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Novel Immunotherapeutic Vaccine for the treatment of Alzheimer’s disease

Periodic Reporting for period 1 - IMMOVA (Novel Immunotherapeutic Vaccine for the treatment of Alzheimer’s disease)

Reporting period: 2017-03-01 to 2017-08-31

Alzinova is developing ALZ-101 – a novel therapeutic Alzheimer´s disease (AD) vaccine candidate designed to prevent neurodegeneration in the brain. The product is based on the proprietary AβCC peptide™ technology which allows for the development of a vaccine that specifically targets the endogenous substance that causes AD – a feat made possible by the unprecedented capability to stabilise the antigen. Through the Phase 1 feasibility assessment, we have developed a business plan that will guide the future clinical studies and commercialisation through out-licensing.

AD is generally considered to be one of the areas of medicine with the highest unmet medical need today. It is a progressive deadly mental disorder afflicting elderly individuals in all parts of the world, including high, low- and middle-income countries, and all socioeconomic classes. Being highly debilitating in later stages, its toll on the individual can be devastating, especially for those of poor socioeconomic status who often lack the social networks that is the most common way of providing both emotional and practical help in dealing with the disease.

Treatment of AD with the existing pharmacotherapies provides only a small window of benefit, but patients always progress to a stage at which the extent of their symptoms overshadows the effects of these drugs. Drugs that can target the underlying mechanisms that contribute to further decline will revolutionize the field of AD therapeutics. This is the market gap that Alzinova will address.
The feasibility study has shown that ALZ-101 as a drug candidate against AD demonstrates a strong commercial and clinical viability due to its unique features as the first ever active vaccine specific for Aβ oligomers, which potentially will lead to the first effective disease modifying AD treatment that can be used for a wide range of the global patient population.
In the feasibility assessment, Alzinova has progressed from the primary market data collection and analysis, through the synthesis of the results, to the finalisation of the financial and out-licensing targets aligned with an overall business development strategy of the company. The data collected has provided valuable insights into the current trends and opportunities on the market for AD therapeutics, activities undertaken by the competitors and the recent clinical development results. The feasibility assessment has been performed with the support from the SME coach who provided valuable insights into the scope of the analyses.

The main conclusion from the feasibility study is that there exists a clear business opportunity to exploit on the global AD treatment market. Due to the significant market size, low number of existing treatment options, the limited number of new similar drugs in development, and a clear regulatory pathway for drug approval, the AD market provides a unique opportunity for Alzinova and the vaccine ALZ-101.

The launch of immunotherapies targeting clearance of Aβ will not fill the existing gap on this market and the high unmet needs related to treatment options for AD patients are forecasted to remain underserved. Thus, the AD market will remain a lucrative opportunity for pharmaceutical companies developing novel efficacious treatments. This is a business opportunity that Alzinova aims to explore.
The analysis has confirmed that there are no regulatory hurdles that can hamper the validation or the market launch of the drug upon delivery of sound clinical data. Importantly, ALZ-101 may qualify for preferential regulatory approval schemes upon successful conduct of the Phase 1 clinical trial. This may drastically shorten approval time and accelerate milestone payments for Alzinova.
Mapping the competitive landscape has made it clear that there is an opportunity to position Alzinova as a “truly oligomer-targeting company”, in contrast to virtually all other companies who have either failed completely or still struggle to develop similar therapies. Alzinova’s products are in fact unique, and have been developed using a proprietary technology. In brief, this “AβCC technology” entails covalent restriction of the Aβ peptide with an intramolecular cysteine bond which renders it completely non-fibrillogenic. Hence, it only forms the soluble oligomers. These oligomers are the antigen in the ALZ-101 vaccine. When presented to the immune system (of mice and rabbits) it stimulates the production of endogenous antibodies with extremely high selectivity for the oligomers. It does not recognize monomeric or fibrillar forms of the peptide at all, which has been demonstrated extensively in in vitro studies.

The unprecedented high specificity of ALZ-101 affords a unique opportunity to address the validity of the “oligomer hypothesis” in AD in both preclinical disease models and, eventually, human subjects. This has not been easily addressed previously based on the lack of truly oligomer-specific tools to aid in the discovery of novel therapies.

If proven successful, the project will have strong socio-economic implications. A cost-effective disease-modifying treatment that can be offered to large numbers of the global population will have a major advantage over any passive immunotherapy.
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