While Dyne Therapeutics Inc.’s DYNE-251 demonstrated high levels of dystrophin expression and functional improvement in boys with Duchenne muscular dystrophy who are amenable to exon 51 skipping, investors focused on three serious adverse events related to the drug, driving shares (NASDAQ:DYNE) down by 31% on Sept. 3.
The BioWorld Biopharmaceutical Index climbed 16.31% by the end of July, continuing its lead over both the Nasdaq Biotechnology Index, which rose 10.82%, and the Dow Jones Industrial Average, up 8.37%. This follows an 8.15% gain in the BBI at the close of May, marking a strong performance throughout 2024.
In the first five months of 2024, the BioWorld Neurological Diseases Index (BNDI) saw a 10.35% decline, underperforming both the Nasdaq Biotechnology Index (NBI), which rose by 0.7%, and the Dow Jones Industrial Average (DJIA), which increased by 2.64%. This represents an additional drop for BNDI, which closed February with a 4.1% decline. In contrast, in 2023, the BNDI closed with a 4.36% increase, outperforming the NBI, which rose by 3.74%, but not matching the 13.7% rise seen in the DJIA.
Sarepta Therapeutics Inc. CEO Douglas Ingram said he expects “ferocious” demand for gene therapy Elevidys (delandistrogene moxeparvovec), granted full approval by the U.S. FDA for Duchenne muscular dystrophy (DMD). Shares of the Cambridge, Mass.-based firm closed June 21 at $16.72, up $37.22, or about 30% on the news.
The good news for Sarepta Therapeutics Inc. is bad news for Pfizer Inc. as the phase III study of its mini-dystrophin gene therapy in Duchenne muscular dystrophy (DMD) has missed its primary endpoint. Now Sarepta’s Elevidys (delandistrogene moxeparvovec), a single-dose, adeno-associated virus-based gene transfer therapy for DMD, is barreling toward a June 21 PDUFA date with the U.S. FDA as the near competition shrinks in the rearview mirror.
A boy participating in the phase II Daylight study of Duchenne muscular dystrophy (DMD) “has passed away suddenly,” according to Pfizer Inc. The participant had received fordadistrogene movaparvovec, a mini-dystrophin gene therapy, in early 2023. The fatal serious adverse event was reported May 3 as a cardiac arrest, Pfizer told BioWorld. Pfizer, together with the independent external data monitoring committee, is reviewing the data to understand the potential cause, the company added.
The U.S. FDA has approved Duvyzat (givinostat), from Italfarmaco SpA, for treating Duchenne muscular dystrophy (DMD). It is the first oral, nonsteroidal drug for treating all of DMD’s genetic variants. The oral treatment is approved for those ages 6 and older.
In 2023, the BioWorld Neurological Diseases Index (BNDI) closed with a 4.36% increase, outperforming the Nasdaq Biotechnology Index (up 3.74%) but falling short of the surge seen in the Dow Jones Industrial Average (up 13.7%). The rebound marked a significant turnaround from November, when BNDI recorded an 18.37% year-to-date decline.
Sarepta Therapeutics Inc.’s next-generation peptide-conjugated PMO therapy, SRP-5051 (vesleteplirsen), looks set to stake its claim in the Duchenne muscular dystrophy (DMD) space, as phase II data unveiled dystrophin expression that outperforms first-generation exon-skipping drug Exondys 51 (eteplirsen). The question is what that space might look like in the wake of a U.S. FDA decision whether to expand labeling and convert to full approval Sarepta’s DMD gene therapy, Elevidys (delandistrogene moxeparvovec).
Edgewise Therapeutics Inc. priced an underwritten offering of 21.8 million shares at $11 each as it looks for about $240 million in gross proceeds to develop its Duchenne and Becker muscular dystrophies treatment. The offering propelled the company’s stock (NASDAQ:EWTX) Jan. 19 to close 34.5% higher at $13.04 each, their highest valuation in the past 12 months.
