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10/30/2019 Novartis announces AVXS-101 intrathecal study update

Basel, October 30, 2019 � Novartis today announced the United States Food & Drug Administration (FDA) placed a partial hold on clinical trials for intrathecal administration of AVXS-101.

10/05/2019 AveXis presents updated STRONG data at WMS demonstrating a higher mean increase in Hammersmith Functional Motor Scale-Expanded (HFMSE) scores among older SMA Type 2 patients following one-time intrathecal administration of AVXS-101

Basel, October 5, 2019 – AveXis, a Novartis company, today announced new interim data from the Phase 1/2 STRONG study for intrathecal (IT) administration of AVXS-101, demonstrating older patients (> 2 years and < 5 years) with spinal muscular atrophy

09/24/2019 Novartis Statement on Form 483 posting by US Food and Drug Administration (FDA)

Today the FDA posted information provided by the company to the FDA in response to its Form 483 issued on August 2, 2019. Our submission, which can be read here at ( https://www.fda.gov/media/131007/download), reiterated our firm commitment to data

09/19/2019 AveXis presents new data at EPNS continuing to show significant therapeutic benefit of Zolgensma® in prolonging event-free survival now up to 5 years of age in patients with spinal muscular atrophy (SMA) Type 1

BASEL, Switzerland , Sept. 19, 2019 /PRNewswire/ -- New interim data from SPR1NT study supports critical importance of early intervention in pre-symptomatic SMA patients, leading to age appropriate major milestone gain Updated results from global STR1VE study demonstrate that Zolgensma®

08/14/2019 AveXis Statement on Changes to Senior Leadership Team

AveXis shares Page Bouchard, DVM named Senior Vice President of Research and Chief Scientific Officer, AveXis, effective August 5, 2019. Dr Bouchard is a 27 year industry veteran with experience in well over 100 Investigational New Drug programs and dozens of New Drug Application/Biologics License

08/06/2019 Novartis stands behind Zolgensma® (onasemnogene abeparvovec- xioi) for the treatment of children less than 2 years of age with spinal muscular atrophy

Basel, August 6, 2019 – Today the FDA released a statement addressing data integrity issues with the Biologics License Application (BLA) for Zolgensma® (onasemnogene abeparvovec-xioi). First and foremost, we are fully confident in the safety, quality and efficacy of Zolgensma.

07/25/2019 AveXis Statement on Access to Zolgensma® (onasemnogene abeparvovec-xioi)

The FDA's May 2019 approval of Zolgensma®, a gene therapy for spinal muscular atrophy (SMA) in pediatric patients less than 2 years of age, marked an important milestone within the SMA community. Zolgensma offers a new treatment option for children with SMA and their families.

05/24/2019 AveXis Announces Innovative Zolgensma® Gene Therapy Access Programs for US Payers and Families

One-time treatment with Zolgensma (onasemnogene abeparvovec-xioi) is designed to replace lifetime of chronic therapy for all pediatric patients with SMA Annualized cost of Zolgensma is USD 425,000 per year for 5 years: 50% less than multiple established value-based pricing benchmarks including

05/24/2019 AveXis receives FDA approval for Zolgensma®, the first and only gene therapy for pediatric patients with spinal muscular atrophy (SMA)

SMA is a rare genetic disease that leads to progressive muscle weakness, paralysis and, when left untreated in its most severe form, permanent ventilation or death for most patients by age 2 [1],[2] Zolgensma (onasemnogene abeparvovec-xioi) is approved for the treatment of pediatric patients less

05/07/2019 New AveXis data at AAN showed long-term durability of Zolgensma® in patients with spinal muscular atrophy (SMA) Type 1

Interim long-term follow-up data showed all enrolled Cohort 2 patients maintained motor function and milestones achieved during the Phase 1 START trial Mean age of follow-up since dosing with Zolgensma ( onasemnogene abeparvovec-xioi; AVXS-101) was nearly four years, with some patients nearing

05/05/2019 AveXis presented robust data at AAN demonstrating efficacy of Zolgensma® in broad spectrum of spinal muscular atrophy (SMA) patients

Interim data reported for the first time from STRONG in SMA Type 2 showed rapid motor function gains and milestone achievements with intrathecal Zolgensma (onasemnogene abeparvovec-xioi; AVXS-101) - New interim data from STR1VE in SMA Type 1 continued to show prolonged event-free survival,

04/16/2019 AveXis data reinforce effectiveness of Zolgensma® in treating spinal muscular atrophy (SMA) Type 1

Ph 3 STR1VE data show prolonged event-free survival, early and rapid increases in CHOP-INTEND and significant milestone achievement in SMA Type 1, consistent with START trial First-in-human biodistribution data show transduction in intended CNS targets and widespread SMN expression comparable

04/01/2019 AveXis expands world-leading gene therapy manufacturing capacity with purchase of advanced biologics therapy manufacturing campus in Longmont, Colorado

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

12/03/2018 Novartis announces FDA filing acceptance and Priority Review of AVXS-101, a one-time treatment designed to address the genetic root cause of SMA Type 1

The AVXS-101, now known as ZOLGENSMA® (onasemnogene abeparvovec-xxxx) 1 , filing is supported by data from the START trial which demonstrated a dramatic increase in survival and transformative improvement in achievement of developmental milestones compared to the natural history of SMA Type 1 2 SMA

05/03/2018 AveXis Reports First Quarter 2018 Financial and Operating Results

Significant progress across operating functions including licensing, clinical trials and regulatory interactions -- -- AVXS-101 pre-BLA meeting with FDA is scheduled to be held in June 2018 -- CHICAGO , May 03, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc.

05/01/2018 AveXis Provides Update on Proposed Acquisition by Novartis AG

CHICAGO , May 01, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the required waiting period under the Hart-Scott-Rodino

04/25/2018 AveXis Announces First Patient Dosed in Phase 3 Trial of AVXS-101 in Pre-Symptomatic SMA Types 1, 2 and 3

CHICAGO , April 25, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that the first patient has been dosed in a Phase 3 trial

04/24/2018 AveXis Presents Initial Data from Pivotal U.S. Trial for SMA Type 1 and 24-Month Follow-Up Data from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

A mean increase in CHOP-INTEND of 17.3 was observed at three months post gene therapy in SMA Type 1 U.S. pivotal trial – – All patients in the therapeutic dose cohort in the Phase 1 trial were alive and event-free at 24-month follow-up – – Patients observed in long-term follow-up from the Phase 1

04/19/2018 AveXis to Present AVXS-101 Data at the Annual Meeting of the American Academy of Neurology

Includes Initial Data from SMA Type 1 Pivotal Trial (STR1VE) and 24-Month Follow-Up Data from Phase 1 Trial CHICAGO , April 19, 2018 (GLOBE NEWSWIRE) -- AveXis , Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening

04/09/2018 AveXis Enters Agreement to be Acquired by Novartis AG for $8.7 billion

Novartis to acquire AveXis for $218 per share in cash AveXis’ lead product candidate, AVXS-101, expected to enhance Novartis’s position as a gene therapy and neuroscience leader Transaction expected to be completed in mid-2018 CHICAGO , April 09, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc.

03/27/2018 AveXis Gene Therapy Awarded SAKIGAKE Designation for Spinal Muscular Atrophy Type 1

CHICAGO , March 27, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that Japan’s Ministry of Health, Labour and Welfare (MHLW)

03/13/2018 AveXis Enters into Licensing Agreement with Genethon

Includes exclusive worldwide rights to AAV9-SMN product and route of administration CHICAGO and ÉVRY, France , March 13, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) and Genethon today announced they have entered into an exclusive, worldwide license agreement for in vivo gene therapy

02/27/2018 AveXis Reports Fourth Quarter and Full Year 2017 Financial and Operating Results

On track to request pre-BLA meeting with FDA in Q2 2018 – – Intends to initiate pivotal trial in SMA Type 1 in Europe and multi-national pre-symptomatic SMA study in the first half of 2018 – – Anticipates IND submissions for Rett syndrome and genetic ALS in late 2018/early 2019 – – Conference

02/20/2018 AveXis to Report Fourth Quarter and Full Year 2017 Financial and Operating Results

Conference call and webcast on February 27 at 4:30 p.m. EST CHICAGO , Feb. 20, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report financial

01/30/2018 AveXis to Initiate Screening for Remaining Patients in Pivotal Trial of AVXS-101 for SMA Type 1 Following Review of Preliminary Data from First Three Patients

CHICAGO , Jan. 30, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that, following review of safety data and early signals of

01/22/2018 AveXis Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

CHICAGO , Jan. 22, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the closing of its previously announced underwritten public offering of 4,509,840 shares of its common stock at a price to the public of $102.00 per share before underwriting discounts and commissions, including

01/16/2018 AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO , Jan. 16, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, up to $400 million of shares of its common stock in an underwritten public offering. The offering is subject to market and other conditions, and

01/16/2018 AveXis Announces Proposed Public Offering of Common Stock

CHICAGO , Jan. 16, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 3,921,600 shares of its common stock at a public offering price of $102.00 per share, before underwriting discounts and commissions.

01/16/2018 AveXis Announces Expanded Clinical Development Program for AVXS-101 in Spinal Muscular Atrophy

– Company to expand study of AVXS-101 into additional SMA populations including pre-symptomatic, older pediatric Type 2 and Type 3 SMA patients – – First patient dosed in Phase 1 trial of AVXS-101 in SMA Type 2 – CHICAGO , Jan. 16, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc.

01/08/2018 REGENXBIO and AveXis Announce Expansion of Relationship through Amended License Agreement for the Development and Commercialization of Treatments for Spinal Muscular Atrophy

AveXis acquires exclusive rights to entire NAV Technology Platform for the development of treatments for SMA Amended agreement permits assignment by AveXis upon a change of control without REGENXBIO’s consent REGENXBIO could receive up to $260 million , including $140 million in guaranteed upfront

01/04/2018 AveXis Announces Alignment with FDA on Next Steps Toward a BLA Submission for AVXS-101 in SMA Type 1

Company to submit information requested by FDA to the IND on an on-going basis – – AveXis plans to request a pre-BLA meeting in Q2 2018 – – Conference call and webcast today at 4:30 pm EST – CHICAGO , Jan. 04, 2018 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy

12/13/2017 AveXis Announces Plan to Initiate Phase 1 Trial in SMA Type 2 Utilizing Intrathecal Delivery of AVXS-101

– The FDA notified AveXis it may initiate the Phase 1 trial in SMA Type 2 based on a review of data provided by the company; trial to commence immediately – – The trial will evaluate safety, dosing and proof of concept for efficacy – – The trial will use product produced

11/09/2017 AveXis Reports Third Quarter 2017 Financial and Operating Results

– First patient has been dosed in pivotal trial of AVXS-101 for SMA Type 1 – – Conference call and webcast November 9 at 4:30 p.m. EST – CHICAGO , Nov. 09, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from

11/02/2017 AveXis to Report Third Quarter 2017 Financial and Operating Results

Conference call and webcast on November 9 at 4:30 p.m. EST CHICAGO , Nov. 02, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report financial

11/01/2017 AveXis Announces New England Journal of Medicine Publication of Phase 1 Data of AVXS-101 Gene Replacement Therapy in Spinal Muscular Atrophy Type 1

– Patients who received a single dose of AVXS-101 resulted in longer survival, superior achievement of motor milestones, and better motor function than in historical cohorts – – As of August 7, 2017 , all patients are alive, event-free and have reached at least 20 months of age – – As of August 7,

10/03/2017 AveXis to Report Top-line Data from the Phase 1 Clinical Trial of AVXS-101 in SMA Type 1 at the International Annual Congress of the World Muscle Society

– All patients were alive and event-free at 20 months of age as of the August 7, 2017 data cut-off – – Patients in Cohort 2 continue to demonstrate improvements in motor milestones – SAINT MALO, France , Oct. 03, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy

09/29/2017 AveXis Announces Plan to Initiate Pivotal Trial of AVXS-101 in SMA Type 1 Using Product from New GMP Commercial Process

– FDA notified AveXis it may initiate pivotal trial based on a review of data provided by the company following CMC Type B meeting – – Product used in the pivotal trial will be from the AveXis GMP manufacturing process; pivotal trial to commence immediately – –

08/10/2017 AveXis Reports Second Quarter 2017 Financial and Operating Results

– AveXis on track to submit potency assay data to FDA in August – – Conference call and webcast August 10 at 4:30 p.m. EDT – CHICAGO , Aug. 10, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and

08/03/2017 AveXis to Report Second Quarter 2017 Financial and Operating Results

-- Conference call and webcast on August 10 at 4:30 p.m. EDT -- CHICAGO , Aug. 03, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report

06/26/2017 AveXis Announces Closing of Public Offering of Common Stock and Full Exercise of Underwriters’ Option to Purchase Additional Shares

CHICAGO , June 26, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the closing of its previously announced underwritten public offering of 4,111,250 shares of its common stock at a price to the public of $70.00 per share before underwriting discounts and commissions, including

06/20/2017 AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO , June 20, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 3,575,000 shares of its common stock. The net proceeds to AveXis from the offering, after deducting the underwriting discounts and commissions and estimated

06/19/2017 AveXis Announces Proposed Public Offering of Common Stock

CHICAGO , June 19, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, up to $200 million of shares of its common stock in an underwritten public offering. The offering is subject to market and other conditions, and

06/14/2017 AveXis Announces Alignment with FDA on GMP Commercial Manufacturing Process for AVXS-101

– Agency is supportive of proposed analytical methods and comparability protocol – – AveXis anticipates initiating U.S. SMA Type 1 and Type 2 trials in Q3 2017 with product from new process – – AveXis manufacturing facility is fully operational for GMP product production – – Conference call and

06/07/2017 AveXis and REGENXBIO Announce New Exclusive Worldwide Licenses for the Treatment of Two Rare Neurological Monogenic Disorders Using NAV AAV9 Vector

REGENXBIO grants AveXis new licenses to NAV AAV9 vector for the development and commercialization of treatments for Rett syndrome (RTT) and amyotrophic lateral sclerosis (ALS) CHICAGO and ROCKVILLE, Md. , June 07, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) and REGENXBIO Inc.

05/11/2017 AveXis Reports First Quarter 2017 Financial and Operating Results

– Conference call of April 25 was in lieu of Q1 2017 earnings call – CHICAGO , May 11, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today reported

05/04/2017 AveXis to Report First Quarter 2017 Financial and Operating Results

Results to be reported on May 11, 2017 CHICAGO , May 04, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will report financial and operating results

04/25/2017 AveXis Presents Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at the Annual Meeting of the American Academy of Neurology

– 10 of 12 patients on proposed therapeutic dose sat unassisted; patient videos demonstrate motor milestone achievement not seen in natural history of disease – – 86% of patients without feeding support and 70% of patients without respiratory support prior to dosing continue without any support –

04/18/2017 AveXis to Host Webcast of Preclinical and Clinical Studies of AVXS-101 as Presented at the Annual Meeting of the American Academy of Neurology

– Includes Results from Phase 1 Trial of AVXS-101 in SMA Type 1 – – Conference call and webcast Tuesday, April 25 at 4:30 p.m. EDT – CHICAGO , April 18, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare

03/16/2017 AveXis Reports Topline Results from Phase 1 Trial of AVXS-101 in SMA Type 1 and Fourth Quarter and Full Year 2016 Financial and Operating Results

– No new treatment-related safety or tolerability findings – – No new events reported and 15 of 15 patients event-free at 13.6 months of age; majority of patients receiving proposed therapeutic dose sit unassisted – – Conference call and webcast March 16 at 4:30 p.m.

03/09/2017 AveXis to Report Topline Phase 1 Trial Results in SMA Type 1 and Fourth Quarter and Full Year 2016 Financial and Operating Results on March 16, 2017

-- Conference call and webcast on March 16 at 4:30 p.m. EDT -- CHICAGO , March 09, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce

03/02/2017 AveXis to Present Results from Phase 1 Trial of AVXS-101 in SMA Type 1 at AAN Annual Meeting

-- Key preclinical and clinical data of AVXS-101 in SMA to be presented -- CHICAGO , March 02, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today

02/15/2017 AveXis Appoints Rick Modi as Senior Vice President and Chief Business Officer

CHICAGO , Feb. 15, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the appointment of Rick Modi to the executive management team as

02/06/2017 AveXis Announces Single-Arm Design for European Pivotal Study of AVXS-101 in SMA Type 1 Patients

Company provides update following receipt of Scientific Advice response from the EMA CHICAGO , Feb. 06, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic

01/31/2017 AveXis Gene Therapy AVXS-101 Granted Access into EMA PRIME Program for Spinal Muscular Atrophy Type 1

PRIME intended to optimize development and speed evaluation of AVXS-101 through enhanced interactions and early dialogue with EMA CHICAGO , Jan. 31, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and

01/03/2017 AveXis to Present at the 35th Annual J.P. Morgan Healthcare Conference

CHICAGO , Jan. 03, 2017 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced Sean Nolan , President and Chief Executive Officer, will present a

12/21/2016 AveXis to Use Intended Commercial GMP Product in SMA Type 2 Study

– SMA Type 2 study to initiate in Q2 2017 – – Streamlines development of Type 2 clinical data set – – Conference call and webcast December 21 , at 4:30 p.m. EST – CHICAGO , Dec. 21, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for

11/10/2016 AveXis Reports Third Quarter 2016 Financial and Operating Results

– AVXS-101 demonstrated continued motor function improvement in ongoing study; majority of patients on proposed therapeutic dose achieved key developmental milestones – – Confirmed single-arm design for pivotal study of SMA Type 1 in U.S. – – Conference call of November 1, 2016 , was in lieu of Q3

11/08/2016 AveXis to Present at Jefferies 2016 London Healthcare Conference

CHICAGO , Nov. 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases , today announced that Sean Nolan , president and chief executive officer, will

11/01/2016 AveXis Announces Single-Arm Design for U.S. Pivotal Study of AVXS-101 in SMA Type 1 Patients

– Company provides update following receipt of FDA minutes from Type B meeting – – Conference call and webcast November 1 at 4:30 p.m. EDT – CHICAGO , Nov. 01, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering

10/31/2016 AveXis to Report Third Quarter 2016 Financial and Operating Results

Conference call and webcast on November 10 at 4:30 p.m. EST CHICAGO , Oct. 31, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce

10/10/2016 AveXis to Host Webcast Update of Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Data, Including Developmental Milestones, as of September 15, 2016 as presented by Jerry Mendell , M.D. at the International Annual Congress of the World Muscle Society -- -- Webcast Today, October 10, 2016 at 8:30 a.m. EDT – CHICAGO , Oct. 10, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

10/08/2016 AveXis Reports Interim Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1 as Presented at the International Annual Congress of the World Muscle Society

-- Majority of patients on the proposed therapeutic dose achieved key developmental milestones including sitting unassisted; two patients walking independently -- -- Conference call and webcast October 10 at 8:30 a.m. EDT -- CHICAGO --(BUSINESS WIRE)--Oct. 8, 2016-- AveXis, Inc.

09/08/2016 AveXis Announces Pricing of Public Offering of Common Stock

CHICAGO , Sept. 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced the pricing of an underwritten public offering of 4,250,000 shares of its common stock, 3,960,145 shares of which are being offered by AveXis and 289,855 shares of which are being offered by PBM Capital

09/06/2016 AveXis Announces Proposed Public Offering of 4,000,000 Shares of Common Stock

CHICAGO , Sept. 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS) today announced that it intends to offer and sell, subject to market conditions, 3,566,474 shares of its common stock in an underwritten public offering. Additionally, PBM Capital Investments, LLC , an existing stockholder of

08/11/2016 AveXis Reports Second Quarter 2016 Financial and Operating Results and Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Interim data through July 1 showed no “events” -- -- Continued motor function improvement with 25% of patients in the proposed therapeutic dose group in the normal range -- -- FDA requested Type B Meeting to discuss SMA Type 1 clinical development pathway -- -- Conference call and webcast August

08/02/2016 AveXis to Report Second Quarter 2016 Financial and Operating Results and Interim Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

Conference call and webcast on August 11 at 4:30 p.m. EDT CHICAGO , Aug. 02, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce financial

07/20/2016 AveXis Receives U.S. FDA Breakthrough Therapy Designation for AVXS-101 Gene Replacement Therapy for Spinal Muscular Atrophy Type 1

CHICAGO , July 20, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. , (Nasdaq:AVXS) a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the U.S. Food and Drug Administration ( FDA ) has granted

07/11/2016 AveXis Appoints Michael B. Johannesen as Senior Vice President, General Counsel and Chief Compliance Officer

CHICAGO , July 11, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (Nasdaq:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the appointment of Michael B.

06/06/2016 AveXis to Present at Goldman Sachs Annual Global Healthcare Conference and Jefferies 2016 Healthcare Conference

CHICAGO , June 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced that members of the management team will present at two

05/16/2016 AveXis Presents Pulmonary Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Richard Shell, MD, Presented Data as of April 1, 2016 at the American Thoracic Society 2016 Conference -- CHICAGO , May 16, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening

05/12/2016 AveXis Reports First Quarter 2016 Financial and Operating Results

Encouraging interim data for lead clinical program, AVXS-101, for Spinal Muscular Atrophy Type 1 Conference call May 12 at 4:30 p.m. EDT CHICAGO , May 12, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from

05/06/2016 AveXis Reports Data from Ongoing Phase 1 Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Jerry Mendell , MD, Presented Data as of April 1, 2016 at the American Society of Gene & Cell Therapy 19 th Annual Meeting -- -- Company to Host Webcast Today at 4:30 p.m. Eastern Daylight Time -- CHICAGO , May 06, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

05/05/2016 AveXis to Report First Quarter 2016 Financial Results on Thursday, May 12

CHICAGO , May 05, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce financial results for the first quarter ended March 31, 2016 on

04/25/2016 AveXis to Report Data from Ongoing Phase 1 Clinical Trial of AVXS-101 in Spinal Muscular Atrophy Type 1

-- Jerry Mendell , M.D., to Present Data as of April 1, 2016 at the American Society of Gene & Cell Therapy 19 th Annual Meeting -- -- Company to Host Webcast on Friday, May 6, 2016 at 4:30 p.m. Eastern Daylight Time -- CHICAGO , April 25, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

03/16/2016 AveXis Reports Fourth Quarter and Full Year 2015 Financial and Operating Results

-- Initial public offering raised $98.2 million in net proceeds -- -- Encouraging interim data for lead clinical program, AVXS-101, for Spinal Muscular Atrophy Type 1 – -- Conference call March 16 at 4:30 p.m. EDT -- CHICAGO , March 16, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc.

03/08/2016 AveXis to Report Full Year and Fourth Quarter 2015 Financial Results on Wednesday, March 16

CHICAGO , March 08, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (NASDAQ:AVXS), a clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, will announce financial results for the fourth quarter and full year ended

03/07/2016 AveXis Announces Appointments of Daniel G. Welch, Terrence C. Kearney and Frank Verwiel, M.D. to Board of Directors

CHICAGO , March 07, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (Nasdaq:AVXS), a leading clinical-stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced three appointments to its Board of Directors: Daniel G.

02/10/2016 AveXis Announces Pricing of Initial Public Offering

CHICAGO , Feb. 10, 2016 (GLOBE NEWSWIRE) -- AveXis, Inc. (Nasdaq:AVXS), a clinical stage gene therapy company developing treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced the pricing of its initial public offering of 4,750,000 shares of

01/04/2016 AveXis Announces Completed Enrollment of Phase 1 Clinical Trial for AVXS-101 in Spinal Muscular Atrophy Type 1

Chicago, Ill. – January 4, 2016 – AveXis, Inc., a clinical-stage gene therapy company developing novel treatments for patients suffering from rare and life-threatening neurological genetic diseases, today announced patient enrollment has completed for the Phase 1 clinical trial of AVXS-101 for the

10/05/2015 Data from Ongoing Study of AVXS-101 in Spinal Muscular Atrophy Type 1 Presented at World Muscle Congress

Brighton, United Kingdom. – October 5, 2015 – AveXis, Inc., a clinical stage gene therapy company developing treatments for rare and life-threatening neurological genetic diseases, presented data from its ongoing Phase 1/2 trial of AVXS-101, the company’s initial product candidate for the treatment

09/16/2015 AveXis Strengthens Executive Leadership Team with Key Appointments

– Thomas J. Dee, Senior Vice President, Chief Financial Officer – – James J. L’Italien, Ph.D., Senior Vice President, Chief Regulatory and Quality Officer – – Andrew Knudten, Senior Vice President, Manufacturing and Supply Chain – – Sukumar Nagendran, M.D., Senior Vice President and Chief Medical

09/08/2015 AveXis Secures $65 Million in Financing

Company builds investor syndicate comprising leading mutual and biotech funds to advance its spinal muscular atrophy gene therapy program Chicago, Ill. – September 8, 2015 – AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening neurological genetic

06/23/2015 AveXis Gains Orphan Drug Designation from the European Medicines Agency for Gene Therapy Treatment in Spinal Muscular Atrophy

Dallas – June 23, 2015 – AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening genetic diseases, today announced that the European Medicines Agency (EMA) has granted Orphan Drug designation to its gene transfer therapeutic currently in clinical development

06/18/2015 AveXis Appoints Sean P. Nolan as Chief Executive Officer

DALLAS, June 18, 2015 — AveXis, Inc., a leading gene therapy company developing treatments for rare and life-threatening genetic diseases, today announced the appointment of Sean P. Nolan as Chief Executive Officer. He will oversee all functions at AveXis, including clinical, regulatory, finance,

04/30/2015 AveXis Announces Management Succession Plan

April 30, 2015, Dallas, Texas: AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases, today announced that John Carbona has resigned from his position as CEO. Mr. Carbona will remain a Director of AveXis.

03/19/2015 AveXis EU Ltd. Granted SME Status by the European Medicines Agency

Dallas – March 19, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced that the European Medicines Agency (EMA) has granted AveXis’ wholly owned

02/25/2015 AveXis Announces the Formation of its European Subsidiary, AveXis EU, Ltd.

Dallas – February 25, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced the formation of its EU-based wholly-owned subsidiary, AveXis EU, Ltd.

01/06/2015 AveXis Closes its Series C Financing Round with Deerfield Management and Roche Venture Fund

Dallas – January 6, 2015 – AveXis, Inc., a biotechnology company focused on using gene therapy to transform the lives of patients with severe genetic and orphan diseases like Spinal Muscular Atrophy (SMA), today announced it has completed a $10 million financing round led by Deerfield Management