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Biotech and Ebola in Cambridge: Chris Garabedian, CEO of Sarepta
NEWS | AUGUST 21, 2014 11:04 AM
________________________________
By Aram Arkun
Mirror-Spectator Staff
CAMBRIDGE, Mass. - Recently, the Ebola virus has been in the news
almost every day, with panicked speculation about the danger of its
spread, and the ethics of treatment. There is no tested and proven
treatment or vaccine available. Christopher "Chris" Nishan Garabedian,
the president and chief executive officer (CEO) of Sarepta
Therapeutics, is frequently sought out by the media in these
discussions. It happens that his company is one of only a handful with
drugs that could treat Ebola victims. Garabedian, in his Cambridge
headquarters, explained that in fact this drug is only one example of
what his company can accomplish. Sarepta employs an approach which is
part of a revolution in biotechnology, using Ribonucleic Acid (RNA) to
specifically target diseases and genetic problems.
This is a different technology from the ZMapp combination of several
antibodies against the Ebola virus, which was in the news because it
was given to two American missionaries infected by Ebola recently.
These antibodies attempt to allow a person's own immune system to
identify and neutralize the virus. However, they had never been given
to humans before, while Sarepta's drugs have been used on non-human
primates and in Phase 1 safety trials for the Federal Drug
Administration (FDA), with promising results. The trials were
supported by the Department of Defense but economic issues halted the
program.
The problem is that treatments for many diseases which are usually
restricted in the numbers of victims do not economically attract
investments necessary to support research and trials. For this reason,
drugs intended to treat rare diseases affecting fewer than 200,000
people in the US are called orphan drugs. Garabedian said, "I think
the US government knows, as governments around the world know, that to
leave it up to the private sector to develop technologies and medical
countermeasures against these threats, whether pandemic threats that
spread naturally or biological ones that someone can manufacture in a
garage, is not feasible. They know they have to foot the bill. The
problem is that they are not funding it fully." He pointed out that
crises come and go, so specific diseases float in and out of the
public's awareness along with a sense of urgency, but the potential
threats remain.
For Ebola in particular, his company has enough supply ready only to
treat 100-150 people, if the FDA would give emergency authorization,
along with permission from the Department of Defense which had
provided initial funding. In case Ebola does spread on a greater
scale, he said, "We hope that this is raising awareness that we have
the technology but need to raise the manufacturing ability to a higher
scale."
The same is true of course for Ebola drugs using different approaches
which are being developed by a few other companies, like Tekmira's
TKM-Ebola, which the FDA very recently allowed to be used on infected
people. However, Garabedian exclaimed that "regardless of what press
or market reactions have been, we still think that we have the most
advanced and robust data set for safety and efficacy that is out there
for Ebola."
There are many companies utilizing various types of technologies that
attempt to modify or suppress RNA. Sarepta uses phosphorodiamidate
morpholino oligomers (PMOs), stable synthetic chemical structures
similar to RNA. They can be used to create drugs specific for either
human or pathogen RNA and can also target specific tissues. PMOs
increase or decrease the production of a protein involved in a
disease. In the case of Ebola or other viruses, the PMOs block access
of the virus to human cellular machinery and thus inhibit its
replication.
Despite the attention that it is garnering lately, the work on Ebola
or related viruses like Marburg is not the main focus of Sarepta's
commercial research. Instead, it is pursuing PMO-based therapeutics
actively for a form of muscular dystrophy called Duchenne (DMD), which
is caused by a mutation or error in the gene for dystrophin, a protein
necessary to protect human cells during muscle function. The PMO
treatment in this case deals with human and not virus RNA, and is
targeted to skip defective mutated sections of the dystrophin gene to
allow the body to make functional dystrophin protein. It can be
targeted to specific mutations, and thus treat different varieties of
the disease. Phase IIb clinical trials on safety and efficacy have
already been done on one of these drugs, eteplirsen, meaning that it
is advancing in the FDA process needed for government approval.
Garabedian stated that the reason for the focus on DMD is that
"Duchenne is a commercially viable market, high on medical need."
Furthermore, the same technology can be used for a series of related
drugs. Garabedian said, "It is the consummate personalized medicine."
This is novel for regulators like the FDA. He continued: "I would say
that this technology demands a new approach from the FDA...If you read
that we have had ups and downs, it is because the FDA sometimes gets
more conservative the more advanced and novel the technology is. I
have been characterized as being aggressive or pushing too hard, but
all we were doing is arguing on behalf of patients who need this
technology."
Sarepta needs at least one successful breakout drug to allow it to
expand the scale of its finances and work. Afterwards, it can expand
its PMO-based approach to many different diseases and genetic
problems, and will be in an enviable position. For example, among its
other ongoing projects, Sarepta is testing a flu drug using the PMO
approach.
There are many companies working on RNA therapeutics with different
approaches. While some others also work with PMOs, Garabedian said,
"We have the lion's share of the real estate of morpholino
intellectual property. We also have the most know-how on scale-up
manufacturing. While we don't have a 100 percent lock on all
morpholino, we are the only one in advanced drug development. We have
patents."
Born in 1966 and raised in the Washington DC area, Garabedian is
Armenian on both his mother and father's sides. His parents were born
in the US but his grandparents fled the Armenian Genocide to the US
via Ellis Island and Cuba. He did not get to know his grandparents
well, as they died either before he was born or while he was still
young. Garabedian's parents tried to instill Armenian culture in their
children, but by the time they came to Chris, it became too difficult.
For this reason, Garabedian said, "Peter Balakian's Black Dog of Fate
resonated with me. I too grew up with American cultural mores, and
came to appreciate my Armenian heritage as an adult." He started to
read books, and saw movies like Atom Egoyan's Ararat, and encountered
in the popular culture efforts at documenting the Armenian plight. He
did work for around a year from 1998 to 1999 for an Armenian, Vaughn
Kailian, CEO and president at COR Therapeutics, but otherwise has had
little opportunity to cultivate his Armenian connections in his
profession.
Garabedian had a business background in college and initially worked
in market research, brand management and new product development. He
described his involvement in biopharmaceuticals as "a little bit of
serendipity." He began learning about this industry in a consulting
company, and from 1994 worked at Abbott Laboratories in Chicago, which
"was a great training ground....Then Gilead Sciences called in 1997 and
I joined them. This was my formative experience in the biotech
industry. They really shaped a vision. At the time, they were less
valued than Sarepta is today. They had a few more employees...They have
about a 140 billion dollar market capitalization today. They built it
on a relatively small footprint. ... I really learned what it takes to
build a successful biotech, how to build in a lean, smart way, and
good, smart drug development. I learned from my mentors, like John
Martin as CEO of Gilead."
Garabedian found out through experience that many companies failed not
due to problems with their technology, but how they applied that
technology, the information they attached to a particular drug, and
how they dealt with the regulatory process.
Garabedian joined Celgene Corporation in 2007, feeling they were on
the same trajectory of success as Gilead, and became Vice President of
Corporate Strategy. He led its first commercial acquisition of a
company called Pharmion. He joined the board of directors of AVI (the
predecessor of Sarepta) in June 2010 and became its president and CEO
in January 2011.
So far, things have worked out pretty well at Sarepta. With a
cutting-edge technology moving toward FDA approval, it had the top
performing stock of all 2012. It rarely used to break over a market
value of 400 million dollars, and now it has been operating at a
valuation between eight hundred million to 1.8 one billion. Garabedian
does not want a buyout, and tells investors that the company is
woefully undervalued, so they should stick with it for the next five
or ten years.
Garabedian nurtures grand ambitions for Sarepta, and for himself. He
learnt from two highly successful chief executive officers, and so
instead of staying as part of a good management team, wanted the
opportunity to build the next great company, the equivalent of a
Google or Apple: "What I'm driven by and what motivates me is to apply
my experience in this industry, to take a technology that can be the
next revolution in medicine. If I can see this developed to treat even
one serious disease like Duchenne, I will feel pretty good. I think
this technology can be applied to many diseases. We all strive to be
part of being something bigger than ourselves. This industry, this
technology and this company give me the opportunity to do that. I hope
my children will be proud of it, that they may have sacrificed time
with their dad for something bigger."
- See more at: http://www.mirrorspectator.com/2014/08/21/biotech-and-ebola-in-cambridge-chris-garabedian-ceo-of-sarepta/#sthash.YfyhsnQV.dpuf
From: A. Papazian
NEWS | AUGUST 21, 2014 11:04 AM
________________________________
By Aram Arkun
Mirror-Spectator Staff
CAMBRIDGE, Mass. - Recently, the Ebola virus has been in the news
almost every day, with panicked speculation about the danger of its
spread, and the ethics of treatment. There is no tested and proven
treatment or vaccine available. Christopher "Chris" Nishan Garabedian,
the president and chief executive officer (CEO) of Sarepta
Therapeutics, is frequently sought out by the media in these
discussions. It happens that his company is one of only a handful with
drugs that could treat Ebola victims. Garabedian, in his Cambridge
headquarters, explained that in fact this drug is only one example of
what his company can accomplish. Sarepta employs an approach which is
part of a revolution in biotechnology, using Ribonucleic Acid (RNA) to
specifically target diseases and genetic problems.
This is a different technology from the ZMapp combination of several
antibodies against the Ebola virus, which was in the news because it
was given to two American missionaries infected by Ebola recently.
These antibodies attempt to allow a person's own immune system to
identify and neutralize the virus. However, they had never been given
to humans before, while Sarepta's drugs have been used on non-human
primates and in Phase 1 safety trials for the Federal Drug
Administration (FDA), with promising results. The trials were
supported by the Department of Defense but economic issues halted the
program.
The problem is that treatments for many diseases which are usually
restricted in the numbers of victims do not economically attract
investments necessary to support research and trials. For this reason,
drugs intended to treat rare diseases affecting fewer than 200,000
people in the US are called orphan drugs. Garabedian said, "I think
the US government knows, as governments around the world know, that to
leave it up to the private sector to develop technologies and medical
countermeasures against these threats, whether pandemic threats that
spread naturally or biological ones that someone can manufacture in a
garage, is not feasible. They know they have to foot the bill. The
problem is that they are not funding it fully." He pointed out that
crises come and go, so specific diseases float in and out of the
public's awareness along with a sense of urgency, but the potential
threats remain.
For Ebola in particular, his company has enough supply ready only to
treat 100-150 people, if the FDA would give emergency authorization,
along with permission from the Department of Defense which had
provided initial funding. In case Ebola does spread on a greater
scale, he said, "We hope that this is raising awareness that we have
the technology but need to raise the manufacturing ability to a higher
scale."
The same is true of course for Ebola drugs using different approaches
which are being developed by a few other companies, like Tekmira's
TKM-Ebola, which the FDA very recently allowed to be used on infected
people. However, Garabedian exclaimed that "regardless of what press
or market reactions have been, we still think that we have the most
advanced and robust data set for safety and efficacy that is out there
for Ebola."
There are many companies utilizing various types of technologies that
attempt to modify or suppress RNA. Sarepta uses phosphorodiamidate
morpholino oligomers (PMOs), stable synthetic chemical structures
similar to RNA. They can be used to create drugs specific for either
human or pathogen RNA and can also target specific tissues. PMOs
increase or decrease the production of a protein involved in a
disease. In the case of Ebola or other viruses, the PMOs block access
of the virus to human cellular machinery and thus inhibit its
replication.
Despite the attention that it is garnering lately, the work on Ebola
or related viruses like Marburg is not the main focus of Sarepta's
commercial research. Instead, it is pursuing PMO-based therapeutics
actively for a form of muscular dystrophy called Duchenne (DMD), which
is caused by a mutation or error in the gene for dystrophin, a protein
necessary to protect human cells during muscle function. The PMO
treatment in this case deals with human and not virus RNA, and is
targeted to skip defective mutated sections of the dystrophin gene to
allow the body to make functional dystrophin protein. It can be
targeted to specific mutations, and thus treat different varieties of
the disease. Phase IIb clinical trials on safety and efficacy have
already been done on one of these drugs, eteplirsen, meaning that it
is advancing in the FDA process needed for government approval.
Garabedian stated that the reason for the focus on DMD is that
"Duchenne is a commercially viable market, high on medical need."
Furthermore, the same technology can be used for a series of related
drugs. Garabedian said, "It is the consummate personalized medicine."
This is novel for regulators like the FDA. He continued: "I would say
that this technology demands a new approach from the FDA...If you read
that we have had ups and downs, it is because the FDA sometimes gets
more conservative the more advanced and novel the technology is. I
have been characterized as being aggressive or pushing too hard, but
all we were doing is arguing on behalf of patients who need this
technology."
Sarepta needs at least one successful breakout drug to allow it to
expand the scale of its finances and work. Afterwards, it can expand
its PMO-based approach to many different diseases and genetic
problems, and will be in an enviable position. For example, among its
other ongoing projects, Sarepta is testing a flu drug using the PMO
approach.
There are many companies working on RNA therapeutics with different
approaches. While some others also work with PMOs, Garabedian said,
"We have the lion's share of the real estate of morpholino
intellectual property. We also have the most know-how on scale-up
manufacturing. While we don't have a 100 percent lock on all
morpholino, we are the only one in advanced drug development. We have
patents."
Born in 1966 and raised in the Washington DC area, Garabedian is
Armenian on both his mother and father's sides. His parents were born
in the US but his grandparents fled the Armenian Genocide to the US
via Ellis Island and Cuba. He did not get to know his grandparents
well, as they died either before he was born or while he was still
young. Garabedian's parents tried to instill Armenian culture in their
children, but by the time they came to Chris, it became too difficult.
For this reason, Garabedian said, "Peter Balakian's Black Dog of Fate
resonated with me. I too grew up with American cultural mores, and
came to appreciate my Armenian heritage as an adult." He started to
read books, and saw movies like Atom Egoyan's Ararat, and encountered
in the popular culture efforts at documenting the Armenian plight. He
did work for around a year from 1998 to 1999 for an Armenian, Vaughn
Kailian, CEO and president at COR Therapeutics, but otherwise has had
little opportunity to cultivate his Armenian connections in his
profession.
Garabedian had a business background in college and initially worked
in market research, brand management and new product development. He
described his involvement in biopharmaceuticals as "a little bit of
serendipity." He began learning about this industry in a consulting
company, and from 1994 worked at Abbott Laboratories in Chicago, which
"was a great training ground....Then Gilead Sciences called in 1997 and
I joined them. This was my formative experience in the biotech
industry. They really shaped a vision. At the time, they were less
valued than Sarepta is today. They had a few more employees...They have
about a 140 billion dollar market capitalization today. They built it
on a relatively small footprint. ... I really learned what it takes to
build a successful biotech, how to build in a lean, smart way, and
good, smart drug development. I learned from my mentors, like John
Martin as CEO of Gilead."
Garabedian found out through experience that many companies failed not
due to problems with their technology, but how they applied that
technology, the information they attached to a particular drug, and
how they dealt with the regulatory process.
Garabedian joined Celgene Corporation in 2007, feeling they were on
the same trajectory of success as Gilead, and became Vice President of
Corporate Strategy. He led its first commercial acquisition of a
company called Pharmion. He joined the board of directors of AVI (the
predecessor of Sarepta) in June 2010 and became its president and CEO
in January 2011.
So far, things have worked out pretty well at Sarepta. With a
cutting-edge technology moving toward FDA approval, it had the top
performing stock of all 2012. It rarely used to break over a market
value of 400 million dollars, and now it has been operating at a
valuation between eight hundred million to 1.8 one billion. Garabedian
does not want a buyout, and tells investors that the company is
woefully undervalued, so they should stick with it for the next five
or ten years.
Garabedian nurtures grand ambitions for Sarepta, and for himself. He
learnt from two highly successful chief executive officers, and so
instead of staying as part of a good management team, wanted the
opportunity to build the next great company, the equivalent of a
Google or Apple: "What I'm driven by and what motivates me is to apply
my experience in this industry, to take a technology that can be the
next revolution in medicine. If I can see this developed to treat even
one serious disease like Duchenne, I will feel pretty good. I think
this technology can be applied to many diseases. We all strive to be
part of being something bigger than ourselves. This industry, this
technology and this company give me the opportunity to do that. I hope
my children will be proud of it, that they may have sacrificed time
with their dad for something bigger."
- See more at: http://www.mirrorspectator.com/2014/08/21/biotech-and-ebola-in-cambridge-chris-garabedian-ceo-of-sarepta/#sthash.YfyhsnQV.dpuf
From: A. Papazian