With its licensing agreement to develop genome-editing programs with Editas Medicine to treat retinal diseases, Allergan has made a move to partner with one of the leading players in the CRISPR genome-editing space at a time when the field is poised to take off.
That’s not Allergan’s hype. A recent report by San Francisco-based Grand View Research, Inc. estimates the global genome-editing market will reach $8.1 billion by 2020. Another report by Inkwood Research predicts a 35% compounded annual growth rate for the global CRISPR market through 2022. Among the 16 major players Inkwood Research has identified in the CRISPR space: Editas Medicine. CRISPR stands for clustered regularly interspaced short palindromic repeats, a technology that can target specific stretches of genetic code and edit DNA at precise locations in the human genome and allows researchers to permanently modify genes.
A Good Week for Editas
Under the agreement, Allergan gains exclusive access and the option to license up to five of Editas’ genome-editing ocular programs, including its lead program for Leber congenital amaurosis type 10 (LCA10), currently in preclinical development.
Last week was a good one for Editas. Not only did it disclose the Allergan licensing agreement and the $90 million up-front payment that comes with it, but a day later it filed for a stock offering that would raise another $90 million to fund development of its LCA10 candidate as well as other programs to treat inherited retinal diseases.
Besides the up-front payment, Allergan would be obligated for a one-time payment “in the low-eight digits” to Editas upon submission of an Investigational New Drug (IND) application for the LCA10 program whether or not Allergan exercises its option to acquire an exclusive license to the product, according to the Securities and Exchange Commission filing. Total payments could exceed $200 million for the LCA10 treatment.
Allergan will have the option to license up to five Editas programs under the agreement and will be responsible for development and commercialization of the optioned products, subject to Editas’ option right to co-develop and co-promote up to two optioned products in the US. Editas will also be eligible to receive development and commercial milestones, as well as royalty payments on a per-program basis. With each option Allergan exercises, it is obligated to pay Editas the same “low-eight digits” as with the IND for LCA10 along with related costs Editas incurs.
The agreement gives Allergan the right to license the programs in any disease except cancer through the use of engineered T cells.
In a conference call with analysts last week, David Nicholson, Allergan’s chief research and development officer, said, “Editas Medicine has built an unparalleled platform for developing CRISPR medicines, while Allergan brings deep capabilities and expertise developing and commercializing medicines for ocular disease.” In the press release announcing the agreement, Nicholson said the LCA10 program “is highly complementary to our ongoing eye care development programs where unmet medical need exists for patients.”
Editas president and CEO Katrine Bosley told the analysts, “We believe that our ophthalmology programs will significantly benefit from Allergan’s expertise and capabilities in ophthalmology R&D, which are very complementary to Editas’ genome-editing capabilities.”
Editas’ work in LCA10 has focused on the CEP290 gene. “We did disclose earlier this year that we did see very encouraging editing of the CEP290 gene, which is the gene underlying LCA10, in a nonhuman primate study,” Charles Albright, PhD, Editas chief scientific officer, said in the analysts call. Editas will report more detail on those findings at an upcoming meeting.
Seeking a Cure for LCA
LCA, a rare congenital retinal dystrophy that leads to blindness, affects 2 to 3 of 100,000 births and represents almost 5% of all retinal dystrophies and 20% of children with visual impairment in special schools, Eye Wiki reports. Seventeen phenotypes with 22 genotypes have been identified that cause LCA, accounting for more than half of all LCA diagnoses.
At least two other biotech companies are involved with finding a cure for LCA, albeit targeting different genes than the Editas program. Spark Therapeutics has been investigating its lead candidate, SPK-RPE65, to target mutations in the RPE65 gene, which is linked to LCA type 2 as well as retinitis pigmentosa (RP). Novelion Therapeutics, formerly known as QLT Inc., has reported on its work on the agent zuretinol, which also targets the RPE65 gene for treatment of LCA and RP.