AbbVie and Regenxbio Inc entered a partnership to develop and commercialize RGX-314, a gene therapy candidate for the treatment of chronic retinal diseases, which would give the latter an upfront payment of $370 million.
Regenxbio may potentially receive up to $1.38 billion in milestone payments, the pharmaceutical companies said on Monday.
RGX-314 is currently in a pivotal trial for the treatment of wet age-related macular degeneration (AMD), an eye disorder causing blurred vision or a blind spot in the visual field. The trial is testing RGX-314 delivered under the retina.
It is also being studied in patients with wet AMD and diabetic retinopathy in two separate mid-stage clinical trials using a different type of delivery.
Regenxbio will be responsible for the completion of the ongoing studies of RGX-314. Both companies will share the costs of additional trials of RGX-314, they said.
AbbVie will lead the clinical development and commercialization of RGX-314 globally.
Trial metprimary endpoint, ATYR1923 was safe and well-tolerated.
Efficacy observed in key endpoints including steroid reduction of 58% in the 5.0 mg/kg treatment group with 33% of patients in the group able to taper completely off of steroids.
Clinically meaningful improvements in forced vital capacity (FVC) of 3.3% and all sarcoidosis symptom measures, including shortness of breath, cough, and fatigue, observed in the 5.0 mg/kg treatment group.
Management to host conference call and webcast today, September 13th at 8:30am ET/5:30am PT
Conference Call and Webcast
aTyr Pharma will host a conference call and webcast to discuss the results today, September 13th at 8:30am ET/5:30am PT. Interested parties may access the call by dialing toll-free 844-358-9116 from the US, or 209-905-5951 internationally and using conference ID 1957829. Links to a live webcast and replay may be accessed on the aTyr website events page at: http://investors.atyrpharma.com/events-and-webcasts. A replay will be available for at least 90 days following the event.
ZOLL Medical Corporation, an Asahi Kasei company, has agreed to acquireItamar Medical Ltd (NASDAQ: ITMR) for a total value of approximately $538 million.
Itamar Medical focuses on the development and commercialization of non-invasive medical devices and solutions to diagnose respiratory sleep disorders.
ZOLL Medical manufactures medical devices and related software solutions.
Under the agreement terms, ZOLL Medical will acquire all outstanding ordinary shares of Itamar Medical for $31 per ADS, or $1.03 (equivalent to approximately NIS 3.31) per ordinary share, in cash.
The offer of $31 per ADS represents a premium of 50.2% over the price of Itamar Medical's ADS on the Nasdaq Stock Market on September 10.
The transaction is expected to close by the end of 2021.
Once the acquisition is completed, Itamar Medical's principal operations will continue at its current location in Caesarea, Israel, including its R&D and Digital Health Technology centers, as well as the production center.
Dynavax Technologies Corporation(Nasdaq: DVAX), a biopharmaceutical company focused on developing and commercializing vaccines, today announced thatValneva SEhas received a termination notice from the United Kingdom Government in relation to Valneva's supply agreement for its COVID-19 vaccine candidate, VLA2001. Valneva stated that they intend to continue clinical development of VLA2001 and the pivotal Phase 3 trial for VLA2001, Cov-Compare, remains ongoing at Public Health England. Based on its portfolio of COVID-19 collaborations Dynavax reiterates its belief that its CpG 1018 supply contracts continue to represent an approximately $300 - $400 million dollars aggregate revenue opportunity in 2021.
Valneva recently announced that its Phase 3 results for VLA2001 are expected to be available early in the fourth quarter of 2021 and these results are expected to form part of Valneva's planned rolling submission for conditional approval of VLA2001 with the UK's Medicines and Healthcare products Regulatory Agency ("MHRA"). Subject to these data and MHRA approval, Valneva has indicated that it believes initial approval for VLA2001 could be granted in late 2021.
Ryan Spencer, Chief Executive Officer of Dynavax commented, "Valneva is one of a number of companies developing COVID vaccines using CpG 1018 as an adjuvant and we continue to look forward to the upcoming Phase 3 clinical trial results for Valneva's inactivated COVID-19 vaccine adjuvanted with CpG 1018. The first COVID-19 vaccine that uses CpG 1018 was recently authorized by regulatory authorities and we look forward to the potential authorization of additional Dynavax-enabled COVID-19 vaccines in the months and quarters ahead."
Under Dynavax's existing supply agreement for CpG 1018, purchase orders submitted by Valneva are cancellable if the UK Government reduces or terminates its order for VLA2001, in which case, Valneva would not be obligated to pay Dynavax the final portion of an outstanding purchase order. Valneva has not yet cancelled any outstanding purchase orders for CpG 1018. Dynavax has the right to retain any portion of the purchase price for CpG 1018 made in advance by Valneva as well as any CpG 1018 manufactured but not yet delivered.
Public outcry about the shockingly high cost of brand-name drugs and a demand for new laws to rein in the cost of these medicines has persisted for years. But more than 90% of all prescriptions —almost 4 billion a year— are filled with generic drugs.
Little attention has been paid to how much insurance companies, pharmacy benefit managers, and pharmacies charge to fill these generic prescriptions. Yet the difference between the highest and lowest price being charged for the same generic drug is so large that many billions of dollars could be saved each year by having prescriptions filled at the lowest-cost pharmacies.
The entry of companies like Amazon and GoodRx into the business of filling prescriptions is a game changer. They are offering prices far lower than the federal government pays insurance plans to have these prescriptions filled under the Medicare Part D drug benefit. Repealing the current Part D benefit design and replacing it with a system in which the government directly reimburses low-cost pharmacies for filling generic prescriptions would save about $18 billion a year. Patients would save an additional $8 billion in out-of-pocket copayments because the prices these pharmacies charge do not require any form of insurance or any additional co-payment.
I compiled information from the Medicare Part D website on how much it spent per pill for the 20 top-selling generic drugs during 2019. I then determined how much Amazon and GoodRx charged for a 90-day prescription for the same 20 drugs and calculated the cost per pill for those prescriptions. For example, Medicare paid 26 cents per tablet for atorvastatin, the generic version of the cholesterol-lowering drug Lipitor. Amazon fills a 90-day prescription for a 20-milligram atorvastatin tablet, the most common dose, for $4.20. That amounts to less than 5 cents per pill. The government paid insurance plans $919 million dollars to dispense 3.6 billion atorvastatin tablets in 2019. Amazon would have dispensed those tablets for $182 million, a savings of $737 million on just one generic drug.
As shown in the table here, Medicare spent $8.8 billion to dispense these 20 generic drugs in 2019. Amazon would have done that for only $4.6 billion, and GoodRx for $5.1 billion. If the prescriptions had been filled by selecting the lowest-cost drug from either Amazon or GoodRx, the cost would have been $3.5 billion. That represents a savings of $5.3 billion (60%) over what Medicare spent.
In 2019, Medicare Part D filled about 1.2 billion generic prescriptions at a total cost of about $30 billion. If the 60% savings applied to all generic drugs dispensed under Part D, the government would have saved $18 billion.
In addition to overcharging the government, Part D insurance plans also charge patients copayments for most generic prescriptions. Iqvia reports that 65% ($8 billion) of the $12 billion in Part D out-of-pocket costs paid by patients are for generic prescriptions. GoodRx estimates that its full price for filling generic prescriptions is less than the Medicare Part D copayment about one-third of the time. The Amazon and GoodRx price is the full price of the drug. There is no copayment. That means patients would save $8 billion in out-of-pocket costs, bringing the total savings from a redesign of the Medicare drug benefit to $26 billion.
Competition between generic manufacturers assures that these pills are offered for sale at a small profit over the actual cost to produce them. Yet, in a few instances where such competition is lacking, as was the case for the generic version of the EpiPen, the cost of a generic drug may sell at only a small discount from the brand name product.
Insurance plans and pharmacy benefit managers price their generic prescriptions at levels the market will bear rather than at a reasonable markup from the actual cost of acquiring and dispensing the prescription. Patients and payers believe they are getting a bargain when they get a 70% discount and pay 30 cents per pill for the generic version of a branded medicine for which they previously paid $1. They have no idea that the pharmacy acquired that pill for only a penny or two and is marking up each pill by 10 to 20 times its acquisition cost. The high markup imposed by insurance plans and pharmacy benefit managers explains why the government pays 26 cents a pill for atorvastatin while Amazon sells it for 5 cents.
Discount pharmacies like Amazon are disrupting the prescription drug market by pricing their prescriptions on a cost-plus basis while still earning a fair profit. A 90-day prescription for a generic medicine acquired at a cost of 1 cent per pill has an ingredient cost of 90 cents. The average dispensing cost — the pharmacy’s cost for the time spent to count and package the pills, label the pills, comply with laws for keeping prescription records, and record the information required for reimbursement from insurance plans — is about $2.40 per prescription. If a discount pharmacy charges $5.00 for this 90-day prescription, it will earn a $1.70 profit over the actual $3.30 cent cost of the prescription. That amounts to a gross profit of 34%, which compares favorably with the reported profit margins of independent pharmacies.
For a 90-day prescription, adding ten cents to the cost of each pill would add $9 to the cost of the prescription. That doesn’t sound like much but doing that for 4 billion prescriptions translates to $36 billion.
Medicare should be able to fill generic prescriptions at the lowest price charged for those prescriptions in the competitive retail market. But the 2003 law that created Part D prohibits the government from shopping for the best price and gives private insurance plans exclusive authority to set the price. The Republican administration that championed the enactment of Part D erroneously believed that competition between insurance plans would result in the lowest prices and widest choices for beneficiaries. No such competition ever materialized. Instead, Part D created windfall profits for private insurance plans and pharmacy benefit managers while needlessly adding complexity to the process of filling generic prescriptions. Patients are now faced with an incomprehensible array of formularies, quantity limitations, and copayments that often make it cheaper to pay for a generic drug out-of-pocket than to use Part D insurance.
The current design of the Part D benefit is ill-suited to a world in which discount pharmacies fill generic drug prescriptions at affordable prices without insurance. A new design that eliminates expensive intermediaries and relies on price competition between pharmacies will not only dramatically lower costs in Part D but will also force all pharmacies to lower their profit margins if they hope to compete for the Part D prescriptions that account for 30% of the retail pharmacy business.
Part D prices for generic drugs would quickly become the benchmark for private payers and drive down the prices and copayments for generic drug prescriptions in private insurance plans. The end result would be that more than 90% of all prescriptions could be filled at a lower cost to payers without burdening patients with significant additional out-of-pocket costs.
Senate Democrats are now negotiating a $3.5 trillion-dollar soft infrastructure package that will include legislation to lower patient out-of-pocket costs in Part D and expand Medicare to cover vision, dental, and hearing services by imposing new taxes. My proposal to lower Medicare’s cost of dispensing generic drugs and the Biden administration’s proposal to limit price increases on brand-name drugs could produce enough savings in the cost of current Medicare prescription drug benefits to pay for the proposed expansion of Medicare benefits without such taxes.
Given the rising resistance of moderate Democrats to the $3.5 trillion price tag of the proposed infrastructure package, paying for new benefits by reducing the cost of existing ones may be the wisest path forward.
Alfred Engelberg is a retired intellectual property lawyer and philanthropist who focuses on efforts to make health care and medicines more affordable. As counsel to the generic drug industry, he played a major role in drafting the Hatch-Waxman Act of 1984, which created the modern generic drug industry.
US Surgeon General Dr. Vivek Murthy on Sunday defended the Biden administration not requiring proof of COVID-19 vaccination to travel on airplanes, saying that unvaccinated people need to be able to fly in emergency situations.
Murthy said it was “reasonable” to consider implementing vaccine rules for travel but also necessary to take into account “equity concerns.”
“We know that when it comes to mandating vaccines for travel there are important issues around equity that would have to be worked out, to ensure that people, for example, if they have to travel in the case of emergency to see a relative who got sick, would be able to do that, even if you know they weren’t vaccinated,” Murthy told CNN anchor Dana Bash on “State of the Union.”
Murthy, however, insisted that President Biden’s new vaccine rules, which will require businesses with 100 or more workersto mandate vaccinations or weekly testing, are necessary steps in fighting the pandemic.
“I think [it’s] an appropriate response for us to recognize that if we want our economy to be back, if we want our schools to stay in session, we’ve got to take steps to make sure workplaces and learning environments are safe and these requirements will help do that,” Murthy told ABC “This Week” anchor George Stephanopoulos.
Under Biden’s mandate, all federal employees will also need to be vaccinated, with few exceptions.
“The COVID virus is a dangerous virus,” Murthy said. “It makes our workplaces and our schools, far less safe than they should be. So this is an appropriate action, we believe, and it’s certainly from a public health perspective — most importantly — will help keep workers safe.”
Fauci, the director of the National Institute of Allergy and Infectious Diseases, said if more people aren't persuaded to get vaccinated by messaging from health officials and "trusted political messengers," additional mandates from schools and businesses may be necessary.
"I believe that's going to turn this around because I don't think people are going to want to not go to work or not go to college ... They're going to do it," Fauci told CNN's Jen Christensen during an interview at the NLGJA, the Association of LGBTQ Journalists, convention Sunday. "You'd like to have them do it on a totally voluntary basis, but if that doesn't work, you've got to go to the alternatives."
The combination of the highly contagious Delta variant and the vaccine holdouts has put the United States in a "very difficult period" of the Covid-19 pandemic, Fauci said.
Of the eligible population in the US, which is currently limited to people 12 and older, 63% are fully vaccinated, according to data from the US Centers for Disease Control and Prevention. Health experts and officials are aiming for the vast majority of the population to be inoculated to control the spread.
Last week, President Joe Biden announced new vaccine requirements, which include a mandate for businesses with more than 100 employees to require vaccination or regular testing for employees. The plan was met with praise and criticism.
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Businesses that want employees to return to work and stay at work will benefit from vaccine requirements, US Surgeon General Dr. Vivek Murthy said. The mandate will benefit employees as well, he added.
"I believe that will not only improve public health, but it will give people some more peace of mind," Murthy told CNN Sunday.
But Arkansas Gov. Asa Hutchinson argued that the requirements may backfire.
"We have to overcome resistance," Hutchinson said Sunday on NBC's "Meet the Press." "This is a very serious, deadly virus and we're all together in trying to get an increased level of vaccination out in the population. The problem is that I'm trying to overcome resistance but the President's actions in a mandate hardens the resistance."
As the debate over mandates continues, some hospitals are feeling the impact of lagging vaccination rates.
Colorado Gov. Jared Polis sounded the alarm Friday, saying, "We actually have the lowest ICU available rate that we've had since the start of this crisis, in part due to the unvaccinated with Covid and just other types of trauma that goes up seasonally this time of year."
Polis said some hospitals in his state "reaching very close to their capacity limits. And that wouldn't be happening if people were vaccinated."
