4 Biotechs Show Off Pipeline Advancements: Dante, Arrowhead, Forma & Nurix

2022-05-26

疫苗细胞疗法合作siRNA信使RNA

Four biotech companies are holding Research-and-Development Day events on Thursday to present and discuss their pipelines. Here’s a look. Dante Genomics Dante Genomics reported progress within its drug discovery pipeline. It currently has six compounds in its pipeline based on its internal siRNA and mRNA technology for both rare and common diseases. Two of the four are under development in partnership with Protelica, one in respiratory disease and the other in ovarian cancer. Its internal programs are in a rare muscle disease, a rare neurological disease, a COVID-19 mRNA vaccine and an mRNA oncology therapeutic. “Personalized medicine needs personalized data, and our progress in the last 18 months alone demonstrates the value of genomic data to research and discovery drug development,” said Andrea Riposati, CEO of Dante. “When we founded Dante Genomics, we felt we had a responsibility to deliver personalized medicine beyond diagnosis so that no patient would be facing a diagnosis with no effective treatments. It is this holistic approach that challenges and motivates us as a company to accelerate science to save more lives.” Arrowhead Pharmaceuticals Arrowhead Pharmaceuticals described its emerging pipeline of RNA interference (RNAi) therapies for pulmonary disease. The company’s technology platform is a Targeted RNAi Molecule (TRIM). At its R&D Day in New York City, the company presented its pipeline candidates, including ARO-MUC5AC, engineered to decrease expression of mucin 5AC (MUC5AC) a possible treatment for various muco-obstructive pulmonary diseases; ARO-RAGE, created to decrease expression of the receptor for advanced glycation end products (RAGE) as a treatment for obstructive inflammatory pulmonary diseases; and ARO-MMP7, its newest and previously undisclosed pipeline compound designed to decrease expression of matrix metalloproteinase 7 (MMP7) as a possible treatment for idiopathic pulmonary fibrosis (IPF). Chris Anzalone, Ph.D., president and chief executive officer of Arrowhead said, “These are each potentially important new investigational medicines that seek to treat muco-obstructive and inflammatory lung diseases and IPF in fundamentally new ways. The pulmonary targeted TRIM™ platform has come a long way since our first clinical candidate, ARO-ENaC. We have now improved siRNA triggers with longer pharmacodynamic duration allowing less frequent dose administration and a lower cumulative exposure in planned tox studies, thus less likely to overload lung clearance mechanisms. This gives us increased confidence as we move forward with upcoming clinical studies and additional toxicology studies.” Forma Therapeutics Holdings Forma Therapeutics Holdings is hosting a virtual R&D Day to discuss its pipeline. This includes its oral PKR activator, etavopivat, which is involved in a range of development programs, including ongoing enrollment in the Phase II/III Hibiscus Study in sickle cell disease (SCD), thalassemia and myelodysplastic syndromes (MDS); a Phase II trial in SCD, and thalassemia; a Phase II trial in lower-risk MDS, and others. The presentation also includes its oral CBP/p300 inhibitor FT-7051, which is being studied in its Phase I trial for metastatic castration-resistant prostate cancer. Its pipeline also includes its USP1 program (FT-3171), targeting a novel DNA damage repair pathway in multiple tumor types. David Cook, Forma’s chief scientific officer, said, “We are also capitalizing on our knowledge of the emerging science of red blood cell health by exploring areas beyond hematologic disease.” Nurix Therapeutics Nurix Therapeutics reported it has initiated the first of several potential Phase Ib expansion cohorts in its ongoing Phase I trial of NX-2127. The drug is an oral BTK degrader with immunomodulatory activity. The first expansion cohorts will target chronic lymphocytic leukemia (CLL). It is also hosting an R&D Day for investors on Thursday. Earlier this month, the company dosed the first patient in its Phase Ia/Ib trial of NX-5948, a BTK degrader in patients with relapsed B-cell malignancies. In addition to those two drugs, it has NX-1607, a CBL-B inhibitor CBL-B inhibitor for immuno-oncology indications and DeTIL-0255, a cell therapy being developed for gynecologic malignancies. It also has a discovery pipeline, some that are wholly owned, and some partnered with Gilead Sciences and Sanofi. “Our decision to advance NX-2127 in patients with CLL is based on the promising efficacy, safety, pharmacokinetic, and pharmacodynamic data from the ongoing Phase Ia dose-escalation trial,” said Dr. Robert J. Brown, M.D., Nurix’s executive vice president of clinical development. “There is a significant unmet need for a therapeutic approach with the potential to address the growing problem of relapse due to the development of BTK inhibitor BTK inhibitor resistance. We aim to meet that need and are encouraged by the emerging data demonstrating the potential of BTK degradation to treat acquired resistance mutations for both standard of care and newly developed BTK inhibitors BTK inhibitors.”