Major biotech stories last week included Isis Pharma’s (ISIS) mid-stage data on its experimental anti-coagulant, a positive opinion for PTC Therapeutics (PTCT) and a billion dollar plus deal for Ophthotech (OPHT). Other updates included the usual regulatory and pipeline events.
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 Lovenox.
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.TO) 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 degeneration.
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 (AMGN) Vectibix: 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 year.
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.
|Company/Index||Last Week||Last 6 Months|
*As of May 23, 2014
InterMune (ITMN) 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 Incyte (INCY) tying up with Bristol-Myers Squibb.
Provectus (PVCT) Plunges as FDA Says No Breakthrough Therapy Status....For Now: Provectus’ shares plunged 62.9% on news that the FDA will not assign Breakthrough Therapy status to the company’s oncology candidate, PV-10.
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