Scientific-stage biotech Alzheon has accomplished a $100 million Collection E financing spherical to advance the event and commercialization of its oral drug for Alzheimer’s illness. The drug, referred to as ALZ-801 (valiltramiprosate), is designed to inhibit the formation of soluble poisonous beta amyloid oligomers, that are implicated within the cognitive decline noticed in Alzheimer’s sufferers. The small molecule acts upstream of different late-stage amyloid-targeting remedies, providing a doubtlessly more practical intervention by addressing the early formation of neurotoxic aggregates.
The brand new funding might be used to finish the present APOLLOE4 Section 3 research to judge the efficacy and security of ALZ-801 in sufferers with early Alzheimer’s illness. It should additionally help Alzheon’s plans to submit a New Drug Software (NDA) in 2024 primarily based on the research’s outcomes, in addition to getting ready the manufacturing processes and industrial launch of ALZ-801, which is doubtlessly the primary oral disease-modifying remedy for Alzheimer’s.
“This newest fundraising ensures that we are going to have enough capital to finish our pivotal Section 3 program and put together commercialization of oral ALZ-801/valiltramiprosate with runway effectively into 2026,” mentioned Ken Mace, CFO of Alzheon. “ALZ-801 has the potential to disrupt the Alzheimer’s therapy paradigm by slowing the development of this relentless and debilitating illness, and the outcomes from our pivotal APOLLOE4 Section 3 trial will set the stage for the potential NDA submitting this yr, adopted by the U.S. industrial launch in 2025.”
The pivotal APOLLOE4 Section 3 research notably targets sufferers with two copies of the APOE4/4 gene, a high-risk group that constitutes roughly 15% of Alzheimer’s sufferers, together with actor Chris Hemsworth. The research, which has screened over 6,000 sufferers and enrolled 325 topics, will conclude within the third quarter of 2024. Constructive outcomes might pave the way in which for regulatory approval and subsequent commercialization.
“We’re on the daybreak of a brand new period within the therapy of Alzheimer’s illness, and our novel therapeutic method has a possibility to remodel the usual of care and enhance entry to therapy for all Alzheimer’s sufferers,” mentioned Dr Martin Tolar, CEO of Alzheon. “Our well-differentiated drug candidate with a positive security profile, exhibiting no elevated threat of vasogenic mind edema in additional than 3,000 AD sufferers, is positioned to doubtlessly turn out to be the primary oral illness modifying remedy for the therapy of Alzheimer’s illness.”
Along with the Section 3 research, ALZ-801 has been evaluated in a two-year Section 2 biomarker trial involving 84 sufferers, together with 31 APOE4/4 homozygotes. This trial, accomplished in late 2023, is at present in a fourth-year extension part. The research’s main objective is to evaluate the consequences of ALZ-801 on biomarkers of Alzheimer’s pathology, alongside its medical efficacy, security, tolerability, and pharmacokinetic profile over 208 weeks of therapy.
Alzheon envisions extending the usage of ALZ-801 past APOE4/4 homozygotes to incorporate sufferers with one copy of the APOE4 gene and even noncarriers. The corporate says it’s also specializing in a future precision medication method, leveraging particular person genetic and biomarker data to tailor therapies that supply the best profit to particular affected person subsets.
The funding spherical, which was led by Alerce Medical Expertise Companions, follows a $50 million Collection D spherical accomplished in 2022.
“At Alerce, we give attention to investing in firms within the late phases of growth which might be growing lifesaving medicine or gadgets with the potential to profit tens of millions of affected person lives,” mentioned Muneer Satter, Managing Associate of Alerce. “Alzheon’s ALZ-801 supplies an progressive precision-medicine answer in an rising Alzheimer’s pipeline with a path to potential approval in 2025.”