Apilimod mesylate is under clinical development by OrphAI Therapeutics and currently in Phase II for Amyotrophic Lateral Sclerosis. According to GlobalData, Phase II drugs for Amyotrophic Lateral Sclerosis have a 65% phase transition success rate (PTSR) indication benchmark for progressing into Phase III. GlobalData’s report assesses how Apilimod mesylate’s drug-specific PTSR and Likelihood of Approval (LoA) scores compare to the indication benchmarks. Buy the report here.

GlobalData tracks drug-specific phase transition and likelihood of approval scores, in addition to indication benchmarks based off 18 years of historical drug development data. Attributes of the drug, company and its clinical trials play a fundamental role in drug-specific PTSR and likelihood of approval.

Apilimod mesylate overview

Apilimod mesylate is under development for the treatment of follicular lymphoma, diffuse large b-cell lymphoma, mantle cell lymphoma, amyotrophic lateral sclerosis, frontotemporal dementia and coronavirus disease 2019 caused by the severe acute respiratory syndrome coronavirus 2 (SARS-CoV-2). The drug candidate is administered through oral route. It acts by targeting 1 phosphatidylinositol 3 phosphate 5 kinase (PIKFYVE). It was also under development for follicular lymphoma, diffuse large b-cell lymphoma, mantle cell lymphoma, marginal zone lymphoma, primary mediastinal b-cell lymphoma, or chronic lymphocytic leukemia/small lymphocytic lymphoma (CLL/SLL).

OrphAI Therapeutics overview

OrphAI Therapeutics (OrphAI), formerly AI Therapeutics Inc, is a biopharmaceutical company that identifies and develops treatments for neurological diseases and cancers. It investigates LAM-001 program against pulmonary sarcoidosis and bronchiolitis obliterans disease caused after lung transplantation resulting in obstruction of the smallest airways of the lungs. The company is also evaluating AIT–101 drugs to treat amyotrophic lateral sclerosis and AIT-102 for rare diseases. OrphAI is headquartered in Guilford, Connecticut, the US.

For a complete picture of Apilimod mesylate’s drug-specific PTSR and LoA scores, buy the report here.

This content was updated on 7 February 2024

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GlobalData, the leading provider of industry intelligence, provided the underlying data, research, and analysis used to produce this article.

GlobalData’s Likelihood of Approval analytics tool dynamically assesses and predicts how likely a drug will move to the next stage in clinical development (PTSR), as well as how likely the drug will be approved (LoA). This is based on a combination of machine learning and a proprietary algorithm to process data points from various databases found on GlobalData’s Pharmaceutical Intelligence Center.