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REYKJAVIK, Iceland, Oct. 29, 2024 /PRNewswire/ --Scientists at deCODE genetics/Amgen, and their collaborators have discovered six novel genes with rare germline variants that associate with cancer risk. The findings are published today in Nature Genetics under the title "Gene-based burden tests of rare germline variants identify six cancer susceptibility genes". Continue Reading Kari Stefansson senior author of the paper, “Gene-based burden tests of rare germline variants identify six cancer susceptibility genes”, published today in Nature Genetics, discusses the paper with Erna V. Ivarsdottir first author on the paper. Kari Stefansson CEO of deCODE genetics and senior author on the paper in Nature Genetics, with Erna V. Ivarsdottir scientists at deCODE genetics and first author. A subset of cancers arises in individuals who are born with rare sequence variants that significantly alter their cancer risk. The discovery of such variants, like those in the BRCA1- and BRCA2 genes, has led to improved early cancer detection and the development of targeted therapies, ultimately reducing the cancer burden and improving prognosis of those carrying these mutations. In this study, the scientists analysed three large genetic datasets from individuals of European descent, including 130,991 cancer patients and 733,486 controls. Through a gene-based burden association analysis across 22 different cancer types, they found four novel genes associated with a risk of developing cancer; the pro-apoptotic BIK for prostate cancer, the autophagy involved ATG12 for colorectal cancer, TG for thyroid cancer, and CMTR2 for both lung cancer and cutaneous melanoma. The relative increase in cancer risk conferred by these variants was substantial (90-295%), but it should be noted that the design of the study does not allow accurate assessment of absolute lifetime cancer risk. Additionally, the researchers found the first genes with rare variants that are associated with a decreased risk of cancer. Specifically, loss of AURKB was found to protect against any cancer type, and loss of PPP1R15A was associated with 53% lower risk of breast cancer. This suggests that inhibition of PPP1R15A may be a therapeutic option for breast cancer. The study revealed new insight into the biological mechanisms involved in cancer predisposition that will hopefully lead to better screening and treatment strategies. Video - Photo - SOURCE deCODE genetics WANT YOUR COMPANY'S NEWS FEATURED ON PRNEWSWIRE.COM? 440k+ Newsrooms & Influencers 9k+ Digital Media Outlets 270k+ Journalists Opted In GET STARTED

Arrowhead Pharmaceuticals said it is refining its cardiometabolic pipeline, choosing to focus its resources on the development of plozasiran, while seeking a partner for zodasiran. The decision comes as the company prepares to advance plozasiran into a Phase III cardiovascular outcomes trial.Plozasiran, an RNAi therapeutic designed to reduce the production of apolipoprotein-CIII (ApoC3), has shown promising results in studies across three distinct patient populations: familial chylomicronaemia syndrome (FCS), severe hypertriglyceridaemia and mixed hyperlipidaemia.The company recently announced positive top-line data from the Phase III PALISADE study, which evaluated plozasiran in patients with FCS. The trial met the primary endpoint of lowering triglycerides (TG) and met all key secondary goals, including reducing the incidence of acute pancreatitis, compared to placebo.Durable triglyceride reductionsArrowhead shared additional data from PALISADE at its R&D day this week. Building on results showing placebo-adjusted median TG reductions of -80% and -78% at month 10 with quarterly doses of 25 mg and 50 mg, respectively, Arrowhead presented curves showing quick and lasting reductions in TG compared to baseline. "Plozasiran, administered once every three months, consistently maintained the median and mean triglyceride levels over the study period with low variability," said chief medical scientist Bruce Given.The company also provided detailed safety data indicating that the incidence of treatment-emergent adverse events (AEs) was comparable between the placebo and active groups, while the rate of serious and severe AEs was slightly higher among placebo patients."We view the incremental update as encouraging," commented analysts at Morgan Stanley. The company will present the full dataset at a future scientific conference.Meanwhile, Arrowhead is preparing to start a Phase III cardiovascular outcomes trial, dubbed CAPITAN, to further evaluate plozasiran in a broader patient population. The study will enroll patients with mixed hyperlipidaemia and residual risk of atherosclerotic cardiovascular disease.Resource considerationsAs for zodasiran, which targets ANGPTL3, the company indicated that while the data generated so far has been "highly encouraging," and warrants further investigation in Phase III, future development will require a partner."Based on our various assessments for both candidates, and resource allocation considerations, we have elected to broadly advance plozasiran ourselves and, at this time, will only conduct further development of zodasiran if we identify a suitable development and commercialisation partner," Given said.

TG Therapeutics says it’s holding on to an option to buy back all rights handed over to Neuraxpharm for a period of two years “in the event of a change in control of TG.” Eight months after Briumvi’s initial green light in the U.S., TG Therapeutics is preparing to debut its multiple sclerosis antibody on the world stage. In a deal unveiled Tuesday, European specialty pharmaceutical company Neuraxpharm will throw TG an upfront payment of $140 million—plus an additional $12.5 million upon Briumvi’s first EU launch—to chip in on commercialization of TG’s CD20-targeting drug outside the U.S. TG is eligible to receive another $492.5 million in launch and commercialization milestones, for a total deal value of $645 million, the companies said in a release. Aside from milestones, TG is in line to receive tiered double-digit percentage royalties based on net product sales, with the royalties capping out at 30%. In exchange, Neuraxpharm is getting its hands on exclusive rights to market Briumvi in areas outside the U.S., Canada and Mexico, which are retained by TG, plus some previously partnered Asian countries. TG also says it’s holding on to an option to buy back all rights handed over to Neuraxpharm for a period of two years “in the event of a change in control of TG.” Briumvi scored in its initial approval in the U.S. back in December. Cleared in relapsing multiple sclerosis, TG’s drug faced an uphill climb against entrenched competitors such as Roche’s Ocrevus, Novartis’ Kesimpta and Biogen’s Tecfidera. But unlike Novartis’ anti-CD20 treatment Kesimpta, which is injected once a month, Briumvi is designed as a one-hour infusion post-starting dose, after which patients need only receive a 450-mg dose administered over one hour every six months. Roche’s Ocrevus is also administered every 24 weeks (PDF) after a series of starting doses. Briumvi won its European green light at the start of June, clearing the way for successive launches across the bloc. The company is also pursuing nods in the U.K. and Switzerland. Neuraxpharm, for its part, figures Briumvi is an “ideal product” to add to its portfolio, according to the group’s CEO Jörg-Thomas Dierks, M.D. As part of the deal, Neuraxpharm is fitting another 100 new multiple sclerosis specialists into its “already deep CNS commercial team,” the company added. For the second quarter of 2023, Briumvi brought home U.S. sales of $16 million, representing 100% quarter-over-quarter growth. According to TG, more than 340 healthcare providers at 225-plus centers across the U.S. have written more than 1,200 Briumvi prescriptions since launch.