Enzyme replacement therapy clinical trials 

Allievex (formerly Biomarin) ERT for Sanfilippo Type B
Allievex is conducting clinical trials of an enzyme replacement therapy (ERT) for Sanfilippo Type B with sites in Germany, Spain, Taiwan, Turkey, USA and the UK.

The early trials of the ERT called “BMN-250” were conducted by Biomarin, but in October 2019 a licencing deal was made with a newly-formed biotechnology company, Allievex Corporation and the therapy was renamed "Tralesinidase Alfa”.
This ERT is administered weekly directly into the cerebrospinal fluid of the brain (ICV) via a port implanted under the scalp. In February 2018 Dr Nicole Muschol from Germany presented preliminary results of the trial at the WORLD Symposium. She reported that six children had been treated in the trial at different dose levels.
Heparan sulfate was shown to decrease into the normal range in the cerebrospinal fluid and participants livers reduced to normal size. At the highest dose there was indication of stabilisation of cognitive ability, but it is still early days and further evidence will need to be gathered to prove the benefit of this therapy. The treatment was generally well tolerated but there were some side effects, mostly due to the invasive way that the therapy is delivered.


The trial is no longer recruiting, however the trial is still ongoing and continuing to collect data. Allievex is preparing to go to the FDA to apply for regulatory approval of this ERT.

More information about the trial.


SOBI's SOB1003 for Sanfilippo Type A 

SOBI, a biopharmaceutical company based in Stockholm, is conducting a clinical trial of an Enzyme Replacement Therapy product which is called "SOBI003". There are trial sites in the USA, Germany and Turkey. The first patient was dosed in August 2018.


SOBI003 is a version of the enzyme that is missing in Sanfilippo Type A which has been chemically modified so that it lasts for longer inside the body, giving it more of a chance to get inside cells and do its job of breaking down GAGs. 
In June 2019 SOBI announced that it is now looking to divest SOBI003, so the future of this program is uncertain.


For more information visit SOBI's website and 


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