Rocket Pharmaceuticals is looking to expand its gene therapy pipeline, exec says

Provided by Mergermarket

Rocket Pharmaceuticals [NASDAQ: RCKT] is looking to grow its gene therapy pipeline, including by bringing in additional assets, according to Claudine Prowse, head of corporate development.

The New York-based drug developer is looking to potentially add early-stage assets in the area of adeno-associated viruses (AAV), she said.

Prowse declined to specify when the company could look to bring in additional assets, noting that it would be when the firm has capacity to do so. Rocket is also interested in acquiring potential technologies that could help its pipeline, she said.

The company reported cash and cash equivalents of USD 159.5m as of 30 September. Rocket has raised USD 175m to date through a reverse-merger with Inotek Pharmaceuticals in September 2017 that resulted in a listing on the NASADQ and a subsequent follow-on offering, according to Prowse.

Rocket has also received a lot of interest from potential suitors, she said, but stated that the company is looking to build its value and to grow the business to become a larger biotech company such as Bluebird Bio [NASDAQ: BLUE], which had a recent market cap of USD 7.2bn.

Rocket currently has five programs. Its only clinical stage drug, RP-L102, is in Phase I as a potential treatment for fanconi anemia, a rare disease that affects bone marrow. The drug developer anticipates that it will have four additional programs in the clinic by the end of next year. The company is pursuing gene therapy targets across two platforms: ex-vivo lentriviral vectors, a type of retrovirus, as well as in-vivo AAV program.

Gene therapy has become one of the most closely watched subsectors in drug development in recent years, with pharma eyeing high price tags for launch products and doling out large sums to acquire new technology platforms in this emerging field.

In April, Novartis [NASDAQ: NVS] acquired AveXis for USD 8.7bn. The Swiss drugmaker recently announced that AveXis’ gene therapy for spinal muscular atrophy could come with a list price of at least USD 4m, which would make it the most expensive drug in the world.

Novartis is not alone in its efforts to expand into gene therapy. In 2016, Pfizer [NYSE: PFE] acquired AAV gene therapy company Bamboo Therapeutics for USD 150m up front and potential milestone payments of USD 495m. Sanofi [NYSE: SNY], too, announced a deal in 2015 with gene therapy developer Voyager Therapeutics [NASDAQ: VYGR] for USD 100m up front and up to USD 745m in milestones.

US regulators are also showing a willingness to help bring gene therapies to market. FDA commissioner Scott Gottlieb said at the 2018 BIO Convention in Boston that the agency expects to approve 40 gene therapies by 2022.

To that end, the agency issued six draft guidance documents in July to establish a framework for approving these novel treatments. The FDA regulator also released three additional disease-specific draft guidance for gene therapies in hemophilia, retinal disorders and rare diseases.

There have been three FDA approved gene therapies to date: Spark Therapeutics’ [NASDAQ: ONCE] retinal dystrophy AAV- based gene therapy Luxturna, Novartis’ genetically modified cell therapy Kymriah and its competitor Gilead Sciences’ [NASDAQ: GILD] Yescarta.

Rocket Pharmaceuticals was recently trading at USD 17.5 per share, with a market cap of USD 694m.

The company was advised by Gibson, Dunn & Crutcher for its deal with Inotek.

by Kevin McCaffrey

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