AB Science reports its revenues for the year 2025 and provides an update on its activities

AB Science Advances Masitinib ALS Phase 3, Reports Strong AB8939 AML Data

AB Science reported its annual financial results for 2025, showing an operating loss of €3.8 million (excluding non-recurring items, down 38%) and a net loss of €1.557 million (down 80%). The company provided extensive updates on its clinical pipeline, notably masitinib in Amyotrophic Lateral Sclerosis (ALS) and AB8939 in Acute Myeloid Leukaemia (AML). For masitinib, a confirmatory Phase 3 trial (AB23005) in ALS received authorizations from European countries and the FDA, building on previous study results showing a 12-month median survival increase. A clinical trial financing insurance offer of up to €39 million was secured for this trial. AB8939 in AML showed promising early Phase 1/2 results, with a 100% partial response rate in 4/4 difficult-to-treat patients with the AB8939 + venetoclax combination, and received Orphan Drug Designation from both EMA and FDA. The company also secured €9.5 million through private placements and renegotiated loan terms, while strategically focusing resources on the masitinib ALS and AB8939 AML programs.

• AB Science advanced its lead compound, masitinib, in Amyotrophic Lateral Sclerosis (ALS) with the authorization of the confirmatory Phase 3 trial (AB23005) by European countries and the FDA. This trial aims to confirm robust results from the prior AB10015 study, which demonstrated a median survival increase of 12 months in an optimal patient population. To de-risk the pivotal trial, AB Science secured a binding offer for clinical trial financing insurance (CTFI) from MCI, providing coverage up to €39 million against clinical failure, recruitment issues, or regulatory suspensions, validating the trial design and regulatory pathway.
• The development program for AB8939 in relapsed/refractory Acute Myeloid Leukaemia (AML) achieved significant milestones. The Phase 1/2 study (AB18001) progressed to its third stage, evaluating AB8939 in combination with venetoclax. Early results from this combination showed a 100% partial response rate (4/4 patients), including one complete remission, in patients with very poor genetic profiles and advanced lines of therapy. This promising efficacy, coupled with a favorable tolerability profile, led to AB8939 receiving Orphan Drug Designation from both the EMA and FDA for AML, recognizing its potential significant benefit.
• AB Science reported a reduced operating loss of €3.8 million (excluding non-recurring items) and a strengthened cash position of €10.2 million, supplemented by €3.2 million from a recent private placement. The company successfully renegotiated loan repayment terms, deferring significant principal payments. Strategically, AB Science announced a temporary suspension of other European clinical trials to focus resources on the pivotal masitinib ALS and AB8939 AML programs, while also strengthening its intellectual property portfolio with new patents granted for masitinib in progressive multiple sclerosis, sickle cell disease, and metastatic hormone-resistant prostate cancer across key markets.

Designing the Confirmatory Phase 3 for Masitinib in ALS

The literature provides comprehensive insights into ALS trial design evolution from early rhIGF-I studies through current approaches, with increasing sophistication in analytical strategies and endpoint selection. Recent trials demonstrate standardization around ALSFRS-R as the primary endpoint while incorporating advanced statistical methods including joint modeling frameworks and interim analysis optimization.

Masitinib's Clinical Efficacy and Biomarker Insights in ALS

Recent clinical studies in ALS have evaluated diverse therapeutic approaches, from novel small molecules to gene therapies and stem cell treatments. These trials have provided valuable insights into both traditional functional endpoints and emerging biomarker strategies for monitoring disease progression and treatment response.

• ROCK-ALS Trial (Fasudil): This multicenter, randomized, double-blind, placebo-controlled phase 2 study evaluated the Rho kinase inhibitor fasudil as add-on therapy to riluzole in 118 ALS patients. At day 90, fasudil significantly reduced the number of newly affected muscles compared to placebo in a dose-dependent manner, with Motor Unit Number Index (MUNIX) assessments demonstrating prognostic value for subsequent ALSFRS-R decline.

• VALOR Trial and Open-Label Extension (Tofersen): This phase 3 trial enrolled 108 participants with SOD1-ALS across 32 sites in 10 countries, with participants receiving intrathecal tofersen (100 mg) or placebo over 24 weeks. Over 148 weeks of follow-up, earlier initiation of tofersen was associated with numerically less decline in ALSFRS-R score (-9.9 vs -13.5 points), respiratory function, and muscle strength, with most adverse events being reversible and consistent with known procedural effects.

• MT-1186-A04 Study (Edaravone Oral Suspension): This phase 3b extension study compared investigational once-daily versus approved on/off dosing of edaravone oral suspension over 96 weeks total treatment duration. The once-daily regimen demonstrated equivalent efficacy to the approved on/off regimen for the primary endpoint of time to ≥12-point ALSFRS-R decrease or death (p=0.78), with no new safety concerns identified in either group.

• Adipose-derived Mesenchymal Stem Cell Trial: This multicenter, randomized, placebo-controlled, double-blinded trial evaluated three intravenous AdMSC doses (1×10⁶, 2×10⁶, 4×10⁶ cells/kg) versus placebo in 40 riluzole-treated ALS patients. While the expected progressive decline characteristic of ALS was observed across all groups with no statistically significant between-group differences in functional outcomes, the treatment demonstrated acceptable safety with similar adverse event rates between groups throughout the 36-month follow-up period.

Masitinib's Broadening Pipeline: MS, Prostate Cancer, and Beyond

Masitinib is being investigated across multiple therapeutic areas beyond ALS, with the most advanced programs targeting progressive multiple sclerosis and Alzheimer's disease. Both indications have completed phase 2/3 trials showing positive efficacy signals, with confirmatory studies planned or initiated.

• Progressive Multiple Sclerosis: Study AB07002 was a randomized, double-blind, placebo-controlled trial conducted across 116 hospital clinics in 20 countries. The study enrolled 611 patients with primary progressive MS or nonactive secondary progressive MS, randomized 2:1 to masitinib 4.5 mg/kg/day or uptitrated 6.0 mg/kg/day versus placebo over 96 weeks. The 4.5 mg/kg/day dose demonstrated significant benefit on EDSS progression with a between-group difference of -0.097 (97% CI -0.192 to -0.002; p=0.0256).

• Alzheimer's Disease: Study AB09004 evaluated masitinib as adjunctive therapy to cholinesterase inhibitors and/or memantine in patients with mild-to-moderate dementia. This randomized, double-blind trial used a two parallel-group design with patients receiving either masitinib 4.5 mg/kg/day or an uptitrated regimen (4.5 mg/kg/day for 12 weeks, then 6.0 mg/kg/day). The 4.5 mg/kg/day group showed significant improvement on ADAS-cog with a between-group difference of -2.15 (97.5% CI -3.48 to -0.81; p<0.001).

• Systemic Mastocytosis: Masitinib is under investigation as a next-generation KIT inhibitor for treating systemic mastocytosis, though specific trial design details were not provided in the available literature.

• Canine Hemangiosarcoma: Preclinical studies evaluated masitinib mesylate against canine hemangiosarcoma cell lines using dose- and time-dependent treatment models (0.01-100 μM concentrations over 24-72 hours). The compound demonstrated IC₅₀ values of 8.56-10.65 μM and induced apoptosis through caspase-3/7 activation, supporting future clinical development.

A Strategic Double-Down on High-Impact Therapies

AB Science's recent financial update, while showing continued losses, signals a pivotal strategic shift, doubling down on two high-potential assets: masitinib for Amyotrophic Lateral Sclerosis (ALS) and AB8939 for Acute Myeloid Leukaemia (AML). This focused approach, backed by significant clinical trial financing and Orphan Drug Designations, underscores a clear intent to address critical unmet medical needs with targeted therapies.

The progression of masitinib into a confirmatory Phase 3 trial for ALS is particularly noteworthy. ALS is a devastating neurodegenerative condition with limited treatment options, and previous studies suggesting a 12-month median survival increase offer a beacon of hope. This tyrosine kinase inhibitor, by targeting neuroinflammation, represents a novel mechanistic approach. However, a meta-analysis of masitinib in neurodegenerative diseases has highlighted a significantly higher incidence of adverse events, including severe ones, compared to placebo. This necessitates careful patient selection and robust safety monitoring in the ongoing trial to ensure an optimal benefit-risk profile.

Concurrently, the early-stage data for AB8939 in AML are highly encouraging. A 100% partial response rate in a small cohort of difficult-to-treat patients, particularly in combination with venetoclax, points to a potentially powerful new option for a rapidly progressing cancer. Orphan Drug Designation further validates the high unmet need and could streamline its development. The AML treatment landscape, especially for FLT3-mutated forms, is increasingly defined by targeted tyrosine kinase inhibitors. AB8939 could carve out a niche, but the inherent limitation of early-phase data means these impressive response rates require validation in larger, more diverse clinical trials. The long-term safety and efficacy of this combination will be crucial.

AB Science's ability to secure substantial financing for these programs, alongside renegotiating loan terms, demonstrates a strategic commitment to de-risk and accelerate these assets. The company's financial health remains closely tied to the success of these two programs, making their clinical and regulatory outcomes paramount for its future trajectory. The coming years will reveal whether this concentrated bet can translate into significant clinical breakthroughs and sustainable commercial success.