Today, the FDA advisory panel will discuss BioMarin (BMRN) product drisapersen for Duchenne muscular dystrophy based on documents released to the committee by the FDA. The FDA said, “The benefit of drisapersen in exon 51 skip amenable patients is inconclusive at this time. Therefore, the benefit-risk assessments were not made.” Analysts interpreted the FDA statement as a condemnation of the drug, which does not really tell what the FDA meant …

Europe Approves Orkambi® The European Commission has granted Marketing Authorization for Orkambi® (lumacaftor/ivacaftor), for cystic fibrosis (CF) in people ages 12 and older who have two copies of the F508del mutation. There are approximately 12,000 people with CF ages 12 and older who have two copies of this mutation. Vertex will now begin the country-by-country reimbursement approval process across the European Union (EU). They said: The …

When we hear about a new approach that promises bringing in hopefulness in patients diagnosed with brain glioma, we try to listen and hope for the best, rather than develop and demonstrate  skepticism. Are we not walking the road of genetic engineering, gene therapy approaches and immunotherapy? Our optimism becomes realistic, especially when a scientific rationale is explained by researchers in scientifically solid firms that …

Food allergies are bad, significant, dangerous and growing in the U.S. and the rest of the world. More than 30 million people in the United States and Europe have a food allergy, and more than five million people in the United States and Europe have peanut allergy, including more than two million children. The prevalence of peanut allergy in children in the United States has increased …

Exelixis (EXEL) received the first of the great news we expected to hear. Today, the U.S. Food and Drug Administration (FDA) approved COTELLIC™ (cobimetinib) a cancer drug discovered by Exelixis in combination with Roche’s drug vemurafenib for BRAF V600E or V600K mutation-positive unresectable or metastatic melanoma. COTELLIC is a selective MEK inhibitor is subject of a worldwide collaboration agreement between Exelixis and Genentech (Roche). Switzerland had …

To shareholders who were stupefied with an instigated selloff of Incyte’s (INCY) stock today, we say do not get panicky. Nothing is wrong with Incyte, no bad news was announced with any of the firm’s 12 ongoing clinical trials or with its approved drug Jakafi, which demonstrated progressive growth since it was launched.  So, a clever correct move now would be taking advantage and accumulate …

– Shire to Acquire Dyax for $5.9million and some possible extras. – Angioedema market has treatments for attacks, but not prophylactic products for the prevention of HAE attacks. – A primary reason for the acquisition, but not the only one, is getting to Dyax’s prophylactic Hereditary Angioedema (HAE) product. – What will be the outcome of Biocryst’s lead HAE prophylactic drugavoralstat? -Will Biocryst be able …

Another Durable Botulinum Toxin with Many Advantages REVANCE THERAPEUTICS As per its self- described profile, Revance Therapeutics (RVNC) manufactures and develops novel botulinum toxin products for multiple aesthetic and therapeutic indications. The company is leveraging its proprietary portfolio of botulinum toxin compounds combined with its patented TransMTS® peptide delivery system to address unmet needs in the growing markets. Revance’s proprietary TransMTS technology seems to enable …

POSITIVE TOP-LINE RESULTS FROM A PHASE 2 TRIAL OF OMECAMTIV MECARBIL IN PATIENTS WITH CHRONIC HEART FAILURE Data Showed Statistically Significant Improvements in Several Pre-Specified Measures of Cardiac Function Amgen (AMGN) and Cytokinetics (CYTK): Data from the expansion phase of COSMIC-HF – a Phase 2 trial evaluating omecamtiv mecarbil in patients with chronic heart failure, showed statistically significant improvements in several measures of cardiac function, including systolic …

The FDA issued a warning that hepatitis C treatment Viekira Pak and Technivie can cause serious side effects and serious liver injury, mostly in patients with underlying advanced liver disease. The drugs in question belong to AbbVie (ABBV) with some of their  ingredients made by Enanta (ENTA), both were subjected to selloffs after the news, in addition to Express Script (ESRX) Holding, which  operates as a …

– Exelixis submitted rolling NDA for cabozantinib. – Exelixis plans to complete the submission in the next two months. – Positive Results from the METEOR trial with cabozantinib on advanced renal Cell carcinoma led the FDA to grant the drug breakthrough designation. Based on good results from a Phase 3 from the METEOR pivotal trial, Exelixis (EXEL) initiated submission of its rolling New Drug Application (NDA) to …

About Duchenne muscular Dystrophy treatments, bloggers tried to explain the reasons that led the FDA to decide upon reviewing BioMarin’s (BMRN) Duchenne muscular dystrophy (DMD) drug Drisapersen a few weeks earlier than reviewing Sarepta (SRPT) DMD drug Eteplirsen. The real reasons for the FDA decision remain inside the FDA mind. No outsider will be allowed to know anything related to NDA reviews. All the talk and …

Incyte (INCY) and Merck (MRK) announced the expansion of their ongoing clinical collaboration to include a Phase 3 study evaluating the combination of epacadostat,Incyte’s investigational selective IDO1 inhibitor, with Keytruda® (pembrolizumab), Merck’s anti-PD-1 therapy. The trial is for the use of the combination as first-line treatment for advanced or metastatic melanoma. The Phase 3 study, which is expected to begin in the first half of 2016, …

Stories from Yesterday’s Market We are not claiming that the market volatility has subsided and the leadership has shown and is in full control of the chaos. But yesterday biotech stock performance made us feel that investors are regaining their common sense and trying to benefit from the catastrophic plummeting of the member firms stock prices. Today’s trading seems negative for the biotech stocks, but …

– Exclusively licenses global rights to its (TGFb) antibody program to Novartis  – Could the lucrative agreement spare Xoma the pain of raising money for developing its programs, or filing bankruptcy?  – Reasons to believe that Xoma’s TGFb therapeutic antibodies might become another cancer immunotherapy used alone or in combination with immune checkpoint protein inhibitors such as PD-1 inhibitors, which are considered game changers in …

Exelixis (EXEL) announced positive results from the METEOR Phase 3 Pivotal Trial of Cabozantinib in advanced renal cell carcinoma, which was presented at the European Cancer Congress 2015. In the study, Cabozantinib met the primary endpoint of improving progression-free survival as compared to Evorulimus. The drug, which has already been approved and marketed for thyroid cancer has also demonstrated a strong trend towards improving overall …

Aerie Pharmaceuticals (AERI), which is specialized in glaucoma treatments reported successful results of its second Phase 3 trial for Rhopressa™ — a novel once-daily, triple-action eye drops for glaucoma. The trial achieved its primary efficacy endpoint demonstrating non-inferiority of Rhopressa™ compared to timolol, the most widely used comparator. Management will host a conference call and provide accompanying slides to discuss these results at 5:00 p.m. …

The biotechnology industry is proving to be the most innovative and the most growing among Wall Street industries. Individual biotech companies’ achievements complement each other. Some are evolving the sequencing of genes and genetic analysis, while others are excelling in designing drugs based on the tremendous amount of facts they are getting on root causes of the diseases and the interaction of proteins. The biotech …

– The second approval of a cancer drug for Exelixis – The approval of Exelixis/Roche drugs combination will save lives. – The melanoma the combination drugs treat is a killer with bad prognosis  – The terms of Exelixis/Roche agreement are favorable Reiterating our enthusiastic feeling about the future of Exelixis (EXEL), our feeling has transformed into reality and here we are, Swissmedic, the Swiss licensing …

but first: Here is some good news from  SAREPTA Yesterday we wrote about BioMarin (BMRN) being granted FDA “Rare Pediatric Disease Designation for its Duchenne potential muscular dystrophy (DMD) drug drisapersen.Today, Sarepta Therapeutics (SRPT) also a developer of RNA-targeted therapeutics has been granted the same for its drug eteplirsen, also a potential treatment for DMD patients who are amenable to skipping exon 51. The Rare …