- AURORA study designed to evaluate bitopertin as a potential
disease-modifying treatment for adults with EPP in the United States
- AURORA study designed to assess changes in protoporphyrin IX
levels, safety, tolerability, photosensitivity and other measures
in a double-blind, placebo-controlled setting; top-line data
expected in 2023
WATERTOWN, Mass., Oct. 31,
2022 /PRNewswire/ -- Disc Medicine, a clinical-stage
biopharmaceutical company focused on the discovery, development and
commercialization of novel treatments for patients suffering from
serious hematologic diseases, announced today the initiation of
AURORA, a Phase 2 clinical study of bitopertin in adults with EPP.
Bitopertin is an oral, selective inhibitor of glycine transporter 1
(GlyT1) designed to modulate heme biosynthesis, and has been shown
in preclinical studies to reduce accumulation of protoporphyrin IX
(PPIX), the toxic metabolite that causes disease pathology in EPP
patients. Bitopertin is also currently being studied in BEACON, an
open-label Phase 2 clinical study in patients with EPP and X-linked
protoporphyria (XLP), which was announced in August 2022 and is being conducted in
Australia.
"We are delighted to initiate AURORA, the first US-based study
of bitopertin in patients with EPP. We are conducting the AURORA
study to enable us to evaluate the effects of bitopertin on PPIX
levels, photosensitivity, pain and other key measures in a
rigorous, blinded trial," said John
Quisel, JD, PhD, Chief Executive Officer at Disc Medicine.
"We have designed AURORA and BEACON to provide us with a robust
assessment of bitopertin's potential as a disease-modifying therapy
for EPP."
The AURORA Phase 2 study is a randomized, double-blind,
placebo-controlled, parallel dosing clinical trial designed to
evaluate the safety, tolerability, and efficacy of bitopertin in
adults with EPP. It is expected to enroll approximately 75 patients
at sites throughout the United
States. Patients will receive orally-administered bitopertin
for 120 days at doses of either 20 mg once-daily or 60 mg
once-daily. Upon completion of the 120-day treatment period,
patients may roll over to the open-label extension portion of the
trial. The study is designed to measure changes in levels of
metal-free PPIX, as well as measures of photosensitivity, daylight
tolerance, pain, safety, and tolerability.
"There is a significant unmet need for disease-modifying
therapies for EPP that address the underlying pathophysiology of
the condition. Patients with EPP experience debilitating painful
reactions, as well as potentially severe hepatobiliary effects, and
the measures they must take to avoid the sun can have a strong,
negative impact on their quality of life," said Cynthia Levy, MD, FAASLD, AGAF, University of Miami Miller School of Medicine. "We
are very excited about the potential for bitopertin to address the
underlying protoporphyrin accumulation that causes EPP symptoms and
hepatopathy, and we look forward to the results."
About Bitopertin
Bitopertin is a clinical-stage, orally administered inhibitor of
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. The safety profile and effects of
bitopertin on heme biosynthesis were previously established in a
comprehensive clinical program comprising over 4,000 individuals
across multiple clinical studies. Disc Medicine is planning to
develop bitopertin as a potential treatment for a range of
hematologic diseases beginning with EPP and XLP. In preclinical
models of EPP and XLP, bitopertin was shown to significantly
decrease PPIX, a toxic intermediate of heme biosynthesis which is
the underlying cause of the disease.
Bitopertin is an experimental 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)
EPP and 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 that
is dedicated to transforming the lives of patients with hematologic
disorders. We are building a portfolio of innovative, potentially
first-in-class therapeutic candidates that affect fundamental
pathways of red blood cell biology. We are committed to developing
treatments that empower and bring hope to the many patients who
suffer from hematologic diseases. In August
2022, Disc announced it entered into a definitive merger
agreement with Gemini Therapeutics, Inc. (NASDAQ:GMTX). For more
information, please visit www.discmedicine.com.
Disc Medicine Cautionary Statement Regarding Forward-Looking
Statements
Certain statements in this press release may constitute
"forward-looking statements" for purposes of the federal securities
laws concerning Disc's expectations with respect to its AURORA
clinical study and other matters, including the proposed
transaction between Disc and Gemini Therapeutics, Inc. (Gemini)
announced in August 2022. These
forward-looking statements include express or implied statements
relating to Disc's management team's expectations, hopes, beliefs,
intentions or strategies regarding the future. In addition, any
statements that refer to projections, forecasts or other
characterizations of future events or circumstances, including any
underlying assumptions, are forward-looking statements. The words
"anticipate," "believe," "contemplate," "continue," "could,"
"estimate," "expect," "intends," "may," "might," "plan,"
"possible," "potential," "predict," "project," "should," "will,"
"would" and similar expressions may identify forward-looking
statements, but the absence of these words does not mean that a
statement is not forward-looking. These forward-looking statements
are based on current expectations and beliefs concerning future
developments and their potential effects. There can be no assurance
that future developments affecting Disc, Gemini or the proposed
transaction will be those that have been anticipated. These
forward-looking statements involve a number of risks, uncertainties
(some of which are beyond Disc's control) or other assumptions that
may cause actual results or performance to be materially different
from those expressed or implied by these forward-looking
statements. These risks and uncertainties include, but are not
limited to, the risk that the conditions to the closing of the
transaction are not satisfied, including the failure to obtain
stockholder approval for the transaction; the risk that the
concurrent financing is not completed in a timely manner or at all;
uncertainties as to the timing of the consummation of the
transaction and the ability of each of Gemini and Disc to
consummate the transaction, including the concurrent financing;
risks related to Gemini's continued listing on the Nasdaq Stock
Market until closing of the proposed transaction; risks related to
Gemini's and Disc's ability to correctly estimate their respective
operating expenses and expenses associated with the transaction, as
well as uncertainties regarding the impact any delay in the closing
would have on the anticipated cash resources of the combined
company upon closing and other events and unanticipated spending
and costs that could reduce the combined company's cash resources;
the occurrence of any event, change or other circumstance or
condition that could give rise to the termination of the merger
agreement; the effect of the announcement or pendency of the merger
on Gemini's or Disc's business relationships, operating results and
business generally; costs related to the merger; the outcome of any
legal proceedings that may be instituted against Gemini, Disc or
any of their respective directors or officers related to the merger
agreement or the transactions contemplated thereby; the ability of
Gemini or Disc to protect their respective intellectual property
rights; competitive responses to the transaction; unexpected costs,
charges or expenses resulting from the transaction; potential
adverse reactions or changes to business relationships resulting
from the announcement or completion of the transaction; and
legislative, regulatory, political and economic developments.
The foregoing list of factors is not exhaustive. You should
carefully consider the foregoing factors and the other risks and
uncertainties described in the "Risk Factors" section of the proxy
statement/prospectus included in the registration statement on Form
S-4 which was initially filed with the SEC in September 2022 in connection with the transaction
and other documents filed by Gemini from time to time with the SEC.
Should one or more of these risks or uncertainties materialize, or
should any of Disc's assumptions prove incorrect, actual results
may vary in material respects from those projected in these
forward-looking statements. Some of these risks and uncertainties
may in the future be amplified by the ongoing COVID-19 pandemic and
there may be additional risks that we consider immaterial or which
are unknown. It is not possible to predict or identify all such
risks. Disc's forward-looking statements only speak as of the date
they are made, and Gemini and Disc do not undertake any obligation
to update or revise any forward-looking statements, whether as a
result of new information, future events or otherwise, except as
may be required under applicable securities laws.
No Offer or Solicitation
This press release is not intended to and does not constitute an
offer to sell or the solicitation of an offer to subscribe for or
buy or an invitation to purchase or subscribe for any securities or
the solicitation of any vote in any jurisdiction pursuant to the
proposed transaction or otherwise, nor shall there be any sale,
issuance or transfer of securities in any jurisdiction in
contravention of applicable law. No offer of securities shall be
made except by means of a prospectus meeting the requirements of
the Securities Act. Subject to certain exceptions to be approved by
the relevant regulators or certain facts to be ascertained, the
public offer will not be made directly or indirectly, in or into
any jurisdiction where to do so would constitute a violation of the
laws of such jurisdiction, or by use of the mails or by any means
or instrumentality (including without limitation, facsimile
transmission, telephone and the internet) of interstate or foreign
commerce, or any facility of a national securities exchange, of any
such jurisdiction.
Important Additional Information Will be Filed with the
SEC
In connection with the proposed transaction between Gemini and
Disc, Gemini intends to file relevant materials with the SEC,
including a registration statement on Form S-4 that contains a
proxy statement/prospectus of Gemini and information statement of
Disc, which was initially filed on September
2, 2022. DISC URGES INVESTORS AND STOCKHOLDERS TO READ THESE
MATERIALS CAREFULLY AND IN THEIR ENTIRETY WHEN THEY BECOME
AVAILABLE BECAUSE THEY WILL CONTAIN IMPORTANT INFORMATION ABOUT
GEMINI, DISC, THE PROPOSED TRANSACTION AND RELATED MATTERS.
Investors and shareholders will be able to obtain free copies of
the proxy statement/prospectus/information statement and other
documents filed by Gemini with the SEC (when they become available)
through the website maintained by the SEC at www.sec.gov. In
addition, investors and shareholders should note that Gemini
communicates with investors and the public using its website
(www.geminitherapeutics.com), the investor relations website
(https://investors.geminitherapeutics.com/) where anyone will be
able to obtain free copies of the proxy
statement/prospectus/information statement and other documents
filed by Gemini with the SEC and stockholders are urged to read the
proxy statement/prospectus/information statement and the other
relevant materials when they become available before making any
voting or investment decision with respect to the proposed
transaction.
Participants in the Solicitation
Gemini and its directors and executive officers may be deemed to
be participants in the solicitation of proxies in connection with
the proposed transaction. Information about Gemini's directors and
executive officers is included in Gemini's most recent Annual
Report on Form 10-K, including any information incorporated therein
by reference, as filed with the SEC, and the registration statement
on Form S-4 initially filed with the SEC on September 2, 2022, and any amendments thereto as
filed with the SEC. These documents can be obtained free of charge
from the sources indicated above.
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