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PTC Therapeutics' (PTCT) Rare Disease Drug Fails in Study

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Shares of PTC Therapeutics (PTCT - Free Report) lost 21.3% on May 24, after management announced that the phase III MOVE-FA study, evaluating vatiquinone in patients with Friedreich ataxia (“FA”), failed to achieve its primary endpoint of a significant change in modified Friedreich Ataxia Rating Scale (“mFARS”) score.

The MOVE-FA study failed to achieve statistically significant change in mFARS score at 72 weeks in the primary analysis population. The mFARS is a clinical assessment which measures disease progression, namely swallowing and speech, upper and lower limb coordination and upright stability.

The MOVE-FA study did show that treatment with vatiquinone led to benefits on key disease subscales and secondary endpoints in both pediatric and adult patients.

Based on signals of clinical benefit observed in the above study, management plans to begin discussions with regulatory authorities to discuss the path for a potential regulatory approval.

FA is an ultra-rare genetic, progressive and neurodegenerative movement disorder that affects approximately 25,000 people across the globe. This disease, usually diagnosed during childhood/adolescence, causes progressive loss of coordination, muscle weakness and fatigue, eventually resulting in loss of mobility and death.

In the year so far, shares of PTC Therapeutics have surged 53% compared with the industry’s 0.8% growth.

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Currently, Reata Pharmaceuticals’ Skyclarys (omaveloxolone) is the only FDA-approved treatment in FA indication. Reata’s Skyclarys, which was granted FDA approval this February, is approved for use in adults and adolescents aged 16 and older. This drug is also Reata’s first commercial drug. Earlier this month, Reata completed the final stages of manufacturing and packaging for Skyclarys but needs an NDA supplement approval to release the drug to the specialty pharmacy. This NDA supplement is currently under FDA review with an approval target action date expected in mid-August 2023. Reata expects Skyclarys to be launched by the same date if the FDA finds no issues with the review.

In a separate press release, PTC Therapeutics also announced that it has decided to discontinue pre-clinical and early research programs in gene therapy. The discontinued programs include preclinical stage programs in FA and Angelman syndrome as well as several other programs targeting rare CNS and ophthalmological disorders of high unmet medical need. However, PTCT will continue its development and global commercial activities on Upstaza, a gene-therapy approved in Europe for treating aromatic L-amino acid decarboxylase (AADC) deficiency in patients aged 18 months and older.

The above decision is a part of a strategic portfolio prioritization undertaken by management that intends to focus resources on areas that are likely to provide a significant return on its R&D investments. As a result of this decision, management expects a reduction of nearly 15% in residual 2023 operating expenses (OPEX). The company will provide guidance on its 2023 OPEX when it reports second-quarter 2023 earnings.

PTC also announced that its chief financial officer (CFO) Emily Hill was relieved of her responsibilities and is leaving the organization.


Zacks Rank & Stocks to Consider

PTC Therapeutics currently carries a Zacks Rank #3 (Hold). A couple of better-ranked stocks in the overall healthcare sector include ANI Pharmaceuticals (ANIP - Free Report) and Allogene Therapeutics (ALLO - Free Report) . While ANI Pharmaceuticals sports a Zacks Rank #1 (Strong Buy), Allogene carries a Zacks Rank #2 (Buy). You can see the complete list of today’s Zacks #1 Rank stocks here.

In the past 60 days, estimates for ANI Pharmaceuticals’ 2023 earnings per share have increased from $2.42 to $3.31. During the same period, the earnings estimates per share for 2024 have risen from $3.76 to $4.32.  Shares of ANI Pharmaceuticals are up 16.0% in the year-to-date period.

Earnings of ANI Pharmaceuticals beat estimates in each of the last four quarters, delivering an average earnings surprise of 68.64%. In the last reported quarter, ANI Pharmaceuticals’ earnings beat estimates by 244.12%.

In the past 60 days, estimates for Allogene Therapeutics’ 2023 loss per share have improved from $2.45 to $2.32. During the same period, the loss estimates per share for 2024 have narrowed from $2.48 to $2.21. Shares of Allogene Therapeuticshave declined 3.8% in the year-to-date period.

Earnings of Allogene Therapeutics beat estimates in three of the last four quarters while missing the mark on one occasion. On average, the company’s earnings witnessed a surprise of 5.08%. In the last reported quarter, Allogene Therapeutics’ earnings beat estimates by 7.94%.

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