mirodenafil (AR1001) / ariBio, Samjin Pharma, Fosun Pharma, Neuco United - LARVOL DELTA

POLARIS-AD: A Global Phase 3 Trial of AR1001 in Early Alzheimer's Disease: Efficacy Outcomes at 52 Weeks and Safety Through the Extension Phase (CTAD 2026) - No abstract available

Clinical • P3 data • Alzheimer's Disease • CNS Disorders

"Aribio Holdings Set to Announce AR1001 Phase 3 Results... Rapid Commercialization Expected" (The Asia Business Daily) - "If the clinical results are positive, there are projections that a New Drug Application (NDA) could be submitted to the U.S. Food and Drug Administration (FDA) as early as 2027...On September 21, ValueFinder anticipated that the topline data for the global Phase 3 clinical trial of AR1001 by Aribio Holdings would be released between October and November this year. The firm did not present an investment opinion or a target price."

FDA filing • P3 data • Alzheimer's Disease

Aribio and ForeverNK join to develop next-gen Alzheimer's drugs in South Korea (Chosun Biz) - "The agreement was pursued to combine Aribio's new drug development capabilities with ForeverNK's NK cell culture and immune evaluation platform technology to discover new therapeutic targets and candidate compounds. Aribio has secured capabilities in developing new drugs for degenerative brain diseases through the global phase 3 clinical trial of AR1001, an Alzheimer's disease treatment candidate....ForeverNK plans to support candidate discovery and preclinical research based on its NK cell therapy research, cell culture technology, and immune function evaluation platform. ForeverNK is an immunology-focused bio corporations with NK cell therapies, cell culture kits, and immune function evaluation technologies."

Licensing / partnership • Alzheimer's Disease

AriBio Independently Completes Global Phase 3 Trial of Oral Alzheimer's Drug Candidate 'AR1001' (BioNews) - "The company particularly highlighted the low discontinuation rate of 12.2% in the long-term Alzheimer's disease trial. In addition, 95.5% of patients who completed the treatment elected to enroll in the subsequent one-year extension trial, resulting in a total extension trial population of more than 1,250 participants....The extension trial is expected to conclude at the end of June 2027...The company plans to announce top-line data containing key clinical results between late September and October...AriBio is moving forward with its planned merger with Solux, a KOSDAQ-listed company, with the merger scheduled to take effect on September 29. "

Commercial • P3 data: top line • Trial completion • Trial status • Alzheimer's Disease

Evaluation Of POLARIS AD-Aligned Enrollment Criteria And Longitudinal Outcomes In A Phase 2 AR1001 Study (AAIC 2026) - No abstract available

P2 data

Polaris-AD: AR1001 Additional Phase 2 Data And Phase 3 Trial Update With Topline Results Expected Later This Year (AAIC 2026) - No abstract available

P2 data • P3 data • P3 data: top line

Aribio joins Lilly TuneLab to boost AI-driven CNS drug development (Chosun Biz) - "Through this participation, Aribio aims to advance data-driven decision-making across its neuroscience pipeline and increase the precision and likelihood of success in developing therapies that improve the disease itself beyond relieving symptoms...Aribio is currently operating its own AI-based drug discovery platform, ARIDD (AI-powered, Rapid and Integrated Drug Development). The platform is being used to develop AR1001, an oral, polypharmacological Alzheimer's disease candidate that targets multiple causal pathways simultaneously, and AR1005, a candidate therapy for dementia with Lewy bodies."

Licensing / partnership • Alzheimer's Disease • Dementia • Lewy Body Disease

On 13 May, Shanghai Fosun Pharmaceutical…jointly announced the signing of an exclusive global option agreement for the development, registration, manufacturing and commercialization of AR1001, an innovative drug candidate for the treatment of Alzheimer's disease (AD) (PRNewswire) - "The transaction adopts an 'option fee plus license payment' structure. Fosun Pharma will pay AriBio a US$60 million option fee and retains the right to decide whether to exercise the option within the agreed exercise period. Upon exercise of the option, Fosun Pharma will pay up to US$180 million in upfront and regulatory milestone payments....AR1001...is currently being evaluated in POLARIS-AD (NCT05531526), a global Phase 3 clinical trial for early AD....Top-line results are anticipated in 2026."

Licensing / partnership • P3 data: top line • Alzheimer's Disease

Phase 3, Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants With Early Alzheimer's Disease (Polaris-AD) (clinicaltrials.gov) - P3 | N=1535 | Active, not recruiting | Sponsor: AriBio Co., Ltd. | N=479 ➔ 1535

Enrollment change • Alzheimer's Disease • CNS Disorders

AriBio "Discloses 'AR1001' Phase 3 Progress at ADPD 2026" [Google translation] (HIT News) - "'The baseline demographic characteristics and cognitive and functional indicators of POLARIS-AD are similar to those of existing global regulatory clinical trials, and no unexpected safety signals have been observed to date.'...'AR1001 has very high blood-brain barrier (BBB) ​​permeability and superior selectivity for PDE5 compared to PDE6 and PDE11, resulting in fewer side effects compared to drugs in its class.'....Meanwhile, AriBio plans to announce top-line data from the global Phase 3 clinical trial of AR1001 in the upcoming third quarter, followed by the goal of submitting a New Drug Application (NDA) to the U.S. Food and Drug Administration (FDA) in the second half of the year."

FDA filing • P3 data: top line • Alzheimer's Disease

AriBio's presentation on its Alzheimer’s therapeutic candidate AR1001 is among the most anticipated. (Korea Biomedical Review) - "AriBio will deliver an oral presentation...detailing progress in its global phase 3 clinical trial...The ongoing Polaris-AD study is a global, multicenter, randomized, double-blind, placebo-controlled clinical trial in patients with early Alzheimer’s disease...Most adverse events reported were mild or moderate. Common events included diarrhea (4.6 percent), urinary tract infection (4.5 percent), falls (3.8 percent), headache (3.6 percent) and dizziness (3.1 percent)...Notably, no ARIA-related optic neuropathy -- an adverse event associated with anti-amyloid antibody therapies -- was reported. Patient retention was high, with 97 percent of participants who completed treatment enrolling in the extension study."

P3 data • Alzheimer's Disease

Aribio signs exclusive sales agreement with Fosun Pharmaceutical worth 630 billion won for AR1001. [Google translation] (HIT News) - "This agreement includes an upfront payment and milestone payments for development, regulatory, and commercialization. A separate royalty payment based on net sales after commercialization is also included. Fosun Pharma will have exclusive rights to manufacture, license, and commercialize AR1001 in the 10 ASEAN countries (Singapore, Indonesia, the Philippines, Thailand, Vietnam, Cambodia, Laos, Malaysia, Myanmar, and Brunei)."

Licensing / partnership • Alzheimer's Disease

Drug Development. (PubMed, Alzheimers Dement) - "The subgroup analysis of participants on AR1001 30 mg without concomitant AD medication suggests potential for AR1001 as a monotherapy for the treatment of Alzheimer's disease."

Biomarker • Clinical • Journal • Alzheimer's Disease • CNS Disorders

Aribio's AR1001 enters final stages of global Phase 3 clinical trials, drawing attention from international scholars. [Google translation] (HIT News) - "The company anticipates that the announcement of Phase 3 clinical trial results, scheduled for the first half of next year..."

P3 data • Trial status • Alzheimer's Disease

Developing Topics. (PubMed, Alzheimers Dement) - P3 | "We found that AR1001 effectively clears oAβ and exhibits anti-inflammatory effects in astrocytes and microglia. It also provides significant protective effects in neurons, reducing p -tau levels. These findings highlight the multiple beneficial effects of AR1001 on key brain components: astrocytes, microglia, and neurons."

Journal • CNS Disorders • CCL2 • CD86 • CXCL1 • CXCL10 • CXCL8 • IFNG • MRC1 • Napsin A

AriBio Co. Ltd. – AR1001 Phase 3 neurodegeneration program (POLARIS-AD) (CTAD 2025) - "Sponsored by AriBio Co. Ltd."

P3 data • CNS Disorders

Protective effects of phosphodiesterase 5 inhibitor, mirodenafil, on traumatic brain injury-induced neuron death. (PubMed, Neuropharmacology) - "These findings suggest that mirodenafil supports neuronal survival by preserving cGMP levels, enhancing NO function, and mitigating oxidative stress related to zinc dysregulation. This study highlights mirodenafil as a potential therapeutic option for limiting TBI-induced neuronal injury and preserving brain function."

Journal • CNS Disorders • Inflammation • Vascular Neurology

SK chemicals said it has signed a memorandum of understanding with AriBio to strengthen cooperation on the development and global commercialization of AR1001 (mirodenafil), an oral Alzheimer’s disease treatment candidate. (Korea Biomedical Review) - "Under the new agreement, the two companies will explore collaboration opportunities in several areas, including the development of next-generation formulations of mirodenafil, global clinical cooperation, and potential manufacturing and export partnerships following AR1001’s commercialization....AriBio’s phase 3 study, POLARIS-AD, has enrolled 1,535 participants across 13 countries. Completion is planned for early 2026, with results expected before an NDA filing to the U.S. FDA."

Licensing / partnership • P3 data • Trial completion date • Alzheimer's Disease

Aribio to Share Results of Dementia Treatment 'AR1001' at BIX 2025 [Google translation] (HIT News) - "In this session, CEO Fred Kim will present the new drug target discovery, candidate design, and development process for AR1001, an Alzheimer's treatment candidate currently undergoing global Phase 3 clinical trials in 13 countries. He will present specific details and data, focusing on key results from AR1001's Phase 2 clinical trial and patient treatment cases from Phase 3 clinical trials."

P2 data • Trial status • Alzheimer's Disease

Phase 3, Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants With Early Alzheimer's Disease (Polaris-AD) (clinicaltrials.gov) - P3 | N=1535 | Active, not recruiting | Sponsor: AriBio Co., Ltd. | Trial primary completion date: Dec 2025 ➔ May 2026

Trial primary completion date • Alzheimer's Disease • CNS Disorders

A phase 2 randomized, placebo-controlled study on the efficacy and safety of AR1001, a phosphodiesterase-5 inhibitor, in patients with mild-to-moderate Alzheimer's disease. (PubMed, J Prev Alzheimers Dis) - P2 | "AR1001 was safe and well tolerated. Although primary efficacy endpoints were not met after 26 weeks of treatment, participants receiving 30 mg AR1001 showed favorable changes in AD-related plasma biomarkers compared to placebo."

Clinical • Journal • P2 data • Alzheimer's Disease • CNS Disorders • Dementia • Depression • Psychiatry • Aβ42 • GFAP • NEFL

Phase 2 Trial Data Support AR1001 as Potential Alzheimer Disease Monotherapy (NeurologyLive) - P2 | N=210 | NCT03625622 | Sponsor: AriBio Co., Ltd. | "New phase 2 data (NCT03625622) presented at the 2025 Alzheimer’s Association International Conference...As an oral PDE5 inhibitor with known blood-brain barrier penetration, AR1001 was assessed not only for cognitive outcomes but also for changes in plasma biomarkers of neurodegeneration. Overall, the study’s findings suggest that AR1001 may exert therapeutic effects through multiple mechanisms beyond traditional symptomatic treatment....In the subgroup of participants not receiving concomitant AD medications, 30 mg AR1001 once daily for 26 weeks was associated with a statistically significant 4.019-point improvement from baseline on the Alzheimer’s Disease Assessment Scale–Cognitive Subscale 13 (ADAS-Cog-13; P = .012), the primary end point. Plasma concentrations of phosphorylated tau (ptau) biomarkers were also significantly reduced: ptau-181 by 1.361 pg/mL (P = .023)..."

P2 data • Alzheimer's Disease

Phase 3, Double-blind, Randomized, Placebo-controlled Trial to Evaluate the Efficacy and Safety of AR1001 in Participants With Early Alzheimer's Disease (Polaris-AD) (clinicaltrials.gov) - P3 | N=1535 | Active, not recruiting | Sponsor: AriBio Co., Ltd. | N=1150 ➔ 1535

Enrollment change • Alzheimer's Disease • CNS Disorders

Fosun Medicine Receives Exclusive License for Alzheimer’s Innovative Drug AR1001 [Google translation] (Fosun Pharma Press Release) - "Shanghai Fosun Pharmaceutical...announced that they have signed an exclusive licensing agreement for the production and commercialization of AR1001 in Greater China, including Mainland China, Hong Kong Special Administrative Region of China and Macau Special Administrative Region of China (License Area). AR1001 is a small molecule oral drug intended to delay the progression of Alzheimer’s disease (AD) disease with a potent, highly selective inhibition of phosphodiesterase-5 (PDE-5)....Under the terms of the agreement, Fosun Pharmaceuticals will receive exclusive production and commercialization rights of AR1001 within the licensed area. Newco continues to coordinate collaboration with AriBio to advance the completion of the global Phase III clinical trial."

Licensing / partnership • Alzheimer's Disease

AriBio licenses Alzheimer’s pill to Arcera in $600 mil. deal as merger partner Solux surges (Korea Biomedical Review) - "Korean biotech AriBio has handed over regional rights to its experimental Alzheimer’s drug in a deal worth up to $600 million, aligning with Arcera to drive commercialization across Latin America, the Middle East, Southern Africa, Ukraine, and parts of Eurasia...the licensing and supply agreement gives Arcera and its Swiss affiliate Acino exclusive rights to market AR1001 (mirodenafil), while AriBio retains manufacturing control...The drug is currently in a global phase 3 trial that has enrolled more than 1,500 patients across 13 countries, exceeding its initial target. Topline data are expected in the first half of 2026."

Licensing / partnership • P3 data: top line • Alzheimer's Disease