By Ransdell Pierson
March 10 (Reuters) – German health regulators have granted
Northwest Biotherapeutics Inc special permission to
sell its experimental brain cancer drug, DCVax-L, in the
country, even though the small U.S. biotechnology company has
not yet completed its late-stage trial of the immunotherapy.
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.
“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)
