Biogen (BIIB) and Ionis Pharmaceuticals’ (IONS) Spinraza has been witnessing improvement in infrastructure and payer access for all types of spinal muscular atrophy (or SMA) patients in the US in 2017. The number of sites administering the drug to patients in the US has risen from 88 in 1Q17 to 145 in 2Q17 and 180 at end of 3Q17. Further, around 250 sites had also submitted start forms for initiaing patients on Spinraza therapy at the end of 3Q17. Additionally, Biogen has also received thousands of start forms from SMA patients, which highlights the strong demand trends for Spinraza.
At the end of 2Q17, approximately 80% of the commercially insured people in the US had access to Spinraza. Further, approximately two-thirds of the commercially covered population had significantly broad access to the drug. Biogen also managed to secure access for two-thirds of those covered by Medicaid, while 60% had broad access to the drug at the end of 2Q17. This improved access has played a major role in increasing the total number of patients on Spinraza therapy by 75% in 3Q17 on a quarter-over-quarter basis. The consistent improvement in the sales metrics of Spinraza could help boost royalty payments for Ionis Pharmaceuticals in 2017. Notably, Ionis Pharmaceuticals accounts for 0.23% of the Vanguard Small-Cap ETF’s (VB) total portfolio holdings.
The above diagram shows results from the phase three trial, CHERISH, which demonstrated the efficacy of Spinraza in improving motor function in children with later onset SMA, which is generally either Type 2 or Type 3 SMA. This was a 15-month study involving 126 non-ambulatory children between two to 12 years of age that experienced SMA symptoms at or above six months of age.
Unlike untreated children who showed a one-point decline in their Hammersmith Functional Motor Scale Expanded (or HFMSE) scores, a tool used to measure motor function in SMA patients, those who were treated with Spinraza saw a 3.9-point improvement over the 15-month period.
Owing to its efficacy and safety profile, Spinraza is expected to enable Ionis Pharmaceuticals to compete effectively with other rare disease players such as United Therapeutics (UTHR) and Alexion Pharmaceuticals (ALXN) for investor attention.
In the next article, we’ll discuss market expansion plans for Spinraza in greater detail.