Replies to post #1067 on Biotech Values

[DewDiligence]

Re: Allergan:

isolution: Allergan’s collaboration with ENMD to develop for Panzem for AMD is all but dead. In more than three hours of discussion at AGN’s R&D Day last week, the Panzem project was mentioned for all of 5 seconds. (That’s not an exaggeration.)

In contrast, the VEGF TKI from Bardeen (now known as AGN-6) is white hot and it received considerable attention during the talk by Larry Wheeler (AGN’s VP of Biologics).

>> Anyway Dew, One again it is local drug delivery story (Alcon, Genentech, Novartis, Eyetech, B&L, Allergan, Genvec, Neurotech, Oxford Biomedica, Oxigene)…<<

As I mentioned previously, Allergan has an oral project in AMD using Tazarotene, a drug already approved in topical form for acne. Pending completion of an IND, oral Tazarotene will enter phase-2 trials in AMD during the second half of 2004. I do not understand why you have chosen to ignore the Tazarotene program in trying to argue that only drugs with localized administration have merit in treating AMD. Regards, Dew

[DewDiligence]

Positive Phase I Data for Neurotech's NT- 501 Encapsulated Cell Delivery of Ciliary Neurotrophic Factor to Eyes of Patients With Retinitis Pigmentosa

[isolution: any comment on this company? They are based in Paris. They are working on wet AMD as well as RP. T.i.a.]

http://biz.yahoo.com/prnews/050501/ukf013.html?.v=4

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Data Presented at the 77th Annual Meeting of the Association for Research in Vision and Ophthalmology (ARVO) Conference in Fort Lauderdale, Florida

FORT LAUDERDALE, Florida, May 1 /PRNewswire/ -- Neurotech SA, a specialist in the development of novel therapies for retinal diseases, today announced positive results from an open-label Phase I clinical trial (03-EI-0234) of its lead product, NT-501. NT-501 uses Neurotech's patented Encapsulated Cell Technology (ECT) as a device to deliver ciliary neurotrophic factor (CNTF) to eyes of visually impaired patients with retinitis pigmentosa (RP). Results confirm that CNTF can be safely delivered into the vitreous of patients with RP and that the ECT device was well tolerated by all patients. Futhermore [sic], some patients experienced more than one-line of improvement in their visual acuity score. These Phase I results were presented at the ARVO annual meeting and the trial was conducted at the National Eye Institute (NEI), Bethesda, USA. Neurotech has confirmed that it will now progress to a multi-center Phase II trial.

ECT, a technique developed and patented by Neurotech, allows for genetically-engineered specific protein delivery without manipulating the patient's genetic information or transferring new genetic information into the target tissue. The Phase I study of NT-501 involved 10 patients with late-stage RP. The study was designed as an open-label safety and tolerability evaluation. Two doses of CNTF (5-fold difference in dose) were evaluated. Phase Ia treated 5 patients with a lower dose; Phase Ib treated 5 patients with a higher dose. The ECT device was implanted in one eye per patient and removed after six months. All explanted devices contained viable cells that continued to produce CNTF.

Commenting on the positive results, Weng Tao, MD, PhD, CSO and VP of R&D with Neurotech said: "These safety and tolerability results are extremely encouraging and strengthen our confidence in pursuing NT-501 for treating RP and other retinal degenerative diseases. I would like to thank the National Eye Institute for conducting this milestone study and for its continued involvement with this technology."

Al Reaves, PhD, VP of Clinical Development with Neurotech confirmed: "This study represents the first use of ECT in human eyes and it is reassuring that the devices were safe and well-tolerated. We are planning Phase II development with well-designed and controlled multi-center clinical studies to help understand the role that NT-501 will play in treating patients with retinitis pigmentosa and other retinal degenerative conditions."

In this trial, the small ECT device was surgically-implanted into the vitreous cavity through the pars plana in one eye per patient. The primary inclusion criteria for the study eye included visual acuity of 20/100 or worse, central visual field diameter of 40 degrees or less, and flicker ERG amplitude of 2 microV or less. The first 2 patients were also required to have visual acuity of 20/400 or worse. After surgical implantation of the device, each patient was followed for six months after which the device was explanted. Safety and tolerability was monitored by an independent Data and Safety Monitoring Committee. All 10 patients completed the study as planned and the devices have been explanted. The ECT devices were well tolerated during the 6 months of implantation and the surgical procedure resulted in minimal or no observed inflammatory reaction. No serious adverse events were reported and in the untreated fellow-eye, there was little change from baseline in the visual acuity score during follow-up. In the treated study-eye, however, the visual acuity score was more variable during follow-up: while some treated eyes showed little change from baseline, some patients experienced more than one-line of improvement in their visual acuity score.

Bernard Davitian, President of Neurotech, said: "We are extremely pleased that the Phase I trial has validated the safe use of the Encapsulated Cell Technology to deliver CNTF to the vitreous. In addition, the Phase I trial has validated ECT as a delivery platform for the back of the eye and the visual acuity outcomes observed in some patients are encouraging enough to pursue the clinical evaluation of NT-501 in Phase II trials. In addition to further studies with NT-501, we are evaluating other neurotrophic factors and agents that can be used with ECT for treating other retinal diseases."

In addition to NT-501 for the treatment of Retinitis Pigmentosa, Neurotech is applying ECT technology to deliver other protein factors for the treatment of other ophthalmic diseases, including anti-angiogenic factors for the treatment of the wet form of age-related macular degeneration (AMD) and diabetic macular edema (DME), and anti-inflammatory factors for the treatment of posterior uveitis.

About Retinitis Pigmentosa (RP)

RP is a group of inherited retinal diseases that affects about 100,000 Americans and 1.5 million people worldwide. It causes the progressive deterioration of specialized, light-absorbing cells in the retina, the paper-thin tissue that lines the back of the eye like film in a camera. As these cells slowly degenerate, people with RP develop night blindness and a gradual loss of peripheral vision. By about age 40, most have tunnel vision, although many may retain good central vision. Between the ages of 50 and 80, however, they typically lose their remaining sight. The extent of vision loss in people of the same age with RP may be different.

About Encapsulated Cell Technology (ECT)

ECT is a unique technology to overcome drug delivery problems into the back of the eye. ECT enables the controlled, continuous, long-term delivery of therapeutic proteins directly to the retina. In addition, the implants can be retrieved, providing an added level of safety as well as the ability to reverse or adjust therapy, if needed. ECT relies on the use of an immunoisolatory hollow fiber membrane technology to allow the implantation of genetically engineered cells that continuously produce the therapeutic protein at the site of implantation. The cells are loaded into the interior volume of the hollow fiber membrane and attach to a proprietary supportive matrix within this space. The genetically modified cells remain captive within the ECT device thus avoiding the risks associated with traditional gene therapy. Long term protein delivery (18 months) in the vitreous cavity of the eye has consistently been achieved when ECT devices containing human retinal pigmented epithelial (RPE) cells genetically engineered to secrete CNTF have been implanted in a highly disparate mammalian species (rabbits).

About Neurotech

Neurotech is a biotechnology company specializing in the development of novel therapeutics to treat diseases of the eye. The company's initial focus is on chronic diseases affecting the back of the eye, especially the retina, because retinal diseases represent the greatest unmet medical need and therefore offer the largest market opportunities in ophthalmology. The Company has one product (NT-501) in development for the treatment of retinitis pigmentosa and other degenerative diseases of the retina, and is evaluating other neurotrophic factors and agents that can be used with ECT for treating other retinal diseases. The company is headquartered in Paris with an American subsidiary, Neurotech USA, Inc., located in Lincoln, RI, south of Boston. Neurotech is supported in its scientific and business strategies by world experts in ophthalmology and by a group of international investors led by Apax Partners and Merlin Biosciences. To learn more about Neurotech, please visit our web site at www.neurotechusa.com
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[DewDiligence]

Neurotech Initiates a Phase II Clinical Trial of NT-501 for the Treatment of Dry Age Related Macular Degeneration

[Aside from a Alcon’s Retaane, this Neurotech trial is the only study I’m aware of that is testing a therapy for the dry form of AMD.

isolution: if you are still reading this board, I’d appreciate your comments.]

http://biz.yahoo.com/prnews/060105/nyth057.html?.v=35

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LINCOLN, R.I., and PARIS, Jan. 5 /PRNewswire/ -- Neurotech, a biotechnology company specializing in the development of sight-saving innovative therapeutics for chronic retinal diseases, announced today the initiation of a Phase II clinical trial of NT-501, the Company's lead Encapsulated Cell Technology (ECT) product for the treatment of visual loss associated with the dry form of age related macular degeneration (dry AMD).

NT-501 is an intraocular, polymer implant containing human retinal epithelial cells genetically modified to secrete Ciliary Neurotrophic Factor (CNTF). The implant is designed to continuously release CNTF directly in the eye to the diseased retinal tissue for sustained periods of time. The Phase II trial is a randomized, double-masked, sham-controlled dose ranging study that will evaluate the efficacy and safety of the CNTF implant and will be conducted at the National Eye Institute (NEI) in Bethesda, Maryland.

"Dry AMD is a form of retinal disease that needs serious attention and we are very pleased to begin the Phase II trial for the indication," stated Ted Danse, CEO of Neurotech. "We believe the improvement in visual acuity observed in our Phase I trial will translate into a similar benefit for patients with advanced stage dry AMD."

An open-label Phase I clinical trial of NT-501 was conducted in patients with retinitis pigmentosa (RP) by Paul A. Sieving, M.D., Ph.D., at the National Eye Institute. Dr. Sieving presented the results last November at the American Academy of Ophthalmology, and the results confirmed that CNTF can be safely delivered into the vitreous of patients with RP. The ECT implants were well tolerated by the RP patients, and some patients experienced improvement in their visual acuity score.

According to Dr. Sieving, "The results from the Phase I trial have encouraged us to study NT-501 in patients with atrophic macular degeneration affecting their central vision, a type of degeneration that is similar to that seen in the late stage RP patients enrolled in the Phase I study." Two Phase II studies of NT-501 in patients with RP are also being planned for initiation in 2006 in conjunction with the NEI.

About Neurotech

Neurotech is a privately-held biotechnology company dedicated to the development of sight-saving therapeutics for chronic retinal diseases. Retinal diseases represent significant unmet medical needs for which new medical therapies are the largest market opportunity in ophthalmology. Neurotech's lead product (NT-501) is in clinical development for the treatment of retinal degeneration, including retinitis pigmentosa, an inherited disease leading to blindness, and the dry form of age related macular degeneration. Neurotech is also evaluating other factors that can be used with its proprietary delivery technology, Encapsulated Cell Technology, to treat additional retinal diseases. The company's U.S. office is headquartered in Lincoln, RI, and European operations are located in Paris. Neurotech is supported by leading world experts in ophthalmology and by a group of international investors led by Apax Partners and Merlin Biosciences. To learn more about Neurotech, please visit our web site at http://www.neurotechusa.com.
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