{"id":36487,"date":"2026-07-15T12:30:00","date_gmt":"2026-07-15T10:30:00","guid":{"rendered":"http:\/\/stocks-future.com\/?guid=d9aab6d4af72c2a2a86576c7e2ea617d"},"modified":"2026-07-15T12:30:00","modified_gmt":"2026-07-15T10:30:00","slug":"alnylam-highlights-progress-with-neuroscience-programs-at-aaic-2026-showcasing-potential-of-rnai-in-neurological-disease","status":"publish","type":"post","link":"https:\/\/stocks-future.com\/?p=36487","title":{"rendered":"Alnylam Highlights Progress with Neuroscience Programs at AAIC 2026, Showcasing Potential of RNAi in Neurological Disease"},"content":{"rendered":"

\n\u2212 Initiates Global Phase 2 APPlauDS Study of Mivelsiran in Down Syndrome\u2013Associated Alzheimer\u2019s Disease (DS-AD) \u2212<\/i><\/p>

\n\u2212 Announces Completion of Enrollment in the Phase 2 cAPPricorn-1 Study of Mivelsiran in Cerebral Amyloid Angiopathy (CAA) \u2212<\/i><\/p>

\n\u2212 Presents Updated Phase 1 Data on Mivelsiran in Early Onset Alzheimer\u2019s Disease (EOAD) Showing Low Incidence of Amyloid-Related Imaging Abnormalities (ARIA) \u2212<\/i><\/p>

\n\u2212 Shares Preclinical Data and Design of Ongoing Phase 1 Study of Tau-Targeting ALN\u20115288 in Alzheimer\u2019s Patients \u2013<\/i><\/p>

CAMBRIDGE, Mass.--(BUSINESS WIRE)--Alnylam Pharmaceuticals, Inc.<\/a> (Nasdaq: ALNY), <\/b>the leading RNAi therapeutics company, today shared advances across its growing neuroscience portfolio at the Alzheimer\u2019s Association International Conference (AAIC) 2026. This scientific progress underscores the potential of RNAi therapeutics to address the needs of patients with debilitating neurological diseases.<\/p><\/a><\/a>

\nMivelsiran: An investigational RNAi therapeutic targeting amyloid precursor protein (APP) in development for Cerebral Amyloid Angiopathy (CAA) and Alzheimer\u2019s disease (AD)<\/i><\/p>

\nThe company announced the initiation of a Phase 2 study of mivelsiran in people with Down syndrome-associated AD and presented a poster detailing the design and rationale. The APPlauDS Study (evaluating APP-lowering to reduce amyloid accumulation in Down syndrome) will be recruiting people with Down syndrome with early-stage AD across approximately 30 global sites.<\/p>

\n\u201cPeople with Down syndrome have a genetically determined form of Alzheimer\u2019s disease that is now the leading cause of death among adults over age 35 years,\u201d said Michael S. Rafii, M.D., Ph.D., Professor of Neurology at the Keck School of Medicine of USC and presenting author. \u201cAPPlauDS is the first clinical trial to evaluate whether an RNAi approach can reduce APP overexpression caused by trisomy 21, where the APP gene is located. By lowering APP expression early in the disease process, this approach has the potential to slow or prevent the progression of Alzheimer\u2019s disease in people with Down syndrome.\u201d<\/p>

\nAn additional presentation included updated data from the Phase 1 trial of mivelsiran in patients with early-onset Alzheimer\u2019s disease. An analysis of safety data from single- and multiple-doses of mivelsiran showed no evidence of increased risk of amyloid-related imaging abnormality (ARIA) events. Results also showed robust, durable reductions in cerebrospinal fluid (CSF) soluble amyloid beta precursor protein (sAPP\u03b2) and amyloid beta 42 (A\u03b242), with up to 30 months of treatment exposure. The mean maximum reductions in the highest dose group were -89.9% and -70.2% change from baseline for sAPP\u03b2 and A\u03b242, respectively. The most common adverse events (AEs) were procedural pain and procedural headache, and no serious or severe AEs were deemed related to study drug. CSF safety labs showed no significant elevations of CSF total protein or white blood cells.<\/p>

\nAlnylam also announced today that it has completed enrollment in the Phase 2 cAPPricorn-1 study of mivelsiran in CAA, a leading cause of hemorrhagic stroke. CAA is defined by progressive deposition of A\u03b2 in blood vessels of the brain. Reducing APP production reduces downstream A\u03b2 accumulation and could potentially slow CAA progression and CAA-related bleeds in the brain. cAPPricorn-1\u2019s primary endpoint is rate of new lobar microbleeds measured by magnetic resonance imaging (MRI) with initial results expected in 2028.<\/p>

\nALN-5288: An investigational RNAi therapeutic targeting Microtubule-Associated Protein Tau (MAPT) in development for Alzheimer\u2019s disease and tauopathies<\/i><\/p>

\nAlnylam also highlighted progress with ALN-5288 \u2013 an investigational asset in development in collaboration with Regeneron Pharmaceuticals \u2013 in presentations of preclinical data and of the design of the ongoing first-in-human Phase 1 trial (NCT07214727), which initiated in the fourth quarter of 2025.<\/p>

\nWith mivelsiran and ALN-5288, Alnylam has two RNAi therapeutics targeting AD in clinical development, and, together with its collaboration partner Regeneron, a total of seven clinical programs<\/a> for neuroscience indications.<\/p>

\n\u201cThe updates we\u2019re sharing at AAIC 2026 mark an important step forward for Alnylam as we continue to build our leadership in neuroscience,\u201d said Toby Ferguson, M.D., Ph.D., Senior Vice President and Head of the Neuroscience Therapeutic Area at Alnylam. \u201cOur RNAi platform is demonstrating strong potential across neurological diseases, with a favorable safety profile and the possibility to reach broad patient populations. By targeting disease-driving proteins like amyloid and tau at their genetic source, we aim to deliver the kind of transformative, disease-modifying therapies patients and families have long awaited.\u201d<\/p>

\nTo view Alnylam\u2019s AAIC 2026 presentations please visit Capella<\/a>.<\/p>

\nAbout RNAi\n<\/b>RNAi (RNA interference) is a natural cellular process of gene silencing that represents one of the most promising and rapidly advancing frontiers in biology and drug development today. Its discovery has been heralded as \u201ca major scientific breakthrough that happens once every decade or so\u201d and was recognized with the award of the 2006 Nobel Prize for Physiology or Medicine. By harnessing the natural biological process of RNAi occurring in our cells, a new class of medicines known as RNAi therapeutics is now a reality. Small interfering RNA (siRNA), the molecules that mediate RNAi and comprise Alnylam\u2019s RNAi therapeutic platform, function upstream of today\u2019s medicines by potently silencing messenger RNA (mRNA) \u2013 the genetic precursors \u2013 that encode for disease-causing or disease pathway proteins, thus preventing them from being made. This is a revolutionary approach with the potential to transform the care of patients with genetic and other diseases.<\/p>

\nAbout Alzheimer\u2019s Disease\n<\/b>Alzheimer\u2019s disease (AD) is the most common neurodegenerative disease and the most common form of dementia, affecting over 30 million people worldwide. Amyloid and tau are widely viewed as the two principal biological drivers of AD, together contributing to the cascade of neurodegeneration and cognitive decline. Disease progression results in progressive loss of independence, increased caregiver burden, institutionalization and premature death. Early-onset Alzheimer\u2019s disease (EOAD), the leading cause of dementia in younger individuals, refers to a subgroup of AD with symptom onset prior to the age of 65, representing approximately 4% to 6% of all AD. People with Down syndrome (DS) have a near-complete risk of developing Alzheimer\u2019s disease (AD) in late adulthood; AD has become the leading cause of death in adults with DS. DS-AD is caused by overexpression of amyloid precursor protein (APP) due to an additional copy of chromosome 21 (Trisomy 21), the locus of the APP gene.<\/p>

\nAbout Cerebral Amyloid Angiopathy\n<\/b>Cerebral amyloid angiopathy (CAA) is the second most-common cause of hemorrhagic stroke. CAA is defined by progressive deposition of amyloid beta (A\u03b2) into the walls of small arteries, arterioles and capillaries in the brain, causing impaired vascular reactivity, focal tissue damage and increased risk for intracerebral hemorrhage. CAA has also been shown to be an independent contributor to cognitive impairment. There are currently no available treatment options for CAA.<\/p>

\nAbout Alnylam Pharmaceuticals\n<\/b>Alnylam (Nasdaq: ALNY) is a leading global biopharmaceutical company and the pioneer of the RNA interference (RNAi) revolution. The Company is focused on developing transformative therapies with the potential to prevent, halt or reverse disease. For more than two decades, Alnylam has advanced the Nobel-Prize-winning science of RNAi, delivering critical breakthroughs and six approved medicines. Alnylam has medicines available in more than 70 countries and a rapidly expanding and robust pipeline, in addition to consistently being recognized as an exceptional workplace and socially responsible organization. The Company is executing on its Alnylam 2030<\/i> strategy to accelerate innovation and scale impact to transform human health.<\/p>

\nAlnylam Forward-Looking Statements<\/b><\/p>

\nThis press release contains forward-looking statements within the meaning of Section 27A of the Securities Act of 1933 and Section 21E of the Securities Exchange Act of 1934. All statements other than historical statements of fact regarding Alnylam\u2019s expectations, beliefs, goals, plans or prospects, including, without limitation, statements regarding the potential of RNAi therapeutics to address the needs of patients with neurological diseases; the potential efficacy or safety of any of Alnylam\u2019s product candidates; the potential for mivelsiran to slow or prevent the progression of Alzheimer\u2019s disease in people with Down syndrome; the timing of the publication of results from any of Alnylam\u2019s clinical trials; the potential for Alnylam\u2019s RNAi platform to treat neurological diseases with a favorable safety profile and the possibility to reach broad patient populations; Alnylam\u2019s ability to deliver transformative, disease-modifying therapies patients and families have long awaited; and Alnylam\u2019s ability to execute on its Alnylam 2030<\/i> strategy to accelerate innovation and scale to transform human health, should be considered forward-looking statements. Actual results and future plans may differ materially from those indicated by these forward-looking statements as a result of various important risks, uncertainties and other factors, including, without limitation, risks and uncertainties relating to: Alnylam\u2019s ability to successfully execute on its \u201cAlnylam 2030<\/i>\u201d strategy; Alnylam\u2019s ability to successfully launch, market and sell Alnylam\u2019s approved products globally; Alnylam\u2019s ability to discover and develop novel drug candidates and delivery approaches and successfully demonstrate the efficacy and safety of its product candidates; the pre-clinical and clinical results for Alnylam\u2019s product candidates; actions or advice of regulatory agencies and Alnylam\u2019s ability to obtain and maintain regulatory approval for its product candidates, as well as favorable pricing and reimbursement; delays, interruptions or failures in the manufacture and supply of Alnylam\u2019s marketed products or its product candidates; obtaining, maintaining and protecting intellectual property; Alnylam\u2019s ability to manage its growth and operating expenses through disciplined investment in operations; Alnylam\u2019s ability to maintain strategic business collaborations; Alnylam\u2019s dependence on third parties for the development and commercialization of certain products; the outcome of litigation and government investigations; the risk of future litigation and government investigations; and unexpected expenditures; as well as those risks and uncertainties more fully discussed in the \u201cRisk Factors\u201d filed with Alnylam\u2019s 2025 Annual Report on Form 10-K filed with the Securities and Exchange Commission (SEC), as may be updated from time to time in Alnylam\u2019s subsequent Quarterly Reports on Form 10-Q, and in other filings that Alnylam makes with the SEC. In addition, any forward-looking statements represent Alnylam\u2019s views only as of today and should not be relied upon as representing its views as of any subsequent date. Alnylam explicitly disclaims any obligation, except to the extent required by law, to update any forward-looking statements.<\/p> Contacts<\/b>

\nAlnylam Pharmaceuticals, Inc.<\/b>Bo Piela\n(Media)\n508-308-9783\n\nJosh Brodsky\n(Investors)\n617-551-8276<\/p>","protected":false},"excerpt":{"rendered":"

\u2212 Initiates Global Phase 2 APPlauDS Study of Mivelsiran in Down Syndrome\u2013Associated Alzheimer\u2019s Disease (DS-AD) \u2212
\n\u2212 Announces Completion of Enrollment in the Phase 2 cAPPricorn-1 Study of Mivelsiran in Cerebral Amyloid Angiopathy (CAA) \u2212
\n\u2212 Presents Up…<\/p>\n","protected":false},"author":2,"featured_media":0,"comment_status":"closed","ping_status":"closed","sticky":false,"template":"","format":"standard","meta":{"footnotes":""},"categories":[1],"tags":[],"class_list":["post-36487","post","type-post","status-publish","format-standard","hentry","category-infos-businesswire"],"_links":{"self":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts\/36487","targetHints":{"allow":["GET"]}}],"collection":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts"}],"about":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/types\/post"}],"author":[{"embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/users\/2"}],"replies":[{"embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcomments&post=36487"}],"version-history":[{"count":1,"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts\/36487\/revisions"}],"predecessor-version":[{"id":36488,"href":"https:\/\/stocks-future.com\/index.php?rest_route=\/wp\/v2\/posts\/36487\/revisions\/36488"}],"wp:attachment":[{"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Fmedia&parent=36487"}],"wp:term":[{"taxonomy":"category","embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Fcategories&post=36487"},{"taxonomy":"post_tag","embeddable":true,"href":"https:\/\/stocks-future.com\/index.php?rest_route=%2Fwp%2Fv2%2Ftags&post=36487"}],"curies":[{"name":"wp","href":"https:\/\/api.w.org\/{rel}","templated":true}]}}