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…
Reata sets $370,000 annual cost for new nerve disorder drug
The Plano, Texas-based Reata Pharmaceuticals has won FDA approval for Skyclarys (omaveloxolone), the first therapy specifically indicated to treat Friedreich’s ataxia. The drug is approved for use in adults and adolescents aged 16 years and older. The approval also comes with a rare pediatric disease priority review voucher. Friedreich’s ataxia is a genetic disease that…
CMS continues to restrict coverage of amyloid-targeting antibodies for Alzheimer’s
CMS has denied the Alzheimer’s Association’s request asking for broader reimbursement support for FDA-approved monoclonal antibodies targeting amyloid. In April 2022, CMS decided to limit coverage of such antibodies for Alzheimer’s to clinical trial volunteers. At that time, aducanumab from Biogen (Nasdaq: BIIB) and Eisai (TYO:4523) was the only FDA-approved antibody for the neurodegenerative condition. CMS…
Canadian company to explore psychedelic as a traumatic brain injury treatment
Algernon Pharmaceuticals (CNSX:AGN) is one of the few companies aiming to develop the classic psychedelic N,N-Dimethyltryptamine (DMT). Most recently, the company’s Algernon NeuroScience announced its plans to explore DMT as a treatment for traumatic brain injury (TBI). The company plans on launching a Phase 2 study in the fourth quarter of 2023 to test its…
Phase 3 study shows zavegepant can relieve migraine pain in 15 minutes
The Lancet has published Phase 3 study results for zavegepant, an investigational nasal spray for the acute treatment of migraine. The study found that a single 10 mg intranasal dose of zavegepant was more effective than placebo for pain relief and freedom from the most bothersome symptom at two hours post-dose. Zavegepant is a small molecule…
Neuron23 launches clinical trial for NEU-723 in Parkinson’s with companion diagnostic in development
South San Francisco–based Neuron23 has started the first-in-human Phase 1 trial of NEU-723 for the treatment of Parkinson’s disease. NEU-723 is a highly potent and selective leucine-rich repeat kinase 2 (LRRK2) inhibitor. This clinical trial will evaluate the safety, tolerability and pharmacokinetics of NEU-723 in healthy volunteers. A targeted approach The company has also collaborated…
Athira Pharma calls for a fresh approach to treating Alzheimer’s disease
The clinical-stage biopharma Athira Pharma (Nasdaq:ATHA) has published a review paper stressing the need for new treatments for Alzheimer’s disease. The paper also states that one of its small molecule drugs, fosgonimeton, holds potential for treating Alzheimer’s. The Journal of Alzheimer’s Disease published the paper titled “The Case for a Novel Therapeutic Approach to Dementia:…
FDA hands Lilly’s Alzheimer’s drug donanemab complete response letter
Last year, Lilly’s (NYSE:LLY) Alzheimer’s monoclonal antibody donanemab appeared to be a superior therapy to aducanumab from Biogen (Nasdaq:BIIB) and Eisai (OTCMKTS:ESALY), the first such antibody to win FDA approval. But facing criticism over its decision to grant accelerated approval to aducanumab, FDA has sent Lilly a complete response letter for the accelerated approval submission of…
QurAlis homes in on drug candidates for subset of ALS patients
The Cambridge, Massachusetts–headquartered biotech QurAlis kicked the year off by launching a Phase 1 clinical study of QRL-101 (QRA-244), a potentially novel selective Kv7.2/7.3 ion channel opener to treat hyperexcitability-induced disease progression in amyotrophic lateral sclerosis (ALS). QRL-101 targets Kv7.2/7.3 potassium channels. Kv7 modulation can potentially lower spinal and cortical motor neuron excitability, potentially improving…
Alzheimer’s drug lecanemab wins accelerated FDA approval
FDA has granted accelerated approval to Leqembi (lecanemab), the amyloid beta-protein inhibitor from Eisai (OTCMKTS:ESALY) and Biogen (Nasdaq:BIIB) at an annual launch price of $26,500. The Institute for Clinical and Economic Review (ICER) had decided that the monoclonal antibody lecanemab should be priced less than $20,600 to be cost-effective. Eisai, which is solely responsible for…
Building on recent progress in Alzheimer’s disease to achieve greater clinical benefit
The recent announcement by Eisai/Biogen of the positive outcome of the CLARITY study of lecanemab — an antibody that preferentially targets soluble toxic beta-amyloid aggregates (protofibrils and oligomers) — supports the decades of research, overwhelming genetic data, and basic understanding that beta-amyloid is a bad actor in the Alzheimer’s disease process. It is also much-welcomed…