Last week, major events in the biotech sector included an
acquisition announcement, a positive FDA panel review for an
insomnia candidate and a delay in the filing of a new drug
application (NDA) for a Duchenne muscular dystrophy (DMD)
Vanda the Clear Winner: Vanda
) was the clear winner last week with shares shooting up 115% on
positive feedback from the FDA regarding insomnia candidate,
tasimelteon (proposed trade name: Hetlioz). The FDA's advisory
panel voted resoundingly in favor of approving the candidate for
the treatment of Non-24-Hour Disorder in the totally blind.
While the FDA is not required to follow the advice of its
advisory panel, it usually does so. So no surprises if
tasimelteon is approved by Jan 31, 2014 (FDA action date).
Sarepta Plunges on Eteplirsen Update: Sarepta's
) hopes of filing an NDA for its DMD candidate, eteplirsen, based
on current data were quashed with the FDA saying that the NDA
filing would be considered premature. The company lost about 64%
of its market value. Sarepta has a long way to go - first up, the
company will be meeting with the FDA later this month to discuss
the confirmatory study design.
With the FDA asking for a placebo-controlled study, patient
enrolment will be a challenge. As things stand now, Sarepta is
looking at a delay of at least two years in the potential
approval of eteplirsen.
Shire Zeroes In on ViroPharma:
) being an acquisition target surfaced earlier this year in June.
At that time, the shares were trading in the $27 - $28 range. But
with acquisition rumors gaining traction, shares shot up to the
late $30s range with a deal finally being struck with Shire at
$50 per share (representing a premium of 27%). Apart from Shire,
companies like Sanofi were also rumored to be interested in
ViroPharma which is well known for its focus on rare
Impact of New ACC/AHA Guidelines:
The new guidelines issued by The American College of Cardiology
(ACC) and the American Heart Association (AHA) released last week
created a lot of buzz with the main discussion being the impact
of the guidelines on PSCK9 inhibitors. The guidelines, which will
benefit statins and lead to an increase in their use, have left a
question mark on the regulatory process for PSCK9 inhibitors, a
new class of cholesterol management drugs currently in
The main concern is that the FDA will require cardiovascular
outcome trials to be conducted before granting approval to PSCK9
inhibitors - this would delay the approval process
However, according to a
article, U.S. regulators will not require companies developing
PSCK9 inhibitors to conduct cardiovascular outcome trials to gain
approval. The drugs need to meet the FDA's current standards for
approval which include cholesterol/blood pressure reduction etc.
But, results from a study (IMPROVE-IT) being conducted by Merck
could very well change this equation. Some biotech companies with
a stake in PSCK9 inhibitors are Amgen, Regeneron and
A Look at the Major Players
The table below captures the price movement of the major
biotech players last week and during the past 6 months. There
wasn't much of an update from these companies last week. The only
news of significance was an update on
) hemophilia A candidate, Eloctate. Eloctate could be facing a
bit of a delay as the FDA has asked the company for additional
information on certain steps in the validation process in the
manufacturing plan. So, Eloctate will now most likely be launched
in mid-2014 instead of sometime in the second quarter of 2014 -
not much of a delay to lose sleep over.
||Last 6 Months
AMEX Biotech Index
CADX Shoots Up on Favorable Ruling:
) investors heaved a sigh of relief with the company receiving a
favorable ruling in its patent infringement lawsuit against Exela
Pharma Sciences for Ofirmev. Shares shot up 34%. The favorable
ruling removes a significant overhang and Cadence should rest
assured that generic competition will not be entering the market
in the near future. The company can now focus on driving Ofirmev
ImmunoGen Gains on ADC Deal:
ImmunoGen continues to sign deals with major pharma companies for
its antibody-drug conjugate (ADC) technology. The latest deal is
with Novartis - ImmunoGen's third with this company - for the
exclusive right to use the ADC technology to develop anticancer
therapeutics to an undisclosed target. Shares rallied 10.3%. This
agreement marks ImmunoGen's fifth this year with a major
FDA Approves Rare Blood Cancer Drug:
) much-awaited blood cancer drug, Imbruvica gained FDA approval.
Imbruvica, developed in collaboration with Johnson and Johnson,
is approved for use as a single agent in patients with mantle
cell lymphoma (MCL) who have received at least one prior therapy.
The drug works by blocking signals that make the cancer grow and
spread. Shares were up 3.5% on the news. Imbruvica is also under
review for chronic lymphocytic leukemia (CLL) with a response due
by Feb 28, 2014.
The Week So Far:
Amgen presented new data on its cholesterol management
candidate, evolocumab (AMG 145) at the American Heart Association
(AHA) Scientific Sessions 2013. Evolocumab works by inhibiting
PCSK9, a protein that reduces the liver's ability to remove "bad"
cholesterol from the blood. The company also presented
encouraging interim overall survival data on its phase III cancer
candidate - talimogene laherparepvec.
Gilead gained EU approval for its HIV drug Vitekta - the FDA
had refused to approve it in April this year.
Regeneron presented data from several studies on Eylea at the
annual meeting of the American Academy of Ophthalmology
BioMarin's Vimzim was backed by the FDA's advisory panel.
Vimzim is under regulatory review for the treatment of patients
with the lysosomal storage disorder Morquio A syndrome.
With quite a few companies attending scientific conferences
like the AHA Scientific Sessions, more data should be out through
the remainder of the week.
Want the latest recommendations from Zacks Investment
Research? Today, you can download
7 Best Stocks for the Next 30 Days
Click to get this free report >>
ALNYLAM PHARMA (ALNY): Free Stock Analysis
BIOGEN IDEC INC (BIIB): Free Stock Analysis
CADENCE PHARMA (CADX): Free Stock Analysis
PHARMACYCLICS (PCYC): Free Stock Analysis
SAREPTA THERAP (SRPT): Free Stock Analysis
VANDA PHARMACT (VNDA): Free Stock Analysis
VIROPHARMA (VPHM): Free Stock Analysis Report
To read this article on Zacks.com click here.