Timbo, I’ll offer my thoughts, but please be aware that much of...

Timbo, I’ll offer my thoughts, but please be aware that much of the scientific stuff goes right over my head!

What are the leading possibilities for PYC’s ASO program, if not DMD?

I’d certainly consider Spinal muscular atrophy (SMA) for the following reasons:

• PYC, in collaboration with Murdoch University, has already completed animal model experiments in SMA which demonstrated preliminary signs of efficacy, lack of toxicity and delivery of ASOs to a range of locations. (Refer PYC Update, Dec 2016)
• Murdoch University has a collaborative research agreement with Murdoch to establish the Sarepta Translational Laboratory, led by Profs. Steve Wilton and Sue Fletcher. The intention of the collaboration is to explore the applicability of PMOs for disease targets beyond Duchenne. Two example targets mentioned in a 2015 press release were cystic fibrosis and SMA.

The most common form of SMA (SMN related SMA) is caused by a deficiency of a motor neuron protein called SMN, a protein expressed in cells which is necessary for the survival of motor neurons. That deficiency is caused by a genetic defect in the SMN1 gene.

There is only one approved drug that I know of which treats SMA - Biogen’s Spinraza (nusinersen), which was approved by the FDA in December 2016. However, AveXis, a company recently acquired by Novartis, filed for FDA approval of its gene therapy for SMA last month, for which approval is expected in H1, 2019.

Spinraza is an ASO drug which is injected into the spine once a month and is already considered a blockbuster drug, having passed the US$1bn annual sales threshold in the September quarter this year. Doubtlessly Sarepta would like a piece of that market.

Just my thoughts… perhaps they’ll go for something totally under the radar!