Tonix, French Research Group Partner to Understand Oxytocin’s Potential in PWS

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by Marta Figueiredo PhD |

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Tonix Pharmaceuticals has entered a research agreement with Inserm Transfert, the private subsidiary of the French National Institute of Health and Medical Research (Inserm), to study the mechanisms behind the reported benefits of the oxytocin hormone in a mouse model of Prader-Willi syndrome (PWS).

Inserm Transfert signed the deal on behalf of Inserm and the Aix-Marseille University in France — which, together with the Toulouse University Hospital, previously signed a licensing agreement providing Tonix with oxytocin-based therapeutic technology.

Based on the earlier deal, the company is developing TNX-2900, a more potent form of oxytocin delivered via the nose (intranasally) as a potential treatment to lessen insatiable hunger in adolescents and adults with PWS.

“Tonix is excited to enter into this new research collaboration, which we hope will expand our understanding of oxytocin’s potential to treat Prader-Willi syndrome,” for which there is no approved treatment to date, Seth Lederman, MD, Tonix’s CEO, said in a press release.

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Pregnancy, Newborn Characteristics May Help Distinguish PWS Infants

PWS is a complex disease associated with changes in feeding behavior, including suckling difficulties and insufficient weight gain in infancy, and insatiable hunger, or hyperphagia, that starts during childhood and typically leads to obesity.

The production of oxytocin, a hormone involved in several behaviors and feelings — including empathy, trust, and satisfaction from food — is deficient in PWS patients.

In animal models of the disease, intranasal oxytocin was shown to improve suckling in newborn animals and to suppress feeding in adult animals, supporting its potential benefit in this patient population.

To date, several clinical trials have evaluated the safety and effectiveness of intranasal oxytocin in infants, children, adolescents, and adults with PWS, producing mixed results.

Several of them were sponsored by and took place at the Toulouse University Hospital, with a placebo-controlled Phase 3 trial, called OTBB3 (NCT04283578), testing intranasal oxytocin in newborns and infants with the disease.

Notably, Levo Therapeutics’ LV-101 (intranasal carbetocin) — a lab-made compound designed to mimic oxytocin — was rejected for approval by the U.S. Food and Drug Administration (FDA). The agency is now asking for an additional clinical trial to confirm the therapy’s efficacy at easing hyperphagia in PWS patients.

Tonix’s patented, potentiated oxytocin formulation is believed to have increased specificity to oxytocin receptors as well as greater potency.

“With this new research collaboration, our goal is to learn how oxytocin-based pharmacological treatment improves and normalizes feeding behavior in infancy,” Lederman said.

The research will be led by Francoise Muscatelli, PhD, of the Institute of Mediterranean Neurobiology, which is affiliated to INSERM and Aix-Marseille University.

Muscatelli and colleagues will use lab-grown mouse cells and a mouse model of PWS to characterize the mechanism by which oxytocin normalizes the suckling and maturation of feeding behavior during infancy.

Results are expected to shed light on how oxytocin regulates feeding behavior in PWS.

Tonix has filed an application to the FDA seeking orphan drug designation for TNX-2900. Previously, the company expressed plans to also ask for fast track status.

These designations are meant to speed TNX-2900’s clinical development and regulatory review by providing financial incentives, a marketing exclusivity period if approved, regulatory support, and the possibility of priority review.

Tonix is also developing TNX-1900, a related intranasal potentiated oxytocin product, to treat chronic migraine. It expects to launch a Phase 2 trial testing the therapy for that indication later this year.