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Ophidion Inc.

Is an early-stage private biotechnology company



Presenter: Julie Miwa PhD, CEO and President


Monday, October 9, 2017

Embassy Suites, Chesterbrook, Pennsylvania (directions)




Ophidion, Inc. is an early-stage private biotechnology company developing a new class of safe and effective drugs targeting some of the most important receptors in the central nervous system. The activity of these targets, nicotinic receptors, are implicated in smoking dependence, anxiety, and cognitive decline associated with neurological disorders. Ophidion leverages its extensive academic ties, deep technical knowledge, and a forward thinking pharmacogenetics approach for early-stage drug development. It has been developing highly innovative drug programs which hold promise for improving the efficacy of CNS therapeutics, for the treatment of neurological disorders, anxiety and nicotine addiction.


Ophidion’s approach is an innovative solution for an intractable problem in the pharmaceutical industry, developing on the promise of cholinergic modulation as a therapeutic approach. Three of the four prescribed medications for Alzheimer’s disease are cholinergic agents, but their design leads to short (6-9 month) windows of clinical efficacy. Follow-on cholinergic agents have been attempted by targeting the receptors of the cholinergic system - neuronal nicotinic receptors - with the first set of nicotinic drugs just emerging in the marketplace. Therefore, Ophidion is pursuing a validated target of high interest in the pharmaceutical industry. Pre-clinical and clinical failures have highlighted the need for different approaches to drugging these promising targets.


In 2008, Dr. Julie Miwa recognized that nicotinic receptors operate in the context of multiple accessory subunits and auxiliary components, each of which can influence how the receptor behaves in its natural milieu in the neuron. She discovered the existence of key regulatory molecules that bind these receptors are control their function, but were completely missing from conventional pharmaceutical approaches. The discovery of these molecules and their function have been published in Science, PNAS, and Neuron, and been featured in mainstream science magazines such as Scientific American and international news broadcasts. Dr. Miwa co-founded a biotech company to take more innovative and comprehensive strategies for targeting nicotinic receptors in their natural context to achieve better clinical outcomes. This understanding has been used to invent a novel brain penetrating technology to enhance the delivery of CNS therapeutics into the brain to achieve superior clinical outcomes, in addition to developing anti-anxiety medications and smoking cessation candidates.


Ophidion, Inc. was formed in 2008 as a C corporation, as a spinout from the Rockefeller University. It uses a semi-virtual model, with wet-labs at the PBC incubator in Pasadena, CA, and headquarters at the Ben Franklin Tech Ventures incubator in Bethlehem, PA.


Ophidion owns four issued patents, two in the US and two in Japan, with a fifth about to issue in the EU and is prosecuting related US patents. Ophidion is prosecuting one patent assigned into Ophidion and is pursuing divisional application on related patent matter.


Short term projection: Ophidion is developing a platform technology that can be applied to
multiple indications and will be amenable to licensing options to multiple companies. The
financing structure will likely follow similar deals in the industry, with upfront payments, and
royalties and/or backend cash payments for successful milestones achieved. Ophidion is
interested in expanding the technology suitable for potential future gene therapies for
monogenic diseases, such as Huntington's, Alzheimer's and Parkinson's Diseases, which lack market attention despite increasing need and demand. Because the technology Ophidion is developing is sequence independent, it is amenable to multiple partnering opportunities representing multiple effector classes and indications (e.g. antibodies, oligos, etc.). Licensing or co-development deals are the most sensible approaches for this program. For business growth, Ophidion will add business advisors to negotiate deals.


Longer term outlook: Ophidion plans to develop a pipeline of its nicotinic receptor-based anxietymedications, once the initial medication is validated through its clinical trial. Staying with its largely virtual business model, it will not expand much beyond its technology development program, but will acquire relevant competencies as needed by outsourcing and collaboration. As each program matures, Ophidion can embark on drug development deals to sell or co-develop the programs with a pharmaceutical company. For each deal, Ophidion can negotiate upfront payments, milestone fees, and royalties. In all these arenas, Ophidion plans to evaluate multiple anxiety-related areas (Generalized anxiety disorder, phobias, PTSD, obsessive-compulsive disorder).



3 Major Issues


  1. What is the best way to value a pre-clinical biotech company? Is there more value to bundle programs together if they are disparate, or is it favored to separate them into different entities?

  2. What are ways to attract management level expertise to an early-stage biotech company? What are the key traits to focus on - experience raising money, clinical/regulatory competency, or experience with exits, etc?
  3. What is the best path to funding, and what should our funding strategy be?





6:30 - Cocktails & Dinner (Cash bar and special 2-entree buffet menu)

8:00 - Julie Miwa PhD, CEO and President,, will deliver Greene Street's "Elevator" Pitch to the Group

8:20 - A Panel will address three major issues crucial to helping the Company reach the next level.

9:00 - Open discussion: members and guests



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Contact: Peter van der Kam, | (610) 296-8086




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Meetings Held at Embassy Suites - Valley Forge

888 Chesterbrook Blvd, Chesterbrook, Pennsylvania 19087

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