We use cookies to understand how you use our site and to improve your experience. This includes personalizing content and advertising. To learn more, click here. By continuing to use our site, you accept our use of cookies, revised Privacy Policy and Terms of Service.
You are being directed to ZacksTrade, a division of LBMZ Securities and licensed broker-dealer. ZacksTrade and Zacks.com are separate companies. The web link between the two companies is not a solicitation or offer to invest in a particular security or type of security. ZacksTrade does not endorse or adopt any particular investment strategy, any analyst opinion/rating/report or any approach to evaluating individual securities.
If you wish to go to ZacksTrade, click OK. If you do not, click Cancel.
Roche (RHHBY) Announces Positive Long-Term Data on SMA Drug
Read MoreHide Full Article
Roche (RHHBY - Free Report) announced new long-term data for the spinal muscular atrophy drug (SMA), Evrysdi (risdiplam), from the open-label extension (n=50) of the pivotal FIREFISH study.
FIREFISH is a two-part study in babies aged 1-7 months at the time of enrolment.
The data reinforced the drug’s sustained efficacy and safety profile in children with type 1 SMA.
Most children enrolled in the study continued to show improvement in their ability to sit, stand and walk without support after four years of treatment with Evrysdi. Importantly, the children who were treated and were alive at the time of the primary analysis were still alive at month 48. Moreover, a majority of infants maintained their ability to feed by mouth and swallow up to month 48.
This was pivotal, as untreated children with type 1 SMA are not expected to survive past two years of age and are never able to sit without support.
Among the infants treated with Evrysdi, 37 infants treated with Evrysdi were able to sit without support for at least 5 seconds at month 48 compared with 35 at month 24; and 36 infants were able to sit without support for at least 30 seconds at month 48, up from 23 infants at month 24. Three infants gained the ability to stand alone and one infant gained the ability to walk alone between months 24 and 48.
Per Roche, Evrysdi’s oral route of administration allows the drug to be distributed throughout the body, systemically increasing SMN protein production, a lack of which is the underlying cause of SMA.
We note that Roche is evaluating Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
Apart from FIREFISH, the drug is currently being evaluated in four other trials in SMA patients.
These studies include SUNFISH, a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA; JEWELFISH, an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi and RAINBOWFISH, an open-label, single-arm, multicentre study investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in babies from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms.
Meanwhile, MANATEE is a global phase II/III study evaluating the safety and efficacy of GYM329, an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age.
Roche’s stock has lost 2.1% in the year so far against the industry’s growth of 2.2%.
Image Source: Zacks Investment Research
We note that Evrysdi is already a key contributor to Roche’s top line. Pharmaceuticals Division sales increased by 9% in the first quarter and Evrysdi was one of the top five contributors to this growth, apart from Vabysmo, Ocrevus (multiple sclerosis), Hemlibra (hemophilia) and Tecentriq (cancer immunotherapy).
Lower COVID-19 product sales continue to impact the top line. Sales are likely to be impacted further due to the expected sharp decline in sales of COVID-19 products of roughly CHF 5 billion in 2023. Competition from biosimilars for established cancer medicines like Avastin, MabThera/Rituxan and Herceptin also harmed sales.
Zacks Rank & Stocks to Consider
Roche currently carries a Zacks Rank #3 (Hold).
Some top-ranked stocks in the healthcare sector are Ligand Pharmaceuticals and Novartis (NVS - Free Report) . While Ligand sports a Zacks Rank #1 (Strong Buy), NVS carries a Zacks Rank #2 (Buy).
Over the past 30 days, earnings estimates for LGND have increased by $1.09 to $5.25. LGND topped earnings estimates in two of the last four quarters and missed in the remaining two, the average surprise being 21.50%.
Over the past 60 days, earnings estimates for NVS have increased to $6.74 from $6.60 for 2023. Novartis surpassed estimates in all the trailing four quarters, the average surprise being 5.15%.
See More Zacks Research for These Tickers
Normally $25 each - click below to receive one report FREE:
Image: Bigstock
Roche (RHHBY) Announces Positive Long-Term Data on SMA Drug
Roche (RHHBY - Free Report) announced new long-term data for the spinal muscular atrophy drug (SMA), Evrysdi (risdiplam), from the open-label extension (n=50) of the pivotal FIREFISH study.
FIREFISH is a two-part study in babies aged 1-7 months at the time of enrolment.
The data reinforced the drug’s sustained efficacy and safety profile in children with type 1 SMA.
Most children enrolled in the study continued to show improvement in their ability to sit, stand and walk without support after four years of treatment with Evrysdi. Importantly, the children who were treated and were alive at the time of the primary analysis were still alive at month 48. Moreover, a majority of infants maintained their ability to feed by mouth and swallow up to month 48.
This was pivotal, as untreated children with type 1 SMA are not expected to survive past two years of age and are never able to sit without support.
Among the infants treated with Evrysdi, 37 infants treated with Evrysdi were able to sit without support for at least 5 seconds at month 48 compared with 35 at month 24; and 36 infants were able to sit without support for at least 30 seconds at month 48, up from 23 infants at month 24. Three infants gained the ability to stand alone and one infant gained the ability to walk alone between months 24 and 48.
Per Roche, Evrysdi’s oral route of administration allows the drug to be distributed throughout the body, systemically increasing SMN protein production, a lack of which is the underlying cause of SMA.
We note that Roche is evaluating Evrysdi as part of a collaboration with the SMA Foundation and PTC Therapeutics.
Apart from FIREFISH, the drug is currently being evaluated in four other trials in SMA patients.
These studies include SUNFISH, a two-part, double-blind, placebo-controlled pivotal study in people aged 2-25 years with Types 2 or 3 SMA; JEWELFISH, an open-label exploratory trial designed to assess the safety, tolerability, pharmacokinetics and pharmacodynamics in people with SMA aged 6 months to 60 years who received other investigational or approved SMA therapies for at least 90 days prior to receiving Evrysdi and RAINBOWFISH, an open-label, single-arm, multicentre study investigating the efficacy, safety, pharmacokinetics and pharmacodynamics of Evrysdi in babies from birth to six weeks of age (at first dose) with genetically diagnosed SMA who are not yet presenting with symptoms.
Meanwhile, MANATEE is a global phase II/III study evaluating the safety and efficacy of GYM329, an anti-myostatin molecule targeting muscle growth, in combination with Evrysdi for the treatment of SMA in patients 2-10 years of age.
Roche’s stock has lost 2.1% in the year so far against the industry’s growth of 2.2%.
Image Source: Zacks Investment Research
We note that Evrysdi is already a key contributor to Roche’s top line. Pharmaceuticals Division sales increased by 9% in the first quarter and Evrysdi was one of the top five contributors to this growth, apart from Vabysmo, Ocrevus (multiple sclerosis), Hemlibra (hemophilia) and Tecentriq (cancer immunotherapy).
Lower COVID-19 product sales continue to impact the top line. Sales are likely to be impacted further due to the expected sharp decline in sales of COVID-19 products of roughly CHF 5 billion in 2023. Competition from biosimilars for established cancer medicines like Avastin, MabThera/Rituxan and Herceptin also harmed sales.
Zacks Rank & Stocks to Consider
Roche currently carries a Zacks Rank #3 (Hold).
Some top-ranked stocks in the healthcare sector are Ligand Pharmaceuticals and Novartis (NVS - Free Report) . While Ligand sports a Zacks Rank #1 (Strong Buy), NVS carries a Zacks Rank #2 (Buy).
You can see the complete list of today’s Zacks #1 Rank stocks here.
Over the past 30 days, earnings estimates for LGND have increased by $1.09 to $5.25. LGND topped earnings estimates in two of the last four quarters and missed in the remaining two, the average surprise being 21.50%.
Over the past 60 days, earnings estimates for NVS have increased to $6.74 from $6.60 for 2023. Novartis surpassed estimates in all the trailing four quarters, the average surprise being 5.15%.