After the Alzheimer's Crash, Should Biogen Turn to M&A to Bolster Its Pipeline?

MARK TERRY | April 26, 2019 | 75 views

Earlier this week, Biogen reported out its first-quarter financial results, showing an 11% increase in revenues, hitting $3.5 billion. But in light of the company’s announcement in late March that it was dropping development of aducanumab for Alzheimer’s, analysts are speculating on what the company needs to do to stay profitable. And the Alzheimer’s pivot wasn’t the only challenge, although that announcement resulted in a stock drop where the company lost $18 billion in value overnight. But one of the company’s top-selling drugs is Spinraza to treat a rare muscular disease called spinal muscular atrophy (SMA). Spinraza came on the market as the first viable treatment, but Novartis is developing its own drug, which in late-stage trials appears to be a one-and-done gene therapy, rather than an ongoing treatment like Spinraza. If Novartis’ Zolgensma is approved, it will likely be the therapy of choice for SMA Type 1 patients, although it is as of now not a treatment for all types of SMA like Spinraza is.

Spotlight

AzurRx BioPharma, Inc.

AzurRx BioPharma aims to become a leader in developing non-systemic, recombinant protein therapies for the treatment of gastrointestinal diseases and related conditions. MS1819 recombinant lipase for exocrine pancreatic insufficiency is the lead development program with additional early stage research being conducted for the prevention of hospital-acquired infection. The company is headquartered in New York, NY, with scientific operations based in Langlade, France.

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Top 10 biotech IPOs in 2019

Article | July 11, 2022

The big question at the start of 2019 was whether the IPO window would stay open for biotech companies, particularly those seeking to pull off ever-larger IPOs at increasingly earlier stages of development. The short answer is yes—kind of. Here’s the long answer: In the words of Renaissance Capital, the IPO market had “a mostly good year.” The total number of deals fell to 159 from 192 the year before, but technology and healthcare companies were standout performers. The latter—which include biotech, medtech and diagnostics companies—led the pack, making up 43% of all IPOs in 2019. By Renaissance’s count, seven companies went public at valuations exceeding $1 billion, up from five the year before

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MEDTECH

Cell Out? Lysate-Based Expression an Option for Personalized Meds

Article | July 16, 2022

Cell-free expression (CFE) is the practice of making a protein without using a living cell. In contrast with cell line-based methods, production is achieved using a fluid containing biological components extracted from a cell, i.e., a lysate. CFE offers potential advantages for biopharma according to Philip Probert, PhD, a senior scientist at the Centre for Process Innovation in the U.K.

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MEDTECH

Closing bacterial genomes from the human gut microbiome using long-read sequencing

Article | July 12, 2022

In our lab, we focus on the impact of the gut microbiome on human health and disease. To evaluate this relationship, it’s important to understand the particular functions that different bacteria have. As bacteria are able to exchange, duplicate, and rearrange their genes in ways that directly affect their phenotypes, complete bacterial genomes assembled directly from human samples are essential to understand the strain variation and potential functions of the bacteria we host. Advances in the microbiome space have allowed for the de novo assembly of microbial genomes directly from metagenomes via short-read sequencing, assembly of reads into contigs, and binning of contigs into putative genome drafts. This is advantageous because it allows us to discover microbes without culturing them, directly from human samples and without reference databases. In the past year, there have been a number of tour de force efforts to broadly characterize the human gut microbiota through the creation of such metagenome-assembled genomes (MAGs)[1–4]. These works have produced hundreds of thousands of microbial genomes that vastly increase our understanding of the human gut. However, challenges in the assembly of short reads has limited our ability to correctly assemble repeated genomic elements and place them into genomic context. Thus, existing MAGs are often fragmented and do not include mobile genetic elements, 16S rRNA sequences, and other elements that are repeated or have high identity within and across bacterial genomes.

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Selexis Cell Line Development Strategies

Article | February 11, 2020

In today’s biotechnology landscape, to be competitive, meet regulations, and achieve market demands, “we must apply Bioprocessing 4.0,” said Igor Fisch, PhD, CEO, Selexis. In fact, in the last decade, “Selexis has evolved from cloning by limiting dilution to automated cell selection to nanofluidic chips and from monoclonality assessment by statistical calculation to proprietary bioinformatic analysis,” he added. Single-use processing systems are an expanding part of the biomanufacturing world; as such, they are a major component of Bioprocessing 4.0. “At Selexis, we use single use throughout our cell line development workflow. Currently, we have incorporated single-use automated bioprocessing systems such as ambr® and the Beacon® optofluidic platform for accelerated cell line development. By using these systems and optimizing our parameters, we were able to achieve high titers in shake flasks. Additionally, the Beacon systems integrate miniaturized cell culture with high-throughput liquid handling automation and cell imaging. This allows us to control, adjust, and monitor programs at the same time,” noted Fisch.

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Spotlight

AzurRx BioPharma, Inc.

AzurRx BioPharma aims to become a leader in developing non-systemic, recombinant protein therapies for the treatment of gastrointestinal diseases and related conditions. MS1819 recombinant lipase for exocrine pancreatic insufficiency is the lead development program with additional early stage research being conducted for the prevention of hospital-acquired infection. The company is headquartered in New York, NY, with scientific operations based in Langlade, France.

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Biogen’s Experimental Drug Shows Promise for Genetic Form of ALS

biospace | May 15, 2019

Amyotrophic lateral sclerosis (ALS) is a devastating disease with no approved treatments. Diagnosis is tantamount to a death sentence and researchers are desperately searching for some kind of therapy. Although there have been numerous failed treatments, Biogen believes it might be on the right track. “ALS is a disease you really want to make a difference in,” Toby Ferguson, M.D., Ph.D, Senior Medical Director for Neuromuscular Research and Early Development at Biogen told BioSpace. Earlier this month, Biogen announced interim results from a Phase I/II study of tofersen, an antisense oligonucleotide the company licensed from Ionis Pharmaceuticals that is being studied as a potential treatment of ALS patients who have a confirmed superoxide dismutase 1 (SOD1) mutation. The interim results showed both a proof-of-biology and proof-of-concept, which supported the start of a Phase III clinical trial to confirm the efficacy and safety of tofersen in that patient population, Ferguson said. Ferguson said this was exciting news as it showed the potential of tofersen to target the genetic driver of this form of ALS.

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Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3

Biogen | November 07, 2016

Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. SPINRAZA demonstrated a favorable safety profile in the study.

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Nature Publishes Results from Pre-Clinical Research and Phase 1b Study of Biogen’s Investigational Alzheimer’s Disease Treatment Aducanumab

Biogen | August 31, 2016

Results from pre-clinical research and PRIME, the Phase 1b study of Biogen’s (NASDAQ: BIIB) investigational treatment for early Alzheimer’s disease (AD), aducanumab, were published today in Nature. The full manuscript titled, “The Antibody Aducanumab Reduces Aβ Plaques in Alzheimer’s Disease,” can be found in the 1 September, 2016 issue of Nature

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Biogen’s Experimental Drug Shows Promise for Genetic Form of ALS

biospace | May 15, 2019

Amyotrophic lateral sclerosis (ALS) is a devastating disease with no approved treatments. Diagnosis is tantamount to a death sentence and researchers are desperately searching for some kind of therapy. Although there have been numerous failed treatments, Biogen believes it might be on the right track. “ALS is a disease you really want to make a difference in,” Toby Ferguson, M.D., Ph.D, Senior Medical Director for Neuromuscular Research and Early Development at Biogen told BioSpace. Earlier this month, Biogen announced interim results from a Phase I/II study of tofersen, an antisense oligonucleotide the company licensed from Ionis Pharmaceuticals that is being studied as a potential treatment of ALS patients who have a confirmed superoxide dismutase 1 (SOD1) mutation. The interim results showed both a proof-of-biology and proof-of-concept, which supported the start of a Phase III clinical trial to confirm the efficacy and safety of tofersen in that patient population, Ferguson said. Ferguson said this was exciting news as it showed the potential of tofersen to target the genetic driver of this form of ALS.

Read More

Biogen and Ionis Pharmaceuticals Announce SPINRAZA (nusinersen) Meets Primary Endpoint at Interim Analysis of Phase 3

Biogen | November 07, 2016

Biogen (NASDAQ:BIIB) and Ionis Pharmaceuticals (NASDAQ:IONS) announced that SPINRAZATM (nusinersen), an investigational treatment for spinal muscular atrophy (SMA), met the primary endpoint at the interim analysis of CHERISH, the Phase 3 study evaluating SPINRAZA in later-onset (consistent with Type 2) SMA. The analysis found that children receiving SPINRAZA experienced a highly statistically significant improvement in motor function compared to those who did not receive treatment. SPINRAZA demonstrated a favorable safety profile in the study.

Read More

Nature Publishes Results from Pre-Clinical Research and Phase 1b Study of Biogen’s Investigational Alzheimer’s Disease Treatment Aducanumab

Biogen | August 31, 2016

Results from pre-clinical research and PRIME, the Phase 1b study of Biogen’s (NASDAQ: BIIB) investigational treatment for early Alzheimer’s disease (AD), aducanumab, were published today in Nature. The full manuscript titled, “The Antibody Aducanumab Reduces Aβ Plaques in Alzheimer’s Disease,” can be found in the 1 September, 2016 issue of Nature

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