BIOWORLD TODAY

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THE DAILY BIOPHARMACEUTICAL NEWS SOURCE

FEBRUARY 11, 2015

BIOTECHS MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARS

VOLUME 26 , NO. 28
SPECIAL REPRINT

Beyond the hype, role of microbiome a long-term investment space

By Jennifer Boggs, Managing Editor
NEW YORK Its probably not an exaggeration to say theres
a lot of hype surrounding the microbiome space, noted Mark
Breidenbach, of H.C. Wainwright & Co., who moderated a panel on
the subject at the annual BIO CEO & Investor Conference.
Though in its infancy, the microbiome already has spawned a
number of biotech start-ups just in the past year. Collectively, they
raised about $170 million in venture funding in 2014, Breidenbach
said.
And that hype is understandable. Though still in its early days,
research on the microbiome the idea that the ecosystem within
the digestive system plays a role in both digestive and nondigestive
diseases and in overall health has exploded. Barely a week goes
by without new data reported. Just last week, in fact, the BBC News
reported that a woman who had received a fecal transplant from
her daughter to treat Clostridium difficile infection immediately
began gaining weight.
She developed an obesity-like phenotype, said panelist Karim
Dabbagh, chief scientific officer at Second Genome Inc. So clearly
the microbiome can transfer traits.
But how and to what extent is still not known. Even microbiology
expert Martin Blaser, of the New York University Langone Medical
Center, author of the book, Missing Microbes: How the Overuse of
Antibiotics Is Fueling Our Modern Plagues, and 2014 Daily Show
guest, conceded that our knowledge is just beginning.
Trying to define characteristics of a healthy microbiome, for example,
also gets tricky. Blaser compared it to the Supreme Courts take on
pornography, that we know it when we see it.
But the opportunity, he said, is there. He offered a few examples
such as one smoker developing lung cancer while another doesnt
and one patient treated for cancer responding while a second
patient, on the exact same therapy doesnt respond. I believe more
and more that well find the answers by studying the microbiome,
he said.
The difference with the microbiome space, compared to other
much-hyped approaches such as gene therapy, which took years
and multiple failures to yield a first approval, is that the technology
for studying the microbiome is already available.
The technology is fully functional in terms of trying to understand
what the microbiome is doing, said Dabbagh, whose company,

Second Genome, is tapping into the microbiome to treat

inflammatory and metabolic diseases.
Researchers are able to type microflora, identify the genes
expressed and can modulate and modify bacteria, he added.
So even though it might be hyped, that doesnt necessarily
mean we dont understand certain functionalities.
And that hype is not getting in the way of trying to take
products forward.
South San Francisco-based Second Genome disclosed last
month that it was moving lead candidate SGM-1019 into
a phase I multiple-ascending-dose trial in inflammatory
bowel disease (IBD). The drug is believed to work by hitting
a target identified by the company as a driver of IBD when
modulated by changes in the microbiome.
Nor has the early stage of microbiome work held off big
pharma. Johnson & Johnsons Janssen unit recently inked a
deal with microbiome company Vedanta Biosciences Inc. to
develop and commercialize a candidate for IBD. That deal
called for an undisclosed up-front payment and up to $241
million in milestones to Vedanta, plus possible additional
consideration related to commercialization.
Johnson & Johnson Innovation previously made an initial
investment in Vedanta in June 2013.
Were very interested in trying to correct the loss of diversity
in the microbiome, said Michael Elliott, vice president
of immunology scientific innovation at J&J Innovation in
Boston. The Vedanta deal also reflects our therapeutic
interests of immunology and immune-mediated disorders.
BEYOND THE GUT

So the role of the microbiome in disease is clear, said Second

Genomes Dabbagh.
The effort now is in trying to elucidate that role, figure out
how it affects diseases such as IBD, type 2 diabetes, obesity
and other indications.
But the potential of the microbiome could go far beyond
intestinal applications.
Its activity also is found in our nasal passages, also in our
skin, said Jeffrey Riley, president and CEO of Synthetic

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WEDNESDAY, FEBRUARY 11, 2015

BIOWORLD TODAY

Biologics Inc. The organisms have co-evolved with us of a

millennium. They all have a function; we just dont know what.
For now, though, Rockville, Md.-based Synthetic Biologics is
tackling the microbiome itself as its lead program. The firm
reported top-line results Monday from a phase Ib trial of SYN004, an oral beta-lactamase enzyme designed to protect
the microbiome and prevent C. difficile infection, antibioticassociated diarrhea and secondary antibiotic-resistant infections
in patients receiving intravenous beta-lactam antibiotic therapy.
The trial in healthy volunteers showed no safety or tolerability
issues. Pharmacokinetic data from the study are expected later
this quarter.
Synthetic Biologics also has an oral statin treatment aimed at
treating constipation-predominant irritable bowel syndrome
by reducing the impact of methane-producing organisms. Too
much methane in the small intestine and large intestine can
cause constipation, Riley explained, adding that the firm would
be starting phase II testing shortly.
Its likely that digestive-related disorders will lead the way, at
least clinically.

PAGE 2 OF 2

From an investor standpoint, the low-hanging fruit involves

the colonic and ileal diseases, said Eric de La Fortelle, venture
partner at Seventure Partners, which last year established a
$164.1 million fund to focus on commercializing emerging
research in the workings of the microbiome and its applications
in therapeutics, biomarkers and companion diagnostics. (See
BioWorld Today, Feb. 12, 2014.)
But beyond those indications lie the tantalizing pieces of
evidence showing connection of the microbiome to diseases
such as diabetes and anorexia, he said.
The regulatory guidances likely will have to evolve for microbial
products, and the industry also will have to figure out the
intellectual property (IP) issues, though for the latter, there
might at least be a little leeway.
Particularly in the U.S., we dont know how strong IP protection
will be, said J&Js Elliott. But the 12-year exclusivity for biologics
included in the Affordable Care Act might apply to microbial
products, too. So one might end up with IP protection or 12
years exclusivity [under ACA] or both, but Im not sure the IP is
guaranteed.

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