As many as 95 percent of experimental medicines and treatments fail during research or testing phases, and, according to recent research, the cost of developing a single new drug can be as high as $5bn. Over the years, the development of new drugs has become a costly and time-consuming enterprise. Due to ever-changing regulatory demands, the biotechnology industry has had to evolve fast in order to remain financially viable while still delivering top results. A 2012 article in Nature Reviews Drug Discovery suggests the number of drugs per billion dollars invested in research and development has dropped by half every nine years for the past 50 years.
With the ever-growing costs of developing successful drugs, the onus of research and development is increasingly falling on the shoulders of smaller, specialised companies that focus on specific disease targets and delivery platforms requiring expert scientists well-versed in their field. Because of this focus, resources can be applied much more efficiently to develop innovative treatments. Rhode Island-based Neurotech Pharmaceuticals is one such company. It is focusing its resources on commercialising protein-based therapies for chronic ophthalmic diseases using a proprietary drug delivery platform. Many of the applications of this platform affect large portions of the ageing population, such as age-related macular degeneration (AMD) and glaucoma, while others target orphan diseases affecting younger patients, such as retinitis pigmentosa and macular telangiectasia.
Potential cost of developing a new drug
Utilising its unique transformational technology platform, Encapsulated Cell Therapy (ECT), Neurotech has developed an intraocular implant that can deliver therapeutic proteins directly to the back of the eye for up to two years. ECT implants are a proven technology and involve the delivery of biologics by means of a miniaturised, implantable protein manufacturing unit. ECT implants contain human retinal pigment epithelium cells that have been engineered to produce and release a desired therapy that is encapsulated in a semi-permeable hollow fibre membrane. The encapsulated implant is surgically inserted into the vitreous body and sutured to the scleral wall during a brief and simple surgical procedure. ECT implants uniquely enable the controlled, continuous, long-term delivery of biologics directly to the retina, bypassing the blood-retinal barrier and overcoming a major obstacle in the treatment of retinal disease.
Neurotech’s lead ECT-based clinical programme (NT-503) is designed to treat wet (neovascular) age-related macular degeneration, the leading cause of irreversible vision loss in industrialised countries. Current treatments for wet AMD have improved immensely over the last decade with the advent of anti-VEGF injections. However, these treatments are administered monthly to every six weeks and must continue for an indefinite period of time in order to manage the disease. This creates a significant burden on the office, creating crowded waiting rooms and the need for additional support staff to handle patient volume. Additional burdens, mostly economic and practical, are placed on patients, their caregivers, and the healthcare system as a whole. Repeat injections can also cause general eye health to decline, with some patients experiencing increased rates of endophthalmitis (a severe eye infection), general ‘injection fatigue’ and pain. Moreover, as the disease progresses over many years, most patients eventually become undertreated and lose the vision benefits they may have gained. A clear unmet need is the ability to administer proven sight-saving therapies in a controlled and long-term delivery system.
NT-503 is designed to continuously secrete effective, long-term levels of VEGF-antagonists to treat wet AMD, which may eliminate the need for injections. According to Neurotech: “The NT-503 ECT product has demonstrated clinically meaningful improvements in best corrected visual acuity and reductions in macular thickening in patients with active neovascular age-related macular degeneration that persist for at least 12 months with a single procedure. The next generation product soon to enter the clinic is designed to have higher protein release rates and to also support combination therapy.” Neurotech is also developing NT-506 for the treatment of wet AMD. NT-506 secretes a PDGF (platelet-derived growth factor) antagonist, which is anticipated to improve efficacy and reduce scarring of the retina when combined with the NT-503 VEGF-antagonist in their latest generation device.
Management and prevention
Another ECT-based programme, known as Renexus, is being developed for the treatment of macular telangiectasia and retinitis pigmentosa. Currently, there are no available treatments for these incapacitating degenerative conditions, so many patients will experience vision loss or blindness. Neurotech is one of the few companies helping to develop therapies specific to these rare, but blinding, diseases.
Renexus is also being tested in glaucoma, another leading cause of irreversible blindness globally. Glaucoma is a neurodegenerative disease resulting from loss of retinal ganglion cells (a type of nerve cell in the retina) and their axons, which causes damage to the optic nerve and subsequent vision loss. In addition to traditional glaucoma treatment – namely topical eye drops that aim to lower pressure inside the eye – therapeutic approaches are now aimed at protecting against cell death through neuroprotection. Results of a study in patients with advanced glaucoma recently presented at the World Ophthalmology Conference by Jeffrey L Goldberg, Professor and Director of Research at the Shiley Eye Centre at the University of California – San Diego, showed for the first time that Renexus was associated with a marked increase in the nerve fibre layer in implanted eyes compared with the control eyes. This suggests a potential neuroprotective role in the management and prevention of glaucoma.
Conservative US market models for wet AMD, macular telangiectasia, retinitis pigmentosa and glaucoma show peak year sales are forecasted to approach $4bn. In addition, the product opportunities for ECT implant programmes can be expanded to include treatment for a number of other significant retinal diseases such as diabetic macular edema, retinal vein occlusion and dry age-related macular degeneration. The worldwide commercial opportunities for the development of ECT implants are significant, considering the number of patients globally suffering from chronic retinal diseases and glaucoma.
Over the next two years, Neurotech will hit a number of significant R&D milestones that will cement the viability of ECT implant treatments by demonstrating their safety and efficacy. Neurotech has initiated Phase 2 clinical studies for Renexus in macular telangiectasia and the latest generation NT-503 product in wet AMD. While additional late-stage clinical development continues, preliminary results and feedback have been positive for treatment across a range of conditions.
Neurotech has designed a unique business strategy to create value through advancing the clinical development of its main existing programmes, and by broadening the pipeline for the development of further ECT-based treatments. It is common at this stage of development for pharmaceutical research and development companies to seek partners that will enable the continuation of clinical development, and Neurotech is no different in that respect. What sets it apart, however, is the quality and uniqueness of its combined innovative delivery platform and robust therapeutic programme.
Qualitative market research, performed by pharmacoeconomic consulting firm McKesson to assess payer attitudes in the US, confirmed the company’s ECT product approach would be well-received. Furthermore, assuming a 12-month treatment effect with the lead NT-503 AMD product, insurers confirmed a premium price would be reasonable and warranted, given the advantages of a proven therapy and sustained delivery. It is all but guaranteed that the treatment – which has not yet been given a commercial name – will be a true blockbuster.
Neurotech is representative of an emerging force in biotechnology; it is a small organisation with top talent, working hard to fill the gaps in the market left by big pharmaceuticals, as they typically shy away from research and development. These companies represent the future of the drug and treatment industry, and their potential for growth is practically boundless. Neurotech is particularly well positioned because of the quality of the treatment it offers: it is, quite literally, a light at the end of the tunnel.