Parents denied access to their children’s medical records by law

A Coralville, Iowa father recently found out he will no longer have access to his 12-year-old daughter’s medical records, so he asked KCRG’s I9 investigative team to investigate.

Kevin Christians, of Coralville, said a letter triggered his concerns alerting him he was losing access to his daughter’s medical records.

At the University of Iowa Hospitals and Clinics, parents are no longer able to see test results, messages from doctors and other information once a child turns 12.

The letter said the hospital wants children at that age to be more active in their own health care. Christians said he believes 12 is too young to keep parents in the dark.

“If you get locked out of being able to see the health care records for your child, that makes parenting even that much more difficult, in my opinion,” said Christians.

The letter from UIHC does not give the full explanation. I9 found the practice stems from federal law and all hospitals have a similar policy but not all of them start at the same age.

I9 has discovered one medical organization where the age is 10.

https://www.kcci.com/article/parents-denied-access-to-their-childrens-medical-records-by-law/26032172

Prostate cancer survivors now live longer than average man if disease caught early

Prostate cancer survivors live longer than men without the disease – if symptoms are caught early, new figures show.

Experts said the statistics suggest a diagnosis of the disease could act as a ‘wake up call,’ with such patients more likely to keep a close eye on their own health, and attempt to improve their lifestyles.

The figures from the Office for National Statistics (ONS) are the first robust estimates showing how survival from a range of cancers varies, depending on when it is diagnosed.

When prostate cancer was spotted early – at stage one – men had five-year survival rates 0.5 per cent higher than men of the same age in the general population.

https://www.telegraph.co.uk/news/2019/01/24/prostate-cancer-survivors-now-live-longer-average-man-long/

High-res genetic map by Amgen’s deCODE offers new insight for disease research

Two mechanisms contribute to human DNA diversity: genetic recombination and de novo mutations, or new gene variants that are not inherited. With a new genetic map developed using whole-genome sequence data, scientists at Amgen’s genomics research subsidiary deCODE Genetics have uncovered patterns in the two processes, offering fresh insights for future medical research.

Drawing on sequence data from more than 155,000 Icelanders, the deCODE researchers examined the location, rate and connection between the two key drivers of human evolution. “What we show in this paper is how tightly linked recombinations and new mutations are,” Kari Stefansson, CEO of deCODE and the senior author of the study published in Science, told FierceBiotechResearch.

The genetic map has a resolution of 682 base (or nucleotide) pairs. That’s likely as high a resolution as any gene map has been and almost “as much as theoretically possible,” Stefansson said.

Stefansson and colleagues pinpointed the exact locations where 4.5 million exchanges of genetic material between chromosomes from parents happen and found more than 200,000 new mutations. As it turns out, most chromosomal crossovers occur in so-called hot spots, which cover less than 2% of the genome.

The most important finding of the study, Stefansson said, is that mutations don’t appear to happen randomly, as scientists have long assumed. “In about a thousand bases flanking the sides of recombinations, a mutation rate is increased almost 50-fold,” Stefansson said. This suggests that crossovers do have a role in the formation of new mutations.

“The classic premise of evolution is that it is powered first by random genetic change. But we see here in great detail how this process is in fact systematically regulated—by the genome itself and by the fact that recombination and de novo mutation are linked,” Stefansson said in a statement.

Moreover, the team identified 35 loci affecting recombination rate and location. Women appear to contribute more to recombination and men to de novo mutation—a common cause of rare diseases of childhood. Recombination rate also increases with the age of the mother but doesn’t appear to be affected by the father’s age, the study showed.

While the current study is not about any specific mutation that contributes to a genetic disease, Stefansson said it sheds light on basic processes of evolution and generation of new diversity while also laying the groundwork for future research on human health.

Iceland-based deCode has largely kept a low profile since it went bankrupt and was bought up by Amgen in 2012 for a handsome $415 million. Soon after that, Amgen spun out part of deCODE’s services that provides genetic diagnostic services to physicians and patients into a separate firm called NextCODE, which itself was later scooped up WuXi AppTec to form what’s now known as WuXi NextCODE.

After those transactions, Amgen was left with deCODE’s library of hereditary information and its ability to analyze large-scale sequence data to help with its drug R&D efforts. Since then, deCODE has focused on genome research. Back in 2015, the company turned up four papers in Nature Genetics, laying out the technology that enabled researchers to collect whole-genome sequencing data as well as some potential therapeutic targets.

The latest research from deCODE could provide “opportunities to make further discoveries on variants that are associated with disease,” Stefansson said. “It is a study that yields background information that makes it easier for us to find drug targets.”

Stefansson also points to the possibility of using the findings to aid in cancer research. “Our study of new mutations and recombinations could be used as a model to try to understand the mutations and genomic rearrangements that happen in cancers,” he said.

https://www.fiercebiotech.com/research/high-res-genetic-map-by-amgen-s-decode-shows-a-pattern-mutations

Vertex strikes deal with Merck KGaA to grow CRISPR/Cas9 toolkit

Vertex has regained limited rights to two DNA-dependent protein kinase (DNA-PK) inhibitors from Merck KGaA. Merck licensed the drugs and other assets for $230 million upfront two years ago but has now granted Vertex the right to use them in certain gene-editing applications.

Germany’s Merck bagged the DNA-PK inhibitors to boost its oncology pipeline and has since wrapped up a phase 1 study of the more advanced candidate, M9831, in subjects with advanced solid tumors. However, researchers have also used DNA-PK inhibitors to modulate mechanisms that repair DNA breaks and thereby improve CRISPR/Cas9 genome editing.

Vertex lost the right to use M9831, formerly known as VX-984, and the other preclinical DNA-PK drug in gene-editing applications when it licensed the assets to Merck in 2017. But it has now returned to Merck to gain certain rights to use the molecules.

The new deal gives Vertex the right to use the two DNA-PK inhibitors in gene-editing applications in six specific, but publicly undisclosed, genetic disease areas. Merck retains the rights to the drugs in all other diseases, including oncology, and can use or license them in gene-editing applications outside of those covered by the agreement. Vertex has an option to add more indications to the agreement.

Vertex has put together an undisclosed package of upfront, milestone and royalty payments to land the deal. In doing so, Vertex has again suggested that it sees CRISPR/Cas9 gene editing as important to its future. Vertex has a clinical-phase gene-editing stem cell therapy, CTX001, through its deal with CRISPR Therapeutics.

For Merck, the deal serves as a way to generate immediate and potentially ongoing revenues from a pair of assets while retaining the chance to advance them in oncology. Merck thinks inhibition of the DNA-PK enzyme could make DNA-damaging agents such as radiotherapy and chemotherapy more effective.

https://www.fiercebiotech.com/biotech/vertex-strikes-deal-merck-kgaa-to-grow-crispr-cas9-toolkit

$20B from Humira? Not there yet, don’t count on it in 2019 either: AbbVie CEO

Analysts and executives had predicted that Humira would be the first drug to ever top $20 billion in annual sales by 2018. But as AbbVie’s whole-year tally shows, the rheumatoid arthritis superstar didn’t cross that target off its wish list—and it may not do so in 2019, either.

The final 2018 figure came in so close—only a couple million dollars left to fill. But still, $19.94 billion is not $20 billion. And based on executives’ predictions for 2019, that number will drop by $1 billion instead of rising as previously expected.

A strong dollar left a dent in 2018’s reported number, but the bigger damage came from “direct biosimilar competition in certain international markets,” according to AbbVie. Outside of the U.S., Humira’s fourth-quarter sales dropped 17.5% to $1.30 billion, dragging the quarter’s total to $4.92 billion, which missed analyst expectations of around $5.03 billion.

Back on the company’s third-quarter earnings call in early November, AbbVie Chairman and CEO Richard Gonzalez projected sales erosion of around 26% to 27% ex-U.S. in 2019. Now that expected cut to sales has been increased to 30%—excluding any unfavorable impact from foreign exchange rates—according to newly minted CFO Robert Michael on the company’s fourth-quarter earnings call.

That means about $2 billion in sales will be lost in 2019, and a projected 7% growth in the U.S. will only compensate by $1 billion. Run the math, and you’ll get that global Humira sales will drop $1 billion this year. That, in turn, will just make it even harder for Humira to reach $21 billion in 2020 sales, a target executives put forward back in October 2017.

On Friday’s fourth-quarter call, Gonzalez said he’d like to see how the market plays out. “In the end, if we look at the long-term targets we put in place, we still feel confident […] whether or not one product is slightly different than another,” he said.

Humira losing exclusivity has long been haunting AbbVie investors; after all, the drug accounted for about 60% of the company’s total revenue. Consider this: It was only mid-October when Humira copycats from Biogen, Amgen, Novartis’ Sandoz and Mylan launched in Europe, and they’ve already caused double-digit losses for AbbVie’s drug in the region.

“We have long been planning and preparing for the event that is now upon us,” Gonzalez said on the call. But despite some unexpectedly aggressive discounting in Europe, his message to concerned investors is that the company’s strategy is working, and it’ll be able to absorb the impact from biosimilar assault.

But its fourth-quarter numbers don’t look good on that front. Both fourth-quarter earnings per share of $1.90 and total sales of $8.31 billion came in below consensus estimates, the first time since the fourth quarter of 2013, according to data cited by Credit Suisse analyst Vamil Divan, M.D., in a Friday note to investors.

At least AbbVie can count on some growth from the U.S. market, where Humira sales jumped 9.1% in the fourth quarter. The company has fended off almost all biosimilar launches in the U.S. to late 2023, recently adding new settlements with Momenta, Pfizer and, on Friday, Coherus BioSciences, leaving Boehringer Ingelheim the only major player still fighting AbbVie’s patent in Humira’s largest market.

Biosimilar competition has been on the company’s mind, and its countermeasure, according to Gonzalez, is to build a strong pipeline. That plan just took a major blow as AbbVie pulled the plug on a phase 3 trial of its troubled cancer drug Rova-T in small-cell lung cancer and registered $4.12 billion in impairment charges.

Plus, its Johnson & Johnson-partnered cancer blockbuster Imbruvica just flunked a pancreatic cancer trial, nixing the pair’s hope to add a seventh indication to the drug’s label. For 2019, AbbVie expects Imbruvica sales to reach $4.4 billion, compared with $3.6 billion in 2018.

The company is now guiding 2019 EPS at $8.65 to $8.75, or 10% growth at the midpoint. Gonzalez touted the company’s ability to pull that off because “this year many of our key pipeline assets will be at the very early stages of their launch trajectory, and therefore providing minimal upside to the biosimilar impact.”

https://www.fiercepharma.com/pharma/20b-from-humira-we-re-not-there-yet-and-don-t-count-it-2019-either-abbvie-ceo

Humira biosimilars catch fire in Europe; could take half the market in a year

AbbVie investors have been bracing for the mother of all patent cliffs—the loss of exclusivity on the company’s $18-billion-per-year Humira—and now its impact is starting to come into view. The mass exodus from the branded product to biosimilars confirms what AbbVie’s executives have already admitted, which is that their initial estimate of a 20% revenue loss in Europe by 2020 was way too optimistic.

That’s one main takeaway from a new Bernstein report on biosimilar adoption rates. Humira biosimilars just hit the market in Europe in October, but pickup has been so strong that Bernstein analyst Ronny Gal took one look at the first full month of sales data and confirmed that the hit on AbbVie’s revenues will come earlier than expected.

“The adoption is essentially all in Germany, with some early noise in France and Italy,” Gal said in a video he posted for investors. With more conversions from brand to biosimilar to come in regions like the United Kingdom, Latin America and Scandinavia, he said it’s reasonable to expect “50% adoption of Humira biosimilars by volume within the first year” of its launch.

AbbVie executives said during the company’s fourth-quarter earnings call today that they expected $2 billion in lost sales outside the U.S. from Humira biosimilar competition this year. AbbVie Chairman and CEO Richard Gonzalez had previously said that he was expecting a total revenue loss in all countries outside of the U.S. of 26% to 27% in 2019 alone, but today the company upped that estimate to 30%.

By Gal’s calculations, the 26%-plus ex-U.S. revenue loss means AbbVie is expecting Humira biosimilars to erode European sales by about 40%.

There are four Humira biosimilars on the market in Europe, with two more to come. So far, Gal said, the main winner is the Biogen-Samsung joint venture, Bioepis, with its low-cost version of the drug, Imraldi. It has taken the biggest share of the European market for Humira biosimilars so far, according to Gal, with Amgen’s Amgevita following close behind.

Gal also reported rapid adoption of other biosimilars in Europe, including breast cancer treatment Herceptin. Copies of that Roche blockbuster have been on the market there for 7 months, but the adoption rate has already hit 14%—making it a faster conversion to biosimilars than what was seen with Roche’s Rituxan, Gal wrote.

The loss of exclusivity on Rituxan, Herceptin and Roche’s other cancer blockbuster, Avastin, has put a cumulative $20 billion in annual sales at risk. During the third quarter of 2018 alone, European sales of Rituxan fell 49% to CHF 206 million ($207 million).

Now Roche is facing new Rituxan competition in the U.S., with the recent FDA approval of Truxima, a biosimilar version from Celltrion and Teva. Then-Roche Pharmaceuticals chief Daniel O’Day predicted during the company’s third-quarter earnings conference call that sales erosion in the U.S. wouldn’t be as fast as it has been in Europe. But he conceded that any action on drug prices from President Donald Trump or Democrats in Congress could accelerate the pickup of biosimilars.

In the new Bernstein report, Gal noted that biosimilars haven’t been embraced in the U.S. to the extent that they have in Europe, but he predicted that could change. Biosimilar versions of Johnson & Johnson’s Remicade have only captured 6% of the market in the U.S., Gal said. Several market forces are fighting biosimilar adoption, not the least of which is fear on the part of insurers that if they require biosimilars, physicians will switch patients to more pricey branded drugs instead, he wrote.

So what might liven up the U.S. market for biosimilars? In October, prompted by new rules from the Center for Medicare and Medicaid Services, UnitedHealthcare introduced “step therapy” requirements for some expensive biologics covered by its Medicare plans, including Remicade. Those requirements start patients on lower-cost medications before they can progress to pricier alternatives, and commercial insurance plans could follow suit with similar tactics to drive biosimilar adoption, Gal said.

The introduction of a biosimilar version of Amgen’s Neulasta in the U.S. indicates that American payers are already ramping up their efforts to adopt the low-cost alternatives. That product, Mylan’s Fulphila, captured 4% of the market in its first five months, Gal noted. That’s not bad, he added, considering it has taken two years for biosimilars of Remicade to grab 6% of the U.S. market.

https://www.fiercepharma.com/pharma/humira-biosimilars-catch-fire-europe-and-could-take-half-market-a-year-report

Lipocine Files IND To Evaluate LPCN 1144 In Biopsy Confirmed NASH

Lipocine Inc. (NASDAQ: LPCN), a specialty pharmaceutical company, today announced that it has filed an Investigational New Drug application (“IND”) with the U.S. Food and Drug Administration (“FDA”) to initiate a Phase 2 clinical study of LPCN 1144 in non-alcoholic steatohepatitis (“NASH”) with biopsy confirmed NASH subjects.

“This IND filing is an important step forward as we pursue further evaluation of LPCN 1144 in biopsy confirmed NASH patients for the treatment of NASH. There is currently no FDA approved product for the treatment of NASH and we believe our well-tolerated oral LPCN 1144 is well positioned to mechanistically address this disease and provide additional unmet benefits,” said Dr. Mahesh Patel, Chairman, President and Chief Executive Officer of Lipocine.

https://www.marketscreener.com/LIPOCINE-INC-16085349/news/Lipocine-Files-IND-To-Evaluate-LPCN-1144-In-Biopsy-Confirmed-NASH-Subjects-27915464/