White House promises more J&J COVID-19 shots after launch stalls

 Johnson & Johnson should come close to hitting its target of delivering 20 million doses of its one-shot COVID-19 vaccine in March after its shipments stalled in recent weeks, a top White House official said on Monday, while declining to say how many will go out this week.

The company shipped four million shots in early March but its rollout has since stalled as facilities involved in the manufacturing await clearance from the U.S. Food and Drug Administration. J&J’s COVID-19 vaccine was authorized by U.S. regulators in February, making it the third to be cleared for use in the United States and the first that requires just one injection for immunization.

The healthcare conglomerate has only shipped around 4.25 million doses in total, according to federal data, far less than the 20 million it promised to deliver by the end of March. Only 2.3 million J&J doses had been administered as of Sunday morning.

“I’m not going to give you precise numbers,” White House COVID-19 adviser Andy Slavitt said on a Monday press call. “I wouldn’t signal to you that they’re going to be far away from the numbers that they have projected,” he said. J&J did not immediately respond to requests for comment. Initial U.S. doses have been made at the company’s plant in Leiden, the Netherlands. J&J has been waiting on the FDA to approve plants of outside contract manufacturers that will produce the vaccine, such as Catalent Inc.

“We’re gonna see a nice increase in Johnson and Johnson this week,” Slavitt said, without specifying. “Obviously, they’ve got a lot of increases that they need to be committed to doing.”

J&J’s vaccine, which can be stored in a standard refrigerator rather than requiring freezing, was 67% effective at stopping moderate-to-severe cases of COVID-19 after 14 days from shot administration, according to trial data. It also prevented hospitalizations and deaths. The United States has delivered more than 150 million COVID-19 shots and administered nearly 125 million doses, according to federal data last updated on Sunday. However, a top health official on Monday said she is “very concerned” that a leveling off of COVID-19 cases after a period of decline could signal a risk of renewed outbreaks as the United States works to get more people vaccinated. U.S. Centers for Disease Control and Prevention Director Rochelle Walensky said public health officials should act now to stop further spread of infections, especially as highly contagious new virus variants continue to infect more people. “I am worried that if you don’t act now, you will have another avoidable surge just as we are seeing in Europe right now,” Walensky said on the press call.

https://www.reuters.com/article/us-health-coronavirus-whitehouse/white-house-promises-more-jj-covid-19-shots-after-launch-stalls-idUSKBN2BE29A

Roche to discontinue Phase 3 dosing in Huntington's disease trial

 Roche (SIX: RO, ROG; OTCQX: RHHBY) today

announced the decision to discontinue dosing in the Phase III GENERATION 
HD1 study of tominersen in manifest Huntington's disease (HD). The 
decision was based on the results of a pre-planned review of the data 
from the Phase III study conducted by an unblinded Independent Data 
Monitoring Committee (iDMC). The iDMC made its recommendation based on 
the investigational therapy's potential benefit/risk profile for study 
participants. No new or emerging safety signals were identified for 
tominersen in the review of the data from this study. Roche intends to 
continue following participants for safety and clinical outcomes, 
without the dosing of the investigational medicine or placebo. Once full 
data from the Phase III study are available and analysed, Roche will 
share learnings and future plans with the HD community. 
 
 
 
   Dosing will be paused in the open-label extension study (GEN-EXTEND) of 
tominersen while data are carefully analysed to inform next steps on 
this study. 
 
 
 
   "This is very unfortunate news to deliver on the tominersen Phase III 
study and we know it will be especially difficult for people with 
Huntington's disease to hear. The HD community currently has no 
treatments to stop or slow the progression of this rare 
neurodegenerative disease that impacts families across generations." 
said Levi Garraway, M.D., Ph.D., Roche's Chief Medical Officer and Head 
of Global Product Development. "GENERATION HD1 is the largest clinical 
trial in Huntington's disease to date and we do know that the data 
generated will significantly advance our understanding of 
huntingtin-lowering as a potential treatment approach. We would like to 
thank all of the individuals and families participating in the study for 
their contribution, as well as the broader HD community for their 
commitment and collaboration." 
 
 
 
   The Phase I PK/PD study (GEN-PEAK) of tominersen and the observational 
Roche HD Natural History Study will continue. 

https://www.marketscreener.com/quote/stock/ROCHE-HOLDING-AG-9364975/news/Press-Release-nbsp-Roche-provides-update-on-tominersen-programme-in-manifest-Huntington-s-disease-32757161/

Tonix on Pre-IND Meeting with FDA on Major Depressive Disorder Treatment

 Tonix Pharmaceuticals Holding Corp. (Nasdaq: TNXP) (Tonix or the Company), a clinical-stage biopharmaceutical company, today announced that it has received the official minutes from a Type B pre-investigational new drug (IND) meeting with the U.S. Food and Drug Administration (FDA) on its development plan for TNX-601 CR (tianeptine oxalate and naloxone controlled-release) tablet for the treatment of major depressive disorder (MDD). Tonix’s TNX-601 CR is a novel oral formulation which is being developed as a potential treatment for MDD, posttraumatic stress disorder, and neurocognitive dysfunction associated with corticosteroid use. Tianeptine sodium (amorphous) immediate release (IR) has been available in Europe and many countries in Asia and Latin America for the treatment of depression for more than three decades, first marketed in France in 1989.

Based on the official minutes, Tonix expects to submit the IND to conduct a human abuse potential study and meet with FDA’s controlled substances staff (CSS) to reach agreement on the details of the abuse potential study. Pending the results of the human abuse potential study and the results of ongoing nonclinical toxicology studies, Tonix expects to be in a position to initiate a Phase 2 study for the treatment of MDD in the fourth quarter of 2021.

https://finance.yahoo.com/news/tonix-pharmaceuticals-announces-results-pre-183000418.html

Anixa, Moffitt Cancer Center File IND Application for Ovarian Cancer CAR-T Therapy

  Anixa Biosciences, Inc. (NASDAQ: ANIX), a biotechnology company focused on the treatment and prevention of cancer and infectious diseases, announced today that an IND (Investigational New Drug) application for its ovarian cancer CAR-T (Chimeric Antigen Receptor T-cell) therapy has been filed with the U.S. Food and Drug Administration.

This CAR-T therapy was invented by a team led by Dr. Jose Conejo-Garcia, while he was at the Wistar Institute. Dr. Conejo-Garcia is currently Chair of the Department of Immunology at Moffitt Cancer Center (MCC), where the IND enabling studies were performed, and where the clinical trial will be conducted, under the leadership of Dr. Robert Wenham, Chair of Gynecological Oncology at MCC. Anixa has a worldwide, exclusive commercial license to the technology.

The technology targets the follicle stimulating hormone receptor (FSHR) with engineered T-cells that use the follicle stimulating hormone as a homing mechanism. FSHR is found at immunological levels exclusively on the granulosa cells of the ovaries. Since the target is a hormone receptor, this technology is also known as CER-T (Chimeric Endocrine Receptor T-cell) therapy, a new type of CAR-T.

https://www.biospace.com/article/releases/anixa-biosciences-and-moffitt-cancer-center-file-ind-application-for-ovarian-cancer-car-t-therapy/

AIM ImmunoTech Adds Ampligen Arm to Study in Cancer Patients with COVID-19

 AIM ImmunoTech Inc. (NYSE American: AIM) today announced that the Institutional Review Board (IRB) of Roswell Park Comprehensive Cancer Center (Roswell Park) has approved a protocol amendment to Roswell Park’s ongoing Phase 1/2a study evaluating the two-drug combination of AIM’s Ampligen and interferon alpha-2b as a potential early-onset treatment for patients with cancer and mild-to-moderate COVID-19.

The IRB-approved amendment calls for randomization of an additional twenty patients. Ten of these patients will receive a single dose of Ampligen, but no interferon treatment, and the other ten will receive best available care only. Ampligen alone has a generally well-tolerated safety profile with lower risk of adverse events than is generally expected with interferon therapy.

"We are excited to see Ampligen tested not only as part of an antiviral treatment combination for COVID-19 among cancer patients, but also as a possible standalone therapy for this terrible virus,” said AIM CEO Thomas K. Equels. “An early-onset treatment is especially critical for cancer patients, who face significantly increased risk of severe symptoms or death."

AIM previously announced in November 2020 that the first patient had been enrolled and received treatment in Roswell Park’s study. Funding for the clinical trial is provided, in part, through grants from the National Cancer Institute and AIM, as well as institutional support from Roswell Park.

Full details about this trial under way at Roswell Park, led by co-Principal Investigators Drs. Brahm Segal and Pawel Kalinski, are available at ClinicalTrials.gov.

https://www.biospace.com/article/releases/aim-immunotech-announces-addition-of-single-agent-ampligen-arm-to-cancer-center-s-ongoing-study-in-cancer-patients-with-covid-19/

FDA Action Alert: Pacira, Zealand, bluebird and Bristol Myers Squibb

 Although the beginning of March was fairly slow, the end of the month shows a busy schedule for PDUFA dates for the U.S. Food and Drug Administration (FDA). Read on to see what’s on the calendar for this week.

Pacira’s Exparel for Use in Pediatric Patients as Postsurgical Analgesia

Pacira Biosciences has a target action date of March 22, 2021, for its supplemental New Drug Application (sNDA) for Exparel’s label to include single-dose infiltration to provide postsurgical analgesia in children aged six and over.

At the time of the FDA acceptance of the sNDA in August 2020, Donald Manning, chief medical officer at Pacira stated, “The FDA acceptance of our sNDA submission confirms and reinforces our unwavering dedication to provide opioid alternatives to as many patients as possible. Currently, postsurgical moderate and severe pain in pediatric patients is principally managed with opioids, which we know come with unwanted and potentially life-threatening side effects. Expanding the Exparel label to the pediatric population will positively impact children, parents, clinicians, hospitals, and payers—as there is a critical need for non-opioid options in this vulnerable population.”

On November 18, 2020, the European Commission granted marketing authorization for Exparel as a brachial plexus block or femoral nerve block for treatment of post-operative pain in adults, as well as a field block for treatment of somatic post-operative pain from small- to medium-sized surgical wounds in adults. The approval was based on data from four pivotal Phase III trials. The drug was originally approved in the U.S. for single-dose infiltration in adults for postsurgical local analgesia and as an interscalene brachial plexus nerve block to produce postsurgical regional analgesia.

Exparel is bupivacaine liposome injectable suspension. It utilizes the company’s DepoFoam to provide product delivery over a desired time period.

Zealand’s HypoPal for Severe Hypoglycemia

Zealand Pharma has a target action date of March 27 for its NDA for the dasiglucagon HypoPal rescue pen for treatment of severe hypoglycemia. The product is designed for diabetes patient for fast and effective treatment for severe hypoglycemia, or low blood sugar. In three Phase III clinical trials in adults and children, the primary and all key secondary endpoints were successfully achieved with a median time to blood glucose recovery of only 10 minutes after injection of 0.6mg of dasiglucagon.

At the company’s March 11 full-year and fourth-quarter 2020 report, Emmanuel Dulac, president and chief executive officer of Zealand, said, “2020 was a pivotal year for Zealand Pharma as transformed from a research and development-focused organization to a fully integrated commercial biopharmaceutical company. We continue to progress our late-stage clinical pipeline across both our metabolic and gastrointestinal franchises and to advance our early-stage pipeline. We are on schedule with our U.S. launch readiness activities in preparation for a potential FDA approval later this month and we are targeting the commercial launch of dasiglucagon auto-injector and pre-filled syringe for severe hypoglycemia in late June.”

Bluebird bio and Bristol Myers Squibb’s Ide-Cel for BCMA-CAR-T

bluebird bio and Bristol Myers Squibb have a target action date of March 27, 2021, for their BLA for idecabtagene vicleucel (ide-cel). The product is an investigational B-cell maturation antigen (BCMA)-directed chimeric antigen receptor (CAR) T-cell immunotherapy. On February 25, 2021, the companies published data from the Phase II KarMMa trial of ide-cel in the New England Journal of Medicine. It demonstrated deep and durable responses in patients with relapsed and refractory multiple myeloma (RRMM) who have had at least three previous therapies, including an immunomodulatory agent, a protease inhibitor and an anti-CD38 antibody.

In March 2020, the FDA declined to review the KarMMa data and issued a Refusal to File letter, saying it needed additional data to review the BLA. It mentioned the Chemistry, Manufacturing and Control (CMC) module as the area that needed the additional data. It was settled shortly afterward.

https://www.biospace.com/article/fda-action-alert-pacira-bluebird-and-bristol-myers-squibb-and-zealand/

Astrazeneca Exec Ruud Dobber: We'll file for U.S. EUA in first half of year

https://www.marketscreener.com/quote/stock/ASTRAZENECA-PLC-4000930/news/Astrazeneca-Exec-Ruud-Dobber-We-ll-file-for-U-S-EUA-in-first-half-of-year-CNBC-32752664/