Gemlapodect

Emalex Biosciences plans to meet with the FDA and other global health authorities to discuss submitting ecopipam for approval later in 2025.\n Emalex Biosciences’ dopamine blocker led to fewer patients with Tourette syndrome relapsing in a phase 3 trial, setting the stage for the Chicago-based firm to submit the drug candidate for approval later this year.The phase 3 study enrolled 167 children and 49 adults, according to a Feb. 25 press release. After a 12-week open-label period, patients who experienced reductions in vocal and motor tics when given the drug were randomized to either continue receiving the drug, called ecopipam, or to switch to placebo for a further 12 weeks. This phase of the trial was double-blind.When continued on ecopipam, 41.9% of children had their symptoms relapse, defined as a 50% loss of the benefit they initially received from the drug in their Yale Global Tic Severity Score. This relapse rate was significantly lower than the 68.1% rate seen in children given placebo, Emalex said in the release. Time to relapse for the pediatric patients was the study’s primary endpoint.Emalex plans to meet with the FDA and other global health authorities to discuss submitting ecopipam for approval later in 2025, the firm said.The most common side effects of ecopipam were drowsiness, insomnia, anxiety, fatigue and headache, according to the release. About 10% of patients experienced drowsiness, also called somnolence, while 7% had insomnia.For the study’s secondary endpoint, Emalex analyzed the time to relapse for children and adult patients together. Here, the relapse rates were similar, with 41.2% of patients relapsing on ecopipam compared to 67.9% on placebo.The phase 3 study builds on Emalex’s prior phase 2 work, which found that ecopipam reduced tic severity by 30% after 12 weeks in 150 children and adolescents with Tourette syndrome.“These results strengthen our confidence in ecopipam as a potential first-in-class treatment for patients with Tourette syndrome,” Frederick Munschauer, M.D., Emalex’s chief medical officer, said in the release. The top-line data “show a statistically significant benefit for ecopipam in maintaining clinically meaningful reductions in vocal and motor tics for pediatric subjects with Tourette syndrome as compared to placebo.” Ecopipam blocks the D1 dopamine receptor, which has been implicated in the repetitive and compulsive behaviors that are characteristic of Tourette syndrome, according to the release. Approved therapies for the disease target the D2 receptor instead.Emalex raised a $250 million series D in 2022 to bring ecopipam into phase 3, a sharp increase in investor interest from the comparatively paltry $35 million series C that preceded it.The Windy City biotech was founded in 2018 by biotech incubator Paragon Biosciences.Swiss biotech Noema Pharma had a Tourette win of its own late last year, when its PDE10A inhibitor gemlapodect—originally developed by Roche—reduced tic severity in a small phase 2 trial that enrolled 15 patients. 

Plus, news about NewAmsterdam, Rocket, ImmunityBio, CG Oncology, Spruce Biosciences, Pharming, Veradermics, Noema Pharma, Ambrosia Biosciences, EsoBiotec, Cellectar Biosciences and Hepion: BenevolentAI’s overhaul: The struggling AI biotech said it plans to lay off staff, consider delisting from the Euronext Amsterdam stock exchange, partner its early-stage assets and undergo other additional organizational shifts as co-founder Ken Mulvany returns . Its Amsterdam-listed stock was down 10% on Wednesday. — Kyle LaHucik Cimeio Therapeutics, Kyowa Kirin make a deal: The Japanese drugmaker is paying up to $300 million as part of an agreement to develop new cell therapies. Additional financial terms were not disclosed. — Jaimy Lee At least three public offerings have been disclosed over the past day, including NewAmsterdam ’s bid to raise $300 million; Rocket ’s upsized $165 million offering ; ImmunityBio ’s $100 million financing ; and CG Oncology ’s offering , which has yet to be priced. — Jaimy Lee Spruce’s Phase 2b fail for endocrine disorder drug: The South San Francisco biotech said tildacerfont administered once daily did not meet the primary endpoint of glucocorticoid reduction in a mid-stage study of people with adult congenital adrenal hyperplasia. But the trial’s principal investigator said the data suggest twice daily dosing may be more effective. Its stock $SPRB was down about 25% on Wednesday morning. — Ayisha Sharma Pharming’s rare disease med passes Phase 3 test in kids: The Dutch drugmaker’s Joenja for activated phosphoinositide 3-kinase delta syndrome (APDS) met the primary endpoints in a late-stage trial of patients aged four to 11 years old. Joenja won FDA approval for APDS patients aged 12 and older in March 2023. Pharming plans to submit global regulatory filings in children starting in 2025. — Ayisha Sharma Veradermics’ $75M Series B: The Connecticut-based dermatology and aesthetics biotech raised the financing as it begins a Phase 2/3 study in hair loss. — Kyle LaHucik Noema adds $35M from EQT Life Sciences: A second close of a Series B first announced in March of last year brings the total for the round to CHF 130 million, which is about $147 million. The cash will fund Noema’s four ongoing Phase 2 trials: mGluR5 negative allosteric modulator basimglurant in trigeminal neuralgia and seizures in tuberous sclerosis complex; gemlapodect, a PDE10a inhibitor, in Tourette syndrome; and NOE-115, a monoamine modulator, in menopause symptoms. — Elizabeth Cairns Merck backs Ambrosia: The New Jersey pharma giant invested in the biotech’s Series A, which now totals $25 million. Ambrosia previously announced plans to take over a former Pfizer site in Boulder as it builds out an oral small-molecule pipeline for obesity and metabolic disorders. — Kyle LaHucik EsoBiotec scores €22M: The Belgian cell therapy specialist raised the equivalent of $23 million from backers that include Thuja Capital, UCB Ventures and Invivo Partners. The company’s immune-shielded lentiviral vector, which is called ESO-T01, is designed to reprogram T lymphocytes into BCMA CAR-T cells in vivo . It will enter an investigator-initiated trial in multiple myeloma in China, its first in humans, and data are expected in the second half of 2025, which could allow expansion to indications including autoimmune diseases. — Elizabeth Cairns Cellectar lays off 60% of its workers: The biotech is prioritizing its pipeline and letting go of staff. Cellectar had 20 employees at the end of 2023. Its stock price $CLRB sank 62% in pre-market trading on Wednesday. — Kyle LaHucik Hepion Pharmaceuticals ends merger plans with Pharma Two B. — Jaimy Lee