As Avidity Biosciences Inc. brought the second-largest follow-on offering of the year to the market, the company also released positive early and midstage stage results of del-zota, an antibody-oligonucleotide conjugate, in treating Duchenne muscular dystrophy. Phase I/II results showed a reversal of disease progression in patients who have been continuously treated for a year, plus improvements in several functional measures.

Pepgen Inc. seems to have gained a leg up on competitors in early data with PGN-EDODM1 in myotonic dystrophy type 1 (DM1), and shares of the Boston-based firm (NASDAQ:PEPG) closed Feb. 24 at $2.29, up 92 cents, or about 67%. The company unveiled initial positive data from the 5- and 10-mg/kg dose cohorts in the ongoing Freedom-DM1 phase I study with PGN-EDODM1, which deploys Boston-based Pepgen’s Enhanced Delivery Oligonucleotide technology to deliver a therapeutic oligonucleotide that is designed to restore the normal function of MBNL1, a key RNA splicing protein.

Pepgen Inc. fell slightly short of its phase II dystrophin goal with PGN-EDO51 for patients with Duchenne muscular dystrophy (DMD) whose mutations are amenable to an exon 51-skipping approach, but Wall Street reacted in a big way, sending the Boston-based firm’s stock (NASDAQ:PEPG) down 33%, or $5.55, to close July 31 at $11.43.

Pepgen Inc. has received a no objection letter from Health Canada for its clinical trial application (CTA) to initiate a phase I study of PGN-EDODM1 in patients with myotonic dystrophy type 1 (DM1). Initial data from the study are expected next year.

IPOs continue to be sluggish but two companies, Pepgen Inc. and Bausch & Lomb Corp., that began trading May 6 managed to sidestep the turbulence despite having to lower their expectations before the market opened.

IPOs continue to be sluggish but two companies, Pepgen Inc. and Bausch & Lomb Corp., that began trading May 6 managed to sidestep the turbulence despite having to lower their expectations before the market opened. Pepgen stock (NASDAQ:PEPG) closed at $12.89 per share May 6, up 7.4% on the day. Bausch & Lomb also had a solid IPO launch May 6 as shares (NYSE:BLCO) closed 11.1% upward at $20 each.