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 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 BitopertinBitopertin 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 MedicineDisc 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.
Disc Medicine Cautionary Statement Regarding
Forward-Looking Statements
This press release contains “forward-looking statements” within
the meaning of the Private Securities Litigation Reform Act of
1995, including, but not limited to, express or implied statements
regarding Disc’s expectations with respect to its BEACON Phase 2
clinical trial of bitopertin and projected timelines for the
initiation and completion of its clinical trials, the timing of
additional data and other activities. The use of words such as, but
not limited to, “aim,” “believe,” “expect,” “estimate,” “project,”
“intend,” “future,” “potential,” “continue,” “may,” “might,”
“plan,” “will,” “should,” “seek,” “anticipate,” or “could” or the
negative of these terms and other similar words or expressions that
are intended to identify forward-looking statements.
Forward-looking statements are neither historical facts nor
assurances of future performance. Instead, they are based on Disc’s
current beliefs, expectations and assumptions regarding the future
of Disc’s business, future plans and strategies, clinical data and
other future conditions. New risks and uncertainties may emerge
from time to time, and it is not possible to predict all risks and
uncertainties. No representations or warranties (expressed or
implied) are made about the accuracy of any such forward-looking
statements.
Disc may not actually achieve the plans, intentions or
expectations disclosed in these forward-looking statements, and
investors should not place undue reliance on these forward-looking
statements. Actual data or events could differ materially from the
plans, intentions and expectations disclosed in the forward-looking
statements as a result of a number of material risks and
uncertainties including but not limited to: the adequacy of Disc’s
capital to support its future operations and its ability to
successfully initiate and complete clinical trials; the nature,
strategy and focus of Disc; the difficulty in predicting the time
and cost of development of Disc’s product candidates; Disc’s plans
to research, develop and commercialize its current and future
product candidates; the timing of initiation of Disc’s planned
preclinical studies and clinical trials; the timing of the
availability of data from Disc’s clinical trials; Disc’s ability to
identify additional product candidates with significant commercial
potential and to expand its pipeline in hematological diseases; the
timing and anticipated data of Disc’s preclinical studies and
clinical trials and the risk that the data of Disc’s preclinical
studies and clinical trials may not be predictive of future data in
connection with future studies or clinical trials and may not
support further development and marketing approval; the other risks
and uncertainties described in the “Risk Factors” section of our
Quarterly Report on Form 10-Q filed with the Securities
and Exchange Commission (SEC) on May 15, 2023 and other documents
filed by Disc from time to time with the SEC, as well as
discussions of potential risks, uncertainties, and other important
factors in Disc’s subsequent filings with the SEC. Any
forward-looking statement speaks only as of the date on which it
was made. None of Disc, nor its affiliates, advisors or
representatives, undertake any obligation to publicly update or
revise any forward-looking statement, whether as result of new
information, future events or otherwise, except as required by
law.
Media Contact
Peg RusconiVerge Scientific
Communicationsprusconi@vergescientific.com
Investor Relations Contact
Christina Tartaglia Stern Investor
Relationschristina.tartaglia@sternir.com
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