Lumos Pharma Reports Third Quarter 2023 Financial Results and Clinical Development Updates
-- Phase 2 Data for Potentially the First Oral Therapeutic for PGHD Met All Primary and Secondary Endpoints with Supportive Evidence to Advance to Phase 3 –
-- OraGrowtH210 Trial Results Show LUM-201 Dose of 1.6 mg/kg Achieves AHVs of 8.2 cm/yr at 6 Months and 8.0 cm/yr at 12 Months, Consistent with Historical Growth Rates for Moderate PGHD Population –
-- OraGrowtH212 Data Confirm Unique Pulsatile Mechanism of Action of LUM-201and Demonstrate Closer to Normal Physiological Growth Hormone Levels and Normalized IGF-1 Levels –
-- Cash of
“With the end of Phase 2 readout from our OraGrowtH210 and OraGrowtH212 trials announced today, we are thrilled that these data support advancing our clinical program towards a pivotal Phase 3 trial for potentially the first oral therapeutic for moderate PGHD,” said
- Phase 2 OraGrowtH210 and PK/PD OraGrowtH212 Trials met all primary and secondary endpoints. Data from the topline Phase 2 OraGrowtH210 Trial demonstrated that the 1.6 mg/kg/day LUM-201 dose produced a mean annualized height velocity (AHV) of 8.2 cm/yr at six months on treatment for moderate PGHD subjects, in line with historical data in moderate PGHD patients1,2,3,4. Additionally, at twelve months on treatment, a durable effect was also observed with LUM-201 achieving AHV of 8.0 cm/yr at the 1.6 mg/kg dose, within the targeted 2 cm/yr margin of the comparator injectable rhGH arm. Data also provided preliminary validation of the PEM strategy, with prespecified primary and secondary outcomes met, de-risking our patient selection for our Phase 3 program. Data from the OraGrowtH212 Trial confirmed that LUM-201’s unique pulsatile mechanism produces an increase in the growth rates by restoring growth hormone secretion and IGF-1 to within normal ranges. The safety profile for LUM-201 remained clean throughout both Phase 2 trials, with no safety concerns identified in either of our Phase 2 trials conducted thus far. For a link to the Company’s conference call and presentation of the data refer to the Events & Presentations page in the Investors & Media section of the Company’s website.
- OraGrowtH212 Trial Data Presented at the 2023 annual meeting of the
European Society for Paediatric Endocrinology. An oral presentation of the deconvolution analysis of growth hormone (GH) concentration sampled over 12 hours at baseline and after 6 months of therapy with daily oral LUM-201 illustrated how treatment with LUM-201 increases AHV, total GH secretion, and serum IGF-1 and IGFBP3 for individuals with moderate PGHD. Measured at 6 months compared to baseline, data showed a 60% increase in GH secretion to a level comparable to established values in normal healthy children and a 62% increase in AHV. Independent Panelof Renowned Pediatric Endocrinologists Discussed PGHD and Therapeutic Landscape. On September 5, 2023, a panel of five pediatric endocrinologists participated in a webinar where they discussed the benefits that the oral therapeutic LUM-201 candidate may provide compared to current injectable therapeutic options.
- Virtual KOL Event Planned. The Company plans to host a virtual KOL Event on
December 6th to discuss topline results from OraGrowtH210 and OraGrowtH212 trials in greater detail and provide updates on clinical and corporate strategy. Management will be joined by the following three esteemed thought leaders in the field of endocrinology: Andrew Dauber, MD, Chief of Endocrinology at Children's National Medical Center, Washington, D.C. Fernando Cassorla, MD, Chief of Pediatric Endocrinology at the Institute of Maternal and Child Research, University of Chile Leslie A. Soyka, MD, Chief of Pediatric Endocrinology, UMass Memorial Medical Center; Associate Professor, UMass Chan Medical School, Worcester, MA
Access information regarding the KOL Event will be provided at a later date.
Financial Results for the Quarter Ended
- Cash Position –
Lumos Pharmaended the quarter on September 30, 2023with cash, cash equivalents and short-term investments totaling $42.7 millioncompared to $67.4 millionon December 31, 2022. The Company expects cash use of approximately $9.0to $10.0 millionin the fourth quarter of 2023. Cash on hand as of September 30, 2023is expected to support operations through the third quarter of 2024, inclusive of the activities related to planning and initiation of a pivotal Phase 3 clinical trial.
- R&D Expenses – Research and development expenses were
$5.0 millionfor the quarter ended September 30, 2023, compared to $4.1 millionfor the same period in 2022, primarily due to an increase of $0.9 millionin clinical trial expenses and $0.2 millionin consulting expenses, offset by a decrease of $0.2 millionin personnel-related expenses.
- G&A Expenses – General and administrative expenses were
$3.9 millionfor the quarter ended September 30, 2023, compared to $3.9 millionfor the same period in 2022, primarily due to an increase of $0.3 millionin personnel-related expenses offset by a $0.3 milliondecrease in royalty expense.
- Net Loss – The net loss for the quarter ended
September 30, 2023was $8.3 millioncompared to a net loss of $7.3 millionfor the same period in 2022. Lumos Pharmaended the third quarter 2023 with 7,914,582 shares outstanding.
About Lumos Pharma’s Clinical Trials
Phase 2 OraGrowtH210 Trial Design
The OraGrowtH210 Trial is a multi-site, global trial evaluating orally administered LUM-201 at three dose levels (0.8, 1.6, 3.2 mg/kg/day) compared to daily injectable recombinant human growth hormone (rhGH) 34 µg/kg/day in 82 subjects diagnosed with moderate PGHD. The trial population was enriched for subjects responsive to LUM-201 during screening by applying the specific PEM cutoffs of a baseline IGF-1 value > 30 ng/ml and a peak growth hormone value of ≥ 5 ng/ml after administering a single dose of 0.8 mg/kg of LUM-201 to treatment-naïve PGHD patients. The study was not designed to evaluate efficacy and demonstrate non-inferiority to daily GH.
OraGrowtH212 Trial Design
The OraGrowtH212 Trial is a single site, open-label trial evaluating the pharmacokinetic (PK) and pharmacodynamic (PD) effects of oral LUM-201 in up to 24 treatment-naïve PGHD subjects at two dose levels, 1.6 and 3.2 mg/kg/day. Every subject in the OraGrowtH212 Trial was PEM-positive and, therefore, enriched for responsiveness to LUM-201.
Switch Study, OraGrowtH213 Trial, Evaluating LUM-201 in OraGrowtH210 Subjects Previously on rhGH
The OraGrowtH213 Trial is an open-label, multi-center, Phase 2 trial evaluating the growth effects and safety of LUM-201 following 12 months of daily rhGH in up to 20 idiopathic PGHD subjects who have completed the OraGrowtH210 Trial. Subjects will be administered LUM-201 at a dose level of 3.2 mg/kg/day for up to 12 months.
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 our Phase 2 data providing supportive evidence to advance oral LUM-201 to Phase 3, the potential for LUM-201 to be the first oral therapeutic for PGHD, data from the OraGrowtH210 Trial supporting the 1.6 mg/kg dose for LUM-201 as the optimal dose for a Phase 3 trial, de-risking our patient selection for our Phase 3 program, expecting cash use of approximately
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 end of Phase 2 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
|Condensed Consolidated Statements of Operations and Comprehensive Loss|
|(In thousands, except share and per share amounts)
|Three Months Ended
||Nine Months Ended
|Research and development||5,046||4,129||15,439||12,995|
|General and administrative||3,893||3,918||12,396||11,221|
|Total operating expenses||8,939||8,047||27,835||24,216|
|Loss from operations||(8,932||)||(7,550||)||(26,610||)||(23,205||)|
|Other income and expense:|
|Other income, net||186||7||429||19|
|Other income, net||632||299||2,004||390|
|Net loss before taxes||(8,300||)||(7,251||)||(24,606||)||(22,815||)|
|Income tax benefit||—||—||29||—|
|Net loss per share:|
|Basic and diluted||$||(1.04||)||$||(0.86||)||$||(3.01||)||$||(2.73||)|
|Weighted average number of common shares outstanding:|
|Basic and diluted||7,978,457||8,388,029||8,161,904||8,371,449|
|Other comprehensive loss:|
|Unrealized gain on short-term investments||7||—||5||—|
|Total comprehensive loss||$||(8,293||)||$||(7,251||)||$||(24,572||)||$||(22,815||)|
|Condensed Consolidated Balance Sheets
|(In thousands, except share and per share amounts)
|Cash and cash equivalents||$||35,557||$||56,007|
|Prepaid expenses and other current assets||4,581||4,427|
|Total current assets||47,447||72,009|
|Property and equipment, net||36||53|
|Liabilities and Stockholders' Equity|
|Current portion of lease liability||126||233|
|Total current liabilities||6,116||6,708|
|Royalty obligation payable to
|Commitments and contingencies|
|Undesignated preferred stock,
|Additional paid-in capital||187,673||187,164|
|Accumulated other comprehensive loss||(4||)||(9||)|
|Total stockholders' equity||35,492||59,584|
|Total liabilities and stockholders' equity||$||47,751||$||72,292|
Source: Lumos Pharma, Inc.