Expects to Report Top-Line Results in First Quarter 2014;
Preparations Underway for European Regulatory Filing in Second Half of
NESS ZIONA, Israel--(BUSINESS WIRE)--
Kamada Ltd. (Nasdaq and TASE:KMDA), a plasma-derived protein
therapeutics company focused on orphan indications, announces completion
of the pivotal Phase II/III clinical trial in Europe and Canada of the
Company's proprietary inhaled Alpha-1 Antitrypsin (AAT) therapy for the
treatment of Alpha-1 Antitrypsin Deficiency (AATD or Inherited
Emphysema). Kamada expects to report top-line results from this study in
the first quarter of 2014.
The multicenter randomized, double-blind, placebo-controlled study is
evaluating the safety and efficacy of Kamada's inhaled formulation of
human AAT to treat AATD in >160 patients. The study involved the
inhalation of 160 mg of human AAT or placebo twice daily via the eFlow®
device for 50 weeks, and eligible patients were given the option to
participate in a 50-week open-label extension study. The primary
endpoint of the study is the difference in exacerbation events between
the two groups at one year and is 80% powered to demonstrate a 20%
difference. Power is based on the number of events collected during the
study. Secondary endpoints include additional parameters of exacerbation
events, pulmonary function tests and safety. Additional exploratory
endpoints include CT densitometry in subset of subjects, Quality of Life
measurements and more.
In September 2013 the Company issued a fifth interim safety report based
on data of all patients in the trial as of that date, which was
supportive and consistent with previous reports and demonstrated a high
safety and tolerability profile. At that time, observed adverse events
were related to the expected known symptoms of Inherited Emphysema. No
allergic reactions or signs of any risk related to the use of the
inhalation device were observed.
"We are proud to have completed this pivotal study as planned and look
forward to realizing the potential of our breakthrough inhaled AAT
product. The controlled portion of this pivotal study is complete, with
patients still being treated in an open-label extension study, for which
enrollment rates are very high. We believe these high enrollment rates,
as well as the additional treatment time on the drug, especially for
patients who were on placebo, further support patient and physician
preference for an inhaled treatment for AATD," noted David Tsur, Chief
Executive Officer of Kamada.
"In conjunction with pre-launch marketing activities performed by
Chiesi, our European marketing partner, we are in the process of
preparing a marketing authorization application (MAA) for the European
Medicines Authority (EMA), which we expect to submit in the second half
of 2014. We look forward to advancing our innovative and potentially
more efficacious treatment for patients suffering with this chronic,
life-threatening, inherited lung disease," added Mr. Tsur.
"The opportunity to build a sizeable market is strong, as patients
suffering from AATD remain under-identified and under-treated with less
than 5% of cases treated in the U.S. and approximately 2% in Europe. The
availability of a simple blood test for the diagnosis of AATD is
expected to further increase awareness and favorably impact demand. We
expect that greater AAT use in Europe and other geographies could also
accelerate market growth. Moreover, we believe this non-invasive,
user-friendly potential alternative to intravenous AAT is highly
attractive as a chronic therapy that represents a compelling commercial
opportunity," concluded Mr. Tsur.
In August 2012Kamada signed an exclusive agreement for the distribution
of its inhaled AAT for the treatment of AATD in Europe and with Chiesi
Farmaceutici S.p.A, a fully integrated European pharmaceutical company
focused on respiratory disease and special care products. Under the
agreement Kamada is eligible to receive milestone payments of up to $60
million, subject to achievement of certain regulatory and sales targets.
The agreement is for 12 years and Kamada estimates that the sales
potential from the agreement, provided its inhaled AAT product is
approved for this indication, may reach hundreds of millions of dollars
in the coming years.
Kamada received approval of its Investigational New Drug application
from the U.S. Food and Drug Administration (FDA) for a Phase II clinical
trial with inhaled AAT for AATD, and expects to initiate that trial in
the coming months.
The Company's flagship product is Glassia®, the first and only liquid,
ready-to-use, intravenous plasma-derived AAT product approved by the
FDA. Glassia is marketed in the U.S. through a strategic partnership
with Baxter International.
About Alpha-1 Antitrypsin Deficiency
Alpha-1 antitrypsin, also called AAT, is a protein made in the liver.
Normally the protein travels through the bloodstream and helps protect
the body's organs from the harmful effects of other proteins. The lungs
are one of the main organs that the AAT protein protects. AAT deficiency
(AATD) occurs if the AAT proteins made in the liver are not the right
shape, and they get stuck inside liver cells and cannot get into the
bloodstream. As a result, not enough AAT proteins travel to the lungs to
protect them, which increases the risk of lung disease. Also, liver
disease can develop because too many AAT proteins are stuck in the
liver. Severe AATD occurs when blood levels of the AAT protein fall
below the lowest amount needed to protect the lungs.
AATD is an inherited condition that occurs in all ethnic groups, yet
most often in Caucasians of European descent. It is not known how many
people have AAT deficiency and many people who have the condition may
not know they have it. According to the National Institutes of Health,
estimates of disease incidence range from about 1 in every 1,600 people
to about 1 in every 5,000 people.
Kamada Ltd. is focused on plasma-derived protein therapeutics for orphan
indications, and has a commercial product portfolio and a robust
late-stage product pipeline. The Company uses its proprietary platform
technology and know-how for the extraction and purification of proteins
from human plasma to produce Alpha-1 Antitrypsin (AAT) in a
highly-purified, liquid form, as well as other plasma-derived proteins.
AAT is a protein derived from human plasma with known and
newly-discovered therapeutic roles given its immunomodulatory,
anti-inflammatory, tissue-protective and antimicrobial properties. The
Company's flagship product is Glassia®, the first and only liquid,
ready-to-use, intravenous plasma-derived AAT product approved by the
U.S. Food and Drug Administration. Kamada markets Glassia in the U.S.
through a strategic partnership with Baxter International. In addition
to Glassia, Kamada has a product line of nine other pharmaceutical
products that are marketed through distributors in more than 15
countries, including Israel, Russia, Brazil, India and other countries
in Latin America, Eastern Europe and Asia. Kamada has five late-stage
plasma-derived protein products in development, including an inhaled
formulation of AAT for the treatment of AAT deficiency that completed a
pivotal Phase II/III clinical trials in Europe and will be entering
Phase II clinical trials in the U.S. Kamada also leverages its expertise
and presence in the plasma-derived protein therapeutics market by
distributing 10 complementary products in Israel that are manufactured
by third parties.
Cautionary Note Regarding Forward-Looking Statements
This release includes forward-looking statements within the meaning of
Section 27A of the U.S. Securities Act of 1933, as amended, Section 21E
of the US Securities Exchange Act of 1934, as amended, and the safe
harbor provisions of the U.S. Private Securities Litigation Reform Act
of 1995. Forward-looking statements are statements that are not
historical facts, such as statements regarding assumptions and results
related to financial results forecast, commercial results, clinical
trials, the EMA and U.S. FDA authorizations and timing of clinical
trials. Forward-looking statements are based on Kamada's current
knowledge and its present beliefs and expectations regarding possible
future events and are subject to risks, uncertainties and assumptions.
Actual results and the timing of events could differ materially from
those anticipated in these forward-looking statements as a result of
several factors including, but not limited to, unexpected results of
clinical trials, delays or denial in the U.S. FDA or the EMA approval
process, additional competition in the AATD market or further regulatory
delays. The forward-looking statements made herein speak only as of the
date of this announcement and Kamada undertakes no obligation to update
publicly such forward-looking statements to reflect subsequent events or
circumstances, except as otherwise required by law.
Source: Kamada Ltd.