Waylivra and the way forward, page-4

'"So scientifically I would imagine moving forwards it would be a matter of getting the correct dosing to avoid the low platelet count"

Thanks Sam,
The problem of low platelet count may be symptomatic of ASOs. In fact, the safety profile of Spinraza includes the observation that thrombocytopenia has been observed after administration of some ASOs and that patients may be at risk of bleeding complications. The complication of low platelet counts, however, wasn't fatal for drug approval. Its all a question of establishing a favourable benefit-risk profile. Spinraza is regarded as a therapeutic breakthrough not only in spinal muscular atrophy but for antisense oligonucleotides. In the early years, it was widely claimed that antisense is nonsense. Today, post Spinraza, antisense is now embraced by scientists and investors as the answer for some devastating genetic diseases.

Returning home to ATL1102. The FDA conservatively approved a 25 mg/week dosage for ATL1102 in a Phase IIb in MS. Is it significant that the decision was made pre-Spinraza? There wasn't much love for antisense during 2014. Spinraza wasn't approved until late 2016. ANP will presumably have data for platelet counts in its current clinical trial in DMD, using a dosage rate of 25 mg/week. The approach by the European regulatory authorities is keenly anticipated and will inform the market of the potential for ATL1102 to treat inflammation in DMD at potentially a higher dosage rate. Remember, Waylivra is prescribed for use at 285 mg/week.

The side effect of low platelet counts will be subject to enhanced monitoring to support early detection and management. Its a question of establishing a favourable benefit-risk profile for the use of ATL1102 in DMD vis-à-vis corticosteroids.