LONDON – “I really believe we can start using the word cure,” said the pioneer of chimeric antigen receptor T-cell (CAR T) therapy Carl June, as he revealed two leukemia patients he treated in a phase I trial have now been in remission for 10 years. Both patients with chronic lymphocytic leukemia achieved complete remission shortly after treatment in 2010. The genetic modification has remained detectable in CAR T-cell descendants for more than 10 years of follow-up, June said, describing details of the case studies published in Nature Feb. 2.
UK challenge trial lays foundation for accelerated COVID-19 studies
LONDON – The first human challenge study of SARS-CoV-2 infection has reported initial results, showing it is safe to infect healthy volunteers with the virus in controlled conditions, and paving way for this model to be used to accelerate clinical trials of COVID-19 vaccines, antivirals and diagnostics.
Gilead paying $1.25B, plus royalties, to Viiv to resolve HIV drug patent claims
Gilead Sciences Inc. is making a one-time $1.25 billion payment, with a commitment for a royalty that analysts predict could add as much as another $1.5 billion, to Viiv Healthcare Ltd., in a deal designed to resolve all global pending or potential patient infringement claims relating to sales of HIV drug Biktarvy (bictegravir/emtricitabine/tenofovir alafenamide). The initial payment, recorded in the fourth quarter of 2021, put a significant dent in Gilead’s earnings per share (EPS) but removes the uncertainty of a trial outcome and clears the way for future bictegravir-containing products.
DOJ claws back $5.6B in False Claims Act litigation in 2021, but standard for dismissal in flux
The U.S. Department of Justice reported Feb. 1 that it had recouped more than $5.6 billion in settlements in connection with False Claims Act litigation last year, with $5 billion of that amount derived from action against the health care industry. Some of these cases begin as whistleblower lawsuits, which the department can dismiss, but the grounds for dismissing these whistleblower suits is in flux and may be headed for a hearing at the U.S. Supreme Court for clarification.
Logicbio study placed on clinical hold due to two SAEs in children
The FDA has placed Logicbio Therapeutics Inc.’s phase I/II clinical trial of LB-001, an AAV genome-editing therapy for treating pediatric patients with methylmalonic acidemia, on a clinical hold. So far, four patients have been dosed in the study and two have had drug-related serious adverse events (SAEs). The Lexington, Mass.-based company’s stock (NASDAQ:LOGC) had staggered 55% downward at midday and is down about 67% since 2022 began.
US FDA begins review of COVID-19 vaccine for youngest Americans
Amid pressure to get a COVID-19 vaccine authorized for infants and toddlers sooner than later, Pfizer Inc. and Biontech SE initiated a rolling submission seeking to amend the emergency use authorization for their mRNA vaccine to include children 6 months through 4 years of age. The submission was made at the request of the U.S. FDA in light of the recent omicron surge, Acting FDA Commissioner Janet Woodcock said. The agency is committed to a “timely review,” Woodcock added. The FDA’s Vaccines and Related Biological Products Advisory Committee will meet Feb. 15 to discuss the data.
Pandemic lessons fuel reignition of US cancer moonshot
U.S. President Joe Biden is reigniting the cancer moonshot he launched as vice president in 2016 to recover momentum lost to COVID-19 and reflect the new tools that have been developed to detect and treat cancer. In a course correction, he is set to issue a call to action today to get back on track after more than 9.5 million cancer screenings were missed in the U.S. due to the pandemic, according to a White House fact sheet issued ahead of the president’s briefing. His plan also calls for government regulators, researchers and industry to apply some of the lessons learned from COVID-19 to the oncology space.
Crossing boundaries: HDT takes the COVID-19 battle to the world
HDT Bio Corp. looked at the world and saw health care inequity, so it built itself to help countries with developing economies help themselves. The company is bringing RNA technology for handling COVID-19 to underserved areas such as Brazil so they can develop, manufacture and distribute their own vaccine instead of relying on big pharma or developed-nation governments.
Therapy for ultra-rare XLHED edges closer as pivotal trial begins
The ultra-rare skin disease X-linked hypohidrotic ectodermal dysplasia (XLHED) can be a heavy burden for children, affecting structures in their skin including sweat glands, respiratory glands, and hair as well as teeth. Though lacking a specific treatment to date, a pivotal trial planned by the Geneva-based nonprofit Esperare Foundation and Pierre Fabre SA will help one potential treatment, ER-004, move one step closer to filing that need in the coming years.
Also in the news
Aardvark, Alvotech, Anakuria, Atea, Aveta, Biontech, Biosanapharma, Brickell, Canntab, Crispr, Evoke, Eversana, Jazz, Juvena, Logicbio, Mosaic, Navitor, Northsea, Oncolytics, PDS, Pfizer, Pharming, Pimera, Qsam, Samsung Bioepis, Tarsus, Theseus, Titan, Tracon, Viacyte