Business Overview ONL Therapeutics, Inc. Company Contact: John Freshley Chief Executive Officer [email protected] 734.945.6344 ONL Therapeutics is developing a first-in-class product to protect photoreceptors from apoptosis caused by retinal diseases. FDA Orphan Drug designation for Retinal Detachment Rapid and capital efficient clinical POC plan De-risked clinical plan with significant upside in major markets Company Product(s): ONL-101 ONL Therapeutics is a biopharmaceutical company dedicated to the discovery and early clinical development of drugs to treat retinal disorders. The current market for retinal drugs is greater than $7 billion and rapidly growing. Existing therapies have significant limitations and none address the initial neural component of vision loss - the death of the photoreceptor cell. ONL Therapeutics is developing a breakthrough class of photoreceptor protective agents to address this large unmet medical need. Intellectual Property: Market Problem/Opportunity Sector: Pharmaceutical/Therapeutic 2 patents issued 1 patent pending Patents cover: use of Fas and FasL inhibitors in the eye and use of Met-12 (and/or analogs) in the eye Operating Team: David Zacks, MD, PhD; CoFounder & President David Kleinman, MD, MBA; Chief Medical Officer Anna Schwendeman, PhD; VP, Preclinical Development Jeff Jamison, PhD; CoFounder & CSO Prospective Customers: Retinal Detachment; $1B+ global market opportunity Age-related Macular Degeneration (AMD); $4B+ global market opportunity Current Investors: Capital Community Angels BRCC Management Investment Sought: $500K in Bridge Round $8M in equity capital Use of Funds: Completion of Series Funding IND Enabling Studies (Toxicology, CMC) Phase I/II POC Retinal Detachment Blindness is primarily caused by the death of photoreceptors initiated by retinal disorders such as Age-Related Macular Degeneration (AMD) and retinal detachment. While drugs and procedures have proven effective at managing the disease progression or surgically repairing anatomic injuries, during the treatment phase photoreceptors die and vision is permanently impaired due to apoptosis. Solution/Value Proposition ONL-101 is a first-in-class inhibitor that blocks FAS-mediated apoptosis in the retina to protect the photoreceptors and preserve vision. The drug is administered via an intravitreal injection and perfectly complements all existing (and developing) therapeutic approaches for retinal diseases (such as anti-VEGF, anti-PDGFR, surgery, gene therapy, etc). ONL-101 will improve the outcomes for all of these therapeutic approaches by preserving the photoreceptors until these other approaches can impact the underlying disease. Marketplace/Competition The current treatment for retinal detachment is surgery that reattaches the retina to its supporting layer. While surgery results in anatomical success in over 85% of the patients, in cases where the central vision (known as macula) is involved only 35% of patients retain driving vision (20/60 or better) post-surgery. Several independent studies involving thousands of patients have shown that for cases of macula-off detachments, the average visual outcome after one year is the loss of 3 lines (on a reading chart) of vision. These poor vision outcomes have not changed despite improved surgical techniques, which means other approaches (including pharmaceuticals) are needed to address this critical unmet medical need. In a similar manner, even though major advances have been made in recent years to treat AMD, and even with the best novel therapies, the average patient still loses 3 lines of vision in the first year to due photoreceptor cell death. ONL’s approach promises to also address this significant unmet need and significantly improve visual outcomes and quality of life for patients. Marketing/Sales Strategy Our initial indication will be retinal detachment for which ONL has received an FDA Orphan Drug designation. This indication will be followed by wet AMD. There are no other drugs in the market or in development that target photoreceptor apoptosis. ONL’s patent position and orphan drug designation provide significant barriers to entry for competition. Retinal detachment represents a $1B+ global market, with an initial market of macula-off detachment representing approximately $300M. AMD represents an annual market opportunity exceeding $4B. Management Team John Freshley, Chief Executive Officer, has 18+ years of leading start-up companies. Most recently, he was Chief Business Officer of Compendia Bioscience, a cancer genomics company that he helped launch as interim CEO in 2006 and was sold to Life Technologies in October of 2012. Previously, he helped start and/or lead multiple drug development companies with a focus on personalized medicine. David Zacks, M.D., Ph.D., Co-Founder & President, is Associate Professor of Ophthalmology and a vitreoretinal surgeon and physicianscientist at University of Michigan’s Kellogg Eye Center and a recognized global leader in retinal neuroprotection. David Kleinman, M.D., M.B.A., FACS, Chief Medical Officer, is a board-certified ophthalmologist with training in vitreoretinal surgery and over 10 years experience in venture creation and ophthalmic drug development. Jeffrey Jamison, Ph.D., Co-Founder & Chief Scientific Officer, has 20 years of drug discovery and development experience in Alcon and Pfizer ophthalmology research units. Anna Schwendeman, Ph.D., Vice President of Preclinical Development, has expertise in drug manufacturing and regulatory affairs, having submitted multiple INDs to FDA and European regulatory agencies. Financial Projections Our clinical proof-of-concept will be demonstrated in macula-off retinal detachment. This is a very capital efficient indication and an attractive commercial opportunity. The drug will be administered with a single injection simplifying our toxicology requirements. Our phase II program will be a robust, randomized, controlled study that will compare the effect of ONL-101 plus the standard of care, which is surgery vs. surgery alone. Because of the acute nature of the disease, we will have rapid results 6 months after completion of trial recruitment. Concurrent with our clinical program in retinal detachment, we will conduct in vivo proof-of-concept studies in AMD. Investment Opportunity ONL is seeking $8-10M Series A investment. These funds will support the following: IND-enabling studies and IND filing in retinal detachment Phase I in retinal detachment (9 - 15 patients) Phase II in retinal detachment (3 arms – 1 placebo, 2 different dose arms – 120+ patients) Corporate operations and legal through 2017 These data will position the company for at a significant inflection point for exit, partnering and/or further investment.
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