We are thrilled to be advancing our small molecule therapeutic candidates, which have promising clinical and preclinical data suggesting neuroprotective, neurotrophic, procognitive, and disease-modifying effects. Our Fosgonimeton Phase 2/3 LIFT-AD study in mild-to-moderate Alzheimer’s disease is on track for completion of targeted enrollment by mid-2023. Excitingly, this will be followed by topline data in early 2024. We are also looking forward to filing an Investigational New Drug (IND) application for our ATH-1105 candidate for amyotrophic lateral sclerosis (ALS) in 2023.
Athira Pharma, Inc. (NASDAQ: ATHA), a late clinical-stage biopharmaceutical company determined to halt the progression of neurodegeneration, announced an update on its pipeline development programs and 2023 outlook. The company is dedicated to developing small molecules that will restore neuronal health and pave the way for a brighter future.
At Athira Pharma, our commitment to developing novel therapeutics to combat neurodegenerative diseases is unwavering. Our data, to be shared in 2022, further bolsters our assurance in our small molecule approach targeting the HGF/MET system for diseases such as Alzheimer’s, Parkinson’s, and ALS. Our discoveries from preclinical models across multiple neurodegenerative diseases, and even Alzheimer’s patients, indicate that our small molecule product candidates may have the potential to be neuroprotective, neurotrophic, procognitive and even disease-modifying. In particular, recently published preclinical results in the Neurotherapeutics journal further support the promise of fosgonimeton.
This year, Athira is poised to make waves in the medical world. We are on track to complete enrollment of the LIFT-AD study for mild-to-moderate Alzheimer’s disease in mid-2023, and in the meantime, we are excited to file an IND application for ATH-1105 as a potential treatment for ALS. The independent, unblinded interim efficacy and futility analysis of the Phase 2/3 LIFT-AD study provides us with confidence in the potential clinical benefits of fosgonimeton treatment, and we can’t wait to see the results. 2021 is sure to be an exciting year for Athira!
Fosgonimeton, an HGF/MET enhancement, may be a viable treatment for neurodegenerative diseases such as Alzheimer’s and Parkinson’s. We are dedicated to investigating the potential of this innovative therapy, and our strong balance sheet allows us to advance our other programs such as ATH-1105 in ALS. As we progress, we will continue to present and publish data that further support the efficacy of this treatment.
Athira’s 2023 Pipeline Outlook: Status and Upcoming Milestones
Fosgonimeton (ATH-1017) is a small molecule with the potential to protect and repair neural networks. It is designed to enhance the activity of hepatocyte growth factor (HGF) and its receptor, MET, allowing the body to effectively regenerate and maintain healthy neural networks. With Fosgonimeton, researchers hope to unlock the power of the brain and revolutionize treatments for neurological diseases.
LIFT-AD Phase 2/3 Study in mild-to-moderate Alzheimer’s Disease
- Following the encouraging results from the exploratory Phase 2 ACT-AD trial, Athira has taken proactive steps to amend the entry criteria for the upcoming LIFT-AD trial in September 2022. The trial will investigate the effects of fosgonimeton compared with placebo without requiring background therapy, in order to further understand the potential of this promising treatment.
- In October 2022, an independent data monitoring committee conducted an unblinded interim efficacy and futility analysis and, based on the results, recommended that the LIFT-AD study be continued in patients with mild-to-moderate Alzheimer’s disease. This milestone is a testament to the dedication and hard work of researchers striving to find new and innovative treatments for this debilitating condition.
- The committee found that the study will be adequately powered to assess the primary endpoint, since the total enrollment of fewer than 300 patients, with no prior treatment, is expected to yield a significant effect size based on preliminary data.
- The LIFT-AD study is nearing completion, and we anticipate enrolling all participants by mid-2023. Our goal is to publish the topline data in early 2024, so stay tuned for exciting updates!
Open Label Extension (OLEX) study
At the end of 2022, an astounding 85% of participants who had completed the ACT-AD and LIFT-AD studies chose to continue their journey with the Open Label Extension (OLEX) study. Currently, over 200 people are taking part in the OLEX study, making it a pivotal advancement in the medical field.
SHAPE Phase 2 Study in mild-to-moderate Parkinson’s disease dementia and Dementia with Lewy bodies
- Athira recently enrolled 28 participants in the exploratory Phase 2 SHAPE study of fosgonimeton for those suffering from Parkinson’s disease dementia or Dementia with Lewy bodies.
- After the completion of the treatment and evaluation of the results, the Company will seek to explore the broader potential of fosgonimeton to improve motor function in Parkinson’s disease.
- The compelling preclinical data presented on the potential of fosgonimeton has inspired the Company to evaluate new study designs that can further explore this complex disease.
Introducing the revolutionary new small molecule, PATH-1020 – a breakthrough potential treatment for neuropathic pain or neurodegenerative disorders that can be taken orally! PATH-1020 is designed to enhance the HGF/MET system, providing an unprecedented level of relief from the effects of these debilitating conditions.
Phase 1 Study in Healthy Volunteers
- Following the successful completion of the Phase 1, single-ascending, dose escalation study of ATH-1020, it has been revealed that the compound was well tolerated in healthy volunteers, displaying a favorable safety profile.
- The Company is now assessing its options with this promising compound and will take into consideration other opportunities and resources as it moves forward with its development.
ATH-1105, a small molecule positive modulator of the HGF/MET system is showing promise as a potential treatment for ALS. Recent preclinical data in December 2022 revealed that ATH-1105 exhibited consistent improvements in motor function, nerve function, and neurodegeneration in a TDP-43 mouse model of ALS.
- With these promising results, the Company is preparing to submit an IND application with the U.S.
- Food and Drug Administration in 2023 to initiate first-in-human studies for this groundbreaking product candidate.
- As of September 30, 2022, Athira reported a healthy balance of cash, cash equivalents and investments, totaling $260.0 million. This impressive figure is a testament to the company’s financial strength and stability.
About Athira Pharma, Inc.
Athira Pharma, Inc., based in the Seattle, Washington area, is a biopharmaceutical company dedicated to discovering small molecules that can restore neuronal health and slow the progression of neurodegenerative diseases. Our novel approach has the potential to provide rapid cognitive improvement, and revolutionize the treatment of Alzheimer’s, Parkinson’s, Dementia with Lewy bodies, and ALS. We are on the cutting edge of research and development, and are excited to be bringing our innovative ideas to the forefront of neurological disease treatment.
At Athira, we are excited about the potential of our product candidates to be a potential treatment for Alzheimer’s disease, Parkinson’s disease dementia, Dementia with Lewy bodies, neuropsychiatric diseases, and other neurodegenerative diseases, such as amyotrophic lateral sclerosis. Our platform technology has the potential to revolutionize these medical fields, and we are pursuing clinical and regulatory objectives in order to bring these therapies to market. We are confident that these products can have a real impact on the lives of those affected by these diseases, and we are dedicated to advancing our product candidates through the necessary stages of development in order to achieve this goal. However, there are risks and uncertainties associated with these efforts, including the impact of the COVID-19 pandemic on our business, research, and clinical development plans and timelines, as well as the regulatory process for our product candidates. Despite these risks, we remain committed to our mission of discovering and developing transformative therapies to improve the lives of patients.