Notes From The Road... Could SLDB Be The Golden Tempo Of Gene Therapy?

Research evidence excerpt

Notes From The Road... Could SLDB Be The Golden Tempo Of Gene Therapy?

● SGT-212: Underappreciated update still to come by YE26. Management noted they completed

SGT-212 dosing in two Friedreich's ataxia (FA) subjects already with early efficacy signals that

were described as unexpected. The first patient had heavy disease burden at baseline (e.g.

no speaking, swallowing or reading) and post-treatment is starting to see restoration of some

function. This coincides with an uneventful adverse event profile. SLDB anticipates completing

dosing in ~5 subjects by YE26 with a data update. SLDB also noted they have two sites with ~100

patients interested in securing treatment with SGT-212. We take this demand signal as a positive

sign and do think the initial clinical data readout could deliver an important valuation inflection.

● SGT-212: What does good look like? For our part, we continue to leave SGT-212 as upside to

our estimates as our valuation derives solely from the SGT-003 program. While the two patients

dosed so far provide an interesting sample, even SLDB management commented that they did

not think there was recovery potential with treatment. The bar here remains Skyclarys which

demonstrated a -2.41-point change on mFARS at week 48. Management noted that recent data

from Design Therapeutics (DSGN, not covered) could be interesting conceptually as a potential

agent that could work synergistically with SGT-212; however, this remains early in conception.

● SGT-212: Keeping the regulatory strategy simple might be best. If SGT-212 demonstrates a

1-2-point improvement it reduces the need to focus on changes in frataxin as a surrogate

endpoint. Instead, it may be more appropriate to advance with a traditional approval focused

on a functional outcome like mFARS.