Amylyx Pharmaceuticals With the advancement of biological sciences, and increased information about the pathways of diseases, we are getting more interested in biotech firms that are focused on treating life-threatening diseases as the risk of failure is decreasing.  The successful outcome of trials would tremendously boost the values of these firms as we have witnessed in many small biotech firms that became large firms and …

Exelixis Positive Results from IMspire 150 Exelixis (EXEL) announced positive results from IMspire 150; the phase 3 trial of atezolizumab (TECENTRIQ®), cobimetinib (COTELLIC®) and vemurafenib (ZELBORAF®) in people with previously untreated BRAF V600 mutation-positive advanced melanoma.  The IMspire150 study met its primary endpoint of progression-free survival (PFS). Adding atezolizumab to cobimetinib and vemurafenib helped reduce the risk of disease worsening or death, compared to placebo plus …

Gilead’s CAR-T Product Yescarta Results Kite, a Gilead (GILD) subsidiary, announced new data from the ZUMA-1 trial of the CAR T Yescarta (axicabtagene ciloleucel) in adult patients with refractory large B-cell lymphoma. The data from the pivotal phase 2 study demonstrate that patients with refractory large B cell lymphoma were alive three years following a single infusion of Gilead’s CAR T immuno-oncology product Yescarta.  Analysis from …

Novartis to Acquire The Medicines Company Novartis (NVS) decided to buy The Medicines Company (MDCO) at $6.8 million. The Medicines Company’s shareholders will be getting $85 per share in cash  – a 45% premium to its unaffected share price and a 57% premium to its unaffected one-week average price. The Transaction is expected to be completed in the first quarter of 2020 The Medicines Company …

Alnylam Granted FDA Early Approval of GIVLAARI™ for AHP Alnylam’s (ALNY) stock outperformed as its subcutaneous injection GIVLAARI™ (givosiran) has been granted FDA early approval. GIVLAARI™ is created and developed for the treatment of adults with acute hepatic porphyria (AHP). AHP is a family of ultra-rare, genetic diseases characterized by debilitating, life-threatening attacks and, for some patients, chronic manifestations that demeans patients’ daily functioning and …

Vertex Pharmaceuticals and CRISPR Therapeutics Rallied Today Vertex Pharmaceuticals (VRTX) and CRISPR Therapeutics (CRSP) announced positive, interim data from the first two patients with severe hemoglobinopathies treated with the investigational CRISPR/Cas9 gene-editing therapy CTX001 in ongoing Phase 1/2 clinical trials. A patient with transfusion-dependent beta-thalassemia (TDT) received CTX001 in the first quarter of 2019. Data for this patient reflect nine months of safety and efficacy follow-up. …

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 …

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 …

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 …

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 …

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 …

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 …

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 …

Moderna Therapeutics Trial Results Moderna Therapeutics (MRNA) trial results mark the first systemic mRNA therapeutic to show the production of a secreted protein in humans.  Moderna’s Product mRNA-1944 successfully encoded for functional antibody (CHKV-24) in humans at all dose levels tested (0.1, 0.3 and 0.6 mg/kg). The antibody level reached is predicted to protect against chikungunya infection achieved within hours. It is projected to be …

Outperforming Biotechs On August 27, 2019 we posted “The Medicines Company Has Agreeable News” under Today’s Highlights.  The Medicines Company We described The Medicines Company (MDCO) as a firm focused on cardiovascular diseases with a goal of halting the progression of atherosclerosis and lowering the cardiovascular risk caused by high levels of LDL Cholesterol (LDL-C). The Medicines Company’s good news emanated from the results of …

Amgen Agreement with Celgene for Otezla® In the News Amgen (AMGN) entered into an agreement with Celgene (CELG) to acquire worldwide rights to Otezla® (apremilast), the only oral, non-biologic treatment for psoriasis and psoriatic arthritis, and certain related assets and liabilities for $13.4 billion in cash. Amgen cited the benefits of this agreement as follows: A strong fit with Amgen’s long-standing expertise in inflammation and …

AstraZeneca: Farxiga for Heart Failure with Reduced Ejection Fraction  AstraZeneca (AZN) has good news. Many of them are extremely positive. However, the firm’s stock is still struggling to get this pharmaceutical company out of the oversold territory where it has been living since the beginning of the year.    Among the recent good news came the results from a Phase 3 trial using AstraZeneca’s SGLT2 inhibitor …

Akari Therapeutics Stock Rallied Autoimmune and inflammatory disease treatments are works in progress with some approved products demonstrating remarkable efficacy. Still there are untreated inflammatory diseases, among them are those where the complement (C5) and/or leukotriene (LTB4) systems are implicated. Akari Therapeutics (AKTX) is a small biopharmaceutical company focused on therapeutics for the orphan autoimmune and inflammatory diseases.The Company announced that the U.S. Food and …