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Pfizer's (PFE) Hemophilia B Gene Therapy Meets Study Goal
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Pfizer (PFE - Free Report) announced positive top-line data from the phase III BENEGENE-2 study, which evaluated fidanacogene elaparvovec, its investigational gene therapy, for the treatment of male adult patients with moderately severe to severe hemophilia B.
The BENGENE-2 study achieved its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post infusion with fidanacogene elaparvovec when compared to those study participants who were administered the current standard-of-care (SOC) of treatment, i.e., a prophylaxis regimen with Factor IX (FIX).
Data from the late-stage study showed a 71% reduction in ABR following a single dose of the gene therapy when compared to SOC. The key secondary endpoints also showed a 78% reduction in treated ABR and a 92% reduction in annualized infusion rate. The company plans to present additional data from the BENGENE-2 study at a future medical meeting.
Pfizer intends to discuss this data with regulatory authorities in early 2023.
Shares of Pfizer have lost 13.1% in the year against the industry’s 11.7% rise.
Image Source: Zacks Investment Research
Hemophilia B is a rare bleeding disorder caused by a missing FIX, known as the clotting protein. Symptoms of the disease include profuse bleeding after an injury, surgery and sometimes even without a clear cause. These bleeding episodes can lead to serious complications for an individual.
Fidanacogene elaparvovec is a one-time gene therapy product consisting of a bio-engineered AAV capsid and a high-activity human coagulation FIX gene. The treatment aims to allow patients to produce FIX themselves without the need to receiving exogenous FIX.
Pfizer has in-licensed fidanacogene elaparvovec gene therapy from Spark Therapeutics, a subsidiary of Roche (RHHBY - Free Report) . The deal was entered into in 2018, a year prior to the acquisition of Spark Therapeutics by Roche in 2019. Per the terms of the licensing deal with the Roche-subsidiary, Pfizer is solely responsible for all pivotal studies as well as the potential commercialization of fidanacogene elaparvovec.
If approved, Pfizer’s gene therapy will face competition from Hemgenix, a one-time gene therapy that was approved by the FDA last month for the treatment of hemophilia B in patients older than 18 years. Hemgenix has been developed by uniQure (QURE - Free Report) in partnership with CSL Behring, the latter being a subsidiary of CSL Limited. Following approval, uniQure/CSL’s Hemgenix became the first FDA-approved gene therapy for treating hemophilia B.
The FDA’s approval of uniQure/CSL’s Hemgenix was based on results from the pivotal HOPE-B study, which demonstrated that following infusion with the gene-therapy, patients produced mean FIX activity of 39% at six months and 36.7% at 24 months. The mean adjusted ABR for all bleeds was also reduced by 54% after seven to 18 months of administering Hemgenix. The clinical development program for HEMGENIX was initially led by uniQure, which was subsequently transferred to CSL after it acquired global rights to commercialize the treatment.
In the past 60 days, estimates for Sanofi’s 2022 earnings per share have risen from $4.04 to $4.27. During the same period, the earnings estimates per share for 2023 have risen from $4.22 to $4.31. Shares of Sanofi have declined 3.1% in the year-to-date period.
Earnings of Sanofi beat estimates in each of the last four quarters, witnessing an earnings surprise of 9.50%, on average. In the last reported quarter, Sanofi’s earnings beat estimates by 9.85%.
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Pfizer's (PFE) Hemophilia B Gene Therapy Meets Study Goal
Pfizer (PFE - Free Report) announced positive top-line data from the phase III BENEGENE-2 study, which evaluated fidanacogene elaparvovec, its investigational gene therapy, for the treatment of male adult patients with moderately severe to severe hemophilia B.
The BENGENE-2 study achieved its primary endpoint of non-inferiority and superiority in the annualized bleeding rate (ABR) of total bleeds post infusion with fidanacogene elaparvovec when compared to those study participants who were administered the current standard-of-care (SOC) of treatment, i.e., a prophylaxis regimen with Factor IX (FIX).
Data from the late-stage study showed a 71% reduction in ABR following a single dose of the gene therapy when compared to SOC. The key secondary endpoints also showed a 78% reduction in treated ABR and a 92% reduction in annualized infusion rate. The company plans to present additional data from the BENGENE-2 study at a future medical meeting.
Pfizer intends to discuss this data with regulatory authorities in early 2023.
Shares of Pfizer have lost 13.1% in the year against the industry’s 11.7% rise.
Image Source: Zacks Investment Research
Hemophilia B is a rare bleeding disorder caused by a missing FIX, known as the clotting protein. Symptoms of the disease include profuse bleeding after an injury, surgery and sometimes even without a clear cause. These bleeding episodes can lead to serious complications for an individual.
Fidanacogene elaparvovec is a one-time gene therapy product consisting of a bio-engineered AAV capsid and a high-activity human coagulation FIX gene. The treatment aims to allow patients to produce FIX themselves without the need to receiving exogenous FIX.
Pfizer has in-licensed fidanacogene elaparvovec gene therapy from Spark Therapeutics, a subsidiary of Roche (RHHBY - Free Report) . The deal was entered into in 2018, a year prior to the acquisition of Spark Therapeutics by Roche in 2019. Per the terms of the licensing deal with the Roche-subsidiary, Pfizer is solely responsible for all pivotal studies as well as the potential commercialization of fidanacogene elaparvovec.
If approved, Pfizer’s gene therapy will face competition from Hemgenix, a one-time gene therapy that was approved by the FDA last month for the treatment of hemophilia B in patients older than 18 years. Hemgenix has been developed by uniQure (QURE - Free Report) in partnership with CSL Behring, the latter being a subsidiary of CSL Limited. Following approval, uniQure/CSL’s Hemgenix became the first FDA-approved gene therapy for treating hemophilia B.
The FDA’s approval of uniQure/CSL’s Hemgenix was based on results from the pivotal HOPE-B study, which demonstrated that following infusion with the gene-therapy, patients produced mean FIX activity of 39% at six months and 36.7% at 24 months. The mean adjusted ABR for all bleeds was also reduced by 54% after seven to 18 months of administering Hemgenix. The clinical development program for HEMGENIX was initially led by uniQure, which was subsequently transferred to CSL after it acquired global rights to commercialize the treatment.
Pfizer Inc. Price
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Zacks Rank & Stocks to Consider
Pfizer currently carries a Zacks Rank #3 (Hold).A better-ranked stock in the overall healthcare sector is Sanofi (SNY - Free Report) , which carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank (Strong Buy) stocks here.
In the past 60 days, estimates for Sanofi’s 2022 earnings per share have risen from $4.04 to $4.27. During the same period, the earnings estimates per share for 2023 have risen from $4.22 to $4.31. Shares of Sanofi have declined 3.1% in the year-to-date period.
Earnings of Sanofi beat estimates in each of the last four quarters, witnessing an earnings surprise of 9.50%, on average. In the last reported quarter, Sanofi’s earnings beat estimates by 9.85%.