Prescribed Reading: PD-1, CAR-T threatened by clinical holds
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.
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Mergers & analysis
Despite being a short trading week for Wall Street, there has been no shortage of activity. Merck & Co. struck a deal to strengthen its immuno-oncology pipeline ahead of the data dump this weekend in Spain at the European Society of Medical Oncology conference, an event where we are going to get a closer look at data for many of the PD-1 inhibitors. (Stay tuned to BioPharma Dive for more on that.)
Merck picked up German biotech Rigontec for approximately $140 million upfront and another $400 million in potential milestones. The company has a pipeline of agonists that activate the gene RIG-I, an immuno-oncology approach that could complement Merck's blockbuster PD-1 inhibitor Keytruda (pembrolizumab). Rigontec's lead candidate is now moving into human trials.
Elsewhere, Biogen spinout Bioverativ and Bicycle Therapeutics are combining resources to discover and develop treatments for hemophilia and sickle cell disorder. Bioverativ is paying $10 million upfront and making a $4 million investment in R&D, as well as agreeing to $400 million in milestones.
And Chinese biotech 3SBio is getting into the contract manufacturing and development business through the acquisition of Therapure Biopharma's CDMO business. The Chinese biotech is hoping that the purchase will help it expand into the North American market.
AstraZeneca plc and Amgen Inc. are prepping to have a first-in-class biologic for their asthma drug tezepelumab after announcing a Phase 2 trial of the drug showed it significantly reduced asthma exacerbation. Analysts called the results "above expectations."
Boston biotech Verastem Inc. also announced a success for its Phase 3 blood cancer drug and intends to seek approval next year. Getting a green light from the Food and Drug Administration would be a major return on investment for the company, which paid only $28 million in milestones to Infinity Pharmaceuticals Inc. for the drug last year.
Insmed Inc. saw its shares spike 115% on positive results for its inhaled antibiotic. The drug was able to treat a rare lung disease that currently has no approved treatment options. It showed significantly better results for patients on the primary endpoint of culture conversion, but didn't show any improvement on a six-minute walk test.
Elsewhere, Alnylam Pharmaceuticals reported some bad news, halting trials of one of its lead compounds in hemophilia after a patient death. The company paused a Phase 2 and Phase 3 trial until it can figure out a risk evaluation and mitigation strategy.
While last week was characterized by a slew of approvals from the FDA, this week saw an uptick in the number of clinical holds the agency had to place.
The most significant was a round of partial and full clinical holds the agency put on nine trials that are currently testing PD-1/L1 inhibitors with other immune modulating agents. While there have been no safety issues seen in the Bristol-Myers Squibb Company trials or those form partners AstraZeneca and Celgene Corp., the FDA is playing it safe.
The agency took this action after patient deaths occurred in a handful of clinical trials being conducted by Merck. While it's still unclear if there is a class-wide effect, the agency is doing more investigating to find out if combining PD-1/L1 inhibitor with certain other agents could create an ill outcome. This could put a major crimp in the commercial strategy of several companies, which have been relying on a combination strategy for their blockbuster PD-1/L1s to be the backbone of their oncology units.
Meanwhile, a patient death in a Phase 1 trial of Cellectis' CAR-T therapy prompted a clinical hold for that company. There have been a number of patient deaths across clinical trials for the highly touted new technology, raising safety concerns for the gene therapy. The most recent death comes on the heels of the first approval in the space and a major deal for another CAR-T company.
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