Closest to market!!!, page-3

The two closest to bringing a drug to market
There are two small caps currently engaged in phase III trials: Neuren Pharmaceuticals (ASX:NEU) and Telix Pharmaceuticals (ASX:TLX).

Neuren announced just last week that its North American licensee ACADIA Pharmaceuticals had kicked off a phase III trial of its drug trofinetide in patients with Rett syndrome in the United States.

Rett syndrome is a rare genetic brain disorder that affects brain development in females.

It currently has no cure and is treated mainly focusing on improving function and addressing symptoms, which can include problems with language, coordination, and repetitive movements, slower growth, smaller head size, seizures, scoliosis and sleeping problems.

In phase II trials, trofinetide was shown to achieve statistically significant improvement compared with placebo on three of five syndrome-specific efficacy measures, in 82 girls with Rett aged between five and 15.

The phase III trial and its associated costs are fully funded by ACADIA and Neuren will commercialise trofinetide outside North America if successful.

Meanwhile, Telix in August reported that it had dosed the first patient in the phase III trial of its drug TLX250-CDx, designed to help detect renal cancer.



The share-moving phase II results
Several small cap biotechs have read out results from phase II trials, which has unsurprisingly resulted in their shares flying — as they all now look to either find a partner to fund third-phase trials or figure out how to afford it themselves.


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