Amyotrophic Lateral Sclerosis (ALS): An area of unmet medical need
ALS is without an effective therapeutic treatment.
- Globally, an estimated 220,000 people suffered from ALS in 2015, and that number is projected to increase by 69% to over 370,000 by 2020
- In the past 20 years, 50 controlled trials, testing ~60 molecules have failed
- There are no diagnostics to predict who will respond to drugs in ALS clinical trials
- There has been no clinical trial success for common sporadic ALS patients
BSP’s Antisense Oligonucleotide (AO) Molecule Focus
Our therapeutic development strategy is driven by the application of genetic science to identify drug targets and design synthetic antisense RNA-based therapeutics. Our lead ALS candidate (BSP0001; SOD1ex3PMO) has a target clinical indication for delay or prevention of ALS progression. BSP0001 is an antisense oligonucleotide (AO), a Phosphorodiamidate Morpholino Oligomer (PMO) which is a novel class of therapeutic suitable for ALS patients and employs an “exon skipping” mechanism of action directed at the activity of the superoxide dismutase (SOD1) gene. Toxic gain of function resulting from mutations in the SOD1 gene have been extensively investigated, and there is strong scientific rationale that reducing the amount of SOD1 protein is likely to rescue motor neurons and slow or halt ALS progression in selected ALS subsets.
BSP0001 employs the “exon skipping” mechanism of action and this methodology has been used in the successful development of the PMO therapeutic Exondys 51 (Eteplirsen) which received accelerated FDA approval in 2016 for the treatment of Duchenne Muscular Dystrophy (DMD). PMOs have been proven to be both safe and effective molecules, and Exondys 51 has dramatically improved the health and wellbeing of DMD patients. These molecules are much more resistant to degradation than other AOs and, therefore, are not only likely to be more potent, but should have a longer therapeutic half-life in the CNS.
In addition to BSP0001 our company is developing a portfolio of AO molecules for a broader range of ALS targets.