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 The special "hospital exemption" in Germany would
allow Northwest to sell DCVax-L for five years, and to seek renewed
approval afterward, Northwest Biotherapeutics Chief Executive
Officer Linda Powers said in an interview. The company has not yet
requested or received formal marketing approval for its product.
The exemption allows Northwest to sell DCVax-L through hospitals and
their outpatient clinics to patients with all severities of cancer
that begin the brain, even though it is only being tested in
patients newly diagnosed with the most severe form of the disease,
called Glioblastoma multiforme (GBM).
It is the first hospital exemption in Germany for a product never
previously on the market, Powers said. She hopes it will encourage
other European nations to look favorably on DCVax-L.
The European Union has encouraged member countries to create the
hospital exemptions as a way of fostering use of breakthrough
biotechnology drugs that have not yet won formal marketing approval.
Patients taking DCVax-L in small informal trials, along with
standard care, lived 2.5 times as long as the typical patient taking
standard treatment alone.
Northwest Biotherapeutics's fortunes are already being closely
watched on Wall Street, where company shares have surged 60 percent
this year to the $6 range. The company has reported promising
results for the drug in a tiny cadre of patients, but skeptics
question whether they will be borne out in a larger population.
Interim data from a late-stage trial had been expected last month,
but has not yet become available, causing concern among investors.
The company said on Friday that an independent data safety
monitoring board had recommended the trial continue, based on its
safety review.
The German regulator, known as the Paul-Ehrlich-Institute (PEI),
said DCVax-L can be used after patients receive the standard care
for brain cancer, which consists of surgery, radiation and treatment
with Merck & Co Inc's Temodar chemotherapy.
At least 3,000 cases of GBM are seen in Germany each year, Powers
estimated. She added that many other patients have lower- grade
brain tumors that will also be eligible for treatment with DCVax-L.
Northwest Biotherapeutics plans to begin selling its drug in the
next few months in Germany, and to ramp up production at its
manufacturing plant in Leipzig, Powers said.
"IMPORTANT VALIDATION," HIGH PRICE
"This is an important validation" of DCVax-L, said Powers, who
described the German regulator as among the toughest in Europe.
"They realize we're still in our Phase III trial, but appear
satisfied with the underlying biology of DCVax-L and the glimpses of
effectiveness that were seen in early-stage trials."
DCVax-L initially could be priced higher than Temodar, which costs
about $65,000 to $70,000 a year. Powers said a premium price for
DCVax-L was warranted because of signs of effectiveness seen in the
informal early-stage trials.
The regulator apparently opened the door to DCVax-L because data
from the small earlier trials suggested advantages over standard
treatments that offer little help to patients, Powers said. Industry
analysts say the treatment could potentially be worth more than a
billion dollars in annual sales if it eventually wins full marketing
approval in Europe and the United States.
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"When you add Temodar to surgery and radiation, you get an additional
2.5 months of survival," Powers said, allowing the average patient
to survive 14.5 months. By contrast, the average patient taking DCVax-L plus
standard treatment in the smaller Phase I/II trial survived three
years, without additional serious side effects. But data from that
study, which involved 20 patients with newly diagnosed GBM, were not
considered statistically significant because of the small size and
informal nature of the trial.
DCVax-L is among an emerging new crop of drugs that coax the body's
own immune system to track down and kill cancer cells. More
specifically, it harnesses dendritic cells, or master cells of the
immune system that give marching orders to its soldiers, including
t-cells and b-cells, which make antibodies. The
treatment consists of an individual patient's immature dendritic
cells being drawn from the blood and mingled in a laboratory dish
with dozens of antigens, or proteins, from brain tumor tissue of the
patient obtained in surgery.
When purified and injected back into the patient, DCVax-L is meant
to prompt t-cells and b-cells to leave the lymph nodes and fan out
through the body, seeking and attacking cells having the target
antigens.
(For a graphic, click:
http://link.reuters.com/cex86v) Northwest
Biotherapeutics expects to report complete data from its formal
late-stage trial of DCVax-L in 2015, Powers said. The Phase III
study, which began in 2008 and is being conducted in Europe and the
United States, involves 312 patients who were newly diagnosed with
GBM and received standard therapies. "If we meet the goals of the
trial, we would plan to apply for marketing approval in Europe and
the United States simultaneously," Powers said.
Separately, German's centralized government reimbursement authority
has authorized Northwest Biotherapeutics to negotiate reimbursement
for DCVax-L, Powers said. She added that six major hospital centers
in Germany, anticipating the hospital exemption, have applied to be
eligible for reimbursement for the product.
At least 12,000 patients a year develop GBM in the United States,
along with a similar number in Europe, Powers said.
Northwest Biotherapeutics is pushing ahead despite two high-profile
disappointments of dendritic cell-based therapies from
ImmunoCellular Therapeutics Ltd and Dendreon Corp.
(Reporting by Ransdell Pierson; editing
by Jeffrey Benkoe)
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