NDA for rusfertide submitted to the US Food and Drug Administration (FDA), with potential approval and launch this year Company expects to opt-out of the 50:50 profit and loss sharing arrangement for rusfertide with Takeda during a 90-day window expected to open in Q2 U.S. regulatory decision for ICOTYDETM (icotrokinra) anticipated in 2026 with potential launch this year PN-881 Phase 1 completion expected by mid- 2026 NEWARK, CALIFORNIA / ACCESS Newswire / February 25, 2026 / Protagonist Therapeutics (Nasda ...

Core Viewpoint - Nicox SA has received positive feedback from the U.S. FDA regarding the pre-NDA meeting for NCX 470, indicating that the data package and proposed NDA content are generally acceptable for submission, with the NDA submission expected in summer 2026 [1][2]. Group 1: FDA Meeting and NDA Submission - The FDA meeting was productive and collaborative, supporting the finalization of the registration dossier, which includes positive results from two Phase 3 studies [2]. - The FDA has requested additional pharmacokinetic data from an ongoing study in Japan, which will not affect the NDA submission timeline [1]. - The NDA submission is on track for summer 2026, with Nicox set to receive a milestone payment from Kowa upon submission [5][6]. Group 2: Product Information - NCX 470 is a novel nitric oxide-donating bimatoprost eye drop designed to lower intraocular pressure in patients with open-angle glaucoma or ocular hypertension [1][7]. - The product is licensed globally to Kowa, except in China, South Korea, and Southeast Asia, where it is licensed to Ocumension Therapeutics [3]. Group 3: Future Milestones - The NDA submission in the U.S. is expected in summer 2026, with a subsequent submission in China anticipated shortly after [6]. - The Phase 3 clinical program for NCX 470 in Japan was initiated in summer 2025 [6].

Core Viewpoint - Grace Therapeutics is advancing GTx-104, a novel injectable formulation of nimodipine for the treatment of aneurysmal subarachnoid hemorrhage (aSAH), with a PDUFA target date set for April 23, 2026, for FDA review of its NDA submission [1][2]. Company Developments - The company is engaged in pre-commercial planning in anticipation of potential FDA approval for GTx-104, supported by positive results from the STRIVE-ON trial, which demonstrated improved clinical outcomes for aSAH patients [2][4]. - The STRIVE-ON trial results were presented at the Society of Vascular and Interventional Neurology annual meeting in November 2025, highlighting the safety and tolerability of GTx-104 compared to oral nimodipine [5][11]. - The company secured approximately $4.0 million in additional funding through the exercise of common warrants, enhancing its financial position for ongoing operations [5]. Financial Performance - For the quarter ended December 31, 2025, the company reported a net loss of approximately $2.3 million, a decrease from a net loss of $4.2 million in the same quarter of the previous year, primarily due to reduced research and development expenses [4][22]. - Research and development expenses for the same period were $0.5 million, significantly lower than $2.2 million in the prior year, reflecting the completion of the STRIVE-ON trial [6]. - General and administrative expenses increased to $2.0 million, up from $1.5 million, attributed to costs associated with GTx-104 pre-commercial planning [7]. Cash Position - As of December 31, 2025, the company had cash and cash equivalents of $18.7 million, a decrease from $22.1 million at the end of March 2025, indicating a need for careful cash management moving forward [8][10]. Market Context - aSAH is a rare and serious condition that has not seen significant innovation in treatment for nearly 40 years, positioning GTx-104 as a potentially transformative therapy if approved [2][12]. - The company holds a strong patent estate that could enhance the long-term market value of GTx-104, benefiting shareholders [2].

Financial Data and Key Metrics Changes - In Q4 2025, research and development expenses decreased to $6.2 million from $7 million in Q4 2024, reflecting the completion of the SHIELD II Phase 3 trial [15] - General and administrative expenses increased to $1.8 million in Q4 2025 from $1 million in Q4 2024, primarily due to non-cash expenses related to performance-based options [16] - The net loss for Q4 2025 was $8.5 million or $0.41 per share, unchanged from the previous year but improved on a per-share basis from $1.13 in Q4 2024 [16] - For the full year 2025, net loss was $34.2 million or $2.09 per share, compared to a net loss of $29 million or $4.91 per share in 2024 [17] - As of December 31, 2025, the company had $12.9 million in cash and equivalents, with additional gross proceeds of $3.7 million generated from warrant exercises post-quarter [18] Business Line Data and Key Metrics Changes - The company successfully completed the SHIELD II Phase 3 trial for D-PLEX100, achieving its primary and key secondary endpoints [5] - The focus remains on advancing the regulatory pathway for D-PLEX100 and commercial partnership discussions in the U.S. [5][7] Market Data and Key Metrics Changes - The company is engaging with potential U.S. commercial partners that have strong hospital-based commercialization experience [11] - Discussions with partners have progressed to advanced stages, reflecting the strong clinical profile of D-PLEX100 [7] Company Strategy and Development Direction - The company is transitioning from a primarily R&D-focused organization to one preparing for commercialization, with a renewed corporate brand to reflect this change [11][12] - The introduction of Kynatrix technology aims to expand capabilities beyond localized delivery, targeting metabolic diseases with a long-acting GLP-1 receptor agonist program [14] Management's Comments on Operating Environment and Future Outlook - Management believes 2026 could be transformative, with the rolling NDA submission expected to begin by the end of Q1 2026 [9] - Positive feedback from the FDA supports the regulatory pathway for D-PLEX100, with expectations for a rolling NDA submission [6][9] Other Important Information - The company appointed Ms. Brooke Story as chairman of the board in December 2025, bringing extensive experience in medical technology [9] Q&A Session Summary Question: Can you share how discussions around the scope of the label progressed? - The company is targeting an initial label for the prevention of surgical site infections in abdominal colorectal surgery, supported by Phase 3 data [22] Question: Any thoughts on plans for broader label expansion? - There may be opportunities for label expansion into broader abdominal surgical applications as the review process progresses [24] Question: What work is being done to prepare for potential approval of D-PLEX100? - The company is focusing on building awareness, market research, and creating a KOL network to prepare for commercialization [32] Question: How do you view the strategic timing for the GLP-1 program? - The company plans to partner at an early stage, leveraging robust preclinical data to attract interest [35] Question: What factors will play a role in identifying the final partner? - The ideal partner will have broad hospital-based capabilities and presence in the surgical suite [41] Question: What percentage of potential target IDNs would likely include D-PLEX on formulary within the first 12 months after approval? - The uptake will take time, with a few months before seeing meaningful updates, but once on formulary, usage is expected to grow steadily [50]

Core Insights - Aquestive Therapeutics, Inc. experienced a significant stock decline of approximately 40% intraday following the disclosure of deficiencies identified by the U.S. Food and Drug Administration (FDA) in its New Drug Application (NDA) for Anaphylm, an experimental treatment for severe allergic reactions [5] Group 1 - The FDA's identification of deficiencies prevents discussions regarding labeling and post-marketing requirements, raising concerns about the approvability of the NDA ahead of the January 31, 2026, PDUFA action date [5] - Faruqi & Faruqi, LLP is investigating potential claims against Aquestive Therapeutics for investors who suffered significant losses [2][4]

Core Viewpoint - Annovis Bio, Inc. is set to launch an Open-Label Extension (OLE) study in January 2026 to assess the long-term safety and efficacy of its drug, buntanetap, in patients with Parkinson's disease [2][3] Study Overview - The OLE study aims to enroll 500 patients over a 36-month treatment period, with participants receiving a once-daily dose of 30mg oral buntanetap [9] - The study will include two cohorts: former participants of previous clinical trials and patients who have been receiving deep brain stimulation (DBS) for at least 12 months [9][10] Objectives and Methodology - The study will evaluate treatment persistence by examining how patient outcomes evolve after discontinuing treatment, providing insights into the long-term effects of buntanetap [5][6] - It will also assess patient responses when treatment is reintroduced, offering a comprehensive view of the drug's effects and durability [6] Addressing Underserved Populations - The inclusion of patients receiving DBS aims to address a gap in clinical research, as this population is often excluded due to complications in outcome assessments [8][10] - The study seeks to evaluate the interaction between buntanetap and DBS, potentially providing additional therapeutic benefits [7][10] Regulatory Advancement - The OLE study is a critical step toward a future New Drug Application (NDA) submission, helping the company meet FDA patient exposure requirements [11] - The study aims to ensure a total of approximately 1,500 treated patients, with specific targets for treatment duration and dosage [11]

Core Insights - Unicycive Therapeutics is advancing its lead investigational treatment, oxylanthanum carbonate (OLC), for patients with hyperphosphatemia and plans to resubmit the New Drug Application (NDA) by the end of 2025 following positive discussions with the FDA [2][8] - The company reported a net loss of $6.0 million for the quarter ended September 30, 2025, an increase from a net loss of $4.1 million in the same period of 2024, primarily due to increased labor and professional services costs [6][9] - Unicycive ended Q3 2025 with $42.7 million in cash, providing a runway into 2027, which positions the company well for regulatory approval and potential product launch [7][9] Financial Results - Research and Development (R&D) expenses for Q3 2025 were $3.0 million, a slight decrease from approximately $3.1 million in Q3 2024, attributed to reduced professional services and drug development costs [4] - General and Administrative (G&A) expenses increased to $4.4 million in Q3 2025 from $3.2 million in Q3 2024, mainly due to higher labor and consulting costs [5] - Other income decreased to $1.3 million in Q3 2025 from $2.2 million in Q3 2024, primarily due to changes in the fair value of warrant liability [6] Upcoming Milestones - The company is on track to resubmit the NDA for OLC by year-end 2025, with a potential new Prescription Drug User Fee Act (PDUFA) date in the first half of 2026 [8] - New data presented at the ASN Kidney Week 2025 indicates that OLC significantly reduces pill burden, with a 7-fold decrease in pill volume and a 2-fold reduction in pill count compared to existing phosphate binders [2][8]

Core Insights - Reviva Pharmaceuticals is advancing its brilaroxazine program towards potential registration, with a planned meeting with the FDA in Q4 2025 to discuss the New Drug Application (NDA) submission targeted for Q2 2026 [2][5] Clinical Program and Business Highlights - The company successfully completed a 1-year open-label extension (OLE) trial for brilaroxazine, demonstrating a well-tolerated safety profile and robust broad-spectrum efficacy across all symptom domains in schizophrenia [1][5] - Brilaroxazine showed a significant reduction in PANSS total score by 18.1, positive symptoms by 5.0, and negative symptoms by 4.4, with a discontinuation rate of 35% after 1 year [5] - Reviva is expanding its patent portfolio, aiming for potential patent and market exclusivity up to 2045 and beyond [5] - A late-breaking poster presentation on the RECOVER 12-month OLE trial was presented at the 2025 ASCP annual meeting [5] Financial Results - For the second quarter ended June 30, 2025, the company reported a net loss of approximately $6.1 million, or $0.12 per share, compared to a net loss of approximately $7.9 million, or $0.26 per share, for the same period in 2024 [10][13] - As of June 30, 2025, cash and cash equivalents totaled approximately $10.4 million, down from approximately $13.5 million as of December 31, 2024 [10][11] Anticipated Milestones and Events - The company plans to initiate a potential registrational Phase 3 RECOVER-2 trial for brilaroxazine pending FDA recommendations [5] - An IND submission for a liposomal-gel formulation of brilaroxazine in psoriasis is expected by Q2 2026 [10]

Core Insights - Nicox SA has announced the completion of the last patient visit in the Denali Phase 3 clinical trial for NCX 470, a treatment for open-angle glaucoma and ocular hypertension [1][3] - The trial enrolled a total of 696 patients, with topline results expected between mid-August and mid-September 2025 [2][6] - NCX 470 is a novel NO-donating bimatoprost eye drop aimed at lowering intraocular pressure, and the trial compares its efficacy to latanoprost [3][6] Company Overview - Nicox SA is an international ophthalmology company focused on developing innovative solutions for ocular health [5][7] - The company is headquartered in Sophia Antipolis, France, and is listed on Euronext Growth Paris [8] Future Milestones - Topline results from the Denali trial are anticipated in mid-August to mid-September 2025 [6] - A New Drug Application (NDA) submission for NCX 470 in the U.S. is targeted for the first half of 2026, contingent on securing a U.S. partner or necessary funding [5][6] - Initiation of NCX 470 Phase 3 clinical trials in Japan is expected in the second half of 2025 [6]

Core Viewpoint - The FDA has identified deficiencies in cGMP compliance at a third-party manufacturing vendor for Unicycive Therapeutics' New Drug Application (NDA) for oxylanthanum carbonate (OLC), with a final decision expected by June 28, 2025 [1][2]. Company Overview - Unicycive Therapeutics is a clinical-stage biotechnology company focused on developing therapies for kidney disease, with its lead investigational treatment being oxylanthanum carbonate (OLC) [6]. - OLC is an oral phosphate binder designed to treat hyperphosphatemia in patients with chronic kidney disease (CKD) on dialysis, utilizing proprietary nanoparticle technology to enhance phosphate binding potency [3][4]. Product Details - OLC aims to reduce the pill burden for patients, potentially improving adherence compared to existing treatments [3]. - The NDA submission for OLC is based on data from three clinical studies and multiple preclinical studies, with a strong global patent portfolio protecting the product until at least 2031, with potential extensions until 2035 [4]. Market Context - Hyperphosphatemia is a critical condition affecting nearly all patients with End Stage Renal Disease (ESRD), with over 450,000 individuals in the U.S. requiring medication annually to manage phosphate levels [5]. - Effective treatment of hyperphosphatemia is essential to reduce associated risks of increased mortality and hospitalization in CKD patients on dialysis [5].