Ionis Plans to Advance Angelman drug Biogen with clinical success

Ionis Pharmaceuticals is set to advance one of its drugs for Angelman syndrome to the third trial phase after proving its efficacy in an earlier trial. Angelman syndrome is a rare genetic disorder that leads to significant impairment of functional and learning abilities.

Ionis dosed ION582 across 51 participants and tested three different doses for its effect. The company stated that the innovative treatment was mostly safe and that the majority of the treated patients tolerated it well, showing a “profound and replicable gain” in communication, cognition, and motor functions.

Researchers used four scores to assess the impact of ION582. One of the scores established that the symptoms of Angelman syndrome were alleviated in 97% of individuals who received a medium or high dose of the drug. Additionally, the gains in cognition, communication, and motor control were significantly greater than those recorded in natural history studies featuring Angelman patients similar to those used in the trial.

With these results in hand, Ionis plans to engage with drug regulators to discuss the blueprint of a pivotal Phase III trial, which the company hopes to commence in the first half of the following year.

Ionis focuses on developing “antisense oligonucleotides,” a type of drug that interferes with the molecular signals the body requires to synthesize proteins. To date, it has commercialized five standalone antisense drugs, two of which were co-created with Biogen. Notably, Biogen recently decided against licensing ION582.

Now, Ionis is trying to market ION582 as a potential unpartnered product to strengthen its position in the industry. The drug is now ready to “be at the forefront of Ionis’ next generation of disruptive, wholly owned therapies for neurological disorders,” noted Brett Monia, the chief executive officer of Ionis.

Data from Ionis indicate that Angelman syndrome prevalence ranges from 1 in 8,000 to 1 in 21,000 persons. This condition occurs when a gene on the maternal chromosome ceases to function properly, affecting a protein that informs a cell which proteins require degradation. This enzyme also plays important roles in the growth and operation of the nervous system.

ION582 is designed to increase the synthesis of the enzyme by interacting with RNAs that regulate the temporal synthesis of the gene located on the paternal chromosome. In a normal cell, that gene is ‘off’ in the brain, but with this Ionis drug, it will turn ‘on’.

The information can now be viewed as a clear positive for Ionis, according to a note for clients written by William Blair analyst Myles Minter. Its Phase III plans place it just within striking distance of Ultragenyx Pharmaceutical, which is developing another potential treatment that might advance to late-stage trials this year.

“Though the development timeline of Ionis is largely similar to a peer program from Ultragenyx, the company could be differentiated based on the safety perspective, making ION582 an attractive asset for future growth, as there is no approved treatment for the target indication currently,” Minter added.

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