{"id":5077,"date":"2016-12-14T09:38:36","date_gmt":"2016-12-14T09:38:36","guid":{"rendered":"https:\/\/clarivate.com\/?p=5077"},"modified":"2016-12-14T09:38:36","modified_gmt":"2016-12-14T09:38:36","slug":"advances-in-alzheimers-but-knowledge-gaps-remain","status":"publish","type":"post","link":"https:\/\/clarivate.com\/life-sciences-healthcare\/blog\/advances-in-alzheimers-but-knowledge-gaps-remain\/","title":{"rendered":"Advances in Alzheimer\u2019s, but Knowledge Gaps Remain"},"content":{"rendered":"

The Regulatory Affairs Professionals Society (RAPS) held its annual conference, the RAPS Regulatory Convergence, from September 17-20 in San Jose, CA. Clarivate Analytics Medical\/Regulatory Writer Meg Egan Auderset attended; she wrote the following is based on a session about Alzheimer\u2019s disease, Delivering the Next Generation of Alzheimer\u2019s Treatments: Are We Prepared?<\/strong><\/em><\/p>\n

At a session on the \u201cnext generation\u201d of treatments for Alzheimer\u2019s disease, Timothy Franson, MD, chief medical officer of the consulting firm YourEncore, bemoaned the existence of \u201czombies\u201d in the field: practices and approaches to Alzheimer\u2019s drug development that should be \u201cdead\u201d but that persist, draining energy and resources.<\/p>\n

\u201cWhat we have here is not the walking dead, but the walking debt,\u201d Franson quipped. The reason for it, he said, is lack of understanding about the causes of Alzheimer\u2019s and the factors that impact its progression\u2014and about the best way to move the field forward. Innovation will ensure the future of the field, Franson said.<\/p>\n

Alzheimer\u2019s disease is an \u201cepidemic,\u201d according to session moderator George Vradenburg, chairman of UsAgainstAlzheimer\u2019s and co-convener of the Global CEO Initiative on Alzheimer\u2019s Disease. Worldwide, nearly 50 million people live with dementia, and that number is expected to triple by 2050, Vradenburg said. Within the US, approximately 5.4 million people have the disease, according to the Alzheimer\u2019s Association.<\/p>\n

The Alzheimer\u2019s field is \u201cdesperately ready\u201d for new innovation, \u201cbut I don\u2019t think we\u2019re prepared,\u201d said Carlos Garner, PhD, senior director for Global Regulatory Affairs at Eli Lilly and Company. First, the field must fill in the knowledge gaps, he said. European colleague Enrica Alteri, head of the Human Medicines R&D Support Division at the European Medicines Agency, agreed. While basic information on the cause of Alzheimer\u2019s and its pathways is evolving, it remains incomplete, she said. Consequently, drug companies have developed potential treatments based on assumptions<\/em> about the disease; some of those assumptions have been false, resulting in failed therapeutic efforts. The field has also not yet identified reliable biomarkers, she said.<\/p>\n

Both speakers expressed hope, however. Garner anticipates \u201cprofound\u201d scientific, medical, and regulatory advances in the next few years. Regulatory advances will be increasingly important as the field fills the holes in scientific understanding. To date, many drug developers have been unable to take advantage of regulatory tools like breakthrough therapy designation, accelerated approval, and priority review, because the science has not been strong enough to support their use. \u201cWe need to be ready,\u201d Garner said.<\/p>\n

According to Garner, more than a dozen new disease-modifying drugs \u201ccould\u201d become available to patients over the next 5 years. Vradenburg referred to 57 compounds currently in phase 2 development; approximately 40% of them have multiple mechanisms of action, according to materials he shared from UsAgainstAlzheimer\u2019s. Of the phase 2 drugs, 22 are either repurposed drugs or nutritional supplements. (Note: in November 2016, Eli Lilly and Company announced that it would no longer \u201cpursue regulatory submissions\u201d for its phase 3 drug, solanezumab, after patients treated with the compound failed to show statistically significant slowing in cognitive decline when compared to placebo. Solanezumab was being developed to treat mild dementia due to Alzheimer\u2019s.)<\/p>\n

 <\/p>\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n\n
Drugs in Phase 3 Development for Alzheimer\u2019s
\nAnticipated to Launch by the End of 2021<\/strong><\/td>\n<\/tr>\n
Compound<\/strong><\/td>\nSponsor<\/strong><\/td>\n<\/tr>\n
aducanumab<\/td>\nBiogen<\/td>\n<\/tr>\n
ALZ-801<\/td>\nAlzheon, Inc<\/td>\n<\/tr>\n
ALZT-OP1<\/td>\nAZTherapies, Inc<\/td>\n<\/tr>\n
aripiprazole<\/td>\nOtsuka Pharmaceutical Co, Ltd<\/td>\n<\/tr>\n
AVP-786<\/td>\nAvanir Pharmaceuticals, Inc<\/td>\n<\/tr>\n
azeliragon<\/td>\nvTv Therapeutics<\/td>\n<\/tr>\n
brexpiprazole<\/td>\nOtsuka Pharmaceutical Co, Ltd; H Lundbeck A\/S<\/td>\n<\/tr>\n
gantenerumab<\/td>\nF Hoffmann-La Roche Ltd<\/td>\n<\/tr>\n
idalopirdine<\/td>\nOtsuka Pharmaceutical Co, Ltd; H Lundbeck A\/S<\/td>\n<\/tr>\n
intepirdine<\/td>\nAxovant Sciences Ltd<\/td>\n<\/tr>\n
masitinib<\/td>\nAB Sciences<\/td>\n<\/tr>\n
nilvadipine<\/td>\nAstellas Pharma Inc; Archer Pharmaceuticals, Inc<\/td>\n<\/tr>\n
pioglitazone<\/td>\nTakeda Pharmaceuticals USA; Zinfandel Pharmaceuticals Inc<\/td>\n<\/tr>\n
sodium oligo-mannurarate<\/td>\nShanghai Greenvalley Pharmaceutical Co, Ltd<\/td>\n<\/tr>\n
TRx0237<\/td>\nTauRx Therapeutics<\/td>\n<\/tr>\n
verubecestat<\/td>\nMerck & Co, Inc<\/td>\n<\/tr>\n
Source: UsAgainstAlzheimer\u2019s<\/em><\/td>\n<\/tr>\n<\/tbody>\n<\/table>\n

 <\/p>\n

Session speakers repeatedly called for increased collaboration. Alteri advocated for new mechanisms to foster data sharing among drug developers, for example, and cited a need to share information on surrogate and intermediate clinical endpoints (ICEs). Drug developers should also exchange information on biomarkers, she said, particularly to identify pre-symptomatic patients and those whose disease is likely to progress, and to predict likely clinical outcomes of interventions.<\/p>\n

Noting his frustration over the current number of \u201cdisparate efforts,\u201d Franson called for collaboration across all stakeholders. While it is important to share best practices, it is even more important to share information about failures, he said. Franson also advised redefining the benefit-risk balance in Alzheimer\u2019s disease, suggesting that caregivers should have a voice in such discussions.<\/p>\n

Garner stressed the importance of involving patients and advised finding new ways to engage them. In particular, researchers must keep in touch with potential study subjects, even when current trials are not suitable for them, he said. Researchers must learn to maintain patient motivation, and that requires active engagement.<\/p>\n

Alteri mentioned efforts by the FDA and EMA to align their respective guidelines on drug development in Alzheimer\u2019s. She reported \u201cvery good\u201d FDA-EMA agreement on underlying concepts, as well as plans to compare revised and finalized documents currently in development.<\/p>\n

To read the original report, please click here<\/a><\/strong><\/p>\n","protected":false},"excerpt":{"rendered":"

The Regulatory Affairs Professionals Society (RAPS) held its annual conference, the RAPS Regulatory Convergence, from September 17-20 in San Jose, CA. Clarivate Analytics Medical\/Regulatory Writer Meg Egan Auderset attended; she wrote the following is based on a session about Alzheimer\u2019s disease, Delivering the Next Generation of Alzheimer\u2019s Treatments: Are We Prepared?<\/p>\n","protected":false},"author":2,"featured_media":4513,"comment_status":"open","ping_status":"open","sticky":false,"template":"","format":"standard","meta":{"_acf_changed":false,"_links_to":"","_links_to_type":0,"footnotes":""},"categories":[19,21],"tags":[34,26],"class_list":["post-5077","post","type-post","status-publish","format-standard","has-post-thumbnail","hentry","category-biopharma","category-life-sciences-healthcare","tag-clinical-trials","tag-regulatory-intelligence","clarivate-product-biopharma"],"acf":[],"lang":"en","translations":{"en":5077},"publishpress_future_workflow_manual_trigger":{"enabledWorkflows":[]},"pll_sync_post":[],"_links":{"self":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/posts\/5077","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/comments?post=5077"}],"version-history":[{"count":0,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/posts\/5077\/revisions"}],"wp:featuredmedia":[{"embeddable":true,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media\/4513"}],"wp:attachment":[{"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/media?parent=5077"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/categories?post=5077"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/clarivate.com\/life-sciences-healthcare\/wp-json\/wp\/v2\/tags?post=5077"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}