The week in industry: Novartis responds to FDA statement on data integrity of ZOLGENSMA®

This week: Automated sample preparation system for cryo-electron microscopy released, orphan drug designation granted for an investigational treatment for Dravet syndrome and exosome patents issued in Europe and the US.

Aug 08, 2019
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This week's industry news:  

Novartis responds to FDA statement on data integrity of ZOLGENSMA® Automated sample preparation system for cryo-EM released
Orphan drug designation granted for investigational treatment for Dravet syndrome
Exosome patents granted in Europe and the US


Novartis responds to FDA statement on data integrity of ZOLGENSMA®

After the US FDA released comments questioning the integrity of data of ZOLGENSMA® from Novartis (Basel, Switzerland), indicating that some data may have been manipulated, Novartis have responded that they are completely confident in the safety, quality and efficacy of the drug.

The FDA’s statement comes after AveXis (IL, USA) disclosed to the FDA – and subsequently other health authorities – that some data submitted may have not been as accurate as previously thought. However, despite this the FDA has said they still supports the continued marketing of Zolgensma.

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Automated sample preparation system for cryo-EM released

TTP Labtech (Cambridge, UK), has introduced chameleon, an automated sample preparation system for cryo-electron microscopy (cryo-EM). The company has developed the system to allow the reliable and scalable application of cryo-EM in protein research for drug discovery. Automation – vs the standard approach – negates the need to undergo the technically demanding and often manual process of sample preparation.

“We are extremely excited to be launching the chameleon system, which enables high quality, repeatable preparation of samples for cryo-electron microscopy. This technology will enable scientists to improve data quality and significantly reduce the time required to prepare protein samples for imaging,” commented David Newbie, Managing Director of TTP Labtach.

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Orphan drug designation granted for investigational treatment for Dravet syndrome

The US FDA has granted orphan drug designation to STK-001, the lead product candidate from Stoke Therapeutics (MA, USA). This investigational drug – a proprietary antisense oligonucleotide – is indicated for the treatment of Dravet syndrome, a genetic form of epilepsy. This decision from the FDA comes after preclinical data from Stoke demonstrates the proof-of-mechanism for STK-001.

“The need for a medicine that will treat the underlying cause of Dravet syndrome is clear to anyone who has seen the devastating effects of the disease, and the significant impact it has on patients and their families,” commented Barry S. Ticho, CMO of Stoke. “Our goal with STK-001 is to slow or even stop disease progression by treating the underlying cause of Dravet syndrome. STK-001 is designed to selectively upregulate one allele of the SCN1A gene to restore the protein expression to near-normal levels. We are on track to submit our investigational new drug application for STK-001 to the FDA in early 2020 and to begin a Phase I/II clinical study in the first half of the year.”

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Exosome patents granted in Europe and the US

In a move that will reinforce the company’s position within exosome-mediated RNA therapeutics and targeted exosome drug delivery space, Evox Therapeutics (Oxford, UK) has been granted three patents in both the US and Europe. Two of the three patents cover exosomes containing genetic material (e.g. RNAi agents) and targeting exosomes to tissues of interest – both of these being in the US. The latest patent has been issued in Europe and gives exclusive rights to the company for the loading of exogenous genetic materials into exosomes, as well as targeted exosome-mediated delivery to tissues and organs of interest.

“We are pleased to have been granted these important, foundational patents that provide significant protection for our internal and partnered exosome therapeutics in the US and Europe. Over the past decade the Company and its founders have built a broad and deep intellectual property estate covering the key applications of exosome therapeutics, including delivery of RNA and protein-based drugs, targeted exosome delivery, and exosome manufacturing,” commented Antonin de Fougerolles, CEO of Evox.

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