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PTCT Stock Rises on Positive Updates on Friedreich Ataxia Program
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PTC Therapeutics, Inc. (PTCT - Free Report) announced positive results on two different long-term extension studies on vatiquinone.
The company also announced regulatory progress on vatiquinone for the treatment of Friedreich ataxia (FA).
Following the announcement, PTCT stock gained 16.52%.
Year to date, shares of PTC Therapeutics have risen 48% against the industry’s 4.6% decline.
Image Source: Zacks Investment Research
PTCT’s Vatiquinone Positive in Long-Term Analysis
PTCT announced that the pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.
A rare, physically debilitating, life-shortening, neuromuscular disorder, FA, mainly affects the central nervous system and the heart.
MOVE-FA was a global registration-directed trial of vatiquinone that enrolled 146 pediatric, adolescent and adult FA patients, the majority of whom were under 18 years of age. The study included a 72-week placebo-controlled phase and a long-term open-label extension.
The primary endpoint of change from baseline in the overall modified Friedreich Ataxia Rating Scale (mFARS) score did not reach statistical significance. Nonetheless, a statistically significant effect was recorded on the mFARS upright stability subscale, which was a pre-specified endpoint. This subscale is the most relevant and sensitive mFARS component for pediatric and young adults.
In addition, the effect on upright stability was concordant with a favorable treatment effect on the 1-minute walk distance test and the functional component of the Modified Fatigue Rating Scale.
Following completion of MOVE-FA, subjects were eligible to enroll in an ongoing long-term open-label extension study.
Analysis of the MOVE-FA long-term extension study showed that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit on the mFARS compared to a matched natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry.
This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over three years.
Further, results confirmed that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial is maintained over 144 weeks of treatment. In addition, vatiquinone continued to be safe and well tolerated without any treatment-related serious adverse events reported.
In addition to analyzing MOVE-FA, PTCT analyzed long-term open-label data from an earlier study of vatiquinone in adults with FA. Subjects had a 4.8-point benefit on the mFARS relative to a matched natural history population following 24 months of treatment with vatiquinone.
PTCT’s Plans for Vatiquinone
PTCT plans to submit the new drug application (NDA) for vatiquinone for the treatment of FA in December 2024. The NDA will include results from the placebo-controlled portion of the MOVE-FA study. It will also include confirmatory evidence from the two long-term treatment analyses, as well mechanistic data demonstrating treatment effect on biomarkers of disease pathology.
PTCT’s portfolio has two approved products — Translarna (ataluren) and Emflaza (deflazacort) — for the treatment of Duchenne muscular dystrophy.
However, the company is facing challenges pertaining to Translarna’s approval in the EU. It has also suffered a setback in the United States.
PTCT developed Upstaza (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic LAmino Acid Decarboxylase deficiency. It has also collaborated with Roche for the spinal muscular atrophy drug Evrysdi.
PTCT Zacks Rank & Stocks to Consider
PTC Therapeutics currently carries a Zacks Rank #3 (Hold).
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.53 to $4.81. Earnings per share estimates for 2025 have improved from $5.38 to $5.86. Year to date, shares of ANIP have risen 4.1%.
ANIP’s earnings beat estimates in each of the trailing four quarters, the average surprise being 31.32%.
In the past 60 days, estimates for Alnylam’s 2024 loss per share have narrowed from $1.20 to 63 cents. Loss per share estimates for 2025 have narrowed from 34 cents to 27 cents. Year to date, shares of ALNY have rallied 41%.
ALNY’s earnings beat estimates in each of the trailing four quarters, the average surprise being 108.53%.
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PTCT Stock Rises on Positive Updates on Friedreich Ataxia Program
PTC Therapeutics, Inc. (PTCT - Free Report) announced positive results on two different long-term extension studies on vatiquinone.
The company also announced regulatory progress on vatiquinone for the treatment of Friedreich ataxia (FA).
Following the announcement, PTCT stock gained 16.52%.
Year to date, shares of PTC Therapeutics have risen 48% against the industry’s 4.6% decline.
Image Source: Zacks Investment Research
PTCT’s Vatiquinone Positive in Long-Term Analysis
PTCT announced that the pre-specified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.
A rare, physically debilitating, life-shortening, neuromuscular disorder, FA, mainly affects the central nervous system and the heart.
MOVE-FA was a global registration-directed trial of vatiquinone that enrolled 146 pediatric, adolescent and adult FA patients, the majority of whom were under 18 years of age. The study included a 72-week placebo-controlled phase and a long-term open-label extension.
The primary endpoint of change from baseline in the overall modified Friedreich Ataxia Rating Scale (mFARS) score did not reach statistical significance. Nonetheless, a statistically significant effect was recorded on the mFARS upright stability subscale, which was a pre-specified endpoint. This subscale is the most relevant and sensitive mFARS component for pediatric and young adults.
In addition, the effect on upright stability was concordant with a favorable treatment effect on the 1-minute walk distance test and the functional component of the Modified Fatigue Rating Scale.
Following completion of MOVE-FA, subjects were eligible to enroll in an ongoing long-term open-label extension study.
Analysis of the MOVE-FA long-term extension study showed that 144 weeks of vatiquinone treatment resulted in a 3.7-point benefit on the mFARS compared to a matched natural history cohort from the FACOMS (Friedreich Ataxia Clinical Outcome Measures) disease registry.
This treatment difference on the primary endpoint represents a clinically meaningful 50% slowing in disease progression over three years.
Further, results confirmed that the slowing of disease progression recorded in the 72-week placebo-controlled MOVE-FA trial is maintained over 144 weeks of treatment. In addition, vatiquinone continued to be safe and well tolerated without any treatment-related serious adverse events reported.
In addition to analyzing MOVE-FA, PTCT analyzed long-term open-label data from an earlier study of vatiquinone in adults with FA. Subjects had a 4.8-point benefit on the mFARS relative to a matched natural history population following 24 months of treatment with vatiquinone.
PTCT’s Plans for Vatiquinone
PTCT plans to submit the new drug application (NDA) for vatiquinone for the treatment of FA in December 2024. The NDA will include results from the placebo-controlled portion of the MOVE-FA study. It will also include confirmatory evidence from the two long-term treatment analyses, as well mechanistic data demonstrating treatment effect on biomarkers of disease pathology.
PTCT’s portfolio has two approved products — Translarna (ataluren) and Emflaza (deflazacort) — for the treatment of Duchenne muscular dystrophy.
However, the company is facing challenges pertaining to Translarna’s approval in the EU. It has also suffered a setback in the United States.
PTCT developed Upstaza (eladocagene exuparvovec), a gene therapy used for the treatment of Aromatic LAmino Acid Decarboxylase deficiency. It has also collaborated with Roche for the spinal muscular atrophy drug Evrysdi.
PTCT Zacks Rank & Stocks to Consider
PTC Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks from the drug/biotech industry are ANI Pharmaceuticals, Inc. (ANIP - Free Report) and Alnylam Pharmaceuticals, Inc. (ALNY - Free Report) , both sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for ANI Pharmaceuticals’ 2024 earnings per share have moved up from $4.53 to $4.81. Earnings per share estimates for 2025 have improved from $5.38 to $5.86. Year to date, shares of ANIP have risen 4.1%.
ANIP’s earnings beat estimates in each of the trailing four quarters, the average surprise being 31.32%.
In the past 60 days, estimates for Alnylam’s 2024 loss per share have narrowed from $1.20 to 63 cents. Loss per share estimates for 2025 have narrowed from 34 cents to 27 cents. Year to date, shares of ALNY have rallied 41%.
ALNY’s earnings beat estimates in each of the trailing four quarters, the average surprise being 108.53%.