PALO ALTO, Calif., Nov. 23, 2020 /PRNewswire/ -- Eiger
BioPharmaceuticals, Inc. (Nasdaq:EIGR), focused on the development
and commercialization of targeted therapies for serious rare and
ultra-rare diseases, today announced that it has entered into a
definitive agreement to sell its Priority Review Voucher (PRV) for
a lump sum payment of $95
million. Eiger will retain fifty percent of the
proceeds, or $47.5 million, under the
terms of the Collaboration and Supply Agreement with The Progeria
Research Foundation (PRF).
The PRV was granted in conjunction with the recent approval by
the U.S. Food and Drug Administration of
ZokinvyTM (lonafarnib) for treatment of Progeria
and processing-deficient Progeroid Laminopathies. The
transaction remains subject to customary closing conditions,
including anti-trust review.
"The sale of the PRV provides Eiger with an important source of
non-dilutive capital and further strengthens our balance
sheet. The proceeds allow us to continue to ensure that all
diagnosed children and young adults worldwide with Progeria and
processing-deficient Progeroid Laminopathies have access to Zokinvy
and to advance our late-stage pipeline that now includes three
breakthrough therapy designated programs," said David Cory, President and CEO of Eiger.
"We are proud that Zokinvy is our first approved product and the
first approved therapy for children and young adults with Progeria
and processing-deficient Progeroid Laminopathies."
For full prescribing information, visit www.zokinvy.com.
About Zokinvy™ (lonafarnib)
Zokinvy blocks
the accumulation of defective, farnesylated proteins which
form tight associations with the nuclear envelope, leading to cellular instability
and the process of premature aging in children and young adults
with Progeria and processing-deficient Progeroid Laminopathies.
Eiger licensed exclusive worldwide rights to lonafarnib from
Merck, known as MSD outside of the United
States and Canada. Merck will not receive any
milestone payments for the development of lonafarnib for the
treatment of Progeria, and has waived royalty obligations from
Eiger for a specified quantity of lonafarnib.
INDICATION
ZOKINVY is indicated in adult and pediatric
patients 12 months of age and older with a body surface area (BSA)
of 0.39 m2 and
above:
- To reduce the risk of mortality in Hutchinson-Gilford Progeria
Syndrome (HGPS)
- For the treatment of processing-deficient Progeroid
Laminopathies with either:
-
- Heterozygous LMNA mutation with progerin-like protein
accumulation
- Homozygous or compound heterozygous ZMPSTE24 mutations
Limitations of Use
ZOKINVY is not indicated for use in
patients with non-HGPS Progeroid Syndromes or with Progeroid
Laminopathies known to be processing-proficient. Based upon its
mechanism of action, ZOKINVY would not be expected to be effective
in these populations.
Contraindications
- Strong or moderate CYP3A inhibitors or inducers
- Midazolam
- Lovastatin, simvastatin, and atorvastatin
IMPORTANT SAFETY INFORMATION
- The most common adverse reactions are vomiting (90%), diarrhea
(81%), infection (78%), nausea (56%), decreased appetite (53%),
fatigue (51%), upper respiratory tract infection (51%), abdominal
pain (48%), musculoskeletal pain (48%), electrolyte abnormalities
(43%), headache (37%), decreased weight (37%), increased aspartate
aminotransferase (35%), myelosuppression (35%), cough (33%),
decreased blood bicarbonate (33%), hypertension (29%), and
increased alanine aminotransferase (27%).
Gastrointestinal Adverse Reactions
- Gastrointestinal adverse reactions were the most frequently
reported adverse reactions. Of the 57 patients (90%) that
experienced vomiting, 30 (53%) patients had mild vomiting, 26 (46%)
patients had moderate vomiting, and 1 (2%) patient had severe
vomiting.
- Of the 35 patients (56%) that experienced nausea, 34 (97%)
patients had mild nausea and 1 (3%) patient had moderate
nausea.
- Of the 51 patients (81%) that experienced diarrhea, the
majority of patients (92%) experienced mild or moderate diarrhea;
38 (75%) patients reported mild diarrhea and 9 (18%) patients
reported moderate diarrhea. Four (8%) patients reported severe
diarrhea.
- Loss of fluids and dehydration can be severe, leading to
hospitalization. As a result, patients should receive therapy for
diarrhea at the earliest signs in order to avoid possible severe
complications.
Alanine Aminotransferase and Aspartate Aminotransferase
Elevations
- Increased alanine aminotransferase was commonly reported (17
[27%] patients). Of the 17 patients with increased alanine
aminotransferase, 14 (82%) patients had mild increases, 1 (6%)
patient had moderate increases, and 2 (12%) patients had severe
increases.
- Increased aspartate aminotransferase was also commonly reported
(22 [35%] patients). Of the 22 patients with increased aspartate
aminotransferase, 21 (95%) patients had mild increases and 1 (5%)
patient had a severe increase.
Hypertension
- Increases in blood pressure have been documented in patients
treated with ZOKINVY. At baseline 22 (35%) patients had either a
systolic blood pressure or a diastolic blood pressure or both above
the 95th percentile. Over the course of the trials, 18 (29%)
patients had hypertension based on systolic blood pressure or
diastolic blood pressure measurements above the 95th percentile on
3 or more occasions. Five (8%) patients who were normotensive at
baseline had either systolic blood pressure or diastolic blood
pressure above the 95th percentile at the end of treatment.
Ophthalmic Adverse Reactions
- Lonafarnib caused retinal toxicity in monkeys at 3.7 times the
human dose based on plasma drug exposure, but not at 2.1 times the
human dose.
Laboratory Abnormalities
Some patients treated with
ZOKINVY developed laboratory abnormalities. These included:
- Electrolyte abnormalities (43%), such as hyperkalemia,
hypokalemia, hyponatremia, or hypercalcemia
- Myelosuppression (35%), such as reductions in absolute
neutrophil count, white blood cell counts, lymphopenia, hemoglobin,
or hematocrit
- Increased liver enzymes, such as aspartate aminotransferase
(35%), or alanine aminotransferase (27%)
These laboratory abnormalities often improved while continuing
ZOKINVY, but it is not possible to exclude ZOKINVY as a cause of
the abnormalities. Periodically monitor electrolytes, complete
blood counts, and liver enzymes, and manage abnormalities
accordingly.
Nephrotoxicity
- Lonafarnib caused nephrotoxicity in rats at plasma drug
exposures approximately equal to that achieved with the human
dose. Monitor renal function at regular intervals during
ZOKINVY therapy.
Retinal Toxicity
- Lonafarnib caused rod-dependent, low-light vision decline in
monkeys at plasma drug exposures similar to that achieved with the
human dose. Perform ophthalmological evaluation at regular
intervals and at the onset of any new visual changes during ZOKINVY
therapy.
Impaired Fertility
- Lonafarnib caused impaired fertility in female rats at 1.2
times the human dose based on plasma drug exposure.
- Lonafarnib caused impaired fertility and testicular toxicity in
male rats at 1.5 times the human dose based on plasma drug
exposure, and toxicity in the male reproductive tract in monkeys at
doses lower than the human dose based on plasma drug exposure.
About Eiger
Eiger is a commercial-stage
biopharmaceutical company focused on the development and
commercialization of first-in-class, well-characterized drugs for
serious rare and ultra-rare diseases for patients with high unmet
medical needs.
Zokinvy for the treatment of Hutchinson-Gilford Progeria
Syndrome (HGPS or Progeria) and processing-deficient Progeroid
Laminopathies is the Company's first FDA approval. A
Marketing Authorization Application (MAA) has been accepted and is
under review by the European Medicines Agency (EMA). Outside
the U.S., Eiger's established global Managed Access Program,
expected to span greater than 40 countries, ensures all children
and young adults with Progeria and Progeroid Laminopathies have
access to treatment.
Eiger's lead clinical programs target Hepatitis Delta Virus
(HDV) infection, the most serious form of human viral
hepatitis. Eiger is developing two complementary treatments
for HDV. Lonafarnib is a first-in-class, oral prenylation
inhibitor in a global Phase 3 trial. Peginterferon lambda is
a first-in-class, well-tolerated type III interferon entering Phase
3.
For additional information about Eiger and its clinical
programs, please visit www.eigerbio.com
Note Regarding Forward-Looking Statements
This press
release contains "forward-looking" statements that involve
substantial risks and uncertainties. All statements other than
statements of historical facts, including statements regarding our
future financial condition, timing for and outcomes of clinical
results, business strategy and plans and objectives for future
operations, are forward-looking statements. These forward-looking
statements include terminology such as "believe," "will," "may,"
"estimate," "continue," "anticipate," "contemplate," "intend,"
"target," "project," "should," "plan," "expect," "predict,"
"could," "potentially" or the negative of these terms.
Forward-looking statements are our current statements regarding our
intentions, beliefs, projections, outlook, analyses or current
expectations concerning, among other things, our anticipating
significant milestones in 2020 and 2021, the timing of our ongoing
and planned clinical development, including our ability to support
the launch of a new product and ship to specialty pharmacies; our
development programs for Zokinvy generally; and the potential
approval of Zokinvy in jurisdictions outside of the U.S., including
the EU; the risks related to the commercialization of Zokinvy, our
ability to manufacture sufficient quantities of Zokinvy, and the
commercial launch of Zokinvy in the U.S., the market potential for
Zokinvy as a treatment for Progeria and processing-deficient
Progeroid Laminopathies; our progression and enrollment of our
Phase 3 D-LIVR study in HDV; our ability to maintain supply of our
commercial and clinical trial materials; our plans to advance
Lambda in HDV in the U.S. and EU; our ability to transition into a
commercial stage biopharmaceutical company; our ability to finance
the continued advancement of our development pipeline products; and
the potential for success of any of our product candidates. These
statements concern product candidates that have not yet been
approved for marketing by the U.S. Food and Drug Administration
(FDA). No representation is made as to their safety or
effectiveness for the purposes for which they are being
investigated. Various important factors could cause actual results
or events to differ materially from the forward-looking statements
that Eiger makes, including additional applicable risks and
uncertainties described in the "Risk Factors" sections in the
Quarterly Report on Form 10-Q for the quarter ended September 30, 2020 and Eiger's subsequent filings
with the SEC. The forward-looking statements contained in this
press release are based on information currently available to Eiger
and speak only as of the date on which they are made. Eiger does
not undertake and specifically disclaims any obligation to update
any forward-looking statements, whether as a result of any new
information, future events, changed circumstances or otherwise.
Investors and Media:
Sri
Ryali
CFO
(650) 272-6138
sryali@eigerbio.com
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SOURCE Eiger BioPharmaceuticals, Inc.