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MARCH 8 , 2016 VOLUME 27, NO. 45BIOTECH’S MOST RESPECTED NEWS SOURCE FOR MORE THAN 20 YEARS
BIOWORLD TODAYTM
THE DAILY BIOPHARMACEUTICAL NEWS SOURCE
See Celldex, page 3 See Abbvie, page 4
See EMA, page 6
See Spark, page 7
See DBV, page 5
RINDO AT INTERMISSION AS CONTROL ARM OVERPERFORMS
IN THE CLINIC
IN THIS ISSUE
REGULATORY
NEWCO NEWS
DEALS AND M&A
See Capella, page 8
Regulatory front, p. 2
Other news to note, p. 2, 4, 5, 9, 11, 12
In the clinic, p. 9-11, 13
Financings, p. 14
Phase II data keep DBV’s
Viaskin peanut allergy
therapy on track
By Cormac Sheridan, Staff Writer
DUBLIN – DBV Technologies SA reported
further progress from its patch-based
peanut allergy therapy, Viaskin Peanut,
at the American Academy of Allergy,
Asthma and Immunology (AAAAI)
Abbvie strikes $595M
deal for Boehringer
anti-IL-23 MAb
By Michael Fitzhugh, Staff Writer
Abbvie Inc. agreed to pay Boehringer
Ingelheim GmbH $595 million up front
for the opportunity to co-develop and
later sell BI 655066, a phase III anti-IL-23
monoclonal antibody for psoriasis that
could eventually help the North Chicago-
based company replace revenue losses
that it will face as its largest product,
Celldex lowers curtain on ACT IV phase III
in glioblastoma; subset data under study
By Randy Osborne, Staff Writer
Regarding the stopped phase III glioblastoma trial with Rintega (rindopepimut),
Celldex Therapeutics Inc. CEO Anthony Marucci said the firm is “well positioned to
weather this temporary setback,” and during the weeks ahead will ransack data from
ACT IV for clues to why control arm patients did just as well as those on the drug.
Thomas Davis, chief medical officer (CMO), said the firm learned only late Friday that
the data safety monitoring board (DSMB) recommended Celldex pull the plug on the
study in patients with newly diagnosed, epidermal growth factor variant III (EGFRvIII)-
Antibody firm Capella
raises $15.5M for novel
targets, good biology
By Nuala Moran, Staff Writer
LONDON – The U.K.’s latest antibody
company, Capella Biosciences Ltd.,
announced the closing of an £11 million
(US$15.5 million) series A round,
providing the funding to take the lead
EMA launches Prime
to accelerate, improve
regulatory process
By Nuala Moran, Staff Writer
LONDON – The EMA has launched
its latest scheme for accelerating the
assessment of drugs, promising support
tailored to the stage of development,
ongoing access to its approvals
‘Spark’ plug: Genable’s
Rhonova adds inherited
retinal disease asset
By Marie Powers, News Editor
Two years after inking an agreement
to provide manufacturing services and
clinical development assistance for
GT038, the lead program at Genable
Technologies Ltd., Spark Therapeutics
Inc. is acquiring the Dublin-based
company and its technology, now known
as Rhonova. Terms call for Spark to pay
Genable shareholders $6 million in cash
and 265,000 shares of Spark’s common
stock (NASDAQ:ONCE), valued at $9.15
million, based on Friday’s closing price
of $34.53. Additional details were not
disclosed, but Genable said its board’s
endorsement of the deal was unanimous.
Although Genable will operate as a
wholly owned subsidiary and remain
in Ireland, the deal gives Philadelphia-
based Spark control over the future of
Rhonova, which targets the treatment of
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REGULATORY FRONT
OTHER NEWS TO NOTE
The FDA is launching a lean management mapping approach
to build a better system to review combination products
that include a device and a drug or biologic. With an eye on
encouraging innovation and supporting the development
of the products, the agency aims to create a review process
that’s efficient, consistent and predictable, according to an
FDAvoice blog posted Monday. As a first step, the agency will
look at how combination products are currently being reviewed,
highlighting areas of delay or redundancy. Creating a baseline
map will allow the FDA to identify metrics for success and
assess the impact of improvements as they’re implemented.
The second step will be to develop a “future state” map
showing a streamlined, efficient process that will eliminate
delays and redundancies.
The National Cancer Institute (NCI) is considering the
development of a specimen bank for tube and ovary tissues
from women undergoing surgery for benign conditions, risk
reduction and early stage cancer. Collecting tube and ovary
tissues containing clinically unsuspected precursors or early
stage cancer has been challenging, especially from women
who are not at increased genetic risk, according to an NCI
notice to be published in Tuesday’s Federal Register. However,
many pathology laboratories have enhanced their processing
protocols for gynecologic surgical specimens removed for
benign indications, which might make a specimen bank
possible. The NCI is proposing a survey to determine the
volume of samples at different pathology labs and the methods
the labs use to process the samples. The survey results would
enable the NCI to determine the feasibility of establishing a
tissue bank for research and provide insights into the design of
a pilot study. Comments on the survey are due by May 7.
The FDA finalized a draft guidance released nearly a year
ago about when sponsors of drugs with potential estrogenic,
androgenic or thyroid (E, A or T) activity should submit an
environmental assessment (EA) or a claim of categorical
exclusion. The guidance also clarifies what information should be
included in an exclusion claim, according to a notice published
in Monday’s Federal Register. Since late cycle requests for
additional environmental information could delay approval,
sponsors should consult with the FDA early on about the
information that may be needed to determine whether an EA or
an exclusion claim would be required. Failure to submit an EA or
exclusion claim for a drug with E, A or T activity could lead to the
FDA refusing to file an application or denying approval.
Adma Biologics Inc., of Ramsey, N.J., presented findings from
preclinical experiments conducted in collaboration with Baylor
University College of Medicine at the American Academy of
Allergy Asthma and Immunology meeting in Los Angeles.
The studies compared the effects of RI-002, an intravenously
administered human immunoglobulin preparation containing
a high titer anti-respiratory syncytial virus (RSV) polyclonal
antibody, to monoclonal anti-RSV antibody Synagis
(palivizumab, Medimmune) in cotton rats infected with a
palivizumab-resistant strain of RSV. The data suggested that
RI-002 and palivizumab were comparable in diminishing the
viral load in the lungs of RSV-infected cotton rats but only RI-
002 prevented RSV infection when a palivizumab-resistant
strain of RSV was administered. Serum neutralization titers
achieved by palivizumab at the approved dose level of 15 mg/
kg were about fourfold lower than those achieved by a dose of
1,500 mg/kg of RI-002. (See BioWorld Today, Feb. 26, 2015.)
Arrowhead Research Corp., of Pasadena, Calif., presented
preclinical data at the American Academy of Allergy, Asthma
and Immunology meeting in Los Angeles, suggesting that
ARC-F12, an RNAi therapeutic that inhibits the production of
factor XII, has the potential to treat hereditary angioedema
and to prevent thrombosis. Data showed that ARC-F12 had the
desired effects of significantly reduced swelling in a rat model
of edema and inhibition of blood clot formation in a mouse
model of thrombosis, without the undesired effect of increased
bleeding risk. The company is conducting studies in order to
advance ARC-F12 into clinical trials.

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Celldex
Continued from page 1
positive disease, since it would not hit statistical significance
on the primary endpoint: overall survival (OS) in those with
minimal residual disease. DSMB investigators found that the
Rintega arm and the control arm data worked out about the
same, and nobody knows why. “Obviously, there’s something
we’re missing,” he said.
Shares of Hampton, N.J.-based Celldex (NASDAQ:CLDX)
closed Monday at $3.79, down $4.40, or 53.7 percent.
Davis called the ACT IV experiment “relatively complicated”
with “high stakes. Hence, we chose a very experienced DSMB,
including two senior statisticians who fully understand all
of the calculations and ramifications. They looked at the full
complement of data available to them at all endpoints before
making a decision” to put the kibosh on the study. “Right now,
we’re just poring through the data so we can’t tell you what
additional signs or signals might be in there,” he said. “We’ll
talk about that later. [But] the DSMB did the job they were
supposed to do in looking at the study overall, and determining
there was not a chance it would be positive at the end of
the day.” He called the outcome “unfortunate” with “broad
ramifications for scientists and patients.”
In ACT IV, Rintega worked like it has in phase II studies, but the
control arm results turned up way better than history would
suggest: hazard ratio = 0.99; median OS: RINTEGA 20.4
months vs. control 21.1 months. All patients on the Rintega arm
of the ACT IV study, as well as those from prior phase II studies
and existing compassionate use recipients, will be offered
ongoing access to Rintega. Individual patients may be getting
benefit from the drug, and “we would hate to pull the rug out
from under [those] patients” Davis said, speaking to investors
during a conference call.
Roth Capital Partners analyst Joseph Pantginis called the ACT
IV results “a major shock,” especially the “unprecedented”
showing of the control arm, since survival with standard-of-
care therapy is generally around 15 months. “The question is,
what now?” he wrote in a research report. “We do not believe a
downgrade is warranted, based on the quality of the company’s
pipeline and upcoming news flow.” Celldex has five active
programs and is “financially strong,” he noted.
Company officials said cash on hand is sufficient through
2017, which had been the case earlier, Brean Capital analyst
Jonathan Aschoff pointed out during the call. “I’m curious
as to why the cash runway isn’t something you can say today
is longer than it was two weeks ago,” he said. CEO Marucci
replied that “we’re going to have to go through the budget,
as you would expect. What we can say, 48 hours after the
[DSMB] event, is that we still have cash through 2017. This is
just a preliminary estimate. I’m sure we’ll tighten our belt even
further.” Asked whether Celldex would “look more aggressively
to purchase something or ramp up internal discovery” post-
ACT IV’s blowup, he said the firm “keep[s] our options open and
we’re always looking at different things.”
NEXT UP, VARLILUMAB
Meanwhile, the mystery remains. “This happens constantly,”
Aschoff said, “where people are blown away by the control-arm”
performance in phase III trials after a smaller, encouraging phase
II studies led researchers to hope for better. He wanted to know if
Celldex has any preliminary theories.
CMO Davis said the matter is “something we spent a lot of
time thinking about this weekend. In most other diseases,
there’s a broader range of survival and you can identify
patient populations within your group who will do well.” In
glioblastoma, though, the survival range is narrower and
usually more predictable. “We can’t come up with any reason
for this right now,” he said.
At last year’s meeting of the American Society of Clinical
Oncology (ASCO) in Chicago, Celldex’s phase II data with
Rintega, an immunotherapy vaccine targeting EGFRvIII, created
a major buzz. The trial called React had yielded first results the
previous November, when investigators reported interim data
through October 2014. Numbers presented at ASCO included
outcomes through March, showing that, of patients given
Rintega plus standard-of-care Avastin (bevacizumab, Roche
AG), 45 percent were alive after 12 months, compared with
31 percent absent Rintega. A median OS rate of 11.6 months
turned up in the Rintega cohort, compared with 9.3 months
in the control group. Patients were Avastin-naïve when they
started the trial. While the data were early and continue to
mature, the company said, at 18 months, 30 percent of Rintega
patients lived vs. 15 percent of control subjects. (See BioWorld
Today, Nov. 17, 2014, and June 2, 2015.)
The drug, which gained breakthrough status from the FDA in
February 2015, seemed set up for a win in phase III. “We will be
looking to identify potential biomarkers or subsets,” Davis said.
“We hope to better understand what happened here, and we’ll
use that information to improve our research moving forward.
We said before that, from a regulatory perspective, this [React]
study really was just too small. It didn’t have adequate power to
justify approval. As disappointing as the ACT IV data are, [they
don’t] change that position.”
Oppenheimer analyst Christopher Marai said Celldex investor
focus likely will shift now to CD27-targeting varlilumab, subject
of a 2014 deal with New York-based Bristol-Myers Squibb Co.
(BMS) for testing with the latter’s approved PD-1 checkpoint
inhibitor Opdivo (nivolumab) in non-small-cell lung cancer
(NSCLC). BMS made a one-time payment of $5 million to
Celldex, and the parties agreed to share development costs
of the effort, which could turn up efficacy in NSCLC and more.
“Varlilumab’s advancement into phase II is promising, in our
view, as BMS is funding half the cost,” Marai wrote in a research
report, and phase I results are expected in the first half of this
See Celldex, page 5

Abbvie
Humira (adalimumab), begins to lose patent protection and
incur competition from biosimilars.
Ingelheim, Germany-based Boehringer will be eligible to
receive additional development and regulatory milestone
payments as part of the deal as well as royalties on net
sales, the terms of which are not disclosed. In the initial
period, the companies will share the responsibility for
development of BI 655066. Abbvie will be solely responsible
for commercialization, while Boehringer will retain an option to
co-promote the compound in asthma, another indication the
partners are exploring, alongside Crohn’s disease and psoriatic
arthritis. The candidate has entered phase II studies for both
asthma and Crohn’s and is about to enter phase II development
for psoriatic arthritis.
As it advances, BI 655066 could also create competition for
Janssen Biotech Inc.’s anti-IL-12/IL-23 psoriasis drug, Stelara
(ustekinumab), a therapy that generated $2.47 billion in 2015
sales. More than 100 million people suffer from psoriasis, a
chronic, systemic, immune-mediated disease.
Boehringer reported phase II data in October showing that BI
655066 demonstrated superior efficacy over Stelara. After nine
months, 69 percent of patients with moderate to severe plaque
psoriasis maintained clear or almost clear skin in the higher
dose group, compared to 30 percent of patients on Stelara.
Patients also achieved skin clearance significantly faster – in
about eight weeks vs. about 16 weeks – and for two months
longer. Completely clear skin was maintained after nine months
in 43 percent of BI 655066 patients compared with 15 percent
of Stelara patients. The data were presented at the 24th
European Academy of Dermatology and Venereology congress
in Copenhagen.
The candidate is administered as a subcutaneous injection and
was generally well tolerated in the 12-week treatment portion
of the phase II study. Serious adverse events were reported for
patients in the BI 655066 18-mg treatment group (7 percent)
and Stelara group (2.5 percent), while there were no serious
adverse events in the 90-mg and 180-mg treatment groups.
One patient discontinued treatment in the BI 655066 18-mg
dose group. The most common adverse events across the BI
655066 treatment groups were common cold and headache,
at 7.3 percent – 11.6 percent across the dose groups and 2.4
percent – 7 percent, respectively. The most common adverse
events in the Stelara group were common cold, injection site
pain and redness, sore throat and myalgia; each of which
occurred in 5 percent of patients.
Abbvie also gained rights to another Boehringer candidate, the
anti-CD-40 antibody, BI 655064, which is currently in phase I
development. Boehringer is retaining responsibility for further
development of that drug, but Abbvie now has the option to
advance the program after it hits certain undisclosed clinical
milestones. //
OTHER NEWS TO NOTE
Antabio SAS, of Labege, France, said it achieved two
milestones in its antibacterial drug discovery collaborations
with the Wellcome Trust, resulting in the release of a further
€3.1 million (US$3.4 million) from the seeding drug discovery
awards. The first collaboration began in February 2013 when
the Wellcome Trust awarded Antabio €4.7 million to fund the
discovery of a safe and efficacious inhibitor of bacterial metallo
beta-lactamases (MBLs). Antabio has identified a highly active
in vivo lead series with drug-like features and MBL coverage
against clinical isolates, including NDM variants. The Wellcome
Trust aims to nominate a preclinical development candidate by
the first quarter of 2017. The second collaboration began in May
last year when the Trust awarded Antabio €4 million to fund
the development of small-molecule drugs for the treatment of
chronic Pseudomonas aeruginosa infections in cystic fibrosis
patients. Antabio said it has identified a selective in vitro lead
series with all the physicochemical properties required for the
achievement of the first milestone within that program.
Astrazeneca plc, of London, said its biologics research and
development arm, Medimmune, has received fast track
designation from the FDA for its human monoclonal antibody,
MEDI8852, for the treatment of patients hospitalized with type
A strain influenza. MEDI8852 is being evaluated in a phase
Ib/IIa trial to investigate the safety and efficacy of a single
intravenous dose in combination with Tamiflu (oseltamivir,
Gilead Sciences Inc. and Roche AG) , and as a monotherapy in
adult patients with confirmed acute, uncomplicated influenza
caused by type A strains.
Biocryst Pharmaceuticals Inc., of Research Triangle Park,
N.C., said a preclinical study of its broad-spectrum antiviral,
BCX4430, in immune-deficient mice infected with Zika virus
showed that overall survival for the standard dose level of
BCX4430 was superior to both placebo and ribavirin treatment
control groups (p < 0.0001). Median survival exceeded 28
days for animals treated with the standard dose of BCX4430
and was 23 days for the low dose, compared to 14 to 17 days
for controls. In the standard-dose group, seven of eight mice
survived through day 28. No animals survived to day 28 in the
low-dose, ribavirin or placebo groups.
Cellectar Biosciences Inc., of Madison, Wis., completed a 1-for-
10 reverse stock split of its common stock, reducing the number
of outstanding common shares to about 860,000.
Citius Pharmaceuticals Inc., of Maynard, Mass., said it entered
a nonbinding letter of intent to acquire all of the outstanding
shares of Leonard Meron Biosciences Inc., (LMB) of Cranford,
N.J., and its lead drug candidate, Mino-Lok, an antibiotic
lock solution used to treat patients with catheter-related
bloodstream infections. Mino-Lok is entering phase III trials.
Citius is expected to issue shares of its stock in exchange for
shares of LMB in which, after the completion of the transaction,
all prior common shareholders of LMB will retain about 49
percent of the common stock in Citius.

DBV
congress in Los Angeles over the weekend, including 52-week
data from a phase II trial conducted by the Consortium of
Food Allergy Research (Cofar) and sponsored by the National
Institute of Allergy and Infectious Disease.
The trial, called Cofar6, met its primary endpoint of
desensitization to peanut protein, defined as an ability to
tolerate 5,044 mg of protein, or a 10-fold increase from
baseline in the maximum tolerated dose.
Shares of DBV (NASDAQ:DBVT) gained $3.96, or 15.1 percent,
Monday to close at $30.11.
The study assigned 75 patients (54 children, ages 4 to 11; and
21 adolescents and young adults, ages 12 to 25) to one of two
dose arms (100 mcg or 250 mcg) or placebo, on a 1-to-1-to-1
basis. Each dose arm attained the primary endpoint. In the
high-dose group, the overall response rate was 48 percent
vs. 12 percent in the placebo group, a difference that was
statistically significant (p=0.03). One-third of those on the high
dose could tolerate a dose of peanut protein of at least 1,000
mg, equivalent to four peanuts.
The response rate increased when children, ages 4 to 11,
only were considered – to 61.1 percent vs. 5.6 percent. “It is
a very strong response,” David Schilansky, chief operating
officer of Paris-based DBV, told BioWorld Today. Treatment
adherence was 97.1 percent, while the study had a dropout
rate of 8 percent. No withdrawals occurred in the high-dose
arm, and no emergency use of epinephrine was recorded. DBV
has dubbed its patch-based delivery system as epicutaneous
immunotherapy. Unlike most other patches, it delivers antigen
directly to intact skin, trapping it within the stratum corneum.
Systemic delivery does not occur.
The study confirms data from the company’s own phase IIb
Vipes trial, in which 50 percent of patients (ages 6 to 55) in the
250-mcg dose arm responded to the therapy – defined as an
ability to tolerate at least 1,000 mg of peanut protein, or a 10-
fold increase over baseline in the amount they could tolerate –
vs. 25 percent in the placebo group.
Significantly, the Cofar data also provided the first evidence
in patients that the Viaskin therapy dampened the Th2
humoral response in recipients without prompting a shift to a
Th1 cellular immune response. A marked decline in levels of
IgE was detected, whereas, CEO and Chairman Pierre-Henri
Benhamou said, a halt in the increase of IgE is more common
in clinical trials. “Here it’s 40 percent below baseline,” he told
BioWorld Today.
At the same meeting, the company also reported two-year
follow-up data from the Vipes study. The fraction of patients
able to consume 1,000 mg or more of peanut protein almost
doubled from 33 percent to 60 percent. “It shows that by
adding another year of therapy, you continue to increase the
therapeutic benefit,” Schilansky said. The overall response rate
at two years was 69.7 percent, up from 50 percent at one year.
In children ages 6 to 11, the two-year response rate was 80
percent, up from 53.6 percent at one year.
The study outcomes augur well for DBV’s ongoing phase III
trial, which is recruiting patients from the 4 to 11 age range
only. Enrollment is due to be completed in the third quarter of
this year, and data are expected before the end of next year. A
regulatory decision could follow in 2018 – the program has fast
track status from the FDA.
Also at AAAAI, the company demonstrated preclinical proof
of concept for the Viaskin technology in desensitizing the
hemophiliac mice to factor VIII during replacement therapy.
“For the moment it’s research data. We need to have further
confirmation to move into the clinic with this program,”
Benhamou said.
DBV is currently valued at €1.1 billion (US$1.2 billion) and
exited 2015 with €324 million on its balance sheet. It plans
to use that cash to complete the clinical development and
commercialization of the product – it aims to market the
therapy itself in U.S. and Europe but will enter deals in other
regions. “We have enough cash to carry out our mission and
bring the product to market,” Schilansky said. //
year. The companies have completed dose-escalation work and
chose 3 mg/kg for varlilumab, with no maximum tolerated dose
reached. Phase II experiments are expected to start enrolling in
the second quarter of this year and include patients with solid
tumors. (See BioWorld Today, May 15, 2014.) //
Celldex
OTHER NEWS TO NOTE
CSL Behring, of King of Prussia, Pa., said the FDA approved
Idelvion (coagulation factor IX), its long-acting albumin
fusion protein linking recombinant coagulation factor IX with
recombinant albumin for the treatment of hemophilia B. The
company said the product is the first and only factor IX therapy
that delivers high-level protection with up to 14-day dosing in
appropriate patients. That dosing interval has been achieved
while maintaining high levels of factor activity, above 5 percent
over 14 days at 75 IU/kg, which the company said reduces the
monthly number of units needed for prophylaxis therapy.
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EMA
committee and more help with plotting a route through to
approval.
The new priority medicines (Prime) designation also will provide
enhanced support at the early stages of development for small
biotechs and academic groups that have limited experience of
the regulatory system.
Small and medium-sized entities (SMEs) will be eligible
to secure Prime designation prior to phase II, while larger
companies can sign up before starting phase III. Anyone
seeking the designation will be required to provide compelling
evidence of a product’s potential public health impact.
Overall, the scheme should lead to better informed
development plans and improve the quality of marketing
submissions. EMA committees will have greater awareness and
knowledge of products coming up the pipeline, enabling the
review process to be abbreviated.
Launching the scheme at EMA’s headquarters in London, Guido
Rasi, executive director of the EMA, said that currently two out
of three development programs are inadequate to produce
the evidence the agency needs to make its assessments. “As
a consequence, many of the very promising medicines fail to
reach patients, resources are wasted, costs increase and more
importantly patients are enrolled in useless trials.” That is
particularly the case for studies sponsored by academic groups
and SMEs, Rasi said.
In addition to honing regulatory strategies, health technology
assessment agencies will provide input on how development
should be shaped to generate information for reimbursement,
pricing and access bodies, a move that the EMA claims will
improve resource planning in Europe’s hard-pressed health
care systems.
“Ultimately, this will promote the possibility of earlier access to
promising new medicines,” the EMA said.
The agency is responding to critics who complain its convoluted
assessment procedures are slowing access to innovative drugs,
both undermining commercial potential and failing patients.
EU Health Commissioner Vytenis Andriukaitis said Prime is a
major step forward in providing earlier access to treatments
for unmet medical needs such as rare cancers, Alzheimer’s
disease and other dementias. “This scheme could also help, for
example, to accelerate the development and authorization of
new classes of antibiotics,” he said.
In addition, Andriukaitis said Prime will optimize the use of
the regulatory framework in order to boost pharmaceutical
innovation and employment in the sector.
Over the past year, the EMA’s Committee for Medicinal
Products for Human Use (CHMP) has worked with other EMA
committees, and in consultation with national drugs regulators,
on re-interpreting the agency’s legislative framework to devise
Prime. The result is a scheme which will take time and cost out
of clinical development and make better use of the broad range
of expertise to which the EMA has access, giving a helping
hand to small biotechs and academics with exciting science but
limited resources. (See BioWorld Today, Nov. 9, 2015.)
The goal is to foster better development strategies to help
companies generate the high quality data the EMA needs, Rasi
said. “Patients with no or insufficient treatments could then
benefit from scientific progress and cutting-edge medicines as
soon as possible.”
Zaide Frias, head of human research and development at
the EMA, told BioWorld Today it is not possible to say to what
extent Prime will cut development timelines. “It is difficult to
predict how much faster Prime will be; we will have to gain
experience,” she said.
Frias noted that to back up the aims of the Prime scheme, the
European Commission has said it will cut the time it takes to
rubber stamp EMA recommendations to approve a drug, from
22 days to 10 days.
“So we are confident there will be earlier access, but at this
time we can’t [quantify] this, Frias said.
Similarly, Hans-Georg Eichler, senior medical officer of the
EMA, told BioWorld Today it is not possible to say if approval
rates will go up. “Prime can’t rescue a molecule that is not
beneficial or causes a nasty adverse event, but it can help avoid
avoidable errors on the part of companies,” Eichler said.
A HIGH LEVEL OF ENGAGEMENT
Only innovative products will make the cut, with the EMA
saying that providing an alternative treatment or incremental
benefit will not qualify for Prime. Rather companies will need
to demonstrate potential to address “to a significant extent” an
unmet medical need.
That could include the duration of effect, as is expected to be
the case for regenerative and gene therapies, where one or a
few treatments may show long-term efficacy.
The review of applications for Prime designation will be
carried out by the Scientific Advice Working Party, with
recommendations forwarded to CHMP for rubber-stamping.
Although the EMA intends to publish details of all applications,
it will withhold the names of products that fail to win Prime
status. The agency said this is because failure to secure the
designation does not signify that a product is no good.
SMEs and academic groups whose products are accepted for
Prime may also get reduced-price scientific advice.
A key feature of the Prime scheme is that products which
qualify will be assigned a rapporteur from CHMP, who will
act as chaperone. There will also be access to other EMA
committees, for example the Committee for Advanced
Therapies, as appropriate.
Chair of CHMP Tomas Salmonson said, “By strengthening
collaboration between the scientific committees, and by
See EMA, page 9

Spark
rhodopsin-linked autosomal-dominant retinitis pigmentosa, or
RHO-adRP, an inherited retinal disease that, in severe cases,
leads to blindness.
“We certainly have an interest in expanding the remit of AAV-
mediated gene therapy,” Katherine High, Spark’s co-founder,
president and chief scientific officer, said of the deal’s rationale.
“If we can tackle autosomal-dominant diseases as well as the
X-linked and autosomal-recessive diseases that we’re working
on now, that just expands our armamentarium.”
Two years ago, Jason Loveridge, Genable’s CEO, called the
collaboration with Spark a “coup,” telling BioWorld Today the
original partnership was “really driven by the quality of the
science. Spark is not a contract manufacturer.” (See BioWorld
Today, March 26, 2014.)
In return for a license to its adeno-associated virus (AAV)
manufacturing intellectual property, Spark received revenues
from the manufacture and supply of the product and was
entitled to development milestones and sales royalties.
Genable, in turn, was able to piggyback on the experience
of Spark’s team. The Irish firm’s gene suppression-and-
replacement approach is designed to address all mutations
in the gene encoding rhodopsin. The protein, a photosensitive
G protein-coupled receptor, is expressed in retinal cells and
plays a central role in the transduction of light to electrical
signals.
By delivering AAV vectors developed and manufactured at
Spark, Rhonova is designed at once to suppress the expression
of a faulty gene by encoding an RNAi molecule to suppress
translation of mutated (and wild-type) RHO transcripts and to
deliver normal copies of the gene by encoding a wild-type RHO
protein from which the RNAi recognition sequence has been
eliminated.
“The ability to address autosomal-dominant diseases by using
a suppression-and-replacement therapy strategy is something
we want to be able to do for a wide range of diseases,” High
told BioWorld Today. “In some ways, the ability to address
it first in an ocular target where you can be very sure that
you’re delivering the vector to the target cells of interest is an
attractive way to start.”
Rhonova has orphan drug designation both in the U.S. and EU,
in addition to advanced therapy medicinal product designation
from the EMA. RHO-adRP, which affects an estimated 12,000
patients in the U.S. and the five major European markets,
currently has no approved pharmacologic treatment.
‘THEY’RE A MODEL OF CLARITY’
Ena Prosser, a partner in the Dublin office of Genable investor
Fountain Healthcare Partners and member of Genable’s
board, credited Jane Farrar, the company’s co-founder and
chief scientific officer, and her team at Trinity College Dublin
(TCD) for shepherding the asset through more than 30 years
of research, “and it really is fueled by the patients and their
families,” Prosser said. “Our main priority is to allow the
Genable team and the product’s dedicated creators to integrate
Rhonova into Spark’s pipeline and see if this really can cure
[RHO-adRP].”
High also praised Farrar, a member of Spark’s scientific
advisory board, and the Genable scientific team.
“We had been working with Jane and her colleagues at
Genable for a number of years, and they’ve been a great
group of collaborators,” High said, singling out TCD’s Sophia
Millington-Ward and Naomi Chadderton as well as Paul
Kenna, an ophthalmologist and Genable’s co-founder and
chief medical officer. “Together, they’ve really driven he work
forward.”
As a fellow scientist, High placed a high priority not only on
identifying “first-rate collaborators, and Jane Farrar definitely
meets that bar,” but also on liaising with researchers who were
focused on the endgame of treating patients.
“Jane really is therapeutically oriented,” she said. “She is
always thinking about how what she’s doing can be safely and
effectively translated for patients. If you look at their papers,
they’re a model of clarity. No controls are left out. That’s just a
great collaborator for a company like Spark.”
The companies have been working together through a joint
steering committee since initiating their 2014 collaboration,
with face-to-face meetings every three months that rotate
between Philadelphia and Dublin. That process will continue,
High said.
Genable had raised €5 million (US$6.8 million) in November
2011 and aimed to raise another €8 million to enable it to
advance Rhonova into a phase I study that aimed to enroll 30
to 35 patients. (See BioWorld Today, Nov. 14, 2011.)
The timetable for a first-in-human trial still depends on a few
interim steps, High said. Genable published proof-of-concept
data on Rhonova and designed a number of experiments
in a large animal model to optimize the relative ratios of
suppression and replacement. The next step is to execute those
studies with the material supplied by Spark.
Genable also has designed some natural history and run-in
studies, “and we’ll be working together on those, too,” High
said.
At first blush, J.P. Morgan analyst Cory Kasimov liked the deal.
“This is probably best characterized as a modest deal (in
terms of price and stage), but it appears to be a prudent
strategic decision for Spark,” he wrote in an email. “In
addition to full rights to the lead program, this acquisition
also gives Spark access to Genable’s suppression/
augmentation approach with potential applications outside
the primary indication.”
See Spark, page 8

Capella
program through to phase IIa development by the end of
2018, and to complete preclinical development of four other
products.
That follows an initial investment of £1.5 million seed funding
in 2014, with which, rather than shaping up an existing body of
academic research, Capella Chief Operating Officer (COO) Steve
Holmes was charged to go out and prospect for five therapeutic
antibody projects around which to form a company.
The seed money came from the founding investors, Advent
Life Sciences and Medicxi Ventures (then Index Ventures). As
Holmes explained, the idea was to draw on his experience, and
that of the VC backers, to select the best monoclonal antibody
projects.
“The key was the targets; they had to be novel, and to have
good biology to go with them,” Holmes said.
Antibody generation has now advanced to the point where a
number of platforms are available and in that sense products
are commoditized.
“We are agnostic about platforms. Making antibodies is
relatively straightforward; it is the targets that make the
difference,” Holmes told BioWorld Today. “[Products] could be
fully human or mouse, it depends what we want to get out of
the antibody,” he said.
From initial prospecting in 2014, London-based Capella has
advanced to the point of having lead panels of antibodies for
its top three programs and has in-licensed target biology for a
further two programs.
The first program, in inflammatory bowel disease, rests on
research from Sanford Burnham Prebys Medical Discovery
Institute in La Jolla, Calif. Although not revealing any details,
Holmes said that is a known target in which Capella is aiming
at a particular aspect of the biology that has not been exploited
previously,
“It happens to be a difficult concept for developing an antibody,
but we’ve got initial [constructs],” Holmes said.
The second program, in fibrosis, is based on intellectual
property from Leeds University.
If target biology is now the key to antibody drug development,
the central requirement in its selection is expertise in
therapeutic targets.
Holmes has some form here, being former COO of murine
antibody company Kymab Ltd. and of Domantis Ltd., and
heading the monoclonal antibody group at Smithkline
Beecham plc and then Glaxosmithkline plc, during the
pioneering decade from 1990 to 2001.
Capella’s clinical and scientific advisory group include antibody
doyen Don Drakeman, founder of two leading U.S. and
European companies, Medarex Inc. (acquired by Bristol-Myers
Squibb Co. for $2.4 billion in 2009) and Genmab A/S.
Meanwhile, Kevin Johnson, partner at Medicxi and board
member at Capella, was head of research at Cambridge
Antibody Technology plc, a corporate foundation stone of the
monoclonal antibody therapeutics sector.
In addition to reflecting the maturity and quality of monoclonal
antibody platforms, Capella exemplifies the asset-based
investment strategies of the founding VCs.
Rather than making an initial investment in forming a
company, the two invest in taking on half-formed assets and
shaping them up to be suitable for pharma pipelines.
The focus then is on conducting a program of research that
will validate – or not - a particular asset, with the aim of
establishing proof of concept in around three years for an
investment of around $20 million.
“The intention with our five therapeutic monoclonal antibodies,
is quick in, quick out. This is a really good model for a start-up;
we have been completely funded by Advent and Medicxi from
nothing,” Holmes said.
“In fact, we started one program and dropped it after five
months and found a substitute - so we are very lethal. But with
the last five targets everything has worked,” said Holmes.
The intention will be to find commercialization partners at an
early stage, and Holmes said he expects to be ready to start the
hunt for partnerships before the end of the year.
The profile and connections of the investors and the board
means Capella “can go straight to the top” when trying to
attract big pharma, he added.
Joining the founding investors Advent and Medicxi in the
series A is Philadelphia-based Osage University Partners, a VC
specializing in the commercialization of academic research.
The series A investment in Capella is Medicxi’s first since
splitting from Index Ventures and launching a new $250 million
fund dedicated to early stage European research in February.
(See BioWorld Today, Feb 3, 2016.) //
This could be a busy year for Spark. Initial efficacy data for
SPK-FIX in hemophilia B, partnered with New York-based Pfizer
Inc., are due midyear along with initial safety and efficacy data
for SPK-CHM in choroideremia due in the second half of 2016.
With a biologics license application filing for lead therapy SPK-
RPE65 (voretigene neparvovec) in RPE65-mediated inherited
retinal dystrophy following positive phase III data last year and
investigational new drug filings for SPK-FVIII in hemophilia A
and SPK-TPP1 in TPP1 deficiency, a form of Batten disease, also
expected in the second half, “we continue to believe that ONCE
is an underappreciated story in SMID biotech,” Kasimov added.
(See BioWorld Today, Oct. 6, 2015.)
On Monday, Spark’s shares closed at $35.69 for a gain of
$1.16. //
Spark

EMA
gaining and sharing knowledge on the medicine throughout the
development, we will not only accelerate patients’ access, but
also ensure an efficient use of available resources.”
That high level of engagement will underpin the generation
of a robust data package that meets marketing authorization
application requirements and supports a thorough risk/benefit
evaluation of a new medicine, the EMA said.
It is expected that if products granted Prime status reach the
point of being submitted for approval, they will then qualify
for the EMA’s accelerated assessment pathway. Currently,
accelerated assessment status is not conferred until just before
an application is submitted.
The EMA was keen to draw the distinctions between Prime and
its Adaptive Pathways pilot, under which the agency is trying to
factor real-world evidence into regulatory development.
While Prime aims to provide early and enhanced scientific and
regulatory support to promising new medicines that fulfill the
criteria for accelerated assessment, Adaptive Pathways is for
treatments in areas of high medical need, where collection of
data via traditional routes is difficult and large clinical trials
would unnecessarily expose patients who are unlikely to benefit
from the medicine.
Instead, products in the Adaptive Pathways pilot will be
developed in a restricted patient population as an initial step,
following by the gathering of real-life data, with a view to
expanding the patient population in which the medicine can be
used. // OTHER NEWS TO NOTE
Geovax Labs. Inc., of Atlanta, said the simian analogue of its
HIV vaccine, GOVX-B11, provided a 76 percent reduction in per-
exposure risk of simian immunodeficiency virus (SIV) infection
in young adult rhesus macaques. Per-exposure risk was
assessed over 12 weekly rectal exposures. The study, supported
by the National Institute of Allergy and Infectious Diseases,
also showed the vaccine provided much better protection for
young adult than middle-aged and older macaques. The test
vaccine used a DNA vaccine expressing non-infectious virus-like
particles (VLPs) to prime the immune response and a modified
vaccinia Ankara (MVA) vaccine, also expressing non-infectious
VLPs, to boost the primed response. The vaccine regimen
included two DNA primes at weeks zero and eight followed by
two MVA boosts at weeks 16 and 32. The efficacy of the vaccine
was tested at six months after the final MVA boost by 12 weekly
rectal exposures to a pathogenic SIV given at a dose that
infected about 30 percent of the unvaccinated animals at each
challenge. Animals that were less than 10 years old (estimated
human age of about 40) had much better protection than
those over 10 years old. The age-dependence for protection
was stronger than typically seen for vaccines and likely reflects
the HIV target for protective antibody being particularly well-
camouflaged.
IN THE CLINIC
3SBio Inc., of Shenyang, China, said the CFDA approved its
investigational new drug application for peg-irinotecan, a long-
acting polymer-drug conjugate that inhibits topoisomerase
I. Peg-irinotecan was developed by Jenkem Technology Co.
Ltd., of Beijing, which inked an exclusive license agreement
with 3SBio in 2014 to develop, manufacture and market the
candidate in mainland China. 3SBio plans to develop peg-
irinotecan as a National Class I drug for relapsed or refractory
cancers, including colorectal, metastatic breast and platinum-
resistant ovarian cancer. (See BioWorld Today, Sept. 19, 2014.)
Acelrx Pharmaceuticals Inc., of Redwood City, Calif., said it
initiated patient enrollment and dosing in an open-label phase
III study (SAP303) of ARX-04 (sufentanil sublingual tablet, 30
mcg) in patients 40 years and older with moderate to severe
acute pain following a surgical procedure. Patients will be
administered one dose of ARX-04 every 60 minutes, as needed
for pain management, for up to 12 hours. The study is expected
to enroll up to about 100 patients who underwent a surgical
procedure with general or spinal anesthesia. The primary
efficacy endpoint is the summed pain intensity difference over
the 12-hour study period. Safety endpoints include adverse
events and vital signs. The study is expected to be completed
by the third quarter. (See BioWorld Today, Nov. 2, 2015.)
Advaxis Inc., of Princeton, N.J., said it dosed the first patient
in the expansion phase of an ongoing phase II trial of its lead
Listeria monocytogenes (Lm) immunotherapy candidate,
axalimogene filolisbac (AXAL), in patients with persistent or
recurrent metastatic (squamous or nonsquamous cell) cervical
cancer. The open-label, nonrandomized, dose-escalation study
is designed to evaluate the safety, efficacy and immunological
effect of AXAL at doses of up to 1 x 1010 colony forming units in
repeat treatment cycles in about 25 women with cervical cancer
whose disease recurred after one prior cytotoxic treatment
regimen. The high-dose expansion phase will include 15
patients. (See BioWorld Today, Dec. 17, 2015.)
Aimmune Therapeutics Inc., of Brisbane, Calif., reported data
from its phase II study of AR101 for the treatment of peanut
allergy, showing that all patients who completed 12 weeks of
low-dose maintenance therapy were desensitized to levels of
peanut protein beyond the 250 mg to 300 mg typically found
in one peanut kernel. Data were presented at the American
Academy of Allergy, Asthma and Immunology meeting in Los
Angeles. The firm is now enrolling patients in the phase III
PALISADE trial testing the drug, a formulation of peanut flour
and pharmaceutical ingredients, in peanut allergy.
Antibe Therapeutics Inc., of Toronto, said it received approval
from Health Canada to start a phase II trial testing lead drug
ATB-346, a hydrogen sulfide-releasing derivative of naproxen,
in osteoarthritis of the knee. The study is set to start this month.

IN THE CLINIC
Beigene Ltd., of Beijing, said the CFDA cleared the firm to
start clinical testing with BGB-3111, a selective small-molecule
Bruton’s tyrosine kinase inhibitor, for the treatment of B-cell
malignancies. Trials already are ongoing in Australia. (See
BioWorld Today, Dec. 10, 2015.)
Brickell Biotech Inc., of Miami, reported phase IIb data at the
American Academy of Dermatology meeting in Washington,
showing that BBI-4000 (sofpironium bromide) for the topical
treatment of primary axillary hyperhidrosis, or excessive
underarm sweating, met its primary endpoint in the intent-
to-treat analysis by achieving a statistically significant two-
grade improvement in the Hyperhidrosis Disease Severity
Score (HDSS), in a dose-related fashion. At the maximum
dose (15 percent), 38.3 percent of participants receiving the
anticholinergic improved more than two points on HDSS at day
29 vs. 12.2 percent with vehicle (p<0.01).
Celgene Corp., of Summit, N.J., said long-term safety findings
from ongoing studies of Otezla (apremilast), presented at the
American Academy of Dermatology meeting in Washington,
showed no increase in exposure-adjusted incidence rates of
adverse events, serious adverse events or drug discontinuations
for the 182-week data compared with the first 52 weeks of
treatment. The rates of major cardiac events, malignancies,
depression and attempted suicide, respectively, were
comparable across the Otezla-exposure periods. No serious
opportunistic infections, reactivation of tuberculosis infection,
completed suicide or clinically meaningful effects on laboratory
measurements were reported. Weight loss above 5 percent of
body weight was experienced by 21.9 percent of participants,
compared with 18.8 percent during the first 52 weeks. Data
were from pooled analyses from the ESTEEM 1 and 2 trials and
the PALACE 1-3 trials testing the phosphodiesterase 4 inhibitor
in patients with plaque psoriasis and psoriatic arthritis.
Cempra Inc., of Chapel Hill, said it received authorization under
its existing contract with the Biomedical Advanced Research
and Development Authority to receive funding of $25 million
through mid-2018 for a phase II/III trial of solithromycin. Cempra
is responsible for an additional designated portion of the cost
of the planned study, which will enroll pediatric patients, ages
2 months to 17 years, with community-acquired bacterial
pneumonia to receive either intravenous, oral suspension or
oral capsule formulations of solithromycin, a fourth-generation
macrolide antibiotic, or a comparator treatment. The study, set
to start in the first half of this year, will enroll about 400 patients,
with full enrollment expected during the first half of 2018.
Chugai Pharmaceutical Co. Ltd., of Tokyo, reported results
from a phase II study of anti-IL-31 receptor A, humanized
monoclonal antibody nemolizumab (CIM331), currently in
development for atopic dermatitis, at the American Academy of
Dermatology meeting in Washington. Data showed the percent
change in pruritus visual analogue scale at week 12, which was
the primary endpoint, was significantly higher in the CIM331
treatment groups than in the placebo group (p<0.01 in all). The
study enrolled 264 patients randomized to one of four CIM331
groups or placebo.
Corbus Pharmaceuticals Holdings Inc., of Norwood, Mass.,
said Resunab, its oral endocannabinoid-mimetic, will be
tested in a phase II trial in systemic lupus erythematosus
(SLE) that was selected for funding by the NIH Autoimmunity
Centers of Excellence program. The study will test the efficacy,
safety, tolerability and biologic effects of Resunab as a non-
immunosuppressive oral treatment to improve signs and
symptoms of SLE. About 100 adult SLE patients with active
musculoskeletal disease will be randomized to receive either
placebo or three different doses of Resunab daily for 84 days,
with 28 days follow-up.
Derma Sciences Inc., of Princeton, N.J., said results from a
peer-reviewed comparative study of Amnioexcel Amniotic
Allograft Membrane plus standard of care vs. standard of
care alone for the closure of hard-to-treat diabetic foot ulcers,
published in Wounds, were both statistically significant and
clinically meaningful, with the closure rate at six weeks in
the Amnioexcel arm far exceeding the standard of care alone
treatment arm.
Eli Lilly and Co., of Indianapolis, said treatment for moderate to
severe plaque psoriasis with ixekizumab, its IgG4 monoclonal
antibody, resulted in clinically meaningful improvements as
early as one week, compared to patients treated with Enbrel
(etanercept, Amgen Inc.) or placebo, according to detailed results
of the combined analysis of UNCOVER-2 and UNCOVER-3 phase
III studies presented at the American Academy of Dermatology
meeting in Washington. Data showed significant differences
in mean percentage improvement of psoriasis plaques among
patients treated with ixekizumab compared to etanercept and
placebo, including one-week data showing a mean percentage
improvement of 32.7 percent in the ixekizumab group, 10.3
percent in the etanercept group and 5.31 percent in the placebo
group (p < 0.001 for all comparisons.) At two weeks, the mean
percentage improvement was 53.6 percent for ixekizumab, 23.3
percent for etanercept and 9.25 percent for placebo (p<0.001 for
all comparisons).
Green Cross Biotherapeutics Corp., of Montreal, a member
of the Green Cross family, said Health Canada approved its
application for a phase III trial of immune globulin intravenous
(human) 10 percent GC5107, intended for the treatment of
primary humoral immunodeficiency. An investigational new
drug application to the FDA is expected to follow.
Index Pharmaceuticals AB, of Stockholm, said the FDA
cleared the firm to start a phase IIb study of lead candidate
cobitolimod (Kappaproct), a Toll-like receptor 9 agonist, in
patients with moderate to severe ulcerative colitis. The trial
will evaluate the safety and efficacy of cobitolimod in inducing
clinical remission in patients with chronic active moderate
to severe ulcerative colitis as compared to placebo and will
evaluate higher doses and more frequent dosing than those
used in previous studies.

OTHER NEWS TO NOTE
Leo Pharma A/S, of Ballerup, Denmark, said data presented at
the American Academy of Dermatology meeting in Washington
demonstrated that calcipotriol/betamethasone dipropionate
50 mcg/0.5 mg/g) foam formulation is a stable supersaturated
solution of the active ingredients, compared to calcipotriol/
betamethasone dipropionate 50 mcg/g/0.5 mg/g ointment in
which the active ingredients are suspended. The data presented
showed that no calcipotriol or betamethasone dipropionate
crystals were identified in the foam spray following application
and for up to 18 hours. However, crystals were present
immediately upon application of the ointment formulation.
Those data may provide a potential explanation for the
improved clinical efficacy observed in clinical trials.
IN THE CLINIC
International Stem Cell Corp., of Carlsbad, Calif., said it is
enrolling patients in the phase I trial if ISC-hpNSC, human
parthenogenetic stem cell-derived neural stem cells, for the
treatment of moderate to severe Parkinson’s disease. The
12-patient, dose-escalation and preliminary efficacy study will
test ISC-hpNSC, intracranially transplanted into patients at
three different dose regimens of 30 million to 70 million neural
cells. Following transplantation, the patients will be monitored
for 12 months at specified intervals, to evaluate safety and
biologic activity. PET scan will be performed at baseline, as
part of the screening assessment, and at six and 12 months
after surgical intervention, and clinical responses compared
to baseline after the administration of ISC-hpNSC will be
evaluated using various neurological assessments such as
Unified Parkinson Disease Rating Scale, Hoehn and Yahr and
other rating scales.
Ix Biopharma Ltd., of Singapore, said its multidose, placebo-
controlled study, called KET0090, testing Wafermine, an
oral, sublingual analgesic employing ketamine as its active
compound, confirmed that Wafermine was well tolerated
with no serious adverse effects at the maximum accumulated
dosage of 315 mg over a 14-hour period. The most frequent
adverse events reported were nausea, vomiting and dizziness,
which are common in postoperative populations. It was noted
that the same adverse events occurred with similar frequency
in all treatment groups. The trial’s objective was to test
Wafermine, administered alone or in combination with opioids,
in subjects undergoing bunionectomy.
Kempharm Inc., of Coralville, Iowa, said it filed an
investigational new drug application with the FDA to start
studies of KP511, its prodrug of hydromorphone that is intended
for development as abuse-deterrent ER medication for
treating moderate to severe pain. Kempharm plans to conduct
a clinical program for KP511 that will assess the product’s
tamper and extraction resistance, intranasal and intravenous
abuse potential, as well as the potential to limit oral abuse
and/or overdose. Additionally, the firm intends to investigate
KP511’s potential for improving or reducing opioid-induced
constipation.
Medicinova Inc., of La Jolla, Calif., presented phase II results
at the Behavior, Biology and Chemistry: Translational Research
in Addiction meeting in San Antonio from a study of MN-
166 (ibudilast) in opioid dependence, showing that ibudilast
significant decreased the reinforcing effects of oxycodone (p <
0.05); significantly decreased the craving for heroin (p < 0.01),
cocaine (p < 0.01) and tobacco (p < 0.05); and significantly
decreased participants’ rating for “liking” oxycodone (p < 0.05).
Ibudilast, an oral, small-molecule phosphodiesterase 4 and 10
inhibitor and macrophage migration inhibitory factor inhibitor,
also was safe and well tolerated.
Novartis AG, of Basel, Switzerland, said data from the head-
to-head CLEAR study, presented at the American Academy of
Dermatology meeting in Washington, demonstrated that its
fully human interleukin 17A inhibitor, Cosentyx (secukinumab),
remains superior to Stelara (ustekinumab, Johnson & Johnson)
in achieving sustained skin clearance (PASI 90 response) at 52
weeks for adults living with moderate to severe psoriasis. At
weeks four, 16 and 52, Cosentyx achieved and sustained PASI
90 responses vs. Stelara (76.2 percent vs. 60.6 percent, p <
0.0001) and achieved greater PASI 100 responses (45.9 percent
vs. 35.8 percent, p = 0.0103) at 52 weeks. The study also
demonstrated Cosentyx had a superior rapid onset of action
compared to Stelara, with half of Cosentyx patients achieving
PASI 75 as early as week four (50 percent vs. 20.6 percent, p <
0.0001).
Nuvo Pharmaceuticals Inc., of Mississauga, Ontario, reported
top-line results from its randomized, placebo-controlled,
double-blind, parallel-group study in patients with grade I or II
ankle sprains, showing that, on average, patients treated with
Pennsaid 2 percent had a larger reduction in pain on movement
scores over the course of the study, though the full analysis
set missed the primary endpoint, as measured on day five
(p=0.2430), while showing a statistically significant difference
on the secondary time point on day three (p=0.0119). In the
per-protocol group, which excluded those patients with a lower
usage of medication, the Pennsaid 2 percent group showed a
statistically significant improvement at both the primary time
point (day five; p=0.0416), as well as the secondary time points
(day three; p=0.0018; and day eight; p=0.0490).
Prexton Therapeutics SA, of Geneva, said it launched a phase
I trial testing its mGluR4 positive allosteric modulator in 72
healthy volunteers. Results are expected in August. Prexton
previously reported preclinical data showing the molecule’s
potential in alleviating motor complications by modulating
glutamate over activity in the central nervous system of
Parkinson’s disease patients. The phase I study will be financed
by a $10 million series A round that closed in February 2015.
(See BioWorld Today, Feb. 25, 2015.)

OTHER NEWS TO NOTE
Matinas BioPharma Holdings Inc., of Bedminster, N.J., said
its investigational drug, MAT2501 (encochleated amikacin),
was granted orphan designation by the FDA for the treatment
of nontuberculous mycobacteria (NTM) infections, its
lead chronic indication. MAT2501, an orally administered,
encochleated formulation of the broad-spectrum intravenous-
only aminoglycoside antibiotic agent amikacin, utilizes the
company’s lipid-crystal, nanoparticle delivery technology. The
company recently received FDA clearance to initiate a phase I
study of MAT2501 for the treatment of NTM infections.
Merck & Co. Inc., of Kenilworth, N.J., said the FDA accepted
for review a new supplemental biologics license application for
Keytruda (pembrolizumab), the company’s anti-PD-1 therapy,
to include data from Keynote-010. The trial was a pivotal
phase II/III study designed to evaluate Keytruda compared to
chemotherapy based on prospective measurement of PD-L1
expression in previously treated patients with advanced non-
small-cell lung cancer. (NSCLC) The drug is currently indicated
in the U.S. for the treatment of patients with metastatic
NSCLC, whose tumors express PD-L1 as determined by an
FDA-approved test, with disease progression on or after
platinum-containing chemotherapy. Patients with EGFR or ALK
genomic tumor aberrations should have disease progression on
FDA-approved therapy for those aberrations prior to receiving
Keytruda. The NSCLC indication, approved under accelerated
approval, was based on tumor response rate and durability of
response in patients with PD-L1 expression on 50 percent or
more of the cancer cells. In accordance with the accelerated
approval process, the data from Keynote-010 was intended
to serve as the confirmatory trial for receiving full approval,
establishing the clinical benefit by demonstrating improved
survival over standard chemotherapy.
Molecular Partners AG, of Zurich-Schlieren, Switzerland,
disclosed details about its immuno-oncology pipeline,
including two early stage multi-Darpin programs. The first
program targets the validated immune checkpoint PD-1 as
well as VEGF-A, aiming to enhance PD-1 efficacy. The second
program is designed to potently activate T cells in the tumor
without activating circulating T cells, thus circumventing
systemic toxicities. Darpins are described as potent, specific
and versatile small-protein therapies that could offer benefits
over conventional monoclonal antibodies or other currently
available protein therapeutics.
Opiant Pharmaceuticals Inc., of Santa Monica, Calif., received
a $2.5 million milestone payment from Adapt Pharma Ltd., of
Dublin, following its first commercial sale of Narcan (naloxone
hydrochloride) for the emergency treatment of known or
suspected opioid overdose.
Oxford Biomedica plc, of Oxford, U.K., disclosed a new and
expanded collaboration with Immune Design Corp., of Seattle,
as well as a nonexclusive, royalty-bearing, intellectual property
license to Immune Design. The license involves the use of
lentiviral vector-based products for the in vivo treatment or
prevention of cancer. The collaboration builds on Immune
Design’s earlier process development collaboration with
Oxford, signed in 2012, which covered the development of
analytic assays. Financial terms were not disclosed.
Peptidream Inc., of Tokyo, said it entered a technology license
agreement with Indianapolis-based Eli Lilly and Co. for
nonexclusive access to Peptidream’s peptide discovery platform
system. Under the terms, Peptidream will receive up-front and
annual technology access payments, and will be eligible to
receive development milestones and royalties on future sales of
products (both peptide and small-molecule therapeutics) that
arise from use of the technology. Similar to previous technology
licenses, this one will exclude work on peptide-drug conjugate
programs, as Peptidream has internal development efforts
ongoing there. Specific financial terms were not disclosed.
Pharmaengine Inc., of Taipei, Taiwan, said the Taiwan FDA
approved the product license of Onivyde (irinotecan liposome
injection, nal-IRI). Onivyde is indicated, in combination with
fluorouracil and leucovorin, for the treatment of patients with
metastatic adenocarcinoma of the pancreas after disease
progression following gemcitabine-based therapy. The
company expects that the product will be available during the
second quarter of this year.
Specialized Therapeutics Pty Ltd., of Melbourne, Australia,
and Mundipharma Pty Ltd., of Cambridge, U.K., disclosed a
sublicense agreement enabling Mundipharma to assume all
commercialization responsibilities for the anti-emetic products
Aloxi (palonosetron hydrochloride) and Akynzeo (netupitant/
palonosetron) in Australia and New Zealand. Financial terms
were not disclosed.
VBI Vaccines Inc., of Cambridge, Mass., reported that
manufacturing characterization data confirmed the purity and
quality of its therapeutic vaccine candidate against glioblastoma
multiforme (GBM). The company said the purity measurements
are expected to meet regulatory requirements for clinical
evaluation, and VBI plans to hold a pre-investigational new drug
meeting with the FDA in the first half of the year. The data were
presented in a poster at the Keystone Symposia Cancer Vaccines
Conference in Whistler, British Columbia. Ex vivo studies have
shown the vaccine candidate’s ability to induce desired antitumor
immunity in peripheral blood mononuclear cells obtained
from healthy subjects and patients with GBM. The candidate
stimulated strong T-cell immunity, including both CD4+ and
CD8+ T-cell responses, as well as secretion of IFN-γ and CCL3,
biomarkers associated with positive clinical outcomes.
Vericel Corp. (formerly Aastrom Biosciences Inc.), of
Cambridge, Mass., said the FDA accepted its biologics license
application for MACI (matrix applied characterized autologous
cultured chondrocytes), its autologous cellular candidate to
treat symptomatic cartilage defects of the knee in adults.
Vericel said the FDA set a PDUFA date of Jan. 3, 2017, and said
it does not plan to hold an advisory committee meeting to
discuss the application.

IN THE CLINIC
Revance Therapeutics Inc., of Newark, Calif., reported results
from its phase II BELMONT trial testing DaxibotulinumtoxinA
for injection (RT002) at the American Academy of Dermatology
meeting in Washington, showing that all three dose levels
of RT002 achieved the primary endpoint by demonstrating
at least a one-point improvement in frown lines based on
the Investigator Global Assessment-Facial Wrinkle Severity
(IGA-FWS) scale at four weeks. That measurement showed
a statistically significantly greater response as compared to
placebo for each dose level. The study demonstrated a six-
month median duration of effect for RT002 based upon at least
a one-point improvement in glabellar lines at maximum frown
on the IGA-FWS scale.
Samumed LLC, of San Diego, presented results from its
phase II androgenetic alopecia trial at the American Academy
of Dermatology meeting in Washington, showing that
topically applied small molecule SM04554 appeared to be
safe and well tolerated, with no adverse events observed in
the treatment groups. Over the 135-day study, both treatment
arms showed an increase in hair count and hair density,
objectively measured by macrophotography. By contrast,
the vehicle group showed a decline in both measures. Under
the a priori statistical analysis plan, between day 90 and
day 135, the 0.15 percent treatment group had a statistically
significantly higher increase in both hair count and hair
density, as compared to vehicle.
Spectrum Pharmaceuticals Inc., of Henderson, Nev., said it
started the planned phase II trial of poziotinib, its pan-HER2
inhibitor. The study is expected to enroll about 70 patients with
HER2-positive metastatic breast cancer who have failed at
least two prior HER2-directed therapies.
Summit Therapeutics plc, of Oxford, U.K., reported data
from the phase II CoDIFy trial showing that narrow-spectrum
antibiotic ridinilazole preserves the gut microbiome in
patients with Clostridium difficile infection (CDI). Ridinilazole-
treated patients in CoDIFy exhibited no further damage
to their microbiome during therapy, with a proportion of
patients showing initial evidence of recovery of key bacterial
groups with roles in protecting from CDI. In stark contrast,
vancomycin-treated patients in the study suffered substantial
damage to their gut microbiome during treatment and that
persisted in many patients during the 30-day post-treatment
period.
Tigenix NV, of Leuven, Belgium, reported that a single
injection of Cx601, its allogeneic expanded stem cell product,
was statistically superior to placebo in achieving combined
remission at week 52 in the phase III ADMIRE-CD trial in
Crohn’s disease patients with complex perianal fistulas with
inadequate response to previous therapies, including anti-
TNFs. Data showed that 54.2 percent of patients treated
with Cx601 achieved combined remission vs. 37.1 percent in
the placebo arm, while 75 percent of Cx601-treated patients
who achieved combined remission at week 24 remained in
combined remission at week 52, compared to 55.9 percent in
the placebo arm. The one-year data also confirm the favorable
safety and tolerability profile of Cx601 reported at week 24.
Vical Inc., of San Diego, said it started a phase I randomized,
double-blind trial testing the safety, tolerability and
pharmacokinetics of antifungal candidate VL-2397 at single
and multiple ascending doses in healthy volunteers. VL-2397 is
in development for treating invasive aspergillosis, for which it
has orphan drug and qualified infectious disease designations
from the FDA.
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FINANCINGS
Argos Therapeutics Inc., of Durham, N.C., said it is selling up
to $60 million of its common stock and warrants in a private
placement financing. Under a securities purchase agreement
the financing will take place in up to three tranches; at the
initial closing, the investors will purchase a total of 3.65
million shares of common stock and warrants to purchase
a total of about 2.74 million shares of common stock for a
total purchase price of $19.88 million. At the second closing,
investors will purchase a total of 5.47 million shares of common
stock and warrants to purchase a total of 4.1 million shares
of common stock at the same price and on the same terms
as the first tranche for an additional purchase price of $29.8
million. The second closing is conditioned on the independent
data monitoring committee (IDMC) for the ADAPT study at,
or following the IDMC’s next regular meeting following the
initial closing, recommending that Argos continue the study
or discontinue it based on favorable efficacy data. Participants
in the financing include Pharmstandard International SA,
Forargos BV, Tianyi Lummy International Holdings Group Ltd.,
China BioPharma Capital I LP, TVM V Life Science Ventures
GmbH & Co. KG and Wasatch Funds Trust. Pharmstandard has
also agreed that, at Argos’s option and the status of the ADAPT
trial and the company’s cash position at such time, it shall
purchase at the third closing up to $10.29 million of shares of
common stock (without warrants) at a price per share to be
determined pursuant to an agreed upon formulation. Argos
expects that proceeds from the initial closing will enable it to
fund the company’s ongoing expenses into the third quarter
of 2016, and that the proceeds from all three closings, if such
closings occur, will enable it to fund the company’s ongoing
expenses into the second quarter of 2017 when it expects final
data from its pivotal phase III ADAPT trial of AGS-003 for the
treatment of metastatic renal cell carcinoma.
Hutchison China Meditech, of Hong Kong, set terms for a
proposed U.S. IPO, aiming to raise $100 million by offering 6.1
million American depositary shares priced at $16.33 apiece. The
company plans to list on Nasdaq under the ticker HCM. (See
BioWorld Today, Feb. 17, 2016.)
ITM Isotopen Technologien Muenchen AG, of Garching,
Germany, said it raised €20 million (US$22 million) in equity
from Medtech Focused Family Office. ITM plans to use the
funds to further develop its pipeline of targeted radionuclide
therapies for therapeutic and diagnostic purposes.
Plx Pharma Inc., of Houston, withdrew its proposed IPO,
previously filed to raise up to $68 million. Founded in 2002,
Plx has a delayed-release aspirin based on its lipid coating
technology.
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