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…
Women in Pharma: 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…
FDA launches 5-year initiative for rare neurodegenerative diseases
FDA has unveiled its Action Plan for Rare Neurodegenerative Diseases, including amyotrophic lateral sclerosis (ALS). The plan will involve the use of public-private partnerships and incorporate feedback from patients. The initiative will encompass regulatory science initiatives, changes to existing programs and new policy initiatives. Milestones of the initiative include the creation of an FDA rare neurodegenerative…
Biogen halts observational post-marketing study of aducanumab
Biogen (Nasdaq:BIIB) has terminated a study of the controversial Alzheimer’s disease drug Aduhelm (aducanumab) as a result of limited national policy for coverage. In a listing on clinicaltrials.gov, the company notes that it anticipates there will limited prescriptions of the drug in routine clinical practice, which would provide limited enrollment for the planned ICARE AD study.…
Inside Canada’s approval of Amylyx’s ALS drug Albrioza
One of the things that differentiates amyotrophic lateral sclerosis (ALS) from other rare diseases is that “it is a catastrophic diagnosis,” said Chris Aiello, Head of Canada and General Manager, Amylyx Pharmaceuticals. “Patients do not really have much to go on in terms of pharmacological treatments.” Last week, Amylyx Pharmaceuticals received Health Canada approval for…
Athira Pharma stock tanks after announcing results from Phase 2 Alzheimer’s study
The neurodegeneration-focused biopharma Athira Pharma (Nasdaq:ATHA) has seen its stock drop more than 90% since December 31, 2020. Today alone, the company’s share price was down 66.73%, falling to $2.81, in mid-day trading after the company announced disappointing results from the Phase 2 ACT-AD study focused on fosgonimeton in Alzheimer’s disease. The company announced that…
Understanding the promise of Albrioza: Q&A with the CEO of ALS Society of Canada
Amyotrophic lateral sclerosis (ALS) remains a bewildering disease. Although it is rare, it is not uncommon. The lifetime risk of developing the condition is roughly 1 in 300 by age 85. The heterogeneity of the disease is also unique, said Tammy Pighin Moore, CEO of the ALS Society of Canada. “About 5% to 10% of the people diagnosed…
AbbVie files sNDA with FDA to expand atogepant label
AbbVie (NYSE:ABBV) has submitted a supplemental New Drug Application (sNDA) to the FDA to support the use of atogepant (Qulipta) as a preventive treatment of both episodic and chronic migraine. FDA approved the drug in September 2021 as a preventive treatment of episodic migraine in adults. In March 2022, AbbVie announced that the Phase 3…
Genentech’s crenezumab stumbles in Alzheimer’s trial
Roche (SIX:RO, ROG; OTCQX:RHHBY) subsidiary Genentech announced that a study of the amyloid beta-protein inhibitor crenezumab failed to show statistically significant clinical benefit in Alzheimer’s patients. The failure is typical. Historically, 99% of Alzheimer’s drug candidate studies fail to show improvement over placebo, according to Alzheimer’s Research & Therapy. In the research, Genentech partnered with the…
Neuron23 names Kathy Dong to its board
The early-stage biotechnology firm Neuron23 has recruited the chief operating officer of Star Therapeutics, Kathy Dong, to join its board of directors. Founded in 2018, Neuron23 (South San Francisco) specializes in the discovery and development of drugs for neurodegenerative and immunological diseases, drawing insights from data science. Star Therapeutics (South San Francisco) focuses on the…
Investigational ALS drug could slow disease progression
Irvine, Calif.-based Eledon Pharmaceuticals has announced promising topline results from a Phase 2a study of tegoprubart, marking the first time an investigational drug has demonstrated a reduction of inflammatory biomarkers associated with progression of amyotrophic lateral sclerosis (ALS). “We have an investigational drug acting based on what we hypothesized it would do based on the…
Roche’s Evrysdi wins FDA approval in newborns with spinal muscular atrophy
Roche (SIX:RO, ROG; OTCQX:RHHBY) has announced that the FDA has approved a label extension to allow the use of Evrysdi (risdiplam) in babies younger than two months old with spinal muscular atrophy (SMA). SMA is a genetic disease associated with progressive muscle wasting and weakness that affects roughly 1 in 11,000 babies. The drug first…
How the CMS decision to limit Aduhelm coverage could impact Medicare pricing
Since winning approval for the Alzheimer’s drug Aduhelm (aducanumab) in June 2021, Biogen (Nasdaq: BIIB) has faced a punishing string of setbacks. As a result, the company’s share price is currently trading near $200 per share — less than half of its $414.71 peak on June 10, 2021. In April 2022, the Centers for Medicare…
Nine out of ten U.S. neurologists have Aduhelm efficacy doubts
A recent physician survey found that 89% of neurologists in the U.S. have concerns about the efficacy of the controversial Alzheimer’s drug Aduhelm (aducanumab) from Biogen (Nasdaq:BIIB) and Eisai Co. (TYO:4523). Nearly as many — 87% — were concerned about the drug’s safety profile. The survey from the physician social media network Sermo (New York City) asked more than…
Athira Pharma to continue open-label Alzheimer’s extension studies
The biopharma Athira Pharma (Nasdaq: ATHA) has lengthened the current open-label extension (OLEX) study for its Phase 3 LIFT-AD and Phase 2 ACT-AD trials of fosgonimeton (ATH-1017, NDX-1017), hepatocyte growth factor receptor agonist, focused on mild-to-moderate Alzheimer’s disease. The Bothell, Washington–based company made the decision based on feedback from its independent data and safety monitoring…
Biogen CEO Michel Vounatsos to step aside as Aduhelm sales continue to sputter
With the blockbuster dreams of the Alzheimer’s drug Aduhelm (aducanumab) dashed for the foreseeable future, Biogen has announced that its CEO Michel Vounatsos will depart. Vounatsos began his tenure as CEO in 2017. Vounatsos’ plans to exit the company came out on the same day that Biogen reported Q1 adjusted earnings per share of $3.62,…
Why Janssen’s nipocalimab could represent a ‘pipeline in a pathway’
At the heart of Johnson & Johnson’s (NYSE:JNJ) $6.5 billion acquisition of Momenta Pharmaceuticals in 2020 is a portfolio of drug candidates for immune-mediated diseases. One of the promising compounds in the acquisition is nipocalimab, which Janssen sees as a potential “pipeline in a pathway.” Nipocalimab holds promise for an array of diseases, ranging from…