TransMedics up on positive heart system news

Thinly traded micro cap TransMedics Group (NASDAQ:TMDX) perks up 3% premarket on light volume on the heels of its announcement of the first successful heart transplants from DCD (donation after circulatory death) donors in the U.S. enabled by its OCS Heart System.

The device is used to resuscitate the donor heart to a normal beating rate before transplantation (based on a clinical assessment at that time).

Heretofore, DCD donors are not considered viable transplant candidates due to the potential injury to the heart once beating ceases.

https://seekingalpha.com/news/3523652-transmedics-up-3-premarket-on-positive-heart-system-news

Zealand Pharma up 6% on dasiglucagon developments

Ultra-thinly traded Zealand Pharma (ZEAL +6.3%) is up, albeit on turnover of only 3,200 shares, in apparent reaction to dasiglucagon-related news, in late-stage development for severe hypoglycemia (low blood sugar).

Earlier today the company announced a second Phase 3 study in children with congenital hyperinsulinism (CHI), an inherited disorder that causes hypoglycemia due to excess amounts of insulin secreted by the pancreas.

It also announced that the FDA has granted Rare Pediatric Disease designation to the glucagon analog for CHI which provides for the issuance of a rare pediatric disease priority review voucher following the agency’s nod. The voucher can be used for accelerated approval of a future application or it can be sold to a third party.

Shares rose 8% in Copenhagen today closing at DKK220.0.

https://seekingalpha.com/news/3523735-zealand-pharma-up-6-on-dasiglucagon-developments

Tandem down 7% on expected headwinds before Control-IQ approval

Tandem Diabetes Care (TNDM -6.9%) slumps on average volume on the heels of its presentation at the Piper Jaffray Healthcare Conference today.

Investors appear to be reacting to a temporary slowdown in business as U.S. customers wait for the FDA nod on Control-IQ, expected in the near future. The company said that it saw a slowdown in late Q3 that will extend through almost all of Q4. About 105K U.S. patients currently use their pump and 80% of those can be updated to Control-IQ (company plans to offer the update at no charge).

There is also looming competition from a new Medtronic device, expected about mid-2020.

U.S. market for insulin pumps currently 30% penetrated compared to 24% ~15 years ago (analyst comment) but the company envisions 50% penetration in the next 3-5 years driven primarily by much improved ease of use.

The company’s income is ~$4K per pump in the U.S. versus ~$2K in Europe (due to broader range of distributor responsibilities there).

Agreement with Abbott should be finalized shortly. May be about a year before commercialization begins on Libre 2.0 device. Meaningful revenue expected no earlier than 2021.

https://seekingalpha.com/news/3523714-tandem-down-7-on-expected-headwinds-control-iq-approval

Gilead’s converting Truvada PrEP users to Descovy faster than expected: analyst

One big task for Gilead Sciences’ HIV franchise in the coming year is to save some $2.5 billion in annual sales by switching as many PrEP patients as possible from Truvada to newly approved Descovy before generics hit.

So far, the company’s doing a good job.

In just two months since the FDA approved Descovy to prevent HIV, Gilead has converted about 10% of Truvada patients to the new drug, a Jefferies team led by analyst Michael Yee noted in a Friday report based on IQVIA scripts data.

For the week ended Nov. 22, Descovy’s total U.S. prescriptions hit 19,295—up about 4,000 from the week ended Oct. 11, the first after its FDA go-ahead. During the same period, Truvada scripts per week dropped by around 3,000 from between 34,000 and 35,000.

At that rate, Gilead could switch about 50% to 60% of PrEP users off Truvada by the time Teva launches its copycat in September 2020, Yee estimates. That’s well ahead of the 35% Wall Street currently expects.

Yee’s team also mapped out a scenario in which all Truvada scripts were transferred at a steady weekly rate to Descovy by Teva’s anticipated launch date, and it found that Descovy is tracking even faster than that. But of course, 100% conversion is almost impossible, and Yee attributed some of Descovy’s growth to organic expansion beyond Truvada switch-overs. Some of that is coming from tandem use with ViiV Healthcare’s Tivicay (dolutgravir) for the treatment of HIV, rather than prevention.

A recent Jefferies doctor survey also suggested the Truvada-Descovy swap would go slower than the HIV treatment switchover from TDF-to-TAF-based regimens, which now stands at about 75%. The difference between Descovy and Truvada is that, besides the shared component of emtricitabine, the newer drug uses tenofovir alafenamide (TAF), which is generally considered a safer option than the tenofovir disoproxil fumarate (TDF) used in the latter.

Gilead’s playing up that advantage in convincing doctors and patients to use the new version. The Big Biotech recently detailed data from the phase 3 Discover trial, showing significantly better bone mineral density and renal safety outcomes for Descovy patients than for those on Truvada.

The company knows what’s at stake: about $2.6 billion in Truvada annual sales is on the line. With that in mind, it has bulked up Descovy’s rep ranks, moving many sales reps off a cardiopulmonary portfolio that’s been hit by generics and retraining them to detail Descovy, Gilead’s chief commercial officer, Johanna Mercier, told investors on a conference call in October.

Mercier noted that over half of PrEP scripts are “very concentrated,” coming from a couple of thousand specialists who are also treating HIV patients. These docs have been Gilead’s initial target for the Descovy swap because they “have experienced converting from TDF to TAF and also understand the value of Descovy and its clinical profile specifically around the safety with bone and renal,” she said.

https://www.fiercepharma.com/marketing/gilead-s-converting-truvada-prep-users-to-descovy-faster-than-expected-analyst

Blockbuster in the balance: Celgene Reblozyl faces FDA panel

In its final days as an independent company, Celgene scored a win last month with the FDA’s approval of rare blood disease med Reblozyl––a potential blockbuster in some analysts’ eyes. Now, the FDA wants a closer look at Reblozyl as a treatment for a group of rare blood cancers.

An FDA advisory committee will review Reblozyl on Dec. 18 to treat patients with myelodysplastic syndromes (MDS), a suite of rare blood cancers in which mutations prevent bone marrow stem cells from making healthy blood cells, leading to chronic anemia.

“Anemia associated with MDS remains a significant area of unmet need for these patients, as current treatment options are limited, consisting primarily of medicines that stimulate the production of erythropoietin and regular RBC transfusions,” new Celgene owner Bristol-Myers Squibb said in a statement.

The FDA set an action date on Reblozyl’s application for April 4. Bristol didn’t disclose what data the FDA was set to review at the advisory committee meeting.

If Reblozly wins an MDS nod, it could reach a $2 billion in yearly sales, according to Jefferies analysts.

In early November, the FDA approved Reblozyl (luspatercept) as an anemia treatment in patients with beta thalassemia who require regular red blood cell transfusions. Though these transfusions are a lifesaving treatment, they can eventually lead to iron overload that can cause organ failure and shorten a patient’s life span.

Reblozyl, co-developed with Cambridge, Massachusetts-based biotech Acceleron, became the first FDA-approved treatment for anemia in beta thalassemia. However, the drug came with label warnings for blood clots, hypertension and embryo-fetal toxicity, the company said.

Serious side effects, including cerebrovascular accidents and deep vein thrombosis, were reported in 1% of trial patients, and one patient reportedly died during clinical testing due to an unconfirmed case of acute myeloid leukemia, Celgene said.

Reblozyl’s win closely followed an FDA approval for Celgene’s JAK inhibitor Inrebic (fedratinib) in August as a first-line or follow-up treatment for myelofibrosis. The once-daily oral drug became the first new treatment in nearly a decade approved to treat the disease, Celgene said.

Inrebic hit the market with a black box warning for serious and fatal encephalopathy, including Wernicke’s. The drugmaker said serious encephalopathy was reported in 1.3% of trial patients treated with Inrebic and one of those patients died.

Both approvals were major wins for Bristol, which is now in the clear on its Celgene merger after the Federal Trade Commission (FTC) voted 3-2 in mid-November to approve the transaction pending the offloading of Celgene psoriasis med Otezla.

Bristol touted Celgene’s “Big 5” late-stage drug candidates––including Reblozly and Inrebic––as major drivers of the merger, with peak sales estimates of more than $5 billion per year. The rest of the list includes multiple sclerosis hopeful ozanimod, which had its application picked up by the FDA in June, and a pair of CAR-T therapies: bluebird bio-partnered bb2121 for multiple myeloma and lisocabtagene maraleucel (liso-cel), formerly called JCAR017, the blood cancer treatment Celgene picked up in its $9 billion Juno Therapeutics buyout.

https://www.fiercepharma.com/pharma/blockbuster-balance-celgene-s-reblozyl-faces-fda-committee-for-mds

Medical marijuana cards often sought by existing heavy users

Young adults who seek enrollment in state medical marijuana programs are often those who already use heavily rather than those with mental or physical issues that could be addressed by the drug. That’s according to new results published in the Journal of Studies on Alcohol and Drugs.

“Making medical marijuana cards easy to obtain for vaguely defined mental or physical health conditions that are not supported by any research evidence has potential for those who use more heavily to claim need for a medical marijuana card solely to have easier access,” says lead author Eric R. Pedersen, Ph.D., of the RAND Corporation in Santa Monica, Calif.

These results have implications for states that have legalized medical marijuana or are considering such legislation. Policymakers, according to the authors, should “design medical marijuana programs in their states that allow card acquisition only for people with mental and physical health problems that have documented evidence of medicinal benefit.”

The researchers analyzed data from a long-term study of substance use prevention in California. Beginning in 2008, participants were followed from middle school through young adulthood. For the current investigation, the study contained data from 264 participants who were 18 to 20 years old in 2015 to 2017, when medical marijuana was legal in California but before outlets in that state were allowed to sell legal recreational marijuana beginning January 2018.

All participants had to have used marijuana at least once in the past month at the beginning of the study, but none had obtained a “medical marijuana card,” which would allow them to buy, possess and use marijuana. Both initially and one year later, they were asked about their marijuana use, symptoms of depression and anxiety, and physical health problems.

At the one-year follow-up, 19 percent of participants had sought out and received a medical marijuana card. Overall, men were 2.91 times as likely as women to have received a medical marijuana card at follow-up.

In addition, for every additional day of marijuana use at the beginning of the study, the odds of receiving a medical marijuana card one year later increased by 7%. After statistically controlling for confounding variables, the authors found that physical and mental health problems at baseline (i.e., the problems ostensibly one would seek a medical marijuana card for) were not significant predictors of receiving a card in that follow-up period.

“It seems that more frequent use of marijuana, and not the physical and mental health problems that one ostensibly seeks a medical marijuana card to address, is what drives acquisition of a medical marijuana card,” Pedersen says.

The authors note that their study does have limitations. Because this was a retrospective analysis of previously reported data, the researchers were unable to specifically ask participants the reasons they may have sought medical marijuana. Further, the timeframe may have been too short, and a more detailed and longer analysis may allow a better understanding of who seeks medical marijuana and why.

“Many individuals . . . struggle with legitimate medical and psychological concerns that can benefit from medical marijuana,” according to the authors. In California, the specific qualifying conditions include chronic pain, glaucoma, AIDS, and seizures, among others. However, the law is also general enough that providers are allowed to recommend the drug to patients with “any other chronic or persistent medical symptom” that may limit “major life activities.”

“It’s not clear to us what participants are telling providers,” Pedersen says, “but we suspect that under this catch all, you can get a recommendation pretty easily.”

He adds that providers should consider reviewing forms of symptom management other than, or in addition to, marijuana and look for signs that the drug has impacted patients in a negative way.

And as more states consider legalizing medical marijuana, policymakers should be aware of both the favorable and adverse outcomes.

“In another paper, we found that young adults who had a medical marijuana card were more likely to report heavy use, greater consequences from use, selling marijuana, and driving under the influence of marijuana compared to young adults who did not have a card,” Pedersen says.

###

Pedersen, E. R., Tucker, J. S., Seelam, R., Rodriguez A., & D’Amico, E. J. (2019). Factors associated with acquiring a medical marijuana card: A longitudinal examination of young adults in California. Journal of Studies on Alcohol and Drugs, 80, 687-692. doi:10.15288/jsad.2019.80.687

https://www.eurekalert.org/pub_releases/2019-12/joso-mmc112619.php

House panel to hold Medicare for All hearing next week

A powerful House committee announced on Tuesday that it will hold a hearing next week on Medicare for All, as well as several other proposals to expand health coverage, in a boost for backers of the progressive policy priority.

The House Energy and Commerce health subcommittee will hold a hearing next Tuesday on the Medicare for All bill introduced by Reps. Pramila Jayapal (D-Wash.) and Debbie Dingell (D-Mich.), along with six other bills, including scaled-back, “public option” proposals.

House committees have already held three hearings on Medicare for All so far this year, but the Energy and Commerce announcement is significant given that panel’s key role on health care.

The panel’s chairman, Rep. Frank Pallone Jr. (D-N.J.), had also been resistant to the idea of holding a hearing on Medicare for All, saying instead that he wanted to focus on strengthening ObamaCare.

“I’ve always been an advocate for Medicare for all or single-payer, but I just don’t think that the votes would be there for that, so I think our priority has to be stabilizing the Affordable Care Act, preventing the sabotage that the Trump administration has initiated,” Pallone said in November 2018, shortly after Democrats took back the House.

Jayapal, in particular, has been pushing for hearings on her bill for months.

Despite getting hearings, Medicare for All is not expected to get a vote in the House this year or next as Democrats increasingly focus on the 2020 White House battle.

Speaker Nancy Pelosi (D-Calif.), while giving her support to airing ideas at a hearing, has been raising increasingly explicit warnings against the policy dominating her party’s presidential race, warning of its cost and that people do not want to lose their private insurance.

“I’m not a big fan of Medicare for All,” she said last month.

Medicare for All will also have to share the stage at the hearing with somewhat less ambitious proposals, including plans for optional government-run insurance backed by Reps. Antonio Delgado (D-N.Y.) and Rosa DeLauro (D-Conn.).

“Universal health care coverage has long been the North Star of the Democratic Party and it’s why the Health Subcommittee will hold a hearing to examine seven legislative proposals that advance universal coverage for the American people,” Pallone and Health Subcommittee Chairwoman Anna Eshoo (D-Calif.) said in a statement on Tuesday.

https://thehill.com/policy/healthcare/472933-powerful-house-panel-to-hold-medicare-for-all-hearing-next-week