2020 p. 7 Progressing development of therapies for retinal vascular disorders and diabetic eye disease - Oxurion
←
→
Page content transcription
If your browser does not render page correctly, please read the page content below
2020 Progressing development of "We focus on non-VEGF Researching the dry AMD therapies for retinal vascular pathways" pathway disorders and diabetic eye disease p. 7 p. 11 p. 32
2020
contents
PROGRESSING DEVELOPMENT
OF THERAPIES FOR RETINAL
VASCULAR DISORDERS AND
DIABETIC EYE DISEASE
Patrik De Haes, M.D., CEO &
Thomas Clay, Chairman of the Board of
Directors
p. 7
"WE FOCUS ON NON-VEGF THR-149, A HIGHLY POTENT THR-687, A POTENT PAN-RGD “OXURION IS WELL-POSITIONED MAKING A DIFFERENCE NEXT GENERATION I/O
PATHWAYS" PLASMA KALLIKREIN INTEGRIN ANTAGONIST IN TO DEVELOP PATHWAYS TO IN THE GLOBAL RETINA CANCER THERAPIES
INHIBITOR TO TREAT DME DEVELOPMENT FOR DME ADDRESS DRY AMD” COMMUNITY
Ongoing clinical research
Jean Feyen, PhD., CSO, Preparing a phase 2 clinical trial Preparing a phase 2 clinical trial Prof. Alan Stitt of Queen’s University Oxurion joins forces with Prevent
and Andy De Deene, M.D., Head of in Belfast Blindness and Retina Global
Development
p. 11 p. 16 p. 24 p. 28 p. 32 p. 37
6 contents
Editorial contents
Editorial 7
Interview Patrik De Haes, M.D., CEO of Oxurion & Thomas Clay, Chairman of the Board
Progressing development of
therapies for retinal vascular
disorders and diabetic eye disease
2019 has been another transformational year for Oxurion. In this first year after the company's
rebranding, we conducted three clinical trials. The results of two were encouraging and promising,
those of the third rather mixed. Oxurion then made the strategic decision to focus its research on
developing non-VEGF compounds to target an unmet medical need for people with diabetic retinal
eye disease.
“2019 has been an important year for Different course, ... “Our molecules are
our clinical developments. We obtained
excellent results from two phase 1 clin- Thomas Clay, Chairman of the Board,
unique, and potentially
ical trials evaluating two distinct and confirms this. “Oxurion had two very first in class.”
wholly-owned non-VEGF compounds: successful phase 1 trials. The data from
THR-149 and THR-687. These have our study evaluating anti-PIGF (THR-317) Patrik De Haes, M.D., CEO of Oxurion
given us a solid basis, a confirmation of for DME were clinically sound but did not
our new focus, and a growth platform for show the outcome we were hoping for.
the company's future development", says The choice to leave that pathway was Finally, in 2019 Oxurion also decided
Patrik De Haes, M.D., CEO of Oxurion. obvious so we took that decision.” to explore and research innovative
new therapies for age-related macular
“The results from the clinical trials for “We also decided to stop actively pro- degeneration (AMD). AMD is a leading
our molecules THR-149 and THR-687 moting Jetrea® ourselves and seek a cause of blindness in the world and
are indeed very promising. Both com- distribution licensee partner", Patrik De comes in two varieties. There are treat-
pounds not only proved to be very safe Haes adds. "With Inceptua Group we've ments for wet AMD, but for dry AMD
and well-tolerated, they also showed found such a partner. In March 2020, there are none. “The focus of the Oxurion
a substantial, clinically relevant early Oxurion and Inceptua Group signed a preclinical research teams is currently
benefit and durability of effect.” This truly global license agreement for further almost exclusively on in-vivo and in-vitro
ticks all the boxes in developing the next- commercialization of Jetrea®. With that, validation of new pathways and com-
generation therapy for retinal disease. we fulfilled our commitment to not pounds for the treatment of dry AMD.
only secure access to Jetrea® for those We hope to be able to present at least
patients we believe can truly benefit from one preclinical proof of concept in 2020",
this first-in-class medicine, but to do it in Patrik continues.
a cost-neutral manner for the Company.”
Perspectives 2020 Perspectives 2020
8 Editorial Editorial 9
... the same commitment strategic one. When 40% of DME patients THR-149 is a plasma kallikrein inhibitor to “Of course, the data will speak for them- Oxurion proudly supports Retina Confidently looking to
respond poorly to an anti-VEGF therapy, treat people with DME. This novel therapy selves, but we feel confident. After all, Global’s efforts to educate people on the future
While Oxurion's strategic course may common sense says we should look yielded encouraging results in the phase the Oxurion team has the important eye disease and proper eye care and
have been fine-tuned in 2019, the at non-VEGF therapies for them. With 1 clinical trial, producing fast and durable experience of successfully developing to send retina specialists to remote and “Oxurion has completed its transfor-
commitment and focus have remained THR-149 and THR-687, Oxurion has two improvement of vision. a medicine from the research bench all less privileged corners of the world to mation to a full-fledged drug develop-
unchanged: developing therapies for unique and wholly owned compounds in the way to the market. We know the way", provide specialized eye care to those ment company with a clear focus.
people with diabetic eye disease or clinical development that are very distinct Read all about the study and clinical says Patrik. in need.
age-related macular degeneration. from any of the anti-VEGF therapies now results of THR-149 on page 16. It now has two exciting phase 2 clinical
available or under development. Not only In the past, we have joined forces with trials under way evaluating two distinct
they represent a great opportunity for Part of a global Prevent Blindness in order to boost and very innovative non-VEGF com-
"We bring experts and many DME patients out there, they also retina family their ‘Diabetic Eye Disease Awareness pounds for treatment of DME. We've
investors together, so offer our shareholders and investors the
best chance for future value creation. It is
The results from the phase 1 evalua-
tion of THR-687, a pan-RGD integrin Whilst advancing its retina and vision
Month’-campaigns. Going forward,
we are looking into how we can assist
also taken our first steps into the dry
AMD arena with its large untapped
the retina experts can obvious that should we succeed in craft- antagonist, are promising. An almost research, Oxurion seeks to preserve and support Prevent Blindness while potential and aim to make a substantial
explain directly the ing a novel pathway towards a therapy
for dry AMD, we expect this to also see
immediate, significant and durable
mean BCVA improvement of vision
close ties with those in the retina com-
munity and the patients they serve. Over
staging their brand-new nationwide
patient engagement and advocacy
impact there", Patrik De Haes states.
need for compounds this translated into additional value for was reported the years it has developed close colla- training program. At Oxurion, we also “We view the future with confidence
we are developing." our shareholders.” borative ventures with US and global
organizations such as Prevent Blindness
believe that patients are among the best
placed to help create awareness about
and ambition", Thomas Clay concludes.
Thomas Clay, Chairman of the Board "Oxurion does not hesitate to bring Read all about the study and clinical and Retina Global. vision-threatening conditions, disease
investors and experts together. We want results of THR-687 on page 24. processes and the impact of these
our shareholders and investors to hear diseases on daily life.
“Our therapeutic approach also means from key opinion leaders familiar with
focusing resources, time, and energy our programs why these are innovative
on compounds we believe have the and vital for patients”, Thomas continues. The two compounds target different
potential to make a life-changing "Meanwhile, we feel very encouraged to patient groups. THR-149 will be evaluated
improvement in a person’s vision. For see key retina opinion leaders in Europe in patients who do not or do not optimally
many DME patients there is still a large and the US also confirm the potential react to anti-VEGF treatment (non-
unmet need. More than 40% of them of THR-149 and THR-687 for treatment responders/poor responders), while anti- In memoriam Claude Sander
have a suboptimal or zero response to of DME.” VEGF treatment naïve patients will be With profound sadness the Board, Management and Staff of Oxurion learned
treatment with an anti-VEGF, currently selected in the THR-687 phase 2 study. about the sudden passing of Dr Claude Sander on December 20th, 2019.
the standard of care for the bulk of Both studies will test whether multiple Claude Sander was Chief Legal Officer and Corporate Secretary to the
DME patients. For them we need Recruiting for two phase 2 doses the compounds can increase and board. He started his professional career as a Senior Researcher at the
to find a solution, and we have two trials prolong the visual improvement observed Center of European Integration Studies in Bonn. He joined Oxurion in 2011
validated options in clinical develop- in the phase 1 studies. and oversaw Legal, Corporate Compliance, Corporate Board and selected
ment", Patrik explains. Oxurion is preparing the start of two fully Market Access related activities at Oxurion.
powered phase 2 clinical trials, next to Oxurion believes that if these fully
the validation of new pathways towards powered phase 2 trials replicate and Patrik De Haes, M.D., CEO of Oxurion: “It was a privilege and pleasure to have
Value creation a therapy for dry AMD. Final approvals maintain the observed benefit from the known Claude and to have worked with him for almost a decade. Claude
for the phase 2 clinical trial evaluating phase 1 studies, both compounds would always demonstrated exceptional leadership in his role as Chief Legal Officer
“This strategy is also interesting for our THR-149 in DME are well underway, and become very compelling drug candidates and more recently as Corporate Secretary to the Board, even in challenging
shareholders and potential investors", Oxurion is making great headway with the with clear value to the market. situations. He was both admired and respected for his relentless commit-
Thomas Clay points out. "Our choice prep work for its phase 2 with THR-687 ment, expertise and achievements. Claude will be deeply missed and will
to develop non-VEGF compounds is a as well. always have a very special place in our hearts.”
Perspectives 2020 Perspectives 2020
10 A strategic focus A strategic focus 11
A strategic focus Interview Jean Feyen, PhD., Chief Scientific Officer and Andy De Deene, M.D., Global Head of Development
Oxurion rises to the opportunity to address an unmet "We focus on non-VEGF pathways"
medical need.
Oxurion has a mission: to prevent loss of vision loss and blindness by developing solutions for
retinal diseases generally, and specifically for diabetic macular edema (DME) and age-related
macular degeneration (AMD). To realize this ambition the company is focusing on non-VEGF
pathways and compounds. Andy De Deene, M.D., Global Head of Development, and Jean Feyen,
PhD., Chief Scientific Officer, explain this strategic direction.
The course may have been fine- DME, meaning there is no effective Two compounds, two
tuned but the goal remains: to find treatment for the other 40%. Exploring target groups
treatments for retinal diseases such as non-VEGF pathways might provide a
DME and AMD. chance to better address the current Th e co m p o un ds THR-149 an d
unmet medical need of those 40% THR-687 use a different pathway than
“The change means first and foremost DME patients." anti-VEGF. “Each of these molecules
leaving the VEGF pathway", Jean says. has its own target group", Jean con-
"The decision to stop the development In parallel with this, Oxurion is seeking tinues. “THR-149, our plasma kallikrein
of our THR-317 compound was com- to lower the burden for patients by inhibitor, is a VEGF-independent com-
pletely data-driven. THR-317 was a clin- developing a treatment requiring less pound specifically used to treat the
ically sound compound, but compared frequent follow-up. “We're looking accumulation of fluids in the retina.
to others it made much more sense to for a way to not just suppress symp- As such it can improve vision. This
focus our time, energy and resources toms but also modify the disease", compound only tackles some aspects
on developing non-VEGF pathways". Andy adds. of DME and is meant for people who
have no or suboptimal response to
The importance of non-VEGF path- anti-VEGF injections".
ways cannot be overestimated. “At
present the standard of care for DME
is a monthly injection into the eye with
an anti-VEGF medicine", Andy explains. "We're a data-driven biotech
"An anti-VEGF is a good product but
it only works for 60% of those with
company. Every decision is
supported by data. We believe
this is the best way to select
which pathways to develop."
Jean Feyen, PhD., Chief Scientific Officer
Perspectives 2020 Perspectives 2020
12 A strategic focus A strategic focus 13
THR- 687 is “a pan -RGD inte g - these clinical trials we hoped for an currently, there is no treatment avail- Once information can be released, every opportunity. That is the only way
rin antagonist and a non-VEGF effect, but were surprised to see these able. We have the chance to address it is crucial that it flows easily to the to give our board well-informed advice
compound. It has a broad mode impressive improvements this fast this problem and improve the lives of scientific community". about molecules and pathways".
of action and addresses different after the injection. One injection with many people. For this we are collab-
aspects of DME such as permeability, THR-149 yielded an improvement orating with, among others, Professor “And the opposite is also true", Jean “We're a data-driven biotech company.
inflammation, angiogenesis, and in vision with 6.4 letters after three Alan Stitt and Queen’s University in adds. “Our team members must know Every one of our decisions is supported
fibrosis. Potentially it could treat dia- months. THR-687 performs even Belfast", Andy explains. Oxurion aspires every development, every novelty, by data. We believe this is the best way
betic retinopathy (with and without better. This kind of improvement is too to bring at least one dry AMD project every breakthrough in this field. We to select which pathways to develop",
DME) and the wet variety of age- significant to be the result of chance", to development. expect them to be on top of their Jean concludes.
related macular degeneration (AMD). Andy explains. game, to test, experiment, and evaluate
THR-687 does what an anti-VEGF Read more about this phase 1
does and more. This allows us to focus Read more about this phase 1 clinical study of THR-149 on
on treating naïve patients: those who clinical study of THR-149 on page 24.
have never received treatment for dia- page 16.
betic eye disease". Leading causes of blindness *
Top team delivering
Fast & A chance to address high-quality work
durable improvement dry AMD 1. 2. 3. 4. 5.
Collaborating with universities, both in Age-related macular Cataract Glaucoma Diabetic Trachoma
In 2019, Oxurion conducted two Oxurion is not staking its future on Europe and the United States, is crucial degeneration (AMD) retinopathy (DME)
phase 1 clinical trials to assess the these two compounds alone. “We're for Oxurion. “To put us on the scien-
safety of both THR-149 and THR-687. always looking for ways to feed the tific map we must deliver high-quality
We are currently preparing for two pipeline and develop novel therapies work. Getting articles published in
phase 2 trials. for treating back of the eye diseases", peer-reviewed scientific journals is one * Source: World Health Organisation: www.who.int
Jean says. In 2019, Oxurion made the measure of quality. We are very lucky
“In testing treatment for eye diseases strategic choice to enter the pathway to work with a team of very talented
you want to see a fast and durable of dry AMD. people who have excellent success in
improvement of vision. Already in the publishing articles in scientific journals",
phase 1 clinical trials we noticed signs “This past year our R&D teams have Andy observes. “Exchanging know-
of efficacy even with the limited group been working on dry AMD. This ledge is critical in scientific research.
of test subjects. When we started disease affects many people and
“Exchanging knowledge is critical in scientific research.
Once information can be released, it is crucial that it
flows easily to the scientific community. We are lucky to
work with a team of talented people who succeed in
sharing results."
Andy De Deene, M.D., Global Head of Development
Perspectives 2020 Perspectives 2020
14 THR-149 THR-149 15
THR-149
In its drive to find solutions to treat diabetic eye disease
and prevent loss of vision and blindness, Oxurion is
developing and testing several compounds. One of
these compounds for treating diabetic macular
edema is THR-149.
What is THR-149? Whom is THR-149 for? What is retinal inflammation?
Inflammation is a biological response of the body. Retinal inflammation is
THR-149 is a plasma kallikrein inhibitor. It’s a non-VEGF that PKal inhibition could be an interesting proposition as an eye condition that causes vision impairment or, in the most severe cases,
inhibits DME independent of the VEGF pathway. a monotherapy in VEGF poor-responding patients or vision loss. Symptoms include blurred vision, sensitivity to light, and floaters in
in combo therapy with VEGF as an effective target for the eye, which may occur in one or both eyes.
Plasma kallikrein (PKal) is an enzyme crucial in the forma- treating DME.
tion of bradykinins. Increased bradykinin levels result in the
formation of edema and inflammation. Over 40% of people with DME have little or no benefit What is vascular permeability?
from anti-VEGF treatment: their vision or the swelling of
the retina does not improve. For them, non-VEGF therapies Vascular permeability means the blood vessel wall is disrupted, causing fluids
How does THR-149 work against DME? might make a big difference. to penetrate the retina. Abnormal permeability causes edema in the retina,
causing vision impairment or even vision loss. Symptoms include increasing
Literature data show that patients with DME have both ele- number of floaters, floaters with flashes, shadows in peripheral vision, grey
vated levels of plasma kallikrein and VEGF in the vitreous curtain over vision and sudden decrease in vision.
compartment. The elevated plasma kallikrein levels occur
in a wide concentration range of VEGF varying from
undetectable to high levels.
Inhibiting plasma kallikrein, as THR-149 does, can stop Collaboration with Bicycle Therapeutics
inflammation, permeability and the formation of micro-
hemorrhages in DME patients. As such it improves eyesight. THR-149 has been developed in partnership with Bicycle Therapeutics
(Nasdaq: BCYC). Oxurion holds the exclusive license to the PKal inhibitor
portfolio originating from this partnership.
Perspectives 2020 Perspectives 2020
16 THR-149 THR-149 17
Clinical update THR-149
One adverse event was deemed
related to study treatment (likely injec-
THR-149, a highly potent plasma tion procedure). All ocular adverse
events were likely due to the injection
procedure, underlying disease pro-
kallikrein inhibitor to treat DME gression or concomitant diseases.
Signs of efficacy
In 2019, Oxurion announced positive topline data for the compound THR-149, a highly potent plasma
kallikrein inhibitor to treat diabetic macular edema (DME). Not only was THR-149 safe and well-tolerated, Signs of efficacy were also observed in
the phase 1 clinical trial also showed fast onset of action in BCVA from day 1, following a single injection. “I this clinical trial. Efficacy of treatments THR-149-001: Mean Change in BCVA From Baseline (Accounted for Rescue)
am very encouraged to see signs of efficacy so early post-treatment, and to observe a clear durable benefit for diabetic macular edema (DME) All Treated Subjects, Overall
to the patient’s vision as measured by BCVA”, explains Dr. Pravin Dugel, M.D. is determined by measuring best
corrected visual acuity (BCVA), mean 15
central subfield of thickness (CST), and 7.5
6.4
10 6.4
Anti-VEGF is the standard of care for macular volume (MV). BCVA relates 4.8 5.2
3.9
DME patients at the moment, but 40% The macula is a very directly to vision, while CST and MV 5
small, sensitive spot at
has little or no benefit of these treat- measure the edema level. 0
the center of the 0
ments. By targeting a VEGF independ- retina. It is responsible DL D1 D7 D14 M1 M2 M3
for our central vision
ent pathway, an alternative treatment and color sight.
Impressively, a single injection with
can be developed for poor or non- THR-149 improved BCVA from day 1
responders to anti-VEGF treatment. with 3.9 letters.
THR-149-001: Mean Change in CST From Baseline (Accounted for Rescue)
THR-149 is a plasma kallikrein (PKal) The mean improvement in BCVA was All Treated Subjects, Overall
inhibitor, a preclinically well validated highest at day 14 (7.5), and improve-
compound to prevent the induction of ment was maintained at 6.4 letters 100
30.4
26.4 30
retinal vascular permeability (leading after 90 days. 50 10.5
20.4
to edema), inflammation and the Vitreous chamber is The retina is a thin layer 0
0
located behind the lens of tissue on the inside of
prevention of microhemorrhages. and before the optic the back of the eye. It
Impact on swelling
-50 -18
Literature data show that patients with nerve. It is filled with a absorbs light and sends
thick, clear gel-like visual signals to the -100
DME have elevated levels of plasma substance called the brain, which processes DL D1 D7 D14 M1 M2 M3
kallikrein and that the vitreous level vitreous body. them into images. Changes in CST were marginal on day
of plasma kallikrein varies less com- 1, followed by increase until study end.
pared to VEGF, making it a potentially Mean CST change was minimal and
more effective target for the treatment within the variability of measurement.
of DME. patients with visual impairment due Safety confirmed This means that changes in BCVA
to center-involved DME. Three doses seem to be unrelated to changes in
were tested: 3 patients were injected In the phase 1 clinical trial for THR-149 CST.
Study set-up with 0.005 mg (‘low dose’), 3 with the no dose-limiting toxicities and no
middle dose (0.021 mg), and 6 with a serious adverse events were reported,
The phase 1 clinical trial for THR-149 high dose (0.125 mg). All 12 completed indicating THR-149 is safe to use and
evaluated the safety of a single the study and attended all study visits well-tolerated. This means THR-149 is
intravitreal injection of THR-149 in 12 from day 0 to day 90. safe to use and well-tolerated.
Perspectives 2020 Perspectives 2020
18 THR-149 THR-149 19
This clinical trial also linked macular treatment seems to be very good at the next stage of THR-149’s clinical
volume with BCVA improvement: lower stopping permeability but doesn’t work development. They also demonstrate
macular volume is apparently indica- on inflammation. Preclinical models of that THR-149 has the clinical profile to
tive of better BCVA response. Macular diabetes show that PKal mediates vas- potentially become the best-in-class
volume was maintained over time in cular hyperpermeability, leukocytosis, PKal inhibitor and VEGF-independent
patients defined as BCVA responders inflammation, and microhemorrhages. therapy for treatment of DME”, says
among patients with 10 or more letters As a Pkal inhibitor, THR-149 is very pro- Patrik De Haes, M.D., CEO of Oxurion.
improvement in at least two con- mising because it allows us to possibly
secutive visits. “Macular volume could find a different mechanism of action”, THR-149 could become a stand-alone
potentially become a new anatomical Dr. Dugel explains. therapy for suboptimal responders to
predictor for BCVA improvement”, the current standard of care (anti-VEGF
Pravin Dugel, M.D. notes. injections). Or it could potentially be a
Potentially best-in-class therapy for all DME patients in com-
bination with anti-VEGF treatment. As
Very promising The results of the phase 1 study such, it could improve the vision (and
take THR-149 to a phase 2 clinical lives) of millions of DME patients.
“Diabetic macular edema evolves from study with multiple injections. “These
being driven by permeability to being positive findings give us the informa-
driven by inflammation. Anti-VEGF tion and confidence needed to plan
Measuring CST or BCVA? BCVA Preparing a phase 2 study
Diabetic macular edema is measured primarily by Best corrected vision acuity (BCVA) is the measurement Oxurion is currently preparing a phase 2 trial for THR-
macular thickness (CST) or macular volume (MV). of the best vision correction achievable with tools like 149 evaluating whether multiple doses of the com-
It is thought that for BCVA to improve CST must glasses or lenses. Vision acuity is measured on the pound can increase and prolong the visual improve-
come down. THR-149 indicates it is more compli- standard Snellen eye chart. 20/20 vision is the aim: ment observed in the phase 1 study. THR-149 will be
cated than this. In this trial, one injection with THR- if you can pass that with your glasses, your BCVA is evaluated in patients who do not or do not optimally
149 did not improve CST, but it did enhance vision 20/20. There are many conditions that can cause bad react to anti-VEGF treatment (non-responders/poor
with more than 6 letters for at least 90 days with a vision, the most common being myopia (nearsighted- responders). Oxurion believes that if the phase 2 trial
single injection. ness) or hyperopia (farsightedness). Retinal disorders like replicates and maintains the observed benefit from
diabetic macular edema (DME) or age-related macular the phase 1 study, the compound can become a com-
degeneration (AMD) are also a leading cause of bad pelling drug candidate with clear value to the market.
visual acuity, so Oxurion is working to solve them.
Perspectives 2020 Perspectives 202020 THR-149 THR-149 21
Interview Pravin Dugel, M.D.
Diabetic macular edema
“Biological signal for efficacy of What is DME?
Diabetic macular edema (DME) is a diabetic eye disease
caused by high glucose (blood sugar) levels. It damages and
Current Treatment of DME
DME can be treated with lasers, surgery, or medications
injected into the eye to decrease or stop swelling of
THR-149” weakens the blood vessels in the eye, causing them to leak
fluids. The result is swelling of the macula and eventually
diabetic retinopathy.
the macula.
Anti-VEGF injections block the protein that can make
abnormal blood vessels grow and leak fluid. Injections of
“As an investigator of THR-149, I was encouraged to see the results from its clinical phase 1 trial. The macula is responsible for sharp detailed vision, so these corticosteroid are also a possible treatment for DME. This
The molecule was found to be safe, very safe even, and we got a biological signal that it worked," leaks can seriously degrade vision and in extreme cases medication reduces inflammation and swelling of the mac-
Pravin Dugel, M.D. says. even cause blindness. ula, but its side effects include pain and bruising around the
injection site, face flushing, thin or pale skin, and insomnia.
Prevalence of DME At present, anti-VEGF medications are the first choice
The improvement in vision Why do we need a What is the future Diabetes affects some 415 million people worldwide. Of of treatment.
was measured only by BCVA, VEGF-independent pathway? of THR-149? these, 21 million (4.6%) were diagnosed with DME in the
not in OCT. Why is that? period 2015 to 2019. DME levels are highest in Africa Why are new therapies needed?
“Because a significant part of DME “THR-149 holds the potential to be (21.5%) and lowest in the USA. The medications currently used to treat DME yield sub-
“That’s a question we get a lot. OCT, patients cannot be treated success- the different method of action that optimal results. Some 40% of patients do not respond to
or optical coherence tomography, fully at the moment. We know that poor- or non-responders to anti-VEGF A third of people with diabetes do not know they have it, so anti-VEGF injections or have a less than ideal response. The
measures permeability, the amount of DME progresses from being driven treatment are waiting for. But first, a these numbers may be underreported. * treatment burden is quite heavy: injections must be given
fluids the retina lets through. As of now by permeability to being driven by clinical phase 2 study will have to be every month, for at least a while, making the treatment
it is the main biomarker to assess effi- inflammation. Anti-VEGF drugs work designed to assess efficacy. Overall, Symptoms of DME time-consuming and costly.
cacy of DME treatment. For anti-VEGF for patients in the permeability phase the phase 1 study's data clearly warrant DME can cause blurriness in the center of vision, appear-
drugs this is perfect. Anti-VEGF drugs but not for those in the inflammation further clinical research with multiple ance of dark spots or patches in the field of vision, dulling of New therapies are needed in order to successfully treat a
stop permeability, so this marker can phase. The problem is that we can’t injections of THR-149." colors, and difficulty in looking at bright light or glare. Objects larger number of patients.
measure how well they work. tell which phase a patient is in because might appear to change shape, size or color, or to disappear.
we have no biomarkers to test this. These symptoms can affect the ability to read, write, drive,
“VEGF-independent medicine possibly Because of this, all patients with DME and recognize faces.
has a different method of action, for get anti-VEGF injections in the eye
which there is no good biomarker at but about 40 percent respond poorly
the moment. The next best thing is or not at all. A different approach is
measuring vision improvement in the necessary to address these patients' 20% of people with diabetes were diagnosed with DED
patients. And even in this small trial need. For them a VEGF-independent (diabetic eye disease)*
of only 12 patients, after 1 injection pathway might be the solution.”
we found quick and maintained vision 7.6% have been diagnosed with DME*
improvement from day 1 to day 90.
This improvement was bigger than
could be expected through normal
variability, so that is very promising.”
Pravin Dugel, M.D. is a clinical pro-
fessor of ophthalmology and retinal
specialist and works with Oxurion
as an investigator and consultant. * Source: https: www.diabetesresearchclinicalpractice.com/article/S0168-8227(19)31257-4/fulltext#secst035)
https://www.diabetesresearchclinicalpractice.com/article/S0168-8227(19)31257-4/fulltext#secst030
Perspectives 2020 Perspectives 202022 THR-687 THR-687 23
THR-687
The second target Oxurion has identified to treat diabetic
macular edema and age-related macular degeneration,
is integrin. To inhibit this target, Oxurion is currently
clinically developing the molecule THR-687, a
compound with a broad potential.
What is THR-687? THR-687 has therefore the potential to attenuate different disease What is angiogenesis?
processes such as vascular leakage, inflammation, neovascu- Angiogenesis is a process by which new blood vessels are formed from
THR-687 is a small molecule pan RGD integrin antagonist. larization and fibrosis induced by multiple stress factors (including pre-existing vessels. Pathological angiogenesis is associated with retinal
Integrins play an important role in various biological processes but not limited to VEGF, cytokines,.….). diseases such as Diabetic Macular Edema, (Proliferative-) Diabetic Retinopathy
including cell differentiation, adhesion, migration, invasion and wet Age-related Macular Degeneration, leading to vision impairment or
and proliferation. Therefore, THR-687 has a broader biological effect than anti- even blindness.
VEGF therapy, the current golden standard of care for patients
Given their broad function, integrins are associated with various with DME or wet-AMD. What is retinal fibrosis?
eye diseases such as diabetic macular edema (DME) and wet
age-related macular degeneration (wet-AMD). Integrins are When retinal fibrosis occurs, abundant tissue (‘scar’) forms in the retina. It
implicated in main pathologic hallmarks like neovascularization, Whom is THR-687 for? is a reparative process in response to retinal injury, which can be caused
inflammation, fibrosis and vascular leakage. by inflammation. Excessive fibrotic scarring can lead to vision impairment or
There is a large unmet medical need in the DME market, since even blindness.
By targeting integrins, THR-687 could attenuate all these a significant proportion of these patients do not respond well to
vision-threatening processes given its broad therapeutic potential. current standard of care therapies.
Given the fact that THR-687 affects multiple signaling pathways
How does THR-687 work? simultaneously (including the VEGF pathway), it has the poten- In-licensed from Galapagos
tial for treating all patients, including naïve patients and patients
THR-687 antagonizes RGD-binding integrin receptors in the low sub-optimally responding to anti-VEGF therapy. THR-687 is derived from the small molecule integrin inhibitor library gen-
nanomolar range. The integrins are involved in the activation of erated and owned by Galapagos, for which Oxurion has an exclusive
multiple pathological pathways of DME and wet-AMD, by interact- Besides DME, THR-687 has also the potential to be very pro- worldwide license.
ing with multiple growth factors or cytokine receptors in a direct mising in wet AMD and other ocular indications.
or indirect manner.
Perspectives 2020 Perspectives 202024 THR-687 THR-687 25
Clinical update THR-687
A mean CST decrease of 106 µm
was reported at day 14 in the highest
THR-687, a potent pan-RGD integrin dose group.
This makes THR-687 a promising can-
THR-687-001: Mean Change in BCVA From Day 0 (Accounted for Rescue)
All Treated Subjects, Overall
antagonist in development for DME didate for treatment of vision-threatening
eye diseases such as DME and wet
AMD. Potentially, it could become a
20
15
9.2 8.8 8.3
7.7
standard of care for all DR patients, with 10 7.2
Oxurion kick-started 2020 with positive topline data from phase 1 clinical trial evaluating THR-687 or without DME, and wet AMD patients 5
0.0
3.1
for treatment of DME. This molecule showed a rapid onset of action across all doses after just one based on its broader biological effect than 0
D0 D1 D7 D14 M1 M2 M3
injection. Improvement was observed in vision, measured by best corrected visual acuity (BCVA), anti-VEGF therapy.
and macular swelling, measured by central subfield thickness (CST) on OCT (optical coherence
tomography). “The positive phase 1 data indicates that THR-687 could potentially be an effective
therapy for patients with DME”, notes Arshad Khanani, M.D., M.A. On track to start phase 2
clinical trial THR-687-001: Mean Change in CST From Day 1 (Accounted for Rescue)
All Treated Subjects, Overall
T H R- 6 87, a p a n - R GD i nte g r i n Each patient received one of three with 9.2 letters gained. Most impressively, “I am very encouraged to see excellent
antagonist, has a broad therapeutic doses: 0.4 mg (3 patients), 1 mg (3 this improvement was mostly maintained signs of efficacy so quickly post-treat- 120
potential. This compound can target patients) and 2.5 mg (6 patients). All post-single injection at month 3 with a ment. The study also demonstrated that 80
4.1
40
multiple disease hallmarks of diabetic 12 patients completed the study, with mean overall gain of 8.3 letters. THR-687 had a rapid positive effect on
0
retinopathy (DR), with and without no dose limiting toxicities or serious best corrected visual acuity (BCVA) that
-40
-3.8
DME, and also with wet age-related adverse effects reported. Overall, a marginal impact on mean CST was durable for up to 3 months after just -80 -43.9 -35.8
-38.9
macular degeneration (AMD). was noted up to month 1, followed by an a single injection. This promising initial data -120
D0 D1 D7 D14 M1 M2 M3
In each dose group, there was one increase until month 3. indicate that THR-687 could potentially be
Preclinical models show that THR-687 subject with a non-serious adverse an effective monotherapy for patients with
is a potent inhibitor of angiogenesis- event that was deemed to be related DME”, says Dr. Arshad Khanani.
induce d vascular leakag e. The probably to the injection procedure.
inhibition of integrins targets multiple Otherwise, there were a few other “THR-687 could Patrik De Haes, M.D., CEO of Oxurion, said:
THR-687-001: Mean Change in BCVA From Day 0 (Accounted for Rescue)
processes involved in pathological
angiogenesis and vascular leakage
side effects likely due to disease pro-
gression or concomitant diseases. In
potentially be an “These encouraging findings from our
phase 1 clinical trial with THR-687, along
All Treated Subjects, Overall
for common retina pathologies. An total, 9 adverse events were reported effective therapy for with the very positive feedback we have 20 THR-687 0.4mg (N=3) THR-687 1.0mg (N=3) THR-687 2.5mg (N=6)
integrin antagonist drug has the poten-
tial to block several pathways, and as
in 5 subjects. Three of them received
rescue therapy.
patients with DME.” received from the retinal community, high-
light the significant potential of this novel
15
12.5
10
such reduces the expression of growth Arshad Khanani, M.D., M.A. integrin antagonist. We are on track to start 6.7
factors as well. a phase 2 clinical study with THR-687 in 5
Immediate
1.7
treatment-naïve DME patients in H1 2021.” 0
D0 D1 D7 D14 M1 M2 M3
vision improvement Dose response
Set-up & safety
At day 1 an immediate improvement A pronounced dose response is observed
The phase 1 study evaluated safety in vision was found. The overall mean for this single injection of THR-687.
and tolerance of a single injection of change in BCVA was rather modest at Patients receiving the highest dose (2.5
THR-687 in 12 patients with DME with day 1 with 3.1 letters gained, but at day mg) noted an improvement in BCVA of
a history of response to prior anti- 7 it was already 7.2 letters. The highest 12.3/12.5 letters at month 3.
VEGF, corticosteroid treatment or laser. mean change was reported at month 1
Perspectives 2020 Perspectives 202026 THR-687 THR-687 27
Interview Arshad Khanani, M.D., M.A.
Diabetic retinopathy
“Initial data point to clinical What is DR?
Diabetic retinopathy (DR) is a consequence of too much
blood sugar, which damages and weakens blood vessels
Prevalence of DR
At present, an estimated 146 million people are affected by
DR and anyone with diabetes is at risk of developing it. Up
efficacy” in the retina. The vessels can swell, leak or clog, or new
vessels can start to grow. DR is a very common eye disease
for people with diabetes and can cause vision impairment
to one third of diabetes sufferers will eventually develop DR.
Symptoms of DR
or, at worst, vision loss. DR is the most common cause of Symptoms are blurriness, floating spots, dark areas of vision,
“The study was phase 1 and designed to assess safety. From evaluating a small number of preventable blindness in the world. and difficulty perceiving colors or contrast. These are typically
subjects, THR-687 showed a signal of clinical efficacy based on best corrected visual acuity," noted absent in the disease's early stages.
Arshad Khanani, M.D., M.A., an investigator in the study. There are two main stages in diabetic retinopathy: early
(non-proliferative diabetic retinopathy, NPDR); and more Treatment of DR
advanced (proliferative diabetic retinopathy, PDR). Current treatment of DR consists of laser treatment, surgery
Dr. Khanani presented the topline What is the future or anti-VEGF injections. These reduce the swelling of the
re s u lt s at t h e Fe b r u a r y 2 02 0 of THR-687? NDPR causes damaged blood vessels and blurred vision. retina and slow vision loss.
Angiogenesis, Exudation and When these vessels start to leak fluids this is called diabetic
Degeneration meeting in Miami, “Preclinical studies show that integrins macular edema (DME). The reverse is also a possibility: Why are new therapies needed?
Florida. “I was very pleased to present work on several pathways linked to blood vessels in the retina close off so blood cannot reach About 40% of patients do not respond to anti-VEGF injec-
the data of the phase 1 clinical trial of diabetic eye disease. As THR-687 is the retina. This is called macular ischemia. tions, or reaction is suboptimal. The treatment burden is also
THR-687. They were well received by a pan-RGD integrin antagonist, it can quite heavy: the injections must be given every month for
my colleagues,” he reported. block these pathways and improve PDR is the more advanced stage of DR and happens when at least a while. This makes treatment time-consuming and
vision. The result of the phase 1 clinical new blood vessels start to grow in response to ischemia. This costly. Lastly, anti-VEGF only suppresses symptoms and
trial argues in favor of a phase 2 study is called neovascularization. does not get at the cause.
Why were you pleased with in naive DME patients to establish
the data? the efficacy and safety of THR-687. In a survey, 79% of respondents said their vision impair- New therapies need to be developed to successfully treat a
These initial data show that THR-687 ment due to DR or DME made everyday activities - driving, larger number of patients.
“The primary endpoint of the study can potentially be an effective mono- working, cooking, cleaning their home - difficult and in some
was safety, and THR-687 was found therapy or adjunctive therapy for cases impossible.
to be safe without any dose-limiting patients with DME."
toxicities. The patients received prior * Source: DR Barometer: https://drbarometer.com
treatment with either anti-VEGF or
steroid injections and we still saw
visual acuity improvements. These
were relatively difficult patients to
treat. The high-dose group showed Dr. Arshad Khanani, M.D., M.A. is an
clinically meaningful gains in vision ophthalmologist and vitreo-retinal spe-
and improvements in OCT, which is cialist and the managing partner and
very encouraging.” director of clinical research at Sierra
Eye Associates, Reno, Nevada, USA.
He is a consultant to the company and
an investigator for both THR-149 and
THR-687 clinical trials.
Perspectives 2020 Perspectives 202028 Researching the AMD pathway Researching the AMD pathway 29
Researching the Interview Alan Stitt
AMD pathway “Oxurion is well-positioned to develop
pathways to address dry AMD”
The focus of the Oxurion research and preclinical
development teams was in 2019 almost In 2019, Oxurion made the strategic decision to also focus on research and development in dry
age-related macular degeneration (AMD): a major cause of vision loss worldwide, but one with no
exclusively on in-vivo and in-vitro validation effective treatment. This represents a huge challenge but Oxurion feels equal to the task. “The
of new pathways and compounds for number of patients affected by dry AMD continues to increase and there is considerable research
effort being channeled into better understanding the disease and, of course, developing an effective
treatment of dry AMD. We hope to be treatment”, explains Professor Alan Stitt, McCauley Chair of Experimental Ophthalmology at
able to present preclinical proof of Queen’s University in Belfast.
concepts in 2020.
Prof. Stitt: “Age-related macular Challenging & with dry AMD although we know this
degeneration occurs as two types, complex disease is influenced by factors like genetics,
often referred to as wet or dry AMD. gender, ethnicity and tobacco smoking.
The vision loss associated with wet An effective treatment for dry AMD There is much work under way to
AMD (also called neovascular AMD) could change many lives, but arriving understand the drivers for this disease,
can be addressed using regular intra- there means navigating a long and especially in the early stages”.
vitreal delivery of drugs called anti- challenging path.
VEGFs. There are efforts to improve “It would be ideal to intervene early
such treatments, such as with Oxurion's “Dry AMD is a very complex disease to when people still see reasonably
THR-687 molecule. Dry AMD (also tackle”, Prof. Stitt points out. “Scientists well. Stopping dry AMD in its tracks
called geographic atrophy) affects a are still trying to get a handle on what will prevent progression to the late,
much greater proportion of patients, causes the onset of dry AMD and we sight-threatening stages of the disease.
and unfortunately today their disease still cannot reliably predict who will Once cells in the retina have died it is
is largely untreatable.” develop the condition and how rapidly hard to replace them”, he adds.
it will progress. There can be distinct
“Dr y AMD is a slow, progressive differences between patients diagnosed
degeneration in the macular region
of the retina causing central vision
deficits. For sufferers this greatly “We want to develop a treatment that
diminishes their everyday life by
reducing the ability to do normal
addresses the underlying causes of dry
activities like reading, driving or even AMD so that progression of the disease
recognizing faces. In the worst-case
scenario it can become impossible to
is stopped or at least slowed down.”
live an independent life.” Professor Alan Stitt
Perspectives 2020 Perspectives 202030 Researching the AMD pathway Researching the AMD pathway 31
Tackling the cause of regularly for an injection into the The company's research teams are Age-related macular degeneration
the disease eye which, of course, is unpleasant working on preclinical models in dry
and inconvenient. The recurring AMD. “I believe the basic knowledge What is AMD?
Regarding treatment, Prof. Stitt is opti- costs are also a burden for health Oxurion has already developed with Age-related macular degeneration (AMD) is a progres- It is not known what causes AMD, although hereditary and
mistic. “Our goal for both wet and dry service providers”. THR-149 and THR-687 in DME and sive eye disease associated with aging. It is caused by environmental factors are thought to play a role. Smoking
AMD is to prevent the disease early wet AMD is very transferable to dry degeneration or deterioration of cells in the macula, the is a known risk factor: it doubles the chance of developing
when the retina's cells are not too While still committed to finding better AMD. Its combination of established region at the back of the eye responsible for central vision AMD. With wet AMD being female, over 60 and Caucasian
badly damaged. Many researchers treatments for DME and wet AMD, expertise in both retinal disease and and critical for activities like reading, writing and driving. AMD also increase the risk.
across the world are seeking to tackle Oxurion recently announced that it is therapeutic target development, plus is the leading cause of blindness is the United States.
the underlying causes of AMD so its exploring new pathways for dry AMD. translation into clinical results, posi- Symptoms of AMD
progression can be stopped or at Since he shares Oxurion’s interest tions Oxurion very well to do this”, Prof. There are two types of AMD: wet and dry. • Reduced central vision in one or both eyes
least slowed. While they have been in these important diseases and its Stitt concludes • Blurry or blind spots in the field of vision
extraordinarily successful, anti-VEGF commitment to improve patients' About 85 to 90 percent of people with AMD have dry AMD. • Decreased intensity or brightness of colors (‘grayness’)
treatment for diabetic macular edema lives, Prof. Stitt was happy to accept . This back of the eye disease occurs when the retina thins • Distortions of center frame of vision (straight lines
(DME) or wet AMD can prevent vision an official partial consultancy role in as part of the aging process. Dry AMD worsens over time, seem bent)
loss but they only suppress symp- the company. although much slower than the wet variety. Dry AMD usually
toms and don’t resolve the underlying affects both eyes. A common early sign of dry AMD is large and numerous
cause. Therefore, patients must return drusen: tiny yellow or white deposits under the retina.
Wet AMD is a more advanced stage of AMD and develops
when abnormal blood vessels grow into the macula and Treatment of AMD
start to leak fluids or blood, leading to formation of scar For dry AMD there is currently no treatment. Wet AMD is
tissue in the macula. The main consequence is rapid loss treated the same way as diabetic retinopathy and diabetic
Alan Stitt is an academic researcher at Queen’s of central vision. macular edema. A complicating factor is that most people
University in Belfast (Northern Ireland). He researches only know they have AMD when they experience vision loss,
diabetic retinopathy, age-related macular degeneration The disease has three stages: early, intermediate, and late. It by which time it is usually too late to effectively treat it. It is
and regenerative medicine, and is internationally recog- is only in late-stage AMD that vision loss becomes noticeable. also very hard to predict the disease's progression.
nized for his research in ophthalmology. In September
2019 he took a partial consultant position at Oxurion. Prevalence of AMD Going on a diet, exercising, wearing sunglasses, and quitting
His role is to advise on target preclinical development Worldwide, 170 million people suffer from AMD. Wet AMD smoking might help in halting the development or pro-
related to macular degeneration, diabetic edema and only affects 10 percent of AMD patients but is responsible gression of AMD.
retinal disease. for 90 percent of all AMD-related vision loss.
* Source: https://yoursightmatters.com/what-is-amd-and-what-are-the-symptoms/
Ambitious plan for dry AMD in 2020
“Oxurion aims at delivering at least one dry AMD pre- Patrik De Haes, M.D., CEO of Oxurion, says. “It must be
clinical proof of concept. In collaboration with top retina clear that as soon as we can craft a value path for those
experts such as Prof. Stitt, we are identifying the most patients, we expect this will automatically translate into
promising pathways for treating dry AMD. Our goal value creation for our shareholders as well”, Thomas Clay,
is to make a difference in the life of these patients”, Chairman of the Board, adds. .
Perspectives 2020 Perspectives 202032 Part of the global eye community Part of the global eye community 33
Part of the global Prevent blindness
eye community “By partnering with Oxurion we
reach more people”
Oxurion joins forces with NGOs and patient
advocacy organizations like Prevent Blindness For the second year in a row, the American non-profit organization Prevent Blindness has
partnered with Oxurion to encourage people to make their eye health a priority. “Partnerships with
and Retina Global to prevent vision loss and companies like Oxurion help us spread the important message to take care of yourself and your
fight blindness worldwide. eyes. And it’s working: each year we notice much more traffic to our website and more requests for
information on eye diseases related to diabetes,” notes Jeff Todd, president & CEO of
Prevent Blindness.
Prevent Blindness has been a patient “Through awareness and
advocate for healthy vision for over
a century. “Through awareness and
education, our goal is to minimize
education, our goal is to minimize the the damaging effects that
damaging effects that diabetes can
have on vision,” Jeff says.
diabetes can have on vision.”
Jeff Todd, President and CEO of Prevent Blindness
Diabetic retinopathy (DR), diabetic
macular edema (DME), and age-
related macular degeneration (AMD) “Our partnership with Oxurion during Empowering patients
are among the eye diseases Prevent Diabetes-Related Eye Disease Awareness
Blindness is working to prevent. Month in November allows us to spread To reach more people and educate them
Oxurion is seeking solutions for these the message, to promote on social media about serious eye diseases that can cause
eye diseases, so the collaboration is and lead more people to our website for significant vision loss and even blindness,
only natural. education,” Jeff concludes. Prevent Blindness will continue to work to
empower patients in 2020.
Jeff Todd: “We want to get patients
involved; we want to hear their voices.
Prevent Blindness has always been
about representing patients with
eye disease. Now we want to also
empower them and their caregivers to
advocate for themselves. By sharing
real life stories we're certain we can
reach more people and educate them
on eye diseases, how to prevent and
treat them.”
Perspectives 2020 Perspectives 2020Part of the global eye community 35
Retina global
Diabetes-related
eye disease
For people with diabetes, preventing
damage to their eyes starts with con-
“Our goal is to train the people on the
trolling blood sugar levels. That means
taking diabetes medication, eating
healthy, exercising, and getting eye
ground to obtain a sustainable outcome”
exams at least yearly. “We cannot stress
enough how important it is for those Retina Global sends retina specialists to parts of the world having a great need for expert eye care. “These
living with diabetes to see their eye specialists volunteer their time, effort and expertise to areas that lack retina specialists. They perform eye
doctor at least once a year. The earlier checks, provide treatment, or do surgery. On top of that they also train medical staff on the ground so they
we can detect these eye diseases, the can work independently”, explains Rajat Agrawal, M.D., CEO of Retina Global. “It is always our goal to have
more successful treatment can be,” a sustainable outcome.”
Jeff emphasizes.
But of course, before they can see Retina Global currently operates in
an eye doctor to check for diabetes- Belize, the Bahamas, Burundi and Bolivia. “Everywhere we go we see
related eye disease people must know
they have diabetes. This isn’t always
Projects in Haiti, Mexico and Ethiopia will
start soon. “In these underserved areas
people with DR. Treatment is
the case. They may not recognize there is a great need to treat diabetic often not available for them.”
the symptoms or realize that their retinopathy (DR). Everywhere we go we
problems are serious. Making people see people with DR, and often treatment Rajat Agrawal, M.D.,
aware of diabetes symptoms is also an is unaffordable or there simply isn’t a retina CEO of Retina Global
important goal. specialist available. Doctors in these coun-
tries don’t always have the knowledge or
the tools to diagnose these eye diseases.” “We are currently focusing our efforts in Public Awareness
Bolivia on the city of Cochabamba, the
country's fourth largest city with some Not only doctors and nurses need
The BOLDR project 650,000 inhabitants. There were no information on these diseases: patients
Frequent Abnormally
hungry
trained retina specialists in public health and patient groups do as well. “Raising
urination
Abnormally Retina Global plans to change that. With care. Most patients with diabetes go to public awareness of diabetic eye disease
thirsty the support of Oxurion it started the a primary care physician, but these don't is another of our goals. People need to
Experiencing BOLDR (Bolivian Diabetic Retinopathy) always know they need to also screen for know they need regular eye screening
blurred vision Tingling feet
or hearing loss and itchy skin
project. BOLDR is committed to reducing eye diseases as. Courses and lectures for and how to recognize the symptoms. By
the number of people going blind due medical staff supported by Oxurion will providing information to patient groups
to the increasing prevalence of diabetic change that”, Rajat Agrawal explains. and running campaigns, we are confi-
retinopathy in Bolivia, and to combating dent we can make people aware of DR
the rising incidence of age-related and get them to turn to their doctors and
macular degeneration (AMD) in this South seek treatment. Of course, we also need
American country. to train doctors and medical staff so they
can do the necessary eye checks and
give treatments.”
Perspectives 202036 Oncurious Oncurious 37
Oncurious Next-generation I/O cancer
treatments
Oncurious is preparing a preclinical proof of concept from its portfolio of immuno-oncology assets
acquired from VIB, the Flemish Institute of Biotechnology. The ambition is to develop next-
generation immunotherapies targeting a very broad spectrum of cancers.
Im m u n o - o n co lo g y i s a ra p i d ly combined expertise pursues a single tumor that is the most common
evolving branch of science. It’s a goal: to develop innovative therapies malignant brain tumor in children.
specific type of immunotherapy that targeting a very broad spectrum of TB-403, an anti-PIGF, blocks the
stimulates the immune system to cancers, thus benefiting a large group PIGF signals and stops the tumor
fight cancer cells and tumors. It could of patients. cell s f ro m g row in g . Pre clinic al
offer an alternative or complementary studies suggest this anti-PIGF leads
treatment to chemotherapy, radiation, Research groups are working on to tumor regression, decreased
and surgical removal of tumors. a preclinical proof of concept for metastasis, and higher survival rates.
a portfolio of innovative immuno- At the same time, anti-PIGF therapy
Unfortunately, the first generation of oncology assets that the company will probably be much less toxic
immunotherapies brought to market plans to present in I/O in 2020. than chemotherapy.
only helps a minority of patients.
Many do not respond to them, and Oncurious is still recruiting patients
for multiple types of cancer they are Clinical trial with relapsed or refractory medullo-
not effective at all. Research on new researching treatment blastoma to conduct a phase 1/
ones is thus urgently needed. of medulloblastoma phase 2a clinical trial to evaluate the
safety of different doses of TB-403.
Oncurious joined forces with VIB two One project Oncurious is to develop
years ago to take immuno-oncology an alternative treatment for medullo-
research to the next level. Their blastoma, a rare life-threatening
Perspectives 2020 Perspectives 2020You can also read
