Category: News & Comments

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

FDA Granted Rare Pediatric Disease Designation for RegenxBio’s Product RGX-181 for CLN2 RegenxBio 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. RegenxBio is a leading clinical-stage biotechnology company that promises to cure genetic-derived diseases with its gene therapy. RegenxBio’s NAV Technology Platform is …
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 …
Loxo Oncology product Vitrakvi® has been granted FDA approval. The real meaning of the approval

Loxo Oncology product Vitrakvi® has been granted FDA approval. The real meaning of the approval

As we expected in our posting yesterday under Today’s Highlights, the U.S. Food and Drug Administration (FDA) approved Loxo Oncology’s (LOXO) Vitrakvi® (larotrectinib), the first oral TRK inhibitor for adult and pediatric patients with solid tumors having a neurotrophic receptor tyrosine kinase (NTRK) gene fusion. The approval is for the cancers that have no known acquired resistance mutation, are metastatic or where surgical resection is likely to result …
What the Aimmune and Nestlé Health Science agreement tells us

What the Aimmune and Nestlé Health Science agreement tells us

Aimmune Therapeutics (AIMT) has news. A lot of it is from a successful phase 3 trial for its peanut allergy drug as well as other news that boosts our conviction that this product is, indeed, important and has a good chance for approval in the U.S. and overseas.   Aimmune is a biopharmaceutical company developing treatments for potentially life-threatening food allergies. The firm’s Characterized Oral …
Jazz Pharmaceuticals has good, not bad news

Jazz Pharmaceuticals has good, not bad news

In today’s news Jazz Pharmaceuticals (JAZZ) announced the submission of a Marketing Authorization Application (MAA) to the European Medicines Agency for the approval of solriamfetol.  Solriamfetol is a selective dopamine and norepinephrine reuptake inhibitor (DNRI) in development for treatment of excessive daytime sleepiness in adult patients with narcolepsy, obstructive sleep apnea, and Parkinson’s disease. In 2014, Jazz Pharmaceuticals acquired a license to develop and commercialize solriamfetol from Aerial Biopharma. The aim of the …
RegenxBio: Another licensing agreement providing additional validation of its NAV platform

RegenxBio: Another licensing agreement providing additional validation of its NAV platform

RegenxBio (RGNX) has granted Abeona Therapeutics (ABEO) an exclusive worldwide license to its NAV AAV9 viral vector (subject to certain non-exclusive rights previously granted for MPS IIIA) for the treatment of four diseases: Sanfilippo syndrome type A (MPS IIIA) and type B (MPS IIIB), Infantile Batten Disease, also known as neuronal ceroid lipofuscinosis type 1 (CLN1 Disease) and Juvenile Batten Disease, also known as neuronal ceroid lipofuscinosis type 3 …
About Illumina’s acquisition

About Illumina’s acquisition

Illumina (ILMN) and Pacific Biosciences (PACB) announced they have signed an agreement for Illumina to acquire Pacific Biosciences at a price of $8.00 per Pacific Biosciences share, in an all-cash transaction. The agreement has already been approved by the board of directors of both firms. The acquisition complements Illumina’s sequencing solutions with accurate long-read sequencing capabilities to answer complex genomic questions. Why the acquisition? While Illumina’s accurate and economic short-read sequencing platforms address the …
Observing Array Biopharma

Observing Array Biopharma

A number of biotech or biopharmaceutical companies have succeeded in the past couple of years in changing their statuses from clinical stage to revenues-generating firms. A number of these firms reside in the Prohost Portfolio, picked up before they generated revenues from their newly approved products’ sales. We are following up on these firms’ news as they come out of their new journeys towards marketing …