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SAN DIEGO, Oct. 17, 2024 /PRNewswire/ -- Amplo Biotechnology, a biotechnology company developing genetic medicines for the treatment of neuromuscular diseases, announced the presentation of three posters at the European Society of Gene and Cell Therapy (ESGCT) 31st Annual Congress, taking place from October 22-25, 2024, in Rome, Italy. Amplo will feature three poster presentations detailing its platform approach to developing programmatic gene therapies, new preclinical data for Dok-7 gene therapy (AMP-101), and lead candidate activities for its novel Collagen Q therapy (AMP-201). "We are pleased to present data at ESGCT demonstrating the potential of our lean drug development strategy that supports the commercial viability of gene therapies in rare- and super-rare diseases by leveraging the ecosystem's knowledge," said Patricio Sepulveda, PhD MBA, Chief Executive Officer at Amplo Biotechnology. Details for the poster presentations: Abstract Title: Development of a platform gene therapy approach for Congenital Myasthenic Syndromes Presenting Author: Patricio V. Sepulveda S., PhD MBA Date: Tuesday, October 22, 2024 Abstract Title: Pre-clinical development of gene augmentation therapy to restore Acetylcholinesterase activity in Collagen Q Congenital Myasthenic Syndrome using a platform approach to AAV gene therapy development. Presenting Author: Claudia Canzonetta, PhD Date: Tuesday, October 22, 2024 Abstract Title: The pre-clinical development of gene augmentation therapy to stabilize the neuromuscular junction in Dok-7 Congenital Myasthenic Syndrome reveals a mechanism of action that has the potential to treat many neuromuscular junction diseases using the same vector. Presenting Author: Paul Al Hawkins Date: Wednesday, October 23, 2024 About Amplo Biotechnology Amplo is a late, pre-clinical genetic medicines company focused on therapies for rare and super-rare diseases. SOURCE Amplo Biotechnology WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Athira said ADAS-Cog11 and ADCS-ADL23 “directionally favored fosgonimeton treatment.” Athira Pharma’s tumultuous journey to phase 2/3 Alzheimer’s disease data has ended in failure. The trial found fosgonimeton was no better than placebo on the primary and key secondary endpoints, driving investors to cut the cord holding up the biotech’s stock price. Washington-based Athira burst onto the scene in 2020, raising an $85 million series B round and pulling off a $204 million in quick succession. The honeymoon was short-lived. In 2021, Leen Kawas, then the CEO of Athira, was accused of altering images in multiple papers. Kawas was gone later that year. In 2022 and 2023, the biotech reported phase 2 failures in Alzheimer’s and dementia.The phase 2/3 study offered Athira a chance to radically improve perceptions of fosgonimeton. The trial didn’t deliver that boost. Rather, the results sent Athira’s share price down 73% to $0.76 in premarket trading, well below the slump following the Kawas scandal, let alone the post-IPO high of over $30.Investors fled the stock after Athira failed to make the case that fosgonimeton can improve outcomes in Alzheimer’s by modulating HGF to promote neuron proliferation and survival. Athira designed the trial to compare the effect of 26 weeks of once-daily subcutaneous fosgonimeton injections to placebo in 312 people with mild to moderate Alzheimer’s who weren’t taking acetylcholinesterase inhibitors. Fosgonimeton was no better than placebo on the Global Statistical Test, a measure that combined scores on assessments of cognition, ADAS-Cog11, and function, ADCS-ADL23. The result caused the trial to miss its primary endpoint. Fosgonimeton failed to beat placebo on ADAS-Cog11 or ADCS-ADL23.However, Athira said ADAS-Cog11 and ADCS-ADL23 “directionally favored fosgonimeton treatment,” with a nonsignificant difference of 0.70 on the cognition scale and 0.67 on the function score. The divergence between the treatment and placebo groups was most pronounced in prespecified subgroups of patients who were expected to progress faster, namely those with moderate disease and APOE4 carriers.Javier San Martin, M.D., chief medical officer at Athira, said in a statement that “the lack of clinical decline in the placebo group, combined with the short duration of the study, may have impacted the trial’s ability to translate the effect of fosgonimeton.” Talking on a webinar in June, Athira CEO Mark Litton said historically ADAS-Cog11 and ADCS-ADL23 scores decline about two points in six months.The lack of that level of decline in the placebo cohort led Athira to run post hoc analyses in patients with worse cognition at baseline for signs of efficacy. Athira argued the totality of the data suggests positive modulation of HGF signaling may work in neurodegenerative diseases. The validity of that position has implications for ATH-1105, a HGF modulator Athira is developing in amyotrophic lateral sclerosis.

A daily injection intended to protect and regenerate brain cells has failed to slow the progression of Alzheimer’s disease in a Phase 2/3 clinical trial run by Athira Pharma, the company announced Tuesday. The drug, fosgonimeton, didn’t meet any of the company’s primary or secondary endpoints in a 26-week study of 549 patients with mild-to-moderate dementia. Plans for a larger follow-up study have been scrapped. “We’re just assessing what is the best way to move forward with this biology,” CEO Mark Litton told Endpoints News in an interview. The Bothell, WA-based biotech had about $75 million at the end of August, enough to keep it running until the end of 2025 “under certain scenarios,” Litton said. The company had a valuation of about $108 million at market close on Tuesday, but its stock $ATHA fell 75% after hours. While most Alzheimer’s drugs have focused on reducing amyloid or tau, the two proteins most strongly linked to dementia, Athira was working on a different protein called hepatocyte growth factor, or HGF. The protein was discovered in the liver but was later found to be vital for neuronal development and survival. Athira’s drug binds to HGF receptors and enhances the growth factor’s activity. In preclinical studies, fosgonimeton showed that it helps neurons make more connections and may even protect brain cells from injury. It also shuts down inflammation and enhances autophagy, a cellular housecleaning process that removes bad proteins like amyloid and tau. Replicating those benefits in people has proven more challenging. The drug already failed a previous clinical test, which the company blamed on the fact that many patients were taking existing Alzheimer’s drugs called acetylcholinesterase inhibitors, even though most doctors believe the drugs only have marginal effects on symptoms of the disease. While Athira enrolled nearly 550 patients in its new study, Tuesday’s results are based on data from just 287 participants. The company excluded people who were on the other Alzheimer’s drugs that foiled its earlier study. It also excluded patients who got the larger of two doses, which it stopped testing due to more frequent injection site reactions — the drug’s most prevalent side effect. The drug was no better than placebo at slowing the disease, as measured by a cognitive test called the ADAS-Cog11 and a functional test called ADCS-ADL23. The drug also failed to significantly change most key biomarkers including neurofilament and amyloid beta, but did lower a form of tau increasingly linked to Alzheimer’s disease. The company said that the drug had slightly bigger effects in subgroups of people with moderate dementia and in people carrying at least one copy of the APOE4 gene linked to the disease. But in all cases, the outcomes between those who got the drug and those who got a placebo were not statistically significant. Despite the trial’s failure, the company isn’t ready to give up on targeting HGF just yet. It’s already testing an oral alternative to fosgonimeton, ATH-1105, in healthy volunteers, with plans to test the compound in ALS patients in 2025. Litton said that it could potentially be tested in Alzheimer’s patients too. “This is the first proof of concept with enhancing HGF, and we truly believe that it’s neuroprotective and should play a role in helping diseases with neurodegeneration,” Litton said.