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BIOWORLD TODAY
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THE DAILY BIOPHARMACEUTICAL NEWS SOURCE
JANUARY 11 , 2016
BIOTECH’S MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARS
ANTI-BOOKMARK DRUG PUT TO TEXT
BIOPHARMA OPTIMISTIC, FLUSH WITH CASH
Despite ‘choppy’ markets, fundamentals
strong as health care world heads to JPM
By Marie Powers, News Editor
SAN FRANCISCO — As investors in biopharma, medtech and affiliated industries
descend on San Francisco for the start of the 34th Annual J.P. Morgan Healthcare
Conference (JPM), invited guests and throngs of hangers-on could be forgiven for
glancing nervously over their shoulders at the global capital markets. In the wake of
geopolitical and macroeconomic uncertainties – currency and stock roiling in China,
oil-fueled power brokering in the Middle East, election year politicking in the U.S.
and saber-rattling in North Korea – the first trading days of 2016 took investors on
$535M Roche cancer
BET more than
‘Tensha’ relief; key to
TNBC in NUTshell?
By Randy Osborne, Staff Writer
With a phase II trial in stubborn triplenegative breast cancer (TNBC) set to
begin soon and results already disclosed
against nuclear protein in testis (NUT)
midline carcinoma, bromodomain
See JPM, page 3
FINANCINGS
DEALS AND M&A
PRESENTING AT J.P. MORGAN
Zai’s series B brings
$100M-plus for R&D in
China, global markets
By Shannon Ellis, Staff Writer
SHANGHAI – Zai Labs Ltd., of Shanghai,
has done it again, this time raising more
than $100 million from seasoned health
care venture firms led by Advantech
By Michael Fitzhugh, Staff Writer
Mylan NV and Momenta Pharmaceuticals
Inc. agreed to work together to develop,
manufacture and commercialize six of
Momenta’s current biosimilar candidates
under an exclusive new deal.
The arrangement provides Mylan with a
maturing biosimilars platform that could
Read this week’s edition
Nice slice of ‘mud’ pie
as Accelerator launches
Lodo with $17M series A
By Marie Powers, News Editor
The New York arm of Accelerator Corp.
launched its second biopharma in less
than a week as Lodo Therapeutics
Corp. set out with a $17 million series A
financing. The round included the same
See Lodo, page 7
See Momenta, page 6
THE BIOWORLD BIOME
BioWorld Science Editor Anette
Breindl takes a closer look at
translational medicine
See Tensha, page 4
NEWCO NEWS
Mylan, Momenta strike
$245M biosimilars deal
See Zai, page 5
BENCH PRESS
VOLUME 27, NO. 6
IN THE CLINIC
THE YEAR FOR NANOMEDICINE?
CALL ME MAYBE
Cardiac variants fail
to predict risk in
prospective study
By Anette Breindl, Senior Science Editor
In findings that illustrate the gap
between risk variants and clinical
disease, scientists from Vanderbilt
See Risk, page 8
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Nanobiotix launches
U.S. trial, enters hot
immuno-oncology field
By John Brosky, Contributing Writer
PARIS – At the start of what promises to
be a big year for nanomedicine developer
Nanobiotix SA, of Paris, the company
reported approval for its first clinical
See Nanobiotix, page 9
MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
FINANCINGS
Aequus Pharmaceuticals Inc., of Vancouver, British Columbia,
said it intends to complete a nonbrokered private placement
in the U.S. of up to 2 million shares and a nonbrokered public
offering in Canada up to 3.5 million shares at a price of C50
cents (US35 cents) each, for aggregate gross proceeds of up to
C$2.75 million. Aequus intends to use funds for research and
development and general working capital purposes. The deal is
expected to close Jan. 12.
Ardelyx Inc., of Fremont, Calif., priced a public offering of 7.5
million shares of its common stock at a public offering price of
$10 each for gross proceeds of $75 million. The firm granted
the underwriters the right to purchase up to about 1.12 more
million shares of common stock at the public offering price.
Ardelyx intends to use its existing cash and cash equivalents
and the net proceeds of the offering to support the tenapanor
and RDX022 phase III programs, including manufacturing of
clinical trial materials, as well as to support the investigational
new drug application filing for RDX009 and to fund additional
research and development for its earlier-stage programs.
Citigroup and Leerink Partners LLC are acting as joint bookrunning managers. Wedbush Pacgrow is acting as lead
manager, and JMP Securities LLC, Cantor Fitzgerald & Co. and
Ladenburg Thalmann are serving as co-managers. Shares of
Ardelyx (NASDAQ:ARDX) closed Friday at $10.72, up 63 cents.
Collegium Pharmaceutical Inc., of Canton, Mass., priced
a public offering of about 2.8 million shares of its common
stock at $20 per share. Gross proceeds are expected to be $55
million. In addition, Collegium granted the underwriters a
30-day option to purchase up to an additional 412,500 shares
of common stock. The offering is expected to close Jan. 13.
Jefferies and Piper Jaffray are acting as joint book-running
managers. William Blair and Needham are acting as co-lead
managers, while Janney Montgomery Scott is acting as a comanager. Shares of Collegium (NASDAQ:COLL) closed Friday
at $19.72, down $1.41. (See BioWorld Today, Sept. 14, 2015.)
Zymeworks Inc., of Vancouver, British Columbia, closed a
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Pihl-Carey (Database Editor), Ann Duncan (Senior Production Editor),
Marie Powers (News Editor), Randy Osborne (Staff Writer) // East Coast Anette Breindl (Senior Science Editor), Mari Serebrov (Regulatory Editor) //
West Coast - Michael Fitzhugh (Staff Writer) // Europe - Nuala Moran &
Cormac Sheridan (Staff Writers) // Asia: John Fox, Shannon Ellis, Catherine
Makino, Alfred Romann, Dave Silver, Cornelia Zou (Staff Writers)
PAGE 2 OF 15
$61.5 million series A mezzanine financing to support the
advancement of its Azymetric therapeutics pipeline. The
financing was supported by a syndicate of U.S. and Canadian
institutional investors. It was co-led by new investors BDC
Capital and Lumira Capital and joined by existing Zymeworks
investors Indianapolis-based Eli Lilly and Co., Summit, N.J.based Celgene Corp., CTI Life Sciences Fund and the Fonds de
solidarité FTQ. New investors in the financing include Perceptive
Advisors, Teralys Capital, Northleaf Venture Catalyst Fund,
Brace Pharma Capital, Merlin Nexus and others. Proceeds of the
financing will support clinical development of Zymeworks’ lead
candidates, the potential cancer therapies ZW25 and ZW33,
which the company plans to move into clinical development
later this year. In separate news, Zymeworks inked a deal making
an undisclosed equity investment in Kairos Therapeutics
Inc., also of Vancouver, which specializes in the discovery and
development of antibody-drug conjugates. Under the terms,
Zymeworks and Kairos also have the option to merge to further
integrate their respective platforms, resources and pipelines
to accelerate the development of cancer biotherapeutics. (See
BioWorld Today, Jan. 22, 2015.)
OTHER NEWS TO NOTE
Ampliphi Biosciences Corp., of San Diego, said it acquired
key assets from U.K.-based Novolytics Ltd., including
bacteriophage-related intellectual property, bacteriophage
libraries, formulation and regulatory know-how as well as GLP
toxicology data. Terms were not disclosed.
Ariad Pharmaceuticals Inc., of Cambridge, Mass., said its
partner, Otsuka Pharmaceutical Co. Ltd., of Tokyo, submitted
a new drug application to the Japanese Pharmaceuticals
and Medical Devices Agency seeking approval for Iclusig
(ponatinib) for the treatment of resistant or intolerant chronic
myeloid leukemia and Philadelphia chromosome-positive acute
lymphoblastic leukemia. Ariad expects Otsuka to receive a
decision on the new drug application in Japan in the second half
of 2016.
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BIOWORLD™ TODAY
JPM
Continued from page 1
a wild ride, and they might need to hold onto their hats for the
foreseeable future.
Despite inching up early Friday, the Dow Jones Industrial
Average closed in the red at 16,346.45, shedding more than 6
percent of its value during the first week of the New Year. The
Nasdaq Composite – arguably the sector’s more important
index – was off more than 7 percent in the week leading to
JPM after losing more ground Friday to close at 4,643.63. The
Nasdaq Biotechnology Index, the subset of 190 of the sector’s
biggest names, also remained in the red Friday for a one-week
drop of nearly 11 percent, finishing at 3,160.21.
Still, the losses didn’t prevent a stunning number of
biopharmas from turning to the markets with IPO filings during
the first week of 2016 – eight offerings collectively seeking
some $680 million – while 22 additional companies filed or
priced follow-ons set to exceed $2.6 billion. (See BioWorld
Today, Jan. 7, 2016, and Jan. 8, 2016.)
“Although biotech investors have headed for the sidelines for
now, the industry’s fundamentals remain strong,” said Peter
Winter, editor of BioWorld Insight and a seasoned observer of
the industry’s financial and business trends.
“Investors will be listening closely to company presentations at
JPM on how executives plan to deliver value going forward,” he
observed. “After a massive inflow of cash in 2015, biopharma
companies are well placed to pull the trigger on acquiring
assets through M&A or ambitious partnering deals, in addition
to the usual share buybacks. If investors like what they hear,
expect them to return to the fold later this month.”
WHAT WILL BIOPHARMA DO WITH ALL THAT CASH?
The industry is poised to see more multibillion-dollar mergers,
which could be announced as early as this quarter – even
during JPM, as the long-simmering offer by Shire plc, of Dublin,
to acquire Baxalta Inc., of Bannockburn, Ill., heated up again
last week. (See BioWorld Today, Aug. 5, 2015.)
Winter also pointed to Gilead Sciences Inc., of Foster City, Calif.,
which is sitting on $10 billion raised in September 2015, only a
fraction of which was used last month to purchase rights to the
Janus kinase 1-selective inhibitor, filgotinib (GLPG0634), from
Galapagos NV, of Mechelen, Belgium. (See BioWorld Today,
Dec. 18, 2015.)
Analysts had questions about what biopharmas will do with all
that cash – 70 pages worth from the J.P. Morgan Healthcare
team. For example, the team wants Alkermes plc, of Dublin,
to explain how the company will prioritize the use of its $800
million in cash in light of its ongoing phase III program for
ALKS 5461 in major depressive disorder and upcoming pivotal
programs for multiple sclerosis candidate ALKS 8700 and
schizophrenia drug ALKS 3831. (See BioWorld Today, Feb. 25,
2015.)
PAGE 3 OF 15
For Celgene Corp., of Summit, N.J., the question is more
about capital allocation and providing the most value from
its cash, reported as $7.5 billion as of Sept. 30. For Dynavax
Technologies Corp., of Berkeley, Calif., which posted upbeat
phase III data last week on its hepatitis B vaccine, Heplisav-B,
the J.P. Morgan team asked whether the cash runway is
sufficient to finance the product launch. (See BioWorld Today,
Jan. 8, 2016.)
For Vertex Pharmaceuticals Inc., of Boston, which is “sitting
on a nice sum of cash,” inquiring minds want to know about
capital allocation – whether the company plans “to keep this
cushion or consider M&A/additional collaborations,” asked the
J.P. Morgan analysts. In October, Vertex paid $105 million up
front, including $75 million in cash and a $30 million equity
investment, to Crispr Therapeutics AG, of Basel, Switzerland, on
a collaboration to discover and develop treatments for genetic
diseases, including cystic fibrosis and sickle cell disease, that
could ultimately exceed $2.6 billion. (See BioWorld Today, Oct.
27, 2015.)
For Mannkind Corp., of Valencia, Calif., questions focus more
on prospects for solvency. Last week, the company lost its
commercial partnership with Paris-based Sanofi SA, for the
inhaled insulin product, Afrezza, and saw shares plummet. The
J.P. Morgan analysts want to know how long the company’s
cash resources will last, how to think about its capital structure
and future financing needs and whether it can raise additional
capital in Israel. (See BioWorld Today, Jan. 6, 2016.)
‘WE’RE LOOKING FOR THE SAME OLD-FASHIONED STUFF’
Although pundits like to look back when assessing the mood at
JPM, participants remain scrupulously consistent. They arrive
with meticulous meeting lists and focus on checking off their
boxes as they slog cheek-to-cheek through the congested halls
of the Westin St. Francis – indeed, through the streets, eateries
and hotels lining Union Square.
There’s no template for success at JPM. The agendas are as
varied as the attendees.
“It’s always a little bit of zoo,” admitted Oleg Nodelman,
founder and managing director of Ecor1 Capital LLC. “But
everyone in the world of health care comes to San Francisco, so
it’s amazingly efficient.”
As he always does, Nodelman will look for “the most interesting
things” – processes, technologies and data – to stand out while
the rest of the PowerPoints fade into background noise.
“We’re fundamentally driven so we’re looking for the same
old-fashioned stuff,” he told BioWorld Today, stressing two of
his favorite questions: “What is the market missing? Or is the
market not missing anything? There’s no magic buzzword.
We’re always looking for companies that are well funded – you
can’t save your way to success in this sector – but at the same
time we like companies that are spending money prudently.”
A staggering amount of public and private capital flowed
See JPM, page 10
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
PAGE 4 OF 15
Tensha
malignancies and advanced solid tumors. (See BioWorld Today,
Dec. 19, 2014.)
and extra terminal domain (BET) inhibitor specialist Tensha
Therapeutics Inc. drew Roche AG to the table for a takeover
valued at up to $535 million.
“Hopefully, we’ve provided them with the foundation of
monotherapy data to take [lead small-molecule TEN-010]
forward in a very smart way, as they’ve historically done in
[other] fields of novel mechanisms,” CEO Douglas Onsi told
BioWorld Today. In Tensha’s hands, the compound has reached
phase I trials in solid tumors and hematological malignancies.
BET drugs bear promise in combination regimens for cancer,
too, making Basel, Switzerland-based Roche a good fit, he said.
The pair is expected to make public their deal today and tie up
loose ends in the first quarter of this year. Specifically, Roche
agreed to pay $115 million up front and as much as $420 million
in milestone payments related to clinical and regulatory goals.
Founded by James Bradner, formerly of the Dana-Farber Cancer
Institute, and managed and funded by Healthcare Ventures,
Tensha brings an epigenetic technology that disrupts BET
proteins, which contribute to cancer cell memory by binding
to the genome as molecular “bookmarks.” An investigatorinitiated phase II trial with TEN-010 in TNBC is set to open at
Dana-Farber shortly. Results in NUT midline carcinoma were
offered by the AACR-NCI-EORTC International Conference on
Molecular Targets and Cancer Therapeutics in November. (See
BioWorld Today, Jan. 7, 2016.)
The chemical probe for BET, JQ1, was freely shared by Bradner
– over the last five years, more than 500 labs have received
it – “with the expectation that the global research community
could provide more scientific support and direction for our
clinical candidate more efficiently and effectively than any
one company, particularly a venture capital-backed biotech
company, could do on its own,” Onsi said. “It’s been a
tremendous success. We and the entire field have benefited
from the publications and the enthusiasm that all of those
investigators have had for JQ1 and for BET inhibition.”
Bradner also was the originator of suberohydroxamic acid
(4-methoxycarbonyl) phenyl ester, or SHAPE, the topical
histone deacetylase inhibitor that ended up with Tetralogic
Pharmaceuticals Corp., of Malvern, Pa., which recently began
an interim analysis of a randomized phase II experiment. The
trial was designed to investigate the safety and efficacy of three
SHAPE dosing regimens in patients with earlier-stage cutaneous
T-cell lymphoma. Onsi said both of Bradner’s programs “went
from his academic labs to clinical trials in two years, and in both
cases were able to demonstrate proof-of-concept data within a
couple of years thereafter.” (See BioWorld Today, Jan. 8, 2016.)
Roche’s move is not the first pharma bid in BET inhibitors.
Kenilworth, N.J.-based Merck & Co. Inc. bought Oncoethix SA,
of Lausanne, Switzerland, for $110 million up front and $265
million in potential milestone payments in late 2014. Oncoethix
had advanced OTX015 to phase Ib trials for hematological
RESISTANCE COULD LIE AHEAD
Continued from page 1
Farthest along clinically in BETs is Resverlogix Corp., of Calgary,
Alberta. In November, the firm dosed the first patient in the
phase III trial called BETonMACE with lead drug apabetalone
(RVX-208) in high-risk patients with coronary artery disease and
type 2 diabetes. The primary outcome measure will assess the
effect of apabetalone on time to first occurrence of major adverse
cardiovascular events (MACEs). In the fall of 2014, Resverlogix
disclosed data showing the compound gained a 77 percent
reduction in MACEs in diabetic patients. Resverlogix spinout
Zenith Epigenetics Inc., of San Francisco, has next-generation
oncology work under way with BET inhibitors.
Others active in the space include Aptose Biosciences Inc., of
San Diego; Foster City, Calif.-based Gilead Sciences Inc.; and
Incyte Pharmaceuticals Inc., of Wilmington, Del. As long ago
as 2013, analyst Andrew Fein with H.C. Wainwright tagged
BET inhibitors as “the most underappreciated early stage
small-molecule class in oncology. Since then,” he wrote in an
April 2014 report, “the BET space has crowded fast, with many
new small companies emerging and several big pharmas also
joining the movement with in-house programs. We count at
least 16 active BET inhibitor programs, although, notably, the
number of companies that have recently ‘dabbled’ in the space
may be twice as large.”
But a paper in Nature recently tempered the optimism,
pointing out that “tumor cells are likely to become resistant
to these drugs. Anticipated mechanisms of resistance have
now been described.” Several reports lately suggest that “BET
inhibitors might have a broader potential than had previously
been realized,” the paper conceded. “They also highlight the
possibility that BET inhibitors could be used in combination
with other drugs to overcome both innate and acquired drug
resistance. Although the reported resistance mechanisms seem
to reflect an adaptation to drug pressure, the root cause of
resistance remains unknown. Does a specific mutation cause
Wnt or CK2 activation, or are these adaptive changes that
drive resistance through reversible epigenetic mechanisms? A
complete mechanistic understanding of resistance remains to
be defined.” //
OTHER NEWS TO NOTE
Bamboo Therapeutics Inc., of Chapel Hill, N.C., said it acquired
the viral gene therapy manufacturing facility (Vector Core) from
the University of North Carolina at Chapel Hill. Financial details
were not disclosed. The Vector Core was founded in 1993 as a
full-service viral vector production organization with extensive
experience in vector design and process development, as
well as manufacturing of research and clinical-grade vectors.
Bamboo has developed a suspension cell-based production
platform that utilizes serum free/chemically defined media to
increase scalability, efficiency and purity.
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
Zai
Continued from page 1
Capital and Orbimed. The trio of Zai’s series A backers –
Qiming Ventures, Sequoia and TF Capital – also continued their
support in this second round.
Zai is a biopharma focused on discovering and developing
new medicines for oncology, inflammatory and autoimmune
diseases for the China market but also with potential for global
markets. The Chinese character for Zai, which means “once
more” or “again,” refers to the history many of its key people
have had in China’s relatively fledgling biotech space, namely
with pioneering firm Hutchison Medipharma.
Not least among that group is Samantha Du, former head of
Hutchison and CEO and founder of Zai. Her goal from the outset
has been to establish a globally respected Chinese biopharma
capable of working to the highest industry standards, and Zai’s
in-licensing deals and strategic moves show the firm has earned
the confidence to be considered among the most relevant
players in the industry after just a few short years. Firms such as
Sanofi SA, UCB SA and Hanmi Pharmaceuticals Co. Ltd. have all
felt comfortable signing over key assets to Zai for local and/or
global development. (See BioWorld Today, Aug. 20, 2014, Sept.
23, 2015, and Nov. 25, 2015.)
Zai’s pipeline consists of ZL2301 for hepatocellular cancer and
ZL2303 for non-small-cell lung cancer (NSCLC), both at a late
stage, entering phase III studies. Three other candidates, ZL
2302 for NSCLC, ZL2102 for asthma and chronic obstructive
pulmonary disease and ZL1101 for graft-vs.-host disease and
inflammatory bowel disease, are all in the preclinical or phase
I stage. Zai has a recently launched internal discovery program
in large molecules for oncology as well.
That diversified, multishots-at-the-target pipeline approach
has allowed Zai to be in the enviable position of picking the
cream of the crop for investment partners, benefiting from an
ongoing trend that there are often more Chinese investors on
the hunt for a good investment vehicle than there are good
Chinese companies in which to invest.
Advantech is a newly formed fund from venture capitalist
Jianming Yu, who has successfully managed one of China’s
largest funds, New Horizon. Advantech Capital will focus on
innovation-driven growth capital, investing in technology,
media and telecommunications, e-services and health care
companies in China.
“What impressed me about Advantech Capital is that Jianming
Yu and I have the same dreams,” Du told BioWorld Today. “He is
looking to make Zai one of the largest pharma companies up to
a global standard coming out of China; we hope to be the best.
He has a lot of patience stemming from his own background
and training – he obtained a doctorate in molecular biology
from Harvard – and has a global network of resources, all sure
to help our company as well.”
With that added financial leverage, Zai’s goal is to continue its
three-prong offensive: advance the pipeline in the clinic, in-
PAGE 5 OF 15
license more candidates for China (and the globe) and expand
its newly formed biological discovery unit. (See BioWorld Today,
Oct. 28, 2015.)
“We still have $20 million left [from previous rounds of
investment],” said Du. “With this over $100 million round we
have a pretty solid financial base to support the company to
move toward the next three years.”
Zai received $30 million in series A financing more than a year
ago. (See BioWorld Today, Sept. 2, 2014.)
The company also joins a relatively exclusive club of
Chinese biotechs that have entered the $100 million-plus
venture capital financing club. Also on the list are Ascletis
Pharmaceuticals Co. Ltd., Innovent Biologics Inc. and Beigene
Co. Ltd. And along with Innovent, Zai is one of the few Chinese
biotechs invited to give a company presentation at this week’s
J.P. Morgan Healthcare Conference in San Francisco.
A ‘VERY BIG AMBITION’
The latest round of financing, Du said, was driven at the outset
from repeated investor interest, prompting the firm to quietly
solicit a few investors with deep knowledge of the industry. It
did not take long to line up Advantech and Orbimed and to see
enthusiastic support from existing series A investors as well.
Du said there are many investors in China looking to enter
the biopharma field, but without the requisite scientific
background, there is a concern they may underestimate the
risks. Over the long term, that could create a dangerous bubble
as those VCs offer capital along with pressure for quick returns,
but little in the way of patience or understanding that it takes
biopharmas a long time to make a return for investors.
While Du said her investors have that patience, with decadelong investment windows, her timetable, if all goes according
to plan, may not require it. Zai is looking to start ramping up
commercial efforts in 2017, with hopes that approvals will be in
hand not long after.
Du pointed to her team’s track record at Hutchison for securing
green channel approval from regulatory authorities and
extensive experience working with the CFDA and the CDE,
as key indicators of that future success. Those relationships
also help potential licensing deals as well, since Zai has
demonstrated local know-how in getting candidates over
China’s considerable regulatory hurdles.
“We hope to license a few more blockbuster drugs – early stage
global rights and later-stage China rights – because the team
by now has very strong capabilities, very good communication
with the CDE and very strong operation and technical
background. We want to continue this momentum and the
team has the capability to handle multiple programs,” said Du.
“Even though we are not a big pharma – we consider ourselves
a medium-sized biotech – we have the know-how to ask the
right questions, which has impressed our global partners. We
have a very big ambition,” she added. “That is why the $100
million can help continue our efforts. It is the right valuation
with the right investors; we really think it is a great move.” //
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
Momenta
Continued from page 1
fuel future growth, while giving Momenta access to a global
distribution and commercial capacity that would be hard to
build on its own.
Mylan will pay Momenta $45 million up front and as much as
$200 million in six contingent early development milestone
payments. Each company will share equally in the costs of the
collaboration and in its profits.
The deal also provides Mylan CEO Heather Bresch with an
opportunity to extend and build upon what Momenta President
and CEO Craig Wheeler said was a close alignment between the
two executives on their vision for the future of the biosimilars
marketplace, an area in which they’ve worked together in
recent years to hammer out favorable policies via participation
in biosimilars industry groups.
Shares of Momenta (NASDAQ:MNTA) closed Friday at $14.48,
up 92 cents, while Mylan shares (NYSE:MYL) dipped, losing
$2.19 before closing at $49.42.
At the head of the deal is Momenta’s lead biosimilar candidate,
M834, a version of the rheumatoid arthritis (RA) drug Orencia
(abatacept, Bristol-Myers Squibb Co.). Clinical trials testing the
biologic are expected to begin midyear. The deal also includes
five other biosimilars for which only the following names were
shared: M615, M706, M710, M730 and M740. Wheeler said all
six programs target branded biologics with aggregate worldwide
2014 sales of greater than $12 billion and could be expected to
launch between 2020 and 2025, starting with M834.
Evercore ISI analyst Umer Raffat said that, based on Momenta’s
patent filings, it’s possible that the remaining five products
may include biosimilars for Rituxan (rituximab, Biogen Inc./
Roche AG), Prolia/Xgeva (denosumab, Amgen Inc.), Vectibix
(panitumumab, Amgen Inc.), Xolair (omalizumab, Genentech
Inc./Roche AG) and Campath (alemtuzumab, Sanofi SA).
The Orencia biosimilar holds big potential for the partners
due to a combination of factors. First, it’s the only CTLA4-Ig
fusion protein approved for use in RA and juvenile idiopathic
arthritis, a position that helped it generate worldwide revenues
of nearly $1.7 billion in 2014. Furthermore, there could be a fair
bit more money on the table: With several other indications in
development, a consensus forecast including models from seven
analysts predicts global sales will peak at $2.4 billion by 2019,
followed by the expiration of a U.S. composition of matter patent
on the drug that year and a very gradual projected decline in
sales after that. Momenta has already filed for an inter partes
review (IPR) challenging BMS’s formulation patent and expects
the Patent Trial and Appeal Board to issue the IPR this month.
Although costs are split equally, the work behind the deal
is divided roughly between early and later tasks. Momenta
is taking primary responsibility for preclinical development,
process development and scale-up for all products, as well
as all clinical development activities for M834 and phase I
development activities for the five other products. It expects
PAGE 6 OF 15
to receive $60 million out of the potential $200 million
this year, Momenta’s chief financial officer, Rick Shea, said.
The extra cash could help Momenta manage its near-term
operating expense increase in the face of both its novel drug
and biosimilar development programs. Momenta will also
be responsible for all regulatory activities in the U.S. through
approval.
Mylan will be responsible for pivotal or phase III activities
for all products except M834, regulatory activities outside
the U.S. and inside the U.S. post-approval and worldwide
commercialization of all products.
Clearance under the Hart-Scott-Rodino Antitrust Improvements
Act is expected by the end of March, after which the partners
will work closely to prioritize each program.
Closing a broad biosimilar deal with economic terms that
would allow it to retain substantial downstream value has been
a top priority for Momenta, said Wheeler, during a conference
call held Friday. He said the Mylan deal accomplished not only
that but marked a “defining moment” in the growth of the
company’s biosimilar business.
Wheeler also spoke to the breadth of the company’s biosimilars
portfolio following the Mylan deal, something he said would
help smooth revenue flow and allow the company to achieve
scale, technology and regulatory synergies. The latter factors
could help Momenta achieve differentiation through early
interchangeability designations from the FDA. “Our common
goal through this collaboration is to reduce development costs
and potentially eliminate the need for significant marketing
investments once the products are launched,” he said.
Mylan’s Bresch said the collaboration with Momenta would be
“highly complementary” to Mylan’s partnership with Bangalore,
India-based Biocon Ltd., which is focused on more near-term
biosimilar opportunities. That partnership, first formed in 2009,
now includes a follow-on of Herceptin (trastuzumab, Roche AG),
launched in India in early 2014, as well as biosimilar versions
of Neulasta (pegfilgrastim, Amgen Inc.), Humira (adalimumab,
Abbvie Inc.), Avastin (bevacizumab, Genentech Inc./Roche AG),
Enbrel (etanercept, Amgen Inc.) and Neupogen (filgrastim,
Amgen Inc.), plus three insulin analogues – a generic of Sanofi
SA’s Lantus, Lispro, a generic of Eli Lilly and Co.’s Humalog and
Aspart, and a generic of Novo Nordisk A/S’ Novolog.
Although both Mylan and Momenta are optimistic about the
endeavor, their hopes could be frustrated by what has been, to
date, a slower than expected uptake for biosimilars. A decade
after the first biosimilar was approved in Europe, research
carried out by IMS Health for the European Commission has
suggested that the market still has lots of room to mature. The
report found that uptake of biosimilars has been slow there.
Furthermore, cost savings that biosimilars are expected to
deliver have not always been as dramatic or market-changing
as expected in the face of complicating factors like price
reductions for reference products or the arrival of new versions
of off-patent biologics that promise more convenient dosing.
(See BioWorld Today, Jan. 7, 2016.) //
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
Lodo
Continued from page 1
investment syndicate partners that days earlier propelled
Petra Pharma Corp. out the door: Abbvie Inc., Alexandria
Venture Investments, Arch Venture Partners, Eli Lilly and
Co., Harris & Harris Group Inc., Innovate NY Fund, Johnson &
Johnson Innovation/JJDC Inc., The Partnership Fund for New
York City, Pfizer Venture Investments, Watson Fund and Wuxi
Pharmatech. (See BioWorld Today, Jan. 6, 2016.)
This time, they were joined by the Bill & Melinda Gates
Foundation, which had a pre-existing collaboration with
Lodo co-founder Sean Brady, associate professor and head
of the Laboratory of Genetically Encoded Small Molecules at
Rockefeller University – an Accelerator partner institution.
Lodo – the Spanish word for mud – will seek to translate
discoveries from Brady’s lab, which focuses on the discovery,
biosynthesis and characterization of genetically encoded small
molecules from microbial sources, particularly those produced
by uncultured soil bacteria and the human microbiome. The
start-up is Brady’s first.
The company’s formation was a case of “perfect timing, where
people are wandering around the world and find each other,”
Brady told BioWorld Today.
His group had worked for roughly a decade on methods to
access the chemical and biosynthetic potential of uncultured,
previously inaccessible bacteria directly from environmental
samples – a field known as metagenomics.
“We had come to the point where we had shown all of the proof
of principle and were thinking of ways to try to scale this,” with
the goal of improving human health, Brady said. “We came
to the conclusion that such work had to be done outside an
academic lab.”
Two years ago, a chance meeting with the Gates Foundation
led to a collaboration on finding, identifying and proving
molecules that could have applications in infectious diseases,
such as tuberculosis, that affect patients in the world’s poorest
countries.
Around the same time, Accelerator was ramping up in New
York and seeking to marshal biotech efforts at the city’s major
research and medical institutions.
“It’s an exciting time in New York, seeing biotech develop,”
Brady said. “Accelerator provided the right people at the right
time.”
‘WE’RE GOING BACK TO THE ROOTS OF DRUG DISCOVERY’
As part of its model, Accelerator will oversee the early
operations of Lodo, which will have office and lab headquarters
in Accelerator’s facilities at New York’s Alexandria Center for
Life Science. Lodo co-founder David Pompliano – a former
senior scientist at Glaxosmithkline plc and Merck & Co. Inc.
before turning to consulting and venture investment – will
serve as interim chief scientific officer. With expertise in the
PAGE 7 OF 15
discovery and development of anti-infective and oncology
drugs, Pompliano sits on numerous scientific boards, including
the Gates Foundation Tuberculosis Drug Accelerator.
Lodo inked a license agreement with Rockefeller to expand
Brady’s technology platform, which also offers potential
therapeutic applications in oncology, metabolic disorders and
rare diseases.
“We think we have an approach that allows us to think about a
global screen for natural products,” Brady explained. “We’re no
longer limited by what one can culture or see in fermentation
broth. The idea of metagenomics is that we can go out and look
and just ask, ‘What does nature have for us?’”
Although “maybe it’s forgotten in the drug development
community,” until a few decades ago the vast majority of
therapeutic agents came from nature, he added. Metagenomics
allows scientists to peek into nature “in excruciating detail, all
the way down to its genetic code, and see very broadly and very
deeply, what’s going on.”
By using a sequencing-based approach, Brady said he believes
Lodo has the opportunity to discover molecules in nature that
are better than those discovered in the lab.
“As productive as the fermentation-based approach has been,
it’s missed most of the chemistry out there,” he maintained.
“We’re taking a systematic look to see if we can have a renewed
age of natural products. We’re going back to the roots of drug
discovery, but with modern tools.”
Thong Le, CEO of Accelerator, called Brady “an incredibly
special researcher” who developed “really, really cool
technology.”
The series A is designed to explore the therapeutic potential of
the platform, but metagenomics also has potential applications
in the food and agriculture industries and Lodo holds the
intellectual property for all uses, Le said.
“The Accelerator model allows us to take on platform
technologies and rapidly, in a step-wise fashion, refine specific
applications,” Le said. “That’s exactly the way we’ve structured
the financing for this company, so we can build on the
technology, expand upon it where appropriate and identify a
small handful of test cases.”
Whether to develop Lodo as a standalone company or spin out
various applications remains to be seen.
“All possibilities are on the table,” Le said. “We have to start
with the science. We have some very specific things that we
need to see the science do. Assuming that the technology
delivers, we have a lot of options for the company.”
For now, Lodo is focused on developing targeted therapeutic
applications, and potential partners are already calling.
“A number of firms in our syndicate and others have reached
out to us in a very aggressive way to work with the technology,”
Le said, citing prospects in pharma, biotech and other
industries. “We’re going to stay as focused as we can and let
the science lead us.” //
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
Risk
Continued from page 1
University have reported that in an unselected population, the
carriers of two gene variants that are considered potentially
pathogenic for heart disease had neither a higher incidence
of arrhythmia nor longer QT intervals than those without such
variants.
The work shows that “these genetic variants are not
deterministic,” first author Sara Van Driest, an assistant
professor of pediatrics at Vanderbilt University Medical Center,
told BioWorld Today.
When a risk variant is detected, “we are talking about
probabilities here, and not about diagnoses.”
Genome and exome sequencing is not quite yet in routine
clinical use. But with massive advances in sequencing
technology has come a deluge of genetic data.
As more and more patients undergo such sequencing, there are
important questions about what to do with so-called incidental
findings – gene variants that have no bearing on the original
reason for the testing, but might indicate a risk for another
disease.
For example, if the whole genome or exome of a cancer patient
is sequenced, the resulting sequence data also include that
patient’s ApoE variant, which predicts their risk of developing
Alzheimer’s disease. Compared to those individuals with two
copies of the protective ApoE2 variant, carriers of two highrisk ApoE4 variants have approximately 10 times the risk of
developing Alzheimer’s disease by age 75.
Most risk-conferring variants, though, have far more modest
effects. And many have been identified by testing individuals
with clinical disease.
A gene variant that is useful for an accurate diagnosis where
disease is already present, though, may not be predictive of that
same disease in a larger, unselected population.
As a result, there is a lively and ongoing debate on what to do
with incidental findings. (See BioWorld Today, Feb. 19, 2013.)
When the evidence for harmful effects of such incidental
findings is weak, there is a complex mix of factors that comes
into play with respect to reporting them. There is an argument
to be made for patient autonomy – patients have a right to their
own medical information, and keeping such information from
them is paternalistic.
On the other hand, when patients are informed of biological
measurements of dubious information value, overtreatment can
cause harm.
The prostate-specific antigen (PSA) test and hormone
treatment for postmenopausal women are two examples of
tests that have led to overtreatment, and there is currently a
heated debate on whether screening mammograms do more
harm than good.
For gene variants, specifically variants of unknown significance,
in the BRCA gene could lead affected women to seek a
PAGE 8 OF 15
mastectomy and/or ovariectomy. The cardiac equivalent
might be a defibrillator, though Van Driest pointed out that in
practice, “in the cardiac rhythm world, the nice thing is that we
have an easier test to do” before making decisions on invasive
procedures – the electrocardiogram, which is noninvasive and
can show whether a risk variant is actually leading to problems.
In the current study, which Van Driest and her colleagues
published in the Jan. 5, 2016, issue of the Journal of the
American Medical Association (JAMA), the team sequenced the
genes for two ion channels, SCN5A and KCNH2, in a group of
more than 2,000 patients.
In its 2013 “Recommendations for Reporting of Incidental
Findings in Clinical Exome and Genome Sequencing,” the
American College of Medical Genetics and Genomics (ACMG)
called variants in SCN5A and KCNH2 known pathogenic or
expected pathogenic in two cardiac syndromes, Long QT
syndrome and Brugada syndrome.
According to the ACMG recommendations, “reporting these
incidental findings to the ordering clinician will offer the
clinician, or an appropriate consulting clinician, the opportunity
to re-evaluate the patient’s personal and family history and
consider appropriate surveillance or intervention for patients
and their family members who are deemed to be at increased
risk for these conditions.”
In the work now published in JAMA, the variants identified in
the sequencing were sent to three different laboratories for
“variant calling,” that is, deciding whether a given variant
within the genes was likely to cause disease. The team also
looked at the database Clinvar to identify variants as potentially
pathogenic.
The first finding of the study was that there was much more
disagreement than agreement on whether variants within the
two genes were problematic. Overall, 223 individuals had at
least one rare variant in one of the two genes. Between them,
the three laboratories and the Clinvar database named 42
variants in 63 individuals as pathogenic or likely pathogenic.
The majority of those variants, however, were called out as
problematic by only one of the four sources.
The team looked for evidence of clinical disease, in the form
of arrhythmias or long QT syndrome, in the electronic medical
records of those individuals where at least one of the labs had
identified a pathogenic gene variant.
That study showed no difference between the group with
variants and the rest of the cohort, which served as a control, in
either the prevalence of arrhythmias or long QT syndrome.
Several uncertainties remain about the relationship between
rare variants in the two channels and disease. The bleakest
possibility is that such variants are harmful after all, and the
reason the study failed to detect that harm was survivor bias
– that is, the patients that had rare variants that had led to
disease did not live long enough to enter the study.
Van Driest and her colleagues said they think it is more likely,
See Risk, page 11
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
Nanobiotix
Continued from page 1
trial in the U.S. and a bold new program stepping into the fastdeveloping field of immuno-oncology.
Late last month, the FDA approved Nanobiotix’s lead product,
NBTXR3, as an investigational new drug (IND) for a clinical
study in prostate cancer at three reference radiation oncology
centers.
Prostate cancer is a new indication for NBTXR3, and the FDA
approval of the nanomedicine as a drug compound is also a
first for the company, as NBTXR3 is approved as a medical
device in clinical programs concurrently being run in Europe
and Asia for soft tissue sarcoma, head and neck cancer, rectal
cancer and liver cancer.
At the same time, Nanobiotix also said it would enter the
burgeoning field of immuno-oncology, exploring the use
of NBTXR3 in combination therapies, a departure from
the approach in current trials testing the efficacy of the
nanoparticles for enhancing the effects of radiotherapy.
Based on inorganic hafnium oxide crystals measuring on
average 50 nanometers, NBTXR3 is injected locally at a tumor
site and binds to cancer cell membranes to amplify the energy
used in radiotherapy by as much as ninefold.
SPANNING THE GLOBE
CEO Laurent Levy said the U.S. approval completes a global
reach for clinical development as the company now has
programs running across Europe, as well as in Asia through
a partnership with Pharmaengine Inc., a biopharmaceutical
company based in Taipei, Taiwan.
While the company reported a cash balance at the end of June
2015 of €25 million (US$27 million), Levy told BioWorld Today
that with the launch of the immuno-oncology program, “we
have reached a point where we are going to stop expanding
clinical development and focus on bringing NBTXR3 to market
by the end of this year.”
Publicly traded on the Euronext Paris exchange, Nanobiotix
(EPA:NANO) is capitalized at €221 million. Shares closed Friday
at €15.92 (US$17.39).
“The stock price is good and it is stable at this moment with
many investors waiting to see our results in 2016,” said Levy,
who added that there will be a steady news flow with four
different clinical trials delivering data this year.
“These results could create an interesting potential for an
inflection in the value of the company,” he said.
Interim findings for the pivotal phase of the soft tissue sarcoma
trial in are expected by the middle of the year.
“This should bring a validation of the proof of concept we
demonstrated in 2014, and these data will allow us to proceed
to the CE mark and the commercialization of the product for
this indication,” said Levy. “This clinical trial is a fast track to
market for our product,” he said.
PAGE 9 OF 15
He views the trial for NBTXR3 in head and neck cancer as an
extension of the product into a new indication, “a validation of
the transferability of the product from one cancer to another.”
Levy said he expects the company will also be able to
communicate on the first findings from that phase I trial ahead
of the soft tissue sarcoma results.
A clinical trial applying NBTXR3 in radiotherapy for several
forms of liver cancer is due in the second half of the year, he
said. That phase I/II trial should help establish the value of the
therapy using NBTXR3 by demonstrating the treatment can kill
tumors and extend the lives of patients where the standard of
care cannot be used or does not exist.
MARKET ACCESS FOCUS SHOULD HASTEN RELEASE
Over the past year, Levy said the focus at Nanobiotix has been
on market access, “preparing all the actions necessary so that
on the day we get the CE mark we can press a button to start
selling and deploying NBTXR3.”
Asked if the FDA approval of NBTXR3 as a drug would slow
the time to market, compared to the designation as a medical
device in Europe, Levy said, “at the end of the day what we
need to put our product on the market is to demonstrate the
benefit to the patient against any risk, and the amount of
data we will need to demonstrate this does not depend on the
regulatory status of the product.”
“The technology is new,” Levy added. “We are not working with
biological or chemical interactions but physics, bringing a new
mode of action to eradicate cancerous cells.
“If you apply cytotoxins to a cancer cell, the cell is equipped to
resist these interactions and at some point cancer cells may
escape, but no cancer cell can build resistance to the physical
effect of heating a cell to 80 degrees Celsius, which will kill it
no matter what biological strategy it develops,” he said.
At the same time, a biological reaction of the tumor to the
physical effect is behind the decision to enter the new frontier
of immuno-oncology.
“As long as there is a tumor that can be recognized by the
immune system, then boosting the immune system to
recognize and then kill the tumor makes sense, yet not every
tumor naturally expresses sufficient proteins or markers that
will identify the tumor for the immune system,” he explained.
“We know that radiotherapy boosts some expression at the
tumor that can be recognized by the immune system. This has
been demonstrated. Our proposal is to show that our product
can boost this expression even greater than radiotherapy alone,
and thereby create a better response from the immune system,”
he said.
“The bet here, which has been confirmed in two clinical
settings, is that when you irradiate part of metastatic
melanoma, and it is combined with an immuno-oncology
therapy, the immune system recognizes the tumors and can
start the attack on the other metastasis present in other parts
of the body.” //
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
JPM
Continued from page 3
into biopharma in 2014 and 2015 – more than $105 billion,
combined, in private rounds, IPOs and follow-ons, according
to BioWorld Snapshots. Some companies plowed those
investments into “really important drugs that are going to have
a significant therapeutic benefit for diseases that have no great
treatment,” Nodelman said. Others, however, allocated funds
inefficiently, and that shakeout “is happening as we speak,” he
added.
But recent wobbles in the capital markets have a silver lining,
according to Nodelman.
“The lower the expectations are, the more upside we could
see,” he observed. Although conversations, and perhaps market
performance, may be dominated this year by political rhetoric,
“the fundamentals are stronger than they’ve ever been,”
Nodelman said. “We have more approvals than ever, M&A is
stronger than ever and innovation is clearly the strongest it’s
ever been.”
He cited CRISPR/Cas9 gene-editing technology as an example
of the compelling platforms propelling biopharma’s trajectory,
calling the system’s use by three independent groups of
researchers to improve the symptoms of Duchenne muscular
dystrophy in mice “mind-bending.” (See BioWorld Today, Jan. 4,
2016.)
Despite the hype around CRISPR, “when you compare it to
other revolutionary technologies, like RNAi or monoclonal
antibodies, the development of CRISPR is moving at an
exponentially faster pace,” he said.
‘WE’RE IN THE MIDDLE OF A LONG INNOVATION CYCLE’
Jim Healy, general partner at Sofinnova Venture Partners,
was more cautious about CRISPR, citing the complicated
intellectual property landscape and lack of clear regulatory
guidance from the FDA.
“We’re intentionally watching from the sidelines at this point in
time,” Healy said.
Nevertheless, he acknowledged the accelerating pace of
innovation in gene therapy, from lentivirus to adeno-associated
virus approaches and now CRISPR – which, he added, lit up the
field with its prospect to offer durable cures even in replicating
cells.
Healy will look to hear more during meetings at JPM.
“Our general approach to J.P. Morgan is to spend a lot of time
interacting with big pharma, looking for what types of new
products they’re looking to acquire or partner with,” he told
BioWorld Today. Healy also attends company presentations
and said he’ll be in the audience for New York-based BristolMyers Squibb Co., which he called “the innovation leader in the
immuno-oncology field.” Sofinnova is tracking that trend “in an
important therapeutic area.”
Sofinnova also uses JPM as an opportunity to touch base
with public companies in its portfolio. Healy cited biosimilars
PAGE 10 OF 15
specialist Coherus Biosciences Inc., of Redwood City, Calif.,
and gene therapy developer Spark Therapeutics Inc., of
Philadelphia, as two that could create some buzz at the
meeting.
With three programs in the clinic and numerous upcoming
milestones, Coherus “should be a beneficiary of discussions
around trying to put cost-containment controls around new
drugs,” he said. That concern is heightened by the trajectory
of more-expensive biologics approvals in the U.S., which have
risen fourfold over the past two years, compared to the 20042008 time period, and now account for more than one-fourth
of FDA drug approvals, according to a BioWorld Today analysis.
(See BioWorld Today, Jan. 8, 2016.)
Spark, meanwhile, reported positive phase III data in October
for its lead program, SPK-RPE65, in blindness-causing, RPE65mediated inherited retinal dystrophies and could be the first
gene therapy approved in the U.S. (See BioWorld Today, Oct. 6,
2015.)
Healy also is optimistic about prospects for 2016, coming on
top of two “terrific” years. The positives, he said, begin with the
regulatory environment, where the FDA picked up the pace of
drug approvals and is seeking to be more transparent about its
decision-making process. M&A activity was steady, and IPOs
remained strong, albeit with a slight falloff in 2015 as markets
turned “choppy” in the second half of the year. If that pullback
continues in 2016, “our expectation is that pharma is going to
use that to their advantage to step in and fill up their pipelines,”
Healy said.
But biotech could have strong staying power. Through
December, in generalist funds, fund flow was negative in
every sector except biotech, he pointed out. Even in the fourth
quarter, when the biotech sector dropped 20 percent, “we were
still seeing positive fund flows,” Healy said. “We hope that’s an
indicator for this year.”
Despite election year rhetoric, macroeconomic jitters and
concerns about drug prices, “we’re in the middle of a long
innovation cycle, and we believe that will continue,” he added.
‘A THREAD THAT’S CONSISTENT AMONG THE PRESENTATIONS’
Scientific innovation also is the draw for Jim Robinson, U.S.
president for Astellas Pharma Inc., of Tokyo, who carves out
time at JPM to attend big and specialty pharma presentations.
“I try to see if there’s a thread that’s consistent among the
presentations,” he said. “Clearly, the bulk of the presentations
are about the innovation that each company is driving –
their future focus in terms of investment and their greatest
opportunities for success in the short and long term.”
Robinson keeps an ear to the ground for trends in therapeutic
areas, regulatory oversight and industry activity. But, from a
personal viewpoint, “I really enjoy the science aspect of the
biopharmaceutical industry,” he said.
At this year’s JPM, Robinson expects to attend a broad crosssection of pharma presentations. He’ll also take in talks by
See JPM, page 11
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
JPM
Continued from page 10
pharmacy benefit managers and retail pharmacy companies to
get their take on industry challenges.
“I don’t have a particular pattern,” he said. “I just try to have
an opportunity to meet with as many folks as possible to
communicate the message about Astellas and to educate
myself on trends in the industry.”
Robinson also was upbeat going in to JPM.
“I’m fairly bullish,” he told BioWorld Today. “If you look at the
investment that’s been made over the last decade in specialty
therapeutics, I’m pretty excited about what is actually coming
from the pipelines of companies around the world. I’m an
optimist by nature, and I’m very optimistic, in light of what I’ve
seen during the last couple of years, about emerging oncology
and specialty products. As an industry, we have an opportunity
to continue to focus on invention, as well as innovation.”
Biopharma investors may have a clearer read of the sector’s
pulse at the end of JPM than the beginning, but the early sense
is that 2016 will play out pretty much as business as usual.
“The biopharma sector is in good shape as it enters the New
Year,” BioWorld’s Winter said. “While we will likely not see a
massive run-up in valuation during 2016, it should be able to
exhibit slow and steady growth.” //
Risk
Continued from page 8
however, that the variants really are not predictive of disease,
and that whether to report them when they show up as
incidental findings should be carefully considered.
Van Driest said her take on the return of incidental findings is
that “we will all be much more enthusiastic about returning
results as we gain more confidence in the results.”
For now, though, “genes are like people,” she said. “They are
more complex than you think.” //
OTHER NEWS TO NOTE
Cytos Biotechnology Ltd., of Schlieren, Switzerland, said it is to
be renamed as Kuros Biosciences Ltd.
Dual Therapeutics LLC, of Cleveland, said it signed a strategic
collaboration with Bristol-Myers Squibb Co., of New York,
to advance small-molecule compounds for the treatment of
cancer and other diseases. Dual’s small-molecule modulators
simultaneously block multiple cancer-promoting pathways,
particularly the growth and survival pathways, allowing a more
comprehensive assault on cancer cells while sparing normal
cells. BMS will gain exclusive, worldwide rights to develop and
commercialize the small-molecule therapeutics discovered
by Dual in exchange for an undisclosed up-front fee and
development costs. Dual will be eligible to receive development
and regulatory milestones that could total more than $255
PAGE 11 OF 15
million for a successful compound approved in multiple
indications in addition to royalties on future sales of products
discovered in the collaboration.
Enumeral Biomedical Holdings Inc., of Cambridge, Mass.,
announced the presentation of research findings for the
company’s class of anti-PD-1 antibodies at the Function of the
Tumor Microenvironment in Cancer Progression conference
in San Diego. The anti-PD-1 antibodies can reverse T-cell
suppression in ex vivo assays by eliciting high levels of T-cell
activation as measured by increased secretion of effector
cytokines such as interferon-gamma and increases in
expression of the IL-2 receptor alpha chain, CD25. Further, in
an in vivo preclinical model harboring a reconstituted human
immune system, the company’s fully humanized anti-PD-1
antibodies exhibited significant antitumor efficacy against a
human lung adenocarcinoma xenograft.
Grünenthal Group, of Aachen, Germany, said it established
a joint global drug development program with Akashi
Therapeutics Inc., of Cambridge, Mass., for HT-100 (delayedrelease halofuginone), an orally available small-molecule drug
candidate designed to reduce fibrosis and inflammation and
to promote healthy muscle fiber regeneration in Duchenne
muscular dystrophy patients. Grünenthal will be responsible
for commercialization in Europe and Latin America, while
Akashi will retain rights for the U.S. and all other markets.
HT-100 is currently in phase Ib/IIa trials. Under the terms of
the agreement, Grünenthal will make up-front and milestone
payments to Akashi. In addition, it will assume all post-phase
II global development costs through commercialization of an
approved product. Akashi will receive royalties on net sales. In
total, Grünenthal plans to commit more than $100 million to
the partnership and will receive royalties on U.S. net sales in
exchange for funding development of Akashi’s U.S. commercial
infrastructure.
APPOINTMENTS AND ADVANCEMENTS
Acorda Therapeutics Inc., of Ardsley, N.Y., appointed Burkhard
Blank interim chief medical officer.
Achillion Pharmaceuticals Inc., of New Haven, Conn., added
Frank Verwiel to its board.
Aclaris Therapeutics Inc., of Malvern, Pa., named Brett Fair
senior vice president of commercial operations.
Aimmune Therapeutics Inc., of Brisbane, Calif., appointed
Jeffrey H. Knapp chief operating officer, effective Feb. 1.
Alimera Sciences Inc., of Alpharetta, Ga., named Richard S.
Eiswirth Jr. president and chief financial officer. C. Daniel Myers
will remain CEO.
Ariad Pharmaceuticals Inc., of Cambridge, Mass., appointed
Paris Panayiotopoulos president and CEO.
Assembly Biosciences Inc., of New York, added Alan J. Lewis to
its board.
Catabasis Pharmaceuticals Inc., of Cambridge, Mass., named
Deirdre Cunnane senior vice president, general counsel.
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
OTHER NEWS TO NOTE
Healios K.K., of Tokyo, and Athersys Inc., of Cleveland, said
they entered a partnership and license agreement that will
focus on the development and commercialization of cell
therapy treatments, including Multistem, in Japan. Multistem,
an off-the-shelf stem cell therapy being developed by Athersys,
initially is being developed for ischemic stroke. Healios will
gain exclusive rights for the development of Multistem for
treating ischemic stroke in Japan, where it will develop
and commercialize the product. Athersys will provide the
manufactured product and support to Healios, while retaining
all rights outside of Japan. In addition, Healios will obtain an
exclusive option for development of two additional Multistem
clinical indications in Japan, including the treatment of acute
respiratory distress syndrome (ARDS), which is currently in
clinical development by Athersys in the U.S. and the U.K, and
another indication in the orthopedic area. Healios will also
obtain an exclusive license to incorporate Athersys technology
in the development and commercialization of its organ bud
technology, initially for transplantation to treat liver disease
or dysfunction, which may be expanded upon exercise of
the option. Athersys will receive an initial license fee of $15
million, as well as have the opportunity to earn milestone
and royalty payments upon the successful accomplishment
of specific development and commercialization objectives,
including the achievement of certain sales milestones. The
milestones for stroke could total $30 million, in addition
to sales milestones that could reach $185 million based
on successful commercialization and the achievement of
substantial sales of an approved product for treating stroke in
Japan. Athersys will also receive tiered, double-digit royalties
increasing into the high teens on product sales and will be
responsible for providing manufactured product to Healios,
subject to receiving reimbursement under a manufacturing
supply arrangement. In addition, if Healios elects to expand the
partnership following the successful completion of Athersys’
ongoing clinical trial in ARDS, it will receive a license expansion
fee of $10 million for the exclusive rights to two additional
indications in Japan, with the corresponding potential for
further milestones based on successful achievement of specific
development and commercialization objectives. Shares of
Athersys (NASDAQ:ATHX) gained 22 cents, or 21.4 percent, to
close Friday at $1.25.
Immune Design Corp., of Seattle, said the FDA granted
orphan status for LV305 and G305 for the treatment of soft
tissue sarcoma. LV305 and G305 are the complementary
agents that comprise CMB305, Immune Design’s prime boost
cancer immunotherapy candidate currently in a phase Ib
trial in patients with locally advanced, relapsed or metastatic
solid cancers whose tumors express NY-ESO-1 and in a phase
II trial in combination with anti-PD-L1 drug atezolizumab
(MPDL3280A, Genentech Inc./Roche AG) in patients with soft
tissue sarcoma.
PAGE 12 OF 15
Moderna Therapeutics Inc., of Cambridge, Mass., and
Pharmaceutical Product Development LLC, of Wilmington,
N.C., said they entered a collaboration to support Moderna’s
evolution as a clinical-stage company. Under the terms
of the agreement, PPD will play a significant role in the
company’s clinical development efforts, including support
for investigational new drug planning, as well as for clinical
trial design and execution. Moderna’s pipeline is composed
of a series of drug modalities that include infectious disease
vaccines, personalized cancer vaccines, intracellular/
transmembrane liver proteins, intratumoral cancer therapy
and secreted antibodies and proteins. Moderna is leveraging
these modalities to advance drugs across a broad spectrum
of therapeutic areas via its four ventures, including Valera,
focused on infectious diseases; Elpidera, focused on rare
diseases; Onkaido, focused on oncology; and Caperna, focused
on personalized cancer vaccines.
Pfizer Inc., of New York, and Adaptive Biotechnologies Corp.,
of Seattle, entered a translational research collaboration to
leverage next-generation sequencing of the adaptive immune
system to advance Pfizer’s growing immuno-oncology
franchise. Under the terms of the agreement, the companies
will seek to combine drug development and platform
technology biomarker expertise to identify patients who may
preferentially benefit from immunotherapy.
PTC Therapeutics Inc., of South Plainfield, N.J., said it
completed its rolling submission of a new drug application to
the FDA for Translarna (ataluren), an oral protein restoration
therapy for the treatment of nonsense mutation Duchenne
muscular dystrophy. PTC has also fulfilled the principal
requirement of the EMA in connection with its approval of
Translarna in August 2014 by submitting the results of its
phase III ACT DMD trial to the agency. (See BioWorld Today,
May 27, 2014.)
Regeneron Pharmaceuticals Inc., of Tarrytown, N.Y., and
Sanofi SA, of Paris, said the FDA has accepted for review the
biologics license application for sarilumab with a target action
date of Oct. 30, 2016. Sarilumab is a human monoclonal
antibody directed against the interleukin-6 (IL-6) receptor that
is intended for the treatment of patients with active, moderate
to severe rheumatoid arthritis (RA). IL-6 is the most abundant
cytokine in the serum and synovial fluid of patients with RA and
levels correlate with both disease activity and joint destruction,
the firms noted.
Theravance Inc., of South San Francisco, changed its name to
Innoviva Inc.
Tiziana Life Science plc, of London, said its research
agreement with Cardiff University, focused on the development
of Bcl-3 inhibitors for cancer, led to the identification of a lead
candidate, CB1, with antimetastatic activity and in vivo efficacy
and safety. The company said it plans to file an investigational
new drug application and move CB1 into the clinic before the
end of this year.
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MONDAY, JANUARY 11, 2016
BIOWORLD™ TODAY
OTHER NEWS TO NOTE
Wuxi Apptec Co. Ltd., of Shanghai, started construction of
a state-of-the-art integrated biologics solution center at the
company’s headquarters. The company plans to invest a total
of $120 million in that dedicated, 250,000-square-foot facility,
which will be operational in 2017 and accommodate 800
scientists. The facility will provide integrated solutions from
ideas to the clinic for biologics discovery, development and
clinical manufacturing on a consolidated campus, Wuxi said.
IN THE CLINIC
Abbvie Inc., of North Chicago, Ill., said it started a large phase
III program to study the use of ABT-494, a once-daily, oral
selective JAK1 inhibitor for the treatment of rheumatoid arthritis
(RA). The program will include adult patients with inadequate
responses to conventional or biologic disease-modifying
antirheumatic drugs (DMARDs), as well as methotrexate-naive
patients. The first two trials out of the five phase III studies
planned have opened for enrollment in the U.S. One study will
evaluate ABT-494 in combination with methotrexate in adult
patients with moderate to severely active RA who have had an
inadequate response to prior treatment with methotrexate, and
will include Humira (adalimumab) as an active comparator. The
second study will include patients who have had an inadequate
response or intolerance to conventional synthetic DMARDs. The
studies will include assessments of safety and tolerability, as
well as key measures of efficacy, including American College
of Rheumatology criteria for responses and levels of disease
activity. The other three phase III trials will begin enrollment
early this year and will include patients with an inadequate
response to biologics and patients who are methotrexate-naive.
Acelrx Pharmaceuticals Inc., of Redwood City, Calif., said
it received comments from the FDA’s Division of Anesthesia,
Analgesia and Addiction Products on the company’s proposed
protocol for a phase III study (IAP312) designed to assess the
overall performance of Zalviso (sufentanil sublingual tablet
system). In response to the comments, the protocol has been
amended to track additional Zalviso use by patients in a clinical
setting. Acelrx plans to initiate the study in the first quarter. The
IAP312 study will include about 310 postoperative patients and
collect information requested by the division to supplement the
three phase III trials already completed. Acelrx is developing
Zalviso for the management of moderate to severe acute pain
in adult patients in the hospital setting.
Beigene Ltd., of Beijing, said the FDA cleared the firm’s
investigational new drug application for phase Ia/IIb studies of
BGB-A317, a humanized monoclonal antibody against immune
checkpoint receptor PD-1. That will allow the firm to include
U.S. sites in the ongoing open-label, dose-escalation study in
patients with relapsed or refractory solid tumors. As of Nov. 30,
a total of 51 patients in Australia had been dosed in the study,
which is evaluating pharmacokinetics and antitumor activities.
Beigene plans to develop BGB-A317 as a monotherapy and
PAGE 13 OF 15
as a combination agent with small-molecule therapeutics for
various solid organ and blood-borne cancers.
Biocryst Pharmaceuticals Inc., of Durham, N.C., said that
when compared to Western subjects administered the same
dose level of BCX7353, its oral plasma kallikrein inhibitor,
plasma drug levels in healthy Japanese trial volunteers were
moderately higher. Kallikrein inhibition on day seven of daily
dosing with 250 mg in Japanese subjects was similar to that
seen at the 350-mg daily and 500-mg daily dose levels in
Western subjects. The patients were added to the phase I
trial to support development of BCX7353 in Japan under the
Sakigake accelerated R&D designation. BCX7353 has been
generally safe and well tolerated in a total of 96 Western and
Japanese healthy volunteers treated, Biocryst said.
Biogaia AB, of Stockholm, Sweden, reported that its subsidiary,
Infant Bacterial Therapeutics (IBT), said an investigational new
drug application seeking permission to test a therapy for the
prevention of necrotizing enterocolitis has been accepted by
the FDA. IBT also received approval from the Medical Product
Agency to conduct a trial in Sweden, it said.
Dermira Inc., of Menlo Park, Calif., said patient enrollment
was completed for the DRM01 phase IIb trial in patients
with facial acne vulgaris, with top-line data expected in the
second quarter. DRM01 is a topical, small-molecule sebum
inhibitor. The company also reported that top-line data from
its two phase III trials of DRM04, a topical, small-molecule
anticholinergic product, in axillary hyperhidrosis are expected
in mid-2016, compared to the previous guidance of the second
half of 2016.
Foamix Pharmaceuticals Ltd., of Rehovot, Israel, reported
top-line results from a phase I maximum use pharmacokinetics
study of FMX-101, a topical foam containing 4 percent
minocycline, in development to treat moderate to severe acne.
The study, intended to characterize the systemic absorption
of minocycline after repeated maximum dose applications,
indicated that the relative bioavailability (systemic exposure) of
FMX-101 was more than 100 times lower than that for Solodyn
(minocycline HCl). FMX-101 was well tolerated, and no serious
adverse events were reported.
Genentech Inc., of South San Francisco, a unit of Roche
AG, said The New England Journal of Medicine published a
letter to the editor reporting results of a 10-year study using
Rituxan (rituximab) in combination with intravenous immune
globulin for the treatment of pemphigus vulgaris, a potentially
fatal autoimmune blistering disease that affects the skin
and mucous membranes. Findings showed that, following
discontinuation of Rituxan, all patients continued to remain in
remission without recurrence of the disease.
Kolltan Pharmaceuticals Inc., of New Haven, Conn., said
KTN0158, a humanized anti-KIT monoclonal antibody drug
candidate, was administered to the first cancer patient in
phase I trial. The open-label, dose-escalating study involves
patients with gastrointestinal stromal tumors and other tumors
expressing KIT.
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MONDAY, JANUARY 11, 2016
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IN THE CLINIC
Molecular Partners AG, of Zurich, Switzerland, said it plans
to conduct a phase II trial of MP0250, a multi-DARPin drug
candidate designed to block vascular endothelial growth
factor and hepatocyte growth factor, in multiple myeloma.
The trial will test MP0250 in combination with Velcade
(bortezomib, Takeda Oncology Inc.) and dexamethasone in
patients who have developed resistance to Velcade and have
received at least two prior regimens, including Velcade and an
immunomodulatory drug. Molecular Partners aims to enroll the
first patient in the second half of this year.
Ocera Therapeutics Inc., of Palo Alto, Calif., reported further
details from a phase I study testing oral formulations of OCR002, ornithine phenylacetate, in healthy subjects. Top-line
data, reported in November, showed a robust, extendedrelease profile for all three pilot OCR-002 formulations. Those
pilot formulations were evaluated on their ability to provide
extended release of phenylacetate (PAA), a potent ammonia
scavenger, and on the formation of phenylacetylglutamine
(PAGN), the end-product responsible for clearing ammonia.
Levels of plasma PAA and PAGN exceeded those achieved with
the ammonia-lowering agent glycerol phenylbutyrate, branded
Ravicti (Horizon Pharma plc), a pre-prodrug of phenylacetate.
The company plans to conduct additional phase I testing after
further formulation optimization.
Oncomed Pharmaceuticals Inc., of Redwood City, Calif.,
said a manuscript published in Cancer Research describes the
company’s efforts to develop therapeutics against R-spondin
(RSPO) targets and elucidates the link between expression
of the RSPO cancer stem cell pathway and tumor growth.
Oncomed is conducting a phase Ia/Ib trial of its anti-RSPO3
antibody, OMP-131R10.
Prescient Therapeutics Ltd., of Melbourne, Australia, said it
secured FDA approval for a phase Ib/II trial in acute myeloid
leukemia (AML). The study will combine the firm’s lead
candidate, Akt inhibitor PTX-200, with chemotherapeutic
agent cytarabine in refractory or relapsed AML. The first part
will enroll 15 to 18 patients in an open-label, dose-escalation
design, while the second part will be an open-label study
testing the recommended dose of PTX-200 for two 21-day
cycles.
Protagonist Therapeutics Inc., of Milpitas, Calif., said it started
treating subjects with lead candidate PTG-100, an orally
stable alpha-4-beta-7 integrin-specific peptide antagonist for
inflammatory bowel disease, in a phase I study. The randomized,
double-blind, placebo-controlled, dose-escalation trial will
involve 70 normal healthy volunteers. The first part of the study
consists of single ascending doses of PTG-100 in 40 volunteers,
while the second part will involve administration of PTG-100 over
14 consecutive days in 30 additional subjects. Primary endpoints
are safety and tolerability, and secondary endpoints are the
identification of the maximally tolerated dose and evaluation of
pharmacokinetic and pharmacodynamic parameters.
PAGE 14 OF 15
Roche AG, of Basel, Switzerland, reported updated results from
its pivotal phase II IMvigor 210 study testing immunotherapy
candidate atezolizumab (MPDL3280A) in people with locally
advanced or metastatic urothelial carcinoma (mUC), with
median overall survival of 11.4 months in people with higher
levels of PD-L1 expression, and 7.9 months in the overall study
population. The study also showed that 84 percent (38/45)
of people who responded to atezolizumab continued to
respond regardless of their PD-L1 status, when the results were
assessed with longer median follow-up of 11.7 months. Median
duration of response has not yet been reached. Atezolizumab
was well tolerated and adverse events were consistent with
those observed in previous updates. Data were presented at the
2016 Genitourinary Cancers Symposium in San Francisco.
Valor Biotherapeutics LLC, of Bryan, Texas, said the first
patient was dosed in a phase I study of lead candidate IGN002
in non-Hodgkin lymphoma. The study is designed to assess the
safety and tolerability of IGN002 administered weekly for up to
26 weeks, evaluate the pharmacokinetic profile of escalating
doses of IGN002 and assess the antitumor activity, objective
response rate and duration of responses. Valor, a joint venture
between Immungene Inc., of Camarillo, Calif., and Caliber
Biotherapeutics LLC, also of Bryan, has partnered with the
Leukemia & Lymphoma Society on preclinical development,
manufacturing and an initial proof-of-concept clinical study of
IGN002.
Vectura Group plc, of Chippenham, U.K., said it completed a
clinical trial for VR315, a generic inhaled combination therapy
comprising fluticasone and salmeterol, in the U.S. for asthma
and chronic obstructive pulmonary disease. VR315 is delivered
using Vectura’s dry powder inhaler and formulation technology.
Xbiotech Inc., of Austin, Texas, said it completed data analysis
for its phase III European study of Xilonix in advanced,
symptomatic colorectal cancer. In addition to previously
announced positive results regarding the primary endpoint of
the pivotal study, complete data analysis further demonstrated
that key secondary measures of antibody activity were also
improved. Inhibition of IL-1 alpha on the surface of platelets
may represent an antitumor, disease-modifying activity, due
to the mechanism of action of Xilonix, and median platelet
counts among placebo patients in the study were found to be
increased fivefold compared to patients who received Xilonix,
whose platelet counts remained near baseline levels during the
treatment cycle (p=0.003). (See BioWorld Today, Dec. 8, 2015.)
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MONDAY, JANUARY 11, 2016
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APPOINTMENTS AND ADVANCEMENTS
Decibel Therapeutics, of Boston, added Jeff Jonas, to its board.
Derma Sciences Inc., of Princeton, N.J., named Stephen
T. Wills interim executive chairman and principal executive
officer.
Dicerna Pharmaceuticals Inc., of Cambridge, Mass., named
John “Jack” Green interim chief financial officer.
Dimerix Ltd., of Melbourne, Australia, added Liz Jazwinska to
its board.
Immunocore plc, of Oxford, U.K., appointed James Sandy
chief development officer and Julian Hirst director of corporate
finance; they will be based in Oxford. The company also opened
a U.S. office in Conshohocken, Pa.
Kite Pharma Inc., of Santa Monica, Calif., appointed Shawn
Tomasello chief commercial officer.
Merus BV, of Utrecht, the Netherlands, appointed Hui Liu chief
business officer.
Novavax Inc., of Gaithersburg, Md., named Mark Twyman vice
president, marketing.
Redhill Biopharma Ltd., of Tel Aviv, Israel, appointed Micha
PAGE 15 OF 15
Ben Chorin chief financial officer, effective March 1.
Rodin Therapeutics Inc., of Cambridge, Mass., added
Samantha Singer to its board.
Theravectys SAS, of Paris, named Alain Clergeot CEO.
Turing Pharmaceuticals AG, of Zug, Switzerland, appointed
Ron Tilles interim CEO.
Uniqure NV, of Amsterdam, the Netherlands, named Dan
Soland CEO.
Vical Inc., of San Diego, added Thomas E. Shenk to its board.
IN THE CLINIC
Xcovery Inc., of Needham, Mass., updated data from its ongoing
phase I/II trial of tyrosine kinase inhibitor (TKI) X-396 in patients
with anaplastic lymphoma kinase (ALK)-positive non-small-cell
lung cancer. Key data showed an objective response rate of 88
percent in ALK-TKI treatment-naïve patients and 84 percent
in Xalkori (crizotinib, Pfizer Inc.)-resistant patients. Data also
showed responses in patients with brain metastases. The most
common adverse event (AE) related to treatment was rash in 49
percent of patients, and no other related AEs were observed in
greater than 30 percent of patients.
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BENCH PRESS
BioWorld looks at translational medicine
By Anette Breindl, Senior Science Editor
Pandemic take 3
Pandemic flu gives insights
into vaccine response
Scientists at the British King’s College London have reported
results from long-term high-throughput immune monitoring
of a cohort of roughly 180 individuals after they were
vaccinated with adjuvanted Pandemrix, the vaccine against
the 2009 pandemic “swine flu” strain. Because seasonal
influenza strains do not vary that much from year to year,
the immune response to seasonal influenza vaccination
is predetermined to a significant extent by an individuals’
vaccination history. By looking at immune responses to
the vaccine for a pandemic flu strain – which is pandemic
precisely because there is not the same kind of immune crossreactivity as with annual strains – the authors were able to
look at how individual immune characteristics determined
the vaccination response. They found that there was an
early complex immune response that included myeloid
cell responses, currently thought to be a late component
of immunity. Furthermore, they were able to show that an
adverse vaccine response did not exacerbate underlying
immune abnormalities, and linked the probability of such
a response to a specific immune signature. Beyond their
findings with respect to the pandemic vaccine, the authors
said that “the trial’s outcomes confirmed the practicality of
high-throughput immune monitoring, which will be important
as immunotherapies are more widely adopted.” The findings
appeared in the Jan. 4, 2016, issue of Nature Immunology.
Pandemic redux
Another team, this one from New York University and the
University of Hong Kong, has looked at the co-transmission
of pandemic H1N1 and seasonal H3N2 flu strains in 2009 to
understand within-host genetic diversity and how it affected
the transmission of the flu. In their work, the authors took
an in-depth look at viral sequences in donor-recipient pairs,
where one caught influenza from the other, within the same
household. They used those data to estimate that during
a typical infection event, about 100 to 200 viral particles
are transmitted, which allowed for the co-transmission of
multiple lineages, The authors said their work “allows a better
understanding of influenza virus transmission in humans and
provides more accurate information for modeling epidemics
and for disease control strategies.” It appeared in the Jan. 4,
2016, issue of Nature Immunology.
MONDAY, JANUARY 11, 2016
Finally, a team at the British Imperial College London has
gained new insights into why bird or avian influenza viruses
cannot easily breach the species barrier. Avian influenza
viruses cause the most serious disease in humans when
they do become easily transmissible, but most fail to do so.
Several reasons for that failure to launch have been identified,
including differences in the anatomical distribution of the
receptors influenza virus uses to enter cells. Another reason
is that avian polymerase, which copies the viral RNA, does
not work particularly well in mammals. Previous work by
the same team had shown that the reason is that avian cells
have a specific factor that turbo-charges the polymerase,
rather than the presence of a specific inhibitory protein in
mammalian cells. In the current follow-up, the team showed
that the difference in polymerase activity is due to species
differences in the protein ANP32A, which interacts with the
viral polymerase. Avian ANP32A has 33 amino acids more
than its mammalian counterpart, and the shorter mammalian
versions led to reduced polymerase activity and, therefore,
reduced viral replication. The exact nature of the interaction
between ANP32A and polymerase remains to be determined,
but the authors concluded that “disrupting the interaction of
the virus with ANP32A may be a novel means for virus control”
of influenza viruses that infect humans, because efficient
replication is paramount to viral success. The findings appeared
in the Jan. 6, 2016, advance online edition of Nature.
CRISPR’s helpmate goes high-fidelity
Scientists from Massachusetts General Hospital have
engineered a Cas9 nuclease that had greatly reduced off-target
effects, which has been one of the major stumbling hurdles
for CRISPR’s use in clinical application. Cas9 is the enzyme
that induces DNA breaks at sites that are determined by the
sequence of CRISPR. CRISPR, however, consists of more than
just the targeting part. The authors originally hypothesized
that the nontargeting part of the enzyme might be responsible
for off-target effects through nonspecific binding, and they
substituted several amino acids that they predicted would be
most likely to be responsible for such effects. The resulting
version of Cas9, which the authors dubbed SpCas9-HF1,
“rendered all or nearly all off-target events undetectable”
for standard targeting sequences, and greatly reduced offtarget effects even for atypical repetitive sequences, where
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off-target activity is harder to prevent. The authors noted that
their original hypothesis for why engineering Cas9 at the sites
they chose should decrease off-target effects was likely too
simple. Nevertheless, they concluded that “with its exceptional
precision, SpCas9-HF1 provides an alternative to wild-type
SpCas9 for research and therapeutic applications. More
broadly, our results suggest a general strategy for optimizing
genome-wide specificities of other CRISPR-RNA-guided
nucleases.” The team published its work in the Jan. 6, 2016,
advance online edition of Nature.
T cells’ expansion strategies, deciphered
Scientists from the University of Pennsylvania have identified
factors that affected whether T cells expanded in vitro. Such
expansion is a necessary prerequisite for emerging T-cell
therapies such as chimeric antigen receptor (CAR) T cells, and
it strongly predicts whether T cells will be active in vivo once
they are re-infused into patients. In the clinic, about a quarter
of children being considered for the University’s CD19 CAR T
clinical trial had T cells that did not expand sufficiently during
testing for them to be able to enter the trial itself. In a new
trial, the team studied 50 children with different leukemias
and lymphomas, both upon enrollment and after each cycle
of chemotherapy the children received. They found that the
ability to expand correlated with higher levels of early lineage
T cells that were still stem-like in some aspects. Chemotherapy
specifically depleted those cells, but culturing cells with the
cytokines interleukin-7 and interleukin-15 could restore their
expansion capabilities. The authors concluded that “early
lineage cells are essential to T cell fitness for expansion,
and enrichment of this population either by timing of T cell
collection or culture method can increase the number of
patients eligible to receive highly active engineered cellular
therapies.” They published their results in the Jan. 6, 2016,
issue of Science Translational Medicine.
Metabolic reprogramming is neuroprotective
Scientists from the Belgian KU Leuven have shown that
inhibiting or deleting the oxygen-sensing protein prolyl
hydroxylase domain 1 (PHD1) could reduce the infarct size in
animal models of stroke. Cell death during and after a stroke
does not occur at one fell swoop. Instead, there is a core zone
where neurons die immediately. But around that zone is the
so-called penumbra, where neurons are damaged but not
necessarily fated to die. In their experiments, the authors
showed that they could limit the damage within that penumbra
through inhibiting PHD1, either through genetic deletion or
through pharmacological inhibitors. Inhibiting PHD1 changed
neuronal metabolism in a way that ultimately allowed the cells
to buffer free radicals, which are a major source of damage
MONDAY, JANUARY 11, 2016
in ischemic stroke. The authors concluded that “these data
identify PHD1 as a regulator of neuronal metabolism and a
potential therapeutic target in ischemic stroke.” They published
their results in the Jan. 7, 2016, online issue of Cell Metabolism.
Treat the liver without making things worse
A team at the University of Texas Southwestern Medical Center
has developed a nanoparticle that improved survival in a
mouse model of late-stage liver cancer. Liver cancer has a high
fatality rate and is particularly difficult to treat because most
drugs are more toxic to a cancerous liver than a healthy one.
MicroRNAs (miRNAs) could potentially be an answer to that
conundrum, but delivering them presents the same toxicity
challenges. The authors tested more than 1,500 dendrimer
nanoparticles to identify those with the best combination
of low toxicity and high delivery efficacy, and tested the five
most promising candidates’ ability to deliver let-7 miRNAs
in an aggressive model of liver cancer. They showed that the
approach significantly extended the animals’ survival. The
authors concluded their particles had promise for the treatment
of liver cancer but also that, more broadly, “the interface
between chemical design, cancer biology, and the clinical
disease setting must be carefully appreciated to develop the
next generation of oligonucleotide-based cancer therapies.”
The findings appeared in the Jan. 4, 2016, online issue of the
Proceedings of the National Academy of Sciences.
Deciphering the stress response
Two independent research teams, one from Duke University
and one from the German Max Planck Institute of Biochemistry,
have gained new insights into the structural features of
heat-shock factors (HSFs). HSFs bind to heat-shock proteins,
chaperone proteins that help other proteins fold correctly. As
such, they play a role in a wide variety of cellular processes.
Their role is further broadened because their interaction
partners include proteins that are critical in the DNA damage
response. There are two HSFs, HSF1 and HSF2, and the authors
looked at the structural details of how both bind to DNA. The
Munich group focused on HSF1 and found that single amino
acid changes can alter its binding characteristics, revealing
the structural basis for its specificity. The Duke team looked at
both HSF1 and HSF2, and found that in addition to assembling
in groups of three identical subunits of one type, the two can
form trimers with each other. Such HSF1-HSF2 interaction,
the authors said, “add an additional layer of complexity to
the stress response but may also provide cells with a rheostat
facilitating more precise transcriptional output.” The papers
appeared back to back in the Jan. 4, 2016, issue of Nature
Structural and Molecular Biology.
LET US KNOW WHAT YOU THINK
We welcome your feedback. Contact Anette
Breindl at [email protected],
or (770) 810-3134.
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