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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
demonstrated efficacy, but then stalled in the pipeline due to side effects or poor bioavailability.
VOLT01 is the first in line. ZA, administered intravenously, slows
lightning – a strategy underpinning the company's name – into the loss of bone mass but is associated with a raft of post-dose
phase III trials for its lead candidate. VOLT01 is a derivative of
side effects, including flushing, fever, joint pains and muscle
zoledronic acid (ZA), a bisphosphonate originally marketed by
aches. The VOLT01 formulation is designed to tamp down those
Novartis AG in a variety of indications, including osteoporosis,
side effects without sacrificing efficacy.
Paget's disease and bone cancers, under the trade names
The market could be sizable. The Novartis drug had peak sales
Reclast in the U.S., Aclasta in Australia and Zometa.
of $2.1 billion in 2011, falling back to less than $1 billion last
Bethlehem, Pa.-based Voltarra is collaborating with the
year, according to Cortellis Competitive Intelligence.
Menzies Research Institute at the University of Tasmania in
Selection of KOA as the drug's initial indication involved a
Hobart, Australia, on the multicenter trial, which is assessing
bit of serendipity. Several years ago, Menzies conducted a
the efficacy and safety of VOLT01 in knee osteoarthritis (KOA).
randomized, placebo-controlled study of ZA vs. placebo in
CEO Richard Becker, whose resume includes the oncology
patients with KOA, an off-label indication, with the trial partly
groups at Bayer Pharmaceuticals, Novartis and Merck & Co.
funded by Novartis, Becker explained. Results, published in
Inc., formed Voltarra in September 2013 by acquiring the assets 2011, showed that a single 5-mg infusion of ZA in patients with
of IMC Biotechnology Inc., which was developing early stage
KOA reduced pain and led to shrinkage of bone marrow lesions
spleen tyrosine kinase inhibitors and anti-tumor necrosis factor
in the study, known as ZAP, which enrolled 59 patients.
drugs, and Renascence Pharmaceuticals, which had a portfolio
However, the Menzies researchers wanted to follow a larger
of late-stage product reformulations. The former CEOs of those group for a longer time period. In June 2013, shortly before
companies, IMC's Renee Stewart and Renascence's Ketan
Voltarra was formed, the group launched a randomized,
Desai, are Voltarra's only other full-time employees, serving as
multicenter phase III study, known as ZAP2, comparing ZA to
chief scientific officer and chief medical officer, respectively.
placebo. The ongoing trial, funded by grants and involving four
Voltarra's early stage assets are on the back burner for now,
sites in Australia, is designed to enroll 264 patients with KOA,
while the company focuses on moving to market derivatives
with efficacy as the primary endpoint.
of compounds that advanced through phase II or beyond,
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
improvements in behavior, attention and sleep were evident in the other three patients. Brain atrophy, measured by nuclear
magnetic resonance imaging, appeared stable in two patients
neurodegenerative disorder arising from impaired breakdown
but increased in two others, including the youngest patient. A
of the cell surface and extracellular matrix component heparan follow-up observation trial is ongoing.
Meanwhile, Lysogene will embark on a slew of activities in
Also called mucopolysaccharidosis III, it is the most prevalent
advance of the pivotal studies, including embarking on a
and severe of four related lysosomal storage disorders that
manufacturing campaign, engaging with drug regulators and
have devastating effects on patients' intellectual and motor
patient groups, and selecting and preparing clinical trial sites.
development and that lead to an early death.
It plans to seek a pre-investigational new drug application
So far, just four Sanfilippo type A patients have received the
meeting with the FDA with a view to including U.S. sites in the
therapy in an open-label phase I/II trial. "The next step for us
development program. Manufacturing will be carried out at the
is to prepare for pivotal studies," Karen Aiach, CEO and co-
UCL Partners Gene Therapy Consortium at University College
founder of Lysogene, told BioWorld Today. The company aims to London. Aiach's co-founder, Olivier Danos, previously was begin the pivotal program by the end of 2015. In rare diseases,
director of that unit and established the AAV facility there.
this kind of trajectory is not uncommon. "We even know of
The funding round was led by Paris-based Sofinnova Partners,
some groups who are aiming to enter directly into phase I/II/III
which was also the company's seed investor. Other participants
studies in rare diseases with innovative therapies," she said.
in the new investment include Bpifrance, through its Innobio
The Paris-based firm also plans to commence development of a fund, and Novo Seeds, the early stage investment arm of Novo second gene therapy treatment in an undisclosed indication. It
aims to file a clinical trial application by the end of 2016.
Sofinnova Partners was attracted to the company on the
SAF-301 comprises an adeno-associated viral (AAV) vector
basis of the therapeutic potential of its lead program, not its
serotype 10 carrying two genes, SGSH and SUMF1, which
technological profile as a gene therapy. "For this disease, we
encode N-sulfoglucosamine sulfohydrolase and sulfatase-
think this is the best approach," Rafaèle Tordjman, managing
modifying factor 1, respectively. The former is part of the
partner at Sofinnova, told BioWorld Today. "It's not more high-
heparan sulfate breakdown pathway and is absent in patients
risk than usual. Obviously gene therapy is challenging," she
with Sanfilippo syndrome type A.
said. But the technical challenge is offset by the more limited
In the SAF-301 trial, the construct was injected directly into
clinical development requirements. The eventual European
the patients' brains via a stereotactic procedure. Each received
approval of Uniqure BV's Glybera (alipogene tiparvovec) to treat
a dose of 7.2 x 1011 viral genomes, delivered over a two-hour
familial lipoprotein lipase deficiency is further encouragement,
period from a 12-needle device. "It's mainly a neurological
notwithstanding the tortuous approval process the dossier
disease, with very mild peripheral symptoms," Aiach said. The
went through. "It's probably not the easiest or best case,
limited scale of the study means that whatever conclusions
because it's not a lethal disease," Tordjman said.
can be drawn must necessarily be tentative. "The primary
There is no approved therapy for any of the Sanfilippo
endpoint was safety," she said. "The second objective of the
syndromes as yet. Dublin-based Shire plc has completed a
study was to explore potential future efficacy endpoints." The
phase I/II trial of an enzyme replacement therapy for type A,
construct appeared safe and well tolerated and, despite being
SHP610, which it plans to move into a phase IIb trial. Uniqure,
on immunosuppressive therapy, the patients did not exhibit an
of Amsterdam, the Netherlands, is collaborating with the
increase in infection.
Pasteur Institute in Paris on a gene therapy approach for
SUMF1 plays a role in the post-translational modification and
Sanfilippo syndrome type B. A phase I/II trial is ongoing. //
catalytic activation of sulfatase enzymes. The company plans to dispense with that aspect of the therapy in the upcoming
OTHER NEWS TO NOTE
pivotal study – it is present in patients in any case.
Imaginab Inc., of Los Angeles, said it formally launched
A report of the trial, titled "Intracerebral Administration
Imaginab Japan KK, a wholly owned subsidiary based in Tokyo,
of Adeno-Associated Viral Vector Serotype rh.10 Carrying
aimed at better meeting the commercial needs of several
Human SGSH and SUMF1 cDNAs in Children with
significantly clinical development and partnering opportunities,
Mucopolysaccharidosis Type IIIA Disease: Results of a Phase I/
the company said. The subsidiary will be led by Shintaro
II Trial," was published online on May 5, 2014, in Human Gene
Therapy. Three of the four patients treated were between 5.5
and 6 years of age, at which point the condition is relatively
BIOWORLD™ IS ON TWITTER
advanced. All were able to walk, but they had abnormal cognitive abilities and exhibited brain atrophy. The fourth was
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2 years and 8 months and, according to the study authors,
"was the most likely to display neurocognitive benefit." Modest
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
Regulatory front
brincidofovir to a dying 7-year-old boy, whose immune system had been compromised following a round of cancer treatments.
The issue for Chimerix was the cost involved, which could delay
drugs and devices.
its ongoing development program and, thus, the approval and
Unanimously passed by the Louisiana Legislature, the bill was
marketing of the drug for other patients. (See BioWorld Today,
sent to Gov. Bobby Jindal Tuesday for his signature. HB 891
mirrors the legislation Colorado Gov. John Hickenlooper signed
South Plainfield, N.J.-based PTC Therapeutics Inc. ended up in
into law Saturday. Other states are following suit. The Missouri
court when a patient sued for access to its investigational drug.
General Assembly is working on a similar bill, and "right-to-try" While PTC won in district court, it lost on appeal. Rather than will be on the November ballot in Arizona.
draining limited resources in court battles and compassionate
The state legislation does nothing to extend compassionate
use programs, "the right thing is to get the drug approved
use beyond what the FDA already permits, but it shows there
and commercially available as rapidly as possible," PTC CEO
is a problem with getting access to promising therapies, Rep.
Stuart Peltz told BioWorld Today. That way the new treatment is
Harold Rogers (R-Ky.) said at Tuesday's House subcommittee
available to all patients, not just a select few.
hearing on ways to speed the development of new cures. (See
One solution would be for the U.S. to adopt something like
BioWorld Today, May 21, 2014.)
the EU's conditional approval program, which grants access
Citing the lengthy approval process of investigational drugs
to some investigational therapies once clinical trials are
and devices, the Colorado and Louisiana legislation noted that
completed, Peltz said. However, PTC was denied conditional
patients with terminal illnesses don't have the luxury of waiting approval in the EU of its ataluren, a protein restoration therapy until a drug or device receives FDA approval.
designed to enable the formation of a functioning protein in
Although the legislation recognizes a right to try, it does
patients with genetic disorders caused by a nonsense mutation.
not force manufacturers to provide investigational drugs or
The company is appealing. (See BioWorld Today, May 14, 2014.)
devices. It also doesn't require third-party payers to cover the
PTC's 48-week, 220-patient confirmatory phase III trial for
unapproved therapies. And it shields health care providers
ataluren is on track to complete enrollment in a few months,
and drug- and devicemakers from liability associated with the
with top-line data expected in mid-2015. The process of
compassionate use of an investigational product.
developing ataluren from the idea stage has taken 15 to 16
When the experts testifying at the subcommittee hearing
years, Peltz said. During that time, the biotech has had to raise
were asked about the right-to-try bills, Garry Neil, head of
money, develop the clinical path for the novel drug and educate
global R&D at Medgenics Inc., said the cost of providing
regulators about the disease and the science behind the drug.
an investigational therapy for compassionate use can be
CMS FINALIZES RULE
prohibitive for small companies.
After withdrawing the most controversial parts of a proposed
Sara Radcliffe, executive vice president of the health section
rule revising the Medicare Part D prescription drug program,
at the Biotechnology Industry Organization, added that
the Centers for Medicare & Medicaid Services (CMS) has
compassionate use is a difficult, complex issue. The written
finalized the rule.
testimony she submitted detailed the growing delays in
The final rule, which is to be published in Friday's Federal
development that are making compassionate use a front-
Register, gives CMS enhanced tools to fight fraud and abuse
burner issue with patients.
in the prescription drug program. To ensure that Part D drugs
For instance, the duration of the clinical phase of drug
are only prescribed by qualified individuals, the rule requires
approvals has steadily lengthened from an average of 4.6 years prescribers to enroll in Medicare by June 1, 2015. It also gives in 1990-1994 to an average of 7.1 years in 2005-2009. That
CMS the authority to revoke a physician's Medicare enrollment
increase has been accompanied by rising protocol complexities
for abusive prescribing practices and patterns.
and declining enrollment and retention rates.
Another provision will broaden public access to privacy-protected
"Confronting the problem of increasing costs and durations of
Part D data, such as unencrypted, prescriber, plan and pharmacy
clinical trials is a daunting task," Radcliffe said in her testimony. identifiers contained in prescription drug event records.
While industry is committed to partnering with Congress,
The nearly 700-page proposed rule CMS released for comment
the FDA, NIH, patients, academia and other stakeholders to
in January also would have reduced the number of Part D
improve the efficiency of clinical trials to reduce the barriers to
plans a sponsor could offer, set standards on requirements
new therapies, patients who are out of options don't have time
to participate in preferred pharmacy networks, clarified
to wait for those talks to produce results. Many of them have
noninterference provisions and lifted the protected class
turned to social media to pressure drug- and devicemakers to
designation for antidepressants, immunosuppressants and, in
grant them compassionate use.
the future, antipsychotics.
Chimerix Inc., of Durham, N.C., was recently the target of a
The agency backed away from those provisions after it was
social media firestorm to get it to provide its antiviral candidate
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
Editor's note: This is an outtake from BioWorld's Bench Press, a weekly addition to the daily news in which BioWorld takes a look
at translational medicine. For more science news like this, look
inhibitor that they used to probe IDE's role more specifically
for the attachment every Monday morning, or visit BioWorld's
than knockout mice could.
dedicated science portal, The BioWorld Biome: Our Habitat for All
In their studies, they showed that in both lean and obese mice,
Things Science at www.bioworld.com.
IDE regulated not just insulin, but also glucagon and amylin, two hormones that control blood sugar levels. When mice were
Regulatory front
fed glucose, acute treatment with IDE improved their blood sugar levels.
"These findings demonstrate the feasibility of modulating IDE
swamped with more than 7,500 comments and Congress
activity as a new therapeutic strategy to treat type 2 diabetes
threatened to pass a bill preventing it from implementing any
and expand our understanding of the roles of IDE in glucose
Part D provision included in the rule. (See BioWorld Today,
and hormone regulation," the authors concluded. Their work
March 4, 2014, and March 12, 2014.)
appeared in the May 22, 2014, issue of Nature.
‘SAMENESS' TOOL NEEDEDThe FDA will accept applications next month for a multiyear grant of up to $1 million to develop a mathematical algorithm or model to use in determining similarity or sameness of biosimilars and generic versions of complex drugs.
In providing a "coherent picture" of the molecular structure of complex macromolecules or drug substances, the project to be funded by the grant could help determine the extent of preclinical and clinical studies that will be needed to develop copies of the drugs.
Initially, "the model will be used to qualitatively and quantitatively determine the degree of sufficiency of in vitro chemical and biological characterization assays, with respect to demonstrating similarity (or sameness) among multiple batches of the reference product," the FDA said.
Details about the project and grant are available on the NIH's
grants webpage. //
OTHER NEWS TO NOTE
Inhibikase Therapeutics Inc., of Atlanta, said it received FDA
orphan drug designation for imatinib to treat progressive
multifocal leukoencephalopathy (PML), a rare side effect of
small-molecule and antibody drugs given to patients with autoimmune diseases such as arthritis and multiple sclerosis
and a disease that occurs in 1 percent to 3 percent of clinical AIDS patients. Imatinib, the active ingredient in the firm's lead
drug, IkT-001Pro, (and also the active ingredient in Novartis AG's Gleevec), is a host-directed protein kinase inhibitor that disrupts
the ability of JC virus, which is activated in cases of PML.
Lanthio Pharma BV, of Groningen, the Netherlands, said it
was awarded an Innovation Credit loan of up to €3.6 million
(US$4.9 million) by Dutch government agency RVO. The loan is risk bearing and will be matched by Lanthio's investors and is expected to cover up to 35 percent of development costs through to completion of phase II trials. It will support the development of LP2, a lanthiopeptide designed to selectively activate the angiotensin type 2 receptor of the renin angiotensin system, for treatment of idiopathic pulmonary fibrosis.
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
and 2009 and has advised a number of other start-ups.
"When I looked at Cour and compared it to other start-ups, they have an enormous amount of data, an impressive patent
veteran Catherine Mackey as chairman of its board.
portfolio, and have only taken nondilutive funding," Mackey
Founded in 2012, the Elmhurst, Ill.-based Northwestern
noted. "They've built a very solid foundation. As a platform, it's
University spinout is still preclinical. But it already has two yet-
not a one-trick pony."
to-be-named big pharma collaborators in place and anticipates Indeed, Cour, which gained its name after company co-founders
adding a third by the end of the year, Cour CEO and president,
voted over dinner to incorporate the ideas of courage and heart,
John Puisis, told BioWorld Today.
may have many tricks ahead. "There are many discoveries yet to
Cour's immune-modifying nanoparticles (IMPs), based on work
be made," Puisis said. "We're still identifying immune pathways
done at the University of Sydney and Northwestern University,
we want to explore further, and there are lot of opportunities
are derived from biodegradable polymer polylactic-co-glycolic-
left to be realized there." //
acid (PLGA) and have the potential to address a variety of inflammatory conditions.
OTHER NEWS TO NOTE
The negatively charged particles bind to the positively charged
Neurotez Inc., of Ridgewater, N.J., said it executed an exclusive
MARCO receptor of monocytes, directing them to the spleen for license deal covering its Leptin derivatives for the treatment
destruction through apoptosis, thus diminishing inflammation.
of Alzheimer's disease and other cognitive disorders with GCA
Inflammatory monocytes are prevented from entering sites
Therapeutics Ltd., of New York. Under the terms, GCA gets
of injury or inflammation, reducing tissue damage and
rights to clinically develop and commercialize Leptin products
accelerating repair and recovery.
in mainland China, exclusive of Hong Kong, and Taiwan, and
The company's tolerizing immune-modifying nanoparticles
will assume all development and regulatory responsibility. In
(TIMPs) also employ PLGA nanoparticles, but load them with
exchange, Neurotez will be eligible for gross sales milestones of
antigen-specific proteins that induce immune tolerance. In the
up to $102.5 million, plus royalties on gross sales. The license
case of specially programmed TIMPs, the modified particles
also has certain minimum annual sales thresholds following
can be used to deliver toleragenic signals to antigen-presenting first commercial sales in mainland China.
cells, resulting in long-term immune tolerance.
Nuvilex Inc., of Silver Spring, Md., said it, along with the
At the heart of the technology are proprietary modifications
Translational Drug Development (TD2) in Scottsdale, Ariz.,
to the nanoparticles that determine the immune target and
started preparing for a U.S.-based preclinical and clinical
mechanism of action, while the particle itself biodegrades and
studies on the pain and accumulation of fluid in the abdominal
is then cleared by the body.
cavity, two commonly occurring symptoms associated with
So far, Cour has filed patents to encapsulate hundreds of
advanced pancreatic cancer. TD2 will study the effectiveness of
epitopes that modulate cell response. The highly specific TIMPs Nuvilex's pancreatic cancer treatment in relieving the pain and are at the core of its pharma partnerships in type 1 diabetes,
fluid accumulation known as ascites. The treatment combines
celiac disease and a third yet-to-be-named autoimmune
the Cell-in-the-Box live-cell encapsulation technology with
disorder. It's diabetes partner has an exclusive option on its
cancer agent ifosfamide and will be studied to see if it can
investigational TIMP, while its celiac partner has an exclusive
improve the quality of life of pancreatic cancer patients.
evaluation option.
Preclinical studies are set to commence shortly.
Supported by the stable foundation of its partnerships, Cour
Supernus Pharmaceuticals Inc., of Rockville, Md., said
plans to advance its internal IMPs in several cardiovascular
United Therapeutics Corp., of Silver Spring, Md., paid the
indications, including acute myocardial infarction, acute
firm a $2 million milestone upon United's launch of Orenitram
encephalitis syndrome and, once lead optimization is complete, (treprostinil) extended-release tablets for the treatment of ischemic stroke and inflammatory bowel disease.
pulmonary arterial hypertension in the U.S. Orenitram uses
So far, Puisis said, the company has raised enough seed capital a Supernus technology platform. In addition to the launch to fund its needs. "Our goal is to see how these collaborations
milestone, Supernus is eligible for royalties on net sales and
play out and determine our capital needs going forward,"
may be entitled to additional milestones. Orenitram gained
he said. As the company further explores applications for its
approval late last year. (See BioWorld Today, Dec. 24, 2013.)
platform and tackles the strategic thinking necessary to build a stable foundation, it was just the right time to bring Mackey on board, he said.
BIOWORLD™ IS ON TWITTER
Mackey, an experienced corporate director, brings 30 years
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of operating experience in the biopharma and agricultural industries. She served as senior vice president of worldwide
R&D and site director for La Jolla Laboratories between 2001
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
VOLT03 will move to market as a neutraceutical, with quick sales – albeit more modest than a prescription drug – creating
a revenue stream to help offset the remainder of the VOLT01
‘PARTNERING CERTAINLY WOULD BE PREFERRED'
clinical development program.
As Voltarra came together, Desai contacted the Menzies team
Voltarra also is in the middle of a pre-seed round that will likely
to discuss comparative data on VOLT01 and the Novartis drug
stay south of $1 million, Becker said, with plans for a larger
to determine whether the company's findings were consistent
series A on tap for later in the year. Once regulatory filings for
with results they had seen in ZAP. Not only were the data
VOLT01 are under way, the company will decide whether to
in line, but the Menzies researchers approached the four
seek a series B.
sponsoring institutions and the country's regulatory agency, the "Partnering certainly would be preferred," Becker said. "I'm
Therapeutic Goods Administration, seeking to add a third arm
going to need a partner to help from the marketing and sales
to the trial, Becker said.
end anyway. If I can find one to help with the clinical end, as
The proposal was accepted, and VOLT01 was added to the two-
well, that would be ideal, but I'm not counting on that." //
year randomized, multicenter, placebo-controlled, double-blind trial, conducted under the auspices of Medicines Australia.
OTHER NEWS TO NOTE
"The hopes are, whoever wins – and I'm assuming it's not going Wilex AG, of Munich, Germany, said a collaboration agreement
to be placebo – the compound would be moved forward to the
with UCB SA, of Brussels, Belgium, for product candidates
Therapeutic Goods Administration for potential registration" in
WX-554 and WX-037 and three preclinical antibody programs
KOA, likely in 2015, Becker told BioWorld Today.
was terminated by mutual agreement due to an extensive
In the meantime, the trial includes a pre-planned six-month
restructuring at Wilex, which the company disclosed in January.
interim analysis after the study is fully enrolled, which Becker
The strategic alliance, inked early in 2009, was worth €10
estimated will occur in the first quarter of 2015. At that point,
million up front (US$13.5 million) and a potential €10 million
"we are going to know how VOLT01 has performed vs. Aclasta"
in two near-term milestones to Wilex, which will return to
in terms of safety and efficacy, he said. Though long-term
UCB all rights and transfer all intellectual property, data and
monitoring will continue in the study, Voltarra plans to package documents generated in connection with the programs. An
the early data and request face-to-face meetings with the
agreement regarding an antibody program for non-oncology
FDA and the EMA to seek guidance on the design of a global
indications that was signed last year was terminated. Wilex
registration study.
will receive a final payment from UCB for R&D expenses, and
Provided the company can conduct those meetings by mid-
UCB agreed to waive repayment of the €2.5 million shareholder
2015, Becker predicted the phase III registration program could
loan granted in December 2010 on completion of the transfer.
be completed by 2017, with subsequent regulatory filings
Termination of the agreement does not affect UCB's role as a
steering the product toward approval in 2018.
Wilex shareholder. (See BioWorld Today, Jan. 12, 2009.)
In the U.S., Voltarra expects to file its new drug application for VOLT01 under the 505(b)(2) pathway, "which saves a
boatload of money, since we don't have to go back and reinvent Amgen, of Thousand Oaks, Calif., said The New England Journal
the wheel," Becker said, noting that the drug's preclinical,
of Medicine published results from a phase I study suggesting
phase I and tox studies are complete. The company hopes
that inhibiting thymic stromal lymphopoietin (TSLP) could
to file concurrently with the EMA, using essentially the same
benefit the treatment of asthma. TSLP is a cytokine thought
data package. The company has filed provisional and non-
to be a key driver of allergic inflammation. Results from the
provisional patents and expects to hold a broad patent estate
31-patient proof-of-concept study showed treatment for 12
for VOLT01 to prevent and treat osteoarthritis.
weeks with AMG 157, a monoclonal antibody that inhibits the
To fund its ambitious program, Voltarra has a complex
activity of TSLP, resulted in statistically significant reductions
curcumin, dubbed VOLT03, that is sitting "in the parking
in early asthmatic responses and late asthmatic responses
lot" while other companies conduct proof-of-concept studies
in the airways following allergen challenges in patients with
with similar compounds across applications in oncology,
allergic asthma. The data also showed statistically significant
inflammatory indications and neurodegenerative diseases.
decreases in baseline markers of inflammation in the airways.
Approximately 80 trials with curcumin alone or in combination
Overall, adverse events were similar across treatment and
with other interventions are under way, according to Cortellis
placebo groups, with no serious adverse events. The compound,
Clinical Trials Intelligence, with most in phase II or III.
in joint development with Astrazeneca plc, of London, through
"We're just sitting back to see if one of those trials is positive,"
its biologics arm Medimmune, is in phase II development
Becker admitted. "Then, potentially, we can move into that
in asthma. The phase I findings also were presented at the
space with our product, which is also reformulated to make the
American Thoracic Society international conference in San
curcumin more soluble."
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
wash), improvements in total mucus weight (the amount of mucus produced by the nose) and also symptom diary score
Antibe Therapeutics Inc., of Toronto, said it submitted a
compared to placebo. The results are being presented during
clinical trial application to Health Canada for lead drug ATB-
a poster discussion session at the American Thoracic Society
346, proposing a phase I study to assess the safety, tolerability
conference in San Diego.
and pharmacokinetics of single and multiple-ascending doses
Intelgenx Corp., of Saint Laurent, Quebec, and Redhill
in healthy subjects. The trial is slated to start this summer. ATB- Biopharma Ltd., of Tel Aviv, Israel, reported data from a
346 is a hydrogen sulfide-releasing derivative of naproxen.
comparative bioavailability study with migraine candidate
Atyr Pharma Inc., of San Diego, said it completed a phase I
Versafilm, an oral thin film formulation of rizatriptan for acute
study for its lead program, Resokine IV, which marked the first
migraines. Results of the study are subject to final quality
ever administration of a physiocrine to humans. The double-
assurance and an independent study report by the Canadian
blinded, placebo-controlled trial was conducted in healthy
clinical research organization that conducted the trial, but the
male and female subjects in the European Union to assess
companies said the data will support the planned submission
the safety and tolerability of Resokine IV, in development
in Europe for the product during the third quarter.
as a potential therapeutic for rare immune disorders. The
Nymox Pharmaceutical Corp., of Hasbrouck Heights,
company said the data from 32 healthy subjects in four
N.J., reported results from the Brief Male Sexual Function
dose cohorts support the basic premise of Resokine IV as
Questionnaire in its recently completed NX03-0040 study of
an immunomodulator rather than a classic interleukin or
NX-1207 in low-grade localized prostate cancer, indicating that
interferon with systemic immunostimulating properties upon
targeted treatment with the drug at either dose – 2.5 mg or
administration. Among their various homeostatic functions,
15 mg – had no significant effect on reported sexual function
some physiocrines act as extracellular signaling molecules
score post-treatment. Nymox also is testing NX-1027 in benign
to orchestrate immunohomeostasis in response to stress and
prostatic hyperplasia and recently completed the second
other physiological changes. Physiocrines comprise naturally
pivotal trial in that indication.
occurring proteins derived from tRNA synthetases that play
Summit plc, of Oxford, UK, said SMT C1100, an oral small-
fundamental roles in the function of human physiology and
molecule utrophin modulator for the treatment of Duchenne
restoring pathophysiological states to a healthier state.
muscular dystrophy (DMD), successfully met its primary
Celsion Corp., of Lawrenceville, N.J., said the FDA has green-
endpoint of safety and tolerability in a phase Ib trial in patients
lighted its planned pivotal phase III trial of Thermodox, its
with the disease. The dose-escalating trial was conducted in 12
heat-activated liposomal encapsulation of doxorubicin, in
patients with DMD, between 5 and 11 years old. The nonplacebo-
combination with radio frequency ablation (RFA) in primary
controlled study also measured levels of creatine kinase (CK), an
liver cancer, or hepatocellular carcinoma. The trial will enroll
enzyme associated with muscle fiber damage that is elevated in
550 patients globally and evaluate Thermodox in combination
boys with DMD, and results found that In the majority of patients
with RFA vs. standardized RFA alone. The primary endpoint
there was a reduction in CK levels during dosing with SMT C1100.
for the trial is overall survival. An independent data monitoring
Those data are consistent with nonclinical in vivo efficacy studies
committee will conduct two interim analyses. Celsion said it
in the mdx model of DMD that showed SMT C1100 reduced CK
expects to launch the study before the end of June.
levels after only 15 days. The company plans to review those
Gilead Sciences Inc., of Foster City, Calif., reported results from preliminary data, which are expected to lead to a revision of
a placebo-controlled, phase IIa challenge study of GS-5806,
future clinical trial plans in order to determine the optimal way,
an investigational oral RSV fusion inhibitor, in healthy adult
either through dietary means or drug formulation changes, to
patients intranasally infected with respiratory syncytial virus
address the drug uptake differences between DMD patients and
(RSV). The trial achieved its primary and secondary endpoints
healthy volunteers. The next patient study is now expected to
of lower viral load (the amount of virus detected in the nasal
start in the fourth quarter.
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THURSDAY, MAY 22, 2014
BIOWORLD™ TODAY
PHARMA: OTHER NEWS TO NOTE
Netherlands, Sweden, Norway, Denmark, Ireland and Italy.
Daiichi Sankyo Co. Ltd., of Tokyo, began a three-year research
Abbvie Inc., of Chicago, said the FDA granted Humira
project with Sanford-Burnham Medical Research Institute, of La
(adalimumab) orphan drug designation for the treatment of
Jolla, Calif., to investigate cardiovascular-metabolic diseases.
non-infectious intermediate, posterior or pan-uveitis, or chronic Terms of the agreement, which will deploy Sanford's screening non-infectious anterior uveitis, a group of rare but serious
capabilities, were not disclosed.
inflammatory diseases of the eye. Abbvie is investigating
Novo Nordisk A/S, of Bagsvaerd, Denmark, said the FDA
the efficacy and safety of Humira for the treatment of non-
tentatively scheduled an advisory committee meeting for Sept.
infectious uveitis, and the clinical program is in phase III
11 to discuss the new drug application (NDA) for liraglutide 3
mg for the treatment of obesity. The NDA was submitted to the
Baxter International Inc., of Deerfield, Ill., said the FDA
FDA last December.
extended the PDUFA date for its review of the biologics
PHARMA: IN THE CLINIC
license application for Hyqvia (immune globulin infusion 10 percent [human] with recombinant human hyaluronidase), the
Boehringer Ingelheim GmbH, of Boehringer, Germany,
company's investigational subcutaneous treatment for patients presented results from the phase III VIVACITO study at the with primary immunodeficiency. The FDA is requiring additional American Thoracic Society meeting in San Diego, showing time to review supplemental data that Baxter provided as part
that the fixed-dose combination of tiotropium and olodaterol,
of the ongoing process for approval. The PDUFA date has been
delivered via the Respimat inhaler, demonstrated a statistically
extended by three months, which is the standard extension
significant improvement over both monotherapy treatment (p
period. As part of the FDA's extended review, Baxter now
< 0.0001) and placebo (p < 0.0001) in the primary endpoint,
expects to participate in a Blood Products Advisory Committee
defined as the forced expiratory volume in one second.
meeting, which the agency has scheduled for July 31. Hyqvia
Secondary endpoints included additional tests measuring
was approved by the European Commission in 2013 and is
breathing over 24 hours. The overall incidence of adverse
available in several European countries, including Germany, the events was comparable among treatment groups.
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