Inozyme Pharma Publishes Preclinical Data Supporting INZ-701 as a Potential Therapy for a Broad Range of Serious Rare Diseases Impacting Bone Health and Blood Vessel Function

Rhea-AI Summary

Inozyme Pharma (Nasdaq: INZY) has published preclinical data in the journal Cells, supporting the potential of INZ-701, their lead ENPP1 enzyme replacement therapy (ERT) candidate, to treat a broad range of rare diseases affecting bone health and blood vessel function. The study demonstrates INZ-701's ability to inhibit intimal proliferation in both ENPP1-deficient and wild-type mice, suggesting potential applications beyond traditional ERT.

Key findings show that INZ-701 increases levels of inorganic pyrophosphate (PPi) and adenosine, addressing deficiencies in the PPi-Adenosine Pathway. This pathway is critical for regulating bone health and blood vessel function. The research reveals ENPP1's role in adenosine production, explaining its dual action in preventing abnormal mineralization and inhibiting intimal proliferation. These results position INZ-701 as a promising therapeutic for multiple rare diseases driven by PPi-Adenosine Pathway disruptions.

Positive

• Preclinical data supports INZ-701's potential to treat a broad range of rare diseases affecting bone health and blood vessel function
• INZ-701 demonstrated ability to inhibit intimal proliferation in both ENPP1-deficient and wild-type mice
• Research reveals ENPP1's role in adenosine production, explaining its dual action in preventing abnormal mineralization and inhibiting intimal proliferation
• Results suggest INZ-701 may have broader therapeutic applications beyond traditional enzyme replacement therapy

Negative

• None.

Insights

Medical Research Analyst

As a Medical Research Analyst, I find the preclinical data on INZ-701 highly intriguing. The study's findings, published in Cells, shed light on the potential of INZ-701 to address a broader range of rare diseases than initially anticipated. The key takeaway is the drug's ability to inhibit intimal proliferation, which is important for treating diseases characterized by vascular smooth muscle cell overgrowth.

What's particularly noteworthy is INZ-701's dual action mechanism. It not only increases levels of inorganic pyrophosphate (PPi) to regulate mineralization but also boosts adenosine production, which plays a role in preventing intimal proliferation. This dual functionality positions INZ-701 as a promising therapeutic candidate for multiple rare diseases stemming from disruptions in the PPi-Adenosine Pathway.

The in vitro experiments demonstrating ENPP1-Fc's ability to reduce vascular smooth muscle cell proliferation through the production of AMP and adenosine are compelling. Moreover, the in vivo studies showing INZ-701's efficacy in both ENPP1-deficient and wild-type mice suggest a broader therapeutic potential beyond traditional enzyme replacement therapy.

While these preclinical results are promising, it's important to note that further clinical studies will be necessary to confirm these findings in humans. The potential applications for INZ-701 in treating conditions such as ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis and ossification of the posterior longitudinal ligament (OPLL) could represent a significant advancement in rare disease therapeutics.

Financial Analyst

From a financial perspective, this preclinical data for Inozyme Pharma's INZ-701 is potentially significant. The expanded therapeutic potential of INZ-701 could substantially increase its market opportunity, which is important for a clinical-stage biopharmaceutical company like Inozyme (Nasdaq: INZY).

The ability of INZ-701 to address multiple rare diseases driven by disruptions in the PPi-Adenosine Pathway could translate into a larger patient population and, consequently, a larger potential revenue stream. Rare disease treatments often command premium pricing due to their specialized nature and patient populations, so expanding the drug's applicability could significantly boost its commercial prospects.

Moreover, the dual mechanism of action demonstrated by INZ-701 - addressing both mineralization and intimal proliferation - could potentially give Inozyme a competitive edge in the rare disease market. This unique selling proposition could be attractive to potential partners or acquirers, possibly opening up opportunities for licensing deals or M&A activity.

However, it's important to temper this optimism with caution. Inozyme is still a clinical-stage company and INZ-701 will need to successfully navigate clinical trials before reaching the market. The path from preclinical to approved drug is long, costly and fraught with risks. Investors should closely monitor the progress of INZ-701 through clinical trials and regulatory processes.

In conclusion, while this preclinical data is promising and could expand Inozyme's market potential, it's important to consider the long road ahead and the inherent risks in drug development when evaluating the company's financial prospects.

- Treatment with INZ-701 shown to prevent intimal proliferation in both ENPP1-deficient and wild-type mice, supporting its potential therapeutic application beyond traditional ERT -

BOSTON, July 25, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage biopharmaceutical company developing novel therapeutics for rare diseases that affect bone health and blood vessel function, today announced the publication of preclinical data supporting the potential of INZ-701, the Company’s lead ENPP1 enzyme replacement therapy (ERT) development candidate, to treat a broad range of diseases mediated by the PPi-Adenosine Pathway, which regulates mineralization and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). The article titled, “Inhibition of Vascular Smooth Muscle Cell Proliferation by ENPP1: The Role of CD73 and the Adenosine Signaling Axis”, published in the journal Cells can be accessed via this link.

“The data published in Cells highlights the potential of INZ-701 to inhibit intimal proliferation, an important advancement for treating rare diseases driven by pathological dysregulation of vascular smooth muscle cell overgrowth,” said Yves Sabbagh, Ph.D., Chief Scientific Officer of Inozyme Pharma and one of the senior authors on this publication. “INZ-701’s ability to address disruptions in the PPi-Adenosine Pathway positions it as a promising therapeutic candidate, offering hope across multiple rare diseases.”

Overview of Findings

The PPi-Adenosine Pathway is critical for regulating bone health and blood vessel function. The ENPP1 enzyme is a vital component of this pathway and plays an essential role in generating inorganic pyrophosphate (PPi), a key regulator of mineralization, and adenosine, a key regulator of intimal proliferation. Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurologic conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL).

INZ-701 is designed to increase PPi and adenosine, addressing deficiencies in these molecules and offering the potential to treat multiple rare diseases driven by disruptions in the PPi-Adenosine Pathway.

The Cells publication highlights the following preclinical findings with INZ-701 (ENPP1-Fc):

In vitro Experiments:

• ENPP1-Fc was tested on vascular smooth muscle cells (VSMCs), which are the major cells in the walls of blood vessels.
• Adding ENPP-1-Fc and ATP to VSMCs led to the production of AMP, which was subsequently converted to adenosine, reducing cell proliferation.
• Both AMP and adenosine independently inhibited VSMC growth.
• Blocking the CD73 enzyme, which converts AMP to adenosine, reduced the accumulation of adenosine, and suppressed the anti-proliferative effects of ENPP1/ATP.

In vivo Experiments:

• In a mouse model where the carotid artery was ligated, treatment with INZ-701 prevented or reduced intimal proliferation in a prophylactic or therapeutic setting, respectively, in both ENPP1-deficienct and wild-type mice.
• These results suggest a certain level of adenosine is necessary to prevent or reduce intimal proliferation.

Conclusions:

• In addition to producing PPi, ENPP1 also plays an important role in the production of adenosine, revealing the mechanisms by which ENPP1 prevents both abnormal mineralization and inhibits intimal proliferation.
• These results suggest that INZ-701 may have broader therapeutic applications beyond traditional enzyme replacement therapy.

About INZ-701
INZ-701, an ENPP1 Fc fusion protein, is an enzyme replacement therapy (ERT) in development for the treatment of rare diseases linked to the PPi-Adenosine Pathway. INZ-701 metabolizes adenosine triphosphate (ATP) to generate inorganic pyrophosphate (PPi), a natural inhibitor of mineralization, and adenosine monophosphate (AMP), which can be processed to phosphate and adenosine, the latter being a natural inhibitor of intimal proliferation. In preclinical studies, INZ-701 has shown potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in devastating disorders such as, ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis and ossification of the posterior longitudinal ligament (OPLL). Clinical data to date have demonstrated that INZ-701 was generally well tolerated, exhibited a favorable safety profile, and meaningfully increased PPi levels in multiple clinical trials.

About Inozyme Pharma
Inozyme Pharma is a pioneering clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics for rare diseases that affect bone health and blood vessel function. Our expertise lies in the PPi-Adenosine Pathway, where the ENPP1 enzyme generates inorganic pyrophosphate (PPi), which regulates mineralization, and adenosine, which controls intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels). Disruptions in this pathway impact the levels of these molecules, leading to severe musculoskeletal, cardiovascular, and neurological conditions, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL).

Our lead candidate, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to increase PPi and adenosine, enabling the potential treatment of multiple diseases caused by deficiencies in these molecules. It is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis. By targeting the PPi-Adenosine Pathway, INZ-701 aims to correct pathological mineralization and intimal proliferation, addressing the significant morbidity and mortality in these devastating diseases.

For more information, please visit https://www.inozyme.com/ or follow Inozyme on LinkedIn, X, and Facebook.

Contacts
Investors:
Inozyme Pharma
Stefan Riley, Senior Director of IR and Corporate Communications
(857) 330-8871
Stefan.riley@inozyme.com

Media:
SmithSolve
Matt Pera
(973) 886-9150
Matt.pera@smithsolve.com

FAQ

What is the purpose of Inozyme Pharma's INZ-701 therapy?

INZ-701 is designed to increase levels of inorganic pyrophosphate (PPi) and adenosine, addressing deficiencies in the PPi-Adenosine Pathway. It aims to treat rare diseases affecting bone health and blood vessel function, including ENPP1 Deficiency, ABCC6 Deficiency, calciphylaxis, and ossification of the posterior longitudinal ligament (OPLL).

What were the key findings of the preclinical study on INZ-701 (INZY)?

The study found that INZ-701 inhibited intimal proliferation in both ENPP1-deficient and wild-type mice. It also demonstrated that ENPP1 plays a role in adenosine production, which helps prevent abnormal mineralization and inhibits intimal proliferation. These results suggest INZ-701 may have broader therapeutic applications beyond traditional enzyme replacement therapy.

How does INZ-701 work to treat rare diseases related to the PPi-Adenosine Pathway?

INZ-701 works by increasing levels of inorganic pyrophosphate (PPi) and adenosine, which are key regulators in the PPi-Adenosine Pathway. This pathway is critical for regulating bone health and blood vessel function. By addressing deficiencies in these molecules, INZ-701 offers potential treatment for multiple rare diseases driven by disruptions in this pathway.

What is the significance of INZ-701's ability to inhibit intimal proliferation?

INZ-701's ability to inhibit intimal proliferation is significant because it addresses the overgrowth of smooth muscle cells inside blood vessels, which is a key factor in various rare diseases. This capability, demonstrated in both ENPP1-deficient and wild-type mice, suggests that INZ-701 could have broader therapeutic applications beyond traditional enzyme replacement therapy.