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Biotech / Medical : Immunomedics (IMMU) - moderated
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jhcimmu
To: Olecranon who wrote (63133)6/12/2025 1:16:27 AM
From: ghettogoulash1 Recommendation  Read Replies (1) of 63284
 
SRPT: A couple of corrections. Elevidys already has Full Approval in ambulatory patients (able to walk) ages 4+. Accelerated Approval was for non-ambulatory patients ages 4+. Also, the confirmatory trial for AA in non-ambulatory is not expected to read out until 2027.

A couple of salient points from CEO Doug Ingram at the BoA conference last month:

-- Sarepta will be profitable and cash flow positive this year.

-- In it's registrational trial for Limb-Girdle Muscular Dystrophy, SRP-9003 hit the primary endpoint. The company will be filing a BLA for AA later in 2025. (Data will be shared at an unnamed medical conference.)

Note: This is the first and most significant of three single-arm trials treating three different mutations of LGMD. Ingram expects approval of each subgroup in 2026, 2027, and 2028. Interestingly, Sarepta is running a concurrent study on LGMD patients with no drug arm to serve as a historical control arm. This is an "external" control the FDA, in theory, will accept as placebo in rare diseases with no Standard of Care.

Chatgpt's valuation model for the three mutations/subtypes of LGMD:



I asked Chatgpt for clarification: Sarepta is leveraging the same gene therapy delivery chassis (AAVrh74) but tailoring each therapy to express the specific sarcoglycan gene that’s missing in that subtype of LGMD. So: same vector, different payload — and each one is a unique biologic drug.
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