XLRPX-Linked Retinitis Pigmentosa (retinal disease)
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MeiraGTx will receive $100 million in cash as an upfront payment and is eligible to receive additional payments for achieving development and sales milestones related to its CNGB3, CNGA3 and XLRP programs of up to $340 million, and royalties on sales for the CNGB3, CNGA3 and XLRP programs.
XLRP is most commonly caused by a mutation on the RPGR gene on the X-chromosome that causes blindness in about one in 15,000 men.
XLRP is a rare orphan disease that occurs due to a congenital genetic defect.
Biotechnology company Applied Genetic Technologies (NasdaqGM:AGTC) revealed on Tuesday that it has received European Commission (EC) orphan medicinal product designation for its investigational gene therapy product candidate for the treatment of X-linked retinitis pigmentosa (XLRP) caused by mutations in the RPGR gene.
Research and development expense was USD3.4m for the three months ended 31 December 2014, a rise by USD1.2m over R&D of USD2.2m the same period in 2013, due to increased activity relating to its XLRS, ACHM, XLRP and other product candidates, including higher facilities and other costs associated with laboratory expansions.
He keeps a Buy rating on the shares but admits Applied Genetic "might be in the penalty box pending positive clinical data from the ACHM and/or XLRP programs to validate this thesis."
AGTC also announced that the company will regain full rights to the XLRS and X-linked retinitis pigmentosa, or XLRP, programs and the three other partnered discovery programs following Biogen's (BIIB) termination of the collaboration agreement, which will be effective on March 8.
Shares of Nightstar Therapeutics should trade higher after presenting positive first data for its new XLRP gene therapy that demonstrates multiple "responders" of vision improvement, solid durability out to six months, and good safety, Jefferies analyst Michael Yee tells investors in a research note.
The XIRIUS trial is intended to evaluate the safety, tolerability and efficacy of NSR-RPGR for the treatment of XLRP in patients with the RPGR mutation.