Porphyria - Pipeline Insight, 2024

This Porphyria - Pipeline Insight, 2024,” report provides comprehensive insights about 10+ companies and 10+ pipeline drugs in Porphyria 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

Porphyria Understanding

Porphyria: Overview

Erythropoietic Protoporphyria (EPP) is an inherited disorder of the heme biosynthetic pathway that results from mutations of the ferrochelatase (FECH) gene or, less commonly X-Linked Protoporphyria (XLP) that results from mutations in the aminolevulinic acid synthase-2 (ALAS2) gene. Both EPP and XLP are characterized by accumulation of protoporphyrin in blood, erythrocytes and tissues and cutaneous photosensitivity. EPP and XLP usually present early in childhood with extremely painful phototoxic reactions which are preceded by a “prodrome” of tingling, stinging, and/or burning of sun-exposed skin. The onset of prodromal symptoms after direct sun exposure varies but may occur in less than 10 minutes.

Porphyria - Pipeline Insight, 2024 report outlays comprehensive insights of present scenario and growth prospects across the indication. A detailed picture of the Porphyria pipeline landscape is provided which includes the disease overview and Porphyria treatment guidelines. The assessment part of the report embraces, in depth Porphyria 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, Porphyria 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 R&D Porphyria. The therapies under development are focused on novel approaches to treat/improve Porphyria.

In September 2021, Disc Medicine, a clinical-stage biotechnology company dedicated to the discovery and development of novel therapies for the treatment of serious and debilitating hematologic diseases, announced a $90 million Series B financing from a leading syndicate of investors. The proceeds of the financing will be used to advance the development of Disc's hematology pipeline of therapeutic candidates. This includes conducting a phase 2 study of its lead program bitopertin, an oral, clinical-stage GlyT1 inhibitor with potential to become the first disease-modifying treatment for erythropoietic porphyrias (EP), a family of rare and debilitating genetic disorders caused by dysregulated heme synthesis.

Porphyria Emerging Drugs

Dersimelagon: Mitsubishi Tanabe PharmaDersimelagon is a novel synthetic, orally-administered, non-peptide small molecule, which acts as a selective agonist of melanocortin-1 receptor (MC1R) with a potential for being effective in the prevention of phototoxicity in erythropoietic protoporphyria (EPP) patients. Mitsubishi Tanabe Pharma Corporation (MTPC) is developing dersimelagon for the treatment of EPP. ENDEAVOR is a Phase II proof of concept study for MT-7117 as a treatment option for EPP. ENDEAVOR met its primary endpoint of change from baseline in average daily time (minutes) to first prodromal symptom associated with sunlight exposure between 1 hour post sunrise and 1 hour pre-sunset at week 16. MT-7117 was generally well tolerated with an acceptable safety profile. Mitsubishi Tanabe Pharma Corporation received Fast Track Designation for dersimelagon by the U.S. Food and Drug Administration in June 2018
DISC-0974: Disc MedicineDISC-0974 is an investigational, first-in-class monoclonal antibody designed to suppress hepcidin production by inhibiting the hemojuvelin (HJV) co-receptor, a highly selective and critical target of the hepcidin pathway. Hepcidin is the primary regulatory hormone of iron homeostasis and plays a central role by restricting iron absorption and preventing deployment from internal iron stores. DISC-0974 is being developed as a potential treatment for anemia of inflammation by suppressing hepcidin and enhancing iron availability for erythropoiesis. Preclinical studies of DISC-0974 and human genetic evidence validate that inhibition of HJV results in potent suppression of hepcidin and increased serum iron.

Porphyria: Therapeutic Assessment

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

Major Players in Porphyria

There are approx. 10+ key companies which are developing the therapies Porphyria. The companies which have their Porphyria drug candidates in the most advanced stage, i.e Phase III include, Mitsubishi Tanabe Pharma.

Phases

This report covers around 10+ 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

Porphyria 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

Molecule Type

Products have been categorized under various Molecule types such as

• Small molecule
• Cell Therapy
• Peptides
• Polymer
• Small molecule
• Gene therapy

Product Type

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

Porphyria: Pipeline Development Activities

The report provides insights into different therapeutic candidates in phase II, I, preclinical and discovery stage. It also analyses Porphyria 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 Porphyria drugs.

Porphyria Report Insights

• Porphyria Analysis
• Therapeutic Assessment
• Unmet Needs
• Impact of Drugs

Porphyria 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 Porphyria drugs?
• How many Porphyria drugs are developed by each company?
• How many emerging drugs are in mid-stage, and late-stage of development for the treatment of Porphyria?
• What are the key collaborations (Industry-Industry, Industry-Academia), Mergers and acquisitions, licensing activities related to the Porphyria 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 Porphyria and their status?
• What are the key designations that have been granted to the emerging drugs?

Key Players

• Clinuvel Pharmaceuticals
• Mitsubishi Tanabe Pharma
• Gilead Sciences
• Disc Medicine
• Atlas Molecular Pharma

Key Products

• Afamelanotide PII
• Dersimelagon PIII
• Ledipasvir/sofosbuvir
• DISC-1459
• Ciclopirox

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