Rocket Pharmaceuticals

At the Cantor Fitzgerald Healthcare Conference, Rocket Pharmaceuticals presented a refocus on three cardiomyopathy programs (Danon, PKP2, BAG3) and reported favorable safety in three Danon patients treated with 3.8E13 vg/kg. The company is preparing approval‑relevant studies with the FDA, cites patient identification as a key hurdle, and runs a free genetic testing program to improve diagnosis. Rocket plans the commercial launch of KRESLADI (for severe LAD‑I) in Q4 2024, is seeking partners for ex‑vivo programs, and expects additional data and regulatory catalysts in the next 9–12 months. » Mais em de.investing.com

Rocket Pharmaceuticals sold a Rare Pediatric Disease Priority Review Voucher for $180 million, bringing pro forma cash, cash equivalents and investments to about $322.6 million and extending expected runway through Q2 2028. The company has restarted dosing in its RP-A501 program, a key step in its near-term recovery and development plan. BofA increased its price target on Rocket to $10 while maintaining a Buy rating following the first-quarter update. » Mais em finance.yahoo.com

Rocket Pharmaceuticals has completed the sale of its priority review voucher for rare pediatric diseases, generating $180 million gross. The FDA had issued the voucher after approval of KRESLADI; pro forma cash rises to about $322.6 million, which the company says is sufficient to fund operations through the second quarter of 2028. The stock is under pressure, trading at $2.66 (market capitalization approx. $290.45 million), down 24% year to date. » Mais em de.investing.com