Executive Summary

Lumos reported FY 2023 results and clinical progress; the Phase 2 OraGrowtH210/212 trials “met all primary and secondary endpoints,” validating the PEM strategy and supporting Phase 3 planning in moderate PGHD, with an End-of-Phase 2 FDA meeting in Q2 2024 and Phase 3 initiation targeted for Q4 2024 .
Year-end cash, cash equivalents, and short-term investments were $36.1M (runway through Q3 2024), down from $67.4M at FY22; net loss widened to $34.0M vs. $31.1M in 2022 .
Q4 2023 quarterly figures implied: royalty revenue ~$0.83M, net loss ~$$9.46M, and operating expenses ~$10.83M, reflecting increased R&D and Phase 2 activities; prior quarter net loss was $8.3M .
Key catalysts: EOP2 FDA meeting (Q2 2024), full 12-month OraGrowtH210 data presentation (Q2 2024), and Phase 3 start (Q4 2024), with strong KOL support cited by management .

What Went Well and What Went Wrong

What Went Well

“The past year was a highly successful one… topline results from Phase 2 OraGrowtH210 and OraGrowtH212… met all of their primary endpoints and provided substantial support for the advancement of LUM-201 toward a registrational Phase 3 trial” (Rick Hawkins) .
Non-inferiority deltas within FDA-suggested margins: AHV delta between 1.6 mg/kg LUM-201 and rhGH comparator was within <2 cm/yr at 6 and 12 months; AHV of 8.2 cm/yr (6m) and 8.0 cm/yr (12m) at the 1.6 mg/kg dose .
PEM strategy validated with 100% reproducibility of PEM-positive classification; LUM-201 normalized IGF-1, achieved comparable growth with ~20% of GH concentration vs rhGH, and showed a clean safety profile (no treatment-related SAEs) .

What Went Wrong

Cash drawdown and widening net loss: cash fell to $36.1M at 12/31/23 (from $67.4M at 12/31/22); FY23 net loss was $34.0M vs. $31.1M FY22, reflecting increased clinical and manufacturing spend .
Management disclosed “substantial doubt” about ability to continue as a going concern without additional financing; Phase 3 execution requires raising capital near-term .
In OraGrowtH210, rhGH comparator AHV was higher than anticipated, which management attributed to dose/outliers; Phase 3 aims for a larger, more statistically robust comparator alignment .

Financial Results

Quarterly P&L (USD thousands)

Metric	Q2 2023	Q3 2023	Q4 2023 (derived)
Revenues (Royalty)	$527	$7	$826
Total Operating Expenses	$10,170	$8,939	$10,830
Net Income (Loss)	$(8,931)	$(8,300)	$(9,457)

Notes: Q4 2023 figures derived from FY 2023 less nine months ended 9/30/2023 based on disclosed financial statements .

EPS

Metric	Q2 2023	Q3 2023	Q4 2023
Diluted EPS	$(1.09)	$(1.04)	Not disclosed in filings

Operating Expense Mix (USD thousands)

Metric	Q2 2023	Q3 2023	Q4 2023 (derived)
R&D Expense	$6,024	$5,046	$6,657
G&A Expense	$4,146	$3,893	$4,173

Balance Sheet / KPIs

Metric	Q2 2023	Q3 2023	Q4 2023 (YE)
Cash, Cash Equivalents & Short-term Investments	$50,900	$42,700	$36,100
Shares Outstanding (End of Period)	8,041,345	7,914,582	8,102,555
AHV (LUM-201 1.6 mg/kg)	6m: 8.2 cm/yr	—	12m: 8.0 cm/yr

Segment breakdown: not applicable (single clinical program focus) .

Guidance Changes

Metric	Period	Previous Guidance	Current Guidance	Change
Cash Runway	Through 2024	“At least 12 months” from Q2 filing (into mid-2024)	“Through Q3 2024”	Maintained/clarified runway timing
Cash Use	Q4 2023	Avg $9.5–$10.5M per quarter in 2023	$9.0–$10.0M in Q4 2023	Slightly lower than prior avg range
Regulatory Milestones	2024	Primary outcome data presentations, EOP2 planning	EOP2 FDA meeting in Q2 2024; Phase 3 initiation in Q4 2024	Firmed timing, added Phase 3 start target

Earnings Call Themes & Trends

Topic	Previous Mentions (Q2 2023)	Previous Mentions (Q3 2023)	Current Period (Q4 2023)	Trend
R&D execution (PGHD)	Confirmed Q4 timeline for Phase 2 readouts; interim data encouraging	Phase 2 topline met endpoints; 1.6 mg/kg dose optimal; pulsatile MOA affirmed	Full-year recap reiterates Phase 2 success; preparing Phase 3	Positive momentum; de-risked via PEM
Regulatory pathway	Phase 3 design concepts and PEM-driven enrichment	Phase 3 planning following topline data; noted rhGH control anomalies	EOP2 meeting in Q2 2024; Phase 3 initiation targeted Q4 2024	Firming timelines
Safety	Clean safety in Phase 2; no significant signals	Clean safety; no treatment-related SAEs; increased appetite common	“No safety signal” reiterated	Stable favorable profile
Cash runway/financing	Runway into Q3 2024; average cash use guidance	Runway through Q3 2024; Q4 cash use guide	Runway through Q3 2024; going concern risk pending financing	Financing need elevating
KOL/support	ENDO/ESPE data and KOL webinar	Additional KOL virtual event planned Dec 6	“Broad support among pediatric endocrinology community”	Strengthening advocacy

Management Commentary

“We expect to present full twelve-month and longer-term data… in Q2… End-of-Phase 2 Meeting… in Q2 2024… initiate this registrational trial in the fourth quarter” (Rick Hawkins, CEO) .
“Delta at 6 and 12-month AHV between optimal LUM-201 dose… was within the non-inferiority margin (< 2 cm/yr) suggested by FDA for recent approvals” .
“With only 20% of the GH concentration of injectable rhGH, LUM-201 achieved similar AHV, illustrating the greater efficiency of LUM-201’s unique pulsatile mechanism of action” .
“Cash on hand… expected to support operations through the third quarter of 2024” .

Q&A Highlights

The company held a results call on March 7, 2024 (4:30 p.m. ET); a transcript is not available in the sourced documents, so Q&A specifics and any guidance clarifications cannot be summarized here .
A separate data call was also held on November 8, 2023 regarding Phase 2 topline; transcript details are unavailable in the sourced documents .

Estimates Context

Wall Street consensus estimates via S&P Global were unavailable due to missing CIQ mapping for LUMO; as a result, a beat/miss assessment versus estimates cannot be provided at this time. Values would have been retrieved from S&P Global if mapping were available.

Key Takeaways for Investors

Phase 2 success and PEM validation de-risk Phase 3 design; AHV deltas were within historical non-inferiority margins, and IGF-1 normalization plus safety profile strengthen the thesis for moderate PGHD .
Near-term catalysts (Q2 EOP2 meeting; Q4 Phase 3 start) are central to stock narrative; outcomes and Phase 3 funding clarity likely to drive valuation .
Cash runway through Q3 2024 underscores urgency to secure financing; the 10-K explicitly cites going concern risk without additional capital, which is a key overhang for the Phase 3 program .
Operating expenses ramped in Q4 (R&D ~$6.7M; G&A ~$4.2M) as the company prepares for Phase 3; expect elevated spend tied to regulatory and manufacturing readiness .
Quarterly royalty revenue remains modest (Q4 ~$0.83M); fundamentals are driven primarily by clinical/regulatory progress rather than near-term revenue .
The higher-than-expected rhGH comparator AHV in OraGrowtH210 is a design consideration; Phase 3 aims to mitigate comparator anomalies via larger, more robust sampling .
KOL and community support for an oral therapy in PGHD is a differentiator, with LUM-201’s pulsatile mechanism potentially improving durability and adherence relative to injections .

Appendix: Additional Data and Trend Analysis

FY Results vs Prior Year (USD thousands, unless noted)

Metric	FY 2022	FY 2023
Revenues (Royalty)	$1,523	$2,051
Total Operating Expenses	$33,563	$38,665
Net Income (Loss)	$(31,062)	$(34,034)
Diluted EPS	$(3.71)	$(4.18)
Cash & Short-term Investments (Year-end)	$67,359	$36,077

Drivers: R&D +$4.2M YoY (clinical trial +$3.3M; manufacturing +$0.9M), G&A +$0.9M (personnel +$0.5M; royalties +$0.4M), partially offset by lower personnel and other expenses .

LUMOS PHARMA, INC. (LUMO)·Q4 2023 Earnings Summary