Key takeaways:

• The FDA granted Orphan Drug Designation to TRN-257 based on the hypothesis that it may be clinically superior to currently available oxybate products.
• The investigational treatment is designed as a once-nightly therapy, eliminating the need for patients to wake up for a second dose.
• If approved, TRN-257 would be the lowest-sodium once-nightly oxybate product available, containing 80 mg of sodium per 9 g dose.

The US Food and Drug Administration (FDA) has granted Orphan Drug Designation to TRN-257 (oxybate for extended-release oral suspension) for the treatment of idiopathic hypersomnia.

Developed by Tris Pharma Inc, TRN-257 received the designation based on the plausible hypothesis that it may be clinically superior to already available oxybate products for the same indication. The clinical superiority hypothesis centers on the drug’s once-nightly dosing, which may provide a major contribution to patient care for those with idiopathic hypersomnia, a chronic sleep disorder that potentially requires lifelong treatment.

“Idiopathic hypersomnia is a serious and often underrecognized disorder of sleep and wakefulness that can substantially impair daily functioning, quality of life, and long-term well-being,” says Maurice M. Ohayon, MD, DSc, PhD, professor and director, Stanford Sleep Epidemiology Research Center at Stanford University, in a release. “The FDA’s orphan drug designation for TRN-257 highlights the continuing need to advance differentiated therapeutic options for this patient population. A once-nightly, low-sodium oxybate formulation may be clinically meaningful if it can reduce nocturnal treatment burden while maintaining efficacy and safety.”

“The granting of orphan drug designation for TRN-257 recognizes the potential for a major contribution to patient care and marks an important milestone for the treatment of patients with idiopathic hypersomnia,” says Ketan Mehta, founder and CEO of Tris Pharma, in a release. “This milestone is a testament to the innovation of Tris scientists using our unique RaftWorks and LiquiXR platforms. TRN-257 is designed to provide clinically meaningful benefits to patients by combining once-nightly dosing with the lowest sodium content among oxybate products in development, which may be an important consideration for patients who are sensitive to or who need to manage their sodium intake.”

The Orphan Drug Designation program is intended to support the development of therapies for rare diseases or conditions affecting fewer than 200,000 people in the US. The designation provides certain development and commercial incentives, including eligibility for a seven-year period of market exclusivity in the US following product approval.

TRN-257 is currently being developed for the treatment of idiopathic hypersomnia, as well as for cataplexy or excessive daytime sleepiness associated with narcolepsy. The formulation utilizes two of Tris Pharma’s proprietary technology platforms to achieve its controlled-release profile and enable the once-nightly, low-sodium dosing regimen:

RaftWorks

• Enables sustained, controlled release and absorption of drugs that have a narrow window of absorption
• Slowly and continuously releases drug from the raft, enabling absorption in the upper gastrointestinal tract
• Employs an inter-penetrating polymer network to create an in situ floating “raft” that disintegrates with the use of pre-determined triggers

LiquiXR

• Particle-based technology that combines principles of ionic chemistry and science of controlled release to enable delivery in oral liquid and solid dosage forms
• Enables sustained, controlled release for oral medicines while maintaining benefits of immediate release
• Over nine NDAs approved in the US in addition to products approved in 33 countries around the world

Tris Pharma is actively exploring US and global partnership opportunities for the commercialization of TRN-257.