National Strategy for Alzheimer’s Clinical Research to Launch this Fall

According to the Alzheimer’s Association, Alzheimer’s disease (AD) is the sixth-leading cause of death in Americans. Between 2010 and 2015, deaths from AD increased 123 percent. The National Institute of Health (NIH) sponsors in whole or in part, approximately 140 active clinical trials into AD. The Alzheimer’s Association indicates their database has more than 250 studies ongoing.

The problem? There aren’t enough people participating in the trials.

This became enough of a concern that the NIH’s National Institute on Aging and the Alzheimer’s Association brought together various experts and stakeholders to create the National Strategy for Recruitment and Participation in Alzheimer’s Disease Clinical Research. This effort will launch this fall. “We anticipate a finished product that will give helpful strategies for recruitment at local, regional, and national levels to other governmental agencies, medical providers, people with Alzheimer’s, and caregivers,” stated Marie Bernard, geriatrician and deputy director of the NIH. “Everyone needs to be more informed and aware of the challenges and opportunities of recruitment.”

Alzheimer’s disease clinical trials pose specific challenges based on varying age requirements and cognitive abilities. Some patients need to be healthy and cognitively unimpaired, while others often involve severely impaired individuals. Clinical Informatics News points out, “While people in cognitive decline require a partner to assist with tasks such as driving and reporting, potential volunteers with no impairment often don’t enroll in AD trials due to a lack of awareness or the stigma of AD.”

The National Strategy is part of the National Plan to Address Alzheimer’s Disease. The research goal of the plan is to prevent and treat Alzheimer’s disease by 2025, a daunting and ambitious goal only seven years away. The NIA states, “Specific actions outlined in the National Plan related to recruitment seek to 1) increase enrollment in clinical trials and other clinical research through community, national, and international outreach, and 2) monitor and identify strategies to increase enrollment of diverse populations in Alzheimer’s disease studies. With development of a National Strategy, the community will also seek to develop ways to track activities and monitor progress.”

Noting that primary physicians are often the access point for involvement in clinical trials, the National Strategy is also working to expand that role to other community leaders. “For example,” Clinical Informatics News writes, “outreach must start with the tribal elders in Native American reservations and with church elders in African-American communities.”

Bernard told Clinical Informatics News, “Researchers must take into consideration the context. They must know the community, work in it and become a part of it. There are differences from site to site; researchers must be cognizant of the nuances within different sites. We must focus on the fact that there needs to be diversity in studies.”

As an example, Indiana University developed a community-based outreach program to increase African-American involvement in clinical trials. It works with a Community Advisory Board made up of clergy, retired volunteers, an elder law attorney, and representatives from the State and County Boards of Health.

The Alzheimer’s Association created TrialMatch in 2010, a matching service on its website that is free and matches healthy individuals to trials and AD patients to caregivers.

Other examples include the Duke Alzheimer’s Disease Prevention Registry (ADPR), and the Banner Alzheimer’s Institute’s GeneMatch program.

The National Strategy also focuses on improving infrastructure, including sufficient staffing to conduct an 18 to 24-month clinical trial. The NIH also has developed $3 million funding opportunity, Examining Diversity Recruitment and Retention in Aging Research, to study the science of Alzheimer’s disease recruitment.

Heather Snyder, Senior Director of Medical and Scientific Operations for the Alzheimer’s Association told Clinical Informatics News, “This is not a one-and-done sort of thing. It must be monitored to see what needs to be tweaked and to further enhance it.”

https://bit.ly/2waRSDI

FDA Suggests Use of Placebo is Necessary Only in Certain Types of Oncology Trials

In a new draft guidance released Thursday, the U.S. Food and Drug Administration (FDA) is questioning whether or not the use of a placebo in a double-blind, randomized clinical trialis always necessary.

The FDA said sometimes the use of a placebo can present practical or ethical concerns in double-blind, randomized trials conducted in development programs for drugs products for the treatment of malignant hematologic and oncologic disease. In its three-page draft guidance, which was first reported by RAPS, the FDA said that because of the toxicity profile of the active treatment, patients and investigators can infer which treatment is being received during a trial. Because of that, the use of a placebo control “may not, in fact, blind the treatment,” the FDA said.

“For patients with hematologic malignancies and oncologic diseases that have standard effective therapy available, use of a placebo (not an active treatment) poses ethical issues. If possible, an active control is often preferred over placebo, and one option has been to conduct an open-label trial with a physician’s choice of one of a few standard therapies as the comparator. Another option has been to compare the investigational drug product to placebo, with each added to the standard of care,” the FDA noted in the draft guidance.

Not only that, but the FDA said the continued blinding of patients and investigators at the time of disease progression or occurrence of serious adverse events presents additional challenges. As an example, the FDA said in an immunotherapy trial, a patient on the control arm who develops adverse events may receive unnecessary treatments, such as immunosuppressive drugs because the adverse events have been “incorrectly attributed to the investigational drug product.” Continuing with a blinded trial after disease progression could also affect subsequent patient therapy. That could prevent a patient on the placebo arm from receiving an approved therapy, or prevent a patient from entering another approved trial, the FDA said.

Unblinding these kinds of trials would allow informed decision making when it comes to additional treatment, the FDA said.

Because of this, the FDA is recommending in its draft guidance that trial sponsors use a placebo-controlled design only in select circumstances, which could include where surveillance is standard of care, or if the trial uses an add-on design “when the endpoint intended to support a labeling claim has a high degree of subjectivity, such as patient-reported outcomes.”

The FDA said when considering a placebo for a clinical trial, sponsors should provide a rationale for the trial design. The FDA noted that it also does not require “patient-level maintenance of blinding” at the time of a disease recurrence or progression.

“Unless there are no available appropriate treatment alternatives, FDA recommends unblinding a patient at the time of documented disease recurrence or progression to ensure optimal patient management,” the agency said in its draft guidance.

https://bit.ly/2NftirP

J&J tells Bloomberg job posting not related to Geron decision

Johnson & Johnson (JNJ) told Bloomberg that its job posting is not related to the expected decision on its licensing pact with Geron (GERN). “The job description outlines responsibilities for a variety of disease areas and potential products/line extensions that are consistent with what has been disclosed as part of the Janssen oncology hematology portfolio,” J&J told Bloomberg. Shares of Geron are off the day’s highs but remain up 25% to $5.06.

https://bit.ly/2PxMVge

Cigna, Express Scripts shareholders approve $52 billion deal

Cigna Corp (CI.N) shareholders on Friday voted in favor of the health insurer’s proposed $52 billion acquisition of pharmacy benefit manager Express Scripts Holding Co (ESRX.O), although the deal still needs clearance from antitrust authorities.

The vote in favor of the merger was on expected lines after activist investor Carl Icahn walked away last week from his 11th-hour attempt to rally shareholders to reject the deal.

The U.S. Department of Justice is still conducting an antitrust review of the combination that is not expected to close until later this year.

According to the preliminary results, about 90 percent of the votes cast were in favor of the merger, the health insurer said.

Cigna expects the merger to close by end of 2018.

The insurer agreed to buy Express Scripts in March saying the two companies could save more on healthcare costs for clients if they better coordinated medical care with prescriptions.

But since then Express Scripts has come under stiffer opposition from the Trump administration, lawmakers and drug makers as being a middleman that drives up drug costs. Icahn had argued that Cigna was overpaying given prospects for reduced profits.

He backed down after shareholder advisory groups recommended that shareholders vote for the deal.

Cigna Corp185.57

CI.NNEW YORK STOCK EXCHANGE

-0.23(-0.12%)

• CI.N
• ESRX.O
• CVS.N
• AET.N
• ANTM.N

Express Scripts shareholders also approved the deal, with about 78 percent of outstanding shares of Express Scripts voted for the deal.

Cigna’s plan for the acquisition comes as Express Scripts’ major rival CVS Health (CVS.N) and Aetna Inc (AET.N) move forward on their own merger. Cigna’s deal to be bought by Anthem Inc (ANTM.N) fell apart last year after it failed to pass antitrust review.

https://reut.rs/2BI1zPq

One Marijuana IPO Gets Set To Report, Another Is Actually Profitable

Tilray (TLRY), the first pure-play marijuana IPO to hit a major U.S. exchange, reports second-quarter results on Tuesday, as another IPO gets set to join the growing list of marijuana stocks.

Tilray Earnings

Wall Street expects Tilray to report a per-share loss of 9 cents on revenue of $9 million. The report will be Tilray’s first since its IPO in July.

Last year’s sales for the company, which sells medical cannabis products in Canada, jumped 62% to $20.5 million, with a per-share loss of 9 cents and a net loss of $7.8 million. The company’s accumulated deficit at the end of the year was $40.5 million.

For the quarter ending March 30, Tilray’s sales rose 55% to $7.8 million, on a loss of 6 cents per share. Net losses during the quarter mushroomed to $5.2 million from $679,000 a year earlier.

Cowen analyst Vivien Azer said Tilray stands to benefit from its relationship with Privateer Holdings, a cannabis-focused private-equity outfit in Seattle. Privateer owns a majority of Tilray, and its portfolio also includes marijuana information website Leafly as well as Marley Natural, the “official cannabis brand of Bob Marley.”

Azer, who started coverage this month on Tilray with an outperform rating, said Tilray’s arrangement with Privateer gives it the opportunity to license “established U.S.-based cannabis brands in Canada.” Tilray also gets around 5.5% of sales from Germany, New Zealand and Australia, with more room to expand, Azer said.

In addition, she said, being under the Privateer umbrella gives Tilray access to a decade’s worth of data from Leafly, a site that tracks consumer preferences for different pot brands and strains. Leafly, which also publishes user reviews and other news about the industry, is the world’s largest cannabis website, Azer said. It draws more than 13 million visitors monthly.

As Canada prepares for recreational legalization in October and products like vapes and edibles next year, Leafly could provide Tilray with an analytical edge over rivals.

“We believe this is critical in the Canadian adult-use market given the form-factor rollout for vapes and edibles in 2019,” she said.

As legalization expands, the language of marketing and connoisseurship has found its way into the marijuana industry. Weed is no longer just weed, the way it was in decades past. More customers could be enticed by the idea of variety, the way they have with, say, craft beer.

Case in point: U.S. marijuana retailer MedMen, in its magazine Ember, this month published an article in which its cannabis “curator” selected his top five strains of the season. Durban Poison, the top pick, was described as “joyful, mellow, floral.” Wedding Cake, the No. 5 selection, was “peppery, effervescent, then sleep-inducing.”

Booze Lifts Marijuana Stocks

Marijuana stocks got a big lift Friday, as Bloomberg reported that Diageo (DEO), the owner of Guinness and maker of Johnnie Walker, has met with at least three pot producers over the last month as it weighs whether to strike a deal to make pot-laden drinks.

Diageo would only say to Bloomberg that “we are monitoring this space closely.”

The news comes after Corona parent Constellation Brands (STZ) announced a $3.8 billion investment in Canopy Growth (CGC) earlier this month.

Molson Coors (TAP) also said this month that its Canadian business unit planned to offer marijuana beverages via a joint venture. The alcohol industry for the past several years has fretted quietly in SEC filings over the threat legal pot presents to their business.

Shares of Tilray jumped 9% in the stock market today. Canopy climbed 8%. Cronos Group(CRON) surged 14%.

Charlotte’s Web Hikes IPO Price

As Tilray prepares to report, Charlotte’s Web Holdings, a company based in Boulder, Colo., is preparing to go public in Canada, likely on the Canadian Securities Exchange.

The company, which makes cannabidiol hemp oils, topicals and capsules, priced its IPO at 7 Canadian dollars per share, the high end of its expectations. The company sold 14.3 million shares, more than anticipated, generating proceeds of 100.1 million Canadian dollars, according to a regulatory filing on Wednesday.

The filing, which reported sales and profits in U.S. dollars, also shows that Charlotte’s Web reported net income of $3.7 million during the quarter ended June 30 and $7.5 million last year. Other marijuana companies, like Canopy and MedMen, have put up losses as they invest in expansion.

Charlotte’s Web’s sales last year soared 172% to $40 million. Sales during its prior quarter were $17.2 million. The company said it believes it “has an opportunity” to reach $65 million to $80 million this year. It expects to have 300 acres of cultivation in 2018.

The company, like others, is also looking abroad, saying that “global distribution is paramount to its growth” over the next two years, according to the filing. Charlotte’s Web, for now, is focusing on markets in the European Union, South America and Asia.

https://bit.ly/2BItf6x

Geron soars after Johnson & Johnson job posting mentions Imetelstat

The move higher in shares of Geron (GERN) is being attributed to a Johnson & Johnson (JNJ) job opening on LinkedIn for an “EMEA Strategic Pricing Manager” role that would “also be accountable for shaping our pricing approach for Imetelstat.” The two companies currently have a partnership for Imetelstat and J&J is expected to make a decision on potentially continuing the pact in Q3, according to Bloomberg. Geron in early trading is up 25%, or $1.01, to $5.04. STAT’s Adam Feuerstein weighed in via Twitter: “$GERN trading +30% on a JNJ job posting that mentions imetelstat. Yes, it’s that ridiculous. Summer Fridays!”

https://bit.ly/2writvw

Bausch Health unit receives FDA approval for Altreno lotion for acne

Ortho Dermatologics, a division of Bausch Health Companies, announced that the U.S. FDA has approved the New Drug Application for ALTRENO lotion, indicated for the topical treatment of acne vulgaris in patients 9 years of age and older. ALTRENO is the first formulation of tretinoin in a lotion, and has been shown to be effective and generally well-tolerated.ALTRENO is expected to be available during the fourth quarter of 2018. “Today’s FDA approval of ALTRENO builds upon our strong acne portfolio, providing physicians and patients a trusted retinoid in a lotion formulated to enhance the user’s experience with the inclusion of moisturizing attributes of hyaluronic acid, glycerin and collagen,” said Bill Humphries, president, Ortho Dermatologics. “ALTRENO lotion spreads easily and is quickly absorbed into the skin allowing acne patients to easily incorporate this once-daily treatment into their skin care regimen.”

https://bit.ly/2o6ACuY