Pasithea Receives Funding to Develop Antibodies for ALS

Margarida Maia, PhD avatar

by Margarida Maia, PhD |

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Pasithea Therapeutics has gained access to AU$1 million (about $694,000) to further its research into anti-integrin antibodies as potential treatments for amyotrophic lateral sclerosis (ALS).

The non-dilutive funds became available following the company’s acquisition of Alpha-5 Integrin, the preclinical biotech company that originally developed the experimental antibodies for ALS. Alpha-5 had received a drug development award from FightMND, an Australian nonprofit patient advocacy group, which now will be transferred to Pasithea.

Non-dilutive funding means that the company receives the money without giving away any of its equity shares or ownership to the foundation.

“With the transfer of this grant, we will have a source of non-dilutive funds to test our monoclonal antibodies in the TDP-43 mouse model and progress the candidate toward the clinic,” Tiago Reis Marques, MD, PhD, CEO and director of Pasithea Therapeutics, said in a company press release.

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“We expect to develop and start human clinical trials by the end of 2023,” Lawrence Steinman, MD, executive chairman and co-founder of Pasithea, said in a webcast announcing the acquisition of Alpha-5. Steinman is a professor of neurology and neurological sciences, pediatrics, and genetics at Stanford Medical School in California.

ALS occurs when motor neurons — the cells that control voluntary movement — become progressively damaged and die. This leads to disease symptoms that range from trouble walking and speaking, to difficulty swallowing and breathing.

The reasons why ALS develops are not completely understood, but inflammation in the central nervous system is believed to play a significant role in disease onset and progression.

Members of the integrin family of proteins are found on the surface of cells, and are important for enabling the circulation of immune cells from the bloodstream into sites of inflammation.

A particular integrin, called alpha-5/beta-1 or fibronectin receptor, has been found at high levels in certain immune cells in an animal model of ALS carrying mutations in the SOD1 gene. Mutations in this gene are estimated to account for up to 20% of familial ALS cases, and up to 3% of sporadic ALS cases.

In people with ALS, this integrin is found in immune cells close to the blood-brain barrier, a semipermeable membrane that controls which substances can travel from the blood into the brain, and near the diseased motor neuron areas. Its levels also appear to increase as the disease progresses.

Slowing disease progression

Researchers at Pasithea believe that antibodies targeting the alpha-5/beta-1 integrin may prevent the overactive immune cells from breaching the blood-brain barrier and reaching motor neurons, thereby slowing disease progression.

Studies in SOD1 animal models found that an antibody against this molecule reduced inflammation in the spinal cord, improved motor function, and helped the animals live longer.

Now, the company plans to test several antibodies with distinct blocking abilities, from partial blockers to strong blockers, in a mouse model of ALS carrying mutations in the TDP-43 gene, and eventually advance a lead candidate into the clinic.

The roadmap to a potential treatment includes selecting a lead candidate by the end of this year and completing toxicology studies by the second quarter of 2023. Then, toward the end of 2023, the company plans to discuss with the regulatory authorities about an orphan drug designation and the filing of an investigational new drug application requesting the start of clinical studies.