We use cookies to understand how you use our site and to improve your experience.
This includes personalizing content and advertising.
By pressing "Accept All" or closing out of this banner, you consent to the use of all cookies and similar technologies and the sharing of information they collect with third parties.
You can reject marketing cookies by pressing "Deny Optional," but we still use essential, performance, and functional cookies.
In addition, whether you "Accept All," Deny Optional," click the X or otherwise continue to use the site, you accept our Privacy Policy and Terms of Service, revised from time to time.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
BEAM Up on FDA's Orphan Drug Status for Gene-Editing Therapy
Read MoreHide Full Article
Key Takeaways
BEAM-101 has received orphan drug designation from the FDA for treating sickle cell disease.
The designation offers BEAM market exclusivity, fee exemptions, and tax credits post-approval.
BEAM-101 showed efficacy in boosting fetal hemoglobin and reducing sickle hemoglobin in early trials.
Beam Therapeutics (BEAM - Free Report) 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%.
Image Source: Zacks Investment Research
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 - Free Report) and Intellia Therapeutics (NTLA - Free Report) .
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 - Free Report) 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.
Image: Bigstock
BEAM Up on FDA's Orphan Drug Status for Gene-Editing Therapy
Key Takeaways
Beam Therapeutics (BEAM - Free Report) 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%.
Image Source: Zacks Investment Research
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 - Free Report) and Intellia Therapeutics (NTLA - Free Report) .
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 - Free Report) 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.