Blueprint Medicines Corp. CFO Mike Landsittel called his firm’s potential $1.25 billion financing deals with Sixth Street and Royalty Pharma “once in a lifetime” arrangements that came at the end of a competitive process in a formidable cash environment.
Trevi Therapeutics Inc. took its place among several late-stage contenders by scoring a win in prurigo nodularis in the form of positive data from the 360-participant, phase IIb/III Pruritus Relief through Itch-Scratch Modulation, or PRISM, trial with oral Haduvio (nalbuphine extended release).
After its $247 million acquisition of Epizyme Inc., Ipsen SA will continue to shop for M&A prospects, said Ipsen CEO David Loew, noting that “the [company’s] firepower has not been absorbed in a significant fashion by this deal,” and the firm might undertake a larger transaction next time in oncology, rare diseases, or neurology.
Sarepta Therapeutics Inc. aims to resolve “very quickly” the clinical hold placed by the U.S. FDA on SRP-5051 (vesleteplirsen) for Duchenne muscular dystrophy (DMD), said Louise Rodino-Klapac, chief scientific officer.
Uniqure NV rolled out stock-pleasing safety and biomarker data from 10 patients enrolled in the low-dose cohort of the ongoing phase I/II trial with AMT-130 for the treatment of Huntington’s disease (HD), but investors must wait for details on MRI scans. “We have communicated that we will discuss the MRI findings in the middle of next year,” CEO Matt Kapusta told investors during a conference call. “That’s all I can say.”
Shares of Athira Pharma Inc. (NASDAQ:ATHA) plunged almost 66.3%, or $5.60, to close at $2.85 on June 22 after the company disclosed top-line results from its exploratory phase II study with fosgonimeton (ATH-1017, fosgo) in mild-to-moderate Alzheimer’s disease (AD).
After an up-and-down day – mostly up, toward the end – during which the phrase “totality of the data” got air time aplenty, shares of PTC Therapeutics Inc. (NASDAQ:PTCT) closed at $34.07, a rise of $5.66, or almost 20%, on word of top-line data from Study 041 with Translarna (ataluren) in nonsense mutation Duchenne muscular dystrophy (DMD).
The U.S. FDA’s Psychopharmacologic Drugs Advisory Committee took up the complicated matter of Acadia Pharmaceuticals Inc.’s sNDA for Nuplazid (pimavanserin) to treat hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP).
The FDA’s Psychopharmacologic Drugs Advisory Committee posted briefing documents related to the June 17 meeting, set to consider Acadia Pharmaceuticals Inc.’s sNDA for Nuplazid (pimavanserin) to treat hallucinations and delusions associated with Alzheimer’s disease psychosis (ADP). Although shares of the San Diego-based firm (NASDAQ:ACAD) stayed in the black, closing at $18.77, up $2.52, or 15.5%, the briefing docs did not bring uniformly good news, echoing some of the concerns spelled out in an earlier complete response letter.
With Alnylam Pharmaceuticals Inc.’s FDA clearance for Amvuttra (vutrisiran) in the rearview mirror, investors are looking ahead to potentially label-widening phase III data related to another, already approved drug: Onpattro (patisiran).
