Avalon-GSK Startup, Iron Horse Therapeutics, to Develop Novel ALS Treatments

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by Charles Moore |

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Brain capillaries and ALS

Avalon Ventures, a venture capital firm that has founded and/or funded more than 127 information technology and life sciences companies, and multinational pharma firm GSK have announced an expansion of their collaboration to fund and launch early-stage life science companies in the San Diego, California, area. The Avalon/GSK collaboration is now worth up to a potential additional $60 million in combined funding and earn-outs per company if the new companies develop marketed products.

Under terms of their expanded collaboration, which was initially established in April 2013, Avalon and GSK will create and fund additional companies through development of clinical candidates. These companies will also receive operational support, fully equipped R&D facilities and an experienced leadership team through COI Pharmaceuticals, a Community Of Innovation established by Avalon Ventures.

“The commitment by GSK has allowed us to move with unprecedented speed in transitioning concepts and ideas into functioning companies addressing unmet clinical needs,” says Jay Lichter, PhD, Avalon Ventures managing director and president of COI Pharmaceuticals in a release. “The newest, Iron Horse Therapeutics, is pioneering a novel approach to ALS, a disease in critical need of solutions.”

“Avalon Ventures and COI Pharmaceuticals foster the type of innovative thinking that the pharmaceutical industry needs,” says Lon Cardon, Senior Vice President, Alternative Discovery & Development at GSK. “The entrepreneurial and nimble companies we have founded in collaboration with Avalon will continue to build GSK’s portfolio of innovative therapeutics.”

You can view a YouTube video of Avalon’s Lichter and GSK’s Cardon discussing the companies’ biotech collaboration at:

Iron Horse Therapeutics, the seventh company launched to date under the Avalon-GSK collaboration, is developing treatments for amyotrophic lateral sclerosis (ALS) focused on compounds that modulate activity of EphA4 — a gene belonging to the ephrin receptor subfamily of the protein-tyrosine kinase family. EPH and EPH-related receptors have been implicated in mediating developmental events, particularly in the nervous system.

The company notes that ALS patients with reduced EphA4 expression or activity have less severe disease and live longer. Interfering with EphA4 signaling protects motor neurons from degeneration, and, in ALS animal models, this approach has been shown to prolong survival and decrease disease severity.

Iron Horse researchers explain that EphA4 expression enhances motor neuron loss with the large motor neurons that are more vulnerable to degeneration in ALS expressing higher levels of EphA4 than the less susceptible small motor neurons. Consequently, inhibiting the activity of EphA4 has been shown to protect the susceptible large motor neurons from degeneration.

Iron Horse was founded based on proprietary small-molecule inhibitors of EphA4 that were identified in the laboratory of Maurizio Pellecchia PhD, Associate Director of Translational Research at the Sanford-Burnham-Prebys Medical Discovery Institute at San Diego (La Jolla), California. Dr. Pellecchia is a recognized leader in NMR methods to develop modulators of protein-protein interactions. The technology he developed has been exclusively licensed from the Sanford Burnham Prebys Medical Discovery Institute to Iron Horse Therapeutics.

As with the other companies founded under the Avalon-GSK collaboration, Iron Horse will receive up to $10 million in Series A financing and R&D support from Avalon and GSK as well as R&D and mentoring support through COI Pharmaceuticals.

The collaboration between Avalon Ventures and GSK previously has launched Sitari Pharma, Silarus Therapeutics, Thyritope Biosciences, Adrenergics Inc., Cadherx Therapeutics, and Calporta Therapeutics.

Under the collaboration, GSK has the option to acquire each of the companies when a clinical candidate is identified. However, if GSK does not elect to exercise this option, Avalon will retain ownership of the company, and the company will be free to enter into other strategic transactions.

COI Pharmaceuticals is an unconventional venture-pharma entity that provides life science inventors an environment in which entrepreneurial and scientific risks are encouraged and rewarded. This Community of Innovation (COI) was established in 2013 to support companies funded through the collaboration between Avalon Ventures and GSK as well as existing Avalon portfolio companies. COI provides intellectual capital needed to launch companies, a fully-equipped R&D infrastructure, and industry mentors prepared to leverage their expertise to help entrepreneurial scientists succeed. COI is located in La Jolla, California. For more information, visit:

Avalon Ventures is passionate about backing talented entrepreneurs seeking to build market-leading companies. Avalon Ventures’ long-standing and successful focus throughout the firm’s 32-year tenure has been on seed and early-stage companies. Avalon’s current fund, launched in 2015, continues the firm’s tradition of pursuing fresh, even disruptive, ideas in ever-changing markets. For more information, visit:

Avalon Ventures
COI Pharmaceuticals, Inc.
Iron Horse Therapeutics
Sanford-Burnham-Prebys Medical Discovery Institute