Our lead therapeutic drug programs target unmet needs in either oncology or neurology, leveraging differentiated mechanisms of action supported by preclinical and clinical evidence.
LeonaBio’s Lead Programs
Disclaimer: Product candidates described in the Pipeline are under development, subject to ongoing testing and clinical evaluation, and have not been approved by any regulatory authority for any commercial use in humans.
In addition to our lead drug programs featured above, LeonaBio’s other product candidates include ATH-1020, fosgonimeton, and LeonaBio’s early compounds.
Click below to learn more.
Lasofoxifene is a novel, nonsteroidal selective estrogen receptor modulator (SERM) with a unique binding profile, designed to confer potent activity against both wild-type and mutant estrogen receptors, including the clinically significant ESR1 mutations commonly associated with resistance to endocrine therapy in metastatic breast cancer. Originally developed for osteoporosis, lasofoxifene has demonstrated promising antitumor activity and favorable tolerability in multiple clinical studies.
Lasofoxifene is being advanced in a Phase 3 clinical trial as a targeted therapy for estrogen receptor-positive (ER+), HER2-negative, ESR1-mutated metastatic breast cancer, a population with limited treatment options following progression on aromatase inhibitors and CDK4/6 inhibitors. The ongoing ELAINE-3 Phase 3 trial is evaluating lasofoxifene in combination with the CDK4/6 inhibitor, abemaciclib, and is aiming to establish a new standard of care for this genetically defined patient group.
ATH-1105 is Athira’s novel, orally available, brain-penetrant, next-generation small molecule drug candidate designed to positively modulate the neurotrophic HGF system for potential treatment of neurodegenerative diseases, including amyotrophic lateral sclerosis (ALS), Alzheimer’s disease, and Parkinson’s disease. ATH-1105 is currently in clinical development for the potential treatment of ALS.
In May 2025, Athira presented data from the first-in-human Phase 1 clinical trial (NCT 06432647) of ATH-1105 at the 4thAnnual ALS Drug Development Summit highlighting ATH-1105 has demonstrated consistent and robust beneficial effects in preclinical models of ALS and showed a favorable safety profile and was well tolerated in both single and multiple ascending dose studies in healthy volunteers. In addition, ATH-1105 showed dose proportional pharmacokinetics and CNS penetration in this first-in-human study.
The first-in-human Phase 1 (NCT 06432647) double-blind, placebo-controlled clinical trial enrolled 80 healthy volunteers to evaluate single and multiple oral ascending doses of ATH-1105. Athira plans to initiate a Phase 2 clinical trial of ATH-1105 in ALS patients in early 2026.
ATH-1020 is a novel, orally available, next-generation small molecule drug candidate designed to positively modulate the neurotrophic HGF system. In preclinical models, ATH-1020 was shown to reduce measures of pain in models of diabetic neuropathy.
LeonaBio completed a Phase 1 clinical trial [NCT05169671] of ATH-1020 in healthy volunteers. ATH-1020 showed a favorable safety profile and was well-tolerated. LeonaBio plans to evaluate options with this compound and will consider its advancement in relation to other opportunities and resources.
Several new compounds are currently in preclinical discovery and development for neurodegenerative diseases and other indications for which LeonaBio believes positive modulation of the neurotrophic HGF system may have therapeutic potential.
Fosgonimeton is a small molecule drug candidate designed to enhance the activity of the neurotrophic hepatocyte growth factor (HGF) system. By targeting the protection and repair of neuronal networks, fosgonimeton offers disease-modifying potential for a broad range of neurodegenerative diseases, including Alzheimer’s disease (AD) and Parkinson’s disease (PD).
Fosgonimeton is currently delivered via a simple at-home injection just under the skin.
For AD, fosgonimeton was evaluated in the completed exploratory Phase 2 ACT-AD clinical trial [NCT04491006] in people with mild-to-moderate Alzheimer’s disease and in the late-stage randomized, double-blind, placebo-controlled, parallel-group clinical trial, LIFT-AD [NCT04488419]. Access the topline data summary of this trial here.
For PD, Fosgonimeton was previously evaluated in an exploratory Phase 2 SHAPE clinical trial [NCT04831281] in people with Parkinson’s disease dementia or dementia with Lewy bodies. Access the topline data summary of this trial here.