Jan 15, 2021 |
Novartis Gene Therapies Recommits to Global Managed Access Program for 2021.
Novartis Gene Therapies Recommits to Global Managed Access Program for 2021.
|
Sep 02, 2020 |
AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy.
AveXis renamed Novartis Gene Therapies, signifying the growing importance of gene therapy to Novartis corporate strategy.
|
Jun 01, 2020 |
AveXis Community Statement on the Global Managed Access Program for
AVXS-101
In January we launched a global Managed Access Program (MAP) for Zolgensma® (onasemnogene abeparvovec). Under this program, we are making up to 100 doses available in 2020 to eligible
patients with SMA under the age of two in countries where Zolgensma
is not approved. Already, the program has enabled children across Asia, Australia, Europe and North America to receive treatment who would not have otherwise had access to the therapy.
|
May 19, 2020 |
AveXis receives EC approval and activates “Day One” access program for Zolgensma®, the
only gene therapy for spinal muscular atrophy (SMA)
Basel, May 19, 2020 — AveXis, a Novartis company, today announced the European Commission (EC) granted conditional approval for Zolgensma® (onasemnogene
abeparvovec) for the treatment of patients with 5q spinal muscular
atrophy (SMA) with a bi-allelic mutation in the SMN1 gene and a clinical diagnosis of SMA Type 1; or for patients with 5q SMA with a bi-allelic mutation in the SMN1 gene and up to
three copies of the SMN2 gene.
|
Apr 21, 2020 |
AveXis Community Statement on the Coronavirus Disease (COVID-19)
Like many organizations across the United States and around the world, AveXis is closely monitoring the coronavirus disease (COVID-19) and its impact on patients, the SMA community,
healthcare providers, and our employees and partners. We know that
life with a rare genetic disease can bring significant challenges, and we want to be as supportive as possible, particularly during this unprecedented time. We are dedicated to the SMA community, and
our patients and their families are the motivation for everything we
do.
|
Mar 27, 2020 |
AveXis
receives positive CHMP opinion for Zolgensma®, the only gene
therapy for spinal muscular atrophy (SMA)
Basel, March 27, 2020 — AveXis, a Novartis
company, today announced that the Committee for Medicinal
Products for Human Use (CHMP) of the European Medicines
Agency (EMA) has adopted a positive opinion recommending
conditional marketing authorization of Zolgensma®
(onasemnogene abeparvovec) for the treatment of patients
with 5q spinal muscular atrophy (SMA) with a bi-allelic
mutation in the SMN1 gene and a clinical diagnosis of SMA
Type 1; or for patients with 5q SMA with a bi-allelic
mutation in the SMN1 gene and up to three copies of the SMN2
gene.
|
Mar 24, 2020 |
Zolgensma®
data shows rapid, significant, clinically meaningful benefit
in SMA including prolonged event-free survival, motor
milestone achievement and durability now up to 5 years
post-dosing
Basel, March 24, 2020 – AveXis, a Novartis
company, today announced a one-time infusion of Zolgensma®
(onasemnogene abeparvovec-xioi) showed rapid, significant
and clinically meaningful therapeutic benefit in patients
with spinal muscular atrophy (SMA) across a range of
studies, including in patients treated presymptomatically,
and sustained durability in patients now up to five years
post-dosing and some patients more than five years of
age.
|
Mar 24, 2020 |
AveXis
presents AVXS-101 IT data demonstrating remarkable increases
in HFMSE scores and a consistent clinically meaningful
response in older patients with SMA Type 2
Basel, March 24, 2020 – AveXis, a Novartis
company, today announced that new data from the Phase 1/2
STRONG study demonstrated a one-time intrathecal (IT)
administration of AVXS-101 in patients ≥2 years and <5 years
old with spinal muscular atrophy (SMA) Type 2 who received
Dose B (1.2 x 1014 vg) met the primary efficacy endpoint,
achieving a remarkable mean increase from baseline of 6.0
points in the Hammersmith Functional Motor Scale-Expanded
(HFMSE), twice the clinically meaningful threshold
established in previous SMA studies and reflecting
improvement in three to six skills.
|
Mar 24, 2020 |
Novartis
receives approval from Japanese Ministry of Health, Labour
and Welfare for Zolgensma the only gene therapy for patients
with spinal muscular atrophy (SMA)
Basel, March 19, 2020 — Novartis Pharma K.K.
(“Novartis Pharma”) today announced that the Japanese
Ministry of Health, Labour and Welfare (MHLW) approved
Zolgensma® (onasemnogene abeparvovec) for the treatment of
spinal muscular atrophy (SMA) in patients under the age of
two, including those who are pre-symptomatic at
diagnosis.
|
Dec 19, 2019 |
AveXis
Community Statement on Global Managed Access Program for
AVXS-101
December 19, 2019 Since its U.S.
approval, AveXis has had increasing demand from families
outside of the U.S. for access to AVXS-101.
|
Oct 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.
|
Oct 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
|
Sep 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
|
Sep 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®
|
Aug 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
|
Aug 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.
|
Jul 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.
|
May 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
|
May 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
|
May 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
|
May 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,
|
Apr 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
|
Apr 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
|
Dec 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
|
May 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.
|
May 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
|
Apr 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
|
Apr 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
|
Apr 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
|
Apr 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.
|
Mar 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)
|
Mar 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
|
Feb 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
|
Feb 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
|
Jan 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
|
Jan 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
|
Jan 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
|
Jan 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.
|
Jan 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.
|
Jan 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
|
Jan 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
|
Dec 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
|
Nov 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
|
Nov 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
|
Nov 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,
|
Oct 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
|
Sep 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 – –
|
Aug 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
|
Aug 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
|
Jun 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
|
Jun 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
|
Jun 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
|
Jun 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
|
Jun 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.
|
May 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
|
May 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
|
Apr 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 –
|
Apr 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
|
Mar 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.
|
Mar 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
|
Mar 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
|
Feb 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
|
Feb 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
|
Jan 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
|
Jan 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
|
Dec 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
|
Nov 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
|
Nov 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
|
Nov 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
|
Oct 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
|
Oct 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.
|
Oct 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.
|
Sep 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
|
Sep 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
|
Aug 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
|
Aug 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
|
Jul 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
|
Jul 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.
|
Jun 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
|
May 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
|
May 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
|
May 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.
|
May 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
|
Apr 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.
|
Mar 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.
|
Mar 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
|
Mar 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.
|
Feb 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
|
Jan 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
|
Oct 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
|
Sep 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
|
Sep 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
|
Jun 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
|
Jun 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,
|
Apr 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.
|
Mar 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
|
Feb 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.
|
Jan 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
|