New Alzheimer’s Drug Slows Cognitive Decline by 27%

A new Alzheimer’s drug from Japanese pharmaceutical company Eisai and US drugmaker Biogen has shown promising results in a large-scale clinical trial. The companies announced the trial’s success in a press release, saying their drug — called lecanemab — was observed to have slowed cognitive decline in Alzheimer’s patients by 27% over 18 months.

The companies said 1,795 patients with early-stage Alzheimer’s were randomly selected to receive a placebo treatment or doses of lecanemab every two weeks. Their cognitive decline was then measured on six fronts, including “memory, orientation, judgment and problem solving, community affairs, home and hobbies, and personal care.” According to the statement, lecanemab significantlyreduced clinical decline” over the 18-month timeframe.

Lecanemab, per Eisai, is a monoclonal antibody treatment, which targets toxic amyloid plaques protein clumps that researchers proposed were the cause of the neurodegeneration seen in Alzheimer’s.

The companies noted that around 21% of the patients who received the lecanemab treatment experienced brain swelling that was visible on PET scans.

Today’s announcement gives patients and their families hope that lecanemab, if approved, can potentially slow the progression of Alzheimer’s disease, and provide a clinically meaningful impact on cognition and function,” said Michel Vounatsos, Biogen‘s chief executive officer in the companies’ joint press release.

Eisai’s chief executive Haruo Naito said in the company’s press release that the lecanemab study’s success was “an important milestone for Eisai in fulfilling our mission to meet the expectations of the Alzheimer’s disease community.”

Source: https://investors.biogen.com/
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F.D.A. Approves Alzheimer’s Drug

Aducanumab, or Aduhelm, is the first new Alzheimer’s treatment in 18 years and the first to attack the disease process. But some experts say there’s not enough evidence it can address cognitive symptomsThe Food and Drug Administration  approved the first new medication for Alzheimer’s disease in nearly two decades, a contentious decision made despite opposition from the agency’s independent advisory committee and some Alzheimer’s experts who said there was not enough evidence that the drug can help patients.

The drug, aducanumab, which will go by the brand name Aduhelm, is a monthly intravenous infusion intended to slow cognitive decline in people with mild memory and thinking problems. It is the first approved treatment to attack the disease process of Alzheimer’s instead of just addressing dementia symptoms. Biogen, its manufacturer, announced that the list price would be $56,000 a year. In addition, there will most likely be tens of thousands of dollars in costs for diagnostic testing and brain imaging. Recognizing that clinical trials of the drug had provided incomplete evidence to demonstrate effectiveness, the F.D.A. granted approval for the drug to be used but required Biogen to conduct a new clinical trial. If the new trial, called a Phase 4 trial, fails to show the drug is effective, the F.D.A. can — but is not required to — rescind its approval.

About six million people in the United States and roughly 30 million globally have Alzheimer’s, a number expected to double by 2050. Currently, five medications approved in the United States can delay cognitive decline for several months in various Alzheimer’s stages. Patient advocacy groups had lobbied vigorously for approval because there are so few treatments available for the debilitating condition. Some other drugs in clinical trials are more promising, but they are most likely three or four years away from potential approval.

The F.D.A. advisory committee, along with an independent think tank and several prominent experts — including some Alzheimer’s doctors who worked on the aducanumab clinical trials — said the evidence raised significant doubts about whether the drug is effective. They also said that even if it could slow cognitive decline in some patients, the benefit suggested by the evidence would be so slight that it would not outweigh the risk of swelling or bleeding in the brain that the drug caused in the trials.

The data included in the applicant’s submission were highly complex and left residual uncertainties regarding clinical benefit,” the F.D.A.’s director of the Center for Drug Evaluation and Research, Dr. Patrizia Cavazzoni, wrote on the agency’s website. But, she said, the agency had decided to approve the drug through a program called accelerated approval, which is designed “to provide earlier access to potentially valuable therapies for patients with serious diseases where there is an unmet need, and where there is an expectation of clinical benefit despite some residual uncertainty regarding that benefit.” Michel Vounatsos, Biogen’s chief executive, hailed the approval and said in a statement, “We are committed to sharing our future insights about Aduhelm with the scientific community as we collect more data from the real-world use of this treatment.

Source: https://www.nytimes.com/

Scribe Therapeutics change the genes responsible for causing diseases

Imagine being able to change the genes responsible for causing diseases. For Scribe Therapeutics, a gene-editing company that develops genetic medicines, this is no longer a dream but a reality. Scribe Therapeutics is one of several companies approaching genetic medicines through Crispr, the now-famous “molecular scissors” employed to cut and edit DNA. But the company is taking a new approach to leveraging Crispr technology. Instead of relying on wild-type or naturally occurring Crispr molecules such as Cas9, Scribe Therapeutics have built their own, highly-specialized varieties.

Founded by Jennifer Doudna, Benjamin Oakes, Brett Staahl, and David Savage, Scribe Therapeutics is creating an advanced platform for Crispr-based genetic medicine.

Crispr is changing how we think about treating diseases,” says co-founder, President, and CEO of Scribe Therapeutics, Benjamin Oakes. “When I finished my undergraduate degree, I shadowed doctors and realized we had no way to treat the underlying causes of diseases. This changed my career path to creating Crispr-based tools that can actually treat the underlying causes.”

Scribe Therapeutics has collaborated with Biogen to create Crispr-based genetic medicines for diseases such as amyotrophic lateral sclerosis (ALS). The company is also studying how to use adeno-associated virus (AAV) vectors to deliver Crispr components to the nervous system, eyes, and muscles. AAV vectors can deliver DNA to specific target cells for therapeutic uses.

Today, Scribe Therapeutics announced a $100 million Series B funding round that will help the company grow and expand. One of the key ways it stands out from other synthetic biology and gene-editing companies is through its approach to doing science. Other companies sometimes create tools without thinking about the problems they can solve, but Scribe Therapeutics is different. Instead of building technology in need of a solution, Scribe Therapeutics finds the problem first and creates the technology to fix it.

We face challenges head-on and continue to inspire people to try the hard things. You have to encourage fearlessness in science. If your experiment failed today, it doesn’t mean you’re a failure. You have to keep trying,” says Oakes.

Scribe Therapeutics‘ “Crispr by designplatform has custom-engineered millions of novel molecules specifically designed for therapeutic uses within the human body. For example, its X-editing (XE) technology is an engineered molecule that offers greater specificity, activity, and deliverability when used therapeutically.

Source: https://www.forbes.com/

Biogen Nabs Speedy FDA Review For Alzheimer’s Drug

Biogen’s aducanumab is inching closer to an FDA decision. The Big Biotech, along with partner Eisai, announced  that the FDA accepted its regulatory submission for aducanumab, its once-failed Alzheimer’s drug—with priority review to boot. The agency expects to decide the fate of the treatment by March 7. Along the way, it will hold an advisory committee meeting. It has not set a date for the meeting, but Jefferies analyst Michael Yee expects it sometime in the first quarter of 2021.

The FDA’s acceptance of the aducanumab BLA with Priority Review is an important step in the path to potentially having a treatment that meaningfully changes the course of Alzheimer’s disease,” said Michel Vounatsos, Chief Executive Officer at Biogen.

How the FDA rules on aducanumab will show how far the FDA and its commissioner, Stephen Hahn, M.D., are willing to diverge from its established approval standards. Under U.S. law, companies need to show “substantial evidence of effectiveness to win approval.

Source: https://investors.biogen.com/

https://www.fiercebiotech.com/

Breakthrough In The Fight Against Alzheimer’s

In a shocking reversal, Biogen (BIIB) said that it would resurrect an Alzheimer’s drug that the company previously said had failed and will ask the Food and Drug Administration (FDA) to approve it. The company said a “new analysis of a larger dataset” showed that the drug, aducanumab, reduced clinical decline in patients with early Alzheimer’s disease on multiple measures of the drug’s effectiveness. That directly contradicts a decision in March to halt studies of the therapy based on the recommendations of an independent monitoring board that was charged with protecting patients in the study. Aducanumab’s failure sent shock waves far beyond Biogen. It was thought to be the last of a series of drugs—the previous ones, from many different drug companies, all failed—that targeted a protein in the brain called beta amyloid. After Biogen’s announcement in March, most researchers and biotechnology executives saw little hope for a drug that would help patients with Alzheimer’s disease even as cases mount.

Biogen said that it conducted a new analysis in consultation with the FDA of a larger data set from the discontinued studies. The new analysis includes additional data that became available after the previous analysis showed the studies were “futile”—that it had no chance of succeeding. Biogen said that the new data show aducanumab is “pharmacologically and clinically active” and that it reduced patients’ clinical decline based on the results of a survey called Clinical Dementia Rating-Sum of Boxes (CDR-SB), which was the main goal of both studies.

With such a devastating disease that affects tens of millions worldwide, today’s announcement is truly heartening in the fight against Alzheimer’s. This is the result of groundbreaking research and is a testament to Biogen’s steadfast determination to follow the science and do the right thing for patients,” Michel Vounatsos, Biogen’s chief executive, said in a statement. “We are hopeful about the prospect of offering patients the first therapy to reduce the clinical decline of Alzheimer’s disease and the potential implication of these results for similar approaches targeting amyloid beta.”

Al Sandrock, Biogen’s head of research and development and chief medical officer, said in his first interview about the new results that his team could only find one previous instance where a trial was stopped for futility and then it turned out to be positive. “I have to pinch myself because I almost don’t believe it yet,” Sandrock said. “It’s so amazing to have this change from March. But I’m also very, very happy because… I know people with mild cognitive impairment and I felt like I had let them all down.

By June, as Biogen analyzed the full data set, researchers started to realize that a different picture was emerging of aducanumab, Sandrock said. The reason was because of changes that Biogen had made to the study late in the game. Initially, the company worried about a potential side effectbrain swelling—and limited the dosage of the drug. But later patients were allowed to receive higher doses of the medicine.

Source: https://www.biogen.com/
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