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PTC Therapeutics' Spinal Muscular Atrophy Study Successful


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Shares of PTC Therapeutics, Inc. PTCT gained 27.5% on Jun 18, following the presentation of encouraging preliminary data from part I of the pivotal FIREFISH study. The study is evaluating its pipeline candidate, risdiplam (RG7916), in babies with type 1 spinal muscular atrophy ("SMA"). The early-stage data has shown promising motor function improvements and milestones achievement in babies. Data was presented over the last weekend at the Annual SMA Researcher Meeting.

Data from the study showed that risdiplam achieved a four-point increase from baseline in CHOP-INTEND score in more than 90% babies below two years at day 182. CHOP-INTEND is a test to determine motor milestone development of patients with SMA Type 1.

The FIREFISH study is a two-part program in collaboration with Roche RHHBY and SMA foundation. The part I was a dose escalation and safety assessment study. Part II will evaluate the selected dose from part I in babies with type I SMA for 24 months. Enrollment in part II of the FIREFISH study is underway.

Following this presentation, shares of rival company, Biogen Inc. BIIB fell more than 5% on Monday. We remind investors that Biogen's Spinraza is available for the treatment of SMA in pediatric and adult patients. A successful development of risdiplam will likely have an unfavorable impact on Spinraza sales, which generated 14.4% of Biogen's total product sales in the first quarter of 2018.

We note that following the completion of acquisition of AveXis, Novartis will also add a SMA candidates to its pipeline, creating a potential large-cap competitor for Biogen and PTC Therapeutics.

So far this year, PTC Therapeutics' shares have skyrocketed 187.1% compared with a 4.6% increase registered by the industry .

PTC Therapeutics is evaluating risdiplam in several clinical studies apart from FIREFISH. The candidate is being evaluated in another two-part pivotal study, SUNFISH, for the treatment of patients with type II or III SMA. An exploratory study is currently evaluating risdiplam in patients who have received treatment with another therapy targeting SMN2 splicing in clinical studies.

SMA is a rare neuromuscular disorder, which causes muscle atrophy due to the loss of motor neurons within the spinal cord leading to impaired movement in patients. Per the press release, one in 11,000 new-born babies has this debilitating disease.

PTC Therapeutics, Inc. Price

PTC Therapeutics, Inc. Price | PTC Therapeutics, Inc. Quote

Zacks Rank & Stocks to Consider

PTC Therapeutics currently carries a Zacks Rank #3 (Hold).

Aeglea BioTherapeutics, Inc. AGLE is a better-ranked stock in the pharma sector carrying a Zacks Rank #2 (Buy). You can see the complete list of today's Zacks #1 Rank (Strong Buy) stocks here .

Aeglea's loss estimates narrowed from $1.93 to $1.67 for 2018 and from $3.86 to $3.57 for 2019 over the last 60 days. The company delivered positive earnings surprise in three of the trailing four quarters with an average beat of 19.32%. Its share price has increased 101.3% so far this year.

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The views and opinions expressed herein are the views and opinions of the author and do not necessarily reflect those of Nasdaq, Inc.



This article appears in: Investing , Business , Stocks
Referenced Symbols: RHHBY , BIIB , AGLE , PTCT



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