Biotech has been depressing lately, but some positive things have recently shown signs of life:
1. ARGX APLS raising $$ on data.
2. Hated IGMS gets a nice deal.
3. SRRA and ATRS M&A.
4. REGN is a buyer and scoops up neglected CMPI
5. Promising early allo CAR-NK data
6. Mavacamten (1st in class, high profile, $13B M&A product) approved.
7. Pfizer acquires $BHVN in the migraine space for $11.6B.
8. Interesting early 'PD-1 knockout' low dose allo data. (100% ORR, 4/5 CR)
9. 100% (9/9) 1 year survival for a lenti gene therapy program in LAD-1, a highly fatal immunodeficiency.
10. Bristol buys Turning Point in oncology for $4.1 billion.
11. Enhertu ‘wows’ #ASCO22 and redefines the treatment of HER2 low expressing breast cancer.
12. Gene therapies for CALD and beta-thalassemia win slam dunk votes during a two-day FDA advisory committee meeting closely watched by industry.
13. CRISPR based gene therapy data presented at #EHA2022 suggests functional cures for sickle cell disease and beta thalassemia.
14. First ever in-vivo gene editing program utilizing CRISPR shows durable serum TTR reduction in transthyretin amyloidosis out to a year. #ILC2022
15. Alnylam announces a positive topline from its P3 of Patisiran in ATTR patients with cardiomyopathy, one of the year's biggest data readouts.
16. CAR-T sales (finally) ramp as products move into earlier stages of treatment for large B-cell lymphoma.
17. Amgen acquires ChemoCentryx for $4 billion and picks up TAVNEOS, a treatment for ANCA-associated vasculitis.
18. Citing the strong data and its alignment with the Cancer Moonshot, FDA approves Enhertu in the HER2 low breast cancer population only two weeks after accepting the application for review. Defining an additional treatment option for an entirely new patient population.
19. Pfizer acquires Global Blood Therapeutics for $5.4 billion, placing a bet on the underserved sickle cell disease community.
20. In a closely watched data readout, Karuna’s muscarinic agonist KarXT succeeds in schizophrenia, potentially offering these patients a first new treatment option mechanism of action in decades.
21. U.S. FDA approves the first gene therapy for the inherited blood disorder beta thalassemia, potentially allowing patients to forgo regular and burdensome blood transfusions.
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