KAPIDEX - Gene€¦ · new business opportunities for licensing or partnerships and analyze...
Transcript of KAPIDEX - Gene€¦ · new business opportunities for licensing or partnerships and analyze...
Launches
GRAZAX
ALK-Abello UK launch for use in
children and adolescents
On 23 February 2009 ALK-Abello
announced that GRAZAX has been
launched in the UK for use in children
and adolescents (aged five to 17 years)
with severe grass pollen-induced
seasonal a l lerg ic rhin it is and
conjunctivitis or severe hay fever.
GRAZAX, a once-daily tablet-based
immunotherapy, was approved in the
EU in September 2006 for the
treatment of adults with grass pollen
allergy, and has subsequently been
launched for this indication in
Germany, the UK, Denmark, Norway,
Sweden and Ireland.
quetiapine
AstraZeneca launches
extended-release formulation for
additional indications
AstraZeneca announced on 23 February
2009 that it has launched once-daily
quetiapine extended-release tablets
(SEROQUEL XR) in the USA for the acute
treatment of depressive episodes
associated with bipolar disorder, manic
and mixed episodes associated with
bipolar I disorder, and as maintenance
therapy for bipolar I disorder as an
adjunct to lithium or divalproex. The
KAPIDEXTakeda marketed, USA (GERD)
On 23 February 2009 Takeda reported that it has launched KAPIDEX (30
and 60 mg capsules) in the USA for the treatment of heartburn
associated with symptomatic non-erosive gastroesophageal reflux
disease (GERD), the healing of erosive esophagitis (EE) and the
maintenance of healed EE. KAPIDEX is a modified-release formulation
of the proton pump inhibitor dexlansoprazole, an enantiomer of
lansoprazole.
APPROVALS
EFIENT & FIRMAGON Approved
in the EU
REMOVAB & CONBRIZA
Recommended for Approval in
the EU
ONRIGIN Submitted for
Approval in the USA
LICENSING
Agreements between
NeuroSearch & Lilly, Chugai &
Romark, Neopharm Ltd &
Orexo, Bial & Eisai, Abbott &
Sucampo, Access & Dong-A,
Actelion & GeneraMedix, CSL &
Xencor
TECHNOLOGY TRANSFERSPOTLIGHT
German Cancer Research Center
PIPELINE NEWS
CNSBio
Shire
PRODUCTS &BIOTECHNOLOGY
Albiglutide Enters PIII
AV 951, EC 145, TB 402 & SGN
35 Enter PII
NKTR 105, DM 1992, ABT 450
and GTx 758 Enter PI
Resatorvid Discontinued
COMPANY FOCUS
Gene Signal International
TABLES
Newly reported drugs
Product phase changes
Volume 18 No 9
2 March 2009
IMS LifeCycle R&D Focus
Essential up-to-date intelligence on drugs in R&D development worldwide
A powerful tool to evaluate the progress of R&D pipelines from discovery phase
to marketed Phase, review registered and recently marketed products, identify
new business opportunities for licensing or partnerships and analyze
acquisitions and investment deals potential
Key Features
• Over 26,000 drugs in R&D with over 9,700 drugs in active development
• Over 5,600 biotechnology products
• Latest phase reached worldwide, with country/region development status in relation
to a particular indication
• Over 3,000 companies, featuring companies’ involvement in the development of a
drug and their relationships as licensor, licensees or developers
• Sales profiles of 123 leading therapeutic classes using IMS sales data, giving some
financial background of the disease area
• Full commercial summaries, providing a commercial overview, regulatory progress,
R&D progress, licensing and partnering deals, analysts sales predictions
• Scientific summaries, detailing preclinical and clinical data
• Licensing offers/partnerships by region/country, with full contact details
Functionality
• Company contact directory
• Substructure searching
• Create user-defined tabular reports
• Save search strategy and results of searches
• Export tabular reports and records into other Windows applications
Updates and availability
• Over 1500 drug updates each month
• Around 100 new drugs added each month
• Available on CD-ROM with monthly or quarterly updates, via www.imsportal.com, via
IMS Knowledge Link with weekly updates
IMS LifeCycle - R&D Focus Drug News
2008 Prices Valid for a 12-month subscription
1-5 users
licence
(access
within
same site)
Additional
5 users
licence
(when 1-5
users licence
already in
place -
access with
same site of
2nd site)
1-20 users
licence
(access in up
to 3
countries)
Country
licence
(unlimited
access in
one
country)
Region
licence
(unlimited
access in
one
Region)
Global
licence
(unlimited
access
Worldwide)
IMS R&D Focus
Drug News
(Weekly PDF)
£500 £140 £780 £1,055 £1,185 £1,435
IMS R&D Focus
(Weekly Web) &
Drug News
(Weekly PDF)
£5,870 £2,770 £10,340 £13,000 £15,515 £36,540
Subscriptions run from 1st April 2008 - 31 March 2009, but can be started at any point
during the year
R&D FOCUS DRUG NEWS
IMS HEALTH
7 Harewood Avenue, London NW1 6JB, UK
Telephone +44 (0)20 3075 5000
Facsimile +44 (0)20 3075 5900
Email: [email protected]
Web: http://www.imshealth.com
Executive EditorCarolyn Hughes
Deputy Executive EditorFrançois Pagnini
Senior EditorHarin Mahadeva
EditorsAmy Austin
Stephanie Cohen
James Harris
David Thomas
David Vincent
Contributing EditorMaleni Chandra
Patent EditorAntonia Lestner
Production AnalystSophie Avery
Marketing ManagerMike Raymond
Advertising ManagerPaul Jenner
Email: [email protected]
Published by IMSworld Publications Ltd.
R&D FOCUS ISSN 1350-1135
Copy right ã 2009. IMS Health In cor po rated or its af fil i ates. All rights
re served
The In for ma tion Ser vice con tained herein is con fi den tial and pro videdsub ject to the IMS Health In for ma tion Ser vices Stan dard Terms andCon di tions. This In for ma tion Ser vice is pro vided to the Cli ent on aper sonal ba sis un der a non-exclusive and non-transferable licence for theCli ent’s own di rect ben e fit and use only, and may not be cop ied ordi vulged to any other party. Whilst ev ery pos si ble care has been taken inthe prep a ra tion of this in for ma tion, the pub lish ers do not holdthem selves re spon si ble for any ex pres sions of opin ion or er ror oromis sion, or any ac tion re sult ing there from.
Subscriber Service
For any subscription information or queries, please contact the relevantnumbers below:
Tel: +44 20 3075 5888
Fax: +44 20 3075 5346
E-mail: [email protected]
Address: IMS HEALTH, 7 Harewood Avenue, London NW1 6JB, UK
Subscriber Rates: £500.00 annually (1-5 users)
For other subscription rates, please contact us or see table opposite.
2
CON TENTSLAUNCHES 1
GRAZAX · · · · · · · · · · · · · · · · · · · · · · · · · · 1quetiapine · · · · · · · · · · · · · · · · · · · · · · · · · 1
AP PROV ALS 4
prasugrel · · · · · · · · · · · · · · · · · · · · · · · · · 4degarelix · · · · · · · · · · · · · · · · · · · · · · · · · 4catumaxomab· · · · · · · · · · · · · · · · · · · · · · · · 4bazedoxifene · · · · · · · · · · · · · · · · · · · · · · · · 4laromustine · · · · · · · · · · · · · · · · · · · · · · · · 4CIP-Tramadol ER · · · · · · · · · · · · · · · · · · · · · · · 5vorinostat · · · · · · · · · · · · · · · · · · · · · · · · · 5drug delivery system, inhaled aztreonam, Gilead Sciences · · · · · · · 5valsartan + amlodipine + hydrochlorothiazide · · · · · · · · · · · · 5efalizumab · · · · · · · · · · · · · · · · · · · · · · · · · 5drug delivery system, topical diclofenac, Nuvo· · · · · · · · · · · · 5BIFERONEX · · · · · · · · · · · · · · · · · · · · · · · · · 6
LI CENS ING 6
ion channel modulators, NeuroSearch/Lilly · · · · · · · · · · · · · 6nitazoxanide · · · · · · · · · · · · · · · · · · · · · · · · 6ABSTRAL · · · · · · · · · · · · · · · · · · · · · · · · · 7eslicarbazepine acetate · · · · · · · · · · · · · · · · · · · · 7lubiprostone · · · · · · · · · · · · · · · · · · · · · · · · 7ProLindac · · · · · · · · · · · · · · · · · · · · · · · · · 7cancer therapy, Stockbridge Pharmaceuticals · · · · · · · · · · · · 8caerulomycin A · · · · · · · · · · · · · · · · · · · · · · · 8drug delivery system, thermostable epoprostenol, Actelion · · · · · · · 8XmAb · · · · · · · · · · · · · · · · · · · · · · · · · · · 8cannabinoid-2 receptor agonists, Reperio · · · · · · · · · · · · · 8drug discovery, HUYA/Beijing Institute of Materia Medica · · · · · · · 9ibritumomab tiuxetan · · · · · · · · · · · · · · · · · · · · · 9MEDUSA · · · · · · · · · · · · · · · · · · · · · · · · · · 9
TECH NOL OGY TRANS FER SPOT LIGHT 9
fusion polypeptides, German Cancer Research Center · · · · · · · · · 9parvovirus NS1 variants, German Cancer Research Center · · · · · · · 10parvovirus H-1 protein, German Cancer Research Center · · · · · · · · 10NS1 protein/VP1 capsid protein, German Cancer Research Center · · · · 10
PIPE LINE NEWS 11
CNSB001 · · · · · · · · · · · · · · · · · · · · · · · · · 11CNSB004 · · · · · · · · · · · · · · · · · · · · · · · · · 11CNSB006 · · · · · · · · · · · · · · · · · · · · · · · · · 11lanthanum carbonate · · · · · · · · · · · · · · · · · · · · 13HGT 2610 · · · · · · · · · · · · · · · · · · · · · · · · · 13SPD 503 · · · · · · · · · · · · · · · · · · · · · · · · · 13icatibant · · · · · · · · · · · · · · · · · · · · · · · · · 13
PROD UCTS & BIO TECH NOL OGY 13
resatorvid· · · · · · · · · · · · · · · · · · · · · · · · · 13NS 2359 · · · · · · · · · · · · · · · · · · · · · · · · · 13albiglutide · · · · · · · · · · · · · · · · · · · · · · · · 14aliskiren + amlodipine + hydrochlorothiazide · · · · · · · · · · · · 14vaccine, Clostridium difficile, Sanofi Pasteur · · · · · · · · · · · · 14nicotinic acid receptor agonist, Merck & Co/Arena · · · · · · · · · · 14AV 951· · · · · · · · · · · · · · · · · · · · · · · · · · 14EC 145 · · · · · · · · · · · · · · · · · · · · · · · · · · 15vaccine, gene-based, HIV infection, GeoVax · · · · · · · · · · · · 15TB 402 · · · · · · · · · · · · · · · · · · · · · · · · · · 15SGN 35· · · · · · · · · · · · · · · · · · · · · · · · · · 15NKTR 105 · · · · · · · · · · · · · · · · · · · · · · · · · 16DM 1992 · · · · · · · · · · · · · · · · · · · · · · · · · 16ABT 450 · · · · · · · · · · · · · · · · · · · · · · · · · 16LX 214 · · · · · · · · · · · · · · · · · · · · · · · · · · 16CB 182804 · · · · · · · · · · · · · · · · · · · · · · · · 16MVA-BN-HER2 · · · · · · · · · · · · · · · · · · · · · · · 16GTx 758 · · · · · · · · · · · · · · · · · · · · · · · · · 17nabiximols · · · · · · · · · · · · · · · · · · · · · · · · 17VGX 3400 · · · · · · · · · · · · · · · · · · · · · · · · · 17mifepristone· · · · · · · · · · · · · · · · · · · · · · · · 17PENNVAX-B & PENNVAX-GP · · · · · · · · · · · · · · · · · · 18ondansetron· · · · · · · · · · · · · · · · · · · · · · · · 18AN 2728 · · · · · · · · · · · · · · · · · · · · · · · · · 18vaccine, NSCLC, Transgene · · · · · · · · · · · · · · · · · · 19Genz 112638 · · · · · · · · · · · · · · · · · · · · · · · 19NPL 2008 · · · · · · · · · · · · · · · · · · · · · · · · · 19rosuvastatin · · · · · · · · · · · · · · · · · · · · · · · · 20UDB · · · · · · · · · · · · · · · · · · · · · · · · · · · 20ALKS 27 · · · · · · · · · · · · · · · · · · · · · · · · · 20ChimeriVax-Dengue · · · · · · · · · · · · · · · · · · · · · 20riociguat · · · · · · · · · · · · · · · · · · · · · · · · · 21MAb, VAP-1, BioTie Therapies · · · · · · · · · · · · · · · · · 21Q 8003IR · · · · · · · · · · · · · · · · · · · · · · · · · 21IPI 940 · · · · · · · · · · · · · · · · · · · · · · · · · 21vaccine, cat allergy, Alk-Abello · · · · · · · · · · · · · · · · · 21
COM PANY FO CUS 23
GS 102 · · · · · · · · · · · · · · · · · · · · · · · · · · 23GS 101 · · · · · · · · · · · · · · · · · · · · · · · · · · 23GS 156 · · · · · · · · · · · · · · · · · · · · · · · · · · 24GS 168 · · · · · · · · · · · · · · · · · · · · · · · · · · 24GS 497c · · · · · · · · · · · · · · · · · · · · · · · · · 24GS 326 · · · · · · · · · · · · · · · · · · · · · · · · · · 24
NEWLY RE PORTED DRUGS IN R&D FO CUS 25
PROD UCT PHASE CHANGES RE PORTED IN R&D FO CUS 27
IMS HEALTH R&D FO CUS drugnews 2 March 2009
See R&Dfocus (Drug Up dates) for full prod uct de tails 3
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
agent was approved for these indications by the US FDA in
October 2008. Quetiapine is available worldwide as a
treatment for schizophrenia.
Approvals
prasugrel
Daiichi Sankyo, Lilly registered, EU (acute coronary
syndrome)
On 23 February 2009 Daiichi Sankyo and Lilly announced that
the European Commission has granted marketing approval to
prasugrel (EFIENT) for the prevention of atherothrombotic
events in patients with acute coronary syndrome (ACS)
undergoing percutaneous coronary intervention (PCI); this
follows a recommendation by the EMEA’s CHMP in December
2008.
Prasugrel, an oral antithrombotic prodrug that inhibits the
ADP receptor, is awaiting marketing approval from the US
FDA for the treatment of patients with ACS managed with PCI.
degarelix
Ferring registered, EU (prostate cancer)
Ferring announced on 19 February 2009 that the European
Commission has granted marketing approval to degarelix
(FIRMAGON), a synthetic gonadotropin-releasing hormone
(GnRH) antagonist, for the treatment of patients with
advanced, hormone-dependent prostate cancer. Phase III
trial data showed that degarelix treatment produced a rapid
and significant reduction in testosterone levels in more than
96% of patients. The agent was approved in the USA for the
treatment of prostate cancer in December 2008.
catumaxomab
Fresenius recommended for approval, EU (cancer)
Fresenius announced on 18 February 2009 that the CHMP of
the EMEA has issued a positive opinion recommending
catumaxomab (REMOVAB) for approval in the intraperitoneal
treatment of malignant ascites. The CHMP’s positive opinion
is based on results from an international phase II/III pivotal
study, which demonstrated a four-fold increase in puncture
free survival over a therapy consisting of puncture alone.
Approval by the European Commission is expected during
second quarter 2009.
bazedoxifene
Wyeth recommended for approval, EU
(osteoporosis)
Wyeth announced on 19 February 2009 that the EMEA’s CHMP
has adopted a positive opinion recommending approval of a
marketing authorization application for bazedoxifene
(CONBRIZA) for the treatment of postmenopausal
osteoporosis in women at increased risk of fracture. Data
from two multicenter, randomized, double-blind,
placebo-controlled phase III trials of bazedoxifene in more
than 9000 postmenopausal women suggested a favorable
endometrial profile for the agent and confirmed its selective
estrogen receptor modulator (SERM) characteristics.
Thromboembolic events were the most serious adverse
reactions reported and were more common with bazedoxifene
treatment than with placebo.
Wyeth is developing bazedoxifene, a once-daily oral SERM,
for the treatment and prevention of postmenopausal
osteoporosis. The agent is also pending approval in the USA
and Japan.
laromustine
Vion submitted for approval, USA (AML)
Vion reported on 17 February 2009 that it has filed an NDA
with the US FDA for laromustine (ONRIGIN) injection as a
monotherapy for remission induction in patients aged 60 or
older with de novo poor-risk acute myelogenous leukemia
(AML). The filing was based on data from an ongoing pivotal
phase II trial of the agent in 85 AML patients aged 65 years
and a previous phase II trial in elderly AML patients. Vion has
requested Priority Review of the submission.
Laromustine, a sulfonyl hydrazine alkylating, is also being
evaluated in a phase III trial in combination with standard
remission induction therapy in patients with AML and
myelodysplastic syndromes, a phase I/II trial in combination
with cytarabine in elderly AML patients and a phase I/II trial
in combination with temozolomide in the treatment of brain
tumors.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
4 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
CIP-Tramadol ER
Cipher receives tentative approval from US FDA
On 17 February 2009 Cipher announced that the US FDA has
granted tentative approval for CIP-Tramadol ER, an orally
administered, once-daily extended-release formulation of
tramadol, for the management of moderate to moderately
severe chronic pain. The agency stipulated that patent issues
relating to the reference product, ULTRAM ER, need to be
resolved before CIP-Tramadol ER can be marketed.
vorinostat
Merck & Co withdraws MAA
On 17 February 2009 Merck Sharp & Dohme, a Merck & Co
company, notified the EMEA of a decision to withdraw its
MAA, seeking centralized EU approval for vorinostat
(ZOLINZA) as a therapy for patients with advanced stage
cutaneous T-cell lymphoma (CTCL) who have progressive,
persistent or recurrent disease, and who have failed at least
two previous systemic therapies. The decision to withdraw
the application was based on a CHMP opinion that the data
provided were insufficient to grant marketing authorization.
Vorinostat, a histone deacetylase (HDAC) inhibitor, is
marketed in the USA for this indication.
drug delivery system, inhaled
aztreonam, Gilead Sciences
Gilead Sciences receives US FDA response to appeal
Gilead Sciences announced on 19 February 2009 that,
following its appeal (submitted under the formal dispute
resolution process in November 2008) regarding the US FDA’s
Complete Response letter to its NDA for aztreonam for
inhalation, the US FDA has restated its position, including
the need for an additional clinical trial to be conducted prior
to an NDA resubmission. Gilead Sciences plans to discuss
whether ongoing trials will meet this requirement.
Gilead Sciences is developing inhaled aztreonam for the
treatment of Gram negative bacterial infections in patients
with cystic fibrosis; the formulation is being developed using
PARI’s proprietary eFLOW Electronic Nebulizer. The agent is
also undergoing regulatory review in Canada and the EU.
valsartan + amlodipine +
hydrochlorothiazide
Novartis anticipates US approval
Novartis reported in a presentation of its Financial Results
2008, on 28 January 2009, that it expects to receive US
marketing approval for a combination of valsartan (an
angiotensin II receptor antagonist), amlodipine (a calcium
channel blocker), and hydrochlorothiazide (a diuretic),
during 2009. The combination therapy was submitted for
approval for the treatment of hypertension.
efalizumab
Merck Serono receives notification of EMEA and
Health Canada recommendations
Merck Serono announced on 19 February 2009 that the EMEA
has recommended to the European Commission the
suspension of Merck Serono’s MAA for efalizumab (RAPTIVA).
This follows three cases of progressive multifocal
leukoencephalopathy in psoriasis patients treated with the
agent since October 2008. The CHMP of the EMEA concluded
that the benefits of efalizumab no longer outweigh its risks.
Merck Serono will discuss next steps with the EMEA, and
health authorities outside the EU will be informed of the
EMEA recommendation. On 23 February 2009, Health Canada
recommended the suspension of the agent in Canada; EMD
Serono Canada, the Canadian affiliate of Merck Serono, will
suspend efalizumab. No new prescriptions will be issued and
the agent will no longer be available in Canada within a few
months.
Efalizumab, an anti-CD11a monoclonal antibody, has been
widely marketed in the EU as a therapy for adults with
moderate-to-severe plaque psoriasis since its approval in
2004. The agent is also marketed in several other countries,
including the USA and Australia for this indication.
drug delivery system, topical
diclofenac, Nuvo
Nuvo resubmits application for US marketing
approval
On 19 February 2009 Nuvo announced that it has resubmitted
an application for US approval of PENNSAID, a topical
See R&Dfocus (Drug Up dates) for full prod uct de tails 5
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
non-steroidal anti-inflammatory drug under development for
the relief of pain and stiffness associated with osteoarthritis.
The resubmission was accepted as a complete response to an
Approvable Letter issued by the US FDA in December 2006.
The US FDA has set a PDUFA date of 5 August 2009. PENNSAID
is available in Canada and several European Markets,
including the UK and Italy, for the treatment of
osteoarthritis.
BIFERONEX
Biopartners to appeal CHMP negative opinion
Following a negative opinion issued by the CHMP of the
EMEA, recommending the refusal of EU approval for
BIFERONEX, Biopartners announced on 19 February 2009 that
it will request a re-evaluation of the negative opinion. The
appeal is expected to last until June 2009.
In August 2007, Biopartners filed an MAA with the EMEA for
BIFERONEX, a recombinant interferon beta-1a, for the
treatment of relapsing remitting multiple sclerosis. The
product is a pH neutral and human serum albumin-free
formulation.
Licensing
ion channel modulators,
NeuroSearch/Lilly
Lilly, NeuroSearch licensing agreement
NeuroSearch announced on 17 February 2009 that it has
signed a collaborative agreement with Lilly relating to the
discovery and development of ion channel modulators for the
treatment of central nervous system disorders. Intellectual
property and know-how from both companies will be used to
develop these agents. Under the terms of the three-year
agreement, drug discovery will be conducted by NeuroSearch;
preclinical development of candidate drugs may also be
carried out by NeuroSearch. Individual agents covered by the
agreement (and related intellectual property) may be
licensed by Lilly by exercising various options. Once the
option has been exercised, Lilly will be responsible for all
development and commercialization. NeuroSearch will
receive a US$5 million upfront payment from Lilly, and an
additional US$8 million in research funding over the next
three years. Lilly will make a US$17 million equity investment
in NeuroSearch. NeuroSearch may receive milestone
payments of up to US$320 million plus royalties on worldwide
sales for each product successfully developed and
commercialized.
nitazoxanide
Chugai, Romark licensing agreement
Romark clinical data (hepatitis C)
Romark has signed a licensing agreement with Chugai, under
which Chugai acquires exclusive Japanese rights to develop
and commercialize nitazoxanide for the treatment of chronic
hepatitis C virus (HCV) infection. Under the terms of the
agreement, announced on 17 February 2009, Romark has
received an undisclosed upfront payment from Chugai, and
wil l be eligible to receive further cl inical- and
regulatory-based milestone payments. Romark will be
entitled to a share of profits from Japanese sales of
nitazoxanide through a supply agreement and royalties.
On 17 February 2009 Romark reported results from a phase I
and a phase II trial of a controlled-release formulation of
nitazoxanide in the treatment of HCV infection. Results from
a randomized, double-blind, crossover phase I trial (OPTIMA
HCN-1) showed that controlled-release nitazoxanide,
administered at 675 mg or 1350 mg bid po with food for seven
days, resulted in trough plasma concentrations of active
metabolite (tizoxanide) that were about three-fold and
12-fold the trough plasma concentrations observed in
previous trials using a standard nitazoxanide 500 mg tablet.
The agent was well tolerated with mild gastrointestinal side
effects reported. Data from the OPTIMA HCN-2 phase II trial,
involving 41 treatment-naive HCV genotype 4 infected
patients, showed that 82% and 100% of patients receiving
the low-dose and high-dose controlled-release nitazoxanide,
respectively, had undetectable serum HCV RNA after 12 weeks
of combination therapy with peginterferon alfa-2a and
ribavirin. Romark expects to report interim results from US
trials of the standard nitazoxanide tablet in patients with
chronic HCV genotype 1 infection during 2009.
Nitazoxanide, a thiazolide, is available in several markets
including Mexico, for the treatment of parasitic infections
and cryptosporidiosis in AIDS patients. Nitazoxanide
(ALINIA) is also marketed in the USA for the treatment of
6 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
diarrhea caused by Cryptosporidium parvum and Giardia
lamblia.
ABSTRAL
Neopharm Ltd, Orexo licensing agreement
On 18 February 2009 Orexo announced that it has entered
into an exclusive distribution agreement with Neopharm Ltd,
under which Neopharm Ltd has been granted the marketing
and sales rights to ABSTRAL in Israel for the treatment of
breakthrough cancer pain. Orexo will provide Neopharm Ltd
with the product in return for significant margins on the
sales. Neopharm Ltd will assume responsibility for the
regulatory approval process in Israel. Orexo is eligible to
receive milestone payments from Neopharm Ltd; financial
details of the agreement were not disclosed.
ABSTRAL, a sublingual mucoadhesive formulation of
fentanyl, was first launched in Sweden in August 2008 for the
treatment of breakthrough cancer pain. The agent was
subsequently launched for the same indication in the UK and
Germany in January 2009.
eslicarbazepine acetate
Bial, Eisai licensing agreement
Bial recommended for approval, EU (epilepsy)
Bial has signed an agreement with Eisai, granting Eisai
exclusive rights to market, promote and distribute
eslicarbazepine acetate (ZEBINIX) in Europe. Under the terms
of the agreement, announced on 19 February 2009,
development and production of the agent will be Bial’s
responsibility. Bial will supply the finished product to Eisai
and will also have an option to co-promote eslicarbazepine
acetate with Eisai in Europe. Upfront payments and milestone
payments for additional epilepsy approvals within Europe
worth EUR95 million will be paid to Bial.
On 19 February 2009, the CHMP of the EMEA issued a positive
opinion, recommending the EU approval of eslicarbazepine
acetate as adjunctive therapy in adults with partial-onset
seizures, with or without secondary generalization.
Eslicarbazepine acetate, an oral, once-daily sodium channel
blocker, is expected to receive EU approval during second
quarter 2009.
lubiprostone
Abbott, Sucampo licensing agreement
Sucampo announced on 19 February 2009 that it has entered
into a license agreement with Abbott, under which Abbott
has been granted exclusive rights to commercialize
lubiprostone (AMITIZA) in Japan for the treatment of chronic
idiopathic constipation (CIC). Abbott will assume
responsibility for all marketing expenses and efforts, and will
have the option of first refusal to any additional indications
for which the agent is developed in Japan. Sucampo will
receive an upfront payment of US$10 million from Abbott,
and is also eligible to receive certain specified development
and commercialization milestone payments. Sucampo will
continue to have responsibility for development of
lubiprostone and the regulatory process in Japan, and for all
development costs for the agent. Following marketing
authorization and pricing approval, Abbott will purchase
lubiprostone from Sucampo for distribution in Japan.
Sucampo will retain the right to co-promote the agent in
Japan. In addition, the two companies have agreed to begin
negotiations for a license, commercialization and supply
agreement for lubiprostone in other available territories.
Lubiprostone, a functional fatty acid chloride channel
opener, is available in the USA for the treatment of CIC in
adults and irritable bowel syndrome with constipation in
adult women. The agent is pending approval in several EU
countries for CIC.
ProLindac
Access, Dong-A licensing agreement
On 19 February 2009 Access and JCOM, an affiliate of Dong-A,
announced that they have entered into a definitive licensing
agreement under which JCOM and Dong-A have been granted
the rights to manufacture, develop and commercialize
ProLindac in South Korea. Under the terms of the agreement,
Access will receive an upfront fee and subsequent milestone
payments from JCOM, in addition to a double-digit royalty
upon commercialization of Prolindac. In cooperation with
Access, JCOM has agreed to fund and conduct a phase II
combination trial of the agent in an indication to be
determined by the companies; this study could cost up to
US$10 million. Dong-A will assume responsibility for
obtaining regulatory approval and for commercialization of
ProLindac in South Korea.
See R&Dfocus (Drug Up dates) for full prod uct de tails 7
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
ProLindac, a DACH-platinum prodrug, is undergoing phase II
evaluation for the treatment of ovarian cancer and phase I
evaluation for head and neck cancer.
cancer therapy, Stockbridge
Pharmaceuticals
Columbia University, Stockbridge Pharmaceuticals
licensing agreement
On 20 February 2009 Science & Technology Ventures, the
technology transfer office at Columbia University (USA),
announced that it has signed an agreement with spin-out
company Stockbridge Pharmaceuticals for the development
of a small molecule-based approach for the treatment of
cancer. The small molecules target a protein receptor
involved in a cell signaling pathway that inhibits
programmed cell death, including cancerous cells.
Significant shrinkage of tumor mass was induced by the
therapy in a mouse model of basal-like breast cancer tumors.
The therapy was developed in collaboration with the
University of Nebraska (USA).
caerulomycin A
Institute of Microbial Technology, Nostrum
licensing agreement
Nostrum reported on 23 February 2009 that it has acquired
worldwide development and commercialization rights to
caerulomycin A, and its proprietary derivatives and
analogues for use in immunosuppression, from the Institute
of Microbial Technology (IMTECH;India). In preclinical
studies, the caeru lomycins demonstrated
immunosuppression of both T- and B-cells in in-vitro and in
rodent models. There were no toxic effects in rodents. The
caerulomycins have potential use in the prevention of organ
transplant rejection and in the treatment of autoimmune
diseases and other indications.
drug delivery system, thermostable
epoprostenol, Actelion
Actelion, GeneraMedix licensing agreement
Actelion announced on 23 February 2009 that it has signed
an agreement with GeneraMedix relating to a thermostable,
intravenous formulation of epoprostenol for the treatment of
pulmonary hypertension. Under the terms of the agreement,
Actelion acquires rights to develop, register and
commercia l i ze the thermostable formulat ion of
epoprostenol, worldwide. Financial details were not
disclosed.
The agent, which is stable at room temperature for up to 24 h,
was approved by the US FDA on 27 June 2008 for the
long-term treatment of primary pulmonary hypertension and
pulmonary hypertension associated with the scleroderma
spectrum of the disease in NYHA class III and class IV
patients who have not responded adequately to standard
therapy.
XmAb
CSL, Xencor licensing agreement
Xencor and CSL have entered into an antibody optimization
collaboration. Under the terms of the agreement, announced
on 23 February 2009, CSL will have access to Xencor’s XmAb
technology platform to enhance the ADCC effector function
of its antibodies. CSL has received several commercial
licenses allowing products that utilize Xencor’s XmAb
technology to be developed and commercialized. Xenocor will
be eligible to receive milestone payments and product
royalt ies for any products f rom this agreement
commercialized by CSL.
XmAb technology utilizes Xencor’s Protein Design
Automation (PDA) technology to generate antibodies
containing engineered Fc regions with increased binding
affinity for the Fc gamma receptors that mediate the immune
system’s cytotoxic effector functions.
cannabinoid-2 receptor agonists,
Reperio
Pharmos, Reperio sign asset purchase agreement
Pharmos announced on 18 February 2009 that it has signed
an asset purchase agreement with Reperio, under which
Pharmos has sold certain cannabinoid-2 (CB2) receptor
agonists to Reperio. Under the terms of the agreement, which
is expected to close on or before 6 June 2009, Reperio
acquires patent rights and technical know-how to PRS
639058 and certain follow-on agents. Pharmos will be
8 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
awarded an equity ownership in Reperio. Pharmos will also be
entitled to receive license and royalty fees.
The agents have been evaluated in preclinical studies for
neuropathic pain; the CB2 agonists may also be useful as
analgesic, neuroprotective, immunomodulatory and
anti-inflammatory agents.
drug discovery, HUYA/Beijing
Institute of Materia Medica
Beijing Institute of Materia Medica, HUYA
collaborate on preclinical and clinical studies
Beijing Institute of Materia Medica, HUYA sign
option agreement
HUYA announced on 18 February 2009 that it has signed a
collaborative agreement with the Beijing Institute of Materia
Medica, Chinese Academy of Medical Sciences and Peking
Union Medical College (China) (known collectively as Beijing
Institute of Materia Medica;BIMM), under which HUYA
acquires the right of first review and negotiation for the
licensing and development of certain BIMM compounds with
therapeutic potential in the areas of cardiology,
endocrinology, oncology, immunology, hematology,
neuroscience and anti-infectives. HUYA and BIMM will jointly
conduct preclinical and clinical testing. Further details of the
agreement were not disclosed.
ibritumomab tiuxetan
Cell Therapeutics, Spectrum option exercised
Cell Therapeutics reported on 20 February 2009 that it has
exercised an option to sell its 50% ownership interest in its
joint venture with Spectrum to develop and commercialize
ibritumomab tiuxetan (ZEVALIN) in the USA, to Spectrum. At
the closing of this sale, Spectrum will pay US$6 million to Cell
Therapeutics; the remainder of the US$18 million sale price
will be paid within 90 days of closing of this sale option
transaction. The joint venture was established in December
2008; Spectrum purchased a 50% membership interest in the
venture for US$15 million and various milestone payments.
Ibritumomab tiuxetan, an anti-CD20 monoclonal antibody
conjugated to yttrium-90, is available in the USA for the
treatment of relapsed or refractory low grade, follicular or
transformed B-cell non-Hodgkin’s lymphoma.
MEDUSA
Flamel Technologies, Merck Serono licensing
agreement modified
Flamel Technologies, Merck Serono milestone
payment
Flamel Technologies announced on 17 February 2009 that it
has entered into a full license agreement with Merck Serono
for the use of Flamel Technologies’ MEDUSA nanoparticle
delivery system in the extended-release delivery of an
already-marketed therapeutic protein; Merck Serono has
exercised its option, under an agreement signed in 2007, to
pursue a partnership with Flamel Technologies. Flamel
Technologies will receive a milestone payment of EUR5
million for the exercise of this option and is eligible to
receive further payments based on research and development
milestones. Research and development activities will be
funded by Merck Serono. Further terms of the agreement were
not disclosed.
Flamel Technologies’ MEDUSA is a self-assembled
poly-aminoacid nanoparticle system which acts as a carrier
for the development of long-acting formulations of peptides,
proteins and other large molecules.
Technology Transfer Spotlight
Opportunities with German
Cancer Research Center
fusion polypeptides, German
Cancer Research Center
German Cancer Research Center partnering
opportunity, Worldwide
Researchers at the German Cancer Research Center (Germany)
are developing fusion polypeptides with potential utility as
cancer therapeutics. The fusion polypeptides consist of an
effector protein, or a binding site thereof, with a targeting
sequence, resulting in targeted cytotoxicity towards tumor
cells. Discovery stage evaluations are ongoing in Germany.
The fusion polypeptides are available for partnering or
licensing, worldwide.
See R&Dfocus (Drug Up dates) for full prod uct de tails 9
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
For further information on the partnering opportunities
available, contact:
Christine Amshoff
German Cancer Research Center
Technologietransfer T010
Im Neuenheimer Feld 280
69120 Heidelberg
Germany
Tel: +49 6221 422950
Fax: +49 6221 422956
Email: [email protected]
parvovirus NS1 variants, German
Cancer Research Center
German Cancer Research Center partnering
opportunity, Worldwide
The German Cancer Research Center’s (Germany) parvovirus
NS1 protein variants, in which the equilibrium between
replication and transcription activities and cytotoxic activity
has been shifted, are available for worldwide licensing or
partnering. Preclinical evaluation of the variants, which have
potential in the treatment of cancer, is ongoing in Germany.
For further information on the partnering opportunities
available, contact:
Christine Amshoff
German Cancer Research Center
Technologietransfer T010
Im Neuenheimer Feld 280
69120 Heidelberg
Germany
Tel: +49 6221 422950
Fax: +49 6221 422956
Email: [email protected]
parvovirus H-1 protein, German
Cancer Research Center
German Cancer Research Center partnering
opportunity, Worldwide
Researchers at the German Cancer Research Center (Germany)
are developing the oncolytic parvovirus H-1 protein for the
potential treatment of brain tumors, particularly
glioblastoma. The parvovirus H-1 protein effectively killed
tumor cells in mouse rat and human glioma cell lines. In
addition, parvovirus H-1 did not elicit inflammatory
responses or tissue damage following intracerebral injection
in healthy rats; however, effective antitumor responses were
observed following injection of the H-1 virus in residing
intracerebral gliomas of rats with brain tumors. Preclinical
studies are ongoing in Germany. The parvovirus H-1 protein is
available for licensing or partnering, worldwide.
For further information on the partnering opportunities
available, contact:
Christine Amshoff
German Cancer Research Center
Technologietransfer T010
Im Neuenheimer Feld 280
69120 Heidelberg
Germany
Tel: +49 6221 422950
Fax: +49 6221 422956
Email: [email protected]
NS1 protein/VP1 capsid protein,
German Cancer Research Center
German Cancer Research Center partnering
opportunity, Worldwide
The German Cancer Research Center’s (Germany) toxin
comprising two parvoviral products, the NS1 protein and part
of the capsid VP1 protein, is available worldwide for
partnering or licensing. The toxin has potential in the
treatment of glioblastomas. Preclinical evaluation is ongoing
in Germany. The intrinsic cytotoxicity of NS1, as measured by
the disappearance of VP1/NS1 containing cells as a function
of time, was significantly increased by the presence of the
VP1 variant. An induction of cytolysis, as demonstrated by
the release of lactate dehydrogenase, was apparent in the
presence of wild-type NS1 and the VP1 specific region (this
was absent upon sole expression of NS1).
For further information on the partnering opportunities
available, contact:
Christine Amshoff
German Cancer Research Center
Technologietransfer T010
Im Neuenheimer Feld 280
69120 Heidelberg
10 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
Germany
Tel: +49 6221 422950
Fax: +49 6221 422956
Email: [email protected]
Pipeline News
Offers from CNSBio
CNSB001
CNSBio partnering opportunity, Worldwide
CNSBio is developing CNSB001 (CNSB 001), a combination
therapy based on the co-administration of the potassium
channel opener flupirtine with an opioid drug, for the
treatment of neuropathic pain. A phase IIa trial of the
technology in the treatment of neuropathic pain in
HIV-infected patients is ongoing. A phase I/IIa trial in
cancer patients with neuropathic pain has completed; phase
IIb evaluation in Europe is planned for commencement in
late 2009. CNSB001 is available for partnering, worldwide.
For further information on the partnering opportunities
available, contact:
Ian Cooke
Managing Director
CNSBio Pty Ltd
Suite 1
651 Victoria Street
Abbotsford
Victoria 3067
Australia
Tel: +61 3 8663 7300
Fax: +61 3 9421 0044
Email: [email protected]
CNSB004
CNSBio partnering opportunity, Worldwide
CNSBio is developing CNSB004 (CNSB 004), an injectable
peptide modulator of N-type voltage sensitive calcium
channels, for the potential treatment of severe pain.
Preclinical studies are ongoing in Australia; phase I/IIa
evaluation is expected to start in late 2009. CNSB004 is
available for partnering, worldwide.
For further information on the partnering opportunities
available, contact:
Ian Cooke
Managing Director
CNSBio Pty Ltd
Suite 1
651 Victoria Street
Abbotsford
Victoria 3067
Australia
Tel: +61 3 8663 7300
Fax: +61 3 9421 0044
Email: [email protected]
CNSB006
CNSBio partnering opportunity, Worldwide
CNSBio is developing CNSB006 (CNSB 006), an oral fixed-dose
combination therapy consisting of a neurokinin 1 antagonist
and an ion channel modulator, for the potential treatment of
inflammatory pain. A phase IIa trial of the agent in pain in
osteoarthritis patients is planned. This program is available
for partnering, worldwide.
For further information on the partnering opportunities
available, contact:
Ian Cooke
Managing Director
CNSBio Pty Ltd
Suite 1
651 Victoria Street
Abbotsford
Victoria 3067
Australia
Tel: +61 3 8663 7300
Fax: +61 3 9421 0044
Email: [email protected]
See R&Dfocus (Drug Up dates) for full prod uct de tails 11
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
©2006 IMS Health Incorporated or its affiliates. All Rights Reserved.
Need an insight into the biotech industry?
The best way to know.
Ask IMS.BioPharmaceutiques accesses the latest biotech information with a resolutely European
angle and interprets the sector’s news from a pharma perspective.This weekly newsletter delivers high-quality content to your e-mail inbox including:
• Biotech-biotech, pharma-biotech alliances and M&A activity• The latest IPOs
• The latest milestones and investment rounds• An overview of the most recent biotech start-ups
• Clinical trial launches• The latest developments in the field of biotech drugs
• News on European directives and regulations • A “movers and shakers” section, with the latest appointments
Subscribers can also access a dedicated website, updated weekly from the latest editions of BioPharmaceutiques, that provides summary tables of molecule, company and disease-specific
overviews of the latest negotiations, alliances and developments. Subscribers only need to purchase one subscription for company-wide access.
For more information, visit www.biopharmaceutiques.com/en or call subscriptions: +33 144 058 300
©2008 IMS Health Incorporated or its affiliates. All rights reserved.
News from Shire's 2008 Results
lanthanum carbonate
Shire registered, Japan (hyperphosphatemia)
Shire reported in a presentation of its results for the year to
31 December 2008, on 19 February 2009, that lanthanum
carbonate (FOSRENOL) was approved in Japan in October
2008. Lanthanum carbonate, a phosphate binding agent for
the treatment of hyperphosphatemia in patients with chronic
kidney failure, was launched in the USA in January 2005 and
has subsequently been marketed for this indication in several
countries worldwide.
Lanthanum carbonate will be marketed in Japan by Bayer
Yakuhin, Bayer’s subsidiary. A Japanese launch is anticipated
first half 2009.
HGT 2610
Shire provides development update
Shire reported in a presentation of its results for the year to
31 December 2008, on 19 February 2009, that it is developing
HGT 2610 as an enzyme replacement therapy for Krabbe
disease (globoid cell leukodystrophy), a lysosomal storage
disorder. Early-stage preclinical development is under way in
Europe.
SPD 503
Shire resubmits NDA to US FDA
In a presentation of its results for the year to 31 December
2008, on 19 February 2009, Shire announced that on 27
January 2009 it resubmitted an NDA to the US FDA for SPD
503 (INTUNIV) for the treatment of attention deficit
hyperactivity disorder (ADHD) in children. In June 2007, the
FDA issued an approvable letter in response to Shire’s August
2006 filing for SPD 503 for ADHD in children and adolescents.
SPD 503, an oral once-daily extended-release formulation of
guanfacine hydrochloride, is a noradrenergic alpha-2 agonist
with specific affinity for the A subtype prevalent in the
cerebral cortex.
icatibant
Shire provides development update
In a presentation of its results for the year to 31 December
2008, on 19 February 2009, Shire reported on a December
2008 meeting between the US FDA and Shire’s subsidiary,
Jerini. In the meeting, to discuss the FDA’s not approvable
letter for icatibant (FIRAZYR), it was agreed that an
additional clinical study would be required. A complete
response to the not approvable letter will be filed following
this trial, which is expected to initiate third quarter 2009.
Icatibant, a bradykinin-2 receptor antagonist, was launched
in September 2008 in the UK and Germany for the treatment
of acute attacks of hereditary angioedema (HAE).
Products & Biotechnology
resatorvid
Takeda discontinued
Takeda reported on 20 February 2009 that it has discontinued
further development of resatorvid (TAK 242). The decision
was taken because the program did not meet Takeda’s criteria
to support continued development; not because of concerns
over safety or efficacy of the agent. Resatorvid, an inhibitor
of inflammatory mediator production, had been under phase
III evaluation in the treatment of severe sepsis.
NS 2359
GlaxoSmithKline discontinued, Worldwide
(depression)
GlaxoSmithKline clinical data (phase II) (depression)
On 23 February 2009 NeuroSearch reported that data from a
phase II proof-of-concept program of NS 2359, conducted by
GlaxoSmithKline in patients with major depressive disorder,
do not support further development of the agent for the
treatment of depression. Results from two ten-week
randomized, double-blind, active- and placebo-controlled
trials in approximately 900 patients demonstrated no
significant difference in efficacy between NS 2359 and
placebo on key efficacy endpoints of the Montgomery-Asberg
Depression Rating Scale (MADRS), Bech 6-item Scale
See R&Dfocus (Drug Up dates) for full prod uct de tails 13
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
(subscale of the HAM-D-17) and IDS-CR (Inventory of
Depressive Symptomatology - Clinicians Rated). However,
treatment with active controls (paroxetine or venlafaxine)
showed statistically significant efficacy compared with
placebo. GlaxoSmithKline is conducting further analysis and
evaluation of the phase II data.
NS 2359, a triple 5HT, noradrenaline and dopamine reuptake
inhibitor, is also undergoing phase II evaluation in the USA
for the potential treatment of attention deficit hyperactivity
disorder (ADHD) and drug addiction.
albiglutide
GlaxoSmithKline phase change III, USA (diabetes)
GlaxoSmithKline announced on 17 February 2009 that it has
begun dosing in a phase III program of albiglutide (SYNCRIA)
in patients with type II diabetes. The program, involving over
4000 patients enrolled in five studies, will aim to evaluate
the efficacy and cardiovascular safety of the agent,
administered as monotherapy or as an adjuvant at a dose of
30 mg weekly. Most studies will include an active comparator
drug (including metformin, sulfonylurea, thiazolidinedione,
insulin and a dipeptidyl peptidase IV inhibitor). The primary
endpoint for all studies will be the change from baseline in
HbA1c compared with placebo and/or active comparator
drugs. Initiation of this phase III program will result in a
US$9 million milestone payment to Human Genome Sciences,
to be paid during first quarter 2009.
Albiglutide, an injectable glucagon-like peptide-1 (GLP-1)
agonist, is a fusion protein consisting of human albumin and
GLP-1 which was originated by Human Genome Sciences
using its proprietary albumin-fusion technology. Human
Genome Sciences licensed albiglutide to GlaxoSmithKline in
October 2004.
aliskiren + amlodipine +
hydrochlorothiazide
Novartis phase change III, USA, Belgium, Slovak
Republic, Egypt, Germany, Poland, Spain, Turkey
(hypertension)
In its annual report 2008, Novartis announced that it is
developing a single pill combination (SPC) of aliskiren,
amlodipine and hydrochlorothiazide for the treatment of
hypertension. Phase III evaluation of the combination is
ongoing, and regulatory submissions in the EU and the USA
are expected in 2010.
vaccine, Clostridium difficile,
Sanofi Pasteur
Sanofi Pasteur phase change II, UK (bacterial
infection)
On 17 February 2009 Sanofi Pasteur announced the initiation
of a proof-of-concept phase II trial of its Clostridium difficile
vaccine (ACAM-Cdiff) in the UK. The study will assess the
safety and efficacy of the vaccine in patients infected with C
difficile. The orally active toxoid vaccine is being developed
for the prevention of C difficile infection. It consists of
native, full length, inactivated toxins A and B, which are
highly immunogenic. This vaccine entered the portfolio of
Sanofi Pasteur via the acquisition of Acambis in September
2008.
nicotinic acid receptor agonist,
Merck & Co/Arena
Merck & Co phase change II, USA (atherosclerosis)
On 18 February 2009 Arena announced that its development
partner, Merck & Co, has initiated a phase II trial of an oral
nicotinic acid (niacin) receptor agonist for the potential
treatment of atherosclerosis. The randomized, double-blind,
placebo-controlled study will evaluate the safety, tolerability
and efficacy of the agent in patients with dyslipidemia.
Arena and Merck & Co entered into their research and
licensing agreement for the development of cardiovascular
therapeutics in October 2002.
AV 951
AVEO phase change II, USA, Europe (breast cancer)
On 19 February 2009 AVEO reported that it has initiated an
open-label phase Ib/IIa trial of AV 951 in the USA and Europe
in patients with advanced breast cancer. The phase Ib section
of the trial will evaluate combined daily escalating,
sequential oral doses of AV 951 with standard weekly
administration of iv paclitaxel in approximately 20 patients
with progressive disease. Primary outcome measures include
safety, tolerability and maximum tolerated dose (MTD).
14 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
Following determination of the MTD, the phase IIa portion of
the study will evaluate the clinical efficacy of a combined AV
951 and paclitaxel regimen in approximately 25 to 50
patients with metastatic breast cancer and no prior exposure
to chemotherapy.
AV 951, a quinoline-urea derivative and specific inhibitor of
VEGF receptors 1, 2 and 3, is also undergoing phase II
evaluation in patients with metastatic renal cell carcinoma.
EC 145
Endocyte phase change II, USA, Europe, Canada
(ovarian cancer)
Endocyte announced on 19 February 2009 that it has
initiated a randomized phase II trial of EC 145 in patients
with p lat inum-res istant ovar ian cancer . This
US/Canadian/European trial (designated PRECEDENT),
involving 122 patients, will aim to evaluate the safety and
efficacy of EC 145 given in combination with pegylated
liposomal doxorubicin compared with pegylated liposomal
doxorubicin monotherapy. Endocyte’s molecular imaging
agent, EC 20, will be used to identify patients who will
benefit from EC 145 therapy.
EC 145 is a folate-targeted drug conjugate comprising folic
acid and the vinca alkaloid, desacetylvinblastine hydrazide
(DAVLBH).
vaccine, gene-based, HIV infection,
GeoVax
GeoVax phase change II, USA, Peru (HIV infection)
On 20 February 2009 GeoVax announced that the first
patients in a phase IIa trial, designated HVTN 205, have
received its candidate HIV/AIDS vaccine. The trial, which is
being conducted by the HIV Vaccine Trials Network (HVTN),
will enroll 225 volunteers across 11 sites in the USA and two
sites in Peru. Of the 225 volunteers, 150 will receive GeoVax’s
vaccine and 75 will receive placebo.
GeoVax’s anti-HIV vaccine employs a prime-boost approach,
with a DNA priming dose, followed by the delivery of a
recombinant modified vaccinia Ankara (rMVA) booster. The
vaccine is designed to stimulate anti-HIV T-cell and anti-HIV
antibody immune responses.
TB 402
BioInvent, ThromboGenics phase change II, Europe
(thrombosis)
On 23 February 2009 ThromboGenics and BioInvent
announced that the first patient has been enrolled in a phase
II trial of TB 402 in Central Europe. A single intravenous bolus
injection of the agent will be assessed in the treatment of
deep vein thrombosis after knee surgery in approximately 300
patients. The safety and efficacy of three escalating doses of
TB 402 will be the primary endpoint of the study. The study is
expected to complete by end of 2010. TB 402, a recombinant
long-acting human monoclonal antibody directed against
factor VIII, has potential in the prevention and treatment of
thrombosis, and the prevention of blood clots associated
with atrial fibrillation.
SGN 35
Seattle Genetics phase change II, USA, Canada,
Europe (Hodgkin's lymphoma)
On 19 February 2009 Seattle Genetics reported that it has
initiated a pivotal phase II trial of SGN 35 in patients with
relapsed or refractory Hodgkin’s lymphoma, under a Special
Protocol Assessment (SPA) with the US FDA. The single-arm
multicenter study in the USA, Canada and Europe, will
evaluate the safety and efficacy of SGN 35 (1.8 mg/kg every
three weeks) in approximately 100 patients who have
previously received autologous stem cell transplantation.
The primary endpoint of the trial will be objective response
rate; secondary endpoints include duration of response,
progression-free survival, overall survival and tolerability.
SGN 35, an anti-CD30 antibody conjugated with eight
molecules of the cytotoxic drug monomethyl auristatin E
(MMAE), is being developed for the potential treatment of
hematological cancers. A phase I dose-escalation trial of SGN
35 is ongoing in the USA; data are expected to be reported
during 2009. Seattle Genetics also plans to initiate a phase II
trial in approximately 55 patients with relapsed or refractory
systemic anaplastic large cell lymphoma (ALCL) in the first
quarter of 2009. An NDA filing for the agent under the
accelerated approval regulations is planned for 2011.
See R&Dfocus (Drug Up dates) for full prod uct de tails 15
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
NKTR 105
Nektar phase change I, USA (solid tumor)
Nektar reported on 17 February 2009 that it has begun a US
phase I trial to assess the safety, pharmacokinetics and
antitumor activity of NKTR 105 in approximately 30 patients
with refractory solid tumors who have failed all prior
treatments. The agent is a pegylated form of docetaxel that
utilizes Nektar’s advanced polymer conjugate technology.
DM 1992
DepoMed phase change I, Russia (Parkinson's
disease)
On 17 February 2009 DepoMed announced that it has
initiated a randomized, open-label, crossover phase I trial in
Russia to compare the pharmacokinetics of two distinct
formulations of DM 1992 and a generic version of Sinumet CR
sustained-release levodopa/carbidopa, in approximately 18
patients with stable Parkinson’s disease. DM 1992 is a gastric
retentive extended-release formulation of levodopa and
carbidopa, which is being developed using DepoMed’s
AcuForm delivery technology. In July 2008, DepoMed was
awarded a grant by the Michael J. Fox Foundation for the
development of DM 1992.
ABT 450
Abbott, Enanta phase change I, USA
Abbott and Enanta announced on 18 February 2009 that they
have initiated a phase I trial of ABT 450. The double-blind,
placebo-controlled study will evaluate the safety,
tolerability, and pharmacokinetics of single, ascending oral
doses of ABT 450 in healthy volunteers.
ABT 450, a hepatitis C virus (HCV) NS3 and NS3/4A protease
inhibitor, was discovered by Abbott and Enanta as part of
their collaboration agreement to develop agents to treat HCV
infection. In preclinical studies, ABT 450 demonstrated
favorable potency across various HCV genotypes and highly
resistant strains in vitro.
LX 214
Lux Biosciences phase change I, USA (eye disease)
Lux Biosciences reported on 17 February 2009 that it has
initiated a phase I trial of LX 214. The study will evaluate the
safety and tolerability of the agent when applied topically to
the eye of patients with keratoconjunctivitis sicca (dry eye
syndrome) and healthy volunteers.
LX 214, a mixed micellar formulation of the calcineurin
inhibitor voclosporin, is being developed for the potential
treatment of dry eye syndrome and other inflammatory eye
diseases.
CB 182804
Cubist phase change I, USA
Cubist reported on 17 February 2009 that it has initiated a US
phase I trial to evaluate the safety and pharmacokinetics of
CB 182804 in healthy volunteers. CB 182804 is a broad
spectrum lipopeptide antibiotic being developed for the
intravenous treatment of multi-drug resistant Gram negative
bacterial infections. Following the results of this study,
Cubist plans to conduct phase II trials to assess the safety
and efficacy of the agent in patients with Gram negative
infections.
MVA-BN-HER2
Bavarian Nordic clinical data (breast cancer)
Pharmexa sells patent rights to Bavarian Nordic
On 23 February 2009 Bavarian Nordic reported updated
results from phase I/II trials of MVA-BN-HER2 in breast
cancer patients. The primary endpoint (safety) was met, and
approximately 70% of evaluated patients had an
MVA-BN-HER2-induced anti-HER2 immune response. To date,
no disease progression has occurred in 15 out of 30 patients
(after a six-month period). One complete response and one
partial response was observed when the vaccine was
administered with chemotherapy. MVA-BN-HER2 was well
tolerated and no vaccine-related serious adverse events were
reported. A moderate overall immune response was observed.
An improved version of the vaccine had a significantly higher
T-cell immune response in preclinical studies compared with
the original vaccine. The improved vaccine was also effective
16 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
in other tumor models in HER2 transgenic mice.
Consequently, Bavarian Nordic has decided to advance the
improved MVA-BN-HER2 vaccine into clinical development; a
single-site US phase I/II trial of this vaccine is expected to
begin during 2009. This study, involving 24 patients, will test
the vaccine in metastatic breast cancer patients and as an
adjuvant in breast cancer therapy.
On 23 February 2009, Pharmexa announced that it has sold
patent rights to MVA-BN-HER2 to Bavarian Nordic. In return,
Pharmexa will receive a single cash payment. MVA-BN-HER2
is a vaccine for the treatment of breast cancer, comprising
Pharmexa’s HER-2 DNA AutoVac vaccine formulated with
Bavarian Nordic’s MVA-BN vector technology.
GTx 758
GTx phase change I, USA
On 23 February 2009 GTx announced the initiation of a
double-blind, placebo-controlled phase I trial of GTx 758 in
healthy men. The study will evaluate the safety, tolerability
and pharmacokinetics of a single ascending dose of the
agent. GTx 758 is an orally-available luteinizing hormone
inhibitor, being developed for the potential treatment of
advanced prostate cancer. In preclinical studies, the agent
rapidly suppressed secretion of luteinizing hormone and then
reduced the production of androgens by the testes in vivo.
GTx plans to initiate a proof-of-concept phase I trial of GTx
758 in 2009.
nabiximols
GW Pharmaceuticals clinical data (muscle spasm)
On 24 February 2009 GW Pharmaceuticals reported results
from a randomized, placebo-controlled, withdrawal study of
nabiximols (SATIVEX) in 36 patients with spasticity
associated with multiple sclerosis. The primary endpoint
(time to treatment failure) was statistically significantly in
favor of nabiximols (p=0.013). In the patient global
impression of change and the carer functional ability global
impression of change, the difference between nabiximols and
placebo was significant (p=0.017 and p=0.001, respectively).
Patients who discontinued nabiximols therapy did not have
withdrawal syndromes, and the severity and frequency of
adverse events were similar for both nabiximols- and
placebo-treated patients. GW Pharmaceuticals expects to
report results from a phase III trial of nabiximols in patients
with spasticity associated with multiple sclerosis end first
quarter 2009. A European regulatory submission for this
indication is expected during second quarter 2009.
The product is marketed in Canada for the treatment of
neuropathic pain in patients with multiple sclerosis and as an
adjunctive analgesic treatment in adult patients with
advanced cancer who experience mild-to-moderate pain.
Nabiximols, a product based on extracts of the plant Cannabis
sativa containing tetrahydrocannabinol and cannabidiol, is
administered as an oral spray.
VGX 3400
VGX preclinical data
VGX reported on 18 February 2009 preclinical data for VGX
3400, an avian influenza DNA vaccine, delivered by
intramuscular or intradermal electroporation (using the
CELLECTRA device). The studies, conducted in macaques in
collaboration with the University of Pennsylvania (USA),
showed that after two vaccinations with VGX 3400, all
macaques in both the intramuscular and intradermal groups
developed hemagglutination inhibition (HAI) titers of
greater than 1:40 against a clade 1 H5N1 virus (sufficient for
protection). The vaccine also elicited HAI titers of greater
than 1:40 against unmatched clade 2 viruses (sub-types 2.1;
2.2; 2.3.4). The vaccine induced significant humoral immune
responses, as well as cellular immune responses to each
component antigen. After post-vaccination challenge with
the A/Vietnam/1203/04 strain, animals achieved significant
reductions in average viral load and disease symptoms
compared with non-vaccinated animals.
VGX 3400 combines three SynCon-based consensus vaccine
constructs targeting HA, NA, and M2e-NP antigens of
H5-influenza; the constructs are not matched to a specific
strain in either of the two main avian influenza families
(clade 1 and clade 2).
mifepristone
Corcept clinical data
Corcept reported on 23 February 2009 data for the main
secondary endpoints of a four-week randomized,
double-blind, controlled clinical trial to evaluate the ability
of mifepristone (CORLUX) to reduce body weight gain and
See R&Dfocus (Drug Up dates) for full prod uct de tails 17
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
other metabolic effects related to risperidone (RISPERDAL)
therapy. In the trial, 75 healthy men with a body mass index
of 23 or less, were given risperidone plus placebo (n=30),
risperidone plus mifepristone (n=30) or mifepristone plus
placebo (n=15). Secondary endpoint results showed that
increases in waist circumference (a measure of abdominal
fat) were 2.03 cm and 3.57 cm in the risperidone plus
mifepristone and risperidone plus placebo groups,
respectively (p less than 0.05). Increases in fasting insulin
levels were 1.80 mU/L and 10.97 mU/L (p less than 0.05) and
increases in triglyceride levels were 3.13 mg/dL and 30.57
mg/dL (p less than 0.01) in the respective groups. Previously
announced primary endpoint data indicated mean increases
in body weight which were 5.1 lbs and 9.2 lbs in the
risperidone plus mifepristone and risperidone plus placebo
groups, respectively (p less than 0.0001).
Mifepristone, a GR-II receptor antagonist, is under phase III
evaluation as a therapy for psychotic depression and
Cushing’s syndrome.
PENNVAX-B & PENNVAX-GP
VGX preclinical data
VGX reported on 18 February 2009 data from preclinical
studies, conducted in collaboration with the University of
Pennsylvania (USA), to assess PENNVAX HIV DNA vaccines in
a simian immunodeficiency virus (SIV) challenge non-human
primate model. The magnitude, frequency, proliferative
capacity and polyfunctionality of elicited immune responses
were significantly enhanced when PENNVAX DNA vaccines
were delivered using the CELLECTRA electroporation device
compared with delivery using a recombinant adenovirus
serotype 5 (Ad5) vector.
VGX is developing the PENNVAX HIV DNA vaccines PENNVAX-B
(targets Clade B) and PENNVAX-GP (targets Clades A, C and
D). The HIV Vaccine Trials Network (HVTN) is evaluating
PENNVAX-B as a prophylaxis for HIV infection; a phase I trial
of the vaccine (not using electroporation) in healthy
volunteers is under way. Researchers at the University of
Pennsylvania and Drexel University (USA) are conducting a
phase I trial of PENNVAX-B (not using electroporation) in the
treatment of HIV infection. Follow-up phase I trials of
PENNVAX-B using the CELLECTRA electroporation device are
planned. Preclinical studies of PENNVAX-GP, delivered using
CELLECTRA, are under way.
ondansetron
Stanford University clinical data (phase I) (drug
addiction)
Stanford University School of Medicine (USA) reported on 18
February 2009 data from its program to develop ondansetron
(ZOFRAN), a 5HT3 receptor antagonist, for the treatment of
opioid addiction. In a clinical trial in eight, healthy,
non-opioid-dependent subjects, a single, large dose of
morphine was given in one session. In another session (at
least one week later) ondansetron was given in combination
with morphine. Withdrawal symptoms were assessed using
questionnaires. Results indicated a significant reduction in
withdrawal signs when ondansetron was given before or
concurrently with morphine, compared with morphine
administration without ondansetron therapy. These data
reflect those of studies in mice in which ondansetron
demonstrated efficacy in treating morphine addiction
(reduction in jumping behavior and pain sensitivity).
Ondansetron is widely marketed by GlaxoSmithKline for the
prevention of chemotherapy-induced nausea and vomiting
and for post-operative nausea and vomiting.
AN 2728
Anacor clinical data (phase II) (psoriasis)
On 19 February 2009 Anacor reported results from a second
phase II trial of AN 2728, an oxaborole being developed for
the topical treatment of psoriasis. The randomized,
double-blind, placebo-controlled study involved 30 patients
who treated one of their psoriasis plaques with AN 2728 5%
ointment twice-daily; a matching psoriasis plaque on the
other side of the body was treated with vehicle ointment
alone. The trial met its objective of defining optimal duration
of treatment. AN 2728-treated plaques had a lower overall
target plaque severity score (OTPSS) in a significantly greater
proportion of patients compared with vehicle-treated
plaques; improvement was observed within two weeks of
therapy (p less than 0.001) and optimal responses were noted
at six to eight weeks (p less than 0.01). Individual signs of
psoriasis and OTPSS were significantly reduced. Complete
clearance of plaque was seen in 13% of treated plaques while
43% of plaques were clear or almost clear with a two-grade
improvement from baseline. AN 2728 ointment was well
tolerated and application site irritation was the most
18 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
common side effect. Anacor plans to begin a dose-ranging
phase IIb trial of AN 2728 during second half 2009.
AN 2728 is a boron-containing small molecule that inhibits
the phosphodiesterase IV enzyme, suppressing the release of
pro-inflammatory cytokines including TNF-alpha, IL-12 and
IL-23.
vaccine, NSCLC, Transgene
Transgene clinical data (phase II) (NSCLC)
Transgene reported on 17 February 2009 21-month median
follow-up data from a European phase IIb trial of its TG 4010
(MVA-MUC1-IL2) immunotherapy in 148 patients with
advanced nonsmall cell lung cancer (NSCLC). Half of the
patients received TG 4010 in combination with cisplatin and
gemcitabine; the remainder received chemotherapy alone.
Results confirmed a statistically significant greater median
survival in the TG 4010/cisplatin/gemcitabine group (17.1
months) compared with the chemotherapy alone group (11.3
months) in patients with normal activated natural killer cell
levels at baseline (approximately 75% of enrolled patients).
This patient sub-population was identified by Transgene’s
biomarker program, data from which provided evidence that
TG 4010 has a Th1-dependent pathway of activity in cancer
patients. The trial met its statistical primary endpoint of at
least a 40% six-month progression-free survival (PFS);
six-month PFS rate was 44% and 35% in the TG
4010/cisplatin/gemcitabine and chemotherapy alone
groups, respectively. Transgene expects to meet with the US
FDA and the EMEA second quarter 2009 regarding plans for a
phase III program in metastatic NSCLC.
TG 4010 comprises a recombinant vaccinia virus (based on
the Modified Virus Ankara (MVA)) that incorporates genes
encoding the MUC-1 tumor-associated antigen and
interleukin-2 (IL-2).
Genz 112638
Genzyme clinical data (phase II) (Gaucher's
disease)
Genzyme reported on 20 February 2009 further data from an
international phase II trial to assess the efficacy, safety and
pharmacokinetics of Genz 112638 (a glucosylceramide
analogue) oral capsules in the treatment of type I Gaucher’s
disease. The composite primary efficacy endpoint of a
clinically meaningful response in at least two of three
endpoints (improvements in spleen size, hemoglobin and
platelet levels) after 52 weeks of the trial period, was met.
Twenty-two (of 26 enrolled) patients completed at least 52
weeks of Genz 112638 therapy. At 12 months there were
mean decreases from baseline of 39% and 17% in spleen and
liver volumes, respectively. Mean increases in blood
hemoglobin levels and platelet counts were 1.62 g/dL and
40% from baseline, respectively. In the 20 patients treated
with chitotriosidase, levels of this enzyme were reduced by a
median of 51% from baseline. These data were comparable to
those observed following one year of imiglucerase
(CEREZYME) treatment. Genz 112638 demonstrated good
tolerability and a favorable safety profile. Of the adverse
events, 91% were unrelated to Genz 112638 therapy.
Drug-related adverse events were mild and transient; they
were experienced by a small number of patients early in
treatment and did not need medical intervention. Related
adverse events in the trial included infrequent abdominal
discomfort and diarrhea, and transient palpitations and
headache. A potential link between asymptomatic
non-sustained ventricular tachycardia and sub-quantifiable
levels of Genz 112638 was thought unlikely by investigators.
After the trial had completed, all 20 eligible patients
remained on Genz 112638 therapy; over 50% of patients have
completed more than two years of treatment. Patient
treatment is ongoing; longer-term efficacy data are being
collected. A phase III trial in untreated patients with type I
Gaucher’s disease and a phase III maintenance trial in which
patients who have achieved their therapeutic goals with
imiglucerase, are expected to start mid 2009.
NPL 2008
Neuropharm clinical data (phase III) (autism)
On 18 February 2009 Neuropharm reported preliminary
results from a US phase III trial (designated SOFIA) of NPL
2008 in 158 patients (aged five to 17 years) with autistic
disorder. Data showed that the CYBOCS-PDD measure of
repetitive behaviors was reduced after 14 weeks of treatment
with either NPL 2008 or placebo. However, the study did not
achieve its primary endpoint of a statistically significant
difference between the two groups. NPL 2008 was generally
well tolerated, and no serious adverse events were observed.
Full analysis of the primary and secondary data from the trial
is ongoing.
See R&Dfocus (Drug Up dates) for full prod uct de tails 19
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
NPL 2008, a formulation of the selective serotonin reuptake
inhibitor fluoxetine delivered through Catalent’s ZYDIS
tablet, is being developed for the potential treatment of
children and adolescents with autism. A rolling NDA for NPL
2008 was submitted to the US FDA in September 2008.
rosuvastatin
AstraZeneca clinical data (phase III)
(cardiovascular disease)
AstraZeneca reported on 19 February 2009 further results
from its phase III JUPITER (Justification for the Use of
statins in Primary prevention: an Intervention Trial
Evaluating Rosuvastatin) trial of rosuvastatin (CRESTOR).
Benefits in higher risk patients, including those older than
70, cigarette smokers, hypertensives, those with an elevated
Framingham risk score and those with a hsCRP
(high-sensitivity C-reactive protein level) at or above 5
mg/dL at baseline, were demonstrated. No increase in the
risk of hemorrhagic stroke was observed, and rosuvastatin
was well tolerated. Initial results, reported in November
2008, showed that treatment with 20 mg of the agent
reduced major cardiovascular events (the combined risk of
myocardial infarction, stroke, arterial revascularization,
hospitalization for unstable angina or death from CV causes)
by 44% compared with placebo (p less than 0.001) in subjects
with elevated hsCRP but low to normal cholesterol levels. The
combined risk of heart attack, stroke or CV death was reduced
by 47% (p less than 0.001), the risk of heart attack by 54% (p
less than 0.001), the risk of stroke by 48% (p=0.002), and
total mortality was reduced by 20% (p=0.02).
Rosuvastatin is available in numerous markets worldwide as a
treatment for hyperlipidemia. AstraZeneca expects to file a
regulatory submission including data from the phase III
JUPITER trial during first half 2009.
UDB
MAP Pharmaceuticals clinical data (phase III)
(asthma)
On 23 February 2009 MAP Pharmaceuticals reported results
from its randomized, double-blind, placebo-controlled phase
III trial of unit dose budesonide (UDB). During the trial, 360
asthmatic children were randomized to receive UDB 0.25 mg,
UDB 0.135 mg or placebo bid for 12 weeks. The co-primary
endpoints (asthma control as assessed by changes from
baseline in nightime and daytime composite symptom
scores) were not achieved for either of the tested doses when
compared with placebo. Drug-related serious adverse events
were not identified in an initial data review. A 52-week safety
trial, evaluating the long-term effects of the two doses, is
ongoing. MAP Pharmaceuticals, together with partner
AstraZeneca, will conduct additional analyses of the data to
evaluate how to progress the program.
UDB, a nebulized formulation of budesonide that utilizes
Elan’s NanoCrystal technology, is being developed as a
potential treatment for pediatric asthma.
ALKS 27
Alkermes begins phase IIa trial
On 18 February 2009 Alkermes announced that it has begun a
randomized, double-blind, crossover, placebo-controlled
phase IIa trial of ALKS 27 in approximately 24 patients with
chronic obstructive pulmonary disease (COPD). Patients will
be given single administrations of three doses of ALKS 27 and
placebo, each separated by a washout period. Efficacy will be
measured using FEV1 (improvement in pulmonary function).
Following the randomized, double-blind, placebo-controlled
portion of the study, all patients will receive ALKS 27 in
combination with formoterol inhalation powder to evaluate
the safety, tolerability and effects of the combination.
Top-line results from this study are expected during second
half 2009.
Alkermes’ ALKS 27 (developed with partner Indevus) is an
inhaled formulation of trospium chloride, a muscarinic
receptor antagonist that relaxes smooth muscle tissue, and is
delivered using Alkermes’ proprietary AIR pulmonary delivery
system.
ChimeriVax-Dengue
Sanofi Pasteur initiates phase IIb trial in Thailand
On 18 February 2009 Sanofi Pasteur announced the initiation
of a phase IIb trial of its recombinant dengue vaccine,
ChimeriVax-Dengue, in Thailand. The study will assess the
efficacy of the vaccine in the prevention of dengue fever in
children. The ChimeriVax-Dengue vaccine was developed
using the ChimeriVax technology licensed from St Louis
University (USA) and is directed against all four serotypes of
20 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
dengue virus. A phase IIa trial of the vaccine was initiated in
2005 in the USA.
riociguat
Bayer Schering receives UK ethical approval for two
phase III trials
Bayer Schering announced on 18 February 2009 that it has
been granted ethical approval to begin two UK phase III trials
of riociguat in patients with chronic thromboembolic
pulmonary hypertension (CTEPH) and pulmonary arterial
hypertension (PAH). The double-blind, randomized,
placebo-controlled CHEST-1 study will enroll 270 CTEPH
patients. Efficacy will be measured as change from baseline in
patients’ exercise capacity using the six-minute walking
distance test. Following 16 weeks of therapy, patients may
elect to enter into an open-label extension study (CHEST-2)
to evaluate long-term safety and efficacy. The second phase
III trial, PATENT-1, will be a randomized, double-blind,
placebo-controlled study in 460 PAH patients who are either
treatment-naive or are receiving an endothelin receptor
antagonist or prostacyclin analogue. The primary endpoint
will be the same as the CHEST-1 trial, and patients may elect
to enter into an open-label extension study (PATENT-2) at
the end of the PATENT-1 study. Initial results from both
studies are expected in 2011.
Riociguat is an oral stimulator of soluble guanylate cyclase.
MAb, VAP-1, BioTie Therapies
BioTie Therapies initiates rheumatoid arthritis trial
On 17 February 2009 BioTie Therapies announced the
initiation of a phase I trial of BTT 1023, its fully human
monoclonal antibody against VAP-1, in patients with
rheumatoid arthritis. The trial, which will be conducted in the
EU, will evaluate the safety and tolerability of the antibody in
up to 36 patients. Results are expected first half 2010. A
phase I trial in psoriasis patients is expected to initiate
during first quarter 2009.
Roche paid BioTie Therapies an option initiation fee of EUR5
million for an exclusive option right to an exclusive,
worldwide license agreement for the VAP-1 monoclonal
antibody, excluding Japan, Taiwan, Singapore, New Zealand
and Australia. This option will end upon the completion of
phase I, but may be extended by Roche to later development
points upon payment of additional fees.
Q 8003IR
QRxPharma initiates comparative US trial
QRxPharma announced on 19 February 2009 the initiation of
a clinical trial comparing the analgesic efficacy of Q 8003IR
(MoxDuoIR) with oxycodone plus acetaminophen (PERCOCET)
and iv morphine (patient controlled analgesia) in patients
with moderate-to-severe pain following total knee
replacement surgery. The trial is expected to enroll 45
patients at three sites in the USA. Patients will receive
treatment every 4 to 6 h for 48 h. Results from the trial will be
used to establish the optimal dose regimen for Q 8003IR, to
select an appropriate control group and in the design of a
pivotal phase III trial.
Q 8003IR is an immediate-release oral formulation of
morphine and oxycodone. A phase III trial of the product in
patients with moderate-to-severe pain after bunionectomy
was initiated in the USA in November 2007.
IPI 940
Infinity selects clinical development candidate
On 9 February 2009 Infinity reported that it has selected IPI
940, a fatty acid amide hydrolase (FAAH) inhibitor, as a
clinical development candidate for the potential treatment
of neuropathic pain.
vaccine, cat allergy, Alk-Abello
Alk-Abello evaluating cat allergen
Alk-Abello is developing an oral tablet formulation of a
recombinant cat allergen, as a vaccine against cat allergy.
Alk-Abello announced on 23 February 2009 that it has signed
an agreement with Novozymes under which the latter will
provide know-how and develop a microbial production
process to manufacture the allergen. Alk-Abello is
responsible for preclinical and clinical evaluation, and
commercialization of the vaccine.
See R&Dfocus (Drug Up dates) for full prod uct de tails 21
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
©2006 IMS Health Incorporated or its affiliates. All Rights Reserved.
Need quick access to pharmaceutical market reports?
The best way to know.
Ask IMS.IMS HEALTH brings you reliable, comprehensive national sales figures,
opinion, analysis and intelligence.
Understand the intricacies of pharma, generic and biotech companies, and markets from Argentina to the USA …
• the breakdown of sales data and growth rates• the complexities of pricing
• the potential of therapy areas• the analysis behind health economics
• the national structures of reimbursement
... all from IMS, online now.
Select what you want from a wide range of reports, view samples, tables of content and have peace of mind that what you purchase is exactly what you want by talking to one of our people before you buy.
IMS reports covering country markets, therapies, companies and pricing now online at www.imshealth.com/reportsonline
©2008 IMS Health Incorporated or its affiliates. All rights reserved.
Company Focus
Focus on Gene Signal
International
Founded in 2000, Gene Signal International (Lausanne,
Switzerland) is a biotechnology company specialized in the
discovery, development, and commercialization of
oligonucleotides, proteins and monoclonal antibodies for the
treatment of a range of conditions where angiogenesis plays
a determinant role. The company uses a patented gene
discovery platform GENE-MAAP (GENE Signal Multiple
Angiogeneis, Angiostatic Platform) which allows the
identification of genes involved in the angiogenic and
angiostatic process. Gene Signal International’s most
advanced compound, GS 101, has entered phase III trials for
the management of neovascularisation associated corneal
graft rejection.
GS 102
Gene Signal International partnering opportunity,
Worldwide
Gene Signal International informed R&D Focus that it is
developing the antisense oligonucleotide GS 102 as a
potential topical therapy for diabetic retinopathy. The agent
was discovered using Gene Signal’s proprietary scientific
platform GENE-MAAP (GENE Signal Multiple Angiogenesis,
Angioplastic Platform). Preclinical studies are ongoing in
Switzerland. The agent is available for partnering worldwide.
For further information on the partnering opportunities
available, contact:
Eric Viaud
CEO
Gene Signal International SA
Parc Scientifique EPFL
A 1015 Lausanne
Switzerland
Tel: +41 21 804 61 64
Fax: +41 21 804 61 65
Email: [email protected]
GS 101
Gene Signal International partnering opportunity,
Worldwide
On 20 February 2009 Gene Signal International informed R&D
Focus that GS 101 is available for partnering in the USA and
Japan for Orphan indications, and worldwide for non-Orphan
indications. GS 101, a topical antisense oligonucleotide
targeting the gene IRS-1, is being developed for the
treatment of various issues related to neovascularisation. GS
101, which was discovered using Gene Signal International’s
proprietary scientific platform GENE-MAAP (GENE Signal
Multiple Angiogenesis, Angioplastic Platform), reduces
corneal neovascularisation. The agent acts via inhibition of
IRS-1 and the modulation of selective transcriptional
activities related to VEGF and IL-1beta expression, while
avoiding complete VEGF suppression. In November 2008 Gene
Signal International initiated a phase III trial of GS 101 in
Germany, Switzerland and France for the prevention of
corneal graft reject ion assoc iated with corneal
neovascularisation. Phase I trials of GS 101 have been
conducted for the treatment of neovascular glaucoma,
retinopathy of prematur ity, age-related macular
degeneration (AMD) as well as in dermatology, for the
treatment of psoriasis and rosacea. The European
Commission has granted GS 101 Orphan designation for the
treatment of neovascular glaucoma, retinopathy of
prematurity and for the prevention of corneal graft rejection.
Gene Signal International plans to initiate phase II trials of
GS 101 in other ophthalmology indications (inhibition of
neovascularization in retina), such as diabetic retinopathy,
in second quarter 2009 in Switzerland, Germany, France and
Spain; a phase II trial of GS 101 for the treatment of psoriasis
and rosacea is planned for second quarter 2009 in Germany
and India.
For further information on the partnering opportunities
available, contact:
Eric Viaud
CEO
Gene Signal International SA
Parc Scientifique EPFL
A 1015 Lausanne
Switzerland
Tel: +41 21 804 61 64
Fax: +41 21 804 61 65
Email: [email protected]
See R&Dfocus (Drug Up dates) for full prod uct de tails 23
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
IMS HEALTH R&D FO CUS drugnews 2 March 2009
GS 156
Gene Signal International partnering opportunity,
Worldwide
Gene Signal International is developing the peptide GS 156
for potential use as a wound healing agent. Preclinical
studies are ongoing in Switzerland. GS 156 is available for
partnering worldwide.
For further information on the partnering opportunities
available, contact:
Eric Viaud
CEO
Gene Signal International SA
Parc Scientifique EPFL
A 1015 Lausanne
Switzerland
Tel: +41 21 804 61 64
Fax: +41 21 804 61 65
Email: [email protected]
GS 168
Gene Signal International partnering opportunity,
Worldwide
Gene Signal International is conducting proof-of-concept
preclinical investigations of the protein GS 168 for the
potential treatment of renal cancer. GS 168 is available for
partnering worldwide.
For further information on the partnering opportunities
available, contact:
Eric Viaud
CEO
Gene Signal International SA
Parc Scientifique EPFL
A 1015 Lausanne
Switzerland
Tel: +41 21 804 61 64
Fax: +41 21 804 61 65
Email: [email protected]
GS 497c
Gene Signal International partnering opportunity,
Worldwide
Gene Signal International is developing the protein GS 497c
for the potential treatment of lung cancer. Proof-of-concept
preclinical studies are ongoing in Switzerland. GS 497c is
available for partnering worldwide.
For further information on the partnering opportunities
available, contact:
Eric Viaud
CEO
Gene Signal International SA
Parc Scientifique EPFL
A 1015 Lausanne
Switzerland
Tel: +41 21 804 61 64
Fax: +41 21 804 61 65
Email: [email protected]
GS 326
Gene Signal International partnering opportunity,
Worldwide
Proof-of-concept preclinical studies of GS 326 have been
initiated. The agent is being developed by Gene Signal
International for the potential treatment of pancreatic
cancer. The agent is available for partnering worldwide.
For further information on the partnering opportunities
available, contact:
Eric Viaud
CEO
Gene Signal International SA
Parc Scientifique EPFL
A 1015 Lausanne
Switzerland
Tel: +41 21 804 61 64
Fax: +41 21 804 61 65
Email: [email protected]
24 See IMS LifeCycle, R&Dfocus for full prod uct de tails
© 2009 IMS Health In cor po rated or its af fil i ates. All rights re served.
2 March 2009 R&D FO CUS drugnews IMS HEALTH
NEWLY REPORTED DRUGS IN R&D FOCUS
Company Product Therapeutic Class Indication Phase
Alk-Abello vaccine, cat allergy, Alk-Abello J7C; V1A allergy Discovery
DepoMeddrug delivery system, levodopa + carbidopa,
DepoMedV7A; N4A Parkinson disease Phase I
Gene Signal
InternationalGS 102 S1X diabetic retinopathy Preclinical
Gene Signal
InternationalGS 101 S1P; D5A; S1E; S1X psoriasis; AMD; glaucoma; retinopathy Phase III
Gene Signal
InternationalGS 156 D3A9 wound Preclinical
Gene Signal
InternationalGS 168 L1X9 renal cancer Preclinical
Gene Signal
InternationalGS 326 L1X9 pancreatic cancer Preclinical
Gene Signal
InternationalGS 497c L1X9 lung cancer Preclinical
German Cancer
Research Center
fusion polypeptides, German Cancer Research
CenterL1X9 cancer Discovery
German Cancer
Research Center
NS1 protein/VP1 capsid protein, German Cancer
Research CenterL1X9 glioblastoma Preclinical
GTx GTx 758 L2B9 prostate cancer Phase I
HUYAdrug discovery, HUYA/Beijing Institute of Materia
MedicaV3X all other therapeutics Discovery
Lilly ion channel modulators, NeuroSearch/Lilly N7X neurological Discovery
Nostrum caerulomycin A L4A transplant rejection; inflammation Preclinical
Novartis aliskiren + amlodipine + hydrochlorothiazide C8A; C3A3; C9X hypertension Phase III
Shire HGT 2610 V3X enzyme deficiency Preclinical
See IMS LifeCycle, R
&Dfo
cus fo
r full p
rod uct d
e tails25
© 2
009 IM
S Health
In co
r po rated
or its af fil i ates. A
ll rights re served
.
2 M
arch 2
009
R&
D FO
CUS d
rugnew
s IM
S HEA
LTH
NEWLY REPORTED DRUGS IN R&D FOCUS
Company Product Therapeutic Class Indication Phase
Stockbridge
Pharmaceuticalscancer therapy, Stockbridge Pharmaceuticals L1X9 cancer Preclinical
VGX vaccine, gene-based, HIV Clade B, VGX J7A9 HIV infection Phase I
VGX vaccine, gene-based, HIV Clades A, C and D, VGX J7A9 HIV infection Preclinical
26
See IMS LifeCycle, R
&Dfo
cus fo
r full p
rod uct d
e tails© 2
009 IM
S Health
In co
r po rated
or its af fil i ates. A
ll rights re served
.
2 M
arch 2
009
R&
D FO
CUS d
rugnew
s IM
S HEA
LTH
PRODUCT PHASE CHANGES REPORTED IN R&D FOCUS
Company Product Therapeutic Class* Indication New PhaseRegion of Phase
ChangeHighest Phase
AVEO AV 951 L1X4 breast cancer Phase II USA; Europe Phase II
Bialeslicarbazepine
acetateN3A epilepsy Recommended EU Pre-registration
Cubist CB 182804 J1X9 bacterial infection Phase I USA Phase I
DepoMed DM 1992 V7A; N4A Parkinson disease Phase I Russia Phase I
Enanta ABT 450 J5B1 hepatitis C Phase I USA Phase I
Endocyte EC 145 L1X9 ovarian cancer Phase IIUSA; Europe;
CanadaPhase II
Ferring degarelix L2B9 prostate cancer Registered EU Registered
Fresenius catumaxomab L1X3 cancer Recommended EU Pre-registration
GeoVaxvaccine, gene-based,
HIV infection, GeoVaxJ5C9 HIV infection Phase II USA; Peru Phase II
GlaxoSmithKline albiglutide A10S diabetes Phase III USA Phase III
GTx GTx 758 L2B9 prostate cancer Phase I USA Phase I
Lilly prasugrel B1C2 acute coronary syndrome Registered EU Registered
Lux Biosciences LX 214 S1X; V7A eye disease Phase I USA Phase I
Merck & Co
nicotinic acid
receptor agonist,
Merck & Co/Arena
C10A9 atherosclerosis Phase II USA Phase II
Nektar NKTR 105 L1C solid tumor Phase I USA Phase I
See IMS LifeCycle, R
&Dfo
cus fo
r full p
rod uct d
e tails27
© 2
009 IM
S Health
In co
r po rated
or its af fil i ates. A
ll rights re served
.
2 M
arch 2
009
R&
D FO
CUS d
rugnew
s IM
S HEA
LTH
PRODUCT PHASE CHANGES REPORTED IN R&D FOCUS
Company Product Therapeutic Class* Indication New PhaseRegion of Phase
ChangeHighest Phase
Novartisaliskiren + amlodipine
+ hydrochlorothiazideC8A; C3A3; C9X hypertension Phase III
USA; Belgium;
Slovak
Republic;
Egypt;
Germany;
Poland; Spain;
Turkey
Phase III
ProStrakan
drug delivery system,
sublingual fentanyl
citrate, Orexo
N2A; V7A pain Marketed UK; Germany Marketed
Sanofi Pasteur
vaccine, Clostridium
difficile, Sanofi
Pasteur
J7A9 bacterial infection Phase II UK Phase II
Seattle Genetics SGN 35 L1X3 Hodgkin lymphoma Phase IIUSA; Canada;
EuropePhase II
Shire lanthanum carbonate V3G hyperphosphatemia Registered Japan Marketed
Takeda KAPIDEX A2B2; V7A GERD Marketed USA Marketed
ThromboGenics TB 402 B1X thrombosis Phase II Europe Phase II
Vion laromustine L1A AML Pre-registration USA Pre-registration
Wyeth bazedoxifene G3J osteoporosis Recommended EU Pre-registration
* A change in phase may not apply to all therapeutic classes and indications
28
See IMS LifeCycle, R
&Dfo
cus fo
r full p
rod uct d
e tails© 2
009 IM
S Health
In co
r po rated
or its af fil i ates. A
ll rights re served
.
2 M
arch 2
009
R&
D FO
CUS d
rugnew
s IM
S HEA
LTH