valproic acid/prednisone (VAL001) / Respiratorius - LARVOL DELTA

Respiratorius has conducted a pre-IND meeting with the FDA (Cision) - "The purpose of the meeting was to discuss strategic considerations ahead of the upcoming clinical development of the drug candidate VAL001, intended for the treatment of previously untreated diffuse large B-cell lymphoma (DLBCL)....The meeting with the FDA primarily focused on two key areas: the study design and the implementation of the phase III trial, with the aim of obtaining marketing authorisation in the United States. The FDA recommended that supplementary data for dose levels lower than the maximum tolerated dose – established in previous phase I/IIa studies – should be incorporated into the phase III study design."

FDA event • Diffuse Large B Cell Lymphoma

Respiratorius Granted Pre-IND Meeting with FDA for VAL001 (Cision) - "Respiratorius AB (publ) today announces that the company has been granted a pre-IND meeting with the U.S. Food and Drug Administration (FDA) ahead of a planned Investigational New Drug (IND) application for its drug candidate VAL001. The purpose of the meeting is to obtain regulatory guidance for the next steps in the clinical development of VAL001 in the strategically important U.S. market. The granted meeting request reflects interest from multiple stakeholders and forms a key part of the company's ongoing efforts to strengthen the positioning of the candidate."

FDA event • Diffuse Large B Cell Lymphoma

Notice of Allowance for European Patent on VAL001 Formulation (Cision) - "Respiratorius AB...announces that the European Patent Office (EPO) has issued a formal Notice of Allowance for its patent application covering the novel oral formulation of sodium valproate that supports VAL001. This milestone marks Respiratorius’ first granted formulation patent, protecting a unique method for administering valproate as a pre‑treatment in oncology. The patent carries a priority date of April 21, 2016, and once formally granted, will confer market exclusivity for at least 20 years from that date. Corresponding patents have already been approved in Japan, Canada and the United States."

Patent • Hematological Malignancies • Oncology

Combined Immediate-Release and Extended-Release Formulation of Sodium Valproate Provides Stable Plasma Levels for Inhibition of Histone Deacetylation. (PubMed, Clin Pharmacol Drug Dev) - "A modified controlled-release sodium valproate formulation (VAL001, test) was compared with an approved enteric-coated tablet formulation (Absenor, reference). No serious or severe adverse events were observed. The novel capsule formulation of valproate, containing a combination of immediate-release granules and extended-release pellets, appears to have suitable pharmacokinetic properties for cancer treatments aiming for histone deacetylase inhibition."

Journal • B Cell Lymphoma • Diffuse Large B Cell Lymphoma • Hematological Malignancies • Lymphoma • Non-Hodgkin’s Lymphoma • Oncology

"BioStock: Sista personen doserad i Respiratorius PK-studie med VAL001 https://t.co/QZ3bokV4Dx" (@CisionNews)

"BioStock: Respiratorius begins production of VAL001 https://t.co/2VeJkx6cjz" (@CisionNews)

"BioStock: Respiratorius inleder produktion av VAL001 https://t.co/bNtg0TNtH9" (@CisionNews)

"BioStock: Respiratorius gets patent approval in Brazil for VAL001 https://t.co/Pkmclrr4SO" (@CisionNews)

Monoclonal Antibodies Prove to Be Novel Therapeutics for Myeloma (OncLive) - "The therapeutic armamentarium has rapidly expanded in recent years because of the approval of novel drugs such as carfilzomib (Kyprolis), ixazomib (Ninlaro), pomalidomide, daratumumab (Darzalex), and selinexor (Xpovio). Others, such as melflufen, likely will come to market in the near future. These drugs have especially improved the treatment options for relapsed disease, with some agents now also being used as frontline therapy. As a result, outcomes for patients with newly diagnosed and relapsed disease continue to improve with prolongation of median survival to greater than 7 years from diagnosis."

Media quote

Early diagnosis improving the outcome of an infant with epileptic encephalopathy with cytoplasmic FMRP interacting protein 2 mutation: Case report and literature review. (PubMed, Medicine (Baltimore)) - "OUR: case confirmed that CYFIP2 was the pathogenic gene of EIEE 65. We also first demonstrated vigabatrin might be effective for control of seizures and helpful for the improved outcomes of these patients."

Clinical • Journal • Review