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.
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.
Wave Life Sciences Ltd. has reported that GSK plc has selected its first two programs to advance to development candidates following achievement of target validation as part of the companies’ ongoing collaboration.
Wave Life Sciences Ltd. and GSK plc have entered into a strategic collaboration to advance oligonucleotide therapeutics, including Wave's preclinical RNA editing program targeting α1-antitrypsin deficiency (AATD), WVE-006. The discovery collaboration has an initial 4-year research term. The first part of the arrangement is a discovery collaboration that enables GSK to advance up to eight programs and Wave to advance up to three programs, leveraging Wave's PRISM oligonucleotide platform and GSK's expertise in genetics and genomics.
Wave Life Sciences Ltd. is partnering again. The company is getting $170 million up front – $120 million in cash and a $50 million equity investment – in a new partnership with GSK plc to advance oligonucleotides in unnamed targets. The agreement, which has a four-year research term, allows for GSK to advance up to eight programs using Wave Life’s platform and for Wave Life to advance three, or more if GSK approves, of its own collaboration programs.
Wave Life Sciences Ltd. has highlighted progress with WVE-006, its preclinical Aimer (A-to-I RNA base editing) oligonucleotide candidate for the treatment of alpha-1 antitrypsin deficiency (AATD).
In a relatively light news flow for the group last month, the gainers and decliners in the BioWorld Neurological Diseases index canceled themselves out during August, with the result that the price-weighted index closed relatively flat and, despite briefly moving into positive territory in July, its value is down 4% for the year.
Investors continue to keep a close eye on the progress of companies involved in developing medicines targeting the central nervous system and, in the main, their reaction has been generally positive. The BioWorld Neurological Diseases index, a price-weighted index of public biopharmaceutical companies that are focused on developing therapies to treat neurological diseases, closed the year up over 16% and after, a dip in January, is now tracking up more than 12% by market close on Friday Feb. 21, well ahead of the general markets for the same period.
Shares of Cambridge, Mass.-based Wave Life Sciences Ltd. (NASDAQ:WVE) lost 49.5% of their value, or $7.82, to close at $7.99 as investors learned of top-line data from the ongoing phase Ib/IIa Precision HD2 trial testing WVE-120102 in Huntington’s disease.
