Regenxbio Inc (RGNX)

Mucopolysaccharidosis Type II (MPS II) · registered primary completion (date passed, results not yet posted) 2026-09-01 · NCT05693142

Published 2026-09-02Market cap $638M9 sourced statements

AFFINITY DUCHENNE: RGX-202 Gene Therapy in Participants With Duchenne Muscular Dystrophy (DMD) · PHASE2,PHASE3 · n=65 · active not recruiting

What this sheet is scored on

call

Cash of $106M against operating outflow of $42M per quarter gives roughly 2.5 quarters of runway. On these figures the company raises capital within four quarters.

Source: 10-Q filed 2026-08-06

Balance sheet

fact

Management has concluded there is substantial doubt about the company's ability to continue as a going concern.

Source: 10-K filed 2026-03-05

“t 12 months. Our existing cash, cash equivalents, and marketable securities may not be sufficient to fund our operating expenses and capital requirements for at least the next 12 months from the issuance date of our financial statements. As a result, there is substantial doubt about our ability to continue as a going concern. Drug development is a lengthy, expensive, and uncertain process, and our product candidates …”

Market

fact

1 approved product already carry Mucopolysaccharidosis Type II (MPS II) on label, including AVLAYAH. Approval would enter an occupied market.

Source: FDA label index

Trial registry

fact

Registered design for NCT05693142: non-randomized, open-label, 65 participants (estimated). Primary outcome: Part 1 Safety measured by incidence of Adverse Events and Serious Adverse Events. 1 further primary outcome(s) registered.

Source: ClinicalTrials.gov record

Share count and dilution

fact

66,013,192 shares outstanding against 80,061,192 if every outstanding option, warrant and convertible were exercised. In that case 17.5% of a gain in enterprise value would accrue to those holders rather than to current shareholders.

Source: antidilutive-securities disclosure, 10-Q filed 2026-08-06

fact

Share count up 31% year over year to 66,013,192 as of 2026-07-31.

Source: cover-page share counts (XBRL)

Regulatory

fact

Designations disclosed: RMAT, Breakthrough Therapy, Fast Track, Orphan Drug, Priority Review Voucher. These accelerate review and, for Orphan, confer exclusivity if approved; none is evidence of efficacy.

Source: 10-Q filed 2026-08-06

Pipeline

fact

2 distinct molecules in 3 active company-sponsored trials; RGX-121 accounts for 2 of them at Phase 2/Phase 3.

Source: ClinicalTrials.gov, company as lead sponsor

Ownership and proceedings

fact

Directors and executive officers as a group hold 14.22%.

Source: DEF 14A filed 2026-04-14

Don't see a name? Request a sheet. Any US-listed biotech with a Phase 3 or a pending FDA decision — it gets built the same way, from the filings.

This sheet is dated and will not be edited. When the registered primary completion passes, its outcome is recorded on the scoreboard and a new dated sheet is published if the filings change materially.