NMD Pharma A/S has announced top-line results showing its orally available ion channel inhibitor, NMD-670, has a positive clinical impact in myasthenia gravis (MG), with patients having stronger hand grip and improvements in quantitative MG scores.
Just nine months after a $55 million series B fundraising round, Billiontoone Inc. delivered an oversubscribed series C of $125 million. Demand for the company’s Unity prenatal test, which can assess fetal risk for common recessive conditions and aneuploidies using one maternal blood sample, tripled over the last year.
LONDON – Neuromuscular disease specialist NMD Pharma A/S has raised €35 million (US$39.7 million) in a new financing, as it awaits initial data from its first clinical trial, in the treatment of myasthenia gravis. The new money enables NMD to complete that phase IIa study and to launch another trial of the same compound, NMD-670, in spinal muscular atrophy. The Aarhus, Denmark-based company is preparing the IND and aims to treat the first patient before the end of 2022.
A splicing defect in the survival of motor neuron gene (SMN) leads to a deficiency of protein function that results in spinal muscular atrophy. It is the second most common autosomal recessive disease, occurring with a prevalence of 1 to as little as 6,000 births.
New top-line data from Scholar Rock Holding Group’s phase II Topaz trial of apitegromab (SRK-015) in patients with type 2 and type 3 spinal muscular atrophy generated enough proof-of-concept results for the company to plan on initiating a phase III for later this year. But the 12-month data didn’t stop the Cambridge, Mass.-based company’s stock (NASDAQ:SRRK) from struggling mightily on April 6 as shares closed 20.3% lower at $35.97 each.
LONDON – The National Health Service (NHS) in the U.K. has agreed to reimburse Zolgensma, the one-off gene therapy treatment for spinal muscular atrophy billed as the most expensive drug in the world, after a confidential deal was struck with Novartis Gene Therapies.
Shares of Cambridge, Mass.-based Scholar Rock Holding Corp. closed Oct. 27 at $30.02, up $16.30, or 119%, on positive six-month interim analysis results from the Topaz phase II trial with inhibitor of myostatin activator SRK-015 in type 2 and type 3 spinal muscular atrophy, and CEO Tony Kingsley pointed to “a rich cascade of data ahead of us.”
More than two weeks ahead of its expected PDUFA date, PTC Therapeutics Inc.’s spinal muscular atrophy (SM) drug, risdiplam, gained FDA approval, making it the first at-home, oral treatment intended for use in adults and children 2 months and older.
DUBLIN – Shares in PTC Therapeutics Inc. dropped as much as 13% during premarket trading Feb. 6 on eagerly awaited 12-month data for its oral spinal muscular atrophy (SMA) drug risdiplam, which is licensed to Genentech. Although patients with type 2 or type 3 SMA demonstrated statistically significant improvements in motor function, the level of that improvement evidently disappointed some investors.
Roche Holding AG and PTC Therapeutics Inc. together with their partner, the SMA Foundation, reported success in part two of the pivotal Sunfish study testing their survival motor neuron-2 splicing modifier risdiplam in patients with type 2 or 3 spinal muscular atrophy (SMA).
