Marinus Pharmaceuticals’ (MRNS) stock rallied following its announcement of top-line data from Phase 2 open-label study in patients with the severe epilepsy disorder known as CDKL5. The disease is a rare  severe genetic derived epilepsy that results in seizures, pervasive neuro-developmental delay and disabling behavioral issues. The disease has no approved treatments. The good news that boosted the stock price early today has come out …

In the news, the FDA has placed a clinical hold on two phase 1 trials of Cellectis’ (CLLS) off- the-shelf product UCART123 after the death of a 78-year old patient. Cellectis is working with the FDA towards finding ways that help mitigate the risks expected to have caused the fatality. We experienced deaths with other CAR T products with the cytokine release syndrome on the …

Eli Lilly and Company (LLY) and Incyte (INCY) announced that, after discussions with the U.S. Food and Drug Administration (FDA) in late August, Lilly will resubmit the New Drug Application (NDA) for baricitinib before the end of January 2018. That was fast, we mean the re-submission. To remind you, the firms had submitted Baricitinib for review seeking marketing approval for rheumatoid arthritis in the U.S., the …

Earlier this month, on Aug. 24, 2017, Propeller Health announced the expansion of its 2015 collaboration with GlaxoSmithKline (GSK), enabling both companies to prepare for and undertake commercial activities using the Propeller clip-on sensor and software platform for use with  ELLIPTA Inhaler. The inhaler, which is marketed by GSK, is produced by Innoviva (INVA) and approved by the FDA in other countries around the world. …

CRISPR Therapeutics (CRSP) and Massachusetts General Hospital Cancer Center (MGHCC) have entered into a two-year research collaboration and license option agreement to develop Novel T cell therapies for cancer. As part of the collaboration, CRISPR/Cas9 gene editing technology will be utilized to improve upon current T cell therapies in development addressing unmet needs in both hematologic and solid tumors.  This improvement is expected to occur by combining the …

About Our New Section On Sunday, August 20, 2017 we will post our first “The Week in Review” article. This current week has carried bad news, political hypocrisy and a catastrophic terrorist attack on the people of Spain. The week started following war threatening words between the U.S. and North Korea; between the politicians around the world and in the U.S.; and between everybody and …

AFTER MANY OF THE PROHOST PICKED STOCKS NEARED REACHING THE PROHOST NEAR-TERM TARGETS, recently, external circumstances and exaggerated delaying news, some of our rallied stocks gave back some of their gains. Two reasons were behind the decline in some precious picked stocks, which occurred one after the other in a very short period of time. The first reason for the declines came through traders’ practice …

Synopsis Most of the Prohost picked stocks gained momentum and closed higher at the end of the week. Some rallied because that’s what they were supposed to do after the quarterly financial when most biotech stocks are subjected to selloffs, including those that have clearly outperformed in real life. The most outperforming of our picked stocks in July and continue to outperform are Illumina (ILMN) …

Amgen’s (AMGN) stock outperformed in the past 6 months for significant reasons and these reasons are still there as we observed listening to the firm’s quarterly results. The firm’s results surpassed analysts’ expectations. The stock outperformed because the firm outperformed, never letting its accomplishments emanate from the past successes, but through updating its technologies and products to where the scientific knowledge has reached. That’s the …

Theravance Biopharma (TBPH) and Mylan N.V. (MYL) announced positive results from a 12-month Phase 3 safety study of revefenacin (TD-4208). The drug is an investigational long-acting muscarinic antagonist (LAMA) - a proposed once-daily, NEBULIZED bronchodilator for chronic obstructive pulmonary disease (COPD). The study demonstrated that revefenacin was generally well-tolerated, and no new safety issues were identified. Rates of adverse events (AEs) and serious adverse events …

Yesterday, Wednesday July 19, Vertex’s (VRTX) stock added over 20% to its value, recording a new all time high. Why? On Tuesday the firm announced a better than technical analysts’ and its own expectations’ results in three trials testing its cystic fibrosis (CF) drugs VX-152, VX-440 and VX-659 combined with two the its cystic fibrosis drugs. The positive data came from Phase 1 and Phase …

Arena (ARNA) announced positive Phase 2 clinical trial results for its drug ralinepag1 for pulmonary arterial hypertension (PAH). In the 61-patient study, the primary efficacy analysis demonstrated a statistically significant absolute change from baseline in pulmonary vascular resistance (PVR) compared to placebo. Ralinepag also demonstrated numerical improvement in 6-minute walk distance (6MWD). Additionally, adverse events observed in the study were consistent with other prostacyclin treatments for …

FROM ARRAY Array’s Drug Binimetinib in Combination Therapy with Merck’s Drug Keytruda for METASTATIC FOR COLORECTAL CANCER   On May 8, 2017, Array BioPharma and Merck announced entering into a clinical trial collaboration to investigate the safety and efficacy of Array's MEK inhibitor, binimetinib, with Merck's anti-PD-1 therapy, Keytruda® (pembrolizumab), in metastatic colorectal cancer patients with microsatellite stable tumors (MSS CRC). The decision on this …

As we wrote yesterday, immunotherapy checkpoint inhibitors represent a great advancement and big changes in the management of cancer. It is pertinent, though, to keep in mind that these immuno-oncology products will not be successful with all cancers. Immune checkpoint inhibitors Keytruda (Merck) and Obdivo, Bristol-Myers Squibb and others are already approved around the world for several cancers. We also know that these immunotherapy products …

Additional Combination Treatment with Vectibix  for Metastatic Colorectal Cancer (mCRC) The Old Drug: VECTIBIX                   The Company: AMGEN Colon cancer is easy to cure when discovered early. Missing the diagnosis of early colon cancer could result in cancer growth, direct spread, distal metastasis and death. In spite of the tremendous improvement of treatments, advanced colon cancer is still …

On June 22, 2017, the U.S. Food and Drug Administration (FDA) has approved Genentech’s Rituxan Hycela™. This product is a combination of Genentech’s drug rituximab and Halozyme's hyaluronidase enzyme through Halozymes’s proprietary human ENHANZE® technology. The aim of the combination is changing Rituxan from an intravenously administered product into a subcutaneous (under the skin) injection. The new version Rituxan Hycela is expected to reduce the …

IMPRESSIVE RESULTS?  Bellicum Pharmaceuticals’ (BLCM) drug BPX-501 is described as a safe and effective product that can offer children with cancer and other serious diseases the opportunity to beat their intractable sicknesses. The drug enables the patients who are in desperate need for a bone marrow stem cell transplant to use unmatched donors’ stem cells through lowering their adverse effects. The most recent clinical trial …

Portola (PTLA) announced that the U.S. Food and Drug Administration (FDA) has approved Bevyxxa (betrixaban), the first anticoagulant for hospital and extended duration prophylaxis (35 to 42 days) of venous thromboembolism (VTE) in adult patients hospitalized for an acute medical illness who are at risk for thromboembolic complications due to moderate or severe restricted mobility and other risk factors for VTE. Bevyxxa’s approval is on …

As we wrote in Prohost Letter #409, a transformation in this year’s American Society of Clinical Oncology meeting (ASCO) is further elaborating on Checkpoint inhibition and chimeric antigen receptor T-cell (CAR-T) approaches’ limitations. Both are attempts towards making Immunotherapy for cancer a successful treatment. Checkpoint inhibitors have reached the market at the hands of Merck, Bristo-Myers Squibb and Roche, with other firms coming forward with products …

Alnylam Pharmaceutical (ALNY) calls RNAi (RNA interference) a revolution in biology. The FDA’s just granted the firm’s RNAi therapy breakthrough for a disease nobody has ever dreamed of conquering confirms this reality in spite of spending years of efforts and money towards turning the RNAi molecules into therapeutic molecules that can be used safely and effectively. RNAi-related treatments represent a completely new approach to drug …