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 By 2007, that backer, Linda Powers, had become
chairman of the Maryland-based company. A venture capitalist, Powers
remains one of its largest shareholders and has invested more than
$20 million in the company and its experimental drugs for brain
cancer and inoperable tumors.
"Biotech is not for the faint of heart," Powers said in an
interview. "Through two major recessions and pummeling in the
capital markets, we've chosen to keep our focus."
Now worth close to $300 million, Northwest's stock has jumped 75
percent this year to the $7 range. Institutional investors include
Goldman Sachs and Franklin Advisers Inc, and more industry analysts
are taking notice.
But many other investors are betting against the company,
highlighting the uncertain, high-risk nature of developing drugs for
hard-to-treat diseases.
The key to Northwest's survival has been delivering strong trial
results in a tiny cadre of patients for its brain-cancer drug,
called DCVax-L. A similar drug called DCVax-Direct has begun testing
for a wide array of solid tumors that cannot be removed by surgery,
including cancers of the colon, pancreas, breast and melanoma.
They are among an emerging new crop of drugs that coax the body's
own immune system into tracking down and killing cancer cells. More
specifically they harness dendritic cells, master cells of the
immune system that give marching orders to its soldiers, including
t-cells and b-cells, which make antibodies.
Northwest Biotherapeutics' entry into the high-stakes field follows
two high-profile disappointments of dendritic cell-based therapies
from ImmunoCellular Therapeutics Ltd and Dendreon Corp.
Powers, chief executive since 2011, is making the case to investors
that the future of its drugs looks far different.
"If (DCVax-L) has a positive result, it could be the new standard of
care for brain cancer and have potential for hundreds of millions of
dollars in sales for newly diagnosed patients," said Carol Werther,
a biotech analyst for Summer Street Research. "And if it works
against other solid tumors, it could be a multibillion-dollar
product."
The company points to early-stage trial data that shows two
brain-cancer patients surviving the disease for a decade after
taking DCVax-L. Others have remained alive for five years or longer,
with virtually no significant side effects.
But Werther cautioned that DCVax-L is a financially "high risk and
speculative product," as investors await data from a late-stage
clinical trial of the drug that involves far more patients and
represents the first formal randomized trial of the medicine.
BOOST FROM A LAB DISH BREW
A graduate of Harvard Law School who was a lead negotiator for the
United States on the NAFTA trade agreement, Powers has become a
self-taught expert in promising new cell-based drugs. She holds
major stakes in more than a dozen biotech companies through several
venture capital funds, including Toucan Partners LLC. But DCVax-L is
her main focus.
The treatment consists of an individual patient's immature dendritic
cells, which are 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, see: http://link.reuters.com/cex86v
In small early trials involving 20 newly diagnosed brain-cancer
patients, those taking DCVax-L lived three years on average, more
than twice the 14.6 months such patients typically live on average
with standard treatment.
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Data from the company's far-larger Phase III brain cancer trial
is due in the first half of 2015. Treatment with the drug will be on
top of standard care, and the main goal is to assess whether DCVax-L
can delay regrowth of the tumor.
But the industry is littered with drugs that crashed after initially
looking like winners. A promising brain-cancer drug from
ImmunoCellular failed its primary goal of significantly prolonging
survival in a midstage trial. Company shares plunged 63 percent on
the setback in December.
"Nobody knows what will happen to them as a company," Powers said of
ImmunoCellular. Northwest's early trials show a more impressive
survival benefit for DCVax-L, she said, which she believes will bode
well for its larger studies.
Northwest Biotherapeutics has also attracted the attention of
investors taking short positions in the stock, betting it will
suffer like other companies that have used dendritic cell
technology. As of January 31, 4.4 million shares were held short,
almost 9 percent of company stock.
Boris Peaker, an analyst with Oppenheimer & Co, noted the ongoing
Phase III trial has the involvement of many more doctors and a more
genetically diverse population of patients.
"If the drug holds up in Phase III, it will be a very big win for
the company because there aren't many good options now for patients,
and DCVax is certainly better tolerated than any chemotherapy,"
Peaker said. "And if it works in brain cancer, it should work in
other solid tumors."
DATA IN MAY?
Early-stage trials of a sister drug, DCVax-Direct, are targeting a
wide range of other solid tumors so advanced or inaccessible that
they are deemed inoperable.
The trials of DCVax-Direct involve 60 U.S. patients. The Phase 1
stage will focus primarily on safety, although hints of efficacy
could emerge. The primary goal of the Phase II part of the study is
to shrink or eliminate tumors.
Dr. Vivek Subbiah, an oncologist at M.D. Anderson Cancer Center in
Houston who is leading the DCVax-Direct trials, said he may present
initial data at the annual meeting of the American Society of
Clinical Oncology that begins May 30.
Subbiah and his colleagues test scores of experimental cancer drugs,
many having new mechanisms of action.
"This is not a 'me-too' trial," Subbiah said. "It is one of the
interesting technologies. Twenty years ago it was chemo, 10 years
ago it was targeted therapies, and for this decade it will be drugs
that boost the immune system."
(Reporting by Ransdell Pierson; Editing
by Michele Gershberg and Prudence Crowther)
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