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…
Hydroxychloroquine tied to lower Alzheimer’s risk in arthritis patients in study
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…
Rilzabrutinib study highlights cognitive impairment in patients with chronic immune thrombocytopenia
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,…
Lilly completes acquisition of Akouos
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…
Eisai prepares to share more findings for Alzheimer’s antibody lecanemab
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…
Biogen names Christopher Viehbacher to CEO post
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…
FDA hands Minerva Neurosciences refusal to file letter for its roluperidone NDA for schizophrenia
The biopharma Minerva Neurosciences (Nasdaq:NERV) announced that it had received a refusal to file letter from the FDA for its New Drug Application (NDA) for roluperidone. The NDA covered the use of the drug candidate as a treatment for the negative symptoms in schizophrenia patients. Negative schizophrenia symptoms can include social withdrawal, mood flatness and depression. Drug…
Athira Pharma’s Phase 2/3 LIFT-AD study wins independent endorsement
The neurodegeneration-focused biopharma Athira Pharma (Nasdaq:ATHA) will continue its Phase 2/3 LIFT-AD study of fosgonimeton (ATH-1017) in patients with mild-to-moderate Alzheimer’s disease. The company recently announced an unblinded interim efficacy and futility analysis of LIFT-AD. In addition, an independent data monitoring committee recommended continuing the study of fosgonimeton (ATH-1017) in patients with mild-to-moderate Alzheimer’s disease…
Innovation1 aims to treat pediatric epilepsy with novel CBD-inspired approach
Cannabidiol (CBD), the phytocannabinoid discovered in 1940, is seemingly everywhere these days. In states where it is legal, it is in drinks, pet products and ointments. FDA frowns on CBD’s ubiquity. Over the years, the agency has warned several companies for selling unapproved CBD-based products. The small molecule drug discovery company Innovation1 Biotech (OTCQB:IVBT) is developing a…
FDA approves Amylyx ALS drug Relyvrio
Amylyx Pharmaceuticals (Nasdaq:AML) has won FDA approval for Relyvrio (AMX0035; sodium phenylbutyrate and ursodoxicoltaurine), notching the first win for amyotrophic lateral sclerosis (ALS) in five years. The drug received Health Canada approval in June. The drug is known as Albrioza in that country. In a summary, FDA reviewers note that the limited clinical data available…
Why Algernon is investigating DMT in stroke rehabilitation
The clinical-stage company Algernon Pharmaceuticals (CSE:AGN; Frankfurt:AGW0; OTCQB: AGNPF) has received approval to run a Phase 1 clinical study of an IV formulation of AP-188. The study will explore the drug candidate’s potential, a formulation of the classic psychedelic N,N-dimethyl tryptamine or DMT, to treat stroke patients in the Netherlands. The Stichting Beoordeling Ethiek Biomedisch…
Eisai and Biogen report positive Phase 3 trial results for new Alzheimer’s drug
Eisai and Biogen (Nasdaq:BIIB) today announced positive topline data from a Phase 3 trial of lecanemab for Alzheimer’s disease. BIIB shares are skyrocketing on the back of the data today. They are up 37.6% at $272.12 apiece in mid-afternoon trading. Lecanemab is an investigational anti-amyloid beta (Aβ) protofibril antibody. The global Phase 3 confirmatory Clarity…
Analysts are bullish on Eli Lilly with its ‘transformative’ drugs
UBS recently issued a report upgrading Eli Lilly (NYSE:LLY) to a “Buy” rating as its diabetes and Alzheimer’s drugs present lucrative opportunities. According to the report, the pharmaceutical giant’s Mounjaro (tirzepatide) “could be the biggest drug ever.” The type 2 diabetes treatment received FDA approval in May. GlobalData said at the time that tirzepatide could…
Ensysce Biosciences’ CEO aims to combat prescription drug abuse
Ensysce Biosciences CEO Lynn Kirkpatrick oversees the development of novel medicines to reduce or eliminate prescription drug abuse. The La Jolla, California-based company’s oxycodone prodrug candidate PF614 is now in a Phase 2 clinical trial. It is designed to offer similar pain relief as prescription opioids with a limited risk of drug abuse and addiction.…
Neuron23 and Qiagen partner on NGS companion diagnostic for LRRK2 inhibitor
The early-stage biotech Neuron23 and Qiagen (NYSE:QGEN) will work together to develop the first next-generation sequencing (NGS) companion diagnostic for Neuron23’s leucine-rich repeat kinase 2 (LRRK2) inhibitor for Parkinson’s disease NEU-723. In the collaboration, Qiagen will focus on developing and validating a clinical trial assay to detect biomarkers that predict the responsiveness of Parkinson’s disease…
U.S. government launches CP-RND, a public-private partnership for rare neurodegenerative diseases
The FDA and NIH have debuted the Critical Path for Rare Neurodegenerative Diseases (CP-RND), a public-private partnership focused on developing new therapies for amyotrophic lateral sclerosis (ALS) and rare neurodegenerative diseases. One of the goals of the CP-RND is to foster innovation in drug discovery and development as well as clinical testing. In addition, CP-RND…
Merck forges Alzheimer’s pact with Cerevance
Merck & Co. (NYSE:MRK) could pay more than $1 billion to the Boston-based biotech startup Cerevance to develop potential drugs for Alzheimer’s disease. The collaboration will center around Cerevance’s Nuclear Enriched Transcript Sort sequencing (NETSseq) technology platform. The platform’s origins trace back to research at the Howard Hughes Medical Institute. Under the terms of the…
Scholar Rock sees continued progress with apitegromab in spinal muscular atrophy
Scholar Rock recently announced Q2 results, highlighting continued progress in its Phase 3 SAPPHIRE trial for the selective myostatin-inhibitor apitegromab in spinal muscular atrophy (SMA). SAPPHIRE is a randomized, double-blind, placebo-controlled study. The Cambridge, Massachusetts–based company also shared new data from its Phase 2 TOPAZ trial focused on apitegromab that showed significant and sustained improvement…
How EEG data and machine learning can inform drug development
EEG data can bolster rare disease drug research and trials. An epilepsy drug trial, for instance, could ask patients to log the number of seizures they experience in a day. “If you look at a seizure diary, there might be 10 seizures a day. But if you look at EEG, there could be 150,” said Dr.…
The pandemic is fueling the demand for natural alternatives to antidepressants
The pandemic, climate change, rapid inflation and a surge in violence are contributing to a mental health crisis. Against that backdrop, demand for antidepressants, anti-anxiety and anti-insomnia drugs is booming. In 2021, there were more than 337 million antidepressant prescriptions in the U.S., according to data from IQVIA. In the first four months of 2022,…
Fluoride nanoparticles could hold promise in treating Alzheimer’s
An international team of researchers has identified a technique that uses fluoride nanoparticles to potentially eliminate protein deformations. The method, which also can promote positive structural changes, could lead to the prevention and treatment of Alzheimer’s disease. The scientists from Tokyo University of Science (TUS) in Japan and Nazarbayev University in Kazakhstan published the research…
FDA places clinical hold on Astellas clinical trial focused on Pompe disease
Astellas Pharma (TSE:4503) has revealed that the FDA has slapped a clinical hold on its Phase 1/2 FORTIS Pompe disease study after observing a serious adverse event (SAE) — peripheral sensory neuropathy — in a trial participant. Peripheral sensory neuropathy results from damage to nerves outside of the central nervous system. The condition can lead…