TSHAMEDIUM SIGNALOPERATIONAL10-K

TSHA completed enrollment in both REVEAL trials for Rett syndrome treatment and reported positive developmental milestone data, while financial losses deepened amid expanded R&D investment.

The completion of enrollment in both pediatric and adolescent REVEAL trials represents a significant operational milestone, with positive clinical data showing patients gaining developmental milestones across communication and motor functions. However, the company's burn rate has accelerated with substantially higher R&D spending, indicating increased investment in advancing their gene therapy pipeline toward potential regulatory approval.

Comparing 2026-03-19 vs 2025-02-26View on EDGAR →
FINANCIAL ANALYSIS

TSHA's financial picture reflects a company in active clinical development, with revenue growing modestly to $9.8M while R&D expenses increased meaningfully to $86.4M. Operating losses widened to $110.5M as the company invested heavily in advancing its clinical programs, and operating cash outflow increased to $93.1M. The financial trajectory suggests intensified development activity as trials progress toward potential pivotal studies.

FINANCIAL STATEMENT CHANGES
Capital Expenditure
Cash Flow
+97.3%
$374K$738K

Capital expenditure jumped 97.3% — major investment cycle underway; assess returns on deployment.

Interest Expense
P&L
+31.6%
$3.8M$5.0M

Interest expense surged 31.6% — significant debt increase or rising rates materially impacting earnings.

R&D Expense
P&L
+30.9%
$66.0M$86.4M

R&D investment increased 30.9% — signals commitment to future product development, though near-term margin impact.

Net Income
P&L
-22.1%
-$89.3M-$109.0M

Net income declined 22.1% — review whether driven by operations, interest costs, or non-recurring items.

Operating Income
P&L
-20.8%
-$91.5M-$110.5M

Operating profitability softening — costs rising faster than revenue, watch for margin recovery plan.

Revenue
P&L
+17.3%
$8.3M$9.8M

Revenue growing 17.3% — solid top-line momentum, watch margins for quality of growth.

Operating Cash Flow
Cash Flow
-14.6%
-$81.2M-$93.1M

Operating cash flow softened — monitor whether temporary working capital timing or structural deterioration.

LANGUAGE CHANGES
NEW — 2026-03-19
PRIOR — 2025-02-26
ADDED
As of March 19, 2026, the registrant had 287,269,885 shares of common stock, $0.00001 par value per share, outstanding.
We are evaluating TSHA-102 for the treatment of females with Rett syndrome in our REVEAL and ASPIRE clinical trials.
A total of six participants aged 15 to 21 years were treated with TSHA-102 in this study, and enrollment for this trial is complete.
Part A also includes the REVEAL Phase 1/2 Pediatric Trial, which is a first-in-human, open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 as a single lumbar intrathecal administration in pediatric females aged 5 to 8 years with Rett syndrome due to MECP2 loss-of-function mutation.
A total of six participants aged 6 to 8 years were treated with TSHA-102 in this study, and enrollment for this trial is also complete.
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REMOVED
As of February 26, 2025, the registrant had 205,001,632 shares of common stock, $0.00001 par value per share, outstanding.
We are also evaluating TSHA-102 in the REVEAL Phase 1/2 pediatric trial, which is an open-label, randomized, dose-escalation and dose-expansion study evaluating the safety and preliminary efficacy of TSHA-102 as a single lumbar intrathecal administration in pediatric females with Rett syndrome due to MECP2 loss-of-function mutation.
The trial is taking place in the United States, Canada and the United Kingdom.
As of the February 17, 2025 data cutoff, TSHA-102 was generally well tolerated, with no treatment-related serious adverse events, or SAEs, or dose-limiting toxicities, or DLTs, in the 10 patients dosed in Part A of the REVEAL trials.
We believe this maturing dataset continues to support advancement toward the Part B registrational trial.
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