The catalysts worth watching this month

• Sep 19, 2026

  RAREUltragenyx PharmaceuticalMust watch

  PDUFA decision for UX111 gene therapy in MPS IIIA (Sanfilippo syndrome type A) — resubmitted BLA

  Mucopolysaccharidosis type IIIA (Sanfilippo syndrome type A)

  Ultragenyx is back in front of the FDA with UX111, its AAV9 gene therapy for Sanfilippo syndrome type A — a fatal pediatric neurodegenerative disease with no approved treatment. The FDA issued a CRL in July 2025 over manufacturing (CMC) issues, not efficacy; the resubmitted BLA was accepted in April 2026 with a September 19 action date.

  UnverifiedCheck itSearch ClinicalTrials.govSEC 8-K filings

  NDA/BLA FiledPDUFA

• Sep 22, 2026

  IONSIonis PharmaceuticalsMust watch

  PDUFA decision for zilganersen in Alexander disease

  Alexander disease (AxD)

  Ionis' antisense oligonucleotide zilganersen is under priority review for Alexander disease, a progressive and often fatal genetic leukodystrophy with no approved treatment. The pivotal study met its primary endpoint, showing statistically significant stabilization of gait speed at week 61, with supportive motor-function results in children ages 2-4.

  UnverifiedCheck itSearch ClinicalTrials.govSEC 8-K filings

  NDA/BLA FiledPDUFA

• Sep 2026

  expected window

  AMLXAmylyx Pharmaceuticals

  Phase 3 LUCIDITY topline data for avexitide in post-bariatric hypoglycemia

  Post-bariatric hypoglycemia (PBH)

  Amylyx — rebuilding after the Relyvrio withdrawal — reads out its Phase 3 LUCIDITY study of avexitide, a GLP-1 receptor antagonist for post-bariatric hypoglycemia, guided for Q3 2026. PBH affects a meaningful share of the growing bariatric-surgery population and has no approved therapy.

  Company-confirmedCompany disclosureSEC 8-K filingsEstimated window, not a confirmed dateVerified 2026-09-05

  Phase 3Data Readout

• Oct 2026

  expected window

  CRSPCRISPR Therapeutics

  CTX112 Phase 1 initial data

  B-cell malignancies

  CRISPR Therapeutics is presenting initial data from a Phase 1 trial of CTX112, an allogeneic CAR-T cell therapy for B-cell malignancies. This is an early-stage readout, but it's significant because CTX112 is designed to improve on existing CAR-T therapies by enhancing efficacy and reducing side effects. The data will show safety and preliminary efficacy in patients who have failed other treatments.

  UnverifiedCheck itSearch ClinicalTrials.govSEC 8-K filingsEstimated window, not a confirmed date

  Phase 1Data Readout

• Oct 2026

  expected window

  NTLAIntellia TherapeuticsMust watch

  Potential accelerated approval filing for NTLA-2002 in HAE

  Hereditary angioedema (HAE)

  Intellia is expected to file for accelerated approval of NTLA-2002, a CRISPR-based therapy for hereditary angioedema, a rare genetic disorder causing severe swelling attacks. This filing is based on promising Phase 1/2 data showing durable reduction in attacks. If approved, it would be the first CRISPR therapy for a non-blood disease, marking a major milestone.

  Company-confirmedCompany disclosureSEC 8-K filingsEstimated window, not a confirmed dateVerified 2026-09-05

  NDA/BLA FiledData Readout

• Oct 2026

  expected window

  NBIXNeurocrine Biosciences

  Phase 3 data for NBI-1065845 in MDD (first trial)

  Major Depressive Disorder (MDD)

  Neurocrine is reporting Phase 3 data for NBI-1065845, an investigational drug for major depressive disorder. This is the first of two pivotal trials, and positive results would support a new treatment option for the millions of patients who don't respond to existing antidepressants. The drug works by a novel mechanism, which could differentiate it from current therapies.

  UnverifiedCheck itSearch ClinicalTrials.govSEC 8-K filingsEstimated window, not a confirmed date

  Phase 3Data Readout

Every tracked event, with filters by significance, phase, and type, is on the full catalyst calendar — also free.

What Pro adds

The dates are free. The analysis is the product.

Anyone can find a PDUFA date. What loses people money is not knowing what the trial has to show, or which risk nobody put in the press release. Pro adds three layers to every entry above.

See the format applied end to end in the sample Pro memo.