Fidanacogene elaparvovec

A new agreement is the culmination of a four-year research collaboration between Beam Therapeutics and Pfizer and provides an opportunity for milestone payments for Beam. \n After dumping its sole remaining gene therapy asset last year, Pfizer has decided to exercise its option for global rights to Beam Therapeutics’ liver-targeted gene editing candidate.  The agreement, announced in Beam’s end-of-year filing, follows Pfizer’s exit from the gene therapy field last February when it discontinued hemophilia product Beqvez less than a year after securing FDA approval. Beam’s announcement follows a four-year research collaboration between the Cambridge-based biotech and the New York pharma giant that focused on in vivo base editing programs. During the J.P. Morgan Healthcare Conference last month, the companies were tight-lipped about whether Pfizer was going to pick up any Beam candidates.At the end of 2021, the partners announced a research collaboration featuring a $300 million upfront payment to Beam. That deal also gave Beam the chance to garner up to $1.05 billion in milestone payments if Pfizer chose to pick up any of the candidates the companies collaborated on.Now, Pfizer has decided to take over development activities, manufacturing and potential commercialization for an unnamed liver disease asset, which uses Beam’s liver-targeting lipid nanoparticle formulation to deliver base editing reagents. Beam will be able to opt into a global codevelopment and co-commercialization agreement with Pfizer once phase 1/2 clinical trials are complete upon payment of an exercise fee. Under that potential arrangement, Pfizer and Beam would split profits and costs, with Pfizer responsible for 65% of costs and receiving the same proportion of profits.  Pfizer’s move for the Beam candidate follows the FDA’s new draft guidance for an approval pathway that focuses on genome editing and RNA-based treatment methods and aims to remove barriers for bespoke drugs targeting rare diseases. In the same financial disclosure, Beam shared that it also entered a $500 million loan agreement with global investment firm Sixth Street, with principal repayment due by 2033. The money is intended to help launch risto-cel, the company’s investigational sickle cell disease treatment that it plans to submit for FDA approval as early as the end of this year.An investor note from analysts at William Blair gave a positive outlook about the Sixth Street financing, which addressed investors’ capital concerns about a potential risto-cel launch. Further, the financing will help the company continue to invest in its in vivo pipeline, the analysts said. “In our view, risto-cel continues to have increasingly best-in-class potential,” the note says. “With the announced Sixth Street financing and an expected minimum draw of $200 million, the company’s current runway should be sufficient to fund operations into mid-2029.”

Bengaluru: A patient who was being treated with Pfizer's ‍hemophilia drug, Hympavzi, ​as part of a long-term study died after ⁠experiencing serious side effects, the company said. The individual died on December 14 after suffering a stroke followed by a brain hemorrhage, according ‌to the ‌European Haemophilia Consortium, a patient support group. The patient was enrolled in a ‌study that was testing Hympavzi in patients with hemophilia A or B with or without inhibitors. "Pfizer, together with the trial investigator and the independent external Data Monitoring Committee, are actively gathering ​information to better understand the ​complex, multi-factorial circumstances surrounding this occurrence," the company said in ‌a statement. The ‍therapy, a once-a-week injection, gained U.S. ‍approval last year to prevent or reduce bleeding ‌episodes in hemophilia A or B patients aged 12 years and older by targeting blood-clotting proteins. Pfizer does not anticipate any impact to safety for patients treated with the drug based on its current knowledge and the overall clinical data collected to date, the ‍company said. People with hemophilia have a defect in a gene that regulates the production of ‍proteins called ⁠clotting factors, causing ⁠spontaneous and severe bleeding following injuries or surgery. Earlier this year, Pfizer said it would halt global development and commercialization of its hemophilia gene therapy, Beqvez, citing soft demand from patients and their doctors. Beqvez, a one-time therapy, was approved in the U.S. for the treatment of adults with moderate to severe hemophilia B. (Reporting by Sneha S K in Bengaluru; Editing by Anil D'Silva)>