Sanders Targets Catalyst Over Price of LEMS Drug Firdapse

U.S. Sen. Bernie Sanders has a history of taking on the pharmaceutical industry and prices for prescription medicines that he sees as unjust. This week, the Vermont senator has found a new target, Florida-based Catalyst Pharmaceuticals.

On Monday, Sanders sent a letter to Patrick J. McEnany, chairman and chief executive officer of Catalyst Pharmaceuticals, in regards to the $375,000 price tag for its recently-approved drug, Firdapse (amifampridine). In November 2018, after a previous rejection, Catalyst won approval from the U.S. Food and Drug Administration for the oral drug, as a first-ever approved treatment for Lambert-Eaton myasthenic syndrome (LEMS). The disorder affects the connection between nerves and muscles and causes weakness and other symptoms in affected patients. It affects approximately one in 100,000 people in the United States and about 3 million people globally. Before finally achieving regulatory approval in the United States, Catalyst had co-marketed marketed a LEMS treatment in Europe it had licensed from BioMarin Pharmaceuticals. Analysts have pegged Firdapse to bring in about $325 million in annual revenue for Catalyst by 2025.

Sanders’ ire was raised over the price tag Catalyst slapped on Firdapse, which had a long road for regulatory approval. For 20 years, Sanders said LEMS patients were able to receive the same drug, known as 3,4-DAP, for free from Jacobus Pharmaceutical under the Food and Drug Administration’s compassionate use program. For years, the family-owned Jacobus has given away treatments to 200 patients battling LEMS, as BioSpace reported in 2016. Jacobus offset giving away the treatment with other drugs it manufactures. Through the compassionate use program, Jacobus was able to sidestep some of the regulatory hurdles associated with marketing a drug. The FDA’s compassionate use program allowed patient access to the Jacobus drug outside of clinical testing, despite the fact it had not been approved by the regulatory agency.

Sanders said the price that Catalyst is charging for Firdapse has many patients worried they will not be able to afford the FDA-approved Firdapse.  On his Senate website, Sanders included video of a conversation he had with a LEMS patient, Rebecca Hovde of Wellman, Iowa, who expressed great concern with the price. Hovde told Sanders that when people are unable to continue to use the Jacobus product, they will “just go to bed.”

Sanders called the $375,000 price tag for Firdapse exploitative. He questioned how many patients will suffer or die due to the cost of the drug.

“By setting such a high price and forcing production and distribution of the older, inexpensive version to cease, you are threatening access that patients had to a cheap version of this product, and handing a completely unwarranted bill to American taxpayers,” Sanders wrote in the letter to Catalyst.

A harsh critic of drug prices, Sanders has used the bully pulpit to take on multiple companies over pricing. In 2017, he chastised Illinois-based Marathon Pharmaceuticals over the pricing of its treatment for patients with Duchenne muscular dystrophy. Earlier that year, Sanders called out Ariad Pharmaceuticals for systematically increasing the price of its leukemia drug, Iclusig, more than 73 percent since the drug’s launch in 2012.

The price of pharmaceuticals is a particularly hot topic now on Capitol Hill, as lawmakers are closely examining the way companies and the insurers set prices. Hearings have been held in both the Senate and House of Representatives and last week, HHS Secretary Alex Azar floated a proposal that could change the way drug rebates are offered in order to benefit consumers.

In the letter to Catalyst, Sanders requested information on what the company is charging patients, private insurers and government payers for the medication.

“The egregious price set by Catalyst cannot be allowed to stand.  Patients in America should not be allowed to suffer or die because of the greed of a drug company. If Catalyst does not substantially lower the price of this medication, Congress must act to ensure it is affordable for every patient,” Sanders said.

Catalyst has not responded to Sanders, according to reports. Shares of Catalyst fell nearly 6 percent Monday to $2.37 per share on news of Sanders’ letter. The stock is down slightly in premarket trading this morning.

https://www.biospace.com/article/bernie-sanders-calls-out-catalyst-pharmaceuticals-over-price-of-lems-drug-firdapse/

Hospital Prices Rising Much Faster Than Physician Fees

Previous reports have pointed to physician prices as a key driver of healthcare costs among the privately insured, but a new analysis shows that hospital prices have risen much faster than physician prices.

Zack Cooper, PhD, an associate professor of health policy in the School of Public Health and of economics in the Department of Economics, both at Yale University, in New Haven, Connecticut, and colleagues analyzed claims data from the Health Care Cost Institute (HCCI) from 2007 to 2014 using prices actually paid — those that insurers negotiated with clinicians.

They found that for inpatient care, hospital prices grew 42% in that time while physician prices grew 18%. For hospital-based outpatient care, hospital prices grew 25% while physician prices grew 6%. All prices were adjusted for inflation to 2014 dollars.

Cooper and colleagues report their findings in an article published onlineFebruary 4 in Health Affairs.

The researchers also compared hospital prices with physician prices for four common services: cesarean delivery, vaginal delivery, hospital-based outpatient colonoscopy, and knee replacements. They found that the facility component of the total increase in price for the procedures ranged from 76.9% of the total price increase for colonoscopies to 96.8% of the total price increase for knee replacements.

Total health spending in the privately insured market in the United States grew by nearly 20% from 2007 to 2014, according to the Henry J. Kaiser Family Foundation.

“Our work suggests that efforts to reduce health care spending should be primarily focused on addressing growth in hospital rather than physician prices,” the authors write.

Addressing hospital price growth could include antitrust enforcement, use of reference pricing (financial incentives to encourage consumers to shop for healthcare), and incentives to encourage physicians to make more cost-effective referrals, the authors suggest.

They note that “vertically integrated physicians often refer their patients to more expensive locations.”

The researchers acknowledge several limitations of the study. Among them are that the HCCI data they used for their study only covered about 28% of employer-sponsored people in the United States. Also, data for people insured by companies other than Aetna, Humana, and UnitedHealthcare were not included.

In addition, hospital price measures were used to calculate costs of implants and medical devices. Therefore, it is possible that the increase in knee replacement costs, for example, reflected the cost of knee implants themselves.

The authors write that they believe this is the first study to systematically compare hospital price and physician price growth rates over time.

The authors’ conclusion is the same as that of another study of insurance claims data from the California Public Employees’ Retirement System. That study, published in 2017, showed that between 2004 and 2011, the compound annualized growth rate for physician prices for infant deliveries was 6% a year, compared with 17% for hospital prices.

The project received support from the Commonwealth Fund and the National Institute for Health Care Management Foundation. A coauthor was previously affiliated with the HCCI. The other authors have disclosed no relevant financial relationships.

Health Aff. Published online February 4, 2019. Abstract

https://www.medscape.com/viewarticle/908784

Altria’s Snuff Safer Than Cigarettes, FDA Panel Agrees

Tobacco giant Altria wants to tell the world that its Copenhagen snuff is a safer alternative to smoking cigarettes, and on Thursday an FDA advisory panel agreed that the science appears to support the claim.

Following a Feb. 6-7 hearing, the Tobacco Products Scientific Advisory Committee (TPSAC) voted 8-0, with 1 abstention, that the manufacturer’s proposed “modified risk” claim for Copenhagen Snuff Fine Cut is scientifically accurate.

Altria has petitioned to include the following on the product’s label: “IF YOU SMOKE, CONSIDER THIS: Switching completely to this product from cigarettes reduces risk of lung cancer.”

“This is an important step for tobacco harm reduction,” said Joe Murillo, vice president of regulatory affairs for Altria Group. “It is critical that adult smokers have accurate information that will help them switch to non-combustible tobacco products.”

The FDA, since 2009, has had the regulatory authority to grant modified risk tobacco product (MRTP) status to those proven to pose lower health risk than traditional combustible cigarettes, but it has never done so.

As of late last year, federal health officials had denied or delayed close to 20 MRTP applications from mostly smokeless tobacco products.

At the two-day public hearing, the advisory panel heard testimony on two pending MRTP applications – Altria’s labeling application for Copenhagen Snuff Fine Cut and a request by “snus” smokeless tobacco manufacturer Swedish Match. TPSAC members reached no consensus on whether the latter’s proposed reduced-risk claims were justified.

In his TPSAC presentation to the committee, Murillo noted that snuff similar to the Copenhagen product has been marketed for almost 200 years.

“Products like Copenhagen snuff are not risk free. But they are substantially less hazardous than cigarettes — specifically, as it relates to lung cancer,” he said. “This is the overwhelming consensus of the scientific, medical and public health communities. It is a scientific fact, but adult smokers don’t understand this fact.”

He said that in survey after survey, most adult smokers erroneously reported that smokeless tobacco products were as harmful or more harmful than cigarettes.

According to the CDC, about 128,000 smokers die of lung cancer every year. Murillo noted that the 23 million adult smokers in the U.S. would be likely to use a reduced harm tobacco product if they actually believed it was safer than combustible cigarettes.

But he said more than half of these smokers don’t know that snuff and other smokeless tobacco products represent reduced harm alternatives to cigarettes.

Murillo conceded that the appeal of Copenhagen snuff and other snuff products is limited.

“Consumers of Copenhagen snuff are by and large adult white males who are 35 years of age and older. We recognize that no single product or category of products will appeal to all adult smokers who are looking for reduced risk alternatives. That is why we believe there must be a portfolio of products with FDA-authorized modified risk claims. We are starting with Copenhagen snuff,” he said. “Authorizing this claim would be an important first step toward solving the dilemma faced by adult smokers. We can give them a reason to switch.”

By emphasizing the older age group, Murillo may have been seeking to distance Altria’s product from Swedish Match’s snus. When the TPSAC discussed the latter product on Wednesday, several panel members hesitated to support the MRTP designation because they feared it could make snus more attractive to young people.

At the close of the Thursday meeting, TPSAC chair Robin Mermelstein, PhD, said the consensus among committee members was the reduced harm claim for Copenhagen snuff was reasonably interpreted by the public, and not misinterpreted to mean that the product was risk free.

“I am hearing a consensus that this was an understandable statement. It was clear. There may be things that need to be tweaked, but there is no great concern that it is a harmful message. I think it is a reasonable place to start in messaging,” she said.

The FDA is not obliged to follow recommendations by its advisory committees, but it usually does.

https://www.medpagetoday.com/primarycare/smoking/77895

HiberCell launches with $60M+ to combat cancer relapse and metastasis

HiberCell, a new startup focused on preventing cancer metastasis and relapse, launched Thursday with research out of Mount Sinai and $60.75 million from the likes of ARCH Venture Partners, Hillhouse Capital and 6 Dimensions Capital.

The company reckons it is the first to exclusively focus on detecting and treating tumor dormancy that can lead to cancer spread.

“While we have made great strides in treating primary tumors, the unfortunate and painful truth is that relapsed or metastatic cancer still claims the lives of most people with cancer, even when their primary tumor has been successfully treated,” said Julio Aguirre-Ghiso, Ph.D., HiberCell’s scientific founder and a professor at The Tisch Cancer Institute at the Icahn School of Medicine at Mount Sinai.

New York-based HiberCell is building on “converging data from multiple labs”—including from Aguirre-Ghiso’s lab at Mount Sinai—showing the prevalence of dormant disseminated tumor cells (DTCs) that remain undetected in the body for long periods of time, as well as how they behave as the “seeds of metastasis.”

It’s not a new idea, Rigby said: “If you think about dissemination, this notion of solitary cells breaking away from the primary tumor, we’ve known that since the ‘50s. This is not a novel mechanism. I think what is novel is our ability to understand the role of those cells when they take up residence in a different niche [in the body.]”

First up, the company is building its knowledge base, figuring out where dormant DTCs live, how they survive and what “wakes them up” again to cause metastatic spread. The hope is that by understanding how dormant DTCs drive metastasis, HiberCell will be able to identify, isolate, genomically and transcriptionally annotate them and, eventually, drug them.

Rigby sees two ways to attack DTCs: by eradicating them or by inducing dormancy.

The first requires the ability to target pathways that are overexpressed in DTCs, which HiberCell has managed to do in animal studies, Rigby said. The second would be a “paradigm shift” in cancer treatment—”the notion of maintenance, maintaining someone’s cancer index, if you will,” he said.

And both could be combined in a one-two punch that would force DTCs into a “very deep sleep state” before being hit with a drug that would eliminate them altogether.

HiberCell will be taking lab space in New York City and is starting out with a team of six. It hopes to boost that number to 10 or 12 by the end of the year. An important piece is its collaboration consortia, which was put in place so it can get its hands on patient material and really pin down the foundational biology, Rigby said.

https://www.fiercebiotech.com/biotech/hibercell-launches-60m-to-combat-cancer-relapse-and-metastasis

Gossamer Bio 17.25M share IPO priced at $16.00

The deal size was upsized

https://thefly.com/landingPageNews.php?id=2861488

Intellia Therapeutics initiated at BTIG

Intellia Therapeutics initiated with a Buy at BTIG. BTIG analyst Amanda Murphy initiated Intellia Therapeutics with a Buy rating and $20 price target. Murphy said Intellia’s in vivo pipeline is the “most well-characterized” of the public gene editing companies, and she sees upside from wholly-owned programs in oncology and CNS.
https://thefly.com/landingPageNews.php?id=2861463

FDA details spike in reports of breast implant-associated lymphomas

The FDA has seen a jump in the number of cases of a rare type of lymphoma linked to breast implants, which the agency says results from its efforts to educate stakeholders of the risks, as well as its work to encourage patients and providers to file reports.

The cases are related to breast implant-associated anaplastic large cell lymphoma, a type of non-Hodgkin’s lymphoma that can develop in the scar tissue surrounding an implant. The agency first alerted the public to the risks of the disease in 2011, and has been tracking known cases of BIA-ALCL, deaths and risk factors.

As of September 2018, the FDA has received 660 adverse event reports regarding cases in the U.S., linked to 457 unique cases of the disease and nine patient deaths, the agency said. The total represents an increase of 246 new reports compared to the year before.

Based on the agency’s education efforts, an agency official said these types of growth “are to be expected and may include past cases that were not previously reported to the FDA.”

“We want to provide patients with the most up-to-date information about the variety of breast implants available so that patients and providers can have thorough and thoughtful discussions weighing the benefits and risks of different products,” Binita Ashar, M.D., director of CDRH’s Division of Surgical Devices, said in an agency statement.

While the FDA’s medical device reporting system allows patients, providers and manufacturers to each file their own reports, even if it’s about the same case, not every report captures thorough information—such as the type of breast implant, reasons for use and characteristics such as surface texture.

This “makes it more difficult to know if any particular breast implant characteristic is associated with BIA-ALCL or if higher reports of BIA-ALCL are simply due to higher implantation rate of a particular manufacturer,” Ashar said.

For transparency, the agency provides a breakdown of the raw data it has collected on its website. The FDA also reviews medical literature and registry data from confirmed patients to get a bigger picture of the risks involved.

In addition, the agency issued a letter to providers, including primary care physicians and gynecologists, encouraging to learn about BIA-ALCL and its risks.

“We want to ensure that all providers who treat patients with breast implants have information regarding identification, diagnosis and treatment,” Ashar said. “Patients are more likely to seek routine care from primary care physicians, gynecologists and others besides their treating plastic surgeon.”

https://www.fiercebiotech.com/medtech/fda-details-spike-reports-breast-implant-associated-lymphomas