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Inozyme Pharma Announces FDA Fast Track Designation for INZ-701 in ABCC6 Deficiency

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Inozyme Pharma announced that the FDA has granted Fast Track designation to INZ-701 for treating ABCC6 Deficiency, a rare, severe condition affecting children and leading to significant clinical events such as stroke and severe neurological and cardiovascular diseases. The decision is based on nonclinical pharmacology data and preliminary safety and efficacy data from the ongoing Phase 1/2 trial in adults. This designation will facilitate more frequent FDA engagement and expedited regulatory review. Inozyme aims to finalize plans for a pivotal pediatric study by the end of 2024.

Positive
  • FDA granted Fast Track designation to INZ-701, facilitating faster development and review.
  • Preliminary safety and efficacy data from the Phase 1/2 trial showed positive results.
  • INZ-701 demonstrated clinical improvements in vascular pathology, visual function, and patient-reported outcomes.
  • Inozyme plans to present development plans and agree on a pivotal pediatric study by year-end 2024.
Negative
  • High disease burden and major clinical events, including stroke and severe neurological and cardiovascular diseases, were observed among pediatric patients with ABCC6 Deficiency.

The FDA's decision to grant Fast Track designation to INZ-701 for ABCC6 Deficiency is a significant milestone for Inozyme Pharma. This designation aims to accelerate the development and review process of drugs that address serious conditions with unmet medical needs. For a biotech company, securing such a designation is emblematic of the potential efficacy and necessity of their therapeutic, which could translate into expedited timelines for drug approval and potentially faster market entry.

From a clinical perspective, the Fast Track status provides several advantages, including more frequent interactions with the FDA and the possibility of a rolling review of the drug application. This can significantly shorten the time to market, which is important for rare diseases where treatment options are limited. It also signals confidence in the preliminary data from the ongoing Phase 1/2 trials, which have shown promising results in adults with ABCC6 Deficiency, including improvements in vascular pathology and visual function. These clinical endpoints are important as they directly impact the quality of life of patients.

ABCC6 Deficiency is associated with severe complications such as stroke and cardiovascular disease, particularly in pediatric populations. The emphasis on pediatric trials indicates a strategic focus on a demographic that is often underrepresented in clinical research but stands to benefit immensely from early intervention. By targeting these early-onset manifestations, INZ-701 could potentially alter the disease course, preventing long-term complications.

Overall, this development positions Inozyme Pharma favorably within the rare disease treatment landscape, potentially leading to enhanced stakeholder confidence and attracting further investment, important for advancing clinical programs.

The Fast Track designation for INZ-701 is not just a clinical milestone but also a strategic financial one. This status can positively influence investor sentiment by reducing perceived risks associated with the lengthy drug approval process. Expedited review timelines can lead to earlier market entry, which is beneficial for a company's revenue streams and can enhance its valuation.

For Inozyme Pharma, this announcement comes on the heels of positive topline data from their Phase 1/2 trials, which further validates the company's scientific approach and enhances its market position. The ability to report ongoing engagement with the FDA also provides transparency and confidence to investors regarding the regulatory pathway of INZ-701. This could potentially lead to an appreciation in the company's stock price as the market often reacts positively to news that could lead to expedited drug approval and commercialization.

Additionally, focusing on rare disease markets can be financially rewarding due to potential benefits like orphan drug status, market exclusivity and the ability to command premium pricing. The pediatric focus could also attract additional funding and collaborations from organizations dedicated to childhood rare diseases.

From a financial perspective, this development might also facilitate easier access to capital markets. Companies with promising drug candidates often use such milestones to engage in secondary offerings or partnerships, further stabilizing their financial footing to fund ongoing and future research endeavors.

Although not directly related to oncology, the implications of Fast Track designation for INZ-701 in a rare disease context can offer insights into the broader realm of drug development for serious conditions. Fast Track status is a strong indicator of the drug's potential to address critical unmet needs, which is a common challenge in oncology as well.

For patients with ABCC6 Deficiency, particularly children, the early onset of severe symptoms like stroke and cardiovascular disease is devastating. Early intervention with a promising therapeutic like INZ-701 could significantly alter disease progression and outcomes. This mirrors the oncology field where early detection and treatment significantly improve prognoses.

The preliminary results indicating improvements in vascular pathology and visual function in adults are encouraging and suggest that INZ-701 could have a substantial impact on patient health and quality of life. These clinical improvements are pivotal, especially in rare diseases where the therapeutic landscape is often barren.

Overall, the pursuit of Fast Track designation and the reported clinical improvements could pave the way for further advancements in treating other severe conditions, underscoring the importance of continued innovation and regulatory engagement in rare disease therapeutics.

Agency program will support and expedite clinical studies addressing severe unmet need in children with ABCC6 Deficiency

BOSTON, July 02, 2024 (GLOBE NEWSWIRE) -- Inozyme Pharma, Inc. (Nasdaq: INZY) (“the Company” or “Inozyme”), a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of pathologic mineralization and intimal proliferation, today announced that the U.S. Food and Drug Administration (FDA) has granted Fast Track designation to INZ-701 for the treatment of ABCC6 Deficiency.

“Through Fast Track designation, the FDA recognizes the potential of INZ-701 in ABCC6 Deficiency. We plan to work closely with the agency to establish an efficient path to approval. Receipt of Fast Track designation underscores our belief that INZ-701 could serve as an important therapy for patients living with ABCC6 Deficiency, notably for pediatric patients in whom this condition increases the risk of major clinical events such as stroke and severe neurological and cardiovascular disease,” said Douglas A. Treco, Ph.D., CEO and Chairman of Inozyme Pharma. “We look forward to presenting our development plans to regulatory agencies and reaching agreement on a pivotal study in pediatric patients with ABCC6 Deficiency by year-end 2024.”

Fast Track is an FDA program designed to facilitate and expedite the development and review of new medicines that are intended to treat or prevent serious conditions and have the potential to address an unmet medical need. The designation has been granted based on nonclinical pharmacology data and preliminary safety and efficacy data from the Company’s ongoing Phase 1/2 trial of INZ-701 in adults with ABCC6 Deficiency. With Fast Track designation, the development of INZ-701 can benefit from more frequent engagement with the FDA and expedited regulatory review.

In April 2024, the Company announced positive topline safety and immunogenicity data from its ongoing Phase 1/2 trial of INZ-701 in adults with ABCC6 Deficiency. Clinical improvements were observed in vascular pathology, visual function, and patient reported outcomes (PROs). The Company also reported initial findings from natural history studies which indicate a substantial disease burden among pediatric patients with ABCC6 Deficiency, manifesting as a high incidence of major clinical events, notably stroke, severe neurological disease, and severe cardiovascular disease, occurring early in life.

About ABCC6 Deficiency

ABCC6 Deficiency is a progressively debilitating condition of the vasculature and soft tissue that is estimated to affect approximately 1 in 25,000 to 1 in 50,000 individuals worldwide. Infants with ABCC6 Deficiency are diagnosed with generalized arterial calcification of infancy (GACI Type 2), a condition that resembles GACI Type 1, the infant form of ENPP1 Deficiency. Pediatric patients who survive the first year of life may develop neurological disease, including stroke, and cardiovascular disease secondary to ongoing vascular calcification and stenosis. In older individuals, ABCC6 Deficiency presents as pseudoxanthoma elasticum (PXE), which is characterized by pathologic mineralization in blood vessels and soft tissues clinically affecting the skin, eyes, and vascular system. There are no approved therapies for ABCC6 Deficiency.

About INZ-701

INZ-701, a recombinant Fc fusion protein, is an ENPP1 enzyme replacement therapy in development for the treatment of rare disorders of the vasculature, soft tissue, and skeleton. In preclinical studies, the experimental therapy has shown potential to prevent pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in devastating genetic disorders such as ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis. INZ-701 is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis.

About Inozyme Pharma

Inozyme Pharma, Inc. is a clinical-stage rare disease biopharmaceutical company developing novel therapeutics for the treatment of diseases impacting the vasculature, soft tissue, and skeleton. Inozyme is developing INZ-701, an enzyme replacement therapy, to address pathologic mineralization and intimal proliferation, which can drive morbidity and mortality in these severe diseases. INZ-701 is currently in clinical development for the treatment of ENPP1 Deficiency, ABCC6 Deficiency and calciphylaxis.

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 availability of data from clinical trials, and 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 and ABCC6 Deficiency; 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

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


FAQ

What is the Fast Track designation granted to INZ-701 for?

The Fast Track designation was granted to INZ-701 for the treatment of ABCC6 Deficiency, a serious condition with significant unmet medical needs.

When does Inozyme plan to finalize the pivotal study for INZ-701?

Inozyme plans to finalize the pivotal study for INZ-701 in pediatric patients by the end of 2024.

What were the preliminary trial results for INZ-701 in adults with ABCC6 Deficiency?

Preliminary trial results showed positive safety and immunogenicity data, with clinical improvements in vascular pathology, visual function, and patient-reported outcomes.

What are the potential benefits of the Fast Track designation for INZ-701?

The Fast Track designation allows for more frequent engagement with the FDA and expedited regulatory review, potentially speeding up the approval process.

Inozyme Pharma, Inc.

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