Rising from a $51 million series A round a year ago to a $1.1 billion acquisition, Kate Therapeutics Inc. has stepped under the umbrella of Novartis AG, which gains preclinical adeno-associated virus-based gene therapies for neuromuscular diseases.
As an array of developers push their Friedreich’s ataxia (FA) drugs along, PTC Therapeutics Inc.’s recent update on its program piqued thirst for the 15-lipoxygenase inhibitor vatiquinone. Warren, N.J.-based PTC said the prespecified endpoint for two different FA long-term extension studies was met, with highly statistically significant evidence of durable treatment benefit on disease progression.
It’s a go for the two phase III Ensure studies of Immunic Inc.’s lead asset in treating relapsing multiple sclerosis. An unblinded data monitoring committee’s interim futility analysis concluded that the placebo-controlled, pivotal studies using vidofludimus calcium may continue as planned, with the program expected by the company to be completed in 2026.
The Japanese government, industry and academia are deliberating health care policies and initiatives to boost Japan’s role in the future of regenerative medicine, experts at Bio Japan 2024 said, as the fruits of cell and gene therapy research come to fruition with new approvals.
Shares of Scholar Rock Holding Corp. (NASDAQ:SRRK) soared $26.86, or 362%, to close Oct. 7 at $34.28, after the Cambridge, Mass.-based firm disclosed positive top-line data from the phase III Sapphire study testing apitegromab in patients with spinal muscular atrophy (SMA). Apitegromab, which Wainwright analyst Andres Maldonado said will “transform SMA” therapy, met the primary endpoint with statistically significant and clinically meaningful improvement in motor function as measured by the Hammersmith Functional Motor Scale Expanded.
Capricor Therapeutics Inc. just wrapped up a visit with the U.S. FDA and is prepping to file a BLA in October for its Duchenne muscular dystrophy treatment. Linda Marbán, the company’s CEO, is the guest on the newest BioWorld Insider podcast and she talks about deramiocel (CAP-1002), the company’s allogeneic cardiac-derived cell therapy, for treating the rare disease and how the FDA has made strong efforts in helping lay the groundwork for deramiocel.
Interim phase II data of Wave Life Sciences Ltd.’s oligonucleotide, WVE-N531, revealed “impressive” dystrophin expression, solid safety and the potential for once-monthly dosing for boys with Duchenne muscular dystrophy who are amenable to exon 53 skipping. The findings drove Wave Life’s stock (NASDAQ:WVE) up by 53.4%, or $2.85, to close Sept. 24 at $8.19, after peaking earlier in the day at $8.35, its 52-week high.
How do exercise and insulin collaborate in metabolism? The European Association for the Study of Diabetes (EASD) and the Novo Nordisk Foundation recognized the work of Juleen Zierath in this topic with the Diabetes Prize for Excellence at their recent annual meeting.
Capricor Therapeutics Inc. has expanded its commercialization and distribution deal with Nippon Shinyaku Co. Ltd. to include the EU and the U.K. for deramiocel, Capricor’s lead asset, in treating Duchenne muscular dystrophy (DMD). In the new agreement, Capricor will receive an up-front payment of $20 million. Capricor will handle development and manufacturing duties for deramiocel while Nippon Shinyaku will be responsible for the sales and distribution.
Predictably, weekly vs. daily subcutaneous injections made a big difference in the achondroplasia space, where shares of Copenhagen, Denmark-based Ascendis Pharma A/S (NASDAQ:ASND) closed Sept. 16 at $139.57, up $20.35, or 17%, on favorable, pivotal top-line data with Transcon CNP (navepegritide), as competitor Biomarin Pharmaceutical Inc.’s stock (NASDAQ:BMRN) ended the day at $69.86, down $15.04, or 17.7%.
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