PARIS—Pharnext SA, a French biopharmaceutical company developing an advanced portfolio of products in the field of neurodegenerative diseases, announced in March a research and development agreement with Galapagos NV. The main goal of this R&D deal is to generate an additional pipeline of novel synergistic drug combinations in a broad set of indications.
The agreement is based on both partners’ R&D capabilities, though the companies acknowledge that it will initially be based on Pharnext’s disruptive technological platform called Pleotherapy. Until now, this platform has enabled the discovery and patenting of low-dose combinations of approved drugs repositioned in new indications and aims to develop more rapidly, safer and more efficacious treatments for unmet medical needs.
According to the partners, the collaboration agreement with Galapagos “highlights another outstanding feature of Pharnext’s platform: its ability to reinforce the potential of new candidate drugs.” More to the point: For a given new preclinical or clinical candidate drug provided by Galapagos, Pharnext’s technology will aim to identify already approved drugs which could be combined at low doses with this new candidate in order to safely increase its efficacy.
This agreement will not only concern therapeutic indications that were primarily envisioned by Galapagos, but also others that Pharnext will identify. Expanding their market to new indications as well as generating new patents should significantly increase the value of the new compounds, they say.
Pharnext’s approach is said to be applicable to a broad spectrum of diseases. Therapeutic indications that will be considered in this R&D agreement will notably include immunoinflammatory and neurodegenerative disorders , both of which represent “potentially substantial markets.” Each of the companies will have priority on indications which will have been previously allocated.
“At Pharnext we have demonstrated that deeper understanding of genetics and molecular networks that underpin pathologies is key to drug discovery. The R&D collaboration with Galapagos validates the value of Pharnext’s innovative platform, Pleotherapy. Thanks to this technology, our team has already produced two novel synergistic drug combinations in late-stage clinical development. Moreover, this collaboration will enlarge our product portfolio with drug combinations including also new compounds,” said Dr. Daniel Cohen, co-founder and CEO of Pharnext. “We are proud to collaborate with Galapagos, an innovative and dynamic company in the biotechnology landscape. We look forward to working with Galapagos to generate new treatment options for patients who have no effective solutions today.”
“Pharnext has already obtained promising clinical results with its cutting-edge technology, which has encouraged us to collaborate,” added Onno van de Stolpe, CEO of Galapagos added. “We are very pleased to collaborate with Pharnext and to benefit from Pharnext’s expertise in deciphering diseases’ molecular networks. We hope that this will reinforce Galapagos’ R&D capabilities to generate new therapeutic approaches in a highly efficient and modern manner.”
Intellectual property with regard to synergistic drug combinations generated by the R&D collaboration will be jointly owned by Pharnext and Galapagos. Financial terms of the agreement have not been disclosed.
In other recent Pharnext news, the French biopharma has announced that the first patients have entered the international Phase 3 extension study PLEO-CMT-FU of PXT3003 at La Timone University Hospital in Marseille, France. PXT3003 is Pharnext’s lead Pleodrug for the treatment of patients with mild-to-moderate Charcot-Marie-Tooth Disease Type 1A (CMT1A), a rare and debilitating inherited peripheral neuropathy for which there are no satisfactory approved treatments available.
Pharnext plans to apply for marketing authorization for PXT3003 in Europe and the United States in the first quarter of 2019. Long-term safety data from PLEO-CMT-FU would then be submitted to regulatory authorities during their review of the marketing authorization application. This should lead to PXT3003 market approval during the second half of 2019, as scheduled.
PXT3003 is an oral fixed-low dose synergistic combination of (RS)-baclofen, naltrexone hydrochloride and D-sorbitol, developed using the Pleotherapy platform. In 2014, PXT3003 was designated an orphan drug for the treatment of CMT1A in adults in Europe and in the United States.
“The initiation of this second international Phase 3 trial marks an important milestone for the whole PXT3003 clinical development program as it aims at confirming the long-term safety and tolerability profile of PXT3003,” said Dr. René Goedkoop, chief medical officer of Pharnext.
As for Belgian biotech Galapagos, another recent bit of news it shared was to announce a new Phase 2 study investigating filgotinib and another investigational agent in cutaneous lupus erythematosus (CLE). This study is being led by filgotinib collaboration partner Gilead Sciences Inc.
“We are very excited with the initiation of this proof-of-concept study with filgotinib in CLE. This is the first time we evaluate filgotinib in an autoimmune skin disorder, and specifically, one with a significant unmet need,” said Dr. Walid Abi-Saab, chief medical officer of Galapagos. “This study represents another cornerstone in Gilead and Galapagos' efforts to explore filgotinib in inflammation. We look forward to seeing whether filgotinib can impact signs and symptoms of CLE.”
Galapagos and Gilead entered into a global collaboration for the development and commercialization of filgotinib in inflammatory indications. This study in CLE is in addition to the ongoing Phase 2 studies in Sjögren's syndrome, ankylosing spondylitis (TORTUGA) and psoriatic arthritis (EQUATOR), as well as the ongoing FINCH Phase 3 program in rheumatoid arthritis, the DIVERSITY Phase 3 study in Crohn's disease (also Phase 2 in small bowel and fistulizing Crohn's disease) and the SELECTION Phase 2b/3 study in ulcerative colitis.