Big Companies Keep Making Big Plays in Gene Therapy

Big Companies Keep Making Big Plays in Gene Therapy

When Allergan and Editas Medicine disclosed this week that they would move forward on their collaboration to develop a genome editing-medicine for treatment of an inherited retinal disorder, it was the latest move into genetic medicine by big ophthalmology players. Already, Novartis had made two significant moves in the gene therapy space, and Roche expanded a research agreement to develop genetic treatments in ophthalmology.

This week’s post, the first of two parts on gene therapy in ophthalmology, explores the moves the big players are making in the space. Next week, we’ll look at smaller companies that are also making moves in this arena.

Under the terms of their agreement, Allergan exercised its option to develop and commercialize EDIT-101, Editas’ experimental CRISPR genome editing-medicine, for the treatment of Leber congenital amaurosis type 10 (LCA10). Editas also exercised its option to co-develop and share equally in the profits and losses from EDIT-101 in the US. The two companies entered a strategic alliance and option agreement in March 2017, under which Allergan received exclusive access and the option to license up to five of Editas’ genome editing-programs for ocular diseases, including EDIT-101.

Spark Moves on Payment Models

With the only Food and Drug Administration-approved gene therapy for eye disease, Spark Therapeutics has spent the past eight months creating three payer programs for its Luxturna (voretigene neparvovec) for treatment of RPE65 mutation-associated retinal dystrophy, priced at $850,000 for treatment in both eyes. Those payer programs include:

  • An outcomes-based rebate arrangement with a long-term durability measure.
  • An innovative contracting model that aims to reduce risk and financial burden for payers and treatment centers.
  • A proposal to the Centers for Medicare and Medicaid Services to create an installment payment model.

Spark has reached an agreement in principle with Harvard Pilgrim to make Luxturna available under the outcomes-based rebate arrangement and the innovative contracting model, and has reached an agreement in principle with Express Scripts affiliates to enable the innovative contracting model.

“We believe that access to therapy is a shared responsibility among Spark Therapeutics, payers, health benefit providers, physicians, and treatment centers,” says Spark CEO Jeffrey D. Marrazzo. “We have been working with stakeholders across the healthcare sector to help ensure that appropriate patients have access to a product that challenges all of the current conventions of how patients are treated, how products are delivered, and how payments are handled.”

Novartis Makes Its Moves

In January, Novartis entered into an agreement with Spark to develop, register, and commercialize Luxturna outside the US, with Spark retaining US rights. The deal has Spark receiving $105 million as an up-front fee, and includes up to $65 million in milestone payments, as well as a royalty on net sales outside the US. FiercePharma quotes Jefferies analyst Michael Yee as saying that Novartis likely sees “meaningful commercial opportunity” in this deal, but that it also may be an “opportunity to jump-start payment discussions with EU regional authorities on the next generation of therapies (e.g., gene therapy and cellular therapy).”

More recently, Novartis made another move in the ophthalmic gene medicine space, buying AveXis, which has a drug in development for treatment of spinal muscular atrophy. A Reuters report says Novartis paid $8.7 billion for AveXis. Novartis hopes to leverage some of AveXis’ know-how in ophthalmology. “We have a robust internal portfolio of gene therapies in ophthalmology and neuroscience in Novartis Institutes for Biomedical Research,” says Novartis chief executive Vas Narasimhan, “and we look forward to using AveXis’ technical development capabilities to be able to advance that portfolio.”

Among the attributes of AveXis that Novartis finds intriguing is its scalable manufacturing to accelerate future gene therapy-programs and launches.

Roche Makes Its Play

Another company, 4D Molecular Therapeutics (4DMT), this year expanded a 2016 research agreement with Roche into a broad long-term partnership to develop and commercialize multiple ophthalmology products. 4DMT describes itself as “a leader in therapeutic vector evolution for adeno-associated virus (AAV) gene therapy vector discovery and product development.” 4DMT is based in the San Francisco Bay area, which is also home to Roche’s Genentech unit.

“We have created clear synergies between 4D’s vector discovery, gene therapy development, and manufacturing capabilities and Roche’s expertise in late-stage clinical development and global biologics commercialization,” says 4DMT CEO and co-founder David Kirn, MD. “The decision to expand our partnership represents validation of our ophthalmology platform, clinical candidates, and team.”

4DMT’s intravitreally delivered choroideremia clinical candidate, 4D-110, is the first collaboration program; investigational new drug-enabling studies and activities are under way. Additional clinical candidate development programs are also underway to treat retinal diseases with high unmet need.

Next week: Smaller players in gene therapy in ophthalmology.

For questions about this article, please contact Steve Lenier at