- Scholar Rock SRRK shares dropped despite the company reporting positive 12-month data on spinal muscular atrophy (SMA) candidate apitegromab.
- The 12-month update was broadly in line with the six-month data that was announced in October last year. Most effects observed at six months have proven to be durable at 12 months.
- In some cases, the efficacy results improved between the six and 12-month readouts.
- A cohort of children with type 2 SMA aged two years and older who began treatment with Biogen Inc BIIB / Ionis Pharmaceuticals Inc’s IONS Spinraza (nusinersen) before turning five years old showed a mean improvement of 7.1 points on a motor scale after receiving 20-mg/kg infusions of apitegromab on top of their existing therapy.
- In the 2 mg/kg dose arm, an improvement of 5.3-point was reported.
- At six months, the improvement was 5.6 points and 2.4 points in 20 mg/kg and 2 mg/kg groups, respectively.
- 35% of the low and high-dose arm participants experienced at least a 10-point improvement on the motor function scale at 12 months. 59% of patients had a 5-point increase.
- The two other cohorts lacked such evidence of enhanced responses.
- In patients with type 2 or non-ambulatory type 3 SMA who began treatment with Spinraza aged five years or older, the mean increase on the motor scale at 12 months was 1.2 points. It excludes a patient who missed three apitegromab doses due to COVID-19 restrictions. At six months, the mean increase across the full cohort was 1.4 points.
- A third cohort saw a worsening of the scores from six to 12 months. On another disease scale, the ambulatory type 3 SMA patients improved by 0.5 points after six months.
- By 12 months, the group had experienced a mean decline of 0.3 points.
- Numerical declines in the proportion of patients with three and five-point improvements were tracked.
- Scholar Rock is now gearing up to evaluate apitegromab through a Phase 3 study set to start by the end of the year.
- Price Action: SRRK shares are down 14.3% at $38.68 during the market trading session on the last check Tuesday.
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