ISIS-814907

Today, a brief rundown of news from Vertex Pharmaceuticals and Merck & Co. as well as updates from Fibrogen, Intellia Therapeutics and Biogen that you may have missed.The Food and Drug Administration has agreed to a speedy review of Vertex Pharmaceuticals closely watched pain tablet. An approval decision should come by Jan. 30. The tablet works differently than available pain medications, instead targeting a kind of microscopic cell tunnel that controls nerve cell communication. Wall Street analysts see Vertexs drug as a valuable, non-opioid option for pain management, and expect annual sales will eventually crest $5 billion. Jacob BellMerck & Co.s prized immunotherapy Keytruda has hit a new milestone. In the companys latest earnings report, quarterly sales of the cancer drug topped $7 billion. The three-month period also saw Keytruda notch its 40th approved indication in the U.S. Jacob BellKeytruda quarterly sales surpass $7 billion for the first timeAs reported by Merck & Co.!function(){“use strict“;window.addEventListener(“message“,(function(a){if(void 0!==a.data[“datawrapper-height“]){var e=document.querySelectorAll(“iframe“);for(var t in a.data[“datawrapper-height“])for(var r=0;r<e.length;r++)if(e[r].contentWindow===a.source){var i=a.data[“datawrapper-height“][t]+“px“;e[r].style.height=i}}}))}(); FibroGen announced Tuesday it is reducing its staff in the U.S. by 75% after disappointing results from a Phase 2/3 trial of its drug pamrevlumab for pancreatic cancer. It's the latest restructuring for the San Francisco biotechnology company, which has faced a series of trial failures and regulatory rejections in recent years. Shares of FibroGen plunged by half in Wednesday morning trading. Gwendolyn WuGene editing specialist Intellia Therapeutics has received clearance from the U.K.'s Medicine and Healthcare products Regulatory Agency to start a Phase 1/2 clinical trial of its treatment NTLA-3001 for alpha-1 antitrypsin deficiency. NTLA-3001 is administered in vivo to insert a healthy copy of the gene that encodes the AAT protein. It would be the third medicine from Intellia to enter clinical trials, following programs for transthyretin amyloidosis and hereditary angioedema. Gwendolyn WuBiogen is teaming up with two diagnostics developers to identify biological markers for Alzheimers disease. Specifically, the collaboration aims to first find and then create tests for blood-based markers that reflect the presence of diseased tau in the brain. Tau is a protein that, while important to brain health, can sometimes form toxic clumps which damage neurons. Early this year, Biogen said it plans to accelerate the development of an experimental, tau-targeting therapy called BIIB080, hoping to add another asset to its portfolio of Alzheimers medicines. Jacob Bell '

Biogen and Ionis Pharmaceuticals are stopping development of an experimental drug for ALS following new clinical trial results that didnt meet the companies expectations.The trial, which enrolled almost 100 people with the nerve-destroying disorder, found the drug actually worked as intended. Biogen and Ionis had designed it to lower levels of ataxin-2, a protein tied to amyotrophic lateral sclerosis, and on that measure it was successful. High-level results released Thursday showed participants who received the drug, named BIIB105, rather than a placebo experienced significant decreases in the protein over a six-month treatment period.However, the drug didnt appear to change the trajectory of the disease. It didnt impact breathing, strength and overall function key health measures that diminish as ALS progresses. BIIB105 also didnt substantially reduce a different protein, neurofilament light chain, that recently became important across this area of research. Qalsody, another ALS medicine developed by Biogen and Ionis, won marketing approval last year based on its ability to lower that protein.“While BIIB105 lowered ATXN2 protein, it did not reduce neurofilament, which gives us confidence that BIIB105 did not slow the disease process,“ said Stephanie Fradette, head of Biogens Neuromuscular Development Unit, in a statement.In the trial, participants who completed the main six-month treatment period were then offered entry into an open-label extension phase, in which they all could receive BIIB105. Biogen and Ionis said data from that phase were similar what was seen earlier in the study, with sustained reductions in the ataxin-2 protein but no impact on neurofilament or clinical outcome measures.The companies said there was no evidence that any subgroup of drug-treated patients benefited clinically.Analysis of the data remains ongoing. Biogen and Ionis plan to present more detailed study results next month at an ALS research convention in Stockholm.The shelving of BIIB105 is the latest setback for a field of drug development marred by failure. ALS, like many central nervous system disorders, is complex and difficult to combat. Scientists first discovered the disease over a century ago; and yet, just a few medicines have ever been approved to treat it. (The list got even smaller this year, as a once-promising medication from Amylyx Pharmaceuticals is being pulled from the market.)Approved in April 2023, Qalsody is the newest offering, though its for a very small fraction of ALS patients with specific genetic mutations. Biogen and Ionis collaborated on early development of the therapy, using the latters antisense oligonucleotide drugmaking technology.Qalsody essentially gums up the production of an ALS-associated protein called SOD1. The therapy actually failed to hit the central goal of the study that led the Food and Drug Administration to clear it. In that trial, Qalsody wasnt any better than a placebo at slowing disease progression. It was, however, better at lowering neurofilament light chain a biological marker of neurological damage.The FDAs decision effectively created a new route to approval for ALS drugmakers, who, since the Qalsody decision, have prioritized showing an effect on neurofilament in their clinical tests.So far, Qalsody is the only clear victory to come out of Biogen and Ionis ALS research. In 2022, the partners discontinued another antisense drug, code-named BIIB078, after an early-stage trial disappointed. Also on Thursday, Ionis revealed that Biogen has opted against licensing a drug the companies have been working on for Angelman syndrome, despite results Ionis described as positive.The longstanding partnership has been fruitful in other areas, though. Its produced a blockbuster medicine in Spinraza, a treatment for a rare, muscle-wasting illness. And over the past couple years, Biogen has highlighted the potential of another experimental drug, BIIB080, which is in mid-stage testing as a possible treatment for Alzheimers disease. '