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Use of ATH-1105, a small molecule being developed by <a href="https://www.athira.com/" rel="nofollow">Athira Pharma</a> to treat <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), protected against nerve damage and improved motor function in a mouse model of the disease, according to data shared in a recent <a href="https://investors.athira.com/static-files/40ed1eb5-fa61-4be4-98fe-1204be7a12a5" rel="nofollow">company presentation</a>.
Athira is planning to file an investigational new drug application with the U.S. Food and Drug Administration (FDA), essentially a request for permission to begin clinical testing of ATH-1105 in people.
&#8220;Our dedication to advancing new therapeutics to impact neurodegenerative diseases remains steadfast,&#8221; Mark Litton, PhD, the company&#8217;s president and CEO, said in a<a href="https://www.globenewswire.com/news-release/2023/01/05/2583521/0/en/Athira-Pharma-Provides-2023-Pipeline-Outlook.html"> press release</a>.
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<h3>Small molecule aiming to treat ALS by maintaining nerve cell health</h3>
ATH-1105 is designed to activate the HGF/MET system, a molecular signaling pathway that plays numerous roles in maintaining the health of nerve cells. Research over the past two decades has shown that increasing the activity of the HGF-MET pathway can reduce disease severity in laboratory models of ALS.
In the December presentation, motor neurons — the specialized nerve cells controlling movement that die in ALS — in lab dishes treated with ATH-1105 were reported to show significantly better survival under toxic conditions.
The experimental therapy also was tested in a mouse model of ALS caused by<a href="https://alsnewstoday.com/als-and-genetics/"> mutations in the TARDBP gene</a> that provides instructions for making the TDP-43 protein. Mutations in this gene account for about 4% of familial ALS cases and 1% of sporadic cases. Yet, atypical clumps of the TDP-43 protein are found in almost all cases of ALS, and thought to help drive the disease.
Compared with untreated mice in the model, those given ATH-1105 maintained significantly higher body weight, indicating generally better health. Treated mice also had significantly lower levels of inflammatory markers, namely TNF-alpha and IL-6, and lower levels of neurofilament light chain (NfL), a marker of nerve damage, researchers reported.
Analyses of the mice&#8217;s nerve cells showed that those treated with ATH-1105 had less degeneration and better electrical activity. These mice also performed better on tests of motor function, although their performance was significantly poorer than that of healthy animals.
&#8220;These results highlight the therapeutic potential of ATH-1105 in ALS and support further development,&#8221; the researchers concluded.
Athira is currently developing several other experimental therapies targeting the HGF/MET pathway, including one called fosgonimeton that is <a href="https://alzheimersnewstoday.com/news/continuation-alzheimers-lift-ad-trial-backed-safety-board/">currently in a clinical trial</a> in <a href="https://alzheimersnewstoday.com/what-is-alzheimers-disease/">Alzheimer&#8217;s</a> patients and a <a href="https://parkinsonsnewstoday.com/news/first-patient-dosed-shape-trial-ath-1017-parkinsons-dementia/">separate trial</a> that includes people with <a href="https://parkinsonsnewstoday.com/what-is-parkinsons-disease/">Parkinson&#8217;s disease</a> dementia.
&#8220;The consistency of data from preclinical models across various neurodegenerative diseases and in Alzheimer’s patients suggest that our small molecule product candidates may be neuroprotective, neurotrophic, procognitive and potentially disease-modifying,&#8221; Litton said.
&#8220;We expect to complete enrollment of the LIFT-AD study [testing fosgonimeton in] mild-to-moderate Alzheimer’s disease … [and] to filing an IND application for ATH-1105 as a potential treatment for ALS,&#8221; Litton added. &#8220;We also plan to publish and present additional data that further support that HGF/MET enhancement may play an important role in treating neurodegenerative disease.&#8221;

Tag: HGF/MET pathway

ATH-1105 Aids Nerve Health and Motor Skills in Mice, Trial Possible