The U.S. FDA has approved Daxxify (daxibotulinumtoxinA-lanm) for treating cervical dystonia in adults nearly a week ahead of its Aug. 19 PDUFA date. Approval of the sBLA for the injectable, peptide-formulated neuromodulator went to Revance Therapeutics Inc. The drug’s original approval was for treating glabellar lines, better known as frown lines, came in September 2022. Daxxify is the first neuromodulator stabilized with Revance’s peptide exchange technology. It is created without human serum albumin or animal-based components.
Modalis Therapeutics Corp. – previously Edigene Corp. – has regained full rights to two gene therapy candidates for muscle disorders, MDL-201 and MDL-202, that were co-developed with Astellas Pharmaceutical Inc. since 2019.
Diabetes has been associated with increased bone fracture risks, however, the cellular and molecular mechanisms underlying diabetic bone fragility are not clear. In the current study, researchers from the Children's Hospital of Philadelphia presented data from a study that aimed to investigate these mechanisms in murine monogenic model of type 1 diabetes (T1D).
South Korean biopharma Connext Co. Ltd. is set on outdoing standard-of-care therapies for Dupuytren’s contracture. The Daegu-headquartered Connext recently secured U.S. FDA IND approval for a phase I/II trial on its recombinant collagenase clostridium histolyticum, called CNT-201, inching closer to its goal of providing an affordable but high-quality therapeutic option for patients with the rare, progressive connective tissue disorder.
Innovo Therapeutics Inc. has prepared and tested new isoindolinone derivative compounds characterized as caspase-1 (IL-1β-converting enzyme), interleukin-1β (IL1B; IL-1β) and apoptosis inhibitors reported to be useful for the treatment of osteoarthritis and pain.
More than two months after Blueprint Medicines Corp. gained a broadened label for Ayvakit (avapritinib), the company is “really pleased to see the diversity of revenue this early in the launch across specialties and across both the academic and community settings,” said Philina Lee, chief commercial officer. The situation “bodes very well for a continued cadence of prescribing [into] the foreseeable future,” she said.
Non-profit Solve GNE LLC has raised over $2.5 million and announced sponsored research agreements to help advance research in hereditary inclusion body myopathy (HIBM), or GNE myopathy (GNEM).
Positive top-line phase II results caused Mymd Pharmaceuticals Inc.’s stock (NASDQ:MYMD) to surge 31.8% on July 31 to close at $1.45 per share. The data behind the push were for MYMD-1 (isomyosmine) for treating chronic inflammation associated with sarcopenia.
Sarcopenia is the loss of muscle mass typically in the elderly that ultimately reduces mobility, diminishes quality of life, and can cause potential fall-related injuries that can become serious. While exercise is an appropriate approach, unfortunately patients with underlying health conditions and low motivation are unlikely to adhere to regimented muscle training. Moreover, there are no available effective cures for addressing age-related muscle loss. Still, it is possible that a failure to repair muscle after injury may be a major contributor to muscle mass loss in the aged that can be addressed by using muscle stem cells.
Epibone Inc. has received IND clearance from the FDA to begin testing its engineered allogenic osteochondral graft in humans. Clinical trials are set to open patient recruitment as soon as early next year.
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