View all newsletters
Receive our newsletter - data, insights and analysis delivered to you
  1. Comment
May 11, 2021

Orphazyme’s arimoclomol fails to show efficacy in a specific ALS population

On 7 May, Orphazyme, a Denmark-based late-stage biopharmaceutical company pioneering heat shock protein therapies for the treatment of rare diseases, announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS.

By GlobalData Healthcare

On 7 May, Orphazyme, a Denmark-based late-stage biopharmaceutical company pioneering heat shock protein therapies for the treatment of rare diseases, announced that the ORARIALS-01 pivotal trial of arimoclomol in amyotrophic lateral sclerosis (ALS) did not meet its primary and secondary endpoints to show benefit in people living with ALS. These topline data will be presented at the European Network to Cure ALS (ENCALS) meeting held virtually on 12–14 May, with complete data from the study expected to be published by the end of the year. While most drugs in development target both patient segments in ALS, namely familial (or hereditary) ALS (FALS) and sporadic ALS (SALS), arimoclomol specifically targets the FALS segment, which accounts for less than 10% of all cases of the disease.

The ORARIALS-01 pivotal trial was a randomised, placebo-controlled Phase III trial conducted among 245 patients in North America and Europe. Participants in the trial received either arimoclomol (248mg three times daily) or a placebo for up to 76 weeks. The primary endpoint, which was assessed by the combined assessment of function and survival (CAFS), determined the efficacy of chronic treatment with arimoclomol compared to the placebo in participants with the disease. Secondary endpoints included survival, change in ALS Functional Rating Scale-Revised (ALSFRS-R) and slow vital capacity (SVC). According to the topline data, no important safety signals were reported in the trial.

There is a large unmet need for a curative agent for the treatment of ALS. In the current treatment algorithm, patients have to take daily medication for the rest of their lives, meaning that a drug preventing the onset of the disease would be revolutionary. The only treatment options currently available are Rilutek (riluzole) and Radicava (edaravone), neither of which can stop or significantly slow the progression of the disease. At best, these drugs may increase a patient’s life span by up to six months. This need, therefore, is completely unmet by the current therapies.

There are multiple leading development strategies in ALS across the pharmaceutical industry. Exploring novel targets with mechanisms of action that differ from the two marketed drugs (Rilutek and Radicava) is the main strategy noticeable within the pipeline products. Other strategies include the use of biologic molecules to help discover a curative therapy.

Types of molecules being developed include stem cell therapies, monoclonal antibodies, antisense oligonucleotides and gene therapies. The hope behind these molecules is that they target the underlying mechanisms of the disease and slow, or potentially even stop, the progression of the disease. Cost is the key barrier to this strategy, however, with the cost of developing biologics far higher than that of developing small molecules and the chance of the drug showing clinical benefit remaining low.

Cell & Gene Therapy Coverage on Clinical Trials Arena supported by Cytiva.

Editorial content is independently produced and follows the highest standards of journalistic integrity. Topic sponsors are not involved in the creation of editorial content.

Free Whitepaper
img

Secure the cell therapy supply chain from bench to bedside

The development of cell therapies is changing healthcare, delivering new hope to thousands of patients around the world. The vein-to-vein workflow for these therapies, however, is not without challenges, many of which will increase as we scale up to treat more patients. Download this free guide from Cytiva to learn more about the challenges and risks associated with the cryogenic supply chain for cell therapies, and how supply chain disruptions can best be mitigated.
by Cytiva Thematic

By clicking the Download Free Whitepaper button, you accept the terms and conditions and acknowledge that your data will be used as described in the Cytiva Thematic privacy policy By downloading this Whitepaper, you acknowledge that we may share your information with our white paper partners/sponsors who may contact you directly with information on their products and services.

Visit our privacy policy for more information about our services, how we may use, process and share your personal data, including information on your rights in respect of your personal data and how you can unsubscribe from future marketing communications. Our services are intended for corporate subscribers and you warrant that the email address submitted is your corporate email address.

NEWSLETTER Sign up Tick the boxes of the newsletters you would like to receive. Key drug pipeline and competitive landscape changes based on the latest clinical activity, sent every Tuesday. Curated analysis and data-driven insights on clinical trials strategy and operations, sent every Thursday. The pharmaceutical industry's most comprehensive news and information delivered every month.
I consent to GlobalData UK Limited collecting my details provided via this form in accordance with the Privacy Policy
SUBSCRIBED

THANK YOU

Thank you for subscribing to Clinical Trials Arena