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New clinical trial in A-T by biopharmaceutical company Intrabio to start soon

IntraBio Inc. is a biopharmaceutical company developing novel treatments for common and rare neurodegenerative diseases. The company has a drug, known as IB1001s, which in a number of pre-clinical and compassionate use studies appears to have made a significant improvement to the symptoms of people with a number of inherited ataxias, including A-T.

The company is now planning to carry out a proper clinical trial to test the effectiveness of the drug in treating A-T and other neurological disorders including Tay Sachs and Nieman Pick type C.

The drug was granted orphan drug designation for ataxia-telangiectasia by the US regulatory body the FDA in October 2018. ‘Orphan drug status’ is granted to drugs for rare conditions which might otherwise not be cost-effective, and offers incentives to companies to develop them.

Recently, with the support of the William Davis and Julie Greenfield of the A-T Society, Intrabio also applied for orphan drug designation from the European Medicine Agency. We are currently awaiting confirmation of the outcome of this application.

On February 11th Intrabio announced that they had received approval from the FDA to start a trial in Tay Sachs and Sandhoff disease. They have also applied for two multinational clinical trials involving IB1001 for the treatment of A-T, and Niemann-Pick disease Type C (NPC).

They are hoping enrolment in all three studies will begin later in 2019 and that there will be a centre for the A-T study in the UK. People already involved in the EryDex trial will not be able to take part in this trial, but as the inclusion criteria are less stringent, it may be possible for people who were not able to participate in the EryDex trial to take part in this one. More information on the A-T trial can be found on the ClinicalTrials.gov website.

William Davis, Chief Executive of the A-T society, says: ‘We warmly welcome the interest of Intrabio in testing their drug in people with A-T and have been supporting the company with their programme. It is looking hopeful that there will be a trial site in the UK and that UK A-T patients will have the opportunity to take part. It is extremely positive that the first ever international clinical trial of a potential treatment for A-T should be so quickly followed by another.’ More information to follow.