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…

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…

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 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…

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 (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…

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…

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, 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, 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…

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 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…

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…

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…

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…

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:…

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…

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…

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…

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…

A recent analysis involving more than 100,000 patients with rheumatoid arthritis concluded that those receiving hydroxychloroquine (HCQ) had a lower risk of developing Alzheimer’s disease than those taking the immunosuppressant methotrexate. Nature‘s Molecular Psychiatry published the analysis. HCQ is a disease-modifying anti-rheumatic drug (DMARD) that can reduce pain and swelling associated with arthritis. It is also…

A Phase 1/2 study (NCT03395210) investigating Sanofi (Nasdaq:SNY) Bruton tyrosine kinase inhibitor rilzabrutinib in immune thrombocytopenia (ITP) has also shed light on the cognitive impairment involved in the disease. Patients with ITP, an autoimmune disease that involves increased platelet destruction, have often complained of fatigue, problems with cognition and other symptoms that physicians have dismissed,…

Eli Lilly and Company (NYSE:LLY) announced today that it successfully completed its acquisition of Akouos (Nasdaq:AKUS). Indianpolis-based Eli Lilly’s acquisition expands its efforts in genetic medicines. Akouos offers a portfolio of potential first-in-class adeno-associated viral gene therapies. The gene therapies treat inner ear conditions, including sensorineural hearing loss. The company initially announced the acquisition in…

Tokyo-headquartered Eisai Co. is preparing to share efficacy, safety and biomarker findings from its Phase 3 confirmatory clinical study of the amyloid beta-protein inhibitor lecanemab. The company will present full results from the Phase 3 confirmatory Clarity AD study of lecanemab in patients with early Alzheimer’s disease in a session at the Clinical Trials on…

In May, Biogen (Nasdaq:BIIB) CEO Michel Vounatsos announced his resignation following a precipitous decline in its share price after failing to achieve material sales of its Alzheimer’s drug Aduhelm (aducanumab). Roughly half a year later, Biogen has installed the German-Canadian executive Christopher Viehbacher to lead the company. Viehbacher will assume the role of president, CEO…