Accepted Manuscript Investing in new therapies for ocular surface disease Gary D. Novack, PhD PII:
S1542-0124(15)00046-4
DOI:
10.1016/j.jtos.2015.04.001
Reference:
JTOS 128
To appear in:
Ocular Surface
Received Date: 5 April 2015 Revised Date:
14 April 2015
Accepted Date: 14 April 2015
Please cite this article as: Novack GD, Investing in new therapies for ocular surface disease, Ocular Surface (2015), doi: 10.1016/j.jtos.2015.04.001. This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain.
ACCEPTED MANUSCRIPT 1 SECTION: Pipeline, Gary D. Novack, PhD, Section Editor TITLE: Investing in new therapies for ocular surface disease BYLINE: GARY D. NOVACK, PHD
RI PT
FOOTNOTES From PharmaLogic Development, Inc., San Rafael CA.
M AN U
SC
Disclosure: Gary D. Novack, PhD consults with numerous pharmaceutical firms.
The Wall Street Journal recently reviewed venture capital funding of health care companies by organ system over the past 15 years. The largest recipient of funding has been “...the eyes and ears, as investors have poured money into treatments for diseases causing blindness, hearing loss and other diseases affecting an aging population. At the same time, venture capital is pulling back from two historical leaders, heart and orthopedic conditions, in part because of difficulties in bringing medical devices for those ailments to market.” In 2013 and 2014, $1.3 billion was invested in ophthalmic
TE D
opportunities in over 50 companies, which is comparable to that invested in cardiovascular and neurology firms (http://graphics.wsj.com/venture-capital-and-the-human-body).1 Some years ago in this journal, I stated “…In capitalistic societies, the cost of drug development
EP
is largely borne by the private rather than governmental sector. Thus, investors must believe that there is a chance of reward for investment to balance the risks, extensive capital, and long drug development process.”2 Thus, as ophthalmic scientists, researchers and clinicians, we should find this continued
AC C
interest in ophthalmology promising.
To be fair, government is becoming more involved in the development of drugs (as contrasted
with basic science and discovery). In the U.S., the National Institutes of Health created the National Center for Advancing Translational Sciences (NCATS). The charter of this center is to address “…scientific and technical challenges to reduce, remove or bypass bottlenecks in the development of new treatments and tests that will ultimately improve human health. The Center aims to make translational science more efficient, less expensive and less risky.” The director of NCATS, Christopher P. Austin, MD, has previous experience in the pharmaceutical industry (http://www.ncats.nih.gov). The NIH also
ACCEPTED MANUSCRIPT 2 funds Small Business Innovation Research (SBIR) grants, giving small firms funds for initial development. In Canada, the Canadian Institutes of Health Research (CIHR) has nine signature initiatives, which support transformative research to improve health, health care and health system outcomes (e.g.,
RI PT
inflammation in chronic diseases, http://www.cihr-irsc.gc.ca). The CIHR funds Proof of Principle (POP) to support projects to advance discoveries/inventions toward commercializable technologies, with a view toward attracting new investment and creating new science-based businesses. Phase 1 is up to 12 months of support. Phase 2 is a co-investment with a non-academic investor. The end product is not required to
SC
generate revenue, but there must be a demonstrated market opportunity for the product. There are other Canadian funding agencies, including the Quebec Consortium for Drug Discovery (CQDM,
M AN U
http://www.cqdm.org).
In the U.K, the National Institute of Health Research (NIHR) is funded through the Department of Health to improve the health and wealth of the U.K. through research (http://www.nihr.ac.uk). The NIHR is funding several translational efforts, including dementia and rare diseases (http://www.nihr.ac.uk/about/nihr-infrastructure.htm).
Other efforts in the U.K. include the newly formed Francis Crick Institute, a consortium of six
TE D
U.K. organizations: the Medical Research Council, Cancer Research U.K., the Wellcome Trust, University College London, Imperial College London, and King's College London. It will be an interdisciplinary medical research institute. Its work will enhance understanding of why disease develops and discovery of new ways to treat, diagnose, and prevent illnesses such as cancer, heart disease, and
EP
stroke, infections, and neurodegenerative diseases (http://www.crick.ac.uk). At Oxford University, Chas Bountra, PhD, formerly Vice President and Head of Biology at GlaxoSmithKline, is creating a multidisciplinary institute wherein all discoveries will be generally available without concern for proprietary
AC C
ownership (http://www.ndm.ox.ac.uk/principal-investigators/researcher/chas-bountra). More generally, pre-competitive research is a growing strategy for efficiency in drug discovery.3 Its extension into proprietary development is yet to be seen, especially given that private investment typically requires a period of exclusivity.
There is continuing debate about how much of government funding research actually contributes
to the development and commercial success of a product, ranging from the classic economic analysis by Professor John Jewkes4 to the more popular press evaluation by Merrill Goozner.5,6 The methods of calculating the cost of pharmaceutical research and development,7 the actual costs,8 drug prices around the world, and who should share in the risks and rewards of drug development are a matter of great debate
ACCEPTED MANUSCRIPT 3 – and beyond the scope of this column. Academia also contributes to post-approval efforts, including work such as post-approval drug safety surveillance.9 Again, we should be pleased that financiers believed that investment in ophthalmic companies might provide a good return. Implicit in this investment is the belief that payers will continue to pay a
RI PT
premium price for ophthalmic therapy. It is further implicit that patients put a high value on vision. Of course, the interests of ophthalmic companies range from anterior to posterior ophthalmic disease. Much of the ophthalmic funding has gone to sight-threatening retinal diseases, such as neovascular macular degeneration. Thus, it behooves us who are interested in ocular surface to continue to discuss the impact
SC
of ocular surface disease on the quality of life10 and to stress that a better understanding of the basic science of ocular surface in health and disease will lead to novel therapeutics in an area of great unmet
M AN U
need. References 1.
Gormley B. Where the money goes: Body part by body part. Wall St J, R4. 9-15-2014
2.
Novack GD. Pipeline: What is a new drug? Ocul Surf 2008;6:143-6
4. Norton, 1968
TE D
3. Wagner JA, Prince M, Wright EC, et al. The Biomarkers Consortium: practice and pitfalls of open-source precompetitive collaboration. Clin Pharmacol Ther 2010;87:539-42 Jewkes J, Sawers D, Stillerman R. The Sources of Invention, 2nd ed. New York, NY, WW
EP
5. Goozner M. The $800 Million Pill: The Truth behind the Cost of New Drugs. Berkeley, CA, University of California, 2004 6. Moore JP. The $800 million pill: The truth behind the cost of new drugs (book review). J Clin Invest 2004;114:1182 Vagelos PR. Are prescription drug prices high? Science 1991;252:1080-4
AC C
7.
8. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs. J Health Econ 2003;22:151-85 9. Hennessy S, Strom BL. Improving postapproval drug safety surveillance: getting better information sooner. Annu Rev Pharmacol Toxicol (in press) 10. Uchino M, Uchino Y, Dogru M, et al. Dry eye disease and work productivity loss in visual display users: the Osaka study. Am J Ophthalmol 2014;157:294-300
ACCEPTED MANUSCRIPT 4
Ophthalmic Products Related to the Ocular Surface •
RI PT
[SUBHEAD: caps and lower case] News from Pharmaceutical and Medical Device Companies
AmorChem licensed from the University of Waterloo a mucoadhesive nanotechnology platform
•
SC
for ophthalmic drug delivery for the treatment of dry eye (February 2015).
Herantus completed enrollment in a Phase 2 study of their drug, cis-UCA eye drops, for the
•
M AN U
treatment of dry eye (March 2015).
Santen received EMA approval for Ikervis® (cyclosporine 1%) for the treatment of severe
keratitis in adult patients with dry eye disease (Jan 2015). •
Shire submitted a New Drug Application to the U.S. Food and Drug Administration (FDA) for
lifitegrast for the treatment of dry eye. They have received a priority review designation (March and April
TE D
2015).
Ophthalmic Products Not Related to the Ocular Surface Aerie completed enrollment in its second of two Phase 3 studies of AR-13324 for the treatment
EP
•
of glaucoma (March 2015). They also announced additional pharmacological effects of AR-13324 in
•
AC C
preclinical models (February 2015).
Alcon received FDA approval for Pazeo™ (olopatadine hydrochloride ophthalmic solution
formulated with a cyclodextrin) for the treatment of ocular itching associated with allergic conjunctivitis (February 2015). •
Allergan received a positive recommendation from the U.K NICE for Ozurdex® (dexamethasone
intravitreal implant) for the treatment of diabetic macular edema (March 2015). •
Applied Genetic Technologies Corporation filed an Investigational New Drug (IND)
application with the FDA to conduct a Phase 1/2 clinical trial of the company's adeno-associated virus gene therapy product candidate for the treatment of X-linked retinoschisis (March 2015).
ACCEPTED MANUSCRIPT 5 •
AqueSys, Inc. received a Class 3 Medical Device License for the XEN Gel Stent, a minimally
invasive glaucoma surgical implant from Health Canada (February 2015). The firm also completed enrollment in a U.S. trial (March 2015). •
Avedro received a positive recommendation from an FDA panel for Photrexa Viscous™
RI PT
(riboflavin ophthalmic solution with 20% dextran)/Photrexa™ (riboflavin ophthalmic solution with 0% dextran) with the KXL system (UVA light) for corneal collagen crosslinking for treatment of progressive keratoconus and corneal ectasia following refractive surgery (February 2015). FDA subsequently decided that additional clinical data would be required for approval (April 2015).
Bayer received a positive recommendation from the U.K National Institute for Health and Care
SC
•
Excellence (NICE) for Eylea® (aflibercept solution for injection) for the treatment of patients with visual
•
M AN U
impairment due to macular edema (February 2015).
Clearside initiated a Phase 2 clinical trial of their suprachoroidal space injection of triamcinolone
acetonide for the treatment of macular edema associated with retinal vein occlusion (March 2015). •
Envisia Therapeutics initiated a Phase 2a clinical trial to investigate the safety and tolerability of
ENV515 (a travoprost drug delivery system) in patients with glaucoma (January 2015). Genentech received approval for Lucentis® (ranibizumab) for the treatment of diabetic
TE D
•
retinopathy in patients with diabetic macular edema (February 2015). GlaxoSmithKline announced that it has research programs in ocular drug delivery (March 2015).
•
Graybug, a drug delivery firm including technology from the Wilmer Eye Institute, will be
EP
•
developing GB-012 for the treatment of wet age-related macular degeneration (AMD, March 2015). Hospira entered an exclusive collaboration with Pfenex Inc. on a biosimilar version of
AC C
•
ranibizumab (Lucentis®) for intravitreal use for ophthalmic indications (February 2015). •
Icon Bioscience completed a Phase 3 study of IBI-10090, its corticosteroid intraocular drug
delivery system for the treatment of post-operative inflammation (April 2015). •
Kala Pharmaceuticals announced results from a Phase 3 clinical trial of KPI-121, its nanoparticle
loteprednol etabonate MPP product candidate, for the treatment of inflammation and pain after cataract surgery (April 2015). •
Lupin received approval for a generic bimatoprost 0.003% in the U.S. (February 2015).
ACCEPTED MANUSCRIPT 6 •
Neurotech submitted an IND for the treatment of wet AMD with NCT-503 encapsulated cell
therapy in a Phase 2 study (March 2015). •
Nicox signed a licensing agreement with InSite Vision for AzaSite® (1% azithromycin),
BromSite™ (0.075% bromfenac) and AzaSite Xtra™ (2% azithromycin) in Europe, Middle East, and
•
RI PT
Africa (February 2015).
Ocular Therapeutix completed two Phase 3 studies of OTX-DP, a punctal plug containing
dexamethasone, for the treatment of post-operative inflammation (April 2015).
pSivida completed enrollment in a Phase 3 study for the treatment of posterior uveitis with
SC
•
Medidur™ (fluocinolone acetonide intravitreal implant, March 2015).
Regeneron received FDA approval for Eylea® (aflibercept) for the treatment of diabetic
M AN U
•
retinopathy (March 2015). •
Santen announced that the EMA accepted its filing for the use of intravitreal sirolimus for the
treatment of noninfectious uveitis of the posterior segment (March 2015).
Second Sight is evaluating a visual cortical prosthesis (Orion I) in an animal study (April 2015).
•
Spark Therapeutics initiated enrollment of a Phase 1/2 clinical trial of its gene therapy, SPK‐
TE D
•
CHM, for patients with choroideremia (January 2015). •
Thrombogenics received a positive opinion from the European Medicines Agency (EMA) for a
AC C
Brazil (March 2015).
EP
ready-diluted formulation of Jetrea® (ocriplasmin). The firm also received approval for this product in
News from the FDA and Other Regulatory Agencies •
Otsuka is suing the FDA over its broadening of the indications for Abilify® (aripiprazole), an
antipsychotic, as it had implications for generic competition (March 2015). •
Margaret Hamburg, MD, has retired as Commissioner of the FDA. Stephen Ostroff, MD, is the
Acting Commissioner (April 2015). Miscellaneous News Notes
ACCEPTED MANUSCRIPT 7 •
International Health Partners, a charity that distributes donated medicines to disaster zones,
celebrated 10 years of operation. A substantial portion of these donations, unfortunately, expire prior to being delivered to patients (March 2015). •
Mapp Biopharmaceuticals, in combination with the U.S. National Institute of Allergy and
RI PT
Infectious Diseases and the Liberian government, will evaluate the experimental drug cocktail ZMapp as a treatment for Ebola virus disease (February 2015). •
Merck, in combination with NewLink Genetics, will commence trials in Guinea for a
•
SC
vaccination for the Ebola virus (March 2015).
Novartis received FDA approval for Zarxio®, a biosimilar of Amgen's Neupogen®, making it
•
M AN U
the first biosimilar medication approved in the U.S. (March 2015).
San Francisco is considering an ordinance to require pharmaceutical manufacturers to institute a
AC C
EP
TE D
drug take-back program (March 2015).
S1542-0124(15)00046-4
DOI:
10.1016/j.jtos.2015.04.001
Reference:
JTOS 128
To appear in:
Ocular Surface
Received Date: 5 April 2015 Revised Date:
14 April 2015
Accepted Date: 14 April 2015
Please cite this article as: Novack GD, Investing in new therapies for ocular surface disease, Ocular Surface (2015), doi: 10.1016/j.jtos.2015.04.001. This is a PDF file of an unedited manuscript that has been accepted for publication. As a service to our customers we are providing this early version of the manuscript. The manuscript will undergo copyediting, typesetting, and review of the resulting proof before it is published in its final form. Please note that during the production process errors may be discovered which could affect the content, and all legal disclaimers that apply to the journal pertain.
ACCEPTED MANUSCRIPT 1 SECTION: Pipeline, Gary D. Novack, PhD, Section Editor TITLE: Investing in new therapies for ocular surface disease BYLINE: GARY D. NOVACK, PHD
RI PT
FOOTNOTES From PharmaLogic Development, Inc., San Rafael CA.
M AN U
SC
Disclosure: Gary D. Novack, PhD consults with numerous pharmaceutical firms.
The Wall Street Journal recently reviewed venture capital funding of health care companies by organ system over the past 15 years. The largest recipient of funding has been “...the eyes and ears, as investors have poured money into treatments for diseases causing blindness, hearing loss and other diseases affecting an aging population. At the same time, venture capital is pulling back from two historical leaders, heart and orthopedic conditions, in part because of difficulties in bringing medical devices for those ailments to market.” In 2013 and 2014, $1.3 billion was invested in ophthalmic
TE D
opportunities in over 50 companies, which is comparable to that invested in cardiovascular and neurology firms (http://graphics.wsj.com/venture-capital-and-the-human-body).1 Some years ago in this journal, I stated “…In capitalistic societies, the cost of drug development
EP
is largely borne by the private rather than governmental sector. Thus, investors must believe that there is a chance of reward for investment to balance the risks, extensive capital, and long drug development process.”2 Thus, as ophthalmic scientists, researchers and clinicians, we should find this continued
AC C
interest in ophthalmology promising.
To be fair, government is becoming more involved in the development of drugs (as contrasted
with basic science and discovery). In the U.S., the National Institutes of Health created the National Center for Advancing Translational Sciences (NCATS). The charter of this center is to address “…scientific and technical challenges to reduce, remove or bypass bottlenecks in the development of new treatments and tests that will ultimately improve human health. The Center aims to make translational science more efficient, less expensive and less risky.” The director of NCATS, Christopher P. Austin, MD, has previous experience in the pharmaceutical industry (http://www.ncats.nih.gov). The NIH also
ACCEPTED MANUSCRIPT 2 funds Small Business Innovation Research (SBIR) grants, giving small firms funds for initial development. In Canada, the Canadian Institutes of Health Research (CIHR) has nine signature initiatives, which support transformative research to improve health, health care and health system outcomes (e.g.,
RI PT
inflammation in chronic diseases, http://www.cihr-irsc.gc.ca). The CIHR funds Proof of Principle (POP) to support projects to advance discoveries/inventions toward commercializable technologies, with a view toward attracting new investment and creating new science-based businesses. Phase 1 is up to 12 months of support. Phase 2 is a co-investment with a non-academic investor. The end product is not required to
SC
generate revenue, but there must be a demonstrated market opportunity for the product. There are other Canadian funding agencies, including the Quebec Consortium for Drug Discovery (CQDM,
M AN U
http://www.cqdm.org).
In the U.K, the National Institute of Health Research (NIHR) is funded through the Department of Health to improve the health and wealth of the U.K. through research (http://www.nihr.ac.uk). The NIHR is funding several translational efforts, including dementia and rare diseases (http://www.nihr.ac.uk/about/nihr-infrastructure.htm).
Other efforts in the U.K. include the newly formed Francis Crick Institute, a consortium of six
TE D
U.K. organizations: the Medical Research Council, Cancer Research U.K., the Wellcome Trust, University College London, Imperial College London, and King's College London. It will be an interdisciplinary medical research institute. Its work will enhance understanding of why disease develops and discovery of new ways to treat, diagnose, and prevent illnesses such as cancer, heart disease, and
EP
stroke, infections, and neurodegenerative diseases (http://www.crick.ac.uk). At Oxford University, Chas Bountra, PhD, formerly Vice President and Head of Biology at GlaxoSmithKline, is creating a multidisciplinary institute wherein all discoveries will be generally available without concern for proprietary
AC C
ownership (http://www.ndm.ox.ac.uk/principal-investigators/researcher/chas-bountra). More generally, pre-competitive research is a growing strategy for efficiency in drug discovery.3 Its extension into proprietary development is yet to be seen, especially given that private investment typically requires a period of exclusivity.
There is continuing debate about how much of government funding research actually contributes
to the development and commercial success of a product, ranging from the classic economic analysis by Professor John Jewkes4 to the more popular press evaluation by Merrill Goozner.5,6 The methods of calculating the cost of pharmaceutical research and development,7 the actual costs,8 drug prices around the world, and who should share in the risks and rewards of drug development are a matter of great debate
ACCEPTED MANUSCRIPT 3 – and beyond the scope of this column. Academia also contributes to post-approval efforts, including work such as post-approval drug safety surveillance.9 Again, we should be pleased that financiers believed that investment in ophthalmic companies might provide a good return. Implicit in this investment is the belief that payers will continue to pay a
RI PT
premium price for ophthalmic therapy. It is further implicit that patients put a high value on vision. Of course, the interests of ophthalmic companies range from anterior to posterior ophthalmic disease. Much of the ophthalmic funding has gone to sight-threatening retinal diseases, such as neovascular macular degeneration. Thus, it behooves us who are interested in ocular surface to continue to discuss the impact
SC
of ocular surface disease on the quality of life10 and to stress that a better understanding of the basic science of ocular surface in health and disease will lead to novel therapeutics in an area of great unmet
M AN U
need. References 1.
Gormley B. Where the money goes: Body part by body part. Wall St J, R4. 9-15-2014
2.
Novack GD. Pipeline: What is a new drug? Ocul Surf 2008;6:143-6
4. Norton, 1968
TE D
3. Wagner JA, Prince M, Wright EC, et al. The Biomarkers Consortium: practice and pitfalls of open-source precompetitive collaboration. Clin Pharmacol Ther 2010;87:539-42 Jewkes J, Sawers D, Stillerman R. The Sources of Invention, 2nd ed. New York, NY, WW
EP
5. Goozner M. The $800 Million Pill: The Truth behind the Cost of New Drugs. Berkeley, CA, University of California, 2004 6. Moore JP. The $800 million pill: The truth behind the cost of new drugs (book review). J Clin Invest 2004;114:1182 Vagelos PR. Are prescription drug prices high? Science 1991;252:1080-4
AC C
7.
8. DiMasi JA, Hansen RW, Grabowski HG. The price of innovation: new estimates of drug development costs. J Health Econ 2003;22:151-85 9. Hennessy S, Strom BL. Improving postapproval drug safety surveillance: getting better information sooner. Annu Rev Pharmacol Toxicol (in press) 10. Uchino M, Uchino Y, Dogru M, et al. Dry eye disease and work productivity loss in visual display users: the Osaka study. Am J Ophthalmol 2014;157:294-300
ACCEPTED MANUSCRIPT 4
Ophthalmic Products Related to the Ocular Surface •
RI PT
[SUBHEAD: caps and lower case] News from Pharmaceutical and Medical Device Companies
AmorChem licensed from the University of Waterloo a mucoadhesive nanotechnology platform
•
SC
for ophthalmic drug delivery for the treatment of dry eye (February 2015).
Herantus completed enrollment in a Phase 2 study of their drug, cis-UCA eye drops, for the
•
M AN U
treatment of dry eye (March 2015).
Santen received EMA approval for Ikervis® (cyclosporine 1%) for the treatment of severe
keratitis in adult patients with dry eye disease (Jan 2015). •
Shire submitted a New Drug Application to the U.S. Food and Drug Administration (FDA) for
lifitegrast for the treatment of dry eye. They have received a priority review designation (March and April
TE D
2015).
Ophthalmic Products Not Related to the Ocular Surface Aerie completed enrollment in its second of two Phase 3 studies of AR-13324 for the treatment
EP
•
of glaucoma (March 2015). They also announced additional pharmacological effects of AR-13324 in
•
AC C
preclinical models (February 2015).
Alcon received FDA approval for Pazeo™ (olopatadine hydrochloride ophthalmic solution
formulated with a cyclodextrin) for the treatment of ocular itching associated with allergic conjunctivitis (February 2015). •
Allergan received a positive recommendation from the U.K NICE for Ozurdex® (dexamethasone
intravitreal implant) for the treatment of diabetic macular edema (March 2015). •
Applied Genetic Technologies Corporation filed an Investigational New Drug (IND)
application with the FDA to conduct a Phase 1/2 clinical trial of the company's adeno-associated virus gene therapy product candidate for the treatment of X-linked retinoschisis (March 2015).
ACCEPTED MANUSCRIPT 5 •
AqueSys, Inc. received a Class 3 Medical Device License for the XEN Gel Stent, a minimally
invasive glaucoma surgical implant from Health Canada (February 2015). The firm also completed enrollment in a U.S. trial (March 2015). •
Avedro received a positive recommendation from an FDA panel for Photrexa Viscous™
RI PT
(riboflavin ophthalmic solution with 20% dextran)/Photrexa™ (riboflavin ophthalmic solution with 0% dextran) with the KXL system (UVA light) for corneal collagen crosslinking for treatment of progressive keratoconus and corneal ectasia following refractive surgery (February 2015). FDA subsequently decided that additional clinical data would be required for approval (April 2015).
Bayer received a positive recommendation from the U.K National Institute for Health and Care
SC
•
Excellence (NICE) for Eylea® (aflibercept solution for injection) for the treatment of patients with visual
•
M AN U
impairment due to macular edema (February 2015).
Clearside initiated a Phase 2 clinical trial of their suprachoroidal space injection of triamcinolone
acetonide for the treatment of macular edema associated with retinal vein occlusion (March 2015). •
Envisia Therapeutics initiated a Phase 2a clinical trial to investigate the safety and tolerability of
ENV515 (a travoprost drug delivery system) in patients with glaucoma (January 2015). Genentech received approval for Lucentis® (ranibizumab) for the treatment of diabetic
TE D
•
retinopathy in patients with diabetic macular edema (February 2015). GlaxoSmithKline announced that it has research programs in ocular drug delivery (March 2015).
•
Graybug, a drug delivery firm including technology from the Wilmer Eye Institute, will be
EP
•
developing GB-012 for the treatment of wet age-related macular degeneration (AMD, March 2015). Hospira entered an exclusive collaboration with Pfenex Inc. on a biosimilar version of
AC C
•
ranibizumab (Lucentis®) for intravitreal use for ophthalmic indications (February 2015). •
Icon Bioscience completed a Phase 3 study of IBI-10090, its corticosteroid intraocular drug
delivery system for the treatment of post-operative inflammation (April 2015). •
Kala Pharmaceuticals announced results from a Phase 3 clinical trial of KPI-121, its nanoparticle
loteprednol etabonate MPP product candidate, for the treatment of inflammation and pain after cataract surgery (April 2015). •
Lupin received approval for a generic bimatoprost 0.003% in the U.S. (February 2015).
ACCEPTED MANUSCRIPT 6 •
Neurotech submitted an IND for the treatment of wet AMD with NCT-503 encapsulated cell
therapy in a Phase 2 study (March 2015). •
Nicox signed a licensing agreement with InSite Vision for AzaSite® (1% azithromycin),
BromSite™ (0.075% bromfenac) and AzaSite Xtra™ (2% azithromycin) in Europe, Middle East, and
•
RI PT
Africa (February 2015).
Ocular Therapeutix completed two Phase 3 studies of OTX-DP, a punctal plug containing
dexamethasone, for the treatment of post-operative inflammation (April 2015).
pSivida completed enrollment in a Phase 3 study for the treatment of posterior uveitis with
SC
•
Medidur™ (fluocinolone acetonide intravitreal implant, March 2015).
Regeneron received FDA approval for Eylea® (aflibercept) for the treatment of diabetic
M AN U
•
retinopathy (March 2015). •
Santen announced that the EMA accepted its filing for the use of intravitreal sirolimus for the
treatment of noninfectious uveitis of the posterior segment (March 2015).
Second Sight is evaluating a visual cortical prosthesis (Orion I) in an animal study (April 2015).
•
Spark Therapeutics initiated enrollment of a Phase 1/2 clinical trial of its gene therapy, SPK‐
TE D
•
CHM, for patients with choroideremia (January 2015). •
Thrombogenics received a positive opinion from the European Medicines Agency (EMA) for a
AC C
Brazil (March 2015).
EP
ready-diluted formulation of Jetrea® (ocriplasmin). The firm also received approval for this product in
News from the FDA and Other Regulatory Agencies •
Otsuka is suing the FDA over its broadening of the indications for Abilify® (aripiprazole), an
antipsychotic, as it had implications for generic competition (March 2015). •
Margaret Hamburg, MD, has retired as Commissioner of the FDA. Stephen Ostroff, MD, is the
Acting Commissioner (April 2015). Miscellaneous News Notes
ACCEPTED MANUSCRIPT 7 •
International Health Partners, a charity that distributes donated medicines to disaster zones,
celebrated 10 years of operation. A substantial portion of these donations, unfortunately, expire prior to being delivered to patients (March 2015). •
Mapp Biopharmaceuticals, in combination with the U.S. National Institute of Allergy and
RI PT
Infectious Diseases and the Liberian government, will evaluate the experimental drug cocktail ZMapp as a treatment for Ebola virus disease (February 2015). •
Merck, in combination with NewLink Genetics, will commence trials in Guinea for a
•
SC
vaccination for the Ebola virus (March 2015).
Novartis received FDA approval for Zarxio®, a biosimilar of Amgen's Neupogen®, making it
•
M AN U
the first biosimilar medication approved in the U.S. (March 2015).
San Francisco is considering an ordinance to require pharmaceutical manufacturers to institute a
AC C
EP
TE D
drug take-back program (March 2015).
