The FDA made public documents related to its controversial accelerated approval of Biogen Inc.’s beta-amyloid-targeting therapy, Aduhelm (aducanumab), for Alzheimer’s disease. Patrizia Cavazzoni, director of the FDA’s Center for Drug Evaluation and Research, said the agency released the materials in order to provide “interested parties with the opportunity to explore the data that helped shape our decision.” In November 2020, members of the Peripheral and Central Nervous System Drugs Advisory Committee voted solidly against clearance of the drug. Three panelists resigned after the FDA greenlighted Aduhelm anyway.

Third Rock-backed Abata scores $95M series A for MS efforts

Abata Therapeutics Inc. pulled down $95 million in series A money to fuel its push in non-relapsing multiple sclerosis as well as other autoimmune and inflammatory disorders. The Boston-based firm is developing autologous regulatory T cells engineered to express T-cell receptors that recognize antigens in tissue where an autoimmune response has been triggered, thereby zeroing in on the site of disease without causing systemic immune suppression. Third Rock Ventures led the round, with participation from Elevatebio LLC, Lightspeed Venture Partners, Invus, Samsara Biocapital and the JDRF T1D Fund.

Tvardi secures $74M financing to advance STAT3 inhibitors

Tvardi Therapeutics Inc., a company developing medicines targeting STAT3, a regulatory protein essential to the survival and immune evasion of cancer cells, has closed a $74 million series B financing. The round will help Tvardi's team advance candidates through multiple clinical readouts in midstage trials for cancer and fibrosis, it said. The Houston-based company’s lead candidate, TTI-101, is in phase I tests in patients with advanced solid tumors who've failed all lines of therapy.

Under-pressure CEO Walmsley outlines GSK’s breakup plans

Glaxosmithkline plc CEO Emma Walmsley has set out further details of the company’s forthcoming split, saying that the firm’s late-stage pipeline could help deliver sales of more than £33 billion by 2031. The announcement scheduled the demerger of its consumer products business for midway through next year. Walmsley announced detailed targets and plans for the “New GSK” ahead of a crunch meeting with investors, at a time where there have been doubts about her leadership of the company based in West London, U.K.

China grants first CAR T approval to Fosun Kite

China’s drug regulator granted a first CAR T therapy approval to Fosun Kite Biotechnology Co. Ltd.’s FKC-876. Marketed as Yescarta (axicabtagene ciloleucel) in the U.S. and EU, the approval by the National Medical Production Administration (NMPA) for the CD19-directed CAR T therapy came 3.5 years after the FDA nod. With a tentative brand name Ejilunsai in China, the CAR T therapy will be indicated for adult patients with relapsed or refractory large B-cell lymphoma after two or more lines of systemic therapy, including diffuse large B-cell lymphoma not otherwise specified, primary mediastinal large B-cell lymphoma, high-grade B-cell lymphoma and DLBCL arising from follicular lymphoma.

Infectious disease companies surge on upcoming FDA decision and COVID-19 wins

It has been a bumpy rollercoaster ride for many biopharma companies throughout the pandemic, but overall, the 17 firms that make up BioWorld’s Infectious Disease Index are coming out ahead this year with stocks up by 44.8%. It is no surprise that the gains are largely driven by successes at three companies at the forefront of fighting COVID-19, as well as an anticipated U.S. FDA approval for a urinary tract infection candidate.

Hutchmed earns NMPA approval for savolitinib amid IPO plan

Hutchmed Ltd.’s savolitinib became the first selective MET inhibitor approved in China to treat patients with non-small-cell lung cancer (NSCLC) with MET exon 14 skipping alterations who have progressed following prior systemic therapy or are unable to receive chemotherapy. This marks the third China approval after Elunate (fruquintinib) and Sulanda (surufatinib) for Hutchmed, which is set to launch a $600 million-plus IPO in Hong Kong this month.

mRNA vaccine trigger for myocarditis, but mechanism unknown

Scientists are still scratching their heads over what’s triggering an increased incidence of heart inflammation in young people after they’ve received a second dose of an mRNA COVID-19 vaccine. Several hypotheses have emerged and are being tested, Matthew Oster, of the U.S. CDC, told the Advisory Committee on Immunization Practices in a review of COVID-19 vaccine safety. Although the vaccine-related reports of myocarditis seem to be milder than other instances of the inflammation, “we don’t know the long-term outcomes,” Oster said. To date, males in their late teens and early 20s seem to be most at risk of developing myocarditis within a few days of receiving the second vaccine dose.

Cures 2.0 includes provisions for real-world evidence for drug postmarket study

The follow-up to the 21st Century Cures Act, dubbed Cures 2.0, encodes several anticipated features such as a Medicare coverage mechanism for breakthrough devices and the Advanced Research Projects Agency for Health (ARPA-H). Less expected was a provision for the use of real-world evidence in evaluation of the safety and effectiveness of drugs and biologics after the FDA grants market access, a provision that could prove useful in post-approval evaluations of products such as Biogen Inc.’s Aduhelm, the Alzheimer’s disease therapy that was recently the subject of a controversial FDA approval.

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