BIOWORLD:Chinese CAR T firm ICT licenses Vericel's cell therapy platform for Asia
来源:BIOWORLD TODAY
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作者:proaf1292
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发布时间: 2017-05-15
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1987 次浏览
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By Shannon Ellis, Staff Writer
SHANGHAI – Innovative Cellular Therapeutics Co. Ltd. (ICT), of Shanghai,
a leader among China’s pack of emerging CAR T oncology companies, has
struck a partnership deal with Vericel Corp., the first biotech to
receive FDA approval for a tissue-engineered autologous cell scaffold.
The deal moves ICT beyond
oncology and CAR T, to give the Chinese firm a portfolio of four cell
therapies for the treatment of knee cartilage repair, burn victims and
heart failure patients.
The deal also means ICT has the Asia rights to three FDA approved assets and one under clinical study.
“This is the first U.S.
FDA cell therapy deal with a Chinese company,” said Jimmy Wei, ICT CEO.
“In China, we are increasing our standard of quality and we think cell
therapy should be more globally standardized.”
A clinical-stage.
VC-backed company, ICT has two clinical programs running in China for
its 19CAR series to treat late stage leukemia and lymphoma as well as
numerous selfdiscovered CAR T oncology candidates. (See BioWorld Today,
May 25, 2016.)
Last year, ICT went
hunting for late-stage or approved cell therapy products outside of
China. With the help of a consulting firm, it came up with a list of 20
potential companies, but Vericel quickly rose to the top due to its
number of FDAapproved therapies. Vericel had acquired its technology
from Sanofi Genzyme, where its knee cartilage therapy had been tested on
patients in China. (See BioWorld Today, March 11, 2016, and Dec. 15,
2016.)
The deal gives ICT the
right to develop and distribute four candidates – Carticel, MACI,
ixmyelocel-T and Epicel – in Greater China, South Korea, Singapore and
other counties in Asia. The company also will be responsible for funding
the development, manufacturing and commercialization in that territory.
“MACI, Epicel and
Carticel are FDA-approved products and have successfully treated
thousands of patients in United States,” said Lei Xiao, ICT chairperson.
“Ixmyelocel-T has been evaluated in a phase II study in the U.S., and
the encouraging data suggest that it may be a treatment option for
millions of heart failure patients.”
Vericel will receive a $6
million up-front payment from ICT and is eligible to receive $8 million
in development and commercial milestones. Vericel may also receive
tiered royalties from ICT equal to a percentage of net sales of each
licensed product in the low to middle double digits.
ICT will also purchase $5 million worth of stock in Vericel.
Intended to be more than a
licensing agreement, the equity arrangement will set the foundation for
a long-term partnership, Wei said.
Vericel will issue to ICT
a warrant, exercisable for the number of shares of Vericel’s common
stock equal to $5 million less any withholding tax payable divided by
Vericel’s closing price on May 9, 2017, with an exercise price of $0.01
per share.
STILL AWAITING COMMERCIAL CLARITY
Currently, the commercial
pathway for cell therapies in China is still uncertain. Biotechs like
ICT are able to conduct smallscale clinical trials under the oversight
of the National Health and Family Planning Commission (NHFPC) – China’s
health ministry – and are able to collect clinical trial data in a much
quicker fashion than the CFDA-regulated process for new drugs. But
getting authorization to charge for cell therapies is not permitted,
although some companies find ways to charge hospitals in a process that
is difficult to scale.
But that grey area makes
it a buyer’s market for forwardthinking Chinese biotechs on the hunt for
China rights to global assets, at least for the time being.
“We should see more
clarity with the regulatory pathway this year,” Wei said. “After that,
we can quickly launch the products. But once the pathway is more clear,
the price of [in-licensing] those products will be much more expensive. .
. . After the pathway is set, you will never get the price we got.”
He said there are
currently three regulatory models for China to consider: the U.S.
system, in which the FDA approves cell therapies similarly to the way it
approves drugs; a system like Japan’s, where cell therapies are
regulated by the MoH as a technology; or a hybrid of the two systems, a
way that could work well in China if it could balance cost-effectiveness
with tight manufacturing quality control.
MANUFACTURING PLATFORM
Manufacturing is a
serious bottleneck for scaling autologous cell therapies, which require
extracting a patient’s cells to be processed before being reinserted
into the patient’s body. ICT has chosen to focus on building a logistics
and manufacturing platform shared across cell therapies rather than
stick strictly to a specific therapeutic area, such as oncology.
“We think the future for
cell therapy companies is to build a core in manufacturing. There needs
to be a cost advantage based on doing more than one product in order to
achieve an economy of scale,” said Wei. “There is a lot of similarity
across therapies. The manufacturing process and systems that we will put
in place [from Vericel] will be very helpful even for our CAR T
product.”
ICT hopes to take
advantage of Vericel’s quality control system that follow U.S. FDA
standards in anticipation that China’s guidelines could closely resemble
those of U.S.
The company also is
looking to bring its portfolio of CAR-T candidates to U.S. and Europe
and is currently in talks with potential partners.
MEETING AN UNMET NEED IN CHINA
In the end, Wei said he
is optimistic that the benefits of Vericel’s therapies – particularly
for burn victims and heart patients who currently have few options in
China – will win over the regulator concerns. Lacking detailed market
data, it is predicted that the Chinese patient population for those two
groups is four to five times as large as in the U.S.
Vericel
FDA-approved Epicel
therapy treats severe burn patients with more than 30 percent of their
total body surface area burned using cultured epidermal autografts, or
CEA. Vericel recently presented data demonstrating an 84 percent
survival rate from the Epicel clinical experience databases in more than
950 patients with a mean total body surface area of 67 percent, which
continues to support a probable survival benefit of Epicel in severe
burn patients.
Ixmyelocel-T is an
investigational autologous expanded multicellular therapy manufactured
from the patient’s own bone marrow using Vericel’s highly automated,
fully closed cell processing system.
That process selectively
expands the population of mesenchymal stromal cells and alternatively
activated macrophages, which are responsible for production of
anti-inflammatory and pro-angiogenic factors known to be important for
repair of damaged tissue. It has been designated as an orphan drug by
the U.S. FDA for use in the treatment of dilated cardiomyopathy (DCM).
The ixCELL-DCM trial was a
multicenter, randomized, double-blind, placebo-controlled phase IIb
study designed to assess the efficacy, safety and tolerability of
ixmyelocel-T compared to placebo when administered via transendocardial
catheter-based injections to participants with end-stage heart failure
due to ischemic DCM, who have no reasonable revascularization options
(either surgical or percutaneous interventional) likely to provide
clinical benefit. All participants were on maximized pharmacological
heart failure treatment and had an automatic implantable cardiac
defibrillator or cardiac resynchronization therapy. The primary endpoint
is the number of all-cause deaths, cardiovascular hospital admissions,
and unplanned outpatient and emergency department visits to treat acute
decompensated heart failure over the 12 months following administration
of ixmyelocel-T compared to placebo.
MACI (autologous cultured
chondrocytes on porcine collagen membrane) is an autologous cellular
scaffold product that is indicated for the repair of symptomatic single
or multiple fullthickness cartilage defects of the knee with or without
bone involvement in adults. The MACI implant consists of autologous
cultured chondrocytes seeded onto a resorbable type I/III collagen membrane.
Autologous cultured chondrocytes are human-derived cells which are
obtained from the patient’s own cartilage for the manufacture of MACI.
An autologous chondrocyte
implant, Carticel is used to treat cartilage defects in the knee in
patients who have had an inadequate response to a prior arthroscopic or
other surgical repair procedure. //