BEAM Up on FDA's Orphan Drug Status for Gene-Editing Therapy

Beam Therapeutics BEAM announced that the FDA has granted an orphan drug designation to its investigational genome-editing candidate, BEAM-101, for the treatment of sickle cell disease (SCD), an inherited blood disorder.

The FDA grants orphan drug designation to support the development of medicines for rare disorders that affect fewer than 200,000 people in the United States. The designation makes the sponsor eligible to receive seven years of market exclusivity following a potential approval, along with the exemption of FDA application fees and tax credits for qualified clinical studies.

Shares of Beam Therapeutics were up 4.3% yesterday following the announcement of the news.

However, the stock has plunged 32% so far this year compared with the industry’s decline of 2.9%.

BEAM’s Ongoing Development Activities With BEAM-101

BEAM-101 is the lead candidate in the company’s hematology franchise. The phase I/II BEACON study is currently evaluating BEAM-101 for the treatment of adult patients with SCD.

In December 2024, the company announced new safety and efficacy data from the BEACON study evaluating BEAM-101 in SCD patients with severe vaso-occlusive crises.

The data showed that treatment with BEAM-101 led to a robust and durable increase in fetal hemoglobin and a reduction in sickle hemoglobin.

The data suggested that the initial safety profile of BEAM-101 was consistent with busulfan conditioning and autologous hematopoietic stem cell transplantation.

Updated data from the BEACON study is expected to be presented at the European Hematology Association conference shortly.

BEAM's Competition in the Target Market

Besides Beam Therapeutics, a few other companies are also utilizing CRISPR/Cas9 nuclease technology, including CRISPR Therapeutics CRSP and Intellia Therapeutics NTLA.

CRISPR Therapeutics is rapidly leveraging its CRISPR/Cas9 gene-editing platform to develop therapies for the treatment of hemoglobinopathies, cancer and other diseases.

CRISPR Therapeutics and partner Vertex Pharmaceuticals’ VRTX CRISPR/Cas9 gene-edited therapy, Casgevy, was approved for two blood disorder indications, SCD and transfusion-dependent beta thalassemia, in several countries in 2023/2024.

VRTX leads global development, manufacturing, and commercialization of Casgevy and splits program costs and profits worldwide in a 60:40 ratio with CRSP.

Intellia Therapeutics is a clinical-stage gene editing company focused on developing drugs with CRISPR-based therapies.

Intellia is developing nexiguran ziclumeran (nex-z, also known as NTLA-2001), an investigational in vivo CRISPR-based therapy for treating transthyretin (ATTR) amyloidosis. NTLA has collaborated with Regeneron for nex-z.

Intellia is also developing its wholly-owned NTLA-2002, an investigational in vivo CRISPR-based therapy for the treatment of hereditary angioedema.

Both nex-z and nex-z are in late-stage development.

BEAM's Zacks Rank

Beam Therapeutics currently carries a Zacks Rank #3 (Hold). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.

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This article originally published on Zacks Investment Research (zacks.com).

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