RAPP — what changed in the latest 10-Q

Text added vs the prior filing · source: 10-Q · 2026-05-07

• Several Phase 1 trials in RAP-219 have been conducted in healthy adult volunteers, including a single ascending dose (“SAD”) trial; a multiple ascending dose (“MAD”) trial; a second MAD trial (“MAD-2”), to assess alternative dosing regimens that may accelerate time to reach therapeutic exposure; a p…

• In September 2025, we announced positive topline results from our Phase 2a proof-of-concept trial of RAP-219 in adult patients with drug-resistant FOS. The trial met its primary and secondary endpoints. The trial demonstrated a statistically significant reduction in long episodes (“LEs”) – an object…

• In late 2025, we initiated an open-label long term safety trial to allow patients enrolled in our Phase 2a proof-of-concept FOS trial to resume taking RAP-219. Data from the open-label trial is expected in the second half of 2026. In December 2025, we received U.S. Food and Drug Administration (“FDA…

• We are also expanding our epilepsy portfolio into PGTCS, the most common type of generalized seizure and an important next step in addressing unmet need in patients with seizure disorders. With proof-of-concept established in FOS, we plan to initiate a Phase 3 trial in PGTCS in the first half of 202…

• We believe RAP-219 also has therapeutic potential in bipolar disorder. Our Phase 2 proof-of-concept trial in bipolar mania is progressing well and topline results are now expected in the fourth quarter of 2026, ahead of the previous guidance of first half of 2027. Additionally, we have modified the …

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• A total of four Phase 1 trials in RAP-219 have been conducted to date in healthy adult volunteers: a single ascending dose (“SAD”) trial; a multiple ascending dose (“MAD”) trial; a second MAD trial (“MAD-2”), to assess dosing regimens that may accelerate time to reach therapeutic exposure; and a hum…

• In September 2025, we announced positive topline results from our Phase 2a proof-of-concept trial of RAP-219 in adult patients with drug-resistant focal onset seizures. The trial met its primary and secondary endpoints. The trial demonstrated a statistically significant reduction in long episodes – …

• We believe RAP-219 also has therapeutic potential in bipolar disorder and our Phase 2 proof-of-concept trial in bipolar mania is currently enrolling patients, with topline results expected in the first half of 2027. We also believe RAP-219 has therapeutic potential in peripheral neuropathic pain. We…

• Additionally, we continue to develop a long-acting injectable (“LAI”) formulation of RAP-219. We believe an LAI formulation has the potential to improve patient adherence and expand the potential clinical utility across all of the RAP-219 indications.

• Beyond TARPγ8, we have two advanced discovery-stage nicotinic acetylcholine receptor (“nAChR”) programs stemming from our RAP technology platform. The first comprises modulators of 6 nAChRs that we are developing in chronic pain; and the second comprises modulators of 910 nAChRs that we are devel…

Text added vs the prior filing · source: 10-Q · 2026-05-07

• We previously entered into a common stock sales agreement (the “Sales Agreement”), dated July 1, 2025, by and between the Company and Leerink Partners LLC and Cantor Fitzgerald & Co., acting as sales agents, which established an at-the-market offering program pursuant to which we may offer and sell …

• For example, in the fourth quarter of 2024, we were notified by the FDA that the IND submitted by us for the initiation of a Phase 2 proof-of-concept trial of RAP-219 in diabetic peripheral neuropathic pain (“DPNP”) was placed on clinical hold. The FDA requested additional information and amendments…

• Furthermore, the length of time necessary to complete clinical trials and submit an application for marketing approval for a final decision by a regulatory authority varies significantly from one product candidate to the next and from one country or jurisdiction to the next and may be difficult to p…

• Our lead product candidate, RAP-219, is in clinical development for focal onset seizures (“FOS”), primary generalized tonic-clonic seizures (“PGTCS”) and bipolar mania, and we continue to develop a long-acting injectable (“LAI”) formulation. Additionally, we have two advanced discovery-stage nAChR p…

• Similar laws have been passed and proposed in numerous states, as well as at the federal and local levels, and we expect more states to pass similar laws in the future. Such proposed legislation, if enacted, may add additional complexity, variation in requirements, restrictions and potential legal r…

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• The risk factors denoted with a “*” are newly added or have been materially updated from those included in our Annual Report on Form 10-K for the year ended December 31, 2024 (our “Annual Report”).

• For example, in the fourth quarter of 2024, we were notified by the FDA that the IND submitted by us for the initiation of a Phase 2 proof-of-concept trial of RAP-219 in diabetic peripheral neuropathic pain (“DPNP”) was placed on clinical hold. The FDA requested additional information and amendments…

• We have completed phase 1 clinical development of our lead product candidate, RAP-219 in multiple indications and announced positive topline results from the Phase 2a trial of RAP-219 in patients with drug-resistant focal onset seizures in September 2025. We plan to hold an end-of-Phase 2 meeting wi…

• Similar laws have been passed and proposed in numerous states, as well as at the federal and local levels, and we expect more states to pass similar laws in the future. Such proposed legislation, if enacted, may add additional complexity, variation in

• requirements, restrictions and potential legal risk, require additional investment of resources in compliance programs, impact strategies and the availability of previously useful data and could result in increased compliance costs and/or changes in business practices and policies. The existence of …

Text added vs the prior filing · source: 10-Q · 2026-05-07

• (c) During the quarter ended March 31, 2026, the following directors and officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted certain trading plans intended to satisfy the requirements of Rule 10b5-1(c):

• Trading plan intended to satisfy the affirmative defense conditions of Securities Exchange Act Rule 10b5-1(c)

• Sale of the Company’s common stock pursuant to the terms of the plan

• No other directors or officers (as defined in Rule 16a-1(f) under the Exchange Act) adopted, modified and/or terminated a “Rule 10b5-1 trading arrangement” or a “non-Rule 10b5-1 trading arrangement,” as defined in Regulation S-K Item 408, during the quarter ended March 31, 2026.

Text removed vs the prior filing · source: 10-Q · 2025-11-06

• (c) During the quarter ended September 30, 2025, none of our directors or officers adopted, modified or terminated a Rule 10b5-1 trading arrangement or non-Rule 10b5-1 trading arrangement, as each term is defined in Item 408 of Regulation S-K.