Edgewise Therapeutics

The FDA declined to consider Edgewise Therapeutics' sevasemten for accelerated approval in Becker muscular dystrophy, citing insufficient data from the Phase II CANYON trial.

• Haya Therapeutics has raised $65 million in Series A funding to develop therapeutics targeting long non-coding RNAs in the previously dismissed "junk DNA" regions of the human genome. • The company's lead candidate, HTX-100, targets a long non-coding RNA called "Wisper" that plays a central role in non-obstructive hypertrophic cardiomyopathy, with clinical trials expected to begin soon. • Unlike competitors' approaches that focus on reducing heart contractions, Haya's technology aims to address the underlying disease mechanisms by suppressing fibrosis and reprogramming disease-driving cell states.

• Pacira BioSciences has reached a settlement agreement with Fresenius, Jiangsu Hengrui Pharmaceuticals, and eVenus Pharmaceuticals regarding patents for its pain management drug Exparel, protecting market exclusivity until 2030. • Under the agreement, Fresenius will begin limited-volume sales of generic bupivacaine liposome injectable suspension in early 2030, with gradual market share increases reaching the high thirties percent by the final years of the agreement. • The settlement strategically preserves Pacira's market dominance for its lead drug Exparel for nearly a decade before allowing controlled generic entry, significantly extending protection beyond potential patent challenges.

Edgewise Therapeutics received an FDA determination that mid-stage study data for its muscular dystrophy drug was insufficient for accelerated approval, though the agency validated the physical ability endpoint for traditional approval.

• Edgewise Therapeutics' novel cardiac sarcomere modulator EDG-7500 demonstrated significant reductions in left ventricular outflow tract gradient in obstructive HCM patients without meaningful changes in ejection fraction. • The CIRRUS-HCM trial showed dose-dependent improvements in both obstructive and nonobstructive HCM, with the 100mg dose reducing NT-proBNP biomarker levels by 62% and 42% respectively. • EDG-7500's mechanism as a sarcomere modulator may offer advantages over existing cardiac myosin inhibitors like Bristol Myers Squibb's Camzyos, with promising efficacy and safety data after four weeks of treatment.

Edgewise Therapeutics is prioritizing the completion of recruitment for the GRAND CANYON trial for Becker muscular dystrophy in early 2025.

The FDA has placed a clinical hold on PepGen's Phase 2 CONNECT2-EDO51 trial for PGN-EDO51 in Duchenne muscular dystrophy (DMD) due to regulatory questions.

Edgewise Therapeutics' sevasemten met the primary endpoint in its Phase 2 CANYON trial, significantly reducing creatine kinase levels in Becker muscular dystrophy patients.

• Edgewise Therapeutics is advancing EDG-7500 for HCM, with a readout expected in September, showing potential to reduce titration needs. • Sevasemten, targeting BMD, has demonstrated encouraging biomarker changes in the DUNE study, estimating a $1 billion market opportunity. • A comprehensive update on the DMD program is anticipated in H2 2024, with a pivotal trial planned for H1 2025, expanding the pipeline. • Analysts view Edgewise favorably, citing novel mechanisms and potential for significant market penetration if clinical trials yield positive results.

• Wave Life Sciences reported positive interim results for WVE-N531, an exon-skipping oligonucleotide, showing promising dystrophin expression in patients with Duchenne muscular dystrophy. • Sarepta Therapeutics expanded the label for its gene therapy Elevidys to include all patients aged four and older with mutations in the dystrophin gene. • Italfarmaco's Duvyzat (givinostat), the first nonsteroidal treatment for all genetic variants of Duchenne, received FDA approval based on phase 3 trial results. • Avidity Biosciences' delpacibart zotadirsen (del-zota) demonstrated a significant increase in dystrophin production and reduction of creatine kinase levels in a phase 1/2 trial.