CEO Eric Dube said Retrophin Inc. will “share more [about clinical development plans] once the deal has closed” in the fourth quarter of this year and Orphan Technologies Ltd. belongs to his firm, which has seen only animal data so far with OT-58, an enzyme replacement therapy (ERT) for classical homocystinuria (HCU).
Second-quarter earnings reports, flowing fast in the last days of July, delivered a bevy of contrasting results, from A to V. Stymied by the pandemic, Aimmune Therapeutics Inc. booked exactly zero net sales during the quarter for its expected blockbuster peanut allergy treatment, Palforzia. Vertex Pharmaceuticals Inc. and Seattle Genetics Inc. beat top-line expectations, driven primarily by strong sales of new products.
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.