Executive Summary

Q2 2025 showed continued pipeline execution with early human PK data for DT-216P2 aligning with NHP profiles, the Phase 1/2 RESTORE-FA MAD trial underway ex-U.S., and initiation of a DT-168 Phase 2 biomarker trial in FECD; operating expenses increased as programs advanced .
EPS was -$0.34 vs Wall Street consensus of -$0.33 (3 estimates), a slight miss of $0.01; revenue remained at $0 consistent with consensus (2 estimates). Values retrieved from S&P Global.*
Cash, cash equivalents and investment securities were $216.3M at quarter-end, supporting continued advancement; net loss was $19.1M, driven by R&D of $15.7M and G&A of $5.8M .
Regulatory setback: FDA placed a clinical hold on the U.S. IND for DT-216P2 (starting dose concerns), while dosing continues outside the U.S.—a key near-term stock narrative catalyst alongside positive PK and FECD trial initiation .

What Went Well and What Went Wrong

What Went Well

Early human PK for DT-216P2 demonstrated favorable translation from NHPs with both IV and SC administration; human plasma exposure profiles were consistent with NHP data .
DT-216P2 exhibited improved exposure vs prior formulation (DT-216P1), including higher AUC and sustained plasma levels; thrombophlebitis seen with P1 is believed no longer limiting development .
FECD program advanced with initiation of a Phase 2 biomarker trial for DT-168 in patients scheduled for corneal transplant surgery; management emphasized progress across GeneTAC candidates: “We’ve made meaningful progress across our pipeline this quarter” (Pratik Shah, Ph.D.) .

What Went Wrong

FDA clinical hold on the U.S. IND for DT-216P2 (starting dose in the U.S.); introduces uncertainty and potential delay for U.S. enrollment, though ex-U.S. dosing continues in RESTORE-FA .
Operating expenses rose meaningfully YoY as programs moved into/through clinical phases (R&D $15.7M vs $10.5M; Total OpEx $21.6M vs $15.0M), widening net loss YoY (-$19.1M vs -$11.8M) .
No earnings call transcript available for Q2 2025; limited forum for real-time guidance clarifications and analyst Q&A this quarter (no transcript found in filings) [ListDocuments: earnings-call-transcript returned 0].

Financial Results

Metric	Q2 2024	Q1 2025	Q2 2025
R&D Expenses ($USD Millions)	$10.5	$15.4	$15.7
G&A Expenses ($USD Millions)	$4.5	$5.0	$5.8
Total Operating Expenses ($USD Millions)	$15.0	$20.4	$21.6
Other Income, net ($USD Millions)	$3.3	$2.7	$2.5
Net Loss ($USD Millions)	$11.8	$17.7	$19.1
Net Loss per Share (basic & diluted) ($USD)	$(0.21)	$(0.31)	$(0.34)

Balance Sheet	Mar 31, 2025	Jun 30, 2025
Cash, Cash Equivalents & Investment Securities ($USD Millions)	$229.7	$216.3
Total Assets ($USD Millions)	$237.5	$222.9
Total Liabilities ($USD Millions)	$9.6	$9.8
Stockholders’ Equity ($USD Millions)	$227.9	$213.0

Notes:

DSGN reports no product revenue; condensed statements present operating expenses, other income, and net loss .

Segment breakdown: Not applicable (no commercial revenue reported) .

KPIs (Operational)

KPI	Q1 2025	Q2 2025
DT-216P2 Phase 1 (SAD) HV status	Ongoing; favorable initial safety/systemic exposure profile	Early human PK consistent with NHP; improved exposure vs P1
RESTORE-FA Phase 1/2 (MAD) patient trial	Anticipated mid-2025 start	Underway ex-U.S.; first patient dosed IV; no AEs reported at initial dosing
DT-168 FECD	Plan Phase 2 biomarker trial in 2H 2025	Phase 2 biomarker trial initiated
Cash runway commentary	“Fund planned operating expenses into 2029”	Not updated; cash $216.3M

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
DT-216P2 RESTORE-FA MAD patient trial timing	2025	Anticipated to begin mid-2025	Underway ex-U.S. (ongoing dosing)	Raised (initiated)
DT-168 Phase 2 biomarker trial	2H 2025	Planned initiation in 2H 2025	Initiated	Raised (initiated earlier in Q2)
DT-216P2 U.S. IND status	2025	Submission planned	Clinical hold (starting dose concerns)	Lowered (regulatory headwind)
Operating runway	Multi-year	“Into 2029” with $229.7M cash at 3/31/25	Not updated; $216.3M at 6/30/25	Maintained (no formal update)

No revenue, margin, OpEx, OI&E, tax rate or dividend guidance provided in Q2 2025 materials .

Earnings Call Themes & Trends

Topic	Previous Mentions (Q1 2025)	Current Period (Q2 2025)	Trend
Regulatory/legal (FDA)	FECD Phase 1 favorable; planning MAD in FA; no U.S. IND commentary	FDA clinical hold on DT-216P2 U.S. IND (starting dose); plan to address with clinical/nonclinical data	Deteriorated (U.S. delay risk)
R&D execution (FA)	Phase 1 SAD HV ongoing; plan MAD in mid-2025	Early human PK favorable; RESTORE-FA MAD underway ex-U.S.	Improving (clinical progress)
R&D execution (FECD)	Phase 1 HV favorable; plan Phase 2 biomarker trial 2H 2025	Phase 2 biomarker trial initiated	Improving (advanced stage)
Cash runway/capital	$229.7M; runway into 2029	$216.3M; runway not restated	Slightly weaker (cash lower; runway unconfirmed)
Pipeline breadth (DM1/HD)	Targeting DM1 dev candidate selection in 2025; HD preclinical ongoing	DM1 candidate selection later in 2025; HD preclinical continues	Stable (timelines reiterated)

No earnings call transcript was available for Q2 2025; themes derived from press releases and 8-K exhibits .

Management Commentary

“We’ve made meaningful progress across our pipeline this quarter… Early human PK data for DT-216P2 demonstrate the consistency of human plasma exposure profiles with NHP data… We’re also pleased to have initiated our Phase 2 biomarker trial in patients with FECD…” — Pratik Shah, Ph.D., CEO .
On clinical hold: the company plans to address FDA’s request on U.S. starting dose with clinical/nonclinical data to initiate U.S. studies; dosing continues ex-U.S. .
On FECD biomarkers and DT-168: Phase 2 biomarker trial initiated to evaluate safety, tolerability, and corneal endothelium biomarkers .

Q&A Highlights

No Q2 2025 earnings call transcript available; no Q&A themes or guidance clarifications could be extracted this quarter [ListDocuments returned 0].

Estimates Context

Metric	Q2 2025 Consensus	Q2 2025 Actual
EPS ($USD)	-0.33*	-0.34
Revenue ($USD Millions)	0.00*	0.00 (no product revenue reported)

Values retrieved from S&P Global.*

Implications:

EPS modestly missed by $0.01; the variance aligns with higher OpEx as trials progressed (R&D +$5.2M YoY) .
Revenue expectations correctly captured as $0 given clinical-stage status .

Key Takeaways for Investors

Near-term narrative hinges on resolving FDA clinical hold for DT-216P2 U.S. IND; ex-U.S. dosing is ongoing, reducing program stoppage risk but delaying U.S. sites .
Positive clinical momentum: early human PK matched NHP, improved exposure vs prior formulation, and Phase 2 FECD biomarker trial initiated—de-risking technical profile across two lead programs .
Cash of $216.3M provides multi-year optionality, though runway into 2029 was not reiterated; watch quarterly burn as OpEx rises with broader clinical activity .
Expect OpEx to remain elevated as MAD patient dosing continues and FECD biomarker trial progresses; this is the primary driver of EPS volatility absent revenue .
Key 2026 catalyst: RESTORE-FA 12-week patient data (FXN expression) could be pivotal for DT-216P2’s path; monitor regulatory feedback timelines on the clinical hold .
No call transcript reduces visibility into timelines and regulatory dialogue; look for subsequent 8-K/press updates or 10-Q risk factor changes to track hold resolution .
Trading lens: balance positive PK/clinical initiation signals against regulatory overhang; headline risk from FDA updates may drive short-term moves, while sustained trial progress supports medium-term thesis .

Appendix: Source Documents Reviewed

Q2 2025 8-K and EX-99.1 press release with financials and program updates .
Q2 2025 additional press release: initiation of RESTORE-FA MAD (first patient dosed), FDA clinical hold notice on U.S. IND .
Q1 2025 8-K press release for prior quarter financials and program status .
Q4 2024 8-K press release for year-end baseline .

Design Therapeutics, Inc. (DSGN)·Q2 2025 Earnings Summary