosphate increases and favorable safety and immunogenicity to date

* ENERGY 3 trial on track for first quarter of 2026 topline data; no patient dropouts, dose adjustments or discontinuations, and no new safety signals

* Updated observations on anti-drug antibody data (ADA) from the ENERGY 1 clinical trial and Expanded Access Program (EAP) showing that In INZ-701-treated infants who developed high-titer ADAs affecting exposure, titers stabilized or declined over time, following transition to twice-weekly dosing or a dose increase

* Agreement with Japan’s Pharmaceuticals and Medical Devices Agenda (PMDA) on a regulatory filing strategy for INZ-701

* Acceptance by the U.S. Centers for Medicare & Medicaid Services (CMS) of a new set of ICD-10 diagnosis codes to support the classification of disorders of pyrophosphate metabolism, including ENPP1 Deficiency

You can read more here: https://bit.ly/4kgr4Ya

Our team is on-site this week at the first ever joint Congress between the European Society for Paediatric Endocrinology ( ) and the European Society of Endocrinology ( ), May 10-13 in Copenhagen. We contributed to a presentation on the evaluation of rickets and are sponsoring a symposium. Come join us!

⚕️Poster Presentation
Quantitative Evaluation of Hypophosphatemic Rickets Due to ENPP1 Deficiency
Leanne Ward, M.D.
Saturday, May 10 | ePoster Session – Bone & Mineral Metabolism -
Multi-center retrospective analysis highlighting persistent FGF23-mediated hypophosphatemia, impaired growth, and delayed bone age in children with ARHR2 under standard of care—underscoring the need for earlier diagnosis and novel therapies.

⚕️Clinical Presentation
Emerging Data in Heterozygous ENPP1 Deficiency
David Weber, M.D., MSCE
New observations suggest monoallelic ENPP1 variants may lead to clinically significant disease - broadening our understanding of ENPP1-related conditions.

⚕️Industry-Sponsored Symposium
Hypophosphatemic Rickets: Practical Tips for Distinguishing ARHR2 from XLH
Sunday, May 11 | 9:55 a.m.
Bella Center, Copenhagen
Led by Dr. Lothar Seefried, the session focused on diagnostic clarity and actionable strategies to improve patient outcomes.

Let’s connect while we’re here!

Today we celebrate , honoring groundbreaking achievements in genetics, particularly the discovery of the DNA double helix by James Watson and Francis Crick in 1953, and the completion of the Human Genome Project in 2003.

At Inozyme Pharma, we’re building upon these critical achievements as we work to better understand and develop treatments for people living with rare diseases that affect bone health and blood vessel function. As part of this commitment, we support no-cost genetic testing and genomic newborn screening programs in a number of countries to facilitate earlier and accurate diagnosis. We’ve also broadened our physician outreach efforts through medical education at key conferences and scientific engagement.

How do public policy and regulatory shifts affect the development of life-saving medicines for people with rare diseases?

Our Chief Operating Officer Matt Winton shared his thoughts with BioXconomy on how market dynamics can lead to “a steady flow of innovation” in the discovery and development of rare diseases medicines.

You can read the article here: https://bit.ly/3R0cU0U

Rare disease investment: Two steps forward, one step back Industry experts point to technological advancements, strategic shifts by pharmaceutical companies, and evolving market dynamics as key drivers of renewed rare disease R&D.

Our CEO Doug Treco, Ph.D., will participate in a fireside chat at the 24th Annual Needham Virtual Healthcare Conference on Monday, April 7, 2025, from 2:15 - 2:55 p.m. ET.

Learn more: https://bit.ly/3RhnqkJ