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After two meetings with the U.S. Food and Drug Administration (FDA), <a href="https://coyatherapeutics.com/">Coya Therapeutics</a> says it&#8217;s received constructive feedback — and has reached an alignment with the agency regarding development plans for COYA 302, its therapy candidate for people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The company held the two meetings — a pre-investigational new drug (IND) meeting and a type C meeting — to seek advice from the FDA. Now, according to COYA, the two parties are in agreement regarding key areas of COYA 302&#8217;s development, to include its manufacturing, preclinical research, and clinical studies, which should support the filing of an IND application seeking clearance to start clinical trials.
That application is expected to be submitted in the first half of 2024, the company said in a <a href="https://www.businesswire.com/news/home/20240105424614/en/Coya-Therapeutics-Announces-Successful-Pre-IND-and-Type-C-Meetings-with-the-FDA-to-Advance-the-Development-of-COYA-302-for-Treatment-of-Amyotrophic-Lateral-Sclerosis-ALS">press release</a>.
If it&#8217;s accepted, Coya will soon thereafter launch a double-blind clinical trial to test COYA 302 in people with ALS. In a double-blind study, neither researchers nor participants know what treatment, if any, patients are receiving.
“This important feedback allows us to advance our development program in ALS with a planned double-blind controlled study, with the potential for bringing forward a much-needed therapy for ALS patients,” said Fred Grossman, Coya’s president and chief medical officer.
[recommended-reading id=82985]
<h3>In proof-of-concept study, COYA 302 showed marked benefits</h3>
ALS is characterized by progressive damage to motor neurons, the nerve cells that control voluntary movements. It renders the brain less able to control muscle movements.
<a href="https://coyatherapeutics.com/science-and-technology/">COYA 302</a> is an experimental approach that combines a low dose of the immune signaling molecule interleukin-2 (IL-2) and a fusion protein named CTLA-4 Ig (abatacept).
IL-2 is intended to reduce inflammation by increasing the levels and activity of anti-inflammatory immune cells called regulatory T-cells, or Tregs. CTLA-4 Ig, meanwhile, aims to decrease the activity of immune cells that promote inflammation and damage in ALS.
The therapy, administered via a subcutaneous or under-the-skin injection, has already been tested in four ALS patients in a proof-of-concept clinical trial. The participants received treatment for 48 weeks, or nearly one year, and were followed for eight more weeks after stopping COYA 302.
Results of the small study showed that ALS progression was markedly slowed during the treatment period, with patients experiencing similar levels of disability after 48 weeks compared with before treatment.
Prior to treatment, participants were losing a mean of 1.1 points per month in their ALS Functional Rating Scale (ALSFRS-R) scores, indicating a lower ability to perform daily tasks each month. During the entire study period, scores dropped a total of 1.5 points, on average.
However, once patients stopped COYA 302, progression rates returned to those observed before the trial started, indicating a need for continuous treatment.
Similarly, there was a statistically significant increase in Treg anti-inflammatory activity during the treatment, which also decreased to starting, or baseline, levels eight weeks after treatment discontinuation.
The study also evaluated biomarkers of inflammation and oxidative stress — a type of cell damage associated with inflammation. Available data up to 16 weeks, or about four months, suggested a decrease in these biomarkers, consistent with the observed increase in Treg function.
[quote=&#8221;center&#8221;]We believe that gaining alignment with FDA through multiple regulatory meetings on the path to filing an IND in [the second quarter of] 2024 is an important next step in advancing the program in patients with ALS.[/quote]
The therapy was generally well tolerated, with no serious side effects reported and no treatment discontinuations. The most common side effects were mild reactions at the injection site.
Coya plans to continue working closely with the FDA throughout COYA 302&#8217;s development program, the company said.
“We believe that gaining alignment with FDA through multiple regulatory meetings on the path to filing an IND in [the second quarter of] 2024 is an important next step in advancing the program in patients with ALS,” said Howard H. Berman, PhD, Coya’s CEO.

Coya aligns with FDA on plans for developing COYA 302 for ALS

<a href="https://brainstorm-cell.com">BrainStorm Cell Therapeutics</a> has had a meeting with the U.S. Food and Drug Administration (FDA) seeking an agreement on its plans for a confirmatory Phase 3b trial of <a href="https://alsnewstoday.com/nurown/">NurOwn</a>, a cell-based treatment the company is developing for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The main goal of the meeting, <a href="https://alsnewstoday.com/news/brainstorm-fda-meeting-dec-6-plans-nurown-als-trial/">announced</a> by BrainStorm in late November, was to discuss a special protocol assessment, or SPA, which ultimately ensures that the design of an upcoming trial is in line with FDA requirements to support a future marketing application.
The company now will submit the needed documents, as defined by the regulatory agency during the meeting, to support the SPA.
“Our meeting with the FDA provided us with valuable input on the Phase 3b trial design, outlining clear steps forward,” Chaim Lebovits, BrainStorm’s president and CEO, said in a <a href="https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-announces-outcome-of-fda-meeting-on-nurown-in-als-302008950.html">company press release</a>.
While the SPA does not guarantee that the trial results will be enough to support a potential approval, it “will serve to help de-risk certain regulatory aspects of the NurOwn clinical program,” Lebovits said.
[recommended-reading id=87237]
<h3>BrainStorm aims to resubmit NurOwn application with new trial data</h3>
The meeting was held a couple of months after BrainStorm <a href="https://alsnewstoday.com/news/brainstorm-drops-application-nurown-als-regroup/">withdrew its application</a> for NurOwn’s approval in the U.S. following a negative opinion by an FDA advisory committee. According to that committee&#8217;s overall findings, there was not enough evidence of the therapy’s efficacy. The negative opinion came just three months before the FDA had been expected to issue a decision on NurOwn&#8217;s approval.
BrainStorm then sought the face-to-face meeting with an aim toward ultimately getting its therapy approved.
“We recognize the urgency in providing ALS patients with safe and efficacious treatments and are committed to agreeing on a regulatory path forward for NurOwn. We are grateful for the FDA’s continued support and guidance and look forward to providing further updates once we have more clarity on an SPA,” Lebovits said.
[quote=&#8221;center&#8221;]We recognize the urgency in providing ALS patients with safe and efficacious treatments and are committed to agreeing on a regulatory path forward for NurOwn.[/quote]
In ALS, motor neurons — the nerve cells that control voluntary movements of muscles — become damaged and die. As a result, people with ALS experience <a href="https://alsnewstoday.com/symptoms-of-als/">symptoms</a> such as muscle twitching, wasting, and weakness, which worsen over time.
NurOwn involves collecting mesenchymal stem cells from a patient’s bone marrow. These stem cells, which can give rise to a variety of cell types, are matured in the lab into cells that produce large amounts of molecules that could help motor neurons survive.
The lab-grown cells are then returned to the patient through injections into the spinal canal, where they are expected to release the beneficial molecules and prevent motor neurons from dying, thereby slowing disease progression.
BrainStorm’s initial application for NurOwn’s approval was mainly based on data from a <a href="https://clinicaltrials.gov/study/NCT03280056">Phase 3 trial (NCT03280056)</a> that enrolled 189 adults with a <a href="https://alsnewstoday.com/als-diagnosis/">diagnosis of ALS</a> and rapidly progressing disease. Participants were given three injections of either NurOwn or a placebo at two-month intervals.
While a greater proportion of NurOwn-treated patients had a slower disease progression, as measured by a 1.25-point slower rate of decline in the ALS Functional Rating Scale (ALSFRS-R) after about seven months, the difference <a href="https://alsnewstoday.com/news/nurown-fails-to-slow-disease-progression-in-als-patients-top-line-data-show/">did not reach statistical significance</a>.
With a Phase 3 trial that failed to meet its main goal, and despite <a href="https://alsnewstoday.com/news/nurown-benefits-patients-less-severe-als-analyses-show/">promising results</a> in trial participants with less advanced disease, the FDA <a href="https://alsnewstoday.com/news/fda-declines-considering-nurown-als/">declined to review</a> an application for NurOwn’s approval, citing insufficient clinical evidence for its efficacy. The advisory committee meeting then backed the agency’s opinion.
The company now hopes that the future Phase 3b trial will provide enough data of the therapy’s benefits in ALS to allow a resubmission of a marketing application seeking regulatory approval.

BrainStorm, FDA map out next steps for Phase 3b testing of NurOwn

A final decision by the U.S. Food and Drug Administration (FDA) on whether or not to approve <a href="https://alsnewstoday.com/nurown/">NurOwn</a> for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is now expected no later than Dec. 8.
But before that, on Sept. 27, an FDA advisory committee will meet to review the potential benefits of the <a href="https://brainstorm-cell.com/">BrainStorm Cell Therapeutics</a> experimental cell therapy, according to a <a href="https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-announces-fda-advisory-committee-meeting-to-review-nurown-biologics-license-application-scheduled-for-september-27-2023-301843416.html">company press release</a>. That ADCOM meeting, to be held by the agency’s Cellular, Tissue and Gene Therapies Advisory Committee, will be available for live streaming.
The committee will provide the FDA with an independent recommendation for its use in considering regulatory approval for the ALS therapy candidate. However, the agency is not required to follow the committee&#8217;s recommendation.
The ADCOM meeting comes after the agency <a href="https://alsnewstoday.com/news/fda-declines-considering-nurown-als/">refused to review</a> a biologics license application (BLA) for NurOwn, filed by BrainStorm in September 2022, due to a lack of strong evidence of the therapy&#8217;s benefits in clinical trials. Brainstorm says that application now has been amended to address most of the FDA’s concerns.
“We remain confident in NurOwn and believe our data support regulatory approval,” said Stacy Lindborg, PhD, co-CEO at BrainStorm, adding, &#8220;As is the case with most ALS research, our clinical program generated complex results, which deserve a thoughtful and holistic review by scientists, ALS experts, FDA reviewers, advocates, and patients.&#8221;
[recommended-reading id=79235]
<h3>ADCOM meeting set for NurOwn after Brainstorm&#8217;s file over protest</h3>
BrainStorm was granted the ADCOM meeting <a href="https://alsnewstoday.com/news/als-therapy-maker-brainstorm-secures-adcom-over-fda-objection/">after its use of the FDA&#8217;s &#8220;file over protest&#8221; procedure</a> following the rejection of the BLA. As its name suggests, the file over protest procedure means that a company proceeds with filing a regulatory application despite the agency’s objection, which makes an advisory committee meeting possible.
Patient groups also <a href="https://alsnewstoday.com/news/als-patients-given-nurown-call-fda-meeting-discuss-refusal/">had called</a> for an ADCOM meeting after the FDA’s refusal to review NurOwn’s BLA. Five individuals with ALS treated in the NurOwn clinical trials and two family members of patients who died from the rare disorder had sent a written letter to the FDA in support of such a meeting.
“We believe this approach honors the needs of those living with ALS and offers the greatest promise for BrainStorm to fulfill our commitment to the ALS community,” Lindborg said of the upcoming meeting.
[quote align=&#8221;center&#8221;]We are encouraged by the regulatory flexibility that the FDA has shown over the last year in ALS broadly, and with respect to NurOwn in particular, and believe an Advisory Committee meeting is good for patients.[/quote]
NurOwn is a cell-based therapy in which mesenchymal stem cells — which can give rise to a variety of cell types — are harvested from a patient’s bone marrow and matured in the lab into cells that produce large amounts of signaling molecules that promote nerve cell health and growth.
The modified cells are then injected into the patient’s spinal canal to promote and support nerve cell repair, which is expected to help slow disease progression.
A <a href="https://clinicaltrials.gov/ct2/show/NCT03280056">Phase 3 clinical trial (NCT03280056)</a>, completed in 2020, had tested NurOwn in patients with rapidly progressing ALS, who were given three injections of either the therapy or a placebo two months apart. The trial involved 189 adults followed for 28 weeks, or about seven months.
The results showed that NurOwn <a href="https://alsnewstoday.com/news/nurown-fails-to-slow-disease-progression-in-als-patients-top-line-data-show/">was not significantly more effective than a placebo</a> at slowing disease progression, measured by the ALS functional rating scale (ALSFR-R). But according to BrainStorm, this was likely due to a large proportion of trial participants with advanced ALS — a population that already has the lowest scores in some ALSFR-R subscales.
Brainstorm said that the participants&#8217; advanced disease likely prevented the proper detection of changes in ALS progression.
Subsequent analysis excluding patients with more severe ALS showed <a href="https://alsnewstoday.com/news/nurown-continues-show-benefit-rapidly-advancing-als/">evidence of the therapy&#8217;s effectiveness in slowing ALS progression</a>, with statistically significant differences in ALSFR-R.
NurOwn <a href="https://alsnewstoday.com/news/als-biomarkers-boosted-nurown-regardless-disease-severity/">consistently reduced</a> markers of inflammation and neurodegeneration in the cerebrospinal fluid, the liquid surrounding the brain and the spinal cord, while increasing markers of neuroprotection. The levels of three of these biomarkers could predict clinical response to NurOwn.
“We are encouraged by the regulatory flexibility that the FDA has shown over the last year in ALS broadly, and with respect to NurOwn in particular, and believe an Advisory Committee meeting is good for patients,” said Chaim Lebovits, president and CEO at BrainStorm.
“We are of course deeply committed to the scientific and regulatory process, which includes continuing research to confirm the results of the NurOwn clinical program and are working with ALS experts in designing a rigorous clinical study to answer important questions about this therapy and inform further research on ALS,” Lebovits added.

FDA final approval decision on NurOwn for ALS due by year’s end

The U.S. Food and Drug Administration (FDA) has granted conditional approval to Biogen&#8217;s <a href="https://alsnewstoday.com/ionis-sod1-rx-biib067/">tofersen</a>, now named Qalsody, for the treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) associated with mutations in the SOD1 gene.
The decision, which marks the first conditional approval for ALS in the country, comes about eight months after the regulatory agency <a href="https://alsnewstoday.com/news/fda-expected-decide-tofersen-approval-sod1-als-january/">accepted the application for review</a> under its accelerated approval pathway. That pathway allows the FDA to give conditional marketing authorization to medications based on early clinical trial data suggesting a likely benefit.
Qalsody&#8217;s clearance had been based on biomarker data from the <a href="https://clinicaltrials.gov/ct2/show/NCT02623699">Phase 1/2/3 VALOR clinical trial (NCT02623699)</a> and its <a href="https://clinicaltrials.gov/ct2/show/NCT03070119">open-label extension study (NCT03070119)</a>. That data showed that the therapy led to <a href="https://alsnewstoday.com/news/early-tofersen-treatment-may-slow-sod1-progression-valor-study/">marked reductions</a> in blood levels of neurofilament light chain (NfL), a marker of nerve cell damage.
&#8220;The findings are reasonably likely to predict a clinical benefit in patients,&#8221; the FDA stated in a <a href="https://www.fda.gov/drugs/news-events-human-drugs/fda-approves-treatment-amyotrophic-lateral-sclerosis-associated-mutation-sod1-gene">press release</a> announcing the approval, adding, &#8220;The observed reduction in NfL was consistent across all subgroups based on sex, disease duration since symptom onset, site of onset, and use of other medications for ALS treatment.&#8221;
&#8220;For more than a decade, Biogen has been steadfast in our commitment to pursuing treatments for ALS, and I want to thank the scientists as well as the entire ALS community who have all worked tirelessly to bring this first-of-its-kind treatment to people with SOD1-ALS,&#8221; said Christopher A. Viehbacher, president and CEO of Biogen, in an email to ALS News Today. &#8220;Today also marks a pivotal moment in ALS research as we gained, for the first time, consensus that neurofilament can be used as a surrogate marker reasonably likely to predict clinical benefit in SOD1-ALS. We believe this important scientific advancement will further accelerate innovative drug development for ALS.”
[recommended-reading id=80852]
<h3>Regulators in Europe now considering Qalsody application for EU</h3>
The members of the FDA&#8217;s Peripheral and Central Nervous System Drugs Advisory Committee last month <a href="https://alsnewstoday.com/news/panel-favors-tofersen-biomarker-data-mixed-on-efficacy-sod1-als/">were mixed</a> in their views on whether the available data provided substantial evidence that Qalsody is effective in people with SOD1-ALS.
However, the experts unanimously considered NfL levels a good surrogate marker of the drug&#8217;s effectiveness — which likely supported the early approval.
According to the FDA announcement, the therapy is now approved at a recommended dose of 100 mg. It&#8217;s given via an injection into the spinal canal, called an intrathecal injection, containing 15 mL of the solution. The first three doses are given two weeks apart, and maintenance doses are then administered monthly.
No information is yet available on Qalsody&#8217;s list price, or how soon it will be available.
A similar application for the medication&#8217;s approval is being reviewed by the European Medicines Agency.
Meanwhile, the treatment is available in 34 countries via early access programs, Biogen has said.
<a href="https://alsnewstoday.com/als-and-genetics/">Mutations in the SOD1 gene</a> are found in as many as 20% of people with <a href="https://alsnewstoday.com/forms-of-als/familial-als/">familial ALS</a> and in up to 2% of <a href="https://alsnewstoday.com/sporadic-als/">sporadic ALS</a> cases. Such mutations result in a toxic form of the SOD1 protein that accumulates and forms clumps that damage nerve cells.
Administered via an intrathecal injection, Qalsody is designed to lower SOD1 levels and preserve nerve cell function. It does that by targeting SOD1’s messenger RNA (mRNA) — an intermediate molecule derived from DNA that guides protein production — for degradation.
By reducing the amount of SOD1 protein that is produced in cells, the therapy has the potential to slow disease progression and extend survival.
Qalsody&#8217;s approval in the U.S. was based on data from a <a href="https://clinicaltrials.gov/ct2/show/NCT03764488?term=BIIB067&amp;draw=2&amp;rank=1">Phase 1 trial (NCT03764488)</a> in healthy volunteers, as well as on the findings from the VALOR trial and its open-label extension study in ALS patients.
The Phase 1/2 part of VALOR found that Qalsody, given in single and multiple ascending doses, <a href="https://alsnewstoday.com/news/tofersen-safe-appears-to-slow-disease-progression-in-sod1-als-patients-phase-1-2-trial-shows/">was generally well tolerated</a> and able to lower SOD1 and NfL levels in the cerebrospinal fluid, the liquid surrounding the brain and spinal cord.
Exploratory analyses also suggested the treatment could slow ALS progression, with patients given the now approved 100 mg dose experiencing slower declines in functional ability, lung function, and muscle strength over 12 weeks compared with those on a placebo. The benefits were particularly evident in patients with rapidly progressing disease.
[recommended-reading id=80302]
<h3>Additional studies of Qalsody already underway</h3>
VALOR&#8217;s Phase 3 portion was designed to confirm the drug&#8217;s benefits in a larger group of patients. It enrolled 108 participants who were randomly assigned to receive eight intrathecal injections of either 100 mg of Qalsody or a placebo over six months.
The main goal was to assess changes in functional disability — as measured with the ALS Functional Rating Scale Revised (ALSFRS-R) — among the 60 participants with rapidly progressing ALS after 28 weeks, or about seven months.
While <a href="https://alsnewstoday.com/news/phase-3-tofersen-trial-fails-slow-sod1-als-trends-hopeful-valor-trial/">that goal was not met</a>, researchers saw some trends toward slower lung function and muscle strength decline. Also, patients given Qalsody experienced marked drops in their SOD1 and NfL CSF levels.
After completing VALOR&#8217;s Phase 3 part, a total of 95 participants opted to join an open-label extension study, in which all are receiving Qalsody for up to seven years. That study is expected to conclude in June 2024.
One-year data spanning the Phase 3 trial and the extension study showed the treatment resulted in a significant and clinically meaningful slowing of ALS progression.
Compared with patients who were initially assigned a placebo and then switched to Qalsody in the extension study, those who were always on Qalsody for one year experienced a smaller decline in their ALSFRS-R scores — 6 vs. 9.5.
Significant differences between these groups also were observed for measures of lung function and muscle strength. The early-start group also experienced significant extensions in survival and in time to death or permanent ventilation.
Additional analyses demonstrated that reductions in blood NfL levels within the first four months of treatment <a href="https://alsnewstoday.com/news/tofersen-clinical-benefits-linked-nfl-levels-trial-data/">predicted slower reductions in ALSFRS-R scores</a> up to 6.5 months. Such early drops also were linked to slower declines in other measures of function and disease severity.
The findings led the FDA&#8217;s advisory committee to unanimously consider blood NfL levels as a good surrogate measure of Qalsody&#8217;s efficacy in ALS patients, which likely contributed to the FDA deeming the available biomarker data enough to support its accelerated approval.
An ongoing Phase 3 trial, called <a href="https://clinicaltrials.gov/ct2/show/NCT04856982">ATLAS (NCT04856982)</a>, is now evaluating Qalsody&#8217;s clinical benefit in people with SOD1 mutations associated with rapidly progressing ALS. These participants have not yet experienced symptoms but show signs of neuronal damage as defined by elevated NfL levels.
A total of 150 adults are expected to enroll at <a href="https://clinicaltrials.gov/ct2/show/NCT04856982#contactlocation">nearly 30 sites worldwide</a>. They will be randomly assigned to receive either Qalsody (100 mg) or a placebo at the approved schedule — three intrathecal injections over the first month followed by monthly injections thereafter — for up to two years.
The main goal is to determine if the treatment can prevent patients from developing ALS symptoms. Top-line data are expected in 2026.

FDA approves tofersen, now Qalsody, as treatment for SOD1-ALS

A Phase 3b study evaluating the long-term safety and effectiveness of two dosing regimens of <a href="https://alsnewstoday.com/radicava-edaravone-for-als/">Radicava ORS</a> (edaravone) — an approved oral formulation of the medicine — in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) is now fully enrolled.
The study was designed to fulfill a post-marketing commitment that <a href="https://www.mt-pharma-america.com/">Mitsubishi Tanabe Pharma America</a> (MTPA) made to the U.S. Food and and Drug Administration (FDA) after the regulatory agency <a href="https://alsnewstoday.com/news/fda-approves-radicava-first-new-als-therapy-in-20-years-and-cause-for-hope/">approved</a> the into-the-vein (intravenous) formulation of Radicava in 2017.
The medication&#8217;s oral formulation, Radicava ORS, <a href="https://alsnewstoday.com/news/fda-approves-radicava-ors-oral-suspension-edaravone-als/">earned</a> its U.S. approval in 2022.
&#8220;We&#8217;re very pleased to announce the completion of trial enrollment in our Phase 3b study of Radicava ORS, representing an important achievement in our ongoing commitment to the fight against ALS,&#8221; Atsushi Fujimoto, president of MTPA, said in a <a href="https://www.prnewswire.com/news-releases/mitsubishi-tanabe-pharma-america-announces-enrollment-completion-for-global-phase-3b-study-of-radicava-ors-edaravone-in-als-301779833.html">company press release</a>.
&#8220;We are deeply grateful to the study participants and their families, as well as study investigators and clinical staff for working so diligently to help us meet this milestone,&#8221; Fujimoto added.
[recommended-reading id=82739]
<h3>Phase 3b study testing 2 different doses of oral therapy</h3>
Radicava ORS is a liquid suspension taken either orally or via a feeding tube. As with the intravenous formulation, it is given in 28-day cycles.
The first cycle consists of daily treatment for 14 days followed by a two-week period (a second 14 days) without the medication. In subsequent cycles, Radicava is taken daily for 10 of the next 14 days, followed again by two weeks without treatment.
Approval of Radicava ORS was based on <a href="https://alsnewstoday.com/news/als-treatment-radicava-ors-fares-well-phase-3-clinical-trial/">clinical trial data</a> indicating that a 105 mg dose of the oral treatment delivers similar amounts of edaravone — Radicava&#8217;s active ingredient — to the body and is as good at slowing disease progression as the intravenous version (60 mg).
Now, the global <a href="https://clinicaltrials.gov/ct2/show/NCT04569084">MT-1186-A02 study (NCT04569084)</a> is designed to compare the approved Radicava ORS on/off dosing regimen against once-daily dosing.
A total of 384 ALS patients, ages 18-75, whose symptoms emerged in the two years prior to the trial, were enrolled across 80 sites in the U.S., Canada, Europe, and Asia.
After an eight-week screening period, participants will either receive Radicava ORS every day, or be given the medication and a placebo. In the medication/placebo group, patients will receive Radicava ORS for 14 days and a placebo for 14 days in the first 28-day cycle, followed by 10 days of treatment and 18 days on a placebo for the next cycles.
The trial&#8217;s main goal is to evaluate changes in disease progression, as assessed by the ALS Functional Rating Scale-Revised (ALSFRS-R), over 48 weeks, or nearly one year. Secondary goals include changes in lung function and in measures of patient-reported quality of life, as well as the time patients live without requiring ventilatory support.
Top-line results from the trial, which already is underway, are expected in 2024.
&#8220;We look forward to seeing how the results of this study evaluating an alternative dosing regimen for Radicava ORS build upon our existing data to support its long-term efficacy and safety profile for people living with ALS, and how it may increase our understanding of the full potential of the medication as a treatment option,&#8221; said Michael D. Weiss, MD, of the University of Washington Medical Center, who is the study&#8217;s lead investigator.
Radicava works by combating oxidative stress, a type of cellular damage that&#8217;s been implicated in ALS. In doing so, it&#8217;s expected to protect nerve cells from damage and slow disease progression.
Intravenous Radicava also is approved in Japan, South Korea, Canada, and Switzerland, among other countries. The oral version has approvals in the U.S., Canada, and Japan.

Tag: U.S. Food and Drug Administration

Coya aligns with FDA on plans for developing COYA 302 for ALS

BrainStorm, FDA map out next steps for Phase 3b testing of NurOwn

FDA final approval decision on NurOwn for ALS due by year’s end

FDA approves tofersen, now Qalsody, as treatment for SOD1-ALS

Post-marketing trial of 2 Radicava ORS doses now fully enrolled