- Novel, targeted radiotherapeutic for malignant
and/or recurrent pheochromocytoma and paraganglioma –
Progenics Pharmaceuticals, Inc. (Nasdaq:PGNX), an oncology company
developing innovative medicines and other products for targeting
and treating cancer, today announced that the Company’s
registrational Phase 2b trial of its novel radiotherapeutic
candidate, AZEDRA® (iobenguane I 131) Injection, has achieved its
primary endpoint. The open-label, multi-center 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 and/or
recurrent pheochromocytoma or paraganglioma, which are rare
neuroendocrine tumors. There are currently no approved therapeutics
in the U.S. for the treatment of malignant and/or recurrent
pheochromocytoma or paraganglioma. AZEDRA has received Breakthrough
Therapy, Orphan Drug and Fast Track designations from the FDA.
The study met the primary endpoint evaluating
the proportion of patients who achieved a 50% or greater reduction
of all antihypertensive medication for at least six months. Under
the study protocol, the primary endpoint is achieved if the lower
limit of the two-sided 95% confidence interval was above 10%.
In the trial, 17 (25%) of the 68 evaluable
patients experienced 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%, thus meeting the
primary endpoint. The upper limit of the 95% confidence
interval was 36.52%.
Progenics also reported favorable data from a
key secondary endpoint, the proportion of patients with overall
tumor response as measured by Response Evaluation Criteria In Solid
Tumors (RECIST) criteria. In this highly pre-treated patient
population, 92.2% of patients who received at least one AZEDRA
therapeutic dose achieved partial response or stable disease.
“The positive data from this trial are
clinically meaningful and provide compelling evidence for the use
of AZEDRA to treat malignant and/or recurrent pheochromocytoma and
paraganglioma,” 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. “Without any approved therapeutics in the U.S. for
these rare and devastating life-threatening tumors, patients face a
poor prognosis and few options. The tumor response data, in
particular for the patients that received two doses, along with the
adverse event profile from this trial, suggest that AZEDRA has the
potential to be a true breakthrough in addressing these
difficult-to-treat patients.”
Phase 2b Trial Topline
ResultsThe Phase 2b open-label trial was designed to
evaluate the efficacy and safety of two therapeutic doses of AZEDRA
administered three months apart to patients with malignant
relapsed/refractory pheochromocytoma or paraganglioma.
Primary Endpoint: Reduction in
Antihypertensive MedicationsUnder the study protocol, the
primary endpoint is achieved if the lower limit of the two-sided
95% confidence interval was above 10%. In order to achieve
this primary endpoint, a minimum of 12 of the total 68 evaluable
patients must have a 50% or greater reduction of all
antihypertensive medication for at least 6 months. As shown
in the table below, 17 patients responded, giving a lower limit of
the 95% confidence interval of 16.15%, thus meeting the primary
endpoint.
|
Responders (%) |
Lower bound ofConfidence Interval |
Upper bound ofConfidence Interval |
Overall (n=68) |
17 (25%) |
16.15 |
% |
36.52 |
% |
Two Doses (n=50) |
16 (32%) |
20.70 |
% |
45.87 |
% |
One Dose (n=18) |
1 (5.6%) |
0.0 |
% |
27.65 |
% |
Secondary Endpoint: Overall Tumor
Response Assessment per RECIST CriteriaBest response over
12 months after first therapeutic dose
|
CompleteResponse |
PartialResponse |
Stable Disease |
ProgressiveDisease |
Unable toEvaluate |
Overall (n=64*) |
0 |
% |
23.4 |
% |
68.8 |
% |
4.7 |
% |
3.1 |
% |
2 doses (n=50) |
0 |
% |
30.0 |
% |
68.0 |
% |
2.0 |
% |
0 |
% |
1 dose(n=14) |
0 |
% |
0 |
% |
71.4 |
% |
14.3 |
% |
14.3 |
% |
*4 patients did not have follow-up scans
AZEDRA was generally well tolerated. The most
common treatment emergent adverse events were nausea,
thrombocytopenia, anemia, fatigue, leukopenia, and
neutropenia. These events are consistent with those observed
in prior AZEDRA studies.
“We intend to move quickly to complete our New
Drug Application submission by mid-2017, as these topline results
underscore the potential of AZEDRA in this ultra-orphan
indication,” said Mark Baker, Chief Executive Officer of Progenics.
“We are grateful to the patients and investigators who participated
in this trial, and are committed to bringing a new treatment option
to this rare cancer population.”
Progenics plans to present additional data from
this trial at a medical meeting in the second half of 2017.
Conference Call and
WebcastProgenics will further review these topline results
from the registrational Phase 2b trial of AZEDRA® in a conference
call today at 8:30 a.m. ET. To participate, please dial (877)
250-8889 (domestic) or (720) 545-0001 (international) and reference
conference ID 99400682. A live webcast will be available in the
Media Center of the Progenics website, www.progenics.com, and a
replay will be available there for two weeks.
About AZEDRA®
AZEDRA® (iobenguane I 131) Injection, 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 registrational 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 secrete 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 Pharmaceuticals, Inc. develops
innovative medicines and other technologies to target and treat
cancer. The Company's pipeline includes: 1) therapeutic agents
designed to precisely target cancer (AZEDRA® and 1095), 2)
PSMA-targeted imaging agents for prostate cancer (1404 and PyLTM),
and 3) imaging analysis tools. Progenics' first commercial product,
RELISTOR® (methylnaltrexone bromide) for opioid-induced
constipation, 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
will be accompanied by words such as "anticipate," "believe,"
"plan," "could," "should," "estimate," "expect," "forecast,"
"outlook," "guidance," "intend," "may," "might," "will,"
"possible," "potential," "predict," "project," or other similar
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, 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; 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
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.
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 statements, whether as a result of new information,
future events or circumstances or otherwise.
Additional information concerning Progenics and
its business may be available in press releases or other public
announcements and public filings made after this release. For more
information, please visit www.progenics.com. Please follow us on
LinkedIn®. Information on or accessed through our website or social
media sites is not included in the company's SEC filings.
(PGNX-F)
Contact:
Melissa Downs
Investor Relations
(646) 975-2533
mdowns@progenics.com
Progenics Pharmaceuticals (NASDAQ:PGNX)
Historical Stock Chart
From Mar 2024 to Apr 2024
Progenics Pharmaceuticals (NASDAQ:PGNX)
Historical Stock Chart
From Apr 2023 to Apr 2024