Inozyme Pharma Presents New Data at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting
Inozyme Pharma (Nasdaq: INZY) presented new data at the ASBMR 2024 Annual Meeting, highlighting the urgent need for therapies addressing cardiovascular and musculoskeletal complications in ENPP1 and ABCC6 Deficiencies. Key findings include:
1. In ENPP1 Deficiency, 88% of patients experienced ectopic calcifications, with 76% showing cardiovascular complications.
2. 95% of surviving ENPP1 Deficiency patients are expected to face lifelong complications.
3. ABCC6 Deficiency patients showed significant cardiovascular issues, with 44% experiencing stroke.
Inozyme and GACI Global launched the PROPEL Registry to further understand these rare diseases. The company is developing INZ-701 as a potential treatment for these conditions.
Inozyme Pharma (Nasdaq: INZY) ha presentato nuovi dati durante la Riunione Annuale ASBMR 2024, evidenziando l'urgenza di terapie per affrontare le complicazioni cardiovascolari e muscoloscheletriche nelle Deficienze di ENPP1 e ABCC6. I principali risultati includono:
1. Nella Deficienza di ENPP1, l'88% dei pazienti ha mostrato calcificazioni ectopiche, con il 76% che presentava complicazioni cardiovascolari.
2. Si prevede che il 95% dei pazienti sopravvissuti alla Deficienza di ENPP1 affronti complicazioni per tutta la vita.
3. I pazienti con Deficienza di ABCC6 hanno mostrato problemi cardiovascolari significativi, con il 44% che ha subito un ictus.
Inozyme e GACI Global hanno lanciato il Registro PROPEL per comprendere meglio queste malattie rare. L'azienda sta sviluppando INZ-701 come potenziale trattamento per queste condizioni.
Inozyme Pharma (Nasdaq: INZY) presentó nuevos datos en la Reunión Anual de ASBMR 2024, destacando la urgente necesidad de terapias para abordar las complicaciones cardiovasculares y musculoesqueléticas en las Deficiencias de ENPP1 y ABCC6. Los hallazgos clave incluyen:
1. En la Deficiencia de ENPP1, el 88% de los pacientes experimentaron calcificaciones ectópicas, y el 76% mostró complicaciones cardiovasculares.
2. Se espera que el 95% de los pacientes sobrevivientes de la Deficiencia de ENPP1 enfrenten complicaciones de por vida.
3. Los pacientes con Deficiencia de ABCC6 presentaron problemas cardiovasculares significativos, con el 44% experimentando un accidente cerebrovascular.
Inozyme y GACI Global lanzaron el Registro PROPEL para comprender mejor estas enfermedades raras. La compañía está desarrollando INZ-701 como un posible tratamiento para estas condiciones.
이노자임 제약(나스닥: INZY)은 2024년 ASBMR 연례 회의에서 심혈관 및 근골격계 합병증을 해결하기 위한 치료제의 시급한 필요성을 강조하는 새로운 데이터를 발표했습니다. 주요 발견 내용은 다음과 같습니다:
1. ENPP1 결핍증 환자의 88%가 이소성 석회화를 경험했으며, 76%는 심혈관 합병증을 보였습니다.
2. 생존한 ENPP1 결핍증 환자의 95%는 평생 동안 합병증에 직면할 것으로 예상됩니다.
3. ABCC6 결핍증 환자는 중요한 심혈관 문제를 보였고, 44%가 뇌졸중을 경험했습니다.
이노자임과 GACI Global은 이러한 희귀 질환을 더 잘 이해하기 위해 PROPEL 등록부를 시작했습니다. 이 회사는 이러한 질환에 대한 잠재적 치료제로 INZ-701을 개발하고 있습니다.
Inozyme Pharma (Nasdaq: INZY) a présenté de nouvelles données lors de la Réunion Annuelle de l'ASBMR 2024, soulignant le besoin urgent de thérapies pour traiter les complications cardiovasculaires et musculo-squelettiques dans les Déficiences en ENPP1 et ABCC6. Les principaux résultats incluent :
1. Dans la Déficience en ENPP1, 88% des patients ont présenté des calcifications ectopiques, avec 76% montrant des complications cardiovasculaires.
2. On s'attend à ce que 95% des patients survivants à la Déficience en ENPP1 fassent face à des complications à vie.
3. Les patients présentant une Déficience en ABCC6 ont montré des problèmes cardiovasculaires significatifs, avec 44% ayant subi un AVC.
Inozyme et GACI Global ont lancé le Registre PROPEL pour mieux comprendre ces maladies rares. L'entreprise développe INZ-701 comme traitement potentiel pour ces conditions.
Inozyme Pharma (Nasdaq: INZY) hat auf dem ASBMR 2024 Jahreskongress neue Daten präsentiert, die den dringenden Bedarf an Therapien zur Bekämpfung von kardiovaskulären und muskuloskelettalen Komplikationen bei ENPP1- und ABCC6-Defizienzen hervorheben. Die wichtigsten Ergebnisse umfassen:
1. Bei ENPP1-Defizienz erlitten 88% der Patienten ektopische Verkalkungen, wobei 76% kardiovaskuläre Komplikationen aufwiesen.
2. 95% der überlebenden ENPP1-Defizienzpatienten werden voraussichtlich lebenslange Komplikationen erleben.
3. ABCC6-Defizienzpatienten wiesen signifikante kardiovaskuläre Probleme auf, wobei 44% einen Schlaganfall erlitten.
Inozyme und GACI Global haben das PROPEL-Register ins Leben gerufen, um diese seltenen Krankheiten besser zu verstehen. Das Unternehmen entwickelt INZ-701 als potenzielle Behandlung für diese Erkrankungen.
- Inozyme presented new data highlighting the urgent need for therapies in ENPP1 and ABCC6 Deficiencies
- Launch of PROPEL Registry to enhance understanding of these rare diseases
- Development of INZ-701 as a potential transformative treatment for patients
- High mortality rate in infants with ENPP1 Deficiency (over 50% die within six months)
- 95% of surviving ENPP1 Deficiency patients expected to face lifelong complications
- Significant cardiovascular and musculoskeletal complications in both ENPP1 and ABCC6 Deficiencies
Insights
This data presentation at ASBMR 2024 provides critical insights into ENPP1 and ABCC6 Deficiencies, rare diseases affecting bone and blood vessel health. Key findings include:
- High mortality rate in infants with ENPP1 Deficiency, with >
50% dying within 6 months 88% of patients experienced ectopic calcifications, primarily arterial (81% ) and aortic (66.7% )76% of patients had cardiovascular complications95% of surviving patients expected to face lifelong complications
The launch of the PROPEL Registry aims to enhance understanding of disease progression and burden. While this research is important for advancing treatment options, the immediate impact on Inozyme's stock may be without specific clinical trial results or regulatory updates for their lead candidate INZ-701.
- Data underscore the urgent need for therapies that address the cardiovascular and musculoskeletal complications and long-term systemic effects of ENPP1 and ABCC6 Deficiencies in children -
- Inozyme and GACI Global launched PROPEL Registry to further understanding of these rare, life-threatening diseases (NCT06302439) -
BOSTON, Sept. 26, 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, is presenting four posters at the American Society for Bone and Mineral Research (ASBMR) 2024 Annual Meeting, including new data that demonstrate the progression and impact of rare bone and blood vessel diseases in children. These presentations include findings highlighting the significant medical burden associated with ENPP1 Deficiency and early-onset ABCC6 Deficiency, new data from a natural history study that provides critical insights into the progression of ENPP1 Deficiency, and the launch of a global registry to enhance our understanding of these diseases.
“Given their high mortality rate, there is an urgent unmet need for patients affected by these rare bone and blood vessel diseases. For instance, more than half of all infants with ENPP1 Deficiency die within six months, and those that survive often face major and potentially fatal complications throughout their lives,” said Kurt Gunter, M.D., Chief Medical Officer of Inozyme Pharma. “These new data underscore the significant and multifaceted impact of ENPP1 and ABCC6 Deficiencies, further highlighting the significant need for therapies that address the immediate and long-term systemic complications associated with these conditions. At Inozyme, we are dedicated to unlocking the full potential of our lead candidate, INZ-701, a potentially transformative treatment for patients and their families facing these diseases.”
“As the data from these retrospective analyses demonstrates, the impact of these life-threatening conditions is more significant in both the short and long-term than we had understood, particularly in terms of cardiovascular and musculoskeletal complications,” said Christine O’Brien, co-president of GACI Global. “We’re excited to work with Inozyme to launch the PROPEL Registry to prospectively collect clinical data and patient-reported outcomes that will provide a comprehensive understanding of the burden of illness and progressive nature of ENPP1 Deficiency and early-onset ABCC6 Deficiency.”
Natural History Study of 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
- In the 84 patients with ENPP1 Deficiency, cardiovascular complications and arterial calcifications developed early in life, predominately within the first few months.
- A striking
88% of patients experienced ectopic calcifications, with81% showing arterial calcification and66.7% showing aortic calcification. Cardiovascular complications, including hypertension, heart valve defects and heart failure, were noted in76% of patients. - Among the 84-patient cohort, 51 were diagnosed with Generalized Arterial Calcification of Infancy (GACI) alone and only 19 survived beyond infancy; 22 were diagnosed with GACI, survived beyond infancy and progressed to Autosomal Recessive Hypophosphatemic Rickets Type 2 (ARHR2); and 11 patients presented initially with ARHR2.
- Musculoskeletal complications, including phosphate-wasting rickets, bowed extremities, and pain, were documented in all patients diagnosed with ARHR2, with
45% of ENPP1 Deficiency survivors showing symptoms by age 3 and75% by age 11. - The data highlight that
95% of surviving ENPP1 Deficiency patients are expected to experience cardiovascular, musculoskeletal, and other organ complications, underscoring the lifelong burden of the disease.
Title: Lifetime Accumulation of Calcification and Clinical Complications in Patients with ENPP1 Deficiency (GACI and ARHR2)
Format: Poster Presentation
Author/Presenter: Carlos Ferreira, M.D., of the National Institutes of Health (NIH)
Session Dates and Times:
Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET
Early Career Spotlight: Friday, September 27, 2024, 4:54 p.m. ET
Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET
Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET
Retrospective Observational Study of ENPP1 Deficiency and Early-onset ABCC6 Deficiency
Like ENPP1 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.
- This study described the clinical events and interventions in patients with ENPP1 Deficiency (n=14) and early-onset ABCC6 Deficiency (n=9).
- ENPP1 Deficiency was associated with a high burden of cardiovascular complications in infancy, with additional frequent medical interventions including orthopedic surgery (
64% ) and hearing aids (43% ). - Early-onset ABCC6 Deficiency patients showed significant cardiovascular complications, and
44% experienced stroke.
Title: The Medical Burden of ENPP1 Deficiency and Early-Onset ABCC6 Deficiency from a Retrospective Observational Study
Format: Poster Presentation
Author/Presenter: David R. Weber, M.D., MSCE, Attending Physician and Medical Director of the Center for Bone Health at Children’s Hospital of Philadelphia
Session Dates and Times:
Rare Bone Disease Symposium: Thursday, September 26, 2024, 8:00 a.m. – 6:00 p.m. ET
Plenary during Welcome Reception: Friday, September 27, 2024, 5:30 – 7:30 p.m. ET
Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET
Additional poster presentations include:
Title: PROPEL: Prospective Observational Registry for ENPP1 Deficiency and Infantile-Onset ABCC6 Deficiency
Format: Poster Presentation
Author/Presenter: Lothar Seefried, M.D., of the Orthopedic Department of the University Hospital at the Julius-Maximilians University Würzburg, Germany
Session Dates and Times:
Energy Metabolism in Skeletal Development and Disease: Thursday, September 26, 2024, 8:30 a.m. – 5:30 p.m. ET
Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET
Title: Therapeutic Application of ENPP1-Fc Prevent Muscle Calcification Following Cardiotoxin-induced-muscle Damage in Abcc6 -/- mice
Format: Poster Presentation
Author/Presenter: Kevin O’Brien, Principal Scientist, Inozyme Pharma
Session Date and Time:
Late Breaking Poster Session I: Saturday, September 28, 2024, 2:15 – 3:45 p.m. ET
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
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 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 potential benefits of INZ-701. 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.
Contacts
Investors:
Inozyme Pharma
Stefan Riley, Senior Director of IR and Corporate Communications
(857) 330-8871
Stefan.riley@inozyme.com
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Todd Cooper
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FAQ
What new data did Inozyme Pharma (INZY) present at the ASBMR 2024 Annual Meeting?
What is the PROPEL Registry launched by Inozyme Pharma (INZY) and GACI Global?
What is the mortality rate for infants with ENPP1 Deficiency according to Inozyme Pharma's (INZY) data?
What potential treatment is Inozyme Pharma (INZY) developing for ENPP1 and ABCC6 Deficiencies?
