PTC Therapeutics Inc. (PTCT) said that it has discontinued pre-clinical and early research programs in gene therapy as part of a strategic portfolio prioritization.
However, PTC said it will continue its development and global commercialization of Upstaza, the first-ever approved gene therapy directly administered to the brain. As a result of the prioritization, estimated reductions of about fifteen percent in residual 2023 operating expenses (OPEX) are expected.
PTCT closed Tuesday's regular trading at $58.41 down $0.69 or 1.17%. In the after-hours trade, the stock further dropped $12.41 or 21.25%.
The discontinuation of the gene therapy programs will also result in a reduction in workforce which will be conducted in accordance with appropriate labor regulations.
The discontinued gene therapy programs include preclinical stage programs in Friedreich ataxia and Angelman syndrome as well as several other programs targeting rare CNS and ophthalmological disorders of high unmet medical need at various stages of preclinical development. The prioritization decision will allow for additional focus on PTC's proprietary splicing platform as well as additional CNS and metabolic disorders that leverage its differentiated and innovative scientific expertise.
In addition, PTC Therapeutics announced that its Chief Financial Officer Emily Hill has been relieved of her responsibilities and will be leaving the organization.
In a separate press release, PTC Therapeutics said that phase 3 trial of vatiquinone in patients with Friedreich ataxia did not meet its primary endpoint of statistically significant change in mFARS score at 72 weeks in the primary analysis population.
However, vatiquinone treatment did demonstrate significant benefit on key disease subscales and secondary endpoints. In addition, in the population of subjects that completed the study protocol, significance was reached in the mFARS endpoint and several secondary endpoints.
Friedreich ataxia is a rare, physically debilitating, life-shortening, neuromuscular disorder that mainly affects the central nervous system and the heart.
The Phase 3 registration-directed trial in Friedreich Ataxia patients, called MOVE-FA, is a randomized, placebo-controlled 72-week trial with the primary endpoint being change in the modified Friedreich Ataxia Rating Scale (mFARS) score.
The mFARS is a clinical assessment that measures disease progression, namely swallowing and speech, upper and lower limb coordination, and upright stability.
The MOVE-FA trial enrolled 146 pediatric and adult patients, the majority of which were under 18 years of age. On a prespecified sensitivity analysis of subjects who completed 72 weeks on assigned therapy, the placebo corrected difference was 2.31, which represents a 75% slowing in disease progression over 72 weeks. Overall, vatiquinone was demonstrated to be well tolerated, adding to the large volume of safety data collected in other pediatric clinical studies.
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