Hypophosphatasia - Pipeline Insight, 2025

DelveInsight's, "Hypophosphatasia - Pipeline Insight, 2025" report provides comprehensive insights about 3+ companies and 5+ pipeline drugs in Hypophosphatasia pipeline landscape. It covers the pipeline drug profiles, including clinical and nonclinical stage products. It also covers the therapeutics assessment by product type, stage, route of administration, and molecule type. It further highlights the inactive pipeline products in this space.

Geography Covered:

• Global coverage

Hypophosphatasia: Understanding

Hypophosphatasia: Overview

Hypophosphatasia (HPP) is a rare, inherited metabolic disorder characterized by defective mineralization of bones and teeth due to mutations in the ALPL gene, which encodes the tissue-nonspecific alkaline phosphatase (TNSALP) enzyme. This enzyme plays a crucial role in bone formation by regulating phosphate and calcium levels. The disease presents with a wide range of clinical severity, from life-threatening forms in infancy to milder forms in adulthood, often involving premature tooth loss, fractures, muscle weakness, and skeletal abnormalities.

The symptoms are highly variable in their clinical expression, which ranges from stillbirth without mineralized bone to early loss of teeth without bone symptoms. Depending on the age at diagnosis, six clinical forms are currently recognized: perinatal (lethal), perinatal benign, infantile, childhood, adult and odontohypophosphatasia. In the lethal perinatal form, the patients show markedly impaired mineralization in utero. In the prenatal benign form these symptoms are spontaneously improved. Clinical symptoms of the infantile form are respiratory complications, premature craniosynostosis, widespread demineralization and rachitic changes in the metaphyses.

The disease is due to mutations in the liver/bone/kidney alkaline phosphatase gene (ALPL; OMIM# 171760) encoding the tissue-nonspecific alkaline phosphatase (TNAP or TNSALP). TNAP is a phosphomonoesterase of 507 residues, anchored at its carboxyl terminus to the plasma membrane by a phosphatidylinositol-glycan moiety. The enzyme is physiologically active in its dimeric form and cleaves extracellular substrates pyridoxal-5'-phosphate (PLP), phosphoethanolamine (PEA) and inorganic pyrophosphates (PPi). Its exact function in bone and dental mineralization is still unclear but involves hydrolysis of PPi, and perhaps mammalian-specific activities such as collagen and calcium binding.

Screening for mutations in the TNAP gene is essential to confirm the hypophosphatasia diagnosis when biochemical and clinical data are not clear enough, to offer genetic counseling or to offer molecular prenatal diagnosis to families affected by severe forms of the disease (see below). Clinical and biochemical data may not always distinguish hypophosphatasia from other skeletal diseases such as osteogenesis imperfecta. Mutation screening may be performed by single-stranded conformation polymorphism (SSCP) or denaturing gradient gel electrophoresis (DGGE) followed by sequencing of exons exhibiting variants, by direct sequencing of the cDNA or by direct sequencing of genomic sequences.

"Hypophosphatasia- Pipeline Insight, 2025" report by DelveInsight outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Hypophosphatasia pipeline landscape is provided which includes the disease overview and Hypophosphatasia treatment guidelines. The assessment part of the report embraces, in depth Hypophosphatasia commercial assessment and clinical assessment of the pipeline products under development. In the report, detailed description of the drug is given which includes mechanism of action of the drug, clinical studies, NDA approvals (if any), and product development activities comprising the technology,

Hypophosphatasia collaborations, licensing, mergers and acquisition, funding, designations and other product related details.

Report Highlights:

• The companies and academics are working to assess challenges and seek opportunities that could influence Hypophosphatasia R&D. The therapies under development are focused on novel approaches to treat/improve Hypophosphatasia.

Hypophosphatasia Emerging Drugs Chapters

This segment of the Hypophosphatasia report encloses its detailed analysis of various drugs in different stages of clinical development, including phase III, II, I, preclinical and Discovery. It also helps to understand clinical trial details, expressive pharmacological action, agreements and collaborations, and the latest news and press releases.

Hypophosphatasia Emerging Drugs

• Efzimfotase alfa: AstraZeneca

Efzimfotase alfa (ALXN-1850) is an advanced recombinant tissue-nonspecific alkaline phosphatase (TNSALP) enzyme replacement therapy developed by Alexion, AstraZeneca Rare Disease, for the treatment of hypophosphatasia (HPP), a rare inherited disorder characterized by deficient TNSALP activity leading to impaired bone mineralization. The drug is designed to substitute the missing or deficient TNSALP enzyme, thereby reducing the accumulation of its substrates such as inorganic pyrophosphate (PPi) and pyridoxal-5'-phosphate (PLP), which inhibit bone mineralization. By restoring alkaline phosphatase activity, ALXN-1850 enhances bone mineralization and addresses systemic manifestations of HPP. Currently, the drug is in Phase III stage of its clinical trial for the treatment of Hypophosphatasia.

Hypophosphatasia: Therapeutic Assessment

This segment of the report provides insights about the different Hypophosphatasia drugs segregated based on following parameters that define the scope of the report, such as:

• Major Players in Hypophosphatasia
• There are approx. 3+ key companies which are developing the therapies for Hypophosphatasia. The companies which have their Hypophosphatasia drug candidates in the most advanced stage, i.e. Phase III include, AstraZeneca.
• Phases

DelveInsight's report covers around 5+ products under different phases of clinical development like

• Late stage products (Phase III)
• Mid-stage products (Phase II)
• Early-stage product (Phase I) along with the details of
• Pre-clinical and Discovery stage candidates
• Discontinued & Inactive candidates
• Route of Administration

Hypophosphatasia pipeline report provides the therapeutic assessment of the pipeline drugs by the Route of Administration. Products have been categorized under various ROAs such as

• Oral
• Intravenous
• Subcutaneous
• Parenteral
• Topical
• Molecule Type

Products have been categorized under various Molecule types such as

• Recombinant fusion proteins
• Small molecule
• Monoclonal antibody
• Peptide
• Polymer
• Gene therapy
• Product Type

Drugs have been categorized under various product types like Mono, Combination and Mono/Combination.

Hypophosphatasia: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Hypophosphatasia therapeutic drugs key players involved in developing key drugs.

Pipeline Development Activities

The report covers the detailed information of collaborations, acquisition and merger, licensing along with a thorough therapeutic assessment of emerging Hypophosphatasia drugs.

Hypophosphatasia Report Insights

• Hypophosphatasia Pipeline Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Hypophosphatasia Report Assessment

• Pipeline Product Profiles
• Therapeutic Assessment
• Pipeline Assessment
• Inactive drugs assessment
• Unmet Needs

Key Questions:

Current Treatment Scenario and Emerging Therapies:

• How many companies are developing Hypophosphatasia drugs?
• How many Hypophosphatasia drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Hypophosphatasia?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Hypophosphatasia therapeutics?
• What are the recent trends, drug types and novel technologies developed to overcome the limitation of existing therapies?
• What are the clinical studies going on for Hypophosphatasia and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• AstraZeneca
• AM-Pharma
• Be Biopharma
• Rallybio

Key Products

• Efzimfotase alfa
• Ilofotase Alfa
• BE-102
• REV102