Celyad (CYAD) is still a clinical-stage biopharmaceutical company developing advanced or improved CAR-T cell-based therapies. Today, Celyad announced that the first patient has received an injection in Phase 1 of the alloSHRINK clinical trial, evaluating its non-gene edited allogeneic CAR-T therapy known as CYAD-101. In this trial, FOLFOX chemotherapy is used simultaneously with CYAD-101 on patients with unresectable metastatic colorectal cancer (mCRC). According to Dr. Christian Homsy, …

Vertex Pharmaceuticals’ triple combination, which added the corrector VX-659 to the tezacaftor and ivacaftor combination, resulted in statistically significant improvements in lung function (percent predicted forced expiratory volume in one second, or ppFEV1) in two Phase 3 studies in people with cystic fibrosis (CF). Data from Vertex’s (VRTX) Phase 3 study in people with one F508del mutation and one minimal function mutation resulted in a mean absolute …

As we expected in our posting yesterday under Today’s Highlights, the U.S. Food and Drug Administration (FDA) approved Loxo Oncology’s (LOXO) Vitrakvi® (larotrectinib), the first oral TRK inhibitor for adult and pediatric patients with solid tumors having a neurotrophic receptor tyrosine kinase (NTRK) gene fusion. The approval is for the cancers that have no known acquired resistance mutation, are metastatic or where surgical resection is likely to result …

We hope you all took advantage of the Thanksgiving holiday to rest and relax. We did. In Today’s NEWS RegenxBio Offers Rocket Pharmaceuticals exclusive, worldwide, rights to adenoviral vector NAV AAV9 for the development of treatments for Danon Disease RegenxBio (RGNX) announced that it awarded an exclusive, worldwide, license agreement to Rocket Pharmaceuticals (RCKT) so it can develop gene therapy for Danon disease. The exclusive license …

This is just to inform that Europe has approved Cabometyx® (cabozantinib) tablets as a monotherapy for hepatocellular carcinoma (HCC). As you know, Cabometyx belongs to Exelixis (EXEL). Today Exelixis announced that its partner, Ipsen, received approval of the drug, from the European Commission (EC), to be used in adults who have HCC and have previously been treated with sorafenib. This approval allows for the marketing of Cabometyx in this indication in …

Aimmune Therapeutics (AIMT) has news. A lot of it is from a successful phase 3 trial for its peanut allergy drug as well as other news that boosts our conviction that this product is, indeed, important and has a good chance for approval in the U.S. and overseas.   Aimmune is a biopharmaceutical company developing treatments for potentially life-threatening food allergies. The firm’s Characterized Oral …

The Week in Review, which we will post soon, is going to provide news that cancer patients, their parents, their oncologists and investors are anxiously waiting to hear.   The news is about studies with products demonstrating huge improvements in immunotherapy efficacy in addition to preventing checkpoint inhibitors such as: Opdivo, Keytruda and others, from causing adverse effects, including autoimmune reactions that are serious and …

In today’s news Jazz Pharmaceuticals (JAZZ) announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency for the approval of solriamfetol.  Solriamfetol is a selective dopamine and norepinephrine reuptake inhibitor (DNRI) in development for treatment of excessive daytime sleepiness in adult patients with narcolepsy, obstructive sleep apnea, and Parkinson’s disease. In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma. The aim of the …

Yesterday, the Stock Market skyrocketed and with it a significant number of Prohost Picks that we keep reminding ourselves are undervalued. The outperformance unveiled the interred strength of firms such as: Illumina (ILMN), Amgen (AMGN) and Vertex (VRTX), the gene therapy firms: RegenxBio (RGNX) and Spark (ONCE), and the gene editing firms: Crispr (CRSP), Intellia (NTLA) and Editas (EDIT), as well as others in the Prohost Portfolio. The rebounding in the …

RegenxBio (RGNX) has granted Abeona Therapeutics (ABEO) an exclusive worldwide license to its NAV AAV9 viral vector (subject to certain non-exclusive rights previously granted for MPS IIIA) for the treatment of four diseases: Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB), Infantile Batten Disease, also known as neuronal ceroid lipofuscinosis type 1 (CLN1 Disease) and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 …

Illumina (ILMN) and Pacific Biosciences (PACB) announced they have signed an agreement for Illumina to acquire Pacific Biosciences at a price of $8.00 per Pacific Biosciences share, in an all-cash transaction. The agreement has already been approved by the board of directors of both firms. The acquisition complements Illumina’s sequencing solutions with accurate long-read sequencing capabilities to answer complex genomic questions. Why the acquisition? While Illumina’s accurate and economic short-read sequencing platforms address the …

Exelixis (EXEL) provided an update that testified to its solid fundamentals, the growth of its approved products’ sales, the professionalism and seriousness of its management. Revenues: Exelixis’ total quarter revenues were $225.4 million vs. $152.5 million for the comparable period in 2017. Its net product sales revenues were $162.9 million for the quarter compared to $96.4 million for the comparable period in 2017, which represents a 69 percent increase year-over-year. …

Celyad (CYAD) announced an exclusive agreement with Horizon Discovery Group plc, for the use of its shRNA technology to generate Celyad’s second non-gene-edited allogeneic platform. The encouragement came from data emanating from preclinical studies demonstrating the versatility of the shRNA platform in the allogeneic setting. The data is expected to be presented at the upcoming 2018 Society for Immunotherapy of Cancer (SITC) annual meeting in Washington, D.C., …

A number of biotech or biopharmaceutical companies have succeeded in the past couple of years in changing their statuses from clinical stage to revenues-generating firms. A number of these firms reside in the Prohost Portfolio, picked up before they generated revenues from their newly approved products’ sales. We are following up on these firms’ news as they come out of their new journeys towards marketing …

Although the tests are for the prevention of a life-threatening metabolic disorder, the prenatal animal testing may be offering the potential to treat many human congenital diseases before birth. The research, which comes from Children's Hospital of Philadelphia (CHOP) and the Perelman School of Medicine at the University of Pennsylvania, is published in Nature Medicine. It offers proof-of-concept for prenatal use of a sophisticated, low-toxicity …

Tanezumab, an investigational humanized monoclonal antibody developed by Pfizer (PFE) and Eli Lilly (LLY), met all three co-primary endpoints in patients with osteoarthritis (OA). The 16-week Phase 3 study, which evaluated subcutaneous administration of tanezumab in patients with OA, demonstrated that the patients who received two doses of tanezumab, separated by eight weeks, experienced a statistically significant improvement in pain, physical function and patients’ overall assessment …

IMMUNOTHERAPY  The Checkpoint Inhibitors The most revered news was the discovery of the checkpoint proteins on the surface of the immune system cells. The checkpoint proteins’ natural job is to put a break on the immune system cells to prevent them from attacking people’s own body cells. An immune attack on host cells can lead to disabling and life-threatening autoimmune diseases. Long before the scientists …

VTV Therapeutics’ (VTVT) stock price added over 48% before trading hours today. The news that some believe is behind investors’ enthusiasm is the firm’s announcement that it will deliver two presentations at the 11th clinical trials on Alzheimer’s Disease (CTAD) Conference in Barcelona, Spain. The oral VTV Therapeutics’ (VTVT) presentations are scheduled for October 24-27, 2018. VTV Therapeutics is a clinical-stage biopharmaceutical company focused on …

In two pivotal Phase 3 placebo-controlled trials Regeneron Pharmaceuticals’ (REGN) and Sanofi’s (SNY) drug, Dupixent® (dupilumab), demonstrated a significant reduction in nasal polyp size, nasal congestion’s severity and the need for systemic corticosteroids and/or surgery. Both trials were evaluating Dupixent® (dupilumab) in adults with inadequately-controlled chronic rhinosinusitis with nasal polyps (CRSwNP). They both met met all their primary and secondary endpoints. Dupixent is a monoclonal antibody designed to …

Amgen (AMGN) announced that AmgevitaTM, a biosimilar to adalimumab, will launch in markets across Europe beginning on October 16, 2018. According to Amgen’s press release, AmgevitaTM is the first adalimumab biosimilar to be approved by the European Commission (EC). The EC approval is based on analytical, pharmacokinetic and clinical data, including results from two Phase 3 confirmatory studies of the product for moderate-to-severe plaque psoriasis and moderate-to-severe rheumatoid arthritis patients. The …