Aptorum Group Receives FDA Orphan Drug Designation for its SACT-1 Repurposed Drug For The Treatment of Neuroblastoma
21 January 2022 - 12:00AM
Business Wire
Regulatory News:
Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM)
(“Aptorum Group” or “Aptorum”), a clinical-stage biopharmaceutical
company, is pleased to announce that the United States Food and
Drug Administration (FDA) Office has granted Orphan Drug
Designation to SACT-1, a repurposed small molecule compound for the
treatment of patients with Neuroblastoma. Aptorum Group plans to
file an Investigational New Drug Application (IND) to commence a
phase 1b/2a clinical trial for SACT-1 to test the drug in
neuroblastoma patients in 2022.
Mr. Darren Lui, President and Executive Director of Aptorum
Group says, “The granting of orphan drug designation for SACT-1 for
the treatment of neuroblastoma is another important step forward in
the development of our drug candidate and reflects both the FDA’s
and Aptorum’s commitment to addressing the unmet clinical needs of
patients with neuroblastoma.” Further to our recently announced
completion of Phase 1 clinical trial and patent grant for SACT-1,
we are currently focusing on our IND preparation for entering into
the exciting Phase Ib/2a clinical trials for SACT-1 in the United
States.”
About SACT-1
SACT-1 is an orally administered repurposed small molecule drug
to target neuroblastoma. SACT-1’s mechanism has been investigated
in our preclinical studies to enhance tumor cell death and suppress
MYCN expression (a common clinical diagnosis in high-risk or
relapsed neuroblastoma patients where an amplification of MYCN is
usually observed). SACT-1 is designed to be used especially in
combination with standard-of-care chemotherapy.
About Neuroblastoma
Neuroblastoma is one of the most prevailing solid tumor cancers
in children, representing 8% - 10% of all childhood tumors,
accounting for c. 15% of all cancer related deaths in the pediatric
population1. For the high-risk patient group, the 5-year survival
rate of this condition is around 40-50% as observed by the American
Cancer Society2 based on existing treatment.
1
https://www.ncbi.nlm.nih.gov/pmc/articles/PMC3668791/#:~:text=Neuroblastoma%20is%20the%20most%20common,deaths%20in%20the%20pediatric%20population.
2
https://www.cancer.org/cancer/neuroblastoma/detection-diagnosis-staging/survival-rates.html
About Aptorum Group Limited
Aptorum Group Limited (Nasdaq: APM, Euronext Paris: APM) is a
clinical stage biopharmaceutical company dedicated to the
discovery, development and commercialization of therapeutic assets
to treat diseases with unmet medical needs, particularly in
oncology (including orphan oncology indications) and infectious
diseases. The pipeline of Aptorum is also enriched through (i) the
establishment of drug discovery platforms that enable the discovery
of new therapeutics assets through, e.g. systematic screening of
existing approved drug molecules, and microbiome-based research
platform for treatments of metabolic diseases; and (ii) the
co-development of a novel molecular-based rapid pathogen
identification and detection diagnostics technology with Accelerate
Technologies Pte Ltd, commercialization arm of the Singapore’s
Agency for Science, Technology and Research.
For more information about Aptorum Group, please visit
www.aptorumgroup.com.
Disclaimer and Forward-Looking Statements
This press release does not constitute an offer to sell or a
solicitation of offers to buy any securities of Aptorum Group.
This press release includes statements concerning Aptorum Group
Limited and its future expectations, plans and prospects that
constitute “forward-looking statements” within the meaning of the
US Private Securities Litigation Reform Act of 1995. For this
purpose, any statements contained herein that are not statements of
historical fact may be deemed to be forward-looking statements. In
some cases, you can identify forward-looking statements by terms
such as “may,” “should,” “expects,” “plans,” “anticipates,”
“could,” “intends,” “target,” “projects,” “contemplates,”
“believes,” “estimates,” “predicts,” “potential,” or “continue,” or
the negative of these terms or other similar expressions. Aptorum
Group has based these forward-looking statements, which include
statements regarding projected timelines for application
submissions and trials, largely on its current expectations and
projections about future events and trends that it believes may
affect its business, financial condition and results of operations.
These forward-looking statements speak only as of the date of this
press release and are subject to a number of risks, uncertainties
and assumptions including, without limitation, risks related to its
announced management and organizational changes, the continued
service and availability of key personnel, its ability to expand
its product assortments by offering additional products for
additional consumer segments, development results, the company’s
anticipated growth strategies, anticipated trends and challenges in
its business, and its expectations regarding, and the stability of,
its supply chain, and the risks more fully described in Aptorum
Group’s Form 20-F and other filings that Aptorum Group may make
with the SEC in the future, as well as the prospectus that received
the French Autorité des Marchés Financiers visa n°20-352 on 16 July
2020. As a result, the projections included in such forward-looking
statements are subject to change and actual results may differ
materially from those described herein.
Aptorum Group assumes no obligation to update any
forward-looking statements contained in this press release as a
result of new information, future events or otherwise.
This announcement is not a prospectus within the meaning of the
Regulation (EU) n°2017/1129 of 14 June 2017 as amended by
Regulations Delegated (EU) n°2019/980 of 14 March 2019 and
n°2019/979 of 14 March 2019.
This press release is provided “as is” without any
representation or warranty of any kind.
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Investor Relations Aptorum Group Limited Investor
Relations Department investor.relations@aptorumgroup.com +44 20
80929299
Redchip – Financial Communications United States Investor
relations Dave Gentry dave@redchip.com +1 407 491 4498
Actifin – Financial Communications Europe Investor relations
Ghislaine Gasparetto ggasparetto@actifin.fr +33 1 56 88 11 22
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