AstraZeneca (NYSE:AZN) and collaboration partner Merck (NYSE:MRK) announce that China’s National Medical Products Administration has approved Lynparza (olaparib) for the first-line maintenance treatment of adult patients with newly diagnosed advanced germline or somatic BRCA-mutated epithelial ovarian, fallopian tube or primary peritoneal cancer who are in complete or partial response to first-line platinum-based chemo.
Thursday, December 5, 2019
Sage Therapeutics’ SAGE-217 flunks depression study
A Phase 3 clinical trial, MOUNTAIN, evaluating Sage Therapeutics’ (NASDAQ:SAGE) GABA modulator SAGE-217 in adults with major depressive disorder (MDD) failed to achieve the primary endpoint.
Specifically, SAGE-217 did not sufficiently separate from placebo as measured by a scale called HAM-D total score at day 15. Patients receiving 30 mg once daily experienced a mean reduction of 12.6 in HAM-D total score versus 11.2 for control (p=0.115), although statistically significant reductions were observed at days 3,8 and 12 (p<0.018 at each timepoint).
A post-hoc analysis revealed that ~9% of patients in the treatment group had no measurable drug concentration implying non-compliance. Excluding these, the separation from placebo was statistically valid at day 15 (p<0.048).
The 20 mg dose failed to sufficiently separate from placebo.
On the safety front, the company says SAGE-217 was generally well-tolerated. The incidence of adverse events during the 14-day treatment period and the 28-day follow-up period was 54.2% for the 30 mg dose, 50.0% for the 20 mg dose and 48.9% for placebo. Two participants receiving 30 mg experienced serious adverse events: one suicide attempt at day 5 (patient has a long history of MDD and a past suicide attempt) and one report of a bile duct stone after day 2 that required removal.
Detailed results will be submitted for presentation at a future medical conference.
Another Phase 3 in MDD is in process with an estimated completion date of April 2021.
In January, the company announced a positive outcome in a Phase 3 study, ROBIN, in women with postpartum depression.
Management will host a conference call this morning at 8:30 am ET to discuss the results.
Shares down 53% premarket on light volume.
Wednesday, December 4, 2019
Acadia Pharma’s psychosis drug proven better than placebo in dementia patients
Acadia Pharmaceuticals Inc said on Wednesday dementia patients taking its drug Nuplazid were nearly three times less prone to a psychotic relapse than those on placebo.
The drugmaker’s late-stage trial testing Nuplazid, stopped in September after positive results, enrolled 392 patients aged 74.5 years on average.
Patients on the drug experienced a 65% reduction rate in relapses of dementia-related psychotic episodes, the company said.
Chief Executive Officer Stephen Davis said Acadia planned to meet with the U.S. regulator in the first half of next year to discuss filing for Nuplazid’s approval to include dementia-linked psychosis on its label.
There are no FDA-approved treatments for the condition, characterized by hallucinations and delusions.
The company estimates there are about 2.4 million dementia-related psychosis patients in the United States, about 70% of whom have Alzheimer’s.
About 1.2 million patients have been diagnosed and are being treated with older-generation anti-psychotics and anti-depressants, Davis noted.
Acadia said 4.8% of patients taking Nuplazid had serious side effects, compared with 3.6% of patients in the placebo group.
Nuplazid was approved to treat psychosis linked to Parkinson’s disease in 2016 and Acadia recorded net product sales of $223.8 million last year.
The drug comes at a list price of $3,100 a month.
Acadia, also testing Nuplazid for use in patients with schizophrenia and major depressive disorder, declined to comment on future pricing.
HHS announces plan to make free doses of Truvada available
The Trump administration is launching a program to provide free access to doses of Truvada, a drug that prevents against HIV, to people who are uninsured and at risk of being infected, officials announced Tuesday.
In a call with reporters Tuesday, Department of Health and Human Services (HHS) Secretary Alex Azar announced the program called Ready, Set, PrEP under which individuals who have tested negative for HIV and have a valid, on-label prescription can access the pre-exposure prophylaxis, or PrEP, medication.
Individuals can find a provider and sign up for the program at www.getyourprep.com or by calling the toll-free number 855-447-8410, he said.
The drug is being donated by the drug’s manufacturer Gilead Sciences in an 11-year deal with HHS. HHS will pay about $200 a bottle in distribution costs for the program up to a total of $6 million over six months to start, Azar said. He said HHS is working to set up a more economical distribution system in the future.
By March 30, CVS Health, Walgreens and Rite Aid will donate their pharmacy dispensing fees, which will help bring down the cost. Qualified patients will be able to obtain PrEP at those pharmacy retailers throughout the U.S. including no-cost delivery via mail, Azar said. He could not provide an estimated value of those donations.
Those companies will also provide patient counseling for the PrEP regimen and take steps to promote adherence.
In May, Gilead first announced it would make up to 2.4 million bottles of Truvada available for free to uninsured Americans at risk of contracting HIV. The announcement is not connected to patent disputes between HHS and Gilead over the drug.
This program is expected to benefit up to 200,000 patients a year and is part of the agency’s broader work aimed at ending the HIV epidemic in the U.S. within the next decade, Azar said. According to a Centers for Disease Control and Prevention report released Tuesday, about 18% of the 1.2 million people who could benefit from PrEP had a prescription.
“We have the tools to stop the spread of HIV in its tracks. Now it’s about the execution,” Azar said. “Ending the HIV epidemic requires significantly increasing the number of Americans receiving PrEP by expanding access, lowering costs and decreasing stigma.”
Ongoing HIV lab testing, which is required for individuals to qualify for Truvada, will not be covered. Azar said community health centers can usually make those services available at no charge to patients who do not have insurance coverage.
Novartis’ digital transformation continues with Amazon supply chain tie
Not long after announcing a wide-ranging partnership with Microsoft to bring artificial intelligence tools to every desk across its R&D departments, Novartis is now teaming up with Amazon’s mammoth cloud computing division to overhaul its manufacturing, supply and business operations.
The new multiyear collaboration with Amazon Web Services dovetails with the drugmaker’s directives to embrace and incorporate digital technology at every point in its product pipeline and value chain.
AWS aims to supply the company with an enterprisewide data analytics platform, including AI and machine learning services, as well as cloud-based “Insight Centers” that will provide real-time metrics to Novartis’ global technical operations staff.
These centers will focus on tracking manufacturing lines and detecting bottlenecks as well as forecasting potential issues in quality and inventory. Novartis hopes this will allow for new, optimized production models, as the company looks to scale up its work in challenging-to-make personalized medicines such as Zolgensma and Kymriah.
Most recently, company opened a new cell and gene therapy manufacturing facility last week in Stein, Switzerland, providing a European base of operations for tailored therapies. Previously, Novartis had to rely on back-and-forth flights across the Atlantic to service patients on the continent, with its sole Kymriah-producing facility being located in New Jersey.
Novartis also plans to use AWS’ internet of things and computer vision offerings to help improve its manufacturing inspections, by digitally scanning images of sites and tracking individual items to spot for potential risks or delays.
“There is a lot we can learn from the AWS team, and while manufacturing is a great starting place, we’re keen to also explore where else we can apply this technology,” Novartis’ chief digital officer, Bertrand Bodson, said in a statement.
“Using data science and digital technologies to reimagine the way we manufacture medicines is not only at the heart of our transformation, but also core to our ambition to bring innovative medicines to patients faster,” Bodson added.
Novartis currently operates more than 60 manufacturing sites worldwide, producing treatments used by nearly 1 billion people. Through its partnership with AWS, the drugmaker hopes to develop standard metrics for site efficiency, deliverable on a single dashboard illustrating global performance.
The company has already been using Amazon’s computer vision products to double-check that batch manufacturing lines are clear and cleaned after use, and prepared to begin their next scheduled set of tasks. Novartis is also employing AI to extract and analyze data from printed manufacturing documents.
The forthcoming site-based Insight Centers look to make these data available in real time to help avoid unnecessary inventory and machine downtime when producing small-batch and personalized treatments.
Pelosi pricing plan will yield 100 fewer new drugs, White House predicts
As debate over drug pricing proposals rolls on in Washington, White House advisers have concluded House Speaker Nancy Pelosi’s aggressive pricing plan would result in 100 fewer new drugs over a decade, lowering Americans’ expected lifespan along the way.
Unveiled this fall, Pelosi’s plan calls for Medicare price negotiations, an international pricing index, potential fines for drugmakers who won’t negotiate and more. President Donald Trump originally tweeted that it was “great to see” the plan, but more recently, the White House has backed away from the proposal and instead supported a bipartisan effort in the Senate.
Now, the White House’s Council of Economic Advisers has rolled out unfavorable findings on Pelosi’s proposal. The bill could lead to 100 fewer drug launches over the next decade, it says, representing about one-third of the total rollouts expected over that span. By reducing access to new drugs, the bill would lower average life expectancy in the U.S. by four months, according to CEA.
The bill would lower drug prices, “but the threat it poses to continued medical innovation will harm American patients in ways that far outweigh any benefits,” the CEA said in a blog post.
In a statement to CNBC, a spokesman for Pelosi said the White House can’t be counted on to provide “accurate numbers or honest analysis.”
“President Trump’s CEA has written a love letter to Big Pharma and letting out-of-control drug prices continue just the way they are,” the spokesman told the publication.
The CEA’s analysis of fewer new drugs resulting from Pelosi’s plan far surpasses the Congressional Budget Office’s estimate. In October, the CBO said the plan would save Medicare $345 billion between 2023 and 2029 but result in eight to 15 fewer new meds out of a group of 300. The New York Times editorial board has said that’s a worthwhile tradeoff, but the pharma industry has pushed back hard against the proposal.
Meanwhile, the Senate bill calls for changes to Medicare Part D and Part B and more transparency for pharma middlemen. Bernstein analyst Ronny Gal recently wrote that the Senate proposal would have a “negligible” effect on pharma operations, while the Pelosi plan would represent a “nuclear winter” for the industry.
All of the debate comes ahead of next year’s elections, and drug pricing promises to be a hot topic on the campaign trail. Gal wrote that in recent months, his team “became convinced that the U.S. will gradually iterate toward more control of drug costs.” What kind of proposal lawmakers and the administration will land on, though, remains an unknown.
Axsome On Track To End 2019 As The Best Performing Stock In Biotech
Axsome Therapeutics Inc AXSM 3.99% shares advanced strongly Tuesday after the company reported a positive mid-stage readout for its investigational compound to treat narcolepsy.
This small-cap biotech has been on a tear since late last year. For the year-to-date period, shares are up a staggering 1,300%.
Benzinga first alerted traders to this hugely successful biotech in late January, when the shares were already up about 200%. Even if the level that prevailed then were to be taken as an entry point, an investor would have made a neat gain of about 300% by now.
The major catalysts for the upside have been clinical readouts, which turned out to be mostly positive.
Source: Axsome
The stock gained about 161% on Jan. 7 when it reported positive Phase 3 results for its lead drug AXS-05 for major depressive disorder, or MDD. The FDA accorded Breakthrough Therapy Designation, or BTD, for AXS-05 in MDD in March. Subsequently in mid-April, the company announced positive results for the mid-stage study of AXS-05 in smoking cessation.
In September, the stock made further gains following the presentation of positive Phase 1 pharmocokinetic data for AXS-07 at the 19th Congress of the International Headache Society in Dublin. AXS-07 is being evaluated for migraine.
The stock, however, came under pressure in early October, although it made good the lost ground subsequently. An announcement in its third-quarter earnings release Nov. 7 concerning a delay in the top-line data readout for AXS-05 in treatment-resistant depression, or TRD, weighed on the stock briefly before it rebounded strongly.
The trigger for Tuesday’s rally was a Phase 2 readout for its AXS-12, which showed that Phase 2 study dubbed CONCERT, met the pre-specified primary endpoint and significantly reduced cataplexy attacks as compared to placebo in patients with narcolepsy. The asset also significantly reduced excessive daytime sleepiness and improved cognitive function, sleep quality and sleep-related symptoms.
Upcoming Catalysts
- Top-line results from the Phase 3 GEMINI placebo-controlled trial of AXS-05 in MDD – year-end 2019
- Top-line results from the Phase 3 MOMENTUM study of AXS-07 in migraine – year-end 2019
- Top-line results from the Phase 3 STRIDE-1 trial of AXS-05 in TRD – Q1 2020
- Top-line results from the Phase 2/3 ADVANCE study of AXS-05 in Alzheimer’s agitation – first half of 2020
Is Stretched Valuation A Concern?
With a slew of clinical readouts between now and the end of the first half of 2020, the stock could be primed for further upside. Regulatory milestones could also act as upside catalysts.
The average analyst rating for the stock is a Buy and the average price target is $39.86, with the Street-high estimate at $50.
Subscribe to:
Comments (Atom)