UsA2 grateful for the immense efforts of researchers and clinical trial participants

WASHINGTON, DC (September 27, 2017) – Following today’s announcement that intepirdine, a drug developed by Axovant Sciences, failed to slow the symptoms of Alzheimer’s in a large trial, UsAgainstAlzheimer’s emphasized that while this news is a setback for the millions of people around the world with Alzheimer’s and dementias, the pipeline could see more successful results in the near future.

“While the news today is not what any of us wanted to hear, we want to offer our heartfelt appreciation to the entire Axovant team, including the tens of thousands of researchers and clinical trials coordinators, in its steadfast commitment, leadership, and unwavering resolve to work to vanquish this complex disease,” said George Vradenburg, UsAgainstAlzheimer’s Co-Founder and Chairman. “We have the utmost gratitude to the clinical trials participants and their families, and we are more focused than ever to sustain this fight. The first person to be cured of Alzheimer’s is someone who will have been in a clinical trial, and we must continue to engage more people to actively participate in clinical research.

“While today’s Alzheimer’s disease drug pipeline has been marred by more than a decade of high failure rates, due to the complex nature of Alzheimer’s, as well as public underinvestment in research, every step in the process – even a setback – brings us closer to a cure. Just as we have before, we will keep working incessantly, passionately, and strategically so that the next round in this fight goes to us.”

Despite today’s results, there are more promising innovations on the horizon. An analysis of the Phase III Alzheimer’s drug pipeline, conducted by ResearchersAgainstAlzheimer’s (RA2), shows that there are now 22 Alzheimer’s drugs in Phase III clinical trials, 18 of which may be on track to launch in the next five years. According to the analysis, a diverse pipeline requires a standing, high-performance clinical trial infrastructure that allows for rapid testing and, thus, fast failure or success.

UsAgainstAlzheimer’s (UsA2) will continue to work zealously for people living with Alzheimer’s and other dementias, and their caregivers, so that a means to prevent, slow and stop Alzheimer’s becomes a reality. UsA2 works collaboratively with industry partners to see near- and long-term progress against this destructive disease.

###

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 advocacy, 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 public funding for Alzheimer’s research over the past few years; (2) drive global efforts that resulted in the leaders of the world’s most powerful nations, the G7, 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. More information can be found at: http://www.usagainstalzheimers.org/.

Contact:

Ranit Schmelzer

202-538-1065

[email protected] 

RA2 Presents Analysis of Late-Stage Alzheimer’s Pipeline at Alzheimer’s Association International Conference in London

LONDON, July 18, 2017 – Twenty-seven Alzheimer’s drugs in Phase III clinical trials and eight drugs in Phase II clinical trials may launch in the next five years, according to a revised Alzheimer’s pipeline analysis presented today at the Alzheimer’s Association International Conference (AAIC) by ResearchersAgainstAlzheimer’s (RA2), an UsAgainstAlzheimer’s network.

“The Alzheimer’s disease pipeline, marred by decades of failures and underinvestment, is due for big victories,” said George Vradenburg, UsAgainstAlzheimer’s Co-Founder and Chairman. “Thanks to growing investment from industry leaders, we remain cautiously optimistic that the current crop of late-stage Alzheimer’s innovations will bring much-needed solutions to families in the near future.”

The pipeline analysis offers projections for drugs currently in Phase II and Phase III clinical trials and identifies critical milestones in drug development, including estimated trial completion, estimated regulatory filing and estimated launch date. The analysis also provides a breakdown of the various mechanisms of action that are being tested to combat the progression of the disease. There are currently 23 drugs in Phase II and III trials targeting amyloid protein buildup in the brain, while 28 drugs are targeting neurotransmitter activity. 

A novel treatment for Alzheimer’s hasn’t received FDA approval since 2003 and regulatory approval in Europe since 2002. However, the RA2 analysis confirms significant momentum by industry to forge ahead with Alzheimer’s disease innovation. According to the analysis, there has been an 18-percent increase in Phase II drugs (49 to 58) and a 7-percent increase in Phase III drugs (30 to 32) in development from 2016 to 2017.

“There is no silver bullet when it comes to treating Alzheimer’s,” said Dr. David Morgan, a founding member of RA2 and a distinguished professor of Molecular Pharmacology and Physiology at the University of South Florida. “The more we learn about the underlying Alzheimer’s pathology, the closer we get to a cure for a disease that is an enormous burden on patients, caregivers and global health systems.”

A recent report by the Centers for Disease Control and Prevention found that Alzheimer’s deaths have increased by 55 percent during a recent 15-year period (1999-2014). The late-stage Alzheimer’s pipeline provides much-needed hope to the 5.5 million Americans with Alzheimer’s and their caregivers who are desperate for a treatment or cure.

According to the analysis, six drugs will conclude Phase III trials in 2017. UsAgainstAlzheimer’s and ResearchersAgainstAlzheimer’s will continue actively tracking the progress of each drug and working across sectors to ensure that clinical trials are efficient and diverse, and that regulatory approaches to approving Alzheimer’s drugs are modernized.

As new, potentially life-changing treatments inch closer to regulatory approval, the analysis, a semi-annual snapshot of the state of the Alzheimer’s drug pipeline, begs the question: is the global health care system prepared to ensure that people with or at risk of Alzheimer’s have access to the latest innovations once they reach the market?

“Alzheimer’s is commonly misdiagnosed, and the United States suffers from a shortage of geriatricians – issues that will only grow as the Baby Boomer generation ages,” Vradenburg said. “Private- and public-sector leaders will need to work closely with insurers in the coming years to ensure patients have access to these drugs when they are available.”   

To view RA2’s AAIC poster presentation and read the full analysis, click here (http://www.usagainstalzheimers.org/sites/default/files/alzheimers-drugs-development-pipeline-2017.pdf)

*While authors of the analysis are affiliated with ResearchersAgainstAlzheimer’s, the viewpoints published in the analysis may not reflect or represent the opinions or positions of all RA2 members.

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UsAgainstAlzheimer's is an innovative, patient-centered non-profit 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 its own patient-centered effective leadership, collaborative advocacy and strategic investments.

ResearchersAgainstAlzheimer’s (RA2) is a global network of more than 450 Alzheimer’s researchers in the United States 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 United States and the G7.

“A cure for Alzheimer’s: a fantasy, a wish, an impossible dream; the same words that were said to Galileo, Edison, Curie, Salk and whoever dreamed up the internet. Yesterday’s dream is today’s reality.” – Trish Vradenburg, Co-Founder and Vice-Chair of UsAgainstAlzheimer’s, who passed away in April 2017. 

WASHINGTON, DC, May 11, 2017 – Leading Alzheimer’s disease researchers and a prominent patient advocate today published an analysis, “Single Endpoint for New Drug Approvals for Alzheimer’s Disease,” urging the Food and Drug Administration (FDA) to clarify and modernize its current approach for approving new treatments for Alzheimer’s disease.

The analysis, authored by George Vradenburg, UsAgainstAlzheimer’s Co-Founder and Chairman, and Drs. Howard Fillit, Dave Morgan, Marwan Sabbagh, Paul Aisen and Richard Mohs, recommends that the FDA approve new medicines that demonstrate a proven benefit on at least one therapeutic endpoint – either cognition or function. The current FDA standards require a new drug to show benefits on both proven endpoints, an unnecessarily challenging hurdle the authors say may be inhibiting investment in new Alzheimer’s treatments.

The authors are members of ResearchersAgainstAlzheimer’s (RA2), an UsAgainstAlzheimer’s global network of more than 450 disease researchers.*

“If the FDA were to state that meaningful efficacy on a single endpoint is sufficient for approval, we believe that it would impact prospective investments in this therapeutic area as well as clinical-trial design,” wrote the authors. “We believe a clarified and modernized FDA approval standard for Alzheimer’s disease would catalyze renewed investment in the discovery and development of new medical advances for Alzheimer’s disease, particularly in early-stage companies and for venture investment.”

The analysis points out that Alzheimer’s disease biopharmaceutical research is lagging well behind that of other diseases, despite the fact that Alzheimer’s diagnoses will triple in future decades and that there is no current means to prevent, treat or cure the disease. The analysis notes that in 2014-2015, there were only 135 ongoing interventional Alzheimer’s disease clinical trials compared to nearly 5,000 similar trials for oncology drugs.

The authors wrote that a modernized FDA standard for Alzheimer’s medications would reflect changes in the field and in treatment since the 1990s, and it would also align with draft guidance issued in 2013 for drug development for early Alzheimer’s. Specifically for Alzheimer’s, the authors argue, if a new drug improves memory but has less positive impact on a patient’s daily functioning skills, the medication should still meet standards for FDA approval, because clinically meaningful improvements in cognition matter importantly to persons with Alzheimer’s independent of functional improvement and vice-versa.

“Irrespective of the degree of impact on secondary measures, the notion that the FDA would deny approval for a safe and well-tolerated drug candidate that achieves its primary endpoint of improving cognition in patients with Alzheimer’s disease is almost unthinkable,” the authors wrote.

The authors emphasized that the new standard is essential at a time in which recent clinical trial failures on Alzheimer’s drugs have adversely affected investment in disease research. The FDA has not approved a novel Alzheimer’s treatment since 2003, and the 5.5 million Americans with Alzheimer’s and their caregivers are desperate for innovation.  

*While authors of the analysis are affiliated with ResearchersAgainstAlzheimer’s, the viewpoints published in the analysis may not reflect or represent the opinions or positions of all RA2 members.

###

UsAgainstAlzheimer's is an innovative, patient-centered non-profit 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 its own patient-centered effective leadership, collaborative advocacy and strategic investments.

ResearchersAgainstAlzheimer’s (RA2) is a global network of more than 450 Alzheimer’s researchers in the United States 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 United States and the G7.

“A cure for Alzheimer’s: a fantasy, a wish, an impossible dream; the same words that were said to Galileo, Edison, Curie, Salk and whoever dreamed up the internet. Yesterday’s dream is today’s reality.” – Trish Vradenburg, Co-Founder and Vice-Chair of UsAgainstAlzheimer’s, who passed away in April 2017. 

Contact: Tim Tassa

Phone: 202-263-2580

Email: [email protected]

UsAgainstAlzheimer’s grateful for the immense efforts of researchers and clinical trial participants

WASHINGTON, DC, February 17, 2017 – The recent announcement that Merck is halting a Phase II/III study for a promising Alzheimer’s treatment (verubecestat) was another setback in the continued and intensive effort to deliver an innovative therapy by 2020. UsAgainstAlzheimer’s, which works collaboratively with industry partners to see near- and long-term progress against this destructive disease, applauds the vast investment of Merck, the diligence of the many principal investigators involved in the study and the courage of the clinical trial participants, as well as their caregivers, for their deep commitment to the fight.

“The spirited work of Merck and other drug companies that are concentrating vast resources into a cure for Alzheimer’s is not underappreciated. Each outcome, while assuredly disheartening, builds our knowledge and leads us closer to a solution for the millions of people with Alzheimer’s or related dementias,” said UsAgainstAlzheimer’s Co-Founder and Chairman George Vradenburg. “Just as we have before, we will keep working incessantly, passionately and strategically so that the next round in this fight goes to us.”

Similar to Eli Lilly’s recent negative Phase III trial of the once-promising therapy solanezumab, verubecestat was assessed by an oversight committee as having “virtually no chance of finding a positive clinical effect” on people with mild to moderate symptoms. Verubecestat, like other drugs in the pipeline, targets the beta amyloid protein that forms most of the plaque that builds up in the brains of Alzheimer’s patient.

“The drug will continue to be studied on people with a very early form of Alzheimer’s disease where we are hopeful for a better outcome,” Vradenburg said. 

Following the solanezumab news, Vradenburg issued a letter, acknowledging lessons learned in the aftermath of negative trials and providing a blueprint for moving forward on the path to a cure. His guidance, directed at policymakers, advocates and researchers, rings true following the verubecestat news. It includes these pivotal next steps:

• Obtaining a minimum of $2 billion in annual U.S. federal funding for Alzheimer’s research, with an insistence that, in the near future, every government in the world provide funding equivalent to one percent of their Alzheimer’s care costs (which, in the United States  would equal $2.3 billion, according to 2016 figures).
• Calling upon President Trump to exercise global leadership in this effort.
• Building a high-speed specialized Alzheimer’s trial network to enable the more rapid and efficient testing of therapies.
• Utilizing “big data” to enable doctors to detect Alzheimer’s disease at the earliest possible moment when prevention therapies now in testing could have their greatest effect.
• Achieving greater racial, income and educational diversity among clinical trial participants, realizing that, by 2030, a majority of Americans with Alzheimer’s are expected to be members of what are today called “minority” populations.

Despite the recent news, there is hope, as there are more promising innovations on the horizon. An analysis of the Phase III Alzheimer’s drug pipeline, conducted by ResearchersAgainstAlzheimer’s (RA2), shows that there are now 21 Alzheimer’s drugs in Phase III clinical trials, 19 of which may be on track to launch in the next five years. In addition, RA2 reports that 10 drugs are scheduled to have trial completion dates in 2017 and 2018.  

“UsAgainstAlzheimer’s was founded because we wanted to turn the pain we experienced with this disease to drive our shared purpose to boldly break down the barriers to a cure,” Vradenburg said. “We are grateful to the researchers and clinical trial participants, as well as the enormous financial contributions of Merck and other industry leaders, who have a similar passion to eradicate this disease.”     

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UsAgainstAlzheimer's 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 advocacy, 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 public funding for Alzheimer’s research over the past few years; (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.

Contact: Tim Tassa

Phone: 202-263-2580

Email: [email protected]

UsAgainstAlzheimer’s thanks Eli Lilly for robust effort; forges ahead in hopeful fight against AD

WASHINGTON, DC, November 23, 2016 —Following today’s announcement that Eli Lilly’s drug that would slow memory loss in people with mild cases of Alzheimer’s disease failed Phase III clinical trials, UsAgainstAlzheimer’s emphasizes that while this news is a setback for the millions of people around the world with dementia, the pipeline could see more successful results in the near future.

In light of the urgency surrounding the critical need to deliver a treatment for a disease that has no cure and is the third leading cause of death in the United States, UsAgainstAlzheimer’s is escalating its calls to action to increase research funding for Alzheimer’s disease and dementias and develop a stronger and faster clinical trial infrastructure.

“While the news today is not what any of us wanted to hear, we want to offer our heartfelt appreciation to the entire Lilly team, including the tens of thousands researchers and clinical trials coordinators, in its steadfast commitment, leadership and unwavering resolve to work to vanquish this complex disease,” said George Vradenburg, UsAgainstAlzheimer’s co-founder and chairman. “We have the utmost gratitude to the 4,000 clinical trials participants and their families, and we are more focused than ever to sustain this fight. The first person to be cured of Alzheimer’s is someone who will have been in a clinical trial, and we must continue to engage more people to actively participate in clinical research. Today we grieve; tomorrow we renew the fight.”

While today’s Alzheimer’s disease drug pipeline has been marred by more than a decade of high failure rates, due to the complex nature of Alzheimer’s, as well as public underinvestment in research, every step in the process – even a setback – brings us closer to a cure.

“Holidays are the hardest for millions of Alzheimer’s families as we try to make memories we know won’t last the moment. But the faith we placed in the researchers and global Alzheimer’s disease team at Lilly remains unabated, despite the outcome of the EXPEDITION3 clinical trial. We have watched them work tirelessly with great integrity, tenacity and transparency,” said Meryl Comer, UsAgainstAlzheimer’s board member, author of “Slow Dancing With A Stranger: Lost and Found in the Age of Alzheimer’s” and President of the Geoffrey Beene Foundation Alzheimer’s Initiative. “Winning the battle against Alzheimer’s is bigger than any one company and asks all of us to share our health data and participate in clinical research.”

Despite today’s results, there is hope, as there are more promising innovations on the horizon. An analysis of the Phase III Alzheimer’s drug pipeline, conducted by ResearchersAgainstAlzheimer’s (RA2), shows that there are now 22 Alzheimer’s drugs in Phase III clinical trials, 18 of which may be on track to launch in the next five years. According to the analysis, a diverse pipeline requires a standing, high-performance clinical trial infrastructure that allows for rapid testing and, thus, fast failure or success.

UsAgainstAlzheimer’s will continue to work zealously for people living with Alzheimer’s and other dementias, and their caregivers, so that a means to prevent, slow and stop Alzheimer’s becomes a reality.

###

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 advocacy, 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 public funding for Alzheimer’s research over the past few years; (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.

CONTACT:
Tim Tassa
202.263.2580
[email protected]

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

WASHINGTON, DC, September 28, 2016—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.”

For more information, the pipeline analysis will be discussed in depth during a session called “Where are We on the Path to 2025” during the 2016 UsAgainstAlzheimer’s National Alzheimer’s Summit on September 28 at the Ronald Reagan Building and International Trade Center, in Washington, DC (register for the 2018 Summit here). The session will feature the following panelists:

• Moderator: Drew Holzapfel, Executive Director, The Global CEO Initiative on Alzheimer’s Disease
• Phyllis Barkman Ferrell, Vice President and GBD Leader Global Alzheimer's Disease Platform team, Eli Lilly and Company
• Alan Gilbert, Director, Global Government and NGO Strategy, GE healthymagination
• Louis Jacques, MD, Chief Clinical Officer, Senior Vice President, ADVI
• David Morgan, PhD, Chief Executive Officer/Executive Director, USF Health Byrd Alzheimer Institute

Reporters interested in attending the session should contact [email protected].

Click here to view the analysis.

FOR IMMEDIATE RELEASE
Contact: Tim Tassa
Phone: 202-263-2580
Email: [email protected]

Health Care System Is Not Prepared to Ensure Patients Will Get Access to First Novel Alzheimer’s Therapies Since 2003 

WASHINGTON, March 31, 2016—Today’s Alzheimer’s disease drug pipeline, marred by more than a decade of high failure rates and public underinvestment, is offering near-term promise with 17 drugs on pace to launch in the next five years, according to a recent analysis conducted by ResearchersAgainstAlzheimer’s (RA2), an UsAgainstAlzheimer’s (UsA2) network. If only some of those drugs are successful in late-stage trials and cleared by the U.S. Food and Drug Administration (FDA), the potential innovations will be a much-needed stimulus to the goal of stopping America’s most costly disease, one that affects approximately 5.3 million Americans, a number that is expected to rise precipitously during the next few decades.

Findings from the analysis underscore an important question: once the therapies are approved, will those affected by the disease be able to access these drugs?

“Despite the recent history of disappointment for Alzheimer’s disease advancements, we are cautiously optimistic about the progress of this next wave of innovation because we are seeing well-funded trials and concerted efforts to learn from past failures,” said Dr. David Morgan, a founding member of RA2, a distinguished professor of the College of Medicine and Molecular Pharmacology and Physiology at the University of South Florida, and CEO of the Byrd Alzheimer's Institute.

“The analysis confirms that while there is a promising pipeline for Alzheimer’s patients, we must, in parallel, be encouraging more physicians to accurately diagnose and treat this disease, and work with insurers to enable access for patients to these new drugs when they are available.” 

According to the RA2 analysis, there are currently 17 Alzheimer’s drugs in Phase 3 clinical trials on course to launch in the next five years. This first-of-its-kind analysis reached its conclusions following extensive examination of SEC filings, company reports, presentations at medical conferences, media releases and interviews with company executives about publicly available information on drugs in late-stage testing. In addition, academic research experts and select RA2 members provided input and review of the analysis. 

In the United States, Alzheimer’s, the most common form of dementia, is the third leading cause of death, according to recent studies. Among the top 10 causes of death, it’s the only one that currently can’t be prevented, stopped or slowed. The RA2 analysis’ findings provide the hope that is sorely needed for Alzheimer’s patients and their numerous caregivers who have not seen a novel Alzheimer’s therapy since 2003, when Namenda was approved by the FDA.

“Alzheimer’s disease must be attacked at the scope and scale equal to the challenge. Alzheimer’s is a cancer-size problem requiring a cancer-size solution,” said George Vradenburg, co-founder and chairman of UsAgainstAlzheimer’s. “Our mission is to stop Alzheimer’s by 2020. Our work has focused on disrupting business as usual – increasing research resources, speeding drug development and assuring access of innovative medicines to those with or at risk of the disease. Should drugs in late-stage development prove successful, insurers and physicians will need to step up their game.”

While the analysis takes a positive approach to the innovation pipeline, it also delineates areas that America’s health care system needs to improve upon before new Alzheimer’s treatments are approved.

These areas include:

1. Enhancing the training and numbers of front-line physicians, including in primary care, to diagnose and treat Alzheimer’s. In addition, the number of geriatricians needs to increase from 7,000 (current) to 30,000 by 2030. Unfortunately, fewer medical students are choosing geriatrics because geriatrics practice is not sustainable financially with a 100 percent Medicare payer mix. Neurologists and psychiatrists typically have a mix of payers.
2. Improving the accuracy of diagnosis. In some settings, up to half of all cases of Alzheimer’s disease are misdiagnosed, and there is currently a significant under-diagnosis of the disease in populations around the world. The lack of an Alzheimer’s diagnosis can also negatively impact the ability of patients to adhere to other chronic care protocols. Visits to memory care specialists in neurology, psychiatry or geriatrics is encouraged.
3. Improving communication between patients and physicians. Just 45 percent of people diagnosed with Alzheimer’s were actually informed of their diagnosis by a doctor, compared to 90 percent of people with cancer and cardiovascular disease who are informed of that diagnosis by their doctor.
4. Treating Alzheimer’s like the fatal disease that it is, rather than as routine clinical care. The average wait time to see a neurologist was 35 days, a 25 percent increase since 2010. In contrast, a study shows that the average wait time for a cardiologist is 16.8 days, about 50 percent less than the wait time to see a neurologist.
5. Addressing payment and reimbursement. There is no assurance that the Centers for Medicare and Medicaid Services (CMS) and other national health payment authorities and insurance providers will pay for or reimburse for Alzheimer’s treatments.

“The studies of the past were a necessary predicate for the future of Alzheimer’s medicine,” said Dr. Rachelle Doody, professor of neurology at Baylor College of Medicine and reviewer of the analysis. “We have learned a great deal about trials methodologies and disease targets from past studies. While the analysis shows a promising future, it is also a sobering wakeup call reminding us of the importance of health system readiness to prevent and combat this crushing disease.”

For more information, the pipeline analysis will be discussed in depth during a future Alzheimer’s Talks Teleconference on April 11 from 4-5 pm (EST). The discussion, which will be live streamed, will include remarks from Vradenburg, Morgan and study lead and RA2 director Drew Holzapfel. Visit the Alzheimer’s Talks page for more information.   

To view the analysis, visit: RA2 Pipeline Report

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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 advocacy, 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 public funding for Alzheimer’s research over the past few years; (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 network of more than 400 Alzheimer’s researchers established by UsAgainstAlzheimer’s to advocate for federal research funding and policy reform 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 institute the policy changes that are necessary.