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Start-Up Previews (10/2011)

Executive Summary

This Month's Profile Group: The New Ophthalmic Drug Players, features profiles of Imagen Biotech, KalVista Pharmaceuticals, ONL Therapeutics and Xcovery Vision. Plus these Start-Ups Across Health Care: Acetylon Pharmaceuticals, Aleva Neurotherapeutics, cermaVEIN and Flex Biomedical.

The New Ophthalmic Drug Players

Unmet medical need and market size continue to drive venture investors to ophthalmic drug plays focused on diseases that blind. This interest is fueled by a stable pool of acquirers and the ability to in-license innovative, clinically validated compounds, providing VCs with a jump start to a potential exit.

(Also see "The New Ophthalmic Drug Players" - Scrip, 1 Oct, 2011.)

With time to exit an increasingly important metric for VCs, jump-starting new companies via in-licensing remains an important and oft-used strategy. Such derisking is one of the defining principles behind ophthalmology focused start-ups like Lux Biosciences, Ophthotech and PanOptica. Imagen Biotech Inc., a new ophthalmic play still operating in stealth mode, is no different. It plans to pursue new leads for sight-threatening diseases, including dry age-related macular degeneration.

(Also see "Imagen Biotech Inc." - Scrip, 1 Oct, 2011.)

Spin-outs can beget further spin-outs. That's what happened in the case of UK ophthalmology start-up KalVista Pharmaceuticals Ltd., launched in August 2011 as a spin out of Vantia Therapeutics. KalVista acquired a portfolio of small-molecule plasma kallikrein inhibitors from Vantia's preclinical store. The compounds will be developed to treat diabetic macular edema, a condition that threatens the vision of up to a quarter of the world's 285 million diabetes sufferers.

(Also see "KalVista Pharmaceuticals Ltd." - Scrip, 1 Oct, 2011.)

ONL Therapeutics LLC is developing an experimental drug to preserve sight in people with detached retinas and other retinal disorders. The start-up aims to leverage understanding about FAS, a cell-based receptor that is a key cause of cell death in photoreceptors. ONL's small peptide is designed to block the FAS receptor on cells of the eye, to keep the natural ligand from binding and keep the cells alive.

(Also see "ONL Therapeutics LLC" - Scrip, 1 Oct, 2011.)

Xcovery Vision LLC is repurposing an anti-angiogenic cancer drug to treat wet age-related macular degeneration. The pharmacokinetics suggest the compound can be delivered orally with no adverse events and still reach the back of the eye in sufficient quantities to stop the growth of blood vessels that is the hallmark of wet AMD.

(Also see "Xcovery Vision LLC" - Scrip, 1 Oct, 2011.)

Start-Ups Across Health Care

There are a number of histone deacetylase inhibitors on the market and in development that regulate gene expression in normal as well as cancer cells but can lead to a number of serious side effects. Acetylon Pharmaceuticals Inc. is developing small-molecule compounds that selectively inhibit HDAC for the treatment of cancers as well as inflammatory neurodegenerative, genetic and infectious disease.

(Also see "Acetylon Pharmaceuticals Inc." - Scrip, 1 Oct, 2011.)

The electrode leads used for deep brain stimulation to treat such conditions as Parkinson's disease, depression and essential tremors have become a lot smarter, thanks to technology developed by Switzerland's Aleva Neurotherapeutics SA. The company's microDBS platform is an evolution of micro-electro-mechanical systems that allows for precise stimulation of targeted brain tissue, leaving the adjacent region unaffected.

(Also see "Aleva Neurotherapeutics SA" - Scrip, 1 Oct, 2011.)

Treatment for often painful and unsightly varicose veins involves shutting down these defective veins and rerouting the blood to nearby healthy veins. For the most prevalent type of varicose vein – the greater saphenous vein – the standard of care in the US is endovascular thermal ablation, whereby the vein is heated from the inside, boiling the blood and sealing the vein closed. But instead of using radiofrequency or laser energy to heat the vein by inserting a catheter with a guidewire, cermaVEIN has found success with simple steam ablation, which avoids boiling the blood and does not require a guidewire.

(Also see "cermaVEIN" - Scrip, 1 Oct, 2011.)

Osteoarthritis, the progressive degeneration of the articular cartilage and synovial fluid in the joints, is one of the largest unmet clinical needs in health care today. Intra-articular injections of hyaluronic acid, which mimics the lubricating properties of natural synovial fluid and lessens friction and pain in the osteoarthritic knee joint, has become increasingly popular. However, of the seven formulations available in the US, none have been proven clinically superior and they have only limited long-term benefit to the patient. Flex Biomedical Inc. is working to address these concerns – and the large and vastly underpenetrated knee OA viscosupplementation market – with a single-injection synthetic polymer alternative to HA.

(Also see "Flex Biomedical Inc." - Scrip, 1 Oct, 2011.)

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