RedHill Biopharma Receives FDA Orphan Drug Designation for YELIVA® for the Treatment of Cholangiocarcinoma
(Thomson Reuters ONE) -
* Orphan Drug designation allows RedHill to benefit from various development
incentives to develop YELIVA(®) (ABC294640) for cholangiocarcinoma, as well
as a seven-year marketing exclusivity period for the indication, if approved
for marketing
* A Phase IIa clinical study with YELIVA(®) in patients with advanced,
unresectable, intrahepatic and extrahepatic cholangiocarcinoma is planned to
be initiated in the third quarter of 2017
* Cholangiocarcinoma (bile duct cancer) is a highly lethal malignancy for
which there is a strong need for more effective systemic treatments; the 5-
year relative survival rate for patients with cholangiocarcinoma ranges
between 2% to 30%, depending on the tumor type and stage at diagnosis
* A Phase I study with YELIVA(®) in patients with advanced solid tumors
successfully met its primary and secondary endpoints; of the three
cholangiocarcinoma patients in the Phase I study, one patient had a
sustained partial response and the other two had prolonged stable disease
* RedHill is pursuing several Phase I/II clinical studies with YELIVA(®),
targeting multiple oncology and inflammatory indications, some of which are
supported by National Cancer Institute (NCI) grants awarded to Apogee
Biotechnology and U.S. universities
* YELIVA(®) is a proprietary, first-in-class, orally-administered sphingosine
kinase-2 (SK2) selective inhibitor, with anti-cancer and anti-inflammatory
activities
TEL-AVIV, Israel, April 04, 2017 (GLOBE NEWSWIRE) -- RedHill Biopharma Ltd.
(NASDAQ:RDHL) (Tel-Aviv Stock Exchange:RDHL) ("RedHill" or the "Company"), a
specialty biopharmaceutical company primarily focused on the development and
commercialization of late clinical-stage, proprietary, orally-administered,
small molecule drugs for gastrointestinal and inflammatory diseases and cancer,
today announced that the U.S. Food and Drug Administration (FDA) has granted
YELIVA(®) (ABC294640) Orphan Drug designation for the treatment of
cholangiocarcinoma.
The Orphan Drug designation allows RedHill to benefit from various development
incentives to develop YELIVA® for this indication, including tax credits for
qualified clinical testing, waiver of a prescription drug user fee (PDUFA fee)
upon submission of a potential marketing application and, if approved, a seven-
year marketing exclusivity period for the treatment of cholangiocarcinoma.
YELIVA(®) is a Phase II-stage, proprietary, first-in-class, orally-administered
sphingosine kinase-2 (SK2) selective inhibitor with anticancer and anti-
inflammatory activities, targeting multiple oncology, inflammatory and
gastrointestinal indications. By inhibiting the SK2 enzyme, YELIVA(®) blocks the
synthesis of sphingosine 1-phosphate (S1P), a lipid signaling molecule that
promotes cancer growth and pathological inflammation.
Mark L. Levitt, MD, PhD, RedHill's Medical Director, Oncology,
said: "Cholangiocarcinoma is a cancer with a poor prognosis. Patients suffering
from this disease have very few treatment options, and they are of limited
efficacy. Based on promising preclinical data, as well as results from three
previously treated cholangiocarcinoma patients who took part in the Phase I
study with YELIVA(®), we are hopeful that YELIVA(®) could potentially provide a
much-needed new treatment option for patients. We are very pleased with the
Orphan Drug designation and are advancing our preparations for a Phase IIa study
to evaluate the safety and efficacy of YELIVA(®) in patient suffering from
unresectable, intrahepatic and extrahepatic cholangiocarcinoma, which we plan to
initiate in the third quarter of this year."
Cholangiocarcinoma (bile duct cancer) is a highly lethal malignancy for which
there is a strong need for more effective systemic treatments. Approximately
8,000 people are diagnosed with intrahepatic and extrahepatic bile duct cancers
annually in the U.S.(1), with recent studies showing an increased incidence of
cholangiocarcinoma, mainly attributed to recent advancements in diagnosis of
this disease(2). Surgery with complete resection remains the only curative
therapy for cholangiocarcinoma, however only a minority of patients are
classified as having a resectable tumor at the time of diagnosis(3). Additional
treatment options include radiation therapy and chemotherapy; however, the
efficacy of these treatments in cholangiocarcinoma patients is also limited.
Despite overall advances in the ability to diagnose and treat patients with
cholangiocarcinoma, the prognosis for these relapse patients who have failed
initial chemotherapy remains very poor, with an overall median survival of
approximately one year(4). The 5-year relative survival rates of intrahepatic
and extrahepatic cholangiocarcinoma patients range between 2% to 30%, depending
on the tumor type and stage at diagnosis(5).
Final results from the Phase I study with YELIVA(®) in patients with advanced
solid tumors confirmed that the study, conducted at the Medical University of
South Carolina (MUSC) Hollings Cancer Center, successfully met its primary and
secondary endpoints, demonstrating that the drug is well-tolerated and can be
safely administered to cancer patients at doses that provide circulating drug
levels that are predicted to have therapeutic activity.
Of the three patients with cholangiocarcinoma treated in the Phase I study, all
of whom had prior therapy, one subject achieved a sustained partial response
(Overall Survival (OS) = 20.3 months) and the other two subjects had prolonged
stable disease (OS = 17.6 and 16.3 months).
RedHill plans to initiate a Phase IIa clinical study with YELIVA(®) in patients
with advanced, unresectable, intrahepatic and extrahepatic cholangiocarcinoma in
the third quarter of 2017. The single-arm study will evaluate YELIVA(®) as a
single agent in cholangiocarcinoma patients with a primary endpoint of
determining the response rate of cholangiocarcinoma to this treatment.
A Phase II study with YELIVA(®) for the treatment of advanced hepatocellular
carcinoma (HCC) is ongoing at MUSC Hollings Cancer Center. The study is
supported by a $1.8 million grant from the NCI, awarded to MUSC, which is
intended to fund a broad range of studies on the feasibility of targeting
sphingolipid metabolism for the treatment of a variety of solid tumor cancers,
with additional support from RedHill.
A Phase Ib/II study with YELIVA(®) for the treatment of refractory or relapsed
multiple myeloma is ongoing at Duke University Medical Center. The study is
supported by a $2 million grant from the NCI Small Business Innovation Research
Program (SBIR) awarded to Apogee Biotechnology Corp. (Apogee), in conjunction
with Duke University, with additional support from RedHill.
A Phase I/II clinical study evaluating YELIVA(®) in patients with
refractory/relapsed diffuse large B-cell lymphoma as well as Kaposi sarcoma
patients is ongoing at the Louisiana State University Health Sciences Center.
The study is supported by a grant from the NCI awarded to Apogee, with
additional support from RedHill.
A Phase Ib study to evaluate YELIVA(®) as a radioprotectant for prevention of
mucositis in head and neck cancer patients undergoing therapeutic radiotherapy
is planned to be initiated in the third quarter of 2017.
A Phase II study to evaluate the efficacy of YELIVA(®) in patients with moderate
to severe ulcerative colitis is planned to be initiated in the second half of
2017.
About YELIVA(®) (ABC294640):
YELIVA(®) (ABC294640) is a Phase II-stage, proprietary, first-in-class, orally-
administered, sphingosine kinase-2 (SK2) selective inhibitor with anti-cancer
and anti-inflammatory activities. RedHill is pursuing with YELIVA(®) multiple
clinical programs in oncology, inflammatory and gastrointestinal indications. By
inhibiting SK2, YELIVA(®) blocks the synthesis of sphingosine 1-phosphate (S1P),
a lipid signaling molecule that promotes cancer growth and pathological
inflammation. SK2 is an innovative molecular target for anticancer therapy
because of its critical role in catalyzing the formation of S1P, which is known
to regulate cell proliferation and activation of inflammatory pathways.
YELIVA(®) was originally developed by U.S.-based Apogee Biotechnology Corp. and
completed multiple successful pre-clinical studies in oncology, inflammation, GI
and radioprotection models, as well as the ABC-101 Phase I clinical study in
cancer patients with advanced solid tumors. The Phase I study included the
first-ever longitudinal analysis of plasma S1P levels as a potential
pharmacodynamic (PD) biomarker for activity of a sphingolipid-targeted drug. The
administration of YELIVA(®) resulted in a rapid and pronounced decrease in S1P
levels, with several patients having prolonged stabilization of disease.
YELIVA(®) received Orphan Drug designation from the U.S. FDA for the treatment
of cholangiocarcinoma. The development of YELIVA(®) was funded to date primarily
by grants and contracts from U.S. federal and state government agencies awarded
to Apogee Biotechnology Corp., including the U.S. National Cancer Institute, the
U.S. Department of Health and Human Services' Biomedical Advanced Research and
Development Authority (BARDA), the U.S. Department of Defense and the FDA Office
of Orphan Products Development.
About RedHill Biopharma Ltd.:
RedHill Biopharma Ltd. (NASDAQ:RDHL) (Tel-Aviv Stock Exchange:RDHL) is a
specialty biopharmaceutical company headquartered in Israel, primarily focused
on the development and commercialization of late clinical-stage, proprietary,
orally-administered, small molecule drugs for the treatment of gastrointestinal
and inflammatory diseases and cancer. RedHill has a U.S. co-promotion agreement
with Concordia for Donnatal(®), a prescription oral adjunctive drug used in the
treatment of IBS and acute enterocolitis. RedHill's clinical-stage pipeline
includes: (i) RHB-105 - an oral combination therapy for the treatment
of Helicobacter pylori infection with successful results from a first Phase III
study; (ii) RHB-104 - an oral combination therapy for the treatment of Crohn's
disease with an ongoing first Phase III study, a completed proof-of-concept
Phase IIa study for multiple sclerosis and QIDP status for nontuberculous
mycobacteria (NTM) infections; (iii) BEKINDA(®) (RHB-102) - a once-daily oral
pill formulation of ondansetron with an ongoing Phase III study for acute
gastroenteritis and gastritis and an ongoing Phase II study for IBS-D; (iv) RHB-
106 - an encapsulated bowel preparation licensed to Salix Pharmaceuticals, Ltd.;
(v) YELIVA(®) (ABC294640) - a Phase II-stage, orally-administered, first-in-
class SK2 selective inhibitor targeting multiple oncology, inflammatory and
gastrointestinal indications; (vi) MESUPRON - a Phase II-stage first-in-class,
orally-administered protease inhibitor, targeting pancreatic cancer and other
solid tumors and (vii) RIZAPORT(®) (RHB-103) - an oral thin film formulation of
rizatriptan for acute migraines, with a U.S. NDA currently under discussion with
the FDA and marketing authorization received in Germany in October 2015. More
information about the Company is available at: www.redhillbio.com.
This press release contains "forward-looking statements" within the meaning of
the Private Securities Litigation Reform Act of 1995. Such statements may be
preceded by the words "intends," "may," "will," "plans," "expects,"
"anticipates," "projects," "predicts," "estimates," "aims," "believes," "hopes,"
"potential" or similar words. Forward-looking statements are based on certain
assumptions and are subject to various known and unknown risks and
uncertainties, many of which are beyond the Company's control, and cannot be
predicted or quantified and consequently, actual results may differ materially
from those expressed or implied by such forward-looking statements. Such risks
and uncertainties include, without limitation, risks and uncertainties
associated with (i) the initiation, timing, progress and results of the
Company's research, manufacturing, preclinical studies, clinical trials, and
other therapeutic candidate development efforts; (ii) the Company's ability to
advance its therapeutic candidates into clinical trials or to successfully
complete its preclinical studies or clinical trials; (iii) the extent and number
of additional studies that the Company may be required to conduct and the
Company's receipt of regulatory approvals for its therapeutic candidates, and
the timing of other regulatory filings, approvals and feedback; (iv) the
manufacturing, clinical development, commercialization, and market acceptance of
the Company's therapeutic candidates; (v) the Company's ability to successfully
market Donnatal(®), (vi) the Company's ability to establish and maintain
corporate collaborations; (vii) the Company's ability to acquire products
approved for marketing in the U.S. that achieve commercial success and build its
own marketing and commercialization capabilities; (viii) the interpretation of
the properties and characteristics of the Company's therapeutic candidates and
of the results obtained with its therapeutic candidates in research, preclinical
studies or clinical trials; (ix) the implementation of the Company's business
model, strategic plans for its business and therapeutic candidates; (x) the
scope of protection the Company is able to establish and maintain for
intellectual property rights covering its therapeutic candidates and its ability
to operate its business without infringing the intellectual property rights of
others; (xi) parties from whom the Company licenses its intellectual property
defaulting in their obligations to the Company; and (xii) estimates of the
Company's expenses, future revenues capital requirements and the Company's needs
for additional financing; (xiii) competitive companies and technologies within
the Company's industry. More detailed information about the Company and the risk
factors that may affect the realization of forward-looking statements is set
forth in the Company's filings with the Securities and Exchange Commission
(SEC), including the Company's Annual Report on Form 20-F filed with the SEC on
February 23, 2017. All forward-looking statements included in this Press Release
are made only as of the date of this Press Release. We assume no obligation to
update any written or oral forward-looking statement unless required by law.
(1) American Cancer Society, Bile Duct
Cancer: www.cancer.org/acs/groups/cid/documents/webcontent/003084-pdf.pdf, Jan
20, 2016.
(2) Gores GJ. Cholangiocarcinoma: current concepts and insights. Hepatology
(Baltimore, Md). 2003 May;37(5):961-9.
(3) Banales JM et al. Expert consensus document: Cholangiocarcinoma: current
knowledge and future perspectives consensus statement from the European Network
for the Study of Cholangiocarcinoma (ENS-CCA), Nat Rev Gastroenterol
Hepatol. 2016;13:261-280.
(4) Valle J, Wasan H, Palmer DH, et al. Cisplatin plus gemcitabine versus
gemcitabine for biliary tract cancer. New Eng J Med 2010;362:1273-81.
(5) Howlader N, Noone AM, Krapcho M, Miller D, Bishop K, Altekruse SF, Kosary
CL, Yu M, Ruhl J, Tatalovich Z, Mariotto A, Lewis DR, Chen HS, Feuer EJ, Cronin
KA (eds). SEER Cancer Statistics Review, 1975-2013, National Cancer Institute.
Bethesda, MD, http://seer.cancer.gov/csr/1975_2013/, based on November 2015 SEER
data submission, posted to the SEER web site, April 2016.
Company contact:
Adi Frish
Senior VP Business Development &
Licensing
RedHill Biopharma
+972-54-6543-112
adi(at)redhillbio.com
IR contact (U.S.):
Marcy Nanus
Senior Vice President
The Trout Group
+1-646-378-2927
Mnanus(at)troutgroup.com
This announcement is distributed by Nasdaq Corporate Solutions on behalf of Nasdaq Corporate Solutions clients.
The issuer of this announcement warrants that they are solely responsible for the content, accuracy and originality of the information contained therein.
Source: RedHill Biopharma Ltd. via GlobeNewswire
Datum: 04.04.2017 - 12:05 Uhr
Sprache: Deutsch
News-ID 534386
Anzahl Zeichen: 18328
contact information:
Town:
Tel-Aviv
Kategorie:
Business News
Diese Pressemitteilung wurde bisher 120 mal aufgerufen.
Die Pressemitteilung mit dem Titel:
"RedHill Biopharma Receives FDA Orphan Drug Designation for YELIVA® for the Treatment of Cholangiocarcinoma"
steht unter der journalistisch-redaktionellen Verantwortung von
RedHill Biopharma Ltd. (Nachricht senden)
Beachten Sie bitte die weiteren Informationen zum Haftungsauschluß (gemäß TMG - TeleMedianGesetz) und dem Datenschutz (gemäß der DSGVO).
