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Sunday, January 12, 2020

Agios sees Tibsovo sales as high as $115M this year

Ahead of its JPM20 presentation, Agios Pharmaceuticals (NASDAQ:AGIOannounces its key milestones and outlook for 2020. Highlights:
Tibsovo (ivosidenib) sales: $105M – 115M.
Receive CHMP opinion Europe on Tibsovo for relapsed/refractory AML with IDH1 mutation by year-end.
Complete enrollment in Phase 3 study, AGILE, evaluating the combination of Tibsovo and azacitidine in treatment-naïve IDH1-positive blood cancers by year-end.
File supplemental marketing application in U.S. by year-end for Tibsovo in previously-treated IDH1-positive cholangiocarcinoma.
Release topline data from ACTIVATE and ACTIVATE-T studies of mitapivat in adults with pyruvate kinase deficiency by year-end.
Achieve proof-of-concept for mitapivat in sickle cell disease by mid-year.
#JPM20

Teladoc in $600M deal for InTouch Health

InTouch Health provides an integrated suite of technology, software, purpose-built devices and a secure global network for global health care providers, according to the Teladoc (NYSE:TDOC) press release. Revenues for 2019 are expected to foot to about $80M.
Terms: $150M in cash and $450M in Teladoc common shares. The deal is seen closing in Q2.
Teladoc presents at #JPM20 tomorrow at 12 ET.

Moderna expands pipeline after accumulation of positive data

Citing positive data from six early-stage studies in its infectious disease portfolio that, it says, builds on the clinical validation of systemic delivery of mRNA, Moderna (NASDAQ:MRNA) has added two programs to its pipeline.
Candidate mRNA-6231: encodes a long-acting selective IL-2 to preferentially expand regulatory T cells that suppress immune activity in autoimmune diseases.
Candidate mRNA-6981: encodes PD-L1 to treat autoimmune disease, initially to be developed in autoimmune hepatitis.
The company has 21 mRNA candidates in its pipeline, including 13 in clinical trials.
#JPM20

Invitae sees 2020 sales as high as $330M

Ahead of its JPM20 presentation, Invitae (NYSE:NVTAannounces preliminary 2019 results and its 2020 outlook.
2019: Revenue: ~$216M (+45%). Quick assets at year-end: ~$400M.
More than 482K (+60%) samples accessioned.
3,500 (+71%) new accounts added.
Final results will be released next month.
2020 guidance: Revenue: at least $330M. Test volume: at least 725K.
#JPM20

Zymeworks teams up with Pfizer in breast cancer study

Zymeworks (NYSE:ZYME) inks a collaboration agreement with Pfizer (NYSE:PFE) aimed at advancing a Phase 2 clinical trial evaluating the combination of lead candidate ZW25, Ibrance (palbociclib) and fulvestrant [AstraZeneca’s (NYSE:AZN) Faslodex] in patients with previously-treated locally advanced/metastatic HER2-positive, HR-positive breast cancer.
Zymeworks is sponsoring the study while Pfizer is supplying Ibrance.
ZW25 is is an HER2-targeted bispecific antibody developed with the company’s Azymetric platform.

Bristol’s Merger With Celgene Is Already Paying Dividends: CEO

Bristol-Myers Squibb Co., fresh off completing one of the largest drug-industry mergers ever, is counting on the acquisition to pay off quickly with new products and ease Wall Street concerns.
Bristol waited nearly 11 months for its controversial $74 billion deal for Celgene to close, in November. During that span, U.S. regulators approved two cancer drugs from Celgene, and a third, for treating multiple sclerosis, could get the go-ahead by March and eventually provide what analysts see as $3 billion in annual sales.
The product additions are timely for the combined company, which is facing patent expirations on some of its big-selling therapies.
“I feel better today than I felt even the day we closed,” Bristol Chief Executive Giovanni Caforio said in an interview. “The power of the innovation engine of the new company is becoming very clear.”
The merger aimed to create a cancer-drug powerhouse with eight products on the market, each generating more than $1 billion in annual sales. Yet investors had their doubts. Bristol shares fell 14% the day of the announcement, and an activist-investor campaign led by hedge fund Starboard Value LP ensued to upend the deal. A handful of other shareholders also expressed displeasure, including the fifth-largest shareholder, Dodge & Cox, The Wall Street Journal reported.
Among Wall Street’s concerns: The two drugmakers’ biggest-selling products faced the prospect of falling sales. Bristol’s Opdivo cancer therapy was losing ground to Merck & Co.’s Keytruda, while Celgene’s Revlimid multiple-myeloma treatment is expected to confront copies in 2022.
In addition, antitrust regulators required that Bristol slough off Celgene’s lucrative psoriasis therapy Otezla, which ultimately went to Amgen Inc. for $13.4 billion.
Now, after initial skepticism, investors appear to be coming around. Bristol shares rose more than 26% in the last three months of 2019, compared with an 11% gain for the NYSE Arca Pharmaceutical Index and an 8.5% uptick for the S&P 500.
“Every single decision, commercially and in the pipeline, looks like it broke in Celgene’s favor,” said Ronny Gal, an analyst at Sanford C. Bernstein & Co.
But analysts also warn that it isn’t all clear sailing for the expanded company. They cite weaker Opdivo sales, intense competition in the cancer-drug market and the challenge to Bristol management of digesting a company as large as Celgene while shepherding drug candidates to approval.
There also are questions about growth several years out, said Tim Anderson, an analyst at Wolfe Research.
“Where is that solution going to come from?” Dr. Anderson said. “It has to be more than what we’re seeing today in the phase-three pipeline. Are there interesting assets on the earlier side of the Celgene pipeline?”
Bristol plans to launch six new drugs over the next two years, said Dr. Caforio, who became CEO in 2015.
He said the company is prepared for the decline in Revlimid sales, which he described as “more of a slope than a cliff.” He also said Opdivo revenue should resume growth in 2021 if the drug wins additional approvals.
In the near term, Dr. Caforio said, the New York-based company will draw growth from legacy products. Those could include Revlimid, which notched $8.1 billion in sales through the first nine months last year, and the blood-thinner Eliquis, which generated $5.9 billion during the same period.
Dr. Caforio said Bristol will look to do smaller acquisitions but probably hold off on larger deals for a couple of years until it has trimmed the $39 billion debt incurred in acquiring Celgene.
Bristol pioneered the development of cancer agents known as immunotherapies, which unleash the body’s immune system on tumors. It now sells two such drugs, Opdivo and Yervoy, which treat cancers including skin and lung.
But the company lost its advantage in the lucrative lung-cancer market to rival Merck, a setback that diminished sales prospects and sent its shares falling.
Meantime, Celgene has transformed the treatment of multiple myeloma starting with the repurposing of the Thalidomide sleeping pill — known for its history of causing birth defects — into Revlimid.
The recent pipeline progress is helping the combined company move past its merger travails. An anemia drug from Celgene called Reblozyl, which U.S. regulators approved last year, is projected by JPMorgan Chase to exceed $1 billion in annual sales.
The Food and Drug Administration has said it would make a decision by March on ozanimod, a multiple-sclerosis therapy that analysts expect to be a multibillion-dollar seller. Bristol is also testing the drug in patients with ulcerative colitis.
Also up for approval is a cellular therapy targeting leukemia that analysts say could surpass $1 billion in yearly sales. Approval of the so-called CAR-T drug, known as liso-cel, would help Bristol remain a big player in the blood-cancer market after Revlimid copies become available.
The company said last month that another CAR-T treatment, in development with Bluebird Bio Inc., was shown to be effective and safe in multiple myeloma patients. A regulatory approval filing is expected this year, Bristol has said.

Reduce Health Costs By Nurturing The Sickest? Much-Touted Idea Disappoints

Improving health and lowering costs for the sickest and most expensive patients in America is a dream harder to realize than many health care leaders had hoped, according to a study published Wednesday by the New England Journal of Medicine.
Researchers tested whether pairing frequently hospitalized patients in Camden, New Jersey, with nurses and social workers could stop that costly cycle of readmissions. The study found no effect: Patients receiving extra support were just as likely to return to the hospital within 180 days as those not receiving that help.
The results are a blow to Dr. Jeffrey Brenner and the Camden Coalition of Healthcare Providers, the organization he founded nearly 20 years ago.
“It’s my life’s work. So, of course, you’re upset and sad,” said Brenner, who now does similar work with health insurance giant UnitedHealthcare.
The model of care, pioneered in part by Brenner and profiled in a widely read 2011 article in The New Yorker, has inspired dozens of similar projects across the country and attracted millions in philanthropic funding.
“This is the messy thing about science,” said Brenner, who won a MacArthur Foundation “Genius Grant” for his efforts. “Sometimes things work the way you want them to do work and sometimes they don’t.”
The Hope
Many hospital and insurance executives have pinned their hopes on this work because it promised to solve a common problem: when patients lives are so complicated by social factors like poverty and addiction that their manageable medical conditions, like diabetes and asthma, lead to expensive, recurring hospital stays.
Writer and physician Atul Gawande introduced Brenner as a brash visionary crusading on behalf of the “worst-of-the-worst patients” in the New Yorker piece, titled “The Hot Spotters.” (Gawande, who now heads Haven, a joint venture of Amazon, Berkshire Hathaway and JPMorgan Chase, declined to be interviewed for this article.)
Brenner’s prescription: Pair these people with front-line care workers who would shepherd them to the social and medical services they needed. Early evidence was promising, the anecdotes inspiring. Brenner boiled the model’s potential down to four words and two tantalizing goals: better care, lower costs.
As word spread, breathless headlines popped up like “Health Care’s Best Hope” and “Could Camden Coalition Save US Healthcare?
“Lots of organizations make claims that their programs work and they’ve never been rigorously tested,” Brenner said.
Instead, Brenner took the unusual step of inviting the scrutiny of respected researchers.
In 2014, Massachusetts Institute of Technology economist Amy Finkelstein began a randomized controlled trial, the same rigorous method used to evaluate new drugs. Over four years, the coalition enrolled 800 patients, all who had been recently hospitalized and struggled with social problems. Half received the usual care patients get when leaving the hospital. The other half got about 90 days of intensive social and medical assistance from the coalition.
And the result: The 400 patients who received the intensive help were just as likely to return to the hospital as the patients who didn’t. In both groups, nearly two-thirds of people were readmitted within 180 days.
So why did the coalition fail? Why did the savings touted in their early data, which Gawande had declared “revolutionary” in The New Yorker nearly a decade ago, disappear when put to this rigorous test?
‘My Daily Routine’
Larry Moore, who has hypertension, alcohol addiction, chronic seizures and difficulty walking, was one of the first people to enroll in the coalition’s trial.
Moore’s experience serves as a road map for understanding why the coalition missed its mark.
His first months were promising: prescriptions filled, medical appointments attended, Social Security benefits claim in process. The 47-year-old even started to trust the team with the details of his deep-seated addiction, confiding how he would consume mouthwash, vanilla extract and even hand sanitizer at times.
“You couldn’t keep anything with alcohol in it” around him, Moore recalled. “That’s addiction.”
But all the progress suddenly stopped when Moore seemed to disappear from the coalition’s radar.
“We didn’t see Mr. Moore after November,” said nurse Jeneen Skinner. “We went to the house. We sent text messages. We [made] phone calls.”
The coalition has learned that for people living in poverty and with poor health, a small hiccup — in Moore’s case, a missed rent payment — can spiral into a major setback.
Moore spent the next 2½ years mostly homeless, completely out of touch with the coalition.
“I was going from place to place. I sleep on a bench or a rock until the next day when the liquor store opens,” remembered Moore. “That was my daily routine.”
Seventy emergency room visits and six hospital admissions later, Skinner reconnected with Moore.
He told her the one thing that would keep him out of the hospital: housing.
The ‘Camden Coalition’
Too many times, during the trial, people ended up back in the hospital despite the intervention. But the coalition is convinced it didn’t fail as much as the larger social safety net did.
“The bottom line is, we built a brilliant intervention to navigate people to nowhere,” said Brenner.
Coalition staff and their patients usually knew what was needed — evidence-based addiction treatment, housing, mental health services — but resources were often in short supply.
Over the past three years, the coalition set out to fill in those gaps, undergoing a kind of metamorphosis.
“We think of ourselves now as the Camden Coalition” steering away from the “health care providers” part of the name, said Kathleen Noonan, who succeeded Brenner as head of the organization.
It has forged partnerships with jails, lawyers and legislators, and started its own housing program. Many of these efforts began as the clinical trial was ongoing — a sign the coalition had seen the writing on the wall.
The Camden Coalition helped Moore get an apartment. (DAN GORENSTEIN/TRADEOFFS)
Moore says it took him a month to sleep in his bed, after years of intermittent homelessness.(DAN GORENSTEIN/TRADEOFFS)
‘I Kid You Not’
“I would have never imagined this,” said Moore, sweeping his arm around his one-bedroom apartment.
A green houseplant sits in the sunshine. A fluorescent-colored stuffed animal decorates the bed.
“When I first moved in here,” Moore explained, “it took me about a month to even sleep in my bed. I slept on a couch.”
Housing made it easier to face his other problem, choosing to try the drug naltrexone, a long-acting injection to treat alcohol addiction.
Moore is nearly two years sober today. He meets with a coalition support group on Wednesdays. He’s becoming a deacon at his church. In the 22 months he has lived in the apartment, Moore’s trips to the hospital have plummeted: just one admission and one ER visit.
“I kid you not, when I saw Mr. Moore probably a month ago, I was standing next to him and did not recognize him,” said nurse Skinner. “He looked at me, and said, ‘Jeneen, it’s me. And I was like, ‘My God, you look amazing!’”
Larry Moore’s story is just that: one story.
Yet it represents a larger trend. Insurers — including UnitedHealthcare under Brenner’s direction — hospitals and many state Medicaid agencies have begun spending millions to meet patients’ social needs.
The new study, though, backs up the skepticism of other researchers that, when it comes to saving money at least, these approaches don’t work well. For one, programs are expensive and hard to scale. The coalition’s housing effort currently serves only 50 people and costs about $14,000 per person per year. Secondly, the data is lacking, cautioned Boston University economist Austin Frakt. “Despite what people would like to believe, there’s not a lot of evidence you can reduce health spending by spending more in other areas.”
Finkelstein said that as health care companies move further beyond the four walls of a hospital, the need for rigorous evaluation grows. “I think a lot of well-intentioned people in health care can’t handle the truth,” she said. “They’re trying to do good, but they don’t have the courage to say, ‘Let’s do a gut check on ourselves.’”