Tag: FDA

ABIO Jumps On Joining COVID-19 Drug Fray, GMAB Hits Trial Goal, IMRA On Watch

Today’s Daily Dose brings you news about ARCA biopharma joining the battle against COVID-19; Genmab’s positive results from amyloidosis trial; MacroGenics’ regulatory update related to breast cancer drug candidate Margetuximab and Simulations Plus receiving the FDA grant award.

Read on…

1. ARCA Jumps Into COVID-19 Drug Fray; Stock Explodes Over 300%

ARCA biopharma Inc. (ABIO) has decided to evaluate AB201, a potent, selective inhibitor of tissue factor, as a potential treatment for COVID-19 associated coagulopathy and the related inflammatory response.

COVID-19 associated coagulopathy or CAC is one of the most serious adverse effects seen in COVID-19 patients.

The Company anticipates filing an Investigational New Drug (IND) application for AB201 with the FDA in the third quarter and initiating late-stage clinical testing in the second half of this year.

As of March 31, 2020, the Company had cash and cash equivalents of only $6.7 million.

ABIO closed Thursday’s trading at $19.21, up 386.33%.

2. Genmab ANDROMEDA Trial Hits Key Goal

Shares of Genmab A/S (GMAB) closed at an all-time high of $30.97 on Thursday, thanks to positive topline results from its ANDROMEDA trial.

ANDROMEDA is a phase III study of subcutaneous Daratumumab in combination with Cyclophosphamide, Bortezomib, and Dexamethasone (CyBorD ) for patients with newly diagnosed light-chain (AL) amyloidosis.

Janssen Biotech Inc. obtained an exclusive worldwide license to develop, manufacture and commercialize Daratumumab from Genmab in 2012.

The ANDROMEDA study, conducted by Janssen, met the primary endpoint of the percentage of patients with hematologic complete response. Patients in the study treated with Daratumumab in combination with CyBorD had a 53.3% hematologic complete response compared to 18.1% of patients who were treated with CyBorD alone.

GMAB closed Thursday’s trading at $30.97, up 4.31%.

3. Imara Gearing Up For The Big Event

Shares of Imara Inc. (IMRA) continues to gain momentum as the Company gears up to present interim data from the ongoing phase IIa study of IMR-687 in patients with sickle cell disease at the 25th Annual European Hematology Association (EHA) Congress to be held virtually June 11-21, 2020.

Enrollment in the IMR-687 phase IIa clinical trial in sickle cell patients was completed in January of this year, with top-line data expected in the fourth quarter of 2020.

IMRA closed Thursday’s trading at $32.38, up 11.27%.

4. MacroGenics To Face FDA In December; No Panel Review

MacroGenics Inc. (MGNX) has been notified that the FDA has no plans to hold an AdComm meeting to review the Company’s Biologics License Application for Margetuximab, proposed for the treatment of patients with pre-treated metastatic HER2-positive breast cancer in combination with chemotherapy.

The regulatory agency’s final decision date set for Margetuximab review remains unchanged, which is December 18, 2020.

MGNX closed Thursday’s trading at $23.00, down 0.61%.

5. Simulations Plus Secures Grant Award

Shares of Simulations Plus Inc. (SLP), a developer of drug discovery/development software, touched an all-time high on Thursday, following the receipt of a new Grant Award from the FDA.

The Company has been awarded a new funded cooperative agreement for up to $400,000 over three years to develop physiologically based pharmacokinetics/pharmacodynamics (PBPK/PD) approaches to support interspecies translation for ocular drug delivery in GastroPlus.

GastroPlus is a mechanistically based simulation software package that simulates intravenous, oral, oral cavity, ocular, inhalation, and dermal/subcutaneous absorption, pharmacokinetics, and pharmacodynamics in humans and animals, according to the Company.

For its second quarter of fiscal year 2020, the period ended February 29, 2020, net revenues increased 22.2% to $10.3 million from $8.5 million in the year-ago period. Net income for the recent second quarter increased to $2.2 million from $2.1 million in the year-ago quarter while earnings per share remained unchanged at $0.12 per share. The second-quarter results were reported last month.

SLP closed Thursday’s trading at $48.43, up 22.61%.

6. Stocks That Moved On No News

Organogenesis Holdings Inc. (ORGO) closed Thursday’s trading at $4.61, up 33.24%.

IDEAYA Biosciences Inc. (IDYA) closed Thursday’s trading at $9.40, up 22.64%.

Flexion Therapeutics Inc. (FLXN) closed Thursday’s trading at $11.65, up 12.83%.

NantKwest Inc. (NK) closed Thursday’s trading at $6.61, down 12.80%.

Verrica Pharmaceuticals Inc. (VRCA) closed Thursday’s trading at $12.20, down 11.98%.

Source: Read Full Article

FDA Halts Bill Gates-backed Coronavirus Testing Program

The Food and Drug Administration has halted an at-home coronavirus testing program by Seattle Coronavirus Assessment Network or SCAN, which is backed by Bill Gates, for lack of proper approval.

In an update of the project on its website, SCAN said its testing of home-based, self-collected samples for COVID-19 is currently paused after the FDA notified that a separate federal emergency use authorization or EUA is required to return results for self-collected tests.

SCAN’s test is authorized by the Washington State Department of Health, but any coronavirus tests in the U.S. requires FDA’s emergency use authorization.

SCAN said, “The FDA has not raised any concerns regarding the safety and accuracy of SCAN’s test, but we have been asked to pause testing until we receive that additional authorization.”

SCAN, a disease surveillance program, is a partnership involving Seattle and King County public health department and a number of institutions such as the University of Washington Medicine and Seattle Children’s Hospital, among others. It receives support from Gates’ private office, Gates Ventures, and as well as The Bill and Melinda Gates Foundation.

The project sends free self-swab test kits including a nasal swab to participants, who would then return it to a lab involved in the program without leaving home. The program has the capacity to analyze 1,000 tests a day.

In a blog post last week, Gates noted that early results from SCAN found many cases of COVID-19 in Seattle that might otherwise have gone undetected among individuals who had experienced some symptoms but had not yet sought medical care. Meanwhile, ongoing COVID-19 medical testing has been largely focused on people with symptoms.

Gates wrote that “as SCAN gathers more test results in the weeks ahead, researchers expect the new data to provide a better sense of the number of infections and serve as one source to help answer other questions, like when physical distancing measures can be relaxed.”

The FDA last week had approved Everlywell Inc.’s at-home sample collection kit for COVID-19 diagnostic testing, the first standalone sample collection kit to receive a proper EUA. The Test kits will be available for individual purchase by the end of the month, which can then send to specified laboratories for COVID-19 diagnostic testing.

Source: Read Full Article

CVS Health To Open More Than 50 COVID-19 Test Sites Today

CVS Health Corp. is set to open more that 50 new COVID-19 drive-thru test sites today at select CVS Pharmacy drive-thru locations across five states – Arizona, Connecticut, Florida, Massachusetts and Pennsylvania.

These sites will use self-swab tests and mark the next phase of the company’s COVID-19 testing strategy.

The healthcare provider is expecting to scale this up to 1,000 new COVID-19 drive-thru test sites by end of May, processing up to 1.5 million tests per month based on the lab capacity and the availability of supplies.

The Self-swab tests will be available to individuals meeting Centers for Disease Control and Prevention (CDC) criteria, in addition to age guidelines. Patients must register online in advance at CVS.com beginning Friday, May 15 to schedule an appointment.

The individuals need to stay in their cars while approaching pharmacy drive-thru window to receive the test kit and instructions will be given by a CVS Pharmacy team member, who will also observe the self-swab process to ensure it is done properly. The sample will be sent to an independent, third-party lab for processing and the results will be available in approximately three days.

CVS said the COVID-19 testing will take place in parking lots or at drive-thru windows and will not take place inside any of their retail locations. CVS Pharmacy, HealthHUB and MinuteClinic locations will continue to serve customers and patients.

Since early April, the company has been successfully operating large-scale COVID-19 drive-thru rapid test sites in coordination with five states, which can process a total of nearly 30,000 tests per week. These sites in Connecticut, Georgia, Massachusetts, Michigan and Rhode Island are using the new Abbott ID NOW point-of-care COVID-19 tests.

The Abbott ID NOW test received emergency use authorization (EUA) from the U.S. Food and Drug Administration (FDA) for the fastest available molecular point-of-care test for the detection of COVID-19. Positive results can be delivered in as little as five minutes and negative results in as little as 13 minutes.

However, the FDA on Thursday warned the public about possible accuracy concerns with Abbott ID NOW test. Specifically, the FDA said the test may return false negative results. This test can still be used as it can correctly identify many positive cases in minutes, but negative results may need to be confirmed with a high-sensitivity authorized molecular test, the FDA added.

The FDA is investigating whether the inaccuracy could be due to the types of swabs used or the type of viral transport media, which is the material used to transport the patient’s specimen. The agency has received 15 adverse event reports.

Source: Read Full Article

FDA Approves NASA-Developed Ventilator For Emergency Use

The U.S. Food and Drug Administration approved a new high-pressure ventilator developed by NASA to treat coronavirus or COVID-19 patients. The space agency is offering the designs for licensing on a royalty-free basis during the time of the pandemic, hoping to increase the availability of life-saving medical devices.

In a statement, NASA said its VITAL (Ventilator Intervention Technology Accessible Locally) was approved for use under the FDA’s March 24 ventilator Emergency Use Authorization.

VITAL was developed by engineers at NASA’s Jet Propulsion Laboratory or JPL in Southern California. VITAL, like all ventilators, requires patients to be sedated and have an oxygen tube inserted into their airway to breathe.

The Office of Technology Transfer and Corporate Partnerships at Caltech, which manages JPL for NASA, is offering a free license for VITAL. It is also seeking manufacturers for the device from commercial medical industry.

NASA said the ventilator can be built faster and maintained more easily than a traditional ventilator. It is composed of fewer parts, many of which are currently available to potential manufacturers through existing supply chains.

With its flexible design, the ventilator can be modified for use in field hospitals being set up in convention centers, hotels and other high-capacity facilities across the world.

The new device will last three or four months, and wouldn’t replace current hospital ventilators that can last years.

Amid the worsening Covid-19 spread, various U.S. car makers recently started manufacturing ventilators using their infrastructure. In mid-April, General Motors said it was preparing to deliver its first batch of ventilators to the U.S. for the treatment of patients with Covid-19. Ford is working with GE Healthcare to produce 50,000 ventilators in Michigan. Tesla also is reported to be producing ventilators at its Gigafactory in Buffalo, New York.

Source: Read Full Article

FDA Recalls Sanofi’s Zantac Heartburn Drug, All Generics

The U.S. Food and Drug Administration has urged to withdraw Sanofi’s heartburn drug Ranitidine as well as its generics from the market with immediate effect for possible contamination. The recall involves all prescription and over-the-counter or OTC ranitidine drugs, commonly known by the brand name Zantac.

The recall includes generic products that contain ranitidine made by Novartis, Endo, Dr. Reddy’s Laboratories, Perrigo, and others.

The FDA said the ranitidine products, which are used to treat stomach acid and ulcers, will not be available for new or existing prescriptions or OTC use in the U.S. FDA is sending letters to all manufacturers of ranitidine asking to withdraw their products.

The decision was taken following an ongoing investigation of a contaminant known as N-Nitrosodimethylamine or NDMA, a probable human carcinogen, in ranitidine medications. NDMA is generally found in water and foods, including meats, dairy products, and vegetables.

In a statement, the heath regulator said it has found that the impurity in some ranitidine products increases over time, when stored at higher than room temperatures. This is likely to result in consumer exposure to unacceptable levels of this impurity.

Janet Woodcock, director of the FDA’s Center for Drug Evaluation and Research, said, “We didn’t observe unacceptable levels of NDMA in many of the samples that we tested. However, since we don’t know how or for how long the product might have been stored, we decided that it should not be available to consumers and patients unless its quality can be assured.”

Independent laboratory testing previously had found the presence of NDMA in ranitidine, but FDA at that did not have enough scientific evidence to recommend to discard its use. The agency in September 2019 had warned the public of the potential risks and to consider alternative OTC and prescription treatments.

Following this, several drug companies in the U.S. had issued recalls for Ranitidine. Sanofi had recalled all Zantac OTC in the U.S., while Dr. Reddy’s, Perrigo, and Sandoz Inc., the generics division of Swiss drug maker Novartis AG, recalled all Ranitidine Tablets.

Retail giant Walmart as well as Walgreens and CVS Pharmacy suspended the sale of OTC Ranitidine medications at their counters.

Woodcock further suggested multiple alternative medications to treat heartburn. FDA said its testing has not found NDMA in famotidine (Pepcid), cimetidine (Tagamet), esomeprazole (Nexium), lansoprazole (Prevacid) or omeprazole (Prilosec).

Source: Read Full Article

AMRN’s Vascepa Woes, AKRO Awaits Big Day, JNJ’s COVID-19 Vaccine Study In Sep

Today’s Daily Dose brings you news about Akero’s big day; Immunovant’s Thyroid Eye Disease trial results; Court ruling in Amarin’s patent suit related to Vascepa, Savara’s update on its ongoing clinical trials in light of the coronavirus crisis; Johnson & Johnson’s progress in its COVID-19 vaccine program.

Read on…

Akero Therapeutics Inc. (AKRO) is all set to announce results from the ongoing phase IIa study of its investigational drug AKR-001 in patients with biopsy-confirmed nonalcoholic steatohepatitis, or NASH, on Tuesday, March 31, 2020, before the market opens.

The phase IIa study, dubbed BALANCED, is a multicenter, randomized, double-blind, placebo-controlled, dose-ranging trial evaluating AKR-001 in biopsy-confirmed patients with NASH.

The trial involves 80 patients who are randomized to receive weekly subcutaneous doses of AKR-001 or placebo for up to 16 weeks, with safety and tolerability followed through week 20.

The primary efficacy endpoint for the study is an absolute change from baseline in hepatic fat fraction measured by magnetic resonance imaging – proton density fat fraction (MRI-PDFF) at week 12. Secondary measures include the change from baseline in ALT (alanine transaminase, a liver enzyme) at 12 weeks, the number of patients who had a decrease of more than 2 points in the NAFLD activity score at 24 weeks and safety and tolerability measures.

AKRO closed Monday’s trading at $17.18, up 6.58%.

Shares of Amarin Corporation plc (AMRN) slumped more than 60 percent in extended trading on Monday, after the Company lost the patent suit, which it had filed against generic competitors Dr. Reddy’s and Hikma Pharmaceuticals.

Dr. Reddy’s and Hikma Pharmaceuticals had filed abbreviated new drug applications, or ANDAs, for Amarin’s VASCEPA capsule franchise.

Vascepa is indicated as an adjunct to diet to reduce triglyceride levels in adult patients with severe hypertriglyceridemia, and as an adjunct to statin therapy to reduce the risk of cardiovascular events.

The drug brought in annual sales of $427 million for Amarin in 2019 compared to $228 million in 2018. According to the Company, Vascepa is said to have patent coverage until 2030.

Commenting on the court ruling, John F. Thero, president and chief executive officer of Amarin said, “Amarin strongly disagrees with the ruling and will vigorously pursue all available remedies, including an appeal of the Court’s decision and a preliminary injunction pending appeal to, if an ANDA is approved by FDA, prevent launch of generic versions of VASCEPA in the United States.”

AMRN closed Monday’s trading at $13.58, up 2.57%. In after-hours, the stock fell 66.86% to $4.50.

Axsome Therapeutics Inc.’s (AXSM) phase III trial of AXS-05 in treatment-resistant depression, dubbed STRIDE 1, has failed to reach statistical significance in the primary endpoint, i.e. the MADRS change from baseline to End of Study (6 weeks).

MADRS or Montgomery-Åsberg Depression Rating Scale is used to assess depressive symptomatology during the previous week. Subjects are rated on 10 items to assess feelings of sadness, lassitude, pessimism, inner tension, suicidality, reduced sleep or appetite, difficulty concentrating and lack of interest. Each item is scored on a 7-point scale. A score of 0 indicates the absence of symptoms, and a score of 6 indicates symptoms of maximum severity. (Source: ClinicalTrials.gov).

However, the trial met key secondary endpoints demonstrating rapid and statistically significant improvements in depressive symptoms on MADRS versus active comparator at Weeks 1, 2, and overall.

The Company also announced that it is on track to file an NDA in the fourth quarter for AXS-05 in the treatment of major depressive disorder.

Data readout from INTERCEPT Phase 3 trial of AXS-07 in the early treatment of migraine is expected very soon while that from ADVANCE-1 Phase 2/3 trial of AXS-05 in Alzheimer’s disease agitation is expected in the early second quarter.

AXSM closed Monday’s trading at $57.58, down 8.99%.

Biohaven Pharmaceutical Holding Company Ltd.’s (BHVN) pivotal clinical trial evaluating oral Rimegepant 75 mg for the preventive treatment of migraine in both episodic and chronic migraine patients has yielded positive topline results.

The study met the primary endpoint, demonstrating a statistically significant reduction from baseline in monthly migraine days in patients treated with oral Rimegepant compared with placebo.

In February of this year, oral Rimegepant 75 mg was approved by the FDA for the acute treatment of migraine in adults. The drug, under brand name NURTEC ODT, was launched on March 12, 2020.

BHVN closed Monday’s trading at $33.31, down 5.58%.

Shares of Immunovant Inc. (IMVT) jumped more than 20 percent on Monday, following encouraging initial results from its ongoing phase IIa study of IMVT-1401 in patients with Thyroid Eye Disease, also known as Graves’ ophthalmopathy.

The trial, dubbed ASCEND GO-1, involved seven adult patients with moderate-to-severe active Thyroid Eye Disease who were treated with two weekly 680mg subcutaneous doses of IMVT-1401 followed by four weekly 340mg subcutaneous doses of IMVT-1401.

According to the initial results, the mean reduction in total IgG levels from baseline to end of treatment was 65%. As evaluated at the end of treatment, four out of the seven patients improved by 2 or more than 2 points on the Clinical Activity Score. Of six patients with baseline diplopia (double vision), 4 patients (67%) demonstrated improvement in diplopia, noted the Company.

IMVT-1401 was safe and generally well-tolerated with no serious adverse events.

Horizon Therapeutics’ (HZNP) Tepezza, approved in January of this year, is the first and only drug for the treatment of adults with thyroid eye disease.

IMVT closed Monday’s trading at $16.55, up 23.14%.

Johnson & Johnson (JNJ) has selected a lead COVID-19 vaccine candidate from constructs it has been working on since January 2020.

The Company expects to initiate phase I human clinical studies of its lead vaccine candidate at the latest by September 2020. The first batches of a COVID-19 vaccine could be available for emergency use authorization in early 2021, a substantially accelerated timeframe in comparison to the typical vaccine development process, JNJ noted.

Through a landmark new partnership, BARDA and Johnson & Johnson together have committed more than $1 billion of investment to co-fund vaccine research, development, and clinical testing.

JNJ closed Monday’s trading at $133.01, up 8.00%.

Reata Pharmaceuticals’ (RETA) phase III study of bardoxolone methyl in patients with connective tissue disease-associated pulmonary arterial hypertension, dubbed CATALYST, and the open-label extension study of bardoxolone in patients with PAH, dubbed RANGER, are being closed.

The decision to stop the CATALYST trial is based on the initial review of available efficacy data provided by the Data and Safety Monitoring Board (DSMB), which suggested that the study is unlikely to meet the primary endpoint of improvement in six-minute walk distance compared to placebo at Week 24.

The Company has temporarily paused enrollment of new patients in the Phase 3 FALCON trial of bardoxolone in patients with autosomal dominant polycystic kidney disease.

However, the phase III CARDINAL trial of bardoxolone in patients with chronic kidney disease caused by Alport syndrome is fully enrolled and ongoing.

RETA closed Monday’s trading at $161.88, up 7.93%.

Savara Inc. (SVRA) has terminated enrollment in its AVAIL and ENCORE studies due to the ongoing COVID-19 pandemic.

AVAIL is a phase III study evaluating AeroVanc in people living with cystic fibrosis who have methicillin-resistant Staphylococcus aureus (MRSA) lung infection. Total target enrollment was 200 patients. A total of 188 patients have been enrolled. Topline results from the study are still expected in early 2021.

ENCORE is a phase IIa study evaluating Molgradex in nontuberculous mycobacterial lung infection in people living with cystic fibrosis. Total target enrollment was approximately 30 patients and 14 patients are currently enrolled.

SVRA closed Monday’s trading at $2.35, up 9.30%. In after-hours, the stock was down 7.66% at $2.17.

Source: Read Full Article