AMLX Stock Overview
Amylyx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in developing various therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases.
Amylyx Pharmaceuticals, Inc. Competitors
Price History & Performance
|Historical stock prices|
|Current Share Price||US$28.15|
|52 Week High||US$33.41|
|52 Week Low||US$6.51|
|1 Month Change||11.53%|
|3 Month Change||44.58%|
|1 Year Change||n/a|
|3 Year Change||n/a|
|5 Year Change||n/a|
|Change since IPO||55.78%|
Recent News & Updates
Amylyx ALS Drug Approval: Unexpected, Welcome, And Conditional - May Put Share Price In Stasis
Summary Amylyx ALS drug candidate AMX0035 has been approved in the US - it will be marketed and sold as Relyvrio. The drug has had a controversial path to approval involving 2 separate FDA Advisory Committee meetings. Initially it looked as though the FDA and its AdComm were against approving AMX0035 until the agency surprised the market. The drug could still be withdrawn from sale however if the results of an ongoing Phase 3 study do not support positive data from a smaller Phase 2 study. It is a unique situation and it will be very interesting to see how this plays out. Hopefully, it will be a win for patients, but there remains much negotiating to be done. Investment Thesis I last covered Amylyx (AMLX) for Seeking Alpha earlier this month, just before the FDA's Advisory Committee convened to discuss the upcoming Prescription Drug User Fee Act ("PDUFA") date for the biotech's Amyotrophic Lateral Sclerosis ("ALS") candidate AMX0035. I suggested that, based on the generally negative briefing documents provided by the FDA in advance of the meeting, the chances of a positive outcome were slim, and that in turn reduced the chances of the drug being approved when the PDUFA arrived on September 29th. As it transpires, I could not have been more wrong! AMX0035 has been approved to treat ALS, or Lou Gehrig's Disease, and will now be marketed and sold under the brand name Relyvrio. Amylyx announced this morning. The company's stock price, which rose from $17 to $30 when the second AdComm voted strongly in favor of approval, has not spiked on the approval news, perhaps owing to the fact that this is a conditional approval and Amylyx still needs to deliver positive results from its ongoing PHOENIX Phase 3 study. Klee and Cohen released a statement calling the approval "an exciting milestone for the ALS community" and "a major step toward achieving our mission to one day end the suffering caused by neurodegenerative diseases". Leading ALS organizations also released a statement as follows: With today's approval, we are encouraged that RELYVRIO can offer people living with ALS and their families the potential of more time with functional independence. This is especially important for a rapidly progressive disease with a median survival time from diagnosis of just two to three years. This is significant for people living with ALS, their loved ones, caregivers, clinicians, researchers, and advocacy, as we now have a new treatment option that could be a big step forward for the future of ALS care Journey To Approval - The First Negative AdComm Vote Relyvrio is a combination of 2 different drugs - a dual UPR-Bax apoptosis inhibitor composed of sodium phenylbutyrate ("PB"), and TURSO (also known as tauroursodeoxycholic acid, or TUDCA). The mechanism of action is described in the company's 10Q submissions as follows: Through the resolution of the unfolded protein response, or UPR, and by inhibiting translocation of the Bcl-2 Associated X-protein, or Bax, to the outer mitochondrial membrane, we have shown in multiple models that AMX0035 can keep neurons alive under a variety of different conditions and stresses, including in in vitro models of neurodegeneration, endoplasmic reticulum stress, mitochondrial dysfunction, oxidative stress and disease-specific models of a variety of other conditions, as well as in vivo models of Alzheimer's Disease, or AD, and multiple sclerosis. The drug has been approved based on data from a Phase 2 CENTAUR study of 137 patients with ALS comprised of a 6-month placebo controlled phase, followed by an open label extension follow-up phase. The primary endpoint of CENTAUR was the rate of decline in total Amyotrophic Lateral Sclerosis Functional Rating Scale ("ALSFRS-R") - a 5-minute questionnaire using an ordinal rating scale of 0-4, covering 12 functional activities relevant to ALS across four functional domains. AMX0035 was able to show a statistically significant slowing of ALS disease progression, and a longer duration of survival of 25 months, compared to 18.5 months in the placebo arm. Initially, the FDA requested that Amylyx complete a further Phase 3 trial, but the agency accepted Amylyx' New Drug Application ("NDA") in the last quarter of 2021. An AdComm was convened for March 30th, and the FDA released briefing documents which were somewhat critical of the CENTAUR trial. The agency queried whether the statistical significance of the improvement in ALSFRS-R scale - 2.32 points on a 48-point scale - was significant enough particularly given that no secondary endpoints were met - and also objected to missing data, the use of 2 already approved ALS therapies - Edaravone and Riluzole - in patients during the study, and issues with randomisation, which meant that the first 18 participants in the study were assigned to the AMX0035 arm. The question put before the AdComm was as follows: Do the data from the single randomized, controlled trial and the open-label extension study [Phase 2 CENTAUR trial] establish a conclusion that sodium phenylbutyrate/taurursodiol [AMX0035] is effective in the treatment of patients with amyotrophic lateral sclerosis (ALS)? The AdComm voted 4-6 against. The Second AdComm And The FDA's Volte Face That was by no means the end of the drug candidates journey to approval however. The FDA took the unusual step of convening a second AdComm, in response to fresh data supplied to the agency by Amylyx. Amylyx shared new biomarker data from a Phase 2 study of AMX-0035 in Alzheimer's Disease, supporting the evidence gained from the CENTAUR trial, and also 3 new pieces of analysis, described by the FDA as follows in its second set of briefing documents. A new analysis utilizing a statistical method to adjust for the effect of treatment crossover; A new analysis comparing observed survival in the CENTAUR study to predicted survival using the ENCALS ALS survival prediction model derived from an ALS natural history database; A new analysis comparing observed survival from CENTAUR treatment group to survival of matched treatment naïve participants from historical clinical trials of ALS. The briefing notes then went on to negatively critique the studies and reject the Alzheimer's biomarker data as relevant to the push for approval in ALS. As most observers drew the conclusion that AMX0035 would again fail to convince the AdComm panel, Amylyx share price sank from ~$25, to ~$18. Perhaps the verdict - shared by myself in my last note on Amylyx - should not have been so negative however - after all, the briefing documents concluded as follows: The placebo controlled survival benefit in the intent-to-treat population provides important confirmatory evidence for the clinical benefit of AMX0035 and new analyses, including comparisons to extend controls confirm and strengthen this finding. CENTAUR is a single adequate and well controlled study with confirmatory evidence that meets the appropriate standards for approval especially in the context of a rare and fatal disease such as ALS. Personally I have to admit that I was too swayed by the critical aspects of the briefing notes and should have paid more attention to these final conclusions, but in my defense I was not alone in believing that the second AdComm would go the way of the first. It did not however. During the AdComm, the FDA's Billy Dunn surprised everyone by pointedly asking Amylyx' co-founders Justin Klee and Josh Cohen if they would be prepared to withdraw AMX0035 from the market if data from an ongoing Phase 3 clinical study of the drug failed to support the positive data from a Phase 2 trial that was being presented as evidence. Both co-founders stated that the would do so - Justin Klee stated: To be clear, if [the trial] is not successful, we will do what is right for patients, which includes voluntarily removing the product from the market." That set the tone for a positive outcome, with the AdComm voting 7:2 in favor in answer to a slightly differently worded question: that the available evidence of effectiveness is sufficient to support approval of AMX0035 (sodium phenylbutyrate and taurursodiol [also known as ursodoxicoltaurine]) for the treatment of amyotrophic lateral sclerosis (ALS). It was an outcome that took most people by surprise, although given the lack of treatment options for patients with ALS, with no disease modifying therapies available, plus Relyvrio's relatively clean safety profile - the most common adverse events occurring in the CENTAUR study (at least 15% of patients, and at least 5% greater than placebo) were diarrhea, abdominal pain, nausea, and upper respiratory tract infection, the approval press release states - perhaps there were more reasons for optimism that most people, including myself, believed. What Happens Now? Getting Relyvrio Into The Hands of Patients AMX0035 has already been approved in Canada, where the drug will be marketed and sold as Albioza, although it is not commercially available yet other than for privately paying patients, as pricing negotiations are apparently yet to be completed.
Amylyx Pharma stock surges 81% after ALS drug gets FDA advisers backing
Amylyx Pharmaceutical (NASDAQ:AMLX) is trading 81% higher premarket after a panel of experts to the U.S. Food and Drug Administration voted in favor to recommend approval of the drug, AMX0035, to treat neurodegenerative disease amyotrophic lateral sclerosis (ALS). If gains hold, the stock is set to reverse its YTD losses. In a rare second meeting, the FDA's panel voted 7 in favor and 2 against for the drug. The advisory committee's nod comes after the panel in March rejected the drug due to issues with trial data. The FDA often follows the advisory committee's recommendations, though it is not obligated to. A decision by the FDA is expected by Sept. 29. Shares of Reata Pharmaceuticals (RETA) and Ardelyx (ARDX), fellow clinical-stage biopharma companies that face their own upcoming AdCom meetings, are up ~10% and 2.5% higher, respectively. Citi research analyst Neena Bitritto-Garg thinks the approval is now "very highly likely", particularly due to the political pressure to approve new therapies for ALS, raises price target to $48 from $35.
Amylyx plunges 28% after FDA briefing documents on ALS drug
Amylyx Pharmaceuticals (NASDAQ:AMLX), a company focused on neurodegenerative diseases, lost ~28% in value on Friday after the FDA posted briefing documents ahead of an advisory committee meeting scheduled for next week on its candidate for amyotrophic lateral sclerosis AMX0035. The shares of Reata Pharmaceuticals (RETA), a developer focused on another neurological disease called Friedreich’s ataxia, have also come under pressure in reaction. Sep. 07 AdCom meeting will be the second review the FDA’s independent experts will conduct on the drug after a negative vote in March. However, the FDA extended the review period of AMLX’s new drug application (NDA) for AMX0035 by three months to analyze additional data extending the PDUFA date to Seep. 29 from the original date of Jun. 29. This is a developing story. Check back for more updates.
|AMLX||US Pharmaceuticals||US Market|
Return vs Industry: Insufficient data to determine how AMLX performed against the US Pharmaceuticals industry.
Return vs Market: Insufficient data to determine how AMLX performed against the US Market.
|AMLX Average Weekly Movement||18.2%|
|Pharmaceuticals Industry Average Movement||11.3%|
|Market Average Movement||6.9%|
|10% most volatile stocks in US Market||15.7%|
|10% least volatile stocks in US Market||2.8%|
Stable Share Price: AMLX is more volatile than 90% of US stocks over the past 3 months, typically moving +/- 18% a week.
Volatility Over Time: AMLX's weekly volatility (18%) has been stable over the past year, but is still higher than 75% of US stocks.
About the Company
Amylyx Pharmaceuticals, Inc., a clinical-stage biopharmaceutical company, engages in developing various therapeutics for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases. The company’s product pipeline includes AMX0035, a dual UPR-Bax apoptosis inhibitor composed of sodium phenylbutyrate and taurursodiol for the treatment of amyotrophic lateral sclerosis. It is also developing AMX0035 for other neurodegenerative diseases.
Amylyx Pharmaceuticals, Inc. Fundamentals Summary
|AMLX fundamental statistics|
Is AMLX overvalued?See Fair Value and valuation analysis
Earnings & Revenue
|AMLX income statement (TTM)|
|Cost of Revenue||US$71.97m|
Last Reported Earnings
Jun 30, 2022
Next Earnings Date
|Earnings per share (EPS)||-2.62|
|Net Profit Margin||-53,824.91%|
How did AMLX perform over the long term?See historical performance and comparison