XOMA Receives Orphan Drug Designation in the European Union for XOMA 358 for Treatment of Congenital Hyperinsulinism
July 20 2016 - 9:00AM
XOMA Corporation (NASDAQ:XOMA), a leader in the discovery and
development of therapeutic antibodies, today announced the European
Medicines Agency (EMA) has granted Orphan Drug Designation to XOMA
358 for the treatment of congenital hyperinsulinism (CHI), a rare
genetic disorder in which the insulin cells of the pancreas (beta
cells) secrete inappropriate and excessive insulin. XOMA 358
is a negative allosteric antibody that binds to the insulin
receptor and down-regulates insulin action. It is in Phase 2
development for CHI with clinical sites in the United States and
European Union (EU) actively enrolling patients.
“Congenital hyperinsulinism is a devastating
disease that presents in infancy and leads to profound
hypoglycemia, which can cause significant morbidities, including
brain damage, seizures and epilepsy. Both the disease and current
treatments have potentially life-long physical and psychological
impacts on patients and their families. The EMA Orphan Drug
Designation for CHI recognizes the significant unmet medical need
for CHI patients, and is an important step in the development
pathway for XOMA 358,” said Paul Rubin, M.D., Senior Vice
President, Research and Development, and Chief Medical Officer at
XOMA. "The CHI community has been very supportive of our efforts to
advance the XOMA 358 clinical program. With Orphan Drug
Designation now secured in both the US and EU, we will continue to
work closely with agencies in both regions to design a regulatory
pathway that will expedite the clinical development for XOMA
358.”
To qualify for orphan designation in the EU, a
therapeutic agent must be intended to treat a life-threatening or
chronically debilitating disease that affects no more than five
people in 10,000, and for which there is no treatment option or the
current treatment options are unsatisfactory. Orphan Drug
Designation in the EU brings numerous benefits to the company
developing the therapeutic agent, including assistance with
clinical protocols, scientific advice, and up to 10 years of
marketing exclusivity upon approval.
About Congenital
HyperinsulinismCHI is a genetic disorder in which the
insulin cells of the pancreas (beta cells) secrete inappropriate
and excessive insulin. In healthy individuals, beta cells secrete
just enough insulin to keep blood sugar in the normal range. In
people with CHI, the secretion of insulin is not properly
regulated, causing excess insulin secretion and frequent episodes
of low blood sugar (hypoglycemia). In infants and young
children, these episodes are characterized by a lack of energy
(lethargy), irritability or difficulty with feeding. Repeated
episodes of low blood sugar increase the risk for serious
complications, such as breathing difficulties, seizures,
intellectual disability, vision loss, brain damage, coma and
possibly death. About 60 percent of infants with CHI experience a
hypoglycemic episode within the first month of life. Other affected
children develop hypoglycemia by early childhood. Current
treatments for CHI are limited to medical therapy and surgical
removal of part or all of the pancreas (pancreatectomy).
About XOMA 358Insulin is the major
physiologic hormone for controlling blood glucose levels. Abnormal
increases in insulin secretion can lead to profound hypoglycemia
(low blood sugar), a state that can result in significant
morbidities, including brain damage, seizures and epilepsy. XOMA,
leveraging its scientific expertise in allosteric monoclonal
antibodies, developed the XMet platform, consisting of separate
classes of selective insulin receptor modulators (SIRMs) that could
have a major effect on treating patients with abnormal metabolic
states. XOMA 358 binds selectively to insulin receptors and
down-regulates insulin action.
XOMA 358, which has received Orphan Drug
Designation from the U.S. Food and Drug Administration and from the
European Medicines Agency, is being investigated as a novel
treatment for non-drug-induced, endogenous hyperinsulinemic
hypoglycemia, as well as hypoglycemia after bariatric surgery. XOMA
recently initiated Phase 2 studies in patients with CHI, and in
patients with hypoglycemia post bariatric surgery. A therapy
that safely and effectively mitigates insulin-induced hypoglycemia
has the potential to address a significant unmet therapeutic need
for certain rare medical conditions associated with
hyperinsulinism. More information on the XOMA 358 clinical
trials can be found at www.clinicaltrials.gov and
www.clinicaltrialsregister.eu.
About XOMA CorporationXOMA
Corporation is a leader in the discovery and development of
therapeutic antibodies. The Company's innovative product
candidates result from its expertise in developing ground-breaking
monoclonal antibodies, including allosteric antibodies, which have
created new opportunities to potentially treat a wide range of
human diseases. XOMA's scientific research has produced a portfolio
of five endocrine assets, each of which has the opportunity to
address multiple indications. The Company's lead product
candidate, XOMA 358, is an allosteric monoclonal antibody that
reduces insulin receptor activity, which could have a major impact
on hyperinsulinism. The Company recently initiated Phase 2
development activities for XOMA 358 in patients with congenital
hyperinsulinism and in patients who experience hypoglycemia
following gastric bypass surgery. For more information, visit
www.xoma.com.
Forward-Looking StatementsCertain
statements contained in this press release including, but not
limited to, statements related to anticipated timing of clinical
trials, anticipated timing of the release of clinical data,
regulatory approval of unapproved product candidates, the
anticipated process of clinical data analysis, the anticipated
success of any clinical trial, cash usage, or statements that
otherwise relate to future periods are forward-looking statements
within the meaning of Section 27A of the Securities Act of 1933 and
Section 21E of the Securities Exchange Act of 1934. These
statements are based on assumptions that may not prove accurate,
and actual results could differ materially from those anticipated
due to certain risks inherent in the biotechnology industry and for
companies engaged in the development of new products in a regulated
market. Potential risks to XOMA meeting these expectations are
described in more detail in XOMA's most recent filing on Form 10-K
and in other SEC filings. Consider such risks carefully when
considering XOMA's prospects. Any forward-looking statement in this
press release represents XOMA's views only as of the date of this
press release and should not be relied upon as representing its
views as of any subsequent date. XOMA disclaims any obligation to
update any forward-looking statement, except as required by
applicable law.
Company and Investor Contact:
Ashleigh Barreto
510-204-7482
barreto@xoma.com
Juliane Snowden
The Oratorium Group, LLC
jsnowden@oratoriumgroup.com
Media Contact:
Taryn Ibach
W2O Group
415-658-9748
tibach@w2ogroup.com
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