The biotech sector was back in focus this week
with quite a few regulatory updates. While Celgeneâs CELG multiple
sclerosis (MS) drug was submitted for approval, Vertex expanded its
collaboration with CRISPR. Meanwhile, shares of both Insys INSY and
CymaBay Therapeutics CBAY plunged on disappointing news.
Recap of the Weekâs
Celgene's MS Drug Accepted for Review
in US/EU: Celgene announced that the FDA has accepted
for review the new drug application (NDA) for ozanimod for the
treatment of people with relapsing forms of multiple sclerosis
(RMS) in the United States. The European Medicines Agency (EMA)
also accepted for review the Marketing Authorization Application
for ozanimod for the treatment of adults with relapsing-remitting
multiple sclerosis in the European Union.
The FDA has set an action date of Mar 25, 2020. A
regulatory decision from the EMA is expected in the first half of
2020. The FDA and EMA applications are based primarily on ozanimod
data from the SUNBEAM and RADIANCE Part B phase III studies. In
February 2018, the company received a Refusal to File letter from
the FDA regarding its NDA for ozanimod for the treatment of people
with RMS. Upon its preliminary review, the FDA determined that the
nonclinical and clinical pharmacology sections in the NDA were
insufficient to permit a complete review. The company then
submitted a NDA again in March 2019 to the FDA based on data from
the SUNBEAM and RADIANCE studies.
Celgene currently carries a Zacks Rank #2 (Buy).
You can see the complete list of today's Zacks #1 Rank
(Strong Buy) stocks here.
Insys Crashes on Bankruptcy
News: Insys Therapeutics plummeted 51.4% after it
filed for bankruptcy protection under Chapter 11 of the U.S.
Bankruptcy Code in the U.S. Bankruptcy Court in the District of
Delaware. The protection will enable the company to facilitate the
sale of substantially all its assets and address its legacy legal
liabilities. Insys has been embroiled in various litigations in
recent times amid allegations of fueling the ongoing opioid endemic
in the United States. The company has also been accused of bribing
doctors to prescribe its lead drug, Subsys, to patients. This
month, Insys signed a settlement with the United States,
formalizing its previously announced settlement relating to certain
civil statutory claims. As part of this agreement, the company
agreed to pay $195 million.
Nightstar: Biogen BIIB announced that it has acquired
clinical-stage gene therapy company Nightstar Therapeutics for $800
million. The acquisition will add NSTâs lead asset, NSR-REP1, to
the companyâs pipeline. The candidate is being evaluated for the
treatment of choroideremia (CHM), a rare, degenerative, X-linked
inherited retinal disorder, which leads to blindness and has no
approved treatments. NSR-RPGR is NSTâs second clinical program for
the treatment of X-linked retinitis pigmentosa (XLRP), which is
also a rare inherited retinal disease, primarily affecting males
with no approved treatments. Biogen now has added two mid- to
late-stage clinical assets, as well as preclinical programs, in
Vertex Expands Collaboration With
CRISPR: Vertex Pharmaceuticals VRTX announced that it
is expanding its collaboration with CRISPR Therapeutics CRSP for an
exclusive licensing agreement to discover and develop gene editing
therapies targeting Duchenne muscular dystrophy (âDMDâ) and
Myotonic dystrophy type 1 (DM1).
Per the expanded collaboration terms, Vertex will
pay CRISPR $1 billion, including $175 million in upfront payment
and potential research, development, regulatory, and commercial
milestone payments for the DMD and DM1 programs. Vertex will get
exclusive worldwide rights to CRISPR Therapeuticsâ existing and
future intellectual property including foundational CRISPR/Cas9
Moreover, Vertex will be responsible for all
research, development, manufacturing, and commercialization
activities and related costs. However, research costs for a
specified guide RNA research related to DM1 program will be shared
by both the companies. Vertex and CRISPR had signed a four-year
research collaboration back to October 2015. Per this agreement,
Vertex and CRISPR are developing gene editing therapies for cystic
fibrosis and sickle cell disease. In December last year, the
companies selected gene therapy, CTX001, to move into clinical
development for sickle cell disease and beta thalassemia.
Concurrently, Vertex announced it will acquire
privately-held Exonics Therapeutics, which is focused on developing
gene editing therapies for DMD and other severe genetic
neuromuscular diseases. With the acquisition of Exonics, Vertex
will gain intellectual property, technology, and scientific
expertise in transformative gene editing therapies. Vertex will pay
$245 million upfront to Exonics for acquiring all its outstanding
shares and form a wholly-owned subsidiary. Shareholders of Exonics
are eligible to receive approximately $1 billion including upfront
and potential milestone payments related to the DMD and DM1
Both the transactions are expected to close in
the third quarter of 2019.
CymaBay Crashes on Disappointing NASH
Data: Shares of CymaBay plunged 45.5% after the
company reported disappointing data from a mid-stage study on lead
candidate, seladelpar for the treatment of nonalcoholic
steatohepatitis (NASH). 12-week top-line results from the ongoing
52-week phase IIb dose-ranging, paired liver biopsy study of
seladelpar showed that treatment with the candidate resulted in
minimal reductions in liver fat that were not significant when
compared to placebo. While the reductions in markers of liver
injury were robust and clinically meaningful, investors were
clearly disappointed with the dismal results, given the potential
of the NASH space.
Medical - Biomedical and Genetics
Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR %
The NASDAQ Biotechnology index lost 0.53% in the
last four trading sessions. Among the major biotech giants,
Alexion lost 0.34% in the period. Over the past six months, shares
of Celgene have surged 38.1% whereas Biogen stock has lost 28.3%.
(See the last biotech stock roundup here: Biotech Stock Roundup:
AMGN's Nuevolution Buyout, CELG, INCY Drugs' Label Expansion)
What's Next in Biotech?
Stay tuned for more pipeline and regulatory
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Insys Therapeutics, Inc.
(INSY) : Free Stock Analysis Report
Celgene Corporation (CELG)
: Free Stock Analysis Report
Biogen Inc. (BIIB) : Free
Stock Analysis Report
Incorporated (VRTX) : Free Stock Analysis Report
CymaBay Therapeutics Inc.
(CBAY) : Free Stock Analysis Report
CRISPR Therapeutics AG
(CRSP) : Free Stock Analysis Report
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