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
-----------------------------------------------------------------------------------------------------------------------
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:
https://www.discmedicine.com/ and the full text of the announcement
from the company is contained below.
[S]
Enquiries
For more information on Arix, please contact:
Arix Bioscience plc
+44 (0)20 7290 1050
ir@arixbioscience.com
Powerscourt Group
Sarah MacLeod, Ibrahim Khalil, Nick Johnson
+44 (0)20 7250 1446
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.
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 inEPP, 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 ofwhole-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 demonstratedsubstantial
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 oftreatment, increasing from 4.5 minutes at baseline to over
6 hours; the participant did not report a prodromeduring any
sunlight challenge after Day 20 ? A participant on 60 mg bitopertin
reported a >200-fold increase in sunlight tolerance on day 74
oftreatment, increasing from 1.25 minutes at baseline to over 4
hours, and did not report a prodrome during anysunlight 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 perday) to 1,200 minutes at Week 24 ? Time to prodrome
during sunlight challenge (averaged over a two-week period):
increased >7-fold, from25 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
treatmentcompared 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 visitreported 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 43visit 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 patientsreported an improvement in the
impact of EPP on quality of life and 4/8 reported no change in the
impact of EPPon quality of life. For patients whose most recent
data was after Day 43, 2/2 reported marked improvement inthe 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 reporteddiscontinuations or
dose reductions, no reported adverse events greater than Grade 1,
and no meaningful changesobserved 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 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
(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.
-----------------------------------------------------------------------------------------------------------------------
Dissemination of a Regulatory Announcement, transmitted by EQS
Group. The issuer is solely responsible for the content of this
announcement.
-----------------------------------------------------------------------------------------------------------------------
ISIN: GB00BD045071
Category Code: PFU
TIDM: ARIX
LEI Code: 213800OVT3AHQCXNIX43
OAM Categories: 3.1. Additional regulated information required to be disclosed under the laws of a Member State
Sequence No.: 249984
EQS News ID: 1653863
End of Announcement EQS News Service
=------------------------------------------------------------------------------------
Image link:
https://eqs-cockpit.com/cgi-bin/fncls.ssp?fn=show_t_gif&application_id=1653863&application_name=news
(END) Dow Jones Newswires
June 09, 2023 09:53 ET (13:53 GMT)
Arix Bioscience (LSE:ARIX)
Historical Stock Chart
From Dec 2024 to Jan 2025
Arix Bioscience (LSE:ARIX)
Historical Stock Chart
From Jan 2024 to Jan 2025
