Scholar Rock (SRRK) Goldman Sachs 45th Annual Global Healthcare Conference summary

Strategic vision and platform differentiation

• Focused on harnessing TGF-β biology with a highly selective approach, targeting unmet needs in SMA and obesity.

• Selectivity in targeting precursor forms of growth factors avoids toxicity and enables safer therapies.

• Robust pipeline includes anti-myostatin programs and TGF-β1 targeting for immuno-oncology and fibrosis.

• Global rights and strong IP protection support commercial ambitions.

• Next 12-24 months expected to be transformative, with key inflection points ahead.

Lead program in spinal muscular atrophy (SMA)

• Apitegromab targets muscle directly, addressing unmet needs not covered by current SMN-targeted therapies.

• Phase II TOPAZ study showed additional functional gains on top of nusinersen, with improved strength and reduced fatigue.

• Over 90% of patients remain on treatment, with no serious adverse events, reflecting strong safety and perceived benefit.

• Phase III SAPPHIRE trial nearing completion, with Q4 readout expected; BLA preparation underway.

• Plans to expand to younger patients and launch in US and Europe, aiming for over $1 billion in revenue.

Commercialization and patient engagement

• Building commercial infrastructure with efficient footprint (30-50 FTEs), targeting US and Europe for initial launch.

• Strong engagement with patient advocacy groups and unbranded campaigns to amplify patient voice.

• Seamless integration into current practice, with monthly dosing and compatibility with existing therapies.

• Focus on world-class customer support and optimizing patient experience.