Pasithea Therapeutics Announces Grant of Rare Pediatric Disease Designation (RPDD) by FDA to PAS-004 for Treatment of Neurofibromatosis Type 1 (NF1)

On April 20, 2026 Pasithea Therapeutics Corp. (NASDAQ: KTTA) ("Pasithea" or the "Company"), a clinical-stage biotechnology company developing PAS-004, a next-generation macrocyclic MEK inhibitor, reported that the U.S. Food and Drug Administration (FDA) has granted Rare Pediatric Disease Designation to PAS-004 for treatment of Neurofibromatosis type-1 (NF1).

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The FDA grants RPDD for serious or life-threatening diseases in which the serious or life-threatening manifestations primarily affect individuals aged from birth to 18 years and affect fewer than 200,000 people in the U.S. There are approximately 115,000 individuals in the U.S living with NF1.

Under the FDA’s Rare Pediatric Disease Priority Review Voucher program, a sponsor who receives an approval of a new drug application (NDA) or biologics license application (BLA) for a rare pediatric disease may be eligible for a Priority Review Voucher ("PRV") which can be redeemed to obtain priority review for a subsequent marketing application for a different product. The PRV may be sold or transferred to another sponsor. In the last 12 months, disclosed PRV sales have ranged from $150–$205 million.

"We are pleased to have received rare pediatric disease designation from the U.S. FDA for our PAS-004 program for patients with NF1," said Dr. Tiago Reis Marques, chief executive officer of Pasithea. "This designation for PAS-004 reinforces the potential of PAS-004 to address this serious condition."

PAS-004 has so far been granted the following FDA regulatory designations: Orphan Drug Designation, Fast Track Designation and Rare Pediatric Disease Designation.

The Company is currently conducting a Phase 1/1b multicenter, open-label, dose escalation trial of PAS-004 in adult participants with symptomatic, inoperable, incompletely resected, or recurrent NF1-PN (NCT06961565).

About NF1- PN
Plexiform neurofibromas (PN) are tumors originating from the nerve sheath that grow through and around nerves and may involve multiple nerve branches. Thirty to fifty percent (30-50%) of patients with NF1 will harbor PNs, which can undergo malignant transformation. PN-related morbidities are primarily caused by the direct impact of the tumor on surrounding structures and can be life-threatening when they compress vital organs or when they become malignant.

(Press release, Pasithea Therapeutics, APR 20, 2026, View Source [SID1234664548])

OXC-101 as a novel therapy in canine blood cancer will be presented at AACR

On April 20, 2026 Oxcia in a collaboration with SLU and Karolinska Institutet, reported that mitotic MTH1 inhibitor OXC-101 is investigated in a pilot clinical study in canine lymphoma and hemangiosarcoma. The early clinical results are presented by Dr. Kumar Sanjiv at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) annual meeting 19-22 April, 2026. The title of the presentation is: "OXC-2101 (karonudib) in canine lymphoma and hemangiosarcoma: Safety, early efficacy and translational potential" (Poster Number 7913) and will be presented Wed 22 April 2026.

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(Press release, Oxcia, APR 20, 2026, View Source;utm_medium=rss&utm_campaign=oxc-101-as-a-novel-therapy-in-canine-blood-cancer-will-be-presented-at-aacr [SID1234664547])

Nykode Therapeutics Presents Additional Immunogenicity Data from VB-C-03 Trial at the 2026 American Association for Cancer Research (AACR) Annual Meeting

On April 20, 2026 Nykode Therapeutics ASA (OSE: NYKD), a clinical-stage biopharmaceutical company dedicated to the discovery and development of novel immunotherapies, reported the poster presentation of additional immunogenicity data from the VB-C-03 clinical trial at the 2026 American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting.

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The data build on results initially presented at the 10th International Congress on Innovative Approaches in Head & Neck Oncology (ICHNO) in March 2026, which demonstrated a confirmed objective response rate of 38.5% and strong immunogenicity signals. The current results support an even more robust immunogenicity, with now 11 evaluable patients, where all 10 patients (100%) in the 6 and 9 mg dose groups showed HPV16-specific vaccine-induced immune responses. The responses were both rapid and durable, with a demonstrated persistence into the last analyzed timepoint at the completed end-of-treatment.

"The consistency and strength of the T-cell responses we are observing across patients underscore that abi-suva has the makings of a best-in-class vaccine for HPV16-positive cancers. For patients with recurrent or metastatic head and neck cancer, where current treatments offer a response rate of only around 19%, a therapy that can generate this quality of immune response alongside meaningful clinical activity could represent a real step forward. We enter the Abili-T trial with a well-defined dose, a strong immune response profile, and a competitive data package that sets abi-suva apart from other approaches in this indication and we are deeply motivated by the opportunity to make a difference for these patients," said Agnete Fredriksen, CSO and Co-founder of Nykode Therapeutics.

The poster will be available after the session at the Company’s Webpage: View Source

(Press release, Nykode Therapeutics, APR 20, 2026, View Source [SID1234664545])

Nanobiotix Announces New Preclinical Data Supporting Improved Systemic Bioavailability and Reduced Toxicity for LNP-Delivered DNA Immunotherapy After Pre-Treatment With Nanoprimer Technology

On April 20, 2026 NANOBIOTIX (Euronext: NANO – NASDAQ: NBTX – the "Company"), a late-clinical stage biotechnology company pioneering nanotherapeutic approaches to expand treatment possibilities for patients with cancer and other major diseases, reported the presentation of new preclinical data evaluating its Nanoprimer platform in sequence with lipid nanoparticle-delivered recombinant DNA ("LNP-DNA") at the 2026 Annual Meeting of the American Association for Cancer Research (AACR) (Free AACR Whitepaper).

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POSTER #6389: Maximizing systemic LNP-DNA delivery for cancer-activated expression of Immunotherapy agents using Nanoprimer technology

Background

A key limitation common to LNP-DNA is rapid hepatic clearance via the mononuclear phagocyte system ("MPS"), which can reduce systemic bioavailability and tumor accumulation, as well as increase hepatic toxicity. In addition, LNP-DNA may trigger transient acute inflammation through activation of the cGAS/STING pathway.

In this evaluation, LNP-DNA vehicles designed for anti-tumor immunotherapy were administered with or without Nanobiotix Nanoprimer pre-treatment in a mouse model. Nanoprimer is designed to transiently occupy hepatic clearance pathways such as MPS to increase bioavailability and target accumulation, while reducing hepatic toxicity. Both agents were administered intravenously (IV).

Key Observations

Reduced hepatic exposure and toxicity:
Decreased liver uptake of LNP-DNA and improved hepatic tolerability
Improved systemic bioavailability:
Increase in circulating levels of LNP-DNA
Mitigation of inflammatory response:
Attenuated activation of cGAS-STING pathway downstream targets
Potentially broad applicability across LNP designs:
Notably, these LNP-DNA formulations were specifically engineered for extrahepatic delivery, supporting the potential of the Nanoprimer to further optimize advanced delivery systems

Conclusions

These data support further evaluation of Nanoprimer in sequence with innovative LNP-delivered therapies
Advanced LNP systems designed for extrahepatic delivery could potentially be further optimized to improve systemic bioavailability and reduce toxicity through sequencing with the Nanoprimer

"We continue our dual path approach to the development of our next-wave Nanoprimer platform in which we are both pursuing external collaborations with partners developing innovative therapeutic candidates that are challenged by liver accumulation, as well as our own proprietary internal pipeline," said Laurent Levy Nanobiotix Chief Executive Officer and Chairman of the Executive Board. "We are encouraged by these preclinical results, generated in collaboration with Earli, which further support our hypothesis that the Nanoprimer may improve therapeutic efficacy while mitigating toxicity when sequenced prior to the administration of advanced therapeutics such as LNP-DNA and an additional layer of proof of concept."

About NANOPRIMER

The Nanoprimer is an early-stage nanotherapeutic platform designed to disrupt the design and development of innovative therapeutics and improve outcomes for patients. The Nanoprimer potentially increases drug bioavailability or decreases unintended off-target effects in the liver, specifically hepatic toxicity. The platform is designed for use in combination with advanced therapeutics across multiple drug classes. The Nanoprimer is being developed through external collaborations and an internal proprietary pipeline.

Nanoprimer is an early-stage nanotherapeutic platform designed to unleash the potential of advanced therapeutics by addressing one of the most common structural limitations in modern medicine: liver uptake and extrahepatic delivery. As therapies become more complex—such as RNA, gene therapies, and advanced biologics—they are increasingly captured by the liver, limiting their ability to reach target tissues and reducing their effectiveness.

Nanoprimer transiently modulates this natural clearance, allowing more drug to circulate longer, reach its intended target, and reduce off-target liver exposure.This approach can both enhance the performance of existing therapies and unlock new therapeutic pathways that were previously not achievable.

Developed as a therapeutic companion platform, Nanobiotix is advancing this technology through strategic external collaborations alongside a proprietary internal pipeline of Nanoprimer-enabled assets.

(Press release, Nanobiotix, APR 20, 2026, View Source [SID1234664544])

FDA Grants Priority Review for KEYTRUDA® (pembrolizumab) and KEYTRUDA QLEX™ (pembrolizumab and berahyaluronidase alfa-pmph), Each with Padcev® (enfortumab vedotin-ejfv), for Cisplatin-Eligible Patients with Muscle-Invasive Bladder Cancer

On April 20, 2026 Merck (NYSE: MRK), known as MSD outside of the United States and Canada, reported that the U.S. Food and Drug Administration (FDA) granted priority review for two supplemental Biologics License Applications (sBLA) for KEYTRUDA (pembrolizumab) and KEYTRUDA QLEX (pembrolizumab and berahyaluronidase alfa-pmph), Merck’s anti-PD-1 therapy, each in combination with Padcev (enfortumab vedotin-ejfv), for the treatment of patients with muscle-invasive bladder cancer (MIBC) who are eligible for cisplatin-based chemotherapy. The FDA set a Prescription Drug User Fee Act (PDUFA), or target action, date of August 17, 2026.

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If approved, these indications would expand the use of KEYTRUDA and KEYTRUDA QLEX, each in combination with Padcev, as the first perioperative treatments for patients with MIBC regardless of cisplatin eligibility and build on the previously approved indications of the combination for the treatment of patients with MIBC who are ineligible for cisplatin-based chemotherapy.

"Results from KEYNOTE-B15 challenge long-held expectations for patients with muscle-invasive bladder cancer," said Dr. Marjorie Green, senior vice president and head of oncology, global clinical development, Merck Research Laboratories. "Even with curative-intent surgery and chemotherapy, patients still experience disease progression or limited survival. These data add to the growing body of evidence demonstrating that KEYTRUDA and KEYTRUDA QLEX, each in combination with Padcev, have the potential to reshape how we approach treatment for these patients and improve outcomes for people facing this aggressive disease."

The sBLAs are based on data from the Phase 3 KEYNOTE-B15 trial (also known as EV-304), which was conducted in collaboration with Pfizer and Astellas, which were presented at the recent American Society of Clinical Oncology (ASCO) (Free ASCO Whitepaper) Genitourinary (GU) Cancers Symposium. The companies plan to share these results with regulatory authorities worldwide for potential regulatory filings.

KEYTRUDA plus Padcev is currently approved for the treatment of adult patients with locally advanced or metastatic urothelial cancer (la/mUC) in the U.S., the European Union (EU), Japan and several other countries around the world. KEYTRUDA plus Padcev is also approved in the U.S. for the treatment of adult patients with MIBC who are ineligible for cisplatin-based chemotherapy. KEYTRUDA as a monotherapy is also approved in the U.S., EU, Japan and other countries for the treatment of certain patients with la/mUC or a type of non-muscle-invasive bladder cancer (NMIBC).

KEYTRUDA plus Padcev has now demonstrated an overall survival (OS) benefit across three Phase 3 trials in bladder cancer. In addition to KEYNOTE-B15, these trials include previously announced positive results from KEYNOTE-905 for the treatment of patients with MIBC who are not eligible for or declined cisplatin-based chemotherapy and positive results from KEYNOTE-A39 for the treatment of adult patients with la/mUC. Three additional Phase 3 studies are currently evaluating KEYTRUDA across all stages of bladder cancer, including non-muscle-invasive, muscle-invasive and metastatic disease. Two of these studies are in MIBC including KEYNOTE-866 (NCT03924856) and KEYNOTE-992 (NCT04241185). KEYTRUDA is also being evaluated in combination with Bacillus Calmette-Guerin (BCG) in patients with NMIBC in KEYNOTE-676 (NCT03711032).

KEYNOTE-B15 is one of six Phase 3 studies of a KEYTRUDA-based regimen in an earlier stage of cancer to demonstrate an OS benefit. It was also the 15th positive pivotal trial for a KEYTRUDA-based regimen in earlier-stage cancers.

About KEYNOTE-B15/EV-304

KEYNOTE-B15, also known as EV-304, is an open-label, randomized Phase 3 trial (ClinicalTrials.gov, NCT04700124) evaluating perioperative KEYTRUDA in combination with Padcev and surgery (radical cystectomy and pelvic lymph node dissection) versus neoadjuvant chemotherapy (gemcitabine plus cisplatin) and surgery in patients with MIBC who are cisplatin-eligible. The trial enrolled 808 patients who were randomized to receive either:

Four cycles (each cycle length is 21 days) of neoadjuvant KEYTRUDA intravenous (IV) infusion plus enfortumab vedotin IV infusion, followed by surgery, followed by 13 cycles of adjuvant KEYTRUDA IV infusion plus five cycles of enfortumab vedotin IV infusion, or;
Four cycles (each cycle is 21 days) of standard of care neoadjuvant chemotherapy followed by surgery.
The primary endpoint is event-free survival (EFS), defined as the time from randomization to the first occurrence of the following events: radiographic disease progression precluding radical cystectomy and pelvic lymph node dissection, failure to undergo surgery in participants with residual disease, gross residual disease left behind at time of surgery, local or distant recurrence based on blinded independent central review or death due to any cause. The key secondary endpoints are OS and pathologic complete response rate.

About bladder cancer

In 2022, bladder cancer changed the lives of more than 600,000 people around the world. According to some clinical practice guidelines, about 25% of newly diagnosed bladder cancer cases are MIBC. The standard of care for patients with MIBC has been neoadjuvant cisplatin-based chemotherapy followed by surgery, which is shown to prolong survival. However, nearly half of patients who undergo this standard treatment experience recurrence.

(Press release, Merck & Co, APR 20, 2026, View Source [SID1234664543])