Amarin is waiting till next year for an FDA decision on Vascepa, but investors are fine

Amarin ($AMRN) will have to wait until the new year to find out whether its fish oil pill Vascepa will be approved for a wider indication. The FDA indefinitely delayed that decision, and it's reviewing the trial design of the New Jersey-based company's ANCHOR design in the meantime. The holdup apparently didn't put off investors, with shares spiking as much as 20% Friday morning

Amarin's ($AMRN) plodding effort to get Vascepa approved for a wider indication just got longer as the FDA delayed its final decision on the fish oil pill, but the company's backers are staying optimistic, and shares jumped as much as 20% on Friday morning.

The company is looking to expand the label of its cholesterol-fighting omega-3 drug, and the FDA was due to make a final ruling Friday. The delay, however, makes procedural sense: In October, the agency backed out of its support for the design of Amarin's ANCHOR study, eliciting an appeal from the company. Regulators would be loath to make a final decision on a drug without first weighing in on its supporting data, and the agency told Amarin it will rule on the study design appeal by Jan. 14, the company said.

Just when the FDA will make a decision on Amarin's supplemental drug application remains unknown, and the company notes in its announcement that "there can be no assurance that Amarin will be successful in this effort."

Investors were much cheerier, however, sending Amarin's now-pilloried shares up in premarket trading. Still, the company has plummeted by about 75% since October, when an FDA panel cast serious doubt on its odds of winning approval to sell Vascepa to patients with less-severe high triglycerides.

The ensuing consternation led Amarin to slash its payroll and may have spelled the demise of CEO Joe Zakrzewski, who will walk out the door Jan. 1 in what the company is calling a "retirement."

Actelion gets second boost for Opsumit with EU approval

Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission

-1 of 3- 20 Dec 2013 16:35:00 UTC  *DJ Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission

   December 20, 2013 11:35 ET (16:35 GMT)- - 11 35 AM EST 12-20-13
-2 of 3- 20 Dec 2013 16:35:00 UTC  Press Release: Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission
Actelion Pharmaceuticals Ltd / Actelion is granted Marketing Authorisation for Opsumit (macitentan) in Pulmonary Arterial Hypertension (PAH) by the European Commission . Processed and transmitted by Nasdaq OMX Corporate Solutions. The issuer is solely responsible for the content of this announcement.
-- Approval granted by European Commission on 20 December 2013
-- Opsumit approved as monotherapy or in combination with another PAH therapy for the long-term treatment of WHO Functional Class II to III PAH patients
-- First EU launch planned in Germany in February 2014
ALLSCHWIL, SWITZERLAND - 20 December 2013 - Actelion Ltd (SIX: ATLN) today announced that Opsumit(R) (macitentan), a novel dual endothelin receptor antagonist (ERA), has been granted marketing authorisation for the long-term treatment of PAH in the EU by the European Commission. Opsumit, as monotherapy or in combination, is indicated for the longterm treatment of pulmonary arterial hypertension (PAH) in adult patients of WHO Functional Class (FC) II to III.
Efficacy has been shown in a PAH population including idiopathic and heritable PAH, PAH associated with connective tissue disorders, and PAH associated with corrected simple congenital heart disease.
Macitentan 10mg is also described as reducing the risk of PAH related death or hospitalization for PAH up to end of treatment (EOT) by 50% (HR 0.50; 97.5% CI: 0.34 to 0.75; logrank p < 0.0001). Macitentan 10mg improved quality of life assessed by the SF-36 questionnaire.
Dr Nazzareno Galiè from the Institute of Cardiology, University of Bologna, Bologna, Italy spoke of the impact of the availability of Opsumit, "We are all very pleased with the approval of Opsumit in Europe. For the first time we can offer patients a therapy that has demonstrated improvement in long term clinical outcome showing a significant effect in naive patients and patients who are already on a specific PAH treatment."
The EU label was based in part on data from the landmark Phase III SERAPHIN study which was published in the New England Journal of Medicine in August 2013, the SERAPHIN study demonstrated that treatment with macitentan 10 mg resulted in a 45% risk reduction (hazard ratio [HR] 0.55; 97.5% CI: 0.39 to 0.76; p < 0.0001) of the composite morbidity-mortality endpoint up to (EOT) when compared to placebo.[1]
Jean-Paul Clozel, M.D. and Chief Executive Officer of Actelion commented: "We are delighted with today's announcement as we believe that Opsumit represents a major step forward for the management of PAH. Our company strategy of sustaining and growing our PAH franchise has yet again been bolstered by this approval. This achievement marks a significant moment for the PAH community in Europe as the first and only PAH treatment with proven long-term efficacy, from controlled clinical trials in PAH, and will now offer hope of a better future to those living with this disease. Actelion is now working to make this important medicine available to patients around European markets in the coming months."
The most commonly reported adverse drug reactions are nasopharyngitis (14.0%), headache (13.6%) and anaemia (13.2%). The majority of adverse reactions are mild to moderate in intensity.
Opsumit was approved by the FDA on 18 October 2013 and by Health Canada in November 2013. It is also undergoing regulatory assessment in other countries including Switzerland.

Genocea brings 2nd vaccine to clinic to rival $4B-a-year Prevnar

Genocea Biosciences, which three months ago announced what it called “unprecedented” results in a trial of its herpes vaccine, today began an early-stage trial of a second potential vaccine based on the same disease-fighting method.

The Cambridge biotech has been focused on fighting infectious diseases through stimulation of T cells in the body since it was founded in 2007. CEO Chip Clark says the approach is superior to existing vaccines (which are based on the so-called B-cell response) in two ways: It can potentially fight all strains of a disease, rather than a select few, and can kill infected cells even before they make their way into the bloodstream and potentially start to make a patient sick.

That approach has resulted in a potential vaccine for herpes simplex virus type 2, and the results from its first-ever human trial were announced in September. The study found that the vaccine reduced viral-shedding, which often results in the disease emerging in lesions.

It has also been behind $73 million in investment to date from backers including Polaris Venture Partners, Lux Capital Management, Johnson & Johnson Development Corp., Skyline Ventures, Cycad Group, Auriga Partners, the Bill & Melinda Gates Foundation, MP Healthcare Ventures, and Morningside.

Today, it’s led to the trial of a potential vaccine against pneumococcus, a disease of the nose and throat which can lead to pneumonia, meningitis and sepsis, and kills between 500,000 and 1 million children every year. The company has begin a a Phase 1 clinical study with GEN-004, which Clark intends to be the first vaccine that would protect against all 90 strains of the disease.

Clark said that the current market-leading vaccine on the market is Prevnar, a $4 billion-a-year drug marketed by Pfizer. (One of the members of Genocea's board, George Sibor, is the former chief scientific officer of Wyeth Vaccines where he lead the development of Prevnar) Clark says the vaccine is “phenomenal” in its effectiveness against 13 of the most important strains of the disease, but says “there is evidence that the 80-plus strains that are not covered by the vaccine are increasing.”

The Phase 1 study will involve about 90 healthy adult volunteers, and will test the safety and effectiveness of GEN-004 in a range of doses. The company expects results in the second quarter of 2014.

Genocea begins a PhI trial of its rival to Pfizer's Prevnar

The huge sales generated by Prevnar 13 mean Pfizer ($PFE) appears to have the pneumococcal vaccine sector sewn up, but Genocea Biosciences sees an opportunity to disrupt the monopoly. This week, the Cambridge, MA-based biotech began a Phase I clinical trial of the vaccine it thinks can unseat Prevnar.

Genocea developed the vaccine, currently called GEN-004, using the antigen discovery technology that spawned its lead candidate, herpes simplex vaccine GEN-003. It is this approach that gives Genocea the chutzpah to take on Pfizer's blockbuster. While the latest version of Prevnar protects against 13 strains of pneumococcus, Genocea is aiming to confer immunity against all forms of the bacteria. Prevnar 7 and 13 have had a huge effect on disease, but the 80 strains not covered by the vaccines are still causing some cases.

A paper published in PLOS ONE in September found that the introduction of Prevnar 7 coincided with a rise in cases of invasive pneumococcal disease caused by the 80 strains not covered by the vaccine. Pfizer subsequently expanded protection by introducing Prevnar 13, but Genocea thinks there is still a need for a more comprehensive vaccine that will stop the impact of strain replacement. "In a way, Prevnar is a victim of its own success," Genocea CEO Chip Clark told FierceVaccines.

Genocea, a 2008 FierceBiotech Fierce 15 company, has heavy hitters in its corner. George Siber, who was chief scientific officer at Wyeth when Prevnar 7 was in development, sits on Genocea's board of directors. Siber sees potential in using a T cell-directed approach to create a universal pneumococcal vaccine that will combat the bacteria in the nasopharynx, the part of the body where it may evolve. The theory is now being put to the test in a Phase I trial of 90 healthy volunteers. Data is due to be presented in the second quarter of 2014.

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Genocea Biosciences is throwing its hat in the growing IPO ring. The biotech has laid out plans to raise $75 million to help fund its work on a pair of early-stage T-cell vaccines aimed at herpes and all strains of the bacteria pneumococcus.

Genocea has built a development platform that hunts up ideal antigens for spurring a T-cell attack on a pathogen. Its work has attracted the high-profile support of the Bill & Melinda Gates Foundation and may have applications in cancer immunotherapy as well.

The Cambridge, MA-based Genocea launched its Phase I study of its new pneumococcus vaccine just days ago, explaining to FierceVaccines that the biotech thinks it can do better than Pfizer's ($PFE) blockbuster Prevnar 13, which guards against 13 strains of the bacteria. Genocea says it has a more comprehensive approach.

Last fall, Genocea--a 2008 Fierce 15 company--landed a $30 million round, with the Gates Foundation joining CVF, Polaris Venture Partners, Lux Capital, SR One, Johnson & Johnson Development Corp., Skyline Ventures, Cycad Group, Auriga Partners, MP Healthcare Ventures and Morningside in bringing its total raise to $76 million.

This year has seen the biggest burst of biotech IPOs in a decade, a welcome turnaround from the drought years that led up to 2013. But in recent months, the market has seen more stumbles as a growing number of developers try to make the leap. Genocea will be among the first to test the waters in 2014.

Ariad storms back with an FDA nod to get Iclusig back on the U.S. market

Losing more than $2.5 billion in market value didn't kill Ariad Pharmaceuticals ($ARIA), and now the biotech is set to relaunch the once-spurned cancer drug Iclusig in the U.S. as the FDA has approved a new label and indication for the company's only product.

Ariad surged more than 30% on the news, announcing it would reintroduce the leukemia treatment by mid-January now that the FDA has OK'd the drug for T315I-positive patients and amended the label to include warnings about Iclusig's risk of blood clotting and heart failure. Those same safety concerns prompted the FDA to ask Ariad to take its drug off the market in October, the heaviest blow in a downward spiral that blasted the company's shares by more than 70%. But that's all in the past, CEO Harvey Berger said.

"We are back on track," Berger told Bloomberg. "We'll enter 2014 largely where we were as we started off the fall in September."

"Largely" being a relative term, of course. Ariad's hellish October led it to lay off about 160 U.S. employees in an effort to save $26 million, and while the company plans to piece together another U.S. sales force, it won't have the same marketing heft as before, Berger said. Furthermore, while the European Medicines Agency hasn't ordered Iclusig off the market, the group's Pharmacovigilance Risk Assessment Committee has reopened the books on the drug's safety profile, putting Ariad's overseas sales in jeopardy.

Still, Iclusig's exit from the tomb is undoubtedly good news for Ariad, as many analysts speculated it might take a year to win over the FDA if it ever happened at all. The company's shares leapt as high as $7.74 on the Friday announcement, by far their highest mark since an October freefall.

"In less than two months of suspending marketing and commercial distribution of Iclusig in the U.S., we addressed the issues raised by the FDA and now are able to market and distribute Iclusig again in the U.S.," Berger said in a statement.

Farmaci per il diabete hanno effetti diversi sul cuore degli uomini e delle donne

Secondo quanto suggerito da un nuovo studio pubblicato sul numero di dicembre dell’American Journal of Physiology - Heart and Circulatory Physiology, alcuni farmaci per il diabete ampiamente utilizzati hanno effetti diversi sul cuore degli uomini e delle donne. Lo studio ha considerato tre trattamenti comunemente prescritti per il diabete di tipo 2: metformina, metformina più rosiglitazone  e metformina più un tipo di olio di pesce.

I ricercatori hanno esaminato la maniera in cui tre trattamenti comunemente prescritti per il diabete di tipo 2 hanno influenzato 78 pazienti che sono stati divisi in tre gruppi. Un gruppo ha assunto metformina da sola, il secondo gruppo ha assunto metformina più rosiglitazone  e il terzo gruppo ha preso metformina più un tipo di olio di pesce.
La Metformina riduce la produzione di zucchero nel sangue dal fegato e migliora la sensibilità all'insulina. Rosiglitazone migliora la sensibilità all'insulina e rimuove gli acidi grassi liberi dal sangue. L’olio di pesce abbassa i livelli ematici dei trigliceridi.

I ricercatori hanno concluso che i farmaci hanno effetti molto diversi e talvolta opposti sui cuori degli uomini e delle donne, anche se i farmaci controllano lo zucchero nel sangue altrettanto bene in entrambi i sessi. Lo studio compare nel numero di dicembre dell’American Journal of Physiology - Heart and Circulatory Physiology.

Anche se la metformina ha avuto effetti positivi sul cuore nelle donne, ha indotto il metabolismo del cuore degli uomini a bruciare meno zucchero e più grassi. La combustione cronica del grasso da parte del cuore può risultare in cambiamenti nocivi che possono portare a insufficienza cardiaca, hanno detto i ricercatori della Scuola di Medicina dell'Università di Washington a St. Louis.
L’Assunzione di rosiglitazone o lovaza con metformina sembrava ridurre alcuni degli effetti negativi sul cuore della metformina assunta da sola negli uomini. L’assunzione di rosiglitazone in aggiunta a metformina ha ulteriormente migliorato il metabolismo del cuore delle donne, rispetto alla metformina da sola.

L'aggiunta dell’olio di pesce alla metformina non ha avuto un forte effetto in entrambi i casi per gli uomini o le donne, hanno aggiunto i ricercatori.
"Il nostro studio suggerisce che abbiamo bisogno di definire meglio quali terapie sono ottimali per le donne con diabete e quali sono ottimali per gli uomini ", ha detto il Dott. Robert Gropler, professore di radiologia. Lo studio non ha tuttavia dimostrato un legame di causa-effetto tra le combinazioni di farmaci e i cambiamenti cardiaci. Ha mostrato solo una associazione.