difficulty swallowing, or who cannot swal-
low tablets,” said Gerald Sakowski, chief
executive officer, CMP Pharma. “Up until
now, these patients have been prescribed a
pharmacy compounded liquid form of spi-
ronolactone. The dosing inconsistencies of
compounded liquids have long been a per-
sistent challenge for physicians.”
CaroSpir will be introduced early in
the fourth quarter of 2017. Spironolactone
oral suspension is the only FDA-approved
oral liquid dosage form of its kind.
Jazz Pharmaceuticals received approv-
al from the FDA for Vyxeos (daunorubicin
and cytarabine) liposome for injection for
the treatment of adults with two types of
Acute Myeloid Leukemia (AML), a rapidly
progressing and life-threatening blood
cancer. Vyxeos is indicated for the treatment of adults with newly-diagnosed t-AML or AML-MRC.
Designed with Jazz’s CombiPlex technology, Vyxeos is a unique liposomal formulation that delivers a fixed-ratio of
daunorubicin and cytarabine to the bone
marrow that has been shown to have synergistic effects at killing leukemia cells in
vitro and in animal models. Vyxeos is the
first product developed with the company’s CombiPlex platform, which enables
the design and rapid evaluation of various
combinations of therapies.
The approval is based on Phase III trial
data that evaluated the efficacy and safety
of Vyxeos compared to cytarabine and
daunorubicin in 309 patients with newly
diagnosed t-AML or AML-MRC. For the
primary endpoint of overall survival, Vyxeos demonstrated an improvement that
was superior to the 7+ 3 treatment regimen. The median overall survival for the
Vyxeos treatment group was 9. 6 months
compared with 5. 9 months for the 7+ 3
treatment group. Vyxeos also demonstrated a statistically significant improvement
in complete response rate of 38 percent
versus 26 percent.
In the Phase III study, the most common adverse reactions were bleeding
events, fever, rash, swelling, nausea, sores
in the mouth or throat, diarrhea, constipation, muscle pain, tiredness, stomach pain,
difficulty breathing, headache, cough,
decreased appetite, irregular heartbeat,
pneumonia, blood infection, chills, sleep
disorders and vomiting.
Eli Lilly and Co.’s lasmiditan, an in-
vestigational, oral, first-in-class molecule
for the acute treatment of migraine, met
its primary endpoint in SPARTAN, a second Phase III study. At two hours following the first dose, a greater percentage
of patients treated with lasmiditan were
migraine pain-free compared to placebo.
These results were statistically significant
across all three studied doses ( 50 mg, 100
mg and 200 mg).
Lasmiditan also met the key secondary endpoint for SPARTAN across all three
studied doses, with a significantly greater
percentage of patients free of their most
bothersome symptom (MBS) compared
with placebo at two hours following the
first dose. In this study, patients chose
their MBS from nausea, sensitivity to
sound or sensitivity to light.
“Lasmiditan represents the first sig-
nificant innovation in the acute treatment
of migraine in more than 20 years, and
could provide a much-needed new treat-
ment option for the 36 million Americans
living with migraine,” said Christi Shaw,
president of Lilly Bio-Medicines. “We are
thrilled with these topline lasmiditan re-
sults, which add to more than 25 years of
Lilly’s research and development of mi-
The most commonly-reported adverse
events after lasmiditan dosing were diz-
ziness, paresthesia, somnolence, fatigue,
nausea and lethargy.
These findings are consistent with
SAMURAI, the first Phase III study evaluating the safety and efficacy of lasmiditan
for the acute treatment of migraine. Lilly
plans to submit a New Drug Application
for lasmiditan to the U.S. FDA in the second half of 2018.
Regeneron Pharmaceuticals, Inc. will
discontinue development Suptavumab
after a Phase III study evaluating suptavumab (REGN2222), an antibody to respiratory syncytial virus (RSV), did not meet
its primary endpoint of preventing medically-attended RSV infections in infants.
Suptavumab did show signs of efficacy
in a subgroup of patients. Adverse events
were generally balanced between suptavumab and placebo. Regeneron plans to
discontinue further clinical development
of this antibody.
“We are disappointed in these results,
as we had hoped suptavumab might offer
a new option for the thousands of infants
impacted by serious RSV infections every
year,” said George D. Yancopoulos, M.D.,
Ph.D., president and chief scientific offi-
cer of Regeneron.“Regeneron has a robust
pipeline across many serious diseases, and
we look forward to important data read-
outs from other programs in the coming
weeks and months.”
The double-blind, placebo-controlled
Phase III study enrolled 1,149 healthy pre-
term infants. Patients were randomized
to one of three study groups: suptavumab
30 mg/kg as a single dose; suptavumab 30
mg/kg administered as two doses 8 weeks
apart; or placebo. Patients were consid-
ered ‘medically-attended’ if they required
hospitalization and/or sought medical
care for a centrally-adjudicated RSV infec-
tion. Pre-term infants in the study had a
gestational age of less than 36 weeks and
were 6 months old or younger at the be-
ginning of the study.
Medivir AB began enrollment in the
company’s Phase I/II study of birinapant
in combination with the anti-PD-1 therapy KEYTRUDA (pembrolizumab), which
is marketed by Merck. The objectives of
the study are to evaluate the safety, tolerability and preliminary efficacy of this
combination in patients with treatment-resistant solid tumors.
The multicenter, single arm, open label
study, will be conducted in two parts. In
the initial dose escalation (Phase I) part of
the study, the objective is to identify the
recommended Phase II dose of birinapant
for use in combination with KEYTRUDA.
Once the recommended Phase II dose
has been identified, the second part of the
study will evaluate the safety and tolerability of birinapant in combination with
KEYTRUDA in several cohorts. Each cohort will be made up of patients with the
same treatment refractory tumor type.
An important secondary objective in the
Phase II part is the preliminary evaluation
of the efficacy of the combination in each
of the cohorts.
Merck will provide KEYTRUDA for this
study at no cost to Medivir. Medivir retains
full rights to birinapant. Additional details
were not disclosed. CP