Mucosal Improvement Observed in 61% of the
Patients and 33% achieved Mucosal Healing
Protalix BioTherapeutics, Inc. (NYSE American:PLX) (TASE:PLX),
announced today that additional positive results from the Company’s
phase II clinical trial of OPRX-106 for the treatment of ulcerative
colitis (UC) were presented at the Digestive Disease Week® (DDW)
2018 Annual Meeting. OPRX-106 is a plant cell-expressed
recombinant human tumor necrosis factor receptor II fused to an
IgG1 Fc domain (TNFRII-Fc), in development for oral administration.
When administered orally and passing through the digestive
tract, the plant cells function as a natural delivery vehicle.
The unique attribute of a cellulose plant cell wall provides
resistance to degradation as opposed to proteins produced via
mammalian cell expression. The Digestive Disease Week® (DDW)
2018 Annual Meeting is taking place in Washington, D.C., June 2-5,
2018.
The phase II clinical trial is a randomized, open label, 2-arm
study of OPRX-106 for the treatment of ulcerative colitis
(UC). A total of 24 patients were enrolled in the study, 18
patients completed the study with 6 patients who did not complete
the study. The dropout rate is consistent with other trials
in similar patient populations, and none of the patients dropped
out due to a side effect or serious adverse event. Patients
were randomized to receive 2 mg or 8 mg of OPRX-106, administered
orally, once daily, for 8 weeks. The average baseline Mayo
score was 7.1 (from a scale of 0-12) and the average baseline
mucosal endoscopy sub score was 2.1 (from a scale of 0-3).
For the 18 patients who completed the study, 89% had a
baseline Mayo score between 6 and 9, which meets the criteria of
moderate disease activity, and 84% had a baseline mucosal endoscopy
sub score of 2 and above indicating moderate to severe disease
based on mucosal appearance.
The key efficacy endpoints of the study were met at week 8:
- 67% of patients experienced a clinical response in each
of the 2mg dose and 8mg dose cohorts;
- 44% of patients experienced a clinical remission in the 8mg
dose and 11% in the 2mg dose for an overall average of 28%.
Clinical response at week 8 is defined as a decrease in the Mayo
score of at least 3 points and either a decrease in the sub-score
for rectal bleeding of at least 1 point from baseline, or rectal
bleeding sub-score of 0 or 1. Clinical remission at week 8 is
defined as clinically symptom free, a Mayo score ≤ 2, with no
individual sub-score exceeding 1 point after treatment.
In addition to clinical response and remission, efficacy was
also observed in mucosal healing, an important prognostic parameter
in ulcerative colitis and other inflammatory bowel diseases,
measured by endoscopy:
- 61% of patients experienced mucosal improvement; and
- 33% of patients experienced mucosal healing.
Mucosal improvement is defined as a decrease in endoscopy
sub-score at week 8. Mucosal healing is defined as a
reduction in, and achievement of, endoscopy sub-score ≤1 at week
8.
Other key efficacy endpoints were also achieved, as follows:
- 72% of patients showed an improvement in rectal bleeding
scores;
- 72% of patients demonstrated an improvement in fecal
calprotectin; and
- 61% of patients showed improved Geboes score (a
histopathological scoring for the assessment of disease activity in
ulcerative colitis).
The positive trend in efficacy is consistent in substantially
all patients. This trend is demonstrated by 89% of the
patients having showed an improvement in Mayo score in both doses,
with an average decrease in Mayo score of 46% at week 8 in the 8mg
dose and 40% in the 2mg dose. In addition, all of the
patients also showed an improvement in at least one of the other
efficacy parameters.
No anti-drug antibodies were detected. In addition, no
systemic absorption was observed. OPRX-106 was safe and well
tolerated with only mild to moderate adverse events, which were
transient in nature. Headaches were the most common adverse
event reported.
“The full set of data from the study is very compelling, and
suggests that OPRX-106 is an effective anti-inflammatory agent.
OPRX-106 is delivered orally and is biologically active in
the gut without triggering the formation of anti-drug or systemic
absorption. OPRX-106 has the potential to address the loss of
response and to avoid critical safety concerns of infections and
malignancies currently seen in anti–TNF alpha treatment, which is
driven by the high presence of neutralizing antibodies and systemic
absorption respectively,” said Professor Yaron Ilan, Chairman of
the Department of Medicine, and an expert in Gastroenterology, at
The Hadassah Hebrew University Medical Center in Jerusalem, and a
consultant to the Company. “I am looking forward to
participating in additional trials of OPRX-106 and to continue to
see this program progress as it offers new hope for patients with
ulcerative colitis by addressing significant unmet medical needs of
patients.”
“We are very pleased by these excellent results, particularly
with respect to the significant percent of patients demonstrating
mucosal healing and clinical remission at eight weeks,” commented
Moshe Manor, Protalix’s President and Chief Executive
Officer. “OPRX-106 may bring a new therapy paradigm in the
ulcerative colitis space potentially offering better safety, longer
term efficacy and oral administration in place of most therapies
which are administered via injection and infusion and bear serious
safety concerns.”
The presentation made at the DDW 2018 Annual Meeting is
available on the Company’s website.
About Protalix BioTherapeutics, Inc.
Protalix is a biopharmaceutical company focused on the
development and commercialization of recombinant therapeutic
proteins expressed through its proprietary plant cell-based
expression system, ProCellEx®. Protalix’s unique expression
system presents a proprietary method for developing recombinant
proteins in a cost-effective, industrial-scale manner.
Protalix’s first product manufactured by ProCellEx, taliglucerase
alfa, was approved for marketing by the U.S. Food and Drug
Administration (FDA) in May 2012 and, subsequently, by the
regulatory authorities of other countries. Protalix has
licensed to Pfizer Inc. the worldwide development and
commercialization rights for taliglucerase alfa, excluding Brazil,
where Protalix retains full rights. Protalix’s development
pipeline includes the following product candidates: pegunigalsidase
alfa, a modified version of the recombinant human alpha-GAL-A
protein for the treatment of Fabry disease; OPRX-106, an
orally-delivered anti-inflammatory treatment; alidornase alfa for
the treatment of Cystic Fibrosis; and others. Protalix has
entered into an ex-United States partnership with Chiesi
Farmaceutici S.p.A. for the development and commercialization of
pegunigalsidase alfa. Protalix maintains full rights to
pegunigalsidase alfa in the United States.
Forward-Looking Statements
To the extent that statements in this press release are not
strictly historical, all such statements are forward-looking, and
are made pursuant to the safe-harbor provisions of the Private
Securities Litigation Reform Act of 1995. The terms
“anticipate,” “believe,” “estimate,” “expect,” “plan” and “intend”
and other words or phrases of similar import are intended to
identify forward-looking statements. These forward-looking
statements are subject to known and unknown risks and uncertainties
that may cause actual future experience and results to differ
materially from the statements made. These statements are
based on our current beliefs and expectations as to such future
outcomes. Drug discovery and development involve a high
degree of risk. Factors that might cause material differences
include, among others: failure or delay in the commencement or
completion of our preclinical and clinical trials which may be
caused by several factors, including: slower than expected rates of
patient recruitment; unforeseen safety issues; determination of
dosing issues; lack of effectiveness during clinical trials;
inability to monitor patients adequately during or after treatment;
inability or unwillingness of medical investigators and
institutional review boards to follow our clinical protocols; and
lack of sufficient funding to finance clinical trials; the risk
that the results of the clinical trials of our product candidates
will not support our claims of superiority, safety or efficacy,
that our product candidates will not have the desired effects or
will be associated with undesirable side effects or other
unexpected characteristics; risks related to the amount and
sufficiency of our cash and cash equivalents; risks related to the
amount of our future revenues, operations and expenditures; risks
relating to our ability to make scheduled payments of the principal
of, to pay interest on or to refinance our outstanding notes or any
other indebtedness; our dependence on performance by third party
providers of services and supplies, including without limitation,
clinical trial services; the inherent risks and uncertainties in
developing drug platforms and products of the type we are
developing; the impact of development of competing therapies and/or
technologies by other companies and institutions; potential product
liability risks, and risks of securing adequate levels of product
liability and other necessary insurance coverage; and other factors
described in our filings with the U.S. Securities and Exchange
Commission. The statements in this press release are valid
only as of the date hereof and we disclaim any obligation to update
this information, except as may be required by law.
Investor Contact
Marcy NanusSolebury Trout
Group646-378-2927mnanus@troutgroup.com
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