Alzheon IPO To Fund New Phase III Trialling Of Failed Alzheimer's Drug
Executive Summary
Alzheon tells prospective investors it believes beta-amyloid blocker ALZ-801 can succeed in pivotal trials by treating APOE4 subgroup.
Alzheon Inc. believes the reworked active ingredient of its in-licensed compound ALZ-801 can prove a success in treating a subset of Alzheimer's disease patients, and plans a NASDAQ listing to raise the funds for a Phase III clinical trial set to start later this year.
But the preliminary funing target of $80m may not be enough to get the drug approved if FDA isn't content with just one pivotal trial and demands another for confirmation.
"Should our initial Phase III trial of ALZ-801 demonstrate efficacy in treating APOE4/4 homozygous patients, we may need to substantiate that evidence through a second adequate and well-controlled clinical study." - Alzheon Inc.Alzheon's lead asset, ALZ-801, is a reformulated, oral version of tramiprosate and supposedly acts via an anti-aggregation mechanism, thus sharing similarities with other amyloid-targeting therapies.
Alzheon in-licensed the compound from Canada-based Bellus Health Inc. after the original tramiprosate therapy failed a big clinical trial in 2007. Using updated analyses from those trials, Alzheon tweaked the product, producing an improved version of the compound, ALZ-801.
The US biotech now plans to study ALZ-801 in a Phase III trial designed for patients that are most likely to respond to it: Patients with two copies of the APOE4 gene on the assumption they have greater beta amyloid burden and develop Alzheimer's earlier.
"If ALZ-801 is approved, we believe it has the potential to be among the first drugs to intervene in an underlying mechanism of Alzheimer’s disease." - Alzheon Inc.
In its SEC filing, Alzheon said it "identified what we believe are key deficiencies in Bellus’ clinical development program: the suboptimal pharmacokinetic (PK) performance of oral tramiprosate and the misdiagnosis of Alzheimer's disease leading to many patients without Alzheimer's disease pathology being enrolled in the trial."
"We believe that our ALZ-801 development program addresses these deficiencies through the improved PK profile of ALZ-801 and through our focus on APOE4/4 homozygous patients who carry a higher burden of [soluble aggregated forms] of Aß oligomers, and are less likely to be misdiagnosed with Alzheimer's Disease," it said.
The company also voiced confidence that ALZ-801 has the potential to be differentiated from other emerging therapies targeting Alzheimer's disease pathology "due to its novel mechanism of action, oral mode of administration, potential efficacy in a genetically-targeted population and observed favorable safety profile."
Doubts Suspended (For Now)
Tramiprosate's chequered history makes some analysts question the rationale for continuing to develop it is purely based on analyses involving subgroups of subgroups.
Regulators in the US have given Alzheon the benefit of the doubt; last October the FDA designated ALZ-801 as a Fast Track development program for the investigation of Alzheimer's Disease. It has also now given Alzheon the go-ahead to start a new Phase III trial using ALZ-801 for the treatment of APOE4/4 homozygous Alzheimer's Disease patients, the company said.
Alzheon in its SEC filing however flagged the possibility of a further Phase III for ALZ-801 .
"Should our initial Phase III trial of ALZ-801 demonstrate efficacy in treating APOE4/4 homozygous patients, we may need to substantiate that evidence through a second adequate and well-controlled clinical study," Alzheon noted.
But it added that "if ALZ-801 is approved, we believe it has the potential to be among the first drugs to intervene in an underlying mechanism of Alzheimer’s disease."