Catalyst Case Studies

Epygenix Therapeutics

Epygenix Therapeutics is a New Jersey-based precision medicine biopharmaceutical company established by Hahn-Jun Lee, M.Sc., Ph.D. in 2016, with technology developed by UCSF Prof. Scott C. Baraban, Ph.D. Epygenix is focused on developing drugs to treat genetic epilepsies, including Dravet syndrome, a rare and catastrophic form of intractable epilepsy that begins in infancy.

Current therapeutic options for Dravet syndrome are not effective. Patients often endure hundreds of seizures in their early years, and the constant care required places a severe burden on families. Dravet syndrome, like most genetic forms of epilepsy, represents an orphan indication with an unmet medical need.

UCSF’s Catalyst Program provided funding and guidance to Baraban, who had published the first high-throughput drug screening using a zebrafish model for Dravet syndrome in 2013. Baraban combined the innovative notion of using zebrafish—which reproduce rapidly and give an early indication of whether a compound holds promise as a treatment—with the concept of purchasing libraries containing large numbers of repurposed compounds. The first round screening identified an antihistamine clemizole (EPX-100) that was approved by the Food and Drug Administration in the 1950s but was no longer manufactured or clinically available. The second round screening identified two other safely used FDA-approved drugs which treat obesity (lorcaserin; EPX-200) and sleep disorder (trazodone; EPX-300).

Epygenix Therapeutics is moving rapidly. The company received Orphan Drug Designations for EPX-100, EPX-200, and EPX-300 from the FDA in 2017, and it expects to receive orphan medicinal product designations from the European Medicines Agency (EMA) in early 2018. Epygenix is preparing to take EPX-100 and EPX-300 to clinical trials in 2018 after completing the necessary preclinical toxicology, formulation, and chemistry, manufacturing, and controls (CMC) studies.

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ChemoFilter, Inc. was a California biotechnology startup established in 2013 by UCSF Prof. Steven Hetts, MD, Anand Patel, MD, a UCSF radiology resident, and Albert K. Chin, MD, a biotechnology inventor and entrepreneur. The company’s main product, also known as ChemoFilter®, is a sponge-like device designed to attract and absorb excess drugs during intra-arterial delivery of chemotherapy for liver cancer, preventing all or most of those chemotherapeutics from entering the bloodstream and thereby improving outcomes while alleviating the worst side effects of chemotherapy.

Intra-arterial chemotherapy (IAC), which delivers high concentrations of drugs directly to tumors, represents a common strategy in fighting many different types of cancers. Current IAC methods do not eliminate side effects such as nausea, vomiting and bone marrow suppression, which limits the ability to use large concentrations of chemotherapeutic drugs to cure a particular cancer. The need to solve this persistent problem represents a large opportunity for innovation.

With guidance from UCSF’s Catalyst Program, ChemoFilter, Inc. was established as a virtual company, and was acquired in 2015 by a global interventional therapies company that designs, manufactures and markets innovative medical devices.

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Kyprolis® (carfilzomib) is a multiple myeloma drug that was developed by a biotech startup, Proteolix, from the stage of basic academic research through Phase II clinical trials. Proteolix went through three rounds of funding before being acquired by Onyx Pharmaceuticals for $851 million in 2009. Kyprolis® ultimately received FDA approval in 2012 and since then has been used to treat thousands of patients. The case study of Kyprolis® highlights some of the challenges and opportunities involved in translating basic research into a commercially viable product. Some key lessons offered by Kyprolis® include strategies for forming a team that maximizes the likelihood of commercial success, the need to pivot in response to investors’ demands, the importance of developing an appropriate target product profile in order to attract investors, and the role of intellectual property (IP) considerations in determining the trajectory of commercialization.

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Intellikine was founded in 2007 and brought three drug candidates to clinical trials in a remarkable four years before being acquired by Millennium Pharmaceuticals in 2012. As of 2015, two of its drugs are moving through clinical trials as part of Millennium’s portfolio, while the third one (duvelisib) was licensed out to Infinity Pharmaceuticals and is in a phase III trial in patients with chronic lymphocytic leukemia. Intellikine’s success story illustrates several noteworthy points. The founders assembled a team with a combination of scientific and business expertise, and recruited a chief executive officer (CEO) to help develop a business plan and negotiate deals before obtaining any venture funding. Once funded, the company accelerated and lowered the cost of their drug-development program by partnering with a contract research organization (CRO) in China. Intellikine’s strategic approach to raising capital and entering into partnerships contributed to their success in a very competitive and fast-paced environment, as did the co-founders’ active management of intellectual property (IP).

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Integrilin® (eptifibatide) is an antiplatelet drug developed by COR Therapeutics from the stage of basic research on snake venom to an FDA-approved drug, which has been used worldwide by millions of patients with cardiovascular disease since its approval in 1998. COR Therapeutics was ultimately acquired by Millennium Pharmaceuticals for $2 billion in 2002. Some key lessons that can be learned from Integrilin® include insights into how to identify unmet clinical needs; the advantages of developing a small molecule aimed at the validated target of a biologic; the choice faced by academic founders between staying in academia, where only part of their time could be dedicated to the new venture, and leaving to become a full-time executive; the importance of partnerships during the drug development and regulatory process; and the crucial role that a successful reimbursement strategy can play in marketing a novel drug.

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