SOUTH PLAINFIELD, N.J.,
April 20, 2017 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today announced it
has completed its acquisition of all rights to Emflaza™
(deflazacort) for the treatment of Duchenne muscular dystrophy
(DMD) in the U.S. Execution of the asset purchase agreement setting
forth the terms of the acquisition was announced on March 16, 2017.
"We are pleased the acquisition was completed ahead of schedule,
following early conclusion of the antitrust review period," stated
Stuart Peltz, Ph.D., chief executive
officer, PTC Therapeutics, Inc. "We've been engaging with key
stakeholders in the DMD community to understand their needs and are
working to make Emflaza commercially available as soon as possible.
PTC is committed to bringing this important therapy to patients
with DMD. Our goal is to enable access for eligible patients
irrespective of insurance status, and we look forward to discussing
commercial launch details on our upcoming quarterly earnings
call."
Financial terms of the acquisition include a total upfront
consideration of $140 million paid to
Marathon Pharmaceuticals. Marathon is also entitled to receive
payments from PTC based on annual net sales of Emflaza beginning in
2018, which PTC expects will range as a percentage of net sales
between the low to mid-20s on a blended average basis. In addition,
Marathon has the opportunity to receive a single $50 million sales-based milestone.
About Duchenne Muscular Dystrophy
Primarily affecting
males, Duchenne muscular dystrophy (DMD) is a rare and fatal
genetic disorder that results in progressive muscle weakness from
early childhood and leads to premature death in the mid-twenties
due to heart and respiratory failure. It is a progressive muscle
disorder caused by the lack of functional dystrophin protein.
Dystrophin is critical to the structural stability of skeletal,
diaphragm, and heart muscles. Patients with DMD can lose the
ability to walk as early as age ten, followed by loss of the use of
their arms. DMD patients subsequently experience life-threatening
lung complications, requiring the need for ventilation support, and
heart complications in their late teens and twenties. More
information regarding DMD is available through the Muscular
Dystrophy Association and the Parent Project Muscular Dystrophy.
Additionally, information and resources are available at
www.duchenneandyou.com.
About PTC Therapeutics
PTC is a global
biopharmaceutical company focused on the discovery, development,
and commercialization of novel medicines using our expertise in RNA
biology. PTC's internally discovered pipeline addresses multiple
therapeutic areas, including rare disorders and oncology. PTC has
discovered all of its compounds currently under development using
its proprietary technologies. Since its founding nearly 20 years
ago, PTC's mission has focused on developing treatments to
fundamentally change the lives of patients living with rare genetic
disorders. The company was founded in 1998 and is headquartered in
South Plainfield, New Jersey. For
more information on the company, please visit our website
www.ptcbio.com.
About Emflaza
EMFLAZA™ is indicated for the treatment
of Duchenne muscular dystrophy in patients 5 years of age and
older.
IMPORTANT SAFETY INFORMATION
Contraindication: Do not
use if you are allergic to deflazacort or any of the inactive
ingredients in EMFLAZA.
Do not stop taking EMFLAZA, or change the amount you are taking,
without first checking with your healthcare provider, as there may
be a need for gradual dose reduction to decrease the risk of
adrenal insufficiency and steroid "withdrawal syndrome". Acute
adrenal insufficiency can occur if corticosteroids are withdrawn
abruptly, and can be fatal. A steroid "withdrawal syndrome,"
seemingly unrelated to adrenocortical insufficiency, may also occur
following abrupt discontinuance of corticosteroids. For patients
already taking corticosteroids during times of stress, the dosage
may need to be increased.
- Hyperglycemia: Corticosteroids can increase blood glucose,
worsen pre-existing diabetes, predispose those on long-term
treatment to diabetes mellitus, and may reduce the effect of
anti-diabetic drugs. Monitor blood glucose at regular intervals.
For patients with hyperglycemia, anti-diabetic treatment should be
initiated or adjusted accordingly.
- Increased Risk of Infection: Tell your healthcare provider if
you have had recent or ongoing infections or if you have recently
received a vaccine or are scheduled for a vaccination. Seek medical
advice at once should you develop fever or other signs of
infection, as some infections can potentially be severe and fatal.
Avoid exposure to chickenpox or measles, but if you are exposed,
medical advice should be sought without delay.
- Alterations in Cardiovascular/Kidney Function: EMFLAZA can
cause an increase in blood pressure, salt and water retention, or a
decrease in your potassium and calcium levels. If this occurs,
dietary salt restriction and potassium supplementation may be
needed.
- Behavioral and Mood Disturbances: There is a potential for
severe behavioral and mood changes with EMFLAZA and you should seek
medical attention if psychiatric symptoms develop.
- Effects on Bones: There is a risk of osteoporosis or decrease
in bone mineral density with prolonged use of EMFLAZA, which can
potentially lead to vertebral and long bone fractures.
- Effects on Growth and Development: Long-term use of
corticosteroids, including EMFLAZA may slow growth and development
in children.
- Ophthalmic Effects: EMFLAZA may cause cataracts or glaucoma and
you should be monitored if corticosteroid therapy is continued for
more than 6 weeks.
- Vaccination: The administration of live or live attenuated
vaccines is not recommended. Killed or inactivated vaccines may be
administered, but the responses cannot be predicted.
- Serious Skin Rashes: Seek medical attention at the first sign
of a rash.
- Drug Interactions: Certain medications can cause an interaction
with EMFLAZA. Tell your healthcare provider of all the medicines
you are taking, including over-the-counter medicines (such as
insulin, aspirin or other NSAIDS), dietary supplements, and herbal
products. Alternate treatment, dosage adjustment, and/or special
test(s) may be needed during the treatment.
Common side effects that could occur with EMFLAZA include:
Facial puffiness or Cushingoid appearance, weight increased,
increased appetite, upper respiratory tract infection, cough,
frequent daytime urination, unwanted hair growth, central obesity,
and colds.
Please see the accompanying full Prescribing Information
You may report side effects to ProPharma Group at 1‑866‑562‑4620
or drugsafety@propharmagroup.com.
You may report side effects to FDA at 1‑800‑FDA‑1088 or
www.fda.gov/medwatch.
For More Information:
Investors:
Emily
Hill
+ 1 (908) 912-9327
ehill@ptcbio.com
Media:
Jane Baj
+1 (908) 912-9167
jbaj@ptcbio.com
Forward Looking Statements:
This press release contains forward-looking statements within
the meaning of The Private Securities Litigation Reform Act of
1995. All statements, other than those of historical fact,
contained in this release are forward-looking statements, including
statements related to PTC's expectations with respect to the future
commercial availability of, and access to, Emflaza; PTC's
expectations with respect to contingent payments to Marathon based
on annual net sales; the future expectations, plans and prospects
for PTC; PTC's strategy, future operations, future financial
position, future revenues or projected costs; and the objectives of
management. Other forward-looking statements may be identified by
the words "look forward", "plan," "anticipate," "believe,"
"estimate," "expect," "intend," "may," "target," "potential,"
"will," "would," "could," "should," "continue," and similar
expressions.
PTC's actual results, performance or achievements could differ
materially from those expressed or implied by forward-looking
statements it makes as a result of a variety of risks and
uncertainties, including those related to: PTC's preparations for a
commercial launch of Emflaza; PTC's ability to realize the
anticipated benefits of the acquisition of Emflaza, including the
possibility that the expected benefits from the acquisition will
not be realized or will not be realized within the expected time
period; negative effects of this announcement on the market price
of PTC's common stock; significant transaction costs, unknown
liabilities, the risk of litigation and/or regulatory actions
related to the acquisition of Emflaza, as well as other business
effects, including the effects of industry, market, economic,
political or regulatory conditions; changes in tax and other laws,
regulations, rates and policies; the eligible patient base and
commercial potential of Translarna™ (ataluren) and Emflaza; the
sufficiency of PTC's cash resources and its ability to obtain
adequate financing in the future for its foreseeable and
unforeseeable operating expenses and capital expenditures; and the
factors discussed in the "Risk Factors" section of PTC's Annual
Report on Form 10-K as well as any updates to these risk factors
filed from time to time in PTC's other filings with the SEC. You
are urged to carefully consider all such factors.
As with any pharmaceutical under development, there are
significant risks in the development, regulatory approval and
commercialization of new products. There are no guarantees that any
product will receive or maintain regulatory approval in any
territory, or prove to be commercially successful, including
Translarna or Emflaza.
The forward-looking statements contained herein represents PTC's
views only as of the date of this press release and PTC does not
undertake or plan to update or revise any such forward-looking
statements to reflect actual results or changes in plans,
prospects, assumptions, estimates or projections, or other
circumstances occurring after the date of this press release except
as required by law.
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SOURCE PTC Therapeutics, Inc.