Target $25.
Monday, June 3, 2019
Natera Test Detects Immunotherapy Response in Metastatic Cancer: ASCO
A new study demonstrates the ability of Natera’s Signatera™ test to assess patient response to immunotherapy in the metastatic setting across multiple cancer types by detecting molecular traces of circulating tumor DNA (ctDNA) in the blood. The study was presented at the American Society of Clinical Oncology (ASCO) Annual Meeting in Chicago on June 1, 2019.
The study, conducted by Princess Margaret Cancer Centre in Toronto, enrolled 70 patients with advanced cancer, including head and neck, triple-negative breast, melanoma, and ovarian cancers. Patients were treated with the single-agent immune checkpoint inhibitor pembrolizumab as part of the phase II INSPIRE trial (NCT02644396). The study used Signatera to assess ctDNA at baseline and again at the start of the third treatment cycle.
Results showed a strong correlation between changes in ctDNA and overall survival (adjusted HR=0.38, p=0.004), progression-free survival (adjusted HR=0.47, p=0.006), and overall clinical response rate, illustrating that ctDNA may be a valuable predictive biomarker for patients with mixed solid tumors treated with checkpoint inhibitors. Signatera, a personalized, tumor-informed blood test, detected ctDNA in 68 out of 70 patients at baseline (97 percent sensitivity).1
The study (abstract 2542), titled “Bespoke Circulating Tumor DNA (ctDNA) Analysis as a Predictive Biomarker in Solid Tumor Patients (pts) Treated With Single Agent Pembrolizumab (P),” can be accessed here.
Forty Seven at ASCO with snapshot of promising CD47 data — and $115M IPO
Here’s a neat trick for up-and-coming oncology biotechs to consider.
Execs for Forty Seven, the CD47 star founded by Stanford’s legendary Irv Weissman, filed for a $115 million IPO on Friday and then — instead of hunkering down for the quiet period — promptly flew out to Chicago to tout a promising set of proof-of-concept data for their lead drug at ASCO.
Execs for Forty Seven, the CD47 star founded by Stanford’s legendary Irv Weissman, filed for a $115 million IPO on Friday and then — instead of hunkering down for the quiet period — promptly flew out to Chicago to tout a promising set of proof-of-concept data for their lead drug at ASCO.
Forty Seven has preliminary data on 22 patients taking 5F9, with an impressive initial snapshot of early data for the small group. Fifteen of the patients suffer from treatment resistant diffuse large B-cell lymphoma (DLBCL) and the other 7 have follicular lymphoma. In DLBCL investigators tracked an objective response rate of 40% with a third achieving a complete response; in follicular lymphoma the ORR was 71% with 43% achieving a CR.
It’s still too early to set a median rate on the duration of response, but the researchers say that just 1 of the 22 saw their cancer progress after 6 months.
5F9 is an antibody that targets the popular CD47 receptor targets, which has inspired a slate of development efforts. Hitting that target is intended to scramble the “don’t-eat-me” signal that cancer cells rely on to avoid being chewed up by macrophages. And the Menlo Park, CA-based biotech has 6 studies underway in solid tumors, acute myeloid leukemia, non-Hodgkin’s lymphoma and colorectal carcinoma.
It’s still too early to set a median rate on the duration of response, but the researchers say that just 1 of the 22 saw their cancer progress after 6 months.
5F9 is an antibody that targets the popular CD47 receptor targets, which has inspired a slate of development efforts. Hitting that target is intended to scramble the “don’t-eat-me” signal that cancer cells rely on to avoid being chewed up by macrophages. And the Menlo Park, CA-based biotech has 6 studies underway in solid tumors, acute myeloid leukemia, non-Hodgkin’s lymphoma and colorectal carcinoma.
The biotech plans to push its studies ahead as a mono therapy as well as in combinations with PD-1/PD-L1 and CTLA-4 checkpoint inhibitors, starting with Genentech’s Tecentriq.
Forty Seven has an interesting past. The 78-year-old Weissman was able to wrangle substantial support for his early research work on CD47 from the California Institute for Regenerative Medicine, even launching early human studies — a rare feat in academic circles. Weissman and former CIRM chief Alan Trounson enjoyed a tight relationship, which extended to Trounson’s appointment to the board of another startup that Weissman had helped found — StemCells —shortly after his departure from the agency. And Forty Seven is still getting money from CIRM under its latest $19 million grant.
Forty Seven has an interesting past. The 78-year-old Weissman was able to wrangle substantial support for his early research work on CD47 from the California Institute for Regenerative Medicine, even launching early human studies — a rare feat in academic circles. Weissman and former CIRM chief Alan Trounson enjoyed a tight relationship, which extended to Trounson’s appointment to the board of another startup that Weissman had helped found — StemCells —shortly after his departure from the agency. And Forty Seven is still getting money from CIRM under its latest $19 million grant.
So far the company has raised $149 million and spent $84 million of that, according to the S-1.
Mark McCamish, a Novartis vet who enjoyed a $3.7 million pay package last year, is the CEO. He also has 3.6% of the stock. Lightspeed Ventures Partners and Sutter Hill Partners each control 16.8% of the stock, followed by Clarus at 15.8%. And Weissman has retained a hefty 9.5% of the equity, which could soon be worth a small fortune.
Forty Seven includes the following companies on its list of rivals in the field: Celgene Corporation, Trillium Therapeutics, Alexo Therapeutics, Arch Therapeutics, Surface Oncology, Novimmune, OSE Immunotherapeutics and Aurigene Discovery Technologies.
Ironwood, Allergan Start Phase 2 for Non-opioid IBS Pain Med
Ironwood Pharmaceuticals, Inc. (NASDAQ:IRWD), a GI-focused healthcare company, today announced the initiation of patient dosing in a Phase II clinical trial evaluating MD-7246 in patients with abdominal pain associated with irritable bowel syndrome with diarrhea (IBS-D). Data from the Phase II trial are expected in the second half of 2020.
Ironwood and Allergan are evaluating MD-7246, an investigational new product, as an oral, intestinal, non-opioid, pain-relieving agent for patients suffering from abdominal pain associated with certain GI diseases. MD-7246 is a delayed-release formulation of linaclotide designed to provide targeted delivery of linaclotide to the colon, where the majority of the abdominal pain associated with IBS-D is believed to originate, and to limit fluid secretion in the small intestine resulting in minimal impact on bowel function.
“Abdominal pain is a dominant symptom experienced by approximately 16 million people in the U.S. living with IBS-D, and there are a limited number of treatment options for patients seeking effective pain relief,” said Michael Shetzline, M.D., Ph.D., chief medical officer, senior vice president and head of drug development at Ironwood. “Through its delayed release and differentiated mechanism of action, we believe that MD-7246 has the potential to target abdominal pain in patients with IBS-D and look forward to advancing it as a potential abdominal pain-relieving agent for this population. If successful, we hope to explore the broader potential of MD-7246 for the more than 50 million patients estimated to suffer from recurring abdominal pain across a broader range of GI disorders.”
TransMedics (TMDX) Reports Second FDA PMA Approval for OCS Lung System
TransMedics Group, Inc. (NASDAQ: TMDX) today announced that it has received an FDA PMA approval for expanded clinical indications.
The top 10 pharma R&D budgets in 2018
Big Pharma companies are still, naturally, the big spenders in drug R&D, and the numbers are impressive: Last year, and for the first time, the top 15 largest companies (by sales) funneled more than $100 billion into research, and we also saw the FDA approve more drugs than ever before.
Big Pharma should get a pat on the back, right? Well, yes and no: The FDA had something of a backlog of drugs from 2017 that helped budge 2018 into more approvals, and some of the ways they classified approvals also slightly flattered the numbers (see our report below).
The tally of more than 50 new molecular entities is an impressive one, and borne from the well-funded R&D engines of life science companies; but earlier this year, a new report by the IQVIA Institute for Human Data Science showed that in fact, large pharma companies (those with more than $10 billion in yearly drug sales) have seen their R&D share drop from 31% to just 20% in the past decade.
And when it comes truly new, innovative, exciting drugs in the pipeline, the report found that the smaller biotechs are punching well above their weight.
Last year, we saw 59 new therapies get the thumbs-up under the leadership of (now ex) FDA Commissioner Scott Gottlieb, M.D. But the report points out that large pharmas were the filing companies for fewer than half of these launches. Emerging biopharma companies, on the other hand, were the originators of 38 of the 59 therapies (64%).
The report says that the “importance of large pharma in originating molecules is decreasing,” but they “remain important partners” for biotech. Still, they see the need for smaller biopharmas to team up with Big Pharma companies—and their large sales teams—decreasing.
“The dynamics of development, M&A and licensing activity seem to be shifting, and emerging companies are retaining control of their assets to a greater degree,” the report’s authors wrote.
Coupled with this is the fact that, according to the index, emerging biopharma companies (those spending less than $200 million annually on R&D and having less than $500 million in sales) account for 72% of all late-stage pipeline activity, up from 61% a decade ago.
Roche, J&J, Novartis and the like are still the unmatched big boys when it comes to R&D budgets, with Roche once again coming out on top with a staggering $11 billion in R&D spend last year (though this also includes its diagnostics business).
The names and numbers don’t change much over the years (in fact, Roche, which has been at the top for a few years now, is down nearly half a billion in terms of R&D spend since 2016), but there is one big exception: Sanofi, which has penned several biotech deals and M&As, is up more than $1 billion in two years, from $5.45 billion in 2016 to $6.6 billion in 2018, as it attempts to get back into oncology, which it seemed to be walking away from.
Merck also deserves a shoutout: While its R&D budget was down from last year (when it spent $10.33 billion), its $9.75 billion spend has put it third, ahead of Novartis, in terms of overall spend, jumping from $7.19 billion, when it was the fifth highest Big Pharma R&D spender, in 2016. This coincides with now more than 1,000 trials for its cancer drug star Keytruda.
With more approvals and big topline numbers in R&D, it’s easy to think the big boys are doing all the heavy lifting, but as the IQVIA report found, don’t forget the smaller teams with supertight budgets that continue to outpace their Big Pharma rivals. — Ben Adams
Top 10 R&D budgets:
1.Roche
2.J&J
3.Merck
4.Novartis
5.Pfizer
6.Sanofi
7.Bristol-Myers Squibb
8.AstraZeneca
9.Eli Lilly
10.GlaxoSmithKline
Scripps study claims rebate system does inflate drug prices
The pharma industry has been claiming for years that the rebate system is a key factor in driving price increases in the US. Now, a study by Scripps Research provides evidence to support that view.
The non-profit scientific institute analysed tens of millions of insurance claims for the top 49 pharmaceutical brands in the US and found “continual, marked, annual increases” that drive up prices by a median of 76% between January 2012 and December 2017.
The study – published in JAMA Network Open – found that 78% of 36 drugs available over the entire study period saw an increase in insurer and out-of-pocket costs of more than 50%, while 44% more than doubled in price.
The main reason? The researchers from the Scripps Research Translational Institute – led by cardiologist and digital medicine authority Eric Topol – say it is in no small part the “current rebate system, which incentivises high list prices for drugs and relies heavily on privately-negotiated rebates to pharmacies.”
The current system is “byzantine and secretive”, they note, and prevents consumers from making informed decisions about purchasing medications.
In January, President Trump proposed a rule that would end the rebate system, a move which was lauded by CEOs from Novartis and Eli Lilly as well as other executives, who claim that pharmacy benefit managers (PBMs) and other “middlemen” in the US supply chain are pocketing discounts instead of passing them on to patients.
It’s not black and white, however. While some drug companies have defended price increases by reasoning that rebates have increased at a similar rate, the researchers found that is not the case. In fact, both increases in list prices and a greater reliance on rebates are making drugs more expensive overall, they suggest.
Scripps is also concerned about the close coordination of price increases among products that can be prescribed interchangeably, citing insulins and TNF inhibitors like AbbVie’s Humira (adalimumab) and Amgen’s Enbrel (etanercept), regardless of competition in the marketplace.
These were “highly synchronised in relative cost changes while demonstrating some of the largest cost increases in the industry over the past six years,” it says. The price of Humira, for instance, rose from $1,940 in January 2012, to $4,338 by December 2017.
Some US lawmakers have suggested that the close correlation between insulins from different manufacturers could suggest collusion, and led to demands that producers produce documentation to explain why the price of these drugs has skyrocketed in recent years.
“It’s bad enough to see the relentless increase in drug prices, but this work underscores it is occurring without transparency or accountability,” says Topol, the Institute. “It is especially concerning to see drugs in the same class having increases that appear to be coordinated.”
The Scripps team obtained the prescription data from a proprietary Blue Cross and Blue Shield data set that includes commercial insurance claims from more than 35 million Americans.
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