Lumos Pharma, Inc. (NASDAQ:LUMO), a biopharmaceutical company
advancing an oral therapeutic candidate for moderate Pediatric
Growth Hormone Deficiency (PGHD) through Phase 2 clinical trials,
announced today that an analysis of interim data from its
OraGrowtH212 Trial was given as an oral presentation at the 2023
annual meeting of the European Society for Paediatric Endocrinology
(ESPE), held in The Hague, Netherlands, September 21-23, 2023.
“These new data and analysis of interim data
from our OraGrowtH212 trial show that oral LUM-201 restored GH
secretion to similar levels seen in normally growing children by
enhancing endogenous pulsatile GH secretion while maintaining
normal feedback control of IGF-1,” said John C. McKew, PhD,
President and Chief Scientific Officer of Lumos. “The presented
results add to the growing body of evidence supporting the
physiologic mechanism of action for LUM-201, as well as its
potential as a treatment for moderate PGHD. As we look ahead to our
topline data readout from these trials in the fourth quarter, these
results and the others we have released recently add to our
confidence that at least one of the LUM-201 dose cohorts will meet
growth expectations based on historical averages, and that the
LUM-201 mechanism of action and potency can elicit sustained
improvements in growth in the moderate PGHD patient population. We
look forward to our topline data announcement and to continuing to
advance our LUM-201 clinical program as potentially the first oral
therapeutic for PGHD.”
The oral presentation, Deconvolution Analysis:
Oral GH secretagogue (LUM-201) enhances growth in individuals with
moderate Pediatric Growth Hormone Deficiency (PGHD) by enhancing
endogenous GH secretion and increasing IGF-1 (Fernando Cassorla,
MD, et al), was given in a late breaking session, Saturday,
September 23, 9:30-10:30 AM CET (Local Time). The slides presented
at ESPE are available on the Posters & Publications page under
the Investors & Media section of Lumos Pharma’s website.
The objective of the analysis was to
characterize growth hormone (GH) profiles, defined by deconvolution
analysis, based on the GH concentration sampled over 12 hours at
baseline and after 6 months of therapy with daily oral LUM-201 to
illustrate how LUM-201 increases annualized height velocity (AHV),
total GH secretion, and serum IGF-1 and IGFBP3 in individuals with
moderate PGHD. Fifteen prepubertal, naive moderate PGHD subjects
were screened with a predictive enrichment marker (PEM) test to
assess their acute response to oral LUM-201 (0.8mg/kg), with a
positive test having a peak GH ≥5 ng/ml with a basal IGF-1 >30
ng/ml. At baseline, subjects (10M:5F) were (mean ± SD) aged 7.9±1.4
years, with IGF-1 SDS -0.82±0.9, and peak GH 7.2±2.2 ng/mL
(clonidine stimulation), consistent with moderate PGHD.
Deconvolution analysis was performed on serum GH
measured every 10 minutes (0800 h to 2000 h). Patients were
randomized to receive 1.6 mg/kg/day or 3.2 mg/kg/day of oral
LUM-201. Both baseline characteristics and acute GH responses to
the PEM test (p=0.9) and the day 1 PEM test doses were not
different between the groups (34.8±6.6 ng/ml for 1.6mg/kg and
38.2±11.2 ng/ml for 3.2mg/kg, p=0.7). The groups were therefore
combined in this analysis.
Results showed that after 6 months of treatment
with LUM-201, GH, IGF parameters, and AHV increased 60-80% (see
Table 1 for means (SD)) from baseline. The study author concluded
that LUM-201 enhanced pulsatile GH secretion to similar levels
observed in normal growing children (estimated at ~3.5 μg/kg/12h;
Albertsson-Wikland et al JCEM 1994), and that restoration of
physiological pulsatile GH secretion and IGF-1 were sufficient to
support normal growth. The study’s author further noted that
LUM-201 in the potential treatment of moderate PGHD has the
advantages of being taken orally, enhancing endogenous pulsatile GH
secretion, and therefore maintaining normal feedback mechanisms to
restore more physiological growth.
Table 1 |
Baseline |
6 Month |
t test, p value |
GH total* |
1.45 (0.89) |
2.32 (1.25) |
0.013 |
GH pulsatile* |
1.28 (0.83) |
1.93 (1.17) |
0.035 |
GH basal * |
0.17 (0.11) |
0.40 (0.28) |
0.008 |
AHV (cm/year) |
4.7 (1.3) |
7.6 (1.1) |
< 0.00001 |
IGF-1 (ng/mL) |
115.5 (46.6) |
205.4 (63.9) |
0.0004 |
IGFBP3 (nmol/L) |
139.3 (32.6) |
169.0 (30.1) |
0.0004 |
IGF-1:IGFBP3 |
0.108 (0.031) |
0.157 (0.050) |
0.0044 |
*daytime secretion μg/kg body weight per 12hr |
About PGHD and the Therapeutic Landscape
PGHD is the consequence of inadequate secretion
of growth hormone from the pituitary gland in children resulting in
low growth hormone (GH) in the body, insufficient production of
downstream signaling molecules required for growth, and the
subsequent lack of growth. The prevailing standard of care for PGHD
consists of a daily injection of exogenous GH administered for
approximately 7 years on average. Several once-weekly injectable GH
therapies have recently been approved in the US and internationally
to treat PGHD. LUM-201, also known as ibutamoren, is an orally
administered investigational small molecule that promotes the
secretion of GH from the pituitary gland and represents an
opportunity for appropriately selected patients to avoid the daily
or weekly injections involved with current or forthcoming
therapies. LUM-201 has been observed to increase the amplitude of
endogenous pulsatile GH secretion, which mimics the natural pattern
of GH secretion.
About Lumos Pharma
Lumos 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 therapeutics that are safe, efficacious,
and offer a 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, progress in our clinical efforts
including the timing of expected results on our LUM-201 trials and
our ability to continue advancing our trials, encouraging interim
data and new analysis presented, that our convictions are further
reinforced that at least one of the LUM-201 dose cohorts will meet
growth expectations based on historical averages, that the LUM-201
mechanism of action and potency can elicit sustained improvements
in growth in the moderate PGHD patient population, continuing to
advance our LUM-201 clinical program for potentially the first oral
therapeutic for PGHD, 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 final results of our
LUM-201 Trials being different than our interim results, the
outcome of our future interactions with regulatory authorities, the
timing and ability of Lumos to raise additional equity capital as
needed to fund our Phase 3 Trial or for other purposes, our ability
to project future cash utilization and reserves needed for
contingent future liabilities and business operations, the ability
to obtain and maintain the necessary patient enrollment for our
product candidate in a 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 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 Annual
Report on Form 10-K for the year ended December 31, 2022, as well
as other reports filed with the SEC. 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
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