Positive data from two studies boosted Edgewise Therapeutics Inc.’s market share and elevated analyst enthusiasm for the company and its treatment of obstructive hypertrophic cardiomyopathy (HCM). Top-line data from the phase I and phase II studies of EDG-7500 in treating HCM, a genetic disease that results in thickened heart muscles, showed the therapy was well-tolerated in healthy volunteers and produced meaningful improvements in those with the disease. The oral, selective, cardiac sarcomere modulator is designed to slow early contraction velocity and address the impaired cardiac relaxation that comes with HCM. The company’s stock (NASDAQ:EWTX) had surged strongly at midday, with shares up 52% at $29.06 each.

Aligos dips on phase II data with MASH treatment

Shares of Aligos Therapeutics Inc. (NASDAQ:ALGS) were trading at $10.70, down $3.46, or 24%, after the South San Francisco-based firm disclosed top-line results from the phase IIa Herald study of ALG-055009, a thyroid hormone receptor beta agonist, in metabolic-dysfunction associated steatohepatitis (MASH) subjects. Doses of 0.5 mg to 0.9 mg turned up statistically significant reductions in liver fat at week 12, with placebo-adjusted median relative reductions up to 46.2% as measured by magnetic resonance imaging proton density fat fraction. Up to 70% of MASH subjects achieved ≥30% relative reduction in liver fat compared to baseline, Aligos said.

Vanda CEO: FDA requests ‘prohibitive’ for gastroparesis drugs

Clearly frustrated by the U.S. FDA’s rejection of tradipitant to treat gastroparesis, Vanda Pharmaceutical Inc. CEO Mihael Polymeropoulos said the agency is discouraging drug development for the serious gastric emptying condition for which there has been no new treatment in 40 years. The complete response letter issued Sept. 18 for the neurokinin-1 receptor antagonist, which Vanda licensed from Eli Lilly and Co. in 2012, requested additional studies that the company said are not in line with the advice of experts in the field. “Gastroparesis is a very difficult disorder to study. It is a waxing and waning disease, which is prone to large placebo effects, and also inaccuracy in measuring effect,” said Polymeropoulos Sept. 19 during the 2024 Cantor Global Healthcare Conference. “So, be that as it may, the reason we’re disappointed is that the designs the FDA seems to ask for [in developing] gastroparesis drugs will be prohibitive for any drug to be developed.”

Cost of using a priority review voucher going up – way up

Using a priority review voucher (PRV) to shave four months off a U.S. FDA drug review will cost 89% more Oct. 1 than it will a day earlier. Although the fiscal 2025 fee of $2,482,446 is significantly higher than the 2024 cost of using a voucher and nearly double what it was just two years ago, it is no record. In fiscal 2018, the FDA fee for using a PRV was $2,830,579. That’s in addition to the PDUFA application fee, which is going up 6% in October. However, as an offset, companies today are paying about one-third of what they once did to buy a transferrable PRV.

ECTRIMS 2024: Time for neuroprotection

The Annual Congress of the European Committee for Treatment and Research in Multiple Sclerosis (ECTRIMS) meeting in Copenhagen this week is celebrating its 40th edition. The hot topic session on neuroprotective therapies set the stage for the subsequent discussions on the latest trends in the management and treatment of multiple sclerosis. Introducing the session, chair Rhonda Voskuhl, from the University of California, highlighted the need to look beyond blood cells and put the focus on CNS cells such as microglia, astrocytes, oligodendrocytes and neurons, that are now recognized as key pieces in the jigsaw puzzle.

Astellas patent survives ‘natural law’ decision for now

The U.S. Court of Appeals for the Federal Circuit reined in a district court that invalidated three claims in an Astellas Pharma Inc. patent protecting bladder drug Myrbetriq (mirabegron). The district court abused its discretion when it determined the claims were not patentable because they recited ‘natural law,’ an argument not raised by the challenger, Sandoz Inc., the Federal Circuit said. The appellate court’s Sept. 18 precedential opinion vacated and returned the case to the lower court to rule on the challenges Sandoz actually raised.

Also in the news

Accord, Altamira, Astrazeneca, Autolus, Awakn, Azitra, Bavarian Nordic, Biomx, Biosyngen, Brenus, Briacell, Bristol Myers Squibb, Cidara, Disc Medicine, Eyconis, Glycomine, GSK, Helsinn, Imcheck, Immix, Innovent, Insilico, Kyverna, Merck, Nanovation, Nexilico, Nicox, Novartis, Novo Nordisk, Pharmajet, Revive, Scancell, Sernova, Siolta, TG, Vaccinex, Valneva