2020-03-23-PharmaCircle-Formulation-DD-Newsletter

2020-03-23-PharmaCircle-Formulation-DD-Newsletter

Week of March 23, 2020
1. PharmaCircle COVID-19 Coverage 2. CRISPR Clinical Trials Begin 3. Kurt’s Drug Delivery & Formulation News Highlights
1. PharmaCircle COVID-19 Coverage
PharmaCircle has initiated specialized COVID-19 coverage. This includes Epidemiology Statistics, Active Pipeline, Diagnostic Tests, Recent News and Clinical Trials. The portal is accessed directly from the PharmaCircle homepage by clicking the COVID-19 image as shown below.
There is so much happening, so quickly, that every attempt is being be made to keep the information updated on a daily basis. If you have any comments or suggestions please contact Customer Support.
2. CRISPR Clinical Trial Begin
With all of the attention being given the the COVID-19 pandemic some news has been falling through the cracks in terms of attention. Editas Medicine and Allergan announced earlier this month the initiation of a clinical trial for AGN-151587 (EDIT-101) for the treatment of Leber congenital amaurosis 10 (LCA10), an inherited form of blindness caused by mutations in the centrosomal protein 290 (CEP290) gene using CRISPR editing. The question we had was – how was this CRISPR medication being delivered? The PharmaCircle drug delivery and formulation team has put together a short summary of the delivery approach and the hoped for therapeutic mechanism. Here is a link to the article. Here is a link to the PharmaCircle product record (PharmaCircle subscription required).
EDIT-101 uses an adeno-associated virus (AAV5) vector to deliver the Staphylococcus aureus Cas9 (the nuclease/gene editor part of CRISPR) and CEP290-specific guide RNAs (gRNAs) (the part of CRISPR which helps the nuclease find the right place to edit) to photoreceptor cells by subretinal injection. The natural tropism of AAV5 for photoreceptor cells, the utilization of the photoreceptor-specific G protein-coupled receptor kinase 1 (GRK1) promoter within the AAV5 vector, and the localized delivery by subretinal injection all serve to limit the expression of the CRISPR/Cas9 system to only the therapeutic target tissue and cell type. The method of viral vector construction and method of administration is given in the publication.
As described in the news, “The goal is that once the virus carrying the CRISPR instructions has been infused into the eye, the gene-editing tool will slice out the mutation that causes a defect in CEP290. That would, the researchers hope, restore production of a crucial protein and prevent the death of cells in the retina, as well as revive other cells — enabling patients to regain at least some vision. The procedure, which takes about an hour to perform, involves making tiny incisions that enable access to the back of the eye. That allows a surgeon to inject three droplets of fluid containing billions of copies of the virus that has been engineered to carry the CRISPR gene-editing instructions under the retina.”
3. Kurt’s Drug Delivery & Formulation News Highlights
(Kurt’s News Highlights as a PDF Download with Links – Week of 2020-03-16) (Kurt’s News Highlights as a PDF Download with Links – Week of 2020-03-09)
Note: only the past week’s news are presented below. The past two weeks’ news highlights are provided as a download using the link above. The downloads include links to the original news item and the PharmaCircle Enhanced Release.
Product Approvals Novartis receives approval from Japanese Ministry of Health, Labour and Welfare for Zolgensma the only gene therapy for patients with spinal muscular atrophy (SMA) (3/19) PharmaCircle Enhanced Release Product Filings Aurinia Pharmaceuticals Initiates Rolling Submission of a New Drug Application to the U.S. Food and Drug Administration for Voclosporin in the Treatment of Lupus Nephritis (3/16) PharmaCircle Enhanced Release Product Deals Atnahs Acquires Rights to Established Brand in Women’s Health Treatment (3/19) PharmaCircle Enhanced Release Tarveda Therapeutics and SciClone Pharmaceuticals International Establish Licensing Agreement for PEN-866 in Greater China (3/19) PharmaCircle Enhanced Release HORAMA Signs Exclusive License Agreement with Leiden University Medical Center Targeting CRB1 Gene Mutations to Treat Inherited Retinal Dystrophies (3/18) PharmaCircle Enhanced Release Emergent BioSolutions Signs Development and Manufacturing Agreement with Vaxart for their Experimental Oral Vaccine Candidate for Coronavirus Disease (3/18) PharmaCircle Enhanced Release Lumosa Knocking on the Door of Turkish Market with LT1001 Long-Acting Analgesic Injection (3/17) PharmaCircle Enhanced Release
Technology Deals Arecor Extends Multi-Product Collaboration With Clinical Stage Biotech (3/19) PharmaCircle Enhanced Release Redbiotec Signs Collaboration Agreement with Large-Cap Pharma to Develop Targeted Living Medicines(3/18) PharmaCircle Enhanced Release PharmaJet Received Multi-Year, Multi-Million Dollar Award to Support Next-Generation Needle-free Delivery Devices (3/17) PharmaCircle Enhanced Release Catalent Partners with Biohaven on New Fast-Dissolve Migraine Treatment (3/16) PharmaCircle Enhanced Release Technology News Applied DNA Leverages COVID-19 Vaccine Development and Proprietary Linear DNA Manufacturing Capability to Design a High Sensitivity Diagnostic Kit for Virus Detection (3/19) – PR Newswire PharmaCircle Enhanced Release Haselmeier receives Medical Device Master File number for D-Flex product platform (3/17) PharmaCircle Enhanced Release Capricor Therapeutics Announces Strategic Plan for Exosome Platform Technology Products Expansion (3/17)PharmaCircle Enhanced Release Drug Delivery Patent News Kuros Strengthens Orthobiologics Patent Portfolio (3/18) PharmaCircle Enhanced Release CURE Pharmaceutical Secures Chinese Patent on its Oral Thin Films to Treat Erectile Dysfunction (3/17) PharmaCircle Enhanced Release Acquisitions Valeritas Receives Court Approval of Sale of Business to Zealand Pharma (3/20) PharmaCircle Enhanced Release AbbVie and Allergan Sign Consent Decree Agreement with Federal Trade Commission Staff on Pending Transaction (3/17) PharmaCircle Enhanced Release Financial/Company News Protalix BioTherapeutics Announces Closing of $43.7 Million Private Placement (3/18) PharmaCircle Enhanced Release Sigilon Therapeutics Announces $80.3 Million Series B Financing to Advance Shielded Living Therapeutics to the Clinic (3/17) PharmaCircle Enhanced Release
Vaxxas Awarded US$5 million [AU$7.5 million] Grant for Clinical Study of Measles and Rubella Vaccination using Vaxxas’ High Density Micro-Array Patch (3/17) PharmaCircle Enhanced Release Seelos Therapeutics Announces Closing of Public Offering of Common Stock (3/16) PharmaCircle Enhanced Release Clarus Therapeutics Announces Financing to Support the Commercial Launch and Availability of JATENZO (testosterone undecanoate) Capsules, CIII for the Treatment of Hypogonadism (3/16) PharmaCircle Enhanced Release Product News Lupin launches Betamethasone Dipropionate Ointment USP (Augmented) (3/20) PharmaCircle Enhanced Release BiomX Discloses Acne Pre-clinical Data Planned for 2020 AAD Annual Meeting (3/20) PharmaCircle Enhanced Release Axsome Therapeutics Accelerates Completion of the ADVANCE-1 Phase 2/3 Trial of AXS-05 in Alzheimer’s Disease Agitation (3/20) PharmaCircle Enhanced Release U.S. Food and Drug Administration (FDA) Grants Emergency IND for Two Coronavirus Patients Treated in New York with CytoDyn’s Leronlimab (3/19) PharmaCircle Enhanced Release
Entos Pharmaceuticals Is Developing a DNA Vaccine Against COVID-19 Using Its Fusogenix Nanomedicine Platform (3/19) PharmaCircle Enhanced Release Algernon to Support Planned Phase 2 Trial of Ifenprodil for Coronavirus (3/19) PharmaCircle Enhanced Release Stabilitech’s COVID-19 Vaccine Intended to Be Delivered in a Disruptive Thermally Stable Oral Capsule (3/19) PharmaCircle Enhanced Release Soligenix Announces Positive Top-line Results for its Pivotal Phase 3 FLASH Trial Evaluating SGX301 in Treatment of Cutaneous T-Cell Lymphoma (3/19) PharmaCircle Enhanced Release BioXcel Therapeutics Provides an Update on its Ongoing Phase 3 SERENITY Trials (3/19) PharmaCircle Enhanced Release Lynparza granted orphan drug designation in Japan for BRCA-mutated metastatic pancreatic cancer (3/19) PharmaCircle Enhanced Release Genentech Initiates Phase III Clinical Trial Of Actemra In Hospitalized Patients With Severe COVID-19 Pneumonia (3/18) PharmaCircle Enhanced Release Aurinia Announces AURORA Phase 3 Clinical Data for Voclosporin in Lupus Nephritis to be Presented at National Kidney Foundation 2020 Spring Clinical Meetings (3/18) PharmaCircle Enhanced Release
Beyond Air Accelerates LungFit BRO COVID-19 Program (3/18) PharmaCircle Enhanced Release Dicerna Receives Orphan Drug Designation From U.S. Food and Drug Administration for DCR-A1AT for Treatment of Alpha-1 Antitrypsin Deficiency (3/18) PharmaCircle Enhanced Release Novartis announces NEJM publication of three pivotal trials showing durable and potent efficacy of inclisiran, an investigational first-in-class siRNA cholesterol-lowering therapy (3/18) PharmaCircle Enhanced Release Novartis Pharmaceuticals Corporation Corrective Action for Certain Blister Packages of SANDIMMUNE and NEORAL 100-Mg Soft Gelatin Capsules in the US Due to Failure to Meet Child-Resistant Packaging Requirements (3/18) PharmaCircle Enhanced Release Follicum pauses recruitment of patients in Phase II clinical trial of FOL-005 as a result of the ongoing COVID-19 pandemic (3/18) PharmaCircle Enhanced Release Biohaven’s Verdiperstat Receives Fast Track Designation For The Treatment Of Multiple System Atrophy(3/18) PharmaCircle Enhanced Release Graybug Vision Concludes Patient Enrollment in its Phase 2b Clinical Trial of GB-102 in Wet Age- Related Macular Degeneration and Accelerates Trial Read-out by Approximately Six Months (3/18) PharmaCircle Enhanced Release Rhythm Pharmaceuticals Receives Orphan Drug Designation from U.S. FDA for Setmelanotide for the Treatment of Alström Syndrome (3/18) PharmaCircle Enhanced Release Synairgen to start trial of SNG001 in COVID-19 imminently (3/18) PharmaCircle Enhanced Release IVERIC bio Provides Update on the Initiation of the Second Pivotal Clinical Trial Enrollment for Zimura in Patients With Geographic Atrophy Secondary to Dry Age-Related Macular Degeneration (3/18) PharmaCircle Enhanced Release IMV Inc. Launches Plans to Advance Clinical Development of a Vaccine Candidate Against COVID- 19 (3/18) PharmaCircle Enhanced Release Regeneron Announces Important Advances in Novel COVID-19 Antibody Program (3/17) PharmaCircle Enhanced Release Epinephrine Nasal Spray Demonstrates Bioequivalent Exposure and Absorption Rate Compared to Epinephrine Injectors (3/17) PharmaCircle Enhanced Release EmphyCorp Nitric Oxide is currently being tested in Clinical Trials as a Potential Treatment for Patients infected with the Coronavirus (3/17) PharmaCircle Enhanced Release SignPath Pharma to Focus on Coronavirus Disease (COVID-19) by Developing Treatment for Acute Respiratory Distress Syndrome (ARDS) (3/17) PharmaCircle Enhanced Release Dr. Reddy’s Laboratories announces the launch of Ziprasidone Mesylate for Injection in the U.S. Market (3/17) PharmaCircle Enhanced Release
Relief Therapeutics Holding SA announces plans to test Aviptadil for the treatment of COVID-induced Acute respiratory distress syndrome (ards). compassionate care protocol to be conducted at rambam healthcare campus in coordination with the government of Israel (3/17) PharmaCircle Enhanced Release Quillivant XR, QuilliChew ER and Dyanavel XR are 100% Made in the U.S.A. and Remain Consistently Supplied During COVID-19 Pandemic (3/17) PharmaCircle Enhanced Release Pneumagen Leverages its Novel Glycan Approach to Target Coronavirus (COVID-19) Infections (3/17) PharmaCircle Enhanced Release Vaxxas Publication in PLoS Medicine Reveals Groundbreaking Clinical Research on Broad Potential of Novel High-Density Microarray Patch (HD-MAP) to Effeciently Deliver Vaccines Directly to the Skin, Enhancing Immunogenicity (3/17) PharmaCircle Enhanced Release EnGeneIC Announces Publication in Cancer Cell of a Scientific Paper Highlighting the Ability of EDV Nanocells to Mount Dual Assault on Cancer Cells (3/16) PharmaCircle Enhanced Release Protalix Biotherapeutics Announces Feasibility Study with Kirin Holdings on the Production of a Novel Complex Protein (3/16) PharmaCircle Enhanced Release The phase II trial of ENERGI-F701, the solution for female hair loss treatment has completed the data analysis(3/16) PharmaCircle Enhanced Release Moderna Announces First Participant Dosed in NIH-led Phase 1 Study of mRNA Vaccine (mRNA-1273) Against Novel Coronavirus (3/16) PharmaCircle Enhanced Release DBV Technologies Provides Update on Viaskin Peanut BLA for Children Ages 4-11 Years (3/16) PharmaCircle Enhanced Release Aptevo Therapeutics Provides Update On Ongoing APVO436 Phase 1 Clinical Trial (3/16) PharmaCircle Enhanced Release Regeneron and Sanofi Begin Global Kevzara (sarilumab) Clinical Trial Program in Patients with Severe COVID-19 (3/16) PharmaCircle Enhanced Release Beyond Air Submits Investigational Device Exemption (IDE) to the United States Food and Drug Administration (FDA) for the Treatment of COVID-19 Patients (3/16) PharmaCircle Enhanced Release Arcutis Announces Inclusion of Children in On-going Pivotal Phase 3 Clinical Trials Evaluating Topical Roflumilast (ARQ-151) as a Potential Once Daily Topical Treatment for Plaque Psoriasis (3/16) PharmaCircle Enhanced Release CytoDyn Files Modified IND and Protocol for Phase 2 Clinical Trial for Treatment of Patients with Coronavirus with Leronlimab (PRO 140) and Advises Correction to Press Release Issued on March 12, 2020 (3/16) PharmaCircle Enhanced Release AGTC Announces Completion of Enrollment in All Adult Dose Groups of its Ongoing Phase 1/2 Clinical
Trials in Patients with Achromatopsia (3/16) PharmaCircle Enhanced Release
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