News Release

Alzheimer's treatment innovation pipeline is building

ResearchersAgainstAlzheimer's releases report detailing 23 drugs in late-stage testing and 57 drugs in mid-stage testing on pace to be on market in the coming years if successful and approved

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A new analysis of the Phase II Alzheimer's drug pipeline, conducted by ResearchersAgainstAlzheimer's (RA2), revealed 57 new Alzheimer's drugs. According to the analysis, nearly twice as many mechanisms of action are being tested in Phase II than in Phase III clinical trials. This diverse pipeline is important as it will give physicians, people with Alzheimer's and their loved ones new ways to combat the disease in the future.

The findings show that as the development of these compounds moves forward and the science behind Alzheimer's drug development continues to advance, policy makers, clinical researchers, drug developers and other Alzheimer's drug partners must build new clinical trial infrastructure and designs that allow for rapid recruitment and testing, consistent high-quality data and prompt data disclosure. Alzheimer's currently has no treatments that stop, slow or prevent the progression of the disease.

"Just as combination treatment proved effective for HIV/AIDS, a similar approach to developing a 'cocktail therapy' for Alzheimer's disease used by several of these compounds may provide the hope and medical progress that millions of people are demanding," said UsAgainstAlzheimer's Co-Founder and Chairman, George Vradenburg. "These potentially game-changing drugs on the horizon may make Alzheimer's a manageable disease. To assure our best shot at success, we must ensure that the necessary investments are being made to build a 21st century infrastructure to test their effectiveness and an innovation-friendly path to market to those in need."

According to the first RA2 pipeline analysis released in March, there were 17 Alzheimer's drugs in Phase III clinical trials on course to launch in the next five years. Since the initial Phase III pipeline report was issued, however, a number of Alzheimer's drug candidates have moved from Phase II to Phase III clinical trials. According to the most recent analysis, there are 23 Alzheimer's drugs in Phase III clinical trials, and ResearchersAgainstAlzheimer's estimate that 19 drugs could reach the market in the next five years.

While promising, many of these drugs will fail due to Alzheimer's complex nature. That is why swift testing and regulatory review of emerging medicines with different therapeutic approaches, such as those in Phase II, will be required to ensure innovative treatments quickly reach patients in need.

The Alzheimer's scientific and commercial field is increasingly turning its focus on disease-modifying prevention drugs, such as those that can be administered to people at risk for disease before Alzheimer's symptoms appear, thereby potentially preventing or delaying the development of dementia symptoms.

This is similar to an approach used in HIV/AIDS to prevent those who are HIV Positive from developing AIDS symptoms. Such drugs represent an approach that is different from so-called symptomatic drugs, which are administered after symptoms appear in order to treat Alzheimer's symptoms, such as agitation, cognitive loss, hallucinations or depression.

According to the analysis, which was reviewed by academic research experts and select RA2 members, a diverse pipeline of both prevention and symptomatic drugs requires changes that include:

  • A standing, high-performance clinical trial infrastructure that allows for rapid testing and thus fast failure or success.
  • Robust biomarkers - or measurable indicators of the presence, severity or progression of a disease - which are able to assess the effectiveness of drugs in populations without any symptoms, thereby potentially preventing the development of symptoms altogether.
  • New endpoints for trials. Many of the currently used clinical trial scales in Alzheimer's trials (e.g., ADAS-Cog and measures of function) will not be effective in people in early stages of the disease, as certain symptoms like cognitive decline may occur late in the disease.
  • Greater understanding of biomarker research, so that researchers and clinicians can better "fit" the right medicine with the right population.
  • Proactive information sharing mechanisms by which clinicians and researchers communicate what treatments work best with certain patient populations.
  • Innovative clinical trial design that increases flexibility for drug developers, including:
    • Adaptive trials or trials that are modified based on patient outcomes.
    • Trials for novel-novel combination treatments, where two new drugs are tested together, including testing a drug with demonstrated target engagement but without efficacy as monotherapy.

"I am encouraged to see such a range of approaches to treating Alzheimer's in Phase II development," said Dr. David Morgan, CEO of the University of South Florida's Health Byrd Alzheimer's Institute. "There is much work that still needs to be done, but the drugs in Phase II clinical trials offer a great deal of hope for the future."

Click here to view the analysis.


UsAgainstAlzheimer's (UsA2) is an innovative non-profit organization demanding - and delivering - a solution to Alzheimer's. Driven by the suffering of millions of families, UsAgainstAlzheimer's presses for greater urgency from government, industry and the scientific community in the quest for an Alzheimer's cure - accomplishing this through effective leadership, collaborative convening and strategic investments.

Founded in 2010, UsAgainstAlzheimer's has worked across sectors to: (1) secure the national goal of preventing and effectively treating Alzheimer's by 2025 and help secure nearly $500 million in additional annual public funding for Alzheimer's research; (2) drive global efforts that resulted in the leaders of the world's most powerful nations, the G7 group, to embrace a similar 2025 goal and to call for greater levels of research investment and collaboration; and (3) forge industry commitments to improve efficiencies for an expedited drug discovery and approval process.

ResearchersAgainstAlzheimer's (RA2) is a global network of more than 400 Alzheimer's researchers in US and abroad established by UsAgainstAlzheimer's to advocate for greater research funding, policy reform and multi-sector collaboration in order to stop Alzheimer's disease. RA2 believes that an effective treatment for Alzheimer's disease is within reach if government, industry and citizens are willing to commit the resources and work together to disrupt business-as-usual to achieve the 2025 goal set by the US and the G7.

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