Press Release: Novartis: AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in...

Novartis International AG / AveXis expands world-leading gene therapy

manufacturing capacity with purchase of advanced biologics therapy

manufacturing campus in Longmont, Colorado. Processed and transmitted

by West Corporation. The issuer is solely responsible for the content of

this announcement.

-- Facility to become the largest of four state-of-the-art sites involved in

manufacturing of AveXis gene therapies for pipeline of rare genetic

diseases including spinal muscular atrophy

-- AveXis plans to offer positions to all approximately 150 employees

previously employed at the site, and to announce further expansion of new

jobs in the near term

-- Investment in the Longmont campus adds to existing $115 million

investment in Durham facility, leading to creation of more than 1,000

US-based, high-tech, biologics manufacturing jobs by the end of 2019

Basel, April 1, 2019 -- AveXis, a Novartis company, today announced it

signed an agreement to purchase an advanced biologics therapy

manufacturing campus in Longmont, Colorado, further expanding AveXis'

production capacity as it prepares to launch Zolgensma(R) (onasemnogene

abeparvovec-xioi[1]) an investigational gene therapy awaiting global

regulatory approvals for the treatment of spinal muscular atrophy (SMA)

Type 1 and for future gene therapy treatments in development.

AveXis currently has a fully-operational state-of-the-art manufacturing

facility in Illinois, is building a facility in North Carolina scheduled

to be operational in 2020 and is expanding its product development

capacity at its San Diego facility. The addition of the six-building

Longmont campus consists of nearly 700,000 square-feet of space for

biologic drug manufacturing, offices, laboratories, warehousing and

utilities. Initial start-up activities in Longmont will include

preparing the facility for scaling, manufacturing and testing of gene

therapies and hiring staff.

"Our Longmont, Colorado, campus, along with our existing manufacturing

sites in Illinois, California and North Carolina, will play a crucial

role in helping us achieve the future manufacturing capacity required to

meet the global patient need for novel gene therapies," said Andrew

Knudten, Senior Vice President, Global Strategic Operations. "We have

built a team with exceptional depth of experience, unified by a common

mission: to positively impact the lives of patients and families

devastated by rare and life-threatening neurological genetic diseases.

We are eager to add the talented team in Longmont to AveXis, and we hope

that they will choose to join us as we build world-leading manufacturing

capabilities in gene therapy."

"AveXis' success requires not just medical breakthroughs, but

innovations in R&D and manufacturing. With the opening of our fourth

location in the US, we will create more than 1,000 high-tech biologics

manufacturing jobs by the end of 2019," said Dave Lennon, President.

"AveXis has now established leading technical manufacturing capabilities

with the capacity to deliver our robust pipeline, as well as the

flexibility to enter into multiple external partnerships as the

development and manufacturing partner of choice in gene therapy."

About Zolgensma(R)

Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) is an

investigational gene therapy currently in development as a one-time

infusion for SMA Type 1. Zolgensma is designed to address the monogenic

root cause of SMA and prevent further muscle degeneration. Zolgensma

represents the first in a proprietary platform to treat rare, monogenic

diseases using gene therapy. In December, the FDA accepted the company's

Biologics License Application for use of Zolgensma with SMA Type 1

patients. The drug previously received Breakthrough Therapy designation

and has been granted Priority Review by the FDA, with regulatory action

anticipated in May 2019. In addition, the drug is anticipated to receive

approval in Japan and the European Union later this year.

About SMA

SMA is a severe neuromuscular disease characterized by the loss of motor

neurons leading to progressive muscle weakness and paralysis. SMA is

caused by a genetic defect in the SMN1 gene that codes SMN, a protein

necessary for survival of motor neurons. The incidence of SMA is

approximately one in 10,000 live births and is the leading genetic cause

of infant mortality. The most severe form of SMA is Type 1, a lethal

genetic disorder characterized by rapid motor neuron loss and associated

muscle deterioration, which results in mortality or the need for

permanent ventilation support by 24 months of age for more than 90

percent of patients.

Disclaimer

This press release contains forward-looking statements within the

meaning of the United States Private Securities Litigation Reform Act of

1995. Forward-looking statements can generally be identified by words

such as "to become," "plans," "to announce," "in the near term,"

"leading to," "awaiting," "building," "scheduled," "expanding," "eager,"

"hope," "anticipated," "potential," "can," "will," "expect," "anticipate,

" "look forward," "believe," "committed," "investigational," "pipeline,"

"launch," or similar terms, or by express or implied discussions

regarding the acquisition of the manufacturing campus in Longmont,

Colorado; regarding potential strategic benefits or opportunities from

the acquisition; regarding potential marketing approvals, new

indications or labeling for the investigational products described in

this press release, or regarding potential future revenues from such

products. You should not place undue reliance on these statements. Such

forward-looking statements are based on our current beliefs and

expectations regarding future events and are subject to significant

known and unknown risks and uncertainties. Should one or more of these

risks or uncertainties materialize, or should underlying assumptions

prove incorrect, actual results may vary materially from those set forth

in the forward-looking statements. There can be no guarantee that

Novartis will be able to realize any of the potential strategic benefits

or opportunities as a result of the acquisition of the manufacturing

campus in Longmont, Colorado within any particular time frame, or at

all. Neither can there be any guarantee that the investigational

products described in this press release will be submitted or approved

for sale or for any additional indications or labelling in any market,

or at any particular time. Nor can there be any guarantee that such

products will be commercially successful in the future. In particular,

our expectations regarding such products and the acquisition of the

manufacturing campus in Longmont, Colorado could be affected by, among

other things, the uncertainties inherent in research and development,

including clinical trial results and additional analysis of existing

clinical data; regulatory actions or delays or government regulation

generally; the potential that the strategic benefits or opportunities

expected from the acquisition may not be realized or may take longer to

realize than expected; global trends toward health care cost containment,

including government, payor and general public pricing and reimbursement

pressures and requirements for increased pricing transparency; our

ability to obtain or maintain proprietary intellectual property

protection; the particular prescribing preferences of physicians and

patients; general political and economic conditions; safety, quality or

manufacturing issues; potential or actual data security and data privacy

breaches, or disruptions of our information technology systems, and

other risks and factors referred to in Novartis AG's current Form 20-F

on file with the US Securities and Exchange Commission. Novartis is

providing the information in this press release as of this date and does

not undertake any obligation to update any forward-looking statements

contained in this press release as a result of new information, future

events or otherwise.

About AveXis

AveXis, a Novartis company, is dedicated to developing and

commercializing novel treatments for patients suffering from rare and

life-threatening neurological genetic diseases. Our initial product

candidate, Zolgensma, is its proprietary gene therapy currently in

development for the treatment of spinal muscular atrophy, or SMA. In

addition to developing Zolgensma to treat SMA, AveXis also plans to

develop other novel treatments for rare neurological diseases, including

Rett syndrome and a genetic form of amyotrophic lateral sclerosis caused

by mutations in the superoxide dismutase 1 (SOD1) gene. For additional

information, please visit www.avexis.com.

About Novartis

Novartis is reimagining medicine to improve and extend people's lives.

As a leading global medicines company, we use innovative science and

digital technologies to create transformative treatments in areas of

great medical need. In our quest to find new medicines, we consistently

rank among the world's top companies investing in research and

development. Novartis products reach more than 800 million people

globally and we are finding innovative ways to expand access to our

latest treatments. About 130,000 people of nearly 150 nationalities work

at Novartis around the world. Find out more at www.novartis.com.

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References

[1] The brand name Zolgensma(R) (onasemnogene abeparvovec-xioi) has

been provisionally approved by the FDA for the investigational product

AVXS-101, but the product itself has not received marketing

authorization or BLA approval from any regulatory authorities.

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Novartis Media Relations

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April 01, 2019 16:05 ET (20:05 GMT)