FDA Approval for Celgene and Acceleron Drug REBLOZYL® The U.S. Food and Drug Administration (FDA) has approved Celgene’s (CELG) and Acceleron Pharma’s (XLRN) drug REBLOZYL® (luspatercept-aamt) for anemia only in adult patients with beta thalassemia who require regular red blood cell (RBC) transfusions.  REBLOZYL® is the first and only FDA-approved erythroid maturation agent, representing a new class of therapy that works by regulating late-stage red blood cell maturation …

Exelixis and Daiichi Sankyo Partner to Prevent and Treat Diabetic Nephropathy Exelixis (EXEL) and Daiichi Sankyo (

Halozyme Therapeutics is Focusing on ENHANZE® Technology Halozyme Therapeutics (HALO) decides to focus on its ENHANZE® drug delivery technology, halt the development activities for PEGPH20 and close its oncology operations. These decisions were made after the failure of HALO-301 Phase 3 clinical study evaluating PEGPH20 as a first-line therapy for patients with metastatic pancreas cancer to reach the primary endpoint of overall survival. The PEGPH20 failure …

Karyopharm Therapeutics Third Quarter Financial Results Generated Revenues For the first time since its establishment Karyopharm Therapeutics (KPTI) financial results report revenues from a strong launch of its first approved product. Indeed, the firm’s third-quarter 2019 financial results are the first quarter in which Karyopharm generated revenues. In early July the U.S. FDA granted the company’s product Xpovio® (selinexor . . . This content is for …

Compugen FDA Approval for COM902 Compugen Announces FDA Clearance of IND Application for COM902 Phase 1 clinical study in patients with advanced malignancies expected to begin in early 2020 Clinical development of COM902 will enable testing the combination treatment with COM701, Compugen’s anti-PVRIG inhibitor  Compugen (CGEN) is a clinical-stage therapeutic discovery and development company utilizing its broadly applicable, predictive computational discovery platforms to identify novel …

IVERIC bio Stock Outperforms IVERIC bio (ISEE) has good news its stock is responding to. A randomized, controlled Phase 2b clinical trial data seem to confirm that the IVERIC bio product Zimura® (avacincaptad pegol), a complement factor C5 inhibitor, has met its prespecified primary endpoint in reducing the rate of geographic atrophy (GA) growth in . . . This content is for paid subscribers. Please …

Regenxbio Misleading Selloff A selloff of Regenxbio (RGNX) stock, following the FDA’s partial hold on the intrathecal administration of AVXS-101, is unwarranted and totally misleading.   Why? Because the selloff of RGNX gave the impression that the gene therapy Zolgensma, using the adeno-associated virus (AAV) vector , developed by Regenxbio, has failed, which is not true. The partial hold was put only on the product …

Spring Bank Pharmaceuticals, Gilead Sciences and HBV If Spring Bank Pharmaceuticals’ (SBPH) product inarigivir in combination with Gilead Sciences’ (GILD) Vemlidy® could validate the current encouraging results of the ongoing trials on chronic hepatitis B virus (HBV) infection, then the small underestimated firm would hand the giant biotech firm a valuable gift that could increase its sales revenues of its liver disease franchise.    The …

Seattle Genetics Announced Positive Results for HER2CLIMB Trial Seattle Genetics (SGEN) announced positive topline results from the HER2CLIMB trial evaluating tucatinib in HER2-positive breast cancer.  The trial compared tucatinib in combination with trastuzumab and capecitabine to trastuzumab and capecitabine

Bayer Decision to Advance IONIS-FXI-LRx for Clotting Disorders On October 9, 2019 Ionis Pharmaceuticals (IONS) announced it had been notified by its partner Bayer (

Hepion Pharmaceuticals CRV431 Decreased Liver Fibrosis and Tumor Burden in Experimental Models EDISON, NJ / ACCESSWIRE / October 16, 2019 / Hepion Pharmaceuticals, Inc. (NASDAQ:HEPA), a biopharmaceutical company focused on the development of therapeutic drugs for the treatment of liver disease . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Reata Pharmaceuticals Omaveloxolone for Friedreich’s Ataxia On October 14, 2019 Reata Pharmaceuticals (RETA) announced the Part 2 portion of its Phase 2 MOXIe trial, with its product omaveloxolone, met its primary endpoint in modifying Friedreich’s ataxia (FA). The largest change occurred in the modified Friedreich’s Ataxia Rating Scale (mFARS) after 48 weeks of treatment.  Patients treated with omaveloxolone 150 mg/day demonstrated a statistically significant, 2.40 point …

RegenxBio Rebounding RegenxBio (RGNX) has the viral vectors which could safely deliver genes into the cells. The firm has improved the adeno-associated viruses making them safe and effective gene delivery vehicles. One of the three currently approved gene therapy products, Zolgensma® for pediatric spinal muscular atrophy (SMA), marketed by Novartis, used RegenxBio’s adeno-associated . . . This content is for paid subscribers. Please click here to …

RegenxBio Interim Data from Phase I/IIa Trial of RGX-314 for Wet AMD RegenxBio (RGNX) announced interim data from the ongoing Phase I/IIa trial of RGX-314 for the treatment of wet age-related macular degeneration (wet AMD). Here are the results as presented by the Company. The results were presented by Jeffrey S. Heier, M.D., Co-President and Director . . . This content is for paid subscribers. Please …

Stock Market Volatility  Stocks can move up and down for various reasons but not all of them have anything to do with the real value of the firms. As a matter of fact, almost all stocks outperform for a while, reverse their course to underperforming, then restart the cycle. The stock market in its entirety does the same thing for reasons that have nothing to …

Nektar Therapeutics Announces Two Studies of NKTR-358 Nektar Therapeutics (NKTR) announced the initiation of two Phase 1b studies of NKTR-358 (LY3471851*), a novel T regulatory (Treg) cell stimulator, one for psoriasis and one for atopic dermatitis. NKTR-358 for Autoimmune and Inflammatory Conditions NKTR-358 is designed to treat autoimmune and inflammatory conditions by correcting the immune system imbalance that results from reduced numbers and impaired function of …

Seattle Genetics Stock Rally Seattle Genetics (SGEN) stock has rallied today. It gained around $10; trading at over $85, nearing the Prohost 2019 $90 target. The reason for investors’ great enthusiasm is Seattle Genetics announcement of a presentation at the European Society for Medical Oncology . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

The Medicines Company ORION-10 Study for ASCVD The Medicines Company (MDCO) announced positive topline results from the ORION-10 Phase 3 clinical trial in patients with atherosclerotic cardiovascular disease (ASCVD). The ORION-10 trial conducted with . . . This content is for paid subscribers. Please click here to subscribe or here to log in.

Vitiligo Vitiligo is a chronic autoimmune disease. The immune system attacks the pigment-producing melanocyte cells causing loss of skin pigmentation. This causes a skin discoloration condition known as vitiligo. The immune system’s attack affects around 0.2% to 2.0% of the world’s population. There are currently no U.S. FDA or European EMA approved products for this condition. Initial vitiligo symptoms start before age 20 and remain …

News from Nektar Nektar Therapeutics (NKTR) announced that new data from patients with metastatic triple-negative breast cancer, who were enrolled in the PIVOT-02 study, will be presented at the upcoming 5th CRI-CIMT-EATI-AACR Cancer Immunotherapy Conference being held from September 25 to September 28, 2019 in Paris, France. The PIVOT-02 study is evaluating NKTR-214 . . . This content is for paid subscribers. Please click here to subscribe …