Novartis Expands Presence in Gene Therapy With Acquisition

– By Barry Cohen

Novartis AG (NYSE:NVS) is expanding its gene therapy footprint by acquiring a venture capital-backed company focusing on treating vision loss that can lead to blindness.

The Swiss pharmaceutical giant will pay shareholders of Vedere Bio $150 million upfront, which could be boosted by $130 million more if the Cambridge, Massachusetts-based company’s programs achieve several development milestones.

Novartis has made huge strides aimed at beefing up its gene therapy program. In 2018, the company bought AveXis for $8.7 billion, inheriting its spinal muscular atrophy therapy Zolgensma, and this year established partnerships with Dyno Therapeutics and Sangamo Therapeutics Inc. (NASDAQ:SGMO).

Vedere is trying to take naturally occurring, light-sensing proteins and use a special gene therapy method to inject them into a certain part of the eye. Once there, they will be sent to retinal cells that are still healthy to help improve the vision of patients, according to an article in BioPharma Dive.

Novartis thinks this approach has much broader uses. The company said the proteins, in conjunction with Vedere’s delivery tools, could “vastly expand” the number of treatable patients with vision loss from photoreceptor death.

Investors should keep in mind that the Vedere treatments are still in the pre-clinical stage so a great deal of development still needs to be done. Working with the acquisition’s scientists, Novartis is preparing to promptly begin human testing.

Novartis Expands Presence in Gene Therapy With Acquisition
Novartis Expands Presence in Gene Therapy With Acquisition

There could be an even bigger population the Vedere platform could address, beyond inherited eye disorders. That would be a new way to treat geographic atrophy, one of the more advanced forms of an age-related vision loss called dry age-related macular degeneration. The American Academy of Ophthalmology notes an estimated 15 million people in North America have AMD, the vast majority with the dry form.

Following the close of the deal, Vedere Bio II will operate as a wholly independent entity from Novartis and Vedere Bio.

Novartis decided to get out of one area of the eye-care business when it spun off Alcon Inc. (NYSE:ALC) last year. The company said it wanted to concentrate on treating diseases of the eye, not eye-care products.

Novartis’ eye drugs include Xidra, treating dry eye disease, and Beovu, which was recently approved for the wet form of AMD. Its third eye drug and the biggest producer is Lucentis, which had sales of $515 million in the third quarter. Xidra and Beovu combined to bring in revenue of about $151 million. Novartis’ biggest competitor is the Regeneron Pharmaceuticals Inc. (NASDAQ:REGN) drug Eylea, a multibillion-dollar annual seller.

The Novartis faithful are hoping for better days ahead. At about $90, the company’s shares are pretty much where they were five years ago. On the plus side, its dividend yields nearly 4%.

Despite the stock being stuck in neutral, Wall Street seems to think Novartis is a good investment, with three recent buy ratings and just one hold. Analysts give it an average target price of more than $106, according to

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Novartis to pursue SMA drug branaplam in Huntington’s disease

ZURICH (Reuters) – Novartis is seeking to repurpose its investigational oral spinal muscular atrophy (SMA) drug branaplam to treat Huntington’s disease, the Swiss drugmaker said on Wednesday, as it plans a clinical trial after winning U.S. orphan drug designation.

Novartis has been studying branaplam in SMA, but last year hinted that it would pivot to new neurological diseases after its head of research Jay Bradner said he did not see a “big opportunity” in its original disease indication.

The drug remains under study for SMA, but now Novartis said it wants to see if trials in humans can duplicate preclinical models in which branaplam was shown to reduce levels of a mutant protein that plays a role in Huntington’s disease, for which there are no approved disease modifying therapies that delay disease onset or slow progression.

“Novartis intends to start a development program for branaplam to determine if it has the potential to be a transformative treatment for people living with this devastating condition,” the company said in a statement.

Roche beat Novartis’s branaplam to the market with an oral SMA therapy, Evrysdi, which was approved earlier this year.

(Reporting by John Miller, editing by John Revill)

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