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Key highlights in this week include Alexion’s efforts to expand lead drug Soliris label gets a boost while Gilead Sciences (GILD - Free Report) looks to launch generics for HCV drugs. On the other hand, a mixed week for Sarepta Therapeutics (SRPT - Free Report) .
Recap of the Week’s Most Important Stories:
Alexion’s lead drug Soliris Successful in Late-stage Study for NMOSD: Alexion announced that the phase III study, PREVENT, on lead drug Soliris was successful. The Prevention of Relapses and Evaluation of Eculizumab in NMOSD Treatment (PREVENT) trial was a multinational, double-blind, parallel-group time-to-event study that evaluated the efficacy and safety of Soliris compared with placebo for the treatment of patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD). The primary endpoint was the time to first on-trial relapse as adjudicated by an independent committee comprised three external experts in neurology/neuro-ophthalmology, who were blinded to treatment. The study met its primary endpoint. Data showed that treatment with Soliris reduced the risk of NMOSD relapse by 94.2% compared to placebo. Treatment with Soliris reduced the adjudicated on-trial annualized relapse rate compared with placebo, a key secondary endpoint, by 95.5%. 97.9% of patients, who received Soliris were free of relapse compared with only 63.2% of patients who received placebo. As there are currently no approved therapies for NMSOD, the company plans to meet regulatory bodies at the earliest and file for approval in the United States, EU and Japan. Soliris is already approved in the United States and EU for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It is also approved in the EU as the first and only treatment of refractory generalized MG (gMG) in adults who are anti-AchR antibody-positive, and in the United States for the treatment of adult patients with gMG who are anti-AchR antibody-positive.
FDA Lifts Clinical Hold On Sarepta’s DMD Therapy, CHMP Still negative on Exondys: It has been a mixed week for Sarepta. The FDA lifted the clinical hold for the company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. The hold was placed in July 2018 due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot. Consequently, the company developed an action plan with Nationwide Children’s Hospital and submitted to the FDA, including an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.
Separately, Sarepta announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion on a Conditional Marketing Application for its sole marketed drug, Exondys (eteplirsen). The opinion was given in May 2018. The canidate is being evaluated for treating DMD patients who have genetic mutations amenable to exon 51 skipping. Sarepta expects the European Commission (EC) to adopt the CHMP opinion by year-end 2018.
Amarin Surges on Positive Data on Vascepa: Shares of Amarin Corporation plc (AMRN - Free Report) soared after the company reported positive top-line results from the cardiovascular (CV) outcomes trial, REDUCE-IT on Vascepa which is derived from fish oil. The REDUCE-IT cardiovascular outcomes study was initiated in 2011. The study enrolled and followed 8,179 randomized patients, and was conducted based on a special protocol assessment agreement with the FDA. The study met its primary endpoint as data showed that the use of Vascepa resulted in an approximately 25% relative risk reduction in major adverse CV events (MACE) in high-risk individuals as compared with placebo. Vascepa was well tolerated with an acceptable safety profile consistent associated with omega-3 fatty acids and current FDA-approved labeling. REDUCE-IT is the first global cardiovascular outcomes study to assess treatment of patients with LDL-C controlled by statin therapy, persistent elevated triglycerides and other cardiovascular risk factors.
Gilead to Launch Generics for Leading HCV Treatments: Gilead announced that it plans to launch authorized generic versions of its leading hepatitis C virus (HCV) treatments — Epclusa (sofosbuvir 400mg/velpatasvir 100mg) and Harvoni (ledipasvir 90mg/sofosbuvir 400mg). The generic versions will be launched through a newly created subsidiary, Asegua Therapeutics LLC at a list price of $24,000 for the most common course of therapy. These will be available from January 2019. Gilead was under the scanner of regulatory bodies for high prices of drugs a couple of years ago. Gilead decided to launch generics as the company believes that the discounts provided by the company do not always translate into lower costs for patients. The main focus of the company is lowering the list price of Epclusa. Per the company, Epclusa was the first HCV therapy proven to effectively treat all six main Hep C genotypes, with a simple one-pill, once-a-day treatment regimen for the majority of patients. The drug was approved in the United States in 2016.
Acadia Soars on Positive FDA Feedback: Shares of ACADIA Pharmaceuticals Inc. (ACAD - Free Report) soared after the FDA issued a clear statement reaffirming the positive benefit-risk profile of Nuplazid for patients with Parkinson’s disease psychosis. The drug was approved by the FDA a couple of years back for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis, (PDP). However, there were reports of deaths and serious adverse events (SAEs) with the use of Nuplazid. Based on post-marketing reports, the FDA concluded that it did not identify any new or unexpected safety findings with Nuplazid, or findings that are inconsistent with the established safety profile currently described in the drug label. The agency stated that the drug’s benefits outweigh its risks for patients with hallucinations and delusions of PDP.
Performance
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 2.18% over the last five trading sessions. Among the major biotech stocks, Alexion has gained 6.7%. Over the past six months, Biogen has rallied 27.4%. (See the last biotech stock roundup here: Biotech Stock Roundup: CELG, REGN Present Data, VKTX Soars on Drug's Success).
What's Next in Biotech?
Stay tuned for regulatory updates and pipeline development news.
The Hottest Tech Mega-Trend of All
Last year, it generated $8 billion in global revenues. By 2020, it's predicted to blast through the roof to $47 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: ALXN Soliris Successful, Respite For Sarepta, AMRN Soars
Key highlights in this week include Alexion’s efforts to expand lead drug Soliris label gets a boost while Gilead Sciences (GILD - Free Report) looks to launch generics for HCV drugs. On the other hand, a mixed week for Sarepta Therapeutics (SRPT - Free Report) .
Recap of the Week’s Most Important Stories:
Alexion’s lead drug Soliris Successful in Late-stage Study for NMOSD: Alexion announced that the phase III study, PREVENT, on lead drug Soliris was successful. The Prevention of Relapses and Evaluation of Eculizumab in NMOSD Treatment (PREVENT) trial was a multinational, double-blind, parallel-group time-to-event study that evaluated the efficacy and safety of Soliris compared with placebo for the treatment of patients with anti-aquaporin-4 (AQP4) auto antibody-positive neuromyelitis optica spectrum disorder (NMOSD). The primary endpoint was the time to first on-trial relapse as adjudicated by an independent committee comprised three external experts in neurology/neuro-ophthalmology, who were blinded to treatment. The study met its primary endpoint. Data showed that treatment with Soliris reduced the risk of NMOSD relapse by 94.2% compared to placebo. Treatment with Soliris reduced the adjudicated on-trial annualized relapse rate compared with placebo, a key secondary endpoint, by 95.5%. 97.9% of patients, who received Soliris were free of relapse compared with only 63.2% of patients who received placebo. As there are currently no approved therapies for NMSOD, the company plans to meet regulatory bodies at the earliest and file for approval in the United States, EU and Japan. Soliris is already approved in the United States and EU for the treatment of patients with paroxysmal nocturnal hemoglobinuria (PNH) and atypical hemolytic uremic syndrome (aHUS). It is also approved in the EU as the first and only treatment of refractory generalized MG (gMG) in adults who are anti-AchR antibody-positive, and in the United States for the treatment of adult patients with gMG who are anti-AchR antibody-positive.
Alexion carries a Zacks Rank #4 (Sell).
You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
FDA Lifts Clinical Hold On Sarepta’s DMD Therapy, CHMP Still negative on Exondys: It has been a mixed week for Sarepta. The FDA lifted the clinical hold for the company’s Duchenne muscular dystrophy (DMD) micro-dystrophin gene therapy program. The hold was placed in July 2018 due to the presence of trace amounts of DNA fragment in research-grade third-party supplied plasmid in a manufacturing lot. Consequently, the company developed an action plan with Nationwide Children’s Hospital and submitted to the FDA, including an audit of the plasmid supplier and a commitment to use GMP-s plasmid for all future production lots.
Separately, Sarepta announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (EMA) maintained its negative opinion on a Conditional Marketing Application for its sole marketed drug, Exondys (eteplirsen). The opinion was given in May 2018. The canidate is being evaluated for treating DMD patients who have genetic mutations amenable to exon 51 skipping. Sarepta expects the European Commission (EC) to adopt the CHMP opinion by year-end 2018.
Amarin Surges on Positive Data on Vascepa: Shares of Amarin Corporation plc (AMRN - Free Report) soared after the company reported positive top-line results from the cardiovascular (CV) outcomes trial, REDUCE-IT on Vascepa which is derived from fish oil. The REDUCE-IT cardiovascular outcomes study was initiated in 2011. The study enrolled and followed 8,179 randomized patients, and was conducted based on a special protocol assessment agreement with the FDA. The study met its primary endpoint as data showed that the use of Vascepa resulted in an approximately 25% relative risk reduction in major adverse CV events (MACE) in high-risk individuals as compared with placebo. Vascepa was well tolerated with an acceptable safety profile consistent associated with omega-3 fatty acids and current FDA-approved labeling. REDUCE-IT is the first global cardiovascular outcomes study to assess treatment of patients with LDL-C controlled by statin therapy, persistent elevated triglycerides and other cardiovascular risk factors.
Gilead to Launch Generics for Leading HCV Treatments: Gilead announced that it plans to launch authorized generic versions of its leading hepatitis C virus (HCV) treatments — Epclusa (sofosbuvir 400mg/velpatasvir 100mg) and Harvoni (ledipasvir 90mg/sofosbuvir 400mg). The generic versions will be launched through a newly created subsidiary, Asegua Therapeutics LLC at a list price of $24,000 for the most common course of therapy. These will be available from January 2019. Gilead was under the scanner of regulatory bodies for high prices of drugs a couple of years ago. Gilead decided to launch generics as the company believes that the discounts provided by the company do not always translate into lower costs for patients. The main focus of the company is lowering the list price of Epclusa. Per the company, Epclusa was the first HCV therapy proven to effectively treat all six main Hep C genotypes, with a simple one-pill, once-a-day treatment regimen for the majority of patients. The drug was approved in the United States in 2016.
Acadia Soars on Positive FDA Feedback: Shares of ACADIA Pharmaceuticals Inc. (ACAD - Free Report) soared after the FDA issued a clear statement reaffirming the positive benefit-risk profile of Nuplazid for patients with Parkinson’s disease psychosis. The drug was approved by the FDA a couple of years back for the treatment of hallucinations and delusions associated with Parkinson’s disease psychosis, (PDP). However, there were reports of deaths and serious adverse events (SAEs) with the use of Nuplazid. Based on post-marketing reports, the FDA concluded that it did not identify any new or unexpected safety findings with Nuplazid, or findings that are inconsistent with the established safety profile currently described in the drug label. The agency stated that the drug’s benefits outweigh its risks for patients with hallucinations and delusions of PDP.
Performance
Medical - Biomedical and Genetics Industry 5YR % Return
Medical - Biomedical and Genetics Industry 5YR % Return
The NASDAQ Biotechnology Index gained 2.18% over the last five trading sessions. Among the major biotech stocks, Alexion has gained 6.7%. Over the past six months, Biogen has rallied 27.4%. (See the last biotech stock roundup here: Biotech Stock Roundup: CELG, REGN Present Data, VKTX Soars on Drug's Success).
What's Next in Biotech?
Stay tuned for regulatory updates and pipeline development news.
The Hottest Tech Mega-Trend of All
Last year, it generated $8 billion in global revenues. By 2020, it's predicted to blast through the roof to $47 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 >>