Lumos Pharma, Inc. (NASDAQ:LUMO) today announced that topline
results from its Phase 2 OraGrowtH210 dose-finding trial and its
Phase 2 OraGrowtH212 Pharmacokinetic/Pharmacodynamic (PK/PD) trial
met all primary and secondary endpoints. Data from the OraGrowtH210
Trial demonstrated annualized height velocity (AHV) on the 1.6
mg/kg dose of orally administered LUM-201 of 8.2 cm/yr at six
months and 8.0 cm/yr at 12 months on treatment,* in line with
historical data in moderate pediatric growth hormone deficiency
(PGHD) patients and within the targeted 2 cm/yr margin of the
comparator injectable recombinant growth hormone (rhGH) arm. Data
also provided preliminary validation of the predictive enrichment
marker (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 growth rates
while restoring growth hormone secretion and IGF-1 to within normal
ranges †, with levels substantially below those produced by
exogenous injectable rhGH.†† Additionally, data from a small subset
of 10 subjects combined 1.6 and 3.2 mg/kg dosage of LUM-201 in both
OraGrowtH210 and OraGrowtH212 trials demonstrated the sustained
effectiveness of AHV up to 24 months. Furthermore, the safety
profile for LUM-201 remained clean throughout both Phase 2 studies,
with no safety concerns identified in either of our Phase 2 trials
conducted thus far.
“Results from our OraGrowtH trials have provided
us with clear proof of concept that oral LUM-201 has the potential
to serve as a viable alternative to injectable therapies in
moderately growth hormone deficient patients. Our data indicates
that LUM-201 can enhance AHVs in line with established standards
for moderate PGHD patients undergoing rhGH therapy, demonstrating a
robust and durable response,” said Rick Hawkins, Chairman and CEO
of Lumos Pharma. “We look forward to discussing these data and
finalizing our plans for a Phase 3 pivotal trial with the FDA in
our end of Phase 2 meeting anticipated in the first half of
2024.”
Renowned pediatric endocrinologist Dr. Ron
Rosenfeld, who also serves as the Chairman of our Clinical and
Scientific Advisory Board, provided insight on the data, stating,
"These findings not only align with historical growth expectations
on therapy but also underscore the distinct advantage of LUM-201's
unique pulsatile mechanism. Demonstrating the ability to achieve
expected growth with oral LUM-201 while exposing patients to only
20% of the growth hormone compared to daily rhGH injections is a
significant scientific breakthrough that has the potential to
revolutionize the approach to treating children with moderate
growth hormone deficiency."
OraGrowtH210 Topline Results
HighlightsThe OraGrowtH210 trial met its primary
objective, with 6-month AHV data of 8.2 cm/yr supporting the 1.6
mg/kg as the optimal dose for a Phase 3 clinical trial.* The
6-month and 12-month AHV on 1.6 mg/kg/day met expectations for
growth and were within the targeted 2.0 cm/yr margin for
non-inferiority against injectable rhGH cohort.
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, HT SDS-MPH SDS Bars represent Least
Squares Mean (LSM), Error bars represent the Standard Error of
LSM
- Dosage at 1.6 mg/kg demonstrates
highest LUM-201 AHV at six months and 12 months
- 1.7 cm/yr difference between 1.6
mg/kg LUM-201 dose and rhGH comparator arm at 12 months falls
within historical non-inferiority Phase 3 margins
- LUM-201 AHVs align with historical
growth rates of rhGH in patient populations with similar
characteristics.
- 12-month AHV data available for
50/81 subjects: Growth rates durable at 12 months
The mean AHVs at 6 months and 12 months observed
in the 1.6 mg/kg dose LUM-201 arm were 8.2 cm/yr and 8.0 cm/yr,
respectively. These AHVs were in line with the Company’s
expectations for 8.3-8.6 cm/yr AHV observed after 12 months of rhGH
treatment in a moderate PGHD patient population.1,2,3
The higher than anticipated AHV seen in this
moderate PGHD population treated in the rhGH control arm of the
OraGrowtH210 Trial was inconsistent with multiple historical trials
which predicted growth in the 8.3-8.6 cm/yr range for moderate
PGHD1-4. This distinctive growth pattern observed in the daily GH
arm of this study is likely due to a higher dosage and the presence
of outliers. We anticipate that in a larger, more statistically
robust Phase 3 trial, the AHV associated with rhGH treatment will
align more closely with historical values for the moderate patient
population.
The OraGrowtH210 Trial met the prespecified
percent responder enrichment providing preliminary validation of
the PEM strategy. Additionally, we have achieved a 100% success
rate in meeting the predetermined outcome for positive PEM
specification classification reproducibility.
OraGrowtH212 Topline Results
HighlightsThe topline results from the OraGrowtH212 Trial
reveal that LUM-201 achieved an expected AHV with only 20% of the
growth hormone (GH) concentration observed using injectable rhGH.
This outcome was achieved through LUM-201's natural pulsatile
mechanism, promoting growth in moderate PGHD subjects that align
with historical norms. Notably, LUM-201 raised circulating GH to
levels closer to normal physiological ranges, whereas treatment
with injectable rhGH has been shown to elevate GH levels to four to
five times that of typical healthy children. Furthermore, it's
important to highlight that during the first 12 months of LUM-201
treatment, no IGF-1 values exceeded 2 standard deviations from the
mean.
Combined 24-Month Data from OraGrowtH210
and OraGrowtH212 Trials
- Eighteen and 24-month growth data
were available for 10 subjects from the OraGrowtH210 and
OraGrowtH212 Trials who met AHV criteria per protocol at 12
months.
- Combined data from the 1.6 mg/kg
and 3.2 mg/kg cohorts of both trials demonstrate sustained AHVs
from 12 to 24 months without a considerable decline in growth
velocity compared to the previously reported ~20% decline in AHV on
rhGH from 12 to 24 months observed in the Pfizer Phase 4 KIGS
dataset.3
Safety & Tolerability
HighlightsThe topline results from both the OraGrowtH210
and OraGrowtH212 trials have shown a clean safety record,
characterized by an absence of treatment-related Serious Adverse
Events (SAEs), no instances of participants discontinuing treatment
due to adverse events (AEs), and the absence of any significant
safety concerns in various parameters such as laboratory values,
adverse event data, or in electrocardiogram (ECG) readings.
† Zadik et al Horm Res 1992†† Adapted from data
in Albertsson-Wikland et al JCEM 1994; 24h exposures listed reflect
absorbance/bioavailability of ~60% of the administered dose, * 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, for graphs HT SDS-MPH SDS Bars
represent Least Squares Mean (LSM), Error bars represent the
Standard Error of LSM1 Blum et al JES 2021, 2 Lechuga-Sancho et al
JPEM 2009, 3 Ranke et al JCEM 2010, 4 Bright et al JES 2021
Conference Call and Webcast
Details
Date: November 8,
2023Time: 8:30 AM ET Dial-in:
1-877-407-9716 or 1-201-493-6779 (international) Conference
ID: 13742617Or Dial-in registration (Available 15
minutes prior to scheduled start time):
https://callme.viavid.com/viavid/?callme=true&passcode=13742617&h=true&info=company-email&r=true&B=6Webcast
link:
https://viavid.webcasts.com/starthere.jsp?ei=1642841&tp_key=d9efda8a69
Slides are available on the Lumos Pharma website in the
“Investors & Media” section under “Events and Presentations”
link:
https://investors.lumos-pharma.com/events-presentations.
A replay of the call will be available
approximately two hours after the completion of the call and can be
accessed by using the same numbers as above for two weeks following
the call.
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.
OraGrowtH210 Trial DesignThe
OraGrowtH210 Trial is a global, multi-site study that assesses the
effects of orally administered LUM-201 at three different dose
levels (0.8, 1.6, 3.2 mg/kg/day) in comparison to daily injections
of recombinant human growth hormone (rhGH) at a dose of 34
µg/kg/day. This trial involves 82 participants diagnosed with
moderate Pediatric Growth Hormone Deficiency (PGHD). To enrich the
trial population with individuals likely to respond to LUM-201,
specific PEM criteria were applied during the screening process.
These criteria included having a baseline IGF-1 value above 30
ng/ml and achieving a peak growth hormone value of 5 ng/ml or
higher after administering a single 0.8 mg/kg dose of LUM-201 to
treatment-naïve PGHD patients. It is important to note that the
primary purpose of this study was not to establish efficacy or
demonstrate non-inferiority compared to daily GH treatment.
OraGrowtH212 Trial DesignThe
OraGrowtH212 Trial is a single-site, open-label study designed to
assess the pharmacokinetic (PK) and pharmacodynamic (PD) impacts of
oral LUM-201. This trial includes up to 24 individuals with no
prior treatment for Pediatric Growth Hormone Deficiency (PGHD), who
are administered LUM-201 at two different dosage levels,
specifically 1.6 and 3.2 mg/kg/day. Every participant in the
OraGrowtH212 Trial met the criteria for Patient PEM positivity,
ensuring their potential responsiveness to LUM-201.
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 StatementsThis 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 supporting evidence to advance oral LUM-201
to Phase 3, clear proof of concept that oral LUM-201 has the
potential to serve as a viable alternative to injectable therapies
in moderately growth hormone deficient patients, the potential for
LUM-201 to enhance AHVs in line with established standards for
moderate PGHD patients undergoing rhGH therapy, looking forward to
discussing these data and finalizing our plans for a Phase 3
pivotal trial with the FDA in our end of Phase 2 meeting
anticipated in the first half of 2024, that this is a significant
scientific breakthrough that has the potential to revolutionize the
approach to treating children with moderate growth hormone
deficiency, data from the OraGrowtH210 Trial supporting the 1.6
mg/kg dose for LUM-201 as the optimal dose for a Phase 3 trial,
that this distinctive growth pattern observed in the daily GH arm
of this study is likely due to a higher dosage and the presence of
outliers, that in a larger, more statistically robust Phase 3
trial, the AHV associated with rhGH treatment will align more
closely with historical values for the moderate patient population,
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
A photo accompanying this announcement is available at
https://www.globenewswire.com/NewsRoom/AttachmentNg/e14ed9b0-f190-41e0-8c43-236ed34b5ceb
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