Biohaven Initiates BHV-0223’s Pivotal Clinical Trial to Demonstrate Bioequivalence to Rilutek

Biohaven Initiates BHV-0223’s Pivotal Clinical Trial to Demonstrate Bioequivalence to Rilutek

Biohaven Pharmaceutical has dosed the first amyotrophic lateral sclerosis (ALS) patient with its sublingual formulation of riluzole, BHV-0223, in a pivotal Phase 2/3 trial. The trial aims to demonstrate that BHV-0223’s effects are at least equivalent to Rilutek, an approved tablet formulation of riluzole.

The trial initiation was supported by the U.S. Food and Drug Administration‘s approval of the investigational new drug (IND) application for BHV-0223, granted earlier this month.

“We are excited to advance the unique BHV-0223 Zydis orally dissolving tablet (ODT) formulation into the clinic,” Vlad Coric, MD, CEO of Biohaven, said in a press release. “If positive, the current study will establish the bioequivalence of sublingually administered BHV-0223 and thereby offer patients an optimized formulation with enhanced pharmaceutic properties.”

BHV-0223 is a sublingual ODT formulation of riluzole, designed to reduce the limitations of Sanofi-Aventis’ Rilutek, an FDA-approved formulation of riluzole.

In addition to having somewhat marginal effects of survival, Rilutek must be swallowed and taken at least one hour before, or two hours after a meal, due to potential loss of stability. The difficulties in swallowing experienced by ALS patients and the limitations in administration time make Rilutek far from ideal for these patients.

The new formulation is expected to overcome these limitations, while retaining its therapeutic potential. The new tablets use Catalent’s Zydis disintegrating technology, which allows the rapid disintegration and absorption of the drug through the small vessels below the tongue. This not only allows patients with swallowing difficulties the ability to take the drug, it also prevents adverse interactions with food.

Results of a Phase 1 trial showed that BHV-0223 was safe overall, and a 35 mg dose was associated with less variability in the drug’s blood levels than 50 mg of Rilutek.

The newly initiated Phase 2/3 study will now assess if BHV-0223 behaves in the body in a similar manner as Rilutek, and induces at least equivalent effectiveness and safety results.

In December 2016, the FDA granted the company orphan drug designation for BHV-0223 for the treatment of ALS. Biohaven also has received regulatory feedback from the FDA stating that after this study, no additional efficacy or toxicology studies are needed for BHV-0223’s approval.

4 comments

  1. Carol Mongiello says:

    So I’m having difficulty understanding the benefits of this drug sublingual. I only know 2 people who have taken this drug in pill form. 1 had GI symptoms and was not able to take the drug. The other, my son had elevated liver enzymes therefore he had to stop. If you look up this drug in the internet it states it may increase survival for 2 to 3 months and delay the onset of ventilator dependence. That being said I know the drug is over 20 years old when no one was looking for a cure just trying to prolong life. At what cost? Did it do anything to improve quality of life? I think not. So tell me factually how many people with als this drug is actually helping? There are so many theories out there and researchers looking into these different theories why not focus on a halt of the disease or cure of the disease? I know this is where I would want my money spent. 2 years ago when my son was diagnosed there was only Riluzole. A drug 20 years old. Today I know there are at least 10 drugs in investigational studies looking for a halt or cure for this disease. I urge everyone to look up THE RIGHT to Try Act. This may give hope for those who have none. Contact your legislatures and demand they support this act. It’s RIGHT. Frankie’s Mom, Carol.

    • Dave Shuey, PhD says:

      Keep fighting Carol. FYI – My wife is taking an NAD+ elevating supplement. We aren’t waiting for the clinic. I urge you and others to look into this safe and neuroprotective therapy. About $1/day. For starters, it works to ameliorate neurodegeneration in SOD- mice. I cant believe there isn’t more buzz about this. Good Luck. Don’t give up!

    • Dave Shuey says:

      It is called “Basis” and is made by Elysium. I encourage you to visit their website at http://www.elysiumhealth.com. Its very good and has plenty of mechanistic information. Their SAB (Scientific Advisory Board) is unparalleled. It costs $480/yr.
      Cheaper NAD+ elevating supplements are out there (Niagen, for example) that provide the same precursor of NAD+ but lack the sirtuin activating compound pterostilbene. The sirtuin regulators essentially carry the NAD+ signal to the gene expression level.

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