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Inozyme Pharma Reports Third Quarter 2024 Financial Results and Provides Business Highlights

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Inozyme Pharma (INZY) reported Q3 2024 financial results with a net loss of $24.6 million ($0.39 per share), compared to $16.6 million ($0.29 per share) in Q3 2023. R&D expenses increased to $19.9 million from $13.3 million year-over-year. The company maintains $131.6 million in cash and equivalents, expected to fund operations into Q4 2025.

The company remains on track to report interim data from the ENERGY 1 Phase 1b trial of INZ-701 in ENPP1 Deficiency by year-end 2024, with topline data from ENERGY 3 pivotal trial expected in early 2026. Inozyme plans to initiate registrational trials in calciphylaxis and ABCC6 Deficiency in 2025, subject to regulatory alignment and funding.

Inozyme Pharma (INZY) ha riportato i risultati finanziari del terzo trimestre 2024, registrando una perdita netta di 24,6 milioni di dollari (0,39 dollari per azione), rispetto a 16,6 milioni di dollari (0,29 dollari per azione) del terzo trimestre 2023. Le spese per R&S sono aumentate a 19,9 milioni di dollari, rispetto a 13,3 milioni di dollari dell'anno precedente. L'azienda mantiene 131,6 milioni di dollari in liquidità e equivalenti, previsti per finanziare le operazioni fino al quarto trimestre 2025.

L'azienda rimane sulla buona strada per riportare dati intermedi dalla sperimentazione ENERGY 1 Fase 1b di INZ-701 nella carenza di ENPP1 entro la fine del 2024, con i dati preliminari dalla sperimentazione pivotale ENERGY 3 attesi all'inizio del 2026. Inozyme prevede di avviare studi registrativi in calcifilassi e carenza di ABCC6 nel 2025, soggetti all'allineamento normativo e al finanziamento.

Inozyme Pharma (INZY) informó sobre los resultados financieros del tercer trimestre de 2024, con una pérdida neta de 24,6 millones de dólares (0,39 dólares por acción), en comparación con 16,6 millones de dólares (0,29 dólares por acción) en el tercer trimestre de 2023. Los gastos de I+D aumentaron a 19,9 millones de dólares desde 13,3 millones de dólares en el año anterior. La compañía mantiene 131,6 millones de dólares en efectivo y equivalentes, que se espera financien las operaciones hasta el cuarto trimestre de 2025.

La empresa sigue en camino de informar datos intermedios del ensayo ENERGY 1 Fase 1b de INZ-701 en la Deficiencia de ENPP1 para finales de 2024, con datos preliminares del ensayo pivotal ENERGY 3 esperados a principios de 2026. Inozyme planea iniciar ensayos de registro en calcifilaxis y Deficiencia de ABCC6 en 2025, sujeto a alineación regulatoria y financiación.

Inozyme Pharma (INZY)는 2024년 3분기 재무 결과를 보고하며 2460만 달러의 순손실(주당 0.39달러)을 기록했습니다. 이는 2023년 3분기의 1660만 달러(주당 0.29달러)에 비해 증가한 수치입니다. 연구개발 비용은 작년의 1330만 달러에서 1990만 달러로 증가했습니다. 회사는 1억3160만 달러의 현금과 현금 등가물을 보유하고 있으며, 이는 2025년 4분기까지 운영 자금을 지원할 것으로 예상됩니다.

회사는 2024년 말까지 ENPP1 결핍증에 대한 INZ-701의 ENERGY 1 1b 단계 시험의 중간 데이터를 보고할 예정이며, ENERGY 3의 주요 시험 데이터는 2026년 초에 예상됩니다. Inozyme는 2025년에 규제 조정과 자금 지원을 조건으로 칼시필락시스와 ABCC6 결핍증에 대한 등록 시험을 시작할 계획입니다.

Inozyme Pharma (INZY) a annoncé ses résultats financiers pour le troisième trimestre 2024, avec une perte nette de 24,6 millions de dollars (0,39 dollar par action), contre 16,6 millions de dollars (0,29 dollar par action) au troisième trimestre 2023. Les dépenses de R&D ont augmenté à 19,9 millions de dollars, contre 13,3 millions de dollars l'année précédente. L'entreprise dispose de 131,6 millions de dollars en liquidités et équivalents, prévus pour financer ses opérations jusqu'au quatrième trimestre 2025.

L'entreprise reste sur la bonne voie pour communiquer des données intermédiaires de l'essai ENERGY 1 Phase 1b de l'INZ-701 dans la défaillance d'ENPP1 d'ici fin 2024, avec des données préliminaires de l'essai pivot ENERGY 3 attendues début 2026. Inozyme prévoit de lancer des essais d'enregistrement en calciphylaxie et en défaillance d'ABCC6 en 2025, sous réserve d'alignement réglementaire et de financement.

Inozyme Pharma (INZY) hat die finanziellen Ergebnisse des 3. Quartals 2024 mit einem Nettoverlust von 24,6 Millionen Dollar (0,39 Dollar pro Aktie) gemeldet, verglichen mit 16,6 Millionen Dollar (0,29 Dollar pro Aktie) im 3. Quartal 2023. Die F&E-Ausgaben sind im Jahresvergleich auf 19,9 Millionen Dollar gestiegen, von 13,3 Millionen Dollar. Das Unternehmen hält 131,6 Millionen Dollar in Zahlungsmitteln und Zahlungsmitteläquivalenten, die voraussichtlich die Betriebskosten bis zum 4. Quartal 2025 finanzieren werden.

Das Unternehmen bleibt auf Kurs, um bis Ende 2024 Zwischenresultate der ENERGY 1 Phase 1b Studie zu INZ-701 bei ENPP1-Mangel zu berichten, während die vorläufigen Daten der ENERGY 3 entscheidenden Studie für Anfang 2026 erwartet werden. Inozyme plant, 2025 registrierungsrelevante Studien zu Kalkifilaxie und ABCC6-Mangel zu initiieren, vorbehaltlich behördlicher Abstimmung und Finanzierung.

Positive
  • Positive interim data showing INZ-701 was well-tolerated in calciphylaxis patients
  • Strong cash position of $131.6 million providing runway into Q4 2025
Negative
  • Increased net loss to $24.6 million from $16.6 million year-over-year
  • R&D expenses increased 49.6% to $19.9 million year-over-year
  • Future trials dependent on additional funding

Insights

The Q3 2024 results reveal significant financial developments for Inozyme Pharma. The company's $131.6 million cash position provides runway into Q4 2025, but increased R&D spending of $19.9 million (up 49.6% YoY) reflects aggressive clinical program advancement. The widening net loss of $24.6 million ($0.39 per share) versus $16.6 million ($0.29 per share) YoY indicates heightened investment in pipeline development.

The company's burn rate and cash position warrant attention, particularly given the planned initiation of multiple registrational trials in 2025. While current funding appears adequate for near-term milestones, additional capital may be needed to support expanded clinical programs, especially the new trials in calciphylaxis and ABCC6 Deficiency which are contingent on "sufficient funding."

The clinical development strategy shows promising momentum across multiple rare disease indications. Key catalysts include:

  • ENERGY 1 trial interim data in Q4 2024 for ENPP1 Deficiency in infants
  • ENERGY 3 pivotal trial completion of enrollment by end of 2024
  • Positive interim data from calciphylaxis program showing INZ-701's ability to increase plasma PPi levels
The company's focus on rare diseases affecting bone health and vessel function represents a significant market opportunity with high unmet medical need. The positive interim calciphylaxis data particularly stands out, as this condition has treatment options and high mortality rates.

- Interim data from ENERGY 1, a Phase 1b trial of INZ-701 in infants with ENPP1 Deficiency, on track for fourth quarter of 2024 –

- Topline data from ENERGY 3, a pivotal trial of INZ-701 in pediatric patients with ENPP1 Deficiency, expected in early 2026 -

- Company plans to initiate registrational trials in calciphylaxis and ABCC6 Deficiency in 2025 subject to regulatory alignment and sufficient funding -

- Cash, cash equivalents, and short-term investments as of September 30, 2024, expected to fund operations into the fourth quarter of 2025 –

BOSTON, Nov. 05, 2024 (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 third quarter ended September 30, 2024, and provided business highlights.

“As we close in on the end of a highly productive year, our focus remains firmly on advancing INZ-701 across each of our clinical programs,” said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. “The recent presentation of promising interim data from our Phase 1 data from our calciphylaxis program highlights our progress, and we remain on track to report interim data from our Phase 1b ENERGY 1 trial in ENPP1 Deficiency by year-end. With a growing body of clinical evidence supporting the potential for INZ-701 to serve as a meaningful therapy across multiple indications of high unmet need, we are committed to our mission to bring novel treatment options to patients facing rare diseases affecting bone health and blood vessel function.”

Recent Highlights

Pipeline

  • Presentations at American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting. In September 2024, the Company presented new data at ASBMR 2024 in Toronto, Canada, demonstrating the progression and impact of ENPP1 Deficiency and ABCC6 Deficiency in children. These findings underscored the urgent need for innovative therapies to address the severe cardiovascular and musculoskeletal complications associated with these conditions. In addition, the Company and GACI Global highlighted the launch of the PROPEL Registry designed to further understanding of the burden of illness and progressive nature of ENPP1 Deficiency and early-onset ABCC6 Deficiency (NCT06302439).

ENPP1 Deficiency

  • ENPP1 Deficiency Review Series Publication. In September 2024, a series of comprehensive review articles on ENPP1 Deficiency was published in the French journal of pediatrics, Archives de Pédiatrie. Authored by expert clinicians and researchers in bone health, the articles collectively highlight the complexities of ENPP1 Deficiency, underscoring both established and emerging insights into the disease’s presentation and management.

Calciphylaxis

  • Phase 1 Trial of INZ-701 in Patients with End-Stage Kidney Disease (ESKD) Undergoing Hemodialysis. In October 2024, the Company announced positive interim data demonstrating INZ-701 was well-tolerated and significantly increased plasma pyrophosphate (PPi) levels in patients with ESKD on dialysis. Low PPi levels are linked to the development of calciphylaxis, a rare and life-threatening complication of ESKD, as well as the associated morbidity and mortality. Data were featured at the American Society of Nephrology’s Kidney Week 2024 in San Diego, CA. Subject to regulatory review and sufficient funding, the Company plans to initiate a registrational study in calciphylaxis in 2025.

Corporate

  • Board of Directors Appointment. In October 2024, the Company announced the appointment of Erik Harris to its Board of Directors. Mr. Harris, who currently serves as Chief Commercial Officer and Executive Vice President at Ultragenyx Pharmaceutical Inc., brings to the Company over 20 years of commercial expertise within the biopharmaceutical industry.

Anticipated Milestones

  • ENPP1 Deficiency
    • Complete enrollment of ENERGY 3 pivotal trial in pediatric patients by the end of 2024
    • Initiation of the ENERGY 2 pivotal trial in infants with ENPP1 Deficiency outside the United States in the fourth quarter of 2024
    • Release interim data from the ENERGY 1 Phase 1b trial in infants in the fourth quarter of 2024
    • Release topline data from the ENERGY 3 pivotal trial in pediatric patients in early 2026
  • ABCC6 Deficiency
    • Initiation of pivotal clinical trial of INZ-701 in pediatric patients with ABCC6 Deficiency in 2025, subject to regulatory alignment and sufficient funding.
  • Calciphylaxis
    • Initiation of a pivotal trial of INZ-701 in patients with calciphylaxis in 2025, subject to regulatory alignment and sufficient funding.

Third Quarter 2024 Financial Results

  • Cash Position and Financial Guidance. Cash, cash equivalents, and short-term investments were $131.6 million as of September 30, 2024. Based on its current plans, the Company anticipates its cash, cash equivalents, and short-term investments as of September 30, 2024, will enable the Company to fund cash flow requirements into the fourth quarter of 2025.
  • Research and Development (R&D) Expenses. R&D expenses were $19.9 million for the quarter ended September 30, 2024, compared to $13.3 million for the prior-year period.
  • General Administrative (G&A) Expenses. G&A expenses were $5.0 million for the quarter ended September 30, 2024, compared to $4.7 million for the prior-year period.
  • Net Loss. Net loss was $24.6 million, or $0.39 net loss per share, for the quarter ended September 30, 2024, compared to $16.6 million or $0.29 net loss per share for the prior-year period.

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 rickets, specifically 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.

Biallelic ENPP1 Deficiency affects approximately 1 in 64,000 pregnancies worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many individuals with just one copy of the mutated gene (monoallelic ENPP1 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ENPP1 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ENPP1 Deficiency.

About ABCC6 Deficiency
ABCC6 Deficiency is a progressive and debilitating rare disease that affects blood vessels and soft tissues. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), which is similar to GACI Type 1, the infant form of ENPP1 Deficiency. Pediatric patients who survive beyond the first year of life may develop neurological disease, including strokes, and cardiovascular diseases due to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency manifests as pseudoxanthoma elasticum (PXE), characterized by abnormal mineralization in blood vessels and soft tissues, affecting the skin, visual function, and vascular system.

Biallelic ABCC6 Deficiency is estimated to affect 1 in 25,000 to 1 in 50,000 individuals worldwide. Initially, it was believed to only impact individuals with two copies of the mutated gene. However, many people with just one copy of the mutated gene (monoallelic ABCC6 Deficiency) also exhibit severe symptoms. This suggests that the worldwide prevalence of ABCC6 Deficiency may be much higher than current estimates, which are based solely on biallelic cases. Currently, there are no approved therapies for ABCC6 Deficiency.

About Calciphylaxis and the PPi-Adenosine Pathway
Calciphylaxis (also known as calcific uremic arteriolopathy, or CUA) is a rare disorder with a high mortality rate that predominantly affects patients with end-stage kidney disease (ESKD). The disease is associated with low levels of inorganic pyrophosphate (PPi) and is characterized by pathologic mineralization (i.e., calcification) and intimal proliferation (the overgrowth of smooth muscle cells inside blood vessels) of the vasculature in the skin and fatty tissue. This leads to poor blood flow, blood clots, painful skin ulcers, serious infections, and often death, with a reported one-year survival rate of approximately 50%. Currently, there are no approved therapies for calciphylaxis. The estimated incidence of calciphylaxis is approximately 3.5 per 1,000 patients with ESKD with approximately 5,000 new patients presenting annually across major addressable markets.

The PPi-Adenosine Pathway plays a critical role in regulating both pathologic mineralization and intimal proliferation. The ENPP1 enzyme generates PPi, a potent inhibitor of pathologic mineralization, by hydrolyzing extracellular adenosine triphosphate. Additionally, adenosine, produced by the CD73 enzyme regulates intimal proliferation, preventing the abnormal growth of smooth muscle cells within blood vessels, which can contribute to vascular occlusion. Recent genetic research has shown that polymorphisms in the ENPP1 or CD73 genes have been linked to an increased risk of arterial calcification in ESKD patients and/or calciphylaxis, further substantiating the role of the PPi-Adenosine Pathway in this condition.

INZ-701 is designed to restore PPi levels and increase adenosine production, addressing both key elements of the PPi-Adenosine Pathway. By normalizing these processes, INZ-701 has the potential to prevent the progression of calciphylaxis, which could offer a promising therapeutic solution for this high-risk and underserved patient population.

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. We are experts 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.

Cautionary Note Regarding Forward-Looking Statements
Statements in this press release about future expectations, plans, and prospects, as well as any other statements regarding matters that are not historical facts, may constitute "forward-looking statements" within the meaning of The Private Securities Litigation Reform Act of 1995. These statements include, but are not limited to, statements relating to the initiation, timing, and design of our planned clinical trials, availability of data from clinical trials, the potential benefits of INZ-701, our regulatory strategy, including our planned pathway to approval for the ABCC6 Deficiency and calciphylaxis programs, and the period over which we believe that our existing cash, cash equivalents, and short-term investments will be sufficient to fund our cash flow requirements. The words "anticipate," "believe," "continue," "could," "estimate," "expect," "intend," "may," "plan," "potential," "predict," "project," "should," "target," "will," "would," and similar expressions are intended to identify forward-looking statements, although not all forward-looking statements contain these identifying words. Any forward-looking statements are based on management's current expectations of future events and are subject to a number of risks and uncertainties that could cause actual results to differ materially and adversely from those set forth in, or implied by, such forward-looking statements. These risks and uncertainties include, but are not limited to, risks associated with the Company's ability to conduct its ongoing clinical trials of INZ-701 for ENPP1 Deficiency, ABCC6 Deficiency, and calciphylaxis; enroll patients in ongoing and planned trials; obtain and maintain necessary approvals from the FDA and other regulatory authorities; continue to advance its product candidates in preclinical studies and clinical trials; replicate in later clinical trials positive results found in preclinical studies and early-stage clinical trials of its product candidates; advance the development of its product candidates under the timelines it anticipates in planned and future clinical trials; obtain, maintain, and protect intellectual property rights related to its product candidates; manage expenses; comply with covenants under its outstanding loan agreement; and raise the substantial additional capital needed to achieve its business objectives. For a discussion of other risks and uncertainties, and other important factors, any of which could cause the Company's actual results to differ from those contained in the forward-looking statements, see the "Risk Factors" section in the Company's most recent Annual Report on Form 10-K filed with the Securities and Exchange Commission, as well as discussions of potential risks, uncertainties, and other important factors, in the Company's most recent filings with the Securities and Exchange Commission. In addition, the forward-looking statements included in this press release represent the Company's views as of the date hereof and should not be relied upon as representing the Company's views as of any date subsequent to the date hereof. The Company anticipates that subsequent events and developments will cause the Company's views to change. However, while the Company may elect to update these forward-looking statements at some point in the future, the Company specifically disclaims any obligation to do so.


Condensed Consolidated Balance Sheet Data
(Unaudited)
    
 September 30,
2024
 December 31,
2023
Cash, cash equivalents and investments$131,608  $188,589 
Total assets$143,361  $200,847 
Total liabilities$60,573  $60,368 
Additional paid-in-capital$443,476  $426,362 
Accumulated deficit$(360,880) $(285,930)
Total stockholders' equity$82,788  $140,479 


Condensed Consolidated Statements of Operations and Comprehensive Loss
(Unaudited)

 
  Three Months Ended September 30,
   2024   2023 
Operating expenses:    
Research and development $19,890  $13,341 
General and administrative  4,961   4,733 
Total operating expenses  24,851   18,074 
Loss from operations  (24,851)  (18,074)
Other income (expense):    
Interest income  1,778   2,369 
Interest expense  (1,416)  (953)
Other (expense)income, net  (81)  20 
Other income, net  281   1,436 
Net loss $(24,570) $(16,638)
Other comprehensive income (loss):    
Unrealized gains on available-for-sale securities  290   53 
Foreign currency translation adjustment  (1)  (182)
Total other comprehensive income (loss)  289   (129)
Comprehensive loss $(24,281) $(16,767)
Net loss attributable to common stockholders—basic
and diluted
 $(24,570) $(16,638)
Net loss per share attributable to common
stockholders—basic and diluted
 $(0.39) $(0.29)
Weighted-average common shares outstanding—basic
and diluted
  63,276,851   56,758,395 
     
  Nine Months Ended September 30,
   2024   2023 
Operating expenses:    
Research and development $60,758  $36,864 
General and administrative  16,101   15,973 
Total operating expenses  76,859   52,837 
Loss from operations  (76,859)  (52,837)
Other income (expense):    
Interest income  6,182   5,306 
Interest expense  (4,136)  (2,052)
Other expense, net  (137)  (42)
Other income (expense), net  1,909   3,212 
Net loss $(74,950) $(49,625)
Other comprehensive income (loss):    
Unrealized gains on available-for-sale securities  137   279 
Foreign currency translation adjustment  8   (152)
Total other comprehensive income  145   127 
Comprehensive loss $(74,805) $(49,498)
Net loss attributable to common stockholders—basic
and diluted
 $(74,950) $(49,625)
Net loss per share attributable to common
stockholders—basic and diluted
 $(1.20) $(1.02)
Weighted-average common shares outstanding—basic
and diluted
  62,334,482   48,494,175 


Contacts

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

Media:
Biongage Communications
Todd Cooper
(617) 840-1637
Todd@biongage.com 


FAQ

What were Inozyme Pharma's (INZY) Q3 2024 financial results?

Inozyme reported a net loss of $24.6 million ($0.39 per share), with R&D expenses of $19.9 million and cash position of $131.6 million as of September 30, 2024.

When will Inozyme (INZY) report ENERGY 1 trial interim data?

Inozyme expects to report interim data from the ENERGY 1 Phase 1b trial in ENPP1 Deficiency in the fourth quarter of 2024.

How long will Inozyme's (INZY) current cash position last?

The company's cash position of $131.6 million is expected to fund operations into the fourth quarter of 2025.

Inozyme Pharma, Inc.

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Biotechnology
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