Major biotech stories last week included
) mid-stage data on its experimental anti-coagulant, a positive
) and a billion dollar plus deal for
). Other updates included the usual regulatory and pipeline
Isis's Pipeline Success Story Continues:
Isis' shares were up 7.54% on positive top-line data from a
mid-stage study on its experimental anti-coagulant, ISIS-FXIRx. Two
doses of ISIS-FXIRx (200mg, 300mg) were compared to Sanofi's
Lovenox. Patients in the 300mg group experienced a seven-fold lower
incidence in venous thrombolic events (VTE) compared to patients on
ISIS-FXIRx works by inhibiting the production of Factor XI -- a
coagulation factor that plays a key role in the formation of blood
clots. High levels of Factor XI increase the risk of thrombosis,
which could lead to heart attacks and strokes. Moreover, high
levels of Factor XI increase the risk of venous thrombosis, a
common problem after surgery, especially major orthopedic
procedures like knee or hip replacement.
The phase II data on ISIS-FXIRx looks good -- Isis could very
well strike a lucrative partnership deal based on these results.
The lower incidence of bleeding is important as bleeding is a major
concern associated with the use of anti-coagulants and could prove
to be fatal. So a lower bleeding rate could provide ISIS-FXIRx with
an edge over existing anti-coagulants. The multi-billion dollar
anti-coagulant market represents huge commercial potential.
CHMP Opinion Boosts PTC Therapeutics:
PTC Therapeutics soared 30.7% on a positive opinion issued by the
European Medicines Agency's (EMA) Committee for Medicinal Products
for Human Use (CHMP) regarding its experimental Duchenne muscular
dystrophy (DMD) treatment, Translarna. What makes this news
significant is that earlier this year, the CHMP had issued a
negative opinion for this very same treatment.
The company is seeking conditional marketing approval for the
treatment of nonsense mutation DMD (nmDMD). So with the CHMP now
adopting a positive stand, Translarna could well gain conditional
approval in the EU within three months. This is great news for PTC
as developing DMD drugs is not easy.
But this doesn't mean that PTC does not need to conduct phase
III studies. In fact, the company expects to finish enrolling
patients in an ongoing phase III study by mid-14 - positive results
from this study would help the company gain full approval in the EU
and the U.S.
Sarepta, another company with a DMD candidate, eteplirsen, in
its pipeline, saw its shares inching up (about 4.6%) on the
positive PTC development. Sarepta is looking to file for FDA
approval by year end.
Billion Dollar Plus Deal for Ophthotech:
Ophthotech signed a deal that could see it receiving more than $1
billion plus royalties from Novartis. Novartis gained exclusive
ex-U.S. commercialization rights to Ophthotech's lead pipeline
candidate, Fovista. Fovista is the most advanced anti-PDGF agent in
development for the treatment of wet age-related macular
The deal looks good for Ophthotech, which stands to gain a lot
financially. Plus, Novartis has lots of experience in the eye care
market given the strong presence of Alcon (a Novartis company).
Ophthotech also managed to retain rights in the U.S. Not
surprisingly, Ophthotech shares were up significantly (24.5%).
But there are still a couple of years to go before initial phase
III data on Fovista will be available.
Label Expansion for Amgen's (
Amgen's colorectal cancer drug, Vectibix, can now be used for the
first-line treatment of KRAS metastatic colorectal cancer in
combination with Folfax chemo. Late-stage data showing that the
addition of Vectibix to Folfax pushed up overall survival rates
significantly (4.4 months) should ensure robust sales for Vectibix
in this new setting. Vectibix delivered sales of $389 million last
Biogen's Plegridy Closer to EU Approval:
Biogen, well known for its strong presence in the multiple
sclerosis (MS) market, could see its experimental MS drug,
Plegridy, gain EU approval shortly. The CHMP has issued a positive
opinion. Plegridy is also under review in the U.S. where a response
should be out in the second half of the year.
Last 6 Months
*As of May 23, 2014
) Gains on Esbriet Data:
InterMune presented pivotal data on its lung disease drug, Esbriet
at the International Conference of the American Thoracic Society
(ATS) and the same was published online in the
New England Journal of Medicine
. Results looked favorable compared to Boehringer Ingelheim's
nintedanib. The market reacted positively to the data sending the
shares up 13.4%.
Esbriet, currently approved in the EU, is yet to gain U.S.
approval. While it is expected that both experimental treatments
will gain FDA approval, Esbriet's label may be more favorable.
Approval in the U.S. would boost sales of the drug
significantly. Esbriet sales were $70.3 million in 2013.
Endocyte Withdraws EU Filings:
Endocyte shares were down 6.01% on news that the company and its
partner, Merck, have withdrawn their marketing applications for
experimental ovarian cancer treatment, vintafolide plus a couple of
companion imaging components.
Ligand Ties Up with Viking:
Ligand is out-licensing a mix of five early- to mid-stage
candidates to another biotech company - Viking Therapeutics. The
out-licensed programs include type II diabetes, muscle wasting,
dyslipidemia and anemia.
The Week So Far:
Dyax Gains on Rival Data:
Dyax's shares jumped 27.4% on news that Biocryst's experimental
hereditary angioedema (HAE) drug, BCX416, fared well in a proof of
concept study. Investors reacted positively to this news as Dyax
too has an HAE candidate in its pipeline.
InterMune Resubmits Esbriet NDA:
InterMune resubmitted the NDA for its lung disease drug, Esbriet.
Esbriet is already approved in the EU.
Immuno-Oncology in Focus:
Immuno-oncology continues to attract more deals with
) tying up with Bristol-Myers Squibb.
) Plunges as FDA Says No Breakthrough Therapy Status....For
Provectus' shares plunged 62.9% on news that the FDA will not
assign Breakthrough Therapy status to the company's oncology
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