On the heels of promising HEALEY-ALS Phase 2 trial data, Prilenia Therapeutics plans to launch a pivotal Phase 3 study of pridopidine, its candidate amyotrophic lateral sclerosis (ALS) treatment, in the second half of this year.
Relative to a placebo, pridopidine was associated with slower disease progression, a better life quality, and a potential to prolong survival in patients in early disease stages. These recently diagnosed ALS patients with rapidly progressing symptoms saw particular benefit in terms of respiratory health and speech, although better speech was evident across all treated patients.
Prilenia reports it has completed discussions with global regulatory agencies regarding the next stage of pridopidines development.
Based on the clinical data generated to date, pridopidine has the potential to become a meaningful treatment for ALS, Michael R. Hayden, PhD, a physician, and CEO and founder of Prilenia, said in a company press release.
We believe it is important for Prilenia to advance our clinical program in ALS, and we are actively planning a single, global, pivotal Phase 3 clinical trial, Hayden said.
Pridopidine is an oral small molecule designed to selectively bind to and activate the sigma-1 receptor (S1R), a protein involved in several processes critical to the function and survival of nerve cells.
Essentially, pridopidine aims to slow ALS progression by boosting the clearance of toxic molecules, increasing energy production, reducing cellular stress, and limiting inflammation.
The HEALEY ALS (NCT04297683) platform trial is designed to simultaneously evaluate multiple experimental ALS treatments against a shared placebo group. It is being led by researchers at the Sean M. Healey & AMG Center for ALS at Massachusetts General Hospital in Boston.
The Phase 2/3 pridopidine arm (NCT04615923) enrolled 163 adults with clinically possible, probable, or definite ALS, whose symptoms had started within the last three years.
Participants were randomly assigned to oral pridopidine (45 mg) or a matched placebo taken twice daily for 24 weeks, or about six months. This was followed by an open-label extension study, where all participants received pridopidine.
The pridopidine trial failed to meet its main goal of demonstrating that treatment could slow functional decline relative to a placebo, reflected by changes in ALS Functional Rating Scale-Revised (ALSFRS-R) scores. Secondary goals related to muscle strength, respiratory function, and survival also were not met.
However, pridopidine led to significant improvements in certain speech measures, including speaking rate (how fast a person can speak) and articulation rate, or how quickly and clearly someone pronounces a word, after six months.
Additional analyses indicated that pridopidines use slowed ALSFRS-R declines in a subgroup of patients with definite ALS who were in an early disease course within 1.5 years of symptom onset.
These patients experienced a significant, 2.4-point slower decline in ALSFRS-R scores compared with those given a placebo. Pridopidine was particularly effective at slowing changes on the ALSFRS-R respiratory domain, mainly on questions related to shortness of breath (dyspnea).
Early ALS patients also experienced significantly slower declines in overall life quality compared with a placebo, with particular benefits in the eating/drinking and communication domains.
Even greater benefits were observed in the subset of probable or definite ALS patients who were early in the disease course but also considered to have fast-progressing disease. In this group, pridopidine was associated with a significant slowing of ALSFRS-R declines starting at week eight of treatment, and with a 5.2-point difference from a placebo after 24 weeks, corresponding to a 41% slower decline.
This subgroup of early, fast-progressing patients also showed the greatest improvements relative to a placebo in terms of changes on the ALSFRS-R respiratory domain, dyspnea, and speech.
Survival analyses spanning the main trial and its extension phase showed that pridopidine may increase survival time for those with definite or probable ALS in early disease phases.
Specifically, participants treated with pridopidine from the main trials start experienced a median survival of about 600 days (around 1.5 years), compared with a 300-day median survival for patients first randomized to a placebo, amounting to a 57% lower risk of death.
Pridopidine also was well tolerated with no serious treatment-related side effects reported.
Support for pridopidines continued development also may come from an ongoing expanded access program. This study, (NCT06069934), is enrolling about 200 ALS patients in the U.S. who are not eligible to participate in clinical trials of pridopidine, all of whom will receive the therapy for up to two years.
Our sincere appreciation to the many ALS patients, caregivers and healthcare providers who have contributed so much to our clinical studies as well as to those who will participate in future trials, Hayden said.
Pridopidine has been designated an orphan drug in the U.S. and Europe for the treatment of ALS, a status that intends to help speed its clinical development. Prilenia is also developing pridopidine for Huntingtons disease.
Read more:
Phase 3 clinical trial of pridopidine in ALS patients likely this year - ALS News Today
- Jake Diekman throws BP session for Rangers | MLB.com - MLB.com - August 6th, 2017 [August 6th, 2017]
- Cerebral palsy: Symptoms, diagnosis, treatment - December 5th, 2017 [December 5th, 2017]
- Hypothalamus - the body's thermostat | ASU - Ask A Biologist - December 6th, 2017 [December 6th, 2017]
- When Stroke Affects the Thalamus - Stroke Connection ... - December 28th, 2017 [December 28th, 2017]
- The Endocrine System: Hypothalamus and Pituitary - December 28th, 2017 [December 28th, 2017]
- Hypothalamus - Anatomy, Blood supply and Function | Kenhub - December 28th, 2017 [December 28th, 2017]
- Ulcerative Colitis - Cedars-Sinai - January 6th, 2018 [January 6th, 2018]
- Ulcerative colitis - Treatment - NHS.UK - January 7th, 2018 [January 7th, 2018]
- Business Directory | Uptown Waterloo Business Improvement Area - January 8th, 2018 [January 8th, 2018]
- Will States Continue To Fund Stem Cell Research? : Shots ... - January 28th, 2018 [January 28th, 2018]
- Spinal Cord Injuries - emedicine.medscape.com - February 15th, 2018 [February 15th, 2018]
- Spinal Cord Injuries - spine.org - February 15th, 2018 [February 15th, 2018]
- Journal of Clinical & Experimental Cardiology - Open Access Journals - April 10th, 2018 [April 10th, 2018]
- Cardiology Treatment - Fortis Healthcare - April 10th, 2018 [April 10th, 2018]
- Biology Junction - April 12th, 2018 [April 12th, 2018]
- Managing Right Ventricular Failure in PAH: An Algorithmic ... - April 14th, 2018 [April 14th, 2018]
- Ticagrelor vs Clopidogrel After Fibrinolytic Therapy in ... - April 22nd, 2018 [April 22nd, 2018]
- Cardiovascular Pharmacotherapeutics: William H. Frishman ... - May 13th, 2018 [May 13th, 2018]
- 13 Super Foods Good for Eczema - Top Eczema Treatments - May 15th, 2018 [May 15th, 2018]
- Diagnosis & Treatment of Parkinson's - ParkinsonsDisease.net - May 30th, 2018 [May 30th, 2018]
- Arizona Heart Rhythm Center - June 10th, 2018 [June 10th, 2018]
- Ayurvedic Medicines for Ulcerative Colitis | Natural ... - June 21st, 2018 [June 21st, 2018]
- Weinstein Cardiovascular Development and Regeneration Meeting - June 30th, 2018 [June 30th, 2018]
- Ulcerative Colitis Information Centre - July 31st, 2018 [July 31st, 2018]
- Sudden Death in Dogs - Pet Health Network | Pet Health ... - August 7th, 2018 [August 7th, 2018]
- Coronary Heart Disease - Ischaemic heart disease - British ... - August 21st, 2018 [August 21st, 2018]
- Cardiac Rehabilitation: Overview, History and Definition ... - August 25th, 2018 [August 25th, 2018]
- Difference between Cardiac Arrest and Respiratory Arrest ... - August 25th, 2018 [August 25th, 2018]
- ECM for Pericardial Closure - Aziyo - August 25th, 2018 [August 25th, 2018]
- Adult Mental Health | Georgia Department of Behavioral Health ... - September 6th, 2018 [September 6th, 2018]
- Mental Health | ECLKC - September 6th, 2018 [September 6th, 2018]
- Mental Health - The Church of Jesus Christ of Latter-day Saints - September 6th, 2018 [September 6th, 2018]
- Center for Vascular Biology - Home | UConn Health - September 22nd, 2018 [September 22nd, 2018]
- # Diabetes Stem Cell Research Diabetes Care Measures - September 30th, 2018 [September 30th, 2018]
- Cardiac Rhythm Disturbances | The Patient Guide to Heart ... - November 2nd, 2018 [November 2nd, 2018]
- Arrhythmia | Cleveland Clinic - November 2nd, 2018 [November 2nd, 2018]
- Hyperbaric Oxygen Therapy for Wound Healing | Johns ... - November 13th, 2018 [November 13th, 2018]
- NIMH Home - November 24th, 2018 [November 24th, 2018]
- What is an interventional cardiologist? | Heart Surgeries ... - November 26th, 2018 [November 26th, 2018]
- Molecular Cardiology Research Institute Boston | Tufts ... - November 29th, 2018 [November 29th, 2018]
- Lower vascular plant | botany | Britannica.com - December 7th, 2018 [December 7th, 2018]
- Welcome to the ABG Tutorial Welcome to Hansen - December 12th, 2018 [December 12th, 2018]
- Cardiac catheterization - Wikipedia - December 12th, 2018 [December 12th, 2018]
- Stem Cell Research & Therapy | Home page - December 22nd, 2018 [December 22nd, 2018]
- Journal of Stem Cell Research and Therapy- Open Access ... - December 22nd, 2018 [December 22nd, 2018]
- Stem-cell therapy - Wikipedia - December 22nd, 2018 [December 22nd, 2018]
- Cardiology and Interventional Cardiology - December 23rd, 2018 [December 23rd, 2018]
- Rhode Island Interventional Cardiology | Cardiovascular ... - December 23rd, 2018 [December 23rd, 2018]
- Interventional Cardiology Certification Policies | ABIM.org - December 23rd, 2018 [December 23rd, 2018]
- Interventional Cardiology - Boston Scientific - December 23rd, 2018 [December 23rd, 2018]
- 9 Benefits of Human Growth Hormone (Along with Side ... - December 27th, 2018 [December 27th, 2018]
- Growth hormone deficiency - Wikipedia - December 27th, 2018 [December 27th, 2018]
- Dr. Lorrie Kirshenbaum, Cardiac Gene Biology | St ... - January 28th, 2019 [January 28th, 2019]
- Cardiomyopathy - CardioSmart - February 8th, 2019 [February 8th, 2019]
- Centenary Institute | Medical Research, Life Saving Research - February 24th, 2019 [February 24th, 2019]
- Cardiovascular Medicine | Department of Medicine - March 29th, 2019 [March 29th, 2019]
- Rapid Test Market 2019 with Recent Trends, Revenue, Demand and Top Manufactures- Bureau Veritas, SGS SA, Intertek Group plc, Eurofins Scientific -... - October 11th, 2019 [October 11th, 2019]
- Medical Imaging Equipment Market Research Report 2019: Global Industry Analysis, Business Development, Size, Share, Trends, Future Growth, Forecast To... - October 11th, 2019 [October 11th, 2019]
- Analysis on the Global DNA Read, Write & Edit Market, 2017-2019 and Forecast to 2024 - Yahoo Finance - October 11th, 2019 [October 11th, 2019]
- Cardiac Surgery Instruments Market is Expected to Tread a Steady Growth Trajectory by Clocking a CAGR of 5.9% from 2017 to 2022 - Health News Office - October 24th, 2019 [October 24th, 2019]
- PEDIATRIC MEDICAL DEVICES MARKET IS PROJECTED TO EXPAND AT A CAGR OF 8.0% FROM 2018 TO 2026 - Health News Office - November 1st, 2019 [November 1st, 2019]
- myTAIHEART Test Provides Evidence for Injury from Biopsy of Heart Transplant Recipients Cardiology2.0 - Cardiology2.0 - November 1st, 2019 [November 1st, 2019]
- Mental health and your heart - British Heart Foundation - November 19th, 2019 [November 19th, 2019]
- BUZZ-U.S. STOCKS ON THE MOVE-Cango, Gulfport Energy, Abiomed, Standard Diversified, Wanda Sports, Eidos - Nasdaq - November 19th, 2019 [November 19th, 2019]
- Cost Analysis of COPD Exacerbations and Cardiovascular Events in SUMMIT - AJMC.com Managed Markets Network - November 19th, 2019 [November 19th, 2019]
- Hundreds walk to defeat ALS in Ancient City - St. Augustine Record - November 22nd, 2019 [November 22nd, 2019]
- Living with Lung Cancer: The Silver Linings - Curetoday.com - November 22nd, 2019 [November 22nd, 2019]
- Building the human spinal cord atlas - SynBioBeta - November 22nd, 2019 [November 22nd, 2019]
- The Week That Wasn't: Keto and the Flu, Human Cyborg, Duvet Disease - Medscape - November 22nd, 2019 [November 22nd, 2019]
- ALS advance offers hope for those diagnosed with deadly disease - WRAL.com - November 22nd, 2019 [November 22nd, 2019]
- ALS Stem Cell Therapy Developer Seeks Amendment to its AstroRx Trial - ALS News Today - November 22nd, 2019 [November 22nd, 2019]
- Former Boston Globe writer, current NHL executive Snow diagnosed with ALS | College Hockey - USCHO - December 23rd, 2019 [December 23rd, 2019]
- AB Science announces the publication of new results in the peer-reviewed journal Glia that further support masitinib's potential mode of action in ALS... - December 23rd, 2019 [December 23rd, 2019]
- Christmas in the City thrills homeless families, takes on poignancy with founders ALS diagnosis - The Boston Globe - December 23rd, 2019 [December 23rd, 2019]
- Amylyx Pharmaceuticals Announces AMX0035 Demonstrated Statistically Significant Treatment Benefit for People with ALS in the CENTAUR Trial - Business... - December 23rd, 2019 [December 23rd, 2019]
- RADICAVA (edaravone) - December 23rd, 2019 [December 23rd, 2019]
- List of Amyotrophic Lateral Sclerosis Medications (5 Compared ... - December 23rd, 2019 [December 23rd, 2019]
- Neuropore Completes Phase 1 Clinical Trial in Healthy Volunteers with NPT520-34, a Therapeutic Candidate Aimed at Treating Parkinson's Disease and... - January 14th, 2020 [January 14th, 2020]
- Comprehensive Analysis on Prostacyclin Market based on types and application - Technology Magazine - January 18th, 2020 [January 18th, 2020]
- N.J. karate legend who inspired thousands loses battle with ALS - NJ.com - January 18th, 2020 [January 18th, 2020]