New BIO Report: More Progress Needed in Innovation of Alzheimer’s Disease Therapeutics

”The growing health and financial impact of Alzheimer’s disease on American patients, caregivers, and our healthcare system makes the need for new treatments more pressing each day,” said BIO President and CEO Jim Greenwood. “The good news is that America’s biopharmaceutical companies currently are conducting 74 clinical-stage programs intended to stop, prevent, or slow the progression of this looming crisis. The bad news is that, while these programs hold real promise, the R&D challenges with this disease are enormous and the level of investment and innovation is not where our nation needs it to be to address this exploding public health crisis. To win this fight, it is essential that our public policies stimulate more investment in research and innovation.”

The analysis, which aims to assess the depth and breadth of innovation to meet the needs of patients suffering from Alzheimer’s disease, also features historical clinical success rates, failed mechanistic strategies, as well as trends in venture financing and investment into new clinical trials.

Key Takeaways

• Lack of Approved Drugs: There are currently no FDA-approved disease-modifying drugs for Alzheimer’s disease.
• Insufficient Venture Investment: Venture capital funding of U.S. companies with lead programs in Alzheimer’s disease is 16 times below oncology funding ($1.0 billion vs. $16.5 billion over the last decade).
• Poor R&D Success Rates: Clinical development success for disease-modifying drug programs for Alzheimer’s has been difficult in late-stage trials, with no disease-modifying drugs moving beyond Phase III to FDA filing. Since 2008, a total of 87 clinical-stage disease-modifying programs have been suspended.
• Inconsistent Clinical Trial Initiations: Clinical trial starts for disease-modifying Alzheimer’s drugs have ranged from 11-21 trial starts per year since 2008, with no detectable trend. Phase III trial starts have been the least consistent, ranging from 0-5 per year.
• Growing & Diverse R&D Pipeline: There are 74 clinical-stage programs with disease-modifying potential in Alzheimer’s disease. The drug candidates in these programs are attempting to stop, prevent, or slow the progression of Alzheimer’s disease using 10 different strategies involving 30 distinct molecular targets. Small biotech companies account for more than three-fourths of these clinical programs.

“With a 100% failure rate for disease-modifying Alzheimer’s Phase III studies, this has been the most challenging R&D hurdle in our industry. The combination of a looming crisis in Alzheimer’s-associated healthcare costs and no disease-modifying drug on the market indicates a desperate need for higher levels of investment and innovative approaches,” said report author David Thomas, Vice President, Industry Research for BIO. “This report shows the venture investment trend is positive, but still significantly lower than other therapeutic areas such as cancer. The biopharmaceutical industry is working hard to make progress, with the majority of targeted therapeutics entering the clinic being conducted by small companies. A more supportive policy environment will be critical to developing new treatments that could improve the lives of the millions of patients and families suffering from this disease.”

The report and other BIO Industry Analysis reports are available for download here: www.bio.org/iareports.

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