According to an analysis conducted by BioWorld of the second-quarter 2020 financial reports filed by 120 public biopharmaceutical companies with market caps greater than $1 billion, and excluding big pharma companies, the amount that was invested in research and development during the period increased by 14% compared to the same six-month period last year.
Passed up for acquisition by former partner Retrophin Inc., Censa Pharmaceuticals Inc. has found a new home in rare disease specialist PTC Therapeutics Inc., which has agreed to pay $10 million up front for the opportunity to develop CNSA-001 (sepiapterin), a candidate for orphan metabolic diseases, starting with phenylketonuria (PKU). The proposed transaction also includes up to 850,000 shares of PTC common stock (NASDAQ:PTCT), valued around $40 million, plus additional rewards for achieving development, regulatory and commercial milestones. Shares of PTC closed at $46.91 on May 7, down $1.31.
DUBLIN – Shares in PTC Therapeutics Inc. dropped as much as 13% during premarket trading Feb. 6 on eagerly awaited 12-month data for its oral spinal muscular atrophy (SMA) drug risdiplam, which is licensed to Genentech. Although patients with type 2 or type 3 SMA demonstrated statistically significant improvements in motor function, the level of that improvement evidently disappointed some investors.
Roche Holding AG and PTC Therapeutics Inc. together with their partner, the SMA Foundation, reported success in part two of the pivotal Sunfish study testing their survival motor neuron-2 splicing modifier risdiplam in patients with type 2 or 3 spinal muscular atrophy (SMA).