Prescribed Reading: Spotlight on CAR-T, Cures
A weekly guide to the goings-on in the biopharma industry.
Biopharma is a complex, rapidly evolving industry that is highly regulated and closely watched — and that means there is constant news. Here's a closer look at the clinical trials, M&A, cool science and regulations that are driving the industry this week.
In case you missed it
- The winners of the 2016 Dive Awards
- Alnylam touts its hemophilia assets
- And Sangamo helps fill Biorativ's pipeline
Mergers & analysis
AstraZeneca continued its deal spree this week , this time signing a multi-target deal with Bicycle Therapeutics — instead of outlicensing any more of its non-core assets. The deal is aimed at identifying and developing the company's proprietary bicyclic peptides into drug candidates for respiratory, metabolic and cardiovascular diseases.
The British big pharma has epitomized the refocusing that has been going on in the industry. AstraZeneca has been shedding assets at a rapid clip over the last quarter as it tries to bring in revenues and cut costs, in hopes of reaching the aggressive $45 million annual goal that was set by CEO Pascal Soriot a couple of years ago.
M&A has been slow this year, but as the year wraps up, expect dealmaking to pick up. Analysts and investors are hoping that the new administration and a glut of cash will mean major M&A going forward.
It's a big week in innovative science. The American Society of Hematology annual meeting is set to start this weekend and there will be news about the up and coming CAR-T immunotherapies. Analysts and investors are eager to get more information from Juno Therapeutics about the recent deaths in its Phase 2 clinical trial.
But don't expect the bad news to overshadow the good; other CAR-T developers including Kite Pharma and Novartis will have plenty to say. And don't forget to keep an eye out for news on the latest developments in hemophilia.
Bluebird Bio is also making news with its CAR-T therapy this week. The company announced the results from a small Phase 1 study that it conducted with partner Celgene on its drug bb2121 in patients with advanced multiple myeloma.
The study showed that seven of nine patients responded to the treatment, despite being heavily pre-treated and those that did not respond were on the lowest dose. No signs of cytokine release syndrome were present – a good sign.
While the study was small and early, it gave the industry and investors hope that CAR-T is still as promising as once thought, despite recent setbacks.
The 21st Century Cures Act passed the House of Representatives by a sweeping margin this week and faces positive odds of passing the Senate and being signed into law by President Obama before the end year.
Although the bill has been touted as providing much-needed funding for medical research, it faces some strong opposition in the form of outspoken Democratic Senators Bernie Sanders and Elizabeth Warren, who both argue the Act will give handouts to the pharmaceutical industry and lower the bar for drug approvals.
Even though the Cures Act could be easing the way for pharma companies, recent guidance issued by the Food and Drug Administration will up the standards at contract manufacturers. According to VisionGain, the global contract pharma and biotech manufacturing market was worth $54.54 billion in 2013, and could reach $79.24 billion in 2019, rising by 7.5% a year. As the use of CMOs increases as more pharma companies look for ways to reduce R&D and manufacturing costs, the FDA has been issuing more warning letters to foreign contractors in an effort to keep standards high.
Off the bench
The microbiome is becoming another one of those really cool areas of science as the industry begins to realize that the bacteria in our guts could have a major impact on how we respond to disease and treatment.
Many companies are now running tests on clinical trial patients to see what sort of bacteria they have in their gut with hopes of using the information later to better understand how the microbiome affects treatment.
Axial Biotherapeutics is taking it one step further. The biotech launched this week with a $19.5 million Series A led by Longwood Fund and Domain Associates. The company is based on science from the labs at Caltech and is hoping to leverage the microbiome to treat central nervous system disorders.
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