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<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.
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<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

Biological samples from <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) patients who took part in the Phase 3 clinical trial of NurOwn, a cell-based therapy, are being donated by <a href="https://brainstorm-cell.com/" rel="nofollow">BrainStorm Cell Therapeutics</a> to a repository for use by the research community.
The samples include blood and spinal fluid (cerebrospinal fluid or CSF) collected from participants on a placebo throughout the trial. They will be given to the <a href="https://neals.org/">Northeast Amyotrophic Lateral Sclerosis Consortium</a> (NEALS) biorepository.
NEALS is a nonprofit group of 147 research sites around the world working to translate clinical research into potential new treatments for ALS. The consortium&#8217;s biorepository includes blood, CSF, DNA, and urine samples from people who took part in various ALS clinical trials.
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<h3>Blood and CSF samples taken in NurOwn trial among those donated</h3>
&#8220;As a clinical research organization, NEALS is committed to using the NEALS Biorepository to help ALS researchers around the globe identify biomarkers and novel therapies as rapidly as possible,&#8221; James Berry, MD, director of the Neurological Clinical Research Institute at Massachusetts General Hospital, said in a <a href="https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-partners-with-neals-the-als-association-and-i-am-als-to-provide-public-access-to-biospecimens-from-nurowns-phase-3-als-study-301717525.html">company press release</a>.
&#8220;We are deeply appreciative of this contribution of samples, which adds to the biorepository and helps facilitate that goal,&#8221; Berry added.
With <a href="https://brainstorm-cell.com/science/#act" rel="nofollow">NurOwn</a>, mesenchymal stem cells are harvested from a patient’s bone marrow and matured in the lab to produce large amounts of signaling molecules that promote nerve cell survival and growth. The modified cells then are returned to the patient via a spinal canal injection to potentially slow disease progression.
The therapy was assessed in a <a href="https://clinicaltrials.gov/ct2/show/NCT03280056">Phase 3 trial (NCT03280056)</a> that enrolled 189 adults with rapidly progressing ALS. Patients were randomized to either three injections of NurOwn or a placebo every other month, and then were followed for up to about seven months (28 weeks).
The trial failed to meet its primary goal of slowing progression across treated patients, but later analyses supported<a href="https://alsnewstoday.com/news/nurown-continues-show-benefit-rapidly-advancing-als/"> benefit in those with less severe ALS</a> at the start of the therapy’s use.
Samples were collected from patients up to seven times over 20 weeks of the trial, the company reported. They were used to evaluate biomarker changes in each patient over time and to compare biomarker levels in the blood and CSF.
&#8220;Our Phase 3 trial included one of the largest longitudinal specimen collection protocols in trial participants with ALS, which has allowed us to perform extensive and unique biomarker analyses,&#8221; said Stacy Lindborg, PhD, co-CEO of BrainStorm.
&#8220;These analyses have greatly increased our understanding of both the mechanisms driving ALS progression, as well as the biological changes in participants treated with NurOwn,&#8221; Lindborg added. &#8220;We are pleased to be providing the research community with access to our clinical samples, which will drive new research and can facilitate the discovery of new breakthroughs for patients with ALS.&#8221;
The donation is connected to a<a href="https://alsnewstoday.com/news/brainstorm-awarded-500k-from-als-association-i-am-als-to-support-biomarker-research/"> $500,000 biomarker research grant</a> awarded to the company in 2020 by <a href="https://www.alsa.org/">The ALS Association</a> and <a href="https://iamals.org/">I AM ALS</a>.
The combined grant aimed to gain insights from clinical measures and tissue samples to identify biomarkers associated with NurOwn response. As part of the award, the company agreed to share samples with the ALS community to further advance biomarker research.
Ultimately, access to biomarker data provides information that could aid in disease understanding and developing targeted therapies.
&#8220;We would like to thank The ALS Association and I AM ALS for providing important funding for our biomarker analyses, as well as NEALS for housing our clinical samples for future use by the research community,” said Chaim Lebovits, BrainStorm&#8217;s co-CEO.

Tag: Phase 3 trial

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

Samples From ALS Patients in Phase 3 NurOwn Trial Open to Researchers