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<a href="https://brainstorm-cell.com/">BrainStorm Cell Therapeutics</a> is withdrawing the application requesting U.S. approval of its cell-based therapy <a href="https://alsnewstoday.com/nurown/">NurOwn</a> for the treatment of <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
The decision comes on the heels of a recent U.S. Food and Drug Administration (FDA) advisory committee meeting, in which experts ruled nearly unanimously that available data for the experimental therapy <a href="https://alsnewstoday.com/news/fda-advisory-committee-votes-not-enough-efficacy-nurown-als/">does not provide enough evidence</a> to support its effectiveness in ALS.
While an official decision from the FDA was expected in December, the withdrawal of the biological license application (BLA) means the agency no will longer review the data. BrainStorm has coordinated its decision to withdraw the BLA with the FDA, which regards the move as a withdrawal without prejudice for a future refiling.
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Meanwhile, the regulatory agency invited the company to request an expedited face-to-face meeting to discuss the next steps for NurOwn&#8217;s development. The company intends to use the meeting to explore a number of approaches, including the publication of emerging clinical data and an additional Phase 3b clinical study.
&#8220;After exploring possible options, we have reached this conclusion that the only path forward of NurOwn for ALS is to conduct another trial. Therefore, we&#8217;re withdrawing our BLA,&#8221; Chaim Lebovits, president and CEO of BrainStorm, said in a <a href="https://www.webcaster4.com/Player/Index?webcastId=49296&amp;g=824e82b3-5502-4572-b94f-9050e434ebcc&amp;uid=6296598&amp;sid=">company webcast</a> announcing the decision.
&#8220;We&#8217;re accepting [the] FDA&#8217;s invitation to have a face-to-face discussion on a Phase 3b trial, with the aim to reach an agreed-upon trial design,&#8221; he added. &#8220;We recognize the need to align with the FDA and will do all we can to deliver a valuable treatment option to the ALS community.&#8221;
Based on data from the earlier <a href="https://clinicaltrials.gov/study/NCT03280056">Phase 3 clinical trial (NCT03280056)</a>, the upcoming study &#8220;likely&#8221; will focus on a mild or less advanced patient population, said Stacy Lindborg, PhD, co-CEO of BrainStorm.
<h3>How NurOwn works</h3>
NurOwn is a cell-based therapy that involves collecting a patient’s stem cells from the bone marrow, engineering them in a laboratory so they produce high levels of signaling molecules that promote nerve health and growth, and then returning the modified cells directly to the patient through an injection into the spine.
A completed Phase 3 trial was designed to study the therapy against a placebo in a group of 189 people with rapidly-progressing ALS.
The trial <a href="https://alsnewstoday.com/news/nurown-fails-to-slow-disease-progression-in-als-patients-top-line-data-show/">failed to meet its primary goal</a> of showing that NurOwn could slow disease progression on the ALS Functional Rating Scale-Revised (ALSFRS-R) scale. But additional analyses showed it could slow the decline in that scale in a group of patients with less-advanced disease.
This prompted BrainStorm to submit the data to the FDA back in 2021, at which point the agency, citing lack of clinical evidence, <a href="https://alsnewstoday.com/news/nurown-trial-data-lacking-brainstorm-fda-approval-review/">discouraged the filing</a> of a regulatory application.
Later findings from the company showed that the lack of a significant effect could be attributable to a so-called <a href="https://alsnewstoday.com/news/mda-2023-issues-als-scale-noted-nurown-trial-failure-analysis/">floor effect</a>, where patients in the trial had such advanced disease that scores could not decrease even further as the disease progressed. That meant a slowdown in disease decline would not be detected.
In line with this, an analysis focusing only on patients with less-severe disease, which were not expected to be affected by this floor effect, suggested that NurOwn <a href="https://alsnewstoday.com/news/nurown-benefits-patients-less-severe-als-analyses-show/">could slow disease progression</a>, compared with a placebo.
This time, <a href="https://alsnewstoday.com/news/brainstorm-plans-filing-nurown-approval-us-corrected-data/">the company submitted an application</a> in August 2022, which the FDA <a href="https://alsnewstoday.com/news/fda-declines-considering-nurown-als/">refused to consider</a> in November of that year. Still, BrainStorm decided to &#8220;<a href="https://alsnewstoday.com/news/als-therapy-maker-brainstorm-secures-adcom-over-fda-objection/">file over protest</a>,&#8221; which granted it an advisory committee meeting where experts reviewed not only the clinical data, but also reports of patient and clinician experiences with NurOwn.
The committee, however, <a href="https://alsnewstoday.com/news/fda-questions-nurown-efficacy-quality-ahead-advisory-panel/">failed to detect evidence of a floor effect</a> in the trial, and cautioned that the analyses conducted after the trial ended are highly prone to false-positive results. That led to a 17-to-1 vote that the available clinical evidence was not enough to support the benefits of NurOwn in ALS patients.
&#8220;We are confident in the data supporting the value of NurOwn as an addition to the options for treating ALS, and we have every desire to bring it to the ALS community,&#8221; Lebovits said in a company <a href="https://www.prnewswire.com/news-releases/brainstorm-cell-therapeutics-to-meet-with-us-fda-to-discuss-development-plan-for-nurown-as-a-treatment-of-als-301960219.html">press release</a>.
<h3>Pivotal trial in the offing</h3>
&#8220;We look forward to working with the FDA to define the path forward. We understand that an additional pivotal trial will be necessary, and we are doing everything in our power to execute on this as quickly as possible,&#8221; he added.
The withdrawal also enables BrainStorm to finally tell patients who participated in the trial whether they were initially given a placebo or the cell therapy, the company said on the webcast.
&#8220;Information regarding randomization will be provided to the principal investigators at each of the clinical sites, who will provide information directly to participants and their families,&#8221; said Lindborg.

Tag: biological license application

BrainStorm drops FDA application for NurOwn