Further Win For Isis's Antisense Pipeline Adds To Buyout Rationale

Isis Pharmaceuticals (ISIS) has long maintained that it intends to develop its triglyceride-lowering agent ISIS-APOCIIIRx without outside assistance, and a recent $170m equity offering will help it get part of the way there. The drug's startlingly positive phase II results revealed on Saturday ought to prompt generous partnership offers, though, and everyone has their price.

These presumed overtures may not be enough to induce the company to depart from its stated plans. But every clinical success further validates the antisense platform on which Isis has built its company, and with a pipeline featuring 18 candidates in phase II or III, Isis itself is starting to look like a takeout target.

Drastic move

Though highly positive, the data, presented at this week's meeting of the European Society of Cardiology in Amsterdam, are early and the trial size small. Top-line results of the monotherapy part of the ongoing 28-patient study showed that the highest dose of the drug, 300mg weekly for 13 weeks, caused a 79% drop in levels of ApoC-III and a 75% fall in triglyceride levels.

Taken alone these results would be encouraging but not conclusive. Added to earlier phase II results, though, a picture of an effective drug starts to take shape. The patients in this study had very high to severely high triglyceride levels, showing the drug to work well in a new population following its success in patients with type 2 diabetes and high triglycerides (ADA – Knockdown result boosts Isis and antisense alike, June 25, 2013).

Isis is now keen to push into phase III, saying that it will discuss plans with regulators and start a trial in patients with severely high triglycerides next year. The company has shown no reluctance to partner its other late-stage drugs; its flagship, Kynamro, was licensed to Sanofi (SNY) in phase III. But management has shown no signs of deviating from its intention to go it alone, despite the magnitude of the undertaking.

If big pharma is really keen to get hold of ISIS-APOCIIIRx, a more drastic move may be necessary. Isis's market cap of $3bn, while not chickenfeed exactly, would present no major problems if Sanofi or one of Isis's other partners – GlaxoSmithKline (GSK) or Biogen Idec (BIIB), for example – wished to acquire a company whose platform is looking increasingly promising.

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Risk of autism in further children - study findings

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Academic Journal
Main Category: Autism
Also Included In: Pediatrics / Children's Health
Article Date: 20 Aug 2013 - 0:00 PDT Current ratings for:
Risk of autism in further children - study findings

A large population-based study from Denmark has followed siblings for the risk for autism spectrum disorders, finding different likelihoods depending on birth year, and also whether brothers or sisters were half- or full-siblings.

The study uses records of all children born in Denmark between 1980 and 2004. It is the first study of its kind, say the authors, to follow such a large number of children - around 1.5 million - and to consider the "recurrence risk" of autism spectrum disorders (ASD) for both full- and half-siblings.

The study - published online in JAMA Pediatrics by researchers from Aarhus University - compared children who had an older sibling with ASD against those whose older sibling did not have ASD.

According to the authors, 30% of all ASD cases are childhood autism, and the prevalence of autism spectrum disorders has increased over the past 20 years.

The study shows that between 1980 and 2004, the recurrence risks for ASDs ranged between 4.5% and 10.5%, higher than the risk of autism spectrum disorders found across the Danish population, of 1.18%.

Additionally, the researchers found there was an almost seven-fold greater risk for an autism spectrum disorder if an older sibling had an ASD diagnosis, compared with families in which the older sibling did not have a disorder.

For children with the same mother, the recurrence risk was 7.5% for full-siblings and 2.4% for half-siblings.

For children with the same father, the recurrence risk was 7.4% for full-siblings, but the researchers not that there was "no statistically significant increased risk" among half-siblings.

The authors note that the reason the risk is higher for half-siblings who share a mother may be due to the fact that they share genes from their mother, and they also share "exposures derived from their mother's intrauterine environment and perinatal history" across her different pregnancies.

An important issue to address from the study, say the researchers, is that parents who have a child with an ASD may choose not to have any more children. This phenomenon is known as stoppage, and they say it may result in an underestimate of the recurrence risk.

The authors conclude the study by saying:

"The difference in the recurrence risk between full- and half-siblings supports the role of genetics in ASDs, while the significant recurrence risk in maternal half-siblings may support the role of factors associated with pregnancy and the maternal intrauterine environment in ASDs."

The results should be reassuring to parents who have a child with an ASD if they are thinking of having other children, note the researchers, because the recurrence risk they found is "substantially lower than recent reports from smaller clinic-based populations."

?Researchers from Duke University recently linked induced labor and autism risk.

How do parents navigate care for their children with disabilities and other needs? Here's a detailed analysis: Getting support for children with disabilities.

Written by Marie Ellis

Copyright: Medical News Today
Not to be reproduced without permission of Medical News Today Visit our autism section for the latest news on this subject.

“Recurrence of Autism Spectrum Disorders in Fulland Half-Siblings and Trends Over Time,” Therese K. Grønborg, et al., JAMA Pediatrics, doi:10.1001/jamapediatrics.2013.2259, 19 August 2013.

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