Johnson & Johnson scores another talc win with California mesothelioma verdict

Johnson & Johnson just chalked up another win against allegations that its talcum powder causes cancer.

A jury in Los Angeles sided with the company in the case of Amy Fong, who alleged J&J’s talc contained asbestos and caused her to develop mesothelioma, Courtroom View Network reports. The company still faces almost 17,000 cases over talc safety.

A J&J spokeswoman said the company sympathizes with “anyone suffering from cancer, and we understand patients and their families are seeking answers.” But the “facts are clear—Johnson’s Baby Powder is safe, does not contain asbestos nor does it cause cancer, as reflected in more than 40 years of scientific evidence,” she said. Monday’s result is the third straight talc verdict in favor of J&J.

The decision marks another win for J&J as it faces around 16,800 lawsuits alleging harm from talc. In the face of the litigation, the drugmaker has always maintained its products are safe.

But earlier this year, the company recalled one lot of the powder out of an “abundance of caution” after the FDA detected “sub-trace” levels of asbestos in one bottle. J&J then tested the bottle and others and found the batch to contain no asbestos. For its part, the FDA stood by the result.

Throughout the litigation, even as J&J has scored numerous trial wins, various juries have also ordered the drugmaker to pay billions of dollars in verdicts. J&J has said it’ll appeal its losses and has successfully overturned numerous prior verdicts. J&J’s spokeswoman said that every verdict that’s been through the appeals process has been overturned.

In the Fong case, J&J lawyers raised concerns of “prejudicial irregularities” throughout the trial. They said plaintiffs’ lawyers “resisted answering” questions about testimony from an expert witness on asbestos testing in talc and more.

Aside from talc, the company also faces litigation over opioids, its antipsychotic medicine Risperdal, plus other drugs and devices; it’s offered to pay $4 billion to settle its opioid litigation. To defend against those issues and others, J&J spent around $900 million on legal costs in the first half of the year, Axios reported in October.

https://www.fiercepharma.com/pharma/johnson-johnson-scores-talc-win-california-mesothelioma-lawsuit

Prevail Therapeutics Gets FDA Orphan Drug Tag for Dementia Treatment

Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) for the company’s gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation (FTD-GRN). PR006 is designed to increase progranulin levels in FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

Orphan Drug designation is granted by the U.S. Food and Drug Administration to drugs or biologics intended to treat a rare disease or condition, which is a disease or condition that affects fewer than 200,000 individuals in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

“FTD with a GRN mutation is characterized by progressive difficulties in decision-making, behavior and language,” said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Prevail. “With no approved treatments for FTD, there is an urgent unmet need for therapies that slow or stop this disease. Orphan drug designation is an important milestone as we prepare to bring PR006 into the clinic in the first half of 2020.”
https://www.biospace.com/article/releases/prevail-therapeutics-announces-fda-orphan-drug-designation-granted-to-pr006-for-the-treatment-of-patients-with-frontotemporal-dementia-with-a-grn-mutation/

Prevail Therapeutics Gets FDA Orphan Drug Tag for Dementia Treatment

 Prevail Therapeutics Inc. (Nasdaq: PRVL), a biotechnology company developing potentially disease-modifying AAV-based gene therapies for patients with neurodegenerative diseases, announced today that the U.S. Food and Drug Administration (FDA) has granted Orphan Drug designation (ODD) for the company’s gene therapy candidate, PR006, for the treatment of patients with frontotemporal dementia (FTD) with a GRN mutation (FTD-GRN). PR006 is designed to increase progranulin levels in FTD-GRN patients by delivering a healthy GRN gene using an AAV9 vector.

Orphan Drug designation is granted by the U.S. Food and Drug Administration to drugs or biologics intended to treat a rare disease or condition, which is a disease or condition that affects fewer than 200,000 individuals in the United States. Programs with Orphan Drug status receive partial tax credit for clinical trial expenditures, waived user fees and eligibility for seven years of marketing exclusivity.

“FTD with a GRN mutation is characterized by progressive difficulties in decision-making, behavior and language,” said Asa Abeliovich, M.D., Ph.D., founder and chief executive officer of Prevail. “With no approved treatments for FTD, there is an urgent unmet need for therapies that slow or stop this disease. Orphan drug designation is an important milestone as we prepare to bring PR006 into the clinic in the first half of 2020.”

https://www.biospace.com/article/releases/prevail-therapeutics-announces-fda-orphan-drug-designation-granted-to-pr006-for-the-treatment-of-patients-with-frontotemporal-dementia-with-a-grn-mutation/

Combining energy treatments for optimal cosmetic outcomes

Using combination therapies with patients is widely accepted as one of the most successful treatment approaches in the aesthetic specialty.

And Michael Gold, M.D., Nashville, Tenn., says there’s a simple reason:

“Combination therapies work really well.”

Dr. Gold says he uses several devices in tandem to get the most successful outcomes for his patients and shared those this year at Cosmetic Surgery Forum 2019 in Nashville, Tenn.

One of those devices, the Venus Versa (Venus Concept) is a multi-treatment system that includes Intense Pulsed Light (IPL), radiofrequency (RF) with microneedling and bulk heating capabilities.

The Venus Versa’s IPL applicators can be used as a photofacial machine, for hair removal or as a dual-light acne treatment.

Used with its Multi-Polar RF and Pulsed Electro Magnetic Fields (PEMF) applicator, these treatments, when done in one session, bring “results [that] are pretty spectacular,” according to Dr. Gold.

“If you do a series of three of these treatments, we’re finding incredible results in wrinkles, texture, tones, tightening, improvement and acne scars or other kinds of imperfections in the skin,” he says.

For facial rejuvenation, there are three InMode technologies — Lumecca, Forma and Fractora — Dr. Gold says he examined clinically and when used together may deliver long-lasting solutions.¹

“We… published a study looking at [InMode’s] IPL which they call Lumecca, their skin tightening device which they call Forma and their RF microneedling device called Fractora,” he says.

Study authors found that after completing alternate treatments of IPL in three sessions, and continuous fractional bipolar RF in three alternating sessions, three weeks apart, a comparison of baseline, six- and 12-week results showed statistically significant improvement in wrinkles (24% at six weeks, 33% at 12 weeks), pigmentation (38% at six weeks, 62% at 12 weeks), vascular lesions (29% at six weeks, 67% at 12 weeks) and laxity (37% at six weeks, 40% at 12 weeks).

According to Dr. Gold, “The improvements in the texture-tone irregularities were spectacular and documented in the long term.”

He also says using Sciton’s Halo device — a combination of non-ablative and ablative fractional resurfacing — in conjunction with their Broad Band Light (BBL) device improves texture and tone.

For the treatment of acne, Dr. Gold was also involved in a Russian study that used Aerolase’s Neo Elite — a 1064 short pulse laser device — with low dose isotretinoin for patients with severe acne. Though noted that the treatment can have many contraindications, the study authors report that “a more current version of treatment protocols and transition to lower dosages have made it possible to reduce the likelihood of side effects of the combination therapy.”²

Dr. Gold says he is always looking for the newest ways to use combination devices, bringing his patients the best possible results.

“We constantly are working on trying to improve the devices that we have,” he says. “And that’s where we need to be thinking.”

References:

1. Gold MH, Biron JA, Sensing W. Facial skin rejuvenation by combination treatment of IPL followed by continuous and fractional radiofrequency. J Cosmet Laser Ther. 2016;18(1):2-6.
2. Gold M, Evgenievna N, Sergeevna L, Vladimirovna E, “Treatment of Moderate to Severe Acne and Post Acne Scars with 650 Microsecond 1064 nm Laser Combined with Low Dose Isotretinoin. Lasers Surg Med. 2019;51(S31):S13-14.

https://www.dermatologytimes.com/aesthetic/combining-energy-treatments-optimal-outcomes

Adamas Pharma plunges after mixed results in MS trial

“As we did not see the scale of clinical benefit we had hoped for in this study we will fully assess the potential for ADS-5102 in MS patients before determining the extent of our continued investment in this program,” says Adamas (ADMS -40.7%) CEO Neil McFarlane after mixed results in the drug’s Phase 3 trial.

The 274 mg dose showed improvement in the timed 25-foot walk after 12 weeks, but the lower did not, and neither dose showed a significant effect on secondary walking measures.

Already bearish on the name, BAML cuts its price target to $3. “Visibility needed on path forward in MSWI,” says the team.

Evercore, meanwhile, is launching a spirited defense of its $45 price target. Analyst Joel Schimmer says this marks the final of many mistakes from prior management. “The new team can execute on Gocovri for LID with a clean slate and lightly explore other pipeline opportunities.”

Shares are currently trading hands at $4.50.

https://seekingalpha.com/news/3526850-adamas-pharma-plunges-after-mixed-results-in-ms-trial

From cancer medication to antibiotic

Antibiotic-resistant bacteria are increasingly the source of deadly infections. A team of scientists from the Technical University of Munich (TUM) and the Helmholtz Center for Infection Research (HZI) in Braunschweig have now modified an approved cancer drug to develop an active agent against multidrug-resistant pathogens.

The methicillin-resistant Staphylococcus aureus (MRSA) is the source of severe and persistent infections. Some strains are even resistant to multiple antibiotics. There is consequently an urgent need for new drugs effective against MRSA infections.

“The industrial development of new antibiotics is stalling and not keeping pace with the spread of antibiotic resistance. We urgently need innovative approaches to meet the need for new infection therapies that do not lead directly to renewed resistance,” says Prof. Eva Medina, director of the HZI Infection Immunology Research Group.

New antibiotic development strategies

One promising strategy is to test the potential effect of approved drugs on bacteria. “Our focus was on a class of human proteins, called kinases, which have many inhibitors to begin with,” explains study leader Stephan Sieber, professor of organic chemistry at TUM.

In this vein, the researchers chemically modified the active ingredient sorafenib, a cancer drug that is effective against MRSA, to achieve a stronger antibiotic effect. This led to the development of PK150, a molecule ten times more effective against MRSA than the original substance.

Multiple attacks prevent the development of resistance

The potent new agent targets various unconventional structures within the bacteria. Two targets were investigated in greater detail: For one, PK150 inhibits an essential protein involved in bacterial energy metabolism. For another, it acts on the cell wall.

In contrast to previously known antibiotics such as penicillin and methicillin, which interfere with cell wall formation, PK150 acts indirectly. It knocks the protein production in bacteria off kilter. As a result, the bacteria release more proteins that control the cell wall thickness to the outside, causing the cells to burst.

In mice, PK150 has proven to be effective against MRSA in a variety of tissues. While staphylococci rapidly develop resistance to other antibiotics, the researchers did not observe the development of any resistance to PK150.

Effectiveness against biofilms and persisters

Eva Medina and Dr. Katharina Rox, a pharmacologist from the Department of Chemical Biology at HZI, showed that PK150 has favorable pharmacological properties. It can be administered as a tablet, for example, and remains stable in the body for several hours. “As a result of the chemical changes to the molecule, PK150 no longer binds to human kinases, but acts very specifically against bacterial targets,” says Sieber.

And PK 150 has another benefit: “MRSA infections are very often chronic, as the bacteria can become dormant. PK150 even kills these, as well as germs protected in biofilms,” says Prof. Dietmar Pieper, head of the HZI research group “Microbial Interactions and Processes”.

In the context of the aBACTER project, Prof. Sieber’s team is now further optimizing PK150 to enter the clinical development phase.

###

The work was funded by the European Community (via the European Research Council (ERC) and the CHEMMINE research program), the German Research Foundation (DFG) within the Cluster of Excellence Center for Integrated Protein Science Munich (CIPSM) and the SFB 1035 Collaborative Research Center, the Chemical Industry Fund, the Boehringer Ingelheim Fund, the German Center for Infection Research (DZIF), the National Science Foundation (NSF) and the National Institute of General Medical Sciences (the latter two, USA). The aBACTER project is funded by the German Federal Ministry of Education and Research (BMBF) as part of the VIP + program.

In addition to the Chair of Organic Chemistry, the Chair of Proteomics and Bioanalytics, and Radiochemistry Munich of the TUM, various working groups of the Helmholtz Center for Infection Research, the German Center for Infection Research, the German Cancer Research Center (DKFZ), Temple University (Philadelphia, USA), and Emory University (Atlanta, USA) participated in the research. Stephan Sieber is also scientifically associated with the Helmholtz Institute for Pharmaceutical Research Saarland (HIPS), an HZI site in cooperation with the University of Saarland, and the Institute for Advanced Study of the TUM.

https://www.eurekalert.org/pub_releases/2019-12/tuom-fcm121219.php

Pacira Bio reports positive results from late-stage Play study

Pacira BioSciences (PCRX) announces positive results from its Phase 3 PLAY study of EXPAREL (bupivacaine liposome injectable suspension) administered as a single-dose infiltration in pediatric patients undergoing spinal or cardiac surgeries. The study enrolled 98 patients.

Overall findings were consistent with the pharmacokinetic and safety profiles for adult patients with no safety concerns identified at a dose of 4 mg/kg.

These results will provide the foundation for the company’s sNDA in H1 2020 seeking expansion of the EXPAREL label to include children aged six and over.

https://seekingalpha.com/news/3526788-pacira-bio-reports-positive-results-from-late-stage-play-study