Multiple Sclerosis News
Today
Today
Opexa Developing Tcelna as a
True Personalized MS Therapy For SPMS, RRMS
True Personalized MS Therapy For SPMS, RRMS
Maureen Newman
After the Phase 2b clinical trial
named Abili-T achieved full enrollment with 190 secondary progress multiple
sclerosis (SPMS) patients, The Woodlands, Texas-based Opexa Therapeutics is
patiently awaiting results for the company’s lead candidate, Tcelna, which are
expected in the second half of 2016. The therapy, which was spun off from a
technology developed at Baylor College of Medicine (BCM) Technologies that was
licensed and developed by Opexa, has shown efficacy in both SPMS and
relapsing-remitting multiple sclerosis (RRMS) patients in earlier clinical
trials.
named Abili-T achieved full enrollment with 190 secondary progress multiple
sclerosis (SPMS) patients, The Woodlands, Texas-based Opexa Therapeutics is
patiently awaiting results for the company’s lead candidate, Tcelna, which are
expected in the second half of 2016. The therapy, which was spun off from a
technology developed at Baylor College of Medicine (BCM) Technologies that was
licensed and developed by Opexa, has shown efficacy in both SPMS and
relapsing-remitting multiple sclerosis (RRMS) patients in earlier clinical
trials.
We have spent a good decade in
clinical trials treating early- and late-stage multiple sclerosis,” said Neil
Warma, President and CEO of Opexa Therapeutics, in an exclusive interview with
Multiple Sclerosis News Today. “In true fashion, we are trying to restore
function of the immune system via personalized therapies that fight the root
cause of disease itself.”
clinical trials treating early- and late-stage multiple sclerosis,” said Neil
Warma, President and CEO of Opexa Therapeutics, in an exclusive interview with
Multiple Sclerosis News Today. “In true fashion, we are trying to restore
function of the immune system via personalized therapies that fight the root
cause of disease itself.”
The focus of Tcelna is the
interaction between T-cells and the rest of the body. “It is similar in concept
to a T-cell vaccine, but these ‘vaccines’ are personalized for patients with
multiple sclerosis,” explained Warma. “In patients with multiple sclerosis,
there is a small population of myelin reactive T-cells (MRTCs) that cross into
the central nervous system and attack nerve fibers. We isolate and identify
those pathogenic T-cells, attenuate them, and reintroduce them back into the
individual.”
interaction between T-cells and the rest of the body. “It is similar in concept
to a T-cell vaccine, but these ‘vaccines’ are personalized for patients with
multiple sclerosis,” explained Warma. “In patients with multiple sclerosis,
there is a small population of myelin reactive T-cells (MRTCs) that cross into
the central nervous system and attack nerve fibers. We isolate and identify
those pathogenic T-cells, attenuate them, and reintroduce them back into the
individual.”
First, a sample of blood is taken
from a patient and sent to Opexa’s Good Manufacturing Practices (GMP) facility
in Houston. Scientists then identify the specific myelin peptide antigens that
the patient’s MRTCs recognize. Out of 109 peptides screened by Opexa, typically
three or four peptides are dominant, and are introduced to a population of
expanding T-cells. After a few more manufacturing steps to attenuate the
reactive T-cells, these are then shipped to the patient’s physician’s office,
who receives a subcutaneous injection of the “T-cell vaccine.”
from a patient and sent to Opexa’s Good Manufacturing Practices (GMP) facility
in Houston. Scientists then identify the specific myelin peptide antigens that
the patient’s MRTCs recognize. Out of 109 peptides screened by Opexa, typically
three or four peptides are dominant, and are introduced to a population of
expanding T-cells. After a few more manufacturing steps to attenuate the
reactive T-cells, these are then shipped to the patient’s physician’s office,
who receives a subcutaneous injection of the “T-cell vaccine.”
“The injection primes the body to
selectively recognize and attack the MRTCs,” said Warma. “It’s telling the
immune system that these attenuated cells should be treated as foreign
attackers.” Although the injection delivers a bolus of MRTCs, the cells are
attenuated, meaning that they pose no risk of further destroying myelin.
Instead, the cells die off and remain in situ long enough for the patient’s
immune system to recognize the cells as damaging and harmful. With a heightened
immune system, the patient’s own healthy T-regulatory cells search for MRTCs to
remove them from the body in a sort of “anti-T-cell T-cell response.”
selectively recognize and attack the MRTCs,” said Warma. “It’s telling the
immune system that these attenuated cells should be treated as foreign
attackers.” Although the injection delivers a bolus of MRTCs, the cells are
attenuated, meaning that they pose no risk of further destroying myelin.
Instead, the cells die off and remain in situ long enough for the patient’s
immune system to recognize the cells as damaging and harmful. With a heightened
immune system, the patient’s own healthy T-regulatory cells search for MRTCs to
remove them from the body in a sort of “anti-T-cell T-cell response.”
Many facets of this process
differentiate Opexa’s therapy from other treatments for multiple sclerosis.
Traditional therapies wipe out the entire immune system and target all T-cells
and other immune cells for destruction. Tcelna targets a select population of
MRTCs, giving the treatment a better side effect profile and greater patient
tolerability.
differentiate Opexa’s therapy from other treatments for multiple sclerosis.
Traditional therapies wipe out the entire immune system and target all T-cells
and other immune cells for destruction. Tcelna targets a select population of
MRTCs, giving the treatment a better side effect profile and greater patient
tolerability.
In contrast to the pharmaceutical
definition of personalized medicine, Tcelna is truly a personalized medicine. “Pharma
looks at ‘personalized’ differently,” commented Warma. “They have a drug
candidate that is effective in 10-20% of patients, and they segment the market
to serve those patients. At Opexa, we look at all multiple sclerosis patients
and find a personalized therapy for every individual.”
definition of personalized medicine, Tcelna is truly a personalized medicine. “Pharma
looks at ‘personalized’ differently,” commented Warma. “They have a drug
candidate that is effective in 10-20% of patients, and they segment the market
to serve those patients. At Opexa, we look at all multiple sclerosis patients
and find a personalized therapy for every individual.”
Personalization of treatment
continues past the first dose of treatment. The optimal dosing schedule was
found to be five subcutaneous injections each year, given in the first six
months. An injection is given at one, two, three, four, and six months. In the
next year, the injection pattern is identical, but patients receive a different
formulation of T-cells. “Each of our preparations is tailored specifically to
match each individual’s profile. Each patient receives a specialized therapy,”
said Warma. “Myelin peptide antigens change over time and are different for
every individual. In year two, three, and beyond, we conduct the antigen
screening process because we know these myelin peptides shift over time. We
deliver a specific T-cell vaccine to each individual, relevant at that time.”
continues past the first dose of treatment. The optimal dosing schedule was
found to be five subcutaneous injections each year, given in the first six
months. An injection is given at one, two, three, four, and six months. In the
next year, the injection pattern is identical, but patients receive a different
formulation of T-cells. “Each of our preparations is tailored specifically to
match each individual’s profile. Each patient receives a specialized therapy,”
said Warma. “Myelin peptide antigens change over time and are different for
every individual. In year two, three, and beyond, we conduct the antigen
screening process because we know these myelin peptides shift over time. We
deliver a specific T-cell vaccine to each individual, relevant at that time.”
According to Opexa, clinical data
so far showed a 37% reduction in annualized relapse rate in patients with RRMS.
A sub-population of studied patients with a more progressed/active disease
profile saw a statistically significant improvement in disability measured by
the Expanded Disability Status Scale (EDSS) score after a year of treatment. In
SPMS patients, 80% of patients showed disease stabilization after two years
according to the EDSS.
so far showed a 37% reduction in annualized relapse rate in patients with RRMS.
A sub-population of studied patients with a more progressed/active disease
profile saw a statistically significant improvement in disability measured by
the Expanded Disability Status Scale (EDSS) score after a year of treatment. In
SPMS patients, 80% of patients showed disease stabilization after two years
according to the EDSS.
The reasons for improvement are
two-fold. First, Tcelna stops destruction of the axonal myelin sheath by
depleting the body of MRTCs. Second, Tcelna stops damage to oligodendrocytes,
which are the producers of myelin in the central nervous system. “We expect to
see stabilization, but we also expect there to be a remyelination process to
take place, as MRTCs are no longer destroying the engine that builds myelin,”
said Warma. “Clinical data support the hypothesis that preventing destruction
of the myelin sheath and oligodendrocytes results in repair. We saw signs of
repair, with a clean side effect profile.”
two-fold. First, Tcelna stops destruction of the axonal myelin sheath by
depleting the body of MRTCs. Second, Tcelna stops damage to oligodendrocytes,
which are the producers of myelin in the central nervous system. “We expect to
see stabilization, but we also expect there to be a remyelination process to
take place, as MRTCs are no longer destroying the engine that builds myelin,”
said Warma. “Clinical data support the hypothesis that preventing destruction
of the myelin sheath and oligodendrocytes results in repair. We saw signs of
repair, with a clean side effect profile.”
With safety and efficacy bundled
together in a personalized T-cell vaccine, Tcelna may be just what the doctor
ordered if approved by the Food and Drug Administration for patients with
multiple sclerosis.
together in a personalized T-cell vaccine, Tcelna may be just what the doctor
ordered if approved by the Food and Drug Administration for patients with
multiple sclerosis.
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