Thoughts on ATL1102 for DMD

Antisense's Duchenne's muscular dystrophy program has somehow snuck underneath investors noses and looks to be grossly undervalued. Here's what we know:

ATL1102 has achieved successful phase 2 results with a strong efficacy signal that is clinically significant.
- Performance of Upper Limb (PUL) 2.0 scores show a mean improvement of 0.9 points over 24 weeks compared to the typical decrease of 2.2 points / yr seen in a comparable group of non-ambulant patients. (1)
- 3/9 patients stabilised according to their PUL 2.0 score, with another 4/9 showing improvement, and 2/9 having disease progression.
- On ATL1102, Grip strength has increased by a mean value of 0.2kg, and pinch strength has stabilised showing no change. A study in European DMD non ambulant patients show a mean decrease in grip strength of 0.39kg and pinch strength of 0.08kg over a year. (2)

Looking at the overall picture, we see a drug with a well understood mechanism of action that looks to have a clinically meaningful impact on established endpoints (PUL 2.0, Myopinch). Essentially, these endpoints will be the same endpoints used in the registration trial.

At the current enterprise value of sub $20m, the risk-reward calculus is quite compelling. There are currently 3 FDA approved drugs for DMD selling in 2019.

Exondys-51 (2019 revenue of $381m USD)
Translarna (2019 revenue of $190m USD)
Emflaza (2019 revenue of $101m USD)

Essentially these are all $1-3bn drugs - and Antisense's ATL1102 is only one trial away from achieving the same outcomes both medically and from a economic value standpoint.

Now with AEF selling, it looks to be a good time to stock up.

Comments and criticisms welcome!

References:

1. Mayhew, A., Mazzone, E. S., Eagle, M., Duong, T., Ash, M., Decostre, V., ... & Bianco, F. (2013). Development of the Performance of the U pper L imb module for D uchenne muscular dystrophy. Developmental Medicine & Child Neurology, 55(11), 1038-1045.

2. Ricotti, V., Selby, V., Ridout, D., Domingos, J., Decostre, V., Mayhew, A., ... & Jansen, M. (2019). Respiratory and upper limb function as outcome measures in ambulant and non-ambulant subjects with Duchenne muscular dystrophy: A prospective multicentre study. Neuromuscular Disorders, 29(4), 261-268.