RPRX Royalty Pharma

tividenofusp alfa · NCT06075537 registry update

countries: {'total_sites': 29, 'us_sites': 8} → {'total_sites': 30, 'us_sites': 8}

pelacarsen · NCT07625306 registry update

status: NOT_YET_RECRUITING → WITHDRAWN; primary_completion: 2028-12-07 → 2029-01-22; completion: 2028-12-07 → 2029-01-22; enrollment: 240 → 0; why_stopped: → Study terminated by sponsor following the outcome of the Lp(a)HORIZON study (NCT04023552), which did not meet its primary endpoint

Zenas BioPharma presents new obexelimab Phase 3 OLE data in IgG4-RD at IgG4 Symposium

New data from the Phase 3 INDIGO open-label extension showed sustained flare-free probability (0.93/0.87) and low glucocorticoid use with obexelimab in IgG4-RD. Additional analyses demonstrated reduced glucocorticoid toxicity versus placebo and preserved vaccine responses after a treatment-free window. These results add to clinical evidence for obexelimab as a reversible, steroid-sparing B-cell inhibitor.

pelacarsen · NCT06813911 registry update

status: RECRUITING → ACTIVE_NOT_RECRUITING; enrollment: 340 → 246; countries: {'total_sites': 95, 'us_sites': 36} → {'total_sites': 94, 'us_sites': 35}

Royalty Pharma: Novartis pelacarsen HORIZON Phase 3 misses primary endpoint

Novartis’ Phase 3 HORIZON outcomes trial for pelacarsen failed to meet its primary composite cardiovascular endpoint versus placebo. Royalty Pharma will not make any milestone payments to Ionis and its Spinraza royalty interest will revert to Ionis after $550 million in payments, yielding a 1.1x return on the $500 million investment.

Zenas: FDA accepts obexelimab BLA for IgG4-RD; PDUFA May 27, 2027

FDA accepted the obexelimab BLA for IgG4-RD with a PDUFA target action date of May 27, 2027. The acceptance triggers a $30 million milestone payment under the Xencor agreement and positions the company for potential U.S. approval and subsequent EMA and PMDA filings. The Phase 3 INDIGO data supporting the BLA showed a 56% reduction in flare risk versus placebo.

Denali sells Rare Pediatric Disease PRV for $195 million

Denali monetized the PRV awarded after AVLAYAH’s accelerated approval. The $195 million proceeds will fund further development of its TransportVehicle pipeline in lysosomal storage and neurodegenerative diseases. No new regulatory milestone or timeline is announced.

Zenas BioPharma submits BLA for obexelimab in IgG4-RD

Zenas BioPharma has submitted a BLA to the FDA for obexelimab in IgG4-RD, supported by positive Phase 3 INDIGO trial data. The submission advances the asset from clinical development to regulatory review. The company highlighted the statistically significant 56% reduction in flare risk (HR 0.44, p=0.0005) and favorable safety profile.

Zenas BioPharma: Phase 3 INDIGO obexelimab data at EULAR 2026

The company will present full safety and efficacy results from the registrational Phase 3 INDIGO trial of obexelimab in IgG4-RD at the EULAR 2026 Congress on June 4, 2026. The trial already met its primary endpoint and all four key secondary endpoints with high statistical significance. This marks the first public disclosure of the detailed Phase 3 data package.

Zenas BioPharma plans obexelimab BLA submission for IgG4-RD this quarter

The company confirmed its marketing applications for obexelimab in IgG4-RD remain on track, with a BLA submission to the FDA expected this quarter and an MAA to the EMA in H2 2026. Phase 3 INDIGO trial results will be presented at EULAR 2026 on June 4.

Denali: FDA approves AVLAYAH (tividenofusp alfa) for Hunter syndrome

FDA granted accelerated approval for AVLAYAH to treat neurologic manifestations of Hunter syndrome in pediatric patients ≥5 kg. The approval marks the first medicine using transferrin receptor to cross the blood-brain barrier. Commercial launch is underway with first patients treated in April 2026.

Denali Therapeutics Inc. · FDA approves Avlayah (tividenofusp alfa-eknm)

FDA lists Avlayah (tividenofusp alfa-eknm) as novel drug approval #8 of 2026 on 2026-03-24: To treat certain individuals with Hunter syndrome (Mucopolysaccharidosis type II or MPS II)

Zenas BioPharma plans obexelimab BLA submission for IgG4-RD in Q2 2026

Following positive Phase 3 INDIGO results, Zenas now expects to submit a BLA to FDA in Q2 2026 and an MAA to EMA in H2 2026 for obexelimab in IgG4-RD. The filing timeline is based on the registrational trial meeting its primary and all key secondary endpoints with high statistical significance.

Denali: tividenofusp alfa PDUFA set for April 5, 2026

The BLA for tividenofusp alfa has a PDUFA target action date of April 5, 2026 under the accelerated approval pathway. Denali has established commercial launch readiness ahead of this date. The ongoing Phase 2/3 COMPASS study is expected to provide confirmatory evidence for global regulatory submissions.

Denali: FDA sets April 5, 2026 PDUFA for tividenofusp alfa BLA

The document states the FDA has assigned a PDUFA target action date of April 5, 2026 for the BLA of tividenofusp alfa under the accelerated approval pathway. This sets the regulatory decision timeline for the first potential commercial launch of a Denali TV-enabled therapy.

Zenas reports positive Phase 3 INDIGO topline for obexelimab in IgG4-RD

The Phase 3 INDIGO trial met its primary endpoint with a 56% reduction in IgG4-RD flare risk (HR 0.44, p=0.0005) and all four key secondary endpoints. Zenas now plans to file a BLA with the FDA in Q2 2026 and an MAA with the EMA in H2 2026.

Royalty Pharma: FDA approves Cytokinetics' MYQORZO (aficamten) for oHCM

FDA approved the NDA for MYQORZO (aficamten) in symptomatic obstructive hypertrophic cardiomyopathy. Royalty Pharma now earns a 4.5% royalty on sales up to $5 billion and 1% thereafter, plus 1.9x repayment on $275 million drawn capital over 10 years. Cytokinetics may draw an additional $175 million within 12 months, triggering further 1.9x repayments.

Denali Therapeutics 2025 Investor Day – ETV franchise launch timeline

The 4 Dec 2025 Investor Day presentation states that tividenofusp alfa (DNL310) regulatory filing is imminent and commercial launch is planned for 2026, with DNL126 (ETV:SGSH) launch targeted for 2027. These timelines and the company’s stated $1 B+ market opportunity for the ETV franchise constitute the key regulatory and commercial milestones disclosed in the document.

Denali: FDA sets April 5, 2026 PDUFA for tividenofusp alfa BLA

The press release discloses that Denali’s BLA for accelerated approval of tividenofusp alfa is under FDA review with a PDUFA target action date of April 5, 2026. The $275 million royalty funding agreement is contingent on FDA accelerated approval and EMA approval by December 31, 2029. This establishes a concrete regulatory timeline for the asset’s potential U.S. approval.

Denali: FDA extends tividenofusp alfa PDUFA to April 5, 2026

The FDA classified Denali’s updated clinical pharmacology submission as a Major Amendment, extending the PDUFA target action date for accelerated approval of tividenofusp alfa in MPS II by three months from January 5, 2026 to April 5, 2026. No new efficacy, safety or biomarker data were requested; the extension is procedural only. Denali states the amendment does not alter the clinical pharmacology or benefit-risk conclusions of the BLA.

Denali: FDA extends PDUFA date for tividenofusp alfa BLA to April 5, 2026

The FDA extended the PDUFA action date for Denali's BLA seeking accelerated approval of tividenofusp alfa in MPS II from January 5, 2026 to April 5, 2026. The extension was triggered by the agency's classification of updated clinical pharmacology data as a Major Amendment to the application.