Cesca started at buy by Wainwright on CAR-T opportunity

Cesca Therapeutics Inc KOOL 5.56% is poised to make much headway with its automated CAR-T manufacturing platform CAR-TXpress, according to H.C. Wainwright.

CAR-T-cell therapy is a method of treating cancer by using a patient’s immune system cells —  T cells — which are then modified by adding a gene in the lab for a special chimeric antigen receptor, or CAR, to attack cancer cells. CAR binds to a certain protein present in the patient’s cancer cell.

The Analyst

Analyst Swayampakula Ramakanth initiated coverage of Cesca with a Buy and $1.50 price target.

The Thesis

Cesca’s CAR-TXpress is likely to address the factors that are behind the high production costs of CAR-T cells, which could impede further advancements in CAR-T research and adoption, Ramakanth said in a Wednesday note.

The production costs are $100,000 per patient, the analyst said, naming five reasons for the high cost:

• Insufficient starting T-cell material.
• Labor- and time-intensive processing steps.
• Expensive reagents and machinery.
• Earge clean room footprints.
• Complicated c-GMP compliance.

CAR-T therapies have a market opportunity of $8.5 billion by 2028, up from $15 million in 2017, according to H.C. Wainwright.

Modular automation — a feature of CAR-TXpress — could be a game-changer, Ramakanth said.

“Currently, we project CAR-TXpress products to generate revenue of $548,000 in 2018, growing to $34 million by 2030.”

H.C. Wainwright projects opportunity for the company in the contract research development manufacturing organization market; Cesca has signed an exclusive license agreement in China and is likely to expand in the U.S., Ramakanth said.

https://bit.ly/2IOWLH6

Danone, Nestle, KKR and more suit up for GSK consumer bidding war

GlaxoSmithKline’s Indian consumer health sale may just touch off a bidding war.

Major players including Danone, Hindustan Unilever and Nestlé are squaring off against private equity firm KKR to nab Glaxo’s 72.5% stake in its Indian consumer products business, The Economic Times reported. PepsiCo, Abbott and Mondelez are reportedly in the mix, too.

The sale could drum up $4 billion, the India-based Times says, which could make it the biggest deal in consumer history there. And a major cash infusion is exactly what GlaxoSmithKline is looking for to help fund its own consumer buy: A $13 billion pickup of Novartis’ share in their consumer joint venture, agreed upon in March.

For some of the rumored bidders in India, such as Danone and Abbott, the Indian company—which markets well-known drink brands Horlicks and Boost—would fit right into their portfolios. KKR, though, could be a tough M&A rival: It’s one of the most aggressive PE investors in India, TET noted, and it’s been scouting for big-time assets in the country.

For now, talks are still in the early stages, the Times’ sources said. The process should pick up steam next month, after initial bids become formal, nonbinding offers and GSK pares down its list of suitors to continue negotiations.

GSK said it was weighing a sale of its share in GlaxoSmithKline Consumer Healthcare Ltd., which is a public company listed on India’s stock exchanges, at the same time it went public with the Novartis deal agreement. But as Glaxo was quick to point out, it’s still focused squarely on the Indian market with its other products.

India “remains a priority market for GSK investment and growth,” the pharma giant said at the time, adding that it would continue to back OTC and oral health brands in the country and invest in pharmaceuticals and vaccines there, too.

https://bit.ly/2GMELv8

Solicitor General urges SCOTUS to hear Merck Fosamax case

Merck & Co.’s effort to take its Fosamax appeal to the Supreme Court just got a boost, and the entire pharma industry should take note. The U.S. Solicitor General is urging the justices to take on the appeal, which centers on a set of closely watched lawsuits alleging Merck failed to fully flag the risks of its osteoporosis drug Fosamax.

If the high court hears the drugmaker’s appeal, its decision could ripple through product liability lawsuits across the drug business. That’s because at its heart is the issue of preemption: whether FDA decisions can protect drugmakers from state-level legal challenges.

Last year, a federal appeals court in New Jersey revived the previously dismissed Fosamax litigation and allowed a jury to examine whether Merck was entitled to protection, based on the FDA’s decision-making on the drug’s official label. In 2009, the agency had rejected Merck’s request to update Fosamax’s label with certain fracture risks, and Merck now argues that the FDA’s decision precludes state tort claims.

After the Supreme Court asked the Solicitor General to weigh in, the nation’s top lawyer sided with Merck, saying that preemption should be determined by a judge, not a jury, according to a brief (PDF).

“Where, as here, FDA renders a decision declining to approve a drug-labeling change, the interpretation of that administrative decision and its significance for a failure-to-warn claim are legal questions for a court to resolve, not factual questions for a jury,” wrote the Solicitor General.

“Judges, rather than lay juries, are best suited to evaluate the scope of an agency’s legal determination in light of the relevant statutory and regulatory context,” the Solicitor General added.

More than 500 patients who took Fosamax suffered femoral fractures before the FDA added a warning of that risk to its label in 2011.

Merck previously argued that preemption should apply because it had attempted to update the label earlier, only to be defeated by the FDA because of wording. But the patients contended that the “stress fracture” term used in Merck’s request did not describe the injuries they experienced. “[Merck] proposed a warning only about a different risk,” therefore, the FDA never rejected a warning of “the risk at issue,” the attorney representing the patients previously said.

In his brief—and in a victory for Merck—the Solicitor General said the FDA’s rejection of the label change was “clear evidence” supporting preemption. “[B]ecause FDA’s decision here prevented [Merck] from modifying the relevant labeling before late 2010, the court of appeals erred in rejecting [Merck’s defense].”

Plus, the Solicitor General argues that the FDA’s decision was actually based on the lack of adequate data to support a warning. “If a warning is warranted, FDA will attempt promptly to identify easily correctable deficiencies in the proposed text and will then develop final labeling text with the manufacturer in an iterative process.”

Now, the Supreme Court will decide whether to review Merck’s case. If it takes the suit, it could become another landmark preemption case after Wyeth v. Levine, where the Supreme Court opens up the possibility of lawsuits against drugmakers even if a medication wins FDA approval.

https://bit.ly/2IKWNPU

Samsung BioLogics gets added FDA OK for its world largest plant’s production

 Samsung Biologics  announced today that a third monoclonal antibody Drug Substance has been licensed by the US FDA (Food and Drug Administration) for its second plant, the world’s largest single plant.

With the approval, Samsung BioLogics has been approved by the FDA for the manufacture of three commercial Biologics Drug Substances at only its second plant in just 26 months since being GMP ready in 2016.

Samsung BioLogics has improved production efficiency through the installation of ten 15,000L bioreactors, a world’s first, breaking industry stereotypes to install and operate only four or six bioreactors in a single bio-manufacturing plant.

Samsung BioLogics has demonstrated that both Plant 1 and Plant 2 are compliant with global GMP standards through receiving 14 global regulatory agency’s approvals including FDA, EMA, and PMDA.

Although Plant 2 is 5 times larger and 10 times more complicated, Samsung BioLogics received initial manufacturing approval 6 months faster than Plant 1. This shortened time in construction and period of approval allows our clients to quickly supply their products to the market.

With its competitiveness, Samsung BioLogics has won the CMO Leadership Award presented by Life Science Leader in all six core categories for two consecutive years.

For the 2018 CMO Leadership Awards, more than 110 CMOs from around the world were evaluated on 23 performance metrics to determine winners in the six categories. Only CMO clients participate in the survey, which enhances the validity of the award.

TH Kim, CEO and President of Samsung BioLogics, said “There has been some cases recently where CMO’s failure of receiving approval affects the clients’ drug marketing schedule.” Then he added that “Samsung BioLogics has once again demonstrated that it possesses world class Quality with Data Integrity which is critical to get global approvals. Samsung BioLogics has been striving to establish a system to completely prevent even tiny data errors that may occur during the whole production process of biopharmaceuticals from the establishment of the company and to set it up as a corporate culture.

TH Kim also said “We will continue to do our best to continuously innovate ourselves so that clients and patients can receive high quality medical products in a timely manner”

Samsung BioLogics will be attending the BIO International 2018 Conference in Boston June 4-7, 2018, as an exhibitor for the 8th year in a row to pursue additional contracts.

https://bit.ly/2x8XZvf

BioMarin gets FDA OK for PKU med

BioMarin Pharmaceutical announced that it received standard approval from the U.S. Food and Drug Administration for Palynziq Injection to reduce blood phenylalanine concentrations in adult patients with phenylketonuria – PKU- who have uncontrolled blood Phe concentrations greater than 600 micromol/L on existing management. Palynziq, a PEGylated recombinant phenylalanine ammonia lyase enzyme, is the first approved enzyme substitution therapy to target the underlying cause of PKU by helping the body to break down Phe. Palynziq is BioMarin’s second approved treatment for this important condition. PKU is a rare genetic disease that manifests at birth and results in a variety of cumulative toxic effects on the brain. PKU affects approximately 1 in 12,500 live births in the United States each year. PKU is marked by an inability to break down Phe, an amino acid that is found in all forms of protein. Left untreated, high levels of Phe become toxic to the brain and may lead to serious neurological and neuropsychiatric-related issues, impacting the way a person thinks, feels, and acts. Due to the seriousness of these symptoms, infants are screened at birth to ensure that they are diagnosed early and treated to avoid intellectual disability and other complications. Patients living with PKU require life-long management, including adherence to a challenging and severely restrictive daily diet of medical foods and formula that avoids the ingestion of Phe that is present in most foods

https://bit.ly/2s4ZdTi

SF To Distribute Opioid Addiction Treatment Directly To Drug Users

San Francisco’s mayor is introducing a first-of-its-kind plan to bring treatment for drug addiction directly to the streets. The program aims to supply drug addicts with buprenorphine, used to help people reduce or quit their opiate use and that could stop cravings immediately.

The program is a follow-up to a 2016 pilot program in which city workers went into homeless encampments to hand out buprenorphine to addicts. The city says 60 percent of the people who received the drug stayed clean.

One of them was Chris Ruffino who was an IV drug user for 30 years. “Buprenorphine saved my life, for sure,” he said.

Ruffino was homeless, addicted to opioids, and almost died five times from overdoses only to be brought back to life by Narcan injections. But he says it’s buprenorphine that finally woke him up.

“Without the buprenorphine I’d still be out here on the streets,” said Ruffino. “I’d still be doing what I was doing.”

The city’s Department of Public Health says there are more than 22,000 IV drug users in San Francisco today. Ruffino says he hopes Thursday’s announcement will give those people the same opportunity he had.

Mayor Mark Farrell committed $3 million and 10 new employees to begin heading into homeless encampments and offering people buprenorphine.

“Because this traditional setting of waiting for people that are addicted to drugs to come to doctors’ offices, to come into clinics and get the treatment they need – it’s not working, plain and simple,” said Farrell.

San Francisco is the first city in the U.S. to offer the program. Farrell says the biggest barrier to getting buprenorphine is that people have to show up to a doctor’s office and get a prescription, but they’re not doing that.

“Ultimately this is about helping these individuals, but it’s also about improving the conditions of our streets,” said Farrell. “They go hand in hand.”

Ruffino is the ultimate success story. He’s clean, lives in the East Bay with his wife, and has a full-time job helping addicts find their way.

Farrell said the money for the program would come out of the city’s general fund, and believes a third of the money could be subsidized through Medi-Cal.

The program is expected to be rolled out over the next two months. While people accustomed to seeing addicts on San Francisco streets likely won’t see a difference right away, Farrell claims when they do see the difference it will be dramatic.

https://cbsloc.al/2x9gaRo

Some Antidepressant, Bipolar, Parkinson’s Meds Linked To Dementia

New research is raising important questions as to whether certain widely-prescribed drugs may be linked to dementia.

Globally, 50 million people are living with dementia, and it’s predicted to increase around the world to 132 million by 2050.

Doctors and public health experts realize that countries need to develop preventive health policies to reduce the neurodegenerative condition.

One strategy: to swap out certain medications or have doctors think twice before prescribing them. These medicines include certain antidepressants, bipolar medications, antispasmodics taken for bladder control, as well as some Parkinson’s drugs.

The drugs in question are called known as anticholinergics. They block a specific neurotransmitter called acetylcholine in the central and peripheral nervous systems.

“We analyzed people who developed dementia and people who didn’t develop dementia,” explained Professor Chris Cox, a researcher at the University of East Anglia in the United Kingdom and one of the authors of the new report.

The study is the largest, most detailed one of its kind. Researchers poured over 300,000 medical records with millions of prescriptions, and then performed a sophisticated statistical analysis.

“So our studies for the first time had indicated a strong association between taking certain anticholinergic drugs and the risk of getting dementia,” said Fox.

The British team found the long-term use of these drugs is linked to dementia, even when the medications are taken up to 20 years before a diagnosis.

The drugs include antidepressants such as Paxil and Elavil, and medications used for bipolar disorder including Zyprexa and Seroquel.

The research also found an associated risk with medications used for bladder conditions, as well as Parkinson’s disease.

“It’s important but it’s not conclusive,” commented Dr. Barbara Sommer, a geriatric psychiatrist at Stanford Medical Center and an expert in anticholinergic medication. She cautioned that the study does not prove these drugs cause dementia. Even so, she said if you take medication, over-the-counter drugs, even supplements, you should reassess if you really need them.

“You have to measure the risk and the benefit of any medication that you take.” said Sommer.

She recommends patients review all prescription drugs, over-the-counter medicines, and supplements with a specialist. “Take them to your doctors and go over each mediation one by one asking how anticholinergic it is, if at all,” said Sommer.

While this new research found no risk with popular over-the-counter antihistamines and sleep aids, Sommer said she doesn’t think they’re off the hook. Previous research shows compelling evidence the long-term use of these drugs may be problematic.

Diphenhydramine is the active ingredient found in Benadryl and other over-the-counter sleep aids. Sommer explained how drugs that contain diphenhydramine get into the brain and sit there for a little while.

“I just think it would be better to use another medication if possible,” she advised.

Patients should not to stop any medication without first talking to their doctor. “Some may have withdrawal symptoms or other adverse effects,” cautioned Fox.

To reduce the chances of dementia in the meantime, researchers advise to exercise, keep a healthy weight, don’t smoke or drink too much alcohol, control your blood pressure, and keep a good social network.

For a list of anticholinergic drugs and how they rate: CLICK HERE

https://cbsloc.al/2koYW9I