$100M Funding – Phase 3 trial of first oral disease-modifying therapy for Alzheimer’s.

Clinical-stage biotech Alzheon has completed a $100 million Series E financing round to advance the development and commercialization of its oral drug for Alzheimer’s disease. The drug, called ALZ-801 (valiltramiprosate), is designed to inhibit the formation of soluble toxic beta amyloid oligomers, which are implicated in the cognitive decline observed in Alzheimer’s patients. The small molecule acts upstream of other late-stage amyloid-targeting treatments, offering a potentially more effective intervention by addressing the early formation of neurotoxic aggregates.

The new funding will be used to complete the current APOLLOE4 Phase 3 study to evaluate the efficacy and safety of ALZ-801 in patients with early Alzheimer’s disease. It will also support Alzheon’s plans to submit a New Drug Application (NDA) in 2024 based on the study’s outcomes, as well as preparing the manufacturing processes and commercial launch of ALZ-801, which is potentially the first oral disease-modifying therapy for Alzheimer’s.

“This latest fundraising ensures that we will have sufficient capital to complete our pivotal Phase 3 program and prepare commercialization of oral ALZ-801/valiltramiprosate with runway well into 2026,” said Ken Mace, CFO of Alzheon. “ALZ-801 has the potential to disrupt the Alzheimer’s treatment paradigm by slowing the progression of this relentless and debilitating disease, and the results from our pivotal APOLLOE4 Phase 3 trial will set the stage for the potential NDA filing this year, followed by the U.S. commercial launch in 2025.”

The pivotal APOLLOE4 Phase 3 study particularly targets patients with two copies of the APOE4/4 gene, a high-risk group that constitutes approximately 15% of Alzheimer’s patients, including actor Chris Hemsworth. The study, which has screened over 6,000 patients and enrolled 325 subjects, will conclude in the third quarter of 2024. Positive results could pave the way for regulatory approval and subsequent commercialization.

“We are at the dawn of a new era in the treatment of Alzheimer’s disease, and our novel therapeutic approach has an opportunity to transform the standard of care and improve access to treatment for all Alzheimer’s patients,” said Dr Martin Tolar, CEO of Alzheon. “Our well-differentiated drug candidate with a favorable safety profile, showing no increased risk of vasogenic brain edema in more than 3,000 AD patients, is positioned to potentially become the first oral disease modifying therapy for the treatment of Alzheimer’s disease.”

In addition to the Phase 3 study, ALZ-801 has been evaluated in a two-year Phase 2 biomarker trial involving 84 patients, including 31 APOE4/4 homozygotes. This trial, completed in late 2023, is currently in a fourth-year extension phase. The study’s primary goal is to assess the effects of ALZ-801 on biomarkers of Alzheimer’s pathology, alongside its clinical efficacy, safety, tolerability, and pharmacokinetic profile over 208 weeks of treatment.

Alzheon envisions extending the use of ALZ-801 beyond APOE4/4 homozygotes to include patients with one copy of the APOE4 gene and even noncarriers. The company says it is also focusing on a future precision medicine approach, leveraging individual genetic and biomarker information to tailor therapies that offer the greatest benefit to specific patient subsets.

The funding round, which was led by Alerce Medical Technology Partners, follows a $50 million Series D round completed in 2022.

“At Alerce, we focus on investing in companies in the late stages of development that are developing lifesaving drugs or devices with the potential to benefit millions of patient lives,” said Muneer Satter, Managing Partner of Alerce. “Alzheon’s ALZ-801 provides an innovative precision-medicine solution in an emerging Alzheimer’s pipeline with a path to potential approval in 2025.”