HONG KONG, Feb 10, 2020 - (ACN Newswire) - Aptorum Group
Limited (Nasdaq: APM) ("Aptorum Group"), a biopharmaceutical
company focused on the development of novel therapeutics to address
global unmet medical needs, announces positive data and development
in relation to its repurposed drug candidate, SACT-1, for the
treatment of neuroblastoma, a rare type of childhood cancer that
develops in infants and young children. Subject to completion of
current validation studies, Aptorum Group plans to leverage the
505(b)(2) pathway and submit an IND submission with the FDA for
SACT-1 in H2 2020.1
SACT-1 is the first repurposed drug candidate to be developed under
the Smart-ACTTM drug discovery platform, which employs a systematic
approach to identify, repurpose and develop existing approved drugs
against a currently identified universe of 7000+ (and increasing)
orphan diseases.2
Through this platform, Aptorum Group intends to accelerate and fast
track repurposed drug candidates, which usually have well
established human safety and toxicity profiles and data, through
the development and clinical phases in order to address the rapidly
growing market of orphan diseases. Aptorum Group aims to screen a
number of orphan disease areas including, but not limited to,
oncology, autoimmune, metabolic and genetic diseases.
Through the Smart-ACTTM platform, Aptorum has successfully
identified potential efficacy for and develops SACT-1 for the
treatment of neuroblastoma, being an entirely new therapeutic area
from its approved indication. In our recent studies, SACT-1 has
been shown to be effective against numerous neuroblastoma cell
lines, of which 2 are MYCN-amplified cells, which represent the
high-risk neuroblastoma patient group. In addition, by using a
combination index as a quantitative measure of the extent of drug
interaction, Aptorum Group has seen a high and robust synergism
between SACT-1 and traditional chemotherapy in vitro, indicating a
potential efficacy enhancement/dose reduction of the chemotherapy.
In addition, in our recent study, the maximum tolerable dose of
SACT-1 in a rodent model was determined to be higher than 400mg/kg.
Compared with the MTD of standard chemotherapy such as paclitaxel
(20-30mg/kg)3 and cisplatin (6mg/kg) 4, the safety profile of
SACT-1 appears to be very impressive.
The reformulation of SACT-1 is a pediatric formulation to better
address the needs of neuroblastoma patients who are exclusively
children younger than 5. Based on our internal observations of
pre-existing information from approved products,5 SACT-1 also
exhibits a well-established safety profile: at 150mg/day, the death
rate was 0% in prior clinical studies) with no dosage related
adverse events.
About neuroblastoma
Neuroblastoma is a rare form of cancer, and classified as an orphan
disease, that forms in certain types of nerve tissue and most
frequently in the adrenal glands as well as spine, chest, abdomen
or neck, predominantly in children, especially for those aged 5
years and below. For the high-risk group, which is close to 20%6 of
total new patient population per year, the 5-year survival rate of
this condition is around 40-50% as observed by the American Cancer
Society7. The current high drug treatment cost for high risk
patients can average USD200,000 per regimen (all 6 cycles)8. In
addition, most pediatric patients often do not tolerate or survive
the relevant chemotherapy stage which, subject to further clinical
studies, may be positively addressed by the SACT-1 candidate due to
the potential synergistic effects when applied with standard
chemotherapy as described above.
For further general presentation, please visit:
http://ir.aptorumgroup.com/static-files/bcf77574-7bd6-4b9d-8110-d53837238f16
For further technical presentation, please visit:
http://ir.aptorumgroup.com/static-files/66346f79-7a03-474a-89be-0eaafaa00d9d
About Aptorum Group Limited
Aptorum Group Limited (Nasdaq: APM) is a pharmaceutical company
dedicated to developing and commercializing novel therapeutics to
tackle unmet medical needs. Aptorum Group is pursuing therapeutic
projects in orphan diseases, infectious diseases, metabolic
diseases and other disease areas.
For more information about Aptorum Group, please visit
www.aptorumgroup.com.
About Smart Pharma Group
Smart Pharma Group is wholly owned by Aptorum Group Limited. Smart
Pharma Group focuses on systematically identifying and repurposing
existing approved drugs for the treatment of a large array of
orphan diseases. Smart Pharma Group conducts both computational
based screening and clinical validations in advancing the
development of its repurposed candidates.
Disclaimer and Forward-Looking Statements
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
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 a result, the projections included
in such forward-looking statements are subject to change. 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.
1 If the FDA deems the 505(b)(2) pathway as an acceptable route for
approval of SACT-1, the Company will be able to leverage existing
clinical and nonclinical data in conjunction with sponsor-initiated
studies to accelerate development and approval of SACT-1.
2 See
https://rarediseases.info.nih.gov/diseases/pages/31/faqs-about-rare-diseases
3 Clin Cancer Res. 5(11):3632-8.
4 BMC Cancer 17: 684 (2017).
5 Subject to FDA's approval and on a case-by-case basis, a
505(b)(2) application can rely in part on existing information from
approved products (such as the FDA's previous findings on safety
and efficacy) or products in literature (such as data available).
However, typically speaking, the applicant is nonetheless required
to carry out a Phase 1 bridging study to compare the Reference
Listed Drug and reference the established safety and efficacy
information.
6 Annu Rev Med. 2015; 66: 49-63.
7https://www.cancer.org/cancer/neuroblastoma/detection-diagnosis-staging/survival-rates.html
8
https://www.cadth.ca/sites/default/files/pcodr/Reviews2019/10154DinutuximabNeuroblastoma_fnEGR_NOREDACT-ABBREV_Post_26Mar2019_final.pdf
Contacts
Investors:
Tel: +852 2117 6611
Email: investor.relations@aptorumgroup.com
Media:
Tel: + 852 2117 6611
Email: info@aptorumgroup.com
Source: Aptorum Group Limited
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