Inozyme Pharma Reports Full Year 2024 Financial Results and Announces Strategic Prioritization of ENPP1 Deficiency Pivotal Program and Recent Business Highlights

- Cash, cash equivalents and short-term investments as of December 31, 2024, and anticipated cost savings from prioritizing ENPP1 Deficiency program, including an approximately 25% workforce reduction, expected to support operations into the first quarter of 2026 -

- Enrollment complete in ENERGY 3 pivotal trial in pediatric patients with ENPP1 Deficiency; topline data expected in first quarter of 2026 -

- Positive interim results from ENERGY 1 trial and Expanded Access Program evaluating INZ-701 in infants and young children with ENPP1 Deficiency showed improvements from baseline in multiple measures of disease -

BOSTON, March 10, 2025 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) ("the Company" or "Inozyme"), a clinical-stage biopharmaceutical company developing innovative therapeutics for rare diseases that affect bone health and blood vessel function, today reported financial results for the full year ended December 31, 2024, and provided recent business highlights.

"2024 marked a transformative year for Inozyme as we achieved critical milestones in our ENPP1 Deficiency program that enabled us to complete enrollment in our pivotal ENERGY 3 trial and announce promising interim data in infants and young children with ENPP1 Deficiency in January 2025," said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. "We are concentrating our resources on advancing INZ-701 toward potential approval in ENPP1 Deficiency. We believe this focus will allow us to most efficiently progress our lead program while building a strong foundation for future growth."

Strategic Prioritization to Extend Cash Runway

As part of its recent strategic review, the Company has prioritized activities to support the planned Biologics License Application $(BLA.AU)$ filing for INZ-701 for the Company's lead indication, ENPP1 Deficiency. Patients with ABCC6 Deficiency being treated in the Company's long-term extension study, the Company's expanded access program, or under investigator-sponsored INDs will continue to receive treatment. Future trials in ABCC6 Deficiency and calciphylaxis will be postponed. These prioritization measures, which include a workforce reduction of approximately 25% of employees implemented in the first quarter of 2025, with the Company's cash, cash equivalents and short-term investments as of December 31, 2024, are expected to extend the Company's cash runway into the first quarter of 2026.

"The strategic refocusing of our portfolio necessitated difficult decisions, including a reduction in our workforce. These organizational changes, while challenging, are essential to extend our operational runway and maximize our ability to advance INZ-701 as a potential treatment for patients with ENPP1 Deficiency," continued Dr. Treco. "I want to express my deepest gratitude to every member of the Inozyme team, both those who will continue our mission and those who will be departing. Their exceptional dedication, talent, and passion have advanced our science and strengthened our commitment to patients with rare diseases.

Enrollment Complete in ENERGY 3 Pivotal Trial

In January 2025, the Company completed enrollment in its ENERGY 3 pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency between the ages of one and less than 13 years across sites globally. With 27 patients enrolled, the trial's 2:1 randomized design provides >90% power to detect meaningful differences in radiographic global impression of change (RGI-C) between treatment and control groups. The Company anticipates completing the one-year dosing period for all patients by January 2026, with topline data expected in first quarter of 2026.

Positive Interim Data from the ENERGY 1 Trial and Expanded Access Program (EAP)

The Company recently announced positive interim data from the ENERGY 1 trial, involving three infants and the EAP, involving two infants and one 2.5-year-old child, which evaluated patients with generalized arterial calcification of infancy (GACI), a severe manifestation of ENPP1 Deficiency. Patients were treated with INZ-701 for periods of three weeks to 22 months. The data presentation can be accessed here on Inozyme's Investor Relations site.

Financial Results for the Year Ended December 31, 2024

   -- Cash Position and Financial Guidance -- Cash, cash equivalents, and 
      short-term investments were $113.1 million as of December 31, 2024. Based 
      on its current plans and including the recent strategic prioritization, 
      the Company anticipates its cash, cash equivalents, and short-term 
      investments as of December 31, 2024, will enable the Company to fund cash 
      flow requirements into the first quarter of 2026. 
 
   -- Research and Development (R&D) Expenses -- R&D expenses were $83.2 
      million for the year ended December 31, 2024, compared to $54.8 million 
      for the year ended December 31, 2023. The increase of $28.4 million was 
      driven by $14.9 million in clinical development and related consulting 
      costs to support our ongoing clinical trials, $10.1 million in chemistry, 
      manufacturing, and controls expense to support our ongoing clinical 
      trials and prepare for potential commercialization, $3.9 million in 
      personnel-related costs, including stock-based compensation expense, 
      offset by a $0.5 million decrease in facilities administrative expense. 
 
   -- General and Administrative (G&A) Expenses -- G&A expenses were $20.8 
      million for the year ended December 31, 2024, remaining consistent with 
      the $20.8 million reported for the year ended December 31, 2023. 
 
   -- Net Loss -- Net loss was $102.0 million, or $1.62 loss per share, for the 
      year ended December 31, 2024, compared to $71.2 million, or $1.37 loss 
      per share, for the year ended December 31, 2023. 

About ENPP1 Deficiency

ENPP1 Deficiency is a serious and progressive rare disease that affects blood vessels, soft tissues, and bones. Individuals who present in utero or in infancy are typically diagnosed with generalized arterial calcification of infancy (GACI Type 1), with about 50% of these infants not surviving beyond six months. Children with this condition typically develop autosomal-recessive hypophosphatemic rickets type 2 (ARHR2), while adolescents and adults may develop osteomalacia, or softened bones. ARHR2 and osteomalacia cause pain and difficulty with movement. Additionally, patients may experience hearing loss, calcification in arteries and joints, and heart problems. ENPP1 Deficiency is an autosomal recessive disease and biallelic mutations are estimated to occur in approximately 1 in 64,000 pregnancies worldwide. Many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) exhibit severe symptoms, suggesting that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates. Currently, there are no approved therapies for ENPP1 Deficiency.

About Inozyme Pharma

Inozyme Pharma is a clinical-stage biopharmaceutical company dedicated to developing innovative therapeutics that target the PPi-Adenosine Pathway, a key regulator of bone health and blood vessel function. Disruptions in this pathway underlie a range of severe diseases, including ENPP1 Deficiency. Our lead investigational therapy, INZ-701, is an ENPP1 Fc fusion protein enzyme replacement therapy (ERT) designed to restore PPi and adenosine levels. INZ-701 is currently in late-stage clinical development in ENPP1 Deficiency, with the potential to expand into additional indications where deficiencies in the PPi-Adenosine Pathway contribute to disease pathology. Through our pioneering work, we aim to transform treatment options for patients affected by these devastating conditions.

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

 
 
                   Condensed Consolidated Balance Sheet 
                              (in thousands) 
 
                                 December 31, 2024     December 31, 2023 
Cash, cash equivalents and 
 investments                     $         113,087    $         188,289 
Total assets                               123,182              200,847 
Total liabilities                           65,356               60,368 
Additional paid-in-capital                 445,705              426,362 
Accumulated deficit                       (387,954)            (285,930) 
Total stockholders' equity                  57,826              140,479 
 
 
         Condensed Consolidated Statements of Operations and 
                          Comprehensive Loss 
           (in thousands, except share and per share data) 
 
                                            Year Ended December 31, 
                                          ---------------------------- 
                                              2024           2023 
                                          ------------  -------------- 
Operating expenses: 
      Research and development            $    83,231   $    54,847 
      General and administrative               20,799        20,798 
Total operating expenses                      104,030        75,645 
                                           ----------    ---------- 
Loss from operations                         (104,030)      (75,645) 
                                           ----------    ---------- 
Other income (expense): 
      Interest income                           7,666         7,837 
      Interest expense                         (5,558)       (3,333) 
      Other expense, net                         (102)          (28) 
Other income, net                               2,006         4,476 
                                           ----------    ---------- 
Net loss                                  $  (102,024)  $   (71,169) 

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