While an “unexpected placebo effect” marred its primary endpoint, the pivotal phase II/III study testing AMO-02 (tideglusib) showed clinically significant benefits across a range of functional and objective assessments, according to developer Amo Pharma Ltd., which is prepping to meet with regulators to discuss potential approval for use in children and adolescents with congenital myotonic dystrophy type 1 (CDM1), an ultra-rare subtype of myotonic dystrophy type 1 for which no treatment options are available.
Abcuro Inc. pulled down an oversubscribed $155 million series B financing co-led by Redmile Group and Bain Capital Life Sciences to advance cytotoxic T and natural killer cells therapies. Specifically, proceeds will back the phase II/III registrational trial of ABC-008, a first-in-class anti-killer cell lectin-like receptor G1 (KLRG1) antibody for inclusion body myositis (IBM) as well as fund continued development of other clinical programs.
There are plenty of companies chasing the first U.S. FDA-approved treatment for the ultra-rare disease fibrodysplasia ossificans progressiva (FOP), but Ipsen SA stayed the course longer and won that approval Aug. 16 with Sohonos (palovarotene). Other companies have struggled to develop a treatment for FOP, which affects about 400 people in the U.S. and about 900 people worldwide. Some potential competitors are still in the clinic, trying to catch up.
The U.S. FDA has given its first approval for treating fibrodysplasia ossificans progressiva (FOP), an ultra-rare disease that creates bone formation outside the skeleton that can lead to immobility, life-threatening respiratory problems and a total locking of the jaw.
Variable muscle expression of frataxin (FXN) protein in muscle plus five adverse events (AEs) of injection-site thrombophlebitis in the multiple ascending-dose (MAD) phase I study hamstrung Design Therapeutics Inc. in its Friedreich’s ataxia (FA) bid with DT-216. Wall Street punished the Carlsbad, Calif.-based firm’s stock (NASDAQ:DSGN) with a knockdown of 70%, or $5.17, leaving shares at $2.17 when the market closed Aug. 15.
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.
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.
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.
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.
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