Variable muscle expression of frataxin (FXN) protein in muscle plus five adverse events (AEs) of injection-site thrombophlebitis in the multiple ascending-dose (MAD) phase I study hamstrung Design Therapeutics Inc. in its Friedreich’s ataxia (FA) bid with DT-216. Wall Street punished the Carlsbad, Calif.-based firm’s stock (NASDAQ:DSGN) with a knockdown of 70%, or $5.17, leaving shares at $2.17 when the market closed Aug. 15.
Harmony Biosciences Holdings Inc.’s agreement to acquire Zynerba Pharmaceuticals Inc. for $60 million cash plus as much as $140 million in contingent value rights (CVRs) didn’t register as one of the larger potential outlays recently, but Wall Street had questions about the term sheet just the same. Plymouth Meeting, Pa.-based Harmony is paying about $1.10 per share in cash for Zynerba, of Devon, Pa., plus one non-tradeable CVR tied to clinical, regulatory and sales milestones.
The accelerated clearance won by Johnson & Johnson (J&J) from the U.S. FDA for Talvey (talquetamab-tgvs) for relapsed or refractory multiple myeloma (r/r MM) in adults brings to the market the bispecific antibody that takes aim at G protein-coupled receptor class C group 5 member D, a target of interest to other developers as well.
Terns Pharmaceuticals Inc.’s unveiled positive top-line data from the phase IIa Duet study testing TERN-501, an oral thyroid hormone receptor-beta (THR-B) agonist, given as a monotherapy or in combination with TERN-101, a liver-distributed farnesoid X receptor agonist, for the treatment of nonalcoholic steatohepatitis (NASH).
Alltrna Inc. raised $109 million in a series B round to back its work with transfer RNA, with the first targeted indication of stop codon disease: a condition that takes in thousands of rare and common afflictions stemming from nonsense mutations where the amino acid code has been mutated into a “stop” codon, also known as a premature termination codon.
Wall Street cheered Vistagen Therapeutics Inc.’s first positive phase III trial with a drug for social anxiety disorder (SAD) in 15 years and sent the South San Francisco-based firm’s shares (NASDAQ:VTGN) on a rocket ride to close Aug. 7 at $13.05, up $11.37, or 677%. Vistagen unveiled top-line data from the phase III study called Palisade-2 testing the efficacy, safety, and tolerability of lead asset fasedienol (PH-94B) nasal spray in adults with SAD.
More than two months after Blueprint Medicines Corp. gained a broadened label for Ayvakit (avapritinib), the company is “really pleased to see the diversity of revenue this early in the launch across specialties and across both the academic and community settings,” said Philina Lee, chief commercial officer. The situation “bodes very well for a continued cadence of prescribing [into] the foreseeable future,” she said.
The notion of taking aim at the galectin pathway across varied therapeutic areas has gained major traction in recent years, with a handful of companies drawing Wall Street’s interest as efforts plow forth in conditions as varied as cancer, liver fibrosis and Alzheimer’s disease.
Investors will have to wait for durability of response (DoR) data from Urogen Pharma Ltd.’s phase III Envision study with UGN-102 (mitomycin intravesical solution) in low-grade, intermediate-risk, non-muscle invasive bladder cancer, but urologists already characterize what’s available so far from that study, as well as Atlas – another late-stage experiment – as practice-changing.
Mersana Therapeutics Inc. followed grim June news with an even worse headline as the phase Ib/II study called Uplift with upifitamab rilsodotin (upri) did not meet its primary endpoint in platinum-resistant ovarian cancer (prOC). The antibody-drug conjugate (ADC) targets the sodium-dependent phosphate transport protein NaPi2b and was developed via the company’s platform called Dolaflexin. Shares of Mersana (NASDAQ:MRSN) tumbled 73%, or $2.84, to close July 27 at $1.07 on word of the failure of the trial.
