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But Vivek Ramaswamy, a 30-year-old former Wall Street hedge fund manager, told investors at a meeting in July that his company Axovant is "a single confirmatory study away" from getting FDA approval for his mild-to-moderate Alzheimer's disease treatment, currently called RVT-101.
Now, that phase 3 clinical trial has begun. The hope is to confirm the results of Axovant's last trial, which showed that patients taking RVT-101 in combination with Aricept, generically known donepezil, a widely used drug already available on the market and used to treat Alzheimer's symptoms like memory loss and confusion. The hope is to make RVT-101 a pill that's taken once a day.
Pharmaceutical giant GlaxoSmithKline started developing the drug, but couldn't get it off their hands fast enough - it had done poorly on its own in clinical trials. That is, except one, which combined the drug with Aricept. Together, the two together slowed the loss of cognition and ability to perform daily tasks than when Aricept was taken alone.
It seems Ramswamy latched onto that study.
Axovant got the rights to the compound in December 2014, paying $5 million for it. Since then, the company had its initial public offering (IPO) in June. It was the biggest in biotech history at $1.4 billion, according to Forbes.
Axovant is in the business of late-stage drug development, which means they don't research and develop their own drugs from scratch. This shaves years off the costly drug development process.
But it's not out of the woods yet. Just because a study has success up through phase 2b does not mean the FDA is ready to hand over approval. Several doctors have shown support for the drug, however. It's not a huge medical advance, but it is a step in the right direction, they say.
Axovant hopes to file for FDA approval by 2017.