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

This issue covers

  • Weekend Wall (Sat, Jan 31): $AQST ( 0.0% ) + $PHAR ( ▲ 1.68% ) PDUFAs — because the action date is Saturday, expect the outcome to most commonly print Fri after-close and/or Mon pre-market (Feb 2).

  • High-impact risk shift: $RGNX ( ▲ 0.74% ) — FDA placed clinical holds on RGX-111 and RGX-121 (tumor-signal investigation). This is now the dominant variable into the Feb 8 setup.

  • Visibility events: $NERV ( ▲ 5.34% ) Feb 3 KOL; $XCUR ( ▼ 1.43% ) Feb 5 (6:30 PM local) poster; $TARA Feb 27 (Fri) ASCO GU Poster Session B.

  • Process carry: $CAPR ( ▲ 7.75% ) — FDA requested full HOPE-3 CSR; company guiding Feb 2026 BLA update → new PDUFA clock likely after acceptance.

Market mood

This is a timing-risk tape. Late-cycle uncertainty is trading like fundamentals: deficiency letters, holds, and amendments are getting priced harder than usual—especially in gene therapy after the RGNX hold headline.

Weekend PDUFA cluster (Sat, Jan 31)

Jan 31 is a Saturday. Expect outcomes to most commonly hit Fri after-close (Jan 30) and/or Mon pre-market (Feb 2) depending on issuer disclosure cadence.

$AQST ( 0.0% ) — Anaphylm (epinephrine sublingual film) | PDUFA: Jan 31

  • PoA (desk band): 30–40 (process risk elevated)

  • Core risk: On Jan 9, FDA notified the company it identified deficiencies that preclude labeling discussions at that time. Review remains ongoing, but this materially raises the probability of CRL and/or an extension versus a clean approval. (Aquestive)

  • Watch for: language implying major amendment / extension vs a straight action-date outcome.

$PHAR ( ▲ 1.68% ) — Joenja (leniolisib) pediatric sNDA (ages 4–11, APDS) | PDUFA: Jan 31

  • PoA (desk band): ~85

  • Setup: Priority Review sNDA for age expansion of an already-approved drug (12+), with target action date Jan 31, 2026. (Pharming Group N.V.)

  • Watch for: dosing/safety label specifics more than approvability.

New / changed since last Friday

$RGNX ( ▲ 0.74% ) — Clinical hold now dominates the Feb 8 setup

  • Update (Jan 28): FDA placed clinical holds on RGX-111 and RGX-121 after a brain tumor was detected in a child treated years earlier in the RGX-111 program; FDA cited potential shared risk across the programs. Company says it has not yet received the full hold letter.

  • Implication: Even if Feb 8 remains on the calendar, the risk regime changed—assume headline volatility + timeline uncertainty until FDA specifics are known.

$KZIA ( ▼ 2.26% ) — “before month-end” trigger is complete

  • Kazia released its paxalisib Phase 1b TNBC clinical update on Jan 27 (no longer a pending “any-day” item).

$CAPR ( ▲ 7.75% ) — process catalyst unchanged

  • FDA requested full HOPE-3 CSR; company expects Feb 2026 BLA update → new target date likely assigned after acceptance/processing.

Calendar (next 2–4 weeks)

Weekend timing note: Saturday/Sunday dates often produce Fri after-close / Mon pre-market disclosure bias.

FDA action dates (PDUFAs)

Tradable visibility events

Closing note

The weekend setup is simple:

  • Jan 31: binary outcomes for $AQST ( 0.0% ) / $PHAR ( ▲ 1.68% ) (with $AQST carrying explicit late-cycle process risk).

  • Post-weekend: attention rotates to $RGNX, but now through clinical-hold mechanics, not a clean “weekend PDUFA” play.

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

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

Keep Reading

No posts found
© 2026 PDUFA Pulse.
Report abusePrivacy policyTerms of use
beehiivPowered by beehiiv