Companies seek a boost to ALS research

Work may eventually extend to other neurodegenerative diseases associated with superoxide dismutase 1 misfolding

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MARLBOROUGH, Mass.—Recently, RXi Pharmaceuticals Corp. and privately held Thera Neuropharma Inc. announced a licensing agreement that would utilize RXi’s sd-rxRNA platform, which uses drug-like properties built into the RNAi itself, to develop and research therapeutics for neurodegenerative diseases. This research will initially focus on superoxide dismutase 1 (SOD1) to develop new treatments for amyotrophic lateral sclerosis (ALS), commonly known as Lou Gehrig’s disease.
The therapy would work to reduce the level of misfolded SOD1 protein using RXi’s self-delivering RNAi compounds in combination with Thera’s Small Molecule Regenerative Therapy (SMRT). Under the agreement, Thera will be responsible for research, development, manufacturing, regulation and commercialization for any licensed products.
Previous to this agreement, RXi’s business plan had focused on dermatology and ophthalmology. Their most advanced compound, RXI-109, is currently in Phase 2 clinical trials for dermal scarring and Phase 1/2 for subretinal fibrosis associated with late-stage, age-related macular degeneration. The sd-rxRNA compounds have features similar to RXI-109, and have had some success in rodents. Thera has been developing a class of disease-modifying molecules in the first and only SMRT to uncover the therapeutic potential of a dual-target approach. This treatment is designed to protect neurons against protein misfolding and cell toxicity, inducing axonal regeneration to restore the neuronal network and delay progression of ALS symptoms.
The companies began their interactions in June of 2015, and it was not long before they realized the potential they possessed by joining their technologies. Over the last several months, the two companies have already filed several joint patents for the use of compounds for use in management of neurodegenerative diseases associated with SOD1.
“We are pleased to announce this licensing deal with Thera for our SOD1 targeting sd-rxRNA compounds,” said Dr. Geert Cauwenbergh, president and CEO of RXi Pharmaceuticals. He added, “Building on the early work that was done at RXi in collaboration with Dr. Robert Brown, chair of the Department of Neurology at the University of Massachusetts Medical School, we are happy to provide Thera with access to our robust self-delivering RNAi technology platform to develop therapeutics targeting SOD1. Thera has a management team and scientific advisory board with a deep knowledge of ALS in particular, and neurodegenerative diseases in general, that well positions the company for success.”
From a business standpoint, this agreement affords RXi, a small company with a tightly focused operational plan, the financial support to advance the technology itself. Their extensive IP portfolio allows them to expand the use of their technology to generate either fee- or equity-based licensing agreements, giving them potential for growth in both revenue and equity in other companies. The equity position RXi received in closing the transaction with Thera gives them the potential for future profit, equity and royalties.
In an interview with DDNews, Cauwenbergh also stated that he hopes this deal will stimulate other companies and investors to think more creatively about development solutions and look into similar combination approaches and accelerate development of new approaches in an area that has seen little progress in decades. “At the heart of what we do,” he says, “ is the goal to improve a patient’s life. We see this collaboration as a great step forward with the potential to develop therapeutics in an area with a significant unmet need. In the end, the major beneficiaries of this deal will be patients and their families.”
Thera currently has a number of other partnerships in order to advance their research and discovery program, and have collaborated, for example, with Southern Research in Birmingham, Ala., and the Vlaams Instituut voor Biotechnologie (VIB) in Ghent, Belgium, to advance research on their technology. In fact, in other recent news from Thera around the same time as the RXi announcement, the company announced that it had been awarded a $1,275,000 grant from the Flemish Agency For Innovation And Entrepreneurship - Vlaamse Agentschap Innoveren En Ondernemen (VLAIO), which will be used to further research on its small-molecule therapeutics and optimize the lead compounds to enter clinical stage in the near future. Part of the research will be conducted in collaboration with Prof. Rudi Beyaert of the VIB.
“We are delighted to have been selected for this funding,” said Dr. Antonella Favit-VanPelt, president and CEO of Thera Neuropharma. “The funding process at VLAIO is highly competitive and this selection underscores the unique opportunity provided by our small-molecule regenerative technology to deliver tangible and decisive treatment options for patients with neurodegenerative disorders.
“We are looking forward to working with Prof. Beyaert and VIB, and we believe that his expertise will be instrumental in enhancing our understanding of the mechanism of action and bring our therapeutic compounds closer to market.”

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