Looking back to look ahead at Inozyme

Dear Friends and Partners

In early April, I passed the baton to Doug Treco , the Chairman of Inozyme, to lead the company as CEO in the next leg of this important race toward the first potential therapy for two rare and devastating disorders. Moving forward, I will serve as a Senior Advisor to the company and continue my role on the board of directors.

I am delighted that Doug shares the excitement for the opportunity in front of us and has agreed to step up to this additional role after having led the Board for several years. He knows the company, the science and the team and we could not have found a better person for the next chapter in the development of Inozyme.

I founded Inozyme almost seven years ago, along with Demetrios Braddock , Ph.D., and Joseph Schlessinger , Ph.D., based on Demetrios’ important work on the ENPP-family of enzymes and, in particular, around his work on ENPP1 and its role in regulating mineralization. With a draft manuscript describing how his ENPP1-Fc construct rescued 100% of the treated Enpp1-deficienct mice, we went on to raise capital and defined a strategic path that is largely unchanged today.

Our Series A round of $49M closed in January 2017, followed by a second private financing in 2019 (Series A2, $67M) and then a public offering in 2020 ($138M) supported by blue-chip investors. The early days were equally exciting and humbling, as Steven Jungles Jungles maneuvered our small team through cell line development and the manufacturing process. We worked through many difficult moments until we finally had - for the first time - grams of product that could treat more than just a handful of mice.?

Henric Bjarke joined as COO, and we began to expand our team and work with the patient community. We eventually grew out of our tiny space in Technology Square and planted ourselves in the Boston Seaport. We also built our research group, initially assembled and led by David Thompson, until Yves Sabbagh, PhD Sabbagh arrived as CSO. The research group continues to deliver on our research objectives to lead the way in understanding mineralization defects and intimal proliferation.

With compelling preclinical data in hand, our IND was cleared in late 2020 – a year we will certainly all remember as the start of the COVID-19 pandemic. As a new publicly held company, starting our first clinical trials, while navigating the difficulties of a global pandemic, we faced many operational headwinds. Nonetheless, we remained steadfast in our commitment to our patient community. By late 2021 a historic milestone was reached - with the first-ever patient with ENPP1 Deficiency receiving INZ-701!??Under the leadership of Kurt Gunter, MD, FASCP , our CMO, our clinical and medical team has grown substantially with multiple clinical trials now in flight.

INZ-701 met our high expectations – behaving safely and raising the critical pharmacodynamic disease marker, inorganic pyrophosphate rapidly, significantly and over a sustained period of time in patients. Based on this promising initial data we raised a follow-on offering of $73M in April 2022, against a decidedly hostile public market backdrop. We were glad to have this lifeline. With continued progress on the clinical, commercial, and regulatory front, we held our first investor and analyst day in early 2023 and people gained a new appreciation of the Inozyme opportunity.

We are now, once again at an inflection point – INZ-701 is accelerating into late-stage development in Q3 2023. Getting INZ-701 to patients in desperate need for a therapeutic option is as urgent as ever and in sight. The question is not?if?but just?when?it will be available to them.

So after almost seven years of building the company and advancing the asset, I feel the company is in an excellent state and ready to be taken forward by a new team.

Inozyme and INZ-701 are close to my heart and I will continue to support them in every way possible.

It has been a most rewarding experience to work with this team of dedicated and smart individuals. I am incredibly grateful for the experience and for the contributions of our partners, KOLs, collaborators, caregivers, patients, vendors, and our investors. I look forward to staying in touch as the journey continues with Inozyme and new opportunities.

#raredisease #enpp1deficiency #abcc6deficiency #PXE