Retrophin, Inc. (NASDAQ: RTRX) today announced that it has entered
into a definitive agreement to acquire Orphan Technologies Limited,
a privately held, clinical-stage biopharmaceutical company focused
on the development of product candidate OT-58 for the treatment of
classical homocystinuria (HCU). OT-58 is a novel investigational
enzyme replacement therapy being evaluated in Phase 1/2 development
for the treatment of classical HCU, a rare metabolic disorder
characterized by elevated levels of plasma homocysteine that can
lead to life-threatening thrombotic events such as stroke and heart
attacks, ophthalmologic and skeletal complications, as well as
developmental delay. Current treatment options, including heavy
dietary restrictions and supplemental use of vitamin B6 and
betaine, are often ineffective in managing homocysteine levels and
a significant unmet need remains.
“Many people with HCU face a continuous risk of
developing life-threatening complications because current treatment
options are largely ineffective in managing homocysteine levels,”
said Eric Dube, Ph.D., chief executive officer of Retrophin. “OT-58
has demonstrated an ability to meaningfully reduce homocysteine
levels in preclinical models and has the potential to ultimately
become the first disease modifying therapy for HCU. This promising,
novel development candidate fits directly with our mission to
identify, develop and deliver life-changing therapies to people
living with rare disease and brings exciting growth potential to
Retrophin.”
OT-58 is a PEGylated, recombinant enzyme
replacement therapy designed to address the underlying cause of
classical HCU — a deficiency in the naturally occurring enzyme
cystathionine beta synthase (CBS). A deficiency in CBS prevents
regular metabolism from occurring and results in elevated levels of
homocysteine. In preclinical studies, OT-58 has demonstrated an
ability to reduce total homocysteine levels and improve clinical
parameters. Specifically, dosing of OT-58 in mouse models corrected
metabolite levels, including up to 90% reduction in homocysteine
levels in plasma and tissues, and appeared to prolong survival,
prevent osteoporosis and rescue ocular structure. OT-58 is
currently advancing in a Phase 1/2 dose escalation study to assess
its safety, tolerability, pharmacokinetics, pharmacodynamics and
clinical effects in patients with classical HCU. OT-58 has been
granted Rare Pediatric Disease and Fast Track designations by the
US Food and Drug Administration (FDA), as well as Orphan Drug
designation in the US and Europe.
“Orphan Technologies’ longstanding mission has
been to reduce the disease burden for people living with HCU,
including debilitating complications of the skeletal,
cardiovascular, ocular, and central nervous systems. Therefore, I
am extremely proud of the Orphan Technologies team for advancing
OT-58 from early stage research and preclinical studies into a
Phase 1/2 trial,” said Frank Glavin, chief executive officer of
Orphan Technologies. “We are now at an ideal juncture to pair the
therapeutic promise of OT-58 with Retrophin’s late-stage
development and commercial capabilities in rare diseases. I believe
that with this new stewardship, we increase the potential for OT-58
to become an impactful new treatment option for patients.”
Under the terms of the agreement, Retrophin will
make an upfront payment of $90 million in cash upon closing of the
transaction. Orphan Technologies shareholders will also be eligible
to receive up to $427 million in additional cash payments
contingent upon the achievement of key milestones in the
development and commercialization of OT-58. Retrophin will also pay
a tiered mid-single digit royalty on future net sales of OT-58 in
the US and Europe, and potentially make a milestone payment in the
event a pediatric rare disease voucher is granted.
The transaction has been approved by the boards
of directors of both companies. It is subject to customary closing
conditions, including consummation of a spinout agreement for
Orphan Technologies’ preclinical OT-15 product candidate, and is
anticipated to close in the fourth quarter of 2020.
Barclays acted as financial advisor, and Cooley
LLP acted as legal counsel to Retrophin. Cantor Fitzgerald &
Co. acted as financial advisor, and Hogan Lovells US LLP acted as
legal counsel to Orphan Technologies.
Conference Call Information
Retrophin will host a conference call and
webcast today, October 22, 2020 at 5:00 p.m. ET to discuss the
acquisition. To participate in the conference call, dial
+1-855-219-9219 (U.S.) or +1-315-625-6891 (International),
confirmation code 5086267 shortly before 5:00 p.m. ET. The webcast
and slides can be accessed at retrophin.com, in the Events and
Presentations section, and will be archived for at least 30 days. A
replay of the call will be available from 8:00 p.m. ET, October 22,
2020 to 8:00 p.m. ET, October 29, 2020. The replay number is +1
(855) 859-2056 (U.S.) or +1 (404) 537-3406 (International),
confirmation code 5086267.
About Classical
Homocystinuria
Classical homocystinuria (HCU) is a rare genetic
metabolic disorder caused by a deficiency in the enzyme
cystathionine beta synthase (CBS). CBS is a pivotal enzyme that is
essential for the management of methionine and cysteine in the
body. Classical HCU leads to toxic levels of homocysteine that can
result in life-threatening thrombotic events such as stroke and
heart attacks, ophthalmologic and skeletal complications, as well
as developmental delay. Current treatment options are limited to
protein-restricted diet and supplemental use of vitamin B6 and
betaine.
About Retrophin
Retrophin is a biopharmaceutical company
specializing in identifying, developing and delivering
life-changing therapies to people living with rare disease. The
Company’s approach centers on its pipeline featuring sparsentan, a
product candidate in late-stage development for focal segmental
glomerulosclerosis (FSGS) and IgA nephropathy (IgAN), rare
disorders characterized by progressive scarring of the kidney often
leading to end-stage renal disease. Research in additional rare
diseases is also underway, including partnerships with leaders in
patient advocacy and government research to identify potential
therapeutics for NGLY1 deficiency and Alagille syndrome, conditions
with no approved treatment options. Retrophin’s R&D efforts are
supported by revenues from the Company’s commercial products
Chenodal®, Cholbam®, Thiola® and Thiola EC®.
Retrophin.com
About Orphan
Technologies
Orphan Technologies is a clinical-stage
biopharmaceutical company dedicated to developing novel therapies
to dramatically improve the lives of patients suffering from the
rare disorder, classical homocystinuria. OT-58 has been optimized
as an investigational enzyme replacement therapy for classical
homocystinuria, a genetic disease characterized by debilitating
cardiovascular, skeletal, neurologic, and ophthalmologic
complications. OT-58 is designed to reduce homocysteine levels via
a targeted mechanism of action and may have therapeutic
applications in other diseases.
Forward-Looking Statements
This press release contains "forward-looking
statements" as that term is defined in the Private Securities
Litigation Reform Act of 1995. Without limiting the foregoing,
these statements are often identified by the words "may", "might",
"believes", "thinks", "anticipates", "plans", "expects", "intends"
or similar expressions. In addition, expressions of our strategies,
intentions or plans are also forward-looking statements. Such
forward-looking statements include, but are not limited to,
references related to Retrophin’s expectations with respect to the
closing of its planned acquisition of Orphan Technologies; the
potential impact upon and benefits to Retrophin from the proposed
acquisition; the potential for OT-58 to ultimately become the first
disease modifying therapy for HCU; and references to future
expectations, plans and prospects for Retrophin. Such
forward-looking statements are based on current information
available to Retrophin and involve inherent risks and
uncertainties, including factors that could delay, divert or change
any such forward-looking statements, and could cause actual
outcomes and results to differ materially from current
expectations. No forward-looking statement can be guaranteed.
Retrophin faces risks associated with, but not limited to: the
parties’ ability to complete the proposed transaction in a timely
manner, if at all, considering the various closing conditions; if
consummated, Retrophin’s ability to realize the anticipated
benefits of the proposed transaction, including the potential
developmental and commercial success of the OT-58 product
candidate; significant and unknown transaction costs; actual or
contingent liabilities; the risk of litigation and/or regulatory
actions related to the proposed transaction; other business effects
outside of either company’s control, including the effects of
industry, market, economic, political or regulatory conditions or
the ongoing COVID-19 pandemic; as well as negative impacts that
could result from changes in tax and other laws, regulations, rates
and policies. In addition, such risks and uncertainties may include
those described in Retrophin’s annual, quarterly and current
reports (i.e., Form 10-K, Form 10-Q and Form 8-K) as filed or
furnished with the Securities and Exchange Commission, which are
available at Retrophin’s website (www.retrophin.com) under
“Investors & Media”. You are cautioned not to place undue
reliance on any forward-looking statements as there are important
factors that could cause actual results to differ materially from
those in any forward-looking statements, many of which are beyond
our control. Except to the extent required by law, Retrophin
undertakes no obligation to publicly update any forward-looking
statement.
Contact:Chris Cline, CFASenior Vice President,
Investor Relations & Corporate
Communications888-969-7879IR@retrophin.com
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