ATH434 / Alterity Therap
Alterity Therapeutics up big on advancement of ATH434 for Parkinsonian disorder (SeekingAlpha) – Jun 30, 2020 – “Alterity Therapeutics…is up 71% premarket after receiving FDA guidance on a development pathway for ATH434…including feedback on the design of a Phase 2 trial. Alterity reached agreement with the agency on the non-clinical investigations required to support the mid-stage study, along with patient population, safety monitoring plan, and strategy for evaluating drug exposure during the trial.”
Trikafta (elexacaftor/tezacaftor/ivacaftor) / Vertex; Kalydeco (ivacaftor) / Vertex
Vertex Announces Expansion of Reimbursement Agreement With NHS England to Include KAFTRIO (ivacaftor/tezacaftor/elexacaftor) in Combination With KALYDECO (ivacaftor) (Vertex Press Release) – Jun 30, 2020 – “Vertex…today announced that it has expanded its reimbursement agreement with NHS England for Vertex’s cystic fibrosis medicines to include KAFTRIO® (ivacaftor/tezacaftor/elexacaftor), in a combination regimen with KALYDECO® (ivacaftor) 150 mg, ahead of the medicine’s anticipated approval by the European Commission.”
Zygel (cannabidiol gel) / Zynerba
Zynerba Pharmaceuticals Announces Top Line Results from Pivotal CONNECT-FX Trial of Zygel (CBD Gel) in Fragile X Syndrome (GlobeNewswire) – Jun 30, 2020 – P2/3, N=204; CONNECT-FX (NCT03614663) ; Sposnor: Zynerba Pharmaceuticals; “Zynerba Pharmaceuticals, Inc….today announced top line results from the 14-week pivotal CONNECT-FX….Zygel did not achieve statistical significance versus placebo in the primary endpoint of improvement in the Social Avoidance subscale….Zygel also did not demonstrate statistical significance versus placebo in the three key secondary endpoints….A pre-planned ad hoc analysis…demonstrated that patients receiving Zygel achieved statistical significance in the primary endpoint of improvement at 12 weeks….Upcoming Corporate Milestones: Fragile X syndrome: Meet with the FDA to discuss CONNECT-FX results as soon as possible; Zynerba intends to meet with FDA to discuss the positive Phase 2 BRIGHT trial results and clinical path forward in 2H2020.”
Melflufen (melphalan flufenamide) / Oncopeptides; dexamethasone / Generic mfg.
Oncopeptides submits a New Drug Application to the FDA for accelerated approval of melflufen in triple-class refractory multiple myeloma patients (Oncopeptides Press Release) – Jun 30, 2020 – “Oncopeptides…today announces that the Company submits a New Drug Application (NDA) to the U.S. Food and Drug Administration, FDA, for accelerated approval of melflufen (INN melphalan flufenamide) in combination with dexamethasone for the treatment of adult patients with multiple myeloma…The submission is based on the results from the pivotal phase 2 study HORIZON….Following the submission to the FDA Oncopeptides will initiate an Expanded Access Program (EAP) in the U.S. to enable melflufen treatment for patients with a significant unmet medical need.”