Applied Therapeutics Inc (APLT)

History and Background

Applied Therapeutics Inc. was founded in 2016 and is a biopharmaceutical company developing novel therapeutics for unmet medical needs in rare diseases. It focuses on discovering, developing, and commercializing small molecule drugs targeting intracellular drug targets.

Core Business Areas

• AT-007 (Gavoreostat) Program: Focuses on developing Gavoreostat for the treatment of Sorbitol Dehydrogenase (SORD) Deficiency, a rare metabolic disease.
• AT-001 Program: Formerly focused on diabetic cardiomyopathy, this program appears to be discontinued or deprioritized based on recent communications.

Leadership and Structure

Shoshana Shendelman is the founder, CEO, and President. The company has a typical structure for a biotech firm, with departments for research and development, clinical trials, regulatory affairs, and commercialization.

Key Offerings

• Gavoreostat (AT-007): A drug candidate in late-stage clinical development for the treatment of SORD deficiency. There are currently no approved therapies for this condition. The potential market is estimated to be substantial given the lack of treatment options and an estimated SORD incidence rate of 1/40,000 live births. Major competitors include other companies developing therapies for rare metabolic diseases, although none directly target SORD deficiency at this moment.

Industry Overview

The industry is focused on rare disease therapeutics. Characteristics include high R&D costs, lengthy regulatory processes, and potential for high returns due to orphan drug status.

Positioning

Applied Therapeutics is positioned as a rare disease specialist, leveraging its expertise to develop and commercialize targeted therapies for unmet medical needs. Its competitive advantage lies in the specificity of its programs and potentially quicker approval due to unmet medical needs.

Total Addressable Market (TAM)

The TAM for SORD deficiency is estimated to be in the hundreds of millions, based on prevalence, pricing of similar orphan drugs and global reach. Applied Therapeutics could capture a substantial portion of this TAM with an approved drug.

Key Competitors

• LLY
• VRTX
• BMY
• GILD
• REGN

Competitive Landscape

Applied Therapeutics competes in the broad rare disease space. Its competitive advantage would be the fact that there are no current FDA approved treatments for SORD deficiency. Thus, APLT could capture most or all of that market. Competitors have bigger cash reserves and a wider product line.

Historical Growth: Historical growth has been driven by pipeline advancement and financing activities.

Future Projections: Future growth depends on the successful development and commercialization of Gavoreostat and other pipeline candidates. Analyst projections vary widely but anticipate significant revenue growth upon potential approval of Gavoreostat.

Recent Initiatives: Recent initiatives include completion of the Phase 3 trial for Gavoreostat in SORD deficiency and preparation for regulatory submissions.

Applied Therapeutics is a high-risk, high-reward biopharmaceutical company focused on rare diseases. Its lead candidate, Gavoreostat, holds significant promise in treating SORD deficiency. However, the company faces significant challenges, including clinical trial risk, regulatory hurdles, and financing needs, making its future uncertain until Gavoreostat is approved and revenue is generated, it will need cash infusions to keep it going and to continue its work and efforts.

Data Sources:

• Company filings (SEC)
• Investor presentations
• Analyst reports
• Company website

Disclaimers:

This analysis is for informational purposes only and should not be considered investment advice. Financial data may be delayed. Market share data are estimates and subject to change. Consult with a qualified financial advisor before making any investment decisions.