Novartis' CTL109 Gets Breakthrough Therapy Designation from the FDA - Analyst Blog


Novartis ( NVS ) announced that the FDA has granted Breakthrough Therapy Designation to its pipeline candidate, CTL019.

Novartis is evaluating CTL019, an experimental chimeric antigen receptor (CAR) therapy, for the treatment of pediatric and adult patients suffering from relapsed/refractory acute lymphoblastic leukemia (r/r ALL).

CTL019 uses CAR technology to reprogram a patient's own T cells to look for cancer cells which express specific proteins, called CD19. Once reprogrammed, the T cells (called CTL019 after reprogramming) are released into the patient's blood so as to proliferate and bind to the targeted CD19+ cancer cells and destroy them.

The filing was submitted by the University of Pennsylvania's Perelman School of Medicine. We note that the university has an exclusive global agreement with Novartis to research, develop and commercialize personalized CAR T cell therapies for the treatment of cancers. The university is currently conducting phase I/II trials on CTL019.

These studies are evaluating the activity and safety of CTL019 in patients with resistant or refractory CD19+ hematologic malignancies, specifically pediatric and adult acute lymphoblastic leukemia and chronic lymphocytic leukemia.

The designation from the FDA should expedite the development and review of the candidate.The designation not only includes all of the fast track program features but a more intensive FDA guidance as well.

In order to focus on advancing innovative cell-based therapies, including the development of CARs, Novartis has established the Cell and Gene Therapies Unit. Moreover, Novartis holds the worldwide rights to CARs which are being developed through the collaboration with Penn for all cancer indications.

We are impressed by Novartis' efforts to realign its portfolio in order to focus on its core business of pharmaceuticals, eye care and generics. The acquisition of oncology products from GlaxoSmithKline ( GSK ) and divestment of Vaccines business is a step in the right direction. It will broaden Novartis' portfolio and enable it to focus better on its core capabilities besides contributing immensely to the top line. Margins are also expected to get a significant boost.

Novartis, a large cap pharma, currently carries a Zacks Rank#3 (Hold). Some better-ranked large cap pharmas include AbbVie, Inc . ( ABBV ) and Johnson & Johnson ( JNJ ). Both the stocks carry a Zacks Rank #2 (Buy).

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of The NASDAQ OMX Group, Inc.

This article appears in: Investing , Business , Stocks

Referenced Stocks: CAR , NVS , JNJ , GSK , ABBV

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