As expected, the FDA granted an approval for Amondys 45 (casimersen), a new Duchenne muscular dystrophy (DMD) therapy developed by Sarepta Therapeutics Inc.
Sarepta Therapeutics Inc.’s miss on a key phase II ambulatory endpoint in its Duchenne muscular dystrophy (DMD) trial may have been caused by a dramatic disparity in functional ability at baseline among older vs. younger patients afflicted with the progressively worsening disorder. In any case, Wall Street had knives out, carving 51% of the value away from shares (NASDAQ:SRPT) of the Cambridge, Mass.-based firm, which closed at $82.29, a loss of $86.66, or 51%.
While everyone will be glad to see 2020 in their rearview mirror, the biopharmaceutical sector can count its blessings, as it emerges from a period where it has surprisingly received record public and private financings and enjoyed strong investor support. This is in sharp contrast to most of the global economy, which has been decimated by the ongoing pandemic.
According to an analysis conducted by BioWorld of the third-quarter 2020 financial reports filed by public biopharmaceutical companies with market caps greater than $1 billion, and excluding big pharma companies, the amount that was invested in research and development (R&D) during the nine-month period increased by almost 21% compared to the same period last year. The spending was bolstered by companies turning their attention to focus their research on therapies and vaccines to tackle COVID-19.
Aavantibio Inc., with the help of a large check from Sarepta Therapeutics Inc., has launched with a $107 million series A to pursue gene therapies for treating rare genetic diseases. The lead program is in Friedreich’s ataxia (FA), a rare genetic disease that typically begins in childhood and causes progressive damage to the nervous system.
One of 19 children dosed with an experimental gene therapy for Sanfilippo syndrome has died following the treatment, given as part of a phase II/III trial of the medicine run by its developer, Paris-based Lysogene SA. The trial has been on clinical hold since June, but the immediate cause of the death is unknown, the company said.
Both Solid Biosciences Inc. and Pfizer Inc. got lifts to their Duchenne muscular dystrophy (DMD) programs from the FDA as the agency released a clinical hold and awarded a fast track designation to their respective adeno-associated viral (AAV) programs.
According to an analysis conducted by BioWorld of the second-quarter 2020 financial reports filed by 120 public biopharmaceutical companies with market caps greater than $1 billion, and excluding big pharma companies, the amount that was invested in research and development during the period increased by 14% compared to the same six-month period last year.
Shares of Sweden-based Hansa Biopharma AB (NASDAQ:HNSA) jumped 32.7% to SEK53.90 (US$5.81) on July 2 as Sarepta Therapeutics Inc. became the first partner to license its lead asset, imlifidase, to enable gene therapies to treat muscular dystrophy patients harboring neutralizing antibodies to the adeno-associated virus (AAV) vectors the medicines employ.
