Building a Portfolio of High Impact New Medicines

AMO Pharma is dedicated to making a positive difference for people affected by serious and debilitating diseases, particularly rare or orphan diseases. To do this, we first work to understand the areas of unmet need and the experience of living with any disease we are targeting. From there, we use our extensive experience in drug development to identify and advance therapies that can significantly improve clinical outcomes while offering the strongest opportunities for approval. Our passion for drug development is strengthened and inspired by many personal connections to the rare disease community held by members of our senior team. With drugs designed to reduce disease burden, slow disease progression or reduce many or all symptoms of a disease, our goal is to give patients an opportunity for improved health and quality of life.

Through a network of global contacts in research and drug development, AMO Pharma is able to identify assets that have demonstrated promising safety and efficacy profiles. We can then position therapies for clinical development, targeting diseases where they have the potential to significantly improve treatment outcomes and quality of care. Using this approach, we are currently advancing clinical research programs for therapies to treat neuromuscular and CNS symptoms for rare diseases. As we expand our portfolio with additional high-potential assets in clinical or pre-clinical stage development, we will remain focused on our mission to address areas of critical unmet need, including treatment of many diseases that have been overlooked and underserved.

Leveraging Opportunities to Minimize Development Risk

In our R&D focus and business strategy, AMO Pharma is reshaping the traditional drug development model to be more efficient and successful. By working to identify promising therapies that have been de-risked in previous pre-clinical and clinical research, assets in our pipeline have the potential to advance more rapidly through the development and regulatory review process. In considering any drug development opportunity, we conduct a comprehensive review of available safety data, predictive pre-clinical models and other scientific evidence. We then match that analysis with deep understanding of the mechanisms of disease progression to carefully assess each asset’s chances of success in clinical development and potential to improve health for patients. Our lead product candidate highlights the advantages of this model. Originally positioned for development in a different indication, it is supported by extensive safety data and efficacy in studies based on a genetic mouse model. Our analysis of previous research revealed that this therapy could have potential applications in the treatment of fragile X syndrome, a rare and devastating disease with no approved therapies available for patients.

The Expertise to Advance Promising Therapies for Novel Indications

In many cases, diseases and patients are underserved or neglected due to a lack of precedent in research and the inability to carefully assess results in pre-clinical and early stage clinical research. AMO Pharma applies a range of innovative clinical trial assessment tools and methodologies to unlock the full development potential of drugs that others might overlook.

The identification of therapies with available safety and efficacy data presents opportunities to “jump start” development programs in new indications. We also apply a more modern approach to clinical research that invites stakeholders including researchers, physicians, regulators and patients to engage in an informed collaborative process of drug development.

This approach presents new opportunities to further de-risk and streamline development programs. By understanding the mechanism of disease and disease pathways, and then matching that understanding to therapies that have been widely studied and that offer proven safety and efficacy profiles, we can more clearly establish protocols and end points in clinical research to produce the targeted results needed for regulatory approval.

Partnering To Achieve the Best Results

As we build our pipeline of assets through acquisition and partnering, we will continue to apply the highest standards in our due diligence and work to develop a network of support from key stakeholders to advance our clinical research programs. We have the global reach and the ability to remain highly flexible in all aspects of asset acquisition and product development, including opportunities to build a global sales organization or partner with leading companies and research centers in our efforts to bring new therapies to the patients who need them.