Platform Compounds



Our Pipeline consists of drugs for chronic neurodegeneration - Alzheimer’s disease (AD), its orphan indication Alzheimer’s disease and dementia in Down syndrome (AD-DS) and Parkinson’s disease (PD). Additionally, we have a compound to treat acute neurodegeneration - traumatic brain injury (TBI) and stroke - and a third compound for advanced AD.

ANVS401 is our lead compound. It is being developed for AD, AD-DS and PD, because in preclinical studies it improved axonal transport in these diseases by inhibiting the neurotoxic proteins that kill nerve cells. It was shown in human and animal studies to lower APP/Aβ, tau/phospho-tau and α-synuclein, the neurotoxic proteins that impair axonal transport and lead to inflammation and cell death. The compound was tested in three Phase 1 clinical studies that show it to be well tolerated [Investigator Brochure 2015]. This safety data is applicable to the clinical development of ANVS401 for AD, AD-DS, PD and other chronic neurodegenerative disorders.

ANVS405 is being developed for acute indications, focused on protecting the brain after TBI and/or stroke. ANVS405 is the same compound as ANVS401, but it is given intravenously in cases of acute head and brain trauma. ANVS405 was given to rats as an injectable after TBI to ensure that it would reach the brain quickly. TBI rats that were treated after the insult exhibited enhanced memory and learning and lowered microglia activation, a measure of inflammation. To date the development of ANVS405 has been funded by the US Army and we plan to further apply for grants to continue the development.

ANVS301 is expected to increase cognitive capability in later stages of AD and dementia. In preclinical studies, ANVS301 improved memory and learning in very old rats. ANVS301 is in a Phase 1 clinical trial that is being conducted and financed by the National Institutes of Health (NIH).



Proof of Concept - ANIMALS

We have shown in a number of animal studies that lowering neurotoxic proteins restores homeostasis and fully recovers the affected function: in AD mice ANVS401 restores brain function, memory and learning; in Parkinson’s mice the drug restores gut motility and in TBI rats the drug protects nerve cells, lowers inflammation and restores memory and learning. In humans, in spinal fluid of mildly cognitive impaired patients, ANVS401 reduces levels of neurotoxic proteins and lowers inflammation.

Proof of Concept - HUMANS

In the human POC study, four patients with MCI were treated for 10 days with ANVS401 with the safe dose of 4x60 mg (240 mg/day). Cerebrospinal fluid (CSF) and plasma were drawn over 12 hours on day 0 and day 11. Levels of ANVS-401 and metabolites were measured in plasma and CSF over the 12 hours. ANVS401 pharmacokinetics (PK) in plasma corresponded to what the Company had seen in the previous clinical safety studies: T1/2 = 5 hrs. In CSF, however, ANVS-401 showed a much longer half-life of T1/2 > 12 hours [Maccecchini, 2012].


Neurotoxic proteins measured in the CSF of MCI patients in the POC study described above. ANVS401 given for 10 days did not just lower APP and Tau but reduced their levels close to the average levels measured in healthy volunteers.



ANVS401 is orally available, well behaved and well qualified as a candidate to treat neurodegeneration (AD,AD-DS and PD). Three clinical studies have been conducted with ANVS401. The first was a single ascending dose (SAD) study in 72 healthy volunteers. The second was a multiple ascending dose (MAD) study in 48 healthy volunteers. The third was a proof of concept (POC) study in four mildly cognitive- impaired (MCI) patients.



Phase 2a Study in AD Patients

We are presently conducting a study in early AD patients in conjunction with the Alzheimer's Disease Cooperative Study (ADCS). We are treating patients for 4 weeks with ANVS401 and measure target and pathway engagement in the spinal fluid. This means that we are measuring levels of neurotoxic proteins, neurotransmitters, neurotrophic factors, inflammation and nerve cell death as well of cognitive improvement.



Phase 2a Study in AD and PD Patients

We are conducting a Phase 2a study in early AD and PD patients, in which we are measuring levels of neurotoxic proteins, neurotransmitters, neurotrophic factors, inflammation and nerve cell death as well of cognitive and functional improvement, i.e. the toxic cascade leading to nerve cell death.

Both studies:

  • Are multi-center, randomized, double-blind, placebo-controlled trials

  • Enroll subjects diagnosed with early AD or early PD

  • Measure target and pathway engagement

  • Will provide optimal information to proceed to pivotal studies, in AD, DS and PD with large scale cognitive endpoints

The successful outcome of both studies will validate ANVS401's inhibition of the toxic cascade that leads to neurodegeneration in AD and PD; it will give us some indications of efficacy; and it will allow the company to move into Phase 3 studies in AD, in AD-DS, an orphan indication of AD, and in PD.

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