Alzheon’s Alzheimer’s asset boards FDA fast track ahead of second swing at phase 3

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The FDA status follows Alzheon’s work to revitalize the once-failed drug.

Alzheon’s Alzheimer’s asset ALZ-801 has secured fast-track status from the FDA. The regulatory status sets the prodrug of tramiprosate up to speed through the FDA review process if it bucks the trend and hits the mark in phase 3. 

Framingham, Massachusetts-based Alzheon has secured the designation for a drug it rescued from the big scrap heap of failed Alzheimer’s drugs a few years ago. The drug, tramiprosate, is one of many that tried to reduce amyloid aggregation but came up short in the clinic.

Alzheon saw promise behind the headline failure, though. That led to the creation of a prodrug formulation and work to position the candidate for use in a genetic subgroup of patients. Alzheon published a paper detailing part of that effort this week.    

“We have built a strong body of original research and clinical analyses that support the initial evaluation of ALZ-801 in the genetically defined high-risk population of APOE4/4 homozygotes, enabling us to ... move toward an upcoming pivotal study and potential approval as quickly as possible,” Alzheon CEO Martin Tolar, M.D., Ph.D., said in a statement.

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That positive statement about the pace of ALZ-801’s advance clashes with Alzheon’s previous predictions about its progress. It is now 15 months since Alzheon said it had all the data it needed to move into phase 3. Back then, the goal was to start the phase 3 program in early 2017. Today, ALZ-801 is still listed as “phase 3 ready.”

The holdup may relate to funding. Alzheon put together a $10 million series A in 2015 and quietly sold $15.8 million worth of stock in June. The biotech has talked up the potential for its precision medicine approach to Alzheimer’s cut down the number of patients—and therefore cost—needed for phase 3. But the final tab for its program will still be a multiple of the amount it raised in June.

Tolar sounded bullish about financing when Alzheon unveiled the data package intended to usher ALZ-801 into phase 3 in June 2016. Back then, an IPO, big private round or partnership were all on the table. 

Today, with IPO window wide open, Alzheon remains on the sidelines.