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…
Biogen gives up seeking European approval for aducanumab
After winning FDA approval for the controversial Alzheimer’s drug Aduhelm (aducanumab) in June 2021, Biogen (Nasdaq:BIIB) has encountered a string of setbacks. Most recently, the company has decided to yank its applications for market approval after the European Medicines Agency (EMA) recommended the refusal of the marketing authorization for Aduhelm in December 2021. At that…
Alzheimer’s group files FOIA request related to CMS decision on amyloid-targeting antibodies
UsAgainstAlzheimer’s, a nonprofit organization committed to stopping Alzheimer’s disease, is hoping to get more information related to CMS’s national coverage determination related to coverage for Aduhelm (aducanumab) from Biogen (Nasdaq:BIIB) and other monoclonal antibodies targeting amyloid to treat Alzheimer’s disease. The organization has filed a Freedom of Information Act (FOIA) request for information about the decision. UsAgainstAlzheimer’s…
Neuron23 names chief medical officer
The early-stage biotech Neuron23 has appointed Dr. Sam Jackson as its first chief medical officer. South San Francisco, California–based Neuron23 specializes in developing precision medicines for genetically-defined neurological and immunological diseases. Jackson is a board-certified emergency physician with fellowship training in medical toxicology. “I am thrilled to welcome Dr. Jackson to our growing Neuron23 leadership…
Eisai amends Aduhelm collaboration with Biogen
Aduhelm (aducanuamab) co-developer Eisai Co. (TYO:4523) has agreed to modify its economic partnership agreement with Biogen (Nasdaq:BIIB). Starting January 1, 2023, Eisai will receive a tiered royalty on net sales of the Alzheimer’s disease therapy Aduhelm (aducanumab) rather than share profits or losses of the drug. Eisai would receive between 2% and 8% of annual sales…
Biogen plays up spinal muscular atrophy pipeline
As Biogen Inc. (Nasdaq: BIIB) continues to struggle to capitalize on the Alzheimer’s drug Aduhelm (aducanumab), the company is highlighting new data from its Spinraza (nusinersen) and spinal muscular atrophy (SMA) research program. First approved in 2016, Spinraza is a survival motor neuron-2 (SMN2)-directed antisense oligonucleotide indicated to treat spinal muscular atrophy (SMA). At the…
AbbVie and Gedeon Richter unveil neuropsychiatric alliance
AbbVie (NYSE:ABBV) and Gedeon Richter have formed a co-development and license agreement focused on novel dopamine receptor modulators with the potential to treat neuropsychiatric diseases. Lake Bluff, Illinois-headquartered AbbVie and Budapest, Hungary-based Gedeon Richter have already collaborated for 15 years in central nervous system projects, including the development of cariprazine (Vraylar/Reagila), which is now authorized to…
iQure Pharma says iQ-008 meets Rule of 5 for neuropathic pain
The biotech iQure Pharma Inc. has announced that the analgesic compound iQ-008 demonstrated efficacy in in vivo experiments. In particular, Wilmington, Delaware-based iQure announced that iQ-008 meets Lipinski’s rule of five criteria. Also known as Pfizer’s rule of five, the Rule of 5 (RO5) helps pharmaceutical companies determine whether a compound is druglike. Additionally, iQure…
AbbVie shares positive Phase 3 atogepant data to prevent chronic migraine
Gepants have gained ground in recent years as a migraine treatment following the FDA’s approval of Allergan’s Ubrelvy (ubrogepant) in 2019. Now, AbbVie (NYSE:ABBV) has announced that a Phase 3 study testing Qulipta (atogepant) met its primary endpoint of reducing mean monthly migraine days over placebo. FDA approved atogepant as a preventive treatment of episodic…
CODA Biotherapeutics appoints new chief scientific officer
South San Francisco, California–based biopharma CODA Biotherapeutics names Susan Catalano as chief scientific officer. Founded in 2014, CODA Biotherapeutics is working on a gene therapy-mediated chemogenetic platform for intractable neurological disorders. Catalano aims to help advance the company’s platform to find treatments for unmet neurological needs. “CODA’s combination of deep expertise in gene therapy and…
Scientists find new potential drug targets for neurodegenerative diseases
Researchers at Oregon State University (OSU) have discovered a new class of potential drug targets for diseases like Alzheimer’s, Parkinson’s and amyotrophic lateral sclerosis (ALS). The scientists are working to identify the best method to attack the targets — oxidized proteins. The most potent oxidant of the bunch is peroxynitrite, which is produced in conditions involving inflammation.…
QurAlis bolsters its executive team with three new leaders
Cambridge, Massachusetts–based biotech QurAlis Corp., has appointed Bryan Boggs as head of regulatory affairs. In addition, Christopher Gerry Lohan will join as head of clinical operations, and Guzide Adhikari will take on the role of head of global supply chain management. The company is focused on developing novel treatments for amyotrophic lateral sclerosis (ALS) and…
Biogen unveils FTC aducanumab probe
In its annual SEC report, Biogen reported that the Federal Trade Commission had sent the company a civil investigative demand related to the controversial Alzheimer’s drug aducanumab (Aduhelm). The company received a similar notice from the SEC. Lawmakers in the U.S. House of Representatives and the Office of Inspector General of HHS are also investigating…