Mar 04, 2025
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Precigen, Inc. announced that the FDA has accepted its Biologics License Application (BLA) for PRGN-2012 (zopapogene imadenovec†), an investigational AdenoVerse gene therapy for adults with recurrent respiratory papillomatosis (RRP). The FDA granted priority review, setting a target action date of August 27, 2025. No advisory committee meeting is currently planned for the application.
If approved, PRGN-2012 would become the first and only FDA-approved therapy for RRP, a rare and lifelong neoplastic disease caused by HPV 6 or HPV 11, which currently requires repeated surgeries. Data from the pivotal Phase I/II study, presented at ASCO 2024 and published in The Lancet Respiratory Medicine, showed that over 50% of patients achieved Complete Response, while 85% experienced a reduction in surgical interventions compared to the previous year. The therapy has also received Breakthrough Therapy, Orphan Drug, and Accelerated Approval Designations from the FDA.
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“The priority review designation is a testament to the FDA’s recognition of the significant unmet need for the RRP patient population,” said Helen Sabzevari, PhD, President and CEO of Precigen. “Treatment with PRGN-2012 has shown significant, durable clinical benefit. We have patients treated with PRGN-2012 who have been surgery-free for more than three years now, bringing hope for an alternative to the cycle of repeated surgeries, which carry immense risk for irreversible damage and significant morbidity.”
She added, “We look forward to working with the FDA over the coming months during their BLA review and hope to introduce the first FDA-approved therapeutic option to the RRP patient population, estimated at more than 27,000 adults in the US, later this year.”
The FDA has granted Fast Track designation to Pyxis Oncology’s antibody-drug conjugate (ADC), PYX-201, for the treatment of adults with recurrent or metastatic head and neck squamous cell carcinoma (R/M HNSCC). The therapy is intended for patients whose disease has progressed after treatment with platinum-based chemotherapy and an anti-PD-L antibody.
“Receiving Fast Track designation for PYX-201 from the FDA marks a significant milestone for Pyxis Oncology, recognizing our potential to address the significant medical need in R/M HNSCC,” said Lara Sullivan, President and CEO of Pyxis Oncology.
PYX-201 targets Extradomain-B Fibronectin (EDB+FN), a structural component in the tumor extracellular matrix, and is currently being evaluated in two clinical trials. The PYX-201-101 study is assessing PYX-201 as a single-agent therapy, while the PYX-201-102 trial, conducted in partnership with MSD, is evaluating the ADC in combination with Keytruda (pembrolizumab) for patients with R/M HNSCC and other advanced solid tumors.
“This designation underscores the urgency of bringing differentiated treatment options to patients and will help accelerate the development of PYX-201 as we recruit patients for our trial,” Sullivan added. “We look forward to working with the FDA to advance this promising therapy as efficiently as possible.”
DeFloria, Inc., a collaboration between Ajna BioSciences PBC and Charlotte’s Web Holdings, Inc., has announced that the FDA has approved its Investigational New Drug (IND) application for AJA001, a multi-cannabinoid botanical drug. This clearance allows the company to proceed with a Phase II clinical trial evaluating AJA001 as a treatment for behavioral symptoms associated with autism spectrum disorder (ASD). The study is set to begin by mid-2025.
“IND submission for AJA001 and the initiation of the Phase II study in the US mark a historic moment in our mission to develop a systemically absorbed botanical drug therapy that meets FDA guidelines,” said Jared Stanley, CEO of DeFloria. “DeFloria is committed to supporting the ASD community and developing AJA001 to treat the behavioral symptoms associated with ASD with a drug that is designed to provide broad therapeutic efficacy.”
AJA001 was developed using Charlotte’s Web’s decade of research, proprietary hemp genetics, and innovative cultivation processes. This botanical drug aims to provide a safer alternative to the two currently approved ASD treatments—atypical antipsychotics, which often cause adverse side effects and have poor patient compliance. The Phase II trial will enroll 60 adolescent and young adult patients (ages 13-29 years old) in an open-label, 12-week study to assess dosage and efficacy signals for future Phase III trials.
“For more than 15 years, my family and our team have been laying the foundation for this botanical drug,” said Joel Stanley, CEO of Ajna BioSciences and Chairman of DeFloria. “Now, with this Phase II milestone, we’re one step closer to delivering a potential treatment option for autism spectrum disorder, a condition for which few effective therapies exist despite impacting millions worldwide.”
Laekna has announced that the FDA has approved its Investigational New Drug (IND) application for LAE120, an internally developed USP1 inhibitor for the treatment of advanced solid tumors. LAE120 is a highly potent, allosteric USP1 inhibitor that has shown strong monotherapy activity and synergy with PARP inhibitors in HRD (homologous recombination deficiency) cancers. It has demonstrated robust tumor inhibition across multiple xenograft models and a favorable safety profile in preclinical toxicology studies.
“Leveraging our deep expertise in drug discovery, Laekna has built an innovative portfolio through the collaboration of our Med Chem, Biology, and AI-driven Drug Discovery (AIDD) teams,” said Dr. Justin Gu, Chief Scientific Officer of Laekna. “We are committed to accelerating the development of novel drugs to bring transformative therapies to patients as swiftly as possible.”
In addition to LAE120, Laekna is advancing LAE118, a potentially best-in-class, mutant-selective PI3Kα inhibitor, which has entered IND-enabling studies. PI3Kα mutations are commonly found in breast, colorectal, lung, and endometrial cancers, but first-generation inhibitors lack selectivity, leading to tolerability concerns. LAE118 has demonstrated superior potency and selectivity against PI3Kα mutants, positioning it as a potential breakthrough therapy.
Laekna presented preclinical data on LAE118 at the San Antonio Breast Cancer Symposium (SABCS) in December 2024 and plans to submit its IND application in Q4 2025. The company continues to actively explore strategic partnerships to accelerate clinical development and expand its innovative oncology pipeline.
Corcept Therapeutics Incorporated has announced that the FDA has officially filed its New Drug Application (NDA) for relacorilant, a selective cortisol modulator intended to treat endogenous hypercortisolism (Cushing’s syndrome). The submission is supported by positive results from the pivotal GRACE trial and additional confirmatory evidence from the Phase III GRADIENT trial, a long-term extension trial, and a Phase II study.
“The FDA’s acceptance of our New Drug Application takes us another step closer to bringing relacorilant to patients with hypercortisolism,” said Joseph Belanoff, M.D., Chief Executive Officer of Corcept. “Relacorilant’s combination of efficacy and safety gives it the potential to become the new standard of care for patients struggling with the devastating impact of this disease.”
Patients treated with relacorilant in clinical trials showed improvements across multiple symptoms of hypercortisolism while avoiding serious adverse effects such as adrenal insufficiency, hypokalemia, and QT prolongation, which are concerns with currently approved treatments. Unlike existing therapies, relacorilant does not exhibit progesterone receptor-related side effects, such as endometrial thickening or vaginal bleeding.
With this regulatory milestone, Corcept moves one step closer to providing a safer and more effective treatment for patients suffering from Cushing’s syndrome, a rare and challenging endocrine disorder.
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