Ganaxolone

Ovid Therapeutics Inc. (Nasdaq: OVID), a New York-based biopharmaceutical company developing small molecule therapies for brain disorders, announced a USD 60 million private placement to fund the expansion of its lead clinical program, OV329, into additional neurological indications. The PIPE financing, expected to close on or about March 19, 2026, will support development of OV329 in tuberous sclerosis complex and infantile spasms, alongside general research and development activities. The company intends to combine the net proceeds with existing cash, cash equivalents, and marketable securities. Under the terms of the securities purchase agreement, Ovid is selling 19,154,321 shares of common stock at USD 2.01 per share and pre-funded warrants to purchase up to 10,701,710 additional shares at a purchase price of USD 2.009 per pre-funded warrant. The pre-funded warrants carry an exercise price of USD 0.001 per share and are immediately exercisable. Point72 led the financing, with participation from existing investors including Adage Capital Management, ADAR1 Capital Management, Affinity Asset Advisors, Ally Bridge Group, Balyasny Asset Management, Coastlands Capital, Eventide Asset Management, Janus Henderson Investors, and RA Capital Management. Leerink Partners served as lead placement agent, with Oppenheimer & Co. and LifeSci Capital acting as co-placement agents. Company overview and pipeline Ovid Therapeutics is focused on developing targeted small molecule candidates that modulate factors involved in neuronal hyperexcitability across multiple neurological and neuropsychiatric disorders. The company’s lead asset, OV329, is a next-generation GABA-aminotransferase inhibitor being developed as a potential therapy for treatment-resistant focal onset seizures and developmental and epileptic encephalopathies, including tuberous sclerosis complex and infantile spasms. The press release did not specify the current clinical phase of OV329 or provide a timeline for upcoming milestones in these new indications. Ovid’s broader pipeline includes OV4071 and a library of related compounds designed to directly activate the KCC2 transporter, targeting multiple central nervous system disorders. The company has previously advanced OV101 (gaboxadol) through Phase III testing in Angelman syndrome, with a completed randomized, double-blind study enrolling 104 patients and an open-label extension study (ELARA) enrolling 170 patients. OV101 was also evaluated in a completed Phase II trial in Fragile X syndrome. In July 2020, Ovid licensed exclusive European rights to OV101 for Angelman syndrome to Angelini Pharma. On the business development side, Ovid has undergone a period of portfolio restructuring. In June 2024, Takeda secured global development and commercialization rights to soticlestat (TAK-935), a cholesterol 24-hydroxylase inhibitor previously co-developed with Ovid for Dravet syndrome and Lennox-Gastaut syndrome. In June 2025, Ovid sold 100% of its ganaxolone royalty rights to Immedica Pharma AB for USD 7 million in cash. The company also holds a collaboration with Graviton Bioscience, established in May 2023, focused on developing brain-penetrant ROCK2 inhibitors for rare neurological diseases marked by seizures. Leave a Reply Cancel reply Your email address will not be published. Required fields are marked * Comment * Name * Email * Website

The FDA has signed off on Immedica Pharma's ultrarare disease treatment Loargys four years after it refused to review a marketing application for the recombinant human enzyme from another drugmaker. Four years after the FDA issued its most heavy-handed form of a rejection to the prior company behind pegzilarginase, the U.S. regulator has now given the treatment a thumbs-up.Scoring the accelerated nod is Immedica Pharma for Loargys as a therapy for hyperargininemia in the ultrarare genetic disorder Arginase 1 deficiency (ARG1-D). The approval covers patients age 2 and older, with the therapy to be used in conjunction with a protein-restricted diet. Loargys, which is also known as pegzilarginase, is a recombinant human enzyme designed to lower levels of arginine in patients who are unable to break down the amino acid. It is the first treatment to address the elevated levels of plasma arginine associated with the disorder. The approval also comes after the FDA issued a complete response letter (CRL) to the company in August of last year citing major deficiencies across efficacy, dosing/administration, safety and labeling.ARG1-D affects an estimated 250 people in the U.S., with the current standard of care relying on symptom management including diet restrictions, arginine-free amino acid supplementation and nitrogen scavenging agents. Patients are often diagnosed in late infancy or early childhood, and the symptoms include spasticity, seizures and intellectual disability. “The accelerated approval of Loargys offers a fundamentally new approach that addresses the enzyme deficiency itself,” Stephen Cederbaum, M.D., a professor of human and medical genetics at the University of California, Los Angeles, said in a release. “Since persistently elevated arginine and its metabolites have been reported to be the proximal or direct driver of disease progression, this is a major advancement in metabolic medicine.” The company expects to make Loargys—which is infused or injected weekly—available in the U.S. in April. The green light comes after the FDA dismissed a marketing application for pegzilarginase in 2022 from Texas-based Aeglea BioTherapeutics, handing down a refusal-to-file (RTF) letter. An RTF is issued when the agency determines an application cannot be reviewed due to deficiencies that cannot be quickly resolved.A year after the rejection, Aeglea sold pegzilarginase to its European partner Immedica for $15 million upfront and $100 million in potential milestones.While a clinical trial of pegzilarginase linked it to an 80% reduction in mean plasma arginine compared to placebo, there were questions about whether it improved the health of patients. While mobility scores for patients improved in the treatment arm and worsened in the placebo group, the difference fell short of statistical significance. To ultimately gain FDA approval of Loargys, Immedica relied on results from the same trial, while also submitting additional "long-term data," it said in an email. "The application was resubmitted with additional data, analyses and clarifications addressing each of the FDA’s objections in the refusal to file and advice in regulatory interactions, which ultimately supported the agency’s decision to accept the filing for review," the company added.Additionally, in the August CRL, the FDA suggested the possibility of an accelerated-approval pathway "only with a postmarketing confirmatory trial designed to validate surrogate endpoints and demonstrate clinical benefit."In December 2023, Sweden-based Immedica earned marketing approval of Loargys from the European Medicines Agency. The private company, which was established in 2018 by Nordic investment firm Impilo, does not report sales of its products.At the end of 2024, Immedica acquired troubled Marinus Pharmaceuticals and its seizure medicine Ztalmy, which was approved in 2022.EDITOR'S NOTE: Immedica's responses to questions about how it ultimately gained FDA approval of Loargys were added to the story after it was initially published.