The tiny U.S. biotech received a special "hospital
exemption" in Germany, allowing Northwest to sell the injectable
drug for five years even though it has not completed its late-stage
trial of the immunotherapy, Chief Executive Officer Linda Powers
said in an interview. She said the company, which would also have
the right to seek renewal of the exemption after five years, has not
yet requested or received formal marketing approval for its product.
Even before Monday's announcement, Northwest Biotherapeutics shares
had surged 60 percent in 2014. Wall Street is closely watching the
company, which has reported promising results for its DCVax-L drug
in a tiny cadre of patients. But skeptics have questioned whether
they will be borne out in a larger population.
Northwest has attracted the interest of large institutional players
such as Goldman Sachs, while some investors have taken short
positions in the stock, at around 9 percent of its shares as of
The exemption in Germany allows Northwest to sell DCVax-L through
hospitals and their outpatient clinics for patients with all
severities of cancer that begin in 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 typically seen with
patients taking standard treatment alone.
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.
Interim data from a late-stage trial of DCVax-L had been expected
last month, but has not yet become available, causing concern among
investors. The company declined to say when it will provide the
interim data on the drug's safety and effectiveness. On Friday, it
said an independent data safety monitoring board had recommended the
trial continue, based on its safety review.
At least 3,000 new cases of GBM are seen in Germany each year,
Powers estimated, as well as another 4,000 cases of 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.
Separately, Germany'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 have applied to be eligible for reimbursement for the
product, anticipating the hospital exemption.
Powers, through different investment entities that include venture
capital firm Toucan Partners LLC and drug manufacturer Cognate
BioServices Inc, owns 33 percent of Northwest Biotherapeutics' 51
million shares. Other major shareholders include Franklin Advisers
Inc, The Vanguard Group and Goldman Sachs.
THE CASE FOR A PREMIUM PRICE
Northwest Biotherapeutics is pushing ahead following two
high-profile disappointments of dendritic cell-based therapies from
ImmunoCellular Therapeutics Ltd and Dendreon Corp.
[to top of second column]
Industry analysts say DCVax-L 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.
Germany's 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.
"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," she said.
DCVax-L initially could be priced higher than Temodar, which costs
about $65,000 to $70,000 a year. Powers said the price for DCVax-L
was warranted because of signs of effectiveness seen in the informal
"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
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
(For a graphic, click:
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," Powers said.
At least 12,000 patients a year develop GBM in the United States,
along with a similar number in Europe, Powers said.
Shares of Northwest Biotherapeutics on Monday closed up $1.79, or 30
percent, on the Nasdaq.
(Reporting by Ransdell Pierson; editing
by Michele Gershberg, Jeffrey Benkoe and David Gregorio)
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