Biogen Inc.(Nasdaq: BIIB) today announced that it has received a Complete Response Letter (CRL) from the U.S. Food and Drug Administration (FDA) for its supplemental Biologic License Application (sBLA) for a new subcutaneous (SC) route of administration of TYSABRI® (natalizumab) to treat relapsing multiple sclerosis (MS). The CRL indicates that the FDA is unable to approve the Company’s filing as submitted. Biogen is evaluating the CRL and will determine next steps in the U.S.
“We are committed to MS and pursuing innovations such as new routes of administration to help provide options that could address the individual needs of patients,” said Maha Radhakrishnan, M.D., Chief Medical Officer at Biogen. “This response from the FDA does not affect the intravenous administration of TYSABRI, a well-established high-efficacy treatment with a well-characterized safety profile, which over the last 15 years has treated more than 200,000 people worldwide with relapsing MS.”
The subcutaneous delivery of TYSABRI was approved by the European Commission in March 2021, and Biogen plans to pursue regulatory filings in additional countries.
Cruising could restart in mid-summer in American waters, the Centers for Disease Control and Prevention said late Wednesday in a letter to the cruise industry that USA TODAY obtained.
"We acknowledge that cruising will never be a zero-risk activity and that the goal of the CSO’s phased approach is to resume passenger operations in a way that mitigates the risk of COVID-19 transmission onboard cruise ships and across port communities," Aimee Treffiletti, head of the Maritime Unit for CDC’s COVID-19 response within its Global Mitigation Task Force for COVID-19, said in the letter.
In a statement about the letter, spokesperson Caitlin Shockey gave USA TODAY a more specific timeline. Cruises could begin passenger voyages from the United States in mid-July, depending on cruise lines' pace and compliance with the CDC's Framework for Conditional Sailing Order.
"CDC looks forward to continued engagement with the industry and urges cruise lines to submit Phase 2A port agreements as soon as possible to maintain the timeline of passenger voyages by mid-July," Shockey said.
A source close to the situation but not authorized to speak on the record told USA TODAY that the industry is cautiously optimistic about moving ahead after receiving the CDC's letter, after more than a year without sailing in U.S. waters due to the COVID-19 pandemic.
"Last night, the CDC notified us of some clarifications and amplifications of their Conditional Sail Order, which addressed uncertainties and concerns we had raised," Fain said. "They have dealt with many of these items in a constructive manner that takes into account recent advances in vaccines and medical science. Although this is only part of a very complex process, it encourages us that we now see a pathway to a healthy and achievable return to service, hopefully in time for an Alaskan season."
The letter came on the heels of a month of twice-weekly meetings with cruise industry representatives. During those meetings, the industry and the health agency discussed the Conditional Sailing Order.
While the CDC outlined a potential restart date for cruises departing from U.S. ports this summer, that doesn't mean that the restrictions on cruises are lifted. The CDC offered clarifications to its guidance based on industry feedback and still expects cruise lines to meet its requirements before sailing can resume.
Based on industry feedback, the CDC landed on five clarifications to its additional guidance issued April 2 to allow a resumption of sailing:
Ships can bypass the required simulated test voyages carrying volunteers and jump to sailings with paying passengers if 98% of crew and 95% of passengers are fully vaccinated.
CDC will review and respond to applications from cruise lines for simulated voyages within 5 days, a review previously expected to take 60 days.
CDC will update its testing and quarantine requirements for passengers and crew on sailings with paying passengers to align with the CDC's guidance for fully vaccinated people. So, for example, instead of taking a PCR lab test ahead of boarding vaccinated passengers can take a rapid antigen test upon embarkation.
CDC has clarified that cruise ship operators may enter into a "multi-port agreement" rather than a single port agreement as long as all port and local authorities sign the agreement.
The CDC has clarified guidance on quarantine guidelines for passengers who may be exposed to or contract COVID-19. For example, local passengers may be able to drive home and passengers who have traveled by air to cruise may quarantine in a hotel.
USA TODAY has reached out to Cruise Lines International Association, the industry's leading trade group, for comment.
Whether to restart cruising in U.S. subject to pressure from all sides
Over the last month or so, the CDC has been subject to pressure from many sides on whether to restart or hold off.
At the end of March, the cruise industry pushed for the CDC to lift its Conditional Sailing Order, calling the agency's restrictions "outdated." Other travel sector members voiced their support for a hastened return to sailing, too.
And politicians have played tug of war with the issue, too. Some legislators are pushing the CDC to allow cruises to restart while others are asking the agency to continue to hold off on allowing ships to sail due to concerns about the spread of COVID-19.
In a letter sent earlier this month, Sen. Richard Blumenthal, D-Conn., and Rep. Doris Matsui, D-Calif., urged the CDC's Walensky to maintain current restrictions on cruising.
United States Secretary of Transportation Pete Buttigieg also weighed in on the cruise industry's slow restart at a White House press briefing on April 9, stating he knows the CDC is "hopeful" for cruise lines to be in a position to sail by midsummer.
"Well, the bottom line is safety," he said. "Airplanes have one safety profile; cruise ships have another, vehicles have another. And each one needs to be treated based on what’s safe for that sector."
The chief executive of BioNTech,Ugur Sahin, has said the company is in the “final stages” before seeking approval from European regulators to administer its Covid-19 vaccine, jointly made withPfizer, to children aged 12 and over.
BioNTech and Pfizer asked regulators in the United States this month to approve emergency use of their vaccine for adolescents aged 12 to 15.
A trial published at the end of March found the companies’ Covid-19 vaccine was safe, effective and produces robust antibody responses in adolescents.
The Pfizer/BioNTech two-shot vaccine is already authorised for use in those aged 16 and above.
Meanwhile, BioNTech expects results by September from trials testing the Covid-19 vaccine in babies as young as six months old, Mr Sahin told German magazine Spiegel.
“In July, the first results could be available for the five to 12 year olds, in September for the younger children,” he said, adding that it takes about four to six weeks to evaluate the data.
“If all goes well, as soon as the data is evaluated, we will be able to submit the application for approval of the vaccine for all children in the respective age group in different countries,” he said.
Young people are less likely to suffer severe cases of Covid-19 and more likely to have asymptomatic infection, allowing them to unwittingly transmit the virus to others.
Patients in areas across the country now have access to Deep Transcranial Magnetic Stimulation (Deep TMS) therapy as an aid for short-term smoking cessation
BrainsWay Ltd. (NASDAQ & TASE: BWAY) (“BrainsWay” or the “Company”), a global leader in advanced noninvasive treatment of brain disorders, today announced the distribution of its first 10 Deep Transcranial Magnetic Stimulation (Deep TMS) devices for smoking addiction to practices across the United States. The recently FDA-cleared aid for short-term smoking cessation offers a noninvasive treatment that administers electromagnetic pulses using BrainsWay’s patented H4-coil through a cushioned helmet, stimulating neurons in brain structures associated with addiction.
Deep TMS is the first FDA-cleared noninvasive treatment in the addiction space for any TMS device, and currently serves as the only TMS platform technology with clinical outcome data for multiple brain disorders. In 2019, a double-blind, sham-controlled, multicenter randomized controlled trial of 262 patients found Deep TMS to be an effective treatment, significantly improving the continuous quit rate, as well as reducing craving and the average number of cigarettes smoked per week. Participants in the study were highly addicted to smoking with a history of smoking an average of over 26 years and multiple failed attempts to quit.
Deep TMS treatment for smoking addiction is currently available at the following clinics:
Phase 2 Study Sponsored by U.S. Military HIV Research Program, Thai Red Cross AIDS Research Centre
ImmunityBio, Inc. (NASDAQ: IBRX), a clinical-stage immunotherapy company, today announced that the U.S. Military HIV Research Program (MHRP) at the Walter Reed Army Institute of Research has launched a Phase 2 clinical trial in Thailand to evaluate ImmunityBio’s interleukin-15 (IL-15) superagonist Anktiva® (also called N-803) administered in combination with antiretroviral therapy (ART) during acute HIV infection as an experimental therapy to target and inhibit early establishment of HIV ‘reservoirs’ in infected individuals. Researchers will compare levels of HIV RNA and DNA in lymph node samples pre- and post-treatment and evaluate the therapy’s effects on CD8+ T and natural killer (NK) immune cells. The study is being conducted at the Thai Red Cross AIDS Research Centre in Bangkok.
The trial’s participants are being recruited through MHRP’s acute HIV infection cohort, which identifies individuals in the earliest post-infection stages. The cohort serves as a foundation to help researchers conduct investigations into long-term remission of HIV and to understand ways to suppress HIV without long-term antiretroviral treatment.
"Our current strategy to not just treat but cure HIV infection involves both inducing HIV out of its latent state in host T cells and removing or killing infected cells via an immune response or immunotherapy," said Patrick Soon-Shiong, M.D., Founder and Executive Chairman of ImmunityBio. "Anktiva is capable of triggering both of these mechanisms, as it can both activate viral transcription in CD4+ T cells—i.e., remove the virus from latency—and activate CD8+ memory cells and natural killer cells that recognize and kill HIV-infected host cells. We look forward to discovering whether or not this activity is correlated with reduction of viral load and inhibition of establishment of the HIV reservoir during acute infection in HIV."
In preliminary data from a separate Phase I study of Anktiva in HIV-infected subjects, the IL-15 superagonist was shown to be safe and to significantly activate proliferation of T cells and natural killer (NK) cells. There was also evidence that Anktiva activated viral transcription and reduced the viral reservoir in peripheral blood mononuclear cells. In addition, in foundational preclinical studies, ImmunityBio observed that Anktiva plus one or two anti-HIV broadly neutralizing antibodies, or bNAbs, suppressed simian/human immunodeficiency virus replication in 9 of 13 animals evaluated. Following these and other preclinical results, the AIDS Clinical Trials Group of the NIH is beginning a Phase I clinical trial (n=46) of Anktiva plus two bNAbs to explore whether the combination affects long-term viral remission.
Quarterly net revenues of $134.7 million with 80% gross margin
Interim analysis for phase 3 pivotal LUNAR trial in non-small cell lung cancer concluded with favorable recommendation to continue the trial with reduced sample size
Novocure (NASDAQ: NVCR) today reported financial results for the quarter ended March 31, 2021, highlighting continued commercial strength despite changes in patterns of care in some regions driven by COVID-19, as well as continued progress across the company’s clinical and product development programs. Novocure is a global oncology company working to extend survival in some of the most aggressive forms of cancer by developing and commercializing its innovative therapy, Tumor Treating Fields (TTFields). TTFields are electric fields that disrupt cancer cell division.
First quarter 2021 highlights include:
Three Months Ended March 31,
2021
2020
% Change
Financial, in millions
Net revenues
$
134,695
$
101,828
32
%
Gross Profit
$
108,310
$
77,332
40
%
Net income (loss)
$
(4,128
)
$
3,952
(204
)
%
Adjusted EBITDA(1)
$
21,145
$
15,064
40
%
Non-financial
Active patients at period end(2)
3,454
3,095
12
%
Prescriptions received in period(3)
1,402
1,409
—
%
Novocure will host a conference call and webcast to discuss first quarter 2021 financial results at 8 a.m. EDT today, Thursday, April 29, 2021. Analysts and investors can participate in the conference call by dialing 855-442-6895 for domestic callers and 509-960-9037 for international callers, using the conference ID 2286525.
The webcast, earnings slides presented during the webcast and the corporate presentation can be accessed live from the Investor Relations page of Novocure’s website, www.novocure.com/investor-relations, and will be available for at least 14 days following the call. Novocure has used, and intends to continue to use, its investor relations website, as a means of disclosing material non-public information and for complying with its disclosure obligations under Regulation FD.
