Beam stock launched to a year high Tuesday as enthusiasm grows for its next-generation form of gene editing.
The company is an expert in base editing, a form of gene editing that differs from CRISPR — popularized by Crispr Therapeutics — in that it doesn't require both strands of DNA's double helix to be broken. In theory, this could reduce the risk for unexpected side effects.
Wedbush analyst David Nierengarten says 2024 will be a busy year for Beam Therapeutics.
In its fourth-quarter earnings release, Beam confirmed plans to have the results of a study in patients with sickle cell disease in the second half of the year. That drug is called BEAM-101. The company is also making headway in studies of gene-editing approaches to liver disease and another rare diseases.
"2024 appears to be a busy year for Beam with the potential initiation of two new clinical programs in 2024 and data for BEAM-101 later this year," he said in a note. "We continue to believe in the potential for base editors for treating sickle cell disease."
On the stock market today, Beam stock soared 25.9% to 45.07.
Beam Stock: Rivaling Crispr, Bluebird
Beam is taking a multipronged approach to sickle cell disease, a blood disease that has captured interest from numerous gene-editing companies.
Crispr and partner Vertex Pharmaceuticals have an approved gene-editing drug called Casgevy. Bluebird Bio, meanwhile, won approval for a one-time gene therapy called Lyfgenia.
The first generation of gene-editing approaches to sickle cell disease use the same tact: making genetic edits to cells outside the body. Then they are infused into patients following a conditioning regimen that completely wipes out their bone marrow. That conditioning regimen can make a patient infertile.
But Wedbush's Nierengarten sees "significant headwinds" for Crispr and Bluebird. Further, "because base editors do not generate double-stranded break intermediates, we believe BEAM-101 should have a superior safety profile compared to the existing sickle cell disease therapies," he said.
Nierengarten has an outperform rating on Beam stock.
Beam's first efforts will mirror Crispr's. But, eventually, Beam expects to do its gene editing inside the body, a process known as in vivo. That move is looming, Nierengarten says. The company plans to begin its first in vivo gene-editing study this year.
Extended Cash Runway
Also this year, Beam plans to begin testing its own conditioning regimen that could be easier on patients.
The company is also hoping to start studies in a liver disease known as alpha-1 antitrypsin deficiency, and a rare disease called glycogen storage disease 1a. In the latter condition, glycogen builds up on organs and tissues, causing systemic problems. Both of these will be in vivo gene-editing drugs, Beam stock analyst Sami Corwin, of William Blair, said in a report.
Financially, Leerink Partners analyst Mani Foroohar noted Beam's operating expenses in the December quarter came in higher than expected.
But a deal with Eli Lilly has extended Beam's cash runway out to 2027. Last October, Lilly agreed to buy Beam's opt-in rights to three gene-editing programs for cardiovascular diseases from Verve Therapeutics for $200 million upfront and a $50 million equity infusion.
Beam is also eligible to receive up to $350 million in milestone payments.
Beam stock recently broke out of a consolidation with a buy point at 31.09, MarketSmith.com shows. At one point, shares were 59% above their entry.
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