Kamada Submits Marketing Authorization Application with the European Medicines Agency for its Proprietary Inhaled Alpha-1 Antitrypsin to Treat Alpha-1 Antitrypsin Deficiency

NESS ZIONA, Israel–(BUSINESS WIRE)– Kamada Ltd. (NASDAQ:KMDA) (TASE:KMDA), a plasma-derived protein
therapeutics company focused on orphan indications, announces the
submission of a Marketing Authorization Application (MAA) with the
European Medicines Agency (EMA) for the Company’s proprietary, inhaled
alpha-1 antitrypsin (AAT) therapy as a treatment for AAT deficiency
(AATD). The filing was validated by the EMA.

“The submission of this application represents an important achievement
that brings us one step closer to our goal of commercializing our
inhaled AAT therapy for the benefit of patients suffering with AATD in
Europe,” stated Amir London, Chief Executive Officer of Kamada. “The EMA
has agreed to evaluate the totality of the data from our innovative
Phase 2/3 study, and based upon orphan designation of the drug, prior
discussions with regulators, the strength of these data, the support we
get from the key opinion leaders and the patient community, and the
persistent unmet need in this chronic disease, we are highly optimistic
of a favorable outcome. Importantly, the combination of lung function
measurements, which are the gold standard for pulmonary diseases, and
symptom improvements, along with the safety profile of the product,
gives us confidence these data meet the risk/benefit balance required by
EMA.”

“The submission of the MAA for inhaled alpha-1 antitrypsin to treat AATD
is a major step toward bringing another treatment to AATD patients. This
study is the first study ever that shows inhaled AAT’s ability to reduce
the decline in FEV₁ in a patient population suffering from
frequent exacerbations of dyspnea and coughing. I believe these results
support the ability to treat AATD patients with Kamada’s inhaled AAT. I
am looking forward to the regulatory authorities’ approval for the
benefit of AATD patients,“ stated Jan Stolk, MD, Department of
Pulmonology, Leiden University Medical Center and Principal Investigator
of the Phase 2/3 clinical trial.

“Kamada’s MAA submission for its inhaled AAT is a major breakthrough
that might affect the critical element of disease progression. This
pioneering study demonstrated a significant improvement in lung
function. It is to be hoped that this novel treatment will soon be
available for the benefit of the AATD patient community,” commented
Robert Stockley, MB, ChB, MD, DSc, FERS, Professor of
Medicine, University Hospital Birmingham, and an Investigator in the
Phase 2/3 clinical trial.

“This submission by Kamada marks a significant milestone in the
management of pulmonary disease caused by severe AATD. Kamada’s inhaled
AAT provides a simple treatment with twice-daily inhalation that offers
improvements in lung function and reduces the discomfort of
exacerbations. Approval of Kamada’s AAT for inhalation will offer a new
and important intervention to protect this vulnerable patient
population,” noted Kenneth R. Chapman, MD, MSc, FRCPC, FACP, FCCP,
Director, Asthma & Airway Centre, University Health Network, Professor
of Medicine, University of Toronto, and an Investigator in the Phase 2/3
clinical trial.

Phase 2/3 Trial Summary

The MAA filing is based upon a Phase 2/3 multicenter randomized,
double-blind, placebo-controlled study that evaluated the safety and
efficacy of Kamada’s inhaled formulation of human AAT to treat AATD in
168 patients. The study involved the inhalation of 80 mg of human AAT or
placebo twice daily via the eFlow® device for 50 weeks. The primary
endpoint of the study was time to the first moderate or severe
exacerbation event. Secondary endpoints included additional parameters
of exacerbation events. Lung function parameters including Forced
Expiratory Volume in One Second (FEV₁) % of Slow Vital
Capacity (SVC), FEV₁ % predicted, FEV₁ (liters)
and Diffusing capacity (DLCO), were collected to support safety
endpoints. Additional exploratory endpoints included CT densitometry in
a subset of subjects, Quality of Life measurements and more.

Despite not meeting the primary or secondary endpoints, lung function
parameters, which were collected to support safety endpoints, showed
concordance of a significant treatment effect in the reduction of the
inflammatory injury to the lung, which is known to be associated with a
reduced loss of respiratory function.

Lung Function Results

Analysis of the lung functions in the safety population as described
below indicate that after one year of daily inhalation of Kamada’s AAT,
clinically and statistically significant improvements were seen in
spirometric measures of lung function, particularly in bronchial airflow
measurements FEV₁ (L), FEV₁% predicted and FEV₁/SVC.

For lung function overall one-year effect:

• FEV₁ (L) rose significantly in AAT-treated patients and
  decreased in placebo-treated patients (+15ml for AAT vs. -27ml for
  placebo, a 42 ml difference, p=0.0268)
• There was a trend towards better FEV₁% predicted (0.54% for
  AAT vs. -0.62% for placebo, a 1.16% difference, p=0.065)
• FEV₁/SVC% rose significantly in AAT-treated patients and
  decreased in placebo-treated patients (0.62% for AAT vs. -0.87% for
  placebo, a 1.49% difference, p=0.0074)

For lung function change at week 50 vs. baseline:

• There was a trend towards reduced FEV₁ (L) decline (-12ml
  for AAT vs. -62ml for placebo, a 50 ml difference, p=0.0956)
• There was a trend towards a reduced decline in FEV₁%
  predicted (-0.1323% for AAT vs. -1.6205% for placebo, a 1.4882%
  difference, p=0.1032)
• FEV₁/SVC% rose significantly in AAT-treated patients and
  decreased in placebo-treated patients (0.61% for AAT vs. -1.07% for
  placebo, a 1.68% difference, p=0.013)

About Alpha-1 Antitrypsin Deficiency

Alpha-1 antitrypsin (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 or
inherited emphysema) 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 populations,
yet most often in Caucasians of European descent. It is not known how
many people have AATD 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.

About eFlow® Technology and PARI Pharma

The Company’s inhaled AAT therapy is delivered by an investigational
eFlow® Nebulizer System developed by PARI Pharma and optimized
specifically for Kamada. The optimized device uses eFlow Technology to
enable highly efficient aerosolization of medication including liposomal
formulations via a vibrating, perforated membrane that includes
thousands of laser-drilled holes. Compared with other nebulization
technologies, eFlow Technology produces aerosols with a very high
density of active drug, a precisely defined droplet size and a high
proportion of respirable droplets delivered in the shortest possible
period of time. eFlow Technology is not an ultrasonic nebulizer
technology and is not a general purpose electronic aerosol generator
nebulizer technology. Combined with its quiet mode of operation, small
size, light weight and battery use, eFlow Technology reduces the burden
of taking daily, inhaled treatments.

About Kamada

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 Immune
globulins. 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 Baxalta. In addition to Glassia,
Kamada has a product line of seven other pharmaceutical products
administered by injection or infusion, 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 its MAA was submitted to the EMA after completing
a pivotal Phase 2/3 clinical trials in Europe and is in Phase 2 clinical
trials in the U.S. and its intravenous AAT to treat type-1 diabetes,
GVHD and prevention of lung transplant rejection. Kamada also leverages
its expertise and presence in the plasma-derived protein therapeutics
market by distributing more than 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 U.S. 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, timing and
results of clinical trials and EMA and U.S. FDA authorizations.
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.

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