Spinal Muscular Atrophy; SMA; Spinal Muscular Atrophy Type 2
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Estimated readout windows: registry primary-completion date + 6-12 weeks. Windows already open are shown from "Now". Drug names link to ClinicalTrials.gov.
Status note Phase 3 SAPPHIRE in SMA (October 2024). BLA submitted January 2025 (priority review); CRL September 2025; resubmitted; FDA accepted with September 30, 2026 PDUFA. Long-term extension ONYX ongoing. A subcutaneous formulation completed a Phase 1 study in healthy volunteers; further development activities ongoing, including planned FDA/EMA regulatory engagements following potential apitegromab approvals.
Status note Subcutaneously administered anti-pro/latent myostatin antibody. Phase 1 study in healthy volunteers underway.
SRK-181
Phase 1 · ActiveModality: monoclonal antibody
Target latent TGFβ1
Indications
Locally advanced or metastatic solid tumors resistant to anti-PD-(L)1 therapies(Phase 1)
Status note Phase 1 DRAGON trial in urothelial carcinoma, melanoma, NSCLC, ccRCC and head and neck SCC completed 2025. Phase 2-ready; company currently seeking partnerships.
SRK-373
Preclinical · ActiveModality: monoclonal antibody
Target latent TGFβ1
Indications
Fibrotic diseases(Preclinical)
Status note Preclinical development. Company currently seeking partnerships.
SRK-256
Preclinical · ActiveModality: monoclonal antibody
Target RGMc (hemojuvelin)
Indications
Iron-restricted anemias(Preclinical)
Status note Preclinical development. Company currently seeking partnerships.
Per-asset detail extracted from the 10-Q filed 2026-08-06. Fields the filing does not state are marked "not disclosed" rather than filled from other sources.
Sponsor-matched active trials from ClinicalTrials.gov (4 as sponsor, 2 as collaborator); "Sponsor" = company is the lead sponsor, "Collaborator" = company is a collaborator. The BP column flags big-pharma involvement as lead sponsor or collaborator (excluding the company itself). The Mgmt guide column shows what management guided for readout timing when a guided program matches the trial (hover for the quote, click for the source). Trial listings are not a company pipeline slide.
Institutional holder positions from 13F filings. Q/Q change is vs the prior quarter's filing; % of portfolio is the fund's own reported portfolio weight.
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