DMD Data by Giant Pharma Pfizer Kept Sarepta In the Driver Seat with Superior Drug

Pfizer (PFE) presented its first set of data (n=6) from the mini-dystrophin gene therapy for DMD (PF-06939926) at the PPMD conference on June 28th. Much speculations surrounded Pfizer’s decision to report the dystrophin quantification using mass-spec. Sarepta (SRPT) has used WB-western blot since 2015 during the Eteplirsen FDA approval process. Sarepta has also used it in […]

SRPT – Again, WMS-2018 Dystrophin Data Beating the Market Expectation

Yesterday 10/04, at 7:00 am ET, Sarepta Therapeutics (SRPT) with Dr. Mendell released the biopsy data from the fourth patient including full 4 patients “Functional Data” from Argentina at #WMS2018 the 23rd International Annual World Muscle Society meeting Link to the full slide deck: https://www.sec.gov/Archives/edgar/data/873303/000119312518292068/d616670dex992.htm The after-hours share price reaction was wild swing, spiking up […]

SRPT – Preparing for WMS-2018 Presentation

Tomorrow 10/04, at 7:00 am ET, Sarepta Therapeutics (SRPT) with Dr. Mendell are hosting a conference call from Argentina at #WMS2018 the 23rd International Annual World Muscle Society meeting, to present and discuss new data from the micro-dystrophin gene-therapy study Ph1/2. Link to SRPT webcast page: http://investorrelations.sarepta.com/events-and-presentations Back in June 19th at the R&D day SRPT […]

XENE – Quick note, Positive TMS Data Presented at The European Congress on Epileptology

Today morning, Xenon Pharmaceutical (XENE) reported positive data from its XEN1101 Phase 1b transcranial magnetic stimulation (TMS) study. (LINK) XEN1101 Demonstrates Statistically Significant Reduction in Corticospinal and Cortical Excitability as Measured by TMS-EMG and TMS-EEG XEN1101 Demonstrates Greater Effect on TMS-EMG Resting Motor Threshold at Significantly Lower Dose When Compared to Historical Ezogabine Data XEN1101 […]

Bellus – David vs. Goliath Can BLU Deliver A Better Chronic Cough Drug Than Merck?

BELLUS Health (BLU.TO) (BLUSF) is a biopharmaceutical development company based in Quebec, Canada, its pipeline includes several partnered clinical-stage drug development programs, but the company’s lead drug candidate is BLU-5937 which is a highly selective P2X3 antagonist for chronic cough and has the potential to be a best-in-class therapeutic for chronic cough patients who do […]

XENE – Epilepsy and Advocacy Under the New FDA

Xenon Pharmaceuticals (XENE) is a clinical stage biopharmaceutical company focusing on discovering and developing innovative therapeutics for neurological disorders. XENE has an extensive knowledge of human genetics and diseases caused by mutations in ion channels, known as channelopathies, focusing their efforts on novel product pipeline of CNS-central nervous system therapies, to address areas of high […]

FDA GT Draft Guidance is Bullish for Sarepta's DMD Pivotal Trial

Yesterday July 11, 2018 the FDA FDA issues 6 new draft guidance documents for gene therapy, clinical development and manufacturing guidance. Human Gene Therapy for Rare Diseases – FDA Draft Guidance for Industry https://www.fda.gov/downloads/BiologicsBloodVaccines/GuidanceComplianceRegulatoryInformation/Guidances/CellularandGeneTherapy/UCM610802.pdf   The FDA guidance noting the pre-existing antibody to GT, this is the strongest SRPT’s & Dr. Mendell point, choosing the AAVrh74 which […]

PSTI – Ahead of IC Phase-2 Top-Line Results

PLURISTEM (PSTI) a cell therapy company, developer of placenta-based cell therapy product candidates for the treatment of multiple ischemic, inflammatory and hematologic conditions, company I’m following for years.  Pluristem trading under the radar and getting even more interesting these days as they already enter late-stage trials in 3 indications, gained the FDA’s Fast Track Designation, EMA’s […]