Latest news with #AmylyxPharmaceuticalsInc
Yahoo
a day ago
- Business
- Yahoo
Amylyx Pharmaceuticals Gains FDA Fast Track for ALS Drug AMX0114
Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) is one of the best biotech stocks to invest in now. On June 3, Amylyx Pharmaceuticals announced that the US FDA granted Fast Track designation to AMX0114. AMX0114 is an investigational antisense oligonucleotide/ASO that is specifically designed to target calpain-2. This protein is believed to be a significant contributor to axonal degeneration, which is a key driver in the progression of amyotrophic lateral sclerosis/ALS. ALS is a rapidly progressive and fatal neurodegenerative disorder with limited therapeutic options. The Fast Track designation is a crucial step for Amylyx in its mission to develop treatments for ALS. The FDA's decision acknowledges both the severity of ALS and the encouraging preclinical data supporting AMX0114's potential. A medical scientist in a lab coat gazing at a microscopic view of a drug in development. Calpain-2 is a calcium-activated protease. Research has indicated that overactive calpain-2 activity might accelerate disease progression in ALS and other neurodegenerative conditions by causing the degeneration of axons, which are vital for transmitting signals between neurons and muscles. Preclinical studies of AMX0114 have demonstrated improved neuronal survival and reduced levels of extracellular neurofilament light (NfL), which is a biomarker for neuronal damage across various disease models. AMX0114 was also well-tolerated in preclinical safety studies. Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) is a clinical-stage pharmaceutical company that discovers and develops treatments for neurodegenerative diseases and endocrine conditions in the US. While we acknowledge the potential of AMLX as an investment, we believe certain AI stocks offer greater upside potential and carry less downside risk. If you're looking for an extremely undervalued AI stock that also stands to benefit significantly from Trump-era tariffs and the onshoring trend, see our free report on the . READ NEXT: and . Disclosure: None. This article is originally published at Insider Monkey.
Yahoo
16-04-2025
- Business
- Yahoo
Is Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) the Best Biotech Penny Stock to Buy According to Hedge Funds?
We recently published a list of the . In this article, we are going to take a look at where Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) stands against other biotech penny stocks. Peter Marks's departure from the FDA has left innovators concerned regarding the current state of the review program at the FDA. Marks had impacts on two key areas. First the vaccine approvals and the subsequent resurgence of new vaccines over the past two decades, and then the establishment of the cell and gene therapy field that he's credited for. He was involved in developing policies aimed at optimizing the process of bringing several innovative products to the market. Following his resignation, a substantial number of other departures are also anticipated, which implies upcoming destabilization at the FDA for its existing expertise and processes. Stifel head of biotech research Paul Matteis suggests looking for biotech names not dependent on the FDA while this sector uncertainty lasts. On March 31, Paul Matteis appeared on CNBC's 'The Exchange' and noted that biotech investors, particularly those who are focused on developmental-stage companies, are now facing risk. These include scientific, clinical trials, commercial, patent, competition, and regulatory risks. Hence, investors now need to be more selective about their stock selections. Previously, the FDA has been a stabilizing force for the biotech sector and is known for accelerating approvals in areas such as rare diseases and biomarkers. But recent developments have introduced unpredictability. Matteis highlighted that there could be short-term delays in drug approvals due to personnel changes at the FDA, such as the recent departure of the head of the Center for Drug Evaluation and Research (CEDAR). Investors may even need to reevaluate agreements between companies and the FDA regarding drug approvals. While this doesn't make biotech investments completely unsustainable, it does shift the focus toward companies with more predictable growth prospects. This could point to specifically commercial-stage firms or those less reliant on FDA consistency. Talking about vaccine stocks particularly, Matteis pointed out that as much as Peter Marks influenced vaccine-related decisions, he also impacted broader areas like gene therapies and oncology. He fostered innovation through programs like breakthrough therapy designations and initiatives that were aimed at streamlining approvals. Investors are now gravitating toward companies with de-risked profiles that are less dependent on FDA flexibility. Matteis noted that the companies relatively outperforming right now are those that have their success tied to commercial launches rather than regulatory decisions. We sifted through ETFs, stock screeners, and financial media reports to compile a list of the top biotech penny stocks that were trading below $5 as of April 14. We then selected the 13 stocks that were the most popular among elite hedge funds and that analysts were bullish on. The stocks are ranked in ascending order of the number of hedge funds that have stakes in them, as of Q4 2024. The hedge fund data was sourced from Insider Monkey's database which tracks the moves of over 1000 elite money managers. Why are we interested in the stocks that hedge funds pile into? The reason is simple: our research has shown that we can outperform the market by imitating the top stock picks of the best hedge funds. Our quarterly newsletter's strategy selects 14 small-cap and large-cap stocks every quarter and has returned 373.4% since May 2014, beating its benchmark by 218 percentage points (see more details here). A medical scientist in a lab coat gazing at a microscopic view of a drug in development. Share Price as of April 14: $3.65 Number of Hedge Fund Holders: 30 Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) discovers and develops treatments for neurodegenerative diseases and endocrine conditions. Its lead product candidates include avexitide, which is an investigational, first-in-class glucagon-like peptide-1; and GLP-1 receptor antagonist, which is in phase 3 clinical trial to treat post-bariatric hypoglycemia and congenital hyperinsulinism. The company is focusing on potentially making avexitide the first approved treatment for post-bariatric hypoglycemia (PBH). Currently, there are no FDA-approved therapies specifically for PBH. This is a significant unmet medical need that impacts ~160,000 individuals in the US alone. These patients suffer from persistent low blood sugar after bariatric surgery. Across five clinical trials in PBH patients, avexitide demonstrated a dose-dependent and statistically significant reduction in hypoglycemic events. Amylyx Pharmaceuticals Inc. (NASDAQ:AMLX) initiated a Phase III clinical trial called LUCIDITY. This is a 16-week study with ~75 participants with topline results anticipated in H1 2026. For the potential launch of avexitide, the company secured ~$65.5 million in financing. If LUCIDITY yields positive results and regulatory approval is granted, Amylyx expects a commercial launch of avexitide in 2027. Overall, AMLX ranks 8th on our list of the best biotech penny stocks to buy according to hedge funds. While we acknowledge the growth potential of AMLX, our conviction lies in the belief that AI stocks hold great promise for delivering high returns and doing so within a shorter time frame. There is an AI stock that went up since the beginning of 2025, while popular AI stocks lost around 25%. If you are looking for an AI stock that is more promising than AMLX but that trades at less than 5 times its earnings, check out our report about the cheapest AI stock. READ NEXT: 20 Best AI Stocks To Buy Now and 30 Best Stocks to Buy Now According to Billionaires. Disclosure: None. This article is originally published at Insider Monkey. Sign in to access your portfolio
