Category: News & Comments

The FDA approves the first ever postpartum depression product. Positive results from Dermira’s atopic dermatitis product Lebrikizumab

The FDA approves the first ever postpartum depression product. Positive results from Dermira’s atopic dermatitis product Lebrikizumab

News We Loved to Hear  The First and Only Approval of a Specific Product for Postpartum Depression The FDA granted approval of Sage Therapeutics’ (SAGE) ZULRESSOTM (brexanolone) injection. A product for the treatment of the condition known as postpartum depression (PPD). Zulresso became the first and only medicine specifically approved to treat PPD; the most common medical complication of childbirth. The product is expected to be available …
Atossa Genetics’ news that staged a huge Rally of this small firm’s stock

Atossa Genetics’ news that staged a huge Rally of this small firm’s stock

Atossa Genetics (ATOS), a clinical-stage biopharmaceutical company developing novel therapeutics and delivery methods to treat breast cancer and other breast conditions,  announced that the FDA has issued a “Safe to Proceed” letter under their “expanded access” program permitting the use of Atossa’s oral Endoxifenas; a post-mastectomy treatment in a pre-menopausal, estrogen-receptor positive (ER+) breast cancer patient. This patient completed a 3-week course of Atossa’s oral …
Spark Therapeutics to be acquired by Roche at $113.47 UP $61.91 

Spark Therapeutics to be acquired by Roche at $113.47 UP $61.91 

We congratulate our subscribers who kept Spark Therapeutics (ONCE) in spite of the stock’s kind of paralysis for no serious reasons. Spark – a fully integrated, commercial gene therapy company; announced today that it has entered into a definitive merger agreement with Roche (RHHBY). The firm is to fully acquire Spark Therapeutics at a price of $114.50 per share in an all-cash transaction. This move represents  a total equity …
Merck’s product Keytruda and Exelixis’ product Cabometyx are both winners

Merck’s product Keytruda and Exelixis’ product Cabometyx are both winners

The pivotal Phase 3 KEYNOTE-240 trial evaluating Merck’s (MRK) checkpoint inhibitor anti-PD-1 therapy plus best supportive care, for the treatment of patients with advanced hepatocellular carcinoma (HCC) who were previously treated with systemic therapy, did not meet its co-primary endpoints of overall survival (OS) and progression-free survival (PFS) compared with placebo plus best supportive care. The improvement of OS results demonstrated in the final analysis did …
RegenxBio: An analyst and investor event focused on the firm’s ophthalmology program.

RegenxBio: An analyst and investor event focused on the firm’s ophthalmology program.

RegenxBio (RGNX), a clinical-stage gene therapy firm with a proprietary NAV® Technology Platform, will host an analyst and investor event focused on its ophthalmology program; including its lead product candidate RGX-314, on Thursday, February 21, 2019, from 9:00 a.m. to 12:00 p.m. EST in New York City. A live webcast will be available on the company’s website. The agenda will include discussion on RegenxBio’s NAV® Technology Platform, a review of the market landscape and unmet need …
Successful results were announced by uniQure for its gene therapy, AMT-061, on hemophilia B patients

Successful results were announced by uniQure for its gene therapy, AMT-061, on hemophilia B patients

Gene therapy firm, uniQure (QURE), announced updated clinical data in patients treated in the ongoing Phase 2b study of AMT-061; an investigational AAV5-based gene therapy containing a patent-protected  FIX9-Padua (Factor 9) for the treatment of patients with severe and moderately severe hemophilia B. The announced data, which were presented in an oral session at the Annual Congress of the European Association for Hemophilia and Allied Disorders (EAHAD) ,demonstrated that therapeutic levels of Factor …
RegenxBio product  RGX-181 has been granted FDA Rare Pediatric Disease Designation for Batten Disease Condition known as CLN2

RegenxBio product RGX-181 has been granted FDA Rare Pediatric Disease Designation for Batten Disease Condition known as CLN2

RegenxBio (RGNX), a development-stage firm that focuses on designing and developing gene therapy based on its proprietary NAV® Technology Platform, announced that the U.S. FDA granted Rare Pediatric Disease Designation to its RGX-181 therapy. RGX-181 is a one-time treatment candidate for late-infantile neuronal ceroid lipofuscinosis type 2 (CLN2) disease, one of the most common forms of Batten disease. The disease is caused by mutations in the tripeptidyl …
OncoCyte liquid biopsy proves its accuracy and superiority in the differential diagnoses of Lung Cancer

OncoCyte liquid biopsy proves its accuracy and superiority in the differential diagnoses of Lung Cancer

Can a liquid biopsy test be accurate enough for physicians to rely on in differentiating between malignant and benign lung nodules in early stage lung cancer and probably other cancers?  A Yes answer has come out from OncoCyte Corporation (OCX) which announced positive results from its key R&D Validation study demonstrating the accuracy of the Company’s DetermaVu™ liquid biopsy test for lung cancer.  The R&D Validation study …
CompuGen demonstrates the importance of its product COM701 targeting PVRIG in immunotherapy

CompuGen demonstrates the importance of its product COM701 targeting PVRIG in immunotherapy

Data published in Cancer Immunology Research indicate monotherapy and combination opportunities for COM701, a first-in-class cancer immunotherapy antibody targeting PVRIG CompuGen (CGEN) announced the online publication of preclinical data demonstrating the role of PVRIG as a novel immune checkpoint and the potential of its first-in-class inhibitory antibody product COM701 targeting PVRIG, to serve as an effective cancer immunotherapy. The findings were published in two peer-reviewed papers in …
Another drug approval for Exelixis. Why Alnylam’s stock is under pressure today

Another drug approval for Exelixis. Why Alnylam’s stock is under pressure today

More good news came  from Exelixis (EXEL). It was expected by many but not by those who ignore the good news in favor of any negative news or lack of news. The announced news is that the FDA approved Exelixis’ Cabometyx® (cabozantinib) for hepatocellular carcinoma (HCC) in patients who have been previously treated with sorafenib. More good news is Exelixis’ announcement that the firm is prepared to fully support the …
Why the new approval of Exelixis’ blood pressure drug Minnebro in Japan is important

Why the new approval of Exelixis’ blood pressure drug Minnebro in Japan is important

Exelixis collaborator, Daiichi Sankyo, Receives Regulatory Approval for MINNEBRO™ (Esaxerenone) Tablets for Hypertension in Japan Exelixis (EXEL) and Daiichi Sankyo announced that the the Japanese Ministry of Health, Labor and Welfare (MHLW) has approved Minnebro™ (esaxerenone) tablets of ,1.25 mg, 2.5 mg, and 5 mg, for hypertension. Minnebro was identified during a prior research collaboration between Exelixis and Daiichi Sankyo, which was entered into in March 2006. The product has been subsequently developed …
The importance of the FDA granting Fast Track Designation to Vertex’s and CRISPR’s Product CTX-001

The importance of the FDA granting Fast Track Designation to Vertex’s and CRISPR’s Product CTX-001

Earlier this month the U.S. Food and Drug Administration (FDA) granted Fast Track Designation for CTX001 for sickle cell disease (SCD). CTX001 is in clinical trials conducted by CRISPR Therapeutics (CRSP) and Vertex Pharmaceuticals (VRTX). It is an investigational, autologous, gene-edited hematopoietic stem cell therapy for patients suffering from severe hemoglobinopathies. The FDA’s Fast Track program is designed to facilitate the development and expedite the review of drugs to …
Rhythm Pharmaceutical: Updates on trial results from genetic-derived obesity

Rhythm Pharmaceutical: Updates on trial results from genetic-derived obesity

Rhythm Pharmaceuticals, Inc. (RYTM) is a biopharmaceutical company focused on the development and commercialization of therapeutics for rare genetic disorders of obesity. It is currently evaluating the efficacy and safety of setmelanotide, the company’s first-in-class MC4R agonist in Phase 3 studies, in patients who suffer from POMC deficiency obesity, LEPR deficiency obesity, BBS and Alström Syndrome. It is obvious that this firm has taken advantage of …
Gilead: On the road towards realizing its goals. A new collaboration for fibrotic diseases

Gilead: On the road towards realizing its goals. A new collaboration for fibrotic diseases

Gilead Sciences (GILD) and Scholar Rock Holding Corporation (SRRK) announced entering into a strategic collaboration to discover and develop highly specific inhibitors of transforming growth factor beta (TGFβ) activation for the treatment of fibrotic diseases. Under the terms of the agreement Gilead has exclusive options to license worldwide rights to product candidates that emerge from the following three Scholar Rock TGFβ programs:  – Inhibitors that target activation of latent …
Agenus: Signing a lucrative and rewarding agreement with Gilead

Agenus: Signing a lucrative and rewarding agreement with Gilead

Agenus (AGEN) announced recently that it entered into an immuno-oncology (I-O) partnership with Gilead Sciences (GILD). The partnership is focused on developing and commercializing up to five novel I-O therapies. The terms of the agreement Agenus is entitled to receive a $120 million upfront cash payment and a $30 million equity investment.   The agreement also includes approximately $1.7 billion in potential future fees and milestones. …
AstraZeneca: Excellent collaborations. Moderna Therapeutics: Turning public. Will Prohost add it to its Portfolio as promised?

AstraZeneca: Excellent collaborations. Moderna Therapeutics: Turning public. Will Prohost add it to its Portfolio as promised?

In the NEWS AstraZeneca (AZN) announced a collaboration with Cancer Research UK to launch a center of excellence in genetic screening, cancer modelling and big data processing aimed at accelerating the discovery of new cancer medicines. During the announcement of the news, Mene Pangalos, the firm’s Executive Vice President, Innovative Medicines & Early Development, said, the best science doesn’t happen in isolation, which is why AstraZeneca …
A follow up on Gilead’s latest news

A follow up on Gilead’s latest news

The Chinese National Medical Products Administration (NMPA) has approved Gilead’s (GILD) HIV combination product Descovy®.  In China, Descovy is to be indicated in combination with other antiretroviral agents for the treatment of human immunodeficiency virus type 1 (HIV-1) in adults and adolescents (aged 12 years and older with a body weight of at least 35 kg). TAF, which is part of the combination treatment, is a novel, targeted prodrug of …
Exelixis initiates a phase 3 trial with cabozantinib combination for previously untreated liver cancer

Exelixis initiates a phase 3 trial with cabozantinib combination for previously untreated liver cancer

Exelixis (EXEL) and Ipsen (Euronext: IPN; ADR: IPSEY) announced the initiation of COSMIC-312. A phase 3 pivotal trial of cabozantinib (CABOMETYX®) in combination with atezolizumab (TECENTRIQ®) versus sorafenib, in previously untreated advanced hepatocellular carcinoma (HCC). The co-primary endpoints of the trial are: progression-free survival and overall survival. An exploratory arm will also evaluate cabozantinib monotherapy in this first-line setting. COSMIC-312 is a multicenter, randomized, controlled phase 3 pivotal trial that …
Celgene has important CAR T presentations at ASH

Celgene has important CAR T presentations at ASH

At the 60th American Society of Hematology (ASH) Annual Meeting   CAR T is Still a Star With all the pessimistic views critics have sprayed around in the past few years about the cell therapy for cancer, the CAR T approach remained a star of prominent cancer gatherings. This phenomenon confirms the value of the FDA decision of approving the CAR T products, in spite of their …
Vertex: Offering proof of leadership in treating Cystic Fibrosis

Vertex: Offering proof of leadership in treating Cystic Fibrosis

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 …