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])

On April 20, 2026 Immunofoco reported the presentation of new preclinical data for its in vivo BCMA-targeting CAR-T candidate, IMV102, at the AACR (Free AACR Whitepaper) Annual Meeting 2026. The data demonstrate that IMV102 achieved potent and durable anti-tumor activity in multiple myeloma models, highlighting its potential to address key limitations of conventional CAR-T therapies.

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Autologous CAR-T therapy has achieved remarkable success in hematologic malignancies, yet its widespread use remains challenging due to complex manufacturing and high costs. To address these challenges, Immunofoco developed the iMAGIC platform, a lentiviral vector-based in vivo CAR-T system composed of a mutated MxV glycoprotein (MxV-G-mut) and a T cell targeting module (TCM3). This platform enables selective targeting and transduction of T cells in vivo.

Leveraging this platform, the company is advancing IMV102, a BCMA-targeting in vivo CAR-T candidate that has demonstrated promising specificity, efficacy, and safety in preclinical models of multiple myeloma.

In vitro, IMV102 showed highly selective transduction of T cells, achieving efficient gene delivery in Jurkat T-cell lines, while minimal transduction was observed in non-target cells such as hepatocytes and Kupffer cells, indicating strong targeting specificity. Furthermore, IMV102-generated CAR-T cells exhibited potent cytotoxic activity against NCI-H929 multiple myeloma cells, accompanied by significant upregulation of IFN-γ.

In vivo, IMV102 induced CAR-T cell generation and achieved potent and durable tumor inhibition in two multiple myeloma xenograft models (H929-Luc and MM.1S-Luc, human PBMC-reconstituted mice). Tumor burden was significantly reduced. Body weight remained stable throughout the study, with no significant safety signals observed. CAR-T cell expansion and plasma IFN-γ levels remained within a manageable range, supporting a favorable balance between immune activity and safety.

Dr. Minmin Sun, Founder, Chairman, and CEO of Immunofoco, commented: "IMV102 further validates the potential of our dual-engine strategy integrating ex vivo and in vivo CAR-T approaches. We believe in vivo CAR-T has the potential to transform the manufacturing and delivery paradigm of cell therapies, enabling a shift from highly personalized treatments to scalable and accessible solutions. We will continue to advance IMV102 into clinical development and expand the application of the iMAGIC platform across oncology and autoimmune diseases."

(Press release, Immunofoco, APR 20, 2026, View Source [SID1234664565])

On April 20, 2026 Orion Corporation (Orion Pharma) reported that its investigational drug ODM-212 has received Orphan Drug Designation (ODD) from the US Food and Drug Administration (FDA) for the treatment of mesothelioma, which is a rare and difficult to treat cancer. The FDA grants orphan drug designation to investigational therapies addressing rare diseases or conditions that affect fewer than 200,000 people in the US.

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ODM-212 is an oral small-molecule pan-TEAD (Transcriptional Enhanced Associate Domain) inhibitor being tested in a Phase 2 clinical study (TEADES) for the treatment of malignant pleural mesothelioma (MPM), epithelioid hemangioendothelioma (EHE) and other solid tumors with dysfunction in Hippo pathway. The trial includes patients who have progressed after receiving standard treatments and have no further treatment options. The primary endpoints of the study are safety and tolerability with secondary endpoints including Overall Response Rate, Progression Free Survival and Overall Survival. This is a global trial conducted at leading oncology centers in the US and Europe.

"Receiving orphan drug designation for mesothelioma is an important milestone for the ODM-212 program. It underscores the importance of developing urgently needed innovative therapies for patients living with mesothelioma" said Praveen Aanur, MD, MPH, MBA, Chief Medical Officer, Oncology Therapy Area, Orion Pharma.

About Orphan Drug Designation
Orphan Drug Designation is granted by FDA to therapies intended to prevent, diagnose, or treat rare diseases or conditions. With this designation for ODM-212, Orion Pharma, the sponsor, is now qualified for incentives including tax credits, exepmption from user fees, and eligibility for a 7-year period of market exclusivity following approval. Orphan Drug Designation neither shortens the development time or regulatory review time of a drug nor gives the drug any advantage in the regulatory review or approval process.

About ODM-212
ODM-212 is an oral small-molecule pan-TEAD (Transcriptional Enhanced Associate Domain) inhibitor developed by Orion Pharma. It targets the Hippo signaling pathway, which regulates cell growth and organ size. Dysregulation of this pathway—particularly through YAP/TAZ activation—can lead to uncontrolled tumor growth and resistance to cancer therapies. ODM-212 works by blocking TEAD transcription factors, disrupting YAP-TEAD protein-protein interactions, and inhibiting TEAD auto-palmitoylation, which is essential for TEAD activity.

(Press release, Orion, APR 20, 2026, View Source [SID1234664582])

On April 20, 2026 Allogene Therapeutics, Inc. (Nasdaq: ALLO), a clinical-stage biotechnology company pioneering the development of allogeneic CAR T (AlloCAR T) products, reported upcoming presentations at the American Association for Cancer Research (AACR) (Free AACR Whitepaper) Annual Meeting, April 17-22, 2026, in San Diego, Calif.

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Allogene’s presentations at AACR (Free AACR Whitepaper) highlight the potential of allogeneic CAR T to expand access, simplify delivery, and enable broader application of cell therapy across diseases when its inherent advantages are fully leveraged. The Company will participate in several AACR (Free AACR Whitepaper) 2026 scientific forums, including:

Poster: Preclinical Evaluation of Allogeneic BCMA/CD70 Dual CAR T Cells for High-Risk Multiple Myeloma
Presenter: Mark K. O’Dair, PhD, Allogene
Session Title: CAR T Cell Targets and TME Reprogramming
Poster Number: 1535
Location: Poster Section 7, Board 17
Session Date and Time: Monday, April 20, 9:00am-12:00pm PT

Based on its foundational work in oncology, Allogene is extending its Dagger technology into autoimmune disease, applying its gene-edited, dual-targeting CAR T approach to target both BCMA and CD70 on malignant plasma cells as well as selectively eliminate alloreactive immune cells to promote durable persistence with reduced need for chemotherapy-based lymphodepletion. This strategy builds on the Company’s expertise in allogeneic cell therapy in advanced renal cell carcinoma and is designed to deliver a readily available, off-the-shelf treatment with the potential to transform how both cancer and autoimmune diseases are treated.

Major Symposium: Off-the-Shelf Cell Therapies for Cancer and Beyond
Presenter: David Chang, M.D., Ph.D., President, CEO and Co-Founder, Allogene Therapeutics
Title: Allogeneic CAR-T: Science at Scale (SY13-03)
Location: Room 28 – Upper Level – Convention Center
Session Date and Time: Tuesday, April 21, 12:30-2:00pm PT

The future of CAR T will be defined by its ability to reach more patients, more reliably and earlier in the course of disease. Allogene’s allogeneic approach is designed to unlock this potential across five key dimensions: speed, with on-demand availability; safety, manageable across care settings; simplicity, as a one-time outpatient treatment; scalability, enabling broad patient access; and survival, with the potential to deliver meaningful clinical outcomes. Together, these attributes represent a path toward making CAR T a more practical and widely accessible therapy.

Forum: Cell Therapy at a Crossroads: Exploring the Evolving Landscape between Autologous, Allogeneic, and In Vivo Engineering (Session: FO06)
Presenter: Zachary Roberts, M.D., Ph.D., EVP, Research and Development, CMO, Allogene Therapeutics
Location: Ballroom 20 AB – Upper Level – Convention Center
Session Date and Time: Tuesday, April 21, 5:00pm-6:30pm PT

Allogeneic CAR T therapy represents a major step forward in the evolution from autologous products. With extensive experience across both hematologic and solid tumors, Allogene has been a singular leader since the earliest days of allogeneic cell therapy and has defined a path through many of the scientific challenges the field has faced. As key trials advance Allogene is positioning allogeneic CAR T as a bridge to biologic-like manufacturing scale necessary to address an ever-growing patient demand for these life-saving products.

(Press release, Allogene, APR 20, 2026, View Source [SID1234664533])

On April 20, 2026 PharmaMar (MSE:PHM) reported that the Australian Therapeutic Goods Administration (TGA) and the Health Sciences Authority (HSA) in Singapore have granted approval for Zepzelca (lurbinectedin) in combination with atezolizumab (Tecentriq) as a first line maintenance treatment for adults with extensive-stage small cell lung cancer (ES-SCLC), whose disease has not progressed after first-line induction therapy with atezolizumab, carboplatin and etoposide.

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The approvals are based on the results from the Phase 3 IMforte[i]trial.

PharmaMar’s partner in Australia and Singapore, Specialised Therapeutics Asia Pte, Ltd (STA), will market and commercialize the product.

These approvals were obtained under the Orbis Project, an initiative of the U.S. Food and Drug Administration’s (FDA) Oncology Center of Excellence, designed to provide a framework for concurrent submission and review of oncology products among international partners. As of today, this treatment is approved in a total of 13 countries: the US, Switzerland, the United Arab Emirates, Oman, Uruguay, Peru, Paraguay, Ecuador, Israel, Taiwan, Australia, Singapore and Dominican Republic.

PharmaMar also received a recommendation for approval from the European Medicines Agency in March. The European Commission will now decide on the marketing authorization in accordance with the established procedure.

(Press release, PharmaMar, APR 20, 2026, View Source [SID1234664549])