Genprex (NASDAQ:GNPX), Inc. (GNPX), a clinical-stage gene therapy company focused on developing life-changing therapies for patients with cancer and diabetes, today provides a review of its 2023 achievements and a preview of plans for advancing its diabetes and oncology gene therapy programs in 2024.
"We are very proud of what we accomplished in 2023, particularly with the successful completion of the Phase 1 portion of our Acclaim-1 clinical trial in lung cancer," said
Oncology Gene Therapy Platform
Genprex's oncology program utilizes its systemic, non-viral Oncoprex® Nanoparticle Delivery System which encapsulates the gene-expressing plasmids using lipid nanoparticles. The resultant product is administered intravenously, where it is taken up by tumor cells that then express tumor suppressor proteins that were deficient in the tumor. The Company's lead product candidate, REQORSA (quaratusugene ozeplasmid), is being evaluated in three clinical trials as a treatment for non-small cell lung cancer ("NSCLC") and small cell lung cancer ("SCLC"), and each of the three lung cancer clinical programs has received a Fast Track Designation from the FDA for the treatment of that patient population.
REQORSA has a multimodal mechanism of action whereby it interrupts cell signaling pathways that cause replication and proliferation of cancer cells, re-establishes pathways for apoptosis (programmed cell death) in cancer cells, and modulates the immune response against cancer cells. In early studies, REQORSA has been shown to be complementary with targeted drugs and immunotherapies. Genprex's strategy is to develop REQORSA in combination with currently approved therapies and believes that REQORSA's unique attributes position it to provide treatments that improve on these current therapies for patients with NSCLC, SCLC, and possibly other cancers.
Important events of the year included:
- In April, at the 2023 Annual Meeting of the American Association for Cancer Research (AACR 2023), Genprex collaborators presented preclinincal gene therapy data with NPRL2, another tumor suppressor gene, that further validates the ONCOPREX Nanoparticle Delivery System as a platform
- In October, Genprex hosted a Key Opinion Leader virtual event, "Bringing Gene Therapy to the Fight Against Lung Cancers" which discussed the use of gene therapies, including REQORSA, in the fight against lung cancer
- In December, Genprex completed the successful production of a new batch of REQORSA thereby securing REQORSA supply for its Acclaim clinical studies
- Collaborators submitted abstracts in 2023 and are expecting to present data at the American Association for Cancer Research (AACR) Meeting in
April 2024
Acclaim-1:
The Acclaim-1 study is a Phase 1/2 clinical trial that has three portions - a Phase 1 dose escalation which has been completed, a Phase 2a expansion, and a Phase 2b randomized portion. Acclaim-1 uses a combination of REQORSA and AstraZeneca's Tagrisso® in patients with late-stage NSCLC that has activating epidermal growth factor receptor mutations and progression after treatment with Tagrisso. This novel approach to targeting lung cancer has demonstrated a strong safety profile with early signs of efficacy.
- In May, Genprex completed the Phase 1 portion of the Acclaim-1 clinical trial and reported encouraging results. The Acclaim-1 Phase 1 study had no Dose Limiting Toxicities and results established a Phase 2 Recommended Dose, as well as provided data showing efficacy of REQORSA in combination with Tagrisso®
- In May, after completion of the Phase 1 portion of the Acclaim-1 trial, the Safety Review Committee ("SRC") approved advancement from the Phase 1 dose escalation portion of the trial to the Phase 2a expansion portion of the trial
- In October, clinical collaborators presented a poster presentation at the 2023 AACR-NCI-EORTC International Conference on Molecular Targets and Cancer Therapeutics showing the Phase 1 results of the Acclaim-1 study
- In
January 2024 , Genprex expects to open the Phase 2a expansion portion of the Acclaim-1 study for enrollment - Genprex expects to complete the enrollment of 19 patients in each cohort of the Phase 2a expansion portion of the study by the end of 2024
Acclaim-2:
The Acclaim-2 study is a Phase 1/2 clinical trial that has three portions - a Phase 1 dose escalation portion, a Phase 2a expansion portion, and a Phase 2b randomized portion. The Acclaim-2 trial uses a combination of REQORSA and Merck & Co.'s Keytruda® in patients with late-stage NSCLC whose disease has progressed after treatment with Keytruda. Patients are treated at the 0.06 mg/kg dose level in the first cohort of patients and, subject to the Acclaim-2 SRC approval, will be treated at successive dose levels of 0.09 mg/kg and 0.12 mg/kg.
- Expanding on the previously granted patents in the
U.S. ,Japan ,Mexico andRussia , Genprex was granted patents inAustralia ,Chile andChina to cover the use of REQORSA in combination with immune checkpoint inhibtors, e.g., PD1 and PDL1 inhibitors. These patents are applicable to Genprex's Acclaim-2 and Acclaim-3 clinical trials. - In the second half of 2024, Genprex expects to complete enrollment in the Phase 1 dose escalation portion of the Acclaim-2 study
Acclaim-3:
The Acclaim-3 study has two portions - a Phase 1 dose escalation portion and a Phase 2 expansion portion. In
- In June, the FDA granted Fast Track Designation for the Acclaim-3 treatment combination of REQORSA and Tecentriq as maintenance therapy in patients with ES-SCLC who did not develop tumor progression after receiving Tecentriq and chemotherapy as initial standard treatment
- In August, the FDA granted Orphan Drug Designation to REQORSA for the treatment of SCLC
- In
January 2024 , Genprex expects to open the Phase 1 portion of the Acclaim-3 study for enrollment, and expects to complete the Phase 1 portion of the study by the second half of 2024 - In the second half of 2024, Genprex expects to start the Phase 2 portion of the Acclaim-3 study
Diabetes Gene Therapy Platform
Genprex's diabetes gene therapy approach is comprised of an infusion process that uses an adeno-associated virus ("AAV") vector to deliver Pdx1 and MafA genes directly to the pancreas. In models of Type 1 diabetes, GPX-002 transforms alpha cells in the pancreas into functional beta-like cells, which can produce insulin but may be distinct enough from beta cells to evade the body's immune system. In a similar approach, GPX-003 for Type 2 diabetes, where autoimmunity is not at play, is believed to rejuvenate and replenish exhausted beta cells. Genprex has exclusively licensed from the University of Pittsburgh of the Commonwealth System of Higher Education ("University of Pittsburgh") multiple technologies relating to the development of a gene therapy product for each of Type 1 and Type 2 diabetes. In
- In February, Genprex's research collaborators at the University of Pittsburgh presented preclinical data in a NHP model of Type 1 diabetes highlighting the therapeutic potential of GPX-002. These data, presented during an oral presentation at the 16th International Conference on Advanced Technologies & Treatments for Diabetes (ATTD 2023), showed statistically significant decreases in insulin requirements, increased c-peptide levels and improved glucose tolerance compared to baseline.
- In April, the Company hosted a Key Opinion Leader virtual event, "Novel Gene Therapy to Treat Type 1 Diabetes," which discussed preclinical data reported at ATTD 2023 supporting gene therapy to treat Ttype 1 diabetes
- Finalized the components of the diabetes construct to take forward for nonclinical studies
- In December, Genprex submitted a request to meet with the FDA to obtain their guidance on the nonclinical studies needed to file an Investigational New Drug application and initiate first-in-human studies. As a result of the FDA's response, the Company will continue with its planned additional nonclinical studies before requesting regulatory guidance in 2024 for the IND-enabling studies.