Shares of San Diego-based Capricor Therapeutics Inc. (NASDAQ: CAPR) closed Dec. 3 at $29.96, up $23.60, or 371%, as investors cheered top-line data from the pivotal phase III Hope-3 trial testing cell therapy deramiocel in Duchenne muscular dystrophy.
Five-year follow-up data for Santhera Pharmaceuticals AG’s Duchenne muscular dystrophy (DMD) drug, Agamree (vamorolone), confirm that its efficacy in preserving muscle function is comparable to standard-of-care corticosteroids, but that the overall side-effect profile is more benign. There was less positive DMD news from Sarepta Therapeutics Inc.
The $12.5 billion acquisition of Avidity Biosciences Inc. by Novartis AG strengthens the company’s neuroscience pipeline and marks the second biggest deal that’s been announced this year. It also is the fifth M&A deal in the past five weeks to top the $1 billion mark, a sign that the market may be strengthening.
Tevard Biosciences Inc. has presented new preclinical data on the use of therapeutic suppressor tRNAs (suptRNAs) for the treatment of Duchenne muscular dystrophy (DMD) and dilated cardiomyopathy (DCM). The data show potent restoration of full-length functional proteins in models of DMD and DCM caused by titin truncations (DCM-TTNtv).
The winding regulatory road for the BLA to Capricor Therapeutics Inc.’s cell therapy for Duchenne muscular dystrophy has more clarity. Out of a recent type A meeting between Capricor and the U.S. FDA, prompted by a complete response letter in July regarding lead asset CAP-1002 (deramiocel), the two are in agreement about a path to potential approval.
NS Pharma Inc.’s NS-051 (NCNP-04) has been awarded orphan drug designation by the FDA for the treatment of Duchenne muscular dystrophy (DMD) in patients amenable to exon 51 skipping.
As Avidity Biosciences Inc. brought the second-largest follow-on offering of the year to the market, the company also released positive early and midstage stage results of del-zota, an antibody-oligonucleotide conjugate, in treating Duchenne muscular dystrophy. Phase I/II results showed a reversal of disease progression in patients who have been continuously treated for a year, plus improvements in several functional measures.
Researchers have identified a potential therapeutic target for muscular dystrophies, a group of genetic disorders characterized by progressive muscle weakness and degeneration. The study reveals that inhibiting the microRNA (miRNA) known as miRNA-33 can significantly improve muscle regeneration and ameliorate the dystrophic phenotype in animal models.
Duchenne muscular dystrophy is the most frequent inherited muscle disease and no cure is available. The condition is currently treated with corticosteroids, which can cause substantial side effects.
Sarepta Therapeutics Inc.’s adventure with the Duchenne muscular dystrophy (DMD) AAV-based gene therapy Elevidys (delandistrogene moxeparvovec) continued as the firm said it would restart shipments of the compound for ambulatory patients “imminently,” with the U.S. FDA’s blessing.
