The Balancing Act® on Lifetime to Feature ENPP1 Deficiency

Rhea-AI Summary

Inozyme Pharma announces the feature of ENPP1 deficiency on The Balancing Act® to raise awareness about this rare, life-threatening disease with no approved treatments. The episode, airing on March 15 and March 24, 2021, will include personal stories from patients and medical professionals. The ENPP1 gene is vital for regulating mineralization and its deficiency can lead to severe health issues, especially in infants. Inozyme aims to develop therapies for these conditions, emphasizing the need for early diagnosis and proper treatment.

Positive

• Inozyme Pharma aims to develop novel therapeutics for ENPP1 deficiency, addressing a significant unmet medical need.
• The segment on The Balancing Act® highlights patient stories, potentially enhancing public awareness and support for the condition.

Negative

• The absence of approved treatments for ENPP1 deficiency indicates a significant challenge in disease management.
• The critical nature of ENPP1 deficiency and its high fatality rate in infants underscores the urgency for effective therapies.

 Educational Episode to Highlight Symptoms of ENPP1 Deficiency and the Importance of Early Diagnosis 

BOSTON, March 11, 2021 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc., a rare disease biopharmaceutical company developing novel therapeutics for the treatment of disorders of abnormal mineralization, announced today that ENPP1 deficiency will be featured on The Balancing Act^® as part of the show’s recurring “Behind the Mystery of Rare and Genetic Diseases” series to raise awareness for this rare, debilitating, and life-threatening mineralization disease for which there are currently no approved treatment options.

The episode will feature Janine Hicks, a patient living with ENPP1 deficiency; her parents, Donna and Peter Hicks; and Professor Zulf Mughal, a treating physician and researcher and Consultant in Pediatric Bone Disorders, Royal Manchester Children’s Hospital, Manchester University NHS Foundation Trust. The segment will cover scientific and medical aspects of ENPP1 deficiency, the importance of making an accurate diagnosis, and the daily challenges faced by patients and families living with this debilitating disease.

“Many rare diseases like ENPP1 deficiency are not easily recognized or well-understood and patients face long delays in getting an accurate diagnosis. It is our hope that this segment will increase awareness of this devastating disease so that patients can receive an accurate diagnosis and appropriate medical treatment as we work to develop a therapy to treat patients who currently lack options,” said Axel Bolte, co-founder, president, and chief executive officer of Inozyme. “We are extremely grateful to Janine, Donna, Peter, and Dr. Mughal for telling this powerful story and bringing a voice to the many challenges that patients and families dealing with ENPP1 deficiency face every day.”

“At GACI Global, we provide information about the complications that can occur with ENPP1 and ABCC6 deficiency and seek to bring hope to families impacted by these rare and life-threatening conditions,” said Christine O’Brien and Liz Molloy, co-presidents of GACI Global. “Our mission is to raise awareness of these rare conditions through research and education, so we welcomed this opportunity to work with Inozyme on this initiative. We, too, are grateful for everyone’s participation in the development of this segment.”

For the past 10 years, Behind the Mystery has spotlighted undiagnosed, misdiagnosed, and complex medical disorders by partnering with pharmaceutical and biotechnology companies who are on a mission to educate the public to bring earlier diagnosis and treatment and provide a sense of community for people living with these disorders.

The segment will air on The Balancing Act^® on Lifetime^® on Monday, March 15, 2021 at 7:30 a.m., ET/PT and again on Wednesday, March 24, 2021 at 7:30 a.m. ET/PT, after which it will be shown in syndication.

About ENPP1 Deficiency
The ENPP1 gene produces a critical enzyme called ectonucleotide pyrophosphatase/ phosphodiesterase 1 (ENPP1), which regulates inorganic pyrophosphate (PPi) levels in plasma. PPi is essential for preventing harmful soft tissue calcification and for regulating normal bone mineralization. ENPP1 Deficiency affects patients across the age spectrum and manifests as either generalized arterial calcification of infancy (GACI) type 1 or autosomal recessive hypophosphatemic rickets type 2 (ARHR2). GACI type 1 is a devastating and often fatal disease affecting infants and is characterized by calcification and narrowing of large and medium-sized arteries, resulting in heart failure and death in about half of patients within the first six months of life. Mutations in the ABCC6 gene can also cause an infantile onset of the disease called GACI Type 2. ARHR2 manifests in the post-infancy stage and causes rickets, weakened bones, repeated bone fractures, skeletal deformities, short stature, muscle weakness, fatigue, and bone pain.

About The Balancing Act
The Balancing Act^® is a morning show created and produced by BrandStar that offers sensible solutions and essential information in a fun, entertaining format; providing resources to help people do life better. The Balancing Act features everything from delicious recipes, style makeovers and dream getaways to parenting tips and the latest news in health and wealth. Tune in to The Balancing Act weekdays at 7:30 a.m. (ET/PT) on Lifetime^® and find all previously aired episodes on TheBalancingAct.com.

About GACI Global
GACI Global is a nonprofit patient advocacy group whose mission is to connect families affected by Generalized Arterial Calcification of Infancy of Hypophosphatemic Rickets caused by ENPP1 or ABCC6 deficiencies to each other and to the medical community. The organization strives to provide current educational resources and supports ongoing research. The goal of this 100% volunteer run organization is to provide not only information about what complications can occur due to ENPP1 and ABCC6 deficiencies, but to provide hope for families impacted by the condition around the world.

About Inozyme Pharma
Inozyme Pharma, Inc. (Nasdaq: INZY), is a rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases of abnormal mineralization impacting the vasculature, soft tissue, and skeleton. Through our in-depth understanding of the biological pathways involved in mineralization, we are pursuing the development of therapeutics to address the underlying causes of these debilitating diseases. It is well established that two genes, ENPP1 and ABCC6, play key roles in a critical mineralization pathway and that defects in these genes lead to abnormal mineralization. We are initially focused on developing a novel therapy to treat the rare genetic diseases of ENPP1 and ABCC6 deficiencies.

Inozyme Pharma was founded in 2017 by Joseph Schlessinger, Ph.D., Demetrios Braddock, M.D., Ph.D., and Axel Bolte, MSc, MBA, with technology developed by Dr. Braddock and licensed from Yale University. For more information, please visit www.inozyme.com.

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 and timing of our future clinical trials and our research and development programs. 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 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; 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, 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
Axel Bolte, co-founder, president, and chief executive officer
ir@inozyme.com

Media:
SmithSolve
Alex Van Rees
(973) 442-1555 ext. 111
alex.vanrees@smithsolve.com 

FAQ

What is ENPP1 deficiency and why is it significant for Inozyme (INZY)?

ENPP1 deficiency is a rare disease that affects mineralization, leading to severe complications. Inozyme Pharma is focused on developing therapies for this condition.

When will the segment about ENPP1 deficiency air on The Balancing Act?

The segment will air on March 15 and March 24, 2021, at 7:30 a.m. ET/PT on Lifetime.

Who are the key participants in the ENPP1 deficiency segment?

The segment will feature patient Janine Hicks, her family, and Professor Zulf Mughal, a treating physician.

What is the goal of the ENPP1 deficiency awareness initiative by Inozyme (INZY)?

The initiative aims to increase understanding and recognition of ENPP1 deficiency to improve diagnosis and treatment options for patients.

How does ENPP1 deficiency impact patients?

ENPP1 deficiency can lead to serious health issues including calcification of arteries, resulting in life-threatening conditions, particularly in infants.