Prescribed Reading: ASH abstracts hit biotech hard

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

Mergers & analysis

Another light week on the deal front, despite an onslaught of earnings calls, but multiple companies talked about how they plan to shift their business development strategies.

Sanofi disappointed investors when it demonstrated further evidence of a directionless trajectory. The company said it will not focus as much on oncology M&A now that its lost out on the Medivation acquisition to Pfizer, and would instead look for deals in spaces that it has more of a presence, like immunology.

Meanwhile, Allergan plans to keep spending its $25 million war chest on bolt-on acquisitions that could help drive its R&D engine. Now that the company is no longer a generics player, and wants to be a major player in the pharma space, it has to build up its pipeline. The company has been focusing most recently on the gastrointestinal space, particularly NASH and eye care, with four deals in those spaces in the last few months.

We're also likely to see an upsurge in biotechs in the next couple years now that Third Rock Ventures has closed its fourth and largest fund, securing $616 million in capital. The investment firm is known for incubating interesting biotechs with their partners at the helm.

Clinically relevant

Abstracts from the American Society of Hematology meeting dropped earlier this week, exposing disappointing results for several biotech companies that sent shares spiraling. While the meeting isn't until early December, the trial recaps are a preview of what's to come.

Bluebird bio will be presenting further results from trials of its Beta-thalassemia gene therapy Lentiglobin. While some patients are responding well to the treatment, others are not showing the same cure rates. Meanwhile, its work in sickle cell disease isn't going as well as planned either.

Spark Therapeutics was hit hard as well, after the biotech and its partner, Pfizer, released an abstract from an early-stage study of SPK-9001 in hemophilia B patients. While most of the seven patients in the study responded well, one developed an immune response and another had to be treated for a bleed.

Highly regulated

The World Health Organization released draft guidance addressing changes to biologic and biosimilar drugs. Ultimately, the guidelines are meant to increase transparency about changes to these types of drugs.

The document suggests that companies should potentially submit a supplemental application to regulators if any changes are conducted to the drugs after they hit the market, such as changes to the manufacturing process.

People have until Dec. 16 to submit comments on the document. The guidelines, at this point, are just to present information and don't represent a regulation.

Off the bench

Earlier this week, Avacta Group announced a research collaboration with Memorial Sloan Kettering Cancer Center to study novel CAR-T cell-based immunotherapies. The focus of the collaboration will use Affimers developed by Avacta that bind to CD-19 and will be led by researchers from Memorial Sloan Kettering.

"CAR-T cell therapy is an emerging and very exciting area of immuno-oncology which holds enormous clinical potential," said Avacta CEO Alastair Smith in a statement. "The generation of positive data in these validated models of disease has the potential to open up highly valuable licensing and partnering opportunities for Avacta in this therapy area which has attracted so much attention in the past couple of years."

Meanwhile, researchers in Japan have improved a treatment for rare genetic diseases by reducing toxicity. A team of researchers at Tokyo Medical and Dental University are working to improve a drug that treats Niemann-Pick type C (NPC) disease, a rare genetic disorder that plays a role in proteins that affect cholesterol and can results in fatal neurodegeneration and enlargement of the liver.

The compound the team developed could have "great potential to improve lysosomal cholesterol accumulation in NPC disease without marked toxicity," said the researchers in a study published in the journal of Science and Technology of Advanced Materials.


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Filed Under: Drug Discovery Clinical Trials Mergers and Acquisitions / Deals Regulatory / Compliance