Biogen (BIIB) continues to drive innovation in neuroscience, using biomarkers, biological mechanisms and other cutting-edge technologies to develop therapies to fight some of the toughest-to-treat diseases, including MS, ALS, Parkinson's and Alzheimer's.
They are fearless in terms of trying new approaches to gene therapy and gene correction as well as biological therapeutics in areas where small molecules have been the norm," Stifel analyst Tom Shrader told IBD.
Cowen analyst Eric Schmidt also is a fan. "From where we sit on Wall Street, everyone appreciates that Biogen is very good at understanding the basic science and underlying biology behind neurological diseases," Schmidt told IBD. "They have been doing it for a long time and have good basic research.
"In order to innovate you need that solid foundation in that space."
Such high praise earned Biogen recognition as an IBD Biotech Innovators award winner and a future observers call bright.
Biogen, which on Friday touched a 27-month high of 338.94 (closing at 337.89) and carries a very high IBD Composite Rating of 96, already has built the leading portfolio of treatments for multiple sclerosis with innovative drugs such as Tecfidera. In 2013, Tecfidera brought to a wide range of MS patients a combination of efficacy, safety, tolerability and ease of administration not seen before in an MS drug.
Tecfidera still leads the MS market, with 38% market share worldwide, according to Biogen.
The biotech pioneer also has a groundbreaker in Spinraza, the only approved treatment for spinal muscular atrophy. SMA, the No. 1 genetic cause of death in infants, is marked by progressive and debilitating muscle weakness.
Drug Could 'Modify' Alzheimer's
In addition, Biogen could have a pioneer treatment with its drug aducanumab, being developed to treat early Alzheimer's disease.
"Should aducanumab work, it would be the first therapy to show a disease-modifying effect in Alzheimer's," analyst Schmidt said.
The "modifying effect" has to do with the way aducanumab works. The memory loss and functional decline in Alzheimer's is linked to amyloid plaques, which are abnormal deposits that build up in the brain.
That's where aducanumab, an antibody that "binds" to amyloid plaque in the brain, comes in: "Based on preclinical and Phase 1B trial data to date, treatment with aducanumab has been shown to reduce amyloid plaque levels," the company said in August, in an analysis of its ongoing trial.
"These early studies of aducanumab show its effectiveness in removing amyloid plaque from the brain as well as its potential effect on the slowing of cognitive decline in patients suffering from Alzheimer's disease," Alfred Sandrock, the company's chief medical officer, said at the time.
Biogen's approach to developing aducanumab is innovative, analysts say.
"It used imaging to enrich the population of patients in the (Phase 1B) trial with those the drug was most likely to help," analyst Shrader explained.
The company acknowledges, however, that any final approvals for aducanumab are still "years away."
Spinal Muscular Atrophy Drug The Driver
For now, Spinraza is Biogen's big growth driver.
"Based on Spinraza's tremendous growth outlook, Biogen's financial outlook for the next couple of years should be good," Schmidt said.
But he also says new drugs will have to pan out at some point.
"Once Spinraza's trajectory begins to flatten, it remains to be seen whether anything in the company's pipeline is capable of driving growth," Schmidt said.
Biogen has licenses with Ionis Pharmaceuticals (IONS) that give Biogen the global rights to develop, manufacture and commercialize Spinraza.
Last December, Spinraza was approved in the U.S. with a full drug label, meaning the drug can treat anyone with SMA. It is also approved in the European Union, Japan, Canada and Brazil.
Shrader says Spinraza was developed using "an unbelievably innovative approach — changing the expression of genes in the patient."
Genes encode proteins, and proteins "dictate" cell function. The genes "expressed" in a particular cell determine what the cell can do.
Spinraza is an antisense oligonucleotide (ASO). It uses Ionis' proprietary antisense technology designed to treat SMA caused by "mutations or deletions" in the SMN1 gene, which lead to SMN protein deficiency, says Biogen.
Through the use of ASO technology, Spinraza has the potential to increase the amount of "full-length" SMN protein in individuals with SMA and regulate gene expression, Biogen says.
As analyst Shrader explained: "Changing the expression of genes means patients start to express a good copy of the protein SMN to replace the expression of a bad copy of the SMN gene."
Spinraza Drug Is A Lifesaver
Kenneth Hobby, president of advocacy organization Cure SMA, calls Spinraza a lifesaver.
"Spinraza is a very powerful drug," Hobby told IBD. "It is a very accurate genetic-based technology that goes to correcting the cause of the disease.
"People are living with the disease now, when they would have passed away at 2 years old. They're breathing and eating, and some are even walking."
Hobby says Biogen and Ionis used an innovative approach to design their clinical trials.
"They worked with the patient community very early on in the process to help design the clinical trials to get critical information on what benefits to measure in the trials," Hobby said. "They were open in communicating results and information.
"This approach of being honest and open with sharing information and goals is what ultimately gave a bigger and better result, which was the data needed to get early approval and a full drug label for all patients from the FDA."
Analysts expect Spinraza will be Biogen's next blockbuster — a drug with annual sales over $1 billion — following its MS drugs. Tecfidera had $3.96 billion in 2016 sales, Avonex $2.31 billion and Tysabri $1.96 billion.
Analyst Schmidt says Spinraza could hit $3 billion to $4 billion in annual sales but cautions "it's still early days."
He estimates Spinraza's sales will hit $1.25 billion in 2018, up from $875 million this year, and peak in five to eight years.
Unlocking Key To Biological Mechanism
Aducanumab also is a potential blockbuster. Stifel's Shrader estimates its yearly sales at $2 billion to as much as $12 billion, "depending on the actual fraction of patients it helps and the final side effects."
Cowen's Schmidt says Wall Street's biggest hopes among Biogen's pipeline candidates rest on aducanumab. "Many of us think it would be a terrific drug," he said.
Biogen would not make an executive available to comment for this story, but its media staff said remarks made by Biogen R&D chief Michael Ehlers on the company's Q2 earnings conference call offer insight into Biogen's approach.
On that July call, Ehlers said Biogen concentrates on four core growth areas: MS and neuroimmunology, Alzheimer's disease and dementia, Parkinson's disease and related movement disorders, and neuromuscular disease including SMA and amyotrophic lateral sclerosis (ALS, also known as Lou Gehrig's disease).
Ehlers said Biogen is succeeding in understanding the basic biological mechanism or disease process of these diseases and developing therapies that target these mechanisms and processes.
Biogen is gaining a better understanding of genetics and "mechanisms of action," he said.
"We are developing insights from longitudinal studies of populations," he said on the call. "For example, we are mining our clinical data and experimental electrophysiological endpoints in ALS, yielding clinical measures with the potential to enable trials that are half the size, twice the speed, and a third the cost."
Biogen continues to invest heavily in "biomarkers" (specific physical traits) for disease pathology and progression, said Ehlers.
"In Parkinson's disease, our advanced imaging biomarker investments are allowing patient selection and detection of disease progression that has not previously been possible," he said. "We have identified genetic modifiers and causal pathways that are providing proprietary insight to produce our next generation of drug candidates."
Analyst Shrader says Biogen uses imaging to understand when neurological drugs are working, saying development of aducanumab depended on its innovative use of imaging.
Biogen licensed aducanumab from Switzerland-based biotech Neurimmune.
In the ongoing Phase 1B trial, researchers found in brain scans that aducanumab reduced the levels of the protein amyloid beta, which is thought to be toxic to brain cells.
Attacks Disease Before Symptoms Apparent
Research shows that amyloid begins to build up in the brain 10 to 20 years before Alzheimer's symptoms appear, James Hendrix, director of global science initiatives for the Alzheimer's Association advocacy organization, told IBD.
"The clinical design of Biogen's Phase 1B trial was innovative," Hendrix said. "One hypothesis of why so many Alzheimer's drugs have negative results in late-stage clinical trials is they were given to people who had already progressed to Alzheimer's dementia and were therefore very sick."
That wasn't the case in Biogen's Phase 1B trial. The patients included in the trial had early Alzheimer's or early memory problems, with high levels of the amyloid protein in their brain based on an amyloid PET scan.
"Biogen's strategy is to treat the disease early or before the memory and thinking symptoms appear," said Hendrix. "How do you find these people? The answer for Biogen is to use brain amyloid PET imaging."
To determine who should be in the trial, Biogen used amyloid PET imaging as a "screening tool," Hendrix said.
"Using amyloid PET scans in this way has ensured that everybody in the Biogen trial had high levels of amyloid in their brain," he said. "After one year, the study showed that the drug did what it was supposed to do, which was remove amyloid from the brain.
"That is, to my knowledge, the first time a trial like this has ever been done."
http://www.investors.com/news/technology/biogen-biomarkers-mark-way-to-alzheimers-parkinsons-treatments/
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