Executive Summary

Q2 2024 was execution-heavy with positive clinical updates across all three programs and a fortified balance sheet; cash, cash equivalents, and marketable securities were $500.9M, extending operating runway well into 2027 .
R&D and G&A rose year over year as the portfolio advanced; net loss was $(26.4)M and diluted EPS was $(1.03), both widening vs Q2 2023 due to higher operating spend .
Clinical readouts at EHA showed efficacy signals: bitopertin reduced toxic PPIX and improved pain-related outcomes; DISC-0974 demonstrated durable hemoglobin responses; DISC-3405 SAD data confirmed mechanism with hepcidin induction and iron reductions .
Stock reaction catalysts ahead: H2 2024 regulatory interactions for bitopertin (EPP) and DISC-0974 (MF), plus multiple-ascending dose (MAD) data for DISC-3405 and additional DISC-0974 NDD-CKD updates .

What Went Well and What Went Wrong

What Went Well

Robust liquidity and extended runway: Completed a $178.0M gross underwritten offering in June; ended Q2 with $500.9M in cash and equivalents, funding operations well into 2027 .
Portfolio momentum across programs: Management highlighted “highly encouraging” EHA data supporting therapeutic potential across bitopertin (EPP), DISC-0974 (MF), and proof-of-mechanism for DISC-3405; “We continue to make great progress…” .
Bitopertin efficacy signals: Statistically significant, dose-dependent PPIX reduction (−21.6% at 20 mg, −40.7% at 60 mg vs placebo); significant reduction in rate of phototoxic reactions with pain at 60 mg; significant PGIC “much better” at 60 mg .

What Went Wrong

AURORA key secondary missed: Cumulative time in sunlight on pain-free days did not reach statistical significance vs placebo due to an outsized placebo response, raising endpoint selection considerations for registration .
Operating expenses elevated: R&D rose to $23.5M (from $12.1M) and G&A to $7.4M (from $5.2M) year over year, reflecting increased headcount, clinical progression, and manufacturing; net loss widened to $(26.4)M .
Estimates unavailable at time of request: Wall Street consensus via S&P Global could not be retrieved due to a temporary request limit; comparisons vs street numbers are deferred (consensus unavailable) (Values would be retrieved from S&P Global).

Financial Results

Income Statement – Year over Year (Q2 2023 vs Q2 2024)

Metric ($USD Millions unless noted)	Q2 2023	Q2 2024
Research & Development Expense	$12.1	$23.5
General & Administrative Expense	$5.2	$7.4
Total Operating Expenses	$17.3	$30.9
Other Income (Expense), net	$1.4	$4.6
Income Tax Expense	$(0.02)	$(0.06)
Net Loss	$(15.9)	$(26.4)
Weighted Avg Shares (Basic & Diluted)	21,484,955	25,649,043
Net Loss per Share (Basic & Diluted)	$(0.74)	$(1.03)

Income Statement – Sequential (Q1 2024 vs Q2 2024)

Metric ($USD Millions unless noted)	Q1 2024	Q2 2024
Research & Development Expense	$23.7	$23.5
General & Administrative Expense	$7.8	$7.4
Total Operating Expenses	$31.5	$30.9
Other Income (Expense), net	$4.5	$4.6
Income Tax Expense	$(0.01)	$(0.06)
Net Loss	$(26.9)	$(26.4)
Weighted Avg Shares (Basic & Diluted)	24,809,869	25,649,043
Net Loss per Share (Basic & Diluted)	$(1.09)	$(1.03)

Balance Sheet – Cash and Equity Trend

Metric ($USD Millions)	Dec 31, 2023	Mar 31, 2024	Jun 30, 2024
Cash, Cash Equivalents & Marketable Securities	$360.4	$342.6	$500.9
Total Stockholders’ Equity	$345.1	$338.1	$488.8
Total Liabilities	$22.9	$16.1	$21.4

Segment breakdown: Not applicable; the company reports no revenue segments in these releases .

Clinical KPIs – AURORA (Bitopertin EPP)

KPI	20 mg	60 mg	Placebo
PPIX Change vs Baseline	−21.6% (p=0.003 vs placebo)	−40.7% (p<0.001 vs placebo)	+8.0%
Phototoxic Reactions with Pain – Incidence Rate vs Placebo	−60% (p=0.109)	−75% (p=0.011)	—
Patients Reporting Any Phototoxic Event	19%	12%	46%
PGIC “Much Better” (Completers)	77%	86% (p=0.022 vs placebo)	50%
Cumulative Time in Sunlight (hrs) on Pain-Free Days	175.1	153.1	133.9
Hemoglobin	Stable (generally well tolerated)	Stable (generally well tolerated)	—

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Operating Runway	Multi-year	“Well into 2026” (as of Q1 2024)	“Well into 2027” (as of Q2 2024)	Raised
Bitopertin (EPP) – Regulatory Interactions	H2 2024	Regulatory interactions anticipated H2 2024	Update on regulatory interactions expected H2 2024	Maintained timing
DISC-0974 (MF) – Regulatory Interactions/Phase 2 Design	H2 2024	Updated MF data in 1H 2024; CKD data H2 2024	Plan for regulator interactions in H2 2024 to determine optimal Phase 2 design (MF); CKD update H2 2024	Clarified next steps
DISC-3405 (TMPRSS6) – MAD Data	H2 2024	Initial SAD data in 1H 2024	MAD cohorts data expected H2 2024	New upcoming milestone

No revenue, margin, OpEx, OI&E, tax-rate or dividend guidance ranges were provided in the quarter’s materials .

Earnings Call Themes & Trends

Note: A Q2 2024 earnings call transcript was not available in our document set; themes are drawn from the Q2 press release and prior period releases.

Topic	Previous Mentions (Q4 2023, Q1 2024)	Current Period (Q2 2024)	Trend
Regulatory/Legal (EPP & MF)	AURORA topline expected Mar/Apr; fast track/orphan designations; regulatory interactions planned H2 2024	Updates on regulatory interactions for bitopertin (EPP) and DISC-0974 (MF) expected H2 2024	Steady cadence toward registration planning
R&D Execution	Positive BEACON (EPP) and MF activity; DISC-3405 initiated; AURORA topline met primary endpoint	EHA data positive across all three programs; DISC-3405 SAD confirms mechanism	Execution broadening across pipeline
Clinical Endpoints/Design	BEACON sunlight tolerance strong; AURORA sunlight time endpoint at risk due to placebo	AURORA key secondary missed due to placebo; plan to optimize registrational endpoints	Endpoint refinement required
Capital Markets/Liquidity	Ended 2023 with ~$360M cash; runway into 2026	$178M offering; $500.9M cash; runway into 2027	Stronger balance sheet
Commercialization Planning	Leadership additions (CFO/CCO/COO) anticipating late-stage dev	Ongoing progress; no new commercialization timelines disclosed	Building capabilities, timeline TBD

Management Commentary

“The data we presented at EHA this past quarter were highly encouraging and supportive of the therapeutic potential of our entire portfolio… We continue to make great progress and look forward to sharing updates on our regulatory interactions around bitopertin and DISC-0974 in the second half of the year.” — John Quisel, President & CEO .
“This study has confirmed that bitopertin significantly reduces the toxic metabolite, PPIX… Given [the placebo response], we will need to conduct an analysis of our final data set and work… to define the optimal registrational endpoints moving forward.” — John Quisel (AURORA topline) .
Q1 context: “Topline data from AURORA… strengthened our belief that bitopertin is active… we look forward to building upon this and sharing additional data… as we move towards regulatory interactions in the second half of the year.” — John Quisel .

Q&A Highlights

Transcript unavailable in our document repository for Q2 2024; no Q&A details could be extracted from the quarter’s filings or furnished materials .

Estimates Context

Consensus EPS and revenue estimates (S&P Global) for Q2 2024 were unavailable due to a temporary request limit at the time of retrieval; results vs street and estimate revisions cannot be assessed in this report. Values would be retrieved from S&P Global.
Given the company’s clinical-stage status and no reported revenue lines in the quarter’s furnished statements, street focus likely centers on cash runway, operating expense trajectory, and clinical/regulatory milestones rather than top-line/earnings comparisons .

Key Takeaways for Investors

Liquidity and runway de-risk near-term financing: $500.9M cash and securities post-offering fund operations well into 2027, reducing financing overhang through multiple data catalysts .
Bitopertin remains promising despite a mixed secondary: Primary endpoint success on PPIX and pain-related outcomes supports disease-modifying potential, but registrational endpoint work is needed given the placebo effect on sunlight-time metrics .
MF and CKD anemia programs advancing: DISC-0974 reported durable hemoglobin responses and reduced transfusion burden in MF; regulator interactions to shape Phase 2 design in H2 2024; CKD anemia update also expected H2 2024 .
Platform validation expanding: DISC-3405 SAD demonstrated hepcidin induction and sustained iron reduction supportive of monthly SC dosing, with MAD data to follow in H2 2024 — a potential incremental valuation driver .
OpEx will stay elevated with pipeline progress: R&D and G&A increases reflect clinical advancement and headcount; sequential spend stabilized in Q2 vs Q1, but absolute levels are higher YoY .
Near-term stock catalysts: H2 2024 regulatory updates for EPP and MF and additional clinical data (DISC-3405 MAD, DISC-0974 CKD) could shift perceptions on registrational path and commercial potential .
Watch for endpoint alignment and patient-reported outcomes: Management’s focus on optimal registrational endpoints, PGIC, and pain-related events could define regulatory strategy and eventual label scope in EPP .

Notes:

No non-GAAP adjustments were furnished; results presented reflect GAAP measures in provided statements .
We searched for Q2 2024 earnings call transcripts and additional Q2 press releases but found none in the document catalog; analysis relies on 8-K press releases and furnished exhibits .

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