WATERTOWN, Mass., Dec. 27,
2022 /PRNewswire/ -- Disc Medicine, Inc. ("Disc"), a
clinical-stage biopharmaceutical company focused on the discovery,
development, and commercialization of novel treatments for patients
suffering from serious hematologic diseases, announced today that
the U.S. Food and Drug Administration ("FDA") granted Orphan Drug
Designation to bitopertin for the treatment of erythropoietic
protoporphyria ("EPP"). Bitopertin is an investigational 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. It
is currently being studied in two ongoing Phase 2 studies in EPP,
AURORA (NCT05308472) and BEACON (ACTRN12622000799752).
"Receiving orphan drug designation for bitopertin is incredibly
encouraging and validates our commitment to bring a potential new
treatment to EPP patients," said John
Quisel, J.D., Ph.D., Chief Executive Officer and President
of Disc. "We are eagerly awaiting the results of our ongoing Phase
2 trials and look forward to collaborating with the FDA to progress
bitopertin through clinical development."
FDA Orphan Drug Designation may be granted to investigational
drugs or biological products which show promise in treating rare
medical diseases or conditions that affect fewer than 200,000
people in the United States. By
receiving Orphan Drug Designation, bitopertin can benefit from
certain development incentives and seven years of market
exclusivity, subject to regulatory approval.
About EPP
EPP is a rare, debilitating and potentially life-threatening
diseases caused by mutations that affect heme biosynthesis,
resulting in the accumulation of a toxic, photoactive intermediate,
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 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 is planning to develop
bitopertin as a potential treatment for a range of hematologic
diseases beginning with EPP and X-linked protoporphyria
("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 Disc Medicine, Inc.
Disc 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, potential
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) ("Gemini").
For more information, please visit www.discmedicine.com.
Disc 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 the proposed transaction between Disc and
Gemini including whether and when the proposed transaction
will be consummated; statements about the structure, timing and
completion of the proposed transaction; and other matters,
including Disc's expectations with respect to its AURORA and BEACON
clinical trials and Phase 1b/2a
clinical study of DISC-0974 in myelofibrosis and anemia, its plans
to initiate a Phase 2 study of DISC-0974 in chronic kidney disease,
and other statements that are not historical in nature. 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 (the "Initial Registration Statement"), which was initially
filed on September 2, 2022, as
amended by Amendment No. 1 to the Initial Registration Statement
filed with the SEC on October 7,
2022, Amendment No. 2 to the Initial Registration Statement
filed with the SEC on November 3,
2022, Amendment No. 3 to the Initial Registration Statement
filed with the SEC on November 23,
2022 and Amendment No. 4 to the Initial Registration
Statement filed with the SEC on December 1,
2022 (together with the Initial Registration Statement, the
"Registration Statement") and declared effective on December 2, 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
In connection with the proposed transaction between Gemini and
Disc, Gemini filed with the SEC a registration statement on Form
S-4, as amended, containing a definitive proxy statement/prospectus
of Gemini. The registration statement was declared effective by the
SEC on December 2, 2022, and the
special meeting of Gemini stockholders is scheduled to be held on
December 28, 2022. GEMINI URGES
INVESTORS AND STOCKHOLDERS TO READ THESE MATERIALS CAREFULLY AND IN
THEIR ENTIRETY BECAUSE THEY CONTAIN IMPORTANT INFORMATION ABOUT
GEMINI, DISC, THE PROPOSED TRANSACTION AND RELATED MATTERS.
Investors and shareholders are able to obtain free copies of the
definitive proxy statement/prospectus and other documents filed by
Gemini with the SEC 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) and the investor relations
website (https://investors.geminitherapeutics.com/) where anyone is
able to obtain free copies of the proxy statement/prospectus 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 before making any voting or
investment decision with respect to the proposed transaction.
Additional Information and Where to Find It
In connection with the proposed transaction between Gemini and
Disc, Gemini filed with the SEC a registration statement on Form
S-4, as amended, containing a definitive proxy statement/prospectus
of Gemini. The registration statement was declared effective by the
SEC on December 2, 2022, and the
special meeting of Gemini stockholders is scheduled to be held on
December 28, 2022. GEMINI URGES
INVESTORS AND STOCKHOLDERS TO READ THESE MATERIALS CAREFULLY AND IN
THEIR ENTIRETY BECAUSE THEY CONTAIN IMPORTANT INFORMATION ABOUT
GEMINI, DISC, THE PROPOSED TRANSACTION AND RELATED MATTERS.
Investors and shareholders are able to obtain free copies of the
definitive proxy statement/prospectus and other documents filed by
Gemini with the SEC 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) and the investor relations
website (https://investors.geminitherapeutics.com/) where anyone is
able to obtain free copies of the proxy statement/prospectus 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 before making any voting or
investment decision with respect to the proposed transaction.
Participants in the Solicitation
Gemini, Disc and their respective 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 definitive proxy/prospectus filed by Gemini with the
SEC on December 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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