Lumos Pharma, Inc. (NASDAQ:LUMO), a clinical-stage
biopharmaceutical company focused on therapeutics for rare
diseases, today announced that topline results met primary and
secondary endpoints for its Phase 2 Dose-Finding OraGrowtH210 Trial
and Phase 2 Pharmacokinetic/Pharmacodynamic (PK/PD) OraGrowtH212
Trial evaluating oral LUM-201 for subjects with moderate pediatric
growth hormone deficiency (PGHD) who screened PEM-positive
utilizing Lumos Pharma’s predictive enrichment marker (PEM)
strategy. Lumos also announced its financial results for the
quarter ended September 30, 2023.
“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 Rick Hawkins, Chairman and CEO
of Lumos Pharma. “Data confirm the optimal dose is 1.6 mg/kg/day
LUM-201 to advance to Phase 3, and preliminary data show durability
out to 24 months of treatment. Additionally, the OraGrowtH212 data
reaffirms our confidence in our oral compound’s unique mechanism of
action, the importance of the natural, pulsatile release of growth
hormone, and its impact on restoring growth.”
Recent Highlights
- 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 Panel of
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.
Upcoming Events
- 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.
1 Blum et al JES 2021, 2 Lechuga-Sancho et al
JPEM 2009, 3 Ranke et al JCEM 2010, 4 For all OraGrowtH Trial AHV
values, ANCOVA Model Terms: treatment, Age at dose 1, Sex, Baseline
HT SDS, Baseline BMI SDS, Baseline IGF-1 SDS, LUM-201 PEM, Baseline
BA Delay
Financial Results for the Quarter Ended
September 30, 2023
- Cash Position – Lumos Pharma ended
the quarter on September 30, 2023 with cash, cash equivalents and
short-term investments totaling $42.7 million compared to $67.4
million on December 31, 2022. The Company expects cash use of
approximately $9.0 to $10.0 million in the fourth quarter of 2023.
Cash on hand as of September 30, 2023 is 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 million for the quarter ended
September 30, 2023, compared to $4.1 million for the same period in
2022, primarily due to an increase of $0.9 million in clinical
trial expenses and $0.2 million in consulting expenses, offset by a
decrease of $0.2 million in personnel-related expenses.
- G&A Expenses – General and
administrative expenses were $3.9 million for the quarter ended
September 30, 2023, compared to $3.9 million for the same period in
2022, primarily due to an increase of $0.3 million in
personnel-related expenses offset by a $0.3 million decrease in
royalty expense.
- Net Loss – The net loss for the
quarter ended September 30, 2023 was $8.3 million compared to a net
loss of $7.3 million for the same period in 2022.
- Lumos Pharma ended the third
quarter 2023 with 7,914,582 shares outstanding.
About Lumos Pharma’s Clinical
TrialsPhase 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.
About Lumos PharmaLumos Pharma,
Inc. is a clinical stage biopharmaceutical company focused on the
development and commercialization of therapeutics for rare
diseases. The Company was founded and is led by a management team
with longstanding experience in rare disease drug development.
Lumos Pharma’s lead therapeutic candidate, LUM-201, is a novel,
oral growth hormone (GH) secretagogue, seeking to transform the
~$3.4B global GH market from injectable to oral therapy. LUM-201 is
currently being evaluated in multiple Phase 2 clinical studies in
Pediatric Growth Hormone Deficiency (PGHD) and has received Orphan
Drug Designation in both the US and EU. For more information,
please visit https://lumos-pharma.com/.
Cautionary Note Regarding
Forward-Looking Statements
This press release contains forward-looking
statements of Lumos Pharma, Inc. that involve substantial risks and
uncertainties. All such statements contained in this press release
are forward-looking statements within the meaning of The Private
Securities Litigation Reform Act of 1995. A law that, in part,
gives us the opportunity to share our outlook for the future
without fear of litigation if it turns out our predictions were not
correct.
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 $9.0 to $10.0 million in the
fourth quarter of 2023, that cash on hand as of September 30, 2023
is expected to support operations through the third quarter of
2024, future financial performance, results of operations, our cash
position and sufficiency of capital resources to fund our operating
requirements through future clinical trials, 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 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 Ukraine-Russia conflict and the Middle East
conflict and other risks that could cause actual results to differ
materially from those matters expressed in or implied by such
forward-looking statements including information in the "Risk
Factors" section and elsewhere in Lumos Pharma’s Quarterly Report
on Form 10-Q for the period ended June 30, 2023, as well as other
reports filed with the SEC including our subsequent Quarterly
Reports on Form 10-Q and Current Reports on Form 8-K. All of these
documents are available on our website. Before making any decisions
concerning our stock, you should read and understand those
documents.
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:
Lisa MillerLumos Pharma Investor
Relations512-792-5454ir@lumos-pharma.com
|
Lumos Pharma, Inc. |
Condensed Consolidated Statements of Operations and
Comprehensive Loss |
(unaudited) |
(In thousands, except share and per share
amounts) |
|
|
|
|
|
|
|
|
|
|
|
Three Months Ended September 30, |
|
Nine Months Ended September 30, |
|
|
|
2023 |
|
|
|
2022 |
|
|
|
2023 |
|
|
|
2022 |
|
Revenues: |
|
|
|
|
|
|
|
|
Royalty revenue |
|
$ |
7 |
|
|
$ |
497 |
|
|
$ |
1,225 |
|
|
$ |
1,011 |
|
Total revenues |
|
|
7 |
|
|
|
497 |
|
|
|
1,225 |
|
|
|
1,011 |
|
Operating expenses: |
|
|
|
|
|
|
|
|
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 |
|
Interest income |
|
|
446 |
|
|
|
292 |
|
|
|
1,575 |
|
|
|
371 |
|
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 |
|
$ |
(8,300 |
) |
|
$ |
(7,844 |
) |
|
$ |
(24,577 |
) |
|
$ |
(15,564 |
) |
|
|
|
|
|
|
|
|
|
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 |
) |
|
Lumos Pharma, Inc. |
Condensed Consolidated Balance Sheets |
(In thousands, except share and per share
amounts) |
|
|
September 30, |
|
December 31, |
|
|
|
2023 |
|
|
|
2022 |
|
|
|
(unaudited) |
|
|
Assets |
|
|
|
|
Current assets: |
|
|
|
|
Cash and cash equivalents |
|
$ |
35,557 |
|
|
$ |
56,007 |
|
Short-term investments |
|
|
7,137 |
|
|
|
11,352 |
|
Prepaid expenses and other current assets |
|
|
4,581 |
|
|
|
4,427 |
|
Other receivables |
|
|
172 |
|
|
|
223 |
|
Total current assets |
|
|
47,447 |
|
|
|
72,009 |
|
Non-current assets: |
|
|
|
|
Property and equipment, net |
|
|
36 |
|
|
|
53 |
|
Right-of-use asset |
|
|
268 |
|
|
|
230 |
|
Total assets |
|
$ |
47,751 |
|
|
$ |
72,292 |
|
Liabilities and
Stockholders' Equity |
|
|
|
|
Current liabilities: |
|
|
|
|
Accounts payable |
|
$ |
488 |
|
|
$ |
275 |
|
Accrued expenses |
|
|
5,502 |
|
|
|
6,200 |
|
Current portion of lease liability |
|
|
126 |
|
|
|
233 |
|
Total current liabilities |
|
|
6,116 |
|
|
|
6,708 |
|
Long-term liabilities: |
|
|
|
|
Royalty obligation payable to Iowa Economic Development
Authority |
|
|
6,000 |
|
|
|
6,000 |
|
Lease liability |
|
|
143 |
|
|
|
— |
|
Total liabilities |
|
|
12,259 |
|
|
|
12,708 |
|
Commitments and
contingencies |
|
|
|
|
Stockholders' equity: |
|
|
|
|
Undesignated preferred stock, $0.01 par value: Authorized shares -
5,000,000 at September 30, 2023 and December 31, 2022;
issued and outstanding shares - 0 at September 30, 2023 and
December 31, 2022 |
|
|
— |
|
|
|
— |
|
Common stock, $0.01 par value: Authorized shares - 75,000,000 at
September 30, 2023 and December 31, 2022; issued shares -
7,937,755 and 8,283,708 at September 30, 2023 and
December 31, 2022, respectively, and outstanding shares -
7,914,582 and 8,267,968 at September 30, 2023 and
December 31, 2022, respectively |
|
|
79 |
|
|
|
82 |
|
Treasury stock, at cost, 23,173 and 15,740 shares at
September 30, 2023 and December 31, 2022,
respectively |
|
|
(196 |
) |
|
|
(170 |
) |
Additional paid-in capital |
|
|
187,673 |
|
|
|
187,164 |
|
Accumulated deficit |
|
|
(152,060 |
) |
|
|
(127,483 |
) |
Accumulated other comprehensive loss |
|
|
(4 |
) |
|
|
(9 |
) |
Total stockholders'
equity |
|
|
35,492 |
|
|
|
59,584 |
|
Total liabilities and
stockholders' equity |
|
$ |
47,751 |
|
|
$ |
72,292 |
|
|
Lumos Pharma (NASDAQ:LUMO)
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