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Arcturus' (ARCT) CF Drug Gets FDA's Orphan Status, Stock Up
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Arcturus Therapeutics Holdings Inc. (ARCT - Free Report) announced that the FDA has granted the Orphan Drug Designation for its product candidate, ARCT-032, for the treatment of cystic fibrosis (CF).
The FDA grants the Orphan Drug Designation to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States. The Orphan Drug Designation will grant Arcturus market exclusivity for ARCT-032 for a predefined time period in the CF indication, along with the exemption of FDA application fees and tax credits for qualified clinical studies, all subject to approval.
Arcturus said that the first CF patient in its early-stage study of ARCT-032 has successfully completed two administrations of the investigational drug candidate. The company is on track to share interim results from the phase Ib study in the first half of 2024.
The company’s stock jumped 7.9% on Monday, as the investors cheered the FDA’s Orphan Drug Designation to ARCT-032 for the CF indication. Year to date, shares of ARCT have surged 52.1% against the industry’s 22.9% fall.
Image Source: Zacks Investment Research
Cystic fibrosis is a highly fatal, inherited disorder that is characterized by abnormal mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, affecting the cells that produce mucus, sweat and digestive juices. Instead of lubricating, the defective gene in CF patients causes the secretions to become sticky and thick, which, in turn, plug up tubes, ducts and passageways, thus severely damaging the lungs, digestive system and other organs in the body.
Arcturus’ ARCT-032 is developed using the company’s proprietary Lunar lipid-mediated aerosolized platform, which follows a novel mechanism of action of delivering CFTR messenger RNA to the lungs. Replacing the absent gene with a functional copy in the lungs of CF patients has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease.
Currently, Vertex Pharmaceuticals (VRTX - Free Report) is the market leader in CF treatments. The company’s lead marketed products are Trikafta, Symdeko/Symkevi, Orkambi and Kalydeco, which are collectively approved to treat more than 75% of the total CF patient population in North America, Europe and Australia.
Trikafta/Kaftrio (brand name of Trikafta in EU), Vertex’s triple combination blockbuster regimen, is approved for the treatment of CF in people aged two years and older who have at least one F508del mutation in the United States and EU. In the nine months ended Sep 30, 2023, VRTX’s CF portfolio generated total revenues worth $7.4 billion.
Vertex is also currently evaluating other candidates in the CF field. It is developing a mRNA therapeutic, VX-522, in partnership with Moderna (MRNA - Free Report) for approximately 5,000 people with CF who cannot benefit from its CFTR modulators. VRTX and MRNA are conducting a single ascending dose (SAD) clinical study on VX-522, after the FDA cleared the investigational new drug application in December 2022. Vertex and Moderna expect to complete the SAD study and initiate the multiple ascending dose study by the end of 2023.
Additionally, VRTX is conducting two pivotal phase III studies evaluating a triple combination of vanzacaftor/VX-561, a CFTR potentiator, deutivacaftor/VX-121, a CFTR corrector and tezacaftor in CF patients aged 12 years and older. This new once-a-day combination medicine has the potential for enhanced patient benefit than those of Trikafta treatment and can potentially treat CF patients who have discontinued Trikafta or other Vertex CF medicines. The studies are expected to be completed by the end of 2023.
Vertex also expects the pivotal studies of vanzacaftor/tezacaftor/deutivacaftor in patients, aged six to 11 years old, to be completed by the end of 2023. Data from all three studies are expected in early 2024.
Arcturus Therapeutics Holdings Inc. Price and Consensus
In the past 30 days, the Zacks Consensus Estimate for Ligand’s 2023 earnings per share has increased from $5.10 to $5.33. During the same time frame, the estimate for Ligand’s 2024 earnings per share has increased from $4.59 to $4.64. Year to date, shares of LGND have lost 11.7%.
LGND’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 67.19%.
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Arcturus' (ARCT) CF Drug Gets FDA's Orphan Status, Stock Up
Arcturus Therapeutics Holdings Inc. (ARCT - Free Report) announced that the FDA has granted the Orphan Drug Designation for its product candidate, ARCT-032, for the treatment of cystic fibrosis (CF).
The FDA grants the Orphan Drug Designation to support the development of medicines for rare disorders that affect fewer than 200,000 patients in the United States. The Orphan Drug Designation will grant Arcturus market exclusivity for ARCT-032 for a predefined time period in the CF indication, along with the exemption of FDA application fees and tax credits for qualified clinical studies, all subject to approval.
Arcturus said that the first CF patient in its early-stage study of ARCT-032 has successfully completed two administrations of the investigational drug candidate. The company is on track to share interim results from the phase Ib study in the first half of 2024.
The company’s stock jumped 7.9% on Monday, as the investors cheered the FDA’s Orphan Drug Designation to ARCT-032 for the CF indication. Year to date, shares of ARCT have surged 52.1% against the industry’s 22.9% fall.
Image Source: Zacks Investment Research
Cystic fibrosis is a highly fatal, inherited disorder that is characterized by abnormal mutations in the cystic fibrosis transmembrane conductance regulator (CFTR) gene, affecting the cells that produce mucus, sweat and digestive juices. Instead of lubricating, the defective gene in CF patients causes the secretions to become sticky and thick, which, in turn, plug up tubes, ducts and passageways, thus severely damaging the lungs, digestive system and other organs in the body.
Arcturus’ ARCT-032 is developed using the company’s proprietary Lunar lipid-mediated aerosolized platform, which follows a novel mechanism of action of delivering CFTR messenger RNA to the lungs. Replacing the absent gene with a functional copy in the lungs of CF patients has the potential to restore CFTR activity and mitigate the downstream effects that cause progressive lung disease.
Currently, Vertex Pharmaceuticals (VRTX - Free Report) is the market leader in CF treatments. The company’s lead marketed products are Trikafta, Symdeko/Symkevi, Orkambi and Kalydeco, which are collectively approved to treat more than 75% of the total CF patient population in North America, Europe and Australia.
Trikafta/Kaftrio (brand name of Trikafta in EU), Vertex’s triple combination blockbuster regimen, is approved for the treatment of CF in people aged two years and older who have at least one F508del mutation in the United States and EU. In the nine months ended Sep 30, 2023, VRTX’s CF portfolio generated total revenues worth $7.4 billion.
Vertex is also currently evaluating other candidates in the CF field. It is developing a mRNA therapeutic, VX-522, in partnership with Moderna (MRNA - Free Report) for approximately 5,000 people with CF who cannot benefit from its CFTR modulators. VRTX and MRNA are conducting a single ascending dose (SAD) clinical study on VX-522, after the FDA cleared the investigational new drug application in December 2022. Vertex and Moderna expect to complete the SAD study and initiate the multiple ascending dose study by the end of 2023.
Additionally, VRTX is conducting two pivotal phase III studies evaluating a triple combination of vanzacaftor/VX-561, a CFTR potentiator, deutivacaftor/VX-121, a CFTR corrector and tezacaftor in CF patients aged 12 years and older. This new once-a-day combination medicine has the potential for enhanced patient benefit than those of Trikafta treatment and can potentially treat CF patients who have discontinued Trikafta or other Vertex CF medicines. The studies are expected to be completed by the end of 2023.
Vertex also expects the pivotal studies of vanzacaftor/tezacaftor/deutivacaftor in patients, aged six to 11 years old, to be completed by the end of 2023. Data from all three studies are expected in early 2024.
Arcturus Therapeutics Holdings Inc. Price and Consensus
Arcturus Therapeutics Holdings Inc. price-consensus-chart | Arcturus Therapeutics Holdings Inc. Quote
Zacks Rank and Stock to Consider
Arcturus currently has a Zacks Rank #3 (Hold).
A better-ranked stock worth mentioning is Ligand Pharmaceuticals (LGND - Free Report) , sporting a Zacks Rank #1 (Strong Buy) at present.
You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 30 days, the Zacks Consensus Estimate for Ligand’s 2023 earnings per share has increased from $5.10 to $5.33. During the same time frame, the estimate for Ligand’s 2024 earnings per share has increased from $4.59 to $4.64. Year to date, shares of LGND have lost 11.7%.
LGND’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 67.19%.