Yale School of Medicine Takes on Promise of Apilimod in Phase 2 Clinical Trial with AI Therapeutics

Jul 28, 2020 | AI Therapeutics, Apilimod, COVID-19, News, Yale School of Medicine

Yale School of Medicine Takes on Promise of Apilimod in Phase 2 Clinical Trial with AI Therapeutics

Yale School of Medicine and biopharmaceutical company AI Therapeutics launched a multi-institutional clinical trial of a drug known as LAM-002A (apilimod), a drug with a proven safety record. In fact, a recent study from Sanford Burnham Prebys Medical Discovery Institute showcased 21 potential drugs targeting SARS-CoV-2 including apilimod ( LAM-002A).

The Drug

LAM-002A is a first in class, highly selective PIKfyve kinase inhibitor1,2 that has demonstrated potent in vitro antiviral activity against several isolates of SARS-CoV-2, the virus responsible for COVID-19. Several studies have revealed that LAM-002A interferes with the entry and trafficking of the SARS-CoV-2 virus in cells. Given its mechanism of action, LAM-002 also has the potential to become part of combination therapies, especially with other drugs that target viral proteins or functions such as viral replication.

The Basis for the Study

Previous trials involving more than 700 patients have shown LAM-002A to be safe for the treatment for autoimmune diseases and follicular lymphoma. The drug has received Fast Track Status and Orphan Drug Designation from the Food & Drug Administration for treatment of lymphoma.

TrialSite recently reported the recent study from Sanford Burnham Prebys Medical Discovery Institute that showcased 21 potential drugs targeting SARS-CoV-2 including apilimod, which is LAM-002A.

The Study

This Phase 2, randomized, double-blind, placebo-controlled study of LAM-002A for the prevention of progression of COVID-19 commenced in July, 2020. All study participants must sign a written informed consent and satisfy the inclusion and exclusion criteria. The actual confirmation of SARS-CoV-2 infection will be done via RT-PCF test, medical history, and current medication is also assessed for each consenting participant at preliminary screening.

Randomized in a 1:1 ratio, study participants receive either the study therapy (LAM-002A, 125 mg [5 capsules/dose]) PO BID or placebo for ten days. Participants who experience an adverse event (AE) considered to be related to study therapy may have a decrease in study dose of the investigational drug to 100mg [4 capsules/dose] PO Bid. After the start of the treatment on Day 1, the study team will follow participants at Days 4,6,8,11,22, and 28.  Of course participants can withdraw from the study at any time reports the sponsor in Clinicaltrials.gov.

The study will incorporate an interim safety analysis after the first 30 participants (15 on LAM-002A and 15 on placebo) have completed treatment and have been followed up for 11 days post-first dose. Recruitment and randomization will continue while this analysis is conducted. Recommendations from an independent Data Safety Monitoring Board (DSMB) will be used for decisions of early termination or study design adaptations.

Non-parametric and parametric statistical analysis will be conducted, as appropriate. For the comparison of the LAM-002A active arm and the control arm for the primary endpoint and secondary endpoints of drug effect, appropriate methods will be employed. Baseline subject characteristics, study therapy administration/compliance, safety, supportive care administration, and pharmacokinetics will be analyzed descriptively.

Yale Point of View

Murat Gunel, professor of neurosurgery and professor of genetics and neuroscience reported, “LAM-002A holds promise to be a powerful new therapy for COVID-19 patients to prevent progression of disease, hopefully avoiding the need for hospitalization.” Professor Gunel does have a financial interest in AI Therapeutics as he serves the company as chief scientific advisor.

A treatment is desperately needed to help prevent the development of COVID-19 after exposure, especially in high-risk segments, such as the elderly in nursing homes, health care and frontline workers or people in underserved communities.

Meanwhile, principal investigator Charles S. Dela Cruz, associate professor of medicine and microbial pathogenesis and director of the Center for Pulmonary Infection Research at Yale University, commented, “We are delighted to be partnering with AI Therapeutics to see if LAM-002A can help ameliorate the devasting impact of this coronavirus pandemic on our society.”

About AI Therapeutics

Founded in 2013, AI Therapeutics promotes itself as an AI-driven company and touts its unique ability to match drugs to indications and prosecute through the clinical development process. Founded originally as LAM Therapeutics, its mission is to advance the clinical development of new therapies to treat Lymphangioleiomyomatosis (LAM) and other diseases.

Lead Research/Investigator

Charles Dela Cruz, MD, PhD

2 Comments

  1. Judith Evans

    Which company provides Lam-002A for the trials?

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