To The Moon

$Ultragenyx Pharmaceutical(RARE)$ $Spdr S&P Biotech Etf(XBI)$  $iShares Biotechnology ETF(IBB)$  🧬🎯 Brittle Endpoint, Resilient Helix 🎯🧬

When a price cut sharpens the aim, not the thesis

🚨📰 Just in: $RARE proves you can put the target on a diet without starving the thesis.

🏦 Wells Fargo cut its price target on $RARE to $45 from $65 while maintaining Overweight. I read this as probability discipline, not loss of conviction. At roughly $19.72, the revised framework still implies about 128% upside, reframing the move as risk calibration rather than thesis erosion.

✂️ The reset followed Ultragenyx’s setrusumab miss in osteogenesis imperfecta, where Phase 3 Orbit and Cosmic trials failed to meet the primary endpoint of fracture reduction. That said, statistically significant gains in bone mineral density were observed, leaving room for salvage pathways rather than outright abandonment. Wells Fargo lowered OI probability to 33%, yet argues the stock is already trading near its non-OI floor, with just $1–2 per share of optionality still priced in. Expectations have been de-risked, not amputated.

📉 The market response was severe, a ~43% single-session drawdown that erased over $1B in market capitalisation. Management has since signalled meaningful expense reductions, aiming to rein in cash burn and target profitability by 2027. That matters for durability, not headlines.

👀 Street caution widened but conviction did not evaporate. Beyond Wells Fargo, multiple firms trimmed targets while largely retaining constructive ratings. Consensus remains Moderate Buy, with mean targets still far above spot, underscoring how sharply valuation has compressed relative to long-term pipeline value.

🧠 The derivatives market is calmer than the equity tape. I’m seeing a bullish risk reversal extending to Jan 2027, with over $500k in premium committed. That positioning reflects patience underwriting recovery, not fast money chasing volatility.

🧬 The pipeline is where the structure still holds.

🧠 GTX-102 for Angelman Syndrome is fully enrolled for Phase 3 Aspire, with topline data expected H2 2026. The programme carries Breakthrough Therapy designation and targets paternal UBE3A reactivation, a mechanism with the potential to materially improve cognitive and motor outcomes. Wells Fargo values GTX-102 alone at roughly $20 per share on a 50% probability, which is almost the entire current stock price quietly making its own case.

🧪 UX111 for Sanfilippo syndrome type A (MPS IIIA) remains active following a Complete Response Letter tied to manufacturing, not efficacy. Biomarker data continues to support clinical relevance, and resubmission targeting 2026 keeps this asset firmly in play rather than shelved.

🔄 So yes, the target was trimmed. But the genome-level thesis remains intact. This looks less like a shattered paradigm and more like a mosaic mid-assembly, volatile in the middle, coherent in design.

⚖️ Biotech rarely moves in straight lines. It moves in probabilities, patience, and pipeline maths. On that score, $RARE still looks appropriately named.

💉📊 Cross-asset context: $XBI $IBB

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Trade like a boss! Happy trading ahead, Cheers, BC 📈🚀🍀🍀🍀

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