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Monday, October 14, 2019

Immuron up on Q1 2020 sales

Immuron (NASDAQ:IMRN) is up 17% premarket on announcing sales results for its OTC gastrointestinal and digestive health supplement Travelan, for Q1 2020 ended September 30, 2019.
Total North American sales for Travelan reached $269K AUD, up 111% YoY.
In U.S., Q1 sales grew to $232K AUD, up 81% YoY.
In Canada, Q1 sales reached $38K AUD.
Global Immuron sales expanded by +54% YoY, reaching $741K AUD.
In Australia, Travelan grew by 34%, achieving sales of $458K AUD in Q1.
https://seekingalpha.com/news/3505466-immuron-plus-17-percent-premarket-q1-2020-sales

FDA accepts Ultragenyx’s triheptanoin NDA for rare oxidation disorders

The FDA accepts for review Ultragenyx’s (NASDAQ:RARE) marketing application seeking approval of UX007 (triheptanoin) for the treatment of long-chain fatty acid oxidation disorders (LC-FAOD), a group of inherited diseases in which the body is unable to convert long-chain fatty acids into energy.
The agency’s action date is July 31, 2020. An advisory committee meeting is not planned.
UX007 is a purified pharmaceutical-grade form of triheptanoin, a triglyceride compound, that provides patients with medium-length odd-chain fatty acids which increase the intermediate substrates in the Krebs cycle, a key energy-generating process, which produces new glucose.
https://seekingalpha.com/news/3505469-fda-accepts-ultragenyxs-triheptanoin-nda-rare-oxidation-disorders

Premarket analyst action, Oct. 14

Allergan (NYSE:AGN) resumed with Market Perform rating and $180 (7% upside) price target at Bernstein.
Amgen (NASDAQ:AMGN) resumed with Outperform rating and $239 (19% upside) price target at Bernstein.
Biogen (NASDAQ:BIIB) resumed with Market Perform rating and $241 (8% upside) price target at Bernstein.
Celgene (NASDAQ:CELG) resumed with Market Perform rating and $100 (flat) price target at Bernstein.
Exagen (NASDAQ:XGN) initiated with Outperform rating and $17 (35% upside) price target at Cowen and Company. Initiated with Overweight rating and $18 price target at Cantor Fitzgerald. Initiated with Outperform rating at William Blair.
Gilead Sciences (NASDAQ:GILD) resumed with Outperform rating and $84 (31% upside) price target at Bernstein.
IGM Biosciences (NASDAQ:IGMS) initiated with Buy rating and $30 (74% upside) price target at Guggenheim. Initiated with Buy rating and $25 price target at Jefferies. Initiated with Buy rating and $32 price target at Stifel. Initiated with Outperform rating and $26 price target at Piper Jaffray.
Jazz Pharmaceuticals (NASDAQ:JAZZ) resumed with Market Perform rating and $150 (26% upside) price target at Bernstein.
Medical Properties Trust (NYSE:MPW) resumed with Neutral rating and $19.50 (flat) price target at BofA Merrill Lynch.
Mylan N.V. (NASDAQ:MYL) resumed with Market Perform rating and $21 (11% upside) price target at Bernstein.
Regeneron Pharmaceuticals (NASDAQ:REGN) resumed with Market Perform rating and $309 (3% upside) price target at Bernstein.
Sabra Health Care REIT (NASDAQ:SBRA) resumed with Neutral rating and $22 (3% downside risk) price target at BofA Merrill Lynch.
Teva Pharmaceutical Industries (NYSE:TEVA) resumed with Market Perform rating and $9 (30% upside) price target at Bernstein. Shares down 2% premarket.
Varian Medical Systems (NYSE:VAR) upgraded to Buy with a $130 (17% upside) price target at BTIG Research.
Canopy Growth (NYSE:CGC) downgraded to Neutral at Seaport Global Securities. Shares down a fraction premarket.
HEXO Corp. (NYSE:HEXO) downgraded to Neutral at Seaport. Shares down 5% premarket.
Insulet (NASDAQ:PODD) downgraded to Neutral at BTIG. Shares down 2% premarket.
Ra Pharmaceuticals (NASDAQ:RARX) downgraded to Hold with a $48 (3% upside) price target at SunTrust.
https://seekingalpha.com/news/3505457-bernstein-sees-19-percent-upside-amgen-premarket-analyst-action

Sunday, October 13, 2019

Biotech week ahead, Oct. 14

Biotech stocks continued to languish for a third straight week. Negative broader market sentiment and company-specific weakness weighed down on the sector.
Pfenex Inc PFNX 3.62% received FDA approval for its biosimilar to Amgen, Inc. AMGN 1.07%‘s Forteo, and Australian biotech Clinuvel Pharmaceuticals Ltd CLVLY 0.44%‘s Scenesse to treat erythropoietic protoporphyria was also given the go-ahead by the regulatory agency.
The week also saw the listing of two biotechs following their initial public offerings.
The following are some of the key catalysts for biotech stocks in the upcoming week.

Conferences

  • American Academy of Ophthalmology (AAO) 2019: Oct. 12-15 in San Francisco, California
  • International Congress And Exhibition on Endocrine And Diabetic Complications: Oct. 14-15 in Sydney, Australia
  • International Conference on Molecular Markers and Cancer Therapeutics: Oct. 14-15 in Abu Dhabi, UAE
  • Annual meeting of Neuro Critical Care Society: Oct. 15-18, in Vancouver
  • 10th Annual Congress on Rare Diseases and Orphan Drugs: Oct. 16-17 in Tokyo, Japan
  • 14th Annual Conference on Nephrology & Renal Care: Oct. 16-17 in Singapore City
  • 2nd International conference on blood disorders: Oct. 17-18 in Osaka, Japan

PDUFA Dates

Sunday
The FDA is expected to rule on Johnson & Johnson JNJ 1.83%‘s Janssen unit’s sNDA for Xarelto for the expanded indication of venous thromboembolism in medically ill patients.
Monday
Flexion Therapeutics Inc FLXN 0.91% awaits FDA verdict on its sNDA for Zilretta, which seeks approval of the drug for repeat administration to treat osteoarthritis of the knee.
Tuesday
The regulatory body will rule on Novartis AG NVS 0.83%‘s BLA for brolucizumab to treat wet-age related macular degeneration.
Friday
The FDA is likely to rule on Amgen, Inc. AMGN 1.07%‘s Nplate, which is being evaluated for treating adult patients with immune thrombocytopenia for 12 months or less.
Saturday
Clearside Biomedical Inc CLSD 6.21% awaits an FDA decision on Xipere to treat macular edema associated with uveitis. The company in August said it expects a complete response letter around the PDUFA date and also gave a schedule for the resubmission of the NDA.
Assertio Therapeutics Inc ASRT 1.64%‘s NDA for Cosyntropin that’s being evaluated as a diagnostic drug for detecting adrenocortical insufficiency is pending before the FDA, with a decision likely on Oct. 19.
The FDA will also rule on Alexion Pharmaceuticals, Inc. ALXN 0.92%‘s sBLA for ultomiris to treat atypical hemolytic uremic syndrome.

Clinical Trial Readouts

Aldeyra Therapeutics Inc ALDX 1.52% is scheduled to present Phase 3 data for reproxalap in allergic conjunctivitis at the AAO meeting Monday.
Lineage Cell Therapeutics Inc LCTX 0.08% on Monday will present updated Phase 1/2 data for OpRegen in dry age-related macular degeneration at the AAO meeting.
IMMUTEP LTD/S ADR IMMP 1.19% is due to present Tuesday with Phase 1 data for eftilagimod alpha and Merck & Co., Inc. MRK 0.64%‘s Keytruda in melanoma.
Marinus Pharmaceuticals Inc MRNS 2.86% will present already released Phase 2 data for ganaxolone in refractory status epilepticus on Wednesday.

Earnings

  • Johnson & Johnson: Tuesday before the market open.
  • Abbott Laboratories ABT 0.67%: Wednesday before the market open.
  • Intuitive Surgical, Inc. ISRG 1.33%: Thursday after the close.

IPOs

Innate Pharma, which develops antibody therapies for cancer, is proposing a 10.67-million-share IPO, with the offering expected to be priced at $7.50. The company seeks to list its shares on the Nasdaq under the ticker symbol “IPHA.”

IPO Quiet Period Expiry

IGM Biosciences Inc IGMS 1.03%
Exagen Inc XGN 3.3%
https://www.benzinga.com/general/biotech/19/10/14575481/the-week-ahead-in-biotech-fda-decisions-johnson-johnson-among-big-pharma-earnings

As Elizabeth Warren Rises, Insurers Tighten Grip On Medicare

The health insurance industry is setting up new sales territories and blitzing seniors with television ads as they market private Medicare Advantage plans in more U.S. counties than ever despite the threat of Democrats to put them out of business.
In the last week, several pollsters showing the majority of Americans wanting Donald Trump to face an impeachment inquiry are also showing U.S. Sens. Elizabeth Warren and Bernie Sanders well ahead of the President should he face either in the general election next November 2020. A Fox News poll last week had Warren leading Trump 50% to 40% in a head-to-head general election matchup. Both Warren and Sanders favor a single payer version of expanded Medicare coverage for all Americans that would uproot the role of private insurers.
But Warren’s potential to challenge Trump should he be the Republican presidential nominee is becoming more real as some new polls of Democratic voters for their party’s nomination show her in a tight race with Joe Biden. The former Vice President is a supporter of privatized Medicare Advantage, which grew in the eight years of the Barack Obama White House thanks in part to new benefits under the Affordable Care Act. New regulations under Trump have allowed private insurers to flourish even more with the ability to sell more supplemental benefits.
Analysts say insurers are looking at the broader picture showing regulations favoring a private health plan’s participation in practically all areas of government-subsidized health benefit administration from Medicare and Medicaid to subsidized commercial coverage under the ACA.
“Aside from single payer, most health reform proposals revolve around broad eligibility for coverage in a regulated and subsidized direct-to-consumer market, so it makes sense that insurers are increasingly eager to participate in real-world opportunities like Medicare Advantage and the individual market,” says Kathy Hempstead, senior policy advisor at the Robert Wood Johnson Foundation. “There may be a sense that this is where the puck is going.”
This month, insurers are ramping up marketing and announcing their expansions in new regions ahead of the annual open enrollment period when seniors eligible for Medicare can choose new benefits or stay with their existing plans. Such changes can be made during Medicare’s open enrollment, which begins this Tuesday, Oct. 15 and runs through Dec. 7.
Already this month, major health insurers including Cigna and UnitedHealth Group have announced they are launching unprecedented expansions into new regions of the country to sell Medicare Advantage plans. Other insurers including CVS Health’s Aetna, Centene and Humana are also launching major expansions of Medicare Advantage products.
There will be about “1,200 more Medicare Advantage plans operating in 2020 than in 2018,” the Centers for Medicare & Medicaid Services said last month. “The average number of Medicare Advantage plan choices per county will increase from about 33 plans in 2019 to 39 plans in 2020. This represents an increase of 49% since 2017.”
Insurers are taking advantage of new rules that allow Medicare Advantage plans to offer more benefits to seniors. Medicare Advantage plans contract with the federal government to provide extra benefits and services to seniors, such as disease management and nurse help hotlines with some also offering vision, dental care and wellness programs.
Enrollment in Medicare Advantage plans surpassed 22 million in 2018, which is 35% of total Medicare beneficiaries. Given the rule changes, one analyst — L.E.K. Consulting — has projected enrollment of seniors in private Medicare Advantage plans could reach 70% of those eligible for federal health benefits for the elderly between 2030 and 2040.
UnitedHealth Group, which has already clashed publicly with Sanders over single payer, said it will expand its Medicare Advantage health plan offerings to seniors in 100 new counties next year to reach an additional 1.2 million people. UnitedHealth will offer Medicare Advantage in 47 states in 2020, up from 45.
UnitedHealth, which is the nation’s largest insurer, currently sells individual Medicare Advantage plans in more than 1,700 counties but that will grow to more than 1,800, the insurer said. The insurer has new ads running across the country.
“In 2020, 90% of people eligible for Medicare will have a choice of UnitedHealthcare Medicare Advantage plans, nationwide,” the company said earlier this month.
Meanwhile, Anthem said its “affiliated health plans” will offer Medicare Advantage plans in 77 new counties for 2020. “These health plans currently sell Medicare Advantage plans in more than 850 counties and that will grow to nearly 950 counties in 2020, across 14 blue states and other affiliated plans,” Anthem said. And Cigna said its Medicare Advantage health maintenance organization plans in 2020 will be in 303 counties, 37 of which are new with the expansion,” a Cigna spokeswoman said. “We were in 266 counties before expansion.”
With the increased competition, premiums are expected to be lower in many areas of the country, the Trump administration has said.
“These dramatically lower costs in Medicare Advantage and Part D, thanks to President Trump’s leadership, means that we are putting dollars back into the pockets of our seniors,” CMS Administrator Seema Verma said last month. “On the contrast, proposals for more government through Medicare for All or a public option, would only harm the progress we have made to protect and strengthen the Medicare program for future generations.”
https://www.forbes.com/sites/brucejapsen/2019/10/13/as-elizabeth-warren-rises-insurers-tighten-grip-on-medicare/#529dcb0527f9

Only 15% of trials could be replicated using real world data

A study published this week in JAMA Network Open finds that currently available real-world data (RWD) sources can only be used to feasibly replicate 15% of clinical trials.
The aim of the study was to determine whether RWD could be used to power observational studies that answer the same clinical questions as traditional clinical trials.
Randomized controlled trials (RCTs) are considered the gold standard for clinical evidence to support the safety and efficacy of medical products due to high levels of internal consistency and reduced bias.
However, as the authors of the study write, “Compared with RCTs, RWE [real-world evidence] better reflects the actual clinical environments in which medical interventions are used, including patient demographics, comorbidities, adherence, and concurrent treatments,” noting that RCTs are costly and time intensive compared to observational studies.
To conduct the study, the authors reviewed 220 clinical trials conducted in the US that were published in the top seven medical journals in 2017 and determined whether RWD obtained from insurance claims and electronic health records (EHRs) contained the information necessary to replicate the studies.
Of those trials, the authors were only able to identify 86 (39%) that “had an intervention that could be ascertained from insurance claims and/or EHR data.” From there, the authors narrowed the trials further to identify ones with an indication and inclusion/exclusion criteria that could be extracted from RWD.
From there the authors were able to identify just 33 (15%) trials with one or more primary endpoints that could be ascertained from available RWD sources.
“This finding suggests the potential for real-world evidence to complement clinical trials, both by examining the concordance between randomized experiments and observational studies and by comparing the generalizability of the trial population with the real-world population of interest,” the authors write.
However, the authors caution that for new products, RWE is unlikely to serve as a replacement for RCTs and point out that many of the trials they looked at could not be replicated because the data necessary to do so “are unlikely to appear in an EHR in structured form if at all.”
But the authors stress that RWE could be used to provide “critical insights” into product safety and efficacy in the postapproval setting and could allow the FDA to identify and act on safety issues more quickly.
Researchers find 15% of trials could be replicated using real world data

In-office gene therapy for wet age-related macular degeneration is coming

Gene therapy is showing promise for one of the most common causes of blindness. Data presented today shows that six patients with wet age-related macular degeneration (AMD) have, so far, gone at least six months without the need for continued injections to control a disease that typically requires treatment every four to six weeks. Researchers say the hope is that gene therapy will free patients from nearly monthly eye injections by offering a potential “one-and-done” treatment. It’s not just about convenience; a more consistent treatment may also help people keep more of their vision. The research will be presented today at AAO 2019, the 123rd Annual Meeting of the American Academy of Ophthalmology.
“This is potentially paradigm shifting,” said lead researcher, Szilárd Kiss, M.D., director of Clinical Research and chief of the Retina Service in the Department of Ophthalmology at Weill Cornell Medical College in New York City. “It’s the next revolutionary leap in treating AMD. When you think about what is science fiction and what is science reality; for AMD is becoming a clinical reality.”
Dr. Kiss expects a gene for wet AMD could be available within the next three to five years, depending upon the results of further clinical studies and regulatory approvals.
AMD is the most common cause of vision loss and blindness in Americans over age 50, affecting about 2.1 million people nationwide; a number that’s expected to explode as the population ages. AMD is a that happens when part of the retina is damaged. The damage happens when new, weak blood vessels form behind the retina at the back of the eye. These abnormal vessels leak, causing scaring and killing off the cells that allow us to see.
The first revolutionary leap in AMD treatment occurred a little over a decade ago, with the introduction of a new medication called anti-VEGF therapy. It was the first treatment to inhibit the formation of those damaging, new blood vessels, allowing more than 90 percent of patients to keep their vision, according to clinical trials.
However, in the real world, the actual percentage is closer to 50 percent. One main reason why is that patients are undertreated. This is because most people with AMD must go to the ophthalmologist’s office every four to eight weeks for an injection directly into their eye (oftentimes in both eyes). This can be a difficult schedule to maintain for many elderly patients struggling with other maladies and reliant on others to get them to their ophthalmologist visits. It’s also unsustainable for the health care system. Last year alone, ophthalmologists performed more than 8 million anti-VEGF injections in the United States.
Researchers have been searching for a better alternative to monthly injections almost from the moment anti-VEGFs were introduced. Gene therapy is emerging as one of the more promising alternatives to long-term anti-VEGF treatment.
The goal of Dr. Kiss’ research is to develop a gene therapy that allows the eye to make its own anti-VEGF medicine. The ideal gene therapy would be administered not through a surgical procedure in an operating room, but through an injection into the eye that can be done in the doctor’s office, just like routine anti-VEGF treatment is done today.
To do this, Dr. Kiss and colleagues have developed a next-generation vector that can insert into the cells of the eye, genetic material that makes a molecule similar to a widely used anti-VEGF medicine called aflibercept. Once inside the cells, the DNA sequence begins making the aflibercept protein.
“Instead of taking a vile of aflibercept and injecting it into the eye, your eye makes the aflibercept,” Dr. Kiss explained. “The goal is a potentially one-and-done treatment. You may need a booster once in a while, but this gene therapy could theoretically last a lifetime.”
In animal studies, Dr. Kiss and colleagues have shown that this gene therapy works just as well as an injection of aflibercept, with a favorable and manageable side-effect profile.
Dr. Kiss will be presenting today early data on the first human subjects treated. The phase 1 clinical trial has thus far enrolled 12 patients who received a single of gene therapy for wet AMD. Prior to the study, the patients had received an average of 35 anti-VEGF injections; one patient had 109. Since entering the gene therapy trial, the patients required no rescue treatments through the first six months.

Explore further
Researchers advance search for safer, easier way to deliver vision-saving gene therapy
https://medicalxpress.com/news/2019-10-in-office-gene-therapy-age-related-macular.html