Alzheimer’s Treatment: Latest Breakthroughs and Advancements in Therapeutic Development

Alzheimer’s disease remains one of the most complex and devastating neurodegenerative disorders, affecting millions worldwide and placing an immense burden on patients, caregivers, and healthcare systems. Characterized by progressive cognitive decline, memory loss, and functional impairment, Alzheimer’s disease currently has no cure, making therapeutic innovation a critical priority. Traditional Alzheimer’s treatments have long focused on symptom management, primarily through cholinesterase inhibitors and NMDA receptor antagonists. However, these approaches offer only temporary relief without addressing the underlying causes of neurodegeneration.

In recent years, Alzheimer’s research and drug development have entered a groundbreaking era, with disease-modifying therapies (DMTs) at the forefront of innovation. Novel therapeutics targeting amyloid-beta plaques, tau protein aggregation, neuroinflammation, and synaptic dysfunction are reshaping the Alzheimer’s treatment landscape. Advances in monoclonal antibodies (mAbs), small molecules, gene therapy, and biomarker-driven approaches are not only improving diagnosis but also offering potential disease-modifying effects that could slow or even halt disease progression. The US FDA has already approved monoclonal antibodies such as Eisai and Biogen’s LEQEMBI and ADUHELM, marking a significant step toward treating the root causes of Alzheimer’s rather than just managing symptoms.

The urgency for new treatments is underscored by the growing prevalence of Alzheimer’s disease. In 2023, the 7MM (United States, EU4 [Germany, France, Italy, Spain], the United Kingdom, and Japan) reported approximately 16 million diagnosed prevalent cases, a number expected to rise with aging populations. As Alzheimer’s cases surge, the demand for innovative therapies that provide long-term efficacy, improved patient outcomes, and better disease management continues to grow.

This article explores the latest breakthroughs in Alzheimer’s treatment, highlighting emerging therapies, key drug developments, clinical advancements, and the evolving regulatory landscape. With a rapidly expanding Alzheimer’s drug pipeline and new technologies shaping the future of treatment, hope is on the horizon for millions of patients and their families.

Want to dive deeper into Alzheimer’s disease, its treatment landscape, and the latest innovations? Read our latest evergreen blog here.

KISUNLA: A Breakthrough in Alzheimer’s Disease Treatment and the Ongoing Race for a Cure

In July 2024, the FDA approved KISUNLA (donanemab-azbt), marking a major milestone in the fight against Alzheimer’s disease. Developed by Eli Lilly, KISUNLA became the first amyloid plaque-targeting therapy with evidence supporting treatment discontinuation once plaques are cleared, potentially reducing costs and infusion burden for patients.

This approval signified a new era in Alzheimer’s treatment. In the TRAILBLAZER-ALZ 2 study, KISUNLA slowed cognitive decline by 35% in early-stage patients, with some showing a 39% lower risk of disease progression. More than just numbers, this breakthrough offered real hope—a chance for patients to remember familiar faces a little longer, manage daily tasks more independently, and hold onto their identity despite the disease’s relentless grip.

But KISUNLA is only part of the story. The race to conquer Alzheimer’s is intensifying, with several biotech and pharma giants pushing boundaries, such as AB Science, Alzheon, AriBio, AgeneBio, Anavex Life Sciences, Annovis Bio, Cerecin, BioVie, Cassava Sciences, Novo Nordisk, Eli Lilly, Neurim Pharmaceuticals, Suven Life Sciences, Bristol Myers Squibb, Karuna Therapeutics, T3D Therapeutics, and others. 

Despite decades of setbacks, the Alzheimer’s research community remains relentless. The goal is no longer just slowing the disease—it’s stopping it altogether. While KISUNLA is a game-changer, the future holds even bigger breakthroughs. Scientists, drug developers, and patients alike are clinging to one powerful word: hope.

Current Commercial Strategies and Innovations in Alzheimer’s Disease Treatment

Along with hope, the relentless pursuit of a cure continues, driving rapid advancements in Alzheimer’s disease research. The treatment landscape is undergoing a transformative shift, with groundbreaking therapies emerging at an unprecedented pace. Recent breakthroughs are reshaping the market, offering new possibilities for patients, caregivers, and the broader healthcare community. As science pushes forward, so does the promise of better treatments, earlier diagnoses, and ultimately—a cure.

February 2025 

On Feb 12, 2025, NKGen Biotech, a clinical-stage biotechnology company specializing in natural killer (NK) cell therapeutics, announced that the FDA has granted Fast Track designation for its investigational therapy, troculeucel, an ex vivo expanded autologous NK cell therapy for moderate Alzheimer’s disease treatment. This designation aims to accelerate the development and regulatory review of Alzheimer’s disease therapies that address serious unmet medical needs.

According to Paul Y. Song, MD, Chairman and CEO of NKGen, the company specifically targeted moderate AD patients, who account for about 30% of Alzheimer’s cases, as most current Alzheimer’s disease treatments focus on early-stage patients. The FDA Fast Track designation follows promising results from a Phase I trial, showing early signs of clinical benefit. NKGen is now enrolling patients in its Phase IIa trial for moderate AD and anticipates sharing updated clinical data by the end of 2025. This milestone marks a step forward in Alzheimer’s disease drugs development, potentially offering a novel AD treatment for patients in need.

January 2025

On Jan 26, 2025, the FDA approved Eisai and Biogen’s Supplemental Biologics License Application (sBLA) for LEQEMBI (lecanemab-irmb), allowing for once-monthly maintenance dosing in patients with early-stage Alzheimer’s disease. This approval offers a more convenient alternative to biweekly dosing after an 18-month initiation phase, helping maintain clinical benefits while reducing treatment burden.

LEQEMBI targets toxic amyloid protofibrils, a key factor in Alzheimer’s disease progression. Clinical data from the Clarity AD trial showed that continued treatment slowed cognitive decline, reinforcing the importance of ongoing therapy. The drug is already approved in multiple countries, with additional regulatory reviews underway.

Additionally, the FDA is reviewing a subcutaneous autoinjector version for weekly maintenance dosing, with a decision expected by August 31, 2025.

Earlier on Jan 15, 2025, Spear Bio Inc. announced that the FDA granted Breakthrough Device Designation for its pTau 217 blood test, recognizing its potential to transform Alzheimer’s disease diagnosis. The designation highlighted the test’s ability to address a significant unmet need by providing a less invasive and more accessible alternative to conventional diagnostic methods, such as PET scans and lumbar punctures.

The pTau 217 biomarker has been widely acknowledged for its role in Alzheimer’s disease pathology, with support from leading research organizations, including the National Institute on Aging and the Alzheimer’s Association. However, accurately detecting low levels of pTau 217 in plasma had remained a challenge. Spear Bio’s SPEAR technology enabled highly sensitive and specific detection, allowing for earlier and more precise identification of Alzheimer’s disease.

By securing FDA Breakthrough Device Designation, Spear Bio strengthened its position in the neurodegenerative disease diagnostics space. This recognition underscored the potential of its advanced immunoassay platform to enhance patient management, accelerate diagnoses, and expand access to timely interventions and emerging Alzheimer’s therapies.

Earlier in January 2025, BioArctic announced that the FDA had accepted Eisai’s Biologics License Application (BLA) for the LEQEMBI subcutaneous autoinjector (SC-AI), setting a PDUFA action date of August 31, 2025.

LEQEMBI, an FDA-approved anti-amyloid therapy for early Alzheimer’s disease, could become the first at-home subcutaneous treatment option if approved. The 15-second injection process aims to enhance convenience, reducing the need for hospital visits.

The BLA submission was supported by Clarity AD (Study 301) open-label extension (OLE) data. LEQEMBI is already approved in multiple countries, with EMA approval pending.

Want to dive deeper into the Alzheimer’s drug pipeline and explore the next wave of potential breakthroughs? Read our latest blog now!

December 2024

In December 2024, China’s National Medical Products Administration (NMPA) approved Eli Lilly’s KISUNLA for adults with early symptomatic Alzheimer’s disease, including mild cognitive impairment and mild dementia with confirmed amyloid pathology.

China marks the fourth major approval for KISUNLA, following the U.S., Japan, and Great Britain, and arrives nearly a year after LEQEMBI gained approval. With a rapidly aging population, China is expected to be a significant market for anti-amyloid therapies.

KISUNLA’s approval is based on the TRAILBLAZER-ALZ 2 trial, where the drug slowed cognitive and functional decline by 35% versus placebo. Unlike LEQEMBI, KISUNLA is administered monthly and only until amyloid plaques are cleared, potentially reducing the treatment burden.

Lilly aims to position KISUNLA as a key Alzheimer’s therapy, despite concerns over cost and side effects. Meanwhile, uptake of anti-amyloid treatments remains under scrutiny, with physician opinions divided on their overall benefits.

Earlier in December 2024, the UK’s NICE ruled Eli Lilly’s KISUNLA too expensive for NHS use, despite MHRA approval. The second approved Alzheimer’s drug in the UK after LEQEMBI (lecanemab), KISUNLA costs $32,000 per year in the U.S.

NICE deemed its benefits insufficient to justify NHS funding, estimating its cost-effectiveness at five to six times above the usual threshold. Given as a monthly infusion, KISUNLA slows cognitive decline by four to seven months, but NICE cited uncertainty about its long-term benefits.

Lilly remains confident in KISUNLA’s value and plans further discussions with NICE, which previously rejected LEQEMBI for similar reasons. If approved, KISUNLA could have reached 70,000 eligible patients in England.

October 2024

On October 10, 2024, Annovis Bio announced a successful End-of-Phase II meeting with the FDA, receiving clearance to advance its investigational drug, buntanetap, into pivotal Phase III trials for early Alzheimer’s disease treatment.

The Phase III program will consist of two studies:

• A 6-month trial to confirm buntanetap’s symptomatic benefits in early AD.
• An 18-month trial to evaluate its potential disease-modifying effects.

While both studies are planned, a well-conducted 6-month trial may be sufficient for an NDA filing within a year of initiation.

The FDA raised no concerns regarding buntanetap’s safety, liver enzymes, drug interactions, pharmacokinetics, or dose selection and approved the use of a new crystal form of the drug for further development.

Annovis Bio CEO Maria Maccecchini emphasized the company’s commitment to launching the Phase III trial in early 2025, ensuring the study meets the highest standards to expedite regulatory approval and bring the therapy to patients as soon as possible.

September 2024

Earlier in 2024, Eli Lilly secured Japanese approval for its Alzheimer’s drug KISUNLA, marking its second major market after the U.S. The drug is indicated for early symptomatic Alzheimer’s, including mild cognitive impairment (MCI) and mild dementia with confirmed amyloid pathology.

Japan’s rapidly aging population makes it a key market, with 5 million dementia cases expected by 2030, two-thirds of which will be Alzheimer’s.

In the TRAILBLAZER-ALZ 2 study, KISUNLA slowed cognitive decline by 35% compared to placebo over 18 months. Nearly half of the patients showed no clinical progression at one year, reinforcing its potential benefits.

KISUNLA follows Eisai and Biogen’s LEQEMBI (lecanemab), which was approved in Japan a year earlier. Lilly highlights KISUNLA’s fixed-duration treatment, stopping once amyloid plaques disappear, which may offer a cost advantage over continuous treatment options like LEQEMBI.

While commercial success remains uncertain, KISUNLA’s U.S. sales performance will become clearer with Lilly’s Q3 earnings update.

From promising breakthroughs to global approvals, the Alzheimer’s treatment landscape is evolving rapidly. While some therapies are gaining regulatory approval, others are making significant strides in clinical development, bringing us closer to transformative treatments. 

While some Alzheimer’s disease drugs secure approvals and progress further, not all make it to the finish line. Discover the ones that fell short in 2024 in our latest blog. Failed Alzheimer’s Drugs – What Went Wrong?

What’s Next in the Azheimer’s Disease Treatment Landscape?

The Alzheimer’s treatment landscape is experiencing a transformative shift, driven by groundbreaking therapies, regulatory milestones, and a deeper understanding of disease pathology. With the FDA approvals of KISUNLA and LEQEMBI paving the way, next-generation treatments like AB Science’s Masitinib, Alzheon’s Valiltramiprosate, AriBio’s Mirodenafil, AgeneBio’s Levetiracetam, Anavex Life Sciences’ Blarcamesine, Annovis Bio’s Buntanetap, Cerecin’s Tricaprilin, BioVie’s Bezisterim, and Cassava Sciences’ Simufilam are pushing the boundaries of innovation. As the fight against Alzheimer’s gains momentum, the future looks more hopeful than ever.

While challenges remain—ranging from cost and accessibility to long-term efficacy and safety concerns—the momentum in Alzheimer’s research has never been stronger. The relentless pursuit of disease-modifying therapies, improved diagnostics, and novel treatment strategies signals that the battle against Alzheimer’s is far from over. With continued innovation, collaboration, and investment, the vision of halting or even reversing this devastating disease no longer seems out of reach.

For millions of patients and caregivers, these breakthroughs represent more than just scientific progress—they offer tangible hope for a future where Alzheimer’s is no longer an inevitable fate, but a conquerable challenge.