Foundation Medicine Launches FoundationOne Liquid Cancer Biopsy Test

– FoundationOne Liquid analyzes 70 genes known to drive cancer growth and reports microsatellite instability high (MSI-high) status to inform the use of precision oncology treatments, including immunotherapies, with a simple blood draw –

 Foundation Medicine, Inc. today announced that FoundationOne^®Liquid, its next-generation liquid biopsy test for solid tumors, is commercially available in the United States. Using a blood sample, FoundationOne Liquid analyzes 70 genes known to drive cancer growth, including homologous recombination deficiency (HRD) genes, and reports the genomic biomarker for microsatellite instability (MSI),¹ to help inform the use of checkpoint inhibitor immunotherapies and multiple targeted therapies, including poly (ADP-ribose) polymerase (PARP) inhibitors, as well as clinical trials for patients with advanced cancer.

“With the commercial launch of FoundationOne Liquid, we are further expanding access to important genomic information that has the potential to match more patients to targeted therapies,” said Tom Civik, Chief Commercial Officer at Foundation Medicine. “For many cancer patients, traditional tissue testing is not feasible, and there is a pressing need for minimally invasive solutions to help inform personalized treatment decisions. FoundationOne Liquid meets our highest standards for sensitivity and analytical validation and offers providers additional insights to help guide treatment options, including immunotherapies and PARP inhibitors. In addition to the clinical advancements this test provides, it will also help our biopharma partners improve trial design and accelerate drug development.”

FoundationOne Liquid is a hybrid capture-based, next-generation sequencing in vitro diagnostic device for the detection of substitutions, insertion and deletion alterations (indels), copy number alterations (CNAs) and select gene rearrangements using circulating cell-free DNA (cfDNA) isolated from plasma derived from peripheral whole blood. The FoundationOne Liquid test expands upon the previous version of the Company’s liquid biopsy test, FoundationACT^®, which has been analytically validated across the four main classes of genomic alterations. Evaluation of the platform using multiple validation methods across a broad range of tumor types demonstrated high sensitivity² and positive predictive value³, even at the low allele frequencies often observed in clinical samples.⁴

https://www.biospace.com/article/releases/foundation-medicine-introduces-foundationone-liquid-the-latest-advance-in-the-company-s-liquid-biopsy-test-for-solid-tumors-in-patients-with-advanced-cancer/

Canopy Growth CEO Talks Tilray’s ‘Technically Weird’ Price Action

The extreme volatility in shares of Tilray Inc TLRY continued Friday, but it could be a result of the limited supply of available shares, according to Canopy Growth Corp CGC CEO Bruce Linton.

What Happened

Tilray’s volatility is likely the result of investors and traders “trying to play a game” with large share positions, Linton told CNBC Friday. For instance, one trader who initiates a 2-million share position would account for a sizable portion of the total daily trading volume and result in the “pretty weird” trading activity seen over the past few days, he said.

Tilray’s production assets might be around 5 percent of what Canopy Growth produces, so it is unlikely the stock’s surge to $300 is “about that company,” the executive said.

It just got “technically weird,” Linton said.

‘Create Things That Don’t Exist’

The cannabis-infused beverage industry will include alcohol alternatives that give the user a “euphoric” sensation, while other beverages will focus on pharmaceutical benefits, the CEO said. Perhaps more important to the cannabis sector, Canopy Growth is in a position to “create things that don’t exist now,” he said.

“Canada is this platform where you can create intellectual property, you can run medical trials, you can file patents without any hesitation — including into the U.S.”

https://www.benzinga.com/markets/cannabis/18/09/12389714/canopy-growth-ceo-talks-tilrays-technically-weird-price-action

Argenx data concerns over rescue therapy unfounded, says Piper Jaffray

Argenx last week reported positive Phase II immune thrombocytopenia data on efgartigimod showing clean safety and clear efficacy, Piper Jaffray analyst Edward Tenthoff tells investors in a research note. He points out that twelve, or 46%, efgartigimod-treated patients achieved clinically meaningful platelet improvement. The analyst views concern over rescue therapy and bleeding events as unfounded and reiterates an Overweight rating on Argenx shares with a $154 price target.
https://thefly.com/landingPageNews.php?id=2794139

FDA Action Alert: Merck, Insmed and Antares Pharma

The U.S. Food and Drug Administration (FDA) has a few target action dates scheduled for this week, including one for Sunday, September 23, which was approved in late August. Let’s take a look.

The FDA gave Merck & Co. a PDUFA target action date of September 23 for its supplemental Biologics License Application (sBLA) for its anti-PD-1 drug Keytruda in combination with pemetrexed (Alimta) and platinum chemotherapy (carboplatin or cisplatin) as a first-line treatment for patients with metastatic nonsquamous non-small cell lung cancer (NSCLC). This was evaluated under Priority Review. The agency approved it for this indication and combination on August 20, 2018.

The supplemental application was based on overall survival (OS) and progression-free survival (PFS) data from the Phase III KEYNOTE-189 clinical trial.

“Keytruda is rapidly becoming a foundation for the treatment of appropriate patients with metastatic non-small cell lung cancer,” said Roger M. Perlmutter, president, Merck Research Laboratories, in a statement in August. “Today’s approval for the expanded label for Keytruda based on data from the KEYNOTE-189 trial is an important milestone, and reinforces our steadfast commitment to improving survival outcomes, and providing hope, for more patients with lung cancer.”

Bridge Water, NJ-based Insmed Incorporated has a target action date of Friday, September 28 for its New Drug Application (NDA) for ALIS (Amikacin Liposome Inhalation Suspension) for adults with nontuberculous mycobacterial (NTM) lung disease caused by Mycobacterium avium complex (MAC). It was granted Priority Review.

NTM is a rare and serious lung disease, which includes multiple symptoms like fever, weight loss, cough, lack of appetite, night sweats, bloody sputum, and fatigue. Though rare, its prevalence increased by about 8 percent a year from 1997 to 2007.

On August 7, the FDA’s Antimicrobial Drugs Advisory Committee voted 12 to 2 in favor of ALIS, as well as in favor of the surrogate endpoint of sputum culture conversion that was used in the Phase III CONVERT trial. However, in a separate vote, the committee voted against the safety and effectiveness of the drug in the broadest population of adults with NTM lung disease caused by MAC.

“We are very pleased by the outcome of today’s advisory committee meeting, which recognized the role ALIS may be able to play in addressing the significant unmet medical need among patients suffering from NTM lung disease caused by MAC, a chronic, debilitating and potentially fatal infection,” stated Will Lewis, Insmed’s president and chief executive officer.

Antares Pharma, based in Ewing, NJ, has a target action date of Saturday, September 29 for its investigational new drug for testosterone replacement therapy, Xyosted. The date was set in April, after the company resubmitted the drug application on March 29, 2018 after receiving a Complete Response Letter from the FDA.

The agency originally rejected the company’s NDA for Xyosted in October 2017. The CRL specified two deficiencies in the clinical data. The FDA indicated they were concerned the drug could cause a clinically problematic increase in blood pressure, as well as possible occurrence of depression and suicidality. There were no issues cited related to manufacturing or efficacy. As part of its resubmission, the company re-analyzed the data, but was not required to conduct additional clinical trials.

At the company’s second-quarter report on August 7, Robert F. Apple, company president and chief executive officer, said in a statement, “The recent launch of AMAG’s Makena auto injector product, the addition of a rescue pen development program to our business alliance pipeline and the potential for a late third quarter approval of our proprietary product Xyosted should continue to drive additional increases in revenue going forward….We are also focused on Xyosted launch planning as we continue to identify highly experienced sales representatives and stand ready to bring them on board contingent upon product approval on the September 29, 2018 target action date.”

https://www.biospace.com/article/fd1a-fda-action-alert-merck-insed-and-antares-pharma/

Some Cancer Cells Hide From Immunotherapy Treatments

Some cancer cells have a remarkable and frightening sense of self-preservation to avoid immunotherapy treatments.

That is the finding from a new study conducted by researchers at the Fred Hutchinson Cancer Research Center in Seattle. The study results, published in Nature Communications, took a close look at why immunotherapies do not always provide lasting treatments for some cancer patients.

The study focused on Merkel cell carcinoma patients and looked at why those patients who had been treated with immunotherapy would sometimes their cancers shrink at first but then come back. For the study, the Fred Hutchison researchers used a new technology that looked at how cancer cells change under the pressure of immunotherapy treatments.

Merkel cell carcinoma is a rare skin cancer caused by a common virus. About 20 percent of Merkel cell patients have an initial positive response to immunotherapy treatment but then relapse. It has been unclear why, Dr. Kelly Paulson, senior fellow at Fred Hutchison said.

By answering the question of “why,” Paulson said it will allow scientists to develop immunotherapies that can get toward long-term tumor control to make cancer a more chronic disease.

“Cancer is really tricky. We’re just starting to learn how tricky it is,” Paulson told BioSpace ahead of the study’s publication.

With the rise of immunotherapies like checkpoint inhibitors, Paulson said it was an exciting time to attempt to harness the body’s immune system in order to take out the cancer without affecting other cells in the microenvironment. Since the checkpoint inhibitors came onto market, she said they have been effective in certain types of cancer. She said with some melanomas, the checkpoint inhibitors have even eradicated some cases of metastatic cancer.

“There’s potential for the immune system to cure cancer. But, it’s challenging because not everybody gets that cure,” she said.

In the case of the Fred Hutchison study, the researchers studied the cancer cells from two patients Merkel cell carcinoma patients who initially responded to therapy, but saw their cancers return. The two patients were treated with a combination of a checkpoint inhibitor and a T-cell therapy that involved removing the cells from patients, multiplying them in a lab and then infusing them back into the patients. The T-cells were not engineered when they had been removed.

The patients responded well at first. Paulson said biopsies showed the T-cells got into the cancers and facilitated the tumor regression.

But then the cancer returned in both of them. In one patient, it was metastatic.

Paulson and colleagues used single-cell RNA sequencing to investigate the molecular changes in T cells, tumor cells and all other cells in the tumor microenvironment to see what had changed in the patients. And what they learned was both fascinating and a bit scary. They saw that the tumor was hiding from the T-cells, Paulson said.

“It was really quite tricky how the cancer came back.”

The tumor was able to accomplish this by removing one piece of human leukocyte antigens (HLA), a gene trio that allows T-cells to find the cancer cells. The single-cell RNA sequencing showed one piece of the HLA trip had been hidden by the tumor and that allowed the cancer cells to return without the T-cells noticing.

With that new knowledge in hand, Paulson and colleagues returned to the lab. They cultured cells from one of the patients and applied drugs already used clinically for other cancers that turned the cancer marker back on. The results from the in-vitro study will potentially be able to help other cancer patients who relapse following immunotherapy treatment.

Paulson said what she has learned is that a multi-pronged approach is the best approach to treating cancer than relying on a single treatment.

Paulson said they have known that other cancers have tricks to avoid destruction, but to her knowledge, this is the first time this particular trick with Merkel cell carcinoma has been reported.

“It’s a new variation on a theme that’s been known,” she said.

With the Fred Hutchison research now published, Paulson said it will likely lead to many questions about other cancers and their responses to immunotherapies. Paulson said researchers can begin to look hard at immunotherapy resistance across cancer settings in order to understand how cancer comes back after immunotherapy and why.

https://www.biospace.com/article/some-cancer-cells-hide-from-immunotherapy-treatments-study-finds/

RELX upgraded to Overweight from Equal Weight at Barclays

Barclays analyst Nick Dempsey upgraded RELX to Overweight and raised his price target for the shares to GBP 17.40 from GBP 17.10. The analyst views the current valuation as attractive.
https://thefly.com/landingPageNews.php?id=2794141

Amarin could have blockbuster after ‘stunning’ data, STAT’s Feuerstein says

https://thefly.com/landingPageNews.php?id=2794145