Biotechnology stocks rallied to a record Monday in reaction to a deluge of data from companies studying blood disease treatments presented at the start of the American Society of Hematology (ASH) annual meeting, one of the year’s largest medical conferences.
Wall Street was quick to reward Fate Therapeutics Inc. and partners Crispr Therapeutics AG and Vertex Pharmaceuticals Inc. as early winners. Fate surged 35% to a record and Crispr jumped to a fresh record of its own, while updated lymphoma results from Trillium Therapeutics Inc., which had skyrocketed more than 5,000% in the past year, caused shares to churn.
Here’s a breakdown of some of ASH’s biggest winners and losers:
Crispr Therapeutics and Vertex Pharmaceuticals
The pair of biotechs rose after presenting updated first-in-human results from a study of their one-time treatment CTX001 in patients with beta thalassemia and sickle cell disease. The companies said a total of 10 patients remained free from a once-routine need for blood transfusions and were rid of painful events related to their diseases.
While Wells Fargo downgraded Crispr, citing better-than-expected results that were already reflected in the stock’s more than $10 billion valuation, bulls said the update showcased the therapy’s benefit and durability. Investors were unphased as the stock jumped 5.6% to another record.
Rival companies that are also studying medicines based on Crispr gene editing technology rallied as the results sparked optimism on the platform’s future. Shares of Intellia Therapeutics Inc. jumped 13% to an all-time high while Editas Medicine Inc. gained 9.7%.
The hedge fund favorite erased most of a 6.4% drop after updated results from a study of TTI-622 in patients with advanced relapsed or refractory lymphoma. Cowen analyst Boris Peaker wrote that the data were “incrementally positive” though receptor occupancy may be plateauing.
The company will present data from a study of TTI-621 in patients with relapsed or refractory hematologic malignancies later today and will host a call to discuss the meeting’s results at 4:30 p.m.
Fate surged as much as 35% Monday after positive updates for a pair of key therapies, FT516 and FT596. The update for an early trial of FT516 combined with rituximab in B-cell lymphoma showed some patients were responding to the treatment. The results from the early-stage study of FT596 also showed patients were improving after a second dose.
Analysts praised the results and said they reinforced expectations that the company can disrupt the cell therapy space. Truist’s Robyn Karnauskas wrote that the updates provide two shots on goal in lymphoma with potential upside for shares given multiple readouts due in 2021.
Constellation Pharma jumped as much as 37%, the most since November 2019, after it gave fresh results for its myelofibrosis medicine both as a treatment by itself and in combination with Incyte Corp.’s Jakafi. The data topped most analyst expectations with BMO’s Do Kim saying the results have a positive read-across to its pivotal study.
Increased patient deaths and a rise in those dropping off of therapy could provide bears with more fuel, but bullish analysts see any initial concerns on CPI-0610’s potential genotoxicity as likely to fade with any impact likely “negligible.”
Legend Biotech and Johnson & Johnson
Shares of the pharmaceutical giant were inched lower while its Somerset, New Jersey-based partner erased initial gains to fall 2.2%. The pair said updated Phase 1b/2 data for their CAR-T cell therapy in multiple myeloma patients drove responses in about 97% of patients in the latest update.
Aptose shed a third of its value after updates for a pair of drugs failed to excite investors despite winning praise from Wall Street analysts. News that a pair of patients in the company’s study of CG-806 as a treatment for chronic lymphocytic leukemia had to stop treatment was shrugged off by analysts. Piper Sandler’s Tyler Van Buren said signs of activity are continuing to emerge though “no formal responses were achieved.”
La Jolla, California-based Kura surged as much as 16% in early trading after presenting data from an early study of KO-539 in patients with relapsed or refractory acute myeloid leukemia. The data “demonstrates clear signs of activity with acceptable safety,” wrote Cowen analyst Phil Nadeau.
The biotech gained 8% to a record after presenting additional data from a chronic graft versus host disease study that showed durable patient benefit. Baird wrote that the safety and effectiveness of the drug are tracking with competitor therapies despite being tested in more unmanageable patients.
Baird’s Madhu Kumar wrote that Kura’s KO-539 data were the “best of both worlds” as they showed a “clear win” for the drug’s class while not having a safety advantage over Syndax’s SNDX-5613.
(Updates share movement throughout)