Category: Today’s Highlights

A Bayer testing of Compugen’s checkpoint protein in human trial boosted our expectations for Compugen’s resurrection

A Bayer testing of Compugen’s checkpoint protein in human trial boosted our expectations for Compugen’s resurrection

Compugen Again On Sept 10, 2018, we posted in The Week in Review #28 news we found very interesting about Compugen (CGEN). The news announced that Compugen has dosed its first patient in Phase 1 clinical trial with its first-in class immunotherapy antibody product COM701 targeting the firm's in house discovered immune checkpoint protein PVRIG. In The Week in Review article we wrote in the beginning, investors …
About Portola’s appointment of a new president. Read Also: Why CRISPR lost $2.90 today

About Portola’s appointment of a new president. Read Also: Why CRISPR lost $2.90 today

Portola Pharmaceuticals Appoints Scott Garland as President and Chief Executive Officer, Effective October 8, 2018. Portola (PTLA) appointed a new president, Mr. Scott Garland, who was Relypsa’s president and previously chief commercial officer and responsible for the integration and growth of the U.S. operations after Relypsa was acquired by Vifor Pharma Group in 2016. Prior to Relypsa, Mr. Garland was executive vice president and chief commercial officer of Exelixis, Inc., …
CRISPR Therapeutics’ new collaboration targeting Diabetes

CRISPR Therapeutics’ new collaboration targeting Diabetes

CRISPR Gene Editing Beyond Bringing Cures to disease-causing genes, CRISPR gene editing technology can change the way diseases are being treated and treat diseases that have yet to find a treatment. CRISPR gene editing can play a key role in solving a lot of problems that prevent breakthrough treatments and approaches from reaching their ultimate goals. A best example is chimeric antigen receptor (CAR T) Cell immunotherapy approach to …
AcelRx Pharmaceuticals: A small currently highlighted firm

AcelRx Pharmaceuticals: A small currently highlighted firm

Today, we see are observing an increased interest in AcelRx Pharmaceuticals (ACRX). The increased investors’ activity happened following the announcement that an Advisory Committee meeting will soon review the firm’s New Drug Application (NDA) for Dsuvia (Sufentanil) developed for the management of moderate-to-severe acute pain in medically supervised settings in adult patients. Sufentanil is an opioid analgesic currently marketed for intravenous (IV) and epidural anesthesia …
CRISPR gene editing firms clear investors’ confusion

CRISPR gene editing firms clear investors’ confusion

Playing buy and sell games with the CRISPR gene editing stocks is sometimes based on scientific articles that meant to caution about possible problems or by patents that were given to some Universities and denied it to others and vice versa. Different Universities back different CRISPR gene-editing firms, which constituted a reason for the volatility of the gene-editing stocks. Finally, CRISPR Therapeutics (CRSP), Intellia Therapeutics …
What happened to Sangamo yesterday?

What happened to Sangamo yesterday?

The disease, mucopolysaccharidosis (MPS) type II, also known as the horrible Hunter syndrome is a killer with no specific treatment anywhere, except enzyme replacement therapy (ERT) that is not stopping the complications of the disease or preventing death. Sangamo (SGMO) made a presentation at the Society for the Study of Inborn Errors of Metabolism in Athens, Greece to demonstrate preliminary results, which included the safety …
Ionis, the FDA and the exaggerated selloff of the firm’s stock

Ionis, the FDA and the exaggerated selloff of the firm’s stock

A complete response letter (CRL) sent by The Division of Metabolism and Endocrinology Products of the United States’ FDA to Ionis Pharmaceuticals (IONS) and its affiliate Akcea Therapeutics (AKCA) regarding Waylivra™ (volanesorsen) carried like usual bad news. The FDA has decided against approving Waylivra, the drug designed to treat the lipid disorder known as familial chylomicronemia syndrome (FCS). Familial chylomicronemia syndrome is an ultra-rare, devastating hereditary …
About Portola

About Portola

Portola (PTLA) has two important approved breakthrough products and an investigational product having promising results on very difficult-to-treat cancers. Instead of soaring following the approval of its two drugs and the good results coming from the investigational third drug, Portola’s stock took several dives, which raised the eyebrows of many investors and motivated more shareholders to sell PTLA. Let us see whether the firm deserves …
Starting the gene therapy chapter in the book of the biotech revolution

Starting the gene therapy chapter in the book of the biotech revolution

WE ARE LIVING AN HISTORIC BIOLOGICAL REVOLUTION. Unfortunately, the undeniably outstanding news has not yet penetrated the ears of many investors. They are still falling victim to the stock market traders’ games and to critics’ and bloggers’ magnifications or fabrications of negative news. Many investors end up selling their thoughtfully chosen highly scientific biotech firms to regret later on what they have done when their …
Vertex has good news from Australia. Why selling solid firms stocks.

Vertex has good news from Australia. Why selling solid firms stocks.

Vertex Pharmaceuticals (VRTX) announced that the Pharmaceutical Benefits Advisory Committee’s (PBAC) in Australia recommendation the listing of Orkambi® (lumacaftor/ivacaftor) on the Pharmaceutical Benefits Scheme (PBS). The recommendation is for cystic fibrosis people ages six and over who have two copies of the F508del mutation in the cystic fibrosis transmembrane conductance regulator (CFTR) gene. Many thousands of patients worldwide are already receiving lumacaftor/ivacaftor in countries where it is reimbursed …
Regeneron: Two good news and a rebound on the road

Regeneron: Two good news and a rebound on the road

The FDA Approves Regeneron’s Product EyLea® (aflibercept) sBLA in Wet Age-Related Macular Degeneration Regeneron (REGN) has two good news. The first is from one of its approved products, EYLEA® announcing that the U.S. Food and Drug Administration(FDA) has approved a supplemental Biologics License Application (sBLA) for EYLEA® (aflibercept) Injection in patients with wet age-related macular degeneration (wet AMD). The sBLA was based on second-year data from the …
Why Portola couldn’t flex its muscles or use them yet

Why Portola couldn’t flex its muscles or use them yet

All the buying and selling of Portola (PTLA) will not make any sense to us until the firm fulfills its obligations towards what is required by the authorities, by health care and by private third-party payers.  So, in the reporting of its Q2 financial results for the three months ending June 30, 2018, in addition to providing a corporate update, nothing matters really except knowing where the …
Spark Therapeutics: A usual reaction to SPK-8011 results should have been a celebration

Spark Therapeutics: A usual reaction to SPK-8011 results should have been a celebration

When an investigational gene therapy gets excellent results, the usual reaction towards the developing firm is a celebration. This is especially true when the developing firm is Spark Therapeutics (ONCE), which has alreadyvalidated its gene therapy approach through the approval of its product LuxturnaTM (voretigene neparvovec-rzyl).         In its quarterly financial report, Spark said that 12 participants in Phase I/II trial investigating SPK-8011 for hemophilia A
Exelixis: Why Q2 results are more than impressive

Exelixis: Why Q2 results are more than impressive

Exciting are the Q2 2018 reported results by Exelixis’ (EXEL) including the firm’s revenues and earnings, compared to 2017. Impressive were Cabometyx’ (cabozantinib) revenues, in addition to the milestone payments from collaborating companies and ex-USA marketing agent. Inspiring also are the efforts the firm is spending on research and development with the aim of further boosting its revenues. Of the Results Revenues and Incomes – …
The FDA approved Neurocrine’s & AbbVie’s Endometriosis drug, Orilissa. Neurocrine stock met & surpassed the Prohost 2018 target

The FDA approved Neurocrine’s & AbbVie’s Endometriosis drug, Orilissa. Neurocrine stock met & surpassed the Prohost 2018 target

AbbVie (NYSE: ABBV), in cooperation with Neurocrine Biosciences (NBIX), announced that the United States FDA approved Orilissa™ (elagolix) under priority review for women with moderate to severe endometriosis pain. Orilissa represents the first FDA-approved oral treatment for moderate to severe endometriosis pain in over a decade. The drug is expected to be available in U.S. retail pharmacies in early August 2018. Endometriosis is a condition where …
Biogen’s Exciting Q2 Results Highlighted Ionis (IONS) Too

Biogen’s Exciting Q2 Results Highlighted Ionis (IONS) Too

The good news from Biogen’s (BIIB) Q2 exciting financial results comprised impressive news for Ionis (IONS) from the commercialization of Ionis’ approved product, Spinraza for Spinal muscular atrophy (SMA).  Biogen is also responsible for the commercialization of Ionis’ products Tegsedi™ (inotersen) and Waylivra™ (volanesorsen) – two antisense drugs that Ionis discovered and successfully advanced through Phase 3 studies. In the second quarter of Biogen’s 2018 …
About PTC Therapeutics’ Acquisition of Agilis Biotherapeutics

About PTC Therapeutics’ Acquisition of Agilis Biotherapeutics

Yesterday’s news unveiled that PTC Therapeutics (PTCT) has entered into an agreement to acquire Agilis Biotherapeutics. The transaction was approved by the Boards of both companies. The acquisition Agilis Biotherapeutics is expected to bring to PTC Therapeutics an innovative gene therapy platform for rare monogenic diseases that affect the central nervous system. It is supposed to add to PTC Therapeutics’ pipeline an advanced gene therapy candidate, …
Once again, we say, CRISPR is here to stay

Once again, we say, CRISPR is here to stay

A Miracle-Like Gene-Editing That People will Sooner or Later Recognize. The Wellcome Sanger Institute’s new findings about CRISPR/Cas9 gene editing is intended to lead to further improvement in the safety of the breakthrough CRISPR gene editing procedure before it is used in clinical practice; the information is not intended to condemn it. Unfortunately, innocent investors got panicky when they heard the Wellcome Sanger findings, which …
Amgen and UCB resubmit BLA to the FDA for the approval of Evenity™ to prevent or heal Osteoporosis bone fracture in post-menopausal women

Amgen and UCB resubmit BLA to the FDA for the approval of Evenity™ to prevent or heal Osteoporosis bone fracture in post-menopausal women

The BLA Includes Data from More Than 11,000 Patients Amgen (AMGN) and UCB (Euronext Brussels: UCB) resubmitted a Biologics License Application (BLA) to the U.S. FDA for the approval of their osteoporosis investigational monoclonal antibody product Evenity™ (romosozumab). The product is indicated for osteoporosis in postmenopausal women at high risk for fracture.The drug increases bone formation and reduces bone resorption simultaneously to increase bone mineral …
Exelixis liver cancer results are gratifying, so do not worry if some pretend they do not like them

Exelixis liver cancer results are gratifying, so do not worry if some pretend they do not like them

The New England Journal of Medicine (NEJM) published results from the CELESTIAL phase 3 pivotal trial of Exelixis (EXEL) product Cabometyx® (cabozantinib) in previously treated patients with advanced hepatocellular carcinoma (HCC). The data demonstrate that Cabometyx provided a statistically significant and clinically meaningful improvement in overall survival (OS) versus placebo. It is important to know that the clinical trial results formed the basis of regulatory …