Regeneron Pharmaceuticals, Inc. (NASDAQ: REGN) today announced
top-line, two-year (96 weeks) data for aflibercept 8 mg from the
pivotal PHOTON trial in patients with diabetic macular edema (DME).
During the trial, aflibercept 8 mg patients were initially
randomized to either 12- or 16-week dosing intervals (after three
initial monthly doses) and were able to shorten or extend dosing
intervals if pre-specified criteria were met. The longer-term data
among aflibercept 8 mg patients who completed the trial
demonstrated that the vast majority of patients were able to
maintain or further extend these dosing intervals through two years
with:
- 89% maintaining ≥12-week dosing intervals through two years,
compared to 93% through one year (48 weeks)
- 83% maintaining ≥16-week dosing intervals through two years,
compared to 89% maintaining a 16-week dosing interval through one
year
- 43% meeting the criteria for ≥20-week dosing intervals by
week 96, including 16% and 27% who were eligible for 20- and
24-week dosing intervals, respectively
“The two-year PHOTON results for aflibercept 8 mg in patients
with diabetic macular edema are extremely compelling,” said Jeffrey
Heier, M.D., Director of the Retina Service and Retina Research
at Ophthalmic Consultants of Boston and a trial
investigator. “To be able to rapidly achieve extended dosing
intervals without any sacrifice of vision gains over two years is a
tremendous benefit in the treatment of diabetic macular edema.”
“The two-year PHOTON results certainly exceeded my expectations
and indicate that the majority of patients may eventually be able
to control their diabetic macular edema with as few as two or three
aflibercept 8 mg injections per year, if approved by regulatory
authorities, with similar excellent visual gains and a safety
profile consistent with EYLEA given every 8 weeks,” said David M.
Brown, M.D., FACS, Director of Research at Retina Consultants of
Texas and a trial investigator. “Reducing the treatment burden in
patients with diabetic macular edema is a critical unmet need, and
the two-year PHOTON results reinforce the potential of aflibercept
8 mg to become the standard of care for the treatment of diabetic
macular edema.”
PHOTON (N=658) is a double-masked, active-controlled pivotal
trial evaluating non-inferiority of aflibercept 8 mg 12-week
(n=328) and 16-week (n=163) dosing regimens after three initial
monthly doses compared to an 8-week dosing regimen for EYLEA
(aflibercept) Injection (n=167) after five initial monthly doses.
In addition to the vast majority of trial patients maintaining
extended dosing intervals through two years, visual gains for
aflibercept 8 mg remained consistent with the first year of the
trial.
|
Through 48 weeks (one year) |
|
Through 96 weeks (two years) |
|
EYLEA8-week regimen |
aflibercept 8
mg12-weekregimen |
aflibercept 8
mg16-weekregimen |
|
EYLEA8-week regimen |
aflibercept 8
mg12-weekregimen |
aflibercept 8
mg16-weekregimen |
Mean number of injections^ |
7.9 |
6.0 |
5.0 |
|
13.8 |
9.5 |
7.8 |
Mean observed BCVA improvement, letters |
9.2 |
8.8 |
7.9 |
|
8.4 |
8.8 |
7.5 |
LS mean (SE) change from baseline, letters |
8.7 (0.7) |
8.1 (0.6) |
7.2 (0.7) |
|
7.7 (0.9) |
8.2 (0.6) |
6.6 (0.8) |
Difference in LS mean (95% CI), letters |
|
-0.6*(-2.3, 1.1) |
-1.4†(-3.3, 0.4) |
|
|
+0.5‡(-1.6, 2.5) |
-1.1§(-3.3, 1.1) |
Proportion of patients losing ≥15 letters, per
LOCF |
1.2% |
2.1% |
0.6% |
|
3.6% |
3.4% |
1.2% |
BCVA: best corrected visual acuity; LS: least squares; SE:
standard error; LOCF: last observation carried forward^Based on
patients completing week 48 or 96 in the trial*Non-inferiority
p-value: p<0.0001†Non-inferiority p-value: p=0.0031‡Nominal
non-inferiority p-value: p<0.0001§Nominal non-inferiority
p-value: p=0.0044
In PHOTON, the safety of aflibercept 8 mg also continued to be
similar to EYLEA through two years and remained consistent with the
known safety profile of EYLEA from previous clinical trials for
DME. Ocular treatment emergent adverse events (TEAE) occurring in
≥5% of patients in any treatment group, in decreasing frequency,
were cataract, vitreous floaters, and conjunctival
hemorrhage. There were no cases of retinal vasculitis,
occlusive retinitis or endophthalmitis. The rate of intraocular
inflammation was 1.2% for both the EYLEA and aflibercept 8 mg
groups. Anti-platelet trialists' collaboration-defined
arterial thromboembolic TEAEs occurred in 7.2% of patients treated
with EYLEA and 6.5% of patients treated with aflibercept 8 mg.
“The aflibercept 8 mg clinical trial program is the first to
demonstrate that patients with diabetic macular edema can
immediately be treated with every 12- or 16-week dosing after their
initial monthly doses and experience lasting vision control,” said
George D. Yancopoulos, M.D., Ph.D., Board Co-Chair, President and
Chief Scientific Officer at Regeneron, and a principal inventor of
EYLEA. “With these two-year results, Regeneron continues to raise
the bar in clinical advancements for retinal treatments and remains
committed to pursuing groundbreaking innovations in
ophthalmology.”
The two-year data from the pivotal PULSAR trial for aflibercept
8 mg in wet age-related macular degeneration are expected in the
third quarter of 2023, and the two-year data from both PHOTON and
PULSAR are planned for presentation at an upcoming medical
meeting.
Aflibercept 8 mg is investigational, and its safety and efficacy
have not been fully evaluated by any regulatory
authority. Aflibercept 8 mg is being jointly developed by
Regeneron and Bayer AG, with Regeneron sponsoring the PHOTON trial.
In the U.S., Regeneron maintains exclusive rights to EYLEA and
aflibercept 8 mg. Bayer has licensed the exclusive marketing rights
outside of the U.S., where the companies share equally the profits
from sales of EYLEA and aflibercept 8 mg following any regulatory
approvals.
About the Aflibercept 8 mg Clinical Trial
Program PULSAR in wAMD and PHOTON in DME are
double-masked, active-controlled pivotal trials that are being
conducted in multiple centers globally. In both trials, patients
were randomized into 3 treatment groups to receive either:
aflibercept 8 mg every 12 weeks, aflibercept 8 mg every 16 weeks,
or EYLEA every 8 weeks. The lead sponsors of the trials were Bayer
for PULSAR and Regeneron for PHOTON.
Patients treated with aflibercept 8 mg in both trials had 3
initial monthly doses, and patients treated with EYLEA received 3
initial doses in PULSAR and 5 in PHOTON. In the first year,
patients in the aflibercept 8 mg groups could have their dosing
intervals shortened down to an every 8-week interval if
protocol-defined criteria for disease progression were observed.
Intervals could not be extended until the second year of the study.
Patients in all EYLEA groups maintained a fixed 8-week dosing
regimen throughout their participation in the trials.
About DMEDME is a common complication in eyes
of people living with diabetes. DME occurs when high levels of
blood sugar lead to damaged blood vessels in the eye that leak
fluid into the macula. This can lead to vision loss and, in some
cases, blindness. Of the nearly 28 million American adults living
with diabetes, an estimated 1.2 million have DME.
About Ophthalmology at RegeneronAt Regeneron,
we relentlessly pursue groundbreaking innovations in eye care
science to help maintain the eye health of the millions of
Americans impacted by vision-threatening conditions. Over a decade
ago, our breakthrough scientific research resulted in the
development of EYLEA, a vascular endothelial growth factor (VEGF)
inhibitor designed to block the growth of new blood vessels and
decrease the ability of fluid to pass through blood vessels in the
eye. EYLEA has since brought fundamental change to the retinal
disease treatment landscape and is supported by a robust body of
research that includes eight pivotal Phase 3 trials, 11 years of
real-world experience, and more than 60 million EYLEA injections
globally.
Regeneron continues to advance our anti-angiogenesis expertise
with new solutions with the aim of offering optimal flexibility for
a broad group of patients and eye care professionals. This includes
aflibercept 8 mg, which has been developed with the aim of
extending the time between injections, while maintaining the vision
gains, anatomic benefits and safety previously observed with
EYLEA.
IMPORTANT EYLEA SAFETY INFORMATION AND
INDICATIONS
INDICATIONSEYLEA (aflibercept) Injection 2 mg
(0.05 mL) is indicated for the treatment of patients with
Neovascular (Wet) Age-related Macular Degeneration (AMD), Macular
Edema following Retinal Vein Occlusion (RVO), Diabetic Macular
Edema (DME), Diabetic Retinopathy (DR) and Retinopathy of
Prematurity (ROP) (0.4 mg [0.01 mL]).
CONTRAINDICATIONS
- EYLEA is contraindicated in patients with ocular or periocular
infections, active intraocular inflammation, or known
hypersensitivity to aflibercept or to any of the excipients in
EYLEA.
WARNINGS AND PRECAUTIONS
- Intravitreal injections, including those with EYLEA, have been
associated with endophthalmitis and retinal detachments. Proper
aseptic injection technique must always be used when administering
EYLEA. Patients and/or caregivers should be instructed to report
any signs and/or symptoms suggestive of endophthalmitis or retinal
detachment without delay and should be managed appropriately.
Intraocular inflammation has been reported with the use of
EYLEA.
- Acute increases in intraocular pressure have been seen within
60 minutes of intravitreal injection, including with EYLEA.
Sustained increases in intraocular pressure have also been reported
after repeated intravitreal dosing with VEGF inhibitors.
Intraocular pressure and the perfusion of the optic nerve head
should be monitored and managed appropriately.
- In infants with ROP, reactivation of abnormal angiogenesis and
tortuosity may occur following treatment with EYLEA. Infants should
be monitored closely after injection with EYLEA until retinal
vascularization has completed or until the examiner is assured that
reactivation of ROP will not occur. Treatment with EYLEA will
necessitate extended periods of ROP monitoring and additional EYLEA
injections and/or laser treatments may be necessary.
- There is a potential risk of arterial thromboembolic events
(ATEs) following intravitreal use of VEGF inhibitors, including
EYLEA. ATEs are defined as nonfatal stroke, nonfatal myocardial
infarction, or vascular death (including deaths of unknown cause).
The incidence of reported thromboembolic events in wet AMD studies
during the first year was 1.8% (32 out of 1824) in the combined
group of patients treated with EYLEA compared with 1.5% (9 out of
595) in patients treated with ranibizumab; through 96 weeks, the
incidence was 3.3% (60 out of 1824) in the EYLEA group compared
with 3.2% (19 out of 595) in the ranibizumab group. The incidence
in the DME studies from baseline to week 52 was 3.3% (19 out of
578) in the combined group of patients treated with EYLEA compared
with 2.8% (8 out of 287) in the control group; from baseline to
week 100, the incidence was 6.4% (37 out of 578) in the combined
group of patients treated with EYLEA compared with 4.2% (12 out of
287) in the control group. There were no reported thromboembolic
events in the patients treated with EYLEA in the first six months
of the RVO studies.
ADVERSE REACTIONS
- Serious adverse reactions related to the injection procedure
have occurred in <0.1% of intravitreal injections with EYLEA
including endophthalmitis and retinal detachment.
- The most common adverse reactions (≥5%) reported in patients
receiving EYLEA were conjunctival hemorrhage, eye pain, cataract,
vitreous detachment, vitreous floaters, and intraocular pressure
increased.
- In pre-term infants with ROP receiving EYLEA the most common
adverse reactions (≥4%) reported were retinal detachment,
conjunctival hemorrhage, and intraocular pressure increased.
Adverse reactions established for adult indications are considered
applicable to pre-term infants with ROP, though not all were
observed in the clinical studies.
- Patients may experience temporary visual disturbances after an
intravitreal injection with EYLEA and the associated eye
examinations. Advise patients not to drive or use machinery until
visual function has recovered sufficiently.
For more information, please see full Prescribing
Information.
About RegeneronRegeneron (NASDAQ: REGN) is a
leading biotechnology company that invents, develops and
commercializes life-transforming medicines for people with serious
diseases. Founded and led for 35 years by physician-scientists, our
unique ability to repeatedly and consistently translate science
into medicine has led to 9 FDA-approved treatments and numerous
product candidates in development, almost all of which were
homegrown in our laboratories. Our medicines and pipeline are
designed to help patients with eye diseases, allergic and
inflammatory diseases, cancer, cardiovascular and metabolic
diseases, pain, hematologic conditions, infectious diseases and
rare diseases.
Regeneron is accelerating and improving the traditional drug
development process through our proprietary VelociSuite®
technologies, such as VelocImmune®, which uses unique genetically
humanized mice to produce optimized fully human antibodies and
bispecific antibodies, and through ambitious research initiatives
such as the Regeneron Genetics Center®, which is conducting one of
the largest genetics sequencing efforts in the world.
For more information, please visit www.Regeneron.com or follow
@Regeneron on Twitter.
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litigation relating thereto (including without limitation the
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EYLEA® (aflibercept) Injection, Praluent® (alirocumab), and
REGEN-COV® (casirivimab and imdevimab)), other litigation and
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Contacts:Media RelationsMary
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