Novartis AG is expanding its position in the optogenetics space with the acquisition of Vedere Bio Inc., bringing with it a program aimed at vision loss prevention and treatment.
Shareholders of Vedere, created in June 2019 through the Atlas Venture incubator, received $150 million up front and are eligible for up to $130 million in milestone payments, bringing the total to $280 million.
The acquired cell and gene therapy technologies include light-sensing proteins for use in the retina and the delivery vectors to get them there. Vedere’s lead preclinical, intravitreally injected AAV gene therapy programs are designed to restore pan-genotypic vision in patients with photoreceptor-based vision loss, which includes inherited retinal dystrophies.
Novartis said it would focus on AAVs, chimeric antigen receptor T cells and CRISPR.
The optogenetics technology is based on work done by Cambridge, Mass.-based Vedere’s scientific founders at the University of California, Berkeley and technology for studying ocular gene therapy delivery developed at Berkeley and the University of Pennsylvania’s school of veterinary medicine.
The scientific founders, Ehud Isacoff and John G. Flannery, injected a green-light receptor into blind eyes of several mice and four weeks later the mice could see movement and detail, showing treatment appeared to reverse the retinal degeneration. The light-sensing proteins, via AAV capsids, go to retinal cells, stimulating them to transmit information to the brain’s visual processing center.
Vedere had a $21 million series A financing to its credit and labs at Labcentral in Cambridge, Mass. The company said it took its lead programs from concept to a development candidate in a year. Prior to the Novartis acquisition, Vedere said that some of its earlier-stage vision restoration and preservation assets that leverage its ocular gene therapy toolbox were spun out in a new entity, Vedere Bio II Inc. The spinout is wholly independent from Novartis and Vedere Bio. Its investors include Atlas Venture, Mission Biocapital and Foundation Fighting Blindness. Vedere Bio II will be known as Vedere, the company said.
The transaction closed in September, according to Vedere, which is the Italian word for “see.”
The latest deal adds to Novartis’ gene therapy efforts. Novartis acquired AAV-oriented Avexis Inc., of Chicago, in 2018 for $218 per share or a total of $8.7 billion in cash. Avexis’ lead candidate was AVXS-101, a gene therapy that in March met the primary endpoint in a phase II trial for spinal muscular atrophy (SMA) type 2. In September, the FDA recommended a pivotal confirmatory study to supplement existing data to treat older individuals with SMA.
It also boosts the big pharma’s work in ocular disease. Novartis’ Beovu (brolucizumab), an antibody fragment targeting VEGF-A for treating diabetic macular edema, had success in September when a phase III study met its primary endpoint of noninferiority for Beovu vs. aflibercept in mean change in best-corrected visual acuity at year one. The drug gained FDA approval in October 2019 in wet age-related macular degeneration. The Basel, Switzerland-based pharma used a priority review voucher to speed up the review process.
In April 2019, Novartis completed the spin-off of its eye care devices business, Alcon Inc., of Geneva. Shareholders in Novartis received one share of Alcon for every five shares or American depositary receipts held. Novartis didn't shepherd its investment in Alcon extremely well over the years, considering its loss in valuation. It gradually acquired pieces of Alcon over 2007 to 2011 for a total of about $51.6 billion. That means its original investment has eroded by almost half in the last decade or so. There is a caveat, though, since Alcon looks a bit different now than when it was acquired. Novartis merged its contact lens unit, Ciba vision, into Alcon, and it pulled out the ophthalmic pharmaceuticals into Novartis, where they remain.