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Biotechs Get Wall Street Nod on New Blood Disorder Drugs Data

Biotechs Get Wall Street Nod on New Blood Disorder Drugs Data

(Bloomberg) -- Biotechnology stocks initially moved higher as Wall Street cheered updates for a range of therapies to treat blood disorders like sickle cell disease and beta thalassemia.

While the gains evaporated as the broader market struggled to rebound from a rocky session on Thursday, biotechnology companies like Crispr Therapeutics AG and partner Vertex Pharmaceuticals Inc., and Bluebird Bio Inc. saw shares rally to start the morning on the back of promising updates. The Nasdaq Biotechnology Index rose as much as 2.4% on Friday before giving up the advance.

Biotechs Get Wall Street Nod on New Blood Disorder Drugs Data

Crispr Therapeutics and Vertex Pharmaceuticals

Among the most closely watched updates at the European Hematology Association’s virtual annual meeting were new data for Crispr and Vertex’s Crispr-Cas9 gene-editing therapy, CTX001. The results included an additional beta thalassemia patient as well as longer follow-up for two other patients.

All three patients were free from the need for blood transfusions and painful events that came with their disorders. Jefferies analyst Michael Yee called the update an “incremental positive” for Vertex as it showed sustained effectiveness and durability and supports the potential for CTX001 to become a blockbuster drug.

Crispr rose as much as 6.1%, the most intraday in nearly a month, before erasing gains, while Vertex gained as much as 2.8%.

Bluebird Bio

Bluebird rose as much as 7.7% after presenting updated results for a range of programs. A study of LentiGlobin in sickle cell disease showed the therapy continued to rid patients of painful events and the need for red blood cell transfusions. An update in transfusion-dependent beta-thalassemia patients also showcased the medicine’s durability.

“The data disclosed today both sets a high bar for competitors, and supports a high likelihood of approval,” Stifel analyst Benjamin Burnett wrote of the sickle cell data. He said the updated durability is “slightly better than we were expecting.”

Apellis Pharmaceuticals Inc.

Apellis rose as much as 7% after presenting additional details on its experimental medicine in a head-to-head study with Alexion Pharmaceuticals Inc.’s Soliris in a rare, life-threatening form of anemia. Breakthrough hemolysis -- when red blood cells break down -- was reported in 9.8% of patients getting Apellis’s pegcetacoplan compared with 23% of those on Soliris.

Constellation Pharmaceuticals Inc.

Constellation erased initial gains of as much as 19% as investors debated updated data for CPI-0610. A combination with Incyte Corp.’s Jakafi produced a meaningful response for myelofibrosis, though the benefits were lower than seen in prior updates.

JPMorgan analyst Anupam Rama said the benefits shown across various lines of myelofibrosis were “an overall win” ahead of a key late-stage trial expected to start later this year. RBC’s Brian Abrahams recommended buying shares and said there’s a “valuation disconnect versus the $1 billion opportunity and potential for M&A interest.”

Agios Pharmaceuticals Inc.

RBC analyst Kennen MacKay said Agios’s data in sickle cell disease “established initial proof of concept” and were the most significant update for the day.

MacKay said mitapivat could enter pivotal studies next year in sickle cell and said commentary from management was a “particularly bullish signal.” The shares fell as much as 4.8% after earlier rising as much as 5%. The company separately announced a partnership with Royalty Pharma AG.

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