Mixed-bag approval hit Sage hard but spared Biogen Sage Therapeutics recently notched an FDA win for the neuroactive steroid zuranolone (Zurzavae) for postpartum depression (PPD). For major depressive disorder (MDD), however, the agency handed the drug a complete response letter. In the wake of the news, Sage’s stock is down 46% to $19.52, while BIIB,…
Epstein-Barr virus: Trigger and driver of multiple sclerosis?
Recent research has indicated a link between Epstein-Barr virus (EBV) and multiple sclerosis (MS), with some researchers going as far as to say that EBV might be a potential trigger of MS. However, it remains unclear whether the virus also drives the progression of the disease. Current treatments focus largely on moderating inflammation. In this…
FDA approves zuranolone for postpartum depression but issues complete response letter for major depressive disorder
FDA’s approval of Zurzuvae for PPD could mark a significant shift in the fight against postpartum depression (PPD). On August 4, the agency gave the green light for the oral medication, although it declined its use for major depressive disorder (MDD), citing insufficient evidence of effectiveness. The former approval represents a milestone as the first ever…
The story behind Biogen’s 1,000 job cuts and R&D refocus
Cambridge-based biotech Biogen has unveiled plans to cut 1,000 jobs, or 11% of its workforce, as it prepares for the launch of its newly approved Alzheimer’s disease drug, Leqembi (lecanemab) that it developed with Eisai. This move follows a pattern of significant layoffs, with the company having cut 885 jobs last year after the troubled…
Phase 3 trial shows donanemab reduces Alzheimer’s symptoms by 35%
According to results from the Phase 3 TRAILBLAZER-ALZ 2 study published in JAMA, the monoclonal antibody donanemab significantly slowed cognitive and functional decline in patients with early symptomatic Alzheimer’s disease by approximately 35% at one year compared to placebo. The trial enrolled 1736 patients across 277 research centers in 8 countries. Another monoclonal antibody, Leqembi…
Apitegromab shows sustained benefits in phase 2 SMA trial
Cambridge, Massachusetts–based biopharma Scholar Rock has been making encouraging progress in the development of apitegromab, a potential new therapy for spinal muscular atrophy (SMA), a genetic disease characterized by progressive muscle weakness and atrophy. Apitegromab, a novel monoclonal antibody, works through a unique mechanism of action — it binds to and inhibits the precursor form…
Leqembi could mark new era in Alzheimer’s treatment progress: An overview of the evolving drug development scene
Today, the FDA granted traditional approved to lecanemab (branded as Leqembi), a monoclonal antibody from Eisai and Biogen for adult patients with Alzheimer’s disease. The agency made the decision on the basis of a confirmatory trial that showed its clinical benefit. The drug, which reduces the formation of amyloid plaques in the brain, is the…
Quantum-inspired power could brighten drug discovery
In the realm of drug discovery, the Harvard-educated chemist Nick Paras, Ph.D. compares the early stages of identifying new compounds to a game of Zelda, in which a player at times must navigate through dark rooms and solve puzzles to progress. Similarly, medicinal chemists and biochemists too must traverse the vast, often obscure landscape of…
The Allen Institute is employing AWS Cloud and machine learning to decode brain mysteries
High-resolution mapping of the human brain involves managing and interpreting a colossal amount of data. Shoaib Mufti, the head of data and technology at the Allen Institute for Brain Science, described the organization’s approach to these challenges in a recent interview. The project uses artificial intelligence to analyze millions of data points from brain imaging…
The Brain Knowledge Platform aims to illumine the brain’s cellular universe
Despite substantial progress in brain research, our understanding of the human brain remains limited. “We really don’t understand the fine circuitry of the brain — even in a relatively simple organism like a mouse,” confessed Ed Lein, a senior investigator at the Allen Institute for Brain Science. But AI in neuroscience research promises to change…
Beyond the trip with non-hallucinogenic psychoplastogens in neuropsychiatry
Interest in ketamine and psilocybin as potential therapies for mood disorders has surged since around 2010. A groundbreaking 2000 study at Yale revealed the powerful antidepressant effects of ketamine, a dissociative anesthetic. Unlike traditional antidepressants which can take weeks or months to have an impact, a single dose of ketamine led to significant improvements in…
PsychoGenics’ SmartCube prompts a reevaluation of CNS drug discovery
In an era of rapid AI progress, the quest to pioneer the first AI-developed drug candidates has led to an increasing number of these drug candidates entering clinical trials. One contender is ulotaront, an antipsychotic drug, that fared well in a phase 3 schizophrenia study published in NEJM in 2020. Sunovion discovered the drug in…
Visualizing the prominence of suspicious medical research publications
A recent exposé in Science magazine concluded that “Fake scientific papers are alarmingly common.” The article quoted a preprint from the German neuropsychologist Bernhard Sabel, which estimated that in 2020, up to one-third of neuroscience papers and nearly a quarter of medicine papers were likely falsified or plagiarized, highlighting a concerning prevalence of suspicious medical…
Natalizumab and PML: The complex dance of benefit and risk for MS
Biogen’s Tysabri (natalizumab), the first humanized monoclonal antibody for multiple sclerosis (MS), sparked optimism among MS patients following its FDA approval in 2004. The drug offered significant benefits, reducing relapses for patients resistant to other treatments. This was a significant milestone in the treatment of MS, but the journey of natalizumab and PML soon took…
Capsida Biotherapeutics to present preliminary findings on engineered AAV capsids at ASGCT annual meeting
The gene therapy platform company Capsida Biotherapeutics (Thousand Oaks, California) is gearing up to unveil initial data on its engineered adeno-associated virus (AAV) capsids at the upcoming American Society of Gene & Cell Therapy (ASGCT) 26th Annual Meeting, held May 16–20 in Los Angeles. In developing these capsids, Capsida Chief Scientific Officer Susan Catalano explained…
Donanemab slows cognitive decline in early Alzheimer’s patients by 35%
Lilly’s (NYSE:LLY) shares were up more than 5.36% to $425.88 after the company announced upbeat results from the phase 3 TRAILBLAZER-ALZ 2 study, which revealed that the experimental Alzheimer’s drug donanemab significantly slowed cognitive decline in patients with early symptomatic Alzheimer’s disease at 18 months. Almost half of patients, 47%, who received donanemab had no…
Janssen exec discusses company’s precision neuroscience strategy and partnerships
In mid-April, San Diego–based Pipeline Therapeutics announced a global license and development agreement with Janssen Pharmaceutica NV (NYSE:JNJ), focusing on PIPE-307, an investigational compound targeting nervous system disorders. As a pioneer in precision neuroscience, Pipeline Therapeutics aims to create targeted treatments for patients. PIPE-307, an oral, highly selective antagonist of the muscarinic M1 receptor, embodies…
Brain breakthroughs: Aprinoia Therapeutics’ harnesses AI and strategic partnerships to propel neurodegenerative disease research
Neurodegenerative disease research is witnessing significant advances. To that end, Hong Kong-headquartered Aprinoia Therapeutics is embracing a ‘precision neuroscience’ approach to neurodegeneration diagnostics. The company’s lead program, APN-1607, represents a new generation of advanced tau positron emission tomography (PET) tracers, which play a crucial role in effective and efficient diagnosis of patients suffering from neurodegenerative…
The 50 best-selling pharmaceuticals of 2022: COVID-19 vaccines poised to take a step back
The COVID-19 pandemic has had a profound impact on the best-selling pharmaceuticals, leading to shifts in the list with Pfizer and BioNTech’s Comirnaty surpassing AbbVie’s Humira for the No. 1 spot in 2021. That momentum continued in 2022, with Pfizer and BioNTech jointly raking in $59.1 billion in revenue from the sales of the COVID-19…
Amylyx Pharmaceuticals launches phase 2 trial for AMX0035 Wolfram syndrome therapy
Cambridge, Massachusetts–based Amylyx has dosed the first participant in its phase 2 HELIOS clinical trial of AMX0035 focusing on Wolfram syndrome. The condition is an ultra-rare genetic disorder involving the endocrine system. Symptoms of Wolfram syndrome can include diabetes insipidus, diabetes mellitus, optic atrophy and hearing loss. In September, Amylyx won FDA approval for AMX0035…
Lonza and ABL Bio partner on bispecific antibody development for immuno-oncology and neurodegenerative diseases
Lonza (LON:0QNO) and South Korea-based ABL Bio (KOSDAQ:298380) have joined forces to accelerate bispecific antibody development for immuno-oncology and neurodegenerative diseases. Over email, Basel, Switzerland–headquartered Lonza noted that its experience with novel molecular formats enables the company to handle ABL Bio’s unique challenges. In the partnership, Lonza will provide submission-ready chemistry, manufacturing and controls (CMC) data…
Accelerating Alzheimer’s research: ADDF’s chief scientific officer reflects on the Lauder Foundation’s $200M gift
The Estée Lauder family has donated $200 million to the Alzheimer’s Drug Discovery Foundation (ADDF), a nonprofit they founded in 1998 to support Alzheimer’s research. The gift is the largest ADDF has received. “We’ve deployed about $270 million so far for about 700 programs in 19 countries of drug discovery and development over the past…
QRL-201, a novel therapy targeting STMN2 expression, enters phase 1 ALS trial
Cambridge, Massachusetts-based QurAlis has dosed the first patient with the biotherapeutic QRL-201 in a Phase 1 clinical trial (ANQUR) in Canada. QRL-201 is a novel therapy targeting the restoration of STATHMIN-2 (STMN2) expression in amyotrophic lateral sclerosis (ALS) patients. QurAlis believes ANQUR the first study to evaluate such a treatment. Stathmin-2 (STMN2), also known as…
QurAlis targets UNC13A RNA mis-splicing in ALS and FTD with novel FlexASO platform
QurAlis, a privately-held company specializing in precision medicines for amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, has debuted its latest program. This initiative targets UNC13A RNA mis-splicing, a critical gene alteration present in ALS and frontotemporal dementia (FTD). FTD has recently gained renewed attention following the disclosure of Bruce Willis’ diagnosis of the condition.…
QurAlis to tap $88M funding to develop ALS product candidates QRL-201 and QRL-101
QurAlis, a clinical-stage biotech focused on amyotrophic lateral sclerosis (ALS) and other neurodegenerative diseases, announced that it has closed an oversubscribed $88 million Series B financing. According to Kasper Roet, CEO and co-founder of QurAlis, the funding round recognizes the company’s scientific track record. “We are breaking down scientific barriers in our quest to bring…