Scilex Has Pricing for ZTlido, Commercial Launch of 1st Product, Available in Oct.

Scilex Pharmaceuticals Inc. (Scilex), a subsidiary of Sorrento Therapeutics, Inc. (NASDAQ: SRNE) (Sorrento), today announced that the Wholesale Acquisition Cost (WAC) for ZTlido (lidocaine topical system) 1.8% (ZTLIDO) is $8.98 per patch and is now listed in pricing compendia.

Scilex expects commercial availability of ZTlido through wholesale distribution in October 2018.

‘We are excited to bring our first commercial product to the market,’ said George Ng, President, Business, of Scilex. ‘With ZTlido, we are proud to offer a non-opioid alternative and option for patients with post-herpetic neuralgia.’

ZTLIDO is a topical product that uses an advanced adhesion technology providing more efficient lidocaine delivery than Lidoderm over a full 12 hours. Post-herpetic neuralgia (PHN), also referred to as post-shingles pain, is a frequent complication of shingles, a condition caused by the herpes zoster (HZ) virus, which afflicts an estimated 1 million people each year in the US.

https://www.marketscreener.com/SORRENTO-THERAPEUTICS-INC-14708339/news/Sorrento-Therapeutics-Scilex-Announces-Pricing-and-Availability-of-ZTlido-for-the-Commercial-Launc-27297930/

J&J Ethicon Agrees To Sell Sterilization Unit for $2.7B

Ethicon Inc., a subsidiary of Johnson & Johnson, has accepted Fortive bid for its sterilization and disinfection products business.

In a securities filing Friday, Fortive said Ethicon agreed to its $2.7 billion offer for the unit on Sept. 20. The price tag is subject to adjustments based on the unit’s inventory book values at the time of closing and the amount of prepaid taxes.

Fortive said in the filing that regulators in the U.S., Germany and Brazil have signed off on the deal. Both sides still must apply for anti-trust clearance from authorities in Israel.

Either side has the right to terminate the deal if it has not been completed by April 5, 2019, the filing says.

Fortive, based in Everett, Wa., announced the offer in June, with an executive at the company saying then the Ethicon sterilization unit would boost its sales and position it in attractive markets.

The business produced about $775 million in sales last year, according to figures from Johnson & Johnson.

Shares of Fortive ticked down 0.4%, to $89.98, on Friday. Johnson & Johnson’s stock rose 0.6%, to $142.88.

https://www.marketscreener.com/JOHNSON-JOHNSON-4832/news/Johnson-Johnson-J-J-Unit-Agrees-To-Sell-Sterilization-Unit-for-2-7-Billion-27298994/

Glaxo fails to warn of pandemic flu vax safety issue: BMJ

GlaxoSmithKline’s H1N1 swine flu vaccine Pandemrix made waves around 2010 after increased cases of narcolepsy emerged in Sweden and Finland among children who got it during the previous pandemic. But a recent report by The BMJ suggests greater safety problems with the vaccine, which neither GSK nor health authorities have made public.

After examining several GSK-made internal safety reports obtained through an ongoing lawsuit alleging that Pandemrix caused the sleep disorder, the medical journal found a large discrepancy in adverse event rates dated years back between Pandemrix and two other GSK pandemic vaccines. Pandemrix have not been available for several years now.

Specifically, by Dec. 2, 2009, for each million doses of vaccine administered, about 76 cases of serious adverse events were reported for people who got Pandemrix, while eight were for Arepanrix and another unadjuvanted vaccine combined. The pattern continued for the next eight reports seen by The BMJ, and by the end of March 2010, the rate for Pandemrix was 72, seven times that for Arepanrix and the third vaccine combined.

The journal cautions that the numbers themselves are not sufficient to draw any causal relationship. But what it finds more problematic is that neither GSK nor health authorities seemed to have made the information public.

“[They] raise fundamental questions about the transparency of information,” about when the public should be alarmed of possible harms of vaccines detected through pharmacovigilance, Peter Doshi, an associate editor of The BMJ, wrote in his report.

In response to a FierceVaccines request for comment, GSK said: “Throughout the development and introduction of the Pandemrix and Arepanrix vaccines GSK fulfilled all requirements established by the authorities for the monitoring and evaluation of safety data. During the pandemic, GSK conducted enhanced evaluation of safety data and supplied all safety data and the company’s assessment to EMA and other authorities, summaries of which were made available to the public weekly on the EMA website.”

The disparity in adverse events reported was “of such striking difference that any person contemplating taking the Pandemrix vaccine would be likely, if in receipt of this information, not to choose to have [it],” Gillian O’Connor, the solicitor representing an Irish woman who’s suing GSK and health agencies, wrote in an affidavit filed in court.

Both Pandemrix and Arepanrix contain an oil-in-water emulsion adjuvant called AS03, but different methods are used to prepare the hemagglutinins used in them.

Pandemrix’s EU approval, first granted on Sept. 25, 2009, along with Novartis’ Focetria and Baxter’s Celvapan, has expired in August 2015 as GSK didn’t apply for a renewal “due to lack of demand,” according to an EMA statement (PDF). Arepanrix was approved in Canada in October 2009 but wasn’t in EU until the next March, at the tail of the pandemic, and its marketing authorization was voluntarily withdrawn at the request of GSK in late 2010.

It was not clear how much the EMA knew about the safety profile of those vaccines. But a press release from the agency in July 2011 said after considering all available data, including analysis of safety surveillance data performed in several member states and case reports from across the EU, its Committee for Medicinal Products for Human Use “confirmed that overall the benefit-risk balance of Pandemrix remains positive.”

“While it might have been possible to estimate reporting rates based on usage data, which are difficult to obtain during a pandemic, EMA does not have a methodology to compare reporting rates between two products (note that the pandemic influenza pharmacovigilance updates included number of reports, not rates),” an EMA spokesperson said, as quoted by The BMJ.

Neither Pandemrix nor Arepanrix has ever been greenlighted in the U.S. Instead, the FDA waved through four nonadjuvanted H1N1 vaccines by CSL, GSK, Novartis and Sanofi Pasteur. In a response to the narcolepsy reports following immunization with Pandemrix, the CDC carried out a population-based study and found the FDA-approved 2009 H1N1 flu shots were not associated with an increased risk for the neurological disorder.

https://www.fiercepharma.com/vaccines/glaxosmithkline-fails-to-warn-pandemic-flu-vaccine-s-alarming-safety-signal-bmj-report

Could Mylan swing sale to private equity buyer? One analyst thinks so

When Mylan said last month that it would consider a sale, analysts were skeptical the company could find an interested buyer. But one of them is changing his tune.

On Friday, RBC Capital Markets analysts Randall Stanicky laid out the case for a private equity player completing a leveraged buyout of the generics leader. It’s a potential move he says “should not be dismissed” thanks to Mylan management’s frustration with the company’s stock price and a generics industry Stanicky thinks is on the up-and-up.

The way he sees it, years without any stock movement—Mylan’s shares have been flat for the last five—may convince the company’s leaders to go private. They’ve already signaled their impatience with U.S. markets that continue “to underappreciate and undervalue the durability, differentiation and strengths of Mylan’s global diversified business,” as Mylan put it in an August statement.

And the generics industry, which has been ravaged by pricing pressure in recent years, could be headed for a turnaround, he figures, which would make now a “favorable” time for a private equity suitor to strike.

Of course, there are some major hurdles—namely, that Mylan’s purchaser, even if prepared to take on a whole lot of debt, would have to put forth $11.6 billion in equity to pull off a deal of about $40 billion, according to Stanicky’s calculations. But while that’s a “large amount, there are plenty of examples of sizable LBOs to point to as support,” he noted.

Mylan first announced last month, amid a spate of bad earnings news, that it would launch a strategic review, keeping all options—including a sale—on the table. At the time, though, industry watchers didn’t get too jazzed about the possibility.

“Many at the time (including us) dismissed this as an attempt to support the stock amidst a sizable miss and guide cut,” Stanicky said.

Even if the generics landscape does improve, though, Mylan still has some of the same problems that made other analysts doubtful it could work out a sale—such as troubles at a Morgantown, West Virginia, manufacturing facility and what Bernstein’s Ronny Gal called slower-than-expected approvals and uptake for the company’s complex generics.

“The U.S. business has been hit twice—once by the broader industry trends” affecting generics makers and “then by company-specific action,” Gal wrote to clients at the time.

Wells Fargo’s David Maris agreed in his own investor note, writing that, “We do not think there is a high likelihood that a public or private buyer for the company or significant parts of the company emerges.”

https://www.fiercepharma.com/pharma/could-troubled-mylan-swing-a-sale-to-private-equity-buyer-one-analyst-thinks-so

Cannabics Pharmaceuticals to Present Trial Results at Medical Cannabis Confab

Cannabics Pharmaceuticals Inc. (OTCQB: CNBX), a leader in personalized cannabinoid medicine focused on Cancer and its side effects, today announced that Dr. Gil Bar-Sela is scheduled to present his findings at the 3^rd International Medical Cannabis Conference. This Conference is held for global leaders in areas of Medical Cannabis to convene, explore and present the latest scientific and clinical research in medical cannabis, held in Tel Aviv from October 14^th-16^th, 2018.

Dr. Bar-Sela is the Deputy Director of the Division of Oncology at Rambam Health Care Campus, Head of the Palliative and Supportive Oncology Unit, and Head of the service for Melanoma and Sarcoma patients.

Dr. Bar-Sela has vast knowledge and experience in clinical evaluation of medical cannabis benefits to Cancer patients. Dr. Bar-Sela was the principal investigator of the company’s clinical study, the first meaningful scientific study regarding the dosage and treatment regimen in oral administration of cannabis and the benefits in treating CACS in Cancer patients. While briefly noted in the Company’s press release of July 3^rd, 2018; this will be the first public scientific presentation of the results of this pilot study.

While this Clinical Study was conducted at Rambam Hospital in Israel, it is registered with the US NIH under “Cannabics Capsules as Treatment to Improve Cancer Related CACS in Advanced Cancer Patients”, Identifier NCT02359123, and may be found at https://clinicaltrials.gov/ct2/show/NCT02359123

Dr. Bar-Sela’s Lecture is scheduled for Monday, October 15th, 2018, and entitled “OPENING THE GATES TO NEW CLINICAL RESEARCH.”

https://www.biospace.com/article/releases/cannabics-pharmaceuticals-to-present-results-of-clinical-trial-at-international-medical-cannabis-conference/

European Regulators Reject Sarepta’s Exondys 51 for Duchenne

In September 2016, the U.S. Food and Drug Administration (FDA) approved Sarepta Therapeutics‘ eteplirsen, now marketed as Exondys 51, for Duchenne muscular dystrophy (DMD). It was a contentious and dramatic approval, one involving internal battles at the agency, media coverage and members of Congress and panels of DMD experts sending public letters to the agency to approve the drug.

And now, two years later, the European Medicines Agency (EMA) has rejected Sarepta’s application for Exondys 51. It wasn’t unexpected. In June, the Committee for Medicinal Products for Human Use (CHMP) at the EMA recommended against Exondys 51.

DMD is a muscle wasting disease caused by mutations in the dystrophin gene. The disease is progressive and typically causes death in early adulthood, with serious complications that include heart or respiratory-related problems. It mostly affects boys, about 1 in ever 3,500 to 5,000 male children.

Sarepta’s application in Europe was built on two clinical trials in 12 boys with DMD between the ages of 7 and 13. The first was a Phase II clinical trial that randomized 12 children to receive Exondys 51 or a placebo weekly over 24 weeks. The primary endpoint was dystrophin production and the assessment of a six-minute walk test. In the trial, there was no significant difference in the walking distance in six minutes between those receiving Exondys 51 or placebo.

Muscular Dystrophy News Today reported in June, “All study participants continued on weekly Exondys 51 treatment the second study, an open-label trial that ran for four years and moved the walk test to a co-primary study objective. Clinical data collected from these Duchenne patients and covering both trials were compared with established DMD natural history data.”

The data indicated that the patients who received Exondys 51 at either 30 mg/kg or 50 mg/kg of body weight had a statistically significant and clinically meaningful decrease in pulmonary function decline. There was also an increase in the amount of dystrophin protein up to 0.44 percent of normal levels after 48 weeks.

However, CHMP, in its negative opinion, expressed doubts about the evidence, especially related to the study’s small size, use of historical data, and lack of comparison data beyond 24 weeks. In their response letter, CHMP wrote, “the methods for comparing results of the main studies with historical data were not satisfactory for showing that the medicine was effective. … further data were needed to show that the very low amounts of shortened dystrophin produced as a result of Exondys treatment bring lasting benefits relevant to the patient.”

And in fact, these were essentially the same controversies within the FDA, specifically the criticisms brought by Ronald Farkas, FDA clinical team leader, who left the agency just before approval, and the agency’s acting chief scientist, Luciana Borio, and Ellis Unger, director of the office of drug evaluation. They were overruled by Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research (CDER). The final decision went to Robert Califf, then the FDA’s Commissioner, and evidence shows Califf had similar reservations as Borio and Unger, but sided with Woodcock.

As part of the FDA approval, Sarepta had to run a confirmatory and open-label Phase III trial into the drug’s efficacy. This trial is ongoing and expected to wrap up in May 2019. The primary endpoint is improvements in the six-minute walk test after 96 weeks of treatment.

Although the drug appears to cause some improvement in dystrophin levels, whether there are any actual clinical improvements is up for debate, particularly given the degree to which the walk test can be affected by a placebo effect. The lack of dystrophin, caused by mutations in the dystrophin gene, is what causes the muscle wasting that characterizes the deadly disease.

Meanwhile, the drug has a yearly price tag, based on the patient’s body weight, of approximately $300,000.

In response to today’s rejection, Doug Ingram, president and chief executive officer of Sarepta stated, “While largely anticipated, we are disappointed with the outcome of the CHMP re-examination and firmly believe that eteplirsen should be made available to patients in Europe, as it is in the United States. We were, however, encouraged by the openness of discussion with the SAG and CHMP and their willingness to engage on different approaches to provide additional data to support an eventual approval in Europe.”

He goes on to say that the company will continue to work to get the drug approved in Europe.

https://www.biospace.com/article/going-its-own-way-european-regulators-reject-sarepta-s-exondys-51-for-dmd-fd1a-/

Bluebird Bio rating change at Goldman Sachs

Bluebird Bio downgraded to Buy from Conviction Buy at Goldman Sachs. Goldman Sachs analyst Salveen Richter removed Bluebird Bio from his firm’s Americas Conviction List but keeps a Buy rating on the name with a $288 price target. The rating change is is not driven by concerns around Bluebird’s pipeline execution or emerging competitive threats, Richter tells investors in a research note. The analyst just believes appreciation of key aspects of his investment thesis are better reflected in the shares at current valuation levels.

https://thefly.com/landingPageNews.php?id=2793601