SOUTH PLAINFIELD, N.J.,
July 19, 2018 /PRNewswire/
-- PTC Therapeutics, Inc. (NASDAQ: PTCT) today
announced that it has entered into an agreement to acquire Agilis
Biotherapeutics, Inc., a biotechnology company advancing an
innovative gene therapy platform for rare monogenic diseases that
affect the central nervous system (CNS). The transaction was
approved by the Boards of both companies.
"The addition of the gene therapy platform transforms PTC and
aligns with our vision of being a leader in the treatment of rare
disorders," said Stuart W. Peltz,
Ph.D., Chief Executive Officer, PTC Therapeutics, Inc. "We are
impressed with the clinical results shown by the AADC program and
are excited with the potential to quickly bring this therapy to
patients. We look forward to advancing the Friedreich ataxia and
Angelman syndrome programs into the clinic in the next two
years."
"I am proud of the accomplishments achieved by Agilis
culminating with this value-creating transaction," said
Mark Pykett, DVM, Ph.D., President
and Chief Executive Officer of Agilis Biotherapeutics, Inc. "PTC
provides a global infrastructure and proven capabilities, which we
believe will enable our goal of providing therapy to patients
suffering from rare CNS disorders. I look forward to joining PTC
and supporting the advancement of the programs to provide value to
patients."
The lead gene therapy candidate, GT-AADC, has compelling
clinical data in treating Aromatic L-Amino Acid Decarboxylase
(AADC) Deficiency. AADC Deficiency is a rare CNS disorder arising
from reductions in the enzyme AADC that result from mutations in
the dopa decarboxylase (DDC) gene. In its severe forms, AADC
Deficiency causes the inability to develop any motor control
(global muscular hypotonia/dystonia) resulting in breathing,
feeding, and swallowing problems, frequent hospitalizations, and
the need for life-long care. Many patients die in the first decade
of life due to profound motor dysfunction and secondary
complications such as choking, hypoxia, and pneumonia. Currently,
no treatment options are available for the underlying cause of the
disorder, and care is limited to palliative options with
significant burden on caregivers.
The results from two prospective clinical studies enrolling 18
subjects with severe AADC Deficiency indicated that treated
subjects with GT-AADC exhibited de novo dopamine production.
Additionally, the subjects demonstrated achievement of motor
development milestones and substantial gains on motor function and
cognitive scales over multiple years following the single gene
therapy treatment. Data from trials that began in 2010 have
demonstrated long-term evidence of durable clinical benefit. Based
on multi-year data from initial clinical studies with the AADC gene
therapy treatment candidate, PTC plans to submit a Biologics
License Application (BLA) to the FDA in 2019.
The product pipeline also includes a gene therapy asset
targeting Friedreich ataxia, a rare and life-shortening
neurodegenerative disease caused by a single defect in the FXN gene
which causes reduced production of the frataxin protein. An IND
submission for this program is expected in 2019. Additionally, the
acquisition brings two other gene therapy programs targeting CNS
disorders, including Angelman syndrome, a rare, genetic,
neurological disorder characterized by severe developmental
delays.
The transaction is expected to close in the third quarter of
2018, pending successful fulfillment of all customary closing
conditions. On completion, PTC plans a smooth transition of
operations and the integration of Agilis' talented and dedicated
employees to continue the mission of bringing the pipeline of gene
therapies for CNS disorders to patients worldwide.
Transaction Details
Under the terms of the merger
agreement, PTC will pay an upfront consideration of $50 million in cash and approximately
$150 million in PTC common stock,
subject to an estimated maximum 9.34 million share limit (with any
shortfall to be made whole with additional cash consideration). In
addition to the upfront payments, potential future consideration
includes $60 million in development
milestones to be paid over the next two years which includes the
acceptance of a BLA. Additionally, the transaction includes up to
$535 million in success-based
milestones in connection with regulatory approvals on the three
most advanced programs and receipt of a priority review voucher, as
well as tiered commercial milestones of $150
million, and 2-6 % of annual net sales for Friedreich ataxia
and Angelman syndrome.
Conference Call and Webcast Details
A conference call
will take place on Thursday, July 19
at 4:30 pm ET and can be accessed by
dialing (877) 303-9216 (domestic) or (973) 935-8152 (international)
five minutes prior to the start of the call and providing the
passcode 8469109. A live, listen-only webcast of the conference
call can be accessed at
https://edge.media-server.com/m6/p/25h8j9nj. A webcast replay of
the call will be available approximately two hours after completion
of the call and will be archived on the company's website for two
weeks.
About Agilis Biotherapeutics, Inc.
Agilis is advancing
innovative gene therapies designed to provide long-term efficacy
for patients with debilitating, often fatal, rare genetic diseases
that affect the central nervous system. Agilis' gene therapies are
engineered to impart sustainable clinical benefits by inducing
persistent expression of a therapeutic gene through precise
targeting and restoration of lost gene function to achieve
long-term efficacy. Agilis' rare disease programs are focused on
gene therapy for AADC deficiency, Friedreich ataxia, and Angelman
syndrome, all rare genetic diseases that include neurological
deficits and result in physically debilitating conditions.
About PTC Therapeutics
PTC is a science-led, global
biopharmaceutical company focused on the discovery, development and
commercialization of clinically-differentiated medicines that
provide benefits to patients with rare disorders. Founded 20 years
ago, PTC Therapeutics has successfully launched two rare
disorder products and has a global commercial footprint. This
success is the foundation that drives investment in a robust
pipeline of transformative medicines and our mission to provide
access to best-in-class treatments for patients who have an unmet
medical need. For more information, please visit our website at
www.ptcbio.com.
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 closing of its planned acquisition
of Agilis and the other transactions contemplated in conjunction
with the acquisition; the potential financial impact and benefits
to PTC of the acquisition, including with respect to the business
of Agilis to be acquired and PTC's expectations with respect to
contingent payments to the Agilis equityholders based on net sales
and the potential achievement of development, regulatory and sales
milestones and contingent payments to the Agilis equityholders with
respect thereto; the future expectations, plans and prospects for
PTC; PTC's strategy, future operations, future financial position,
future revenues or projected costs; the integration of Agilis'
operations and employees; 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: satisfaction of the
conditions to closing the acquisition (including the failure to
obtain necessary Agilis stockholder and regulatory approvals) in
the anticipated timeframe or at all; PTC's ability to realize the
anticipated benefits of the acquisition, 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 the announcement of the acquisition 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; 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 TranslarnaTM (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; the
integration of Agilis' operations and employees and the factors
discussed in the "Risk Factors" section of PTC's most recent
Quarterly Report on Form 10-Q or 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.
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 candidate will receive or maintain regulatory approval in
any territory, or prove to be commercially successful, including
Translarna or Emflaza, or any product candidates acquired in the
transaction from Agilis.
The forward-looking statements contained herein represent the
Company'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.