Category: Today’s Highlights

After being approved for Eczema, Regeneron’s and Sanofi’s product, Dupixent, succeeded in treating chronic rhinosinusitis, nasal polyps and asthma.

After being approved for Eczema, Regeneron’s and Sanofi’s product, Dupixent, succeeded in treating chronic rhinosinusitis, nasal polyps and asthma.

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 …
Spark Therapeutics: An encouraging attempt to dethrone enzyme replacement as the standard of care treatment for Pompe Disease.

Spark Therapeutics: An encouraging attempt to dethrone enzyme replacement as the standard of care treatment for Pompe Disease.

On Saturday, October 6, 2018, Spark Therapeutics (ONCE) presented positive data from a preclinical trial with SPK-3006 for the treatment of Pompe disease in an oral presentation at the 23rd International Congress of the World Muscle Society in Argentina. SPK-3006 is an investigational liver-directed adeno-associated viral (AAV) gene therapy being developed for Pompe disease. Pompe disease is caused by the buildup of a complex sugar, glycogen, in the body’s cells. It is …
Today’s Highlighted news is from …

Today’s Highlighted news is from …

Today, Prohost is highlighting news that could be transformative for a clinical-stage immunotherapy firm. The firm with promising news focuses on creating and developing safer and far-reaching Chimeric Antigen Receptor T-cell (CAR T) Therapy.   The firm we are highlighting today is Celyad (CYAD), whose investigational CAR T products are allogeneic, rather than autologous, which makes it easier to use and less expensive than the autologous products. …
PTC Therapeutics: Solid data from its oral product Risdiplam in spinal muscular atrophy

PTC Therapeutics: Solid data from its oral product Risdiplam in spinal muscular atrophy

PTC Therapeutics’ (PTCT) product Risdiplam demonstrated safety and tolerability at all the doses used in the studies on Type 1, 2 and 3 spinal muscular atrophy (SMA). The good news emanated from interim data of the Part 1, open-label studies of FIREFISH and SUNFISH trials, with the oral drug Risdiplam on SMA patients. Risdiplam was dubbed effective,  substantiated by a confirmed increase in motor function. …
Seattle Genetics and Takeda Pharmaceutical Limited announced a successful trial on mature T-Cell lymphoma

Seattle Genetics and Takeda Pharmaceutical Limited announced a successful trial on mature T-Cell lymphoma

Seattle Genetics (SGEN) and Takeda Pharmaceutical Limited (TKPYY) announced results from Phase 3 ECHELON-2 clinical trial evaluating Adcetris as part of a frontline combination chemotherapy regimen in patients with previously untreated CD30-expressing peripheral T-cell lymphoma (PTCL), known also as mature T-cell lymphoma (MTCL). The data from the trial with Seattle Genetics' conjugated monoclonal antibody Adcetris (brentuximab vedotin) in combination with CHP (cyclophosphamide, doxorubicin, prednisone) versus the control …
AstraZeneca: Advancing and improving Stage III lung cancer treatments

AstraZeneca: Advancing and improving Stage III lung cancer treatments

Stage III (locally-advanced) NSCLC is commonly divided into three sub-categories: IIIA, IIIB and IIIC, defined by how much the cancer has spread locally and the possibility of surgery. The difference between Stage III and Stage IV disease (cancer (metastasized to distant organs)) is that Stage III is treated now with curative intent. The majority of Stage III NSCLC patients are diagnosed with unresectable tumors. It …
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 – …