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It has been a low-key week for the biotech sector, with not many key updates. Bigwig Regeneron (REGN - Free Report) initiated a study on its investigational COVID-19 antibody cocktail. Other pipeline updates and collaboration deals also grabbed the spotlight.
Recap of the Week’s Most Important Stories:
Regeneron Initiates Study on Antibody Cocktail for Coronavirus: Regeneron has initiated the first study on its experimental dual antibody cocktail, REGN-COV2, for the prevention and treatment of COVID-19.
This program consists of four separate study populations — hospitalized COVID-19 patients, non-hospitalized symptomatic COVID-19 patients, uninfected patients in groups that are at high risk of exposure (such as healthcare workers or first responders) and uninfected patients with close exposure to a COVID-19 patient (such as the patient's housemate). The placebo-controlled studies will be conducted at multiple sites. The first two adaptive phase I/II/III studies are evaluating REGN-COV2 (REGN10933+REGN10987) as a treatment for hospitalized and non-hospitalized patients with COVID-19. The phase I portion will focus on virologic and safety endpoints, while the phase II portion will focus on virologic and clinical endpoints. Data from the phase I and phase II studies will be used to refine the endpoints and determine the size for the phase III studies.
The antibody cocktail approach may also have long-term utility for elderly and immuno-compromised patients, who often do not respond well to vaccines.
Momenta Surges on Pipeline Updates: Momenta Pharmaceuticals surged after it announced positive top-line data from an interim analysis of its mid-stage study on nipocalimab (M281), a high-affinity, fully-human, aglycosylated, effectorless IgG1 anti-FcRn monoclonal antibody.
The multicenter, randomized double-blind, placebo-controlled study, Vivacity-MG, is evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of nipocalimab in 68 patients with moderate-to-severe generalized myasthenia gravis (gMG). The 8-week treatment period included four active arms (5 mg/kg every 4 weeks, 30 mg/kg every 4 weeks, 60 mg/kg every two weeks and a 60 mg/kg single dose) and one placebo arm. Results showed that all four treatment arms demonstrated efficacy in the myasthenia gravis activities of daily living (MG-ADL) score. The study met its primary endpoint with a strong relationship between Immunoglobulin G (IgG) reduction and MG-ADL clinical benefit. Additionally, all dosing arms showed strong safety and tolerability profiles. The study is expected to be completed in the third quarter of 2020.
Vertex Gets EC Approval for Kalydeco: Vertex Pharmaceuticals (VRTX - Free Report) announced that the European Commission has granted label extension for its cystic fibrosis (CF) drug, Kalydeco (ivacaftor). The drug’s label has been expanded to include children and adolescents aged six months to 18 years with the R117H mutation in the CFTR gene, the most common residual function mutation. In Europe, Kalydeco was, until now, approved to treat CF in children aged six months and older with at least one of the specified nine mutations in the CFTR gene. The drug also got a regulatory nod for CF patients aged 18 years and above with R117H mutation in the CFTR gene.
Moreover, the company and partner CRISPR Therapeutics announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 phase I/II trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. Data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress. Data demonstrated clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated.
Ideaya Surged on Partnership With GlaxoSmithKline: Shares of IDEAYA Biosciences, Inc. (IDYA - Free Report) surged after it announced a strategic partnership with pharma bigwig GlaxoSmithKline plc (GSK - Free Report) in Synthetic Lethality, an emerging field in oncology. The collaboration includes IDEAYA's Synthetic Lethality programs — MAT2A, Pol Theta and Werner Helicase — which are projected to reach clinical trials within the next three years. IDEAYA will receive a $100-million upfront cash payment and $20 million from equity purchase of IDEAYA common stock in a direct private placement, and a potential $50 million cash option exercise fee for the MAT2A program. IDEAYA is also entitled to receive other milestone payments. IDEAYA will receive a 50% profit share in the United States for the MAT2A and Werner Helicase programs and is responsible for 20% of global development costs for products being developed with Glaxo.
Biogen Announces Results From NURTURE Study: Biogen Inc. (BIIB - Free Report) announced new results from NURTURE in patients with spinal muscular atrophy (SMA). NURTURE is an ongoing, phase II, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2), who received their first dose of Spinraza before 6 weeks. New data demonstrated that early and sustained treatment with Spinraza for up to 4.8 years enabled unprecedented survival in infants genetically diagnosed with SMA. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease, with 96% now able to walk with assistance. The NURTURE study was recently extended to evaluate the longer-term efficacy and safety of Spinraza in patients up to 8 years of age.
Performance
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index lost 0.29% in the last five trading sessions. Among the biotech giants, Regeneron lost 2.58% during this period. Over the past six months, shares of Regeneron have gained 57.68%. (See the last biotech stock roundup here: Biotech Stock Roundup: GILD's Coronavirus Drug Update, NTLA-REGN Deal & More).
What's Next in Biotech?
Stay tuned for more pipeline updates, with a focus on treatments for the novel COVID-19.
The Hottest Tech Mega-Trend of All
Last year, it generated $24 billion in global revenues. By 2020, it's predicted to blast through the roof to $77.6 billion. Famed investor Mark Cuban says it will produce "the world's first trillionaires," but that should still leave plenty of money for regular investors who make the right trades early.
Image: Bigstock
Biotech Stock Roundup: REGN Begins Coronavirus Study, VRTX, BIIB Give Updates
It has been a low-key week for the biotech sector, with not many key updates. Bigwig Regeneron (REGN - Free Report) initiated a study on its investigational COVID-19 antibody cocktail. Other pipeline updates and collaboration deals also grabbed the spotlight.
Recap of the Week’s Most Important Stories:
Regeneron Initiates Study on Antibody Cocktail for Coronavirus: Regeneron has initiated the first study on its experimental dual antibody cocktail, REGN-COV2, for the prevention and treatment of COVID-19.
This program consists of four separate study populations — hospitalized COVID-19 patients, non-hospitalized symptomatic COVID-19 patients, uninfected patients in groups that are at high risk of exposure (such as healthcare workers or first responders) and uninfected patients with close exposure to a COVID-19 patient (such as the patient's housemate). The placebo-controlled studies will be conducted at multiple sites. The first two adaptive phase I/II/III studies are evaluating REGN-COV2 (REGN10933+REGN10987) as a treatment for hospitalized and non-hospitalized patients with COVID-19. The phase I portion will focus on virologic and safety endpoints, while the phase II portion will focus on virologic and clinical endpoints. Data from the phase I and phase II studies will be used to refine the endpoints and determine the size for the phase III studies.
The antibody cocktail approach may also have long-term utility for elderly and immuno-compromised patients, who often do not respond well to vaccines.
Momenta Surges on Pipeline Updates: Momenta Pharmaceuticals surged after it announced positive top-line data from an interim analysis of its mid-stage study on nipocalimab (M281), a high-affinity, fully-human, aglycosylated, effectorless IgG1 anti-FcRn monoclonal antibody.
The multicenter, randomized double-blind, placebo-controlled study, Vivacity-MG, is evaluating the safety, efficacy, pharmacokinetics and pharmacodynamics of nipocalimab in 68 patients with moderate-to-severe generalized myasthenia gravis (gMG). The 8-week treatment period included four active arms (5 mg/kg every 4 weeks, 30 mg/kg every 4 weeks, 60 mg/kg every two weeks and a 60 mg/kg single dose) and one placebo arm. Results showed that all four treatment arms demonstrated efficacy in the myasthenia gravis activities of daily living (MG-ADL) score. The study met its primary endpoint with a strong relationship between Immunoglobulin G (IgG) reduction and MG-ADL clinical benefit. Additionally, all dosing arms showed strong safety and tolerability profiles. The study is expected to be completed in the third quarter of 2020.
Vertex Gets EC Approval for Kalydeco: Vertex Pharmaceuticals (VRTX - Free Report) announced that the European Commission has granted label extension for its cystic fibrosis (CF) drug, Kalydeco (ivacaftor). The drug’s label has been expanded to include children and adolescents aged six months to 18 years with the R117H mutation in the CFTR gene, the most common residual function mutation. In Europe, Kalydeco was, until now, approved to treat CF in children aged six months and older with at least one of the specified nine mutations in the CFTR gene. The drug also got a regulatory nod for CF patients aged 18 years and above with R117H mutation in the CFTR gene.
Moreover, the company and partner CRISPR Therapeutics announced new clinical data for CTX001, an investigational CRISPR/Cas9 gene-editing therapy, from the CLIMB-111 and CLIMB-121 phase I/II trials in transfusion-dependent beta thalassemia (TDT) and severe sickle cell disease (SCD), and highlighted recent progress in the CTX001 development program. Data were presented during an oral presentation at the European Hematology Association (EHA) virtual congress. Data demonstrated clinical proof-of-concept for CTX001 in TDT. Data include longer-duration follow-up data for the first patient with TDT treated with CTX001 and new data for the second TDT patient treated.
Vertex currently sports a Zacks Rank #1 (Strong Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.
Ideaya Surged on Partnership With GlaxoSmithKline: Shares of IDEAYA Biosciences, Inc. (IDYA - Free Report) surged after it announced a strategic partnership with pharma bigwig GlaxoSmithKline plc (GSK - Free Report) in Synthetic Lethality, an emerging field in oncology. The collaboration includes IDEAYA's Synthetic Lethality programs — MAT2A, Pol Theta and Werner Helicase — which are projected to reach clinical trials within the next three years. IDEAYA will receive a $100-million upfront cash payment and $20 million from equity purchase of IDEAYA common stock in a direct private placement, and a potential $50 million cash option exercise fee for the MAT2A program. IDEAYA is also entitled to receive other milestone payments. IDEAYA will receive a 50% profit share in the United States for the MAT2A and Werner Helicase programs and is responsible for 20% of global development costs for products being developed with Glaxo.
Biogen Announces Results From NURTURE Study: Biogen Inc. (BIIB - Free Report) announced new results from NURTURE in patients with spinal muscular atrophy (SMA). NURTURE is an ongoing, phase II, open-label study of 25 pre-symptomatic patients with the genetic diagnosis of SMA (most likely to develop SMA Type 1 or 2), who received their first dose of Spinraza before 6 weeks. New data demonstrated that early and sustained treatment with Spinraza for up to 4.8 years enabled unprecedented survival in infants genetically diagnosed with SMA. Patients continued to maintain and make progressive gains in motor function compared to the natural course of the disease, with 96% now able to walk with assistance. The NURTURE study was recently extended to evaluate the longer-term efficacy and safety of Spinraza in patients up to 8 years of age.
Performance
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The Nasdaq Biotechnology index lost 0.29% in the last five trading sessions. Among the biotech giants, Regeneron lost 2.58% during this period. Over the past six months, shares of Regeneron have gained 57.68%. (See the last biotech stock roundup here: Biotech Stock Roundup: GILD's Coronavirus Drug Update, NTLA-REGN Deal & More).
What's Next in Biotech?
Stay tuned for more pipeline updates, with a focus on treatments for the novel COVID-19.
The Hottest Tech Mega-Trend of All
Last year, it generated $24 billion in global revenues. By 2020, it's predicted to blast through the roof to $77.6 billion. Famed investor Mark Cuban says it will produce "the world's first trillionaires," but that should still leave plenty of money for regular investors who make the right trades early.
See Zacks' 3 Best Stocks to Play This Trend >>