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Novartis (NVS) Announces Data on Zolgensma, Acquires Coalesce

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Novartis (NVS - Free Report) has announced new positive data on Zolgensma (onasemnogene abeparvovec), a one-time treatment for spinal muscular atrophy (SMA), Zolgensma (onasemnogene abeparvovec).

Zolgensma is an approved gene therapy for the treatment of SMA, a rare, genetic neuromuscular disease.

The late-stage study SPR1NT showed that children with three copies of the SMN2 back-up gene who were treated presymptomatically achieved age-appropriate motor milestones, including standing and walking.

In addition, a descriptive post-hoc analysis of START, STR1VE-EU and STR1VE-US (n=65) showed that children with SMA Type 1 achieved or maintained important measures of bulbar function following treatment with Zolgensma, including the ability to speak, swallow and meet nutritional needs and maintain airway protection.

In the three-copy cohort of SPR1NT, 93% of children went on to walk independently, most (11/15, 73 percent) within the World Health Organization’s window of normal development.  However, without treatment, most children with three copies of the SMN2 back-up gene develop SMA Type 2, which is characterized by the inability to walk independently.  Zolgensma is now approved in more than 40 countries.

Shares of Novartis have lost 3.4% in the year so far compared with the industry’s 3.1%  decline.

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Additionally, Novartis’ generic division, Sandoz, announced that it has acquired respiratory device company Coalesce Product Development Limited.

Coalesce is a U.K.-based medical and drug delivery device development company.

The financial terms of the transaction were not disclosed.

Sandoz has acquired the capabilities and assets of Coalesce, which will enable it to strengthen its existing portfolio of respiratory medicines and further develop the pipeline. The company currently has an existing portfolio of six in market-products in its respiratory franchise.

We remind investors that Novartis is still evaluating whether to retain or sell its Sandoz business and expects to provide an update on the same by the end of this year.

Novartis has streamlined its business significantly and looking forward to focusing on its core business.

Earlier in the month, Novartis also announced an option agreement with gene therapy company Voyager Therapeutics (VYGR - Free Report) . Under the terms of the agreement, Novartis has the right to evaluate novel capsids from Voyager’s TRACER platform and exercise options to license capsids for exclusive use with specific targets in its development of AAV gene therapies.  NVS also gets options to access capsids for two additional targets.

In exchange, Voyager will receive $54 million upfront and is entitled to get up to $37.5 million as exercise fees for options for three initial CNS targets, which can be exercised by Novartis within 12 months of signing.

Novartis may also elect to evaluate capsids for up to two additional targets. The development of additional drugs and treatments bode well for Novartis.

Novartis currently carries a Zacks Rank #3 (Hold).

Some better-ranked stocks in the healthcare space are Vertex Pharmaceuticals (VRTX - Free Report) and BioDelivery Sciences , both carrying a Zacks Rank #2 (Buy) at present. You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

Vertex Pharmaceuticals’ earnings per share estimates have increased to $14.52 from $13.39 for 2022 over the past 60 days. Shares of Vertex have gained 9.7% in the past year.

BioDelivery Sciences’ earnings per share estimates for 2022 have increased from 33 cents to 38 cents in the past 30 days. Shares of BDSI have surged 79.7% year to date.

Earnings of BioDelivery Sciences beat estimates in three of the last four quarters, missing the mark once, the average surprise being 33.7%.


 


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