Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX), an oncology company
developing innovative medicines and other products for targeting
and treating cancer, today announced that additional clinical data
from the Company’s pivotal Phase 2b trial of its novel
radiotherapeutic candidate, AZEDRA® (iobenguane I 131), in patients
with malignant, recurrent, or unresectable pheochromocytoma and
paraganglioma (pheo/para) will be presented at the 5th
International Symposium on Pheochromocytoma and Paraganglioma in
Sydney, Australia. AZEDRA has not been approved for use in the
United States.
“The positive outcomes from this study, which is
the largest prospective clinical trial in pheo/para to date,
indicate the clinical benefit and anti-tumor effects of AZEDRA,”
said Dr. Daniel Pryma, Associate Professor of Radiology &
Radiation Oncology and Chief, Division of Nuclear Medicine &
Clinical Molecular Imaging at the Perelman School of Medicine at
the University of Pennsylvania, the trial’s lead investigator. “A
meaningful proportion of patients treated in this study with AZEDRA
achieved a sustained reduction of antihypertensive medications
which was correlated with favorable tumor responses, including
radiographic tumor responses, tumor biomarker response, and overall
survival. The clinical response and antitumor effects observed in
this heavily pre-treated study population, along with the
acceptable adverse event profile from this trial, provide a strong
rationale for the potential use of AZEDRA.”
Dr. Pryma will review the data in an oral
presentation entitled “AZEDRA® (iobenguane I 131) in Patients with
Malignant and/or Recurrent and/or Unresectable Pheochromocytoma or
Paraganglioma: Final Results of a Multi-Center, Open-Label, Pivotal
Phase 2b Study.”
Trial Design and Patient Baseline
Characteristics
The Phase 2b study was conducted under a Special
Protocol Assessment (SPA) agreement with the Food and Drug
Administration (FDA). The trial was designed to evaluate the
efficacy and safety of AZEDRA in patients with malignant,
recurrent, and/or unresectable pheochromocytoma or paraganglioma,
which are rare neuroendocrine tumors.
A majority of patients enrolled in this study
(68.9%) have had two (41.9%) or more (27.0%) prior treatments,
these include surgery (89.2%), conventional MIBG I-131 (30%), and
chemotherapy (37.8%). The most common locations of metastases were
the lymph nodes (54.1%), bone (52.7%), lung and/or liver
(50.0%).
Primary Endpoint: Reduction in Antihypertensive
Medications
The study met the primary endpoint, with 17
(25%) of the 68 evaluable patients experiencing a 50% or greater
reduction of all antihypertensive medication for at least 6 months.
The lower limit of the 95% confidence interval was 16.15%, and the
upper limit of the 95% confidence interval was 36.52%. The
reduction of antihypertensive medication was sustained, with a
median duration of clinical benefit of 13.3 months, ranging from
8.0 to 60.2 months. In patients who received two therapeutic doses,
clinical benefit was achieved in 32%, compared to 5.6% of patients
who received only one therapeutic dose.
31.4% of study patients who were not considered
as responders for the primary endpoint also experienced ≥50%
reduction of antihypertensive medication(s), albeit for <6
months, suggesting that there was a continuum of this clinical
benefit. Secondary
Endpoints: Tumor Response Data
Treatment with AZEDRA produced favorable data
for a key secondary endpoint, the proportion of study patients with
overall tumor response as measured by Response Evaluation Criteria
In Solid Tumors (RECIST) criteria. Of the 64 patients with
evaluable scans, confirmed partial response (PR) was achieved in
23.4% of patients (30.0% of patients receiving two doses; 0% of
patients receiving one dose). Stable disease (SD) was achieved in
68.8% of all patients (68.0% of patients receiving two doses; 71.4%
of patients receiving one dose). Overall, 92.2% of patients
achieved tumor response of confirmed PR or SD. Persistence of
antitumor effects were also evident. The proportion of study
patients who experienced PR increased over time (5.9% at 3 months,
to 23.5% at 12 months).
All primary endpoint responders achieved tumor
response of PR/SD and 89.3% of primary endpoint
non-responders also achieved tumor response of PR/SD, suggesting
the potential of AZEDRA to confer multiple clinical benefits to
pheo/para patients.
Tumor biomarkers were analyzed in patients that
had individual tumor biomarkers above 1.5 x ULN at baseline. At 12
months following the first therapeutic dose of AZEDRA, urine and
blood norepinephrine showed best response (CR/PR) rates of 42.1%
and 31.0%, respectively, and urine and blood normetanephrine
responder rates of 36.0% and 44.0%, respectively. In
addition, serum CgA showed best response rate of 67.9%.
Overall Survival
Median survival time as of March 10, 2017 was
36.7 months (95% CI 29.9 – 49.1) from first AZEDRA therapeutic
dosing in the overall study population, and 48.73 months among
patients who received two therapeutic doses, compared to 17.42
months among patients who received only one therapeutic dose. The
Kaplan-Meier estimates of survival were 91.0%, 66.8%, and 51.5% at
1, 2, and 3 years, respectively, following initial therapeutic
dosing. Long term follow-up continues.
The study data also suggest the potential for
AZEDRA to extend survival in patients with liver or lung
metastasis, which is generally considered in the literature to be
less than 24 months. In this study, median survival time was
similar in patients with lung or liver metastasis compared to those
without (42.55 vs. 41.09 months).
Safety and Tolerability Profile
This study showed AZEDRA to be generally well
tolerated. The most common treatment emergent adverse events were
nausea, thrombocytopenia, anemia, fatigue, leukopenia, and
neutropenia. No severe acute hypertension or hypertensive
crises were observed in study patients during or immediately
following AZEDRA infusion. These events are consistent with those
observed in prior AZEDRA studies.
“These results are entirely consistent with the
topline data announced earlier this year and further demonstrate
the profound significant clinical benefit that study patients with
pheo/para achieved with AZEDRA,” said Mark Baker, Chief Executive
Officer of Progenics. “We are working to complete our rolling New
Drug Application for AZEDRA to the FDA, and if our filing is
approved, AZEDRA would be the first therapy for these rare tumors
approved in the U.S.”
About AZEDRA®
AZEDRA® (iobenguane I-131), a radiotherapeutic
product candidate in development as a treatment for malignant
and/or recurrent pheochromocytoma and paraganglioma, rare tumors
found in the adrenal glands and outside of the adrenal glands,
respectively. AZEDRA has been granted Breakthrough Therapy and
Orphan Drug designations, as well as Fast Track status in the U.S.
Under a SPA agreement with the U.S. Food and Drug Administration
(FDA), a Phase 2 pivotal study has been completed in patients with
malignant and/or recurrent pheochromocytoma and
paraganglioma. There are currently no FDA-approved therapies
for the treatment of these ultra-orphan diseases.
About Pheochromocytoma and
Paraganglioma
Pheochromocytoma and paraganglioma are rare
neuroendocrine tumors that arise from cells of the sympathetic
nervous system. When pheochromocytomas are located outside the
adrenal glands, they are called paragangliomas. Standard treatment
options for these tumors include surgery, palliative therapy and
symptom management. Pheochromocytoma and paraganglioma tumors
frequently secret high levels of hormones that can lead to life
threatening hypertension, heart failure, and stroke in these
patients. Malignant and recurrent pheochromocytoma and
paraganglioma may result in unresectable disease with a poor
prognosis, representing a significant management challenge with
very limited treatment options and no approved anti-tumor
therapies.
About Progenics
Progenics develops innovative medicines and
other technologies to target and treat cancer. Progenics' pipeline
includes: 1) therapeutic agents designed to precisely target cancer
(AZEDRA® and 1095), 2) PSMA-targeted imaging agents for prostate
cancer (1404 and PyL™), and 3) imaging analysis tools. Progenics'
first commercial product, RELISTOR® (methylnaltrexone bromide) for
OIC, is partnered with Valeant Pharmaceuticals International,
Inc.
This press release may contain projections and
other "forward-looking statements" regarding future events.
Statements contained in this communication that refer to Progenics'
estimated or anticipated future results or other non-historical
facts are forward-looking statements that reflect Progenics'
current perspective of existing trends and information as of the
date of this communication. Forward looking statements generally
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"outlook," "guidance," "intend," "may," "might," "will,"
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words, phrases or expressions. Such statements are predictions
only, and are subject to risks and uncertainties that could cause
actual events or results to differ materially. These risks and
uncertainties include, among others, the cost, timing and
unpredictability of results of clinical trials and other
development activities and collaborations, including our clinical
trials for AZEDRA and our other product candidates such as our
collaboration with Valeant on the RELISTOR oral formulation and the
Phase 3 clinical program for 1404; our ability to successfully
integrate EXINI Diagnostics AB and to develop and commercialize its
products; the unpredictability of the duration and results of
regulatory review of New Drug Applications and Investigational
NDAs, including our NDA for AZEDRA; market acceptance for approved
products; the effectiveness of the efforts of our partners to
market and sell products on which we collaborate and the royalty
revenue generated thereby; generic and other competition; the
possible impairment of, inability to obtain and costs of obtaining
intellectual property rights; possible product safety or efficacy
concerns, general business, financial, regulatory and accounting
matters, litigation and other risks. More information concerning
Progenics and such risks and uncertainties is available on its
website, and in its press releases and reports it files with the
U.S. Securities and Exchange Commission, including in its Quarterly
Report on Form 10-Q for the period ending June 30, 2017. Progenics
is providing the information in this press release as of its date
and, except as expressly required by law, Progenics disclaims any
intent or obligation to update or revise any forward-looking
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or circumstances or otherwise.
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(PGNX-F)
Contact:
Melissa Downs
Investor Relations
(646) 975-2533
mdowns@progenics.com
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