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Shares of Rapport Therapeutics RAPP surged 61.6% in a week after the company reported positive top-line data from a mid-stage study of its lead candidate, RAP-219, in patients with drug-resistant focal onset seizures (FOS), a type of epileptic seizure. RAP-219 is a potential first-in-class, investigational TARPγ8-specific AMPAR negative allosteric modulator.
The phase IIa RAP-219-FOS-201 study of RAP-219 is evaluating the candidate’s efficacy, safety, and tolerability in adults with drug-resistant FOS. The study enrolled thirty FOS patients, who received 0.75 mg of RAP-219 daily for the first five days, followed by 1.25 mg daily for the remainder of the eight-week treatment period. The study also entails another 8-week follow-up period, which is currently ongoing.
Rapport Therapeutics reported that the phase IIa FOS study met its primary endpoint, showing a statistically significant reduction in long episodes (LEs) — an electrographic biomarker tied to seizure activity — compared with baseline, over the eight-week treatment period. RAP-219 also delivered a statistically significant and clinically meaningful reduction in clinical seizures during the study.
Across the eight-week treatment period, 85.2% of patients achieved a ≥30% reduction in LEs, 72% achieved a ≥50% reduction in clinical seizures, and 24% of patients reached seizure freedom. RAP-219 was also found to be well-tolerated in the mid-stage study. Most adverse events related to treatment with the candidate were mild to moderate in severity. This further supports RAP-219’s potential advancement as a new anti-seizure medication.
Year to date, RAPP stock has gained 30.8% compared with the industry’s 5.6% growth.
Per Rapport Therapeutics, as many as 40% of focal epilepsy patients continue to experience seizures despite a range of available therapies, underscoring the need for more effective treatments. Management believes that the magnitude of clinical seizure reduction and the corroborating biomarker data support RAP-219’s potential as a highly effective new therapy for patients with drug-resistant FOS.
Rapport Therapeutics plans to meet with the FDA in late 2025 to discuss the phase IIa FOS study top-line data and is targeting the launch of pivotal studies for RAP-219 in the third quarter of 2026. The company expects to share further efficacy analyses and eight-week follow-up data in 2026.
Rapport Therapeutics is also preparing to launch a long-term safety study of RAP-219 by the end of 2025, to enable FOS patients from the RAP-219-FOS-201 study to continue treatment with the candidate. Preliminary data from this open-label study are expected in the second half of 2026. In parallel, the company is advancing development of a long-acting injectable formulation of RAP-219, aimed at addressing patient nonadherence to oral anti-seizure medications, a key barrier to sustained treatment benefit.
Beyond epilepsy, Rapport Therapeutics is broadening RAP-219’s clinical program into additional indications. A separate phase II study evaluating the candidate in bipolar mania is currently underway, with top-line results anticipated in the first half of 2027. The company also plans to provide an update later in 2025 on the timeline for starting yet another mid-stage study of RAP-219 for diabetic peripheral neuropathic pain.
Rapport Therapeutics, Inc. price-consensus-chart | Rapport Therapeutics, Inc. Quote
Rapport Therapeutics currently carries a Zacks Rank #3 (Hold).
Some better-ranked stocks in the biotech sector are CorMedix CRMD, Pharming Group PHAR and Kiniksa Pharmaceuticals KNSA, each sporting a Zacks Rank #1 (Strong Buy) at present. You can see the complete list of today’s Zacks #1 Rank stocks here.
In the past 60 days, estimates for CorMedix’s earnings per share have increased from $1.10 to $1.52 for 2025. During the same time, earnings per share estimates for 2026 have increased from $1.46 to $2.12. Year to date, shares of CRMD have surged 61.6%.
CorMedix’s earnings beat estimates in each of the trailing four quarters, delivering an average surprise of 34.85%.
In the past 60 days, estimates for Pharming Group’s 2025 loss per share have narrowed from 40 cents to 10 cents. For 2026, PHAR’s earnings per share estimate has improved from 7 cents to 27 cents. PHAR stock has risen 47% year to date.
Pharming Group’s earnings beat estimates in two of the trailing four quarters and missed on the remaining two occasions, delivering an average negative surprise of 39.14%.
In the past 60 days, estimates for Kiniksa Pharmaceuticals’ 2025 earnings per share have increased from 74 cents to $1.03. Earnings per share estimate for 2026 has increased from $1.19 to $1.60 during the same period. KNSA stock has surged 87.1% year to date.
Kiniksa Pharmaceuticals’ earnings beat estimates in two of the trailing four quarters and missed on the remaining two occasions, delivering an average negative surprise of 330.56%.
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This article originally published on Zacks Investment Research (zacks.com).
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