Ultragenyx Pharmaceutical Inc (NASDAQ:RARE) Fails In A Mid stage, Sources Say


Ultragenyx Pharmaceutical Inc (NASDAQ:RARE), one of the top Biotech Stocks, has come forward to make the announcement about its most recent failure. The drug which it had hoped would do much to treat complications failed in the mid stage of the study and that was quite frustrating to all those scientists that had delved into the study and the company in general. The drug by the name UX007 had earlier on led the company in investing as well as directing much in terms of efforts and resources in seeking out the cure for the condition.

The phase 2 study gave kits data for evaluation. It was meant to help seek top solutions for handling patients with transporter type-1 deficiency syndrome .After the tedious task, the drug miserably failed to reach the primary endpoints thus rendering it inappropriate to serve the targeted patients’ needs.

The latest trial was seeking to try and identify the drug’s efficacy.

Some patients had been placed in a placebo to help come to a good conclusion. A comparison was made with this group where the results showed that there was no major difference in the various patients on UX007 during the eight week assessment period.

It is important to point out that the drug treatment showcased a decrease in absence seizures, as captured on EEG when the different seizures were evaluated independently. The drug did pretty well on safety and tolerability considering that there were no cases of death reported. There were also no adverse effects that arose fro the treatments.

Glut1 DS happens to be a nervous system impacting disorder which usually results from a genetic defect that occurs following the transportation of glucose to the various brain tissues. There are usually a lot of neurological signs and symptoms as well that come with the condition. They include epilepsy, seizures and the delayed neurological development.

Basically, a seizure presents itself as a sudden surge of electrical activity in the brain and its effects are adverse. Any affected person behaves differently and acts different for some time. UX007 operates by converting to new glucose and that helps patients obtain the necessary therapeutic benefits especially on those instances when the glucose levels decline a huge deal. It is not the end of the road for this company because it will continue developing some top solutions. Incase something great comes up; it will be aired out in the Biotech Stock News.


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