The BioWorld Drug Developers Index underperformed both the Nasdaq Biotechnology Index (NBI) and the Dow Jones Industrial Average (DJIA) throughout February and March. However, it showed some recovery by the end of April, finishing the month down 4.67%, slightly worse than the DJIA’s 4.41% decline. The NBI ended April slightly up from both, with a year-to-date drop of 1.16%.
Positive early stage data for Verve Therapeutics Inc.’s base editing therapy points to a range of development options, including bringing partner Eli Lilly and Co. in a little closer. The new data helped ease the company’s pain from the April 2 enrollment pause of a similarly designed therapy from Verve. Verve’s Heart-2 phase Ib of VERVE-102 in treating 14 patients with heterozygous familial hypercholesterolemia and/or premature coronary artery disease showed one infusion led to dose-dependent decreases in blood PCSK9 protein levels and low density lipoprotein cholesterol.
Alongside the release of abstracts related to the American Society of Hematology meeting next month in San Diego, and as part of the firm’s third-quarter update, Beam Therapeutics Inc. disclosed that one patient died in the phase I/II trial testing BEAM-101 in sickle cell disease (SCD).
Trouble continues to dog Verve Therapeutics Inc.’s base editor of the PCSK9 gene, VERVE-101, so the company paused enrollment in a phase Ib study in cholesterol lowering to focus on the similarly designed VERVE-102.
Beam Therapeutics Inc. has received clearance of its clinical trial authorization (CTA) application by the U.K.’s Medicines and Healthcare Products Regulatory Agency for BEAM-302, an in vivo base editor, as a potential treatment for patients with α1-antitrypsin deficiency (AATD).
Though down nearly 6% in January, the BioWorld Drug Developers Index (BDDI) rebounded in February, finishing the month with a 4.41% increase and outperforming both the Nasdaq Biotechnology Index (NBI; up 1.33%) and the Dow Jones Industrial Average (DJIA; up 3.47%). In 2023, BDDI concluded the year down 11.35%, trailing behind the NBI and DJIA.
Inhibrx Inc. and Sanofi SA have agreed to a deal worth up to $2.2 billion, whereby the latter’s Aventis Inc. subsidiary will acquire INBRX-101, an optimized, recombinant alpha-1 antitrypsin (AAT) augmentation therapy undergoing a registrational trial for AAT deficiency (AATD), an inherited genetic disorder caused by single nucleotide variants in the SERPINA1 gene.
Beam Therapeutics Inc. has offered a progress update on its genetic disease franchise. BEAM-302, the company’s priority genetic disease program, is a potential treatment for α1-antitrypsin deficiency (AATD).
Beam Therapeutics Inc. has released new preclinical data demonstrating the ability of its in vivo drug candidate, BEAM-302, to directly correct the PiZ mutation, the primary disease-causing mutation associated with severe α1-antitrypsin deficiency (AATD).
