Aldeyra Therapeutics Inc. CEO Todd Brady said that, with new data from a crossover trial with reproxalap in dry eye disease (DED), the question of “approvability has been put to bed,” and the company plans a pre-NDA meeting with U.S. FDA in the third quarter of this year.
Wall Street cheered a potential, long hoped-for breakthrough in idiopathic pulmonary fibrosis (IPF), and shares of Pliant Therapeutics Inc. (NASDAQ:PLRX) closed at $23, up $14.12, or 159%, after investors learned of positive phase IIa data with PLN-74809. The trial met its primary and secondary endpoints, proving PLN-74809, a dual integrin alpha-V/beta-1/6 antagonist, well-tolerated with a favorable pharmacokinetic (PK) profile. Exploratory efficacy endpoints measured changes in forced vital capacity (FVC) and quantitative lung fibrosis (QLF) imaging, and the drug turned up a dose-dependent treatment effect on FVC and QLF vs. placebo over 12 weeks of treatment. Serum biomarkers were examined, too.
The recent win in Japan by Otsuka Holdings Co. Ltd. subsidiary Taiho Pharmaceutical Co. Ltd. with oral heat shock protein 90 inhibitor Jeselhy (pimitespib) put gastrointestinal stromal tumors (GIST) at center stage. Jeselhy was cleared for GIST cases that have progressed after chemotherapy. A handful of companies line the runway with candidates meant to defeat the resistance that GIST often develops to approved tyrosine kinase inhibitors.
Attention has turned to Cytomx Therapeutics Inc.’s CX-2029 candidate after the company held up its program with the CD166-directed antibody-drug conjugate CX-2009, based on phase II data in patients with hormone receptor-positive/HER2-non-amplified breast cancer.
Treating sooner in the disease course – “hit them hard, hit them early,” as CEO Tahir Mahmood put it – may change outcomes for the better with AMT-101, the candidate from Applied Molecular Transport Inc. (AMT) tested in a combo trial against ulcerative colitis (UC), officials said during a conference call with investors.
The Hail Mary pass by Syros Pharmaceuticals Inc. – shares of which have dwindled severely since Jan. 3 – foreseen by some on Wall Street, came in the form of a $130 million financing and the merger with Tyme Technologies Inc., bringing in-house pipeline assets as well as net cash in a deal that, after accounting for wind-down and transaction expenses, is worth about $60 million.
Recently published real-world data with Firazyr (icatibant), the selective B2 bradykinin receptor antagonist from Takeda Pharmaceutical Co. Ltd. to treat acute hereditary angioedema (HAE) attacks, trained a spotlight on the space, where a handful of players compete. Among the prospects is the early stage but intriguing Star-0215 from Astria Therapeutics Inc., which could be the next advance in the kallikrein-inhibitor class.
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.
