REG-Arix Bioscience PLC Portfolio company Disc Medicine presents positive initial data from Phase 2 BEACON trial at EHA
09/06/2023 15:00
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Arix Bioscience PLC (ARIX)
Portfolio company Disc Medicine presents positive initial data from Phase
2 BEACON trial at EHA
09-Jun-2023 / 14:53 GMT/BST
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Arix Bioscience plc
Portfolio company Disc Medicine presents positive initial data from Phase
2 BEACON trial at EHA
LONDON, 09 June 2023: Arix Bioscience plc (“Arix” or the “Company”) (LSE:
ARIX), a transatlantic venture capital company focused on investing in
breakthrough biotechnology companies, notes that its portfolio company,
Disc Medicine, today announced preliminary findings from its Phase 2
open-label BEACON trial evaluating bitopertin, an orally administered
glycine transporter 1 (GlyT1) inhibitor, in patients with erythropoietic
protoporphyria (EPP) and X-linked protoporphyria (XLP) at the European
Haematology Association (EHA) 2023 Congress in Frankfurt, Germany.
The initial trial data from the BEACON study demonstrated consistent
decreases in protoporphyrin IX (the disease-causing metabolite in EPP),
significant increases in reported sunlight tolerance and improvements in
measures of patient quality of life.
The BEACON trial is a randomised, open-label, parallel-arm trial enrolling
up to 22 patients with EPP or XLP at trial sites in Australia. This trial
was designed to assess changes in levels of PPIX, as well as measures of
photosensitivity, quality of life, and safety and tolerability.
Robert Lyne, CEO of Arix Bioscience, said: “We are very encouraged by the
positive data from the BEACON trial. This is an important clinical
milestone for the company and a major development for patients who suffer
from erythropoietic protoporphyria. We look forward to supporting Disc
Medicine as they continue to advance the BEACON study through clinical
development. We are also thrilled that Disc are achieving these important
clinical milestones less than two years after our initial investment,
demonstrating a success of our strategy in action.”
The announcement can be accessed on Disc Medicine’s website at:
1 https://www.discmedicine.com/ and the full text of the announcement
from the company is contained below.
ENDS
Enquiries
For more information on Arix, please contact:
Arix Bioscience plc
+44 (0)20 7290 1050
ir 2 @arixbioscience.com
Powerscourt Group
Sarah MacLeod, Ibrahim Khalil, Nick Johnson
+44 (0)20 7250 1446
3 arix@powerscourt-group.com
About Arix Bioscience plc
Arix Bioscience plc is a global venture capital company focused on
investing in breakthrough biotechnology companies around cutting-edge
advances in life sciences.
We collaborate with exceptional entrepreneurs and provide the capital,
expertise, and global networks to help accelerate their ideas into
important new treatments for patients. As a listed company, we are able to
bring this exciting growth phase of our industry to a broader range of
investors. 4 www.arixbioscience.com
Disc Medicine Press Release:
Disc Presents Positive Initial Data from Phase 2 BEACON Trial of
Bitopertin in Patients with Erythropoietic Protoporphyria (EPP) at
European Hematology Association (EHA) 2023 Congress
Jun 09, 2023
• Consistent and dose-dependent reductions of protoporphyrin IX (PPIX),
the disease-causing metabolite in EPP, were observed in patients
treated with bitopertin
• Patients reported significant improvements in sunlight tolerance and
measures of quality-of-life
• Bitopertin was well-tolerated, with no meaningful changes in
hemoglobin observed
• Disc Medicine to host an investor conference call today at 7:30 AM ET
WATERTOWN, Mass., June 09, 2023 (GLOBE NEWSWIRE) -- Disc Medicine, Inc.
(NASDAQ:IRON), a clinical-stage biopharmaceutical company focused on the
discovery, development, and commercialization of novel treatments for
patients suffering from serious hematologic diseases, today presented
preliminary findings from its ongoing, Phase 2 open-label BEACON trial
evaluating bitopertin, an orally administered glycine transporter 1
(GlyT1) inhibitor, in patients with erythropoietic protoporphyria (EPP)
and X-linked protoporphyria (XLP) at the European Hematology Association
(EHA) 2023 Congress in Frankfurt, Germany. The initial trial data
demonstrated consistent decreases in PPIX, significant increases in
reported sunlight tolerance and improvements in measures of patient
quality of life.
“We’re delighted to share these initial, positive data from BEACON, which
provide the first clinical evidence supporting our therapeutic hypothesis
of bitopertin in EPP. Over the next 12 months, we plan to build on this
momentum with a series of additional clinical read-outs across our
portfolio,” said John Quisel, J.D., Ph.D., Chief Executive Officer and
President of Disc Medicine. “This is an important moment for Disc as a
company, and I want to extend my gratitude to our team, collaborators, and
most importantly, the patients and families participating in BEACON.”
“We are excited to share these initial data from the BEACON trial, where
we observed consistent and sustained suppression of PPIX, the
disease-causing metabolite in EPP, in patients treated with bitopertin,”
said Will Savage, M.D., Ph.D., Chief Medical Officer at Disc Medicine.
“Importantly, this reduction translated into significant improvements in
the time that patients can spend in sunlight without reporting pain or
symptoms related to their disease. We’re encouraged by the data and plan
to present additional data at the end of the year.”
The BEACON trial is a randomized, open-label, parallel-arm trial enrolling
up to 22 patients with EPP or XLP at trial sites in Australia. This trial
was designed to assess changes in levels of PPIX, as well as measures of
photosensitivity, quality of life, and safety and tolerability. Subjects
are randomized to receive either 20 mg or 60 mg of bitopertin once-daily
for 24 weeks, after which patients have the option of continuing in an
open-label extension of the trial for up to an additional 24 weeks. The
trial is ongoing and these data reflect initial data from 15 subjects
enrolled as of the data cutoff of May 8, 2023, with a range of treatment
durations from 18 days to 6 months. Due to batch processing of samples,
the data cutoff for PPIX data was April 7, 2023.
Highlights of the initial data presented:
• Protoporphyrin IX (PPIX) levels: Significant, consistent,
dose-dependent, and sustained reductions of whole-blood, metal-free
PPIX; mean reduction of >40% when compared to baseline
• Measures of light tolerance (individual) from two participants with
the longest follow-up demonstrated substantial increases in sunlight
tolerance as measured by time in sunlight without experiencing a
prodrome (initial symptoms that signal a pain attack), or “sunlight
challenge”:
◦ A participant on 20 mg bitopertin reported a >80-fold increase in
sunlight tolerance on day 88 of treatment, increasing from 4.5
minutes at baseline to over 6 hours; the participant did not
report a prodrome during any sunlight challenge after Day 20
◦ A participant on 60 mg bitopertin reported a >200-fold increase
in sunlight tolerance on day 74 of treatment, increasing from
1.25 minutes at baseline to over 4 hours, and did not report a
prodrome during any sunlight challenge after Day 120
• Measures of light tolerance (aggregated across participants from whom
data was available in the trial):
◦ Average weekly total time spent in sunlight: increased from 344
minutes (approximately 49 minutes per day) to 1,200 minutes at
Week 24
◦ Time to prodrome during sunlight challenge (averaged over a
two-week period): increased >7-fold, from 25 minutes at baseline
to 182 minutes at Week 24
◦ Increased proportion of days without symptoms: 75% vs. 25%
(baseline)
◦ Increased proportion of sunlight challenges without prodromes:
50% vs. 0% (baseline)
◦ Phototoxic reactions: 96% reduction in patient-reported
phototoxic reactions while on treatment compared to baseline
(n=15)
• Measures of patient quality of life
◦ Patient Global Impression of Change (PGIC): All 10 patients that
had completed a day 43 visit reported their disease was much
better (n=8) or a little better (n=2) in the last 7 days
◦ Patient Global Impression of Severity (PGIS): Nine out of 10
patients that had completed a day 43 visit reported their EPP was
mild (n=3) or not at all severe (n=6)
◦ EPP Impact Questionnaire (EPIQ): For patients whose most recent
data was Day 43, 4/8 patients reported an improvement in the
impact of EPP on quality of life and 4/8 reported no change in
the impact of EPP on quality of life. For patients whose most
recent data was after Day 43, 2/2 reported marked improvement in
the impact of EPP on quality of life, reporting no impact of EPP
on quality of life.
• Bitopertin was well-tolerated at both dose levels with no reported
serious adverse events, no reported discontinuations or dose
reductions, no reported adverse events greater than Grade 1, and no
meaningful changes observed in mean hemoglobin levels
These data were presented at the European Hematology Association 2023
Congress in Frankfurt, Germany and the poster is available on the EHA
Congress platform at 5 www.ehaweb.org.
Management will host a call to review the presented data on Friday, June
9th at 7:30 am ET. Please register for the event on the Events and
Presentations page of Disc’s website ( 6 https://ir.discmedicine.com/).
About Bitopertin
Bitopertin is an investigational, clinical-stage, orally-administered
inhibitor of glycine transporter 1 (GlyT1) that is designed to modulate
heme biosynthesis. GlyT1 is a membrane transporter expressed on developing
red blood cells and is required to supply sufficient glycine for heme
biosynthesis and support erythropoiesis. Disc is planning to develop
bitopertin as a potential treatment for a range of hematologic diseases
including erythropoietic porphyrias, where it has potential to be the
first disease-modifying therapy. There are currently two ongoing Phase 2
clinical trials of bitopertin in patients with erythropoietic porphyria,
including an open-label trial called BEACON and a randomized, double-blind
placebo-controlled trial called AURORA.
Bitopertin is an investigational agent and is not approved for use as a
therapy in any jurisdiction worldwide. Disc obtained global rights to
bitopertin under a license agreement from Roche in May 2021.
About Erythropoietic Protoporphyria (EPP) and X-linked Protoporphyria
(XLP)
Erythropoietic protoporphyria (EPP) and X-linked Protoporphyria (XLP) are
rare, debilitating and potentially life-threatening diseases caused by
mutations that affect heme biosynthesis, resulting in the accumulation of
a toxic, photoactive intermediate called protoporphyrin IX (PPIX). This
causes severe reactions when patients are exposed to sunlight,
characterized by excruciating pain, edema, burning sensations and
potential blistering and disfigurement. PPIX also accumulates in the
hepatobiliary system and can result in complications including gallstones,
cholestasis, and liver damage in 20-30% of patients and in extreme cases
liver failure. Current standard of care involves extreme measures to avoid
sunlight, including restricting outdoor activities to nighttime, use of
protective clothing and opaque shields, and pain management. This has a
significant impact on the psychosocial development, quality of life, and
daily activities of patients, particularly in young children and families.
There is currently no cure for EPP and only one FDA-approved therapy, a
surgically implanted synthetic hormone designed to stimulate melanin
production called Scenesse® (afamelanotide).
About Disc Medicine
Disc Medicine is a clinical-stage biopharmaceutical company committed to
discovering, developing, and commercializing novel treatments for patients
who suffer from serious hematologic diseases. We are building a portfolio
of innovative, potentially first-in-class therapeutic candidates that aim
to address a wide spectrum of hematologic diseases by targeting
fundamental biological pathways of red blood cell biology, specifically
heme biosynthesis and iron homeostasis. For more information, please visit
www.discmedicine.com.
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══════════════════════════════════════════════════════════════════════════
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TIDM: ARIX
LEI Code: 213800OVT3AHQCXNIX43
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