{"id":5146,"date":"2024-10-01T16:03:03","date_gmt":"2024-10-01T16:03:03","guid":{"rendered":"https:\/\/leonabio.com\/?page_id=5146"},"modified":"2026-07-10T17:29:33","modified_gmt":"2026-07-10T17:29:33","slug":"pipeline","status":"publish","type":"page","link":"https:\/\/leonabio.com\/pipeline\/","title":{"rendered":"Pipeline"},"content":{"rendered":"

[vc_row row_height_percent=”0″ overlay_alpha=”50″ gutter_size=”3″ column_width_percent=”100″ shift_y=”0″ z_index=”0″ content_parallax=”0″ uncode_shortcode_id=”144091″ back_color_type=”uncode-gradient” back_color_gradient=”{“css“:“background: linear-gradient(to left , rgb(32, 51, 76) 31% , rgb(51, 158, 140) 85%);“,“sliders“:`{`{“color“:“rgb(32, 51, 76)“,“position“:31},{“color“:“rgb(51, 158, 140)“,“position“:85}`}`,“type“:“linear“,“direction“:“right“}”][vc_column column_width_percent=”100″ position_vertical=”middle” gutter_size=”3″ overlay_alpha=”50″ shift_x=”0″ shift_y=”0″ shift_y_down=”0″ z_index=”0″ medium_width=”0″ mobile_width=”0″ width=”1\/2″ uncode_shortcode_id=”509687″][vc_column_text text_color=”color-xsdn” css_animation=”left-t-right” animation_delay=”400″ uncode_shortcode_id=”137406″ text_color_type=”uncode-palette”]Our Pipeline<\/span>[\/vc_column_text][\/vc_column][vc_column column_width_percent=”100″ position_vertical=”middle” gutter_size=”3″ overlay_alpha=”50″ shift_x=”0″ shift_y=”0″ shift_y_down=”0″ z_index=”0″ medium_width=”0″ mobile_width=”0″ width=”1\/2″ uncode_shortcode_id=”969935″][vc_single_image media=”5678″ media_width_percent=”50″ alignment=”center” no_lazy=”yes” uncode_shortcode_id=”286893″][\/vc_column][\/vc_row][vc_row row_height_percent=”0″ overlay_alpha=”50″ gutter_size=”3″ column_width_percent=”100″ shift_y=”0″ z_index=”0″ content_parallax=”0″ uncode_shortcode_id=”305225″ css=”.vc_custom_1767565088453{margin-top: 90px !important;margin-bottom: 90px !important;}”][vc_column width=”1\/1″][vc_column_text uncode_shortcode_id=”353470″]<\/p>\n

Our lead therapeutic drug programs target unmet needs in either oncology or neurology, leveraging differentiated mechanisms of action supported by preclinical and clinical evidence.<\/h2>\n

[\/vc_column_text][\/vc_column][\/vc_row][vc_row row_height_percent=”0″ back_color=”color-lxmt” overlay_alpha=”50″ gutter_size=”3″ column_width_percent=”100″ shift_y=”0″ z_index=”0″ content_parallax=”0″ uncode_shortcode_id=”206935″ back_color_type=”uncode-palette” css=”.vc_custom_1767564222939{padding-top: 60px !important;padding-bottom: 60px !important;}”][vc_column column_width_percent=”100″ gutter_size=”3″ overlay_alpha=”50″ shift_x=”0″ shift_y=”0″ shift_y_down=”0″ z_index=”0″ medium_width=”0″ mobile_width=”0″ width=”1\/1″ uncode_shortcode_id=”169970″][vc_column_text uncode_shortcode_id=”187896″ css=”.vc_custom_1767564254262{margin-bottom: 10px !important;}”]<\/p>\n

LeonaBio\u2019s Lead Programs<\/h1>\n

[\/vc_column_text][vc_single_image media=”5990″ media_width_percent=”100″ alignment=”center” advanced=”yes” media_overlay_opacity=”50″ media_overlay_anim=”no” media_text_anim=”no” media_image_anim=”no” media_padding=”1″ uncode_shortcode_id=”887040″][vc_column_text uncode_shortcode_id=”845862″ css=”.vc_custom_1767564346671{margin-top: 30px !important;}”]<\/p>\n

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.<\/span><\/p>\n

[\/vc_column_text][\/vc_column][\/vc_row][vc_row row_height_percent=”0″ overlay_alpha=”50″ gutter_size=”3″ column_width_percent=”100″ shift_y=”0″ z_index=”0″ content_parallax=”0″ uncode_shortcode_id=”138949″ css=”.vc_custom_1767564381461{margin-top: 60px !important;margin-bottom: 150px !important;}”][vc_column width=”1\/1″][vc_column_text uncode_shortcode_id=”549604″]<\/p>\n

In addition to our lead drug programs featured above, LeonaBio\u2019s other product candidates include ATH-1020, fosgonimeton, and LeonaBio\u2019s early compounds.<\/h3>\n

Click below to learn more.[\/vc_column_text][vc_accordion sign=”plus” active_tab=”0″][vc_accordion_tab gutter_size=”2″ column_padding=”2″ title=”Lasofoxifene” tab_id=”1766434216777-4-1″][vc_column_text uncode_shortcode_id=”211045″]Lasofoxifene\u202fis 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\u202fESR1 mutations\u202fcommonly 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.<\/p>\n

Lasofoxifene is being advanced in a Phase 3 clinical trial as a targeted therapy for\u202festrogen 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\u202fELAINE-3 Phase 3 trial\u202fis 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.[\/vc_column_text][\/vc_accordion_tab][vc_accordion_tab gutter_size=”2″ column_padding=”2″ title=”Brelgometon (ATH-1105)” tab_id=”1671666987768-3-9″][vc_column_text uncode_shortcode_id=”107036″]Brelgometon (ATH-1105) is LeonaBio\u2019s 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\u2019s disease, and Parkinson\u2019s disease. Brelgometon is currently in clinical development for the potential treatment of ALS.<\/p>\n

In May 2025, LeonaBio presented data from the first-in-human Phase 1 clinical trial (NCT 06432647<\/a>) of brelgometon at the 4th<\/sup>Annual ALS Drug Development Summit\u00a0highlighting\u00a0brelgometon has demonstrated consistent and robust beneficial effects in preclinical models of ALS\u00a0and\u00a0showed a favorable safety profile and was well tolerated in both single and multiple ascending dose studies in healthy volunteers. In addition,\u00a0brelgometon showed dose proportional pharmacokinetics and\u00a0CNS penetration\u00a0in this first-in-human study.<\/p>\n

The\u00a0first-in-human Phase 1 (NCT 06432647<\/a>) double-blind, placebo-controlled clinical trial enrolled 80 healthy volunteers to evaluate single and multiple oral ascending doses of brelgometon. A Phase 2 proof-of-concept study is targeted for initiation in the second half of 2026.[\/vc_column_text][\/vc_accordion_tab][vc_accordion_tab gutter_size=”2″ column_padding=”2″ title=”ATH-1020″ tab_id=”1640377031-2-83″][vc_column_text uncode_shortcode_id=”277250″]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.<\/p>\n

LeonaBio completed a Phase 1 clinical trial [NCT05169671<\/a>] 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.[\/vc_column_text][\/vc_accordion_tab][vc_accordion_tab gutter_size=”2″ column_padding=”2″ title=”Early Compounds” tab_id=”1640377417192-3-3″][vc_column_text uncode_shortcode_id=”518777″]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.[\/vc_column_text][\/vc_accordion_tab][vc_accordion_tab gutter_size=”2″ column_padding=”2″ title=”Fosgonimeton (ATH-1017)” tab_id=”1640377031-1-47″][vc_column_text uncode_shortcode_id=”328981″ css=”.vc_custom_1762978131303{margin-bottom: 30px !important;}”]<\/p>\n

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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\u2019s disease (AD) and Parkinson\u2019s disease (PD).<\/p>\n<\/div>\n

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Fosgonimeton is currently delivered via a simple at-home injection just under the skin.<\/p>\n<\/div>\n

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For AD, fosgonimeton was evaluated in the completed exploratory Phase 2 ACT-AD clinical trial [NCT04491006<\/a>] in people with mild-to-moderate Alzheimer\u2019s disease and in the late-stage randomized, double-blind, placebo-controlled, parallel-group clinical trial, LIFT-AD [NCT04488419<\/a>]. Access the topline data summary of this trial here<\/a>.<\/p>\n<\/div>\n

For PD, Fosgonimeton was previously evaluated in an exploratory Phase 2 SHAPE clinical trial [NCT04831281<\/a>] in people with Parkinson\u2019s disease dementia or dementia with Lewy bodies. Access the topline data summary of this trial here<\/a>.[\/vc_column_text][\/vc_accordion_tab][\/vc_accordion][\/vc_column][\/vc_row]<\/p>\n<\/div>","protected":false},"excerpt":{"rendered":"

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