Pramana Pharmaceuticals Inc. has been awarded $1 million from the Alzheimer’s Association to support IND-enabling development of PRM-914, the company’s once-daily oral small-molecule therapeutic for Alzheimer’s disease. The funding will support final toxicology and translational studies required to advance PRM-914 into first-in-human evaluation.
Sarepta Therapeutics Inc. has filed a clinical trial application (CTA) in New Zealand seeking clearance to initiate a first-in-human trial of SRP-1005 (formerly ARO-HTT). Pending approval, the INSIGHTT trial is anticipated to begin in the second quarter of this year.
Kallyope Inc. has outlined plans to initiate phase I studies this year with K-554, a non-incretin peptide candidate offering a new mechanism of action in obesity.
Glioblastoma (GBM) is the most common and highly aggressive primary brain tumor in adults. MicroRNAs (miRNAs) are pleiotropic post-transcriptional regulators of oncogenic pathways, and frequently lose their tumor-suppressive function in GBM. Researchers from the Istituto Italiano di Tecnologia previously identified a group of 11 pro-neurogenic miRNAs that supports adult neurogenesis by jointly regulating multiple targets in mouse neural stem cells. Writing in Molecular Therapy Nucleic Acids, they present a study where they examined the expression of these 11 miRNAs across glioma grades and GBM subtypes in patients.
Ottimo Pharma Ltd. has obtained IND clearance from the FDA and advanced OTP-01 (jankistomig), an anti-PD-1/VEGFR2 antibody, into phase I for solid tumors. The first patient has been dosed and the study is open at sites in the U.S. and Australia.
Arbele Pte Ltd.’s ARB-1002 has been awarded orphan drug designation by the FDA for the treatment of pancreatic cancer. ARB-1002 is an antibody-drug conjugate comprising an anti-CDH17 monoclonal antibody chemically linked to a potent cytotoxic agent.
Mediar Therapeutics Inc. has announced an oversubscribed $76 million series B financing to support its novel anti-fibrotics through clinical studies. The funding will also be used to advance MTX-439 into phase I for the treatment of chronic kidney disease (CKD)-associated fibrosis.
Cystic fibrosis (CF) is an autosomal recessive genetic disorder caused by mutations in the CF transmembrane conductance regulator (CFTR) gene. Researchers from Sorbonne University and Hôpital Trousseau developed a targeted approach based on antisense oligonucleotides as a way to improve CF management.
The concept of the 3 Rs – reducing, refining and replacing animal research – has been championed since the 1950s, when William Russel and Rex Burch argued in their book “The Principles of Humane Experimental Technique” that the 3 Rs could simultaneously improve the treatment of research animals and advance the quality of scientific and medical research and testing. Current standard practices of animal research undeniably cause animal suffering at the same time that they have prioritized replicability over translatability.
Securing a second major partnership with Sanofi SA – this time worth a potential $2.56 billion – Earendil Labs will provide its AI-driven discovery platform to find new therapeutics for autoimmune and inflammatory diseases.
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