Dosing of healthy adults begins in Phase 1 trial of Alzheimer’s therapy

OLX-07010 designed to block tau protein clumping at its earliest stages

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by Andrea Lobo |

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Dosing has begun in a first group of healthy adults in a Phase 1a clinical trial of OLX-07010, Oligomerix’s lead treatment candidate for Alzheimer’s disease and other neurodegenerative disorders.

OLX-07010 is an oral small molecule inhibitor of tau self-association — one of the initial steps leading to tau aggregation, a process thought to be implicated in the development of Alzheimer’s and similar disorders.

“With its targeted safety and efficacy profile, OLX-07010 has the potential to fill a significant void in treatment options for patients suffering from devastating neurodegenerative diseases,” James Moe, PhD, CEO at Oligomerix, said in a company press release.

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Early studies in mice show OLX-07010 prevented toxic tau clumping

While the exact causes of Alzheimer’s disease elude scientists, its symptoms are attributed to the toxic accumulation of beta-amyloid and tau protein aggregates inside nerve cells in the brain, resulting in their premature death.

Tau is a protein that normally transports nutrients and other molecules to brain cells. In Alzheimer’s, tau acquires abnormal shapes that aggregate into “tau tangles,” disrupting normal cellular transport and eventually killing brain cells.

By targeting tau self-association, OLX-07010 is expected to help mitigate the effects of tau aggregation.

Use of the investigational therapy effectively prevented tau aggregation in early studies involving mouse models of neurodegeneration, Oligomerix reported.

“Over the last two years, Oligomerix has made significant progress in demonstrating how inhibiting tau self-association can have remarkable effects in preclinical models of [Alzheimer’s disease] and neurodegenerative conditions,” said William Erhardt, MD, the company’s president and head of development and operations.

A 2020 study in a mouse model of tauopathy — a condition characterized by the deposition of abnormal tau protein in the brain — reported that treatment with OLX-07010 significantly lowered self-associated tau levels, as well as the total number of insoluble tau aggregates.

Additional experiments in another mouse model of tauopathy, with “more aggressive” tau misfolding and accumulation, showed similar benefits with five months of OLX-07010 given to the animals, Oligomerix reported.

“The activity in these two models … [show] that targeting tau-self association can inhibit the entire tau aggregation pathway,” it stated on a therapy webpage.

The Phase 1a study (NCT05696483) is evaluating the safety, tolerability, and pharmacological properties of single- and, later, multiple-ascending doses of OLX-07010 in healthy adults, ages 18-50. If findings are favorable, a single dose also will be given to older healthy adults, those ages 51 to 75 and making up the treatment’s target population.

The trial is enrolling adults by invitation at its single site in California.

“We look forward to working closely with our stakeholders, from patients to caregivers to regulatory officials, as we characterize the clinical profile of OLX-07010,” Erhardt said.

As an oral small molecule, OLX-07010 is expected to be easy to administer and may allow for lower manufacturing costs, the company stated on its website.

OLX-07010 also may complement current antibody-based therapies for Alzheimer’s, such as Leqembi (lecanemab) and Aduhelm (aducanamab), which are designed to bind and clear toxic versions of beta-amyloid, Oligomerix stated.