Fact or Fiction - Part 2

Should I start a Facts or Fiction series?

Anyway, recently, Vertex announced that the company's NDA for Kalydeco has been completed and the FDA has agreed to given it priority review. Vertex even said in its press release that the PDUFA date is in April of 2012.

In case you are not familiar, this is definitely different from having the NDA approved.

So take a look at this picture and see what's wrong with the article was written by

Valeant's Proposal to Ista

It's Friday and many times this year whenever I heard about Valeant, I start thinking about another possible acquisition. This stems from the many acquisition that the company has made this year.

This time the company has taken its proposal public after the board of directors repeatedly reject its offer and would like ISTA's shareholders to know about it.

Valeant announced that it has made a proposal to the board of directors of ISTA Pharmaceuticals to acquire the company's outstanding share for $6.5 per share in cash. This represents a premium of over 60$ of current value of ISTA's share which closed at $3.87

BioSannte's shares hit new low with the results of the phase III LibiGel efficacy trials

We all know that investors are not forgiving..
BioSante recently announced the results of the efficacy trials evaluating LibiGel (testosterone gels) for the treatment of hypoactive sexual desire disorder in postmenopausal women. The company's share has taken a tumble and dropped to $0.48 a share (from $2.1 a share).  This represents a decrease of 75% in value. So what goes wrong?

The company's two pivotal trials showed that the medication is not more effective than placebo given in postmenopausal women experienced hypoactive sexual desire.

The BLOOM-1 trial, which enrolled 597 patients, showed that patients taking Libigel reported an increase of 1.47 days with a satisfying sexual event when compared to baseline.  Patients taking placebo in the same trial had an increase of 1.26 days when compared to baseline.  The difference between the two groups was not statistically significant (and even if it is, it might not be clinically meaningful either).  Even though there isn't significant change in clinical endpoint, pharmacokinetic data showed an increase in free testosterone levels in patients taking LibiGel after six months when they were compared to baseline and placebo groups (4.01 vs 1.1 vs 1.19 respectively).

The BLOOM-2 trial, which enrolled 575 patients, showed that patients taking LibiGel reported an increase of 1.0 day with a satisfying sexual event when compared

Kalydeco of Vertex gets priority review from the US FDA

Vertex announced that the FDA has given the company's Kalydeco (ivacaftor) for the treatment of cystic fibrosis priority review. The target review date will be April 18, 2012. 

Vertex has also announced that the marketing authorization application for Kalydeco submitted to the European Medicines Agency has been validated.

The company has completed two phase III trials,

Dicerna received milestone payment from Kyowa

Kyowa Hakko Kirin  has selected the first DsiRNA therapeutic candidate, which was developed under the collaborative agreement with Dicerna, to advance into formal development studies. 

The announcement triggers a $5M milestone payment that Dicerna will receive from Kyowa. At the same time, Dicerna has received an undisclosed payment from Kyowa as it has decided to include a second oncology target into the collaboration.

The companies signed a research collaboration and license agreement in Jan 2010 to utilize Dicerna's prorietary Dicer Substrate Technology to develop therapies for oncology indications.  The agreement was expanded in December 2010 to include therapies for immunologic and inflammatory diseases.

Kyowa Hakko Kirin will be responsible for the continuing development of the DsiRNA therapeutic candidate.  Dicerna has retained rights to commercialize and to have profit-share opt-in for the therapy.

Under the original agreement, Dicerna received $4M in upfront cash payments including research funding. Kyowa will receive exclusive rights to one target for oncology indication and Dicerna would be eligible to receive up to $120M from Kyowa for research funding and development and commercial milestones. Dicerna has option to co-promote and profit-share with Kyowa for the initial target in the US.  The agreement was expanded in December 7, 2010 to include immunologic and inflammatory disease.

Isis hosts a conference call to discuss about the three newly added agents

Isis announced that it has added three new agents to its development pipeline and will be hosting a conference called on December 15 at 12:00 PM Eastern Time (for information about the conference call, see below).

• ISIS-APOArx works by reducing apolipoproteina( in the liver) and is being developed for the treatment of Atherosclerosis
• ISIS-DGAT2Rx works by inhibiting diacylglycerol acyltransferase-2, which is the main component in the synthesis of triglyceride, and is currently being developed or the treatment of NASH
• ISIS-FVIIRx works by reducing factor VI, which is an important factor in the coagulation pathway, and is currently being investigated for the treatment of clotting disorders

 Conference call through telephone

• Phone number: 866-825-3308
• Passcode: Isis2011

Conference call through website

• www.isispharma.com

Health Canada approved Eisai's Halaven

Health Canada has approved Eisai's Halaven (eribulin mesylate) for the treatment of metastatic breast cancer in patients who have previously received at least two chemotherapeutic agents for the approved indication. Eisai has recently announced its official entrance into Canadian market.  The company currently has two products marketed in Canada: Benzel (an antiepiletic agent) and Gliadel Wafer (for malignant brain tumors).

Eisai Canada plans to launch the product before the end of its 2012 fiscal year on March 31, 2012.  The company plans to submit New Drug Submissions for perampanel and other products starting next years.

Halaven has been approved in 35 countries including Canada, the US, EU member countries, Japan, Switzerland and Singapore.

Halaven (eribulin mesylate) is a non-taxane microtubule inhibitor. The compound belongs to a class of compound called halichondrins which are isolated from the marine sponge Halichondria okadai.

Interesting fact about Halaven: the molecule has a molecular weight of 826 and 19 chiral carbons.  The synthesis of the molecule requires approximately 62 steps.

Seattle Genetics and Millennium announced interim results of the phase I trial evaluating Adcetris

Seattle Genetics and Millennium (Takeda) announced the interim results of the phase I trial evaluating ADCETRIS (brentixumab) given in combination with chemotherapy for the treatment of newly diagnosed advanced stage  Hodgkin lymphoma patients.

Adcetris was given in combination with one of the following chemotherapy regimens:

• ABVD: adriamycin, bleomycin, vinblastine, and dacarbazine
• AVD: adriamycin, vinblastine and dacarbazine

Twenty-five patients were enrolled in the Adcetris and ABVD group

Salix submitted the NDA for crofelemer

Salix announced that it has submitted the new drug application for crofelemer for the treatment of HIV-associated diarrhea. 

Crofelemer was developed by Napo Pharmaceuticals, with whom Salix signed a collaboration agreement to develop crofelemer.  Under the terms of the agreement,  Salix has exclusive license to market crofelemer for the HIV-associated diarrhea, pediatric diarrhea, and acute infectious

Alexza's Adasuve received positive opinions from the FDA Drug Advisory Committee

Alexza announced that the FDA Psychopharmacologic Drugs Advisory Committee has recommended approval of its Adasuve (Staccato loxapine) for the treatment of agitation in patients with schizophrenia or bipolar mania.  Once approved, the medication will need to be used with the FDA recommended Risk Evaluation and Mitigation Strategy (REMS).

The PDUFA date for Adasuve has been set on Feb 4, 2012. 


The FDA Psychopharmacologic Drugs Advisory Committee

Vanda partnered with Megapharm in Israel

Vanda Pharmaceuticals has entered into an exclusive license agreement with Megapharm. Under the terms of the agreement, Fanapt will be commercialized by Megapharm who will also be seeking regulatory approval for the product. The regulatory approval application has been filed in Israel in 2011 for the treatment of schizophrenia.

Iloperidone (Fanapt) is a psychotropic agent that belongs to the class of chemical called piperidinyl-benzisoxazole derivatives.  The exact mechanism of this compound has not been fully understood.  One of the proposed mechanism that resulted in clinical efficacy of the medication was its antagonism on both dopamine type 2 and serotonin type 2.

Fanapt is currently available in the US in the following strengths: 1 mg, 2 mg, 4 mg, 6 mg, 8 mg, 10 mg, and 12 mg tablets.

MegaPharm was founded in 1989 and has since become partners with many international companies.  It's a private biotech marketing company in Israel with many products available in different therapeutic areas.

Icon acquired BeijingWits Medical Consulting Ltd

Icon announced that it has acquired BeijingWits Medical Consulting Ltd, a contract research organization in China. The move will increase Icon's presence in China where the demand for clinical research is on the rise.

BeijingWits was founded in 1997 and has over 100 highly qualified and experienced professionals stationed in Beijing, Shanghai, Chengdu, Guangzhou, Wuhan and Hong Kong. 

Financial terms were not disclosed and the transaction is expected to be completed in the first quarter of 2012.

Icon is a global contract research organization.  The company stock is currently traded as ICLR in the NASDAQ.

Regulus Therapeutics initiated the discovery of new anti-miRs for the treatment of glioblastoma

Regulus Therapeutics announced that it has initiated the process to discover chemically modified oligonucleotide anti-miRs using its expertise in microRNA therapeutics for the treatment of glioblastoma multiforme.

The company has been award a research grant from the Accelerate Brain Cancer Cure to accelerate the process. The compound will be tested at the Samsung Medical Center at which the compound will be trialed in preclinical models that have been shown to mimic human brain cancer.

Anti-miRs are antisense oligonucleotide inhibitors of microRNAs which have been shown to play roles in different biological processes.  By inhibiting microRNAs, the company hopes its therapy will be able to inhibit disease progression of certain disease or to treat certain conditions.

Abbott expanded its agreement with Reata

Abbott has entered into a worldwide collaboration with Reata Pharmaceuticals to develop and commercialize Reata's second-generation oral antioxidant inflammation modulators.  The newly signed agreement is the expansion of the previous collaboration in which Abbott has obtained exclusive rights to develop and commercialize Reata's bardoxolone methyl outside the US (excluding several Asian markets).  The companies plan to move its first compound into clinical trial in 2012.  Reata continues to have US development and commercialization rights to bardoxolone methyl which is currently being studied in the phase III trial (BEACON study) in patients with stage 4 chronic kidney disease and type 2 diabetes.

Under the new agreement, Reata will receive $400M one-time license payment.  Both companies will equally share the costs and profits spent and generated from all new AIMs for the following therapeutic areas pulmonary, central nervous system disorders and certain immunological disorders.  Abbott will also be responsible for 70% of the development cost and take 70% of profits in compounds being developed for rheumatoid arthritis and other autoimmune diseases while Reata will be responsible for the other 30%.

Both companies have also agreed to discover new molecules that demonstrate similar pharmacology seen in AIMs that are already present in Reata's portfolio.

Antioxidant inflammation modulators are potent activators of transcription factor Nrf2 which have been shown to increase the production of antioxidant, detoxification and anti-inflammatory genes.  The activation of Nfr2 also inhibits NF-kB which is a pro-inflammatory enzyme regulator.  As a result, by activating Nrf2, the companies hope these compounds will be able to decrease inflammation that is often associated with chronic diseases including multiple sclerosis, rheumatoid arthritis, chronic kidney disease, neurodegenerative disease and COPD. 

Endo announced the approval of Opana(R) ER crush-resistant tablet

Endo Pharmaceuticals announced that the FDA has approved the company's Opana (R) ER, which has been designed to be crush-resistant, for the management of pain.

Oxymorphone, Opana(R) ER active ingredient, is classified as a CII controlled substance with high abuse potential.

Opana(R) ER is designed using the proprietary INTAC technology owned by the company's partner, Grunenthal.

The medicine will be marketed with the same dose strengths, color and packaging as well as similar tablet size and shape to the currently market Opana(R) ER. The current Opana(R) ER tablets are currently available in 5 mg, 7.5 mg, 10 mg, 15 mg, 20 mg, 30 mg, and 40 mg tablets.

The company also said that US Patent and Trademark Office will issue patent covering the new formulation of Opana (R) ER on December 13, 2011. The patent number will be called 8,075,872 and the patent will provide protection to the product until November 2023.

The company currently markets Opana in different formulation: regular tablet, extended release tablet and injectable.
 

Janssen Biotech entered into a collaboration with Pharmacyclics

Janssen Biotech (part of Johnson and Johnson) has signed an agreement with Pharmacyclics to allow both companies working together to develop and market PCI-32765, currently being investigated for multiple B-cell hematologic malignancies. Under the agreement, the companies will equally share profit and loss as well as cost of development and commercialization related to PCI-32765.  Pharmacyclics has received an upfront payment of $150M and is eligible to receive additional payment as PCI-32765 achieves certain development and regulatory milestones.

PCI-32765 is currently being studied in phase 1 and 2 trials for the treatment of B-cell malignancy disorders, including chronic lymphocytic leukemia, mantel cell lymphoma and diffuse large B-cell lymphoma.  

PCI-32765 of Pharmacyclic is an orally active, small molecule that inhibits Bruton’s tyrosine kinase which is an essential element of the B-cell antigen receptor signaling pathway that has been shown to play a significant role in tumor expansion and proliferation.  As an inhibitor of an important element of the pathway, PCI-32765 has been shown to induce cell death.