Lumos Pharma Announces Positive End-of-Phase 2 Meeting with FDA and Reports First Quarter 2024 Financial Results
Outcome from End-of-Phase 2 Meeting Supportive of a Placebo-Controlled Phase 3 Trial
Updated 12 and 24-Month Data from Phase 2 OraGrowtH210 and OraGrowtH212 Trials Continue to Show Oral LUM-201 Achieves Significant Increase in Growth from Baseline, Durable Effect to 24 Months
Company to Host Conference Call
"We are pleased to announce that, earlier this quarter, we had a very productive End-of-Phase 2 meeting with the FDA," said
"In addition to our encouraging engagement with the FDA, we are also very pleased to share updated data from our Phase 2 OraGrowtH trials. These data continue to show that LUM-201 produces a significant increase in growth from baseline in annualized height velocity (AHV) at 6 and 12 months in per protocol analysis. Combined data also suggest durable benefit out to 24 months.
"We believe these developments have positioned us to advance LUM-201 toward both a Phase 3 registrational trial and potential approval of LUM-201 as the first oral therapeutic for moderate pediatric growth hormone deficiency,"
Recent Highlights
- End of Phase 2 Meeting Held with FDA
- FDA indicated that a placebo-controlled trial design is an appropriate option for a Phase 3 trial for LUM-201. We believe this reflects FDA’s recognition of unique qualities of LUM-201’s mechanism of action as a growth hormone secretagogue.
- Proposal for a Phase 3 trial to include a 12-month double-blinded, placebo-controlled design with 2:1 randomization, ~150 patients with the placebo-controlled portion of the study lasting six months, which we believe will improve the likelihood of success when compared to a non-inferiority study.
- Planning is ongoing, and the Company expects to initiate a Phase 3 trial of LUM-201 in Q4 2024, subject to FDA approval.
- Updated LUM-201 Data from Combined OraGrowtH210 and OraGrowtH212 Trials
- Additional data continue to show durable LUM-201 treatment effect to 12 and 24 months.
- Full 12-month data from OraGrowtH210 demonstrated LUM-201 produces significant increase in growth from baseline with AHVs of 8.2 cm/yr (N=22) and 7.6 cm/yr (N=21) at 6 and 12 months, respectively, at the 1.6 mg/kg dose vs. 4.7 cm/yr baseline growth (N=13).*
- Full 12-month data from OraGrowtH210 continued to show durable effect to 12 months for all LUM-201 cohorts and 1.6 mg/kg/day as optimal dose to advance to Phase 3.
- Updated combined data from OraGrowtH210 and OraGrowtH212 trials continued to demonstrate LUM-201 AHV durable to 24 months with per protocol-24M (N=12) AHV of 8.1 cm/yr and 7.3 cm/yr at 12 and 24 months, respectively.
- More moderate year-2 decline in AHV of 9.9% for LUM-201 compared to year-2 decline in AHV of 19.7% observed in historical rhGH benchmarks likely due to LUM-201 restoration of GH and IGF-1 to normal levels via amplification of physiologic pulsatile secretion of growth hormone within the natural endocrine feedback loop.
- Investigational safety profile continues to be favorable.
- Data from Phase 2 OraGrowtH210 and OraGrowtH212 Trials Presented at Medical Meetings in US and
Europe Pediatric Endocrinology Society (PES)- 10th
International Congress of theGrowth Hormone Research Society (GRS) European Congress of Endocrinology (ECE)- Data presented at these medical conferences demonstrate that, by augmenting the natural pulsatile secretion of growth hormone, LUM-201 produces comparable growth to injectable rhGH with significantly less exposure to circulating growth hormone.
- Additional Data from Phase 2 OraGrowtH Trials to be Presented in Q2 2024
- Full 12-Month OraGrowtH212 data, additional analyses of OraGrowtH210 data, and updated combined 24-month data to be presented in Q2 2024
- Two abstracts accepted for poster presentation at the
Endocrine Society (ENDO) Annual Meeting
*Baseline AHV data were not required for enrollment; baseline data available for N=13 subjects.
Financial Results for Quarter Ended
Cash Position –
R&D Expenses – Research and development expenses for the quarter ended
G&A Expenses – General and administrative expenses for the quarter ended
Net Loss – The net loss for the quarter ended
Conference Call and Webcast Details
Date:
Time:
Dial-in: 1-877-407-9716 or 1-201-493-6779 (International)
Conference ID: 13746447
Dial-in registration (Available 15 minutes prior to scheduled start time): Click Here
Webcast: Click Here
About
Cautionary Note Regarding Forward-Looking Statements
This press release contains forward-looking statements of
We are passionate about our business - including LUM-201 and the potential it may have to help patients in the clinic. This passion feeds our optimism that our efforts will be successful and bring about meaningful change for patients. Please keep in mind that actual results or events could differ materially from the plans, intentions and expectations disclosed in the forward-looking statements that we make.
We have attempted to identify forward-looking statements by using words such as "projected," "upcoming," "will," "would," "plan," "intend," "anticipate," "approximate," "expect," "potential," "imminent," and similar references to future periods or the negative of these terms. Not all forward-looking statements contain these identifying words. Examples of forward-looking statements include, among others, statements we make regarding that our FDA meeting was supportive of a placebo-controlled Phase 3 trial, that we expect to finalize design details with the FDA in the third quarter and to be in position to initiate this trial before the end of this year, that the data continue to show that LUM-201 produces a significant increase in growth from baseline in annualized height velocity (AHV) at 6 and 12 months in per protocol analysis, that combined data also suggest durable benefit out to 24 months, that we plan to move forward with a proposal for a Phase 3 study that will be a double-blinded, placebo-controlled clinical trial with a 2:1 randomization in approximately 150 patients, that we believe this reflects the FDA’s recognition of unique qualities of LUM-201’s mechanism of action as a growth hormone secretagogue, that we believe these developments have positioned us to advance LUM-201 towards a Phase 3 registrational trial and toward potential approval of LUM-201 as the first oral therapeutic for moderate pediatric growth hormone deficiency, that we believe the study design will improve the likelihood of success when compared to a non-inferiority study, that the investigational safety profile continues to be favorable, that cash on hand is expected to support operations through Q3 2024, which is inclusive of Phase 3 planning and preparatory activities, and any other statements other than statements of historical fact.
We wish we were able to predict the future with 100% accuracy, but that just is not possible. Our forward-looking statements are neither historical facts nor assurances of future performance. You should not rely on any of these forward-looking statements and, to help you make your own risk determinations, we have provided an extensive discussion of risks that could cause actual results to differ materially from our forward-looking statements including risks related to the continued analysis of data from our LUM-201 Trials, the timing and outcome of our future interactions with regulatory authorities including our Type C meeting with the FDA, the timing and ability of Lumos to raise additional equity capital as needed to fund our Phase 3 Trial, our ability to project future cash utilization and reserves needed for contingent future liabilities and business operations, the ability to structure our Phase 3 trial in an effective and timely manner, the ability to successfully develop our product candidate, the effects of pandemics, other widespread health problems or military conflicts including the
We anticipate that subsequent events and developments will cause our views to change. We may choose to update these forward-looking statements at some point in the future, however, we disclaim any obligation to do so. As a result, you should not rely on these forward-looking statements as representing our views as of any date subsequent to the date of this press release.
Investor & Media Contact:
Lumos Pharma Investor Relations
512-792-5454
ir@lumos-pharma.com
| Condensed Consolidated Statements of Operations and Comprehensive Loss | ||||||||
| (unaudited) | ||||||||
| (In thousands, except share and per share amounts) |
||||||||
| Three Months Ended |
||||||||
| 2024 | 2023 | |||||||
| Revenues: | ||||||||
| Royalty revenue | $ | 165 | $ | 691 | ||||
| Total revenues | 165 | 691 | ||||||
| Operating expenses: | ||||||||
| Research and development | 7,248 | 4,369 | ||||||
| General and administrative | 3,779 | 4,357 | ||||||
| Total operating expenses | 11,027 | 8,726 | ||||||
| Loss from operations | (10,862 | ) | (8,035 | ) | ||||
| Other income and expense: | ||||||||
| Other income, net | 263 | 119 | ||||||
| Interest income | 158 | 570 | ||||||
| Other income, net | 421 | 689 | ||||||
| Net loss | $ | (10,441 | ) | $ | (7,346 | ) | ||
| Net loss per share: | ||||||||
| Basic and diluted | $ | (1.29 | ) | $ | (0.89 | ) | ||
| Weighted average number of common shares outstanding: | ||||||||
| Basic and diluted | 8,104,905 | 8,239,941 | ||||||
| Other comprehensive income: | ||||||||
| Unrealized gain on short-term investments | — | 4 | ||||||
| Total comprehensive loss | $ | (10,441 | ) | $ | (7,342 | ) | ||
| Condensed Consolidated Balance Sheets | |||||||
| (In thousands, except share and per share amounts) |
|||||||
| 2024 | 2023 | ||||||
| (unaudited) | |||||||
| Assets | |||||||
| Current assets: | |||||||
| Cash and cash equivalents | $ | 23,179 | $ | 35,078 | |||
| Short-term investments | — | 999 | |||||
| Prepaid expenses and other current assets | 4,184 | 3,748 | |||||
| Income tax receivable | 181 | 210 | |||||
| Total current assets | 27,544 | 40,035 | |||||
| Non-current assets: | |||||||
| Right-of-use asset | 534 | 603 | |||||
| Total assets | $ | 28,078 | $ | 40,638 | |||
| Liabilities and Stockholders' Equity | |||||||
| Current liabilities: | |||||||
| Accounts payable | $ | 569 | $ | 890 | |||
| Accrued expenses | 3,552 | 5,858 | |||||
| Current portion of lease liability | 293 | 282 | |||||
| Total current liabilities | 4,414 | 7,030 | |||||
| Long-term liabilities: | |||||||
| Royalty obligation payable to |
6,000 | 6,000 | |||||
| Lease liability | 225 | 303 | |||||
| Total liabilities | 10,639 | 13,333 | |||||
| Commitments and contingencies: | |||||||
| Stockholders' equity: | |||||||
| Undesignated preferred stock, |
— | — | |||||
| Common stock, |
81 | 81 | |||||
| (201 | ) | (196 | ) | ||||
| Additional paid-in capital | 189,517 | 188,937 | |||||
| Accumulated deficit | (171,958 | ) | (161,517 | ) | |||
| Total stockholders' equity | 17,439 | 27,305 | |||||
| Total liabilities and stockholders' equity | $ | 28,078 | $ | 40,638 | |||
Source: Lumos Pharma, Inc.