PoA = probability-of-approval band (directional only; not FDA guidance).

This issue covers

  • New desk note (process): $CAPR ( ▲ 7.75% ) deramiocel BLA — FDA requested the full HOPE-3 Clinical Study Report (CSR); company guiding BLA updates in Feb 2026, after which a new PDUFA target should be assigned.

  • Weekend Wall setup: Jan 31 is a Saturday ($AQST ( 0.0% ) , $PHAR ( ▲ 1.68% ) ) — expect Fri after-close or Mon pre-market outcomes.

  • Late-Jan “visit-completion” zone: $KOD ( ▼ 0.33% ) — management continues to guide topline in 1Q 2026; primary endpoint visits are expected to wrap late January, but topline remains a Q1 event.

  • Early Feb docket: $RGNX ( ▲ 0.74% ) PDUFA Feb 8 (Sunday) + $XCUR ( ▼ 1.43% ) Tandem poster Feb 5 (6:30 PM local).

  • High-alert “any day” signal: $KZIA ( ▼ 2.26% ) expects a clinical/translational update before month-end (paxalisib Phase 1b TNBC).

  • Quick context: $AXSM ( ▲ 0.79% ) still guiding AXS-12 NDA submission in January; separately, AXS-05 (Alzheimer’s agitation) PDUFA is April 30, 2026 (not the focus this week, but relevant for positioning).

Market mood

This is a lighter tape for hard-dated catalysts, and the market is still hypersensitive to “process extensions” (labeling stops, amendment clocks, CSR requests). Net: traders are pricing timeline risk more aggressively than usual, even when the underlying clinical story is intact.

Desk notes (new / worth carrying)

$CAPR ( ▲ 7.75% ) — Deramiocel BLA (process update)

  • FDA has requested the HOPE-3 CSR; company expects to submit BLA updates in February 2026.

  • Practical implication: new PDUFA clock likely follows once the Feb update is accepted/processed. This is the archetypal “process extension” tape is reacting to.

$KZIA ( ▼ 2.26% ) — Paxalisib Phase 1b TNBC (timing trigger)

  • Company has guided a clinical + translational update before the end of January.

  • Treat as high-alert next-week item: any-day release risk into month-end positioning.

$OSTX ( ▲ 1.44% ) — Floor note (if you’re tracking the BLA path)

  • Positive Phase 2b biomarker update (Jan 15) is worth holding as the “fundamental floor” headline into the BLA submission watch.

Late-January + February Catalyst Calendar

Weekend timing note: for Saturday/Sunday PDUFAs, expect “press release limbo” (Fri after close or Mon pre-market).

FDA Action Dates (PDUFAs)

  • $AQST ( 0.0% ) — Jan 31 (Sat)PoA: 40%
    Anaphylm (epinephrine sublingual film): FDA letter (Jan 9) flagged deficiencies that preclude labeling discussions for now; review is ongoing. This is primarily a process/timeline risk setup.

  • $PHAR ( ▲ 1.68% ) — Jan 31 (Sat)PoA: 85%
    Leniolisib pediatric sNDA (ages 4–11) for APDS: Priority Review; still binary on label specifics, but structurally higher-conviction than most.

  • $RGNX ( ▲ 0.74% ) — Feb 8 (Sun)PoA: 65%
    RGX-121 (MPS II / Hunter syndrome): extended goal date; apply Weekend Wall logic.

  • $ETON ( ▲ 1.45% ) — Feb 25PoA: 85%
    ET-600 (desmopressin oral solution): hard-dated FDA target.

  • $ASND ( ▲ 0.48% ) — Feb 28PoA: 85%
    TransCon CNP (navepegritide) in achondroplasia: hard-dated FDA target.

*Weekend timing: action/news may land Fri, Jan 30 after close or Mon, Feb 2 pre-market (for the Jan 31 cluster); similarly Fri, Feb 6 / Mon, Feb 9 behavior is plausible for Feb 8.

Non-PDUFA “tradable” dates / visibility events

  • $XCUR ( ▼ 1.43% ) — Feb 5 — 6:30 PM (Tandem Meetings; Abstract #29283)
    Burixafor (GPC-100) + propranolol with G-CSF mobilization poster.

  • $KOD ( ▼ 0.33% ) — Late Jan (visit-completion zone) → 1Q topline
    Track for “data readiness” messaging, but keep expectations anchored: topline is still guided as 1Q.

  • $KZIA ( ▼ 2.26% ) — “Before month-end” (any day)
    Paxalisib TNBC update expected before Jan 31.

What changed vs last week (quick audit)

Tools we use: Affiliate disclosure: Market Chameleon (options/IV + unusual activity). We may earn a commission at no extra cost to you: [Market Chameleon]

Disclaimer
This newsletter is for informational purposes only. Biotech securities involve substantial risk, including total loss of capital. Always conduct your own due diligence.

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