Portage Biotech Inc. (Portage), engaged in identifying, financing and developing novel therapeutics in indications with high unmet medical need, and Biohaven Pharmaceutical Holding Company Limited (Biohaven), a privately-held biopharmaceutical company, announced positive results from a phase I study with BHV-0223, a glutamate modulating agent. The final study results confirm that the pharmacokinetic, safety, and tolerability data with sublingual BHV-0223 support advancement of its clinical development. Additionally, lower doses of BHV-0223 appear to have a similar exposure profile to the current standard of care medication.

BHV-0223 is a unique formulation of riluzole, a glutamate modulating agent, that utilizes the Zydis ODT fast-dissolve technology under an exclusive worldwide agreement with Catalent.

The phase I trial was designed to demonstrate the safety and unique pharmacokinetic characteristics of BHV-0223, in single and then multiple dosing in humans. A comparison arm with the generic standard of care riluzole was included in the study to demonstrate potential advantages of the unique BHV-0223 formulation and route of administration. In the first phase of the study, approximately 10 participants were treated with varying doses of BHV-0223 on four separate occasions. In the second phase of the trial, participants received multiple daily doses of BHV-0223. The study tested three doses of BHV-0223 along with the standard oral tablet formulation of riluzole.

Dosing with BHV-0223 showed favorable pharmacokinetic properties. That is, as compared to the oral tablet formulation, BHV-0223 demonstrated faster absorption and, on a dose-normalized basis, higher peak concentrations and greater exposure. With regard to AEs, the most common AE was transient and mild oral numbness. Of note, there were no severe or serious adverse events (AEs), and the vast majority of AEs were considered mild.

Robert Berman, M.D., chief medical officer of Biohaven commented, "This data is exciting as it shows that our novel formulation is able to achieve equivalent exposures with lower doses of the active pharmaceutical ingredient, thereby reducing overall drug burden to patients. In addition, BHV-0223 provides a new route of administration, especially important for those patients with difficulty swallowing. We believe that BHV-0223 offers tangible benefits over the standard oral formulation." Biohaven's plans for BHV-0223 will hinge on feedback from the FDA regarding its previously submitted Pre-Investigational New Drug Application (PIND) meeting request with the FDA for Amyotrophic Lateral Sclerosis.