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Biogen's (BIIB) Rare Disease Drug Skyclarys Gets CHMP Nod in EU
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Biogen Inc. (BIIB - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (“EMA”) has adopted a positive opinion, recommending the marketing authorization for Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in patients aged 16 years and older.
FA is an ultra-rare genetic, progressive and neurodegenerative movement disorder. This disease, which is usually diagnosed during adolescence, causes progressive loss of coordination, muscle weakness and fatigue, eventually resulting in loss of mobility and finally death.
Subject to approval by the European Commission (EC), Skyclarys will become the first treatment authorized for FA within the EU.
The CHMP’s recommendation, although not legally binding, will be considered by the EC in its decision-making process for the marketing authorization of Skyclarys in the EU for FA. A final decision from the regulatory body is expected in the first quarter of 2024.
Per the press release, the positive CHMP opinion was based on upbeat efficacy and safety data from the MOXIe Part 2 study of Skyclarys for the treatment of FA. After 48 weeks, data from the study showed that treatment with Skyclarys resulted in a reduction of physical impairment compared to patients who received a placebo, as measured by the modified Friedreich Ataxia Rating Scale (MFARS). mFARS is a metric used in the assessment of the severity of neurologic features of FA.
Several other benefits across subscales of mFARS were also observed in FA patients treated with Skyclarys. The positive CHMP opinion was also fueled by additional long-term data from the MOXIe (Parts 1 and 2) clinical program, where patients treated with Skyclarys had lower mFARS scores at three years, as compared to a matched natural history group. Common side effects of treatment with the drug were also mild in severity.
Year to date, shares of Biogen have lost 10.4% compared with the industry’s 17.7% decline.
Image Source: Zacks Investment Research
Skyclarys was added to Biogen’s portfolio with the September 2023 acquisition of Reata Pharmaceuticals. It was approved by the FDA in February 2023 for the treatment of FA in patients aged 16 years.
Biogen expects Skyclarys to provide potential operating synergies with the company’s spinal muscular atrophy drug, Spinraza (nusinersen) and amyotrophic lateral sclerosis drug, Qalsody (tofersen).
On the last earnings call, Biogen’s management said that Skyclarys generated sales of $43 million in the third quarter of 2023, which appears strong. However, Skyclarys sales were not included in the third-quarter revenues as the acquisition closed in September. The sales indicate that the drug has the potential to boost Biogen’s revenues in the quarters ahead.
It is important to note that another company that is making a drug for FA is PTC Therapeutics (PTCT - Free Report) .
PTC Therapeutics is developing vatiquinone in phase III pivotal studies for children and young adults with FA. PTC Therapeutics’ vatiquinone has been granted Orphan Drug Designation and Fast Track Designation by the FDA. In May 2023, PTCT reported top-line results from its MOVE-FA study on vatiquinone for the treatment of FA.
The study failed to achieve its primary endpoint of statistically significant change in mFARS score at week 72 in the primary analysis population. However, significant benefits on key disease subscales and secondary endpoints were observed upon treatment with vatiquinone. Furthermore, PTCT reported that the mFARS score and several secondary endpoints reached significance in the population of patients who completed the study protocol.
PTC Therapeutics is currently holding dialogues with the FDA to figure out a regulatory pathway for vatiquinone in the treatment of FA. The company believes that a confirmatory study would be required to support a regulatory filing for the investigational drug in the United States. Simultaneously, the company is also holding discussions with the EMA for the regulatory pathway of vatiquinone in the EU.
In the past 30 days, the Zacks Consensus Estimate for Puma Biotech’s 2023 earnings per share (EPS) has decreased from 73 cents to 72 cents. During the same time frame, the consensus estimate for Puma Biotech’s 2024 EPS has increased from 62 cents to 64 cents. In the year so far, shares of PBYI have lost 7.3%.
PBYI’s earnings beat estimates in three of the last four quarters while missing on one occasion, delivering a four-quarter average earnings surprise of 76.55%.
In the past 30 days, the Zacks Consensus Estimate for ADMA Biologics’ 2023 loss per share has remained constant at 3 cents. The consensus estimate for ADMA Biologics’ 2024 EPS is pegged at 16 cents. In the year so far, shares of ADMA have gained 8.2%.
ADMA beat estimates in three of the trailing four quarters and matched in one, delivering an average earnings surprise of 63.57%.
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Biogen's (BIIB) Rare Disease Drug Skyclarys Gets CHMP Nod in EU
Biogen Inc. (BIIB - Free Report) announced that the Committee for Medicinal Products for Human Use (CHMP) of the European Medicines Agency (“EMA”) has adopted a positive opinion, recommending the marketing authorization for Skyclarys (omaveloxolone) for the treatment of Friedreich’s ataxia (FA) in patients aged 16 years and older.
FA is an ultra-rare genetic, progressive and neurodegenerative movement disorder. This disease, which is usually diagnosed during adolescence, causes progressive loss of coordination, muscle weakness and fatigue, eventually resulting in loss of mobility and finally death.
Subject to approval by the European Commission (EC), Skyclarys will become the first treatment authorized for FA within the EU.
The CHMP’s recommendation, although not legally binding, will be considered by the EC in its decision-making process for the marketing authorization of Skyclarys in the EU for FA. A final decision from the regulatory body is expected in the first quarter of 2024.
Per the press release, the positive CHMP opinion was based on upbeat efficacy and safety data from the MOXIe Part 2 study of Skyclarys for the treatment of FA. After 48 weeks, data from the study showed that treatment with Skyclarys resulted in a reduction of physical impairment compared to patients who received a placebo, as measured by the modified Friedreich Ataxia Rating Scale (MFARS). mFARS is a metric used in the assessment of the severity of neurologic features of FA.
Several other benefits across subscales of mFARS were also observed in FA patients treated with Skyclarys. The positive CHMP opinion was also fueled by additional long-term data from the MOXIe (Parts 1 and 2) clinical program, where patients treated with Skyclarys had lower mFARS scores at three years, as compared to a matched natural history group. Common side effects of treatment with the drug were also mild in severity.
Year to date, shares of Biogen have lost 10.4% compared with the industry’s 17.7% decline.
Image Source: Zacks Investment Research
Skyclarys was added to Biogen’s portfolio with the September 2023 acquisition of Reata Pharmaceuticals. It was approved by the FDA in February 2023 for the treatment of FA in patients aged 16 years.
Biogen expects Skyclarys to provide potential operating synergies with the company’s spinal muscular atrophy drug, Spinraza (nusinersen) and amyotrophic lateral sclerosis drug, Qalsody (tofersen).
On the last earnings call, Biogen’s management said that Skyclarys generated sales of $43 million in the third quarter of 2023, which appears strong. However, Skyclarys sales were not included in the third-quarter revenues as the acquisition closed in September. The sales indicate that the drug has the potential to boost Biogen’s revenues in the quarters ahead.
It is important to note that another company that is making a drug for FA is PTC Therapeutics (PTCT - Free Report) .
PTC Therapeutics is developing vatiquinone in phase III pivotal studies for children and young adults with FA. PTC Therapeutics’ vatiquinone has been granted Orphan Drug Designation and Fast Track Designation by the FDA. In May 2023, PTCT reported top-line results from its MOVE-FA study on vatiquinone for the treatment of FA.
The study failed to achieve its primary endpoint of statistically significant change in mFARS score at week 72 in the primary analysis population. However, significant benefits on key disease subscales and secondary endpoints were observed upon treatment with vatiquinone. Furthermore, PTCT reported that the mFARS score and several secondary endpoints reached significance in the population of patients who completed the study protocol.
PTC Therapeutics is currently holding dialogues with the FDA to figure out a regulatory pathway for vatiquinone in the treatment of FA. The company believes that a confirmatory study would be required to support a regulatory filing for the investigational drug in the United States. Simultaneously, the company is also holding discussions with the EMA for the regulatory pathway of vatiquinone in the EU.
Biogen Inc. Price and Consensus
Biogen Inc. price-consensus-chart | Biogen Inc. Quote
Zacks Rank and Stocks to Consider
Biogen currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks worth mentioning are Puma Biotechnology, Inc. (PBYI - Free Report) and ADMA Biologics (ADMA - Free Report) . While PBYI sports a Zacks Rank #1 (Strong Buy), ADMA carries a Zacks Rank #2 (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 Puma Biotech’s 2023 earnings per share (EPS) has decreased from 73 cents to 72 cents. During the same time frame, the consensus estimate for Puma Biotech’s 2024 EPS has increased from 62 cents to 64 cents. In the year so far, shares of PBYI have lost 7.3%.
PBYI’s earnings beat estimates in three of the last four quarters while missing on one occasion, delivering a four-quarter average earnings surprise of 76.55%.
In the past 30 days, the Zacks Consensus Estimate for ADMA Biologics’ 2023 loss per share has remained constant at 3 cents. The consensus estimate for ADMA Biologics’ 2024 EPS is pegged at 16 cents. In the year so far, shares of ADMA have gained 8.2%.
ADMA beat estimates in three of the trailing four quarters and matched in one, delivering an average earnings surprise of 63.57%.