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Up to three years of daily treatment with the investigational therapy <a href="https://alsnewstoday.com/ipl344/">IPL344</a> was well tolerated and slowed disease progression among adults with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), according to top-line data from a small Phase 1/2a clinical trial.
Preliminary efficacy analyses also indicated that IPL344 might be able to increase body weight, preserve respiratory function, and prolong survival among ALS patients, as shown relative to an external placebo group.
The findings were announced by the therapy&#8217;s developer, <a href="https://immunitypharma.com/">Immunity Pharma</a>, which now plans to submit the data for publication in a scientific journal.
&#8220;These early-stage data support further development of IPL344 as a treatment for ALS, and merit further investigation in a large number of participants,&#8221; Eran Ovadia, Immunity&#8217;s CEO, said in a <a href="https://pipelinereview.com/index.php/2024011785237/Proteins-and-Peptides/Immunity-Pharma-Announces-Positive-Top-Line-Results-from-Phase-2a-Trial-with-IPL344-in-ALS-Patients.html">company press release</a>.
&#8220;We intend to progress [IPL344] to a pivotal clinical trial in ALS,&#8221; Ovadia added. That would support an application for the drug&#8217;s approval, pending positive results.
[recommended-reading id=88327]
<h3>Monthly declines in ALS progression of 48% seen with IPL344</h3>
IPL344 is a peptide designed to stimulate cellular pathways thought to be protective in ALS. It specifically activates the Akt pathway that&#8217;s involved in the survival of motor neurons — the specialized nerve cells that communicate with muscle cells to coordinate voluntary movements — which are progressively lost in ALS.
Studies indicate that <a href="https://pubmed.ncbi.nlm.nih.gov/16507768/#:~:text=Human%20skeletal%20muscle%20atrophy%2C%20as,or%20via%20another%20signaling%20pathway.">Akt signaling is diminished</a> in people with ALS. By boosting Akt activity, it&#8217;s believed that IPL344 may help to protect motor neurons and slow ALS progression.
In preclinical studies, IPL344 treatment <a href="https://www.hilarispublisher.com/proceedings/peptide-ipl344-an-akt-activator-treats-sod1-als-model-mice-to-improve-neuromuscular-function-36691.html">improved neuromuscular function</a>, prevented weight loss, and extended survival in a mouse model of ALS.
The investigational therapy has received <a href="https://immunitypharma.com/news/61-immunity-pharma-announces-that-its-novel-als-treatment-was-granted-an-orphan-drug-designation-by-both-the-fda-and-the-european-medicines-agency">orphan drug designation</a> in the U.S. and Europe — a status intended to accelerate a treatment&#8217;s clinical development by offering regulatory support and financial incentives.
The Phase 1/2a trial evaluated IPL344&#8217;s safety and preliminary efficacy among nine ALS patients, ages 18-80, at a single site in Israel. All were experiencing quickly progressing disease, defined as at least a 0.55-point monthly decline in scores on the ALS Functional Rating Scale-Revised (ALSFRS-R).
In the <a href="https://clinicaltrials.gov/ct2/show/NCT03652805">Phase 1 portion (NCT03652805)</a>, all participants received increasing daily doses of IPL344, delivered as an injection directly into the bloodstream, for 28 days, or about one month). The starting dose of 1.7 mg/kg was increased by 0.5 mg/kg every 3-4 days up to a maximum dose of 3.2 mg/kg. <a href="https://alsnewstoday.com/news-posts/2020/09/17/investigational-treatment-ipl344-safely-slows-als-progression-in-small-phase-1-2a-trial/">Interim results</a> indicated the treatment was safe and well tolerated.
Participants then continued receiving daily IPL344 for up to 36 months, or three years, in the <a href="https://clinicaltrials.gov/ct2/show/NCT03755167">Phase 2 part of the trial (NCT03755167)</a>.
Immunity now reports that IPL344-treated participants lost a mean of 0.53 points per month in the ALS Functional Rating Scale Revised (ALSFRS-R) with up to three years of treatment, reflecting a significant, 48% slowing in disease progression.
[quote=&#8221;center&#8221;]If confirmed in a subsequent larger Phase 3 study, the magnitude of reduction in ALSFRS-R progression observed in this study is greater than that achieved with currently approved drugs.[/quote]
When disease stage and other influential factors were considered in the statistical analysis, monthly declines were found to be 64% slower.
&#8220;If confirmed in a subsequent larger Phase 3 study, the magnitude of reduction in ALSFRS-R progression observed in this study is greater than that achieved with currently approved drugs,&#8221; said Ilana Cohen, PhD, Immunity Pharma&#8217;s vice president of research and development.
[recommended-reading id=88415]
<h3>Study&#8217;s participants see longer survival time compared with placebo group</h3>
Patients also experienced increases in body weight, a significant difference from a pooled group of placebo-treated patients involved in previous ALS clinical trials, in which patients continuously lost weight as the disease progressed. Those natural history data were housed in the PRO-ACT ALS patient database.
Moreover, average monthly declines in respiratory function were 44% slower on IPL344 than in the historical placebo group, though this difference was not statistically significant.
The median survival time on IPL344 was 29 months, or nearly 2.5 years, compared with 19 months, or a little longer than 1.5 years, in an external placebo group from a clinical trial of a different therapy. This comparison suggests a trend toward prolonged survival with IPL344.
For the eight patients with available data, <a href="https://immunitypharma.com/images/publications/ImmunityPharma%20NfL%20December%202023%20Poster.pdf">reductions in neurofilament light chain</a> (NfL) — a marker of nerve damage — also were observed for all but one. That patient experienced a stabilization in NfL levels. This reflects a possible neuroprotective effect of the therapy, according to Immunity.
IPL344 reportedly was well tolerated, with no major serious adverse events associated with the therapy and no treatment discontinuations due to side effects from the medication.
&#8220;We are excited that treatment with IPL344 was well tolerated in people with ALS and demonstrated encouraging signs of efficacy across multiple parameters,&#8221; Cohen said.
Long-term survival follow-up for these nine patients is ongoing. The trial initially planned to enroll 15 participants, but was suspended early due to a shortage in the investigational therapy.

Daily IPL344 for up to 3 years seen to slow ALS progression in trial

NeuroSense Therapeutics’ PrimeC was found to be safe and well tolerated in a Phase 2b trial, and use of the oral therapy for six months led to &#8220;meaningful slowing of disease progression&#8221; in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS), the company said in a <a href="https://www.prnewswire.com/news-releases/neurosenses-phase-2b-als-trial-achieves-primary-safety-and-tolerability--secondary-clinical-efficacy-endpoints-302005837.html">press release</a> announcing top-line results.
The new data comes from <a href="https://clinicaltrials.gov/study/NCT05357950">PARADIGM (NCT05357950)</a>, a <a href="https://alsnewstoday.com/news/neurosense-paradigm-trial-primec-als-completes-enrollment-patients/">fully enrolled</a> clinical trial that&#8217;s testing PrimeC against a placebo in 69 adults with a <a href="https://alsnewstoday.com/als-diagnosis/">diagnosis of ALS</a>.
The positive findings will now support the launch of a pivotal and global Phase 3 trial that&#8217;s <a href="https://alsnewstoday.com/news/primec-potential-als-therapy-earns-patents-in-europe-japan-israel/">planned for 2024</a>. The U.S. Food and Drug Administration (FDA) already has <a href="https://alsnewstoday.com/news/fda-oks-neurosenses-cmc-primec-ahead-phase-3-trial/">agreed to the manufacturing practices</a> and product specifications set in place ahead of the trial.
“The release of this portion of the top-line results of the PARADIGM trial marks an exciting milestone for NeuroSense as we take another step toward helping people suffering from this dire disease,” said Alon Ben-Noon, <a href="https://www.neurosense-tx.com">NeuroSense</a>’s CEO.
According to the release, PrimeC &#8220;achieved primary safety and tolerability endpoints [goals] with a safety and tolerability profile comparable to [a] placebo.&#8221;
[recommended-reading id=87331]
<h3>Use of PrimeC in PARADIGM leads to 29% slower disease progression</h3>
NeuroSense also is planning to report the results of biomarker analyses from the PARADIGM trial in the first half of 2024.
These include changes in ALS blood biomarkers TDP-43 and prostaglandin J2 after six months — one of the trial&#8217;s main goal. The company also expects to share its findings on changes in neurofilament blood levels, a biomarker of nerve cell damage. That analysis is being done in collaboration with <a href="https://www.biogen.com/">Biogen</a>.
“We look forward to meeting with the FDA to determine the best path forward and to advancing discussions with strategic partners who share our vision for PrimeC to benefit people living with ALS,” Ben-Noon said.
PrimeC contains a fixed-dose combination of two FDA-approved medications: ciprofloxacin, an antibiotic used to treat bacterial infection, and celecoxib, a medicine for relieving pain and inflammation. It is expected to slow <a href="https://alsnewstoday.com/stages-of-als/">disease progression</a> by blocking key mechanisms behind ALS.
PARADIGM, slated for completion in fall of 2024, is testing a long-acting formulation of PrimeC, which releases the medications in the body over time. In the trial, PrimeC was given at a total daily dose of 1,496 mg, taken as two tablets twice daily.
A total of 69 adults with ALS — taking part in the study at sites in Canada, Italy, and Israel — were randomly assigned to receive the medication or a placebo for six months. Data was gathered from 68 patients, due to one misdiagnosis.
The trial&#8217;s main goals are to determine if PrimeC is safe and well tolerated in ALS patients, and whether it reduces the amount of ALS biomarkers in circulation, indicating a potential effect on disease processes.
Secondary measures include changes in the ability to perform daily tasks, as assessed with the ALS Functional Rating Scale-Revised (ALSFRS-R), as well as changes in lung function, quality of life, and survival.
Over the trial&#8217;s six months, safety and tolerability profile of PrimeC was comparable to that of the placebo.
[quote=&#8221;center&#8221;]In addition to the safety and tolerability profile observed, we believe the 29% difference observed in ALSFRS-R in favor of PrimeC as well as the 13% decline in [lung function] compared to the placebo arm, illustrate PrimeC&#8217;s potential to render a meaningful clinical benefit to people living with ALS.[/quote]
Also, patients treated with PrimeC experienced a 29% slower disease progression on the ALSFRS-R scale, as well as a 13% slower decline in lung function, compared with those given a placebo. These differences failed to reach statistical significance, but the company noted that this trial was not powered to detect such significant difference.
&#8220;In addition to the safety and tolerability profile observed, we believe the 29% difference observed in ALSFRS-R in favor of PrimeC as well as the 13% decline in [lung function] compared to the placebo arm, illustrate PrimeC&#8217;s potential to render a meaningful clinical benefit to people living with ALS,&#8221; said Ferenc Tracik, MD, NeuroSense’s chief medical officer.
After completing the six months of dosing, most participants (96%) chose to enter an open-label extension, in which all are being treated with PrimeC for up to one year. So far, all who completed 1.5 years of treatment have opted to continue taking PrimeC by joining an ongoing investigator-initiated trial.
“I am excited by the top-line clinical data from PARADIGM, as this is an important milestone for the patients I care for and for the entire ALS community,” said Merit Cudkowicz, MD, chair of neurology at the Massachusetts General Hospital and director of the Healey &amp; AMG Center for ALS in Boston.
“The positive results support moving forward to a Phase 3 pivotal trial. The biomarker data will also be very informative, and I look forward to seeing those results in early 2024,” added Cudkowicz, who is also a member of NeuroSense’s scientific advisory board.

PrimeC safe, shows trends toward slowing ALS progression: Trial data

<a href="https://www.neurosense-tx.com/" rel="nofollow">NeuroSense Therapeutics</a> is planning to soon announce top-line safety and efficacy results from its PARADIGM trial, which is testing PrimeC in people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
News of trial findings being expected in December follows the dosing of a final patient in the randomized portion of the <a href="https://clinicaltrials.gov/study/NCT05357950">Phase 2b trial (NCT05357950)</a>. A total of 69 people have received the experimental therapy or a placebo for six months, and most (96%) have chosen to enter an open-label extension where all are being treated with PrimeC for up to one year.
The company also plans to report findings across a number of PARADIGM biomarker analyses in the first half of 2024.
&#8220;We are excited to soon begin sharing the results of the double-blind segment of our Phase 2b trial,&#8221; Alon Ben Noon, NeuroSense’s CEO, said in a <a href="https://www.prnewswire.com/il/news-releases/neurosense-completes-dosing-of-last-patient-in-the-double-blind-segment-of-phase-2b-als-trial-topline-clinical-efficacy-results-expected-december-2023-301978417.html">company press release</a>.
[recommended-reading id=87783]
<h3>PARADIGM trial assessing a longer-acting formulation of PrimeC</h3>
&#8220;We would like to thank the trial participants, their caregivers and families, as well as the sites&#8217; Principal Investigators and study coordinators for their tremendous contribution to PARADIGM,&#8221; Ben Noon added.
PrimeC is an oral, fixed-dose combination of two medications approved in the U.S. for other indications, with established safety profiles. One is ciprofloxacin, an antibiotic, and the other is celecoxib, an anti-inflammatory.
Together, these compounds are designed to target several key mechanisms that contribute to the nerve cell degeneration that <a href="https://alsnewstoday.com/causes-of-als/">causes ALS</a>. Specifically, it aims to block inflammation, iron accumulation, and impaired RNA regulation to slow <a href="https://alsnewstoday.com/stages-of-als/">disease progression</a>.
In a previous <a href="https://clinicaltrials.gov/study/NCT04165850">Phase 2a clinical trial (NCT04165850)</a> in Israel, an initial formulation of PrimeC was found to be <a href="https://alsnewstoday.com/news/primec-safe-lowers-als-disease-biomarkers-phase-2-clinical-study/">safe and well tolerated</a> in 15 ALS patients. The therapy also slowed functional and respiratory decline compared with an historical group of untreated patients, and lowered ALS biomarkers, particularly TDP-43.
PARADIGM is testing a longer-acting formulation of PrimeC that releases the medications over an extended period of time, requiring less frequent dosing.
Participants, whose symptoms of muscle weakness began less than 30 months before screening, were randomly assigned to either PrimeC or a placebo for six months. The active medication was given at a total daily dose of 1,496 mg, taken as two tablets twice daily, and patients were allowed to continue their standard <a href="https://alsnewstoday.com/als-treatment/">ALS therapies</a>.
<h3>Efficacy measured by changes in disability, lung health with treatment</h3>
The study&#8217;s primary goals are to determine PrimeC&#8217;s safety and its effects on key ALS blood biomarkers, TDP-43 and prostaglandin J2. Secondary goals include changes in the ALS Functional Rating Scale-Revised (ALSFRS-R), a measure of functional disability, changes in lung health and quality of life, and survival outcomes.
Data covering these secondary efficacy measures, as well as safety results, are expected to be announced in December, and the biomarker findings by June. NeuroSense also expects to report, by the close of March, results of a blood biomarker analysis being conducted under a collaboration with Biogen.
This analysis is looking at changes in plasma levels of neurofilaments, established markers of nerve damage, among people treated with PrimeC. Biogen is conducting the analysis, and under the agreement has a right of first refusal to co-develop and co-commercialize PrimeC depending on the results.
PrimeC has been <a href="https://alsnewstoday.com/news/als-combination-therapy-primec-receives-fdas-orphan-drug-designation-enters-clinical-trials/">designated an orphan drug in the U.S.</a> and <a href="https://alsnewstoday.com/news/primec-orphan-drug-designation-europe-treatment-als/">the European Union</a>, a status meant to accelerate the development and regulatory review of therapies for rare diseases.

Top-line results of PARADIGM trial into PrimeC expected by year’s end

An advisory committee of the European Medicines Agency has maintained its recommendation not to approve <a href="https://alsnewstoday.com/amx0035/">AMX0035</a> for <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS).
In its <a href="https://alsnewstoday.com/news/chmp-opposes-approval-amx0035s-als-treatment-europe/">initial assessment issued in June</a>, the Committee for Medicinal Products for Human Use (CHMP) said data from the <a href="https://clinicaltrials.gov/ct2/show/NCT03127514">CENTAUR Phase 2 trial (NCT03127514)</a>, which formed the basis for the application, did not convincingly show that AMX0035 was effective at slowing ALS progression.
<a href="https://www.amylyx.com/">Amylyx Pharmaceuticals</a>, the company developing the treatment, then requested a formal re-examination, in which available clinical data were evaluated by a different set of examiners, but this second review process has confirmed the initial opinion.
An official decision on AMX0035 from the European Medicines Agency is now set to be handed down by year&#8217;s end. The regulatory agency is not obligated to abide by the committee’s opinion, but it usually does.
“We are disappointed to learn of this outcome, as it is a further setback for the more than 30,000 people <a href="https://alsnewstoday.com/living-with-als-2/">living with ALS</a> and their loved ones in Europe who have not seen therapeutic progress for this devastating disease in over 25 years,” the patients and carers board of the <a href="https://als.eu/">European Organization for Professionals and Patients with ALS</a> said in a <a href="https://www.businesswire.com/news/home/20231012969323/en/Amylyx-Pharmaceuticals-Receives-Negative-CHMP-Opinion-on-its-Marketing-Authorisation-Application-for-AMX0035-for-the-Treatment-of-ALS-in-the-European-Union-Following-Re-Examination-Process">press release</a>.
[recommended-reading id=86997]
<h3>Other paths to AMX0035&#8217;s approval in Europe being explored</h3>
Meanwhile, the company says it will continue to explore other paths to AMX0035&#8217;s approval in Europe.
The treatment is being evaluated in a larger Phase 3 trial called <a href="https://clinicaltrials.gov/ct2/show/NCT05021536">PHOENIX (NCT05021536)</a>, whose data was not included in the initial application. Pending positive results, Amylyx is planning to submit an application to the European agency as soon as possible.
AMX0035, a fixed-dose combination of tauroursodeoxycholic acid and sodium phenylbutyrate, is already available in the U.S. under the brand name Relyvrio. It has also gained <a href="https://alsnewstoday.com/news/amx0035-oral-als-therapy-called-albrioza-approved-canada/">conditional approval in Canada</a>, where it is sold as Albrioza — that brand name will also be used in Europe if it&#8217;s approved.
“We share the frustration felt by the European ALS community, who has no time to wait for new, safe, and effective treatment options,” said Stéphanie Hoffmann-Gendebien, general manager and head of Europe, the Middle East, and Africa at Amylyx.
“We remain committed to exploring all potential paths forward in support of the company’s goal of getting AMX0035 to people living with ALS in the EU as quickly as possible,” she added.
The safety and effectiveness of AMX0035 was initially assessed in the CENTAUR trial, which enrolled 137 adults with early and rapidly progressing ALS. Data showed Relyvrio significantly <a href="https://alsnewstoday.com/news-posts/2020/09/03/amx0035-safe-effective-in-slowing-als-functional-decline-trial-data-shows/">slowed functional decline</a> and <a href="https://alsnewstoday.com/news-posts/2020/10/19/amylyxs-amx0035-significantly-extends-als-patients-lives-long-term-trial-data-show-also-slows-functional-decline/">extended survival</a>, compared with a placebo.
“AMX0035 is the first and only drug to show an effect on both function and survival in the same trial. Since the medication’s approval with conditions in Canada and full approval in the U.S., thousands of people have been prescribed AMX0035 in North America,&#8221; said Joshua Cohen and Justin Klee, co-CEOs of Amylyx.
[quote=&#8221;center&#8221;]We are disappointed to learn of this outcome, as it is a further setback for the more than 30,000 people living with ALS and their loved ones in Europe who have not seen therapeutic progress for this devastating disease in over 25 years.[/quote]
<h3>Phase 3 PHOENIX trial fully enrolled with 664 participants in US, Europe</h3>
The PHOENIX trial was then launched to confirm the benefits seen in CENTAUR in a larger group of ALS patients. The trial is <a href="https://alsnewstoday.com/news/phoenix-trial-approved-als-therapy-relyvrio-amx0035-enrolled/">fully enrolled</a>, with 664 participants recruited across 69 clinical sites in the U.S. and Europe.
Participants were randomly assigned to Relyvrio or a placebo, given daily for 48 weeks, or nearly one year. They will then be given the option to join an open-label extension part, where all will the receive the medication for two years.
The trial&#8217;s main goal is to determine if AMX0035 can slow disease progression, as assessed by changes in the ALS Functional Rating Scale-Revised (ALSFRS-R). Secondary measures include changes in lung function and quality of life, and an extension in survival time.
Top-line data are expected by June 2024.
&#8220;ALS has no geographical boundaries, and we are working with urgency toward providing timely, broad, and sustainable access to AMX0035 for eligible people living with ALS who may benefit,” Cohen and Klee added.

CHMP confirms its initial position against approving AMX0035 in EU

A Phase 2b trial of PrimeC for people with <a href="https://alsnewstoday.com/what-is-als/">amyotrophic lateral sclerosis</a> (ALS) has completed patient enrollment, according to <a href="https://www.neurosense-tx.com/">NeuroSense Therapeutics</a>, the therapy&#8217;s developer.
Called <a href="https://clinicaltrials.gov/ct2/show/NCT05357950">PARADIGM (NCT05357950)</a>, the study enrolled 69 adults <a href="https://alsnewstoday.com/als-diagnosis/">diagnosed with ALS</a>, ages 18-75, at sites in Israel, Italy, and Canada. The company anticipates top-line results late this year.
The trial is testing an extended-release formulation of PrimeC, designed to require less frequent dosing for patients.
“Based on the new formulation, we are aiming for efficacy and safety results that will meet and exceed the results achieved in the Phase 2a study,” Vivian Drory, MD, principal investigator of the PARADIGM trial at Tel Aviv Sourasky Medical Center, in Israel, said in a <a href="https://www.prnewswire.com/news-releases/neurosense-completes-enrollment-in-phase-2b-als-clinical-trial-301824559.html">company press release</a>.
Results from this study will inform the design of a future pivotal Phase 3 clinical trial of PrimeC, the company said.
[recommended-reading id=78033]
<h3>PARADIGM trial testing 17 outcome measures for Prime C</h3>
PrimeC is an oral, fixed-dose combination of two medications approved in the U.S. that both have well-established safety profiles. One, ciprofloxacin, is an antibiotic, while the other, celecoxib, is an anti-inflammatory agent.
The combination is thought to potentially inhibit disease progression by targeting key mechanisms in ALS motor degeneration. Specifically, the therapy aims to block inflammation, iron accumulation, and impaired RNA processing.
An initial formulation of PrimeC was found to be <a href="https://alsnewstoday.com/news/primec-safe-lowers-als-disease-biomarkers-phase-2-clinical-study/">safe and well tolerated</a>, and showed some promising trends toward less functional and respiratory decline, in a previous <a href="https://clinicaltrials.gov/ct2/show/NCT04165850">Phase 2a trial (NCT04165850)</a>. That trial involved 15 ALS patients.
The PARADIGM trial was designed to test a longer-acting formulation of PrimeC, which may be administered less frequently because it releases the two medications over an extended period of time.
The trial enrolled ALS patients whose symptoms of muscle weakness began less then 30 months (about 2.5 years) before screening. All participants were randomly assigned to receive Prime C, at a total daily dose of 1,496 mg — two tablets twice daily — or a placebo, for six months. All patients maintained their standard <a href="https://alsnewstoday.com/als-treatment/">ALS medication</a> regimen during the trial.
The study primarily aims to evaluate the treatment&#8217;s safety and tolerability, as well as changes in ALS biomarkers associated with PrimeC treatment. Secondary measures include changes in patients&#8217; functional disability, lung function, quality of life, and survival.
“PARADIGM is tracking 17 different outcome measures while the primary endpoint remains focused on biomarkers,“ said Ferenc Tracik, MD, chief medical officer at NeuroSense.
“This wealth of outcome data will not only be used to optimize the design of a future pivotal Phase 3 study for PrimeC, but we believe it will provide greater insight into the mechanisms of ALS for the scientific community as we advance toward a day when ALS will become a highly treatable disease,&#8221; Tracik said.
After completing the trial’s randomized phase, patients will have the option to enter an open-label extension phase, during which all participants will receive PrimeC for one year. To date, 26 participants completed the randomized phase, and 25 chose to continue into the extension part.
&#8220;It is highly encouraging that nearly all participants who completed the study have elected to continue in the [open label extension] phase with the treatment of PrimeC,&#8221; Drory said.
&#8220;We look forward to seeing the topline data from this study,&#8221; Drory added, noting the trial is testing the &#8220;optimized formulation of PrimeC.&#8221;
PrimeC has been granted orphan drug status in both the U.S. and the European Union. This designation is awarded to therapies that aim to treat rare diseases.
&#8220;We are pleased to share this milestone for the PARADIGM clinical trial,&#8221; said Alon Ben-Noon, CEO of NeuroSense, adding, &#8220;We are grateful to the participants living with ALS, their families, and the study investigators for their commitment to the PARADIGM trial and for taking part in our mission to develop an effective therapy for ALS.&#8221;

Tag: top-line data

Daily IPL344 for up to 3 years seen to slow ALS progression in trial

PrimeC safe, shows trends toward slowing ALS progression: Trial data

Top-line results of PARADIGM trial into PrimeC expected by year’s end

CHMP confirms its initial position against approving AMX0035 in EU

PARADIGM trial of PrimeC for ALS now fully enrolled with 69 adults