CLINICAL NEWS
The FDA noted that a drug’s appearance
on a quarterly FAERS watch list does not
mean that the agency has determined a
causal relationship between that product
and the AE, but it does indicate the need
for further study. If the FDA determines
that there is a causal link between the
product and the AE, the agency can
collect more data to better describe the
risk, change the drug’s label, order a risk
evaluation and mitigation strategy, or
pull the product from the market.
“Time is critical
for ... seriously
ill patients who
do not have
alternative thera-
pies and who
cannot take part
in a clinical trial.” FDA Grants Orphan-
Drug Designation to
—SCOTT GOTTLIEB, MD
PCM-075 for AML
Source: U.S. Food and Drug Administration watch list, October 6, 2017.
ensure the safety of blood in the U.S. and
its territories.”
In August 2016, the FDA issued a
final guidance document recommending
that states and territories screen blood
donations for the Zika virus using a
screening test under an investigational
new drug (IND) application or an ap-
proved test when available. Prior to that
approval, several blood-collection estab-
lishments used the cobas test under IND;
collected data demonstrated its efficacy
in detecting the virus.
Source: U.S. Food and Drug Administration press release, October
5, 2017.
Several Anti-Cancer
Drugs on Updated
FDA “Watch List”
The FDA released a watch list that in-
cludes 19 drugs or drug classes, seven of
which are cancer therapies, which may
have serious risks or have new safety
information. The list is derived from
FAERS.
Drugs relevant to hematology and
oncology and their associated risks
include:
• alemtuzumab for acute acalculous
cholecystitis
• imatinib mesylate for decline in
renal function
• pembrolizumab for Stevens-Johnson
syndrome and toxic epidermal
necrolysis and complications of al-
logeneic hematopoietic cell trans-
plantation (alloHCT)
• nivolumab for complications of
alloHCT
• pomalidomide for ischemic colitis
ASHClinicalNews.org
The FDA granted orphan-drug designa-
tion to PCM-075 for the treatment of
acute myeloid leukemia (AML). PCM-075
is an oral, highly selective adenosine
triphosphate competitive inhibitor of
the serine/threonine Polo-like Kinase 1
enzyme.
The drug’s manufacturers are plan-
ning an open-label, phase Ib/II trial to
evaluate the safety and anti-leukemic
activity of PCM-075 in combination with
standard of care for patients with AML
at 10 U.S. research sites. The study, which
is not yet recruiting, will determine
whether PCM-075 is safe and tolerable
in adult patients with relapsed/refractory
AML when administered orally for five
consecutive days every 28 days. The
study will also determine the maximum
tolerated or recommended dose of PCM-075
for phase II testing in combination with
decitabine and/or low-dose cytarabine.
The projected study completion date is
mid-2020.
more high-quality trials to be conducted.
The 11 partner organizations will
contribute up to $1 million per year
for five years for a total private-sector
contribution of up to $55 million; NIH
will contribute $160 million during the
five-year partnership – pending availabil-
ity of funds – for a total of $215 million
in funding.
“We have seen dramatic responses from
immunotherapy, often eradicating cancer
completely for some patients,” said NIH
Director Francis S. Collins, MD, PhD.
“We need to bring that kind of success –
and hope – to more people and more types
of cancers, and we need to do it quickly. A
systematic approach like PACT will help us
to achieve success faster.”
The Foundation for the NIH will
manage the program, and the FDA will
provide an advisory role. PACT partners
are AbbVie, Amgen, Boehringer Ingelheim,
Bristol-Myers Squibb, Celgene, Genentech,
Gilead Sciences, GlaxoSmithKline, Janssen
Pharmaceuticals, Novartis, and Pfizer.
Additional support has been provided by
Pharmaceutical Research and Manufac-
turers of America.
“We have
seen dramatic
responses from
immunotherapy.
... We need to
bring that kind
of success – and
hope – to more
people and more
NIH Announces
Program to Accelerate types of cancers.”
—FRANCIS S. COLLINS, MD, PhD
Development of
New Cancer
In addition, the National Cancer In-
Immunotherapies
stitute awarded cooperative agreements
Source: Trovagene, Inc., press release, October 9, 2017.
The National Institutes of Health (NIH)
joined with 11 biopharmaceutical
companies to launch the Partnership for
Accelerating Cancer Therapies (PACT), a
five-year, public-private research collabo-
ration, as part of the Cancer Moonshot
Initiative. PACT will initially focus on
identifying, developing, and validating
biomarkers to advance immunotherapy op-
tions. PACT will also facilitate information-
sharing among all stakeholders to better
coordinate clinical efforts, align investigative
approaches, reduce duplication, and enable
– totaling $53.6 million in funding over
five