A SUPER-powered WP Engine Atlas Test Site

Joe&#039;s Atlas Experimentation Site

While the now-discontinued <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) therapy <a href="https://alsnewstoday.com/amx0035">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) failed to show a meaningful effect on disease progression, lung function, or life quality in the Phase 3 PHOENIX clinical trial, ongoing analyses will look at other outcomes such as biomarkers and survival.
That’s according to a presentation from one of the trial’s primary investigators, Leonard H. Van den Berg, MD, PhD, given at the American Academy of Neurology (AAN) 2024 Annual Meeting. The AAN meeting is being held April 13-18, in Denver, and virtually.
“PHOENIX was a well-powered and a well-executed trial,” Van den Berg said in the presentation, titled “Results From a Global Phase 3 Trial Evaluating an Oral, Fixed-Dose Combination of Sodium Phenylbutyrate and Taurursodiol in Amyotrophic Lateral Sclerosis.”
“We will continue to learn from PHOENIX to inform future ALS trials,” Van den Berg said.
Relyvrio&#8217;s developer <a href="https://www.amylyx.com/">Amylyx Pharmaceuticals</a> voluntarily <a href="https://alsnewstoday.com/news/relyvrio-withdrawn-from-us-canada-after-phase-3-trial-results/">took the therapy off the market</a> in the U.S. and Canada — where it was marketed as Albrioza — earlier this month after the company first announced that PHOENIX had <a href="https://alsnewstoday.com/news/als-treatment-relyvrio-fails-trial-may-be-withdrawn-market/">failed to meet its main goal</a>.
The medication no longer is available to new patients in either country. However, patients already on Relyvrio as of the April 4 withdrawal date who wish to remain on treatment may be able to transition to a free drug program in consultation with their doctor.
[recommended-reading id=88465]
<h3>PHOENIX trial was intended to confirm positive results from CENTAUR</h3>
Formerly known as AMX0035, Relyvrio is a fixed-dose oral combination of sodium phenylbutyrate and taurursodiol. The two medications are believed to work together to prevent nerve cell death in ALS.
Relyvrio was approved in both the <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">U.S.</a> and <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">Canada</a> in 2022 based on findings from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">Phase 2 CENTAUR study (NCT03127514)</a>, which involved adults with recently diagnosed and rapidly progressing ALS.
In CENTAUR, the oral medication was associated with <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slower functional declines</a> compared with a placebo, with evidence that it may also <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">extend survival</a>.
Still, experts were not altogether convinced of the treatment’s efficacy for ALS patients. Indeed, the European Medicines Agency <a href="https://alsnewstoday.com/news/chmp-confirms-initial-recommendation-not-approve-amx0035-eu/">voted against</a> the approval of Relyvrio in the European Union based on data from CENTAUR alone. The treatment’s clearance in Canada also was conditional, meaning regulators wanted to see more clinical trial data before converting to a full approval.
<a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a> was the Phase 3 trial intended to confirm Relyvrio’s safety and effectiveness in a larger group of patients across the U.S. and Europe. A total of 664 people with definite or probable ALS, who were within two years of symptom onset, were enrolled and randomly assigned to receive oral Relyvrio or a placebo for 48 weeks, or about a year.
The primary goal was to confirm the main finding from CENTAUR — that Relyvrio significantly slowed functional declines, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), compared with a placebo.
According to Van den Berg’s presentation, the demographic and clinical characteristics of participants in the Relyvrio and placebo groups were well-balanced. In both groups, the average age was about 60, most patients were men (60%-63%), and nearly all (91%-93%) were taking a stable dose of riluzole (sold as <a href="https://alsnewstoday.com/als-treatment/rilutek-riluzole/">Rilutek</a> among others) upon entering the trial. There also were 3% of patients in each group receiving edaravone, which is approved as <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava or Radicava ORS</a>.
Ultimately, however, the results of the trial&#8217;s primary outcome measure showed that ALSFRS-R scores dropped by an average of 14.98 points in the Relyvrio group and by 15.32 points in the placebo group — a non-significant difference.
Likewise, there was no significant benefit of Relyvrio in pre-specified subgroups, including patients who were not concurrently using edaravone, and patients enrolled only at European sites.
[recommended-reading id=87417]
<h3>Further survival data on Relyvrio likely not available before 2026</h3>
The researchers also looked at a subgroup of “CENTAUR-like” patients, or those who matched the enrollment criteria for the earlier Phase 2 trial. Specifically, those patients had definite ALS and greater than 60% of normal lung function. These individuals also were within 1.5 years of symptom onset.
While those patients did “somewhat better” in terms of disease progression when given Relyvrio, Van den Berg noted that the PHOENIX findings still failed to reach statistical significance in comparison to the placebo in that subgroup.
Secondary goals related to life quality and lung function also were not met.
Overall, despite “promising results” from CENTAUR, “there were no differences between the groups for the primary endpoints and the secondary endpoints in the Phase 3 study,&#8221; Van den Berg concluded.
In terms of survival, the scientist said there&#8217;s still not enough data to adequately evaluate Relyvrio’s effects. An open-label extension phase of the trial is still ongoing, in which participants will receive Relyvrio for up to about two more years. However, Van den Berg said mature survival data may not be available before February 2026.
“We will continue to collect survival data, as survival is an important outcome measure in ALS trials,” Van den Berg said.
[quote=&#8221;center&#8221;]As a community, we all work together to find better treatments for our patients, and we will do that even harder after these results.[/quote]
Other analyses also are still ongoing, including biomarker and subgroup analyses, particularly within the CENTAUR-like population.
Relyvrio was generally well tolerated, with safety findings consistent with the CENTAUR trial. Only diarrhea was more common with Relyvrio versus the placebo.
Van den Berg extended a thank you to PHOENIX participants, investigators, and clinical sites.
“As a community, we all work together to find better treatments for our patients, and we will do that even harder after these results,” the scientist concluded.

AAN 2024: Trial investigator shares details from Relyvrio failure in ALS

<a href="https://alsnewstoday.com/amx0035">Relyvrio</a> (sodium phenylbutyrate and taurursodiol), an approved treatment for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), was voluntarily removed from the U.S. and Canadian markets.
<a href="https://www.amylyx.eu/">Amylyx Pharmaceuticals</a>, the therapy&#8217;s developer, made the decision after <a href="https://alsnewstoday.com/news/als-treatment-relyvrio-fails-trial-may-be-withdrawn-market/">top-line results</a> from the <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">Phase 3 PHOENIX trial (NCT05021536)</a> showed that patients who received Relyvrio as an add-on to standard ALS medications experienced a similar rate of disease progression as those given a standard treatment alone.
As of April 4, Relyvrio is no longer available for new patients. Patients receiving the therapy in the U.S. and in Canada, where it was marketed under the brand name Albrioza, who want to continue on treatment can transition to a free drug program after consulting with their physicians.
&#8220;While this is a difficult moment for the ALS community, we reached this path forward in partnership with the stakeholders who will be impacted and in line with our steadfast commitment to people living with ALS and other neurodegenerative diseases,&#8221; said Joshua Cohen and Justin Klee, co-CEOs of Amylyx, in a <a href="https://www.businesswire.com/news/home/20240404501040/en/Amylyx-Pharmaceuticals-Announces-Formal-Intention-to-Remove-RELYVRIOALBRIOZA-from-the-Market-Provides-Updates-on-Access-to-Therapy-Pipeline-Corporate-Restructuring-and-Strategy">company press release</a>.
&#8220;The decision to remove Relyvrio/Albrioza from the market and provide therapy free of charge for those who wish to continue was informed by the PHOENIX trial results, engagement with regulatory authorities, and discussions with the ALS community,&#8221; Cohen and Klee said. &#8220;Thank you to each and every person who shared feedback with us and continues to support our commitment to the ALS community.&#8221;
[recommended-reading id=90368]
<h3>Phase 2 results suggested slowing of functional decline</h3>
Relyvrio, formerly known as AMX0035, <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">was approved</a> in the U.S. and Canada in 2022 based on data from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">Phase 2 CENTAUR study (NCT03127514)</a>. The study enrolled 137 adults with recently diagnosed, rapidly progressing ALS.
After six months, there was <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">significantly less decline in physical function</a>, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), among adults given Relyvrio than those on a placebo. Data also suggested the therapy may <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">extend survival</a>.
Still, an advisory committee with the U.S. Food and Drug Administration <a href="https://alsnewstoday.com/news/fda-committee-votes-against-recommending-als-therapy-amx0035/">initially voted</a> there was not enough substantial evidence to support Relyvrio’s efficacy, and European authorities also <a href="http://recommended">recommended against approval</a> based on these data alone.
Although the treatment was ultimately approved in the U.S. based on the early data, the company said it would consider withdrawing the marketing application if Relyvrio failed to prove beneficial in a confirmatory Phase 3 trial.
The PHOENIX trial was launched to confirm Relyvrio&#8217;s effectiveness over one year, enrolling 664 people with early ALS. However, top-line data showed the trial failed to meet its primary goal of slowing the decline in ALSFRS-R scores compared with a placebo. These results were a key factor in the decision to remove Relyvrio from the market.
Top-line PHOENIX data will be presented at the American Academy of Neurology (AAN) annual meeting this month and will be available on <a href="https://www.amylyx.com/publications">Amylyx&#8217;s website</a> following the presentation.
[recommended-reading id=90100]
<h3>PHOENIX results to inform future research</h3>
The company will evaluate and share PHOENIX&#8217;s findings to help inform future ALS research and collect survival data at the encouragement of ALS specialists. The <a href="https://classic.clinicaltrials.gov/ct2/show/NCT05619783">PHOENIX open-label extension study (NCT05619783)</a> in Europe is ongoing.
Relyvrio also is being evaluated as a potential treatment for other neurodegenerative conditions, including Wolfram syndrome and progressive supranuclear palsy.
Amylyx will continue to advance its experimental ALS treatment AMX0114, which targets calpain-2, a protein involved in nerve fiber degeneration. In preclinical studies, AMX0114 achieved a dose-dependent reduction of calpain-2 protein levels in human motor neurons, the nerve cells affected in ALS. The company <a href="https://alsnewstoday.com/news/als-mnd-2023-clinical-tests-aso-treatment-als-happen-next-year/">expects to launch a clinical trial</a> to test AMX0114 in ALS later this year.
&#8220;We also remain focused on ALS and believe AMX0114 has strong potential for the treatment of ALS and other diseases,&#8221; said Camille L. Bedrosian, MD, chief medical officer of Amylyx. &#8220;Calpain-2 is considered an essential protein in the process of axonal degeneration.&#8221;
&#8220;In our preclinical studies of AMX0114 and in multiple independent published studies, inhibition of calpain-2 has reduced cell death and degeneration,&#8221; Bedrosian said. &#8220;We expect to initiate a clinical trial studying AMX0114 in ALS in the second half of this year.&#8221;

Relyvrio withdrawn from US, Canada after Phase 3 trial results

<a href="https://alsnewstoday.com/amx0035">Relyvrio</a> (sodium phenylbutyrate and taurursodiol), an approved treatment for people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), has failed to meet its primary goal of slowing disease progression in an ongoing Phase 3 clinical trial designed to confirm its effectiveness.
That&#8217;s according to new data from the trial, dubbed PHOENIX, released today by the therapy&#8217;s developer, <a href="https://www.amylyx.com/">Amylyx Pharmaceuticals</a>.
Essentially, the data show that patients who received Relyvrio in addition to their standard ALS medications in PHOENIX experienced a similar rate of disease progression as did those given a standard treatment alone.
Based on the data, Amylyx is voluntarily pausing promotion of Relyvrio, and said it will work with regulatory authorities and the broader ALS community to determine the appropriate next steps for the therapy — which may include voluntarily withdrawing it from the market, according to a <a href="https://www.businesswire.com/news/home/20240308462671/en/Amylyx-Pharmaceuticals-Announces-Topline-Results-From-Global-Phase-3-PHOENIX-Trial-of-AMX0035-in-ALS">company press release</a>.
Until then, the therapy will remain available to ALS patients in the U.S. and in Canada, where it&#8217;s marketed under the brand name Albrioza. The company also will continue running its support programs to help patients on the medication, formerly known as AMX0035 for ALS, which has been <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approved since 2022</a> in both countries.
[quote=&#8221;center&#8221;]Over the next eight weeks, our team will continue to engage with regulatory authorities and the ALS community to discuss the results from PHOENIX.[/quote]
Justin Klee and Joshua Cohen, co-CEOs of Amylyx, said they were &#8220;surprised and deeply disappointed by the PHOENIX results.&#8221;
&#8220;Our main priority at the moment is sharing the information with people living with ALS and their treating physicians; this is part of our continued commitment to them and our mission. Over the next eight weeks, our team will continue to engage with regulatory authorities and the ALS community to discuss the results from PHOENIX,&#8221; Klee and Cohen said.
The CEOs said their discussions with regulators will be informed by &#8220;two key principles: doing what is right for people living with ALS, informed by regulatory authorities and the ALS community, and by what the science tells us.&#8221;
[recommended-reading id=87478]
<h3>Regulators in Europe had recommended against therapy&#8217;s approval</h3>
Relyvrio was approved in the U.S. and Canada based mainly on data from a Phase 2 study called <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR (NCT03127514)</a>. It tested the therapy against a placebo in 137 adults with recently-diagnosed, rapidly progressing ALS.
The results showed that, after six months, patients given Relyvrio had <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">significantly less decline in physical function</a> than those on a placebo<a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">,</a> as measured by scores on the ALS Functional Rating Scale-Revised (ALSFRS-R).
Data from the Phase 2 study even hinted Relyvrio <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">might extend survival</a> for ALS patients. The robustness of the results were questioned, however, with authorities in Europe recommending against approval based on data from the Phase 2 study alone.
The <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">Phase 3 PHOENIX trial (NCT05021536)</a> was launched with the hope of definitively confirming that Relyvrio was effective as an ALS treatment. The study enrolled <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">664 people with early ALS</a> who were randomly assigned to take Relyvrio or a placebo for about one year.
Because the Phase 3 trial included more patients and followed them for a longer period of time, it was expected that the results would be statistically more robust than in the smaller, shorter Phase 2 study.
The main goal of the PHOENIX trial was to show that patients given Relyvrio had less decline in ALSFRS-R scores, just as was seen in the Phase 2 CENTAUR study. However, the results showed that the Phase 3 trial failed to meet this goal: Rates of ALSFRS-R decline were not significantly different for patients given the therapy or the placebo.
[recommended-reading id=89981]
<h3>PHOENIX trial tested Relyvrio vs. a placebo in over 650 ALS patients</h3>
According to Amylyx, Relyvrio also failed to outperform the placebo across several secondary endpoints, or trial goals, which included patient-reported quality of life, assessments of lung function, and overall survival.
Still, Relyvrio was generally well tolerated in PHOENIX, and no unexpected safety issues were reported in the trial.
Full results from the Phase 3 trial are expected to be presented at an upcoming meeting and published in detail later this year. The company also is continuing to run an extension to the study in which European participants who completed PHOENIX could continue to receive Relyvrio.
The Amylyx CEOs thanked all those who took part in the therapy&#8217;s development, regardless of this trial&#8217;s outcome.
&#8220;On behalf of the entire Amylyx team, we are grateful to the ALS community and for the dedication of trial participants, investigators, and study site teams,&#8221; Klee and Cohen said.
&#8220;With data collected from 664 participants in PHOENIX, we are certain there will be important learnings that will help inform future ALS research,&#8221; they added.
While the next step for Relyvrio in ALS remain to be determined, the therapy will continue to be developed for other neurological disorders. Amylyx also will continue to advance its experimental ALS treatment <a href="https://alsnewstoday.com/news/als-mnd-2023-clinical-tests-aso-treatment-als-happen-next-year/">AMX0114</a>, which targets the calpain-2 protein.
&#8220;We are steadfast in our commitment to the ALS community and our mission, including with AMX0035 where it has shown potential in neurodegenerative diseases such as Wolfram syndrome and progressive supranuclear palsy, and with AMX0114, our investigational antisense oligonucleotide targeting calpain-2, in ALS,&#8221; Klee and Cohen said.

ALS treatment Relyvrio fails trial, may be withdrawn from market

Throughout 2023, <a href="https://alsnewstoday.com/">ALS News Today</a> brought you daily coverage of the latest clinical research and scientific breakthroughs related to <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
Here are the year&#8217;s top 10 most-read articles, each with a brief description. We&#8217;re excited to remain a dependable resource for the ALS community in 2024.
<h3>No. 10 – Omega-3 fatty acid slows ALS progression, extends survival</h3>
A diet rich in omega-3 fatty acids has been tied to a lower risk of developing ALS, but whether it also might affect the rate of disease progression is not known. Researchers at Harvard found that <a href="https://alsnewstoday.com/news/alpha-linolenic-acid-slower-als-progression-better-survival/">higher blood levels of alpha-linolenic acid</a> (ALA) — an omega-3 fatty acid found in flaxseed, walnuts, chia, canola, and soybean oils — linked with a slower disease progression and a 50% lower risk of death.
Two other fatty acids also were tied to a lower mortality risk. This discovery suggests that adding to the diet more foods containing these fatty acids could be beneficial for ALS patients. Researchers hope to confirm these findings in a clinical trial.
<h3>No. 9 – Further questions set back European approval of AMX0035</h3>
Amylyx Pharmaceuticals&#8217; application seeking the approval of AMX0035 in treating ALS patients across Europe faced <a href="https://alsnewstoday.com/news/european-approval-amx0035-als-faces-new-hurdle-review/">a second round of questions</a> from the Committee for Medicinal Products for Human Use (CHMP), an arm of the European Medicines Agency. The committee still had major objections to AMX0035 that needed to be addressed, after a scientific advisory group meeting of experts provided recommendations based on current data.
The therapy is approved in the U.S. and available under the brand name <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> (sodium phenylbutyrate and taurursodiol), and approved in Canada as Albrioza. CHMP,  however, later confirmed that it <a href="https://alsnewstoday.com/news/chmp-confirms-initial-recommendation-not-approve-amx0035-eu/">remained opposed to AMX0035&#8217;s approval in Europe</a>.
<h3>No. 8 – Masitinib as add-on therapy seen to benefit mild to moderate ALS</h3>
<a href="https://alsnewstoday.com/masitinib/">Masitinib</a> is an experimental ALS therapy that intends to slow disease progression and ease <a href="https://alsnewstoday.com/symptoms-of-als/">ALS symptoms</a> by blocking certain enzymes needed by immune cells thought to drive inflammation and neurodegeneration in ALS. An analysis of AB Science&#8217;s completed <a href="https://clinicaltrials.gov/ct2/show/NCT02588677">AB10015 Phase 2/3 clinical trial (NCT02588677)</a> showed that treatment with masitinib as an add-on to <a href="https://alsnewstoday.com/rilutek-riluzole-for-als/">Rilutek</a> (riluzole) benefited most of its <a href="https://alsnewstoday.com/news/als-therapy-masitinib-works-best-add-on-moderate-disease-analysis/">ALS patients with mild to moderate disease severity</a>.
Although the 48-week study failed to show better survival rates overall with masitinib compared with a placebo, treated patients with moderate ALS experienced a 25-month extension in survival. The combination treatment also significantly slowed disease progression, improved lung function, and enhanced life quality for patients who still retained some degree of physical function at the study&#8217;s start.
<h3>No. 7 – Stem cell transplants seen to extend survival by 4 years</h3>
A stem cell transplant using mesenchymal stromal cells (MSCs) taken from bone marrow <a href="https://alsnewstoday.com/news/msc-stem-cell-transplants-extend-survival-4-years-als-patients/">significantly extended survival for ALS patients</a> beyond their anticipated lifespan, a new analysis of data from two Phase 1 trials, both conducted in the early 2000s, showed.
Treated patients lived about 118 months, or nearly 10 years, without needing permanent breathing support, surpassing a predicted survival time of around 70 months by about four years. At the time of the analysis, four of the trials&#8217; 19 total patients were alive, including one still living without any need for breathing or feeding assistance at 303 months — more than 20 years later.
<h3>No. 6 – Neurologists frustrated with current ALS treatments</h3>
Neurologists, while quick to prescribe their patients new treatments showing a potential to delay ALS progression in clinical trials, often <a href="https://alsnewstoday.com/news/neurologists-frustrated-with-als-treatments-now-on-market-study/">remain frustrated with current available therapies</a>. Fewer than 20% of these doctors responding to a survey expressed full satisfaction with responses to treatments now on the market.
This so-called first market landscape study on ALS, conducted by Spherix Global Insights, involved 103 neurologists. It found that many already were prescribing Relyvrio and <a href="https://alsnewstoday.com/ionis-sod1-rx-biib067/">Qalsody</a> (tofersen), two ALS medications recently approved in the U.S. Spherix data also showed that neurologists lacked awareness of <a href="https://alsnewstoday.com/experimental-treatments-for-als/">experimental ALS treatments</a>, possibly because many such therapies fail to win approval.
<h3>No. 5 – COYA 302 seen to slow ALS progression in proof-of-concept trial</h3>
COYA 302, an experimental immune-modulating therapy under development by Coya Therapeutics, markedly <a href="https://alsnewstoday.com/news/mda-2023-coya-302-therapy-slows-als-progression-early-trial/">slowed disease progression among four ALS patients</a> treated in a small, 48-week proof-of-concept clinical trial. Data also showed a treatment-related increase in the activity of <a href="https://alsnewstoday.com/tregs/">regulatory T-cells</a>, an anti-inflammatory immune cell, demonstrating that the therapy worked as designed.
Coya intends to open larger ALS trials of the therapy, administered via a subcutaneous (under-the-skin) injection. The company announced plans to file early this year a request for clearance to <a href="https://alsnewstoday.com/news/coya-dr-reddys-collaborate-advance-coya-302-als-therapy-globally/">launch a Phase 2 clinical study</a>.
<h3>No. 4 – Tofersen, now Qalsody, approved by the FDA for SOD1-ALS</h3>
In April, the <a href="https://alsnewstoday.com/news/fda-approves-tofersen-now-qalsody-treatment-adults-sod1-als/">U.S. Food and Drug Administration conditionally approved</a> Biogen&#8217;s Qalsody (tofersen) for treating ALS linked to SOD1 gene mutations. The decision marked the country&#8217;s first conditional approval for an ALS therapy.
Qalsody&#8217;s approval relied on biomarker data from the <a href="https://clinicaltrials.gov/ct2/show/NCT02623699">VALOR Phase 1/2/3 clinical trial (NCT02623699)</a> and its <a href="https://clinicaltrials.gov/ct2/show/NCT03070119">extension study (NCT03070119)</a>. Data demonstrated significant, treatment-related reductions in blood neurofilament light chain (NfL) levels, indicating a decline in nerve cell damage. Exploratory analyses also suggested the treatment&#8217;s use could slow ALS progression, particularly in people with rapidly progressing disease.
<h3>No. 3 – Chinese herbal skin patch, as add-on therapy, slows progression</h3>
A skin patch containing a mixture of Chinese herbs called Ji Wu Li (JWL), combined with standard treatments, <a href="https://alsnewstoday.com/news/herbal-skin-patch-add-on-therapy-slows-als-progression/">significantly slowed ALS progression</a> in a placebo-controlled clinical trial involving 120 adult patients. Benefits were seen after eight weeks of treatment and sustained for up to 20 weeks, or about five months.
Administered via a self-adhesive patch placed on the patient&#8217;s upper back, the transdermal, or on-the-skin, therapy may provide a way to deliver a concentrated formulation of JWL without swallowing, which can be challenging for some with ALS. Use of the patch, worn for six consecutive days each week and for six hours each day, also was reported to slow quality of life declines and ease weight loss.
<h3>No. 2 – Gene editing &#8216;cuts out&#8217; ALS-linked mutation in early study</h3>
In mouse models and human cells, the gene-editing tool CRISPR/Cas9 <a href="https://alsnewstoday.com/news/crispr-cas9-gene-editing-used-cut-out-mutation-early-study/">removed the repeat expansion defect in the C9ORF72 gene</a>, the most common genetic cause of ALS, scientists in a proof-of concept study reported.
While not interfering with the production of the C9ORF72 protein, such gene editing notably lowered levels of defective RNA molecules and small proteins associated with nerve cell death in ALS. RNA acts as the intermediary molecule derived from a DNA sequence, functioning as a template for protein production.
<h3>No. 1 – Poorer ALS survival linked with higher &#8216;good&#8217; cholesterol levels</h3>
A Netherlands-based population review study found that elevated levels of high-density lipoprotein, also known as &#8220;good&#8221; cholesterol, <a href="https://alsnewstoday.com/news/lower-als-survival-ratel-linked-higher-good-cholesterol-levels/">significantly correlated with a reduced survival rate</a> in people with ALS. Individuals in more advanced disease stages also were seen to have lower levels of total cholesterol and low-density lipoprotein, commonly referred to as &#8220;bad&#8221; cholesterol.
Its researchers acknowledged their study contrasts with previous research suggesting that a <a href="https://alsnewstoday.com/news/higher-good-cholesterol-blood-levels-linked-lower-als-risk/">lower ALS risk was tied to higher levels of good cholesterol</a>. Still, its findings contribute to insights into fat-like lipid metabolism and ALS severity, as their levels appear to be related to disease progression.
***
At ALS News Today, we hope our stories and reporting throughout 2023 positively impacted the lives of those affected by ALS. We look forward to serving the community further in 2024, seeking to provide ongoing support and information.
A very happy new year to all our readers!

Relyvrio reduces inflammatory biomarkers in ALS: CENTAUR data

People with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) who are treated daily with 1,000 mg or higher doses of <a href="https://alsnewstoday.com/tudca-tauroursodeoxycholic-acid/">tauroursodeoxycholic acid</a> (TUDCA) tend to live longer than those given standard care alone, according to an analysis of registry data.
&#8220;In our &#8216;real-world&#8217; study, patients who received TUDCA at the higher dose survived longer,&#8221; the researchers wrote.
&#8220;Since this was not a controlled study, we cannot rule out additional confounding factors,&#8221; the researchers noted. But &#8220;pending the results of ongoing [Phase 3 clinical trials], this study seems to confirm&#8221; TUDCA&#8217;s potential benefit to ALS patients, they added.
The study, &#8220;<a href="https://www.thelancet.com/journals/eclinm/article/PIIS2589-5370(23)00433-9">Effect of tauroursodeoxycholic acid on survival and safety in amyotrophic lateral sclerosis: a retrospective population-based cohort study</a>,&#8221; was published in <a href="https://www.thelancet.com/journals/eclinm/home">eClinicalMedicine</a>.
[recommended-reading id=87568]
<h3>TUDCA thought to help to protect nerve cells in ALS patients</h3>
TUDCA is a bile acid salt normally made in the liver. As an oral therapy, it&#8217;s long been used to treat liver disease, and accumulating evidence suggest it also may have nerve-protecting effects that can be beneficial in ALS.
The European Commission is sponsoring the Phase 3 <a href="https://clinicaltrials.gov/ct2/show/NCT03800524">TUDCA-ALS (NCT03800524)</a> clinical trial to evaluate the oral treatment&#8217;s efficacy, given on top of standard treatment with riluzole (sold as <a href="https://alsnewstoday.com/als-treatment/rilutek-riluzole/">Rilutek</a>, <a href="https://alsnewstoday.com/exservan-riluzole-oral-film/">Exservan</a>, and <a href="https://alsnewstoday.com/tiglutik-riluzole-oral-suspension/">Tiglutik</a>), in people with recent ALS. The <a href="https://alsnewstoday.com/news/results-phase-3-trial-tudca-als-likely-next-year/">trial is expected to conclude</a> later this year.
TUDCA is part of <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a>, alongside sodium phenylbutyrate. Relyvrio is approved to treat ALS in the U.S. and in Canada, where it&#8217;s sold under the brand name Albrioza. <a href="https://www.amylyx.com/" rel="nofollow">Amylyx Pharmaceuticals</a>, Relyvrio&#8217;s developer, is running a Phase 3 study called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a> to confirm the treatment&#8217;s efficacy.
While these large studies are ongoing, it&#8217;s not possible to say definitively whether TUDCA has a beneficial effect in ALS. Nonetheless, in some parts of Italy, it&#8217;s been common practice since 2015 to prescribe it off label for certain people with ALS.
Scientists in Italy analyzed available data from 86 ALS patients treated with TUDCA, as well as data covering 172 patients not using it. The two groups were matched for factors like sex, age at onset, diagnostic delay, and measures of disease severity and progression at <a href="https://alsnewstoday.com/als-diagnosis/">ALS diagnosis</a>.
Among those given TUDCA, treatment on average was started about eight months after diagnosis, or slightly more than 1.5 years after the first appearance of <a href="https://alsnewstoday.com/symptoms-of-als/">ALS symptoms</a>. The median duration of TUDCA treatment was about a year.
TUDCA doses varied markedly from person to person, the researchers noted, though patients given lower doses tended to be older, and more commonly had <a href="https://alsnewstoday.com/forms-of-als/#Clinical_onset">bulbar-onset ALS</a> where the disease initially affects muscles around the throat and mouth.
Over a median follow-up of more than three years, roughly half of all the patients in the analysis died or required a tracheostomy to assist breathing.
<h3>Median survival longer in ALS patients given TUDCA at 1 g or higher doses</h3>
Analyses showed that patients given TUDCA at daily doses of at least 1,000 mg had longer median survival times (56.5 months) compared to patients given lower doses (29.7 months) or patients not treated with TUDCA (36.2 months).
&#8220;High-dose TUDCA treatment yielded superior results in terms of survival both versus controls and low-dose treatment,&#8221; the researchers wrote.
In analyses comparing high-dose TUDCA to no TUDCA, statistical models indicated that treatment reduced the risk of death or tracheostomy by 55%. Analyses comparing patients on TUDCA for at least a year with untreated patients also found treatment prolonged survival.
&#8220;In our retrospective propensity score matched study, we found that treatment with TUDCA was associated with reduced risk of death and/or tracheotomy over 50% in patients treated with daily doses [of at least] 1000 mg in comparison to controls,&#8221; the researchers wrote.
About 1 in 5 patients given TUDCA experienced side effects that required stopping treatment or reducing the dose. The most common was diarrhea; some patients also reported abdominal pain or rash. In two cases, TUDCA-related side effects were serious enough to necessitate medical attention, but they resolved after treatment was stopped in both cases.
&#8220;Our study showed that the safety profile of TUDCA, even with a longer treatment duration, was mainly characterized by gastrointestinal side effects reported in almost 20% of TUDCA-exposed patients. &#8230; Drug reduction was sufficient for most patients presenting with side effects, but 35% of them (corresponding to 7% of the entire treated cohort) judged these effects intolerable and discontinued the treatment,&#8221; the researchers wrote.
This analysis is limited by the use of real-world data, which often is messier than those of clinical trials with stringent enrollment and measurement criteria, the researchers noted. Findings from ongoing trials will be needed to conclusively determine the likely efficacy and safety of TUDCA in ALS.

TUDCA at higher daily dose seen to extend survival: ALS registry study

Treatment with <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) in the CENTAUR trial significantly extended the median survival — by 10.4 months — of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) patients compared with an historical patient control group, a post-hoc analysis of study data reported.
This finding more than doubles the 4.8 months of extended survival initially observed in treated patients versus those randomized to a placebo in the 24-week trial, a majority of whom then were given Relyvrio in an open-label extension study.
The new analysis also found treatment lowered the risk of death over follow-up by 52% relative to the study’s external control group. The earlier analysis comparing treated CENTAUR patients with those who moved from placebo to Relyvrio reported a 36% lower risk of death.
“This [post-hoc] analysis is important because the survival difference seen helps support the robustness and clinical meaningfulness of the ITT [intent-to-treat] overall survival analysis on CENTAUR,” Machelle Manuel, PhD, head of global medical affairs at <a href="https://www.amylyx.com/">Amylyx Pharmaceuticals</a>, the treatment&#8217;s developer, said in a <a href="https://www.businesswire.com/news/home/20231010489409/en/Amylyx-Pharmaceuticals-Announces-Publication-of-Survival-Analysis-Comparing-CENTAUR-to-Historical-Clinical-Trial-Control">company press release</a>.
[recommended-reading id=81996]
<h3>ALS patients with rapid progression treated in CENTAUR and extension study</h3>
The study “<a href="https://onlinelibrary.wiley.com/doi/full/10.1002/acn3.51915">Analysis of sodium phenylbutyrate and taurursodiol survival effect in ALS using external controls</a>,” was published in the journal <a href="https://onlinelibrary.wiley.com/journal/23289503">Annals of Clinical and Translational Neurology</a>.
In this post hoc analysis of CENTAUR data, patients given the oral medication were compared with matched control participants from previously completed clinical trials.
These controls were part of the <a href="https://ncri1.partners.org/proact">Pooled Resource Open-Access ALS Clinical Trials (PRO-ACT) database</a>, the largest publicly available collection of ALS clinical trial data. It currently contains nearly 12,000 de-identified patient records from 29 Phase 2 and 3 trials.
“Analyses like the one &#8230; further our shared mission of accelerating the discovery of treatments and a cure for ALS,” said Alex Sherman, leader of the team that created the PRO-ACT database.
Relyvrio contains a fixed-dose combination of tauroursodeoxycholic acid and sodium phenylbutyrate, two compounds believed to protect nerve cells from certain forms of cellular stress.
Approved in the U.S. and in Canada — where it is sold as Albrioza — it is designed to slow disease progression and potentially extend survival.
Relyvrio&#8217;s approval was supported by data from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR Phase 2 trial (NCT03127514)</a>, which evaluated the treatment&#8217;s safety and efficacy against a placebo in adults recently diagnosed with rapidly progressing ALS.
Its 137 participants randomly were assigned to either Relyvrio or a placebo for six months. Then, a total of 90 patients who completed the main trial chose to enter an <a href="https://clinicaltrials.gov/ct2/show/NCT03488524">open-label extension (OLE) study (NCT03488524)</a>, in which all were treated for up to 30 months, or about 2.5 years.
Analyses spanning about 42 months in both the CENTAUR and OLE studies demonstrated that patients given Relyvrio in the main trial lived about 4.8 months longer than those who moved from a placebo to the active treatment. This meant that starting Relyvrio six months earlier lowered the risk of death by 36%.
<h3>Majority of placebo group patients opted for Relyvrio in long-term extension</h3>
However, such an analysis is unable to determine exactly how much longer patients survive when given Relyvrio versus those not using this treatment, as most placebo patients — 71% — entered the OLE and received Relyvrio.
To have a better idea of the therapy’s true effectiveness, researchers at the Sean M. Healey and AMG Center for ALS and the Neurological Clinical Research Institute (NCRI) at Massachusetts General Hospital conducted a post hoc analysis of CENTAUR data.
This analysis, done after the trial finished and all the data had been gathered, was funded by Amylyx. It specifically compared survival outcomes among Relyvrio-treated patients in CENTAUR with a matched group of patients from PRO-ACT — which includes <a href="https://alsnewstoday.com/news/amylyx-donates-centaur-records-pro-act-trials-database-als/">data from CENTAUR&#8217;s placebo arm</a>.
“The PRO-ACT initiative merges data from existing publicly and privately conducted ALS clinical trials to generate an invaluable resource for accelerating discovery in the field of ALS. This database allows anyone interested in moving ALS research forward an aid to do so quickly,” said Sherman, who also is director of the Center for Innovation and Biomedical Informatics at the NCRI.
In total, the post-hoc analysis included 89 ALS patients treated with Relyvrio and 85 controls from PRO-ACT, who were matched for factors such as disease duration, rate of disease progression, lung function, and age.
Notably, this control group had a similar rate of disease progression as the placebo group in CENTAUR — a monthly decline of 1.66 points in ALS Functional Rating Scale-Revised (ALSFRS-R) scores.
After a follow-up of 42 months, the median survival time in the Relyvrio group was found to be 23.54 months, significantly longer than the 13.15 months observed for the PRO-ACT control group.
Here, Relyvrio resulted in a 10.39-month longer survival, and a 52% lower risk of death compared with the PRO-ACT controls.
Overall, these findings are consistent with a <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">previous CENTAUR analysis</a>, where researchers estimated the likely survival of placebo group patients had they stayed on placebo in the OLE. In that analysis, Relyvrio resulted in a 10.6-month survival gain, which translated in a 61% reduction in the risk of death.
“The PRO-ACT database is comprised of thousands of harmonized longitudinal records from ALS clinical trials and helps advance scientific research by providing access to robust longitudinal participant data,” said Sabrina Paganoni, MD, PhD, with the Sean M. Healey and AMG Center for ALS &amp; the Neurological Clinical Research Institute at Massachusetts General Hospital, and Melanie Quintana, PhD, a senior statistical scientist with Berry Consultants.
“Analyses using well-matched external controls may provide additional context for evaluating survival effects in future ALS trials,” added the scientists, both lead authors of the study.

Relyvrio extends survival by 10.4 months versus external ALS group

New Brunswick is reimbursing, through its public health insurance program, the cost of <a href="https://www.amylyx.ca/document/albrioza-product-monograph.pdf" rel="nofollow">Albrioza</a> (sodium phenylbutyrate and ursodoxicoltaurine) for eligible residents living with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The Canadian province is the country&#8217;s fourth to provide public coverage for the oral therapy — marketed in the U.S. as <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a>. Similar reimbursement decisions recently were announced in <a href="https://alsnewstoday.com/news/treat-als-ontario-quebec-add-albrioza-public-reimbursement-list/">Ontario and Quebec</a>, followed by British Columbia.
“We sincerely appreciate the New Brunswick government&#8217;s collaboration and support in providing public coverage for Albrioza, to make timely treatment possible for individuals living with ALS,” Chris Aiello, general manager and head of Canada at <a href="https://www.amylyx.com/" rel="nofollow">Amylyx Pharmaceuticals</a>, the therapy’s developer, said in a <a href="https://www.newswire.ca/news-releases/new-brunswick-to-publicly-reimburse-albrioza-tm-for-the-treatment-of-als-879073652.html">company press release</a>.
“Recognizing the speed at which this devastating disease progresses, our aim is to ensure equitable access to this treatment for all eligible Canadians, regardless of their location,” Aiello said. “We are actively working with other provincial, territorial, and federal drug plans to expedite the process and make it a reality as quickly as possible.”
[recommended-reading id=85602]
<h3>ALS oral treatment given conditional approval in Canada in 2022</h3>
Previously called AMX0035, Albrioza is an oral therapy containing a combination of two molecules that are thought to help reduce damage to nerve cells. It was granted <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">conditional approval in Canada</a> for treating people with ALS in June 2022, based on clinical trial data suggesting it can <a href="https://alsnewstoday.com/news/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slow ALS progression</a> and <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">prolong survival</a>.
Full study results were published in <a href="https://www.nejm.org/doi/full/10.1056/NEJMoa1916945">The New England Journal of Medicine</a> and the journal <a href="https://onlinelibrary.wiley.com/doi/10.1002/mus.27569">Muscle &amp; Nerve</a>.
The medication also is <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">approved for ALS patients in the U.S.</a>, while a European Union committee <a href="https://alsnewstoday.com/news/chmp-opposes-approval-amx0035s-als-treatment-europe/">recommended against conditional approval</a> last month. Amylix plans to request a formal reconsideration of its application before the European Commission makes a final decision.
Albrioza has been available in Canada commercially for nearly a year, but only recently have patients begun to gain access to reimbursement formulas through public health insurance programs, run by each province and territory individually.
Announcements of reimbursement deals, in New Brunswick and elsewhere, come on the heels of Amylyx <a href="https://alsnewstoday.com/news/amylyx-agrees-deal-albrioza-als-treatment-reimbursement-canada/">finalizing an</a><a href="https://alsnewstoday.com/news/amylyx-agrees-deal-albrioza-als-treatment-reimbursement-canada/"> agreement</a> with the pan-Canadian Pharmaceutical Alliance, a government agency that collaborates with the country&#8217;s provinces and territories in establishing public drug plan funding of approved medications.
<h3>Advocates welcome news, but argue for therapy&#8217;s inclusion in plans nationwide</h3>
Advocates welcomed the decision, though they emphasized the importance of making the therapy available at lower cost to ALS patients throughout Canada.
“We applaud the government of New Brunswick for its decision to offer public coverage” for Albrioza, as “it is of utmost importance that eligible individuals can promptly access approved treatments,” said Kimberly Carter, president and CEO of the <a href="https://www.facebook.com/ALSNBNS/">ALS Society of New Brunswick and Nova Scotia</a>.
“We express our hope that other public drug plans, especially those in Atlantic Canada, will follow suit in making this treatment accessible,” Carter added. “This will ensure that Canadians dependent on public coverage will have the opportunity to benefit from this innovative treatment.”
ALS patients “critically need timely access to treatment,” said Colleen O&#8217;Connell, MD, medical director and research chief at Horizon&#8217;s Stan Cassidy Centre for Rehabilitation in New Brunswick.
“With few existing treatment options for ALS, I and others in the community are eager to hear news of Albrioza&#8217;s listing on other public drug plans to enable wider access to this treatment and greater hope for people living with this disease,” O&#8217;Connell added.
In Canada, prescription drugs are provided at no charge to all hospitalized patients. For others, each province and territory&#8217;s <a href="https://www.canada.ca/en/health-canada/services/health-care-system/pharmaceuticals/access-insurance-coverage-prescription-medicines.html">public health insurance plan</a> can cover at least part of a prescription drug that&#8217;s included in its plan.

New Brunswick adds Albrioza for ALS to its public health plan

The Committee for Medicinal Products for Human Use (CHMP) has issued an opinion opposing <a href="https://www.amylyx.com/" rel="nofollow">Amylyx Pharmaceuticals</a>’ bid for conditional European Union approval of AMX0035, its oral treatment to slow the progression of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The negative recommendation from the advisory committee, an arm of the European Medicines Agency (EMA), is not binding. But the EMA will consider the opinion when making its final approval decision, while Amylyx intends to request that CHMP reconsider its findings.
According to the EMA, <a href="https://www.ema.europa.eu/en/medicines/human/summaries-opinion/albrioza">the committee had concerns</a> that data from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR trial (NCT03127514)</a>, on which the application was based, did not &#8220;convincingly show&#8221; that AMX0035 was effective at <a href="https://alsnewstoday.com/living-with-als-2/">slowing ALS progression</a>.
[recommended-reading id=85161]
<h3>Oral ALS treatment has conditional approval in US and Canada, not Europe</h3>
Survival findings also were &#8220;unreliable,&#8221; the opinion stated, given how these data were collected and analyzed. In support of its decision, <a href="https://alsnewstoday.com/news/european-approval-amx0035-als-faces-new-hurdle-review/">advice from expert groups</a>, including neurology specialists and patient representatives, was considered.
The therapy is approved in the U.S. under the name <a href="https://alsnewstoday.com/amx0035/">Relyvrio</a> (sodium phenylbutyrate and taurursodiol) and in Canada as Albrioza — the brand name that will be used in Europe should it be approved.
“We are confident in the strength of our CENTAUR trial data, which we believe meets the criteria for conditional approval. These data were also the basis of the full approval received from the U.S. Food and Drug Administration and the approval with conditions from Health Canada,” Tammy Sarnelli, global head of regulatory affairs and clinical compliance at Amylyx, said in a <a href="https://www.businesswire.com/news/home/20230622200968/en/Amylyx-Pharmaceuticals-Receives-CHMP-Negative-Opinion-on-its-Conditional-Marketing-Authorisation-Application-for-AMX0035-for-the-Treatment-of-ALS-in-the-European-Union">company press release</a>. “We disagree with the CHMP’s opinion and will request a formal re-examination procedure of the current Marketing Authorisation Application (MAA).”
Filing for a formal reconsideration of the application starts a process that takes about four months. Based on an evaluation by a different set of examiners than those of the initial assessment, the process will close with CHMP adopting a final opinion.
“We will continue to engage with CHMP and EMA through the re-examination process with the goal of making Albrioza available in Europe as it is in the United States and Canada,” said Joshua Cohen and Justin Klee, Amylyx’s co-CEOs. “We know how precious time is for people with ALS and their families.”
Although it is not clear how AMX0035 works, its two active ingredients, sodium phenylbutyrate and ursodoxicoltaurine (taurursodiol in the U.S), are thought to reduce the nerve cell damage that <a href="https://alsnewstoday.com/causes-of-als/">causes ALS</a> and slow the progression of <a href="https://alsnewstoday.com/symptoms-of-als/">disease symptoms</a>.
<h3>Findings of Phase 3 trial of AMX0035 in ALS patients likely in mid-2024</h3>
Data from CENTAUR, a Phase 2 trial that enrolled 137 adults with rapidly progressing ALS, supported its <a href="https://alsnewstoday.com/news/amx0035-albrioza-oral-als-therapy-available-canada/">conditional approval in Canada</a> and <a href="https://alsnewstoday.com/news/relyvrio-amx0035-approved-us-als-treatment/">its U.S approval.</a>
Meeting its primary goal, the trial showed that AMX0035 significantly <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slowed disability progression</a> compared with a placebo. Data spanning CENTAUR and its open-label extension study also supported a <a href="https://alsnewstoday.com/news/amx0035-survival-als-patients-centaur-clinical-trial-analysis-amylyx/">10-month survival benefit</a> in people assigned to AMX0035 from the start.
A Phase 3 trial called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a> is underway to confirm AMX0035’s safety and efficacy in 664 ALS patients at sites across the U.S. and Europe. The new <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">trial is fully enrolled</a>, with top-line data expected by June 2o24.
“While our MAA continues to be under review, we will also work towards completing our global Phase 3 PHOENIX study, with topline results anticipated in mid-2024, which will provide important additional data on the efficacy and safety profile of Albrioza,” said Stéphanie Hoffmann-Gendebien, general manager at Amylyx. “There have been no new innovations approved in Europe for this devastating disease in over 25 years, and we recognize the urgent need of the ALS community in Europe to access new treatment options.”
CHMP’s negative opinion will not affect patients currently enrolled in clinical trials of AMX0035, Amylyx stated. It encourages study participants needing further information to speak with their clinical trial physician.

Tag: Relyvrio