November 12, 2018
Illumina is acquiring Pacific Biosciences for $1.2 billion in an all-cash merger of the two gene sequencing biotechs, the companies have announced.
Illumina, based in San Diego, has coveted Pacific Biosciences’ long-read sequencing technologies and expects the merger will allow the company “to reach more applications, accelerate the pace of genomic discovery and bolster our innovation engine,” Illumina President and CEO Francis deSouza said in a statement announcing the deal.
Short-read gene sequences — in essence, smaller strands of DNA, and the kind of product Illumina has specialized in — can produce high-quality, deep coverage of genomes, scientists say. But they also have a limited capacity, especially in mapping complex genetic regions, because they have to be pinpointed precisely. Long-read sequencing — in which strands can be up to 100 times larger than their short-read counterparts — offer a lot more flexibility, experts say.
Illumina says it expects the deal to close mid-next year.
UK: Sanction Sponsors Who Don’t Publish Trial Results
Health regulators should force drug sponsors to publish clinical trial data and sanction those who flout the rules, UK lawmakers say in a new report.
The House of Commons’ Science and Technology Committee says a recent probe found that more than half of clinical trials fail to publish findings within a year of wrapping up as required by European Union rules, creating a “publication bias” that could distort research results.
The UK is scheduled to leave the EU early next year but the lawmakers urged their colleagues to adopt and adhere to its regulations on trials publication — and set aside funding to create audit trails for compliance.
The panel also called on regulators to keep a list of violators and set up a system of sanctions designed to “drive improvements in clinical trials transparency, such as withdrawing favorable ethical opinion or preventing further trials from taking place,” and give health regulators the authority to fine trial sponsors who don’t publish findings.
The report — released last week — also calls out two publicly funded organizations, Public Health England and the National Health Service’s Foundation Trusts, for having a “particularly disappointing” record of compliance.
“Public trust in medicine could easily be eroded by failures in clinical trials transparency from such important parts of the health system,” the committee warns.
You can read the report here: https://bit.ly/2D7ysnD.
Merck Teams with FLX Bio for Keytruda Combinations Trials
Merck is teaming with California-based FLX Bio for a Phase I/II trial to test the effectiveness of a combination of Merck’s cancer drug Keytruda (pemborlizumab) and FLX’s experimental immunotherapy, the companies have announced.
Keytruda is already approved as a treatment for several cancers. FLX has developed an oral cancer immunotherapy, FLX475. The companies want to test FLX475 on its own but also in combination with Keytruda in an open-label, dose-escalation and cohort expansion trial with sites in the U.S., Australia and Asia.
FLX475 is designed to inhibit, selectively, regulatory T cells in tumors. FLX Bio says it has already demonstrated the med’s safety in trials on healthy patients and has shown in preclinical studies that it can slow and even shrink tumors.
You can read about the proposed Merck-FLX trial here: https://bit.ly/2RHpEJh.
Sponsors, CRO Ask FDA How to Un-Blind Blood Cancer Trials
Drug companies and a CRO are urging the FDA to sharpen its recommendations on how to reveal to blood cancer patients whether they’re taking placebos or the real thing in clinical trials.
The agency in August issued draft guidance suggesting that blood cancer patients be told if they’re taking experimental drugs or placebos and their disease comes back or worsens. It also said sponsors should alert patients and researchers if they’re worried meds are causing bad reactions.
Bristol-Myers Squibb in comments asked the FDA to allow un-blinded patients to drop out of a trial, but let sponsors keep their data. Novartis, on the other hand, said patients should only be told about an experimental drug if it causes a serious (not milder) adverse reaction, as the original draft suggests.
AstraZeneca believes there should also be guidelines on un-blinding the sponsor, suggesting drug v. placebo only be unveiled if cancer recurrence or progression can be documented “by objective measurements.”
Syneos Health said patients should only be let in on a drug trial’s secrets if unmasking them is necessary for their care. It also splits with AstraZeneca on sponsor intel, noting that “care should be taken within the un-blinding process to limit sponsor access to treatment assignment.”
You can read comments on the FDA’s draft guidance here: https://bit.ly/2zAE4mX.
FDA Final Guidance on Trial Design for Hypertension Drugs
The FDA says sponsors should conduct a single Phase III randomized clinical trial to determine if combinations of previously approved blood pressure meds are effective.
The trial should include participants identified as likely to benefit from two-drug therapy to keep a lid on high blood pressure, the agency says in final guidance released last week.
The FDA says sponsors can consider the combo therapy to be effective if it significantly improves patients’ systolic and diastolic blood pressure. That’s a switch from its suggestion in draft guidance for just one of the measures — or their mean — to be used as a primary endpoint.
Read the final guidance here: https://bit.ly/2z0TIZn.
EMA Promotes Novel Therapies
The European Medicines Agency (EMA) is set to release draft guidelines for sponsors by the end of the year designed to increase patient access to novel therapies through enrollment in clinical trials.
“The EU is committed to support the development of [advanced therapy medicinal products] and will keep monitoring developments in the field to ensure that the regulatory framework supports — and not hinders — the development” of biologics and the opportunity for patients to use them, the agency said last week in releasing an updated action plan to develop ATMPs.
Read the full action plan here: https://bit.ly/2D8w7bS.
FDA Redesigns Web Page for Expanded Access to Clinical Trials
The FDA’s web page for expanded access to clinical trials has a new look.
The tweaked website makes it even more user-friendly, FDA Commissioner Scott Gottlieb said in a statement last week announcing the redesign.
The FDA says it has granted 9,000 patients access to trial meds over the past five years, greenlighting more than 99 percent of applications it has received for expanded access.
Nonetheless, the agency has found itself hard-pressed by patient groups who say it’s still not enough.
Earlier this year, President Trump signed “right to try” legislation, which patient advocates say will cut out some of the FDA’s red tape and help get desperate patients into trials. The legislation protects drug sponsors from liability lawsuits over experimental drugs given to patients who wouldn’t otherwise qualify for a trial.
Read the new site here: https://bit.ly/2xNlVVv.
China Begins Brain Cancer Trials
Chinese scientists say they’re ready to begin phase I clinical trials on a gene inhibitor they hope will help treat a rare and deadly brain cancer.
Researchers recently found that the proposed treatment can penetrate the blood-brain barrier — a critical obstacle to successfully treating brain cancer. PLB-1001 appears to target a rare genetic mutation that’s been discovered in 14 percent of patients who suffer from sGBM, which affects up to 5 million people worldwide every year.
The five-year survival rate of sGBM patients is less than 10 percent.
The trials, which haven’t formally opened yet, will recruit sGBM patients who test positive for the dangerous mutations.